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Time to deterioration in cancer randomized clinical trials for patient-reported outcomes data: a systematic review

  • E. ChartonEmail author
  • B. Cuer
  • F. Cottone
  • F. Efficace
  • C. Touraine
  • Z. Hamidou
  • F. Fiteni
  • F. Bonnetain
  • M.-C. Woronoff-Lemsi
  • C. Bascoul-Mollevi
  • A. Anota
Review
  • 15 Downloads

Abstract

Purpose

The time to deterioration (TTD) approach has been proposed as a modality of longitudinal analysis of patient-reported outcomes (PROs) in cancer randomized clinical trials (RCTs). The objective of this study was to perform a systematic review of how the TTD approach has been used in phase III RCTs to analyze longitudinal PRO data.

Methods

A systematic literature search was conducted in PubMed/MEDLINE, the Cochrane Library and through manual search to identify studies published between January 2014 and June 2018. All phase III cancer RCTs including a PRO endpoint using the TTD approach were considered. We collected general information about the study, PRO assessment and the TTD approach, such as the event definition, the choice of reference score and whether the deterioration was definitive or not.

Results

A total of 1549 articles were screened, and 39 studies were finally identified as relevant according to predefined criteria. Among these 39 studies, 36 (92.3%) were in advanced and/or metastatic cancer. Several different deterioration definitions were used in RCTs, 10 studies (25.6%) defined the deterioration as “definitive”, corresponding to a deterioration maintained over time until the last PRO assessment available for each patient. The baseline score was explicitly stated as the reference score to qualify the deterioration for most studies (n = 31, 79.5%).

Conclusion

This review highlights the lack of standardization of the TTD approach for the analysis of PRO data in RCTs. Special attention should be paid to the definition of “deterioration”, and this should be based on the specific cancer setting.

Keywords

Patient-reported outcomes Randomized clinical trials Time to deterioration Systematic review 

Notes

Acknowledgements

This work was supported by a grant from the “Institut National du Cancer (INCA 11862)”. The authors thank François Calais (University of Franche-Comté, Besançon, France) for developing and executing the search strategy. The authors thank Fiona Ecarnot (EA3920, University Hospital Besançon, University of Franche-Comté, Besançon, France) for editorial assistance.

Funding

This work was funded by the “Institut National du Cancer (Grant INCA 11862)”.

Compliance with ethical standards

Conflict of interest

The authors declare that they have no conflict of interest.

Ethical approval

This article does not contain any studies with human participants or animals performed by any of the authors.

Supplementary material

11136_2019_2367_MOESM1_ESM.docx (36 kb)
Supplementary material 1 (DOCX 38 kb)

References

  1. 1.
    Efficace, F., Fayers, P., Pusic, A., Cemal, Y., Yanagawa, J., Jacobs, M., et al. (2015). Quality of patient-reported outcome reporting across cancer randomized controlled trials according to the CONSORT patient-reported outcome extension: A pooled analysis of 557 trials. Cancer,121(18), 3335–3342.  https://doi.org/10.1002/cncr.29489.CrossRefPubMedPubMedCentralGoogle Scholar
  2. 2.
    Secord, A. A., Coleman, R. L., Havrilesky, L. J., Abernethy, A. P., Samsa, G. P., & Cella, D. (2015). Patient-reported outcomes as end points and outcome indicators in solid tumours. Nature Reviews. Clinical Oncology,12(6), 358–370.  https://doi.org/10.1038/nrclinonc.2015.29.CrossRefPubMedGoogle Scholar
  3. 3.
    Bonnetain, F., Fiteni, F., Efficace, F., & Anota, A. (2016). Statistical challenges in the analysis of health-related quality of life in cancer clinical trials. Journal of Clinical Oncology,34(16), 1953–1956.  https://doi.org/10.1200/JCO.2014.56.7974.CrossRefPubMedGoogle Scholar
  4. 4.
    Cnaan, A., Laird, N. M., & Slasor, P. (1997). Using the general linear mixed model to analyse unbalanced repeated measures and longitudinal data. Statistics in Medicine,16(20), 2349–2380.CrossRefGoogle Scholar
  5. 5.
    Fairclough, D. L. (2010). Design and analysis of quality of life studies in clinical trials. London: Chapman and Hall/CRC.CrossRefGoogle Scholar
  6. 6.
    Anota, A., Hamidou, Z., Paget-Bailly, S., Chibaudel, B., Bascoul-Mollevi, C., Auquier, P., 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.  https://doi.org/10.1007/s11136-013-0583-6.CrossRefPubMedGoogle Scholar
  7. 7.
    Bonnetain, F., Dahan, L., Maillard, E., Ychou, M., Mitry, E., Hammel, P., 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.  https://doi.org/10.1016/j.ejca.2010.07.023.CrossRefPubMedGoogle Scholar
  8. 8.
    Hamidou, Z., Dabakuyo, T. S., Mercier, M., Fraisse, J., Causeret, S., Tixier, H., et al. (2011). Time to deterioration in quality of life score as a modality of longitudinal analysis in patients with breast cancer. The Oncologist,16(10), 1458–1468.  https://doi.org/10.1634/theoncologist.2011-0085.CrossRefPubMedPubMedCentralGoogle Scholar
  9. 9.
    Cortes, J., Hudgens, S., Twelves, C., Perez, E. A., Awada, A., Yelle, L., et al. (2015). Health-related quality of life in patients with locally advanced or metastatic breast cancer treated with eribulin mesylate or capecitabine in an open-label randomized phase 3 trial. Breast Cancer Research and Treatment,154(3), 509–520.  https://doi.org/10.1007/s10549-015-3633-7.CrossRefPubMedPubMedCentralGoogle Scholar
  10. 10.
    Verma, S., O’Shaughnessy, J., Burris, H. A., Campone, M., Alba, E., Chandiwana, D., et al. (2018). Health-related quality of life of postmenopausal women with hormone receptor-positive, human epidermal growth factor receptor 2-negative advanced breast cancer treated with ribociclib + letrozole: Results from MONALEESA-2. Breast Cancer Research and Treatment,170(3), 535–545.  https://doi.org/10.1007/s10549-018-4769-z.CrossRefPubMedPubMedCentralGoogle Scholar
  11. 11.
    Mathoulin-Pelissier, S., Gourgou-Bourgade, S., Bonnetain, F., & Kramar, A. (2008). Survival end point reporting in randomized cancer clinical trials: A review of major journals. Journal of Clinical Oncology,26(22), 3721–3726.  https://doi.org/10.1200/JCO.2007.14.1192.CrossRefPubMedGoogle Scholar
  12. 12.
    Higgins, J., & Green, S. (2014). Cochrane handbook for systematic reviews of interventions Version 5.1. 0 [updated March 2011]. 2011. The Cochrane Collaboration.Google Scholar
  13. 13.
    Al-Batran, S. E., Van Cutsem, E., Oh, S. C., Bodoky, G., Shimada, Y., Hironaka, S., et al. (2016). Quality-of-life and performance status results from the phase III RAINBOW study of ramucirumab plus paclitaxel versus placebo plus paclitaxel in patients with previously treated gastric or gastroesophageal junction adenocarcinoma. Annals of Oncology,27(4), 673–679.  https://doi.org/10.1093/annonc/mdv625.CrossRefPubMedPubMedCentralGoogle Scholar
  14. 14.
    Bang, Y.-J., Xu, R.-H., Chin, K., Lee, K.-W., Park, S. H., Rha, S. Y., et al. (2017). Olaparib in combination with paclitaxel in patients with advanced gastric cancer who have progressed following first-line therapy (GOLD): A double-blind, randomised, placebo-controlled, phase 3 trial. The Lancet Oncology,18(12), 1637–1651.  https://doi.org/10.1016/S1470-2045(17)30682-4.CrossRefPubMedGoogle Scholar
  15. 15.
    Bascoul-Mollevi, C., Gourgou, S., Galais, M. P., Raoul, J. L., Bouche, O., Douillard, J. Y., et al. (2017). Health-related quality of life results from the PRODIGE 5/ACCORD 17 randomised trial of FOLFOX versus fluorouracil-cisplatin regimen in oesophageal cancer. European Journal of Cancer,84, 239–249.  https://doi.org/10.1016/j.ejca.2017.07.038.CrossRefPubMedGoogle Scholar
  16. 16.
    Bodoky, G., Scheulen, M. E., Rivera, F., Jassem, J., Carrato, A., Moiseyenko, V., et al. (2015). Clinical benefit and health-related quality of life assessment in patients treated with cisplatin/S-1 versus cisplatin/5-FU: Secondary end point results from the First-Line Advanced Gastric Cancer Study (FLAGS). Journal of Gastrointestinal Cancer,46(2), 109–117.  https://doi.org/10.1007/s12029-014-9680-1.CrossRefPubMedGoogle Scholar
  17. 17.
    Boye, M., Wang, X., Srimuninnimit, V., Kang, J. H., Tsai, C.-M., Orlando, M., et al. (2016). First-line pemetrexed plus cisplatin followed by gefitinib maintenance therapy versus gefitinib monotherapy in East Asian never-smoker patients with locally advanced or metastatic nonsquamous non-small-cell lung cancer: Quality of life results from a randomized phase III trial. Clinical Lung Cancer,17(2), 150–160.  https://doi.org/10.1016/j.cllc.2015.12.004.CrossRefPubMedGoogle Scholar
  18. 18.
    Brahmer, J. R., Rodríguez-Abreu, D., Robinson, A. G., Hui, R., Csőszi, T., Fülöp, A., et al. (2017). Health-related quality-of-life results for pembrolizumab versus chemotherapy in advanced, PD-L1-positive NSCLC (KEYNOTE-024): A multicentre, international, randomised, open-label phase 3 trial. The Lancet Oncology,18(12), 1600–1609.  https://doi.org/10.1016/S1470-2045(17)30690-3.CrossRefPubMedGoogle Scholar
  19. 19.
    Cella, D., Escudier, B., Tannir, N. M., Powles, T., Donskov, F., Peltola, K., et al. (2018). Quality of life outcomes for cabozantinib versus everolimus in patients with metastatic renal cell carcinoma: METEOR phase III randomized trial. Journal of Clinical Oncology,36(8), 757–764.  https://doi.org/10.1200/JCO.2017.75.2170.CrossRefPubMedPubMedCentralGoogle Scholar
  20. 20.
    Chi, K. N., Protheroe, A., Rodríguez-Antolín, A., Facchini, G., Suttman, H., Matsubara, N., et al. (2018). Patient-reported outcomes following abiraterone acetate plus prednisone added to androgen deprivation therapy in patients with newly diagnosed metastatic castration-naive prostate cancer (LATITUDE): An international, randomised phase 3 trial. The Lancet Oncology,19(2), 194–206.  https://doi.org/10.1016/S1470-2045(17)30911-7.CrossRefPubMedGoogle Scholar
  21. 21.
    Ellis, P. M., Shepherd, F. A., Millward, M., Perrone, F., Seymour, L., Liu, G., et al. (2014). Dacomitinib compared with placebo in pretreated patients with advanced or metastatic non-small-cell lung cancer (NCIC CTG BR.26): A double-blind, randomised, phase 3 trial. The Lancet Oncology,15(12), 1379–1388.  https://doi.org/10.1016/s1470-2045(14)70472-3.CrossRefPubMedGoogle Scholar
  22. 22.
    Felip, E., Hirsh, V., Popat, S., Cobo, M., Fülöp, A., Dayen, C., et al. (2018). Symptom and quality of life improvement in LUX-Lung 8, an open-label phase III study of second-line afatinib versus erlotinib in patients with advanced Squamous Cell carcinoma of the lung after first-line platinum-based chemotherapy. Clinical Lung Cancer,19(1), 74–83.e11.  https://doi.org/10.1016/j.cllc.2017.06.002.CrossRefPubMedGoogle Scholar
  23. 23.
    Fiteni, F., Anota, A., Bonnetain, F., Oster, J.-P., Pichon, E., Wislez, M., et al. (2016). Health-related quality of life in elderly patients with advanced non-small cell lung cancer comparing carboplatin and weekly paclitaxel doublet chemotherapy with monotherapy. The European Respiratory Journal,48(3), 861–872.  https://doi.org/10.1183/13993003.01695-2015.CrossRefPubMedGoogle Scholar
  24. 24.
    Fizazi, K., Scher, H. I., Miller, K., Basch, E., Sternberg, C. N., Cella, D., et al. (2014). Effect of enzalutamide on time to first skeletal-related event, pain, and quality of life in men with castration-resistant prostate cancer: Results from the randomised, phase 3 AFFIRM trial. The Lancet Oncology,15(10), 1147–1156.  https://doi.org/10.1016/S1470-2045(14)70303-1.CrossRefPubMedGoogle Scholar
  25. 25.
    Geater, S. L., Xu, C. R., Zhou, C., Hu, C. P., Feng, J., Lu, S., et al. (2015). Symptom and quality of life improvement in LUX-Lung 6: An open-label phase III study of afatinib versus cisplatin/gemcitabine in asian patients with EGFR mutation-positive advanced non-small-cell lung cancer. Journal of Thoracic Oncology,10(6), 883–889.  https://doi.org/10.1097/JTO.0000000000000517.CrossRefPubMedGoogle Scholar
  26. 26.
    Gill, S., Ko, Y. J., Cripps, C., Beaudoin, A., Dhesy-Thind, S., Zulfiqar, M., et al. (2016). PANCREOX: A randomized phase III study of fluorouracil/leucovorin with or without oxaliplatin for second-line advanced pancreatic cancer in patients who have received gemcitabine-based chemotherapy. Journal of Clinical Oncology,34(32), 3914–3920.  https://doi.org/10.1200/JCO.2016.68.5776.CrossRefPubMedGoogle Scholar
  27. 27.
    Guimbaud, R., Louvet, C., Ries, P., Ychou, M., Maillard, E., Andre, T., et al. (2014). Prospective, randomized, multicenter, phase III study of fluorouracil, leucovorin, and irinotecan versus epirubicin, cisplatin, and capecitabine in advanced gastric adenocarcinoma: A French intergroup (Federation Francophone de Cancerologie Digestive, Federation Nationale des Centres de Lutte Contre le Cancer, and Groupe Cooperateur Multidisciplinaire en Oncologie) study. Journal of Clinical Oncology,32(31), 3520–3526.  https://doi.org/10.1200/JCO.2013.54.1011.CrossRefPubMedGoogle Scholar
  28. 28.
    Hagiwara, Y., Ohashi, Y., Okusaka, T., Ueno, H., Ioka, T., Boku, N., et al. (2017). Health-related quality of life in a randomised phase III study of gemcitabine plus S-1, S-1 alone and gemcitabine alone for locally advanced or metastatic pancreatic cancer: GEST study. ESMO Open,2(1), e000151.  https://doi.org/10.1136/esmoopen-2016-000151.CrossRefPubMedPubMedCentralGoogle Scholar
  29. 29.
    Hamidou, Z., Chibaudel, B., Hebbar, M., Hug de Larauze, M., André, T., Louvet, C., et al. (2016). Time to definitive health-related quality of life score deterioration in patients with resectable metastatic colorectal cancer treated with FOLFOX4 versus sequential dose-dense FOLFOX7 followed by FOLFIRI: The MIROX randomized phase III trial. PLoS ONE,11(6), e0157067.  https://doi.org/10.1371/journal.pone.0157067.CrossRefPubMedPubMedCentralGoogle Scholar
  30. 30.
    Harbeck, N., Iyer, S., Turner, N., Cristofanilli, M., Ro, J., Andre, F., et al. (2016). Quality of life with palbociclib plus fulvestrant in previously treated hormone receptor-positive, HER2-negative metastatic breast cancer: Patient-reported outcomes from the PALOMA-3 trial. Annals of Oncology,27(6), 1047–1054.  https://doi.org/10.1093/annonc/mdw139.CrossRefPubMedPubMedCentralGoogle Scholar
  31. 31.
    Harrington, K. J., Ferris, R. L., Blumenschein, G., Colevas, A. D., Fayette, J., Licitra, L., et al. (2017). Nivolumab versus standard, single-agent therapy of investigator’s choice in recurrent or metastatic squamous cell carcinoma of the head and neck (CheckMate 141): Health-related quality-of-life results from a randomised, phase 3 trial. The Lancet Oncology,18(8), 1104–1115.  https://doi.org/10.1016/S1470-2045(17)30421-7.CrossRefPubMedPubMedCentralGoogle Scholar
  32. 32.
    Lee, C. K., Novello, S., Rydén, A., Mann, H., & Mok, T. (2018). Patient-reported symptoms and impact of treatment with osimertinib versus chemotherapy in advanced non-small-cell lung cancer: The AURA3 trial. Journal of Clinical Oncology,36(18), 1853–1860.  https://doi.org/10.1200/JCO.2017.77.2293.CrossRefPubMedGoogle Scholar
  33. 33.
    Long, G. V., Atkinson, V., Ascierto, P. A., Robert, C., Hassel, J. C., Rutkowski, P., et al. (2016). Effect of nivolumab on health-related quality of life in patients with treatment-naive advanced melanoma: Results from the phase III CheckMate 066 study. Annals of Oncology,27(10), 1940–1946.  https://doi.org/10.1093/annonc/mdw265.CrossRefPubMedPubMedCentralGoogle Scholar
  34. 34.
    Loriot, Y., Miller, K., Sternberg, C. N., Fizazi, K., De Bono, J. S., Chowdhury, S., et al. (2015). Effect of enzalutamide on health-related quality of life, pain, and skeletal-related events in asymptomatic and minimally symptomatic, chemotherapy-naive patients with metastatic castration-resistant prostate cancer (PREVAIL): Results from a randomised, phase 3 trial. The Lancet Oncology,16(5), 509–521.  https://doi.org/10.1016/S1470-2045(15)70113-0.CrossRefPubMedGoogle Scholar
  35. 35.
    Machiels, J.-P. H., Haddad, R. I., Fayette, J., Licitra, L. F., Tahara, M., Vermorken, J. B., et al. (2015). Afatinib versus methotrexate as second-line treatment in patients with recurrent or metastatic squamous-cell carcinoma of the head and neck progressing on or after platinum-based therapy (LUX-Head & Neck 1): An open-label, randomised phase 3 trial. The Lancet Oncology,16(5), 583–594.  https://doi.org/10.1016/S1470-2045(15)70124-5.CrossRefPubMedGoogle Scholar
  36. 36.
    Taphoorn, M. J., Dirven, L., Kanner, A. A., Lavy-Shahaf, G., Weinberg, U., Taillibert, S., et al. (2018). Influence of treatment with tumor-treating fields on health-related quality of life of patients with newly diagnosed glioblastoma: A secondary analysis of a randomized clinical trial. JAMA Oncology,4(4), 495–504.  https://doi.org/10.1001/jamaoncol.2017.5082.CrossRefPubMedPubMedCentralGoogle Scholar
  37. 37.
    Novello, S., Kaiser, R., Mellemgaard, A., Douillard, J. Y., Orlov, S., Krzakowski, M., et al. (2015). Analysis of patient-reported outcomes from the LUME-Lung 1 trial: A randomised, double-blind, placebo-controlled, Phase III study of second-line nintedanib in patients with advanced non-small cell lung cancer. European Journal of Cancer,51(3), 317–326.  https://doi.org/10.1016/j.ejca.2014.11.015.CrossRefPubMedGoogle Scholar
  38. 38.
    Pavel, M. E., Singh, S., Strosberg, J. R., Bubuteishvili-Pacaud, L., Degtyarev, E., Neary, M. P., et al. (2017). Health-related quality of life for everolimus versus placebo in patients with advanced, non-functional, well-differentiated gastrointestinal or lung neuroendocrine tumours (RADIANT-4): A multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet Oncology,18(10), 1411–1422.  https://doi.org/10.1016/S1470-2045(17)30471-0.CrossRefPubMedGoogle Scholar
  39. 39.
    Perol, M., Ciuleanu, T. E., Arrieta, O., Prabhash, K., Syrigos, K. N., Goksel, T., et al. (2016). Quality of life results from the phase 3 REVEL randomized clinical trial of ramucirumab-plus-docetaxel versus placebo-plus-docetaxel in advanced/metastatic non-small cell lung cancer patients with progression after platinum-based chemotherapy. Lung Cancer,93, 95–103.  https://doi.org/10.1016/j.lungcan.2016.01.007.CrossRefPubMedGoogle Scholar
  40. 40.
    Petrella, T. M., Robert, C., Richtig, E., Miller, W. H., Jr., Masucci, G. V., Walpole, E., et al. (2017). Patient-reported outcomes in KEYNOTE-006, a randomised study of pembrolizumab versus ipilimumab in patients with advanced melanoma. European Journal of Cancer,86, 115–124.  https://doi.org/10.1016/j.ejca.2017.08.032.CrossRefPubMedGoogle Scholar
  41. 41.
    Qin, S., Bi, F., Jin, J., Cheng, Y., Guo, J., Ren, X., et al. (2015). Axitinib versus sorafenib as a second-line therapy in Asian patients with metastatic renal cell carcinoma: Results from a randomized registrational study. OncoTargets and Therapy,8, 1363–1373.  https://doi.org/10.2147/OTT.S83302.CrossRefPubMedPubMedCentralGoogle Scholar
  42. 42.
    Reck, M., Taylor, F., Penrod, J. R., DeRosa, M., Morrissey, L., Dastani, H., et al. (2018). Impact of nivolumab versus docetaxel on health-related quality of life and symptoms in patients with advanced squamous non-small cell lung cancer: results from the CheckMate 017 study. Journal of Thoracic Oncology,13(2), 194–204.  https://doi.org/10.1016/j.jtho.2017.10.029.CrossRefPubMedGoogle Scholar
  43. 43.
    Saad, F., Fizazi, K., Jinga, V., Efstathiou, E., Fong, P. C., Hart, L. L., et al. (2015). Orteronel plus prednisone in patients with chemotherapy-naive metastatic castration-resistant prostate cancer (ELM-PC 4): A double-blind, multicentre, phase 3, randomised, placebo-controlled trial. The Lancet Oncology,16(3), 338–348.  https://doi.org/10.1016/S1470-2045(15)70027-6.CrossRefPubMedGoogle Scholar
  44. 44.
    Satoh, T., Bang, Y.-J., Gotovkin, E. A., Hamamoto, Y., Kang, Y.-K., Moiseyenko, V. M., et al. (2014). Quality of life in the trastuzumab for gastric cancer trial. The Oncologist,19(7), 712–719.  https://doi.org/10.1634/theoncologist.2014-0058.CrossRefPubMedPubMedCentralGoogle Scholar
  45. 45.
    Shiroiwa, T., Fukuda, T., Shimozuma, K., Mouri, M., Hagiwara, Y., Doihara, H., et al. (2017). Long-term health status as measured by EQ-5D among patients with metastatic breast cancer: Comparison of first-line oral S-1 and taxane therapies in the randomized phase III SELECT BC trial. Quality of Life Research,26(2), 445–453.  https://doi.org/10.1007/s11136-016-1388-1.CrossRefPubMedGoogle Scholar
  46. 46.
    Taphoorn, M. J., Henriksson, R., Bottomley, A., Cloughesy, T., Wick, W., Mason, W. P., et al. (2015). Health-related quality of life in a randomized phase III study of bevacizumab, temozolomide, and radiotherapy in newly diagnosed glioblastoma. Journal of Clinical Oncology,33(19), 2166–2175.  https://doi.org/10.1200/JCO.2014.60.3217.CrossRefPubMedGoogle Scholar
  47. 47.
    Vaughn, D. J., Bellmunt, J., Fradet, Y., Lee, J. L., Fong, L., Vogelzang, N. J., et al. (2018). Health-related quality-of-life analysis from KEYNOTE-045: A phase III study of pembrolizumab versus chemotherapy for previously treated advanced urothelial cancer. Journal of Clinical Oncology,36(16), 1579–1587.  https://doi.org/10.1200/JCO.2017.76.9562.CrossRefPubMedGoogle Scholar
  48. 48.
    Vinik, A., Bottomley, A., Korytowsky, B., Bang, Y.-J., Raoul, J.-L., Valle, J. W., et al. (2016). Patient-reported outcomes and quality of life with sunitinib versus placebo for pancreatic neuroendocrine tumors: Results from an international phase III trial. Targeted Oncology,11(6), 815–824.  https://doi.org/10.1007/s11523-016-0462-5.CrossRefPubMedGoogle Scholar
  49. 49.
    Yang, Y.-P., Ma, Y.-X., Huang, Y., Zhao, Y.-Y., Fang, W.-F., Hong, S.-D., et al. (2015). QoL analyses from INFORM study, a phase III study of gefitinib versus placebo as maintenance therapy in advanced NSCLC. Scientific Reports,5, 11934.  https://doi.org/10.1038/srep11934.CrossRefPubMedPubMedCentralGoogle Scholar
  50. 50.
    Fiteni, F., Westeel, V., Pivot, X., Borg, C., Vernerey, D., & Bonnetain, F. (2014). Endpoints in cancer clinical trials. Journal of Visceral Surgery,151(1), 17–22.CrossRefGoogle Scholar
  51. 51.
    Montori, V. M., Permanyer-Miralda, G., Ferreira-González, I., Busse, J. W., Pacheco-Huergo, V., Bryant, D., et al. (2005). Validity of composite end points in clinical trials. BMJ (Clinical Research ed.),330(7491), 594–596.  https://doi.org/10.1136/bmj.330.7491.594.CrossRefGoogle Scholar
  52. 52.
    Freemantle, N., Calvert, M., Wood, J., Eastaugh, J., & Griffin, C. (2003). Composite outcomes in randomized trials: Greater precision but with greater uncertainty? JAMA,289(19), 2554–2559.  https://doi.org/10.1001/jama.289.19.2554.CrossRefPubMedGoogle Scholar
  53. 53.
    Anota, A., Savina, M., Bascoul-Mollevi, C., & Bonnetain, F. (2017). Qolr: An r package for the longitudinal analysis of health-related quality of life in oncology. Journal of Statistical Software, Articles,77(12), 1–30.Google Scholar
  54. 54.
    Bottomley, A., Pe, M., Sloan, J., Basch, E., Bonnetain, F., Calvert, M., et al. (2016). Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: A start in setting international standards. The Lancet Oncology,17(11), e510–e514.  https://doi.org/10.1016/S1470-2045(16)30510-1.CrossRefPubMedGoogle Scholar
  55. 55.
    Ajani, J. A., Moiseyenko, V. M., Tjulandin, S., Majlis, A., Constenla, M., Boni, C., et al. (2007). Quality of life with docetaxel plus cisplatin and fluorouracil compared with cisplatin and fluorouracil from a phase III trial for advanced gastric or gastroesophageal adenocarcinoma: The V-325 Study Group. Journal of Clinical Oncology,25(22), 3210–3216.  https://doi.org/10.1200/JCO.2006.08.3956.CrossRefPubMedGoogle Scholar
  56. 56.
    Panageas, K. S., Ben-Porat, L., Dickler, M. N., Chapman, P. B., & Schrag, D. (2007). When you look matters: The effect of assessment schedule on progression-free survival. Journal of the National Cancer Institute,99(6), 428–432.  https://doi.org/10.1093/jnci/djk091.CrossRefPubMedGoogle Scholar
  57. 57.
    Kiebert, G. M., Curran, D., & Aaronson, N. K. (1998). Quality of life as an endpoint in EORTC clinical trials. European Organization for Research and Treatment for Cancer. Statistics in Medicine,17(5–7), 561–569.CrossRefGoogle Scholar
  58. 58.
    Fiteni, F., Pam, A., Anota, A., Vernerey, D., Paget-Bailly, S., Westeel, V., et al. (2015). Health-related quality-of-life as co-primary endpoint in randomized clinical trials in oncology. Expert Review of Anticancer Therapy,15(8), 885–891.CrossRefGoogle Scholar

Copyright information

© Springer Nature Switzerland AG 2019

Authors and Affiliations

  • E. Charton
    • 1
    • 2
    Email author
  • B. Cuer
    • 3
    • 4
  • F. Cottone
    • 5
  • F. Efficace
    • 5
  • C. Touraine
    • 3
  • Z. Hamidou
    • 6
    • 7
  • F. Fiteni
    • 3
    • 8
  • F. Bonnetain
    • 1
    • 2
    • 7
  • M.-C. Woronoff-Lemsi
    • 2
    • 9
  • C. Bascoul-Mollevi
    • 3
    • 4
    • 7
  • A. Anota
    • 1
    • 2
    • 7
  1. 1.Methodology and Quality of Life Unit in Oncology (INSERM UMR 1098)University Hospital of BesançonBesançonFrance
  2. 2.University Bourgogne Franche-Comté, INSERM, EFS BFC, UMR1098, Interactions Hôte-Greffon-Tumeur/Ingénierie Cellulaire et GéniqueBesançonFrance
  3. 3.Montpellier Cancer Institute (ICM)University of MontpellierMontpellierFrance
  4. 4.Institute of Cancer Research of Montpellier (IRCM), ICM, INSERMMontpellierFrance
  5. 5.Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA)RomeItaly
  6. 6.EA3279 Self-perceived Health Assessment Research UnitAix-Marseille UniversityMarseilleFrance
  7. 7.French National Platform Quality of Life and CancerBesançonFrance
  8. 8.Department of Medical OncologyUniversity Hospital of NîmesNîmesFrance
  9. 9.Department of Clinical Research and InnovationUniversity Hospital of BesançonBesançonFrance

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