PATRO children, a multi-center, non-interventional study of the safety and effectiveness of Omnitrope® (somatropin) treatment in children: update on the United States cohort.

Philippe Backeljauw,Markus Zabransky,Kim Campbell,Hichem Zouater,Richard Levy,Bradley S Miller,Kenneth Mccormick

doi:10.1515/jpem-2020-0360

Abstract

Omnitrope® (somatropin, Sandoz Inc.) is one of several recombinant human growth hormones (rhGH) approved in the United States (US) for use in pediatric indications, including growth hormone deficiency (GHD) and idiopathic short stature (ISS). We report data on the effectiveness and safety of Omnitrope® in the US cohort of the PATRO Children (international, longitudinal, non-interventional) study. All visits and assessments are carried out according to routine clinical practice, and doses of Omnitrope® are given according to country-specific prescribing information. By September 2018, 294 US patients were recruited; the two largest groups were GHD (n=193) and ISS (n=62). Across all indications, HSDS improvement (ΔHSDS) from baseline at three years was +1.0 (rhGH-naïve, +1.2; pre-treated, +0.7). In pre-pubertal patients, ΔHSDS from baseline at three years was +0.94 (rhGH-naïve, +1.3; pre-treated, +0.7). Following three years of treatment, ΔHSDS from baseline was +1.3 in rhGH-naïve GHD patients and +1.1 in rhGH-naïve ISS patients. In pre-pubertal rhGH-naïve patients, ΔHSDS from baseline was +1.3 and +1.2 in GHD and ISS patients, respectively. Overall, 194 patients (66.0%) experienced adverse events (AEs; n=886 events); most were of mild-moderate intensity. Five patients (1.7%) had AEs that were suspected to be treatment-related (n=5 events). All reported neoplasms were benign, non-serious, and considered unrelated to rhGH therapy. No AEs of diabetes mellitus or hyperglycemia were reported. Omnitrope® appears to be well tolerated and effective in the majority of patients, without evidence of an increased risk of developing unexpected AEs, diabetes mellitus, or new malignancies during treatment.

Highlights

Omnitrope® is a recombinant human growth hormone approved by the United States Food and Drug Administration (US FDA) in 2006
Five patients (1.7%) had adverse events (AEs) that were suspected to be treatment-related (n=5 events)
A recombinant human growth hormones (rhGH) was first approved in 1985 to treat children with growth failure caused by inadequate secretion of endogenous growth hormone [1, 2]. rhGH therapy was subsequently approved in the US for use in various other pediatric indications, including Turner syndrome (TS) in 1996, Prader–Willi syndrome (PWS) in 2000, children born small for gestational age (SGA) in 2001, and children with idiopathic short stature (ISS) in 2003 [2]

Summary

Introduction

Omnitrope® (somatropin, Sandoz Inc.) is a recombinant human growth hormone (rhGH) approved by the United States Food and Drug Administration (US FDA) in 2006. A rhGH was first approved in 1985 to treat children with growth failure caused by inadequate secretion of endogenous growth hormone (growth hormone deficiency; GHD) [1, 2]. TS, SGA, and ISS are not characterized by a deficiency in endogenous GH; rhGH treatment has been shown to improve linear growth and adult height in patients with these conditions [3]. RhGH has been used for many years to treat growth disorders in children, some concerns have been raised about its safety, in relation to its. The PAtients TReated with Omnitrope® (PATRO) Children study is a post-marketing surveillance program for Omnitrope®, which began in 2006 [5]. We report effectiveness and safety results from an analysis (September 2018) of patients enrolled in the PATRO Children study in the US

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