Caplacizumab as an Emerging Treatment for Patients With Thrombotic Thrombocytopenic Purpura Presenting With Acute Ischemic Stroke.

Abstract

Objectives: Thrombotic thrombocytopenic purpura (TTP) is rare, life-threatening autoimmune disorder characterized by microvascular thrombosis, severe thrombocytopenia, and hemolytic anemia. It can lead to organ ischemia and increase the risk of thromboembolic events, including acute ischemic stroke (AIS). Caplacizumab, an essential adjunct in TTP management, rapidly inhibits platelet aggregation and prevents disease progression.

Methods: We present 3 cases of TTP diagnosed in patients with AIS. Treatment included plasma exchange (PLEX), corticosteroids, and caplacizumab.

Results: All 3 patients exhibited acute neurologic deficits, with brain MRI confirming AIS. Laboratory tests revealed thrombocytopenia, hemolytic anemia, and ADAMTS-13 activity <1%, confirming TTP. Two patients initially treated only with PLEX and corticosteroids experienced thrombocytopenia exacerbation, requiring caplacizumab for stabilization. The third patient, treated with caplacizumab from stroke onset, maintained stable platelet counts without exacerbation. No adverse events or deaths occurred, emphasizing caplacizumab's role in sustained hematologic recovery.

Discussion: This case series underscores caplacizumab's potential role in stabilizing platelet counts, reducing exacerbation rates, and improving clinical outcomes in TTP-associated AIS. Although all patients experienced favorable neurologic outcomes, a faster recovery cannot be directly attributed to caplacizumab given the multimodal treatment approach. These findings are suggestive of its early use as first-line adjunct, potentially optimizing treatment strategies and improving prognosis.