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

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, parathyroid 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 treatments (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.