Growth hormone replacement does not increase mortality in patients with childhood-onset growth hormone deficiency

Agnethe Berglund1, Claus Højbjerg Gravholt1,2, Morten Smaerup Olsen3

  • 1Department of Endocrinology and Internal Medicine, Aarhus University Hospital, Denmark.

Insights

Growth hormone (GH) replacement therapy in childhood-onset GH deficiency (CO GHD) patients was associated with significantly decreased mortality, particularly from malignancy. This study indicates GH treatment does not increase mortality risks in these patients.

Area of Science:

  • Endocrinology
  • Pediatric Endocrinology
  • Clinical Research

Background:

  • Long-term safety of growth hormone (GH) treatment remains debated.
  • Previous studies suggested potential increased mortality or stroke risk with GH treatment in children, including those with non-GH deficiency (GHD) causes.
  • Concerns exist regarding the safety of GH therapy in pediatric populations.

Purpose of the Study:

  • To investigate the impact of GH replacement on overall and cause-specific mortality in patients with childhood-onset GHD (CO GHD).
  • To address the ongoing debate surrounding the long-term safety of GH treatment in pediatric patients.
  • To compare mortality rates between GH-treated and non-GH-treated CO GHD patients and the general population.

Main Methods:

  • A nationwide, population-based registry study comparing 494 CO GHD patients with 100 general population controls each.
  • Cox regression analysis was used to compute mortality hazard ratios (HRs) between patients and controls, and between GH-replaced and non-GH-replaced patients.
  • Adjustments were made for confounders including birth year, diagnosis year, gender, irradiation, ACTH insufficiency, and primary disease.

Main Results:

  • Mortality was substantially increased in CO GHD patients compared to the general population (HR = 7.51).
  • GH replacement was associated with significantly decreased mortality in CO GHD patients, both overall (HR = 0.27) and due to malignancy (HR = 0.14).
  • After adjusting for confounders, the decreased mortality remained significant (overall HR = 0.56; malignancy HR = 0.33), with highest mortality observed in non-GH-replaced patients.

Conclusions:

  • Data from this national cohort of CO GHD patients do not support the hypothesis that GH replacement increases mortality.
  • GH replacement therapy appears to be safe concerning overall and cause-specific mortality in childhood-onset GHD.
  • The findings suggest that GH treatment may offer survival benefits in patients with CO GHD.
Abstract

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