Myeloproliferative neoplasms and acute myeloid leukemia

In patients with advanced myeloproliferative CMML, first-line decitabine treatment does not improve EFS compared with hydroxyurea.

A randomized phase III trial compared decitabine (DAC) and HY in advanced myeloproliferative chronic myelomonocytic leukemias (CMML). Newly diagnosed myeloproliferative CMML patients with advanced disease were randomly assigned 1:1 to intravenous DAC (20 mg/m2/d days 1-5) or HY (1-4 g/d) in 28-day cycles. The primary endpoint was event-free survival (EFS), events being death and acute myelomonocytic leukemia (AML) transformation or progression. One hundred seventy patients received DAC (n = 84) or HY (n = 86). The mean age was 72 and 74 years in the DAC and HY groups, respectively. Thirty-three percent of DAC and 31% of HY patients had CMML-2. Patients received a median of five DAC and six HY cycles. With a median follow-up of 17.5 months, the median EFS was 12.1 months in the DAC group and 10.3 months in the HY group. Fifty-three (63%) DAC patients achieved a response compared with 30 (35%) HY patients. The median duration of response was similar in both arms (DAC, 16.3 months; HY, 17.4 months). Median overall survival was 18.4 months in the DAC group and 21.9 months in the HY group. Compared with HY, DAC significantly reduced the risk of CMML progression or transformation to acute myelomonocytic leukemia at the expense of death without progression or transformation. Compared with HY, first-line treatment with DAC did not improve EFS in patients with advanced myeloproliferative CMML.

Decitabine Versus Hydroxyurea for Advanced Proliferative Chronic Myelomonocytic Leukemia: Results of a Randomized Phase III Trial Within the EMSCO Network. Itzykson R, Santini V, Thepot S, Ades L, Chaffaut C, et al.  J Clin Oncol. 2023 Apr 1;41(10):1888-1897.

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