Hematology Times

Antihemophilic factor

In a survey of 200 adults with hemophilia A, most were not aware that factor VIII (FVIII) products can be stored at room temperature.

A majority of patients surveyed thought FVIII products must be stored in the refrigerator at all times.

The minority of

patients who did store FVIII at room temperature felt significantly

more satisfied with their treatment and less restricted in their daily

lives than patients who refrigerated FVIII.

“People living with hemophilia A can feel restricted in their daily life based on the misconception that all FVIII treatments must be stored in the refrigerator,” said Mariasanta Napolitano, MD, of the University of Palermo in Italy.

“It is important that healthcare professionals communicate to patients that some FVIII products can be used portably and at room temperature, to enable them to go about their daily lives as actively as they wish.”

Dr Napolitano and her colleagues presented the results of this survey at the 10th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD, poster P024).

The researchers conducted the survey in adults with hemophilia A in 7 countries. Face-to-face surveys were conducted with 90 people in the European Union (France, Italy, and UK) and 90 people in Latin America (Argentina, Brazil, and Mexico). Online surveys were conducted with 20 people in Japan.

The questionnaire covered current use of FVIII products, patient satisfaction, and restrictions in daily life using verbalized rating scales (eg, “not restricted at all / somewhat restricted / restricted / strongly restricted”).

Seventy-four percent of patients used FVII as prophylaxis, and 26% used on-demand treatment. Sixty-seven percent used recombinant FVIII, and 33% used plasma-derived FVIII.

Storage

Most patients—85%—stored their FVIII product in the refrigerator, with 15% storing their treatment at room temperature.

Of those storing FVIII in the refrigerator, 88% said FVIII must be stored in the refrigerator at all times. Seventy-nine percent of patients said they worry about the temperature at which FVIII is stored.

Seventy-four percent said they wait for their FVIII product to reach room temperature before mixing and injecting it. The median wait time was 10 minutes.

Sixty-two percent of patients said an injection with cold FVIII is more unpleasant than an injection with room temperature FVIII.

Satisfaction

Patients storing FVIII at room temperature were significantly more likely than those storing FVIII in the refrigerator to report overall satisfaction with their FVIII product (52% vs 28%, P<0.05).

Patients storing FVIII at room temperature were also significantly more likely to report satisfaction with:

• Product efficacy (61% vs 43%, P<0.05)
• The procedure of mixing the product before injection (39% vs 22%, P<0.05)
• The flexibility of storage depending on the patients’ needs (55% vs 18%, P<0.05)
• The length of time the product can be stored outside the refrigerator (45% vs 19%, P<0.05)
• The size of vials (42% vs 18%, P<0.05)
• Storage temperature (39% vs 15%, P<0.05).

Daily life

Patients storing FVIII at room temperature travelled significantly more often—16 times per year vs 9 times per year (P<0.01).

Nineteen percent of these patients felt that storing, preparing, and mixing FVIII is disruptive to the normal course of the day, compared to 35% of patients who store FVIII in the refrigerator (P<0.05).

Significantly fewer patients in the room-temperature group said they feel restricted when it comes to sports activities, traveling, and their evening routine (P<0.05).

Forty-eight percent of patients storing FVIII at room temperature said they feel relaxed using FVIII in their daily life, compared to 20% of patients storing FVIII in the refrigerator (P<0.05).

Antihemophilic factor

In a survey of 200 adults with hemophilia A, most were not aware that factor VIII (FVIII) products can be stored at room temperature.

A majority of patients surveyed thought FVIII products must be stored in the refrigerator at all times.

The minority of

patients who did store FVIII at room temperature felt significantly

more satisfied with their treatment and less restricted in their daily

lives than patients who refrigerated FVIII.

“People living with hemophilia A can feel restricted in their daily life based on the misconception that all FVIII treatments must be stored in the refrigerator,” said Mariasanta Napolitano, MD, of the University of Palermo in Italy.

“It is important that healthcare professionals communicate to patients that some FVIII products can be used portably and at room temperature, to enable them to go about their daily lives as actively as they wish.”

Dr Napolitano and her colleagues presented the results of this survey at the 10th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD, poster P024).

The researchers conducted the survey in adults with hemophilia A in 7 countries. Face-to-face surveys were conducted with 90 people in the European Union (France, Italy, and UK) and 90 people in Latin America (Argentina, Brazil, and Mexico). Online surveys were conducted with 20 people in Japan.

The questionnaire covered current use of FVIII products, patient satisfaction, and restrictions in daily life using verbalized rating scales (eg, “not restricted at all / somewhat restricted / restricted / strongly restricted”).

Seventy-four percent of patients used FVII as prophylaxis, and 26% used on-demand treatment. Sixty-seven percent used recombinant FVIII, and 33% used plasma-derived FVIII.

Storage

Most patients—85%—stored their FVIII product in the refrigerator, with 15% storing their treatment at room temperature.

Of those storing FVIII in the refrigerator, 88% said FVIII must be stored in the refrigerator at all times. Seventy-nine percent of patients said they worry about the temperature at which FVIII is stored.

Seventy-four percent said they wait for their FVIII product to reach room temperature before mixing and injecting it. The median wait time was 10 minutes.

Sixty-two percent of patients said an injection with cold FVIII is more unpleasant than an injection with room temperature FVIII.

Satisfaction

Patients storing FVIII at room temperature were significantly more likely than those storing FVIII in the refrigerator to report overall satisfaction with their FVIII product (52% vs 28%, P<0.05).

Patients storing FVIII at room temperature were also significantly more likely to report satisfaction with:

• Product efficacy (61% vs 43%, P<0.05)
• The procedure of mixing the product before injection (39% vs 22%, P<0.05)
• The flexibility of storage depending on the patients’ needs (55% vs 18%, P<0.05)
• The length of time the product can be stored outside the refrigerator (45% vs 19%, P<0.05)
• The size of vials (42% vs 18%, P<0.05)
• Storage temperature (39% vs 15%, P<0.05).

Daily life

Patients storing FVIII at room temperature travelled significantly more often—16 times per year vs 9 times per year (P<0.01).

Nineteen percent of these patients felt that storing, preparing, and mixing FVIII is disruptive to the normal course of the day, compared to 35% of patients who store FVIII in the refrigerator (P<0.05).

Significantly fewer patients in the room-temperature group said they feel restricted when it comes to sports activities, traveling, and their evening routine (P<0.05).

Forty-eight percent of patients storing FVIII at room temperature said they feel relaxed using FVIII in their daily life, compared to 20% of patients storing FVIII in the refrigerator (P<0.05).

Antihemophilic factor

In a survey of 200 adults with hemophilia A, most were not aware that factor VIII (FVIII) products can be stored at room temperature.

A majority of patients surveyed thought FVIII products must be stored in the refrigerator at all times.

The minority of

patients who did store FVIII at room temperature felt significantly

more satisfied with their treatment and less restricted in their daily

lives than patients who refrigerated FVIII.

“People living with hemophilia A can feel restricted in their daily life based on the misconception that all FVIII treatments must be stored in the refrigerator,” said Mariasanta Napolitano, MD, of the University of Palermo in Italy.

“It is important that healthcare professionals communicate to patients that some FVIII products can be used portably and at room temperature, to enable them to go about their daily lives as actively as they wish.”

Dr Napolitano and her colleagues presented the results of this survey at the 10th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD, poster P024).

The researchers conducted the survey in adults with hemophilia A in 7 countries. Face-to-face surveys were conducted with 90 people in the European Union (France, Italy, and UK) and 90 people in Latin America (Argentina, Brazil, and Mexico). Online surveys were conducted with 20 people in Japan.

The questionnaire covered current use of FVIII products, patient satisfaction, and restrictions in daily life using verbalized rating scales (eg, “not restricted at all / somewhat restricted / restricted / strongly restricted”).

Seventy-four percent of patients used FVII as prophylaxis, and 26% used on-demand treatment. Sixty-seven percent used recombinant FVIII, and 33% used plasma-derived FVIII.

Storage

Most patients—85%—stored their FVIII product in the refrigerator, with 15% storing their treatment at room temperature.

Of those storing FVIII in the refrigerator, 88% said FVIII must be stored in the refrigerator at all times. Seventy-nine percent of patients said they worry about the temperature at which FVIII is stored.

Seventy-four percent said they wait for their FVIII product to reach room temperature before mixing and injecting it. The median wait time was 10 minutes.

Sixty-two percent of patients said an injection with cold FVIII is more unpleasant than an injection with room temperature FVIII.

Satisfaction

Patients storing FVIII at room temperature were significantly more likely than those storing FVIII in the refrigerator to report overall satisfaction with their FVIII product (52% vs 28%, P<0.05).

Patients storing FVIII at room temperature were also significantly more likely to report satisfaction with:

• Product efficacy (61% vs 43%, P<0.05)
• The procedure of mixing the product before injection (39% vs 22%, P<0.05)
• The flexibility of storage depending on the patients’ needs (55% vs 18%, P<0.05)
• The length of time the product can be stored outside the refrigerator (45% vs 19%, P<0.05)
• The size of vials (42% vs 18%, P<0.05)
• Storage temperature (39% vs 15%, P<0.05).

Daily life

Patients storing FVIII at room temperature travelled significantly more often—16 times per year vs 9 times per year (P<0.01).

Nineteen percent of these patients felt that storing, preparing, and mixing FVIII is disruptive to the normal course of the day, compared to 35% of patients who store FVIII in the refrigerator (P<0.05).

Significantly fewer patients in the room-temperature group said they feel restricted when it comes to sports activities, traveling, and their evening routine (P<0.05).

Forty-eight percent of patients storing FVIII at room temperature said they feel relaxed using FVIII in their daily life, compared to 20% of patients storing FVIII in the refrigerator (P<0.05).

Prescription pills

New research published in Blood suggests young patients with acute lymphoblastic leukemia (ALL)—and their parents—are likely to over-report treatment adherence.

In a study of 416 pediatric ALL patients, 84% of patients or their parents said the patients took more 6-mercaptopurine (6MP) than they actually did.

6MP is prescribed for 2 years after chemotherapy with the goal of producing durable remissions, but studies show that over 95% of the prescribed doses must be taken for the medication to be effective.

“Because this therapy is administered orally every day, we cannot supervise our patients to make sure they are taking their pills,” said study author Smita Bhatia, MD, of the University of Alabama at Birmingham.

“Findings from this study suggest that we need better ways to monitor intake of medications as prescribed.”

This study included 416 pediatric ALL patients followed over 4 months. Patients received prescription pill bottles for their 6MP that contained a microprocessor chip in the cap to log the date and time the bottle was opened.

The logs were compared to self-report questionnaires completed by patients or their parents and provided to physicians at monthly check-ups.

Patients were classified as:

• Perfect reporters—self-report corresponded to data from the Medication Event Monitoring System (MEMS)
• Over-reporters—self-report was greater than MEMS data by more than 5 days per month for more than 50% of the study months
• Others—all patients not meeting criteria for perfect- or over-reporter.

Twelve percent of patients were classified as perfect reporters, 23.6% were over-reporters, 0.5% were under-reporters, and 63.9% were classified as “others.”

Self-reported 6MP intake exceeded MEMS data at least some of the time in 84% of patients.

“We observed that there was an inverse relationship between over-reporting and the extent of non-adherence,” Dr Bhatia noted.

Of the patients who adhered to their regimens (defined as taking their prescribed dose 95% of the time), a small percentage (8%) over-reported their intake.

However, among those found to be non-adherent, a substantially larger percent (47%) over-reported.

In order to find out why patients were not taking their prescribed medication, study author Wendy Landier, PhD, RN, also of the University of Alabama at Birmingham, organized interviews with patients and their parents.

In these interviews, Dr Landier noted that forgetfulness was the primary reason for non-adherence.

“We found that partnering with a parent was very conducive to our patients’ adherence, playing a key role in combatting forgetfulness,” she said.

Recognizing the importance of parental involvement in patient adherence, the researchers were inspired to conduct further research to make parents more involved.

In a trial that builds on this study, physicians send personalized text messages to both patients and their parents at a prescribed time reminding them to take their medicine. Both patient and parent then report back, via text, that the indicated dose was taken.

“Our first study showed that non-adherence was prevalent, increasing the risk of relapse,” Dr Bhatia said. “We then found that parental vigilance was an important facilitator to adherence, while forgetting to take the medication was the most commonly reported barrier.”

“We used these findings to inform the intervention trial to help families improve adherence to treatment. We hope that this will be a real step in improving outcomes in children with leukemia.”

Prescription pills

New research published in Blood suggests young patients with acute lymphoblastic leukemia (ALL)—and their parents—are likely to over-report treatment adherence.

In a study of 416 pediatric ALL patients, 84% of patients or their parents said the patients took more 6-mercaptopurine (6MP) than they actually did.

6MP is prescribed for 2 years after chemotherapy with the goal of producing durable remissions, but studies show that over 95% of the prescribed doses must be taken for the medication to be effective.

“Because this therapy is administered orally every day, we cannot supervise our patients to make sure they are taking their pills,” said study author Smita Bhatia, MD, of the University of Alabama at Birmingham.

“Findings from this study suggest that we need better ways to monitor intake of medications as prescribed.”

This study included 416 pediatric ALL patients followed over 4 months. Patients received prescription pill bottles for their 6MP that contained a microprocessor chip in the cap to log the date and time the bottle was opened.

The logs were compared to self-report questionnaires completed by patients or their parents and provided to physicians at monthly check-ups.

Patients were classified as:

• Perfect reporters—self-report corresponded to data from the Medication Event Monitoring System (MEMS)
• Over-reporters—self-report was greater than MEMS data by more than 5 days per month for more than 50% of the study months
• Others—all patients not meeting criteria for perfect- or over-reporter.

Twelve percent of patients were classified as perfect reporters, 23.6% were over-reporters, 0.5% were under-reporters, and 63.9% were classified as “others.”

Self-reported 6MP intake exceeded MEMS data at least some of the time in 84% of patients.

“We observed that there was an inverse relationship between over-reporting and the extent of non-adherence,” Dr Bhatia noted.

Of the patients who adhered to their regimens (defined as taking their prescribed dose 95% of the time), a small percentage (8%) over-reported their intake.

However, among those found to be non-adherent, a substantially larger percent (47%) over-reported.

In order to find out why patients were not taking their prescribed medication, study author Wendy Landier, PhD, RN, also of the University of Alabama at Birmingham, organized interviews with patients and their parents.

In these interviews, Dr Landier noted that forgetfulness was the primary reason for non-adherence.

“We found that partnering with a parent was very conducive to our patients’ adherence, playing a key role in combatting forgetfulness,” she said.

Recognizing the importance of parental involvement in patient adherence, the researchers were inspired to conduct further research to make parents more involved.

In a trial that builds on this study, physicians send personalized text messages to both patients and their parents at a prescribed time reminding them to take their medicine. Both patient and parent then report back, via text, that the indicated dose was taken.

“Our first study showed that non-adherence was prevalent, increasing the risk of relapse,” Dr Bhatia said. “We then found that parental vigilance was an important facilitator to adherence, while forgetting to take the medication was the most commonly reported barrier.”

“We used these findings to inform the intervention trial to help families improve adherence to treatment. We hope that this will be a real step in improving outcomes in children with leukemia.”

Prescription pills

New research published in Blood suggests young patients with acute lymphoblastic leukemia (ALL)—and their parents—are likely to over-report treatment adherence.

In a study of 416 pediatric ALL patients, 84% of patients or their parents said the patients took more 6-mercaptopurine (6MP) than they actually did.

6MP is prescribed for 2 years after chemotherapy with the goal of producing durable remissions, but studies show that over 95% of the prescribed doses must be taken for the medication to be effective.

“Because this therapy is administered orally every day, we cannot supervise our patients to make sure they are taking their pills,” said study author Smita Bhatia, MD, of the University of Alabama at Birmingham.

“Findings from this study suggest that we need better ways to monitor intake of medications as prescribed.”

This study included 416 pediatric ALL patients followed over 4 months. Patients received prescription pill bottles for their 6MP that contained a microprocessor chip in the cap to log the date and time the bottle was opened.

The logs were compared to self-report questionnaires completed by patients or their parents and provided to physicians at monthly check-ups.

Patients were classified as:

• Perfect reporters—self-report corresponded to data from the Medication Event Monitoring System (MEMS)
• Over-reporters—self-report was greater than MEMS data by more than 5 days per month for more than 50% of the study months
• Others—all patients not meeting criteria for perfect- or over-reporter.

Twelve percent of patients were classified as perfect reporters, 23.6% were over-reporters, 0.5% were under-reporters, and 63.9% were classified as “others.”

Self-reported 6MP intake exceeded MEMS data at least some of the time in 84% of patients.

“We observed that there was an inverse relationship between over-reporting and the extent of non-adherence,” Dr Bhatia noted.

Of the patients who adhered to their regimens (defined as taking their prescribed dose 95% of the time), a small percentage (8%) over-reported their intake.

However, among those found to be non-adherent, a substantially larger percent (47%) over-reported.

In order to find out why patients were not taking their prescribed medication, study author Wendy Landier, PhD, RN, also of the University of Alabama at Birmingham, organized interviews with patients and their parents.

In these interviews, Dr Landier noted that forgetfulness was the primary reason for non-adherence.

“We found that partnering with a parent was very conducive to our patients’ adherence, playing a key role in combatting forgetfulness,” she said.

Recognizing the importance of parental involvement in patient adherence, the researchers were inspired to conduct further research to make parents more involved.

In a trial that builds on this study, physicians send personalized text messages to both patients and their parents at a prescribed time reminding them to take their medicine. Both patient and parent then report back, via text, that the indicated dose was taken.

“Our first study showed that non-adherence was prevalent, increasing the risk of relapse,” Dr Bhatia said. “We then found that parental vigilance was an important facilitator to adherence, while forgetting to take the medication was the most commonly reported barrier.”

“We used these findings to inform the intervention trial to help families improve adherence to treatment. We hope that this will be a real step in improving outcomes in children with leukemia.”

Huiqiang Huang, MD
Photo by Larry Young

SAN FRANCISCO—Interim results of a phase 3 trial suggest 2 treatment regimens may provide comparable efficacy in patients with extranodal natural killer/T-cell lymphoma (ENKTL), though 1 regimen appears more toxic than the other.

In this ongoing trial, investigators are comparing pegaspargase, gemcitabine, oxaliplatin, and thalidomide (P-Gemox+Thal) to pegaspargase, methotrexate, calcium folinate, and dexamethasone (AspaMetDex).

In some patients, either regimen may be followed by extensive involved-field radiotherapy (EIFRT) or autologous hematopoietic stem cell transplant (ASCT).

Thus far, P-Gemox+Thal and AspaMetDex have proven similarly effective for patients with newly diagnosed, stage I/II ENKTL.

And both regimens have produced unsatisfying survival outcomes in patients with advanced or relapsed/refractory ENKTL, according to investigator Huiqiang Huang, MD, of Sun Yat-sen Institute of Hematology in Guangzhou, China.

In addition, P-Gemox+Thal seems to be less toxic, overall, than AspaMetDex.

However, Dr Huang said it is still too early to draw any firm conclusions about these regimens. 

He presented results from this trial at the 9th Annual T-cell Lymphoma Forum. Results were previously presented at the 2016 ASH Annual Meeting (abstract 1819).

Dr Huang noted that AspaMetDex and SMILE (dexamethasone, methotrexate, ifosfamide, lL-asparaginase, and etoposide) are frequently administered to patients with ENKTL. And P-Gemox is recommended in the 2016 NCCN guidelines.

“But optimal regimens still have not been fully defined,” he said.

Therefore, he and his colleagues decided to compare AspaMetDex to P-Gemox+Thal in this non-inferiority trial, which has enrolled 110 patients from 12 centers in China.

Treatment

Fifty-six patients have been randomized to receive P-Gemox+Thal. Every 3 weeks, they received pegaspargase at 2000 U/m² on day 1, gemcitabine at 1000 mg/m² on days 1 and 8, and oxaliplatin at 130 mg/m² on days 1 and 8. They also received thalidomide at 100 mg every day for 1 year.

Fifty-four patients have been randomized to receive AspaMetDex. Every 3 weeks, they received pegaspargase at 2000 U/m² on day 1, methotrexate at 3000 mg/m² on day 1, calcium folinate at 30 mg every 6 hours until a safe serum methotrexate concentration was reached, and dexamethasone at 40 mg every day on days 1 to 4.

Newly diagnosed patients with stage I/II disease received either regimen as induction for a maximum of 4 cycles. Responders went on to receive EIFRT at 56 Gy in 28 fractions over 4 weeks.

Patients with newly diagnosed, stage III/IV ENKTL or relapsed/refractory ENKTL received either chemotherapy regimen for a maximum of 6 cycles. If they achieved a complete response (CR), these patients could proceed to ASCT.

Stage I/II ENKTL

Of the 63 stage I/II patients, 33 were randomized to P-Gemox+Thal, and 30 received AspaMetDex. In both arms, most patients were male (69.7% and 70%, respectively) and younger than 60 (78.8% and 90%, respectively).

Ninety-seven percent of patients in the P-Gemox+Thal arm had an ECOG status of 0-1, as did 100% of patients in the AspaMetDex arm.

The overall response rate (ORR) was 85.2% in the P-Gemox+Thal arm and 81.5% in the AspaMetDex arm. The CR rate was 59.3% in both arms. The rate of stable disease was 3.7% in the P-Gemox+Thal arm and 11.1% in the AspaMetDex arm.

After EIFRT, the ORR increased to 92.6% in the P-Gemox+Thal arm, and the CR rate increased to 88.8%. In the AspaMetDex arm, the ORR increased to 88.8%, and the CR rate increased to 85.1%.

At a median follow-up of 13.5 months, the 2-year progression-free survival rate was 82.9% in the P-Gemox+Thal arm and 84.5% in the AspaMetDex arm (P=0.791).

The 2-year overall survival rates were 95.0% in the P-Gemox+Thal arm and 75.8% in the AspaMetDex arm (P=0.089).

Advanced, rel/ref ENKTL

Of the 47 patients with stage III/IV or relapsed/refractory ENKTL, 24 were randomized to P-Gemox+Thal, and 23 to AspaMetDex. In both arms, most patients were male (75% and 87%, respectively) and younger than 60 (95.8% and 91.3%, respectively).

ECOG status was 0 for 62.5% of patients in the P-Gemox+Thal arm and 73.9% of those in the AspaMetDex arm. ECOG status was 1 for 33.3% and 17.4%, respectively.

The ORR was 86.3% in the P-Gemox+Thal arm and 70% in the AspaMetDex arm. The CR rate was 50% in both arms.

The partial response rate was 36.3% in the P-Gemox+Thal arm and 20% in the AspaMetDex arm. And the rate of stable disease was 13.6% and 15%, respectively.

Three patients in each treatment arm went on to ASCT after CR. A total of 3 patients relapsed within 6 months of ASCT—2 in the P-Gemox+Thal arm and 1 in the AspaMetDex arm. Two patients died of disease progression.

At a median follow-up of 14.5 months, the 2-year progression-free survival was 12.2 months in the P-Gemox+Thal arm and 7.6 months in the AspaMetDex arm (P=0.365).

The 2-year overall survival was 52.5% in the P-Gemox+Thal arm and 48.9% in the AspaMetDex arm (P=0.935).

Overall safety

Rates of leukopenia, thrombocytopenia, and ALT/AST increase were all significantly higher with P-Gemox+Thal than with AspaMetDex—100% vs 66.7% (P<0.001), 64.2% vs 35.2% (P=0.005), and 69.6% vs 64.8% (P=0.004), respectively.

Rates of anemia and edema were significantly higher with AspaMetDex than with P-Gemox+Thal—51.8% vs 77.8% (P=0.005) and 37.5% vs 66.7% (P=0.003), respectively.

There were 3 treatment-related deaths in the AspaMetDex arm but none in the P-Gemox+Thal arm. Two of the treatment-related deaths—from severe acute renal failure and sepsis—occurred in the first cycle, and 1 death—due to severe sepsis—occurred in the third cycle.

The median hospitalization time was significantly shorter in the P-Gemox+Thal arm than the AspaMetDex arm—1.9 days and 4.9 days, respectively (P<0.01).

Based on these results, Dr Huang said P-Gemox+Thal may be more tolerable and provide more convenient administration than AspaMetDex.

Huiqiang Huang, MD
Photo by Larry Young

SAN FRANCISCO—Interim results of a phase 3 trial suggest 2 treatment regimens may provide comparable efficacy in patients with extranodal natural killer/T-cell lymphoma (ENKTL), though 1 regimen appears more toxic than the other.

In this ongoing trial, investigators are comparing pegaspargase, gemcitabine, oxaliplatin, and thalidomide (P-Gemox+Thal) to pegaspargase, methotrexate, calcium folinate, and dexamethasone (AspaMetDex).

In some patients, either regimen may be followed by extensive involved-field radiotherapy (EIFRT) or autologous hematopoietic stem cell transplant (ASCT).

Thus far, P-Gemox+Thal and AspaMetDex have proven similarly effective for patients with newly diagnosed, stage I/II ENKTL.

And both regimens have produced unsatisfying survival outcomes in patients with advanced or relapsed/refractory ENKTL, according to investigator Huiqiang Huang, MD, of Sun Yat-sen Institute of Hematology in Guangzhou, China.

In addition, P-Gemox+Thal seems to be less toxic, overall, than AspaMetDex.

However, Dr Huang said it is still too early to draw any firm conclusions about these regimens. 

He presented results from this trial at the 9th Annual T-cell Lymphoma Forum. Results were previously presented at the 2016 ASH Annual Meeting (abstract 1819).

Dr Huang noted that AspaMetDex and SMILE (dexamethasone, methotrexate, ifosfamide, lL-asparaginase, and etoposide) are frequently administered to patients with ENKTL. And P-Gemox is recommended in the 2016 NCCN guidelines.

“But optimal regimens still have not been fully defined,” he said.

Therefore, he and his colleagues decided to compare AspaMetDex to P-Gemox+Thal in this non-inferiority trial, which has enrolled 110 patients from 12 centers in China.

Treatment

Fifty-six patients have been randomized to receive P-Gemox+Thal. Every 3 weeks, they received pegaspargase at 2000 U/m² on day 1, gemcitabine at 1000 mg/m² on days 1 and 8, and oxaliplatin at 130 mg/m² on days 1 and 8. They also received thalidomide at 100 mg every day for 1 year.

Fifty-four patients have been randomized to receive AspaMetDex. Every 3 weeks, they received pegaspargase at 2000 U/m² on day 1, methotrexate at 3000 mg/m² on day 1, calcium folinate at 30 mg every 6 hours until a safe serum methotrexate concentration was reached, and dexamethasone at 40 mg every day on days 1 to 4.

Newly diagnosed patients with stage I/II disease received either regimen as induction for a maximum of 4 cycles. Responders went on to receive EIFRT at 56 Gy in 28 fractions over 4 weeks.

Patients with newly diagnosed, stage III/IV ENKTL or relapsed/refractory ENKTL received either chemotherapy regimen for a maximum of 6 cycles. If they achieved a complete response (CR), these patients could proceed to ASCT.

Stage I/II ENKTL

Of the 63 stage I/II patients, 33 were randomized to P-Gemox+Thal, and 30 received AspaMetDex. In both arms, most patients were male (69.7% and 70%, respectively) and younger than 60 (78.8% and 90%, respectively).

Ninety-seven percent of patients in the P-Gemox+Thal arm had an ECOG status of 0-1, as did 100% of patients in the AspaMetDex arm.

The overall response rate (ORR) was 85.2% in the P-Gemox+Thal arm and 81.5% in the AspaMetDex arm. The CR rate was 59.3% in both arms. The rate of stable disease was 3.7% in the P-Gemox+Thal arm and 11.1% in the AspaMetDex arm.

After EIFRT, the ORR increased to 92.6% in the P-Gemox+Thal arm, and the CR rate increased to 88.8%. In the AspaMetDex arm, the ORR increased to 88.8%, and the CR rate increased to 85.1%.

At a median follow-up of 13.5 months, the 2-year progression-free survival rate was 82.9% in the P-Gemox+Thal arm and 84.5% in the AspaMetDex arm (P=0.791).

The 2-year overall survival rates were 95.0% in the P-Gemox+Thal arm and 75.8% in the AspaMetDex arm (P=0.089).

Advanced, rel/ref ENKTL

Of the 47 patients with stage III/IV or relapsed/refractory ENKTL, 24 were randomized to P-Gemox+Thal, and 23 to AspaMetDex. In both arms, most patients were male (75% and 87%, respectively) and younger than 60 (95.8% and 91.3%, respectively).

ECOG status was 0 for 62.5% of patients in the P-Gemox+Thal arm and 73.9% of those in the AspaMetDex arm. ECOG status was 1 for 33.3% and 17.4%, respectively.

The ORR was 86.3% in the P-Gemox+Thal arm and 70% in the AspaMetDex arm. The CR rate was 50% in both arms.

The partial response rate was 36.3% in the P-Gemox+Thal arm and 20% in the AspaMetDex arm. And the rate of stable disease was 13.6% and 15%, respectively.

Three patients in each treatment arm went on to ASCT after CR. A total of 3 patients relapsed within 6 months of ASCT—2 in the P-Gemox+Thal arm and 1 in the AspaMetDex arm. Two patients died of disease progression.

At a median follow-up of 14.5 months, the 2-year progression-free survival was 12.2 months in the P-Gemox+Thal arm and 7.6 months in the AspaMetDex arm (P=0.365).

The 2-year overall survival was 52.5% in the P-Gemox+Thal arm and 48.9% in the AspaMetDex arm (P=0.935).

Overall safety

Rates of leukopenia, thrombocytopenia, and ALT/AST increase were all significantly higher with P-Gemox+Thal than with AspaMetDex—100% vs 66.7% (P<0.001), 64.2% vs 35.2% (P=0.005), and 69.6% vs 64.8% (P=0.004), respectively.

Rates of anemia and edema were significantly higher with AspaMetDex than with P-Gemox+Thal—51.8% vs 77.8% (P=0.005) and 37.5% vs 66.7% (P=0.003), respectively.

There were 3 treatment-related deaths in the AspaMetDex arm but none in the P-Gemox+Thal arm. Two of the treatment-related deaths—from severe acute renal failure and sepsis—occurred in the first cycle, and 1 death—due to severe sepsis—occurred in the third cycle.

The median hospitalization time was significantly shorter in the P-Gemox+Thal arm than the AspaMetDex arm—1.9 days and 4.9 days, respectively (P<0.01).

Based on these results, Dr Huang said P-Gemox+Thal may be more tolerable and provide more convenient administration than AspaMetDex.

Huiqiang Huang, MD
Photo by Larry Young

SAN FRANCISCO—Interim results of a phase 3 trial suggest 2 treatment regimens may provide comparable efficacy in patients with extranodal natural killer/T-cell lymphoma (ENKTL), though 1 regimen appears more toxic than the other.

In this ongoing trial, investigators are comparing pegaspargase, gemcitabine, oxaliplatin, and thalidomide (P-Gemox+Thal) to pegaspargase, methotrexate, calcium folinate, and dexamethasone (AspaMetDex).

In some patients, either regimen may be followed by extensive involved-field radiotherapy (EIFRT) or autologous hematopoietic stem cell transplant (ASCT).

Thus far, P-Gemox+Thal and AspaMetDex have proven similarly effective for patients with newly diagnosed, stage I/II ENKTL.

And both regimens have produced unsatisfying survival outcomes in patients with advanced or relapsed/refractory ENKTL, according to investigator Huiqiang Huang, MD, of Sun Yat-sen Institute of Hematology in Guangzhou, China.

In addition, P-Gemox+Thal seems to be less toxic, overall, than AspaMetDex.

However, Dr Huang said it is still too early to draw any firm conclusions about these regimens. 

He presented results from this trial at the 9th Annual T-cell Lymphoma Forum. Results were previously presented at the 2016 ASH Annual Meeting (abstract 1819).

Dr Huang noted that AspaMetDex and SMILE (dexamethasone, methotrexate, ifosfamide, lL-asparaginase, and etoposide) are frequently administered to patients with ENKTL. And P-Gemox is recommended in the 2016 NCCN guidelines.

“But optimal regimens still have not been fully defined,” he said.

Therefore, he and his colleagues decided to compare AspaMetDex to P-Gemox+Thal in this non-inferiority trial, which has enrolled 110 patients from 12 centers in China.

Treatment

Fifty-six patients have been randomized to receive P-Gemox+Thal. Every 3 weeks, they received pegaspargase at 2000 U/m² on day 1, gemcitabine at 1000 mg/m² on days 1 and 8, and oxaliplatin at 130 mg/m² on days 1 and 8. They also received thalidomide at 100 mg every day for 1 year.

Fifty-four patients have been randomized to receive AspaMetDex. Every 3 weeks, they received pegaspargase at 2000 U/m² on day 1, methotrexate at 3000 mg/m² on day 1, calcium folinate at 30 mg every 6 hours until a safe serum methotrexate concentration was reached, and dexamethasone at 40 mg every day on days 1 to 4.

Newly diagnosed patients with stage I/II disease received either regimen as induction for a maximum of 4 cycles. Responders went on to receive EIFRT at 56 Gy in 28 fractions over 4 weeks.

Patients with newly diagnosed, stage III/IV ENKTL or relapsed/refractory ENKTL received either chemotherapy regimen for a maximum of 6 cycles. If they achieved a complete response (CR), these patients could proceed to ASCT.

Stage I/II ENKTL

Of the 63 stage I/II patients, 33 were randomized to P-Gemox+Thal, and 30 received AspaMetDex. In both arms, most patients were male (69.7% and 70%, respectively) and younger than 60 (78.8% and 90%, respectively).

Ninety-seven percent of patients in the P-Gemox+Thal arm had an ECOG status of 0-1, as did 100% of patients in the AspaMetDex arm.

The overall response rate (ORR) was 85.2% in the P-Gemox+Thal arm and 81.5% in the AspaMetDex arm. The CR rate was 59.3% in both arms. The rate of stable disease was 3.7% in the P-Gemox+Thal arm and 11.1% in the AspaMetDex arm.

After EIFRT, the ORR increased to 92.6% in the P-Gemox+Thal arm, and the CR rate increased to 88.8%. In the AspaMetDex arm, the ORR increased to 88.8%, and the CR rate increased to 85.1%.

At a median follow-up of 13.5 months, the 2-year progression-free survival rate was 82.9% in the P-Gemox+Thal arm and 84.5% in the AspaMetDex arm (P=0.791).

The 2-year overall survival rates were 95.0% in the P-Gemox+Thal arm and 75.8% in the AspaMetDex arm (P=0.089).

Advanced, rel/ref ENKTL

Of the 47 patients with stage III/IV or relapsed/refractory ENKTL, 24 were randomized to P-Gemox+Thal, and 23 to AspaMetDex. In both arms, most patients were male (75% and 87%, respectively) and younger than 60 (95.8% and 91.3%, respectively).

ECOG status was 0 for 62.5% of patients in the P-Gemox+Thal arm and 73.9% of those in the AspaMetDex arm. ECOG status was 1 for 33.3% and 17.4%, respectively.

The ORR was 86.3% in the P-Gemox+Thal arm and 70% in the AspaMetDex arm. The CR rate was 50% in both arms.

The partial response rate was 36.3% in the P-Gemox+Thal arm and 20% in the AspaMetDex arm. And the rate of stable disease was 13.6% and 15%, respectively.

Three patients in each treatment arm went on to ASCT after CR. A total of 3 patients relapsed within 6 months of ASCT—2 in the P-Gemox+Thal arm and 1 in the AspaMetDex arm. Two patients died of disease progression.

At a median follow-up of 14.5 months, the 2-year progression-free survival was 12.2 months in the P-Gemox+Thal arm and 7.6 months in the AspaMetDex arm (P=0.365).

The 2-year overall survival was 52.5% in the P-Gemox+Thal arm and 48.9% in the AspaMetDex arm (P=0.935).

Overall safety

Rates of leukopenia, thrombocytopenia, and ALT/AST increase were all significantly higher with P-Gemox+Thal than with AspaMetDex—100% vs 66.7% (P<0.001), 64.2% vs 35.2% (P=0.005), and 69.6% vs 64.8% (P=0.004), respectively.

Rates of anemia and edema were significantly higher with AspaMetDex than with P-Gemox+Thal—51.8% vs 77.8% (P=0.005) and 37.5% vs 66.7% (P=0.003), respectively.

There were 3 treatment-related deaths in the AspaMetDex arm but none in the P-Gemox+Thal arm. Two of the treatment-related deaths—from severe acute renal failure and sepsis—occurred in the first cycle, and 1 death—due to severe sepsis—occurred in the third cycle.

The median hospitalization time was significantly shorter in the P-Gemox+Thal arm than the AspaMetDex arm—1.9 days and 4.9 days, respectively (P<0.01).

Based on these results, Dr Huang said P-Gemox+Thal may be more tolerable and provide more convenient administration than AspaMetDex.

DNA helices
Image courtesy of the
National Institute of
General Medical Sciences

A gene therapy known as DTX101 has shown early promise for the treatment of adults with hemophilia B, according to the company developing the therapy.

DTX101 is designed to deliver stable expression of factor IX (FIX) in patients with hemophilia B.

Preliminary results from a phase 1/2 trial showed that DTX101 can increase FIX expression in these patients, allowing some to forgo prophylactic and on-demand treatment.

However, 5 of the 6 patients enrolled in this trial experienced elevations in alanine aminotransferase (ALT), with 1 patient experiencing a grade 4 adverse event as a result.

These results were released by Dimension Therapeutics, Inc., the company developing DTX101.

DTX101 is a non-replicating, recombinant adeno-associated viral vector, AAVrh10, with a codon-optimized FIX gene expressing wild-type FIX protein.

The phase 1/2 study of DTX101 has enrolled 6 patients, ages 28 to 70, with moderate/severe to severe hemophilia B. They had baseline FIX expression of ≤ 2%, which requires either prophylactic or on-demand recombinant FIX therapy.

These 6 patients were divided into 2 dose cohorts. Cohort 1 (n=3) received DXT101 at 1.6 x 10¹² GC/kg. And cohort 2 (n=3) received 5 x 10¹² GC/kg.

All patients have been in post-treatment follow-up ranging from 6 weeks to 52 weeks.

Efficacy

Researchers observed evidence of efficient liver transduction of DTX101 across the 2 cohorts.

Patients in the low-dose cohort achieved peak FIX expression levels of 10% to 11%, which stabilized to between 3% and 4% at last follow-up (weeks 24, 48, and 52).

Patients in the second dose cohort achieved peak FIX expression of 13%, 20%, and 12% at weeks 4, 8, and 8, respectively. FIX activity was 5% and 8% in 2 patients at 12 weeks of follow-up. It was 7% for the third patient at 7 weeks.

None of the patients in cohort 2 have required prophylactic or on-demand recombinant FIX therapy for spontaneous bleeds post-dosing.

Safety

None of the patients experienced a drug-related serious adverse event as of the January 28, 2017, data cutoff.

Five of the 6 patients had elevations in ALT. All elevated liver enzymes were clinically asymptomatic with no elevations of gamma-glutamyl transferase, alkaline phosphatase, or bilirubin.

Patient 3 in cohort 2 experienced a grade 4 adverse event due to an elevated laboratory ALT (defined as > 800 IU/L).

Preliminary findings from 2 patients in each cohort prompted the administration of a standard tapering course of corticosteroids to treat mild, asymptomatic elevations in ALT (52-98 IU/L).

The third patient in cohort 2 also received corticosteroids, experiencing a peak ALT of 914 IU/L, and was at 431 IU/L at 6 weeks post-dosing.

As of the January 28, 2017, data cutoff, 2 of 3 patients in cohort 2 had ALT levels in the normal range. Cohort 1 patients were all clinically stable and off steroids, with ALT levels in the normal range.

Dimension Therapeutics said it expects cohort 2 will continue to receive a standard tapering course of corticosteroid therapy.

As required by the trial protocol, the company reported the ALT levels for patient 3 in cohort 2 to the Data Safety Monitoring Committee, the US Food and Drug Administration, and the appropriate regulatory authorities.

The company said it will await their feedback prior to initiating dosing of cohort 3.

“We are encouraged by the apparent efficiency of gene transduction and the early trend we are seeing in sustained FIX activity across both cohorts with our wild-type FIX AAVrh10 vector in patients,” said Annalisa Jenkins, MBBS, chief executive officer of Dimension Therapeutics.

“We continue to explore the therapeutic window for DTX101 as our data mature and in light of the ALT rises that appear to be associated with a decline in FIX activity.”

DNA helices
Image courtesy of the
National Institute of
General Medical Sciences

A gene therapy known as DTX101 has shown early promise for the treatment of adults with hemophilia B, according to the company developing the therapy.

DTX101 is designed to deliver stable expression of factor IX (FIX) in patients with hemophilia B.

Preliminary results from a phase 1/2 trial showed that DTX101 can increase FIX expression in these patients, allowing some to forgo prophylactic and on-demand treatment.

However, 5 of the 6 patients enrolled in this trial experienced elevations in alanine aminotransferase (ALT), with 1 patient experiencing a grade 4 adverse event as a result.

These results were released by Dimension Therapeutics, Inc., the company developing DTX101.

DTX101 is a non-replicating, recombinant adeno-associated viral vector, AAVrh10, with a codon-optimized FIX gene expressing wild-type FIX protein.

The phase 1/2 study of DTX101 has enrolled 6 patients, ages 28 to 70, with moderate/severe to severe hemophilia B. They had baseline FIX expression of ≤ 2%, which requires either prophylactic or on-demand recombinant FIX therapy.

These 6 patients were divided into 2 dose cohorts. Cohort 1 (n=3) received DXT101 at 1.6 x 10¹² GC/kg. And cohort 2 (n=3) received 5 x 10¹² GC/kg.

All patients have been in post-treatment follow-up ranging from 6 weeks to 52 weeks.

Efficacy

Researchers observed evidence of efficient liver transduction of DTX101 across the 2 cohorts.

Patients in the low-dose cohort achieved peak FIX expression levels of 10% to 11%, which stabilized to between 3% and 4% at last follow-up (weeks 24, 48, and 52).

Patients in the second dose cohort achieved peak FIX expression of 13%, 20%, and 12% at weeks 4, 8, and 8, respectively. FIX activity was 5% and 8% in 2 patients at 12 weeks of follow-up. It was 7% for the third patient at 7 weeks.

None of the patients in cohort 2 have required prophylactic or on-demand recombinant FIX therapy for spontaneous bleeds post-dosing.

Safety

None of the patients experienced a drug-related serious adverse event as of the January 28, 2017, data cutoff.

Five of the 6 patients had elevations in ALT. All elevated liver enzymes were clinically asymptomatic with no elevations of gamma-glutamyl transferase, alkaline phosphatase, or bilirubin.

Patient 3 in cohort 2 experienced a grade 4 adverse event due to an elevated laboratory ALT (defined as > 800 IU/L).

Preliminary findings from 2 patients in each cohort prompted the administration of a standard tapering course of corticosteroids to treat mild, asymptomatic elevations in ALT (52-98 IU/L).

The third patient in cohort 2 also received corticosteroids, experiencing a peak ALT of 914 IU/L, and was at 431 IU/L at 6 weeks post-dosing.

As of the January 28, 2017, data cutoff, 2 of 3 patients in cohort 2 had ALT levels in the normal range. Cohort 1 patients were all clinically stable and off steroids, with ALT levels in the normal range.

Dimension Therapeutics said it expects cohort 2 will continue to receive a standard tapering course of corticosteroid therapy.

As required by the trial protocol, the company reported the ALT levels for patient 3 in cohort 2 to the Data Safety Monitoring Committee, the US Food and Drug Administration, and the appropriate regulatory authorities.

The company said it will await their feedback prior to initiating dosing of cohort 3.

“We are encouraged by the apparent efficiency of gene transduction and the early trend we are seeing in sustained FIX activity across both cohorts with our wild-type FIX AAVrh10 vector in patients,” said Annalisa Jenkins, MBBS, chief executive officer of Dimension Therapeutics.

“We continue to explore the therapeutic window for DTX101 as our data mature and in light of the ALT rises that appear to be associated with a decline in FIX activity.”

DNA helices
Image courtesy of the
National Institute of
General Medical Sciences

A gene therapy known as DTX101 has shown early promise for the treatment of adults with hemophilia B, according to the company developing the therapy.

DTX101 is designed to deliver stable expression of factor IX (FIX) in patients with hemophilia B.

Preliminary results from a phase 1/2 trial showed that DTX101 can increase FIX expression in these patients, allowing some to forgo prophylactic and on-demand treatment.

However, 5 of the 6 patients enrolled in this trial experienced elevations in alanine aminotransferase (ALT), with 1 patient experiencing a grade 4 adverse event as a result.

These results were released by Dimension Therapeutics, Inc., the company developing DTX101.

DTX101 is a non-replicating, recombinant adeno-associated viral vector, AAVrh10, with a codon-optimized FIX gene expressing wild-type FIX protein.

The phase 1/2 study of DTX101 has enrolled 6 patients, ages 28 to 70, with moderate/severe to severe hemophilia B. They had baseline FIX expression of ≤ 2%, which requires either prophylactic or on-demand recombinant FIX therapy.

These 6 patients were divided into 2 dose cohorts. Cohort 1 (n=3) received DXT101 at 1.6 x 10¹² GC/kg. And cohort 2 (n=3) received 5 x 10¹² GC/kg.

All patients have been in post-treatment follow-up ranging from 6 weeks to 52 weeks.

Efficacy

Researchers observed evidence of efficient liver transduction of DTX101 across the 2 cohorts.

Patients in the low-dose cohort achieved peak FIX expression levels of 10% to 11%, which stabilized to between 3% and 4% at last follow-up (weeks 24, 48, and 52).

Patients in the second dose cohort achieved peak FIX expression of 13%, 20%, and 12% at weeks 4, 8, and 8, respectively. FIX activity was 5% and 8% in 2 patients at 12 weeks of follow-up. It was 7% for the third patient at 7 weeks.

None of the patients in cohort 2 have required prophylactic or on-demand recombinant FIX therapy for spontaneous bleeds post-dosing.

Safety

None of the patients experienced a drug-related serious adverse event as of the January 28, 2017, data cutoff.

Five of the 6 patients had elevations in ALT. All elevated liver enzymes were clinically asymptomatic with no elevations of gamma-glutamyl transferase, alkaline phosphatase, or bilirubin.

Patient 3 in cohort 2 experienced a grade 4 adverse event due to an elevated laboratory ALT (defined as > 800 IU/L).

Preliminary findings from 2 patients in each cohort prompted the administration of a standard tapering course of corticosteroids to treat mild, asymptomatic elevations in ALT (52-98 IU/L).

The third patient in cohort 2 also received corticosteroids, experiencing a peak ALT of 914 IU/L, and was at 431 IU/L at 6 weeks post-dosing.

As of the January 28, 2017, data cutoff, 2 of 3 patients in cohort 2 had ALT levels in the normal range. Cohort 1 patients were all clinically stable and off steroids, with ALT levels in the normal range.

Dimension Therapeutics said it expects cohort 2 will continue to receive a standard tapering course of corticosteroid therapy.

As required by the trial protocol, the company reported the ALT levels for patient 3 in cohort 2 to the Data Safety Monitoring Committee, the US Food and Drug Administration, and the appropriate regulatory authorities.

The company said it will await their feedback prior to initiating dosing of cohort 3.

“We are encouraged by the apparent efficiency of gene transduction and the early trend we are seeing in sustained FIX activity across both cohorts with our wild-type FIX AAVrh10 vector in patients,” said Annalisa Jenkins, MBBS, chief executive officer of Dimension Therapeutics.

“We continue to explore the therapeutic window for DTX101 as our data mature and in light of the ALT rises that appear to be associated with a decline in FIX activity.”

Pill production

Photo courtesy of FDA

AMSTERDAM—New research suggests some generic cancer drugs could be manufactured for less than 1% of the prices currently charged in the US and UK.

For example, researchers calculated that manufacturing a 400 mg tablet of imatinib costs $0.92.

Charging $1.04 per tablet would cover costs and allow for a 10% profit margin.

However, the current price of imatinib is $84.36 per tablet in the UK and $247.74 per tablet in the US.

Melissa Barber, of the London School of Hygiene and Tropical Medicine in the UK, reported these findings at ECCO 2017: European Cancer Congress (abstract 1032).

Barber and her colleagues collected data on per-kilogram costs of exported active pharmaceutical ingredients (APIs) from an online database of Indian export logs.

The team then estimated generic prices for tablets through an established costing algorithm. They calculated per-dose API costs and added excipient costs of $2.63 per kg of finished pharmaceutical product and per-tablet costs of production of $0.01, plus a 10% profit margin accounting for a 26.6% average tax on profits (assuming manufacture in India.)

Finally, the researchers compared the calculated price to current unit prices in the US, UK, Spain, and India.

For imatinib, the team determined the cost of the API to be $2284 per kg and the API cost per tablet to be $0.91. They then added excipient cost ($0.002 per tablet), conversion cost ($0.01 per tablet), and a 10% profit margin accounting for a 26.6% tax on profits.

This resulted in the estimated generic price of $1.04 per tablet. The per-tablet price is below the estimated price in India ($0.22) but much higher than the estimated price in Spain ($57.53), the UK ($84.36), and the US ($247.74).

Barber noted that, according to her group’s calculations, imatinib could be produced for $54 a month.

Another drug that could be produced for a low cost is etoposide. Barber and her colleagues calculated a generic price for etoposide of $0.97 per 100 mg tablet.

However, the per-tablet price is $1.50 in India, $8.65 in Spain, $11.34 in the UK, and $87.14 in the US.

The researchers calculated a generic price for mercaptopurine of $0.03 per 50 mg tablet, which is the same as the per-tablet price in India. However, a 50 mg mercaptopurine tablet costs $3.14 in Spain, $2.56 in the UK, and $0.40 in the US.

“Showing that certain cancers could be treated for very low prices could transform the future of people with these cancers in very low-income countries where there are usually few or no treatment options,” Barber said.

Pill production

Photo courtesy of FDA

AMSTERDAM—New research suggests some generic cancer drugs could be manufactured for less than 1% of the prices currently charged in the US and UK.

For example, researchers calculated that manufacturing a 400 mg tablet of imatinib costs $0.92.

Charging $1.04 per tablet would cover costs and allow for a 10% profit margin.

However, the current price of imatinib is $84.36 per tablet in the UK and $247.74 per tablet in the US.

Melissa Barber, of the London School of Hygiene and Tropical Medicine in the UK, reported these findings at ECCO 2017: European Cancer Congress (abstract 1032).

Barber and her colleagues collected data on per-kilogram costs of exported active pharmaceutical ingredients (APIs) from an online database of Indian export logs.

The team then estimated generic prices for tablets through an established costing algorithm. They calculated per-dose API costs and added excipient costs of $2.63 per kg of finished pharmaceutical product and per-tablet costs of production of $0.01, plus a 10% profit margin accounting for a 26.6% average tax on profits (assuming manufacture in India.)

Finally, the researchers compared the calculated price to current unit prices in the US, UK, Spain, and India.

For imatinib, the team determined the cost of the API to be $2284 per kg and the API cost per tablet to be $0.91. They then added excipient cost ($0.002 per tablet), conversion cost ($0.01 per tablet), and a 10% profit margin accounting for a 26.6% tax on profits.

This resulted in the estimated generic price of $1.04 per tablet. The per-tablet price is below the estimated price in India ($0.22) but much higher than the estimated price in Spain ($57.53), the UK ($84.36), and the US ($247.74).

Barber noted that, according to her group’s calculations, imatinib could be produced for $54 a month.

Another drug that could be produced for a low cost is etoposide. Barber and her colleagues calculated a generic price for etoposide of $0.97 per 100 mg tablet.

However, the per-tablet price is $1.50 in India, $8.65 in Spain, $11.34 in the UK, and $87.14 in the US.

The researchers calculated a generic price for mercaptopurine of $0.03 per 50 mg tablet, which is the same as the per-tablet price in India. However, a 50 mg mercaptopurine tablet costs $3.14 in Spain, $2.56 in the UK, and $0.40 in the US.

“Showing that certain cancers could be treated for very low prices could transform the future of people with these cancers in very low-income countries where there are usually few or no treatment options,” Barber said.

Pill production

Photo courtesy of FDA

AMSTERDAM—New research suggests some generic cancer drugs could be manufactured for less than 1% of the prices currently charged in the US and UK.

For example, researchers calculated that manufacturing a 400 mg tablet of imatinib costs $0.92.

Charging $1.04 per tablet would cover costs and allow for a 10% profit margin.

However, the current price of imatinib is $84.36 per tablet in the UK and $247.74 per tablet in the US.

Melissa Barber, of the London School of Hygiene and Tropical Medicine in the UK, reported these findings at ECCO 2017: European Cancer Congress (abstract 1032).

Barber and her colleagues collected data on per-kilogram costs of exported active pharmaceutical ingredients (APIs) from an online database of Indian export logs.

The team then estimated generic prices for tablets through an established costing algorithm. They calculated per-dose API costs and added excipient costs of $2.63 per kg of finished pharmaceutical product and per-tablet costs of production of $0.01, plus a 10% profit margin accounting for a 26.6% average tax on profits (assuming manufacture in India.)

Finally, the researchers compared the calculated price to current unit prices in the US, UK, Spain, and India.

For imatinib, the team determined the cost of the API to be $2284 per kg and the API cost per tablet to be $0.91. They then added excipient cost ($0.002 per tablet), conversion cost ($0.01 per tablet), and a 10% profit margin accounting for a 26.6% tax on profits.

This resulted in the estimated generic price of $1.04 per tablet. The per-tablet price is below the estimated price in India ($0.22) but much higher than the estimated price in Spain ($57.53), the UK ($84.36), and the US ($247.74).

Barber noted that, according to her group’s calculations, imatinib could be produced for $54 a month.

Another drug that could be produced for a low cost is etoposide. Barber and her colleagues calculated a generic price for etoposide of $0.97 per 100 mg tablet.

However, the per-tablet price is $1.50 in India, $8.65 in Spain, $11.34 in the UK, and $87.14 in the US.

The researchers calculated a generic price for mercaptopurine of $0.03 per 50 mg tablet, which is the same as the per-tablet price in India. However, a 50 mg mercaptopurine tablet costs $3.14 in Spain, $2.56 in the UK, and $0.40 in the US.

“Showing that certain cancers could be treated for very low prices could transform the future of people with these cancers in very low-income countries where there are usually few or no treatment options,” Barber said.

White blood cells

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has announced that Sandoz GmbH withdrew its marketing authorization application (MAA) for Zioxtenzo.

The active ingredient of Zioxtenzo is pegfilgrastim, and the product was intended to be biosimilar to Amgen’s Neulasta.

The intended use for Zioxtenzo was to reduce the duration of neutropenia and the occurrence of febrile neutropenia in cancer patients.

In its application for Zioxtenzo, Sandoz presented results of studies designed to show the product is highly similar to Neulasta in terms of chemical structure, purity, the way it works, and how the body handles the drug.

In addition, there were 2 studies comparing the safety and effectiveness of Zioxtenzo and Neulasta in patients receiving cancer drugs.

Sandoz withdrew the MAA for Zioxtenzo after the CHMP had evaluated the initial documentation provided by the company and formulated a list of questions. The company had not responded to the questions at the time of the withdrawal. 

Based on a review of the data, at the time of the withdrawal, the CHMP had 2 main concerns and was of the provisional opinion that Zioxtenzo could not have been approved as a biosimilar of Neulasta.

One concern was that study results were not able to show that the concentrations of pegfilgrastim in blood were the same after taking Zioxtenzo and Neulasta.

The other concern was the lack of a certificate of Good Manufacturing Practice for Zioxtenzo’s manufacturing site. An inspection of the site would therefore be needed before the drug could be approved.

At the time of the MAA withdrawal, Sandoz had not demonstrated that Zioxtenzo is highly similar to Neulasta, and an inspection to confirm that Zioxtenzo was being manufactured according to Good Manufacturing Practice standards had not yet taken place. 

In its letter notifying the CHMP of the MAA withdrawal, Sandoz said it would not be able to provide the additional data required by the CHMP within the timeframe allowed for the procedure.

The company also said the withdrawal of Zioxtenzo will not impact ongoing clinical trials, and there are no compassionate use programs for Zioxtenzo.

White blood cells

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has announced that Sandoz GmbH withdrew its marketing authorization application (MAA) for Zioxtenzo.

The active ingredient of Zioxtenzo is pegfilgrastim, and the product was intended to be biosimilar to Amgen’s Neulasta.

The intended use for Zioxtenzo was to reduce the duration of neutropenia and the occurrence of febrile neutropenia in cancer patients.

In its application for Zioxtenzo, Sandoz presented results of studies designed to show the product is highly similar to Neulasta in terms of chemical structure, purity, the way it works, and how the body handles the drug.

In addition, there were 2 studies comparing the safety and effectiveness of Zioxtenzo and Neulasta in patients receiving cancer drugs.

Sandoz withdrew the MAA for Zioxtenzo after the CHMP had evaluated the initial documentation provided by the company and formulated a list of questions. The company had not responded to the questions at the time of the withdrawal. 

Based on a review of the data, at the time of the withdrawal, the CHMP had 2 main concerns and was of the provisional opinion that Zioxtenzo could not have been approved as a biosimilar of Neulasta.

One concern was that study results were not able to show that the concentrations of pegfilgrastim in blood were the same after taking Zioxtenzo and Neulasta.

The other concern was the lack of a certificate of Good Manufacturing Practice for Zioxtenzo’s manufacturing site. An inspection of the site would therefore be needed before the drug could be approved.

At the time of the MAA withdrawal, Sandoz had not demonstrated that Zioxtenzo is highly similar to Neulasta, and an inspection to confirm that Zioxtenzo was being manufactured according to Good Manufacturing Practice standards had not yet taken place. 

In its letter notifying the CHMP of the MAA withdrawal, Sandoz said it would not be able to provide the additional data required by the CHMP within the timeframe allowed for the procedure.

The company also said the withdrawal of Zioxtenzo will not impact ongoing clinical trials, and there are no compassionate use programs for Zioxtenzo.

White blood cells

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has announced that Sandoz GmbH withdrew its marketing authorization application (MAA) for Zioxtenzo.

The active ingredient of Zioxtenzo is pegfilgrastim, and the product was intended to be biosimilar to Amgen’s Neulasta.

The intended use for Zioxtenzo was to reduce the duration of neutropenia and the occurrence of febrile neutropenia in cancer patients.

In its application for Zioxtenzo, Sandoz presented results of studies designed to show the product is highly similar to Neulasta in terms of chemical structure, purity, the way it works, and how the body handles the drug.

In addition, there were 2 studies comparing the safety and effectiveness of Zioxtenzo and Neulasta in patients receiving cancer drugs.

Sandoz withdrew the MAA for Zioxtenzo after the CHMP had evaluated the initial documentation provided by the company and formulated a list of questions. The company had not responded to the questions at the time of the withdrawal. 

Based on a review of the data, at the time of the withdrawal, the CHMP had 2 main concerns and was of the provisional opinion that Zioxtenzo could not have been approved as a biosimilar of Neulasta.

One concern was that study results were not able to show that the concentrations of pegfilgrastim in blood were the same after taking Zioxtenzo and Neulasta.

The other concern was the lack of a certificate of Good Manufacturing Practice for Zioxtenzo’s manufacturing site. An inspection of the site would therefore be needed before the drug could be approved.

At the time of the MAA withdrawal, Sandoz had not demonstrated that Zioxtenzo is highly similar to Neulasta, and an inspection to confirm that Zioxtenzo was being manufactured according to Good Manufacturing Practice standards had not yet taken place. 

In its letter notifying the CHMP of the MAA withdrawal, Sandoz said it would not be able to provide the additional data required by the CHMP within the timeframe allowed for the procedure.

The company also said the withdrawal of Zioxtenzo will not impact ongoing clinical trials, and there are no compassionate use programs for Zioxtenzo.

Session at the 9th Annual

T-cell Lymphoma Forum

Photo by Larry Young

SAN FRANCISCO—Researchers have used data from the T-Cell Project (TCP) to create a prognostic model for peripheral T-cell lymphoma not otherwise specified (PTCL-NOS).

Analyses have suggested the TCP model is more accurate for PTCL-NOS than 4 other prognostic models—the International Prognostic Index (IPI), the Prognostic Index for T-cell Lymphoma (PIT), the International Peripheral T-cell Lymphoma Project score (IPTCLP), and the modified PIT (mPIT).

Massimo Federico, MD, of the University of Modena and Reggio Emilia in Italy, described the TCP model at the 9th Annual T-cell Lymphoma Forum.

Creating the model

TCP is a prospective registry that includes data from T-cell lymphoma patients in 15 countries located in 5 different regions of the world. As of December 31, 2016, 1523 cases of T-cell lymphoma have been registered with TCP.

Dr Federico and his colleagues used these data to create their prognostic model. There were 311 patients with PTCL-NOS who had adequate data for analysis. The 5-year overall survival (OS) for these patients was 36%.

The researchers chose 13 variables from the literature that have been reported to have a prognostic impact on survival in PTCL-NOS:

• Age > 60
• Lactate dehydrogenase > upper limit of normal
• Albumin < 3.5 g/dL
• Hemoglobin < 12 g/dL
• Platelets < 150/mm³
• Lymphocyte to monocyte ratio ≤ 2.1
• Neutrophil to lymphocyte ratio > 6.5
• Absolute neutrophil count (ANC) > 6.5/mm³
• ECOG performance status > 1
• Stage III-IV disease
• B symptoms
• Extra nodal sites > 1
• Male gender.

In univariate analysis, nearly all of these factors were significantly associated with OS in the cohort of TCP patients. (The 2 exceptions were age older than 60 and having more than 1 extranodal site.)

However, Dr Federico and his colleagues said the factors with the greatest prognostic impact were:

• ECOG performance status > 1, with a hazard ratio (HR) of 2.12 (P<0.001)
• Albumin < 3.5 g/dL, with an HR of 2.03 (P<0.001)
• ANC > 6.5/mm³, with an HR of 1.85 (P<0.001)
• Stage III-IV disease, with an HR of 1.74 (P=0.010).

So the researchers used these factors in their model, which has 3 risk categories.

Risk categories

Patients were considered low-risk if they had 0 of the 4 risk factors. These patients had a 3-year OS of 76% and a 5-year OS of 69%.

Patients were considered intermediate-risk if they had 1 to 2 risk factors. These patients had a 3-year OS of 43% and a 5-year OS of 31%. Compared to low-risk patients, the HR was 3.08 (P<0.001).

Patients were considered high-risk if they had 3 to 4 risk factors. The 3-year OS was 11% for these patients, and the 5-year OS was 8%.

The HR was 8.88 (P<0.001) for high-risk compared to low-risk patients and 2.88 (P<0.001) for high-risk compared to intermediate-risk patients.

Validation

The researchers tested the TCP model in a validation cohort of 98 patients from the COMPLETE registry. As with the training cohort of TCP patients, the model revealed 3 different risk groups (in terms of OS) in the validation cohort.

Dr Federico noted that there were no significant differences between the training and validation cohorts, except when it came to follow-up. The median follow-up was 46 months in the TCP group and 18 months in the COMPLETE group.

The researchers also found the TCP could classify patients into 3 different risk groups according to progression-free survival.

Comparison

Finally, Dr Federico and his colleagues compared the TCP model to the IPI, PIT, IPTCLP, and mPIT models using 208 patients.

“The discriminant power of the proposed model is superior to the others in terms of all of the statistical tests we adopted,” Dr Federico said.

In closing, Dr Federico said the TCP model clearly defines risk groups in PTCL-NOS and identifies patients with relatively good prognosis.

However, there is a need for emerging biologic variables to be tested for prognostic value and included in prognostic tools to allow for better risk stratification.

*c-Harrel: Harrell’s concordance index, 95% CI: confidence interval, D-Royston: Royston/Sauerbrei’s D statistic (Stat Med 2004 Mar 15, 23[5]:723-48), SE: standard error, R²: explained randomness, AIC: Akaike information criterion, AUC: area under the curve (according to Heagerty et al, Biometrics, 2000 Jun, 56[2]:337-44).

Session at the 9th Annual

T-cell Lymphoma Forum

Photo by Larry Young

SAN FRANCISCO—Researchers have used data from the T-Cell Project (TCP) to create a prognostic model for peripheral T-cell lymphoma not otherwise specified (PTCL-NOS).

Analyses have suggested the TCP model is more accurate for PTCL-NOS than 4 other prognostic models—the International Prognostic Index (IPI), the Prognostic Index for T-cell Lymphoma (PIT), the International Peripheral T-cell Lymphoma Project score (IPTCLP), and the modified PIT (mPIT).

Massimo Federico, MD, of the University of Modena and Reggio Emilia in Italy, described the TCP model at the 9th Annual T-cell Lymphoma Forum.

Creating the model

TCP is a prospective registry that includes data from T-cell lymphoma patients in 15 countries located in 5 different regions of the world. As of December 31, 2016, 1523 cases of T-cell lymphoma have been registered with TCP.

Dr Federico and his colleagues used these data to create their prognostic model. There were 311 patients with PTCL-NOS who had adequate data for analysis. The 5-year overall survival (OS) for these patients was 36%.

The researchers chose 13 variables from the literature that have been reported to have a prognostic impact on survival in PTCL-NOS:

• Age > 60
• Lactate dehydrogenase > upper limit of normal
• Albumin < 3.5 g/dL
• Hemoglobin < 12 g/dL
• Platelets < 150/mm³
• Lymphocyte to monocyte ratio ≤ 2.1
• Neutrophil to lymphocyte ratio > 6.5
• Absolute neutrophil count (ANC) > 6.5/mm³
• ECOG performance status > 1
• Stage III-IV disease
• B symptoms
• Extra nodal sites > 1
• Male gender.

In univariate analysis, nearly all of these factors were significantly associated with OS in the cohort of TCP patients. (The 2 exceptions were age older than 60 and having more than 1 extranodal site.)

However, Dr Federico and his colleagues said the factors with the greatest prognostic impact were:

• ECOG performance status > 1, with a hazard ratio (HR) of 2.12 (P<0.001)
• Albumin < 3.5 g/dL, with an HR of 2.03 (P<0.001)
• ANC > 6.5/mm³, with an HR of 1.85 (P<0.001)
• Stage III-IV disease, with an HR of 1.74 (P=0.010).

So the researchers used these factors in their model, which has 3 risk categories.

Risk categories

Patients were considered low-risk if they had 0 of the 4 risk factors. These patients had a 3-year OS of 76% and a 5-year OS of 69%.

Patients were considered intermediate-risk if they had 1 to 2 risk factors. These patients had a 3-year OS of 43% and a 5-year OS of 31%. Compared to low-risk patients, the HR was 3.08 (P<0.001).

Patients were considered high-risk if they had 3 to 4 risk factors. The 3-year OS was 11% for these patients, and the 5-year OS was 8%.

The HR was 8.88 (P<0.001) for high-risk compared to low-risk patients and 2.88 (P<0.001) for high-risk compared to intermediate-risk patients.

Validation

The researchers tested the TCP model in a validation cohort of 98 patients from the COMPLETE registry. As with the training cohort of TCP patients, the model revealed 3 different risk groups (in terms of OS) in the validation cohort.

Dr Federico noted that there were no significant differences between the training and validation cohorts, except when it came to follow-up. The median follow-up was 46 months in the TCP group and 18 months in the COMPLETE group.

The researchers also found the TCP could classify patients into 3 different risk groups according to progression-free survival.

Comparison

Finally, Dr Federico and his colleagues compared the TCP model to the IPI, PIT, IPTCLP, and mPIT models using 208 patients.

“The discriminant power of the proposed model is superior to the others in terms of all of the statistical tests we adopted,” Dr Federico said.

In closing, Dr Federico said the TCP model clearly defines risk groups in PTCL-NOS and identifies patients with relatively good prognosis.

However, there is a need for emerging biologic variables to be tested for prognostic value and included in prognostic tools to allow for better risk stratification.

*c-Harrel: Harrell’s concordance index, 95% CI: confidence interval, D-Royston: Royston/Sauerbrei’s D statistic (Stat Med 2004 Mar 15, 23[5]:723-48), SE: standard error, R²: explained randomness, AIC: Akaike information criterion, AUC: area under the curve (according to Heagerty et al, Biometrics, 2000 Jun, 56[2]:337-44).

Session at the 9th Annual

T-cell Lymphoma Forum

Photo by Larry Young

SAN FRANCISCO—Researchers have used data from the T-Cell Project (TCP) to create a prognostic model for peripheral T-cell lymphoma not otherwise specified (PTCL-NOS).

Analyses have suggested the TCP model is more accurate for PTCL-NOS than 4 other prognostic models—the International Prognostic Index (IPI), the Prognostic Index for T-cell Lymphoma (PIT), the International Peripheral T-cell Lymphoma Project score (IPTCLP), and the modified PIT (mPIT).

Massimo Federico, MD, of the University of Modena and Reggio Emilia in Italy, described the TCP model at the 9th Annual T-cell Lymphoma Forum.

Creating the model

TCP is a prospective registry that includes data from T-cell lymphoma patients in 15 countries located in 5 different regions of the world. As of December 31, 2016, 1523 cases of T-cell lymphoma have been registered with TCP.

Dr Federico and his colleagues used these data to create their prognostic model. There were 311 patients with PTCL-NOS who had adequate data for analysis. The 5-year overall survival (OS) for these patients was 36%.

The researchers chose 13 variables from the literature that have been reported to have a prognostic impact on survival in PTCL-NOS:

• Age > 60
• Lactate dehydrogenase > upper limit of normal
• Albumin < 3.5 g/dL
• Hemoglobin < 12 g/dL
• Platelets < 150/mm³
• Lymphocyte to monocyte ratio ≤ 2.1
• Neutrophil to lymphocyte ratio > 6.5
• Absolute neutrophil count (ANC) > 6.5/mm³
• ECOG performance status > 1
• Stage III-IV disease
• B symptoms
• Extra nodal sites > 1
• Male gender.

In univariate analysis, nearly all of these factors were significantly associated with OS in the cohort of TCP patients. (The 2 exceptions were age older than 60 and having more than 1 extranodal site.)

However, Dr Federico and his colleagues said the factors with the greatest prognostic impact were:

• ECOG performance status > 1, with a hazard ratio (HR) of 2.12 (P<0.001)
• Albumin < 3.5 g/dL, with an HR of 2.03 (P<0.001)
• ANC > 6.5/mm³, with an HR of 1.85 (P<0.001)
• Stage III-IV disease, with an HR of 1.74 (P=0.010).

So the researchers used these factors in their model, which has 3 risk categories.

Risk categories

Patients were considered low-risk if they had 0 of the 4 risk factors. These patients had a 3-year OS of 76% and a 5-year OS of 69%.

Patients were considered intermediate-risk if they had 1 to 2 risk factors. These patients had a 3-year OS of 43% and a 5-year OS of 31%. Compared to low-risk patients, the HR was 3.08 (P<0.001).

Patients were considered high-risk if they had 3 to 4 risk factors. The 3-year OS was 11% for these patients, and the 5-year OS was 8%.

The HR was 8.88 (P<0.001) for high-risk compared to low-risk patients and 2.88 (P<0.001) for high-risk compared to intermediate-risk patients.

Validation

The researchers tested the TCP model in a validation cohort of 98 patients from the COMPLETE registry. As with the training cohort of TCP patients, the model revealed 3 different risk groups (in terms of OS) in the validation cohort.

Dr Federico noted that there were no significant differences between the training and validation cohorts, except when it came to follow-up. The median follow-up was 46 months in the TCP group and 18 months in the COMPLETE group.

The researchers also found the TCP could classify patients into 3 different risk groups according to progression-free survival.

Comparison

Finally, Dr Federico and his colleagues compared the TCP model to the IPI, PIT, IPTCLP, and mPIT models using 208 patients.

“The discriminant power of the proposed model is superior to the others in terms of all of the statistical tests we adopted,” Dr Federico said.

In closing, Dr Federico said the TCP model clearly defines risk groups in PTCL-NOS and identifies patients with relatively good prognosis.

However, there is a need for emerging biologic variables to be tested for prognostic value and included in prognostic tools to allow for better risk stratification.

*c-Harrel: Harrell’s concordance index, 95% CI: confidence interval, D-Royston: Royston/Sauerbrei’s D statistic (Stat Med 2004 Mar 15, 23[5]:723-48), SE: standard error, R²: explained randomness, AIC: Akaike information criterion, AUC: area under the curve (according to Heagerty et al, Biometrics, 2000 Jun, 56[2]:337-44).

Massimo Federico, MD
Photo by Larry Young

SAN FRANCISCO—The T-Cell Project has provided information that can enhance our understanding of T-cell lymphomas, according to a presentation at the 9th Annual T-cell Lymphoma Forum.

The project is a prospective registry that includes data from T-cell lymphoma patients in 15 countries located in 5 different regions of the world. 

The data showed that peripheral T-cell lymphoma not otherwise specified (PTCL-NOS) is the most common subtype of T-cell lymphoma in all 5 regions, although the distribution of other subtypes varies.

A majority of patients in the registry received chemotherapy as induction, and anthracycline-containing regimens were the most popular treatment choice.

Although 60% of patients in the registry had low-risk or low/intermediate-risk disease, progression-free survival (PFS) and overall survival (OS) rates were low. The 5-year PFS was 32%, and the 5-year OS was 42%.

Massimo Federico, MD, of the University of Modena and Reggio Emilia in Italy, presented these data at the meeting.

About the project

Dr Federico said the goals of the T-Cell Project are to determine if prospective data collection provides more accurate information to better define prognosis of the most frequent subtypes of T-cell lymphoma and to improve our knowledge of clinical and biological characteristics, as well as outcomes, of the more uncommon subtypes.

“Why did we choose to propose a prospective registry for the collection of information in T-cell lymphoma?” Dr Federico asked. “Because it is, by far, less expensive than a clinical trial, but also because it can offer excellent data for generating new research programs and is a great opportunity for academic cooperation.”

As of December 31, 2016, the registry included 1523 patients. There were 75 sites (with at least 1 patient) active in the registry.

Fifteen countries in 5 geographic regions were represented. Europe was the greatest contributor (44%), followed by North America (US only, 25%), South America (20%), the Far East (9%), the Middle East (2%), and Oceania (<1%).

Subtypes

Overall, the distribution of the different T-cell lymphoma subtypes is as follows:

PTCL-NOS—36%
Angioimmunoblastic T-cell lymphoma (AITL)—17%
ALK- anaplastic large-cell lymphoma (ALCL)—16%
NK/T-cell lymphoma (NKTCL)—11%
ALK+ ALCL—8%
Enteropathy-associated T-cell lymphoma—4%
Unclassifiable T-cell lymphoma—3%
Hepatosplenic T-cell lymphoma—2%
Subcutaneous panniculitis-like T-cell lymphoma—2%
Peripheral gamma delta T-cell lymphoma—1%

Geographic distribution

The most common T-cell lymphoma subtypes in Europe were PTCL-NOS (37%), AITL (21%), and ALK- ALCL (14%). Likewise, the most common subtypes in the US were PTCL-NOS (35%), AITL (21%), and ALK- ALCL (13%).

In the Middle East, the most common subtypes were PTCL-NOS (40%), AITL (16%), and ALK+ ALCL (13%). In South America, they were PTCL-NOS (41%), ALK- ALCL (26%), and NKTCL (10%). And in Asia, they were PTCL-NOS (29%), NKTCL (29%), and AITL (17%).

Patient characteristics

Dr Federico presented data on patient characteristics for 1391 individuals, validated as of April 30, 2016.

The patients’ median age was 56 (range, 18-89). Forty-four percent were 60 or older, and 60% were male. Twenty-six percent had ECOG performance status > 1, 50% had B symptoms, and 72% had disease-related discomfort.

Sixty percent had low-risk or low/intermediate-risk disease according to the International Prognostic Index (IPI) and the Prognostic Index for T-cell Lymphoma (PIT).

Treatment

Treatment details are available for 1022 patients. Ninety-two percent received therapy with curative intent.

For induction, 76% of patients received chemotherapy alone, 14% received chemotherapy and radiotherapy, 8% received best supportive care, and 2% received radiotherapy alone.

Seventy-one percent of patients who received chemotherapy had an anthracycline-containing regimen, 13% received etoposide-containing chemotherapy, 9% received chemotherapy containing an anthracycline and etoposide, and 7% of patients received other therapy.

Thirteen percent of patients received a transplant as salvage treatment, and 7% received a transplant as consolidation.

Outcomes

Data on patient responses to initial treatment were available for 888 individuals. The 84 patients who received best supportive care were not included, and 50 patients were not evaluable for response.

The complete response/unconfirmed complete response rate was 53%, and the partial response rate was 19%. Twenty-eight percent of patients had no response or progressed.

The median PFS was 16 months. The 5-year PFS rate was 32% overall, 23% for PTCL-NOS, 28% for AITL, 39% for ALK- ALCL, and 57% for ALK+ ALCL.

The median OS was 36 months. The 5-year OS was 42% overall, 34% for PTCL-NOS, 42% for AITL, 46% for ALK- ALCL, and 76% for ALK+ ALCL.

Dr Federico and his colleagues have used these data to develop a prognostic model for PTCL-NOS that, they say, is more accurate than current models.

Massimo Federico, MD
Photo by Larry Young

SAN FRANCISCO—The T-Cell Project has provided information that can enhance our understanding of T-cell lymphomas, according to a presentation at the 9th Annual T-cell Lymphoma Forum.

The project is a prospective registry that includes data from T-cell lymphoma patients in 15 countries located in 5 different regions of the world. 

The data showed that peripheral T-cell lymphoma not otherwise specified (PTCL-NOS) is the most common subtype of T-cell lymphoma in all 5 regions, although the distribution of other subtypes varies.

A majority of patients in the registry received chemotherapy as induction, and anthracycline-containing regimens were the most popular treatment choice.

Although 60% of patients in the registry had low-risk or low/intermediate-risk disease, progression-free survival (PFS) and overall survival (OS) rates were low. The 5-year PFS was 32%, and the 5-year OS was 42%.

Massimo Federico, MD, of the University of Modena and Reggio Emilia in Italy, presented these data at the meeting.

About the project

Dr Federico said the goals of the T-Cell Project are to determine if prospective data collection provides more accurate information to better define prognosis of the most frequent subtypes of T-cell lymphoma and to improve our knowledge of clinical and biological characteristics, as well as outcomes, of the more uncommon subtypes.

“Why did we choose to propose a prospective registry for the collection of information in T-cell lymphoma?” Dr Federico asked. “Because it is, by far, less expensive than a clinical trial, but also because it can offer excellent data for generating new research programs and is a great opportunity for academic cooperation.”

As of December 31, 2016, the registry included 1523 patients. There were 75 sites (with at least 1 patient) active in the registry.

Fifteen countries in 5 geographic regions were represented. Europe was the greatest contributor (44%), followed by North America (US only, 25%), South America (20%), the Far East (9%), the Middle East (2%), and Oceania (<1%).

Subtypes

Overall, the distribution of the different T-cell lymphoma subtypes is as follows:

PTCL-NOS—36%
Angioimmunoblastic T-cell lymphoma (AITL)—17%
ALK- anaplastic large-cell lymphoma (ALCL)—16%
NK/T-cell lymphoma (NKTCL)—11%
ALK+ ALCL—8%
Enteropathy-associated T-cell lymphoma—4%
Unclassifiable T-cell lymphoma—3%
Hepatosplenic T-cell lymphoma—2%
Subcutaneous panniculitis-like T-cell lymphoma—2%
Peripheral gamma delta T-cell lymphoma—1%

Geographic distribution

The most common T-cell lymphoma subtypes in Europe were PTCL-NOS (37%), AITL (21%), and ALK- ALCL (14%). Likewise, the most common subtypes in the US were PTCL-NOS (35%), AITL (21%), and ALK- ALCL (13%).

In the Middle East, the most common subtypes were PTCL-NOS (40%), AITL (16%), and ALK+ ALCL (13%). In South America, they were PTCL-NOS (41%), ALK- ALCL (26%), and NKTCL (10%). And in Asia, they were PTCL-NOS (29%), NKTCL (29%), and AITL (17%).

Patient characteristics

Dr Federico presented data on patient characteristics for 1391 individuals, validated as of April 30, 2016.

The patients’ median age was 56 (range, 18-89). Forty-four percent were 60 or older, and 60% were male. Twenty-six percent had ECOG performance status > 1, 50% had B symptoms, and 72% had disease-related discomfort.

Sixty percent had low-risk or low/intermediate-risk disease according to the International Prognostic Index (IPI) and the Prognostic Index for T-cell Lymphoma (PIT).

Treatment

Treatment details are available for 1022 patients. Ninety-two percent received therapy with curative intent.

For induction, 76% of patients received chemotherapy alone, 14% received chemotherapy and radiotherapy, 8% received best supportive care, and 2% received radiotherapy alone.

Seventy-one percent of patients who received chemotherapy had an anthracycline-containing regimen, 13% received etoposide-containing chemotherapy, 9% received chemotherapy containing an anthracycline and etoposide, and 7% of patients received other therapy.

Thirteen percent of patients received a transplant as salvage treatment, and 7% received a transplant as consolidation.

Outcomes

Data on patient responses to initial treatment were available for 888 individuals. The 84 patients who received best supportive care were not included, and 50 patients were not evaluable for response.

The complete response/unconfirmed complete response rate was 53%, and the partial response rate was 19%. Twenty-eight percent of patients had no response or progressed.

The median PFS was 16 months. The 5-year PFS rate was 32% overall, 23% for PTCL-NOS, 28% for AITL, 39% for ALK- ALCL, and 57% for ALK+ ALCL.

The median OS was 36 months. The 5-year OS was 42% overall, 34% for PTCL-NOS, 42% for AITL, 46% for ALK- ALCL, and 76% for ALK+ ALCL.

Dr Federico and his colleagues have used these data to develop a prognostic model for PTCL-NOS that, they say, is more accurate than current models.

Massimo Federico, MD
Photo by Larry Young

SAN FRANCISCO—The T-Cell Project has provided information that can enhance our understanding of T-cell lymphomas, according to a presentation at the 9th Annual T-cell Lymphoma Forum.

The project is a prospective registry that includes data from T-cell lymphoma patients in 15 countries located in 5 different regions of the world. 

The data showed that peripheral T-cell lymphoma not otherwise specified (PTCL-NOS) is the most common subtype of T-cell lymphoma in all 5 regions, although the distribution of other subtypes varies.

A majority of patients in the registry received chemotherapy as induction, and anthracycline-containing regimens were the most popular treatment choice.

Although 60% of patients in the registry had low-risk or low/intermediate-risk disease, progression-free survival (PFS) and overall survival (OS) rates were low. The 5-year PFS was 32%, and the 5-year OS was 42%.

Massimo Federico, MD, of the University of Modena and Reggio Emilia in Italy, presented these data at the meeting.

About the project

Dr Federico said the goals of the T-Cell Project are to determine if prospective data collection provides more accurate information to better define prognosis of the most frequent subtypes of T-cell lymphoma and to improve our knowledge of clinical and biological characteristics, as well as outcomes, of the more uncommon subtypes.

“Why did we choose to propose a prospective registry for the collection of information in T-cell lymphoma?” Dr Federico asked. “Because it is, by far, less expensive than a clinical trial, but also because it can offer excellent data for generating new research programs and is a great opportunity for academic cooperation.”

As of December 31, 2016, the registry included 1523 patients. There were 75 sites (with at least 1 patient) active in the registry.

Fifteen countries in 5 geographic regions were represented. Europe was the greatest contributor (44%), followed by North America (US only, 25%), South America (20%), the Far East (9%), the Middle East (2%), and Oceania (<1%).

Subtypes

Overall, the distribution of the different T-cell lymphoma subtypes is as follows:

PTCL-NOS—36%
Angioimmunoblastic T-cell lymphoma (AITL)—17%
ALK- anaplastic large-cell lymphoma (ALCL)—16%
NK/T-cell lymphoma (NKTCL)—11%
ALK+ ALCL—8%
Enteropathy-associated T-cell lymphoma—4%
Unclassifiable T-cell lymphoma—3%
Hepatosplenic T-cell lymphoma—2%
Subcutaneous panniculitis-like T-cell lymphoma—2%
Peripheral gamma delta T-cell lymphoma—1%

Geographic distribution

The most common T-cell lymphoma subtypes in Europe were PTCL-NOS (37%), AITL (21%), and ALK- ALCL (14%). Likewise, the most common subtypes in the US were PTCL-NOS (35%), AITL (21%), and ALK- ALCL (13%).

In the Middle East, the most common subtypes were PTCL-NOS (40%), AITL (16%), and ALK+ ALCL (13%). In South America, they were PTCL-NOS (41%), ALK- ALCL (26%), and NKTCL (10%). And in Asia, they were PTCL-NOS (29%), NKTCL (29%), and AITL (17%).

Patient characteristics

Dr Federico presented data on patient characteristics for 1391 individuals, validated as of April 30, 2016.

The patients’ median age was 56 (range, 18-89). Forty-four percent were 60 or older, and 60% were male. Twenty-six percent had ECOG performance status > 1, 50% had B symptoms, and 72% had disease-related discomfort.

Sixty percent had low-risk or low/intermediate-risk disease according to the International Prognostic Index (IPI) and the Prognostic Index for T-cell Lymphoma (PIT).

Treatment

Treatment details are available for 1022 patients. Ninety-two percent received therapy with curative intent.

For induction, 76% of patients received chemotherapy alone, 14% received chemotherapy and radiotherapy, 8% received best supportive care, and 2% received radiotherapy alone.

Seventy-one percent of patients who received chemotherapy had an anthracycline-containing regimen, 13% received etoposide-containing chemotherapy, 9% received chemotherapy containing an anthracycline and etoposide, and 7% of patients received other therapy.

Thirteen percent of patients received a transplant as salvage treatment, and 7% received a transplant as consolidation.

Outcomes

Data on patient responses to initial treatment were available for 888 individuals. The 84 patients who received best supportive care were not included, and 50 patients were not evaluable for response.

The complete response/unconfirmed complete response rate was 53%, and the partial response rate was 19%. Twenty-eight percent of patients had no response or progressed.

The median PFS was 16 months. The 5-year PFS rate was 32% overall, 23% for PTCL-NOS, 28% for AITL, 39% for ALK- ALCL, and 57% for ALK+ ALCL.

The median OS was 36 months. The 5-year OS was 42% overall, 34% for PTCL-NOS, 42% for AITL, 46% for ALK- ALCL, and 76% for ALK+ ALCL.

Dr Federico and his colleagues have used these data to develop a prognostic model for PTCL-NOS that, they say, is more accurate than current models.

Prescription drugs
Photo by Steven Harbour

AMSTERDAM—The UK has seen substantial price increases for some generic cancer drugs over the last few years, according to a study presented at ECCO 2017: European Cancer Congress (abstract 966).

Of the 89 drugs analyzed in this study, 21 of them—including 17 generics—had price increases from 2011 to 2016.

Fourteen of the generic cancer drugs had price increases over 100%, and 2 of the drugs had increases exceeding 1000%.

“We were surprised to find several companies consistently raising the prices of cancer treatment,” said study investigator Andrew Hill, PhD, of the University of Liverpool in the UK.

“Twenty treatments have shown rises of over 100% in the last 5 years, and in 2—busulfan (used to treat leukemia) and tamoxifen (breast cancer)—prices have increased by over 1000%. We have found that some companies take over the supply of some generic cancer medicines and then raise the price progressively.”

Dr Hill and his co-investigator Melissa Barber, of the London School of Hygiene and Tropical Medicine in the UK, analyzed prices for 190 formulations of 89 cancer drugs.

Twenty-eight formulations of 21 drugs had price increases from 2011 to 2016. Seventeen of these 21 drugs were generic in 2016.

Twenty formulations of 14 generic cancer drugs had price increases exceeding 100%.

For example, the cost per tablet or injection increased for:

• Ifosfamide (2 g vial)—from £89 to £180, or 103%.
• Melphalan (50 mg vial)—from £33 to £137, or 315%.
• Chlorambucil (2 mg)—from £0.33 to £1.62, or 390%.
• Cyclophosphamide (50 mg)—from £0.20 to £1.39, or 695%.
• Busulfan (2 mg)—from £0.21 to £2.61, or 1227%.

Dr Hill said the UK’s Department of Health is aware of this issue and has introduced the Health Services Medical Supplies (Costs) Bill to enable price regulation in the future.

Companies found to be raising prices with no clear justification will be referred to the Competition and Markets Authority, and they could face fines.

However, Dr Hill and Barber said they found large price increases for generic cancer drugs in other European countries as well.

In Spain and Italy, failure to accept the high prices demanded for some generic drugs has led to warnings from companies that they could stop the supply of these drugs.

For instance, Italy fined the generic company Aspen €5 million after a 1500% increase in the price of cancer drugs, including melphalan and chlorambucil. Aspen then threatened Italy with drug shortages unless higher prices were accepted. 

In Spain, Aspen demanded a 4000% increase in melphalan prices.

“We hope that, by explaining what we have found in the UK, other European countries will take note and protect themselves against these kinds of price rises,” Dr Hill said. “At a time when cancer patients are living longer and better lives due to effective treatments, this situation is particularly worrying.”

Prescription drugs
Photo by Steven Harbour

AMSTERDAM—The UK has seen substantial price increases for some generic cancer drugs over the last few years, according to a study presented at ECCO 2017: European Cancer Congress (abstract 966).

Of the 89 drugs analyzed in this study, 21 of them—including 17 generics—had price increases from 2011 to 2016.

Fourteen of the generic cancer drugs had price increases over 100%, and 2 of the drugs had increases exceeding 1000%.

“We were surprised to find several companies consistently raising the prices of cancer treatment,” said study investigator Andrew Hill, PhD, of the University of Liverpool in the UK.

“Twenty treatments have shown rises of over 100% in the last 5 years, and in 2—busulfan (used to treat leukemia) and tamoxifen (breast cancer)—prices have increased by over 1000%. We have found that some companies take over the supply of some generic cancer medicines and then raise the price progressively.”

Dr Hill and his co-investigator Melissa Barber, of the London School of Hygiene and Tropical Medicine in the UK, analyzed prices for 190 formulations of 89 cancer drugs.

Twenty-eight formulations of 21 drugs had price increases from 2011 to 2016. Seventeen of these 21 drugs were generic in 2016.

Twenty formulations of 14 generic cancer drugs had price increases exceeding 100%.

For example, the cost per tablet or injection increased for:

• Ifosfamide (2 g vial)—from £89 to £180, or 103%.
• Melphalan (50 mg vial)—from £33 to £137, or 315%.
• Chlorambucil (2 mg)—from £0.33 to £1.62, or 390%.
• Cyclophosphamide (50 mg)—from £0.20 to £1.39, or 695%.
• Busulfan (2 mg)—from £0.21 to £2.61, or 1227%.

Dr Hill said the UK’s Department of Health is aware of this issue and has introduced the Health Services Medical Supplies (Costs) Bill to enable price regulation in the future.

Companies found to be raising prices with no clear justification will be referred to the Competition and Markets Authority, and they could face fines.

However, Dr Hill and Barber said they found large price increases for generic cancer drugs in other European countries as well.

In Spain and Italy, failure to accept the high prices demanded for some generic drugs has led to warnings from companies that they could stop the supply of these drugs.

For instance, Italy fined the generic company Aspen €5 million after a 1500% increase in the price of cancer drugs, including melphalan and chlorambucil. Aspen then threatened Italy with drug shortages unless higher prices were accepted. 

In Spain, Aspen demanded a 4000% increase in melphalan prices.

“We hope that, by explaining what we have found in the UK, other European countries will take note and protect themselves against these kinds of price rises,” Dr Hill said. “At a time when cancer patients are living longer and better lives due to effective treatments, this situation is particularly worrying.”

Prescription drugs
Photo by Steven Harbour

AMSTERDAM—The UK has seen substantial price increases for some generic cancer drugs over the last few years, according to a study presented at ECCO 2017: European Cancer Congress (abstract 966).

Of the 89 drugs analyzed in this study, 21 of them—including 17 generics—had price increases from 2011 to 2016.

Fourteen of the generic cancer drugs had price increases over 100%, and 2 of the drugs had increases exceeding 1000%.

“We were surprised to find several companies consistently raising the prices of cancer treatment,” said study investigator Andrew Hill, PhD, of the University of Liverpool in the UK.

“Twenty treatments have shown rises of over 100% in the last 5 years, and in 2—busulfan (used to treat leukemia) and tamoxifen (breast cancer)—prices have increased by over 1000%. We have found that some companies take over the supply of some generic cancer medicines and then raise the price progressively.”

Dr Hill and his co-investigator Melissa Barber, of the London School of Hygiene and Tropical Medicine in the UK, analyzed prices for 190 formulations of 89 cancer drugs.

Twenty-eight formulations of 21 drugs had price increases from 2011 to 2016. Seventeen of these 21 drugs were generic in 2016.

Twenty formulations of 14 generic cancer drugs had price increases exceeding 100%.

For example, the cost per tablet or injection increased for:

• Ifosfamide (2 g vial)—from £89 to £180, or 103%.
• Melphalan (50 mg vial)—from £33 to £137, or 315%.
• Chlorambucil (2 mg)—from £0.33 to £1.62, or 390%.
• Cyclophosphamide (50 mg)—from £0.20 to £1.39, or 695%.
• Busulfan (2 mg)—from £0.21 to £2.61, or 1227%.

Dr Hill said the UK’s Department of Health is aware of this issue and has introduced the Health Services Medical Supplies (Costs) Bill to enable price regulation in the future.

Companies found to be raising prices with no clear justification will be referred to the Competition and Markets Authority, and they could face fines.

However, Dr Hill and Barber said they found large price increases for generic cancer drugs in other European countries as well.

In Spain and Italy, failure to accept the high prices demanded for some generic drugs has led to warnings from companies that they could stop the supply of these drugs.

For instance, Italy fined the generic company Aspen €5 million after a 1500% increase in the price of cancer drugs, including melphalan and chlorambucil. Aspen then threatened Italy with drug shortages unless higher prices were accepted. 

In Spain, Aspen demanded a 4000% increase in melphalan prices.

“We hope that, by explaining what we have found in the UK, other European countries will take note and protect themselves against these kinds of price rises,” Dr Hill said. “At a time when cancer patients are living longer and better lives due to effective treatments, this situation is particularly worrying.”

Blood donation

The Swiss Agency for Therapeutic Products (Swissmedic) has agreed to lift the ban on blood donations from men who have sex with men (MSM).

Instead, MSMs will be allowed to donate blood if it has been at least 12 months since their last sexual contact with another man.

Swiss Transfusion SRC Inc. expects to implement the modified donation criteria for MSMs in regional blood transfusion services starting in mid-2017.

However, Swissmedic’s decision is subject to certain conditions.

Specifically, blood transfusion services will have to record additional data on the effects of the modified donation criteria and donors’ compliance with them, as well as closely monitor the risk trend.

Swissmedic said that, since January 2016, the tests for specific pathogens in donated blood in Switzerland have been further refined, resulting in a higher level of sensitivity.

The diagnostic window—the period in which any infections carried by blood donors cannot yet be discovered—for the relevant pathogens has been further narrowed. Depending on the virus, the diagnostic window is 3 days to 15 days after infection.

Therefore, Swissmedic believes that a 12-month deferral period for MSM blood donors would not expose recipients of blood transfusions to an increased risk of contracting a blood-borne infection.

Swissmedic noted that approximately half of all new HIV infections in Switzerland are attributable to MSMs. This is one of the reasons MSMs have been permanently excluded from giving blood since 1977.

The new 12-month deferral period for MSMs is in line with the precautionary measures applicable to various other behaviors that have been shown to increase the risk of HIV transmission, such as changing sexual partners, staying in countries with a high AIDS rate, and sexual contact with partners who have stayed in countries with a high AIDS rate for a lengthy period.

The change to a 1-year deferral period for MSM blood donors brings Switzerland into line with other nations that have adopted similar policies, such as Ireland, Canada, the US, and the UK.

Blood donation

The Swiss Agency for Therapeutic Products (Swissmedic) has agreed to lift the ban on blood donations from men who have sex with men (MSM).

Instead, MSMs will be allowed to donate blood if it has been at least 12 months since their last sexual contact with another man.

Swiss Transfusion SRC Inc. expects to implement the modified donation criteria for MSMs in regional blood transfusion services starting in mid-2017.

However, Swissmedic’s decision is subject to certain conditions.

Specifically, blood transfusion services will have to record additional data on the effects of the modified donation criteria and donors’ compliance with them, as well as closely monitor the risk trend.

Swissmedic said that, since January 2016, the tests for specific pathogens in donated blood in Switzerland have been further refined, resulting in a higher level of sensitivity.

The diagnostic window—the period in which any infections carried by blood donors cannot yet be discovered—for the relevant pathogens has been further narrowed. Depending on the virus, the diagnostic window is 3 days to 15 days after infection.

Therefore, Swissmedic believes that a 12-month deferral period for MSM blood donors would not expose recipients of blood transfusions to an increased risk of contracting a blood-borne infection.

Swissmedic noted that approximately half of all new HIV infections in Switzerland are attributable to MSMs. This is one of the reasons MSMs have been permanently excluded from giving blood since 1977.

The new 12-month deferral period for MSMs is in line with the precautionary measures applicable to various other behaviors that have been shown to increase the risk of HIV transmission, such as changing sexual partners, staying in countries with a high AIDS rate, and sexual contact with partners who have stayed in countries with a high AIDS rate for a lengthy period.

The change to a 1-year deferral period for MSM blood donors brings Switzerland into line with other nations that have adopted similar policies, such as Ireland, Canada, the US, and the UK.

Blood donation

The Swiss Agency for Therapeutic Products (Swissmedic) has agreed to lift the ban on blood donations from men who have sex with men (MSM).

Instead, MSMs will be allowed to donate blood if it has been at least 12 months since their last sexual contact with another man.

Swiss Transfusion SRC Inc. expects to implement the modified donation criteria for MSMs in regional blood transfusion services starting in mid-2017.

However, Swissmedic’s decision is subject to certain conditions.

Specifically, blood transfusion services will have to record additional data on the effects of the modified donation criteria and donors’ compliance with them, as well as closely monitor the risk trend.

Swissmedic said that, since January 2016, the tests for specific pathogens in donated blood in Switzerland have been further refined, resulting in a higher level of sensitivity.

The diagnostic window—the period in which any infections carried by blood donors cannot yet be discovered—for the relevant pathogens has been further narrowed. Depending on the virus, the diagnostic window is 3 days to 15 days after infection.

Therefore, Swissmedic believes that a 12-month deferral period for MSM blood donors would not expose recipients of blood transfusions to an increased risk of contracting a blood-borne infection.

Swissmedic noted that approximately half of all new HIV infections in Switzerland are attributable to MSMs. This is one of the reasons MSMs have been permanently excluded from giving blood since 1977.

The new 12-month deferral period for MSMs is in line with the precautionary measures applicable to various other behaviors that have been shown to increase the risk of HIV transmission, such as changing sexual partners, staying in countries with a high AIDS rate, and sexual contact with partners who have stayed in countries with a high AIDS rate for a lengthy period.

The change to a 1-year deferral period for MSM blood donors brings Switzerland into line with other nations that have adopted similar policies, such as Ireland, Canada, the US, and the UK.