Development of a core outcome measurement set for studies of interventions to support secretion clearance in patients with neuromuscular disorders in the community.

Patients with neuromuscular disorders often develop respiratory muscle weakness. These neuromuscular disorders include congenital diseases such as the muscular dystrophies, and non-congenital diseases such as multiple sclerosis, and amyotrophic lateral sclerosis. High spinal cord injuries can also result in respiratory muscle weakness. Irrespective of the cause, these patients experience weakened cough strength, impeding adequate secretion clearance and leading to increased risk of respiratory infection, a common cause of death (1, 2).

To reduce the risk of respiratory infection, patients with neuromuscular disorders are often supported with interventions to augment secretion clearance. Methods used to augment secretion clearance can be categorised into proximal (‘cough augmentation’) or distal techniques (‘sputum mobilisation’). Proximal techniques include assisted inspiration (e.g., intermittent positive pressure breathing, air stacking, glossopharyngeal breathing, lung volume recruitment), assisted expiration (e.g., manual assisted cough) and assisted inspiration and expiration (e.g., mechanical insufflation-exsufflation). Distal techniques include high frequency chest wall oscillation or compression, intrapulmonary percussive ventilation, and chest wall strapping (3).

Use of secretion clearance techniques in patients with a neuromuscular disorder is varied in terms of choice of intervention, technique used, and availability of equipment between sites and countries (4-6). A frequently cited reason for this practice variation is the lack of evidence for the efficacy of these techniques or guidelines based on consensus opinion. Assessing the comparative efficacy of secretion clearance techniques is currently challenging due to the lack of a consensus in outcomes and measurement instruments. A key stakeholder informed core outcome set COS would assist with homogeneity in the outcomes reported in future trials.


Aim

To establish a core outcome set (COS) for studies of interventions designed to improve secretion clearance in patients with neuromuscular disorders in the community.


Objectives

1. To define and characterise outcomes reported in existing studies of interventions designed to improve secretion clearance in patients with neuromuscular disorders in the community.

2. To obtain consensus on a core outcome set for future studies of interventions designed to improve secretion clearance in patients with neuromuscular disorders.


References
1. Van Ruiten HJ, Marini Bettolo C, Cheetham T, Eagle M, Lochmuller H, Straub V, et al. Why are some patients with Duchenne muscular dystrophy dying young: An analysis of causes of death in North East England. Eur J Paediatr Neurol. 2016;20(6):904-9.
2. Savic G, DeVivo MJ, Frankel HL, Jamous MA, Soni BM, Charlifue S. Causes of death after traumatic spinal cord injury-a 70-year British study. Spinal Cord. 2017;55(10):891-7.
3. Chatwin M, Toussaint M, Goncalves MR, Sheers N, Mellies U, Gonzales-Bermejo J, et al. Airway clearance techniques in neuromuscular disorders: A state of the art review. Respir Med. 2018;136:98-110.
4. Rose L, Adhikari NK, Poon J, Leasa D, McKim DA. Cough Augmentation Techniques in the Critically Ill: A Canadian National Survey. Respir Care. 2016;61(10):1360-8.
5. Swingwood E, Tume L, Cramp F. A survey examining the use of mechanical insufflation-exsufflation on adult intensive care units across the UK. J Intensive Care Soc. 2020;21(4):283-9.
6. Szczepanski R, Cox M, Hashmi-Greenwood M. UK survey of current cough augmentation management in patients with motor neurone disease. European Respiratory Journal. 2020;56(suppl 64):1272.

Contributors

Dr Neeraj Shah (co-PI) Guy’s & St. Thomas’ NHS Foundation Trust, London
Ms Chloe Apps (co-PI) Guy’s & St. Thomas’ NHS Foundation Trust, London
Prof Nicholas Hart, Guy's & St Thomas' NHS Foundation Trust, London
Dr Georgios Kaltsakas, Guy's & St Thomas' NHS Foundation Trust, London
Dr Patrick Murphy, Guy's & St Thomas' NHS Foundation Trust, London
Prof Louise Rose (King’s College London)

Further Study Information

Current Stage: Ongoing
Date: January 2022 - 2022
Funding source(s): To be confirmed


Health Area

Disease Category: Lungs & airways

Disease Name: Neuromuscular disorders

Target Population

Age Range: 14 - 100

Sex: Either

Nature of Intervention: Any

Stakeholders Involved

- Charities
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Families
- Patient/ support group representatives
- Researchers
- Service providers

Study Type

- COS for clinical trials or clinical research
- COS for practice

Method(s)

- 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.

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