Treatment with Hypomethylating Agents (HMA)

  • Ulrich Germing
  • Pierre FenauxEmail author
Part of the Hematologic Malignancies book series (HEMATOLOGIC)


In 1993 Silverman and coworkers [1] reported on a phase 2 trial on 43 patients with higher-risk MDS (medullary blast count >10%) using 75 mg/m2 5-Azacytidine (5-Aza) for 7 days every 28 days for 6 cycles. Forty-nine percent of the patients responded, with 12% CR (CR and PR 37%, and 5% improved hematologically without achieving CR or PR), and 51% were nonresponders. This report was the first to show that 5-Aza can result in remission in a reasonable proportion of the patients, and results were very closely reproduced in phase 2 and 3 trials [2–18]. The drug was regarded as differentiating agent, and the authors speculated if 5-Aza was the first drug with benefit for older MDS patients. In 1997 Wijermans et al. [19] published promising phase 2 data on decitabine in high-risk MDS with similar results and subsequently worked on different schedules and on cytogenetic responses as well. Having in mind the very poor prognosis of older high-risk MDS patients after induction chemotherapy [20, 21], the efficacy of HMA appears to be an attractive alternative.


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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Department of Hematology, Oncology and Clinical ImmunologyHeinrich-Heine-UniversityDüsseldorfGermany
  2. 2.Hôpital St Louis (Assistance Publique –Hôpitaux de Paris) and Paris 7 UniversityParisFrance

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