ESPE Abstracts (2015) 84 P-2-197

Evaluation of Medical Treatment in the First 2 Years of Life with a New Dutch National Longitudinal Registry for Children with Congenital Adrenal Hyperplasia (CAH)

Annelieke van der Lindea, Erica van den Akkerf, Willy Bakker- van Waardee, Sabine Hannemaj,f, Gea Hoorweg-Nijmank, Hetty van de Kampc, Daniel Klinkg, Roelof Odinkh, Saartje Straetemansi, Paul van Trotsenburgd, Paul Verkerkb & Hedi Claahsen-van der Grintena


aRadboudumc, Nijmegen, The Netherlands; bTNO, Zeist, The Netherlands; cUMC Utrecht, Utrecht, The Netherlands; dAMC, Amsterdam, The Netherlands; eUMCG, Groningen, The Netherlands; fErasmusmc, Rotterdam, The Netherlands; gVUmc, Amsterdam, The Netherlands; hCatharina Hospital Eindhoven, Eindhoven, The Netherlands; iMUMC, Maastricht, The Netherlands; jLUMC, Leiden, The Netherlands; kSt Antonius Hospital, Nieuwegein, The Netherlands


Background: Recently, a national database has been developed to register yearly data from all children detected with CAH in the neonatal screening program from 2002 onwards. So far longitudinal data of 105 children have been registered (roughly 65% of Dutch CAH patients) to evaluate medical treatment and long-term effects in CAH. A national CAH work group developed guidelines for diagnostics and follow up in CAH.

Objective and hypotheses: Aim of our current study is to evaluate the dosage of hydrocortisone, fludrocortisones and salt supplementation in CAH children in the first 2years of life.

Method: This is a descriptive study in 105 children. The hydrocortisone, fludrococrtisone and salt medication was evaluated at the age of six months (T 0.5), 12 months (T1) and 24 months (T2). The treatment approaches of different centers was compared.

Results: T 0.5: (n=95): median HC dosage (n=89) 4 mg/day. Interquartile range (IQR) 2 mg/day. FC (n=82) median 93.75 mcg/day; IQR 37.5 mcg/day and NaCl (n=46) median 500 mg; IQR 400 mg/day. T 1: (n=90) median HC dosage (n=88) 4 mg/day; IQR 1 mg/day, FC (n=84) median 62.5 mcg/day; IQR 30.3 mcg/day and NaCl (n=25) median 375 mg/day; IQR 400 mg/day. T2: (n=88) median HC dosage (n=84) 5.4 mg/day; IQR 2.14 mg/day, FC (n=78) median 62.5 mcg/day; IQR 37.5 mcg/day. No salt medication was used after the first year of life. No significant differences between centers are observed.

Conclusion: Our analysis showed a stable slightly supraphysiological HC dosage over the first 2 years of life. FC shows a more widespread range in dosage suggesting a more individualized treatment approach without a clear difference between national centres. Further studies will focus on the relation between medication dosage and biometrical data. Our longitudinal database gives the opportunity to establish a more standardized care and long-term treatment evaluation.