Low Dose Decitabine for Elderly High Risk MDS Patients: Who Will Respond?

Reviewer: Tracy d'Entremont, MD
Last Modified: December 9, 2002

Presenter: P.W. Wijermans
Presenter's Affiliation: The Hague; Nethrelands
Type of Session: Scientific


    MDS is a rare disease with an inevitably fatal outcome. The treatment is cumbersome, especially in the elderly. Previous studies of treatment for this disease in the elderly have demonstrated lower response rates (RR) and shorter durations of response. A therapy which is based on the biology of the disease is needed. This is a summary of three phase 2 studies utilizing Decitabine, a DNA-hypomethylating agent, for MDS in an elderly population.

Materials and Methods

  • This study enrolled patients with MDS without prior therapy (RAEB, RAEB-T, CMML, and RA/RARS who were transfusion dependent
  • Low dose Decitabine at 40-50mg/m2/day for three days was used in one study and the second and third studies used 15mg/m2 over 4hours t.i.d
  • Cycles were repeated every 6 weeks
  • Treatment was given for a max of 6-8 cycles.


    Data on 169 patients was reported
  • RR 49%
  • CR 23%
  • PR 12%
  • HI 14% (Hematologic Improvement)
  • The median duration of response was 40 weeks
  • The median overall survival was 15 months.
  • The 2-year survival rate was 34%
  • The responses were seen equally in both genders, in all ages, in all IPSS, in all initial LDH levels, and in patients with varying numbers of blasts at diagnosis.
  • The OS was seen to correlate significantly with the following prognostic factors: age < 65 (43% vs. 28%) p=0.046 IPSS score (49% vs. 25% vs. 28%) p=0.012 low risk cytogenetics (43% vs. 24%) p=0.01

Author's Conclusions

  • Decitabine is a mild therapy that can be used even in older patients with high risk disease.
  • The response rate is approximately 50%
  • Cytogenetic remission is seen in approx 31% of patients.

Clinical/Scientific Implications

  • Decitabine may be an effective treatment for elderly high risk patients with MDS. Data from an ongoing randomozed phase 3 trial by the EORTC of Decitabine vs. best supportive care is ongoing to look at survival in these patients and the data is anxiously awaited.

Oncolink's ASH Coverage made possible by an unrestricted Educational Grant from Ortho Biotech.