Acquired hemophilia A (AHA) is caused by autoantibodies neutralizing coagulation factor VIII (FVIII). In the presence of inhibitors against FVIII, acute bleeds can be managed with bypassing agents, including recombinant factor VIIa (eptacog alfa activated, NovoSeven) and activated prothrombin complex concentrate (FEIBA), as well as recombinant porcine FVIII (susoctocog alfa, Obizur). Studies comparing these agents directly are not available, and indirect evidence suggests an overall similar efficacy. Selecting an agent in clinical practice therefore depends on (1) availability of agent, (2) safety profile, (3) monitoring requirements, (4) cost, and (5) personal experience. This review examines available data and collects additional considerations to support decision making for bleeding emergencies in AHA.
BACKGROUND: Acquired haemophilia A (AHA) is a rare acquired bleeding disorder that can present with life threatening bleeding. OBJECTIVE: To describe recent Australian use of recombinant porcine FVIII (rpFVIII) replacement therapy as a haemostatic agent in patients with acquired haemophilia. PATIENTS/METHOD: Four patients with acquired haemophilia treated in three different institutions around Australia in the last 12 (twelve) months were included in the study. Haemostatic efficacy of Obizur was...
Last. Andreas Tiede(MHH: Hannover Medical School)H-Index: 30
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BACKGROUND: Recombinant porcine factor VIII (rpFVIII, OBI-1, susoctocog alfa) is used for the treatment of acute bleeds in patients with acquired hemophilia A (AHA). Inhibitors in AHA can sometimes cross-react with rpFVIII. OBJECTIVES: To assess the frequency, strength, and determinants of cross-reactivity. PATIENTS/METHODS: Baseline samples from 70 patients of the prospective, observational cohort study GTH-AH 01/2010 were assessed for anti-human FVIII and anti-rpFVIII inhibitors using modified...
Last. Craig M. Kessler(GUMC: Georgetown University Medical Center)H-Index: 64
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BACKGROUND: Acquired haemophilia (AH) is a rare bleeding disorder with significant morbidity and mortality. Most patients initially present to physicians without experience of the disease, delaying diagnosis and potentially worsening outcomes. Existing guidance in AH is limited to clinical opinion of few experts and does not address monitoring bleeds in specific anatomical locations. AIM: Derive consensus from a large sample of experts around the world in monitoring bleeding patients with AH. ME...
#1Sonha Nguyen(UCSD: University of California, San Diego)H-Index: 3
#2P. Teh(UCR: University of California, Riverside)H-Index: 1
Last. A. von Drygalski(UCSD: University of California, San Diego)H-Index: 10
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Acquired hemophilia A (AHA) is a rare autoimmune disorder caused by autoantibodies against Factor VIII (FVIII). It has a high mortality due to bleeding complications. FVIIa-based bypassing agents are the first line of treatment but not always effective. Recombinant porcine (rp) FVIII (Obizur®) was recently approved for rescue treatment but with little evidence-based information regarding efficacy. We report a case of papillary thyroid cancer associated with AHA malignancy that responded to a sin...
#1Patrick Möhnle(LMU: Ludwig Maximilian University of Munich)H-Index: 16
#2Isabell Pekrul(LMU: Ludwig Maximilian University of Munich)H-Index: 5
Last. Claudia Dechant(LMU: Ludwig Maximilian University of Munich)H-Index: 9
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The prognosis of acquired haemophilia A (AHA) is severe and treatment options are limited. Emicizumab is a novel bispecific humanized monoclonal antibody in the treatment of inherited AHA with inhibitors. An 83-year-old AHA patient with congestive heart failure and a high risk for thromboembolic and cardiac events who had initially been treated successfully with steroids and substitution of recombinant B-domain-deleted porcine FVIII developed severe bleeding complications and a secondary increas...
To conduct a systematic review of the literature reporting efficacy and safety of recombinant factor VIIa (rFVIIa) for the treatment of bleeding in acquired haemophilia and, if data permitted, undertake a meta-analysis of the current evidence. MEDLINE®, Embase®, and the Cochrane Central Register of Controlled Trials (CENTRAL) databases were searched for all studies on rFVIIa treatment in acquired haemophilia. Heterogeneity of included studies was measured using the inconsistency index (I2). Of t...
#1Emma Fosbury(Royal Free London NHS Foundation Trust)H-Index: 3
#2Anja B Drebes(Royal Free London NHS Foundation Trust)H-Index: 3
Last. Pratima Chowdary(Royal Free London NHS Foundation Trust)H-Index: 26
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Acquired haemophilia A (AHA) is a rare, serious bleeding disorder most often encountered in elderly patients. The mainstay of haemostatic management is with bypassing agents (BPAs) including recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrates (aPCCs). Their major limitation is incomplete efficacy, potential risk for thrombosis and the lack of routine laboratory assays for monitoring treatment response. Plasma-derived porcine FVIII (pd-pFVIII, Hyate C®), first...
Acquired hemophilia A (AHA) is a rare disease resulting from autoantibodies (inhibitors) against endogenous factor VIII (FVIII) that leads to bleeding, which is often spontaneous and severe. AHA tends to occur in elderly patients with comorbidities and is associated with high mortality risk from underlying comorbidities, bleeding, or treatment complications. Treatment, which consists of hemostatic management and eradication of the inhibitors, can be challenging to manage. Few data are available ...
Introduction A recombinant porcine factor VIII B-domain-deleted product (rpFVIII; OBIZUR, Baxalta Incorporated, Deerfield, IL 60015, USA) was recently approved for treatment of bleeding episodes in adults with acquired haemophilia A (AHA) in the United States. To date, no clinical experience outside the registration study has been reported. Aim To describe early clinical experience using rpFVIII for AHA. Methods A retrospective chart review of seven patients with AHA treated with rpFVIII at four...
Introduction: Acquired hemophilia A (AHA) is a rare autoimmune disease caused by autoantibodies that bind and inactivate factor VIII (FVIII), predisposing to a potentially life-threatening bleeding...
Last. Paul Knöbl(Medical University of Vienna)H-Index: 52
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Emicizumab is currently approved to prevent bleeding in patients with congenital hemophilia A with or without neutralizing antibodies (inhibitors) against factor VIII (FVIII). Here, we present a case-based discussion of its potential use in acquired hemophilia A (AHA), a severe bleeding disorder caused by autoantibodies against FVIII. State-of-the-art management is based on bypassing agents (recombinant factor VIIa, activated prothrombin complex concentrate) and recombinant porcine FVIII; immuno...
