Moderately Severe Hemophilia A

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Retrieved

2021-01-23

Source

Orphanet

Trials

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Genes

F8, F9, TFPI, F10, AK3, F2, VWF, COX8A, ST14, F5, F3, PROC, IL10, CCR5, AAVS1, HLA-DRB1, TNF, MS4A1, CCND1, GP1BA, G6PD, LMAN1, RBM45, KRT20, ABO, CPB2, VIPR1, PROCR, ISG20, IFNA2

F8, F9, TFPI, F10, AK3, F2, VWF, COX8A, ST14, F5, F3, PROC, IL10, CCR5, AAVS1, HLA-DRB1, TNF, MS4A1, CCND1, GP1BA, G6PD, LMAN1, RBM45, KRT20, ABO, CPB2, VIPR1, PROCR, ISG20, IFNA2, IL2, TGFB1, IL2RA, SELP, IL4, IFNL3, MAPK9, PF4, HAL, PROS1, FOXP3, STATH, PRNP, ADAMTS5, WDHD1, MTHFR, F7, AMBP, ABR, DMD, ALB, GH1, ARTN, DKK1, APOH, BPI, BRCA1, ABCB6, CD163, FCGR2C, TNFRSF11A, HAP1, TRBV16, IKBKG, SLC14A2, LAP, BTK, C4BPA, VEGFA, TPT1, C6, THBD, NT5C2, HFE, TRBV7-9, CALR, MIR1246, C20orf181, C4orf3, CA13, ASPG, AGRP, IL33, MCFD2, CARD14, FUNDC2, UPF3B, HPSE2, IMPACT, SCLY, SERPINA10, SOST, APC, ICOS, TRBC1, CA6, TRBV20OR9-2, PRDX2, FUT1, LTF, LRP1, F11, KRT7, KIT, FCGR2A, FCGR2B, CXCL8, IL6, UTS2R, HLA-A, IL1B, IFNG, GPT, ICAM1, HNF4A, HMOX1, GUSB, HLA-DRA, HLA-B, MBL2, MRC1, NFYA, NHS, TRB, TAL1, CD28, CD68, SPRR2A, SLC4A1, SELPLG, CLTC, CCL3L1, REST, PSMA7, CSF1R, CSF2, CTLA4, SERPINE2, DPP4, SERPINA5, SERPINE1, TNFRSF11B, MIR4521

Drugs

Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene, Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII, Anti-inhibitor coagulant complex ( FEIBA ), Antihemophilic factor / Von Willebrand factor complex (human) ( ALPHANATE, HUMATE -P ), Autologous dendritic cells incubated ex vivo with zebularine and factor VIII, Desmopressine acetate ( DESMOPRESSIN ACETATE AVENTIS BEHRING L.L.C., OCTIM, MINIRIN ), Efmoroctocog alfa ( ELOCTA, ELOCTATE ), Factor VIII mimetic bispecific antibody, Fitusiran, Human coagulation factor IX (antihemophilic B factor) ( BETAFACT, ALPHANINE, NONAFACT ), Human coagulation factor VIII (antihemophilic A factor) ( FACTANE ), Human monoclonal IgG1 antibody against tissue factor pathway inhibitor, Human monoclonal IgG2 antibody against tissue factor pathway inhibitor, Humanised monoclonal IgG4 antibody against tissue factor pathway inhibitor, Humanised monoclonal modified IgG4 antibody with bispecific structure targeting factors IX, IXa, X and Xa ( HEMLIBRA ), Long acting recombinantFactor VIIa-CTP3, Moroctocog alpha ( REFACTO AF ), Octocog alfa (liposomal), Octocog alpha ( ADVATE, HELIXATE NEXGEN, KOGENATE BAYER, KOVALTRY, IBLIAS ), Pegylated B-domain-deleted sequence-modified recombinant human factor VIII, Pegylated recombinant factor VIIa, Recombinant adeno-associated viral vector serotype 6 encoding the B-domain-deleted human factor VIII, Recombinant coagulation factor VIIa (rFVIIa) ( NOVOSEVEN, NOVOSEVEN RT ), Recombinant factor VIIa modified with three terminal repeats derived from the beta chain of human chorionic gonadotropin, Recombinant fusion protein linking human coagulation factor VIIa with human albumin (rVIIa-FP), Recombinant porcine factor VIII (B domain deleted) ( OBIZUR ), Sequence-modified human recombinant factor VIIa, Turoctocog alfa pegol ( ESPEROCT ), Vatreptacog alfa (activated), nonacog alfa ( BENEFIX ), recombinant AAV vector with hu37 serotype capsid (AAVhu37) encoding human FVIII, recombinant adeno-associated viral vector containing a bioengineered capsid and a codon-optimised expression cassette to drive the expression of the SQ form of a B-domain deleted human coagulation factor VIII, recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein

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Moderately severe hemophilia A is a form of hemophilia A (see this term) characterized by factor VIII deficiency leading to abnormal bleeding as a result of minor injuries, or following surgery or tooth extraction.

Epidemiology

Moderately severe hemophilia A accounts for around 20% of all cases of hemophilia A.

Clinical description

The biological activity of factor VIII is between 1% and 5%. Spontaneous hemorrhages are rare.

Etiology

The disorder is caused by mutations in the F8 gene (Xq28) encoding coagulation factor VIII.

Genetic counseling

Transmission is X-linked recessive.