Articles related to MYELODYSPLASTIC SYNDROMES

Even after allogeneic hematopoietic stem-cell transplant (HCT), poor outcomes are typically the case in patients with TP53-mutant (mTP53) acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The data from recent trials using the combination of eprenetapopt (APR-246) and azacitidine showed positive preclinical results in mTP53 AML/MDS, and this trial sought to assess the efficacy and safety of the eprenetapopt and azacitidine combination as maintenance therapy after allogeneic HCT in patients with mTP53 AML or MDS.

In an integrated analysis of mutations and clinical outcomes, comprising 2,200 patients with TP53-mutated myelodysplastic syndrome (MDS) with excess blasts (EB) or TP53-mutated acute myeloid leukemia (AML), the authors state that mutant TP53 AML and MDS-EB “do not differ with respect to molecular characteristics and survival” and argue these entities should be considered a single molecular disease entity. In a commentary to the above paper, TP53 and the star-crossed lovers MDS and AML, John Welch, MD, PhD of Washington University School of Medicine writes, “As a junior Hematology/Oncology fellow, I was told there were two types of physicians: splitters and mergers. That is, clinicians either seek to diagnose increasingly homogenously narrow groups of patients based on increasingly refined, shared characteristics, or they seek to find broad, overarching patterns that unite diagnostic classifications. Hematologic malignancies have been fertile ground for the diagnostic splitters of the world. On the other hand, there have been some noteworthy exceptions. Sometimes it is a technological advance that allows for the synthesis of disparate diagnoses.”

The authors analyzed the outcome of 349 patients with primary or secondary myelofibrosis undergoing reduced intensity transplantation, of whom 35 had accelerated-phase myelofibrosis. After a median follow-up of 5.9 years, estimated 5-year overall survival was between the two groups, and median overall survival was not reached. In terms of relapse, five-year incidence was 30% for the accelerated-phase group versus 15% for the chronic-phase group. Reduced intensity transplantation showed excellent survival but higher relapse for accelerated-phase myelofibrosis.

A study published in Blood Advances finds that higher levels of NETosis in myeloproliferative neoplasms (MPNs) are not linked to thrombotic events. The study determined that while NETosis may play a role in facilitating thrombosis, it is not a sole causative determinant in MPN-associated thrombophilia.

Bone marrow failure in children and young adults is often suspected to be inherited; however, in many cases, diagnosis remains uncertain. A new study involving 179 patients (from 173 families) found that implementing precision medicine at diagnosis can improve patient management, along with the use of family counseling. This study published in Blood expands the “portrait” of IBMF syndromes and explores some newly recognized disease entities.

This article presents a review and commentary of selected presentations from the all-virtual 62nd American Society of Hematology (ASH) Meeting and Exposition, which took place in December 2020. Coverage includes safety and efficacy results from various trials and studies, including MEDALIST, DACOTA, P-2001, M15-531, ACE-536-MF-001, Sintra-REV, and COMMANDS.