Jakafi for HLH
Hemophagocytic lymphohistiocytosis (HLH) in adults is a clinical challenge due to its rapid progress and high mortality. The current first-line treatment regimen such as HLH-94 or HLH-2004 are largely extrapolated from pediatric HLH; however, the prognosis of adult HLH is worse than that of children. Ruxolitinib, an oral JAK inhibitor, has shown favorable efficacy and safety in murine models and clinical trials in adult HLH. Moreover, ruxolitinib can reverse the resistance of CD8 positive T cells to dexamethasone in hypercytokinemia of HLH. Multiple small studies and case series have been published.
Keenan C, Nichols KE, Albeituni S. Use of the JAK Inhibitor Ruxolitinib in the Treatment of Hemophagocytic Lymphohistiocytosis. Front Immunol. 2021 Feb 16;12:614704.
Yue Song et al, Dose-escalating ruxolitinib for refractory hemophagocytic lymphohistiocytosis. Front. Immunol., 29 June 2023
De Zhou, Xianbo Huang, Xueying Li, Lixia Zhu, Mixue Xie, Xin Huang, Fang Yu, Juying Wei, Liya Ma, Xiudi Yang, Jianai Sun, Li Li, Wanzhuo Xie, Hongyan Tong, Jie Jin, Honghu Zhu, Xiujin Ye, Ruxolitinib Combined with Dexamethasone in Adult Patients with Newly Diagnosed Hemophagocytic Lymphohistiocytosis: A Prospective, Single-Center, Single-Arm, Phase 2 Clinical Trial. Blood (2022) 140 (Supplement 1): 2620–2621.