Perfil demográfico e de resposta molecular dos pacientes com Leucemia Mieloide Crônica em tratamento com inibidores de tirosinoquinase na Fundação HEMOAM

Abstract

Chronic Myeloid Leukemia (CML) is a neoplasm of hematopoietic stem cells caused by a reciprocal translocation between chromosomes 9;22 that gives rise to an abnormal chromosome 22 called Philadelphia (Ph). This episode results in a BCR::ABL1 fusion oncogene responsible for the pathogenesis of the disease. The incidence of the disease corresponds to 15% of all leukemias in adults, with an average age of 57 years and a predominance of males. Recently, the classification of its phases was modified to biphasic (chronic phase and blast crisis), with the chronic phase being the one that presents the best treatment results. The diagnosis is confirmed by the presence of the Ph chromosome and the BCR::ABL1 gene and the current therapeutic goal is to achieve minimal residual disease (MRD) and treatment-free remission (TRL). Objective: To highlight the incidence rate of new cases per year, demographic, clinical and molecular response profile of patients with CML treated with 1st and 2nd generation ITKs at the Fundação de Hematologia e Hemoterapia do Amazonas (F. HEMOAM). Methodology: The study was observational, retrospective, longitudinal and descriptive and evaluated patients with Ph+ CML diagnosed from 2011 to 2020 at F. HEMOAM. The data were tabulated according to the number of new cases per year, age, sex and origin, stage of the disease, Sokal score, type of transcript and BCR::ABL1 results. Results: The results demonstrated a total of 176 patients with CML, a mean age of 46.49 years and a predominance of males. The chronic phase of the disease was the most common at diagnosis (166/94.3%) and the Sokal score, among those classified, had the predominant high risk with 77 cases (43.7%). Regarding transcript subtypes, only 155 were classified, the most common being e14a2 (92/52.27%) followed by e13a2 (60/34.1%) and e13a2/e14a2 (3/1.70%). The molecular response found was confirmed to be earlier and more profound with 2nd generation ITKs. Of the 47 patients with more than four years of treatment, 24 achieved a sustained deep molecular response (18 with imatinib, 3 with dasatinib and 3 with nilotinib). Conclusion: The present study demonstrated the incidence of CML in a younger population in Amazonas and molecular response rates to 1st and 2nd line TKI that coincide with the literature