Ricerca
Trapianto di polmone in fibrosi cistica
Lung transplantation in cystic fibrosis: a regional Center experience
Anna Agrusti1, Matteo Bramuzzo2, Massimo Maschio2, Alessandro Ventura1,2
1Università di Trieste; 2IRCCS Materno-Infantile “Burlo-Garofolo”, Trieste
Febbraio 2017 - pagg. 99 -106
Abstract
Background - Cystic fibrosis (CF) is a rare genetic disease that progressively involves the
pancreatic and pulmonary functions: as respiratory failure develops, lung transplantation
is the only therapeutic option available nowadays.
Objective - The purpose of the paper is to describe the efficacy and the safety of bilateral lung transplantation in patients with cystic fibrosis.
Materials and Methods - The clinical records of the patients followed by the Regional Centre of Diagnosis and Treatment of CF in Trieste (Italy) who underwent lung transplantation between 1970 and June 2015 were analysed. Survival rate, respiratory, metabolic and nutritional parameters, quality of life and complications after transplantation were evaluated.
Results - Ten (12%) out of 83 patients underwent bilateral lung transplantation (average age 28.3 ± 9.5 years old, M:F=5:5) and 2 (20%) were in paediatric age (11 and 18 years). Five-year survival rate was 80%: one patient (10%) died because of a primary graft dysfunction and one patient (10%) died of Burkholderia Cepacia sepsis (10%). Indication for lung transplantation was chronic respiratory failure in 80% of the patients and acute respiratory failure in 20%. The mean time spent in the waiting list was 18.8 ± 13.2 months; the mean post-transplantation follow-up time was 4.5 ± 4 years. After transplantation, mean FEV1%, FVC%, FEF25-75% and oxygen saturation significantly improved (p value < 0.05) and no patient needed NIV or oxygen supplementation. Weight and BMI also improved (p value=0.3). The most frequent complications after transplantation were CMV infections, arterial hypertension, acute rejection, neutropoenia, dyslipidemia, gastro-intestinal problems and renal dysfunction. Scores in the Cystic Fibrosis Quality of Life questionnaire significantly improved in all the 12 fields explored.
Conclusions - Lung transplantation is the only life-saving option available for end-stage pulmonary disease in patients with CF: it can improve the pulmonary function, the metabolic and nutritional profile and the quality of life, even if monitoring for post-transplantation complications is required.
Objective - The purpose of the paper is to describe the efficacy and the safety of bilateral lung transplantation in patients with cystic fibrosis.
Materials and Methods - The clinical records of the patients followed by the Regional Centre of Diagnosis and Treatment of CF in Trieste (Italy) who underwent lung transplantation between 1970 and June 2015 were analysed. Survival rate, respiratory, metabolic and nutritional parameters, quality of life and complications after transplantation were evaluated.
Results - Ten (12%) out of 83 patients underwent bilateral lung transplantation (average age 28.3 ± 9.5 years old, M:F=5:5) and 2 (20%) were in paediatric age (11 and 18 years). Five-year survival rate was 80%: one patient (10%) died because of a primary graft dysfunction and one patient (10%) died of Burkholderia Cepacia sepsis (10%). Indication for lung transplantation was chronic respiratory failure in 80% of the patients and acute respiratory failure in 20%. The mean time spent in the waiting list was 18.8 ± 13.2 months; the mean post-transplantation follow-up time was 4.5 ± 4 years. After transplantation, mean FEV1%, FVC%, FEF25-75% and oxygen saturation significantly improved (p value < 0.05) and no patient needed NIV or oxygen supplementation. Weight and BMI also improved (p value=0.3). The most frequent complications after transplantation were CMV infections, arterial hypertension, acute rejection, neutropoenia, dyslipidemia, gastro-intestinal problems and renal dysfunction. Scores in the Cystic Fibrosis Quality of Life questionnaire significantly improved in all the 12 fields explored.
Conclusions - Lung transplantation is the only life-saving option available for end-stage pulmonary disease in patients with CF: it can improve the pulmonary function, the metabolic and nutritional profile and the quality of life, even if monitoring for post-transplantation complications is required.
Parole chiave
Suggerite dall'AI
Classificazione MeSH
Contenuto riservato
Per leggere l'articolo completo è necessario effettuare il login.
Non sei ancora registrato? Registrati
Bibliografia
1. O’Sullivan BP, Freedman SD. Cystic fibrosis.
Lancet 2009;373(9678):1891-904.
2. Kim RJ. Cystic fibrosis-related diabetes in
children: an update. Pediatr Ann 2016;45(9):e
321-6.
3. Smyth AR, Bell SC, Bojcin S, et al.; European
Cystic Fibrosis Society. European Cystic
Fibrosis Society Standards of Care: Best Practice
guidelines. J Cyst Fibros 2014;13(Suppl
1):S23-42.
4. Schindler T, Michel S, Wilson AW. Nutrition
management of cystic fibrosis in the 21st
century. Nutr Clin Pract 2015;30(4):488-500.
5. Ramsey BW, Davies J, McElvaney G, 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(18):
1663-72.
6. Boyle MP, Bell SC, Konstan MW, et al.;
VX09-809-102 study group. A CFTR corrector
(lumacaftor) and a CFTR potentiator (ivacaftor)
for treatment of patients with cystic fibrosis
who have a phe508del CFTR mutation: a
phase 2 randomised controlled trial. Lancet
Respir Med 2014;2(7):527-38.
7. Wainwright CE, Elborn JS, Ramsey BW, et
al.; TRAFFIC Study Group; TRANSPORT
Study Group. Lumacaftor-Ivacaftor in Patients
with Cystic Fibrosis Homozygous for
Phe508del CFTR. N Engl J Med 2015;373:220-
31.
8. Hirche TO, Knoop C, Hebestreit H, et al.; ECORN-
CF Study Group. Practical guidelines:
lung transplantation in patients with cystic fibrosis.
Pulm Med 2014;2014:621342.
9. Lane JT, Dagogo-Jack S. Approach to the
patient with new-onset diabetes after transplant
(NODAT). J Clin Endocrinol Metab
2011;96(11):3289-97.
10. Lovric S, Kielstein JT, Kayser D, et al. Combination
of everolimus with calcineurin inhibitor
medication resulted in post-transplant haemolytic
uraemic syndrome in lung transplant
recipients - a case series. Nephrol Dial Transplant
2011;26(9):3032-8.
11. Modi AC, Quittner AL. Validation of a disease-
specific measure of health-related Quality
of life for children with cystic fibrosis. J Pediatr
Psychol 2003;28(8):535-45.
12. Quittner AL, Buu A, Messer MA, Modi AC,
Watrous M. Development and validation of
The Cystic Fibrosis Questionnaire in the United
States: a health-related quality-of-life measure
for cystic fibrosis. Chest 2005;128(4):
2347-54.
13. Hinchey J, Chaves C, Appignani B, et al. A
reversible posterior leukoencephalopathy syndrome.
N Engl J Med 1996;334(8):494-500.
14. Covarrubias DJ, Luetmer PH, Campeau
NG. Posterior reversible encephalopathy syndrome:
prognostic utility of quantitative diffusion-
weighted MR images. AJNR Am J Neuroradiol
2002;23(6):1038-48.
15. Fugate JE, Claassen DO, Cloft HJ, Kallmes
DF, Kozak OS, Rabinstein AA. Posterior Reversible
Encephalopathy Syndrome: associated
clinical and radiologic findings. Mayo Clin
Proc 2010;85(5):427-32.
16. Cullinan N, Gorman KM, Riordan M, Waldron
M, Goodship TH, Awan A. Case report:
benefits and challenges of long-term eculizumab
in atypical hemolytic uremic syndrome.
Pediatrics 2015;135(6):e1506-9.
17. Barker AF, Bergeron A, Rom WN, Hertz
MI. Obliterative bronchiolitis. N Engl J Med
2014;370(19):1820-8.
18. Sato M, Waddell TK, Wagnetz U, et al. Restrictive
allograft syndrome (RAS): a novel
form of chronic lung allograft dysfunction. J
Heart Lung Transplant 2011;30(7):735-42.
19. Verleden SE, Ruttens D, Vandermeulen E,
et al. Restrictive chronic lung allograft dysfunction:
where we are now? J Heart Lung
Transplant 2015;34(5):625-30.
20. Wollin L, Wex E, Pautsch A, et al. Mode of
action of nintedanib in the treatment of idiopathic
pulmonary fibrosis. Eur Respir J 2015;
45(5):1434-45.
21. Suhling H, Bollmann B, Gottlieb J. Nintedanib
in restrictive chronic lung allograft dysfunction
after lung transplantation. J Heart
Lung Transplant 2016;35(7):939-40.
22. Al-Mansour Z, Nelson BP, Evens AM. Post-
transplant lymphoproliferative disease (PTLD):
risk factors, diagnosis, and current
treatment strategies. Curr Hematol Malig Rep
2013;8(3):173-83.
23. International Society for Heart and Lung
Transplantation. Registries > Heart/Lung
Transplant Registry > Slides > Adult lung transplantation
statistics. ISHLT 2016;35(10):1149-
205. www.ishlt.org/registries.
24. International Society for Heart and Lung
Transplantation. Registries > Heart/Lung
Transplant Registry > Slides > Pediatric lung
transplantation statistics. ISHLT 2016;35(10):
1149-205. www.ishlt.org/registries.
25. Chaparro C, Keshavjee S. Lung transplantation
for cystic fibrosis: an update. Expert Rev
Respir Med 2016;10(12):1269-80.
26. Aurora P, Spencer H, Moreno-Galdó A.
Lung transplantation in children with cystic fibrosis:
a view from Europe. Am J Respir Crit
Care Med 2008;177(9):935-40:27.
27. Robinson W, Waltz DA. FEV(1) as a guide
to lung transplant referral in young patients
with cystic fibrosis. Pediatr Pulmonol 2000;30
(3):198-202.
28. Corris PA. Lung transplantation for cystic
fibrosis and bronchiectasis. Semin Respir Crit
Care Med 2013;34(3):297-304.
29. Hayes D Jr, McCoy KS, Whitson BA, Mansour
HM, Tobias JD. High-risk age window for
mortality in children with cystic fibrosis after
lung transplantation. Pediatr Transplant 2015;
19(2):206-10.
30. Hayes D Jr, Glanville AR, McGiffin D, Tobias
JD, Tumin D. Age-related survival disparity
associated with lung transplantation in cystic
fibrosis: An analysis of the registry of the
International Society for Heart and Lung Transplantation.
J Heart Lung Transplant 2016;35
(9):1108-15.
31. International Society for Heart and Lung
Transplantation. Registries > Heart/Lung
Transplant Registry > Quarterly data report about
lung transplantation. www.ishlt.org/registries.
32. NHS Blood and Transplant. Annual report
on cardiothoracic transplantation for 2014/
2015. July 2016.
33. Valapour M, Skeans MA, Smith JM, et al.
Lung. Am J Transplant 2016;16(Suppl 2):141-
68.
Corrispondenza: anna.agrusti88@gmail.com