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RNS May Reduce the Risk of SUDEP
Responsive neurostimulation (RNS) appears to reduce the risk of sudden unexpected death in epilepsy (SUDEP), according to research published online ahead of print January 16 in Epilepsia. The results provide evidence that treatments that reduce seizures decrease the risk of SUDEP, according to the authors. The data also indicate that not every SUDEP follows a seizure.
Orrin Devinsky, MD, Director of the Comprehensive Epilepsy Center at New York University Langone Medical Center, and colleagues examined data for all deaths in patients with epilepsy who received RNS in clinical trials or following FDA approval through May 5, 2016, and adjudicated them for SUDEP. In all, 256 patients received treatment during trials, and 451 received treatment after FDA approval. 
The investigators observed 14 deaths, including two possible, one probable, and four definite cases of SUDEP. The rate of probable or definite SUDEP was 2.0/1,000 over 2,036 patient stimulation years and 2.3/1,000 over 2,208 patient implant years. The age-adjusted standardized mortality ratio for definite and probable SUDEP was 0.75.
“The SUDEP rate of 2.0/1,000 patient stimulation years among patients undergoing treatment with RNS is favorable relative to patients [with treatment-resistant epilepsy] randomized to the placebo arm of add-on drug studies (SUDEP rate of 6.1/1,000 patient years), patients who were referred for epilepsy surgery but did not receive epilepsy surgery (SUDEP rate of 6.3/1,000 patient years), and patients with recurrent seizures after epilepsy surgery (SUDEP rate 6.3/1,000 patient years),” said the authors. “Future explorations can examine whether certain seizure patterns are particularly associated with SUDEP.”
—Erik Greb
Suggested Reading
Devinsky O, Friedman D, Duckrow RB, et al. Sudden unexpected death in epilepsy in patients treated with brain-responsive neurostimulation. Epilepsia. 2018 Jan 16 [Epub ahead of print].
Responsive neurostimulation (RNS) appears to reduce the risk of sudden unexpected death in epilepsy (SUDEP), according to research published online ahead of print January 16 in Epilepsia. The results provide evidence that treatments that reduce seizures decrease the risk of SUDEP, according to the authors. The data also indicate that not every SUDEP follows a seizure.
Orrin Devinsky, MD, Director of the Comprehensive Epilepsy Center at New York University Langone Medical Center, and colleagues examined data for all deaths in patients with epilepsy who received RNS in clinical trials or following FDA approval through May 5, 2016, and adjudicated them for SUDEP. In all, 256 patients received treatment during trials, and 451 received treatment after FDA approval.
The investigators observed 14 deaths, including two possible, one probable, and four definite cases of SUDEP. The rate of probable or definite SUDEP was 2.0/1,000 over 2,036 patient stimulation years and 2.3/1,000 over 2,208 patient implant years. The age-adjusted standardized mortality ratio for definite and probable SUDEP was 0.75.
“The SUDEP rate of 2.0/1,000 patient stimulation years among patients undergoing treatment with RNS is favorable relative to patients [with treatment-resistant epilepsy] randomized to the placebo arm of add-on drug studies (SUDEP rate of 6.1/1,000 patient years), patients who were referred for epilepsy surgery but did not receive epilepsy surgery (SUDEP rate of 6.3/1,000 patient years), and patients with recurrent seizures after epilepsy surgery (SUDEP rate 6.3/1,000 patient years),” said the authors. “Future explorations can examine whether certain seizure patterns are particularly associated with SUDEP.”
—Erik Greb
Suggested Reading
Devinsky O, Friedman D, Duckrow RB, et al. Sudden unexpected death in epilepsy in patients treated with brain-responsive neurostimulation. Epilepsia. 2018 Jan 16 [Epub ahead of print].
Responsive neurostimulation (RNS) appears to reduce the risk of sudden unexpected death in epilepsy (SUDEP), according to research published online ahead of print January 16 in Epilepsia. The results provide evidence that treatments that reduce seizures decrease the risk of SUDEP, according to the authors. The data also indicate that not every SUDEP follows a seizure.
Orrin Devinsky, MD, Director of the Comprehensive Epilepsy Center at New York University Langone Medical Center, and colleagues examined data for all deaths in patients with epilepsy who received RNS in clinical trials or following FDA approval through May 5, 2016, and adjudicated them for SUDEP. In all, 256 patients received treatment during trials, and 451 received treatment after FDA approval.
The investigators observed 14 deaths, including two possible, one probable, and four definite cases of SUDEP. The rate of probable or definite SUDEP was 2.0/1,000 over 2,036 patient stimulation years and 2.3/1,000 over 2,208 patient implant years. The age-adjusted standardized mortality ratio for definite and probable SUDEP was 0.75.
“The SUDEP rate of 2.0/1,000 patient stimulation years among patients undergoing treatment with RNS is favorable relative to patients [with treatment-resistant epilepsy] randomized to the placebo arm of add-on drug studies (SUDEP rate of 6.1/1,000 patient years), patients who were referred for epilepsy surgery but did not receive epilepsy surgery (SUDEP rate of 6.3/1,000 patient years), and patients with recurrent seizures after epilepsy surgery (SUDEP rate 6.3/1,000 patient years),” said the authors. “Future explorations can examine whether certain seizure patterns are particularly associated with SUDEP.”
—Erik Greb
Suggested Reading
Devinsky O, Friedman D, Duckrow RB, et al. Sudden unexpected death in epilepsy in patients treated with brain-responsive neurostimulation. Epilepsia. 2018 Jan 16 [Epub ahead of print].
Diagnostic delays, morbidity, and epidural abscesses
Clinical question: What is the frequency of diagnostic delays in epidural abscesses, and what factors may contribute to these delays?
Background: Diagnostic evaluation of back pain can be challenging. Missed diagnosis of serious conditions such as epidural abscesses can lead to significant morbidity.
Setting: Veterans Affairs Electronic Medical Record database from more than 1,700 VA outpatient and inpatient facilities in the United States.
Synopsis: Of the 119 patients with a new diagnosis of spinal epidural abscess, 55.5% were felt to have experienced a diagnostic error, defined by the study authors as a missed opportunity to evaluate a red flag (e.g. weight loss, neurologic deficit, fever) in a timely or appropriate manner. There was a significant difference in the time to diagnosis between patients with and without a diagnostic error (4 versus 12 days, P less than .01). Of those cases involving diagnostic error, 60.6% were felt to have resulted in serious patient harm and 12.1% in patient death. The most commonly missed red flags were fever, focal neurological deficits, and signs of active infection.
Based on these findings, the authors suggest that future intervention focus on improving information gathering during patient-physician encounter and physician education about existing guidelines.
The limitations of this study include its use of data from a single health system, and the employment of chart reviews instead of a root cause analysis based on provider and patient interviews.
Bottom line: A delay in diagnosis resulting in patient harm or death may occur frequently in cases of epidural abscesses. Further work on targeted interventions to reduce error and prevent harm are needed.
Citation: Bhise V, Meyer A, Singh H, et al. Diagnosis of spinal epidural abscesses in the era of electronic health records. Am J Med. 2017;130(8):975-81.
Dr. Pizza is a hospitalist, Beth Israel Deaconess Medical Center, and instructor in medicine, Harvard Medical School, Boston.
Clinical question: What is the frequency of diagnostic delays in epidural abscesses, and what factors may contribute to these delays?
Background: Diagnostic evaluation of back pain can be challenging. Missed diagnosis of serious conditions such as epidural abscesses can lead to significant morbidity.
Setting: Veterans Affairs Electronic Medical Record database from more than 1,700 VA outpatient and inpatient facilities in the United States.
Synopsis: Of the 119 patients with a new diagnosis of spinal epidural abscess, 55.5% were felt to have experienced a diagnostic error, defined by the study authors as a missed opportunity to evaluate a red flag (e.g. weight loss, neurologic deficit, fever) in a timely or appropriate manner. There was a significant difference in the time to diagnosis between patients with and without a diagnostic error (4 versus 12 days, P less than .01). Of those cases involving diagnostic error, 60.6% were felt to have resulted in serious patient harm and 12.1% in patient death. The most commonly missed red flags were fever, focal neurological deficits, and signs of active infection.
Based on these findings, the authors suggest that future intervention focus on improving information gathering during patient-physician encounter and physician education about existing guidelines.
The limitations of this study include its use of data from a single health system, and the employment of chart reviews instead of a root cause analysis based on provider and patient interviews.
Bottom line: A delay in diagnosis resulting in patient harm or death may occur frequently in cases of epidural abscesses. Further work on targeted interventions to reduce error and prevent harm are needed.
Citation: Bhise V, Meyer A, Singh H, et al. Diagnosis of spinal epidural abscesses in the era of electronic health records. Am J Med. 2017;130(8):975-81.
Dr. Pizza is a hospitalist, Beth Israel Deaconess Medical Center, and instructor in medicine, Harvard Medical School, Boston.
Clinical question: What is the frequency of diagnostic delays in epidural abscesses, and what factors may contribute to these delays?
Background: Diagnostic evaluation of back pain can be challenging. Missed diagnosis of serious conditions such as epidural abscesses can lead to significant morbidity.
Setting: Veterans Affairs Electronic Medical Record database from more than 1,700 VA outpatient and inpatient facilities in the United States.
Synopsis: Of the 119 patients with a new diagnosis of spinal epidural abscess, 55.5% were felt to have experienced a diagnostic error, defined by the study authors as a missed opportunity to evaluate a red flag (e.g. weight loss, neurologic deficit, fever) in a timely or appropriate manner. There was a significant difference in the time to diagnosis between patients with and without a diagnostic error (4 versus 12 days, P less than .01). Of those cases involving diagnostic error, 60.6% were felt to have resulted in serious patient harm and 12.1% in patient death. The most commonly missed red flags were fever, focal neurological deficits, and signs of active infection.
Based on these findings, the authors suggest that future intervention focus on improving information gathering during patient-physician encounter and physician education about existing guidelines.
The limitations of this study include its use of data from a single health system, and the employment of chart reviews instead of a root cause analysis based on provider and patient interviews.
Bottom line: A delay in diagnosis resulting in patient harm or death may occur frequently in cases of epidural abscesses. Further work on targeted interventions to reduce error and prevent harm are needed.
Citation: Bhise V, Meyer A, Singh H, et al. Diagnosis of spinal epidural abscesses in the era of electronic health records. Am J Med. 2017;130(8):975-81.
Dr. Pizza is a hospitalist, Beth Israel Deaconess Medical Center, and instructor in medicine, Harvard Medical School, Boston.
Parental leave not available to all academic surgeons
Paid parental leave policies have been unevenly adopted among academic medical centers, according to a study published in the Journal of Surgical Research. These policies, or their lack, may have important ramifications for recruiting and specialty selection by surgeons, and for women of child-rearing age in particular.
Parental leave for surgeons has been championed by the American College of Surgeons, among other professional societies, in formal statements and supportive policies in recent years.
Dina S. Itum, MD, a fifth-year resident in the department of surgery, University of Texas Southwestern Medical Center, Houston, and a research team looked into how widespread parental leave is for surgeons in US medical centers. Their sample was the 91 top-ranked academic medical centers identified by U.S. News & World Report in 2016. The method used by U.S. News & World Report for ranking medical centers is based on student selectivity, dean and residency directors’ peer assessment of national institutions, faculty to student ratio, and the dollar amount in NIH research grants received.
“Parental leave” was defined by the research team as any leave dedicated to new mothers, fathers and/or primary caregivers after childbirth or adoption. “Paid leave” was defined as some protected leave with salary without mandated use of sick leave or vacation leave.
The study found that among the top-ranked 91 institutions, 48 (53%) offered some form of paid parental leave to faculty surgeons. The higher the rating, the more likely the institution offered paid parental leave: 77% of those in the top third of rankings vs. 53% in the middle third and 29% in the bottom third. Private institutions were more likely to offer paid leave of 6 weeks or longer; 67% vs. 33% of public institutions.
The investigators posed a question: Did these institutions implement the policy to attract the top talent, or did the policy improve faculty morale and productivity leading to a higher ranking? The study does not answer the question, but the investigators consider it an important issue for further study.
The investigators also suggested that surgeons of child-rearing age use parental leave information their in their own employment negotiations. “As physicians, we are aware of the health care benefits associated with parental leave, and as leaders within our communities, we should be at the forefront of supporting further advancement of this benefit,” the investigators wrote.
The investigators reported having no financial disclosures.
SOURCE: Itum DS et al. J Surg Res. 2018 Feb 3. doi. org/10.1016/j.jss.2018.01.001.
Paid parental leave policies have been unevenly adopted among academic medical centers, according to a study published in the Journal of Surgical Research. These policies, or their lack, may have important ramifications for recruiting and specialty selection by surgeons, and for women of child-rearing age in particular.
Parental leave for surgeons has been championed by the American College of Surgeons, among other professional societies, in formal statements and supportive policies in recent years.
Dina S. Itum, MD, a fifth-year resident in the department of surgery, University of Texas Southwestern Medical Center, Houston, and a research team looked into how widespread parental leave is for surgeons in US medical centers. Their sample was the 91 top-ranked academic medical centers identified by U.S. News & World Report in 2016. The method used by U.S. News & World Report for ranking medical centers is based on student selectivity, dean and residency directors’ peer assessment of national institutions, faculty to student ratio, and the dollar amount in NIH research grants received.
“Parental leave” was defined by the research team as any leave dedicated to new mothers, fathers and/or primary caregivers after childbirth or adoption. “Paid leave” was defined as some protected leave with salary without mandated use of sick leave or vacation leave.
The study found that among the top-ranked 91 institutions, 48 (53%) offered some form of paid parental leave to faculty surgeons. The higher the rating, the more likely the institution offered paid parental leave: 77% of those in the top third of rankings vs. 53% in the middle third and 29% in the bottom third. Private institutions were more likely to offer paid leave of 6 weeks or longer; 67% vs. 33% of public institutions.
The investigators posed a question: Did these institutions implement the policy to attract the top talent, or did the policy improve faculty morale and productivity leading to a higher ranking? The study does not answer the question, but the investigators consider it an important issue for further study.
The investigators also suggested that surgeons of child-rearing age use parental leave information their in their own employment negotiations. “As physicians, we are aware of the health care benefits associated with parental leave, and as leaders within our communities, we should be at the forefront of supporting further advancement of this benefit,” the investigators wrote.
The investigators reported having no financial disclosures.
SOURCE: Itum DS et al. J Surg Res. 2018 Feb 3. doi. org/10.1016/j.jss.2018.01.001.
Paid parental leave policies have been unevenly adopted among academic medical centers, according to a study published in the Journal of Surgical Research. These policies, or their lack, may have important ramifications for recruiting and specialty selection by surgeons, and for women of child-rearing age in particular.
Parental leave for surgeons has been championed by the American College of Surgeons, among other professional societies, in formal statements and supportive policies in recent years.
Dina S. Itum, MD, a fifth-year resident in the department of surgery, University of Texas Southwestern Medical Center, Houston, and a research team looked into how widespread parental leave is for surgeons in US medical centers. Their sample was the 91 top-ranked academic medical centers identified by U.S. News & World Report in 2016. The method used by U.S. News & World Report for ranking medical centers is based on student selectivity, dean and residency directors’ peer assessment of national institutions, faculty to student ratio, and the dollar amount in NIH research grants received.
“Parental leave” was defined by the research team as any leave dedicated to new mothers, fathers and/or primary caregivers after childbirth or adoption. “Paid leave” was defined as some protected leave with salary without mandated use of sick leave or vacation leave.
The study found that among the top-ranked 91 institutions, 48 (53%) offered some form of paid parental leave to faculty surgeons. The higher the rating, the more likely the institution offered paid parental leave: 77% of those in the top third of rankings vs. 53% in the middle third and 29% in the bottom third. Private institutions were more likely to offer paid leave of 6 weeks or longer; 67% vs. 33% of public institutions.
The investigators posed a question: Did these institutions implement the policy to attract the top talent, or did the policy improve faculty morale and productivity leading to a higher ranking? The study does not answer the question, but the investigators consider it an important issue for further study.
The investigators also suggested that surgeons of child-rearing age use parental leave information their in their own employment negotiations. “As physicians, we are aware of the health care benefits associated with parental leave, and as leaders within our communities, we should be at the forefront of supporting further advancement of this benefit,” the investigators wrote.
The investigators reported having no financial disclosures.
SOURCE: Itum DS et al. J Surg Res. 2018 Feb 3. doi. org/10.1016/j.jss.2018.01.001.
FROM THE JOURNAL OF SURGICAL RESEARCH
Key clinical point: Parental leave policies have been unevenly adopted by U.S. medical schools.
Major finding: Among the top 91 ranked medical schools, 53% offer paid parental leave.
Data source: Survey of 91 top academic medical centers identified in U.S. News & World Report 2016 listing.
Disclosures: The investigators reported having no financial disclosures.
SOURCE: Itum DS et al. J Surg Res. 2018 Feb 3. doi. org/10.1016/j.jss.2018.01.001.
FDA grants priority review for AML drug
The Food and Drug Administration has granted priority review status to ivosidenib for the treatment of patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase 1 mutation.
The drug, marketed by Agios Pharmaceuticals, was given a Prescription Drug User Free Act action date of Aug. 21, 2018.
Results from a phase 1 dose-escalation and expansion study (AG120-C-001) presented at the annual meeting of the American Society of Hematology showed a complete response and complete response with partial hematologic recovery rate of 30.4% in 125 patients with relapsed/refractory AML who received the drug, according to Agios.
The Food and Drug Administration has granted priority review status to ivosidenib for the treatment of patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase 1 mutation.
The drug, marketed by Agios Pharmaceuticals, was given a Prescription Drug User Free Act action date of Aug. 21, 2018.
Results from a phase 1 dose-escalation and expansion study (AG120-C-001) presented at the annual meeting of the American Society of Hematology showed a complete response and complete response with partial hematologic recovery rate of 30.4% in 125 patients with relapsed/refractory AML who received the drug, according to Agios.
The Food and Drug Administration has granted priority review status to ivosidenib for the treatment of patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase 1 mutation.
The drug, marketed by Agios Pharmaceuticals, was given a Prescription Drug User Free Act action date of Aug. 21, 2018.
Results from a phase 1 dose-escalation and expansion study (AG120-C-001) presented at the annual meeting of the American Society of Hematology showed a complete response and complete response with partial hematologic recovery rate of 30.4% in 125 patients with relapsed/refractory AML who received the drug, according to Agios.
Mortality estimates put pancreatic cancer on the map
The alphabet may put Vermont right after Utah, but pancreatic cancer mortality has them on opposite sides of the country.
The expected number of deaths for 2018 and the current U.S. population estimate of nearly 326 million produce an expected death rate of 13.6 per 100,000 population. The Census Bureau estimates for the state populations, the deaths projected by the ACS, and a little math result in expected death rates of 8.7 per 100,000 for Utah and 17.7 for Vermont.
The incidence of pancreatic cancer increased by about 1% per year in whites and was stable in blacks from 2005 to 2014, although the rate of new cases in blacks is still about 25% higher than it is for whites. An estimated 55,440 new cases are expected in the United States in 2018, the ACS reported.
Although the survival rate for cancer of the pancreas has tripled since the mid-1970s, it is still much lower than for any other cancer. Five-year relative survival went from 3% in 1975-1977 to 9% in 2007-2013 for pancreatic cancer, while liver and intrahepatic bile duct cancer, which had the next-lowest survival rate, went from 3% to 19%, the ACS said.
The alphabet may put Vermont right after Utah, but pancreatic cancer mortality has them on opposite sides of the country.
The expected number of deaths for 2018 and the current U.S. population estimate of nearly 326 million produce an expected death rate of 13.6 per 100,000 population. The Census Bureau estimates for the state populations, the deaths projected by the ACS, and a little math result in expected death rates of 8.7 per 100,000 for Utah and 17.7 for Vermont.
The incidence of pancreatic cancer increased by about 1% per year in whites and was stable in blacks from 2005 to 2014, although the rate of new cases in blacks is still about 25% higher than it is for whites. An estimated 55,440 new cases are expected in the United States in 2018, the ACS reported.
Although the survival rate for cancer of the pancreas has tripled since the mid-1970s, it is still much lower than for any other cancer. Five-year relative survival went from 3% in 1975-1977 to 9% in 2007-2013 for pancreatic cancer, while liver and intrahepatic bile duct cancer, which had the next-lowest survival rate, went from 3% to 19%, the ACS said.
The alphabet may put Vermont right after Utah, but pancreatic cancer mortality has them on opposite sides of the country.
The expected number of deaths for 2018 and the current U.S. population estimate of nearly 326 million produce an expected death rate of 13.6 per 100,000 population. The Census Bureau estimates for the state populations, the deaths projected by the ACS, and a little math result in expected death rates of 8.7 per 100,000 for Utah and 17.7 for Vermont.
The incidence of pancreatic cancer increased by about 1% per year in whites and was stable in blacks from 2005 to 2014, although the rate of new cases in blacks is still about 25% higher than it is for whites. An estimated 55,440 new cases are expected in the United States in 2018, the ACS reported.
Although the survival rate for cancer of the pancreas has tripled since the mid-1970s, it is still much lower than for any other cancer. Five-year relative survival went from 3% in 1975-1977 to 9% in 2007-2013 for pancreatic cancer, while liver and intrahepatic bile duct cancer, which had the next-lowest survival rate, went from 3% to 19%, the ACS said.
Legacy Society members sustain research
AGA Legacy Society members share a desire to guarantee long-term support for digestive disease research. Through their foresight and generosity, they help ensure the continued momentum of discovery and patient education that has characterized GI medicine in recent decades. Legacy Society member donations directly support young GI investigators as they establish independent research careers.
Legacy Society members are the most generous individual donors to the AGA Research Foundation. Members of the AGA Legacy Society provide tax-deductible gifts to the AGA Research Foundation of $5,000 or more per year for 5 years ($25,000 total) or $50,000 or more in a planned gift, such as a bequest. All Legacy Society contributions go directly to support research awards.
AGA members support young researchers at a critical decision point in their lives – when many consider giving up their research careers due to a lack of funding. “I am honored to be a recipient of the Research Scholar Award. I would like to thank the foundation for their generous contribution that will fund a crucial transition in my career,” said Jose Saenz, MD, PhD, Washington University School of Medicine and 2017 AGA – Gastric Cancer Foundation Research Scholar Award recipient.
The AGA Research Foundation’s mission is to raise funds to support young researchers in gastroenterology and hepatology. Gifts to the foundation support researchers working towards developing new treatments and diagnostics for patients with GI conditions.
“I am extremely grateful to be selected for this award. I would like to thank the foundation donors for their generous support. This award will me build a research program to better understand mechanisms that promote growth of cholangiocarcinoma,” remarks Silvia Affe, PhD, Columbia University, 2017 AGA Research Scholar recipient.
Donors who make gifts at the Legacy Society level before DDW will receive an invitation to the annual Benefactors’ Dinner at the Folger Shakespeare Library in Washington, DC. Individuals interested in learning more about Legacy Society membership may contact Stacey Hinton Tuneski, Senior Director of Development at [email protected] or via phone (301) 222-4005. More information on the AGA Legacy Society including the current roster and acceptance form is available on the foundation’s website at www.gastro.org/legacysociety.
The makings of a grand celebration
Beginning with a memorable gathering at the United States Library of Congress in 2007, the AGA Benefactors’ Dinner has welcomed members of the AGA Legacy Society and other AGA dignitaries to special locations nationwide. The Folger Shakespeare Library will be the location of the 2018 AGA Research Foundation Benefactors Dinner during DDW in Washington, DC. Just steps from the Capitol, the Great Hall and Pastor Reading room are a spectacular setting for an enjoyable evening with friends. Members of the AGA Legacy Society will be among the distinguished honorees at the annual event.
AGA Legacy Society members share a desire to guarantee long-term support for digestive disease research. Through their foresight and generosity, they help ensure the continued momentum of discovery and patient education that has characterized GI medicine in recent decades. Legacy Society member donations directly support young GI investigators as they establish independent research careers.
Legacy Society members are the most generous individual donors to the AGA Research Foundation. Members of the AGA Legacy Society provide tax-deductible gifts to the AGA Research Foundation of $5,000 or more per year for 5 years ($25,000 total) or $50,000 or more in a planned gift, such as a bequest. All Legacy Society contributions go directly to support research awards.
AGA members support young researchers at a critical decision point in their lives – when many consider giving up their research careers due to a lack of funding. “I am honored to be a recipient of the Research Scholar Award. I would like to thank the foundation for their generous contribution that will fund a crucial transition in my career,” said Jose Saenz, MD, PhD, Washington University School of Medicine and 2017 AGA – Gastric Cancer Foundation Research Scholar Award recipient.
The AGA Research Foundation’s mission is to raise funds to support young researchers in gastroenterology and hepatology. Gifts to the foundation support researchers working towards developing new treatments and diagnostics for patients with GI conditions.
“I am extremely grateful to be selected for this award. I would like to thank the foundation donors for their generous support. This award will me build a research program to better understand mechanisms that promote growth of cholangiocarcinoma,” remarks Silvia Affe, PhD, Columbia University, 2017 AGA Research Scholar recipient.
Donors who make gifts at the Legacy Society level before DDW will receive an invitation to the annual Benefactors’ Dinner at the Folger Shakespeare Library in Washington, DC. Individuals interested in learning more about Legacy Society membership may contact Stacey Hinton Tuneski, Senior Director of Development at [email protected] or via phone (301) 222-4005. More information on the AGA Legacy Society including the current roster and acceptance form is available on the foundation’s website at www.gastro.org/legacysociety.
The makings of a grand celebration
Beginning with a memorable gathering at the United States Library of Congress in 2007, the AGA Benefactors’ Dinner has welcomed members of the AGA Legacy Society and other AGA dignitaries to special locations nationwide. The Folger Shakespeare Library will be the location of the 2018 AGA Research Foundation Benefactors Dinner during DDW in Washington, DC. Just steps from the Capitol, the Great Hall and Pastor Reading room are a spectacular setting for an enjoyable evening with friends. Members of the AGA Legacy Society will be among the distinguished honorees at the annual event.
AGA Legacy Society members share a desire to guarantee long-term support for digestive disease research. Through their foresight and generosity, they help ensure the continued momentum of discovery and patient education that has characterized GI medicine in recent decades. Legacy Society member donations directly support young GI investigators as they establish independent research careers.
Legacy Society members are the most generous individual donors to the AGA Research Foundation. Members of the AGA Legacy Society provide tax-deductible gifts to the AGA Research Foundation of $5,000 or more per year for 5 years ($25,000 total) or $50,000 or more in a planned gift, such as a bequest. All Legacy Society contributions go directly to support research awards.
AGA members support young researchers at a critical decision point in their lives – when many consider giving up their research careers due to a lack of funding. “I am honored to be a recipient of the Research Scholar Award. I would like to thank the foundation for their generous contribution that will fund a crucial transition in my career,” said Jose Saenz, MD, PhD, Washington University School of Medicine and 2017 AGA – Gastric Cancer Foundation Research Scholar Award recipient.
The AGA Research Foundation’s mission is to raise funds to support young researchers in gastroenterology and hepatology. Gifts to the foundation support researchers working towards developing new treatments and diagnostics for patients with GI conditions.
“I am extremely grateful to be selected for this award. I would like to thank the foundation donors for their generous support. This award will me build a research program to better understand mechanisms that promote growth of cholangiocarcinoma,” remarks Silvia Affe, PhD, Columbia University, 2017 AGA Research Scholar recipient.
Donors who make gifts at the Legacy Society level before DDW will receive an invitation to the annual Benefactors’ Dinner at the Folger Shakespeare Library in Washington, DC. Individuals interested in learning more about Legacy Society membership may contact Stacey Hinton Tuneski, Senior Director of Development at [email protected] or via phone (301) 222-4005. More information on the AGA Legacy Society including the current roster and acceptance form is available on the foundation’s website at www.gastro.org/legacysociety.
The makings of a grand celebration
Beginning with a memorable gathering at the United States Library of Congress in 2007, the AGA Benefactors’ Dinner has welcomed members of the AGA Legacy Society and other AGA dignitaries to special locations nationwide. The Folger Shakespeare Library will be the location of the 2018 AGA Research Foundation Benefactors Dinner during DDW in Washington, DC. Just steps from the Capitol, the Great Hall and Pastor Reading room are a spectacular setting for an enjoyable evening with friends. Members of the AGA Legacy Society will be among the distinguished honorees at the annual event.
MDedge Daily News: The Nonallergen of the Year
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
Parabens are named “nonallergen” of the year, a gene test guides need for sentinel node biopsy in elderly melanoma patients, select atopic dermatitis patients need patch testing, and try these real solutions for delusional parasitosis.
Listen to the MDedge Daily News podcast for all the details on today’s top news.
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
Parabens are named “nonallergen” of the year, a gene test guides need for sentinel node biopsy in elderly melanoma patients, select atopic dermatitis patients need patch testing, and try these real solutions for delusional parasitosis.
Listen to the MDedge Daily News podcast for all the details on today’s top news.
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
Parabens are named “nonallergen” of the year, a gene test guides need for sentinel node biopsy in elderly melanoma patients, select atopic dermatitis patients need patch testing, and try these real solutions for delusional parasitosis.
Listen to the MDedge Daily News podcast for all the details on today’s top news.
AGA Pres. Sheila Crowe Spends the Day on Capitol Hill
AGA President Sheila Crowe, MD, FRCPC, FACP, FACG, AGAF, recently spent the day on Capitol Hill meeting with lawmakers to advocate for AGA legislative priorities including increasing funding for NIH and biomedical research, support for the Removing Barriers to Colorectal Cancer Screening Act, and support for the Restoring the Patient’s Voice Act. Dr. Crowe met with eight congressional offices and received helpful feedback on the upcoming agenda in Congress and how it impacts AGA’s priorities.
NIH funding
Removing Barriers to Colorectal Cancer Screening Act
Fixing the current coinsurance problem for Medicare beneficiaries who undergo a screening colonoscopy that becomes therapeutic remains a top AGA priority. Most of the offices that Dr. Crowe met with were cosponsors of the legislation, the Removing Barriers to Colorectal Cancer Screening Act (HR 1017/S.479), that would waive coinsurance payment regardless of the screening outcome. Dr. Crowe shared her experience with patients and the financial burden this places on beneficiaries who need to be screened. Rep. Raul Ruiz, D-CA, and Rep. Scott Peters, D-CA, both members of the House Energy and Commerce Committee and supporters of the bill, will continue to advocate that the bill receive a hearing this year to help move it through Congress. The bill continues to have wide bipartisan support. Read more about the issue and how you can explain it to your patients.
Step therapy
More and more patients are being subject to step therapy protocols, also known as “fail first” under which they are required to try and fail sometimes two or three therapies before receiving coverage of the initial therapy recommended by their physician. With the emergence of new biologics to treat diseases like inflammatory bowel disease, more and more digestive disease patients are being subject to these protocols, which can have adverse effects on their health. Restoring the Patient’s Voice Act (HR 2077) would provide patients and providers with a fair and equitable appeals process when step therapy has been imposed and provides common sense exceptions for the provider to appeal. Dr. Crowe spoke of the impact this policy is having on digestive disease patients and the burden it puts on physician practices that have to take time away from patients to navigate the convoluted insurance appeals process. We are hopeful that many of the offices that we met with will support HR 2077. Read more about the issue.
Food is Medicine Working Group
Dr. Crowe also had a productive meeting with Rep. Jim McGovern’s, D-MA, office and learned more about the recently created Food is Medicine Working Group that he has initiated. McGovern is the Ranking Member of the Agriculture Committee’s Subcommittee on Nutrition which is responsible for our nation’s nutrition guidelines and the Supplemental Nutrition Assistance Program. The Working Group will focus on costs related to hunger and the importance of nutrition in treating chronic illness and disease. AGA looks forward to working with McGovern and members of the Working Group on this bipartisan initiative.
Capitol Hill needs to hear the voice of GI
In conjunction with Dr. Crowe’s visit, AGA launched a Virtual Advocacy Day to encourage members to contact their legislators in support of the issues that Dr. Crowe was advocating during her meetings. We thank those members who took time out of their schedules to take action.
AGA President Sheila Crowe, MD, FRCPC, FACP, FACG, AGAF, recently spent the day on Capitol Hill meeting with lawmakers to advocate for AGA legislative priorities including increasing funding for NIH and biomedical research, support for the Removing Barriers to Colorectal Cancer Screening Act, and support for the Restoring the Patient’s Voice Act. Dr. Crowe met with eight congressional offices and received helpful feedback on the upcoming agenda in Congress and how it impacts AGA’s priorities.
NIH funding
Removing Barriers to Colorectal Cancer Screening Act
Fixing the current coinsurance problem for Medicare beneficiaries who undergo a screening colonoscopy that becomes therapeutic remains a top AGA priority. Most of the offices that Dr. Crowe met with were cosponsors of the legislation, the Removing Barriers to Colorectal Cancer Screening Act (HR 1017/S.479), that would waive coinsurance payment regardless of the screening outcome. Dr. Crowe shared her experience with patients and the financial burden this places on beneficiaries who need to be screened. Rep. Raul Ruiz, D-CA, and Rep. Scott Peters, D-CA, both members of the House Energy and Commerce Committee and supporters of the bill, will continue to advocate that the bill receive a hearing this year to help move it through Congress. The bill continues to have wide bipartisan support. Read more about the issue and how you can explain it to your patients.
Step therapy
More and more patients are being subject to step therapy protocols, also known as “fail first” under which they are required to try and fail sometimes two or three therapies before receiving coverage of the initial therapy recommended by their physician. With the emergence of new biologics to treat diseases like inflammatory bowel disease, more and more digestive disease patients are being subject to these protocols, which can have adverse effects on their health. Restoring the Patient’s Voice Act (HR 2077) would provide patients and providers with a fair and equitable appeals process when step therapy has been imposed and provides common sense exceptions for the provider to appeal. Dr. Crowe spoke of the impact this policy is having on digestive disease patients and the burden it puts on physician practices that have to take time away from patients to navigate the convoluted insurance appeals process. We are hopeful that many of the offices that we met with will support HR 2077. Read more about the issue.
Food is Medicine Working Group
Dr. Crowe also had a productive meeting with Rep. Jim McGovern’s, D-MA, office and learned more about the recently created Food is Medicine Working Group that he has initiated. McGovern is the Ranking Member of the Agriculture Committee’s Subcommittee on Nutrition which is responsible for our nation’s nutrition guidelines and the Supplemental Nutrition Assistance Program. The Working Group will focus on costs related to hunger and the importance of nutrition in treating chronic illness and disease. AGA looks forward to working with McGovern and members of the Working Group on this bipartisan initiative.
Capitol Hill needs to hear the voice of GI
In conjunction with Dr. Crowe’s visit, AGA launched a Virtual Advocacy Day to encourage members to contact their legislators in support of the issues that Dr. Crowe was advocating during her meetings. We thank those members who took time out of their schedules to take action.
AGA President Sheila Crowe, MD, FRCPC, FACP, FACG, AGAF, recently spent the day on Capitol Hill meeting with lawmakers to advocate for AGA legislative priorities including increasing funding for NIH and biomedical research, support for the Removing Barriers to Colorectal Cancer Screening Act, and support for the Restoring the Patient’s Voice Act. Dr. Crowe met with eight congressional offices and received helpful feedback on the upcoming agenda in Congress and how it impacts AGA’s priorities.
NIH funding
Removing Barriers to Colorectal Cancer Screening Act
Fixing the current coinsurance problem for Medicare beneficiaries who undergo a screening colonoscopy that becomes therapeutic remains a top AGA priority. Most of the offices that Dr. Crowe met with were cosponsors of the legislation, the Removing Barriers to Colorectal Cancer Screening Act (HR 1017/S.479), that would waive coinsurance payment regardless of the screening outcome. Dr. Crowe shared her experience with patients and the financial burden this places on beneficiaries who need to be screened. Rep. Raul Ruiz, D-CA, and Rep. Scott Peters, D-CA, both members of the House Energy and Commerce Committee and supporters of the bill, will continue to advocate that the bill receive a hearing this year to help move it through Congress. The bill continues to have wide bipartisan support. Read more about the issue and how you can explain it to your patients.
Step therapy
More and more patients are being subject to step therapy protocols, also known as “fail first” under which they are required to try and fail sometimes two or three therapies before receiving coverage of the initial therapy recommended by their physician. With the emergence of new biologics to treat diseases like inflammatory bowel disease, more and more digestive disease patients are being subject to these protocols, which can have adverse effects on their health. Restoring the Patient’s Voice Act (HR 2077) would provide patients and providers with a fair and equitable appeals process when step therapy has been imposed and provides common sense exceptions for the provider to appeal. Dr. Crowe spoke of the impact this policy is having on digestive disease patients and the burden it puts on physician practices that have to take time away from patients to navigate the convoluted insurance appeals process. We are hopeful that many of the offices that we met with will support HR 2077. Read more about the issue.
Food is Medicine Working Group
Dr. Crowe also had a productive meeting with Rep. Jim McGovern’s, D-MA, office and learned more about the recently created Food is Medicine Working Group that he has initiated. McGovern is the Ranking Member of the Agriculture Committee’s Subcommittee on Nutrition which is responsible for our nation’s nutrition guidelines and the Supplemental Nutrition Assistance Program. The Working Group will focus on costs related to hunger and the importance of nutrition in treating chronic illness and disease. AGA looks forward to working with McGovern and members of the Working Group on this bipartisan initiative.
Capitol Hill needs to hear the voice of GI
In conjunction with Dr. Crowe’s visit, AGA launched a Virtual Advocacy Day to encourage members to contact their legislators in support of the issues that Dr. Crowe was advocating during her meetings. We thank those members who took time out of their schedules to take action.
Headlines from the 2018 Gastrointestinal Cancers Symposium
The 2018 Gastrointestinal Cancers Symposium took place Jan. 18-20, 2018, in San Francisco. During the meeting, investigators presented groundbreaking research designed to improve the diagnosis and treatment of GI cancers. Here are some of the most noteworthy headlines from the 2018 meeting.
Promising Results Using Liquid Biopsy to Improve CRC Early Detection
Researchers in Taiwan developed a screening test for early colorectal cancer (CRC) detection that requires a simple blood draw to assess for circulating tumor cells in the blood. The test demonstrates 88% accuracy to detect all stages of colorectal illness, including precancerous lesions. If validated and made commercially available, this test could be readily integrated into a patient’s routine physical exam, thereby increasing CRC screening compliance.
CELESTIAL Results May Lead to Cabozantinib Approval in Second-Line HCC
The phase III CELESTIAL trial met its primary endpoint by demonstrating a survival advantage with cabozantinib in patients with advanced hepatocellular carcinoma (HCC) that progressed following prior systemic therapy. Other outcomes included improvements in progression-free survival and objective response rate, as well as an acceptable safety profile, thus positioning cabozantinib for potential approval in the second-line setting in HCC.
RAINFALL Meets Primary Endpoint, But Ramucirumab Will Not Be Pursued for a First-Line Indication in G-GEJ Cancer
Results of the global, randomized, double-blind, placebo-controlled, phase III RAINFALL trial established the statistical benefit of ramucirumab, a monoclonal antibody targeting VEGFR-2, added to standard chemotherapy for patients with previously untreated metastatic gastric or gastroesophageal junction (G-GEJ) adenocarcinoma. The findings revealed a significant 25% reduction in the risk of disease progression or death for the primary endpoint of progression-free survival (PFS). However, the reduction corresponded to only a 9-day improvement in median PFS, so the clinical benefit of frontline ramucirumab is debatable.
The Gastrointestinal Cancers Symposium is cosponsored by AGA, the American Society of Clinical Oncology (ASCO), the American Society for Radiation Oncology (ASTRO) and the Society of Surgical Oncology (SSO).
More news from the 2018 Gastrointestinal Cancers Symposium is available at gicasym.org/daily-news.
The 2018 Gastrointestinal Cancers Symposium took place Jan. 18-20, 2018, in San Francisco. During the meeting, investigators presented groundbreaking research designed to improve the diagnosis and treatment of GI cancers. Here are some of the most noteworthy headlines from the 2018 meeting.
Promising Results Using Liquid Biopsy to Improve CRC Early Detection
Researchers in Taiwan developed a screening test for early colorectal cancer (CRC) detection that requires a simple blood draw to assess for circulating tumor cells in the blood. The test demonstrates 88% accuracy to detect all stages of colorectal illness, including precancerous lesions. If validated and made commercially available, this test could be readily integrated into a patient’s routine physical exam, thereby increasing CRC screening compliance.
CELESTIAL Results May Lead to Cabozantinib Approval in Second-Line HCC
The phase III CELESTIAL trial met its primary endpoint by demonstrating a survival advantage with cabozantinib in patients with advanced hepatocellular carcinoma (HCC) that progressed following prior systemic therapy. Other outcomes included improvements in progression-free survival and objective response rate, as well as an acceptable safety profile, thus positioning cabozantinib for potential approval in the second-line setting in HCC.
RAINFALL Meets Primary Endpoint, But Ramucirumab Will Not Be Pursued for a First-Line Indication in G-GEJ Cancer
Results of the global, randomized, double-blind, placebo-controlled, phase III RAINFALL trial established the statistical benefit of ramucirumab, a monoclonal antibody targeting VEGFR-2, added to standard chemotherapy for patients with previously untreated metastatic gastric or gastroesophageal junction (G-GEJ) adenocarcinoma. The findings revealed a significant 25% reduction in the risk of disease progression or death for the primary endpoint of progression-free survival (PFS). However, the reduction corresponded to only a 9-day improvement in median PFS, so the clinical benefit of frontline ramucirumab is debatable.
The Gastrointestinal Cancers Symposium is cosponsored by AGA, the American Society of Clinical Oncology (ASCO), the American Society for Radiation Oncology (ASTRO) and the Society of Surgical Oncology (SSO).
More news from the 2018 Gastrointestinal Cancers Symposium is available at gicasym.org/daily-news.
The 2018 Gastrointestinal Cancers Symposium took place Jan. 18-20, 2018, in San Francisco. During the meeting, investigators presented groundbreaking research designed to improve the diagnosis and treatment of GI cancers. Here are some of the most noteworthy headlines from the 2018 meeting.
Promising Results Using Liquid Biopsy to Improve CRC Early Detection
Researchers in Taiwan developed a screening test for early colorectal cancer (CRC) detection that requires a simple blood draw to assess for circulating tumor cells in the blood. The test demonstrates 88% accuracy to detect all stages of colorectal illness, including precancerous lesions. If validated and made commercially available, this test could be readily integrated into a patient’s routine physical exam, thereby increasing CRC screening compliance.
CELESTIAL Results May Lead to Cabozantinib Approval in Second-Line HCC
The phase III CELESTIAL trial met its primary endpoint by demonstrating a survival advantage with cabozantinib in patients with advanced hepatocellular carcinoma (HCC) that progressed following prior systemic therapy. Other outcomes included improvements in progression-free survival and objective response rate, as well as an acceptable safety profile, thus positioning cabozantinib for potential approval in the second-line setting in HCC.
RAINFALL Meets Primary Endpoint, But Ramucirumab Will Not Be Pursued for a First-Line Indication in G-GEJ Cancer
Results of the global, randomized, double-blind, placebo-controlled, phase III RAINFALL trial established the statistical benefit of ramucirumab, a monoclonal antibody targeting VEGFR-2, added to standard chemotherapy for patients with previously untreated metastatic gastric or gastroesophageal junction (G-GEJ) adenocarcinoma. The findings revealed a significant 25% reduction in the risk of disease progression or death for the primary endpoint of progression-free survival (PFS). However, the reduction corresponded to only a 9-day improvement in median PFS, so the clinical benefit of frontline ramucirumab is debatable.
The Gastrointestinal Cancers Symposium is cosponsored by AGA, the American Society of Clinical Oncology (ASCO), the American Society for Radiation Oncology (ASTRO) and the Society of Surgical Oncology (SSO).
More news from the 2018 Gastrointestinal Cancers Symposium is available at gicasym.org/daily-news.
Tamibarotene shows strong results in high-risk APL patients
ATLANTA – Maintenance therapy with the synthetic retinoid tamibarotene is more effective than all-trans retinoic acid (ATRA), for decreasing the relapse rate in patients with acute promyelocytic leukemia (APL) – a subtype of acute myeloid leukemia, according to 7-year findings from the JALSG-APL204 randomized controlled trial.
The relapse-free survival findings were particularly pronounced among high-risk patients with leukocyte counts of at least 10,000 per microliter, Akihiro Takeshita, MD, PhD, reported at the annual meeting of the American Society of Hematology.
“These results could lead to a new strategy for the treatment of high-risk patients, which is one of the recent priority issues in the treatment of APL,” said Dr. Takeshita of Hamamatsu (Japan) University.
Of 344 eligible patients aged 15-70 years with newly diagnosed APL and documented cytogenetic and/or molecular evidence of chromosomal translocation t(15;17) or PML/RAR-alpha gene expression, 269 entered the maintenance phase of the study after completing three courses of consolidation therapy and were assigned to receive ATRA or tamibarotene. At a mean follow-up of 7 years, the relapse-free survival rate was 84% in the 135 patients in the ATRA arm, compared with 93% among the 134 patients in the tamibarotene arm.
The difference between the groups was statistically significant, but an even greater difference was seen when the analysis was restricted to 52 high-risk patients with an initial leukocyte count of at least 10,000 per microliter (62% vs. 89%).
Both treatments were generally well tolerated, Dr. Takeshita reported.
Study subjects received ATRA at a daily dose of 45 mg/m2 for remission induction. Once complete remission was achieved, they received chemotherapy based on their initial leukocyte and blast count in the peripheral blood. Those who achieved molecular remission after consolidation chemotherapy were included in the current maintenance phase of the study. During this phase, ATRA was given at a daily dose of 45 mg/m2 divided into 3 doses for 14 days, and tamibarotene was given at a daily dose of 6 mg/m2 divided into 2 doses for 14 days. Each cycle of treatment was repeated every 3 months for 2 years.
Adverse events included secondary hematopoietic disorders in 12 cases, malignancies in 9 cases, and late cardiac complications of grade 3 or higher in 5 cases, but no significant difference in the rates of these events was seen between the two treatment groups, Dr. Takeshita noted.
Tamibarotene was studied in this trial because, compared with ATRA, it has been shown to have about a 10-fold increase in potency for inducing in vitro differentiation of NB-4 cells, enhanced chemical stability, and low affinity for cellular RA-binding protein.
“The clinical efficacy of tamibarotene for the treatment of APL has also been reported,” Dr. Takeshita added.
In the initial phases of the trial, no difference was seen between ATRA and tamibarotene with respect to 4-year relapse-free survival, but there did appear to be improved efficacy with tamibarotene in high-risk patients, which warranted further investigation, he said.
The current findings demonstrate the efficacy of tamibarotene vs. ATRA for decreasing the relapse rate at the 7-year observation point, and confirm the benefit in high-risk patients that was seen in earlier analyses, he concluded.
Dr. Takeshita reported receiving research funding from Chugai Pharmaceutical, Astellas Pharma, Pfizer Japan, and Takeda Pharmaceutical.
SOURCE: Takeshita A et al., ASH 2017, abstract 642.
ATLANTA – Maintenance therapy with the synthetic retinoid tamibarotene is more effective than all-trans retinoic acid (ATRA), for decreasing the relapse rate in patients with acute promyelocytic leukemia (APL) – a subtype of acute myeloid leukemia, according to 7-year findings from the JALSG-APL204 randomized controlled trial.
The relapse-free survival findings were particularly pronounced among high-risk patients with leukocyte counts of at least 10,000 per microliter, Akihiro Takeshita, MD, PhD, reported at the annual meeting of the American Society of Hematology.
“These results could lead to a new strategy for the treatment of high-risk patients, which is one of the recent priority issues in the treatment of APL,” said Dr. Takeshita of Hamamatsu (Japan) University.
Of 344 eligible patients aged 15-70 years with newly diagnosed APL and documented cytogenetic and/or molecular evidence of chromosomal translocation t(15;17) or PML/RAR-alpha gene expression, 269 entered the maintenance phase of the study after completing three courses of consolidation therapy and were assigned to receive ATRA or tamibarotene. At a mean follow-up of 7 years, the relapse-free survival rate was 84% in the 135 patients in the ATRA arm, compared with 93% among the 134 patients in the tamibarotene arm.
The difference between the groups was statistically significant, but an even greater difference was seen when the analysis was restricted to 52 high-risk patients with an initial leukocyte count of at least 10,000 per microliter (62% vs. 89%).
Both treatments were generally well tolerated, Dr. Takeshita reported.
Study subjects received ATRA at a daily dose of 45 mg/m2 for remission induction. Once complete remission was achieved, they received chemotherapy based on their initial leukocyte and blast count in the peripheral blood. Those who achieved molecular remission after consolidation chemotherapy were included in the current maintenance phase of the study. During this phase, ATRA was given at a daily dose of 45 mg/m2 divided into 3 doses for 14 days, and tamibarotene was given at a daily dose of 6 mg/m2 divided into 2 doses for 14 days. Each cycle of treatment was repeated every 3 months for 2 years.
Adverse events included secondary hematopoietic disorders in 12 cases, malignancies in 9 cases, and late cardiac complications of grade 3 or higher in 5 cases, but no significant difference in the rates of these events was seen between the two treatment groups, Dr. Takeshita noted.
Tamibarotene was studied in this trial because, compared with ATRA, it has been shown to have about a 10-fold increase in potency for inducing in vitro differentiation of NB-4 cells, enhanced chemical stability, and low affinity for cellular RA-binding protein.
“The clinical efficacy of tamibarotene for the treatment of APL has also been reported,” Dr. Takeshita added.
In the initial phases of the trial, no difference was seen between ATRA and tamibarotene with respect to 4-year relapse-free survival, but there did appear to be improved efficacy with tamibarotene in high-risk patients, which warranted further investigation, he said.
The current findings demonstrate the efficacy of tamibarotene vs. ATRA for decreasing the relapse rate at the 7-year observation point, and confirm the benefit in high-risk patients that was seen in earlier analyses, he concluded.
Dr. Takeshita reported receiving research funding from Chugai Pharmaceutical, Astellas Pharma, Pfizer Japan, and Takeda Pharmaceutical.
SOURCE: Takeshita A et al., ASH 2017, abstract 642.
ATLANTA – Maintenance therapy with the synthetic retinoid tamibarotene is more effective than all-trans retinoic acid (ATRA), for decreasing the relapse rate in patients with acute promyelocytic leukemia (APL) – a subtype of acute myeloid leukemia, according to 7-year findings from the JALSG-APL204 randomized controlled trial.
The relapse-free survival findings were particularly pronounced among high-risk patients with leukocyte counts of at least 10,000 per microliter, Akihiro Takeshita, MD, PhD, reported at the annual meeting of the American Society of Hematology.
“These results could lead to a new strategy for the treatment of high-risk patients, which is one of the recent priority issues in the treatment of APL,” said Dr. Takeshita of Hamamatsu (Japan) University.
Of 344 eligible patients aged 15-70 years with newly diagnosed APL and documented cytogenetic and/or molecular evidence of chromosomal translocation t(15;17) or PML/RAR-alpha gene expression, 269 entered the maintenance phase of the study after completing three courses of consolidation therapy and were assigned to receive ATRA or tamibarotene. At a mean follow-up of 7 years, the relapse-free survival rate was 84% in the 135 patients in the ATRA arm, compared with 93% among the 134 patients in the tamibarotene arm.
The difference between the groups was statistically significant, but an even greater difference was seen when the analysis was restricted to 52 high-risk patients with an initial leukocyte count of at least 10,000 per microliter (62% vs. 89%).
Both treatments were generally well tolerated, Dr. Takeshita reported.
Study subjects received ATRA at a daily dose of 45 mg/m2 for remission induction. Once complete remission was achieved, they received chemotherapy based on their initial leukocyte and blast count in the peripheral blood. Those who achieved molecular remission after consolidation chemotherapy were included in the current maintenance phase of the study. During this phase, ATRA was given at a daily dose of 45 mg/m2 divided into 3 doses for 14 days, and tamibarotene was given at a daily dose of 6 mg/m2 divided into 2 doses for 14 days. Each cycle of treatment was repeated every 3 months for 2 years.
Adverse events included secondary hematopoietic disorders in 12 cases, malignancies in 9 cases, and late cardiac complications of grade 3 or higher in 5 cases, but no significant difference in the rates of these events was seen between the two treatment groups, Dr. Takeshita noted.
Tamibarotene was studied in this trial because, compared with ATRA, it has been shown to have about a 10-fold increase in potency for inducing in vitro differentiation of NB-4 cells, enhanced chemical stability, and low affinity for cellular RA-binding protein.
“The clinical efficacy of tamibarotene for the treatment of APL has also been reported,” Dr. Takeshita added.
In the initial phases of the trial, no difference was seen between ATRA and tamibarotene with respect to 4-year relapse-free survival, but there did appear to be improved efficacy with tamibarotene in high-risk patients, which warranted further investigation, he said.
The current findings demonstrate the efficacy of tamibarotene vs. ATRA for decreasing the relapse rate at the 7-year observation point, and confirm the benefit in high-risk patients that was seen in earlier analyses, he concluded.
Dr. Takeshita reported receiving research funding from Chugai Pharmaceutical, Astellas Pharma, Pfizer Japan, and Takeda Pharmaceutical.
SOURCE: Takeshita A et al., ASH 2017, abstract 642.
REPORTING FROM ASH 2017
Key clinical point:
Major finding: The 7-year relapse-free survival was 62% vs. 89% with ATRA vs. tamibarotene in high-risk patients.
Study details: Long-term maintenance results in 344 patients from a randomized controlled trial.
Disclosures: Dr. Takeshita reported receiving research funding from Chugai Pharmaceutical, Astellas Pharma, Pfizer Japan, and Takeda Pharmaceutical.
Source: Takeshita A et al. ASH 2017, abstract 642.