Growth Hormone Dose Modulation and Final Height in Short Children Born Small for Gestational Age: French Real-Life Data

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Abstract

Introduction

Growth hormone (GH) therapy improves height outcomes in short children born small for gestational age (SGA); however, real-world data on long-term GH exposure are few.

Methods

We report results from an observational study (NCT01578135) including children born SGA, treated with GH at 126 sites in France, and followed up for >5 years until achieving final adult height (FAH) or until study termination. Primary endpoints were the proportion of patients with normal (>−2) height standard deviation score (SDS) at the last visit and with normal FAH SDS. Post hoc analyses were performed by multivariate logistic regression analysis with stepwise elimination to identify factors associated with GH dose modulation and normal height SDS achievement.

Results

Of 1,408 registered patients, a representative sample ( n = 291) was selected for long-term follow-up. At the last visit, 193/291 (66.3%) children achieved normal height SDS and 72/291 (24.7%) reached FAH. FAH SDS was >–2 for chronological age in 48 (66.7%) children and >–2 for adult age in 40 (55.6%) children. In the post hoc analyses, height SDS at the last visit was a significant determinant of whether GH dose had been modulated. Factors significantly associated with reaching normal height SDS were baseline height SDS (taller, better), age at treatment start (younger, better), treatment duration excluding discontinuation periods (longer, better), and absence of a chronic disease. Most (70%) adverse events were non-serious, with 39% considered possibly/probably related to GH treatment.

Conclusions

GH therapy was fairly effective in most short children born SGA. No new safety concerns were identified.

Related collections

Most cited references 38

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World Medical Association Declaration of Helsinki: ethical principles for medical research involving human subjects.

(2013)

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International Small for Gestational Age Advisory Board consensus development conference statement: management of short children born small for gestational age, April 24-October 1, 2001.

P. A. Lee, S Chernausek, A Hokken-Koelega … (2003)

To provide pediatric endocrinologists, general pediatricians, neonatologists, and primary care physicians with recommendations for the management of short children born small for gestational age (SGA). A 13-member independent panel of pediatric endocrinologists was convened to discuss relevant issues with respect to definition, diagnosis, and clinical management of short children born SGA. Panel members convened over a series of 3 meetings to thoroughly review, discuss, and come to consensus on the identification and treatment of short children who are born SGA. SGA is defined as birth weight and/or length at least 2 standard deviations (SDs) below the mean for gestational age ( 2 SD below the mean; this catch-up process is usually completed by the time they are 2 years of age. A child who is SGA and older than 3 years and has persistent short stature (ie, remaining at least 2 SD below the mean for chronologic age) is not likely to catch up and should be referred to a pediatrician who has expertise in endocrinology. Bone age is not a reliable predictor of height potential in children who are SGA. Nevertheless, a standard evaluation for short stature should be performed. A diagnosis of SGA does not exclude growth hormone (GH) deficiency, and GH assessment should be performed if there is clinical suspicion or biochemical evidence of GH deficiency. At baseline, insulin-like growth factor-I, insulin-like growth factor binding protein-3, fasting insulin, glucose, and lipid levels as well as blood pressure should be measured, and all aspects of SGA-not just stature-should be addressed with parents. The objectives of GH therapy in short children who are SGA are catch-up growth in early childhood, maintenance of normal growth in childhood, and achievement of normal adult height. GH therapy is effective and safe in short children who are born SGA and should be considered in those older than 2 to 3 years. There is long-term experience of improved growth using a dosage range from 0.24 to 0.48 mg/kg/wk. Higher GH doses (0.48 mg/kg/wk [0.2 IU/kg/d]) are more effective for the short term. Whether the higher GH dose is more efficacious than the lower dose in terms of adult height results is not yet known. Only adult height results of randomized dose-response studies will give a definite answer. Monitoring is necessary to ensure safety of medication. Children should be monitored for changes in glucose homeostasis, lipids, and blood pressure during therapy. The frequency and intensity of monitoring will vary depending on risk factors such as family history, obesity, and puberty.

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Management of the child born small for gestational age through to adulthood: a consensus statement of the International Societies of Pediatric Endocrinology and the Growth Hormone Research Society.

Alan Rogol, S Cianfarani, P Czernichow … (2007)

Low birth weight remains a major cause of morbidity and mortality in early infancy and childhood. It is associated with an increased risk of health problems later in life, particularly coronary heart disease and stroke. A meeting was convened to identify the key health issues facing a child born small for gestational age (SGA) and to propose management strategies. There were 42 participants chosen for their expertise in obstetrics, peri- and neonatal medicine, pediatrics, pediatric and adult endocrinology, epidemiology, and pharmacology. Written materials were exchanged, reviewed, revised, and then made available to all. This formed the basis for discussions at the meeting. Where published data were not available or adequate, discussion was based on expert clinical opinions. Each set of questions was considered by all and then discussed in plenary sessions with consensus and unresolved issues identified. The consensus statement was prepared in plenary sessions and then edited by the group chairs and shared with all participants. The diagnosis of SGA should be based on accurate anthropometry at birth including weight, length, and head circumference. We recommend early surveillance in a growth clinic for those without catch-up. Early neurodevelopment evaluation and interventions are warranted in at-risk children. Endocrine and metabolic disturbances in the SGA child are recognized but infrequent. For the 10% who lack catch-up, GH treatment can increase linear growth. Early intervention with GH for those with severe growth retardation (height sd score, <-2.5; age, 2-4 yr) should be considered at a dose of 35-70 microg/kg x d. Long-term surveillance of treated patients is essential. The associations at a population level between low birth weight, including SGA, and coronary heart disease and stroke in later life are recognized, but there is inadequate evidence to recommend routine health surveillance of all adults born SGA outside of normal clinical practice.

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Author and article information

Journal

Journal ID (nlm-ta): Horm Res Paediatr

Journal ID (iso-abbrev): Horm Res Paediatr

Journal ID (hwp): HRP

Journal ID (publisher-id): HRP

Title: Hormone Research in Pædiatrics

Publisher: S. Karger AG (Basel, Switzerland )

ISSN (Print): 1663-2818

ISSN (Electronic): 1663-2826

Publication date (Electronic): 11 April 2023

Publication date (Print): October 2023

Volume: 96

Issue: 5

Pages: 495-508

Affiliations

[a ]Department of Paediatric Medicine, University Hospital Angers, Angers, France

[b ]Department of Clinical Genetics and Infant Medicine, Medical School and University Hospital of the University of Lorraine, Nancy, France

[c ]Division of Pediatric Endocrinology and Metabolism, Lyon University Pediatric Hospital, Lyon, France

[d ]Pediatric and Adult Congenital Cardiology Unit, Children’s Hospital, Toulouse University Hospital, Toulouse, France

Author notes

Correspondence to: Régis Coutant, ReCoutant@ 123456chu-angers.fr

Article

Publisher ID: 530572

DOI: 10.1159/000530572

PMC ID: 10614259

PubMed ID: 37040726

SO-VID: 2f66ebc9-61f8-4aa1-9952-1199f47ee8db

License:

This article is licensed under the Creative Commons Attribution 4.0 International License (CC BY) ( http://www.karger.com/Services/OpenAccessLicense). Usage, derivative works and distribution are permitted provided that proper credit is given to the author and the original publisher.

History

Date received : 13 May 2022

Date accepted : 16 March 2023

Date: 2023

Page count

Figures: 2, Tables: 8, References: 39, Pages: 14

Funding

This work was supported by Novo Nordisk. The funder was involved in study design, collection, analysis and interpretation of data, and reviewing of the report.

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