From the (1)Department of Endocrinology, National Health Commission Key Laboratory of Endocrinology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China, and the.
Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China.
Endocr Pract. 2020 Dec;26(12):1477-1485. doi: 10.4158/EP-2020-0073.
Duchenne muscular dystrophy (DMD) is a severe X-linked progressive neuromuscular disease that brings a significantly increased risk of osteoporosis and bone fractures. We prospectively evaluated the effects of oral and intravenous bisphosphonates on the bones of children with DMD.
This study included a total of 52 children with DMD. They were divided into zoledronic acid (ZOL), alendronate (ALN), and control groups according to bone mineral density (BMD) and history of fragility fractures. For 2 years, all patients took calcium, vitamin D, and calcitriol. Meanwhile, 17 patients received infusions of ZOL, and 18 patients received ALN. BMD, serum levels of alkaline phosphatase (ALP) and the cross-linked C-telopeptide of type I collagen (β-CTX) were evaluated.
After 24 months of treatment, the percentage changes in lumbar spine BMD were 23.2 ± 9.7% and 23.6 ± 8.8% in the ZOL and ALN groups (all P<.01 vs. baseline). The increases did not differ between the ZOL and ALN groups, but were significantly larger than those of the control group (P<.01). Serum β-CTX and ALP levels, respectively, were decreased by 44.4 ± 18.0% and 31.9 ± 26.7% in the ZOL group and by 36.0 ± 20.3% and 25.8 ± 14.4% in the ALN group (all P<.01 vs. baseline).
Zoledronic acid and alendronate had similar protective effects to increase bone mineral density and reduce bone resorption in children with DMD, which were superior to treatment of calcium, vitamin D, and calcitriol.
25OHD = 25 hydroxyvitamin D; ALN = alendro-nate; ALP = alkaline phosphatase; ALT = alanine aminotransferase; BMD = bone mineral density; BP = bisphosphonate; Ca = calcium; β-CTX = cross-linked C-telopeptide of type I collagen; DMD = Duchenne muscular dystrophy; FN = femoral neck; GC = glucocorticoid; LS = lumbar spine; ZOL = zoledronic acid.
杜氏肌营养不良症(DMD)是一种严重的 X 连锁进行性神经肌肉疾病,会显著增加骨质疏松症和骨折的风险。我们前瞻性评估了口服和静脉用双膦酸盐对 DMD 患儿骨骼的影响。
本研究共纳入 52 例 DMD 患儿。根据骨密度(BMD)和脆性骨折史,将他们分为唑来膦酸(ZOL)、阿仑膦酸钠(ALN)和对照组。2 年内,所有患者均服用钙、维生素 D 和骨化三醇。同时,17 例患者接受 ZOL 输注,18 例患者接受 ALN 治疗。评估 BMD、血清碱性磷酸酶(ALP)和Ⅰ型胶原交联 C 端肽(β-CTX)水平。
治疗 24 个月后,ZOL 和 ALN 组腰椎 BMD 百分比变化分别为 23.2%±9.7%和 23.6%±8.8%(均 P<.01 与基线相比)。ZOL 和 ALN 组之间的增加没有差异,但明显大于对照组(P<.01)。ZOL 组血清β-CTX 和 ALP 水平分别下降 44.4%±18.0%和 31.9%±26.7%,ALN 组分别下降 36.0%±20.3%和 25.8%±14.4%(均 P<.01 与基线相比)。
唑来膦酸和阿仑膦酸钠对增加 DMD 患儿的骨密度和减少骨吸收具有相似的保护作用,优于钙、维生素 D 和骨化三醇治疗。