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Longterm Outcomes of Living Donor Liver Transplantation for Glycogen Storage Disease Type 1b
Author(s) -
Shimizu Seiichi,
Sakamoto Seisuke,
Horikawa Reiko,
Fukuda Akinari,
Uchida Hajime,
Takeda Masahiro,
Yanagi Yusuke,
Irie Rie,
Yoshioka Takako,
Kasahara Mureo
Publication year - 2020
Publication title -
liver transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.814
H-Index - 150
eISSN - 1527-6473
pISSN - 1527-6465
DOI - 10.1002/lt.25649
Subject(s) - medicine , glycogen storage disease , liver transplantation , glycogen storage disease type i , living donor liver transplantation , transplantation , liver disease , glycogen
Glycogen storage disease (GSD) type 1b (Online Mendelian Inheritance in Man [OMIM] 232220) is an autosomal recessive inborn error of carbohydrate metabolism caused by defects in glucose‐6‐phosphate translocase. GSD1b patients have severe hypoglycemia with several clinical manifestations of hepatomegaly, obesity, a doll‐like face, and neutropenia. Liver transplantation (LT) has been indicated for severe glucose intolerance, poor metabolic control (PMC), and poor growth (PG). We retrospectively reviewed 11 children with GSD1b who underwent living donor liver transplantation (LDLT) at the National Center for Child Health and Development in Tokyo, Japan. Between November 2005 and December 2018, 495 children underwent LDLT with an overall 10‐year patient and graft survival of 90.6% and 88.9%, respectively. Of these, LT was indicated for 11 patients with GSD1b. All patients are doing well with the stabilization of glucose intolerance and decreased hospitalization for infectious complications. Demand for granulocyte colony‐stimulating factor significantly decreased. However, although LT stabilized the blood glucose level, the platelet function was not improved. The posttransplant developmental quotient (DQ) remained similar to the pretransplant DQ without deterioration. LDLT is a feasible procedure for GSD1b patients with regard to the longterm prognosis. LT should be considered for patients with severe glucose intolerance to protect the cognitive function against hypoglycemic encephalopathy and to ameliorate PMC and PG.