Top

Gepubliceerd in:

23-01-2025

Meaningful change threshold estimation for the non-small cell lung cancer symptom assessment questionnaire (NSCLC-SAQ): psychometric analysis from a phase 3 trial (LIBRETTO-431)

Auteurs: Nathan Clarke, Gill Worthy, Nalin Payakachat, Adrienne M. Gilligan, Kim Cocks

Gepubliceerd in: Quality of Life Research | Uitgave 4/2025

Abstract

Purpose

Meaningful change thresholds are important to help interpret patient-reported outcome scores. To date, meaningful within-patient change (MWPC) thresholds have only been proposed for NSCLC-SAQ total score. This study proposed clinically MWPC thresholds, and group-level minimal important change/difference (MIC/MID) thresholds for both improvement and worsening for the Non-Small Cell Lung Cancer- Symptom Assessment Questionnaire (NSCLC-SAQ) total and symptom scores.

Methods

Blinded patient data (N = 246) from the Phase 3 LIBRETTO-431 clinical trial were used in a pre-specified meaningful change analysis. A combination of anchor- and supportive distribution-based methods were used to estimate the MWPC, MIC, and MID thresholds. Triangulation across anchor estimates was then performed using a correlation-weighted average to provide a single MWPC, MIC, and MID estimate for improvement and worsening.

Results

NSCLC-SAQ total score and symptom scores showed moderate to high correlations with various anchors (ranging from 0.306 to 0.890), with threshold estimates being provided from multiple anchors (except for cough). Triangulation suggested MWPC, MIC, and MID thresholds for improved total score were − 2.5, -3.5, and − 2.0, respectively. For worsening, the proposed thresholds were 2.0, 0.5, and 2.0, respectively. The MWPC, MIC, and MID thresholds for improved symptom scores ranged from − 0.5 to -1.5, and the worsening thresholds for symptom scores ranged from 0.5 to 1.0.

Conclusion

This study provides the first worsening and improvement estimates of MWPC, MIC, and MID for NSCLC-SAQ total and symptom scores. The thresholds proposed in this study can be used to inform interpretation of NSCLC-SAQ scores in clinical trials.

vorige artikel Investigating item response theory model performance in the context of evaluating clinical outcome assessments in clinical trials

volgende artikel Clinical cut-offs for hip- and knee arthroplasty outcome - minimal clinically important improvement (MCII) and patient acceptable symptom state (PASS) of patient-reported outcome measures (PROM)

Alleen toegankelijk voor geautoriseerde gebruikers

Sung, H., Ferlay, J., Siegel, R. L., Laversanne, M., Soerjomataram, I., Jemal, A., & Bray, F. (2021). Global Cancer statistics 2020: GLOBOCAN estimates of incidence and Mortality Worldwide for 36 cancers in 185 countries. C Ca: A Cancer Journal for Clinicians, 71(3), 209–249. https://doi.org/10.3322/caac.21660CrossRef

American Cancer Society. Facts &, & Figures (2023). from https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2023/2023-cancer-facts-and-figures.pdf

McCarrier, K. P., Atkinson, T. M., DeBusk, K. P., Liepa, A. M., Scanlon, M., & Coons, S. J. (2016). Qualitative development and content validity of the non-small cell Lung Cancer Symptom Assessment Questionnaire (NSCLC-SAQ), a patient-reported Outcome Instrument. Clinical Therapeutics, 38(4), 794–810. https://doi.org/10.1016/j.clinthera.2016.03.012CrossRefPubMedPubMedCentral

Cleeland, C. S., Mendoza, T. R., Wang, X. S., Woodruff, J. F., Palos, G. R., Richman, S. P., Nazario, A., Lynch, G. R., Liao, K. P., Mobley, G. M., & Lu, C. (2011). Levels of symptom burden during chemotherapy for advanced lung cancer: Differences between public hospitals and a tertiary cancer center. Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology, 29(21), 2859–2865. https://doi.org/10.1200/jco.2010.33.4425CrossRefPubMed

Yang, P., Cheville, A. L., Wampfler, J. A., Garces, Y. I., Jatoi, A., Clark, M. M., Cassivi, S. D., Midthun, D. E., Marks, R. S., Aubry, M. C., Okuno, S. H., Williams, B. A., Nichols, F. C., Trastek, V. F., Sugimura, H., Sarna, L., Allen, M. S., Deschamps, C., & Sloan, J. A. (2012). Quality of life and symptom burden among long-term lung cancer survivors. Journal of Thoracic Oncology: Official Publication of the International Association for the Study of Lung Cancer, 7(1), 64–70. https://doi.org/10.1097/JTO.0b013e3182397b3eCrossRefPubMed

Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Retrieved Jun 06 (2023). from https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-reported-outcome-measures-use-medical-product-development-support-labeling-claims

Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. Retrieved Septemeber 09 (2023). from https://www.fda.gov/media/159500/download

Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints For Regulatory Decision-Making. Retrieved September 04 (2023). from https://www.fda.gov/media/166830/download

Bergman, B., Aaronson, N. K., Ahmedzai, S., Kaasa, S., & Sullivan, M. (1994). The EORTC QLQ-LC13: A modular supplement to the EORTC Core Quality of Life Questionnaire (QLQ-C30) for use in lung cancer clinical trials. EORTC Study Group on Quality of Life. European Journal of Cancer, 30a(5), 635–642. https://doi.org/10.1016/0959-8049(94)90535-5CrossRefPubMed

10.

Cella, D. F., Bonomi, A. E., Lloyd, S. R., Tulsky, D. S., Kaplan, E., & Bonomi, P. (1995). Reliability and validity of the Functional Assessment of Cancer Therapy-Lung (FACT-L) quality of life instrument. Lung Cancer, 12(3), 199–220. https://doi.org/10.1016/0169-5002(95)00450-fCrossRefPubMed

11.

Hollen, P. J., Gralla, R. J., Kris, M. G., & Potanovich, L. M. (1993). Quality of life assessment in individuals with lung cancer: Testing the Lung Cancer Symptom Scale (LCSS). European Journal of Cancer, 29A(Suppl 1), 51–58. https://doi.org/10.1016/s0959-8049(05)80262-xCrossRef

12.

Mendoza, T. R., Wang, X. S., Lu, C., Palos, G. R., Liao, Z., Mobley, G. M., Kapoor, S., & Cleeland, C. S. (2011). Measuring the symptom burden of lung cancer: The validity and utility of the lung cancer module of the M. D. Anderson Symptom Inventory Oncologist, 16(2), 217–227. https://doi.org/10.1634/theoncologist.2010-0193CrossRefPubMed

13.

Bushnell, D. M., Atkinson, T. M., McCarrier, K. P., Liepa, A. M., DeBusk, K. P., & Coons, S. J. (2021). Non-small Cell Lung Cancer Symptom Assessment Questionnaire: Psychometric Performance and Regulatory qualification of a Novel patient-reported Symptom measure. Curr Ther Res Clin Exp, 95, 100642. https://doi.org/10.1016/j.curtheres.2021.100642CrossRefPubMedPubMedCentral

14.

Basch, E., Geoghegan, C., Coons, S. J., Gnanasakthy, A., Slagle, A. F., Papadopoulos, E. J., & Kluetz, P. G. (2015). Patient-reported outcomes in Cancer Drug Development and US Regulatory Review: Perspectives from industry, the Food and Drug Administration, and the patient. JAMA Oncol, 1(3), 375–379. https://doi.org/10.1001/jamaoncol.2015.0530CrossRefPubMed

15.

Plan for Issuance of Patient-Focused Drug Development Guidance. Retrieved Jun 11 (2023). from https://www.fda.gov/files/about%20fda/published/Plan-for-Issuance-of-Patient%E2%80%90Focused-Drug-Development-Guidance.pdf

16.

Coons, S. J., Kothari, S., Monz, B. U., & Burke, L. B. (2011). The patient-reported outcome (PRO) consortium: Filling measurement gaps for PRO end points to support labeling claims. Clinical Pharmacology and Therapeutics, 90(5), 743–748. https://doi.org/10.1038/clpt.2011.203CrossRefPubMed

17.

Williams, P., Burke, T., Norquist, J. M., Daskalopoulou, C., Speck, R. M., Samkari, A., Eremenco, S., & Coons, S. J. (2022). Non-small Cell Lung Cancer Symptom Assessment Questionnaire (NSCLC-SAQ): Measurement Properties and estimated clinically meaningful thresholds from a phase 3 study. JTO Clin Res Rep, 3(4), 100298. https://doi.org/10.1016/j.jtocrr.2022.100298CrossRefPubMedPubMedCentral

18.

(March, G. K. (2021). FP14.05 LIBRETTO-431: Selpercatinib in Treatment-Naïve patients with RET Fusion-positive Non-small Cell Lung Cancer (NSCLC). Journal of Thoracic Oncology, S228–S229. https://doi.org/10.1016/j.jtho.2021.01.148. 16(3 (Supplement).

19.

Solomon, B. J., Zhou, C. C., Drilon, A., Park, K., Wolf, J., Elamin, Y., Davis, H. M., Soldatenkova, V., Sashegyi, A., Lin, A. B., Lin, B. K., HH, F. L., Novello, S., Arriola, E., Pérol, M., Goto, K., & Santini, F. C. (2021). Phase III study of selpercatinib versus chemotherapy ± pembrolizumab in untreated RET positive non-small-cell lung cancer. Future Oncology, 17(7), 763–773. https://doi.org/10.2217/fon-2020-0935CrossRefPubMed

20.

PM, F. (2001). The EORTC QLQ-C30 Scoring Manual (3rd edn.). Brussels: European Organisation for Research and Treatment of Cancer.

21.

FACIT Item GP5, Functional Assessment of Chronic Illness Therapy - Item GP5. Retrieved Jul 31 (2023). from https://www.facit.org/measures/FACIT-Item-GP5

22.

Development and Testing of the PRO-CTCAE Measurement System. Retrieved Jul 31 (2023). from https://healthcaredelivery.cancer.gov/pro-ctcae/testing.html

23.

Devlin, N., Pickard, S., & Busschbach, J. (2022). The development of the EQ-5D-5L and its value sets. In N. Devlin, B. Roudijk, & K. Ludwig (Eds.), Value sets for EQ-5D-5L: A compendium, comparative Review & user guide (pp. 1–12). Springer International Publishing.

24.

Trigg, A., Lenderking, W. R., & Boehnke, J. R. (2023). Introduction to the special section: Methodologies and considerations for meaningful change. Quality of Life Research, 32(5), 1223–1230. https://doi.org/10.1007/s11136-023-03413-1CrossRefPubMed

25.

Revicki, D., Hays, R. D., Cella, D., & Sloan, J. (2008). Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. Journal of Clinical Epidemiology, 61(2), 102–109. https://doi.org/10.1016/j.jclinepi.2007.03.012CrossRefPubMed

26.

Vanier, A., Sébille, V., Blanchin, M., & Hardouin, J. B. (2021). The minimal perceived change: A formal model of the responder definition according to the patient’s meaning of change for patient-reported outcome data analysis and interpretation. Bmc Medical Research Methodology, 21(1), 128. https://doi.org/10.1186/s12874-021-01307-9CrossRefPubMedPubMedCentral

27.

Froud, R., & Abel, G. (2014). Using ROC curves to choose minimally important change thresholds when sensitivity and specificity are valued equally: The forgotten lesson of pythagoras. Theoretical considerations and an example application of change in health status. PLoS One, 9(12), e114468. https://doi.org/10.1371/journal.pone.0114468CrossRefPubMedPubMedCentral

28.

Norman, G. R., Sloan, J. A., & Wyrwich, K. W. (2004). The truly remarkable universality of half a standard deviation: Confirmation through another look. Expert Rev Pharmacoecon Outcomes Res, 4(5), 581–585. https://doi.org/10.1586/14737167.4.5.581CrossRefPubMed

29.

King, M. T. (2011). A point of minimal important difference (MID): A critique of terminology and methods. Expert Rev Pharmacoecon Outcomes Res, 11(2), 171–184. https://doi.org/10.1586/erp.11.9CrossRefPubMed

30.

Trigg, A., & Griffiths, P. (2021). Triangulation of multiple meaningful change thresholds for patient-reported outcome scores. Quality of Life Research, 30(10), 2755–2764. https://doi.org/10.1007/s11136-021-02957-4CrossRefPubMed

31.

Ren, J., Bushmakin, A. G., Cislo, P. R., Abraham, L., Cappelleri, J. C., Dworkin, R. H., & Farrar, J. T. (2023). Meaningful within-patient change for clinical outcome assessments: Model-based approach versus cumulative distribution functions. Journal of Biopharmaceutical Statistics, 1–13. https://doi.org/10.1080/10543406.2023.2281575

32.

Cocks, K., King, M. T., Velikova, G., St-James, M., Fayers, M., 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. https://doi.org/10.1200/jco.2010.28.0107CrossRefPubMed

33.

Ringash, J., O’Sullivan, B., Bezjak, A., & Redelmeier, D. A. (2007). Interpreting clinically significant changes in patient-reported outcomes. Cancer, 110(1), 196–202. https://doi.org/10.1002/cncr.22799CrossRefPubMed

34.

Cella, D., Hahn, E. A., & Dineen, K. (2002). Meaningful change in cancer-specific quality of life scores: Differences between improvement and worsening. Quality of Life Research, 11(3), 207–221. https://doi.org/10.1023/a:1015276414526CrossRefPubMed

35.

Cocks, K., & Buchanan, J. (2022). How scoring limits the usability of minimal important differences (MIDs) as responder definition (RD): An exemplary demonstration using EORTC QLQ-C30 subscales. Quality of Life Research. https://doi.org/10.1007/s11136-022-03181-4CrossRefPubMed

36.

HCWW, V. (2011). Measurement in Medicine: A practical guide (practical guides to Biostatistics and Epidemiology) (1st edn.). Cambridge University Press.

Titel: Meaningful change threshold estimation for the non-small cell lung cancer symptom assessment questionnaire (NSCLC-SAQ): psychometric analysis from a phase 3 trial (LIBRETTO-431)
Auteurs: Nathan Clarke
Gill Worthy
Nalin Payakachat
Adrienne M. Gilligan
Kim Cocks
Publicatiedatum: 23-01-2025
Uitgeverij: Springer International Publishing
Gepubliceerd in: Quality of Life Research / Uitgave 4/2025
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI: https://doi.org/10.1007/s11136-025-03895-1

Andere artikelen Uitgave 4/2025

Clinical cut-offs for hip- and knee arthroplasty outcome - minimal clinically important improvement (MCII) and patient acceptable symptom state (PASS) of patient-reported outcome measures (PROM)

Open Access

Exploring the content validity of the EQ-5D-5L and four bolt-ons (skin irritation, self-confidence, sleep, social relationships) in atopic dermatitis and chronic urticaria

Open Access

Content and face validity of preference-based quality of life instruments in indigenous youth experiencing mental health problems: a qualitative study

Comparison of patient-reported symptoms with multi-item patient-reported outcome measures of fatigue, anxiety, and depression in the clinical care of women undergoing chemotherapy for early breast cancer

Physical activity, and improvement in health-related quality of life among Australian middle-aged and older adults living with type 2 diabetes mellitus

In varietate concordia – custer analysis of EQ-5D-5L value sets in European Union countries

Open Access

Bohn Stafleu van Loghum

Welkom bij Scalda & Bohn Stafleu van Loghum

Registreer

Login

Meaningful change threshold estimation for the non-small cell lung cancer symptom assessment questionnaire (NSCLC-SAQ): psychometric analysis from a phase 3 trial (LIBRETTO-431)

Abstract

Purpose

Methods

Results

Conclusion

Andere artikelen Uitgave 4/2025

Clinical cut-offs for hip- and knee arthroplasty outcome - minimal clinically important improvement (MCII) and patient acceptable symptom state (PASS) of patient-reported outcome measures (PROM)

Exploring the content validity of the EQ-5D-5L and four bolt-ons (skin irritation, self-confidence, sleep, social relationships) in atopic dermatitis and chronic urticaria

Content and face validity of preference-based quality of life instruments in indigenous youth experiencing mental health problems: a qualitative study

Comparison of patient-reported symptoms with multi-item patient-reported outcome measures of fatigue, anxiety, and depression in the clinical care of women undergoing chemotherapy for early breast cancer

Physical activity, and improvement in health-related quality of life among Australian middle-aged and older adults living with type 2 diabetes mellitus

In varietate concordia – custer analysis of EQ-5D-5L value sets in European Union countries

Bohn Stafleu van Loghum

Welkom bij Scalda & Bohn Stafleu van Loghum

Registreer

Login

Deel dit onderdeel of sectie (kopieer de link)

Abstract

Purpose

Methods

Results

Conclusion

Log in om toegang te krijgen

Clinical cut-offs for hip- and knee arthroplasty outcome - minimal clinically important improvement (MCII) and patient acceptable symptom state (PASS) of patient-reported outcome measures (PROM)

Exploring the content validity of the EQ-5D-5L and four bolt-ons (skin irritation, self-confidence, sleep, social relationships) in atopic dermatitis and chronic urticaria

Content and face validity of preference-based quality of life instruments in indigenous youth experiencing mental health problems: a qualitative study

Comparison of patient-reported symptoms with multi-item patient-reported outcome measures of fatigue, anxiety, and depression in the clinical care of women undergoing chemotherapy for early breast cancer

Physical activity, and improvement in health-related quality of life among Australian middle-aged and older adults living with type 2 diabetes mellitus

In varietate concordia – custer analysis of EQ-5D-5L value sets in European Union countries