SERPINC1

gene
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Also known as ATIIIMGC22579

Summary

SERPINC1 (serpin family C member 1, HGNC:775) is a protein-coding gene on chromosome 1q25.1, encoding Antithrombin-III (P01008). Most important serine protease inhibitor in plasma that regulates the blood coagulation cascade.

The protein encoded by this gene, antithrombin III, is a plasma protease inhibitor and a member of the serpin superfamily. This protein inhibits thrombin as well as other activated serine proteases of the coagulation system, and it regulates the blood coagulation cascade. The protein includes two functional domains: the heparin binding-domain at the N-terminus of the mature protein, and the reactive site domain at the C-terminus. The inhibitory activity is enhanced by the presence of heparin. Numerous mutations have been identified for this gene, many of which are known to cause antithrombin-III deficiency which constitutes a strong risk factor for thrombosis. A reduction in the serum level of this protein is associated with severe cases of Coronavirus Disease 19 (COVID-19).

Source: NCBI Gene 462 — RefSeq curated summary.

At a glance

  • Gene–disease (curated): hereditary antithrombin deficiency (Definitive, ClinGen)
  • GWAS associations: 2
  • Clinical variants (ClinVar): 480 total — 95 pathogenic, 67 likely-pathogenic
  • Phenotypes (HPO): 20
  • Druggable target: yes — 1 molecules with ChEMBL bioactivity
  • MANE Select transcript: NM_000488

Identifiers

Gene identifiers

FieldValue
HGNC IDHGNC:775
Approved symbolSERPINC1
Nameserpin family C member 1
Location1q25.1
Locus typegene with protein product
StatusApproved
AliasesATIII, MGC22579
Ensembl geneENSG00000117601
Ensembl biotypeprotein_coding
OMIM107300
Entrez462

Gene structure

Transcript identifiers

Ensembl transcripts: 14 — 12 protein_coding, 2 protein_coding_CDS_not_defined

ENST00000367698, ENST00000487183, ENST00000494024, ENST00000874322, ENST00000874323, ENST00000874324, ENST00000874325, ENST00000874326, ENST00000874327, ENST00000874328, ENST00000874329, ENST00000874330, ENST00000874331, ENST00000874332

RefSeq mRNA: 7 — MANE Select: NM_000488 NM_000488, NM_001365052, NM_001386302, NM_001386303, NM_001386304, NM_001386305, NM_001386306

CCDS: CCDS1313

Canonical transcript exons

ENST00000367698 — 7 exons

ExonStartEnd
ENSE00000789908173909552173909942
ENSE00000789909173910754173910891
ENSE00001383315173907450173907514
ENSE00001876994173903800173904065
ENSE00001944151173917219173917327
ENSE00003578986173914553173914919
ENSE00003644352173911799173912014

Expression profiles

Bgee: expression breadth ubiquitous, 153 present calls, max score 99.73.

FANTOM5 (CAGE): breadth tissue_specific, TPM avg 11.3159 / max 5017.9498, expressed in 63 samples.

FANTOM5 promoters (9 alternative TSS)

Promoter IDTPM avgSamples expressed
159716.041047
159704.254146
159740.356911
159670.32189
159720.195411
159750.04739
159690.04679
159730.04378
159680.00904

Top tissues by expression

291 total, by Bgee expression score (0-100, higher = more expressed):

TissueAnatomy IDExpression scoreQuality
right lobe of liverUBERON:000111499.73gold quality
liverUBERON:000210799.72gold quality
adrenal tissueUBERON:001830362.15gold quality
colonic epitheliumUBERON:000039761.46gold quality
primordial germ cell in gonadCL:0000670 ∩ UBERON:000099161.22gold quality
lower esophagus mucosaUBERON:003583461.20gold quality
stromal cell of endometriumCL:000225561.04gold quality
adult mammalian kidneyUBERON:000008260.56gold quality
oocyteCL:000002358.33gold quality
islet of LangerhansUBERON:000000657.44gold quality
kidneyUBERON:000211357.22gold quality
renal glomerulusUBERON:000007456.22gold quality
nephron tubuleUBERON:000123155.71gold quality
metanephric glomerulusUBERON:000473655.26gold quality
kidney epitheliumUBERON:000481954.58gold quality
gastrocnemiusUBERON:000138854.55gold quality
mucosa of stomachUBERON:000119954.29gold quality
metanephrosUBERON:000008153.86silver quality
bloodUBERON:000017853.16gold quality
paraflocculusUBERON:000535152.96gold quality
cortex of kidneyUBERON:000122552.38gold quality
muscle of legUBERON:000138351.98gold quality
right coronary arteryUBERON:000162551.82gold quality
descending thoracic aortaUBERON:000234551.62gold quality
quadriceps femorisUBERON:000137751.55gold quality
muscle organUBERON:000163051.19gold quality
pancreasUBERON:000126451.08gold quality
granulocyteCL:000009451.07silver quality
lower esophagusUBERON:001347350.95gold quality
lower esophagus muscularis layerUBERON:003583350.91gold quality

Single-cell (SCXA)

Detected in 4 experiment(s), a significant marker in 3.

ExperimentMarker?Max mean expression
E-GEOD-130473yes2859.31
E-HCAD-9yes54.72
E-MTAB-10553yes31.71
E-ANND-3no0.00

Regulation

Is transcription factor: no

Upstream regulators (CollecTRI, top): ATF4, CEBPA, FOXA1, FOXA2, HNF4A, NR2F1, RXRA, SP1, THRA, THRB

Literature-anchored findings (GeneRIF, showing 40)

  • We report 5 children from 3 families with homozygous antithrombin deficiency type II affecting the heparin binding site (99 Leu to Phe mutation). (PMID:11686316)
  • The single base substitution of G to A at nucleotide 2709 was found in a proband with a family history of venous thrombosis. This results in a substitution of 82 Ser by Asn, creating a new glycosylation site causing type I antithrombin deficiency. (PMID:11686319)
  • The karyotype showed 46XY, del(1)(q23q31.2), the antithrombin III (AT3, 1q23-q25.1) serum level and activity were half of normal. (PMID:11754060)
  • A specific pentasaccharide sequence (H5) in high affinity heparin induces a conformational change in antithrombin that is particularly important for factor Xa (fXa) inhibition (factor Xa) (PMID:11854268)
  • Antithrombin prevents endotoxin-induced hypotension by inhibiting the induction of nitric oxide synthase in rat lung. (PMID:11861278)
  • Molecular biological basis and diagnosis of hereditary defects (review) (PMID:12193972)
  • a new natural antithrombin mutation that confirms the critical contribution of lysine 114 to the binding of the core heparin pentasaccharide, with the replacement of lysine 114 by glutamate causing a complete loss in affinity (PMID:12353073)
  • Arg is required in position 129 for high-affinity pentasaccharide binding of heparin to antithrombin, whereas Lys125 and Lys114 do not participate in intramolecular interactions of this type and can be acceptably replaced with Arg. (PMID:12369826)
  • Data show that anti-thrombin is widely expressed in prostate cancer but is gradually lost in tumors of high Gleason grade. (PMID:12466122)
  • hydrophobic residues Phe-122 and Phe-121 play a critical role in heparin binding and activation of antithrombin (PMID:12556442)
  • A new antithrombin mutation, Phe229Leu, spontaneously polymerized in the proband’s circulation, causing cerebral venous thrombosis. Molecular modeling suggested how the mutation might destabilize the protein and favor polymerization. (PMID:12595305)
  • study of the possibility that all pentasaccharide AT-binding sequences in antithrombin-heparin complex may catalyze factor Xa inhibition (PMID:12695507)
  • Five novel and two previously reported point mutations in the antithrombin gene causing venous thrombosis were identified; the novel L409P mutation is a type 2 pleiotropic effects (PE) deficiency and it is highly conserved among other serpins. (PMID:12894857)
  • findings suggest that antithrombin exosites responsible for enhancing the rates of factor Xa and factor IXa inhibition in the conformationally activated inhibitor lie in strand 3 of beta-sheet C of the serpin (PMID:14532267)
  • endotoxin-induced adhesion of leukocytes to endothelium can be reversed by ligation of syndecan-4 with antithrombin’s heparin-binding site (PMID:14652650)
  • identified two missense mutations affecting the shutter region of ATIII: P80S and G424R; the mutant ATIII is an inactive disulfide-linked dimer whose properties are consistent with head-to-head insertion of the reactive loop (PMID:15140129)
  • Renal hypertension was caused by renal artery obstruction due to thrombus formation. This thrombus formation was attributed to antithrombin deficiency caused by a novel SERPINCI gene mutation (AT III Akita, M352R). (PMID:15338392)
  • hAT3 might increase hepatic tissue levels of PGI2 via enhancement of hepatic I/R-induced activation of capsaicin-sensitive sensory neurons, thereby reducing liver injury in rats (PMID:15630490)
  • clinical and biological features of heterozygotes who carry the N187D or N187K mutations; also investigated AT thermostability as well as the presence of latent or polymerized AT in plasma (PMID:15630491)
  • The two mutated antithrombins were the same as that of wild type, but the specific activity of the E381del mutant decreased significantly compared with that of wild type. (PMID:15733967)
  • This review assembles the available experimental and clinical data on biological mechanisms of antithrombin in inflammatory coagulation activation. (PMID:16542481)
  • 22 novel SERPINC1 mutations are associated with antithrombin deficiency. (PMID:16705712)
  • These results suggest that heparin-binding proteins influence the conformational equilibrium of iduronic acid residues that are directly or indirectly involved in binding. (PMID:16796563)
  • tyrosine 131 in the native conformation of antithrombin III activates it for factor Xa inhibition (PMID:16940049)
  • This study reports that the poor inhibitory activity of the circulating of AT is partially conferred by intramolecular contacts that restrain the RCL, orient the P1 residue away from attacking proteases, and block the exosite used in protease recognition. (PMID:16973611)
  • Antithrombin A384S mutation is a prevalent genetic risk factor for venous thrombosis and is the most frequent cause of antithrombin deficiency in white populations. (PMID:17244682)
  • pulmonary endothelium contributes to the antithrombin III metabolism with a 0.023 breakdown rate; The data support the hypothesis of a main endothelial catabolism of antithrombin III (PMID:17413759)
  • findings suggested that FS-3Stop and Met-32Thr mutations are responsible for type I antithrombin deficiency, whereas Ser116Pro and Ala59Val mutations contribute to type II antithrombin deficiency (PMID:17492649)
  • REVIEW OF AT3 BINDING INTERACTIONS, HEREDITARY AT3 DEFICIENCY, LOW LEVEL IN DIC (PMID:17597998)
  • Report novel antithrombin heparin-binding site variant associated with low thrombotic risk. (PMID:17849067)
  • High antithrombin III in cerebrospinal fluid is associated with CNS lymphoma (PMID:18056677)
  • A previously unreported heterozygous large deletion spanning exons 1-6 of the SERPINC1 gene was responsible for hereditary antithrombin deficiency in a Korean family. (PMID:18208532)
  • Identification in human milk of complexes of matriptase with ATIII, A1AT, or A2AP, provides evidence that the proteolytic activity of matriptase, from cells that express no or low levels of HAI-1, may be controlled by secreted serpins. (PMID:18550704)
  • crystallographic structure of a stable serpin dimer which reveals a domain swap of more than 50 residues, including two long antiparallel beta-strands inserting in the centre of the principal beta-sheet of the neighbouring monomer (PMID:18923394)
  • Prevalence of genetic mutations in SERPINC1 in Japanese patients with venous thrombosis is reported. (PMID:18954896)
  • functional effect of the rs2227589 polymorphism not explained by its linkage with the promoter polymorphism that supports the moderate thrombotic risk associated with the A allele. (PMID:19229049)
  • The study shows that Antithrombin Cambridge II (A384S) significantly increases the risk of myocardial infarction . (PMID:19277409)
  • AT-III directly acts on platelets and suppresses ADP-induced platelet granule secretion due to inhibiting HSP27 phosphorylation via p44/p42 MAPK and p38 MAPK. (PMID:19631608)
  • These results indicate that P2-Gly plays a key role in determining the S2 sub-site specificity and target protease selectivity of AT in circulation. (PMID:19715676)
  • Data show that large deletions explain almost half of otherwise unexplained type I AT-inherited deficiency cases. (PMID:19760264)

Cross-species orthologs

3 orthologs

OrganismSymbolGene ID
danio_rerioserpinc1ENSDARG00000042684
mus_musculusSerpinc1ENSMUSG00000026715
rattus_norvegicusSerpinc1ENSRNOG00000002783

Paralogs (36): SERPINB1 (ENSG00000021355), SERPINB3 (ENSG00000057149), SERPIND1 (ENSG00000099937), SERPINA4 (ENSG00000100665), SERPINE1 (ENSG00000106366), SERPINI2 (ENSG00000114204), SERPINA7 (ENSG00000123561), SERPINB6 (ENSG00000124570), SERPINF1 (ENSG00000132386), AGT (ENSG00000135744), SERPINE2 (ENSG00000135919), SERPINA10 (ENSG00000140093), SERPING1 (ENSG00000149131), SERPINH1 (ENSG00000149257), SERPINI1 (ENSG00000163536), SERPINA12 (ENSG00000165953), SERPINB7 (ENSG00000166396), SERPINB8 (ENSG00000166401), SERPINB12 (ENSG00000166634), SERPINF2 (ENSG00000167711), SERPINA9 (ENSG00000170054), SERPINA6 (ENSG00000170099), SERPINB9 (ENSG00000170542), SERPINA11 (ENSG00000186910), SERPINA5 (ENSG00000188488), SERPINA3 (ENSG00000196136), SERPINA1 (ENSG00000197249), SERPINB2 (ENSG00000197632), SERPINB13 (ENSG00000197641), SERPINB11 (ENSG00000206072), SERPINB4 (ENSG00000206073), SERPINB5 (ENSG00000206075), HMSD (ENSG00000221887), SERPINB10 (ENSG00000242550), SERPINE3 (ENSG00000253309), SERPINA2 (ENSG00000258597)

Protein

Protein identifiers

Antithrombin-IIIP01008 (reviewed: P01008)

Alternative names: Serpin C1

All UniProt accessions (2): A0A2S0BDD1, P01008

UniProt curated annotations — full annotation on UniProt →

Function. Most important serine protease inhibitor in plasma that regulates the blood coagulation cascade. AT-III inhibits thrombin, matriptase-3/TMPRSS7, as well as factors IXa, Xa and XIa. Its inhibitory activity is greatly enhanced in the presence of heparin.

Subunit / interactions. Forms protease inhibiting heterodimer with TMPRSS7.

Subcellular location. Secreted. Extracellular space.

Tissue specificity. Found in plasma.

Post-translational modifications. Phosphorylated by FAM20C in the extracellular medium.

Disease relevance. Antithrombin III deficiency (AT3D) [MIM:613118] An important risk factor for hereditary thrombophilia, a hemostatic disorder characterized by a tendency to recurrent thrombosis. Antithrombin-III deficiency is classified into 4 types. Type I: characterized by a 50% decrease in antigenic and functional levels. Type II: has defects affecting the thrombin-binding domain. Type III: alteration of the heparin-binding domain. Plasma AT-III antigen levels are normal in type II and III. Type IV: consists of miscellaneous group of unclassifiable mutations. The disease is caused by variants affecting the gene represented in this entry.

Similarity. Belongs to the serpin family.

RefSeq proteins (7): NP_000479, NP_001351981, NP_001373231, NP_001373232, NP_001373233, NP_001373234, NP_001373235 (=MANE)

Domains & families (InterPro)

IDNameType
IPR000215Serpin_famFamily
IPR023795Serpin_CSConserved_site
IPR023796Serpin_domDomain
IPR033829Antithrombin_3_serpin_domainDomain
IPR036186Serpin_sfHomologous_superfamily
IPR042178Serpin_sf_1Homologous_superfamily
IPR042185Serpin_sf_2Homologous_superfamily

Pfam: PF00079

UniProt features (174 total): sequence variant 102, strand 26, helix 15, sequence conflict 8, turn 6, glycosylation site 4, disulfide bond 3, binding site 3, mutagenesis site 2, modified residue 2, signal peptide 1, chain 1, site 1

Structure

Experimental structures (PDB)

27 structures.

PDBMethodResolution (Å)
3KCGX-RAY DIFFRACTION1.7
2B5TX-RAY DIFFRACTION2.1
1TB6X-RAY DIFFRACTION2.5
1E04X-RAY DIFFRACTION2.6
1JVQX-RAY DIFFRACTION2.6
1NQ9X-RAY DIFFRACTION2.6
2ANTX-RAY DIFFRACTION2.6
1E05X-RAY DIFFRACTION2.62
1OYHX-RAY DIFFRACTION2.62
1R1LX-RAY DIFFRACTION2.7
2BEHX-RAY DIFFRACTION2.7
1T1FX-RAY DIFFRACTION2.75
1DZGX-RAY DIFFRACTION2.8
1LK6X-RAY DIFFRACTION2.8
2B4XX-RAY DIFFRACTION2.8
2ZNHX-RAY DIFFRACTION2.8
4EB1X-RAY DIFFRACTION2.8
1DZHX-RAY DIFFRACTION2.85
1AZXX-RAY DIFFRACTION2.9
1BR8X-RAY DIFFRACTION2.9
1E03X-RAY DIFFRACTION2.9
2HIJX-RAY DIFFRACTION2.9
1ANTX-RAY DIFFRACTION3
3EVJX-RAY DIFFRACTION3
1SR5X-RAY DIFFRACTION3.1
1ATHX-RAY DIFFRACTION3.2
2GD4X-RAY DIFFRACTION3.3

Predicted structure (AlphaFold)

ModelpLDDTFraction very-high
AF-P01008-F185.340.70

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Catalytic / active sites (1): 425–426 (reactive bond)

Ligand- & substrate-binding residues (3): 81; 161; 177

Post-translational modifications (2): 63, 68

Disulfide bonds (3): 40–160, 53–127, 279–462

Glycosylation sites (4): 187, 224, 128, 167

Mutagenesis-validated functional residues (2):

PositionPhenotype
414reduces interaction with thrombin by 99%.
414reduces interaction with thrombin by 80%.

Function

Pathways and Gene Ontology

Reactome pathways

12 pathways

IDPathway
R-HSA-381426Regulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs)
R-HSA-8957275Post-translational protein phosphorylation
R-HSA-9769733Fibrin formation
R-HSA-9769735Initiation of coagulation cascade
R-HSA-9769739Regulation of clotting cascade
R-HSA-9769743Amplification and propagation of coagulation cascade
R-HSA-140837
R-HSA-140875
R-HSA-109582Hemostasis
R-HSA-140877
R-HSA-392499Metabolism of proteins
R-HSA-597592Post-translational protein modification

MSigDB gene sets: 230 (showing top): GOBP_REGULATION_OF_WOUND_HEALING, GOBP_REGULATION_OF_COAGULATION, GNF2_GSTM1, GNF2_HPN, TGACCTY_ERR1_Q2, SHEPARD_BMYB_MORPHOLINO_DN, COUP_01, GOBP_WOUND_HEALING, VART_KSHV_INFECTION_ANGIOGENIC_MARKERS_UP, HOSHIDA_LIVER_CANCER_SUBCLASS_S3, GOBP_REGULATION_OF_RESPONSE_TO_WOUNDING, GNF2_LCAT, INGRAM_SHH_TARGETS_UP, HSIAO_LIVER_SPECIFIC_GENES, GOBP_REGULATION_OF_RESPONSE_TO_STRESS

GO Biological Process (3): blood coagulation (GO:0007596), regulation of blood coagulation (GO:0030193), hemostasis (GO:0007599)

GO Molecular Function (6): protease binding (GO:0002020), serine-type endopeptidase inhibitor activity (GO:0004867), heparin binding (GO:0008201), identical protein binding (GO:0042802), protein binding (GO:0005515), peptidase inhibitor activity (GO:0030414)

GO Cellular Component (7): extracellular region (GO:0005576), obsolete extracellular space (GO:0005615), endoplasmic reticulum lumen (GO:0005788), plasma membrane (GO:0005886), extracellular matrix (GO:0031012), extracellular exosome (GO:0070062), blood microparticle (GO:0072562)

Reactome top-level categories

Rollup of top-3 pathways:

CategoryPathways
Coagulation pathway4
Metabolism of proteins2
Post-translational protein modification1

GO top-level categories

Rollup of top GO terms by namespace:

CategoryTerms
cellular anatomical structure2
hemostasis1
wound healing1
coagulation1
blood coagulation1
regulation of response to external stimulus1
regulation of coagulation1
regulation of wound healing1
regulation of hemostasis1
regulation of body fluid levels1
enzyme binding1
serine-type endopeptidase activity1
endopeptidase inhibitor activity1
glycosaminoglycan binding1
sulfur compound binding1
protein binding1
binding1
enzyme inhibitor activity1
peptidase activity1
peptidase regulator activity1
endoplasmic reticulum1
intracellular organelle lumen1
membrane1
cell periphery1
external encapsulating structure1
extracellular vesicle1
extracellular region1

Protein interactions and networks

STRING

1718 interactions, top by confidence (×1000):

Protein AProtein BPartner UniProtScore
SERPINC1F2P00734980
SERPINC1PLGP00747960
SERPINC1F3P13726958
SERPINC1TFPIP10646935
SERPINC1VTNP01141924
SERPINC1PLATP00750915
SERPINC1THBDP07204913
SERPINC1A2MP01023912
SERPINC1F7P08709882
SERPINC1F9P00740864
SERPINC1KLK4Q9Y5K2864
SERPINC1ALBP02768863
SERPINC1F8P00451809
SERPINC1PF4V1P10720803
SERPINC1FGAP02671800

IntAct

50 interactions, top by confidence:

ABTypeScore
RNF146TNKSpsi-mi:“MI:0914”(association)0.790
CFTRESYT2psi-mi:“MI:0914”(association)0.710
GPX7GAKpsi-mi:“MI:0914”(association)0.640
CFTRHAX1psi-mi:“MI:0914”(association)0.610
SERPINC1SERPINC1psi-mi:“MI:0407”(direct interaction)0.560
DDX31IGLL5psi-mi:“MI:0914”(association)0.530
FCGRTGOLIM4psi-mi:“MI:0914”(association)0.530
SERPINC1BTDpsi-mi:“MI:0914”(association)0.530
ING4KAT7psi-mi:“MI:0914”(association)0.530
LGALS1PODXLpsi-mi:“MI:0914”(association)0.530
SPSB4ARHGEF10psi-mi:“MI:0914”(association)0.530
C1QBPF2psi-mi:“MI:0914”(association)0.460
SERPINC1FAM20Cpsi-mi:“MI:0217”(phosphorylation reaction)0.440
SERPINC1F9psi-mi:“MI:0407”(direct interaction)0.440
SERPINC1F2psi-mi:“MI:0915”(physical association)0.400
PLCG1SERPINC1psi-mi:“MI:0915”(physical association)0.400
CD5Lpsi-mi:“MI:0915”(physical association)0.400
LECT2psi-mi:“MI:0915”(physical association)0.400
HRSERPINC1psi-mi:“MI:0914”(association)0.350

BioGRID (65): TUBB8 (Affinity Capture-MS), TUBB1 (Affinity Capture-MS), TEX15 (Affinity Capture-MS), OS9 (Affinity Capture-MS), ISCA2 (Affinity Capture-MS), SUCLG1 (Affinity Capture-MS), BTD (Affinity Capture-MS), PFAS (Co-fractionation), TUBB1 (Affinity Capture-MS), TEX15 (Affinity Capture-MS), SERPINC1 (Affinity Capture-MS), BTD (Affinity Capture-MS), SERPINC1 (Affinity Capture-MS), SUCLG1 (Affinity Capture-MS), OS9 (Affinity Capture-MS)

ESM2 similar proteins: A0A090BX51, A0A0K8RCY5, A0A0K8RJ89, A0A0K8RJV9, A0A7H0DNF9, B3RFC3, E2RVI8, O73860, O75635, P01008, P01012, P01014, P05120, P07092, P07093, P07385, P0DSW3, P12388, P13909, P14754, P15059, P19104, P20961, P22777, P22922, P42926, P50449, P68565, P68566, P79335, P80034, Q06B72, Q06B74, Q06B75, Q07235, Q27085, Q27086, Q5MGH0, Q5R5A3, Q6J201

Diamond homologs: A0A090BX51, A0A0K8RCY5, A0A0K8RJ89, A0A0K8RJV9, A5PJK0, A9RA96, B0CMB0, B1MTB7, B1MTC3, B2KI30, B3RFC3, B4USX2, E2RVI8, O02739, O08800, O35684, O54757, O54758, O54759, O54760, O73790, O73860, O75635, O75830, P01008, P01011, P01012, P01014, P05120, P05619, P12388, P17475, P19104, P22323, P22325, P22922, P23035, P29508, P29524, P30740

SIGNOR signaling

6 interactions.

AEffectBMechanism
SERPINC1up-regulatesLRP1binding
SERPINC1“down-regulates activity”F2cleavage
SERPINC1“down-regulates activity”F11cleavage
SERPINC1“down-regulates activity”F10cleavage
SERPINC1“down-regulates activity”F12cleavage
SERPINC1“down-regulates activity”F9cleavage

Disease & clinical

Clinical variants and AI predictions

ClinVar

480 variants total. Per-class counts are floors (≥ shown; pagination cap):

ClassificationCount (floor)
Pathogenic95
Likely pathogenic67
Uncertain significance160
Likely benign107
Benign19

Top pathogenic / likely-pathogenic (30)

Variant IDHGVSClassification
100923NM_000488.4(SERPINC1):c.1016G>A (p.Trp339Ter)Pathogenic
1321887NC_000001.11:g.173896668_173942868delPathogenic
1321892NC_000001.11:g.173914640_173922032delPathogenic
1321893NM_000488.4(SERPINC1):c.84_409-536delPathogenic
1321894NM_000488.4(SERPINC1):c.408+948_763-386delPathogenic
1321895NM_000488.4(SERPINC1):c.42-486_1154-846delPathogenic
1321900NC_000001.11:g.173916704_173935703delPathogenic
1321902NM_000488.4(SERPINC1):c.763-379_1084delPathogenic
1321903NM_000488.4(SERPINC1):c.1154-898_1218+410delPathogenic
1321904NM_000488.4(SERPINC1):c.1154-40_1218+127dupPathogenic
1321905NM_000488.4(SERPINC1):c.1154-324_1218+475dupPathogenic
1321907NM_000488.4(SERPINC1):c.41+297_55delPathogenic
1321909NC_000001.11:g.173905922_173905923ins[NC_000006.11:56893618_56896059]Pathogenic
1321911NC_000001.11:g.173879820_173925989delPathogenic
1330290NM_000488.4(SERPINC1):c.436A>G (p.Lys146Glu)Pathogenic
1335876NC_000001.11:g.173908412_173919816dupPathogenic
1434089NM_000488.4(SERPINC1):c.381T>A (p.Cys127Ter)Pathogenic
1456446NM_000488.4(SERPINC1):c.607C>T (p.Gln203Ter)Pathogenic
18004NM_000488.4(SERPINC1):c.235C>T (p.Arg79Cys)Pathogenic
18005AT-III RomaPathogenic
18006AT-III TrentoPathogenic
18007NM_000488.4(SERPINC1):c.1246G>C (p.Ala416Pro)Pathogenic
18008AT-III FontainebleuPathogenic
18010NM_000488.4(SERPINC1):c.1277C>T (p.Ser426Leu)Pathogenic
18011NM_000488.4(SERPINC1):c.218C>T (p.Pro73Leu)Pathogenic
18012NM_000488.4(SERPINC1):c.89T>A (p.Val30Glu)Pathogenic
18013AT-III BarcelonaPathogenic
18014NM_000488.4(SERPINC1):c.236G>A (p.Arg79His)Pathogenic
18016NM_000488.4(SERPINC1):c.1273C>T (p.Arg425Cys)Pathogenic
18019NM_000488.4(SERPINC1):c.1274G>A (p.Arg425His)Pathogenic

SpliceAI

976 predictions. Top by Δscore:

VariantEffectΔscore
1:173907442:GTACT:Gdonor_loss1.0000
1:173907444:ACT:Adonor_loss1.0000
1:173907445:CTC:Cdonor_loss1.0000
1:173907446:TCAC:Tdonor_loss1.0000
1:173907447:CAC:Cdonor_loss1.0000
1:173907448:ACCTC:Adonor_loss1.0000
1:173907449:C:Tdonor_loss1.0000
1:173907512:TACC:Tacceptor_loss1.0000
1:173907515:C:CGacceptor_loss1.0000
1:173907516:T:Aacceptor_loss1.0000
1:173909540:T:TAdonor_gain1.0000
1:173909938:AGGCC:Aacceptor_gain1.0000
1:173909939:GGCC:Gacceptor_gain1.0000
1:173909940:GCC:Gacceptor_gain1.0000
1:173909941:CC:Cacceptor_gain1.0000
1:173909941:CCC:Cacceptor_gain1.0000
1:173909942:CC:Cacceptor_gain1.0000
1:173909943:C:CCacceptor_gain1.0000
1:173909943:C:Tacceptor_gain1.0000
1:173909943:CTG:Cacceptor_loss1.0000
1:173909944:T:Gacceptor_loss1.0000
1:173910806:T:TAdonor_gain1.0000
1:173911795:TCAC:Tdonor_loss1.0000
1:173911797:ACCT:Adonor_loss1.0000
1:173914554:T:TAdonor_gain1.0000
1:173914916:CTTC:Cacceptor_gain1.0000
1:173914917:TTC:Tacceptor_gain1.0000
1:173914918:TC:Tacceptor_gain1.0000
1:173914919:CC:Cacceptor_gain1.0000
1:173914920:C:CCacceptor_gain1.0000

AlphaMissense

3067 scored. Top likely-pathogenic:

VariantProtein changeam_pathogenicity
1:173914619:A:CS114R0.998
1:173914619:A:TS114R0.998
1:173914621:T:GS114R0.998
1:173909936:A:GW257R0.996
1:173909936:A:TW257R0.996
1:173909934:C:AW257C0.995
1:173909934:C:GW257C0.995
1:173914585:C:GA126P0.995
1:173910769:G:CN249K0.994
1:173910769:G:TN249K0.994
1:173911893:C:GR177P0.994
1:173914560:A:GL134P0.994
1:173914587:C:TG125D0.994
1:173911950:A:GL158P0.993
1:173914584:G:TA126D0.993
1:173907481:A:TV396D0.992
1:173910782:A:GL245P0.992
1:173911841:A:CS194R0.992
1:173911841:A:TS194R0.992
1:173911843:T:GS194R0.992
1:173914588:C:GG125R0.992
1:173909605:A:GL367P0.991
1:173911886:A:CF179L0.991
1:173911886:A:TF179L0.991
1:173911888:A:GF179L0.991
1:173911890:A:GL178P0.991
1:173914603:C:GA120P0.991
1:173914713:A:GL83P0.991
1:173914602:G:TA120D0.989
1:173904044:C:GA414P0.988

dbSNP variants (sampled 300 via entrez): RS1000202171 (1:173910078 T>C,G), RS1000332785 (1:173904290 T>A), RS1000582649 (1:173908014 TAATA>T), RS1000949668 (1:173911362 A>G), RS1001057744 (1:173907678 A>C), RS1001156482 (1:173914357 A>G), RS1001555339 (1:173910275 T>C), RS1001654948 (1:173909315 T>A), RS1001880914 (1:173917165 T>C), RS1001992574 (1:173910573 T>C,G), RS1002022677 (1:173903632 C>A), RS1002105677 (1:173904787 A>G), RS1002424360 (1:173908529 TCTAATTA>T), RS1002517609 (1:173914757 T>G), RS1002618543 (1:173908360 G>A)

Disease associations

OMIM: gene MIM:107300 | disease phenotypes: MIM:613118, MIM:613254, MIM:120430

GenCC curated gene-disease

DiseaseClassificationInheritance
hereditary antithrombin deficiencyDefinitiveAutosomal dominant

ClinGen Gene-Disease Validity (1)

Expert-panel classifications — Definitive > Strong > Moderate > Limited > Disputed > Refuted.

DiseaseClassificationInheritance
hereditary antithrombin deficiencyDefinitiveSD

Mondo (4): hereditary antithrombin deficiency (MONDO:0013144), tuberous sclerosis 2 (MONDO:0013199), thrombocytopenia (MONDO:0002049), coloboma of optic nerve (MONDO:0007354)

Orphanet (4): Hereditary thrombophilia due to congenital antithrombin deficiency (Orphanet:82), Tuberous sclerosis complex (Orphanet:805), Morning glory disc anomaly (Orphanet:35737), Coloboma of optic disc (Orphanet:98947)

HPO phenotypes

20 total (21 of 20 shown, HPO-id order):

HPOTerm
HP:0000006Autosomal dominant inheritance
HP:0000007Autosomal recessive inheritance
HP:0001976Reduced antithrombin III activity
HP:0002204Pulmonary embolism
HP:0002625Deep venous thrombosis
HP:0002638Superficial thrombophlebitis
HP:0004419Recurrent thrombophlebitis
HP:0004420Arterial thrombosis
HP:0004831Recurrent thromboembolism
HP:0005268Miscarriage
HP:0005305Cerebral venous thrombosis
HP:0012636Retinal venous occlusion
HP:0025324Arterial occlusion
HP:0030242Portal vein thrombosis
HP:0030243Hepatic vein thrombosis
HP:0030248Mesenteric venous thrombosis
HP:0031437Pregnancy exposure
HP:0040226Decreased level of heparin co-factor II
HP:0040246Reduced antithrombin antigen
HP:0200067Recurrent spontaneous abortion
HP:0000588Optic disc coloboma

GWAS associations

2 associations (top):

StudyTraitp-value
GCST006944_27Experiencing mood swings4.000000e-08
GCST006979_962Heel bone mineral density2.000000e-12

EFO canonical traits (2, from GWAS)

EFO IDTrait name
EFO:0008475mood instability measurement
EFO:0009270heel bone mineral density

MeSH disease descriptors (4)

DescriptorNameTree numbers
D020152Antithrombin III DeficiencyC15.378.100.100.075; C15.378.147.150; C15.378.925.075; C16.320.099.075
D013921ThrombocytopeniaC15.378.140.855; C15.378.243.937
C535970Coloboma of optic nerve (supp.)
C566021Tuberous Sclerosis 2 (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (2): CHEMBL1950 (SINGLE PROTEIN), CHEMBL2111449 (PROTEIN COMPLEX)

Molecules with ChEMBL bioactivity

1 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 3 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

MoleculeNamePhasePatents
CHEMBL5314374IDRAPARINUX SODIUM33

PharmGKB: 1 entry (VIP=true, CPIC=false)

PharmGKB clinical annotations

1 annotations.

VariantTypeLevelDrugsPhenotypes
rs5877Toxicity3carboplatin;gemcitabineNon-Small Cell Lung Carcinoma;Thrombocytopenia

PharmGKB variants

1 variants.

VariantGenesLevelScore#Clin annotsDrugs
rs5877SERPINC132.501carboplatin;gemcitabine

GtoPdb / IUPHAR curated pharmacology

(IUPHAR/BPS Guide to Pharmacology — expert-curated)

Target class: enzyme — Blood coagulation components

Most potent curated ligand interactions (2 total), top 2:

LigandActionAffinityParameter
heparinActivation7.85pKd
fondaparinuxActivation7.49pKd

Binding affinities (BindingDB)

6 measured of 6 human assays (6 total across all organisms); most potent 6 below. Values come from heterogeneous assays and are not directly comparable.

LigandMeasureValuePatent
N-decyl-N-(3,4-dihydroxyphenyl)sulfonyl-3,4-dihydroxybenzenesulfonamideIC5044400 nMUS-9718760: Plasminogen activator inhibitor-1 inhibitors and methods of use thereof
N-(3,4-dihydroxyphenyl)sulfonyl-3,5-difluoro-4-hydroxy-N-octylbenzenesulfonamideIC5087000 nMUS-9718760: Plasminogen activator inhibitor-1 inhibitors and methods of use thereof
N-(3,4-dihydroxyphenyl)sulfonyl-N-dodecyl-3,4-dihydroxybenzenesulfonamideIC50547000 nMUS-9718760: Plasminogen activator inhibitor-1 inhibitors and methods of use thereof
3,4,5-trihydroxy-N-octylbenzamideIC501.67e+06 nMUS-9718760: Plasminogen activator inhibitor-1 inhibitors and methods of use thereof
N-(3,5-difluoro-4-hydroxyphenyl)sulfonyl-3,5-difluoro-4-hydroxy-N-octylbenzenesulfonamideIC502.33e+06 nMUS-9718760: Plasminogen activator inhibitor-1 inhibitors and methods of use thereof
N-benzyl-3,4-dihydroxybenzenesulfonamideIC502.33e+06 nMUS-9718760: Plasminogen activator inhibitor-1 inhibitors and methods of use thereof

ChEMBL bioactivities

6 potent at pChembl≥5 of 6 total, top 6 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChemblTypeValueUnitMolecule
9.00Kd1nMIDRAPARINUX SODIUM
8.72Kd1.9nMCHEMBL2303815
8.55Kd2.8nMCHEMBL2303814
8.15Kd7nMCHEMBL1909451
7.24Kd58nMCHEMBL609990
6.24IC50570nMCHEMBL51173

PubChem BioAssay actives

4 with measured affinity, of 59 total; 4 most potent distinct compounds. Largely complementary to BindingDB; screening values are coarse (µM, 4 dp), so sub-nM hits tie at the floor.

CompoundAssayTypeValueUnit
nonasodium;(2S,3S,4S,5R,6R)-6-[(2R,3R,4S,5R,6R)-6-[(2R,3S,4S,5R,6R)-2-carboxylato-4,5-dimethoxy-6-[(2R,3R,4S,5R,6S)-6-methoxy-4,5-disulfonatooxy-2-(sulfonatooxymethyl)oxan-3-yl]oxyoxan-3-yl]oxy-4,5-disulfonatooxy-2-(sulfonatooxymethyl)oxan-3-yl]oxy-4,5-dimethoxy-3-[(2R,3R,4S,5R,6R)-3,4,5-trimethoxy-6-(sulfonatooxymethyl)oxan-2-yl]oxyoxane-2-carboxylate527395: Binding affinity to antithrombin 3kd0.0010uM
decasodium;(2R,3S,4S,5R,6R)-6-[(2S,3R,4S,5R,6S)-6-[(2S,3S,4S,5R,6R)-2-carboxylato-6-[(2S,3S,4S,5S,6S)-4,6-dimethoxy-5-(sulfonatoamino)-2-(sulfonatooxymethyl)oxan-3-yl]oxy-4-methoxy-5-sulfonatooxyoxan-3-yl]oxy-4,5-disulfonatooxy-2-(sulfonatooxymethyl)oxan-3-yl]oxy-3-[(2R,3R,4R,5S,6S)-4,5-dimethoxy-3-(sulfonatoamino)-6-(sulfonatooxymethyl)oxan-2-yl]oxy-4,5-dimethoxyoxane-2-carboxylate38738: Dissociation constant for Antithrombin-IIIkd0.0070uM
decasodium;(3S,4R,6R)-3-[(2S,4S,5R)-5-[(2R,5S,6R)-6-carboxylato-5-[(2R,5S,6S)-4,5-dihydroxy-3-(sulfonatoamino)-6-(sulfonatooxymethyl)oxan-2-yl]oxy-3,4-dihydroxyoxan-2-yl]oxy-3,4-disulfonatooxy-6-(sulfonatooxymethyl)oxan-2-yl]oxy-6-[(2S,3S,6S)-4,6-dihydroxy-5-(sulfonatoamino)-2-(sulfonatooxymethyl)oxan-3-yl]oxy-4-hydroxy-5-(sulfonatoamino)oxane-2-carboxylate38738: Dissociation constant for Antithrombin-IIIkd0.0580uM
N-[(2R)-3-(4-carbamimidoylphenyl)-1-oxo-1-piperidin-1-ylpropan-2-yl]-2-(naphthalen-2-ylsulfonylamino)acetamide38735: Antithrombin III (ATIII)-mediated anti-Xa activity was determinedic500.5700uM

CTD chemical–gene interactions

73 total (human), top 30 by PubMed support.

ChemicalActions (top 5)PubMed papers
Ethinyl Estradiolaffects binding, decreases expression, affects cotreatment, decreases activity8
ethinyl estradiol-desogestrel combinationdecreases activity, decreases expression, increases expression7
Heparindecreases expression, affects binding, increases expression4
Cyclosporinedecreases expression, increases expression3
Aflatoxin B1decreases expression, affects expression3
Levonorgestreldecreases expression, affects cotreatment, decreases activity3
sodium arseniteincreases methylation, decreases expression2
Gestodenedecreases activity, decreases expression, affects cotreatment2
Acetaminophendecreases expression2
Benzo(a)pyrenedecreases expression2
Contraceptives, Oraldecreases activity, affects response to substance2
Estrogensdecreases activity, decreases expression2
Hydrogen Peroxideaffects expression, increases expression2
Tobacco Smoke Pollutiondecreases expression, affects expression2
testosterone enanthateincreases expression1
methyleugenoldecreases expression1
bisphenol Aaffects expression1
calcium heparinincreases activity1
potassium perchloratedecreases expression1
tris(1,3-dichloro-2-propyl)phosphateincreases expression1
norgestimateaffects cotreatment, decreases expression1
cobaltous chlorideincreases expression1
nickel chloridedecreases expression1
dienogestaffects cotreatment, decreases activity1
benzo(e)pyreneincreases methylation1
periodate-oxidized adenosineaffects expression1
2,3-dimethoxy-1,4-naphthoquinonedecreases expression1
ethinyl estradiol, levonorgestrel drug combinationdecreases expression1
estradiol, norethindrone drug combinationdecreases expression1
estradiol valerate-dienogestaffects cotreatment, decreases activity1

ChEMBL screening assays

19 unique, capped per target: 19 binding

Representative assays (with source publication via chembl_document):

Assay IDTypeDescriptionSource paper
CHEMBL1072392BindingInhibition of antithrombin-3 assessed as residual alpha-thrombin activityNovel bis-arylsulfonamides and aryl sulfonimides as inactivators of plasminogen activator inhibitor-1 (PAI-1). — Bioorg Med Chem Lett

Cellosaurus cell lines

3 cell lines: 2 induced pluripotent stem cell, 1 transformed cell line

First 10 cell lines (id-ordered, not curated):

CellosaurusNameCategorySex
CVCL_A9YMZZUNEUi014-AInduced pluripotent stem cellFemale
CVCL_C9LXSFPHi001-AInduced pluripotent stem cellFemale
CVCL_IR13CHO-13D-35DTransformed cell lineFemale

Clinical trials (associated diseases)

257 trials via MONDO — disease-level, not drug-specific.

TrialPhaseStatusTitle
NCT02278575PHASE4WITHDRAWNAtenativ Effect on Uterine Blood Flow and Preeclampsia
NCT00039858PHASE4COMPLETEDEvaluation of Argatroban Injection in Pediatric Patients Requiring Anticoagulant Alternatives to Heparin
NCT00239733PHASE4TERMINATEDAnti-D for Treating Thrombocytopenia in Adults Infected With Hepatitis C Virus With or Without HIV Co-Infection
NCT00907478PHASE4COMPLETEDStudy on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)
NCT01727401PHASE4TERMINATEDThromboprophylaxis of Venous Thromboembolism in Acutely-ill Medical Inpatients With Thrombocytopenia
NCT02032134PHASE4TERMINATEDProtocol for the Infusion of Buffy Coat-derived Cryopreserved Platelets in Patients With Severe Thrombocytopenia
NCT02267993PHASE4COMPLETEDEfficacy and Safety of rhTPO for the Treatment of Thrombocytopenia After Chemotherapy in AML Patients
NCT03633019PHASE4UNKNOWNHigh-dose Use of rhTPO in CIT Patients
NCT03688191PHASE4UNKNOWNStudy of Sirolimus in CTD-TP in China
NCT04906083PHASE4UNKNOWNAvatrombopag in Patients With End-stage Liver Disease and Thrombocytopenia
NCT05217719PHASE4UNKNOWNEffects of Recombinant Human Thrombopoietin on Platelet Levels in ICU Patients
NCT05255003PHASE4RECRUITINGSTrategies for Anticoagulation in Patients With thRombocytopenia and Cancer-associated Thrombosis
NCT05382013PHASE4UNKNOWNEfficacy and Safety of Avatrombopag for Treating TCP in HBV-ACLF Patients Receiving ALSS Treatment
NCT05944458PHASE4COMPLETEDEfficacy of Intravenous N-Acetylcysteine in Preventing Linezolid-Induced Thrombocytopenia in Critically Ill Patients
NCT06562738PHASE4RECRUITINGClinical Study on Efficacy and Safety of Hetrombopag in the Preoperative Patients of Thrombocytopenia
NCT00110513PHASE3COMPLETEDRecombinant Human Antithrombin (rhAT) in Patients With Hereditary Antithrombin Deficiency Undergoing Surgery or Delivery
NCT04918173PHASE3RECRUITINGEfficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery
NCT06096116PHASE3RECRUITINGPhase 3 Study on the Efficacy and Safety of Human Plasma Derived Antithrombin (Atenativ) in Heparin-Resistant Patients Scheduled to Undergo Cardiac Surgery Necessitating Cardiopulmonary Bypass
NCT00037791PHASE3COMPLETEDSafety and Efficacy of (PN-152,243)/PN-196,444 in the Prevention of Thrombocytopenia
NCT00039910PHASE3COMPLETEDSafety and Efficacy of (PN-152,243)/PN-196,444 in the Prevention of Thrombocytopenia
NCT00073580PHASE3COMPLETEDAngiomax in Patients With HIT/HITTS Type II Undergoing Off-Pump Coronary Artery Bypass Grafting (CABG) (CHOOSE)
NCT00102323PHASE3COMPLETEDAMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Refractory to Splenectomy
NCT00102336PHASE3COMPLETEDAMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Prior to Splenectomy
NCT00116688PHASE3COMPLETEDOpen Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
NCT00128713PHASE3COMPLETEDOptimal Platelet Dose Strategy for Management of Thrombocytopenia
NCT00151866PHASE3COMPLETEDEfficacy of Transfusions With Platelets Stored in Platelet Additive Solution II Versus Plasma
NCT00261924PHASE3COMPLETEDEfficacy and Safety Study of Platelets Treated for Pathogen Inactivation and Stored for Up to Seven Days
NCT00415532PHASE3COMPLETEDRomiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura
NCT00420914PHASE3TERMINATEDStrategies for Transfusion of Platelets (SToP)
NCT00501345PHASE3TERMINATEDAspirin in Patients With Myocardial Infarction and Thrombocytopenia
NCT00508820PHASE3COMPLETEDAn Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP
NCT00678587PHASE3TERMINATEDEltrombopag To Reduce The Need For Platelet Transfusion In Subjects With Chronic Liver Disease And Thrombocytopenia Undergoing Elective Invasive Procedures
NCT01438840PHASE3COMPLETEDEfficacy and Safety of Oral E5501 Plus Standard of Care for the Treatment of Thrombocytopenia in Adults With Chronic Immune Thrombocytopenia (Amendment 02)
NCT01444417PHASE3COMPLETEDSafety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients
NCT01805648PHASE3UNKNOWNEfficacy and Safety Study of Maintenance Treatment With rhTPO in Thrombocytopenic Subjects With ITP
NCT02244658PHASE3UNKNOWNRecombinant Human Thrombopoietin (rhTPO) in Management of Chemotherapy-induced Thrombocytopenia in Acute Myelocytic Leukemia
NCT02389621PHASE3COMPLETEDSafety and Efficacy Study of Lusutrombopag for Thrombocytopenia in Patients With Chronic Liver Disease Undergoing Elective Invasive Procedures
NCT02444728PHASE3TERMINATEDCyclophosphamide and Hydroxychloroquine for Thrombocytopenia in SLE
NCT02487563PHASE3COMPLETEDProspective Study of Patients With Thrombocytopenia Following HSCT
NCT02578901PHASE3COMPLETEDAmerican Trial Using Tranexamic Acid in Thrombocytopenia