Danazol for paroxysmal nocturnal hemoglobinuria.
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.
Harrington
W J
WJ
Department of Medicine, University of Miami School of Medicine, Florida 33101, USA.
Kolodny
L
L
Horstman
L L
LL
Jy
W
W
Ahn
Y S
YS
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
D003613:Danazol