Serious bleeds in pediatric persons with hemophilia A on emicizumab prophylaxis.
Serious bleeds in pediatric persons with hemophilia A on emicizumab prophylaxis.
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在预防症中，血友病A的小儿患者的严重出血。
DOI：
10.1016/j.rpth.2023.102238
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发表时间：
2023-11
影响因子：
4.6
通讯作者：
Zia, Ayesha
中科院分区：
文献类型：
--
作者：
Garcia, Jessica;Hammer, Matthew R.;Zia, Ayesha
临床试验表明，emicizumab在预防出血方面取得了成功，但emicizumab在预防儿科人群严重出血方面的有效性的实际数据缺乏。为了报告Emicizumab对儿童a型血友病患者有效性的真实数据，我们完成了一项回顾性图表回顾，在德克萨斯州达拉斯儿童医疗中心，37名年龄≤18岁的儿童男性患者接受Emicizumab预防治疗，中位持续时间为30.5个月。我们确定了4名患有严重血友病A的儿童，有或没有抑制剂，他们经历了诱发性或非诱发性严重出血，需要住院治疗。本研究强调，严重出血，无论是诱发性的还是非诱发性的，都可能发生在患有严重血友病A的儿童患者中。这些发现对于临床医生通过共同决策为患有严重血友病A的儿童患者提供适当的咨询/教育和治疗建议具有重要意义。对于出血控制不佳的血友病患者或参与中度至高风险活动的患者，需要考虑提高emicizumab滴度或替换因子VIII。关于emicizumab预防严重出血的有效性的实际数据缺乏。我们回顾了37例使用emicizumab的儿童A型血友病患者的图表。我们发现4名儿童患有严重血友病A，服用半单抗并伴有严重出血。对于严重出血患者，需要考虑提高艾美单抗滴度或替换因子VIII。
Clinical trials have shown success in bleed prevention with emicizumab, but real-world data on the effectiveness of emicizumab in preventing serious bleeds in the pediatric population are lacking. To report real-world data on the effectiveness of Emicizumab in pediatric persons with hemophilia A. We completed a retrospective chart review of 37 pediatric male patients aged ≤18 years on emicizumab prophylaxis for a median duration of 30.5 months at Children’s Medical Center in Dallas, Texas. We identified 4 pediatric persons with severe hemophilia A with and without inhibitors who experienced a provoked or unprovoked serious bleed requiring hospitalization. This study highlights that serious bleeds, both provoked and unprovoked, can occur in pediatric persons with severe hemophilia A. These findings are important for clinicians to provide appropriate counseling/education and recommendation of treatment for pediatric persons with severe hemophilia A through shared decision making. Up-titration of emicizumab or factor VIII replacement needs consideration in persons with hemophilia with suboptimal bleeding control or who participate in activities categorized as moderate- to high-risk activities. Real-world data on the effectiveness of emicizumab in preventing serious bleeds are lacking. We reviewed charts of 37 pediatric persons with hemophilia A on emicizumab. We found 4 pediatric persons with severe hemophilia A on emicizumab with a serious bleed. Up-titration of emicizumab or factor VIII replacement needs consideration for serious bleeds.
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影响因子：
3.9
作者：
Barg, Assaf A.;Budnik, Ivan;Kenet, Gili
通讯作者：
Kenet, Gili
影响因子：
158.5
作者：
von Drygalski, Annette;Chowdary, Pratima;Knobe, Karin
通讯作者：
Knobe, Karin
影响因子：
3.9
作者：
Ebbert, Patrick T.;Xavier, Frederico;Ragni, Margaret V.
通讯作者：
Ragni, Margaret V.
关于血友病A的出血模式的现实世界数据。
影响因子：
3.9
作者：
Levy-Mendelovich S;Brutman-Barazani T;Budnik I;Avishai E;Barg AA;Levy T;Misgav M;Livnat T;Kenet G
通讯作者：
Kenet G
DOI：
10.1111/hae.14222
发表时间：
2021-03
期刊：
Haemophilia : the official journal of the World Federation of Hemophilia
影响因子：
--
作者：
Kempton C;Trask P;Parnes A;Niggli M;Campinha-Bacote A;U Callaghan M;O'Connell N;Paz-Priel I;Mahlangu JN
通讯作者：
Mahlangu JN