妊娠期预防性使用重组ADAMTS-13治疗先天性血栓性血小板减少性紫癜

IF 3.4 3区医学 Q2 HEMATOLOGY

Research and Practice in Thrombosis and Haemostasis Pub Date : 2025-01-01 DOI:10.1016/j.rpth.2025.102687

Éloïse Colliou , Agnès Ribes , Clotilde Gaible , Mathilde Marlas , David Ribes , Isabelle Labadens , Paul Guerby , Stanislas Faguer

{"title":"妊娠期预防性使用重组ADAMTS-13治疗先天性血栓性血小板减少性紫癜","authors":"Éloïse Colliou , Agnès Ribes , Clotilde Gaible , Mathilde Marlas , David Ribes , Isabelle Labadens , Paul Guerby , Stanislas Faguer","doi":"10.1016/j.rpth.2025.102687","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><div>Congenital thrombotic thrombocytopenic purpura (cTTP) related to ADAMTS-13 deficiency is associated with a maternal risk of death of 10% and a risk of fetal loss greater than 50% without treatment.</div></div><div><h3>Key Clinical Question</h3><div>Is prophylactic use of recombinant (r)ADAMTS-13 during pregnancy in patients with cTTP safe and effective in preventing cTTP relapse?</div></div><div><h3>Clinical Approach</h3><div>rADAMTS-13 was given intravenously weekly (40 Units/kg) from 17 weeks’ gestation. ADAMTS-13 activity was undetectable before the first administration, reached 60% to 90% of normal levels 2 hours after, and became undetectable between days 4 and 6. A full dose was given in the hours preceding the delivery and on day 3. No flare-up of cTTP occurred during the pregnancy, and rADAMTS-13 was tolerated well. No anti–ADAMTS-13 antibodies developed.</div></div><div><h3>Conclusion</h3><div>Prophylactic use of rADAMTS-13 during pregnancy may prevent relapse of cTTP and reduce the risk of fetal loss, but an optimal regimen requires further attention.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 1","pages":"Article 102687"},"PeriodicalIF":3.4000,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Prophylactic use of recombinant ADAMTS-13 during pregnancy for congenital thrombotic thrombocytopenic purpura\",\"authors\":\"Éloïse Colliou , Agnès Ribes , Clotilde Gaible , Mathilde Marlas , David Ribes , Isabelle Labadens , Paul Guerby , Stanislas Faguer\",\"doi\":\"10.1016/j.rpth.2025.102687\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Background</h3><div>Congenital thrombotic thrombocytopenic purpura (cTTP) related to ADAMTS-13 deficiency is associated with a maternal risk of death of 10% and a risk of fetal loss greater than 50% without treatment.</div></div><div><h3>Key Clinical Question</h3><div>Is prophylactic use of recombinant (r)ADAMTS-13 during pregnancy in patients with cTTP safe and effective in preventing cTTP relapse?</div></div><div><h3>Clinical Approach</h3><div>rADAMTS-13 was given intravenously weekly (40 Units/kg) from 17 weeks’ gestation. ADAMTS-13 activity was undetectable before the first administration, reached 60% to 90% of normal levels 2 hours after, and became undetectable between days 4 and 6. A full dose was given in the hours preceding the delivery and on day 3. No flare-up of cTTP occurred during the pregnancy, and rADAMTS-13 was tolerated well. No anti–ADAMTS-13 antibodies developed.</div></div><div><h3>Conclusion</h3><div>Prophylactic use of rADAMTS-13 during pregnancy may prevent relapse of cTTP and reduce the risk of fetal loss, but an optimal regimen requires further attention.</div></div>\",\"PeriodicalId\":20893,\"journal\":{\"name\":\"Research and Practice in Thrombosis and Haemostasis\",\"volume\":\"9 1\",\"pages\":\"Article 102687\"},\"PeriodicalIF\":3.4000,\"publicationDate\":\"2025-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Research and Practice in Thrombosis and Haemostasis\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2475037925000111\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Research and Practice in Thrombosis and Haemostasis","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2475037925000111","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}

引用次数: 0

摘要

背景：先天性血栓性血小板减少性紫癜（cTTP）与ADAMTS-13缺乏相关，如果不进行治疗，产妇死亡风险为10%，胎儿死亡风险大于50%。妊娠期cTTP患者预防性使用重组(r)ADAMTS-13预防cTTP复发安全有效吗？临床方法：radamts -13从妊娠17周开始每周静脉滴注（40单位/公斤）。第一次给药前无法检测到ADAMTS-13的活性，2小时后达到正常水平的60%至90%，在第4天至第6天无法检测到。在分娩前数小时和第3天给予全剂量。妊娠期间未发生cTTP发作，rADAMTS-13耐受性良好。未产生抗adamts -13抗体。结论妊娠期预防性使用rADAMTS-13可预防cTTP复发，降低胎儿丢失风险，但需进一步关注最佳方案。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Prophylactic use of recombinant ADAMTS-13 during pregnancy for congenital thrombotic thrombocytopenic purpura

Background

Congenital thrombotic thrombocytopenic purpura (cTTP) related to ADAMTS-13 deficiency is associated with a maternal risk of death of 10% and a risk of fetal loss greater than 50% without treatment.

Key Clinical Question

Is prophylactic use of recombinant (r)ADAMTS-13 during pregnancy in patients with cTTP safe and effective in preventing cTTP relapse?

Clinical Approach

rADAMTS-13 was given intravenously weekly (40 Units/kg) from 17 weeks’ gestation. ADAMTS-13 activity was undetectable before the first administration, reached 60% to 90% of normal levels 2 hours after, and became undetectable between days 4 and 6. A full dose was given in the hours preceding the delivery and on day 3. No flare-up of cTTP occurred during the pregnancy, and rADAMTS-13 was tolerated well. No anti–ADAMTS-13 antibodies developed.

Conclusion

Prophylactic use of rADAMTS-13 during pregnancy may prevent relapse of cTTP and reduce the risk of fetal loss, but an optimal regimen requires further attention.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊