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

IF 3.4 3区医学 Q2 HEMATOLOGY Research and Practice in Thrombosis and Haemostasis Pub Date : 2025-01-01 Epub Date: 2025-01-21 DOI:10.1016/j.rpth.2025.102687

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

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Abstract

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.

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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

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。

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