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. 2022 Mar;35(2):653-656.
doi: 10.1007/s40620-021-00992-5. Epub 2021 Feb 22.

Immune-mediated thrombotic thrombocytopenic purpura in childhood treated by caplacizumab, about 3 cases

Julie Boudali  1 , Benjamin Hallak  2 , Marie Haeck  3 , Anne-Laure Sellier-Leclerc  4 , Marc Ulrich  3 , Paul Coppo  5 , Stéphanie Tellier  2 , François Provôt  6
Affiliations

Immune-mediated thrombotic thrombocytopenic purpura in childhood treated by caplacizumab, about 3 cases

Julie Boudali et al. J Nephrol. 2022 Mar.

Abstract

Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare disease in adults and exceptional in childhood. Caplacizumab has proven its effectiveness in the treatment of iTTP in adulthood in association with standard of care. Unfortunately, this treatment is restricted to adults. We report our experience in three children who were treated successfully with caplacizumab.

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