Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP)

Frontiers in Medicine

Lucia Dora Notarangelo¹  Andrea Pession²  Antonio Ruggiero³  Giovanna Russo⁴  Paola Saracco⁵  Marco Spinelli⁶  Alessandra Tolva⁷  Assunta Tornesello⁸  Maurizio Miano⁹  Ilaria Fotzi^1,10  Saverio Ladogana^1,11  Angelica Barone^1,12  Fiorina Giona^1,13  Simone Cesaro^1,14  Margherita Nardi^1,15  Antonio Marzollo^1,16  Giovanni Carlo Del Vecchio^1,17  Paola Giordano^1,17  Giuseppe Lassandro^1,17  Valentina Palladino^1,17 

[1] 0Hemato-Oncology Unit, Children Hospital, Spedali Civili, Brescia, Italy;1Department of Pediatrics, Sant'Orsola-Malpighi Hospital, University of Bologna, Bologna, Italy;2Pediatric Oncology Unit, Fondazione Policlinico Universitario Gemelli IRCCS, Università Cattolica del Sacro Cuore, Rome, Italy;3Pediatric Hemato-Oncology Unit, Department of Clinical and Experimental Medicine, University of Catania, Catania, Italy;4Pediatric Hematology, Department of Pediatrics, University Hospital Città della Salute e della Scienza, Turin, Italy;5Hemato-Oncology Unit, Fondazione MBBM, San Gerardo Hospital, Monza, Italy;6Pediatric Hematology/Oncology, IRCCS Policlinico San Matteo Foundation, University of Pavia, Pavia, Italy;7Pediatric Hematology Oncology, Presidio Ospedaliero Vito Fazzi, Lecce, Italy;Clinical and Experimental Hematology Unit, “G. Gaslini” Children's Hospital, Genoa, Italy;Department Pediatric Hematology Oncology, Azienda Ospedaliero Universitaria A. Meyer Children Hospital, Florence, Italy;Department of Hematology, IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy;Department of Pediatric Onco-Hematology, University Hospital of Parma, Parma, Italy;Department of Translational and Precision Medicine, Sapienza University of Rome, Rome, Italy;Pediatric Hematology Oncology, Department of Mother and Child, Azienda Ospedaliera Universitaria Integrata, Verona, Italy;Pediatric Hematology, Azienda Ospedaliero Universitaria Pisana, Pisa, Italy;Pediatric Hematology-Oncology Unit, Department of Women's and Children's Health, Azienda Ospedaliera-University of Padova, Padua, Italy;Pediatric Unit, Department of Biomedical Science and Human Oncology, University of Bari “Aldo Moro”, Bari, Italy;

关键词: eltrombopag;    children;    immune thrombocytopenia;    thrombopoietin receptor agonists;    bleeding disorders;   

DOI  :  10.3389/fmed.2020.00066

来源: DOAJ

【 摘 要 】

Background: The thrombopoietin receptor agonist eltrombopag has been shown to be safe and effective for children with chronic immune thrombocytopenia (ITP). The aim of the present study was to characterize eltrombopag use in current clinical practice.Material and Methods: This is a retrospective multicenter study conducted in 17 centers affiliated to the Italian Association of Pediatric Hematology and Oncology (AIEOP). The primary objective of the study was to determine the prevalence of eltrombopag use in Italian children affected by chronic ITP, after EMA authorization for pediatric age. The secondary objective was to assess efficacy in the first 6 months and safety during the whole period of eltrombopag treatment in current clinical practice. A total of 386 children with chronic ITP were retrospectively enrolled and eligible for analysis. Among these patients, 71 received eltrombopag.Results: The prevalence of eltrombopag use was 19% (95% CI 0.15–0.23). Thirty-one patients (44%) were male and 40 patients (56%) were female. The median age at the first dose of eltrombopag was 12 years (3–17 years). The median duration of eltrombopag treatment was 11 months (1–32 months) and the median starting dose was 50 mg/day (12, 5–75 mg/day). Thirty-two patients (45%) required one or more concomitant ITP medications during the first 6 months of treatment with eltrombopag. Thirty-nine patients (55%) never required concomitant medications. Median platelet counts and proportion of patients achieving the target platelet count of at least 30 × 109/L and 100 × 109/L significantly increased during the first 6 months of treatment (p < 0.0001). Additionally, eltrombopag has been proved effective in the absence of concomitant therapies. The most common Adverse Events were headache (7%) and thrombocytosis (6%).Conclusion: Our study highlighted the crucial role of eltrombopag as second line treatment in children with chronic ITP.

【 授权许可】

Unknown