Drug updated on 4/10/2024
| Dosage Form | Tablet (oral; 5 mg, 10 mg, 15 mg, 20 mg, 25 mg) |
| Drug Class | Kinase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis in adults.
- For treatment of polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea.
- For treatment of steroid-refractory acute graft-versus-host disease in adult and pediatric patients 12 years and older.
- For treatment of chronic graft-versus-host disease after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.
Summary
- Ruxolitinib (Jakafi) is a selective inhibitor of Janus Kinase 1 and 2, used for treating myelofibrosis, polycythemia vera, and steroid-refractory acute and chronic graft-versus-host disease in both adult and pediatric patients aged 12 years or older.
- Analysis was based on 11 systematic reviews/meta-analyses concerning Ruxolitinib.
- In the treatment of steroid-refractory graft-versus-host disease (SR-GvHD), Ruxolitinib showed promising effectiveness with an overall response rate ranging from 45% to100%, yielding significant results compared to other second-line therapies such as mesenchymal stroma cell addition or inolimomab.
- For myelofibrosis treatment, earlier initiation of Ruxolitinib post-diagnosis resulted in improved overall survival rates, fewer cytopenia events, durable spleen volume response (SVR), making it more effective than placebo or the best available therapy.
- When comparing different Janus kinase inhibitors for myelofibrosis treatment, like fedratinib and momelotinib; efficacy-wise, they were comparable, but side effect profiles varied, which might guide the choice between them.
- In treating polycythemia vera patients who are intolerant or have had an inadequate response to hydroxyurea, thrombosis reduction favored Ruxolitinib over the best available therapy, although differences did not reach statistically significant levels. It also improved quality-of-life outcomes compared to conventional treatments.
- The safety profile involves considerations for treatment-related toxicities, including cytopenia, liver toxicity, and infections. Ruxolitinib has been associated with increased risks of specific infections, notably herpes zoster, in the context of Myeloproliferative neoplasm (MPN) patients. However, the safety profile is deemed manageable and comparable to other options.
- Ruxolitinib's advantages over other treatments include improved response rates and clinical outcomes, particularly for SR-GvHD and myelofibrosis. It offers an essential therapeutic choice for patients who have limited options due to resistance or intolerance to other therapies but requires careful monitoring for specific side effects, including increased risks of infections and cytopenia.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Jakafi (ruxolitinib) Prescribing Information. | 2023 | Incyte Corporation, Wilmington, DE |
Systematic Reviews / Meta-Analyses
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| ERS/EBMT clinical practice guideline on treatment of pulmonary chronic graft-versus-host disease in adults. | 2024 | European Respiratory Journal |
| Management of Patients with Early Myelofibrosis: A Discussion of Best Practices. | 2024 | Current Hematologic Malignancy Reports |
| Myeloproliferative Neoplasms, Version 3.2022, NCCN Clinical Practice Guidelines in Oncology. | 2022 | Journal of the National Comprehensive Cancer Network |
| Graft-Versus-Host Disease. | 2021 | Journal of the National Comprehensive Cancer |
| A guideline for the management of specific situations in polycythaemia vera and secondary erythrocytosis. A British Society for Haematology Guideline. | 2019 | British Journal of Haematology |