Ruxolitinib is an alternative to etoposide for patient with hemophagocytic lymphohistiocytosis complicated by acute renal injury: A case report.

INTRODUCTION: 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. CASE REPORT: 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. MANAGEMENT AND OUTCOME: 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. DISCUSSION: 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.

Ruxolitinib Treatment of Steroid-Refractory Graft-versus-Host Disease in Children: A Case Series and Review of the Literature.

BACKGROUND: Ruxolitinib has been increasingly used in the treatment of steroid-refractory graft-versus-host disease (SR-GVHD) in allogeneic hematopoietic stem cell transplantation (allo-HSCT) patients. However, there are limited data on the use of ruxolitinib in children. OBJECTIVE: This study aimed to assess the efficacy and toxicity of ruxolitinib in the treatment of SR-GVHD in children. PATIENTS AND METHODS: Data of patients who suffered from SR-GVHD after allo-HSCT and received ruxolitinib treatment between June 2018 and December 2020 at our center were analyzed retrospectively. The characteristics of patients, the dosage of ruxolitinib, the response, toxicity, and the survival data were collected. RESULTS: A total of 14 pediatric patients were diagnosed with SR-GVHD after allo-HSCT and received ruxolitinib. The age of the patients ranged from 3 months to 12 years old. The dosage of ruxolitinib ranged from 2.5 mg twice daily to 7.5 mg twice daily, mainly according to patient weight. The total overall response rate (ORR) was 64.3% (9/14), with 63.6% (7/11) in aGVHD and 67% (2/3) in cGVHD. Of the 14 patients, adverse effects were observed in 9 patients (64.3%), including cytopenia, infection, and elevated alanine aminotransferase. In addition, seven reports on the treatment of SR-GVHD in children with ruxolitinib were included for systematic analysis, with the ORR ranging from 45 to 87% in aGVHD and 70-91% in cGVHD. CONCLUSION: Given its effectiveness and safety, ruxolitinib could be used to treat SR-GVHD in children after HSCT.