U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Familial glucocorticoid deficiency

MedGen UID:
885955
Concept ID:
C4054695
Disease or Syndrome
Synonyms: Familial Glucocorticoid Deficiency; familial glucocorticoid deficiency; GCCD; Isolated Glucocorticoid Deficiency
SNOMED CT: Familial glucocorticoid deficiency (765326001)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Monarch Initiative: MONDO:0008733
OMIM® Phenotypic series: PS202200
Orphanet: ORPHA361

Definition

Disease that is characterised clinically by neonatal hyperpigmentation, hypoglycaemia, failure to thrive, and recurrent infections and biochemically by glucocorticoid deficiency without mineralocorticoid deficiency. The disease usually presents in infancy or early childhood. Hypoglycaemic crises resulting in convulsions can lead to coma or death if untreated and recurrent hypoglycaemia may lead to neurological sequelae. Most cases are caused by defects in the adrenocorticotropin receptor, or its signalling pathway resulting in a failure of the cells of zona fasciculata in the adrenal cortex to respond appropriately to adrenocorticotrophic hormone, leading to a glucocorticoid deficiency. These defects are most commonly caused by mutations in MC2R (18p11.2) and MRAP (21q22.1). Other mutations reported include MCM4 (8q12-q13), NNT (5p12) and TXNRD2 (22q11.21). Inherited in an autosomal recessive manner. [from SNOMEDCT_US]

Professional guidelines

PubMed

Metwalley K, Farghaly H
Acta Biomed 2021 Sep 2;92(4):e2021255. doi: 10.23750/abm.v92i4.11613. PMID: 34487068Free PMC Article
Çamtosun E, Dündar İ, Akıncı A, Kayaş L, Çiftci N
J Clin Res Pediatr Endocrinol 2021 Feb 26;13(1):88-99. Epub 2020 Sep 17 doi: 10.4274/jcrpe.galenos.2020.2020.0132. PMID: 32938577Free PMC Article
Malikova J, Flück CE
Horm Res Paediatr 2014;82(3):145-57. Epub 2014 Aug 1 doi: 10.1159/000363107. PMID: 25096886

Recent clinical studies

Etiology

Hasenmajer V, Ferrigno R, Minnetti M, Pellegrini B, Isidori AM, Lenzi A, Salerno M, Cappa M, Chan L, De Martino MC, Savage MO
Rev Endocr Metab Disord 2023 Apr;24(2):345-363. Epub 2023 Feb 10 doi: 10.1007/s11154-023-09784-7. PMID: 36763264Free PMC Article
Capalbo D, Moracas C, Cappa M, Balsamo A, Maghnie M, Wasniewska MG, Greggio NA, Baronio F, Bizzarri C, Ferro G, Di Lascio A, Stancampiano MR, Azzolini S, Patti G, Longhi S, Valenzise M, Radetti G, Betterle C, Russo G, Salerno M
J Clin Endocrinol Metab 2021 Mar 8;106(3):762-773. doi: 10.1210/clinem/dgaa881. PMID: 33247909
Çamtosun E, Dündar İ, Akıncı A, Kayaş L, Çiftci N
J Clin Res Pediatr Endocrinol 2021 Feb 26;13(1):88-99. Epub 2020 Sep 17 doi: 10.4274/jcrpe.galenos.2020.2020.0132. PMID: 32938577Free PMC Article
Cooray SN, Chan L, Metherell L, Storr H, Clark AJL
Endocr Dev 2008;13:99-116. doi: 10.1159/000134828. PMID: 18493136
Huebner A, Elias LL, Clark AJ
J Pediatr Endocrinol Metab 1999 Apr;12 Suppl 1:277-93. PMID: 10698592

Diagnosis

Maharaj AV
Front Endocrinol (Lausanne) 2023;14:1268345. Epub 2023 Dec 21 doi: 10.3389/fendo.2023.1268345. PMID: 38189052Free PMC Article
Hasenmajer V, Ferrigno R, Minnetti M, Pellegrini B, Isidori AM, Lenzi A, Salerno M, Cappa M, Chan L, De Martino MC, Savage MO
Rev Endocr Metab Disord 2023 Apr;24(2):345-363. Epub 2023 Feb 10 doi: 10.1007/s11154-023-09784-7. PMID: 36763264Free PMC Article
Maharaj A, Maudhoo A, Chan LF, Novoselova T, Prasad R, Metherell LA, Guasti L
J Steroid Biochem Mol Biol 2019 May;189:73-80. Epub 2019 Feb 25 doi: 10.1016/j.jsbmb.2019.02.012. PMID: 30817990
Bertorini TE, Perez A
Handb Clin Neurol 2014;120:749-71. doi: 10.1016/B978-0-7020-4087-0.00050-4. PMID: 24365350
Meimaridou E, Hughes CR, Kowalczyk J, Chan LF, Clark AJ, Metherell LA
Endocr Dev 2013;24:57-66. Epub 2013 Feb 1 doi: 10.1159/000342504. PMID: 23392095

Therapy

Ferrigno R, Cioffi D, Pellino V, Savanelli MC, Klain A
Endocrine 2023 Apr;80(1):1-9. Epub 2022 Oct 30 doi: 10.1007/s12020-022-03236-z. PMID: 36309634
Tunkara H, Topor LS
J Pediatr 2022 May;244:e5. doi: 10.1016/j.jpeds.2022.01.022. PMID: 35534171
Luo Y, Bai R, Wang Z, Zhu X, Xing J, Li X
Mol Med Rep 2020 Aug;22(2):681-686. Epub 2020 May 7 doi: 10.3892/mmr.2020.11140. PMID: 32627004Free PMC Article
Elias LL, Huebner A, Metherell LA, Canas A, Warne GL, Bitti ML, Cianfarani S, Clayton PE, Savage MO, Clark AJ
Clin Endocrinol (Oxf) 2000 Oct;53(4):423-30. doi: 10.1046/j.1365-2265.2000.01122.x. PMID: 11012566
Thistlethwaite D, Darling JA, Fraser R, Rees LH, Harkness RA
J Endocrinol 1974 Nov;63(2):48P-49P. PMID: 4374490

Prognosis

Maharaj AV
Front Endocrinol (Lausanne) 2023;14:1268345. Epub 2023 Dec 21 doi: 10.3389/fendo.2023.1268345. PMID: 38189052Free PMC Article
Jazayeri O, Liu X, van Diemen CC, Bakker-van Waarde WM, Sikkema-Raddatz B, Sinke RJ, Zhang J, van Ravenswaaij-Arts CM
Eur J Med Genet 2015 Dec;58(12):642-9. Epub 2015 Nov 6 doi: 10.1016/j.ejmg.2015.11.001. PMID: 26548497
Habeb AM, Hughes CR, Al-Arabi R, Al-Muhamadi A, Clark AJ, Metherell LA
Eur J Pediatr 2013 Oct;172(10):1407-10. Epub 2013 May 26 doi: 10.1007/s00431-013-2044-1. PMID: 23708259
Aza-Carmona M, Barreda-Bonis AC, Guerrero-Fernández J, González-Casado I, Gracia R, Heath KE
J Pediatr Endocrinol Metab 2011;24(5-6):395-7. doi: 10.1515/jpem.2011.024. PMID: 21823545
Elias LL, Huebner A, Metherell LA, Canas A, Warne GL, Bitti ML, Cianfarani S, Clayton PE, Savage MO, Clark AJ
Clin Endocrinol (Oxf) 2000 Oct;53(4):423-30. doi: 10.1046/j.1365-2265.2000.01122.x. PMID: 11012566

Clinical prediction guides

Maharaj AV
Front Endocrinol (Lausanne) 2023;14:1268345. Epub 2023 Dec 21 doi: 10.3389/fendo.2023.1268345. PMID: 38189052Free PMC Article
Ferrigno R, Cioffi D, Pellino V, Savanelli MC, Klain A
Endocrine 2023 Apr;80(1):1-9. Epub 2022 Oct 30 doi: 10.1007/s12020-022-03236-z. PMID: 36309634
Capalbo D, Moracas C, Cappa M, Balsamo A, Maghnie M, Wasniewska MG, Greggio NA, Baronio F, Bizzarri C, Ferro G, Di Lascio A, Stancampiano MR, Azzolini S, Patti G, Longhi S, Valenzise M, Radetti G, Betterle C, Russo G, Salerno M
J Clin Endocrinol Metab 2021 Mar 8;106(3):762-773. doi: 10.1210/clinem/dgaa881. PMID: 33247909
Elias LL, Huebner A, Metherell LA, Canas A, Warne GL, Bitti ML, Cianfarani S, Clayton PE, Savage MO, Clark AJ
Clin Endocrinol (Oxf) 2000 Oct;53(4):423-30. doi: 10.1046/j.1365-2265.2000.01122.x. PMID: 11012566
Allolio B, Reincke M
Horm Res 1997;47(4-6):273-8. doi: 10.1159/000185476. PMID: 9167964

Supplemental Content

Table of contents

    Clinical resources

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Recent activity

    Your browsing activity is empty.

    Activity recording is turned off.

    Turn recording back on

    See more...