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Inherited pseudoxanthoma elasticum

MedGen UID:
697574
Concept ID:
C1274225
Disease or Syndrome
Synonyms: inherited Gronblad Strandberg syndrome; inherited pseudoxanthoma elasticum; inherited PXE
SNOMED CT: Inherited pseudoxanthoma elasticum (402782006)
 
Related gene: ABCC6
 
Monarch Initiative: MONDO:0100091

Definition

An inheritable form of pseudoxanthoma elasticum (PXE), that causes calcium and other minerals to accumulate in the elastic fibers of the skin, eyes, and blood vessels, and less frequently in other areas such as the digestive tract. PXE may cause the following symptoms: growth of yellowish bumps on the skin of the neck, under the arms, or in the groin area; reduced vision; periodic weakness in the legs (claudication); or bleeding in the gastrointestinal tract, particularly the stomach. A clinical diagnosis of PXE can be made when an individual is found to have both the characteristic eye findings and yellow bumps on the skin. ABCC6 is the only gene known to be associated with this condition. Currently, there is no treatment for this condition, but affected individuals may benefit from routine visits to an eye doctor who specializes in retinal disorders, and by having regular physical examinationswith their primary physician. [from MONDO]

Professional guidelines

PubMed

Nitschke Y, Rutsch F
Curr Osteoporos Rep 2017 Aug;15(4):255-270. doi: 10.1007/s11914-017-0370-3. PMID: 28585220
De Vilder EY, Debacker J, Vanakker OM
Int J Mol Sci 2017 Jan 25;18(2) doi: 10.3390/ijms18020240. PMID: 28125048Free PMC Article
Georgalas I, Tservakis I, Papaconstaninou D, Kardara M, Koutsandrea C, Ladas I
Clin Exp Optom 2011 Mar;94(2):169-80. Epub 2010 Dec 29 doi: 10.1111/j.1444-0938.2010.00559.x. PMID: 21198842

Recent clinical studies

Diagnosis

Valbuena V, Assaad D, Yeung J
J Cutan Med Surg 2017 Jul/Aug;21(4):345-347. Epub 2017 Mar 10 doi: 10.1177/1203475417699407. PMID: 28282240
Aessopos A, Farmakis D, Loukopoulos D
Blood 2002 Jan 1;99(1):30-5. doi: 10.1182/blood.v99.1.30. PMID: 11756149
Coatesworth AP, Darnton SJ, Green RM, Cayton RM, Antonakopoulos GN
J Clin Pathol 1998 Feb;51(2):169-71. doi: 10.1136/jcp.51.2.169. PMID: 9602696Free PMC Article

Therapy

Valbuena V, Assaad D, Yeung J
J Cutan Med Surg 2017 Jul/Aug;21(4):345-347. Epub 2017 Mar 10 doi: 10.1177/1203475417699407. PMID: 28282240
Coatesworth AP, Darnton SJ, Green RM, Cayton RM, Antonakopoulos GN
J Clin Pathol 1998 Feb;51(2):169-71. doi: 10.1136/jcp.51.2.169. PMID: 9602696Free PMC Article

Prognosis

Valbuena V, Assaad D, Yeung J
J Cutan Med Surg 2017 Jul/Aug;21(4):345-347. Epub 2017 Mar 10 doi: 10.1177/1203475417699407. PMID: 28282240

Clinical prediction guides

Aessopos A, Floudas CS, Kati M, Tsironi M, Giakoumi X, Livir-Rallatos C, Farmakis D
Int J Hematol 2008 Jan;87(1):35-8. Epub 2007 Dec 11 doi: 10.1007/s12185-007-0014-y. PMID: 18224411

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