Myelofibrosis Year in Review With Dr. Raajit Rampal

Raajit Rampal, MD, PhD, of the Memorial Sloan Kettering Cancer Center, highlighted notable myelofibrosis research presented at the 67th American Society of Hematology Annual Meeting & Exposition, from MANIFEST-2 to RALLY-MF.

The phase 3 MANIFEST-2 trial evaluated ruxolitinib as monotherapy or in combination with pelabresib in patients with janus kinase (JAK) inhibitor naïve myelofibrosis. Notably, the combination improved spleen and symptom response compared with ruxolitinib alone at 96 weeks without additive toxicities.

Next, Dr. Rampal covered the phase 2 RALLY-MF trial, which evaluated DISC-0974—an anti-hemojuvelin antibody—in patients with myelofibrosis and anemia. The researchers observed benefits in terms of improvement in hemoglobin and reduction in transfusion burden.

“This is a study that is addressing one of the major unmet needs in patients with myelofibrosis, which is anemia,” he explained. “Reduction in transfusion burden is a very clinically meaningful end point. If we take a patient who is getting transfused once a week and cut that down to twice a month, that's a significant savings in terms of the patient's time and infusion share.”

Finally, Dr. Rampal shared what’s on the horizon in myelofibrosis research in 2026, including additional data on calreticulin targeted agents, phase 3 results on selinexor, and further follow-up data from MANIFEST-2.