Guo Shu-Yi, Han Jun-Yan, Qiu Kun-Yin, Wang Jian, Fang Jian-Pei, Zhou Dun-Hua
Department of Hematology/Oncology, Children's Medical Center, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China.
Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, People's Republic of China.
Hematology. 2025 Dec;30(1):2526124. doi: 10.1080/16078454.2025.2526124. Epub 2025 Aug 4.
To retrospectively evaluate the efficacy and safety of ruxolitinib, with or without intensive chemotherapy, in pediatric patients with hemophagocytic syndrome. Pediatric patients with newly diagnosed hemophagocytic lymphohistiocytosis treated with ruxolitinib between January 2021 and December 2024 were analyzed. A total of 34 patients were included, with a median age of 45.6 months. The median ruxolitinib treatment duration was 62 days, with a median daily dosage of 0.63 mg/kg. Twenty-four patients received VP-16, while ten did not. Significant differences were observed in hemoglobin, sCD25, LDH, and ALT levels between the groups. The overall response rates at weeks 2, 4, and 8 were 73.5%, 76.4%, and 70.6% respectively. Platelets, ferritin, triglycerides, IL-6, IL-10, TNF-α, and liver function markers also changed significantly. The median follow-up was 502 days. Seven patients died, with a two-year overall survival rate of 78.3%, higher in the non-VP-16 group (90.0%) than the VP-16 group (73.2%). The two-year event-free survival rate was 63.2%, with 80.0% in the non-VP-16 group and 58.3% in the VP-16 group. Poor prognostic factors included high IL-6 levels, lack of response to initial ruxolitinib, and bone marrow hemophagocytic cells. The VP-16 dosage was reduced compared to HLH-1994, and no patients discontinued ruxolitinib due to side effects. Ruxolitinib is effective and safe for pediatric hemophagocytic syndrome, potentially reducing the need for etoposide. Its initial treatment response can serve as an important factor for prognostic analysis.
回顾性评估芦可替尼联合或不联合强化化疗在小儿噬血细胞综合征患者中的疗效和安全性。分析了2021年1月至2024年12月期间接受芦可替尼治疗的新诊断噬血细胞性淋巴组织细胞增生症的小儿患者。共纳入34例患者，中位年龄为45.6个月。芦可替尼的中位治疗持续时间为62天，中位日剂量为0.63mg/kg。24例患者接受了依托泊苷，10例未接受。两组之间在血红蛋白、可溶性CD25、乳酸脱氢酶和谷丙转氨酶水平上观察到显著差异。第2、4和8周的总体缓解率分别为73.5%、76.4%和70.6%。血小板、铁蛋白、甘油三酯、白细胞介素-6、白细胞介素-10、肿瘤坏死因子-α和肝功能指标也有显著变化。中位随访时间为502天。7例患者死亡，两年总生存率为78.3%，非依托泊苷组（90.0%）高于依托泊苷组（73.2%）。两年无事件生存率为63.2%，非依托泊苷组为80.0%，依托泊苷组为58.3%。不良预后因素包括白细胞介素-6水平高、对初始芦可替尼无反应和骨髓噬血细胞。与HLH-1994相比，依托泊苷剂量减少，且无患者因副作用停用芦可替尼。芦可替尼对小儿噬血细胞综合征有效且安全，可能减少对依托泊苷的需求。其初始治疗反应可作为预后分析的重要因素。
