Proposing a Core Outcome Set for Physical Activity and Exercise Interventions in People With Rare Neurological Conditions.

Gita Ramdharry,Michael Robling,Valentina Buscemi,Monica Busse,Lynn Rochester,Lorna Paul,Annette Boaz,Thomas Jaki,Rebecca Playle,Jonathan Marsden,Helen Dawes,Elizabeth Randell,Fiona Jones

doi:10.3389/fresc.2021.705474

Abstract

Rare neurological conditions (RNCs) encompass a variety of diseases that differ in progression and symptoms but typically include muscle weakness, sensory and balance impairment and difficulty with coordinating voluntary movement. This can limit overall physical activity, so interventions to address this are recommended. The aim of this study was to agree a core outcome measurement set for physical activity interventions in people living with RNCs. We followed established guidelines to develop core outcome sets. Broad ranging discussions in a series of stakeholder workshops led to the consensus that (1) physical well-being; (2) psychological well-being and (3) participation in day-to-day activities should be evaluated in interventions. Recommendations were further informed by a scoping review of physical activity interventions for people living with RNCs. Nearly 200 outcome measures were identified from the review with a specific focus on activities or functions (e.g, on lower limb function, ability to perform daily tasks) but limited consideration of participation based outcomes (e.g., social interaction, work and leisure). Follow on searches identified two instruments that matched the priority areas: the Oxford Participation and Activities Questionnaire and the Sources of Self-Efficacy for Physical Activity. We propose these scales as measures to assess outcomes that are particularly relevant to assess when evaluating physical activity interventions mong people with RNCs. Validation work across rare neurological conditions is now required to inform application of this core outcome set in future clinical trials to facilitate syntheses of results and meta-analyses.

Highlights

Rare neurological conditions (RNCs), where cases are ≤40 per 100,000 population [1], collectively incur a significant cost burden to healthcare, social care services and informal care [2]
This study focuses on the development of an agreed standardized set of outcomes termed a “core outcome set” [21] that should be at a minimum measured and reported in trials of physical activity interventions in people living with RNCs
We focused on groups of progressive RNCs, namely neuromuscular diseases, Ataxias, Huntington’s Disease (HD), Atypical Parkinson Diseases (AP), including Progressive Supranuclear Palsy, Multiple Systems Atrophy, Corticobasal Degeneration, Motor Neuron Diseases (MND) and Hereditary Spastic Paraparesis (HSP)

Summary

Introduction

Rare neurological conditions (RNCs), where cases are ≤40 per 100,000 population [1], collectively incur a significant cost burden to healthcare, social care services and informal care [2]. Common approaches to improve fitness (e.g., cardiovascular and strength training), activity (e.g., balance and gait training) and participation levels (e.g., supported self-management) are often implemented in clinical practice [4,5,6,7,8]. Whilst trials of physical activity interventions in RNCs highlight the potential of physical activity interventions to improve fitness and function [10,11,12,13] they are often small studies that fail to influence clinical practice. If we are to ensure that research is relevant and able to influence clinical practice and future research, we need to ensure the use (and reporting) of standardized, relevant outcome measures within the field that are applicable to people living with the conditions [15, 16]. Core outcome sets have been agreed for specific target conditions for example cancer, rheumatology and chronic pain as well as for specific care pathways for example maternity care [17, 18]

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