Джерело: Drucy Borowitz et al. Cystic Fibrosis Foundation Evidence-Based Guidelines for Management of Infants with Cystic Fibrosis J Pediatr 2009;155:S73-93
References
1. Robinson KA, Saldanha I, McKoy N. Management of infants with cystic
fibrosis: A summary of the evidence for the Cystic Fibrosis Foundation
Working Group on Care of Infants with Cystic Fibrosis. J Pediatr
(Insert proper citation for the submitted methodology paper in this
supplement).
2. Sawaya GF, Guirguis-Blake J, LeFevre M, Harris R, Petitti D. Update
on the methods of the US Preventive Services Task Force: estimating
certainty and magnitude of net benefit. Ann Intern Med 2007;147:
871-5.
3. Giglio L, Candusso M, D’Orazio C, Mastella G, Faraguna D. Failure to
thrive: the earliest feature of cystic fibrosis in infants diagnosed by neonatal
screening. Acta Paediatr 1997;86:1162-5.
4. Accurso FJ, Sontag MK, Wagener JS. Complications associated with
symptomatic diagnosis in infants with cystic fibrosis. J Pediatr 2005;
147(suppl):S37-41.
5. Jedlicka-Kohler I, Gotz M, Eichler I. Parents’ recollection of the initial
communication of the diagnosis of cystic fibrosis. Pediatrics 1996;97:
204-9.
6. Tluczek A,Koscik RL,Modaff P, Pfeil D, Rock MJ, FarrellPM, et al. Newborn screening for cystic fibrosis: parents’ preferences regarding counseling at the time of infants’ sweat test. J Genet Couns 2006;15:277-91.
7. Sims EJ, Mugford M, Clark A, Aitken D, McCormick J, Mehta G, et al.
Economic implications of newborn screening for cystic fibrosis: a cost
of illness retrospective cohort study. Lancet 2007;369:1187-95.
8. 9Stallings VA, Stark LJ, Robinson KA, Feranchak AP, Quinton H. Evidence-
based practice recommendations for nutrition-relatedmanagement
of children and adults with cystic fibrosis and pancreatic insufficiency: results
of a systematic review. J Am Diet Assoc 2008;108:832-9.
9. Konstan MW, Butler SM, Wohl ME, Stoddard M, Matousek R,
Wagener JS, et al. Growth and nutritional indexes in early life predict
pulmonary function in cystic fibrosis. J Pediatr 2003;142:624-30.
10. Feranchak AP, Sontag MK, Wagener JS, Hammond KB, Accurso FJ,
Sokol RJ. Prospective, long-term study of fat-soluble vitamin status
in children with cystic fibrosis identified by newborn screen. J Pediatr
1999;135:601-10.
11. Shepherd RW, Holt TL, Greer R, Cleghorn GJ, Thomas BJ. Total body
potassium in cystic fibrosis. J Pediatr Gastroenterol Nutr 1989;9:200-5.
12. Borowitz D. Update on the evaluation of pancreatic exocrine status in
cystic fibrosis. Curr Opin Pulm Med 2005;11:524-7.
13. Bronstein MN, Sokol RJ, Abman SH, Chatfield BA, Hammond KB,
Hambridge KM, et al. Pancreatic insufficiency, growth, and nutrition
in infants identified by newborn screening as having cystic fibrosis.
J Pediatr 1992;120:533-40.
14. Walkowiak J, Sands D, Nowakowska A, Piotrowski R, Zybert K,
Herzig KH, et al. Early decline of pancreatic function in cystic fibrosis
patients with class 1 or 2 CFTR mutations. J Pediatr Gastroenterol Nutr
2005;40:199-201.
15. Gaskin K, Waters D, Dorney S, Gruca M, O’Halloran M, Wilcken B. Assessment
of pancreatic function in screened infants with cystic fibrosis.
Pediatr Pulmonol Suppl 1991;7:69-71.
16. Bellentani S, Grisendi A, Rinaldi M, Bertolani P, Costa G, Agostini M,
et al. BT-Paba test in the diagnosis of pancreatic exocrine insufficiency
in cystic fibrosis: urinary and serum determinations compared. Eur J
Pediatr 1984;143:145-8.
17. Greer R, Shepherd R, Cleghorn G, Bowling FG, Holt T. Evaluation of
growth and changes in body composition following neonatal diagnosis
of cystic fibrosis. J Pediatr Gastroenterol Nutr 1991;13:52-8.
18. Durie PR, Largman C, Brodrick JW, Johnson JH, Gaskin KJ,
Forstner GG, et al. Plasma immunoreactive pancreatic cationic trypsinogen
in cystic fibrosis: a sensitive indicator of exocrine pancreatic dysfunction.
Pediatr Res 1981;15:1351-5.
19. Gillard BK, Cox KL, Pollack PA, Geffner ME. Cystic fibrosis serum pancreatic
amylase: useful discriminator of exocrine function. Am J Dis
Child 1984;138:577-80.
20. Watkins JB, Schoeller DA, Klein PD, Ott DG, Newcomer AD,
Hofmann AF. 13C-trioctanoin: a nonradioactive breath test to detect
fat malabsorption. J Lab Clin Med 1977;90:422-30.
21. Colombo C, Maiavacca R, Ronchi M, Consalvo E, Amoretti M,
Giunta A. The steatocrit: a simple method for monitoring fat malabsorption
in patients with cystic fibrosis. J Pediatr Gastroenterol Nutr
1987;6:926-30.
22. Remtulla MA, Durie PR, Goldberg DM. Stool chymotrypsin activity
measured by a spectrophotometric procedure to identify pancreatic
disease in infants. Clin Biochem 1986;19:341-7.
23. Beharry S, Ellis L, Corey M, Marcon M, Durie P. How useful is fecal
pancreatic elastase 1 as a marker of exocrine pancreatic disease? J Pediatr
2002;141:84-90.
Джерело: Peter J. Mogayzel et al. Cystic Fibrosis Pulmonary Guidelines Chronic Medications for Maintenance of Lung Health Am J Respir Crit Care Med Vol 187, Iss. 7, pp 680–689, Apr 1, 2013
1. Flume PA, O’Sullivan BP, Robinson KA, Goss CH, Mogayzel PJ Jr,
Willey-Courand DB, Bujan J, Finder J, Lester M, Quittell L, et al.
Cystic fibrosis pulmonary guidelines: chronic medications for maintenance
of lung health. Am J Respir Crit Care Med 2007;176:957–969.
2. Sawaya GF, Guirguis-Blake J, LeFevre M, Harris R, Petitti D. Update on
the methods of the US Preventive Services Task Force: estimating certainty
and magnitude of net benefit. Ann Intern Med 2007;147:871–875.
3. Halfhide C, Evans HJ, Couriel J. Inhaled bronchodilators for cystic fibrosis.
Cochrane Database Syst Rev 2005;CD003428.
4. König P, Poehler J, Barbero GJ. A placebo-controlled, double-blind trial
of the long-term effects of albuterol administration in patients with
cystic fibrosis. Pediatr Pulmonol 1998;25:32–36.
5. Eggleston PA, Rosenstein BJ, Stackhouse CM, Mellits ED, Baumgardner
RA. A controlled trial of long-term bronchodilator therapy in cystic
fibrosis. Chest 1991;99:1088–1092.
6. Holzer FJ, Olinsky A, Phelan PD. Variability of airways hyper-reactivity
and allergy in cystic fibrosis. Arch Dis Child 1981;56:455–459.
7. Konstan MW, Byard PJ, Hoppel CL, Davis PB. Effect of high-dose
ibuprofen in patients with cystic fibrosis. N Engl J Med 1995;332:
848–854.
8. Konstan MW, Hoppel CL, Chai BL, Davis PB. Ibuprofen in children
with cystic fibrosis: pharmacokinetics and adverse effects. J Pediatr
1991;118:956–964.
9. Sordelli DO, Macri CN, Maillie AJ, Cerquetti MC. A preliminary study
on the effect of anti-inflammatory treatment in cystic fibrosis patients
with Pseudomonas aeruginosa lung infection. Int J Immunopathol
Pharmacol 1994;7:109–117.
10. Lands LC, Milner R, Cantin AM, Manson D, Corey M. High-dose
ibuprofen in cystic fibrosis: Canadian safety and effectiveness trial.
J Pediatr 2007;151:249–254.
686 AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE VOL 187 2013
11. Lands LC, Stanojevic S. Oral non-steroidal anti-inflammatory drug therapy
for cystic fibrosis. Cochrane Database Syst Rev 2007;CD001505.
12. Konstan MW, Krenicky JE, Finney MR, Kirchner HL, Hilliard KA,
Hilliard JB, Davis PB, Hoppel CL. Effect of ibuprofen on neutrophil
migration in vivo in cystic fibrosis and healthy subjects. J Pharmacol
Exp Ther 2003;306:1086–1091.
13. Clement A, Tamalet A, Leroux E, Ravilly S, Fauroux B, Jais JP. Long
term effects of azithromycin in patients with cystic fibrosis: a double
blind, placebo controlled trial. Thorax 2006;61:895–902.
14. Rotschild M, Elias N, Berkowitz D, Pollak S, ShinawiM, Beck R, Bentur
L. Autoantibodies against bactericidal/permeability–increasing protein
(BPI-ANCA) in cystic fibrosis patients treated with azithromycin.
Clin Exp Med 2005;5:80–85.
15. Saiman L, Marshall BC, Mayer-Hamblett N, Burns JL, Quittner AL,
Cibene DA, Coquillette S, Fieberg AY, Accurso FJ, Campbell PW
III. Azithromycin in patients with cystic fibrosis chronically infected
with Pseudomonas aeruginosa: a randomized controlled trial. JAMA
2003;290:1749–1756.
16. Wolter J, Seeney S, Bell S, Bowler S, Masel P, McCormack J. Effect of
long term treatment with azithromycin on disease parameters in cystic
fibrosis: a randomised trial. Thorax 2002;57:212–216.
17. Saiman L, Anstead M, Mayer-Hamblett N, Lands LC, Kloster M,
Hocevar-Trnka J, Goss CH, Rose LM, Burns JL, Marshall BC, et al.
Effect of azithromycin on pulmonary function in patients with cystic
fibrosis uninfected with Pseudomonas aeruginosa: a randomized controlled
trial. JAMA 2010;303:1707–1715.
18. Equi A, Balfour-Lynn IM, Bush A, Rosenthal M. Long term azithromycin
in children with cystic fibrosis: A randomised, placebocontrolled
crossover trial. Lancet 2002;360:978–984.
19. Southern K, Barker P, Solis-Moya A, Patel L. Macrolide antibiotics for
cystic fibrosis. Cochrane Database Syst Rev 2004;CD002203.
20. Loening-Baucke VA, Mischler E, Myers MG. A placebo-controlled trial
of cephalexin therapy in the ambulatory management of patients with
cystic fibrosis. J Pediatr 1979;95:630–637.
21. Stutman HR, Lieberman JM, Nussbaum E, Marks MI. Antibiotic prophylaxis
in infants and young children with cystic fibrosis: a randomized
controlled trial. J Pediatr 2002;140:299–305.
22. Smyth A, Walters S. Prophylactic antibiotics for cystic fibrosis. Cochrane
Database Syst Rev 2003;CD001912.
23. Yu H, Burton B, Huang CJ, Worley J, Cao D, Johnson JP Jr, Urrutia A,
Joubran J, Seepersaud S, Sussky K, et al. Ivacaftor potentiation of
multiple CFTR channels with gating mutations. J Cyst Fibros 2012;11:
237–245.
24. Yang Y, Devor DC, Engelhardt JF, Ernst SA, Strong TV, Collins FS,
Cohn JA, Frizzell RA, Wilson JM. Molecular basis of defective anion
transport in L cells expressing recombinant forms of CFTR. Hum Mol
Genet 1993;2:1253–1261.
25. Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel
SD, Hornick DB, Konstan MW, Donaldson SH, et al. Effect ofVX-770
in persons with cystic fibrosis and the G551D-CFTR mutation.NEngl J
Med 2010;363:1991–2003.
26. Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P,
Griese M, McKone EF, Wainwright CE, Konstan MW, et al. A CFTR
potentiator in patients with cystic fibrosis and the G551D mutation.
N Engl J Med 2011;365:1663–1672.
27. Aherns RS, Yen K, Davies JC. VX-770 in subjects 6 to 11 years with
cystic fibrosis and the G551D-CFTR mutation [abstract]. Pediatr
Pulmonol 2011;46 (S34):283.
28. Flume PA, Liou TG, Borowitz DS, Li H, Yen K, Ordonez CL, Geller
DE. Ivacaftor in subjects with cystic fibrosis who are homozygous for
the F508del-CFTR mutation. Chest 2012;142:718–724.
29. Emerson J, Rosenfeld M, McNamara S, Ramsey B, Gibson RL. Pseudomonas
aeruginosa and other predictors of mortality and morbidity in
young children with cystic fibrosis. Pediatr Pulmonol 2002;34:91–100.
30. McCoy KS, Quittner AL, Oermann CM, Gibson RL, Retsch-Bogart GZ,
Montgomery AB. Inhaled aztreonam lysine for chronic airway Pseudomonas
aeruginosa in cystic fibrosis. Am J Respir Crit Care Med 2008;
178:921–928.
Джерело: Alan R. Smith et. al. European Cystic Fibrosis Society Standards of Care: Best Practice guidelines Journal of Cystic Fibrosis 13 (2014) S23-S42
[1] DiSant'Agnese PA. Cystic fibrosis of the pancreas. Am J Med
1956;21:406–22.
[2] Shwachman H, Kulczycki LL. Long-term study of one hundred five
patients with cystic fibrosis; studies made over a five- to fourteen-year
period. AMA J Dis Child 1958;96:6–15.
[3] Doershuk CF, Matthews LW, Tucker AS, Nudleman H, Eddy G, Wise
M, et al. A 5 year clinical evaluation of a therapeutic program for
patients with cystic fibrosis. J Pediatr 1964;65:677–93.
[4] Warwick WJ, Pogue RE. The prognosis for children with cystic
fibrosis based on reasoned approaches to therapy: past, present, and
future. J Asthma Res 1968;5:277–84.
[5] Shwachman H, Kowalski M, Khaw KT. Cystic fibrosis: a new
outlook. 70 patients above 25 years of age. Medicine 1977;56:129–49.
[6] Crozier DN. Cystic fibrosis: a not-so-fatal disease. Pediatr Clin North
Am 1974;21:935–50.
[7] Gurwitz D, Corey M, Francis PW, Crozier D, Levison H. Perspectives
in cystic fibrosis. Pediatr Clin North Am 1979;26:603–15.
[8] Corey M, McLaughlin FJ, Williams M, Levison H. A comparison of
survival, growth, and pulmonary function in patients with cystic
fibrosis in Boston and Toronto. J Clin Epidemiol 1988;41:583–91.
[9] Cystic Fibrosis Foundation. Cystic Fibrosis Foundation Patient
Registry 2011 annual data report; 2012 [Bethesda, Maryland].
[10] Cystic Fibrosis Canada. Canadian Cystic Fibrosis Patient Data
Registry report. 2010; 2011 [Toronto].
[11] Cystic Fibrosis Foundation. http://www.cff.org/LivingWithCF/Care
CenterNetwork/QualityImprovement/; 2013.
[12] Salvatore D, Buzzetti R, Baldo E, Furnari ML, Lucidi V, Manunza D,
et al. An overview of international literature from cystic fibrosis
registries. Part 4: update 2011. J Cyst Fibros 2012;11:480–93.
[13] European cystic fibrosis society patient registry newsletter.
http://www.ecfs.eu/projects/ecfspr/newsletters; January 2013 . [Issue
3].
[14] The ECFS Patient Registry annual data report. 2008–2009 data.
version 03. Available from:
https://www.ecfs.eu/files/webfm/webfiles/File/ecfs_
registry/ECFSPR_Report0809_v32012.pdf; 2012.
[15] Kerem E, Viviani L, Zolin A, Macneill S, Hatziagorou E, Ellemunter
H, et al. Factors associated with FEV1 decline in cystic fibrosis:
analysis of the data of the ECFS Patient Registry. Eur Respir J 2013.
http:// dx.doi.org/10.1183/09031936.00166412 [Epub ahead of print].
[16] Wiedemann B, Steinkamp G, Sens B, Stern M. The German Cystic
Fibrosis Quality Assurance Project: clinical features in children and
adults. Eur Respir J Eur Respir J 2001;17:1187–94.
M. Stern et al. / Journal of Cystic Fibrosis 13 (2014) S43–S59 S59
[17] Gutierrez HH, Sanchez I, Schidlow DV. Cystic fibrosis care in Chile.
Curr Opin Pulm Med 2009;15:632–7.
[18] Morgan WJ, Butler SM, Johnson CA, Colin AA, FitzSimmons SC,
Geller DE, et al. Epidemiologic study of cystic fibrosis: design and
implemen- tation of a prospective, multicentre, observational study of
patients with cystic fibrosis in the U.S. and Canada. Pediatr Pumonol
1999;28:231–41.
[19] Schechter MS. Benchmarking to improve the quality of cystic fibrosis
care. Curr Opin Pulm Med 2012;18:596–601.
[20] Rosenfeld M, Emerson J, McNamara S, Joubran K, Retsch-Bogart G,
Graff GR, et al. Baseline characteristics and factors associated with
nutritional and pulmonary status at enrollment in the cystic fibrosis
EPIC observational cohort. Pediatr Pulmonol 2010;45:934–44.
[21] Byrnes CA, Vidmar S, Cheney JL, Carlin JB, Armstrong DS, Cooper
PJ, et al. Prospective evaluation of respiratory exacerbations in
children with cystic fibrosis from newborn screening to 5 years of age.
Thorax 2013;68:643–51.
[22] Martin B, Schechter MS, Jaffe A, Cooper P, Bell SC, Ranganathan S.
Comparison of the US and Australian cystic fibrosis registries: the
impact of newborn screening. Pediatrics 2012;129:48–55.
[23] Closing the quality gap: revisiting the state of the science series:
quality improvement interventions to address health disparities.
Review protocol. Rockville, MD: Agency for Healthcare Research
and Quality; 2011 [http://www.ahrq.gov/clinic/tp/gapdisptp.htm].
[24] Berwick DM. The science of improvement. JAMA 2008;299:1182–4.
[25] Shojania KG, Grimshaw JM. Evidence-based quality improvement:
the state of the science. Health Aff (Millwood) 2005;24:138–50.
[26] Berwick DM. Continuous improvement as an ideal in health care. N
Engl J Med 1989;320:53–6.
[27] Nelson EC, Godfrey MM, Batalden PB, Berry SA, Bothe AE,
McKinley KE, et al. Clinical microsystems, Part1: The building
blocks of health systems. Jt Comm J Qual Patient Saf 2008;34:367–
78.
[28] Schechter MS, Margolis P. Improving subspecialty healthcare: lessons
from cystic fibrosis. J Pediatr 2005;147:295–301.
[29] Quinton HB, O'Connor GT, for the Northern New England CV Cystic
Fibrosis Consortium. Current issues in quality improvement in cystic
fibrosis. Clin Chest Med 2007;28:459–72.
[30] Stern M, Wiedemann B, Wenzlaff P, on behalf of the German Cystic
Fibrosis Quality Assessment group. From registry to quality management:
the German Cystic Fibrosis Quality Assessment project 1995–
2006. Eur Respir J 2008;31:29–35.
[31] Stern M. The use of a cystic fibrosis patient registry to assess
outcomes and improve cystic fibrosis care in Germany. Curr Opin
Pulm Med 2011;17:477–573.
[32] Ellemunter H, Eder J, Steinkamp G. Structured care in an ISO
certified Centre for patients with cystic fibrosis and their families.
Pneumologie 2011;65:615–23.
[33] Liou TG, Elkin EP, Pasta DJ, Jacobs JR, Konstan MW, Morgan WJ,
et al. Year-to-year changes in lung function in individuals with cystic
fibrosis. J Cyst Fibros 2010;9:250–6.
[34] Taylor-Robinson D, Whitehead M, Diderichsen F, Vebert Olesen H,
Pressler T, Smyth RL, et al. Understanding the natural progression in
%FEV1 decline in patients with cystic fibrosis: a longitudinal study.
Thorax 2012;67:860–6.
[35] Pillarisetti N, Williamson E, Linnane B, Skoric B, Robertson CF,
Robinson P, et al. Infection, inflammation and lung function decline in
infants with cystic fibrosis. Am J Respir Crit Care Med 2011;184:75–
81.
[36] Kraynack NC, McBride JT. Improving care at cystic fibrosis centers
through quality improvement. Semin Respir Crit Care Med
2009;30:547–58.
[37] Zemanick ET, Harris JK, Conway S, Konstan MW, Marshall B,
Quittner AL, et al. Measuring and improving respiratory outcomes in
cystic fibrosis lung disease: opportunities and challenges to therapy. J
Cyst Fibros 2010;9:1–16.
[38] Glauser TA, Nevins PH, Williamson JC, Abdolrasulnia M, Salinas
GD, Zhang J, et al. Adherence to the 2007 cystic fibrosis pulmonary
guidelines: a national survey of CF care centers. Pediatr Pulmonol
2012;47:434–40.
[39] McPhail GL, Weiland J, Acton JD, Ednick M, Chima A, VanDyke R,
et al. Improving evidence-based care in cystic fibrosis through quality
improvement. Arch Pediatr Adolesc Med 2010;164:957–60.
[40] Stallings VA, Stark LJ, Robinson KA, Feranchak AP, Quinton H.
Evidence-based practice recommendations for nutrition-related
manage- ment of children and adults with cystic fibrosis and
pancreatic insufficiency: results of a systematic review. J Am Diet
Assoc 2008;108:832–9.
[41] Moen IE, Nilsson K, Andersson A, Fagerland MW, Fluge G, Hollsing
A, et al. Dietary intake and nutritional status in a Scandinavian adult
cystic fibrosis population compared with recommendations. Food Nutr
Res 2011;55:7561.
[42] Leonard A, Davis E, Rosenstein BJ, Zeitlin PL, Paranjape SM, Peeler
D, et al. Description of a standardized nutrition classification plan and
its relation to nutritional outcomes in children with cystic fibrosis. J
Pediatr Psychol 2010;35:6–13.
[43] Stark LJ, Opipari-Arrigan L, Quittner AL, Bean J, Powers SW. The
effects of an intensive behavior and nutrition intervention compared to
standard of care on weight outcomes in CF. Pediatr Pulmonol
2011;46:31–5.
[44] Davis PB. Cystic fibrosis since 1938. Am J Respir Crit Care Med
2006;173:475–82.
[45] CF Europe. http://www.cf-europe.eu/.
[46] Cystic Fibrosis Foundation Patient Registry. 2011 Annual Data
Report. Bethesda, Maryland: Cystic Fibrosis Foundation; 2011
[http://www.cff.
org/research/ClinicalResearch/PatientRegistryReport].
[47] Goss HG, Quittner AL. Patient-reported outcomes in cystic fibrosis.
Proc Am Thorac Soc 2007;4:378–86.
[48] Mayer-Hamblett N, Ramsey BW, Kronmal AK. Advancing outcome
measures for the new era of drug development in cystic fibrosis. Proc
Am Thorac Soc 2007;4:370–7.
[49] European Medicines Agency. Committee for medicinal products for
Human Use (CHMP). Guidelines on the Clinical Development of
Medicinal Products for the Treatment of Cystic Fibrosis; Oct. 22 2009
[London, http://www.ema.europa.eu/docs/en_GB/document_library/
Scientific_guideline/2009/12/WC500017055.pdf].
[50] Kerem E, Conway S, Elborn S, Heijerman H, for the Consensus
Committee. Standards of care for patients with cystic fibrosis: a
European consensus. J Cyst Fibros 2005;4(7–26):20.
[51] Steinkamp G, Schwarz C, Stahl K, Busche MCF. Care through the
patient's eyes — a nationwide survey on patients' experience and
satisfaction with services using a CF-specific questionnaire. Pediatr
Pulmonol 2012;47(S35):404 (abstract 492).
[52] Quon BS, Goss CH. A story of success: continuous quality
improvement in cystic fibrosis care in the USA. Thorax
2011;66:1106–8.
[53] http://www.cftrust.org.uk/aboutus/annualreview/CF_Trust_Annual_Review_
2011_%28web%29.pdf.
[54] https://www.cysticfibrosis.org.uk/media/82070/CD_Standards_of_Care_
Dec_11.pdf.
[55] http://www.cff.org/treatments/CFCareGuidelines/AgeSpecificCare/.
[56] Batalden PB, Davidoff F. What is “quality improvement” and how can
it transform healthcare? Qual Saf Health Care 2007;16:2–3.
[57] Sermet-Gaudelus I, Mayell SJ, Southern KW. Guidelines on the early
management of infants diagnosed with cystic fibrosis following
newborn screening. J Cyst Fibros 2010;9:323–9.
[58] Mehta G, Macek Jr M, Mehta A, European RegistryWorking Group.
Cystic fibrosis across Europe: EuroCareCF analysis of demographic
data from 35 countries. J Cyst Fibros 2010;9(Suppl. 2):5–21.
[59] World Health Organization. Guidance on developing quality and
safety strategies with a health system approach. Copenhagen: World
Health Organization Regional Office for Europe; 2008.
[60] Stern M, Niemann N, Wiedemann B, Wenzlaff P, German CFQA
Group. Benchmarking improves quality in cystic fibrosis care: a pilot
project involving 12 Centres. Int J Qual Health Care 2011;23:349–56.
[61] Ayers LR, Beyea SC, Godfrey MM, Harper DC, Nelson EC, Batalden
PB. Quality improvement learning collaboratives. Qual Manag Health
Care 2005;14:234–47.
[62] Rault G, Pougheon-Bertrand D, Gueganton L, Minguet G, Lombrail P.
CF quality improvement program: a pilot phase to experiment the US
QIP approach in France. Poster; ECFS Conference, Dublin, Ireland;
June 6–9 2012.
S60 M. Stern et al. / Journal of Cystic Fibrosis 13 (2014) S43–S59
[63] Schechter MS, Leonard A, Nash J, Quinton H, Richards K, Sabadosa
K, et al. Benchmarking: signature themes. Pediatr Pulmonol
2006(Suppl. 29):122–3.
[64] Homchick RG, Hayward LR. Chapter 16. Peer review and quality
assurance requirements. In: Tremaine DW, JD Organization, editors.
3rd ed. Washington Health Law Manual; 2009.
[65] World Health Organization. Quality of care: a process for making
strategic choices in health systems. Geneva: World Health
Organization; 2006.
[66] Nelson EC, Batalden PB, Godfrey M. Measurement and monitoring.
Quality by design: a clinical microsystems approach. Hoboken, NJ:
John Wiley & Sons; 2007.
[67] McIntyre K, Shojania KG. The challenges of quality improvements
reports and the urgent need for more of them. Thorax 2011;66:1020-2.
[68] Shaw CD, Kalo I. A background for national quality policies in health
systems. Copenhagen: World Health Organization Regional Office for
Europe; 2002.
[69] Kulich M, Rosenfeld M, Goss CH, Wilmott R. Improved survival
among young patients with cystic fibrosis. J Pediatr 2003;142:631–6.
[70] Mayer-Hamblett N, Rosenfeld M, Emerson J, Goss CH, Aitken ML.
Developing cystic fibrosis lung transplant referral criteria using
predictors of two year mortality. Am J Respir Crit Care Med
2002;166:1550–5.
[71] Dasenbrook EC, Checkley W, Merlo CA, Konstan MW, Lechtzin N,
Boyle MP. Association between respiratory tract methicillin-resistant
Staphylococcus aureus and survival in cystic fibrosis. JAMA
2010;303:2386–92.
[72] Quon BS, Mayer-Hamblett N, Aitken ML, Smyth AR, Goss CH. Risk
factors for chronic kidney disease in adults with cystic fibrosis. Am J
Respir Crit Care Med 2011;184:1147–52.
[73] Smyth A, Lewis S, Bertenshaw C, Choonara I, McGaw J, Watson A.
Case–control study of acute renal failure in patients with cystic
fibrosis in the UK. Thorax 2008;63:532–5.
[74] Chamnan P, Shine BS, Haworth CS, Bilton D, Adler AI. Diabetes as a
determinant of mortality in cystic fibrosis. Diabetes Care
2010;33:311–6.
[75] Johnson C, Butler SM, Konstan MW, Morgan W, Wohl ME. Factors
influencing outcomes in cystic fibrosis: a center-based analysis. Chest
2003;123:20–7.
[76] Döring G, Flume P, Heijerman H, Elborn JS. Treatment of lung
infection in patients with cystic fibrosis: current and future strategies.
J Cyst Fibros 2012;11:461–79.
[77] Schechter MS, Gutierrez HH. Improving the quality of care for
patients with cystic fibrosis. Curr Opin Pediatr 2010;22:296–301.
[78] Kraynack NC, Gothard MD, Falletta LM, McBride JT. Approach to
treating cystic fibrosis pulmonary exacerbations varies widely across
US CF care centers. Pediatr Pulmonol 2011;46:870–81.
[79] Mott LS, Park J, Murray CP, Gangell CL, de Klerk NH, Robinson PJ,
et al. Progression of early structural lung disease in young children
with cystic fibrosis assessed using CT. Thorax 2012;67:509–16.
[80] Sanders DB, Lai HJ, Rock MJ, Farrell PM. Comparing age of cystic
fibrosis diagnosis and treatment initiation after newborn screening
with two common strategies. J Cyst Fibros 2012;11:150–3.
[81] Calvin J, Hogg SL, McShane D, McAuley SA, Iles R, Ross-Russell R,
et al. Thirty-years of screening for cystic fibrosis in East Anglia. Arch
Dis Child 2012;97:1043–7.
[82] Vernooij-van Langen AM, Loeber JG, Elvers B, Triepels RH, Gille JJ,
Van der Ploeg CP, et al. Novel strategies in newborn screening for
cystic fibrosis: a prospective controlled study. Thorax 2012;67:289–
95.
[83] Walshe K. International comparisons of the quality of health care:
what do they tell us? Qual Saf Health Care 2003;12:4–5.
[84] Dreachslin JL, Zernott M, Mendenhall S, Nieuwenjuijsen ER. Data
comparability issues in international research and quality assurance.
Qual Assur Health Care 1993;5:143–56.
[85] Kerr EA, Asch SM, Hamilton EG, McGlynn EA, editors. Quality of
care for general medical conditions: a review of the literature and
quality indicators. Santa Monica, CA: RAND Corporation; 2000.
[86] Marshall MN, Shekelle PG, McGlynn EA, Campbell S, Brook RH,
Roland MO. Can health care quality indicators be transferred between
countries? Qual Saf Health Care 2003;12:8–12.
[87] Ellis J. Sharing the evidence: clinical practice benchmarking to
improve continuously the quality of care. J Adv Nurs 2000;32:215-25.
[88] Goddard M, Mannion R, Smith P. Enhancing performance in health
care: a theoretical perspective on agency and the role of information.
Health Econ 2000;9:95–107.
[89] Sims G. International benchmarking from Centre level cystic fibrosis
data. Pediatr Pulmonol 2010;45(S33):527.
[90] Conway S, Balfour-Lynn IM, De Rijcke K, Drevinek P, Foweraker J,
Havermans T, et al. European Cystic Fibrosis Society Standards of
Care: framework for the cystic fibrosis centre. J Cyst Fibros
2014;13:3–22.
[91] Gawande A. The bell curve. N Y Ann Med 2004 The New Yorker.
[92] Delaisi B, Grosskopf C, Reignault E, Goehrs JM, Navarro J.
International registry on mucoviscidosis: comparison of the French
data with the European data for 1995. Arch Pediatr 1998;5:384–8.
[93] Lai HC, Corey M, FitzSimmons S, Kosorok MR, Farrell PM.
Comparison of growth status of patients with cystic fibrosis between
the United States and Canada. Am J Clin Nutr 1999;69:531–8.
[94] Fogarty A, Hubbard R, Britton J. International comparison of median
age at death from cystic fibrosis. Chest 2000;117:1656–60.
[95] Koch C, Cuppens H, Rainisio M, Madessani U, Harms H, Hodson M,
et al. European Epidemiologic Registry of Cystic Fibrosis (ERCF):
comparison of major disease manifestations between patients with
different classes of mutations. Pediatr Pulmonol 2001;31:1–12.
[96] McCormick J, Mehta G, Olesen HV, Viviani L, Macek Jr M, Mehta
A, et al.
Comparative demographics of the European cystic fibrosis population:
a cross-sectional database analysis. Lancet 2010;375:1007–13.
[97] Knudsen PK, Olesen HV, Høiby N, Johannesson M, Karpati F,
Laerum BN, et al. Differences in prevalence and treatment of
Pseudomonas aeruginosa in cystic fibrosis Centres in Denmark,
Norway and Sweden. J Cyst Fibros 2009;8:135–42.
[98] Jackson AD, Daly L, Kelleher C, Marshall BC, Quinton HB, Foley L,
et al.
The application of current life table methods to compare cystic
fibrosis median survival internationally is limited. J Cyst Fibros
2011;10:62–5.
[99] Boelle PY, Viviani L, Busson PF, Olesen H, Ravilly S, Stern M, et al.
Reference percentiles for FEV1 and BMI in European children and
adults with cystic fibrosis. Orphanet J Rare Dis 2012;7:64.
[100] Viviani L, Zolin A, Olesen HV. ECFSPR Annual Report 2008–2009.
European Cystic Fibrosis Society Patient Registry; 2012.
[101] McCormick J, Sims EJ, Green MW, Mehta G, Culross F, Mehta A.
Comparative analysis of Cystic Fibrosis Registry data from the UK
with USA, France and Australasia. J Cyst Fibros 2005;4:115–22.
[102] Sims G. International benchmarking for cystic fibrosis patient
outcomes using registry data. J Cyst Fibros 2009;8(S2):S103.
[103] Cystic fibrosis data network.
http://www.cysticfibrosisdata.org/metadata- and-methodology.htm.
[104] Sims G. Outline and proposal for an international Cystic Fibrosis Data
Network. http://www.cfww.org/programs/article/1319/Outline_and_
proposal_for_an_international_Cystic_Fibrosis_Data_Network_April
_ 2010; April 2010