Exploring quality of life in people with slowly-progressive neuromuscular disease

Abstract

Purpose: A qualitative work is conducted to enable later the construction of a health-related quality of life (HRQL) questionnaire for patients with slowly-progressive neuromuscular disease (NMD) such as myopathies and muscular dystrophies.

Methods: The formation of focus groups is an efficient method to perform an in-depth exploration of the aspects of HRQL potentially impaired by NMD. Patients were recruited in France by 4 NMD reference centers. To ensure adequate representativeness in terms of severity, three types of focus groups were formed: (1) Patients able to walk (WP). (2) Patients using a wheelchair (WCP). (3) Patients using a wheelchair and requiring continuous mechanical ventilation (WCMVP). All verbal interactions among group participants were recorded. A qualitative analysis of the verbatim was performed using the framework of the International Classification of Functioning, Disability and Health model (ICF).

Results: A total of 41 patients distributed across five focus groups were interviewed. The verbatim provided 2424 ICF categories. The percentages of mentions of the different ICF categories were calculated and graphically displayed.

Conclusion: The results enabled to identify and quantify the aspects of life that are altered by NMD according to patients. This qualitative work was the first phase of a more ambitious project to develop a new NMD-specific HRQL questionnaire.

Implication of rehabilitation

• Patients with NMD have more to say about the quality of their environment, their social relationships and their psychological state than about their physical symptoms.

• This observation should be compared to clinician perceptions which often focus mainly on the physical symptoms, overlooking those elements that they cannot assess directly.

• Many patients reported relationship issues with various people from their surrounding (e.g., health professionals, acquaintances, colleagues, and strangers).

• In particular, it is essential that health professionals are careful not to make adult patients with NMD feel infantilized.

• This issue can be addressed by making all relevant medical information available and asking for the patient's opinion on any important change in their medical care.

Keywords:

• Focus group
• neuromuscular disease
• patient-reported outcome
• quality of life
• qualitative study

Acknowledgements

We would like to thank all the participating patients who were so kind as to share personal experiences about the consequences of their disease. We also thank Dr Aurore Wolak-Thierry, Dr Moustapha Dramé, Dr Coralie Barbe, Pr Francis Guillemin, Dr Laeticia Percebois-Macadré and Dr Jean-Benoit Hardouin for their valuable help. Special thanks to Sylvie Brimont, our outstanding secretary.

Disclosure statement

The authors have declared no potential conflicts of interest with respect to the research, authorship and publication of this article.

Funding information

This study was supported both financially and logistically by the French Muscular Dystrophy Association (Association française contre les myopathies/AFM-téléthon).