title: Stem cell transplantation for paroxysmal nocturnal haemoglobinuria in childhood.
abstract: Paroxysmal nocturnal haemoglobinuria (PNH) is a clonal haematopoietic disorder characterized by chronic or intermittent intravascular haemolysis, variable cytopenia and an increased risk of thrombosis. Stem cell transplantation (SCT) is a curative therapeutic option, but its risks must be carefully weighed against the natural course of PNH. World-wide experience with SCT for PNH in the paediatric age group is scarce. We report on two adolescents suffering from PNH with life-threatening complications who were successfully transplanted from unrelated donors. Indications and techniques of SCT in childhood PNH are discussed and an overview of the literature is given.
authors.affiliation: Children's Hospital, University of Freiburg, Freiburg, Germany.
authors.forename: Christian
authors.initials: C
authors.lastname: Flotho
authors.forename: Brigitte
authors.initials: B
authors.lastname: Strahm
authors.forename: Udo
authors.initials: U
authors.lastname: Kontny
authors.forename: Ulrich
authors.initials: U
authors.lastname: Duffner
authors.forename: Anke M J
authors.initials: AM
authors.lastname: Peters
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Dupuis
authors.forename: Charlotte M
authors.initials: CM
authors.lastname: Niemeyer
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000741:Anemia, Aplastic / Q000150:complications / Q000601:surgery; D002648:Child; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006457:Hemoglobinuria, Paroxysmal / Q000150:complications / Q000601:surgery*; D006801:Humans; D007951:Leukemia, Myeloid / Q000150:complications / Q000601:surgery; D008297:Male; D018579:Patient Selection; D014184:Transplantation, Homologous; D016896:Treatment Outcome