Treatment of vitamin D deficiency in transfusion-dependent thalassemia

The survival of patients with thalassemia major has progressively improved with advances in therapy; however, osteoporosis remains a frequent, unresolved issue [1]. Adequate circulating levels of vitamin D are essential for optimal skeletal health and reducing fracture risk [2]. Vitamin D insufficiency is reported in the majority of patients with thalassemia in the USA [3] and elsewhere [4-10], despite routine prescription of 400-1,000 IU vitamin D per day. In this study, assessment of serum 25-hydroxy vitamin D (25-OH D) levels in 96 patients with thalassemia revealed that 70 (73%) were either deficient (<20 ng/ml, 43%) or insufficient (20-29 ng/ml, 30%). Significantly more transfusion-independent patients were deficient compared with the transfusion-dependent group (60% versus 33%, P = 0.014). Supervised administration of high-dose (50,000 IU) oral vitamin D-2 every 3 weeks during transfusion visits in 32 transfusion-dependent patients increased the 25-OH D level from 18.4 to 24.2 ng/ml (P < 0.001) over a 4-month period. Each dose of vitamin D-2, given at 3-week intervals, increased 25-OH D levels by 1.4 +/- 2.0 ng/ml. These results show that vitamin D deficiency remains widespread despite daily low-dose supplementation. Supervised high-dose oral vitamin D supplementation is a safe and noninvasive method for predictable improvement of vitamin D status in thalassemia.