Gdf -11 a New Target to Improve Anemia in Thalassemia

MILAN, June 13, 2014 /PRNewswire/ --

New possible treatment options for thalassemia patients will be presented by Dr Olivier Hermine at the 19^th Congress of EHA. 

β-thalassemias are characterized by ineffective red blood cell (RBC) production, leading to anemia, iron overload, and organ failure. As current treatment options for β-thalassemia are limited, there is a clear unmet need for alternative therapies. We have previously shown that in animal models that GDF-11, a cytokine, is produced in large quantities and participates in the decreased production of red cells.  In this study, 32 adult patients with β-thalassemia received 0.1 (n = 8), 0.3 (n = 9), 0.5 (n = 8), or 0.75 (n = 7) mg/kg sotatercept(a fusion protein that inhibits GDF-11) subcutaneously once every 3 weeks.

Three (9%) patients reported grade ≥ 2 treatment-related adverse events: 2 (25%) in the 0.1 mg/kg dose cohort (bone pain and superficial thrombophlebitis), and 1 (13%) in the 0.5 mg/kg dose cohort (ventricular extra systoles).

Among patients with non-transfusion-dependent β-thalassemia (n = 22), a higher proportion of patients achieved a maximum hemoglobin increase ≥ 1 g/dL in the 0.3 (67%), 0.5 (83%), and 0.75 (100%) mg/kg dose cohorts versus the 0.1 mg/kg dose cohort (0%).

Furthermore, among patients with RBC transfusion-dependent β-thalassemia (n = 10), a higher proportion of patients achieved a transfusion burden reduction ≥ 20% in the 0.3 (33%), 0.5 (50%), and 0.75 (67%) mg/kg dose cohorts versus the 0.1 mg/kg dose cohort (0%).

Sotatercept may be of benefit to β-thalassemia patients with a favorable safety profile, and is also undergoing phase 2 trials for treatment of anemia in myelodysplastic syndromes, diamond blackfan anemia, chronic myelomonocytic leukemia, myelofibrosis, and end-stage renal disease.

Presenter: Dr Olivier Hermine

Affiliation: Department of Hematology, Imagine institute, INSERM U 1163 CNRS ERL 8564, Labex Red Cells, Hôpital Necker-Enfants Malades, Paris, France

Topic: Gdf -11 a new target to improve anemia in thalassemia.

Abstract S662 will be presented by Dr John Porter on Saturday June 14, 2014, 08:00 - 09:15 at Room Brown 3 (SW - Level 2)

About the EHA Annual Congress 

Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and developments will be presented. The topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders.