Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by anti-ADAMTS13 antibodies. Caplacizumab is approved for adults with an acute episode of iTTP in conjunction with PEX and immunosuppression. The objective of this study is to analyze and compare the safety and efficacy of caplacizumab versus the standard of care and assess the effect of the concomitant use of rituximab. A retrospective study from the Spanish TTP Registry of patients treated with caplacizumab vs those who did not receive it was conducted. A total of 155 iTTP patients (77 caplacizumab, 78 no caplacizumab) were included. Patients initially treated with caplacizumab had fewer exacerbations (4.5% vs 20.5% p<0.05) and less refractoriness (4.5% vs 14.1% p<0.05) than those that were not treated. Time to clinical response was shorter when caplacizumab was used as initial treatment vs caplacizumab used after refractoriness or exacerbation. The multivariate analysis showed that its use in the first 3 days after plasma exchange (PEX) was associated with a lower number of PEX (OR 7.5, CI 2.3-12.7; p<0.05) and days of hospitalization (OR 11.2, CI 5.6-16.9; p<0.001) compared to standard therapy. There was no difference in time to clinical remission in patients treated with caplacizumab compared to the use of rituximab. No severe adverse event was described in the caplacizumab group. In summary, caplacizumab reduced exacerbations and refractoriness compared to standard of care regimens. When administered within the first 3 days after PEX it also provided a faster clinical response, reducing hospitalization time and the need for PEX.

El mieloma múltiple (MM) es la segunda neoplasia hematológica maligna más frecuente y se presenta generalmente con dolor óseo, fracturas patológicas y anemia 1 pleural 2 . Si bien el derrame pleural se observa en el 6% de pacientes con mieloma 3 , el derrame pleural por infiltración de células plasmáticas es poco frecuente, con una incidencia del 0,8% y menos de 100 casos descritos hasta la fecha. A un varón de 60 años se le diagnosticó de mieloma múltiple IgA lambda con plasmocitoma extraóseo supraclavicular en enero de 2016. Se trató con inicialmente con ciclos de VRD (bortezomib, lenalidomida y dexametasona), tras el 3º ciclo se constató progresión extramedular con adenopatías axilar, supraclavicular y cervical derecho a las que se realizó una biopsia con aguja gruesa detectándose por citogenética hiperploidía con t(14;16) en el 90% de los núcleos. Recibió posteriormente tratamiento con 2 ciclos de VRD-PACE (VRD-cisplatino, adriamicina, ciclofosfamida y etopésido) y radioterapia en los plasmocitomas, alcanzando remisión completa estricta. En ju Se presentó en septiembre de 2016 con dolor torácico derecho de carácter pleurítico, disnea y astenia. La radiografía de tórax mostró derrame pleural (Fig. 1, flecha) y atelectasia pasiva de los lóbulos medio e inferior derechos.

Immune thrombotic thrombocytopenic purpura (iTTP) is caused by severely reduced activity of ADAMTS13 due to the development of autoantibodies against ADAMTS13. The median age at diagnosis of iTTP is around the fourth decade of life and patients ≥60 years account for less than 25% of cases. Current treatment of iTTP consists of the association of daily plasma exchange (PEx), immunosuppressive therapy with glucocorticoids ± rituximab, and caplacizumab. However, there is scarce information about the safety and efficacy of caplacizumab in patients with iTTP over 60 years of age.