Understanding the perceptions and experiences of adults with early-onset neuromuscular disorders using non-invasive ventilation in making healthcare decisions
Introduction Adults with early-onset neuromuscular disorders (NMDs) experience increasing physical limitations and multi-system complications which require ongoing, multifaceted decisions about their care and support. Contemporary and evolving treatment options have introduced greater prognostic uncertainty, adding further complexity. This study addresses the lack of data drawn directly from affected individuals regarding their actual and preferred roles in healthcare-related decision-making, and aims to provide an in-depth understanding of the supportive factors and barriers that they identify. Methods A purposive sample of participants was taken from a larger quantitative study. Semi-structured individual interviews were used to obtain qualitative data. Interviews were transcribed verbatim and evaluated using inductive thematic analysis. Results Twelve participants (50% female, aged 25–55 years) with a range of NMDs ( e.g. , muscular dystrophies, spinal muscular atrophy) were interviewed. All required long-term non-invasive ventilation to manage chronic respiratory failure. Four themes were identified: 1) Empowerment and independence, 2) Being known and understood by others, 3) Taking a pragmatic approach, and 4) Barriers to holistic long-term care. Conclusions Adults with early-onset NMDs are active, informed decision-makers whose autonomy is supported by trusted relationships, relevant information, and opportunities to reflect on changing health needs. They balance clinical priorities with independence, wellbeing, and daily life. Peer, family, and clinical partnerships facilitate meaningful engagement. Fragmented care and limited support during changes and transitions can hinder timely and person-centred decisions. Embedding psychological support, and strengthening continuity and coordination of services offer opportunities to enhance shared decision-making and deliver care aligned with patients’ values and lived experience.
Authors
- Lydia Spurr (ORCID: https://orcid.org/0000-0002-0386-0802)
- Hui‐Leng Tan (ORCID: https://orcid.org/0000-0001-6917-3434)
- James Tighe
- Anita K Simonds
Institutions
- Royal Marsden NHS Foundation Trust (GB)
- Royal Brompton Hospital (GB)
Publication Details
- Journal
- ERJ Open Research
- Published
- 2026-03-12
- DOI
- https://doi.org/10.1183/23120541.01100-2025
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00