Paternal Split-Liver Transplantation Followed by Haploidentical Hematopoietic Cell Transplantation in an Adult Patient With Protoporphyria-Induced Liver Failure

Overview

Journal EJHaem

Specialty Hematology

Date 2025 Feb 19

PMID 39967799

Authors

Ulrich Stolzel

Lasse Jost

Daniel Seehofer

Katharina Egger-Heidrich

Uwe Scheuermann

Kristina Holig

Thomas Stauch

Desiree Kunadt

Detlef Schuppan

Johannes Schetelig

Nils Wohmann

Martin Bornhauser

Friedrich Stolzel

Affiliations

Soon will be listed here.

Abstract

Introduction: Erythropoietic Protoporphyria (EPP) caused skin light sensitivity and liver cirrhosis in a 35-year-old patient who subsequently developed liver-failure.

Methods: In absence of a human leukocyte antigens (HLA)-matched-unrelated donor, the father consented in donating for split liver transplantation (SLT) and allogeneic hematopoietic cell transplantation (HCT).

Results: After bridging therapy and successful SLT a first paternal HCT resulted in graft failure. For a second haploidentical HCT a different regimen was applied leading to engraftment while protoporphyrin (PP) blood-levels decreased to normal and skin light sensitivity skin disappeared, leading to complete remission in an immunosuppressive-free patient.

Conclusion: Haploidentical transplantation is a feasible treatment approach in EPP-patients.

Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.

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