What If Acute Myeloid Leukemia Doesn’t Respond or Comes Back After Treatment?

For most types of acute myeloid leukemia

If acute myeloid leukemia (AML) doesn’t go away with the first treatment, newer drugs or more intensive doses of chemotherapy (chemo) drugs may be tried, if they can be tolerated. A stem cell transplant may be tried in younger patients if a suitable stem cell donor can be found. 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 patient’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) rarely will be the first place it recurs, but if it does, it is often treated with chemo given directly into the CSF (during a lumbar puncture/spinal tap).

For those whose remission lasted longer than 12 months, it is sometimes possible to put the leukemia into remission again with more chemo, although this is not likely to be long-lasting. For younger patients (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 may also be considered.

If AML comes back sooner than 12 months, most doctors will 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 patient may want to consider taking part in a clinical trial of newer treatments.

For AML with an IDH2 gene mutation

If the leukemia cells have an IDH2 gene mutation, one option if the leukemia doesn’t go away or if it comes back later might be treatment with a targeted drug such as enasidenib (Idhifa). Other options might include chemo or a stem cell transplant.

For acute promyelocytic leukemia

For patients with acute promyelocytic leukemia (APL) who don’t respond to initial treatment with chemo plus ATRA or who relapse, arsenic trioxide (Trisenox) is often very effective. A stem cell transplant may be another option if a donor can be found.

If treatment with arsenic trioxide achieves a remission, further courses of this drug may be given. An autologous stem cell transplant may also be an option. If a second remission is not achieved, treatment options may include an allogeneic stem cell transplant or taking part in a clinical trial.

Palliative treatment

If further treatment or a clinical trial is not an option, the focus of treatment may shift to controlling symptoms caused by the leukemia, rather than trying to cure the leukemia. This is called palliative treatment or supportive care. For example, the doctor may advise less intensive chemo to try to slow the leukemia growth 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 and appropriate pain-relieving medicines. If medicines such as aspirin and ibuprofen don’t help with the pain, stronger opioid medicines such as morphine 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. It’s very important to let your cancer care team know if you are having pain so that it can be treated.

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.

The American Cancer Society medical and editorial content team
Our team is made up of doctors and master's-prepared nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Last Medical Review: December 9, 2014 Last Revised: November 21, 2017

American Cancer Society medical information is copyrighted material. For reprint requests, please see our Content Usage Policy.