Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms
BackgroundMyelodysplastic neoplasms (MDS) are characterized by ineffective hematopoiesis, peripheral blood cytopenias, and an increased risk of progression to acute myeloid leukemia. One of the main treatment goals is improving quality of life (QoL), particularly for patients with lower-risk MDS (LR...
Saved in:
| Main Authors: | , , , , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2025-02-01
|
| Series: | Frontiers in Oncology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fonc.2025.1507854/full |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1825206692700225536 |
|---|---|
| author | Esther Natalie Oliva Shien Guo Jennifer Lord-Bessen Aylin Yucel Roberto Latagliata Massimo Breccia Giuseppe A. Palumbo Grazia Sanpaolo Marta Riva Valeria Santini Uwe Platzbecker Guillermo Garcia-Manero Pierre Fenaux Christopher G. Pelligra |
| author_facet | Esther Natalie Oliva Shien Guo Jennifer Lord-Bessen Aylin Yucel Roberto Latagliata Massimo Breccia Giuseppe A. Palumbo Grazia Sanpaolo Marta Riva Valeria Santini Uwe Platzbecker Guillermo Garcia-Manero Pierre Fenaux Christopher G. Pelligra |
| author_sort | Esther Natalie Oliva |
| collection | DOAJ |
| description | BackgroundMyelodysplastic neoplasms (MDS) are characterized by ineffective hematopoiesis, peripheral blood cytopenias, and an increased risk of progression to acute myeloid leukemia. One of the main treatment goals is improving quality of life (QoL), particularly for patients with lower-risk MDS (LR-MDS) who may live longer with compromised QoL. The QOL-E© is a patient-reported outcome (PRO) measure specifically developed to address the lack of a health-related QoL questionnaire for patients with MDS. The objective of this study was to evaluate the psychometric performance of the QOL-E in patients with LR-MDS.MethodsData from four clinical trials in MDS (MEDALIST, DARB-MDS, EQoL-MDS, and RevMDS trials) were used to assess construct validity, reliability, and responsiveness. The QOL-E was validated by the European Organization for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire – Core 30 (QLQ-C30) and clinical outcomes. It contains 29 items with the first two items assessing the patient’s general well-being and the 27 remaining items grouped into six domain scores: physical well-being (QOL-FIS), functional well-being (QOL-FUN), social/family well-being (QOL-SOC), sexual well-being (QOL-SEX), fatigue (QOL-FAT), and MDS-specific disturbances (QOL-MDSS). Additionally, meaningful within-patient change (MWPC) thresholds were determined for the domains and summary scores of the QOL-E using anchor-based analyses, supported by distribution-based analyses.ResultsA total of 458 patients were included in the analyses. The QOL-E domain/summary scores demonstrated acceptable convergent/divergent and known-groups validity. Test-retest reliability and internal consistency was confirmed with intraclass correlation coefficients and Cronbach alpha exceeding 0.70 across most QOL-E domains/summary scores. The QOL-E domains/summary scores, except for QOL-SEX, had an adequate ability to detect change from baseline to Week 24. MWPC thresholds were proposed for all other domains and summary scores.ConclusionThe study results demonstrate that the QOL-E is generally fit for purpose to assess treatment effects in populations with LR-MDS and the proposed MWPC thresholds can be used to assess within-patient treatment effect on PROs, as assessed by the QOL-E, in future studies. |
| format | Article |
| id | doaj-art-18cd17e1a9a34359a5be412d7e79bb2b |
| institution | Kabale University |
| issn | 2234-943X |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Oncology |
| spelling | doaj-art-18cd17e1a9a34359a5be412d7e79bb2b2025-02-07T06:49:30ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2025-02-011510.3389/fonc.2025.15078541507854Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasmsEsther Natalie Oliva0Shien Guo1Jennifer Lord-Bessen2Aylin Yucel3Roberto Latagliata4Massimo Breccia5Giuseppe A. Palumbo6Grazia Sanpaolo7Marta Riva8Valeria Santini9Uwe Platzbecker10Guillermo Garcia-Manero11Pierre Fenaux12Christopher G. Pelligra13Hematology Unit, Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, ItalyEvidera, Waltham, MA, United StatesBristol Myers Squibb, Lawrence, NJ, United StatesBristol Myers Squibb, Lawrence, NJ, United StatesHematology Unit, Belcolle Hospital, Viterbo, ItalyHematology, Department of Translational and Precision Medicine, Az. Policlinico Umberto I-Sapienza University, Rome, ItalyDepartment of Medical, Surgical Sciences and Advanced Technologies “GF Ingrassia”, University of Catania, Catania, ItalyDepartment of Hematology and Stem Cell Transplantation Unit, IRCCS Casa Sollievo della Sofferenza Hospital, San Giovanni Rotondo, ItalyDepartment of Hematology and Oncology, Niguarda Cancer Center, ASST Grande Ospedale Metropolitano Niguarda, Milan, ItalyMDS Unit, Hematology, DMSC, University of Florence, AOUC, Florence, Italy0Medical Clinic and Policlinic 1, Hematology and Cellular Therapy, University Hospital Leipzig, Leipzig, Germany1Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX, United States2Service d’Hématologie Séniors, Hôpital Saint-Louis, Université Paris 7, Paris, France3Evidera, Atlanta, GA, United StatesBackgroundMyelodysplastic neoplasms (MDS) are characterized by ineffective hematopoiesis, peripheral blood cytopenias, and an increased risk of progression to acute myeloid leukemia. One of the main treatment goals is improving quality of life (QoL), particularly for patients with lower-risk MDS (LR-MDS) who may live longer with compromised QoL. The QOL-E© is a patient-reported outcome (PRO) measure specifically developed to address the lack of a health-related QoL questionnaire for patients with MDS. The objective of this study was to evaluate the psychometric performance of the QOL-E in patients with LR-MDS.MethodsData from four clinical trials in MDS (MEDALIST, DARB-MDS, EQoL-MDS, and RevMDS trials) were used to assess construct validity, reliability, and responsiveness. The QOL-E was validated by the European Organization for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire – Core 30 (QLQ-C30) and clinical outcomes. It contains 29 items with the first two items assessing the patient’s general well-being and the 27 remaining items grouped into six domain scores: physical well-being (QOL-FIS), functional well-being (QOL-FUN), social/family well-being (QOL-SOC), sexual well-being (QOL-SEX), fatigue (QOL-FAT), and MDS-specific disturbances (QOL-MDSS). Additionally, meaningful within-patient change (MWPC) thresholds were determined for the domains and summary scores of the QOL-E using anchor-based analyses, supported by distribution-based analyses.ResultsA total of 458 patients were included in the analyses. The QOL-E domain/summary scores demonstrated acceptable convergent/divergent and known-groups validity. Test-retest reliability and internal consistency was confirmed with intraclass correlation coefficients and Cronbach alpha exceeding 0.70 across most QOL-E domains/summary scores. The QOL-E domains/summary scores, except for QOL-SEX, had an adequate ability to detect change from baseline to Week 24. MWPC thresholds were proposed for all other domains and summary scores.ConclusionThe study results demonstrate that the QOL-E is generally fit for purpose to assess treatment effects in populations with LR-MDS and the proposed MWPC thresholds can be used to assess within-patient treatment effect on PROs, as assessed by the QOL-E, in future studies.https://www.frontiersin.org/articles/10.3389/fonc.2025.1507854/fullhealth-related quality of lifemyelodysplastic syndromesmyelodysplastic neoplasmspatient-reported outcomespsychometric analysisQOL-E |
| spellingShingle | Esther Natalie Oliva Shien Guo Jennifer Lord-Bessen Aylin Yucel Roberto Latagliata Massimo Breccia Giuseppe A. Palumbo Grazia Sanpaolo Marta Riva Valeria Santini Uwe Platzbecker Guillermo Garcia-Manero Pierre Fenaux Christopher G. Pelligra Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms Frontiers in Oncology health-related quality of life myelodysplastic syndromes myelodysplastic neoplasms patient-reported outcomes psychometric analysis QOL-E |
| title | Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms |
| title_full | Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms |
| title_fullStr | Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms |
| title_full_unstemmed | Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms |
| title_short | Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms |
| title_sort | psychometric properties and meaningful change thresholds for the qol e instrument in patients with myelodysplastic neoplasms |
| topic | health-related quality of life myelodysplastic syndromes myelodysplastic neoplasms patient-reported outcomes psychometric analysis QOL-E |
| url | https://www.frontiersin.org/articles/10.3389/fonc.2025.1507854/full |
| work_keys_str_mv | AT esthernatalieoliva psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT shienguo psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT jenniferlordbessen psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT aylinyucel psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT robertolatagliata psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT massimobreccia psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT giuseppeapalumbo psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT graziasanpaolo psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT martariva psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT valeriasantini psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT uweplatzbecker psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT guillermogarciamanero psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT pierrefenaux psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms AT christophergpelligra psychometricpropertiesandmeaningfulchangethresholdsfortheqoleinstrumentinpatientswithmyelodysplasticneoplasms |