OBJECTIVES: Although FLT3 mutations are common in acute myeloid leukemia (AML), their impact on patients’ health-related quality of life (HRQoL) is unclear. In order to inform health economic analyses of gilteritinib—a targeted therapy for FLT3-mutated (FLT3^mut+) relapsed or refractory (R/R) AML—a systematic literature review (SLR) regarding the humanistic burden was conducted to identify data on HRQoL in R/R AML.

METHODS: Embase, MEDLINE, MEDLINE In-Process, and Cochrane databases were searched from inception to September 2019 using terms related to R/R AML, HRQoL, and patient-reported outcomes (PROs). ClinicalTrials.gov and selected conference proceedings were also reviewed. Studies of any design were included if they reported HRQoL or other PROs for adults with R/R AML treated with current standard of care, gilteritinib, or products in phase 3 development.

RESULTS: Of the 452 unique publications identified, 10 publications from seven studies were included in the SLR: one phase 3 randomized controlled trial (gilteritinib; ADMIRAL), three cross-sectional studies, one phase 1 non-randomized study, one observational study, and one qualitative study. Study populations differed, with studies recruiting either patients with R/R AML only or both newly diagnosed and R/R AML. ADMIRAL was the only study that reported data for FLT3^mut+ R/R AML and was the only study to compare the HRQoL of patients treated with specific therapies. Although studies administered different PRO instruments, all highlighted the negative impact of R/R AML on HRQoL, which is typically worse than that of de novo AML. No study compared the humanistic burden of FLT3^mut+ and FLT3 wild‑type R/R AML.

CONCLUSIONS: This SLR highlighted the lack of published evidence regarding the humanistic burden of R/R AML. Only seven studies met the inclusion criteria and only one study (ADMIRAL) focused on patients with FLT3^mut+ R/R AML. Further research is therefore required to inform health economic analyses in FLT3^mut+ R/R AML.