63rd ASH Annual Meeting and Exposition
December 11-14, 2021
  • Low Risk Myelodysplastic Syndrome Prognosis and Treatment
  • Treatment of High Risk Myelodysplastic Syndrome
  • Treatment of HIgh Risk and Relapsed/Refractory Myelodysplastic Syndrome

637. Myelodysplastic Syndromes – Clinical and Epidemiological: Low Risk Myelodysplastic Syndrome Prognosis and Treatment


Elsa Bernard, Heinz Tuechler, Peter L. Greenberg, et al.

The IPSS-M risk score can be described as continuous, interpretable, and reproducible. It integrates information from 38 gene mutations. This leads to improved discrimination compared to the IPSS-R score. In addition, the IPSS-M risk score restratifies almost half of the MDS patients.



Marie Sebert, Thomas Cluzeau, Odile Beyne Rauzy, et al.

Ivosidenib achieved a significant response in MDS patients, with 91 percent particularly high in treatment-naive MDS patients at higher risk and IDH1 mutations (cohort B). Ivosidenib was well tolerated in all cohorts. Results of the molecular monitoring of IDH1 mutations will be presented in the oral session.



Lionel Ades, Sophie Dimicoli-Salazar, Marie Sebert, et al.

Enasidenib in patients with MDS does not show any limiting toxicity. 42 percent of patients respond to treatment. This response is particularly encouraging in first-line (low and high-risk) patients. Updates to the study are presented in the oral session.




Jesus D Gonzalez-Lugo, Suman Kambhampati, Abdulraheem Yacoub, et al.

In evaluable patients with MDS with a low / int risk, lenalidomide plus eltrombopag demonstrate an ORR of just under 41 percent and with a sustained response. The security profile is acceptable. See more info in the abstract.


66 ASH denotes this abstract as clinically relevant

Guillermo Garcia-Manero, James K McCloskey, Elizabeth A. Griffiths, et al.

An oral fixed-dose combination of decitabine/cedazuridine showed an equivalent PK exposure of 20 mg / m2 IV DEC in MDS patients and led to mlFS and mOS of more than 32 months. For MDS patients with lower risk and indicated treatment, the active ingredient was generally well tolerated after prolonged treatment.

637. Myelodysplastic Syndromes – Clinical and Epidemiological: Treatment of High Risk Myelodysplastic Syndrome

241 ASH denotes this abstract as clinically relevant

Jacqueline S. Garcia, Andrew H. Wei, Meagan A. Jacoby, et al.In MDS with a higher risk, Venetoclax plus Azacitidine leads to a rapid and sustained response and high remission rates. Meaningful clinical and molecular responses were observed in all major mutation profiles. Updated data will be presented in the oral session.


243 ASH denotes this abstract as clinically relevant

Pierre Peterlin, Pascal Turlure, Patrice Chevallier, et al.

The study authors show that CPX-351 is effective for MDS/CMML with higher risk. This is particularly true for achieving blast clearance and as a bridge to allo SCT.


244 ASH denotes this abstract as clinically relevant

Andrew M. Brunner, Jordi Esteve, Kimmo Porkka, et al.

Sabatolimab + HMA demonstrated a sustained clinical response in patients with vHR / HR-MDS and ND-AM. According to the study authors, the combination is safe and well-tolerated.



Sangeetha Venugopal, Hagop Kantarjian, Abhishek Maiti, et al.

The combination Venetoclax + ASTX727 (cedazuridine/decitabine) appears to be safe according to the study authors. It demonstrates preliminary efficacy in patients with MDS or CMML at increased risk and excess blasts.



David A. Sallman, Rami S. Komrokji, Amy E. DeZern, et al.

Treatment of eprenetapopt (APR-246) and azacitidine (AZA) in mTP53 MDS/AML was well tolerated and high response rates were achieved.

637. Myelodysplastic Syndromes – Clinical and Epidemiological: Treatment of HIgh Risk and Relapsed/Refractory Myelodysplastic Syndrome


Alexandre Bazinet, Elias J. Jabbour, Hagop Kantarjian, et al.

Treatment with AZA-VEN was tolerable and effective in this high-risk patient group, with a very high ORR. AZA-VEN was shown to be an effective bridge to aHSCT.



Rami S. Komrokji, Najla Al Ali, Onyee Chan, et al.

In the higher risk MDS, first-line hypomethylating agents / Venetoclax achieved significantly higher complete response rates including the mutated ASXL-1 MDS compared to first-line hypomethylating agents (HMA) alone. The data suggest promising activity in those who received first-line HMA / Venetoclax and AHSCT (2-year OS 91%). The Venetoclax-add-back strategy to HMA after first-line HMA failure has clinical activity and has been associated with an overall survival benefit.


537 ASH denotes this abstract as clinically relevant

Amer M. Zeidan, Uma Borate, Daniel A. Pollyea, et al.

In this very difficult-to-treat patient population, an ORR of 39 percent and an RBC and platelet TI rate of 36 percent were achieved. The median OS was 12.6 months. The study authors suggest that the Ven + Aza treatment leads to significant clinical benefits. Additional analyzes will be presented in the oral session.



Mrinal M. Patnaik, Haris Ali, Eunice S. Wang, et al.

TAG monotherapy first-line in patients with CMML shows significant clinical activity in patients with high-risk disease as well as in patients with R/R disease. The well-tolerated TAG therapy has a manageable and predictable safety profile. The study authors suggest that TAG could offer a novel targeted approach for patients with CMML.



Xinping Zhou, Fanjun Meng, Yanjuan Lin, et al.

Decitabine plus (all-trans retinoic acid) ATRA achieved an OR rate of 85.2 percent in the MDS subtype EB. The study authors conclude that this therapy may be a new treatment option for EB patients.



Meagan A. Jacoby, David A. Sallman, Bart L. Scott, et al.

CPX-351 (a dual-drug liposomal encapsulation of daunorubicin and cytarabine in a synergistic 1: 5 molar drug ratio) shows a tolerable safety and a promising efficacy profile in an MDS population with a higher risk suitable for transplantation. Updated safety and efficacy results will be presented in the oral session.


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