期刊
AMERICAN JOURNAL OF HEMATOLOGY
卷 93, 期 6, 页码 824-840出版社
WILEY
DOI: 10.1002/ajh.25104
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类别
资金
- Gerstner Family Career Development Award
- Mayo Clinic Center for Individualized Medicine, Mayo Clinic, Rochester, MN, USA
- CTSA from the National Center for Advancing Translational Science (NCATS) [KL2 TR000136]
Disease overview: Chronic myelomonocytic leukemia (CMML) is a clonal hematopoietic stem cell disorder with overlapping features of myelodysplastic syndromes and myeloproliferative neoplasms, with an inherent risk for leukemic transformation (similar to 15%-20% over 3-5 years). Diagnosis: Diagnosis is based on the presence of sustained (>3 months) peripheral blood monocytosis (>= 1 x 10(9)/L; monocytes >= 10%), along with bone marrow dysplasia. Clonal cytogenetic abnormalities occur in similar to 30% of patients, while >90% have gene mutations. Mutations involving TET2 (similar to 60%), SRSF2 (similar to 50%), ASXL1 (similar to 40%) and the oncogenic RAS pathway (similar to 30%) are frequent; while the presence of ASXL1 and DNMT3A mutations and the absence of TET2 mutations negatively impact over-all survival. Risk stratification: Molecularly integrated prognostic models include; the Groupe Francais des Myelodysplasies (GFM), Mayo Molecular Model (MMM), and the CMML specific prognostic model (CPSS-Mol). Risk factors incorporated into the MMM include presence of nonsense or frameshift ASXL1 mutations, absolute monocyte count >10 x 10(9)/L, hemoglobin <10 gm/dL, platelet count <100 x 10(9)/L and the presence of circulating immature myeloid cells. The MMM stratifies CMML patients into 4 groups; high (>= 3 risk factors), intermediate-2 (2 risk factors), intermediate-1 (1 risk factor), and low (no risk factors), with median survivals of 16, 31, 59, and 97 months, respectively. Risk-adapted therapy: Hypomethylating agents such as 5-azacitidine and decitabine are commonly used, with overall response rates of similar to 30%-40% and complete remission rates of similar to 7%-17%; with no impact on mutational allele burdens. Allogeneic stem cell transplant is the only potentially curative option, but is associated with significant morbidity and mortality.
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