Danazol for paroxysmal nocturnal hemoglobinuria.

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.

journal_name

Am J Hematol

authors

Harrington WJ Sr,Kolodny L,Horstman LL,Jy W,Ahn YS

doi

10.1002/(sici)1096-8652(199702)54:2<149::aid-ajh9>

subject

Has Abstract

pub_date

1997-02-01 00:00:00

pages

149-54

issue

2

eissn

0361-8609

issn

1096-8652

pii

10.1002/(SICI)1096-8652(199702)54:2<149::AID-AJH9>

journal_volume

54

pub_type

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