Best Practices for Initiating and Optimizing Growth Hormone Therapy in Pediatric GHD

In this segment of an HCPLive special report on the practical management of growth hormone deficiency (GHD), pediatric endocrinologist Joshua Yang, MD, discusses contemporary best practices for initiating and optimizing growth hormone therapy in children.

He emphasizes that the foundation of appropriate treatment is an accurate diagnosis paired with a clear understanding of growth potential and shared treatment goals. For pediatric patients, Yang notes that diagnosis typically relies on a combination of auxologic assessment, growth velocity, biochemical testing, and, when appropriate, imaging studies to confirm GHD and rule out alternative explanations for short stature.

Once the diagnosis of GHD is established, Yang describes a treatment framework centered on 3 key clinical variables: age at treatment initiation, severity of deficiency, and expected adherence. Earlier treatment onset is associated with more robust growth responses, and children with more severe GHD likewise tend to experience greater height gains with therapy.

He underscores the importance of counseling families about likely growth responses based on these factors so that expectations are aligned with the biologic potential and clinical realities of each child. This shared decision-making process helps families understand the trajectory of therapy and remain engaged over the long term.

Yang further highlights adherence as a critical determinant of treatment success and identifies long-acting growth hormone formulations as an emerging tool to address the burden of daily injections. He references contemporary expert consensus recommendations that encourage individualized therapy, including explicit consideration of treatment burden and challenges with daily dosing.

In his practice, he typically begins with guideline-supported weight-based dosing and then individualizes treatment using insulinlike growth factor 1 (IGF-1) levels, clinical response, pubertal status, and adherence. Importantly, he cautions that IGF-1 should not be used as a standalone decision point; rather, clinicians should “treat the patient, not the number,” integrating IGF-1 with auxologic data and the broader clinical picture when optimizing therapy.