ArticleHaematologica2026
A retrospective analysis of upfront treatment strategies in chronic myelomonocytic leukemia: impact on survival and response patterns.
Article in Haematologica, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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15 authors.
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Abstract
In a retrospective analysis of 457 Mayo Clinic patients (median age 72 years) with chronic myelomonocytic leukemia (CMML) (proliferative 38%; CMML-2 15%), overall survival (OS), calculated from time of diagnosis, was not differentially affected by treatment exposure to: i) either cytotoxic or non-cytotoxic drugs (i.e., untreated; N=155; median 29 months); ii) non-cytotoxic drugs (N=95; median 25 months); iii) hydroxyurea (HU; N=102; median 23 months) or hypomethylating agent (HMA; N=78; median 35 months), as the first-line choice of cytotoxic therapy; or iv) cytotoxic drugs other than HU or HMA (N=27; median 18 months) (P=0.2). Blast transformation (BT) was more frequent in patients exposed to cytotoxic (26%) versus non-cytotoxic (11%) drugs (P<0.01), confirmed in multivariable analysis (HR [Hazard Ratio] 2.0; 95% Confidence Interval [CI]: 1.2-3.3) that accounted for other risk factors. Comparison of patients receiving HU or HMA favored HMA for control of leukocytosis (P<0.01), anemia (P=0.02), and thrombocytopenia (P=0.06); however, there was no difference in OS (P=0.3) or BTfree survival (P=0.7) between the two treatment arms. RUNX1 mutation was associated with lower response rates in leukocytosis (P=0.03) and thrombocytopenia (P=0.03) in HU/HMA treated patients. Allogeneic stem cell transplantation was undertaken in 49 patients with median post-transplant survival of 69 months. Our study confirms that current drug therapy in CMML has no impact on OS. Instead, it suggests a significant association between BT and the need for cytotoxic drug therapy. Our observations regarding higher response rates with HMA versus HU are also in line with a previously published controlled study.
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