Retrospective analysis of prognostic factors associated with response and overall survival by baseline marrow blast percentage in patients with myelodysplastic syndromes treated with decitabine

Abstract

Background: After the World Health Organization (WHO) changed the definition of acute myeloid leukemia (AML) to ≥ 20% blasts, the International Working Group (IWG) response criteria for myelodysplasia were updated. This retrospective analysis evaluated response to decitabine using updated IWG criteria in patients pooled from 2 decitabine trials.

Patients and methods: Outcomes for patients with myelodysplastic syndrome (MDS) with baseline marrow blasts ≥ 20% and < 30% (RAEB-t group) and < 20% (MDS group) were compared.

Results: Patients with RAEB-t (n = 26) had a significantly shorter time from diagnosis to study treatment (7.3 vs. 18.3 months), a higher International Prognostic Scoring System (IPSS) risk (77% vs. 16% high-risk patients), and lower median baseline platelet count (62.3 vs. 112.7 × 10(3)/μL) vs. patients with MDS (n = 157), yet no significant difference in overall response rate (ORR) (15.4% vs. 28.0%). Patients with MDS had better duration of response (9.9 vs. 5 months; P = .024) and overall survival (OS) (16.6 vs. 9.0 months; P = .021) compared with patients with RAEB-t.

Conclusion: Decitabine is active in and may benefit patients with > 20% blasts (RAEB-t).

Trial registration: ClinicalTrials.gov NCT00043381 NCT00260065.

Keywords: Acute myeloid leukemia; FAB classification; Myelodysplastic syndrome; RAEB-t; Survival.

Conflict of interest statement

CONFLICT OF INTEREST

H.K. has received research grants from Eisai Inc. T.K. has received research grants from GlaxoSmithKline. A.T. is an employee of Eisai Inc. J.C. has served on a speaker’s bureau and has received honoraria and research grants from Ariad, Bristol-Myers Squibb, Chemgenex, and Novartis. F.R. has served as a remunerated consultant for Sunesis and Genzyme, has received honoraria from Bristol-Myers Squibb, Celgene, Genzyme, Novartis, and Sunesis, and has received research grants from Bristol-Myers Squibb, Bayer-Onyx, Celgene, EMD Serono, Incyte, Merck, Serono, and Sanofi. A.M., E.J., S.O’B, M.A.W., and G.G-M. declare no competing financial interests. The authors received no honoraria or other form of financial support related to the development of this manuscript.

Copyright © 2013 Elsevier Inc. All rights reserved.

Figures

Figure 1. Pooled analysis of decitabine response rates in patients with RAEB-t and in patients with myelodysplastic syndromes (MDS)

Significant differences in response rates between the RAEB-t and MDS groups were observed only for complete response (P = .028). CR, complete response; HI, hematologic improvement; mCR, marrow complete response; OIR, overall improvement rate; ORR, overall response rate; PD, progressive disease; PR, partial response; RAEB-t, refractory anemia with excess of blasts in transformation; SD, stable disease.