Application of the ENCALS predictive survival model in assessing the effect of the 24/44 inclusion criteria in FORTITUDE-ALS.

IF 3.4 4区医学 Q2 CLINICAL NEUROLOGY

Journal of neuromuscular diseases Pub Date : 2025-09-01 Epub Date: 2025-05-05 DOI:10.1177/22143602251336058

Tyrell Simkins Lead, Jeremy M Shefner, Stuart Kupfer, Fady I Malik, Lisa Meng, Stacy A Rudnicki, Jenny Wei, Ruben Pa van Eijk

引用次数: 0

Abstract

FORTITUDE-ALS was a study evaluating reldesemtiv in people living with ALS. Post-hoc analysis identified larger treatment effects in those with symptom onset ≤24 months and baseline ALSFRS-R ≤ 44 (24/44 criteria). Using the ENCALS risk score (RS), we analyzed how the 24/44 criteria changed the eligible population. Of the 272 participants meeting the 24/44 criteria, 73% had very short to intermediate RS compared to 18% not meeting the criteria. Though the 24/44 criteria enriched the FORTITUDE-ALS population with rapidly progressing patients, they did not completely exclude all patients with a very long predicted survival.

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应用ENCALS预测生存模型评估24/44纳入标准对FORTITUDE-ALS的影响。

“坚忍-ALS”是一项评估ALS患者自我认知能力的研究。事后分析发现，症状发作≤24个月且基线ALSFRS-R≤44（24/44标准）的治疗效果更大。使用ENCALS风险评分（RS），我们分析了24/44标准如何改变符合条件的人群。在符合24/44标准的272名参与者中，73%的人有非常短到中等的RS，而18%的人不符合标准。虽然24/44标准丰富了FORTITUDE-ALS人群中快速进展的患者，但他们并没有完全排除所有预测生存期很长的患者。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of neuromuscular diseases Medicine-Neurology (clinical)

CiteScore

5.10

自引率

6.10%

发文量

102

期刊介绍： The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.