Original Article from The New England Journal of Medicine — Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1
NEJMEight infants with SCID-X1 had multilineage immune reconstitution after autologous hematopoietic stem-cell transplantation of marrow stem cells transfected with a lentiviral vector containing IL2RG cDNA after busulfan conditioning. See the results
Correspondence from The New England Journal of Medicine — Treatment Strategies for Deficiency of Adenosine Deaminase 2
NEJMThe manifestations of a deficiency of adenosine deaminase 2, a genetic disease, include early-onset lacunar stroke. This open-label study examined the effects of FFP administration and anti-TNF initiation in patients with ADA2 deficiency. Learn more
Perspective from The New England Journal of Medicine — Diabetes and Disclosure
NEJMAs a medical student with type 1 #diabetes, Kaitlyn Rogers, BS, has to develop workarounds for her surgical rotation, choose her specialty carefully…and decide whether, when, and how to disclose her condition to her educators and to patients.
Perspective from The New England Journal of Medicine — Measles in 2019 — Going Backward
NEJMRecent increases in measles cases in the United States mirror patterns in other countries that had previously eliminated the virus. This resurgence is all the more frustrating since the disease is entirely preventable through vaccination.
Clinical Implications of Basic Research from The New England Journal of Medicine — Boning Up on Irisin
NEJMSkeletal muscle is an endocrine organ that synthesizes and releases myokines (muscle-derived cytokines) in response to contraction; myokines can influence metabolism in other tissues and organs. Learn more
Correspondence from The New England Journal of Medicine — Preliminary Results of a Trial of Atabecestat in Preclinical Alzheimer’s Disease
NEJMThis letter reports on a preliminary analysis of a trial involving participants with brain amyloid deposition who are at risk for #dementia due to Alzheimer’s disease. See the results of this interim analysis of the EARLY trial
Perspective from The New England Journal of Medicine — Lactates and Local Knowledge — A Parable of Teamwork
NEJMOur patient looked bad; his labs worse. We’d never seen such a high lactate level. What was going on? The diagnosis would soon emerge, along with a lesson: the different perspectives and types of expertise within our team were crucial to solving the case.
Original Article from The New England Journal of Medicine — Canagliflozin and Renal Outcomes in Type 2 Diabetes and Nephropathy
NEJMThis double-blind, randomized trial compared canagliflozin with placebo in patients with type 2 diabetes and evidence of kidney disease that was treated with renin–angiotensin system blockade. Read the article abstract: nej.md/2KuM41q #ISNWCN
Review Article from The New England Journal of Medicine — Hepatocellular Carcinoma
NEJMPrimary #livercancer is the fourth most common tumor worldwide. Hepatocellular carcinoma occurs mainly in the context of cirrhosis, hepatitis B or C virus infection, or nonalcoholic steatohepatitis. Underlying liver disease limits therapeutic efficacy.
NEJMPatients with chronic #liverdisease have sustained hepatic inflammation, fibrosis, and aberrant hepatocyte regeneration. Learn more in the review of hepatocellular carcinoma
Perspective from The New England Journal of Medicine — Physicians as Border Guards — The Troubling Exam for Immigrants
NEJMRules proposed by the Trump administration depend on physicians performing “exams” to spot immigrants who are sick enough to become reliant on the government for living costs. The impact would be devastating for immigrants who are denied visas.
Clinical Practice from The New England Journal of Medicine — Obstructive Sleep Apnea in Adults
NEJMObstructive sleep apnea is common and is associated with daytime sleepiness and increased risks of motor vehicle accidents and cardiovascular disease. CPAP is considered first-line therapy for symptomatic or moderate-to-severe obstructive sleep apnea.
Correspondence from The New England Journal of Medicine — Treatment of Progressive Multifocal Leukoencephalopathy with Pembrolizumab
NEJMA patient with variable immunodeficiency and progressive multifocal leukoencephalopathy was treated with five pembrolizumab infusions. The CSF JC viral load went from 119,000 copies per milliliter to undetectable levels. Read more