已入深夜,您辛苦了!由于当前在线用户较少,发布求助请尽量完整地填写文献信息,科研通机器人24小时在线,伴您度过漫漫科研夜!祝你早点完成任务,早点休息,好梦!

Real‐world experience on the tolerability and safety of emicizumab prophylaxis in paediatric patients with severe haemophilia A with and without FVIII inhibitors

医学 耐受性 不利影响 血友病 药物警戒 恶心 儿科 队列 上市后监督 内科学
作者
Eman Hassan,Lancashire Jonathan,Motwani Jayashree
出处
期刊:Haemophilia [Wiley]
卷期号:27 (6) 被引量:16
标识
DOI:10.1111/hae.14432
摘要

Abstract Background Emicizumab is a bispecific monoclonal antibody that bridges activated factor (F) IX and FX, and maintains haemostasis in patients with haemophilia A (PwHA). As a novel agent, many questions remain unanswered about the loss of emicizumab efficacy due to anti‐drug antibody (ADA) development, the incidence of inhibitor recurrence in previously tolerized patients, and the risk of de novo inhibitor development. Aim To present real‐world experience regarding tolerability, side effects, and outcomes of adverse events of emicizumab prophylaxis in paediatric PwHA. Methods Data on tolerability, compliance, adverse events, and laboratory results of paediatric patients receiving emicizumab prophylaxis, treated at the Haemophilia Comprehensive Care Centre, at Birmingham Children's Hospital between March 2018 and June 2021, were collected. Results Our results showed that out of 52 patients, four experienced minor adverse events, two developed headaches, one developed abdominal pain and nausea, and one developed injection site reactions. Moreover, four patients experienced major adverse events, including severe headaches, major bleeding events, development of ADAs, and recurrence of inhibitors. Emicizumab prophylaxis was discontinued in three patients (5.7% of the cohort) due to adverse events. In addition, emicizumab was discontinued in one patient because of poor compliance. No adverse events were reported in previously untreated/minimally treated patients, represented by four patients in our cohort. Conclusions The real‐world experience of emicizumab prophylaxis in our cohort showed that emicizumab was safe and well tolerated in paediatric PwHA with and without inhibitors. Long‐term assessment is crucial to monitor major adverse events, recurrence of inhibitors, and development of ADAs.
最长约 10秒,即可获得该文献文件

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
1秒前
苗条的立果完成签到 ,获得积分10
2秒前
snapu发布了新的文献求助10
2秒前
小阳阳5010完成签到 ,获得积分10
3秒前
3秒前
tanhaowen完成签到 ,获得积分10
4秒前
Dravia完成签到,获得积分10
4秒前
lair发布了新的文献求助10
5秒前
song发布了新的文献求助10
10秒前
12秒前
ganson完成签到 ,获得积分10
16秒前
思源应助walalalla采纳,获得10
17秒前
田一点发布了新的文献求助10
18秒前
天真的不凡完成签到 ,获得积分10
20秒前
顾矜应助wuhao88采纳,获得10
24秒前
董羽佳完成签到,获得积分10
25秒前
江城一霸完成签到,获得积分10
26秒前
共享精神应助song采纳,获得10
26秒前
ZZZ完成签到 ,获得积分10
28秒前
科研通AI2S应助标致的问晴采纳,获得10
29秒前
33秒前
34秒前
35秒前
walalalla发布了新的文献求助10
38秒前
Jyy77完成签到 ,获得积分10
39秒前
隐形曼青应助lizigongzhu采纳,获得10
39秒前
调皮冷梅完成签到 ,获得积分10
40秒前
黎乐荷发布了新的文献求助10
40秒前
normankasimodo完成签到 ,获得积分10
41秒前
刘刘完成签到 ,获得积分10
42秒前
43秒前
钱小豪完成签到,获得积分20
44秒前
彭于晏应助www采纳,获得10
46秒前
一直向前发布了新的文献求助10
48秒前
汉堡包应助Yn_采纳,获得10
48秒前
shjyang完成签到,获得积分10
51秒前
科研达人发布了新的文献求助10
54秒前
yyc完成签到,获得积分10
57秒前
Niercol完成签到,获得积分10
1分钟前
walalalla完成签到,获得积分10
1分钟前
高分求助中
A new approach to the extrapolation of accelerated life test data 1000
ACSM’s Guidelines for Exercise Testing and Prescription, 12th edition 500
Indomethacinのヒトにおける経皮吸収 400
Phylogenetic study of the order Polydesmida (Myriapoda: Diplopoda) 370
基于可调谐半导体激光吸收光谱技术泄漏气体检测系统的研究 350
Robot-supported joining of reinforcement textiles with one-sided sewing heads 320
Aktuelle Entwicklungen in der linguistischen Forschung 300
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 3989949
求助须知:如何正确求助?哪些是违规求助? 3532017
关于积分的说明 11255865
捐赠科研通 3270829
什么是DOI,文献DOI怎么找? 1805053
邀请新用户注册赠送积分活动 882233
科研通“疑难数据库(出版商)”最低求助积分说明 809216