Rationale and design of PATRO Adults, a multicentre, noninterventional study of the long-term efficacy and safety of Omnitrope^® for the treatment of adult patients with growth hormone deficiency

Abstract

Abstract:Objective: To describe the rationale and design of PATRO Adults, a postmarketing surveillance study of the long-term efficacy and safety of somatropin (Omnitrope ® ) for the treatment of adult patients with growth hormone deficiency (GHD). Methods: PATRO Adults is an observational, multicentre, open, longitudinal, noninterventional study being conducted in hospitals and specialized endocrinology clinics across several European countries. The primary objective is to assess the safety and efficacy of Omnitrope ® in adults treated in routine clinical practice. Eligible patients are male or female adults who are receiving treatment with Omnitrope ® and who have provided informed consent. Patients who have been treated with another human growth hormone (hGH) product before starting Omnitrope ® therapy will also be eligible for inclusion. Efficacy assessments will be based on the analysis of the following: insulin-like growth factor-1 levels within age-and gender-adjusted normal ranges; anthropometric measures (weight, waist circumference, total fat mass, lean body mass, total body water); bone mineral density; lipids; effects on cardiovascular risk factors such as glucose metabolism, blood pressure and inflammatory markers (e.g. C-reactive protein); and quality of life. All adverse events will be monitored and recorded. Particular emphasis will be placed on long-term safety, the recording of malignancies, the occurrence and clinical impact of antirecombinant hGH antibodies, the incidence, severity and duration of hyperglycaemia, and the development of diabetes during treatment with Omnitrope ® .

Long-term safety and efficacy of Omnitrope® in adults with growth hormone deficiency: Italian interim analysis of the PATRO Adults study

“…The design and methods of this study have been published in detail previously [9]. Briefly, eligible patients are adults (≥15 years old) receiving treatment with Omnitrope® for GHD (isolated or combined with other hormone deficiencies) and who provided written informed consent.…”

Malignancy risk in adults with growth hormone deficiency undergoing long-term treatment with biosimilar somatropin (Omnitrope®): data from the PATRO Adults study

“…The design of the PATRO Adults study has been described in detail elsewhere. 11 Briefly, eligible patients are male and female adults in receipt of rhGH treatment in accordance with the recommendations in the Summary of Product Characteristics. 12 The study is performed in accordance with the ethical standards as laid down in the 1964 Declaration of Helsinki and its later amendments or comparable ethical standards.…”

“…Secondary objectives include monitoring effectiveness parameters, including IGF-I levels, lipid profile, body composition and quality of life. 11 As Omnitrope ® was approved in Europe as a biosimilar rhGH, the study is also important for confirming that its long-term safety profile in adults with GHD is comparable to that of the reference medicine. This paper focuses on safety, and specifically the occurrence of malignancies in the PATRO Adults population, based on an interim data analysis performed in July 2018.…”

“…PAtients TReated with Omnitrope ® (PATRO) Adults is an ongoing post-marketing surveillance study conducted since 2007 in hospitals and specialized endocrinology clinics across Europe. 11 The primary objective of PATRO Adults is to assess the long-term safety of rhGH (Omnitrope ® ) in adults with GHD treated in routine clinical practice. Secondary objectives include monitoring effectiveness parameters, including IGF-I levels, lipid profile, body composition and quality of life.…”

Multidisciplinary approach to evaluating immunogenicity of biosimilars: lessons learnt and open questions based on 10 years' experience of the European Union regulatory pathway

“…21 In accordance with the provisions of the risk management plan, the sponsor has initiated noninterventional post-marketing studies in children and in adults that will include monitoring for anti-growth hormone antibodies and the potential clinical effects of such antibodies. 22,23 Given the known association between anti-infliximab antibodies, reduced efficacy, and increased incidence of infusion-related reactions, it is pertinent to question whether there could be a differential risk for a biosimilar product relative to the reference product in alternative therapeutic settings. For example, controlled clinical studies performed using patients who receive less immunosuppressive medication might reveal differences in immunogenicity that are not apparent in a patient population receiving a more intensive concomitant immunosuppressive regimen.…”