Cystic Fibrosis: Recent Successes Present New Challenges
Authors: Joseph M. Pilewski, Darren B. Taichman
Journal: Annals of Internal Medicine (2014)

Editorials19 August 2014Cystic Fibrosis: Recent Successes Present New ChallengesJoseph M. Pilewski, MD and Darren B. Taichman, MD, PhD, Executive Deputy EditorJoseph M. Pilewski, MDFrom University of Pittsburgh, Pittsburgh, Pennsylvania; and American College of Physicians, Philadelphia, Pennsylvania.Search for more papers by this author and Darren B. Taichman, MD, PhD, Executive Deputy EditorFrom University of Pittsburgh, Pittsburgh, Pennsylvania; and American College of Physicians, Philadelphia, Pennsylvania.Search for more papers by this authorAuthor, Article, and Disclosure Informationhttps://doi.org/10.7326/M14-1534 SectionsAboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail Remarkable progress has been made in the care and survival of patients with cystic fibrosis (CF). First described in 1938 as a life-shortening disease characterized by cystic changes of the pancreas, CF is now known to be caused by any of many known mutations in a single gene, resulting in abnormal function of a chloride channel and multisystem disease (1). Before the development of pancreatic enzymes and nutritional supplements, most patients with CF died early from malnutrition related to pancreatic insufficiency. After this early threat became more controllable, progressive pulmonary disease characterized by chronic airway infection, inflammation, bronchiectasis, and respiratory ...References1. O'Sullivan BP, Freedman SD. Cystic fibrosis. Lancet. 2009;373:1891-904. [PMID: 19403164] doi:10.1016/S0140-6736(09)60327-5 CrossrefMedlineGoogle Scholar2. MacKenzie T, Gifford AH, Sabadosa KA, Quinton HB, Knapp EA, Goss CH, et al. Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the Cystic Fibrosis Foundation Patient Registry. Ann Intern Med. 2014;161:233-41. doi:10.7326/M13-0636 LinkGoogle Scholar3. Ramsey BW, Pepe MS, Quan JM, Otto KL, Montgomery AB, Williams-Warren J, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. N Engl J Med. 1999;340:23-30. [PMID: 9878641] CrossrefMedlineGoogle Scholar4. Fuchs HJ, Borowitz DS, Christiansen DH, Morris EM, Nash ML, Ramsey BW, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group. N Engl J Med. 1994;331:637-42. [PMID: 7503821] CrossrefMedlineGoogle Scholar5. Elkins MR, Robinson M, Rose BR, Harbour C, Moriarty CP, Marks GB, et al; National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med. 2006;354:229-40. [PMID: 16421364] CrossrefMedlineGoogle Scholar6. Donaldson SH, Bennett WD, Zeman KL, Knowles MR, Tarran R, Boucher RC. Mucus clearance and lung function in cystic fibrosis with hypertonic saline. N Engl J Med. 2006;354:241-50. [PMID: 16421365] CrossrefMedlineGoogle Scholar7. Treggiari MM, Retsch-Bogart G, Mayer-Hamblett N, Khan U, Kulich M, Kronmal R, et al; Early Pseudomonas Infection Control (EPIC) Investigators. Comparative efficacy and safety of 4 randomized regimens to treat early Pseudomonas aeruginosa infection in children with cystic fibrosis. Arch Pediatr Adolesc Med. 2011;165:847-56. [PMID: 21893650] doi:10.1001/archpediatrics.2011.136 CrossrefMedlineGoogle Scholar8. Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevínek P, et al; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365:1663-72. [PMID: 22047557] doi:10.1056/NEJMoa1105185 CrossrefMedlineGoogle Scholar9. Jackson GL, Powers BJ, Chatterjee R, Bettger JP, Kemper AR, Hasselblad V, et al. The patient centered medical home.A systematic review. Ann Intern Med. 2013;158:169-78. [PMID: 24779044] doi:10.7326/0003-4819-158-3-201302050-00579 LinkGoogle Scholar10. Warm EJ, Goetz C. Too smart for primary care? Ann Intern Med. 2013;159:709-10. [PMID: 24247675] doi:10.7326/0003-4819-159-10-201311190-00009 LinkGoogle Scholar Author, Article, and Disclosure InformationAffiliations: From University of Pittsburgh, Pittsburgh, Pennsylvania; and American College of Physicians, Philadelphia, Pennsylvania.Disclosures: Disclosures can be viewed at www.acponline.org/authors/icmje/ConflictOfInterestForms.do?msNum=M14-1534.Corresponding Author: Joseph M. Pilewski, MD, University of Pittsburgh, Department of Medicine, PACCM Division, NW 628 MUH, 3459 Fifth Avenue, Pittsburgh, PA 15213.Current Author Addresses: Dr. Pilewski: University of Pittsburgh, Department of Medicine, PACCM Division, NW 628 MUH, 3459 Fifth Avenue, Pittsburgh, PA 15213.Dr. Taichman: American College of Physicians, 190 N. Independence Mall West, Philadelphia, PA 19106. PreviousarticleNextarticle Advertisement FiguresReferencesRelatedDetailsSee AlsoLongevity of Patients With Cystic Fibrosis in 2000 to 2010 and Beyond: Survival Analysis of the Cystic Fibrosis Foundation Patient Registry Todd MacKenzie , Alex H. Gifford , Kathryn A. Sabadosa , Hebe B. Quinton , Emily A. Knapp , Christopher H. Goss , and Bruce C. Marshall Longevity of Patients With Cystic Fibrosis in 2000 to 2010 and Beyond: Survival Analysis of the Cystic Fibrosis Foundation Patient Registry Todd MacKenzie , Alex H. Gifford , Kathryn A. Sabadosa , Hebe B. Quinton , Emily A. Knapp , Christopher H. Goss , and Bruce C. Marshall Metrics Cited byPerspectives in Pediatric Pathology, Chapter 22. Testicular Involvement in Systemic Diseases 19 August 2014Volume 161, Issue 4Page: 298-299KeywordsChildrenCystic fibrosisDisclosureHealth careLungsMorbidityMortalityMucusNutritionOutpatients ePublished: 19 August 2014 Issue Published: 19 August 2014 Copyright & PermissionsCopyright © 2014 by American College of Physicians. All Rights Reserved.PDF downloadLoading ...