ArticleJournal of patient-reported outcomes2019
A pragmatic patient-reported outcome strategy for rare disease clinical trials: application of the EORTC item library to myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia.
Article in Journal of patient-reported outcomes, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
13 citing papers in PubMed.
- Patient-centred and fit-for-purpose clinical trials in rare cancers: lessons from sarcomas for operationalising 2024 WHO Guidance and ICH E6(R3).EClinicalMedicine · 2026Review
- Mapping the EORTC item library to the World Health Organization international classification of functioning, disability and health: a content analysis.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026Article
- Understanding data visualization techniques in qualitative studies used to develop and validate patient-reported outcome measures: a targeted literature review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Review
- Mixed-methods research to support the use of new lymphoma-specific patient-reported symptom measures derived from the EORTC item library.Journal of patient-reported outcomes · 2024Review
- [Application of patient-reported-outcome in myelodysplastic syndromes].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2022Article
- Measuring the Patient Experience in Rare Disorders: Benefit of Pragmatic Mixed-Methods Research in NUT Carcinoma.Oncology and therapy · 2022Article
- Consideration of quality of life in the health technology assessments of rare disease treatments.The European journal of health economics : HEPAC : health economics in prevention and care · 2022Article
- Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists.Blood advances · 2022Article
- The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment.The patient · 2021Review
- Measuring patient-reported physical functioning and fatigue in myelodysplastic syndromes using a modular approach based on EORTC QLQ-C30.Journal of patient-reported outcomes · 2021Article
- Health-Related Quality of Life Assessment in Patients with Myelodysplastic Syndromes: Evidence from Randomized Clinical Trials.Clinical practice and epidemiology in mental health : CP & EMH · 2021Review
- Developing Symptom Lists for People with Cancer Treated with Targeted Therapies.Targeted oncology · 2021Article
- Article
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundNovel, pragmatic, patient-centered strategies are needed to ensure fit-for-purpose patient-reported outcomes (PRO) instruments in clinical trial research for rare diseases such as myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML). The objective of the current study was to select supplemental items to add to the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life-Core 30 (QLQ-C30) to ensure content coverage of all important clinical concepts in patients with higher-risk (HR) MDS, low-blast count (LB) AML, and CMML, thus, improving the instrument's ability to detect clinically meaningful treatment benefit for this context of use.
methodsOur mixed methods approach comprised literature review, clinician consultation (n = 3), and qualitative and quantitative analysis of two stages of patient interview data (n = 14, n = 18) to select library bank items to supplement a generic cancer PRO, the EORTC QLQ-C30.
resultsUnique symptom (n = 54) and impact (n = 72) concepts were organized into conceptual frameworks of treatment benefit, compared with EORTC QLQ-C30 items and conceptual gaps identified. Supplemental items (n = 13) addressing those gaps were selected from the EORTC Item Library and tested with patients. Supplemental item endorsement frequencies met World Health Organization Quality of Life criteria, suggesting good targeting and relevance for this sample. However, three supplemental items were confirmed as problematic based upon cognitive debriefing results, and expert clinical consultations. Ultimately, 10 supplemental items (n = 7 symptom; n = 3 impact) were selected for the MDS/AML/CMML context.
conclusionSupplemental items were selected to enhance the conceptual coverage of the EORTC QLQ-C30 in the areas of fatigue, shortness of breath, and functioning.
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