Treatment Development of Chronic Myeloid Leukemia


The treatment chosen for your CML largely depends on the phase of your disease, your age and general health. Most people with CML will be treated with drugs called tyrosine kinase inhibitors (TKIs). TKIs work by blocking the activity of an enzyme called bcr-abl and thereby preventing the growth and proliferation of the leukemic cells. The most common decision to be made at diagnosis is which of the three available TKI drugs is most suited to you.  This varies from person to person and your doctor will examine all the information about you that is available to them to decide on the most suitable option.

While these drugs are very effective at controlling the disease, most people are required to take these medications for life to keep the disease under control. Only a minority of patients may be ‘cured’ by TKI therapy and are able to safely stop taking them. People with well-controlled CML are expected to have a normal life expectancy.

In a very small number of patients, a stem cell transplant from a matched donor may be considered. This is only considered in patients who do not respond well to TKI therapy and have progressive CML. This treatment, although offering the prospect of a cure, carries serious risks.

Chronic phase

While you are in the chronic phase of CML, treatment is aimed at controlling your disease, prolonging this phase and delaying the onset of symptoms and complications for as long as possible. When you are first diagnosed with CML it can take one to two weeks to start therapy with a TKI. Patients with an elevated white cell count at diagnosis may be given a short course of chemotherapy tablets called hydroxyurea to reduce the CML count.  Most people tolerate hydroxyurea well.

Adherence also commonly called compliance, to treatment with TKIs for CML is very important for the drugs to work effectively. If there is not enough drug in the body (due to skipped doses), it is possible that the CML cells may become resistant via a process called mutation. Some mutations do not respond well to TKIs; therefore, it is very important to take your medication as directed and do not make any changes without discussing it first with your hematologists.

Advanced phase

Although uncommon, some people already have advanced disease at diagnosis, while others may experience disease progression. Advanced phase treatment is aimed at re-establishing the chronic phase of CML and reducing any troublesome symptoms. There are several treatment options that may be used. The treatment of accelerated and blast phase CML usually involves a more intensive approach. These include more intensive chemotherapy using a combination of drugs like those used to treat acute leukemia in combination with a different TKI.

A stem cell transplant is now generally only used for people in whom the CML has not responded to TKIs, or who were diagnosed in the accelerated phase. Some patients may benefit by participating in a clinical trial.

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