New possible treatment options for thalassemia patients have been presented by Dr Olivier Hermine (Hôpital Necker-Enfants Malades, Paris, France).

β-thalassemias are characterised by ineffective red blood cell (RBC) production, leading to anaemia, iron overload, and organ failure.

As current treatment options for β-thalassemia are limited, there is a clear unmet need for alternative therapies.

It has been shown previously 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 haemoglobin 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 favourable safety profile, and is also undergoing phase 2 trials for treatment of anaemia in myelodysplastic syndromes, diamond blackfan anaemia, chronic myelomonocytic leukaemia, myelofibrosis, and end-stage renal disease.

Source: EHA