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Jeune thoracic dystrophy(ATD1)

MedGen UID:
78548
Concept ID:
C0265275
Disease or Syndrome
Synonyms: Asphyxiating Thoracic Dystrophy; ATD1; Chondroectodermal dysplasia-like syndrome; Infantile thoracic dystrophy; Jeune syndrome; Jeune's syndrome; Short-rib thoracic dysplasia; Thoracic pelvic phalangeal dystrophy
SNOMED CT: Jeune thoracic dysplasia (75049004); Jeune syndrome (75049004); Jeune thoracic dystrophy (75049004); Asphyxiating thoracic dystrophy (75049004)
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).
 
Related genes: TTC21B, DYNC2H1, WDR19, IFT80
 
Monarch Initiative: MONDO:0018770
OMIM®: 208500
OMIM® Phenotypic series: PS208500
Orphanet: ORPHA474

Definition

Asphyxiating thoracic dystrophy, also known as Jeune syndrome, is an inherited disorder of bone growth characterized by a narrow chest, short ribs, shortened bones in the arms and legs, short stature, and extra fingers and toes (polydactyly). Additional skeletal abnormalities can include unusually shaped collarbones (clavicles) and pelvic bones, and and cone-shaped ends of the long bones in the arms and legs. Many infants with this condition are born with an extremely narrow, bell-shaped chest that can restrict the growth and expansion of the lungs. Life-threatening problems with breathing result, and people with asphyxiating thoracic dystrophy may live only into infancy or early childhood. However, in people who survive beyond the first few years, the narrow chest and related breathing problems can improve with age.

Some people with asphyxiating thoracic dystrophy are born with less severe skeletal abnormalities and have only mild breathing difficulties, such as rapid breathing or shortness of breath. These individuals may live into adolescence or adulthood. After infancy, people with this condition may develop life-threatening kidney (renal) abnormalities that cause the kidneys to malfunction or fail. Heart defects and a narrowing of the airway (subglottic stenosis) are also possible. Other, less common features of asphyxiating thoracic dystrophy include liver disease, fluid-filled sacs (cysts) in the pancreas, dental abnormalities, and an eye disease called retinal dystrophy that can lead to vision loss. [from MedlinePlus Genetics]

Professional guidelines

PubMed

Tsukahara K, Mayer OH
Paediatr Respir Rev 2022 Dec;44:78-84. Epub 2022 Mar 7 doi: 10.1016/j.prrv.2022.02.003. PMID: 35339395Free PMC Article
Adamo L, Kassif E, Jacobson JM, Achiron R
J Ultrasound Med 2020 Sep;39(9):1869-1871. Epub 2020 Mar 10 doi: 10.1002/jum.15266. PMID: 32154601
Mayer OH
Paediatr Respir Rev 2015 Jan;16(1):30-4. Epub 2014 Nov 5 doi: 10.1016/j.prrv.2014.10.012. PMID: 25434647

Recent clinical studies

Diagnosis

Smith C, Lamont RE, Wade A, Bernier FP, Parboosingh JS, Innes AM
Am J Med Genet A 2016 Mar;170(3):760-5. Epub 2015 Dec 22 doi: 10.1002/ajmg.a.37514. PMID: 26691894

Prognosis

Smith C, Lamont RE, Wade A, Bernier FP, Parboosingh JS, Innes AM
Am J Med Genet A 2016 Mar;170(3):760-5. Epub 2015 Dec 22 doi: 10.1002/ajmg.a.37514. PMID: 26691894
Kalappanavar NK, Bidhu P, Kannan B, Devanand M, Chidanand S
Indian J Pediatr 2005 Mar;72(3):269. PMID: 15812132

Clinical prediction guides

Smith C, Lamont RE, Wade A, Bernier FP, Parboosingh JS, Innes AM
Am J Med Genet A 2016 Mar;170(3):760-5. Epub 2015 Dec 22 doi: 10.1002/ajmg.a.37514. PMID: 26691894

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