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EC grants drug orphan designation for CTCL
The European Commission (EC) has granted orphan designation to MRG-106 for the treatment of cutaneous T-cell lymphoma (CTCL).
MRG-106 is a locked nucleic acid-modified oligonucleotide inhibitor of miR-155-5p.
miRagen Therapeutics, Inc., the company developing MRG-106, is currently testing the drug in a phase 1 trial of CTCL patients.
Early results from this trial were presented at the 2016 ASH Annual Meeting.
Researchers presented results in 6 patients with stage I-III mycosis fungoides.
The patients received 4 or 5 intratumoral injections of MRG-106 (at 75 mg) over 2 weeks. Four patients received saline injections in a second lesion on the same schedule.
There were 3 adverse events related to MRG-106—pain during injection, burning sensation during injection, and tingling at the injection site.
Adverse events considered possibly related to MRG-106 were pruritus, erythema, skin inflammation, sore on hand, nausea, decrease in white blood cells, neutropenia, and prolonged partial thromboplastin time.
One patient was taken off the trial due to rapid disease progression. The other 5 patients completed the dosing period.
All 5 patients had a reduction in the baseline Composite Assessment of Index Lesion Severity score in MRG-106-treated and saline-treated lesions.
The average maximal reduction was 55% (range, 33% to 77%) in MRG-106-treated lesions and 39% (range, 13% to 75%) in saline-treated lesions.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission. 
The European Commission (EC) has granted orphan designation to MRG-106 for the treatment of cutaneous T-cell lymphoma (CTCL).
MRG-106 is a locked nucleic acid-modified oligonucleotide inhibitor of miR-155-5p.
miRagen Therapeutics, Inc., the company developing MRG-106, is currently testing the drug in a phase 1 trial of CTCL patients.
Early results from this trial were presented at the 2016 ASH Annual Meeting.
Researchers presented results in 6 patients with stage I-III mycosis fungoides.
The patients received 4 or 5 intratumoral injections of MRG-106 (at 75 mg) over 2 weeks. Four patients received saline injections in a second lesion on the same schedule.
There were 3 adverse events related to MRG-106—pain during injection, burning sensation during injection, and tingling at the injection site.
Adverse events considered possibly related to MRG-106 were pruritus, erythema, skin inflammation, sore on hand, nausea, decrease in white blood cells, neutropenia, and prolonged partial thromboplastin time.
One patient was taken off the trial due to rapid disease progression. The other 5 patients completed the dosing period.
All 5 patients had a reduction in the baseline Composite Assessment of Index Lesion Severity score in MRG-106-treated and saline-treated lesions.
The average maximal reduction was 55% (range, 33% to 77%) in MRG-106-treated lesions and 39% (range, 13% to 75%) in saline-treated lesions.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission.
The European Commission (EC) has granted orphan designation to MRG-106 for the treatment of cutaneous T-cell lymphoma (CTCL).
MRG-106 is a locked nucleic acid-modified oligonucleotide inhibitor of miR-155-5p.
miRagen Therapeutics, Inc., the company developing MRG-106, is currently testing the drug in a phase 1 trial of CTCL patients.
Early results from this trial were presented at the 2016 ASH Annual Meeting.
Researchers presented results in 6 patients with stage I-III mycosis fungoides.
The patients received 4 or 5 intratumoral injections of MRG-106 (at 75 mg) over 2 weeks. Four patients received saline injections in a second lesion on the same schedule.
There were 3 adverse events related to MRG-106—pain during injection, burning sensation during injection, and tingling at the injection site.
Adverse events considered possibly related to MRG-106 were pruritus, erythema, skin inflammation, sore on hand, nausea, decrease in white blood cells, neutropenia, and prolonged partial thromboplastin time.
One patient was taken off the trial due to rapid disease progression. The other 5 patients completed the dosing period.
All 5 patients had a reduction in the baseline Composite Assessment of Index Lesion Severity score in MRG-106-treated and saline-treated lesions.
The average maximal reduction was 55% (range, 33% to 77%) in MRG-106-treated lesions and 39% (range, 13% to 75%) in saline-treated lesions.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission.
Assay available to screen donated blood for Zika
Blood banks in countries that accept the CE mark can now use the Procleix Zika Virus Assay to screen blood donations for the presence of Zika virus.
CE marking means a product conforms to relevant legislation for sale in the European economic area.
The Procleix Zika Virus Assay, which was developed by Hologic, Inc. and Grifols, is designed to run on the Procleix Panther System, an automated, nucleic acid technology (NAT) blood screening platform.
NAT enables detection of infectious agents in blood and plasma donations.
The Procleix Panther system automates all aspects of NAT-based blood screening on a single, integrated platform.
The system has received regulatory approvals in countries around the world, and it is in development for the US market.
In the US, the Procleix Zika Virus Assay is being used under an investigational new drug protocol in response to the US Food and Drug Administration’s recommendation to screen all US blood donations for Zika virus.
“The CE marking of the Procleix Zika virus assay is a further step in our mission to support safer blood donations, the result of our passion for innovation and the role we play as market leaders in transfusion medicine,” said Grifols Diagnostic Division President Carsten Schroeder.
About Zika virus
Zika is a mosquito-borne virus that was first identified in rhesus monkeys in Uganda in 1947 and in humans in 1952. Outbreaks of Zika virus have been recorded in Africa, the Americas, Asia, and the Pacific.
Zika virus is transmitted to humans primarily through the bite of an infected mosquito from the Aedes genus, mainly Aedes aegypti, in tropical regions.
Sexual transmission of Zika virus is also possible, and reports have suggested Zika can be transmitted via transfusion of blood products. Other modes of transmission are being investigated as well.
In total, 64 countries and territories have reported transmission of Zika virus since January 1, 2007.
Blood banks in countries that accept the CE mark can now use the Procleix Zika Virus Assay to screen blood donations for the presence of Zika virus.
CE marking means a product conforms to relevant legislation for sale in the European economic area.
The Procleix Zika Virus Assay, which was developed by Hologic, Inc. and Grifols, is designed to run on the Procleix Panther System, an automated, nucleic acid technology (NAT) blood screening platform.
NAT enables detection of infectious agents in blood and plasma donations.
The Procleix Panther system automates all aspects of NAT-based blood screening on a single, integrated platform.
The system has received regulatory approvals in countries around the world, and it is in development for the US market.
In the US, the Procleix Zika Virus Assay is being used under an investigational new drug protocol in response to the US Food and Drug Administration’s recommendation to screen all US blood donations for Zika virus.
“The CE marking of the Procleix Zika virus assay is a further step in our mission to support safer blood donations, the result of our passion for innovation and the role we play as market leaders in transfusion medicine,” said Grifols Diagnostic Division President Carsten Schroeder.
About Zika virus
Zika is a mosquito-borne virus that was first identified in rhesus monkeys in Uganda in 1947 and in humans in 1952. Outbreaks of Zika virus have been recorded in Africa, the Americas, Asia, and the Pacific.
Zika virus is transmitted to humans primarily through the bite of an infected mosquito from the Aedes genus, mainly Aedes aegypti, in tropical regions.
Sexual transmission of Zika virus is also possible, and reports have suggested Zika can be transmitted via transfusion of blood products. Other modes of transmission are being investigated as well.
In total, 64 countries and territories have reported transmission of Zika virus since January 1, 2007.
Blood banks in countries that accept the CE mark can now use the Procleix Zika Virus Assay to screen blood donations for the presence of Zika virus.
CE marking means a product conforms to relevant legislation for sale in the European economic area.
The Procleix Zika Virus Assay, which was developed by Hologic, Inc. and Grifols, is designed to run on the Procleix Panther System, an automated, nucleic acid technology (NAT) blood screening platform.
NAT enables detection of infectious agents in blood and plasma donations.
The Procleix Panther system automates all aspects of NAT-based blood screening on a single, integrated platform.
The system has received regulatory approvals in countries around the world, and it is in development for the US market.
In the US, the Procleix Zika Virus Assay is being used under an investigational new drug protocol in response to the US Food and Drug Administration’s recommendation to screen all US blood donations for Zika virus.
“The CE marking of the Procleix Zika virus assay is a further step in our mission to support safer blood donations, the result of our passion for innovation and the role we play as market leaders in transfusion medicine,” said Grifols Diagnostic Division President Carsten Schroeder.
About Zika virus
Zika is a mosquito-borne virus that was first identified in rhesus monkeys in Uganda in 1947 and in humans in 1952. Outbreaks of Zika virus have been recorded in Africa, the Americas, Asia, and the Pacific.
Zika virus is transmitted to humans primarily through the bite of an infected mosquito from the Aedes genus, mainly Aedes aegypti, in tropical regions.
Sexual transmission of Zika virus is also possible, and reports have suggested Zika can be transmitted via transfusion of blood products. Other modes of transmission are being investigated as well.
In total, 64 countries and territories have reported transmission of Zika virus since January 1, 2007.
EMA recommends orphan designation for AML drug
The European Medicines Agency (EMA) has recommended orphan designation for Actimab-A, a product intended to treat patients with newly diagnosed acute myeloid leukemia (AML) who are over the age of 60 and are ineligible for standard induction therapy.
Actimab-A targets CD33, a protein expressed on the surface of AML cells, via the monoclonal antibody, HuM195, which carries the cytotoxic radioisotope actinium-225 to the AML cells.
Actinium Pharmaceuticals, Inc., the company developing Actimab-A, is testing the drug in a phase 2 trial.
Results from a phase 1 trial of the drug were presented at the 2016 ASH Annual Meeting.
At that time, researchers reported results in 18 patients who had been newly diagnosed with AML and were age 60 and older. Their median age was 77 (range, 68-87).
The patients received Actimab-A in combination with low-dose cytarabine. Actimab-A was given at 0.5 μCi/kg/fraction (n=3), 1 μCi/kg/fraction (n=6), 1.5 μCi/kg/fraction (n=3), or 2 μCi/kg/fraction (n=6).
Two patients experienced dose-limiting toxicities. Both had grade 4 thrombocytopenia with marrow aplasia for more than 6 weeks after therapy. One patient was in the 1 µCi/kg/fraction cohort, and the other was in the 2 µCi/kg/fraction cohort.
The maximum-tolerated dose was not reached, but 2 µCi/kg/fraction was chosen as the phase 2 dose.
Grade 3/4 toxicities included neutropenia (n=5), thrombocytopenia (n=9), febrile neutropenia (n=6), pneumonia (n=5), other infections (n=3), atrial fibrillation/syncope (n=1), transient creatinine increase (n=1), generalized fatigue (n=1), hypokalemia (n=1), mucositis (n=1), and rectal hemorrhage (n=1).
Twenty-eight percent of patients (5/18) had objective responses to treatment. Two patients achieved a complete response (CR), 1 had a CR with incomplete platelet recovery, and 2 had a CR with incomplete marrow recovery.
The median duration of response was 9.1 months (range, 4.1-16.9).
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval. The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The European Commission typically makes a decision within 30 days.
The European Medicines Agency (EMA) has recommended orphan designation for Actimab-A, a product intended to treat patients with newly diagnosed acute myeloid leukemia (AML) who are over the age of 60 and are ineligible for standard induction therapy.
Actimab-A targets CD33, a protein expressed on the surface of AML cells, via the monoclonal antibody, HuM195, which carries the cytotoxic radioisotope actinium-225 to the AML cells.
Actinium Pharmaceuticals, Inc., the company developing Actimab-A, is testing the drug in a phase 2 trial.
Results from a phase 1 trial of the drug were presented at the 2016 ASH Annual Meeting.
At that time, researchers reported results in 18 patients who had been newly diagnosed with AML and were age 60 and older. Their median age was 77 (range, 68-87).
The patients received Actimab-A in combination with low-dose cytarabine. Actimab-A was given at 0.5 μCi/kg/fraction (n=3), 1 μCi/kg/fraction (n=6), 1.5 μCi/kg/fraction (n=3), or 2 μCi/kg/fraction (n=6).
Two patients experienced dose-limiting toxicities. Both had grade 4 thrombocytopenia with marrow aplasia for more than 6 weeks after therapy. One patient was in the 1 µCi/kg/fraction cohort, and the other was in the 2 µCi/kg/fraction cohort.
The maximum-tolerated dose was not reached, but 2 µCi/kg/fraction was chosen as the phase 2 dose.
Grade 3/4 toxicities included neutropenia (n=5), thrombocytopenia (n=9), febrile neutropenia (n=6), pneumonia (n=5), other infections (n=3), atrial fibrillation/syncope (n=1), transient creatinine increase (n=1), generalized fatigue (n=1), hypokalemia (n=1), mucositis (n=1), and rectal hemorrhage (n=1).
Twenty-eight percent of patients (5/18) had objective responses to treatment. Two patients achieved a complete response (CR), 1 had a CR with incomplete platelet recovery, and 2 had a CR with incomplete marrow recovery.
The median duration of response was 9.1 months (range, 4.1-16.9).
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval. The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The European Commission typically makes a decision within 30 days.
The European Medicines Agency (EMA) has recommended orphan designation for Actimab-A, a product intended to treat patients with newly diagnosed acute myeloid leukemia (AML) who are over the age of 60 and are ineligible for standard induction therapy.
Actimab-A targets CD33, a protein expressed on the surface of AML cells, via the monoclonal antibody, HuM195, which carries the cytotoxic radioisotope actinium-225 to the AML cells.
Actinium Pharmaceuticals, Inc., the company developing Actimab-A, is testing the drug in a phase 2 trial.
Results from a phase 1 trial of the drug were presented at the 2016 ASH Annual Meeting.
At that time, researchers reported results in 18 patients who had been newly diagnosed with AML and were age 60 and older. Their median age was 77 (range, 68-87).
The patients received Actimab-A in combination with low-dose cytarabine. Actimab-A was given at 0.5 μCi/kg/fraction (n=3), 1 μCi/kg/fraction (n=6), 1.5 μCi/kg/fraction (n=3), or 2 μCi/kg/fraction (n=6).
Two patients experienced dose-limiting toxicities. Both had grade 4 thrombocytopenia with marrow aplasia for more than 6 weeks after therapy. One patient was in the 1 µCi/kg/fraction cohort, and the other was in the 2 µCi/kg/fraction cohort.
The maximum-tolerated dose was not reached, but 2 µCi/kg/fraction was chosen as the phase 2 dose.
Grade 3/4 toxicities included neutropenia (n=5), thrombocytopenia (n=9), febrile neutropenia (n=6), pneumonia (n=5), other infections (n=3), atrial fibrillation/syncope (n=1), transient creatinine increase (n=1), generalized fatigue (n=1), hypokalemia (n=1), mucositis (n=1), and rectal hemorrhage (n=1).
Twenty-eight percent of patients (5/18) had objective responses to treatment. Two patients achieved a complete response (CR), 1 had a CR with incomplete platelet recovery, and 2 had a CR with incomplete marrow recovery.
The median duration of response was 9.1 months (range, 4.1-16.9).
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval. The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The European Commission typically makes a decision within 30 days.
Patients with thrombocytosis should be tested for cancers, team says
Researchers have found evidence to suggest that thrombocytosis is a strong predictor of cancer, particularly lung and colorectal cancer.
The team therefore believes patients with thrombocytosis should be evaluated for an underlying malignancy, as such investigation could speed up cancer diagnosis and save lives.
“We know that early diagnosis is absolutely key in whether people survive cancer,” said Sarah Bailey, PhD, of the University of Exeter Medical School in the UK.
“Our research suggests that substantial numbers of people could have their cancer diagnosed up to 3 months earlier if thrombocytosis prompted investigation for cancer. This time could make a vital difference in achieving earlier diagnosis.”
Dr Bailey and her colleagues described their research in the British Journal of General Practice.
The team conducted a prospective cohort study using Clinical Practice Research Datalink data spanning the period from 2000 to 2013.
They compared the 1-year cancer incidence in 40,000 patients (age 40 and older) with thrombocytosis (platelet count >400 × 109/L) and 10,000 matched controls with normal platelet counts.
Patients with thrombocytosis had a higher incidence of cancer than individuals with normal platelet counts.
The cancer incidence was 6.2% (1355/21,826) in women with thrombocytosis and 2.2% (119/5370) in women with normal platelet counts.
The cancer incidence was 11.6% (1098/9435) in men with thrombocytosis and 4.1% (106/2599) in men with normal platelet counts.
If patients in the thrombocytosis group had a second raised platelet count recorded within 6 months of their index date, the risk of cancer increased to 18.1% for men and 10.1% for women.
Lung and colorectal cancer were more common among patients with thrombocytosis than among individuals with normal platelet counts.
And about one-third of patients with thrombocytosis and lung/colorectal cancer had no other symptoms that would indicate they had cancer.
In addition, the researchers found that “substantial proportions” of lung/colorectal cancer diagnoses could be expedited if thrombocytosis were routinely investigated.
The team calculated that if 5% of patients with cancer have thrombocytosis before a cancer diagnosis, one-third of them have the potential to have their diagnosis expedited by at least 3 months if their doctor investigates the possibility of cancer based on the presence of thrombocytosis. This equates to 5500 earlier diagnoses annually.
“The UK lags well behind other developed countries on early cancer diagnosis,” said study author Willie Hamilton, MD, of the University of Exeter Medical School.
“In 2014, 163,000 people died of cancer in this country. Our findings on thrombocytosis show a strong association with cancer, particularly in men—far stronger than that of a breast lump for breast cancer in women. It is now crucial that we roll out cancer investigation of thrombocytosis. It could save hundreds of lives each year.”
Researchers have found evidence to suggest that thrombocytosis is a strong predictor of cancer, particularly lung and colorectal cancer.
The team therefore believes patients with thrombocytosis should be evaluated for an underlying malignancy, as such investigation could speed up cancer diagnosis and save lives.
“We know that early diagnosis is absolutely key in whether people survive cancer,” said Sarah Bailey, PhD, of the University of Exeter Medical School in the UK.
“Our research suggests that substantial numbers of people could have their cancer diagnosed up to 3 months earlier if thrombocytosis prompted investigation for cancer. This time could make a vital difference in achieving earlier diagnosis.”
Dr Bailey and her colleagues described their research in the British Journal of General Practice.
The team conducted a prospective cohort study using Clinical Practice Research Datalink data spanning the period from 2000 to 2013.
They compared the 1-year cancer incidence in 40,000 patients (age 40 and older) with thrombocytosis (platelet count >400 × 109/L) and 10,000 matched controls with normal platelet counts.
Patients with thrombocytosis had a higher incidence of cancer than individuals with normal platelet counts.
The cancer incidence was 6.2% (1355/21,826) in women with thrombocytosis and 2.2% (119/5370) in women with normal platelet counts.
The cancer incidence was 11.6% (1098/9435) in men with thrombocytosis and 4.1% (106/2599) in men with normal platelet counts.
If patients in the thrombocytosis group had a second raised platelet count recorded within 6 months of their index date, the risk of cancer increased to 18.1% for men and 10.1% for women.
Lung and colorectal cancer were more common among patients with thrombocytosis than among individuals with normal platelet counts.
And about one-third of patients with thrombocytosis and lung/colorectal cancer had no other symptoms that would indicate they had cancer.
In addition, the researchers found that “substantial proportions” of lung/colorectal cancer diagnoses could be expedited if thrombocytosis were routinely investigated.
The team calculated that if 5% of patients with cancer have thrombocytosis before a cancer diagnosis, one-third of them have the potential to have their diagnosis expedited by at least 3 months if their doctor investigates the possibility of cancer based on the presence of thrombocytosis. This equates to 5500 earlier diagnoses annually.
“The UK lags well behind other developed countries on early cancer diagnosis,” said study author Willie Hamilton, MD, of the University of Exeter Medical School.
“In 2014, 163,000 people died of cancer in this country. Our findings on thrombocytosis show a strong association with cancer, particularly in men—far stronger than that of a breast lump for breast cancer in women. It is now crucial that we roll out cancer investigation of thrombocytosis. It could save hundreds of lives each year.”
Researchers have found evidence to suggest that thrombocytosis is a strong predictor of cancer, particularly lung and colorectal cancer.
The team therefore believes patients with thrombocytosis should be evaluated for an underlying malignancy, as such investigation could speed up cancer diagnosis and save lives.
“We know that early diagnosis is absolutely key in whether people survive cancer,” said Sarah Bailey, PhD, of the University of Exeter Medical School in the UK.
“Our research suggests that substantial numbers of people could have their cancer diagnosed up to 3 months earlier if thrombocytosis prompted investigation for cancer. This time could make a vital difference in achieving earlier diagnosis.”
Dr Bailey and her colleagues described their research in the British Journal of General Practice.
The team conducted a prospective cohort study using Clinical Practice Research Datalink data spanning the period from 2000 to 2013.
They compared the 1-year cancer incidence in 40,000 patients (age 40 and older) with thrombocytosis (platelet count >400 × 109/L) and 10,000 matched controls with normal platelet counts.
Patients with thrombocytosis had a higher incidence of cancer than individuals with normal platelet counts.
The cancer incidence was 6.2% (1355/21,826) in women with thrombocytosis and 2.2% (119/5370) in women with normal platelet counts.
The cancer incidence was 11.6% (1098/9435) in men with thrombocytosis and 4.1% (106/2599) in men with normal platelet counts.
If patients in the thrombocytosis group had a second raised platelet count recorded within 6 months of their index date, the risk of cancer increased to 18.1% for men and 10.1% for women.
Lung and colorectal cancer were more common among patients with thrombocytosis than among individuals with normal platelet counts.
And about one-third of patients with thrombocytosis and lung/colorectal cancer had no other symptoms that would indicate they had cancer.
In addition, the researchers found that “substantial proportions” of lung/colorectal cancer diagnoses could be expedited if thrombocytosis were routinely investigated.
The team calculated that if 5% of patients with cancer have thrombocytosis before a cancer diagnosis, one-third of them have the potential to have their diagnosis expedited by at least 3 months if their doctor investigates the possibility of cancer based on the presence of thrombocytosis. This equates to 5500 earlier diagnoses annually.
“The UK lags well behind other developed countries on early cancer diagnosis,” said study author Willie Hamilton, MD, of the University of Exeter Medical School.
“In 2014, 163,000 people died of cancer in this country. Our findings on thrombocytosis show a strong association with cancer, particularly in men—far stronger than that of a breast lump for breast cancer in women. It is now crucial that we roll out cancer investigation of thrombocytosis. It could save hundreds of lives each year.”
PK-guided prophylaxis reduces dosing, still prevents bleeds
Personalized prophylaxis with a recombinant factor VIII product allowed for reduced dosing while still providing protection from bleeds in a small study of patients with severe hemophilia A.
Researchers tested pharmacokinetic (PK)-guided prophylaxis with simoctocog alfa in previously treated patients with severe hemophilia A.
The approach reduced the dose administered and increased the dosing interval compared to standard prophylaxis, without compromising protection from bleeds.
Researchers reported these results in the journal Haemophilia. The research was sponsored by Octapharma AG, the company marketing simoctocog alfa as Nuwiq.
Simoctocog alfa is a fourth-generation recombinant human factor VIII product produced in a human cell line.
In this phase 3b study, researchers tested PK-guided prophylaxis with simoctocog alfa in 66 previously treated adults with severe hemophilia A.
The patients’ mean age at baseline was 33.6. Most (62%) had received on-demand treatment in the 6 months prior to starting the study. Prophylaxis was largely irregular in the 38% of patients who received prophylaxis.
At baseline, the mean annualized bleeding rates (ABRs) were 38.9 in the entire cohort, 45.6 in the on-demand cohort, and 27.8 in the prophylaxis cohort.
Study design
The study had 3 phases. The first was the 72-hour PK phase. Patients received a single dose of simoctocog alfa (60 ± 5 IU kg–1) and had blood samples taken at various time points. This helped the researchers determine the patients’ personalized prophylaxis regimen.
The second phase of the study was the standard prophylaxis phase, which lasted 1 to 3 months. In this phase, patients received simoctocog alfa at a dose of 30 to 40 IU kg–1 every other day or 3 times a week until they began personalized prophylaxis.
The third phase was the 6-month personalized prophylaxis phase. A patient’s personalized prophylaxis regimen was based on individual PK modeling for each patient according to whether their PK profile most closely fit a 1- or 2-compartment model. In cases of uncertainty, the researchers used a non-compartment model.
A 2-compartment PK model was used for 36 patients, a 1-compartment model was used for 23 patients, and a non-compartment model was used for 7 patients.
Dosing
In total, the patients received 6612 infusions of simoctocog alfa.
During standard prophylaxis, the mean treatment duration was 2.7 months. Patients received a mean of 34.0 infusions and a total dose of 1157.6 IU kg–1.
During the personalized prophylaxis phase, the mean treatment duration was 6.2 months. Patients received a mean of 58.8 infusions and a total dose of 2574.4 IU kg–1.
The median dosing interval was 3.5 days during personalized prophylaxis, 3.5 days in the 1- and 2-compartment model groups, and 2.3 days in the non-compartment group. Fifty-seven percent of patients had twice-weekly dosing or less.
In the standard prophylaxis period, patients were dosed every other day or 3 times a week.
The median weekly dose was 100.0 IU kg–1 during standard prophylaxis and 95.0 IU kg–1 during personalized prophylaxis. It was 97.5 IU kg–1 during months 1 to 4 of personalized prophylaxis and 92.8 IU kg–1 during months 5 and 6 of personalized prophylaxis.
Bleeding and safety
Similar percentages of patients were bleed-free during the roughly 3-month standard prophylaxis period and the roughly 6-month personalized prophylaxis period—76.9% and 73.8%, respectively.
However, the mean ABR was 3.16 for the standard prophylaxis period and 1.45 for the personalized prophylaxis period. The median ABR was 0 for both periods.
In all, there were 95 breakthrough bleeds—46 during standard prophylaxis and 49 during personalized prophylaxis—in 23 of the patients. All of these bleeds were treated with at least 1 dose of simoctocog alfa.
There were no serious adverse events associated with treatment, and none of the patients developed factor VIII inhibitors.
One patient experienced malaise and dizziness after a single infusion during the standard prophylaxis period. These events were considered treatment-related, but both events resolved.
Personalized prophylaxis with a recombinant factor VIII product allowed for reduced dosing while still providing protection from bleeds in a small study of patients with severe hemophilia A.
Researchers tested pharmacokinetic (PK)-guided prophylaxis with simoctocog alfa in previously treated patients with severe hemophilia A.
The approach reduced the dose administered and increased the dosing interval compared to standard prophylaxis, without compromising protection from bleeds.
Researchers reported these results in the journal Haemophilia. The research was sponsored by Octapharma AG, the company marketing simoctocog alfa as Nuwiq.
Simoctocog alfa is a fourth-generation recombinant human factor VIII product produced in a human cell line.
In this phase 3b study, researchers tested PK-guided prophylaxis with simoctocog alfa in 66 previously treated adults with severe hemophilia A.
The patients’ mean age at baseline was 33.6. Most (62%) had received on-demand treatment in the 6 months prior to starting the study. Prophylaxis was largely irregular in the 38% of patients who received prophylaxis.
At baseline, the mean annualized bleeding rates (ABRs) were 38.9 in the entire cohort, 45.6 in the on-demand cohort, and 27.8 in the prophylaxis cohort.
Study design
The study had 3 phases. The first was the 72-hour PK phase. Patients received a single dose of simoctocog alfa (60 ± 5 IU kg–1) and had blood samples taken at various time points. This helped the researchers determine the patients’ personalized prophylaxis regimen.
The second phase of the study was the standard prophylaxis phase, which lasted 1 to 3 months. In this phase, patients received simoctocog alfa at a dose of 30 to 40 IU kg–1 every other day or 3 times a week until they began personalized prophylaxis.
The third phase was the 6-month personalized prophylaxis phase. A patient’s personalized prophylaxis regimen was based on individual PK modeling for each patient according to whether their PK profile most closely fit a 1- or 2-compartment model. In cases of uncertainty, the researchers used a non-compartment model.
A 2-compartment PK model was used for 36 patients, a 1-compartment model was used for 23 patients, and a non-compartment model was used for 7 patients.
Dosing
In total, the patients received 6612 infusions of simoctocog alfa.
During standard prophylaxis, the mean treatment duration was 2.7 months. Patients received a mean of 34.0 infusions and a total dose of 1157.6 IU kg–1.
During the personalized prophylaxis phase, the mean treatment duration was 6.2 months. Patients received a mean of 58.8 infusions and a total dose of 2574.4 IU kg–1.
The median dosing interval was 3.5 days during personalized prophylaxis, 3.5 days in the 1- and 2-compartment model groups, and 2.3 days in the non-compartment group. Fifty-seven percent of patients had twice-weekly dosing or less.
In the standard prophylaxis period, patients were dosed every other day or 3 times a week.
The median weekly dose was 100.0 IU kg–1 during standard prophylaxis and 95.0 IU kg–1 during personalized prophylaxis. It was 97.5 IU kg–1 during months 1 to 4 of personalized prophylaxis and 92.8 IU kg–1 during months 5 and 6 of personalized prophylaxis.
Bleeding and safety
Similar percentages of patients were bleed-free during the roughly 3-month standard prophylaxis period and the roughly 6-month personalized prophylaxis period—76.9% and 73.8%, respectively.
However, the mean ABR was 3.16 for the standard prophylaxis period and 1.45 for the personalized prophylaxis period. The median ABR was 0 for both periods.
In all, there were 95 breakthrough bleeds—46 during standard prophylaxis and 49 during personalized prophylaxis—in 23 of the patients. All of these bleeds were treated with at least 1 dose of simoctocog alfa.
There were no serious adverse events associated with treatment, and none of the patients developed factor VIII inhibitors.
One patient experienced malaise and dizziness after a single infusion during the standard prophylaxis period. These events were considered treatment-related, but both events resolved.
Personalized prophylaxis with a recombinant factor VIII product allowed for reduced dosing while still providing protection from bleeds in a small study of patients with severe hemophilia A.
Researchers tested pharmacokinetic (PK)-guided prophylaxis with simoctocog alfa in previously treated patients with severe hemophilia A.
The approach reduced the dose administered and increased the dosing interval compared to standard prophylaxis, without compromising protection from bleeds.
Researchers reported these results in the journal Haemophilia. The research was sponsored by Octapharma AG, the company marketing simoctocog alfa as Nuwiq.
Simoctocog alfa is a fourth-generation recombinant human factor VIII product produced in a human cell line.
In this phase 3b study, researchers tested PK-guided prophylaxis with simoctocog alfa in 66 previously treated adults with severe hemophilia A.
The patients’ mean age at baseline was 33.6. Most (62%) had received on-demand treatment in the 6 months prior to starting the study. Prophylaxis was largely irregular in the 38% of patients who received prophylaxis.
At baseline, the mean annualized bleeding rates (ABRs) were 38.9 in the entire cohort, 45.6 in the on-demand cohort, and 27.8 in the prophylaxis cohort.
Study design
The study had 3 phases. The first was the 72-hour PK phase. Patients received a single dose of simoctocog alfa (60 ± 5 IU kg–1) and had blood samples taken at various time points. This helped the researchers determine the patients’ personalized prophylaxis regimen.
The second phase of the study was the standard prophylaxis phase, which lasted 1 to 3 months. In this phase, patients received simoctocog alfa at a dose of 30 to 40 IU kg–1 every other day or 3 times a week until they began personalized prophylaxis.
The third phase was the 6-month personalized prophylaxis phase. A patient’s personalized prophylaxis regimen was based on individual PK modeling for each patient according to whether their PK profile most closely fit a 1- or 2-compartment model. In cases of uncertainty, the researchers used a non-compartment model.
A 2-compartment PK model was used for 36 patients, a 1-compartment model was used for 23 patients, and a non-compartment model was used for 7 patients.
Dosing
In total, the patients received 6612 infusions of simoctocog alfa.
During standard prophylaxis, the mean treatment duration was 2.7 months. Patients received a mean of 34.0 infusions and a total dose of 1157.6 IU kg–1.
During the personalized prophylaxis phase, the mean treatment duration was 6.2 months. Patients received a mean of 58.8 infusions and a total dose of 2574.4 IU kg–1.
The median dosing interval was 3.5 days during personalized prophylaxis, 3.5 days in the 1- and 2-compartment model groups, and 2.3 days in the non-compartment group. Fifty-seven percent of patients had twice-weekly dosing or less.
In the standard prophylaxis period, patients were dosed every other day or 3 times a week.
The median weekly dose was 100.0 IU kg–1 during standard prophylaxis and 95.0 IU kg–1 during personalized prophylaxis. It was 97.5 IU kg–1 during months 1 to 4 of personalized prophylaxis and 92.8 IU kg–1 during months 5 and 6 of personalized prophylaxis.
Bleeding and safety
Similar percentages of patients were bleed-free during the roughly 3-month standard prophylaxis period and the roughly 6-month personalized prophylaxis period—76.9% and 73.8%, respectively.
However, the mean ABR was 3.16 for the standard prophylaxis period and 1.45 for the personalized prophylaxis period. The median ABR was 0 for both periods.
In all, there were 95 breakthrough bleeds—46 during standard prophylaxis and 49 during personalized prophylaxis—in 23 of the patients. All of these bleeds were treated with at least 1 dose of simoctocog alfa.
There were no serious adverse events associated with treatment, and none of the patients developed factor VIII inhibitors.
One patient experienced malaise and dizziness after a single infusion during the standard prophylaxis period. These events were considered treatment-related, but both events resolved.
FDA approves new formulation of deferasirox
The US Food and Drug Administration (FDA) has approved a new formulation of deferasirox known as Jadenu Sprinkle granules.
The granules are approved for use in the same population as Jadenu film-coated tablets.
Both formulations of Jadenu have accelerated approval from the FDA for the treatment of chronic iron overload due to blood transfusions in patients age 2 and older.
The formulations also have accelerated FDA approval for the treatment of chronic iron overload in patients age 10 and older with non-transfusion-dependent-thalassemia and a liver iron concentration of at least 5 mg Fe per gram of dry weight and a serum ferritin greater than 300 mcg/L.
Continued FDA approval for Jadenu in these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Jadenu Sprinkle granules are intended for patients who have difficulty swallowing whole tablets. The granules can be sprinkled over soft foods (eg, yogurt or applesauce) prior to consumption.
Like Jadenu film-coated tablets, Jadenu Sprinkle granules are available in 3 strengths—90 mg, 180 mg, and 360 mg.
Both formulations of Jadenu are products of Novartis. For more details on Jadenu, see the prescribing information.
The US Food and Drug Administration (FDA) has approved a new formulation of deferasirox known as Jadenu Sprinkle granules.
The granules are approved for use in the same population as Jadenu film-coated tablets.
Both formulations of Jadenu have accelerated approval from the FDA for the treatment of chronic iron overload due to blood transfusions in patients age 2 and older.
The formulations also have accelerated FDA approval for the treatment of chronic iron overload in patients age 10 and older with non-transfusion-dependent-thalassemia and a liver iron concentration of at least 5 mg Fe per gram of dry weight and a serum ferritin greater than 300 mcg/L.
Continued FDA approval for Jadenu in these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Jadenu Sprinkle granules are intended for patients who have difficulty swallowing whole tablets. The granules can be sprinkled over soft foods (eg, yogurt or applesauce) prior to consumption.
Like Jadenu film-coated tablets, Jadenu Sprinkle granules are available in 3 strengths—90 mg, 180 mg, and 360 mg.
Both formulations of Jadenu are products of Novartis. For more details on Jadenu, see the prescribing information.
The US Food and Drug Administration (FDA) has approved a new formulation of deferasirox known as Jadenu Sprinkle granules.
The granules are approved for use in the same population as Jadenu film-coated tablets.
Both formulations of Jadenu have accelerated approval from the FDA for the treatment of chronic iron overload due to blood transfusions in patients age 2 and older.
The formulations also have accelerated FDA approval for the treatment of chronic iron overload in patients age 10 and older with non-transfusion-dependent-thalassemia and a liver iron concentration of at least 5 mg Fe per gram of dry weight and a serum ferritin greater than 300 mcg/L.
Continued FDA approval for Jadenu in these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Jadenu Sprinkle granules are intended for patients who have difficulty swallowing whole tablets. The granules can be sprinkled over soft foods (eg, yogurt or applesauce) prior to consumption.
Like Jadenu film-coated tablets, Jadenu Sprinkle granules are available in 3 strengths—90 mg, 180 mg, and 360 mg.
Both formulations of Jadenu are products of Novartis. For more details on Jadenu, see the prescribing information.
Why fewer blood cancer patients receive hospice care
New research provides an explanation for the fact that US patients with hematologic malignancies are less likely to enroll in hospice care than patients with solid tumor malignancies.
Results of a national survey suggest that concerns about the adequacy of hospice may prevent hematologic oncologists from referring their patients.
Researchers say this finding, published in Cancer, points to potential means of improving end-of-life care for patients with hematologic malignancies.
Oreofe Odejide, MD, of the Dana-Farber Cancer Institute in Boston, Massachusetts, and her colleagues carried out this study.
The team conducted a survey of a national sample of hematologic oncologists listed in the publicly available clinical directory of the American Society of Hematology.
More than 57% of physicians who were contacted provided responses, for a total of 349 respondents.
The survey included questions about views regarding the helpfulness and adequacy of home hospice services for patients with hematologic malignancies, as well as factors that would impact oncologists’ likelihood of referring patients to hospice.
More than 68% of hematologic oncologists strongly agreed that hospice care is “helpful” for patients with hematologic malignancies.
However, 46% of the oncologists felt that home hospice is “inadequate” for the needs of patients with hematologic malignancies, when compared to inpatient hospice.
Still, most of the respondents who believed home hospice is inadequate said they would be more likely to refer patients if platelet and red blood cell transfusions were readily available.
“Our findings are important as they shed light on factors that are potential barriers to hospice referrals,” Dr Odejide said. “These findings can be employed to develop targeted interventions to address hospice underuse for patients with blood cancers.”
New research provides an explanation for the fact that US patients with hematologic malignancies are less likely to enroll in hospice care than patients with solid tumor malignancies.
Results of a national survey suggest that concerns about the adequacy of hospice may prevent hematologic oncologists from referring their patients.
Researchers say this finding, published in Cancer, points to potential means of improving end-of-life care for patients with hematologic malignancies.
Oreofe Odejide, MD, of the Dana-Farber Cancer Institute in Boston, Massachusetts, and her colleagues carried out this study.
The team conducted a survey of a national sample of hematologic oncologists listed in the publicly available clinical directory of the American Society of Hematology.
More than 57% of physicians who were contacted provided responses, for a total of 349 respondents.
The survey included questions about views regarding the helpfulness and adequacy of home hospice services for patients with hematologic malignancies, as well as factors that would impact oncologists’ likelihood of referring patients to hospice.
More than 68% of hematologic oncologists strongly agreed that hospice care is “helpful” for patients with hematologic malignancies.
However, 46% of the oncologists felt that home hospice is “inadequate” for the needs of patients with hematologic malignancies, when compared to inpatient hospice.
Still, most of the respondents who believed home hospice is inadequate said they would be more likely to refer patients if platelet and red blood cell transfusions were readily available.
“Our findings are important as they shed light on factors that are potential barriers to hospice referrals,” Dr Odejide said. “These findings can be employed to develop targeted interventions to address hospice underuse for patients with blood cancers.”
New research provides an explanation for the fact that US patients with hematologic malignancies are less likely to enroll in hospice care than patients with solid tumor malignancies.
Results of a national survey suggest that concerns about the adequacy of hospice may prevent hematologic oncologists from referring their patients.
Researchers say this finding, published in Cancer, points to potential means of improving end-of-life care for patients with hematologic malignancies.
Oreofe Odejide, MD, of the Dana-Farber Cancer Institute in Boston, Massachusetts, and her colleagues carried out this study.
The team conducted a survey of a national sample of hematologic oncologists listed in the publicly available clinical directory of the American Society of Hematology.
More than 57% of physicians who were contacted provided responses, for a total of 349 respondents.
The survey included questions about views regarding the helpfulness and adequacy of home hospice services for patients with hematologic malignancies, as well as factors that would impact oncologists’ likelihood of referring patients to hospice.
More than 68% of hematologic oncologists strongly agreed that hospice care is “helpful” for patients with hematologic malignancies.
However, 46% of the oncologists felt that home hospice is “inadequate” for the needs of patients with hematologic malignancies, when compared to inpatient hospice.
Still, most of the respondents who believed home hospice is inadequate said they would be more likely to refer patients if platelet and red blood cell transfusions were readily available.
“Our findings are important as they shed light on factors that are potential barriers to hospice referrals,” Dr Odejide said. “These findings can be employed to develop targeted interventions to address hospice underuse for patients with blood cancers.”
Global study reveals healthcare inequity, preventable deaths
A global study has revealed inequity of access to and quality of healthcare among and within countries and suggests people are dying from causes with well-known treatments.
“What we have found about healthcare access and quality is disturbing,” said Christopher Murray, MD, DPhil, of the University of Washington in Seattle.
“Having a strong economy does not guarantee good healthcare. Having great medical technology doesn’t either. We know this because people are not getting the care that should be expected for diseases with established treatments.”
Dr Murray and his colleagues reported these findings in The Lancet.
For this study, the researchers assessed access to and quality of healthcare services in 195 countries from 1990 to 2015.
The group used the Healthcare Access and Quality Index, a summary measure based on 32 causes* that, in the presence of high-quality healthcare, should not result in death. Leukemia and Hodgkin lymphoma are among these causes.
Countries were assigned scores for each of the causes, based on estimates from the annual Global Burden of Diseases, Injuries, and Risk Factors study (GBD), a systematic, scientific effort to quantify the magnitude of health loss from all major diseases, injuries, and risk factors by age, sex, and population.
In addition, data were extracted from the most recent GBD update and evaluated using a Socio-demographic Index based on rates of education, fertility, and income.
Results
The 195 countries were assigned scores on a scale of 1 to 100 for healthcare access and quality. They received scores for the 32 causes as well as overall scores.
In 2015, the top-ranked nation was Andorra, with an overall score of 95. Its lowest treatment score was 70, for Hodgkin lymphoma.
The lowest-ranked nation was Central African Republic, with a score of 29. Its highest treatment score was 65, for diphtheria.
Nations in much of sub-Saharan Africa, as well as in south Asia and several countries in Latin America and the Caribbean, also had low rankings.
However, many countries in these regions, including China (score: 74) and Ethiopia (score: 44), have seen sizeable gains since 1990.
‘Developed’ nations falling short
The US had an overall score of 81 (in 2015), tied with Estonia and Montenegro. As with many other nations, the US scored 100 in treating common vaccine-preventable diseases, such as diphtheria, tetanus, and measles.
However, the US had 9 treatment categories in which it scored in the 60s: lower respiratory infections (60), neonatal disorders (69), non-melanoma skin cancer (68), Hodgkin lymphoma (67), ischemic heart disease (62), hypertensive heart disease (64), diabetes (67), chronic kidney disease (62), and the adverse effects of medical treatment itself (68).
“America’s ranking is an embarrassment, especially considering the US spends more than $9000 per person on healthcare annually, more than any other country,” Dr Murray said.
“Anyone with a stake in the current healthcare debate, including elected officials at the federal, state, and local levels, should take a look at where the US is falling short.”
Other nations with strong economies and advanced medical technology are falling short in some areas as well.
For example, Norway and Australia each scored 90 overall, among the highest in the world. However, Norway scored 65 in its treatment for testicular cancer, and Australia scored 52 for treating non-melanoma skin cancer.
“In the majority of cases, both of these cancers can be treated effectively,” Dr Murray said. “Shouldn’t it cause serious concern that people are dying of these causes in countries that have the resources to address them?”
*The 32 causes are:
- Adverse effects of medical treatment
- Appendicitis
- Breast cancer
- Cerebrovascular disease (stroke)
- Cervical cancer
- Chronic kidney disease
- Chronic respiratory diseases
- Colon and rectum cancer
- Congenital anomalies
- Diabetes mellitus
- Diarrhea-related diseases
- Diphtheria
- Epilepsy
- Gallbladder and biliary diseases
- Hodgkin lymphoma
- Hypertensive heart disease
- Inguinal, femoral, and abdominal hernia
- Ischemic heart disease
- Leukemia
- Lower respiratory infections
- Maternal disorders
- Measles
- Neonatal disorders
- Non-melanoma skin cancer
- Peptic ulcer disease
- Rheumatic heart disease
- Testicular cancer
- Tetanus
- Tuberculosis
- Upper respiratory infections
- Uterine cancer
- Whooping cough.
A global study has revealed inequity of access to and quality of healthcare among and within countries and suggests people are dying from causes with well-known treatments.
“What we have found about healthcare access and quality is disturbing,” said Christopher Murray, MD, DPhil, of the University of Washington in Seattle.
“Having a strong economy does not guarantee good healthcare. Having great medical technology doesn’t either. We know this because people are not getting the care that should be expected for diseases with established treatments.”
Dr Murray and his colleagues reported these findings in The Lancet.
For this study, the researchers assessed access to and quality of healthcare services in 195 countries from 1990 to 2015.
The group used the Healthcare Access and Quality Index, a summary measure based on 32 causes* that, in the presence of high-quality healthcare, should not result in death. Leukemia and Hodgkin lymphoma are among these causes.
Countries were assigned scores for each of the causes, based on estimates from the annual Global Burden of Diseases, Injuries, and Risk Factors study (GBD), a systematic, scientific effort to quantify the magnitude of health loss from all major diseases, injuries, and risk factors by age, sex, and population.
In addition, data were extracted from the most recent GBD update and evaluated using a Socio-demographic Index based on rates of education, fertility, and income.
Results
The 195 countries were assigned scores on a scale of 1 to 100 for healthcare access and quality. They received scores for the 32 causes as well as overall scores.
In 2015, the top-ranked nation was Andorra, with an overall score of 95. Its lowest treatment score was 70, for Hodgkin lymphoma.
The lowest-ranked nation was Central African Republic, with a score of 29. Its highest treatment score was 65, for diphtheria.
Nations in much of sub-Saharan Africa, as well as in south Asia and several countries in Latin America and the Caribbean, also had low rankings.
However, many countries in these regions, including China (score: 74) and Ethiopia (score: 44), have seen sizeable gains since 1990.
‘Developed’ nations falling short
The US had an overall score of 81 (in 2015), tied with Estonia and Montenegro. As with many other nations, the US scored 100 in treating common vaccine-preventable diseases, such as diphtheria, tetanus, and measles.
However, the US had 9 treatment categories in which it scored in the 60s: lower respiratory infections (60), neonatal disorders (69), non-melanoma skin cancer (68), Hodgkin lymphoma (67), ischemic heart disease (62), hypertensive heart disease (64), diabetes (67), chronic kidney disease (62), and the adverse effects of medical treatment itself (68).
“America’s ranking is an embarrassment, especially considering the US spends more than $9000 per person on healthcare annually, more than any other country,” Dr Murray said.
“Anyone with a stake in the current healthcare debate, including elected officials at the federal, state, and local levels, should take a look at where the US is falling short.”
Other nations with strong economies and advanced medical technology are falling short in some areas as well.
For example, Norway and Australia each scored 90 overall, among the highest in the world. However, Norway scored 65 in its treatment for testicular cancer, and Australia scored 52 for treating non-melanoma skin cancer.
“In the majority of cases, both of these cancers can be treated effectively,” Dr Murray said. “Shouldn’t it cause serious concern that people are dying of these causes in countries that have the resources to address them?”
*The 32 causes are:
- Adverse effects of medical treatment
- Appendicitis
- Breast cancer
- Cerebrovascular disease (stroke)
- Cervical cancer
- Chronic kidney disease
- Chronic respiratory diseases
- Colon and rectum cancer
- Congenital anomalies
- Diabetes mellitus
- Diarrhea-related diseases
- Diphtheria
- Epilepsy
- Gallbladder and biliary diseases
- Hodgkin lymphoma
- Hypertensive heart disease
- Inguinal, femoral, and abdominal hernia
- Ischemic heart disease
- Leukemia
- Lower respiratory infections
- Maternal disorders
- Measles
- Neonatal disorders
- Non-melanoma skin cancer
- Peptic ulcer disease
- Rheumatic heart disease
- Testicular cancer
- Tetanus
- Tuberculosis
- Upper respiratory infections
- Uterine cancer
- Whooping cough.
A global study has revealed inequity of access to and quality of healthcare among and within countries and suggests people are dying from causes with well-known treatments.
“What we have found about healthcare access and quality is disturbing,” said Christopher Murray, MD, DPhil, of the University of Washington in Seattle.
“Having a strong economy does not guarantee good healthcare. Having great medical technology doesn’t either. We know this because people are not getting the care that should be expected for diseases with established treatments.”
Dr Murray and his colleagues reported these findings in The Lancet.
For this study, the researchers assessed access to and quality of healthcare services in 195 countries from 1990 to 2015.
The group used the Healthcare Access and Quality Index, a summary measure based on 32 causes* that, in the presence of high-quality healthcare, should not result in death. Leukemia and Hodgkin lymphoma are among these causes.
Countries were assigned scores for each of the causes, based on estimates from the annual Global Burden of Diseases, Injuries, and Risk Factors study (GBD), a systematic, scientific effort to quantify the magnitude of health loss from all major diseases, injuries, and risk factors by age, sex, and population.
In addition, data were extracted from the most recent GBD update and evaluated using a Socio-demographic Index based on rates of education, fertility, and income.
Results
The 195 countries were assigned scores on a scale of 1 to 100 for healthcare access and quality. They received scores for the 32 causes as well as overall scores.
In 2015, the top-ranked nation was Andorra, with an overall score of 95. Its lowest treatment score was 70, for Hodgkin lymphoma.
The lowest-ranked nation was Central African Republic, with a score of 29. Its highest treatment score was 65, for diphtheria.
Nations in much of sub-Saharan Africa, as well as in south Asia and several countries in Latin America and the Caribbean, also had low rankings.
However, many countries in these regions, including China (score: 74) and Ethiopia (score: 44), have seen sizeable gains since 1990.
‘Developed’ nations falling short
The US had an overall score of 81 (in 2015), tied with Estonia and Montenegro. As with many other nations, the US scored 100 in treating common vaccine-preventable diseases, such as diphtheria, tetanus, and measles.
However, the US had 9 treatment categories in which it scored in the 60s: lower respiratory infections (60), neonatal disorders (69), non-melanoma skin cancer (68), Hodgkin lymphoma (67), ischemic heart disease (62), hypertensive heart disease (64), diabetes (67), chronic kidney disease (62), and the adverse effects of medical treatment itself (68).
“America’s ranking is an embarrassment, especially considering the US spends more than $9000 per person on healthcare annually, more than any other country,” Dr Murray said.
“Anyone with a stake in the current healthcare debate, including elected officials at the federal, state, and local levels, should take a look at where the US is falling short.”
Other nations with strong economies and advanced medical technology are falling short in some areas as well.
For example, Norway and Australia each scored 90 overall, among the highest in the world. However, Norway scored 65 in its treatment for testicular cancer, and Australia scored 52 for treating non-melanoma skin cancer.
“In the majority of cases, both of these cancers can be treated effectively,” Dr Murray said. “Shouldn’t it cause serious concern that people are dying of these causes in countries that have the resources to address them?”
*The 32 causes are:
- Adverse effects of medical treatment
- Appendicitis
- Breast cancer
- Cerebrovascular disease (stroke)
- Cervical cancer
- Chronic kidney disease
- Chronic respiratory diseases
- Colon and rectum cancer
- Congenital anomalies
- Diabetes mellitus
- Diarrhea-related diseases
- Diphtheria
- Epilepsy
- Gallbladder and biliary diseases
- Hodgkin lymphoma
- Hypertensive heart disease
- Inguinal, femoral, and abdominal hernia
- Ischemic heart disease
- Leukemia
- Lower respiratory infections
- Maternal disorders
- Measles
- Neonatal disorders
- Non-melanoma skin cancer
- Peptic ulcer disease
- Rheumatic heart disease
- Testicular cancer
- Tetanus
- Tuberculosis
- Upper respiratory infections
- Uterine cancer
- Whooping cough.
FDA supports continued, cautious use of GBCAs
The US Food and Drug Administration (FDA) said it has not found any evidence of adverse health effects from gadolinium retention in the brain following the use of gadolinium-based contrast agents (GBCAs) for magnetic resonance imaging (MRI).
The agency noted that all GBCAs may be associated with some gadolinium retention in the brain and other body tissues.
However, an FDA review showed no evidence that gadolinium retention in the brain is harmful.
Therefore, the FDA said it will not restrict GBCA use, although the agency will continue to assess the safety of GBCAs and plans to have a public meeting on the issue in the future.
The manufacturer of OptiMARK (gadoversetamide), a linear GBCA, updated its label with information about gadolinium retention. The FDA said it is reviewing the labels of other GBCAs to determine if changes are needed.
Recommendations
The FDA said its recommendations regarding GBCAs have not changed.
The agency advises healthcare professionals to limit GBCA use to circumstances in which the contrast agent can provide necessary information. Professionals should also consider the necessity of repetitive MRIs with GBCAs.
Patients, parents, and caregivers with any questions or concerns about GBCAs should discuss the agents with their healthcare professionals.
The FDA is also urging patients and healthcare professionals to report side effects involving GBCAs to the agency’s MedWatch program.
About the FDA review
For its review, the FDA looked at scientific publications and adverse event reports submitted to agency.
These data showed that gadolinium is retained in organs and suggested that linear GBCAs cause retention of more gadolinium in the brain than macrocyclic GBCAs. However, the data did not show adverse health effects related to this brain retention.
The only known adverse health effect related to gadolinium retention is nephrogenic systemic fibrosis (NSF), a disease characterized by thickening of the skin, which can involve the joints and limit motion.
NSF is known to occur in patients with pre-existing kidney failure. However, recent publications have shown reactions involving thickening and hardening of the skin and other tissues in patients with normal kidney function who received GBCAs and did not have NSF. Some of these patients also had evidence of gadolinium retention.
The FDA said it is evaluating such reports to determine if these fibrotic reactions are an adverse health effect of retained gadolinium.
The agency is also continuing its assessment of GBCAs, investigating how gadolinium is retained in the body. And the FDA’s National Center for Toxicological Research is conducting a study on brain retention of GBCAs in rats.
PRAC review
A recent review by the European Medicines Agency’s Pharmacovigilance Risk Assessment Committee (PRAC) suggested there are no adverse health effects associated with gadolinium retention in the brain.
However, the PRAC recommended suspending the marketing authorization of certain linear GBCAs because they cause greater retention of gadolinium in the brain than macrocyclic GBCAs.
The PRAC’s recommendation is undergoing an appeal, which will be further reviewed by the PRAC and the Committee for Medicinal Products for Human Use.
The US Food and Drug Administration (FDA) said it has not found any evidence of adverse health effects from gadolinium retention in the brain following the use of gadolinium-based contrast agents (GBCAs) for magnetic resonance imaging (MRI).
The agency noted that all GBCAs may be associated with some gadolinium retention in the brain and other body tissues.
However, an FDA review showed no evidence that gadolinium retention in the brain is harmful.
Therefore, the FDA said it will not restrict GBCA use, although the agency will continue to assess the safety of GBCAs and plans to have a public meeting on the issue in the future.
The manufacturer of OptiMARK (gadoversetamide), a linear GBCA, updated its label with information about gadolinium retention. The FDA said it is reviewing the labels of other GBCAs to determine if changes are needed.
Recommendations
The FDA said its recommendations regarding GBCAs have not changed.
The agency advises healthcare professionals to limit GBCA use to circumstances in which the contrast agent can provide necessary information. Professionals should also consider the necessity of repetitive MRIs with GBCAs.
Patients, parents, and caregivers with any questions or concerns about GBCAs should discuss the agents with their healthcare professionals.
The FDA is also urging patients and healthcare professionals to report side effects involving GBCAs to the agency’s MedWatch program.
About the FDA review
For its review, the FDA looked at scientific publications and adverse event reports submitted to agency.
These data showed that gadolinium is retained in organs and suggested that linear GBCAs cause retention of more gadolinium in the brain than macrocyclic GBCAs. However, the data did not show adverse health effects related to this brain retention.
The only known adverse health effect related to gadolinium retention is nephrogenic systemic fibrosis (NSF), a disease characterized by thickening of the skin, which can involve the joints and limit motion.
NSF is known to occur in patients with pre-existing kidney failure. However, recent publications have shown reactions involving thickening and hardening of the skin and other tissues in patients with normal kidney function who received GBCAs and did not have NSF. Some of these patients also had evidence of gadolinium retention.
The FDA said it is evaluating such reports to determine if these fibrotic reactions are an adverse health effect of retained gadolinium.
The agency is also continuing its assessment of GBCAs, investigating how gadolinium is retained in the body. And the FDA’s National Center for Toxicological Research is conducting a study on brain retention of GBCAs in rats.
PRAC review
A recent review by the European Medicines Agency’s Pharmacovigilance Risk Assessment Committee (PRAC) suggested there are no adverse health effects associated with gadolinium retention in the brain.
However, the PRAC recommended suspending the marketing authorization of certain linear GBCAs because they cause greater retention of gadolinium in the brain than macrocyclic GBCAs.
The PRAC’s recommendation is undergoing an appeal, which will be further reviewed by the PRAC and the Committee for Medicinal Products for Human Use.
The US Food and Drug Administration (FDA) said it has not found any evidence of adverse health effects from gadolinium retention in the brain following the use of gadolinium-based contrast agents (GBCAs) for magnetic resonance imaging (MRI).
The agency noted that all GBCAs may be associated with some gadolinium retention in the brain and other body tissues.
However, an FDA review showed no evidence that gadolinium retention in the brain is harmful.
Therefore, the FDA said it will not restrict GBCA use, although the agency will continue to assess the safety of GBCAs and plans to have a public meeting on the issue in the future.
The manufacturer of OptiMARK (gadoversetamide), a linear GBCA, updated its label with information about gadolinium retention. The FDA said it is reviewing the labels of other GBCAs to determine if changes are needed.
Recommendations
The FDA said its recommendations regarding GBCAs have not changed.
The agency advises healthcare professionals to limit GBCA use to circumstances in which the contrast agent can provide necessary information. Professionals should also consider the necessity of repetitive MRIs with GBCAs.
Patients, parents, and caregivers with any questions or concerns about GBCAs should discuss the agents with their healthcare professionals.
The FDA is also urging patients and healthcare professionals to report side effects involving GBCAs to the agency’s MedWatch program.
About the FDA review
For its review, the FDA looked at scientific publications and adverse event reports submitted to agency.
These data showed that gadolinium is retained in organs and suggested that linear GBCAs cause retention of more gadolinium in the brain than macrocyclic GBCAs. However, the data did not show adverse health effects related to this brain retention.
The only known adverse health effect related to gadolinium retention is nephrogenic systemic fibrosis (NSF), a disease characterized by thickening of the skin, which can involve the joints and limit motion.
NSF is known to occur in patients with pre-existing kidney failure. However, recent publications have shown reactions involving thickening and hardening of the skin and other tissues in patients with normal kidney function who received GBCAs and did not have NSF. Some of these patients also had evidence of gadolinium retention.
The FDA said it is evaluating such reports to determine if these fibrotic reactions are an adverse health effect of retained gadolinium.
The agency is also continuing its assessment of GBCAs, investigating how gadolinium is retained in the body. And the FDA’s National Center for Toxicological Research is conducting a study on brain retention of GBCAs in rats.
PRAC review
A recent review by the European Medicines Agency’s Pharmacovigilance Risk Assessment Committee (PRAC) suggested there are no adverse health effects associated with gadolinium retention in the brain.
However, the PRAC recommended suspending the marketing authorization of certain linear GBCAs because they cause greater retention of gadolinium in the brain than macrocyclic GBCAs.
The PRAC’s recommendation is undergoing an appeal, which will be further reviewed by the PRAC and the Committee for Medicinal Products for Human Use.
Severe hospital-acquired anemia linked to readmission, death
Severe hospital-acquired anemia (HAA) may increase a person’s risk of hospital readmission and death, a new study suggests.
Researchers studied more than 11,000 patients admitted to 6 Texas hospitals and found that a third of the patients developed HAA.
The team also found that severe HAA was associated with a higher risk of death or readmission, even after the researchers adjusted for other factors.
They reported these findings in the Journal of Hospital Medicine.
“This study shines a spotlight on a very common but underappreciated risk of hospitalization, hospital-acquired anemia, which has traditionally been viewed as an incidental change in the red blood count of no significance,” said study author Ethan Halm, MD, of the University of Texas Southwestern Medical Center in Dallas.
“However, our results showed that hospital-acquired anemia was associated with worse clinical outcomes after leaving the hospital, so it needs to be taken more seriously.”
Dr Halm and his colleagues looked at consecutive medicine discharges between November 1, 2009, and October 30, 2010, from 6 hospitals in Texas (safety-net, teaching, and nonteaching).
Of the 11,309 patients studied, 33.1% developed HAA. Most (21.6%) had mild HAA, followed by moderate HAA (10.1%), and severe HAA (1.4%).
The study’s primary outcome was a composite of 30-day mortality and nonelective readmission. This occurred in 9.7% of patients without HAA and 16.4% of those with severe HAA.
The researchers found that severe HAA was independently associated with a 39% increase in the odds of meeting the primary outcome (readmission or 30-day mortality).
The team noted that 85% of patients with severe HAA underwent a major procedure, had a discharge diagnosis of hemorrhage, and/or a discharge diagnosis of hemorrhagic disorder.
The researchers identified 2 potentially modifiable predictors of moderate or severe HAA. These were length of hospital stay (adjusted odds ratio=1.26 per day) and undergoing a major procedure (adjusted odds ratio=5.09).
“Our findings suggest that reducing blood loss during major surgeries and reducing unnecessary testing during hospital stays may lower a patient’s risk of developing severe hospital-acquired anemia, and potentially improve their recovery,” said Anil N. Makam, MD, of the University of Texas Southwestern Medical Center.
In the future, the researchers hope to examine other patient-centered outcomes that may be related to HAA, such as fatigue, functional impairment, and the trajectory of post-hospital recovery.
Severe hospital-acquired anemia (HAA) may increase a person’s risk of hospital readmission and death, a new study suggests.
Researchers studied more than 11,000 patients admitted to 6 Texas hospitals and found that a third of the patients developed HAA.
The team also found that severe HAA was associated with a higher risk of death or readmission, even after the researchers adjusted for other factors.
They reported these findings in the Journal of Hospital Medicine.
“This study shines a spotlight on a very common but underappreciated risk of hospitalization, hospital-acquired anemia, which has traditionally been viewed as an incidental change in the red blood count of no significance,” said study author Ethan Halm, MD, of the University of Texas Southwestern Medical Center in Dallas.
“However, our results showed that hospital-acquired anemia was associated with worse clinical outcomes after leaving the hospital, so it needs to be taken more seriously.”
Dr Halm and his colleagues looked at consecutive medicine discharges between November 1, 2009, and October 30, 2010, from 6 hospitals in Texas (safety-net, teaching, and nonteaching).
Of the 11,309 patients studied, 33.1% developed HAA. Most (21.6%) had mild HAA, followed by moderate HAA (10.1%), and severe HAA (1.4%).
The study’s primary outcome was a composite of 30-day mortality and nonelective readmission. This occurred in 9.7% of patients without HAA and 16.4% of those with severe HAA.
The researchers found that severe HAA was independently associated with a 39% increase in the odds of meeting the primary outcome (readmission or 30-day mortality).
The team noted that 85% of patients with severe HAA underwent a major procedure, had a discharge diagnosis of hemorrhage, and/or a discharge diagnosis of hemorrhagic disorder.
The researchers identified 2 potentially modifiable predictors of moderate or severe HAA. These were length of hospital stay (adjusted odds ratio=1.26 per day) and undergoing a major procedure (adjusted odds ratio=5.09).
“Our findings suggest that reducing blood loss during major surgeries and reducing unnecessary testing during hospital stays may lower a patient’s risk of developing severe hospital-acquired anemia, and potentially improve their recovery,” said Anil N. Makam, MD, of the University of Texas Southwestern Medical Center.
In the future, the researchers hope to examine other patient-centered outcomes that may be related to HAA, such as fatigue, functional impairment, and the trajectory of post-hospital recovery.
Severe hospital-acquired anemia (HAA) may increase a person’s risk of hospital readmission and death, a new study suggests.
Researchers studied more than 11,000 patients admitted to 6 Texas hospitals and found that a third of the patients developed HAA.
The team also found that severe HAA was associated with a higher risk of death or readmission, even after the researchers adjusted for other factors.
They reported these findings in the Journal of Hospital Medicine.
“This study shines a spotlight on a very common but underappreciated risk of hospitalization, hospital-acquired anemia, which has traditionally been viewed as an incidental change in the red blood count of no significance,” said study author Ethan Halm, MD, of the University of Texas Southwestern Medical Center in Dallas.
“However, our results showed that hospital-acquired anemia was associated with worse clinical outcomes after leaving the hospital, so it needs to be taken more seriously.”
Dr Halm and his colleagues looked at consecutive medicine discharges between November 1, 2009, and October 30, 2010, from 6 hospitals in Texas (safety-net, teaching, and nonteaching).
Of the 11,309 patients studied, 33.1% developed HAA. Most (21.6%) had mild HAA, followed by moderate HAA (10.1%), and severe HAA (1.4%).
The study’s primary outcome was a composite of 30-day mortality and nonelective readmission. This occurred in 9.7% of patients without HAA and 16.4% of those with severe HAA.
The researchers found that severe HAA was independently associated with a 39% increase in the odds of meeting the primary outcome (readmission or 30-day mortality).
The team noted that 85% of patients with severe HAA underwent a major procedure, had a discharge diagnosis of hemorrhage, and/or a discharge diagnosis of hemorrhagic disorder.
The researchers identified 2 potentially modifiable predictors of moderate or severe HAA. These were length of hospital stay (adjusted odds ratio=1.26 per day) and undergoing a major procedure (adjusted odds ratio=5.09).
“Our findings suggest that reducing blood loss during major surgeries and reducing unnecessary testing during hospital stays may lower a patient’s risk of developing severe hospital-acquired anemia, and potentially improve their recovery,” said Anil N. Makam, MD, of the University of Texas Southwestern Medical Center.
In the future, the researchers hope to examine other patient-centered outcomes that may be related to HAA, such as fatigue, functional impairment, and the trajectory of post-hospital recovery.