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. 2022 Dec 27;6(24):6219-6227.
doi: 10.1182/bloodadvances.2022008028.

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

Cristina Pascual Izquierdo  1 , María Eva Mingot-Castellano  2 , Ana E Kerguelen Fuentes  3 , José García-Arroba Peinado  4 , Joan Cid  5 , Maria Moraima Jimenez  6 , David Valcarcel  6 , Inés Gómez-Seguí  7 , Javier de la Rubia  7 , Paz Martin  8 , Rosa Goterris  9 , Luis Hernández  10 , Inmaculada Tallón  11 , Sara Varea  12 , Marta Fernández  13 , Nadia García-Muñoz  14 , Míriam Vara  15 , Miguel Fernández Zarzoso  16 , Faustino García-Candel  17 , María Liz Paciello  18 , Irene García-García  18 , Saioa Zalba  19 , Verónica Campuzano  20 , José María Gala  21 , Julia Vidán Estévez  22 , Gemma Moreno Jiménez  23 , José Luis López Lorenzo  24 , Elena González Arias  25 , Carmen Freiría  26 , María Solé  27 , Laura Francisca Ávila Idrovo  28 , José Carlos Hernández Castellet  29 , Naylen Cruz  30 , Esperanza Lavilla  31 , Albert Pérez-Montaña  32 , Jon Ander Atucha  33 , María Esperanza Moreno Beltrán  34 , Juán Ramón Moreno Macías  35 , Ramón Salinas  36 , Julio Del Rio-Garma  37
Affiliations

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

Cristina Pascual Izquierdo et al. Blood Adv. .

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.

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Conflict of interest statement

Conflict-of-interest disclosure: M.E.M.-C. has received honoraria for consulting and as a speaker from Sobi, Amgen, Takeda, Sanofi, Novo Nordisk, Grifols, Novartis, CSL Behring, Werfen; and grant founding from Novo Nordisk, Amgen, Takeda, Alexion. C.P.I. has served as a consultant and provided expert testimony for Sanofi and Takeda. A.E.K.F. has received honoraria for consulting and as a speaker for Sanofi and Novartis. J.G.-A.P. has received honoraria for consulting and as a speaker for Sanofi. J.C. has received research funding from Cerus, Kawasumi Laboratories, and Sanofi; and has received speaker or advisory fees from Cerus, Fresenius Kabi, Grifols, MacoPharma, Sanofi, and TerumoBCT. R.G. has received honoraria from Sanofi for consulting activities. J.d.l.R. has served as a consultant and has provided expert testimony for Sanofi. J.d.R.-G. has received honoraria for consulting from Takeda and Sanofi. D.V. has received honoraria from Sanofi as speaker and for consulting activities. I.G.-S. has received honoraria for consulting and as a speaker from Terumo BCT, Takeda and Sanofi. J.R.R.M. has received honoraria for consultancy on advisory boards and as a speaker from Janssen, AbbVie, AstraZeneca, CSL-Behring, Takeda, and GSK. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Patient distribution.
Figure 2.
Figure 2.
Survival analysis. Time to clinical response in patients treated with caplacizumab as initial treatment (n = 43∗) vs caplacizumab used in refractoriness or exacerbation (n = 32∗). (It should be noted that all patients also received PEX and corticosteroids). ∗Missing values.
Figure 3.
Figure 3.
Survival analysis. Time to clinical response in patients treated with rituximab + PEX, with or without the addition of caplacizumab (n = 111∗). (It should be noted that all patients also received corticosteroids.) ∗Seven patients were lost to follow-up: 2 patients receiving rituximab and 5 patients receiving rituximab and caplacizumab. Capla, caplacizumab.
Figure 4.
Figure 4.
Time to relapse in patients treated with caplacizumab + rituximab vs patients treated with caplacizumab alone (n = 75∗). (It should be noted that all patients also received PEX and corticosteroids.) ∗Two patients were lost to follow-up in the caplacizumab + rituximab group. Capla, caplacizumab.
See this image and copyright information in PMC

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