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A Randomized, Multicenter, Open-label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus No Prophylaxis in Persons with Hemophilia A in the Asia-Pacific region (HAVEN 5)

S. Wang1, X. Zhao2, X. Wang3, J. Sun4, A. Chuansumrit5, J. Zhou6, L. Li7, W. Hsu7, J. Xu7, P. Barrington8, R. Yang9

1Peking Union Medical College Hospital, Beijing, China, 2Xiangya Hospital of Centre-South University, Changsha, China, 3Ruijin Hospital Shanghai, Jiaotong University School of Medicine, Shanghai, China, 4Nanfang Hospital, Southern Medical University, Guangzhou, China, 5Ramathibodi Hospital, Bangkok, Thailand, 6Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, Wuhan, China, 7F. Hoffmann-La Roche Ltd, Shanghai, China, 8TranScrip Partners LL, Reading, United Kingdom, 9Tianjin Institute of Hematology & Blood Diseases Hospital, Tianjin, China

Abstract Number: PB0957

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Emicizumab bridges activated factor IX (FIXa) and FX to restore hemostasis in persons with hemophilia A (PwHA), and is widely approved for prophylaxis in PwHA with/without FVIII inhibitors.

Aims: To present the efficacy, safety and pharmacokinetics of emicizumab from the Phase III HAVEN 5 (NCT03315455) trial in PwHA in the Asia-Pacific region.

Methods: Following informed consent and ethics committee approval, PwHA (≥12 years, with/without FVIII inhibitors) were randomized 2:2:1 to receive subcutaneous (SC) emicizumab prophylaxis: 1.5 mg/kg once-weekly (QW), Arm A; 6 mg/kg once every four weeks (Q4W), Arm B, or; no prophylaxis, Arm C. Loading doses were administered for the first four weeks (3 mg/kg QW SC emicizumab; Arms A and B). The primary endpoint was annualized bleeding rate (ABR) for treated bleeds, calculated using a negative binomial-regression model. Secondary endpoints included additional bleeding-related endpoints, adverse events (AEs) and pharmacokinetic analyses.

Results: At clinical cut-off (06/21/2019), 70 participants aged 12-66 (median 29.0) years were enrolled and had completed 24 weeks of treatment. Significant reductions in treated bleed ABRs of 96% were observed with emicizumab prophylaxis (Arm: A, 1.0; B, 1.0) versus no prophylaxis (Arm C, 27.0; p< 0.0001; Table 1). In Arms A and B, 65.5% (19/29) and 55.6% (15/27) of participants had zero treated bleeds respectively, versus 7.1% (1/14) of participants in Arm C. Efficacious mean emicizumab trough plasma concentrations >30 µg/mL were sustained from Week 5. Emicizumab was well tolerated; the most common AEs were upper respiratory tract infection (Arm: A, 31.0%; B, 18.5%; C, 14.3%) and injection site reaction (Arm: A, 13.8%; B, 18.5%; C, 0.0%). No fatalities, thrombotic microangiopathies or thrombotic events were reported. Eight participants developed treatment-induced anti-drug antibodies; one with transient neutralizing potential.

Conclusions: Emicizumab QW or Q4W prophylaxis offers a highly efficacious, well tolerated, flexible option for PwHA from the Asia-Pacific region.


[Table 1. Duration of Exposure and ABRs in the HAVEN 5 Study]

To cite this abstract in AMA style:

Wang S, Zhao X, Wang X, Sun J, Chuansumrit A, Zhou J, Li L, Hsu W, Xu J, Barrington P, Yang R. A Randomized, Multicenter, Open-label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus No Prophylaxis in Persons with Hemophilia A in the Asia-Pacific region (HAVEN 5) [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/a-randomized-multicenter-open-label-phase-iii-clinical-trial-to-evaluate-the-efficacy-safety-and-pharmacokinetics-of-prophylactic-emicizumab-versus-no-prophylaxis-in-persons-with-hemophilia-a-in/. Accessed September 21, 2023.

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