Unmet Needs in Growth Hormone Deficiency Management

This episode takes stock of the most significant gaps in the diagnosis and management of GHD across both pediatric and adult populations, drawing on the panel’s collective clinical experience.

Dr. Alter leads the pediatric discussion, identifying diagnostic imprecision as the most pressing unmet need. Growth hormone stimulation testing remains an imperfect tool — producing false negatives in truly GHD children and false positives in normal children. He envisions a future in which advanced genetic testing could isolate the underlying cause of poor growth and predict GH responsiveness without relying on dynamic tests.

The panel also raises the question of whether GH should be used in children who would respond well physiologically but do not meet current diagnostic criteria — a philosophically and ethically challenging question intersecting with indications, insurance coverage, and the expanding availability of more affordable daily GH formulations.

On the adult side, Dr. Agrawal identifies three major unmet needs: the lack of reliable biomarkers (IGF-1 is normal in up to 10–20% of GHD patients), the absence of FDA-approved and commercially available testing options (macimorelin), and the profound underdiagnosis of adult GHD in both general and endocrine practice. She calls for expanded education at the fellow and practicing-physician level.

Dr. Garcia reinforces the need for broader physician engagement. He notes that adult endocrinologists — particularly those without pituitary disease training — often lack the comfort level and resources to diagnose and manage adult GHD. The panel emphasizes that this is fundamentally a workforce and education problem as much as a scientific one.

In the next episode, “Initiating Growth Hormone Therapy in Pediatric Patients,” the panel transitions to treatment, examining how and when to start GH therapy and the growing role of long-acting weekly formulations.