Population preference values for health states in relapsed or refractory B-precursor acute lymphoblastic leukemia in the United Kingdom

Abstract

BackgroundTo date, reliable and comprehensive health-related quality of life data for patients with relapsed or refractory B-precursor acute lymphoblastic leukemia (ALL) have not been collected in clinical trials of the disease, and no utility studies have been published. The purpose of this study was to define and validate health states experienced by adults with relapsed/refractory B-precursor ALL, and to assign utility values to these health states using time-trade off methodology.MethodsThis study was conducted in the UK in three phases. In the first phase, five health state descriptions were developed based on a recent clinical trial. The second phase validated the health state descriptions with clinicians and patients with experience of relapsed/refractory B-precursor ALL. The third phase involved prospective health state valuation using time-trade off methodology in a sample of the general public. The study was approved by the UK National Health Service Research Ethics Committee.ResultsIn total, 123 participants were recruited and included in the final analysis; all participants gave written, informed consent. Complete remission was the most preferred health state (mean utility [SEM], 0.86 [0.01]), followed by complete remission with partial hematological recovery (with minimal risk of bleeding or developing infection) (0.75 [0.02]); aplastic bone marrow (0.59 [0.02]); partial remission (0.50 [0.03]); and progressive disease (0.30 [0.04]).ConclusionsThis is the first study to report utility values for health states associated with relapsed/refractory B-precursor ALL. It was designed and conducted to align with NICE guidance on alternative methods for generating health state utility values when EQ-5D data are either unavailable or inappropriate. These utilities can be applied in future cost-effectiveness analyses of treatment for relapsed/refractory B-precursor ALL.Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-015-0377-3) contains supplementary material, which is available to authorized users.

Cost-Effectiveness of Chimeric Antigen Receptor T-Cell Therapy in Pediatric Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia

“…Health utility characterizes quality of life, which ranges from 0 (death) to 1 (perfect health). We obtained health utility scores for separate health states from the literature (37)(38)(39)(40)(41)(42). For patients experiencing toxicity, we subtracted health utility from their baseline scores.…”

Social preferences for health states associated with acute myeloid leukemia for patients undergoing treatment in the United Kingdom

“…Although preference values aligned with clinical perception for AML as a disease, they generally represented other acute leukemias as milder disease. Health state utility values derived from a preference elicitation study conducted in the UK for patients with relapsed/refractory acute lymphoblastic leukemia (R/R ALL) demonstrated higher score values compared with AML preference values obtained in this study [ 26 ]. Although progressive disease (0.3) was not an HS defined in this study, it is akin to the relapse HS (0.1) used herein and was valued higher in patients with ALL.…”

Estimating health state utilities in hemophagocytic lymphohistiocytosis

“…These searches suggested that most studies reported data from patients only after they have recovered [12]. Aristides et al report values for adults with acute lymphoblastic anemia; with 0.50 for partial recovery after BMT and 0.30 for progressive disease [13]. Similarly, in acute myeloid leukemia Castejón et al (2018) report utility estimates of 0.28 for BMT; and 0.36 for chemotherapy during a month long hospital stay [14].…”