Luspatercept considerations1-6
| . | Strengths . | Limitations . |
|---|---|---|
| Subcutaneous administration | Does not require intravenous access | Requires infusion center visit every 3 weeks |
| Reduction of transfusion in TDT | 21.4% had >33% reduction 7.6% had >50% reduction | Most patients without significant reduction Majority still have visits every 3 weeks |
| Increase in hemoglobin in NTDT | 52.1% had 1.5-g/dL increase | Many patients without significant increase Requires increased appointment burden Not yet approved by regulatory agencies |
| Risk of thromboembolism | Rates low: 8 in 223 (3.6%) in trial | Patients with thalassemia already at increased risk of thromboembolism, especially if prior splenectomy |
| Impact on iron loading | Preliminary data showed lower ferritin and LIC in some patients, including some who did not meet primary end point | Additional data needed to verify findings |
| . | Strengths . | Limitations . |
|---|---|---|
| Subcutaneous administration | Does not require intravenous access | Requires infusion center visit every 3 weeks |
| Reduction of transfusion in TDT | 21.4% had >33% reduction 7.6% had >50% reduction | Most patients without significant reduction Majority still have visits every 3 weeks |
| Increase in hemoglobin in NTDT | 52.1% had 1.5-g/dL increase | Many patients without significant increase Requires increased appointment burden Not yet approved by regulatory agencies |
| Risk of thromboembolism | Rates low: 8 in 223 (3.6%) in trial | Patients with thalassemia already at increased risk of thromboembolism, especially if prior splenectomy |
| Impact on iron loading | Preliminary data showed lower ferritin and LIC in some patients, including some who did not meet primary end point | Additional data needed to verify findings |