PORTLAND, ORE. – A new device worn on one ear may help individuals with Parkinson’s disease to overcome speech deficits associated with the disease. When triggered by the wearer’s voice, the device, SpeechVive, provides a simulated room crowd noise or “babble” only when they are talking. Because it capitalizes on natural reflexes to this simulated background noise, improvement can occur independent of speech training.

Researchers led by Jessica Huber, PhD, a professor of speech, language, and hearing sciences at Purdue University in West Lafayette, Ind., investigated changes to speech after 3 months of daily use of the SpeechVive device by 16 individuals. The cohort had a mean age of 64.5 years (range: 56-78 years) and a mean disease duration of 8.1 years (range: 2-17 years). Six participants had previously had speech therapy (five with LSVT [Lee Silverman Voice Treatment] LOUD speech training exercises), and two had deep brain stimulation implants.

Available speech therapies

People with Parkinson’s disease (PD) often have reduced vocal loudness, increased speech rate, and slurred articulation. Available speech therapies have included adduction exercises, vocal function exercises, and LSVT LOUD speech training exercises. For some individuals with PD, these behavioral treatments may not carry over into daily life.

The SpeechVive consists of a piece placed into the ear canal and a piece that sits behind the ear, similar to a slightly larger version of a modern hearing aid. It is designed large enough that many patients, who have motor deficits, can don it themselves. Because it is on just one ear, users can hear other people talking and what is going on around them.

Dr. Huber noted several strengths of the device. First, it does not impose any cognitive load on the user because its effect is based on an automatic reflex. Second, little training is necessary. It is easy for the user to put on the device, and many of its beneficial effects occur as soon as it is activated. Third, compliance can be tracked using data stored by the device itself.

In a previous study, she saw that after using the device for 8 weeks, participants used more effective patterns with their respiratory and laryngeal mechanisms to produce louder speech. “It’s not like they’re working super hard after 8 weeks.” She thinks they are just not usually using their speech apparatus in the most efficient manner, and they become more efficient after using the device. They may also not sense how loud they are without it, so it helps patients “recalibrate” a sense of their own voices, something the LSVT LOUD training also does.

Jori Fleisher, MD, a movement disorders neurologist at the New York University Langone Medical Center, commented that the study shows an interesting approach to the common and difficult problem of hypophonia and dysphonia in people with Parkinson’s disease. “It can be very difficult to speak loud enough to be heard, and it often leads to people withdrawing socially and not speaking as much in conversation as they normally would,” she said. “LSVT LOUD works in practice when you’re actually doing the exercises, but often when people stop doing the exercises, their voice goes back toward their normal. So this is something that you can keep with you and sort of have constant prompting to overcome that tendency toward quieter voice. It has a lot of promise.”

She said she would like to see the data on compliance or adherence with using the device “because it seems like a great idea, but how much are people actually using it? ... Having more long-term follow-up to see whether this is something that people want to put into practice in their daily life would be very useful.” She also said she would like to see how long the carry-over effect lasts once people take off the device to be able to compare it with sessions of speech therapy or doing speech exercises.

SpeechVive is $2,495, which includes the device, a charger, and earpiece fittings. It is available only in the United States, but the company now has clearance to market it in Canada.

The research was funded by a grant from SpeechVive. Dr. Huber is the inventor of SpeechVive, has a patent on it, has a financial interest in the SpeechVive company, and sits on its board of directors. Dr. Fleisher reported having no financial conflicts.

PORTLAND, ORE. – A new device worn on one ear may help individuals with Parkinson’s disease to overcome speech deficits associated with the disease. When triggered by the wearer’s voice, the device, SpeechVive, provides a simulated room crowd noise or “babble” only when they are talking. Because it capitalizes on natural reflexes to this simulated background noise, improvement can occur independent of speech training.

Researchers led by Jessica Huber, PhD, a professor of speech, language, and hearing sciences at Purdue University in West Lafayette, Ind., investigated changes to speech after 3 months of daily use of the SpeechVive device by 16 individuals. The cohort had a mean age of 64.5 years (range: 56-78 years) and a mean disease duration of 8.1 years (range: 2-17 years). Six participants had previously had speech therapy (five with LSVT [Lee Silverman Voice Treatment] LOUD speech training exercises), and two had deep brain stimulation implants.

Available speech therapies

People with Parkinson’s disease (PD) often have reduced vocal loudness, increased speech rate, and slurred articulation. Available speech therapies have included adduction exercises, vocal function exercises, and LSVT LOUD speech training exercises. For some individuals with PD, these behavioral treatments may not carry over into daily life.

The SpeechVive consists of a piece placed into the ear canal and a piece that sits behind the ear, similar to a slightly larger version of a modern hearing aid. It is designed large enough that many patients, who have motor deficits, can don it themselves. Because it is on just one ear, users can hear other people talking and what is going on around them.

Dr. Huber noted several strengths of the device. First, it does not impose any cognitive load on the user because its effect is based on an automatic reflex. Second, little training is necessary. It is easy for the user to put on the device, and many of its beneficial effects occur as soon as it is activated. Third, compliance can be tracked using data stored by the device itself.

In a previous study, she saw that after using the device for 8 weeks, participants used more effective patterns with their respiratory and laryngeal mechanisms to produce louder speech. “It’s not like they’re working super hard after 8 weeks.” She thinks they are just not usually using their speech apparatus in the most efficient manner, and they become more efficient after using the device. They may also not sense how loud they are without it, so it helps patients “recalibrate” a sense of their own voices, something the LSVT LOUD training also does.

Jori Fleisher, MD, a movement disorders neurologist at the New York University Langone Medical Center, commented that the study shows an interesting approach to the common and difficult problem of hypophonia and dysphonia in people with Parkinson’s disease. “It can be very difficult to speak loud enough to be heard, and it often leads to people withdrawing socially and not speaking as much in conversation as they normally would,” she said. “LSVT LOUD works in practice when you’re actually doing the exercises, but often when people stop doing the exercises, their voice goes back toward their normal. So this is something that you can keep with you and sort of have constant prompting to overcome that tendency toward quieter voice. It has a lot of promise.”

She said she would like to see the data on compliance or adherence with using the device “because it seems like a great idea, but how much are people actually using it? ... Having more long-term follow-up to see whether this is something that people want to put into practice in their daily life would be very useful.” She also said she would like to see how long the carry-over effect lasts once people take off the device to be able to compare it with sessions of speech therapy or doing speech exercises.

SpeechVive is $2,495, which includes the device, a charger, and earpiece fittings. It is available only in the United States, but the company now has clearance to market it in Canada.

The research was funded by a grant from SpeechVive. Dr. Huber is the inventor of SpeechVive, has a patent on it, has a financial interest in the SpeechVive company, and sits on its board of directors. Dr. Fleisher reported having no financial conflicts.

PORTLAND, ORE. – A new device worn on one ear may help individuals with Parkinson’s disease to overcome speech deficits associated with the disease. When triggered by the wearer’s voice, the device, SpeechVive, provides a simulated room crowd noise or “babble” only when they are talking. Because it capitalizes on natural reflexes to this simulated background noise, improvement can occur independent of speech training.

Researchers led by Jessica Huber, PhD, a professor of speech, language, and hearing sciences at Purdue University in West Lafayette, Ind., investigated changes to speech after 3 months of daily use of the SpeechVive device by 16 individuals. The cohort had a mean age of 64.5 years (range: 56-78 years) and a mean disease duration of 8.1 years (range: 2-17 years). Six participants had previously had speech therapy (five with LSVT [Lee Silverman Voice Treatment] LOUD speech training exercises), and two had deep brain stimulation implants.

Available speech therapies

People with Parkinson’s disease (PD) often have reduced vocal loudness, increased speech rate, and slurred articulation. Available speech therapies have included adduction exercises, vocal function exercises, and LSVT LOUD speech training exercises. For some individuals with PD, these behavioral treatments may not carry over into daily life.

The SpeechVive consists of a piece placed into the ear canal and a piece that sits behind the ear, similar to a slightly larger version of a modern hearing aid. It is designed large enough that many patients, who have motor deficits, can don it themselves. Because it is on just one ear, users can hear other people talking and what is going on around them.

Dr. Huber noted several strengths of the device. First, it does not impose any cognitive load on the user because its effect is based on an automatic reflex. Second, little training is necessary. It is easy for the user to put on the device, and many of its beneficial effects occur as soon as it is activated. Third, compliance can be tracked using data stored by the device itself.

In a previous study, she saw that after using the device for 8 weeks, participants used more effective patterns with their respiratory and laryngeal mechanisms to produce louder speech. “It’s not like they’re working super hard after 8 weeks.” She thinks they are just not usually using their speech apparatus in the most efficient manner, and they become more efficient after using the device. They may also not sense how loud they are without it, so it helps patients “recalibrate” a sense of their own voices, something the LSVT LOUD training also does.

Jori Fleisher, MD, a movement disorders neurologist at the New York University Langone Medical Center, commented that the study shows an interesting approach to the common and difficult problem of hypophonia and dysphonia in people with Parkinson’s disease. “It can be very difficult to speak loud enough to be heard, and it often leads to people withdrawing socially and not speaking as much in conversation as they normally would,” she said. “LSVT LOUD works in practice when you’re actually doing the exercises, but often when people stop doing the exercises, their voice goes back toward their normal. So this is something that you can keep with you and sort of have constant prompting to overcome that tendency toward quieter voice. It has a lot of promise.”

She said she would like to see the data on compliance or adherence with using the device “because it seems like a great idea, but how much are people actually using it? ... Having more long-term follow-up to see whether this is something that people want to put into practice in their daily life would be very useful.” She also said she would like to see how long the carry-over effect lasts once people take off the device to be able to compare it with sessions of speech therapy or doing speech exercises.

SpeechVive is $2,495, which includes the device, a charger, and earpiece fittings. It is available only in the United States, but the company now has clearance to market it in Canada.

The research was funded by a grant from SpeechVive. Dr. Huber is the inventor of SpeechVive, has a patent on it, has a financial interest in the SpeechVive company, and sits on its board of directors. Dr. Fleisher reported having no financial conflicts.

The calcium channel blocker amlodipine, added to iron chelation therapy, significantly reduced excess myocardial iron concentration in patients with thalassemia major, compared with chelation alone, according to results from a randomized trial.

The findings (Blood. 2016;128[12]:1555-61) suggest that amlodipine, a cheap, widely available drug with a well-established safety profile, may serve as an adjunct to standard treatment for people with thalassemia major and cardiac siderosis. Cardiovascular disease caused by excess myocardial iron remains a major cause of morbidity and mortality in thalassemia major.

Juliano L. Fernandes, MD, PhD, of the Jose Michel Kalaf Research Institute in Campinas, Brazil, led the study, which randomized 62 patients already receiving chelation treatment for thalassemia major to 1 year of chelation plus placebo (n = 31) or chelation plus 5 mg daily amlodipine (n = 31).

Patients in each arm were subdivided into two subgroups: those whose baseline myocardial iron concentration was within normal thresholds, and those with excess myocardial iron concentration as measured by magnetic resonance imaging (above 0.59 mg/g dry weight or with a cardiac T2* below 35 milliseconds).

In the amlodipine arm, patients with excess cardiac iron at baseline (n = 15) saw significant reductions in myocardial iron concentrations at 1 year, compared with those randomized to placebo (n = 15). The former had a median reduction of –0.26 mg/g (95% confidence interval, –1.02 to –0.01) while the placebo group saw an increase of 0.01 mg/g (95% CI, 20.13 to 20.23; P = .02).

The investigators acknowledged that some of the findings were limited by the study’s short observation period.

Patients without excess myocardial iron concentration at baseline did not see significant changes associated with amlodipine. While Dr. Fernandes and his colleagues could not conclude that the drug prevented excess cardiac iron from accumulating, “our data cannot rule out the possibility that extended use of amlodipine might prevent myocardial iron accumulation with a longer observation period.”

Secondary endpoints of the study included measurements of iron storage in the liver and of serum ferritin, neither of which appeared to be affected by amlodipine treatment, which the investigators said was consistent with the drug’s known mechanism of action. No serious adverse effects were reported related to amlodipine treatment.

Dr. Fernandes and his colleagues also did not find improvements in left ventricular ejection fraction associated with amlodipine use at 12 months. This may be due, they wrote in their analysis, to a “relatively low prevalence of reduced ejection fraction or severe myocardial siderosis upon trial enrollment, limiting the power of the study to assess these outcomes.”

The government of Brazil and the Sultan Bin Khalifa Translational Research Scholarship sponsored the study. Dr. Fernandes reported receiving fees from Novartis and Sanofi. The remaining 12 authors disclosed no conflicts of interest.

The calcium channel blocker amlodipine, added to iron chelation therapy, significantly reduced excess myocardial iron concentration in patients with thalassemia major, compared with chelation alone, according to results from a randomized trial.

The findings (Blood. 2016;128[12]:1555-61) suggest that amlodipine, a cheap, widely available drug with a well-established safety profile, may serve as an adjunct to standard treatment for people with thalassemia major and cardiac siderosis. Cardiovascular disease caused by excess myocardial iron remains a major cause of morbidity and mortality in thalassemia major.

Juliano L. Fernandes, MD, PhD, of the Jose Michel Kalaf Research Institute in Campinas, Brazil, led the study, which randomized 62 patients already receiving chelation treatment for thalassemia major to 1 year of chelation plus placebo (n = 31) or chelation plus 5 mg daily amlodipine (n = 31).

Patients in each arm were subdivided into two subgroups: those whose baseline myocardial iron concentration was within normal thresholds, and those with excess myocardial iron concentration as measured by magnetic resonance imaging (above 0.59 mg/g dry weight or with a cardiac T2* below 35 milliseconds).

In the amlodipine arm, patients with excess cardiac iron at baseline (n = 15) saw significant reductions in myocardial iron concentrations at 1 year, compared with those randomized to placebo (n = 15). The former had a median reduction of –0.26 mg/g (95% confidence interval, –1.02 to –0.01) while the placebo group saw an increase of 0.01 mg/g (95% CI, 20.13 to 20.23; P = .02).

The investigators acknowledged that some of the findings were limited by the study’s short observation period.

Patients without excess myocardial iron concentration at baseline did not see significant changes associated with amlodipine. While Dr. Fernandes and his colleagues could not conclude that the drug prevented excess cardiac iron from accumulating, “our data cannot rule out the possibility that extended use of amlodipine might prevent myocardial iron accumulation with a longer observation period.”

Secondary endpoints of the study included measurements of iron storage in the liver and of serum ferritin, neither of which appeared to be affected by amlodipine treatment, which the investigators said was consistent with the drug’s known mechanism of action. No serious adverse effects were reported related to amlodipine treatment.

Dr. Fernandes and his colleagues also did not find improvements in left ventricular ejection fraction associated with amlodipine use at 12 months. This may be due, they wrote in their analysis, to a “relatively low prevalence of reduced ejection fraction or severe myocardial siderosis upon trial enrollment, limiting the power of the study to assess these outcomes.”

The government of Brazil and the Sultan Bin Khalifa Translational Research Scholarship sponsored the study. Dr. Fernandes reported receiving fees from Novartis and Sanofi. The remaining 12 authors disclosed no conflicts of interest.

The calcium channel blocker amlodipine, added to iron chelation therapy, significantly reduced excess myocardial iron concentration in patients with thalassemia major, compared with chelation alone, according to results from a randomized trial.

The findings (Blood. 2016;128[12]:1555-61) suggest that amlodipine, a cheap, widely available drug with a well-established safety profile, may serve as an adjunct to standard treatment for people with thalassemia major and cardiac siderosis. Cardiovascular disease caused by excess myocardial iron remains a major cause of morbidity and mortality in thalassemia major.

Juliano L. Fernandes, MD, PhD, of the Jose Michel Kalaf Research Institute in Campinas, Brazil, led the study, which randomized 62 patients already receiving chelation treatment for thalassemia major to 1 year of chelation plus placebo (n = 31) or chelation plus 5 mg daily amlodipine (n = 31).

Patients in each arm were subdivided into two subgroups: those whose baseline myocardial iron concentration was within normal thresholds, and those with excess myocardial iron concentration as measured by magnetic resonance imaging (above 0.59 mg/g dry weight or with a cardiac T2* below 35 milliseconds).

In the amlodipine arm, patients with excess cardiac iron at baseline (n = 15) saw significant reductions in myocardial iron concentrations at 1 year, compared with those randomized to placebo (n = 15). The former had a median reduction of –0.26 mg/g (95% confidence interval, –1.02 to –0.01) while the placebo group saw an increase of 0.01 mg/g (95% CI, 20.13 to 20.23; P = .02).

The investigators acknowledged that some of the findings were limited by the study’s short observation period.

Patients without excess myocardial iron concentration at baseline did not see significant changes associated with amlodipine. While Dr. Fernandes and his colleagues could not conclude that the drug prevented excess cardiac iron from accumulating, “our data cannot rule out the possibility that extended use of amlodipine might prevent myocardial iron accumulation with a longer observation period.”

Secondary endpoints of the study included measurements of iron storage in the liver and of serum ferritin, neither of which appeared to be affected by amlodipine treatment, which the investigators said was consistent with the drug’s known mechanism of action. No serious adverse effects were reported related to amlodipine treatment.

Dr. Fernandes and his colleagues also did not find improvements in left ventricular ejection fraction associated with amlodipine use at 12 months. This may be due, they wrote in their analysis, to a “relatively low prevalence of reduced ejection fraction or severe myocardial siderosis upon trial enrollment, limiting the power of the study to assess these outcomes.”

The government of Brazil and the Sultan Bin Khalifa Translational Research Scholarship sponsored the study. Dr. Fernandes reported receiving fees from Novartis and Sanofi. The remaining 12 authors disclosed no conflicts of interest.

LONDON – Measuring serum levels of the extracellular protein periostin could help identify patients with asthma who have comorbid upper-airway disease, according to research from a late-breaking oral abstract presented at the annual congress of the European Respiratory Society.

Periostin is known to be involved in the pathophysiology of upper-airway disease, linked to both airway remodeling and inflammation. Levels of periostin have been shown to be higher in patients with asthma, allergic rhinitis, and chronic rhinosinusitis than in healthy individuals, and there are data suggesting that it could be linked to severity. However, little is known about whether serum periostin can be used to detect and perhaps monitor upper-airway disease in patients with asthma.

Serum periostin levels were found to be higher in asthma patients with comorbid chronic rhinosinusitis, compared with those with no comorbid upper-airway disease (119.8 vs. 86.3 ng/mL; P = .04).

Levels of periostin in the serum were also significantly higher in asthma patients with chronic rhinosinusitis who also had nasal polyps than in those who did not have nasal polyps (143.3 vs. 94.0 ng/mL; P = .01).

“Serum periostin is a sensitive biomarker for detecting comorbid chronic rhinosinusitis, especially in patients with chronic rhinosinusitis with nasal polyps,” said Dr. Takamitsu Asano, who reported the findings at the meeting. Dr. Asano of Nagoya (Japan) City University Graduate School of Medical Sciences noted that the serum periostin could also reflect the severity of chronic rhinosinusitis in patients with asthma.

“Upper-airway diseases are considered risk factors for the exacerbation and poor control of asthma,” Dr. Asano explained. It is thought, he said, that 40%-50% of patients with asthma have comorbid chronic rhinosinusitis, and 57%-70% have comorbid allergic rhinitis.

Dr. Asano and colleagues at Nagoya City University and Saga (Japan) Medical School recruited 65 patients with stable asthma, with or without comorbid upper-airway disease, between July 2014 and December 2015. Of these patients, 20 had no comorbid upper-airway disease, 22 had rhinitis, 21 had chronic rhinosinusitis, and two had confirmed nasal polyps. Of the 21 patients with chronic rhinosinusitis, 10 had and 11 did not have nasal polyps. Patients’ diagnoses were checked by otorhinolaryngology specialists.

The recruited patients, who were a mean age of 56 years, with an asthma disease duration of 9 years, underwent the following tests: fractional exhaled nitric oxide testing, spirometry, sputum induction, and sinus computed tomography scanning. They also had their blood tested for serum periostin, eosinophils, eotaxin, and immunoglobulin E levels.

Serum periostin levels were found to correlate with the results of fractional exhaled nitric oxide testing, and with blood and sputum eosinophil counts, with respective correlation coefficients of 0.36 (P = .003), 0.35 (P = .005), and 0.44 (P = .004).

In a separate study presented by Dr. Cecile Holweg of Genentech, several patient factors were found to influence the level of serum periostin. Using data from trials of the experimental asthma therapy lebrikizumab and the asthma therapy omalizumab (Xolair), the biologic variability in serum periostin and blood eosinophils was characterized according to patient demographics, asthma severity, concomitant medication use, and comorbidities, including upper-airway diseases.

Multivariate analysis showed that serum periostin levels were higher in patients with nasal polyps and in patients from South America and Asia. Conversely, serum periostin levels were lower in patients with high body mass index and in patients who were current smokers.

Raised eosinophil counts were also seen in patients with nasal polyps and lower eosinophil counts were observed in current smokers.

“These factors should be taken into account when making treatment decisions based on these biomarkers,” Dr. Holweg concluded.

Dr. Asano has received financial support from Novartis, AstraZeneca, Merck Sharp & Dohme, and Eisai Co. Dr. Holweg is an employee of Genentech.

chestphysician@frontlinemedcom.com

LONDON – Measuring serum levels of the extracellular protein periostin could help identify patients with asthma who have comorbid upper-airway disease, according to research from a late-breaking oral abstract presented at the annual congress of the European Respiratory Society.

Periostin is known to be involved in the pathophysiology of upper-airway disease, linked to both airway remodeling and inflammation. Levels of periostin have been shown to be higher in patients with asthma, allergic rhinitis, and chronic rhinosinusitis than in healthy individuals, and there are data suggesting that it could be linked to severity. However, little is known about whether serum periostin can be used to detect and perhaps monitor upper-airway disease in patients with asthma.

Serum periostin levels were found to be higher in asthma patients with comorbid chronic rhinosinusitis, compared with those with no comorbid upper-airway disease (119.8 vs. 86.3 ng/mL; P = .04).

Levels of periostin in the serum were also significantly higher in asthma patients with chronic rhinosinusitis who also had nasal polyps than in those who did not have nasal polyps (143.3 vs. 94.0 ng/mL; P = .01).

“Serum periostin is a sensitive biomarker for detecting comorbid chronic rhinosinusitis, especially in patients with chronic rhinosinusitis with nasal polyps,” said Dr. Takamitsu Asano, who reported the findings at the meeting. Dr. Asano of Nagoya (Japan) City University Graduate School of Medical Sciences noted that the serum periostin could also reflect the severity of chronic rhinosinusitis in patients with asthma.

“Upper-airway diseases are considered risk factors for the exacerbation and poor control of asthma,” Dr. Asano explained. It is thought, he said, that 40%-50% of patients with asthma have comorbid chronic rhinosinusitis, and 57%-70% have comorbid allergic rhinitis.

Dr. Asano and colleagues at Nagoya City University and Saga (Japan) Medical School recruited 65 patients with stable asthma, with or without comorbid upper-airway disease, between July 2014 and December 2015. Of these patients, 20 had no comorbid upper-airway disease, 22 had rhinitis, 21 had chronic rhinosinusitis, and two had confirmed nasal polyps. Of the 21 patients with chronic rhinosinusitis, 10 had and 11 did not have nasal polyps. Patients’ diagnoses were checked by otorhinolaryngology specialists.

The recruited patients, who were a mean age of 56 years, with an asthma disease duration of 9 years, underwent the following tests: fractional exhaled nitric oxide testing, spirometry, sputum induction, and sinus computed tomography scanning. They also had their blood tested for serum periostin, eosinophils, eotaxin, and immunoglobulin E levels.

Serum periostin levels were found to correlate with the results of fractional exhaled nitric oxide testing, and with blood and sputum eosinophil counts, with respective correlation coefficients of 0.36 (P = .003), 0.35 (P = .005), and 0.44 (P = .004).

In a separate study presented by Dr. Cecile Holweg of Genentech, several patient factors were found to influence the level of serum periostin. Using data from trials of the experimental asthma therapy lebrikizumab and the asthma therapy omalizumab (Xolair), the biologic variability in serum periostin and blood eosinophils was characterized according to patient demographics, asthma severity, concomitant medication use, and comorbidities, including upper-airway diseases.

Multivariate analysis showed that serum periostin levels were higher in patients with nasal polyps and in patients from South America and Asia. Conversely, serum periostin levels were lower in patients with high body mass index and in patients who were current smokers.

Raised eosinophil counts were also seen in patients with nasal polyps and lower eosinophil counts were observed in current smokers.

“These factors should be taken into account when making treatment decisions based on these biomarkers,” Dr. Holweg concluded.

Dr. Asano has received financial support from Novartis, AstraZeneca, Merck Sharp & Dohme, and Eisai Co. Dr. Holweg is an employee of Genentech.

chestphysician@frontlinemedcom.com

LONDON – Measuring serum levels of the extracellular protein periostin could help identify patients with asthma who have comorbid upper-airway disease, according to research from a late-breaking oral abstract presented at the annual congress of the European Respiratory Society.

Periostin is known to be involved in the pathophysiology of upper-airway disease, linked to both airway remodeling and inflammation. Levels of periostin have been shown to be higher in patients with asthma, allergic rhinitis, and chronic rhinosinusitis than in healthy individuals, and there are data suggesting that it could be linked to severity. However, little is known about whether serum periostin can be used to detect and perhaps monitor upper-airway disease in patients with asthma.

Serum periostin levels were found to be higher in asthma patients with comorbid chronic rhinosinusitis, compared with those with no comorbid upper-airway disease (119.8 vs. 86.3 ng/mL; P = .04).

Levels of periostin in the serum were also significantly higher in asthma patients with chronic rhinosinusitis who also had nasal polyps than in those who did not have nasal polyps (143.3 vs. 94.0 ng/mL; P = .01).

“Serum periostin is a sensitive biomarker for detecting comorbid chronic rhinosinusitis, especially in patients with chronic rhinosinusitis with nasal polyps,” said Dr. Takamitsu Asano, who reported the findings at the meeting. Dr. Asano of Nagoya (Japan) City University Graduate School of Medical Sciences noted that the serum periostin could also reflect the severity of chronic rhinosinusitis in patients with asthma.

“Upper-airway diseases are considered risk factors for the exacerbation and poor control of asthma,” Dr. Asano explained. It is thought, he said, that 40%-50% of patients with asthma have comorbid chronic rhinosinusitis, and 57%-70% have comorbid allergic rhinitis.

Dr. Asano and colleagues at Nagoya City University and Saga (Japan) Medical School recruited 65 patients with stable asthma, with or without comorbid upper-airway disease, between July 2014 and December 2015. Of these patients, 20 had no comorbid upper-airway disease, 22 had rhinitis, 21 had chronic rhinosinusitis, and two had confirmed nasal polyps. Of the 21 patients with chronic rhinosinusitis, 10 had and 11 did not have nasal polyps. Patients’ diagnoses were checked by otorhinolaryngology specialists.

The recruited patients, who were a mean age of 56 years, with an asthma disease duration of 9 years, underwent the following tests: fractional exhaled nitric oxide testing, spirometry, sputum induction, and sinus computed tomography scanning. They also had their blood tested for serum periostin, eosinophils, eotaxin, and immunoglobulin E levels.

Serum periostin levels were found to correlate with the results of fractional exhaled nitric oxide testing, and with blood and sputum eosinophil counts, with respective correlation coefficients of 0.36 (P = .003), 0.35 (P = .005), and 0.44 (P = .004).

In a separate study presented by Dr. Cecile Holweg of Genentech, several patient factors were found to influence the level of serum periostin. Using data from trials of the experimental asthma therapy lebrikizumab and the asthma therapy omalizumab (Xolair), the biologic variability in serum periostin and blood eosinophils was characterized according to patient demographics, asthma severity, concomitant medication use, and comorbidities, including upper-airway diseases.

Multivariate analysis showed that serum periostin levels were higher in patients with nasal polyps and in patients from South America and Asia. Conversely, serum periostin levels were lower in patients with high body mass index and in patients who were current smokers.

Raised eosinophil counts were also seen in patients with nasal polyps and lower eosinophil counts were observed in current smokers.

“These factors should be taken into account when making treatment decisions based on these biomarkers,” Dr. Holweg concluded.

Dr. Asano has received financial support from Novartis, AstraZeneca, Merck Sharp & Dohme, and Eisai Co. Dr. Holweg is an employee of Genentech.

chestphysician@frontlinemedcom.com

Sometimes it seems like cardiac troponin testing has become nearly as ubiquitous as the CBC and the BMP. Concern over atypical presentations of MI has contributed to widespread use in emergency departments and hospitalized patients. But once the test comes back elevated, what do you do with that information?

Typically, the next step is to consult Cardiology, which is a reasonable request with or without a suspicion of MI. Frequently, invasive management is not an option; or perhaps the diagnosis is “type 2 MI.”¹

Courtesy University of Florida

1. Thygesen K, Alpert JS, et al. Third universal definition of myocardial infarction. J Am Coll Cardiol. 2012;60:1581-98.

2. Alcalai R, Planer D, Culhaoglu A, Osman A, Pollak A and Lotan C. Acute coronary syndrome vs nonspecific troponin elevation: clinical predictors and survival analysis. Arch Intern Med. 2007;167:276-81.

3. Winchester DE, Burke L, Agarwal N, Schmalfuss C and Pepine CJ. Predictors of short- and long-term mortality in hospitalized veterans with elevated troponin. J Hosp Med. 2016 Jun 3. doi: 10.1002/jhm.2619.

David Winchester, MD, is assistant professor in the division of cardiovascular medicine at the University of Florida (Gainesville), and practices general cardiology at the Malcom Randall VA Medical Center, Gainesville.

Sometimes it seems like cardiac troponin testing has become nearly as ubiquitous as the CBC and the BMP. Concern over atypical presentations of MI has contributed to widespread use in emergency departments and hospitalized patients. But once the test comes back elevated, what do you do with that information?

Typically, the next step is to consult Cardiology, which is a reasonable request with or without a suspicion of MI. Frequently, invasive management is not an option; or perhaps the diagnosis is “type 2 MI.”¹

Courtesy University of Florida

1. Thygesen K, Alpert JS, et al. Third universal definition of myocardial infarction. J Am Coll Cardiol. 2012;60:1581-98.

2. Alcalai R, Planer D, Culhaoglu A, Osman A, Pollak A and Lotan C. Acute coronary syndrome vs nonspecific troponin elevation: clinical predictors and survival analysis. Arch Intern Med. 2007;167:276-81.

3. Winchester DE, Burke L, Agarwal N, Schmalfuss C and Pepine CJ. Predictors of short- and long-term mortality in hospitalized veterans with elevated troponin. J Hosp Med. 2016 Jun 3. doi: 10.1002/jhm.2619.

David Winchester, MD, is assistant professor in the division of cardiovascular medicine at the University of Florida (Gainesville), and practices general cardiology at the Malcom Randall VA Medical Center, Gainesville.

Sometimes it seems like cardiac troponin testing has become nearly as ubiquitous as the CBC and the BMP. Concern over atypical presentations of MI has contributed to widespread use in emergency departments and hospitalized patients. But once the test comes back elevated, what do you do with that information?

Typically, the next step is to consult Cardiology, which is a reasonable request with or without a suspicion of MI. Frequently, invasive management is not an option; or perhaps the diagnosis is “type 2 MI.”¹

Courtesy University of Florida

1. Thygesen K, Alpert JS, et al. Third universal definition of myocardial infarction. J Am Coll Cardiol. 2012;60:1581-98.

2. Alcalai R, Planer D, Culhaoglu A, Osman A, Pollak A and Lotan C. Acute coronary syndrome vs nonspecific troponin elevation: clinical predictors and survival analysis. Arch Intern Med. 2007;167:276-81.

3. Winchester DE, Burke L, Agarwal N, Schmalfuss C and Pepine CJ. Predictors of short- and long-term mortality in hospitalized veterans with elevated troponin. J Hosp Med. 2016 Jun 3. doi: 10.1002/jhm.2619.

David Winchester, MD, is assistant professor in the division of cardiovascular medicine at the University of Florida (Gainesville), and practices general cardiology at the Malcom Randall VA Medical Center, Gainesville.

LONDON – The vast majority of surgical lung biopsies currently used to diagnose interstitial lung diseases (ILDs) could be avoided, suggests research presented at the annual congress of the European Respiratory Society.

During an oral presentation, Benjamin Bondue, MD, of Hopital Erasme, Brussels, presented the preliminary results of a Belgian prospective study evaluating the role of transbronchial lung cryobiopsies in 24 patients with undefined ILD treated at three participating centers.

Cryobiopsies were found to have a diagnostic yield of 79%, meaning that patients might be able to avoid undergoing a more invasive surgical removal of tissue in many cases. Compared with surgical biopsy, cryobiopsies offered the potential advantage of lower morbidity and shorter hospitalization time, Dr. Bondue said. He reported that patients needed to stay in hospital just 1.2 days after the procedure in the study.

“Our data also show that there is some benefit of surgical lung biopsy after cryobiopsy if we identify an NSIP [nonspecific interstitial pneumonia] pattern or idiopathic conditions, or if we cannot obtain a clear pathological diagnosis,” he reported. Acknowledging the study was small and conducted in a single center, he said the use of cryobiopsies following surgical biopsy might be worth further study.

Transbronchial lung cryobiopsy is a relatively new technique that uses a cryoprobe inserted down through a bronchoscope about 1-2 cm from the thoracic wall. Once in place, the probe is cooled for between 3 and 6 seconds, lung tissue freezes to the probe, and the probe and bronchoscope are removed together. This method allows for larger samples of tissue to be taken than does traditional transbronchial biopsy, which involves using large forceps to obtain tissue samples (Respirology. 2014;19:645-54).

In the Belgian study, Dr. Bondue noted that a Fogarty balloon was used to control any bleeding and that four transbronchial lung cryobiopsies were obtained from two different segments of the same lobe of a patient’s lungs. All biopsies were then analyzed by an expert pathologist in ILDs, and reviewed by two other expert pathologists when needed. The mean sample size obtained was 16 mm².

The patients included in the study had undergone chest X-ray and had inconclusive findings in the majority (84%) of cases. They then had the option to undergo cryobiopsy or surgical lung biopsy, with the latter performed following discussion among a multidisciplinary team’s members.

Following cryobiopsy, 16 of the 24 patients – who were a mean age of 62 years, and over half of whom were past (56%) or current (12%) smokers – were diagnosed with a specific pattern of ILD not due to NSIP. Of the 16 cases, 6 were due to hypersensitive pneumonitis, 4 were due to interstitial pulmonary fibrosis, and 2 were due to sarcoidosis. The other four cases included patients with one of the following conditions: adenocarcinoma, desquamative interstitial pneumonia, eosinophilic pneumonia, and amyloidosis.

Six of the 24 cases were defined as NSIP, with 2 reclassified as definite and 1 as probable hypersensitive pneumonitis, after discussion within the multidisciplinary team.

Five patients – three who had been diagnosed with NSIP and two who had been given no pathological diagnosis after cryobiopsy – underwent surgical lung biopsy. Of these, following the surgical biopsies, only one patient was considered to have NSIP and the other four were eventually diagnosed with interstitial pulmonary fibrosis.

In terms of safety, five patients experienced pneumothorax, two patients required chest drainage, two needed simple aspiration and one underwent observation. In the majority of cases, patients experienced mild bleeding, with only one patient having experienced severe bleeding. During this study, none of the participants experienced significant chest pain, acute exacerbations, or infections, and none of them died.

Dr. Bondue has received research grants and fees for consulting from Boehringer Ingelheim and Roche.

LONDON – The vast majority of surgical lung biopsies currently used to diagnose interstitial lung diseases (ILDs) could be avoided, suggests research presented at the annual congress of the European Respiratory Society.

During an oral presentation, Benjamin Bondue, MD, of Hopital Erasme, Brussels, presented the preliminary results of a Belgian prospective study evaluating the role of transbronchial lung cryobiopsies in 24 patients with undefined ILD treated at three participating centers.

Cryobiopsies were found to have a diagnostic yield of 79%, meaning that patients might be able to avoid undergoing a more invasive surgical removal of tissue in many cases. Compared with surgical biopsy, cryobiopsies offered the potential advantage of lower morbidity and shorter hospitalization time, Dr. Bondue said. He reported that patients needed to stay in hospital just 1.2 days after the procedure in the study.

“Our data also show that there is some benefit of surgical lung biopsy after cryobiopsy if we identify an NSIP [nonspecific interstitial pneumonia] pattern or idiopathic conditions, or if we cannot obtain a clear pathological diagnosis,” he reported. Acknowledging the study was small and conducted in a single center, he said the use of cryobiopsies following surgical biopsy might be worth further study.

Transbronchial lung cryobiopsy is a relatively new technique that uses a cryoprobe inserted down through a bronchoscope about 1-2 cm from the thoracic wall. Once in place, the probe is cooled for between 3 and 6 seconds, lung tissue freezes to the probe, and the probe and bronchoscope are removed together. This method allows for larger samples of tissue to be taken than does traditional transbronchial biopsy, which involves using large forceps to obtain tissue samples (Respirology. 2014;19:645-54).

In the Belgian study, Dr. Bondue noted that a Fogarty balloon was used to control any bleeding and that four transbronchial lung cryobiopsies were obtained from two different segments of the same lobe of a patient’s lungs. All biopsies were then analyzed by an expert pathologist in ILDs, and reviewed by two other expert pathologists when needed. The mean sample size obtained was 16 mm².

The patients included in the study had undergone chest X-ray and had inconclusive findings in the majority (84%) of cases. They then had the option to undergo cryobiopsy or surgical lung biopsy, with the latter performed following discussion among a multidisciplinary team’s members.

Following cryobiopsy, 16 of the 24 patients – who were a mean age of 62 years, and over half of whom were past (56%) or current (12%) smokers – were diagnosed with a specific pattern of ILD not due to NSIP. Of the 16 cases, 6 were due to hypersensitive pneumonitis, 4 were due to interstitial pulmonary fibrosis, and 2 were due to sarcoidosis. The other four cases included patients with one of the following conditions: adenocarcinoma, desquamative interstitial pneumonia, eosinophilic pneumonia, and amyloidosis.

Six of the 24 cases were defined as NSIP, with 2 reclassified as definite and 1 as probable hypersensitive pneumonitis, after discussion within the multidisciplinary team.

Five patients – three who had been diagnosed with NSIP and two who had been given no pathological diagnosis after cryobiopsy – underwent surgical lung biopsy. Of these, following the surgical biopsies, only one patient was considered to have NSIP and the other four were eventually diagnosed with interstitial pulmonary fibrosis.

In terms of safety, five patients experienced pneumothorax, two patients required chest drainage, two needed simple aspiration and one underwent observation. In the majority of cases, patients experienced mild bleeding, with only one patient having experienced severe bleeding. During this study, none of the participants experienced significant chest pain, acute exacerbations, or infections, and none of them died.

Dr. Bondue has received research grants and fees for consulting from Boehringer Ingelheim and Roche.

LONDON – The vast majority of surgical lung biopsies currently used to diagnose interstitial lung diseases (ILDs) could be avoided, suggests research presented at the annual congress of the European Respiratory Society.

During an oral presentation, Benjamin Bondue, MD, of Hopital Erasme, Brussels, presented the preliminary results of a Belgian prospective study evaluating the role of transbronchial lung cryobiopsies in 24 patients with undefined ILD treated at three participating centers.

Cryobiopsies were found to have a diagnostic yield of 79%, meaning that patients might be able to avoid undergoing a more invasive surgical removal of tissue in many cases. Compared with surgical biopsy, cryobiopsies offered the potential advantage of lower morbidity and shorter hospitalization time, Dr. Bondue said. He reported that patients needed to stay in hospital just 1.2 days after the procedure in the study.

“Our data also show that there is some benefit of surgical lung biopsy after cryobiopsy if we identify an NSIP [nonspecific interstitial pneumonia] pattern or idiopathic conditions, or if we cannot obtain a clear pathological diagnosis,” he reported. Acknowledging the study was small and conducted in a single center, he said the use of cryobiopsies following surgical biopsy might be worth further study.

Transbronchial lung cryobiopsy is a relatively new technique that uses a cryoprobe inserted down through a bronchoscope about 1-2 cm from the thoracic wall. Once in place, the probe is cooled for between 3 and 6 seconds, lung tissue freezes to the probe, and the probe and bronchoscope are removed together. This method allows for larger samples of tissue to be taken than does traditional transbronchial biopsy, which involves using large forceps to obtain tissue samples (Respirology. 2014;19:645-54).

In the Belgian study, Dr. Bondue noted that a Fogarty balloon was used to control any bleeding and that four transbronchial lung cryobiopsies were obtained from two different segments of the same lobe of a patient’s lungs. All biopsies were then analyzed by an expert pathologist in ILDs, and reviewed by two other expert pathologists when needed. The mean sample size obtained was 16 mm².

The patients included in the study had undergone chest X-ray and had inconclusive findings in the majority (84%) of cases. They then had the option to undergo cryobiopsy or surgical lung biopsy, with the latter performed following discussion among a multidisciplinary team’s members.

Following cryobiopsy, 16 of the 24 patients – who were a mean age of 62 years, and over half of whom were past (56%) or current (12%) smokers – were diagnosed with a specific pattern of ILD not due to NSIP. Of the 16 cases, 6 were due to hypersensitive pneumonitis, 4 were due to interstitial pulmonary fibrosis, and 2 were due to sarcoidosis. The other four cases included patients with one of the following conditions: adenocarcinoma, desquamative interstitial pneumonia, eosinophilic pneumonia, and amyloidosis.

Six of the 24 cases were defined as NSIP, with 2 reclassified as definite and 1 as probable hypersensitive pneumonitis, after discussion within the multidisciplinary team.

Five patients – three who had been diagnosed with NSIP and two who had been given no pathological diagnosis after cryobiopsy – underwent surgical lung biopsy. Of these, following the surgical biopsies, only one patient was considered to have NSIP and the other four were eventually diagnosed with interstitial pulmonary fibrosis.

In terms of safety, five patients experienced pneumothorax, two patients required chest drainage, two needed simple aspiration and one underwent observation. In the majority of cases, patients experienced mild bleeding, with only one patient having experienced severe bleeding. During this study, none of the participants experienced significant chest pain, acute exacerbations, or infections, and none of them died.

Dr. Bondue has received research grants and fees for consulting from Boehringer Ingelheim and Roche.

HIV incidence in the United States declined by approximately 26% since 2003, based on a study using a new and simple method of estimating the number of new cases.

“Previously, the number of newly diagnosed HIV cases in a year was used as a proxy measure for HIV incidence but has never been used as the sole data source to calculate HIV incidence, because HIV infection can remain asymptomatic for many years and newly reported cases represent both recent and long-standing infections,” wrote Qiang Xia, MD, of the New York City Department of Health and Mental Hygiene, and coauthors.

©grandeduc/Thinkstock

HIV incidence in the United States declined by approximately 26% since 2003, based on a study using a new and simple method of estimating the number of new cases.

“Previously, the number of newly diagnosed HIV cases in a year was used as a proxy measure for HIV incidence but has never been used as the sole data source to calculate HIV incidence, because HIV infection can remain asymptomatic for many years and newly reported cases represent both recent and long-standing infections,” wrote Qiang Xia, MD, of the New York City Department of Health and Mental Hygiene, and coauthors.

©grandeduc/Thinkstock

HIV incidence in the United States declined by approximately 26% since 2003, based on a study using a new and simple method of estimating the number of new cases.

“Previously, the number of newly diagnosed HIV cases in a year was used as a proxy measure for HIV incidence but has never been used as the sole data source to calculate HIV incidence, because HIV infection can remain asymptomatic for many years and newly reported cases represent both recent and long-standing infections,” wrote Qiang Xia, MD, of the New York City Department of Health and Mental Hygiene, and coauthors.

©grandeduc/Thinkstock

The Food and Drug Administration has approved ustekinumab (Stelara) for the treatment of moderately to severely active Crohn’s disease in certain adults.

Specifically, the new approval for the human monoclonal antibody, which was previously approved for the treatment of psoriasis and psoriatic arthritis, is for Crohn’s disease patients aged 18 years or older who fail or cannot tolerate treatment with immunomodulators or corticosteroids but who never failed treatment with a tumor necrosis factor (TNF) blocker, or who fail or cannot tolerate treatment with one or more TNF blockers, according to a statement from the drug’s maker, Janssen Biotech.

sworcester@frontlinemedcom.com

The Food and Drug Administration has approved ustekinumab (Stelara) for the treatment of moderately to severely active Crohn’s disease in certain adults.

Specifically, the new approval for the human monoclonal antibody, which was previously approved for the treatment of psoriasis and psoriatic arthritis, is for Crohn’s disease patients aged 18 years or older who fail or cannot tolerate treatment with immunomodulators or corticosteroids but who never failed treatment with a tumor necrosis factor (TNF) blocker, or who fail or cannot tolerate treatment with one or more TNF blockers, according to a statement from the drug’s maker, Janssen Biotech.

sworcester@frontlinemedcom.com

The Food and Drug Administration has approved ustekinumab (Stelara) for the treatment of moderately to severely active Crohn’s disease in certain adults.

Specifically, the new approval for the human monoclonal antibody, which was previously approved for the treatment of psoriasis and psoriatic arthritis, is for Crohn’s disease patients aged 18 years or older who fail or cannot tolerate treatment with immunomodulators or corticosteroids but who never failed treatment with a tumor necrosis factor (TNF) blocker, or who fail or cannot tolerate treatment with one or more TNF blockers, according to a statement from the drug’s maker, Janssen Biotech.

sworcester@frontlinemedcom.com

The SVS has announced the launch of the SVS Member Affinity Program, expanding member benefits to eventually provide products and services to assist members at every step throughout their career, from medical school to retirement.

The first new benefit is a student loan refinancing partnership with SoFi, the market leader in student loan refinancing.

Benefits include average savings of more than $600 a month for physician borrowers plus a $300 welcome bonus for SVS borrowers (terms and conditions apply). Visit here for more information.

Short informational webinars will be held at 5 p.m. CST Tuesday, Oct. 11, and at 11 a.m. CST Friday, Oct. 14. Register here. 

The SVS has announced the launch of the SVS Member Affinity Program, expanding member benefits to eventually provide products and services to assist members at every step throughout their career, from medical school to retirement.

The first new benefit is a student loan refinancing partnership with SoFi, the market leader in student loan refinancing.

Benefits include average savings of more than $600 a month for physician borrowers plus a $300 welcome bonus for SVS borrowers (terms and conditions apply). Visit here for more information.

Short informational webinars will be held at 5 p.m. CST Tuesday, Oct. 11, and at 11 a.m. CST Friday, Oct. 14. Register here. 

The SVS has announced the launch of the SVS Member Affinity Program, expanding member benefits to eventually provide products and services to assist members at every step throughout their career, from medical school to retirement.

The first new benefit is a student loan refinancing partnership with SoFi, the market leader in student loan refinancing.

Benefits include average savings of more than $600 a month for physician borrowers plus a $300 welcome bonus for SVS borrowers (terms and conditions apply). Visit here for more information.

Short informational webinars will be held at 5 p.m. CST Tuesday, Oct. 11, and at 11 a.m. CST Friday, Oct. 14. Register here. 

New federal regulations aim to strengthen the current requirements on reporting of clinical trial information.

The new rule offers a checklist to help determine which clinical trials are subject to regulations and who is responsible for information that appears on ClinicalTrials.gov. In addition, the rule does the following:

• Expands the scope of trials for which results must be submitted to include Food and Drug Administration–regulated products that have not yet been approved, licensed, or cleared.

• Requires additional information be submitted to ClinicalTrials.gov, including demographic information about trial participants.

• Expands adverse-event reporting.

• Adds potential penalties for noncompliance.

ClinicalTrials.gov

gtwachtman@frontlinemedcom.com

New federal regulations aim to strengthen the current requirements on reporting of clinical trial information.

The new rule offers a checklist to help determine which clinical trials are subject to regulations and who is responsible for information that appears on ClinicalTrials.gov. In addition, the rule does the following:

• Expands the scope of trials for which results must be submitted to include Food and Drug Administration–regulated products that have not yet been approved, licensed, or cleared.

• Requires additional information be submitted to ClinicalTrials.gov, including demographic information about trial participants.

• Expands adverse-event reporting.

• Adds potential penalties for noncompliance.

ClinicalTrials.gov

gtwachtman@frontlinemedcom.com

New federal regulations aim to strengthen the current requirements on reporting of clinical trial information.

The new rule offers a checklist to help determine which clinical trials are subject to regulations and who is responsible for information that appears on ClinicalTrials.gov. In addition, the rule does the following:

• Expands the scope of trials for which results must be submitted to include Food and Drug Administration–regulated products that have not yet been approved, licensed, or cleared.

• Requires additional information be submitted to ClinicalTrials.gov, including demographic information about trial participants.

• Expands adverse-event reporting.

• Adds potential penalties for noncompliance.

ClinicalTrials.gov

gtwachtman@frontlinemedcom.com

NEW ORLEANS – For adolescents and children with type 2 diabetes, there aren’t a lot of therapeutic options other than insulin and metformin. And the situation isn’t likely to change without extraordinary collaboration, Kristen J. Nadeau, MD, research director of the department of pediatric endocrinology at Children’s Hospital Colorado, Colorado Springs, said at the annual scientific sessions of the American Diabetes Association.

“Type 2 diabetes in youth appears to differ not only from pediatric type 1 diabetes, but also from adult type 2 diabetes, and current treatment options are limited,” Dr. Nadeau said. The estimated number of type 2 diabetes cases in the United States per year stands at 1,469,000 cases (12.3 per 100) in adults, compared with 5,100 (0.5 per 100) in youth. In adults there is a slight male predominance, whereas in kids girls are almost twice as likely as boys to be affected. Moreover, beta cell function declines faster in youth with type 2 diabetes.

The required safety and efficacy studies are too difficult “because of the combination of unique challenges of the target population, study design concerns, and a lack of collaboration between agencies,” Dr. Nadeau said during a session that focused on the conclusions of the American Diabetes Association’s consensus conference on youth with type 2 diabetes, which took place on Oct. 20, 2015 in Alexandria, Va.

The consensus report was published online in Diabetes Care, and addresses the current status of type 2 diabetes in youth, the challenges of treatment, and priorities for research. Dr. Nadeau co-chaired the effort along with Dr. Philip Zeitler, section head of pediatric endocrinology at Children’s Hospital Colorado and medical director of the Children’s Hospital Colorado Clinical and Translational Research Center, Denver. Collaborators included the American Academy of Pediatrics, the International Society for Pediatric and Adolescent Diabetes, and the Pediatric Endocrine Society.

One example of the research challenges is evident in data from the Today trial, which found that only about 39% of kids with type 2 diabetes live with both parents (J Clin Endocrinol Metab. 2011 Jan; 96[1]:159-67). “Whenever you have only one parent in the home, there are difficulties with transportation by definition, so it’s a lot harder for these kids to participate in studies,” Dr. Nadeau said. “In addition, only 17% of their parents had a college or advanced education and 41% had a household income of less than $25,000 per year.”

The social environment is critical, she continued, because the lifestyle factors associated with type 2 diabetes often result in poor outcomes. “It’s very hard to make lifestyle changes if there is a socioeconomic challenge,” she said. “We can’t make change without understanding the community and culture that these youth live in. It’s also critical that we have participation of minorities and other research participants with diverse backgrounds in order for [clinical] trials to be effective for the population that this disease is affecting.”

Another issue keeping drug trials of youth with type 2 diabetes from being completed is the entry criteria. Some studies require youth to be drug naive and have a hemoglobin A1c greater than 7%. “This is difficult, because many youth that are referred to our diabetes center already come in on metformin, leaving only about 7% of subjects available for this criteria,” Dr. Nadeau explained. Another common study entry criterion is being on metformin and having a hemoglobin A1c of about 7%, “so basically being a metformin failure,” she said. “This is difficult to meet because metformin is relatively effective in the early stages of diabetes.”

“We need clear strategies for research, prevention, and treatment. Clarifying unique pathophysiology, complications, and psychosocial impact will enable industry, academia, funding agencies, advocacy groups, and regulators to collectively evaluate the best approaches to research, treatment, and prevention,” Dr. Nadeau said.

The consensus conference participants recommended the following objectives: clarify the biology of type 2 diabetes in youth, obtain new pediatric information on drugs, encourage the use of appropriate medications, and inform clinical decision-making. “We have a desperate need to understand the actions of drugs in type 2 diabetes youth,” Dr. Nadeau said. “Our current approach is not working. Potential solutions include considering efficacy outcomes besides A1c, potentially looking at improvement in insulin sensitivity, preservation of beta-cell function, trying to prevent the A1c increase instead of looking for an A1c reduction, and trying to extrapolate from effects in adults, if we can understand enough to do that.”

The conference participants also called for infrastructure changes, such as creating a resource for patients with type 2 diabetes in the model of the Type 1 Diabetes Exchange. “We need to have collaborations internationally,” she said. “We also need support for teams and clinical groups to work together to be able to accomplish these collaboratively.”

Dr. Nadeau reported having no financial disclosures.

dbrunk@frontlinemedcom.com

NEW ORLEANS – For adolescents and children with type 2 diabetes, there aren’t a lot of therapeutic options other than insulin and metformin. And the situation isn’t likely to change without extraordinary collaboration, Kristen J. Nadeau, MD, research director of the department of pediatric endocrinology at Children’s Hospital Colorado, Colorado Springs, said at the annual scientific sessions of the American Diabetes Association.

“Type 2 diabetes in youth appears to differ not only from pediatric type 1 diabetes, but also from adult type 2 diabetes, and current treatment options are limited,” Dr. Nadeau said. The estimated number of type 2 diabetes cases in the United States per year stands at 1,469,000 cases (12.3 per 100) in adults, compared with 5,100 (0.5 per 100) in youth. In adults there is a slight male predominance, whereas in kids girls are almost twice as likely as boys to be affected. Moreover, beta cell function declines faster in youth with type 2 diabetes.

The required safety and efficacy studies are too difficult “because of the combination of unique challenges of the target population, study design concerns, and a lack of collaboration between agencies,” Dr. Nadeau said during a session that focused on the conclusions of the American Diabetes Association’s consensus conference on youth with type 2 diabetes, which took place on Oct. 20, 2015 in Alexandria, Va.

The consensus report was published online in Diabetes Care, and addresses the current status of type 2 diabetes in youth, the challenges of treatment, and priorities for research. Dr. Nadeau co-chaired the effort along with Dr. Philip Zeitler, section head of pediatric endocrinology at Children’s Hospital Colorado and medical director of the Children’s Hospital Colorado Clinical and Translational Research Center, Denver. Collaborators included the American Academy of Pediatrics, the International Society for Pediatric and Adolescent Diabetes, and the Pediatric Endocrine Society.

One example of the research challenges is evident in data from the Today trial, which found that only about 39% of kids with type 2 diabetes live with both parents (J Clin Endocrinol Metab. 2011 Jan; 96[1]:159-67). “Whenever you have only one parent in the home, there are difficulties with transportation by definition, so it’s a lot harder for these kids to participate in studies,” Dr. Nadeau said. “In addition, only 17% of their parents had a college or advanced education and 41% had a household income of less than $25,000 per year.”

The social environment is critical, she continued, because the lifestyle factors associated with type 2 diabetes often result in poor outcomes. “It’s very hard to make lifestyle changes if there is a socioeconomic challenge,” she said. “We can’t make change without understanding the community and culture that these youth live in. It’s also critical that we have participation of minorities and other research participants with diverse backgrounds in order for [clinical] trials to be effective for the population that this disease is affecting.”

Another issue keeping drug trials of youth with type 2 diabetes from being completed is the entry criteria. Some studies require youth to be drug naive and have a hemoglobin A1c greater than 7%. “This is difficult, because many youth that are referred to our diabetes center already come in on metformin, leaving only about 7% of subjects available for this criteria,” Dr. Nadeau explained. Another common study entry criterion is being on metformin and having a hemoglobin A1c of about 7%, “so basically being a metformin failure,” she said. “This is difficult to meet because metformin is relatively effective in the early stages of diabetes.”

“We need clear strategies for research, prevention, and treatment. Clarifying unique pathophysiology, complications, and psychosocial impact will enable industry, academia, funding agencies, advocacy groups, and regulators to collectively evaluate the best approaches to research, treatment, and prevention,” Dr. Nadeau said.

The consensus conference participants recommended the following objectives: clarify the biology of type 2 diabetes in youth, obtain new pediatric information on drugs, encourage the use of appropriate medications, and inform clinical decision-making. “We have a desperate need to understand the actions of drugs in type 2 diabetes youth,” Dr. Nadeau said. “Our current approach is not working. Potential solutions include considering efficacy outcomes besides A1c, potentially looking at improvement in insulin sensitivity, preservation of beta-cell function, trying to prevent the A1c increase instead of looking for an A1c reduction, and trying to extrapolate from effects in adults, if we can understand enough to do that.”

The conference participants also called for infrastructure changes, such as creating a resource for patients with type 2 diabetes in the model of the Type 1 Diabetes Exchange. “We need to have collaborations internationally,” she said. “We also need support for teams and clinical groups to work together to be able to accomplish these collaboratively.”

Dr. Nadeau reported having no financial disclosures.

dbrunk@frontlinemedcom.com

NEW ORLEANS – For adolescents and children with type 2 diabetes, there aren’t a lot of therapeutic options other than insulin and metformin. And the situation isn’t likely to change without extraordinary collaboration, Kristen J. Nadeau, MD, research director of the department of pediatric endocrinology at Children’s Hospital Colorado, Colorado Springs, said at the annual scientific sessions of the American Diabetes Association.

“Type 2 diabetes in youth appears to differ not only from pediatric type 1 diabetes, but also from adult type 2 diabetes, and current treatment options are limited,” Dr. Nadeau said. The estimated number of type 2 diabetes cases in the United States per year stands at 1,469,000 cases (12.3 per 100) in adults, compared with 5,100 (0.5 per 100) in youth. In adults there is a slight male predominance, whereas in kids girls are almost twice as likely as boys to be affected. Moreover, beta cell function declines faster in youth with type 2 diabetes.

The required safety and efficacy studies are too difficult “because of the combination of unique challenges of the target population, study design concerns, and a lack of collaboration between agencies,” Dr. Nadeau said during a session that focused on the conclusions of the American Diabetes Association’s consensus conference on youth with type 2 diabetes, which took place on Oct. 20, 2015 in Alexandria, Va.

The consensus report was published online in Diabetes Care, and addresses the current status of type 2 diabetes in youth, the challenges of treatment, and priorities for research. Dr. Nadeau co-chaired the effort along with Dr. Philip Zeitler, section head of pediatric endocrinology at Children’s Hospital Colorado and medical director of the Children’s Hospital Colorado Clinical and Translational Research Center, Denver. Collaborators included the American Academy of Pediatrics, the International Society for Pediatric and Adolescent Diabetes, and the Pediatric Endocrine Society.

One example of the research challenges is evident in data from the Today trial, which found that only about 39% of kids with type 2 diabetes live with both parents (J Clin Endocrinol Metab. 2011 Jan; 96[1]:159-67). “Whenever you have only one parent in the home, there are difficulties with transportation by definition, so it’s a lot harder for these kids to participate in studies,” Dr. Nadeau said. “In addition, only 17% of their parents had a college or advanced education and 41% had a household income of less than $25,000 per year.”

The social environment is critical, she continued, because the lifestyle factors associated with type 2 diabetes often result in poor outcomes. “It’s very hard to make lifestyle changes if there is a socioeconomic challenge,” she said. “We can’t make change without understanding the community and culture that these youth live in. It’s also critical that we have participation of minorities and other research participants with diverse backgrounds in order for [clinical] trials to be effective for the population that this disease is affecting.”

Another issue keeping drug trials of youth with type 2 diabetes from being completed is the entry criteria. Some studies require youth to be drug naive and have a hemoglobin A1c greater than 7%. “This is difficult, because many youth that are referred to our diabetes center already come in on metformin, leaving only about 7% of subjects available for this criteria,” Dr. Nadeau explained. Another common study entry criterion is being on metformin and having a hemoglobin A1c of about 7%, “so basically being a metformin failure,” she said. “This is difficult to meet because metformin is relatively effective in the early stages of diabetes.”

“We need clear strategies for research, prevention, and treatment. Clarifying unique pathophysiology, complications, and psychosocial impact will enable industry, academia, funding agencies, advocacy groups, and regulators to collectively evaluate the best approaches to research, treatment, and prevention,” Dr. Nadeau said.

The consensus conference participants recommended the following objectives: clarify the biology of type 2 diabetes in youth, obtain new pediatric information on drugs, encourage the use of appropriate medications, and inform clinical decision-making. “We have a desperate need to understand the actions of drugs in type 2 diabetes youth,” Dr. Nadeau said. “Our current approach is not working. Potential solutions include considering efficacy outcomes besides A1c, potentially looking at improvement in insulin sensitivity, preservation of beta-cell function, trying to prevent the A1c increase instead of looking for an A1c reduction, and trying to extrapolate from effects in adults, if we can understand enough to do that.”

The conference participants also called for infrastructure changes, such as creating a resource for patients with type 2 diabetes in the model of the Type 1 Diabetes Exchange. “We need to have collaborations internationally,” she said. “We also need support for teams and clinical groups to work together to be able to accomplish these collaboratively.”

Dr. Nadeau reported having no financial disclosures.

dbrunk@frontlinemedcom.com