OBJECTIVES: Acute myeloid leukemia (AML) is a rapidly progressing blood cancer for which new treatments are needed. We sought to inform regulatory agencies of this unmet need by documenting and comparing the worries of patients and caregivers affected by AML.

METHODS: A national survey of individuals from the Leukemia and Lymphoma Society database incorporated a previously developed and validated Best-Worst Scaling (BWS) instrument to prioritize respondent worries. Using a balanced incomplete block design (BIBD), respondents assessed subsets of 13 AML-related worries that were identified through rigorous community engagement. In each task, respondents chose which item they worried about the most and the least. Priorities were assessed using standardized best-worst scores (ranging from 0 to 100), and compared across three groups: patients, caregivers of a patient who was alive, and caregivers of a patient who had passed.

RESULTS: We had 892 patients, 158 caregivers of living patients, and 122 caregivers of deceased patients complete the survey (response rate=18%). Patients were most worried about “the possibility of dying from AML” (BW score=74.47, SE=0.60) and the “long-term side effects of treatments” (BW score=70.61, SE=0.53). Patients were least worried about “communicating openly with doctors” (BW score=24.34, SE=0.50). Patient and caregiver responses were highly correlated, Spearman’s rho=0.91. The prioritization of worries between caregiver groups had subtle differences; caregivers of deceased patients reported more worry about “being a burden to others” and “knowing about all the treatment option” and less worry about “the possibility of dying from AML” and the “overall financial cost of AML.”

CONCLUSIONS: The caregivers of patients with AML generally understand the worries of their patients and are able to reliably prioritize them. There are subtle differences in the worries reported by caregivers of living patients and caregivers of deceased patients that merit further investigation.