Growth hormone therapy in children born small for gestational age: results from the ANSWER program.

Robert Rapaport,Giuseppe Piccoli,Judith Ross,Peter Lee,Paul Saenger,Vlady Ostrow

doi:10.1530/ec-18-0286

Abstract

Growth hormone (GH) is used to treat short stature and growth failure associated with growth disorders. Birth size and GH status variably modulate response to GH therapy. The aim of this study was to determine the effect of birth size on response to GH therapy, and to determine the impact of GH status in patients born small for gestational age (SGA) on response to GH therapy. Data from the prospective, non-interventional American Norditropin Studies: Web-Enabled Research (ANSWER) Program was analyzed for several growth outcomes in response to GH therapy over 3 years. GH-naïve children from the ANSWER Program were included in this analysis: SGA with peak GH ≥10 ng/mL (20 mIU/L), SGA with peak GH <10 ng/mL (20 mIU/L), isolated growth hormone deficiency (IGHD) born SGA, IGHD not born SGA and idiopathic short stature. For patients with IGHD, those who did not meet criteria for SGA at birth showed greater improvements in height SDS and BMI SDS than patients with IGHD who met criteria for SGA at birth. For patients born SGA, response to GH therapy varied with GH status. Therefore, unlike previous guidelines, we recommend that GH status be established in patients born SGA to optimize GH therapy.

Highlights

Treatment with recombinant human growth hormone (GH) is widely utilized for improving height in children with growth failure and conditions in which it is efficacious, including isolated growth hormone deficiency (IGHD), idiopathic short stature (ISS) and small for gestational age (SGA) (1, 2)
The analysis described here evaluated growth outcomes (HSDS, IGF-I standard deviation scores (SDS) and BMI SDS), bone age per chronological age (BA/CA) and GH dose over 3 years of treatment in the IGHD, ISS and SGA (GH-sufficient or -deficient) pediatric populations
The subpopulations in this analysis were SGA and GH sufficient, SGA with GH deficiency, IGHD born SGA, IGHD not born SGA and ISS. The majority of these patients were diagnosed with IGHD and not born SGA (n = 709); the second largest subpopulation was those diagnosed with IGHD who were born SGA (n = 86) (Table 1)

Summary

Introduction

Treatment with recombinant human growth hormone (GH) is widely utilized for improving height in children with growth failure and conditions in which it is efficacious, including isolated growth hormone deficiency (IGHD), idiopathic short stature (ISS) and small for gestational age (SGA) (1, 2). Clinical characteristics of these growth disorders often overlap, criteria for SGA can be distinguished from those of other GH disorders in that diagnosis is defined by having a birth weight and/or length of

Objectives

Methods

Results