Sustained response off treatment in eltrombopag for children with persistent/chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China.

Abstract

Eltrombopag (ELT) is effective and safe in adult persistent/chronic immune thrombocytopenia (p/cITP); a proportion could achieve a sustained response off treatment (SRoT); however, data on children are lacking. We attempted to analyse SRoT of ELT in children with p/cITP in this study. A multicentre retrospective observational study was performed in November 2022 for children with p/cITP who used ELT alone for >2 months between January 2017 and November 2021. Clinical data of pre-, during and post-ELT were collected. SRoT was defined as maintaining a platelet count of ≥30 × 109 /L without rescue therapy for at least 6 months off ELT. There were 143 patients enrolled; 69.2% (99/143) achieved an overall response of 43.3% and 25.9% achieved complete response (CR) and response (R). Among the 35 patients analysed from whom ELT was withdrawn, 71.4% (25/35) showed SRoT after discontinuing ELT without additional ITP therapy, with a median follow-up of 0.94 (range, 0.53-3.8) years, equal to 17.5% (25/143) in all patients treated with ELT. Compared with the patients with relapse (n = 10), the SRoT patients (n = 25) had a higher rate of CR (80% [20/25] vs. 40% [4/10]), shorter interval time from initiation to taper (6.4 months vs. 9.4 months), longer time from taper to withdrawal (1.1 years vs. 0.3 years) and a longer duration of ELT treatment (1.6 years vs. 0.5 years) with p < 0.05. Patients who achieved CR could attain SRoT more easily (p = 0.02). ELT had a response in 69.2% of children with p/cITP and 17.5% of them attained SRoT with good tolerance. The patients who achieved CR and began ELT treatment as early as possible, with a longer treatment duration and slower tapering, had a higher probability of SRoT.

• Authors: Zhifa Wang, Lijuan Wang, Yan Liu, Jinxi Meng, Shuyue Dong, Jingyao Ma, Yu Hu, Zhenping Chen, Xiaoling Cheng, Runhui Wu
• Journal: British journal of haematology (Br J Haematol) Vol. 202 Issue 2 Pg. 422-428 (07 2023) ISSN: 1365-2141 [Electronic] England
• PMID: 37128832 (Publication Type: Observational Study, Multicenter Study, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2023 British Society for Haematology and John Wiley & Sons Ltd.

Chemical References

• eltrombopag
• Receptors, Thrombopoietin
• Benzoates
• Hydrazines

Topics

• Adult
• Humans
• Child
• Purpura, Thrombocytopenic, Idiopathic (drug therapy, chemically induced)
• Retrospective Studies
• Treatment Outcome
• Receptors, Thrombopoietin
• Benzoates
• Hydrazines
• China