Eur J Pediatr Surg 2012; 22(01): 060-066
DOI: 10.1055/s-0031-1291288
Original Article
Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Liver Transplantation in Children with Cystic Fibrosis: Experience in our Centre and Preliminary Results with a Combined En Bloc Liver-Pancreas Graft

M. Miguel
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
,
A.M. Andres
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
,
M. Lopez-Santamaria
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
,
S. Barrena
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
,
L. Hierro
2  Hospital Universitario La Paz, Servicio de Hepatología Infantily Trasplante Hepático Infantil, Madrid, Spain
,
F. Hernandez
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
,
M. Ramírez
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
,
E. Frauca
2  Hospital Universitario La Paz, Servicio de Hepatología Infantily Trasplante Hepático Infantil, Madrid, Spain
,
J.L. Encinas
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
,
S. Lopez-Fernandez
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
,
P. Jara
2  Hospital Universitario La Paz, Servicio de Hepatología Infantily Trasplante Hepático Infantil, Madrid, Spain
,
J.A. Tovar
1  Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain
› Author Affiliations
Further Information

Publication History

16 May 2011

21 September 2011

Publication Date:
07 December 2011 (online)

Abstract

Aim of the study Cystic fibrosis (CF) is a multisystemic disease, with some patients developing end-stage liver disease (ESLD), requiring liver transplantation (LT). These children usually present with severe mutations of the CFTR gene. Almost 100% of patients with severe mutations develop exocrine pancreatic insufficiency, leading later to endocrine insufficiency. Immunosuppression accelerates the development of insulin-dependent diabetes (IDD) in transplanted children with CF. Our aims were: 1) to analyze our experience with CF-related ESLD children who received LT, and the relationship to the development of IDD; 2) to report our preliminary results with en bloc liver-pancreas transplantation (CLPT).

Methods 9 children (6M/3F) with CF and ESLD underwent LT between 1993 and 2010; median age and weight were 12.3 years (range: 5.4–17.0) and 36.7 kg (range: 14.2–58.5), respectively. 4 patients received a whole graft, 4 had reduced grafts (1 split) and 1 underwent CLPT. Immunosuppression followed the protocols at the time of transplantation.

Results Liver function was restored in all patients and none of them needed re-transplantation. Median follow-up was 105 months (range: 4–206). 1 child died of respiratory failure at 23 months after transplantation while awaiting pulmonary transplantation. Survival (Kaplan-Meier) at 105 months was 87.5%. 4 children already had IDD before transplantation and 3 developed diabetes immediately after transplantation. 2 had not developed IDD at the end of the study: the youngest at the time of LT (5.4 years, follow-up 7.1 years) and the girl who had had CLPT and who recovered normal exocrine and endocrine pancreatic function after transplantation.

Conclusions LT is a realistic option to treat CF-related ESLD children. IDD is common in these patients. En bloc liver-pancreas transplantation is an appealing option, since it simultaneously restores exocrine function and prevents IDD. This procedure has clear technical advantages over simultaneous isolated liver and pancreas transplantation.