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Clinical trial end point and surrogate

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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
#1
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
#2
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
#3
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
#4
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
#5
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
#6
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
#7
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
#8
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
#9
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
#10
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
#11
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
#12
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)...
June 22, 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
#13
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
#14
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
#15
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
https://www.readbyqxmd.com/read/28465118/development-and-validation-of-a-major-adverse-transplant-event-mate-score-to-predict-late-graft-loss-in-pediatric-heart-transplantation
#16
Christopher S Almond, Helena Hoen, Joseph W Rossano, Chesney Castleberry, Scott R Auerbach, Lingyao Yang, Ashwin K Lal, Melanie D Everitt, Matthew Fenton, Seth A Hollander, Elfriede Pahl, Elizabeth Pruitt, David N Rosenthal, Doff B McElhinney, Kevin P Daly, Manisha Desai
BACKGROUND: There is inadequate power to perform a valid clinical trial in pediatric heart transplantation (HT) using a conventional end-point, because the disease is rare and hard end-points, such as death or graft loss, are infrequent. We sought to develop and validate a surrogate end-point involving the cumulative burden of post-transplant complications to predict death/graft loss to power a randomized clinical trial of maintenance immunosuppression in pediatric HT. METHODS: Pediatric Heart Transplant Study (PHTS) data were used to identify all children who underwent an isolated orthotopic HT between 2005 and 2014 who survived to 6 months post-HT...
March 24, 2017: Journal of Heart and Lung Transplantation
https://www.readbyqxmd.com/read/28450365/critical-issues-for-the-translation-of-cardioprotection
#17
REVIEW
Gerd Heusch
The translation from numerous successful animal experiments on cardioprotection beyond that by reperfusion to clinical practice has to date been disappointing. Animal experiments often use reductionist approaches and are mostly performed in young and healthy animals which lack the risk factors, comorbidities, and comedications which are characteristics of patients suffering an acute myocardial infarction or undergoing cardiovascular surgery. Conceptually, it is still unclear by how much the time window for successful reperfusion is extended by preconditioning, and how long the duration of ischemia can be so that adjunct cardioprotection by postconditioning at reperfusion still protects...
April 28, 2017: Circulation Research
https://www.readbyqxmd.com/read/28417858/-18-f-fdg-pet-ct-imaging-of-thoracic-and-extrathoracic-tuberculosis-in-children
#18
REVIEW
Matthieu Pelletier-Galarneau, Patrick Martineau, Lionel S Zuckier, Xuan Pham, Raymond Lambert, Sophie Turpin
Tuberculosis (TB) remains a major health problem, affecting approximately one-third of the world׳s population. The tubercle bacillus can affect virtually any organ of the human body and if left untreated can lead to severe morbidity and death. Diagnosis of active TB is challenging, especially in children. As a "great imitator," the disease can mimic numerous other pathologies, both clinically and at imaging. Although recognition of active TB is crucial to initiate adequate treatment in a timely fashion, thereby preventing transmission of disease, differentiation of active and quiescent disease is not always straightforward...
May 2017: Seminars in Nuclear Medicine
https://www.readbyqxmd.com/read/28397190/surrogate-end-points-for-overall-survival-in-metastatic-locally-advanced-or-unresectable-pancreatic-cancer-a-systematic-review-and-meta-analysis-of-24-randomized-controlled-trials
#19
Eleftherios A Makris, Regina MacBarb, Danielle J Harvey, George A Poultsides
BACKGROUND: Overall survival (OS) has traditionally been the primary end point in studies evaluating the clinical benefit of first-line chemotherapy in metastatic, locally advanced, or unresectable pancreatic cancer (MLAUPC). Given the prolonged follow-up assessment required to obtain OS and its potential to be confounded by second-line treatments, this study sought to determine whether progression-free survival (PFS), response rate (RR), or disease control rate (DCR) can serve as a reliable surrogate for OS...
August 2017: Annals of Surgical Oncology
https://www.readbyqxmd.com/read/28337673/progression-free-survival-as-a-surrogate-for-overall-survival-in-clinical-trials-of-targeted-therapy-in-advanced-solid-tumors
#20
Stefan Michiels, Everardo D Saad, Marc Buyse
Over the past 15 years, targeted therapy has revolutionized the systemic treatment of cancer. In parallel, there has been a growing debate on the choice of end points in clinical trials in oncology. This debate basically hinges on the choice between overall survival (OS) and progression-free survival (PFS). PFS is advantageous because it is measured earlier than OS, requires a smaller sample size than OS to achieve the desired power, and is not influenced by cross-over. On the other hand, PFS is prone to measurement error and bias, and may not capture the entire treatment effect on the outcomes of most interest to patients with an incurable disease: a prolonged survival and improved quality of life...
May 2017: Drugs
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