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Corticosterone 18-monooxygenase deficiency(FHHA1A)

MedGen UID:
82784
Concept ID:
C0268293
Disease or Syndrome
Synonyms: 18 alpha hydroxylase deficiency; 18 Hydroxylase deficiency; Aldosterone deficiency 1; Aldosterone deficiency due to defect in 18 hydroxylase; ALDOSTERONE DEFICIENCY DUE TO DEFECT IN STEROID 18-HYDROXYLASE; ALDOSTERONE DEFICIENCY I; CMO 1 deficiency; CMO I DEFICIENCY; Corticosterone methyloxidase type 1 deficiency; STEROID 18-HYDROXYLASE DEFICIENCY
SNOMED CT: 18-Hydroxycorticosterone dehydrogenase deficiency (47757001); Aldosterone deficiency due to 18-hydroxysteroid dehydrogenase deficiency (47757001); CAH - 18-hydroxylase deficiency (47757001); Corticosterone 18-monooxygenase deficiency (47757001); 18-Hydroxylase deficiency (47757001); Aldosterone deficiency due to 18-hydroxylase defect (47757001); Corticosterone methyl oxidase type I deficiency (47757001); CMO I deficiency (47757001)
 
Gene (location): CYP11B2 (8q24.3)
 
Monarch Initiative: MONDO:0008751
OMIM®: 203400

Definition

CMO type I deficiency is an autosomal recessive disorder caused by a defect in the penultimate biochemical step of aldosterone biosynthesis, the 18-hydroxylation of corticosterone (B) to 18-hydroxycorticosterone (18-OHB). This enzymatic defect results in decreased aldosterone and salt-wasting. In CMO I deficiency, aldosterone is undetectable, whereas its immediate precursor, 18-OHB, is low or normal. These patients have an increased ratio of corticosterone to 18-OHB (Portrat-Doyen et al., 1998). The CYP11B2 gene product also catalyzes the final step in aldosterone biosynthesis: the 18-oxidation of 18-OHB to aldosterone. A defect in that enzymatic step results in CMO type II deficiency (610600), an allelic disorder with an overlapping phenotype but distinct biochemical features. In CMO II deficiency, aldosterone can be low or normal, but at the expense of increased secretion of 18-OHB. These patients have a low ratio of corticosterone to 18-OHB (Portrat-Doyen et al., 1998). [from OMIM]

Additional description

From MedlinePlus Genetics
Corticosterone methyloxidase deficiency, also known as aldosterone synthase deficiency, is a disorder characterized by excessive amounts of sodium released in the urine (salt wasting), along with insufficient release of potassium in the urine, usually beginning in the first few weeks of life. This imbalance leads to low levels of sodium and high levels of potassium in the blood (hyponatremia and hyperkalemia, respectively). Individuals with corticosterone methyloxidase deficiency can also have high levels of acid in the blood (metabolic acidosis).

The hyponatremia, hyperkalemia, and metabolic acidosis associated with corticosterone methyloxidase deficiency can cause nausea, vomiting, dehydration, low blood pressure, extreme tiredness (fatigue), and muscle weakness. Affected infants often experience failure to thrive, which means they do not gain weight and grow at the expected rate. Severe cases of corticosterone methyloxidase deficiency can result in seizures and coma and can be life-threatening. However, affected individuals who survive infancy generally have a normal life expectancy, and the signs and symptoms of the disorder typically become milder or disappear by adulthood.  https://medlineplus.gov/genetics/condition/corticosterone-methyloxidase-deficiency

Clinical features

From HPO
Renal salt wasting
MedGen UID:
375868
Concept ID:
C1846347
Finding
A high concentration of one or more electrolytes in the urine in the presence of low serum concentrations of the electrolyte(s).
Hypotension
MedGen UID:
5715
Concept ID:
C0020649
Finding
Low Blood Pressure, vascular hypotension.
Growth delay
MedGen UID:
99124
Concept ID:
C0456070
Pathologic Function
A deficiency or slowing down of growth pre- and postnatally.
Failure to thrive
MedGen UID:
746019
Concept ID:
C2315100
Disease or Syndrome
Failure to thrive (FTT) refers to a child whose physical growth is substantially below the norm.
Vomiting
MedGen UID:
12124
Concept ID:
C0042963
Sign or Symptom
Forceful ejection of the contents of the stomach through the mouth by means of a series of involuntary spasmic contractions.
Feeding difficulties in infancy
MedGen UID:
436211
Concept ID:
C2674608
Finding
Impaired feeding performance of an infant as manifested by difficulties such as weak and ineffective sucking, brief bursts of sucking, and falling asleep during sucking. There may be difficulties with chewing or maintaining attention.
Dehydration
MedGen UID:
8273
Concept ID:
C0011175
Disease or Syndrome
A condition resulting from the excessive loss of water from the body. It is usually caused by severe diarrhea, vomiting or diaphoresis.
Hyperkalemia
MedGen UID:
5691
Concept ID:
C0020461
Finding
An abnormally increased potassium concentration in the blood.
Hyponatremia
MedGen UID:
6984
Concept ID:
C0020625
Finding
An abnormally decreased sodium concentration in the blood.
Recurrent fever
MedGen UID:
811468
Concept ID:
C3714772
Sign or Symptom
Periodic (episodic or recurrent) bouts of fever.
Increased circulating renin concentration
MedGen UID:
66818
Concept ID:
C0240783
Finding
An increased level of renin in the blood.
Decreased circulating aldosterone concentration
MedGen UID:
208996
Concept ID:
C0857899
Finding
Abnormally reduced levels of aldosterone.

Professional guidelines

PubMed

Gurpinar Tosun B, Kendir Demirkol Y, Seven Menevse T, Kaygusuz SB, Ozbek MN, Altincik SA, Mammadova J, Cayir A, Doger E, Bayramoglu E, Nalbantoglu O, Yesiltepe Mutlu G, Aghayev A, Turan S, Bereket A, Guran T
J Clin Endocrinol Metab 2022 Jan 1;107(1):e106-e117. doi: 10.1210/clinem/dgab619. PMID: 34415991

Recent clinical studies

Etiology

Concolino P
Mol Diagn Ther 2024 Mar;28(2):215-224. Epub 2024 Feb 7 doi: 10.1007/s40291-024-00697-y. PMID: 38324138
Polat S, Karaburgu S, Unluhizarci K, Dundar M, Ozkul Y, Arslan YK, Karaca Z, Kelestimur F
J Endocrinol Invest 2023 Nov;46(11):2367-2377. Epub 2023 Apr 13 doi: 10.1007/s40618-023-02093-5. PMID: 37055708
Gurpinar Tosun B, Kendir Demirkol Y, Seven Menevse T, Kaygusuz SB, Ozbek MN, Altincik SA, Mammadova J, Cayir A, Doger E, Bayramoglu E, Nalbantoglu O, Yesiltepe Mutlu G, Aghayev A, Turan S, Bereket A, Guran T
J Clin Endocrinol Metab 2022 Jan 1;107(1):e106-e117. doi: 10.1210/clinem/dgab619. PMID: 34415991
Wijaya M, Ma H, Zhang J, Du M, Li Y, Chen Q, Guo S
BMC Endocr Disord 2021 Jul 9;21(1):149. doi: 10.1186/s12902-021-00811-9. PMID: 34243750Free PMC Article
Martín-Rivada Á, Argente J, Martos-Moreno GÁ
J Pediatr Endocrinol Metab 2020 Nov 26;33(11):1501-1505. doi: 10.1515/jpem-2020-0239. PMID: 32809961

Diagnosis

Concolino P
Mol Diagn Ther 2024 Mar;28(2):215-224. Epub 2024 Feb 7 doi: 10.1007/s40291-024-00697-y. PMID: 38324138
Gurpinar Tosun B, Kendir Demirkol Y, Seven Menevse T, Kaygusuz SB, Ozbek MN, Altincik SA, Mammadova J, Cayir A, Doger E, Bayramoglu E, Nalbantoglu O, Yesiltepe Mutlu G, Aghayev A, Turan S, Bereket A, Guran T
J Clin Endocrinol Metab 2022 Jan 1;107(1):e106-e117. doi: 10.1210/clinem/dgab619. PMID: 34415991
Martín-Rivada Á, Argente J, Martos-Moreno GÁ
J Pediatr Endocrinol Metab 2020 Nov 26;33(11):1501-1505. doi: 10.1515/jpem-2020-0239. PMID: 32809961
Cerame BI, New MI
J Pediatr Endocrinol Metab 2000 Nov-Dec;13(9):1537-47. doi: 10.1515/jpem.2000.13.9.1537. PMID: 11154148
Peter M, Dubuis JM, Sippell WG
Horm Res 1999;51(5):211-22. doi: 10.1159/000023374. PMID: 10559665

Therapy

Polat S, Karaburgu S, Unluhizarci K, Dundar M, Ozkul Y, Arslan YK, Karaca Z, Kelestimur F
J Endocrinol Invest 2023 Nov;46(11):2367-2377. Epub 2023 Apr 13 doi: 10.1007/s40618-023-02093-5. PMID: 37055708
Brown NJ
Nat Rev Nephrol 2013 Aug;9(8):459-69. Epub 2013 Jun 18 doi: 10.1038/nrneph.2013.110. PMID: 23774812Free PMC Article
Hakki T, Bernhardt R
Pharmacol Ther 2006 Jul;111(1):27-52. Epub 2006 Jan 19 doi: 10.1016/j.pharmthera.2005.07.006. PMID: 16426683
Warnock DG
Curr Opin Nephrol Hypertens 2001 Jul;10(4):493-9. doi: 10.1097/00041552-200107000-00003. PMID: 11458030
White PC
Endocrinol Metab Clin North Am 2001 Mar;30(1):61-79, vi. doi: 10.1016/s0889-8529(08)70019-7. PMID: 11344939

Prognosis

Gurpinar Tosun B, Kendir Demirkol Y, Seven Menevse T, Kaygusuz SB, Ozbek MN, Altincik SA, Mammadova J, Cayir A, Doger E, Bayramoglu E, Nalbantoglu O, Yesiltepe Mutlu G, Aghayev A, Turan S, Bereket A, Guran T
J Clin Endocrinol Metab 2022 Jan 1;107(1):e106-e117. doi: 10.1210/clinem/dgab619. PMID: 34415991
Wijaya M, Ma H, Zhang J, Du M, Li Y, Chen Q, Guo S
BMC Endocr Disord 2021 Jul 9;21(1):149. doi: 10.1186/s12902-021-00811-9. PMID: 34243750Free PMC Article
Martín-Rivada Á, Argente J, Martos-Moreno GÁ
J Pediatr Endocrinol Metab 2020 Nov 26;33(11):1501-1505. doi: 10.1515/jpem-2020-0239. PMID: 32809961
Jia M, Yang B, Li Z, Shen H, Song X, Gu W
PLoS One 2014;9(8):e104311. Epub 2014 Aug 7 doi: 10.1371/journal.pone.0104311. PMID: 25102047Free PMC Article
Müller J
Steroids 1995 Jan;60(1):2-9. doi: 10.1016/0039-128x(94)00021-4. PMID: 7792810

Clinical prediction guides

Pignatti E, Kollar J, Hafele E, Schuster D, Steele RE, Vogt B, Schumacher C, Groessl M
J Steroid Biochem Mol Biol 2023 Dec;235:106409. Epub 2023 Oct 10 doi: 10.1016/j.jsbmb.2023.106409. PMID: 37827219
Jia M, Yang B, Li Z, Shen H, Song X, Gu W
PLoS One 2014;9(8):e104311. Epub 2014 Aug 7 doi: 10.1371/journal.pone.0104311. PMID: 25102047Free PMC Article
Luo Y, Kumar P, Chen CC, Latham J, Wang L, Tudela C, Alexander JM, Shelton JM, McKown L, Mendelson CR
Mol Endocrinol 2014 Jun;28(6):965-75. Epub 2014 Apr 11 doi: 10.1210/me.2014-1003. PMID: 24725083Free PMC Article
Nishimoto K, Nakagawa K, Li D, Kosaka T, Oya M, Mikami S, Shibata H, Itoh H, Mitani F, Yamazaki T, Ogishima T, Suematsu M, Mukai K
J Clin Endocrinol Metab 2010 May;95(5):2296-305. Epub 2010 Mar 3 doi: 10.1210/jc.2009-2010. PMID: 20200334
Lee PD, Patterson BD, Hintz RL, Rosenfeld RG
J Clin Endocrinol Metab 1986 Jan;62(1):225-9. doi: 10.1210/jcem-62-1-225. PMID: 3510001

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