Jorge Cortes, MD, Director of the Georgia Cancer Center, provides an overview of chronic myeloid leukemia (CML), discusses treatment options, and offers advice to newly diagnosed patients.
CML is a myeloproliferative neoplasm characterized by the expression of the BCR-ABL1 fusion gene. It presents with neutrophilic leukocytosis. Around 90% to 95% of patients have a shortened chromosome 22, also called the Philadelphia chromosome.
The condition can manifest at any age but most commonly affects middle aged and older individuals. Patients usually present with fatigue, weight loss, anemia, night sweats, and splenomegaly. Approximately 50% of patients diagnosed with CML are asymptomatic and diagnosed due to routine complete blood count.
Allogeneic stem cell transplantation and tyrosine kinase inhibitors are traditional first-lines of therapy for this condition. There are currently four U.S. Food and Drug Administration (FDA) approved tyrosine kinase inhibitors for first-line therapies for patients with CML: imatinib, bosutinib, dasatinib, and nilotinib. There are also several other therapies approved, or in development, as second- and third-line therapies for this rare cancer.
Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) is a chronic condition requiring long-term treatment. Nilotinib has been a trusted treatment for over 17 years, primarily available as Tasigna capsules, which requires strict fasting, posing challenges to patients’ routines and treatment adherence. In November 2024, the FDA approved Danziten, a new nilotinib tablet formulation that eliminates mealtime restrictions while maintaining efficacy and safety. Unlike Tasigna, Danziten can be taken with or without food and uses a re-engineered formulation to enhance bioavailability and reduce food-related risks, including QT prolongation. Danziten is now included in the National Comprehensive Cancer Network (NCCN) guidelines for CML, but it is not interchangeable with other nilotinib products due to differences in formulation and dosing.
For more information on CML and other rare cancers, visit https://checkrare.com/diseases/cancers/