End points for Phase II trials in recurrent glioblastoma: the cornerstone for a new era

References

• Batchelor T, Mulholland P, Neyns B et al. The efficacy of cediranib as monotherapy and in combination with lomustine compared to lomustine alone in patients with recurrent glioblastoma: a Phase III randomized study. Neuro-Oncology12(Suppl. 4), iv75 (2010).
   Google Scholar
• Wick W, Puduvalli VK, Chamberlain MC et al. Phase III study of enzastaurin compared With lomustine in the treatment of recurrent intracranial glioblastoma. J. Clin. Oncol.28(7), 1168–1174 (2010).
   PubMed Web of Science ®Google Scholar
• Brada M, Hoang-Xuan K, Rampling R et al. Multicenter Phase II trial of temozolomide in patients with glioblastoma multiforme at first relapse. Ann. Oncol.12(2), 259–266 (2001).
   PubMed Web of Science ®Google Scholar
• Grothey A, Hedrick EE, Mass RD et al. Response-independent survival benefit in metastatic colorectal cancer: a comparative analysis of N9741 and AVF2107. J. Clin. Oncol.26(2), 183–189 (2008).
   PubMedGoogle Scholar
• Ratain MJ, Eisen T, Stadler WM et al. Phase II placebo-controlled randomized discontinuation trial of sorafenib in patients with metastatic renal cell carcinoma. J. Clin. Oncol.24(16), 2505–2512 (2006).
   PubMed Web of Science ®Google Scholar
• Abou-Alfa GK, Schwartz L, Ricci S et al. Phase II study of sorafenib in patients with advanced hepatocellular carcinoma. J. Clin. Oncol.24(26), 4293–4300 (2006).
   PubMed Web of Science ®Google Scholar
• Escudier B, Eisen T, Stadler WM et al. Sorafenib in advanced clear-cell renal-cell carcinoma. N. Engl. J. Med.356(2), 125–134 (2007).
   PubMed Web of Science ®Google Scholar
• Llovet JM, Ricci S, Mazzaferro V et al. Sorafenib in advanced hepatocellular carcinoma. N. Engl. J. Med.359(4), 378–390 (2008).
   PubMed Web of Science ®Google Scholar
• Burris HA 3rd, Moore MJ, Andersen J et al. Improvements in survival and clinical benefit with gemcitabine as first-line therapy for patients with advanced pancreas cancer: a randomized trial. J. Clin. Oncol.15(6), 2403–2413 (1997).
   PubMed Web of Science ®Google Scholar
• Wong ET, Hess KR, Gleason MJ et al. Outcomes and prognostic factors in recurrent glioma patients enrolled onto Phase II clinical trials. J. Clin. Oncol.17(8), 2572–2578 (1999).
   PubMed Web of Science ®Google Scholar
• Ballman KV, Buckner JC, Brown PD et al. The relationship between six-month progression-free survival and 12-month overall survival end points for Phase II trials in patients with glioblastoma multiforme. Neuro Oncol.9(1), 29–38 (2007).
   PubMed Web of Science ®Google Scholar
• Lamborn KR, Yung WK, Chang SM et al. Progression-free survival: an important end point in evaluating therapy for recurrent high-grade gliomas. Neuro Oncol.10(2), 162–170 (2008).
   PubMed Web of Science ®Google Scholar
• Brandes AA, Franceschi E. New agents and new end points for recurrent gliomas. J. Clin. Oncol. (2010).
   Google Scholar
• Franceschi E, Brandes AA, Tosoni A et al. Can OS-6 replace PFS-6 as a primary endpoint in Phase II studies on glioblastoma patients given antiangiogenetic drugs? J. Clin. Oncol. (Meeting Abstracts)28(15 Suppl.), 2022 (2010).
   Google Scholar
• Schilsky RL. End points in cancer clinical trials and the drug approval process. Clin. Cancer Res.8(4), 935–938 (2002).
   PubMed Web of Science ®Google Scholar
• Parulekar WR, Eisenhauer EA. Novel endpoints and design of early clinical trials. Ann. Oncol.13(Suppl. 4), 139–143 (2002).
   PubMed Web of Science ®Google Scholar
• Adjei AA, Christian M, Ivy P. Novel designs and end points for Phase II clinical trials. Clin. Cancer Res.15(6), 1866–1872 (2009).
   PubMed Web of Science ®Google Scholar
• Chi AS, Sorensen AG, Jain RK, Batchelor TT. Angiogenesis as a therapeutic target in malignant gliomas. Oncologist14(6), 621–636 (2009).
   PubMedGoogle Scholar
• Kreisl TN, Kim L, Moore K et al. Phase II trial of single-agent bevacizumab followed by bevacizumab plus irinotecan at tumor progression in recurrent glioblastoma. J. Clin. Oncol.27, 740–745 (2008).
   PubMedGoogle Scholar
• Friedman HS, Prados MD, Wen PY et al. Bevacizumab alone and in combination with irinotecan in recurrent glioblastoma. J. Clin. Oncol.27(28), 4733–4740 (2009).
   PubMed Web of Science ®Google Scholar
• Chen W, Delaloye S, Silverman DH et al. Predicting treatment response of malignant gliomas to bevacizumab and irinotecan by imaging proliferation with [18F] fluorothymidine positron emission tomography: a pilot study. J. Clin. Oncol.25(30), 4714–4721 (2007).
   PubMed Web of Science ®Google Scholar
• Paesmans M, Sculier JP, Libert P et al. Response to chemotherapy has predictive value for further survival of patients with advanced non-small cell lung cancer: 10 years experience of the European Lung Cancer Working Party. Eur. J. Cancer33(14), 2326–2332 (1997).
   Google Scholar
• Torri V, Simon R, Russek-Cohen E, Midthune D, Friedman M. Relationship between response and survival in patients with advanced ovarian cancer. J. Natl Cancer Inst.84(11), 899–900 (1992).
   Google Scholar
• Graf W, Pahlman L, Bergstrom R, Glimelius B. The relationship between an objective response to chemotherapy and survival in advanced colorectal cancer. Br. J. Cancer70(3), 559–563 (1994).
   PubMed Web of Science ®Google Scholar
• Macdonald DR, Cascino TL, Schold SC Jr, Cairncross JG. Response criteria for Phase II studies of supratentorial malignant glioma. J. Clin. Oncol.8(7), 1277–1280 (1990).
   PubMed Web of Science ®Google Scholar
• Wen PY, Macdonald DR, Reardon DA et al. Updated response assessment criteria for high-grade gliomas: response assessment in neuro-oncology working group. J. Clin. Oncol.28(11), 1963–1972 (2010).
   PubMed Web of Science ®Google Scholar
• Pazdur R. Endpoints for assessing drug activity in clinical trials. Oncologist13(Suppl. 2), 19–21 (2008).
   PubMed Web of Science ®Google Scholar
• Panageas KS, Ben-Porat L, Dickler MN, Chapman PB, Schrag D. When you look matters: the effect of assessment schedule on progression-free survival. J. Natl Cancer Inst.99(6), 428–432 (2007).
   PubMed Web of Science ®Google Scholar
• Committee for Medical Products for Human Use. Appendix 1 to the Guideline on the Evaluation of Anticancer Medicinal Products in Man. EMEA CPMP/EWP/205/95/Rev 3, London, UK (2008).
   Google Scholar
• Federal Drug Administration.Guidance for Industry. Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics. US Department of Health and Human Services, Rockville, MD, USA (2007).
   Google Scholar
• Michaelis LC, Ratain MJ. Measuring response in a post-RECIST world: from black and white to shades of grey. Nat. Rev. Cancer6(5), 409–414 (2006).
   PubMed Web of Science ®Google Scholar
• Rubinstein LV, Korn EL, Freidlin B, Hunsberger S, Ivy SP, Smith MA. Design issues of randomized Phase II trials and a proposal for Phase II screening trials. J. Clin. Oncol.23(28), 7199–7206 (2005).
   PubMed Web of Science ®Google Scholar
• Ocana A, Amir E, Vera F, Eisenhauer EA, Tannock IF. Addition of bevacizumab to chemotherapy for treatment of solid tumors: similar results but different conclusions. J. Clin. Oncol.29(3), 254–256 (2010).
   PubMedGoogle Scholar
• Montagnani F, Migali C, Fiorentini G. Progression-free survival in bevacizumab-based first-line treatment for patients with metastatic colorectal cancer: is it a really good end point? J. Clin. Oncol.27(28), e132–e133; author reply e134–e135 (2009).
   Google Scholar
• Di Leo A, Buyse M, Bleiberg H. Is overall survival a realistic primary end point in advanced colorectal cancer studies? A critical assessment based on four clinical trials comparing fluorouracil plus leucovorin with the same treatment combined either with oxaliplatin or with CPT-11. Ann. Oncol.15(4), 545–549 (2004).
   PubMed Web of Science ®Google Scholar
• Biomarkers Definitions Working Group.Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin. Pharmacol. Ther.69(3), 89–95 (2001).
   PubMed Web of Science ®Google Scholar
• Sargent DJ, Patiyil S, Yothers G et al. End points for colon cancer adjuvant trials: observations and recommendations based on individual patient data from 20,898 patients enrolled onto 18 randomized trials from the ACCENT Group. J. Clin. Oncol.25(29), 4569–4574 (2007).
   PubMed Web of Science ®Google Scholar
• Sargent DJ, Wieand HS, Haller DG et al. Disease-free survival versus overall survival as a primary end point for adjuvant colon cancer studies: individual patient data from 20,898 patients on 18 randomized trials. J. Clin. Oncol.23(34), 8664–8670 (2005).
   PubMed Web of Science ®Google Scholar
• Tang PA, Bentzen SM, Chen EX, Siu LL. Surrogate end points for median overall survival in metastatic colorectal cancer: literature-based analysis from 39 randomized controlled trials of first-line chemotherapy. J. Clin. Oncol.25(29), 4562–4568 (2007).
   PubMed Web of Science ®Google Scholar
• Buyse M, Burzykowski T, Carroll K et al. Progression-free survival is a surrogate for survival in advanced colorectal cancer. J. Clin. Oncol.25(33), 5218–5224 (2007).
   PubMed Web of Science ®Google Scholar
• Brandes AA, Tosoni A, Amista P et al. How effective is BCNU in recurrent glioblastoma in the modern era? A Phase II trial. Neurology63(7), 1281–1284 (2004).
   PubMed Web of Science ®Google Scholar
• Brandes AA, Tosoni A, Franceschi E et al. Fotemustine as second-line treatment for recurrent or progressive glioblastoma after concomitant and/or adjuvant temozolomide: a Phase II trial of Gruppo Italiano Cooperativo di Neuro-Oncologia (GICNO). Cancer Chemother. Pharmacol.64(4), 769–775 (2009).
   PubMed Web of Science ®Google Scholar