Understanding Unmet Needs in Myelofibrosis

Announcer:
Welcome to Project Oncology on ReachMD. On this episode, sponsored by GSK, we’ll hear from Dr. Aaron T. Gerds, who’s an Associate Professor of Medicine for Hematology and Medical Oncology and Deputy Director for Clinical Research at the Cleveland Clinic Taussig Cancer Institute. He’ll be discussing the current state of myelofibrosis management. Here’s Dr. Gerds now.

Dr. Gerds:
Well, the incidence and prevalence of myelofibrosis here in the United States is not terribly high. It’s not a very common disease. We estimate there are roughly 300,000 people in the United States living with an MPN. That includes PV – polycythemia vera – essential thrombocythemia, or ET, as well as myelofibrosis. And we tend to lump these all together because they are very related diseases, and often people’s diagnoses can change from one to the other. For example, someone diagnosed with polycythemia may have developed post-polycythemia vera myelofibrosis at some point during their disease course.

There are really two key unmet needs in the treatment of myelofibrosis. The first being anemia. Anemia is very common. It’s present in roughly a third of patients at the time of diagnosis. And that proportion of patients increases with time, with roughly 60 percent of patients getting transfusions within the first year after diagnosis, and virtually every patient will need a transfusion or become anemic at some point in time during their disease course. And along with that, many of the medications that we use to treat myelofibrosis have anemia as a side effect. So certainly, we think about anemia as a key unmet need in the treatment and care of patients with myelofibrosis.

The second major element of unmet need in myelofibrosis are therapies that truly change the disease course. As of today, the only therapy we know that can lead to long-term, durable remissions and responses, or cure that is, is allogeneic bone marrow or stem cell transplant. And of course, that procedure comes with a heavy price tag of morbidity and mortality, so it's not available to everyone.

And so we need to develop therapeutics that not only improve spleen size and symptom burden, improve quality of life, and extend survival to some degree, but therapeutics that also truly modify the disease: clean out myelofibrosis cells, reverse scar tissue, improve bone marrow function, and substantially improve survival in these patients.

Announcer:
This episode of Project Oncology was sponsored by GSK. To access other episodes in this series, visit ReachMD.com/Project Oncology, where you can Be Part of the Knowledge. Thanks for listening!