title: Allogeneic CD34-enriched peripheral blood stem cell transplantation in a patient with paroxysmal nocturnal haemoglobinuria.
abstract: Paroxysmal nocturnal haemoglobinuria (PNH) is an acquired clonal disorder of haematopoietic stem cells associated with a somatic mutation in the phosphatidylinositol glycan complementation class A (PIG-A) gene. The only curative option is an allogeneic stem cell transplant (SCT), although treatment is hazardous. A 46-year-old male patient with PNH and obvious signs of severe, progressive haemolysis was transplanted in July 2002 with highly purified CD34 T-cell depleted peripheral blood stem cells from his HLA-identical brother. Prior to transplantation, the PNH was resistant to immunosuppressive therapy. The patient received 6.1 x 10(6)/kg bodyweight CD34-positive cells with a proportion of CD3-positive cells of 0.81 x 10(4)/kg bodyweight. After engraftment, 12 days post transplant (neutrophils>1.0/nl) the patient's physical condition steadily improved and parameters of haemolysis decreased. No glycophosphatidylinositol-deficient cells in peripheral blood could be detected by flow cytometry 40 and 100 days after transplant. We conclude that PNH may be cured by allogeneic CD34-enriched SCT from a sibling donor attempting to avoid acute GVHD and to reduce cumulative organ toxicity by using this transplantation modality.
authors.affiliation: Department for Bone Marrow Transplantation, University Hospital of Essen, Essen, Germany. markus.ditschkowski@uni-essen.de
authors.forename: M
authors.initials: M
authors.lastname: Ditschkowski
authors.forename: R
authors.initials: R
authors.lastname: Trenschel
authors.forename: G
authors.initials: G
authors.lastname: Kummer
authors.forename: A H
authors.initials: AH
authors.lastname: Elmaagacli
authors.forename: D W
authors.initials: DW
authors.lastname: Beelen
mesh_terms: D018952:Antigens, CD34; D018572:Disease-Free Survival; D006457:Hemoglobinuria, Paroxysmal / Q000628:therapy*; D006801:Humans; D008212:Lymphocyte Depletion; D008297:Male; D008875:Middle Aged; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods*; D012074:Remission Induction; D035781:Siblings; D014184:Transplantation, Homologous
chemical_list: D018952:Antigens, CD34