Data analysis of REPAR – country-wide register of growth hormone treatment recipients

Česká verzia

Authors: A. Kodýtková ¹; E. Al Lababidi ²; I. Čermáková ³; J. Černá ⁴; J. Čížek ⁵; M. Dvořáková ³; B. Kalvachová ³; O. Magnová ⁶; D. Neumann ⁷; D. Novotná ⁶; B. Obermannová ¹; I. Plášilová ⁷; R. Pomahačová ⁸; Š. Průhová ¹; J. Strnadel ⁴; J. Škvor ⁹; M. Šnajderová ¹; Z. Šumník ¹; J. Zapletalová ¹⁰; K. Kusalová ¹¹; J. Šilar ¹¹; D. Zemková ¹; J. Lebl ¹; S. Koloušková ¹
Authors place of work: Pediatrická klinika 2. lékařské fakulty Univerzity Karlovy a Fakultní nemocnice Motol, Praha ¹; Klinika dětí a dorostu 3. lékařské fakulty Univerzity Karlovy a Fakultní nemocnice Královské Vinohrady, Praha ²; Endokrinologický ústav, Praha ³; Klinika dětského lékařství Lékařské fakulty Ostravské univerzity a Fakultní nemocnice s poliklinikou, Ostrava ⁴; Dětské oddělení nemocnice České Budějovice ⁵; Pediatrická klinika Lékařské fakulty Masarykovy univerzity a Fakultní nemocnice Brno ⁶; Dětská klinika Lékařské fakulty Univerzity Karlovy a Fakultní nemocnice Hradec Králové ⁷; Dětská klinika Lékařské fakulty Univerzity Karlovy a Fakultní nemocnice Plzeň ⁸; Dětská klinika Masarykovy nemocnice, Krajská zdravotní, a. s., Ústí nad Labem ⁹; Klinika dětského lékařství Lékařské fakulty Univerzity Palackého a Fakultní nemocnice Olomouc ¹⁰; Institut biostatistiky a analýz, s. r. o., Brno ¹¹
Published in the journal: Čes-slov Pediat 2020; 75 (4): 205-212.
Category:

Summary

Background: Since 2014, the Czech national registry REPAR has been collecting longitudinal data on patients treated with recombinant growth hormone (rhGH). It contains converted data from terminated international databases KIGS, Nordinet® IOS, GeNeSIS, ECOS and new subsequently added patients.

Objective: The aim of this study is to present the basic demographic data of patients included in the REPAR database and to analyze selected longitudinal data.

Methods: Data from all ten pediatric clinical centers for the rhGH treatment in the Czech Republic are entered into web-based database via web case report forms. The database contains data before the start of the treatment and data from follow-up visits until treatment termination. Patients belong to the one of six pediatric indication groups – growth hormone deficiency (GHD), Turner syndrome (TS), chronic renal failure (CRI), postnatal growth failure following intrauterine growth restriction (SGA), Prader-Willi syndrome (PWS) and SHOX deficiency.

Results: By January 1st, 2020, the REPAR register contained data on 4330 pediatric patients, of which 1896 were currently treated (1130 boys, 766 girls). The dose of rhGH varied from 0.020 to 0.046 mg/kg/day (5th and 95th percentiles; median 0.033 mg/kg/day). In currently treated patients, treatment was initiated at age 5.8 years (median; 0.2–13.4 years, 5th and 95th percentiles), the earliest in PWS (girls 1.5 years, boys 1.3 years) and at the latest in boys with CRI (8.0 years) and also in girls with SHOX deficiency (8.3 years). The age at treatment onset was 7.2 years (girls) and 7.5 years (boys) in GHD, 6.6 years in TS, and 6.4 years (boys) and 6.2 years (girls) in children born SGA on average. In GHD, TS and PWS, the age at treatment initiation decreases significantly over time (p<0.001). At the end of treatment, the median height reaches -1.8 SDS in SGA patients and -1.5 SDS in girls with TS.

Conclusion: Analyses of data from the registry contribute to an overview of numbers of treated patients, but also to the evaluation of the clinical recognition of growth disorders in pediatric practice. They can serve to better understanding of physiological effects of growth hormone, to analyzing predictors of therapy success, and also to pharmacoeconomic assessments.

Keywords:

Czech Republic – treatment – database – REPAR – Growth hormone

Zdroje

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