EUH Krakow Conference: Treatment of thrombotic thrombocytopenic purpura (TTP).

The Bottom Line: Treat TTP as a medical emergency.  For full treatment details, see the TTP section in DynaMed Plus.

Acquired TTP:

  • Initiate plasma exchange
  • Give methylprednisolone or high-dose oral prednisolone
  • If plasma exchange is unavailable/delayed, consider large plasma infusions if tolerated in conjuction with pulsed methyl prednisolone
  • Consider rituximab with plasma exchange and corticosteroids for patients with neurological/cardiac pathology

Congenital TTP:

  • Treatment regime should be individualized according to patient’s phenotype
  • Give solvent/detergent-treated plasma infusion or ADAMTS13-containing intermediate purity factor VIII
  • Refer pregnant women to specialist treatment centers and give ADAMTS13 supplementation regularly throughout pregnancy and postpartum period

Secondary TTP and ADAMTS13 < 10%:

  • Treat with standard therapy – plasma exchange and corticosteroids
  • Treat underlying disorder and/or remove underlying trigger

Refractory/Relapsing TTP:

  • Increase frequency of plasma exchange
  • Rituximab

Emerging therapies include bortezomib, recombinant ADAMTS13, and anti-von Willebrand factor therapy.

(DyanMed Plus, 2017)

References: DynaMed Plus [Internet]. Ipswich (MA): EBSCO Information Services. Record No. 115863, Thrombotic thrombocytopenic purpura (TTP); [updated 2017 Oct 04, cited 2017 Oct 27]; [about 16 screens].

Saha M, McDaniel JK, Zheng XL. Thrombotic thrombocytopenic purpura: pathogenesis, diagnosis and potential novel therapeutics. J Thromb Haemost. 2017 Oct;15(10):1889-1900. doi:10.1111/jth.13764.

Summary: TTP is a potentially fatal clinical syndrome and is primarily caused by autoantibodies against the von Willebrand factor (VWF)-cleaving metalloprotease ADAMTS13. Severe deficiency of plasma ADAMTS13 activity with or without detectable inhibitory autoantibodies against ADAMTS13 supports diagnosis of TTP (Saha, McDaniel, and Zheng, 2017).

Prophylactic plasma infusion remains the mainstay for treatment of hereditary TTP resulting from mutations in ADAMTS13. According to Saha et al (2017), 5 mL kg-1 bi-weekly fresh frozen plasma seems sufficient to prevent acute episodes. However, when acute TTP develops, 10 mL kg-1 every 12 weeks may be necessary for treatment.

Therapeutic plasma exchange (TPE) is the treatment of choice for acquired TTP. Other immunosuppressive therapies including corticosteroids, vincristine, cyclophosphamide, cyclosporien, and rituximab, and bortezomib may be considered in patients with acquired TTP to eliminate antibody formation and sustain a long-term remission. Some physicians are reluctant to use platelet transfusion; Swisher et al (2009) suggest it should be minimized but can be considered in those with overt bleeding and also during invasive procedures in patients with severe thrombocytopenia (Saha, McDaniel, and Zheng, 2017).