Search Results (4)

Background and Clinical Significance: Acquired hemophilia A (AHA) and acquired von Willebrand syndrome (AVWS) are rare bleeding disorders that do not often present concurrently. Here, we report a coexisting AHA and AVWS case due to underlying autoantibodies to factor VIII (FVIII) and von Willebrand factor (VWF). Case Presentation: A patient with gastrointestinal bleeding and prolonged aPTT was diagnosed with AHA and AVWS. The patient was started on immunosuppression with prednisone, cyclophosphamide, and intravenous immunoglobulin, alongside recombinant porcine FVIII replacement, susoctocog alfa. AVWS reduced the half-life of susoctocog alfa, requiring more frequent dosing and laboratory monitoring until AVWS resolved. The patient had two further relapses; the most recent was treated with Rituximab, following which remission has been maintained. Conclusions: Given the potential therapeutic implications, VWF testing should be considered as part of the diagnostic workup for AHA. Full article

Background: Acquired hemophilia A (AHA) is a rare bleeding disease due to autoantibodies directed against clotting factor VIII (FVIII). Treatment of AHA consists of inhibitor eradication with immunosuppressive therapy (IST) and prompt control of bleeding obtained with bypassing agents or recombinant porcine FVIII (rpFVIII). The latter has recently been licensed for management of acute bleeding in AHA. Unlike treatment with bypassing agents, rpFVIII can be monitored to provide a successful hemostatic effect and avoid overtreatment. Correlation between rpFVIII inhibitor titers and efficacy of rpFVIII treatment remains a matter of debate. Methods: We report three cases of AHA in which rpFVIII was successfully used with an unconventional schedule despite the presence of medium–high titers of the rpFVIII. The modified Nijmegen–Bethesda inhibitor assay (NBA) was used to dose porcine FVIII inhibitors. Result: The presence of rpFVIII inhibitors prior to the exposition to susoctocog-alfa, that may suggest a cross-reactivity with human FVIII inhibitors, did not affect hemostasis. Conclusion: In our experience, rpFVIII demonstrates safety and efficacy in the presence of rpFVIII inhibitors and using an unconventional schedule in both the perioperative and outpatient settings. Laboratory measurement of inhibitors against rpFVIII during treatment is described for the first time. Full article

Acquired hemophilia A (AHA) is a bleeding disorder due to the presence of neutralizing autoantibodies named inhibitors in patients with a previously normal hemostasis. Recent international recommendations suggest the use of bypassing agents or substitutive therapy as the first-line treatment, usually preferring the former. The adequate hemostatic therapy needs an accurate balance between bleeding and thrombotic risks. We report a clinical case of acquired hemophilia A successfully treated with recombinant porcine factor VIII (Susoctocog alfa) as the first-line treatment. Despite the patient having a high-risk thrombotic score and a history of recent myocardial infarction, our experience showed the absence of thrombotic complications related to the use of Susoctocog alfa and a complete restoration of hemostatic parameters. Limited literature is present on the use of recombinant porcine factor VIII as a first-line treatment, and our report supports its use, especially when the thrombotic risk is high. Full article

Recombinant porcine factor VIII (rpFVIII) is indicated for treating bleeding episodes in acquired haemophilia A, but there are few data regarding laboratory methods to adequately monitor treatment. This study involving three Italian laboratories aimed to evaluate the analytical performance of different assays for measuring rpFVIII. Five spiked rpFVIII samples (0.5–1.5 IU/mL) were analysed on three days, in triplicate, with eleven combinations of reagents (Werfen, Boston, MA, USA: SynthasIL and SynthaFax for one-stage assay, Chromogenix Coamatic FVIII for chromogenic assay), FVIII depleted plasmas (with or without von Willebrand factor—VWF) and calibrators (HemosIL human calibrator plasma, porcine calibrator diluted in FVIII deficient plasma with or without VWF). The assays were performed on ACL TOP analysers (Werfen, Boston, MA, USA). Intra- and inter-assay and inter-laboratory Coefficient of Variation (CV%) were calculated together with percentage of recovery (% recovery) on the expected value. The results showed that the reagent combinations reaching satisfactory analytical performance are: SynthasIL/human calibrator/deficient plasma+VWF (total recovery 99.4%, inter-laboratory CV 4.04%), SynthasIL/porcine calibrator/deficient plasma+VWF (total recovery 111%, inter-laboratory CV 2.75%) and Chromogenic/ porcine calibrator/deficient plasma+VWF (total recovery 96.6%, inter-laboratory CV 8.32%). This study highlights that the use of porcine standard (when available) and FVIII deficient plasma with VWF should be recommended. Full article