title: Danazol for paroxysmal nocturnal hemoglobinuria.
abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal stem-cell disorder in which blood cells lack complement inhibiting membrane proteins, and become susceptible to complement-mediated injury, leading to chronic intravascular hemolysis and pancytopenia. Glucocorticoids have been a mainstay of therapy. For patients refractory to glucocorticoids and requiring blood transfusions, an alternative therapy is needed. We studied danazol therapy in 5 patients refractory to other treatments. Four of the 5 benefited, showing rise in hematocrit and eventual cessation of transfusion requirements. Remissions lasted > or =2 years in 3 and 10 years in 1 patient. Danazol was well-tolerated without serious side effects. Danazol appears to be a good alternative treatment in PNH.
authors.affiliation: Department of Medicine, University of Miami School of Medicine, Florida 33101, USA.
authors.forename: W J
authors.initials: WJ
authors.lastname: Harrington
authors.forename: L
authors.initials: L
authors.lastname: Kolodny
authors.forename: L L
authors.initials: LL
authors.lastname: Horstman
authors.forename: W
authors.initials: W
authors.lastname: Jy
authors.forename: Y S
authors.initials: YS
authors.lastname: Ahn
mesh_terms: D000293:Adolescent; D000328:Adult; D002940:Circadian Rhythm; D003613:Danazol / Q000627:therapeutic use*; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000188:drug therapy* / Q000503:physiopathology*; D006801:Humans; D008297:Male; D008875:Middle Aged; D012074:Remission Induction
chemical_list: D003613:Danazol