Panel calls prenatal treatment for congenital adrenal hyperplasia ‘experimental’
Clinical practice guidelines for treating children with congenital adrenal hyperplasia take a dim view of prenatal steroid treatment that may prevent ambiguous genitalia in girls.
CAH is an inherited disorder involving malfunctioning adrenal glands that requires lifelong treatment. In girls with severe cases, the disorder can cause ambiguous genitalia, including the development of a penis even though these babies usually have a uterus and ovaries. However, the disorder has also been the subject of recent, highly publicized debate because some doctors propose treating pregnant women who know they are at risk for having a child with CAH with the steroid dexamethasone in order to reduce the risk of masculinized genitalia in girls and, a few researcher say, later predisposition to being lesbian.
In the new guidelines, experts say prenatal treatment is experimental, should take place only as part of clinical trials governed by Institutional Review Boards and only in “centers of excellence” that have expertise with the treatment. The panel criticized as inadequate the few studies on the treatment’s risks and benefits. They noted, however, that “the groups advocating and performing prenatal treatment appear to agree that it is effective in reducing and often eliminating virilization of female fetal genitalia and that the success rate is about 80% to 85%.”
The panel said, however, that it places a higher value on preventing unnecessary risks from prenatal dexamethasone exposure to the mother and baby and a lower value on “minimizing the emotional toll of ambiguous genitalia on parents and patients.” The guidelines appear in the September issue of the Journal of Clinical Endocrinology & Metabolism.
The issue is part of a larger controversy about how to address ambiguous genital in newborns. Experts disagree about what kinds of surgeries should be conducted to correct physical problems and when those operations should take place. The new guidelines support early surgery for genital repair of severely virilized girls and warned against experimental therapies to promote growth or delay puberty.
Perhaps more important to families, a paper explaining a shared decision-making process for dealing with disorders of sexual development was published in the August issue of the Journal of Pediatric Endocrinology & Metabolism. The paper notes that there is no standard approach or best practice for doctors and families to follow when a child is born with ambiguous genitalia.
-- Shari Roan / Los Angeles Times
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