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.
Highlights
To 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
Of the techniques that can be used for preference elicitation, time trade-off (TTO) is the preferred method according to the guidelines of the National Institute for Health and Care Excellence (NICE) in the UK [8]
Development of the health state descriptions Five health state descriptions were developed to describe the burden of B-precursor ALL and response to treatment: complete remission (CR), complete remission with partial hematological recovery (with minimal risk of bleeding or developing infection: platelets > 50,000/μl, and absolute neutrophil count (ANC) > 500/μl) (CRh), aplastic bone marrow, partial remission (PR), and progressive disease (PD)
Summary
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. HTA agencies prefer utility data derived directly from patients in a clinical trial, using the EQ-5D [6,7,8]. Preference elicitation may be appropriate for determining utility values for rare diseases such as B-precursor ALL, where generating data from clinical trials can be challenging due to trial sample size or patient fitness and questionnaire burden.
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