Safety of Pediatric rhGH Therapy

Growth hormone (GH) therapy has been applied in the yesteryear; it has demonstrated excellent safety when used for short-term treatments ever since 1958. However, reports from observational studies in the recent past depict inconsistent outcomes in the pediatric population. The potential risks that demand attention are – cancer, cardio and cerebrovascular manifestations, along with diabetes. 

The aim of a review published in the Frontiers in Endocrinology was to overview the main studies reporting long-term safety in children treated with recombinant human GH (rhGH) therapy, to emphasize the evidence for or against the risk of cancer, cardio and cerebrovascular complications and diabetes – associated with this therapy.

RhGH was first derived from E. coli, and was approved by the United States Food and Drug Administration (US FDA) for treating childhood GH deficiency. However, evidence suggests that such therapy is safe only on short-term use. Most studies that report the long-term effects disclose that rhGH therapy is associated with increased risk of type 2 diabetes (T2D) in patients with risk factors for metabolic diseases like obesity, genetic predisposition, and sedentary lifestyle. In addition, in patients with a higher predilection, such as those with Turner syndrome and organic GHD, rhGH therapy may accelerate diabetes pathogenesis. 

Another group of among the pediatric population who are at an increased risk for T2D and metabolic syndrome are those born small for gestational age. Yet, literature indicates long-term metabolic safety of rhGH therapy in this cohort. 

Source: Frontiers in Endocrinology. 2021 Dec 24. doi: 10.3389/fendo.2021.811846