An escalating treatment strategy for children with severe chronic immune thrombocytopenia: Preliminary report from a single center
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Lingling Fu1,2,3, Jie Ma1,2,3, Hao Gu1,2,3
1Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing Key Laboratory of Pediatric Hematology Oncology, Beijing, China.
Insights
An escalating treatment strategy for severe chronic immune thrombocytopenia (SCITP) in children demonstrated effectiveness, with a 36.7% remission rate and 68.2% sustained response. Eltrombopag combined with rituximab helped reduce relapse rates.
Area of Science:
- Pediatric Hematology
- Immunology
- Pharmacology
Background:
- Severe chronic immune thrombocytopenia (SCITP) presents a significant challenge in pediatric care.
- Escalating treatment strategies are being explored to improve outcomes for children with SCITP.
Purpose of the Study:
- To evaluate the efficacy of a three-step escalating treatment strategy for SCITP in children.
- To analyze remission and sustained response rates at different treatment stages.
Main Methods:
- A retrospective cohort study was conducted at a single center.
- Data from 30 children with SCITP treated between June 2017 and August 2019 were analyzed.
- The escalating strategy involved high-dose dexamethasone (HDD), followed by HDD with rituximab, and finally eltrombopag.
Main Results:
- The overall remission rate was 36.7% (11/30), with a sustained response rate of 68.2% (15/22).
- Step I (HDD) showed a 33.3% remission rate.
- Step III (eltrombopag) resulted in 47.5% of patients maintaining a platelet count ≥50 × 10^9/L, with 25% discontinuing medication.
- The total relapse rate was 26.7%.
Conclusions:
- The escalating treatment strategy effectively improved outcomes in children with SCITP.
- Approximately 30% of patients benefit from and maintain sustained response with high-dose dexamethasone.
- Combining eltrombopag can decrease relapse rates associated with low-dose rituximab treatment.
Objective:
To analyze the effects of escalating treatment strategy in children with severe chronic immune thrombocytopenia (SCITP).
Methods:
This was a single-center, retrospective cohort study. Data from children with SCITP who received escalating treatment strategy in our center were collected between June 2017 and August 2019. The escalating strategy included three steps: Step I (six courses of high-dose dexamethasone [HDD]), Step II (HDD combined with low-dose rituximab), and Step III (eltrombopag).
Results:
A total of 30 cases (18 males and 12 females) were included, with duration of immune thrombocytopenia (ITP) of 20.5 (12.0-96.0) months. After treatment, the remission rate was 36.7% (11/30) and the sustained response (SR) rate was 68.2% (15/22). The distribution (remission rates) from Step I to III was as follows: nine of 30 (33.3%, 3/9); four of 30 (50%, 2/4); 17/30 (29.4%, 5/17), respectively. In eltrombopag (Step III) cases, 47.5% (8/17) maintained a platelet count of ≥50 × 109 /L, 37.5% (3/8) had dose tapering, and 25% (2/8) have successfully discontinued the medication. The number of patients at 12, 24, and 36 months were 30, seven, and two, with a total response and remission rates of 80% (36.7%), 57.1% (28.6%), and 50% (50%), respectively. The total relapse rate was 26.7% (8/30), and three cases from Step II and five cases from Step III.
Conclusion:
The escalating strategy for children SCITP showed an effective improvement rate with 36.7% remission and 68.2% SR, and 30% could benefit and retain SR from HDD treatment. Combined treatment with eltrombopag can reduce the relapse rate of low-dose rituximab.
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