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Impact of Bisphosphonates on Survival for Patients With Duchenne Muscular Dystrophy

2011; American Academy of Pediatrics; Volume: 127; Issue: 2 Linguagem: Inglês

10.1542/peds.2010-1666

1098-4275

Kevin Gordon, Joseph M. Dooley, Kayla M. Sheppard, Judith MacSween, Michael J. Esser,

Adipose Tissue and Metabolism

In this article we describe the association of bisphosphonate therapy on survival within a regional cohort of patients with Duchenne muscular dystrophy (DMD) who received steroid therapy and were managed in a single center.The records of all patients with confirmed DMD who were born between 1963 and 2006 and who had received at least 1 year of steroid therapy were reviewed from birth until they reached the study end points (death, loss to follow-up, or the last follow-up was in 2009). A survival analysis was used to account for the variable follow-up duration within this cohort.Forty-four boys from this cohort with DMD were exposed to continuous steroid use. Bisphosphonate therapy was initiated for 16 patients (36%) between 1997 and 2007 at a median age of 12.5 years (range: 7-23 years). At the time of the last follow-up in 2009, 13 patients had died (30%) at a median age of 16 years (range: 14-27 years). Survival curves demonstrate that the prescription of bisphosphonates was associated with a significant improvement in survival rate (P = .005, log-rank test). Furthermore, a possible therapy-duration effect could be shown for bisphosphonate use (P = .007, log-rank test).The treatment of patients with DMD with steroids and bisphosphonates seems to be associated with significantly improved survival compared with treatment with steroids alone.