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Christopher Palmer has been an associate editor at MDedge News since 2017. When he's not tidying grammar, he writes short pieces about breaking FDA announcements and approvals, as well as journal articles. He proudly holds a BA in English and philosophy. Follow him on Twitter @cmacmpalm.
FDA approves Tecentriq plus Abraxane in breast cancer
The Food and Drug Administration has granted accelerated approval for the combination of atezolizumab (Tecentriq) plus nanoparticle albumin–bound paclitaxel (nab-paclitaxel; Abraxane) for the treatment of adults with unresectable locally advanced or metastatic programmed death-ligand 1 (PD-L1)–positive triple-negative breast cancer (TNBC).
This conditional approval is granted to medicines that fill an unmet medical need for serious or life-threatening diseases or conditions, but the FDA may require confirmatory trials to provide verification and description of clinical benefit to allow continued approval.
The approval is based on the phase 3 IMpassion130 trial (NCT02425891), which enrolled 902 patients with unresectable, locally advanced or metastatic TNBC who had not received prior lines of chemo for metastatic disease, according to Genentech.
The multicenter, randomized, double-blind study has been evaluating the drug combination’s efficacy, safety, and pharmacokinetics. Compared with placebo plus nab-paclitaxel, atezolizumab/nab-paclitaxel demonstrated significantly superior progression-free survival (median PFS, 7.4 months vs. 4.8 months; hazard ratio, 0.60; 95% confidence interval, 0.48-0.77; P less than .0001).
The overall survival data for the intention-to-treat population remains immature, but further data will be shared with the FDA in the future, according to Genentech.
No new safety signals were seen in the atezolizumab/nab-paclitaxel arm, and the combination’s safety appeared consistent with the known safety profiles of each medicine individually.
The most common grade 3-4 events (occurring in more than 2% of patients) in the combination arm included low red blood cells, low white blood cells, feeling tired, low blood potassium level, and pneumonia.
The most common side effects (occurring in more than 20% of patients) in the combination arm included hair loss, tingling, nausea, diarrhea, headache, low red blood cells, low white blood cells, and decreased appetite.
Atezolizumab is a monoclonal antibody that binds to the PD-L1 receptor, which could possibly lead to the reactivation of T cells; however, atezolizumab also may interact with other cells in the body. Nab-paclitaxel is an injectable suspension of the common chemotherapy drug.
The Food and Drug Administration has granted accelerated approval for the combination of atezolizumab (Tecentriq) plus nanoparticle albumin–bound paclitaxel (nab-paclitaxel; Abraxane) for the treatment of adults with unresectable locally advanced or metastatic programmed death-ligand 1 (PD-L1)–positive triple-negative breast cancer (TNBC).
This conditional approval is granted to medicines that fill an unmet medical need for serious or life-threatening diseases or conditions, but the FDA may require confirmatory trials to provide verification and description of clinical benefit to allow continued approval.
The approval is based on the phase 3 IMpassion130 trial (NCT02425891), which enrolled 902 patients with unresectable, locally advanced or metastatic TNBC who had not received prior lines of chemo for metastatic disease, according to Genentech.
The multicenter, randomized, double-blind study has been evaluating the drug combination’s efficacy, safety, and pharmacokinetics. Compared with placebo plus nab-paclitaxel, atezolizumab/nab-paclitaxel demonstrated significantly superior progression-free survival (median PFS, 7.4 months vs. 4.8 months; hazard ratio, 0.60; 95% confidence interval, 0.48-0.77; P less than .0001).
The overall survival data for the intention-to-treat population remains immature, but further data will be shared with the FDA in the future, according to Genentech.
No new safety signals were seen in the atezolizumab/nab-paclitaxel arm, and the combination’s safety appeared consistent with the known safety profiles of each medicine individually.
The most common grade 3-4 events (occurring in more than 2% of patients) in the combination arm included low red blood cells, low white blood cells, feeling tired, low blood potassium level, and pneumonia.
The most common side effects (occurring in more than 20% of patients) in the combination arm included hair loss, tingling, nausea, diarrhea, headache, low red blood cells, low white blood cells, and decreased appetite.
Atezolizumab is a monoclonal antibody that binds to the PD-L1 receptor, which could possibly lead to the reactivation of T cells; however, atezolizumab also may interact with other cells in the body. Nab-paclitaxel is an injectable suspension of the common chemotherapy drug.
The Food and Drug Administration has granted accelerated approval for the combination of atezolizumab (Tecentriq) plus nanoparticle albumin–bound paclitaxel (nab-paclitaxel; Abraxane) for the treatment of adults with unresectable locally advanced or metastatic programmed death-ligand 1 (PD-L1)–positive triple-negative breast cancer (TNBC).
This conditional approval is granted to medicines that fill an unmet medical need for serious or life-threatening diseases or conditions, but the FDA may require confirmatory trials to provide verification and description of clinical benefit to allow continued approval.
The approval is based on the phase 3 IMpassion130 trial (NCT02425891), which enrolled 902 patients with unresectable, locally advanced or metastatic TNBC who had not received prior lines of chemo for metastatic disease, according to Genentech.
The multicenter, randomized, double-blind study has been evaluating the drug combination’s efficacy, safety, and pharmacokinetics. Compared with placebo plus nab-paclitaxel, atezolizumab/nab-paclitaxel demonstrated significantly superior progression-free survival (median PFS, 7.4 months vs. 4.8 months; hazard ratio, 0.60; 95% confidence interval, 0.48-0.77; P less than .0001).
The overall survival data for the intention-to-treat population remains immature, but further data will be shared with the FDA in the future, according to Genentech.
No new safety signals were seen in the atezolizumab/nab-paclitaxel arm, and the combination’s safety appeared consistent with the known safety profiles of each medicine individually.
The most common grade 3-4 events (occurring in more than 2% of patients) in the combination arm included low red blood cells, low white blood cells, feeling tired, low blood potassium level, and pneumonia.
The most common side effects (occurring in more than 20% of patients) in the combination arm included hair loss, tingling, nausea, diarrhea, headache, low red blood cells, low white blood cells, and decreased appetite.
Atezolizumab is a monoclonal antibody that binds to the PD-L1 receptor, which could possibly lead to the reactivation of T cells; however, atezolizumab also may interact with other cells in the body. Nab-paclitaxel is an injectable suspension of the common chemotherapy drug.
fMRI might help differentiate MDD and bipolar disorder
The hippocampal functional connectivity (FC) found in patients with major depressive disorder is different from the FC found in patients with bipolar disorder, an analysis of functional MRI data shows.
“ Ahmed Ameen Fateh and his associates wrote in Psychiatry Research: Neuroimaging.
Mr. Fateh and his associates recruited 29 participants with depression and 30 with bipolar disorder, from a mental health center of Chengdu, Sichuan, China. An additional 30 healthy controls were recruited through advertisements, reported Mr. Fateh, a doctoral student at the University of Electronic Science and Technology of China, Chengdu.
Using one-way analysis of variance, the investigators looked at possible differences in the participants’ hippocampal FC. The results showed that some regions exhibited significant differences in the hippocampal FC among the patients with major depression and bipolar disorder.
If their results could be replicated, the researchers wrote, “such differences may yield future trends towards improving the clinical differentiation between these two types of depression with significant therapeutic and prognostic implications.”
SOURCE: Fateh AA et al. Psychiatry Res Neuroimaging. 2019 Jan 12. doi: 10.1016/j.pscychresns.2019.01.004.
The hippocampal functional connectivity (FC) found in patients with major depressive disorder is different from the FC found in patients with bipolar disorder, an analysis of functional MRI data shows.
“ Ahmed Ameen Fateh and his associates wrote in Psychiatry Research: Neuroimaging.
Mr. Fateh and his associates recruited 29 participants with depression and 30 with bipolar disorder, from a mental health center of Chengdu, Sichuan, China. An additional 30 healthy controls were recruited through advertisements, reported Mr. Fateh, a doctoral student at the University of Electronic Science and Technology of China, Chengdu.
Using one-way analysis of variance, the investigators looked at possible differences in the participants’ hippocampal FC. The results showed that some regions exhibited significant differences in the hippocampal FC among the patients with major depression and bipolar disorder.
If their results could be replicated, the researchers wrote, “such differences may yield future trends towards improving the clinical differentiation between these two types of depression with significant therapeutic and prognostic implications.”
SOURCE: Fateh AA et al. Psychiatry Res Neuroimaging. 2019 Jan 12. doi: 10.1016/j.pscychresns.2019.01.004.
The hippocampal functional connectivity (FC) found in patients with major depressive disorder is different from the FC found in patients with bipolar disorder, an analysis of functional MRI data shows.
“ Ahmed Ameen Fateh and his associates wrote in Psychiatry Research: Neuroimaging.
Mr. Fateh and his associates recruited 29 participants with depression and 30 with bipolar disorder, from a mental health center of Chengdu, Sichuan, China. An additional 30 healthy controls were recruited through advertisements, reported Mr. Fateh, a doctoral student at the University of Electronic Science and Technology of China, Chengdu.
Using one-way analysis of variance, the investigators looked at possible differences in the participants’ hippocampal FC. The results showed that some regions exhibited significant differences in the hippocampal FC among the patients with major depression and bipolar disorder.
If their results could be replicated, the researchers wrote, “such differences may yield future trends towards improving the clinical differentiation between these two types of depression with significant therapeutic and prognostic implications.”
SOURCE: Fateh AA et al. Psychiatry Res Neuroimaging. 2019 Jan 12. doi: 10.1016/j.pscychresns.2019.01.004.
FROM PSYCHIATRY RESEARCH: NEUROIMAGING
FDA approves liquid colchicine for gout
for prophylaxis of gout flares in adults, according to a statement from Romeg Therapeutics.
Colchicine has been used in capsule and tablet forms to treat this form of arthritis for decades. An advantage to the new formulation is that it allows physicians to “easily make dose adjustments,” according to the statement.
“Existing therapies do not adequately address the physician’s need to adjust dosages of colchicine to manage the toxicity profile for patients with renal and liver impairments, side effects, common drug-to-drug interactions, and age-related health disorders,” said Naomi Vishnupad, PhD, chief scientific officer of Romeg Therapeutics, in the statement.
According to the prescribing information for the drug on the FDA website, this formulation is indicated for prophylaxis rather than acute treatment of gout flares because the safety profile of acute treatment with it has not yet been studied. It is contraindicated in patients with hepatic and/or renal impairment. Gastrointestinal symptoms were the most commonly reported adverse reactions.
The drug is expected to be available this summer.
for prophylaxis of gout flares in adults, according to a statement from Romeg Therapeutics.
Colchicine has been used in capsule and tablet forms to treat this form of arthritis for decades. An advantage to the new formulation is that it allows physicians to “easily make dose adjustments,” according to the statement.
“Existing therapies do not adequately address the physician’s need to adjust dosages of colchicine to manage the toxicity profile for patients with renal and liver impairments, side effects, common drug-to-drug interactions, and age-related health disorders,” said Naomi Vishnupad, PhD, chief scientific officer of Romeg Therapeutics, in the statement.
According to the prescribing information for the drug on the FDA website, this formulation is indicated for prophylaxis rather than acute treatment of gout flares because the safety profile of acute treatment with it has not yet been studied. It is contraindicated in patients with hepatic and/or renal impairment. Gastrointestinal symptoms were the most commonly reported adverse reactions.
The drug is expected to be available this summer.
for prophylaxis of gout flares in adults, according to a statement from Romeg Therapeutics.
Colchicine has been used in capsule and tablet forms to treat this form of arthritis for decades. An advantage to the new formulation is that it allows physicians to “easily make dose adjustments,” according to the statement.
“Existing therapies do not adequately address the physician’s need to adjust dosages of colchicine to manage the toxicity profile for patients with renal and liver impairments, side effects, common drug-to-drug interactions, and age-related health disorders,” said Naomi Vishnupad, PhD, chief scientific officer of Romeg Therapeutics, in the statement.
According to the prescribing information for the drug on the FDA website, this formulation is indicated for prophylaxis rather than acute treatment of gout flares because the safety profile of acute treatment with it has not yet been studied. It is contraindicated in patients with hepatic and/or renal impairment. Gastrointestinal symptoms were the most commonly reported adverse reactions.
The drug is expected to be available this summer.
New BinaxNOW influenza test gets CLIA waiver
The Food and Drug Administration has granted the reformulated BinaxNOW Influenza A & B Card 2 waived status under the Clinical Laboratory Improvements Amendments for use with Abbott’s DIGIVAL reader, which means the system is relatively simple and has a low risk of erroneous results.
According to Abbott’s press release, the DIGIVAL reader (formerly known as the Alere Reader) automatically interprets this rapid influenza diagnostic test’s results in seconds and is intended to remove subjectivity from the diagnostic process. The BinaxNOW Influenza Card A & B Card 2 is a Class II assay and complies with the FDA’s new reclassification requirements for rapid influenza diagnostic tests.
The Food and Drug Administration has granted the reformulated BinaxNOW Influenza A & B Card 2 waived status under the Clinical Laboratory Improvements Amendments for use with Abbott’s DIGIVAL reader, which means the system is relatively simple and has a low risk of erroneous results.
According to Abbott’s press release, the DIGIVAL reader (formerly known as the Alere Reader) automatically interprets this rapid influenza diagnostic test’s results in seconds and is intended to remove subjectivity from the diagnostic process. The BinaxNOW Influenza Card A & B Card 2 is a Class II assay and complies with the FDA’s new reclassification requirements for rapid influenza diagnostic tests.
The Food and Drug Administration has granted the reformulated BinaxNOW Influenza A & B Card 2 waived status under the Clinical Laboratory Improvements Amendments for use with Abbott’s DIGIVAL reader, which means the system is relatively simple and has a low risk of erroneous results.
According to Abbott’s press release, the DIGIVAL reader (formerly known as the Alere Reader) automatically interprets this rapid influenza diagnostic test’s results in seconds and is intended to remove subjectivity from the diagnostic process. The BinaxNOW Influenza Card A & B Card 2 is a Class II assay and complies with the FDA’s new reclassification requirements for rapid influenza diagnostic tests.
Brilinta reduces MACE in THEMIS trial
The maker of ticagrelor has released top-line results from the phase 3 trial known as THEMIS.
AstraZeneca announced that THEMIS met its primary endpoint of reduction of major adverse cardiovascular events (MACE) among patients with coronary artery disease (CAD) and type 2 diabetes with no history of heart attack or stroke. (MACE is a composite of cardiovascular death, heart attack, and stroke.)
The trial (NCT01991795) explored these risks by comparing ticagrelor (Brilinta) plus aspirin versus aspirin alone. The oral, reversibly binding, direct-acting P2Y12 receptor antagonist is currently indicated for reducing MACE among patients with acute coronary syndrome or a history of MI. According to the press release, the multinational, randomized, double-blind trial is exploring the use of ticagrelor/aspirin among patients with CAD and type 2 diabetes because of this population’s high risk and the lack of treatment options.
Gabriel Steg, MD, THEMIS cochair and professor at Université Paris–Diderot, said in the release that “patients who have both stable coronary artery disease and diabetes are a sizable group which remains at particularly high risk of major adverse cardiac events. The optimal long-term antiplatelet therapy in that group is not fully established.” He added that the full results from the THEMIS trial will be presented later this year.
Ticagrelor comes with risks of significant and sometimes fatal bleeding; as such it is contraindicated for patients with pathological bleeding risk or history of intracranial hemorrhage. Its use is also discouraged among patients with severe hepatic impairment and in patients who are breastfeeding. Although usually self-limiting when related to ticagrelor use, dyspnea was reported in about 14% of patients taking the drug. Dyspnea and bleeding were among the most common adverse reactions seen with ticagrelor.
The maker of ticagrelor has released top-line results from the phase 3 trial known as THEMIS.
AstraZeneca announced that THEMIS met its primary endpoint of reduction of major adverse cardiovascular events (MACE) among patients with coronary artery disease (CAD) and type 2 diabetes with no history of heart attack or stroke. (MACE is a composite of cardiovascular death, heart attack, and stroke.)
The trial (NCT01991795) explored these risks by comparing ticagrelor (Brilinta) plus aspirin versus aspirin alone. The oral, reversibly binding, direct-acting P2Y12 receptor antagonist is currently indicated for reducing MACE among patients with acute coronary syndrome or a history of MI. According to the press release, the multinational, randomized, double-blind trial is exploring the use of ticagrelor/aspirin among patients with CAD and type 2 diabetes because of this population’s high risk and the lack of treatment options.
Gabriel Steg, MD, THEMIS cochair and professor at Université Paris–Diderot, said in the release that “patients who have both stable coronary artery disease and diabetes are a sizable group which remains at particularly high risk of major adverse cardiac events. The optimal long-term antiplatelet therapy in that group is not fully established.” He added that the full results from the THEMIS trial will be presented later this year.
Ticagrelor comes with risks of significant and sometimes fatal bleeding; as such it is contraindicated for patients with pathological bleeding risk or history of intracranial hemorrhage. Its use is also discouraged among patients with severe hepatic impairment and in patients who are breastfeeding. Although usually self-limiting when related to ticagrelor use, dyspnea was reported in about 14% of patients taking the drug. Dyspnea and bleeding were among the most common adverse reactions seen with ticagrelor.
The maker of ticagrelor has released top-line results from the phase 3 trial known as THEMIS.
AstraZeneca announced that THEMIS met its primary endpoint of reduction of major adverse cardiovascular events (MACE) among patients with coronary artery disease (CAD) and type 2 diabetes with no history of heart attack or stroke. (MACE is a composite of cardiovascular death, heart attack, and stroke.)
The trial (NCT01991795) explored these risks by comparing ticagrelor (Brilinta) plus aspirin versus aspirin alone. The oral, reversibly binding, direct-acting P2Y12 receptor antagonist is currently indicated for reducing MACE among patients with acute coronary syndrome or a history of MI. According to the press release, the multinational, randomized, double-blind trial is exploring the use of ticagrelor/aspirin among patients with CAD and type 2 diabetes because of this population’s high risk and the lack of treatment options.
Gabriel Steg, MD, THEMIS cochair and professor at Université Paris–Diderot, said in the release that “patients who have both stable coronary artery disease and diabetes are a sizable group which remains at particularly high risk of major adverse cardiac events. The optimal long-term antiplatelet therapy in that group is not fully established.” He added that the full results from the THEMIS trial will be presented later this year.
Ticagrelor comes with risks of significant and sometimes fatal bleeding; as such it is contraindicated for patients with pathological bleeding risk or history of intracranial hemorrhage. Its use is also discouraged among patients with severe hepatic impairment and in patients who are breastfeeding. Although usually self-limiting when related to ticagrelor use, dyspnea was reported in about 14% of patients taking the drug. Dyspnea and bleeding were among the most common adverse reactions seen with ticagrelor.
FDA approves combo Lonsurf for gastric and GEJ adenocarcinomas
The Food and Drug Administration has approved Taiho’s trifluridine/tipiracil combination Lonsurf for adult patients with either gastric or gastroesophageal junction adenocarcinomas. Specifically, these patients will have been previously treated with at least two lines of chemotherapy that included fluoropyrimidine, a platinum, either a taxane or irinotecan, and if appropriate, HER2/neu-targeted therapy, according to a statement from the company.
The approval was based on the TAGS trial (NCT02500043), which was a global, randomized, phase 3 study that evaluated Lonsurf plus best supportive care versus placebo plus best supportive care. The trial demonstrated prolonged overall survival with the drug combo, compared with that seen with placebo, and thereby met its primary and secondary endpoint. The safety profile seen in the trial was consistent with what’s previously been seen with the drug. Results from this trial were published in The Lancet Oncology.
Warnings and precautions for the drug combination include severe myelosuppression and embryo-fetal toxicity, as well as fatigue, nausea, diarrhea, infections, pyrexia, alopecia, and others. The full prescribing information can be found on the Taiho website.
The Food and Drug Administration has approved Taiho’s trifluridine/tipiracil combination Lonsurf for adult patients with either gastric or gastroesophageal junction adenocarcinomas. Specifically, these patients will have been previously treated with at least two lines of chemotherapy that included fluoropyrimidine, a platinum, either a taxane or irinotecan, and if appropriate, HER2/neu-targeted therapy, according to a statement from the company.
The approval was based on the TAGS trial (NCT02500043), which was a global, randomized, phase 3 study that evaluated Lonsurf plus best supportive care versus placebo plus best supportive care. The trial demonstrated prolonged overall survival with the drug combo, compared with that seen with placebo, and thereby met its primary and secondary endpoint. The safety profile seen in the trial was consistent with what’s previously been seen with the drug. Results from this trial were published in The Lancet Oncology.
Warnings and precautions for the drug combination include severe myelosuppression and embryo-fetal toxicity, as well as fatigue, nausea, diarrhea, infections, pyrexia, alopecia, and others. The full prescribing information can be found on the Taiho website.
The Food and Drug Administration has approved Taiho’s trifluridine/tipiracil combination Lonsurf for adult patients with either gastric or gastroesophageal junction adenocarcinomas. Specifically, these patients will have been previously treated with at least two lines of chemotherapy that included fluoropyrimidine, a platinum, either a taxane or irinotecan, and if appropriate, HER2/neu-targeted therapy, according to a statement from the company.
The approval was based on the TAGS trial (NCT02500043), which was a global, randomized, phase 3 study that evaluated Lonsurf plus best supportive care versus placebo plus best supportive care. The trial demonstrated prolonged overall survival with the drug combo, compared with that seen with placebo, and thereby met its primary and secondary endpoint. The safety profile seen in the trial was consistent with what’s previously been seen with the drug. Results from this trial were published in The Lancet Oncology.
Warnings and precautions for the drug combination include severe myelosuppression and embryo-fetal toxicity, as well as fatigue, nausea, diarrhea, infections, pyrexia, alopecia, and others. The full prescribing information can be found on the Taiho website.
FDA clears test to monitor residual disease in CML
The Food and Drug Administration has cleared Bio-Rad’s digital polymerase chain reaction (PCR) testing solution to monitor patients’ molecular response to treatment for chronic myeloid leukemia.
The QXDx AutoDG ddPCR System combines Bio-Rad’s Droplet Digital PCR technology and the QXDx BCR-ABL %IS Kit, according to the company.
This so-called liquid biopsy test can “precisely and reproducibly” monitor the molecular response to tyrosine kinase inhibitor therapy. The current standard – reverse transcription quantitative PCR – can have variable results, especially at low levels of disease, according to Bio-Rad.
FDA clearance means that the product is “substantially equivalent” to an already-approved product and can be sold in the United States, according to the agency.
The Food and Drug Administration has cleared Bio-Rad’s digital polymerase chain reaction (PCR) testing solution to monitor patients’ molecular response to treatment for chronic myeloid leukemia.
The QXDx AutoDG ddPCR System combines Bio-Rad’s Droplet Digital PCR technology and the QXDx BCR-ABL %IS Kit, according to the company.
This so-called liquid biopsy test can “precisely and reproducibly” monitor the molecular response to tyrosine kinase inhibitor therapy. The current standard – reverse transcription quantitative PCR – can have variable results, especially at low levels of disease, according to Bio-Rad.
FDA clearance means that the product is “substantially equivalent” to an already-approved product and can be sold in the United States, according to the agency.
The Food and Drug Administration has cleared Bio-Rad’s digital polymerase chain reaction (PCR) testing solution to monitor patients’ molecular response to treatment for chronic myeloid leukemia.
The QXDx AutoDG ddPCR System combines Bio-Rad’s Droplet Digital PCR technology and the QXDx BCR-ABL %IS Kit, according to the company.
This so-called liquid biopsy test can “precisely and reproducibly” monitor the molecular response to tyrosine kinase inhibitor therapy. The current standard – reverse transcription quantitative PCR – can have variable results, especially at low levels of disease, according to Bio-Rad.
FDA clearance means that the product is “substantially equivalent” to an already-approved product and can be sold in the United States, according to the agency.
Trametinib effectively treats case of giant congenital melanocytic nevus
according to a case report presented in Pediatrics.
Her nevus covered most of her back and much of her torso and had thickened significantly over the years since initial presentation to the point of disfigurement, even invading the fascia and musculature of the trunk and pelvis, reported Adnan Mir, MD, PhD, of the University of Texas Southwestern Medical Center, Dallas, and his coauthors. Furthermore, she presented with intractable pruritus and pain that interfered with sleep and responded minimally to treatments. Although initial immunohistochemical staining and gene sequencing did not reveal any mutations, such as BRAF V600E, further testing uncovered an AKAP9-BRAF fusion.
There are few if any effective ways of treating GCMNs. With that knowledge, as well as general theories of the mechanism GCMNs in mind, the patient’s health care team decided to try a 0.5-mg daily dose of trametinib when she was 7 years old. Her pruritus and pain resolved completely, and after 6 months of treatment with trametinib, repeat MRI “revealed decreased thickening of the dermis and near resolutions of muscular invasion.” According to the patient’s family, her quality of life improved dramatically.
SOURCE: Mir A et al. Pediatrics. 2019;143(3):e20182469.
according to a case report presented in Pediatrics.
Her nevus covered most of her back and much of her torso and had thickened significantly over the years since initial presentation to the point of disfigurement, even invading the fascia and musculature of the trunk and pelvis, reported Adnan Mir, MD, PhD, of the University of Texas Southwestern Medical Center, Dallas, and his coauthors. Furthermore, she presented with intractable pruritus and pain that interfered with sleep and responded minimally to treatments. Although initial immunohistochemical staining and gene sequencing did not reveal any mutations, such as BRAF V600E, further testing uncovered an AKAP9-BRAF fusion.
There are few if any effective ways of treating GCMNs. With that knowledge, as well as general theories of the mechanism GCMNs in mind, the patient’s health care team decided to try a 0.5-mg daily dose of trametinib when she was 7 years old. Her pruritus and pain resolved completely, and after 6 months of treatment with trametinib, repeat MRI “revealed decreased thickening of the dermis and near resolutions of muscular invasion.” According to the patient’s family, her quality of life improved dramatically.
SOURCE: Mir A et al. Pediatrics. 2019;143(3):e20182469.
according to a case report presented in Pediatrics.
Her nevus covered most of her back and much of her torso and had thickened significantly over the years since initial presentation to the point of disfigurement, even invading the fascia and musculature of the trunk and pelvis, reported Adnan Mir, MD, PhD, of the University of Texas Southwestern Medical Center, Dallas, and his coauthors. Furthermore, she presented with intractable pruritus and pain that interfered with sleep and responded minimally to treatments. Although initial immunohistochemical staining and gene sequencing did not reveal any mutations, such as BRAF V600E, further testing uncovered an AKAP9-BRAF fusion.
There are few if any effective ways of treating GCMNs. With that knowledge, as well as general theories of the mechanism GCMNs in mind, the patient’s health care team decided to try a 0.5-mg daily dose of trametinib when she was 7 years old. Her pruritus and pain resolved completely, and after 6 months of treatment with trametinib, repeat MRI “revealed decreased thickening of the dermis and near resolutions of muscular invasion.” According to the patient’s family, her quality of life improved dramatically.
SOURCE: Mir A et al. Pediatrics. 2019;143(3):e20182469.
FROM PEDIATRICS
Switching long-acting injectables deemed safe in schizophrenia
Patients with schizophrenia who have efficacy or tolerability concerns with paliperidone palmitate or risperidone long-acting injection can be switched safely to aripiprazole lauroxil, a small prospective, open-label study suggests.
“To our knowledge, this is the first prospective study of the safety of switching from other long-acting injectable antipsychotics to [aripiprazole lauroxil],” wrote Brian J. Miller, MD, and his associates.
The 6-month study included 51 patients (mean age, 40.6 years; 72.5% male) who were switched to aripiprazole lauroxil from either of the other long-acting injectables, reported Dr. Miller of Augusta University, Georgia, and his associates. They observed rates of discontinuation for any reason and discontinuation related to the new medication regimen. The study found that, at 6 months, all-cause discontinuation was 30.4% and medication-related discontinuation was 9.2% (Schizophr Res. 2019 Feb 7. doi: 10.1016/jschres.2019.01.38).
Statistically significant improvements were seen with aripiprazole lauroxil based on scores on the Clinical Global Impressions–Severity and the Brief Psychiatric Rating Scale. Safety was assessed by tracking adverse events; the observed adverse events were consistent with aripiprazole lauroxil’s known safety profile. Those improvements and tolerability seen with aripiprazole lauroxil are important, because the reasons for the switch had included experiencing residual symptoms or issues of tolerability with the previous treatment.
“The clinical benefit observed in the study occurred irrespective of the investigator-determined [aripiprazole lauroxil] dosing regimen, suggesting that clinicians have the flexibility to select the regimen that is most compatible with the individual needs of their patients,” the authors added.
The full report can be found in Schizophrenia Research.
Patients with schizophrenia who have efficacy or tolerability concerns with paliperidone palmitate or risperidone long-acting injection can be switched safely to aripiprazole lauroxil, a small prospective, open-label study suggests.
“To our knowledge, this is the first prospective study of the safety of switching from other long-acting injectable antipsychotics to [aripiprazole lauroxil],” wrote Brian J. Miller, MD, and his associates.
The 6-month study included 51 patients (mean age, 40.6 years; 72.5% male) who were switched to aripiprazole lauroxil from either of the other long-acting injectables, reported Dr. Miller of Augusta University, Georgia, and his associates. They observed rates of discontinuation for any reason and discontinuation related to the new medication regimen. The study found that, at 6 months, all-cause discontinuation was 30.4% and medication-related discontinuation was 9.2% (Schizophr Res. 2019 Feb 7. doi: 10.1016/jschres.2019.01.38).
Statistically significant improvements were seen with aripiprazole lauroxil based on scores on the Clinical Global Impressions–Severity and the Brief Psychiatric Rating Scale. Safety was assessed by tracking adverse events; the observed adverse events were consistent with aripiprazole lauroxil’s known safety profile. Those improvements and tolerability seen with aripiprazole lauroxil are important, because the reasons for the switch had included experiencing residual symptoms or issues of tolerability with the previous treatment.
“The clinical benefit observed in the study occurred irrespective of the investigator-determined [aripiprazole lauroxil] dosing regimen, suggesting that clinicians have the flexibility to select the regimen that is most compatible with the individual needs of their patients,” the authors added.
The full report can be found in Schizophrenia Research.
Patients with schizophrenia who have efficacy or tolerability concerns with paliperidone palmitate or risperidone long-acting injection can be switched safely to aripiprazole lauroxil, a small prospective, open-label study suggests.
“To our knowledge, this is the first prospective study of the safety of switching from other long-acting injectable antipsychotics to [aripiprazole lauroxil],” wrote Brian J. Miller, MD, and his associates.
The 6-month study included 51 patients (mean age, 40.6 years; 72.5% male) who were switched to aripiprazole lauroxil from either of the other long-acting injectables, reported Dr. Miller of Augusta University, Georgia, and his associates. They observed rates of discontinuation for any reason and discontinuation related to the new medication regimen. The study found that, at 6 months, all-cause discontinuation was 30.4% and medication-related discontinuation was 9.2% (Schizophr Res. 2019 Feb 7. doi: 10.1016/jschres.2019.01.38).
Statistically significant improvements were seen with aripiprazole lauroxil based on scores on the Clinical Global Impressions–Severity and the Brief Psychiatric Rating Scale. Safety was assessed by tracking adverse events; the observed adverse events were consistent with aripiprazole lauroxil’s known safety profile. Those improvements and tolerability seen with aripiprazole lauroxil are important, because the reasons for the switch had included experiencing residual symptoms or issues of tolerability with the previous treatment.
“The clinical benefit observed in the study occurred irrespective of the investigator-determined [aripiprazole lauroxil] dosing regimen, suggesting that clinicians have the flexibility to select the regimen that is most compatible with the individual needs of their patients,” the authors added.
The full report can be found in Schizophrenia Research.
FROM SCHIZOPHRENIA RESEARCH
LGBTQ youth struggle in foster or unstable housing situations
Lesbian, gay, bisexual, transgender, and questioning (LGBTQ) youth living in foster care or unstable housing are at greater risk for mental health problems, victimization, and getting into fights at school, compared with LGBTQ youth in stable housing and heterosexual youth in foster care, reported Laura Baums, PhD, of the University of Texas at Austin and her coauthors.
This was the finding of analyses of nested data of more than 493,000 students aged 10-18 years from the cross-sectional California Healthy Kids Survey for 2013-2015; 13% identified as LGBTQ. The analyses published in Pediatrics showed LGBTQ youth also were overrepresented in those living situations as compared with the general population.
Less than 1% of the overall sample was in foster care, but 30% of those youth identified as LGBTQ. About 4% of the overall sample lived in unstable housing, and 25% of those youth identified as LGBTQ. So the proportion of LGBTQ youth in foster care or unstable housing was two to three times greater than would be expected than the estimates of LGBTQ youth in nationally representative adolescent samples (that is 11%), Dr. Baums and her associates said.
LGBTQ youth in unstable housing reported lower grades, higher substance/alcohol abuse, higher rates of absenteeism, more fights in school, and more victimization, compared with heterosexual youth in unstable housing and LGBTQ youth in stable housing. Both LGBTQ youth in unstable housing and those in foster care reported higher rates of depression and suicidality in the past year, but the rates for depression were not different from LGBTQ youth in stable housing. Furthermore, African American LGBTQ youth in unstable housing showed poorer outcomes than non-Hispanic white LGBTQ youth in unstable housing, they said.
“” concluded Dr. Baums and her associates. “The findings of this study point to the need for care that is affirming and respectful of youth’s sexual orientation and gender identity.”
The authors reported no relevant financial disclosures. The study was funded by a Eunice Kennedy Shriver National Institute of Child Health and Human Development grant and supported by the Communities for Just Schools Fund and the Priscilla Pond Flawn Endowment at the university.
SOURCE: Baum L et al. Pediatrics. 2019 Feb 11. doi: 10.1542/peds.2017-4211.
Lesbian, gay, bisexual, transgender, and questioning (LGBTQ) youth living in foster care or unstable housing are at greater risk for mental health problems, victimization, and getting into fights at school, compared with LGBTQ youth in stable housing and heterosexual youth in foster care, reported Laura Baums, PhD, of the University of Texas at Austin and her coauthors.
This was the finding of analyses of nested data of more than 493,000 students aged 10-18 years from the cross-sectional California Healthy Kids Survey for 2013-2015; 13% identified as LGBTQ. The analyses published in Pediatrics showed LGBTQ youth also were overrepresented in those living situations as compared with the general population.
Less than 1% of the overall sample was in foster care, but 30% of those youth identified as LGBTQ. About 4% of the overall sample lived in unstable housing, and 25% of those youth identified as LGBTQ. So the proportion of LGBTQ youth in foster care or unstable housing was two to three times greater than would be expected than the estimates of LGBTQ youth in nationally representative adolescent samples (that is 11%), Dr. Baums and her associates said.
LGBTQ youth in unstable housing reported lower grades, higher substance/alcohol abuse, higher rates of absenteeism, more fights in school, and more victimization, compared with heterosexual youth in unstable housing and LGBTQ youth in stable housing. Both LGBTQ youth in unstable housing and those in foster care reported higher rates of depression and suicidality in the past year, but the rates for depression were not different from LGBTQ youth in stable housing. Furthermore, African American LGBTQ youth in unstable housing showed poorer outcomes than non-Hispanic white LGBTQ youth in unstable housing, they said.
“” concluded Dr. Baums and her associates. “The findings of this study point to the need for care that is affirming and respectful of youth’s sexual orientation and gender identity.”
The authors reported no relevant financial disclosures. The study was funded by a Eunice Kennedy Shriver National Institute of Child Health and Human Development grant and supported by the Communities for Just Schools Fund and the Priscilla Pond Flawn Endowment at the university.
SOURCE: Baum L et al. Pediatrics. 2019 Feb 11. doi: 10.1542/peds.2017-4211.
Lesbian, gay, bisexual, transgender, and questioning (LGBTQ) youth living in foster care or unstable housing are at greater risk for mental health problems, victimization, and getting into fights at school, compared with LGBTQ youth in stable housing and heterosexual youth in foster care, reported Laura Baums, PhD, of the University of Texas at Austin and her coauthors.
This was the finding of analyses of nested data of more than 493,000 students aged 10-18 years from the cross-sectional California Healthy Kids Survey for 2013-2015; 13% identified as LGBTQ. The analyses published in Pediatrics showed LGBTQ youth also were overrepresented in those living situations as compared with the general population.
Less than 1% of the overall sample was in foster care, but 30% of those youth identified as LGBTQ. About 4% of the overall sample lived in unstable housing, and 25% of those youth identified as LGBTQ. So the proportion of LGBTQ youth in foster care or unstable housing was two to three times greater than would be expected than the estimates of LGBTQ youth in nationally representative adolescent samples (that is 11%), Dr. Baums and her associates said.
LGBTQ youth in unstable housing reported lower grades, higher substance/alcohol abuse, higher rates of absenteeism, more fights in school, and more victimization, compared with heterosexual youth in unstable housing and LGBTQ youth in stable housing. Both LGBTQ youth in unstable housing and those in foster care reported higher rates of depression and suicidality in the past year, but the rates for depression were not different from LGBTQ youth in stable housing. Furthermore, African American LGBTQ youth in unstable housing showed poorer outcomes than non-Hispanic white LGBTQ youth in unstable housing, they said.
“” concluded Dr. Baums and her associates. “The findings of this study point to the need for care that is affirming and respectful of youth’s sexual orientation and gender identity.”
The authors reported no relevant financial disclosures. The study was funded by a Eunice Kennedy Shriver National Institute of Child Health and Human Development grant and supported by the Communities for Just Schools Fund and the Priscilla Pond Flawn Endowment at the university.
SOURCE: Baum L et al. Pediatrics. 2019 Feb 11. doi: 10.1542/peds.2017-4211.
FROM PEDIATRICS