For patients with suspected small-bowel bleeding, a three-variable scoring system predicted the diagnostic outcomes of video capsule endoscopy, according to the findings of a multicenter study.

“Admission to the hospital with overt bleeding and having a hemoglobin [level] of less than 6.4 g/dL were two positive predictors of a diagnosis. Being younger than 54 years old at the time of the video capsule endoscopy exam was a significant negative predictor of a diagnosis,” Neil B. Marya, MD, of the University of California, Los Angeles, and associates wrote in Techniques and Innovations in Gastrointestinal Endoscopy.

To develop the scoring system, they analyzed retrospective data from 162 adults with suspected small-bowel bleeding who received video capsule endoscopies at the University of Massachusetts or the University of California, Los Angeles, in 2016 or 2017. Most of these individuals were outpatients with occult gastrointestinal bleeding, but nearly one-third were inpatients with overt bleeding.

In all, 70 (43%) patients had a relevant finding on video capsule endoscopy, most frequently arteriovenous malformation (26%), blood (10.5%), or ulceration (10.5%). On multivariable analysis, the odds of positive video capsule endoscopy were significantly higher when patients had been admitted to the hospital with overt bleeding (adjusted odds ratio, 2.38; 95% confidence interval, 1.05-5.39) or had a baseline hemoglobin level less than or equal to 6.4 g/dL (aOR, 2.68; 95% CI, 1.11-6.48). In contrast, patients who were younger than 54 years were significantly less likely to have diagnostic lesions (aOR, 0.25; 95% CI, 0.11-0.58). After designating these variables as A, B, and C, respectively, the researchers derived the following equation to produce the score: (0.87 x A) + (0.99 x B) – (1.38 x C). Each variable was scored as 1 (present) or 0 (absent). Based on this equation, the highest score possible score was 1.86, and the lowest possible score was –1.38.

The researchers validated this scoring system by analyzing data from 152 adults with suspected small-bowel bleeding who were examined prospectively at the two centers. The development and validation cohorts resembled each other except that the validation cohort had lower mean hemoglobin levels (8.2 g/dL versus 9.0 g/dL in the development cohort; P < .01) and higher mean blood urea nitrogen levels (25.3 mg/dL vs. 19.9 mg per dL; P =.03). Receiver operating curves were similar between the two groups (respective C-statistics, 0.70 and 0.69; P = .91).

However, the scoring system’s maximum specificity was only 30.6%, yielding a positive predictive value of only 48.6%. The associated cutoff score was greater than or equal to 0. At this cutoff, sensitivity was “at least 90%,” and negative predictive value was 83.6%. Thus, the scoring system is best suited for identifying patients who are unlikely to have a diagnostic lesion found on video capsule endoscopy, the researchers said.

“Patients with scores of less than 0 could conceivably have capsule examinations deferred,” they concluded. “For example, consider a clinician taking care of a hospitalized patient who meets criteria for undergoing video capsule endoscopy for the indication of suspected small intestinal bleeding, but finds that the patient has a suspected small-bowel bleeding capsule diagnosis score of less than 0. The clinician could decide to have their patient undergo the video capsule endoscopy as an outpatient, since the likelihood of detecting an actionable lesion is low. This decision could have a [beneficial] financial impact.”

No external funding sources were reported. Dr. Marya disclosed a recent consulting relationship with AnX Robotica. Two coinvestigators disclosed a consulting relationship with Medtronic and research support from Olympus and Medtronic.

SOURCE: Marya NB et al. Tech Innov Gastrointest Endosc. 2020 Jun 19. doi: 10.1016/j.tige.2020.06.001.

For patients with suspected small-bowel bleeding, a three-variable scoring system predicted the diagnostic outcomes of video capsule endoscopy, according to the findings of a multicenter study.

“Admission to the hospital with overt bleeding and having a hemoglobin [level] of less than 6.4 g/dL were two positive predictors of a diagnosis. Being younger than 54 years old at the time of the video capsule endoscopy exam was a significant negative predictor of a diagnosis,” Neil B. Marya, MD, of the University of California, Los Angeles, and associates wrote in Techniques and Innovations in Gastrointestinal Endoscopy.

To develop the scoring system, they analyzed retrospective data from 162 adults with suspected small-bowel bleeding who received video capsule endoscopies at the University of Massachusetts or the University of California, Los Angeles, in 2016 or 2017. Most of these individuals were outpatients with occult gastrointestinal bleeding, but nearly one-third were inpatients with overt bleeding.

In all, 70 (43%) patients had a relevant finding on video capsule endoscopy, most frequently arteriovenous malformation (26%), blood (10.5%), or ulceration (10.5%). On multivariable analysis, the odds of positive video capsule endoscopy were significantly higher when patients had been admitted to the hospital with overt bleeding (adjusted odds ratio, 2.38; 95% confidence interval, 1.05-5.39) or had a baseline hemoglobin level less than or equal to 6.4 g/dL (aOR, 2.68; 95% CI, 1.11-6.48). In contrast, patients who were younger than 54 years were significantly less likely to have diagnostic lesions (aOR, 0.25; 95% CI, 0.11-0.58). After designating these variables as A, B, and C, respectively, the researchers derived the following equation to produce the score: (0.87 x A) + (0.99 x B) – (1.38 x C). Each variable was scored as 1 (present) or 0 (absent). Based on this equation, the highest score possible score was 1.86, and the lowest possible score was –1.38.

The researchers validated this scoring system by analyzing data from 152 adults with suspected small-bowel bleeding who were examined prospectively at the two centers. The development and validation cohorts resembled each other except that the validation cohort had lower mean hemoglobin levels (8.2 g/dL versus 9.0 g/dL in the development cohort; P < .01) and higher mean blood urea nitrogen levels (25.3 mg/dL vs. 19.9 mg per dL; P =.03). Receiver operating curves were similar between the two groups (respective C-statistics, 0.70 and 0.69; P = .91).

However, the scoring system’s maximum specificity was only 30.6%, yielding a positive predictive value of only 48.6%. The associated cutoff score was greater than or equal to 0. At this cutoff, sensitivity was “at least 90%,” and negative predictive value was 83.6%. Thus, the scoring system is best suited for identifying patients who are unlikely to have a diagnostic lesion found on video capsule endoscopy, the researchers said.

“Patients with scores of less than 0 could conceivably have capsule examinations deferred,” they concluded. “For example, consider a clinician taking care of a hospitalized patient who meets criteria for undergoing video capsule endoscopy for the indication of suspected small intestinal bleeding, but finds that the patient has a suspected small-bowel bleeding capsule diagnosis score of less than 0. The clinician could decide to have their patient undergo the video capsule endoscopy as an outpatient, since the likelihood of detecting an actionable lesion is low. This decision could have a [beneficial] financial impact.”

No external funding sources were reported. Dr. Marya disclosed a recent consulting relationship with AnX Robotica. Two coinvestigators disclosed a consulting relationship with Medtronic and research support from Olympus and Medtronic.

SOURCE: Marya NB et al. Tech Innov Gastrointest Endosc. 2020 Jun 19. doi: 10.1016/j.tige.2020.06.001.

For patients with suspected small-bowel bleeding, a three-variable scoring system predicted the diagnostic outcomes of video capsule endoscopy, according to the findings of a multicenter study.

“Admission to the hospital with overt bleeding and having a hemoglobin [level] of less than 6.4 g/dL were two positive predictors of a diagnosis. Being younger than 54 years old at the time of the video capsule endoscopy exam was a significant negative predictor of a diagnosis,” Neil B. Marya, MD, of the University of California, Los Angeles, and associates wrote in Techniques and Innovations in Gastrointestinal Endoscopy.

To develop the scoring system, they analyzed retrospective data from 162 adults with suspected small-bowel bleeding who received video capsule endoscopies at the University of Massachusetts or the University of California, Los Angeles, in 2016 or 2017. Most of these individuals were outpatients with occult gastrointestinal bleeding, but nearly one-third were inpatients with overt bleeding.

In all, 70 (43%) patients had a relevant finding on video capsule endoscopy, most frequently arteriovenous malformation (26%), blood (10.5%), or ulceration (10.5%). On multivariable analysis, the odds of positive video capsule endoscopy were significantly higher when patients had been admitted to the hospital with overt bleeding (adjusted odds ratio, 2.38; 95% confidence interval, 1.05-5.39) or had a baseline hemoglobin level less than or equal to 6.4 g/dL (aOR, 2.68; 95% CI, 1.11-6.48). In contrast, patients who were younger than 54 years were significantly less likely to have diagnostic lesions (aOR, 0.25; 95% CI, 0.11-0.58). After designating these variables as A, B, and C, respectively, the researchers derived the following equation to produce the score: (0.87 x A) + (0.99 x B) – (1.38 x C). Each variable was scored as 1 (present) or 0 (absent). Based on this equation, the highest score possible score was 1.86, and the lowest possible score was –1.38.

The researchers validated this scoring system by analyzing data from 152 adults with suspected small-bowel bleeding who were examined prospectively at the two centers. The development and validation cohorts resembled each other except that the validation cohort had lower mean hemoglobin levels (8.2 g/dL versus 9.0 g/dL in the development cohort; P < .01) and higher mean blood urea nitrogen levels (25.3 mg/dL vs. 19.9 mg per dL; P =.03). Receiver operating curves were similar between the two groups (respective C-statistics, 0.70 and 0.69; P = .91).

However, the scoring system’s maximum specificity was only 30.6%, yielding a positive predictive value of only 48.6%. The associated cutoff score was greater than or equal to 0. At this cutoff, sensitivity was “at least 90%,” and negative predictive value was 83.6%. Thus, the scoring system is best suited for identifying patients who are unlikely to have a diagnostic lesion found on video capsule endoscopy, the researchers said.

“Patients with scores of less than 0 could conceivably have capsule examinations deferred,” they concluded. “For example, consider a clinician taking care of a hospitalized patient who meets criteria for undergoing video capsule endoscopy for the indication of suspected small intestinal bleeding, but finds that the patient has a suspected small-bowel bleeding capsule diagnosis score of less than 0. The clinician could decide to have their patient undergo the video capsule endoscopy as an outpatient, since the likelihood of detecting an actionable lesion is low. This decision could have a [beneficial] financial impact.”

No external funding sources were reported. Dr. Marya disclosed a recent consulting relationship with AnX Robotica. Two coinvestigators disclosed a consulting relationship with Medtronic and research support from Olympus and Medtronic.

SOURCE: Marya NB et al. Tech Innov Gastrointest Endosc. 2020 Jun 19. doi: 10.1016/j.tige.2020.06.001.

Atezolizumab (Tecentriq) plus paclitaxel is ineffective in patients with previously untreated, inoperable, locally advanced or metastatic triple-negative breast cancer (TNBC), according to an alert from the Food and Drug Administration.

The FDA said the phase 3 IMpassion131 trial showed that atezolizumab plus paclitaxel did not significantly reduce the risk of cancer progression and death, when compared with paclitaxel plus placebo, in programmed death-ligand 1 (PD-L1)–positive patients.

“Additionally, interim overall survival results favored paclitaxel plus placebo over paclitaxel plus atezolizumab in both the PD-L1-positive population and total population,” the FDA noted.

As a result, “health care professionals should not replace paclitaxel protein-bound (Abraxane) with paclitaxel in clinical practice,” the FDA advised.

Atezolizumab is approved for use in combination with protein-bound paclitaxel, also known as nanoparticle albumin–bound paclitaxel (nab-paclitaxel), to treat patients with unresectable, locally advanced, or metastatic TNBC whose tumors express PD-L1, as detected by an FDA-approved test. The combination was granted accelerated approval for this indication last year.

Atezolizumab plus nab-paclitaxel is the combination most often used in PD-L1-positive TNBC, as opposed to atezolizumab and unbound paclitaxel, said Melinda L. Telli, MD, an associate professor of medicine and director of the Stanford Cancer Institute Breast Cancer Program at Stanford (Calif.) University.

However, as the FDA noted, “continued approval of atezolizumab in combination with [nab-paclitaxel] may be contingent on proven benefit of the treatment in additional trials.”

Dr. Telli explained that atezolizumab was granted accelerated approval for the treatment of PD-L1-positive TNBC based on results of the phase 3 IMpassion130 trial, which compared nab-paclitaxel plus atezolizumab with nab-paclitaxel plus placebo.

“Additional data from IMpassion131 was hoped to be used to support the conversion of the accelerated approval to a full approval. Since IMpassion131 was negative, it unfortunately places the status of atezolizumab in [TNBC] in jeopardy as the benefits were not corroborated. The FDA may move to revoke the approval of atezolizumab for [TNBC],” Dr. Telli said.

In its alert, the FDA stated that it “will review the findings of IMpassion131 and will communicate new information regarding the IMpassion131 results and any potential changes to prescribing information.”

“We need to wait for full presentation and publication of the study results, but, in my assessment, the negative results in IMpassion131 are most likely due to differences in patient selection [from IMpassion130],” Dr. Telli said.

Results from IMpassion131 are scheduled to be presented at the ESMO Virtual Congress 2020.

The IMpassion trials were funded by Roche, maker of atezolizumab. Dr. Telli disclosed relationships with AbbVie, AstraZeneca, Merck, PharmaMar, Pfizer, and Tesaro.
 

[email protected]

Atezolizumab (Tecentriq) plus paclitaxel is ineffective in patients with previously untreated, inoperable, locally advanced or metastatic triple-negative breast cancer (TNBC), according to an alert from the Food and Drug Administration.

The FDA said the phase 3 IMpassion131 trial showed that atezolizumab plus paclitaxel did not significantly reduce the risk of cancer progression and death, when compared with paclitaxel plus placebo, in programmed death-ligand 1 (PD-L1)–positive patients.

“Additionally, interim overall survival results favored paclitaxel plus placebo over paclitaxel plus atezolizumab in both the PD-L1-positive population and total population,” the FDA noted.

As a result, “health care professionals should not replace paclitaxel protein-bound (Abraxane) with paclitaxel in clinical practice,” the FDA advised.

Atezolizumab is approved for use in combination with protein-bound paclitaxel, also known as nanoparticle albumin–bound paclitaxel (nab-paclitaxel), to treat patients with unresectable, locally advanced, or metastatic TNBC whose tumors express PD-L1, as detected by an FDA-approved test. The combination was granted accelerated approval for this indication last year.

Atezolizumab plus nab-paclitaxel is the combination most often used in PD-L1-positive TNBC, as opposed to atezolizumab and unbound paclitaxel, said Melinda L. Telli, MD, an associate professor of medicine and director of the Stanford Cancer Institute Breast Cancer Program at Stanford (Calif.) University.

However, as the FDA noted, “continued approval of atezolizumab in combination with [nab-paclitaxel] may be contingent on proven benefit of the treatment in additional trials.”

Dr. Telli explained that atezolizumab was granted accelerated approval for the treatment of PD-L1-positive TNBC based on results of the phase 3 IMpassion130 trial, which compared nab-paclitaxel plus atezolizumab with nab-paclitaxel plus placebo.

“Additional data from IMpassion131 was hoped to be used to support the conversion of the accelerated approval to a full approval. Since IMpassion131 was negative, it unfortunately places the status of atezolizumab in [TNBC] in jeopardy as the benefits were not corroborated. The FDA may move to revoke the approval of atezolizumab for [TNBC],” Dr. Telli said.

In its alert, the FDA stated that it “will review the findings of IMpassion131 and will communicate new information regarding the IMpassion131 results and any potential changes to prescribing information.”

“We need to wait for full presentation and publication of the study results, but, in my assessment, the negative results in IMpassion131 are most likely due to differences in patient selection [from IMpassion130],” Dr. Telli said.

Results from IMpassion131 are scheduled to be presented at the ESMO Virtual Congress 2020.

The IMpassion trials were funded by Roche, maker of atezolizumab. Dr. Telli disclosed relationships with AbbVie, AstraZeneca, Merck, PharmaMar, Pfizer, and Tesaro.
 

[email protected]

Atezolizumab (Tecentriq) plus paclitaxel is ineffective in patients with previously untreated, inoperable, locally advanced or metastatic triple-negative breast cancer (TNBC), according to an alert from the Food and Drug Administration.

The FDA said the phase 3 IMpassion131 trial showed that atezolizumab plus paclitaxel did not significantly reduce the risk of cancer progression and death, when compared with paclitaxel plus placebo, in programmed death-ligand 1 (PD-L1)–positive patients.

“Additionally, interim overall survival results favored paclitaxel plus placebo over paclitaxel plus atezolizumab in both the PD-L1-positive population and total population,” the FDA noted.

As a result, “health care professionals should not replace paclitaxel protein-bound (Abraxane) with paclitaxel in clinical practice,” the FDA advised.

Atezolizumab is approved for use in combination with protein-bound paclitaxel, also known as nanoparticle albumin–bound paclitaxel (nab-paclitaxel), to treat patients with unresectable, locally advanced, or metastatic TNBC whose tumors express PD-L1, as detected by an FDA-approved test. The combination was granted accelerated approval for this indication last year.

Atezolizumab plus nab-paclitaxel is the combination most often used in PD-L1-positive TNBC, as opposed to atezolizumab and unbound paclitaxel, said Melinda L. Telli, MD, an associate professor of medicine and director of the Stanford Cancer Institute Breast Cancer Program at Stanford (Calif.) University.

However, as the FDA noted, “continued approval of atezolizumab in combination with [nab-paclitaxel] may be contingent on proven benefit of the treatment in additional trials.”

Dr. Telli explained that atezolizumab was granted accelerated approval for the treatment of PD-L1-positive TNBC based on results of the phase 3 IMpassion130 trial, which compared nab-paclitaxel plus atezolizumab with nab-paclitaxel plus placebo.

“Additional data from IMpassion131 was hoped to be used to support the conversion of the accelerated approval to a full approval. Since IMpassion131 was negative, it unfortunately places the status of atezolizumab in [TNBC] in jeopardy as the benefits were not corroborated. The FDA may move to revoke the approval of atezolizumab for [TNBC],” Dr. Telli said.

In its alert, the FDA stated that it “will review the findings of IMpassion131 and will communicate new information regarding the IMpassion131 results and any potential changes to prescribing information.”

“We need to wait for full presentation and publication of the study results, but, in my assessment, the negative results in IMpassion131 are most likely due to differences in patient selection [from IMpassion130],” Dr. Telli said.

Results from IMpassion131 are scheduled to be presented at the ESMO Virtual Congress 2020.

The IMpassion trials were funded by Roche, maker of atezolizumab. Dr. Telli disclosed relationships with AbbVie, AstraZeneca, Merck, PharmaMar, Pfizer, and Tesaro.
 

[email protected]

A history of liver transplant conveyed no increased risk of death from COVID-19 infections, according to data from a multicenter cohort study of 151 transplant recipients who became infected.

Although current data suggest a possible increased risk of adverse outcomes if liver transplant patients develop COVID-19 infections, the effects remain unclear, wrote Gwilym J. Webb, PhD, of the University of Oxford (England) and colleagues.

In a study published in the Lancet Gastroenterology & Hepatology, the researchers identified adults from 18 countries who had laboratory-confirmed COVID-19 infections between March 25, 2020, and June 26, 2020. The average age of the patients was 60 years, and 68% were men. A contemporaneous group of 627 consecutive adults with confirmed COVID-19 infections who had not undergone liver transplants served as controls.

Overall, 28 of the liver transplant patients and 167 of the controls died (19% vs. 27%; P = .046).

In addition, no differences appeared between infected transplant patients and infected controls in terms of hospitalization (82% vs. 76%) and the need for intensive care (31% vs. 30%), although the transplant patients were significantly more likely to require invasive ventilation (20% vs. 5%).

However, in a multivariate analysis, older age, serum creatinine concentration, and the presence of nonliver cancers were independently associated with increased risk of death in the liver transplant patients, with odds ratios of 1.06 for each year increase in age, 1.57 for each mg/dL increase in serum creatinine concentration, and 18.30 with the presence of a nonliver cancer.

The study findings were limited by several factors including the potential overreporting of severe COVID-19 cases because of reporting bias in the transplant registry, as well as the inability of the sample size to rule out mortality differences, the differences in comorbidities between the transplant patients and controls, and the impact of unmeasured confounding variables such as diet, physical activity, or fibrosis or cirrhosis in recipient grafts, the researchers noted. However, the results suggest that a history of liver transplantation does not increase the risk of death following COVID-19 infection, they wrote.

“Thus, traditional risk factors for adverse outcomes from COVID-19 should be preferentially considered when considering the risks and benefits of hospital attendance, immunosuppression, and social-distancing requirements for liver transplant recipients during the ongoing COVID-19 pandemic,” they concluded.

Focus on comorbidities and combined transplants

“Given that age and presence of comorbidities were significantly associated with risk of death in the cohorts of patients who had and had not undergone liver transplantation, greater emphasis should be placed on other coexisting comorbidities, rather than transplantation status per se, when risk-stratifying liver transplant recipients,” the researchers noted. “Indirectly, these findings suggest that liver transplantation, where indicated, should not be delayed during the COVID-19 pandemic, and that supportive care should not be limited for patients with existing liver transplants with COVID-19,” they suggested.

Given the high prevalence of COVID-19 in many countries, “it is inevitable that liver transplant patients will become infected,” said Wajahat Mehal, MD, of Yale University, New Haven, Conn., in an interview.

Going forward, “it is important to know the natural history of COVID in the immunocompromised population,” he emphasized.

One of the study limitations was the lack of data on how patients’ immunosuppression regimens were changed, if at all, while they were infected. “Since some other immunocompromised patients have had a higher rate of complications [in the wake of COVID-19 infections], I was pleasantly surprised that liver transplant recipients did so well,” Dr. Mehal said.

Dr. Mehal noted that additional research is needed to promote safety in patients with liver disease in the context of COVID-19. “It would be important to evaluate combined transplants, particularly combined liver/kidney transplants,” he said.

The study was supported by the European Association for the Study of the Liver, the National Institutes of Health, and the United Kingdom National Institute for Health Research. Lead author Dr. Webb had no financial conflicts to disclose. One coauthor disclosed unrelated fees from AbbVie and grants from the Fondation du Centre Hospitalier de l’Université de Montréal. Dr. Mehal had no financial conflicts to disclose.

SOURCE: Webb GJ et al. Lancet Gastroenterol Hepatol. 2020 Aug 28. doi: 10.1016/ S2468-1253(20)30271-5.

A history of liver transplant conveyed no increased risk of death from COVID-19 infections, according to data from a multicenter cohort study of 151 transplant recipients who became infected.

Although current data suggest a possible increased risk of adverse outcomes if liver transplant patients develop COVID-19 infections, the effects remain unclear, wrote Gwilym J. Webb, PhD, of the University of Oxford (England) and colleagues.

In a study published in the Lancet Gastroenterology & Hepatology, the researchers identified adults from 18 countries who had laboratory-confirmed COVID-19 infections between March 25, 2020, and June 26, 2020. The average age of the patients was 60 years, and 68% were men. A contemporaneous group of 627 consecutive adults with confirmed COVID-19 infections who had not undergone liver transplants served as controls.

Overall, 28 of the liver transplant patients and 167 of the controls died (19% vs. 27%; P = .046).

In addition, no differences appeared between infected transplant patients and infected controls in terms of hospitalization (82% vs. 76%) and the need for intensive care (31% vs. 30%), although the transplant patients were significantly more likely to require invasive ventilation (20% vs. 5%).

However, in a multivariate analysis, older age, serum creatinine concentration, and the presence of nonliver cancers were independently associated with increased risk of death in the liver transplant patients, with odds ratios of 1.06 for each year increase in age, 1.57 for each mg/dL increase in serum creatinine concentration, and 18.30 with the presence of a nonliver cancer.

The study findings were limited by several factors including the potential overreporting of severe COVID-19 cases because of reporting bias in the transplant registry, as well as the inability of the sample size to rule out mortality differences, the differences in comorbidities between the transplant patients and controls, and the impact of unmeasured confounding variables such as diet, physical activity, or fibrosis or cirrhosis in recipient grafts, the researchers noted. However, the results suggest that a history of liver transplantation does not increase the risk of death following COVID-19 infection, they wrote.

“Thus, traditional risk factors for adverse outcomes from COVID-19 should be preferentially considered when considering the risks and benefits of hospital attendance, immunosuppression, and social-distancing requirements for liver transplant recipients during the ongoing COVID-19 pandemic,” they concluded.

Focus on comorbidities and combined transplants

“Given that age and presence of comorbidities were significantly associated with risk of death in the cohorts of patients who had and had not undergone liver transplantation, greater emphasis should be placed on other coexisting comorbidities, rather than transplantation status per se, when risk-stratifying liver transplant recipients,” the researchers noted. “Indirectly, these findings suggest that liver transplantation, where indicated, should not be delayed during the COVID-19 pandemic, and that supportive care should not be limited for patients with existing liver transplants with COVID-19,” they suggested.

Given the high prevalence of COVID-19 in many countries, “it is inevitable that liver transplant patients will become infected,” said Wajahat Mehal, MD, of Yale University, New Haven, Conn., in an interview.

Going forward, “it is important to know the natural history of COVID in the immunocompromised population,” he emphasized.

One of the study limitations was the lack of data on how patients’ immunosuppression regimens were changed, if at all, while they were infected. “Since some other immunocompromised patients have had a higher rate of complications [in the wake of COVID-19 infections], I was pleasantly surprised that liver transplant recipients did so well,” Dr. Mehal said.

Dr. Mehal noted that additional research is needed to promote safety in patients with liver disease in the context of COVID-19. “It would be important to evaluate combined transplants, particularly combined liver/kidney transplants,” he said.

The study was supported by the European Association for the Study of the Liver, the National Institutes of Health, and the United Kingdom National Institute for Health Research. Lead author Dr. Webb had no financial conflicts to disclose. One coauthor disclosed unrelated fees from AbbVie and grants from the Fondation du Centre Hospitalier de l’Université de Montréal. Dr. Mehal had no financial conflicts to disclose.

SOURCE: Webb GJ et al. Lancet Gastroenterol Hepatol. 2020 Aug 28. doi: 10.1016/ S2468-1253(20)30271-5.

A history of liver transplant conveyed no increased risk of death from COVID-19 infections, according to data from a multicenter cohort study of 151 transplant recipients who became infected.

Although current data suggest a possible increased risk of adverse outcomes if liver transplant patients develop COVID-19 infections, the effects remain unclear, wrote Gwilym J. Webb, PhD, of the University of Oxford (England) and colleagues.

In a study published in the Lancet Gastroenterology & Hepatology, the researchers identified adults from 18 countries who had laboratory-confirmed COVID-19 infections between March 25, 2020, and June 26, 2020. The average age of the patients was 60 years, and 68% were men. A contemporaneous group of 627 consecutive adults with confirmed COVID-19 infections who had not undergone liver transplants served as controls.

Overall, 28 of the liver transplant patients and 167 of the controls died (19% vs. 27%; P = .046).

In addition, no differences appeared between infected transplant patients and infected controls in terms of hospitalization (82% vs. 76%) and the need for intensive care (31% vs. 30%), although the transplant patients were significantly more likely to require invasive ventilation (20% vs. 5%).

However, in a multivariate analysis, older age, serum creatinine concentration, and the presence of nonliver cancers were independently associated with increased risk of death in the liver transplant patients, with odds ratios of 1.06 for each year increase in age, 1.57 for each mg/dL increase in serum creatinine concentration, and 18.30 with the presence of a nonliver cancer.

The study findings were limited by several factors including the potential overreporting of severe COVID-19 cases because of reporting bias in the transplant registry, as well as the inability of the sample size to rule out mortality differences, the differences in comorbidities between the transplant patients and controls, and the impact of unmeasured confounding variables such as diet, physical activity, or fibrosis or cirrhosis in recipient grafts, the researchers noted. However, the results suggest that a history of liver transplantation does not increase the risk of death following COVID-19 infection, they wrote.

“Thus, traditional risk factors for adverse outcomes from COVID-19 should be preferentially considered when considering the risks and benefits of hospital attendance, immunosuppression, and social-distancing requirements for liver transplant recipients during the ongoing COVID-19 pandemic,” they concluded.

Focus on comorbidities and combined transplants

“Given that age and presence of comorbidities were significantly associated with risk of death in the cohorts of patients who had and had not undergone liver transplantation, greater emphasis should be placed on other coexisting comorbidities, rather than transplantation status per se, when risk-stratifying liver transplant recipients,” the researchers noted. “Indirectly, these findings suggest that liver transplantation, where indicated, should not be delayed during the COVID-19 pandemic, and that supportive care should not be limited for patients with existing liver transplants with COVID-19,” they suggested.

Given the high prevalence of COVID-19 in many countries, “it is inevitable that liver transplant patients will become infected,” said Wajahat Mehal, MD, of Yale University, New Haven, Conn., in an interview.

Going forward, “it is important to know the natural history of COVID in the immunocompromised population,” he emphasized.

One of the study limitations was the lack of data on how patients’ immunosuppression regimens were changed, if at all, while they were infected. “Since some other immunocompromised patients have had a higher rate of complications [in the wake of COVID-19 infections], I was pleasantly surprised that liver transplant recipients did so well,” Dr. Mehal said.

Dr. Mehal noted that additional research is needed to promote safety in patients with liver disease in the context of COVID-19. “It would be important to evaluate combined transplants, particularly combined liver/kidney transplants,” he said.

The study was supported by the European Association for the Study of the Liver, the National Institutes of Health, and the United Kingdom National Institute for Health Research. Lead author Dr. Webb had no financial conflicts to disclose. One coauthor disclosed unrelated fees from AbbVie and grants from the Fondation du Centre Hospitalier de l’Université de Montréal. Dr. Mehal had no financial conflicts to disclose.

SOURCE: Webb GJ et al. Lancet Gastroenterol Hepatol. 2020 Aug 28. doi: 10.1016/ S2468-1253(20)30271-5.

Knowing it may be met with some skepticism, the Trump administration Thursday announced a sweeping plan that officials say will transform health care in rural America.

Even before the coronavirus pandemic reached into the nation’s less-populated regions, rural Americans were sicker, poorer, and older than the rest of the country. Hospitals are shuttering at record rates, and health care experts have long called for changes.

The new plan, released by the Department of Health & Human Services Secretary Alex M. Azar, II, acknowledges the gaps in health care and other problems facing rural America. It lists a litany of projects and directives, with many already underway or announced within federal agencies.

“We cannot just tinker around the edges of a rural healthcare system that has struggled for too long,” Azar said in a prepared statement.

Yet, that is exactly what experts say the administration continues to do.

“They tinker around the edges,” said Tommy Barnhart, former president of the National Rural Health Association. And he added, “there’s a lot of political hype” that has happened under President Trump, as well as previous presidents.

In the past few months, rural health care has increasingly become a focus for Mr. Trump, whose polling numbers are souring as COVID-19 kills hundreds of Americans every day, drives down restaurant demand for some farm products, and spreads through meatpacking plants. Rural states including Iowa and the Dakotas are reporting the latest surges in cases.

This announcement comes in response to Mr. Trump’s executive order last month calling for improved rural health and telehealth access. Earlier this week, three federal agencies also announced they would team up to address gaps in rural broadband service – a key need because large portions of the plan seek to expand telehealth.

The plan is more than 70 pages long and the word “telehealth” appears more than 90 times, with a focus on projects across HHS, including the Health Resources and Services Administration and the Centers for Medicare & Medicaid Services.

Mr. Barnhart said CMS has passed some public health emergency waivers since the beginning of the pandemic that helped rural facilities get more funding, including one that specifically was designed to provide additional money for telehealth services. However, those waivers are set to expire when the coronavirus emergency ends. Officials have not yet set a date for when the federal emergency will end.

Andrew Jay Schwartzman, senior counselor to the Benton Institute for Broadband & Society, a private foundation that works to ensure greater Internet access, said there are multiple challenges with implementing telehealth across the nation. Many initiatives for robust telehealth programs need fast bandwidth, yet getting the money and setting up the necessary infrastructure is very difficult, he said.

“It will be a long time before this kind of technology will be readily available to much of the country,” he said.

Ge Bai, associate professor of accounting and health policy at Johns Hopkins University in Baltimore, noted that telehealth was short on funding in the HHS initiative. However, she said, the focus on telehealth, as well as a proposed shift in payment for small rural hospitals and changing workforce licensing requirements, had good potential.

“We are so close to the election that this is probably more of a messaging issue to cater to rural residents,” Ms. Bai said. “But it doesn’t matter who will be president. This report will give the next administration useful guidance.”

The American Hospital Association, representing 5,000 hospitals nationwide, sent a letter to Mr. Trump last week recommending a host of steps the administration could take. As of late Thursday, AHA was still reviewing the HHS plan but said it was “encouraged by the increased attention on rural health care.”

Buried within the HHS announcement are technical initiatives, such as a contract to help clinics and hospitals integrate care, and detailed efforts to address gaps in care, including a proposal to increase funding for school-based mental health programs in the president’s 2021 budget.

A senior HHS official said that, while some actions have been taken in recent months to improve rural health — such as the $11 billion provided to rural hospitals through coronavirus relief funding — more is needed.

“We’re putting our stake in the ground that the time for talk is over,” he said. “We’re going to move forward.”

Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of KFF (Kaiser Family Foundation), which is not affiliated with Kaiser Permanente.

Knowing it may be met with some skepticism, the Trump administration Thursday announced a sweeping plan that officials say will transform health care in rural America.

Even before the coronavirus pandemic reached into the nation’s less-populated regions, rural Americans were sicker, poorer, and older than the rest of the country. Hospitals are shuttering at record rates, and health care experts have long called for changes.

The new plan, released by the Department of Health & Human Services Secretary Alex M. Azar, II, acknowledges the gaps in health care and other problems facing rural America. It lists a litany of projects and directives, with many already underway or announced within federal agencies.

“We cannot just tinker around the edges of a rural healthcare system that has struggled for too long,” Azar said in a prepared statement.

Yet, that is exactly what experts say the administration continues to do.

“They tinker around the edges,” said Tommy Barnhart, former president of the National Rural Health Association. And he added, “there’s a lot of political hype” that has happened under President Trump, as well as previous presidents.

In the past few months, rural health care has increasingly become a focus for Mr. Trump, whose polling numbers are souring as COVID-19 kills hundreds of Americans every day, drives down restaurant demand for some farm products, and spreads through meatpacking plants. Rural states including Iowa and the Dakotas are reporting the latest surges in cases.

This announcement comes in response to Mr. Trump’s executive order last month calling for improved rural health and telehealth access. Earlier this week, three federal agencies also announced they would team up to address gaps in rural broadband service – a key need because large portions of the plan seek to expand telehealth.

The plan is more than 70 pages long and the word “telehealth” appears more than 90 times, with a focus on projects across HHS, including the Health Resources and Services Administration and the Centers for Medicare & Medicaid Services.

Mr. Barnhart said CMS has passed some public health emergency waivers since the beginning of the pandemic that helped rural facilities get more funding, including one that specifically was designed to provide additional money for telehealth services. However, those waivers are set to expire when the coronavirus emergency ends. Officials have not yet set a date for when the federal emergency will end.

Andrew Jay Schwartzman, senior counselor to the Benton Institute for Broadband & Society, a private foundation that works to ensure greater Internet access, said there are multiple challenges with implementing telehealth across the nation. Many initiatives for robust telehealth programs need fast bandwidth, yet getting the money and setting up the necessary infrastructure is very difficult, he said.

“It will be a long time before this kind of technology will be readily available to much of the country,” he said.

Ge Bai, associate professor of accounting and health policy at Johns Hopkins University in Baltimore, noted that telehealth was short on funding in the HHS initiative. However, she said, the focus on telehealth, as well as a proposed shift in payment for small rural hospitals and changing workforce licensing requirements, had good potential.

“We are so close to the election that this is probably more of a messaging issue to cater to rural residents,” Ms. Bai said. “But it doesn’t matter who will be president. This report will give the next administration useful guidance.”

The American Hospital Association, representing 5,000 hospitals nationwide, sent a letter to Mr. Trump last week recommending a host of steps the administration could take. As of late Thursday, AHA was still reviewing the HHS plan but said it was “encouraged by the increased attention on rural health care.”

Buried within the HHS announcement are technical initiatives, such as a contract to help clinics and hospitals integrate care, and detailed efforts to address gaps in care, including a proposal to increase funding for school-based mental health programs in the president’s 2021 budget.

A senior HHS official said that, while some actions have been taken in recent months to improve rural health — such as the $11 billion provided to rural hospitals through coronavirus relief funding — more is needed.

“We’re putting our stake in the ground that the time for talk is over,” he said. “We’re going to move forward.”

Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of KFF (Kaiser Family Foundation), which is not affiliated with Kaiser Permanente.

Knowing it may be met with some skepticism, the Trump administration Thursday announced a sweeping plan that officials say will transform health care in rural America.

Even before the coronavirus pandemic reached into the nation’s less-populated regions, rural Americans were sicker, poorer, and older than the rest of the country. Hospitals are shuttering at record rates, and health care experts have long called for changes.

The new plan, released by the Department of Health & Human Services Secretary Alex M. Azar, II, acknowledges the gaps in health care and other problems facing rural America. It lists a litany of projects and directives, with many already underway or announced within federal agencies.

“We cannot just tinker around the edges of a rural healthcare system that has struggled for too long,” Azar said in a prepared statement.

Yet, that is exactly what experts say the administration continues to do.

“They tinker around the edges,” said Tommy Barnhart, former president of the National Rural Health Association. And he added, “there’s a lot of political hype” that has happened under President Trump, as well as previous presidents.

In the past few months, rural health care has increasingly become a focus for Mr. Trump, whose polling numbers are souring as COVID-19 kills hundreds of Americans every day, drives down restaurant demand for some farm products, and spreads through meatpacking plants. Rural states including Iowa and the Dakotas are reporting the latest surges in cases.

This announcement comes in response to Mr. Trump’s executive order last month calling for improved rural health and telehealth access. Earlier this week, three federal agencies also announced they would team up to address gaps in rural broadband service – a key need because large portions of the plan seek to expand telehealth.

The plan is more than 70 pages long and the word “telehealth” appears more than 90 times, with a focus on projects across HHS, including the Health Resources and Services Administration and the Centers for Medicare & Medicaid Services.

Mr. Barnhart said CMS has passed some public health emergency waivers since the beginning of the pandemic that helped rural facilities get more funding, including one that specifically was designed to provide additional money for telehealth services. However, those waivers are set to expire when the coronavirus emergency ends. Officials have not yet set a date for when the federal emergency will end.

Andrew Jay Schwartzman, senior counselor to the Benton Institute for Broadband & Society, a private foundation that works to ensure greater Internet access, said there are multiple challenges with implementing telehealth across the nation. Many initiatives for robust telehealth programs need fast bandwidth, yet getting the money and setting up the necessary infrastructure is very difficult, he said.

“It will be a long time before this kind of technology will be readily available to much of the country,” he said.

Ge Bai, associate professor of accounting and health policy at Johns Hopkins University in Baltimore, noted that telehealth was short on funding in the HHS initiative. However, she said, the focus on telehealth, as well as a proposed shift in payment for small rural hospitals and changing workforce licensing requirements, had good potential.

“We are so close to the election that this is probably more of a messaging issue to cater to rural residents,” Ms. Bai said. “But it doesn’t matter who will be president. This report will give the next administration useful guidance.”

The American Hospital Association, representing 5,000 hospitals nationwide, sent a letter to Mr. Trump last week recommending a host of steps the administration could take. As of late Thursday, AHA was still reviewing the HHS plan but said it was “encouraged by the increased attention on rural health care.”

Buried within the HHS announcement are technical initiatives, such as a contract to help clinics and hospitals integrate care, and detailed efforts to address gaps in care, including a proposal to increase funding for school-based mental health programs in the president’s 2021 budget.

A senior HHS official said that, while some actions have been taken in recent months to improve rural health — such as the $11 billion provided to rural hospitals through coronavirus relief funding — more is needed.

“We’re putting our stake in the ground that the time for talk is over,” he said. “We’re going to move forward.”

Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of KFF (Kaiser Family Foundation), which is not affiliated with Kaiser Permanente.

In the very near future, clinicians injecting dermal fillers on the face will have the benefit of a guided “smart” needle that senses blood vessels, thereby dramatically reducing the risk of adverse events such as necrosis and blindness.

That is the goal of an experienced team composed of leading clinicians, academics, and researchers developing S³ Inject, a first-in-class safety innovation that has entered human trials.

“When physicians inject the fillers, they hope experience and technique will enable them to avoid adverse events,” Irina Erenburg, PhD, said during the virtual annual Masters of Aesthetics Symposium. “If they inadvertently hit a blood vessel, the filler can actually occlude that vessel and cause either an infarct of the skin or, in certain serious cases, blindness. This is a challenging adverse event that every injector is focused on avoiding. While hyaluronidase is used as a rescue [medication] in certain cases, the risk is real,” she added.

Vision abnormalities, including blindness, and necrosis are among the adverse events associated with dermal fillers that have been reported to the Food and Drug Administration.

S³ Inject is a sensing needle that can differentiate tissues such as fat, blood vessels, and muscle. Its proprietary algorithms provide immediate feedback via a micro LED light embedded in the needle hub. Results from recent human trials demonstrate that, as the needle tip passes through different biological tissues and fluids, “it senses changes in specific electrical properties and with that information sends a very precise signal to the needle hub,” said Dr. Erenburg, CEO and President of Waltham, Mass.–based Blossom Innovations, a company focused on developing early stage medical devices in dermatology. “With that information, the physician can make real-time treatment decisions.”

Currently, in order to determine if the needle is in a blood vessel, physicians pull back on the syringe and look for a flash of blood. “In speaking with physicians, the pull back technique has limitations, in part, because filler in the syringe can limit easy pull back to check the presence of a blood vessel,” she said. “Our needles provide an immediate response for a safer injection.”

Blossom Innovations has developed a proprietary manufacturing process that will initially target 27 gauge needles, but over time it plans to introduce multiple sizes, as well as cannulas.

“The physicians in our industry are committed to patient safety and they’re looking for better outcomes with a solution that does not impact their technique,” said Dr. Erenburg, who founded Blossom Innovations along with R. Rox Anderson, MD, director of the Wellman Center for Photomedicine at Massachusetts General Hospital, Boston; Dieter Manstein, MD, PhD, also at Massachusetts General Hospital; and Henry H.L. Chan, MD, PhD, of the Hong Kong Dermatology and Laser Center. During market research for S³ Inject, which was conducted with 15 leading injectors, thought leaders, and trend makers, the country’s leading injectors expressed strong interest in “solutions that allow them to provide additional safety for their patients and provide personal reassurance to the physician,” she said. “They definitely would want to train all their physicians and injectors on its use.”

As clinical testing continues, the company is preparing to submit data to the FDA’s Premarket Notification program, known as the 510(k) process. “Our intent is to create a scale-up manufacturing over the course of the coming year in time for our clearance, with a planned launch at the end of 2021,” Dr. Erenburg said. “Based on our clinical research and physician discussions, we are confident that S³ Inject is a breakthrough safety technology which will drive a better outcome for patients.”

Dr. Erenburg is an employee of Blossom Innovations.

[email protected]

In the very near future, clinicians injecting dermal fillers on the face will have the benefit of a guided “smart” needle that senses blood vessels, thereby dramatically reducing the risk of adverse events such as necrosis and blindness.

That is the goal of an experienced team composed of leading clinicians, academics, and researchers developing S³ Inject, a first-in-class safety innovation that has entered human trials.

“When physicians inject the fillers, they hope experience and technique will enable them to avoid adverse events,” Irina Erenburg, PhD, said during the virtual annual Masters of Aesthetics Symposium. “If they inadvertently hit a blood vessel, the filler can actually occlude that vessel and cause either an infarct of the skin or, in certain serious cases, blindness. This is a challenging adverse event that every injector is focused on avoiding. While hyaluronidase is used as a rescue [medication] in certain cases, the risk is real,” she added.

Vision abnormalities, including blindness, and necrosis are among the adverse events associated with dermal fillers that have been reported to the Food and Drug Administration.

S³ Inject is a sensing needle that can differentiate tissues such as fat, blood vessels, and muscle. Its proprietary algorithms provide immediate feedback via a micro LED light embedded in the needle hub. Results from recent human trials demonstrate that, as the needle tip passes through different biological tissues and fluids, “it senses changes in specific electrical properties and with that information sends a very precise signal to the needle hub,” said Dr. Erenburg, CEO and President of Waltham, Mass.–based Blossom Innovations, a company focused on developing early stage medical devices in dermatology. “With that information, the physician can make real-time treatment decisions.”

Currently, in order to determine if the needle is in a blood vessel, physicians pull back on the syringe and look for a flash of blood. “In speaking with physicians, the pull back technique has limitations, in part, because filler in the syringe can limit easy pull back to check the presence of a blood vessel,” she said. “Our needles provide an immediate response for a safer injection.”

Blossom Innovations has developed a proprietary manufacturing process that will initially target 27 gauge needles, but over time it plans to introduce multiple sizes, as well as cannulas.

“The physicians in our industry are committed to patient safety and they’re looking for better outcomes with a solution that does not impact their technique,” said Dr. Erenburg, who founded Blossom Innovations along with R. Rox Anderson, MD, director of the Wellman Center for Photomedicine at Massachusetts General Hospital, Boston; Dieter Manstein, MD, PhD, also at Massachusetts General Hospital; and Henry H.L. Chan, MD, PhD, of the Hong Kong Dermatology and Laser Center. During market research for S³ Inject, which was conducted with 15 leading injectors, thought leaders, and trend makers, the country’s leading injectors expressed strong interest in “solutions that allow them to provide additional safety for their patients and provide personal reassurance to the physician,” she said. “They definitely would want to train all their physicians and injectors on its use.”

As clinical testing continues, the company is preparing to submit data to the FDA’s Premarket Notification program, known as the 510(k) process. “Our intent is to create a scale-up manufacturing over the course of the coming year in time for our clearance, with a planned launch at the end of 2021,” Dr. Erenburg said. “Based on our clinical research and physician discussions, we are confident that S³ Inject is a breakthrough safety technology which will drive a better outcome for patients.”

Dr. Erenburg is an employee of Blossom Innovations.

[email protected]

In the very near future, clinicians injecting dermal fillers on the face will have the benefit of a guided “smart” needle that senses blood vessels, thereby dramatically reducing the risk of adverse events such as necrosis and blindness.

That is the goal of an experienced team composed of leading clinicians, academics, and researchers developing S³ Inject, a first-in-class safety innovation that has entered human trials.

“When physicians inject the fillers, they hope experience and technique will enable them to avoid adverse events,” Irina Erenburg, PhD, said during the virtual annual Masters of Aesthetics Symposium. “If they inadvertently hit a blood vessel, the filler can actually occlude that vessel and cause either an infarct of the skin or, in certain serious cases, blindness. This is a challenging adverse event that every injector is focused on avoiding. While hyaluronidase is used as a rescue [medication] in certain cases, the risk is real,” she added.

Vision abnormalities, including blindness, and necrosis are among the adverse events associated with dermal fillers that have been reported to the Food and Drug Administration.

S³ Inject is a sensing needle that can differentiate tissues such as fat, blood vessels, and muscle. Its proprietary algorithms provide immediate feedback via a micro LED light embedded in the needle hub. Results from recent human trials demonstrate that, as the needle tip passes through different biological tissues and fluids, “it senses changes in specific electrical properties and with that information sends a very precise signal to the needle hub,” said Dr. Erenburg, CEO and President of Waltham, Mass.–based Blossom Innovations, a company focused on developing early stage medical devices in dermatology. “With that information, the physician can make real-time treatment decisions.”

Currently, in order to determine if the needle is in a blood vessel, physicians pull back on the syringe and look for a flash of blood. “In speaking with physicians, the pull back technique has limitations, in part, because filler in the syringe can limit easy pull back to check the presence of a blood vessel,” she said. “Our needles provide an immediate response for a safer injection.”

Blossom Innovations has developed a proprietary manufacturing process that will initially target 27 gauge needles, but over time it plans to introduce multiple sizes, as well as cannulas.

“The physicians in our industry are committed to patient safety and they’re looking for better outcomes with a solution that does not impact their technique,” said Dr. Erenburg, who founded Blossom Innovations along with R. Rox Anderson, MD, director of the Wellman Center for Photomedicine at Massachusetts General Hospital, Boston; Dieter Manstein, MD, PhD, also at Massachusetts General Hospital; and Henry H.L. Chan, MD, PhD, of the Hong Kong Dermatology and Laser Center. During market research for S³ Inject, which was conducted with 15 leading injectors, thought leaders, and trend makers, the country’s leading injectors expressed strong interest in “solutions that allow them to provide additional safety for their patients and provide personal reassurance to the physician,” she said. “They definitely would want to train all their physicians and injectors on its use.”

As clinical testing continues, the company is preparing to submit data to the FDA’s Premarket Notification program, known as the 510(k) process. “Our intent is to create a scale-up manufacturing over the course of the coming year in time for our clearance, with a planned launch at the end of 2021,” Dr. Erenburg said. “Based on our clinical research and physician discussions, we are confident that S³ Inject is a breakthrough safety technology which will drive a better outcome for patients.”

Dr. Erenburg is an employee of Blossom Innovations.

[email protected]

The human growth hormone formulation somapacitan for adults with growth hormone deficiency was approved by the Food and Drug Administration on Sept. 1. The drug is injected once a week, while other FDA-approved human growth hormone formulations require daily jabs.

Somapacitan contains an albumin-binding element attached to the growth hormone, causing the reversible binding to albumin proteins in the body. This reduces clearance and increases the half-life of the hormone. The formulation has previous demonstrated safety and efficacy in children with growth hormone deficiency (J Clin Endocrinol Metab. 2020 Apr 1. doi: 10.1210/clinem/dgz310).

Growth hormone treatment can counter abdominal obesity, reduced lean body mass, fatigue, osteopenia, cardiovascular risks, and other manifestations of growth hormone deficiency in adults, but daily injections can be burdensome for patients. That makes long-acting versions attractive, but the lifelong nature of the treatment makes it important to characterize safety and tolerability.

The approval comes on the strength of a randomized, placebo-controlled phase 3 trial (REAL 1) of 300 adult patients in 17 countries with growth hormone deficiency (J Clin Endocrinol Metab. 2020 Apr 1. doi: 10.1210/clinem/dgaa049). Participants had either never received growth hormone treatment, or had stopped taking one at least 6 months before starting the trial. Subjects received once-weekly somapacitan, once-weekly placebo, or daily somatropin, which is FDA approved.

The primary endpoint was percentage change of truncal fat, which is regulated by growth hormone, and can lead to medical problems. After 34 weeks, subjects in the somapacitan group experienced a 1.06% decrease in truncal fat, compared with a 0.47% increase in the placebo group (P = .009) and a 2.23% decrease in the daily somatropin group.

After 34 weeks, a 52-week extension trial began. The somapacitan group continued on the drug and the placebo group was offered somapacitan. Patients on daily somatropin were randomized to continue daily treatment with somatropin or to switch to somapacitan.

At the end of the extension trial, those taking somapacitan for the full 86-week duration had an average reduction of 1.52% in truncal fat. After 86 weeks, the somapacitan and daily somatropin groups had similar values for percentage change in visceral fat, lean body mass, or appendicular skeletal muscle mass.

Common side effects of somapacitan were back pain, joint paint, indigestion, a sleep disorder, dizziness, tonsillitis, swelling in the arms or lower legs, vomiting, adrenal insufficiency, hypertension, increase in blood creatine phosphokinase, weight increase, and anemia.

Somapacitan, marketed as Sogroya by Novo Nordisk, is contraindicated in patients with an allergy to the drug, as well as those with an active malignancy, diabetic eye disease where increases in blood sugars could lead to retinal damage, acute critical illness, or acute respiratory failure.

The FDA recommends that providers perform an eye examination before drug initiation, as well as periodically while the patient is taking the drug, to rule out preexisting papilledema. This could be a sign of intracranial hypertension, which could be caused or worsened by growth hormones.

The human growth hormone formulation somapacitan for adults with growth hormone deficiency was approved by the Food and Drug Administration on Sept. 1. The drug is injected once a week, while other FDA-approved human growth hormone formulations require daily jabs.

Somapacitan contains an albumin-binding element attached to the growth hormone, causing the reversible binding to albumin proteins in the body. This reduces clearance and increases the half-life of the hormone. The formulation has previous demonstrated safety and efficacy in children with growth hormone deficiency (J Clin Endocrinol Metab. 2020 Apr 1. doi: 10.1210/clinem/dgz310).

Growth hormone treatment can counter abdominal obesity, reduced lean body mass, fatigue, osteopenia, cardiovascular risks, and other manifestations of growth hormone deficiency in adults, but daily injections can be burdensome for patients. That makes long-acting versions attractive, but the lifelong nature of the treatment makes it important to characterize safety and tolerability.

The approval comes on the strength of a randomized, placebo-controlled phase 3 trial (REAL 1) of 300 adult patients in 17 countries with growth hormone deficiency (J Clin Endocrinol Metab. 2020 Apr 1. doi: 10.1210/clinem/dgaa049). Participants had either never received growth hormone treatment, or had stopped taking one at least 6 months before starting the trial. Subjects received once-weekly somapacitan, once-weekly placebo, or daily somatropin, which is FDA approved.

The primary endpoint was percentage change of truncal fat, which is regulated by growth hormone, and can lead to medical problems. After 34 weeks, subjects in the somapacitan group experienced a 1.06% decrease in truncal fat, compared with a 0.47% increase in the placebo group (P = .009) and a 2.23% decrease in the daily somatropin group.

After 34 weeks, a 52-week extension trial began. The somapacitan group continued on the drug and the placebo group was offered somapacitan. Patients on daily somatropin were randomized to continue daily treatment with somatropin or to switch to somapacitan.

At the end of the extension trial, those taking somapacitan for the full 86-week duration had an average reduction of 1.52% in truncal fat. After 86 weeks, the somapacitan and daily somatropin groups had similar values for percentage change in visceral fat, lean body mass, or appendicular skeletal muscle mass.

Common side effects of somapacitan were back pain, joint paint, indigestion, a sleep disorder, dizziness, tonsillitis, swelling in the arms or lower legs, vomiting, adrenal insufficiency, hypertension, increase in blood creatine phosphokinase, weight increase, and anemia.

Somapacitan, marketed as Sogroya by Novo Nordisk, is contraindicated in patients with an allergy to the drug, as well as those with an active malignancy, diabetic eye disease where increases in blood sugars could lead to retinal damage, acute critical illness, or acute respiratory failure.

The FDA recommends that providers perform an eye examination before drug initiation, as well as periodically while the patient is taking the drug, to rule out preexisting papilledema. This could be a sign of intracranial hypertension, which could be caused or worsened by growth hormones.

The human growth hormone formulation somapacitan for adults with growth hormone deficiency was approved by the Food and Drug Administration on Sept. 1. The drug is injected once a week, while other FDA-approved human growth hormone formulations require daily jabs.

Somapacitan contains an albumin-binding element attached to the growth hormone, causing the reversible binding to albumin proteins in the body. This reduces clearance and increases the half-life of the hormone. The formulation has previous demonstrated safety and efficacy in children with growth hormone deficiency (J Clin Endocrinol Metab. 2020 Apr 1. doi: 10.1210/clinem/dgz310).

Growth hormone treatment can counter abdominal obesity, reduced lean body mass, fatigue, osteopenia, cardiovascular risks, and other manifestations of growth hormone deficiency in adults, but daily injections can be burdensome for patients. That makes long-acting versions attractive, but the lifelong nature of the treatment makes it important to characterize safety and tolerability.

The approval comes on the strength of a randomized, placebo-controlled phase 3 trial (REAL 1) of 300 adult patients in 17 countries with growth hormone deficiency (J Clin Endocrinol Metab. 2020 Apr 1. doi: 10.1210/clinem/dgaa049). Participants had either never received growth hormone treatment, or had stopped taking one at least 6 months before starting the trial. Subjects received once-weekly somapacitan, once-weekly placebo, or daily somatropin, which is FDA approved.

The primary endpoint was percentage change of truncal fat, which is regulated by growth hormone, and can lead to medical problems. After 34 weeks, subjects in the somapacitan group experienced a 1.06% decrease in truncal fat, compared with a 0.47% increase in the placebo group (P = .009) and a 2.23% decrease in the daily somatropin group.

After 34 weeks, a 52-week extension trial began. The somapacitan group continued on the drug and the placebo group was offered somapacitan. Patients on daily somatropin were randomized to continue daily treatment with somatropin or to switch to somapacitan.

At the end of the extension trial, those taking somapacitan for the full 86-week duration had an average reduction of 1.52% in truncal fat. After 86 weeks, the somapacitan and daily somatropin groups had similar values for percentage change in visceral fat, lean body mass, or appendicular skeletal muscle mass.

Common side effects of somapacitan were back pain, joint paint, indigestion, a sleep disorder, dizziness, tonsillitis, swelling in the arms or lower legs, vomiting, adrenal insufficiency, hypertension, increase in blood creatine phosphokinase, weight increase, and anemia.

Somapacitan, marketed as Sogroya by Novo Nordisk, is contraindicated in patients with an allergy to the drug, as well as those with an active malignancy, diabetic eye disease where increases in blood sugars could lead to retinal damage, acute critical illness, or acute respiratory failure.

The FDA recommends that providers perform an eye examination before drug initiation, as well as periodically while the patient is taking the drug, to rule out preexisting papilledema. This could be a sign of intracranial hypertension, which could be caused or worsened by growth hormones.

One of every 10 pregnant or recently pregnant women in hospital was diagnosed with COVID-19, yet up to three-quarters were asymptomatic at the time of diagnosis, according to a living systematic review from the PregCOV-19 Living Systematic Review Consortium.

dimarik/iStock/Getty Images

The study, published in BMJ, shows an increased risk of preterm delivery, as well as the need for invasive ventilation in these women, wrote John Allotey, PhD, of the University of Birmingham (England) and colleagues. The findings “will produce a strong evidence base for living guidelines on COVID-19 and pregnancy,” they noted.

The systematic review included 77 studies, one-third each from the United States and China, with the remaining studies from Belgium, Brazil, Denmark, France, Israel, Italy, Japan, Mexico, the Netherlands Portugal, Spain, and the United Kingdom.

The studies included women with COVID-19, of whom 13,118 were either pregnant or in the postpartum or postabortion period and 83,486 were of reproductive age but not pregnant. Some studies also included healthy pregnant women for comparison.

In the pregnant and recently pregnant women, the most common COVID-19 symptoms were fever (40%) and cough (39%), with lymphopenia (35%) and raised C reactive protein levels (49%) being the most common laboratory findings. Pregnant and recently pregnant women with COVID-19 were less likely to have fever (odds ratio, 0.43) and myalgia (OR, 0.48), compared with nonpregnant women of reproductive age with COVID-19, reported the authors.

The overall preterm and spontaneous preterm birth rates in the COVID-19–positive women were 17% and 6% respectively. Dr. Allotey and authors noted that “these preterm births could be medically indicated, as the overall rates of spontaneous preterm births in pregnant women with COVID-19 was broadly similar to those observed in the pre-pandemic period.” There were 18 stillbirths and 6 neonatal deaths in the COVID-19 cohort.

Overall, 73 (0.1%) of pregnant women with confirmed COVID-19 died from any cause, and severe COVID-19 infection was diagnosed in 13%. Maternal risk factors associated with severe infection included older age (OR, 1.78), high body mass index (OR, 2.3), chronic hypertension (OR, 2.0), and preexisting diabetes (OR, 2.51). Compared with nonpregnant women with COVID-19, pregnant or recently pregnant women with the infection were at increased risk of admission to intensive care (OR, 1.62) and needing invasive ventilation (OR, 1.88).

The report included studies published between December 1, 2019, and June 26, 2020, but the living systematic review will involve weekly search updates, with analysis performed every 2-4 weeks and reported through a dedicated website.
 

The value of a living meta-analysis

Asked to comment on the findings, Torri Metz, MD, a maternal-fetal medicine subspecialist at the University of Utah, Salt Lake City, expressed surprise at the 10% rate of infection in the pregnant or recently pregnant population. “This is higher than currently observed at many hospitals in the United States,” she said in an interview. “This may overestimate the actual risk as many of these studies were published early in the pandemic and did not universally sample women who were pregnant for SARS-CoV-2.”

She noted the value of a living meta-analysis in that it will be updated on a regular basis as new evidence emerges. “During this time of rapidly accumulating publications about COVID-19 infection, clinicians will find it useful to have a resource in which the available data can be combined in one source.”

And there are still some outstanding questions that new studies hopefully will shed light on, she added. “The authors found that many of the risk factors for severe disease, like diabetes, obesity and high blood pressure, in nonpregnant adults are the same in the pregnant population. What remains unknown is if pregnant patients with COVID-19 infection are at higher risk than those who are not pregnant. The authors note that this information is still limited and largely influenced in this published analysis by a CDC [Centers for Disease Control and Prevention] study in which the majority of patients had unknown pregnancy status. We also do not know if COVID-19 infection is associated with any birth defects since the majority of women with COVID-19 infection in the first trimester have not yet delivered.”

Malavika Prabhu, MD, an obstetetrician/gyneologist at Weill Cornell Medicine in New York City added that “this systematic review and meta analysis, which is a compilation of other studies done around the globe, confirms that pregnant women with preexisting medical conditions such as diabetes, hypertension, and obesity, are at increased risk of severe COVID-19 and that pregnant women with COVID-19 are at increased risk of invasive ventilation, compared to nonpregnant women with COVID-19, particularly if they have a preexisting medical condition.”

She said the preterm delivery rate of COVID-positive women is “challenging to interpret given that the total preterm birth rate potentially included many medically indicated preterm deliveries – which is to be expected – and there is no comparison group for spontaneous preterm birth presented”.

Other outstanding questions about COVID-19 pregnancies include whether they are associated with preeclampsia or smaller/growth restricted infants and why the cesarean delivery rate is high, she said. “But some of these questions are tough to answer with this data because it primarily reflects a COVID infection close to the delivery, not one that occurred several months prior to a delivery.”

Deborah Money, MD, professor of obstetrics and gynecology, medicine, and the school of population and public health, University of British Columbia, Vancouver, commented that “this is a group that have been doing ongoing living systematic reviews of the literature scanning for pregnancy outcomes. They post their information in real time on their website, so many of us in this area follow these postings as their methodology is robust and they work hard to only include high-quality literature and avoid duplication of cases in multiple papers. There has been a problem of re-reporting the same severe cases of COVID-19 in the literature.”

This “amplifies the importance of collecting Canadian-specific data to ensure that we understand if these kind of outcomes will also be found in Canada. The data presented in this paper represent outcomes from a broad range of countries with different methods of collecting information on pregnancy and highly variable prenatal care systems. This makes our pan-Canadian study of outcomes of COVID-19 for pregnant women and their infants, CANCOVID-Preg, even more important,” she said.

“Globally, we all must continue to monitor outcomes of COVID-19 in pregnancy to minimize adverse impact on women and their infants,” said Dr. Money, who was not involved in the study.

The study was partially funded by the World Health Organization and supported by Katie’s Team, a dedicated patient and public involvement group in Women’s Health. Dr. Metz is principal investigator for the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Maternal-Fetal Medicine Units Network COVID-19 study; the study is funded by NICHD and enrollment is ongoing. Dr. Prabhu had no relevant financial disclosures. Dr. Money received funding from the Canadian Institutes for Health Research and the Public Health Agency of Canada and received a small grant from theBC Women’s Foundation for COVID-19 in pregnancy research.

SOURCE: Allotey J et al. BMJ. 2020;370:m3320.

One of every 10 pregnant or recently pregnant women in hospital was diagnosed with COVID-19, yet up to three-quarters were asymptomatic at the time of diagnosis, according to a living systematic review from the PregCOV-19 Living Systematic Review Consortium.

dimarik/iStock/Getty Images

The study, published in BMJ, shows an increased risk of preterm delivery, as well as the need for invasive ventilation in these women, wrote John Allotey, PhD, of the University of Birmingham (England) and colleagues. The findings “will produce a strong evidence base for living guidelines on COVID-19 and pregnancy,” they noted.

The systematic review included 77 studies, one-third each from the United States and China, with the remaining studies from Belgium, Brazil, Denmark, France, Israel, Italy, Japan, Mexico, the Netherlands Portugal, Spain, and the United Kingdom.

The studies included women with COVID-19, of whom 13,118 were either pregnant or in the postpartum or postabortion period and 83,486 were of reproductive age but not pregnant. Some studies also included healthy pregnant women for comparison.

In the pregnant and recently pregnant women, the most common COVID-19 symptoms were fever (40%) and cough (39%), with lymphopenia (35%) and raised C reactive protein levels (49%) being the most common laboratory findings. Pregnant and recently pregnant women with COVID-19 were less likely to have fever (odds ratio, 0.43) and myalgia (OR, 0.48), compared with nonpregnant women of reproductive age with COVID-19, reported the authors.

The overall preterm and spontaneous preterm birth rates in the COVID-19–positive women were 17% and 6% respectively. Dr. Allotey and authors noted that “these preterm births could be medically indicated, as the overall rates of spontaneous preterm births in pregnant women with COVID-19 was broadly similar to those observed in the pre-pandemic period.” There were 18 stillbirths and 6 neonatal deaths in the COVID-19 cohort.

Overall, 73 (0.1%) of pregnant women with confirmed COVID-19 died from any cause, and severe COVID-19 infection was diagnosed in 13%. Maternal risk factors associated with severe infection included older age (OR, 1.78), high body mass index (OR, 2.3), chronic hypertension (OR, 2.0), and preexisting diabetes (OR, 2.51). Compared with nonpregnant women with COVID-19, pregnant or recently pregnant women with the infection were at increased risk of admission to intensive care (OR, 1.62) and needing invasive ventilation (OR, 1.88).

The report included studies published between December 1, 2019, and June 26, 2020, but the living systematic review will involve weekly search updates, with analysis performed every 2-4 weeks and reported through a dedicated website.
 

The value of a living meta-analysis

Asked to comment on the findings, Torri Metz, MD, a maternal-fetal medicine subspecialist at the University of Utah, Salt Lake City, expressed surprise at the 10% rate of infection in the pregnant or recently pregnant population. “This is higher than currently observed at many hospitals in the United States,” she said in an interview. “This may overestimate the actual risk as many of these studies were published early in the pandemic and did not universally sample women who were pregnant for SARS-CoV-2.”

She noted the value of a living meta-analysis in that it will be updated on a regular basis as new evidence emerges. “During this time of rapidly accumulating publications about COVID-19 infection, clinicians will find it useful to have a resource in which the available data can be combined in one source.”

And there are still some outstanding questions that new studies hopefully will shed light on, she added. “The authors found that many of the risk factors for severe disease, like diabetes, obesity and high blood pressure, in nonpregnant adults are the same in the pregnant population. What remains unknown is if pregnant patients with COVID-19 infection are at higher risk than those who are not pregnant. The authors note that this information is still limited and largely influenced in this published analysis by a CDC [Centers for Disease Control and Prevention] study in which the majority of patients had unknown pregnancy status. We also do not know if COVID-19 infection is associated with any birth defects since the majority of women with COVID-19 infection in the first trimester have not yet delivered.”

Malavika Prabhu, MD, an obstetetrician/gyneologist at Weill Cornell Medicine in New York City added that “this systematic review and meta analysis, which is a compilation of other studies done around the globe, confirms that pregnant women with preexisting medical conditions such as diabetes, hypertension, and obesity, are at increased risk of severe COVID-19 and that pregnant women with COVID-19 are at increased risk of invasive ventilation, compared to nonpregnant women with COVID-19, particularly if they have a preexisting medical condition.”

She said the preterm delivery rate of COVID-positive women is “challenging to interpret given that the total preterm birth rate potentially included many medically indicated preterm deliveries – which is to be expected – and there is no comparison group for spontaneous preterm birth presented”.

Other outstanding questions about COVID-19 pregnancies include whether they are associated with preeclampsia or smaller/growth restricted infants and why the cesarean delivery rate is high, she said. “But some of these questions are tough to answer with this data because it primarily reflects a COVID infection close to the delivery, not one that occurred several months prior to a delivery.”

Deborah Money, MD, professor of obstetrics and gynecology, medicine, and the school of population and public health, University of British Columbia, Vancouver, commented that “this is a group that have been doing ongoing living systematic reviews of the literature scanning for pregnancy outcomes. They post their information in real time on their website, so many of us in this area follow these postings as their methodology is robust and they work hard to only include high-quality literature and avoid duplication of cases in multiple papers. There has been a problem of re-reporting the same severe cases of COVID-19 in the literature.”

This “amplifies the importance of collecting Canadian-specific data to ensure that we understand if these kind of outcomes will also be found in Canada. The data presented in this paper represent outcomes from a broad range of countries with different methods of collecting information on pregnancy and highly variable prenatal care systems. This makes our pan-Canadian study of outcomes of COVID-19 for pregnant women and their infants, CANCOVID-Preg, even more important,” she said.

“Globally, we all must continue to monitor outcomes of COVID-19 in pregnancy to minimize adverse impact on women and their infants,” said Dr. Money, who was not involved in the study.

The study was partially funded by the World Health Organization and supported by Katie’s Team, a dedicated patient and public involvement group in Women’s Health. Dr. Metz is principal investigator for the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Maternal-Fetal Medicine Units Network COVID-19 study; the study is funded by NICHD and enrollment is ongoing. Dr. Prabhu had no relevant financial disclosures. Dr. Money received funding from the Canadian Institutes for Health Research and the Public Health Agency of Canada and received a small grant from theBC Women’s Foundation for COVID-19 in pregnancy research.

SOURCE: Allotey J et al. BMJ. 2020;370:m3320.

One of every 10 pregnant or recently pregnant women in hospital was diagnosed with COVID-19, yet up to three-quarters were asymptomatic at the time of diagnosis, according to a living systematic review from the PregCOV-19 Living Systematic Review Consortium.

dimarik/iStock/Getty Images

The study, published in BMJ, shows an increased risk of preterm delivery, as well as the need for invasive ventilation in these women, wrote John Allotey, PhD, of the University of Birmingham (England) and colleagues. The findings “will produce a strong evidence base for living guidelines on COVID-19 and pregnancy,” they noted.

The systematic review included 77 studies, one-third each from the United States and China, with the remaining studies from Belgium, Brazil, Denmark, France, Israel, Italy, Japan, Mexico, the Netherlands Portugal, Spain, and the United Kingdom.

The studies included women with COVID-19, of whom 13,118 were either pregnant or in the postpartum or postabortion period and 83,486 were of reproductive age but not pregnant. Some studies also included healthy pregnant women for comparison.

In the pregnant and recently pregnant women, the most common COVID-19 symptoms were fever (40%) and cough (39%), with lymphopenia (35%) and raised C reactive protein levels (49%) being the most common laboratory findings. Pregnant and recently pregnant women with COVID-19 were less likely to have fever (odds ratio, 0.43) and myalgia (OR, 0.48), compared with nonpregnant women of reproductive age with COVID-19, reported the authors.

The overall preterm and spontaneous preterm birth rates in the COVID-19–positive women were 17% and 6% respectively. Dr. Allotey and authors noted that “these preterm births could be medically indicated, as the overall rates of spontaneous preterm births in pregnant women with COVID-19 was broadly similar to those observed in the pre-pandemic period.” There were 18 stillbirths and 6 neonatal deaths in the COVID-19 cohort.

Overall, 73 (0.1%) of pregnant women with confirmed COVID-19 died from any cause, and severe COVID-19 infection was diagnosed in 13%. Maternal risk factors associated with severe infection included older age (OR, 1.78), high body mass index (OR, 2.3), chronic hypertension (OR, 2.0), and preexisting diabetes (OR, 2.51). Compared with nonpregnant women with COVID-19, pregnant or recently pregnant women with the infection were at increased risk of admission to intensive care (OR, 1.62) and needing invasive ventilation (OR, 1.88).

The report included studies published between December 1, 2019, and June 26, 2020, but the living systematic review will involve weekly search updates, with analysis performed every 2-4 weeks and reported through a dedicated website.
 

The value of a living meta-analysis

Asked to comment on the findings, Torri Metz, MD, a maternal-fetal medicine subspecialist at the University of Utah, Salt Lake City, expressed surprise at the 10% rate of infection in the pregnant or recently pregnant population. “This is higher than currently observed at many hospitals in the United States,” she said in an interview. “This may overestimate the actual risk as many of these studies were published early in the pandemic and did not universally sample women who were pregnant for SARS-CoV-2.”

She noted the value of a living meta-analysis in that it will be updated on a regular basis as new evidence emerges. “During this time of rapidly accumulating publications about COVID-19 infection, clinicians will find it useful to have a resource in which the available data can be combined in one source.”

And there are still some outstanding questions that new studies hopefully will shed light on, she added. “The authors found that many of the risk factors for severe disease, like diabetes, obesity and high blood pressure, in nonpregnant adults are the same in the pregnant population. What remains unknown is if pregnant patients with COVID-19 infection are at higher risk than those who are not pregnant. The authors note that this information is still limited and largely influenced in this published analysis by a CDC [Centers for Disease Control and Prevention] study in which the majority of patients had unknown pregnancy status. We also do not know if COVID-19 infection is associated with any birth defects since the majority of women with COVID-19 infection in the first trimester have not yet delivered.”

Malavika Prabhu, MD, an obstetetrician/gyneologist at Weill Cornell Medicine in New York City added that “this systematic review and meta analysis, which is a compilation of other studies done around the globe, confirms that pregnant women with preexisting medical conditions such as diabetes, hypertension, and obesity, are at increased risk of severe COVID-19 and that pregnant women with COVID-19 are at increased risk of invasive ventilation, compared to nonpregnant women with COVID-19, particularly if they have a preexisting medical condition.”

She said the preterm delivery rate of COVID-positive women is “challenging to interpret given that the total preterm birth rate potentially included many medically indicated preterm deliveries – which is to be expected – and there is no comparison group for spontaneous preterm birth presented”.

Other outstanding questions about COVID-19 pregnancies include whether they are associated with preeclampsia or smaller/growth restricted infants and why the cesarean delivery rate is high, she said. “But some of these questions are tough to answer with this data because it primarily reflects a COVID infection close to the delivery, not one that occurred several months prior to a delivery.”

Deborah Money, MD, professor of obstetrics and gynecology, medicine, and the school of population and public health, University of British Columbia, Vancouver, commented that “this is a group that have been doing ongoing living systematic reviews of the literature scanning for pregnancy outcomes. They post their information in real time on their website, so many of us in this area follow these postings as their methodology is robust and they work hard to only include high-quality literature and avoid duplication of cases in multiple papers. There has been a problem of re-reporting the same severe cases of COVID-19 in the literature.”

This “amplifies the importance of collecting Canadian-specific data to ensure that we understand if these kind of outcomes will also be found in Canada. The data presented in this paper represent outcomes from a broad range of countries with different methods of collecting information on pregnancy and highly variable prenatal care systems. This makes our pan-Canadian study of outcomes of COVID-19 for pregnant women and their infants, CANCOVID-Preg, even more important,” she said.

“Globally, we all must continue to monitor outcomes of COVID-19 in pregnancy to minimize adverse impact on women and their infants,” said Dr. Money, who was not involved in the study.

The study was partially funded by the World Health Organization and supported by Katie’s Team, a dedicated patient and public involvement group in Women’s Health. Dr. Metz is principal investigator for the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Maternal-Fetal Medicine Units Network COVID-19 study; the study is funded by NICHD and enrollment is ongoing. Dr. Prabhu had no relevant financial disclosures. Dr. Money received funding from the Canadian Institutes for Health Research and the Public Health Agency of Canada and received a small grant from theBC Women’s Foundation for COVID-19 in pregnancy research.

SOURCE: Allotey J et al. BMJ. 2020;370:m3320.

A second drug is now available in the United States for use in the treatment of patients with metastatic non–small cell lung cancer (NSCLC) that tests positive for rearranged during transfection (RET) fusions.

The new drug is pralsetinib (Gavreto). The Food and Drug Administration granted it an accelerated approval for this indication on the basis of response rate data. Continued approval for this indication depends on clinical benefit in a confirmatory trial.

Pralsetinib joins selpercatinib (Retevmo), which was approved in the United States in May 2020 as the first RET-targeted therapy. Selpercatinib is also indicated for use in RET+ NSCLC and was approved for use in RET+ medullary thyroid cancer and RET+ thyroid cancer.

Pralsetinib is still awaiting approval for these thyroid cancer indications.

Both drugs are taken orally; pralsetinib is taken once daily, and selpercatinib is taken twice daily.

For both drugs, before treatment is initiated, laboratory testing is needed to show that a RET gene alteration is present in the tumor.

RET fusions are found in approximately 1%-2% of patients with NSCLC.

They are the latest of a number of tumor-specific gene alterations found in NSCLC that are targeted with an approved drug.

“Targeted therapies have dramatically improved care for patients with non–small cell lung cancer driven by oncogenes, including EGFR and ALK, and the approval of the selective RET inhibitor pralsetinib, or Gavreto, marks another milestone in a paradigm shift toward precision medicine,” Vivek Subbiah, MD, of the University of Texas MD Anderson Cancer Center, Houston, said in a press release.

Dr. Subbiah was an investigator of the phase 1/2 clinical trial known as ARROW, which provided the data on which the accelerated approval was based. In this trial, patients with RET+ NSCLC were found by testing with next-generation sequencing, FISH (fluorescence in situ hybridization), or other methods.

The ARROW trial involved one cohort of 87 patients who had previously been treated with platinum-based chemotherapy. In these patients, the overall response rate (ORR) was 57%, the complete response (CR) rate was 5.7%, and the median duration of response (DOR) was not estimable, according to the manufacturer, Blueprint Medicines.

The trial also involved 27 treatment-naive patients who were ineligible for platinum-based chemotherapy per the study protocol. In this group, the ORR was 70%, and the CR rate was 11%. The median DOR was 9.0 months.

“Patients treated with [pralsetinib] had durable clinical responses, with a subset achieving complete responses characterized by the resolution of all target lesions, an uncommon outcome in metastatic lung cancer,” Dr. Subbiah commented.

“We observed this activity with or without prior therapy and regardless of RET fusion partner or the presence of brain metastases. This approval represents an important advance with the potential to change standards of care for patients with RET fusion-positive NSCLC, who have historically had limited treatment options,” Dr. Subbiah added.

Product information for pralsetinib has warnings and precautions of interstitial lung disease/pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, risk for impaired wound healing, and risk for embryo-fetal toxicity.

This article first appeared on Medscape.com.

A second drug is now available in the United States for use in the treatment of patients with metastatic non–small cell lung cancer (NSCLC) that tests positive for rearranged during transfection (RET) fusions.

The new drug is pralsetinib (Gavreto). The Food and Drug Administration granted it an accelerated approval for this indication on the basis of response rate data. Continued approval for this indication depends on clinical benefit in a confirmatory trial.

Pralsetinib joins selpercatinib (Retevmo), which was approved in the United States in May 2020 as the first RET-targeted therapy. Selpercatinib is also indicated for use in RET+ NSCLC and was approved for use in RET+ medullary thyroid cancer and RET+ thyroid cancer.

Pralsetinib is still awaiting approval for these thyroid cancer indications.

Both drugs are taken orally; pralsetinib is taken once daily, and selpercatinib is taken twice daily.

For both drugs, before treatment is initiated, laboratory testing is needed to show that a RET gene alteration is present in the tumor.

RET fusions are found in approximately 1%-2% of patients with NSCLC.

They are the latest of a number of tumor-specific gene alterations found in NSCLC that are targeted with an approved drug.

“Targeted therapies have dramatically improved care for patients with non–small cell lung cancer driven by oncogenes, including EGFR and ALK, and the approval of the selective RET inhibitor pralsetinib, or Gavreto, marks another milestone in a paradigm shift toward precision medicine,” Vivek Subbiah, MD, of the University of Texas MD Anderson Cancer Center, Houston, said in a press release.

Dr. Subbiah was an investigator of the phase 1/2 clinical trial known as ARROW, which provided the data on which the accelerated approval was based. In this trial, patients with RET+ NSCLC were found by testing with next-generation sequencing, FISH (fluorescence in situ hybridization), or other methods.

The ARROW trial involved one cohort of 87 patients who had previously been treated with platinum-based chemotherapy. In these patients, the overall response rate (ORR) was 57%, the complete response (CR) rate was 5.7%, and the median duration of response (DOR) was not estimable, according to the manufacturer, Blueprint Medicines.

The trial also involved 27 treatment-naive patients who were ineligible for platinum-based chemotherapy per the study protocol. In this group, the ORR was 70%, and the CR rate was 11%. The median DOR was 9.0 months.

“Patients treated with [pralsetinib] had durable clinical responses, with a subset achieving complete responses characterized by the resolution of all target lesions, an uncommon outcome in metastatic lung cancer,” Dr. Subbiah commented.

“We observed this activity with or without prior therapy and regardless of RET fusion partner or the presence of brain metastases. This approval represents an important advance with the potential to change standards of care for patients with RET fusion-positive NSCLC, who have historically had limited treatment options,” Dr. Subbiah added.

Product information for pralsetinib has warnings and precautions of interstitial lung disease/pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, risk for impaired wound healing, and risk for embryo-fetal toxicity.

This article first appeared on Medscape.com.

A second drug is now available in the United States for use in the treatment of patients with metastatic non–small cell lung cancer (NSCLC) that tests positive for rearranged during transfection (RET) fusions.

The new drug is pralsetinib (Gavreto). The Food and Drug Administration granted it an accelerated approval for this indication on the basis of response rate data. Continued approval for this indication depends on clinical benefit in a confirmatory trial.

Pralsetinib joins selpercatinib (Retevmo), which was approved in the United States in May 2020 as the first RET-targeted therapy. Selpercatinib is also indicated for use in RET+ NSCLC and was approved for use in RET+ medullary thyroid cancer and RET+ thyroid cancer.

Pralsetinib is still awaiting approval for these thyroid cancer indications.

Both drugs are taken orally; pralsetinib is taken once daily, and selpercatinib is taken twice daily.

For both drugs, before treatment is initiated, laboratory testing is needed to show that a RET gene alteration is present in the tumor.

RET fusions are found in approximately 1%-2% of patients with NSCLC.

They are the latest of a number of tumor-specific gene alterations found in NSCLC that are targeted with an approved drug.

“Targeted therapies have dramatically improved care for patients with non–small cell lung cancer driven by oncogenes, including EGFR and ALK, and the approval of the selective RET inhibitor pralsetinib, or Gavreto, marks another milestone in a paradigm shift toward precision medicine,” Vivek Subbiah, MD, of the University of Texas MD Anderson Cancer Center, Houston, said in a press release.

Dr. Subbiah was an investigator of the phase 1/2 clinical trial known as ARROW, which provided the data on which the accelerated approval was based. In this trial, patients with RET+ NSCLC were found by testing with next-generation sequencing, FISH (fluorescence in situ hybridization), or other methods.

The ARROW trial involved one cohort of 87 patients who had previously been treated with platinum-based chemotherapy. In these patients, the overall response rate (ORR) was 57%, the complete response (CR) rate was 5.7%, and the median duration of response (DOR) was not estimable, according to the manufacturer, Blueprint Medicines.

The trial also involved 27 treatment-naive patients who were ineligible for platinum-based chemotherapy per the study protocol. In this group, the ORR was 70%, and the CR rate was 11%. The median DOR was 9.0 months.

“Patients treated with [pralsetinib] had durable clinical responses, with a subset achieving complete responses characterized by the resolution of all target lesions, an uncommon outcome in metastatic lung cancer,” Dr. Subbiah commented.

“We observed this activity with or without prior therapy and regardless of RET fusion partner or the presence of brain metastases. This approval represents an important advance with the potential to change standards of care for patients with RET fusion-positive NSCLC, who have historically had limited treatment options,” Dr. Subbiah added.

Product information for pralsetinib has warnings and precautions of interstitial lung disease/pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, risk for impaired wound healing, and risk for embryo-fetal toxicity.

This article first appeared on Medscape.com.

Treatment with a fixed-dose combination of sodium phenylbutyrate and taurursodiol (AMX0035, Amylyx Pharmaceuticals) slows the rate of decline in physical function in patients with amyotrophic lateral sclerosis (ALS), according to results of the phase 2/3 CENTAUR study.

Patients with a fast-progressing form of ALS who were treated with AMX0035 “retained higher levels of physical function over 6 months compared with those who received placebo,” reported principal investigator Sabrina Paganoni, MD, PhD, of the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital, Boston.

“This is very hopeful news for people affected by ALS, especially because we were able to see a treatment effect in a relatively short period of time,” Dr. Paganoni said.

The study was published online Sept. 3 in the New England Journal of Medicine.

In this study, AMX0035 demonstrated a “clinically meaningful benefit and a favorable safety profile for people living with ALS,” Josh Cohen, co-CEO, chairman, and cofounder at Amylyx, said in a news release. The company is “working collaboratively and expeditiously with agencies worldwide to bring this potential new treatment option forward.”

“The data ... makes a clear and compelling case that AMX0035 should be made available to people with ALS as soon as possible,” Calaneet Balas, president and CEO of The ALS Association, said in the release.

The CENTAUR trial

Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. AMX0035 combines 3 g sodium phenylbutyrate and 1 g of taurursodiol.

The CENTAUR trial tested AMX0035 against placebo in 137 ALS patients with symptom onset within the prior 18 months, with 89 patients in the AMX0035 group and 48 in the placebo group. AMX0035 or matching placebo were administered once daily for 3 weeks and then twice daily for a planned duration of 24 weeks.

In a modified intention-to-treat analysis, the mean rate of change in the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ALSFRS-R) score was −1.24 points per month with AMX0035 and −1.66 points per month with placebo (difference, 0.42 points per month; 95% CI, 0.03 - 0.81; P = .03). After 24 weeks, patients treated with AMX0035 scored on average 2.32 points higher on the ALSFRS-R than their peers on placebo group (P = .03).

“The score, consisting of four subdomains, showed a change that was most prominent for the fine-motor subscale and less apparent for the other subscales,” the investigators said.

Treatment with AMX0035 led to slowing of disease progression in a population in which many participants were receiving riluzole (Tiglutik), edaravone (Radicava) or both, they pointed out.

The secondary outcomes were rate of decline in isometric muscle strength and breathing function; change in plasma phosphorylated axonal neurofilament H subunit (pNF-H) levels; and the time to composite events of death, tracheostomy, permanent ventilation, and hospitalization. These outcomes did not differ significantly between the two groups.

Open-label extension ongoing

AMX0035 was generally well tolerated. Nearly all patients in both groups had one or more adverse events. Events occurring at 2% or greater frequency in the AMX0035 group were primarily gastrointestinal (diarrhea, nausea, salivary hypersecretion, and abdominal discomfort). Serious adverse events were more common in the placebo group (19% vs. 12%). The incidence of respiratory serious adverse events was 8% in the placebo group and 3% in the AMX0035 group.

More patients on active treatment than placebo (19% vs. 8%) stopped the trial regimen early owing to adverse events. The most common adverse events leading to discontinuation of the trial regimen were diarrhea and respiratory failure.

The trial was “too short for us to detect an effect on survival,” Dr. Paganoni said in an interview. Most of the participants who completed the trial elected to enroll in an open-label extension study and receive AMX0035 long-term. “This is important because it will teach us about the impact of AMX0035 on survival,” said Dr. Paganoni.

Interim data from the ongoing open-label extension study are being submitted to a peer-reviewed journal shortly and will be published in the coming months.
 

A cause for hope

“There has been understandable frustration with the slow pace of development of therapy for ALS,” Michael Benatar, MD, PhD, University of Miami, and Michael McDermott, PhD, University of Rochester (N.Y.), said in an accompanying editorial.

“Despite dozens of trials, few pharmacologic agents have emerged that affect functional decline or survival – and all only modestly so. Although the effects of sodium phenylbutyrate–taurursodiol are similarly modest, the incremental gains that they provide in the battle against ALS are a cause for hope,” they wrote.

They caution, however, that this study was enriched for patients with more rapidly progressive disease, which “raises questions about generalizability to the broader population of patients with ALS.

“Although the patients who were enrolled in the trial may not be biologically different from the broader population of patients with ALS, the magnitude of therapeutic effect may be smaller in the latter,” Dr. Benatar and Dr. McDermott noted.

They said that in light of “residual questions about efficacy and the ability of patients to continue taking the drug,” they agree with the authors’ conclusion that “longer and larger trials are needed to evaluate the efficacy and safety of sodium phenylbutyrate–taurursodiol in persons with ALS.”

Given these “tantalizing preliminary data,” Dr. Benatar and Dr. McDermott said they look forward to “a confirmatory phase 3 trial.” 

The study was supported by Amylyx Pharmaceuticals, the ALS Finding a Cure Foundation, and the ALS Association. Dr. Paganoni has received grants from Revalesio, Ra Pharma, Biohaven, Clene, and Prilenia. A complete list of disclosures for authors and editorialists is available with the original article.

A version of this article originally appeared on Medscape.com.

Treatment with a fixed-dose combination of sodium phenylbutyrate and taurursodiol (AMX0035, Amylyx Pharmaceuticals) slows the rate of decline in physical function in patients with amyotrophic lateral sclerosis (ALS), according to results of the phase 2/3 CENTAUR study.

Patients with a fast-progressing form of ALS who were treated with AMX0035 “retained higher levels of physical function over 6 months compared with those who received placebo,” reported principal investigator Sabrina Paganoni, MD, PhD, of the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital, Boston.

“This is very hopeful news for people affected by ALS, especially because we were able to see a treatment effect in a relatively short period of time,” Dr. Paganoni said.

The study was published online Sept. 3 in the New England Journal of Medicine.

In this study, AMX0035 demonstrated a “clinically meaningful benefit and a favorable safety profile for people living with ALS,” Josh Cohen, co-CEO, chairman, and cofounder at Amylyx, said in a news release. The company is “working collaboratively and expeditiously with agencies worldwide to bring this potential new treatment option forward.”

“The data ... makes a clear and compelling case that AMX0035 should be made available to people with ALS as soon as possible,” Calaneet Balas, president and CEO of The ALS Association, said in the release.

The CENTAUR trial

Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. AMX0035 combines 3 g sodium phenylbutyrate and 1 g of taurursodiol.

The CENTAUR trial tested AMX0035 against placebo in 137 ALS patients with symptom onset within the prior 18 months, with 89 patients in the AMX0035 group and 48 in the placebo group. AMX0035 or matching placebo were administered once daily for 3 weeks and then twice daily for a planned duration of 24 weeks.

In a modified intention-to-treat analysis, the mean rate of change in the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ALSFRS-R) score was −1.24 points per month with AMX0035 and −1.66 points per month with placebo (difference, 0.42 points per month; 95% CI, 0.03 - 0.81; P = .03). After 24 weeks, patients treated with AMX0035 scored on average 2.32 points higher on the ALSFRS-R than their peers on placebo group (P = .03).

“The score, consisting of four subdomains, showed a change that was most prominent for the fine-motor subscale and less apparent for the other subscales,” the investigators said.

Treatment with AMX0035 led to slowing of disease progression in a population in which many participants were receiving riluzole (Tiglutik), edaravone (Radicava) or both, they pointed out.

The secondary outcomes were rate of decline in isometric muscle strength and breathing function; change in plasma phosphorylated axonal neurofilament H subunit (pNF-H) levels; and the time to composite events of death, tracheostomy, permanent ventilation, and hospitalization. These outcomes did not differ significantly between the two groups.

Open-label extension ongoing

AMX0035 was generally well tolerated. Nearly all patients in both groups had one or more adverse events. Events occurring at 2% or greater frequency in the AMX0035 group were primarily gastrointestinal (diarrhea, nausea, salivary hypersecretion, and abdominal discomfort). Serious adverse events were more common in the placebo group (19% vs. 12%). The incidence of respiratory serious adverse events was 8% in the placebo group and 3% in the AMX0035 group.

More patients on active treatment than placebo (19% vs. 8%) stopped the trial regimen early owing to adverse events. The most common adverse events leading to discontinuation of the trial regimen were diarrhea and respiratory failure.

The trial was “too short for us to detect an effect on survival,” Dr. Paganoni said in an interview. Most of the participants who completed the trial elected to enroll in an open-label extension study and receive AMX0035 long-term. “This is important because it will teach us about the impact of AMX0035 on survival,” said Dr. Paganoni.

Interim data from the ongoing open-label extension study are being submitted to a peer-reviewed journal shortly and will be published in the coming months.
 

A cause for hope

“There has been understandable frustration with the slow pace of development of therapy for ALS,” Michael Benatar, MD, PhD, University of Miami, and Michael McDermott, PhD, University of Rochester (N.Y.), said in an accompanying editorial.

“Despite dozens of trials, few pharmacologic agents have emerged that affect functional decline or survival – and all only modestly so. Although the effects of sodium phenylbutyrate–taurursodiol are similarly modest, the incremental gains that they provide in the battle against ALS are a cause for hope,” they wrote.

They caution, however, that this study was enriched for patients with more rapidly progressive disease, which “raises questions about generalizability to the broader population of patients with ALS.

“Although the patients who were enrolled in the trial may not be biologically different from the broader population of patients with ALS, the magnitude of therapeutic effect may be smaller in the latter,” Dr. Benatar and Dr. McDermott noted.

They said that in light of “residual questions about efficacy and the ability of patients to continue taking the drug,” they agree with the authors’ conclusion that “longer and larger trials are needed to evaluate the efficacy and safety of sodium phenylbutyrate–taurursodiol in persons with ALS.”

Given these “tantalizing preliminary data,” Dr. Benatar and Dr. McDermott said they look forward to “a confirmatory phase 3 trial.” 

The study was supported by Amylyx Pharmaceuticals, the ALS Finding a Cure Foundation, and the ALS Association. Dr. Paganoni has received grants from Revalesio, Ra Pharma, Biohaven, Clene, and Prilenia. A complete list of disclosures for authors and editorialists is available with the original article.

A version of this article originally appeared on Medscape.com.

Treatment with a fixed-dose combination of sodium phenylbutyrate and taurursodiol (AMX0035, Amylyx Pharmaceuticals) slows the rate of decline in physical function in patients with amyotrophic lateral sclerosis (ALS), according to results of the phase 2/3 CENTAUR study.

Patients with a fast-progressing form of ALS who were treated with AMX0035 “retained higher levels of physical function over 6 months compared with those who received placebo,” reported principal investigator Sabrina Paganoni, MD, PhD, of the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital, Boston.

“This is very hopeful news for people affected by ALS, especially because we were able to see a treatment effect in a relatively short period of time,” Dr. Paganoni said.

The study was published online Sept. 3 in the New England Journal of Medicine.

In this study, AMX0035 demonstrated a “clinically meaningful benefit and a favorable safety profile for people living with ALS,” Josh Cohen, co-CEO, chairman, and cofounder at Amylyx, said in a news release. The company is “working collaboratively and expeditiously with agencies worldwide to bring this potential new treatment option forward.”

“The data ... makes a clear and compelling case that AMX0035 should be made available to people with ALS as soon as possible,” Calaneet Balas, president and CEO of The ALS Association, said in the release.

The CENTAUR trial

Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. AMX0035 combines 3 g sodium phenylbutyrate and 1 g of taurursodiol.

The CENTAUR trial tested AMX0035 against placebo in 137 ALS patients with symptom onset within the prior 18 months, with 89 patients in the AMX0035 group and 48 in the placebo group. AMX0035 or matching placebo were administered once daily for 3 weeks and then twice daily for a planned duration of 24 weeks.

In a modified intention-to-treat analysis, the mean rate of change in the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ALSFRS-R) score was −1.24 points per month with AMX0035 and −1.66 points per month with placebo (difference, 0.42 points per month; 95% CI, 0.03 - 0.81; P = .03). After 24 weeks, patients treated with AMX0035 scored on average 2.32 points higher on the ALSFRS-R than their peers on placebo group (P = .03).

“The score, consisting of four subdomains, showed a change that was most prominent for the fine-motor subscale and less apparent for the other subscales,” the investigators said.

Treatment with AMX0035 led to slowing of disease progression in a population in which many participants were receiving riluzole (Tiglutik), edaravone (Radicava) or both, they pointed out.

The secondary outcomes were rate of decline in isometric muscle strength and breathing function; change in plasma phosphorylated axonal neurofilament H subunit (pNF-H) levels; and the time to composite events of death, tracheostomy, permanent ventilation, and hospitalization. These outcomes did not differ significantly between the two groups.

Open-label extension ongoing

AMX0035 was generally well tolerated. Nearly all patients in both groups had one or more adverse events. Events occurring at 2% or greater frequency in the AMX0035 group were primarily gastrointestinal (diarrhea, nausea, salivary hypersecretion, and abdominal discomfort). Serious adverse events were more common in the placebo group (19% vs. 12%). The incidence of respiratory serious adverse events was 8% in the placebo group and 3% in the AMX0035 group.

More patients on active treatment than placebo (19% vs. 8%) stopped the trial regimen early owing to adverse events. The most common adverse events leading to discontinuation of the trial regimen were diarrhea and respiratory failure.

The trial was “too short for us to detect an effect on survival,” Dr. Paganoni said in an interview. Most of the participants who completed the trial elected to enroll in an open-label extension study and receive AMX0035 long-term. “This is important because it will teach us about the impact of AMX0035 on survival,” said Dr. Paganoni.

Interim data from the ongoing open-label extension study are being submitted to a peer-reviewed journal shortly and will be published in the coming months.
 

A cause for hope

“There has been understandable frustration with the slow pace of development of therapy for ALS,” Michael Benatar, MD, PhD, University of Miami, and Michael McDermott, PhD, University of Rochester (N.Y.), said in an accompanying editorial.

“Despite dozens of trials, few pharmacologic agents have emerged that affect functional decline or survival – and all only modestly so. Although the effects of sodium phenylbutyrate–taurursodiol are similarly modest, the incremental gains that they provide in the battle against ALS are a cause for hope,” they wrote.

They caution, however, that this study was enriched for patients with more rapidly progressive disease, which “raises questions about generalizability to the broader population of patients with ALS.

“Although the patients who were enrolled in the trial may not be biologically different from the broader population of patients with ALS, the magnitude of therapeutic effect may be smaller in the latter,” Dr. Benatar and Dr. McDermott noted.

They said that in light of “residual questions about efficacy and the ability of patients to continue taking the drug,” they agree with the authors’ conclusion that “longer and larger trials are needed to evaluate the efficacy and safety of sodium phenylbutyrate–taurursodiol in persons with ALS.”

Given these “tantalizing preliminary data,” Dr. Benatar and Dr. McDermott said they look forward to “a confirmatory phase 3 trial.” 

The study was supported by Amylyx Pharmaceuticals, the ALS Finding a Cure Foundation, and the ALS Association. Dr. Paganoni has received grants from Revalesio, Ra Pharma, Biohaven, Clene, and Prilenia. A complete list of disclosures for authors and editorialists is available with the original article.

A version of this article originally appeared on Medscape.com.

“Nomophobia” – the fear of being without a mobile phone or out of mobile phone contact – is extremely prevalent among college students and is associated with poor sleep habits and fatigue. In a study of more than 300 college students, nearly 9 in 10 (89%) were classified as having moderate to severe nomophobia. Greater levels of nomophobia were significantly linked to daytime sleepiness and more behaviors associated with poor sleep hygiene.

“My undergraduate research team came up with the idea for this study,” said study investigator Jennifer Peszka, PhD, professor of psychology at Hendrix College, Conway, Ark. She explained that her students had been looking at the impact of technology use in the 2 hours before bed, and hypothesized that ‘cell phone addiction’ might play a role in sleep problems.

Incidentally, “that group of students were all pretty high on nomophobia themselves so they were really interested in the outcome,” Dr. Peszka said.

The study findings were presented at the virtual annual meeting of the Associated Professional Sleep Societies.
 

A likely suspect

The study involved 327 undergraduates (mean age, 19.7 years) recruited from introductory psychology courses and campus newsletters. They completed several questionnaires, including the Nomophobia Questionnaire, the Epworth Sleepiness Scale, and the Sleep Hygiene Index.

Nomophobia was prevalent, with mild, moderate, and severe nomophobia reported by 10%, 83%, and 7% of students, respectively. Only one student reported no nomophobia at all. Dr. Peszka said the fact that 89% of students had moderate or severe nomophobia is “concerning,” given a 2012 study suggesting that 77% of 18- to 24-year-olds had nomophobia. This phobia “very well may be on a rapid rise,” she lamented.

Greater severity of nomophobia was significantly correlated with greater sleepiness measured by both the Epworth Sleepiness Scale (P < .05) and the Associated Features of Poor Sleep Hygiene daytime sleepiness item (P < .05). More severe nomophobia was also related to decreased motivation (a commonly reported symptom of insufficient sleep) and with more maladaptive sleep hygiene behaviors (including using technology during sleep time, long daytime naps, inconsistent wake and bed times, using bed for nonsleep purposes, uncomfortable bed, and bedtime cognitive rumination).

Prior research has shown that smartphones may lead to compulsive “checking” habits, compulsive usage, increased distress, and potentially addictive behaviors. Active phone use at bedtime has also been implicated in disrupted sleep. Nomophobia is likely to be an important consideration when treating sleep disorders and/or making any sleep hygiene recommendations, Dr. Peszka said.
 

Proliferation of ‘night owls’

Reached for comment, Rajkumar (Raj) Dasgupta, MD, University of Southern California, Los Angeles, said this is a “very timely study with COVID-19. Right now, more than ever, technology is a double-edged sword. I’m a father of three kids and, for now, technology is the only way some kids are going to be socializing and learning.”

Yet a foundation of good sleep hygiene is keeping a nightly sleep routine, said Dr. Dasgupta, who was not involved in the study. “Right now, it seems like all my sleep patients are becoming night owls and sleep time is becoming more and more delayed because there is so much news to keep up with. Also, you may be stressed at night and you may not have the motivation to wake up early in the morning.”

He said it is important to counsel patients to “put technology away at night. That goes for kids and adults.”

Support for the study was provided by Hendrix College Charles Brewer Fund for Psychology. Dr. Peszka and Dr. Dasgupta disclosed no relevant financial relationships.

A version of this article originally appeared on Medscape.com.

“Nomophobia” – the fear of being without a mobile phone or out of mobile phone contact – is extremely prevalent among college students and is associated with poor sleep habits and fatigue. In a study of more than 300 college students, nearly 9 in 10 (89%) were classified as having moderate to severe nomophobia. Greater levels of nomophobia were significantly linked to daytime sleepiness and more behaviors associated with poor sleep hygiene.

“My undergraduate research team came up with the idea for this study,” said study investigator Jennifer Peszka, PhD, professor of psychology at Hendrix College, Conway, Ark. She explained that her students had been looking at the impact of technology use in the 2 hours before bed, and hypothesized that ‘cell phone addiction’ might play a role in sleep problems.

Incidentally, “that group of students were all pretty high on nomophobia themselves so they were really interested in the outcome,” Dr. Peszka said.

The study findings were presented at the virtual annual meeting of the Associated Professional Sleep Societies.
 

A likely suspect

The study involved 327 undergraduates (mean age, 19.7 years) recruited from introductory psychology courses and campus newsletters. They completed several questionnaires, including the Nomophobia Questionnaire, the Epworth Sleepiness Scale, and the Sleep Hygiene Index.

Nomophobia was prevalent, with mild, moderate, and severe nomophobia reported by 10%, 83%, and 7% of students, respectively. Only one student reported no nomophobia at all. Dr. Peszka said the fact that 89% of students had moderate or severe nomophobia is “concerning,” given a 2012 study suggesting that 77% of 18- to 24-year-olds had nomophobia. This phobia “very well may be on a rapid rise,” she lamented.

Greater severity of nomophobia was significantly correlated with greater sleepiness measured by both the Epworth Sleepiness Scale (P < .05) and the Associated Features of Poor Sleep Hygiene daytime sleepiness item (P < .05). More severe nomophobia was also related to decreased motivation (a commonly reported symptom of insufficient sleep) and with more maladaptive sleep hygiene behaviors (including using technology during sleep time, long daytime naps, inconsistent wake and bed times, using bed for nonsleep purposes, uncomfortable bed, and bedtime cognitive rumination).

Prior research has shown that smartphones may lead to compulsive “checking” habits, compulsive usage, increased distress, and potentially addictive behaviors. Active phone use at bedtime has also been implicated in disrupted sleep. Nomophobia is likely to be an important consideration when treating sleep disorders and/or making any sleep hygiene recommendations, Dr. Peszka said.
 

Proliferation of ‘night owls’

Reached for comment, Rajkumar (Raj) Dasgupta, MD, University of Southern California, Los Angeles, said this is a “very timely study with COVID-19. Right now, more than ever, technology is a double-edged sword. I’m a father of three kids and, for now, technology is the only way some kids are going to be socializing and learning.”

Yet a foundation of good sleep hygiene is keeping a nightly sleep routine, said Dr. Dasgupta, who was not involved in the study. “Right now, it seems like all my sleep patients are becoming night owls and sleep time is becoming more and more delayed because there is so much news to keep up with. Also, you may be stressed at night and you may not have the motivation to wake up early in the morning.”

He said it is important to counsel patients to “put technology away at night. That goes for kids and adults.”

Support for the study was provided by Hendrix College Charles Brewer Fund for Psychology. Dr. Peszka and Dr. Dasgupta disclosed no relevant financial relationships.

A version of this article originally appeared on Medscape.com.

“Nomophobia” – the fear of being without a mobile phone or out of mobile phone contact – is extremely prevalent among college students and is associated with poor sleep habits and fatigue. In a study of more than 300 college students, nearly 9 in 10 (89%) were classified as having moderate to severe nomophobia. Greater levels of nomophobia were significantly linked to daytime sleepiness and more behaviors associated with poor sleep hygiene.

“My undergraduate research team came up with the idea for this study,” said study investigator Jennifer Peszka, PhD, professor of psychology at Hendrix College, Conway, Ark. She explained that her students had been looking at the impact of technology use in the 2 hours before bed, and hypothesized that ‘cell phone addiction’ might play a role in sleep problems.

Incidentally, “that group of students were all pretty high on nomophobia themselves so they were really interested in the outcome,” Dr. Peszka said.

The study findings were presented at the virtual annual meeting of the Associated Professional Sleep Societies.
 

A likely suspect

The study involved 327 undergraduates (mean age, 19.7 years) recruited from introductory psychology courses and campus newsletters. They completed several questionnaires, including the Nomophobia Questionnaire, the Epworth Sleepiness Scale, and the Sleep Hygiene Index.

Nomophobia was prevalent, with mild, moderate, and severe nomophobia reported by 10%, 83%, and 7% of students, respectively. Only one student reported no nomophobia at all. Dr. Peszka said the fact that 89% of students had moderate or severe nomophobia is “concerning,” given a 2012 study suggesting that 77% of 18- to 24-year-olds had nomophobia. This phobia “very well may be on a rapid rise,” she lamented.

Greater severity of nomophobia was significantly correlated with greater sleepiness measured by both the Epworth Sleepiness Scale (P < .05) and the Associated Features of Poor Sleep Hygiene daytime sleepiness item (P < .05). More severe nomophobia was also related to decreased motivation (a commonly reported symptom of insufficient sleep) and with more maladaptive sleep hygiene behaviors (including using technology during sleep time, long daytime naps, inconsistent wake and bed times, using bed for nonsleep purposes, uncomfortable bed, and bedtime cognitive rumination).

Prior research has shown that smartphones may lead to compulsive “checking” habits, compulsive usage, increased distress, and potentially addictive behaviors. Active phone use at bedtime has also been implicated in disrupted sleep. Nomophobia is likely to be an important consideration when treating sleep disorders and/or making any sleep hygiene recommendations, Dr. Peszka said.
 

Proliferation of ‘night owls’

Reached for comment, Rajkumar (Raj) Dasgupta, MD, University of Southern California, Los Angeles, said this is a “very timely study with COVID-19. Right now, more than ever, technology is a double-edged sword. I’m a father of three kids and, for now, technology is the only way some kids are going to be socializing and learning.”

Yet a foundation of good sleep hygiene is keeping a nightly sleep routine, said Dr. Dasgupta, who was not involved in the study. “Right now, it seems like all my sleep patients are becoming night owls and sleep time is becoming more and more delayed because there is so much news to keep up with. Also, you may be stressed at night and you may not have the motivation to wake up early in the morning.”

He said it is important to counsel patients to “put technology away at night. That goes for kids and adults.”

Support for the study was provided by Hendrix College Charles Brewer Fund for Psychology. Dr. Peszka and Dr. Dasgupta disclosed no relevant financial relationships.

A version of this article originally appeared on Medscape.com.