[Experimental therapy of hypoplastic paroxysmal nocturnal hemoglobinuria].
Paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia are associated either as a PNH-aplasia syndrome or the emergence of glycosylphosphatidylinositol-(GPI)deficient blood cells in patients with severe aplastic anemia (SAA). It could be demonstrated that SAA patients with GPI-deficient cells in comparison to those without have a worse response to classical immunosuppressive therapy. Therefore, we treated a female PNH patient with severe thrombocytopenia, anemia and granulopenia with G-CSF and cyclosporine. Within 8 weeks, a trilineage response of hematopoiesis was observed. In addition, the proportion of normal to GPI-deficient granulocytes and monocytes increased significantly.
Schubert
J
J
Abteilung Klinische Immunologie, Medizinischen Hochschule Hannover.
Scholz
C
C
Schmidt
R E
RE
D000328:Adult; D015703:Antigens, CD / Q000378:metabolism; D018952:Antigens, CD34; D018957:CD59 Antigens; D016572:Cyclosporine / Q000008:administration & dosage*; D005260:Female; D017261:Glycosylphosphatidylinositols / Q000172:deficiency; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage*; D006457:Hemoglobinuria, Paroxysmal / Q000188:drug therapy*; D006801:Humans; D008562:Membrane Glycoproteins / Q000378:metabolism; D013921:Thrombocytopenia / Q000188:drug therapy
D015703:Antigens, CD; D018952:Antigens, CD34; D018957:CD59 Antigens; D017261:Glycosylphosphatidylinositols; D008562:Membrane Glycoproteins; D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine