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  3. Real-world effectiveness of caplacizumab vs standard of care in immune thrombotic thrombocytopenic purpura.

Real-world effectiveness of caplacizumab vs standard of care in immune thrombotic thrombocytopenic purpura.

Fecha de publicación: Fecha Ahead of Print:

Autores de IIS La Fe

Participantes ajenos a IIS La Fe

  • Pascual Izquierdo, Maria Cristina
  • Kerguelen Fuentes, Ana Esther
  • Garcia-Arroba Peinado, Jose
  • Cid, Joan
  • Jimenez, Maria Moraima
  • Valcarcel, David
  • Martin, Paz
  • Goterris, Rosa
  • Hernandez, Luis
  • Tallon, Inmaculada
  • Varea, Sara
  • Fernandez, Marta
  • Garcia-Munoz, Nadia
  • Vara, Miriam
  • Fernandez Zarzoso, Miguel
  • Garcia-Candel, Faustino
  • Paciello, Maria Liz
  • Garcia-Garcia, Irene
  • Zalba, Saioa
  • Campuzano, Veronica
  • Garcia-Gala, Jose M M
  • Vidan, Julia
  • Moreno, Gemma
  • Lopez Lorenzo, Jose Luis
  • Gonzalez Arias, Elena
  • Freiria, Carmen
  • Sole, Maria
  • Avila Idrovo, Laura Francisca
  • Hernandez Castellet, Jose Carlos
  • Cruz, Naylen
  • Lavilla, Esperanza
  • Perez-Montana, Albert
  • Atucha, Jon Ander
  • Moreno Beltran, Maria Esperanza
  • Romero Macias, Juan-Ramon
  • Salinas, Ramon
  • Del Rio-Garma, Julio

Grupos

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 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.

Copyright © 2022 American Society of Hematology.

Datos de la publicación

ISSN/ISSNe:
2473-9529, 2473-9537

Blood Advances  ELSEVIER

Tipo:
Article
Páginas:
6219-6227
PubMed:
35930694
Factor de Impacto:
2,795 SCImago ℠
Cuartil:
Q1 SCImago ℠

Citas Recibidas en Web of Science: 46

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Keywords

  • TTP; CONSENSUS

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