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Thrombocytopenia 3(THC3)

MedGen UID:
437174
Concept ID:
C2678311
Disease or Syndrome
Synonyms: THC3; THROMBOCYTOPENIA, AUTOSOMAL RECESSIVE, 3
 
Gene (location): FYB1 (5p13.1)
 
Monarch Initiative: MONDO:0010120
OMIM®: 273900

Definition

Thrombocytopenia-3 (THC3) is an autosomal recessive hematologic disorder characterized by onset of small-platelet thrombocytopenia in infancy. Patients may show variable bleeding tendency, manifest as petechiae, epistaxis, or heavy menstrual bleeding (summary by Levin et al., 2015). For a general phenotypic description and a discussion of genetic heterogeneity of thrombocytopenia, see 313900. [from OMIM]

Clinical features

From HPO
Epistaxis
MedGen UID:
4996
Concept ID:
C0014591
Pathologic Function
Epistaxis, or nosebleed, refers to a hemorrhage localized in the nose.
Gingival bleeding
MedGen UID:
42218
Concept ID:
C0017565
Pathologic Function
Hemorrhage affecting the gingiva.
Menorrhagia
MedGen UID:
44358
Concept ID:
C0025323
Pathologic Function
Prolonged and excessive menses at regular intervals in excess of 80 mL or lasting longer than 7 days.
Thrombocytopenia
MedGen UID:
52737
Concept ID:
C0040034
Disease or Syndrome
A reduction in the number of circulating thrombocytes.
Prolonged bleeding time
MedGen UID:
56231
Concept ID:
C0151529
Finding
Prolongation of the time taken for a standardized skin cut of fixed depth and length to stop bleeding.
Decreased mean platelet volume
MedGen UID:
853132
Concept ID:
C1096368
Finding
Average platelet volume below the lower limit of the normal reference interval.
Abnormal bleeding
MedGen UID:
264316
Concept ID:
C1458140
Pathologic Function
An abnormal susceptibility to bleeding, often referred to as a bleeding diathesis. A bleeding diathesis may be related to vascular, platelet and coagulation defects.
Megakaryocytopenia
MedGen UID:
346881
Concept ID:
C1858312
Finding
A reduced count of megakaryocytes.
Abnormal megakaryocyte morphology
MedGen UID:
868626
Concept ID:
C4023026
Finding
Any structural anomaly of megakaryocytes. Mature blood platelets are released from the cytoplasm of megakaryocytes, which are bone-marrow resident cells.
Splenomegaly
MedGen UID:
52469
Concept ID:
C0038002
Finding
Abnormal increased size of the spleen.
Petechiae
MedGen UID:
10680
Concept ID:
C0031256
Disease or Syndrome
Petechiae are pinpoint-sized reddish/purple spots, resembling a rash, that appear just under the skin or a mucous membrane when capillaries have ruptured and some superficial bleeding into the skin has happened. This term refers to an abnormally increased susceptibility to developing petechiae.
Purpura
MedGen UID:
19584
Concept ID:
C0034150
Disease or Syndrome
Purpura (from Latin
Spontaneous hematomas
MedGen UID:
306397
Concept ID:
C1697453
Disease or Syndrome
Spontaneous development of hematomas (hematoma) or bruises without significant trauma.

Professional guidelines

PubMed

Epstein RS, Nelms J, Moran D, Girman C, Huang H, Chioda M
Cancer Treat Res Commun 2022;31:100555. Epub 2022 Apr 4 doi: 10.1016/j.ctarc.2022.100555. PMID: 35421820
Obstet Gynecol 2019 Mar;133(3):589-591. doi: 10.1097/AOG.0000000000003101. PMID: 30801464
Radin M, Ugolini-Lopes MR, Sciascia S, Andrade D
Semin Arthritis Rheum 2018 Aug;48(1):117-120. Epub 2018 Jan 5 doi: 10.1016/j.semarthrit.2017.12.006. PMID: 29395258

Recent clinical studies

Etiology

Bayly-McCredie E, Treisman M, Fiorenza S
Int J Mol Sci 2024 Sep 9;25(17) doi: 10.3390/ijms25179736. PMID: 39273684Free PMC Article
Seifert R, Telli T, Lapa C, Desaulniers M, Hekimsoy T, Weber WA, Pfob C, Hadaschik B, Bögemann M, Schäfers M, Herrmann K, Rahbar K, Eiber M, Fendler WP
J Nucl Med 2024 Jun 3;65(6):909-916. doi: 10.2967/jnumed.123.267321. PMID: 38697669
Shuster M, Konopka CI, Verlinden NJ
ASAIO J 2022 Sep 1;68(9):1135-1140. Epub 2022 Feb 1 doi: 10.1097/MAT.0000000000001625. PMID: 36066355
Natal RA, Carneiro HC, de Carvalho Barra FF, Pagnano KBB, Soares FA, Vassallo J
Hum Pathol 2022 Aug;126:55-62. Epub 2022 May 10 doi: 10.1016/j.humpath.2022.05.007. PMID: 35550832
del-Rio-Vellosillo M, Garcia-Medina JJ
Acta Anaesthesiol Scand 2016 Feb;60(2):144-57. Epub 2015 Oct 8 doi: 10.1111/aas.12639. PMID: 26446688

Diagnosis

Bosio A, Cerretti G, Padovan M, Caccese M, Denaro L, Chioffi F, Della Puppa A, Aldegheri V, Guarneri V, Zagonel V, Lombardi G
Clin Oncol (R Coll Radiol) 2023 May;35(5):e319-e327. Epub 2023 Feb 2 doi: 10.1016/j.clon.2023.01.012. PMID: 36858930
Natal RA, Carneiro HC, de Carvalho Barra FF, Pagnano KBB, Soares FA, Vassallo J
Hum Pathol 2022 Aug;126:55-62. Epub 2022 May 10 doi: 10.1016/j.humpath.2022.05.007. PMID: 35550832
Li A, Yin D, Huang X, Zhang L, Lv T, Yi Q, Xiang P
Cardiology 2021;146(2):253-257. Epub 2020 Dec 30 doi: 10.1159/000512512. PMID: 33378749
Marsico C, Aban I, Kuo H, James SH, Sanchez PJ, Ahmed A, Arav-Boger R, Michaels MG, Ashouri N, Englund JA, Estrada B, Jacobs RF, Romero JR, Sood SK, Whitworth S, Jester PM, Whitley RJ, Kimberlin DW; Collaborative Antiviral Study Group (CASG)
J Infect Dis 2019 Apr 16;219(9):1398-1406. doi: 10.1093/infdis/jiy695. PMID: 30535363Free PMC Article
del-Rio-Vellosillo M, Garcia-Medina JJ
Acta Anaesthesiol Scand 2016 Feb;60(2):144-57. Epub 2015 Oct 8 doi: 10.1111/aas.12639. PMID: 26446688

Therapy

Bayly-McCredie E, Treisman M, Fiorenza S
Int J Mol Sci 2024 Sep 9;25(17) doi: 10.3390/ijms25179736. PMID: 39273684Free PMC Article
Seifert R, Telli T, Lapa C, Desaulniers M, Hekimsoy T, Weber WA, Pfob C, Hadaschik B, Bögemann M, Schäfers M, Herrmann K, Rahbar K, Eiber M, Fendler WP
J Nucl Med 2024 Jun 3;65(6):909-916. doi: 10.2967/jnumed.123.267321. PMID: 38697669
Shuster M, Konopka CI, Verlinden NJ
ASAIO J 2022 Sep 1;68(9):1135-1140. Epub 2022 Feb 1 doi: 10.1097/MAT.0000000000001625. PMID: 36066355
Li A, Yin D, Huang X, Zhang L, Lv T, Yi Q, Xiang P
Cardiology 2021;146(2):253-257. Epub 2020 Dec 30 doi: 10.1159/000512512. PMID: 33378749
del-Rio-Vellosillo M, Garcia-Medina JJ
Acta Anaesthesiol Scand 2016 Feb;60(2):144-57. Epub 2015 Oct 8 doi: 10.1111/aas.12639. PMID: 26446688

Prognosis

Wang L, Luo Y, Li X, Li Y, Xia Y, He T, Huang Y, Xu Y, Yang Z, Ling J, Weng R, Zhu X, Qi Z, Yang J
Mycopathologia 2022 Dec;187(5-6):455-467. Epub 2022 Sep 30 doi: 10.1007/s11046-022-00659-0. PMID: 36180657Free PMC Article
Shuster M, Konopka CI, Verlinden NJ
ASAIO J 2022 Sep 1;68(9):1135-1140. Epub 2022 Feb 1 doi: 10.1097/MAT.0000000000001625. PMID: 36066355
Natal RA, Carneiro HC, de Carvalho Barra FF, Pagnano KBB, Soares FA, Vassallo J
Hum Pathol 2022 Aug;126:55-62. Epub 2022 May 10 doi: 10.1016/j.humpath.2022.05.007. PMID: 35550832
Li A, Yin D, Huang X, Zhang L, Lv T, Yi Q, Xiang P
Cardiology 2021;146(2):253-257. Epub 2020 Dec 30 doi: 10.1159/000512512. PMID: 33378749
Marsico C, Aban I, Kuo H, James SH, Sanchez PJ, Ahmed A, Arav-Boger R, Michaels MG, Ashouri N, Englund JA, Estrada B, Jacobs RF, Romero JR, Sood SK, Whitworth S, Jester PM, Whitley RJ, Kimberlin DW; Collaborative Antiviral Study Group (CASG)
J Infect Dis 2019 Apr 16;219(9):1398-1406. doi: 10.1093/infdis/jiy695. PMID: 30535363Free PMC Article

Clinical prediction guides

Bayly-McCredie E, Treisman M, Fiorenza S
Int J Mol Sci 2024 Sep 9;25(17) doi: 10.3390/ijms25179736. PMID: 39273684Free PMC Article
Seifert R, Telli T, Lapa C, Desaulniers M, Hekimsoy T, Weber WA, Pfob C, Hadaschik B, Bögemann M, Schäfers M, Herrmann K, Rahbar K, Eiber M, Fendler WP
J Nucl Med 2024 Jun 3;65(6):909-916. doi: 10.2967/jnumed.123.267321. PMID: 38697669
Shuster M, Konopka CI, Verlinden NJ
ASAIO J 2022 Sep 1;68(9):1135-1140. Epub 2022 Feb 1 doi: 10.1097/MAT.0000000000001625. PMID: 36066355
Ramachandran RM, Srinivasan R
J Trop Pediatr 2022 Jan 7;68(1) doi: 10.1093/tropej/fmac004. PMID: 35084035
Steinberg O, Gilad G, Dgany O, Krasnov T, Zoldan M, Laor R, Kapelushnik J, Gabriel H, Churi C, Stein J, Yaniv I, Tamary H
J Pediatr Hematol Oncol 2007 Dec;29(12):822-5. doi: 10.1097/MPH.0b013e318158152e. PMID: 18090929

Recent systematic reviews

Bayly-McCredie E, Treisman M, Fiorenza S
Int J Mol Sci 2024 Sep 9;25(17) doi: 10.3390/ijms25179736. PMID: 39273684Free PMC Article

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