Hematology & Oncology Department, The Children's Hospital of Zhejiang University School of Medicine, Hangzhou, PR China.
J Oncol Pharm Pract. 2022 Jan;28(1):222-227. doi: 10.1177/10781552211020821. Epub 2021 Jun 1.
Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening syndrome characterized by excessive production of inflammatory cytokines and multiple organs injury. Ruxolitinib, an oral selective JAK1/2 inhibitor, has recently shown efficacy and safety in the treatment of secondary HLH, which may be an alternative to intensive chemotherapy.
We report a case of a 2-year-old boy who presented to our institution with recurrent fever and acute renal failure. We made the diagnosis of Epstein-Barr virus related HLH based on the HLH-2004 protocol, and gave the treatment of ruxolitinib instead of etoposide.
The patient received dexamethasone and continuous renal replacement therapy due to renal failure, but he still had fever and anuria. Given that the use of etoposide may deteriorate renal function, ruxolitinib was administered instead of etoposide. After 5 days of ruxolitinib treatment, the patient's fever was resolved and renal function also gradually recovered 14 days later.
Currently, dexamethasone, etoposide and cyclosporine A are the main drugs in HLH treatment. However, cytotoxic chemotherapy can temporally deteriorate organ damage and induce serious myelosuppression, which makes clinicians hesitate to implement these regimens. Ruxolitinib has shown efficacy in treating HLH without much toxicity in clinical trials. Thus, we suggest that ruxolitinib constitutes a treatment option for secondary HLH complicated by severe renal damage which may reduce toxic effects compared with intense chemotherapy.
噬血细胞性淋巴组织细胞增生症（HLH）是一种危及生命的综合征，其特征是炎症细胞因子过度产生和多器官损伤。鲁索利替尼（ruxolitinib）是一种口服选择性 JAK1/2 抑制剂，最近在治疗继发性 HLH 方面显示出疗效和安全性，可能是强化化疗的替代方案。
我们报告了一例 2 岁男孩，因反复发热和急性肾衰竭就诊于我院。根据 HLH-2004 方案，我们做出了 EBV 相关 HLH 的诊断，并给予 ruxolitinib 治疗，而不是依托泊苷。
由于肾衰竭，患者接受了地塞米松和持续肾脏替代治疗，但仍有发热和无尿。由于依托泊苷的使用可能会使肾功能恶化，因此给予 ruxolitinib 代替依托泊苷。在 ruxolitinib 治疗 5 天后，患者的发热得到缓解，肾功能在 14 天后逐渐恢复。
目前，地塞米松、依托泊苷和环孢素 A 是 HLH 治疗的主要药物。然而，细胞毒性化疗可能会暂时加重器官损伤并引起严重的骨髓抑制，这使得临床医生在实施这些方案时犹豫不决。鲁索利替尼在临床试验中已被证明在治疗 HLH 方面有效，且毒性较小。因此，我们建议 ruxolitinib 可作为治疗继发性 HLH 的一种选择，对于伴有严重肾损伤的患者，与强化化疗相比，可能会减少毒性作用。
