Living Donor Liver Transplantation as Salvage Therapy for Steroid‐Refractory Chronic Hepatic <scp>GVHD</scp> Following Allogeneic Hematopoietic Stem Cell Transplantation

BACKGROUND: Hepatic graft-versus-host disease (GVHD) is a rare but life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT). Steroid-refractory hepatic GVHD carries a high risk of mortality with limited salvage options. Liver transplantation after HSCT poses unique challenges due to dual alloimmunity and is rarely pursued, particularly in pediatric patients. We report a pediatric patient with steroid-refractory chronic hepatic GVHD successfully treated with living donor liver transplantation (LDLT) from a parental donor. CASE PRESENTATION: A 13-year-old male underwent allogeneic HSCT for idiopathic severe aplastic anemia and developed progressive chronic hepatic GVHD characterized by ductopenia and severe cholestasis. His disease was refractory to corticosteroids and multiple additional therapies, including ruxolitinib, mesenchymal stromal cells, infliximab, sirolimus, anti-thymocyte globulin, alpha-1 antitrypsin, cyclophosphamide, and emapalumab. His course was complicated by Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disorder (PTLD), successfully treated with rituximab. Fifteen months after HSCT, and seven months after clearance of EBV viremia, he underwent LDLT from a parental donor. Donor selection preserved the option for future donor-derived EBV-specific cytotoxic T lymphocyte therapy if needed. Eleven months following LDLT, the patient remains clinically well, with normal liver function, sustained full donor chimerism, and no evidence of recurrent GVHD or PTLD. CONCLUSION: LDLT may represent a viable option in select pediatric patients with irreversible chronic hepatic GVHD. This case underscores the importance of early multidisciplinary planning and highlights the absence of liver-specific biomarkers or targeted therapies to guide management. Dedicated research into preventive and regenerative strategies is warranted.

Authors

Institutions

Publication Details

Journal
Pediatric Transplantation
Published
2026-07-30
DOI
https://doi.org/10.1111/petr.70415
Primary Topic
Hematopoietic Stem Cell Transplantation
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Living Donor Liver Transplantation as Salvage Therapy for Steroid‐Refractory Chronic Hepatic GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation

Steven Lobritto, Urvi Kapoor, Monica Bhatia
Pediatric Transplantation
Hematopoietic Stem Cell Transplantation
article

Living Donor Liver Transplantation as Salvage Therapy for Steroid‐Refractory Chronic Hepatic GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation

Steven Lobritto, Urvi Kapoor, Monica Bhatia
article en

Abstract

BACKGROUND: Hepatic graft-versus-host disease (GVHD) is a rare but life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT). Steroid-refractory hepatic GVHD carries a high risk of mortality with limited salvage options. Liver transplantation after HSCT poses unique challenges due to dual alloimmunity and is rarely pursued, particularly in pediatric patients. We report a pediatric patient with steroid-refractory chronic hepatic GVHD successfully treated with living donor liver transplantation (LDLT) from a parental donor. CASE PRESENTATION: A 13-year-old male underwent allogeneic HSCT for idiopathic severe aplastic anemia and developed progressive chronic hepatic GVHD characterized by ductopenia and severe cholestasis. His disease was refractory to corticosteroids and multiple additional therapies, including ruxolitinib, mesenchymal stromal cells, infliximab, sirolimus, anti-thymocyte globulin, alpha-1 antitrypsin, cyclophosphamide, and emapalumab. His course was complicated by Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disorder (PTLD), successfully treated with rituximab. Fifteen months after HSCT, and seven months after clearance of EBV viremia, he underwent LDLT from a parental donor. Donor selection preserved the option for future donor-derived EBV-specific cytotoxic T lymphocyte therapy if needed. Eleven months following LDLT, the patient remains clinically well, with normal liver function, sustained full donor chimerism, and no evidence of recurrent GVHD or PTLD. CONCLUSION: LDLT may represent a viable option in select pediatric patients with irreversible chronic hepatic GVHD. This case underscores the importance of early multidisciplinary planning and highlights the absence of liver-specific biomarkers or targeted therapies to guide management. Dedicated research into preventive and regenerative strategies is warranted.

Pediatric TransplantationVol. 30(8)
Columbia University Irving Medical Center (US)
Good health and well-being
Openalex Percentile: Top 9%
Hematopoietic Stem Cell Transplantation
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.