key: cord-1024548-fj7roabz authors: Zemanick, Edith T.; Daines, Cori L.; Dellon, Elisabeth P.; Esthe, Charles R.; BreAnna, Kinghorn; Ong, Thida; Muhlebach, Marianne S. title: HIGHLIGHTS FROM THE 2016 NORTH AMERICAN CYSTIC FIBROSIS CONFERENCE date: 2017-07-11 journal: Pediatric Pulmonology DOI: 10.1002/ppul.23707 sha: 6281a9063f678875fa8f6522f5e84b78f0d414c1 doc_id: 1024548 cord_uid: fj7roabz The 30th annual North American Cystic Fibrosis Conference (NACFC) was held in Orlando, FL, on Oct. 27-29, 2016. Abstracts were published in a supplement to Pediatric Pulmonology.(1) This review summarizes several major topic areas addressed at the conference: the pathophysiology of cystic fibrosis (CF) lung disease,clinical trials, clinical management issues, and quality improvement. We sought to provide an overview of emerging concepts in several areas of CF research and care, rather than a comprehensive review of the conference. Citations from the conference are by first author and abstract number or symposium number, as designated in the supplement. only be addressed via genetic approaches. The most common method for gene therapy has been use of viral vectors, and a study in the CF pig demonstrated the feasibility of using lentivirus or a hybrid piggyback/AAV vector to restore CFTR activity 194) . More recently, CRISPR/Cas9 technology has emerged as an exciting new approach toward targeted gene repair, and this methodology was shown to correct a deep intronic splicing mutant in human tracheal epithelial cells (Sanz et al., 195) . While promising, this approach will likely need to be paired with stem cell technologies to deliver cells with repaired CFTR to the lung (Ghaedi, S12.4), which remains an emerging technology. 3, 6 An alternative approach is to utilize targeted RNA editing to correct mutations after translation, which was shown to restore functional CFTR activity in a PTC mutation 194) , though utility is somewhat limited by potential off target effects. While strategies to improve CFTR function offer the best hope for an eventual cure for CF, even the best drugs to date do not completely eliminate disease. There remains an urgent need for improved therapies to address the airway mucus abnormalities, infection, and inflammation that characterize CF. One promising novel approach lies in use of the sphingosine-1-phosphate lyase inhibitor LX2931, which was shown to reduce airway inflammation in a F508del-CFTR mutant Developing new therapies requires appropriate models and biomarkers to assess therapeutic effects, and several investigators presented recent advances in these tools. One emerging model is the use of specialized culture techniques to grow epithelial cells obtained from patient biopsies into spheroid "organoids" that can swell or shrink in response to CFTR modulators (Berkers et al., S12.3). Assays based on organoids obtained from airway cells were shown to predict responses of rare CFTR mutations to modulators better than total protein or chloride current measures (Cholon et al., 11) , and the viscoelastic properties of mucus from these organoids correlates with the efficacy of CFTR modulators (Mellnick et al., 149). In addition, intestinal organoids were shown to be useful in developing patient specific, high-throughput screening platforms for novel therapies (Vonk et al., 200) . Ideally, new therapeutic strategies would be tested in animal models, but generating a cost-effective animal model that faithfully recapitulates human disease remains elusive. One potentially exciting new approach is using CRISPR/Cas9 technology to generate CFTR deficient rabbits, which appear to exhibit many of the airway and liver phenotypes of clinical disease but are less expensive to maintain than the CF pig or other larger animal models (Xu et al., S12.2). While concern exists for outcomes of the highest risk patients, 16, 17 one center reported no difference in survival between relatively stable patients and "high risk" patients needing substantial respiratory support and bridging therapies (Gray et al., 486). These studies may help transform our approach to referral, listing, and pre-and posttransplant management. While a study of advance care planning (ACP) among US CF centers actually suggests more attention to this issue in recent years than in the past, we know that practices vary from center to center. 18 Basile Mental health received a great deal of attention at NACFC 2016, with many interesting abstracts and presentations in follow up to the international guidelines established by the CFF and the European CF Society. 19 In a symposium session, members of the CFF Mental Health Guidelines Advisory Committee presented an overview of tasks for education and training, consultation and guidance, and research work groups that will continue to move this important work forward (Smith, S11.4). The role of the mental health coordinator was reviewed, with attention to how mental health providers with different backgrounds and training could effectively serve in this role (Kooney, S11.1; Sher and Dvorak, S11.2). Collaborative and other improvement training. The inaugural CFF QI Fair just prior to the NACFC was an electric showcase of a multitude of QI interventions throughout the CF community, including the introduction of the CF Pilot Learning Network (PLN). The PLN is a QI network of an initial 14 CF programs with a common aim to improve CF care delivery and outcomes and build an infrastructure for collaborative learning. The QI Fair also featured Virtual Improvement Program Fundamentals (VIP-F). The VIP-F promises to provide a remote way for all centers to participate in QI learning and to stay informed on the best techniques for performing QI in their centers. The CFFPR and other tools provide the means to measure improvement efforts. The rigor of QI in CF is evolving toward implementation science, the study of mechanisms by which evidencebased health care interventions are adopted and disseminated. Through the interface and collaboration of QI and biomedical research, the CF community remains at the forefront of its mission to deliver the best care to people living with CF. 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