Department of Rehabilitation, Józef Piłsudski University of Physical Education in Warsaw, Warsaw, Poland.
Department of Neurology, University Clinical Centre of Medical University of Warsaw, Warsaw, Poland.
Neurol Res. 2023 Aug;45(8):708-716. doi: 10.1080/01616412.2023.2194184. Epub 2023 Mar 28.
To date, there are no evidence-based recommendations for physical therapy in amyotrophic lateral sclerosis (ALS). The reason is a low number of related clinical trials (CTs), restricted sample sizes and a high dropout rate. It may influence the profile of the participants, while the final results might not translate to the general ALS population.
To analyze factors affecting the ALS patients' enrollment and retention to the study, and to describe a profile of participants as compared to the eligible group.
A total of 104 ALS patients were offered participation in a CT of low-intensity exercises at home. Forty-six patients were recruited. Demographic and clinical data (El Escorial criteria, site of onset, diagnosis delay, disease duration, amyotrophic lateral sclerosis functional rating scale - revised [ALSFRS-R], Medical Research Council [MRC], hand-held dynamometry) were analyzed every 3 months.
Male gender, younger age and a higher ALSFRS predicted enrollment, while male gender, higher ALSFRS-R and MRC predicted retention in the study. A long commute to the study site and a fast disease progression were the main reasons influencing both enrollment and retention. Despite a high dropout rate, study participants were representative for the general ALS population.
The above demographic, clinical and logistic factors need to be considered when designing studies in ALS population.
迄今为止，尚无针对肌萎缩侧索硬化症（ALS）的物理治疗的循证推荐。原因是相关临床试验（CT）数量较少，样本量有限且脱落率较高。这可能会影响参与者的特征，而最终结果可能无法转化为一般 ALS 人群。
分析影响 ALS 患者参与研究和保留率的因素，并描述与合格组相比参与者的特征。
共向 104 名 ALS 患者提供了参加家庭低强度运动 CT 的机会，其中有 46 名患者被录取。分析了人口统计学和临床数据（埃尔埃斯科里亚尔标准、发病部位、诊断延迟、疾病持续时间、肌萎缩侧索硬化功能评定量表修订版 [ALSFRS-R]、医学研究委员会 [MRC]、手持式测力计），每 3 个月评估一次。
男性、年轻和更高的 ALSFRS 预测了入组，而男性、更高的 ALSFRS-R 和 MRC 预测了研究中的保留率。前往研究地点的通勤时间长和疾病快速进展是影响入组和保留率的主要原因。尽管脱落率很高，但研究参与者仍能代表一般 ALS 人群。
在设计 ALS 人群的研究时，需要考虑上述人口统计学、临床和逻辑因素。
