Treatment Options for Relapsed/Refractory Acute Myeloid Leukemia (AML)1,2

Treatment options for patients with relapsed or refractory AML include1:

  • Clinical trials (strongly preferred per NCCN Guidelines)
  • Targeted therapies for patients with actionable mutations followed by matched sibling or alternative donor HCT
  • Chemotherapy followed by matched sibling or alternative donor HCT
  • A repeat of the initial successful induction regimen may be used if ≥12 months since induction regimen
  • Best supportive care

Treatment options are thus restricted by mutation status, overall health, response to previous therapeutic agents, clinical trial eligibility, and availability of a matched donor.

Up to 25%-50% of patients with AML will require a re-induction therapy after experiencing induction failure.2

  • Four-year survival in a cohort of 150 patients in induction failure was 23%3
  • Four-year survival rates were significantly higher in patients who received a transplant versus those who did not (48% vs 4%)3
  • Patients who fail primary induction and are treated with a second round of induction therapy have CR rates below 50%2

Response rates are reduced with additional rounds of therapy; 5-year survival is lower in patients achieving CR after second induction (23%) versus after first induction (40%).4

For approximately 25 years, there were no advances in the treatment for AML—until 2017. But despite these new advances, this population of patients has an unmet need because the 5-year survival rate is ~30%.5,6

References: 1. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia V.3.2021. © National Comprehensive Cancer Network, Inc. 2021. All rights reserved. Accessed April 22, 2021. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 2. Döhner H, Estey E, Grimwade D, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129(4):424-447. 3. De Kouchkovsky I, Abdul-Hay M. Acute myeloid leukemia: a comprehensive review and 2016 update. Blood Cancer J. 2016;6(7):e441. 4. Othus M, Appelbaum FR, Petersdorf SH, et al. Fate of patients with newly diagnosed acute myeloid leukemia who fail primary induction therapy. Biol Blood Marrow Transplant. 2015;21(3):559-564. 5. Bloomfield CD, Estey E, Pleyer L, et al. Time to repeal and replace response criteria for acute myeloid leukemia. Blood Rev. 2018;32(5):416-425. 6. Saygin C, Carraway HE. Emerging therapies for acute myeloid leukemia. J Hematol Oncol. 2017;10(1):93-107. 7. Cancer Stat Facts: Leukemia - Acute Myeloid Leukemia (AML). National Cancer Institute website. http://seer.cancer.gov/statfacts/html/amyl.html. Accessed May 22, 2021.