ESPE Yearbook of Paediatric Endocrinology

J Clin Endocrinol Metab. 2025 Mar 8:dgaf156. PMID: 40056454 doi: 10.1210/clinem/dgaf156. [email protected].

Brief summary: This cross-sectional analysis of patients enrolled in the St. Jude Lifetime Cohort Study (SJLIFE) investigated the prevalence of severe adult growth hormone deficiency (aGHD) and its treatment, explored potential associations with socioeconomic factors, and examined the impact of aGHD on physical, psychosocial, and neurocognitive outcomes.

Detailed information regarding growth hormone deficiency (GHD) diagnosis, as well as the dose and/or duration of growth hormone therapy (GHT), was not available. Therefore, the authors used IGF-1 z-scores as a surrogate marker of GHD/GHT status in cases where a confirmed diagnosis of GHD or documentation of GHT was lacking. Based on this approach, severe aGHD was identified in 354 of 3,902 patients. However, only 9% (32 patients) reported receiving GHT, 286 had mild untreated aGHD, and 3,262 did not have aGHD. Patients in the aGHD group who received GHT had higher socioeconomic status and were more likely to have health insurance compared to the other three groups. This finding was also confirmed in analyses restricted to the aGHD group, showing differences between treated and untreated patients. Low IGF-1 levels, used as a surrogate marker for untreated severe adult growth hormone deficiency (aGHD), were associated with a higher prevalence of abnormal body composition, reduced handgrip strength, adverse metabolic and cardiovascular outcomes, increased neurocognitive disorders, diminished health-related quality of life (QOL), and a higher incidence of depression.

Comment: Among the endocrine consequences in childhood cancer survivors (CCSs), growth hormone deficiency (GHD) is one of the most common, particularly when the hypothalamic–pituitary region is directly affected by the tumor itself and/or its treatment (e.g., radiotherapy). GHD may begin in childhood and persist into adulthood, and if left untreated, is associated with a higher risk of complications compared to adult-onset GHD. This study highlights that untreated adult growth hormone deficiency (aGHD) among adult survivors of childhood cancer is common and associated with a wide range of adverse outcomes. There are several possible reasons for the relatively high number of untreated GHD cases. These include missed diagnoses during routine follow-up, limited awareness regarding indications, benefits, and safety of treatment, and socioeconomic barriers, especially in countries where GH therapy (GHT) is costly and requires health insurance coverage. The value of this large cohort study lies in its focus on the risk of complications in untreated aGHD patients among CCSs, whose survival and QOL are impacted by multiple health issues and may be further compromised by the absence of GHT when needed.

It is essential to implement strategies that ensure appropriate GHT for eligible patients. These include timely diagnosis, even in cases where the tumor did not directly affect the hypothalamic–pituitary axis, clear communication about the importance of GHT for overall health, reassurance regarding potential side effects, socioeconomic support when needed, and, last but not least, a collaborative approach among all specialists involved in the care of CCSs.