Oxandrolone treatment of childhood hereditary angioedema.

Ann Allergy Asthma Immunol. 2004 Mar;92(3):377-8.

Oxandrolone treatment of childhood hereditary angioedema.

Church JA.

Division of Clinical Immunology and Allergy, Department of Pediatrics, Childrens
Hospital Los Angeles and Keck of School of Medicine, The University of Southern
California, Los Angeles, California 90027, USA. jchurch@chla.usc.edu

BACKGROUND: The virilizing effects of danazol, stanozolol, and methyltestosterone
significantly restrict the usefulness of these agents in the treatment of
children with hereditary angioedema (HAE). Oxandrolone is a synthetic anabolic
steroid with limited virilizing effects that has been used in a variety of
pediatric conditions and has an acceptable safety profile.

OBJECTIVE: To report
the effective use of oxandrolone in a 6-year-old boy with recurrent,
life-threatening episodes of angioedema.

METHODS: Oxandrolone was administered at
a dose of 0.1 mg/kg per day. Symptoms and laboratory findings were evaluated by
parental report and laboratory analysis of serum C1 esterase inhibitor and C4
levels, respectively.

RESULTS: Oxandrolone therapy resulted in a marked reduction
in clinical episodes and normalization of serum complement levels; cessation of
oxandrolone therapy resulted in recurrence of symptoms and decreased complement
levels. However, early signs of virilization were noted.

CONCLUSIONS: Oxandrolone
treatment was associated with significant clinical and laboratory evidence of a
therapeutic effect in a prepuberal boy with HAE. It is imperative to treat HAE
with the lowest dose of oxandrolone that controls life-threatening episodes of
angioedema