Article
Hematopoietic cell transplantation from related and unrelated donors after minimal conditioning as a curative treatment modality for severe paroxysmal nocturnal hemoglobinuria.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation - 1 Nov 2003
Hegenbart U, Niederwieser D, Forman S, Holler E, Leiblein S, Johnston L, Pönisch W, Epner E, Witherspoon R, Blume K, Storb R
Abstract excerpt
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal disorder caused by a somatic mutation of the X-linked phosphatidylinositol glycan class A gene. Allogeneic hematopoietic cell transplantation (HCT) after high-dose conditioning is the only curative treatment; however, it is associated with high treatment-related mortality. Here, we report on allogeneic HCT for PNH after minimal conditioning. Seven adult...
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