From the FDA Drug Label
CABLIVI is indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.
The treatment for active Thrombotic Thrombocytopenic Purpura (TTP) is caplacizumab in combination with:
- Plasma exchange
- Immunosuppressive therapy 1 Key points about the treatment:
- Caplacizumab is administered as a single 11 mg bolus intravenous injection prior to the first plasma exchange, followed by a daily subcutaneous injection of 11 mg after completion of plasma exchange.
- Treatment duration with caplacizumab is for the duration of the daily plasma exchange period and for 30 days thereafter, with possible extension for 7-day intervals for a maximum of 28 days if signs of persistent underlying disease remain present.
From the Research
The primary treatment for active Thrombotic Thrombocytopenic Purpura (TTP) is therapeutic plasma exchange (TPE) combined with immunosuppressive therapy, including caplacizumab, as evidenced by the most recent study 2.
Treatment Overview
The treatment approach should prioritize reducing morbidity, mortality, and improving quality of life.
- Therapeutic plasma exchange (TPE) should be initiated immediately upon diagnosis or strong suspicion of TTP, with daily exchanges of 1-1.5 plasma volumes until platelet count normalizes and clinical improvement occurs.
- Concurrently, corticosteroids (typically methylprednisolone 1 mg/kg/day or prednisone 1 mg/kg/day) should be administered.
- Rituximab (375 mg/m² weekly for 4 weeks) is often added to the initial treatment regimen to reduce relapse rates.
- Caplacizumab, an anti-von Willebrand factor nanobody, is recommended at 10 mg daily (after an initial 10 mg IV loading dose) throughout the TPE period and for 30 days afterward to rapidly improve platelet counts and reduce thrombotic complications, as supported by 3 and 2.
Additional Considerations
For refractory cases, additional immunosuppressants like cyclophosphamide, vincristine, cyclosporine, or bortezomib may be considered.
- Platelet transfusions should generally be avoided unless life-threatening bleeding occurs, as they may worsen microvascular thrombosis.
- The effectiveness of these treatments stems from addressing the underlying pathophysiology: TPE removes ADAMTS13 autoantibodies and provides functional ADAMTS13, while immunosuppressive therapy inhibits autoantibody production against ADAMTS13, the enzyme deficient in TTP that normally cleaves von Willebrand factor multimers, as discussed in 4, 5, and 6.