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Myelofibrosis (MF) is characterized by ineffective and hepatosplenic extramedullary hematopoiesis due to fibrotic changes in the bone marrow and systemic manifestations due to aberrant cytokine release. Ruxolitinib (RUX) is the first JAK1/JAK2 inhibitor that is clinically approved to treat splenomegaly by ameliorating inflammatory cytokines and myeloproliferation in MF. Patients with less advanced MF may also achieve better outcome and successful treatment with RUX. However, approximately 40% of the patients failed to achieve a stable response or have shown to be intolerant to RUX, and most of them discontinued RUX. In patients who need to discontinue or reduce the dose of RUX for any reason, RUX is known to induce a paradoxical accumulation of JAK activation loop phosphorylation that is causing RUX discontinuation syndrome (RDS). To review the topic of MF and RUX, we searched relevant literatures using PubMed. RUX treatment in lower IPSS risk patients who present with splenomegaly and disease-associated symptoms can be helpful. A careful discontinuation strategy with steroids may reduce the probability of RDS, and the recognition of RDS with early re-introduction of RUX is important in the treatment of severe cases of RDS.

Light and shade of ruxolitinib: positive role of early treatment with ruxolitinib and ruxolitinib withdrawal syndrome in patients with myelofibrosis