Background: Emicizumab, a bispecific factor VIII mimetic antibody, was approved in 2018 for bleeding prophylaxis in congenital hemophilia A with or without inhibitors. Since then, several case reports and case series have described the off-label use of emicizumab in acquired hemophilia A (AHA), and data from two clinical trials were recently published (AGEHA, GTH-AHA-EMI).
Objectives: To describe the reported data on the outcomes of emicizumab, highlighting its benefit/risk profile in treatment.
Methods: We conducted a literature search in PubMed, Scopus, Cochrane, and Google Scholar up to August 2024, including all scientific articles reporting clinical outcomes of emicizumab use in patients with AHA.
Results: Thirty-two studies were included in the final review, covering a total of 171 AHA patients. The majority started emicizumab for active bleeding management and prophylaxis with various regimens. Follow-up duration and remission criteria varied. Two clinical trials supported the use of emicizumab for bleeding prophylaxis with a new dosing regimen and completion criteria. Bleeding was well managed in all cases, with no major recurrent bleeds. Some adverse events were reported : 3 cases of deep venous thrombosis, 2 cases of stroke, and 2 cases of anti-emicizumab drug antibodies developing in patients with thromboembolic risk factors.
Conclusions: Based on published data, emicizumab appears to be effective in bleeding management and prophylaxis in AHA patients, with a favorable benefit/risk profile.
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http://dx.doi.org/10.1177/10760296241298661 | DOI Listing |
Haemophilia
December 2024
Department of Medicine, University of Colorado School of Medicine, Aurora, Colorado, USA.
Introduction: People with haemophilia A (PwHA) experience acute and chronic pain associated with reduced quality of life (QoL).
Aims: This post hoc analysis of pooled data from the HAVEN 1 (NCT02622321), 3 (NCT02847637), 4 (NCT03020160) and STASEY (NCT0319179) studies assessed the impact of emicizumab prophylaxis on pain-related QoL in PwHA.
Methods: PwHA received emicizumab during the four studies.
Ther Adv Hematol
November 2024
Haemostas-Frankfurt, Frankfurt am Main, Germany.
Adv Ther
November 2024
Sanofi, Cambridge, MA, USA.
Introduction: The phase 3 XTEND-1 trial (NCT04161495) demonstrated that efanesoctocog alfa prophylaxis provided superior bleed protection compared with pre-trial factor VIII (FVIII) prophylaxis in patients with severe haemophilia A. The aim of this study was to indirectly compare the efficacy of efanesoctocog alfa with non-factor replacement therapy emicizumab in adolescent and adult patients with severe haemophilia A without inhibitors.
Methods: A systematic literature review was conducted to identify phase 3 trials of emicizumab.
Clin Appl Thromb Hemost
November 2024
Faculty of Medecine of Monastir Tunisia, University of Monastir, Monastir, Tunisia.
Background: Emicizumab, a bispecific factor VIII mimetic antibody, was approved in 2018 for bleeding prophylaxis in congenital hemophilia A with or without inhibitors. Since then, several case reports and case series have described the off-label use of emicizumab in acquired hemophilia A (AHA), and data from two clinical trials were recently published (AGEHA, GTH-AHA-EMI).
Objectives: To describe the reported data on the outcomes of emicizumab, highlighting its benefit/risk profile in treatment.
J Hematol
October 2024
Department of Pediatric Hematology Oncology, Post Graduate Institute of Child Health, Noida, India.
Background: The real-world data on outcome of hemophilia A patients with inhibitors (HAI) is sparse, especially from developing countries. In a setting of inequitable healthcare opportunities for hemophilia patients, especially those with inhibitors, low-dose practices of emicizumab are emerging. In the present article, we describe our experience of managing HAI patients on low-dose emicizumab over a period of 56 months (from December 2019 to August 2024).
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