Ruxolitinib Might Be an Effective Hormone Reduction and Replacement Drug in Children With Secondary Hemophagocytic Lymphohistiocytosis 

Abstract

Background: To analyze the effects of ruxolitinib on children with secondary hemophagocytic lymphohistiocytosis (HLH).Methods: Eleven pediatric patients diagnosed with HLH and treated with ruxolitinib (ruxolitinib group: group R) between November 2017 and August 2018 were retrospectively analyzed. Eleven age-matched pediatric patients with HLH undergoing conventional treatment (control group: group C) were also analyzed.Results: In group R, three patients who did not respond to conventional treatment (dexamethasone and etoposide) were treated with Ruxolitinib and their temperature decreased to normal levels. Four patients had normal temperature after conventional treatment, but they had severe organ involvement, including obvious yellowing of the skin, increased liver enzyme levels and neuropsychiatric symptoms, and they were all ameliorated with ruxolitinib treatment. Four patients were relieved with ruxolitinib therapy alone. In group C, the body temperature of eleven patients all decreased to normal levels after conventional treatment. The body temperature of group R patients decreased to normal levels more rapidly than that of group C patients. The hormone dosage in group R was significantly lower than that in group C. Both groups were followed up for 2–2.5 years. No obvious adverse drug reactions of ruxolitinib were observed during treatment and follow-up.Conclusion: Ruxolitinib might be an effective drug in controlling body temperature and reducing inflammation indicators. It might be a potential replacement for hormone therapy for HLH treatment in children, thereby reducing or avoiding hormone-related adverse reactions.