Emicizumab reduces re‐hospitalization for bleeding in acquired haemophilia A

Chen, Sheh-Li; Ellsworth, Patrick; Kasthuri, Raj S.; Moll, Stephan; Alice, D.; Key, Nigel S.

doi:10.1111/hae.14335

Cited by 7 publications

(28 citation statements)

References 8 publications

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“…After screening citations on PubMed, EMBASE and Scopus by titles and abstracts, 60 full‐text and conference abstracts were screened for full‐text review for inclusion. Seventeen studies (11 full text, six conference abstracts) were included in the final review, for a total of 41 patients (33 AHA, eight type 3 VWD) 15–31 . The flowchart that describes our study selection is shown in Figure 1.…”

Section: Resultsmentioning

confidence: 99%

“…Of the 33 patients, 26 (78.8%) received haemostatic therapy prior to emicizumab initiation 15–19,22–26 . Haemostatic therapy was not required in five (15.2%) patients 23,26 and not reported in two (6%) patients 20,21 .…”

Section: Resultsmentioning

confidence: 99%

“…Seventeen patients were initiated on the standard loading dose regimen of 3 mg/kg weekly for 4 weeks, 15,17,19,20,22,24,26 whereas 14 patients received a modified loading dose regimen 18,21,23 (Table 1). In two patients, the loading dose regimen was not specified 16,25 .…”

Section: Resultsmentioning

confidence: 99%

“…Haemostatic therapy was not required in five (15.2%) patients 23,26 and not reported in two (6%) patients 20,21 . rFVIIa was the most commonly used haemostatic agent (20/26, 76.9%), 16–19,23–26 followed by aPCC (7/26, 26.9%) 15,18,22,23,25 and rp‐FVIII (7/26, 26.9%) 18,22–24 . Immunosuppressive therapy was used in 31 patients.…”

Section: Resultsmentioning

confidence: 99%

“…Emicizumab is administered subcutaneously at least once a week, which allows for home administration as compared to bypassing agents and rpFVIII, both of which typically require hospitalisation for frequent intravenous infusions. As such, the use of emicizumab in AHA has been shown to lead to reduced days of hospitalisations and potentially overall health care costs 23,24 . Despite the advantages of emicizumab as a first‐line treatment for AHA, a prospective clinical trial comparing emicizumab versus standard haemostatic agents is needed to answer the aforementioned question.…”

Section: Discussionmentioning

confidence: 99%

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Off‐label use of emicizumab in persons with acquired haemophilia A and von Willebrand disease: A scoping review of the literature

2021

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Introduction Since the approval of emicizumab, a bispecific, factor VIII‐mimetic antibody, for use in persons with congenital haemophilia A in 2018, there have been increasing case reports and case series of off‐label use of emicizumab in other bleeding disorders, including acquired haemophilia A (AHA) and von Willebrand disease (VWD). Aim We conducted a scoping review on the use of emicizumab in AHA and VWD, focusing on the clinical presentation and outcomes. Methods We conducted a comprehensive search in PubMed, EMBASE and Scopus up to July 15, 2021. The following criteria were applied to the studies identified in the initial search: patients had a diagnosis of AHA or VWD; and the study reported on the clinical outcome of emicizumab use. Results Seventeen studies were included in the final review for a total of 41 patients (33 AHA, eight type 3 VWD). The majority of AHA patients and all type 3 VWD patients were started on emicizumab for active/recurrent bleeds. The dosing regimen of emicizumab used varied significantly in AHA patients. All patients had a clinical response to emicizumab use. One AHA patient developed a stroke on emicizumab use in association with concomitant recombinant FVIIa use for surgery. Data on adverse events from emicizumab use were not specifically reported in 24.4% of patients (four AHA, six type 3 VWD). Conclusion Based on published case reports and case series, emicizumab appears to be an effective haemostatic therapy for AHA and VWD. Larger confirmatory clinical trials are needed to confirm these findings.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Off‐label use of emicizumab in persons with acquired haemophilia A and von Willebrand disease: A scoping review of the literature

2021

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Recombinant porcine factor VIII in acquired hemophilia A: Experience from two patients and literature review

Hayden

Candelario

Moyer

2022

Research and Practice in Thrombosis and Haemostasis

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Background Acquired hemophilia A (AHA) is a disease caused by antibody formation inhibiting the function of factor VIII, causing bleeding. Recombinant porcine factor VIII (rpFVIII) escapes human FVIII antibody recognition and can provide life‐saving hemostasis. However, the development of antibodies against pFVIII can limit its use. We report two cases in which loss of response to rpFVIII occurred, likely because of inhibiting antibodies. In case 1, the patient achieved hemostasis but lost response to rpFVIII within a few days. In the second case, rpFVIII controlled bleeding but the patient experienced diminishing half‐life of rpFVIII infusions over time, necessitating a switch to emicizumab which provided lasting hemostasis. Key Clinical Question Based on our experience with these cases, we reviewed the available literature regarding the use of rpFVIII in AHA. The Key Clinical Question was to determine how often inhibitors were associated with rpFVIII treatment failure. Clinical approach and conclusions We identified 43 AHA patients across five studies who were treated with rpFVIII. Twenty‐two patients (51%) developed pFVIII inhibitors and seven cases (16%) reported loss of efficacy associated with an inhibitor. In conclusion, rpFVIII can be a life‐saving therapy in AHA. However, clinicians should be aware that pFVIII antibody development can reduce the efficacy and duration of response. Recombinant pFVIII’s limitations support the utility of further investigation of alternative therapies such as emicizumab in early AHA management.

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Acquired Haemophilia A in four north European countries: survey of 181 patients

et al. 2022

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Summary Acquired haemophilia A (AHA) is a rare bleeding disorder caused by acquired antibodies against coagulation factor VIII. In the Nordic countries, treatment and outcomes have not been studied in recent times. To collect retrospective data on patients diagnosed with AHA in the Nordic countries between 2006 and 2018 and compare demographic data and clinical outcomes with previously published reports, data were collected by six haemophilia centres: three Swedish, one Finnish, one Danish and one Estonian. The study included 181 patients. Median age at diagnosis was 76 (range 5–99) years, with even gender distribution. Type and severity of bleeding was comparable to that in the large European Acquired Haemophilia Registry study (EACH2). Bleedings were primarily treated with activated prothrombin complex concentrate (aPCC) with a high success rate (91%). For immunosuppressive therapy, corticosteroid monotherapy was used most frequently and this may be the cause of the overall lower clinical remission rate compared to the EACH2 study (57% vs. 72%). Survey data on 181 patients collected from four north European countries showed similar demographic and clinical features as in previous studies on AHA. aPCC was used more frequently than in the EACH2 study and the overall remission rate was lower.

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Emicizumab reduces re‐hospitalization for bleeding in acquired haemophilia A

Cited by 7 publications

References 8 publications

Off‐label use of emicizumab in persons with acquired haemophilia A and von Willebrand disease: A scoping review of the literature

Off‐label use of emicizumab in persons with acquired haemophilia A and von Willebrand disease: A scoping review of the literature

Recombinant porcine factor VIII in acquired hemophilia A: Experience from two patients and literature review

Acquired Haemophilia A in four north European countries: survey of 181 patients

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