Danazol for paroxysmal nocturnal hemoglobinuria

William J. Harrington, Luciano Kolodny, Lawrence L. Horstman, Wenche Jy, Yeon S. Ahn

Research output: Contribution to journalArticle

17 Scopus citations

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 ≤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.

Original languageEnglish (US)
Pages (from-to)149-154
Number of pages6
JournalAmerican Journal of Hematology
Volume54
Issue number2
DOIs
StatePublished - Feb 26 1997

Keywords

  • androgen therapy
  • danazol
  • PNH

ASJC Scopus subject areas

  • Hematology

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