In a Phase 1 trial, patients with refractory lymphoid cancers, an antibody-drug complex directed against ROR1 had no unexpected toxicities. About half of the patients with mantle cell lymphoma and diffuse large B-cell lymphoma had clinically meaningful responses.
In January 2022, the NEJM Group will be publishing a new journal, NEJM Evidence. This monthly, peer-reviewed, online-only, general medical journal will publish original research, along the full spectrum of clinical investigation, that takes ideas and turns them into reality.
Furian and colleagues report on the results of two randomized controlled trials testing the use of acetazolamide to prevent the adverse effects of altitude on healthy older persons and in people with COPD. They find that acetazolamide decreased the incidence of altitude related adverse health events (primarily hypoxemia) in both populations with no evidence of adverse events.
The effect of increased water intake on kidney cyst growth in patients with polycystic kidney disease was compared for two groups randomly assigned to either prescribed or ad libitum water intake. Over 3 years, there was no difference in height-corrected total kidney volume between the groups.
This phase 1b/2a, randomized, double-blind, placebo-controlled, dose-escalation trial tested low-dose subcutaneous aldesleukin (recombinant IL-2) in patients with ischemic heart disease. Low-dose IL-2 expanded Tregs, without adverse events of major concern. Single-cell RNA-sequencing of circulating immune cells was used to provide mechanistic assessment of the treatment’s effects.
If a patient with autosomal dominant polycystic kidney disease could drink enough water to suppress arginine vasopressin release, would cyst growth be attenuated, thereby reducing the decline in kidney function over time? Louise M. Moist, M.D. discusses this randomized controlled trial.
Screening for colorectal cancer is widespread and successful but screening programs across the globe differ in their recommendations. In this article, Helsingen and Kalager review the evidence for different approaches to colorectal cancer screening and propose a framework for the evaluation of screening programs going forward.
This randomized placebo-controlled trial tested MV140, a sublingual preparation of whole-cell inactivated bacteria, in women with recurrent urinary tract infection (UTI). MV140 administered sublingually for either 3 or 6 months decreased UTI incidence and prevented recurrence for up to 1 year compared with placebo, without serious adverse events.
This article presents a frequentist (showing no signifiicant difference) and Bayesian (in which the posterior probability that balanced crystalloids reduced mortality was 89.5%) systematic review of randomized clinical trials comparing balanced crystalloids with saline in critically ill adults with the primary outcome of 90-day mortality.
In a patient with early-stage lung cancer, is resection by video-assisted thoracoscopic surgery (VATS) versus open resection superior with respect to the postoperative recovery? This question was addressed in a multicenter randomized trial in more than 500 patients. At 5 weeks after surgery, physical function at 5 weeks mean was 73 in the VATS group and 67 in the open surgery group (function scores range from 0 to 100, with higher scores indicating better function). Of the participants allocated to VATS, 30.7% had serious adverse events after discharge compared with 37.8% of those allocated to open surgery. At 52 weeks, there were no differences in cancer progression-free survival.
DSMBs look after the welfare of patients enrolled in interventional clinical trials. DSMBs monitor for early establishment of efficacy, findings of harm, futility in obtaining a meaningful outcome, or changes in the ecology of care that render moot the question a trial aims to answer. This article opens a series of NEJM Evidence reviews about DSMBs.
When investigators in a 2013 RCT received their unblinded results, the data forced them to make a difficult decision – do they stay within conventional guidelines for data analysis or forge a new path? This Clinical Trial Case Study tells the story of how they dealt with the data dilemma for a heart failure treatment that affects millions of people.
A 34-year-old woman is scheduled to undergo surgery to manage a torn anterior cruciate ligament in her left knee. Her only medication is an estrogen- and progestin-containing oral contraceptive pill (OCP). Should she stop her combined oral contraception to reduce the risk of a postoperative blood clot?
This Patient Platform is from Moses Taylor, a participant in the Systolic Blood Pressure Intervention Trial (SPRINT) that was published in the New England Journal of Medicine in November 2015.