CPX-351 for higher risk myelodysplastic syndrome: cytarabine and daunorubicin in disguise?

Abstract

Higher-risk myelodysplastic syndrome is a heterogenous disease with a poor prognosis that occurs in older people. 1 Greenberg PL Myelodysplastic syndromes, thy name is heterogeneity. Br J Haematol. 2023; 201: 381-382 Crossref PubMed Scopus (0) Google Scholar Current treatment regimens usually result in a transient and often less than complete response. Only allogeneic haematopoietic stem-cell transplantation (HSCT) can potentially cure a select group of relatively young and fit patients. These patients usually receive remission induction therapy before allogeneic HSCT, but in the absence of well designed, prospective randomised clinical trials, its value remains uncertain. 2 de Witte T Bowen D Robin M et al. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. Blood. 2017; 129: 1753-1762 Crossref PubMed Scopus (213) Google Scholar CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 studyCPX-351 appears to be active and safe in patients with higher-risk myelodysplastic syndrome and chronic myelomonocytic leukaemia, allowing bridging to allogenic HSCT in most patients. Full-Text PDF