2024 American Academy of Neurology Abstract Website

Eufrosina Young¹, Dongliang Wang², Birendra Sah⁴, Dragos Manta⁴, Urvi Desai⁵, George Slavinski⁴, Lena Deb³, Marielle Posmik³, Takuya Kudou⁶, Kinjal Patel⁷, Stephen Apple⁷, Benjamin Brooks⁸
¹Upstate University Hospital, ²Biostatistics, ³Neurology, Upstate University Hospital, ⁴Pulmonary, Upstate Medical University, ⁵Dept of Neurology, CMC, ⁶Mitsubishi Tanabe Pharma Corporation, ⁷Mitsubishi Tanabe Pharma America, ⁸Clinnical Trials Planning LLC

Objective:

Background:

Pilot Implementation study of At-Home Telespirometry (AHT) measurement of Erect-seated forced vital capacity (eFVC) demonstrated excellent precision (Bland-Altman), repeatability, feasibility of longitudinal measurement, increased assessment of supine forced vital capacity (sFVC) in home situation and differential rate of loss of eFVC as function of initial eFVC at baseline [ Young 2023, Narapureddy 2023 ].

Slope measurements of eFVC change differ by stratification of initial eFVC at baseline in parallel with increased rate of eFVC loss and increased rate of number of eFVC 3 % predicted monthly drops in cohort with eFVC < 60 % predicted at baseline [ Young 2023, Brooks 2023 ].

Design/Methods:

98 ALS subjects from two ALSA-certified ALS Clinics in the US (SUNY Upstate, Atrium Health) measured erect-seated and supine slow vital capacity [ eSVC, sSVC ] from home using a portable spirometer (MIR, Italy), ZEPHYRx mobile application and ZEPHYRx Provider Dashboard over 6 months. Cohorts stratified according to <60%, 60-80% and > 80% predicted eSVC at baseline are followed longitudinally to define eSVC and sSVC changes between cannonical quarterly ( q3month ) in-clinic visits to assess whether clinically significant changes in eSVC and sSVC identify respiratory complications requiring initiation of ventilator support before the next in-clinic visit

Results:

This prospective study [NCT05106569] has nearly completed recruitment of 100 ALS subjects. Baseline characteristics of enrolled ALS subjects are similar to ALS subjects completing the pilot FVC study [ Young 2023]. An interim analysis of the first 50 subjects to complete the study will be presented.

Conclusions:

Stratification of eSVC obtained from baseline in-clinic measurement is predictive of ALS disease trajectory. This clinical study, using AHT to measure eSVC and sSVC between canonical in-clinic visits at home, will identify clinically significant, between clinic visit eSVC and sSVC changes, that may require earlier respiratory management than would occur from quarterly in-clinic visits.