Brazilian experience with caplacizumab in acquired thrombotic thrombocytopenic purpura: outcomes of the expanded access program

Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare disease with an acute and severe clinical presentation. The anti-von Willebrand factor caplacizumab was licensed for adults with aTTP based on prospective controlled trials. However, until now, there was no Brazilian experience with this new treatment modality. This retrospective, multicenter, single-arm, expanded access program (EAP) with caplacizumab, plasma exchange (PEX), and immunosuppression was conducted between 02/24/21 and 04/14/21, and enrolled 5 Brazilian patients with aTTP. EAP allowed access to caplacizumab in Brazil and real-world data was collected, at a time when the medication was not commercially available in Brazil. The median age was 31 years old, most patients were women (80%), and neurological manifestation was observed in 80% of cases. The median of laboratory tests was hemoglobin (Hb) of 11 g/dL, platelets (16.1 × 10(9)/L), lactic dehydrogenase (LDH) of 1471 U/L, creatinine (0.7 mg/dL), ADAMTS13 activity lower than 0.71%, and PLASMIC score of 6. All patients received immunosuppression, PEX, and caplacizumab. Until clinical response was achieved, the median was 3 sessions of PEX and 3 days of treatment. The median time of caplacizumab use was 35 days, with platelet normalization in 2 days after starting the drug. The median total length of stay was 8 days. All patients achieved clinical response and clinical remission, with a good safety profile. There was rapid clinical response, few PEX sessions were necessary, and there were short hospital stay, absence of refractoriness, little exacerbation, no death, and resolution of signs and symptoms at diagnosis.