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Anauxetic dysplasia(ANXD)

MedGen UID:
375972
Concept ID:
C1846796
Disease or Syndrome
Synonym: SPONDYLOMETAEPIPHYSEAL DYSPLASIA, ANAUXETIC TYPE
SNOMED CT: Anauxetic dysplasia (764460003); Spondyloepimetaphyseal dysplasia Menger type (764460003); Spondyloepimetaphyseal dysplasia anauxetic type (764460003)
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:0011773
OMIM®: 607095
OMIM® Phenotypic series: PS607095
Orphanet: ORPHA93347

Definition

Anauxetic dysplasia (ANXD1) is a form of spondylometaepiphyseal dysplasia characterized by the prenatal onset of extreme short stature, an adult height of less than 85 cm, hypodontia, and mild mental retardation. Major radiographic characteristics are late-maturing ovoid vertebral bodies with concave dorsal surfaces in the lumbar region; small capital femoral epiphyses; hypoplastic femoral necks; hypoplastic iliac bodies and shallow acetabulae; irregular metaphyseal mineralization and demarcation of the long tubular bones; short first and fifth metacarpals with widened shafts; very short and broad phalanges with small, late-ossifying epiphyses and bullet-shaped middle phalanges; and midface hypoplasia. The number of chondrocytes is severely reduced in the resting and proliferating cartilage, with diminished columnization of the hypertrophic zone (summary by Thiel et al., 2005). Mutations in RMRP also cause 2 milder types of short stature with susceptibility to cancer, cartilage-hair hypoplasia (CHH; 250250) and metaphyseal dysplasia without hypotrichosis (250460). Genetic Heterogeneity of Anauxetic Dysplasia Anauxetic dysplasia-2 (ANXD2; 617396) is caused by mutation in the POP1 gene (602486) on chromosome 8q22. ANXD3 (618853) is caused by mutation in the NEPRO gene (617089) on chromosome 3q13. [from OMIM]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVAnauxetic dysplasia
Follow this link to review classifications for Anauxetic dysplasia in Orphanet.

Recent clinical studies

Etiology

Remmelzwaal PC, Verhagen MV, Jongbloed JDH, van den Akker PC, Veenstra-Knol HE, Hitzert MM
Am J Med Genet A 2023 Sep;191(9):2440-2445. Epub 2023 Jun 9 doi: 10.1002/ajmg.a.63316. PMID: 37294112
Akgün-Doğan Ö, Şimsek-Kiper PÖ, Utine GE, Boduroğlu K
Turk J Pediatr 2018;60(1):89-93. doi: 10.24953/turkjped.2018.01.014. PMID: 30102486
Garcia-Tarodo S, Bottani A, Merlini L, Kaelin A, Schwitzgebel VM, Parvex P, Dayer R, Lascombes P, Korff CM
Eur J Paediatr Neurol 2015 May;19(3):367-71. Epub 2015 Jan 3 doi: 10.1016/j.ejpn.2014.12.016. PMID: 25596067
Thiel CT, Mortier G, Kaitila I, Reis A, Rauch A
Am J Hum Genet 2007 Sep;81(3):519-29. Epub 2007 Aug 6 doi: 10.1086/521034. PMID: 17701897Free PMC Article

Diagnosis

Park JH, Im M, Kim YJ, Jang JH, Lee SM, Kim MS, Cho SY
Medicine (Baltimore) 2024 May 24;103(21):e37247. doi: 10.1097/MD.0000000000037247. PMID: 38787970Free PMC Article
Remmelzwaal PC, Verhagen MV, Jongbloed JDH, van den Akker PC, Veenstra-Knol HE, Hitzert MM
Am J Med Genet A 2023 Sep;191(9):2440-2445. Epub 2023 Jun 9 doi: 10.1002/ajmg.a.63316. PMID: 37294112
Hall CM, Liu B, Haworth A, Reed L, Pryce J, Mansour S
Eur J Med Genet 2021 Mar;64(3):104162. Epub 2021 Feb 7 doi: 10.1016/j.ejmg.2021.104162. PMID: 33567347
Elalaoui SC, Laarabi FZ, Mansouri M, Mrani NA, Nishimura G, Sefiani A
Am J Med Genet A 2016 Sep;170(9):2462-5. Epub 2016 Jul 6 doi: 10.1002/ajmg.a.37839. PMID: 27380734
Thiel CT, Mortier G, Kaitila I, Reis A, Rauch A
Am J Hum Genet 2007 Sep;81(3):519-29. Epub 2007 Aug 6 doi: 10.1086/521034. PMID: 17701897Free PMC Article

Prognosis

Narayanan DL, Shukla A, Kausthubham N, Bhavani GS, Shah H, Mortier G, Girisha KM
Am J Med Genet A 2019 Sep;179(9):1709-1717. Epub 2019 Jun 27 doi: 10.1002/ajmg.a.61267. PMID: 31250547
Garcia-Tarodo S, Bottani A, Merlini L, Kaelin A, Schwitzgebel VM, Parvex P, Dayer R, Lascombes P, Korff CM
Eur J Paediatr Neurol 2015 May;19(3):367-71. Epub 2015 Jan 3 doi: 10.1016/j.ejpn.2014.12.016. PMID: 25596067
Thiel CT, Mortier G, Kaitila I, Reis A, Rauch A
Am J Hum Genet 2007 Sep;81(3):519-29. Epub 2007 Aug 6 doi: 10.1086/521034. PMID: 17701897Free PMC Article

Clinical prediction guides

Narayanan DL, Shukla A, Kausthubham N, Bhavani GS, Shah H, Mortier G, Girisha KM
Am J Med Genet A 2019 Sep;179(9):1709-1717. Epub 2019 Jun 27 doi: 10.1002/ajmg.a.61267. PMID: 31250547
Barraza-García J, Rivera-Pedroza CI, Hisado-Oliva A, Belinchón-Martínez A, Sentchordi-Montané L, Duncan EL, Clark GR, Del Pozo A, Ibáñez-Garikano K, Offiah A, Prieto-Matos P, Cormier-Daire V, Heath KE
Clin Genet 2017 Jul;92(1):91-98. Epub 2017 Feb 22 doi: 10.1111/cge.12964. PMID: 28067412
Thiel CT, Mortier G, Kaitila I, Reis A, Rauch A
Am J Hum Genet 2007 Sep;81(3):519-29. Epub 2007 Aug 6 doi: 10.1086/521034. PMID: 17701897Free PMC Article

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