How short is short enough?

October 10, 2004

Unintended consequences are alive and active in pediatric endocrinology. When the Food and Drug Administration approved the use of growth hormone to treat idiopathic short stature in 1993, pediatricians found themselves trying to decide how short is short enough to treat.

Unintended consequences are alive and active in pediatric endocrinology. When the Food and Drug Administration approved the use of growth hormone to treat idiopathic short stature in 1993, pediatricians found themselves trying to decide how short is short enough to treat. One expert, speaking to the press on Saturday at the AAP 2004 National Conference and Exhibition, offered a broad but useful philosophy for clinicians to consider in counseling parents and treating their patients.

The FDA indication for growth hormone requires that children be at least 2.25 standard deviations shorter than what is considered normal for age, explained Ron Rosenfeld, MD, senior vice president for medical affairs at the Lucile Packard Foundation for Children's Health at Stanford University. That equates to the shortest 1% of children in the US population.

The agency may have believed it was creating an objective standard of treatment, Dr. Rosenfeld said, "but nobody accepts this as an endorsement that 1% of American kids should be treated. There are enormous ethical and financial issues."

Growth hormone treatment costs about $20,000 per child per year, Dr. Rosenfeld noted. Insurers are willing to cover treatment for children suffering from growth hormone deficiency, chronic renal failure, Turner syndrome, and other disease-related causes of short stature. But they do not want to open the door to unrestricted use or create incentives for dubious "short clinics."

"Shortness is a physical trait, not a disease," Dr. Rosenfeld emphasized. "The question becomes who is short enough to treat."

His answer is to reserve treatment for children who are significantly shorter than normal and who are suffering a demonstrable handicap from their short stature.

"I am not in the business of making tall people," Dr. Rosenfeld said, in inviting other pediatricians to consider his approach. "I am in the business of getting people into the normal range of height and alleviating any psychosocial disadvantage of short stature."