Ruxolitinib for the treatment of MF: Case-based recommendations for adverse event management

While ruxolitinib remains the standard of care for intermediate-2 and high-risk myelofibrosis (MF), its use is frequently complicated by cytopenias, infections, and progressive disease. 

In a case-based review published in eJHaem, Butler et al. present seven anonymized clinical scenarios that reflect common, real-world challenges associated with ruxolitinib, such as anemia, thrombocytopenia, infection risk, secondary malignancy, and disease progression. The authors provide real-world clinical insights from over 15 years of experience managing patients with MF treated with ruxolitinib.

Key learnings¹

Anemia and thrombocytopenia are common with ruxolitinib; management strategies include ESAs, transfusions, and dose adjustments tailored to symptom burden and erythropoietin levels.

Ruxolitinib increases infection risk, including reactivation of latent infections; pre-treatment screening, vaccination, and prophylaxis are essential in at-risk patients.

Progressive splenomegaly or symptom relapse warrant reassessment for disease progression, molecular testing, and consideration of alternative JAK inhibitors or clinical trials.

As MF treatment evolves, ruxolitinib remains a key option; real-world data continue to inform strategies to mitigate toxicities and optimize long-term outcomes. Novel therapies are being explored in the context of ruxolitinib failure.