Novel trial designs for high-risk myelodysplastic syndromes
Mené sur 299 patients atteints d'un syndrome myélodysplasique à haut risque, cet essai randomisé international de phase III évalue l'efficacité, du point de vue de la survie globale, et la toxicité du rigosertib après l'échec d'un traitement à l'aide d'azacitidine ou de décitabine
Myelodysplastic syndromes are a heterogeneous group of clonal disorders of bone marrow characterised by peripheral blood cytopenia and can progress to leukeamia. Until recently, conventional treatments had not been shown to have a clear survival benefit, with the exception of a small proportion of patients eligible for allogeneic transplantation. In early-stage disease, standard care is based on supportive transfusions and growth factors. When disease progresses to leukaemia, prognosis is poor.