Evaluation of Minimal Important Difference and Responder Definition in the EORTC QLQ-PAN26 Module for Assessing Health-Related Quality of Life in Patients with Surgically Resected Pancreatic Adenocarcinoma.
Journal
Annals of surgical oncology
ISSN: 1534-4681
Titre abrégé: Ann Surg Oncol
Pays: United States
ID NLM: 9420840
Informations de publication
Date de publication:
Nov 2021
Nov 2021
Historique:
received:
13
10
2020
accepted:
16
02
2021
pubmed:
5
4
2021
medline:
21
10
2021
entrez:
4
4
2021
Statut:
ppublish
Résumé
Although the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-PAN26 is widely used to assess health-related quality of life (HRQoL), its group-level minimal important difference (MID) and individual-level responder definition (RD) are not established; we calculated MID and RD using HRQoL data from the APACT trial in patients with surgically resected pancreatic cancer who received adjuvant chemotherapy. HRQoL was assessed using EORTC QLQ-C30 and QLQ-PAN26 at baseline, during treatment, at end of treatment, and during follow-up. Distribution-based MIDs were estimated using 0.5 × baseline standard deviation (SD) and reliability-based (intraclass correlation) standard error of measurement (SEM). Anchor-based MIDs and RDs (anchor, QLQ-C30 overall health) were estimated using a linear mixed model. Overall, 772 patients completed the baseline assessment. Distribution-based MIDs (0.5 × SD) for QLQ-PAN26 scales ranged from 12 to 13, except hepatic symptoms (≈8), pancreatic pain (≈10), and sexual dysfunction (≈17); those for stand-alone items ranged from 12 to 16. The SEM values were similar. Among scales/items sufficiently correlated (r > 0.30) with the anchor, MIDs ranged from 5 to 9. Within-patient QLQ-PAN26 RD estimates varied by direction (deterioration vs. improvement) and scale/item, but all values were lower than the true possible within-patient change (e.g. 16.7 points for a two-item scale) given a one-category change on the raw scale. Compared with distribution-based MIDs, anchor-based MIDs were twice as sensitive in detecting group-level changes in QLQ-PAN26 scales/items. For interpreting clinically meaningful change, RDs cannot be less than the true minimum of the scale. The group-level MID may help clinicians/researchers interpret HRQoL changes. ClinicalTrials.gov NCT01964430; Eudra CT 2013-003398-91.
Sections du résumé
BACKGROUND
BACKGROUND
Although the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-PAN26 is widely used to assess health-related quality of life (HRQoL), its group-level minimal important difference (MID) and individual-level responder definition (RD) are not established; we calculated MID and RD using HRQoL data from the APACT trial in patients with surgically resected pancreatic cancer who received adjuvant chemotherapy.
METHODS
METHODS
HRQoL was assessed using EORTC QLQ-C30 and QLQ-PAN26 at baseline, during treatment, at end of treatment, and during follow-up. Distribution-based MIDs were estimated using 0.5 × baseline standard deviation (SD) and reliability-based (intraclass correlation) standard error of measurement (SEM). Anchor-based MIDs and RDs (anchor, QLQ-C30 overall health) were estimated using a linear mixed model.
RESULTS
RESULTS
Overall, 772 patients completed the baseline assessment. Distribution-based MIDs (0.5 × SD) for QLQ-PAN26 scales ranged from 12 to 13, except hepatic symptoms (≈8), pancreatic pain (≈10), and sexual dysfunction (≈17); those for stand-alone items ranged from 12 to 16. The SEM values were similar. Among scales/items sufficiently correlated (r > 0.30) with the anchor, MIDs ranged from 5 to 9. Within-patient QLQ-PAN26 RD estimates varied by direction (deterioration vs. improvement) and scale/item, but all values were lower than the true possible within-patient change (e.g. 16.7 points for a two-item scale) given a one-category change on the raw scale.
CONCLUSIONS
CONCLUSIONS
Compared with distribution-based MIDs, anchor-based MIDs were twice as sensitive in detecting group-level changes in QLQ-PAN26 scales/items. For interpreting clinically meaningful change, RDs cannot be less than the true minimum of the scale. The group-level MID may help clinicians/researchers interpret HRQoL changes.
TRIAL REGISTRATION
BACKGROUND
ClinicalTrials.gov NCT01964430; Eudra CT 2013-003398-91.
Identifiants
pubmed: 33813673
doi: 10.1245/s10434-021-09816-z
pii: 10.1245/s10434-021-09816-z
doi:
Banques de données
ClinicalTrials.gov
['NCT01964430']
Types de publication
Clinical Trial
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
7545-7554Informations de copyright
© 2021. Society of Surgical Oncology.
Références
American Cancer Society. Cancer Facts & Figures 2019. 2019. Available at: https://www.cancer.org/research/cancer-facts-statistics/all-cancer-facts-figures/cancer-facts-figures-2019.html . Accessed 3 Feb 2021.
Surveillance, Epidemiology, and End Results Program. Cancer Stat Facts: Pancreatic Cancer. Available at: https://seer.cancer.gov/statfacts/html/pancreas.html . Accessed 3 Feb 2021.
Bray F, Ferlay J, Soerjomataram I, et al. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2018;68:394–424.
doi: 10.3322/caac.21492
Ducreux M, Cuhna AS, Caramella C, et al. Cancer of the pancreas: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2015;26(Suppl 5):v56–68.
doi: 10.1093/annonc/mdv295
Smeenk HG, Tran TC, Erdmann J, et al. Survival after surgical management of pancreatic adenocarcinoma: does curative and radical surgery truly exist? Langenbecks Arch Surg. 2005;390:94–103.
doi: 10.1007/s00423-004-0476-9
Eaton AA, Gonen M, Karanicolas P, et al. Health-related quality of life after pancreatectomy: results from a randomized controlled trial. Ann Surg Oncol. 2016;23:2137–45.
doi: 10.1245/s10434-015-5077-z
Maharaj AD, Samoborec S, Evans SM, et al. Patient-reported outcome measures (PROMs) in pancreatic cancer: a systematic review. HPB (Oxford). 2020;22:187–203.
doi: 10.1016/j.hpb.2019.09.002
Fitzsimmons D, Johnson CD, George S, et al. Development of a disease specific quality of life (QoL) questionnaire module to supplement the EORTC core cancer QoL questionnaire, the QLQ-C30 in patients with pancreatic cancer. EORTC Study Group on Quality of Life. Eur J Cancer. 1999;35:939–41.
Schmidt J, Abel U, Debus J, et al. Open-label, multicenter, randomized phase III trial of adjuvant chemoradiation plus interferon alfa-2b versus fluorouracil and folinic acid for patients with resected pancreatic adenocarcinoma. J Clin Oncol. 2012;30:4077–83.
doi: 10.1200/JCO.2011.38.2960
Serrano PE, Herman JM, Griffith KA, et al. Quality of life in a prospective, multicenter phase 2 trial of neoadjuvant full-dose gemcitabine, oxaliplatin, and radiation in patients with resectable or borderline resectable pancreatic adenocarcinoma. Int J Radiat Oncol Biol Phys. 2014;90:270–7.
doi: 10.1016/j.ijrobp.2014.05.053
Toyama Y, Yoshida S, Saito R, et al. Successful adjuvant bi-weekly gemcitabine chemotherapy for pancreatic cancer without impairing patients’ quality of life. World J Surg Oncol. 2013;11:3.
doi: 10.1186/1477-7819-11-3
US Food and Drug Administration. Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Bethesda, MD: US FDA; 2009.
US Food and Drug Administration. Patient-Focused Drug Development Guidance Public Workshop. 2018. Available at: https://www.fda.gov/drugs/news-events-human-drugs/patient-focused-drug-development-guidance-methods-identify-what-important-patients-and-select . Accessed 3 Feb 2021.
European Medicines Agency. The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. 2020. Available at: https://www.ema.europa.eu/en/partners-networks/international-activities/multilateral-organisations-initiatives/international-council-harmonisation-technical-requirements-registration-pharmaceuticals-human-use . Accessed 3 Feb 2021.
World Medical Association. WMA Declaration of Helsinki—Ethical Principles for Medical Research Involving Human Subjects. 2018. Available at: https://www.wma.net/policies-post/wma-declaration-of-helsinki-ethical-principles-for-medical-research-involving-human-subjects/ . Accessed 3 Feb 2021.
Tempero MA, Reni M, Riess H, et al. APACT: phase III, multicenter, international, open-label, randomized trial of adjuvant nab-paclitaxel plus gemcitabine vs gemcitabine for surgically resected pancreatic adenocarcinoma. J Clin Oncol. 2019;37(15 suppl):abstract 4000.
Aaronson NK, Ahmedzai S, Bergman B, et al. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst. 1993;85:365–76.
doi: 10.1093/jnci/85.5.365
Ousmen A, Touraine C, Deliu N, et al. Distribution- and anchor-based methods to determine the minimally important difference on patient-reported outcome questionnaires in oncology: a structured review. Health Qual Life Outcomes. 2018;16:228.
doi: 10.1186/s12955-018-1055-z
Norman GR, Sloan JA, Wyrwich KW. Interpretation of changes in health-related quality of life: the remarkable universality of half a standard deviation. Med Care. 2003;41:582–92.
pubmed: 12719681
Wyrwich KW, Tierney WM, Wolinsky FD. Further evidence supporting an SEM-based criterion for identifying meaningful intra-individual changes in health-related quality of life. J Clin Epidemiol. 1999;52:861–73.
doi: 10.1016/S0895-4356(99)00071-2
de Vet HC, Terwee CB, Ostelo RW, et al. Minimal changes in health status questionnaires: distinction between minimally detectable change and minimally important change. Health Qual Life Outcomes. 2006;4:54.
doi: 10.1186/1477-7525-4-54
Hahn EA, Cella D, Chassany O, et al. Precision of health-related quality-of-life data compared with other clinical measures. Mayo Clin Proc. 2007;82:1244–54.
doi: 10.4065/82.10.1244
McGraw KO, Wong SP. Forming inferences about some intraclass correlation coefficients. Psychol Methods. 1996;1:30–46.
doi: 10.1037/1082-989X.1.1.30
Shrout PE, Fleiss JL. Intraclass correlations: uses in assessing rater reliability. Psychol Bull. 1979;86:420–8.
doi: 10.1037/0033-2909.86.2.420
Revicki D, Hays RD, Cella D, Sloan J. Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidemiol. 2008;61:102–9.
doi: 10.1016/j.jclinepi.2007.03.012
Sirls LT, Tennstedt S, Brubaker L, et al. The minimum important difference for the International Consultation on Incontinence Questionnaire-Urinary Incontinence Short Form in women with stress urinary incontinence. Neurourol Urodyn. 2015;34:183–7.
doi: 10.1002/nau.22533
Zeiger RS, Mellon M, Chipps B, et al. Test for Respiratory and Asthma Control in Kids (TRACK): clinically meaningful changes in score. J Allergy Clin Immunol. 2011;128:983–8.
doi: 10.1016/j.jaci.2011.08.010