Novel targets in MDS

The study authors conclude that CA-4948 monotherapy is so far well tolerated and with signs of clinical activity in this group of patients with advanced disease. Three of 3 patients with spliceosome mutations achieved a marrow CR or better. The study continues to define RP2D and to obtain definitive PK and PD profiles.


The study authors conclude that their data suggest that the combination of pevonedistat plus azacytidine (PEVO+AZA) reduces mutation burden and the likelihood of treatment-emergent resistance or progression mutations versus AZA, thereby maintaining response for a longer period. The combination of PEVO+AZA may be beneficial in higher-risk MDS and LB-AML, and potentially as a maintenance treatment in AML. This will be further assessed in the ongoing randomized Phase 3 trial (NCT03268954).


The study authors conclude that enasidenib is well tolerated and shows promising efficacy in IDH2-mutated higher risk MDS. Follow-up and accrual is ongoing to better define duration and biomarkers of response.


The study authors conclude that Sabatolimab+HMA showed favorable tolerability in MDS/AML, including in patients with grade 4 cytopenias at baseline (BL). Promising remission rates were seen irrespective of BL blast burden and in older patients and patients with adverse risk mutation. This supports the development of sabatolimab+HMA in the STIMULUS trial program in MDS/AML.


Presentation ID p413-5


Presenter: Ahmad Nanaa, Rochester, United States

The study authors describe the spectrum and characteristics of GATA2 mutations across myeloid neoplasms. There was an impact of different GATA2-mutation types, single versus several GATA2 mutations, and co- mutations patterns on survival. their work was limited by the small number of patients and the inability to completely rule out germline mutation possibility.