儿童暴发性肝豆状核变性：未移植行血浆置换后恢复。

Fulminant Wilson Disease in Children: Recovery After Plasma Exchange Without Transplantation.

机构信息

Department of Pediatrics.

Department of Hepatology of the Unversity Hospitals.

出版信息

J Pediatr Gastroenterol Nutr. 2020 Dec;71(6):720-725. doi: 10.1097/MPG.0000000000002894.

DOI:10.1097/MPG.0000000000002894

PMID:32804913

Abstract

OBJECTIVES

Since 2005, a New Wilson Index (NWI) ≥11 is used as a predictor of death without transplantation in fulminant Wilson disease (WD). Plasma exchange is advocated as a new treatment modality.

METHODS

We present a patient with fulminant WD treated with plasma exchange. All published cases applying plasma exchange for fulminant WD were reviewed systematically.

RESULTS

A 14-year-old girl presented with hemolysis and fulminant liver failure. She had no encephalopathy; NWI was 14. As a bridge to transplantation plasma exchange was started immediately. Complete remission was achieved with plasma exchange and later chelation therapy with D-penicillamine. She is now at 3-year transplant-free survival. Literature review identified 37 patients presenting with fulminant WD and NWI ≥11 who were treated with plasma exchange. Seventeen of these patients (ie, 46%) recovered without transplantation.

CONCLUSIONS

Multiple case reports and case series demonstrate transplant free survival after plasma exchange and subsequent chelation therapy, despite a NWI ≥11. Plasma exchange affects the clinical course and is a therapeutic option in children and young adults presenting with fulminant WD.

摘要

目的

自 2005 年以来，新的威尔逊指数（NWI）≥11 被用作暴发性肝豆状核变性（WD）患者无移植死亡的预测因子。提倡血浆置换作为一种新的治疗方式。

方法

我们介绍了一位接受血浆置换治疗的暴发性 WD 患者。系统回顾了所有应用血浆置换治疗暴发性 WD 的已发表病例。

结果

一名 14 岁女孩因溶血性贫血和暴发性肝功能衰竭就诊。她没有脑病；NWI 为 14。作为肝移植的桥梁，立即开始进行血浆置换。完全缓解是通过血浆置换和随后的 D-青霉胺螯合疗法实现的。目前，她无肝移植生存 3 年。文献复习发现，37 例 NWI≥11 的暴发性 WD 患者接受了血浆置换治疗。其中 17 例（46%）未经移植而康复。