The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
Study Type
OBSERVATIONAL
Enrollment
200
According to the product label
Prince Muhammad bin Nasser Hospital
Jizan, Saudi Arabia
Change in transfusion burden
Time frame: Baseline and up to week 144
Change in mean pre-transfusion hemoglobin level
Time frame: Baseline and up to week 144
Change in transfusion-related visits
Time frame: Baseline and up to week 144
Proportion of participants achieving ≥33% reduction in red blood cell (RBC) transfusion burden (number of RBC units transfused) plus a reduction of ≥2 units
Time frame: Baseline and up to week 144
Proportion of participants achieving ≥50% reduction in red blood cell transfusion burden plus a reduction of ≥2 units
Time frame: Baseline and up to week 144
Time from first luspatercept dosing date to the first erythroid response
Time frame: Baseline and up to week 144
Time from the date the erythroid response is first observed until the last day of response
Time frame: Baseline and up to week 144
Participant pretransfusion hemoglobin level
Time frame: Baseline and up to week 144
Participant baseline transfusion burden
Time frame: Baseline
Proportion of participants without red blood cell transfusion during any consecutive 12-week or 24-week treatment period
Time frame: Baseline and up to week 144
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Time from first luspatercept dosing date to first onset of red blood cell-transfusion independence ≥12 weeks and ≥24 weeks
Time frame: Baseline and up to week 144
Time from the date a 12-week and 24-week red blood cell-transfusion independence is first observed until the date the participant has a subsequently documented red blood cell transfusion
Time frame: Baseline and up to week 144
Change in mean serum ferritin (SF) level
Time frame: Baseline and up to week 144
Change in proportion of participants with mean serum ferritin <1,000, 1000-2500, and >2,500 μg/L
Time frame: Baseline and up to week 144
Types (drug formulation - mono and combination therapy) of iron chelation therapy received
Time frame: Baseline and up to week 144
Change in mean daily dose of iron chelation therapy from baseline
Time frame: Baseline and up to week 144
Number of medical encounters (inpatient hospitalizations, emergency department attendances, hospital outpatient visits, visits at office-based physicians)
Time frame: Baseline and up to week 144
Inpatient length of stay
Time frame: Baseline and up to week 144
Proportion of participants remaining on luspatercept treatment
Time frame: Up to week 144
Length of time from initiation to discontinuation of luspatercept treatment
Time frame: Up to week 144
Frequencies of reasons for discontinuation of luspatercept treatment
Time frame: Up to week 144
Participant sociodemographics
Sociodemographics of participants such as age, sex, ethnicity, country of treatment, height, weight, and body mass index
Time frame: Baseline
Participant disease characteristics
Disease characteristics of participants, describing age of diagnosis of β-thalassemia, genotype (β0/β0, non-β0/β0), splenectomy status (yes/no), pretransfusion hemoglobin level (mean of all documented), transfusion burden (total number of red blood cell units transfused).
Time frame: Baseline
Participant comorbidities
Disease- and non-disease-related comorbid conditions
Time frame: Baseline
Concomitant treatment(s) received
Disease-related best supportive care (BSC) and treatments for other comorbidities.
Time frame: Baseline
Number of luspatercept doses administered
Time frame: Up to week 144
Number of luspatercept dose modifications
Time frame: Up to week 144
Frequencies of reasons for luspatercept dose modifications
Time frame: Up to week 144