Abstract
Objective
Patient reported outcome measures (PROMs) can provide researchers with a direct view of patients’ experiences. They are becoming increasingly important tools for evaluating clinical care and research outcomes. There has been little data on the application of PROMs to amyotrophic lateral sclerosis (ALS) care. The objective of this study was to examine the feasibility of PROM collection in an academic ALS clinic and to measure correlations between PROMs and standard ALS clinical outcome measures.
Methods
PROMs were gathered from tablet-based surveys offered to adult patients in the waiting room, prior to ALS outpatient clinic visits. They included a demographic section and two validated surveys: the patient reported outcome measurement information system (PROMIS-10), which generates physical health and mental health subscores, and the quality of life in neurological disorders-fatigue subscale (NeuroQoL-fatigue). The ALS functional rating scale-revised (ALSFRS-R) and other ALS measures were collected by clinic staff as part of routine clinical care.
Results
PROMIS-10 physical and mental health scores correlated positively with the ALSFRS-R score (physical: R = 0.85, p < 0.001; mental: R = 0.58, p = 0.02). NeuroQoL-fatigue scores were inversely correlated with the ALSFRS-R scores—higher fatigue correlated with lower function (R = − 0.72, p = 0.004).
Conclusion
Collection of PROMs is feasible in the context of routine ALS care. PROM scores are highly correlated with validated ALS outcome measures.
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Data availability
The datasets generated during analysis of this study are available from the corresponding author on reasonable request.
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Acknowledgements
We would like to thank all study participants and their families and caregivers for their dedication and contribution to ALS research.
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Fabiola De Marchi: reports no disclosure. James D. Berry has been a consultant to Denali Therapeutics and has research funding from Amylyx Pharmaceuticals, Anelixis Pharmaceuticals, Biogen, Brainstorm Cell Therapeutics, Genentech, MT Pharma of America, ALS Association, ALS Finding A Cure, ALS One, EGL Foundation, and Muscular Dystrophy Association. James Chan: reports no disclosure. Sarah Caldwell: reports no disclosure. Amy Ellrodt: reports no disclosure. Jennifer Scalia: reports no disclosure. Katherine Burke: reports no disclosure. Ton Fang: reports no disclosure. Rachel Clark Sisodia: reports no disclosure. Lidia M.V.R. Moura: reports no disclosure. Lee Schwamm: reports no disclosure. Sabrina Paganoni has received research funding from the Salah Foundation, the Spastic Paraplegia Foundation, the ALS Association, ALS Finding a Cure, the American Academy of Neurology and Amylyx.
Ethical statement
This study has been approved by the appropriate ethics committee and has, therefore, been performed in accordance with the ethical standards (Declaration of Helsinki). In particular, this retrospective chart review and analysis was approved by the Partners Healthcare Human Research Committee.
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De Marchi, F., Berry, J.D., Chan, J. et al. Patient reported outcome measures (PROMs) in amyotrophic lateral sclerosis. J Neurol 267, 1754–1759 (2020). https://doi.org/10.1007/s00415-020-09774-8
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DOI: https://doi.org/10.1007/s00415-020-09774-8