Emicizumab: a novel drug in hemophilia A prophylaxis – a narrative review

Mazurkiewicz, Łukasz; Czernikiewicz, Krystian; Rupa‐Matysek, Joanna; Gil, Lidia

doi:10.1080/17474086.2022.2131526

Cited by 3 publications

(2 citation statements)

References 67 publications

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“…Both bypassing drugs have shown a decrease in the frequency of bleeding and amelioration of QoL . In patients with hemophilia A, prophylaxis with subcutaneous emicizumab has also proved its efficacy in many publications [5][6][7][8].…”

Section: Rs In Individuals With Inhibitorsmentioning

confidence: 99%

“…In patients with hemophilia A (deficit of factor VIII), emicizumab prophylaxis has led to greater treatment satisfaction compared with FVIII prophylaxis, reflecting in part the low treatment burden of emicizumab associated with its infrequent, subcutaneous administration. Emicizumab can also be used in patients with inhibitors [5][6][7][8].…”

Section: Introductionmentioning

confidence: 99%

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Radiosynovectomy for the Treatment of Chronic Hemophilic Synovitis: An Old Technique, but Still Very Effective

Rodríguez-Merchán

Corte‐Rodríguez

Álvarez‐Román

et al. 2022

JCM

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A radiosynovectomy (RS) should be indicated when recurrent articular bleeds related to chronic hemophilia synovitis (CHS) exist, established by clinical examination, and confirmed by imaging techniques that cannot be constrained with hematological prophylaxis. RS can be performed at any point in life, mainly in adolescents (>13–14 years) and adults. Intraarticular injection (IAI) of a radioactive material in children might be arduous since we need child collaboration which might include general anesthesia. RS is our initial option for management of CHS. For the knee joint we prescribe Yttrium-90, while for the elbow and ankle we prescribe Rhenium-186 (1 to 3 IAIs every 6 months). The procedure is greatly cost efficient when compared to surgical synovectomy. Chemical synovectomy with rifampicin has been reported to be efficacious, inexpensive, simple, and especially practical in developing countries where radioactive materials are not easily available. Rifampicin seems to be more efficacious when it is utilized in small joints (elbows and ankles), than when utilized in bigger ones (knees). When RS and/or chemical synovectomy fail, arthroscopic synovectomy (or open synovectomy in some cases) should be indicated. For us, surgery must be performed after the failure of 3 RSs with 6-month interims. RS is an effective and minimally invasive intervention for treatment of repeated articular bleeds due to CHS. Although it has been published that the risk of does not increase, and that the amount of radioactive material used in RS is insignificant, the issue of chromosomal and/or deoxyribonucleic acid (DNA) changes remains a concern and continued surveillance is critical. As child and adulthood prophylaxis becomes more global, RS might become obsolete in the long-term.

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Section: Rs In Individuals With Inhibitorsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Radiosynovectomy for the Treatment of Chronic Hemophilic Synovitis: An Old Technique, but Still Very Effective

Rodríguez-Merchán

Corte‐Rodríguez

Álvarez‐Román

et al. 2022

JCM

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Knowledge and attitudes toward gene therapy of a cohort of Italian patients with hemophilia

Cutica,

Mortarino,

Garagiola

et al. 2024

Journal of Thrombosis and Haemostasis

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A bispecific inhibitor of complement and coagulation blocks activation in complementopathy models via a novel mechanism

Andersen¹,

Lei

Strayer

et al. 2023

Blood

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Inhibitors of complement and coagulation are present in the saliva of a variety of blood feeding arthropods that transmit parasitic and viral pathogens. Here we describe the structure and mechanism of action of the sand fly salivary protein lufaxin, which inhibits formation of the central alternative C3 convertase (C3bBb) and inhibits coagulation factor Xa (fXa). Surface plasmon resonance experiments show that lufaxin stabilizes the binding of the serine protease factor B (FB) to C3b but does not detectably bind either C3b or FB alone. The crystal structure of the inhibitor reveals a novel all β-sheet fold containing two domains. A structure of the lufaxin-C3bB complex obtained by cryo-electron microscopy shows that lufaxin binds via its N-terminal domain at an interface containing elements of both C3b and FB. By occupying this spot, the inhibitor locks FB into a closed conformation where proteolytic activation of FB by FD cannot occur. C3bB-bound lufaxin binds fXa at a separate site in its C-terminal domain. In the cryo-EM structure of a C3bB-lufaxin-fXa complex the inhibitor binds both targets simultaneously and lufaxin inhibits fXa through substrate-like binding of a C-terminal peptide at the active site as well as other interactions in this region. Lufaxin inhibits complement activation in ex vivo models of atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH) as well as thrombin generation in plasma, providing rationale for the development of a bispecific inhibitor to treat complement-related diseases in which thrombosis is a prominent manifestation.

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Emicizumab: a novel drug in hemophilia A prophylaxis – a narrative review

Cited by 3 publications

References 67 publications

Radiosynovectomy for the Treatment of Chronic Hemophilic Synovitis: An Old Technique, but Still Very Effective

Radiosynovectomy for the Treatment of Chronic Hemophilic Synovitis: An Old Technique, but Still Very Effective

Knowledge and attitudes toward gene therapy of a cohort of Italian patients with hemophilia

A bispecific inhibitor of complement and coagulation blocks activation in complementopathy models via a novel mechanism

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