Efficacy and tolerability of ruxolitinib in Egyptian patients with intermediate and high-risk myelofibrosis: a single center study

Background Treatment options in patients with myelofibrosis (MF) were limited until the approval of ruxolitinib as a potent Janus kinase1/Janus kinase2 inhibitor associated with improved splenomegaly, MF-related symptoms, and better survival, according to many studies. The purpose of this study was to assess the safety and effectiveness of ruxolitinib in treating MF in 34 Egyptian patients. Patients and methods This is a retro-prospective study carried out on 34 patients aged 18 years or older who were diagnosed with MF either primary, postpolycythemia vera or post-essential thrombocytosis MF. Reductions in spleen size and improvements in constitutional symptoms were used to evaluate treatment response. The first [Myeloproliferative neoplasms Symptom Assessment Form Total Symptom Score; myeloproliferative neoplasms-10) was used to evaluate constitutional symptoms. Results Of 34 patients (mean age 43–83 years), 61.76% were male. Most (70.6%) began ruxolitinib at 20 mg BID; 91.2% completed the study. At 6 months, 69% showed splenomegaly reduction; 44.8% had more than or equal to 35% spleen size decrease. Median spleen size and myeloproliferative neoplasm-10 score changes were 18 cm and 15, respectively ( P =0.0001). Treatment-related AEs led to discontinuation in 8.8%. Early initiation was linked to fewer nonhematological AEs. Survival rate was 97.1%. Conclusions Ruxolitinib is an effective drug in patients with intermediate and high-risk MF and provides spleen size reduction and systemic symptom control in our patients with manageable side effects. Larger prospective studies are warranted with long follow-up periods and evaluation of higher doses’ effect on spleen response.