Colliou Éloïse, Ribes Agnès, Gaible Clotilde, Marlas Mathilde, Ribes David, Labadens Isabelle, Guerby Paul, Faguer Stanislas
Department of Nephrology and Organ Transplantation, National Referral Centre for Rare Kidney Diseases, University Hospital of Toulouse, Toulouse, France.
Laboratory for Hematology and Hemostasis, University Hospital of Toulouse, Toulouse, France.
Res Pract Thromb Haemost. 2025 Jan 21;9(1):102687. doi: 10.1016/j.rpth.2025.102687. eCollection 2025 Jan.
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.
Is prophylactic use of recombinant (r)ADAMTS-13 during pregnancy in patients with cTTP safe and effective in preventing cTTP relapse?
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.
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.
与ADAMTS - 13缺乏相关的先天性血栓性血小板减少性紫癜（cTTP），在未治疗的情况下，母亲的死亡风险为10%，胎儿丢失风险大于50%。
对于cTTP患者，孕期预防性使用重组（r）ADAMTS - 13在预防cTTP复发方面是否安全有效？
从妊娠17周起每周静脉注射rADAMTS - 13（40单位/千克）。首次给药前ADAMTS - 13活性检测不到，给药后2小时达到正常水平的60%至90%，并在第4至6天检测不到。在分娩前数小时和第3天给予全剂量。孕期未发生cTTP复发，rADAMTS - 13耐受性良好。未产生抗ADAMTS - 13抗体。
孕期预防性使用rADAMTS - 13可能预防cTTP复发并降低胎儿丢失风险，但最佳方案仍需进一步关注。
