Practical tools to identify short children born small-for-gestational-age eligible for rhGH treatment according to Italian regulation

Tornese, Gianluca; Pricci, Flavia; Pellegrin, Maria Chiara; Villa, Marika; Rotondi, Daniela; Agazio, Elvira; Barbi, Egidio

doi:10.1186/s13052-019-0715-x

Cited by 3 publications

(8 citation statements)

References 10 publications

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“…The prevalence in the general pediatric population of children born SGA who qualify for rhGH treatment at 4 years of age in our cohort was 1:3250, smaller than the hypothetical one (1:417) [ 17 ] and than previously reported (1:1800 in Japan at 3 years of age) [ 2 ], but still very far from the stated prevalence of children treated with rhGH with SGA indication in Italy (0.37/100,000) [ 15 ]. Intriguingly, all children with short stature at the age of 4 years were born at term, and if we had considered the age of 2 years (as in the USA) or 3 years (as in Japan), no children would have qualified for rhGH treatment, considering all the remaining criteria.…”

Section: Discussioncontrasting

confidence: 74%

“…The hypothetical prevalence of short children born SGA at the age of 2 years would be 0.24% (12% of 2%, 1:417) [ 15 ]; however, no study evaluated the prevalence of SGA children with short stature who qualify for rhGH treatment in Europe, so far. Only one Japanese study on a cohort of nearly 30,000 children re-evaluated at 3 years of life verified the prevalence of children with short stature born SGA of 0.06% (1:1800) (notably higher in preterm births < 34 weeks of GA, 0.39%, 1:256) [ 2 ].…”

Section: Introductionmentioning

confidence: 99%

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Prevalence of children born small for gestational age with short stature who qualify for growth hormone treatment

Tamaro

Pizzul²,

Gaeta³

et al. 2021

Ital J Pediatr

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Background Recombinant human growth hormone (rhGH) is approved in Europe as a treatment for short children born small for gestational age (SGA) since 2003. However, no study evaluated the prevalence of SGA children with short stature who qualify for rhGH in Europe so far. This study aimed to investigate in an Italian population the prevalence of children born SGA, of short stature in children born SGA, and of SGA children who qualify for rhGH treatment at 4 years of age. Methods We conducted a population-based study on primary care pediatricians’ databases in Trieste, Italy. Data was collected on 3769 children born between 2004 and 2014. SGA was defined as birth weight and/or birth length ≤ − 2 SDS. Data on height and weight were registered at the closest well-being visit to 1, 2, 3, 4 years of age. Short stature was defined as height ≤ − 2 SDS. Short children born SGA who qualify for rhGH treatment were identified according to Note AIFA #39 criteria (age ≥ 4 years; height ≤ − 2.5 SDS; growth velocity < 50th percentile). Results Full data at birth were available for 3250 children. The SGA prevalence was 3.6% (0.8% SGA for weight, 2.2% SGA for length, 0.6% SGA for both weight and length). The prevalence of short stature among SGA children was 9% at 1 year of age, 6% at 2 years (significantly higher in preterm in the first 2 years), 4% at 3 years, 3% at 4 years (all born at term). At 4 years of age, median height SDS was − 0.52. One child born SGA was eligible for GH treatment (0.8% among SGA children). Conclusions The prevalence in a general pediatric population of children born SGA who qualify for GH treatment was 1:3250. Although the prevalence of SGA in our population was similar to previous studies, catch-up growth was recorded earlier in our sample compared to previous reports, and term babies had late catch-up. Height SDS of children born SGA at 4 years of age was lower than expected (− 0.52 SDS).

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Section: Discussioncontrasting

confidence: 74%

Section: Introductionmentioning

confidence: 99%

Prevalence of children born small for gestational age with short stature who qualify for growth hormone treatment

Tamaro

Pizzul²,

Gaeta³

et al. 2021

Ital J Pediatr

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“…Twenty-five articles were excluded after full-text screening, while 25 articles were included in the final analysis. A list of excluded studies along with reasons for exclusion is provided in S2 Table . Among the 25 included studies, 9 studies [7,13,14,17,19,25,[28][29][30] were used to address the research question 1 (epidemiology), 10 studies [6, 8-12, 18, 20, 22, 23] the research question 2 (adherence), 5 studies [15,16,21,26,28] the research question 3 (economic impact), and 4 studies [8,21,24,27] the research question 4 (quality of life); three studies [8,21,28] contributed for more than one research question. The literature selection process is depicted in the Fig 1…”

Section: Resultsmentioning

confidence: 99%

“…To address the research question 1 (epidemiology), we identified 9 studies [7,13,14,17,19,25,[28][29][30], 5 of which were secondary studies [14,25,[28][29][30] and 4 were primary studies [7,13,17,19] reporting epidemiological data about the considered diseases. The main characteristics of the included studies are described in the Table 1 (secondary studies) and Table 2 (primary studies).…”

Section: Epidemiologymentioning

confidence: 99%

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Pediatric growth hormone treatment in Italy: A systematic review of epidemiology, quality of life, treatment adherence, and economic impact

Orso¹,

Polistena

Granato

et al. 2022

PLoS ONE

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Objectives This systematic review aims to describe 1) the epidemiology of the diseases indicated for treatment with growth hormone (GH) in Italy; 2) the adherence to the GH treatment in Italy and factors associated with non-adherence; 3) the economic impact of GH treatment in Italy; 4) the quality of life of patients treated with GH and their caregivers in Italy. Methods Systematic literature searches were performed in PubMed, Embase and Web of Science from January 2010 to March 2021. Literature selection process, data extraction and quality assessment were performed by two independent reviewers. Study protocol has been registered in PROSPERO (CRD42021240455). Results We included 25 studies in the qualitative synthesis. The estimated prevalence of growth hormone deficiency (GHD) was 1/4,000–10,000 in the general population of children; the prevalence of Short Stature HOmeoboX Containing gene deficiency (SHOX-D) was 1/1,000–2,000 in the general population of children; the birth prevalence of Turner syndrome was 1/2,500; the birth prevalence of Prader-Willi syndrome (PWS) was 1/15,000. Treatment adherence was suboptimal, with a range of non-adherent patients of 10–30%. The main reasons for suboptimal adherence were forgetfulness, being away from home, pain/discomfort caused by the injection. Economic studies reported a total cost for a complete multi-year course of GH treatment of almost 100,000 euros. A study showed that drug wastage can amount up to 15% of consumption, and that in some Italian regions there could be a considerable over- or under-prescribing. In general, patients and caregivers considered the GH treatment acceptable. There was a general satisfaction among patients with regard to social and school life and GH treatment outcomes, while there was a certain level of intolerance to GH treatment among adolescents. Studies on PWS patients and their caregivers showed a lower quality of life compared to the general population, and that social stigma persists. Conclusion Growth failure conditions with approved GH treatment in Italy constitute a significant burden of disease in clinical, social, and economic terms. GH treatment is generally considered acceptable by patients and caregivers. The total cost of the GH treatment is considerable; there are margins for improving efficiency, by increasing adherence, reducing drug wastage and promoting prescriptive appropriateness.

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Long-term safety and effectiveness of a somatropin biosimilar (Omnitrope®) in children requiring growth hormone therapy: analysis of final data of Italian patients enrolled in the PATRO children study

Iughetti,

Antoniazzi,

Giavoli

et al. 2024

Endocrine

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Practical tools to identify short children born small-for-gestational-age eligible for rhGH treatment according to Italian regulation

Cited by 3 publications

References 10 publications

Prevalence of children born small for gestational age with short stature who qualify for growth hormone treatment

Prevalence of children born small for gestational age with short stature who qualify for growth hormone treatment

Pediatric growth hormone treatment in Italy: A systematic review of epidemiology, quality of life, treatment adherence, and economic impact

Long-term safety and effectiveness of a somatropin biosimilar (Omnitrope®) in children requiring growth hormone therapy: analysis of final data of Italian patients enrolled in the PATRO children study

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