An overview of international literature from cystic fibrosis registries. Part 4: Update 2011

Journal of Cystic Fibrosis

Volumn 11, Issue 6, 2012, Pages 480-493

  Salvatore, Donatello ^a   Buzzetti, Roberto ^b   Baldo, Ermanno ^c   Furnari, Maria Lucia ^d   Lucidi, Vincenzina ^e   Manunza, Daniela ^f   Marinelli, Italo ^g   Messore, Barbara ^h   Neri, Anna Silvia ⁱ   Raia, Valeria ^j   Mastella, Gianni ^b  

^a SAN CARLO HOSPITAL   (Italy)

^b Italian Cystic Fibrosis Research Foundation   (Italy)

^c Ospedale Santa Maria del Carmine   (Italy)

^d Ospedale dei Bambini G Di Cristina   (Italy)

^e BAMBINO GESÙ CHILDREN S HOSPITAL   (Italy)

^f BROTZU HOSPITAL   (Italy)

^g Ospedale Alto Chiascio   (Italy)

^h SAN LUIGI GONZAGA HOSPITAL   (Italy)

ⁱ UNIVERSITY OF FLORENCE   (Italy)

^j UNIVERSITY OF NAPLES FEDERICO II   (Italy)

more..

Author keywords

CF related diabetes; Cystic fibrosis; Demographics; Diagnosis factors; Epidemiology; Gender gap; Lung disease factors; Microbiology; Nutrition; Patient registry; Socioeconomic status; Therapy evaluation

Indexed keywords

ANTIBIOTIC AGENT; ANTIDIABETIC AGENT; BRONCHODILATING AGENT; COLISTIN; CORTICOSTEROID; INSULIN; LEUKOTRIENE RECEPTOR BLOCKING AGENT; MACROLIDE; SODIUM CHLORIDE; TOBRAMYCIN;

CHRONIC KIDNEY DISEASE; CLINICAL FEATURE; CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; DIABETES MELLITUS; DIET SUPPLEMENTATION; DISEASE COURSE; DISEASE EXACERBATION; DISEASE REGISTRY; ENVIRONMENTAL FACTOR; ETHNIC GROUP; FOLLOW UP; HEMOPTYSIS; HUMAN; LIFE EXPECTANCY; LONG TERM SURVIVAL; LUNG FIBROSIS; MEDICAL LITERATURE; MICROBIOLOGICAL EXAMINATION; MORBIDITY; OUTCOME ASSESSMENT; PREVALENCE; QUALITY OF LIFE; REVIEW; SOCIOECONOMICS; TREATMENT PLANNING;

CYSTIC FIBROSIS; HUMANS; INCIDENCE; INTERNATIONALITY; PREVALENCE; REGISTRIES;

EID: 84869083220     PISSN: 15691993     EISSN: 18735010     Source Type: Journal    
DOI: 10.1016/j.jcf.2012.07.005     Document Type: Review

References (82)

1. Registries for evaluating patient outcomes: a user's guide. (Prepared by Outcome DEcIDE Center [Outcome Sciences, Inc. d/b/a Outcome] under Contract No. HHSA29020050035I TO3.) AHRQ Publication No.10-EHC049. 2010, Agency for Healthcare Research and Quality, Rockville, MD, Available from: http://www.effectivehealthcare.ahrq.gov/ehc/products/74/531/Registries 2nd ed final to Eisenberg 9-15-10.pdf. 2nd ed. R.E. Gliklich, N.A. Dreyer (Eds.).
2. Buzzetti R., Salvatore D., Baldo E., et al. An overview of international literature from cystic fibrosis registries: 1. mortality and survival studies in cystic fibrosis. J Cyst Fibros 2009, 8:229-237.
3. Salvatore D., Buzzetti R., Baldo E., et al. An overview of international literature from cystic fibrosis registries 2. neonatal screening and nutrition/growth. J Cyst Fibros 2010, 9:75-83.
4. Salvatore D., Buzzetti R., Baldo E., et al. An overview of international literature from cystic fibrosis registries part 3. disease incidence, genotype/phenotype correlation, microbiology, pregnancy, clinical complications, lung transplantation, and miscellanea. J Cyst Fibros 2011, 10:71-85.
5. McCormick J., Mehta G., Olesen H.V., Viviani L., Macek M., Mehta A., et al. Demographics of the European cystic fibrosis population, a cross sectional study. Lancet 2010, 375:1007-1013.
6. Fogarty A., Hubbard R., Britton J. International comparison of median age at death from cystic fibrosis. Chest 2000, 117:1656-1660.
7. Mehta G., Macek M., Mehta A. Cystic fibrosis across Europe: EuroCareCF analysis of demographic data from 35 countries. J Cyst Fibros 2010, 9(Suppl. 2):S5-S21. on behalf of the European Registry Working Group.
8. http://www.ecfs.eu/projects/ecfs-patient-registry/intro.
9. Bell S.C., Bye P.T., Cooper P.J., et al. Cystic fibrosis in Australia, 2009: results from a data registry. Med J Aust 2011, 195:396-400.
10. Farrell P.M. The prevalence of cystic fibrosis in the European Union. J Cyst Fibros 2008, 7:450-453.
11. Danieli Watts K., Seshadri R., Sullivan C., McColley S.A. Increased prevalence of risk factors for morbidity and mortality in the US Hispanic CF population. Pediatr Pulmonol 2009, 44:594-601.
12. Jackson A.D., Daly L., Kelleher C., et al. The application of current lifetable methods to compare median survival internationally is limited. J Cyst Fibros 2011, 10:62-65.
13. Jackson A.D., Daly L., Al Jackson, et al. Validation and use of a parametric model for projecting cystic fibrosis survivorship beyond observed data: a birth cohort analysis. Thorax 2011, 66:674-679.
14. Viviani L., Bossi A., Assael B.M., et al. Absence of a gender gap in survival. An analysis of the italian registry for cystic fibrosis in the paediatric age. J Cyst Fibros 2011, 10:313-317.
15. Olesen H.V., Pressler T., Hjelte L., et al. Gender differences in the Scandinavian cystic fibrosis population. Pediatr Pulmonol 2010, 45:959-965.
16. Pittman J.E., Calloway E.H., Kiser M., et al. Age of Pseudomonas aeruginosa acquisition and subsequent severity of cystic fibrosis lung disease. Pediatr Pulmonol 2011, 46:497-504.
17. Barr H.L., Britton J., Smyth A.R., Fogarty A.W. Association between socioeconomic status, sex, and age at death from cystic fibrosis in England and Wales (1959 to 2008): cross sectional study. BMJ 2011, 343:d4662.
18. Burgel P.R., Fajac I., Hubert D., et al. Non-classic cystic fibrosis associated with D1152H CFTR mutation. Clin Genet 2010, 77:355-364.
19. Castellani C., Cuppens H., Macek M.J., et al. Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice. J Cyst Fibros 2008, 7:179-196.
20. Lai H.J., Shoff S.M. Classification of malnutrition in cystic fibrosis: implications for evaluating and benchmarking clinical practice performance. Am J Clin Nutr 2008, 88:161-166.
21. Hirche T.O., Hirche H., Jungblut S., Stern M., Wagner T.O., Wiedemann B., et al. Group. Statistical limitations of percent ideal body weight as measure for nutritional failure in patients with cystic fibrosis. J Cyst Fibros 2009, 8:238-244.
22. Zhang Z., Shoff S.M., Lai H.J. Incorporating genetic potential when evaluating stature in children with cystic fibrosis. J Cyst Fibros 2010, 9:135-142.
23. Arrigo T., DeLuca F., Sferlazzas C., et al. Young adults with cystic fibrosis are shorter than healthy peers because their parents are also short. Eur J Pediatr 2005, 164:781-782.
24. Falkner F., Tanner J.M. Human growth 1986, 104-107. Plenum Press, New York, 3. second edition.
25. Himes J.H., Roche A.F., Thissen D., Moore W.M. Parent-specific adjustments for evaluation of recumbent length and stature of children. Pediatrics 1985, 75:304-313.
26. LiPuma J.J. The changing microbial epidemiology in cystic fibrosis. Clin Microbiol Rev 2010, 23:299-323.
27. Murray S., Charbeneau J., Marshall B.C., LiPuma J.J. Impact of Burkholderia infection on lung transplantation in cystic fibrosis. Am J Respir Crit Care Med 2010, 178:363-371.
28. De Soyza A., McDowell A., Archer L., et al. Burkholderia cepacia complex genomovars and pulmonary transplantation outcomes in patients with cystic fibrosis. Lancet 2001, 358:1780-1781.
29. Aris R.M., Routh J.C., LiPuma J.J., Heath D.G., Gilligan P.H. Lung transplantation for cystic fibrosis patients with Burkholderia cepacia complex: survival linked to genomovar type. Am J Respir Crit Care Med 2001, 164:2102-2106.
30. Razvi S., Quittell L., Sewall A., Quinton H., Marshall B., Saiman L. Respiratory microbiology of patients with cystic fibrosis in the United States, 1995 to 2005. Chest 2009, 136:1554-1560.
31. Millar F.A., Simmonds N.J., Hodson M.E. Trends in pathogens colonising the respiratory tract of adult patients with cystic fibrosis, 1985-2005. J Cyst Fibros 2009, 8:386-391.
32. Coffin S., Rettig S., Bell L., Scanlin T., St John K. Comparison of prevalence calculations using infection control surveillance methods and cystic fibrosis foundation patient registry guidelines. Am J Infect Control 2004, 32:E85.
33. Sawicki G.S., Rasouliyan L., Pasta D.J., Regelmann W.R., Wagener J.L., Waltz D.A., et al. The impact of incident methicillin resistant Staphylococcus aureus detection on pulmonary function in cystic fibrosis. Pediatr Pulmonol 2008, 43:1117-1123.
34. Dasenbrook E.C., Merlo C.A., Diener-West M., Lechtzin N., Boyle M.P. Persistent methicillin-resistant Staphylococcus aureus and rate of FEV1 decline in cystic fibrosis. Am J Respir Crit Care Med 2008, 178:814-821.
35. Sawicki G.S., Rasouliyan L., Ren C.L. The impact of MRSA on lung function in patients with cystic fibrosis. Am J Respir Crit Care Med 2009, 179:734-735. [author reply 735].
36. Dasenbrook E.C., Checkley W., Merlo C.A., Konstan M.W., Lechtzin N., Boyle M.P. Association between respiratory tract methicillin-resistant Staphylococcus aureus and survival in cystic fibrosis. JAMA 2010, 303:2386-2392.
37. Harrison F. Microbial ecology of the cystic fibrosis lung. Microbiology 2007, 153:917-923.
38. Sagel S.D., Gibson R.L., Emerson J., McNamara S., Burns J.L., Wagener J.S., et al. Inhaled tobramycin in young children study group; cystic fibrosis foundation therapeutics development network. Impact of Pseudomonas and Staphylococcus infection on inflammation and clinical status in young children with cystic fibrosis. J Pediatr 2009, 154:183-188.
39. Adler A.I., Shine B.S., Chamnan P., Haworth C.S., Bilton D. Genetic determinants and epidemiology of cystic fibrosis-related diabetes: results from a British cohort of children and adults. Diabetes Care 2008, 31:1789-1794.
40. Chamnan P., Shine B.S., Haworth C.S., Bilton D., Adler A.I. Diabetes as a determinant of mortality in cystic fibrosis. Diabetes Care 2010, 33:311-316.
41. Adler A.I., Shine B., Haworth C., Leelarathna L., Bilton D. Hyperglycemia and death in cystic fibrosis-related diabetes. Diabetes Care 2011, 34:1577-1578.
42. Moran A., Dunitz J., Nathan B., Saeed A., Holme B., Thomas W. Cystic fibrosis-related diabetes: current trends in prevalence, incidence, and mortality. Diabetes Care 2009, 32:1626-1631.
43. Efrati O., Harash O., Rivlin J., et al. Hemoptysis in Israeli CF patients - prevalence, treatment and clinical characteristics. J Cyst Fibros 2008, 7:301-306.
44. Quon B.S., MayerHamblett N., Aitken M.L., Smyth A.R., Goss C.H. Risk factors for chronic kidney disease in adults with cystic fibrosis. AJRCCM 2011, 184:1147-1152.
45. Jackson A., Foley L., Daly L., et al. Delayed cystic fibrosis presentation in children in the absence of newborn screening. Ir Med J 2010, 103:113-116.
46. Keating C.L., Liu X., DiMango E.A. Classic respiratory disease but atypical diagnostic testing distinguishes adult presentation of cystic fibrosis. Chest 2010, 137:1157-1163.
47. Yan J., Cheng Y., Fine J.P., Lai H.J. Uncovering symptom progression history from disease registry data with application to young cystic fibrosis patients. Biometrics 2010, 66:594-602.
48. Sanders D.B., Lai H.J., Rock M.J., Farrell P.M. Comparing age of cystic fibrosis diagnosis and treatment initiation after newborn screening with two common strategies. J Cyst Fibros 2012, 11:150-153.
49. VanDevanter D.R., Rasouliyan L., Murphy T.M., Morgan W.J., Ren C.L., Konstan M.W., et al. Investigators, coordinators of the epidemiologic study of cystic fibrosis. Trends in the clinical characteristics of the U.S. cystic fibrosis patient population from 1995 to 2005. Pediatr Pulmonol 2008, 43:739-744.
50. Konstan M.W., Morgan W.J., Butler S.M. Scientific Advisory Group and the Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. Risk factors for rate of decline in forced expiratory volume in one second in children and adolescents with cystic fibrosis. J Pediatr 2007, 151:134-139. [(*) This study was evaluated to be included in the previous review article (4), but it was excluded by editing mistake. We decided to include it in the present paper].
51. VanDevanter D.R., Wagener J.S., Pasta D.J., Elkin E., Jacobs J.R., Morgan W.J., et al. Pulmonary outcome prediction (POP) tools for cystic fibrosis patients. Pediatr Pulmonol 2010, 45:1156-1166.
52. Liou T.G., Elkin E.P., Pasta D.J., Jacobs J.R., Konstan M.W., Morgan W.J., et al. Year-to-year changes in lung function in individuals with cystic fibrosis. J Cyst Fibros 2010, 9:250-256.
53. Konstan M.W., Wagener J.S., VanDevanter D.R. Characterizing aggressiveness and predicting future progression of CF lung disease. J Cyst Fibros 2009, 8(Suppl. 1):S15-S19.
54. Buzzetti R., Alicandro G., Minicucci L., et al. Validation of a predictive survival model in Italian patients with cystic fibrosis. J Cyst Fibros 2012, 11:24-29.
55. Sanders D.B., Bittner R.C., Rosenfeld M., Hoffman L.R., Redding G.J., Goss C.H. Failure to recover to baseline pulmonary function after cystic fibrosis pulmonary exacerbation. Am J Respir Crit Care Med 2010, 182:627-632.
56. Sanders D.B., Bittner R.C., Rosenfeld M., Redding G.J., Goss C.H. Pulmonary exacerbations are associated with subsequent FEV1 decline in both adults and children with cystic fibrosis. Pediatr Pulmonol 2011, 46:393-400.
57. Ortiz J.R., Neuzil K.M., Victor J.C., Wald A., Aitken M.L., Goss C.H. Influenza-associated cystic fibrosis pulmonary exacerbations. Chest 2010, 137:852-860.
58. Ortiz J.R., Neuzil K.M., Victor J.C., Aitken M.L., Goss C.H. Predictors of influenza vaccination in the Cystic Fibrosis Foundation patient registry, 2006 through 2007. Chest 2010, 138:1448-1455.
59. Stephenson A., Hux J., Tullis E., Austin P.C., Corey M., Ray J. Higher risk of hospitalization among females with cystic fibrosis. J Cyst Fibros 2011, 10:93-99.
60. Nick J.A., Chacon C.S., Brayshaw S.J., et al. Effects of gender and age at diagnosis on disease progression in long-term survivors of cystic fibrosis. Am J Respir Crit Care Med 2010, 182:614-626.
61. Ji S., Peng L., Cheng Y., Lai H. Quantile regression for doubly censored data. Biometrics 2012, 68:101-112.
62. Collaco J.M., McGready J., Green D.M., et al. Effect of temperature on cystic fibrosis lung disease and infections: a replicated cohort study. PLoS One 2011, 6:e27784.
63. Schechter M.S., McColley S.A., Silva S., Haselkorn T., Konstan M.W., Wagener J.S. Association of Socioeconomic Status with the Use of Chronic Therapies and Healthcare Utilization in Children with Cystic Fibrosis. J Pediatr 2009, 155:634-639.
64. Schechter M.S., McColley S.A., Regelmann W., et al. Socioeconomic status and the likelihood of antibiotic treatment for signs and symptoms of pulmonary exacerbation in children with cystic fibrosis. J Pediatr 2011, 159:819-824.
65. Quittner A.L., Schechter M.S., Rasouliyan L., Haselkorn T., Pasta D.J., Wagener J.S. Impact of socioeconomic status, race, and ethnicity on quality of life in patients with cystic fibrosis in the United States. Chest 2010, 137:642-650.
66. Stephenson A., Hux J., Tullis E., Austin P.C., Corey M., Ray J. Socioeconomic status and risk of hospitalization among individuals with cystic fibrosis in Ontario, Canada. Pediatr Pulmonol 2011, 46:376-384.
67. Sawicki G.S., Rasouliyan L., McMullen A.H., Wagener J.S., McColley S.A., Pasta D.J., et al. Longitudinal assessment of health-related quality of life in an observational cohort of patients with cystic fibrosis. Pediatr Pulmonol 2011, 46:36-44.
68. Goldbeck L., Besier T., Hinz A., Singer S. Quittner AL; TIDES Group. Prevalence of symptoms of anxiety and depression in German patients with cystic fibrosis. Chest 2010, 138:929-936.
69. Quittner A.L., Sawicki G.S., McMullen A., et al. Psychometric evaluation of the cystic fibrosis questionnaire - revised in a national sample. Qual Life Res 2012, 10.1007/s11136-011-0091-5.
70. Ren C.L., Pasta D.J., Rasouliyan L., Wagener J.S., Konstan M.W., Morgan W.J. Scientific advisory group and the investigators and coordinators of the epidemiologic study of cystic fibrosis. Relationship between inhaled corticosteroid therapy and rate of lung function decline in children with cystic fibrosis. J Pediatr 2008, 153:746-751.
71. De Boeck K., Vermeulen F., Wanyama S. Thomas M; members of the Belgian CF Registry. Inhaled corticosteroids and lower lung function decline in young children with cystic fibrosis. Eur Respir J 2011, 37:1091-1095.
72. Balfour-Lynn I.M., Lees B., Hall P., et al. Multicenter randomized controlled trial of withdrawal of inhaled corticosteroids in cystic fibrosis. Am J Respir Crit Care Med 2006, 173:1356-1362.
73. Schmoor C., Caputo A., Schumacher M. Evidence from nonrandomized studies: a case study on the estimation of causal effects. Am J Epidemiol 2008, 167:1120-1129.
74. Schechter M.S. Patient registry analyses: seize the data, but caveat lector. J Pediatr 2008, 153:733-735.
75. Konstan M.W., VanDevanter D.R., Rasouliyan L. Scientific advisory group; investigators and coordinators of the epidemiologic study of cystic fibrosis. Trends in the use of routine therapies in cystic fibrosis: 1995-2005. Pediatr Pulmonol 2010, 45:1167-1172.
76. Moskowitz S.M., Silva S.J., Mayer-Hamblett N., et al. Shifting patterns of inhaled antibiotic use in cystic fibrosis. Pediatr Pulmonol 2008, 43:874-881.
77. Konstan M.W., Wagener J.S., Pasta D.J., et al. Scientific advisory group and investigators and coordinators of epidemiologic study of cystic fibrosis. Clinical use of dornase alpha is associated with a slower rate of FEV1 decline in cystic fibrosis. Pediatr Pulmonol 2011, 46:545-553.
78. Jones A.P., Wallis C. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev 2010 Mar, 17(3):CD001127.
79. Sawicki G.S., Signorovitch J.E., Zhang J., et al. Reduced mortality in cystic fibrosis patients treated with tobramycin inhalation solution. Pediatr Pulmonol 2012, 47:44-52.
80. Konstan M.W., Wagener J.S., Yegin A., Millar S.J., Pasta D.J., VanDevanter D.R. Design and powering of cystic fibrosis clinical trials using rate of FEV(1) decline as an efficacy endpoint. J Cyst Fibros 2010, 9:332-338.
81. Davis P.B., Byard P.J., Konstan M.W. Identifying treatments that halt progression of pulmonary disease in cystic fibrosis. Pediatr Res 1997, 41:161-165.
82. VanDevanter D.R., Yegin A., Morgan W.J., Millar S.J., Pasta D.J., Konstan M.W. Design and powering of cystic fibrosis clinical trials using pulmonary exacerbation as an efficacy endpoint. J Cyst Fibros 2011, 10:453-459.