Among patients with advanced, proliferative chronic myelomonocytic leukemia (CMML), front-line decitabine does not appear to improve overall survival or event-free survival (EFS) compared with hydroxyurea, according to research published in the Journal of Clinical Oncology.

In patients with myeloproliferative CMML for whom transplantation is not a clinical option, cytoreductive treatment is frequently used; hydroxyurea has, in previous studies, yielded favorable outcomes in this patient population.

There is, however, an open question to what degree patients may benefit from hypomethylating agents, including decitabine. For this randomized phase 3 study ( Identifier: NCT02214407), researchers compared the safety and efficacy of hydroxyurea with that of decitabine among patients with advanced, proliferative CMML in the front-line setting. The primary endpoint was EFS.

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Overall, 170 patients were randomly assigned to receive decitabine (84 patients) or hydroxyurea (86 patients). In the decitabine and hydroxyurea groups, the median ages were 72 and 74 years, 67% and 71% of patients were male sex, and 74% and 65% of patients had low-risk cytogenetic scores, respectively.

The median follow-up was 17.5 months. At this point, the median EFS was 12.1 months with decitabine vs 10.3 months with hydroxyurea (hazard ratio, 0.83; P =.27). Treatment group did not, furthermore, predict for blasts, platelet count, or likelihood of anemia.

The median duration of response was similar between the groups; median overall survival was 18.4 months with the decitabine group vs 21.9 months in the hydroxyurea group (P =.67)

The rate of response was, however, higher with decitabine (63% vs 35% with hydroxyurea; P =.0004). Decitabine also reduced the risk of progression/transformation to acute myelomonocytic leukemia (cause-specific hazard ratio, 0.62; P =.005).

Decitabine was linked with a higher rate of some adverse events, including infection and cardiovascular events.

“Future studies will aim at translating the superior response rate noted with [decitabine] in this study into a significant long-term survival benefit,” the authors wrote in their report. “Our results stress the need for international, randomized clinical trials in rare and heterogeneous neoplasms such as CMML.”

Disclosures: This research was supported by Janssen Pharmaceuticals. Please see the original reference for a full list of disclosures.


Itzykson R, Santini V, Thepot S, et al. Decitabine versus hydroxyurea for advanced proliferative chronic myelomonocytic leukemia: results of a randomized phase III trial within the EMSCO network. J Clin Oncol. 2023;41(10):1888-1897. doi:10.1200/JCO.22.00437