Dr. Michaelis on Unanswered Questions in Myelofibrosis Treatment

Laura C. Michaelis, MD, associate professor, Medical College of Wisconsin, discusses the direction of future myelofibrosis research.

The myelofibrosis treatment paradigm has progressed significantly since the approval of ruxolitinib (Jakafi), yet the clone still has not been successfully eradicated, leaving some remaining questions in the field, says Michaelis. One such question has to do with whether it’s even necessary to eradicate the clone. Better stratification systems exist now and some patients with myelofibrosis can live with the disease for a long time, making treatment potentially unnecessary, according to Michaelis. Many patients can be observed with no treatment, she adds.

For someone with higher-risk disease, how is the disease being modified? Ruxolitinib alone or JAK inhibitors as a whole are not going to answer that question, according to Michaelis, although they are good for splenic response and reduction symptom response. Another unanswered question is whether more drugs should be used earlier, even in patients who have lower risk of disease, to prevent the clone from expanding. Additionally, “Are new drugs needed to tackle the clone at different areas of vulnerability, not just the JAK-STAT pathway?”

Another area of research should focus on making transplant safer and performing transplants earlier before patients develop resistant, damaged marrow and other organs, such as liver or lungs, as that can make the procedure more dangerous, concludes Michaelis.