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Danazol for paroxysmal nocturnal hemoglobinuria
W J Harrington1, L Kolodny, L L Horstman
1Department of Medicine, University of Miami School of Medicine, Florida 33101, USA.
American Journal of Hematology
|February 1, 1997
Summary
Danazol therapy offers a promising alternative for patients with paroxysmal nocturnal hemoglobinuria (PNH) refractory to glucocorticoids. This treatment led to improved hematocrit and reduced transfusion needs in most patients, with good tolerability.
Area of Science:
- Hematology
- Immunology
- Pharmacology
Background:
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal stem cell disorder characterized by complement-mediated red blood cell destruction.
- Current treatments like glucocorticoids are not effective for all PNH patients, necessitating alternative therapies, especially for those requiring transfusions.
Observation:
- A study investigated danazol therapy in five patients with PNH who were refractory to other treatments.
- These patients exhibited resistance to conventional therapies and depended on blood transfusions.
Findings:
- Four out of five patients treated with danazol showed significant clinical improvement.
- Improvements included an increased hematocrit level and a cessation of transfusion requirements.
- Remissions were sustained for at least two years in three patients and ten years in one patient.
Implications:
- Danazol demonstrates potential as an effective and well-tolerated alternative treatment for PNH.
- This finding is particularly relevant for glucocorticoid-refractory PNH patients who require transfusions.
- Further research may solidify danazol's role in managing PNH