Biopsy‐Proven Reversal of F4 Cirrhosis in Classic Hepatic Glycogen Storage Disease Type IV: A 42‐Year Follow‐Up Without Transplantation

This unprecedented case demonstrates that biochemical and histological remission is possible in classic hepatic GSD-IV despite persistently deficient branching-enzyme activity. Early, aggressive management of portal hypertension may therefore permit long-term survival and full histological recovery, challenging the transplant-first paradigm and supporting individualized, transplant-sparing care.

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Publication Details

Journal
Hepatology Research
Published
2025-12-22
DOI
https://doi.org/10.1111/hepr.70084
Primary Topic
Glycogen Storage Diseases and Myoclonus
Type
article
Field-Weighted Citation Impact
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article

Biopsy‐Proven Reversal of F4 Cirrhosis in Classic Hepatic Glycogen Storage Disease Type IV: A 42‐Year Follow‐Up Without Transplantation

Masaaki Mino, Masataka Tsuge, Taiji Yamazoe, D. Miki et al.
Hepatology Research
Glycogen Storage Diseases and Myoclonus
article

Biopsy‐Proven Reversal of F4 Cirrhosis in Classic Hepatic Glycogen Storage Disease Type IV: A 42‐Year Follow‐Up Without Transplantation

Masaaki Mino, Masataka Tsuge, Taiji Yamazoe, D. Miki, Nami Mori, Shiro Oka, Yumi Shimomura, Shintaro Takaki, Kazuko Hamamoto, Tokiko Fukuda, Hideo Sugie, Keiji Tsuji
article en

Abstract

This unprecedented case demonstrates that biochemical and histological remission is possible in classic hepatic GSD-IV despite persistently deficient branching-enzyme activity. Early, aggressive management of portal hypertension may therefore permit long-term survival and full histological recovery, challenging the transplant-first paradigm and supporting individualized, transplant-sparing care.

Hepatology Research
Hiroshima University (JP), Tokoha Gakuen University (JP), Hamamatsu University School of Medicine (JP), National Center for Global Health and Medicine (JP), Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital (JP)
Good health and well-being
Openalex Percentile: Top 29%
Glycogen Storage Diseases and Myoclonus
DOIOpen Access PDFOpenAlex
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Biopsy‐Proven Reversal of F4 Cirrhosis in Classic Hepatic Glycogen Storage Disease Type IV: A 42‐Year Follow‐Up Without Transplantation — Masaaki Mino, Masataka Tsuge, et al. · Hepatology Research (2025) | TGRS Research Map | TGRS