Stem cell transplantation for paroxysmal nocturnal haemoglobinuria in childhood |
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Authors: | Flotho Christian Strahm Brigitte Kontny Udo Duffner Ulrich Peters Anke M J Dupuis Wolfgang Niemeyer Charlotte M |
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Affiliation: | Children's Hospital, University of Freiburg, Freiburg, Germany. |
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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. |
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Keywords: | paroxysmal nocturnal haemoglobinuria trans-plantation PNH SCT BMT |
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