An opinionated take on NEJM highlights for November 2019

A triumph for cystic fibrosis patients (and for Vertex)

Phase 3 confirmation that the benefits seen in early trials from triple-combination therapy of elexacaftor, tezacaftor, and ivacaftor, are sustained and applicable to 90% of the cystic fibrosis patients.  Every metric is unambiguously better: sweat chloride concentration, forced expiration volume (FEV1), respiratory symptom questionnaire, pulmonary exacerbations (down 60%), hospitalizations (down 70%). If anything, with 400 subjects, the study was probably overpowered by at least a factor of 4 to show effect.  It is great news for CF patients most of whom are likely to live a quasi-normal life if they begin treatment early. But it will cost a fortune.  Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele; Realizing the Dream of Molecularly Targeted Therapies for Cystic Fibrosis


A massively scalable clinical study for a massively scalable health intervention

In the right circumstances, the Apple Watch can detect the pulse of its wearer with reasonable accuracy and so it has the potential to detect clinically significant abnormalities of heart rhythm of which the most common is atrial fibrillation, a disorder that leads to a high risk of stroke.   This has now been tested in a remarkable study which enrolled 419,297 participants without using a single clinical site. Everything was managed remotely through the download of an app including patient inclusion criteria, patient consent etc. After about 4 months of monitoring, 2167 patients were flagged with a potential abnormal rhythm, and received an invitation to a telehealth consultation, following which 685 received in the mail an ECG patch capable of recording electrocardiograms for a more definitive diagnosis. Of the 415 patches that yielded data, 153 showed atrial fibrillation and were referred for local care. There are so many implications of this approach that this would deserve a long-form write-up but to begin, I would guess that a study of this type conducted under a classical clinical study model would have a cost well north of 100 million dollars, whereas this was probably executed for less than 10 million (and would cost much less in “production” mode). In addition, if among the 153 patients with a-fib, this eventually avoids 20-50 strokes, it is pretty good value for money.  Large-Scale Assessment of a Smartwatch to Identify Atrial Fibrillation; Watched by Apple


Treatments for Ebola on the horizon

One of the things that make the control of an Ebola epidemic in Sub-Saharan Africa so challenging is that while a mainstay of outbreak management is to isolate those affected, in the absence of good therapeutic options, there are fewer incentives for the sick to let themselves be quarantined and so the spread of the disease is harder to contain.  This may change with promising results for 2 out of 4 anti-viral agents field tested in the DRC that show a relative decrease of mortality by about 30%, and probably better than that for patients treated earlier in the course of the disease. Unfortunately, these are antibodies which means they are expensive to produce, but combined with a vaccine with some efficacy, may lead to a new dynamic for Ebola outbreaks that promotes quicker control and resolution. A Randomized, Controlled Trial of Ebola Virus Disease Therapeutics; Monoclonal Antibody Therapy for Ebola Virus Disease

The New England Journal of Medicine is a premier weekly medical journal covering many topics of interest to the health sector. In this monthly series we offer an opinionated perspective on selected highlights that might be of interest to our clients and others.

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