treatment

Key Points
NK cells phenotyping reveals impaired cytotoxicity, chronic activation and exhaustion in VEXAS syndrome.

Decreased circulating NK cells were independently associated with an increased risk of severe infections in VEXAS syndrome.

Their research led to the 2022 approval of olutasidenib for certain patients with IDH1-mutant AML, which occurs in about 10% of AML. After that success, Dr. Watts and his team began to focus closely on testing the drug in MDS, a related condition that often progresses to AML.

IDH-1 mutations also occur in MDS, at a frequency of 3 to 5%.

Strong Responses
In the current study, the researchers tested olutasidenib in 22 MDS patients with IDH1-mutant tumors. All patients were classified with intermediate to high-risk disease, with 86% being high or very high risk.

A phase 2 study found that eprenetapopt plus azacitidine induced responses in patients with TP53-mutated

Abstract

Steroid-refractory GvHD (SR-GvHD) following allogeneic transplant is a major clinical challenge and is associated with substantial mortality [1].

A phase 2 study found that eprenetapopt plus azacitidine induced responses in patients with TP53-mutated

In this issue of Blood, Takashima et al show that reduced STAT1 abundance in SRSF2-mutant myelodysplastic syndrome (MDS) cells confers protection against interferon (IFN)-driven cell suppression relative to normal wild-type (WT) cells.1 Importantly, the authors demonstrate that treatment with the proteasome inhibitor bortezomib in vitro increases STAT1 abundance and sensitizes SRSF2-mutant cells to IFN. These findings provide potential rationale for using bortezomib therapy in SRSF2-mutated MDS, which is characterized by poor outcomes.

Abstract