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Danazol for paroxysmal nocturnal hemoglobinuria

✍ Scribed by Harrington, William J.; Kolodny, Luciano; Horstman, Lawrence L.; Jy, Wenche; Ahn, Yeon S.

Publisher
John Wiley and Sons
Year
1997
Tongue
English
Weight
45 KB
Volume
54
Category
Article
ISSN
0361-8609

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✦ Synopsis

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 Υ†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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