Prospective trial evaluates ruxolitinib safety and efficacy in children with hemophagocytic lymphohistiocytosis, suggesting benefit for newly diagnosed cases.
Key Points
This trial aims to evaluate the safety and effectiveness of ruxolitinib in children with hemophagocytic lymphohistiocytosis (HLH).
Prospective multi-institutional trial
Patients received ruxolitinib 25 mg/m2 twice daily combined with dexamethasone, for eight weeks.
Pharmacokinetic studies conducted on days one and eight.
All eight patients demonstrated clinical stabilization and improvement in serum ferritin levels after one week.
Five patients with newly diagnosed HLH achieved a complete response after eight weeks and were alive at one year.
One patient with relapsed/refractory HLH also achieved a complete response, while two with relapsed/refractory pHLH unfortunately died.