Skip to main content
Top
Gepubliceerd in:

29-04-2023

Psychometrics of patient-reported bother from side effects of treatment single-items in industry-sponsored oncology trials

Auteurs: Peter C. Trask, Tracy Holt, Timothy Pearman

Gepubliceerd in: Quality of Life Research | Uitgave 9/2023

Log in om toegang te krijgen
share
DELEN

Deel dit onderdeel of sectie (kopieer de link)

  • Optie A:
    Klik op de rechtermuisknop op de link en selecteer de optie “linkadres kopiëren”
  • Optie B:
    Deel de link per e-mail

Abstract

Background

Improvements in cancer treatment have increased the number of cancer survivors, but also increased the long-term and late effects from cancer therapy. Patient reported “side effect bother” could be used to measure the burden of treatment, and the risk for negative outcomes such as dose reduction, treatment delay or discontinuation. The current study addresses the psychometric properties of a single item, determines what represents a “meaningful change”, and evaluates the correlation to safety endpoints and functioning.

Methods

Results from 5911 patients enrolled in 8 clinical trials representing 5 disease types in oncology and hematology who completed either the Functional Assessment of Cancer Therapy (FACT) GP5 item or a modified bother item (MBI) were assessed.

Results

Patients ranged in age from 18 to 93 years, with all cancer stages represented and approximately equal numbers of males and females. Test–retest reliability was acceptable, as were convergent and known groups validity. The GP5 and MBI effectively demonstrated sensitivity to change over time and established meaningful thresholds.

Conclusions

The results indicate that these single-items are psychometrically sound, capable of distinguishing known groups, responsive to change and can identify meaningful change over time in terms of treatment-related symptoms. It extends the findings of recent scientific groups by providing analyses not included in prior studies, and further supports the FDA’s recommendation to include a single item question in clinical trials.
Literatuur
1.
go back to reference American Cancer Society. (2022). Cancer facts & figures 2022. American Cancer Society. American Cancer Society. (2022). Cancer facts & figures 2022. American Cancer Society.
2.
go back to reference Sciotto, F., Py, C., & Tsantoulis, P. (2017). Follow up of the long-term cancer survivor. Revue Médicale Suisse, 13, 1044–1048.CrossRefPubMed Sciotto, F., Py, C., & Tsantoulis, P. (2017). Follow up of the long-term cancer survivor. Revue Médicale Suisse, 13, 1044–1048.CrossRefPubMed
3.
go back to reference Pearman, T. P., Garcia, S., Penedo, F., Yanez, B., Wagner, L. I., & Cella, D. (2015). Implementation of distress screening in an oncology setting. The Journal of Community and Supportive Oncology, 13, 423–428.CrossRefPubMed Pearman, T. P., Garcia, S., Penedo, F., Yanez, B., Wagner, L. I., & Cella, D. (2015). Implementation of distress screening in an oncology setting. The Journal of Community and Supportive Oncology, 13, 423–428.CrossRefPubMed
4.
go back to reference Schirrmacher, V. (2019). From chemotherapy to biological therapy: A review of novel concepts to reduce the side effects of systemic cancer treatment. International Journal of Oncology, 54, 407–419.CrossRefPubMed Schirrmacher, V. (2019). From chemotherapy to biological therapy: A review of novel concepts to reduce the side effects of systemic cancer treatment. International Journal of Oncology, 54, 407–419.CrossRefPubMed
5.
go back to reference Cheung, W. Y., White, M. K., Bayliss, M. S., Stroupe, A., Lovley, A., King-Kalimannis, B. L., & Lasch, K. (2019). Patient-reported treatment-related symptom burden for patients with advanced melanoma in Canada. Supportive Care in Cancer, 27, 219–227.CrossRefPubMed Cheung, W. Y., White, M. K., Bayliss, M. S., Stroupe, A., Lovley, A., King-Kalimannis, B. L., & Lasch, K. (2019). Patient-reported treatment-related symptom burden for patients with advanced melanoma in Canada. Supportive Care in Cancer, 27, 219–227.CrossRefPubMed
6.
go back to reference Bernhard, J., Maibach, R., Thurlimann, B., Sessa, C., & Aapro, M. S. (2002). Patients’ estimation of overall treatment burden: Why not ask the obvious? Journal of Clinical Oncology., 20(1), 65–72.PubMed Bernhard, J., Maibach, R., Thurlimann, B., Sessa, C., & Aapro, M. S. (2002). Patients’ estimation of overall treatment burden: Why not ask the obvious? Journal of Clinical Oncology., 20(1), 65–72.PubMed
7.
go back to reference Griffin, A. M., Butow, P. N., Coates, A. S., Childs, A. M., Ellis, P. M., Dunn, S. M., & Tattersall, M. H. (1996). On the receiving end: Patient perceptions of the side effects of cancer chemotherapy in 1993. Annals of Oncology, 7, 189–195.CrossRefPubMed Griffin, A. M., Butow, P. N., Coates, A. S., Childs, A. M., Ellis, P. M., Dunn, S. M., & Tattersall, M. H. (1996). On the receiving end: Patient perceptions of the side effects of cancer chemotherapy in 1993. Annals of Oncology, 7, 189–195.CrossRefPubMed
8.
go back to reference Portenoy, R. K., Thaler, H. T., Kornblith, A. B., Lepore, J. M., Friedlander-Klar, H., Coyle, N., Smart-Curley, T., Kemeny, N., Norton, L., & Hoskins, W. (1994). Symptom prevalence, characteristics and distress in a cancer population. Quality of Life Research, 3, 183–189.CrossRefPubMed Portenoy, R. K., Thaler, H. T., Kornblith, A. B., Lepore, J. M., Friedlander-Klar, H., Coyle, N., Smart-Curley, T., Kemeny, N., Norton, L., & Hoskins, W. (1994). Symptom prevalence, characteristics and distress in a cancer population. Quality of Life Research, 3, 183–189.CrossRefPubMed
9.
go back to reference Slevin, M. L., Plant, H., Lynch, D., Drinkwater, J., & Gregory, W. M. (1988). Who should measure quality of life, the doctor or the patient? British Journal of Cancer, 1988(57), 109–112.CrossRef Slevin, M. L., Plant, H., Lynch, D., Drinkwater, J., & Gregory, W. M. (1988). Who should measure quality of life, the doctor or the patient? British Journal of Cancer, 1988(57), 109–112.CrossRef
10.
go back to reference Sprangers, M. A., & Aaronson, N. K. (1992). The role of health care providers and significant others in evaluating the quality of life of patients with chronic disease: A review. Journal of Clinical Epidemiology, 45, 743–760.CrossRefPubMed Sprangers, M. A., & Aaronson, N. K. (1992). The role of health care providers and significant others in evaluating the quality of life of patients with chronic disease: A review. Journal of Clinical Epidemiology, 45, 743–760.CrossRefPubMed
11.
go back to reference Pearman, T. P., Beaumont, J. L., Mroczek, D., O’Connor, M., & Cella, D. (2018). Validity and usefulness of a single-item measure of patient-reported bother from side effects of cancer therapy. Cancer, 124, 991–997.CrossRefPubMed Pearman, T. P., Beaumont, J. L., Mroczek, D., O’Connor, M., & Cella, D. (2018). Validity and usefulness of a single-item measure of patient-reported bother from side effects of cancer therapy. Cancer, 124, 991–997.CrossRefPubMed
12.
go back to reference FDA. (2021, June). Core patient-reported outcomes in cancer clinical trials guidance for industry. Draft guidance. U.S. Department of Health and Human Services Food and Drug Administration Oncology Center of Excellence (OCE). FDA. (2021, June). Core patient-reported outcomes in cancer clinical trials guidance for industry. Draft guidance. U.S. Department of Health and Human Services Food and Drug Administration Oncology Center of Excellence (OCE).
13.
go back to reference Bennett, B. K., Park, S. B., Lin, C. S., Friedlander, M. L., Kiernan, M. C., & Goldstein, D. (2012). Impact of oxaliplatin-induced neuropathy: A patient perspective. Supportive Care Cancer., 20, 2959–2967.CrossRef Bennett, B. K., Park, S. B., Lin, C. S., Friedlander, M. L., Kiernan, M. C., & Goldstein, D. (2012). Impact of oxaliplatin-induced neuropathy: A patient perspective. Supportive Care Cancer., 20, 2959–2967.CrossRef
14.
go back to reference Basch, E., Iasonos, A., McDonough, T., Barz, A., Culkin, A., Kris, M. G., Scher, H. I., & Schrag, D. (2006). Patient versus clinician symptom reporting using the national cancer institute common terminology criteria for adverse events: Results of a questionnaire-based study. The lancet Oncology, 7, 903–909.CrossRefPubMed Basch, E., Iasonos, A., McDonough, T., Barz, A., Culkin, A., Kris, M. G., Scher, H. I., & Schrag, D. (2006). Patient versus clinician symptom reporting using the national cancer institute common terminology criteria for adverse events: Results of a questionnaire-based study. The lancet Oncology, 7, 903–909.CrossRefPubMed
15.
go back to reference Nyrop, K. A., Deal, A. M., et al. (2020). Congruence of patient- and clinician-reported toxicity in women receiving chemotherapy for early breast cancer. Cancer, 126, 3084–3093.CrossRefPubMed Nyrop, K. A., Deal, A. M., et al. (2020). Congruence of patient- and clinician-reported toxicity in women receiving chemotherapy for early breast cancer. Cancer, 126, 3084–3093.CrossRefPubMed
16.
go back to reference Schuurhuizen, C., Marino, P., Braamse, A., et al. (2018). Impact of patient- and clinician-reported cumulative toxicity on quality of life in patients with metastatic castration-naïve prostate cancer. Journal of the National Comprehensive Cancer Network, 16, 1481–1488.CrossRefPubMed Schuurhuizen, C., Marino, P., Braamse, A., et al. (2018). Impact of patient- and clinician-reported cumulative toxicity on quality of life in patients with metastatic castration-naïve prostate cancer. Journal of the National Comprehensive Cancer Network, 16, 1481–1488.CrossRefPubMed
17.
go back to reference Basch, E., Reeve, B. B., & Mitchell, S. A. (2014). Development of the national cancer institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE). JNCI: Journal of National Cancer Institue., 106, 244.CrossRef Basch, E., Reeve, B. B., & Mitchell, S. A. (2014). Development of the national cancer institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE). JNCI: Journal of National Cancer Institue., 106, 244.CrossRef
18.
go back to reference Dueck, A. C., Mendoza, T. R., Mitchell, S. A., et al. (2015). Validity and reliability of the US national cancer institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE). JAMA Oncology, 1, 1051–1059.CrossRefPubMedPubMedCentral Dueck, A. C., Mendoza, T. R., Mitchell, S. A., et al. (2015). Validity and reliability of the US national cancer institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE). JAMA Oncology, 1, 1051–1059.CrossRefPubMedPubMedCentral
19.
go back to reference Griffiths, P., Peipert, J. D., Leith, A., Rider, A., Morgan, L., Cella, D., & Cocks, K. (2022). Validity of a single-item indicator of treatment side effect bother in a diverse sample of cancer patients. Support Care Cancer; online ahead of print. Griffiths, P., Peipert, J. D., Leith, A., Rider, A., Morgan, L., Cella, D., & Cocks, K. (2022). Validity of a single-item indicator of treatment side effect bother in a diverse sample of cancer patients. Support Care Cancer; online ahead of print.
20.
go back to reference Roydhouse, J. K., King-Kallimanis, B. L., Roy, P., Weinstock, C., Krol, D., Daniels, S. R., Suzman, D. L., Beaver, J. A., & Kluetz, P. G. (2020). Exploration of baseline patient-reported side effect bother from cancer therapy. Clinical Trials, 17, 332–337.CrossRefPubMed Roydhouse, J. K., King-Kallimanis, B. L., Roy, P., Weinstock, C., Krol, D., Daniels, S. R., Suzman, D. L., Beaver, J. A., & Kluetz, P. G. (2020). Exploration of baseline patient-reported side effect bother from cancer therapy. Clinical Trials, 17, 332–337.CrossRefPubMed
21.
go back to reference Cella, D. F., Tulsky, D. S., Gray, G., Sarafian, B., Linn, E., Bonomi, A., Silberman, M., Yellen, S. B., Winicour, P., & Brannon, J. (1993). The functional assessment of cancer therapy scale: Development and validation of the general measure. Journal of Clinical Oncology, 11, 570–579.CrossRefPubMed Cella, D. F., Tulsky, D. S., Gray, G., Sarafian, B., Linn, E., Bonomi, A., Silberman, M., Yellen, S. B., Winicour, P., & Brannon, J. (1993). The functional assessment of cancer therapy scale: Development and validation of the general measure. Journal of Clinical Oncology, 11, 570–579.CrossRefPubMed
22.
go back to reference Hlubocky, F. J., Webster, K., Cashy, J., Beaumont, J., Cella, D. (2013). The development and validation of a measure of health-related quality of life for non-Hodgkin’s Lymphoma: The Functional Assessment of Cancer Therapy—Lymphoma (FACT-Lym). Lymphoma. Hlubocky, F. J., Webster, K., Cashy, J., Beaumont, J., Cella, D. (2013). The development and validation of a measure of health-related quality of life for non-Hodgkin’s Lymphoma: The Functional Assessment of Cancer Therapy—Lymphoma (FACT-Lym). Lymphoma.
23.
go back to reference Basen-Engquist, K., Bodurka-Bevers, D., & Fitzgerald, M. A. (2001). Reliability and validity of the functional assessment of cancer therapy-ovarian. Journal of Clinical Oncology, 19, 1809–1817.CrossRefPubMed Basen-Engquist, K., Bodurka-Bevers, D., & Fitzgerald, M. A. (2001). Reliability and validity of the functional assessment of cancer therapy-ovarian. Journal of Clinical Oncology, 19, 1809–1817.CrossRefPubMed
24.
go back to reference Aaronson, N. K., Ahmedzai, S., & Bergman, B. (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, 365–376.CrossRefPubMed Aaronson, N. K., Ahmedzai, S., & Bergman, B. (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, 365–376.CrossRefPubMed
25.
go back to reference Sprangers, M. A., Groenvold, M., Arraras, J. I., Franklin, J., teVelde, A., Muller, M., Franzini, L., Williams, A., de Haess, H. C., Hopwood, P., Cull, A., & Aaronson, N. K. (1996). The EORTC breast cancer-specific quality-of-life questionnaire module: First results from a three-country field study. Journal of Clinical Oncology, 14, 2756–2768.CrossRefPubMed Sprangers, M. A., Groenvold, M., Arraras, J. I., Franklin, J., teVelde, A., Muller, M., Franzini, L., Williams, A., de Haess, H. C., Hopwood, P., Cull, A., & Aaronson, N. K. (1996). The EORTC breast cancer-specific quality-of-life questionnaire module: First results from a three-country field study. Journal of Clinical Oncology, 14, 2756–2768.CrossRefPubMed
26.
go back to reference Greimel, E., Bottomley, A., Cull, A., Waldenstrom, A.-C., Arraras, J., Chauvenet, L., Holzner, B., Kuljanic, K., Lebrec, J., & D’haese, S. (2003). EORTC Quality of Life Group and the Quality of Life Unit: An international field study of the reliability and validity of a disease-specific questionnaire module (the QLQ-OV28) in assessing the quality of life of patients with ovarian cancer. European Journal of Cancer, 39, 1402–1408.CrossRefPubMed Greimel, E., Bottomley, A., Cull, A., Waldenstrom, A.-C., Arraras, J., Chauvenet, L., Holzner, B., Kuljanic, K., Lebrec, J., & D’haese, S. (2003). EORTC Quality of Life Group and the Quality of Life Unit: An international field study of the reliability and validity of a disease-specific questionnaire module (the QLQ-OV28) in assessing the quality of life of patients with ovarian cancer. European Journal of Cancer, 39, 1402–1408.CrossRefPubMed
27.
go back to reference Janssen, M. F., Pickard, A. S., Golicki, D., Gudex, C., Nieuwada, M., Scalone, M., Swinburn, P., & Bussbach, J. (2013). Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L across eight patient groups: A multi-country study. Quality of Life Research, 22, 1717–1727.CrossRefPubMed Janssen, M. F., Pickard, A. S., Golicki, D., Gudex, C., Nieuwada, M., Scalone, M., Swinburn, P., & Bussbach, J. (2013). Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L across eight patient groups: A multi-country study. Quality of Life Research, 22, 1717–1727.CrossRefPubMed
28.
go back to reference Rabin, R., & de Charro, F. (2001). EQ-5D: A measure of health status from the EuroQol Group. Annals of Medicine, 33, 337–343.CrossRefPubMed Rabin, R., & de Charro, F. (2001). EQ-5D: A measure of health status from the EuroQol Group. Annals of Medicine, 33, 337–343.CrossRefPubMed
29.
go back to reference Terwee, C. B., Bot, S. D. M., de Boer, M. R., van der Windt, D. A. W. M., Knol, D. L., Dekker, J., Bouter, L. M., & de Vet, H. C. W. (2007). Quality criteria were proposed for measurement properties of health status questionnaires. Journal of Clinical Epidemiology, 60, 34–42.CrossRefPubMed Terwee, C. B., Bot, S. D. M., de Boer, M. R., van der Windt, D. A. W. M., Knol, D. L., Dekker, J., Bouter, L. M., & de Vet, H. C. W. (2007). Quality criteria were proposed for measurement properties of health status questionnaires. Journal of Clinical Epidemiology, 60, 34–42.CrossRefPubMed
30.
go back to reference Cella, D., Hahn, E. A., & Dineen, K. (2022). Meaningful change in cancer-specific quality of life scores: Differences between improvement and worsening. Quality of Life Research, 11, 207–221.CrossRef Cella, D., Hahn, E. A., & Dineen, K. (2022). Meaningful change in cancer-specific quality of life scores: Differences between improvement and worsening. Quality of Life Research, 11, 207–221.CrossRef
31.
go back to reference Osoba, D., Rodrigues, G., Myles, J., Zee, B., & Pater, J. (1998). Interpreting the significance of changes in health-related quality-of-life scores. Journal of Clinical Oncology, 16, 139–144.CrossRefPubMed Osoba, D., Rodrigues, G., Myles, J., Zee, B., & Pater, J. (1998). Interpreting the significance of changes in health-related quality-of-life scores. Journal of Clinical Oncology, 16, 139–144.CrossRefPubMed
32.
go back to reference Koo, T. K., & Li, M. Y. (2016). A guideline of selecting and reporting intraclass correlation coefficients for reliability research. Journal of Chiropractic Medicine, 15(2), 155–163.CrossRefPubMedPubMedCentral Koo, T. K., & Li, M. Y. (2016). A guideline of selecting and reporting intraclass correlation coefficients for reliability research. Journal of Chiropractic Medicine, 15(2), 155–163.CrossRefPubMedPubMedCentral
33.
go back to reference Botsch, R. (2011). Chapter 12: Significance and measures of association. Scopes and Methods of Political Science Botsch, R. (2011). Chapter 12: Significance and measures of association. Scopes and Methods of Political Science
34.
go back to reference Copay, A. G., Subach, B. R., Glassman, S. D., Polly, D. W., Jr., & Schuler, T. C. (2007). Understanding the minimum clinically important difference: A review of concepts and methods. Spine J., 7(5), 541–546.CrossRefPubMed Copay, A. G., Subach, B. R., Glassman, S. D., Polly, D. W., Jr., & Schuler, T. C. (2007). Understanding the minimum clinically important difference: A review of concepts and methods. Spine J., 7(5), 541–546.CrossRefPubMed
35.
go back to reference Ousmen, A., Touraine, C., Deliu, N., Cottone, F., Bonnetain, F., Efficace, F., Brédart, A., Mollevi, C., & Anota, A. (2018). Distribution- and anchor-based methods to determine the minimally important difference on patient-reported outcome questionnaires in oncology: A structured review. Health and Quality of Life Outcomes, 16, 228.CrossRefPubMedPubMedCentral Ousmen, A., Touraine, C., Deliu, N., Cottone, F., Bonnetain, F., Efficace, F., Brédart, A., Mollevi, C., & Anota, A. (2018). Distribution- and anchor-based methods to determine the minimally important difference on patient-reported outcome questionnaires in oncology: A structured review. Health and Quality of Life Outcomes, 16, 228.CrossRefPubMedPubMedCentral
36.
go back to reference 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, 102–109.CrossRefPubMed 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, 102–109.CrossRefPubMed
37.
go back to reference Wyrwich, K. W., Norquist, J. M., Lenderking, W. R., & Acaster, S. (2013). Industry advisory committee of international society for quality of life R: Methods for interpreting change over time in patient-reported outcome measures. Quality of Life Research, 22, 475–483.CrossRefPubMed Wyrwich, K. W., Norquist, J. M., Lenderking, W. R., & Acaster, S. (2013). Industry advisory committee of international society for quality of life R: Methods for interpreting change over time in patient-reported outcome measures. Quality of Life Research, 22, 475–483.CrossRefPubMed
38.
go back to reference Lee, K. M., Lee, J., & Chung, C. Y. (2012). Pitfalls and important issues in testing reliability using intraclass correlation coefficients in orthopaedic research. Clinics in Orthopedic Surgery, 4, 149–155.CrossRefPubMedPubMedCentral Lee, K. M., Lee, J., & Chung, C. Y. (2012). Pitfalls and important issues in testing reliability using intraclass correlation coefficients in orthopaedic research. Clinics in Orthopedic Surgery, 4, 149–155.CrossRefPubMedPubMedCentral
Metagegevens
Titel
Psychometrics of patient-reported bother from side effects of treatment single-items in industry-sponsored oncology trials
Auteurs
Peter C. Trask
Tracy Holt
Timothy Pearman
Publicatiedatum
29-04-2023
Uitgeverij
Springer International Publishing
Gepubliceerd in
Quality of Life Research / Uitgave 9/2023
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-023-03426-w

Andere artikelen Uitgave 9/2023

Quality of Life Research 9/2023 Naar de uitgave