ABSTRACT
Objectives: The aim of this study is to evaluate the efficacy and safety of Ruxolitinib among Myelofibrosis patients in real life compared to published data. Methods: In this retrospective observational chart review study we reviewed the medical records for all patients diagnosed with Myelofibrosis in King Abdulaziz Medical City who has received Ruxolitinib from March 1, 2012-December 1, 2015. The efficacy and safety results of Ruxolitinib were compared with published data. Results: A total of 20 patients were included. The average age was 63.3 (SD=11.6) years, with 55% of females. Efficacy: At week 24, only 20% of the study participant achieved spleen size reduction equal to or more than 20% with total average of 8% reduction in the spleen size as compared to 31.60% in COMFORT-1 study (p ≤ 0.001), The highest symptoms reduction observed with fatigue and bone pain (45% and 40% of the affected patients respectively) followed by Abdominal distress (35%) whereas no statistically significant difference observed in early satiety and night sweat. Weight loss showed improvement in 15% of the patients. Safety: Fatigue was observed in 45% of the patients, diarrhea (5%), dyspnea (15%), dizziness (5%), nausea (5%), constipation (10%), vomiting (5%), pain in extremities (5%), arthralgia (5%), pyrexia (5%), and abdominal pain (35%). At week 24, Platelet count decreased by 26% and hemoglobin decreased by 5% from the baseline. In general, only three reported cases for temporal or permanent drug discontinuation. Conclusion: Ruxolitinib therapy in real life as compared to published trials was associated with significant improvement in Myelofibrosis related symptoms and splenomegaly with an acceptable safety profile