Rituximab-first versus eltrombopag monotherapy in pediatric persistent/chronic immune refractory thrombocytopenia...

Immune thrombocytopenia (ITP) in children is an autoimmune disorder in which a significant subset develops persistent or chronic disease requiring second-line therapies, yet the optimal sequencing of agents such as rituximab (RTX) and eltrombopag (ELT) to achieve sustained remission off-treatment remains (SRoT) undefined.

This study assessed whether RTX-first sequential therapy outperforms upfront ELT monotherapy in achieving durable remission for pediatric persistent/chronic ITP (p/cITP) after first-line failure.

This nonrandomized multicenter prospective cohort study enrolled 55 children (April 2020 to July 2023) with refractory p/cITP until first-line therapy. They were assigned to RTX-first (RfG, n = 35: single RTX dose, then ELT for nonresponders) or ELT monotherapy (ELT-first group [EfG], n = 20) groups, with 12-month follow-up. Primary endpoints were SRoT and treatment duration (TD), and secondary endpoints were response rate and safety. The study is registered at ChiCTR2100048162.

Among the 55 enrolled children (RfG = 35, EfG = 20), RfG had superior SRoT (30%; 95% CI, 15.6%-48.7%) than ELT-monotherapy group (0%; 95% CI, 0%-18.7%; P = .016), although 12-month remission rates were comparable (84.8% vs 87.5%; P = .804). RfG had a shorter TD (median 18 vs 34 weeks; P = .009), even in severe cases (baseline platelets 20 ×10⁹/L vs 29 ×10⁹/L; P = .034). Early ELT (≤96 days) in RfG nonresponders improved outcomes (P = .024). Both treatments had favorable safety (grade 1-2 adverse events, <15%).

RTX-first treatment yields a better SRoT and shorter TD than ELT monotherapy for pediatric p/cITP, with comparable remission and safety.