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Does shortness need a cure?

Small but healthy children can now receive growth hormones. Some parents see salvation, others misuse.

August 11, 2003|Melissa Healy | Times Staff Writer

Patricia Costa's friends told her Nicole was a perfect little girl. Sure, she was tiny for a 3-year-old, but she was adorable and perfectly proportioned. She had a charming feistiness about her -- a determination that seemed to grow as Nicole confronted the childhood taunts of bigger peers and the indignities of being the smallest kid in school.

Like many other children who rank at the bottom of growth charts, Nicole had no identifiable disorder depressing her growth. So what if she's tiny, Patricia's friends insisted. The tests say she's healthy. Why worry?

But Patricia saw a tiny girl on track to become a tiny woman -- 4 feet, 8 inches, by her doctors' estimates -- and that prospect galvanized her to consider a course of costly and intrusive human growth hormone injections for her then 6-year-old daughter. "I'm thinking about when she's 25 and walking into a courtroom with an attache case, or into a hospital with a stethoscope," Patricia Costa said. "Being 4-foot-8 -- that just didn't cut it."

Today, Nicole is 18, and her first bid for power and influence has ended with success. After a seven-year course of human growth hormone injections, Nicole, now 5 feet, 2 inches, recently marched into a meeting room filled with federal regulators, physicians and medical ethicists and appealed to the Food and Drug Administration to sanction the use of human growth hormone by "all the children who now walk in the shoes I outgrew."

In late July, the FDA did just that, expanding its approval of the synthetic human growth hormone -- Eli Lilly and Co.'s Humatrope -- to include children with "idiopathic short stature," kids such as Nicole, whose shortness does not appear to be the result of underlying disease.

The decision could mean that as many as 400,000 U.S. children could get medication for a problem that lies in the ambiguous territory between personal obstacle, social prejudice and yet-to-be discovered medical disorder.

Like the FDA's deliberations on drugs that treat complaints from shyness to depressed libido to acne, its latest decision takes the agency into some ethically tricky territory: Where do consumers' often poignant demands and pharmaceutical companies' desire to expand their markets fit into the process, next to safety and efficacy? And whose responsibility is it -- doctors', patients', insurance companies' or the government's -- to weigh whether a course of medical treatment, especially one that can bring only modest results, is worth the expense, the health risks and the attention of the medical community?

The FDA's deliberations on Humatrope went beyond the traditional confines of technical reports about a drug's safety and effectiveness. The FDA also reviewed various research that showed how short people are more likely to be bullied and teased in school, to consider themselves lonely, to have reduced marriage rates and to be perceived as having lower competence than people of ordinary height. Among these supportive studies were clinical comparisons showing increased self-esteem and decreased behavior problems among those whose height was enhanced with Humatrope's use.

But Humatrope use had downsides as well. For some kids in the target group (children 7 to 15 years old), Humatrope does not work, and for many, even years of treatment will draw them just inside the range of "normal height." A few children developed diabetes-like symptoms or curvature of the spine because of rapid growth. And researchers found 27 cases in which children taking Humatrope developed increased intracranial pressure that mimicked the symptoms of a brain tumor -- an apparent side effect that was reversible, but which required a painful and lengthy exam to rule out cancer.

The science of promoting growth has flourished since the late 1950s, when doctors first began administering a natural form of growth hormone derived from human cadavers to patients with growth deficits. In 1985, Lilly and several other drug firms began producing a synthetic form of human growth hormone, considered safer and easier to manufacture. Although thousands appear to have undertaken such treatment safely, its long-term effects remain uncertain, since many who took it as children are still in their 20s and 30s.

But other testimony considered by the FDA struck a decidedly more personal chord: There were painful accounts of playground taunting fests, of always being chosen last for a team, of being stuffed into lockers and garbage cans and dressed up like a baby. Nicole Costas recalled how lonely she felt waiting on the bench at an amusement park while friends went on rides she was too small for. How embarrassed she was when a kindergarten chair was brought in for her in second grade, because the teacher required all feet on the floor when children were at their desks. How thirsty she was because she couldn't reach the water fountain.

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