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  • 04133 Paternal split-liver transplantation followed by haploidentical hematopoietic cell transplantation in an adult patient with protoporphyria-induced liver failure

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Poster 8
04133 Paternal split-liver transplantation followed by haploidentical hematopoietic cell transplantation in an adult patient with protoporphyria-induced liver failure
  1. Stölzel Ulrich1,
  2. Jost Lasse2,
  3. Daniel Seehofer3,
  4. Egger-Heidrich Katharina4,
  5. Scheuermann Uwe3,
  6. Hölig Kristina4,
  7. Stauch Thomas5,
  8. Kunadt Desiree4,
  9. Detlef Schuppan6,
  10. Schetelig Johannes4,
  11. Wohmann Nils1,
  12. Bornhäuser Martin4,
  13. Stölzel Friedrich2,4
  1. 1Department of Internal Medicine II and Porphyria Center, Klinikum Chemnitz, Chemnitz, Germany
  2. 2Division of Stem Cell Transplantation and Cellular Immunotherapies, Department of Internal Medicine II, University Hospital Schleswig Holstein Kiel, Kiel University, Germany
  3. 3Department of Visceral, Transplantation, Vascular and Thoracic Surgery, University Hospital of Leipzig, Leipzig, Germany
  4. 4Department of Internal Medicine I, University Hospital Carl Gustav Carus, TU Dresden, Dresden, Germany
  5. 5Deutsches Kompetenz-Zentrum für Porphyriediagnostik und Konsultation, MVZ Labor Volkmann und Kollegen GbR, Karlsruhe, Germany
  6. 6Institute of Translational Immunology, Johannes Gutenberg University Mainz, Mainz, Germany

Abstract

Erythropoietic Protoporphyria (EPP) caused lifelong grave light sensitivity on skin and induced liver cirrhosis with liver-failure in a 35-year-old patient. Since an unrelated donor search for hematopoietic cell transplantation (HCT) was not successful, the haploidentical and otherwise healthy father was identified as an alternative stem cell donor. However, during the Covid-19 pandemic HCT was postponed and the patient developed severe liver dysfunction and subsequent liver-failure. The 60-year-old father consented in donating split liver and hematopoietic stem cells and was cleared medically for donating both. Repeated plasma- and erythrocyte-apheresis were used to diminish toxic protoporphyrin IX (PP) and as a bridging strategy to paternal split-liver transplantation (SLT). Six months after SLT and with improved liver function, a first paternal haploidentical bone marrow transplantation resulted in non-donor-specific-antibody-associated primary graft failure (PGF) with subsequent autologous recovery. For a second successful paternal haploidentical peripheral-blood HCT, hydroxyurea was implemented and a more immunosuppressive total-body irradiation-based conditioning regimen was applied.

After haploidentical HCT, PP levels decreased to normal blood concentrations and light sensitivity of the skin disappeared.

The results presented herein describe the successful treatment of a patient with EPP causing lifelong grave light sensitivity on skin and induced liver cirrhosis with life-threatening complications.

This is a) the first patient reported with EPP who underwent haploidentical HCT using post-transplant cyclophosphamide as immunosuppressive backbone, and b) the first adult patient with EPP in general who has been treated with this subsequent SLT-HCT regimen.

The report might be of high interest for clinicians and currently discussed guidelines for management of protoporphyria-induced liver failure.

Abstract 04133 Figure 1
Abstract 04133 Figure 1
http://creativecommons.org/licenses/by-nc/4.0/

This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.

https://doi.org/10.1136/bmjgast-2024-ICPP.39

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