SMA社区未来药物治疗的优先事项：代表410名SMA成人的横断面调查结果。
IF 3.9
3区 医学
Q1 CLINICAL NEUROLOGY
引用次数: 0
摘要
导读：尽管脊髓性肌萎缩症（SMA）的治疗领域取得了进展，但这种疾病患者的需求仍未得到满足。本研究旨在描述成人SMA患者未满足的需求，希望未来的治疗能够解决这些需求，并探讨报告的需求与健康状况和人口统计学特征之间的联系。方法：采用2021年至2023年治愈SMA社区更新调查数据的封闭式问题，评估与肌肉和运动功能、肺功能和球功能以及一般功能相关的需求的重要性。根据SMA类型和活动状态对数据进行分层，并使用Fisher精确检验来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、最大流动性和药物治疗状况的回归，进一步探讨报告需求的差异。结果：样本包括410名成年人，他们回答了未满足需求的问题。大多数为2型或3型SMA（分别为48%和45%）。获得肌肉力量是最常见的未满足需求，其次是改善日常功能、获得新的运动功能和稳定运动功能。分层和回归确定了基于健康状况和人口统计学特征的治疗需求在统计学上的显著差异(p结论：本研究突出了成人SMA患者的治疗重点。本文章由计算机程序翻译，如有差异，请以英文原文为准。
SMA Community Priorities for Future Drug Therapies: Cross-Sectional Survey Findings Representing 410 Adults with SMA.
Introduction: 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.
Methods: 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.
Results: 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.
Conclusion: This research highlights treatment priorities for adults with SMA.
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来源期刊
Neurology and Therapy
CLINICAL NEUROLOGY-
CiteScore
5.40
自引率
8.10%
发文量
103
审稿时长
6 weeks
期刊介绍：
Aims and Scope
Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice.
Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed.
The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Neurology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.
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