Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study

Ruan, Yongsheng; Cao, Wenming; Luo, Tingting; Li, Xuan; Liu, Qiujun; Xiao, Yao; Chen, Wu; Xie, Danfeng; Ren, Yu; Wang, Xuedong; Feng, Xi‐Qiao

doi:10.3389/fped.2023.1099372

Cited by 4 publications

(3 citation statements)

References 29 publications

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“…14 However, it is not approved in the paediatric population; therefore, the treatment of ITP with AVA has only been reported in case (series) reports in this patient population. 8,15 The results of the phase 2, 28-day, double-blind, randomised clinical trial of AVA (20 mg) in adult patients with ITP showed that 80% patients achieved a platelet count response ≥50 × 10 9 /L with a 20 × 10 9 /L increase above baseline on day 28. In a randomised phase 3 clinical trial, 63% of patients treated with AVA achieved at least one response (platelet count ≥50 000/μL) by day 8.…”

Section: Discussionmentioning

confidence: 99%

“…It has been approved by the FDA and EMA for the treatment of primary chronic ITP in adult patients and has demonstrated efficacy and safety in a previous study 14 . However, it is not approved in the paediatric population; therefore, the treatment of ITP with AVA has only been reported in case (series) reports in this patient population 8,15 …”

Section: Discussionmentioning

confidence: 99%

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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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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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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“…Another real-world study revealed the efficacy of eltrombopag in providing a rapid, sustained response that improves haematopoietic function in PGF patients [ 44 ]. Avatrombopag [ 45 , 46 ], hetrombopag [ 47 ], and romiplostim [ 48 ] have all been reported to improve platelet engraftment post-HSCT. A recent systematic review and meta-analysis has concluded that eltrombopag could provide an effective treatment option for PGF with an acceptable toxicity profile [ 49 ].…”

Section: Discussionmentioning

confidence: 99%