Patients
This retrospective study included patients with TDT accepted thalidomide in Department of Pediatrics, Sun Yat-sen Memorial Hospital between March 2018 and December 2019. They have been followed up at least for 6 months since the application of thalidomide. All patients were diagnosed according to the clinical characteristics and genetic test. Clinical data were obtained from patients’ medical records and the hospital electronic database. Those who did not have transfusion volume record before and during thalidomide treatment, or who did not have first month, third month visit were excluded from efficacy and safety analysis.
The application of thalidomide to the patients was approved by the off-label medicine committee. The retrospective study protocol was approved by the Ethics Committee of Sun Yat-sen Memorial Hospital and was in accordance with the 1964 Declaration of Helsinki and its later amendments. The clinical trial registration number is ChiCTR2000031436.