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Published on: August 15, 2019
Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for
Lingling Fu1, Xi Lin1, Zhenping Chen2
1Hematology Department, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Insights
A new stepwise treatment protocol for children with severe chronic immune thrombocytopenia (ITP) shows higher sustained response rates off-treatment and lower costs compared to standard TPO-RA therapy. This individualized approach reduces the medical burden for pediatric ITP patients.
Area of Science:
- Pediatric Hematology
- Immunology
- Pharmacology
Background:
- The current international recommendation for second-line treatment of severe persistent/chronic immune thrombocytopenia (P/CITP) in children involves thrombopoietin receptor agonists (TPO-RAs).
- However, less than 30% of children achieve a sustained response off-treatment (SRoT) with TPO-RA therapy, resulting in a significant medical burden.
Purpose of the Study:
- To evaluate the efficacy of a stepwise response-guided treatment protocol compared to standard TPO-RA-based second-line therapy for pediatric severe P/CITP.
- To assess outcomes including platelet count, bleeding control, side effects, sustained response off-treatment (SRoT), and treatment costs.
Main Methods:
- A prospective, multicenter clinical cohort study was conducted with 143 children diagnosed with severe P/CITP.
- Participants were followed for 1 year.
- The study compared a stepwise protocol (dexamethasone, rituximab, TPO-RAs based on response) with a TPO-RA-based second-line treatment group.
Main Results:
- No significant differences were observed in response/remission rates or bleeding grades between the stepwise and TPO-RA groups.
- The stepwise group exhibited significantly fewer treatment-related side effects (9.0%) compared to the TPO-RA group.
- A notable 74% of patients in the stepwise group achieved SRoT, whereas none in the TPO-RA group did.
- Treatment costs were substantially lower in the stepwise group (USD 68.26/kg) versus the TPO-RA group (USD 384.76/kg) over 12 months.
Conclusions:
- The stepwise response-guided treatment protocol effectively stratifies children with severe P/CITP, allowing for individualized treatment strategies.
- This protocol demonstrates comparable efficacy and safety to TPO-RA-based therapy while significantly reducing the overall treatment burden.
- The stepwise approach is a preferable treatment option for children suffering from severe P/CITP.
Background:
The first second-line international recommendation for children with severe persistent/chronic immune thrombocytopenia is thrombopoietin receptor agonist (TPO-RA)-based treatment; however, <30% can achieve sustained response off-treatment (SRoT), leading to a heavy medical burden.
Objectives:
This study aimed to confirm the efficacy of the stepwise response-guided treatment protocol compared with TPO-RA-based second-line therapy for children with severe P/CITP.
Methods:
The stepwise response-guided treatment protocol is an individualized stratified immune thrombocytopenia treatment starting with high-dose dexamethasone, then adding rituximab and TPO-RAs in sequential order according to treatment response. A prospective, multicenter clinical cohort study enrolled severe P/CITP children with a 1-year follow-up. We compared the treatment outcome response of platelet count, bleeding control, and treatment-related side effects and cost outcomes (escalation status, SRoT, and treatment costs) between the stepwise group and the TPO-RA-based second-line treatment group (TPO-RA group).
Results:
The study enrolled 143 cases of severe P/CITP children with a 12-month follow-up period. There were no differences in baseline characteristics between the stepwise and TPO-RA groups (P > .05). Response/remission rates and bleeding grades showed no differences (P > .05), but there were fewer side effects related to treatment in the stepwise group (9.0%; P < .00). A total of 74% in the stepwise group achieved SRoT while none in the TPO-RA group did. The cost of treatment was significantly lower in the stepwise group compared with the TPO-RA group over the 12-month follow-up period (USD 68.26/kg vs USD 384.76/kg, P < .00).
Conclusion:
The stepwise response-guided treatment protocol effectively stratifies children with severe P/CITP based on treatment response, enabling individualized treatment strategies. This protocol achieves comparable efficacy and safety while reducing the treatment burden compared with TPO-RA-based second-line therapy, making it a preferable option for children with severe P/CITP.
