Treatment of MDS/MPN and the MDS/MPN IWG International Trial: ABNL MARRO.

ABSTRACT

PURPOSE OF REVIEW MDS/MPNs comprise a group of rare hematologic malignancies that balance features of myeloproliferation and bone marrow failure. Given overlapping clinical features and rarity of incidence, MDS/MPNs have long posed a diagnostic and therapeutic challenge. Herein, we sought to review recent advances in diagnosis and emerging therapeutic strategies and highlight the upcoming ABNL MARRO study which aims to individualize therapy for patients with MDS/MPN. RECENT FINDINGS: Focused study of molecular mutations in MDS/MPNs has provided improved diagnostic clarity. Specific gene mutation or patterns of mutation have been increasingly described and have helped to distinguish between clinically similar diseases. While the current treatment landscape consists largely of therapies that have been co-opted from related disease, the emergence of prospective clinical trials specifically focused on MDS/MPN and the increased use of targeted agents represent progress for patients with MDS/MPN. An improved understanding of the molecular drivers of myeloid diseases has provided diagnostic clarity and renewed hope of targeted therapies for MDS/MPN patients. The upcoming ABNL MARRO study hopes to leverage this knowledge to match patients with targeted therapeutic options specific to molecular drivers of their disease.