Purpose

To assess the efficacy and tolerability of lower and conventional dose regimens of decitabine in patients with selected lower risk myelodysplastic syndrome (MDS).

Patients and Methods

The patients recruited in this study, in addition to having an IPSS score ≤ 1.0, also had to have at least one of the following indicators (with the tendency of malignant clonal expansion): (1) more than 2% marrow blasts, or more than 2% of CD34+ cells in CD45 positive subset. (2) poor karyotype by IPSS scoring, (3) unfit for immunosuppressive treatment(IST), (4) not benefited from best supportive care. The tested patients received decitabine by two different regimens: group A: 15mg/m2×5 days every 28 days , and group B: 20mg/m2×5 days every 28 days. Primary efficacy end point was overall response rate (ORR), complete remission [CR], marrow CR [mCR], CR without platelet recover [CRp], hematologic improvement [HI] or marrow CR plus hematologic improvement [mCR+HI]), cytogenetic response, hematological adverse effects .Secondary end points were progression free survival (PFS) and overall survival (OS).

Results

Total 34 patients were treated by decitabine（Group A, n = 23; group B, n=11）.

The median time of the 34 patients from MDS diagnosis to treatment was 6 months and 100% of them were de novo MDS. The total ORR and CR rate for the 34 patients were 70.6% (24/34) and 26.5% (9/34) respectively. ORR was 69.6% for group A and 72.7% for group B, and CR was 30.4% and 18.2% for group A and B respectively. The cytogenetic response in group A and group B were 55.6% (5/9) and 100%(1/1) respectively. All the difference was statistically insignificant. Also, no statistically differences were observed in secondary end points, OS from the diagnosis of MDS was 43 months in group A and not reached in group B respectively. And PFS were 44 months in group B and not reached in group A respectively. There were no difference between two groups in incidence of grade 3-4 neutropenia and thrombocytopenia. No therapy related death occurred in the 34 patients.

Conclusion

Decitabine is effective and safe in selected patients with lower-risk MDS. Lower and conventional dose of decitabine showed the same efficiency and safety in these patients.

Disclosures:

No relevant conflicts of interest to declare.

Topics:
decitabine, myelodysplastic syndrome, risk reduction, prostatic hypertrophy risk score, surrogate endpoints, adverse effects, cd45 antigens, complete remission, c-reactive protein, cyclic amp receptor protein

Author notes

*

Asterisk with author names denotes non-ASH members.

© 2013 by The American Society of Hematology
2013
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