Growth hormone replacement therapy in childhood-onset craniopharyngioma

Area of Science:

• Pediatric Endocrinology
• Neuro-oncology
• Endocrinology

Background:

• Childhood onset craniopharyngioma (cCP) frequently leads to growth hormone (GH) deficiency due to the tumor or its treatment.
• Growth hormone replacement therapy (GHRT) is vital for optimizing growth velocity and final height in affected children.
• Continuing GHRT into adulthood offers significant benefits for metabolic, bone, mental health, and quality of life.

Purpose of the Study:

• To review the current evidence on the safety and efficacy of GH replacement therapy (GHRT) in children with craniopharyngioma (cCP).
• To assess the impact of GHRT on tumor progression, recurrence, secondary neoplasms, and mortality in cCP patients.
• To provide guidance on the optimal timing for initiating GHRT in newly diagnosed cCP patients.

Main Methods:

• Systematic review of existing literature and clinical evidence regarding GHRT in cCP.
• Analysis of studies examining long-term outcomes, including tumor behavior and patient survival.
• Evaluation of data on the benefits of GHRT for growth and metabolic parameters.

Main Results:

• Current evidence suggests GHRT does not increase the risk of tumor progression, recurrence, secondary cancers, or mortality in cCP patients.
• Early initiation of GHRT (as early as three months post-surgery) can effectively address linear growth deficits and metabolic issues.
• GHRT demonstrates positive effects on bone health, mental well-being, and overall quality of life in cCP survivors.

Conclusions:

• GH replacement therapy is a safe and beneficial treatment for children with craniopharyngioma experiencing GH deficiency.
• GHRT should be considered for early initiation to manage growth and metabolic disturbances, with potential for long-term continuation.
• Further research is warranted on the long-term effects of early GHRT initiation and the use of long-acting GH preparations in cCP patients.