A discussion of pediatric growth hormone deficiency with Andrew Dauber, MD, MMSc

Andrew Dauber, MD, MSSc, Chief of Endocrinology at Children’s National Hospital in Washington, DC, discussed standard of care for pediatric growth hormone deficiency and how the general provider can play a role in a multidisciplinary team when it comes to treatment.

“For many years, the standard of care treatment for growth hormone deficiency was to receive daily injections of growth hormone,” Dauber explained. “Recombinant human growth hormone was made for the first time in the mid-1980s, so that's been around for around 40 years, and is very effective.” However, despite its efficacy, the treatment regimen has downsides.

“The obvious downside of it is that it's a daily injection treatment, and kids need to be on it for many, many years, right? So if they get diagnosed at age 6 or 7 or 8, they can continue on daily injections through all of puberty, until they're done growing and sometimes even into adulthood,” said Dauber. This long-term daily treatment can be challenging for both patients and their families.

“In the last few years, there's now been weekly formulations of growth hormone approved,” Dauber noted. “There are actually in America 3 different once-a-week injectables, and those all work quite well.”

While these weekly injections offer a more convenient alternative, they still come with certain challenges. “The major downside of those are still that it's an injection, so there's some kids, patients, and families who really would prefer, obviously, to avoid injection medicine,” Dauber stated. Additionally, the cost of these weekly injections remains a barrier for many families. “And they're also still quite expensive. So those are, I would say, the 2 major barriers.”

Dauber emphasized that compliance remains an issue with any long-term medication, especially when injections are involved. “We know, with any long-term medicine, and especially injectable medicines, there's a decrease in compliance over time, and that really affects outcomes for these patients,” he explained.

The introduction of weekly growth hormone injections represents a significant advance in the treatment of GHD, offering greater convenience and efficacy. However, challenges related to cost, injection discomfort, and compliance still need to be addressed to optimize patient care and outcomes.

Pediatricians, in collaboration with endocrinologists, continue to play an essential role in monitoring patient progress and ensuring optimal outcomes for children with GHD.