Ruxolitinib ( Jakafa) for hemaphagocytic lymphocytosis – pro
The pathogenesis and cellular origin of Langerhans cell histiocytosis (LCH) are debated. Recently, mutations on MAPK and PI3K pathways have been linked to disrupted cell proliferation in LCH At present, there is no unanimously recommended salvage HLH treatment regimen, and there are few case reports or clinical reports with small sample sizes concerning salvage therapy after first line treatment failure . Janus Kinase 2 (JAK2) mutations play the same role in Philadelphia-negative chronic myeloproliferative neoplasms. Hence, ruxolitinb has been tried for JAK-2+ cases.
A recent sustematic review by Shazad et al concluded: “Ruxolitinib appears to have superior survival and reduced HLH symptoms compared to standard treatments alone, with an acceptable safety profile. These findings support further investigation and potential inclusion of Ruxolitinib in HLH treatment protocols to enhance patient outcomes and quality of life. Future studies should focus on long-term effects and optimal dosing strategies.”
Moazzam Shahzad,et al, Efficacy of Ruxolitinib in Patients with Hemophagocytic Lymphohistiocytosis: A Systematic Review. Blood (2024) 144 (Supplement 1): 5372.
Bonometti A, Bagnoli F, Fanoni D, Venegoni L, Corti L, Bianchi P, Elli EM, Isimbaldi G, L’Imperio V, Nazzaro G, Passoni E, Berti E. JAK2-mutated Langerhans cell histiocytosis associated with primary myelofibrosis treated with ruxolitinib. Hum Pathol. 2018 Mar;73:171-175. doi: 10.1016/j.humpath.2017.10.017. Epub 2017 Oct 28. PMID: 29107666.
Wang J, Wang Y, Wu L, Wang X, Jin Z, Gao Z, Wang Z. Ruxolitinib for refractory/relapsed hemophagocytic lymphohistiocytosis. Haematologica. 2020 May;105(5):e210-e212. doi: 10.3324/haematol.2019.222471. Epub 2019 Sep 12. PMID: 31515353; PMCID: PMC7193462.