Long‐term outcomes after cholecystocolostomy for progressive familial intrahepatic cholestasis

Chen, Long; Xiao, Hui; Ren, Xiaobin; Li, Long

doi:10.1111/hepr.13222

Cited by 2 publications

(1 citation statement)

References 35 publications

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“…Recently, external fistulae have been intubated with gastrostomy devices for intermittent drainage[ 49 ]. IE[ 50 , 51 ] and PIBD[ 52 , 53 ] are therapeutic options in those with failed PEBD or for better cosmetic results, especially in adolescents. Recent studies on SBD with their outcome and complications have been tabulated in Table 3 [ 51 - 55 ].…”

Section: Therapeutic Strategies For Pficmentioning

confidence: 99%

Recent updates on progressive familial intrahepatic cholestasis types 1, 2 and 3: Outcome and therapeutic strategies

Alam¹,

Lal²

2022

WJH

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Recent evidence points towards the role of genotype to understand the phenotype, predict the natural course and long term outcome of patients with progressive familial intrahepatic cholestasis (PFIC). Expanded role of the heterozygous transporter defects presenting late needs to be suspected and identified. Treatment of pruritus, nutritional rehabilitation, prevention of fibrosis progression and liver transplantation (LT) in those with end stage liver disease form the crux of the treatment. LT in PFIC has its own unique issues like high rates of intractable diarrhoea, growth failure; steatohepatitis and graft failure in PFIC1 and antibody-mediated bile salt export pump deficiency in PFIC2. Drugs inhibiting apical sodium-dependent bile transporter and adenovirus-associated vector mediated gene therapy hold promise for future.

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Section: Therapeutic Strategies For Pficmentioning

confidence: 99%

Recent updates on progressive familial intrahepatic cholestasis types 1, 2 and 3: Outcome and therapeutic strategies

Alam¹,

Lal²

2022

WJH

View full text Add to dashboard Cite

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Epidemiology and burden of progressive familial intrahepatic cholestasis: a systematic review

Jones-Hughes¹,

Campbell²,

Crathorne³

2021

Orphanet J Rare Dis

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Background Progressive familial intrahepatic cholestasis is a rare, heterogeneous group of liver disorders of autosomal recessive inheritance, characterised by an early onset of cholestasis with pruritus and malabsorption, which rapidly progresses, eventually culminating in liver failure. For children and their parents, PFIC is an extremely distressing disease. Significant pruritus can lead to severe cutaneous mutilation and may affect many activities of daily living through loss of sleep, irritability, poor attention, and impaired school performance. Methods Databases including MEDLINE and Embase were searched for publications on PFIC prevalence, incidence or natural history, and the economic burden or health-related quality of life of patients with PFIC. Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines were followed. Results Three systematic reviews and twenty-two studies were eligible for inclusion for the epidemiology of PFIC including a total of 2603 patients. Study periods ranged from 3 to 33 years. Local population prevalence of PFIC was reported in three studies, ranging from 9.0 to 12.0% of children admitted with cholestasis, acute liver failure, or splenomegaly. The most detailed data come from the NAPPED study where native liver survival of >15 years is predicted in PFIC2 patients with a serum bile acid concentration below 102 µmol/L following bile diversion surgery. Burden of disease was mainly reported through health-related quality of life (HRQL), rates of surgery and survival. Rates of biliary diversion and liver transplant varied widely depending on study period, sample size and PFIC type, with many patients have multiple surgeries and progressing to liver transplant. This renders data unsuitable for comparison. Conclusion Using robust and transparent methods, this systematic review summarises our current knowledge of PFIC. The epidemiological overview is highly mixed and dependent on presentation and PFIC subtype. Only two studies reported HRQL and mortality results were variable across different subtypes. Lack of data and extensive heterogeneity severely limit understanding across this disease area, particularly variation around and within subtypes.

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Long‐term outcomes after cholecystocolostomy for progressive familial intrahepatic cholestasis

Abstract: This is the first long-term, large-scale analysis of cholecystocolostomy approaches for PFIC. Approaches single and well tolerated, and generally result in improvement of pruritus and cholestasis.

Cited by 2 publications

References 35 publications

Recent updates on progressive familial intrahepatic cholestasis types 1, 2 and 3: Outcome and therapeutic strategies

Recent updates on progressive familial intrahepatic cholestasis types 1, 2 and 3: Outcome and therapeutic strategies

Epidemiology and burden of progressive familial intrahepatic cholestasis: a systematic review

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