Contributions
Abstract: PB1726
Type: Publication Only
Session title: Myeloproliferative neoplasms - Clinical
Background
ruxolitinib is a potent Janus Associated Kinase (JAK) inhibitor that mediates the signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. It acts by inhibiting dysregulated JAK signaling associated with myelofibrosis (MF). Also, it has been shown to be an effective drug in alleviating systemic symptoms and reducing spleen size with significant improvement in patients (pts) quality of life.
Aims
report the efficacy and safety of ruxolitinib in a cohort of MF pts treated in routine clinical practice.
Methods
we retrospectively analyzed a total of 16 pts with MF treated with ruxolitinib in our hospital, between 2015 and 2020. For toxicity grading (gr) we used CTCAE criteria v5.0.
Results
62.5% were males and the median age at diagnosis was 69.5 years (22-77). Ten pts (62.5%) were diagnosed as primary MF, 4 (25%) as post-essential thrombocythemia MF and 2 (12.5%) as post-polycythemia vera MF. Ten pts (62.5%) were JAK2 V617F mutated, 1 (6.3%) was CALR and 5 (31.3%) were triple negative. When analyzing risk stratification, 50% were DIPSS intermediate II and 25% high risk. Indications for treatment were constitutional symptoms plus splenomegaly in 10 (62.5%) pts, splenomegaly alone in 5 (31.3%) and intolerance to hydroxycarbamide in 1 (6.25%). Median time from diagnosis to start of ruxolitinib was 32.9 months with a median initial dose of 30mg/day. At baseline of treatment, 75% pts experienced constitutional symptoms among which 58.3% had complete symptomatic resolution. An average reduction in the spleen length of 60.6%, with almost all patients having a spleen reduction, was observed. Seven pts (53.8%) required a dose reduction: 3 (42.9%) due to anemia gr4, 1 (14.3%) due to thrombocytopenia gr2 and 2 (28.6%) due to both anemia and thrombocytopenia gr3. Five pts (25%) discontinued ruxolitinib, 2 due to leukemic transformation, 2 due to anemia g4 and gr5 and 1 due to no response.
Conclusion
our analysis confirms the efficacy and safety of ruxolitinib outside clinical trials with more than half of treated pts achieving and maintaining a clinical benefit and most of them reporting relief from symptoms. Clinicians are usually more alert towards thrombocytopenia but in our series, anemia was the most frequent and serious toxicity having occurred in more than half of the cases. Although, we consider that it is manageable, we would like to propose more strict indications for ruxolitinib dose adjustment according to the hemoglobin cut-off value.
Keyword(s): Myelofibrosis, Myeloproliferative disorder, Ruxolitinib
Abstract: PB1726
Type: Publication Only
Session title: Myeloproliferative neoplasms - Clinical
Background
ruxolitinib is a potent Janus Associated Kinase (JAK) inhibitor that mediates the signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. It acts by inhibiting dysregulated JAK signaling associated with myelofibrosis (MF). Also, it has been shown to be an effective drug in alleviating systemic symptoms and reducing spleen size with significant improvement in patients (pts) quality of life.
Aims
report the efficacy and safety of ruxolitinib in a cohort of MF pts treated in routine clinical practice.
Methods
we retrospectively analyzed a total of 16 pts with MF treated with ruxolitinib in our hospital, between 2015 and 2020. For toxicity grading (gr) we used CTCAE criteria v5.0.
Results
62.5% were males and the median age at diagnosis was 69.5 years (22-77). Ten pts (62.5%) were diagnosed as primary MF, 4 (25%) as post-essential thrombocythemia MF and 2 (12.5%) as post-polycythemia vera MF. Ten pts (62.5%) were JAK2 V617F mutated, 1 (6.3%) was CALR and 5 (31.3%) were triple negative. When analyzing risk stratification, 50% were DIPSS intermediate II and 25% high risk. Indications for treatment were constitutional symptoms plus splenomegaly in 10 (62.5%) pts, splenomegaly alone in 5 (31.3%) and intolerance to hydroxycarbamide in 1 (6.25%). Median time from diagnosis to start of ruxolitinib was 32.9 months with a median initial dose of 30mg/day. At baseline of treatment, 75% pts experienced constitutional symptoms among which 58.3% had complete symptomatic resolution. An average reduction in the spleen length of 60.6%, with almost all patients having a spleen reduction, was observed. Seven pts (53.8%) required a dose reduction: 3 (42.9%) due to anemia gr4, 1 (14.3%) due to thrombocytopenia gr2 and 2 (28.6%) due to both anemia and thrombocytopenia gr3. Five pts (25%) discontinued ruxolitinib, 2 due to leukemic transformation, 2 due to anemia g4 and gr5 and 1 due to no response.
Conclusion
our analysis confirms the efficacy and safety of ruxolitinib outside clinical trials with more than half of treated pts achieving and maintaining a clinical benefit and most of them reporting relief from symptoms. Clinicians are usually more alert towards thrombocytopenia but in our series, anemia was the most frequent and serious toxicity having occurred in more than half of the cases. Although, we consider that it is manageable, we would like to propose more strict indications for ruxolitinib dose adjustment according to the hemoglobin cut-off value.
Keyword(s): Myelofibrosis, Myeloproliferative disorder, Ruxolitinib