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

Wang, Zhifa; Wang, Limin; Liu, Yan; Meng, Jinxi; Dong, Shuyue; Ma, Jingyao; Hu, Yu; Chen, Zhenping; Cheng, Xiaoling; Wu, Runhui

doi:10.1111/bjh.18842

Cited by 8 publications

(3 citation statements)

References 25 publications

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“…Eltrombopag stimulates thrombopoiesis and is considered suitable for use in in pediatric patients [21] . In our study, 67.0% of patients achieved OR, 55.3% of patients achieved CR, 11.7% of patients achieved R, which is consistent with previously reported for children with ITP [22] .Although, our OR rate is lower than what has been reported in the children with chronic ITP [18] .During the follow-up period, we observed a considerable number of parents had high expectations for the platelet count, leading to the poor compliance. Three children discontinued the treatment at 1 week and four children at 2 weeks.…”

Section: E Cacy Of Eltrombopag Under Six Months Patientssupporting

confidence: 92%

The long-term efficacy of eltrombopag in children with immune thrombocytopenia

Yang,

Sang,

Yang

et al. 2024

Preprint

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Immune thrombocytopenia (ITP) is the most common autoimmune disorder characterized by decreased platelet counts and impaired platelet production. Eltrombopag has been demonstrated to be safe and effective for children with ITP. It is reported eltrombopag can achieve a sustained response off treatment. However, data on its overall efficacy and safety profile are scarce in children. This study aimed to investigate the long-term efficacy of eltrombopag in children with ITP. Treatment overall response (OR), complete response (CR), response (R), durable response (DR), no response (NR), treatment free remission (TFR), and relapse rate, were assessed in 103 children with ITP during eltrombopag therapy. The OR rate, CR rate, R rate, DR rate, NR rate, TFR, and relapse rate were 67.0%, 55.3%, 11.7%, 56.3%, 33.0%, 60%, 36.2%, respectively. Importantly, we discovered that newly diagnosed ITP patients showed a higher DR rate, TFR rate and lower relapse rate compared to persistent and chronic ITP patients. Furthermore, the CR rate, DR rate, and TFR rate of 5 patients under six months were 100%. None of them suffered relapse. The most common adverse event (AEs) was hepatotoxicity (7.77%). Our study highlighted the critical role of eltrombopag as the second-line treatment in children with ITP who were intolerant to first-line therapy.

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Section: E Cacy Of Eltrombopag Under Six Months Patientssupporting

confidence: 92%

The long-term efficacy of eltrombopag in children with immune thrombocytopenia

Yang,

Sang,

Yang

et al. 2024

Preprint

View full text Add to dashboard Cite

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“…However, this finding may provide an idea and direction for further exploring the sensitizer of TPO-RAs, as about 37% of patients had no response or relapse after discontinuing TPO-RAs in our previous study. 13 Sun's study found that the addition of diacerein as a sensitizer to ELT has improved the initial response for ELT-inefficient or relapsed ITP patients by increased the level of TGF-β and promoted megakaryocyte PI3K phosphorylation. 14 The previous study also showed COVID-19 significant increase in the tissue biomarkers of TGF-β in the paraffin lung samples from patients who died of COVID-19.…”

Section: Discussionmentioning

confidence: 99%

Transient increase in platelet counts associated with COVID‐19 infection during TPO‐RA as the second‐line treatment in children with ITP

Wang,

Cheng,

Wang

et al. 2023

Br J Haematol

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SummaryThe thrombopoietin receptor agonists (TPO‐RA) were recommended for primary immune thrombocytopenia (ITP) during the pandemic of COVID‐19. However, the incidence of thrombocytosis and thrombosis was sporadically reported in the chronic immune thrombocytopenia (CITP) patients receiving TPO‐RA during the COVID‐19 infection. With the local prevalence of COVID‐19 in December 2022 in the Beijing area, we got more powerful evidence about the change in platelet (Plt) counts associated with COVID‐19 infection. A single‐centre observational cohort study was performed from the beginning of December 2022 to the end of February 2023 to enrol CITP children treated with TPO‐RA alone as the second‐line treatment and suffering from the COVID‐19 infection in December 2022. The Plt counts before, during and after COVID‐19 infection were collected. In total, 67 (34 males and 33 females) patients with 8.10 (2.15, 15.70) years of age were enrolled. Sixty‐three patients who had responded to the TPO‐RA showed a transient increase in Plt counts after the infection of COVID‐19. The time of starting to increase was on Day 3 (2, 7), and to the peak level on Day 14 (7, 19) of infection with the peak Plt count was 289 (88, 1974) × 109/L. With at least 2 months observation period from COVID‐19 infection, the Plt counts of 100% (63/63) patients declined to the baseline on Day 25 (14, 41). The phenomenon of transient increase in Plt counts has been shown in the CITP children who responded to TPO‐RA when suffering from COVID‐19 infection.

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“…9 The median time to first response was >12 days in a children's eltrombopag study, 16 and the median time to first response was 16 days in our previous eltrombopag study. 17 In this study, the median time to first response (platelet count ≥30 × 10 9 /L with at least a twofold increase from baseline and no bleeding at 4 weeks) was 7 days. This study also showed that attaining the required platelet levels was slightly quicker than that observed in our previous AVA switch study, in which the median time to response was 21 days.…”

Section: Discussionmentioning

confidence: 55%

Efficacy and safety of avatrombopag in Chinese children with persistent and chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China

Wang,

Zhang,

et al. 2024

Br J Haematol

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SummaryAvatrombopag (AVA) is a novel thrombopoietin receptor agonist (TPO‐RA) that has been recently approved as a second‐line therapy for immune thrombocytopenia (ITP) in adults; however, its safety and efficacy data in children are lacking. Here, we demonstrated the efficacy and safety of AVA as second‐line therapy in children with ITP. A multicentre, retrospective, observational study was conducted in children with persistent or chronic ITP who did not respond to or relapsed from previous treatment and were treated with AVA for at least 12 weeks between August 2020 and December 2022. The outcomes were the responses (defined as achieving a platelet count ≥30 × 109/L, twofold increase in platelet count from baseline and absence of bleeding), including rapid response within 4 weeks, sustained response at weeks 12 and 24, bleeding control and adverse events (AEs). Thirty‐four (18 males) patients with a mean age of 6.3 (range: 1.9–15.3) years were enrolled. The median number of previous treatment types was four (range: 1–6), and 41.2% patients switched from other TPO‐RAs. Within 4 weeks, overall response (OR) was achieved in 79.4% patients and complete response (CR, defined as a platelet count ≥100 × 109/L and the absence of bleeding) in 67.7% patients with a median response time of 7 (range: 1–27) days. At 12 weeks, OR was achieved in 88.2%, CR in 76.5% and sustained response in 44% of patients. At 24 weeks, 22/34 (64.7%) patients who achieved a response and were followed up for 24 weeks were evaluated; 12/22 (54.55%) achieved a sustained response. During AVA therapy, median platelet counts increased by week 1 and were maintained throughout the treatment period. The proportion of patients with grade 1–3 bleeding decreased from 52.95% at baseline to 2.94% at 12 weeks, while concomitant ITP medications decreased from 36.47% at baseline to 8.82% at 12 weeks, with only 9 (26.47%) patients receiving rescue therapy 23 times within 12 weeks. There were 61.8% patients with 59 AEs: 29.8% with Common Terminology Criteria for Adverse Events grade 1 and the rest with grade 2. These findings show that AVA could achieve a rapid and sustained response in children with persistent or chronic ITP as a second‐line treatment, with good clinical bleeding control and reduction of concomitant ITP therapy, without significant AEs.

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

Cited by 8 publications

References 25 publications

The long-term efficacy of eltrombopag in children with immune thrombocytopenia

The long-term efficacy of eltrombopag in children with immune thrombocytopenia

Transient increase in platelet counts associated with COVID‐19 infection during TPO‐RA as the second‐line treatment in children with ITP

Efficacy and safety of avatrombopag in Chinese children with persistent and chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China

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