Danazol for paroxysmal nocturnal hemoglobinuria

William J. Harrington, Luciano Kolodny, Lawrence L. Horstman, Wenche Jy, Yeon S. Ahn
In: American Journal of Hematology · 1997 · vol. 54(2) , pp. 149–154 · doi:10.1002/(sici)1096-8652(199702)54:2<149::aid-ajh9>3.0.co;2-x · PMID:9034290 · W1984400657
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Danazol therapy improved hematocrit and reduced transfusion needs in four of five patients with paroxysmal nocturnal hemoglobinuria refractory to other treatments.

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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.

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