Onkológia S1/2024

First administration of caplacizumab in first-line treatment for acquired acute thrombotic thrombocytopenic purpura – casuistry

Introduction: Thrombotic thrombocytopenic purpura (TTP) is a rare, life-threatening disease belonging to the group of thrombotic microangiopathies (TMA). The cause is a congenital or acquired deficiency of ADAMTS13 activity. Examining the activity of ADAMTS13 helps us to make a quick diagnosis and timely targeted treatment. The current standard in therapy for acquired TTP is therapeutic plasma exchange (TPE) and immunosuppressive therapy. In the future, a triple combination of treatment consisting of therapeutic exchange plasmapheresis (TPE), immunosuppressive treatment and timely addition of the drug caplacizumab should become the standard of treatment, thanks to which the morbidity and mortality of patients are significantly reduced. Acquired TTP with the presence of autoantibodies can be idiopathic or secondary. Secondary TTP is associated, among other conditions, with malignancies, medication, infections, pregnancy, and hematopoietic cell transplantation. Treatment options for secondary TTP, mainly associated with malignancies, are much more complex. Case description: Our case report describes the case of a patient with a newly diagnosed acute form of acquired TTP, the diagnostic procedure, complex treatment management and the successful use of caplacizumab at an early stage in the 1st line of treatment with a rapid onset of treatment response. Conclusion: Early diagnosis and early initiation of treatment are essential for successful TTP therapy. Therefore, close cooperation with centers specialized in the diagnosis and treatment of hemostasis disorders with the possibility of acute examination of ADAMTS13 activity and with specialized hematology and transfusion departments equipped to perform TPE (apheretic unit) is essential. Due to the introduction of TPE and steroids in the treatment, the mortality of patients from this disease has decreased from 90% to 20%, and its further reduction is expected from the current new approaches in the diagnosis and treatment of TTP.

Keywords: Thrombotic thrombocytopenic purpura, deficiency of ADAMTS13 activity, caplacizumab, therapeutic plasma exchange, immunosuppressive therapy, malignancy