Peller Julián, Trevisan Marcos A, Bujia Gaston, Aguirre Felipe, Shalom Diego E, Taitz Alan, Henze Stephanie, Bastola Silviya, Osik Jason, Shewcraft Ryan A, Jiang Peng, Schwartz Joel, Heiman-Patterson Terry, Sherman Michael E, Wipperman Matthew F, Levy Oren, Shou Guofa, Sillay Karl A, Ostrow Lyle W, Fraenkel Ernest, Berry James D, Navar Bingham Indu, Roitberg Esteban G
Peter Cohen Foundation dba Everything ALS, Seattle, WA, United States.
Universidad de Buenos Aires, Facultad de Ciencias Exactas y Naturales, Departamento de Física - CONICET - Universidad de Buenos Aires, Instituto de Física Interdisciplinaria y Aplicada (INFINA), Buenos Aires, Argentina.
Front Neurol. 2025 Jun 24;16:1588992. doi: 10.3389/fneur.2025.1588992. eCollection 2025.
Monitoring respiratory function is essential for assessing the progression of Amyotrophic Lateral Sclerosis (ALS) and planning interventions. Remote pulmonary function testing offers a promising alternative to in-clinic visits by reducing participant burden and enabling more frequent and accessible measurements.
To evaluate the feasibility and reliability of home-based spirometry in ALS, we built on the Radcliff Study, a fully remote, longitudinal, exploratory study conducted at home by 67 people with ALS (pALS). After an initial training period, participants managed their coaching autonomously, performing spirometry independently or requesting assistance from trained personnel.
We demonstrate that combining flexible coaching with a predefined automatic quality control protocol yields consistent and reliable spirometry results for tracking respiratory function over time. This approach reveals that home-measured Slow Vital Capacity (SVC) and Forced Vital Capacity (FVC) evolve similarly and follow a linear trajectory throughout the study period (7.7 ± 4.0 months), in both slow and fast progressor subpopulations.
The observed linearity in respiratory trajectories supports the potential for early and accurate estimation of progression, reinforcing the feasibility of less frequent monitoring without compromising assessment precision and reducing the burden on both pALS and the healthcare system. Furthermore, our results align with reported in-clinic pulmonary tests, validating remote monitoring as a means to promote more equitable and accessible clinical trial designs.
监测呼吸功能对于评估肌萎缩侧索硬化症（ALS）的进展和规划干预措施至关重要。远程肺功能测试通过减轻参与者负担并实现更频繁、更便捷的测量，为门诊就诊提供了一种有前景的替代方案。
为了评估家庭肺量计在ALS中的可行性和可靠性，我们以拉德克利夫研究为基础，这是一项完全远程的纵向探索性研究，由67名ALS患者（pALS）在家中进行。在初始培训期后，参与者自主管理指导，独立进行肺量计检查或请求受过培训的人员协助。
我们证明，将灵活的指导与预定义的自动质量控制协议相结合，可产生一致且可靠的肺量计结果，用于长期跟踪呼吸功能。这种方法表明，在整个研究期间（7.7±4.0个月），无论是进展缓慢还是进展迅速的亚组人群，在家中测量的慢肺活量（SVC）和用力肺活量（FVC）的变化相似且呈线性轨迹。
观察到的呼吸轨迹线性支持了早期准确估计疾病进展的可能性，加强了减少监测频率而不影响评估精度以及减轻pALS和医疗保健系统负担的可行性。此外，我们的结果与报告的门诊肺功能测试结果一致，验证了远程监测作为促进更公平、更便捷临床试验设计手段的有效性。
