851 Luspatercept Increases Hemoglobin, Decreases Transfusion Burden and Improves Iron Overload in Adults with Beta-Thalassemia Thalassemia and Globin Gene Regulation Program: Oral and Poster Abstracts Type: Oral Session: 112. Thalassemia and Globin Gene Regulation: Clinical Advances in Thalassemia Monday, December 5, 2016: 3:45 PM Room 7AB (San Diego Convention Center)
Antonio G. Piga, MD1, Immacolata Tartaglione, MD2*, Rita Gamberini, MD3*, Ersi Voskaridou, MD4*, Angela Melpignano, MD5*, Paolo Ricchi, MD6*, Vincenzo Caruso, MD7*, Antonello Pietrangelo, MD8*, Xiaosha Zhang9*, Dawn M. Wilson9*, Ashley Leneus9*, Abderrahmane Laadem, MD10, Matthew L. Sherman, MD9 and Kenneth M. Attie, MD9 Turin University, Turin, Italy
1
Second University of Naples, Naples, Italy
2
Arcispedale S. Anna, Cona, Ferrara, Italy
3
Laiko General Hospital, Athens, Greece
4
Ospedale "A. Perrino", Brindisi, Italy
5
AORN "A. Cardarelli", Naples, Italy
6
ARNAS Garibaldi, Catania, Italy
7
CEMEF, Medicina 2, Modena, Italy
8
Acceleron Pharma, Cambridge, MA
9
Celgene Corporation, Summit, NJ
10
Background.Luspatercept (ACE-536) is a modified activin receptor type IIB fusion protein that promotes late-stage erythroid differentiation. In beta-thalassemia, imbalanced production of alpha and beta globin chains in erythroid precursors causes ineffective erythropoiesis (IE) leading to anemia and dysregulated iron homeostasis. In a mouse model of beta-thalassemia, luspatercept corrected the effects of ineffective erythropoiesis and in a phase 1 clinical study with healthy volunteers, was well tolerated and increased hemoglobin (Suragani R, Nat Med, 2014; Suragani R, Blood, 2014; Attie K, Am J Hematol, 2014).
Aims.This is an ongoing, phase 2, multicenter, open-label, dose-finding study followed by a longterm extension study to evaluate the effects of luspatercept in patients (pts) with either transfusiondependent (TD) or non-transfusion dependent (NTD) beta-thalassemia with the following key