利妥昔单抗治疗儿童重症慢性免疫性血小板减少症2例并文献复习

Rituximab Treatment for Severe Chronic Immune Thrombocytopenia in Children: A Report of 2 Cases and Literature Review

摘要: 目的：探讨利妥昔单抗(rituximab, RTX)治疗儿童重症慢性免疫性血小板减少症(chronic immune thrombocytopenia, CITP)的疗效及安全性。方法：回顾分析2020~2023年间青岛大学附属医院儿童血液肿瘤科收治的2例重症慢性ITP患儿利妥昔单抗治疗的临床资料，并文献复习。结果：病例1，男性患儿，13岁，病史2年7月，反复皮肤粘膜出血点，严重时伴有血尿，骨髓巨核细胞数正常，成熟障碍；病例2，女性患儿，15岁，病史3年，反复皮肤粘膜出血点瘀斑、月经量大，病程中出现血尿、血便、卵巢出血，失血性贫血，骨髓增生活跃，巨核细胞明显增多，成熟障碍。均排除其他疾病，血小板反复低于10 × 109/L，诊断为重症慢性ITP，反复糖皮质激素、丙种球蛋白以及联合艾曲波帕治疗，均不能持续有效。病例1应用血浆置换及RTX治疗，持续有效超过6个月；病例2应用RTX治疗，达完全反应8个月，持续有效超过2年2月。均无严重不良反应及严重感染发生。结论：重症慢性ITP患儿应用利妥昔单抗治疗是安全有效的。

Abstract: Objective: This study aims to explore the effectiveness and safety of rituximab (RTX) treatment in children with severe chronic immune thrombocytopenia (CITP). Methods: We retrospectively ana-lyzed clinical data from two cases treated with rituximab in the Pediatric Hematology-Oncology De-partment at Qingdao University Affiliated Hospital between 2020 and 2023, and conducted a liter-ature review. Results: Case 1 involves a 13-year-old boy with a medical history of 2 years and 7 months. He had recurring bleeding spots on the skin and mucous membranes, sometimes accom-panied by blood in the urine. The bone marrow showed a normal number of megakaryocytes but with maturation issues. Case 2 features a 15-year-old girl with a medical history of 3 years. She ex-perienced recurring bleeding spots, ecchymosis, and heavy menstrual bleeding. During the course of the disease, she had episodes of hematuria, bloody stools, and ovarian bleeding, leading to hem-orrhagic anemia. Increased bone marrow activity and significant megakaryocyte proliferation with maturation issues were observed in her case. Other diseases were ruled out, both cases had platelet counts repeatedly lower than 10 × 109/L, leading to a diagnosis of severe chronic ITP. Despite re-peated treatment with corticosteroids, intravenous immunoglobulin, and combination therapy with eltrombopag, no sustained improvement was achieved. In Case 1, treatment with plasma exchange and RTX led to sustained effectiveness for over 6 months. In Case 2, RTX treatment achieved com-plete response within 8 months and remained effective for over 2 years and 2 months. No severe adverse reactions or infections occurred in either case. Conclusion: The use of rituximab in children with severe chronic ITP proves to be a safe and effective approach.