Table 2.

Therapeutic options for thrombotic thrombocytopenic purpura

Therapeutic option	Indication	Dose	Mechanism of action
Established options
Plasma exchange	Initial therapy in all types of TMA Treatment of choice in autoimmune TTP	60–80 ml/kg/day	Elimination of autoantibodies, immune complexes, UL-VWF MM, sludge Replacement of ADAMTS13 and regularly composed VWF
Plasma infusion	Congenital ADAMTS13 deficiency (Upshaw–Schulman syndrome)	10–40 ml/kg every 2–3 weeks	Replacement of ADAMTS13
Corticosteroids	Autoantibody-induced TTP	1–2 mg/kg/day	Immunosuppression
Rituximab	Autoantibody-induced TTP	4 doses of 100–375 mg/m2/week	Immunosuppression
Immunomodulators (vincristine, MMF, cyclosporine, cyclophosphamide)	Autoantibody-induced TTP (3rd line immunotherapy)	As indicated	Immunosuppression
Anti-platelet agents (ASS, clopidogrel, prasugrel, ticagrelor)	TTP with severe organ damage	Clopidogrel: 75–150 mg/day	Inhibition of platelet aggregation
Splenectomy	Refractory TTP (after rituximab failure)	–	Unknown. Elimination of memory cells?
Supportive therapy	Anemia: RBC transfusion organ failure: ICU	–	(Details: see text)
Future options
Caplacizumaba	Acute autoimmune TTP	10 mg/day sc	Blocking VWF A1 domains, competition with platelet GP Ib/IX
Recombinant ADAMTS13b	Congenital deficiency of ADAMTS13 (Upshaw-Schulman syndrome)	20–40 U/kg every 2–4 weeks	Replacement of ADAMTS13
Recombinant ADAMTS13b	Autoimmune TTP?	Unknown	Replacement of ADAMTS13 to overcome inhibitors
N-acetylcysteineb	Acute and chronic TTP	300 mg/kg/day	Cleavage of UL-VWF MM

TMA thrombotic microangiopathy; TTP thrombotic thrombocytopenic purpura; UL-VWF MM unusually large von Willebrand factor multimers; RBC red blood cells; ICU intensive care unit; ADAMTS13 a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13; GP glycoprotein

^aEarly access program available

^bIn development