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.
Study Type
OBSERVATIONAL
Enrollment
61
As per product label
Local Institution - 02
Kaohsiung City, Taiwan
Local Institution - 04
Tainan, Taiwan
Local Institution - 01
Taipei, Taiwan
Local Institution - 03
Taipei, Taiwan
Local Institution - 05
Number of Red Blood Cell (RBC) transfusion units received by participants
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant hemoglobin (Hb) level results
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant serum ferritin results
Time frame: Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96
Participant platelet level results
Time frame: Baseline, and every 3 weeks thereafter up to week 96
Participant white blood cell count results
Time frame: Baseline, and every 3 weeks thereafter up to week 96
Participant reticulocytes/normoblast count results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant Hemoglobin F (HbF) levels
Time frame: Baseline and weeks 6 and 12
Participant uric acid level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant lactate dehydrogenase (LDH) level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ratio
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Taoyuan District, Taiwan
Participant creatinine level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant bilirubin (total and direct) laboratory values
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Type of iron chelating therapy prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
Iron chelating therapy treatment dosage prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant Adverse Events (AEs)
Time frame: 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
Time frame: Baseline, week 12, 24, 48, 72 and 96
Participant health-related quality of life as assessed by EQ-5D-5L questionnaire
Time frame: Baseline, week 12, 24, 48, 72 and 96
Luspatercept treatment dosage prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
Duration of luspatercept treatment
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant reason(s) for luspatercept treatment discontinuation as assessed by the treating clinician
Time frame: Baseline, every 3 weeks thereafter until week 96
Participant demographics
Time frame: Baseline
Participant weight
Time frame: Baseline
Participant β0/β0 genotype status
Time frame: Baseline
Participant disease status
Time frame: Baseline
Participant comorbidities
Time frame: Baseline
Participant concomitant medication
Time frame: Baseline
Participant β-thalassemia treatment history
Time frame: Baseline
Participant surgical history (splenectomy and/or cholecystectomy)
Time frame: Baseline