Real-Life Data of Patients with Chronic Myeloid Leukemia in Amazonas

Background Chronic myeloid leukemia (CML) is a myeloproliferative neoplasm caused by the (t9;22) (q34;q11.2) translocation, known as the Philadelphia chromosome (Ph+), which generates the BCR::ABL1 fusion oncogene. This oncogene is central to CML pathogenesis. The disease accounts for 15% of adult leukemias, predominantly affecting males, with a median diagnosis age of 57 years. CML progresses from a chronic phase to a blast crisis, with risk stratification guiding treatment decisions. Tyrosine kinase inhibitors (TKIs) are the primary treatment, aiming for molecular disease control, deep sustained response, and potential treatment-free remission. This study evaluated the demographic, clinical, and molecular response profiles of CML patients treated with first- and second-generation TKIs at Fundação Hospitalar de Hematologia e Hemoterapia do Amazonas (HEMOAM). Methods This retrospective, longitudinal study analyzed medical records of 176 CML patients diagnosed between 2011 and 2020. Demographic features, clinical status and laboratory findings were obtained from medical records. Results Amongst these patients, 122 were analyzed for treatment outcomes. The mean diagnosis age was 49.6 years, with male predominance. Most patients were in the chronic phase, with high-risk Sokal scores being the most frequent prognostic category. The e14a2 (b3a2) transcript was the most prevalent molecular subtype. Second-generation TKIs achieved higher molecular response rates, reinforcing their therapeutic relevance. Conclusions These findings align with existing evidence and enhance the understanding of the epidemiological, clinical, and therapeutic profiles of Ph + CML in Amazonas state, providing a valuable basis for future research.