Background: Several treatments are used in the community to improve secretion clearance for patients with neuromuscular disease (NMD). However, the optimal intervention remains unclear with further research required. We aimed to develop a core outcome set (COS) for studies investigating secretion clearance interventions used in the community by patients with NMD.
Methods: We conducted a scoping review, qualitative interviews with patients/family, modified e-Delphi survey, and consensus meeting. We recruited health care professionals, patients, and caregivers. Delphi participants were provided a 9-point Likert scale to score outcomes as “not important” (1– 3), “important but not critical” (4–6), or “critical” (7–9). Those scored as critical for inclusion were discussed at the consensus meeting using nominal group technique methods to achieve final consensus.
Results: Ninety participants were recruited for the e-Delphi. Twenty-nine outcomes identified from the scoping review and qualitative interviews were taken forward to Round 1. Eleven additional outcomes were suggested by participants during Round 1. Forty outcomes were presented in Round 2. Sixteen outcomes were voted as critical for inclusion and taken forward to the consensus meeting (20 participants). The final COS includes measured cough strength/power, burden of respiratory illness, patient-reported effectiveness of secretion clearance, patient-reported experience of airway clearance, quality of life, adherence to secretion clearance intervention, and adverse events related to secretion clearance intervention.
Conclusions: This COS should now be included in all trials investigating secretion clearance interventions in the community for patients with NMD. Next steps are to identify measurement tools and characteristics such as measurement time points for these outcomes using COSMIN methodology.
Neeraj M. Shah, Chloe Apps, Reshma Amin, Georgios Kaltsakas, Nicholas Hart, Patrick B. Murphy, and Louise Rose
Disease Category: Lungs & airways
Disease Name: Neuromuscular disorders
Age Range: 14 - 100
Sex: Either
Nature of Intervention: Any
- Charities
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Families
- Patient/ support group representatives
- Researchers
- Service providers
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Interview
- Literature review
- Nominal group technique (NGT)
- Systematic review
Item generation
1 Scoping Review: we will extract data on primary and secondary outcomes reported, measures used and measurement time-points from studies using a scoping review methodology.
2. Interviews with key stakeholders and end users including patients with a neuromuscular disorder and family members: we will conduct qualitative interviews with patients with neuromuscular disorders and their family members to elicit outcomes they consider important to measure in future studies of interventions designed to improve secretion clearance.
Consensus Building
1. We will conduct a modified Delphi study recruiting an international expert panel representing key stakeholders and end users including patients and family members.
2. We will establish consensus on outcomes for inclusion in a Core Outcome Set (COS).
3. If consensus on a core outcome set is not achieved via the Delphi process, we will host a virtual consensus meeting using nominal group technique and voting to establish the core outcomes.