Background: The first international recommendations for second-line therapy in children with severe persistent/chronic thrombocytopenia (SP/CITP) are thrombopoietin receptor agonists (TRAs) or TRAs-based strategies, however, only 30% of them could achieve sustain response off treatment (SRoT), leading to a heavy medical burden, especially for the SP/CITP children in developing countries. The result of our previous escalating treatment pilot study showed the possibility to solve this problem.

Objective: Comparing the efficacy of the escalating treatment to TRAs-based second-line treatment, to provide a better treatment option for children SP/CITP.

Methods: A prospective, multicenter clinical cohort study enrolled SP/CITP children, with one-year follow-up, we compared the escalating group to the group using TRAs-based second-line treatment directly, for the treatment outcomes (response/remission of platelet count, bleeding control, side effects related to treatment) and costs outcomes (escalation status, SRoT and treatment costs).

Results: A total of 143 analyzable cases were enrolled (72 males, 71 females), with 110 cases in the escalating group (EG) and 33 patients in the TRAs-based second-line treatment group (TG) (12 cases using TRAs monotherapy and 21cases using TRAs-based combination therapies). There were without differences in baseline characteristics (Ps>0.05).

Comparison between EG and TG showed that without differences in duration response(DR), remission(R) rates and also the grades of bleeding at 6 and 12 months (Ps>0.05), but with fewer side effects related to treatment (9.0% in EG and 39.4% in TG, P＜0.001).

In EG, with 57% responding to HDD and 26% to RTX, only 16% of them needed to escalate to add TRAs, leading to 74.5% achieving SRoT within 150（75-180）treatment days whereas 0% in TG. The cost of treatment was significantly lower in EG ($68.26/kg/year) than either the monotherapy group ($395.11/kg/year) or the combination therapies group ($373.85/kg/year) from TG during 12 months of follow-up period (P<0.001).

Conclusion: The escalating strategy achieved comparable clinical efficacy with fewer treatment-related side effects to TRA-based second-line treatment. By utilizing individualized and stratified treatment, the escalating strategy could shorten the treatment duration, attain treatment discontinuation, and save treatment costs significantly. It is a better option second-line treatment strategy for children SP/CITP.

Disclosures

No relevant conflicts of interest to declare.

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2024
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