Abstract
Background Thalidomide has been reported as a promising
treatment for reducing transfusion volume in adults with β-thalassemia.
However, the evidence about the safety and efficacy of thalidomide on
children with transfusion dependent β-thalassemia (TDT) is scarce.
Methods Seventy-seven children with TDT treated with
thalidomide at least for 6 months were included and retrospectively
analyzed. Oral dose was started at 2.5 mg·kg-1·d-1. Blood volume for
maintenance of hemoglobin above 90 g·L-1 compared with pre-treatment
volume is the evaluation index for response.
Results After the sixth month treatment, 51/77 (66.2%)
maintained Hb over 90 g·L-1 without transfusion. Adverse events were
reported in 48 (63.2%) patients. Age, sex, genotype category, dosage
and transfusion interval before thalidomide treatment were not
correlated to treatment response. The AUC was 0.806 for the HbF at the
third month of treatment in predicting probability of major responders
at the sixth month treatment. Based on Youden’s index algorithm in the
ROC curve, 47.298 g·L-1 was the optimal cut-off value of the HbF at the
third month of treatment in predicting major responders at the sixth
month treatment, with sensitivity of 67.5% and specificity of 93.3%.
Conclusions The dose of thalidomide between 2.5 mg·kg-1·d-1to
3.6 mg·kg-1·d-1 is effective in TDT children. Severe side effects are
uncommon. HbF concentration of 47.298 g·L-1 at the third month is
recommended as the predictor for further major responders.