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Tele-neurology visits deemed as effective as office visits for Parkinson’s patients
A new study finds tele-neurology visits are just as effective as in-person visits for patients with Parkinson’s disease, while also saving time and mileage.
Lead author Christopher A. Beck, PhD, of the University of Rochester (N.Y.), and his colleagues studied 195 patients for 1 year who were either provided with their usual medical care or their usual medical care supplemented by four virtual visits via video conferencing from a remote specialist. Investigators evaluated the feasibility of telemedicine visits, as measured by the proportion of patients who completed at least one virtual visit and the proportion of virtual visits completed on time. Efficacy was also evaluated, as measured by the change in the Parkinson’s Disease Questionnaire–39, a quality of life scale. Secondary outcomes included quality of care, caregiver burden, and time and travel savings.
Eligible participants had a clinical diagnosis of Parkinson’s disease, had a private, Internet-enabled device, and lived in a state where a site investigator was licensed to practice. Patients randomized to the telemedicine group received up to four virtual visits over 12 months from a neurologist. Physicians and patients determined the specific content and frequency of each visit, but the format generally included a medical history, a Parkinson’s disease–specific examination (including assessment of tremor and gait), physician recommendations, and time to address patients’ concerns (Neurology. 2017 Aug 16. doi: 10.1212/WNL.0000000000004357).
The study findings showed that the virtual house calls were as effective as were in-person visits, with quality of life no better or worse for patients receiving care at home than for those receiving care in the office. Of telemedicine patients, 98% completed at least one virtual visit, and 91% of all virtual visits scheduled were completed. Researchers also found that participants’ overall quality of care and the burden felt by caregivers was no different whether they had virtual or in-person visits. Each virtual house call saved patients a median of 88 minutes and 38 miles per visit.
Ninety-seven percent of patients and 86% of participating neurologists said they were satisfied with the virtual visits, with 55% of patients stating they preferred virtual visits over in-person visits.
The study – the first national randomized controlled trial of telemedicine to connect remote specialists to patients at home – shows that tele-neurology is a practical and effective model of care for Parkinson patients, said study coauthor E. Ray Dorsey, MD, of the University of Rochester.
“People were very interested in taking part in this study, and the results showed that these virtual house calls were feasible for people with Parkinson’s disease,” Dr. Dorsey said in a statement. “People’s care was as effective as with the in-office visits, and the virtual house calls provided the participants with convenience and comfort.”
Dr. Dorsey noted that 73% of the study participants had visited a Parkinson’s disease specialist in the past year and 83% said they were satisfied with their care, which may have impacted the quality of life finding.
“The fact that adding the virtual house calls to people’s care did not improve their quality of life could be because a large proportion were already seeing a specialist and were satisfied with that care,” he said. “Of course, it’s also possible that virtual house calls are not enough to improve quality of life.”
Limitations of the study included that study participants were primarily well-educated and more familiar with the Internet than was the general population, so results may not be relevant for all people with Parkinson’s disease. In addition, the study population, of whom 96% were white with a mean age of 66 years, did not include people with the disease who live in nursing homes, who account for nearly 25% of all Medicare beneficiaries with Parkinson’s disease.
The study was supported by the Patient-Centered Outcomes Research Institute. Dr. Dorsey serves on the medical advisory board of, and has stock options in, Grand Rounds. No other relevant disclosures relevant were reported by study authors.
[email protected]
On Twitter @legal_med
Patients with Parkinson’s disease (PD) show more improvements in motor symptoms and less frequently receive inappropriate medications when treated by a specialist. However, reduced mobility due to PD, combined with the physical distance to the specialist’s office, poses substantial travel burdens and may limit access to care.
Telemedicine, utilizing video and audio conferencing technology to connect patients and clinicians, can mitigate this problem. However, the Centers for Medicare & Medicaid Services currently only covers telemedicine encounters under specific criteria: The patient lives in a rural area seen in a covered entity by a consulting physician licensed in the state in which the patient resides. Even if the rural designation were removed, requiring travel to a center that offers telemedicine consultation may preclude many patients from using this approach.
The study by Dr. Beck and his colleagues provides a good first step in demonstrating how neurologists can use available technology to reduce patient burdens while still maintaining high-quality neurologic care. If more evidence mounts, and the regulatory environment improves, home-based telemedicine has the potential to revolutionize modern health care for PD and other chronic debilitating diseases.
David Shprecher, DO, is with Banner Sun Health Research Institute in Sun City, Ariz. Jennifer Majersik, MD, is with the University of Utah, Salt Lake City. Dr. Shprecher reported financial relationships with a variety of companies that market or are developing drugs for Parkinson’s disease. Dr. Majersik reported receiving significant research support from the National Institute of Neurological Disorders and Stroke. These comments are adapted from an accompanying editorial (Neurology. 2017 Aug 16. doi: 10.1212/WNL.0000000000004366).
Patients with Parkinson’s disease (PD) show more improvements in motor symptoms and less frequently receive inappropriate medications when treated by a specialist. However, reduced mobility due to PD, combined with the physical distance to the specialist’s office, poses substantial travel burdens and may limit access to care.
Telemedicine, utilizing video and audio conferencing technology to connect patients and clinicians, can mitigate this problem. However, the Centers for Medicare & Medicaid Services currently only covers telemedicine encounters under specific criteria: The patient lives in a rural area seen in a covered entity by a consulting physician licensed in the state in which the patient resides. Even if the rural designation were removed, requiring travel to a center that offers telemedicine consultation may preclude many patients from using this approach.
The study by Dr. Beck and his colleagues provides a good first step in demonstrating how neurologists can use available technology to reduce patient burdens while still maintaining high-quality neurologic care. If more evidence mounts, and the regulatory environment improves, home-based telemedicine has the potential to revolutionize modern health care for PD and other chronic debilitating diseases.
David Shprecher, DO, is with Banner Sun Health Research Institute in Sun City, Ariz. Jennifer Majersik, MD, is with the University of Utah, Salt Lake City. Dr. Shprecher reported financial relationships with a variety of companies that market or are developing drugs for Parkinson’s disease. Dr. Majersik reported receiving significant research support from the National Institute of Neurological Disorders and Stroke. These comments are adapted from an accompanying editorial (Neurology. 2017 Aug 16. doi: 10.1212/WNL.0000000000004366).
Patients with Parkinson’s disease (PD) show more improvements in motor symptoms and less frequently receive inappropriate medications when treated by a specialist. However, reduced mobility due to PD, combined with the physical distance to the specialist’s office, poses substantial travel burdens and may limit access to care.
Telemedicine, utilizing video and audio conferencing technology to connect patients and clinicians, can mitigate this problem. However, the Centers for Medicare & Medicaid Services currently only covers telemedicine encounters under specific criteria: The patient lives in a rural area seen in a covered entity by a consulting physician licensed in the state in which the patient resides. Even if the rural designation were removed, requiring travel to a center that offers telemedicine consultation may preclude many patients from using this approach.
The study by Dr. Beck and his colleagues provides a good first step in demonstrating how neurologists can use available technology to reduce patient burdens while still maintaining high-quality neurologic care. If more evidence mounts, and the regulatory environment improves, home-based telemedicine has the potential to revolutionize modern health care for PD and other chronic debilitating diseases.
David Shprecher, DO, is with Banner Sun Health Research Institute in Sun City, Ariz. Jennifer Majersik, MD, is with the University of Utah, Salt Lake City. Dr. Shprecher reported financial relationships with a variety of companies that market or are developing drugs for Parkinson’s disease. Dr. Majersik reported receiving significant research support from the National Institute of Neurological Disorders and Stroke. These comments are adapted from an accompanying editorial (Neurology. 2017 Aug 16. doi: 10.1212/WNL.0000000000004366).
A new study finds tele-neurology visits are just as effective as in-person visits for patients with Parkinson’s disease, while also saving time and mileage.
Lead author Christopher A. Beck, PhD, of the University of Rochester (N.Y.), and his colleagues studied 195 patients for 1 year who were either provided with their usual medical care or their usual medical care supplemented by four virtual visits via video conferencing from a remote specialist. Investigators evaluated the feasibility of telemedicine visits, as measured by the proportion of patients who completed at least one virtual visit and the proportion of virtual visits completed on time. Efficacy was also evaluated, as measured by the change in the Parkinson’s Disease Questionnaire–39, a quality of life scale. Secondary outcomes included quality of care, caregiver burden, and time and travel savings.
Eligible participants had a clinical diagnosis of Parkinson’s disease, had a private, Internet-enabled device, and lived in a state where a site investigator was licensed to practice. Patients randomized to the telemedicine group received up to four virtual visits over 12 months from a neurologist. Physicians and patients determined the specific content and frequency of each visit, but the format generally included a medical history, a Parkinson’s disease–specific examination (including assessment of tremor and gait), physician recommendations, and time to address patients’ concerns (Neurology. 2017 Aug 16. doi: 10.1212/WNL.0000000000004357).
The study findings showed that the virtual house calls were as effective as were in-person visits, with quality of life no better or worse for patients receiving care at home than for those receiving care in the office. Of telemedicine patients, 98% completed at least one virtual visit, and 91% of all virtual visits scheduled were completed. Researchers also found that participants’ overall quality of care and the burden felt by caregivers was no different whether they had virtual or in-person visits. Each virtual house call saved patients a median of 88 minutes and 38 miles per visit.
Ninety-seven percent of patients and 86% of participating neurologists said they were satisfied with the virtual visits, with 55% of patients stating they preferred virtual visits over in-person visits.
The study – the first national randomized controlled trial of telemedicine to connect remote specialists to patients at home – shows that tele-neurology is a practical and effective model of care for Parkinson patients, said study coauthor E. Ray Dorsey, MD, of the University of Rochester.
“People were very interested in taking part in this study, and the results showed that these virtual house calls were feasible for people with Parkinson’s disease,” Dr. Dorsey said in a statement. “People’s care was as effective as with the in-office visits, and the virtual house calls provided the participants with convenience and comfort.”
Dr. Dorsey noted that 73% of the study participants had visited a Parkinson’s disease specialist in the past year and 83% said they were satisfied with their care, which may have impacted the quality of life finding.
“The fact that adding the virtual house calls to people’s care did not improve their quality of life could be because a large proportion were already seeing a specialist and were satisfied with that care,” he said. “Of course, it’s also possible that virtual house calls are not enough to improve quality of life.”
Limitations of the study included that study participants were primarily well-educated and more familiar with the Internet than was the general population, so results may not be relevant for all people with Parkinson’s disease. In addition, the study population, of whom 96% were white with a mean age of 66 years, did not include people with the disease who live in nursing homes, who account for nearly 25% of all Medicare beneficiaries with Parkinson’s disease.
The study was supported by the Patient-Centered Outcomes Research Institute. Dr. Dorsey serves on the medical advisory board of, and has stock options in, Grand Rounds. No other relevant disclosures relevant were reported by study authors.
[email protected]
On Twitter @legal_med
A new study finds tele-neurology visits are just as effective as in-person visits for patients with Parkinson’s disease, while also saving time and mileage.
Lead author Christopher A. Beck, PhD, of the University of Rochester (N.Y.), and his colleagues studied 195 patients for 1 year who were either provided with their usual medical care or their usual medical care supplemented by four virtual visits via video conferencing from a remote specialist. Investigators evaluated the feasibility of telemedicine visits, as measured by the proportion of patients who completed at least one virtual visit and the proportion of virtual visits completed on time. Efficacy was also evaluated, as measured by the change in the Parkinson’s Disease Questionnaire–39, a quality of life scale. Secondary outcomes included quality of care, caregiver burden, and time and travel savings.
Eligible participants had a clinical diagnosis of Parkinson’s disease, had a private, Internet-enabled device, and lived in a state where a site investigator was licensed to practice. Patients randomized to the telemedicine group received up to four virtual visits over 12 months from a neurologist. Physicians and patients determined the specific content and frequency of each visit, but the format generally included a medical history, a Parkinson’s disease–specific examination (including assessment of tremor and gait), physician recommendations, and time to address patients’ concerns (Neurology. 2017 Aug 16. doi: 10.1212/WNL.0000000000004357).
The study findings showed that the virtual house calls were as effective as were in-person visits, with quality of life no better or worse for patients receiving care at home than for those receiving care in the office. Of telemedicine patients, 98% completed at least one virtual visit, and 91% of all virtual visits scheduled were completed. Researchers also found that participants’ overall quality of care and the burden felt by caregivers was no different whether they had virtual or in-person visits. Each virtual house call saved patients a median of 88 minutes and 38 miles per visit.
Ninety-seven percent of patients and 86% of participating neurologists said they were satisfied with the virtual visits, with 55% of patients stating they preferred virtual visits over in-person visits.
The study – the first national randomized controlled trial of telemedicine to connect remote specialists to patients at home – shows that tele-neurology is a practical and effective model of care for Parkinson patients, said study coauthor E. Ray Dorsey, MD, of the University of Rochester.
“People were very interested in taking part in this study, and the results showed that these virtual house calls were feasible for people with Parkinson’s disease,” Dr. Dorsey said in a statement. “People’s care was as effective as with the in-office visits, and the virtual house calls provided the participants with convenience and comfort.”
Dr. Dorsey noted that 73% of the study participants had visited a Parkinson’s disease specialist in the past year and 83% said they were satisfied with their care, which may have impacted the quality of life finding.
“The fact that adding the virtual house calls to people’s care did not improve their quality of life could be because a large proportion were already seeing a specialist and were satisfied with that care,” he said. “Of course, it’s also possible that virtual house calls are not enough to improve quality of life.”
Limitations of the study included that study participants were primarily well-educated and more familiar with the Internet than was the general population, so results may not be relevant for all people with Parkinson’s disease. In addition, the study population, of whom 96% were white with a mean age of 66 years, did not include people with the disease who live in nursing homes, who account for nearly 25% of all Medicare beneficiaries with Parkinson’s disease.
The study was supported by the Patient-Centered Outcomes Research Institute. Dr. Dorsey serves on the medical advisory board of, and has stock options in, Grand Rounds. No other relevant disclosures relevant were reported by study authors.
[email protected]
On Twitter @legal_med
FROM NEUROLOGY
Key clinical point:
Major finding: Overall, 98% of tele-neurology patients completed at least one virtual visit, and 91% of all virtual visits scheduled were completed.
Data source: National randomized controlled trial of 195 patients with Parkinson’s disease.
Disclosures: E. Ray Dorsey, MD, serves on the medical advisory board of and has stock options in Grand Rounds. No other relevant disclosures were reported by the researchers. The study was supported by the Patient-Centered Outcomes Research Institute.
Identifying Gene Variants Linked to Epilepsy
Mutations in SCN1A, SCN2A, and SCN8A, which code for neuronal voltage-gated sodium channel alpha-subunits, have been associated with certain early onset epilepsy syndromes. Despite this association, many clinicians are uncertain about the value of missense genetic variants in the management of epilepsy because many mutations are classified as having unknown significance. A recent database analysis identified gene variants that are pathogenic and benign, giving clinicians a better understanding of how to interpret SCN test results. Details of the investigation include the following:
- Investigators used 8 algorithms to evaluate the pathogenicity of various genetic variants. They also used logistic regression to help determine if combining algorithms might improve their ability to predict pathogenicity.
- 440 variants were considered pathogenic or likely pathogenic.
- Most computer algorithms that attempt to determine the value of SCN test results are very sensitive but also suffer from low specificity.
- The Mendelian Clinically Applicable Pathogenicity algorithm proved most valuable, with an accuracy of 0.90.
Holland KD, Bouley TM, Horn PS. Comparison and optimization of in silico algorithms for predicting the pathogenicity of sodium channel variants in epilepsy. Epilepsia. 2017;58(7):1190-1198.
Mutations in SCN1A, SCN2A, and SCN8A, which code for neuronal voltage-gated sodium channel alpha-subunits, have been associated with certain early onset epilepsy syndromes. Despite this association, many clinicians are uncertain about the value of missense genetic variants in the management of epilepsy because many mutations are classified as having unknown significance. A recent database analysis identified gene variants that are pathogenic and benign, giving clinicians a better understanding of how to interpret SCN test results. Details of the investigation include the following:
- Investigators used 8 algorithms to evaluate the pathogenicity of various genetic variants. They also used logistic regression to help determine if combining algorithms might improve their ability to predict pathogenicity.
- 440 variants were considered pathogenic or likely pathogenic.
- Most computer algorithms that attempt to determine the value of SCN test results are very sensitive but also suffer from low specificity.
- The Mendelian Clinically Applicable Pathogenicity algorithm proved most valuable, with an accuracy of 0.90.
Holland KD, Bouley TM, Horn PS. Comparison and optimization of in silico algorithms for predicting the pathogenicity of sodium channel variants in epilepsy. Epilepsia. 2017;58(7):1190-1198.
Mutations in SCN1A, SCN2A, and SCN8A, which code for neuronal voltage-gated sodium channel alpha-subunits, have been associated with certain early onset epilepsy syndromes. Despite this association, many clinicians are uncertain about the value of missense genetic variants in the management of epilepsy because many mutations are classified as having unknown significance. A recent database analysis identified gene variants that are pathogenic and benign, giving clinicians a better understanding of how to interpret SCN test results. Details of the investigation include the following:
- Investigators used 8 algorithms to evaluate the pathogenicity of various genetic variants. They also used logistic regression to help determine if combining algorithms might improve their ability to predict pathogenicity.
- 440 variants were considered pathogenic or likely pathogenic.
- Most computer algorithms that attempt to determine the value of SCN test results are very sensitive but also suffer from low specificity.
- The Mendelian Clinically Applicable Pathogenicity algorithm proved most valuable, with an accuracy of 0.90.
Holland KD, Bouley TM, Horn PS. Comparison and optimization of in silico algorithms for predicting the pathogenicity of sodium channel variants in epilepsy. Epilepsia. 2017;58(7):1190-1198.
Siblings Respond Similarly to the Same AED
Siblings tend to have a similar response to antiepileptic drugs (AEDs) suggests a study that compared drug response in sibling pairs. Details of the investigation include the following:
- Investigators collected records from a single-center database that included patients with a diagnosis of epilepsy in which their last names, addresses, and parents’ names were matched to determine the existence of siblings with the same disease.
- Twenty-eight sibling pairs were identified, along with 2 sibling trios with epilepsy.
- Seventeen of the sibling pairs had been taking the same initial AED, while 15 pairs had the same type of epilepsy.
- When at least one sibling in a pair improved on an initial AED, the other sibling was more likely to respond if they were taking the same AED compared with those who were taking a different AED.
- While a positive response to an AED predicted success in a sibling taking the same drug, investigators pointed out that their study was retrospective and involved a small sample, which is why they recommended larger prospective trials.
Ueda K, Serajee F, Rajilich J, Taraman S, Steckling L, Huq AM. Sibling response to initial antiepileptic medication predicts treatment success. Epilepsy Res. 2017;136:84-87.
Siblings tend to have a similar response to antiepileptic drugs (AEDs) suggests a study that compared drug response in sibling pairs. Details of the investigation include the following:
- Investigators collected records from a single-center database that included patients with a diagnosis of epilepsy in which their last names, addresses, and parents’ names were matched to determine the existence of siblings with the same disease.
- Twenty-eight sibling pairs were identified, along with 2 sibling trios with epilepsy.
- Seventeen of the sibling pairs had been taking the same initial AED, while 15 pairs had the same type of epilepsy.
- When at least one sibling in a pair improved on an initial AED, the other sibling was more likely to respond if they were taking the same AED compared with those who were taking a different AED.
- While a positive response to an AED predicted success in a sibling taking the same drug, investigators pointed out that their study was retrospective and involved a small sample, which is why they recommended larger prospective trials.
Ueda K, Serajee F, Rajilich J, Taraman S, Steckling L, Huq AM. Sibling response to initial antiepileptic medication predicts treatment success. Epilepsy Res. 2017;136:84-87.
Siblings tend to have a similar response to antiepileptic drugs (AEDs) suggests a study that compared drug response in sibling pairs. Details of the investigation include the following:
- Investigators collected records from a single-center database that included patients with a diagnosis of epilepsy in which their last names, addresses, and parents’ names were matched to determine the existence of siblings with the same disease.
- Twenty-eight sibling pairs were identified, along with 2 sibling trios with epilepsy.
- Seventeen of the sibling pairs had been taking the same initial AED, while 15 pairs had the same type of epilepsy.
- When at least one sibling in a pair improved on an initial AED, the other sibling was more likely to respond if they were taking the same AED compared with those who were taking a different AED.
- While a positive response to an AED predicted success in a sibling taking the same drug, investigators pointed out that their study was retrospective and involved a small sample, which is why they recommended larger prospective trials.
Ueda K, Serajee F, Rajilich J, Taraman S, Steckling L, Huq AM. Sibling response to initial antiepileptic medication predicts treatment success. Epilepsy Res. 2017;136:84-87.
MRI-detected Neuronal Connectivity Predicts Surgical Outcomes
Identifying a seizure propagation network in patients who have undergone surgery for unilateral temporal lobe epilepsy (TLE) may help determine which patients are most likely to remain seizure free over time, according to an analysis of patients who underwent magnetic resonance imaging (MRI) scans. Details of the investigation include the following:
- MRI of 22 unilateral TLE patients found functional connectivity that encompassed the ipsilateral (to seizure focus) and contralateral hippocampus, thalamus, and insula. 35 healthy individuals were included in the study to act as controls.
- Resting state functional and diffusion-weighted 3T magnetic resonance imaging was used in the study.
- The investigators discovered a consistent connectivity pattern representing the network expected in patients who remained seizure free. This was found in 8 patients who did not have seizures one year after surgery.
- Using this model, they were able to differentiate patients with unfavorable outcomes from those with long-term freedom from seizures.
This study provides evidence that network connectivity could be a clinical tool for epilepsy surgery outcome prediction.
Morgan VL, Englot DJ, Rogers BP, et al. Magnetic resonance imaging connectivity for the prediction of seizure outcome in temporal lobe epilepsy. Epilepsia. 2017;58(7):1251-1260.
Identifying a seizure propagation network in patients who have undergone surgery for unilateral temporal lobe epilepsy (TLE) may help determine which patients are most likely to remain seizure free over time, according to an analysis of patients who underwent magnetic resonance imaging (MRI) scans. Details of the investigation include the following:
- MRI of 22 unilateral TLE patients found functional connectivity that encompassed the ipsilateral (to seizure focus) and contralateral hippocampus, thalamus, and insula. 35 healthy individuals were included in the study to act as controls.
- Resting state functional and diffusion-weighted 3T magnetic resonance imaging was used in the study.
- The investigators discovered a consistent connectivity pattern representing the network expected in patients who remained seizure free. This was found in 8 patients who did not have seizures one year after surgery.
- Using this model, they were able to differentiate patients with unfavorable outcomes from those with long-term freedom from seizures.
This study provides evidence that network connectivity could be a clinical tool for epilepsy surgery outcome prediction.
Morgan VL, Englot DJ, Rogers BP, et al. Magnetic resonance imaging connectivity for the prediction of seizure outcome in temporal lobe epilepsy. Epilepsia. 2017;58(7):1251-1260.
Identifying a seizure propagation network in patients who have undergone surgery for unilateral temporal lobe epilepsy (TLE) may help determine which patients are most likely to remain seizure free over time, according to an analysis of patients who underwent magnetic resonance imaging (MRI) scans. Details of the investigation include the following:
- MRI of 22 unilateral TLE patients found functional connectivity that encompassed the ipsilateral (to seizure focus) and contralateral hippocampus, thalamus, and insula. 35 healthy individuals were included in the study to act as controls.
- Resting state functional and diffusion-weighted 3T magnetic resonance imaging was used in the study.
- The investigators discovered a consistent connectivity pattern representing the network expected in patients who remained seizure free. This was found in 8 patients who did not have seizures one year after surgery.
- Using this model, they were able to differentiate patients with unfavorable outcomes from those with long-term freedom from seizures.
This study provides evidence that network connectivity could be a clinical tool for epilepsy surgery outcome prediction.
Morgan VL, Englot DJ, Rogers BP, et al. Magnetic resonance imaging connectivity for the prediction of seizure outcome in temporal lobe epilepsy. Epilepsia. 2017;58(7):1251-1260.
AMA’s stance on choline, prenatal vitamins could bring ‘staggering’ results
For quite some time now, I’ve been urging my colleagues to follow the science on the powerful impact of choline on the brain.
In May 2017, based on studies using genetically altered mice that show the developmental changes of Down syndrome and Alzheimer’s disease at 6 months, I raised the question of whether prenatal choline could lead to the prevention of Alzheimer’s.
Thanks to the leadership of Niva Lubin-Johnson, MD, now president-elect of the National Medical Association, while a member and immediate past chair of the American Medical Association’s minority affairs section governing council*, the AMA ’s delegates passed a resolution to support an increase in choline in prenatal vitamins.
If the prenatal vitamin companies take the AMA’s resolution to heart and put more choline in their prenatal vitamins or if physicians in the United States pay attention to the AMA’s action and recommend pregnant women ensure they get adequate choline in their diets, the benefit to Americans’ public health could be staggering. Currently, it is known that choline deficiency – usually brought about by fetal alcohol exposure – is a public health problem, and choline deficiency is the leading preventable cause of intellectual disability. Public health efforts aimed at preventing intellectual disabilities from fetal alcohol exposure are designed to warn women about the risks of drinking during pregnancy; while this effort is commendable, it does not solve a very common problem – namely, women’s engaging in social drinking before they realize they are pregnant. (Psychiatric Serv. 2015 66[5]:539-42).
The late Julius B. Richmond, MD, former director of the Institute for Juvenile Research, surgeon general under former President Jimmy Carter, and one of the founders of Head Start under former President Lyndon B. Johnson, used to say that, in order to institutionalize a public policy, you need a solid scientific basis for the policy, a mechanism to actualize the policy, and the “political will” to do so. The AMA’s recommendation has the Institute of Medicine’s science behind it, so putting choline in prenatal vitamins or having physicians recommend that pregnant women get adequate doses of choline should be pretty easy to actualize. The political will to do this extremely important, biotechnical preventive intervention should be a no-brainer.
Should this AMA recommendation gain the traction it deserves, the American people might see a substantial decrease in the prevalence of premature and low-birth-weight infants, intellectual disability, ADHD, speech and language difficulties, epilepsy, heart defects, schizophrenia, Alzheimer’s disease, depression, school failure, juvenile delinquency, violence, and suicide – all of which seem to be tied to choline deficiency.
*This story was updated August 17, 2017.
For quite some time now, I’ve been urging my colleagues to follow the science on the powerful impact of choline on the brain.
In May 2017, based on studies using genetically altered mice that show the developmental changes of Down syndrome and Alzheimer’s disease at 6 months, I raised the question of whether prenatal choline could lead to the prevention of Alzheimer’s.
Thanks to the leadership of Niva Lubin-Johnson, MD, now president-elect of the National Medical Association, while a member and immediate past chair of the American Medical Association’s minority affairs section governing council*, the AMA ’s delegates passed a resolution to support an increase in choline in prenatal vitamins.
If the prenatal vitamin companies take the AMA’s resolution to heart and put more choline in their prenatal vitamins or if physicians in the United States pay attention to the AMA’s action and recommend pregnant women ensure they get adequate choline in their diets, the benefit to Americans’ public health could be staggering. Currently, it is known that choline deficiency – usually brought about by fetal alcohol exposure – is a public health problem, and choline deficiency is the leading preventable cause of intellectual disability. Public health efforts aimed at preventing intellectual disabilities from fetal alcohol exposure are designed to warn women about the risks of drinking during pregnancy; while this effort is commendable, it does not solve a very common problem – namely, women’s engaging in social drinking before they realize they are pregnant. (Psychiatric Serv. 2015 66[5]:539-42).
The late Julius B. Richmond, MD, former director of the Institute for Juvenile Research, surgeon general under former President Jimmy Carter, and one of the founders of Head Start under former President Lyndon B. Johnson, used to say that, in order to institutionalize a public policy, you need a solid scientific basis for the policy, a mechanism to actualize the policy, and the “political will” to do so. The AMA’s recommendation has the Institute of Medicine’s science behind it, so putting choline in prenatal vitamins or having physicians recommend that pregnant women get adequate doses of choline should be pretty easy to actualize. The political will to do this extremely important, biotechnical preventive intervention should be a no-brainer.
Should this AMA recommendation gain the traction it deserves, the American people might see a substantial decrease in the prevalence of premature and low-birth-weight infants, intellectual disability, ADHD, speech and language difficulties, epilepsy, heart defects, schizophrenia, Alzheimer’s disease, depression, school failure, juvenile delinquency, violence, and suicide – all of which seem to be tied to choline deficiency.
*This story was updated August 17, 2017.
For quite some time now, I’ve been urging my colleagues to follow the science on the powerful impact of choline on the brain.
In May 2017, based on studies using genetically altered mice that show the developmental changes of Down syndrome and Alzheimer’s disease at 6 months, I raised the question of whether prenatal choline could lead to the prevention of Alzheimer’s.
Thanks to the leadership of Niva Lubin-Johnson, MD, now president-elect of the National Medical Association, while a member and immediate past chair of the American Medical Association’s minority affairs section governing council*, the AMA ’s delegates passed a resolution to support an increase in choline in prenatal vitamins.
If the prenatal vitamin companies take the AMA’s resolution to heart and put more choline in their prenatal vitamins or if physicians in the United States pay attention to the AMA’s action and recommend pregnant women ensure they get adequate choline in their diets, the benefit to Americans’ public health could be staggering. Currently, it is known that choline deficiency – usually brought about by fetal alcohol exposure – is a public health problem, and choline deficiency is the leading preventable cause of intellectual disability. Public health efforts aimed at preventing intellectual disabilities from fetal alcohol exposure are designed to warn women about the risks of drinking during pregnancy; while this effort is commendable, it does not solve a very common problem – namely, women’s engaging in social drinking before they realize they are pregnant. (Psychiatric Serv. 2015 66[5]:539-42).
The late Julius B. Richmond, MD, former director of the Institute for Juvenile Research, surgeon general under former President Jimmy Carter, and one of the founders of Head Start under former President Lyndon B. Johnson, used to say that, in order to institutionalize a public policy, you need a solid scientific basis for the policy, a mechanism to actualize the policy, and the “political will” to do so. The AMA’s recommendation has the Institute of Medicine’s science behind it, so putting choline in prenatal vitamins or having physicians recommend that pregnant women get adequate doses of choline should be pretty easy to actualize. The political will to do this extremely important, biotechnical preventive intervention should be a no-brainer.
Should this AMA recommendation gain the traction it deserves, the American people might see a substantial decrease in the prevalence of premature and low-birth-weight infants, intellectual disability, ADHD, speech and language difficulties, epilepsy, heart defects, schizophrenia, Alzheimer’s disease, depression, school failure, juvenile delinquency, violence, and suicide – all of which seem to be tied to choline deficiency.
*This story was updated August 17, 2017.
Safety alert for intragastric balloon systems
The Food and Drug Administration announced a safety alert on Aug. 10, 2017, for liquid-filled intragastric balloon systems, as they have caused five reports of unanticipated deaths that occurred from 2016 to present in patients.
The cause or incidence of patient death is still unknown, and the FDA has not been able to definitively attribute the deaths to the devices or the insertion procedures for these devices. All five reports show that patient deaths occurred within a month or less of balloon placement. In three of the reports, death occurred as soon as 1-3 days after balloon placement. The FDA has also received two additional reports of deaths in the same time period related to potential complications associated with balloon treatment.
The FDA continues to recommend that health care providers closely monitor patients treated with these devices for complications. Any adverse events related to intragastric balloon systems should be reported through MedWatch. The FDA will keep the public informed as new information becomes available.
Read the full safety alert on the FDA’s website.
As past chair of the AGA Center for GI Innovation and Technology, I have been closely following balloon-based obesity devices as they’ve entered the marketplace. The center has welcomed the introduction of these noninvasive devices that can be managed by GIs, and we’ve worked closely with device companies and the FDA for the past several years to ensure these devices were introduced to the market in a safe and efficient manner.
We do not have enough information now to connect these recent patient deaths to these devices. That said, the FDA’s letter reinforces a few important points. Foremost, the fact that complications and adverse events can occur with any procedure. For physicians using intragastric balloons, each patient must be appropriately evaluated prior to the decision to place the balloon, especially for the potential risks of anesthesia and an endoscopic procedure. Patients must be monitored closely during the entire term of treatment, and following the procedure, in order to detect the development of possible complications, and each patient should be instructed to contact his or her physician immediately upon the onset of any unexpected symptoms.
Michael Kochman MD, AGAF, is the Wilmott Family Professor of Medicine, professor of medicine in surgery, gastroenterology division, University of Pennsylvania, Philadelphia.
As past chair of the AGA Center for GI Innovation and Technology, I have been closely following balloon-based obesity devices as they’ve entered the marketplace. The center has welcomed the introduction of these noninvasive devices that can be managed by GIs, and we’ve worked closely with device companies and the FDA for the past several years to ensure these devices were introduced to the market in a safe and efficient manner.
We do not have enough information now to connect these recent patient deaths to these devices. That said, the FDA’s letter reinforces a few important points. Foremost, the fact that complications and adverse events can occur with any procedure. For physicians using intragastric balloons, each patient must be appropriately evaluated prior to the decision to place the balloon, especially for the potential risks of anesthesia and an endoscopic procedure. Patients must be monitored closely during the entire term of treatment, and following the procedure, in order to detect the development of possible complications, and each patient should be instructed to contact his or her physician immediately upon the onset of any unexpected symptoms.
Michael Kochman MD, AGAF, is the Wilmott Family Professor of Medicine, professor of medicine in surgery, gastroenterology division, University of Pennsylvania, Philadelphia.
As past chair of the AGA Center for GI Innovation and Technology, I have been closely following balloon-based obesity devices as they’ve entered the marketplace. The center has welcomed the introduction of these noninvasive devices that can be managed by GIs, and we’ve worked closely with device companies and the FDA for the past several years to ensure these devices were introduced to the market in a safe and efficient manner.
We do not have enough information now to connect these recent patient deaths to these devices. That said, the FDA’s letter reinforces a few important points. Foremost, the fact that complications and adverse events can occur with any procedure. For physicians using intragastric balloons, each patient must be appropriately evaluated prior to the decision to place the balloon, especially for the potential risks of anesthesia and an endoscopic procedure. Patients must be monitored closely during the entire term of treatment, and following the procedure, in order to detect the development of possible complications, and each patient should be instructed to contact his or her physician immediately upon the onset of any unexpected symptoms.
Michael Kochman MD, AGAF, is the Wilmott Family Professor of Medicine, professor of medicine in surgery, gastroenterology division, University of Pennsylvania, Philadelphia.
The Food and Drug Administration announced a safety alert on Aug. 10, 2017, for liquid-filled intragastric balloon systems, as they have caused five reports of unanticipated deaths that occurred from 2016 to present in patients.
The cause or incidence of patient death is still unknown, and the FDA has not been able to definitively attribute the deaths to the devices or the insertion procedures for these devices. All five reports show that patient deaths occurred within a month or less of balloon placement. In three of the reports, death occurred as soon as 1-3 days after balloon placement. The FDA has also received two additional reports of deaths in the same time period related to potential complications associated with balloon treatment.
The FDA continues to recommend that health care providers closely monitor patients treated with these devices for complications. Any adverse events related to intragastric balloon systems should be reported through MedWatch. The FDA will keep the public informed as new information becomes available.
Read the full safety alert on the FDA’s website.
The Food and Drug Administration announced a safety alert on Aug. 10, 2017, for liquid-filled intragastric balloon systems, as they have caused five reports of unanticipated deaths that occurred from 2016 to present in patients.
The cause or incidence of patient death is still unknown, and the FDA has not been able to definitively attribute the deaths to the devices or the insertion procedures for these devices. All five reports show that patient deaths occurred within a month or less of balloon placement. In three of the reports, death occurred as soon as 1-3 days after balloon placement. The FDA has also received two additional reports of deaths in the same time period related to potential complications associated with balloon treatment.
The FDA continues to recommend that health care providers closely monitor patients treated with these devices for complications. Any adverse events related to intragastric balloon systems should be reported through MedWatch. The FDA will keep the public informed as new information becomes available.
Read the full safety alert on the FDA’s website.
Helpful schedules ease task of tapering opioids
ESTES PARK, COLO. – Now that the opioid epidemic has formally been declared a national emergency, physicians can expect to encounter growing pressure to taper opioids in their chronic pain patients, Sunny Linnebur, PharmD, predicted at a conference on internal medicine sponsored by the University of Colorado.
As an example of what physicians around the country might expect, she added, Colorado state health officials recently announced that coverage of opioid therapy for Medicaid patients will be reduced. State health officials recommended that physicians taper down their patients’ opioids.
Fortunately, helpful tools for doing so are just a few mouse clicks away, according to Dr. Linnebur, professor of clinical pharmacy at the University of Colorado, Aurora.
Indications for opioid tapering as described in a guide provided by the Centers for Disease Control and Prevention include lack of a sustained or clinically meaningful improvement in pain and functioning as defined, for example, by at least a 30% improvement on the three-item PEG scale; use of opioids at a daily dosage of 50 morphine equivalent doses or more without evidence of benefit; signs of a substance use disorder other than tobacco dependence; warning signs of harms, such as drowsiness, slurred speech, or difficulty controlling use of the medication; patient request; and any situation where the physician deems that the benefits no longer outweigh the risks (www.cdc.gov/drugoverdose/pdf/clinical_pocket_guide_tapering-a.pdf).
General principles of tapering opioids as outlined by the CDC include reducing the dosage by about 10% a week at a time – although if a patient has been on opioids for years, then at a slower rate, perhaps 10% per month, may be more appropriate. If a patient has been using a 12.5 mcg/hour fentanyl patch, a switch to an oral opioid is recommended to complete the taper. When the smallest dosage has been reached, the interval between doses can be stretched; and once the medication is being taken less than once per day, it can be stopped.
She highlighted an opioid tapering schedule form developed by experts at the Washington State Health Care Authority as being particularly useful.
“If you type in a patient’s opioid medication and dose, it will give you a week-to-week calendar schedule for tapering,” she explained. “We know that getting patients on the safest dose of opioid is important, but it’s also difficult. This is an objective taper schedule that will prevent the patient from withdrawing from their opioid and hopefully will help in tolerating the reduction.”
Dr. Linnebur reported having no financial conflicts of interest regarding her presentation.
ESTES PARK, COLO. – Now that the opioid epidemic has formally been declared a national emergency, physicians can expect to encounter growing pressure to taper opioids in their chronic pain patients, Sunny Linnebur, PharmD, predicted at a conference on internal medicine sponsored by the University of Colorado.
As an example of what physicians around the country might expect, she added, Colorado state health officials recently announced that coverage of opioid therapy for Medicaid patients will be reduced. State health officials recommended that physicians taper down their patients’ opioids.
Fortunately, helpful tools for doing so are just a few mouse clicks away, according to Dr. Linnebur, professor of clinical pharmacy at the University of Colorado, Aurora.
Indications for opioid tapering as described in a guide provided by the Centers for Disease Control and Prevention include lack of a sustained or clinically meaningful improvement in pain and functioning as defined, for example, by at least a 30% improvement on the three-item PEG scale; use of opioids at a daily dosage of 50 morphine equivalent doses or more without evidence of benefit; signs of a substance use disorder other than tobacco dependence; warning signs of harms, such as drowsiness, slurred speech, or difficulty controlling use of the medication; patient request; and any situation where the physician deems that the benefits no longer outweigh the risks (www.cdc.gov/drugoverdose/pdf/clinical_pocket_guide_tapering-a.pdf).
General principles of tapering opioids as outlined by the CDC include reducing the dosage by about 10% a week at a time – although if a patient has been on opioids for years, then at a slower rate, perhaps 10% per month, may be more appropriate. If a patient has been using a 12.5 mcg/hour fentanyl patch, a switch to an oral opioid is recommended to complete the taper. When the smallest dosage has been reached, the interval between doses can be stretched; and once the medication is being taken less than once per day, it can be stopped.
She highlighted an opioid tapering schedule form developed by experts at the Washington State Health Care Authority as being particularly useful.
“If you type in a patient’s opioid medication and dose, it will give you a week-to-week calendar schedule for tapering,” she explained. “We know that getting patients on the safest dose of opioid is important, but it’s also difficult. This is an objective taper schedule that will prevent the patient from withdrawing from their opioid and hopefully will help in tolerating the reduction.”
Dr. Linnebur reported having no financial conflicts of interest regarding her presentation.
ESTES PARK, COLO. – Now that the opioid epidemic has formally been declared a national emergency, physicians can expect to encounter growing pressure to taper opioids in their chronic pain patients, Sunny Linnebur, PharmD, predicted at a conference on internal medicine sponsored by the University of Colorado.
As an example of what physicians around the country might expect, she added, Colorado state health officials recently announced that coverage of opioid therapy for Medicaid patients will be reduced. State health officials recommended that physicians taper down their patients’ opioids.
Fortunately, helpful tools for doing so are just a few mouse clicks away, according to Dr. Linnebur, professor of clinical pharmacy at the University of Colorado, Aurora.
Indications for opioid tapering as described in a guide provided by the Centers for Disease Control and Prevention include lack of a sustained or clinically meaningful improvement in pain and functioning as defined, for example, by at least a 30% improvement on the three-item PEG scale; use of opioids at a daily dosage of 50 morphine equivalent doses or more without evidence of benefit; signs of a substance use disorder other than tobacco dependence; warning signs of harms, such as drowsiness, slurred speech, or difficulty controlling use of the medication; patient request; and any situation where the physician deems that the benefits no longer outweigh the risks (www.cdc.gov/drugoverdose/pdf/clinical_pocket_guide_tapering-a.pdf).
General principles of tapering opioids as outlined by the CDC include reducing the dosage by about 10% a week at a time – although if a patient has been on opioids for years, then at a slower rate, perhaps 10% per month, may be more appropriate. If a patient has been using a 12.5 mcg/hour fentanyl patch, a switch to an oral opioid is recommended to complete the taper. When the smallest dosage has been reached, the interval between doses can be stretched; and once the medication is being taken less than once per day, it can be stopped.
She highlighted an opioid tapering schedule form developed by experts at the Washington State Health Care Authority as being particularly useful.
“If you type in a patient’s opioid medication and dose, it will give you a week-to-week calendar schedule for tapering,” she explained. “We know that getting patients on the safest dose of opioid is important, but it’s also difficult. This is an objective taper schedule that will prevent the patient from withdrawing from their opioid and hopefully will help in tolerating the reduction.”
Dr. Linnebur reported having no financial conflicts of interest regarding her presentation.
EXPERT ANALYSIS FROM THE ANNUAL INTERNAL MEDICINE PROGRAM
FDA advisory committee to consider adjuvant sunitinib for RCC
The Oncologic Drugs Advisory Committee to the Food and Drug Administration will meet on Sept. 19 to discuss a supplemental new drug application for sunitinib (Sutent), for the adjuvant treatment of adult patients at high risk of recurrent renal cell carcinoma (RCC) following nephrectomy.
Sunitinib is an oral antiangiogenic agent that has been approved for the treatment of advanced RCC since 2006.
The FDA accepted the new drug application in May and is expected to issue a decision by January 2018.
Results for sunitinib as adjuvant treatment have been mixed. No significant differences in disease-free survival or overall survival were found in the phase 3 ASSURE between patients receiving adjuvant sunitinib and those receiving placebo, according to results published in The Lancet. However, adjuvant sunitinib prolonged disease-free survival by 1.2 years, compared with placebo, in the phase 3 S-TRAC trial, presented at the 2016 ESMO Congress and published in the New England Journal of Medicine. S-TRAC results are the basis for the new drug application submitted by Pfizer, Inc., according to a press release.
The advisory committee will consider comments from the public if submitted by Sept. 5, as electronic comments through the electronic filing system or by mail/hand delivery/courier at Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
The docket number is FDA-2017-N-1063.
The Oncologic Drugs Advisory Committee to the Food and Drug Administration will meet on Sept. 19 to discuss a supplemental new drug application for sunitinib (Sutent), for the adjuvant treatment of adult patients at high risk of recurrent renal cell carcinoma (RCC) following nephrectomy.
Sunitinib is an oral antiangiogenic agent that has been approved for the treatment of advanced RCC since 2006.
The FDA accepted the new drug application in May and is expected to issue a decision by January 2018.
Results for sunitinib as adjuvant treatment have been mixed. No significant differences in disease-free survival or overall survival were found in the phase 3 ASSURE between patients receiving adjuvant sunitinib and those receiving placebo, according to results published in The Lancet. However, adjuvant sunitinib prolonged disease-free survival by 1.2 years, compared with placebo, in the phase 3 S-TRAC trial, presented at the 2016 ESMO Congress and published in the New England Journal of Medicine. S-TRAC results are the basis for the new drug application submitted by Pfizer, Inc., according to a press release.
The advisory committee will consider comments from the public if submitted by Sept. 5, as electronic comments through the electronic filing system or by mail/hand delivery/courier at Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
The docket number is FDA-2017-N-1063.
The Oncologic Drugs Advisory Committee to the Food and Drug Administration will meet on Sept. 19 to discuss a supplemental new drug application for sunitinib (Sutent), for the adjuvant treatment of adult patients at high risk of recurrent renal cell carcinoma (RCC) following nephrectomy.
Sunitinib is an oral antiangiogenic agent that has been approved for the treatment of advanced RCC since 2006.
The FDA accepted the new drug application in May and is expected to issue a decision by January 2018.
Results for sunitinib as adjuvant treatment have been mixed. No significant differences in disease-free survival or overall survival were found in the phase 3 ASSURE between patients receiving adjuvant sunitinib and those receiving placebo, according to results published in The Lancet. However, adjuvant sunitinib prolonged disease-free survival by 1.2 years, compared with placebo, in the phase 3 S-TRAC trial, presented at the 2016 ESMO Congress and published in the New England Journal of Medicine. S-TRAC results are the basis for the new drug application submitted by Pfizer, Inc., according to a press release.
The advisory committee will consider comments from the public if submitted by Sept. 5, as electronic comments through the electronic filing system or by mail/hand delivery/courier at Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
The docket number is FDA-2017-N-1063.
Role of fidaxomicin for C. difficile infection continues to evolve
SAN FRANCISCO– The role of fidaxomicin for treating mild to moderate Clostridium difficile infection is still finding its way, according to Sarah Doernberg, MD.
For now, fidaxomicin, a narrow spectrum macrocyclic antibiotic, may be appropriate for those at high risk for relapse and/or those requiring concomitant antibiotics – but its high price tag may be prohibitive.
“I think a lot of centers have taken the fidaxomicin data, which were based on patients with initial infection or a single relapse, and extrapolated it to patients with multiple relapses,” Dr. Doernberg, medical director of adult antimicrobial stewardship at UCSF Medical Center, said at the UCSF Annual Advances in Internal Medicine meeting.
In a recent analysis, researchers collected real-world data on implementation of fidaxomicin for CDI patients at seven hospitals in the United Kingdom (Eur J Clin Microbiol Infect Dis. 2016 Feb;35[2]:251-9). In two hospitals where fidaxomicin was used as the first-line treatment for all primary and recurrent episodes, the recurrence rate reduced from 10.6% to 3.1% and from 16.3% to 3.1%, with a significant difference in 28-day mortality, from 18.2% to 3.1%; (P less than .05) and 17.3% to 6.3% (P less than .05). In the remaining five hospitals that used fidaxomicin in selected patients only, the changes in recurrence rates and mortality were less marked.
Other studies have found that fidaxomicin has a similar cure rate, compared with vancomycin (around 88%) but a lower recurrence rate (13%-15%, compared with 25%-27%, respectively; see N Engl J Med. 2011 Feb 3;364[5]:422-31). However, the general cost for a course is significantly more, compared with metronidazole and vancomycin, making fidaxomicin less cost effective as a first-line agent in most cases (Clin Infect Dis. 2013 Aug 15; 57[4]:555-61).
“Our guidelines recommend it in patients who have a very high risk for relapse who would not be candidates for fecal transplant down the line, which generally means immunocompromised patients,” Dr. Doernberg said.
Additional considerations when treating CDI include stopping unnecessary antibiotics, shortening the antibiotic course, narrowing the antibiotic spectrum, and stopping acid-suppressive medication when possible, especially proton pump inhibitors. “Do no use anti-peristaltic agents until acute symptoms of CDI improve,” she said.
Dr. Doernberg disclosed that she is a consultant for Actelion. She has also conducted prior research studies with Cerexa, Cubist, and Merck.
SAN FRANCISCO– The role of fidaxomicin for treating mild to moderate Clostridium difficile infection is still finding its way, according to Sarah Doernberg, MD.
For now, fidaxomicin, a narrow spectrum macrocyclic antibiotic, may be appropriate for those at high risk for relapse and/or those requiring concomitant antibiotics – but its high price tag may be prohibitive.
“I think a lot of centers have taken the fidaxomicin data, which were based on patients with initial infection or a single relapse, and extrapolated it to patients with multiple relapses,” Dr. Doernberg, medical director of adult antimicrobial stewardship at UCSF Medical Center, said at the UCSF Annual Advances in Internal Medicine meeting.
In a recent analysis, researchers collected real-world data on implementation of fidaxomicin for CDI patients at seven hospitals in the United Kingdom (Eur J Clin Microbiol Infect Dis. 2016 Feb;35[2]:251-9). In two hospitals where fidaxomicin was used as the first-line treatment for all primary and recurrent episodes, the recurrence rate reduced from 10.6% to 3.1% and from 16.3% to 3.1%, with a significant difference in 28-day mortality, from 18.2% to 3.1%; (P less than .05) and 17.3% to 6.3% (P less than .05). In the remaining five hospitals that used fidaxomicin in selected patients only, the changes in recurrence rates and mortality were less marked.
Other studies have found that fidaxomicin has a similar cure rate, compared with vancomycin (around 88%) but a lower recurrence rate (13%-15%, compared with 25%-27%, respectively; see N Engl J Med. 2011 Feb 3;364[5]:422-31). However, the general cost for a course is significantly more, compared with metronidazole and vancomycin, making fidaxomicin less cost effective as a first-line agent in most cases (Clin Infect Dis. 2013 Aug 15; 57[4]:555-61).
“Our guidelines recommend it in patients who have a very high risk for relapse who would not be candidates for fecal transplant down the line, which generally means immunocompromised patients,” Dr. Doernberg said.
Additional considerations when treating CDI include stopping unnecessary antibiotics, shortening the antibiotic course, narrowing the antibiotic spectrum, and stopping acid-suppressive medication when possible, especially proton pump inhibitors. “Do no use anti-peristaltic agents until acute symptoms of CDI improve,” she said.
Dr. Doernberg disclosed that she is a consultant for Actelion. She has also conducted prior research studies with Cerexa, Cubist, and Merck.
SAN FRANCISCO– The role of fidaxomicin for treating mild to moderate Clostridium difficile infection is still finding its way, according to Sarah Doernberg, MD.
For now, fidaxomicin, a narrow spectrum macrocyclic antibiotic, may be appropriate for those at high risk for relapse and/or those requiring concomitant antibiotics – but its high price tag may be prohibitive.
“I think a lot of centers have taken the fidaxomicin data, which were based on patients with initial infection or a single relapse, and extrapolated it to patients with multiple relapses,” Dr. Doernberg, medical director of adult antimicrobial stewardship at UCSF Medical Center, said at the UCSF Annual Advances in Internal Medicine meeting.
In a recent analysis, researchers collected real-world data on implementation of fidaxomicin for CDI patients at seven hospitals in the United Kingdom (Eur J Clin Microbiol Infect Dis. 2016 Feb;35[2]:251-9). In two hospitals where fidaxomicin was used as the first-line treatment for all primary and recurrent episodes, the recurrence rate reduced from 10.6% to 3.1% and from 16.3% to 3.1%, with a significant difference in 28-day mortality, from 18.2% to 3.1%; (P less than .05) and 17.3% to 6.3% (P less than .05). In the remaining five hospitals that used fidaxomicin in selected patients only, the changes in recurrence rates and mortality were less marked.
Other studies have found that fidaxomicin has a similar cure rate, compared with vancomycin (around 88%) but a lower recurrence rate (13%-15%, compared with 25%-27%, respectively; see N Engl J Med. 2011 Feb 3;364[5]:422-31). However, the general cost for a course is significantly more, compared with metronidazole and vancomycin, making fidaxomicin less cost effective as a first-line agent in most cases (Clin Infect Dis. 2013 Aug 15; 57[4]:555-61).
“Our guidelines recommend it in patients who have a very high risk for relapse who would not be candidates for fecal transplant down the line, which generally means immunocompromised patients,” Dr. Doernberg said.
Additional considerations when treating CDI include stopping unnecessary antibiotics, shortening the antibiotic course, narrowing the antibiotic spectrum, and stopping acid-suppressive medication when possible, especially proton pump inhibitors. “Do no use anti-peristaltic agents until acute symptoms of CDI improve,” she said.
Dr. Doernberg disclosed that she is a consultant for Actelion. She has also conducted prior research studies with Cerexa, Cubist, and Merck.
AT THE ANNUAL ADVANCES IN INTERNAL MEDICINE
Off-the-shelf T cells used to treat viral infections after HSCT
One-size-fits-all T cells designed to recognize and mount an immune response against five common viral pathogens may help to reduce the incidence of severe viral infections and treatment-related deaths in patients who have undergone hematopoietic stem cell transplants (HSCT), investigators reported.
Among 37 evaluable patients who had undergone an allogeneic HSCT, a single infusion of banked virus-specific T cells (VSTs) directed against adenovirus, BK virus, cytomegalovirus (CMV), Epstein-Barr virus (EBV), and human herpesvirus 6 (HHV-6) was associated with a 92% cumulative complete or partial response rate, reported Ifigeneia Tzannou, MD, and her colleagues from Baylor College of Medicine in Houston.
“Although a randomized trial will be required to definitively assess the value of banked VSTs, this study strongly suggests that off-the-shelf, multiple-virus–directed VSTs are a safe and effective broad-spectrum approach to treat severe viral infections after HSCT. These VSTs can be rapidly and cost effectively produced in scalable quantities with excellent long-term stability, which facilitates the broad implementation of this therapy,” they wrote in the Journal of Clinical Oncology (2017 Aug 7. doi: 10.1200/JCO.2017.73.0655).
Although adoptive transfer of VSTs derived from donor stem cells has been shown to protect patients against viral pathogens, the technique is hampered by costs, complexity, the time-consuming manufacturing process, and the need for seropositive donors, Dr. Tzannou and her colleagues pointed out.
“One way to overcome these limitations and to supply antiviral protection to recipients of allogeneic HSCT would be to prepare and cryopreserve banks of VST lines from healthy seropositive donors, which would be available for immediate use as an off-the-shelf product,” they wrote.
They tested this concept in a phase 2 clinical trial in 38 patients with a total of 45 infections.
A single infusion was associated with cumulative complete and partial responses rates of 71% in 7 patients with adenoviral infections, 100% in 16 patients with BK virus infections, 94% in 17 patients with CMV infections, 100% for 2 patients with EBV infections, and 67% for 3 patients with HHV-6 infections.
Seven of the 38 patients received VSTs for two viral infections, and all patients had viral control after a single infusion. All cases of CMV, adenovirus, and EBV infections were cleared from serum. One patient with HHV-6 encephalitis had complete resolution of encephalitis after one infusion and resolution of hemorrhagic cystitis after a second infusion; 14 patients with BK virus–associated hemorrhagic cystitis had clinical improvement or resolution of disease.
The infusions were delivered safely. After infusion, one patient developed recurrent grade 3 gastrointestinal graft versus host disease (GVHD) after a rapid corticosteroid taper, three patients had recurrent grade 1 or 2 skin GVHD, and two patients had de novo skin GVHD. Of the five cases of skin GVHD, four resolved with the administration of topical treatments and one with the reinstitution of corticosteroids after a taper.
“More widespread and earlier use of this modality could minimize both drug-related and virus-associated complications and thereby decrease treatment-related mortality in recipients of allogeneic HSCT,” the investigators wrote.
The study was supported by the National Heart, Lung, and Blood Institute; Conquer Cancer Foundation; and Dan L. Duncan Comprehensive Cancer Center. Dr. Tzannou disclosed having a consulting or advisory role with ViraCyte, and several coauthors reported financial ties with various companies.
One-size-fits-all T cells designed to recognize and mount an immune response against five common viral pathogens may help to reduce the incidence of severe viral infections and treatment-related deaths in patients who have undergone hematopoietic stem cell transplants (HSCT), investigators reported.
Among 37 evaluable patients who had undergone an allogeneic HSCT, a single infusion of banked virus-specific T cells (VSTs) directed against adenovirus, BK virus, cytomegalovirus (CMV), Epstein-Barr virus (EBV), and human herpesvirus 6 (HHV-6) was associated with a 92% cumulative complete or partial response rate, reported Ifigeneia Tzannou, MD, and her colleagues from Baylor College of Medicine in Houston.
“Although a randomized trial will be required to definitively assess the value of banked VSTs, this study strongly suggests that off-the-shelf, multiple-virus–directed VSTs are a safe and effective broad-spectrum approach to treat severe viral infections after HSCT. These VSTs can be rapidly and cost effectively produced in scalable quantities with excellent long-term stability, which facilitates the broad implementation of this therapy,” they wrote in the Journal of Clinical Oncology (2017 Aug 7. doi: 10.1200/JCO.2017.73.0655).
Although adoptive transfer of VSTs derived from donor stem cells has been shown to protect patients against viral pathogens, the technique is hampered by costs, complexity, the time-consuming manufacturing process, and the need for seropositive donors, Dr. Tzannou and her colleagues pointed out.
“One way to overcome these limitations and to supply antiviral protection to recipients of allogeneic HSCT would be to prepare and cryopreserve banks of VST lines from healthy seropositive donors, which would be available for immediate use as an off-the-shelf product,” they wrote.
They tested this concept in a phase 2 clinical trial in 38 patients with a total of 45 infections.
A single infusion was associated with cumulative complete and partial responses rates of 71% in 7 patients with adenoviral infections, 100% in 16 patients with BK virus infections, 94% in 17 patients with CMV infections, 100% for 2 patients with EBV infections, and 67% for 3 patients with HHV-6 infections.
Seven of the 38 patients received VSTs for two viral infections, and all patients had viral control after a single infusion. All cases of CMV, adenovirus, and EBV infections were cleared from serum. One patient with HHV-6 encephalitis had complete resolution of encephalitis after one infusion and resolution of hemorrhagic cystitis after a second infusion; 14 patients with BK virus–associated hemorrhagic cystitis had clinical improvement or resolution of disease.
The infusions were delivered safely. After infusion, one patient developed recurrent grade 3 gastrointestinal graft versus host disease (GVHD) after a rapid corticosteroid taper, three patients had recurrent grade 1 or 2 skin GVHD, and two patients had de novo skin GVHD. Of the five cases of skin GVHD, four resolved with the administration of topical treatments and one with the reinstitution of corticosteroids after a taper.
“More widespread and earlier use of this modality could minimize both drug-related and virus-associated complications and thereby decrease treatment-related mortality in recipients of allogeneic HSCT,” the investigators wrote.
The study was supported by the National Heart, Lung, and Blood Institute; Conquer Cancer Foundation; and Dan L. Duncan Comprehensive Cancer Center. Dr. Tzannou disclosed having a consulting or advisory role with ViraCyte, and several coauthors reported financial ties with various companies.
One-size-fits-all T cells designed to recognize and mount an immune response against five common viral pathogens may help to reduce the incidence of severe viral infections and treatment-related deaths in patients who have undergone hematopoietic stem cell transplants (HSCT), investigators reported.
Among 37 evaluable patients who had undergone an allogeneic HSCT, a single infusion of banked virus-specific T cells (VSTs) directed against adenovirus, BK virus, cytomegalovirus (CMV), Epstein-Barr virus (EBV), and human herpesvirus 6 (HHV-6) was associated with a 92% cumulative complete or partial response rate, reported Ifigeneia Tzannou, MD, and her colleagues from Baylor College of Medicine in Houston.
“Although a randomized trial will be required to definitively assess the value of banked VSTs, this study strongly suggests that off-the-shelf, multiple-virus–directed VSTs are a safe and effective broad-spectrum approach to treat severe viral infections after HSCT. These VSTs can be rapidly and cost effectively produced in scalable quantities with excellent long-term stability, which facilitates the broad implementation of this therapy,” they wrote in the Journal of Clinical Oncology (2017 Aug 7. doi: 10.1200/JCO.2017.73.0655).
Although adoptive transfer of VSTs derived from donor stem cells has been shown to protect patients against viral pathogens, the technique is hampered by costs, complexity, the time-consuming manufacturing process, and the need for seropositive donors, Dr. Tzannou and her colleagues pointed out.
“One way to overcome these limitations and to supply antiviral protection to recipients of allogeneic HSCT would be to prepare and cryopreserve banks of VST lines from healthy seropositive donors, which would be available for immediate use as an off-the-shelf product,” they wrote.
They tested this concept in a phase 2 clinical trial in 38 patients with a total of 45 infections.
A single infusion was associated with cumulative complete and partial responses rates of 71% in 7 patients with adenoviral infections, 100% in 16 patients with BK virus infections, 94% in 17 patients with CMV infections, 100% for 2 patients with EBV infections, and 67% for 3 patients with HHV-6 infections.
Seven of the 38 patients received VSTs for two viral infections, and all patients had viral control after a single infusion. All cases of CMV, adenovirus, and EBV infections were cleared from serum. One patient with HHV-6 encephalitis had complete resolution of encephalitis after one infusion and resolution of hemorrhagic cystitis after a second infusion; 14 patients with BK virus–associated hemorrhagic cystitis had clinical improvement or resolution of disease.
The infusions were delivered safely. After infusion, one patient developed recurrent grade 3 gastrointestinal graft versus host disease (GVHD) after a rapid corticosteroid taper, three patients had recurrent grade 1 or 2 skin GVHD, and two patients had de novo skin GVHD. Of the five cases of skin GVHD, four resolved with the administration of topical treatments and one with the reinstitution of corticosteroids after a taper.
“More widespread and earlier use of this modality could minimize both drug-related and virus-associated complications and thereby decrease treatment-related mortality in recipients of allogeneic HSCT,” the investigators wrote.
The study was supported by the National Heart, Lung, and Blood Institute; Conquer Cancer Foundation; and Dan L. Duncan Comprehensive Cancer Center. Dr. Tzannou disclosed having a consulting or advisory role with ViraCyte, and several coauthors reported financial ties with various companies.
FROM THE JOURNAL OF CLINICAL ONCOLOGY
Key clinical point: Off-the-shelf virus-specific T-cell preparations can effectively treat infections following HSCT.
Major finding: Banked T cells directed against five common viruses were associated with a 92% cumulative partial or complete response rate.
Data source: Phase 2 clinical trial in 38 patients with viral infections following HSCT.
Disclosures: The study was supported by the National Heart, Lung, and Blood Institute; Conquer Cancer Foundation; and Dan L. Duncan Comprehensive Cancer Center. Dr. Tzannou disclosed having a consulting or advisory role with ViraCyte, and several coauthors reported financial ties with various companies.