Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids

• Published in: Neuromuscular disorders : NMD Vol. 28; no. 11; pp. 897 - 909
• Main Authors: McDonald, Craig M., Gordish-Dressman, Heather, Henricson, Erik K., Duong, Tina, Joyce, Nanette C., Jhawar, Sanjay, Leinonen, Mika, Hsu, Fengming, Connolly, Anne M., Cnaan, Avital, Abresch, Richard T., Dubrovsky, A., Kornberg, A., Ryan, M., Webster, R., Biggar, W.D., McAdam, L.C., Mah, J.K., Kolski, H., Vishwanathan, V., Chidambaranathan, S., Nevo, Y., Gorni, K., Carlo, J., Tulinius, M., Lotze, T., Bertorini, T.E., Day, J.W., Karachunski, P., Clemens, P.R., Abdel-Hamid, H., Teasley, J., Kuntz, N., Driscoll, S., Bodensteiner, J.B., Connolly, A.M., Pestronk, A., Abresch, R.T., Henricson, E.K., Joyce, N.C., McDonald, C.M., Cnaan, A., Morgenroth, L.P., Leshner, R., Tesi-Rocha, C., Thangarajh, M., Duong, T.
• Format: Journal Article
• Language: English
• Published: England Elsevier B.V 01-11-2018
• Subjects: Duchenne; Mortality; Muscular dystrophy; Pulmonary; Steroids; Mortality; Muscular dystrophy; Pulmonary; Duchenne; Steroids
• ISSN: 0960-8966; 1873-2364
• DOI: 10.1016/j.nmd.2018.07.004

•Natural history changes in pulmonary function tests across time in Duchenne muscular dystrophy.•Treatment with glucocorticoids (steroids) > 1 year was compared to steroid naïve treatment.•Steroid treatment slowed the rate of pulmonary decline as measured by FVC%p in 7–9.9 year olds;•Steroid treatment resulted in higher peak absolute FVC and PEFr values with later onset of decline.•Time to FVC < 1 liter was delayed by steroid treatment; FVC < 1 liter increased risk of death 4-fold.
We describe changes in pulmonary function measures across time in Duchenne muscular dystrophy patients treated with glucocorticoids (GCs) > 1 year compared to GC naïve patients in the Cooperative International Research Group Duchenne Natural History Study, a multicenter prospective cohort study. 397 participants underwent 2799 pulmonary function assessments over a period up to 10 years. Fifty-three GC naïve participants (< 1 month exposure) were compared to 322 subjects with > 1 year cumulative GC treatment. Forced vital capacity (FVC), peak expiratory flow rate (PEFr), maximal inspiratory and expiratory pressures were performed and calculated as a percent predicted (%p). GC treatment slowed the rate of pulmonary decline as measured by FVC%p, in patients aged 7–9.9 years. GC treatment slowed 12 and 24-month progression of percent predicted spirometry to a greater degree in those with baseline FVC%p from < 80–34%. GC treatment resulted in higher peak absolute FVC and PEFr values with later onset of decline. Progression to an absolute FVC < 1 liter was delayed by GC treatment. Patients who reached a FVC below 1 L were 4.1 times more likely to die (p = 0.017). Long-term glucocorticoid treatment slows pulmonary disease progression in Duchenne dystrophy throughout the lifespan.