Clinical Utility of Subcutaneous Factor VIII Replacement Therapies in Hemophilia A: A Review of the Evidence

Dargaud, Yesim; Janbain, Maissaa

doi:10.2147/jbm.s260923

Cited by 6 publications

(15 citation statements)

References 27 publications

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“…Obstetric management for women with vWD, hemophilia carriers and those with inherited platelet disorders requires counselling (preferably before conception), choice, informed consent and multidisciplinary care providers ( Table 3 , 2 , 59 Table 6 , 2 , 40 , 59 , 72 – 81 Table 7 , 2 , 40 , 59 , 72 – 81 Table 8 42 , 82 – 90 ). There are substantial hemostatic changes in a normal pregnancy whose role is to preserve placental function and prevent excessive bleeding at delivery.…”

Section: Managementmentioning

confidence: 99%

“…Directed obstetrical delivery management for fetal vWD type 1–3 and hemophilia A and B risk (using a stratification process for estimated obstetric bleeding risk levels of mild, moderate and high [ Table 3 , Tables 6 – 8 ]) suggests that, in pregnant people with associated mild or unlikely risk, a recommendation for avoidance of ventouse use and external cephalic version, and selected use of rotational forceps, fetal scalp blood sampling and fetal scalp electrodes should be considered. 2 , 40 , 59 , 72 – 81 In those with associated moderate risk, care providers should consider a recommendation for avoidance of use of midcavity or rotational forceps, ventouse use procedure and external cephalic version, and selected use of fetal scalp blood sampling and fetal scalp electrodes. In those with associated high risk, a recommendation for avoidance of use of all forceps, external cephalic version, ventouse procedure, fetal scalp blood sampling and fetal scalp electrodes should be considered.…”

Section: Managementmentioning

confidence: 99%

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Preoperative diagnosis and management of inherited bleeding disorders in female adolescents and adults

Wilson

2023

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Although inherited bleeding disorders (IBDs) affect both females and males, this review of the preoperative diagnosis and management of IBDs focuses on genetic and gynecologic screening, diagnosis and management of affected and carrier females. A PubMed literature search was conducted, and the peer-reviewed literature on IBDs was evaluated and summarized. Best-practice considerations for screening, diagnosis and management of IBDs in female adolescents and adults, with GRADE (Grading of Recommendations, Assessment, Development, and Evaluations) evidence level and ranking of recommendation strength, are presented. Health care providers need to increase their recognition of and support for female adolescents and adults with IBDs. Improved access to counselling, screening, testing and hemostatic management is also required. Patients should be educated and encouraged to report abnormal bleeding symptoms to their health care provider when they have a concern. It is hoped that this review of preoperative IBD diagnosis and management will enhance access to women-centred care to increase patients’ understanding of IBDs and decrease their risk of IBD-related morbidity and mortality.

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Section: Managementmentioning

confidence: 99%

Section: Managementmentioning

confidence: 99%

Preoperative diagnosis and management of inherited bleeding disorders in female adolescents and adults

Wilson

2023

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“…The treatment of patients with hemophilia A has evolved over time, from fresh frozen plasma transfusion, and cryoprecipitate to FVIII concentrates (in the 1970s), purified FVIII (ref. 5,6 ), recombinant FVIII (in the 1990s) and prolonged half-life recombinant FVIII products 5,7 . Among replacement therapies, those bioengineered using XTEN fusion technology seem to be the most promising, according to the results from phase 1/2 studies 8 (the polypeptide XTEN can be produced using E. coli, is less immunogenic, and is potentially biodegradable) (ref.…”

Section: Introductionmentioning

confidence: 99%

“…Emicizumab mimics the function of activated FVIII (ref. 6 ), is administered subcutaneously, as prophylactic treatment of patients with severe hemophilia A, and has at least similar efficacy to FVIII replacement therapy 7 . Other investigational pipeline therapies suppress specific natural anticoagulant pathways (ie, anti-TFPI antibodies or siRNA antithrombin) (ref.…”

Section: Introductionmentioning

confidence: 99%

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From a bispecific monoclonal antibody to gene therapy: A new era in the treatment of hemophilia A

Mihăilă¹

2023

Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub

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The treatment of hemophilia A has progressed amazingly in recent years. Emicizumab, a bispecific-humanized monoclonal antibody, is able to improve coagulation by bridging activated factor IX and factor X. Emicizumab is administered subcutaneously and much less often compared to factor VIII products. It has low immunogenicity, does not require dose adjustment, and can be administered regardless of the presence of factor VIII inhibitors. Thrombin generation assays but not factor VIII activity are indicated to guide and monitor the treatment. Emicizumab has enabled the conversion of patients with severe forms into patients with milder forms of hemophilia A. It has reduced the number of bleeding episodes compared to both on-demand and prophylactic substitution therapy and has an excellent safety profile. Gene therapy can elevate factor VIII plasma levels for many years after a single treatment course, could offer longterm protection from bleeding episodes, and minimize or eliminate the need for substitutive treatment with factor VIII concentrates. Gene therapy can provoke an immune response, manifested by an increase in common liver enzymes, that require immunotherapy. Long term monitoring is necessary to identify possible adverse effects. Future objectives are: the development of an ideal viral vector, the possibility of its re-administration, the use of gene therapy in hemophiliac children, and determining whether it can be successfully used to induce immune tolerance to factor VIII ceteri paribus. The future will determine the place of each type of treatment and group of patients for which it is indicated.

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CHIEF: A retrospective self‐control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis

Mashiach,

Mead,

Carneiro

et al. 2024

Pediatric Blood & Cancer

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BackgroundHemophilia A (HA) is an X‐linked bleeding disorder diagnosed by a deficiency in factor VIII (FVIII). For severe HA (SHA), prophylaxis clotting factor concentrates (CFC) has become the standard of care; however, it imparts a high treatment burden and typically results in an annualized bleeding rate (ABR) of 2–6. Emicizumab, a subcutaneously administered FVIII substitute, has become the de facto standard‐of‐care prophylaxis for children with SHA in many countries. Previous clinical trials of emicizumab have assessed ABR in patients greater than 12 years without inhibitors, and in children less than 12 years with inhibitors; however, there is little information published regarding the ABR of emicizumab compared to CFC in non‐inhibitor SHA children.MethodsUsing a retrospective electronic medical record chart review, we conducted a self‐control analysis of 15 patients less than 12 years of age during equivalent periods of CFC versus emicizumab prophylaxis.ResultsThe mean ABR on CFC and emicizumab was 1.79 and 1.13 (p = .092), respectively, with a substantially decreased rate of joint bleeds (CFC 0.94; emicizumab 0.33; p = .001) and spontaneous bleeds (CFC 0.79; emicizumab 0.23; p = .008). No safety events were recorded for patients while administering emicizumab. The mean annual cost of CFC prophylaxis was $515,340 (SD $199,540), compared to $328,410 (SD $137,230) for emicizumab prophylaxis (p < .001).ConclusionEmicizumab resulted in an improved ABR compared to CFC, especially for joint and spontaneous bleeds, had fewer administration complications, and was substantially less expensive compared to CFC prophylaxis; however, more research is necessary for a complete understanding of the effect of emicizumab on joint health and muscle bleeds.

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Clinical Utility of Subcutaneous Factor VIII Replacement Therapies in Hemophilia A: A Review of the Evidence

Cited by 6 publications

References 27 publications

Preoperative diagnosis and management of inherited bleeding disorders in female adolescents and adults

Preoperative diagnosis and management of inherited bleeding disorders in female adolescents and adults

From a bispecific monoclonal antibody to gene therapy: A new era in the treatment of hemophilia A

CHIEF: A retrospective self‐control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis

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