Stem cell transplantation for paroxysmal nocturnal haemoglobinuria in childhood

Br J Haematol. 2002 Jul;118(1):124-7. doi: 10.1046/j.1365-2141.2002.03543.x.

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.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Acute Disease
  • Adolescent
  • Anemia, Aplastic / complications
  • Anemia, Aplastic / surgery
  • Child
  • Female
  • Hematopoietic Stem Cell Transplantation*
  • Hemoglobinuria, Paroxysmal / complications
  • Hemoglobinuria, Paroxysmal / surgery*
  • Humans
  • Leukemia, Myeloid / complications
  • Leukemia, Myeloid / surgery
  • Male
  • Patient Selection
  • Transplantation, Homologous
  • Treatment Outcome