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

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

This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.

Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, being injected subcutaneously every 3 weeks ,by the dose of 1-1.25 mg/kg. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.

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.