Thalassemia Major
Conditions
Brief summary
An observational study to evaluate long-term safety and efficacy of luspatercept in subjects with transfusion dependent who received the first dose of luspatercept after its introduction in the clinical practice
Detailed description
Luspatercept represents the first and only erythroid maturation agent (EMA) approved by the European Commission (EC) and the Food and Drug Administration (FDA) capable of enhancing advanced erythrocyte maturation. The efficacy of luspatercept was demonstrated in the phase III clinical trial called BELIEVE. With market authorization, the drug is used in less selected patients with potentially more complex medical histories than those enrolled in the registration study. In addition, it is now also possible to prescribe the drug in patients with blood consumption at baseline between 20 and 24 blood units in the 24 weeks before the drug was prescribed, who could not be included in the BELIEVE study. Gathering data on the clinical characteristics of patients who are prescribed luspatercept in normal clinical practice, and of safety and efficacy in this setting, is essential to enrich the available information on this new therapy.
Interventions
Luspatercept (ACE-536)
Sponsors
Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Subjects who received at least one dose of luspatercept after its introduction in the clinical practice
Exclusion criteria
* Subjects who received luspatercept after participating in the compassionate use program * Subjects unwilling to sign informed consent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | 52 WEEK | \- To evaluate safety of luspatercept in subjects with transfusion-dependent beta thalassemia who received the first dose of luspatercept after its introduction in the clinical practice |
| Tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | 52 WEEK | \- To evaluate tolerability of luspatercept in subjects with transfusion-dependent beta thalassemia who received the first dose of luspatercept after its introduction in the clinical practice |
| Efficacy of luspatercept - Number of subjects who showed a ≥ 33% reduction from baseline in the number of transfused blood units during any 12-week interval period of luspatercept treatment | 12 weeks | \- To evaluate efficacy of luspatercept in subjects with transfusion-dependent beta thalassemia who received the first dose of luspatercept after its introduction in the clinical practice |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| impact of luspatercept on iron accumulation cardiac T2* | 52 weeks | \- To increase available data on the impact of luspatercept on iron accumulation by assessing the change in serum ferritin and cardiac T2\* value on nuclear magnetic resonance imaging |
| impact of luspatercept on iron accumulation hepatic T2* | 52 weeks | \- To increase available data on the impact of luspatercept on iron accumulation by assessing the change in serum ferritin and hepatic T2\* value on nuclear magnetic resonance imaging |
| incidence of endocrinological changes | 52 weeks | \- To evaluate the incidence of endocrinological changes in patients treated with luspatercept |
| safety of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | 52 weeks | \- To evaluate safety of luspatercept in subjects with transfusion-dependent beta thalassemia with baseline transfusion needs and comorbidities that were exclusion criteria in formal clinical trials |
| positively affect drug response. | 52 weeks | \- To identify the factors that positively affect drug response. |
| negatively affect drug response. | 52 weeks | \- To identify the factors that negatively affect drug response. |
| incidence of thromboembolic events | 52 weeks | \- To assess the incidence of thromboembolic events in this category of patients |
| tolerability of luspatercept - Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | 52 weeks | \- To evaluate tolerability of luspatercept in subjects with transfusion-dependent beta thalassemia with baseline transfusion needs and comorbidities that were exclusion criteria in formal clinical trials |
| Efficacy of luspatercept - Number of subjects who showed a ≥ 33% reduction from baseline in the number of transfused blood units during any 12-week interval period of luspatercept treatment | 52 weeks | \- To evaluate efficacy of luspatercept in subjects with transfusion-dependent beta thalassemia with baseline transfusion needs and comorbidities that were exclusion criteria in formal clinical trials |
Countries
Italy
Outcome results
None listed