Effects of stem cell transplantation on bone mineral density and vitamin D status in children with thalassemia major

HSCT is a curative treatment in TM, but conditioning and immunosuppressive treatment may affect bone metabolism. In this retrospective study, we aimed to compare BMD, vitamin D status, and growth in children with TM who underwent HSCT to those in children with TD TM. Twenty‐three children with TM who underwent HSCT (mean age 7.1 years [1.03‐14.7]) and 24 children with TD thalassemia (mean age 9.8 years [1.6‐14]) were recruited. Lumbar spine BMD of TD thalassemia patients was higher than those in patients who had HSCT at both baseline and second‐year assessments (P=.009, P<.001, respectively). However, BMD Z scores or serum 25‐OH vitamin D levels were not different in two groups. Being >10 years of age was a significant risk factor for low BMD, height, and weight Z score for both groups. Patients who underwent HSCT with Pesaro risk class II or III had higher risk for low BMD compared to those risk class I patients (P=.044). In conclusion, children with TM who were >10 years at HSCT are at risk for low BMD and growth retardation. HSCT had no effect on BMD deficit in children with TM.