Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from China
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. Obj...
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Elsevier
2025-02-01
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| Series: | Research and Practice in Thrombosis and Haemostasis |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2475037925000263 |
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| author | Lingling Fu Xi Lin Zhenping Chen Zhifa Wang Yan Liu Lijuan Wang Yu Hu Jingyao Ma Nan Wang Xiaoling Cheng Jie Ma Runhui Wu |
| author_facet | Lingling Fu Xi Lin Zhenping Chen Zhifa Wang Yan Liu Lijuan Wang Yu Hu Jingyao Ma Nan Wang Xiaoling Cheng Jie Ma Runhui Wu |
| author_sort | Lingling Fu |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-8e88e99cb6bb4d0c89091a90312c49bf |
| institution | DOAJ |
| issn | 2475-0379 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Research and Practice in Thrombosis and Haemostasis |
| spelling | doaj-art-8e88e99cb6bb4d0c89091a90312c49bf2025-08-20T03:18:58ZengElsevierResearch and Practice in Thrombosis and Haemostasis2475-03792025-02-019210270210.1016/j.rpth.2025.102702Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from ChinaLingling Fu0Xi Lin1Zhenping Chen2Zhifa Wang3Yan Liu4Lijuan Wang5Yu Hu6Jingyao Ma7Nan Wang8Xiaoling Cheng9Jie Ma10Runhui Wu11Hematology Department, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaHematology Department, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaDepartment of Clinical Laboratory Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaHematology Department, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaHematology Department, Baoding Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaHematology Department, Henan Province Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaHematology Department, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaHematology Department, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaPharmacology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaPharmacology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaHematology Department, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, ChinaHematology Department, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, China; Correspondence Runhui Wu, Hematology Department, Beijing Children's Hospital, Affiliated to Capital Medical University, Beijing, China.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.http://www.sciencedirect.com/science/article/pii/S2475037925000263childrenpersistent/chronic immune thrombocytopeniathrombopoietin receptor agonistssecond-line therapystepwise treatment |
| spellingShingle | Lingling Fu Xi Lin Zhenping Chen Zhifa Wang Yan Liu Lijuan Wang Yu Hu Jingyao Ma Nan Wang Xiaoling Cheng Jie Ma Runhui Wu Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from China Research and Practice in Thrombosis and Haemostasis children persistent/chronic immune thrombocytopenia thrombopoietin receptor agonists second-line therapy stepwise treatment |
| title | Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from China |
| title_full | Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from China |
| title_fullStr | Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from China |
| title_full_unstemmed | Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from China |
| title_short | Stepwise response-guided treatment protocol superior to thrombopoietin receptor agonist-based second-line therapy for severe persistent/chronic immune thrombocytopenia: a multicenter prospective study from China |
| title_sort | stepwise response guided treatment protocol superior to thrombopoietin receptor agonist based second line therapy for severe persistent chronic immune thrombocytopenia a multicenter prospective study from china |
| topic | children persistent/chronic immune thrombocytopenia thrombopoietin receptor agonists second-line therapy stepwise treatment |
| url | http://www.sciencedirect.com/science/article/pii/S2475037925000263 |
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