Dataset Information

Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France.

ABSTRACT: Aim: To map patient-level data collected on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC) QLQ-C30 to EQ-5D-5L data for estimating health-state utilities in patients with paroxysmal nocturnal hemoglobinuria (PNH). Materials & methods: European cross-sectional PNH patient survey data populated regression models mapping EORTC QLQ-C30 domains (covariates: sex and baseline age) to utilities calculated with the EQ-5D-5L French value set. A genetic algorithm allowed selection of the best-fitting between a set of models with and without interaction terms. We validated the selected algorithm using EQ-5D-5L utilities converted from EORTC QLQ-C30 data collected in the PEGASUS phase III, randomized controlled trial of pegcetacoplan versus eculizumab in adults with PNH. Results: Selected through the genetic algorithm, the ordinary least squares model without interactions provided highly stable results across study visits (mean [±SD] utilities 0.58 [±0.42] to 0.89 [±0.10]), and showed the best predictive validity. Conclusion: The new PNH EQ-5D-5L direct mapping developed using a genetic algorithm enabled calculation of reliable health-state utility data required for cost-utility analysis in health technology assessments supporting treatments of PNH.

SUBMITTER: Wojciechowski P

PROVIDER: S-EPMC10402747 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature

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Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France.

Wojciechowski Piotr P Wdowiak Marlena M Hakimi Zalmai Z Wilson Koo K Fishman Jesse J Nazir Jameel J Toumi Mondher M

Journal of comparative effectiveness research 20230413 5

<b>Aim:</b> To map patient-level data collected on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC) QLQ-C30 to EQ-5D-5L data for estimating health-state utilities in patients with paroxysmal nocturnal hemoglobinuria (PNH). <b>Materials & methods:</b> European cross-sectional PNH patient survey data populated regression models mapping EORTC QLQ-C30 domains (covariates: sex and baseline age) to utilities calculated with the EQ-5D-5L French value ...[more]

PMID: 37052120

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Project description:BackgroundThe minimal important difference (MID) is a useful tool to interpret changes in patients' health-related quality of life. This study aims to estimate MIDs for interpreting within-patient change for both components of the EQ-5D-5L questionnaire [EQ-Visual Analogue Scale (EQ-VAS) and utility index] and domains of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) for cancer patients.MethodsData were obtained from the Cancer 2015 dataset, a longitudinal cohort of Australian cancer patients. Anchor-based approaches were used to estimate MIDs for the EQ-5D-5L index-based utility index [Australia and the United States (US) tariff sets], EQ-VAS scores, and the EORTC QLQ-C30. Clinical [Eastern Cooperative Oncology Group (ECOG) performance status] and patient-reported (items 29 and 30 of the EORTC QLQ-C30 and the EQ-VAS) anchors were assessed for appropriateness by their correlation strength. Clinical change groups (CCGs) were defined a priori for improvement and deterioration based on estimates used in previous literature. MIDs were estimated via linear regression and distribution-based methods.ResultsFor the index-based utility scores in Australia, the anchor-defined MID estimates were 0.01 to 0.06 for improvement and - 0.04 to -0.03 for deterioration, with a weighted value of 0.03 for improvement and deterioration. The EQ-VAS MID estimate was 5 points for both improvement and deterioration. For the EORTC QLQ-C30, changes of at least 3.64 (improvement) and - 4.28 (deterioration) units on the physical functioning scale, 6.31 (improvement) and - 7.11 (deterioration) units on the role functioning scale, 4.65 (improvement) and - 3.41 (deterioration) units on the emotional functioning scale, and 5.41 (improvement) and - 5.56 (deterioration) units on the social functioning scale were estimated to be meaningful.ConclusionThis study identified lower MIDs for the EQ-5D-5L utility index, EQ-VAS, and EORTC QLQ-C30 domain scores, than those reported previously. The use of a real-world cancer-specific panel dataset may reflect smaller MID estimates that are more applicable to cancer patients in the clinical practice, rather than using MIDs that have been estimated from clinical trials.

Project description:BackgroundIn oncology, health-related quality of life (HRQoL) data are often collected using disease-specific patient questionnaires while generic, patient-level utility data required for health economic modeling are often not collected.MethodsWe developed a mapping algorithm for multiple myeloma that relates HRQoL scores from the European Organization for Research and Treatment of Cancer (EORTC) questionnaires QLQ-C30 and QLQ-MY20 to a utility value from the European QoL-5 Dimensions (EQ-5D) questionnaire. Data were obtained from 154 multiple myeloma patients who had participated in a multicenter cohort study in the UK or Germany. All three questionnaires were administered at a single time point. Scores from all 19 domains of the QLQ-C30 and QLQ-MY20 instruments were univariately tested against EQ-5D values and retained in a multivariate regression model if statistically significant. A 10-fold cross-validation model selection method was also used as an alternative testing means. Two models were developed: one based on QLQ-C30 plus QLQ-MY20 scores and one based on QLQ-C30 scores alone. Adjusted R-squared, correlation coefficients, and plots of observed versus predicted EQ-5D values were presented for both models.ResultsMapping revealed that Global Health Status/QoL, Physical Functioning, Pain, and Insomnia were significant predictors of EQ-5D utility values. Similar results were observed when QLQ-MY20 scores were excluded from the model, except that Emotional Functioning and became a significant predictor and Insomnia was no longer a significant predictor. Adjusted R-squared values were of similar magnitude with or without inclusion of QLQ-MY20 scores (0.70 and 0.69, respectively), suggesting that the EORTC QLQ-MY20 adds little in terms of predicting utility values in multiple myeloma.ConclusionsThis algorithm successfully mapped EORTC HRQoL data onto EQ-5D utility in patients with multiple myeloma. Current mapping will aid in the analysis of cost-effectiveness of novel therapies for this indication.

Project description:BackgroundParoxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening terminal-complement-mediated disease resulting in intravascular hemolysis and thrombosis with significant morbidity and premature mortality. There exists no disease-specific quality-of-life (QOL) measure for PNH. Its QOL effects resemble those of hematologic cancers, which supports the use of cancer-specific QOL measures in PNH clinical trials. The European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 has published norms for many European and North American countries, but not for Asian countries. We investigated differences by Asian ethnicity in scores and item function on the EORTC QLQ-C30.MethodsThis secondary analysis focused on two non-inferiority PNH trials (301 and 302) comparing eculizumab and ravulizumab (n = 441). Analysis of covariance examined the main effect of Asian ethnicity on baseline EORTC QLQ-C30 scores, after adjusting for propensity scores encompassing trial, demographic and clinical factors. Mixed modeling of longitudinal data compared subscale scores in Asian vs. non-Asian patients, after propensity adjustment. Differential item function (DIF) was examined using ordinal regression models at baseline and longitudinally, to predict item score from total score, ethnicity, and their interaction to test for uniform DIF (significant main effect for Asian) and non-uniform DIF (significant Asian-by-total-score interaction).ResultsOf the 15 baseline domains, Asian patients scored slightly better on role and emotional functioning and slightly worse on constipation and diarrhea (0.22 < Cohen's d < 0.36). In longitudinal models, Asians reported slightly higher appetite loss, diarrhea, and financial difficulties than non-Asians (R2 increment < 0.0005). There was negligible uniform and non-uniform DIF, i.e., R2 0 to 0.018, far below Zumbo's (1999) criterion of 0.13. On average there were larger differences from norms for Asians (mean = 0.05, sd = 0.44) than non-Asians (mean = -0.07, sd = 0.36), but the size and direction of the differences varied considerably by domain, age, and gender.ConclusionWhen compared to norms, Asian patients showed no systematic biases. DIF results supported this finding. We conclude that Asian ethnicity does not impact interpretation of EORTC QLQ-C30 scores.

Project description:BackgroundThe types of outcomes measured collected in clinical studies and those required for cost-effectiveness analysis often differ. Decision makers routinely use quality adjusted life years (QALYs) to compare the benefits and costs of treatments across different diseases and treatments using a common metric. QALYs can be calculated using preference-based measures (PBMs) such as EQ-5D-3L, but clinical studies often focus on objective clinician or laboratory measured outcomes and non-preference-based patient outcomes, such as QLQ-C30. We model the relationship between the generic, preference-based EQ-5D-3L and the cancer specific quality of life questionnaire, QLQ-C30 in patients with breast cancer. This will result in a mapping that allows users to convert QLQ-C30 scores into EQ-5D-3L scores for the purposes of cost-effectiveness analysis or economic evaluation.MethodsWe use data from a randomized trial of 602 patients with HER2-positive advanced breast cancer provided 3766 EQ-5D-3L observations. Direct mapping using adjusted, limited dependent variable mixture models (ALDVMM) is compared to a random effects linear regression and indirect mapping using seemingly unrelated ordered probit models. EQ-5D-3L was estimated as a function of the summary scales of the QLQ-C30 and other patient characteristics.ResultsA four component mixture model outperformed other models in terms of summary fit statistics. A close fit to the observed data was observed across the range of disease severity. Simulated data from the model closely aligned to the original data and showed that mapping did not significantly underestimate uncertainty. In the simulated data, 22.15% were equal to 1 compared to 21.93% in the original data. Variance was 0.0628 in the simulated data versus 0.0693 in the original data. The preferred mapping is provided in Excel and Stata files for the ease of users.ConclusionA four component adjusted mixture model provides reliable, non-biased estimates of EQ-5D-3L from the QLQ-C30, to link clinical studies to economic evaluation of health technologies for breast cancer. This work adds to a growing body of literature demonstrating the appropriateness of mixture model based approaches in mapping.

Project description:BackgroundCost-effectiveness models require quality of life utilities calculated from generic preference-based questionnaires, such as EQ-5D. We evaluated the performance of available algorithms for QLQ-C30 conversion into EQ-5D-3L based utilities in a metastatic colorectal cancer (mCRC) patient population and subsequently developed a mCRC specific algorithm. Influence of mapping on cost-effectiveness was evaluated.MethodsThree available algorithms were compared with observed utilities from the CAIRO3 study. Six models were developed using 5-fold cross-validation: predicting EQ-5D-3L tariffs from QLQ-C30 functional scale scores, continuous QLQ-C30 scores or dummy levels with a random effects model (RE), a most likely probability method on EQ-5D-3L functional scale scores, a beta regression model on QLQ-C30 functional scale scores and a separate equations subgroup approach on QLQ-C30 functional scale scores. Performance was assessed, and algorithms were tested on incomplete QLQ-C30 questionnaires. Influence of utility mapping on incremental cost/QALY gained (ICER) was evaluated in an existing Dutch mCRC cost-effectiveness model.ResultsThe available algorithms yielded mean utilities of 1: 0.87 ± sd:0.14,2: 0.81 ± 0.15 (both Dutch tariff) and 3: 0.81 ± sd:0.19. Algorithm 1 and 3 were significantly different from the mean observed utility (0.83 ± 0.17 with Dutch tariff, 0.80 ± 0.20 with U.K. tariff). All new models yielded predicted utilities drawing close to observed utilities; differences were not statistically significant. The existing algorithms resulted in an ICER difference of €10,140 less and €1765 more compared to the observed EQ-5D-3L based ICER (€168,048). The preferred newly developed algorithm was €5094 higher than the observed EQ-5D-3L based ICER. Disparity was explained by minimal diffences in incremental QALYs between models.ConclusionAvailable mapping algorithms sufficiently accurately predict utilities. With the commonly used statistical methods, we did not succeed in developping an improved mapping algorithm. Importantly, cost-effectiveness outcomes in this study were comparable to the original model outcomes between different mapping algorithms. Therefore, mapping can be an adequate solution for cost-effectiveness studies using either a previously designed and validated algorithm or an algorithm developed in this study.

Dataset Information

Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France.

Publications

Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France.

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