Partial External Biliary Diversion in Children With Progressive Familial Intrahepatic Cholestasis and Alagille Disease

Yang, Huiqi; Porte, Robert J.; Verkade, Henkjan J.; Langen, Zacharias J. de; Hulscher, Jan B F

doi:10.1097/mpg.0b013e31819a4e3d

Cited by 59 publications

(79 citation statements)

References 17 publications

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“…Liver transplantation, which is the only definitive treatment of this condition, is limited by organ availability and may be associated with significant morbidity and mortality [3,4]. Alternative surgical treatments include partial biliary diversion (PBD) via a hepaticojejunal or a cholecystojejunal Roux-en-Y with a cutaneous loop stoma, a cutaneous end cholecystojejunostomy or cholecystoappendicostomy, a partial internal biliary diversion via a jejunal conduit connecting the gallbladder with the colon, and an ileal bypass [5][6][7]. Several reports have suggested that children exhibit accelerated (catch-up) growth velocity and improved liver histology associated with reduced symptoms after PBD, thus, avoiding or delaying the need for liver transplantation [5,6].…”

mentioning

confidence: 99%

Long-term outcome after partial external biliary diversion for progressive familial intrahepatic cholestasis

Halaweish

Chwals

2010

Journal of Pediatric Surgery

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confidence: 99%

Long-term outcome after partial external biliary diversion for progressive familial intrahepatic cholestasis

Halaweish

Chwals

2010

Journal of Pediatric Surgery

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“…Previous reports [3,[5][6][7][9][10][11][12][13] have noted therapeutic effect from PEBD in patients with PFIC1. However, such benefits are not seen in all PFIC1 patients.…”

Section: Discussionmentioning

confidence: 99%

“…Immunostaining demonstrated expression of the bile salt export pump (BSEP) protein. A limited response of PEBD sometimes is attributed to advanced liver fibrosis already present before PEBD [11,14] . However, pre-existing fibrosis clearly could not be blamed for Patient 2's disappointing response.…”

Section: Discussionmentioning

confidence: 99%

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Clinical Course Following Partial External Biliary Diversion for Progressive Familial Intrahepatic Cholestasis 1: A Report of Two Patients

Kimura

Abukawa²,

Takei³

et al. 2016

Journal of Gastroenterology and Hepatology Research

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AIM:To better understand the effect of partial external biliary diversion (PEBD) in patients with progressive familial intrahepatic cholestasis 1 (PFIC1). MATERIALS AND METHODS:We analyzed quantitative changes in the concentrations of individual bile acids in serum, urine, and biliary bile, and concomitant changes in symptoms such as pruritus, in 2 patients whose PFIC1 clinically evolved to resemble the course of benign recurrent intrahepatic cholestasis (BRIC) upon treatment with PEBD. RESULTS: Patient 1 showed good therapeutic effect after PEBD including improved liver function test results and decreased total bile acids in serum, as well as decreased pruritus. The cholic acid (CA)/chenodeoxycholic acid (CDCA) ratio in biliary bile also decreased. Patient 2, however, experienced little decrease in pruritus after PEBD, and showed no clear change in the CA/CDCA ratio. Nonetheless, this patient's course did not rapidly worsen. CONCLUSION: In PFIC1, increased bile secretion and a decreased CA/CDCA ratio in biliary bile may be the most important responses after PEBD. Relief of pruritus is also an important sign of therapeutic effect. Moreover, progression of symptoms in PFIC1 sometimes can be delayed by PEBD, resulting in a relatively mild BRIC-like course, even when the CA/CDCA ratio and pruritus show little change. However, mechanisms underlying clinical benefits remain obscure. Abbreviations PFIC1: progressive familial intrahepatic cholestasis 1; GGT: γ-glutamyltransferase;

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“…This innovative technique was quickly adopted by other centres worldwide and so far sixteen additional casereports/series addressing the effect and technique of PEBD in the treatment of PFIC have been published (Table 3). Except for one small series of five patients in which PEBD did not have any effect [104], all others report normalization or improvement of liver function in 75-100% of the patients with low GGT-PFIC, indicated by at least improved liver tests and reduced pruritus [106,108,110,112,[136][137][138][139][140][141][142][143]. The response in patients with an undefined subtype of PFIC seems to be less [111,144].…”

Section: Biliary Diversionmentioning

confidence: 96%

Liver disease associated with canalicular transport defects: Current and future therapies

Stapelbroek

Erpecum

Klomp

et al. 2010

Journal of Hepatology

153

152

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Bile formation at the canalicular membrane is a delicate process. This is illustrated by inherited liver diseases due to mutations in ATP8B1, ABCB11, ABCB4, ABCC2 and ABCG5/8, all encoding hepatocanalicular transporters. Effective treatment of these canalicular transport defects is a clinical and scientific challenge that is still ongoing. Current evidence indicates that ursodeoxycholic acid (UDCA) can be effective in selected patients with PFIC3 (ABCB4 deficiency), while rifampicin reduces pruritus in patients with PFIC1 (ATP8B1 deficiency) and PFIC2 (ABCB11 deficiency), and might abort cholestatic episodes in BRIC (mild ATP8B1 or ABCB11 deficiency). Cholestyramine is essential in the treatment of sitosterolemia (ABCG5/8 deficiency). Most patients with PFIC1 and PFIC2 will benefit from partial biliary drainage. Nevertheless liver transplantation is needed in a substantial proportion of these patients, as it is in PFIC3 patients. New developments in the treatment of canalicular transport defects by using nuclear receptors as a target, enhancing the expression of the mutated transporter protein by employing chaperones, or by mutation specific therapy show substantial promise. This review will focus on the therapy that is currently available as well as on those developments that are likely to influence clinical practice in the near future.

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Partial External Biliary Diversion in Children With Progressive Familial Intrahepatic Cholestasis and Alagille Disease

Abstract: At median follow-up of 3.1 years after PEBD, pruritus has been relieved in 75%. Bile salts level and growth are improved in most patients. Longer follow-up is needed to determine whether PEBD can postpone or avoid the demand for liver transplantation.

Cited by 59 publications

References 17 publications

Long-term outcome after partial external biliary diversion for progressive familial intrahepatic cholestasis

Long-term outcome after partial external biliary diversion for progressive familial intrahepatic cholestasis

Clinical Course Following Partial External Biliary Diversion for Progressive Familial Intrahepatic Cholestasis 1: A Report of Two Patients

Liver disease associated with canalicular transport defects: Current and future therapies

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