Drug may be new option for transfusion-dependent β-thalassemia

Grade 3 or higher TEAEs occurred in 29.1% of patients in the luspatercept arm and 15.6% of those in the placebo arm. Serious TEAEs occurred in 15.2% and 5.5%, respectively.

One patient in the placebo arm had a TEAE-related death (acute cholecystitis), but there were no treatment-related deaths in the luspatercept arm.

TEAEs leading to treatment discontinuation occurred in 5.4% of luspatercept-treated patients and 0.9% of placebo-treated patients.

TEAEs that occurred more frequently in the luspatercept arm than in the placebo arm (respectively) included bone pain (19.7% and 8.3%), arthralgia (19.3% and 11.9%), and dizziness (11.2% and 4.6%).

Grade 3/4 TEAEs (in the luspatercept and placebo arms, respectively) included anemia (3.1% and 0%), increased liver iron concentration (2.7% and 0.9%), hyperuricemia (2.7% and 0%), hypertension (1.8% and 0%), syncope (1.8% and 0%), back pain (1.3% and 0.9%), bone pain (1.3% and 0%), blood uric acid increase (1.3% and 0%), increased aspartate aminotransferase (1.3% and 0%), increased alanine aminotransferase (0.9% and 2.8%), and thromboembolic events (0.9% and 0%).

Dr. Cappellini noted that thromboembolic events occurred in eight luspatercept-treated patients and one placebo-treated patient. In all cases, the patients had multiple risk factors for thrombosis.

This study was sponsored by Celgene Corporation and Acceleron Pharma. Dr. Cappellini reported relationships with Novartis, Celgene, Sanofi-Genzyme, and Vifor.