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Mandibulofacial dysostosis

MedGen UID:
505796
Concept ID:
CN004722
Disease or Syndrome
Synonyms: Bilateral and symmetric oto-mandibular dysplasia; bilateral and symmetric oto-mandibular dysplasia; mandibulofacial dysostosis; Treacher Collins syndrome
 
Related genes: POLR1B, POLR1D, POLR1C, TCOF1, EDNRA
 
HPO: HP:0005321
Monarch Initiative: MONDO:0015483
Orphanet: ORPHA155899

Definition

Treacher Collins syndrome (TCS) is a disorder of craniofacial development. The features include downslanting palpebral fissures, coloboma of the eyelid, micrognathia, microtia and other deformity of the ears, hypoplastic zygomatic arches, and macrostomia. Conductive hearing loss and cleft palate are often present (summary by Dixon, 1996). Genetic Heterogeneity of Treacher Collins Syndrome Treacher Collins syndrome-2 (TCS2; 613717) is caused by mutation in the POLR1D gene (613715) on chromosome 13q12. Treacher Collins syndrome-3 (TCS3; 248390) is caused by mutation in the POLR1C gene (610060) on chromosome 6p21. Treacher Collins syndrome-4 (TCS4; 618939) is caused by mutation in the POLR1B gene (602000) on chromosome 2q14. [from OMIM]

Conditions with this feature

Mandibulofacial dysostosis with ptosis, autosomal dominant
MedGen UID:
331276
Concept ID:
C1842349
Disease or Syndrome
Treacher Collins syndrome 3
MedGen UID:
340868
Concept ID:
C1855433
Disease or Syndrome
Treacher Collins syndrome (TCS) is characterized by bilateral and symmetric downslanting palpebral fissures, malar hypoplasia, micrognathia, and external ear abnormalities. Hypoplasia of the zygomatic bones and mandible can cause significant feeding and respiratory difficulties. About 40%-50% of individuals have conductive hearing loss attributed most commonly to malformation of the ossicles and hypoplasia of the middle ear cavities. Inner ear structures tend to be normal. Other, less common abnormalities include cleft palate and unilateral or bilateral choanal stenosis or atresia. Typically intellect is normal.
Mandibulofacial dysostosis-microcephaly syndrome
MedGen UID:
355264
Concept ID:
C1864652
Disease or Syndrome
Mandibulofacial dysostosis with microcephaly (MFDM) is characterized by malar and mandibular hypoplasia, microcephaly (congenital or postnatal onset), intellectual disability (mild, moderate, or severe), malformations of the external ear, and hearing loss that is typically conductive. Associated craniofacial malformations may include cleft palate, choanal atresia, zygomatic arch cleft (identified on cranial CT scan), and facial asymmetry. Other relatively common findings (present in 25%-35% of individuals) can include cardiac anomalies, thumb anomalies, esophageal atresia/tracheoesophageal fistula, short stature, spine anomalies, and epilepsy.
Mandibulofacial dysostosis-macroblepharon-macrostomia syndrome
MedGen UID:
355927
Concept ID:
C1865181
Disease or Syndrome
Mandibulofacial dysostosis-macroblepharon-macrostomia syndrome is a rare developmental defect during embryogenesis disorder characterized by macroblepharon, ectropion, and facial dysmorphism which includes severe hypertelorism, downslanting palpebral fissures, posteriorly rotated ears, broad nasal bridge, long and smooth philtrum, and macrostomia with thin upper lip vermilion border. Other features may include large fontanelles, prominent metopic ridge, thick eyebrows, mild synophrys, increased density of upper eyelashes, anterverted nares, abnormal dentition and capillary hemangioma.
Diamond-Blackfan anemia 10
MedGen UID:
412873
Concept ID:
C2750080
Disease or Syndrome
Diamond-Blackfan anemia (DBA) is characterized by a profound normochromic and usually macrocytic anemia with normal leukocytes and platelets, congenital malformations in up to 50%, and growth deficiency in 30% of affected individuals. The hematologic complications occur in 90% of affected individuals during the first year of life. The phenotypic spectrum ranges from a mild form (e.g., mild anemia or no anemia with only subtle erythroid abnormalities, physical malformations without anemia) to a severe form of fetal anemia resulting in nonimmune hydrops fetalis. DBA is associated with an increased risk for acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS), and solid tumors including osteogenic sarcoma.
Acrofacial dysostosis, Catania type
MedGen UID:
419487
Concept ID:
C2931762
Disease or Syndrome
The Catania type of acrofacial dysostosis is characterized by intrauterine growth retardation, short stature, microcephaly, intellectual disability, widow's peak, mandibulofacial dysostosis without cleft palate, ear anomalies, mild pre- and postaxial limb hypoplasia with brachydactyly, mild interdigital webbing, dental anomalies, and cryptorchidism and hypospadias in males (Opitz et al., 1993; Wulfsberg et al., 1996).
Mandibulofacial dysostosis with alopecia
MedGen UID:
898794
Concept ID:
C4225349
Disease or Syndrome
A rare mandibulofacial dysostosis with the association with scalp alopecia and sparse eyebrows and eyelashes. Craniofacial dysmorphic features include zygomatic and mandibular dysplasia or hypoplasia, cleft palate, micrognathia, dental anomalies, auricular dysmorphism and eyelid anomalies among others. Patients may experience limited jaw mobility, glossoptosis, upper airway obstruction and conductive hearing loss.
Patterson-Stevenson-Fontaine syndrome
MedGen UID:
1808766
Concept ID:
C5574964
Disease or Syndrome
A very rare variant of acrofacial dysostosis with characteristics of mandibulofacial dysostosis and limb anomalies. It has been described in less than ten patients. The mandibulofacial dysostosis consists of retrognathism, complete or occult posterior cleft palate and anomalies of the external ears. Limb anomalies consist of split-foot deformity with syndactyly of some toes. The condition is transmitted as an autosomal dominant trait with variable penetrance and expressivity.

Professional guidelines

PubMed

Marszałek-Kruk BA, Wójcicki P, Dowgierd K, Śmigiel R
Genes (Basel) 2021 Sep 9;12(9) doi: 10.3390/genes12091392. PMID: 34573374Free PMC Article
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Recent clinical studies

Etiology

Beaumont CA, Dunaway DJ, Padwa BL, Forrest C, Koudstaal MJ, Caron CJJM
Int J Oral Maxillofac Surg 2021 Nov;50(11):1471-1476. Epub 2021 Mar 19 doi: 10.1016/j.ijom.2021.03.001. PMID: 33752939
Amirhassankhani S, Lloyd MS
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Diagnosis

Marszałek-Kruk BA, Wójcicki P, Dowgierd K, Śmigiel R
Genes (Basel) 2021 Sep 9;12(9) doi: 10.3390/genes12091392. PMID: 34573374Free PMC Article
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Therapy

Neophytou P, Artemiadis A, Hadjigeorgiou GM, Zis P
Acta Neurol Belg 2023 Oct;123(5):1693-1701. Epub 2023 Jul 19 doi: 10.1007/s13760-023-02336-5. PMID: 37468803Free PMC Article
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Prognosis

Neophytou P, Artemiadis A, Hadjigeorgiou GM, Zis P
Acta Neurol Belg 2023 Oct;123(5):1693-1701. Epub 2023 Jul 19 doi: 10.1007/s13760-023-02336-5. PMID: 37468803Free PMC Article
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Clinical prediction guides

Smallwood K, Watt KEN, Ide S, Baltrunaite K, Brunswick C, Inskeep K, Capannari C, Adam MP, Begtrup A, Bertola DR, Demmer L, Demo E, Devinsky O, Gallagher ER, Guillen Sacoto MJ, Jech R, Keren B, Kussmann J, Ladda R, Lansdon LA, Lunke S, Mardy A, McWalters K, Person R, Raiti L, Saitoh N, Saunders CJ, Schnur R, Skorvanek M, Sell SL, Slavotinek A, Sullivan BR, Stark Z, Symonds JD, Wenger T, Weber S, Whalen S, White SM, Winkelmann J, Zech M, Zeidler S, Maeshima K, Stottmann RW, Trainor PA, Weaver KN
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Splendore A, Silva EO, Alonso LG, Richieri-Costa A, Alonso N, Rosa A, Carakushanky G, Cavalcanti DP, Brunoni D, Passos-Bueno MR
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Recent systematic reviews

Neophytou P, Artemiadis A, Hadjigeorgiou GM, Zis P
Acta Neurol Belg 2023 Oct;123(5):1693-1701. Epub 2023 Jul 19 doi: 10.1007/s13760-023-02336-5. PMID: 37468803Free PMC Article
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Amirhassankhani S, Lloyd MS
J Craniofac Surg 2018 Mar;29(2):372-375. doi: 10.1097/SCS.0000000000004201. PMID: 29239919
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