Abstract
Introduction: PATRO Adults is an international, longitudinal, non-interventional study of the long-term safety and efficacy of Omnitrope® (recombinant human growth hormone, rhGH; Sandoz) in adults treated in routine clinical practice. The study provides data on the long-term safety of rhGH in adult patients with severe GH deficiency (GHD). Methods: The study includes adult patients who are receiving treatment with Omnitrope® and have provided informed consent. Patients treated with another rhGH before starting Omnitrope® therapy are also eligible for inclusion. Here we report the latest safety data, with a focus on the risk of diabetes. Results: As of September 2018, 1298 patients had been enrolled on the study over >10 years; 1084 (83.5%) had adulthood-onset GHD (combined GHD, n=956) and 204 (15.7%) had childhood-onset GHD. Overall, 659 (50.8%) patients had been pre-treated with another rhGH. Mean (standard deviation [SD]) age was 49.3 (15.3) years, and mean (SD) body mass index was 29.5 (6.4) kg/m(2). In total, 4009 adverse events (AEs) in 889 patients have been reported, with 643 (353 [27.2%] patients) of these regarded as serious. 154 AEs in 92 (7.1%) patients were suspected to be related to Omnitrope®; these included general disorders/administration site conditions in 23 patients, nervous system disorders in 23 patients and musculoskeletal/connective tissue disorders in 36 patients. A total of 20 serious AEs (SAEs) in 17 (1.3%) patients were suspected to be related to Omnitrope®, leading to treatment discontinuation in 5 patients. Overall, 328 patients (282 with combined GHD) have discontinued the study, 73 (61 with combined GHD) due to AEs (26 were considered related to rhGH treatment). To date, 22 cases of diabetes have been reported (21 in patients with combined GHD; 20 in patients with adult-onset GHD). Nine of these reports were SAEs, 2 of which were considered to be treatment-related. The first case was diabetes mellitus aggravation in a 45 year old male with adulthood-onset combined GHD, following 4-6 months of GH therapy; Omnitrope® was permanently discontinued. The second case was worsening of diabetes mellitus in a male aged 72 years with adulthood-onset combined GHD, following 19 years of GH therapy; Omnitrope® treatment was interrupted. Conclusions: Based on this interim analysis, Omnitrope® treatment in adults with GHD is well tolerated in a real-life clinical practice setting, irrespective of pre-treatment status. The ongoing PATRO Adults study will provide further data on the diabetogenic potential and overall safety of long-term GH treatment in this population.