Lingling Fu1,2,3, Jie Ma1,2,3, Hao Gu1,2,3, Jingyao Ma1,2,3, Yunyun Wei1,2,3, Zhengping Chen1,2,3, Runhui Wu1,2,3. 1. Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing Key Laboratory of Pediatric Hematology Oncology, Beijing, China. 2. National Key Discipline of Pediatrics (Capital Medical University), Beijing, China. 3. Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing, China.
Abstract
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.
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 childrenSCITP 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.