Progression-free survival, disease-free survival and other composite end points in oncology: improved reporting is needed

Del Paggio, J. C. et al. Evolution of the randomized clinical trial in the era of precision oncology. JAMA Oncol. 7, 728–734 (2021).

Article  PubMed  Google Scholar 

Le-Rademacher, J. & Wang, X. Time-to-event data: an overview and analysis considerations. J. Thorac. Oncol. 16, 1067–1074 (2021).

Article  PubMed  Google Scholar 

McCoy, C. E. Understanding the use of composite endpoints in clinical trials. West J. Emerg. Med. 19, 631–634 (2018).

Article  PubMed  PubMed Central  Google Scholar 

Kim, C. & Prasad, V. Cancer drugs approved on the basis of a surrogate end point and subsequent overall survival: an analysis of 5 years of US Food and Drug Administration approvals. JAMA Intern. Med. 175, 1992–1994 (2015).

Article  PubMed  Google Scholar 

Eisenhauer, E. A. et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur. J. Cancer 45, 228–247 (2009).

Article  CAS  PubMed  Google Scholar 

Delgado, A. & Guddati, A. K. Clinical endpoints in oncology–a primer. Am. J. Cancer Res. 11, 1121–1131 (2021).

PubMed  PubMed Central  Google Scholar 

Robinson, A. G., Booth, C. M. & Eisenhauer, E. A. Disease-free survival as an end-point in the treatment of solid tumours – perspectives from clinical trials and clinical practice. Eur. J. Cancer 50, 2298–2302 (2014).

Article  PubMed  Google Scholar 

Cohen, R. et al. Guidelines for time-to-event end-point definitions in adjuvant randomised trials for patients with localised colon cancer: results of the DATECAN initiative. Eur. J. Cancer 130, 63–71 (2020).

Article  PubMed  PubMed Central  Google Scholar 

Gourgou-Bourgade, S. et al. Guidelines for time-to-event end point definitions in breast cancer trials: results of the DATECAN initiative (Definition for the Assessment of Time-to-event Endpoints in CANcer trials). Ann. Oncol. 26, 873–879 (2015).

Article  CAS  PubMed  Google Scholar 

Fleming, M. T., Morris, M. J., Heller, G. & Scher, H. I. Post-therapy changes in PSA as an outcome measure in prostate cancer clinical trials. Nat. Rev. Clin. Oncol. 3, 658–667 (2006).

Article  CAS  Google Scholar 

Bubley, G. et al. Eligibility and response guidelines for phase II clinical trials in androgen-independent prostate cancer: recommendations from the Prostate-Specific Antigen Working Group. J. Clin. Oncol. 17, 3461–3467 (1999).

Article  CAS  PubMed  Google Scholar 

Therasse, P. et al. New guidelines to evaluate the response to treatment in solid tumors. J. Natl Cancer Inst. 92, 205–216 (2000).

Article  CAS  PubMed  Google Scholar 

Scher, H. I. et al. Design and end points of clinical trials for patients with progressive prostate cancer and castrate levels of testosterone: recommendations of the Prostate Cancer Clinical Trials Working Group. J. Clin. Oncol. 26, 1148–1159 (2008).

Article  PubMed  Google Scholar 

Scher, H. I. et al. Trial design and objectives for castration-resistant prostate cancer: updated recommendations from the Prostate Cancer Clinical Trials Working Group 3. J. Clin. Oncol. 34, 1402–1418 (2016).

Article  PubMed  PubMed Central  Google Scholar 

Ryan, C. J. et al. Abiraterone in metastatic prostate cancer without previous chemotherapy. N. Engl. J. Med. 368, 138–148 (2013).

Article  CAS  PubMed  Google Scholar 

Franzese, C. et al. The efficacy of stereotactic body radiation therapy and the impact of systemic treatments in oligometastatic patients from prostate cancer. Cancer Med. 7, 4379–4386 (2018).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Gafita, A. et al. Early prostate-specific antigen changes and clinical outcome after 177Lu-PSMA radionuclide treatment in patients with metastatic castration-resistant prostate cancer. J. Nucl. Med. 61, 1476–1483 (2020).

Article  CAS  PubMed  Google Scholar 

Fizazi, K. et al. Phase III, randomized, placebo-controlled study of docetaxel in combination with zibotentan in patients with metastatic castration-resistant prostate cancer. J. clin. oncol. 31, 1740–1747 (2013).

Article  CAS  PubMed  Google Scholar 

Woo, S. et al. Correlation between imaging-based intermediate endpoints and overall survival in men with metastatic castration-resistant prostate cancer: analysis of 28 randomized trials using the Prostate Cancer Clinical Trials Working Group (PCWG2) criteria in 16,511 patients. Clin. Genitourin. Cancer 20, 69–79 (2022).

Article  PubMed  Google Scholar 

Shore, N. D. et al. Prostate-specific antigen (PSA) progression-free survival (PFS): a comparison of degarelix versus leuprolide in patients with prostate cancer [abstract]. J. Clin. Oncol. 29 (Suppl. 7), 12 (2011).

Article  Google Scholar 

Kato, H. et al. Consequences of an early PSA response to enzalutamide treatment for Japanese patients with metastatic castration-resistant prostate cancer. Anticancer Res. 36, 6141–6150 (2016).

Article  CAS  PubMed  Google Scholar 

Zhao, J. et al. AKR1C3 expression in primary lesion rebiopsy at the time of metastatic castration-resistant prostate cancer is strongly associated with poor efficacy of abiraterone as a first-line therapy. Prostate 79, 1553–1562 (2019).

Article  CAS  PubMed  Google Scholar 

Kim, W. et al. Sequential use of the androgen synthesis inhibitors ketoconazole and abiraterone acetate in castration-resistant prostate cancer and the predictive value of circulating androgens. Clin. Cancer Res. 20, 6269–6276 (2014).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Saad, F. et al. Apalutamide plus abiraterone acetate and prednisone versus placebo plus abiraterone and prednisone in metastatic, castration-resistant prostate cancer (ACIS): a randomised, placebo-controlled, double-blind, multinational, phase 3 study. Lancet Oncol. 22, 1541–1559 (2021).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Rao, A. et al. Impact of clinical versus radiographic progression on clinical outcomes in metastatic castration-resistant prostate cancer. ESMO Open 5, e000943 (2020).

Article  PubMed  PubMed Central  Google Scholar 

Choi, S. W. & Cheung, C. W. The case of the misleading composite – one outcome is better than two. Anaesthesia 71, 1101–1103 (2016).

Article  CAS  PubMed  Google Scholar 

Freemantle, N., Calvert, M., Wood, J., Eastaugh, J. & Griffin, C. Composite outcomes in randomized trials: greater precision but with greater uncertainty? JAMA 289, 2554–2559 (2003).

Article  PubMed  Google Scholar 

Lei, N. et al. Docetaxel-based therapy with and without antiangiogenic agents as first-line chemotherapy for castration-resistant prostate cancer: a meta-analysis of nine randomized controlled trials. Mol. Clin. Oncol. 2, 1182–1188 (2014).

Article  PubMed  PubMed Central  Google Scholar 

Kelly, W. K. et al. Randomized, double-blind, placebo-controlled phase III trial comparing docetaxel and prednisone with or without bevacizumab in men with metastatic castration-resistant prostate cancer: CALGB 90401. J. Clin. Oncol. 30, 1534–1540 (2012).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Quinn, D. I. et al. Docetaxel and atrasentan versus docetaxel and placebo for men with advanced castration-resistant prostate cancer (SWOG S0421): a randomised phase 3 trial. Lancet Oncol. 14, 893–900 (2013).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Pili, R. et al. Phase II study on the addition of ASA404 (vadimezan; 5,6-dimethylxanthenone-4-acetic acid) to docetaxel in CRMPC. Clin. Cancer Res. 16, 2906–2914 (2010).

Article  CAS  PubMed  Google Scholar 

Heidenreich, A. et al. A randomized, double-blind, multicenter, phase 2 study of a human monoclonal antibody to human αν integrins (intetumumab) in combination with docetaxel and prednisone for the first-line treatment of patients with metastatic castration-resistant prostate cancer. Ann. Oncol. 24, 329–336 (2013).

Article  CAS  PubMed  Google Scholar 

Younes, A. et al. International Working Group consensus response evaluation criteria in lymphoma (RECIL 2017). Ann. Oncol. 28, 1436–1447 (2017).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cheson, B. D. et al. Recommendations for initial evaluation, staging, and response assessment of Hodgkin and non-Hodgkin lymphoma: the Lugano classification. J. Clin. Oncol. 32, 3059–3067 (2014).

Article  PubMed  PubMed Central  Google Scholar 

Kostakoglu, L. et al. Recil 2017 criteria demonstrated similar prognostic value and detected a comparable treatment difference between obinutuzumab- and rituximab-chemotherapy compared with Cheson 2007 and Lugano 2014 criteria in patients with previously untreated advanced-stage follicular lymphoma [abstract]. Blood 136 (Suppl. 1), 25–26 (2020).

Article  Google Scholar 

Berzaczy, D. et al. RECIL versus Lugano for treatment response assessment in FDG-avid non-Hodgkin lymphomas: a head-to-head comparison in 54 patients. Cancers 12, 9 (2019).

Article  PubMed  PubMed Central  Google Scholar 

Wen, P. Y. et al. Updated response assessment criteria for high-grade gliomas: Response Assessment in Neuro-Oncology Working Group. J. Clin. Oncol. 28, 1963–1972 (2010).

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