Summary

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• Ruxolitinib (Jakafi) is indicated for the treatment of intermediate or high-risk myelofibrosis, polycythemia vera in adults intolerant to hydroxyurea, and steroid-refractory acute and chronic graft-versus-host disease in patients 12 years and older.
• The review included a total of 11 studies from systematic reviews/meta-analyses.
• In pediatric patients with steroid-refractory GvHD, Ruxolitinib showed an overall response rate (ORR) ranging from 45% to 100%, with complete response rates between 9% to 67% for acute GvHD and up to 28% for chronic GvHD. Approximately 20% experienced treatment-related toxicities such as cytopenia, liver toxicity, and infections.
• For adult patients with steroid-refractory GvHD, Ruxolitinib demonstrated higher ORR and CR rates at day 28 compared to other regimens. Common adverse events included cytopenia and infectious complications.
• In treating intermediate or high-risk myelofibrosis based on COMFORT-I/II trials data analysis, early initiation (\<=12 months from diagnosis) led to improved overall survival (OS), durable spleen volume reduction (SVR), and reduced thrombocytopenia/anemia rates compared to placebo/best available therapy.
• Safety concerns include an increased risk of herpes zoster infection during initial phases but decreased overall infection risk over extended use periods in myeloproliferative neoplasms treatments, indicating better long-term adaptation by patients.
• Comparatively among JAK inhibitors, while Fedratinib/Momelotinib had similar efficacy on spleen reduction/symptom score, Momelotinib was safer regarding anemia issues whereas Pacritinib showed potential as second-line post-JAK inhibitor exposure despite being less effective initially than Ruxolitinib in first-line usage against myelofibrosis symptoms.