The Role of Ruxolitinib Around Transplant in Myelofibrosis

Ruxolitinib (Jakafi, Incyte) was the first FDA-approved treatment for intermediate or high-risk myelofibrosis. It is a JAK inhibitor that can help treat an enlarged spleen caused by myelofibrosis and improve symptoms.

Research has shown that ruxolitinib is beneficial for some people with myelofibrosis  at different times in their care, including  during stem cell transplant and as a long-term maintenance therapy. 

In this article, you’ll learn about a recent studiy that has strengthened the evidence that ruxolitinib not only reduces spleen size and improves symptoms but can also help improve survival and reduce complications for people with myelofibrosis.

Ruxolitinib around transplant is safe and can improve survival according to phase 2 study

A phase II multicenter study evaluated the safety and effectiveness of using ruxolitinib before, during, and after allogeneic hematopoietic cell transplantation (HCT) in 43 people with primary or secondary myelofibrosis. Participants received reduced-intensity conditioning and standard GVHD prevention with tacrolimus and methotrexate.

• Participants showed 1-year graft-versus-host disease (GVHD)-free, relapse-free survival of 71%.
• The overall survival at two years was 82%, and progression-free survival at two years was 72%. Progression-free survival is the amount of time after treatment begins when a disease does not get worse.
• Severe acute GVHD occurred in only 2.4% of patients. Moderate to severe chronic GVHD occurred in 15%.

People with an enlarged spleen that measures more than 20 cm before transplant had lower survival rates, which may indicate the importance of early intervention. Continuing ruxolitinib through the transplant process is feasible, safe, and may reduce GVHD while maintaining strong survival outcomes for people with myelofibrosis undergoing HCT.

The importance of dose optimization and long-term treatment

Dose optimization of ruxolitinib is essential to achieve the best results . Many people benefit from a step-up dosing approach, where treatment starts at a lower dose and is gradually increased to the target amount.

Starting ruxolitinib early after diagnosis allows people to remain on therapy longer. It can be used for many years safely, and, if temporarily stopped, it can often be reintroduced or combined with other therapies later. Remarkably, 40% of patients treated for three or more years were alive 10 years after beginning ruxolitinib.

These results conclude that early initiation and careful dose management help maximize both safety and long-term survival benefits.

Ruxolitinib can provide long-lasting effects on people before or after transplantation

Across multiple studies, ruxolitinib has consistently demonstrated durable benefits for people with myelofibrosis, ranging from improving transplant outcomes to extending survival in both high- and low-risk groups. With proper dose management and early use, many patients experience long-term safety, improved quality of life, and extended survival.

For people considering ruxolitinib, these findings provide reassurance that it remains a cornerstone therapy for myelofibrosis, backed by strong clinical evidence.

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Sources: 

• Phase II Study of Peri- and Post-Transplant Ruxolitinib for Patients With Myelofibrosis Receiving Allogeneic Hematopoietic Cell Transplantation
• Safety and efficacy of ruxolitinib in an open-label, multicenter, single-arm phase 3b expanded-access study in patients with myelofibrosis: a snapshot of 1144 patients in the JUMP trial 
• A pooled analysis of overall survival in COMFORT-I and COMFORT-II, 2 randomized phase III trials of ruxolitinib for the treatment of myelofibrosis