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

Sun Yat-sen University

Status and phase

Enrolling

Phase 4

Conditions

Transfusion-dependent Anemia

Thalassemia Major

Treatments

Drug: Luspatercept Injectable Product

Study type

Interventional

Funder types

Other

Identifiers

NCT05462548

SYSKY-2022-088-02

Details and patient eligibility

About

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.

Full description

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.

Enrollment

20 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.