Outcomes of switching to avatrombopag following treatment failure with eltrombopag in paediatric immune thrombocytopenia: A real‐world study in China

Abstract

Primary immune thrombocytopenia (ITP) is an autoimmune condition characterized by immune-mediated platelet destruction combined with impaired platelet production in the bone marrow that results in thrombocytopenia and a bleeding tendency. 1,2 Primary ITP is defined as a platelet (PLT) count <100 × 10 9 /L in the absence of underlying causes, with an estimated incidence of 4.2 per 100 000 person-years 2-4 ; and is the most common cause of thrombocytopenia in children. ITP is mostly a selflimiting process for most of paediatric patients; 5,6 hence, a watch-and-wait policy is generally recommended for incident paediatric cases of ITP. However, it may become chronic in up to 20% of patients. [7][8][9] Paediatric chronic ITP is commonly associated with limited activities and fear of bleeding that can obviously affect the quality of life. 10

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

“…In the adult phase 3 AVA study, 50% of patients maintained a platelet response for >12 weeks. In our previous study, 8 5/11 patients maintained treatment for >6 months after achieving a response. Herein, we also found that 15/34 and 12/22 patients maintained a sustained response at 12 weeks and 6 months, respectively.…”

“…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. 8 This may be related to changing from other ineffective TPO-RAs to AVA in the switch 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 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.…”

Knowledge map of thrombopoietin receptor agonists: A bibliometric analysis

“…The core position of ITP identified in TPO-RAs research also follows the fact that almost each TPO-RA has been extensively examined in various clinical trials, prospective or retrospective studies for the efficacy and safety in ITP patients from different countries and age groups [ 12 , 13 , 17 , [29] , [30] , [31] , [32] ]. Additionally, there are emerging studies comparing the efficacy and safety between TPO-RAs in adult and paediatric ITP patients [ 33 , 34 ].…”

Update on the Use of Thrombopoietin-Receptor Agonists in Pediatrics

“…Notably, treatment was terminated in 7 out of 11 patients within 3 to 6 months after switching to avatrombopag. 86 Importantly, recent studies generally indicate that pharmacologic differences among TPO-RA therapies may have real-life effects on the efficacy of agents in the individual patient. 12,74,83,84 In addition, the pharmacokinetics of different TPO-RAs vary considerably, particularly in terms of maximum concentration (C max ), area under the curve (AUC oinf ), and time to maximum concentration (T max ).…”