A prospective, multicenter, open-label phase III study of emicizumab prophylaxis in patients with acquired hemophilia A

打开标签 医学 多中心研究 前瞻性队列研究 开放标签研究 内科学 儿科 临床试验 不利影响 随机对照试验
作者
Midori Shima,Kagehiro Amano,Yoshiyuki Ogawa,Koichiro Yoneyama,Ryoto Ozaki,Ryota Kobayashi,Emiko Sakaida,Makoto Saito,Takashi Okamura,Toshihiro Ito,Norimichi Hattori,Satoshi Higasa,Nobuaki Suzuki,Yoshinobu Seki,Keiji Nogami
出处
期刊:Journal of Thrombosis and Haemostasis [Elsevier BV]
卷期号:21 (3): 534-545 被引量:53
标识
DOI:10.1016/j.jtha.2022.10.004
摘要

Emicizumab is a bispecific antibody that mimics the cofactor function of activated factor (F) VIII. It prevents bleeds in patients with congenital hemophilia A regardless of the inhibitor status; however, no prospective clinical studies have been conducted for emicizumab in patients with acquired hemophilia A (PwAHA).To describe the primary analysis results from a prospective, multicenter, open-label phase III study evaluating the efficacy, safety, and pharmacokinetics of emicizumab in PwAHA (AGEHA; JapicCTI-205151).Emicizumab was administered subcutaneously at 6 mg/kg on day 1 and 3 mg/kg on day 2, followed by 1.5 mg/kg once weekly from day 8 onward. Predefined criteria for the completion of dosing included FVIII activity of >50 IU/dL.By the cutoff date (April 23, 2021), 12 patients on immunosuppressive therapy were enrolled, and 11 of them (91.7%) completed emicizumab treatment. The mean trough plasma emicizumab concentration rapidly reached a steady state (1 week), achieving the efficacious level that was established in patients with congenital hemophilia A (>30 μg/mL). Before first emicizumab administration, 7 patients (58.3%) experienced 77 major bleeds. During emicizumab treatment, no major bleeds occurred in any patient. Neither death due to bleeding or infection nor any study treatment-related serious adverse event was reported. One asymptomatic, nonserious deep vein thrombosis was discovered with no laboratory findings indicating any trend toward hypercoagulation.These results suggest that emicizumab prophylaxis with the tested dosing regimen and completion criteria may have a favorable benefit-risk profile in PwAHA.

科研通智能强力驱动
Strongly Powered by AbleSci AI
更新
PDF的下载单位、IP信息已删除 (2025-6-4)

科研通是完全免费的文献互助平台,具备全网最快的应助速度,最高的求助完成率。 对每一个文献求助,科研通都将尽心尽力,给求助人一个满意的交代。
实时播报
兴奋奇异果完成签到,获得积分10
刚刚
1秒前
六月发布了新的文献求助10
1秒前
燕子应助沈年年采纳,获得10
2秒前
江夏清完成签到,获得积分10
3秒前
liangshuang完成签到,获得积分20
3秒前
dandan完成签到,获得积分10
4秒前
望北完成签到 ,获得积分10
5秒前
5秒前
chenwei完成签到,获得积分10
6秒前
6秒前
6秒前
7秒前
小柯完成签到,获得积分10
7秒前
liangshuang发布了新的文献求助10
7秒前
戴晓倩发布了新的文献求助10
7秒前
简简单单完成签到,获得积分20
7秒前
8秒前
年轻半雪完成签到,获得积分10
8秒前
光之霓裳完成签到 ,获得积分10
8秒前
zp6666tql完成签到 ,获得积分10
9秒前
9秒前
大个应助zhangjx采纳,获得10
9秒前
ding应助小米采纳,获得10
10秒前
优秀的千柳完成签到,获得积分10
10秒前
Linden_bd完成签到 ,获得积分10
11秒前
胖虎完成签到,获得积分10
11秒前
冷酷严青发布了新的文献求助10
11秒前
Nuyoah发布了新的文献求助10
11秒前
guhuihaozi发布了新的文献求助10
12秒前
xue完成签到,获得积分10
12秒前
共享精神应助糟糕的惜萱采纳,获得10
13秒前
今后应助小九九采纳,获得10
13秒前
13秒前
梦梦发布了新的文献求助10
13秒前
XT666完成签到,获得积分10
13秒前
新威宝贝完成签到,获得积分10
14秒前
oak完成签到,获得积分10
14秒前
15秒前
16秒前
高分求助中
【重要!!请各位用户详细阅读此贴】科研通的精品贴汇总(请勿应助) 10000
Three plays : drama 1000
International Code of Nomenclature for algae, fungi, and plants (Madrid Code) (Regnum Vegetabile) 1000
Semantics for Latin: An Introduction 999
Psychology Applied to Teaching 14th Edition 600
Robot-supported joining of reinforcement textiles with one-sided sewing heads 580
Apiaceae Himalayenses. 2 500
热门求助领域 (近24小时)
化学 材料科学 医学 生物 工程类 有机化学 生物化学 物理 内科学 纳米技术 计算机科学 化学工程 复合材料 遗传学 基因 物理化学 催化作用 冶金 细胞生物学 免疫学
热门帖子
关注 科研通微信公众号,转发送积分 4087773
求助须知:如何正确求助?哪些是违规求助? 3626669
关于积分的说明 11499781
捐赠科研通 3339508
什么是DOI,文献DOI怎么找? 1836012
邀请新用户注册赠送积分活动 904153
科研通“疑难数据库(出版商)”最低求助积分说明 822092