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.
Keywords
cystic fibrosis - liver transplantation - pancreas transplantation - diabetes mellitus
type I
