Chronic myeloid leukemia constitutes a model in Onco-hematology, because it was the first chromosomal anomaly described in a malignant disease, but whose leukemogenesis has been particularly well studied, thus allowing major therapeutic advances. It is associated with a recurrent genetic anomaly: the Philadelphia chromosome, balanced reciprocal translocation t (9; 22), which results in a BCR-ABL fusion gene, then an abnormal protein with tyrosine kinase activity, responsible for leukemic transformation. It is also the first disease where molecular targeted therapy has been used successfully; including imatinib; these TKIs showed most mutations, except the dreaded T315I mutation. This led scientists to develop a third generation TKI; It’s Ponatinib. These outpatient treatments allowed for better hematological, cytogenetic and molecular responses, associated with a better quality of life for patients. However, TKIs do not eradicate the disease. However, allogeneic hematopoietic stem cell transplantation remains the only curative treatment at present.
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