Aerosolized lancovutide in adolescents (≥12 years) and adults with cystic fibrosis - a randomized trial.

Background: Lancovutide activates a chloride channel (TMEM-16A) other than the cystic fibrosis (CF) transmembrane conductance regulator protein and could benefit CF patients.

Methods: In this randomized, multi-center, double-blind, placebo-controlled, parallel-group trial 161 patients ≥12 years with a confirmed diagnosis of CF were randomized to either placebo (saline) or active drug in 3 different dosing schemes of 2.5mg inhaled lancovutide (once daily, every other day or twice a week) for eight weeks.

Phenotype of children with inconclusive cystic fibrosis diagnosis after newborn screening.

Objective: To characterize the phenotypic expression of children with conductance regulator-related metabolic syndrome (CRMS)/cystic fibrosis screen positive inconclusive diagnosis (CFSPID) designation after positive newborn screening, reassign labeling if applicable and better define these children's prognosis.

Methods: A multicenter cohort with CRMS/CFSPID designation was matched with cystic fibrosis (CF)-diagnosed cohort. Cohorts were prospectively compared on baseline characteristics, cumulative data and when they reached 6 to 7 years at endpoint assessment.

Gait abnormalities in people with Dravet syndrome: A cross-sectional multi-center study.

Objective: To quantify gait abnormalities in people with Dravet syndrome (DS).

Methods: Individuals with a confirmed diagnosis of DS were enrolled, and stratified according to knee flexion at initial contact (IC) and range of motion (ROM) during stance (atypical crouch: knee flexion >20° at IC and knee ROM >15° during stance; straight: knee flexion <20° at IC). A 1D ANOVA (α = 0.

Intra-articular botulinum toxin injection in complex regional pain syndrome: Case report and review of the literature.

Complex regional pain syndrome (CRPS) is characterized by hyperalgesia, autonomic and trophic alterations of bones, muscles and skin. It is supported by neurogenic inflammation and impairment of sympathetic nervous system. Botulinum Toxin (BTX) is an option for the management of pain, with level B evidence of efficacy in neuropathic, joint and myofascial pain syndrome.

LRP1 expression in colon cancer predicts clinical outcome.

Unlabelled: LRP1 (low-density lipoprotein receptor-related protein 1), a multifunctional endocytic receptor, has recently been identified as a hub within a biomarker network for multi-cancer clinical outcome prediction. As its role in colon cancer has not yet been characterized, we here investigate the relationship between LRP1 and outcome.

Materials And Methods: mRNA expression was determined in colon adenocarcinoma and paired colon mucosa samples, as well as in stromal and tumor cells obtained after laser capture microdissection.

Sensitivity and specificity of different methods for cystic fibrosis-related diabetes screening: is the oral glucose tolerance test still the standard?

Background: Cystic fibrosis-related diabetes (CFRD) is a late cystic fibrosis (CF)-associated comorbidity whose prevalence is increasing sharply lifelong. Guidelines for glucose metabolism (GM) monitoring rely on the oral glucose tolerance test (OGTT). However, this test is neither sensitive nor specific.

Moderate intake of docosahexaenoic acid raises plasma and platelet vitamin E levels in cystic fibrosis patients.

Patients with cystic fibrosis have increased oxidative stress and impaired antioxidant systems. Moderate intake of docosahexaenoic acid (DHA) may favor the lowering of oxidative stress. In this randomized, double-blind, cross-over study, DHA or placebo capsules, were given daily to 10 patients, 5mg/kg for 2 weeks then 10mg/kg DHA for the next 2 weeks (or placebo).

Intercomparison of methods of coupling between convection and large-scale circulation: 2. Comparison over nonuniform surface conditions.

As part of an international intercomparison project, the weak temperature gradient (WTG) and damped gravity wave (DGW) methods are used to parameterize large-scale dynamics in a set of cloud-resolving models (CRMs) and single column models (SCMs). The WTG or DGW method is implemented using a configuration that couples a model to a reference state defined with profiles obtained from the same model in radiative-convective equilibrium. We investigated the sensitivity of each model to changes in SST, given a fixed reference state.

Intercomparison of methods of coupling between convection and large-scale circulation: 1. Comparison over uniform surface conditions.

As part of an international intercomparison project, a set of single-column models (SCMs) and cloud-resolving models (CRMs) are run under the weak-temperature gradient (WTG) method and the damped gravity wave (DGW) method. For each model, the implementation of the WTG or DGW method involves a simulated column which is coupled to a reference state defined with profiles obtained from the same model in radiative-convective equilibrium. The simulated column has the same surface conditions as the reference state and is initialized with profiles from the reference state.

Biobanking of Fresh-Frozen Human Adenocarcinomatous and Normal Colon Tissues: Which Parameters Influence RNA Quality?

Medical research projects become increasingly dependent on biobanked tissue of high quality because the reliability of gene expression is affected by the quality of extracted RNA. Hence, the present study aimed to determine if clinical, surgical, histological, and molecular parameters influence RNA quality of normal and tumoral frozen colonic tissues. RNA Quality Index (RQI) was evaluated on 241 adenocarcinomas and 115 matched normal frozen colon tissues collected between October 2006 and December 2012.

Design, Synthesis, and Use of MMP-2 Inhibitor-Conjugated Quantum Dots in Functional Biochemical Assays.

The development of chemically designed matrix metalloprotease (MMP) inhibitors has advanced the understanding of the roles of MMPs in different diseases. Most MMP probes designed are fluorogenic substrates, often suffering from photo- and chemical instability and providing a fluorescence signal of moderate intensity, which is difficult to detect and analyze when dealing with crude biological samples. Here, an inhibitor that inhibits MMP-2 more selectively than Galardin has been synthesized and used for enzyme labeling and detection of the MMP-2 activity.

[Analysis of hospital outpatient care of a rare disease: cystic fibrosis].

Introduction: Following the generalization of neonatal screening, the French CF Care Network has become structured around 45 qualified centres, the French CF Society, 2 national expertise centres, the Patient Registry and the National Protocol of CF Care in collaboration with the Vaincre Ia Mucoviscidose patient association. This organization and progress in treatment have resulted in the outpatient follow-up of a growing number of patients. Since 2010, the CF Network representatives have been conducting an assessment of outpatient follow-up to identify difficulties in complying with national and international clinical practice guidelines.

Analytical methods for measuring collagen XIX in human cell cultures, tissue extracts, and biological fluids.

Type XIX collagen is a minor collagen associated with basement membranes in vascular, neuronal, mesenchymal, and epithelial tissues. We demonstrated that the NC1, C-terminal, domain of collagen XIX inhibits the migration capacities of tumor cells and exerts a strong inhibition of tumor growth. Other basement membrane collagens or derived fragments were measured in biological fluids such as blood and urine of patients and appeared to be useful for diagnosis, prognosis, or treatment monitoring.

Pooled analysis of two large randomised phase III inhaled mannitol studies in cystic fibrosis.

Background: To evaluate safety and efficacy of inhaled mannitol treatment in subgroups of a large global CF population.

Methods: Data were pooled from two multicentre, double-blind, randomised, controlled, parallel group phase III studies in which 600 patients inhaled either mannitol (400 mg) or control (mannitol 50 mg) twice a day for 26 weeks.

Results: Both the mean absolute change in FEV(1) (mL) and relative change in FEV(1) by % predicted from baseline for mannitol (400 mg) versus control were statistically significant (73.

Exercise with incorporated expiratory manoeuvres was as effective as breathing techniques for airway clearance in children with cystic fibrosis: a randomised crossover trial.

Question: : Can a session of exercise with incorporated expiratory manoeuvres substitute for a session of breathing techniques for airway clearance in children with cystic fibrosis? Are children with cystic fibrosis as co-operative and satisfied with the exercise regimen as with the breathing techniques?.

Design: Randomised, cross-over trial with concealed allocation and intention-to-treat analysis.

Participants: 34 children with cystic fibrosis in a stable clinical state.

[Late onset Ondine syndrome: literature review on a case report].

Ondine syndrome is the central congenital hypoventilation syndrome (CCHS) caused by the mutation of the PHOX2B gene. In late onset cases, the symptomatology often appears after an acute event (infection, general anesthesia, drug intake), increasing hypoventilation. We report a case of late onset Ondine curse in a 9-year-old girl.

[Cystic fibrosis: centers and care networks organization].

More than 20 years after the gene discovery, without specific treatment, the observed improvement of the cystic fibrosis prognosis appears due to management's organization as well as early diagnosis (neonatal screening) or progress in symptomatic treatment. The CF Centers (CRCM) official recognition was a necessary step before generalization of routine neonatal screening (October, 2002). Actually French CF management relies on three levels of organization: CF centers, regional care networks and French CF Society, in close relationship with patients association (Vaincre la Mucoviscidose).

Long-term inhaled dry powder mannitol in cystic fibrosis: an international randomized study.

Rationale: New treatment strategies are needed to improve airway clearance and reduce the morbidity and the time burden associated with cystic fibrosis (CF).

Objectives: To determine whether long-term treatment with inhaled mannitol, an osmotic agent, improves lung function and morbidity.

Methods: Double-blind, randomized, controlled trial of inhaled mannitol, 400 mg twice a day (n = 192, "treated" group) or 50 mg twice a day (n = 126, "control" group) for 26 weeks, followed by 26 weeks of open-label treatment.

[Intrafamilial transmission of Staphylococcus aureus Panton-Valentine leukocidin responsible for two cases of neonatal necrotizing pneumonia].

Necrotizing Staphylococcus aureus Panton-Valentine leukocidin (SA-PLV+) accounts for less than 1% of community-acquired lung diseases in children and young adults. Neonatal cases are exceptional. We report the observations of two newborn female twins, who were not breastfed, presenting a necrotizing lung disease due to the same strain of SA-PVL+ despite nasal decolonization measures taken.

Impact of practice recommendations on patient follow-up and cystic fibrosis centres' activity in France.

Introduction: The French cystic fibrosis (CF) practice recommendations were published at the end of 2002. They advise each patient to be checked up at least once every 3 months in a reference centre for cystic fibrosis.

Objective: To describe the activity of the four reference centres in the Rhône-Alpes area and the patients' follow-up.

Relative contribution of Prevotella intermedia and Pseudomonas aeruginosa to lung pathology in airways of patients with cystic fibrosis.

Background: Patients with cystic fibrosis (CF) with Pseudomonas aeruginosa lung infections produce endobronchial mucus plugs allowing growth of obligate anaerobes including Prevotella spp. Whether obligate anaerobes contribute to the pathophysiology of CF lung disease is unknown.

Methods: The virulence of Prevotella intermedia and Ps aeruginosa was investigated in vitro and in mice, antibodies against P intermedia in CF sera were assessed and a culture-independent detection method for P intermedia/P nigrescens in CF sputum was tested.