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Clinical trial end point and surrogate

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https://www.readbyqxmd.com/read/29055841/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
#1
Giandomenico Roviello, Fabrice Andre, Sergio Venturini, Barbara Pistilli, Giuseppe Curigliano, Massimo Cristofanilli, Pietro Rosellini, Daniele Generali
INTRODUCTION: To assess the role of the tumour response rate (RR) after immune checkpoint inhibitors-based therapy as a potential surrogate end-point of progression-free survival (PFS) and overall survival (OS) in patients with solid tumours, we performed a trial-based meta-regression of randomised studies comparing different immune checkpoint inhibitors-based treatments. METHODS: The systematic literature search included the electronic databases and the proceedings of oncologic meetings...
November 2017: European Journal of Cancer
https://www.readbyqxmd.com/read/29020339/can-viral-load-be-used-as-a-surrogate-marker-in-clinical-studies-of-cytomegalovirus-in-solid-organ-transplantation-a-systematic-review-and-meta-analysis
#2
Yoichiro Natori, Ali Alghamdi, Mahmood Tazari, Veronica Miller, Shahid Husain, Takashi Komatsu, Paul Griffiths, Per Ljungman, Ani Orchanian-Cheff, Deepali Kumar, Atul Humar
Symptomatic CMV disease has been the standard endpoint for clinical trials in organ transplant recipients. Viral load may be a more relevant endpoint due to low frequency of disease. We performed a meta-analysis and systematic review of the literature. We found several lines of evidence to support the validity of viral load as an appropriate surrogate end-point including 1)viral loads in CMV disease are significantly greater than in asymptomatic viremia (OR 9.3, 95%CI 4.6 to 19.3); 2)kinetics of viral replication are strongly associated with progression to disease; 3)pooled incidence of CMV viremia and disease is significantly lower during prophylaxis compared with the full patient followup period (viremia incidence: 3...
September 5, 2017: Clinical Infectious Diseases: An Official Publication of the Infectious Diseases Society of America
https://www.readbyqxmd.com/read/29020259/report-of-an-esc-eapci-task-force-on-the-evaluation-and-use-of-bioresorbable-scaffolds-for-percutaneous-coronary-intervention-executive-summary
#3
Robert A Byrne, Giulio G Stefanini, Davide Capodanno, Yoshinobu Onuma, Andreas Baumbach, Javier Escaned, Michael Haude, Stefan James, Michael Joner, Peter Jüni, Adnan Kastrati, Semih Oktay, William Wijns, Patrick W Serruys, Stephan Windecker
A previous Task Force of the European Society of Cardiology (ESC) and European Association of Percutaneous Cardiovascular Interventions (EAPCI) provided a report on recommendations for the non-clinical and clinical evaluation of coronary stents. Following dialogue with the European Commission, the Task Force was asked to prepare an additional report on the class of devices known as bioresorbable scaffolds (BRS). Five BRS have CE-mark approval for use in Europe. Only one device-the Absorb bioresorbable vascular scaffold-has published randomized clinical trial data and this data show inferior outcomes to conventional drug-eluting stents (DES) at 2-3 years...
August 28, 2017: European Heart Journal
https://www.readbyqxmd.com/read/29019452/minimal-residual-disease-analysis-in-myeloma-when-why-and-where
#4
Uday Yanamandra, Shaji K Kumar
The primary hurdle in the path to curing multiple myeloma (MM) is defining a validated minimal residual disease (MRD) and its utility in the therapeutic decision making. A better definition of MRD will aid in tailoring MM therapy further to address the clonal heterogeneity and genomic instability and overcome patient's ineffective immune surveillance. MRD analysis can define the logical endpoint for maintenance therapy, in addition also aids in providing a better clinical end point for studies comparing novel agents in myeloma...
October 11, 2017: Leukemia & Lymphoma
https://www.readbyqxmd.com/read/28948934/report-of-an-esc-eapci-task-force-on-the-evaluation-and-use-of-bioresorbable-scaffolds-for-percutaneous-coronary-intervention-executive-summary
#5
Robert A Byrne, Giulio F Stefanini, Davide Capodanno, Yoshinobu Onuma, Andreas Baumbach, Javier Escaned, Michael Haude, Stefan James, Michael Joner, Peter Ju Ni, Adnan Kastrati, Semih Oktay, William Wijns, Patrick W Serruys, Stephan Windecker
A previous Task Force of the European Society of Cardiology (ESC) and European Association of Percutaneous Cardiovascular Interventions (EAPCI) provided a report on recommendations for the nonclinical and clinical evaluation of coronary stents. Following dialogue with the European Commission, the Task Force was asked to prepare an additional report on the class of devices known as bioresorbable scaffolds (BRS). Five BRS have CE-mark approval for use in Europe. Only one device - the Absorb bioresorbable vascular scaffold - has published randomized clinical trial data and this data show inferior outcomes to conventional drug-eluting stents (DES) at 2-3 years...
September 25, 2017: EuroIntervention
https://www.readbyqxmd.com/read/28889347/decline-in-estimated-glomerular-filtration-rate-is-associated-with-risk-of-end-stage-renal-disease-in-type-2-diabetes-with-macroalbuminuria-an-observational-study-from-jdncs
#6
Miho Shimizu, Kengo Furuichi, Tadashi Toyama, Tomoaki Funamoto, Shinji Kitajima, Akinori Hara, Daisuke Ogawa, Daisuke Koya, Kenzo Ikeda, Yoshitaka Koshino, Yukie Kurokawa, Hideharu Abe, Kiyoshi Mori, Masaaki Nakayama, Yoshio Konishi, Ken-Ichi Samejima, Masaru Matsui, Hiroyuki Yamauchi, Tomohito Gohda, Kei Fukami, Daisuke Nagata, Hidenori Yamazaki, Yukio Yuzawa, Yoshiki Suzuki, Shouichi Fujimoto, Shoichi Maruyama, Sawako Kato, Takero Naito, Kenichi Yoshimura, Hitoshi Yokoyama, Takashi Wada
BACKGROUND: There is increased interest in surrogate endpoints for clinical trials of chronic kidney disease. METHODS: In this nationwide observational study of 456 patients with type 2 diabetes and clinically suspected diabetic nephropathy followed for a median of 4.2 years, we evaluated the association between estimated glomerular filtration rate (eGFR) and albuminuria at baseline or during follow-up and risk of ESRD. RESULTS: Low eGFR (<60 mL/min/1...
September 9, 2017: Clinical and Experimental Nephrology
https://www.readbyqxmd.com/read/28818493/progression-free-survival-and-time-to-progression-as-real-surrogate-end-points-for-overall-survival%C3%A2-in-advanced-breast-cancer-a-meta-analysis-of-37-trials
#7
Ling Li, Zhanyu Pan
BACKGROUND: Progression-free survival (PFS) and time to progression (TTP) have been reported to correlate with overall survival (OS) in several cancer types. To our knowledge, however, the correlation between them is unclear. METHODS: A literature-based meta-analysis was performed to assess whether PFS and TTP can be considered reliable surrogate end points for OS in a phase 3 clinical trial of advanced breast cancer (ABC). The median hazard ratios of PFS/TTP and OS were analyzed by determining their nonparametric Spearman rank correlation coefficients (Rs)...
July 25, 2017: Clinical Breast Cancer
https://www.readbyqxmd.com/read/28810023/characteristics-of-preapproval-and-postapproval-studies-for-drugs-granted-accelerated-approval-by-the-us-food-and-drug-administration
#8
Huseyin Naci, Katelyn R Smalley, Aaron S Kesselheim
Importance: Drugs treating serious or life-threatening conditions can receive US Food and Drug Administration (FDA) accelerated approval based on showing an effect in surrogate measures that are only reasonably likely to predict clinical benefit. Confirmatory trials are then required to determine whether these effects translate to clinical improvements. Objective: To characterize preapproval and confirmatory clinical trials of drugs granted accelerated approval...
August 15, 2017: JAMA: the Journal of the American Medical Association
https://www.readbyqxmd.com/read/28810022/characteristics-of-clinical-studies-used-for-us-food-and-drug-administration-approval-of-high-risk-medical-device-supplements
#9
Sarah Y Zheng, Sanket S Dhruva, Rita F Redberg
Importance: High-risk medical devices often undergo modifications, which are approved by the US Food and Drug Administration (FDA) through various kinds of premarket approval (PMA) supplements. There have been multiple high-profile recalls of devices approved as PMA supplements. Objective: To characterize the quality of the clinical studies and data (strength of evidence) used to support FDA approval of panel-track supplements (a type of PMA supplement pathway that is used for significant changes in a device or indication for use and always requires clinical data)...
August 15, 2017: JAMA: the Journal of the American Medical Association
https://www.readbyqxmd.com/read/28796988/microvessel-density-as-a-surrogate-prognostic-marker-in-patients-with-multiple-myeloma-a-meta-analysis
#10
Panagiotis Ntellas, Konstantinos Perivoliotis, Katerina Dadouli, Georgios K Koukoulis, Maria Ioannou
BACKGROUND/AIMS: Bone marrow (BM) angiogenesis is considered a hallmark of multiple myeloma (MM) development and progression, and can be quantified with the use of microvessel density (MVD). The purpose of this study is to provide a review and a meta-analysis of the current literature regarding the prognostic value of MVD in the overall survival (OS) of MM patients. METHODS: MEDLINE was screened for studies evaluating the OS of MM patients with regard to their MVD count in BM trephine...
2017: Acta Haematologica
https://www.readbyqxmd.com/read/28796587/metastasis-free-survival-is-a-strong-surrogate-of-overall-survival-in-localized-prostate-cancer
#11
Wanling Xie, Meredith M Regan, Marc Buyse, Susan Halabi, Philip W Kantoff, Oliver Sartor, Howard Soule, Noel W Clarke, Laurence Collette, James J Dignam, Karim Fizazi, Wendy R Paruleker, Howard M Sandler, Matthew R Sydes, Bertrand Tombal, Scott G Williams, Christopher J Sweeney
Purpose Adjuvant therapy for intermediate-risk and high-risk localized prostate cancer decreases the number of deaths from this disease. Surrogates for overall survival (OS) could expedite the evaluation of new adjuvant therapies. Methods By June 2013, 102 completed or ongoing randomized trials were identified and individual patient data were collected from 28 trials with 28,905 patients. Disease-free survival (DFS) and metastasis-free survival (MFS) were determined for 21,140 patients from 24 trials and 12,712 patients from 19 trials, respectively...
September 20, 2017: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28790165/prognostic-value-of-fractional-flow-reserve-measured-immediately-after-drug-eluting-stent-implantation
#12
Zsolt Piroth, Gabor G Toth, Pim A L Tonino, Emanuele Barbato, Soheila Aghlmandi, Nick Curzen, Gilles Rioufol, Nico H J Pijls, William F Fearon, Peter Jüni, Bernard De Bruyne
BACKGROUND: The predictive value of fractional flow reserve (FFR) measured immediately after percutaneous coronary intervention (PCI) with drug-eluting stent placement has not been prospectively investigated. We investigated the potential of post-PCI FFR measurements to predict clinical outcome in patients from FAME 1 and 2 trials (Fractional Flow Reserve or Angiography for Multivessel Evaluation). METHODS AND RESULTS: All patients of FAME 1 and FAME 2 who had post-PCI FFR measurement were included...
August 2017: Circulation. Cardiovascular Interventions
https://www.readbyqxmd.com/read/28750119/minimal-residual-disease-in-chronic-lymphocytic-leukemia-in-the-era-of-novel-agents-a-review
#13
Meghan Thompson, Danielle Brander, Chadi Nabhan, Anthony Mato
Importance: The landscape of chronic lymphocytic leukemia (CLL) treatment has changed considerably since the first reported assessment of minimal residual disease (MRD) by flow cytometry in 1992. Chemoimmunotherapy (CIT) combinations have become the standard of care for most patients, and novel targeted agents are rapidly being incorporated into the front-line and relapsed settings. Minimal residual disease status has been shown to be a predictor of both progression-free survival (PFS) and overall survival (OS) at the time of response assessment following CIT, but less is known about the relationship between MRD and outcomes after novel oral therapeutics...
July 27, 2017: JAMA Oncology
https://www.readbyqxmd.com/read/28730989/users-guide-to-the-surgical-literature-how-to-assess-an-article-using-surrogate-end-points
#14
Lucas Gallo, Cagla Eskicioglu, Luis H Braga, Forough Farrokhyar, Achilleas Thoma
Phase 3 randomized controlled trials are the widely accepted gold standard through which treatment decisions are made, as they assess the efficacy of a novel treatment against the control on the relevant patient population. The effectiveness of the novel treatment should be derived by measuring patient-important outcomes; however, to accurately assess these outcomes, clinical trials often require extensive patient follow-up and large sample sizes that can incur substantial expense. For this reason, investigators substitute surrogate end points to reduce the sample size and duration of a trial, ultimately reducing cost...
August 2017: Canadian Journal of Surgery. Journal Canadien de Chirurgie
https://www.readbyqxmd.com/read/28718326/cns-anticancer-drug-discovery-and-development-2016-conference-insights
#15
Victor A Levin, Lauren E Abrey, Timothy P Heffron, Peter J Tonge, Arvin C Dar, William A Weiss, James M Gallo
CNS Anticancer Drug Discovery and Development November 2016, AZ, USA The 2016 second CNS Anticancer Drug Discovery and Development Conference addressed diverse viewpoints about why new drug discovery/development focused on CNS cancers has been sorely lacking. Despite more than 70,000 individuals in the USA being diagnosed with a primary brain malignancy and 151,669-286,486 suffering from metastatic CNS cancer, in 1999, temozolomide was the last drug approved by the US FDA as an anticancer agent for high-grade gliomas...
July 18, 2017: CNS Oncology
https://www.readbyqxmd.com/read/28712621/dealing-with-uncertainty-and-accounting-for-social-value-judgments-in-assessments-of-orphan-drugs-evidence-from-four-european-countries
#16
Elena Nicod, Karen Berg Brigham, Isabelle Durand-Zaleski, Panos Kanavos
OBJECTIVES: To better understand the reasons for differences in reimbursement decisions for orphan drugs in four European countries that were not readily apparent from health technology assessment (HTA) reports and operating procedures. METHODS: Semistructured interviews with representatives of HTA bodies in England, Scotland, Sweden, and France were conducted. An interview topic guide was developed on the basis of findings from a systematic comparison of HTA decisions for 10 orphan drugs...
July 2017: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/28648303/risk-of-esrd-and-mortality-associated-with-change-in-filtration-markers
#17
Casey M Rebholz, Lesley A Inker, Yuan Chen, Menglu Liang, Meredith C Foster, John H Eckfeldt, Paul L Kimmel, Ramachandran S Vasan, Harold I Feldman, Mark J Sarnak, Chi-Yuan Hsu, Andrew S Levey, Josef Coresh
BACKGROUND: Using change in estimated glomerular filtration rate (eGFR) based on creatinine concentration as a surrogate outcome in clinical trials of chronic kidney disease has been proposed. Risk for end-stage renal disease (ESRD) and all-cause mortality associated with change in concentrations of other filtration markers has not been studied in chronic kidney disease populations. STUDY DESIGN: Observational analysis of 2 clinical trials. SETTING & PARTICIPANTS: Participants in the MDRD (Modification of Diet in Renal Disease; n=317) Study and AASK (African American Study of Kidney Disease and Hypertension; n=373)...
October 2017: American Journal of Kidney Diseases: the Official Journal of the National Kidney Foundation
https://www.readbyqxmd.com/read/28640707/response-assessment-in-neuro-oncology-clinical-trials
#18
REVIEW
Patrick Y Wen, Susan M Chang, Martin J Van den Bent, Michael A Vogelbaum, David R Macdonald, Eudocia Q Lee
Development of novel therapies for CNS tumors requires reliable assessment of response and progression. This requirement has been particularly challenging in neuro-oncology for which contrast enhancement serves as an imperfect surrogate for tumor volume and is influenced by agents that affect vascular permeability, such as antiangiogenic therapies. In addition, most tumors have a nonenhancing component that can be difficult to accurately quantify. To improve the response assessment in neuro-oncology and to standardize the criteria that are used for different CNS tumors, the Response Assessment in Neuro-Oncology (RANO) working group was established...
July 20, 2017: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28611186/success-failure-and-transparency-in-biomarker-based-drug-development-a-case-study-of-cholesteryl-ester-transfer-protein-inhibitors
#19
Spencer Phillips Hey, Jessica M Franklin, Jerry Avorn, Aaron S Kesselheim
BACKGROUND: Although biomarkers are used as surrogate measures for drug targeting and approval and are generally based on plausible biological hypotheses, some are found to not correlate well with clinical outcomes. Over-reliance on inadequately validated biomarkers in drug development can lead to harm to trial subjects and patients and to research waste. To shed greater light on the process and ethics of biomarker-based drug development, we conducted a systematic portfolio analysis of cholesterol ester transfer protein inhibitors, a drug class designed to improve lipid profiles and prevent cardiovascular events...
June 2017: Circulation. Cardiovascular Quality and Outcomes
https://www.readbyqxmd.com/read/28571579/how-could-we-enhance-translation-of-sepsis-immunology-to-inform-immunomodulation-trials-in-sepsis
#20
EDITORIAL
M Shankar-Hari
Sepsis results in complex alterations to the immune system. Our understanding of how these alterations in immune responses could help characterize extreme immune phenotypes, identify biomarkers with the ability to stratify patients for therapeutic interventions, surrogates in the causal pathway of clinical end-points, and treatable traits are still rudimentary. A methodologically rigorous, consensus-based approach should enrich sepsis immune subpopulations to increase the probability of successful trials.
May 26, 2017: Critical Care: the Official Journal of the Critical Care Forum
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