Yazarlar : Lars Jorgensen
Yayın : DGNews : Presented at EHA
Pubmed Linki : http://www.docguide.com/novel-gdf-11-inhibitor-shows-efficacy-beta-thalassemia-related-anaemia-treatment?tsid=5
Konu : Talasemi
Literatür İçeriği : MILAN, Italy -- June 20, 2014 -- Sotatercept, a novel fusion protein that targets the inhibition of GDF-11, shows potential benefit in the treatment of anaemia in patients with beta thalassemia, for whom treatment options are lacking, according to research presented on June 14 at the 19th Congress of the European Hematology Association (EHA).
“By improving ineffective erythropoiesis, sotatercept may provide clinical benefit to patients with beta-thalassemia, thereby addressing a significant unmet need,” stated co-author Olivier Hermine, MD, PhD, Imagine Institute, Hopital Necker-Enfants Malades, Paris, France.
“Preliminary data suggest that sotatercept, given subcutaneously once every 3 weeks, increases serum haemoglobin, improving anaemia,” Dr. Hermine added. “A dose-dependent effect for sotatercept was demonstrated, supporting further study of the exposure-effect relationship of thalessemia.”
As of February 2014, Dr. Hermine and colleagues had treated 32 adults with thalassemia with 0.1 mg/kg (n = 8), 0.3 mg/kg (n = 9), 0.5 mg/kg (n = 8), or 0.75 mg/kg (n = 7) of the drug, subcutaneously, once every 3 weeks.
Patients who were not dependent on transfusions (n = 22) showed the highest increases in haemoglobin, with a maximum increase of 1 g/dL or greater in 67% of patients in the 0.3-dose group; 83% in the 0.5-dose group; and 100% in the 0.75-mg/kg dose cohort, compared with 0% in the 0.1 mg/kg dose cohort.
Among 10 patients who were dependent on red-blood-cell transfusions, 33% achieved a transfusion burden reduction of 20% or greater in the 0.3-dose group, 50% in the 0.5-group, and 67% in the 0.75-mg/kg dose cohorts versus 0% in the 0.1-mg/kg dose cohort.
Three patients reported grade 2 or greater treatment-related adverse events: 2 in the 0.1-mg/kg dose cohort (including bone pain and superficial thrombophlebitis), and 1 in the 0.5 mg/kg dose cohort (ventricular extra systoles).
“The safety profile is acceptable, however longer follow-up is needed to assess long-term study,” Dr. Hermine added.
Sotatercept is currently undergoing phase 2 trials for the treatment of anaemia in various other conditions, including myelodysplastic syndromes, diamond blackfan anaemia, chronic myelomonocytic leukemia, myelofibrosis, and end-stage renal disease.
Beta-thalassemia is characterised by ineffective red blood cell production, which places patients at risk for anaemia, excessive iron, and organ failure.
Dr. Hermine and colleagues have previously shown in animal studies that the cytokine GDF-11 is involved in the decreased production of red blood cells.
[Presentation title: Interim Results from a Phase 2A Open-Label, Dose-Finding Study to Determine the Safety, Efficacy, and Tolerability of Sotatercept (ACE-011) in Adults With Beta-Thalassemia. Abstract S662]
| Sunumlar | Videolar | Olgu Tartışması |
