Combining an immunomodulatory drug with a TPO‐RA to treat multirefractory ITP patients: The Spanish ITP Group experience

Multirefractory immune thrombocytopenia (ITP) is difficult to manage. The simultaneous administration of an immunosuppressive/immunomodulatory drug (IS/IM) and a thrombopoietin receptor agonist (TPO-RA) is increasingly used. We present the experience of the Spanish ITP Group in this retrospective, observational study. Ninety-seven patients with ≥3 failed treatment lines were administered combination therapy (CT) with an IS/IM and a TPO-RA between January 2021 and December 2023. The IS/IMs were steroids, purine synthesis inhibitors and fostamatinib. The TPO-RAs were romiplostim, eltrombopag and avatrombopag. Seventy-five (77.3%) patients responded to treatment in a median (interquartile range [IQR]) time of 14 (7-52) days. An inverse correlation between previous failures and response was observed (Rho = -0.290, p = 0.009). After 840 (448-1054) days, relapse was reported in 35 (46.7%) of 75 responders (time to relapse: 197 [47-402] days). Relapse was more frequent in chronic ITP patients. Twenty-one patients received a second CT after failure of the first one. Eleven were administered a third CT for the same reason. Response and relapse were similar to those observed with the first CT. Three thromboembolic events and 14 infections that required hospitalization were reported, none fatal. The combination of IS/IMs and TPO-RAs arises as an alternative option of treatment in multirefractory ITP.

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Journal
British Journal of Haematology
Published
2026-01-02
DOI
https://doi.org/10.1111/bjh.70306
Citations
1
Primary Topic
Platelet Disorders and Treatments
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article
Field-Weighted Citation Impact
35.41
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article

Combining an immunomodulatory drug with a TPO‐RA to treat multirefractory ITP patients: The Spanish ITP Group experience

Rocío Pérez‐Montes, Daniel Martínez‐Carballeira, Sunil Lakhwani, Francesc García‐Pallarols et al.
1 citations
British Journal of Haematology
Platelet Disorders and Treatments
35.41
article

Combining an immunomodulatory drug with a TPO‐RA to treat multirefractory ITP patients: The Spanish ITP Group experience

Rocío Pérez‐Montes, Daniel Martínez‐Carballeira, Sunil Lakhwani, Francesc García‐Pallarols, Patricia Alcalde‐Mellado, Laura Entrena‐Ureña, Mariana Canaro Hirnyk, Begoña Pedrote Amador, Dolores Piquer‐Monsonis, Cristina Pascual, J. Bastida, Marta García‐Culebras, Denis Zafra‐Torres, Blanca Sánchez‐González, Manuel Fernández‐Villalobos, Silvia Bernat‐Pablo, the Spanish ITP Group (GEPTI), Marta Canet‐Maldonado, Carlos Puerta‐Vázquez, María‐Eva Mingot‐Castellano, Beatriz Chiclana‐Rodríguez, María‐del‐Carmen Gómez‐del‐Castillo‐Solano, Isabel‐Socorro Caparrós‐Miranda, Amalia Cuesta‐García, María Poza‐Santaella, Paula Sánchez‐Llorca
article en
1 citations

Abstract

Multirefractory immune thrombocytopenia (ITP) is difficult to manage. The simultaneous administration of an immunosuppressive/immunomodulatory drug (IS/IM) and a thrombopoietin receptor agonist (TPO-RA) is increasingly used. We present the experience of the Spanish ITP Group in this retrospective, observational study. Ninety-seven patients with ≥3 failed treatment lines were administered combination therapy (CT) with an IS/IM and a TPO-RA between January 2021 and December 2023. The IS/IMs were steroids, purine synthesis inhibitors and fostamatinib. The TPO-RAs were romiplostim, eltrombopag and avatrombopag. Seventy-five (77.3%) patients responded to treatment in a median (interquartile range [IQR]) time of 14 (7-52) days. An inverse correlation between previous failures and response was observed (Rho = -0.290, p = 0.009). After 840 (448-1054) days, relapse was reported in 35 (46.7%) of 75 responders (time to relapse: 197 [47-402] days). Relapse was more frequent in chronic ITP patients. Twenty-one patients received a second CT after failure of the first one. Eleven were administered a third CT for the same reason. Response and relapse were similar to those observed with the first CT. Three thromboembolic events and 14 infections that required hospitalization were reported, none fatal. The combination of IS/IMs and TPO-RAs arises as an alternative option of treatment in multirefractory ITP.

British Journal of HaematologyVol. 208(3)
Hospital Universitario de Canarias (ES), Hospital General Universitario Gregorio Marañón (ES), Research Institute Hospital 12 de Octubre (ES), University Hospital Mútua de Terrassa (ES), Complejo Hospitalario de Salamanca (ES), Ayuntamiento de Castellón de la Plana (ES), Hospital Sierrallana (ES), Hospital Universitario Virgen de las Nieves (ES), Hospital Del Mar (ES), Instituto de Biomedicina de Sevilla (ES), Hospital Universitario Central de Asturias (ES), Instituto de Investigación Biomédica de Salamanca (ES), Complexo Hospitalario Universitario A Coruña (ES), Hospital Universitario Son Espases (ES), Hospital Universitario Virgen del Rocío (ES), Hospital Clínico Universitario Virgen de la Victoria (ES)
Good health and well-being
Openalex Percentile: Top 2%
Platelet Disorders and Treatments
35.41
DOISourceOpenAlex
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