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.