Side effects from acute myeloid leukemia treatment: results from a national survey

Curr Med Res Opin. 2019 Nov;35(11):1965-1970. doi: 10.1080/03007995.2019.1631149. Epub 2019 Jun 26.


Objective: Acute myeloid leukemia (AML) is experiencing a therapeutic renaissance due to the heightened biomedical understanding of AML and patient-focused drug development (PFDD). Many AML patients now live long-term with the side effects of treatment. This study documents the prevalence and severity of AML treatment-related side effects. Methods: A national cross-sectional survey designed with the Leukemia & Lymphoma Society assessed patients' experiences with short-term (nausea/vomiting, diarrhea, hair loss, mouth sores, infection, rash) and long-term (organ dysfunction, chemobrain, fatigue, neuropathy) treatment side effects. Patient and caregiver participants rated side effect severity (none-severe). Results: Survey participants (n = 1182) were mostly female (65%), AML patients (76%), and had undergone chemotherapy (94%). Eighty-seven per cent of participants reported severe short-term effects, and 33% reported severe long-term effects of treatment. Only 11% of respondents did not have any severe effects. Hair loss and fatigue were the most common severe short- and long-term side effects (78%, 33%). There was a moderate correlation between having short- and long-term adverse effects (r = 0.41, p < 0.001). Caregivers were more likely than patients to report severe organ dysfunction, fatigue, and neuropathy (p-values < 0.05). Conclusions: Survivors experience a high burden of side effects from AML treatments highlighting the need for the development of less toxic therapies. Differences in patients' and caregivers' experiences illustrate the importance of sampling from diverse sources to understand the full burden of AML treatment, and the need for less toxic drugs. This study informs patients, patient-advocacy groups, clinicians, and regulators about AML treatment burdens and provides the community with information to inform PFDD.

Keywords: Adverse effects; Long-term adverse effects; Patient-focused drug development; Quality-of-life: Treatment outcome.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Alopecia / chemically induced
  • Antineoplastic Agents / adverse effects*
  • Cross-Sectional Studies
  • Female
  • Humans
  • Leukemia, Myeloid, Acute / drug therapy*
  • Male
  • Middle Aged


  • Antineoplastic Agents