Effect of Molecular Adsorbents Recirculating System Treatment in Children With Acute Liver Failure Caused by Wilson Disease

Rustom, Najla; Bost, Muriel; Cour-Andlauer, Fleur; Lachaux, Alain; Brunet, Anne-Sophie; Boillot, Olivier; Bordet, Fabienne; Valla, Frédéric V.; Richard, Nathalie; Javouhey, Étienne

doi:10.1097/mpg.0b013e3182a853a3

Cited by 37 publications

(28 citation statements)

References 21 publications

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“…It is also used as a ligand for therapy acting as adsorbent for treatment of patients with liver failure such as Wilson disease (Rustom et al . ). Over the past decades, albumin has emerged as a versatile carrier for therapeutic and diagnostic agents, primarily for diagnosing and treating diabetes, cancer, rheumatoid arthritis and infectious diseases.…”

Section: What Could Be the Consequences?mentioning

confidence: 97%

New insight in impaired binding capacity for albumin in uraemic patients

Stegmayr

2015

Acta Physiologica

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. 2015. Guanidinylations of albumin decreased binding capacity of hydrophobic metabolites. Acta Physiol 215, 13-23. New information presented by Rueth et al. in the current issue of Acta Physiologica extends our knowledge regarding decreased binding capacity of albumin of hydrophobic metabolites especially in uraemic patients. The authors were able to use recent developments in selectivity and sensitivity in mass spectrometry to identify and quantify endogenous posttranslational modifications of albumin. The technique enabled the authors to clarify that in uraemic patients, albumin has lower binding affinity due to conformational changes in an allosteric mechanism. A decreased binding capacity of albumin in patients suffering from chronic kidney injury has been described (Watanabe et al. 2012); however, the molecular reasons have been controversial. In the article by Rueth et al., the reason is suggested to be due to uraemic metabolites. Conformational changes can also be caused by post-translational modifications such as oxidation, glycosylation and carbamylation (Meijers et al. 2008).The study by Rueth et al. builds on the previous studies, and it showed that the alteration of albumin in CKD patients had a strong impact on total specific and unspecific binding. Thereby, the number of hydrophobic binding sites carrying indoxyl sulphate and especially tryptophan were significantly decreased.

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Section: What Could Be the Consequences?mentioning

confidence: 97%

New insight in impaired binding capacity for albumin in uraemic patients

Stegmayr

2015

Acta Physiologica

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“…Our PubMed search strategy identified 9 MEDLINE‐indexed reports on the outcomes of MARS therapy in a series of pediatric patients (Table ). Combined, these reports describe the outcomes of MARS therapy in a total of 44 children . Almost all of these patients (92%) suffered from ALF or ACLF, and only 4 patients (8%) underwent MARS treatment for GD.…”

Section: Resultsmentioning

confidence: 99%

“…Combined, these reports describe the outcomes of MARS therapy in a total of 44 children. 7,[30][31][32][33][34][35][36][37] Almost all of these patients (92%) suffered from ALF or ACLF, and only 4 patients (8%) underwent MARS treatment for GD. Recovery of the native liver was reported for 10 chil-dren with ALF (21%), including 4 patients with mushroom intoxication and 3 with an acetaminophen overdose.…”

Section: Comparison With Published Case Seriesmentioning

confidence: 99%

Experience with molecular adsorbent recirculating system treatment in 20 children listed for high‐urgency liver transplantation

et al. 2015

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For more than 10 years, children at our national center for pediatric liver transplantation (LT) have been treated with Molecular Adsorbent Recirculating System (MARS) liver dialysis as a bridging therapy to high-urgency LT. Treatment was reserved for 20 patients with the highest degrees of hepatic encephalopathy (HE; median grade 5 3.5). Death from neurological sequelae was considered imminent for these patients, and this was further reflected in significantly higher international normalized ratios and ammonia levels and worse prognostic liver indices (Model for End-Stage Liver Disease/Pediatric End-Stage Liver Disease scores and liver injury units) in comparison with 32 wait-listed patients who did not receive MARS dialysis. MARS therapy was generally well tolerated, with a reduction in thrombocytes and hemorrhaging as the most common side effects. HE improvement was documented in 30% of the treated patients, but progression to grade IV encephalopathy occurred in 45% of the patients despite the treatment. Serum ammonia, bilirubin, bile acid, and creatinine levels significantly decreased during treatment. Eighty percent of MARS-treated patients survived to undergo LT, and their survival was equivalent to that of non-MARStreated patients with severe liver failure (69%, P 5 0.52). The heterogeneity between MARS-treated patients and non-MARStreated patients in our cohort precluded a statistical evaluation of a benefit from MARS for patient survival. Our data demonstrate the safety of MARS even in the most severely ill patients awaiting LT, but strategies that promote the more rapid and widespread availability of high-quality donor organs remain of critical importance for improving patient survival in cases of severe acute liver failure. Liver Transpl 21:369-380, 2015. V C 2015 AASLD.

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“…Albeit some degree of improvement has been reported, the need for liver transplantation has not been obviated in numerous cases, although successful treatment without transplantation has been reported [131] . The molecular adsorbent recycling system ultrafiltration device, which combines ion exchange with albumin dialysis might provide some therapeutic efficacy in this setting, achieving copper removal and clinical stabilization, that might constitute a bridge for liver transplantation [132][133][134][135] .…”

Section: Fulminant Hepatic Failurementioning

confidence: 99%

Wilson’s disease: A review of what we have learned

Rodríguez–Castro

Hevia‐Urrutia

Sturniolo

2015

WJH

140

123

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Wilson's disease (WD), which results from the defective ATP7B protein product, is characterized by impaired copper metabolism and its clinical consequences vary from an asymptomatic state to fulminant hepatic failure, chronic liver disease with or without cirrhosis, neurological, and psychiatric manifestations. A high grade of suspicion is warranted to not miss cases of WD, especially less florid cases with only mild elevation of transaminases, or isolated neuropsychiatric involvement. Screening in first and second relatives of index cases is mandatory, and treatment must commence upon establishment of diagnosis. Treatment strategies include chelators such as D-penicillamine and trientine, while zinc salts act as inductors of methallothioneins, which favor a negative copper balance and a reduction of free plasmatic copper. As an orphan disease, research is lacking in this field, especially regarding therapeutic strategies which are associated with better patient compliance and which could eventually also reverse established injury.

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Effect of Molecular Adsorbents Recirculating System Treatment in Children With Acute Liver Failure Caused by Wilson Disease

Abstract: MARS is able to remove copper and to stabilise children with ALF secondary to WD, allowing bridging to LT.

Cited by 37 publications

References 21 publications

New insight in impaired binding capacity for albumin in uraemic patients

New insight in impaired binding capacity for albumin in uraemic patients

Experience with molecular adsorbent recirculating system treatment in 20 children listed for high‐urgency liver transplantation

Wilson’s disease: A review of what we have learned

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