International Council for Harmonisation (2019) Addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials
Enrico D, Waisberg F, Burton J et al (2021) Analysis of adverse events attribution and reporting in cancer clinical trials: a systematic review. Crit Rev Oncol Hematol 160:103296. https://doi.org/10.1016/j.critrevonc.2021.103296
Péron J, Maillet D, Gan HK et al (2013) Adherence to CONSORT adverse event reporting guidelines in randomized clinical trials evaluating systemic cancer therapy: a systematic review. J Clin Oncol off J Am Soc Clin Oncol 31:3957–3963. https://doi.org/10.1200/JCO.2013.49.3981
Sivendran S, Latif A, McBride RB et al (2014) Adverse event reporting in cancer clinical trial publications. J Clin Oncol off J Am Soc Clin Oncol 32:83–89. https://doi.org/10.1200/JCO.2013.52.2219
Maillet D, Blay JY, You B et al (2016) The reporting of adverse events in oncology phase III trials: a comparison of the current status versus the expectations of the EORTC members. Ann Oncol off J Eur Soc Med Oncol 27:192–198. https://doi.org/10.1093/annonc/mdv485
Thanarajasingam G, Hubbard JM, Sloan JA, Grothey A (2015) The Imperative for a New Approach to Toxicity Analysis in Oncology clinical trials. J Natl Cancer Inst 107:djv216. https://doi.org/10.1093/jnci/djv216
Lopes GS, Tournigand C, Olswold CL et al (2021) Adverse event load, onset, and maximum grade: a novel method of reporting adverse events in cancer clinical trials. Clin Trials Lond Engl 18:51–60. https://doi.org/10.1177/1740774520959313
Motzer RJ, Hutson TE, Cella D et al (2013) Pazopanib versus sunitinib in metastatic renal-cell carcinoma. N Engl J Med 369:722–731. https://doi.org/10.1056/NEJMoa1303989
Article CAS PubMed Google Scholar
Brahmer JR, Lacchetti C, Schneider BJ et al (2018) Management of Immune-related adverse events in patients treated with Immune checkpoint inhibitor therapy: American Society of Clinical Oncology Clinical Practice Guideline. J Clin Oncol off J Am Soc Clin Oncol 36:1714–1768. https://doi.org/10.1200/JCO.2017.77.6385
Weber JS, Hodi FS, Wolchok JD et al (2017) Safety Profile of Nivolumab Monotherapy: a pooled analysis of patients with Advanced Melanoma. J Clin Oncol off J Am Soc Clin Oncol 35:785–792. https://doi.org/10.1200/JCO.2015.66.1389
Cabarrou B, Boher JM, Bogart E et al (2016) How to report toxicity associated with targeted therapies? Ann Oncol off J Eur Soc Med Oncol 27:1633–1638. https://doi.org/10.1093/annonc/mdw218
Gong Q, Tong B, Strasak A, Fang L (2014) Analysis of safety data in clinical trials using a recurrent event approach. Pharm Stat 13:136–144. https://doi.org/10.1002/pst.1611
Hengelbrock J, Gillhaus J, Kloss S, Leverkus F (2016) Safety data from randomized controlled trials: applying models for recurrent events. Pharm Stat 15:315–323. https://doi.org/10.1002/pst.1757
Major PP, Cook RJ, Lipton A et al (2009) Natural history of malignant bone disease in breast cancer and the use of cumulative mean functions to measure skeletal morbidity. BMC Cancer 9:272. https://doi.org/10.1186/1471-2407-9-272
Article PubMed PubMed Central Google Scholar
Cook RJ, Major P (2006) Multistate Analysis of Skeletal Events in patients with bone metastases. Clin Cancer Res 12:6264s–6269s. https://doi.org/10.1158/1078-0432.CCR-06-0654
Phillips R, Sauzet O, Cornelius V (2020) Statistical methods for the analysis of adverse event data in randomised controlled trials: a scoping review and taxonomy. BMC Med Res Methodol 20:288. https://doi.org/10.1186/s12874-020-01167-9
Article PubMed PubMed Central Google Scholar
Brahmer JR, Abu-Sbeih H, Ascierto PA et al (2021) Society for Immunotherapy of Cancer (SITC) clinical practice guideline on immune checkpoint inhibitor-related adverse events. J Immunother Cancer 9:e002435. https://doi.org/10.1136/jitc-2021-002435
Article PubMed PubMed Central Google Scholar
Cabarrou B, Gomez-Roca C, Viala M et al (2020) Modernizing adverse events analysis in oncology clinical trials using alternative approaches: rationale and design of the MOTIVATE trial. Invest New Drugs 38:1879–1887. https://doi.org/10.1007/s10637-020-00938-x
Pepe MS, Longton G, Thornquist M (1991) A qualifier Q for the survival function to describe the prevalence of a transient condition. Stat Med 10:413–421. https://doi.org/10.1002/sim.4780100313
Article CAS PubMed Google Scholar
Prentice RL, Kalbfleisch JD, Peterson AV et al (1978) The analysis of failure times in the presence of competing risks. Biometrics 34:541–554
Article CAS PubMed Google Scholar
Ghosh D, Lin DY (2000) Nonparametric analysis of recurrent events and death. Biometrics 56:554–562. https://doi.org/10.1111/j.0006-341x.2000.00554.x
Article CAS PubMed Google Scholar
Cabarrou B, Jouin A, Boher JM et al (2015) Assessment of health status over time by Prevalence and Weighted Prevalence functions: interface in R. Comput Methods Programs Biomed 118:298–308. https://doi.org/10.1016/j.cmpb.2014.12.006
Article CAS PubMed Google Scholar
Fine JP, Gray RJ (1999) A proportional hazards model for the subdistribution of a competing risk. J Am Stat Assoc 94:496–509. https://doi.org/10.2307/2670170
Pepe MS, Fleming TR (1989) Weighted Kaplan-Meier statistics: a class of distance tests for censored survival data. Biometrics 45:497–507
Article CAS PubMed Google Scholar
Weber JS, Kähler KC, Hauschild A (2012) Management of immune-related adverse events and kinetics of response with ipilimumab. J Clin Oncol off J Am Soc Clin Oncol 30:2691–2697. https://doi.org/10.1200/JCO.2012.41.6750
Borghaei H, Paz-Ares L, Horn L et al (2015) Nivolumab versus Docetaxel in Advanced Nonsquamous non–small-cell Lung Cancer. N Engl J Med 373:1627–1639. https://doi.org/10.1056/NEJMoa1507643
Article CAS PubMed PubMed Central Google Scholar
Lancar R, Kramar A, Haie-Meder C (1995) Non-parametric methods for analysing recurrent complications of varying severity. Stat Med 14:2701–2712. https://doi.org/10.1002/sim.4780142409
Article CAS PubMed Google Scholar
Longué M, Cabarrou B, Wallet J et al (2018) The importance of jointly analyzing treatment administration and toxicity associated with targeted therapies: a case study of regorafenib in soft tissue sarcoma patients. Ann Oncol off J Eur Soc Med Oncol 29:1588–1593. https://doi.org/10.1093/annonc/mdy168
Thanarajasingam G, Atherton PJ, Novotny PJ et al (2016) Longitudinal adverse event assessment in oncology clinical trials: the toxicity over Time (ToxT) analysis of Alliance trials NCCTG N9741 and 979254. Lancet Oncol 17:663–670. https://doi.org/10.1016/S1470-2045(16)00038-3
Article CAS PubMed PubMed Central Google Scholar
Thanarajasingam G, Leonard JP, Witzig TE et al (2020) Longitudinal toxicity over Time (ToxT) analysis to evaluate tolerability: a case study of lenalidomide in the CALGB 50401 (Alliance) trial. Lancet Haematol 7:e490–e497. https://doi.org/10.1016/S2352-3026(20)30067-3
Article PubMed PubMed Central Google Scholar
Goldhirsch A, Gelber RD, Simes RJ et al (1989) Costs and benefits of adjuvant therapy in breast cancer: a quality-adjusted survival analysis. J Clin Oncol off J Am Soc Clin Oncol 7:36–44. https://doi.org/10.1200/JCO.1989.7.1.36
Beaumont JL, Salsman JM, Diaz J et al (2016) Quality-adjusted time without symptoms or toxicity analysis of pazopanib versus sunitinib in patients with renal cell carcinoma. Cancer 122:1108–1115. https://doi.org/10.1002/cncr.29888
Comments (0)