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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
1-1.25mg/kg every 3 weeks subcutaneous injection
The second affiliated hospital of Sun Yat-sen University
Guangzhou, Guangdong, China
RECRUITINGA 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline
the incidence of 33% reduction in RBC transfusion
Time frame: 24 weeks
The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment
incidence of reported adverse events
Time frame: 24weeks
the incidence of 50% reduction in transfusion in any 12 consecutive weeks
the incidence of 50% reduction in transfusion
Time frame: 24 weeks
Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24
volume of transfusion
Time frame: 24 weeks
The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population
volume of transfusion
Time frame: 24 weeks
Changes in mean serum ferritin (SF) levels in the population from baseline
serum ferritin (SF) levels
Time frame: 24 weeks
Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)
MR T2\*
Time frame: 24 weeks
changes in reticulocyte levels from baseline during treatment
blood routine
Time frame: 24 weeks
changes in fetal hemoglobin levels from baseline during treatment
Hemoglobin electrophoresis
Time frame: 24weeks
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