Ostrom QT, Price M, Neff C, Cioffi G, Waite KA, Kruchko C, et al. CBTRUS statistical report: primary brain and other central nervous system tumors diagnosed in the United States in 2016–2020. Neuro-Oncol. 2023;25(Supplement_4):iv1-99.

Article  PubMed  Google Scholar 

Stupp R, Mason WP, van den Bent MJ, Weller M, Fisher B, Taphoorn MJB, et al. Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med. 2005;352(10):987–96.

Article  CAS  PubMed  Google Scholar 

Wen PY, Weller M, Lee EQ, Alexander BM, Barnholtz-Sloan JS, Barthel FP, et al. Glioblastoma in adults: a Society for Neuro-Oncology (SNO) and European Society of Neuro-Oncology (EANO) consensus review on current management and future directions. Neuro-Oncol. 2020;22(8):1073–113.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Lamborn KR, Yung WKA, Chang SM, Wen PY, Cloughesy TF, DeAngelis LM, et al. Progression-free survival: an important end point in evaluating therapy for recurrent high-grade gliomas. Neuro-Oncol. 2008;10(2):162–70.

Article  PubMed  PubMed Central  Google Scholar 

Wong ET, Hess KR, Gleason MJ, Jaeckle KA, Kyritsis AP, Prados MD, et al. Outcomes and prognostic factors in recurrent glioma patients enrolled onto phase II clinical trials. J Clin Oncol. 1999;17(8):2572–2572.

Article  CAS  PubMed  Google Scholar 

Han K, Ren M, Wick W, Abrey L, Das A, Jin J, et al. Progression-free survival as a surrogate endpoint for overall survival in glioblastoma: a literature-based meta-analysis from 91 trials. Neuro-Oncol. 2014;16(5):696–706.

Article  PubMed  Google Scholar 

Ellingson BM, Wen PY, Chang SM, van den Bent M, Vogelbaum MA, Li G, et al. Objective response rate targets for recurrent glioblastoma clinical trials based on the historic association between objective response rate and median overall survival. Neuro-Oncol. 2023;25(6):1017–28.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Friedman HS, Prados MD, Wen PY, Mikkelsen T, Schiff D, Abrey LE, et al. Bevacizumab alone and in combination with irinotecan in recurrent glioblastoma. J Clin Oncol Off J Am Soc Clin Oncol. 2009;27(28):4733–40.

Article  CAS  Google Scholar 

Thiesse P, Ollivier L, Di Stefano-Louineau D, Négrier S, Savary J, Pignard K, et al. Response rate accuracy in oncology trials: reasons for interobserver variability. Groupe Français d’Immunothérapie of the Fédération Nationale des Centres de Lutte Contre le Cancer. J Clin Oncol Off J Am Soc Clin Oncol. 1997;15(12):3507–14.

Article  CAS  Google Scholar 

Provenzale JM, Ison C, Delong D. Bidimensional measurements in brain tumors: assessment of interobserver variability. AJR Am J Roentgenol. 2009;193(6):W515–522.

Article  PubMed  Google Scholar 

Hygino da Cruz LC, Rodriguez I, Domingues RC, Gasparetto EL, Sorensen AG. Pseudoprogression and pseudoresponse: imaging challenges in the assessment of posttreatment glioma. AJNR Am J Neuroradiol. 2011;32(11):1978–85.

Article  PubMed  PubMed Central  Google Scholar 

Lin NU, Lee EQ, Aoyama H, Barani IJ, Barboriak DP, Baumert BG, et al. Response assessment criteria for brain metastases: proposal from the RANO group. Lancet Oncol. 2015;16(6):e270–8.

Article  PubMed  Google Scholar 

Alexander BM, Brown PD, Ahluwalia MS, Aoyama H, Baumert BG, Chang SM, et al. Clinical trial design for local therapies for brain metastases: a guideline by the Response Assessment in Neuro-Oncology Brain Metastases working group. Lancet Oncol. 2018;19(1):e33-42.

Article  PubMed  Google Scholar 

Camidge DR, Lee EQ, Lin NU, Margolin K, Ahluwalia MS, Bendszus M, et al. Clinical trial design for systemic agents in patients with brain metastases from solid tumours: a guideline by the Response Assessment in Neuro-Oncology Brain Metastases working group. Lancet Oncol. 2018;19(1):e20-32.

Article  PubMed  Google Scholar 

Chamberlain M, Junck L, Brandsma D, Soffietti R, Rudà R, Raizer J, et al. Leptomeningeal metastases: a RANO proposal for response criteria. Neuro Oncol. 2017;19(4):484–92.

Warren KE, Vezina G, Poussaint TY, Warmuth-Metz M, Chamberlain MC, Packer RJ, et al. Response assessment in medulloblastoma and leptomeningeal seeding tumors: recommendations from the Response Assessment in Pediatric Neuro-Oncology committee. Neuro-Oncol. 2018;20(1):13–23.

Article  CAS  PubMed  Google Scholar 

Levin VA, Crafts DC, Norman DM, Hoffer PB, Spire JP, Wilson CB. Criteria for evaluating patients undergoing chemotherapy for malignant brain tumors. J Neurosurg. 1977;47(3):329–35.

Article  CAS  PubMed  Google Scholar 

Cairncross JG, Macdonald DR, Pexman JH, Ives FJ. Steroid-induced CT changes in patients with recurrent malignant glioma. Neurology. 1988;38(5):724–6.

Article  CAS  PubMed  Google Scholar 

Miller AB, Hoogstraten B, Staquet M, Winkler A. Reporting results of cancer treatment. Cancer. 1981;47(1):207–14.

Article  CAS  PubMed  Google Scholar 

Therasse P, Arbuck SG, Eisenhauer EA, Wanders J, Kaplan RS, Rubinstein L, et al. New Guidelines to evaluate the response to treatment in solid tumors. J Natl Cancer Inst. 2000;92(3):205–16.

Eisenhauer EA, Therasse P, Bogaerts J, Schwartz LH, Sargent D, Ford R, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009;45(2):228–47.

Article  CAS  PubMed  Google Scholar 

Macdonald DR, Cascino TL, Schold SC, Cairncross JG. Response criteria for phase II studies of supratentorial malignant glioma. J Clin Oncol. 1990;8(7):1277–80.

Article  CAS  PubMed  Google Scholar 

Wen PY, Macdonald DR, Reardon DA, Cloughesy TF, Sorensen AG, Galanis E, et al. Updated response assessment criteria for high-grade gliomas: response assessment in neuro-oncology working group. J Clin Oncol. 2010;28(11):1963–72. This paper reports RANO-HGG, the most widely used response assessment criteria in high-grade gliomas.

Article  PubMed  Google Scholar 

Norden AD, Young GS, Setayesh K, Muzikansky A, Klufas R, Ross GL, et al. Bevacizumab for recurrent malignant gliomas: efficacy, toxicity, and patterns of recurrence. Neurology. 2008;70(10):779–87.

Article  CAS  PubMed  Google Scholar 

Norden AD, Drappatz J, Wen PY. Novel anti-angiogenic therapies for malignant gliomas. Lancet Neurol. 2008;7(12):1152–60.

Article  CAS  PubMed  Google Scholar 

Nowosielski M, Wiestler B, Goebel G, Hutterer M, Schlemmer HP, Stockhammer G, et al. Progression types after antiangiogenic therapy are related to outcome in recurrent glioblastoma. Neurology. 2014;82(19):1684–92.

Article  CAS  PubMed  Google Scholar 

Hagiwara A, Schlossman J, Shabani S, Raymond C, Tatekawa H, Abrey LE, et al. Incidence, molecular characteristics, and imaging features of “clinically-defined pseudoprogression” in newly diagnosed glioblastoma treated with chemoradiation. J Neurooncol. 2022;159(3):509–18.

Article  PubMed  Google Scholar 

Youssef G, Rahman R, Bay C, Wang W, Lim-Fat MJ, Arnaout O, et al. Evaluation of standard response assessment in neuro-oncology, modified response assessment in neuro-oncology, and immunotherapy response assessment in neuro-oncology in newly diagnosed and recurrent glioblastoma. J Clin Oncol. 2023;41(17):3160–71. Evaluation of standard response assessment in neuro-oncology, modified response assessment in neuro-oncology, and immunotherapy response assessment in neuro-oncology in newly diagnosed and recurrent glioblastoma.

Article  CAS  PubMed  Google Scholar 

Wen PY, Chang SM, Van den Bent MJ, Vogelbaum MA, Macdonald DR, Lee EQ. Response assessment in neuro-oncology clinical trials. J Clin Oncol. 2017;35(21):2439–49.

Article  CAS  PubMed  PubMed Central  Google Scholar 

van den Bent MJ, Wefel JS, Schiff D, Taphoorn MJB, Jaeckle K, Junck L, et al. Response assessment in neuro-oncology (a report of the RANO group): assessment of outcome in trials of diffuse low-grade gliomas. Lancet Oncol. 2011;12(6):583–93.

Article  PubMed  Google Scholar 

Avila EK, Chamberlain M, Schiff D, Reijneveld JC, Armstrong TS, Ruda R, et al. Seizure control as a new metric in assessing efficacy of tumor treatment in low-grade glioma trials. Neuro-Oncol. 2017;19(1):12–21.

Article  PubMed  Google Scholar 

Hodi FS, O’Day SJ, McDermott DF, Weber RW, Sosman JA, Haanen JB, et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med. 2010;363(8):711–23.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hamid O, Robert C, Daud A, Hodi FS, Hwu WJ, Kefford R, et al. Safety and tumor responses with lambrolizumab (anti-PD-1) in melanoma. N Engl J Med. 2013;369(2):134–44.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Wolchok JD, Hoos A, O’Day S, Weber JS, Hamid O, Lebbé C, et al. Guidelines for the evaluation of immune therapy activity in solid tumors: immune-related response criteria. Clin Cancer Res. 2009;15(23):7412–20.

Article  CAS  PubMed  Google Scholar 

Okada H, Kalinski P, Ueda R, Hoji A, Kohanbash G, Donegan TE, et al. Induction of CD8+ T-cell responses against novel glioma-associated antigen peptides and clinical activity by vaccinations with -type 1 polarized dendritic cells and polyinosinic-polycytidylic acid stabilized by lysine and carboxymethylcellulose in patients with recurrent malignant glioma. J Clin Oncol Off J Am Soc Clin Oncol. 2011;29(3):330–6.

Article  CAS  Google Scholar 

Pollack IF, Jakacki RI, Butterfield LH, Hamilton RL, Panigrahy A, Potter DM, et al. Antigen-specific immune responses and clinical outcome after vaccination with glioma-associated antigen peptides and polyinosinic-polycytidylic acid stabilized by lysine and carboxymethylcellulose in children with newly diagnosed malignant brainstem and nonbrainstem gliomas. J Clin Oncol Off J Am Soc Clin Oncol. 2014;32(19):2050–8.

Article  CAS  Google Scholar 

Sampson JH, Heimberger AB, Archer GE, Aldape KD, Friedman AH, Friedman HS, et al. Immunologic escape after prolonged progression-free survival with epidermal growth factor receptor variant III peptide vaccination in patients with newly diagnosed