Addressing ‘A Main Problem’ Becomes Center of MPN Treatment Research


Despite the several classes of drugs either approved, or in development, for the treatment of myeloproliferative neoplasms, a big question remains.

Despite the several classes of drugs either approved, or in development, for the treatment of myeloproliferative neoplasms (MPN), a big question remains.

“The main problem right now is…in whom should we develop these medications for, in whom should these medications be tested, and what should be the end points,” Srdan Verstovsek, MD, PhD, of The University of Texas MD Anderson Cancer Center, said during a presentation during the 2021 Society of Hematologic Oncology (SOHO) Annual Meeting.

“We are moving…from looking at the spleen and symptoms and anemia to survival benefit,” he explained. “I would expect that this trend will continue; we will see perhaps more survival benefit designed studies or time-to-progression or prevention of symptoms and signs in the first place—not to treat only patients that need therapy but prevent symptoms or signs from becoming a problem.”

The issue, Verstovsek noted, is that strategies are needed to identify how to target the newer therapies that are being developed and whether to study them as single agents, or in combination with other standard-of-care therapies.

Overall, Verstovsek reviewed several topics and provided insight into the possible future of the field of myeloproliferative neoplasms.


First, Verstovsek discussed prognostication factors in individuals with myelofibrosis. He explained that there are several questions healthcare providers must ask themselves when doing a prognosis of a patient. The main question he asks is if a patient qualifies for hematopoietic transplant.

Another question, which Verstovsek said he does not weigh as high, is whether the patient needs therapy. Because, he said, providers should be looking at the phenotype of the patients and not what the risk of dying is. The goal is to see when the patient would need therapy, according to Verstovsek.

“We should clinically assess the need for therapy rather than looking at risk of dying when we decide on the new medication,” he said. “Therefore, the question is, do we really need to be so precise and develop, as I see it now, 10 different prognostic scoring systems?”

The answer, according to Verstovsek, is no. Instead, prognostics for identifying treatment options for patients should be more individualized and rely on genetic information.

“That's why [these] type[s] of algorithms are more likely to exist in the future; for us to use rather than arguing which 1 of the multiple different prognostic systems is better than the other,” he said.

Looking Ahead

He noted that the advent of certain vaccinations may aid in enhancing the immune system and control the disease in its early stages, so the disease remains a prefibrotic myelofibrosis.

However, disease evolution and progression often happens.

In those instances, to gain valuable information for the application of new therapies, Verstovsek noted that the field of MPNs is shifting to subgrouping patients by their genetic profile.

In fact, he highlighted how significant clinical responses with IDH1/2 inhibitors has previously been reported. This is in addition to ongoing or planned studies assessing IDH inhibitors with JAK inhibitors.

“Therefore, the new way of looking at the algorithms for treatment of accelerated/blastic phase has to do with the implementation of [next-generation sequencing] analysis to include IDH1/2, [and] possibly even P53 mutation,” he concluded. “P53 mutations [are a] bad prognostic factor for transplant; [hypomethylating agent]-based therapy should be implemented for that. So IDH inhibitors for IDH mutations; hypomethylation otherwise, many times combined with a JAK inhibitor for control of the symptoms and signs.”

Verstovsek S. Next Questions: Myeloproliferative Neoplasms. Presented at: Society of Hematologic Oncology 2021 Annual Meeting; September 8-10. Accessed September 11, 2021.

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