Q&A: Guideline provides better respiratory care for patients with neuromuscular diseases

Patients with neuromuscular diseases face an increased risk for respiratory muscle weakness, which can contribute to various health and mental problems that take a significant toll on their lives.

There is limited guidance on managing respiratory muscle weakness associated with neuromuscular diseases, Akram Khan, MD, associate professor of pulmonary, allergy and critical care medicine at Oregon Health & Science University, told Healio.

With this knowledge, Khan and 11 clinicians collaborated to write a clinical practice guideline, published in CHEST, on managing respiratory issues in patients with neuromuscular weakness.

Through a systematic review of 128 studies related to this topic, the clinicians established 15 graded recommendations, a good practice statement and a consensus-based statement that can be used in practice. In the recommendations section, researchers cover patients with neuromuscular diseases who also have/are at risk for chronic respiratory failure, sleep-related breathing disorders, sialorrhea and reduced cough effectiveness and offer individualized suggestions.

Healio spoke with Khan, the guideline’s lead author, to learn more about respiratory muscle weakness in this patient population, recommendations for those suffering/at risk and how clinicians can implement the guideline into their practice.

Healio: How prevalent is respiratory muscle weakness in patients with neuromuscular diseases? What challenges related to respiratory muscle weakness may these patients face daily?

Khan: The prevalence of respiratory muscle weakness varies depending on the specific disease, its severity and the stage of the disease. Some neuromuscular diseases associated with respiratory muscle weakness include muscular dystrophy, spinal muscular atrophy and amyotrophic lateral sclerosis, or ALS. Most patients with these disorders will eventually have some progressive neuromuscular weakness as the disease progresses.

In patients with neuromuscular diseases, respiratory muscle weakness can cause difficulties in breathing, coughing and clearing the airways of secretions. This can result in chronic respiratory failure, recurrent infections and other complications that can significantly affect daily life.

Patients with respiratory muscle weakness also experience fatigue, reduced endurance and exercise intolerance. Additionally, these patients may face social and psychological challenges, such as anxiety, depression and social isolation, due to their dependence on medical equipment and limitations in their ability to perform daily activities. It is also a burden on their families.

Healio: What prompted the creation of this clinical guideline?

Khan: Besides the answers provided in the first question, no treatments are available for these progressive disorders. Therefore, the best we can do is provide symptomatic relief, reduce complications and improve their quality of life using a shared decision-making approach with patients and their families. We hoped to do that by providing improved guidance to treating clinicians.

Healio: How did you and your colleagues establish the 15 recommendations that are presented in the guideline?

Khan: Panel members are practicing clinicians with technical expertise in this area. Through a Delphi process, the panel members identified the issues faced by these patients in clinical practice to choose the 15 areas to discuss.

Healio: Some recommendations are described as strong and some are described as conditional. Could you briefly explain some of the strong recommendations, as well as some of the conditional recommendations featured in the guideline?

Khan: The grade of recommendation, such as strong vs. conditional, is based on the GRADE (Grading of Recommendations, Assessment, Development and Evaluations) methodology. GRADE is an evidence-based approach to developing and presenting clinical practice guidelines. It is a framework that helps guideline developers assess evidence quality, make recommendations and communicate those recommendations to clinicians and patients. CHEST uses this established methodology. For example, we recommend using noninvasive ventilation for treatment for patients with neuromuscular diseases and chronic respiratory failure (strong recommendation, very low certainty of evidence). In this case, we provide both the strength of the recommendation and the level of evidence it is based on. Those interested in guideline methodology can read more about it on CHEST’s website.

Healio: The guideline also features a good practice statement that suggests pulmonary function testing (PFT) for these patients. Why is PFT the best tool when assessing these patients for respiratory complications?

Khan: Good-practice statements can be a valuable tool for clinicians and patients, as they can provide guidance on practices that are generally accepted as beneficial or practical. PFT or spirometry can be done very easily in outpatient clinics, it is relatively cheap, widely available and it can even be done at home by patients. This swayed the panel to recommend it, despite limited evidence.

Healio: How will everyday clinicians benefit from this guidance? How should they implement these recommendations into their practice?

Khan: Neuromuscular disorders are relatively rare, and an individual physician or other providers may not see many of these patients regularly. This guideline allows providers to assess their management plan against expert recommendations and best use available local resources. One of the things we have strongly recommended is shared decision-making with patients and their families.

For more information:

Akram Khan, MD, can be reached at khana@ohsu.edu.

References:

• CHEST releases clinical practice guideline on respiratory management of patients with neuromuscular weakness. https://www.chestnet.org/Newsroom/Press-Releases/2023/03/CHEST-releases-clinical-practice-guideline-on-respiratory-management-of-neuromuscular-weakness. Published March 14, 2023. Accessed March 15, 2023.
• Khan A, et al. CHEST. 2023;doi:10.1016/j.chest.2023.03.011.