Alman Benjamin A, Raza S Naweed, Biggar W Douglas
Department of Surgery and Program in Developmental Biology, The Hospital for Sick Children, 555 University Avenue, Toronto, ON M5G 1X8, Canada.
J Bone Joint Surg Am. 2004 Mar;86(3):519-24. doi: 10.2106/00004623-200403000-00009.
Scoliosis due to progressive muscle weakness occurs in almost all males with Duchenne muscular dystrophy, and it progresses relentlessly. Previous studies have shown that corticosteroid treatment slows the decline in muscle strength and stabilizes muscle strength in patients with this disease. We hypothesized that steroids may also attenuate the development of scoliosis. The purpose of this study was to compare the prevalence of scoliosis in male patients with Duchenne muscular dystrophy who received steroids with a control group of such patients who did not.
A group of seven to ten-year-old boys with Duchenne muscular dystrophy who were able to walk were enrolled in a nonrandomized comparative study to determine the effect of deflazacort (a derivative of prednisone) on muscle strength and pulmonary function. Thirty patients were treated with deflazacort (treatment group), and twenty-four were not (control group). The patients were matched for age and pulmonary function at baseline. To assess the development of scoliosis, the patients in each group were followed for at least five years. Survival curves were plotted to determine the chance of scoliosis of >/==" BORDER="0">20 degrees developing. The difference between the groups with respect to the chance of scoliosis developing was determined with Kaplan-Meier analysis.
A curve of >/==" BORDER="0">20 degrees developed during the follow-up period in sixteen (67%) of the twenty-four patients in the control group but in only five (17%) of the thirty patients in the treatment group. Fifteen of the twenty-four patients in the control group underwent spine surgery, at a mean age of thirteen years, whereas only five of the thirty patients in the treatment group underwent spine surgery, at a mean age of fifteen years. Kaplan-Meier analysis demonstrated a significant difference between the two groups with regard to development of scoliosis of >/==" BORDER="0">20 degrees (p < 0.001). Cataracts developed in ten patients in the treatment group, and stress fractures developed in three patients in the treatment group. Patients in the treatment group weighed a mean of 3.7 kg more than did those in the control group.
Steroid treatment slows the progression of scoliosis in males with Duchenne muscular dystrophy; however, longer-term evaluation will be necessary to determine if the treatment prevents the development of scoliosis or just delays its onset. At the very least, steroid treatment delays the need for spinal surgery.
Therapeutic study, Level II-1 (prospective cohort study). See Instructions to Authors for a complete description of levels of evidence.
进行性肌无力导致的脊柱侧弯几乎见于所有杜氏肌营养不良症男性患者，且病情持续进展。既往研究表明，皮质类固醇治疗可减缓此类疾病患者肌肉力量的下降并稳定其肌肉力量。我们推测类固醇可能还会减轻脊柱侧弯的发展。本研究的目的是比较接受类固醇治疗的杜氏肌营养不良症男性患者与未接受治疗的对照组患者脊柱侧弯的患病率。
一组7至10岁能行走的杜氏肌营养不良症男孩被纳入一项非随机对照研究，以确定地夫可特（泼尼松的一种衍生物）对肌肉力量和肺功能的影响。30例患者接受地夫可特治疗（治疗组），24例未接受治疗（对照组）。患者在基线时按年龄和肺功能进行匹配。为评估脊柱侧弯的发展，每组患者至少随访5年。绘制生存曲线以确定发生≥20度脊柱侧弯的几率。采用Kaplan-Meier分析确定两组在脊柱侧弯发生几率方面的差异。
对照组24例患者中有16例（67%）在随访期间出现了≥20度的脊柱侧弯曲线，而治疗组30例患者中只有5例（17%）出现。对照组24例患者中有15例在平均13岁时接受了脊柱手术，而治疗组30例患者中只有5例在平均15岁时接受了脊柱手术。Kaplan-Meier分析表明，两组在发生≥20度脊柱侧弯方面存在显著差异（p<0.001）。治疗组有10例患者出现白内障，3例患者出现应力性骨折。治疗组患者的平均体重比对照组患者重3.7千克。
类固醇治疗可减缓杜氏肌营养不良症男性患者脊柱侧弯的进展；然而，需要进行长期评估以确定该治疗是预防了脊柱侧弯的发生还是仅仅延迟了其发病。至少，类固醇治疗延迟了脊柱手术的需求。
治疗性研究，II-1级（前瞻性队列研究）。有关证据水平的完整描述，请参阅《作者须知》。
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