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Danazol for paroxysmal nocturnal hemoglobinuria
Author(s) -
Harrington William J.,
Kolodny Luciano,
Horstman Lawrence L.,
Jy Wenche,
Ahn Yeon S.
Publication year - 1997
Publication title -
american journal of hematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.456
H-Index - 105
eISSN - 1096-8652
pISSN - 0361-8609
DOI - 10.1002/(sici)1096-8652(199702)54:2<149::aid-ajh9>3.0.co;2-x
Subject(s) - paroxysmal nocturnal hemoglobinuria , danazol , hemoglobinuria , medicine , anemia , endometriosis
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 ≥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. Am. J. Hematol. 54:149–154, 1997 © 1997 Wiley‐Liss, Inc.