Novel targets in MDS
LINKS TO ABSTRACTS:
Presentation ID p413-1
Presenter: Guillermo Garcia-Manero, Houston, United States
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.
Presentation ID p413-2
Presenter: Mikkael Sekeres, Miami, United States
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).
Presentation ID p413-3
Presenter: Sangeetha Venugopal, Houston, United States
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.
Presentation ID p413-4
(S168) SABATOLIMAB PLUS HYPOMETHYLATING AGENTS (HMAS) IN PATIENTS (PTS) WITH HIGH-/VERY HIGH-RISK MYELODYSPLASTIC SYNDROME (HR/VHR-MDS) AND ACUTE MYELOID LEUKEMIA (AML): SUBGROUP ANALYSIS OF A PHASE 1 STUDY
Presenter: Andrew Wei, Melbourne, Australia
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.