An Overview of Chronic Myeloid Leukemia (CML)

Expert overview of chronic myeloid leukemia, including considerations for the unmet needs in management as they exist today.


Daniel J. DeAngelo, MD, PhD: There’s been a paradigm shift for the treatment of patients with chronic phase CML [chronic myeloid leukemia]. It’s important to remember this is a rare disease. [There are] around 6000 to 7000 cases in the United States, and about 90% of patients present with chronic phase or stable phase CML. It’s still important to conduct and document a bone marrow exam to absolutely exclude accelerated phase and blast crisis. It’s also important to do a diagnostic marrow to ascertain a full karyotype. Diagnosis should not be based solely on a BCR-ABL PCR [polymerase chain reaction].

Having said that, the average age is 60 years, but it ranges from pediatric patients to patients in their 90s. [There’s] no cause for CML that we’re aware of other than a high dose of radiation exposure. There doesn’t seem to be a familial inheritance and no racial or ethnic propensity to developing chronic phase CML. In the TKI [tyrosine kinase inhibitor] era in 2020, 2021, and 2022, the average life expectancy of a patient with stable phase CML is roughly equal to those patients without CML. We’re really fixing this disease, but there are clearly outliers; that’s where the art lies, if you will. In terms of making sure a patient is adherent or compliant with the therapy, and that has to do with minimizing toxicities. Even low-grade toxicities can interfere with compliance from the patient. And then ascertain the patient’s milestones and make sure the patient is progressing the way he or she should with the therapy you’ve chosen. That’s the overview of CML. Patients should expect a normal life expectancy, but the issue is minimizing toxicities to maximize compliance and then assessing for the milestones.

The major unmet need for many patients comes in 2 flavors. One is tolerability. Despite all the randomized phase 3 trials, roughly a quarter, depending on the study, but roughly a quarter of patients will come off study. The assumption is the patients are coming off to pursue alternative therapy. But when you’re on the study and then you come off study, there’s very limited information. That’s very important. That’s true of all the phase 3 studies, this discontinuation and the assumption that it’s because of toxicity.

The second unmet need, specifically for younger patients, is the ability to enter into a TKI discontinuation. I’ll rephrase that: to enter into a successful TKI discontinuation. When you’re a young patient, the idea of being on a therapy for the rest of your life can be very daunting, specifically if you want to preserve fertility, such as in a young woman who is yet to have her family. The ability to be rapidly treated, rapidly into a molecular remission, and then have a successful TKI discontinuation is another unmet need.

Transcript edited for clarity.

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