Enhancing the Content of the EORTC QLQ-C30 to Maximize the Detection of Treatment Benefit in Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, and Low-Blast Acute Myeloid Leukemia Using the EORTC Item Bank

INTRODUCTION

Myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and acute myelogenous leukemia (AML) are rare hematological stem cell disorders resulting in cytopenias, disease-related complications, and substantial impact on health-related quality of life (HRQOL). A literature review, clinician interviews, and qualitative interviews with high risk (HR) MDS, CMML, and low blast (LB) AML patients confirmed the relevance of concepts assessed by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) for measuring HRQL, but also suggested that concepts important to evaluate the complete experience of patients with these conditions were missing, or only addressed in a limited fashion.

The present study involved testing supplemental items from the EORTC item bank with patients to confirm item relevance and aimed to enhance the content of the EORTC QLQ-C30 to detect treatment benefit in patients with HR MDS, CMML and LB AML.

METHODS

Supplemental items were selected from the EORTC item bank to address the conceptual gaps of the core EORTC QLQ-C30 instrument in this patient population. Items were selected based on concepts drawn from the literature review and prior qualitative work with patients. After ethical approval from an independent review board, HR MDS, CMML, and LB AML patients were identified through clinical sites and the MDS Foundation and screened for eligibility. Using a semi-structured interview guide, 45-minute cognitive debriefing interviews (CDIs) were conducted with each patient to evaluate item understanding, response option clarity, and item relevance. Audio-recorded interviews were transcribed, coded, and analyzed. Patient responses to the EORTC QLQ-C30 and supplemental items were collected for quantitative analysis (i.e., item level endorsement frequencies). An interview with a hematologist provided additional clinical perspective on the results.

RESULTS

CDIs were performed with 18 patients (mean age 68 (±10.1); 56% female; n=14 HR MDS, n=3 CMML, and n=1 LB AML). Most patients were treated with azacitidine (n=12; 67%), had an ECOG status of 1 (n=6, 33%) or a patient-rated ECOG of 2 (n=6, 33%), and were diagnosed within the last 12 months (n=10, 56%).

To address conceptual gaps identified in our initial qualitative work, 9 supplemental symptom items (bone pain, weakness, feeling slowed down, easily tired, lacking energy, bruising/petechiae, dizziness, and shortness of breath [2 items]) and 4 supplemental impact items (limitations with climbing stairs or getting up out of a chair, travel limitations, doing housework, and shopping or running errands) were selected from the EORTC item bank. These items were presented to patients for additional testing. Supplemental items were generally well understood and considered relevant, with two exceptions. Bone pain was often attributed to injury or arthritis rather than MDS, CMML, or AML or its treatments; travel limitations lacked clarity as patients were unsure what type of travel to consider (i.e., car travel vs. air travel). Item level endorsement frequencies showed good distribution across most response options without skewness, indicating that most supplemental items assessed concepts relevant to this sample (Table 1). Clinician feedback confirmed the relevance of most supplemental items and reinforced that the bone pain and travel limitation items were problematic. Based on the qualitative and quantitative analysis of the CDIs, the bone pain and travel limitation supplemental items were removed (Table 2). A total of 11 supplemental items were retained to enhance the content of EORTC QLQ-C30 instrument to better capture treatment benefit from the patient perspective.

CONCLUSIONS

Our study suggests that engagement with patients to address conceptual gaps in EORTC QLQ-C30 coverage provided valuable information regarding supplemental items from the EORTC item bank that could potentially provide more clinically relevant and better targeted measurement of HRQOL in HR MDS, CMML, and LB AML patients, thus improving the detection of treatment benefit. To confirm this, the EORTC QLQ-C30 and the proposed supplemental items should be tested in a larger clinically-defined sample to evaluate their combined measurement properties in these patient populations.

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