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Surrogate endpoints

Paneez Khoury, Praveen Akuthota, Steven J Ackerman, Joseph R Arron, Bruce S Bochner, Margaret H Collins, Jean-Emmanuel Kahn, Patricia C Fulkerson, Gerald J Gleich, Rashmi Gopal-Srivastava, Elizabeth A Jacobsen, Kristen M Leiferman, Levi-Schaffer Francesca, Sameer K Mathur, Michael Minnicozzi, Calman Prussin, Marc E Rothenberg, Florence Roufosse, Kathleen Sable, Dagmar Simon, Hans-Uwe Simon, Lisa A Spencer, Jonathan Steinfeld, Andrew J Wardlaw, Michael E Wechsler, Peter F Weller, Amy D Klion
BACKGROUND: Eosinophil-associated diseases (EADs) are rare, heterogeneous disorders characterized by the presence of eosinophils in tissues and/or peripheral blood resulting in immunopathology. The heterogeneity of tissue involvement, lack of sufficient animal models, technical challenges in working with eosinophils, and lack of standardized histopathologic approaches have hampered progress in basic research. Additionally, clinical trials and drug development for rare EADs are limited by the lack of primary and surrogate endpoints, biomarkers, and validated patient-reported outcomes...
April 19, 2018: Journal of Leukocyte Biology
Patricia de Cremoux, Lucie Biard, Brigitte Poirot, Philippe Bertheau, Luis Teixeira, Jacqueline Lehmann-Che, Fatiha A Bouhidel, Pascal Merlet, Marc Espié, Matthieu Resche-Rigon, Christos Sotiriou, David Groheux
Background: The efficacy of neoadjuvant chemotherapy regimens in advanced luminal breast cancer patients is difficult to predict. Intrinsic properties of breast tumors, including altered gene expression profile and dynamic evaluation of metabolic properties of tumor cells using positron emission tomography/computed tomography (PET/CT) of tumor cells, have been identified to guide patient's prognosis. The aim of this study is to determine if both analyses may improve the prediction of response to neoadjuvant chemotherapy in ER-positive / HER2-negative breast cancers (BCs) patients...
March 27, 2018: Oncotarget
Wei Wei, Qianqian Sun, Yiwen Deng, Yufeng Wang, Guanhuan Du, Chencheng Song, Chenxi Li, Mengxue Zhu, Guangjie Chen, Guoyao Tang
OBJECTIVE: The aim of this study was to measure T helper (Th) 1/Th2-related cytokine expression in saliva from patients with oral lichen planus (OLP), compared with healthy controls (HC group) and controls with recurrent aphthous ulcers (RAU group). STUDY DESIGN: Saliva was collected from 41 patients with OLP, 14 HCs, and 14 controls with RAU for Th1/Th2-related cytokines analysis with cytometric bead array. Disease activity in OLP was recorded by reticulation/keratosis, erythema, and ulceration scores...
March 6, 2018: Oral Surgery, Oral Medicine, Oral Pathology and Oral Radiology
Madhukar Pai, Samuel G Schumacher, Seye Abimbola
No abstract text is available yet for this article.
2018: BMJ Global Health
Elisabeth S Bergen, Rupert Bartsch
A combination of anthracyclines and taxanes remains the standard of care for neoadjuvant chemotherapy (NACT) resulting in increased breast conservation rate (BCR) and decreased recurrence rate [1]. Whether pathological complete response (pCR) could be an appropriate surrogate parameter for long-term survival is still a matter of debate. In patients with triple-negative breast cancer (TNBC) and HER2-positive breast cancer (BC), a six to nine times higher risk for relapse has been reported if no pCR was achieved [2, 3]...
2018: Memo
Gianluigi Savarese, Erwan Donal, Camilla Hage, Emmanuel Oger, Hans Persson, Jean-Claude Daubert, Cecilia Linde, Lars H Lund
No abstract text is available yet for this article.
March 27, 2018: International Journal of Cardiology
Katharina Schilbach, Daniel S Olsson, Margaret C S Boguszewski, Martin Bidlingmaier, Gudmundur Johannsson, Jens-Otto Lunde Jørgensen
Growth hormone (GH) and IGF-I levels in serum are used as biomarkers in the diagnosis and management of GH-related disorders but have not been subject to structured validation. Auxological parameters in children and changes in body composition in adults, as well as metabolic parameters and patient related outcomes are used as clinical and surrogate endpoints. New treatment options, such as long acting GH and GH antagonists, require reevaluation of the currently used biochemical biomarkers. This article will review biomarkers, surrogate endpoints and clinical endpoints related to GH treatment in children and adults as well as in acromegaly...
March 20, 2018: Growth Hormone & IGF Research
Maryam B Lustberg, Daniel G Stover, Jeffrey J Chalmers
A primary goal of personalized medicine is to develop tumor-specific biomarkers to aid in treatment selection and to better evaluate response to targeted therapies. The assessment of circulating blood markers as surrogate real-time biopsies of disease status, termed liquid biopsies, has been under investigation. There are many different types of liquid biopsies each with different functionalities and limitations. These include tumor markers, circulating tumor cells, cell-free DNA, and extracellular vesicles including exosomes...
March 2018: Cancer Journal
Franz H Messerli, Sripal Bangalore, Chirag Bavishi, Stefano F Rimoldi
Most guidelines for the management of patients with cardiovascular disease recommend angiotensin-converting enzyme (ACE) inhibitors as first-choice therapy, whereas angiotensin receptor blockers (ARBs) are merely considered an alternative for ACE inhibitor-intolerant patients. The aim of this review was to compare outcomes and adverse events between ACE inhibitors and ARBs in patients. In patients with hypertension and hypertension with compelling indications, we found no difference in efficacy between ARBs and ACE inhibitors with regard to the surrogate endpoint of blood pressure and outcomes of all-cause mortality, cardiovascular mortality, myocardial infarction, heart failure, stroke, and end-stage renal disease...
April 3, 2018: Journal of the American College of Cardiology
Gianluigi Savarese, Nicola Orsini, Camilla Hage, Ulf Dahlström, Ola Vedin, Giuseppe M C Rosano, Lars H Lund
BACKGROUND: Aim was to characterize N-terminal pro-B-type natriuretic peptide (NT-proBNP) in terms of determinants of levels and of its prognostic and discriminatory role in heart failure with mid-range (HFmrEF) vs. preserved (HFpEF) and reduced (HFrEF) ejection fraction. METHODS AND RESULTS: In 9,847 out-patients with HFpEF (n=1,811;18%), HFmrEF (n=2,122;22%) and HFrEF (n=5,914;60%) enrolled in the Swedish Heart Failure Registry, median NT-proBNP was 1,428, 1,540 and 2,288 pg/ml, respectively...
March 26, 2018: Journal of Cardiac Failure
Claudia C Dobler, Rebecca L Morgan, Yngve Falck-Ytter, Victor M Montori, M Hassan Murad
Surrogate endpoints are often used in clinical trials, as they allow for indirect measures of outcomes (eg, shorter trials with less participants). Improvements in surrogate endpoints (eg, reduction in low density lipoprotein cholesterol, normalisation of glycated haemoglobin) achieved with an intervention are, however, not always associated with improvements in patient-important outcomes. The common tendency in evidence-based medicine is to view results based on surrogate endpoints as less certain than results based on long term, final patient-important outcomes and rate them as 'lower quality evidence'...
April 2018: BMJ evidence-based medicine
Mototsugu Shimokawa, Takahiro Kogawa, Takako Shimada, Toshiaki Saito, Hozumi Kumagai, Masafumi Ohki, Tsunehisa Kaku
Purpose: A growing number of treatment options and active compounds in treatments have led to better outcomes for patients with advanced or recurrent epithelial ovarian cancer. We examined the association between progression-free survival (PFS), post-progression survival (PPS) and overall survival (OS) in phase III trials of second- and third-line chemotherapy for advanced or recurrent epithelial ovarian cancer. We aim to determine whether PFS or PPS is a surrogate of OS so that we can decide progress of disease is optimal endpoint for ovarian cancer...
2018: Journal of Cancer
Justin Stebbing, Yauheni Baranau, Alexey Manikhas, Sang Joon Lee, Paul Thiruchelvam, Daniel Leff, Francisco J Esteva
Trastuzumab is a key drug in the neoadjuvant treatment of breast cancers that overexpress the human epidermal growth factor receptor 2 (HER2). Pathological complete response (pCR) is commonly used as an endpoint in neoadjuvant clinical trials of trastuzumab as evidence suggests it may be a surrogate for long-term survival. Several biosimilar candidates of originator or 'reference' trastuzumab are in development and have used pCR as a primary endpoint to assess therapeutic equivalence between treatments. The exact definition of pCR has varied across studies...
March 27, 2018: Expert Review of Anticancer Therapy
Nicholas McAndrew, Angela DeMichele
The optimal neoadjuvant chemotherapy (NACT) regimen in triple-negative breast cancer (TNBC) has not been clearly defined. Achieving a pathologic complete response (pCR) provides important prognostic information, and, especially in TNBC, is considered a surrogate endpoint for event-free survival. Thus, many neoadjuvant studies in TNBC focus on this as a primary endpoint, and such information may be used for accelerated US Food and Drug Administration approval. Current controversies in the field include: (1) the role of platinum-based compounds; (2) the optimal chemotherapy backbone; and (3) the benefits of additional therapy after surgery...
February 2018: Journal of Targeted Therapies in Cancer
Frank Weber, Laurence C Walhout, Johanna C Escher
BACKGROUND: Propofol is often used for procedural sedation in children undergoing gastrointestinal endoscopy. Reliable assessment of the depth of hypnosis during the endoscopic procedure is challenging. Processed electroencephalography using the Narcotrend Index can help titrating propofol to a predefined sedation level. AIMS: The aim of this trial was to investigate the impact of Narcotrend Index-guided titration of propofol delivery on the speed of recovery. METHODS: Children, aged 12-17 years, undergoing gastrointestinal endoscopy under procedural sedation, had propofol delivered via target controlled infusion either based on Narcotrend Index guidance (group NI) or standard clinical parameters (group C)...
March 25, 2018: Paediatric Anaesthesia
Cyriel Y Ponsioen, Keith D Lindor, Ruby Mehta, Lara Dimick-Santos
Primary sclerosing cholangitis is a rare and chronic liver disease for which there is no effective therapy. There has been growing interest in developing treatments for this condition with several agents being proposed as potential therapies. However, there is lack of clarity about how to measure clinical benefit in trials in patients with this complex and rare disease. This article reviews regulatory information, the available literature on natural history, as well as potential candidate clinical and surrogate endpoints...
March 25, 2018: Hepatology: Official Journal of the American Association for the Study of Liver Diseases
Davide Maccagni, Susanna Benincasa, Barbara Bellini, Luciano Candilio, Enrico Poletti, Mauro Carlino, Antonio Colombo, Lorenzo Azzalini
Chronic total occlusions (CTO) percutaneous coronary intervention (PCI) is associated with high radiation dose. Our study aim was to evaluate the impact of the implementation of a noise reduction technology (NRT) on patient radiation dose during CTO PCI. A total of 187 CTO PCIs performed between February 2016 and May 2017 were analyzed according to the angiographic systems utilized: Standard (n = 60) versus NRT (n = 127). Propensity score matching (PSM) was performed to control for differences in baseline characteristics...
March 23, 2018: International Journal of Cardiovascular Imaging
Lisa Stamp, Melanie B Morillon, William J Taylor, Nicola Dalbeth, Jasvinder A Singh, Marissa Lassere, Robin Christensen
OBJECTIVES: The primary efficacy outcome in trials of urate lowering therapy (ULT) for gout is serum urate (SU). The aim of this study was to examine the strength of the relationship between SU and patient-important outcomes to determine whether SU is an adequate surrogate endpoint for clinical trials. METHODS: Multiple databases through October 2017 were searched. Randomized controlled trials comparing any ULT in people with gout with any control or placebo, ≥three months duration were included...
February 21, 2018: Seminars in Arthritis and Rheumatism
Joshua D Wallach, Oriana Ciani, Alison M Pease, Gregg S Gonsalves, Harlan M Krumholz, Rod S Taylor, Joseph S Ross
BACKGROUND: The U.S. Food and Drug Administration (FDA) often approves new drugs based on trials that use surrogate markers for endpoints, which involve certain trade-offs and may risk making erroneous inferences about the medical product's actual clinical effect. This study aims to compare the treatment effects among pivotal trials supporting FDA approval of novel therapeutics based on surrogate markers of disease with those observed among postapproval trials for the same indication...
March 21, 2018: BMC Medicine
P Scott Pine, Steven P Lund, Sanford A Stass, Debra Kukuruga, Feng Jiang, Lynn Sorbara, Sudhir Srivastava, Marc Salit
BACKGROUND: We demonstrate the feasibility of creating a pair of reference samples to be used as surrogates for clinical samples measured in either a research or clinical laboratory setting. The reference sample paradigm presented and evaluated here is designed to assess the capability of a measurement process to detect true differences between two biological samples. Cell-based reference samples can be created with a biomarker signature pattern designed in silico. Clinical laboratories working in regulated applications are required to participate in proficiency testing programs; research laboratories doing discovery typically do not...
March 20, 2018: BMC Biotechnology
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