Successful treatment of congenital erythropoietic porphyria using matched unrelated hematopoietic stem cell transplantation in an adult: A case report

• Main Authors: Pierre Peterlin (Author), Julia Bonnelye (Author), Alice Garnier (Author), Amandine Le Bourgeois (Author), Thierry Guillaume (Author), Maxime Jullien (Author), Hervé Dutartre (Author), Marie Le Moigne (Author), Caroline Schmitt (Author), Laurent Gouya (Author), Antoine Poli (Author), Sebastien Barbarot (Author), Patrice Chevallier (Author)
• Format: Book
• Published: Wiley, 2024-04-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Congenital erythropoietic porphyria (CEP), or Gunther disease, is a rare genetic disease responsible for severe dermatologic, hepatic and/or haematological damages related to the deficient activity of the uroporphyrinogen III synthase. Allogeneic stem cell transplantation (Allo‐SCT) represe...