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CMML: finally a target in sight?
22 septembre 2025
By Ana Espino | Published on september 22, 2025 | 3 min read
#Leukemia #CMML #CD123 #Tagraxofusp
Chronic myelomonocytic leukemia (CMML) is a rare and aggressive malignant blood disorder, characterized by clonal proliferation of monocytes and marked clinical heterogeneity. It mainly affects older individuals, carries a high risk of transformation into acute myeloid leukemia, and remains associated with limited median survival.
Current treatments, mainly based on hypomethylating agents such as azacitidine or decitabine, induce only partial and temporary responses, without significantly altering the natural history of the disease. To date, no targeted therapy has been approved for this indication, despite improved understanding of molecular profiles. A major challenge in the management of this condition therefore lies in developing innovative therapeutic strategies capable of selectively targeting leukemic cells without impairing normal hematopoiesis. Among the avenues being explored, CD123, which is overexpressed in most CMML cases, represents a promising target.
#Leukemia #CMML #CD123 #Tagraxofusp
Chronic myelomonocytic leukemia (CMML) is a rare and aggressive malignant blood disorder, characterized by clonal proliferation of monocytes and marked clinical heterogeneity. It mainly affects older individuals, carries a high risk of transformation into acute myeloid leukemia, and remains associated with limited median survival.
Current treatments, mainly based on hypomethylating agents such as azacitidine or decitabine, induce only partial and temporary responses, without significantly altering the natural history of the disease. To date, no targeted therapy has been approved for this indication, despite improved understanding of molecular profiles. A major challenge in the management of this condition therefore lies in developing innovative therapeutic strategies capable of selectively targeting leukemic cells without impairing normal hematopoiesis. Among the avenues being explored, CD123, which is overexpressed in most CMML cases, represents a promising target.
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Scientific reference
Patnaik, M. M., et al. (2025). Tagraxofusp, a CD123-targeted therapy, for chronic myelomonocytic leukemia: final results of a phase 1/2 study. Blood neoplasia, 2(4), 100115
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