对于患有严重慢性免疫性血小板减少症的儿童，采用升级治疗策略：来自单个中心的初步报告。
An escalating treatment strategy for children with severe chronic immune thrombocytopenia: Preliminary report from a single center.
机构信息
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.
National Key Discipline of Pediatrics (Capital Medical University), Beijing, China.
出版信息
Pediatr Blood Cancer. 2021 Jun;68(6):e29006. doi: 10.1002/pbc.29006. Epub 2021 Mar 15.
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 × 10 /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.
目的
分析儿童严重慢性免疫性血小板减少症（SCITP）递增治疗策略的效果。
方法
这是一项单中心回顾性队列研究。收集 2017 年 6 月至 2019 年 8 月在我院接受递增治疗策略的 SCITP 患儿的数据。递增策略包括三个步骤：步骤 I（6 个疗程的高剂量地塞米松[HDD]）、步骤 II（HDD 联合低剂量利妥昔单抗）和步骤 III（艾曲波帕）。
结果
共纳入 30 例患儿（男 18 例，女 12 例），免疫性血小板减少症（ITP）病程 20.5（12.0-96.0）个月。治疗后，缓解率为 36.7%（11/30），持续反应（SR）率为 68.2%（15/22）。从步骤 I 到步骤 III 的分布（缓解率）如下：30 例中 9 例（33.3%，3/9）；4 例（50%，2/4）；17 例（29.4%，5/17）。在艾曲波帕（步骤 III）病例中，47.5%（8/17）的血小板计数≥50×10/L，37.5%（3/8）的剂量减少，25%（2/8）的药物成功停药。12、24 和 36 个月的患者数分别为 30、7 和 2，总反应率和缓解率分别为 80%（36.7%）、57.1%（28.6%）和 50%（50%）。总复发率为 26.7%（8/30），其中来自步骤 II 的 3 例和步骤 III 的 5 例。
结论
儿童 SCITP 的递增治疗策略显示出有效的改善率，缓解率为 36.7%，SR 为 68.2%，30%可从 HDD 治疗中获益并保持 SR。艾曲波帕联合治疗可降低低剂量利妥昔单抗的复发率。
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