Respiratory decline in adult patients with Becker muscular dystrophy: A longitudinal study.
Becker muscular dystrophy (BMD) is a rare hereditary neuromuscular disease, caused by a genetic defect in the Duchenne Muscular Dystrophy (DMD) gene. We studied the natural history of respiratory function and its affecting factors in 23 adult BMD patients. These important data are needed for (future) clinical trials in BMD but are largely lacking. Patients had a median age of 51 years (28-78y) and median follow-up duration of 14 years (2-25y). We analysed 190 pulmonary function measurements with a median interval of one year (1-17y) and measured a 1.00% decline of Forced Vital Capacity percent predicted (FVC%pred) per year (p = 0.004). Loss of ambulation significantly increased the annual rate of FVC decline and was dependent of patient's body mass index (BMI; p = 0.015), with increases in BMI correlating with an even more rapid deterioration of FVC. A decline in Medical Research Council (MRC) sum score was significantly correlated with a decline in FVC (p = 0.003). We conclude that adult BMD patients experience a significant but mild respiratory decline. However, this decline is significantly more rapid and clinically relevant after loss of ambulation, which warrants a more vigilant follow-up of respiratory function in this subgroup.
['De Wel|Bram|B|', 'Willaert|Sofie|S|', 'Nadaj-Pakleza|Aleksandra|A|', 'Aubé-Nathier|Anne-Catherine|AC|', 'Testelmans|Dries|D|', 'Buyse|Bertien|B|', 'Claeys|Kristl G|KG|']
D000328:Adult D000368:Aged D015992:Body Mass Index D005260:Female D006801:Humans D008137:Longitudinal Studies D008168:Lung D008297:Male D008875:Middle Aged D020388:Muscular Dystrophy, Duchenne D012119:Respiration D014797:Vital Capacity D016138:Walking
BMD Natural history FVC BMI DMD-gene spirometry