Startseite Kongressberichte 2020 Virtual Edition of the 25th Annual Congress of the European Hematology Association Oral Sessions Chronic Myeloid Neoplasms MDS: MDS:

Novel treatments for MDS I

These IPSS INT-1 LR-MDS pts have unfavorable disease features, including RBC-TD and thrombocytopenia, and poor prognosis, indicating a need for disease-modifying Tx. CC-486 met the primary endpoint of RBC-TI and induced durable bilineage Hgb and PLT improvements. AEs were more frequent with CC-486. Pts with severe neutropenia pre-Tx are at higher risk for hematologic toxicity during early CC-486 Tx and may benefit from a modified dosing regimen. 
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Thomas Cluzeau, Marie Sebert, Ramy Rahmé, et al.
 
The authors of the abstract conclude: 
In this very high-risk elderly population of TP53m MDS and AML, a promising 64% CR/CRi/mCR rate was reached in the evaluable population with AZA+ APR 246 combination, including 57% CR rate in MDS patients. We observed manageable neurologic AEs, mainly in elderly patients with reduced renal function, who therefore require close monitoring and dose reduction if necessary. A phase III international trial comparing AZA vs AZA+ APR-246 in TP53m MDS is ongoing.
 
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Lionel Ades, Justin Watts, Atanas Radinoff, et al.
 
The authors of the abstract conclude: 
Compared with A, P+A led to improved EFS and CR rate in higher-risk MDS pts and numerically longer OS in the ITT population. Notable clinical activity was observed with P+A in poor-risk genetic subgroups. AE rates were similar and there were fewer on-study deaths with P+A vs A. A randomized phase 3 trial (NCT03268954) is in progress to further evaluate P+A vs A.
 
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Uwe Platzbecker, Pierre Fenaux, David P. Steensma, et al.
 
The authors of the abstract conclude: 
Imetelstat achieved an 8-w TI rate of 42% for a median duration of 20 mo, the longest so far reported with any agent in non-del 5q LR-MDS, and 29% of pts achieved TI ≥ 1 y. Furthermore, a high and durable HI-E rate (68% for median of 21 mo) was also achieved in this population of heavily RBC TD, ESA-R/R LR-MDS. Enrollment is ongoing in the Phase 3 portion of IMerge, a placebo-controlled trial of the efficacy and safety of imetelstat, including potential predictive biomarkers of response.
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Tomas Stopka, Lubomir Minarik, Adela Schaffartzik, et al.
 
The authors of the abstract conclude: 

Our data indicate expected beneficial effect of G-CSF pre-treatment to standard AZA therapy in higher risk MDS patients  at the levels of OS and ORR. Biomarker study indicates adverse affects of variants in EZH2, DNMT3A, TP53, & CSF3R.

Support: NV19-08-00144, 18-01687S, 19-03586S, UNCE/MED/016, RVO-VFN64165, RVO 68378050, LM2015040, LQ1604, CZ.1.05/2.1.00/19.0395.