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Treatment for relapsed or refractory acute myeloid leukemia (most types)

If acute myeloid leukemia (AML) doesn’t go away completely with induction treatment, sometimes a second, similar course of chemotherapy (chemo), often called reinduction, can be tried. If this isn't helpful, treatment with other chemo drugs or more intensive doses of chemo may be tried, if a person can tolerate them. A stem cell transplant may be an option for some people, as it can allow higher doses of chemo to be used. Clinical trials of new treatment approaches may also be an option.

If the leukemia went away and has now come back, the treatment options depend on the person’s age and health, and on how long the leukemia was in remission. AML most often recurs in the bone marrow and blood. The brain or cerebrospinal fluid (CSF) is rarely the first place where it recurs, but if this happens, it is often treated with chemo given directly into the CSF.

If remission lasted at least a year, it's sometimes possible to put the leukemia into remission again with more chemo, although this is not likely to be long-lasting. For younger people (generally those younger than 60), most doctors would then advise a stem cell transplant if a suitable donor can be found. Clinical trials of new treatment approaches might also be an option.

If AML comes back sooner than 12 months, most doctors would advise a stem cell transplant for younger patients, if possible. Taking part in a clinical trial is another option.

Another option for AML that doesn’t go away or comes back after treatment might be the targeted drug gemtuzumab ozogamicin (Mylotarg).

If the leukemia keeps coming back or doesn’t go away, further chemo treatment will probably not be very helpful. If a stem cell transplant is not an option, a person might want to consider taking part in a clinical trial of newer treatments.

For AML with a mutation in the FLT3 gene

If the leukemia cells have a mutation in the FLT3 gene and the leukemia doesn’t go away or if it comes back later, one option might be treatment with an FLT3 inhibitor such as gilteritinib (Xospata), which is a type of targeted drug.

For AML with a mutation in the IDH1 or IDH2 gene

If the leukemia cells have an IDH1 or IDH2 gene mutation and the leukemia doesn’t go away or if it comes back later, an option might be treatment with a targeted drug called an IDH inhibitor, such as ivosidenib (Tibsovo) or olutasidenib (Rezlidhia) for AML with an IDH1 mutation, or enasidenib (Idhifa) for AML with an IDH2 mutation. Other options might include chemo or a stem cell transplant.

Treatment for relapsed or refractory acute promyelocytic leukemia (APL)

Treatment options for APL that doesn't go away with initial treatment or that relapses depend on which treatments were used before, as well as other factors.

For people whose initial treatment was with the non-chemo drugs all-trans retinoic acid (ATRA) and arsenic trioxide (ATO):

• If the relapse occurs early (usually within about 6 months), treatment will most likely be with some of the same chemo drugs used to treat other types of AML. Another option might be the targeted drug gemtuzumab ozogamicin (Mylotarg).
• If the remission lasts longer, ATO might be used again, possibly along with other treatments such as ATRA and either chemo or the targeted drug gemtuzumab ozogamicin.

If the initial treatment was ATRA plus chemo, ATO (sometimes along with ATRA) is often very effective.

At some point, a stem cell transplant might be a good option if a person is healthy enough. Another option might be taking part in a clinical trial.

Supportive treatment for leukemia that won't go away

If further treatment or a clinical trial is not a good option, the focus of treatment may shift to controlling symptoms caused by the leukemia, rather than trying to cure it. This is called palliative treatment or supportive care. For example, the doctor may advise less intensive chemo to try to keep the leukemia under control instead of trying to cure it.

As the leukemia grows in the bone marrow it may cause pain. It’s important that you be as comfortable as possible. Treatments that may be helpful include radiation therapy to the bones and appropriate pain-relieving medicines. If medicines such as aspirin and ibuprofen don’t help with the pain, stronger opioid medicines are likely to be helpful. Some people may worry about taking stronger drugs for fear of being sleepy all the time or becoming addicted to them. But many people get very effective pain relief from these medicines without serious side effects.

Other common symptoms from leukemia are low blood counts and fatigue. Medicines or blood transfusions may be needed to help correct these problems.

Nausea and loss of appetite can be treated with medicines and high-calorie food supplements.

Infections that occur may be treated with antibiotics.

It’s very important to let your cancer care team know if you are having pain or any other symptoms so that they can be treated.