Oxandrolone treatment of childhood hereditary angioedema.
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.
Church
Joseph A
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
D045930:Anabolic Agents / Q000627:therapeutic use*; D000799:Angioedema / Q000188:drug therapy*; D002648:Child; D003174:Complement C1 Inactivator Proteins / Q000187:drug effects; D003181:Complement C4 / Q000187:drug effects; D006801:Humans; D008297:Male; D010074:Oxandrolone / Q000627:therapeutic use*
D045930:Anabolic Agents; D003174:Complement C1 Inactivator Proteins; D003181:Complement C4; D010074:Oxandrolone