ESPE Abstracts

The availability of biosynthetic growth hormone (GH) ensures that children who are deficient can have replacement therapy, but it also has created the opportunity to treat children who are short but do not have a deficiency. Non-GH deficient short stature, without treatment, the height outcomes in most studies have failed to reach mid-parental target height. GH therapy resulted in mixed height outcomes; some reached genetic target height whereas others failed. The aim of this ...

Background: Pre- and postnatal growth retardation of unknown pathogenesis is a common clinical feature in patients with Williams-Beuren syndrome (WBS). However, growth hormone deficiency (GHD) has not been considered a major cause of growth retardation.Case reports: We report two female patients with confirmed WBS who had defective GH secretion in response to two provocative tests and low IGF-I level and their growth response to GH therapy for 9 years. T...

Introduction: Treatment with recombinant growth hormone (rGH) is safe and has greatly improved the approach of children and adolescents with growth hormone deficiency (GHD) and other growth disorders. Some studies show that most of the height gain associated with GH treatment occurs in prepubertal years. The aim of our study was to evaluate the effect of age at start of the treatment on final height in children with isolated or GHD in a Portuguese cohort.<p class="abstext"...

Background: Recombinant human growth hormone (r-hGH) treatment can optimise growth potential; however, optimum outcomes are not always achieved owing to several reasons, including poor adherence. This analysis sought to operationalize psychosocial drivers of non-adherence in GH patients, using the three-component, evidence-based capability, opportunity and motivation behavioural framework (COM-b). The framework allows for matching of specific interventions to help modify behav...

Introduction: Growth prediction algorithms (i.e. iGRO), provide an estimate of a patients’ likely growth in the first year, and subsequent years, of GH treatment at a given dose, taking into account the patient’s combination of physical characteristics. Comparing a patient’s actual growth with their predicted growth after the first year of GH treatment, it is possible to determine whether the patient is responding to GH as expected (Index of responsiveness; IoR)...

The Easypod™ Connect Observational Study (ECOS) was the first global study of easypod™, currently the only electronic injection device for recombinant human growth hormone (r-hGH; Saizen®). ECOS reported accurate and robust real-time adherence data in a large cohort of patients. In this analysis, we assess the adherence of r-hGH administered via easypod™ in a cohort of Greek patients from ECOS (EMR200104-520, NCT01363674). Patients aged 2–18...

Introduction: Short stature is frequently associated to neurofibromatosis (NF). In these patients this condition is often caused by growth hormone (GH) deficiency. We describe four boys affected by NF type 1 and GH deficiency treated with GH replacement therapy.Case report: GH deficiency was diagnosed in four patients with NF type 1, who were referred to our Pediatric Endocrinology Unit for short stature. Patient 1 started GH replacement therapy at the C...

Introduction: The nutritional status of a child is essential for the overall development and in particular for the statural growth. It was previously reported that growth hormone (GH) administration in children with growth hormone deficiency (GHD) could have a beneficial effect on body mass index (BMI) in both underweight and overweight children, suggesting a complex interplay between nutrition and growth. Therefore, the aim of the study was to analyze the influence of BMI on ...

Objective: Premature GH treatment discontinuation of SGA is usually linked to safety or ineffectiveness. However, this population is poorly addressed compared to those with final adult height (FAH). Authors investigated the journey of SGA prematurely discontinued Norditropin® treatment in a French real-life.Methods: Observational prospective ongoing study: 291 Norditropin®-treated SGA. Annual FU up to FAH. Descriptive anal...

Objectives: This study was performed to evaluate effectiveness on near-adult height (NAH) and safety of recombinant human growth hormone (rhGH) (Eutropin® Inj., Eutropin®Plus Inj., Eutropin®AQ Inj., LG Chem, Ltd.) treatment in children with growth hormone deficiency (GHD) and Turner syndrome (TS).Methods: The LG Growth Study (LGS) is a multicenter, long-term, observational study designed to evaluate the lon...

This study aimed to evaluate final adult height after recombinant growth hormone (GH) treatment in girls with Turner syndrome (TS) and elucidate the contributing factors to growth response. (Seventy-four patients with Turner syndrome who were treated with GH and reached adult height and 18 patients without treatment were enrolled in this study. To determine final height gain, we assessed the difference between the final height standard deviation score (SDS) and height SDS at t...

Background: Severe primary deficiency of insulin-like growth factor-1 (IGFD) being characterized by growth failure and short stature in children, constitutes an indication to recombinant human IGF-1 (mecasermin) treatment. It is defined by serum insulin-like-growth factor-1 (IGF-1) levels less than or equal to 2.5 th percentile, height less than or equal to −3 S.D.S., normal growth hormone (GH) secretion and exlusion of secondary causes of IGFD.<p clas...

Introduction: Short stature is defined as stature less than −2 standars deviations (SD) for a person’s age and sex of the reference population. Short for gestational age children (SGA) represent 20% of all children with short stature. 10% of these can not catch-up and remains their height below −2 SD. Growth hormone (GH) treatment is a recognized therapy for SGA children authorized in Europe at 4 years old. There are studies that support that younger children ...

Objective: Age and height at treatment start, target height, GH dose, and first year treatment response are among known criteria of GH deficiency (GHD) good responders (final adult height [FAH] >−2 standard deviation score [S.D.S.]) to GH treatment (GHT). The authors investigated whether the same criteria are applicable to SGA patients based on real-life ongoing French registry data.Methods: 291 SGA children treated with Norditro...

Introduction: Growth velocity is reduced in patients with GH deficiency and this may result in an increase in Body Mass Index (BMI). Treatment performed with Growth Hormone (GH) while accelerating growth velocity, might reduce BMI. The objective of this study was to evaluate BMI in patients with GHD at diagnosis, 1 y and 2 y after started treatment with GH and to compare if there is difference between the BMI of the patients with and without pituitary abnormalities.<p clas...

Background/Aims: An increase in growth rate in children suffering from growth hormone deficiency (GHD) subjected to recombinant growth hormone treatment (rGHT) was shown to be accompanied by acceleration of metabolic processes that may stimulate hematopoiesis. Therefore, the aim of the present study was to examine the effects of one year rGHT on erythropoietin (EPO) and Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) levels in GHD children.Meth...

Background: Growth hormone deficiency (GHD) in children is a rare condition, which requires pathogenic therapy. In Russia GH treatment (GHT) is part of a federal program called “Seven high expenditure diseases” (7HED) and is fully state funded. In the rare cases when a GHD child cannot be treated with GH, financial and medical support for the child and its family is provided by the state. It is therefore important to understand the cost-effectiveness of GHT for child...

Background: Vitamin D status in children with isolated GHD has been analyzed in few studies with controversial results. The aim of the study was to assess vitamin D status in children with idiopathic GHD in North and Central Greece.Materials and methods: 128 children (M/F: 76/61, mean age 9.5 (S.D.±3.5 years) with isolated GHD were compared with 65 controls (M/F: 46/3, mean age 9.3 (S.D.±3.2 years). Children were d...

Background: CinnaTropin® (CinnaGen, Iran) is a recombinant human GH manufactured in Iran. Herein, we compared the efficacy and safety of the CinnaTropin® with the corresponding reference (Nordilet® Norditropin, Novo Nordisk, Denmark) in children with idiopathic GH deficiency (IGHD) and multiple pituitary hormone deficiency (MPHD).Methods: This was a randomized, open-label and cross-over study. Eligible pati...

Introduction: Recombinant GH is an effective treatment for short children who are born small for gestational age(SGA). Short children SGA who fail catch-up growth by 4 years of age are candidates for GH treatment, at a dose of 35–70 μg/kg per day. Factors associated with response to GH treatment during the initial 2–3 years of therapy include age and height standard deviation scores at the start of therapy, midparental height, and GH dose. It is important to kno...

Objective: Bang et al. showed that Δ height and growth velocity (GV) the first year of treatment could be predictive factors of statural response in SGA (n=54). Poor responders showed GV <1SDS (55%) and Δ height <0.5SDS (45%). Moreover, Ortego et al. seems to confirm the suitability interest of KIGS mathematical model in a retrospective SGA cohort (n=103) showing that the percentage of good responders in the first year varies betwee...

Introduction: Growth retardation as clinical manifestation among children with acquired hypothyroidism is observed later in the course of the illness. Thyroid hormones along with insulin like growth factors (IGF) have important role in somatic and skeletal growth. Studies addressing role of IGF’s towards growth retardation in children with acquired hypothyroidism are limited.Objective: To evaluate effect of Insulin like growth factors in children wi...

2 siblings were referred for evaluation fo short stature and failure to thrive. Both were born of 3rd degree consanguinity, first and second in birth order. the first sibling was 2 1/2 year old at time of referral and had birthweight of 3.1 kg and had gross motor delay. Present height was 65 cm (SDS −6.2 S.D.)and weight was 6 kg (<3rd centile) Second sibling was 1.5 years old, with gross motor delay with height of 57 cm (SDS −6.5 S.D.</sma...

Treatment with Recombinant Human GH (rhGH) has been of significant value in promoting quality of life in children with GH deficiency. However, it has been associated with several side-effects in the literature, including hypothyroidism, usually transient during the replacement therapy. The aim of this study was to evaluate the side effects of hGH replacement therapy, among children who were followed up at the Pediatric Endocrinology Outpatients Unit of our hospital during the ...

This study aims to investigate the efficacy and safety of recombinant human GH (rhGH) in the treatment of idiopathic short stature (ISS).Methods: The data of 200 ISS children, who were treated with rhGH from January 2008 to December 2016, were collected and retrospectively analyzed. The data of height, bone age, blood glucose, insulin, thyroid function and IGF-1 were collected, and annual growth rate (AGR), height standard deviation score (HtSDS) and rel...

Background: The efficacy and safety of rhGH treatment on ISS and GHD were not reported in Chinese children. In this study, we aimed to compare the efficacy and safety of rhGH therapy in ISS and GHD.Methods: The clinical data in children with ISS and GHD who were treated with rhGH for more than one year from 2005 to 2016 were retrospectively analyzed. Growth velocity (GV), HtSDS, IGF1 SDS, BMI and the incidence of fasting hyperglycemia, fasting hyperinsul...

Background: Although the beneficial effects of GH treatment on statural growth are well known, the impacts on skeletal maturation are not fully understood. In the current study, we aimed to investigate the progression of bone age (BA) in children with idiopathic GH deficiency (iGHD) and idiopathic short stature (ISS) based on a LG Growth Study (LGS). We also evaluated the progression rate of BA relative to chronologic age (CA) between iGHD and ISS and to find their associated ...

Several studies have evaluated the effects of growth hormone (GH) on auxological and biochemical parameters in children with non-GH-deficient, idiopathic short stature (ISS). This study evaluated the efficacy and safety of GrowtropinR-II (recombinant human GH) in Korean patients with ISS. This was a 1-year, open-label, multicenter, phase III randomized trial of Growtropin-II in Korean patients with ISS. In total, 70 prepubertal subjects (39 males, 31 females) betwee...