The clinical efficacy of ruxolitinib in treating myelofibrosis was reviewed in a new article by American researchers that was recently published in Leukemia & Lymphoma

More than 10 years have passed since the US Food and Drug Administration (FDA) first approved the treatment, sold under the brand name Jakafi®, for myelofibrosis. Since then, many clinical trials, pooled analyses, and real-world studies have shown that the treatment is effective to treat the disease, especially if administered early.

Read more about ruxolitinib for the treatment of myelofibrosis

“Despite the proven benefits, ruxolitinib treatment is sometimes delayed or avoided in favor of . . . other therapeutic approaches,” the authors of the review article said. “Over the next 10 years and beyond, the importance of optimizing therapeutic strategies, including how best to use ruxolitinib as monotherapy or in combination with other agents for further disease modification, will continue to be a topic of utmost importance.”

Ruxolitinib is a Janus kinase (JAK) 1 and JAK2 inhibitor and the first agent that the FDA approved for myelofibrosis. It is still the standard of care for the disease. 

The review article contains information about the effects of ruxolitinib on spleen volume and patient survival. It provides information about the best timing for the initiation of the treatment and how symptoms of the disease return if ruxolitinib is discontinued. It also compares the effect of the treatment with that of hydroxyurea, which is recommended by the National Comprehensive Cancer Network (NCCN) guidelines for some patients with lower-risk symptomatic myelofibrosis as well as other JAK inhibitors.

The review article also contains a comprehensive section about the side effects of the treatment before finishing with an overall historical perspective and future directions that include combination therapy based on ruxolitinib.

Myelofibrosis is a rare disease characterized by bone marrow scarring caused by the excessive production of fibrous tissue, which interferes with the production of blood cells. 


Pemmaraju N, Bose P, Rampal R, Gerds AT, Fleischman A, Verstovsek S. Ten years after ruxolitinib approval for myelofibrosis: a review of clinical efficacy. Leuk Lymphoma. Published online April 20, 2023. doi:10.1080/10428194.2023.2196593