Stem Cell Transplant for Chronic Myeloid Leukemia (CML)
A stem cell transplant allows doctors to use high doses of chemotherapy (chemo) and/or radiation therapy to treat some types of blood cancer. Stem cell transplant is sometimes used to treat chronic myeloid leukemia (CML), although it’s not common.
What is a stem cell transplant?
For a stem cell transplant (SCT), doctors use very high doses of chemotherapy (sometimes along with radiation therapy) to destroy the cells in a person’s bone marrow. This includes both healthy cells and the leukemia cells.
After these treatments, the person gets a transplant (transfusion) of blood-forming stem cells to restore the cells in their bone marrow.
Stem cell transplant for CML
The type of transplant used to treat CML is known as an allogeneic stem cell transplant. In this type of transplant, the stem cells come from someone else (a donor).
Where do the stem cells come from?
Blood-forming stem cells used for an allogeneic transplant can come from:
- Blood (called a peripheral blood stem cell transplant or PBSCT)
- Bone marrow (called a bone marrow transplant or BMT)
- Umbilical cord blood
Bone marrow transplant was more common in the past, but today it has largely been replaced by PBSCT.
Lowering the chance of serious side effects
To lower the chance of serious health problems, your donor needs to closely match your tissue type. A close relative, like a brother or sister, is often a good match. Less often, a matched unrelated donor or umbilical cord blood is used.
This type of transplant can cause severe or even life-threatening complications and side effects, so it's often not a good option for people who are older or have other serious health problems.
When might a stem cell transplant be used to treat CML?
Targeted drugs called tyrosine kinase inhibitors (TKIs) are usually very effective in treating CML. They are typically the first treatment used. But these drugs need to be taken daily, and they might not cure most people. They might also become less effective over time if the CML cells start to change.
A stem cell transplant is more likely to be recommended for advanced CML (in accelerated or blast phase) that isn’t responding well to TKIs.
An allogeneic SCT offers the best chance to cure CML, so doctors might also recommend a transplant for younger people, especially children. But TKIs are still likely to be the first treatment used.
A stem cell transplant is more likely to be considered if a matched donor is available, such as a well-matched sibling.
More information about stem cell transplant
To learn more about stem cell transplants, including how they are done and their potential side effects, see Stem Cell Transplant for Cancer.
For more general information about side effects and how to manage them, see Managing Cancer-related Side Effects.
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- References
Developed by the 91黑料不打烊 medical and editorial content team with medical review and contribution by the American Society of Clinical Oncology (ASCO).
Kantarjian H, Cortes J. Chapter 98: Chronic myeloid leukemia. In: Niederhuber JE, Armitage JO, Doroshow JH, Kastan MB, Tepper JE, eds. Abeloff's Clinical Oncology. 6th ed. Philadelphia, Pa: Elsevier; 2020.
National Cancer Institute. Chronic Myeloid Leukemia Treatment (PDQ?)–Health Professional Version. 2025. Accessed at https://www.cancer.gov/types/leukemia/hp/cml-treatment-pdq on April 25, 2025.
National Comprehensive Cancer Network, Clinical Practice Guidelines in Oncology (NCCN Guidelines?): Chronic Myeloid Leukemia. Version 3.2025. Accessed at https://www.nccn.org on April 25, 2025.
Schiffer CA, Atallah E. Overview of the treatment of chronic myeloid leukemia. UpToDate. 2025. Accessed at https://www.uptodate.com/contents/overview-of-the-treatment-of-chronic-myeloid-leukemia on April 25, 2025.
Last Revised: June 16, 2025
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