Dados do Trabalho

Título

SURVIVAL AMONG PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY RECEIVING ETEPLIRSEN FOR UP TO 8 YEARS AND CONTEXTUALIZATION WITH NATURAL HISTORY CONTROLS

Introdução

Eteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip-amenable variants, is associated with attenuation of ambulatory and pulmonary decline versus DMD natural history (NH).

Objetivo

To report overall survival in a US cohort receiving eteplirsen and contextualize these outcomes versus DMD NH.

Método

Data on age at eteplirsen initiation and death/end of follow-up were collected from US patients with DMD through a patient support program. Individual DMD NH data were extracted by digitizing Kaplan–Meier (KM) curves from published systematic and targeted literature reviews. Overall survival age was analyzed using KM curves, contextualized with DMD NH survival curves, and subanalyses considered age groups and duration of eteplirsen exposure. Overall survival time from treatment initiation was also evaluated.

Resultados

Among 579 patients, mean age at eteplirsen initiation was 11.9 years (range, 1.0–35.0) and mean exposure was 3.7 years (0.0–8.6). During a total follow-up of 2119 person-years, median survival age was 32.8 years. DMD NH survival curves extracted from 2 US-based and 2 European study publications (follow-up for 1224 DMD NH controls) showed overall pooled median survival age of 27.4 years. Eteplirsen-treated patients had significantly longer survival from treatment initiation versus age-matched controls (age-adjusted hazard ratio [HR], 0.65; 95% confidence interval [CI], 0.44–0.98; P<0.05). Longer treatment exposure was associated with improved survival (HR, 0.15; 95% CI, 0.05–0.41; P<0.001). Comparisons using different DMD NH cohorts to address common risks of bias yielded consistent findings.

Conclusão

These real-world data suggest that eteplirsen may prolong survival in patients with DMD across a wide age range. As more data become available, the impact of eteplirsen on survival will be further elucidated.

Referências

none

Fonte de Fomento (se houver)

Sarepta Therapeutics, Inc.

Palavras Chave

Duchenne muscular dystrophy; eteplirsen; survival