Clinical patterns of thalidomide in the treatment of transfusion-dependent β-thalassaemia in children: a prospective single-arm study in China

Key Points

• Thalidomide treatment leads to significant reductions in transfusion volume for children with β-thalassaemia.
• Evidence from this study indicates potential long-term benefits and manageable side effects associated with thalidomide.
• This is a prospective single-arm study conducted to evaluate thalidomide's efficacy in managing TDT.
• Findings suggest that thalidomide may offer a viable treatment pathway for children needing long-term therapy.