Considerations and challenges for patients with refractory and relapsed acute myeloid leukaemia

•Relapsed and refractory AML remains an area of unmet clinical need.•Cytogenetics and duration of first CR remain the most important prognostic factors.•Treatment decisions should be based on patient characteristics and performance status.•Intensive chemotherapy can induce second remission in up to 76% of younger adults.•Novel agents need further randomised studies to confirm clinical benefit.

Despite advances in understanding the complexities of acute myeloid leukaemia (AML), the treatment of refractory or relapsed AML (rrAML) remains a daunting clinical challenge. Numerous clinical trials have failed to identify new treatments or combinations of existing therapies that substantially improve outcomes and survival. This may be due, at least in part, to heterogeneity among study patients with respect to multiple inter-related factors that have been shown to affect treatment outcomes for patients with rrAML; such factors include age, cytogenetics, immunophenotypic changes, and (in the case of relapsed AML) duration of first complete remission, or if the patient has had a previous blood and marrow transplant (BMT). A clear understanding of disease characteristics and patient-related factors that influence treatment response, as well as expected outcomes with existing and emerging therapies, can aid clinicians in helping their patients navigate through this complex disease state.