Peterson Ilse S, Chitale Ramaa, Curry Mary A, Belter Lisa T
Faegre Drinker Consulting, Washington, DC, USA.
Contractor to Faegre Drinker Consulting, Washington, DC, USA.
Neurol Ther. 2025 Jun;14(3):1083-1092. doi: 10.1007/s40120-025-00753-7. Epub 2025 May 3.
Despite advances in the therapeutic landscape for spinal muscular atrophy (SMA), unmet needs for those with this condition persist. This study seeks to characterize unmet needs that adults with SMA hope future therapies will address and explores associations between reported needs and health status and demographic characteristics.
Close-ended questions from 2021 to 2023 Cure SMA Community Update Survey data were used to assess the importance of needs related to muscle and motor function, lung function and bulbar function, and general functioning. Data was stratified by SMA type and mobility status, and Fisher's exact tests were used to assess for statistically significant differences based on these characteristics. Variations in reported needs were further explored with regressions controlling for sex, education, maximum mobility, and drug treatment status.
The sample included 410 adults who answered questions on unmet needs. Most had type 2 or type 3 SMA (48% and 45%, respectively). Gaining muscle strength was the most frequently reported unmet need, and followed by improving daily functioning, achieving new motor function, and stabilizing motor function. Stratifications and regressions identified statistically significant differences in treatment needs based on health status and demographic characteristics (p < 0.05). Most notably, people with more severe types of SMA and lower mobility were more likely to report items related to lung function and bulbar function as important.
This research highlights treatment priorities for adults with SMA.
尽管脊髓性肌萎缩症（SMA）的治疗领域取得了进展，但患有这种疾病的患者仍存在未满足的需求。本研究旨在描述成年SMA患者希望未来治疗能够解决的未满足需求，并探讨报告的需求与健康状况和人口统计学特征之间的关联。
使用2021年至2023年治愈SMA社区最新调查数据中的封闭式问题，评估与肌肉和运动功能、肺功能和延髓功能以及总体功能相关需求的重要性。数据按SMA类型和活动状态分层，并使用Fisher精确检验评估基于这些特征的统计学显著差异。通过控制性别、教育程度、最大活动能力和药物治疗状态的回归分析，进一步探讨报告需求的差异。
样本包括410名回答了未满足需求问题的成年人。大多数人患有2型或3型SMA（分别为48%和45%）。增强肌肉力量是最常报告的未满足需求，其次是改善日常功能、获得新的运动功能和稳定运动功能。分层和回归分析确定了基于健康状况和人口统计学特征的治疗需求存在统计学显著差异（p<0.05）。最值得注意的是，SMA类型更严重且活动能力较低的人更有可能报告与肺功能和延髓功能相关的项目很重要。
本研究突出了成年SMA患者的治疗重点。
