Expert Answers to Common Questions on Response Matters: Transforming the Standard of Care in CML by Mastering Response-Guided Treatment
Chronic myeloid leukemia is a myeloproliferative neoplasm characterized by disordered growth of myeloid cells. The hallmark of chronic myeloid leukemia (CML) is an acquired reciprocal translocation between the long arms of chromosomes 9 and 22. This translocation results in the BCR-ABL1 fusion protein, with constitutively active tyrosine kinase activity, and is the underlying driver of CML. BCR-ABL1 testing is widely used to confirm the clinical diagnosis of CML and to assess response to TKI therapy. The development of BCR-ABL1 tyrosine kinase inhibitors (TKIs) has revolutionized the treatment of CML, dramatically improving patient outcomes. Although most CML patients experience excellent clinical outcomes, some CML patients (20-30%) exhibit an acquired resistance to treatment during the disease course, often requiring second- or third-line therapy. Novel TKIs designed to overcome TKI resistance have shown efficacy in early clinical trials and are offering promise to CML patients who do not respond to any of the multiple therapeutic options currently available.
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