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.