Clinical presentation and outcome of Wilson’s disease patients in a monocentric cohort of liver reference center

Sobesky, Rodolphe; Darce, M.; Agostini, Hélène; Fernandez, I.; Usardi, Alessia; Adam, R D; Cherqui, Daniel; Gonzalès, Emmanuel; Jacquemin, E.; Samuel, Douglas B.; Poujois, Aurélia; Woimant, F.; Duclos-Vallée, J.-C.

doi:10.1016/s0168-8278(18)31516-2

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“…A pediatric patient with WD could present-as the very first evidence of WD-with severely decompensated cirrhosis, displaying marked intractable coagulopathy but no HE, thus fulfilling the PALF criteria. These patients may respond to intensive medical treatment and avoid LT (22)(23)(24)(25). Such may have been the case with one of the excluded articles which employed the PALF definition and reported an extremely high rate of spontaneous recovery (14).…”

Section: Discussionmentioning

confidence: 99%

Pediatric Wilson Disease Presenting as Acute Liver Failure

Vandriel

Ayoub

Ricciuto

et al. 2020

J. pediatr. gastroenterol. nutr.

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Objectives: Wilson disease (WD) presenting as acute liver failure (ALF) is rare and typically fatal without liver transplantation (LT). Its rarity has hindered comprehensive studies. We undertook an individual patient data meta-analysis to characterize a cohort of pediatric patients presenting with ALF whose final diagnosis was WD to examine outcomes and identify predictors of poor outcomes. Methods: Database searches were conducted in PubMed, ScienceDirect, and Google Scholar, restricted to English-language articles published between January 1984 and May 2018. Articles were excluded if pediatric (<18 years old) data were not extractable or if LT was not readily available at reporting institutions. Extracted data included clinical and biochemical characteristics, genotype, treatment, and outcome. Results: Data were available on 249 subjects from 52 articles, plus 7 additional subjects identified from our institution's WD database (N = 256). Females represented 69% (n = 170/245). Median age at presentation was 13.4 years (n = 204, range 4.0–17.9). Of the total 256 subjects, 87% underwent LT, 11% achieved spontaneous recovery and 2% died before LT. International normalized ratio >2.0 at presentation was a predictor of LT/death (odds ratio 7.6, 95% confidence interval 1.5–28), with a trend observed for hepatic encephalopathy (HE) (odds ratio 4.18, 95% confidence interval 0.99–18). Arithmetic diagnostic scores proved inferior in the pediatric age-bracket compared to adults. Conclusions: This large international pediatric cohort has permitted an individual patient data analysis of WD presenting as ALF. Notably, 11% of subjects achieved spontaneous survival; the rest required LT. Coagulopathy (international normalized ratio >2:0) and HE at presentation heralded poor outcomes. Further prospective studies may identify additional early predictors of outcomes.

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