Understanding the needs of people with ALS: a national survey of patients and caregivers

Abstract

Objective: Amyotrophic lateral sclerosis (ALS) has profound effects on people with ALS (PALS) and caregivers. There is a paucity of research detailing and comparing PALS and caregiver day-to-day perspectives of ALS. Methods: A survey developed collaboratively by The ALS Association and a panel of experts in ALS care was designed to broadly sample the experience of PALS and caregivers with respect to physical and emotional symptoms, the efficacy of treatment approaches, and goals for future treatments. Specific physical symptoms assessed consisted of fatigue, pain, weakness, shortness of breath, difficulty sleeping, speech problems, depression and other mood changes, and cognitive changes. PALS, caregivers of living patients with ALS (C-LPALS), and caregivers of deceased patients with ALS (C-DPALS) were contacted by email to participate in a 30-minute online survey. Results: 887 PALS, 444 C-LPALS, and 193 C-DPALS responded to the survey. In comparison to PALS, C-LPALS perceived that PALS had significantly higher rates of all surveyed symptoms except for pain and weakness. Caregivers self-reported higher stress levels than PALS (p < 0.001). 35% (135/383) of caregivers reported experiencing a devastating or near devastating financial impact of ALS and 64% (247/383) of caregivers felt their own health had worsened. Caregivers were significantly less likely to perceive a positive response to treatment in comparison to PALS (p < 0.001). Conclusions: PALS and caregivers report a number of symptoms beyond weakness that affect daily life which may be targets of future interventions. There are opportunities to improve services and care for caregivers to reduce the burden of illness.

Keywords:

• Therapy
• epidemiology
• dementia
• ALS
• caregivers
• quality of life
• patient-reported outcome measures

Acknowledgements

We would like to acknowledge all of the people with ALS and caregivers of people with ALS who participated in this study. We would like to acknowledge the contributions of the following people to the design and/or interpretation of the study: Brian Kennedy (Caregiver Representative), Madeline Kennedy, RN, MSN, PhD (PALS Representative), Allison Martin (Consultant, Faegre Baker Daniels Consulting), James Valentine (Hyman, Phelps, & McNamara, PC), David Zook (Faegre Baker Daniels Consulting), Kristina Bowyer (Industry Partner, Ionis), Bonnie Charpentier (Industry Partner, Cytokinetics), Amy Laverdiere (Industry Partner, Cytokinetics), Lasair O’ Callaghan (Industry Partner, Biogen).

Declaration of interest

Kate T. Brizzi and John Bridges report no conflict of interest. Jill Yersak, Calaneet Balas, and Neil Thakur are employees of The ALS Association. Miriam Galvin reports no conflict of interest. Orla Hardiman is Editor in Chief of the ALS and Frontotemporal Degeneration Journal. She has been a consultant for Cytokinetics, Alexion, AveXis, Biogen, and Novartis. Chad Heatwole, MD, MS-CI. Chad Heatwole receives royalties for the use of multiple disease specific instruments. He has provided consultation to Biogen Idec, Ionis Pharmaceuticals, aTyr Pharma, AMO Pharma, Acceleron Pharma, Cytokinetics, Expansion Therapeutics, Harmony Biosciences, Regeneron Pharmaceuticals, Astellas Pharmaceuticals, AveXis and the Marigold Foundation. He receives grant support from Duchenne UK, Parent Project Muscular Dystrophy, Recursion Pharmaceuticals, the National Institute of Neurological Disorders and Stroke, the Muscular Dystrophy Foundation, the Friedreich’s Ataxia Research Alliance, Cure Spinal Muscular Atrophy, and the Amyotrophic Lateral Sclerosis Association. John Ravits reports no conflict of interest. Zachary Simmons has received consulting fees from Cytokinetics and Biohaven, research support from Cytokinetics, Biohaven, Biogen, and Mallinckrodt, and a stipend from Wiley for duties as Editor of Muscle & Nerve. Lucie Bruijn has been a consultant to Ossianix, AveXis, Biohaven, Orthogonal, and Takeda. James Chan reports no conflicts of interest. Richard Bedlack has received research support from the ALS Association, Cytokinetics, Orion, and Ultragenyx. He has received consulting fees from the ALS Association, Biohaven, Biogen, Brainstorm, ITF Pharma, Mallinkrodt, MT Pharma, New Biotic, Woolsey Pharma. James D. Berry has been a consultant for Alexion Therapeutics and Denali Therapeutics. He has received research funding from Amylyx Therapeutics, Anelixis Therapeutics, Biogen, Brainstorm, Cytokinetics, Genentech, ALS Association, Muscular Dystrophy Association, ALS Finding A Cure, and ALS One. He has also been a consultant to CLEAN Nanomedicine.

Additional information

Funding

This work was supported by The ALS Association.