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Ataxia-telangiectasia without immunodeficiency

MedGen UID:
865539
Concept ID:
C4017102
Finding
Synonym: ATAXIA-TELANGIECTASIA WITHOUT IMMUNODEFICIENCY
 
OMIM®: 607585

Professional guidelines

PubMed

Amirifar P, Ranjouri MR, Lavin M, Abolhassani H, Yazdani R, Aghamohammadi A
Expert Rev Clin Immunol 2020 Sep;16(9):859-871. Epub 2020 Oct 15 doi: 10.1080/1744666X.2020.1810570. PMID: 32791865
Schon K, van Os NJH, Oscroft N, Baxendale H, Scoffings D, Ray J, Suri M, Whitehouse WP, Mehta PR, Everett N, Bottolo L, van de Warrenburg BP, Byrd PJ, Weemaes C, Willemsen MA, Tischkowitz M, Taylor AM, Hensiek AE
Ann Neurol 2019 Feb;85(2):170-180. doi: 10.1002/ana.25394. PMID: 30549301Free PMC Article
van Os NJH, Haaxma CA, van der Flier M, Merkus PJFM, van Deuren M, de Groot IJM, Loeffen J, van de Warrenburg BPC, Willemsen MAAP; A-T Study Group
Dev Med Child Neurol 2017 Jul;59(7):680-689. Epub 2017 Mar 20 doi: 10.1111/dmcn.13424. PMID: 28318010

Recent clinical studies

Etiology

Kim J, Woo S, de Gusmao CM, Zhao B, Chin DH, DiDonato RL, Nguyen MA, Nakayama T, Hu CA, Soucy A, Kuniholm A, Thornton JK, Riccardi O, Friedman DA, El Achkar CM, Dash Z, Cornelissen L, Donado C, Faour KNW, Bush LW, Suslovitch V, Lentucci C, Park PJ, Lee EA, Patterson A, Philippakis AA, Margus B, Berde CB, Yu TW
Nature 2023 Jul;619(7971):828-836. Epub 2023 Jul 12 doi: 10.1038/s41586-023-06277-0. PMID: 37438524Free PMC Article
Amirifar P, Ranjouri MR, Lavin M, Abolhassani H, Yazdani R, Aghamohammadi A
Expert Rev Clin Immunol 2020 Sep;16(9):859-871. Epub 2020 Oct 15 doi: 10.1080/1744666X.2020.1810570. PMID: 32791865
Schon K, van Os NJH, Oscroft N, Baxendale H, Scoffings D, Ray J, Suri M, Whitehouse WP, Mehta PR, Everett N, Bottolo L, van de Warrenburg BP, Byrd PJ, Weemaes C, Willemsen MA, Tischkowitz M, Taylor AM, Hensiek AE
Ann Neurol 2019 Feb;85(2):170-180. doi: 10.1002/ana.25394. PMID: 30549301Free PMC Article
van Os NJH, Haaxma CA, van der Flier M, Merkus PJFM, van Deuren M, de Groot IJM, Loeffen J, van de Warrenburg BPC, Willemsen MAAP; A-T Study Group
Dev Med Child Neurol 2017 Jul;59(7):680-689. Epub 2017 Mar 20 doi: 10.1111/dmcn.13424. PMID: 28318010
Micol R, Ben Slama L, Suarez F, Le Mignot L, Beauté J, Mahlaoui N, Dubois d'Enghien C, Laugé A, Hall J, Couturier J, Vallée L, Delobel B, Rivier F, Nguyen K, Billette de Villemeur T, Stephan JL, Bordigoni P, Bertrand Y, Aladjidi N, Pedespan JM, Thomas C, Pellier I, Koenig M, Hermine O, Picard C, Moshous D, Neven B, Lanternier F, Blanche S, Tardieu M, Debré M, Fischer A, Stoppa-Lyonnet D; CEREDIH Network Investigators
J Allergy Clin Immunol 2011 Aug;128(2):382-9.e1. Epub 2011 Jun 12 doi: 10.1016/j.jaci.2011.03.052. PMID: 21665257

Diagnosis

Fortuna J, Rodrigues AL, Pires P
Pediatr Neonatol 2022 Sep;63(5):551-552. Epub 2022 Mar 23 doi: 10.1016/j.pedneo.2022.01.004. PMID: 35431147
Amirifar P, Ranjouri MR, Lavin M, Abolhassani H, Yazdani R, Aghamohammadi A
Expert Rev Clin Immunol 2020 Sep;16(9):859-871. Epub 2020 Oct 15 doi: 10.1080/1744666X.2020.1810570. PMID: 32791865
van Os NJH, Haaxma CA, van der Flier M, Merkus PJFM, van Deuren M, de Groot IJM, Loeffen J, van de Warrenburg BPC, Willemsen MAAP; A-T Study Group
Dev Med Child Neurol 2017 Jul;59(7):680-689. Epub 2017 Mar 20 doi: 10.1111/dmcn.13424. PMID: 28318010
Rothblum-Oviatt C, Wright J, Lefton-Greif MA, McGrath-Morrow SA, Crawford TO, Lederman HM
Orphanet J Rare Dis 2016 Nov 25;11(1):159. doi: 10.1186/s13023-016-0543-7. PMID: 27884168Free PMC Article
Perlman SL, Boder Deceased E, Sedgewick RP, Gatti RA
Handb Clin Neurol 2012;103:307-32. doi: 10.1016/B978-0-444-51892-7.00019-X. PMID: 21827897

Therapy

Kim J, Woo S, de Gusmao CM, Zhao B, Chin DH, DiDonato RL, Nguyen MA, Nakayama T, Hu CA, Soucy A, Kuniholm A, Thornton JK, Riccardi O, Friedman DA, El Achkar CM, Dash Z, Cornelissen L, Donado C, Faour KNW, Bush LW, Suslovitch V, Lentucci C, Park PJ, Lee EA, Patterson A, Philippakis AA, Margus B, Berde CB, Yu TW
Nature 2023 Jul;619(7971):828-836. Epub 2023 Jul 12 doi: 10.1038/s41586-023-06277-0. PMID: 37438524Free PMC Article
Fischer A
Curr Opin Immunol 1989-1990 Feb;2(3):439-44. doi: 10.1016/0952-7915(89)90157-x. PMID: 2483886
Cunlift PN, Mann JR, Cameron AH, Roberts KD, Ward HN
Br J Radiol 1975 May;48(569):374-6. doi: 10.1259/0007-1285-48-569-374. PMID: 1139093
Hitzig WH
J Clin Pathol Suppl (Assoc Clin Pathol) 1975;6:83-91. doi: 10.1136/jcp.s1-6.1.83. PMID: 802876Free PMC Article
Rosen FS
Pediatr Clin North Am 1974 Aug;21(3):533-49. doi: 10.1016/s0031-3955(16)33023-1. PMID: 4137895

Prognosis

Amirifar P, Ranjouri MR, Lavin M, Abolhassani H, Yazdani R, Aghamohammadi A
Expert Rev Clin Immunol 2020 Sep;16(9):859-871. Epub 2020 Oct 15 doi: 10.1080/1744666X.2020.1810570. PMID: 32791865
Pearson TS, Pons R, Ghaoui R, Sue CM
Mov Disord 2019 May;34(5):625-636. Epub 2019 Mar 26 doi: 10.1002/mds.27655. PMID: 30913345
van Os NJH, Haaxma CA, van der Flier M, Merkus PJFM, van Deuren M, de Groot IJM, Loeffen J, van de Warrenburg BPC, Willemsen MAAP; A-T Study Group
Dev Med Child Neurol 2017 Jul;59(7):680-689. Epub 2017 Mar 20 doi: 10.1111/dmcn.13424. PMID: 28318010
Rothblum-Oviatt C, Wright J, Lefton-Greif MA, McGrath-Morrow SA, Crawford TO, Lederman HM
Orphanet J Rare Dis 2016 Nov 25;11(1):159. doi: 10.1186/s13023-016-0543-7. PMID: 27884168Free PMC Article
Nissenkorn A, Ben-Zeev B
Handb Clin Neurol 2015;132:199-214. doi: 10.1016/B978-0-444-62702-5.00014-7. PMID: 26564081

Clinical prediction guides

Kim J, Woo S, de Gusmao CM, Zhao B, Chin DH, DiDonato RL, Nguyen MA, Nakayama T, Hu CA, Soucy A, Kuniholm A, Thornton JK, Riccardi O, Friedman DA, El Achkar CM, Dash Z, Cornelissen L, Donado C, Faour KNW, Bush LW, Suslovitch V, Lentucci C, Park PJ, Lee EA, Patterson A, Philippakis AA, Margus B, Berde CB, Yu TW
Nature 2023 Jul;619(7971):828-836. Epub 2023 Jul 12 doi: 10.1038/s41586-023-06277-0. PMID: 37438524Free PMC Article
Jackson TJ, Chow G, Suri M, Byrd P, Taylor MR, Whitehouse WP
Dev Med Child Neurol 2016 Jul;58(7):690-7. Epub 2016 Feb 19 doi: 10.1111/dmcn.13052. PMID: 26896183
Teraoka SN, Telatar M, Becker-Catania S, Liang T, Onengüt S, Tolun A, Chessa L, Sanal O, Bernatowska E, Gatti RA, Concannon P
Am J Hum Genet 1999 Jun;64(6):1617-31. doi: 10.1086/302418. PMID: 10330348Free PMC Article
Radford DM, Zehnbauer BA
Surg Clin North Am 1996 Apr;76(2):205-20. doi: 10.1016/s0039-6109(05)70434-5. PMID: 8610259
Teplitz RL
Arch Neurol 1978 Sep;35(9):553-4. doi: 10.1001/archneur.1978.00500330001001. PMID: 687181

Recent systematic reviews

Tiet MY, Guțu BI, Springall-Jeggo P, Coman D, Willemsen M, Van Os N, Doria M, Donath H, Schubert R, Dineen RA, Biagiotti S, Prayle AP, Group ATBW, Hensiek AE, Horvath R
J Neurol 2025 Jan 15;272(2):110. doi: 10.1007/s00415-024-12766-7. PMID: 39812834Free PMC Article
Petley E, Yule A, Alexander S, Ojha S, Whitehouse WP
PLoS One 2022;17(3):e0264177. Epub 2022 Mar 15 doi: 10.1371/journal.pone.0264177. PMID: 35290391Free PMC Article
Fakhri S, Moradi SZ, Ash-Rafzadeh A, Bishayee A
Pharmacol Res 2022 Mar;177:105961. Epub 2021 Oct 28 doi: 10.1016/j.phrs.2021.105961. PMID: 34718135
Moeini Shad T, Yazdani R, Amirifar P, Delavari S, Heidarzadeh Arani M, Mahdaviani SA, Sadeghi-Shabestari M, Aghamohammadi A, Rezaei N, Abolhassani H
Front Immunol 2021;12:779502. Epub 2022 Jan 14 doi: 10.3389/fimmu.2021.779502. PMID: 35095854Free PMC Article
van Os NJ, Roeleveld N, Weemaes CM, Jongmans MC, Janssens GO, Taylor AM, Hoogerbrugge N, Willemsen MA
Clin Genet 2016 Aug;90(2):105-17. Epub 2016 Jan 20 doi: 10.1111/cge.12710. PMID: 26662178

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