Caplacizumab Therapy in Immune Thrombotic Thrombocytopenic Purpura

Effectiveness of Adjunctive Caplacizumab Treatment in Immune Thrombotic Thrombocytopenic Purpura in Real-Life Setting. Retrospective Monocentric Cohort

Effect of Caplacizumab in the Recovery of ADAMTS13 Level in Patients with Autoimmune Thrombotic Thrombocytopenic Purpura. Analysis from the Spanish Registry (REPTT)

Immune thrombotic thrombocytopenic purpura in Armenia: A case series highlighting the role of early empiric therapy and multidisciplinary management

Thrombotic thrombocytopenic purpura and its diagnosis

Daratumumab for Refractory and Frequently Relapsing Immune Thrombotic Thrombocytopenic Purpura - a Case Series with Long-Term Follow-up

Caplacizumab in Immune-Mediated Acquired Thrombotic Thrombocytopenic Purpura: A Systematic Literature Review and Meta-Analyses of Clinical Trials and Observational Studies

Detection of Monoclonal Immunoglobulin By Mass Spectrometry in Patients Evaluated for Thrombotic Microangiopathy (TMA)

Thrombotic Thrombocytopenic Purpura (ADAMTS13 [a Disintegrin and Metalloproteinase With a Thrombospondin Type 1 Motif, Member 13] Deficiency) as Cause of Recurrent Multiterritory Ischemic Strokes.

Treatment of immune thrombotic thrombocytopenic purpura (iTTP) includes plasma exchange (PEX) and immunosuppression (glucocorticoids and rituximab). The addition of caplacizumab to therapy has improved treatment outcomes in iTTP. However, the available therapies focus on the duration of drug administration and clinical response rather than ADAMTS13 activity. To evaluate the efficacy of therapy for iTTP targeting ADAMTS13 activity. Treatment of patients with iTTP was started with PEX, prednisolone (1 mg/kg) and caplacizumab (10 mg/day). PEX was discontinued after an increase of platelet count > 150×109/L. Only after PEX cessation treatment with rituximab (375 mg/m2 weekly) was started. Caplacizumab was discontinued when partial remission (ADAMTS13 > 20%) was achieved. Rituximab and glucocorticoids were discontinued when complete remission (ADAMTS13 > 40%) was achieved. Platelet count, schistocyte count, haemoglobin, haptoglobin, lactate dehydrogenase activity, ADAMTS13, ADAMTS13 inhibitor titre, number of PEX, plasma volume replaced, time to increase platelet count > 150×109/L, achievement of partial and complete remission were analyzed. Data are presented as median and interquartile range. From 2021 to 2025, the diagnosis of TTP was confirmed in 102 patients. 35 patients were included in the study. Platelet counts > 150×109/L were achieved after 4 (3-5) PEX procedures in 4 (3-4.5) days. In total, 11 395 (7241-16 343) ml of plasma were exchanged. Partial remission was achieved in 100% of patients, the duration of caplacizumab therapy was 23 (12-30) days. Rituximab was administered from 4 to 8 times (median 4), complete remission was achieved in 33 out of 35 patients, 2 patients achieved only partial remission, they were treated with bortezomib and 1 with anti-CD38 monoclonal antibody. The probability of complete remission was 97.1%. The duration of therapy with caplacizumab, rituximab and glucocorticoids in patients with iTTP should be determined by the achievement of target ADAMTS13 activity.

Randomized clinical trials in thrombotic thrombocytopenic purpura: where do we go from here?

Guidelines on the diagnosis and management of the thrombotic microangiopathic haemolytic anaemias.

A Descriptive Analysis of Immune Thrombotic Thrombocytopenic Purpura (iTTP) Patients with Fatal Outcomes in the U.S. Thrombotic Microangiopathy (USTMA) TTP Registry

Diagnostic and Prognostic Values Of ADAMTS13 Activity Measured During Daily Plasma Exchange Therapy In Patients With Acquired TTP

Caplacizumab as a pre-emptive therapy in thrombotic thrombocytopenic purpura: A proactive strategy to reduce morbidity

Correction of ADAMTS13 Deficiency by In Utero Gene Transfer of Lentiviral Vector encoding ADAMTS13 Genes