Naveen Pemmaraju, MD, an associate professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, discusses the current understandings of how myelofibrosis (MF) presents in patients; he highlights the treatment options and where research is heading in the future.
MF is a heterogenous disease and can present in a number of different ways, says Pemmaraju. For patients with low-risk disease, according to the International Prognostic Scoring System, MF will present similarly to a chronic disease, such as essential thrombocytopenia or polycythemia vera. Life expectancy can be measured in decades for these patients. However, patients that present with intermediate- to high-risk disease are more ill when they come into clinic; they present with various symptoms and can become sick quickly from the disease itself.
How physicians score prognosis continues to evolve as more scoring systems become available, says Pemmaraju. He adds that the bottom-line is clinical presentation should include age, cytogenetics, and molecular markers, all of which could help determine low-, intermediate-, and high-risk disease. From there, physicians can decide what clinical trial or targeted therapy a patient with MF is eligible for.
The only FDA-approved agent for the treatment of MF is ruxolitinib (Jakafi), a JAK inhibitor. While other JAK inhibitors are still in development, physicians are interested in developing treatment options beyond JAK inhibition. There are a number of clinical trials that may signal a new treatment option outside the JAK pathway, but it is important to see the long-term follow-up first, Pemmaraju concludes.
For more resources and information regarding anticancer targeted therapies in myelofibrosis: [ Ссылка ]
