Background: Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease with average survival of 3-5 years from onset.
Objectives: The objective of the current study was to estimate the time from disease onset to key disease milestones for people living with ALS using a multinational real-world data cohort. These disease milestones reflect the escalating level of healthcare resource utilization associated with disease progression and functional loss in patients with ALS.
Methods: Data were drawn from the Adelphi ALS Disease Specific Programme™, a multinational survey of neurologists and their consulting patients with ALS in France, Germany, Italy, Spain, UK, and the USA. The data were collected between July 2020 and March 2021. Neurologists completed questionnaires on their consulting patients’ demographics and clinical milestones. From disease onset, we report the mean (95% confidence interval) time in months to the following milestones: first consultation for ALS related symptoms, diagnosis, need for caregiver, employment change, need for care facility, and need for walking aid, wheelchair, communication aid, respiratory aid, eye gaze technology, and gastrostomy.
Results: A total of 1003 patients with ALS were included in the analysis. The average age was 61.4 years and 63% were males. The mean (95% CI) time from symptom onset to first consultation was 3.8 (3.5–4.2) months and to diagnosis was 8.0 (7.3–8.8) months. The interval from symptom onset to employment change was 16.2 (13.6–18.8), need for walking aid 17.5 (15.8–19.2), caregiver 18.5 (16.7–20.3) and wheelchair 22.8 (19.9–25.7) months. The time to the need for communication aid was 24.6 (20.8–28.5), respiratory aid 27.3 (24.2–30.4), gastrostomy 28.6 (24.4–32.9), eye gaze technology 29.7 (21.4–38.0) and care facility 30.3 (21.9–38.6) months.
Patients rapidly transitioned through various disease milestones, which demonstrates the rapid progression of ALS. Effective therapies that slow down disease progression may have the potential to reduce humanistic burden of ALS and would help patients, physicians, and caregivers to plan better around these transitions and spread out the costs over time.