How scoring limits the usability of minimal important differences (MIDs) as responder definition (RD): an exemplary demonstration using EORTC QLQ-C30 subscales.
EORTC QLQ-C30
Meaningful change
Responder definition
State change
Journal
Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation
ISSN: 1573-2649
Titre abrégé: Qual Life Res
Pays: Netherlands
ID NLM: 9210257
Informations de publication
Date de publication:
May 2023
May 2023
Historique:
accepted:
18
06
2022
medline:
25
4
2023
pubmed:
10
7
2022
entrez:
9
7
2022
Statut:
ppublish
Résumé
The recommended method for establishing a meaningful threshold for individual changes in patient-reported outcome (PRO) scores over time uses an anchor-based method. The patients assess their perceived level of change and this is used to define a threshold on the PRO score which may be considered meaningful to the patient. In practice, such an anchor may not be available. In the absence of alternative information often the meaningful change threshold for assessing between-group differences, the minimally important difference, is used to define meaningful change at the individual level too. This paper will highlight the issues with this, especially where the underlying measurement scale is not continuous. Using the EORTC QLQ-C30 as an example, plausible score increments ("state changes") are calculated for each subscale highlighting why commonly used thresholds may be misleading, including leading to sensitivity analyses that are inadvertently testing the same underlying threshold. The minimal possible individual score change varies across subscales; 6.7 for Physical Functioning, 8.3 for Global Health Scale and Emotional Functioning, 11.1 for fatigue, 16.7 for role functioning, cognitive functioning, social functioning, nausea and vomiting, pain and 33.3 for single items. The determination of meaningful change for an individual patient requires input from the patients but being mindful of the underlying scale ensures that these thresholds are also guided by what is a plausible change for patients to achieve on the scale.
Identifiants
pubmed: 35809136
doi: 10.1007/s11136-022-03181-4
pii: 10.1007/s11136-022-03181-4
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1247-1253Informations de copyright
© 2022. The Author(s), under exclusive licence to Springer Nature Switzerland AG.
Références
Food and Durg Administration. (2009). Guidance for industry: Patient-reported outcome measures: use in medical product development to support labeling claims. Federal Register, 74(235), 65132–65133.
Anota, A., Hamidou, Z., Paget-Bailly, S., et al. (2015). Time to health-related quality of life score deterioration as a modality of longitudinal analysis for health-related quality of life studies in oncology: Do we need RECIST for quality of life to achieve standardization? Quality of Life Research, 24(1), 5–18.
doi: 10.1007/s11136-013-0583-6
pubmed: 24277234
Jaeschke, R., Singer, J., & Guyatt, G. H. (1989). Measurement of health status: Ascertaining the minimal clinically important difference. Controlled Clinical Trials, 10(4), 407–415.
doi: 10.1016/0197-2456(89)90005-6
pubmed: 2691207
Wyrwich, K. W., Tierney, W. M., Babu, A. N., Kroenke, K., & Wolinsky, F. D. (2005). A comparison of clinically important differences in health-related quality of life for patients with chronic lung disease, asthma, or heart disease. Health Services Research, 40(2), 577–592.
doi: 10.1111/j.1475-6773.2005.0l374.x
pubmed: 15762908
pmcid: 1361158
Beckerman, H., Roebroeck, M. E., Lankhorst, G. J., Becher, J. G., Bezemer, P. D., & Verbeek, A. L. (2001). Smallest real difference, a link between reproducibility and responsiveness. Quality of Life Research., 10(7), 571–578.
doi: 10.1023/A:1013138911638
pubmed: 11822790
Aaronson, N. K., Ahmedzai, S., Bergman, B., et al. (1993). The European Organization for Research and Treatment of Cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. Journal of the National Cancer Institute., 85(5), 365–376.
doi: 10.1093/jnci/85.5.365
pubmed: 8433390
Fayers, P., Aaronson, N., Bjordal, K., Groenvold, M., Curran, D., & Bottomley, A. (2001). The EORTC QLQ-C30 scoring manual (3rd ed.). European Organization for Research and Treatment of Cancer.
Lenz, H.-J., Argiles, G., Yoshino, T., et al. (2019). Health-related quality of life in the Phase III LUME-Colon 1 study: Comparison and interpretation of results from EORTC QLQ-C30 analyses. Clinical Colorectal Cancer, 18, 269–279.
doi: 10.1016/j.clcc.2019.08.005
pubmed: 31628043
pmcid: 7505163
Anota, A., Mouillet, G., Trouilloud, I., et al. (2015). Sequential FOLFIRI 3+ Gemcitabine improves health-related quality of life deterioration-free survival of patients with metastatic pancreatic adenocarcinoma: a randomized phase II trial. PLoS ONE, 10(5), e0125350.
doi: 10.1371/journal.pone.0125350
pubmed: 26010884
pmcid: 4444351
Stockler, M. R., Hilpert, F., Friedlander, M., et al. (2014). Patient-reported outcome results from the open-label phase III AURELIA trial evaluating bevacizumab-containing therapy for platinum-resistant ovarian cancer. Journal of Clinical Oncology., 32(13), 1309.
doi: 10.1200/JCO.2013.51.4240
pubmed: 24687829
pmcid: 4876313
Harrison, C. N., Mesa, R. A., Kiladjian, J. J., et al. (2013). Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy. British Journal of Haematology, 162(2), 229–239.
doi: 10.1111/bjh.12375
pubmed: 23672349
Bonnetain, F., Dahan, L., Maillard, E., et al. (2010). Time until definitive quality of life score deterioration as a means of longitudinal analysis for treatment trials in patients with metastatic pancreatic adenocarcinoma. European Journal of Cancer., 46(15), 2753–2762.
doi: 10.1016/j.ejca.2010.07.023
pubmed: 20724140
Eisenhardt, A., Schneider, T., Scheithe, K., Colling, C., & Heidenreich, A. (2015). Health-related quality of life in patients with advanced prostate cancer undergoing treatment with TRIPTOrelin Pamoate SIX month formulation: Results of the non-interventional TRIPTOSIX study. Journal of Clinical Oncology, 33, 287.
doi: 10.1200/jco.2015.33.7_suppl.287
Osoba, D., Bezjak, A., Brundage, M., et al. (2005). Analysis and interpretation of health-related quality-of-life data from clinical trials: Basic approach of The National Cancer Institute of Canada Clinical Trials Group. European Journal of Cancer., 41(2), 280–287.
doi: 10.1016/j.ejca.2004.10.017
pubmed: 15661554
Osoba, D., Bezjak, A., Brundage, M., Pater, J., National Cancer Institute of Canada Clinical Trials Group. (2007). Evaluating health-related quality of life in cancer clinical trials: The National Cancer Institute of Canada Clinical Trials Group experience. Value Health., 10, S138–S145.
doi: 10.1111/j.1524-4733.2007.00278.x
pubmed: 17995472
Brundage, M., Osoba, D., Bezjak, A., Tu, D., Palmer, M., & Pater, J. (2007). Lessons learned in the assessment of health-related quality of life: Selected examples from the National Cancer Institute of Canada Clinical Trials Group. Journal of Clinical Oncology., 25(32), 5078–5081.
doi: 10.1200/JCO.2007.11.4645
pubmed: 17991924
Kawahara, T., Shimozuma, K., Shiroiwa, T., et al. (2018). Patient-reported outcome results from the open-label randomized phase III select bc trial evaluating first-line s-1 therapy for metastatic breast cancer. Oncology, 94(2), 107–115.
doi: 10.1159/000484142
pubmed: 29145211
Cocks, K., King, M. T., Velikova, G., Martyn St-James, M., Fayers, P. M., & Brown, J. M. (2011). Evidence-based guidelines for determination of sample size and interpretation of the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30. Journal of Clinical Oncology., 29(1), 89–96.
doi: 10.1200/JCO.2010.28.0107
pubmed: 21098316
SF-36 scoring. Retrieved from https://www.rand.org/health-care/surveys_tools/mos/36-item-short-form/scoring.html .
Sully, K., Trigg, A., Bonner, N., Moreno-Koehler, A., Trennery, C., Shah, N., Yucel, E., Panjabi, S., & Cocks, K. (2019). Estimation of minimally important differences and responder definitions for EORTC QLQ-MY20 scores in multiple myeloma patients. European Journal of Haematology, 103(5), 500–509. https://doi.org/10.1111/ejh.13316
doi: 10.1111/ejh.13316
pubmed: 31444815
pmcid: 6852250