Congenital thrombotic thrombocytopenic purpura

Section editor
	Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA LinkedIn

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All lines of therapy

rADAMTS13 monotherapy

Regimen

Study	Dates of enrollment	Evidence	Comparator	Comparative Efficacy
Scully et al. 2024 (cTTP)	2010-10 to 2014-01	Phase 3 (E-switch-ooc)	Standard treatment	No acute TTP events

Supportive therapy

• ADAMTS13, recombinant (Adzynma)

References

1. cTTP: Scully M, Antun A, Cataland SR, Coppo P, Dossier C, Biebuyck N, Hassenpflug WA, Kentouche K, Knöbl P, Kremer Hovinga JA, López-Fernández MF, Matsumoto M, Ortel TL, Windyga J, Bhattacharya I, Cronin M, Li H, Mellgård B, Patel M, Patwari P, Xiao S, Zhang P, Wang LT; cTTP Phase 3 Study Investigators. Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2024 May 2;390(17):1584-1596. link to original article PubMed NCT03393975