CPX-351 therapy

We described the case of a patient with SM with associated AML-MRC, who was successfully treated with CPX-351 followed by allo-SCT. The concomitant diagnosis of SM-AML with MDS genetic or cytogenetic related mutations is a particularly rare entity, where the transformation usually occurs as a second step during the disease evolution. At present, there are no specific guidelines for the treatment of SM-AML and data from literature mostly report an unsatisfactory outcome. IAs far as we know, this is the first case showing that CPX-351 followed by allo-SCT can result in the achievement of a persistent CR in MDS-related SM-AML. This observation provides the rationale for future prospective studies exploring the efficacy of this therapeutic option for the treatment of these rare cases of SM.