Frequentist and Bayesian meta‐regression of health state utilities for multiple myeloma incorporating systematic review and analysis of individual patient data

Abstract

This analysis presents the results of a systematic review for health state utilities in multiple myeloma, as well as analysis of over 9,000 observations taken from registry and trial data. The 27 values identified from 13 papers are then synthesised in a frequentist nonparametric bootstrap model and a Bayesian meta‐regression. Results were similar between the frequentist and Bayesian models with low utility on disease diagnosis (approximately 0.55), raising to approximately 0.65 on first line treatment and declining slightly with each subsequent line. Stem cell transplant was also found to be a significant predictor of health‐related quality of life in both individual patient data and meta‐regression, with an increased utility of approximately 0.06 across different models. The work presented demonstrates the feasibility of Bayesian methods for utility meta‐regression, whilst also presenting an internally consistent set of data from the analysis of registry data. To facilitate easy updating of the data and model, data extraction tables and model code are provided as Data S1. The main limitations of the model relate to the low number of studies available, particularly in highly pretreated patients.

Health-related quality of life results from the IFM 2009 trial: treatment with lenalidomide, bortezomib, and dexamethasone in transplant-eligible patients with newly diagnosed multiple myeloma

“…This analysis focused on the following domains considered to be most relevant for this treatment setting, namely QLQ-C30 (i.e. global QoL, physical functioning, role functioning, fatigue, and pain) and side effects of treatment and disease symptoms domains from the QLQ-MY20 [3]. Results from other QLQ-C30 and QLQ-MY20 domains are presented in Supplementary Tables S2 and S3. All the scales in the QLQ-C30 and QLQ-MY20 range in score from 0 to 100.…”

“…Multiple myeloma (MM) is one of the most debilitating hematological diseases, and patients experience substantial pain and fatigue along with significant reductions in physical functioning, role functioning, and overall quality of life (QoL) [1][2][3]. High-dose therapy (HDT) with melphalan followed by autologous stem cell transplantation (ASCT), and subsequent lenalidomide maintenance therapy is one of the standard treatment approaches for newly diagnosed MM (NDMM) in adults who are < 65 years of age and clinically fit [4].…”

EQ-5D–Derived Health State Utility Values in Hematologic Malignancies: A Catalog of 796 Utilities Based on a Systematic Review

“…An example of such an approach in multiple myeloma was presented recently by Hatswell and colleagues. 108 The clinical trials that were included do not always reflect current clinical practice; for instance, some results were made obsolete by more recent trials. In other examples, the results of the trials did not translate into clinical practice or the trials that constituted the basis for treatment standards lacked EQ-5D assessment.…”

Cost-Effectiveness of First-Line Versus Second-Line Use of Daratumumab in Older, Transplant-Ineligible Patients With Multiple Myeloma

“…Our utility values were based on a study by Hatswell et al, which used metaregression to synthesize quality-of-life measurements for multiple myeloma patients from several studies across multiple lines of therapy. 37 In our base case, first-line therapy was associated with a utility of 0.659, second-line therapy had a utility of 0.620, third-line therapy had a utility of 0.606, and fourth-line therapy and best supportive care were assigned a utility of 0.494. QALYs were calculated by weighting patient survival based on utility estimates for each health state.…”