Feasibility of lung function testing & results in children with spinal muscular atrophy type II & III

Abstract

Introduction/Aim: Respiratory function testing is important for children with spinal muscular atrophy (SMA) as a way of monitoring disease progression and timing of interventions. However, it is unclear which tests are most appropriate for this patient group. Our objectives were (1) to assess feasibility of a variety of lung function tests in children with SMA type II and III, and (2) establish baseline data in this unique cohort for future clinical research. Method: Subjects (n=18) were prospectively recruited when they had a respiratory outpatient appointment or overnight sleep study. Forced oscillation technique (FOT), Lung clearance index (LCI) via Multiple breath washout, spirometry, peak cough flow (PCF), sniff nasal inspiratory pressure (SNIP), maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) were attempted on all subjects. Results: Subjects able to perform and achieve results with PCF, MIP and MEP were comparatively lower so data was excluded from analysis. Table highlights the difference in the number of patients able to adequately perform each test and subsequently achieve acceptable results. Median of all results reported (IQR). (Table presented) Conclusion: Establishing which tests are more appropriate for children with SMA will help to improve the quality of lung function results and better optimise patient management. While MIP, MEP and PCF may have clinical relevance, subjects ability to perform these tests were more variable compared with reported results. This suggests that these tests may not be useful for monitoring patient progress.L6220915172018-05-16 <br />