The natural history of primary sclerosing cholangitis in 781 children: A multicenter, international collaboration

Deneau, Mark; El‐Matary, Wael; Valentino, Pamela L.; Abdou, Reham; Alqoaer, Khaled; Amin, Mansi; Amir, Achiya; Auth, Marcus; Bazerbachi, Fateh; Broderick, Annemarie; Chan, Albert C. Y.; Cotter, Jillian M.; Doan, Sylvia; El–Youssef, Mounif; Ferrari, Federica; Furuya, Katryn N.; Gottrand, Madeleine; Gottrand, F.; Gupta, Nitika; Homan, Matjaž; Kamath, Binita M.; Kim, Kyung Mo; Kolho, Kaija‐Leena; Konidari, Anastasia; Koot, Bart; Iorio, Raffaele; Ledder, Oren; Mack, Cara L.; Martínez, Mercedes; Miloh, Tamir; Mohan, Parvathi; O’Cathain, Niamh; Papadopoulou, Alexandra; Ricciuto, Amanda; Saubermann, Lawrence J.; Sathya, P; Shteyer, Eyal; Smolka, Vratislav; Tanaka, Ayaka; Varier, Raghu; Venkat, Veena; Vitola, Bernadette; Vos, Miriam B.; Woynarowski, Marek; Yap, Jason; Jensen, M. Kyle

doi:10.1002/hep.29204

Cited by 162 publications

(221 citation statements)

References 39 publications

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“…All retrospective studies recognize a form of SC with strong autoimmune features, but while in some this condition is reported to respond favourably to immunosuppression, having a better prognosis than PSC, in others the prognosis of AIH/PSC overlap is reported to be severe and not ameliorated by immunosuppressive treatment or similar to that of PSC irrespective of treatment . In agreement with the current study by Deneau et al, small‐duct SC is reported to have a more favorable prognosis …”

supporting

confidence: 91%

“…In this issue of H epatology , Deneau et al report on the 5‐ and 10‐year outcome of an impressive number of children diagnosed with primary sclerosing cholangitis (PSC) recruited in 36 transplant and nontransplant referral centers from Europe, North America, the Middle East, and Asia . The study shows the importance of multicenter collaboration and data collection in dedicated registries.…”

mentioning

confidence: 99%

“…As shown by the prospective study, often cited negatively to support a worse prognosis for ASC compared to AIH, immunosuppressive treatment is effective in controlling both parenchymal and biliary disease in 50% of ASC cases, suggesting that the real prognosis of ASC compared to PSC cannot be adequately established in retrospective cohorts with variable diagnostic approaches and treatment protocols. Thus, in the series by Deneau et al, it would be interesting to know to which immunosuppressive drugs the PSC+IBD patients, who have a lower proportion of AIH overlap compared to PSC with no IBD, had been exposed before the hepatic presentation, given that immunosuppression might have modified their disease phenotype. It would be also interesting to search for possible exposure to vancomycin treatment, recently reported to have a beneficial effect in patients with PSC and IBD …”

mentioning

confidence: 99%

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The Riddle of Juvenile Sclerosing Cholangitis

Mieli‐Vergani

Vergani

2017

Hepatology

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supporting

confidence: 91%

mentioning

confidence: 99%

mentioning

confidence: 99%

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The Riddle of Juvenile Sclerosing Cholangitis

Mieli‐Vergani

Vergani

2017

Hepatology

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“…To meet the knowledge gap in pediatric PSC, a multicenter data collection on PSC patients from over 36 pediatric centers worldwide was initiated. Results show that elevations in bilirubin, gamma‐glutamyltransferase (gGT), and aspartate aminotransferase‐to‐platelet ratio index at diagnosis can identify patients at highest risk; small‐duct PSC and concurrent IBD are more favorable disease phenotypes . Clinical trials in pediatric patients with PSC may need innovative trial designs.…”

Section: Scope Of the Problemmentioning

confidence: 99%

Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis

et al. 2018

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Primary sclerosing cholangitis (PSC) is a rare and chronic liver disease for which there is no effective therapy. Interest has grown in developing treatments for this condition, with several agents proposed as potential therapies. However, there is a lack of clarity about how to measure clinical benefit in trials involving patients with this complex and rare disease. This article reviews regulatory information, the available literature on natural history, as well as potential candidate clinical and surrogate endpoints for PSC. (Hepatology 2018; 00:000-000).

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“…It is suggested by several authors that paediatric‐onset SC runs a milder course and has a more favourable outcome compared to adult‐onset PSC . The time horizon in these papers could have been restricted by transfer of patients to adult‐oriented care.…”

Section: Introductionmentioning

confidence: 99%

Disease progression in paediatric‐ and adult‐onset sclerosing cholangitis: Results from two independent Dutch registries

Joosse

Haisma

Sterk

et al. 2019

Liver International

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Background & Aims Sclerosing cholangitis (SC) is a severe liver disease leading to destruction of bile ducts. It is believed to run a milder course in children than in adults. To test this assumption, we evaluated time‐to‐complication curves in two independent paediatric‐onset cohorts from the same geographical area. Methods Short‐term disease outcomes were evaluated with an online clinical registry that was filled with data on children with SC diagnosed between 2000 and 2017 and who were followed bi‐annually thereafter. Long‐term disease outcomes were evaluated in a paediatric‐onset subcohort derived from a previously published population‐based study from the Netherlands. Time‐to‐complication in the first cohort was defined as the time from diagnosis until portal hypertension, biliary obstructions and infections, development of malignancy, or liver transplantation, whichever came first. In the second cohort time‐to‐complication was defined as the time until liver transplantation or PSC‐related death. Results Median age at diagnosis in the first cohort (n = 86) was 12.3 years. In the first 5 years post‐diagnosis 23% of patients developed complications. The patients in the population‐based study (n = 683) were stratified into those diagnosed before the age of 18 years (‘paediatric‐onset’ subcohort, n = 43) and those diagnosed after the age of 18 years (‘adult‐onset’ subcohort, n = 640). Median age at diagnosis was 14.6 and 40.2 years, respectively. Median time‐to‐complication in the paediatric‐onset and adult‐onset subcohorts was not statistically different. Conclusion Paediatric and adult‐onset SC run a similar long‐term disease course. Paediatricians who treat children with SC should monitor them closely to recognize early complications and control long‐term sequelae.

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The natural history of primary sclerosing cholangitis in 781 children: A multicenter, international collaboration

Cited by 162 publications

References 39 publications

The Riddle of Juvenile Sclerosing Cholangitis

The Riddle of Juvenile Sclerosing Cholangitis

Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis

Disease progression in paediatric‐ and adult‐onset sclerosing cholangitis: Results from two independent Dutch registries

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