This real-world study will assess the efficacy and safety of luspatercept treatment for β-thalassemia major in Taiwan as well as the impact on quality of life.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Participants ≥18 years of age.
* Participants with the diagnosis of transfusion-dependent β-thalassemia major who are eligible to the treatment of luspatercept.
* Before luspatercept treatment, participant's transfusion burden ≥ 24 Red Blood Cell units in 24 weeks, without ≥35-day transfusion free.
Exclusion Criteria:
* Hypersensitivity to the active substance or to any of the excipients.
* Pregnancy.
* Participants requiring treatment to control the growth of extramedullary hematopoiesis (EMH) masses.
Primary outcome measure(s)
Number of Red Blood Cell (RBC) transfusion units received by participants — Baseline, every 3 weeks thereafter until week 96
Participant hemoglobin (Hb) level results — Baseline, every 3 weeks thereafter until week 96
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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