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
We are an independent charity and are not backed by a large company or society. We raise every penny ourselves to improve the standards of cancer care through education. You can help us continue our work to address inequalities in cancer care by making a donation.
Any donation, however small, contributes directly towards the costs of creating and sharing free oncology education.
Together we can get better outcomes for patients by tackling global inequalities in access to the results of cancer research.
Thank you for your support.