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CC BY-NC-ND 4.0 · Thromb Haemost 2025; 125(05): 449-459
DOI: 10.1055/a-2384-3585
Coagulation and Fibrinolysis

Final Analysis Results from the AGEHA Study: Emicizumab Prophylaxis for Acquired Hemophilia A with or without Immunosuppressive Therapy

Midori Shima
1   Thrombosis and Hemostasis Research Center, Nara Medical University, Kashihara, Japan
2   Department of Pediatrics, Nara Medical University, Kashihara, Japan
,
Nobuaki Suzuki
3   Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan
,
Hidekazu Nishikii
4   Department of Hematology, Faculty of Medicine, University of Tsukuba, Tsukuba, Japan
,
Kagehiro Amano
5   Department of Laboratory Medicine, Tokyo Medical University Hospital, Tokyo, Japan
,
Yoshiyuki Ogawa
6   Department of Hematology, Gunma University Graduate School of Medicine, Maebashi, Japan
,
Ryota Kobayashi 
7   Chugai Pharmaceutical Co., Ltd., Tokyo, Japan
,
Ryoto Ozaki 
7   Chugai Pharmaceutical Co., Ltd., Tokyo, Japan
,
Koichiro Yoneyama 
7   Chugai Pharmaceutical Co., Ltd., Tokyo, Japan
,
Narumi Mizuno
7   Chugai Pharmaceutical Co., Ltd., Tokyo, Japan
,
Emiko Sakaida
8   Department of Hematology, Chiba University Hospital, Chiba, Japan
,
Makoto Saito
9   Blood Disorders Center, Aiiku Hospital, Sapporo, Japan
,
Takashi Okamura
10   Department of Hematology, St. Mary's Hospital, Kurume, Japan
,
Toshihiro Ito
11   Department of Infectious Diseases, National Hospital Organization Sendai Medical Center, Sendai, Japan
,
Norimichi Hattori
12   Division of Hematology, Department of Medicine, Showa University School of Medicine, Tokyo, Japan
,
Satoshi Higasa
13   Department of Hematology, Hyogo College of Medicine Hospital, Nishinomiya, Japan
,
Yoshinobu Seki
14   Department of Hematology, Uonuma Institute of Community Medicine, Niigata University Medical and Dental Hospital, Minami-uonuma, Japan
,
Keiji Nogami
2   Department of Pediatrics, Nara Medical University, Kashihara, Japan
› Author Affiliations
Funding This study was funded by Chugai Pharmaceutical Co., Ltd.
› Further Information
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Abstract

Background

Primary analysis of the phase III AGEHA study suggested a favorable benefit–risk profile for emicizumab prophylaxis in patients with acquired hemophilia A (PwAHA); however, only patients undergoing immunosuppressive therapy (IST; Cohort 1) were included.

Objectives

To present final analysis results of AGEHA, including data on IST-ineligible patients (Cohort 2) and on long-term prophylaxis with emicizumab.

Methods

For patients in both Cohorts 1 and 2, emicizumab was administered subcutaneously at 6 mg/kg on Day 1, 3 mg/kg on Day 2, and 1.5 mg/kg once weekly from Day 8 onward.

Results

Twelve patients (Cohort 1) and two patients (Cohort 2) were enrolled. Duration of emicizumab treatment was 8 to 639 days (median: 44.5 days) in Cohort 1 and 64 and 450 days in Cohort 2. In both cohorts, no major bleeds were observed after initial emicizumab administration. Six patients started their first rehabilitation sessions during emicizumab treatment and no rehabilitation-related bleeds occurred. Twenty-three surgeries were performed under emicizumab prophylaxis and there were no bleeds related to surgeries. Although asymptomatic deep vein thrombosis was reported in one patient in the primary analysis, no other thrombotic events occurred thereafter. Two patients developed anti-emicizumab antibodies, one of whom showed accelerated emicizumab clearance. Tailored IST approaches (delayed initiation, no use, or reduced dose) were successfully executed in three patients undergoing emicizumab prophylaxis.

Conclusion

These results suggest that emicizumab prophylaxis has a favorable benefit–risk profile in PwAHA regardless of eligibility for IST.

Keywords

Factor VIII deficiency, acquired - long-term care - immunosuppressive therapy - rehabilitation - surgery

Data Availability Statement

Chugai's clinical trial data sharing policy is available at www.chugai-pharm.co.jp/english/profile/rd/ctds_request.html.


Authors' Contribution

M. Shima, R.K., and N.M. wrote and edited the manuscript. M. Shima, K.A., Y.O., R.K., R.O., K.Y., and K.N. designed the study. N.S., H.N., K.A., Y.O., E.S., M. Saito, T.O., T.I., N.H., S.H., and Y.S. acquired the data. K.Y. and R.O. analyzed the data. All authors interpreted the data, critically reviewed the manuscript, approved the final version, and supported the publication.


Supplementary Material



Publication History

Received: 21 February 2024

Accepted: 04 August 2024

Accepted Manuscript online:
12 August 2024

Article published online:
12 September 2024

© 2024. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/)

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