Search results for "Surrogate endpoint"

Measurement of exhaled nitric oxide: comparison of 3 different analyzers

2018

Exhaled nitric oxide (FeNO) is used as a surrogate marker to monitor airway inflammation. Aim of this study was to compare the new FeNO analyzer Vivatmo pro from Bosch (BV) with the Niox Vero from Circassia (CN) and the CLD from Ecomedics (EC). In 106 asthma patients (median age 54 years (range 20-87), 60 % female, median ACT of 16, 85 % on inhaled corticosteroids (mean 1300 μg BDP), 36 % on therapy with biologics) 2 FeNO measurements per patient and per device were performed using each analyzer in a random sequence according to the ATS / ERS guidelines. Additionally, the success rate to achieve a valid NO value was evaluated. 70 % of the patients had FeNO values In conclusion, for the rang…

Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients with Low-Intermediate Risk Myelodysplastic Syndromes (MDS): Long-Term Re…

2016

Abstract Background: Management of anemia is a common therapeutic challenge in patients with MDS. Luspatercept (ACE-536), a fusion protein containing modified activin receptor type IIB, is being developed for treatment of anemia in lower-risk MDS. Luspatercept binds GDF11 and other TGF-β superfamily ligands to promote late-stage erythroid differentiation and increase hemoglobin (Hgb) levels (Suragani R, Nat Med, 2014 and Attie K, Am J Hematol, 2014). Aims: This is an ongoing, phase 2, multicenter, open-label, long-term extension study to evaluate the effects of luspatercept in patients (pts) with low-intermediate risk MDS. Endpoints include long-term safety and tolerability, erythroid respo…

Pioneer: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent or Smoldering Systemic Mastocytosis w…

2019

Background: The gain-of function mutation KIT D816V plays a central role in the pathogenesis of systemic mastocytosis (SM). Among subtypes of SM, indolent SM (ISM) is the most frequent and is associated with normal/near-normal life expectancy; smoldering SM (SSM) is a subtype with a relatively higher burden of mast cells and may be associated with an increased risk of progression to advanced mastocytosis. However, both ISM and SSM patients may experience severe symptoms associated with mast cell mediator release, such as pruritus, diarrhea, anaphylaxis, and bone pain, which can be severely debilitating and have a profoundly negative impact on quality of life. Symptomatic treatments (eg, ant…

Recognition and characterization of small colonic neoplasia with high-definition colonoscopy using i-Scan is as precise as chromoendoscopy

2010

Abstract Background The EPKi system (Pentax, Japan) enables resolution above HDTV. Aim of the study was to test the efficacy of HD+ alone and with the new post-processing digital filter i-Scan or chromoendoscopy (Methylene blue 0.1%) in screening for colorectal cancer. We focused on lesions less than 5 mm as a surrogate marker for the optical possibilities of the EPKi system. Methods The last 30 cm of the colon in a screening population were inspected with HD+ alone, in combination with i-Scan (2:1 randomisation) and subsequently with chromoendoscopy. All lesions were characterized and targeted biopsies were performed. Results i-Scan augmented in 69 patients the identification of lesions fr…

Subclinical atherosclerosis: independent predictor of cardiovascular events in a 20-years follow up of patients with or without metabolic syndrome

2013

Purpose: To evaluate the influence of carotid atherosclerosis and metabolic syndrome (MetS) on the prediction of cardiovascular events during a 20-years follow-up. Methods: We studied a population of 529 asymptomatic patients, divided into 2 groups according to the results of the carotid ultrasound evaluation: one without atherosclerotic lesions (198 patients) and a second one with an increased carotid IMT or asymptomatic carotid plaque (331 patients). In each of these groups, we identified two subgroups of subjects with and without MetS. Cardiovascular endpoints were investigated in a 20-years follow-up: acute myocardial infarction, angina, transient ischemic attack, ischemic stroke, abdom…

Effects of empagliflozin on CA125 trajectory in patients with chronic congestive heart failure

2021

INTRODUCTION: We aimed to evaluate the trajectory of two surrogates of fluid overload -antigen carbohydrate 125 (CA125) and amino-terminal pro-brain natriuretic peptide (NT-proBNP)- after the addition of oral empagliflozin to usual care in a cohort of patients with chronic heart failure (CHF) and type 2 diabetes (T2D). METHODS AND RESULTS: From October 2015 to February 2019, 60 ambulatory patients with CHF and T2D were retrospectively included. The primary endpoint was to assess the longitudinal trajectory of plasma levels of CA125 and NT-proBNP after empagliflozin initiation. Changes in quantitative variables were evaluated using linear mixed regression. Median CA125 and NT-proBNP at basel…

Randomized clinical trial comparing liver resection with and without perioperative assessment of liver function

2018

Background Liver function tests may help to predict outcomes after liver surgery. The aim of this study was to evaluate the clinical impact on postoperative outcome and patient management of perioperative liver function testing using the LiMAx® test. Methods A multicentre RCT was conducted in six academic liver centres. Patients with intrahepatic tumours scheduled for open liver resection of at least one segment were eligible. Patients were randomized to undergo additional perioperative liver function tests (LiMAx® group) or standard care (control group). Patients in the intervention arm received two perioperative LiMAx® tests, one before the operation for surgical planning and another afte…

P275 Development and reliability of the new endoscopic virtual chromoendoscopy score: the PICaSSO score (the Paddington International Virtual Chromoe…

2017

CA125 but not NT-proBNP predicts the presence of a congestive intrarenal venous flow in patients with acute heart failure

2021

Abstract Background Intrarenal venous flow (IRVF) measured by Doppler ultrasound has gained interest as a potential surrogate marker of renal congestion and adverse outcomes in heart failure. In this work, we aimed to determine if antigen carbohydrate 125 (CA125) and plasma amino-terminal pro-B-type natriuretic peptide (NT-proBNP) are associated with congestive IRVF patterns (i.e., biphasic and monophasic) in acute heart failure (AHF). Methods and results We prospectively enrolled a consecutive cohort of 70 patients hospitalized for AHF. Renal Doppler ultrasound was assessed within the first 24-h of hospital admission. The mean age of the sample was 73.5 ± 12.3 years; 47.1% were female, and…

Antihypertensive treatment: should it be titrated to blood pressure reduction or to target organ damage regression?

2005

PURPOSE OF REVIEW The success of an antihypertensive treatment is difficult to estimate by the extent of blood pressure reduction alone. Above and beyond blood pressure values in an individual patient, it is necessary to monitor the impact of antihypertensive treatment on the development or regression of hypertension-induced early end-organ damage. The intermediate objectives or surrogate endpoints may provide additional valuable information about the level of success of a given therapy in a particular patient. Moreover, monitoring intermediate objectives may provide scientific evidence for delineating the best antihypertensive treatment. RECENT FINDINGS The importance of assessing left ven…