ABSTRACT

Rationale and Objectives Current therapies for Hemophagocytic Lymphohistiocytosis (HLH) have limited success and are associated with side effects such as serious myelosuppression, immunosuppression, and the risk for secondary malignancy. Given the mechanism of action and safety profile of ruxolitinib, this systematic review assessed its efficacy and safety as a potential treatment for newly diagnosed and relapsed/refractory HLH.

Research Design and Methodology

A comprehensive search of electronic databases was done to identify relevant prospective and retrospective studies. The quality of the evidence was assessed using MINORS for non-RCT studies.

Results

Ten articles were selected for full-text review: six prospective, two retrospective, and two case-control studies. Data threshold was not reached for meta-analysis. The studies showed a moderate risk of bias. Ruxolitinib, either alone or in combination with chemotherapies, exhibited an overall response rate (ORR) ranging from 69% to 100%, with an average of 85%. Complete remission (CR) rates ranged from 15% to 83%, with an average of 45%. Clinical and laboratory improvements were observed within the first week of treatment, although the duration of response varied significantly, ranging from three days to almost nine months. Regarding safety, ruxolitinib demonstrated manageable adverse events (AE), with hematologic and gastrointestinal events being the most common.

Discussion and Conclusion

Overall, the available evidence indicates that ruxolitinib is a potential treatment option with a satisfactory safety profile for newly diagnosed and relapsed/refractory HLH patients. However, the findings should be interpreted cautiously, with randomized studies needed to further elucidate the role of ruxolitinib in HLH treatment.