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Re: Re: Recent advances in cystic fibrosis

Dear Editor

Doull's review of recent advances in cystic fibrosis [1] gives insufficient recognition to the development of liver transplantation as an effective treatment for decompensating liver disease [2-5].

Liver disease is an important problem in children with cystic fibrosis (CF). After cardiorespiratory problems and transplantation complications, liver disease is the commonest cause of death accounting for 2.3% of overall CF mortality [6]:

Isolated liver transplantation in children has an excellent survival rate with 100% reported in two paediatric series [3 and Dr Deirdre Kelly Birmingham Children's Hospital, personal communication] and 75% survival in another series where the subjects clearly had more advanced liver disease at the time of liver transplantation [4].

Improved outcomes depend on careful selection. Delaying liver transplantation until malnutrition, encephalopathy and jaundice develop has been associated with more morbidity and mortality [4]. Transplantation is indicated for decompensating liver disease, evidenced by progressive hypoalbuminaemia, coagulopathy and malnutrition unresponsive to management [3]. Children with severe pulmonary disease require heart, lung and liver transplantation where outcomes are poor. Thus, isolated liver transplantation is reserved for children with only moderate lung disease (usually taken to mean FEV1 and FVC greater than 50% of expected for age).

Lung function is maintained or improves after liver transplantation, and fears of an increased incidence of pulmonary infection due to immunosuppression have proved unfounded. Reduced pulmonary oedema, intrapulmonary shunting and diaphragmatic splinting have been postulated to explain the effect on respiratory function. Preoperative use of DNAase may reduce perioperative pulmonary complications [2].

Nutritional status probably improves too but the evidence for this is less certain. Infant recipients of liver grafts for other indications show a dramatic increase in mid arm circumference within twelve to eighteen months of transplantation [7]. However, 20% of transplant recipients have persistent behavioural problems requiring long term nasogastric tube feeding. Our impression is that this may be more common in recipients with CF.

The management of decompensated CF liver disease has dramatically improved in the past 20 years with refinements in management of portal hypertension and the availability of isolated liver transplantation for selected children and adults.

VIN DIWAKAR Specialist Registrar

PETER WELLER Consultant Paediatrician

SUE BEATH Consultant Paediatric Hepatologist

DEIRDRE KELLY Reader in Paediatric Hepatology

References

(1) Doull IJM. Recent Advances in Cystic Fibrosis. Arch Dis Child 2001;85: 62-6

(2) Noble Jamieson G, Valente J, Barnes N D, Friend P J, Jamieson N V, Rasmussen A, Calne R Y. Liver transplantation for hepatic cirrhosis in cystic fibrosis. Arch Dis Child 1994;71: 349-352

(3) Noble Jamieson G, Barnes N, Jamieson N, Friend P, Calne R. Liver transplantation for hepatic cirrhosis in cystic fibrosis. J Roy Soc Med 1996; 89: 31-7

(4) Mack D, Traystaman M, Colombo J, Sammut P, Kauffman S S, Vanderhoof J A et al. Clinical denouement and mutation analysis of patients with cystic fibrosis undergoing liver transplantation for biliary fibrosis. J Pediatr 1995; 127: 881-7

(5) Molmenti E, Nagata D, Roden J, Squires R et al. Pediatric liver transplantation for cystic fibrosis. Transplant Proc 2001; 33: 1738

(6) Fitzsimmons S C. Cystic Fibrosis Foundation, Patient Registry 1996 Annual Data Report. Bethesda, Maryland. August 1997.