Top Three Questions for MPN Researchers With Dr. Pemmaraju

“The HemOnc Pulse” was live in Chicago, Illinois, on May 3-4, and host Chadi Nabhan, MD, MBA, FACP, sat down with Naveen Pemmaraju, MD, of the University of Texas MD Anderson Cancer Center, to discuss his presentation on unanswered questions in myeloproliferative neoplasms (MPN).
Dr. Pemmaraju outlined the three major questions that he covered in his session. Firstly, he discussed whether the existing response criteria in patients with MPN can be improved.
“We started out with spleen size and symptom burden, but now we have combination therapies, mutant-specific therapies, and immune therapies,” he noted. “I asked the question to the panel, ‘Shouldn’t we be considering something beyond that?’ [Shouldn't we be considering] overall survival (OS), progression-free survival (PFS), or event-free survival (EFS), as is done in other tumors?”
Dr. Pemmaraju then questioned the Janus kinase inhibitor monotherapy standard, asking the MPN panel, “Is two drugs better than one?”
He pointed out that treatment strategies for myeloma, acute myeloid leukemia, and acute lymphoblastic leukemia clinicians already include combination therapies in upfront treatment, while strategies for lymphocytic leukemia and MPN have not expanded beyond single-agent therapy.
Lastly, Dr. Pemmaraju highlighted the need for more advanced disease measures to guide treatment decisions. “Surrogate endpoints, biomarkers, something to tell us that our drugs are working and the disease is reducing,” he explained.
“I want my patients to live longer, and I want them to feel better; but I want to capture that with metrics that I don’t think we are doing right now,” Dr. Pemmaraju continued. “To capture OS, PFS, EFS, we may need something at the level of fibrosis reduction, allele burden reduction, or cytokine modulation.”
“Most of the drugs we have so far have not met that bar, but we are not following them long enough,” he suggested. “I’m calling for longer follow-up, more clinical translational studies, and the identification of some of these new markers. We may be missing something in plain sight that can guide us for future drug development.”