Dados do Trabalho

Título

Effect of ataluren on upper limb function in nmDMD patients from the STRIDE Registry

Introdução

Strategic Targeting of Registries and International Database of Excellence (STRIDE; NCT02369731) is an international, observational registry evaluating the long-term safety and effectiveness of ataluren in individuals with nonsense mutation Duchenne muscular dystrophy (nmDMD) in real-world clinical practice.

Objetivo

This study investigated whether patients in the STRIDE Registry experienced a slower decline in upper limb function versus patients with DMD receiving standard of care (SoC) alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (NCT00468832).

Método

Data were extracted on 31 January 2023. Upper limb function was assessed using the PUL module in the STRIDE Registry and the Brooke Scale in the CINRG cohort. Kaplan–Meier analyses estimated the age at loss of upper limb function (loss of overhead reach, loss of hand-to-mouth function and loss of distal hand function).

Resultados

The mean age at the last assessment was 14.0 years and 15.2 years in propensity score-matched STRIDE and CINRG cohorts (N=277 per cohort), respectively. The median (95% CI) ages at loss of overhead reach (STRIDE vs CINRG) were 15.8 (12.1, 19.2) and 12.7 (10.3, 14.3) years, respectively (HR [95% CI], 0.412 [0.220, 0.771]; p=0.0095). In CINRG patients, the median (95% CI) age at loss of hand-to-mouth function was 20.5 years (17.1, NC) and the median age at loss of distal hand function was 30.1 years (27.6, NC). These outcomes were not yet estimable for STRIDE Registry patients.

Conclusão

These results indicate that treatment with ataluren plus SoC in routine clinical practice may help preserve upper limb function in patients with advanced nmDMD.

Referências

1.Mercuri E, Osorio AN, Muntoni F, Buccella F, Desguerre I, Kirschner J, et al. Safety and effectiveness of ataluren in patients with
nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022
interim analysis. J Neurol. 2023;270(8):3896–913.
2.Data on file

Palavras Chave

DMD; ataluren; Standard-of-care