Development of clinical practice guidelines for allied health and nursing assessment and management of Duchenne muscular dystrophy

Abstract

Purpose

To provide evidence-based guidance specific to allied health and nursing practice for the assessment and management of individuals with Duchenne muscular dystrophy (DMD).

Materials and methods

Thirteen key focus areas were identified in consultation with health professionals and consumer advocacy groups. A series of systematic literature reviews were conducted to identify assessment and management strategies for each key focus area. A consensus process using modified Delphi methodology, including an Australia-New Zealand expert consensus meeting, was conducted. Recommendations underwent consultative review with key groups before being finalised and prepared for dissemination.

Results

This clinical practice guideline (CPG) generated 19 evidence-based recommendations, 117 consensus-based recommendations and five research recommendations across the 13 focus areas to inform allied health assessment and management of individuals with DMD.

Conclusions

The resulting recommendations can be used in conjunction with existing medical CPGs to improve, standardise and advocate for allied health and rehabilitation care in DMD. The process used here may be useful for the development of CPGs in other rare diseases.

Implications for rehabilitation

• Implementation-ready evidence-based statements to guide clinical care of individuals with DMD are provided with the potential to improve participation, function in the community and quality of life.

• A model for developing best practice statements for other rare neurological diseases is described.

• Allied health and nursing health professionals should focus research efforts to generate quality evidence to support rehabilitation practice.

Keywords:

• Neuromuscular disease
• rehabilitation
• evidence based
• GRADE
• COSMIN

Correction Statement

This article has been corrected with minor changes. These changes do not impact the academic content of the article.

Acknowledgements

We would like to thank the following organisations for their input to the development of the scope of the guidelines and their review of the recommendations: Duchenne Foundation*, Save Our Sons*, Muscular Dystrophy Association Western Australia, Muscular Dystrophy Australia Victoria, Muscular Dystrophy New Zealand, Muscular Dystrophy Association South Australia, Muscular Dystrophy Association New South Wales, Muscular Dystrophy Association Queensland. *After the scope consultation process, these two organisations merged to form Save Our Sons Duchenne Foundation.

Disclosure statement

Dr. Davidson reports grants from Neuromuscular Research Foundation Trust, grants from Richdale Charitable Trust, grants from National Health & Medical Research Council RARE-Bestpractices [1055131], grants from National Health & Medical Research Council Centre of Research Excellence in Neuromuscular Disorders [ID #1031893], during the conduct of the study; and Lead Investigator on Nutriceuticals in DMD, an investigator-initiated trial of nutritional supplements in DMD funded by Duchenne Foundation, Muscular Dystrophy Australia (VIC), Save Our Sons Duchenne Foundation. Dr. Bray reports grants from Neuromuscular Research Foundation Trust, grants from Richdale Charitable Trust, grants from National Health & Medical Research Council RARE-Bestpractices [1055131], grants from National Health & Medical Research Council Centre of Research Excellence in Neuromuscular Disorders [ID #1031893], during the conduct of the study. Dr. Rose reports personal fees from ATOM International Ltd, outside the submitted work. Miriam Rodrigues reports grants from Neuromuscular Research Foundation Trust, grants from Richdale Charitable Trust, during the conduct of the study. A/Prof. Corben reports grants from Medical Research Futures Fund Next Generation Clinical Researchers Program, grants from Friedreich's Ataxia Research Alliance, USA, grants from Medical Research Futures Fund Project Grant, grants from Rebecca L Cooper Project Grant, outside the submitted work. Dr. North has nothing to disclose. Prof. Ryan reports grants from National Health & Medical Research Council Centre of Research Excellence in Neuromuscular Disorders [ID #1031893], during the conduct of the study; grants and personal fees from Biogen, personal fees from Roche, personal fees from Novartis, personal fees from PTC Therapeutics, outside the submitted work. Dr. Burns reports grants from Neuromuscular Research Foundation Trust, grants from Richdale Charitable Trust, during the conduct of the study; personal fees from Pharnext SA, outside the submitted work.

Additional information

Funding

This work was supported by the Neuromuscular Research Foundation Trust and the Richdale Charitable Trust to support the expert consensus meeting. Zoe Davidson and Paula Bray were supported by funding from the Australian National Health & Medical Research Council RARE-Bestpractices [ID# 1055131] and from the Australian National Health & Medical Research Council Centre of Research Excellence in Neuromuscular Disorders [ID #1031893].