Avatrombopag in immune thrombocytopenia: A real‐world study of the Spanish ITP Group (GEPTI)

Avatrombopag is the newest thrombopoietin receptor agonist (TPO-RA) approved to treat immune thrombocytopenia (ITP). Real-world evidence regarding effectiveness/safety is limited. The Spanish ITP Group (GEPTI) performed a retrospective study with patients starting avatrombopag for the first time. A total of 268 ITP patients were recruited. The median (interquartile range [IQR]) follow-up time was 47.5 (30.4-58.9) weeks. Among the 193 patients with baseline platelet counts <50 × 10<sup>9</sup>/L, 174 (90.1%) of them achieved response (PC ≥50 × 10<sup>9</sup>/L), and 113 (87.6%) of the 129 who persisted on avatrombopag at last visit had platelet levels above such threshold. Results were similar when only those patients switching to avatrombopag due to previous treatment failure were considered (n = 104). Patients reached response in 13 (7-21) days. Among patients with baseline levels ≥50 × 10<sup>9</sup>/L, 73/75 (97.3%) reported response, which was maintained by 53 (94.6%) of the 56 who continued on avatrombopag at the end of the study. Loss-of-response was always <10%. ITP duration did not influence response. Approximately 79% (34/43) of heavily pretreated (≥4 lines) patients with baseline platelet counts <50 × 10<sup>9</sup>/L switching after previous failure achieved PC ≥50 × 10<sup>9</sup>/L. Previous use of eltrombopag and/or romiplostim did not influence response, regardless of whether previous TPO-RA(s) succeeded or failed. Avatrombopag allowed dose-reduction/suspension of corticosteroids in 40/50 (80.0%) patients with baseline platelet counts <50 × 10<sup>9</sup>/L. Overall, 40/268 (14.9%) thrombocytosis and 12/268 (4.5%) thromboembolic events were reported. Our real-world cohort supports the use of avatrombopag to manage ITP, regardless of disease severity and treatment history.

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Journal
American Journal of Hematology
Published
2024-10-12
DOI
https://doi.org/10.1002/ajh.27498
Citations
9
Primary Topic
Platelet Disorders and Treatments
Type
article
Field-Weighted Citation Impact
4.09

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article

Avatrombopag in immune thrombocytopenia: A real‐world study of the Spanish ITP Group (GEPTI)

M. Marquez Aguirre, Silvia Bernat, Daniel Martínez‐Carballeira, María Eva Mingot‐Castellano et al.
9 citations
American Journal of Hematology
Platelet Disorders and Treatments
4.09
article

Avatrombopag in immune thrombocytopenia: A real‐world study of the Spanish ITP Group (GEPTI)

M. Marquez Aguirre, Silvia Bernat, Daniel Martínez‐Carballeira, María Eva Mingot‐Castellano, Teresa Arquero, Sunil Lakhwani, Gloria García‐Donas, Montserrat Alvarellos, Almudena de‐la‐Iglesia, Elsa López‐Ansoar, Reyes Jiménez‐Bárcenas, María‐José Llácer, Gonzalo Caballero‐Navarro, Blanca Sánchez‐González, Amalia Cuesta, Juan‐José Gil‐Fernández, Isabel‐Teresa González‐Gascón‐y‐Marín, Isidro Jarque‐Ramos, Silvia Monsalvo‐Saornil, Begoña Pedrote Amador, Marta Moreno‐Carbonell, Cristina Pascual, J. Bastida, Isabel Socorro Caparrós-Miranda, Denis Zafra‐Torres, Ariana Ortúzar‐Pasalodos, Nuria Revilla‐Calvo, Esther Chica‐Gullón, María‐Esperanza Moreno‐Beltrán, Álvaro Lorenzo‐Vizcaya, Maialen Lasa‐Eguialde, Mariana‐Isabel Canaro‐Hirnyk, María Menor‐Gómez, Marta Díaz‐López, Marta Canet
article en
9 citations

Abstract

Avatrombopag is the newest thrombopoietin receptor agonist (TPO-RA) approved to treat immune thrombocytopenia (ITP). Real-world evidence regarding effectiveness/safety is limited. The Spanish ITP Group (GEPTI) performed a retrospective study with patients starting avatrombopag for the first time. A total of 268 ITP patients were recruited. The median (interquartile range [IQR]) follow-up time was 47.5 (30.4-58.9) weeks. Among the 193 patients with baseline platelet counts <50 × 10<sup>9</sup>/L, 174 (90.1%) of them achieved response (PC ≥50 × 10<sup>9</sup>/L), and 113 (87.6%) of the 129 who persisted on avatrombopag at last visit had platelet levels above such threshold. Results were similar when only those patients switching to avatrombopag due to previous treatment failure were considered (n = 104). Patients reached response in 13 (7-21) days. Among patients with baseline levels ≥50 × 10<sup>9</sup>/L, 73/75 (97.3%) reported response, which was maintained by 53 (94.6%) of the 56 who continued on avatrombopag at the end of the study. Loss-of-response was always <10%. ITP duration did not influence response. Approximately 79% (34/43) of heavily pretreated (≥4 lines) patients with baseline platelet counts <50 × 10<sup>9</sup>/L switching after previous failure achieved PC ≥50 × 10<sup>9</sup>/L. Previous use of eltrombopag and/or romiplostim did not influence response, regardless of whether previous TPO-RA(s) succeeded or failed. Avatrombopag allowed dose-reduction/suspension of corticosteroids in 40/50 (80.0%) patients with baseline platelet counts <50 × 10<sup>9</sup>/L. Overall, 40/268 (14.9%) thrombocytosis and 12/268 (4.5%) thromboembolic events were reported. Our real-world cohort supports the use of avatrombopag to manage ITP, regardless of disease severity and treatment history.

American Journal of HematologyVol. 99(12)
Hospital Universitario La Paz (ES), University Hospital Complex Of Vigo (ES), Hospital General Universitario Gregorio Marañón (ES), Hospital Universitari i Politècnic La Fe (ES), Hospital Costa del Sol (ES), Hospital Universitario Virgen Macarena (ES), Complejo Hospitalario de Salamanca (ES), Hospital Universitario Rey Juan Carlos (ES), Hospital Universitario 12 De Octubre (ES), Hospital Sierrallana (ES), Hospital Universitario de Getafe (ES), Hospital Universitario Puerta de Hierro Majadahonda (ES), Hospital Universitario Príncipe de Asturias (ES), Hospital Universitario Miguel Servet (ES), Hospital Universitario Fundación Jiménez Díaz (ES), Mútua Terrassa (ES), Hospital Del Mar (ES), Instituto de Biomedicina de Sevilla (ES), Hospital Universitario Central de Asturias (ES), Donostiako Unibertsitate Ospitalea (ES), Hospital Universitario Lucus Augusti (ES), Hospital Universitario Son Espases (ES), Hospital Universitario Virgen del Rocío (ES), Hospital Clínico Universitario Virgen de la Victoria (ES), Hospital Universitario Infanta Leonor (ES), Instituto de Investigación Sanitaria del Principado de Asturias (ES), Universidad de Sevilla (ES)
Sociedad Española de Hematología y Hemoterapia
Good health and well-being
Openalex Percentile: Top 5%
Platelet Disorders and Treatments
4.09
DOIOpen Access PDFOpenAlex
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