From the IQVIA (E.L.), Stockholm, Sweden; Division of Endocrinology (K.P., L.M.W.), Department of Pediatrics, University of Ottawa, Children's Hospital of Eastern Ontario, Canada; Department of Women's and Children's Health (F.Z., E.Å., T.S.), Karolinska Institutet, Stockholm, Sweden; IQVIA (S.A.), London, United Kingdom; and Children's Hospital of Eastern Ontario Research Institute (H.L.); Division of Neurology, Department of Medicine, the Ottawa Hospital, Canada.
Neurology. 2024 Jan 23;102(2):e207948. doi: 10.1212/WNL.0000000000207948. Epub 2023 Dec 18.
Bisphosphonates are routinely used to treat osteoporosis in patients with Duchenne muscular dystrophy (DMD), a rare, severely debilitating neuromuscular disease. We sought to synthesize and grade benefits and harms evidence of bisphosphonates in glucocorticoid-treated patients with DMD.
In this systematic review (PROSPERO identifier: CRD42020157606), we searched MEDLINE, CINAHL, Embase, PsycINFO, Web of Science, and CENTRAL for articles published from inception up to and including March 31, 2023, reporting results in any language from any study type. Quality of evidence was assessed using the Grading of Recommendations, Assessment, Development, and Evaluations framework.
We identified 19 publications involving 1,010 children and adults from 12 countries across all inhabited continents except South America. We found high-quality evidence that bisphosphonates significantly increase the areal lumbar spine bone mineral density (BMD) Z score in glucocorticoid-treated patients with DMD. The greatest improvements were recorded in controlled settings among patients treated with intravenous zoledronate. Evidence of benefits to fracture risks was inconclusive and/or of low quality, primarily due to lack of controlled data and small samples. Bisphosphonates were generally well-tolerated, although adverse events related to the first infusion (i.e., "acute phase reaction") were frequently reported.
There is high-quality evidence supporting the use of bisphosphonates to increase the areal lumbar spine BMD Z score in patients with DMD and glucocorticoid-induced osteoporosis. Our synthesis and grading affirm current recommendations put forward in the 2018 DMD Clinical Care Considerations and should be helpful in raising awareness about anticipated benefits of bisphosphonates, prevailing unmet needs, and potential safety issues in their use.
双膦酸盐通常用于治疗杜兴氏肌营养不良症（DMD）患者的骨质疏松症，DMD 是一种罕见的严重神经肌肉疾病。我们旨在综合评估并分级双膦酸盐治疗 DMD 糖皮质激素治疗患者的获益和危害证据。
在这项系统评价（PROSPERO 标识符：CRD42020157606）中，我们检索了 MEDLINE、CINAHL、Embase、PsycINFO、Web of Science 和 CENTRAL，检索时间从建库至 2023 年 3 月 31 日，检索语种不限，纳入了所有研究类型的报告结果。使用推荐、评估、发展和评估（GRADE）框架评估证据质量。
我们共确定了 19 项出版物，这些出版物涉及来自除南美洲以外所有有人居住的大陆的 12 个国家的 1010 名儿童和成人。我们发现高质量证据表明，双膦酸盐可显著增加 DMD 糖皮质激素治疗患者的腰椎骨面积骨密度（BMD）Z 评分。在静脉注射唑来膦酸盐的患者中，在对照环境下记录到最大改善。骨折风险获益的证据不确定和/或质量较低，主要是由于缺乏对照数据和小样本量。双膦酸盐通常耐受良好，尽管首次输注相关的不良事件（即“急性期反应”）经常报告。
有高质量证据支持在 DMD 患者和糖皮质激素诱导的骨质疏松症中使用双膦酸盐来增加腰椎骨面积 BMD Z 评分。我们的综合评估和分级证实了 2018 年 DMD 临床护理考虑提出的当前建议，这应该有助于提高对双膦酸盐预期获益、当前未满足的需求以及其使用的潜在安全问题的认识。
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