POSTER 6
EFFICACY AND SAFETY OF RUXOLITINIB IN MYELOFIBROSIS – A SINGLE
CENTER EXPERIENCE
Sara Duarte1, Diana Mota2, Mónica Santos3, Rita S. Gomes1, José Pedro Carda4 and Letícia Ribeiro1
(1)Hematology, University Hospital Center of Coimbra, Coimbra, Portugal
(2)District Hospital of Figueira da Foz, Figueira da Foz, Portugal
(3)Tondela-Viseu Hospital Center, Viseu, Portugal
(4)Hematology Department, Coimbra Hospital and University Centre (CHUC), Coimbra, Portugal
Background: Ruxolitinib is a Janus kinase (JAK) 1/JAK2 inhibitor that showed to reduce
splenomegaly and symptomatic burden in patients with myelofibrosis (MF), with
improvement in quality of life and survival.
Aim: To evaluate efficacy and safety of Ruxolitinib in patients with myelofibrosis.
Methods: Retrospective analysis of patients with MF treated with Ruxolitinib between 2014
and 2019 (follow-up until December 2020), in our department.
Results: We analysed a cohort of 26 patients, 73% were males, median age was 64 years old
(30-79). MF was classified as primary in 17 patients, post-polycythemia vera in 6, and post-essential
thrombocytosis in 3 patients. Nineteen patients were in a prefibrotic stage and 7
with overt fibrosis, as demonstrated by bone marrow biopsy. JAK2V617F mutation was
detected in 77% of the cases. According to the Dinamic International Prognostic Scoring
System (DIPSS), 2 patients had low risk disease, 12 had intermediate-1, 10 had intermediate-
2 and 2 had high risk disease, at diagnosis. The majority of patients (77%) were initially treated
with alternative drugs, mostly with hydroxyurea. However, Ruxolitinib was initiated due to
systemic symptoms persistence and/or symptomatic splenomegaly. B symptoms improved in
80% of the patients. Spleen size reduction >35% at physical examination was observed in
19/21 cases, 89.5% already after 3 months of treatment, while 1 patient died due to
progressive disease (spleen size information was not available for 4 patients). Ruxolitinib-related
toxicities were observed in 17/26 patients, being the most frequent: anemia (2 grade
1, 4 grade 2 and 5 grade 3), thrombocytopenia (3 grade 1, 2 grade 2 and 1 grade 3),
decompensation of chronic kidney disease /acute kidney disease (3 cases), hepatotoxicity (2
cases) and infections (2 cases). Dose adjustment was necessary in 72% of the cases, since half
of the patients developed thrombocytopenia and one patient had severe anemia.
Nevertheless, in any case the discontinuation of the drug was needed. Median follow-up from
diagnosis was 60.3 months, and from the beginning of treatment with Ruxolitinib was 36.2
months.
Conclusion: We conclude that Ruxolitinib is a potent drug in symptomatic burden relieve in
patients with MF, with significant reduction of spleen size and constitutional symptoms. Also,
Ruxolitinib is a safe and well-tolerated drug. Anemia and thrombocytopenia were the main
toxicities, however they were manageable with dose reduction and never led to treatment
discontinuation.