Skip to main content
Top
Gepubliceerd in:

11-03-2021

Study objectives in clinical trials in older patients with solid malignancies: do we measure what matters?

Auteurs: E. R. M. Scheepers, L. H. van Huis-Tanja, M. H. Emmelot-Vonk, M. E. Hamaker

Gepubliceerd in: Quality of Life Research | Uitgave 7/2021

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

Purpose

We set out to determine study objectives of clinical trials which included older patients with the four most common malignancies, to assess the extent to which the inclusion of patient-related outcomes (PROs) has changed over the last fifteen years.

Method

A search of the National Institutes of Health clinical trial registry was performed to identify currently recruiting or completed phase II or III clinical trials started between 2005 and 2020, which addressed chemotherapy or immunotherapy in patients aged > 65 years with the four most common solid malignancies. Trial characteristics and study objectives were extracted from the registry website.

Results

Compared to disease- and treatment-related outcomes, PROs were the least measured outcomes. Of the 1,663 trials, PROs were addressed in only 21% of all trials, in which quality of life as primary objective was found in less than 1% of all trials. Compared to all trials, trials exclusively for older patients addressed more often PROs (respectively, 30% vs 21%, p < 0.001). Over the last fifteen years, there was an incremental trend in the reporting of PROs from 17 to 24% of all trials (p = 0.007).

Conclusion

Despite a slight incremental trend over the past 15 years, PROs appear to be underrepresented in clinical trials which include patients with a solid malignancy. In order to provide physicians and older patients with cancer realistic information about the impact of chemo- or immunotherapy on quality of life or functioning, researchers should strongly consider including PROs in their future clinical trials.
Bijlagen
Alleen toegankelijk voor geautoriseerde gebruikers
Literatuur
3.
go back to reference Scher, K. S., & Hurria, A. (2012). Under-representation of older adults in cancer registration trials: Known problem, little progress. Journal of Clinical Oncology, 30(17), 2036–8.CrossRef Scher, K. S., & Hurria, A. (2012). Under-representation of older adults in cancer registration trials: Known problem, little progress. Journal of Clinical Oncology, 30(17), 2036–8.CrossRef
4.
go back to reference Fried, T. R., Bradley, E. H., Towle, V. R., & Allore, H. (2002). Understanding the Treatment Preferences of Seriously Ill Patients. The New England Journal of Medicine, 346(14), 1061–5.CrossRef Fried, T. R., Bradley, E. H., Towle, V. R., & Allore, H. (2002). Understanding the Treatment Preferences of Seriously Ill Patients. The New England Journal of Medicine, 346(14), 1061–5.CrossRef
5.
go back to reference Hurria, A., Mohile, S. G., & Dale, W. (2012). Research priorities in geriatric oncology: Addressing the needs of an aging population. JNCCN Journal of the National Comprehensive Cancer Network, 10(2), 286–8.CrossRef Hurria, A., Mohile, S. G., & Dale, W. (2012). Research priorities in geriatric oncology: Addressing the needs of an aging population. JNCCN Journal of the National Comprehensive Cancer Network, 10(2), 286–8.CrossRef
6.
go back to reference Pallis, A. G., Ring, A., Fortpied, C., Penninckx, B., van Nes, M. C., Wedding, U., et al. (2011). Eortc workshop on clinical trial methodology in older individuals with a diagnosis of solid tumors. Annals of Oncology, 22(8), 1922–6.CrossRef Pallis, A. G., Ring, A., Fortpied, C., Penninckx, B., van Nes, M. C., Wedding, U., et al. (2011). Eortc workshop on clinical trial methodology in older individuals with a diagnosis of solid tumors. Annals of Oncology, 22(8), 1922–6.CrossRef
7.
go back to reference Van Leeuwen, K. M., Van Loon, M. S., Van Nes, F. A., Bosmans, J. E., De Veti, H. C. W., Ket, J. C. F., et al. (2019). What does quality of life mean to older adults? A thematic synthesis. PLoS One, 14(3), 1–39. Van Leeuwen, K. M., Van Loon, M. S., Van Nes, F. A., Bosmans, J. E., De Veti, H. C. W., Ket, J. C. F., et al. (2019). What does quality of life mean to older adults? A thematic synthesis. PLoS One, 14(3), 1–39.
8.
go back to reference Yellen, S. B., Cella, D. F., & Leslie, W. T. (1994). Age and clinical decision making in oncology patients. Journal of the National Cancer Institute, 86, 1766–70.CrossRef Yellen, S. B., Cella, D. F., & Leslie, W. T. (1994). Age and clinical decision making in oncology patients. Journal of the National Cancer Institute, 86, 1766–70.CrossRef
9.
go back to reference McKenna, S. P. (2011). Measuring patient-reported outcomes: Moving beyond misplaced common sense to hard science. BMC Medicine, 9, 86.CrossRef McKenna, S. P. (2011). Measuring patient-reported outcomes: Moving beyond misplaced common sense to hard science. BMC Medicine, 9, 86.CrossRef
10.
go back to reference Schnipper, L. E., Davidson, N. E., Wollins, D. S., Blayney, D. W., Dicker, A. P., Ganz, P. A., et al. (2016). Updating the American society of clinical oncology value framework: Revisions and reflections in response to comments received. Journal of Clinical Oncology, 34(24), 2925–34.CrossRef Schnipper, L. E., Davidson, N. E., Wollins, D. S., Blayney, D. W., Dicker, A. P., Ganz, P. A., et al. (2016). Updating the American society of clinical oncology value framework: Revisions and reflections in response to comments received. Journal of Clinical Oncology, 34(24), 2925–34.CrossRef
11.
go back to reference Cherny, N. I., Sullivan, R., Dafni, U., Kerst, J. M., Sobrero, A., Zielinski, C., et al. (2015). A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: The European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Annals of Oncology, 26, 1547–73.CrossRef Cherny, N. I., Sullivan, R., Dafni, U., Kerst, J. M., Sobrero, A., Zielinski, C., et al. (2015). A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: The European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Annals of Oncology, 26, 1547–73.CrossRef
13.
go back to reference Food and Drug Administration. (2009). Guidance for Industry: Patient-reported outcome measures: use in medical product development to support labeling claims. Food and Drug Administration. (2009). Guidance for Industry: Patient-reported outcome measures: use in medical product development to support labeling claims.
14.
go back to reference Devlin NJ, Appleby J, Buxton M, Vallance-Owen A. (2010). Getting the most out of PROMS. Putting health outcomes at the heart of NHS decision making. Health Economics. Devlin NJ, Appleby J, Buxton M, Vallance-Owen A. (2010). Getting the most out of PROMS. Putting health outcomes at the heart of NHS decision making. Health Economics.
15.
go back to reference Nivel, Nederland Z. (2014). Handreiking voor het meten van kwaliteit van zorg met Patient Reported Outcome Measures. Nivel, Nederland Z. (2014). Handreiking voor het meten van kwaliteit van zorg met Patient Reported Outcome Measures.
16.
go back to reference Wildiers, H., Mauer, M., Pallis, A., Hurria, A., Mohile, S. G., Luciani, A., et al. (2013). End points and trial design in geriatric oncology research: A joint European Organisation for Research and Treatment of Cancer-Alliance for clinical trials in oncology-international society of geriatric oncology position article. Journal of Clinical Oncology, 31(29), 3711–8.CrossRef Wildiers, H., Mauer, M., Pallis, A., Hurria, A., Mohile, S. G., Luciani, A., et al. (2013). End points and trial design in geriatric oncology research: A joint European Organisation for Research and Treatment of Cancer-Alliance for clinical trials in oncology-international society of geriatric oncology position article. Journal of Clinical Oncology, 31(29), 3711–8.CrossRef
17.
go back to reference Scotté F, Bossi P, Carola E, Cudennec T, Dielenseger P, Gomes F, et al. (2018). Addressing the quality of life needs of older patients with cancer: A SIOG consensus paper and practical guide. Annals of Oncology. Scotté F, Bossi P, Carola E, Cudennec T, Dielenseger P, Gomes F, et al. (2018). Addressing the quality of life needs of older patients with cancer: A SIOG consensus paper and practical guide. Annals of Oncology.
18.
go back to reference Hamaker, M. E., Schulkes, K. J., ten Bokkel, H. D., van Munster, B. C., van Huis, L. H., & van den Bos, F. (2017). Evaluation and reporting of quality of life outcomes in phase III chemotherapy trials for poor prognosis malignancies. Quality of Life Research, 26, 65–71.CrossRef Hamaker, M. E., Schulkes, K. J., ten Bokkel, H. D., van Munster, B. C., van Huis, L. H., & van den Bos, F. (2017). Evaluation and reporting of quality of life outcomes in phase III chemotherapy trials for poor prognosis malignancies. Quality of Life Research, 26, 65–71.CrossRef
19.
go back to reference Schulkes, K. J. G., Nguyen, C., van den Bos, F., Hamaker, M. E., & van Elden, L. J. R. (2016). Patient-centered outcome measures in lung cancer trials. Lung., 194(4), 647–52.CrossRef Schulkes, K. J. G., Nguyen, C., van den Bos, F., Hamaker, M. E., & van Elden, L. J. R. (2016). Patient-centered outcome measures in lung cancer trials. Lung., 194(4), 647–52.CrossRef
20.
go back to reference Hamaker ME, Stauder R, van Munster B. (2013). Ongoing clinical trials in elderly patients with a haematological malignancy: are we addressing the right outcome measures? Journal of Geriatric Oncology. Hamaker ME, Stauder R, van Munster B. (2013). Ongoing clinical trials in elderly patients with a haematological malignancy: are we addressing the right outcome measures? Journal of Geriatric Oncology.
21.
go back to reference van Bekkum ML, van Munster BC, Thunnissen PLM, Smorenburg CH, Hamaker ME. (2015). Current palliative chemotherapy trials in the elderly neglect patient-centred outcome measures. Journal of Geriatric Oncology. van Bekkum ML, van Munster BC, Thunnissen PLM, Smorenburg CH, Hamaker ME. (2015). Current palliative chemotherapy trials in the elderly neglect patient-centred outcome measures. Journal of Geriatric Oncology.
23.
go back to reference MacLeod, S., Musich, S., Hawkins, K., Alsgaard, K., & Wicker, E. R. (2016). The impact of resilience among older adults. Geriatric Nursing (Minneap)., 37, 266–72.CrossRef MacLeod, S., Musich, S., Hawkins, K., Alsgaard, K., & Wicker, E. R. (2016). The impact of resilience among older adults. Geriatric Nursing (Minneap)., 37, 266–72.CrossRef
24.
go back to reference Mercieca-Bebber, R., King, M. T., Calvert, M. J., Stockler, M. R., & Friedlander, M. (2018). The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Related Outcome Measures, 9, 353–67.CrossRef Mercieca-Bebber, R., King, M. T., Calvert, M. J., Stockler, M. R., & Friedlander, M. (2018). The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Related Outcome Measures, 9, 353–67.CrossRef
25.
go back to reference Fayers P, Aaronson N, Bjordal K. (2001). EORTC QLQ-C30 scoring manual. Europen Organization for Research and Treatment of cancer. Fayers P, Aaronson N, Bjordal K. (2001). EORTC QLQ-C30 scoring manual. Europen Organization for Research and Treatment of cancer.
26.
go back to reference Cocks K, King MT, Velikova G, St-James MM, Fayers PM, Brown JM. (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 Clincal Oncology. Cocks K, King MT, Velikova G, St-James MM, Fayers PM, Brown JM. (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 Clincal Oncology.
27.
go back to reference Schwartz, C. E., Bode, R., Repucci, N., Becker, J., Sprangers, M. A. G., & Fayers, P. M. (2006). The clinical significance of adaptation to changing health: A meta-analysis of response shift. Quality of Life Research, 15(9), 1533–50.CrossRef Schwartz, C. E., Bode, R., Repucci, N., Becker, J., Sprangers, M. A. G., & Fayers, P. M. (2006). The clinical significance of adaptation to changing health: A meta-analysis of response shift. Quality of Life Research, 15(9), 1533–50.CrossRef
28.
go back to reference Park SB, Kwok JB, Asher R, Lee CK, Beale P, Selle F, et al. (2017). Clinical and genetic predictors of paclitaxel neurotoxicity based on patient- versus clinicianreported incidence and severity of neurotoxicity in the ICON7 trial. Annals of Oncology, 28(2733–40). Park SB, Kwok JB, Asher R, Lee CK, Beale P, Selle F, et al. (2017). Clinical and genetic predictors of paclitaxel neurotoxicity based on patient- versus clinicianreported incidence and severity of neurotoxicity in the ICON7 trial. Annals of Oncology, 28(2733–40).
31.
go back to reference Calvert, M., Brundage, M., Jacobsen, P. B., Schünemann, H. J., & Efficace, F. (2013). The CONSORT patient-reported outcome (PRO) extension: Implications for clinical trials and practice. Health Quality of Life Outcomes, 11, 184.CrossRef Calvert, M., Brundage, M., Jacobsen, P. B., Schünemann, H. J., & Efficace, F. (2013). The CONSORT patient-reported outcome (PRO) extension: Implications for clinical trials and practice. Health Quality of Life Outcomes, 11, 184.CrossRef
32.
go back to reference Calvert, M., Kyte, D., Mercieca-Bebber, R., Slade, A., Chan, A. W., & King, M. T. (2018). Guidelines for inclusion of patient-reported outcomes in clinical trial protocols the spirit-pro extension. JAMA – Journal of American Medical Association, 319, 483–94.CrossRef Calvert, M., Kyte, D., Mercieca-Bebber, R., Slade, A., Chan, A. W., & King, M. T. (2018). Guidelines for inclusion of patient-reported outcomes in clinical trial protocols the spirit-pro extension. JAMA – Journal of American Medical Association, 319, 483–94.CrossRef
33.
go back to reference Field, J., Holmes, M. M., & Newell, D. (2019). PROMs data: can it be used to make decisions for individual patients? A narrative review. Patient Related Outcome Measures, 10, 233–41.CrossRef Field, J., Holmes, M. M., & Newell, D. (2019). PROMs data: can it be used to make decisions for individual patients? A narrative review. Patient Related Outcome Measures, 10, 233–41.CrossRef
Metagegevens
Titel
Study objectives in clinical trials in older patients with solid malignancies: do we measure what matters?
Auteurs
E. R. M. Scheepers
L. H. van Huis-Tanja
M. H. Emmelot-Vonk
M. E. Hamaker
Publicatiedatum
11-03-2021
Uitgeverij
Springer International Publishing
Gepubliceerd in
Quality of Life Research / Uitgave 7/2021
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-021-02791-8