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

Julie Boudali; Benjamin Hallak; Marie Haeck; Anne-Laure Sellier-Leclerc; Marc Ulrich; Paul Coppo; Stéphanie Tellier; François Provôt

doi:10.1007/s40620-021-00992-5

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

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

Authors

Julie Boudali¹, Benjamin Hallak², Marie Haeck³, Anne-Laure Sellier-Leclerc⁴, Marc Ulrich³, Paul Coppo⁵, Stéphanie Tellier², François Provôt⁶

Affiliations

¹ Department of Nephrology, CHRU Lille, Lille, France.
² Department of Pediatric Nephrology and Rheumatolgy, French Reference Center of Rare Renal Diseases (SORARE), CHU Toulouse, Toulouse, France.
³ Department of Nephrology, CHG Valenciennes, Valenciennes, France.
⁴ Department of Pediatric Nephrology, Hôpital Femme Mère Enfant, Bron, France.
⁵ French Reference Center for Thrombotic Microangiopathies, Hôpital Saint Antoine, AP-HP, Paris, France.
⁶ Department of Nephrology, CHRU Lille, Lille, France. francois.provot@chru-lille.fr.

PMID: 33616856
DOI: 10.1007/s40620-021-00992-5

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.

MeSH terms

Adult
Child
Humans
Plasma Exchange
Purpura, Thrombotic Thrombocytopenic* / diagnosis
Purpura, Thrombotic Thrombocytopenic* / drug therapy
Rare Diseases / therapy
Single-Domain Antibodies* / therapeutic use

Substances

Single-Domain Antibodies
caplacizumab