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Adult height in children born small for gestational age (SGA) with persistent short stature (SGA-SS). Growth hormone treatment outcomes from data of the Czech countrywide REPAR database


Authors: Toni Ledjona 1,AnetaKodytková 1;  Plachý Lukáš 1;  Jírová Barbora 1;  Hainerová Aldhoon Irena 2;  Aleksijevi Darina 3;  Černá Jana 4;  Dvořáková Marcela 5;  El-Lababidi Eva 2;  Kalvachová Božena 5;  Kocourková Kamila 6;  Kotvalová Ivana 7;  Magnová Olga 8;  Novotná Dana 8;  Obermannová Barbora 1;  Pomahačová Renata 9;  Průhová Štěpánka 1;  Souček Ondřej 1;  Strnadel Jiří 4;  Šíchová Lucie 10;  Škvor Jaroslav 10;  Šnajderová Marta 1;  Šumník Zdeněk 1;  Zapletalová Jiřina 3;  Šilar Jiří 11;  Koloušková Stanislava 1;  Lebl Jan 1
Authors‘ workplace: Pediatrická klinika, 2. lékařská fakulta, Univerzita Karlova a Fakultní nemocnice v Motole, Praha 1;  Klinika dětí a dorostu, 3. lékařská fakulta, Univerzita Karlova a Fakultní nemocnice Královské Vinohrady, Praha 2;  Dětská klinika, Lékařská fakulta, Univerzita Palackého a Fakultní nemocnice Olomouc 3;  Klinika dětského lékařství, Lékařská fakulta, Ostravská univerzita a Fakultní nemocnice Ostrava 4;  Endokrinologický ústav, Praha 5;  Dětské oddělení, Nemocnice České Budějovice 6;  Dětská klinika, Lékařská fakulta, Univerzita Karlova a Fakultní nemocnice Hradec Králové 7;  Pediatrická klinika, Lékařská fakulta, Masarykova univerzita a Fakultní nemocnice Brno ¨ 8;  Dětská klinika, Lékařská fakulta, Univerzita Karlova a Fakultní nemocnice Plzeň 9;  Dětská klinika, Masarykova nemocnice, Krajská zdravotní, a. s., Ústí nad Labem 10;  Institut biostatistiky a analýz, s. r. o., Brno #tyto dvě autorky přispěly ke vzniku článku stejným dílem 11
Published in: Čes-slov Pediat 2023; 78 (S3): 22-29.
Category: Original Papers
doi: https://doi.org/10.55095/CSPediatrie2023/059

Overview

Of children born small for gestational age (SGA) with birth weight and/or birth length below −2 SD, about 10% fail to catch-up after birth. These children remain short over childhood (with height standard deviation score [ht-SDS] below −2.5 SDS) and end up as short adults. They are assigned as SGA-SS (small for gestational age with short stature). European Medicine Agency approved growth hormone therapy in SGA-SS already in 2003.

Out of 1502 SGA-SS children that are registered in the Czech national database of growth hormone recipients REPAR, 397 children (221 boys) already completed growth and achieved near-final height. Growth hormone therapy (median dose 0.034 mg/kg/day) was initiated at age 8.5 years in boys (median; P5-95: 3.2-15.0) and 7.3 years in girls (3.9-13.5). The mean ht-SDS at treatment initiation was −3.21 (SD: 0.91). Treatment was terminated at age 16.2 years in boys (median; P5-95: 14.0-18.7) and 14.3 years in girls (12.4-17.3).

By treatment termination, the patients have had gained 1.59 ht-SDS (mean) and have achieved near-final height −1.62 ht-SDS (SD: 0.97; p<0.0001 if compared to pre-treatment). Thus, majority of patients exceeded −2 SDS at treatment completion and achieved height within normal limits. After adjusting for ht-SDS at age 18 years, their height corresponded to ht-SDS −2.30 (SD: 1.09; p < 0.0001 if compared to pre-treatment). Growth hormone therapy was successful even after this adjustment that mildly underestimates the treatment effect, and led to mean height increase of 0.91 ht-SDS.

Analysis results of the REPAR database confirm efficacy of growth hormone therapy in SGA-SS children and its potential to significantly improve the adult height.


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