Impact of Amyotrophic Lateral Sclerosis Disease Progression on Health Care Resource Use and Health-Related Quality of Life Using MiToS Staging and ALSFRS-R: Analysis of a Real-World Cross-Sectional Survey

OBJECTIVES: To evaluate healthcare resource use (HCRU) and health-related quality of life (HRQOL) in people living with amyotrophic lateral sclerosis (ALS) according to Milano-Torino staging (MiToS) and ALS Functional Rating Scale-Revised (ALSFRS-R) total scores.

METHODS: In the Adelphi ALS Disease-Specific Programme™, a multinational point-in-time survey (July 2020 to March 2021) of neurologists and their consulting patients with ALS, neurologists completed questionnaires for demographics and HCRU, and patients (or caregivers) completed the EQ-5D-5L. The cohort was analyzed according to MiToS staging, a tool measuring ALS progression from stages 0–5 (0–4 reflecting 0–4 functional domains lost; stage 5 is death). Mean HCRU (ALS-related hospitalization and durable medical equipment [DME] use) and HRQOL (EQ-5D-5L, US value set) were evaluated for each stage. The analysis was repeated using ALSFRS-R quartiles as a measure of progression.

RESULTS: Of 1003 patients (mean 61.4 years; 63% males), 68% were in MiToS stage 0, 11% stage 1, 8% stage 2, 5% stage 3, and 8% stage 4. The mean number of ALS-related hospitalizations in the past 12 months increased from stage 0 to 2 and then declined, perhaps reflecting transition to hospice care (stage 0=0.18, stage 1=0.54, stage 2=1.05, stage 3=0.97, stage 4=0.84). Hospitalization duration, healthcare provider consultations, and professional caregiver support increased with MiToS stages. DME (mobility, communication, therapeutic, and respiratory aids) items used increased across stages 0–4 (mean 1.43, 3.41, 5.00, 5.34, 5.84). The EQ-5D-5L worsened from stage 0 to 2, plateauing at stages 3 and 4 (mean 0.53, 0.28, -0.05, 0.01, 0.01). Analysis using ALSFRS-R quartiles showed consistent results.

CONCLUSIONS: In people with ALS, HCRU increased and HRQOL deteriorated with disease progression, as measured by MiToS staging and ALSFRS-R. Effective treatments that lower HCRU and delay HRQOL deterioration are needed to reduce the economic and humanistic burden of ALS for patients as well as healthcare systems.