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Congenital short bowel syndrome, autosomal recessive

MedGen UID:
979371
Concept ID:
CN296805
Disease or Syndrome
Synonyms: congenital short bowel syndrome 1; congenital short bowel syndrome due to CLMP variation; congenital short bowel syndrome, autosomal recessive
 
Gene (location): CLMP (11q24.1)
 
Monarch Initiative: MONDO:0020718
OMIM®: 615237

Definition

Infants with congenital short bowel syndrome (CSBS) are born with a shortened small intestine, with a mean length of 50 cm compared to the normal length of 190 to 280 cm, and intestinal malrotation. Severe malnutrition develops as a result of the hugely reduced absorptive surface of the small intestine, and infants require parenteral nutrition for survival; however, parenteral nutrition itself causes life-threatening complications such as sepsis and liver failure which are associated with a high rate of mortality early in life (summary by van der Werf et al., 2012). A possible form of congenital short bowel syndrome (see 300048) is caused by mutation in the FLNA gene (300017) on chromosome Xq28. [from OMIM]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  

Professional guidelines

PubMed

Alves MM, Halim D, Maroofian R, de Graaf BM, Rooman R, van der Werf CS, Van de Vijver E, Mehrjardi MY, Aflatoonian M, Chioza BA, Baple EL, Dehghani M, Crosby AH, Hofstra RM
Eur J Hum Genet 2016 Nov;24(11):1627-1629. Epub 2016 Jun 29 doi: 10.1038/ejhg.2016.58. PMID: 27352967Free PMC Article

Recent clinical studies

Therapy

Bax BE, Bain MD, Scarpelli M, Filosto M, Tonin P, Moran N
Neurology 2013 Oct 1;81(14):1269-71. Epub 2013 Aug 21 doi: 10.1212/WNL.0b013e3182a6cb4b. PMID: 23966250Free PMC Article
Antonucci A, Fronzoni L, Cogliandro L, Cogliandro RF, Caputo C, De Giorgio R, Pallotti F, Barbara G, Corinaldesi R, Stanghellini V
World J Gastroenterol 2008 May 21;14(19):2953-61. doi: 10.3748/wjg.14.2953. PMID: 18494042Free PMC Article

Prognosis

Devavarapu PKV, Uppaluri KR, Nikhade VA, Palasamudram K, Sri Manjari K
Clin J Gastroenterol 2024 Jun;17(3):383-395. Epub 2024 Mar 9 doi: 10.1007/s12328-024-01934-x. PMID: 38461165
Nakamura H, O'Donnell AM, Puri P
Pediatr Surg Int 2019 Feb;35(2):175-180. Epub 2018 Nov 1 doi: 10.1007/s00383-018-4390-6. PMID: 30386895
Farrell SA, Sodhi S, Marshall CR, Guerin A, Slavotinek A, Paton T, Chong K, Sirkin WL, Scherer SW, Bérubé-Simard FA, Pilon N
Am J Med Genet A 2017 Nov;173(11):3070-3074. Epub 2017 Sep 12 doi: 10.1002/ajmg.a.38354. PMID: 28898547
Halter JP, Michael W, Schüpbach M, Mandel H, Casali C, Orchard K, Collin M, Valcarcel D, Rovelli A, Filosto M, Dotti MT, Marotta G, Pintos G, Barba P, Accarino A, Ferra C, Illa I, Beguin Y, Bakker JA, Boelens JJ, de Coo IF, Fay K, Sue CM, Nachbaur D, Zoller H, Sobreira C, Pinto Simoes B, Hammans SR, Savage D, Martí R, Chinnery PF, Elhasid R, Gratwohl A, Hirano M
Brain 2015 Oct;138(Pt 10):2847-58. Epub 2015 Aug 10 doi: 10.1093/brain/awv226. PMID: 26264513Free PMC Article
Cucchiara S, Borrelli O
J Pediatr Gastroenterol Nutr 2009 Apr;48 Suppl 2:S83-5. doi: 10.1097/MPG.0b013e3181a15bfe. PMID: 19300134

Clinical prediction guides

Farrell SA, Sodhi S, Marshall CR, Guerin A, Slavotinek A, Paton T, Chong K, Sirkin WL, Scherer SW, Bérubé-Simard FA, Pilon N
Am J Med Genet A 2017 Nov;173(11):3070-3074. Epub 2017 Sep 12 doi: 10.1002/ajmg.a.38354. PMID: 28898547
Moreno CA, Metze K, Lomazi EA, Bertola DR, Barbosa RH, Cosentino V, Sobreira N, Cavalcanti DP
Am J Med Genet A 2016 Nov;170(11):2965-2974. Epub 2016 Aug 2 doi: 10.1002/ajmg.a.37857. PMID: 27481187Free PMC Article
Moore SW
Pediatr Surg Int 2012 Nov;28(11):1045-58. Epub 2012 Sep 23 doi: 10.1007/s00383-012-3175-6. PMID: 23001136
Garone C, Tadesse S, Hirano M
Brain 2011 Nov;134(Pt 11):3326-32. Epub 2011 Sep 20 doi: 10.1093/brain/awr245. PMID: 21933806Free PMC Article
Hirano M, Garcia-de-Yebenes J, Jones AC, Nishino I, DiMauro S, Carlo JR, Bender AN, Hahn AF, Salberg LM, Weeks DE, Nygaard TG
Am J Hum Genet 1998 Aug;63(2):526-33. doi: 10.1086/301979. PMID: 9683610Free PMC Article

Recent systematic reviews

Negri E, Coletta R, Morabito A
J Pediatr Surg 2020 Sep;55(9):1809-1814. Epub 2020 Mar 23 doi: 10.1016/j.jpedsurg.2020.03.009. PMID: 32278545
Nakamura H, O'Donnell AM, Puri P
Pediatr Surg Int 2019 Feb;35(2):175-180. Epub 2018 Nov 1 doi: 10.1007/s00383-018-4390-6. PMID: 30386895
Mc Laughlin D, Puri P
Pediatr Surg Int 2013 Sep;29(9):947-51. doi: 10.1007/s00383-013-3357-x. PMID: 23955298

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