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The combination of lumacaftor and ivacaftor (LUM/IVA) has been reported to induce a mean acute absolute drop of -4.1% predicted forced expiratory volume in 1s (FEV1) after a unique administration in healthy subjects. The aim of the present study was to assess acute FEV1 changes after the first dose of LUM/IVA in CF patients. A total of 32 pediatric patients were included. Respiratory manifestations occurred in only 3 patients (9.4%), but FEV1 consistently decreased (-10.4±4.6%, range: -1.5; -21.8%). FEV1 only partially resumed after salbutamol inhalation. Patients with previously known significant reversible airway obstruction and low FEV1 were more at risk of FEV1 decrease.

The ECFS-CTN Standardisation Committee has undertaken this review of lung clearance index as part of the group's work on evaluation of clinical endpoints with regard to their use in multicentre clinical trials in CF. The aims were 1) to review the literature on reliability, validity and responsiveness of LCI in patients with CF, 2) to gain consensus of the group on feasibility of LCI and 3) to gain consensus on answers to key questions regarding the promotion of LCI to surrogate endpoint status. It was concluded that LCI has an attractive feasibility and clinimetric properties profile and is particularly indicated for multicentre trials in young children with CF and patients with early or mild CF lung disease. This is the first article to collate the literature in this manner and support the use of LCI in clinical trials in CF. © 2013 European Cystic Fibrosis Society. Published by Elsevier B.V.

Objective: CFRD is the most common comorbidity in CF. The recommended treatment of CFRD is Insulin, but other options are also in use. Only few data exist related to other treatment options. Therefore we analyzed the German CFregister for the documented therapy of CFRD. Methods: Data from the German CF-register were used from patients with CFRD in 2010 in a retrospective observational study. Delta FEV1% and delta BMI-Zscore from 2 year before diagnosis of CFRD to year of diagnosis and from year of diagnosis to 2 years after were calculated for each treatment group (Insulin, oral anti-diabetic drugs, no therapy with drugs) and compared using ANOVA-analysis. Results: 798 patients with CFRD were documented in 2010. 51.9%.were female; age (mean±SD) at diagnosis 23.8±9.3 years; CFRD duration 5.8±4.5 years. Treatment: 57.6% Insulin, 9.8% oral anti-diabetic drugs, 2.1% with both and 30.2% without any drug treatment. The mean (±SD) CFRD duration in the non-treated patients was 4.5±4.0 years. ANOVA tests showed no differences between treatment groups regarding changes in FEV1% and BMI-Z-score from 2 years before to the year of diagnosis and delta FEV1 and delta BMI-Z-score and from year of diagnosis to 2 years later. Conclusions: The percentage of patients treated with oral anti-diabetic drugs is in the international published range. We know the weakness of retrospective studies. Nevertheless the numbers are high and the observation time is long. Our data point to the question if insulin is the only successful initial treatment of CFRD in all CF patients. At least a part of patients with CFRD seems to be well treated with other regimes. Supported by German CF Foundation.

Background In 2008, ECFS-CTN selected 6 multisite centres out of the 7 French inter-regions. Objectives The French CF Society (SFM) and the patients’ association setup (2009) a NPRC in order to optimize the contribution to Clinical Research (CR) of the 45 CF centres following 6200 patients recorded in the French CF Registry. Methods Establishment of i. a Steerco including the principal investigator and a clinical research coordinator (CRC) of each inter-region, SFM and patients’ association representatives; ii. a website, http://www.recherchecliniquemuco.fr/ ; iii. the CRC in each inter-region has the following main tasks: – collect/enter data related to patients’ inclusions into a software – run CR protocols within the multisite centres – assist the development of clinical studies – prepare investiqator's responses to CTN requests Results Advices by the Steerco for the setup of CR projects. The Webcast CR data show 31 industrial and 75 academic projects either carried out or in progress since 2010. Intervention of Steerco to setup a Vertex LEAD action for LCI training in France. Regular improvement in ECFS assessments of French multisite CTN centres. Conclusion The NPCR may help meet ECFS-CTN multisite centres challenges by: – giving an overview of CR projects – implementing tools for patient's screening and inclusion – sharing effective experiences of multisite centres – reinforcing the link between centres by greater involvement into a project regarding a priority topic (e.g. antibiotic treatment/LCI/physical activity) – identifying projects that have resulted in a publication and sometimes in a change in care practices – coordinating the ECFS eQUIP project deployment.

Objectives The aim of DIAMUCO study is to describe the natural history of the glucose tolerance (GT) and to identify predictive factors of the changes in GT in cystic fibrosis (CF) patients for a three-year period. Methods We used a cross-sectional study design and included a total of 228 patients, 111 children (between 10 and 18 years) and 117 adults between 2009 and 2011. All patients had an annual screening. Patients were classified as having normal glucose tolerance (NGT), impaired glucose tolerance (IGT), or CF-related diabetes mellitus (CFRD) using the 2-h oral glucose tolerance test (OGTT). Results At baseline, 67.1% (n = 153) patients were classified as NGT, 25.4% (n = 58) as IGT and 7.4% (n = 17) as CFRD. Over half of patients changed at least once of glucose tolerance status during follow-up. The mean decline of FEV1 was 1.54 per year without difference between female and male. A decrease in FEV1 (p TableClinical characteristicsTotalCFRDIGTNTpAge20.1±8.124.3±9.319.3±7.420.0±8.20.1FEV172.4±24.165.0±22.173.8±23.972.6±24.50.4Weight z-score0.5±1.5−0.70±1.24−0.79±1.18−0.37±1.50.1 Conclusion We confirm the variability of glucose tolerance status over time. The impact of glucose tolerance status on respiratory deterioration seems to concern only patients with permanently CFRD status. The early treatment decision must therefore be careful.

Chronic colonization with Pseudomonas aeruginosa (PA) in CF patients shows more rapidly decline in pulmonary function (PF). Objective: To compare the PF and clinical characteristics of CF patients according to the colonization with PA. Methods: Observational cross-sectional study. We collected data from CF patients who were performed at least four sputum cultures from September 2009 to September 2010. We divided them into four groups according to Leeds Criteria: Chronic infection, Intermittent colonization, Free and Never. We considered: age, genetics, pulmonary function, nutritional status, intravenous (IV) antibiotics and PA antibodies. Results: We evaluated 97 CF patients: 26% Chronic, 25% Intermittent, 27% Free and 19 % Never. The current age (median and interquartile range) was 10.7 (8.5 to 16.2), 5.8 (2.78 to 7.67), 5.5 (4.7 to 8.9) and 4.2 (0.97 to 5.3), respectively (p< 0.001). The homozygous p.508 del was present in 34%, 56%, 29%, and 31% of patients, respectively (p =NS). PA antibodies was 7.6 (4.5 to 16.5), 1.6 (1 to 1.8), 1.5 (1to 2.6) and 0.8 (0.6 to 1), respectively (p< 0.001). The Zscore BMI was −0.39 (−1.14 to 0.13), −0.1 (−0.81 to 0.31), 0.19 (−0.28 to 0.83) and 0.11 (−0.94 to 0.37) in each group (p =NS). The FEV1 was 63 (47.5 to 92.2), 79 (54.7 to 108), 99 (85 to 105) and 109 (105.7 to 112), respectively (p = 0.06). (Chronic vs. Free p = 0.007 and Chronic vs. Never p< 0.001). Iv Antibiotic treatments were required in 1 (0−1), 1 (1−2), 0 (0−1) and 0 (0−0), respectively (p< 0.01). Conclusion: Chronic infection with PA determined lower pulmonary function, more iv antibiotics treatments and had higher level of antibodies than the others groups.

Radiological tools are critical for monitoring cystic fibrosis (CF) patients’ thoracic, abdominal, rhinosinusal, and bone systems. Given the likelihood of increasing demand for ionizing techniques for research purposes, we sought to determine how clinicians use radiological tools in their centers. Methods: A 39-item questionnaire was sent to 49 pediatric and adult CF centers in July 2011. Questions were asked on frequency and indications for thoracic, abdominal, rhinosinusal, and bone imaging, as well as the radiological devices available. Results: Thirty-three of 49 (68%) centers answered the questionnaire. Among them, 36% were pediatric centers, 33% mixed, and 31% adult centers. An annual chest x-ray was done routinely in more than 95% of centers, starting at the initial stage of the disease (neonatal screening). While adult clinicians did not routinely perform chest CT, 72% of the pediatricians requested it routinely. Pediatricians declared doing the first chest CT at a mean of 4.9±1.2 years of age, and every 2 or 3 years thereafter. Respectively, 32% and 20% of pediatric and adult centers regularly indicated the cumulative doses received by patients on medical charts. MRI was used in seven out of 33 centers mainly for abdominal indications. Annual chest x-ray is part of routine follow-up for most centers; however, divergent attitudes have emerged regarding chest CT use between pediatric and adult centers. The reasons for the routine use of chest CT in pediatric patients will need further investigations. Furthermore, efforts should be made by clinicians to regularly monitored cumulative doses received by their patients, particularly in the pediatric population.