title: Evaluation of danazol, cyclosporine, and prednisolone as single agent or in combination for paroxysmal nocturnal hemoglobinuria.
abstract: OBJECTIVE
  The responses of 32 patients with paroxysmal nocturnal hemoglobinuria (PNH) were assessed after the patients were put on various combinations of danazol, prednisolone, and cyclosporine.
  
  
  METHODS
  Nineteen males and 13 females aged between 14 and 60 years with confirmed diagnosis of PNH were treated with danazol (4), danazol + cyclosporine (7), cyclosporine (1), and prednisolone + danazol (20). Response to these interventions was assessed regularly. Danazol was added to cyclosporine in patients with aplastic bone marrow after 3 months of cyclocporine use only unless the former therapy was successful. Four patients with aplastic marrow received only danazol because they had renal insufficiency at presentation. Patients were evaluated with regular complete blood count and routine liver and renal function tests.
  
  
  RESULTS
  One patient responded to cyclosporine only. Thirteen of 32 patients (40%) had complete response, 12/32 patients (37%) had partial response leading to freedom from red cell transfusion, and 2/32 (7%) had no response. Five patients (16%) died due to thrombosis or hemorrhage within 3 months of therapy before their response to therapy could be assessed. The median period of review of the cases was 4 years and 6 months.
  
  
  CONCLUSIONS
  Danazol is a useful addition to PNH therapy both in combination with cyclosporine for hypoplastic PNH and with prednisolone for other forms of PNH, and this therapy could be a good alternative where eculizumab and anti-lymphocyte globulin cannot be used for various reasons.
  
  
  BACKGROUND
  None declared.
authors.affiliation: National Institute of Immunohaematology, 13th Floor, NMS Building, KEM Hospital Campus, Parel, Mumbai, India and Department of Haematology, 10th Floor, NMS Building, KEM Hospital Campus, Parel, Mumbai, India.
authors.forename: Kanjaksha
authors.initials: K
authors.lastname: Ghosh
authors.affiliation: National Institute of Immunohaematology, 13th Floor, NMS Building, KEM Hospital Campus, Parel, Mumbai, India and Department of Haematology, 10th Floor, NMS Building, KEM Hospital Campus, Parel, Mumbai, India.
authors.forename: Manisha
authors.initials: M
authors.lastname: Madkaikar
authors.affiliation: National Institute of Immunohaematology, 13th Floor, NMS Building, KEM Hospital Campus, Parel, Mumbai, India and Department of Haematology, 10th Floor, NMS Building, KEM Hospital Campus, Parel, Mumbai, India.
authors.forename: Maya
authors.initials: M
authors.lastname: Gupta
authors.affiliation: National Institute of Immunohaematology, 13th Floor, NMS Building, KEM Hospital Campus, Parel, Mumbai, India and Department of Haematology, 10th Floor, NMS Building, KEM Hospital Campus, Parel, Mumbai, India.
authors.forename: Farah
authors.initials: F
authors.lastname: Jijina
keywords: Danazol; Hemoglobinuria; Immunosuppression; Paroxysmal; Prednisolone; cyclosporine