Measuring patient-reported physical functioning and fatigue in myelodysplastic syndromes using a modular approach based on EORTC QLQ-C30

Farrah Pompilus,Rafael Bejar,Jill A Bell,Antoine Regnault,Robert J Fram,Flora Mazerolle,Douglas V Faller,Patrick Marquis,Anna Ciesluk

doi:10.1186/s41687-021-00334-w

Abstract

PurposePhysical functioning and fatigue are key patient concerns in myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML). The objective of this research was to generate supportive quantitative evidence for modular physical functioning and fatigue measures based on the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 items (QLQ-C30) and a customized selection of 10 supplemental items from the EORTC Item Library.MethodsThe 40 items were completed online cross-sectionally by 51 patients (higher risk [HR] MDS: 53%; CMML: 26%; AML: 10%). Psychometric analyses based on Rasch measurement theory (RMT) were conducted on the QLQ-C30 physical functioning and fatigue domains as well as measures combining QLQ-C30 and supplemental items. A measure of anemia-related symptoms composed of QLQ-C30 and supplemental items covering fatigue, dyspnea, and dizziness was also investigated.ResultsThe QLQ-C30 physical functioning and fatigue domains showed good targeting to the sample and adequate reliability, with few conceptual gaps identified. Combining the QLQ-C30 and supplemental physical functioning and fatigue items improved the conceptual coverage and the reliability of the measures. The patient-reported anemia-related symptom measure showed good measurement performance, underpinned by a clinically meaningful characterization of severity of these symptoms over a spectrum, starting with fatigue, then dyspnea, and finally dizziness (most severe).ConclusionThe modular measurement approach of combining EORTC QLQ-C30 and Item Library offers a promising pragmatic solution to the measurement of physical functioning and fatigue, as well as anemia-related symptoms in clinical trials conducted in HR MDS, CMML, and AML.

Highlights

Myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML) are rare blood cancers that affect the myeloid cells
Patient demographics A total of 51 patients participated in the online survey, with most patients diagnosed with higher risk (HR) MDS (HR MDS: 53%; CMML: 26%; AML: 10%)
All patients confirmed a diagnosis of HR MDS, CMML, and AML to be included in the study

Summary

Introduction

Myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML) are rare blood cancers that affect the myeloid cells. Three core PRO concepts have been identified for oncology trials and should be measured to integrate the experience of patients and to demonstrate the benefit of new treatments as well as inform decision making: physical functioning, disease-related symptoms, and symptomatic adverse events [4]. The measurement of these core PRO concepts in oncology trials is increasingly performed using a modular approach. In a modular measurement approach, only the key concepts for a specific context of use are carefully selected and measured in the study, using items that are thoughtfully selected from existing static questionnaires and item banks or libraries [4, 5]. This approach allows a bespoke and efficient measurement of the concepts that are meaningful to patients

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