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 efficacy of CABLIVI for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy was established in a pivotal multicenter, randomized, double-blind, placebo-controlled trial (HERCULES)
The diagnosis of Thrombotic Thrombocytopenic Purpura (TTP) is not directly stated in the provided drug labels. The treatment for TTP is caplacizumab (CABLIVI) in combination with plasma exchange and immunosuppressive therapy 1, 1, 1. Key points about the treatment include:
- CABLIVI is administered as an 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, for the duration of the daily plasma exchange period and for 30 days thereafter.
- Treatment may be extended for 7 day intervals for a maximum of 28 days if signs of persistent underlying disease remain present.
From the Research
Thrombotic thrombocytopenic purpura (TTP) is a rare, life-threatening blood disorder requiring immediate treatment with plasma exchange therapy (PEX), as evidenced by the most recent study 2. The standard approach involves daily PEX (1-1.5 plasma volumes) combined with corticosteroids (methylprednisolone 1 mg/kg/day or prednisone 1 mg/kg/day) [ 3, 2 ]. Some studies suggest that caplacizumab and rituximab may be beneficial in certain cases of TTP [not directly mentioned in provided studies, but relevant to current medical knowledge]. Key points to consider in the diagnosis and treatment of TTP include:
- TTP is characterized by thrombocytopenia, microangiopathic hemolytic anemia, and often neurological abnormalities, renal dysfunction, and fever [ 3, 2, 4 ]
- Plasma exchange is the mainstay of treatment and should be initiated promptly [ 3, 2, 5, 6 ]
- The goal of treatment is to normalize the platelet count and prevent further thrombotic complications [ 3, 2 ]
- Daily plasma exchange should be continued until the platelet count exceeds 150,000/μL for at least 2 consecutive days, with ADAMTS13 activity monitoring to guide treatment duration [ 3, 2 ]
- TTP can be fatal if left untreated, but with prompt treatment, survival rates can exceed 80% [ 3, 2, 6 ]