The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study

Inclusion Criteria:

-≥18 years old

• The patient was clearly diagnosed as transfusion-dependent β -thalassemia, the blood transfusion period was ≤60 days, and the red blood cell infusion volume was not less than 3-12U in the 12 weeks before enrollment, which could provide the red blood cell infusion volume record in the 12 weeks before enrollment
• Patients who were treated with thalidomide before enrollment were required to stop the treatment for more than 4 weeks, and their hemoglobin decreased to below 90g/L. Blood transfusion was required, and records of blood transfusion within 12 weeks could be provided
• Voluntarily participate in the study and sign the informed consent;

Exclusion Criteria:

• (1) Pregnant or lactating women
• persons known to be allergic to Luspatercept and/or Luspatercept excipients for injection
• Severe liver dysfunction (liver enzyme (ALT or AST) ≥3 TIMES ULN)
• Severe renal impairment (eGFR < 30 ml/min/1.73m3 or patients with end-stage renal disease)
• Heart disease, heart failure classified as Class 3 or higher by the New York Heart Association (NYHA), or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization.
• The patient has uncontrolled hypertension. According to NCI CTCAE version 5.0, controlled hypertension in this protocol is considered to be ≤1.
• Patients with a history of deep vein thrombosis or stroke within 24 weeks prior to enrollment
• Erythropoietin, Luspatercept or hydroxyurea were used 8 weeks before enrollment
• Any other significant medical condition, laboratory abnormality, or mental illness that the Investigator deems inappropriate for inclusion.