Duchenne muscular dystrophy respiratory profiles from real world registry data

Abstract

IntroductionUnderstanding real‐world profiles from neuromuscular databases is helpful for optimizing clinical care and planning research studies. The Canadian Neuromuscular Disease Registry (CNDR) has respiratory data from a population of boys with Duchenne Muscular Dystrophy (DMD).ObjectivesTo describe cross‐sectional respiratory profiles from a national DMD real‐world dataset. To explore the relationship between forced vital capacity percent predicted (FVC%) and disease severity parameters: scoliosis, ambulation and ventilation status.MethodsDescriptive statistics summarized the respiratory profiles. The CNDR registry enrolls and collects DMD clinic data from 36 Canadian centers.ResultsThere were 414 participants enrolled. The age ranged from 2 to 36 years old. Pulmonary function test data were available for 323 participants. The use of ventilatory support was seen in a significant proportion (19.5%) of subjects by age 14–16 years and was used by the majority (69.2%) by age 20–22 years. FVC% declined at a rate of 3.19% per year with every 1‐year increase in age. FVC% declined annually by 2.47% in nonambulatory participants versus by 0.96% in ambulatory participants. FVC% did not significantly change over age with the presence of scoliosis or use of ventilatory technology.ConclusionsThe data from this large cohort are valuable for understanding real‐world patterns of clinical care and disease progression. There is a significant association between the loss of ambulation and the rate of FVC% decline. Further longitudinal studies are needed to better understand the impact of disease parameters on pulmonary function decline and the need for ventilatory support.

Real world respiratory profiles and Duchenne muscular dystrophy

“…If the CNDR control data had been used instead, it is unlikely that those studies would have found a positive respiratory effect. This illustrates what may be the most important implication of the article from Hnaini et al 11 -that the use of historical controls, including historical data derived from academic consortiums and marketed to industry, 24,25 is a potentially misleading way to study new drugs for DMD.…”