Novel treatments for MDS II

MBG453 combined with Dec or Aza was safe and well tolerated in pts with HR-MDS and AML, with the most common TEAEs consistent with that for single-agent HMA. Both combinations showed antileukemic activity with encouraging response rates and emerging durability, supporting further development of MBG453 combined with HMAs in MDS/AML. (A Brunner & AH Wei contributed equally
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Nico Gagelmann, Anita Badbaran, Rashit Bogdanov, et al.
 
The authors of the abstract conclude: 
This proposed model incorporates genetic and clinical information being the first that was developed and externally validated specifically in CMML undergoing HCT. Furthermore, the model suggests improved prognostic ability for post-HCT outcome and significantly reclassifies patients' individual risk. Therefore, this model may facilitate personalized counseling and treatment stratification.
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Sallman David, Monzr Al Malki, Adam Asch, et al.
 
The authors of the abstract conclude: 
Magrolimab is a novel macrophage targeting immunotherapy that with AZA is well tolerated with durable efficacy in MDS.  A potential registration single arm MDS cohort is ongoing (NCT03248479).  ENHANCE, a randomized Ph3 MDS trial of magrolimab + AZA vs. AZA is being initiated. Additional patients, follow up and translational analyses will be reported at time of presentation. Funded by Forty Seven and the California Institute for Regenerative Medicine
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Amer M. Zeidan*1, Daniel A. Pollyea2, Jacqueline S. Garcia, et al. 
 
The authors of the abstract conclude: 
Ven+Aza combination therapy was well tolerated in pts with RR-MDS and the AEs were manageable. The CR+mCR of 40% and estimated 12-month OS of 65% with Ven+Aza is encouraging. Updated analyses on safety, efficacy, genetic alterations, and BCL-2 family member expression with outcomes will be presented.
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Jacqueline S. Garcia, Andrew H. Wei, Uma Borate, et al.
 
The authors of the abstract conclude: 
This preliminary analysis shows promising efficacy of Ven+Aza combination treatment across all IPSS-R cytogenetic risk categories, and suggests prognostic relevance of the IPSS-R cytogenetic scoring system. Further analyses evaluating the association of genetic alterations and BCL-2 family member expression on efficacy outcomes will be presented.

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