Department of Pediatrics, Dalhousie University, Halifax, Nova Scotia, Canada.
Pediatrics. 2011 Feb;127(2):e353-8. doi: 10.1542/peds.2010-1666. Epub 2011 Jan 17.
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.
本文描述了在接受类固醇治疗并在单一中心接受管理的杜兴氏肌营养不良症（DMD）患者的区域队列中，双膦酸盐治疗与生存的相关性。
对所有 1963 年至 2006 年出生、至少接受 1 年类固醇治疗的确诊 DMD 患者的记录进行了回顾性研究，随访至研究终点（死亡、失访或最后一次随访时间为 2009 年）。在该队列中，生存分析用于考虑随访时间的变化。
该 DMD 队列中的 44 名男孩持续接受类固醇治疗。1997 年至 2007 年间，16 名患者（36%）开始接受双膦酸盐治疗，中位年龄为 12.5 岁（范围：7-23 岁）。在 2009 年最后一次随访时，13 名患者（30%）死亡，中位年龄为 16 岁（范围：14-27 岁）。生存曲线表明，双膦酸盐的处方与生存率的显著提高相关（P =.005，对数秩检验）。此外，双膦酸盐使用可能存在治疗持续时间的影响（P =.007，对数秩检验）。
与单独使用类固醇治疗相比，用类固醇和双膦酸盐治疗 DMD 患者似乎与生存率的显著提高相关。
