β-Thalassemia Major
Conditions
Keywords
β-Thalassemia major
Brief summary
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.
Interventions
DRUGLuspatercept
As per product label
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Participant serum ferritin results | Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96 |
| 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 |
Secondary
| Measure | Time frame |
|---|---|
| Participant creatinine level results | Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96 |
| Participant bilirubin (total and direct) laboratory values | Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96 |
| Type of iron chelating therapy prescribed to participants | Baseline, every 3 weeks thereafter until week 96 |
| Participant platelet level results | Baseline, and every 3 weeks thereafter up to week 96 |
| Participant white blood cell count results | Baseline, and every 3 weeks thereafter up to week 96 |
| Participant reticulocytes/normoblast count results | Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96 |
| Participant Hemoglobin F (HbF) levels | Baseline and weeks 6 and 12 |
| Iron chelating therapy treatment dosage prescribed to participants | Baseline, every 3 weeks thereafter until week 96 |
| Participant Adverse Events (AEs) | Baseline and every 3 weeks until week 12, then weeks 24, 36, 48, 60, 72, 84 and 96 |
| Participant health-related quality of life as assessed by Transfusion-dependent Quality of Life (TranQoL) questionnaire | Baseline, week 12, 24, 48, 72 and 96 |
| Participant uric acid level results | Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96 |
| Luspatercept treatment dosage prescribed to participants | Baseline, every 3 weeks thereafter until week 96 |
| Duration of luspatercept treatment | Baseline, every 3 weeks thereafter until week 96 |
| Participant reason(s) for luspatercept treatment discontinuation as assessed by the treating clinician | Baseline, every 3 weeks thereafter until week 96 |
| Participant demographics | Baseline |
| Participant weight | Baseline |
| Participant β0/β0 genotype status | Baseline |
| Participant disease status | Baseline |
| Participant comorbidities | Baseline |
| Participant concomitant medication | Baseline |
| Participant β-thalassemia treatment history | Baseline |
| Participant surgical history (splenectomy and/or cholecystectomy) | Baseline |
| Participant health-related quality of life as assessed by EQ-5D-5L questionnaire | Baseline, week 12, 24, 48, 72 and 96 |
| Participant lactate dehydrogenase (LDH) level results | Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96 |
| Participant aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ratio | Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96 |
Countries
Taiwan
Outcome results
None listed