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Stem Cell Transplantation

For certain patients with CML, allogeneic stem cell transplantation (the infusion of donor stem cells into a patient) is their best treatment option. However, this type of transplant can cause serious or even life-threatening complications and side effects. In addition, it is often not a good option for older patients or for patients who have other health problems.

The decision to pursue allogeneic transplantation has become more complicated because many patients have very good responses to TKIs. It is true that stem cell transplantation has been proven to be curative for some CML patients; but today, treatment with TKIs may control the disease for very long periods and preserve quality of life without the serious side effects of transplantation.

Doctors consider many important factors when deciding if an allogeneic transplant is the preferred choice of treatment for a patient. These factors include the patient’s age, general health, phase of CML, history of poor response to other treatments, and the availability of a well-matched donor. Results of transplants using stem cells from matched sibling donors are very similar to those of transplants that use stem cells from matched unrelated donors. Stem cell transplantation is considered for patients who have resistance to at least two types of TKIs, for patients whose CML is in accelerated or blast phase, and for patients who are intolerant to all TKIs.

The most important prognostic factor for post-transplant survival is the patient’s phase of CML. Approximately 80 percent of patients with chronic phase CML will be disease-free for 5 years after transplant. In patients with accelerated phase CML, approximately 40 to 50 percent are disease-free after 5 years, and only 10 to 20 percent of blast phase patients are alive and disease-free after 5 years.

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