Williams Kristen M, Lee Margaret T, Licursi Maureen, Brittenham Gary M, Fennoy Ilene
Division of Pediatric Endocrinology, Columbia University Medical Center.
Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Columbia University Medical Center, New York, NY.
J Pediatr Hematol Oncol. 2018 Aug;40(6):458-461. doi: 10.1097/MPH.0000000000001155.
Patients with sickle cell disease (SCD) are at risk for bone fragility from multiple factors including vitamin D deficiency. To date, no studies have evaluated the efficacy and safety of long-term vitamin D therapy for bone disease in children with SCD. We report a cohort of 4 children with SCD found to have severe vitamin D deficiency, secondary hyperparathyroidism, and abnormal bone mineral density treated with monthly high-dose oral cholecalciferol over 2 years. All patients exhibited a positive response to therapy without hypervitaminosis D or hypercalcemia. Further studies are needed to standardize guidelines for optimal vitamin D dosing and prevention of toxicity.
镰状细胞病（SCD）患者因多种因素（包括维生素D缺乏）而有骨质脆弱的风险。迄今为止，尚无研究评估长期维生素D治疗对SCD儿童骨病的疗效和安全性。我们报告了一组4名SCD儿童，他们被发现患有严重维生素D缺乏、继发性甲状旁腺功能亢进和骨矿物质密度异常，接受了为期2年的每月高剂量口服胆钙化醇治疗。所有患者对治疗均表现出阳性反应，且无维生素D过多症或高钙血症。需要进一步研究以规范最佳维生素D剂量指南和预防毒性。
