Estimation of minimally important differences and responder definitions for EORTC QLQ‐MY20 scores in multiple myeloma patients

Abstract

Objectives: Thresholds for the minimally important difference (MID) or responder definition (RD) in health-related quality-of-life (HRQoL) scores are required to interpret the impact of an intervention or change in the trajectory of the condition which is meaningful to patients. This study aimed to establish MID and RD for the European Organisation for Research and Treatment of Cancer Quality of Life Multiple Myeloma questionnaire (EORTC QLQ-MY20).Methods: A novel mixed-methods approach was applied by utilizing both existing clinical trial data and prospective patient interviews. Anchor-based, distributionbased, and qualitative-based estimates of meaningful change were triangulated to form recommended RDs for each scale of the EORTC QLQ-MY20. Anchor-based MIDs were summarized using weighted correlation.Results: Recommended MIDs were as follows: Disease Symptoms (DS 10 points), Side Effects of Treatment (SE 10 points), Body Image (BI 13 points), and Future Perspective (FP 9 points). Recommended RDs were as follows: DS (16 improvement; 11 worsening), SE (6 improvement; 9 worsening), BI (33 improvement; 33 worsening), and FP (11 improvement; 11 worsening). Conclusions:The study generated estimates of the MID and RD for each scale of the EORTC QLQ-MY20. Published estimates will enable investigators and clinicians to adopt these as standard for interpretation and for hypothesis testing. Consequently, analyses from trials of different interventions can be more comparable. K E Y W O R D SEORTC QLQ-MY20, interpretation guidelines, meaningful change, minimally important difference, mixed methods, multiple myeloma, qualitative interviews, quality of life, responder definition Kate Sully and Andrew Trigg are contributed equally to this work.This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.

Real-World Evaluation of Health-Related Quality of Life in Patients With Multiple Myeloma From Germany

“…Nonetheless, many of the subgroup differences seen on the EORTC QLQ-C30 questionnaire approached or exceeded the ≥6 points considered to represent clinically meaningful differences. [30][31][32] Likewise, some of the changes observed on the QLQ-MY20 tool also exceeded those proposed to represent clinically meaningful differences (10 points for Disease Symptoms and Side Effects of Treatment, 13 points for Body Image, and 9 points for Future Perspectives). 32 Our data highlight the considerable negative impact of MM on patient HRQoL.…”

“…[30][31][32] Likewise, some of the changes observed on the QLQ-MY20 tool also exceeded those proposed to represent clinically meaningful differences (10 points for Disease Symptoms and Side Effects of Treatment, 13 points for Body Image, and 9 points for Future Perspectives). 32 Our data highlight the considerable negative impact of MM on patient HRQoL. A recent global study including 15,386 people from the general population reported normative HRQoL data for the EORTC QLQ-C30 questionnaire; 33 mean (SD) GHS/QoL scores were 66.1 (21.7) overall and 67.0 (21.8) for German participants.…”

Meaningful improvement thresholds in measures of pain and quality of life in oral lichen planus

“…However, the present study adopted a triangulation process, which has been recently recommended by a group of authors, to establish recommended thresholds for further references (Coon & Cappelleri, 2016). It was often observed that the magnitude of within‐patient change (MIC) was generally greater than that of between‐group difference (MID) (Sully et al., 2019). The present results, however, showed that the values of MIC and MID of studied measures are relatively comparable.…”

PERCEPT myeloma: a protocol for a pilot randomised controlled trial of exercise prehabilitation before and during autologous stem cell transplantation in patients with multiple myeloma

“…This study will provide estimates of means/SD for the intended outcomes of a fully powered RCT, to support a sample size calculation for a larger trial. Meaningful clinically important differences are known for a number of the secondary end points, including the FACIT-F,44 EORTC-QLQ-30 and MY20 module45 46 and 6MWT47 and will be used to inform future sample size calculations. For a pilot study, including >30 participants in each arm is considered to be an acceptable number 48 49…”