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儿科患者的止血和血栓形成障碍

Hemostatic and thrombotic disorders in the pediatric patient.

作者信息

O'Brien Sarah H, Zia Ayesha

机构信息

Division of Pediatric Hematology/Oncology and.

Department of Pediatrics, The Ohio State University, Columbus, OH.

出版信息

Blood. 2022 Aug 11;140(6):533-541. doi: 10.1182/blood.2020006477.

DOI:10.1182/blood.2020006477
PMID:34724564
Abstract

This review focuses on significant advances in the field of pediatric hemostasis and thrombosis, with a focus on published studies within the past decade. The evaluation and management of patients with excessive bleeding remain cornerstones of consultative hematology. We will describe the development of validated bleeding assessment tools relevant to pediatric practice, laboratory advances in the evaluation of von Willebrand disease, and a shift in clinical practice regarding the interpretation of normal coagulation studies in patients with significant bleeding phenotypes. There have also been critical advances in the management of hemostatic disorders. This review highlights new treatment paradigms in hemophilia and the rise of multidisciplinary medical homes for women living with bleeding disorders. Given the continued increase in the incidence of thrombosis, particularly in the hospital setting, a full call to arms against pediatric venous thromboembolism is now essential. We will describe recently completed clinical trials of direct oral anticoagulants in children and adolescents and ongoing work to elucidate the appropriate duration of therapy for children with provoked thrombosis. Recent work regarding the prevention of pediatric venous thromboembolism is highlighted, including studies of thromboprophylaxis and the development of risk prediction models for hospital-acquired thrombosis. Finally, we review advances in our understanding of thrombotic sequelae and the need for continued refinement of our evaluation tools. Despite the significant advances in pediatric hemostasis and thrombosis over the past decade, many unanswered questions remain for the next generation of investigators.

摘要

本综述聚焦于儿科止血与血栓形成领域的重大进展,重点关注过去十年内发表的研究。对出血过多患者的评估与管理仍是血液学咨询的基石。我们将描述与儿科实践相关的有效出血评估工具的发展、血管性血友病评估的实验室进展,以及在具有显著出血表型患者中正常凝血检查解读方面临床实践的转变。止血障碍的管理也取得了关键进展。本综述强调了血友病的新治疗模式以及出血性疾病女性患者多学科医疗之家的兴起。鉴于血栓形成的发病率持续上升,尤其是在医院环境中,现在全面呼吁应对儿科静脉血栓栓塞至关重要。我们将描述近期完成的儿童和青少年直接口服抗凝剂的临床试验,以及为阐明诱因性血栓形成儿童的适当治疗时长而正在进行的工作。重点介绍了近期关于预防儿科静脉血栓栓塞的工作,包括血栓预防研究以及医院获得性血栓形成风险预测模型的开发。最后,我们回顾了在血栓形成后遗症理解方面的进展以及持续完善评估工具的必要性。尽管过去十年儿科止血与血栓形成领域取得了重大进展,但仍有许多问题有待下一代研究人员解答。

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JAMA. 2022 Jan 11;327(2):129-137. doi: 10.1001/jama.2021.23182.
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Whole-exome analysis of adolescents with low VWF and heavy menstrual bleeding identifies novel genetic associations.对低 VWF 和月经过多青少年进行全外显子组分析鉴定出新的遗传关联。
Blood Adv. 2022 Jan 25;6(2):420-428. doi: 10.1182/bloodadvances.2021005118.
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"If I wasn't a girl": Experiences of adolescent girls with heavy menstrual bleeding and inherited bleeding disorders.
“如果我不是个女孩”:青春期女孩月经过多及遗传性出血性疾病的经历
Res Pract Thromb Haemost. 2022 May 30;6(4):e12727. doi: 10.1002/rth2.12727. eCollection 2022 May.
Pharmacokinetic modeling and simulation support for age- and weight-adjusted dosing of dabigatran etexilate in children with venous thromboembolism.
达比加群酯在儿童静脉血栓栓塞症中按年龄和体重调整剂量的药代动力学建模与模拟支持。
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Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies.HAVEN 1-4 研究中依库珠单抗预防伴或不伴 FVIII 抑制剂的血友病 A 的长期疗效。
Blood. 2021 Apr 22;137(16):2231-2242. doi: 10.1182/blood.2020009217.
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Dabigatran etexilate for the treatment of acute venous thromboembolism in children (DIVERSITY): a randomised, controlled, open-label, phase 2b/3, non-inferiority trial.达比加群酯治疗儿童急性静脉血栓栓塞症(DIVERSITY):一项随机、对照、开放标签、2b/3 期、非劣效性试验。
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