Premium
Stem cell transplantation for paroxysmal nocturnal haemoglobinuria in childhood
Author(s) -
Flotho Christian,
Strahm Brigitte,
Kontny Udo,
Duffner Ulrich,
Peters Anke M. J.,
Dupuis Wolfgang,
Niemeyer Charlotte M.
Publication year - 2002
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.2002.03543.x
Subject(s) - medicine , cytopenia , haemolysis , transplantation , stem cell , pediatrics , thrombosis , hematopoietic stem cell transplantation , immunology , bone marrow , biology , genetics
Summary. 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.