Successful Treatment of Acquired Thrombotic Thrombocytopenic Purpura With Caplacizumab Combined With Plasma Exchanges and Immune Suppression in 3 Children.

Autor:	Kalinina II; Department of General Hematology, Dmitri Rogachev National Research Center for Pediatric Hematology, Oncology and Immunology., Antonova KS; Department of General Hematology, Dmitri Rogachev National Research Center for Pediatric Hematology, Oncology and Immunology., Avdonin PV; Laboratory of the Physiology of Receptors and Signal Pathways, N.K. Koltsov Institute of Developmental Biology., Klebanova EE; Express-Laboratory of ICU, National Research Center for Hematology, Moscow, Russian Federation., Kotskaya NN; Department of General Hematology, Dmitri Rogachev National Research Center for Pediatric Hematology, Oncology and Immunology., Kurnikova EE; Department of Blood Transfusion, Dmitri Rogachev National Research Center for Pediatric Hematology, Oncology and Immunology., Shutova AD; Department of General Hematology, Dmitri Rogachev National Research Center for Pediatric Hematology, Oncology and Immunology., Matveev VE; Department of General Hematology, Dmitri Rogachev National Research Center for Pediatric Hematology, Oncology and Immunology., Maschan AA; Department of General Hematology, Dmitri Rogachev National Research Center for Pediatric Hematology, Oncology and Immunology.
Jazyk:	angličtina
Zdroj:	Journal of pediatric hematology/oncology [J Pediatr Hematol Oncol] 2024 Apr 01; Vol. 46 (3), pp. e220-e222. Date of Electronic Publication: 2024 Feb 29.
DOI:	10.1097/MPH.0000000000002843
Abstrakt:	Acquired thrombotic thrombocytopenic (aTTP) purpura is a life-threatening condition that can lead to devastating thromboembolic events. Recently, caplacizumab has been shown to rapidly restore platelet numbers and reduce the risk of severe end-organ damage when added to plasma exchanges (PEXs) and immunosuppression (IST). Here, we report the outcomes in 3 children with aTTP who were treated with caplacizumab in combination with PEXs and IST. In all 3 patients, platelet count increased to >15,000/mm 3 in 24 h and normalized on day 4, whereas normalization of ADAMTS13 activity >50% and elimination of the inhibitor was achieved after 18 to 89 days. Epistaxis was observed in 2 patients and was the only side effect related to caplacizumab. Caplacizumab is a promising agent for first-line treatment of children with aTTP. Competing Interests: The authors declare no conflict of interest. (Copyright © 2024 Wolters Kluwer Health, Inc. All rights reserved.)
Databáze:	MEDLINE
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