PS-195-Predicting long-term outcome after surgical biliary diversion in Bsep-deficiency patients: Results from the NAPPED consortium

Wessel, Daan van; Thompson, Richard; Grammatikopoulos, Tassos; Kadaristiana, Agustina; Jankowska, Irena; Lipineski, Patryk; Czubkowski, Piotr; Gonzalès, Emmanuel; Jacquemin, Emmanuel; Spraul, Anne; Sokal, Étienne; Shagrani, Mohammad; Bröering, Dieter C.; Algoufi, Talal; Mazhar, Nejat; Nicastro, Emanuele; Kelly, Déirdre; Nebbia, Gabriella; Arnell, Henrik; Fischler, Björn; Hulscher, J. B. F.; Serranti, Daniele; Arıkan, Çiğdem; Polat, Esra; Debray, Dominique; Lacaille, Florence; Gonçalves, Ana Cristina; Bartolo, Gema Muñoz; Mozer‐Glassberg, Yael; Brecelj, Jernej; Dezsőfi, Antal; Calvo, Pier Luigi; Grabhorn, Enke; Sturm, Ekkehard; Woerd, Wendy Van der; Kamath, Binita M.; Wang, Jianshe; Liting, null Li; Durmaz, Özlem; Hansen, Bettina E.; Verkade, Henkjan J.

doi:10.1016/s0618-8278(19)30213-0

Cited by 4 publications

(3 citation statements)

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“…This review, however, adds insight to PFIC and BRIC disease progression, following on from Baker et al 2019 [ 5 ] in particular, where selected treatments and/or surgery may be more suitable to one form of PFIC and at a specific point in the progression of the disease over another. In addition, this review includes a detailed summary of the NAPPED study, which continues to add crucial data to aid our understanding [ 14 , 15 , 18 , 19 , 55 , 60 ].…”

Section: Discussionmentioning

confidence: 99%

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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Section: Discussionmentioning

confidence: 99%

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

Jones-Hughes¹,

Campbell²,

Crathorne³

2021

Orphanet J Rare Dis

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“…Mean improvements in serum bile acids and pruritus scores with odevixibat were generally accompanied by mean decreases in hepatic laboratory values that are commonly associated with paediatric cholestasis. 20 End-stage liver disease resulting from prolonged cholestasis, or pruritus unresponsive to medical therapy or surgical diversion, are indications for liver transplantation in patients with PFIC, 4 , 21 , 22 and preclinical evidence suggests IBAT inhibition may reduce hepatic inflammation or fibrosis. 19 , 23 , 24 …”

Section: Discussionmentioning

confidence: 99%

Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis

Artan

Baumann

et al. 2023

JHEP Reports

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“…A recent retrospective follow-up study from the NAPPED (Natural Course and Prognosis of PFIC and Effect of Biliary Diversion) consortium found that native liver survival was significantly higher in BSEP-deficient patients who had undergone biliary diversion surgery than in those who had not (29), and a significant association was identified between lower postsurgical serum bile acids and long-term native liver survival (30).…”

Section: Discussionmentioning

confidence: 99%

Systematic Review and Meta‐analysis

Verkade¹,

Thompson

Arnell

et al. 2020

J. pediatr. gastroenterol. nutr.

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Objectives: We assessed available data on impact of partial external biliary diversion (PEBD) surgery on clinical outcomes in patients with progressive familial intrahepatic cholestasis (PFIC). Methods: We performed a systematic literature review (PubMed) and metaanalysis to evaluate relationships between liver biochemistry parameters (serum bile acids, bilirubin, and alanine aminotransferase [ALT]) and early response (pruritus improvement) or long-term outcomes (need for liver transplant) in patients with PFIC who underwent PEBD. Results: Searches identified 175 publications before September 2018; 16 met inclusion criteria. Receiver operating characteristic (ROC) analysis examined ability of liver biochemistry parameters to discriminate patients who demonstrated early and long-term response to PEBD from those who did not. Regarding pruritus improvement in 155 included patients in aggregate, 104 (67%) were responders, 14 (9%) had partial response, and 37 (24%) were nonresponders. In ROC analyses of individual patient data, post-PEBD serum concentration of bile acids, in particular, could discriminate responders from nonresponders for pruritus improvement (area under the curve, 0.99; P < 0.0001; n ¼ 42); to a lesser extent, this was also true for bilirubin (0.87; P ¼ 0.003; n ¼ 31), whereas ALT could not discriminate responders from nonresponders for pruritus improvement (0.74; P ¼ 0.06; n ¼ 28). Reductions from pre-PEBD values in serum bile acid concentration (0.89; P ¼ 0.0003; n ¼ 32) and bilirubin (0.98; P ¼ 0.002; n ¼ 18) but not ALT (0.62; P ¼ 0.46; n ¼ 18) significantly discriminated decreased aggregate need for liver transplant. Conclusion: Changes in bile acids seem particularly useful in discriminating early and long-term post-PEBD outcomes and may be potential biomarkers of response to interruption of enterohepatic circulation in patients with PFIC.

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PS-195-Predicting long-term outcome after surgical biliary diversion in Bsep-deficiency patients: Results from the NAPPED consortium

Cited by 4 publications

References 0 publications

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

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

Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis

Systematic Review and Meta‐analysis

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