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

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 2 patients (Cohort 2) were enrolled. Duration of emicizumab treatment was 8-639 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 1 patient in the primary analysis, no other thrombotic events occurred thereafter. Two patients developed anti-emicizumab antibodies, 1 of whom showed accelerated emicizumab clearance. Tailored IST approaches (delayed initiation, no use, or reduced dose) were successfully executed in 3 patients undergoing emicizumab prophylaxis.

CONCLUSIONS

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

Informations de copyright

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/).

Déclaration de conflit d'intérêts

M. Shima has received research funding from Chugai Pharmaceutical Co., Ltd., CSL Behring, and Takeda; has received honoraria from Bayer, Chugai Pharmaceutical Co., Ltd., CSL Behring, Fujimoto Seiyaku, Sanofi, Novo Nordisk, Pfizer, and Takeda; holds patents with Chugai Pharmaceutical Co., Ltd.; and has participated on a data safety monitoring board or advisory board for Chugai Pharmaceutical Co., Ltd., Fujimoto Seiyaku, KYORIN Pharmaceutical Co., Ltd., Novo Nordisk, and Pfizer. N.S. has received honoraria from Bayer, Chugai Pharmaceutical Co., Ltd., CSL Behring, Japan Blood Products Organization, KM Biologics, Novo Nordisk, Pfizer, Sanofi, and Takeda. K.A. has received research funding from KM Biologics; has received consulting fees from Chugai Pharmaceutical Co., Ltd.; has received honoraria from Bayer, Chugai Pharmaceutical Co., Ltd., CSL Behring, Fujimoto Pharmaceutical Corporation, Japan Blood Products Organization, KM Biologics, Novo Nordisk, Pfizer, Sanofi, and Takeda; has participated on a data safety monitoring board or advisory board for Chugai Pharmaceutical Co., Ltd.; and is belong to endowed chair for CSL Behring. Y.O. has received consulting fees and honoraria from Chugai Pharmaceutical Co., Ltd. R.K. and R.O. are employees of Chugai Pharmaceutical Co., Ltd. K.Y. is an employee of Chugai Pharmaceutical Co., Ltd. and an inventor of patents related to anti-activated FIX/FX bispecific antibodies. N.M. is an employee of Chugai Pharmaceutical Co., Ltd. and has stocks of Chugai Pharmaceutical Co., Ltd. E.S. has received research funding from Eisai; has received honoraria from Janssen, Novartis, Pfizer, Sanofi and Takeda and has leadership or fiduciary role in other board, society, committee or advocacy group (unpaid) for Japan Adult Leukemia Study Group and Japanese Society of Myeloma. S.H. has received research funding from Chugai Pharmaceutical Co., Ltd. and has received honoraria from Bayer, Chugai Pharmaceutical Co., Ltd., CSL Behring, Novo Nordisk, Sanofi, and Takeda. K.N. has received research funding from Bayer, Chugai Pharmaceutical Co., Ltd., CSL Behring, KM Biologics, Novo Nordisk, Sanofi, and Takeda; has received consulting fees from Chugai Pharmaceutical Co., Ltd.; and has received honoraria from Bayer, Chugai Pharmaceutical Co., Ltd., CSL Behring, KM Biologics, Novo Nordisk, Sanofi, and Takeda. The remaining authors have no conflicts of interest to declare.