Treatment for atypical CML – pro
Atypical chronic myeloid leukemia (aCML) is included in the group of myelodysplastic/myeloproliferative neoplasms by the International Consensus Classification and has been renamed as MDS/MPN with neutrophilia by the fifth edition of World Health Organization classification. aCML is a rare disease with an incidence of 1% to 2%; it affects elderly patients with a median age ranging between 60 and 70 years, with a male predominance.5-7 The disease is associated with a poor outcome, with a median overall survival (OS) of 10 to 28 months8 and a high rate of leukemic transformation (>15%–20% at 5 years). There is no standard tetmetn for this rare disease. Allogeneic transplantation is the only known curative strategy. The review by Braccia concludes: ” Unfortunately, no consensus on treatment has been reported until now, and allogeneic transplant remains the only valid option. Palliative chemotherapy (HU, IFN) is the most common treatment in patients not eligible for the transplant procedure. HMAs can be a bridge to the transplant approach, but the results reported, based on small cohort of patients, are not encouraging. Targeted therapies with JAK2 inhibitor (ruxolitinib), SRC kinase inhibitor (dasatinib), and MEK inhibitor (trametinib) in patients with aCML with actionable target mutations have shown some interesting responses, but large cohorts of patients are needed to understand if they could represent a future therapeutic strategy.”
There are three completed trials that can serve as guides to treatment, but I do not see a currently open trial – https://clinicaltrials.gov/search?cond=atypical%20CML
Chen H et al,A case report of atypical chronic myeloid leukemia with complete hematological and major molecular response to Venetoclax/Azacitidine treatment. Front. Oncol., 24 March 2024, Sec. Hematologic Malignancies, Volume 14 – 2024 | https://doi.org/10.3389/fonc.2024.1327834
Breccia M. Atypical CML: diagnosis and treatment. Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):476-482. doi: 10.1182/hematology.2023000448. PMID: 38066919; PMCID: PMC10727105.