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GATA2 deficiency with susceptibility to MDS/AML

MedGen UID:
981493
Concept ID:
CN300066
Disease or Syndrome
Synonym: GATA2 deficiency
 
Monarch Initiative: MONDO:0042982

Definition

A disorder arising from deficiency in the GATA2 with a wide spectrum of phenotypes. Autosomal dominant mutations of GATA2 cause a haploinsufficiency, which, in consequence, cause individuals to develop hematological, immunological, lymphatic, or other presentations. These often progress to severe organ (e.g. lung) failure, opportunistic infections, myelodysplastic syndrome, and/or acute myeloid leukemia. The most common clinical denominator is the propensity for myeloid neoplasia (myelodysplastic syndrome [MDS], myeloproliferative neoplasms [MPN], chronic myelomonocytic leukemia [CMML], acute myeloid leukemia [AML]). [from MONDO]

Recent clinical studies

Etiology

Marvin-Peek J, Mason EF, Kishtagari A, Jayani RV, Dholaria B, Kim TK, Engelhardt BG, Chen H, Strickland S, Savani B, Ferrell B, Kassim A, Savona M, Mohan S, Byrne M
Transplant Cell Ther 2023 Jun;29(6):390.e1-390.e10. Epub 2023 Mar 9 doi: 10.1016/j.jtct.2023.03.008. PMID: 36906277
Kotmayer L, Romero-Moya D, Marin-Bejar O, Kozyra E, Català A, Bigas A, Wlodarski MW, Bödör C, Giorgetti A
Br J Haematol 2022 Nov;199(4):482-495. Epub 2022 Jun 26 doi: 10.1111/bjh.18330. PMID: 35753998Free PMC Article
Moon WY, Powis SJ
Front Immunol 2019;10:1703. Epub 2019 Jul 19 doi: 10.3389/fimmu.2019.01703. PMID: 31379882Free PMC Article
Simonis A, Fux M, Nair G, Mueller NJ, Haralambieva E, Pabst T, Pachlopnik Schmid J, Schmidt A, Schanz U, Manz MG, Müller AMS
Ann Hematol 2018 Oct;97(10):1961-1973. Epub 2018 Jun 13 doi: 10.1007/s00277-018-3388-4. PMID: 29947977
Spinner MA, Sanchez LA, Hsu AP, Shaw PA, Zerbe CS, Calvo KR, Arthur DC, Gu W, Gould CM, Brewer CC, Cowen EW, Freeman AF, Olivier KN, Uzel G, Zelazny AM, Daub JR, Spalding CD, Claypool RJ, Giri NK, Alter BP, Mace EM, Orange JS, Cuellar-Rodriguez J, Hickstein DD, Holland SM
Blood 2014 Feb 6;123(6):809-21. Epub 2013 Nov 13 doi: 10.1182/blood-2013-07-515528. PMID: 24227816Free PMC Article

Diagnosis

Marvin-Peek J, Mason EF, Kishtagari A, Jayani RV, Dholaria B, Kim TK, Engelhardt BG, Chen H, Strickland S, Savani B, Ferrell B, Kassim A, Savona M, Mohan S, Byrne M
Transplant Cell Ther 2023 Jun;29(6):390.e1-390.e10. Epub 2023 Mar 9 doi: 10.1016/j.jtct.2023.03.008. PMID: 36906277
McReynolds LJ, Yang Y, Yuen Wong H, Tang J, Zhang Y, Mulé MP, Daub J, Palmer C, Foruraghi L, Liu Q, Zhu J, Wang W, West RR, Yohe ME, Hsu AP, Hickstein DD, Townsley DM, Holland SM, Calvo KR, Hourigan CS
Leuk Res 2019 Jan;76:70-75. Epub 2018 Dec 4 doi: 10.1016/j.leukres.2018.11.013. PMID: 30578959Free PMC Article
Spinner MA, Sanchez LA, Hsu AP, Shaw PA, Zerbe CS, Calvo KR, Arthur DC, Gu W, Gould CM, Brewer CC, Cowen EW, Freeman AF, Olivier KN, Uzel G, Zelazny AM, Daub JR, Spalding CD, Claypool RJ, Giri NK, Alter BP, Mace EM, Orange JS, Cuellar-Rodriguez J, Hickstein DD, Holland SM
Blood 2014 Feb 6;123(6):809-21. Epub 2013 Nov 13 doi: 10.1182/blood-2013-07-515528. PMID: 24227816Free PMC Article

Prognosis

Marvin-Peek J, Mason EF, Kishtagari A, Jayani RV, Dholaria B, Kim TK, Engelhardt BG, Chen H, Strickland S, Savani B, Ferrell B, Kassim A, Savona M, Mohan S, Byrne M
Transplant Cell Ther 2023 Jun;29(6):390.e1-390.e10. Epub 2023 Mar 9 doi: 10.1016/j.jtct.2023.03.008. PMID: 36906277
Kotmayer L, Romero-Moya D, Marin-Bejar O, Kozyra E, Català A, Bigas A, Wlodarski MW, Bödör C, Giorgetti A
Br J Haematol 2022 Nov;199(4):482-495. Epub 2022 Jun 26 doi: 10.1111/bjh.18330. PMID: 35753998Free PMC Article
Spinner MA, Sanchez LA, Hsu AP, Shaw PA, Zerbe CS, Calvo KR, Arthur DC, Gu W, Gould CM, Brewer CC, Cowen EW, Freeman AF, Olivier KN, Uzel G, Zelazny AM, Daub JR, Spalding CD, Claypool RJ, Giri NK, Alter BP, Mace EM, Orange JS, Cuellar-Rodriguez J, Hickstein DD, Holland SM
Blood 2014 Feb 6;123(6):809-21. Epub 2013 Nov 13 doi: 10.1182/blood-2013-07-515528. PMID: 24227816Free PMC Article

Clinical prediction guides

Marvin-Peek J, Mason EF, Kishtagari A, Jayani RV, Dholaria B, Kim TK, Engelhardt BG, Chen H, Strickland S, Savani B, Ferrell B, Kassim A, Savona M, Mohan S, Byrne M
Transplant Cell Ther 2023 Jun;29(6):390.e1-390.e10. Epub 2023 Mar 9 doi: 10.1016/j.jtct.2023.03.008. PMID: 36906277
Kotmayer L, Romero-Moya D, Marin-Bejar O, Kozyra E, Català A, Bigas A, Wlodarski MW, Bödör C, Giorgetti A
Br J Haematol 2022 Nov;199(4):482-495. Epub 2022 Jun 26 doi: 10.1111/bjh.18330. PMID: 35753998Free PMC Article
Moon WY, Powis SJ
Front Immunol 2019;10:1703. Epub 2019 Jul 19 doi: 10.3389/fimmu.2019.01703. PMID: 31379882Free PMC Article

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