Curated News: NEJM

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Limiting first-year medical residents to 16-hour work shifts, compared to “flexing” them to allow for some longer shifts, generally makes residents more satisfied with their training and work-life balance, but their training directors more dissatisfied with curtailed educational opportunities. That’s one conclusion of a new study published online March 20 in the New England Journal of Medicine.

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Children with mild to moderate asthma do not benefit from a common practice of increasing their inhaled steroids at the first signs of an asthma exacerbation, according to clinical trial results published in The New England Journal of Medicine. Researchers found short-term increases in inhaled steroids did not prevent attacks in children aged 5 to 11, and may even slow a child’s growth.

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African-American men successfully lowered their high blood pressure to healthy levels when aided by a pharmacist and their local barber, according to a new study from the Smidt Heart Institute.

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Gun injuries fall by 20 percent during the dates of the National Rifle Association’s annual convention. Some 80,000 gun owners attend the NRA’s national convention, including many experienced users. A brief period of gun abstinence, even by experienced, well-trained gun owners, appears to yield safety benefits.

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Larotrectinib, a highly selective TRK kinase inhibitor, shows rapid, potent, and durable efficacy in both adult and pediatric patients with solid tumors that harbor TRK fusions, regardless of tumor type or patient age, according to results from three clinical trials published in the New England Journal of Medicine. The landmark data support the foundation of precision oncology by creating a treatment option for a genetically defined cancer while continuing to validate the concept that comprehensive molecular profiling should be strongly considered in people of all ages with advanced solid tumors.

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Three quarters of patients with a variety of advanced cancers occurring in different sites of the body responded to larotrectinib, a novel therapy that targets a specific genetic mutation. The oral treatment is based on the genetic traits of the tumor and not the organ where the cancer originated.

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A historic study involving researchers from UT Southwestern’s Harold C. Simmons Comprehensive Cancer Center demonstrates the effectiveness of CAR-T therapy, which uses genetically modified immune cells to treat acute lymphoblastic leukemia in children and young adults.

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A new study revealed patients receiving radiofrequency catheter ablation compared to traditional drug therapies for atrial fibrillation (AF), a contributing factor to heart failure, had significantly lower hospitalization and mortality rates. The findings are published in the February 1 issue of the New England Journal of Medicine.

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Updated results from a global clinical trial of the CAR T-cell therapy, tisagenlecleucel, a landmark personalized treatment for a high-risk form of acute lymphoblastic leukemia (ALL), reveal that children and young adults continued to show high rates of durable, complete remission of their disease. Most side effects were short-lived and reversible.

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Results of the global, multicenter, pivotal phase 2 study that led to the first FDA approval of a gene therapy/cell therapy approach known as CAR T-cell therapy, were published today in the New England Journal of Medicine.

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A 25-year-old drug discount program aimed at boosting resources for hospitals treating low-income patients did not deliver on its promise to enhance care for the needy, according to research from Harvard Medical School and the NYU School of Medicine.

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A paper published today in the New England Journal of Medicine reports that solanezumab, a monoclonal antibody-based treatment for Alzheimer’s disease developed by Eli Lilly that targets amyloid plaques, did not significantly slow cognitive decline.

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Smoking is an issue that has been at the heart of public health concerns for decades, with many efforts to restrict tobacco sales, tax cigarettes and sometimes hard-hitting campaigns to get people to quit smoking. But if the tobacco control community has long agreed on the harms of smoking, the place of reducing, rather than eliminating, harm has been hotly contested.

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Patients with monoclonal gammopathy of undetermined significance are at risk of progressing to multiple myeloma or a related cancer ─ even after 30 years of stability. These are the findings of a study by Mayo Clinic researchers published in the Wednesday, Jan. 17, issue of the New England Journal of Medicine.

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Recent studies testing multivalent combinations of three broadly neutralizing antibodies, or bnAbs, have yielded promising results in animal models of HIV prevention. Two investigators at the University of North Carolina at Chapel Hill describe the potential of bnAbs to inform HIV prevention, treatment and cure strategies in a recent article in the New Journal of Medicine.

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Los pacientes con gammapatía monoclonal de significado incierto corren más riesgo de avanzar hacia mieloma múltiple u otro cáncer afín, incluso después de 30 años de estabilidad.

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The approach could represent the first new treatment to improve survival in patients with severe scleroderma in more than four decades.

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Freezing and subsequent transfer of embryos gives infertile couples just as much of a chance of having a child as using fresh embryos for in vitro fertilization (IVF), research from Ho Chi Minh City, Vietnam, and Adelaide, Australia has found.

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In a commentary published in the Jan. 4 issue of the New England Journal of Medicine, UC Davis researcher William Murphy expressed cautious optimism about efforts to genetically engineer hematopoietic stem cells (HSCs) to temporarily resist cell death during transplantation. While these gene therapy approaches could dramatically improve patient outcomes, Murphy argues that their risks must be carefully studied in diverse models.

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Duke Health researchers, publishing in the Jan. 4 issue of the New England Journal of Medicine, found significantly improved survival among patients with a severe form of scleroderma who underwent chemotherapy, whole body radiation and a stem cell transplant. Patients also had less need for immune suppressant drugs after transplant.

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The "mutational burden," or the number of mutations present in a tumor's DNA, is a good predictor of whether that cancer type will respond to a class of cancer immunotherapy drugs known as checkpoint inhibitors, a new study led by Johns Hopkins Kimmel Cancer Center researchers shows. The finding, published in the Dec. 21 New England Journal of Medicine, could be used to guide future clinical trials for these drugs.

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Radiation therapy often is used to treat cancer patients. Now, doctors at Washington University School of Medicine in St. Louis have shown that radiation therapy — aimed directly at the heart — can be used to treat patients with a life-threatening heart rhythm. They treated five patients with irregular heart rhythms, called ventricular tachycardia, who had not responded to standard treatments. The therapy resulted in a dramatic reduction in the number of ventricular tachycardia episodes.

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A study involving the recently approved CD19-targeting chimeric antigen receptor (CAR) T cell therapy shows that 42 percent of patients with aggressive large B-cell lymphoma remained in remission at 15 months following treatment with axi-cel (marketed as Yescarta™).

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Loyola University Medical Center is the only Chicago center that participated in the pivotal clinical trial of a groundbreaking cancer treatment that genetically engineers a patient's immune system to attack cancer cells.

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About half of people with blood clots in the deep veins of their legs develop a complication that involves chronic limb pain and swelling, making it difficult to walk and perform daily activities. A large-scale clinical trial has shown that a risky, costly procedure to remove such clot fails to reduce the likelihood that patients will develop the debilitating complication.

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A team of gene therapy researchers has reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered gene therapy treatment enabled adult participants to safely produce sustained levels of clotting factor that prevented debilitating bleeding episodes. Patients were able to terminate prophylactic treatments--the gene therapy nearly universally eliminated their need for intravenous infusions of manufactured clotting factor.

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Spark Therapeutics (NASDAQ: ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer Inc. (NYSE:PFE), today announced that The New England Journal of Medicine has published interim data as of July 25, 2017, from the Phase 1/2 clinical trial of SPK-9001, an investigational gene therapy for hemophilia B.

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An antibody therapy against a key inflammatory molecule involved in migraines reduces the number of headaches that chronic migraine patients experience per month in a phase III trial.

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Experts from Johns Hopkins Medicine are available to talk about AIDS/HIV, and there is new research from Johns Hopkins on the topic.

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A steep drop in the local incidence of new HIV infections accompanied the rollout of a U.S.-funded anti-HIV program in a large East-African population, according to a study led by researchers at Johns Hopkins Bloomberg School of Public Health and Johns Hopkins School of Medicine.

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The final results of the DAWN study, published in The New England Journal of Medicine, show that select patients with stroke caused by a blood clot can be effectively treated with a procedure to remove the clot mechanically – and that this can be done up to 24 hours after the onset of symptoms.

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A procedure to remove clots from blocked brain vessels – known as thrombectomy - may be beneficial for some stroke patients even if they come in to the emergency room beyond the 6-hour treatment window that current guidelines endorse, according to a groundbreaking study conducted by an international team of physicians and researchers.

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Even 20 years after a diagnosis, women with a type of breast cancer fueled by estrogen still face a substantial risk of cancer returning or spreading, according to a new analysis from an international team of investigators published in the New England Journal of Medicine.

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High protein diets may lead to long-term kidney damage among those suffering from chronic chronic kidney disease, according to research led by nephrologist Kamyar Kalantar-Zadeh, MD, MPH, PhD, of the University of California, Irvine.

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More than half of the babies with infantile-onset spinal muscular atrophy (SMA) who were treated with nusinersen (Spinraza) gained motor milestones, compared to none of the babies in the control group. Infants treated with the drug also had 63 percent lower risk of death. These final results from the 13-month, international, randomized, multicenter, sham procedure-controlled, phase 3 trial called ENDEAR were published in the New England Journal of Medicine.

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A phase 3 trial studying the effects of tolvaptan has found that the drug slowed the rate of decline in kidney function in patients with the most common form of polycystic kidney disease, a condition with no cure.

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Studies show a two-drug combination therapy for cystic fibrosis, using tezacaftor and ivacaftor, is effective. UAB researchers say triple combination therapy is close behind.

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Results from the prospective, randomized, multicenter CULPRIT-SHOCK trial found that an initial strategy of culprit lesion only percutaneous coronary intervention (PCI) reduces the composite of 30-day mortality and/or severe renal failure in patients with multivessel disease and cardiogenic shock complicating acute myocardial infarction.

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New study results from the EXCEL trial comparing the quality of life (QoL) of patients with left main coronary artery disease (LMCAD) receiving percutaneous coronary intervention (PCI) versus coronary artery bypass graft surgery (CABG) found significant and similar QoL improvement at three years, although a greater benefit was observed with PCI at one month.

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A new generation DNA-based Zika vaccine is the first to demonstrate both safety and the ability to elicit an immune response against Zika in humans, according to new research from the Perelman School of Medicine at the University of Pennsylvania, conducted in partnership with The Wistar Institute, Inovio Pharmaceuticals, and GeneOne Life Science, Inc. In results published today in the New England Journal of Medicine, the phase 1 clinical trial showed for the first time that humans who received up to three doses of the vaccine candidate produced an immune response against Zika with minimal adverse effects, opening the door to further clinical trials for this important vaccine candidate.

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Co-Chair of Consensus Committee Advising Trump Administration on Global Health Outlines How the United States Can Bolster Its Global Health Efforts

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The pending termination of DACA may reverse these mental health benefits for the 800,000 DACA beneficiaries, and trigger a public health crisis, according to an essay in the New England Journal of Medicine, co-authored by Atheendar. S. Venkataramani, MD, PhD, an assistant professor of medical ethics and health policy at Perelman School of Medicine at the University of Pennsylvania.

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Antibody treatment reduces rate of flare-ups in patients with a subgroup of treatment-resistant COPD.

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A new drug that boosts bone formation has been shown to reduce the risk of fracture in women with osteoporosis when compared to one of the most commonly used osteoporosis medications, according to findings from UAB reported online in NEJM.

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The immunotherapy drug nivolumab is safer and more effective than ipilimumab—the current standard of care—in treating patients with resected stage III and stage IV melanoma.

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Mucin levels – the proteins that make mucus thick – is abnormally high in chronic bronchitis and mucin concentrations are associated with disease severity. This finding could become the first-ever objective marker of chronic bronchitis and lead to the creation of diagnostic and prognostic tools.

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A major international study has found that the combination of two drugs – rivaroxaban and aspirin -- is superior to aspirin alone in preventing further heart complications in people with vascular disease.

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Two studies provide additional support for lowering systolic blood pressure to an intensive goal of 120 mmHg – far below the standard guidelines of 140 mmHg – to reduce the risk of heart disease in high-risk patients with hypertension. The new research shows that intensive blood pressure control is well-tolerated by patients and is cost-effective in terms of health-related quality of life and financial costs to the healthcare system, and appears online in NEJM on Aug. 24.

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An experimental treatment derived from a potentially deadly microorganism may provide lifesaving help for kidney transplant patients, according to an international study led by investigators at Cedars-Sinai. The study found that treating patients with the drug IdeS® before transplantation significantly reduced, and in most cases eliminated, donor-specific antibodies that can cause rejection or failure of the new organ.

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A major 20-year study provides further evidence that prostate cancer surgery offers negligible benefits to many men with early-stage disease. In such men, who account for most cases of newly diagnosed prostate cancer, surgery did not prolong life and often caused serious complications such as infection, urinary incontinence and erectile dysfunction. The research team included Washington University School of Medicine in St. Louis.