Unit:
Κατεύθυνση Κλινικές Μελέτες: Σχεδιασμός και ΕκτέλεσηLibrary of the School of Health Sciences
Author:
Diamantopoulou Lydia
Supervisors info:
Γαβριατοπούλου Μαρία, Επίκουρη Καθηγήτρια, Ιατρική Σχολή, Εθνικό και Καποδιστριακό Πανεπιστήμιο Αθηνών, Επιβλέπουσα
Τέρπος Ευάγγελος, Καθηγητής, Ιατρική Σχολή, Εθνικό και Καποδιστριακό Πανεπιστήμιο Αθηνών
Ζαγουρή Φλώρα, Αναπληρώτρια Καθηγήτρια, Ιατρική Σχολή, Εθνικό και Καποδιστριακό Πανεπιστήμιο Αθηνών
Original Title:
Ο ρόλος των "παγίδων" των συνδετών του μετατρεπτικού αυξητικού παράγοντα β, sotatercept και luspatercept, στη θαλασσαιμία
Translated title:
The role of Transforming Growth Factor β ligand “traps”, sotatercept and luspatercept, in thalassemia
Summary:
B thalassemia is a chronic disease characterized by anemia due to ineffective erythropoiesis and peripheral haemolysis. The treatment of b thalassemia major includes chronic transfusions that may be complicated by iron overload. Patients with thalassemia intermedia may become transfusion dependent overtime. Hematopoietic stem cell transplantation and gene therapy are the only curative treatment options, but the associated morbidity and mortality rates are high. During the past few years, two newly introduced agents, sotatercept and luspatercept, have been under investigation. They constitute TGFb ligand traps and they have been proven effective in stimulating erythropoiesis.
The present study analyzes the available clinical trials on sotatercept and luspatercept. An internet-based search was performed, and the resulting data on design, implementation, and outcomes of the published trials were analyzed.
One study on sotatercept and four on luspatercept were found. Published data were available on the sotatercept trial and on three of the luspatercept trials. The sotatercept trial showed a significant improvement in erythropoiesis, hemoglobin levels, and the transfusion load of treated patients. The phase II studies on luspatercept confirmed the safety of the drug, while the preliminary results on the efficacy of the drug from the phase III study that followed were impressive (21.4% in the luspatercept group achieved the primary endpoint versus 4,5% in the placebo group, p<0,0001 and 19,6% a reduction of 33% in the transfusion load versus 3,6% respectively, p<0,0001). The innovative mechanism of action along with the efficacy and safety results are expected to lead to the approval of luspatercept for b thalassemia patients.
Main subject category:
Health Sciences
Keywords:
Thalassemia, b-thalassemia, Ligand traps, Sotatercept, Luspatercept
