International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: Data from the I-CAH registry

Irina Bacila; Nicole Freeman; Eleni Daniel; Marija Sandrk; Jillian Bryce; Salma Rashid Ali; Zehra Yavas Abali; Navoda Atapattu; Tania A. Bachega; Antonio Balsamo; Niels Birkebæk; Oliver Blankenstein; Walter Bonfig; Martine Cools; Eduardo Correa Costa; Feyza Darendeliler; Silvia Einaudi; Heba Hassan Elsedfy; Martijn J.J. Finken; Evelien Gevers; Hedi L. Claahsen-Van der Grinten; Tulay Guran; Ayla Güven; Sabine E. Hannema; Claire E. Higham; Violeta Iotova; Hetty J. van der Kamp; Marta Korbonits; Ruth E. Krone; Corina Lichiardopol; Andrea Luczay; Berenice Bilharinho Mendonca; Tatjana Milenkovic; Mirela C. Miranda; Klaus Mohnike; Uta Neumann; Rita Ortolano; Sukran Poyrazoglu; Ajay Thankamony; Jeremy W. Tomlinson; Ana Vieites; Liat de Vries; S. Faisal Ahmed; Richard J. Ross; Nils P. Krone

doi:10.1530/EJE-20-1249

International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: Data from the I-CAH registry

Irina Bacila
, Nicole Freeman
, Eleni Daniel
, Marija Sandrk
, Jillian Bryce
, Salma Rashid Ali
, Zehra Yavas Abali
, Navoda Atapattu
, Tania A. Bachega
, Antonio Balsamo
, Niels Birkebæk
, Oliver Blankenstein
, Walter Bonfig
, Martine Cools
, Eduardo Correa Costa
, Feyza Darendeliler
, Silvia Einaudi
, Heba Hassan Elsedfy
, Martijn J.J. Finken
, Evelien Gevers

Hedi L. Claahsen-Van der Grinten, Tulay Guran, Ayla Güven, Sabine E. Hannema, Claire E. Higham, Violeta Iotova, Hetty J. van der Kamp, Marta Korbonits, Ruth E. Krone, Corina Lichiardopol, Andrea Luczay, Berenice Bilharinho Mendonca, Tatjana Milenkovic, Mirela C. Miranda, Klaus Mohnike, Uta Neumann, Rita Ortolano, Sukran Poyrazoglu, Ajay Thankamony, Jeremy W. Tomlinson, Ana Vieites, Liat de Vries, S. Faisal Ahmed, Richard J. Ross, Nils P. Krone^*

^*Corresponding author for this work

Pediatrics

University of Sheffield
University of Glasgow
Marmara University
Lady Ridgeway Hospital
Universidade de São Paulo
S. Orsola-Malpighi Hospital
Aarhus University Hospital
Charité – Universitätsmedizin Berlin
Technical University of Munich
Klinikum Wels - Grieskirchen GmbH
Universidade Federal do Rio Grande do Sul
Istanbul University
University of Turin
Ain Shams University
Vrije Universiteit Amsterdam
Barts and The London School of Medicine and Dentistry
Barts Health NHS Trust
Radboud University Medical Center
University of Health Sciences
University of Manchester and Christie NHS Foundation Trust
Medical University of Varna
Wilhelmina Kinderziekenhuis
Birmingham Women's and Children's Hospital
Craiova University of Medicine and Pharmacy
Semmelweis University
Institute for Mother and Child Healthcare of Serbia “Dr Vukan Čupić”
Otto von Guericke University Magdeburg
University of Cambridge
Oxford University Hospitals NHS Foundation Trust
Hospital de Niños Dr. Ricardo Gutiérrez
Schneider Childrens Medical Center Israel
Tel Aviv University
Technische Universität Dresden
Ghent University Hospital
Leiden University Medical Centre

Research output: Contribution to journal › Article › Academic › peer-review

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55 Downloads (Pure)

Abstract

Objective: Despite published guidelines no unified approach to hormone replacement in congenital adrenal hyperplasia (CAH) exists. We aimed to explore geographical and temporal var iations in the treatment with glucocorticoids and mineralocorticoids in CAH. Design: This retrospective multi-center study, including 31 centers (1 6 countries), analyzed data from the International-CAH Registry. Methods: Data were collected from 461 patients aged 0-18 years with cla ssic 21-hydroxylase deficiency (54.9% females) under follow-up between 1982 and 2018. Type, dose and timing of glucocorticoid and mineralocorticoid replacement were analyzed from 4174 patient visits. Results: The most frequently used glucocorticoid was hydrocortisone (87.6%). Overall, there were significant differences between age groups with regards to daily hydrocortisone-equival ent dose for body surface, with the lowest dose (median with interquartile range) of 12.0 (10.0-14.5) mg/m 2/day at age 1-8 years and the highest dose of 14.0 (11.6- 17.4) mg/m2/day at age 12-18 years. Glucocorticoid doses decreased after 2 010 in patients 0-8 years (P < 0.001) and remained unchanged in patients aged 8-18 years. Fludrocortisone was used in 92% of patients, with relative doses decreasing with age. A wide variation was observed among countr ies with regards to all aspects of steroid hormone replacement. Conclusions: Data from the I-CAH Registry suggests international variations in hormone replacement therapy, with a tendency to treatment with high doses in children.

Original language	English
Pages (from-to)	553-563
Number of pages	11
Journal	European Journal of Endocrinology
Volume	184
Issue number	4
DOIs	https://doi.org/10.1530/EJE-20-1249
Publication status	Published - Apr 2021

Bibliographical note

Funding Information:
This project has received support from the 阀-CAH Registry project that receives unrestricted education grants from Diurnal Ltd and Neurocrine Biosciences. The initial development of the Registry was supported by the Medical Research Council (G1100236), the Seventh European Union Framework Program (201444) and the European Society for Paediatric Endocrinology Research Unit. S R A is supported by the Gardiner Lectureship at the University of Glasgow. N P K is supported by the German Research Foundation (KR3363/3-1).

Publisher Copyright:
© 2021 The authors.

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International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia data from the I-CAH registryFinal published version, 0.98 MBLicence: CC BY

Cite this

Bacila, I., Freeman, N., Daniel, E., Sandrk, M., Bryce, J., Ali, S. R., Abali, Z. Y., Atapattu, N., Bachega, T. A., Balsamo, A., Birkebæk, N., Blankenstein, O., Bonfig, W., Cools, M., Costa, E. C., Darendeliler, F., Einaudi, S., Elsedfy, H. H., Finken, M. J. J., ... Krone, N. P. (2021). International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: Data from the I-CAH registry. European Journal of Endocrinology, 184(4), 553-563. https://doi.org/10.1530/EJE-20-1249

@article{bcb930c2fb414d86bb75da39aa6191be,

title = "International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: Data from the I-CAH registry",

abstract = "Objective: Despite published guidelines no unified approach to hormone replacement in congenital adrenal hyperplasia (CAH) exists. We aimed to explore geographical and temporal var iations in the treatment with glucocorticoids and mineralocorticoids in CAH. Design: This retrospective multi-center study, including 31 centers (1 6 countries), analyzed data from the International-CAH Registry. Methods: Data were collected from 461 patients aged 0-18 years with cla ssic 21-hydroxylase deficiency (54.9\% females) under follow-up between 1982 and 2018. Type, dose and timing of glucocorticoid and mineralocorticoid replacement were analyzed from 4174 patient visits. Results: The most frequently used glucocorticoid was hydrocortisone (87.6\%). Overall, there were significant differences between age groups with regards to daily hydrocortisone-equival ent dose for body surface, with the lowest dose (median with interquartile range) of 12.0 (10.0-14.5) mg/m 2/day at age 1-8 years and the highest dose of 14.0 (11.6- 17.4) mg/m2/day at age 12-18 years. Glucocorticoid doses decreased after 2 010 in patients 0-8 years (P < 0.001) and remained unchanged in patients aged 8-18 years. Fludrocortisone was used in 92\% of patients, with relative doses decreasing with age. A wide variation was observed among countr ies with regards to all aspects of steroid hormone replacement. Conclusions: Data from the I-CAH Registry suggests international variations in hormone replacement therapy, with a tendency to treatment with high doses in children. ",

author = "Irina Bacila and Nicole Freeman and Eleni Daniel and Marija Sandrk and Jillian Bryce and Ali, \{Salma Rashid\} and Abali, \{Zehra Yavas\} and Navoda Atapattu and Bachega, \{Tania A.\} and Antonio Balsamo and Niels Birkeb{\ae}k and Oliver Blankenstein and Walter Bonfig and Martine Cools and Costa, \{Eduardo Correa\} and Feyza Darendeliler and Silvia Einaudi and Elsedfy, \{Heba Hassan\} and Finken, \{Martijn J.J.\} and Evelien Gevers and \{Claahsen-Van der Grinten\}, \{Hedi L.\} and Tulay Guran and Ayla G{\"u}ven and Hannema, \{Sabine E.\} and Higham, \{Claire E.\} and Violeta Iotova and \{van der Kamp\}, \{Hetty J.\} and Marta Korbonits and Krone, \{Ruth E.\} and Corina Lichiardopol and Andrea Luczay and Mendonca, \{Berenice Bilharinho\} and Tatjana Milenkovic and Miranda, \{Mirela C.\} and Klaus Mohnike and Uta Neumann and Rita Ortolano and Sukran Poyrazoglu and Ajay Thankamony and Tomlinson, \{Jeremy W.\} and Ana Vieites and \{de Vries\}, Liat and Ahmed, \{S. Faisal\} and Ross, \{Richard J.\} and Krone, \{Nils P.\}",

note = "Funding Information: This project has received support from the 阀-CAH Registry project that receives unrestricted education grants from Diurnal Ltd and Neurocrine Biosciences. The initial development of the Registry was supported by the Medical Research Council (G1100236), the Seventh European Union Framework Program (201444) and the European Society for Paediatric Endocrinology Research Unit. S R A is supported by the Gardiner Lectureship at the University of Glasgow. N P K is supported by the German Research Foundation (KR3363/3-1). Publisher Copyright: {\textcopyright} 2021 The authors.",

year = "2021",

month = apr,

doi = "10.1530/EJE-20-1249",

language = "English",

volume = "184",

pages = "553--563",

number = "4",

}

Bacila, I, Freeman, N, Daniel, E, Sandrk, M, Bryce, J, Ali, SR, Abali, ZY, Atapattu, N, Bachega, TA, Balsamo, A, Birkebæk, N, Blankenstein, O, Bonfig, W, Cools, M, Costa, EC, Darendeliler, F, Einaudi, S, Elsedfy, HH, Finken, MJJ, Gevers, E, Claahsen-Van der Grinten, HL, Guran, T, Güven, A, Hannema, SE, Higham, CE, Iotova, V, van der Kamp, HJ, Korbonits, M, Krone, RE, Lichiardopol, C, Luczay, A, Mendonca, BB, Milenkovic, T, Miranda, MC, Mohnike, K, Neumann, U, Ortolano, R, Poyrazoglu, S, Thankamony, A, Tomlinson, JW, Vieites, A, de Vries, L, Ahmed, SF, Ross, RJ & Krone, NP 2021, 'International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: Data from the I-CAH registry', European Journal of Endocrinology, vol. 184, no. 4, pp. 553-563. https://doi.org/10.1530/EJE-20-1249

TY - JOUR

T1 - International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia

T2 - Data from the I-CAH registry

AU - Bacila, Irina

AU - Freeman, Nicole

AU - Daniel, Eleni

AU - Sandrk, Marija

AU - Bryce, Jillian

AU - Ali, Salma Rashid

AU - Abali, Zehra Yavas

AU - Atapattu, Navoda

AU - Bachega, Tania A.

AU - Balsamo, Antonio

AU - Birkebæk, Niels

AU - Blankenstein, Oliver

AU - Bonfig, Walter

AU - Cools, Martine

AU - Costa, Eduardo Correa

AU - Darendeliler, Feyza

AU - Einaudi, Silvia

AU - Elsedfy, Heba Hassan

AU - Finken, Martijn J.J.

AU - Gevers, Evelien

AU - Claahsen-Van der Grinten, Hedi L.

AU - Guran, Tulay

AU - Güven, Ayla

AU - Hannema, Sabine E.

AU - Higham, Claire E.

AU - Iotova, Violeta

AU - van der Kamp, Hetty J.

AU - Korbonits, Marta

AU - Krone, Ruth E.

AU - Lichiardopol, Corina

AU - Luczay, Andrea

AU - Mendonca, Berenice Bilharinho

AU - Milenkovic, Tatjana

AU - Miranda, Mirela C.

AU - Mohnike, Klaus

AU - Neumann, Uta

AU - Ortolano, Rita

AU - Poyrazoglu, Sukran

AU - Thankamony, Ajay

AU - Tomlinson, Jeremy W.

AU - Vieites, Ana

AU - de Vries, Liat

AU - Ahmed, S. Faisal

AU - Ross, Richard J.

AU - Krone, Nils P.

N1 - Funding Information: This project has received support from the 阀-CAH Registry project that receives unrestricted education grants from Diurnal Ltd and Neurocrine Biosciences. The initial development of the Registry was supported by the Medical Research Council (G1100236), the Seventh European Union Framework Program (201444) and the European Society for Paediatric Endocrinology Research Unit. S R A is supported by the Gardiner Lectureship at the University of Glasgow. N P K is supported by the German Research Foundation (KR3363/3-1). Publisher Copyright: © 2021 The authors.

PY - 2021/4

Y1 - 2021/4

N2 - Objective: Despite published guidelines no unified approach to hormone replacement in congenital adrenal hyperplasia (CAH) exists. We aimed to explore geographical and temporal var iations in the treatment with glucocorticoids and mineralocorticoids in CAH. Design: This retrospective multi-center study, including 31 centers (1 6 countries), analyzed data from the International-CAH Registry. Methods: Data were collected from 461 patients aged 0-18 years with cla ssic 21-hydroxylase deficiency (54.9% females) under follow-up between 1982 and 2018. Type, dose and timing of glucocorticoid and mineralocorticoid replacement were analyzed from 4174 patient visits. Results: The most frequently used glucocorticoid was hydrocortisone (87.6%). Overall, there were significant differences between age groups with regards to daily hydrocortisone-equival ent dose for body surface, with the lowest dose (median with interquartile range) of 12.0 (10.0-14.5) mg/m 2/day at age 1-8 years and the highest dose of 14.0 (11.6- 17.4) mg/m2/day at age 12-18 years. Glucocorticoid doses decreased after 2 010 in patients 0-8 years (P < 0.001) and remained unchanged in patients aged 8-18 years. Fludrocortisone was used in 92% of patients, with relative doses decreasing with age. A wide variation was observed among countr ies with regards to all aspects of steroid hormone replacement. Conclusions: Data from the I-CAH Registry suggests international variations in hormone replacement therapy, with a tendency to treatment with high doses in children.

AB - Objective: Despite published guidelines no unified approach to hormone replacement in congenital adrenal hyperplasia (CAH) exists. We aimed to explore geographical and temporal var iations in the treatment with glucocorticoids and mineralocorticoids in CAH. Design: This retrospective multi-center study, including 31 centers (1 6 countries), analyzed data from the International-CAH Registry. Methods: Data were collected from 461 patients aged 0-18 years with cla ssic 21-hydroxylase deficiency (54.9% females) under follow-up between 1982 and 2018. Type, dose and timing of glucocorticoid and mineralocorticoid replacement were analyzed from 4174 patient visits. Results: The most frequently used glucocorticoid was hydrocortisone (87.6%). Overall, there were significant differences between age groups with regards to daily hydrocortisone-equival ent dose for body surface, with the lowest dose (median with interquartile range) of 12.0 (10.0-14.5) mg/m 2/day at age 1-8 years and the highest dose of 14.0 (11.6- 17.4) mg/m2/day at age 12-18 years. Glucocorticoid doses decreased after 2 010 in patients 0-8 years (P < 0.001) and remained unchanged in patients aged 8-18 years. Fludrocortisone was used in 92% of patients, with relative doses decreasing with age. A wide variation was observed among countr ies with regards to all aspects of steroid hormone replacement. Conclusions: Data from the I-CAH Registry suggests international variations in hormone replacement therapy, with a tendency to treatment with high doses in children.

UR - https://www.scopus.com/pages/publications/85102965211

U2 - 10.1530/EJE-20-1249

DO - 10.1530/EJE-20-1249

M3 - Article

C2 - 33460392

AN - SCOPUS:85102965211

SN - 0804-4643

VL - 184

SP - 553

EP - 563

JO - European Journal of Endocrinology

JF - European Journal of Endocrinology

IS - 4

ER -

International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: Data from the I-CAH registry

Abstract

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