Efficacy and safety of oral decitabine/cedazuridine in the chronic myelomonocytic leukaemia subpopulations from phase 2 and 3 studies

DNA methyltransferase inhibitors (DNMTis) are commonly used in treating chronic myelomonocytic leukaemia (CMML); however, data from prospective studies of DNMTis in CMML are limited. The present analysis evaluated the efficacy, safety and pharmacodynamics of the oral DNMTi decitabine/cedazuridine in the subset of patients with CMML from the phase 2 and 3 trials, which led to the approval of this agent for myelodysplastic syndromes and CMML in the United States and Canada. Potential prognostic factors also were analysed. In all, 34 patients with CMML were screened and 33 were treated. Most patients (76% [n = 25]) had myelodysplastic type-CMML and 77% (n = 24/31 with DNA available for sequencing) had intermediate-2 or high-risk disease noted by CMML-specific prognostic scoring systems. The overall response rate was 76%, with 21% (n = 7) of patients achieving a complete response. Nearly half of the 11 patients who were red blood cell-transfusion dependent at baseline (46%) attained transfusion independence for ≥12 weeks, which was associated with survival. Median overall and transformation-free survival were 35.7 and 28.3 months, respectively, and the safety profile was similar to that previously reported for decitabine. This analysis described the use of decitabine/cedazuridine in CMML from consecutive, prospective, randomised trials and illustrated a median survival of nearly 3 years.

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Publication Details

Journal
British Journal of Haematology
Published
2025-06-16
DOI
https://doi.org/10.1111/bjh.20203
Citations
5
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
8.85
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article

Efficacy and safety of oral decitabine/cedazuridine in the chronic myelomonocytic leukaemia subpopulations from phase 2 and 3 studies

Aram Oganesian, Mary‐Margaret Keating, Gail J. Roboz, Brian F. Leber et al.
5 citations
British Journal of Haematology
Acute Myeloid Leukemia Research
8.85
article

Efficacy and safety of oral decitabine/cedazuridine in the chronic myelomonocytic leukaemia subpopulations from phase 2 and 3 studies

Aram Oganesian, Mary‐Margaret Keating, Gail J. Roboz, Brian F. Leber, Amy Elizabeth Dezern, Harold Keer, Somedeb Ball, Bhavana Bhatnagar, Rena J. Buckstein, Michael Robert Savona, Olatoyosi M. Odenike, Elizabeth A. Griffiths, Guillermo Garcia‐Manero, Amer Methqal Zeidan, Kim‐Hien Dao, Yuri Sano, Harshad Amin
article en
5 citations

Abstract

DNA methyltransferase inhibitors (DNMTis) are commonly used in treating chronic myelomonocytic leukaemia (CMML); however, data from prospective studies of DNMTis in CMML are limited. The present analysis evaluated the efficacy, safety and pharmacodynamics of the oral DNMTi decitabine/cedazuridine in the subset of patients with CMML from the phase 2 and 3 trials, which led to the approval of this agent for myelodysplastic syndromes and CMML in the United States and Canada. Potential prognostic factors also were analysed. In all, 34 patients with CMML were screened and 33 were treated. Most patients (76% [n = 25]) had myelodysplastic type-CMML and 77% (n = 24/31 with DNA available for sequencing) had intermediate-2 or high-risk disease noted by CMML-specific prognostic scoring systems. The overall response rate was 76%, with 21% (n = 7) of patients achieving a complete response. Nearly half of the 11 patients who were red blood cell-transfusion dependent at baseline (46%) attained transfusion independence for ≥12 weeks, which was associated with survival. Median overall and transformation-free survival were 35.7 and 28.3 months, respectively, and the safety profile was similar to that previously reported for decitabine. This analysis described the use of decitabine/cedazuridine in CMML from consecutive, prospective, randomised trials and illustrated a median survival of nearly 3 years.

British Journal of HaematologyVol. 207(2)
Roswell Park Comprehensive Cancer Center (US), Sunnybrook Health Science Centre (CA), NewYork–Presbyterian Hospital (US), The University of Texas MD Anderson Cancer Center (US), Johns Hopkins University (US), Oregon Health & Science University (US), Vanderbilt University (US), Cornell University (US), Queen Elizabeth II Health Sciences Centre (CA), New York Hospital Queens (US), Health Sciences Centre (CA), Yale Cancer Center (US), Yale University (US), University of Chicago (US), Presbyterian Hospital (US), Wheeling Hospital (US), Taiho Oncology (United States) (US), BRCR Global (United States) (US), Sidney Kimmel Comprehensive Cancer Center (US), McMaster University (CA)
Good health and well-being
Openalex Percentile: Top 2%
Acute Myeloid Leukemia Research
8.85
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