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Published on: March 14, 2017
Response to Long-term Vitamin D Therapy for Bone Disease in Children With Sickle Cell Disease
Kristen M Williams1, Margaret T Lee2, Maureen Licursi2
1Division of Pediatric Endocrinology, Columbia University Medical Center.
Insights
Long-term vitamin D therapy improved bone health in children with sickle cell disease (SCD) who had severe vitamin D deficiency. The treatment was safe and effective over two years, showing no signs of toxicity.
Area of Science:
- Pediatrics
- Hematology
- Endocrinology
Background:
- Sickle cell disease (SCD) is associated with bone fragility.
- Vitamin D deficiency is a common contributing factor to bone disease in SCD patients.
- Optimal long-term vitamin D therapy for pediatric SCD bone disease remains unevaluated.
Purpose of the Study:
- To evaluate the efficacy and safety of long-term vitamin D therapy in children with SCD.
- To assess the impact of high-dose oral cholecalciferol on bone mineral density and related parameters.
Main Methods:
- A cohort of 4 children with SCD and severe vitamin D deficiency was treated.
- Monthly high-dose oral cholecalciferol was administered for 2 years.
- Bone mineral density, vitamin D levels, and markers of hyperparathyroidism were monitored.
Main Results:
- All patients showed a positive response to vitamin D therapy.
- Bone mineral density and related parameters improved with treatment.
- No cases of hypervitaminosis D or hypercalcemia were observed during the study period.
Conclusions:
- Long-term, high-dose vitamin D therapy appears safe and effective for managing bone disease in children with SCD.
- Further research is necessary to establish standardized guidelines for vitamin D dosing and toxicity prevention in this population.
Abstract:
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.
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