Nationwide survey of ruxolitinib administration for Japanese children with hemophagocytic lymphohistiocytosis

Le résumé fourni par la source

In the treatment of hemophagocytic lymphohistiocytosis (HLH), the effectiveness of ruxolitinib has been reported internationally. However, in Japan, ruxolitinib use has been limited due to its off-label status. We conducted a nationwide, multicenter, retrospective survey in Japan on the use of ruxolitinib in pediatric HLH. Seventeen patients from nine institutions were included: primary HLH (n = 5), Epstein–Barr virus-associated HLH (EBV-HLH; n = 3), and post-transplant HLH (PT-HLH; n = 9). Ruxolitinib was used for refractory disease (n = 14) as well as for maintenance therapy in primary HLH/EBV-HLH (n = 3). In most patients, ruxolitinib was employed in combination with conventional therapies. Among refractory cases, the overall response rate was 80% for primary HLH/EBV-HLH and 89% for PT-HLH. The three patients who received ruxolitinib as maintenance therapy achieved sustained remission: two remained in remission until subsequent transplantation, and one did not require transplantation. Considering the results, we propose that ruxolitinib is a promising therapeutic option for pediatric HLH.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.