“A category 1 designation from the NCCN is not just a footnote change — it’s a signal flare,” said Dr. Aaron Gerds, MD, a hematologist, clinical investigator in MPNs, and Chair of the NCCN Myeloproliferative Neoplasms Panel. “It tells clinicians, patients, and payers that the data have matured to the point where this is no longer an interesting option; it’s a standard.”
For people living with essential thrombocythemia (ET), the recent update to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology signals an important shift.
In its 2026 Clinician Guidelines, the NCCN now lists ropeginterferon alfa-2b-njft (Besremi®) as a Category 1 preferred regimen for patients with high-risk ET who had an inadequate response to, or lost response to, prior cytoreductive therapy. Category 1 is the highest level of NCCN recommendation. It reflects high-level evidence and uniform expert consensus that an intervention is appropriate.
Although ropeginterferon remains under review by the U.S. Food and Drug Administration (FDA) for ET, the guideline designation carries real weight in clinical practice and payer decisions.
Moving beyond platelet control
ET is a myeloproliferative neoplasms (MPN) cancer characterized by overproduction of platelets in the bone marrow, increasing the risk of blood clots and bleeding. Standard first-line cytoreductive therapy often includes hydroxyurea. However, some patients on cytoreductive therapies do not achieve adequate control of their symptoms, or their response to treatments diminish over time.
The results from the phase III SURPASS-ET clinical trial helped shape the new NCCN recommendation and how ET symptoms are considered. According to one of the Guideline’s authors, Dr. Anand A. Patel, MD, the clinical trial’s design reflects how the field is redefining meaningful disease control.
“The primary endpoint for the study is one that reflects durable disease control beyond just platelet count as it incorporated white blood cell count, spleen response, and symptom response,” Patel explained.
This composite endpoint broadens the defining features of ET beyond platelet numbers. Physicians are encouraged to view white blood cell counts, symptom burden, and spleen enlargement as all contributing to long-term risk and quality of life. While evaluating multiple dimensions of disease activity, SURPASS-ET generated the type of comprehensive evidence that supports a new Category 1 treatment listing.
Interferon’s expanding role across MPNs
Interferon therapy is not new to the MPN field. Ropeginterferon alfa-2b-njft is already FDA-approved for polycythemia vera, a related MPN. What is new is the formal recognition of its role in a defined treatment for certain ET patients.
Over the past decade, interferon has moved from a niche therapy to a more strategic option within the MPN treatment landscape.
“In the right patient, particularly earlier in disease, it offers the possibility of clonal containment rather than just count management,” Gerds said.
That distinction reflects a deeper understanding of MPN biology. Rather than focusing only on lowering blood counts, researchers are increasingly asking whether therapies can influence the underlying disease state (malignant clone) and potentially alter long-term disease trajectory.
What this update means for patients and clinicians
Options matter in chronic, rare blood cancers like MPNs. When disease status or life circumstances change, having more than one path helps a care team personalize treatment.
For clinicians, NCCN inclusion often influences real-world practice. Guidelines are widely used at the point of care and frequently referenced by insurers when determining coverage.
For patients with high-risk ET who have exhausted standard cytoreductive options, the update expands the evidence-based conversation. It also reinforces that treatment decisions are no longer limited to managing platelet counts alone.
Still, the guideline change reflects a broader trend in MPN biology. As shared mechanisms between PV and ET become clearer, therapies may increasingly cross traditional disease boundaries.
At MPN Research Foundation, we view this update as an example of how sustained clinical investigation translates into practical shifts in care. The science of MPNs continues to mature. As it does, treatment frameworks are evolving to reflect not only symptom control, but deeper questions about disease biology and long-term outcomes.
For people living with ET, more evidence-based options mean more informed choices — and that is meaningful progress.
Importantly, the use of ropeginterferon in ET remains investigational, pending FDA review. Patients considering interferon therapy should discuss potential risks, benefits, and monitoring requirements with their hematology team.
While the NCCN Guidelines for Clinicians treating Myeloproliferative Neoplasms were updated this year, the National Comprehensive Cancer Network (NCCN) Guidelines for Patients- Myeloproliferative Neoplasms from 2024 are available to all and remain a valuable resource where expert information is presented in plain language with visuals, charts, and definitions to empower people with MPN and caregivers to talk with their clinicians about the best treatment option. The guidelines are available in multiple languages.
