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

EJHaem Pub Date : 2025-02-18 DOI:10.1002/jha2.1092

Ulrich Stölzel, Lasse Jost, Daniel Seehofer, Katharina Egger-Heidrich, Uwe Scheuermann, Kristina Hölig, Thomas Stauch, Desiree Kunadt, Detlef Schuppan, Johannes Schetelig, Nils Wohmann, Martin Bornhäuser, Friedrich Stölzel

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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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父亲分裂肝移植后单倍体造血细胞移植在原发性卟啉症肝衰竭成人患者中的应用

在一个35岁的患者中，红细胞生成性原生卟啉症（EPP）导致皮肤光敏和肝硬化，随后发展为肝衰竭。方法在没有人类白细胞抗原（HLA）匹配的无亲缘关系供体的情况下，父亲同意捐献肝移植（SLT）和异基因造血细胞移植（HCT）。结果在桥接治疗和SLT成功后，首次父本HCT导致移植物失败。对于第二次单倍体HCT，应用不同的方案导致植入，同时原卟啉（PP）血液水平降至正常，皮肤光敏性皮肤消失，导致无免疫抑制剂患者完全缓解。结论单倍体移植治疗epp是一种可行的治疗方法。试验注册作者已确认该提交不需要临床试验注册

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EJHaem

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