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Related Experiment Videos

[Long-term danazol therapy for hereditary angioedema].

R Höpfl1, S Schwarz, P Fritsch

  • 1Klinik für Dermatologie und Venerologie, Universität Innsbruck.

Deutsche Medizinische Wochenschrift (1946)
|January 26, 1990
PubMed
Summary

Danazol effectively treats hereditary angio-oedema (HAE) by reducing attack frequency and severity. This androgen preparation shows minimal side effects and can improve C1-esterase inhibitor levels in HAE patients.

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Area of Science:

  • Immunology
  • Endocrinology
  • Pharmacology

Background:

  • Hereditary angio-oedema (HAE) is a rare genetic disorder characterized by recurrent episodes of severe swelling.
  • Current treatments aim to manage acute attacks and prevent future occurrences.
  • Danazol, an androgen derivative, has been used for HAE prophylaxis due to its C1-esterase inhibitor-boosting properties.

Observation:

  • A 10-year treatment study involved three HAE patients (19-year-old male, 35-year-old male, 69-year-old female).
  • Danazol dosages varied, with maintenance therapy and occasional boosters for the young male, while others received regular weekly doses.
  • Patients experienced significant reductions in attack frequency and severity, with one becoming symptom-free and another fathering a child.

Findings:

Related Experiment Videos

  • Danazol treatment normalized sexual maturation in a male patient treated from age nine through puberty.
  • C1-esterase inhibitor levels increased from approximately 30% to 50% of normal in all treated patients.
  • Minimal endocrine, metabolic, and toxic side effects were observed during danazol therapy.

    • Implications:

    • Danazol demonstrates long-term efficacy and safety in managing hereditary angio-oedema across different age groups and sexes.
    • The observed increase in C1-esterase inhibitor levels suggests a direct mechanism for HAE symptom control.
    • Danazol represents a viable therapeutic option for HAE, potentially improving quality of life and reducing disease burden.