BOSTON – Bezafibrate was superior to placebo for ameliorating pruritus in patients with chronic cholestatic liver diseases, investigators reported at the annual meeting of the American Association for the Study of Liver Diseases.

Improvements in itch were reported by four times as many patients treated with the peroxisome proliferator-activated receptor (PPAR) agonist, compared with those treated with placebo, according to results of the FITCH (Fibrates for cholestatic ITCH) trial.

That finding from FITCH is very encouraging for patients with this “vexing” clinical issue, which can be highly distressing and is a common feature of cholestatic liver diseases, said Michael R. Charlton, MBBS, FRCP, director of the Transplant Institute and hepatology chief at the University of Chicago.

“It’s generally a misery-making condition,” Dr. Charlton said in a podium discussion of the FITCH study results at the Liver Meeting 2019. “I had a patient tell me that they felt like the subject of Edvard Munch’s ‘Scream’ painting.”

As of this meeting, bezafibrate should be considered superior to placebo for treatment of pruritus in cholangiopathies and should be “considered as first-line treatment” for pruritus in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), added Dr. Charlton, who was not involved in the study.

Investigators in FITCH recruited a total of 74 patients – all enrolled in the Netherlands or Spain – with cholestasis-induced pruritus who reported itch with an intensity of least 5 out of 10 on a visual analogue scale (VAS). Of the 70 patients who completed the trial, 44 had PSC, 24 had PBC, and 2 had secondary sclerosing cholangitis. Patients were randomly allocated to receive bezafibrate 400 mg once daily or placebo for 21 days.

The hypothesis was that PPAR agonist treatment would relieve itch by alleviating hepatobiliary inflammation and reducing formation of a biliary itch factor, according to the investigators, led by Elsemieke de Vries, MD, of the department of gastroenterology and hepatology, Amsterdam University Medical Centers.

“Guideline-approved pharmacological strategies show limited efficacy and can provoke serious side effects,” Dr. de Vries and coauthors said in the published abstract on the study.

The primary study endpoint, a 50% reduction in pruritus VAS score, was achieved in 45% of patients in the bezafibrate treatment arm (17 of 38 patients) versus just 11% in the placebo arm (4 of 36 patients; P = .003), according to updated results presented at the meeting.

The mean VAS score, comparable at baseline, was significantly lower in the bezafibrate group vs. the placebo group at day 21 (P < .001), the results showed.

Authors of the FITCH study reported disclosures related to Intercept Pharmaceuticals, Gilead, Takeda, Tillotts, Pliant, and Dr. Falk GmbH.

SOURCE: de Vries E et al. The Liver Meeting 2019. Abstract 13.

BOSTON – Bezafibrate was superior to placebo for ameliorating pruritus in patients with chronic cholestatic liver diseases, investigators reported at the annual meeting of the American Association for the Study of Liver Diseases.

Improvements in itch were reported by four times as many patients treated with the peroxisome proliferator-activated receptor (PPAR) agonist, compared with those treated with placebo, according to results of the FITCH (Fibrates for cholestatic ITCH) trial.

That finding from FITCH is very encouraging for patients with this “vexing” clinical issue, which can be highly distressing and is a common feature of cholestatic liver diseases, said Michael R. Charlton, MBBS, FRCP, director of the Transplant Institute and hepatology chief at the University of Chicago.

“It’s generally a misery-making condition,” Dr. Charlton said in a podium discussion of the FITCH study results at the Liver Meeting 2019. “I had a patient tell me that they felt like the subject of Edvard Munch’s ‘Scream’ painting.”

As of this meeting, bezafibrate should be considered superior to placebo for treatment of pruritus in cholangiopathies and should be “considered as first-line treatment” for pruritus in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), added Dr. Charlton, who was not involved in the study.

Investigators in FITCH recruited a total of 74 patients – all enrolled in the Netherlands or Spain – with cholestasis-induced pruritus who reported itch with an intensity of least 5 out of 10 on a visual analogue scale (VAS). Of the 70 patients who completed the trial, 44 had PSC, 24 had PBC, and 2 had secondary sclerosing cholangitis. Patients were randomly allocated to receive bezafibrate 400 mg once daily or placebo for 21 days.

The hypothesis was that PPAR agonist treatment would relieve itch by alleviating hepatobiliary inflammation and reducing formation of a biliary itch factor, according to the investigators, led by Elsemieke de Vries, MD, of the department of gastroenterology and hepatology, Amsterdam University Medical Centers.

“Guideline-approved pharmacological strategies show limited efficacy and can provoke serious side effects,” Dr. de Vries and coauthors said in the published abstract on the study.

The primary study endpoint, a 50% reduction in pruritus VAS score, was achieved in 45% of patients in the bezafibrate treatment arm (17 of 38 patients) versus just 11% in the placebo arm (4 of 36 patients; P = .003), according to updated results presented at the meeting.

The mean VAS score, comparable at baseline, was significantly lower in the bezafibrate group vs. the placebo group at day 21 (P < .001), the results showed.

Authors of the FITCH study reported disclosures related to Intercept Pharmaceuticals, Gilead, Takeda, Tillotts, Pliant, and Dr. Falk GmbH.

SOURCE: de Vries E et al. The Liver Meeting 2019. Abstract 13.

BOSTON – Bezafibrate was superior to placebo for ameliorating pruritus in patients with chronic cholestatic liver diseases, investigators reported at the annual meeting of the American Association for the Study of Liver Diseases.

Improvements in itch were reported by four times as many patients treated with the peroxisome proliferator-activated receptor (PPAR) agonist, compared with those treated with placebo, according to results of the FITCH (Fibrates for cholestatic ITCH) trial.

That finding from FITCH is very encouraging for patients with this “vexing” clinical issue, which can be highly distressing and is a common feature of cholestatic liver diseases, said Michael R. Charlton, MBBS, FRCP, director of the Transplant Institute and hepatology chief at the University of Chicago.

“It’s generally a misery-making condition,” Dr. Charlton said in a podium discussion of the FITCH study results at the Liver Meeting 2019. “I had a patient tell me that they felt like the subject of Edvard Munch’s ‘Scream’ painting.”

As of this meeting, bezafibrate should be considered superior to placebo for treatment of pruritus in cholangiopathies and should be “considered as first-line treatment” for pruritus in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), added Dr. Charlton, who was not involved in the study.

Investigators in FITCH recruited a total of 74 patients – all enrolled in the Netherlands or Spain – with cholestasis-induced pruritus who reported itch with an intensity of least 5 out of 10 on a visual analogue scale (VAS). Of the 70 patients who completed the trial, 44 had PSC, 24 had PBC, and 2 had secondary sclerosing cholangitis. Patients were randomly allocated to receive bezafibrate 400 mg once daily or placebo for 21 days.

The hypothesis was that PPAR agonist treatment would relieve itch by alleviating hepatobiliary inflammation and reducing formation of a biliary itch factor, according to the investigators, led by Elsemieke de Vries, MD, of the department of gastroenterology and hepatology, Amsterdam University Medical Centers.

“Guideline-approved pharmacological strategies show limited efficacy and can provoke serious side effects,” Dr. de Vries and coauthors said in the published abstract on the study.

The primary study endpoint, a 50% reduction in pruritus VAS score, was achieved in 45% of patients in the bezafibrate treatment arm (17 of 38 patients) versus just 11% in the placebo arm (4 of 36 patients; P = .003), according to updated results presented at the meeting.

The mean VAS score, comparable at baseline, was significantly lower in the bezafibrate group vs. the placebo group at day 21 (P < .001), the results showed.

Authors of the FITCH study reported disclosures related to Intercept Pharmaceuticals, Gilead, Takeda, Tillotts, Pliant, and Dr. Falk GmbH.

SOURCE: de Vries E et al. The Liver Meeting 2019. Abstract 13.

More than 6,000 veterans die by suicide every year—more than the total number of combat deaths in Iraq and Afghanistan combined.

In recently released Improving the Quality of Mental Health Care for Veterans: Lessons from Rand Research, RAND researchers say the overwhelming message from 10 years of research, including a comprehensive evaluation of the US Department of Veterans Affairs (VA) mental health system and a congressionally mandated analysis of VA health care vs other health care systems, is, We could do more to save the lives of veterans.

RAND research has found that the VA outperforms other health systems on most measures of health care, including mental health care. The report also acknowledges that the VA and US Department of Defense (DoD) have both invested heavily in public-awareness campaigns and efforts to better identify those at risk of suicide, but “the research shows it’s not enough.”

Improving the Quality of Mental Health Care for Veterans has 6 recommendations:

1. Increase the number of highly trained mental health providers within the VA and in private practice. The VA has continued to hire more providers, integrate mental health into primary care settings, and expand the use of telemental health. However, quality varies “considerably” across facilities, the report says, with best practices not universally delivered.

Moreover, fewer than half of all veterans get their care at the VA. Most use local hospitals and health clinics. RAND research has shown those community health providers are often not prepared to address the needs of veterans. Few community providers even ask their patients if they ever served, the researchers say. Recently, the report notes, the VA has taken steps to help private providers serve veterans more effectively, by providing toolkits, training programs, and other resources.

2. Reduce barriers to care by educating veterans about treatment and expanding access to high-quality treatment. The report notes that patients may believe that admitting a mental health problem is a “sign of weakness,” or that they may be skeptical about the effectiveness of treatment.

3. Adopt and enforce appropriate, consistent quality-of-care standards by creating incentives and disincentives that support best practices.

4. Improve monitoring and performance measurement for VA community care programs. According to the report, “Little is known about the timeliness or quality of care that veterans receive through these programs as mandated.”

5. Continue to develop and test new models of care, particularly as new interventions become available and show promise. The report lists examples of evidence-based practices that have proven effective, including narrative exposure therapy for PTSD, mindfulness-based therapies for depression, and behavioral couples therapy for alcohol use disorder.

6. Strengthen the evidence base for understanding the effectiveness of complementary and alternative therapies for mental health conditions.

The research also supports the concept that policies that change the environment and attitudes can reduce and prevent suicides. For instance, a RAND essay advocates policies that promote better sleep and address a “culture of stress.” Policies that promote safe gun storage, encourage health care providers to ask their patients about guns, and remove guns from those at highest risk could help: Nearly 70% of veterans who die by suicide use a firearm. And because sexual assault within the military is a major risk factor for suicide among female veterans, a zero-tolerance policy on sexual assault could make a difference for thousands of service members.

In 2011, RAND researchers published The War Within, a comprehensive look at suicide in the military. One of the driving forces behind the research was senior behavioral scientist Terri Tanielian, whose father, a veteran, committed suicide. Evidence-based treatment not only improves recovery rates but saves money, Tanielian says. In 2008, she and other researchers estimated the 2-year societal costs of postdeployment mental health problems among veterans who had served since the September 11, 2001, attacks at approximately $6.2 billion (in 2007 dollars). If all veterans received high-quality care for depression, posttraumatic stress disorder, and other conditions, those costs could be reduced by $1.2 billion, they found.

The RAND report emphasizes that suicide and mental health issues are a national problem, not just a VA problem. “We can’t think about addressing these issues in the veteran population without thinking about them for the larger American population,” Tanielian says. “We can’t keep pointing a finger at the DoD and the VA. We have to think about this as the national public health crisis that it is.”

More than 6,000 veterans die by suicide every year—more than the total number of combat deaths in Iraq and Afghanistan combined.

In recently released Improving the Quality of Mental Health Care for Veterans: Lessons from Rand Research, RAND researchers say the overwhelming message from 10 years of research, including a comprehensive evaluation of the US Department of Veterans Affairs (VA) mental health system and a congressionally mandated analysis of VA health care vs other health care systems, is, We could do more to save the lives of veterans.

RAND research has found that the VA outperforms other health systems on most measures of health care, including mental health care. The report also acknowledges that the VA and US Department of Defense (DoD) have both invested heavily in public-awareness campaigns and efforts to better identify those at risk of suicide, but “the research shows it’s not enough.”

Improving the Quality of Mental Health Care for Veterans has 6 recommendations:

1. Increase the number of highly trained mental health providers within the VA and in private practice. The VA has continued to hire more providers, integrate mental health into primary care settings, and expand the use of telemental health. However, quality varies “considerably” across facilities, the report says, with best practices not universally delivered.

Moreover, fewer than half of all veterans get their care at the VA. Most use local hospitals and health clinics. RAND research has shown those community health providers are often not prepared to address the needs of veterans. Few community providers even ask their patients if they ever served, the researchers say. Recently, the report notes, the VA has taken steps to help private providers serve veterans more effectively, by providing toolkits, training programs, and other resources.

2. Reduce barriers to care by educating veterans about treatment and expanding access to high-quality treatment. The report notes that patients may believe that admitting a mental health problem is a “sign of weakness,” or that they may be skeptical about the effectiveness of treatment.

3. Adopt and enforce appropriate, consistent quality-of-care standards by creating incentives and disincentives that support best practices.

4. Improve monitoring and performance measurement for VA community care programs. According to the report, “Little is known about the timeliness or quality of care that veterans receive through these programs as mandated.”

5. Continue to develop and test new models of care, particularly as new interventions become available and show promise. The report lists examples of evidence-based practices that have proven effective, including narrative exposure therapy for PTSD, mindfulness-based therapies for depression, and behavioral couples therapy for alcohol use disorder.

6. Strengthen the evidence base for understanding the effectiveness of complementary and alternative therapies for mental health conditions.

The research also supports the concept that policies that change the environment and attitudes can reduce and prevent suicides. For instance, a RAND essay advocates policies that promote better sleep and address a “culture of stress.” Policies that promote safe gun storage, encourage health care providers to ask their patients about guns, and remove guns from those at highest risk could help: Nearly 70% of veterans who die by suicide use a firearm. And because sexual assault within the military is a major risk factor for suicide among female veterans, a zero-tolerance policy on sexual assault could make a difference for thousands of service members.

In 2011, RAND researchers published The War Within, a comprehensive look at suicide in the military. One of the driving forces behind the research was senior behavioral scientist Terri Tanielian, whose father, a veteran, committed suicide. Evidence-based treatment not only improves recovery rates but saves money, Tanielian says. In 2008, she and other researchers estimated the 2-year societal costs of postdeployment mental health problems among veterans who had served since the September 11, 2001, attacks at approximately $6.2 billion (in 2007 dollars). If all veterans received high-quality care for depression, posttraumatic stress disorder, and other conditions, those costs could be reduced by $1.2 billion, they found.

The RAND report emphasizes that suicide and mental health issues are a national problem, not just a VA problem. “We can’t think about addressing these issues in the veteran population without thinking about them for the larger American population,” Tanielian says. “We can’t keep pointing a finger at the DoD and the VA. We have to think about this as the national public health crisis that it is.”

More than 6,000 veterans die by suicide every year—more than the total number of combat deaths in Iraq and Afghanistan combined.

In recently released Improving the Quality of Mental Health Care for Veterans: Lessons from Rand Research, RAND researchers say the overwhelming message from 10 years of research, including a comprehensive evaluation of the US Department of Veterans Affairs (VA) mental health system and a congressionally mandated analysis of VA health care vs other health care systems, is, We could do more to save the lives of veterans.

RAND research has found that the VA outperforms other health systems on most measures of health care, including mental health care. The report also acknowledges that the VA and US Department of Defense (DoD) have both invested heavily in public-awareness campaigns and efforts to better identify those at risk of suicide, but “the research shows it’s not enough.”

Improving the Quality of Mental Health Care for Veterans has 6 recommendations:

1. Increase the number of highly trained mental health providers within the VA and in private practice. The VA has continued to hire more providers, integrate mental health into primary care settings, and expand the use of telemental health. However, quality varies “considerably” across facilities, the report says, with best practices not universally delivered.

Moreover, fewer than half of all veterans get their care at the VA. Most use local hospitals and health clinics. RAND research has shown those community health providers are often not prepared to address the needs of veterans. Few community providers even ask their patients if they ever served, the researchers say. Recently, the report notes, the VA has taken steps to help private providers serve veterans more effectively, by providing toolkits, training programs, and other resources.

2. Reduce barriers to care by educating veterans about treatment and expanding access to high-quality treatment. The report notes that patients may believe that admitting a mental health problem is a “sign of weakness,” or that they may be skeptical about the effectiveness of treatment.

3. Adopt and enforce appropriate, consistent quality-of-care standards by creating incentives and disincentives that support best practices.

4. Improve monitoring and performance measurement for VA community care programs. According to the report, “Little is known about the timeliness or quality of care that veterans receive through these programs as mandated.”

5. Continue to develop and test new models of care, particularly as new interventions become available and show promise. The report lists examples of evidence-based practices that have proven effective, including narrative exposure therapy for PTSD, mindfulness-based therapies for depression, and behavioral couples therapy for alcohol use disorder.

6. Strengthen the evidence base for understanding the effectiveness of complementary and alternative therapies for mental health conditions.

The research also supports the concept that policies that change the environment and attitudes can reduce and prevent suicides. For instance, a RAND essay advocates policies that promote better sleep and address a “culture of stress.” Policies that promote safe gun storage, encourage health care providers to ask their patients about guns, and remove guns from those at highest risk could help: Nearly 70% of veterans who die by suicide use a firearm. And because sexual assault within the military is a major risk factor for suicide among female veterans, a zero-tolerance policy on sexual assault could make a difference for thousands of service members.

In 2011, RAND researchers published The War Within, a comprehensive look at suicide in the military. One of the driving forces behind the research was senior behavioral scientist Terri Tanielian, whose father, a veteran, committed suicide. Evidence-based treatment not only improves recovery rates but saves money, Tanielian says. In 2008, she and other researchers estimated the 2-year societal costs of postdeployment mental health problems among veterans who had served since the September 11, 2001, attacks at approximately $6.2 billion (in 2007 dollars). If all veterans received high-quality care for depression, posttraumatic stress disorder, and other conditions, those costs could be reduced by $1.2 billion, they found.

The RAND report emphasizes that suicide and mental health issues are a national problem, not just a VA problem. “We can’t think about addressing these issues in the veteran population without thinking about them for the larger American population,” Tanielian says. “We can’t keep pointing a finger at the DoD and the VA. We have to think about this as the national public health crisis that it is.”

Three federal agencies have jointly issued a price transparency proposal that would require most employer-based health plans and health insurance issuers to disclose price and cost-sharing information up front.

utah778/Thinkstock

The goal behind the proposal is to give consumers accurate estimates about the out-of-pocket costs they may incur for medical services, giving them the opportunity to shop around for medical treatment.

“Under the status quo, health care prices are about as clear as mud to patients,” Seema Verma, administrator of the Centers for Medicare & Medicaid Services said in a statement, adding that “we are throwing open the shutters and bringing to light the price of care for American consumers. Kept secret, these prices are simply dollar amounts on a ledger; disclosed, they deliver fuel to the engines of competition among hospitals and insurers.”

The “Transparency in Coverage” proposed rule, was released online on Nov. 15 jointly by the Department of Health & Human Services, the Department of Labor, and the Department of the Treasury. If finalized, it would give consumers “real-time, personalized access to cost-sharing information, including an estimate of their cost-sharing liability for all covered health care items and services through an online tool that most group health plans and health insurance issuers would be required to make available to all of their members, and in paper form, at the consumer’s request,” a fact sheet outlining the features of the proposed rule states.

Health plans would also “be required to disclose on a public website their negotiated rates for in-network providers and allowed amounts paid for out-of-network providers,” the fact sheet continues.

The proposal comes as the CMS finalized transparency-related rules in the 2020 update to the hospital outpatient prospective payment system (OPPS). The price transparency portion of the OPPS is scheduled to go into effect on Jan. 1, 2021.

A fact sheet on the OPPS states that each hospital in the United States will be required to “establish (and update) and make public a yearly list of the hospital’s standard charges for items and services provided by the hospital.”

That list must include all standard charges, including gross charges, discounted cash prices, payer-specific negotiated charges, and deidentified minimum and maximum negotiated charges for all hospital items and services, as well as cash prices, payer-specific negotiated charges, and deidentified minimum and maximum negotiated charges for 300 shoppable services (70 identified by CMS and 230 selected by the hospital). A shoppable service is a service that can be scheduled in advance.

“This final rule and the proposed rule will bring forward the transparency we need to finally begin reducing the overall health care costs,” Ms. Verma said. “Today’s rules usher in a new era that upends the status quo to empower patients and put them first.”

America’s Health Insurance Plans said in a statement that it is evaluating the proposal and the final OPPS rule through a lens of three core principles: that consumers deserve transparency about out-of-pocket costs to help them make informed decisions; that transparency should be achieved in a way that encourages, not undermines, competitive negotiations to lower costs; and that public programs and the free market work together to deliver on our commitments to affordable, quality, and value.

“Neither of these rules, together or separately, satisfies these principles,” AHIP stated.

The Federation of American Hospitals is already anticipating a legal challenge to the rules.

“Patients should have readily available and easy-to-understand cost-sharing information when they need to make health care decisions,” FAH President and CEO Chip Kahn said in a statement. “Health care pricing transparency ought to be defined by the right information at the right time. This final regulation on hospital transparency fails to meet the definition of price transparency useful for patients. Instead, it will only result in patient overload of useless information while distorting the competitive market for purchasing hospital care.”

Mr. Kahn said FAH plans “on joining with hospitals to file a legal challenge,” asserting that CMS has exceeded it’s authority with these rules.

[email protected]

Three federal agencies have jointly issued a price transparency proposal that would require most employer-based health plans and health insurance issuers to disclose price and cost-sharing information up front.

utah778/Thinkstock

The goal behind the proposal is to give consumers accurate estimates about the out-of-pocket costs they may incur for medical services, giving them the opportunity to shop around for medical treatment.

“Under the status quo, health care prices are about as clear as mud to patients,” Seema Verma, administrator of the Centers for Medicare & Medicaid Services said in a statement, adding that “we are throwing open the shutters and bringing to light the price of care for American consumers. Kept secret, these prices are simply dollar amounts on a ledger; disclosed, they deliver fuel to the engines of competition among hospitals and insurers.”

The “Transparency in Coverage” proposed rule, was released online on Nov. 15 jointly by the Department of Health & Human Services, the Department of Labor, and the Department of the Treasury. If finalized, it would give consumers “real-time, personalized access to cost-sharing information, including an estimate of their cost-sharing liability for all covered health care items and services through an online tool that most group health plans and health insurance issuers would be required to make available to all of their members, and in paper form, at the consumer’s request,” a fact sheet outlining the features of the proposed rule states.

Health plans would also “be required to disclose on a public website their negotiated rates for in-network providers and allowed amounts paid for out-of-network providers,” the fact sheet continues.

The proposal comes as the CMS finalized transparency-related rules in the 2020 update to the hospital outpatient prospective payment system (OPPS). The price transparency portion of the OPPS is scheduled to go into effect on Jan. 1, 2021.

A fact sheet on the OPPS states that each hospital in the United States will be required to “establish (and update) and make public a yearly list of the hospital’s standard charges for items and services provided by the hospital.”

That list must include all standard charges, including gross charges, discounted cash prices, payer-specific negotiated charges, and deidentified minimum and maximum negotiated charges for all hospital items and services, as well as cash prices, payer-specific negotiated charges, and deidentified minimum and maximum negotiated charges for 300 shoppable services (70 identified by CMS and 230 selected by the hospital). A shoppable service is a service that can be scheduled in advance.

“This final rule and the proposed rule will bring forward the transparency we need to finally begin reducing the overall health care costs,” Ms. Verma said. “Today’s rules usher in a new era that upends the status quo to empower patients and put them first.”

America’s Health Insurance Plans said in a statement that it is evaluating the proposal and the final OPPS rule through a lens of three core principles: that consumers deserve transparency about out-of-pocket costs to help them make informed decisions; that transparency should be achieved in a way that encourages, not undermines, competitive negotiations to lower costs; and that public programs and the free market work together to deliver on our commitments to affordable, quality, and value.

“Neither of these rules, together or separately, satisfies these principles,” AHIP stated.

The Federation of American Hospitals is already anticipating a legal challenge to the rules.

“Patients should have readily available and easy-to-understand cost-sharing information when they need to make health care decisions,” FAH President and CEO Chip Kahn said in a statement. “Health care pricing transparency ought to be defined by the right information at the right time. This final regulation on hospital transparency fails to meet the definition of price transparency useful for patients. Instead, it will only result in patient overload of useless information while distorting the competitive market for purchasing hospital care.”

Mr. Kahn said FAH plans “on joining with hospitals to file a legal challenge,” asserting that CMS has exceeded it’s authority with these rules.

[email protected]

Three federal agencies have jointly issued a price transparency proposal that would require most employer-based health plans and health insurance issuers to disclose price and cost-sharing information up front.

utah778/Thinkstock

The goal behind the proposal is to give consumers accurate estimates about the out-of-pocket costs they may incur for medical services, giving them the opportunity to shop around for medical treatment.

“Under the status quo, health care prices are about as clear as mud to patients,” Seema Verma, administrator of the Centers for Medicare & Medicaid Services said in a statement, adding that “we are throwing open the shutters and bringing to light the price of care for American consumers. Kept secret, these prices are simply dollar amounts on a ledger; disclosed, they deliver fuel to the engines of competition among hospitals and insurers.”

The “Transparency in Coverage” proposed rule, was released online on Nov. 15 jointly by the Department of Health & Human Services, the Department of Labor, and the Department of the Treasury. If finalized, it would give consumers “real-time, personalized access to cost-sharing information, including an estimate of their cost-sharing liability for all covered health care items and services through an online tool that most group health plans and health insurance issuers would be required to make available to all of their members, and in paper form, at the consumer’s request,” a fact sheet outlining the features of the proposed rule states.

Health plans would also “be required to disclose on a public website their negotiated rates for in-network providers and allowed amounts paid for out-of-network providers,” the fact sheet continues.

The proposal comes as the CMS finalized transparency-related rules in the 2020 update to the hospital outpatient prospective payment system (OPPS). The price transparency portion of the OPPS is scheduled to go into effect on Jan. 1, 2021.

A fact sheet on the OPPS states that each hospital in the United States will be required to “establish (and update) and make public a yearly list of the hospital’s standard charges for items and services provided by the hospital.”

That list must include all standard charges, including gross charges, discounted cash prices, payer-specific negotiated charges, and deidentified minimum and maximum negotiated charges for all hospital items and services, as well as cash prices, payer-specific negotiated charges, and deidentified minimum and maximum negotiated charges for 300 shoppable services (70 identified by CMS and 230 selected by the hospital). A shoppable service is a service that can be scheduled in advance.

“This final rule and the proposed rule will bring forward the transparency we need to finally begin reducing the overall health care costs,” Ms. Verma said. “Today’s rules usher in a new era that upends the status quo to empower patients and put them first.”

America’s Health Insurance Plans said in a statement that it is evaluating the proposal and the final OPPS rule through a lens of three core principles: that consumers deserve transparency about out-of-pocket costs to help them make informed decisions; that transparency should be achieved in a way that encourages, not undermines, competitive negotiations to lower costs; and that public programs and the free market work together to deliver on our commitments to affordable, quality, and value.

“Neither of these rules, together or separately, satisfies these principles,” AHIP stated.

The Federation of American Hospitals is already anticipating a legal challenge to the rules.

“Patients should have readily available and easy-to-understand cost-sharing information when they need to make health care decisions,” FAH President and CEO Chip Kahn said in a statement. “Health care pricing transparency ought to be defined by the right information at the right time. This final regulation on hospital transparency fails to meet the definition of price transparency useful for patients. Instead, it will only result in patient overload of useless information while distorting the competitive market for purchasing hospital care.”

Mr. Kahn said FAH plans “on joining with hospitals to file a legal challenge,” asserting that CMS has exceeded it’s authority with these rules.

[email protected]

Screening youth to identify those at risk for suicide is particularly important in medical settings, given the increasing rates of adolescent suicide, and screening can take as little as 20 seconds, according to Lisa Horowitz, PhD, MPH, a staff scientist and clinical psychologist at the National Institute of Mental Health, Bethesda, Md.

Steve Debenport/Getty Images

But clinicians need to use validated screening instruments that are both population specific and site specific, and they need practice guidelines to treat patients screening positive.

Currently, many practitioners use depression screens – such as question #9 on suicide ideation and self harm on the Patient Health Questionnaire for Adolescents (PHQ-A) – to identify suicide risk, but preliminary data suggest these screens often are inadequate, Dr. Horowitz said. Just one question, especially one without precise language, does not appear to identify as many at-risk youths as more direct questions about suicidal thoughts and behaviors.

A Pathways to Clinical Care suicide risk screening work group therefore designed a three-tiered clinical pathway for suicide risk screenings in emergency departments, inpatient care, and outpatient primary care. It begins with the Ask Suicide-Screening Questions (ASQ), which takes about 20 seconds and was specifically developed for pediatric patients in the emergency department and validated in both inpatient and outpatient settings.

Dr Horowitz, also the lead principal investigator for development of the ASQ, currently is leading six National Institute of Mental Health studies to validate and implement the screening tool in medical settings. She explained the three-tiered system during a session on youth suicide screening at the Pediatric Academic Societies annual meeting in Baltimore this year.

If a patient screens positive on the ASQ, a trained clinician should conduct a brief suicide safety assessment (BSSA), which takes approximately 10 minutes, Dr Horowitz said. Those who screen positive on the BSSA should receive the Patient Resource List and then be referred for a full mental health and safety evaluation, which takes about 30 minutes. Resources, such as nurse scripts and parent/guardian flyers, are available at the NIMH website, as well as translations of the ASQ in Arabic, Chinese, Dutch, French, Hebrew, Italian, Japanese, Korean, Portuguese, Russian, Somali, Spanish, and Vietnamese.

Acknowledging the importance of suicide screening

During the same session, John V. Campo, MD, an assistant dean for behavioral health and professor of behavioral medicine and psychiatry at West Virginia University in Morgantown, discussed why suicide risk screening is so crucial in general medical settings. As someone who trained as a pediatrician before crossing over to behavioral health, he acknowledged that primary care physicians already have many priorities to cover in short visits, and that the national answer to most public health problems is to deal with it in primary care.

“Anyone who has done primary care pediatrics understands the challenges involved with screening for anything – particularly when you identify someone who is extensively at risk,” he said.

But suicide has a disproportionately high impact on young populations, and “identifying youth at risk for suicide identifies a group of young people who are at risk for a variety of threats to their health and well-being,” he said.

For youth aged 10-19 years in 2016, suicide was the second leading cause of death behind accidents, according to the Centers for Disease Control and Prevention (Natl Vital Stat Rep. 2018 Jun;67[4]:1-16). In fact, accidents, suicide, and homicide account for three-quarters of deaths among youth aged 10-24 years (Natl Vital Stat Rep. 2019 Jun;68[6]:1-77), yet it’s typically the other 25% that most physicians trained for in residency.

“Suicide kills more kids than cancer, heart disease, infections – all kinds, sepsis, meningitis, pneumonia, influenza, HIV, respiratory conditions. Suicide kills more young people every year than all of that [combined],” Dr. Campo said. “And yet, when you walk through a modern emergency department, we see all these specialized programs for those who present with physical trauma or chest pain or all these other things, but zero specialized mental health services. There’s a disconnect.”

There is some good news in the data, he said. Observational data have shown that suicide rates negatively correlate with indicators of better access to health and medical health services, and researchers increasingly are identifying proven strategies that help prevent suicide in young people – once they have been identified.

But that’s the problem, “and we all know it,” Dr. Campo continued. “Most youth who are at risk for suicide aren’t recognized, and those who are recognized most often are untreated or inadequately treated,” he said. Further, “the best predictor of future behavior is past behavior,” but most adolescents die by suicide on their first attempt.

Again, however, Dr. Campo pivoted to the good news. Data also have shown that most youth who die by suicide had at least one health contact in the previous year, which means there are opportunities for screening and intervention.

The most common risk factor for suicide is having a mental health or substance use condition, present in about 90% of completed suicides and affecting approximately one in five youth. Prevalence is even higher in those with physical health conditions and among those with Medicaid or no insurance (J Child Psychol Psychiatry. 2006 Mar-Apr;47[3-4]372-94).

Yet, “the majority of them have not been treated at all for mental disorder, which seems to be the most important remediable risk factor for suicide, and even fewer are in current treatment at the time of the death,” Dr. Campo said. Suicide also is correlated with a number of other high-risk behaviors or circumstances, such as “vulnerabilities to substance abuse, riding in a car with someone who is intoxicated, carrying a weapon to school, fighting, and meeting criteria for depression” (Pediatrics. 2010 May;125[5]:945-52). Screening for suicide risk therefore allows physicians to identify youth vulnerable to a wide range of risks, conditions, or death.

Overcoming barriers to suicide screening in primary care

Given the high prevalence of suicide and its link to so many other risks for youth, screening in primary care can send the message that suicide screening “really is a part of health care,” Dr. Campo said. Incorporating screening into primary care also can help overcome distrust of behavioral health specialists in the general public and stigma associated with behavioral health disorders.

Primary care screening emphasizes the importance and credibility of mental health and challenges attitudinal barriers to care, he said.

At the same time, however, he acknowledged that providers themselves often are uneasy about addressing behavioral health. Therefore, “having the guideline and the expectation [of suicide risk screening] really drives home the point that this needs to be integrated into the rest of primary care,” he said. “It’s also consistent with the idea of the medical home.” With suicide the second leading cause of death among youth, “if there’s anything that we’re going to be thinking about screening for, one would think suicide would be high on the list.”

In fact, observational evidence has shown that educating and training primary care providers to recognize people with depression or a high risk for suicide can reduce suicide attempts and the suicide rate, Dr. Campo said (JAMA Psychiatry. 2017 Jun 1;74[6]:563-70). It also can help with the mismatch between where at-risk patients are and where behavioral health specialists are. About 90% of behavioral health specialists work only in specialty settings, and only 5% typically work in general medical settings, he said. Yet “most people who are in mental distress or in crisis don’t present in specialty behavioral health settings. They present in general medical settings.”

More data are needed to demonstrate more definitively whether and how much suicide risk screening changes outcomes, but we know a few things, Dr. Campo said, summing up his key points: “We know suicide’s a major source of mortality in youth that’s been relatively neglected in pediatric health care. Second, we know that suicide risk is associated with risk for other important causes of death, for mental disorders, and for alcohol and substance use.

“We know that most suicide decedents are unrecognized prior to the time of death, and those who are recognized often are not treated. We know that the majority of suicide deaths occur on the very first attempt. We also know that we increasingly have treatments, mental disorders that can be identified, and remediable risk factors, and [that at-risk youth] typically present at general medical settings. Beyond that, focusing on the general medical setting has both conceptual and practical advantages as a site for really helping us to detect patients at risk and then managing them.”

No funding was used for the presentations. Dr. Horowitz and Dr. Campo had no relevant financial disclosures.

Screening youth to identify those at risk for suicide is particularly important in medical settings, given the increasing rates of adolescent suicide, and screening can take as little as 20 seconds, according to Lisa Horowitz, PhD, MPH, a staff scientist and clinical psychologist at the National Institute of Mental Health, Bethesda, Md.

Steve Debenport/Getty Images

But clinicians need to use validated screening instruments that are both population specific and site specific, and they need practice guidelines to treat patients screening positive.

Currently, many practitioners use depression screens – such as question #9 on suicide ideation and self harm on the Patient Health Questionnaire for Adolescents (PHQ-A) – to identify suicide risk, but preliminary data suggest these screens often are inadequate, Dr. Horowitz said. Just one question, especially one without precise language, does not appear to identify as many at-risk youths as more direct questions about suicidal thoughts and behaviors.

A Pathways to Clinical Care suicide risk screening work group therefore designed a three-tiered clinical pathway for suicide risk screenings in emergency departments, inpatient care, and outpatient primary care. It begins with the Ask Suicide-Screening Questions (ASQ), which takes about 20 seconds and was specifically developed for pediatric patients in the emergency department and validated in both inpatient and outpatient settings.

Dr Horowitz, also the lead principal investigator for development of the ASQ, currently is leading six National Institute of Mental Health studies to validate and implement the screening tool in medical settings. She explained the three-tiered system during a session on youth suicide screening at the Pediatric Academic Societies annual meeting in Baltimore this year.

If a patient screens positive on the ASQ, a trained clinician should conduct a brief suicide safety assessment (BSSA), which takes approximately 10 minutes, Dr Horowitz said. Those who screen positive on the BSSA should receive the Patient Resource List and then be referred for a full mental health and safety evaluation, which takes about 30 minutes. Resources, such as nurse scripts and parent/guardian flyers, are available at the NIMH website, as well as translations of the ASQ in Arabic, Chinese, Dutch, French, Hebrew, Italian, Japanese, Korean, Portuguese, Russian, Somali, Spanish, and Vietnamese.

Acknowledging the importance of suicide screening

During the same session, John V. Campo, MD, an assistant dean for behavioral health and professor of behavioral medicine and psychiatry at West Virginia University in Morgantown, discussed why suicide risk screening is so crucial in general medical settings. As someone who trained as a pediatrician before crossing over to behavioral health, he acknowledged that primary care physicians already have many priorities to cover in short visits, and that the national answer to most public health problems is to deal with it in primary care.

“Anyone who has done primary care pediatrics understands the challenges involved with screening for anything – particularly when you identify someone who is extensively at risk,” he said.

But suicide has a disproportionately high impact on young populations, and “identifying youth at risk for suicide identifies a group of young people who are at risk for a variety of threats to their health and well-being,” he said.

For youth aged 10-19 years in 2016, suicide was the second leading cause of death behind accidents, according to the Centers for Disease Control and Prevention (Natl Vital Stat Rep. 2018 Jun;67[4]:1-16). In fact, accidents, suicide, and homicide account for three-quarters of deaths among youth aged 10-24 years (Natl Vital Stat Rep. 2019 Jun;68[6]:1-77), yet it’s typically the other 25% that most physicians trained for in residency.

“Suicide kills more kids than cancer, heart disease, infections – all kinds, sepsis, meningitis, pneumonia, influenza, HIV, respiratory conditions. Suicide kills more young people every year than all of that [combined],” Dr. Campo said. “And yet, when you walk through a modern emergency department, we see all these specialized programs for those who present with physical trauma or chest pain or all these other things, but zero specialized mental health services. There’s a disconnect.”

There is some good news in the data, he said. Observational data have shown that suicide rates negatively correlate with indicators of better access to health and medical health services, and researchers increasingly are identifying proven strategies that help prevent suicide in young people – once they have been identified.

But that’s the problem, “and we all know it,” Dr. Campo continued. “Most youth who are at risk for suicide aren’t recognized, and those who are recognized most often are untreated or inadequately treated,” he said. Further, “the best predictor of future behavior is past behavior,” but most adolescents die by suicide on their first attempt.

Again, however, Dr. Campo pivoted to the good news. Data also have shown that most youth who die by suicide had at least one health contact in the previous year, which means there are opportunities for screening and intervention.

The most common risk factor for suicide is having a mental health or substance use condition, present in about 90% of completed suicides and affecting approximately one in five youth. Prevalence is even higher in those with physical health conditions and among those with Medicaid or no insurance (J Child Psychol Psychiatry. 2006 Mar-Apr;47[3-4]372-94).

Yet, “the majority of them have not been treated at all for mental disorder, which seems to be the most important remediable risk factor for suicide, and even fewer are in current treatment at the time of the death,” Dr. Campo said. Suicide also is correlated with a number of other high-risk behaviors or circumstances, such as “vulnerabilities to substance abuse, riding in a car with someone who is intoxicated, carrying a weapon to school, fighting, and meeting criteria for depression” (Pediatrics. 2010 May;125[5]:945-52). Screening for suicide risk therefore allows physicians to identify youth vulnerable to a wide range of risks, conditions, or death.

Overcoming barriers to suicide screening in primary care

Given the high prevalence of suicide and its link to so many other risks for youth, screening in primary care can send the message that suicide screening “really is a part of health care,” Dr. Campo said. Incorporating screening into primary care also can help overcome distrust of behavioral health specialists in the general public and stigma associated with behavioral health disorders.

Primary care screening emphasizes the importance and credibility of mental health and challenges attitudinal barriers to care, he said.

At the same time, however, he acknowledged that providers themselves often are uneasy about addressing behavioral health. Therefore, “having the guideline and the expectation [of suicide risk screening] really drives home the point that this needs to be integrated into the rest of primary care,” he said. “It’s also consistent with the idea of the medical home.” With suicide the second leading cause of death among youth, “if there’s anything that we’re going to be thinking about screening for, one would think suicide would be high on the list.”

In fact, observational evidence has shown that educating and training primary care providers to recognize people with depression or a high risk for suicide can reduce suicide attempts and the suicide rate, Dr. Campo said (JAMA Psychiatry. 2017 Jun 1;74[6]:563-70). It also can help with the mismatch between where at-risk patients are and where behavioral health specialists are. About 90% of behavioral health specialists work only in specialty settings, and only 5% typically work in general medical settings, he said. Yet “most people who are in mental distress or in crisis don’t present in specialty behavioral health settings. They present in general medical settings.”

More data are needed to demonstrate more definitively whether and how much suicide risk screening changes outcomes, but we know a few things, Dr. Campo said, summing up his key points: “We know suicide’s a major source of mortality in youth that’s been relatively neglected in pediatric health care. Second, we know that suicide risk is associated with risk for other important causes of death, for mental disorders, and for alcohol and substance use.

“We know that most suicide decedents are unrecognized prior to the time of death, and those who are recognized often are not treated. We know that the majority of suicide deaths occur on the very first attempt. We also know that we increasingly have treatments, mental disorders that can be identified, and remediable risk factors, and [that at-risk youth] typically present at general medical settings. Beyond that, focusing on the general medical setting has both conceptual and practical advantages as a site for really helping us to detect patients at risk and then managing them.”

No funding was used for the presentations. Dr. Horowitz and Dr. Campo had no relevant financial disclosures.

Screening youth to identify those at risk for suicide is particularly important in medical settings, given the increasing rates of adolescent suicide, and screening can take as little as 20 seconds, according to Lisa Horowitz, PhD, MPH, a staff scientist and clinical psychologist at the National Institute of Mental Health, Bethesda, Md.

Steve Debenport/Getty Images

But clinicians need to use validated screening instruments that are both population specific and site specific, and they need practice guidelines to treat patients screening positive.

Currently, many practitioners use depression screens – such as question #9 on suicide ideation and self harm on the Patient Health Questionnaire for Adolescents (PHQ-A) – to identify suicide risk, but preliminary data suggest these screens often are inadequate, Dr. Horowitz said. Just one question, especially one without precise language, does not appear to identify as many at-risk youths as more direct questions about suicidal thoughts and behaviors.

A Pathways to Clinical Care suicide risk screening work group therefore designed a three-tiered clinical pathway for suicide risk screenings in emergency departments, inpatient care, and outpatient primary care. It begins with the Ask Suicide-Screening Questions (ASQ), which takes about 20 seconds and was specifically developed for pediatric patients in the emergency department and validated in both inpatient and outpatient settings.

Dr Horowitz, also the lead principal investigator for development of the ASQ, currently is leading six National Institute of Mental Health studies to validate and implement the screening tool in medical settings. She explained the three-tiered system during a session on youth suicide screening at the Pediatric Academic Societies annual meeting in Baltimore this year.

If a patient screens positive on the ASQ, a trained clinician should conduct a brief suicide safety assessment (BSSA), which takes approximately 10 minutes, Dr Horowitz said. Those who screen positive on the BSSA should receive the Patient Resource List and then be referred for a full mental health and safety evaluation, which takes about 30 minutes. Resources, such as nurse scripts and parent/guardian flyers, are available at the NIMH website, as well as translations of the ASQ in Arabic, Chinese, Dutch, French, Hebrew, Italian, Japanese, Korean, Portuguese, Russian, Somali, Spanish, and Vietnamese.

Acknowledging the importance of suicide screening

During the same session, John V. Campo, MD, an assistant dean for behavioral health and professor of behavioral medicine and psychiatry at West Virginia University in Morgantown, discussed why suicide risk screening is so crucial in general medical settings. As someone who trained as a pediatrician before crossing over to behavioral health, he acknowledged that primary care physicians already have many priorities to cover in short visits, and that the national answer to most public health problems is to deal with it in primary care.

“Anyone who has done primary care pediatrics understands the challenges involved with screening for anything – particularly when you identify someone who is extensively at risk,” he said.

But suicide has a disproportionately high impact on young populations, and “identifying youth at risk for suicide identifies a group of young people who are at risk for a variety of threats to their health and well-being,” he said.

For youth aged 10-19 years in 2016, suicide was the second leading cause of death behind accidents, according to the Centers for Disease Control and Prevention (Natl Vital Stat Rep. 2018 Jun;67[4]:1-16). In fact, accidents, suicide, and homicide account for three-quarters of deaths among youth aged 10-24 years (Natl Vital Stat Rep. 2019 Jun;68[6]:1-77), yet it’s typically the other 25% that most physicians trained for in residency.

“Suicide kills more kids than cancer, heart disease, infections – all kinds, sepsis, meningitis, pneumonia, influenza, HIV, respiratory conditions. Suicide kills more young people every year than all of that [combined],” Dr. Campo said. “And yet, when you walk through a modern emergency department, we see all these specialized programs for those who present with physical trauma or chest pain or all these other things, but zero specialized mental health services. There’s a disconnect.”

There is some good news in the data, he said. Observational data have shown that suicide rates negatively correlate with indicators of better access to health and medical health services, and researchers increasingly are identifying proven strategies that help prevent suicide in young people – once they have been identified.

But that’s the problem, “and we all know it,” Dr. Campo continued. “Most youth who are at risk for suicide aren’t recognized, and those who are recognized most often are untreated or inadequately treated,” he said. Further, “the best predictor of future behavior is past behavior,” but most adolescents die by suicide on their first attempt.

Again, however, Dr. Campo pivoted to the good news. Data also have shown that most youth who die by suicide had at least one health contact in the previous year, which means there are opportunities for screening and intervention.

The most common risk factor for suicide is having a mental health or substance use condition, present in about 90% of completed suicides and affecting approximately one in five youth. Prevalence is even higher in those with physical health conditions and among those with Medicaid or no insurance (J Child Psychol Psychiatry. 2006 Mar-Apr;47[3-4]372-94).

Yet, “the majority of them have not been treated at all for mental disorder, which seems to be the most important remediable risk factor for suicide, and even fewer are in current treatment at the time of the death,” Dr. Campo said. Suicide also is correlated with a number of other high-risk behaviors or circumstances, such as “vulnerabilities to substance abuse, riding in a car with someone who is intoxicated, carrying a weapon to school, fighting, and meeting criteria for depression” (Pediatrics. 2010 May;125[5]:945-52). Screening for suicide risk therefore allows physicians to identify youth vulnerable to a wide range of risks, conditions, or death.

Overcoming barriers to suicide screening in primary care

Given the high prevalence of suicide and its link to so many other risks for youth, screening in primary care can send the message that suicide screening “really is a part of health care,” Dr. Campo said. Incorporating screening into primary care also can help overcome distrust of behavioral health specialists in the general public and stigma associated with behavioral health disorders.

Primary care screening emphasizes the importance and credibility of mental health and challenges attitudinal barriers to care, he said.

At the same time, however, he acknowledged that providers themselves often are uneasy about addressing behavioral health. Therefore, “having the guideline and the expectation [of suicide risk screening] really drives home the point that this needs to be integrated into the rest of primary care,” he said. “It’s also consistent with the idea of the medical home.” With suicide the second leading cause of death among youth, “if there’s anything that we’re going to be thinking about screening for, one would think suicide would be high on the list.”

In fact, observational evidence has shown that educating and training primary care providers to recognize people with depression or a high risk for suicide can reduce suicide attempts and the suicide rate, Dr. Campo said (JAMA Psychiatry. 2017 Jun 1;74[6]:563-70). It also can help with the mismatch between where at-risk patients are and where behavioral health specialists are. About 90% of behavioral health specialists work only in specialty settings, and only 5% typically work in general medical settings, he said. Yet “most people who are in mental distress or in crisis don’t present in specialty behavioral health settings. They present in general medical settings.”

More data are needed to demonstrate more definitively whether and how much suicide risk screening changes outcomes, but we know a few things, Dr. Campo said, summing up his key points: “We know suicide’s a major source of mortality in youth that’s been relatively neglected in pediatric health care. Second, we know that suicide risk is associated with risk for other important causes of death, for mental disorders, and for alcohol and substance use.

“We know that most suicide decedents are unrecognized prior to the time of death, and those who are recognized often are not treated. We know that the majority of suicide deaths occur on the very first attempt. We also know that we increasingly have treatments, mental disorders that can be identified, and remediable risk factors, and [that at-risk youth] typically present at general medical settings. Beyond that, focusing on the general medical setting has both conceptual and practical advantages as a site for really helping us to detect patients at risk and then managing them.”

No funding was used for the presentations. Dr. Horowitz and Dr. Campo had no relevant financial disclosures.

China’s National Medical Products Administration has approved a new therapy for patients with mild to moderate Alzheimer’s disease – a seaweed extract thought to alter the gut microbiome profile and subsequently decrease microbiome-driven neuroinflammation.

Sodium oligomannate – dubbed GV-971 – won approval based on a 36-week, placebo-controlled, phase 3 study of 818 patients with mild to moderate Alzheimer’s disease (AD). The study hit its primary endpoint of change on the Alzheimer’s Disease Assessment Scale cognitive portion (ADAS-cog12). It did not meet any of the trial’s other cognitive or functional secondary endpoints.

A portion of the data were presented last year at the Clinical Trials on Alzheimer’s Disease meeting in Barcelona. But the full study has never appeared in a peer-reviewed journal. A truncated version is publicly available on the website of Shanghai Green Valley Pharmaceuticals, the company developing the molecule.

Shanghai Green Valley contends that it reduces neuroinflammation by improving a proinflammatory microbiome profile that it says is characteristic of AD. However, the mechanism by which GV-971 alters intestinal bacterial composition is unclear – or at least it is not fully described in the public literature.

In the United States, some key researchers appraised the news with a cautiously optimistic eye, while others pointed noted that the AD-microbiome link is an unproven concept, and that it was evaluated in a study of questionable worth.

“The company has presented data that suggest there is a modest cognitive benefit to this treatment,” Paul S. Aisen, MD, said in an interview. “The key secondary endpoint was missed, and the other secondary endpoints showed no benefit. It’s a single trial and the mechanism is still unclear.”

“We do need to pursue all possible leads, and I’m glad the company is pursuing additional studies, but I wouldn’t draw a firm conclusion from these data. And they certainly would not be enough to win approval in the U.S.,” said Dr. Aisen, founding director Alzheimer’s Therapeutic Research Institute at the University of Southern California, Los Angeles.

Preclinical findings on GV-971

In commenting on preclinical findings of GV-971 published in Cell Research in September 2019, David Holtzman, MD, associate director of the Alzheimer’s disease research center at Washington University, St. Louis, and coauthors observed that the data support research exploring treatments that modulate the gut microbiome but leave it unclear as to whether GV-971 has AD-specific effects.

“[The company shows] that GV-971 decreases amyloid beta-related pathologies by reconditioning the gut microbiota, providing further evidence that gut-targeted interventions may serve as novel strategies to tackle AD,” Dr. Holtzman and coauthors wrote. “Whether this potential mechanism represents an AD-specific process is not clear, since there is great overlap in immunological changes and gut dysbiosis with other diseases. … In addition, although this study reveals that gut reconditioning may be one mechanism of action of the drug GV-971, it does not rule out other possible mechanisms. For example, GV-971 may attenuate AD pathogenesis by directly inhibiting neuroinflammation or amyloid-beta fibril formation. However, there is no question that [these] data further [support] the emerging idea that modulation of the gut microbiome via treatments such as GV-971 or other strategies should be further explored as novel strategies to slow the progression of AD.”

Sodium oligomannate is a long-chain saccharide extracted from brown sea algae and consists of acidic linear oligosaccharides with structures ranging from dimers to decamers. Related molecules without the sugar backbone were inactive, suggesting that the saccharides are the active portion, Xinyi Wang of Shanghai Green Valley and colleagues wrote in the Cell Research paper.

Based on these studies, the company contends that Alzheimer’s progression is accompanied by a characteristic microbiome change to a proinflammatory profile. And indeed, two transgenic Alzheimer’s mouse models – one with five familial AD mutations (5xFAD) and one with mutations of amyloid precursor protein and presenilin 1 (APP/PS1) – showed similar gradual age- and progression-related decreases of Bacteroides and Verrucomicrobia, two components of a normal microbiome. Bacteroides species perform key functions necessary for survival, including sensing and adapting to nutrient variability, expelling toxins, and stimulating the immune system). Species of the Verrucomicrobia phylum are important in glucose homeostasis. The decline in Bacteroides and Verrucomicrobia species is accompanied by an increase in concomitant proinflammatory species.

The investigators then explored the relationship between the microbiome composition and cognitive function in both transgenic models and a wild-type mouse.

First, they showed that the bacterial populations shifted as the mice aged and their AD pathology developed. This was accompanied by an uptick in activated microglia and, in turn, proinflammatory T1 helper cells that migrated through the intestinal membranes and into the periphery, then cross the blood-brain barrier to enter the brain.

Then the investigators used a cocktail of powerful antibiotics to disturb the intestinal flora in both transgenic and wild-type mice. After this, the 5xFAD mice showed fewer activated microglia and fewer infiltrating T cells. Later, they gave wild-type mice a fecal transplant from the 5xFAD mice. The wild-type mice developed more activated and infiltrating cells and their microbiome began to resemble that of the transgenic mice. Conversely, when the transgenic mice received a transplant from the wild-type mice, their microbiome changed to resemble the donors’, and their activated and infiltrating cells declined.

After this, the team gave GV-971 to the mice. The APP/PS1 mice improved cognitively, and the 5xFAD mice had fewer activated and infiltrating cells, fewer amyloid brain plaques, and less tau phosphorylation. These changes were accompanied by higher levels of two amino acids, phenylalanine and isoleucine. These proteins appear to act on T-cell proliferation and differentiation, they said.

“Taken together, these analyses suggest the idea that gut dysbiosis contributes to [phenylalanine and isoleucine] elevation, which drives the proliferation/differentiation and brain infiltration of [T1 helper] cells,” Dr. Holtzman and coauthors wrote. “These infiltrating Th1 cells may then further activate microglia and contribute to amyloid-related pathogenesis.”

The phase 3 study

The approval of GV-971 was based on the subsequent 36-week phase 3, placebo-controlled study of 818 patients with mild to moderate AD, which was reported last year. Patients were randomized to placebo or to GV-971 450 mg twice daily. Amyloid PET imaging was not required at entrance to the study, so there was no measure of baseline amyloid load. However, all patients showed MRI evidence of cortical atrophy. The Mini Mental State Exam (MMSE) ranged from 11 to 26, indicating mild to moderate AD.

Secondary endpoints included change on the Alzheimer’s Disease Cooperative Study Activities of Daily Living, the Clinician’s Interview-Based Impression of Change–Plus, and the Neuropsychiatric Index.

Patients taking GV-971 experienced a statistically significant 2.54-point difference on the ADAS-cog12, compared with placebo. The difference was apparent by week 4 and was seen at every clinical visit. When patients were grouped according to baseline MMSE (11-14, 15-19, and 20-26), the drug performed similarly.

Michele G Sullivan/MDedge News

But none of the secondary endpoints significantly favored of GV-971. And the placebo group behaved in an unexpected way, which could throw the data interpretation off-kilter somewhat, David Knopman, MD, said in an interview.

“It was a very weird and unusual-looking trajectory of the placebo and treated groups,” said Dr. Knopman of the Mayo Clinic, Rochester, Minn., with those taking placebo staying relatively stable for some time before the cognitive scores dropped precipitously. “The data were unconvincing to me.”

He also pointed out that although the extensive preclinical data appeared in the recent peer-reviewed Cell Research paper, the phase 3 data has not appeared in any peer-reviewed forum, “even though the trial has been completed for some time now. Furthermore, the duration of the study was inadequate.”

Finally, none of the study subjects were taking the standard-of-care cholinesterase inhibitors, which virtually every AD patient in the United States does take.

“This makes it almost completely inapplicable to the U.S.,” he said. “It’s not bad news. I’m just not convinced.”

Dr. Holtzman is a cofounder of C2N Diagnostics. He is on the scientific advisory board of Genentech, Denali, and C2N Diagnostics. He consults for Idorsia. Dr. Knopman is a consultant for the Bluefield Project to Cure Frontotemporal Dementia and for Lundbeck.

China’s National Medical Products Administration has approved a new therapy for patients with mild to moderate Alzheimer’s disease – a seaweed extract thought to alter the gut microbiome profile and subsequently decrease microbiome-driven neuroinflammation.

Sodium oligomannate – dubbed GV-971 – won approval based on a 36-week, placebo-controlled, phase 3 study of 818 patients with mild to moderate Alzheimer’s disease (AD). The study hit its primary endpoint of change on the Alzheimer’s Disease Assessment Scale cognitive portion (ADAS-cog12). It did not meet any of the trial’s other cognitive or functional secondary endpoints.

A portion of the data were presented last year at the Clinical Trials on Alzheimer’s Disease meeting in Barcelona. But the full study has never appeared in a peer-reviewed journal. A truncated version is publicly available on the website of Shanghai Green Valley Pharmaceuticals, the company developing the molecule.

Shanghai Green Valley contends that it reduces neuroinflammation by improving a proinflammatory microbiome profile that it says is characteristic of AD. However, the mechanism by which GV-971 alters intestinal bacterial composition is unclear – or at least it is not fully described in the public literature.

In the United States, some key researchers appraised the news with a cautiously optimistic eye, while others pointed noted that the AD-microbiome link is an unproven concept, and that it was evaluated in a study of questionable worth.

“The company has presented data that suggest there is a modest cognitive benefit to this treatment,” Paul S. Aisen, MD, said in an interview. “The key secondary endpoint was missed, and the other secondary endpoints showed no benefit. It’s a single trial and the mechanism is still unclear.”

“We do need to pursue all possible leads, and I’m glad the company is pursuing additional studies, but I wouldn’t draw a firm conclusion from these data. And they certainly would not be enough to win approval in the U.S.,” said Dr. Aisen, founding director Alzheimer’s Therapeutic Research Institute at the University of Southern California, Los Angeles.

Preclinical findings on GV-971

In commenting on preclinical findings of GV-971 published in Cell Research in September 2019, David Holtzman, MD, associate director of the Alzheimer’s disease research center at Washington University, St. Louis, and coauthors observed that the data support research exploring treatments that modulate the gut microbiome but leave it unclear as to whether GV-971 has AD-specific effects.

“[The company shows] that GV-971 decreases amyloid beta-related pathologies by reconditioning the gut microbiota, providing further evidence that gut-targeted interventions may serve as novel strategies to tackle AD,” Dr. Holtzman and coauthors wrote. “Whether this potential mechanism represents an AD-specific process is not clear, since there is great overlap in immunological changes and gut dysbiosis with other diseases. … In addition, although this study reveals that gut reconditioning may be one mechanism of action of the drug GV-971, it does not rule out other possible mechanisms. For example, GV-971 may attenuate AD pathogenesis by directly inhibiting neuroinflammation or amyloid-beta fibril formation. However, there is no question that [these] data further [support] the emerging idea that modulation of the gut microbiome via treatments such as GV-971 or other strategies should be further explored as novel strategies to slow the progression of AD.”

Sodium oligomannate is a long-chain saccharide extracted from brown sea algae and consists of acidic linear oligosaccharides with structures ranging from dimers to decamers. Related molecules without the sugar backbone were inactive, suggesting that the saccharides are the active portion, Xinyi Wang of Shanghai Green Valley and colleagues wrote in the Cell Research paper.

Based on these studies, the company contends that Alzheimer’s progression is accompanied by a characteristic microbiome change to a proinflammatory profile. And indeed, two transgenic Alzheimer’s mouse models – one with five familial AD mutations (5xFAD) and one with mutations of amyloid precursor protein and presenilin 1 (APP/PS1) – showed similar gradual age- and progression-related decreases of Bacteroides and Verrucomicrobia, two components of a normal microbiome. Bacteroides species perform key functions necessary for survival, including sensing and adapting to nutrient variability, expelling toxins, and stimulating the immune system). Species of the Verrucomicrobia phylum are important in glucose homeostasis. The decline in Bacteroides and Verrucomicrobia species is accompanied by an increase in concomitant proinflammatory species.

The investigators then explored the relationship between the microbiome composition and cognitive function in both transgenic models and a wild-type mouse.

First, they showed that the bacterial populations shifted as the mice aged and their AD pathology developed. This was accompanied by an uptick in activated microglia and, in turn, proinflammatory T1 helper cells that migrated through the intestinal membranes and into the periphery, then cross the blood-brain barrier to enter the brain.

Then the investigators used a cocktail of powerful antibiotics to disturb the intestinal flora in both transgenic and wild-type mice. After this, the 5xFAD mice showed fewer activated microglia and fewer infiltrating T cells. Later, they gave wild-type mice a fecal transplant from the 5xFAD mice. The wild-type mice developed more activated and infiltrating cells and their microbiome began to resemble that of the transgenic mice. Conversely, when the transgenic mice received a transplant from the wild-type mice, their microbiome changed to resemble the donors’, and their activated and infiltrating cells declined.

After this, the team gave GV-971 to the mice. The APP/PS1 mice improved cognitively, and the 5xFAD mice had fewer activated and infiltrating cells, fewer amyloid brain plaques, and less tau phosphorylation. These changes were accompanied by higher levels of two amino acids, phenylalanine and isoleucine. These proteins appear to act on T-cell proliferation and differentiation, they said.

“Taken together, these analyses suggest the idea that gut dysbiosis contributes to [phenylalanine and isoleucine] elevation, which drives the proliferation/differentiation and brain infiltration of [T1 helper] cells,” Dr. Holtzman and coauthors wrote. “These infiltrating Th1 cells may then further activate microglia and contribute to amyloid-related pathogenesis.”

The phase 3 study

The approval of GV-971 was based on the subsequent 36-week phase 3, placebo-controlled study of 818 patients with mild to moderate AD, which was reported last year. Patients were randomized to placebo or to GV-971 450 mg twice daily. Amyloid PET imaging was not required at entrance to the study, so there was no measure of baseline amyloid load. However, all patients showed MRI evidence of cortical atrophy. The Mini Mental State Exam (MMSE) ranged from 11 to 26, indicating mild to moderate AD.

Secondary endpoints included change on the Alzheimer’s Disease Cooperative Study Activities of Daily Living, the Clinician’s Interview-Based Impression of Change–Plus, and the Neuropsychiatric Index.

Patients taking GV-971 experienced a statistically significant 2.54-point difference on the ADAS-cog12, compared with placebo. The difference was apparent by week 4 and was seen at every clinical visit. When patients were grouped according to baseline MMSE (11-14, 15-19, and 20-26), the drug performed similarly.

Michele G Sullivan/MDedge News

But none of the secondary endpoints significantly favored of GV-971. And the placebo group behaved in an unexpected way, which could throw the data interpretation off-kilter somewhat, David Knopman, MD, said in an interview.

“It was a very weird and unusual-looking trajectory of the placebo and treated groups,” said Dr. Knopman of the Mayo Clinic, Rochester, Minn., with those taking placebo staying relatively stable for some time before the cognitive scores dropped precipitously. “The data were unconvincing to me.”

He also pointed out that although the extensive preclinical data appeared in the recent peer-reviewed Cell Research paper, the phase 3 data has not appeared in any peer-reviewed forum, “even though the trial has been completed for some time now. Furthermore, the duration of the study was inadequate.”

Finally, none of the study subjects were taking the standard-of-care cholinesterase inhibitors, which virtually every AD patient in the United States does take.

“This makes it almost completely inapplicable to the U.S.,” he said. “It’s not bad news. I’m just not convinced.”

Dr. Holtzman is a cofounder of C2N Diagnostics. He is on the scientific advisory board of Genentech, Denali, and C2N Diagnostics. He consults for Idorsia. Dr. Knopman is a consultant for the Bluefield Project to Cure Frontotemporal Dementia and for Lundbeck.

China’s National Medical Products Administration has approved a new therapy for patients with mild to moderate Alzheimer’s disease – a seaweed extract thought to alter the gut microbiome profile and subsequently decrease microbiome-driven neuroinflammation.

Sodium oligomannate – dubbed GV-971 – won approval based on a 36-week, placebo-controlled, phase 3 study of 818 patients with mild to moderate Alzheimer’s disease (AD). The study hit its primary endpoint of change on the Alzheimer’s Disease Assessment Scale cognitive portion (ADAS-cog12). It did not meet any of the trial’s other cognitive or functional secondary endpoints.

A portion of the data were presented last year at the Clinical Trials on Alzheimer’s Disease meeting in Barcelona. But the full study has never appeared in a peer-reviewed journal. A truncated version is publicly available on the website of Shanghai Green Valley Pharmaceuticals, the company developing the molecule.

Shanghai Green Valley contends that it reduces neuroinflammation by improving a proinflammatory microbiome profile that it says is characteristic of AD. However, the mechanism by which GV-971 alters intestinal bacterial composition is unclear – or at least it is not fully described in the public literature.

In the United States, some key researchers appraised the news with a cautiously optimistic eye, while others pointed noted that the AD-microbiome link is an unproven concept, and that it was evaluated in a study of questionable worth.

“The company has presented data that suggest there is a modest cognitive benefit to this treatment,” Paul S. Aisen, MD, said in an interview. “The key secondary endpoint was missed, and the other secondary endpoints showed no benefit. It’s a single trial and the mechanism is still unclear.”

“We do need to pursue all possible leads, and I’m glad the company is pursuing additional studies, but I wouldn’t draw a firm conclusion from these data. And they certainly would not be enough to win approval in the U.S.,” said Dr. Aisen, founding director Alzheimer’s Therapeutic Research Institute at the University of Southern California, Los Angeles.

Preclinical findings on GV-971

In commenting on preclinical findings of GV-971 published in Cell Research in September 2019, David Holtzman, MD, associate director of the Alzheimer’s disease research center at Washington University, St. Louis, and coauthors observed that the data support research exploring treatments that modulate the gut microbiome but leave it unclear as to whether GV-971 has AD-specific effects.

“[The company shows] that GV-971 decreases amyloid beta-related pathologies by reconditioning the gut microbiota, providing further evidence that gut-targeted interventions may serve as novel strategies to tackle AD,” Dr. Holtzman and coauthors wrote. “Whether this potential mechanism represents an AD-specific process is not clear, since there is great overlap in immunological changes and gut dysbiosis with other diseases. … In addition, although this study reveals that gut reconditioning may be one mechanism of action of the drug GV-971, it does not rule out other possible mechanisms. For example, GV-971 may attenuate AD pathogenesis by directly inhibiting neuroinflammation or amyloid-beta fibril formation. However, there is no question that [these] data further [support] the emerging idea that modulation of the gut microbiome via treatments such as GV-971 or other strategies should be further explored as novel strategies to slow the progression of AD.”

Sodium oligomannate is a long-chain saccharide extracted from brown sea algae and consists of acidic linear oligosaccharides with structures ranging from dimers to decamers. Related molecules without the sugar backbone were inactive, suggesting that the saccharides are the active portion, Xinyi Wang of Shanghai Green Valley and colleagues wrote in the Cell Research paper.

Based on these studies, the company contends that Alzheimer’s progression is accompanied by a characteristic microbiome change to a proinflammatory profile. And indeed, two transgenic Alzheimer’s mouse models – one with five familial AD mutations (5xFAD) and one with mutations of amyloid precursor protein and presenilin 1 (APP/PS1) – showed similar gradual age- and progression-related decreases of Bacteroides and Verrucomicrobia, two components of a normal microbiome. Bacteroides species perform key functions necessary for survival, including sensing and adapting to nutrient variability, expelling toxins, and stimulating the immune system). Species of the Verrucomicrobia phylum are important in glucose homeostasis. The decline in Bacteroides and Verrucomicrobia species is accompanied by an increase in concomitant proinflammatory species.

The investigators then explored the relationship between the microbiome composition and cognitive function in both transgenic models and a wild-type mouse.

First, they showed that the bacterial populations shifted as the mice aged and their AD pathology developed. This was accompanied by an uptick in activated microglia and, in turn, proinflammatory T1 helper cells that migrated through the intestinal membranes and into the periphery, then cross the blood-brain barrier to enter the brain.

Then the investigators used a cocktail of powerful antibiotics to disturb the intestinal flora in both transgenic and wild-type mice. After this, the 5xFAD mice showed fewer activated microglia and fewer infiltrating T cells. Later, they gave wild-type mice a fecal transplant from the 5xFAD mice. The wild-type mice developed more activated and infiltrating cells and their microbiome began to resemble that of the transgenic mice. Conversely, when the transgenic mice received a transplant from the wild-type mice, their microbiome changed to resemble the donors’, and their activated and infiltrating cells declined.

After this, the team gave GV-971 to the mice. The APP/PS1 mice improved cognitively, and the 5xFAD mice had fewer activated and infiltrating cells, fewer amyloid brain plaques, and less tau phosphorylation. These changes were accompanied by higher levels of two amino acids, phenylalanine and isoleucine. These proteins appear to act on T-cell proliferation and differentiation, they said.

“Taken together, these analyses suggest the idea that gut dysbiosis contributes to [phenylalanine and isoleucine] elevation, which drives the proliferation/differentiation and brain infiltration of [T1 helper] cells,” Dr. Holtzman and coauthors wrote. “These infiltrating Th1 cells may then further activate microglia and contribute to amyloid-related pathogenesis.”

The phase 3 study

The approval of GV-971 was based on the subsequent 36-week phase 3, placebo-controlled study of 818 patients with mild to moderate AD, which was reported last year. Patients were randomized to placebo or to GV-971 450 mg twice daily. Amyloid PET imaging was not required at entrance to the study, so there was no measure of baseline amyloid load. However, all patients showed MRI evidence of cortical atrophy. The Mini Mental State Exam (MMSE) ranged from 11 to 26, indicating mild to moderate AD.

Secondary endpoints included change on the Alzheimer’s Disease Cooperative Study Activities of Daily Living, the Clinician’s Interview-Based Impression of Change–Plus, and the Neuropsychiatric Index.

Patients taking GV-971 experienced a statistically significant 2.54-point difference on the ADAS-cog12, compared with placebo. The difference was apparent by week 4 and was seen at every clinical visit. When patients were grouped according to baseline MMSE (11-14, 15-19, and 20-26), the drug performed similarly.

Michele G Sullivan/MDedge News

But none of the secondary endpoints significantly favored of GV-971. And the placebo group behaved in an unexpected way, which could throw the data interpretation off-kilter somewhat, David Knopman, MD, said in an interview.

“It was a very weird and unusual-looking trajectory of the placebo and treated groups,” said Dr. Knopman of the Mayo Clinic, Rochester, Minn., with those taking placebo staying relatively stable for some time before the cognitive scores dropped precipitously. “The data were unconvincing to me.”

He also pointed out that although the extensive preclinical data appeared in the recent peer-reviewed Cell Research paper, the phase 3 data has not appeared in any peer-reviewed forum, “even though the trial has been completed for some time now. Furthermore, the duration of the study was inadequate.”

Finally, none of the study subjects were taking the standard-of-care cholinesterase inhibitors, which virtually every AD patient in the United States does take.

“This makes it almost completely inapplicable to the U.S.,” he said. “It’s not bad news. I’m just not convinced.”

Dr. Holtzman is a cofounder of C2N Diagnostics. He is on the scientific advisory board of Genentech, Denali, and C2N Diagnostics. He consults for Idorsia. Dr. Knopman is a consultant for the Bluefield Project to Cure Frontotemporal Dementia and for Lundbeck.

The Food and Drug Administration has approved crizanlizumab-tmca (Adakveo) to reduce the frequency of vaso-occlusive crisis, a common complication of sickle cell disease.

The drug is approved for patients aged 16 years and older. It was approved on the strength of the SUSTAIN trial, which randomized 198 patients with sickle cell disease and a history of vaso-occlusive crisis to crizanlizumab or placebo. Patients who received crizanlizumab had a median annual rate of 1.63 health care visits for vaso-occlusive crises, compared with patients who received placebo and had a median annual rate of 2.98 visits. The drug also delayed the first vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months, according to the FDA.

“Adakveo is the first targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement. “Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease.”

Common adverse events associated with crizanlizumab included back pain, nausea, pyrexia, and arthralgia. The FDA advised physicians to monitor patients for infusion-related reactions.

[email protected]

The Food and Drug Administration has approved crizanlizumab-tmca (Adakveo) to reduce the frequency of vaso-occlusive crisis, a common complication of sickle cell disease.

The drug is approved for patients aged 16 years and older. It was approved on the strength of the SUSTAIN trial, which randomized 198 patients with sickle cell disease and a history of vaso-occlusive crisis to crizanlizumab or placebo. Patients who received crizanlizumab had a median annual rate of 1.63 health care visits for vaso-occlusive crises, compared with patients who received placebo and had a median annual rate of 2.98 visits. The drug also delayed the first vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months, according to the FDA.

“Adakveo is the first targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement. “Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease.”

Common adverse events associated with crizanlizumab included back pain, nausea, pyrexia, and arthralgia. The FDA advised physicians to monitor patients for infusion-related reactions.

[email protected]

The Food and Drug Administration has approved crizanlizumab-tmca (Adakveo) to reduce the frequency of vaso-occlusive crisis, a common complication of sickle cell disease.

The drug is approved for patients aged 16 years and older. It was approved on the strength of the SUSTAIN trial, which randomized 198 patients with sickle cell disease and a history of vaso-occlusive crisis to crizanlizumab or placebo. Patients who received crizanlizumab had a median annual rate of 1.63 health care visits for vaso-occlusive crises, compared with patients who received placebo and had a median annual rate of 2.98 visits. The drug also delayed the first vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months, according to the FDA.

“Adakveo is the first targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement. “Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease.”

Common adverse events associated with crizanlizumab included back pain, nausea, pyrexia, and arthralgia. The FDA advised physicians to monitor patients for infusion-related reactions.

[email protected]

LAS VEGAS – Be alert for hidden cases of hyperhidrosis in patients, Seemal R. Desai, MD, said during a presentation on this topic at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

During an examination for another condition, he said, patients may be “sweating and dripping.” However, “you look over that diagnosis because that’s not what they’re there for,” said Dr. Desai, a dermatologist at the University of Texas Southwestern Medical Center in Dallas.

He described one of his patients, who only revealed that she suffered from “horrible, devastating” hyperhidrosis after he’d treated her for years for melasma. The sweating especially affected her because it prevented her from wearing the skin-exposing clothing of her Indian culture.

Delays in treatment are common in hyperhidrosis, which is believed to affect 5% of the world’s population. According to Dr. Desai, research suggests that 85% of patients with hyperhidrosis wait more than 3 years to bring it up with doctors, and half wait more than a decade.

There are many treatments for hyperhidrosis. Some are fairly simple: over-the-counter or prescription antiperspirants, said Dr. Desai, who likes the over-the-counter brand Certain Dri), iontophoresis (application of electric current), topical anticholinergics (including glycopyrronium tosylate cloth wipes, recently approved by the FDA for topical treatment of primary axillary hyperhidrosis for ages 9 years and older), and systemic management. Others are minimally invasive: Botox injections and the miraDry medical device (which relies on thermolysis). And surgical strategies may be an option for severe cases.

On its website, the International Hyperhidrosis Society provides a chart of options for hyperhidrosis in various parts of the body. Treatments tend to focus on the underarms, however, and “we’ve got huge unmet needs for patient options,” Dr. Desai said.
 

• During his presentation, he provided the following pearls regarding hyperhidrosis treatments:
• Distinguish between antiperspirants, which block sweating, and deodorants, which cover up body odor. “Sometimes I get caught up in the middle of a busy office visit and use these terms interchangeably. They’re really different, but patients and the public tend to equate those together,” he commented.
• Make sure patients understand how to properly use antiperspirants and explain that antiperspirants must be applied to dry skin. “Antiperspirant is forming a clog in the drain” to prevent the release of sweat, he said. “If you apply it to wet skin, you will block that chemical reaction in the duct.”
• Massage in the antiperspirant, he advises, and don’t occlude the skin. Apply twice daily, including before bedtime. “They can use antiperspirant on the hands and the bottom of the feet,”Dr. Desai said. “You want to ensure that they’re using the spray on the surface and in the web space. They can also use antiperspirants on the face, but avoid contact with the eyes.”
• Be careful if you prescribe glycopyrronium cloths off label. These wipes are helpful and they can be used outside the FDA-approved use in the underarms, said Dr. Desai, who said he has palmar hyperhidrosis and has successfully used them on his palms, but he hasn’t found them to be helpful on the soles of his feet.

Dr. Desai recommends 5-minute applications on the palms because the treatment can irritate the face and eyes.

Linda F. Stein Gold, MD, of Henry Ford Health System in Detroit, told the audience about the case of a teacher who touched his eyes after applying the treatment. He went to school, felt ill, and ended up in an emergency department because he had an enlarged pupil. “You just have to tell people this can happen,” she said.

Dr. Desai reported no relevant disclosures.

SDEF and this news organization are owned by the same parent company.

LAS VEGAS – Be alert for hidden cases of hyperhidrosis in patients, Seemal R. Desai, MD, said during a presentation on this topic at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

During an examination for another condition, he said, patients may be “sweating and dripping.” However, “you look over that diagnosis because that’s not what they’re there for,” said Dr. Desai, a dermatologist at the University of Texas Southwestern Medical Center in Dallas.

He described one of his patients, who only revealed that she suffered from “horrible, devastating” hyperhidrosis after he’d treated her for years for melasma. The sweating especially affected her because it prevented her from wearing the skin-exposing clothing of her Indian culture.

Delays in treatment are common in hyperhidrosis, which is believed to affect 5% of the world’s population. According to Dr. Desai, research suggests that 85% of patients with hyperhidrosis wait more than 3 years to bring it up with doctors, and half wait more than a decade.

There are many treatments for hyperhidrosis. Some are fairly simple: over-the-counter or prescription antiperspirants, said Dr. Desai, who likes the over-the-counter brand Certain Dri), iontophoresis (application of electric current), topical anticholinergics (including glycopyrronium tosylate cloth wipes, recently approved by the FDA for topical treatment of primary axillary hyperhidrosis for ages 9 years and older), and systemic management. Others are minimally invasive: Botox injections and the miraDry medical device (which relies on thermolysis). And surgical strategies may be an option for severe cases.

On its website, the International Hyperhidrosis Society provides a chart of options for hyperhidrosis in various parts of the body. Treatments tend to focus on the underarms, however, and “we’ve got huge unmet needs for patient options,” Dr. Desai said.
 

• During his presentation, he provided the following pearls regarding hyperhidrosis treatments:
• Distinguish between antiperspirants, which block sweating, and deodorants, which cover up body odor. “Sometimes I get caught up in the middle of a busy office visit and use these terms interchangeably. They’re really different, but patients and the public tend to equate those together,” he commented.
• Make sure patients understand how to properly use antiperspirants and explain that antiperspirants must be applied to dry skin. “Antiperspirant is forming a clog in the drain” to prevent the release of sweat, he said. “If you apply it to wet skin, you will block that chemical reaction in the duct.”
• Massage in the antiperspirant, he advises, and don’t occlude the skin. Apply twice daily, including before bedtime. “They can use antiperspirant on the hands and the bottom of the feet,”Dr. Desai said. “You want to ensure that they’re using the spray on the surface and in the web space. They can also use antiperspirants on the face, but avoid contact with the eyes.”
• Be careful if you prescribe glycopyrronium cloths off label. These wipes are helpful and they can be used outside the FDA-approved use in the underarms, said Dr. Desai, who said he has palmar hyperhidrosis and has successfully used them on his palms, but he hasn’t found them to be helpful on the soles of his feet.

Dr. Desai recommends 5-minute applications on the palms because the treatment can irritate the face and eyes.

Linda F. Stein Gold, MD, of Henry Ford Health System in Detroit, told the audience about the case of a teacher who touched his eyes after applying the treatment. He went to school, felt ill, and ended up in an emergency department because he had an enlarged pupil. “You just have to tell people this can happen,” she said.

Dr. Desai reported no relevant disclosures.

SDEF and this news organization are owned by the same parent company.

LAS VEGAS – Be alert for hidden cases of hyperhidrosis in patients, Seemal R. Desai, MD, said during a presentation on this topic at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

During an examination for another condition, he said, patients may be “sweating and dripping.” However, “you look over that diagnosis because that’s not what they’re there for,” said Dr. Desai, a dermatologist at the University of Texas Southwestern Medical Center in Dallas.

He described one of his patients, who only revealed that she suffered from “horrible, devastating” hyperhidrosis after he’d treated her for years for melasma. The sweating especially affected her because it prevented her from wearing the skin-exposing clothing of her Indian culture.

Delays in treatment are common in hyperhidrosis, which is believed to affect 5% of the world’s population. According to Dr. Desai, research suggests that 85% of patients with hyperhidrosis wait more than 3 years to bring it up with doctors, and half wait more than a decade.

There are many treatments for hyperhidrosis. Some are fairly simple: over-the-counter or prescription antiperspirants, said Dr. Desai, who likes the over-the-counter brand Certain Dri), iontophoresis (application of electric current), topical anticholinergics (including glycopyrronium tosylate cloth wipes, recently approved by the FDA for topical treatment of primary axillary hyperhidrosis for ages 9 years and older), and systemic management. Others are minimally invasive: Botox injections and the miraDry medical device (which relies on thermolysis). And surgical strategies may be an option for severe cases.

On its website, the International Hyperhidrosis Society provides a chart of options for hyperhidrosis in various parts of the body. Treatments tend to focus on the underarms, however, and “we’ve got huge unmet needs for patient options,” Dr. Desai said.
 

• During his presentation, he provided the following pearls regarding hyperhidrosis treatments:
• Distinguish between antiperspirants, which block sweating, and deodorants, which cover up body odor. “Sometimes I get caught up in the middle of a busy office visit and use these terms interchangeably. They’re really different, but patients and the public tend to equate those together,” he commented.
• Make sure patients understand how to properly use antiperspirants and explain that antiperspirants must be applied to dry skin. “Antiperspirant is forming a clog in the drain” to prevent the release of sweat, he said. “If you apply it to wet skin, you will block that chemical reaction in the duct.”
• Massage in the antiperspirant, he advises, and don’t occlude the skin. Apply twice daily, including before bedtime. “They can use antiperspirant on the hands and the bottom of the feet,”Dr. Desai said. “You want to ensure that they’re using the spray on the surface and in the web space. They can also use antiperspirants on the face, but avoid contact with the eyes.”
• Be careful if you prescribe glycopyrronium cloths off label. These wipes are helpful and they can be used outside the FDA-approved use in the underarms, said Dr. Desai, who said he has palmar hyperhidrosis and has successfully used them on his palms, but he hasn’t found them to be helpful on the soles of his feet.

Dr. Desai recommends 5-minute applications on the palms because the treatment can irritate the face and eyes.

Linda F. Stein Gold, MD, of Henry Ford Health System in Detroit, told the audience about the case of a teacher who touched his eyes after applying the treatment. He went to school, felt ill, and ended up in an emergency department because he had an enlarged pupil. “You just have to tell people this can happen,” she said.

Dr. Desai reported no relevant disclosures.

SDEF and this news organization are owned by the same parent company.

LAS VEGAS – It is important to think outside the box and consider whether secondary causes of hyperhidrosis are at play when a patient complains of sweating too much, a dermatologist told his colleagues.

“Look at where the patient fits into the sweating paradigm,” advised Seemal R. Desai, MD, of University of Texas Southwestern Medical Center in Dallas, and consider factors such as where and how often patients are oversweating.

In cases of secondary hyperhidrosis – those that are caused by another condition – “the key is to really find the underlying cause of the problem and not just try to treat the sweating,” said Dr. Desai, who spoke in a presentation at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

According to Dr. Desai, the answers to several questions can help pinpoint a diagnosis of primary hyperhidrosis (also known as focal or primary focal hyperhidrosis) or secondary hyperhidrosis:

• Where does the sweating occur?

Sweating occurs over large parts of the body in patients with secondary hyperhidrosis, Dr. Desai said, although it is typically limited to certain areas, such as the armpits, palms, or soles in the primary form.

• When did the sweating begin?

When sweating begins in adulthood, he said, there’s a good chance that it has a secondary cause. Sweating that began in childhood is more likely to be the primary form.

• How does sweating occur at night?

Dr. Desai advised: “Ask about sleep patterns. Do you sweat during your sleep or wake up feeling like you’re sweating?” Sweating throughout a sleep cycle – not “night sweats” that are brief in nature – indicate a probable secondary cause, he said.

According to Dr. Desai, the causes of secondary hyperhidrosis are numerous, including hypoglycemia, neural tumors, and cardiovascular conditions. “Typically, if I’m trying to figure out why a patient is having generalized sweating, the No. 1 cause is medications.”

Dr. Desai reported no relevant disclosures. SDEF and this news organization are owned by the same parent company.

LAS VEGAS – It is important to think outside the box and consider whether secondary causes of hyperhidrosis are at play when a patient complains of sweating too much, a dermatologist told his colleagues.

“Look at where the patient fits into the sweating paradigm,” advised Seemal R. Desai, MD, of University of Texas Southwestern Medical Center in Dallas, and consider factors such as where and how often patients are oversweating.

In cases of secondary hyperhidrosis – those that are caused by another condition – “the key is to really find the underlying cause of the problem and not just try to treat the sweating,” said Dr. Desai, who spoke in a presentation at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

According to Dr. Desai, the answers to several questions can help pinpoint a diagnosis of primary hyperhidrosis (also known as focal or primary focal hyperhidrosis) or secondary hyperhidrosis:

• Where does the sweating occur?

Sweating occurs over large parts of the body in patients with secondary hyperhidrosis, Dr. Desai said, although it is typically limited to certain areas, such as the armpits, palms, or soles in the primary form.

• When did the sweating begin?

When sweating begins in adulthood, he said, there’s a good chance that it has a secondary cause. Sweating that began in childhood is more likely to be the primary form.

• How does sweating occur at night?

Dr. Desai advised: “Ask about sleep patterns. Do you sweat during your sleep or wake up feeling like you’re sweating?” Sweating throughout a sleep cycle – not “night sweats” that are brief in nature – indicate a probable secondary cause, he said.

According to Dr. Desai, the causes of secondary hyperhidrosis are numerous, including hypoglycemia, neural tumors, and cardiovascular conditions. “Typically, if I’m trying to figure out why a patient is having generalized sweating, the No. 1 cause is medications.”

Dr. Desai reported no relevant disclosures. SDEF and this news organization are owned by the same parent company.

LAS VEGAS – It is important to think outside the box and consider whether secondary causes of hyperhidrosis are at play when a patient complains of sweating too much, a dermatologist told his colleagues.

“Look at where the patient fits into the sweating paradigm,” advised Seemal R. Desai, MD, of University of Texas Southwestern Medical Center in Dallas, and consider factors such as where and how often patients are oversweating.

In cases of secondary hyperhidrosis – those that are caused by another condition – “the key is to really find the underlying cause of the problem and not just try to treat the sweating,” said Dr. Desai, who spoke in a presentation at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

According to Dr. Desai, the answers to several questions can help pinpoint a diagnosis of primary hyperhidrosis (also known as focal or primary focal hyperhidrosis) or secondary hyperhidrosis:

• Where does the sweating occur?

Sweating occurs over large parts of the body in patients with secondary hyperhidrosis, Dr. Desai said, although it is typically limited to certain areas, such as the armpits, palms, or soles in the primary form.

• When did the sweating begin?

When sweating begins in adulthood, he said, there’s a good chance that it has a secondary cause. Sweating that began in childhood is more likely to be the primary form.

• How does sweating occur at night?

Dr. Desai advised: “Ask about sleep patterns. Do you sweat during your sleep or wake up feeling like you’re sweating?” Sweating throughout a sleep cycle – not “night sweats” that are brief in nature – indicate a probable secondary cause, he said.

According to Dr. Desai, the causes of secondary hyperhidrosis are numerous, including hypoglycemia, neural tumors, and cardiovascular conditions. “Typically, if I’m trying to figure out why a patient is having generalized sweating, the No. 1 cause is medications.”

Dr. Desai reported no relevant disclosures. SDEF and this news organization are owned by the same parent company.

LAS VEGAS – Merkel cell carcinoma, an extremely rare form of skin cancer, is often caused by a subclinical virus that routinely inhabits the skin. Now, a serum test of virus antibody levels is offering insight into the state of the disease, according to one dermatologist.

“If you have these antibodies, you have a better prognosis. You can follow those antibodies to test for recurrence or progression,” Isaac Brownell, MD, PhD, of the Dermatology Branch of the National Institutes of Health said at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

The cancer appears in the skin’s Merkel cells, which contribute to our sense of touch by helping us to discriminate textures. “When you put your hand in your pocket, and you can tell the difference between the front and back of a quarter,” he said, “you’re using the Merkel cells in your fingertips.”

Only about 2,500 cases of Merkel cell carcinoma appear in the United States each year, Dr. Brownell said. It appears more often in elderly white patients, is more common in men than women, and is more likely among immunosuppressed patients, whose risk is increased 15- to 20-fold. Cases are more common in sunnier regions – at least in men – and lesions frequently appear on the head, face, and neck.

Five-year survival is estimated at 51% if the cancer is localized, according to a 2016 study of 9,387 cases that Dr. Brownell highlighted. But survival declines dramatically if it has spread to lymph nodes or distant sites (Ann Surg Oncol. 2016 Oct;23[11]:3564-71).

In recent years, researchers have linked 80% of Merkel cell carcinoma cases to the Merkel cell polyomavirus, he said. The virus normally inhabits our skin with no ill effects, he said. “We all have this virus on our skin. It’s everywhere, and even children have antibodies,” he said. But mutations can lead to Merkel cell carcinoma.

Does it matter if cases are polyomavirus positive or polyomavirus negative? Not really, Dr. Brownell said, since the presence of the virus doesn’t appear to affect overall prognosis. However, he said, serum antibody testing can be helpful in polyomavirus-positive patients because it offers insight into prognosis and tumor burden. For example, “if the baseline titer falls and then starts to go up, they’re likely to have a recurrence, and you’ll want to look out for that,” he said.

Dr. Brownell offered another bit of advice: Be prepared to respond to patients who worry that they have a contagious virus and could be a danger to others. The proper answer, he said, is this: “You don’t have to worry about infecting people. Your tumor is not making the virus, you’re not infectious, and we have the virus on us already.”

For more information about the antibody test, visit merkelcell.org/sero.

Dr. Brownell reported having no relevant disclosures. SDEF and this news organization are owned by the same parent company.

LAS VEGAS – Merkel cell carcinoma, an extremely rare form of skin cancer, is often caused by a subclinical virus that routinely inhabits the skin. Now, a serum test of virus antibody levels is offering insight into the state of the disease, according to one dermatologist.

“If you have these antibodies, you have a better prognosis. You can follow those antibodies to test for recurrence or progression,” Isaac Brownell, MD, PhD, of the Dermatology Branch of the National Institutes of Health said at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

The cancer appears in the skin’s Merkel cells, which contribute to our sense of touch by helping us to discriminate textures. “When you put your hand in your pocket, and you can tell the difference between the front and back of a quarter,” he said, “you’re using the Merkel cells in your fingertips.”

Only about 2,500 cases of Merkel cell carcinoma appear in the United States each year, Dr. Brownell said. It appears more often in elderly white patients, is more common in men than women, and is more likely among immunosuppressed patients, whose risk is increased 15- to 20-fold. Cases are more common in sunnier regions – at least in men – and lesions frequently appear on the head, face, and neck.

Five-year survival is estimated at 51% if the cancer is localized, according to a 2016 study of 9,387 cases that Dr. Brownell highlighted. But survival declines dramatically if it has spread to lymph nodes or distant sites (Ann Surg Oncol. 2016 Oct;23[11]:3564-71).

In recent years, researchers have linked 80% of Merkel cell carcinoma cases to the Merkel cell polyomavirus, he said. The virus normally inhabits our skin with no ill effects, he said. “We all have this virus on our skin. It’s everywhere, and even children have antibodies,” he said. But mutations can lead to Merkel cell carcinoma.

Does it matter if cases are polyomavirus positive or polyomavirus negative? Not really, Dr. Brownell said, since the presence of the virus doesn’t appear to affect overall prognosis. However, he said, serum antibody testing can be helpful in polyomavirus-positive patients because it offers insight into prognosis and tumor burden. For example, “if the baseline titer falls and then starts to go up, they’re likely to have a recurrence, and you’ll want to look out for that,” he said.

Dr. Brownell offered another bit of advice: Be prepared to respond to patients who worry that they have a contagious virus and could be a danger to others. The proper answer, he said, is this: “You don’t have to worry about infecting people. Your tumor is not making the virus, you’re not infectious, and we have the virus on us already.”

For more information about the antibody test, visit merkelcell.org/sero.

Dr. Brownell reported having no relevant disclosures. SDEF and this news organization are owned by the same parent company.

LAS VEGAS – Merkel cell carcinoma, an extremely rare form of skin cancer, is often caused by a subclinical virus that routinely inhabits the skin. Now, a serum test of virus antibody levels is offering insight into the state of the disease, according to one dermatologist.

“If you have these antibodies, you have a better prognosis. You can follow those antibodies to test for recurrence or progression,” Isaac Brownell, MD, PhD, of the Dermatology Branch of the National Institutes of Health said at Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.

The cancer appears in the skin’s Merkel cells, which contribute to our sense of touch by helping us to discriminate textures. “When you put your hand in your pocket, and you can tell the difference between the front and back of a quarter,” he said, “you’re using the Merkel cells in your fingertips.”

Only about 2,500 cases of Merkel cell carcinoma appear in the United States each year, Dr. Brownell said. It appears more often in elderly white patients, is more common in men than women, and is more likely among immunosuppressed patients, whose risk is increased 15- to 20-fold. Cases are more common in sunnier regions – at least in men – and lesions frequently appear on the head, face, and neck.

Five-year survival is estimated at 51% if the cancer is localized, according to a 2016 study of 9,387 cases that Dr. Brownell highlighted. But survival declines dramatically if it has spread to lymph nodes or distant sites (Ann Surg Oncol. 2016 Oct;23[11]:3564-71).

In recent years, researchers have linked 80% of Merkel cell carcinoma cases to the Merkel cell polyomavirus, he said. The virus normally inhabits our skin with no ill effects, he said. “We all have this virus on our skin. It’s everywhere, and even children have antibodies,” he said. But mutations can lead to Merkel cell carcinoma.

Does it matter if cases are polyomavirus positive or polyomavirus negative? Not really, Dr. Brownell said, since the presence of the virus doesn’t appear to affect overall prognosis. However, he said, serum antibody testing can be helpful in polyomavirus-positive patients because it offers insight into prognosis and tumor burden. For example, “if the baseline titer falls and then starts to go up, they’re likely to have a recurrence, and you’ll want to look out for that,” he said.

Dr. Brownell offered another bit of advice: Be prepared to respond to patients who worry that they have a contagious virus and could be a danger to others. The proper answer, he said, is this: “You don’t have to worry about infecting people. Your tumor is not making the virus, you’re not infectious, and we have the virus on us already.”

For more information about the antibody test, visit merkelcell.org/sero.

Dr. Brownell reported having no relevant disclosures. SDEF and this news organization are owned by the same parent company.

The Food and Drug Administration announced that it has approved cefiderocol (Fetroja), an IV antibacterial drug to treat complicated urinary tract infections (cUTIs), including kidney infections, caused by multidrug-resistant gram-negative microorganisms in patients 18 years of age or older.

The safety and effectiveness of cefiderocol was demonstrated in a pivotal study of 448 patients with cUTIs. Published results indicated that 73% of patients had resolution of symptoms and eradication of the bacteria approximately 7 days after completing treatment, compared with 55% in patients who received an alternative antibiotic.

The approval is for patients who have limited or no alternative treatment options and includes a label warning regarding cefiderocol’s higher all-cause mortality observed in comparison to patients treated with other antibiotics in a trial of critically ill patients having multidrug-resistant gram-negative bacterial infections (clinical trials. gov NCT02714595).

The cause of the increase in mortality has not been determined, according to the FDA. Some of the deaths in the study were attributable to worsening or complications of infection, or underlying comorbidities, in patients treated for hospital-acquired/ventilator-associated pneumonia (i.e., nosocomial pneumonia), bloodstream infections, or sepsis. Thus, safety and efficacy of cefiderocol has not been established for the treating these types of infections, according to the announcement.

Adverse reactions observed in patients treated with cefiderocol included diarrhea, constipation, nausea, vomiting, elevations in liver tests, rash, infusion-site reactions, and candidiasis. The FDA added that cefiderocol should not be used in persons known to have a severe hypersensitivity to beta-lactam antibacterial drugs.

“A key global challenge the FDA faces as a public health agency is addressing the threat of antimicrobial-resistant infections, like cUTIs. This approval represents another step forward in the FDA’s overall efforts to ensure safe and effective antimicrobial drugs are available to patients for treating infections,” John Farley, MD, acting director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research said in the FDA press statement.

Fetroja is a product of Shionogi.

[email protected]

The Food and Drug Administration announced that it has approved cefiderocol (Fetroja), an IV antibacterial drug to treat complicated urinary tract infections (cUTIs), including kidney infections, caused by multidrug-resistant gram-negative microorganisms in patients 18 years of age or older.

The safety and effectiveness of cefiderocol was demonstrated in a pivotal study of 448 patients with cUTIs. Published results indicated that 73% of patients had resolution of symptoms and eradication of the bacteria approximately 7 days after completing treatment, compared with 55% in patients who received an alternative antibiotic.

The approval is for patients who have limited or no alternative treatment options and includes a label warning regarding cefiderocol’s higher all-cause mortality observed in comparison to patients treated with other antibiotics in a trial of critically ill patients having multidrug-resistant gram-negative bacterial infections (clinical trials. gov NCT02714595).

The cause of the increase in mortality has not been determined, according to the FDA. Some of the deaths in the study were attributable to worsening or complications of infection, or underlying comorbidities, in patients treated for hospital-acquired/ventilator-associated pneumonia (i.e., nosocomial pneumonia), bloodstream infections, or sepsis. Thus, safety and efficacy of cefiderocol has not been established for the treating these types of infections, according to the announcement.

Adverse reactions observed in patients treated with cefiderocol included diarrhea, constipation, nausea, vomiting, elevations in liver tests, rash, infusion-site reactions, and candidiasis. The FDA added that cefiderocol should not be used in persons known to have a severe hypersensitivity to beta-lactam antibacterial drugs.

“A key global challenge the FDA faces as a public health agency is addressing the threat of antimicrobial-resistant infections, like cUTIs. This approval represents another step forward in the FDA’s overall efforts to ensure safe and effective antimicrobial drugs are available to patients for treating infections,” John Farley, MD, acting director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research said in the FDA press statement.

Fetroja is a product of Shionogi.

[email protected]

The Food and Drug Administration announced that it has approved cefiderocol (Fetroja), an IV antibacterial drug to treat complicated urinary tract infections (cUTIs), including kidney infections, caused by multidrug-resistant gram-negative microorganisms in patients 18 years of age or older.

The safety and effectiveness of cefiderocol was demonstrated in a pivotal study of 448 patients with cUTIs. Published results indicated that 73% of patients had resolution of symptoms and eradication of the bacteria approximately 7 days after completing treatment, compared with 55% in patients who received an alternative antibiotic.

The approval is for patients who have limited or no alternative treatment options and includes a label warning regarding cefiderocol’s higher all-cause mortality observed in comparison to patients treated with other antibiotics in a trial of critically ill patients having multidrug-resistant gram-negative bacterial infections (clinical trials. gov NCT02714595).

The cause of the increase in mortality has not been determined, according to the FDA. Some of the deaths in the study were attributable to worsening or complications of infection, or underlying comorbidities, in patients treated for hospital-acquired/ventilator-associated pneumonia (i.e., nosocomial pneumonia), bloodstream infections, or sepsis. Thus, safety and efficacy of cefiderocol has not been established for the treating these types of infections, according to the announcement.

Adverse reactions observed in patients treated with cefiderocol included diarrhea, constipation, nausea, vomiting, elevations in liver tests, rash, infusion-site reactions, and candidiasis. The FDA added that cefiderocol should not be used in persons known to have a severe hypersensitivity to beta-lactam antibacterial drugs.

“A key global challenge the FDA faces as a public health agency is addressing the threat of antimicrobial-resistant infections, like cUTIs. This approval represents another step forward in the FDA’s overall efforts to ensure safe and effective antimicrobial drugs are available to patients for treating infections,” John Farley, MD, acting director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research said in the FDA press statement.

Fetroja is a product of Shionogi.

[email protected]