ABSTRACT
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 plasma exchange (PEX) and immunosuppression. The objective of this study was to analyze and compare the safety and efficacy of caplacizumab vs 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 patients with iTTP (77 caplacizumab, 78 no caplacizumab) were included. Patients initially treated with caplacizumab had fewer exacerbations (4.5% vs 20.5%; P < .05) and less refractoriness (4.5% vs 14.1%; P < .05) than those who 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 PEX was associated with a lower number of PEX (odds ratio, 7.5; CI, 2.3-12.7; P < .05) and days of hospitalization (odds ratio, 11.2; CI, 5.6-16.9; P < .001) compared with standard therapy. There was no difference in time to clinical remission in patients treated with caplacizumab compared with the use of rituximab. No severe adverse event was described in the caplacizumab group. In summary, caplacizumab reduced exacerbations and refractoriness compared with 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.
Subject(s)
Purpura, Thrombocytopenic, Idiopathic , Purpura, Thrombotic Thrombocytopenic , Thrombosis , Adult , Humans , Purpura, Thrombotic Thrombocytopenic/drug therapy , Rituximab/adverse effects , Retrospective Studies , Standard of CareABSTRACT
El mieloma múltiple (MM) puede afectar al tórax en forma de lesiones óseas, plasmocitomas, infiltraciones pulmonares y derrame pleural. 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.Presentamos el caso de un varón de 60 años que se le diagnosticó de mieloma múltiple. Tras recibir tratamiento y alcanzando remisión completa, se constata recaída precoz en forma de derrame pleural. Mostramos imágenes radiológicas, citológicas y de la traslocación t(14;16) por FISH. Se ha reportado que esta traslocación denota mal pronóstico, una clínica más agresiva y, a diferencia de otros subgrupos moleculares, su resultado no mejora con la introducción de bortezomib.
