The utility of glucose area under the curve from the oral glucose tolerance test as a screening tool for cystic fibrosis-related diabetes.

Background: Consistently abnormal glucose levels on oral glucose tolerance test (OGTT) are the most effective screening tool for cystic fibrosis-related diabetes (CFRD). However, some cystic fibrosis (CF) patients demonstrate abnormal glucose profiles not reaching levels required for CFRD diagnosis and are, therefore, left untreated. Since CFRD is associated with disease deterioration, early diagnosis and treatment are desirable.

Respiratory physiotherapy in patients with cystic fibrosis and upper limb deep vein thrombosis.

We report physiotherapy management of two patients with severe cystic fibrosis (CF) lung disease and upper limb deep vein thrombosis (DVT). These patients were admitted due to a pulmonary exacerbation. Following peripherally inserted central catheters, they were diagnosed with an upper limb DVT.

Increasing Vitamin D Serum Levels Is Associated With Reduced Pulmonary Exacerbations in Patients With Cystic Fibrosis.

Context: In 2012, The North American Cystic Fibrosis Foundation (NACFF) published new guidelines for the treatment of vitamin D deficiency in individuals with cystic fibrosis (CF).

Objective: The objectives of our study were to assess the efficacy of these guidelines, and to test the effect of increasing vitamin D dosage on pulmonary function and exacerbations.

Design: Pulmonary function tests and serum concentrations of 25-hydroxyvitamin D [25(OH)D] were measured 1 year before increasing vitamin D dosage according to the guidelines and at least 1 year later.

Eradication failure of newly acquired Pseudomonas aeruginosa isolates in cystic fibrosis.

Unlabelled: Eradication of Pseudomonas aeruginosa (PA) is critical in cystic fibrosis (CF) patients.

Objectives: To determine eradication success rate of newly acquired PA and to identify characteristics associated with eradication failure.

Methods: In an observational study, data from patients with newly acquired PA infection from 2007 to 2013 were collected.

Adherence pattern to study drugs in clinical trials by patients with cystic fibrosis.

Background: Clinical trials are all based on the assumption that patients are adherent to the study protocol. Many reports indicate that general adherence of patients with CF to their daily routine therapies is poor. However, no data exists on adherence to study drug regimens.

CFTR potentiator therapy ameliorates impaired insulin secretion in CF patients with a gating mutation.

Objective: To investigate the effect of treatment with ivacaftor on insulin secretion in patients with cystic fibrosis (CF) (ΔF508\S549R) having CFRD/impaired insulin secretion.

Methods: A standard OGTT was performed before and after 16weeks of treatment with ivacaftor in 2 sibling patients with CF carrying the S549R gating mutation. The area under the curve (AUC) for glucose and insulin was calculated using the trapezoidal estimation.

Evidence of intestinal inflammation in patients with cystic fibrosis.

Objectives: Treatment with pancreatic enzymes fails to completely correct malabsorption and gastrointestinal symptoms in patients with cystic fibrosis (CF). The aim of the present study was to examine the small intestine of patients with CF without overt evidence of gastrointestinal disease using capsule endoscopy (CE).

Methods: Patients with CF received the agile patency capsule and, depending on the result of that procedure, then underwent standard CE using the PillCam SB capsule (Given Imaging, Yokneam, Israel).

18F-fluorodeoxyglucose-PET/CT imaging of lungs in patients with cystic fibrosis.

Background: Airway inflammation plays a critical role in the progression of cystic fibrosis (CF) lung disease, and in the destruction of airways and lung parenchyma. Current methods to assess CF lung disease (BAL, spirometry, and high-resolution CT scanning), do not always accurately reflect actual disease states. Fluorodeoxyglucose (FDG)-PET scanning has been used previously to image infection and inflammation.

Intravenous monthly pulse methylprednisolone treatment for ABPA in patients with cystic fibrosis.

Background: Allergic bronchopulmonary aspergillosis (ABPA) in patients with CF is associated with frequent exacerbations and deterioration of lung function. Oral corticosteroids are standard therapy for ABPA and are associated with severe side effects. Monthly pulses of high-dose intravenous methylprednisolone (HDIVPM) are an effective therapy for autoimmune diseases with fewer side effects compared to oral prednisone, implicating its use for patients with CF who suffer from ABPA.

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.

Background: In about 10% of patients worldwide and more than 50% of patients in Israel, cystic fibrosis results from nonsense mutations (premature stop codons) in the messenger RNA (mRNA) for the cystic fibrosis transmembrane conductance regulator (CFTR). PTC124 is an orally bioavailable small molecule that is designed to induce ribosomes to selectively read through premature stop codons during mRNA translation, to produce functional CFTR.

Methods: This phase II prospective trial recruited adults with cystic fibrosis who had at least one nonsense mutation in the CFTR gene.