Authors’ contributions
Xinyu Li, Shuting Hu contributed equally to the manuscript. Jianpei Fang, Xinyu Li and Shuting Hu designed the study. Xinyu Li, Shuting Hu analyzed the data and wrote the manuscript. Yong Liu, Junjiu Huang, Lvhong Xu, Honggui Xu contributed to the retrieval and analysis of essential data. All authors contributed to the charts, critical revision and final approval of the manuscript.
Trial registration: www.chictr.org.cn. Identifier: ChiCTR2000031436.
What is already known about this subject: Thalidomide has been reported as a promising treatment for reducing transfusion volume in adults with β-thalassemia. For reducing blood transfusion volume, thalidomide is a potential applicable agent in children. However, the evidence about the safety and efficacy of thalidomide in children with TDT is scarce.
What this study adds: After six months treatment, thalidomide (2.5 to 3.6 mg·kg-1·d-1) is effective for 51/77 (66.2%), maintaining Hb over 90 g·L-1 without transfusion. Severe side effects are uncommon. HbF concentration of 47.298 g·L-1 at the third month is the predictor for further major responders.