Kết quả điều trị bằng nilotinib (Tasigna) trong bệnh bạch cầu mạn dòng tủy tại Viện Huyết học – Truyền máu Trung ương

Chronic myeloid leukemia (CML) remains a prevalent myeloproliferative neoplasm, posing significant therapeutic challenges despite advances. The advent of Imatinib marked a pivotal breakthrough, dramatically enhancing patient survival and quality of life. However, a critical issue arises with a subset of patients who develop Imatinib resistance or intolerance, primarily due to ABL gene mutations, necessitating alternative strategies. Nilotinib, a second-generation tyrosine kinase inhibitor, was specifically designed to address these limitations by promoting deeper and more durable molecular responses. In Vietnam, Nilotinib has become an established therapeutic choice for patients failing first-line Imatinib. This paper aims to evaluate the treatment outcomes of Nilotinib in chronic myeloid leukemia patients who have shown resistance or intolerance to Imatinib.

Clinical hematologists, oncologists, medical researchers, and pharmaceutical professionals specializing in chronic myeloid leukemia and targeted therapies.

This study assessed the therapeutic efficacy of Nilotinib in 52 chronic myeloid leukemia (CML) patients who experienced resistance or intolerance to first-line Imatinib treatment, a common myeloproliferative neoplasm. Conducted as a case series at the National Institute of Hematology and Blood Transfusion, the research aimed to quantify molecular responses at critical 3-month and 12-month intervals. Patient demographics revealed an average age of 49.6 years, with a predominance of younger patients (55.8% ≤ 50 years) and males (63.5%), consistent with CML epidemiological patterns. The findings demonstrate significant early molecular response, with 92.3% of patients achieving molecular response at 3 months. By 12 months, a major molecular response was observed in 36.5% of the cohort. While the 3-month response rate aligns favorably with or exceeds findings from comparable international studies like ENESTnd, the 12-month major molecular response rate, although lower than some primary Nilotinib studies, was notably higher than other Vietnamese studies specifically on Imatinib-resistant CML patients. This suggests that Nilotinib provides a robust and valuable second-line treatment option, offering effective disease control and contributing to improved long-term outcomes for patients with Imatinib resistance or intolerance. The study underscores the importance of second-generation tyrosine kinase inhibitors in managing complex CML cases and reinforces Nilotinib's role in therapeutic strategies for this patient subgroup.