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 answer to the question about thrombotic thrombocytopenia purpura is that caplacizumab (IV), also known as CABLIVI, is indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy 1.
- Key points:
- Indication: treatment of adult patients with aTTP
- Combination therapy: plasma exchange and immunosuppressive therapy
- Drug name: caplacizumab (IV) or CABLIVI The efficacy of CABLIVI for the treatment of adult patients with aTTP in combination with plasma exchange and immunosuppressive therapy was established in a pivotal multicenter, randomized, double-blind, placebo-controlled trial (HERCULES) 1.
From the Research
Thrombotic thrombocytopenic purpura (TTP) is a rare but life-threatening blood disorder requiring immediate treatment with plasma exchange therapy (PEX) and high-dose corticosteroids, as well as consideration of caplacizumab and rituximab in certain cases, as supported by the most recent and highest quality study 2.
Key Points
- TTP results from severe ADAMTS13 deficiency (activity <10%), usually due to autoantibodies that inhibit this enzyme, leading to unchecked von Willebrand factor multimers that cause platelet aggregation and microvascular thrombosis 3.
- The standard approach involves daily PEX (1-1.5 plasma volumes) until platelet count normalizes and hemolysis resolves, typically for at least 2-3 days after platelet recovery 4.
- Concurrent treatment with caplacizumab (10mg IV loading dose followed by 10mg subcutaneous daily) should be initiated during the first PEX session and continued for 30 days after PEX cessation, as recommended by recent studies 2.
- High-dose corticosteroids (methylprednisolone 1g/day IV for 3 days, followed by prednisone 1mg/kg/day) are also recommended, with consideration of rituximab (375mg/m² weekly for 4 weeks) for refractory or relapsing cases 3, 2.
Diagnosis and Management
- TTP is characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and ischemic end organ injury due to microvascular platelet-rich thrombi 2.
- Testing should include a complete blood count, complete metabolic panel, blood smear, coagulation panel, fibrinogen, D-dimer, lactate dehydrogenase, ADAMTS13 level, troponin, human immunodeficiency virus assessment, urinalysis, pregnancy test as appropriate, and electrocardiogram 5.
- Management includes hematology consultation if available, plasma exchange and corticosteroids, and treatment of end-organ complications, with all patients requiring admission for treatment and close monitoring 5.
Prognosis and Outcome
- Without prompt treatment, mortality exceeds 90%, but with appropriate therapy, survival rates exceed 80% [@Example@].
- Long-term follow-up after the acute episode is critical to monitor for relapse and to diagnose and manage chronic sequelae of this disease 2.