Liver transplantation and the management of progressive familial intrahepatic cholestasis in children

Table 1 Patients Intervention Comparator Outcomes table for assessment of progressive familial intrahepatic cholestasis

P	I	C	O
Pediatric patients with PFIC	Liver transplantation	Biliary diversion and medical management	Patients survival, graft survival, post operative morbidity

PFIC: Progressive familial intrahepatic cholestasis; P: Patients; I: Intervention; C: Comparator; O: Outcomes.

Table 2 Review of documented liver transplantation outcomes for progressive familial intrahepatic cholestasis patients (≥ 3 patients)

Ref.	PFIC type	Age at transplant (years old)	Previous management	Graft survival	Patient survival	Notes
Soubrane et al[45]	14 “byler disease” PFIC type unspecified	6.5 (0.4-13)	NR	93.3%	92.8%	Consanguineous to the 2nd degree in 8 cases
Emond et al[91]	11 PFIC unspecified type	4.6 ± 3.4	2 had previous partial biliary diversion procedures	76.9%	73%	LT performed on those with advanced cirrhosis (6 received diversion procedures only)
Ismail et al[80]	8 PFIC of unspecified type	Unknown	1 patient PEBD, all received cholestyramine, phenobarbital, rifampicin, UDCA	100%	85.7%	6 cadaver livers, 2 living donors
Kondo et al[63]	4 PFIC of unspecified type	2-7	NR	75%	75%
Bassas et al[56]	5 PFIC3 8 “low GGT PFIC” PFIC1/2	10-40 mo	NR	84.6%	84.6%	Parents of 12 out of 13 were 1st cousins
Cutillo et al[57]	6 PFIC1/2 1 PFIC3	4-53 mo	NR	100%	75%
Englert et al[44]	33 patients PFIC2 and 3	Unknown	UDCA 10 of 33 received biliary diversion then LT	100% with prior diversion 89% without prior diversion	100%
Aydogdu et al[52]	10 PFIC1/2 2 PFIC3	43.2 ± 27 mo	UDCA	69.2%	75%	Surviving patients show good quality of life, exacerbation of diarrhea as the exception, mix of LDLT and cadaveric
Hori et al[50,51]	11 PFIC1	0.6-18.2 years old	Total external biliary diversion performed at time of re-transplantation in one PFIC1 patient	82.4% total graft survival (14/17)	PFIC1 - 90.9% at 5 yr, 72.7% at 10 yr, 54.5% at 15 yr; PFIC2 - 100% at 5 yr	Digestive symptoms in 10 out of 11 PFIC1; 8 out of 11 PFIC1 recipients exhibited steatosis; 9 out of 11 PFIC1 recipients exhibited fibrosis
Miyagawa-Hayashino et al[54], Egawa et al[53]	3 PFIC2
Kaur et al[58]	2 PFIC3 2 PFIC1/2	2, 2.5, 6 and 9 years old males	UDCA, phenobarbital and ondansetron	100%	75%

LT: Liver transplantation; PEBD: Partial external biliary diversion; UDCA: Ursodeoxycholic acid; PFIC: Progressive familial intrahepatic cholestasis; GGT: Gamma-glutamyl transpeptidase; LDLT: Living donor liver transplantation; NR: Not reported.