期刊
WORLD JOURNAL OF HEPATOLOGY
卷 13, 期 10, 页码 1351-1366出版社
BAISHIDENG PUBLISHING GROUP INC
DOI: 10.4254/wjh.v13.i10.1351
关键词
Pediatric metabolic liver disease; Liver transplantation; Liver based metabolic disorders; Inherited; Cell therapy; Gene therapy
Liver transplantation is still the gold standard treatment for end stage liver disease in children, improving both survival and quality of life. This review summarizes the clinical presentation, treatment, and indications for transplantation for LBMDs, as well as outlining new therapies and addressing dilemmas in the field.
Liver transplantation (LT) remains the gold standard treatment for end stage liver disease in the pediatric population. For liver based metabolic disorders (LBMDs), the decision for LT is predicated on a different set of paradigms. With improved outcomes post-transplantation, LT is no longer merely life saving, but has the potential to also significantly improve quality of life. This review summarizes the clinical presentation, medical treatment and indications for LT for some of the common LBMDs. We also provide a practical update on the dilemmas and controversies surrounding the indications for transplantation, surgical considerations and prognosis and long terms outcomes for pediatric LT in LBMDs. Important progress has been made in understanding these diseases in recent years and with that we outline some of the new therapies that have emerged.
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