Patients with high-risk chronic myelomonocytic leukemia (CMML) may experience prolonged overall survival (OS) and increased overall response rate (ORR) from treatment with decitabine, according to a study published in Leukemia.

CMML is a highly complex clonal hematologic disorder that does not have many effective therapeutic options, and management is usually based in supportive care and cytoreductive therapy. CMML is classified among myelodysplastic (MDS)/myeloproliferative neoplasms. Evidence from previous studies suggests that patients with CMML may experience a good response with decitabine.

This phase 2 study ( Identifier: NCT01251627) enrolled 43 patients with CMML to receive decitabine 20 mg/m2 on days 1 to 5 of a 28-day cycle. More than 50% of study patients were considered to be high risk. Follow-up was performed at the end of treatment cycles 4 and 6, and patients who responded to treatment at the end of 6 cycles would continue treatment.

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After a median duration of 51.5 months, the median OS was significantly longer at 17.0 months compared with patients who did not receive decitabine (P =.02).

After 6 cycles of treatment, the ORR was 47.6%. Nineteen percent of patients had marrow responses, 16.6% of patients had complete responses, 9.5% of patients demonstrated hematologic improvements, and 2.4% of patients had a partial response.

The most frequently reported grade 3 or 4 adverse events were thrombocytopenia, anemia, and neutropenia, occurring in 38%, 28.6%, and 50% of patients, respectively.

The authors of the study concluded that “decitabine appears to be an effective treatment for patients with high-risk CMML, including those with proliferative disease. Further research is needed to determine whether there is a difference in response between low- and high-risk patients.”


1. Santini V, Allione B, Zini G, et al. A phase II, multicentre trial of decitabine in higher-risk chronic myelomonocytic leukemia [published online July 7, 2017]. Leukemia. doi: 10.1038/leu.2017.186