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A Prospective Study of Children Aged 0–8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone Granules
CONTEXT: Children with congenital adrenal hyperplasia (CAH) and adrenal insufficiency (AI) require daily hydrocortisone replacement with accurate dosing. OBJECTIVE: Prospective study of efficacy and safety of hydrocortisone granules in children with AI and CAH monitored by 17-OHP (17-hydroxyprogeste...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7947757/ https://www.ncbi.nlm.nih.gov/pubmed/32888021 http://dx.doi.org/10.1210/clinem/dgaa626 |
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author | Neumann, Uta Braune, Katarina Whitaker, Martin J Wiegand, Susanna Krude, Heiko Porter, John Digweed, Dena Voet, Bernard Ross, Richard J M Blankenstein, Oliver |
author_facet | Neumann, Uta Braune, Katarina Whitaker, Martin J Wiegand, Susanna Krude, Heiko Porter, John Digweed, Dena Voet, Bernard Ross, Richard J M Blankenstein, Oliver |
author_sort | Neumann, Uta |
collection | PubMed |
description | CONTEXT: Children with congenital adrenal hyperplasia (CAH) and adrenal insufficiency (AI) require daily hydrocortisone replacement with accurate dosing. OBJECTIVE: Prospective study of efficacy and safety of hydrocortisone granules in children with AI and CAH monitored by 17-OHP (17-hydroxyprogesterone) saliva profiles. METHODS: Seventeen children with CAH (9 male) and 1 with hypopituitarism (male), aged from birth to 6 years, had their hydrocortisone medication changed from pharmacy compounded capsules to hydrocortisone granules. Patients were followed prospectively for 2 years. In children with CAH, the therapy was adjusted by 17-OHP salivary profiles every 3 months. The following parameters were recorded: hydrocortisone dose, height, weight, pubertal status, adverse events, and incidence of adrenal crisis. RESULTS: The study medication was given thrice daily, and the median duration of treatment (range) was 795 (1–872) days, with 150 follow-up visits. Hydrocortisone doses were changed on 40/150 visits, with 32 based on salivary measurements and 8 on serum 17-OHP levels. The median daily mg/m(2) hydrocortisone dose (range) at study entry for the different age groups 2–8 years, 1 month to 2 years, <28 days was 11.9 (7.2–15.5), 9.9 (8.6–12.2), and 12.0 (11.1–29.5), respectively, and at end of the study was 10.2 (7.0–14.4), 9.8 (8.9–13.1), and 8.6 (8.2–13.7), respectively. There were no trends for accelerated or reduced growth. No adrenal crises were observed despite 193 treatment-emergent adverse events, which were mainly common childhood illnesses. INTERPRETATION: This first prospective study of glucocorticoid treatment in children with AI and CAH demonstrates that accurate dosing and monitoring from birth results in hydrocortisone doses at the lower end of the recommended dose range and normal growth, without occurrence of adrenal crises. |
format | Online Article Text |
id | pubmed-7947757 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | Oxford University Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-79477572021-03-16 A Prospective Study of Children Aged 0–8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone Granules Neumann, Uta Braune, Katarina Whitaker, Martin J Wiegand, Susanna Krude, Heiko Porter, John Digweed, Dena Voet, Bernard Ross, Richard J M Blankenstein, Oliver J Clin Endocrinol Metab Clinical Research Articles CONTEXT: Children with congenital adrenal hyperplasia (CAH) and adrenal insufficiency (AI) require daily hydrocortisone replacement with accurate dosing. OBJECTIVE: Prospective study of efficacy and safety of hydrocortisone granules in children with AI and CAH monitored by 17-OHP (17-hydroxyprogesterone) saliva profiles. METHODS: Seventeen children with CAH (9 male) and 1 with hypopituitarism (male), aged from birth to 6 years, had their hydrocortisone medication changed from pharmacy compounded capsules to hydrocortisone granules. Patients were followed prospectively for 2 years. In children with CAH, the therapy was adjusted by 17-OHP salivary profiles every 3 months. The following parameters were recorded: hydrocortisone dose, height, weight, pubertal status, adverse events, and incidence of adrenal crisis. RESULTS: The study medication was given thrice daily, and the median duration of treatment (range) was 795 (1–872) days, with 150 follow-up visits. Hydrocortisone doses were changed on 40/150 visits, with 32 based on salivary measurements and 8 on serum 17-OHP levels. The median daily mg/m(2) hydrocortisone dose (range) at study entry for the different age groups 2–8 years, 1 month to 2 years, <28 days was 11.9 (7.2–15.5), 9.9 (8.6–12.2), and 12.0 (11.1–29.5), respectively, and at end of the study was 10.2 (7.0–14.4), 9.8 (8.9–13.1), and 8.6 (8.2–13.7), respectively. There were no trends for accelerated or reduced growth. No adrenal crises were observed despite 193 treatment-emergent adverse events, which were mainly common childhood illnesses. INTERPRETATION: This first prospective study of glucocorticoid treatment in children with AI and CAH demonstrates that accurate dosing and monitoring from birth results in hydrocortisone doses at the lower end of the recommended dose range and normal growth, without occurrence of adrenal crises. Oxford University Press 2020-09-04 /pmc/articles/PMC7947757/ /pubmed/32888021 http://dx.doi.org/10.1210/clinem/dgaa626 Text en © The Author(s) 2020. Published by Oxford University Press on behalf of the Endocrine Society. https://creativecommons.org/licenses/by/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) ), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Clinical Research Articles Neumann, Uta Braune, Katarina Whitaker, Martin J Wiegand, Susanna Krude, Heiko Porter, John Digweed, Dena Voet, Bernard Ross, Richard J M Blankenstein, Oliver A Prospective Study of Children Aged 0–8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone Granules |
title | A Prospective Study of Children Aged 0–8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone Granules |
title_full | A Prospective Study of Children Aged 0–8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone Granules |
title_fullStr | A Prospective Study of Children Aged 0–8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone Granules |
title_full_unstemmed | A Prospective Study of Children Aged 0–8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone Granules |
title_short | A Prospective Study of Children Aged 0–8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone Granules |
title_sort | prospective study of children aged 0–8 years with cah and adrenal insufficiency treated with hydrocortisone granules |
topic | Clinical Research Articles |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7947757/ https://www.ncbi.nlm.nih.gov/pubmed/32888021 http://dx.doi.org/10.1210/clinem/dgaa626 |
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