GH safety workshop position paper: a critical appraisal of recombinant human GH therapy in children and adults

Abstract

Recombinant human GH (rhGH) has been in use for 30 years, and over that time its safety and efficacy in children and adults has been subject to considerable scrutiny. In 2001, a statement from the GH Research Society (GRS) concluded that ‘for approved indications, GH is safe’; however, the statement highlighted a number of areas for on-going surveillance of long-term safety, including cancer risk, impact on glucose homeostasis, and use of high dose pharmacological rhGH treatment. Over the intervening years, there have been a number of publications addressing the safety of rhGH with regard to mortality, cancer and cardiovascular risk, and the need for long-term surveillance of the increasing number of adults who were treated with rhGH in childhood. Against this backdrop of interest in safety, the European Society of Paediatric Endocrinology (ESPE), the GRS, and the Pediatric Endocrine Society (PES) convened a meeting to reappraise the safety of rhGH. The ouput of the meeting is a concise position statement.

Growth hormone treatment in children born small for gestational age: One center experience

“…Although the thyroid function test showed no significant changes, one patient developed subclinical hypothyreosis, requiring supplementation therapy. These results are consistent with the literature data (14).…”

Growth response to growth hormone treatment in patients with SHOX deficiency can be predicted by the Cologne prediction model

“…Based on the fact that the growth response in the first year is associated with the long-term outcome, it is reasonable to evaluate the growth response as early as possible to predict the long-term outcome [28][29][30]. Although the experience with GH therapy over 30 years suggests that GH treatment is safe [31,32], discussions about adverse effects especially in the long-term flared up again recently because of results from the French cohort of the Safety and Appropriateness of Growth Hormone Treatments in Europe (SAGhE) study [33,34]. Moreover, treatment costs are very high.…”

Growth hormone, insulin-like growth factor system and carcinogenesis

“…The constant need to address the long-term safety of rhGH treatment was the main motivation for the European Society of Paediatric Endocrinology (ESPE), the Growth Hormone Research Society (GRS), and the Paediatric Endocrine Society (PES) to convene an international meeting in 2014, which resulted in the publication of a concise position statement [87]. The document has addressed important issues related to rhGH therapy and cancer risk, as summarised in Table III.…”

“…dren, but made a recommendation to discuss this possibility with patients and their families [87]. In patients surviving adult-onset malignancies, data were not sufficient to reach a conclusion.…”

“…Neurofibromatosis type 1, Fanconi anaemia, or Down syndrome). In these clinical situations, the authors recommended that the decision to start therapy should be carefully considered and discussed with families [87].…”