Time from amyotrophic lateral sclerosis symptom onset to key disease milestones: analysis of data from a multinational cross-sectional survey

Abstract

Objective

To determine the average time from Amyotrophic Lateral Sclerosis (ALS) symptom onset to 11 pre-defined milestones, overall and according to ALS progression rate and geographic location.

Methods

Data were drawn from the Adelphi Real World ALS Disease-Specific Programme^TM, a point-in-time survey of neurologists caring for people living with ALS (pALS) conducted in France, Germany, Italy, Spain, the United Kingdom and the United States from 2020–2021. ALS progression rate was calculated using time since symptom onset and ALS Functional Rating Scale Revised score.

Results

Survey results were available for N = 1003 pALS (progression rate for N = 867). Mean time from symptom onset was 3.8 months to first consultation, 8.0 months to diagnosis, 16.2 months to employment change (part-time/sick leave/retirement/unemployment), 17.5 months to use of a walking aid, 18.5 months to first occurrence of caregiver support, 22.8 months to use of a wheelchair, 24.6 months to use of a communication aid, 27.3 months to use of a respiratory aid, 28.6 months to use of gastrostomy feeding, 29.7 months to use of eye gaze technology and 30.3 months to entering a care facility. Multivariate analysis indicated significant effects of fast (versus slow) progression rate on time to reach all 11 milestones, as well as US (versus European) location, age, body mass index and bulbar onset (versus other) on time to reach milestones.

Conclusions

pALS rapidly reached clinical and disease-related milestones within 30 months from symptom onset. Milestones were reached significantly faster by pALS with fast versus slow progression. Geographic differences were observed.

Keywords:

• Amyotrophic lateral sclerosis
• clinical milestones
• progression
• multinational
• ALS

Disclosure statement

The authors report no conflicts of interest. The authors alone are responsible for the content and writing of this article.

PG, JB and SAR own stock in and are employees of Cytokinetics Inc.

JM, JW, LE, NB, HI, OT and GC are employees of Adelphi Real World

Data collection was undertaken by Adelphi Real World as part of an independent survey, entitled the Adelphi ALS Disease Specific Programme™, sponsored by multiple pharmaceutical companies, one of which was Cytokinetics Inc. The study described here using data from the Adelphi ALS Disease Specific Programme was funded by Cytokinetics Inc.

Medical writing support under the guidance of the authors was provided by Bethan Hahn, PhD, on behalf of Adelphi Real World and Cytokinetics, in accordance with Good Publication Practice guidelines (31).

Data deposition

All data, i.e. methodology, materials, data and data analysis, that support the findings of this survey are the intellectual property of Adelphi Real World. All requests for access should be addressed directly to Jennifer Mellor at [email protected].

Table 5. Time from symptom onset to milestones according to region (US or Europe).