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
{"title":"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","authors":"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","doi":"10.1002/jha2.1092","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Introduction</h3>\n \n <p>Erythropoietic Protoporphyria (EPP) caused skin light sensitivity and liver cirrhosis in a 35-year-old patient who subsequently developed liver-failure.</p>\n </section>\n \n <section>\n \n <h3> Methods</h3>\n \n <p>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).</p>\n </section>\n \n <section>\n \n <h3> Results</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> Conclusion</h3>\n \n <p>Haploidentical transplantation is a feasible treatment approach in EPP-patients.</p>\n </section>\n \n <section>\n \n <h3> Trial Registration</h3>\n \n <p>The authors have confirmed clinical trial registration is not needed for this submission</p>\n </section>\n </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2025-02-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.1092","citationCount":"0","resultStr":"{\"title\":\"Paternal Split-Liver Transplantation Followed by Haploidentical Hematopoietic Cell Transplantation in an Adult Patient With Protoporphyria-Induced Liver Failure\",\"authors\":\"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\",\"doi\":\"10.1002/jha2.1092\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div>\\n \\n \\n <section>\\n \\n <h3> Introduction</h3>\\n \\n <p>Erythropoietic Protoporphyria (EPP) caused skin light sensitivity and liver cirrhosis in a 35-year-old patient who subsequently developed liver-failure.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Methods</h3>\\n \\n <p>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).</p>\\n </section>\\n \\n <section>\\n \\n <h3> Results</h3>\\n \\n <p>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.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Conclusion</h3>\\n \\n <p>Haploidentical transplantation is a feasible treatment approach in EPP-patients.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Trial Registration</h3>\\n \\n <p>The authors have confirmed clinical trial registration is not needed for this submission</p>\\n </section>\\n </div>\",\"PeriodicalId\":72883,\"journal\":{\"name\":\"EJHaem\",\"volume\":\"6 1\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-02-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.1092\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"EJHaem\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1002/jha2.1092\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"EJHaem","FirstCategoryId":"1085","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/jha2.1092","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Paternal Split-Liver Transplantation Followed by Haploidentical Hematopoietic Cell Transplantation in an Adult Patient With Protoporphyria-Induced Liver Failure
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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