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CEBPE-associated autoinflammation-immunodeficiency-neutrophil dysfunction syndrome

MedGen UID:
1799987
Concept ID:
C5568564
Disease or Syndrome
Synonyms: CAIN; CAIN (CEBPE-associated autoinflammation, immunodeficiency, neutrophil dysfunction) syndrome; CCAAT enhancer binding protein epsilon-associated autoinflammation, immunodeficiency, neutrophil dysfunction syndrome; CEBPE-associated autoinflammation, immunodeficiency, neutrophil dysfunction syndrome
SNOMED CT: CCAAT enhancer binding protein epsilon-associated autoinflammation, immunodeficiency, neutrophil dysfunction syndrome (1186720006); CAIN (CEBPE-associated autoinflammation, immunodeficiency, neutrophil dysfunction) syndrome (1186720006); CEBPE-associated autoinflammation, immunodeficiency, neutrophil dysfunction syndrome (1186720006)
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:0035437
Orphanet: ORPHA566067

Definition

A rare genetic syndrome with a combination of autoinflammation, immunodeficiency and neutrophil dysfunction, as well as mild bleeding diathesis. Patients present with recurrent attacks of abdominal pain, high fever, and systemic inflammation lasting four to five days and occurring every few weeks. Attacks may be accompanied by nailbed, tongue, submandibular and gluteal abscesses, intra-abdominal granulomas, pyoderma gangrenosum and buccal ulcerations. Frequent episodes of purulent paronychia, superficial skin and mucosal infections and purulent upper respiratory tract infections have also been reported. [from SNOMEDCT_US]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVCEBPE-associated autoinflammation-immunodeficiency-neutrophil dysfunction syndrome

Professional guidelines

PubMed

Lacy BE, Pimentel M, Brenner DM, Chey WD, Keefer LA, Long MD, Moshiree B
Am J Gastroenterol 2021 Jan 1;116(1):17-44. doi: 10.14309/ajg.0000000000001036. PMID: 33315591
Kellmann M, Bertollo M, Bosquet L, Brink M, Coutts AJ, Duffield R, Erlacher D, Halson SL, Hecksteden A, Heidari J, Kallus KW, Meeusen R, Mujika I, Robazza C, Skorski S, Venter R, Beckmann J
Int J Sports Physiol Perform 2018 Feb 1;13(2):240-245. Epub 2018 Feb 19 doi: 10.1123/ijspp.2017-0759. PMID: 29345524
Spasovski G, Vanholder R, Allolio B, Annane D, Ball S, Bichet D, Decaux G, Fenske W, Hoorn EJ, Ichai C, Joannidis M, Soupart A, Zietse R, Haller M, van der Veer S, Van Biesen W, Nagler E; Hyponatraemia Guideline Development Group
Eur J Endocrinol 2014 Mar;170(3):G1-47. Epub 2014 Feb 25 doi: 10.1530/EJE-13-1020. PMID: 24569125

Recent clinical studies

Etiology

Lambeck J, Hieber M, Dreßing A, Niesen WD
Dtsch Arztebl Int 2019 Sep 2;116(35-36):600-606. doi: 10.3238/arztebl.2019.0600. PMID: 31587708Free PMC Article
Rosenfeld E, Ganguly A, De Leon DD
Am J Med Genet C Semin Med Genet 2019 Dec;181(4):682-692. Epub 2019 Aug 14 doi: 10.1002/ajmg.c.31737. PMID: 31414570Free PMC Article
Fazl A, Fleisher J
Semin Pediatr Neurol 2018 Apr;25:2-9. Epub 2017 Dec 27 doi: 10.1016/j.spen.2017.12.005. PMID: 29735113Free PMC Article
Poh TY, Mac Aogáin M, Chan AK, Yii AC, Yong VF, Tiew PY, Koh MS, Chotirmall SH
Expert Rev Respir Med 2017 Apr;11(4):285-298. Epub 2017 Mar 24 doi: 10.1080/17476348.2017.1305895. PMID: 28282995
Ronco C, Haapio M, House AA, Anavekar N, Bellomo R
J Am Coll Cardiol 2008 Nov 4;52(19):1527-39. doi: 10.1016/j.jacc.2008.07.051. PMID: 19007588

Diagnosis

Kerber KA
Semin Neurol 2020 Feb;40(1):59-66. Epub 2020 Jan 28 doi: 10.1055/s-0039-3402739. PMID: 31994145
Lambeck J, Hieber M, Dreßing A, Niesen WD
Dtsch Arztebl Int 2019 Sep 2;116(35-36):600-606. doi: 10.3238/arztebl.2019.0600. PMID: 31587708Free PMC Article
Sahrmann S, Azevedo DC, Dillen LV
Braz J Phys Ther 2017 Nov-Dec;21(6):391-399. Epub 2017 Sep 27 doi: 10.1016/j.bjpt.2017.08.001. PMID: 29097026Free PMC Article
Poh TY, Mac Aogáin M, Chan AK, Yii AC, Yong VF, Tiew PY, Koh MS, Chotirmall SH
Expert Rev Respir Med 2017 Apr;11(4):285-298. Epub 2017 Mar 24 doi: 10.1080/17476348.2017.1305895. PMID: 28282995
Adityanjee, Munshi KR, Thampy A
Clin Neuropharmacol 2005 Jan-Feb;28(1):38-49. doi: 10.1097/01.wnf.0000150871.52253.b7. PMID: 15714160

Therapy

Traish AM
Fertil Steril 2020 Jan;113(1):21-50. doi: 10.1016/j.fertnstert.2019.11.030. PMID: 32033719
Kagan R, Kellogg-Spadt S, Parish SJ
Drugs Aging 2019 Oct;36(10):897-908. doi: 10.1007/s40266-019-00700-w. PMID: 31452067Free PMC Article
Sosa-Reina MD, Nunez-Nagy S, Gallego-Izquierdo T, Pecos-Martín D, Monserrat J, Álvarez-Mon M
Biomed Res Int 2017;2017:2356346. Epub 2017 Sep 20 doi: 10.1155/2017/2356346. PMID: 29291206Free PMC Article
Sahrmann S, Azevedo DC, Dillen LV
Braz J Phys Ther 2017 Nov-Dec;21(6):391-399. Epub 2017 Sep 27 doi: 10.1016/j.bjpt.2017.08.001. PMID: 29097026Free PMC Article
Adityanjee, Munshi KR, Thampy A
Clin Neuropharmacol 2005 Jan-Feb;28(1):38-49. doi: 10.1097/01.wnf.0000150871.52253.b7. PMID: 15714160

Prognosis

Roger VL
Circ Res 2021 May 14;128(10):1421-1434. Epub 2021 May 13 doi: 10.1161/CIRCRESAHA.121.318172. PMID: 33983838
Berthome P, Tixier R, Briand J, Geoffroy O, Babuty D, Mansourati J, Jesel L, Dupuis JM, Bru P, Kyndt F, Guyomarch B, Thollet A, Behar N, Mabo P, Sacher F, Probst V, Gourraud JB
Heart Rhythm 2019 Feb;16(2):260-267. Epub 2018 Sep 5 doi: 10.1016/j.hrthm.2018.08.032. PMID: 30193851
Razuin R, Effat O, Shahidan MN, Shama DV, Miswan MF
Malays J Pathol 2013 Jun;35(1):87-90. PMID: 23817399
Ageno W, Dentali F
Front Neurol Neurosci 2012;30:191-4. Epub 2012 Feb 14 doi: 10.1159/000333644. PMID: 22377893
Steinlein OK
Expert Rev Neurother 2009 Jun;9(6):825-33. doi: 10.1586/ern.09.49. PMID: 19496686

Clinical prediction guides

Kattah JC
Stroke Vasc Neurol 2018 Dec;3(4):190-196. Epub 2018 Jun 23 doi: 10.1136/svn-2018-000160. PMID: 30637123Free PMC Article
Poh TY, Mac Aogáin M, Chan AK, Yii AC, Yong VF, Tiew PY, Koh MS, Chotirmall SH
Expert Rev Respir Med 2017 Apr;11(4):285-298. Epub 2017 Mar 24 doi: 10.1080/17476348.2017.1305895. PMID: 28282995
Karussis D
J Autoimmun 2014 Feb-Mar;48-49:134-42. Epub 2014 Feb 10 doi: 10.1016/j.jaut.2014.01.022. PMID: 24524923
Wijdicks EF, Bamlet WR, Maramattom BV, Manno EM, McClelland RL
Ann Neurol 2005 Oct;58(4):585-93. doi: 10.1002/ana.20611. PMID: 16178024
Chrousos GP, Gold PW
JAMA 1992 Mar 4;267(9):1244-52. PMID: 1538563

Recent systematic reviews

Biswas S, Gomez J, Horgan R, Sibai BM, Saad A, Powel JE, Al-Kouatly HB
Am J Obstet Gynecol MFM 2023 Sep;5(9):101067. Epub 2023 Jun 28 doi: 10.1016/j.ajogmf.2023.101067. PMID: 37385374
Menon K, Schlapbach LJ, Akech S, Argent A, Biban P, Carrol ED, Chiotos K, Jobayer Chisti M, Evans IVR, Inwald DP, Ishimine P, Kissoon N, Lodha R, Nadel S, Oliveira CF, Peters M, Sadeghirad B, Scott HF, de Souza DC, Tissieres P, Watson RS, Wiens MO, Wynn JL, Zimmerman JJ, Sorce LR; Pediatric Sepsis Definition Taskforce of the Society of Critical Care Medicine
Crit Care Med 2022 Jan 1;50(1):21-36. doi: 10.1097/CCM.0000000000005294. PMID: 34612847Free PMC Article
Sosa-Reina MD, Nunez-Nagy S, Gallego-Izquierdo T, Pecos-Martín D, Monserrat J, Álvarez-Mon M
Biomed Res Int 2017;2017:2356346. Epub 2017 Sep 20 doi: 10.1155/2017/2356346. PMID: 29291206Free PMC Article
Payne LE, Gagnon DJ, Riker RR, Seder DB, Glisic EK, Morris JG, Fraser GL
Crit Care 2017 Nov 14;21(1):276. doi: 10.1186/s13054-017-1856-1. PMID: 29137682Free PMC Article
Sorensen CJ, DeSanto K, Borgelt L, Phillips KT, Monte AA
J Med Toxicol 2017 Mar;13(1):71-87. Epub 2016 Dec 20 doi: 10.1007/s13181-016-0595-z. PMID: 28000146Free PMC Article

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