Size vs safety

Neal Barnard, president and founder of the Physicians Committee for Responsible Medicine, has teamed up with the Lawson Wilkins Pediatric Endocrine Society to assess the prevalence of growth-suppression therapy using oral estrogens (J. Ped. Adol. Gyn. 2002, 15 (1), 23–26). They sent out questionnaires to 715 pediatric endocrinologists in the United States, surveying whether they offered this therapy in their practices, how many patients each clinician had recently treated using oral estrogens, criteria for initiating and terminating treatment, choice of estrogen, and typical therapy regimens.

Out of 411 valid responses, 137 respondents (33.3%) reported offering the growth-suppression treatment to their patients, 92 (22%) reported treating 1–5 girls during the preceding five years, and 4 reported treating more than 5 cases during this period.

Barnard explains that patients are given an oral estrogen, such as Premarin, in the same manner as women who receive hormone replacement therapy after menopause. “The difference is that the dose is much, much higher, and that’s because the intention is not to supplement a little estrogen that might have [been] reduced after menopause,” explains Barnard. “The intention is to flood the body with estrogen.” A girl who is expected to grow to 6 feet or higher will typically be treated for 2–3 years.

The estrogen affects the cartilage growth plate in the long bones. “When you have a large amount of estrogen, it causes that cartilage to mature into the adult form, and so growth stops,” says Barnard.

Because no one has studied the long-term effects of growth-suppression therapy, physicians are unable to give patients a comprehensive list of risks and benefits. Barnard explains that the most serious threats are hormone-related diseases such as breast and uterine cancer. Other concerns include weight gain, and the treatment often causes girls to start menstruating prematurely. Informal surveys show that girls who receive this estrogen treatment go on to experience reproductive health problems, including miscarriage, endometriosis, and infertility.

Barnard and his colleagues hope to partner with a pharmaceutical company to study the long-term effects of this therapy. In the meantime, they are petitioning the FDA to change the language on the prescribing information so that physicians and patients know that hormones are not approved for growth-suppression purposes. “That doesn’t mean it’s illegal,” he explains, “but it means it’s not been proven to be safe and effective. Its long-term safety in that context has not been established.”