Chemotherapy is generally used only in patients with blast phase disease as a way to get the CML back into chronic phase. Very high-dose chemotherapy is sometimes used in the protocol that prepares patients for an allogeneic stem cell transplantation.
Omacetaxine mepesuccinate (Synribo®), a chemotherapy drug, is a treatment option for adults with chronic or accelerated phase CML with resistance to and/or intolerance to two or more TKIs. Omacetaxine can be used to treat all mutations resistant to TKIs including the T315I mutation. In general, its use is limited to patients who have exhausted all other TKI options and who are not candidates for allogeneic transplant.
Omacetaxine is given as a liquid that is injected under the skin. The most common side effects include
- Low red blood cell, white blood cell and platelet counts
- Reaction at the injection site
Asciminib (Scemblix®) has a distinct mechanism of action and is the first-in-class STAMP (Specifically Targeting the ABL Myristoyl Pocket) inhibitor. It is indicated for the treatment of adult patients with:
- Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs).
This indication is approved under accelerated approval based on major molecular response (MMR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
- Ph+ CML in CP with the T315I mutation.
For information about the drugs listed on this page, visit Drug Listings.
- Download or order The Leukemia & Lymphoma Society's free booklet, Chronic Myeloid Leukemia