Ruxolitinib Rechallenge Can Improve Constitutional Symptoms and Splenomegaly in Patients with Myelofibrosis: A Case Series

Publication date: Available online 28 June 2018Source: Clinical Lymphoma Myeloma and LeukemiaAuthor(s): Aaron Gerds, Derrick Su, Anastasia Martynova, Benjamin Pannell, Sudipto Mukherrjee, Caitlin O’Neill, Mikkael Sekeres, Casey O’ConnellAbstractMyelofibrosis (MF) is one of the classic myeloproliferative neoplasms (MPNs) and can occur de novo or following transformation from polycythemia vera (PPV MF) or essential thrombocythemia (PET MF). It can be associated with constitutional symptoms and splenomegaly, both of which can greatly affect quality of life. The only curative option for MF is allogeneic stem cell transplantation. Studies have shown JAK2 inhibitors such as ruxolitinib are effective in reducing both splenomegaly and symptom burden. Although there is no approved treatment for patients who progress on ruxolitinib, anecdotal evidence suggests patients may respond to a re-challenge of ruxolitinib after drug cessation. We conducted a multi-institutional, retrospective case series to study patients who were re-challenged with ruxolitinib after inadequate response to or loss of response with an initial treatment course. Thirteen patients were identified. Six patients had primary MF, 3 patients had PPV MF, and 4 patients had PET MF. Ten patients were JAK2-positive, 2 were CALR-positive and one patient had neither mutation. Nine patients received one ruxolitinib re-challenge and four received two re-challenges. Response was defined as improvement in constitutional sympt...
Source: Clinical Lymphoma Myeloma and Leukemia - Category: Cancer & Oncology Source Type: research