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Prophylactic use of recombinant ADAMTS-13 during pregnancy for congenital thrombotic thrombocytopenic purpura
Éloïse Colliou1, Agnès Ribes2, Clotilde Gaible1
1Department of Nephrology and Organ Transplantation, National Referral Centre for Rare Kidney Diseases, University Hospital of Toulouse, Toulouse, France.
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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