The clinical trial compared REBLOZYL to placebo (no active medication)
- This trial included adults with β-thalassemia who needed regular red blood cell transfusions of 6 to 20 units every 24 weeks
The main goal of the study was to see if REBLOZYL could reduce transfusion burden by at least one-third (at least 2 red blood cell units) during a set 12-week period (weeks 13–24)
Patients in the clinical trial were divided into 2 groups:
REBLOZYL was continued for as long as a reduction in transfusions was observed or until unacceptable toxicity.
*Subcutaneous means under the skin.
Patients included in the study needed to:
- Receive regular red blood cell transfusions (6–20 red blood cell units per 24 weeks) with no transfusion-free period greater than 35 days during that period
- Be at least 18 years old
Patients could not be a part of the study if they had:
- Hemoglobin S/β-thalassemia or alpha (α)-thalassemia
- Major organ damage (liver disease, heart disease, lung disease, poor kidney function)
- Recent deep vein thrombosis or stroke, or recent use of erythropoiesis-stimulating agent, immunosuppressant, or hydroxyurea therapy
Characteristics of the 336 patients in the clinical trial