Effect of cyclic pamidronate administration on osteoporosis in children with β-thalassemia major: a single-center study

Abstract

Background: Osteopenia and osteoporosis represent a prominent cause of morbidity in children with thalassemia. Multiple factors are responsible for the pathogenesis of bone loss in thalassemia, including diabetes, hypothyroidism, para-thyroid gland dysfunction, accelerated hemopoiesis, direct iron toxicity of osteoblasts, iron chelators, and deficiencies of growth hormone or insulin growth factors. Purpose: To assess the effect of pamidronate administration on β-thalassemia major-induced osteoporosis in children. Methods: This study assessed the effects of different treat-ments (calcium and vitamin D versus calcium, vitamin D, and pamidronate) on patients with β-thalassemia major and osteoporosis. Bone mineral density (BMD) and z scores were measured at baseline and after 1 year of treatment using dual-energy x-ray absorptiometry. Results: The mean baseline BMD values of the lumbar spine were 0.71±0.07 (g/cm²) and 0.74±0.07 (g/cm²), respectively, while those at the end of the study were 0.81±0.07 (g/cm²) (P<0.001) and 0.78±0.07 (g/cm²) (P>0.05), respectively. The mean baseline z scores of the lumbar spine were-3.53±0.55 and-3.17±0.61, while those after treatment were-2.1±0.32 (P=0.001) and-3.11±0.67 (P>0.05), respectively. The baseline alkaline phosphatase levels were 351.5±86.07 µg/dL and 357.6±89.7 µg/dL, while those after treatment were 220.4± 59.26.07 µg/dL (P<0.001) and 320.3±83.99 µg/dL (P>0.05), respectively. Conclusion: Pamidronate administration effectively increased the BMD and z scores of children with β-thalassemia major. Pamidronate had a favorable safety profile with no related serious adverse events during the study period.