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Complete androgen insensitivity syndrome(CAIS)

MedGen UID:
183188
Concept ID:
C0936016
Disease or Syndrome
Synonyms: CAIS; Complete androgen insensitivity syndrome, complete
SNOMED CT: Complete androgen insensitivity syndrome (368851000119102); Complete androgen resistance syndrome (368851000119102); CAIS - complete androgen insensitivity syndrome (368851000119102)
Modes of inheritance:
X-linked recessive inheritance
MedGen UID:
375779
Concept ID:
C1845977
Finding
Source: Orphanet
A mode of inheritance that is observed for recessive traits related to a gene encoded on the X chromosome. In the context of medical genetics, X-linked recessive disorders manifest in males (who have one copy of the X chromosome and are thus hemizygotes), but generally not in female heterozygotes who have one mutant and one normal allele.
 
Monarch Initiative: MONDO:0021023
OMIM®: 313700
Orphanet: ORPHA99429

Definition

A form of androgen insensitivity syndrome (AIS) characterized by the presence of female external genitalia in a 46,XY individual with normal testis development but undescended testes and unresponsiveness to age-appropriate levels of androgens. The condition is due to mutations in the androgen receptor (AR) gene which is located on the long arm of the X-chromosome (Xq11-12). The AR is a nuclear transcription factor comprising three functional domains. Mutations are distributed throughout the gene, predominantly in 5 of the 8 exons that code for the ligand binding domain. The CAIS phenotype is associated with an AR mutation that completely disrupts AR function. The condition is X-linked recessive. [from SNOMEDCT_US]

Professional guidelines

PubMed

Lanciotti L, Cofini M, Leonardi A, Bertozzi M, Penta L, Esposito S
Int J Environ Res Public Health 2019 Apr 9;16(7) doi: 10.3390/ijerph16071268. PMID: 30970592Free PMC Article
Bertelloni S, Dati E, Baroncelli GI, Hiort O
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Recent clinical studies

Etiology

Tyutyusheva N, Mancini I, Baroncelli GI, D'Elios S, Peroni D, Meriggiola MC, Bertelloni S
Int J Mol Sci 2021 Jan 27;22(3) doi: 10.3390/ijms22031264. PMID: 33514065Free PMC Article
Godfrey LM
J Health Psychol 2021 Jan;26(1):40-59. Epub 2020 Mar 5 doi: 10.1177/1359105320909863. PMID: 32133887
Lanciotti L, Cofini M, Leonardi A, Bertozzi M, Penta L, Esposito S
Int J Environ Res Public Health 2019 Apr 9;16(7) doi: 10.3390/ijerph16071268. PMID: 30970592Free PMC Article
Hughes IA, Werner R, Bunch T, Hiort O
Semin Reprod Med 2012 Oct;30(5):432-42. Epub 2012 Oct 8 doi: 10.1055/s-0032-1324728. PMID: 23044881
Nakhal RS, Creighton SM
J Pediatr Adolesc Gynecol 2012 Dec;25(6):352-7. Epub 2011 Aug 27 doi: 10.1016/j.jpag.2011.06.003. PMID: 21872517

Diagnosis

Bakker J
J Neuroendocrinol 2022 Feb;34(2):e13050. Epub 2021 Oct 27 doi: 10.1111/jne.13050. PMID: 34708466
Babu R, Shah U
J Pediatr Urol 2021 Feb;17(1):39-47. Epub 2020 Nov 12 doi: 10.1016/j.jpurol.2020.11.017. PMID: 33246831
Kubo H, Kozan H, Kawai M
Pediatr Int 2021 Jan;63(1):122-123. doi: 10.1111/ped.14404. PMID: 32735744
Lanciotti L, Cofini M, Leonardi A, Bertozzi M, Penta L, Esposito S
Int J Environ Res Public Health 2019 Apr 9;16(7) doi: 10.3390/ijerph16071268. PMID: 30970592Free PMC Article
Hughes IA, Werner R, Bunch T, Hiort O
Semin Reprod Med 2012 Oct;30(5):432-42. Epub 2012 Oct 8 doi: 10.1055/s-0032-1324728. PMID: 23044881

Therapy

Auer MK, Birnbaum W, Hartmann MF, Holterhus PM, Kulle A, Lux A, Marshall L, Rall K, Richter-Unruh A, Werner R, Wudy SA, Hiort O
Endocrine 2022 Jun;76(3):722-732. Epub 2022 Mar 8 doi: 10.1007/s12020-022-03017-8. PMID: 35258786Free PMC Article
Tyutyusheva N, Mancini I, Baroncelli GI, D'Elios S, Peroni D, Meriggiola MC, Bertelloni S
Int J Mol Sci 2021 Jan 27;22(3) doi: 10.3390/ijms22031264. PMID: 33514065Free PMC Article
Lanciotti L, Cofini M, Leonardi A, Bertozzi M, Penta L, Esposito S
Int J Environ Res Public Health 2019 Apr 9;16(7) doi: 10.3390/ijerph16071268. PMID: 30970592Free PMC Article
King TFJ, Wat WZM, Creighton SM, Conway GS
Clin Endocrinol (Oxf) 2017 Aug;87(2):136-140. Epub 2017 Jun 8 doi: 10.1111/cen.13368. PMID: 28493277
Bertelloni S, Dati E, Baroncelli GI, Hiort O
Horm Res Paediatr 2011;76(6):428-33. Epub 2011 Nov 29 doi: 10.1159/000334162. PMID: 22156544

Prognosis

Kumar A, Sharma R, Faruq M, Kumar M, Sharma S, Werner R, Hiort O, Vandana J
Sex Dev 2022;16(1):34-45. Epub 2021 Oct 22 doi: 10.1159/000519047. PMID: 34689141
Lanciotti L, Cofini M, Leonardi A, Bertozzi M, Penta L, Esposito S
Int J Environ Res Public Health 2019 Apr 9;16(7) doi: 10.3390/ijerph16071268. PMID: 30970592Free PMC Article
Cools M, Looijenga L
Sex Dev 2017;11(4):175-181. Epub 2017 Jul 19 doi: 10.1159/000477921. PMID: 28719895
Kaprova-Pleskacova J, Stoop H, Brüggenwirth H, Cools M, Wolffenbuttel KP, Drop SL, Snajderova M, Lebl J, Oosterhuis JW, Looijenga LH
Mod Pathol 2014 May;27(5):721-30. Epub 2013 Nov 1 doi: 10.1038/modpathol.2013.193. PMID: 24186138
Shroff R, Rajesh U, Srivastava G, Morgan AB, Kevelighan E
J Obstet Gynaecol 2005 Oct;25(7):719-20. doi: 10.1080/01443610500307334. PMID: 16263555

Clinical prediction guides

Liu Q, Yin X, Li P
J Endocrinol Invest 2023 Jan;46(1):151-158. Epub 2022 Aug 16 doi: 10.1007/s40618-022-01894-4. PMID: 35974208Free PMC Article
Kumar A, Sharma R, Faruq M, Kumar M, Sharma S, Werner R, Hiort O, Vandana J
Sex Dev 2022;16(1):34-45. Epub 2021 Oct 22 doi: 10.1159/000519047. PMID: 34689141
Hornig NC, Holterhus PM
Mol Cell Endocrinol 2021 Mar 1;523:111146. Epub 2020 Dec 29 doi: 10.1016/j.mce.2020.111146. PMID: 33385475
King TFJ, Wat WZM, Creighton SM, Conway GS
Clin Endocrinol (Oxf) 2017 Aug;87(2):136-140. Epub 2017 Jun 8 doi: 10.1111/cen.13368. PMID: 28493277
Wisniewski A, Aston CE
Curr Pediatr Rev 2015;11(1):27-35. doi: 10.2174/1573396311666150501003541. PMID: 25938375

Recent systematic reviews

Babu R, Shah U
J Pediatr Urol 2021 Feb;17(1):39-47. Epub 2020 Nov 12 doi: 10.1016/j.jpurol.2020.11.017. PMID: 33246831
Godfrey LM
J Health Psychol 2021 Jan;26(1):40-59. Epub 2020 Mar 5 doi: 10.1177/1359105320909863. PMID: 32133887

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