OBJECTIVES : In recent years, minimal residual disease (MRD) has become widely used in the management of haematological malignancies. Several studies have shown that MRD negativity significantly correlates with survival outcomes. However, little is known about the correlation between MRD status and other patient-related outcomes. Therefore, a targeted literature review (TLR) was conducted to identify published studies that evaluated MRD in relation to patient-centered outcomes (PCOs), including health-related quality of life (HRQoL), utilities, health care resource use (HCRU) and cost-effectiveness across a range of haematological malignancies.

METHODS : Publications were identified using PubMed, Embase, health technology assessment (HTA) reports and manual search of conference abstracts. Full text publications (last 10 years), conference abstracts and HTA submissions (last 5 years) reporting MRD and PCOs were included.

RESULTS : Eight publications were included. Most studies evaluated MRD at 10^-4 sensitivity threshold. In one study, patients with similar baseline status who achieved MRD negative (MRD-) reported better HRQoL than those achieved MRD positive (MRD+) in multiple myeloma (MM). Two studies in chronic lymphoblastic leukemia (CLL) and acute lymphoblastic leukemia (ALL) showed MRD- patients having better HRQoL. However, 1 study in chronic myeloid leukemia (CML) reported that patients who reached MRD- at 24 months have a marginally higher symptom burden compared to MRD+ patients. In relation to costs, four studies reported that MRD- patients had lower HCRU and costs compared to MRD+ patients in CML and ALL. Two studies stated that the cost-effectiveness of Blinatumomab in treating ALL patients was mainly driven by the high MRD response rate and the lower cost and longer relapse-free duration of being MRD-.

CONCLUSIONS : The beneficial impact of deeper responses in the form of MRD negativity on PCOs was demonstrated in these few studies. However, these data are limited and the relationship between MRD status and PCOs needs to be explored further in future studies.