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
Abstract Background Novel, 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 (...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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SpringerOpen
2019-06-01
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| Series: | Journal of Patient-Reported Outcomes |
| Subjects: | |
| Online Access: | http://link.springer.com/article/10.1186/s41687-019-0123-4 |
| _version_ | 1818067503785443328 |
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| author | Jill A. Bell Aaron Galaznik Farrah Pompilus Sara Strzok Rafael Bejar Fatima Scipione Robert J. Fram Douglas V. Faller Stefan Cano Patrick Marquis |
| author_facet | Jill A. Bell Aaron Galaznik Farrah Pompilus Sara Strzok Rafael Bejar Fatima Scipione Robert J. Fram Douglas V. Faller Stefan Cano Patrick Marquis |
| author_sort | Jill A. Bell |
| collection | DOAJ |
| description | Abstract Background Novel, 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. Methods Our 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. Results Unique 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. Conclusion Supplemental items were selected to enhance the conceptual coverage of the EORTC QLQ-C30 in the areas of fatigue, shortness of breath, and functioning. |
| first_indexed | 2024-12-10T15:24:43Z |
| format | Article |
| id | doaj.art-5c00d5c9314c4874932bf05918b400b6 |
| institution | Directory Open Access Journal |
| issn | 2509-8020 |
| language | English |
| last_indexed | 2024-12-10T15:24:43Z |
| publishDate | 2019-06-01 |
| publisher | SpringerOpen |
| record_format | Article |
| series | Journal of Patient-Reported Outcomes |
| spelling | doaj.art-5c00d5c9314c4874932bf05918b400b62022-12-22T01:43:35ZengSpringerOpenJournal of Patient-Reported Outcomes2509-80202019-06-013111210.1186/s41687-019-0123-4A 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 leukemiaJill A. Bell0Aaron Galaznik1Farrah Pompilus2Sara Strzok3Rafael Bejar4Fatima Scipione5Robert J. Fram6Douglas V. Faller7Stefan Cano8Patrick Marquis9Millennium Pharmaceuticals, Inc., (a wholly owned subsidiary of Takeda Pharmaceutical Company Limited)Millennium Pharmaceuticals, Inc., (a wholly owned subsidiary of Takeda Pharmaceutical Company Limited)Modus OutcomesModus OutcomesUC San Diego Moores Cancer Center – MDS Center of ExcellenceMillennium Pharmaceuticals, Inc., (a wholly owned subsidiary of Takeda Pharmaceutical Company Limited)Millennium Pharmaceuticals, Inc., (a wholly owned subsidiary of Takeda Pharmaceutical Company Limited)Millennium Pharmaceuticals, Inc., (a wholly owned subsidiary of Takeda Pharmaceutical Company Limited)Modus OutcomesModus OutcomesAbstract Background Novel, 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. Methods Our 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. Results Unique 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. Conclusion Supplemental items were selected to enhance the conceptual coverage of the EORTC QLQ-C30 in the areas of fatigue, shortness of breath, and functioning.http://link.springer.com/article/10.1186/s41687-019-0123-4Patient-reported outcomesQuality of lifeMyelodysplastic syndromesAcute myeloid leukemiaChronic myelomonocytic leukemia |
| spellingShingle | Jill A. Bell Aaron Galaznik Farrah Pompilus Sara Strzok Rafael Bejar Fatima Scipione Robert J. Fram Douglas V. Faller Stefan Cano Patrick Marquis 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 Journal of Patient-Reported Outcomes Patient-reported outcomes Quality of life Myelodysplastic syndromes Acute myeloid leukemia Chronic myelomonocytic leukemia |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| topic | Patient-reported outcomes Quality of life Myelodysplastic syndromes Acute myeloid leukemia Chronic myelomonocytic leukemia |
| url | http://link.springer.com/article/10.1186/s41687-019-0123-4 |
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