Assessing Treatment Benefit in Immuno-oncology

[1]  R. Pazdur,et al.  Response Rate, Event-Free Survival, and Overall Survival in Newly Diagnosed Acute Myeloid Leukemia: US Food and Drug Administration Trial-Level and Patient-Level Analyses , 2021, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[2]  B. Freidlin,et al.  Methods for Accommodating Nonproportional Hazards in Clinical Trials: Ready for the Primary Analysis? , 2019, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[3]  Patrick C. Staples,et al.  An Online Calculator for the Prediction of Survival in Glioblastoma Patients Using Classical Statistics and Machine Learning , 2019, Neurosurgery.

[4]  H. Quan,et al.  Design and monitoring of survival trials based on restricted mean survival times , 2019, Clinical trials.

[5]  Satrajit Roychoudhury,et al.  Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group , 2019, Statistics in Biopharmaceutical Research.

[6]  J. Taube,et al.  Major pathologic response on biopsy (MPRbx) in patients with advanced melanoma treated with anti-PD-1: evidence for an early, on-therapy biomarker of response. , 2019, Annals of oncology : official journal of the European Society for Medical Oncology.

[7]  P. Royston,et al.  Combined test versus logrank/Cox test in 50 randomised trials , 2019, Trials.

[8]  D. Maucort-Boulch,et al.  Assessing long-term survival benefits of immune checkpoint inhibitors using the net survival benefit. , 2019, Journal of the National Cancer Institute.

[9]  V. Servois,et al.  Novel patterns of response under immunotherapy , 2019, Annals of oncology : official journal of the European Society for Medical Oncology.

[10]  M. Buyse Multiple Prioritized Outcomes , 2019, Wiley StatsRef: Statistics Reference Online.

[11]  J. Wolchok,et al.  Five-year survival outcomes for patients with advanced melanoma treated with pembrolizumab in KEYNOTE-001 , 2019, Annals of oncology : official journal of the European Society for Medical Oncology.

[12]  O. V. Matorin,et al.  Pembrolizumab versus methotrexate, docetaxel, or cetuximab for recurrent or metastatic head-and-neck squamous cell carcinoma (KEYNOTE-040): a randomised, open-label, phase 3 study , 2019, The Lancet.

[13]  D. Magirr,et al.  Modestly weighted logrank tests , 2018, Statistics in medicine.

[14]  D. Planchard,et al.  Hyperprogressive Disease in Patients With Advanced Non–Small Cell Lung Cancer Treated With PD-1/PD-L1 Inhibitors or With Single-Agent Chemotherapy , 2018, JAMA oncology.

[15]  Laura A. Levit,et al.  Trial Reporting in Immuno-Oncology (TRIO): an American society of clinical oncology-society for immunotherapy of cancer statement , 2018, Journal of Immunotherapy for Cancer.

[16]  L. Schwartz,et al.  Evaluation of classical clinical endpoints as surrogates for overall survival in patients treated with immune checkpoint blockers: a systematic review and meta-analysis , 2018, Journal of Cancer Research and Clinical Oncology.

[17]  V. Velculescu,et al.  Pathologic features of response to neoadjuvant anti-PD-1 in resected non-small-cell lung carcinoma: a proposal for quantitative immune-related pathologic response criteria (irPRC) , 2018, Annals of oncology : official journal of the European Society for Medical Oncology.

[18]  B. Freidlin,et al.  Interim Futility Monitoring Assessing Immune Therapies With a Potentially Delayed Treatment Effect. , 2018, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[19]  L. Trippa,et al.  Violations of the proportional hazards assumption in randomized phase III oncology clinical trials. , 2018 .

[20]  L. Tian,et al.  Evaluating Treatment Effect Based on Duration of Response for a Comparative Oncology Study , 2018, JAMA oncology.

[21]  L. Tian,et al.  Efficiency of two sample tests via the restricted mean survival time for analyzing event time observations , 2018, Biometrics.

[22]  L. Howie,et al.  A 25-Year Experience of US Food and Drug Administration Accelerated Approval of Malignant Hematology and Oncology Drugs and Biologics: A Review , 2018, JAMA oncology.

[23]  W. Li,et al.  Treatment effects measured by restricted mean survival time in trials of immune checkpoint inhibitors for cancer , 2018, Annals of oncology : official journal of the European Society for Medical Oncology.

[24]  Bo Huang,et al.  Comparison of the restricted mean survival time with the hazard ratio in superiority trials with a time‐to‐event end point , 2018, Pharmaceutical statistics.

[25]  I. Marschner,et al.  Defining the Most Appropriate Primary End Point in Phase 2 Trials of Immune Checkpoint Inhibitors for Advanced Solid Cancers: A Systematic Review and Meta-analysis , 2018, JAMA oncology.

[26]  M. Buyse,et al.  An extension of generalized pairwise comparisons for prioritized outcomes in the presence of censoring , 2018, Statistical methods in medical research.

[27]  David C. Smith,et al.  Five-Year Follow-Up of Nivolumab in Previously Treated Advanced Non-Small-Cell Lung Cancer: Results From the CA209-003 Study. , 2018, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[28]  P. Hegde,et al.  Atezolizumab versus chemotherapy in patients with platinum-treated locally advanced or metastatic urothelial carcinoma (IMvigor211): a multicentre, open-label, phase 3 randomised controlled trial , 2018, The Lancet.

[29]  B. Chmielowski How Should We Assess Benefit in Patients Receiving Checkpoint Inhibitor Therapy? , 2018, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[30]  Bo Huang,et al.  Some statistical considerations in the clinical development of cancer immunotherapies , 2018, Pharmaceutical statistics.

[31]  J. Wolchok,et al.  Immune-Modified Response Evaluation Criteria In Solid Tumors (imRECIST): Refining Guidelines to Assess the Clinical Benefit of Cancer Immunotherapy. , 2018, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[32]  Zheng Su,et al.  Is it time for the weighted log-rank test to play a more important role in confirmatory trials? , 2017, Contemporary clinical trials communications.

[33]  F. Bidault,et al.  Patterns of responses in metastatic NSCLC during PD-1 or PDL-1 inhibitor therapy: Comparison of RECIST 1.1, irRECIST and iRECIST criteria. , 2018, European journal of cancer.

[34]  Vanessa M. Hubbard-Lucey,et al.  Comprehensive analysis of the clinical immuno-oncology landscape , 2018, Annals of oncology : official journal of the European Society for Medical Oncology.

[35]  H. Uno,et al.  Interpretability of Cancer Clinical Trial Results Using Restricted Mean Survival Time as an Alternative to the Hazard Ratio , 2017, JAMA oncology.

[36]  F. André,et al.  Response rate as a potential surrogate for survival and efficacy in patients treated with novel immune checkpoint inhibitors: A meta-regression of randomised prospective studies. , 2017, European journal of cancer.

[37]  D. Schadendorf,et al.  Nivolumab for Patients With Advanced Melanoma Treated Beyond Progression: Analysis of 2 Phase 3 Clinical Trials , 2017, JAMA oncology.

[38]  L. F. León,et al.  Estimation of treatment effects in weighted log-rank tests , 2017, Contemporary clinical trials communications.

[39]  A. Ravaud,et al.  Treatment Beyond Progression in Patients with Advanced Renal Cell Carcinoma Treated with Nivolumab in CheckMate 025. , 2017, European urology.

[40]  V. Anagnostou,et al.  Immuno-oncology Trial Endpoints: Capturing Clinically Meaningful Activity , 2017, Clinical Cancer Research.

[41]  V. Servois,et al.  Hyperprogression during anti-PD-1/PD-L1 therapy in patients with recurrent and/or metastatic head and neck squamous cell carcinoma , 2017, Annals of oncology : official journal of the European Society for Medical Oncology.

[42]  B. Freidlin,et al.  Assessing treatment efficacy in the subset of responders in a randomized clinical trial , 2017, Annals of oncology : official journal of the European Society for Medical Oncology.

[43]  J. Wargo,et al.  Hallmarks of response to immune checkpoint blockade , 2017, British Journal of Cancer.

[44]  J. Crowley,et al.  End points and statistical considerations in immuno-oncology trials: impact on multiple myeloma , 2017, Future oncology.

[45]  Robert Ford,et al.  iRECIST: guidelines for response criteria for use in trials testing immunotherapeutics. , 2017, The Lancet. Oncology.

[46]  Zhenzhen Xu,et al.  Designing therapeutic cancer vaccine trials with delayed treatment effect , 2017, Statistics in medicine.

[47]  S. Culine,et al.  Pembrolizumab as Second‐Line Therapy for Advanced Urothelial Carcinoma , 2017, The New England journal of medicine.

[48]  P. Keegan,et al.  Characterization of outcomes in patients with metastatic non-small cell lung cancer treated with programmed cell death protein 1 inhibitors past RECIST version 1.1-defined disease progression in clinical trials. , 2017, Seminars in oncology.

[49]  Carlos Barrios,et al.  Atezolizumab versus docetaxel in patients with previously treated non-small-cell lung cancer (OAK): a phase 3, open-label, multicentre randomised controlled trial , 2017, The Lancet.

[50]  Charles Ferté,et al.  Hyperprogressive Disease Is a New Pattern of Progression in Cancer Patients Treated by Anti-PD-1/PD-L1 , 2016, Clinical Cancer Research.

[51]  Y. Shentu,et al.  Pembrolizumab versus Chemotherapy for PD-L1-Positive Non-Small-Cell Lung Cancer. , 2016, The New England journal of medicine.

[52]  D. Follmann,et al.  Using Outcomes to Analyze Patients Rather than Patients to Analyze Outcomes: A Step Toward Pragmatism in Benefit:Risk Evaluation , 2016, Statistics in biopharmaceutical research.

[53]  L. Trinquart,et al.  Comparison of Treatment Effects Measured by the Hazard Ratio and by the Ratio of Restricted Mean Survival Times in Oncology Randomized Controlled Trials. , 2016, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[54]  R. A’Hern Restricted Mean Survival Time: An Obligatory End Point for Time-to-Event Analysis in Cancer Trials? , 2016, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[55]  J. Wolchok,et al.  Evaluation of Immune-Related Response Criteria and RECIST v1.1 in Patients With Advanced Melanoma Treated With Pembrolizumab. , 2016, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[56]  R. Motzer,et al.  Safety and Efficacy of Nivolumab in Patients With Metastatic Renal Cell Carcinoma Treated Beyond Progression: A Subgroup Analysis of a Randomized Clinical Trial. , 2016, JAMA oncology.

[57]  L. Nardo,et al.  Tumor immune profiling predicts response to anti-PD-1 therapy in human melanoma. , 2016, The Journal of clinical investigation.

[58]  T. Karrison Versatile Tests for Comparing Survival Curves Based on Weighted Log-rank Statistics , 2016 .

[59]  M. Buyse,et al.  The Net Chance of a Longer Survival as a Patient-Oriented Measure of Treatment Benefit in Randomized Clinical Trials. , 2016, JAMA oncology.

[60]  R. Pazdur,et al.  Response Rate as an Approval End Point in Oncology: Back to the Future. , 2016, JAMA oncology.

[61]  S. Barni,et al.  Early analysis of surrogate endpoints for metastatic melanoma in immune checkpoint inhibitor trials , 2016, Medicine.

[62]  Y. Shentu,et al.  Pembrolizumab versus docetaxel for previously treated, PD-L1-positive, advanced non-small-cell lung cancer (KEYNOTE-010): a randomised controlled trial , 2016, The Lancet.

[63]  Axel Hoos,et al.  Non-alcoholic steatohepatitis: emerging molecular targets and therapeutic strategies , 2016, Nature Reviews Drug Discovery.

[64]  M Buyse,et al.  Statistical controversies in clinical research: end points other than overall survival are vital for regulatory approval of anticancer agents. , 2016, Annals of oncology : official journal of the European Society for Medical Oncology.

[65]  Patrick Royston,et al.  Augmenting the logrank test in the design of clinical trials in which non-proportional hazards of the treatment effect may be anticipated , 2016, BMC Medical Research Methodology.

[66]  J. Radford Nivolumab for recurrent squamous-cell carcinoma of the head and neck , 2016, BDJ.

[67]  Rosemarie Mick,et al.  Statistical Challenges in the Design of Late-Stage Cancer Immunotherapy Studies , 2015, Cancer Immunology Research.

[68]  A. Ravaud,et al.  Nivolumab versus Everolimus in Advanced Renal-Cell Carcinoma. , 2015, The New England journal of medicine.

[69]  C. Rudin,et al.  Nivolumab versus Docetaxel in Advanced Nonsquamous Non-Small-Cell Lung Cancer. , 2015, The New England journal of medicine.

[70]  Tai-Tsang Chen Milestone Survival: A Potential Intermediate Endpoint for Immune Checkpoint Inhibitors. , 2015, Journal of the National Cancer Institute.

[71]  M. Burotto,et al.  Pseudoprogression and Immune-Related Response in Solid Tumors. , 2015, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[72]  Troy Guthrie,et al.  Talimogene Laherparepvec Improves Durable Response Rate in Patients With Advanced Melanoma. , 2015, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[73]  Sean Khozin,et al.  Overall response rate, progression-free survival, and overall survival with targeted and standard therapies in advanced non-small-cell lung cancer: US Food and Drug Administration trial-level and patient-level analyses. , 2015, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[74]  J. Wolchok,et al.  Five-year survival rates for treatment-naive patients with advanced melanoma who received ipilimumab plus dacarbazine in a phase III trial. , 2015, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[75]  D. Schadendorf,et al.  Nivolumab in previously untreated melanoma without BRAF mutation. , 2015, The New England journal of medicine.

[76]  Sy Han Chiou,et al.  Fitting Accelerated Failure Time Models in Routine Survival Analysis with R Package aftgee , 2014 .

[77]  Pamela A Shaw,et al.  Chimeric antigen receptor T cells for sustained remissions in leukemia. , 2014, The New England journal of medicine.

[78]  R. Emerson,et al.  PD-1 blockade induces responses by inhibiting adaptive immune resistance , 2014, Nature.

[79]  Deepak Mittal,et al.  New insights into cancer immunoediting and its three component phases--elimination, equilibrium and escape. , 2014, Current opinion in immunology.

[80]  David C. Smith,et al.  Survival, durable tumor remission, and long-term safety in patients with advanced melanoma receiving nivolumab. , 2014, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[81]  Patrick Royston,et al.  Restricted mean survival time: an alternative to the hazard ratio for the design and analysis of randomized trials with a time-to-event outcome , 2013, BMC Medical Research Methodology.

[82]  W. Henley,et al.  Bias and Sensitivity Analysis When Estimating Treatment Effects from the Cox Model with Omitted Covariates , 2013, Biometrics.

[83]  Tai-Tsang Chen Statistical issues and challenges in immuno-oncology , 2013, Journal of Immunotherapy for Cancer.

[84]  A. Giobbie-Hurder,et al.  Developing a Common Language for Tumor Response to Immunotherapy: Immune-Related Response Criteria Using Unidimensional Measurements , 2013, Clinical Cancer Research.

[85]  D. Sargent,et al.  Innovative estimation of survival using log-normal survival modelling on ACCENT database , 2013, British Journal of Cancer.

[86]  I. Tannock,et al.  Comparison of absolute benefits of anticancer therapies determined by snapshot and area methods. , 2012, Annals of oncology : official journal of the European Society for Medical Oncology.

[87]  P. Kawalec,et al.  Sipuleucel-T immunotherapy for castration-resistant prostate cancer. A systematic review and meta-analysis , 2012, Archives of medical science : AMS.

[88]  O. Finn,et al.  Immuno-oncology: understanding the function and dysfunction of the immune system in cancer. , 2012, Annals of oncology : official journal of the European Society for Medical Oncology.

[89]  S. Pocock,et al.  The win ratio: a new approach to the analysis of composite endpoints in clinical trials based on clinical priorities , 2011, European heart journal.

[90]  George Coukos,et al.  Cancer immunotherapy comes of age , 2011, Nature.

[91]  P. Royston,et al.  The use of restricted mean survival time to estimate the treatment effect in randomized clinical trials when the proportional hazards assumption is in doubt , 2011, Statistics in medicine.

[92]  Axel Hoos,et al.  Ipilimumab plus dacarbazine for previously untreated metastatic melanoma. , 2011, The New England journal of medicine.

[93]  M. Buyse Generalized pairwise comparisons of prioritized outcomes in the two‐sample problem , 2010, Statistics in medicine.

[94]  J. Banchereau,et al.  Assessing oncologic benefit in clinical trials of immunotherapy agents. , 2010, Annals of oncology : official journal of the European Society for Medical Oncology.

[95]  Sylvia Janetzki,et al.  Improved Endpoints for Cancer Immunotherapy Trials , 2010, Journal of the National Cancer Institute.

[96]  D. Schadendorf,et al.  Improved survival with ipilimumab in patients with metastatic melanoma. , 2010, The New England journal of medicine.

[97]  P. Kantoff,et al.  Sipuleucel-T immunotherapy for castration-resistant prostate cancer. , 2010, The New England journal of medicine.

[98]  Song Yang,et al.  Improved Logrank‐Type Tests for Survival Data Using Adaptive Weights , 2010, Biometrics.

[99]  T. Mok,et al.  Gefitinib or carboplatin-paclitaxel in pulmonary adenocarcinoma. , 2009, The New England journal of medicine.

[100]  Axel Hoos,et al.  Guidelines for the Evaluation of Immune Therapy Activity in Solid Tumors: Immune-Related Response Criteria , 2009, Clinical Cancer Research.

[101]  L. Schwartz,et al.  New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). , 2009, European journal of cancer.

[102]  K. Carroll,et al.  Analysis of duration of response in oncology trials. , 2008, Contemporary clinical trials.

[103]  M. Buyse,et al.  Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer. , 2008, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[104]  K. Pritchard,et al.  Ascertaining Prognosis for Breast Cancer in Node‐Negative Patients with Innovative Survival Analysis , 2006, The breast journal.

[105]  S. Rosenberg,et al.  Autoimmunity correlates with tumor regression in patients with metastatic melanoma treated with anti-cytotoxic T-lymphocyte antigen-4. , 2005, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[106]  Min-Hsiao Tsai,et al.  SOME VERSATILE TESTS BASED ON THE SIMULTANEOUS USE OF WEIGHTED LOGRANK AND WEIGHTED KAPLAN-MEIER STATISTICS , 2001 .

[107]  Geert Molenberghs,et al.  Relation between tumour response to first-line chemotherapy and survival in advanced colorectal cancer: a meta-analysis , 2000, The Lancet.

[108]  Jae Won Lee,et al.  SOME VERSATILE TESTS BASED ON THE SIMULTANEOUS USE OF WEIGHTED LOG-RANK STATISTICS , 1996 .

[109]  K. Anderson,et al.  New models for predicting cardiovascular events. , 1994, Journal of clinical epidemiology.

[110]  K. Anderson,et al.  A nonproportional hazards Weibull accelerated failure time regression model. , 1991, Biometrics.

[111]  D. Zucker,et al.  Weighted log rank type statistics for comparing survival curves when there is a time lag in the effectiveness of treatment , 1990 .

[112]  M S Pepe,et al.  Weighted Kaplan-Meier statistics: a class of distance tests for censored survival data. , 1989, Biometrics.

[113]  T. Morgan Analysis of duration of response: a problem of oncology trials. , 1988, Controlled clinical trials.

[114]  D. Harrington A class of rank test procedures for censored survival data , 1982 .

[115]  I. James,et al.  Linear regression with censored data , 1979 .