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

Midori Shima; Nobuaki Suzuki; Hidekazu Nishikii; Kagehiro Amano; Yoshiyuki Ogawa; Ryota Kobayashi; Ryoto Ozaki; Koichiro Yoneyama; Narumi Mizuno; Emiko Sakaida; Makoto Saito; Takashi Okamura; Toshihiro Ito; Norimichi Hattori; Satoshi Higasa; Yoshinobu Seki; Keiji Nogami

doi:10.1055/a-2384-3585

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

Thromb Haemost. 2024 Sep 12. doi: 10.1055/a-2384-3585. Online ahead of print.

Authors

Midori Shima^{1

2}, Nobuaki Suzuki³, Hidekazu Nishikii⁴, Kagehiro Amano⁵, Yoshiyuki Ogawa⁶, Ryota Kobayashi⁷, Ryoto Ozaki⁷, Koichiro Yoneyama⁷, Narumi Mizuno⁷, Emiko Sakaida⁸, Makoto Saito⁹, Takashi Okamura¹⁰, Toshihiro Ito¹¹, Norimichi Hattori¹², Satoshi Higasa¹³, Yoshinobu Seki¹⁴, Keiji Nogami²

Affiliations

¹ Thrombosis and Hemostasis Research Center, Nara Medical University, Kashihara, Japan.
² Department of Pediatrics, Nara Medical University, Kashihara, Japan.
³ Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan.
⁴ Department of Hematology, Faculty of Medicine, University of Tsukuba, Tsukuba, Japan.
⁵ Department of Laboratory Medicine, Tokyo Medical University Hospital, Tokyo, Japan.
⁶ Department of Hematology, Gunma University Graduate School of Medicine, Maebashi, Japan.
⁷ Chugai Pharmaceutical Co., Ltd., Tokyo, Japan.
⁸ Department of Hematology, Chiba University Hospital, Chiba, Japan.
⁹ Blood Disorders Center, Aiiku Hospital, Sapporo, Japan.
¹⁰ Department of Hematology, St. Mary's Hospital, Kurume, Japan.
¹¹ Department of Infectious Diseases, National Hospital Organization Sendai Medical Center, Sendai, Japan.
¹² Division of Hematology, Department of Medicine, Showa University School of Medicine, Tokyo, Japan.
¹³ Department of Hematology, Hyogo College of Medicine Hospital, Nishinomiya, Japan.
¹⁴ Department of Hematology, Uonuma Institute of Community Medicine, Niigata University Medical and Dental Hospital, Minami-uonuma, Japan.

PMID: 39134043
DOI: 10.1055/a-2384-3585

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.

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