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

Publisher: Springer Netherlands Country of Publication: Netherlands NLM ID: 9210257 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2649 (Electronic) Linking ISSN: 09629343 NLM ISO Abbreviation: Qual Life Res Subsets: MEDLINE

Publication: 2005- : Netherlands : Springer Netherlands
Original Publication: Oxford, UK : Rapid Communications of Oxford, Ltd, c1992-

Clinical Deterioration* ; Patient Reported Outcome Measures*; Neoplasms/*pathology ; Neoplasms/*therapy; Humans ; Quality of Life ; Randomized Controlled Trials as Topic

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.

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 . (PMID: 10.1002/cncr.29489260791974755292)
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 . (PMID: 10.1038/nrclinonc.2015.2925754949)
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 . (PMID: 10.1200/JCO.2014.56.797427091712)
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. (PMID: 10.1002/(SICI)1097-0258(19971030)16:20<2349::AID-SIM667>3.0.CO;2-E)
Fairclough, D. L. (2010). Design and analysis of quality of life studies in clinical trials. London: Chapman and Hall/CRC. (PMID: 10.1201/9781420061185)
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 . (PMID: 10.1007/s11136-013-0583-624277234)
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 . (PMID: 10.1016/j.ejca.2010.07.02320724140)
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 . (PMID: 10.1634/theoncologist.2011-0085219486503228064)
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 . (PMID: 10.1007/s10549-015-3633-7265670104661183)
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 . (PMID: 10.1007/s10549-018-4769-z296544156022531)
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 . (PMID: 10.1200/JCO.2007.14.119218669458)
Higgins, J., & Green, S. (2014). Cochrane handbook for systematic reviews of interventions Version 5.1. 0 [updated March 2011]. 2011. The Cochrane Collaboration.
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 . (PMID: 10.1093/annonc/mdv625267478594803452)
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 . (PMID: 10.1016/S1470-2045(17)30682-429103871)
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 . (PMID: 10.1016/j.ejca.2017.07.03828829992)
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 . (PMID: 10.1007/s12029-014-9680-125707610)
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 . (PMID: 10.1016/j.cllc.2015.12.00426809984)
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 . (PMID: 10.1016/S1470-2045(17)30690-329129441)
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 . (PMID: 10.1200/JCO.2017.75.2170293777556804841)
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 . (PMID: 10.1016/S1470-2045(17)30911-729326030)
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 . (PMID: 10.1016/s1470-2045(14)70472-325439692)
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 . (PMID: 10.1016/j.cllc.2017.06.00228729180)
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 . (PMID: 10.1183/13993003.01695-201527338193)
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 . (PMID: 10.1016/S1470-2045(14)70303-125104109)
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 . (PMID: 10.1097/JTO.000000000000051725933111)
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 . (PMID: 10.1200/JCO.2016.68.577627621395)
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 . (PMID: 10.1200/JCO.2013.54.101125287828)
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 . (PMID: 10.1136/esmoopen-2016-000151287617315519786)
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 . (PMID: 10.1371/journal.pone.0157067273102054910973)
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 . (PMID: 10.1093/annonc/mdw139270297044880065)
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 . (PMID: 10.1016/S1470-2045(17)30421-7286519296461049)
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 . (PMID: 10.1200/JCO.2017.77.229329733770)
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 . (PMID: 10.1093/annonc/mdw265274053225035785)
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 . (PMID: 10.1016/S1470-2045(15)70113-025888263)
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 . (PMID: 10.1016/S1470-2045(15)70124-525892145)
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 . (PMID: 10.1001/jamaoncol.2017.5082293922805885193)
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 . (PMID: 10.1016/j.ejca.2014.11.01525534294)
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 . (PMID: 10.1016/S1470-2045(17)30471-028838862)
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 . (PMID: 10.1016/j.lungcan.2016.01.00726898621)
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 . (PMID: 10.1016/j.ejca.2017.08.03228987768)
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 . (PMID: 10.2147/OTT.S83302260896864467642)
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 . (PMID: 10.1016/j.jtho.2017.10.02929129758)
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 . (PMID: 10.1016/S1470-2045(15)70027-625701170)
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 . (PMID: 10.1634/theoncologist.2014-0058249516094077451)
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 . (PMID: 10.1007/s11136-016-1388-127517267)
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 . (PMID: 10.1200/JCO.2014.60.321726014298)
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 . (PMID: 10.1200/JCO.2017.76.956229590008)
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 . (PMID: 10.1007/s11523-016-0462-527924459)
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 . (PMID: 10.1038/srep11934261379164490396)
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. (PMID: 10.1016/j.jviscsurg.2013.10.001)
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 . (PMID: 10.1136/bmj.330.7491.594)
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 . (PMID: 10.1001/jama.289.19.255412759327)
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.
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 . (PMID: 10.1016/S1470-2045(16)30510-127769798)
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 . (PMID: 10.1200/JCO.2006.08.395617664468)
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 . (PMID: 10.1093/jnci/djk09117374832)
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. (PMID: 10.1002/(SICI)1097-0258(19980315/15)17:5/7<561::AID-SIM803>3.0.CO;2-S)
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. (PMID: 10.1586/14737140.2015.1047768)

INCA 11862 Institut National du Cancer

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

Date Created: 20191129 Date Completed: 20200629 Latest Revision: 20210321

20221213

10.1007/s11136-019-02367-7

31776827