Only some genes count in breast cancer panels

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Some genetic variants included in commercially available gene panels for breast cancer susceptibility matter a great deal, while others appear to be irrelevant or of uncertain significance, investigators suggested.

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Some genetic variants included in commercially available gene panels for breast cancer susceptibility matter a great deal, while others appear to be irrelevant or of uncertain significance, investigators suggested.

 

Some genetic variants included in commercially available gene panels for breast cancer susceptibility matter a great deal, while others appear to be irrelevant or of uncertain significance, investigators suggested.

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FROM JAMA ONCOLOGY

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Key clinical point: Variants in 5 of 16 genes included in breast cancer panels were associated with increased risk.

Major finding: Germline variants in PALB2 were associated with a more than sevenfold greater risk for breast cancer, and four other variants were associated with moderate increases in risk.

Data source: Retrospective case control study of 65,057 women and a validation sample of 38,326 cases and 26,911 controls.

Disclosures: The study was supported by grants from the National Institutes of Health and Breast Cancer Research Foundation, and was sponsored by Ambry Genetics. Six of the coauthors are current or former employees of Ambry. Couch and his colleagues reported no conflicts of interest.

CHEST names Stephen J. Welch as EVP and CEO

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Tue, 10/23/2018 - 16:10

 

The Board of Regents of the American College of Chest Physicians (CHEST) has finalized the appointment of Stephen J. Welch as executive vice president and chief executive officer.

Stephen J. Welch
Stephen J. Welch
Welch had been serving as the interim Executive Vice President and CEO for the Glenview, Illinois–based organization since May 2016. Prior to this appointment, he served in senior staff roles at CHEST for 22 years. One of these roles was publisher and senior vice president of Publications and Digital Content, which includes managing the organization’s flagship scientific journal, CHEST®.

“We appreciate the exceptional performance of Steve, his senior team, and the entire CHEST staff during this transition in executive leadership. We are excited about the opportunity to work with Steve in his new role going forward, as we begin outlining CHEST’s strategic plan for the next 5 years,” said CHEST President Gerard A. Silvestri, MD, MS, FCCP, in a statement.

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The Board of Regents of the American College of Chest Physicians (CHEST) has finalized the appointment of Stephen J. Welch as executive vice president and chief executive officer.

Stephen J. Welch
Stephen J. Welch
Welch had been serving as the interim Executive Vice President and CEO for the Glenview, Illinois–based organization since May 2016. Prior to this appointment, he served in senior staff roles at CHEST for 22 years. One of these roles was publisher and senior vice president of Publications and Digital Content, which includes managing the organization’s flagship scientific journal, CHEST®.

“We appreciate the exceptional performance of Steve, his senior team, and the entire CHEST staff during this transition in executive leadership. We are excited about the opportunity to work with Steve in his new role going forward, as we begin outlining CHEST’s strategic plan for the next 5 years,” said CHEST President Gerard A. Silvestri, MD, MS, FCCP, in a statement.

 

The Board of Regents of the American College of Chest Physicians (CHEST) has finalized the appointment of Stephen J. Welch as executive vice president and chief executive officer.

Stephen J. Welch
Stephen J. Welch
Welch had been serving as the interim Executive Vice President and CEO for the Glenview, Illinois–based organization since May 2016. Prior to this appointment, he served in senior staff roles at CHEST for 22 years. One of these roles was publisher and senior vice president of Publications and Digital Content, which includes managing the organization’s flagship scientific journal, CHEST®.

“We appreciate the exceptional performance of Steve, his senior team, and the entire CHEST staff during this transition in executive leadership. We are excited about the opportunity to work with Steve in his new role going forward, as we begin outlining CHEST’s strategic plan for the next 5 years,” said CHEST President Gerard A. Silvestri, MD, MS, FCCP, in a statement.

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VIDEO: Innovation fuels push toward therapeutic, safety advances in gastroenterology

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BOSTON – In the next 5 years there is going to be a “huge swing” in gastroenterology to minimally invasive therapies, improved diagnostics, and additional therapeutics and disease management strategies that can be applied on an ambulatory basis and, perhaps, even in the office.

That’s the prediction of Michael L. Kochman, MD, AGAF, of the University of Pennsylvania, Philadelphia, who is the executive committee chair of the American Gastroenterological Association Center for GI Innovation and Technology. In a video interview conducted at the 2017 AGA Tech Summit, sponsored by the center, Dr. Kochman discussed five emerging trends in gastroenterology.

The trends include advances in endoscopic technologies and an expansion of the role of personalized medicine that would allow gastroenterologists to better target medications to individual patients. Office-based physicians will be able to evaluate patient risk for various diseases more easily through the use of stool or blood samples or cheek swabs and, then, steer toward additional tests for those found to be at high risk. Improvements in patient safety also are on the horizon, he said, as new technologies will provide earlier warning signs of failed therapies and potential complications.

Finally, as innovation continues in the area of minimally invasive surgery and transorifice surgery, “we need to look more and more to what we will end up calling organ-preserving surgeries, where we are able to treat just the area of pathology,” he noted. “We will be moving to leaving the patient without lasting scars and remembrances of having to have an organ removed.”

Dr. Kochman disclosed that he serves as a consultant to Boston Scientific and Dark Canyon Labs.

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BOSTON – In the next 5 years there is going to be a “huge swing” in gastroenterology to minimally invasive therapies, improved diagnostics, and additional therapeutics and disease management strategies that can be applied on an ambulatory basis and, perhaps, even in the office.

That’s the prediction of Michael L. Kochman, MD, AGAF, of the University of Pennsylvania, Philadelphia, who is the executive committee chair of the American Gastroenterological Association Center for GI Innovation and Technology. In a video interview conducted at the 2017 AGA Tech Summit, sponsored by the center, Dr. Kochman discussed five emerging trends in gastroenterology.

The trends include advances in endoscopic technologies and an expansion of the role of personalized medicine that would allow gastroenterologists to better target medications to individual patients. Office-based physicians will be able to evaluate patient risk for various diseases more easily through the use of stool or blood samples or cheek swabs and, then, steer toward additional tests for those found to be at high risk. Improvements in patient safety also are on the horizon, he said, as new technologies will provide earlier warning signs of failed therapies and potential complications.

Finally, as innovation continues in the area of minimally invasive surgery and transorifice surgery, “we need to look more and more to what we will end up calling organ-preserving surgeries, where we are able to treat just the area of pathology,” he noted. “We will be moving to leaving the patient without lasting scars and remembrances of having to have an organ removed.”

Dr. Kochman disclosed that he serves as a consultant to Boston Scientific and Dark Canyon Labs.

BOSTON – In the next 5 years there is going to be a “huge swing” in gastroenterology to minimally invasive therapies, improved diagnostics, and additional therapeutics and disease management strategies that can be applied on an ambulatory basis and, perhaps, even in the office.

That’s the prediction of Michael L. Kochman, MD, AGAF, of the University of Pennsylvania, Philadelphia, who is the executive committee chair of the American Gastroenterological Association Center for GI Innovation and Technology. In a video interview conducted at the 2017 AGA Tech Summit, sponsored by the center, Dr. Kochman discussed five emerging trends in gastroenterology.

The trends include advances in endoscopic technologies and an expansion of the role of personalized medicine that would allow gastroenterologists to better target medications to individual patients. Office-based physicians will be able to evaluate patient risk for various diseases more easily through the use of stool or blood samples or cheek swabs and, then, steer toward additional tests for those found to be at high risk. Improvements in patient safety also are on the horizon, he said, as new technologies will provide earlier warning signs of failed therapies and potential complications.

Finally, as innovation continues in the area of minimally invasive surgery and transorifice surgery, “we need to look more and more to what we will end up calling organ-preserving surgeries, where we are able to treat just the area of pathology,” he noted. “We will be moving to leaving the patient without lasting scars and remembrances of having to have an organ removed.”

Dr. Kochman disclosed that he serves as a consultant to Boston Scientific and Dark Canyon Labs.

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FROM THE 2017 AGA TECH SUMMIT

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VIDEO: Five ‘revolutionary’ market forces poised to impact medical practice

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– Five “revolutionary” forces in medicine are going to impact patients and physicians, two health care consultants say, and the federal government will act either as a brake or an accelerator on those forces.

In a video interview conducted during the 2017 AGA Tech Summit, Patrick D. Pilch, managing director and national healthcare advisory leader at the BDO Center for Healthcare Excellence and Innovation, New York, and his colleague David B. Friend, MD, MBA, managing director and chief medical officer at the center, discussed these market forces and their potential effects on the practice of medicine.

One such force is the convergence of molecular biology and computer science, which will lead to the creation of new medical devices that use software and will require Food and Drug Administration approval, including devices that can remotely monitor patient compliance with taking medication. Some health care apps also would need FDA approval.

“Increasingly, health care will be mobile,” Dr. Friend continued. “The concept of the right care at the right place at the right time at the right cost is going to increase, and the ability to deliver this in the mobile setting is going to be very powerful.”

A change in who bears financial risk is another important driver. Insurance companies are decreasing their risk, Dr. Friend said, while health care providers and health systems are bearing more financial risk as they increasingly accept bundled payments and value-based payments. Physicians will need to understand and be prepared for this shift in risk, he warned.

Dr. Friend also noted a move toward an integrated supply chain model that will have “profound implications” throughout the delivery process and a movement of care away from the hospital to a nonhospital setting such as home care or skilled nursing facilities.

The summit was sponsored by the AGA Center for GI Innovation and Technology.

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– Five “revolutionary” forces in medicine are going to impact patients and physicians, two health care consultants say, and the federal government will act either as a brake or an accelerator on those forces.

In a video interview conducted during the 2017 AGA Tech Summit, Patrick D. Pilch, managing director and national healthcare advisory leader at the BDO Center for Healthcare Excellence and Innovation, New York, and his colleague David B. Friend, MD, MBA, managing director and chief medical officer at the center, discussed these market forces and their potential effects on the practice of medicine.

One such force is the convergence of molecular biology and computer science, which will lead to the creation of new medical devices that use software and will require Food and Drug Administration approval, including devices that can remotely monitor patient compliance with taking medication. Some health care apps also would need FDA approval.

“Increasingly, health care will be mobile,” Dr. Friend continued. “The concept of the right care at the right place at the right time at the right cost is going to increase, and the ability to deliver this in the mobile setting is going to be very powerful.”

A change in who bears financial risk is another important driver. Insurance companies are decreasing their risk, Dr. Friend said, while health care providers and health systems are bearing more financial risk as they increasingly accept bundled payments and value-based payments. Physicians will need to understand and be prepared for this shift in risk, he warned.

Dr. Friend also noted a move toward an integrated supply chain model that will have “profound implications” throughout the delivery process and a movement of care away from the hospital to a nonhospital setting such as home care or skilled nursing facilities.

The summit was sponsored by the AGA Center for GI Innovation and Technology.

– Five “revolutionary” forces in medicine are going to impact patients and physicians, two health care consultants say, and the federal government will act either as a brake or an accelerator on those forces.

In a video interview conducted during the 2017 AGA Tech Summit, Patrick D. Pilch, managing director and national healthcare advisory leader at the BDO Center for Healthcare Excellence and Innovation, New York, and his colleague David B. Friend, MD, MBA, managing director and chief medical officer at the center, discussed these market forces and their potential effects on the practice of medicine.

One such force is the convergence of molecular biology and computer science, which will lead to the creation of new medical devices that use software and will require Food and Drug Administration approval, including devices that can remotely monitor patient compliance with taking medication. Some health care apps also would need FDA approval.

“Increasingly, health care will be mobile,” Dr. Friend continued. “The concept of the right care at the right place at the right time at the right cost is going to increase, and the ability to deliver this in the mobile setting is going to be very powerful.”

A change in who bears financial risk is another important driver. Insurance companies are decreasing their risk, Dr. Friend said, while health care providers and health systems are bearing more financial risk as they increasingly accept bundled payments and value-based payments. Physicians will need to understand and be prepared for this shift in risk, he warned.

Dr. Friend also noted a move toward an integrated supply chain model that will have “profound implications” throughout the delivery process and a movement of care away from the hospital to a nonhospital setting such as home care or skilled nursing facilities.

The summit was sponsored by the AGA Center for GI Innovation and Technology.

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More Baby Boomers Need HCV Testing

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Wed, 08/22/2018 - 11:09
Eighty-percent of 3.5 million adults with hepatitis C are born between 1945 and 1965, and most of them are not getting tested.

About 3.5 million U.S. adults are chronically infected with hepatitis C (HCV), and 80% of those are baby boomers. As many as 3 out of 4 infected people are not aware of it, according to the CDC, putting them at risk for liver disease, cancer, and death. And most baby boomers aren’t getting tested for the HCV virus.

Between 2013 (when the U.S. Preventive Services Task Force issued a recommendation that all people born between 1945 and 1965 be tested) and 2015, the rate of testing among baby boomers rose only from 12.3% to 13.8%. About 10.5 million of the 76.2 million baby boomers have been tested for HCV, say American Cancer Society researchers who analyzed data from the CDC’s National Health Interview Survey.

Half of Americans identified as ever having had HCV received follow-up testing showing they were still infected, suggesting that even among those who receive an initial antibody test, half may not know for sure whether they still carry the virus.

“Hepatitis C has few noticeable symptoms,” says John Ward, MD, director of the CDC’s Viral Hepatitis Program, and left undiagnosed it threatens the health not only of the person with the virus, but those the disease might be transmitted to. Identifying those who are infected is important, he adds, because new treatments can cure the infection and eliminate the risk of transmission.

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Eighty-percent of 3.5 million adults with hepatitis C are born between 1945 and 1965, and most of them are not getting tested.
Eighty-percent of 3.5 million adults with hepatitis C are born between 1945 and 1965, and most of them are not getting tested.

About 3.5 million U.S. adults are chronically infected with hepatitis C (HCV), and 80% of those are baby boomers. As many as 3 out of 4 infected people are not aware of it, according to the CDC, putting them at risk for liver disease, cancer, and death. And most baby boomers aren’t getting tested for the HCV virus.

Between 2013 (when the U.S. Preventive Services Task Force issued a recommendation that all people born between 1945 and 1965 be tested) and 2015, the rate of testing among baby boomers rose only from 12.3% to 13.8%. About 10.5 million of the 76.2 million baby boomers have been tested for HCV, say American Cancer Society researchers who analyzed data from the CDC’s National Health Interview Survey.

Half of Americans identified as ever having had HCV received follow-up testing showing they were still infected, suggesting that even among those who receive an initial antibody test, half may not know for sure whether they still carry the virus.

“Hepatitis C has few noticeable symptoms,” says John Ward, MD, director of the CDC’s Viral Hepatitis Program, and left undiagnosed it threatens the health not only of the person with the virus, but those the disease might be transmitted to. Identifying those who are infected is important, he adds, because new treatments can cure the infection and eliminate the risk of transmission.

About 3.5 million U.S. adults are chronically infected with hepatitis C (HCV), and 80% of those are baby boomers. As many as 3 out of 4 infected people are not aware of it, according to the CDC, putting them at risk for liver disease, cancer, and death. And most baby boomers aren’t getting tested for the HCV virus.

Between 2013 (when the U.S. Preventive Services Task Force issued a recommendation that all people born between 1945 and 1965 be tested) and 2015, the rate of testing among baby boomers rose only from 12.3% to 13.8%. About 10.5 million of the 76.2 million baby boomers have been tested for HCV, say American Cancer Society researchers who analyzed data from the CDC’s National Health Interview Survey.

Half of Americans identified as ever having had HCV received follow-up testing showing they were still infected, suggesting that even among those who receive an initial antibody test, half may not know for sure whether they still carry the virus.

“Hepatitis C has few noticeable symptoms,” says John Ward, MD, director of the CDC’s Viral Hepatitis Program, and left undiagnosed it threatens the health not only of the person with the virus, but those the disease might be transmitted to. Identifying those who are infected is important, he adds, because new treatments can cure the infection and eliminate the risk of transmission.

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Study supports use of tPA in stroke patients with SCD

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Vials and a syringe

A new study suggests that having sickle cell disease (SCD) should not prevent patients from receiving tissue plasminogen activator (tPA) to treat ischemic stroke if they otherwise qualify for the treatment.

Researchers compared outcomes of tPA treatment in stroke patients with and without SCD and found no significant differences between the groups with regard to serious complications, length of hospital stay, or in-hospital mortality.

“Having sickle cell disease did not adversely affect any of the indicators we measured,” said Robert J. Adams, MD, of the Medical University of South Carolina in Charleston.

The SCD patients did have a higher rate of intracranial hemorrhage (ICH) than patients without SCD, although the rate was not significantly higher. Still, the researchers said further study is needed to look more closely at this outcome.

Dr Adams and his colleagues reported their findings in the journal Stroke.

The team noted that use of tPA has never been contraindicated in SCD, but guidelines recommend acute exchange transfusion for stroke in SCD, rather than tPA.

To gain more insight into the effects of tPA in patients with SCD, the researchers analyzed in-hospital data compiled by the quality improvement program Get With The Guidelines – Stroke.

The data included 2,016,652 stroke patients seen at 1952 participating US hospitals between January 2008 and March 2015. From these patients, the researchers identified 832 with SCD and 3328 age-, sex-, and race-matched controls.

There was no significant difference between the 2 cohorts in the rate of tPA use—8.2% for SCD patients and 9.4% for controls (P=0.3024).

Likewise, there was no significant difference in the timeliness of tPA administration. The median door-to-needle time was 73 minutes for SCD patients and 79 minutes for controls (P=0.3891).

Among patients who received tPA, there was no significant difference in the overall rate of serious complications, which occurred in 6.6% of the SCD patients and 6.0% of controls (P=0.7732).

Serious complications included symptomatic ICH, which occurred in 4.9% of the SCD patients and 3.2% of controls who received tPA (P= 0.4502).

Although this difference was not significant, the researchers said additional studies are needed to track the ICH rate in SCD patients receiving tPA.

The researchers also calculated the odds ratios (ORs) for various outcomes in tPA-treated SCD patients compared to controls.

In an analysis adjusted for multiple covariates, the OR for in-hospital mortality was 1.21 for SCD patients (P=0.4150), the OR for being discharged home was 0.90 (P=0.2686), and the OR for having a hospital stay lasting beyond 4 days was 1.15 (P=0.1151).

“People with sickle cell disease and an acute stroke who would otherwise qualify for tPA did not have worse outcomes than stroke patients who did not have sickle cell disease,” Dr Adams noted.

He and his colleagues said these findings suggest tPA is safe for patients with SCD and could potentially be used as a complementary therapy to red blood cell exchange, the current guideline-recommended frontline therapy for ischemic stroke in patients with SCD.

“These findings suggest that a future randomized trial that compares using red blood cell exchange alone versus combination therapy with tPA and red blood cell exchange should be undertaken to evaluate the outcomes of [ischemic stroke] in patients with sickle cell disease,” said study author Julie Kanter, MD, of the Medical University of South Carolina in Charleston.

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Vials and a syringe

A new study suggests that having sickle cell disease (SCD) should not prevent patients from receiving tissue plasminogen activator (tPA) to treat ischemic stroke if they otherwise qualify for the treatment.

Researchers compared outcomes of tPA treatment in stroke patients with and without SCD and found no significant differences between the groups with regard to serious complications, length of hospital stay, or in-hospital mortality.

“Having sickle cell disease did not adversely affect any of the indicators we measured,” said Robert J. Adams, MD, of the Medical University of South Carolina in Charleston.

The SCD patients did have a higher rate of intracranial hemorrhage (ICH) than patients without SCD, although the rate was not significantly higher. Still, the researchers said further study is needed to look more closely at this outcome.

Dr Adams and his colleagues reported their findings in the journal Stroke.

The team noted that use of tPA has never been contraindicated in SCD, but guidelines recommend acute exchange transfusion for stroke in SCD, rather than tPA.

To gain more insight into the effects of tPA in patients with SCD, the researchers analyzed in-hospital data compiled by the quality improvement program Get With The Guidelines – Stroke.

The data included 2,016,652 stroke patients seen at 1952 participating US hospitals between January 2008 and March 2015. From these patients, the researchers identified 832 with SCD and 3328 age-, sex-, and race-matched controls.

There was no significant difference between the 2 cohorts in the rate of tPA use—8.2% for SCD patients and 9.4% for controls (P=0.3024).

Likewise, there was no significant difference in the timeliness of tPA administration. The median door-to-needle time was 73 minutes for SCD patients and 79 minutes for controls (P=0.3891).

Among patients who received tPA, there was no significant difference in the overall rate of serious complications, which occurred in 6.6% of the SCD patients and 6.0% of controls (P=0.7732).

Serious complications included symptomatic ICH, which occurred in 4.9% of the SCD patients and 3.2% of controls who received tPA (P= 0.4502).

Although this difference was not significant, the researchers said additional studies are needed to track the ICH rate in SCD patients receiving tPA.

The researchers also calculated the odds ratios (ORs) for various outcomes in tPA-treated SCD patients compared to controls.

In an analysis adjusted for multiple covariates, the OR for in-hospital mortality was 1.21 for SCD patients (P=0.4150), the OR for being discharged home was 0.90 (P=0.2686), and the OR for having a hospital stay lasting beyond 4 days was 1.15 (P=0.1151).

“People with sickle cell disease and an acute stroke who would otherwise qualify for tPA did not have worse outcomes than stroke patients who did not have sickle cell disease,” Dr Adams noted.

He and his colleagues said these findings suggest tPA is safe for patients with SCD and could potentially be used as a complementary therapy to red blood cell exchange, the current guideline-recommended frontline therapy for ischemic stroke in patients with SCD.

“These findings suggest that a future randomized trial that compares using red blood cell exchange alone versus combination therapy with tPA and red blood cell exchange should be undertaken to evaluate the outcomes of [ischemic stroke] in patients with sickle cell disease,” said study author Julie Kanter, MD, of the Medical University of South Carolina in Charleston.

Vials and a syringe

A new study suggests that having sickle cell disease (SCD) should not prevent patients from receiving tissue plasminogen activator (tPA) to treat ischemic stroke if they otherwise qualify for the treatment.

Researchers compared outcomes of tPA treatment in stroke patients with and without SCD and found no significant differences between the groups with regard to serious complications, length of hospital stay, or in-hospital mortality.

“Having sickle cell disease did not adversely affect any of the indicators we measured,” said Robert J. Adams, MD, of the Medical University of South Carolina in Charleston.

The SCD patients did have a higher rate of intracranial hemorrhage (ICH) than patients without SCD, although the rate was not significantly higher. Still, the researchers said further study is needed to look more closely at this outcome.

Dr Adams and his colleagues reported their findings in the journal Stroke.

The team noted that use of tPA has never been contraindicated in SCD, but guidelines recommend acute exchange transfusion for stroke in SCD, rather than tPA.

To gain more insight into the effects of tPA in patients with SCD, the researchers analyzed in-hospital data compiled by the quality improvement program Get With The Guidelines – Stroke.

The data included 2,016,652 stroke patients seen at 1952 participating US hospitals between January 2008 and March 2015. From these patients, the researchers identified 832 with SCD and 3328 age-, sex-, and race-matched controls.

There was no significant difference between the 2 cohorts in the rate of tPA use—8.2% for SCD patients and 9.4% for controls (P=0.3024).

Likewise, there was no significant difference in the timeliness of tPA administration. The median door-to-needle time was 73 minutes for SCD patients and 79 minutes for controls (P=0.3891).

Among patients who received tPA, there was no significant difference in the overall rate of serious complications, which occurred in 6.6% of the SCD patients and 6.0% of controls (P=0.7732).

Serious complications included symptomatic ICH, which occurred in 4.9% of the SCD patients and 3.2% of controls who received tPA (P= 0.4502).

Although this difference was not significant, the researchers said additional studies are needed to track the ICH rate in SCD patients receiving tPA.

The researchers also calculated the odds ratios (ORs) for various outcomes in tPA-treated SCD patients compared to controls.

In an analysis adjusted for multiple covariates, the OR for in-hospital mortality was 1.21 for SCD patients (P=0.4150), the OR for being discharged home was 0.90 (P=0.2686), and the OR for having a hospital stay lasting beyond 4 days was 1.15 (P=0.1151).

“People with sickle cell disease and an acute stroke who would otherwise qualify for tPA did not have worse outcomes than stroke patients who did not have sickle cell disease,” Dr Adams noted.

He and his colleagues said these findings suggest tPA is safe for patients with SCD and could potentially be used as a complementary therapy to red blood cell exchange, the current guideline-recommended frontline therapy for ischemic stroke in patients with SCD.

“These findings suggest that a future randomized trial that compares using red blood cell exchange alone versus combination therapy with tPA and red blood cell exchange should be undertaken to evaluate the outcomes of [ischemic stroke] in patients with sickle cell disease,” said study author Julie Kanter, MD, of the Medical University of South Carolina in Charleston.

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Drug granted orphan designation for MM

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multiple myeloma
Micrograph showing

The US Food and Drug Administration (FDA) has granted orphan drug designation for tasquinimod as a treatment for multiple myeloma (MM).

Tasquinimod is an immunomodulatory, anti-metastatic, and anti-angiogenic compound being developed by Active Biotech AB.

The company says tasquinimod works by inhibiting the function of S100A9, a pro-inflammatory protein that is elevated in MM and other malignancies.

S100A9 is believed to aid cancer development by recruiting and activating immune cells such as myeloid-derived suppressor cells.

Active Biotech AB says that, by targeting the S100A9 pathway, tasquinimod interferes with the accumulation and activation of myeloid-derived suppressor cells in the tumor microenvironment, which decreases immune suppression and angiogenesis.

Tasquinimod also inhibits the hypoxic response in the tumor by binding to HDAC4, according to Active Biotech AB.

The company says tasquinimod has produced “robust results” in animal models of MM, and research presented at the AACR Annual Meeting 2015 supports this statement.

Investigators found that tasquinimod reduced tumor growth and improved survival in mouse models of MM. And these effects were associated with reduced angiogenesis in the bone marrow.

Tasquinimod was previously under development as a treatment for prostate cancer, but research suggested the drug did not have a favorable risk-benefit ratio in this patient population.

About orphan designation

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases. This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

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multiple myeloma
Micrograph showing

The US Food and Drug Administration (FDA) has granted orphan drug designation for tasquinimod as a treatment for multiple myeloma (MM).

Tasquinimod is an immunomodulatory, anti-metastatic, and anti-angiogenic compound being developed by Active Biotech AB.

The company says tasquinimod works by inhibiting the function of S100A9, a pro-inflammatory protein that is elevated in MM and other malignancies.

S100A9 is believed to aid cancer development by recruiting and activating immune cells such as myeloid-derived suppressor cells.

Active Biotech AB says that, by targeting the S100A9 pathway, tasquinimod interferes with the accumulation and activation of myeloid-derived suppressor cells in the tumor microenvironment, which decreases immune suppression and angiogenesis.

Tasquinimod also inhibits the hypoxic response in the tumor by binding to HDAC4, according to Active Biotech AB.

The company says tasquinimod has produced “robust results” in animal models of MM, and research presented at the AACR Annual Meeting 2015 supports this statement.

Investigators found that tasquinimod reduced tumor growth and improved survival in mouse models of MM. And these effects were associated with reduced angiogenesis in the bone marrow.

Tasquinimod was previously under development as a treatment for prostate cancer, but research suggested the drug did not have a favorable risk-benefit ratio in this patient population.

About orphan designation

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases. This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

multiple myeloma
Micrograph showing

The US Food and Drug Administration (FDA) has granted orphan drug designation for tasquinimod as a treatment for multiple myeloma (MM).

Tasquinimod is an immunomodulatory, anti-metastatic, and anti-angiogenic compound being developed by Active Biotech AB.

The company says tasquinimod works by inhibiting the function of S100A9, a pro-inflammatory protein that is elevated in MM and other malignancies.

S100A9 is believed to aid cancer development by recruiting and activating immune cells such as myeloid-derived suppressor cells.

Active Biotech AB says that, by targeting the S100A9 pathway, tasquinimod interferes with the accumulation and activation of myeloid-derived suppressor cells in the tumor microenvironment, which decreases immune suppression and angiogenesis.

Tasquinimod also inhibits the hypoxic response in the tumor by binding to HDAC4, according to Active Biotech AB.

The company says tasquinimod has produced “robust results” in animal models of MM, and research presented at the AACR Annual Meeting 2015 supports this statement.

Investigators found that tasquinimod reduced tumor growth and improved survival in mouse models of MM. And these effects were associated with reduced angiogenesis in the bone marrow.

Tasquinimod was previously under development as a treatment for prostate cancer, but research suggested the drug did not have a favorable risk-benefit ratio in this patient population.

About orphan designation

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases. This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

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Topical HDAC inhibitor shows activity in MF

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Topical HDAC inhibitor shows activity in MF

mycosis fungoides
Micrograph showing

Results of a phase 2 trial suggest a topical, skin-directed histone deacetylase (HDAC) inhibitor can elicit responses in patients with early stage mycosis fungoides (MF).

The drug, remetinostat, was designed to be active in the skin but rapidly broken down and inactivated in blood in order to limit the adverse effects associated with systemic exposure to HDAC inhibitors.

The trial included 60 MF patients who were randomized to receive 0.5% remetinostat gel twice daily, 1% remetinostat gel once daily, or 1% remetinostat gel twice daily for between 6 and 12 months.

Results from this trial were recently released by Medivir AB, the company developing remetinostat.

The primary endpoint of the study was the proportion of patients with either a complete or partial confirmed response to therapy, assessed using the Composite Assessment of Index Lesion Severity.

Based on an intent-to-treat analysis, patients receiving the 1% remetinostat gel twice daily arm had the highest proportion of confirmed responses. Eight of 20 patients (40%) responded, which included 1 complete response.

Five of 20 patients (25%) receiving 0.5% remetinostat gel twice daily responded, as did 4 of 20 (20%) patients receiving 1% remetinostat gel once daily. None of these responses were complete responses.

Remetinostat was well-tolerated across all the dose groups, according to Medivir. There were no signs of systemic adverse effects, including those associated with systemic HDAC inhibitors.

Based on these data, Medivir expects to initiate discussions with regulatory authorities with the aim of initiating a phase 3 study later this year, and to present full phase 2 trial data at scientific meetings in the second half of 2017.

“Remetinostat was designed to effectively inhibit HDACs within cutaneous lesions but to be rapidly broken down in the bloodstream, preventing the side effects associated with systemically administered HDAC inhibitors,” said Richard Bethell, Medivir’s chief scientific officer.

“Based on the efficacy and safety data from this phase 2 study, we believe that remetinostat is capable of meeting a very important unmet need in patients with this chronic and poorly treated orphan disease.”

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mycosis fungoides
Micrograph showing

Results of a phase 2 trial suggest a topical, skin-directed histone deacetylase (HDAC) inhibitor can elicit responses in patients with early stage mycosis fungoides (MF).

The drug, remetinostat, was designed to be active in the skin but rapidly broken down and inactivated in blood in order to limit the adverse effects associated with systemic exposure to HDAC inhibitors.

The trial included 60 MF patients who were randomized to receive 0.5% remetinostat gel twice daily, 1% remetinostat gel once daily, or 1% remetinostat gel twice daily for between 6 and 12 months.

Results from this trial were recently released by Medivir AB, the company developing remetinostat.

The primary endpoint of the study was the proportion of patients with either a complete or partial confirmed response to therapy, assessed using the Composite Assessment of Index Lesion Severity.

Based on an intent-to-treat analysis, patients receiving the 1% remetinostat gel twice daily arm had the highest proportion of confirmed responses. Eight of 20 patients (40%) responded, which included 1 complete response.

Five of 20 patients (25%) receiving 0.5% remetinostat gel twice daily responded, as did 4 of 20 (20%) patients receiving 1% remetinostat gel once daily. None of these responses were complete responses.

Remetinostat was well-tolerated across all the dose groups, according to Medivir. There were no signs of systemic adverse effects, including those associated with systemic HDAC inhibitors.

Based on these data, Medivir expects to initiate discussions with regulatory authorities with the aim of initiating a phase 3 study later this year, and to present full phase 2 trial data at scientific meetings in the second half of 2017.

“Remetinostat was designed to effectively inhibit HDACs within cutaneous lesions but to be rapidly broken down in the bloodstream, preventing the side effects associated with systemically administered HDAC inhibitors,” said Richard Bethell, Medivir’s chief scientific officer.

“Based on the efficacy and safety data from this phase 2 study, we believe that remetinostat is capable of meeting a very important unmet need in patients with this chronic and poorly treated orphan disease.”

mycosis fungoides
Micrograph showing

Results of a phase 2 trial suggest a topical, skin-directed histone deacetylase (HDAC) inhibitor can elicit responses in patients with early stage mycosis fungoides (MF).

The drug, remetinostat, was designed to be active in the skin but rapidly broken down and inactivated in blood in order to limit the adverse effects associated with systemic exposure to HDAC inhibitors.

The trial included 60 MF patients who were randomized to receive 0.5% remetinostat gel twice daily, 1% remetinostat gel once daily, or 1% remetinostat gel twice daily for between 6 and 12 months.

Results from this trial were recently released by Medivir AB, the company developing remetinostat.

The primary endpoint of the study was the proportion of patients with either a complete or partial confirmed response to therapy, assessed using the Composite Assessment of Index Lesion Severity.

Based on an intent-to-treat analysis, patients receiving the 1% remetinostat gel twice daily arm had the highest proportion of confirmed responses. Eight of 20 patients (40%) responded, which included 1 complete response.

Five of 20 patients (25%) receiving 0.5% remetinostat gel twice daily responded, as did 4 of 20 (20%) patients receiving 1% remetinostat gel once daily. None of these responses were complete responses.

Remetinostat was well-tolerated across all the dose groups, according to Medivir. There were no signs of systemic adverse effects, including those associated with systemic HDAC inhibitors.

Based on these data, Medivir expects to initiate discussions with regulatory authorities with the aim of initiating a phase 3 study later this year, and to present full phase 2 trial data at scientific meetings in the second half of 2017.

“Remetinostat was designed to effectively inhibit HDACs within cutaneous lesions but to be rapidly broken down in the bloodstream, preventing the side effects associated with systemically administered HDAC inhibitors,” said Richard Bethell, Medivir’s chief scientific officer.

“Based on the efficacy and safety data from this phase 2 study, we believe that remetinostat is capable of meeting a very important unmet need in patients with this chronic and poorly treated orphan disease.”

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Portal allows hemophilia patients to share data with providers

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National Cancer Institute
Doctor using a smartphone Photo by Daniel Sone,

Novo Nordisk has launched a web-based portal that allows hemophilia patients to share real-time data on their treatment and bleeding events with their healthcare providers.

The portal, HemaGo™ XChange, is an extension of Novo Nordisk’s HemaGo™ mobile application and website, which were launched in 2012.

Data a patient enters into the HemaGo™ diary can be shared through the HemaGo™ XChange with the patient’s hemophilia treatment network.

Patients may also choose to have these data entered into the American Thrombosis and Hemostasis Network’s (ATHN) national database of bleeding disorder treatment information.

“We developed HemaGo™ XChange to help drive progress in hemophilia management by turning static data into usable information for people with hemophilia, their care teams, and even researchers,” said John Spera, vice-president of biopharmaceuticals marketing at Novo Nordisk Inc.

“With timely information about the daily experiences of patients, including bleeds, healthcare providers can adjust their care to better fit patient lives.”

Patients using HemaGo™ can:

  • Provide information to their healthcare team, including access to treatment and bleed data, in real time through the HemaGo™ XChange web portal
  • Choose to email data directly from the app or website at any time
  • Opt-in through their hemophilia treatment center to have their data integrated into ATHN’s national database of bleeding disorder treatment information. ATHN will use and share these data with hemophilia treatment centers to foster its mission of advancing knowledge and transforming care for the bleeding and clotting disorders community.

Providers invited by patients to connect via the HemaGo™ XChange portal can:

  • View details about treatments, bleeds, and more for multiple patients
  • Track when and how much factor is used; the type of infusion, vial, and dosing amounts; and information about any other medications
  • View data on the type, location, duration, frequency, and status of bleeds
  • View patient and caregiver life experiences, such as pain and health scores or how a bleeding disorder has affected work, school, or other activities
  • Download in-depth reports for all recorded information.

Novo Nordisk does not have access to patient-specific information. The company’s access is restricted to de-identified data in which the individual sources of the data cannot be identified, in accordance with Health Insurance Portability and Accountability Act of 1996 (HIPAA) Privacy and Security Rules.

To download the HemaGo™ app or join the HemaGo™ XChange, visit www.HemaGo.com and www.HGXchange.com. The HemaGo™ app is available for iPhone and Android phones.

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National Cancer Institute
Doctor using a smartphone Photo by Daniel Sone,

Novo Nordisk has launched a web-based portal that allows hemophilia patients to share real-time data on their treatment and bleeding events with their healthcare providers.

The portal, HemaGo™ XChange, is an extension of Novo Nordisk’s HemaGo™ mobile application and website, which were launched in 2012.

Data a patient enters into the HemaGo™ diary can be shared through the HemaGo™ XChange with the patient’s hemophilia treatment network.

Patients may also choose to have these data entered into the American Thrombosis and Hemostasis Network’s (ATHN) national database of bleeding disorder treatment information.

“We developed HemaGo™ XChange to help drive progress in hemophilia management by turning static data into usable information for people with hemophilia, their care teams, and even researchers,” said John Spera, vice-president of biopharmaceuticals marketing at Novo Nordisk Inc.

“With timely information about the daily experiences of patients, including bleeds, healthcare providers can adjust their care to better fit patient lives.”

Patients using HemaGo™ can:

  • Provide information to their healthcare team, including access to treatment and bleed data, in real time through the HemaGo™ XChange web portal
  • Choose to email data directly from the app or website at any time
  • Opt-in through their hemophilia treatment center to have their data integrated into ATHN’s national database of bleeding disorder treatment information. ATHN will use and share these data with hemophilia treatment centers to foster its mission of advancing knowledge and transforming care for the bleeding and clotting disorders community.

Providers invited by patients to connect via the HemaGo™ XChange portal can:

  • View details about treatments, bleeds, and more for multiple patients
  • Track when and how much factor is used; the type of infusion, vial, and dosing amounts; and information about any other medications
  • View data on the type, location, duration, frequency, and status of bleeds
  • View patient and caregiver life experiences, such as pain and health scores or how a bleeding disorder has affected work, school, or other activities
  • Download in-depth reports for all recorded information.

Novo Nordisk does not have access to patient-specific information. The company’s access is restricted to de-identified data in which the individual sources of the data cannot be identified, in accordance with Health Insurance Portability and Accountability Act of 1996 (HIPAA) Privacy and Security Rules.

To download the HemaGo™ app or join the HemaGo™ XChange, visit www.HemaGo.com and www.HGXchange.com. The HemaGo™ app is available for iPhone and Android phones.

National Cancer Institute
Doctor using a smartphone Photo by Daniel Sone,

Novo Nordisk has launched a web-based portal that allows hemophilia patients to share real-time data on their treatment and bleeding events with their healthcare providers.

The portal, HemaGo™ XChange, is an extension of Novo Nordisk’s HemaGo™ mobile application and website, which were launched in 2012.

Data a patient enters into the HemaGo™ diary can be shared through the HemaGo™ XChange with the patient’s hemophilia treatment network.

Patients may also choose to have these data entered into the American Thrombosis and Hemostasis Network’s (ATHN) national database of bleeding disorder treatment information.

“We developed HemaGo™ XChange to help drive progress in hemophilia management by turning static data into usable information for people with hemophilia, their care teams, and even researchers,” said John Spera, vice-president of biopharmaceuticals marketing at Novo Nordisk Inc.

“With timely information about the daily experiences of patients, including bleeds, healthcare providers can adjust their care to better fit patient lives.”

Patients using HemaGo™ can:

  • Provide information to their healthcare team, including access to treatment and bleed data, in real time through the HemaGo™ XChange web portal
  • Choose to email data directly from the app or website at any time
  • Opt-in through their hemophilia treatment center to have their data integrated into ATHN’s national database of bleeding disorder treatment information. ATHN will use and share these data with hemophilia treatment centers to foster its mission of advancing knowledge and transforming care for the bleeding and clotting disorders community.

Providers invited by patients to connect via the HemaGo™ XChange portal can:

  • View details about treatments, bleeds, and more for multiple patients
  • Track when and how much factor is used; the type of infusion, vial, and dosing amounts; and information about any other medications
  • View data on the type, location, duration, frequency, and status of bleeds
  • View patient and caregiver life experiences, such as pain and health scores or how a bleeding disorder has affected work, school, or other activities
  • Download in-depth reports for all recorded information.

Novo Nordisk does not have access to patient-specific information. The company’s access is restricted to de-identified data in which the individual sources of the data cannot be identified, in accordance with Health Insurance Portability and Accountability Act of 1996 (HIPAA) Privacy and Security Rules.

To download the HemaGo™ app or join the HemaGo™ XChange, visit www.HemaGo.com and www.HGXchange.com. The HemaGo™ app is available for iPhone and Android phones.

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Dry, thickened skin on hand

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The FP asked the patient to show him how he moved about and immediately noticed that the involved area corresponded directly to the part of the hand that pressed upon his cane. He then diagnosed the patient with unilateral hand eczema related to friction.

The FP asked the patient if he would be willing to get a soft glove to wear on his hand while walking. The patient was amenable to this suggestion, but also asked if something could be done for the dry, thickened area that had already built up on his palm.

The FP prescribed ammonium lactate 12% to be applied twice daily, as it is a good moisturizing keratolytic that helps to break down keratin and soften the skin. He also gave the patient a prescription for 0.1% triamcinolone ointment to rub into the affected area at night before going to sleep. The FP recommended not using this during the daytime as it might make the patient’s hand slippery, leading to a fall if he lost his grip on the cane. At a follow-up visit 2 months later, the patient had improved and was very happy with the result.

 

Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Usatine R. Hand eczema. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013:597-602.

To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/

You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com

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The FP asked the patient to show him how he moved about and immediately noticed that the involved area corresponded directly to the part of the hand that pressed upon his cane. He then diagnosed the patient with unilateral hand eczema related to friction.

The FP asked the patient if he would be willing to get a soft glove to wear on his hand while walking. The patient was amenable to this suggestion, but also asked if something could be done for the dry, thickened area that had already built up on his palm.

The FP prescribed ammonium lactate 12% to be applied twice daily, as it is a good moisturizing keratolytic that helps to break down keratin and soften the skin. He also gave the patient a prescription for 0.1% triamcinolone ointment to rub into the affected area at night before going to sleep. The FP recommended not using this during the daytime as it might make the patient’s hand slippery, leading to a fall if he lost his grip on the cane. At a follow-up visit 2 months later, the patient had improved and was very happy with the result.

 

Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Usatine R. Hand eczema. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013:597-602.

To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/

You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com

 

The FP asked the patient to show him how he moved about and immediately noticed that the involved area corresponded directly to the part of the hand that pressed upon his cane. He then diagnosed the patient with unilateral hand eczema related to friction.

The FP asked the patient if he would be willing to get a soft glove to wear on his hand while walking. The patient was amenable to this suggestion, but also asked if something could be done for the dry, thickened area that had already built up on his palm.

The FP prescribed ammonium lactate 12% to be applied twice daily, as it is a good moisturizing keratolytic that helps to break down keratin and soften the skin. He also gave the patient a prescription for 0.1% triamcinolone ointment to rub into the affected area at night before going to sleep. The FP recommended not using this during the daytime as it might make the patient’s hand slippery, leading to a fall if he lost his grip on the cane. At a follow-up visit 2 months later, the patient had improved and was very happy with the result.

 

Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Usatine R. Hand eczema. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013:597-602.

To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/

You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com

Issue
The Journal of Family Practice - 66(4)
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