Design of, and first data from, PATRO Children, a multicentre, noninterventional study of the long-term efficacy and safety of Omnitrope^® in children requiring growth hormone treatment

Abstract

The efficacy and safety profile of Omnitrope(®) in the PATRO Children study so far are as expected. The ongoing study will extend the safety database for Omnitrope(®), and rhGH products more generally, in paediatric indications. Of particular interest, PATRO Children will add important information on the diabetogenic potential of rhGH in children born SGA, the risk of malignancies in children receiving rhGH, and AEs with a possible causal relationship to rhGH treatment in children with PWS.

Is safety of childhood growth hormone therapy related to dose? Data from a large observational study

“…The safety profile of GH treatment reported in our study is consistent with findings from other observational studies (11,29,33,34). Furthermore, our data collected from a large, multinational, observational study demonstrate no association between GH dose up to the event and the occurrence of ADRs in patients with short stature across various indications.…”

“…Comparision of the IRs for AEs reported in NordiNet ® IOS with those reported in other GH observational studies can be seen in Table 3 (11,26,26,27,28,29,30,31,32). With the exception of SAEs in the intermediaterisk group, the IR for all AEs was independent of GH dose ( Figs 1B and 2B).…”

“…Headache, a relatively frequent complication among children receiving GH treatment, was reported across all risk groups with duration of GH therapy to start of AE showing wide variation as has previously been reported in KIGS (41,42). Benign intracranial hypertension, previously reported in association with GH therapy (29,41,42), was reported in a single patient with SGA in our study, although it is recognized to be more common among children with GHD, Turner syndrome, Prader-Willi syndrome and CRI (11,29,41). Hypertension, which is frequent in patients with Turner syndrome, and may be unrelated to GH therapy (43), was reported in a single patient in our study.…”

Review of growth hormone therapy in adolescents and young adults with Prader–Willi syndrome

“…Treatment with recombinant GH therapy has been utilised for over 20 years for a number of different conditions [58]. Consensus guidelines advise monitoring patients with PWS on GH treatment for a variety of different possible complications, including changes in physical appearance, joint pain, sleep apnoea, headache, benign intracranial hypertension, slipped capital femoral epiphysis, insulin resistance and decreased levels of thyroid hormone, all of which have been reported in paediatric patients with or without PWS who were receiving GH treatment [1].…”

Long-term safety and efficacy of Omnitrope®, a somatropin biosimilar, in children requiring growth hormone treatment: Italian interim analysis of the PATRO Children study

“…It has been conducted as part of the risk management plan for Omnitrope®, to fulfil the commitment with the EMA. Interim 1-year results of the patients included in this study up to September 2012 ( n  = 1837) have previously been reported [15] and annual updates have been presented at international meetings [16]. Up to January 2016, 4675 patients have been recruited from 291 sites across 14 countries (Austria, Czech Republic, France, Germany, Italy, Poland, Romania, Slovenia, Spain, Sweden, Taiwan, UK, Canada and USA) in the PATRO Children study.…”