Endocrine Abstracts

JOINT3686

Introduction: The transition period in patients undergoing recombinant human growth hormone (rhGH) therapy is a critical developmental phase, beginning at the end of puberty and lasting until early adulthood, typically 6–7 years after reaching final height. Despite slowed linear growth, rhGH remains vital for bone and lipid metabolism, body composition, and quality of life. Re-evaluating the somatotropic axis during this phase is essential, as some individuals diagnosed in childhood may exhibit normal secretion upon retesting. If deficiency persists, therapy continuation ensures complete bone mineralization and mitigates metabolic complications. However, many adolescents discontinue treatment or abandon medical follow-up, highlighting the need for coordinated care between pediatric and adult endocrinologists.

Objective: To characterize adults on rhGH replacement therapy for childhood-onset growth hormone deficiency (GHD) followed at a tertiary endocrinology center.

Methods: A retrospective observational study analyzing clinical records of patients followed until October 2024.

Results: The sample included 18 patients, with a median age of 22.5 (18–48) years, and 66.7% (n = 12) were female. The mean age at diagnosis and therapy initiation was 5.4 (±4.6) years, with a mean final height of 1.61 (±0.12) meters. The most frequent etiology was congenital structural abnormalities (66.7%), followed by sequelae-related conditions (22.2%), mainly secondary to space-occupying CNS lesions. Multiple hormone deficiencies were present in 88.9% (n = 16). Axis re-evaluation occurred at a mean age of 16.9 (±1.2) years, with a mean IGF-1 level of 97.2 (±53.9) ng/ml. Dyslipidemia was present in 55.6% (n = 10), and 83.3% of those assessed had low bone mineral density (BMD) or were at risk. Fifteen patients continued rhGH therapy without confirmatory testing, while three discontinued due to normal axis function. However, all three resumed therapy following reassessment due to cardiovascular comorbidities, decreased BMD or worsening quality of life.

Conclusion: rhGH replacement is indicated in patients with persistent deficiency during transition, offering long-term benefits. Most patients with persistent GHD had congenital structural abnormalities and multiple hormone deficiencies. Even those who discontinue therapy require active monitoring in adulthood. Continuous management is essential to optimize quality of life and prevent complications associated with chronic GH deficiency.