Refractory AML: Most patients achieve a remission (an absence of signs and symptoms) after initial treatment for acute myeloid leukemia (AML). However, some patients have residual leukemic cells in their marrow even after intensive treatment. Patients who have not achieved complete remission after two cycles of induction chemotherapy are usually diagnosed as having "refractory AML."
Relapsed AML: Some patients reach remission and then have a return of leukemia cells in the marrow and a decrease in normal blood cells. This is called “relapsed leukemia.” At the time of relapse, repeat genetic testing of the leukemia cells is considered “standard of care,” since the mutational pattern at the time of relapse may be different from when the disease was first diagnosed. This can affect treatment decisions.
Treatment options for patients with refractory or relapsed AML include:
- Clinical trials
- Re-treatment with the same induction regimen that produced the patient's first remission
- Allogeneic stem cell transplantation
- Allogeneic stem cell transplantation remains the only potential curative option for patients with relapsed AML, and they must be considered fit enough to undergo the procedure. However, recent approval of several new treatments may help patients who cannot undergo a stem cell transplant to live longer with high quality of life.
- Targeted therapy
- Gilteritinib (Xospata®) for AML with an FLT3 mutation
- Low-intensity therapy (azacytidine or decitabine) plus sorafenib (Nexavar®) for AML with an FLT3 mutation
- Enasidenib (Idhifa®) for AML with an IDH2 mutation
- Ivosidenib (Tibsovo®) for AML with an IDH1 mutation
- Gemtuzumab ozogamicin (MylotargTM) for CD33-positive AML
- Other drug combinations
Research is ongoing to determine optimal drug combinations, doses and administration schedules.
For information about the drugs listed on this page, visit Drug Listings.
For some patients with relapsed or refractory AML, the best treatment route may be one that is being studied in a clinical trial. Several drugs and drug combinations used to treat AML are currently being studied.
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