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

• Tác giả: Martin Bornhäuser, Katharina Egger-Heidrich, Kristina Hölig, Lasse Jost, Desiree Kunadt, Johannes Schetelig, Uwe Scheuermann, Detlef Schuppan, Daniel Seehofer, Thomas Stauch, Friedrich Stölzel, Ulrich Stölzel, Nils Wohmann
• Ngôn ngữ: eng
• Thông tin xuất bản: United States : EJHaem , 2025
• Bộ sưu tập: NCBI
• ID: 209833

Tóm tắt

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.