NYU Langone doctors often recommend a stem cell transplant for people with primary myelofibrosis, because the condition can permanently scar the bone marrow. However, if you are not healthy enough to have this procedure, doctors prescribe medication.
Doctors may prescribe the JAK2 inhibitor ruxolitinib to people who aren’t eligible for stem cell transplantation. The JAK2 gene mutation can contribute to anemia, in which a person has low levels of healthy red blood cells. It’s also associated with abnormal levels of platelets and white blood cells and the formation of scar tissue in the bone marrow.
Ruxolitinib interferes with the function of JAK2 and a related protein called JAK1. This helps normalize blood cell levels and reduce symptoms, such as an enlarged spleen.
New JAK2 inhibitors for primary myelofibrosis are available through clinical trials. Your doctor can discuss whether a clinical trial is right for you.