Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura

Congenital thrombotic thrombocytopenic purpura results from hereditary deficiency of ADAMTS13. Prophylactic administration of recombinant ADAMTS13 achieved normal blood levels with limited toxicity and no neutralizing antibodies.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2024-05, Vol.390 (17), p.1584-1596
Hauptverfasser:	Scully, Marie, Antun, Ana, Cataland, Spero R., Coppo, Paul, Dossier, Claire, Biebuyck, Nathalie, Hassenpflug, Wolf-Achim, Kentouche, Karim, Knöbl, Paul, Kremer Hovinga, Johanna A., López-Fernández, M. Fernanda, Matsumoto, Masanori, Ortel, Thomas L., Windyga, Jerzy, Bhattacharya, Indranil, Cronin, Michael, Li, Hong, Mellgård, Björn, Patel, Munjal, Patwari, Parth, Xiao, Shan, Zhang, Pinghai, Wang, Linda T.
Format:	Artikel
Sprache:	eng
Schlagworte:	ADAMTS13 Protein - deficiency ADAMTS13 Protein - genetics Adolescent Adult Adverse events Antibodies Body weight Child Childhood Diseases Coagulation Congenital diseases Cross-Over Studies Disease prevention Female Hematology Humans Hypothesis testing Male Middle Aged Oncology Patients Pediatrics Pharmacokinetics Plasma Prophylaxis Purpura Purpura, Thrombotic Thrombocytopenic - drug therapy Purpura, Thrombotic Thrombocytopenic - genetics Recombinant Proteins - administration & dosage Recombinant Proteins - adverse effects Recombinant Proteins - therapeutic use Thrombocytopenia Thrombocytopenic purpura Thrombotic thrombocytopenic purpura Young Adult
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Beschreibung
Zusammenfassung:	Congenital thrombotic thrombocytopenic purpura results from hereditary deficiency of ADAMTS13. Prophylactic administration of recombinant ADAMTS13 achieved normal blood levels with limited toxicity and no neutralizing antibodies.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2314793