From collaboration to publication: How the Progression Research Network is advancing MPN progression research
One of the greatest challenges in myeloproliferative neoplasm (MPN) research is understanding why some patients experience disease progression and how that progression can be identified earlier and more accurately. While physicians can recognize advanced disease states, researchers are still working to determine which early biological changes signal or predict progression.
A new publication from the Progression Research Network (PRN) takes stock of what researchers know about MPN disease progression and, importantly, what they still need to learn.
Published in Leukemia, the publication titled ‘Defining and predicting disease progression in myeloproliferative neoplasms: a proposed biomarker-driven approach’ explores how the field can better define and predict progression, with the goal of identifying meaningful disease changes earlier.
The article was led in part by Dr. Raajit Rampal, MD, and Dr. Florian Heidel, MD, on behalf of the PRN. “Disease progression remains a major concern for MPN patients and those who take care of them,” explains author Dr. Rampal, who serves as Medical Advisor to MPN Research Foundation. “It is a complex problem that will require concerted focused efforts to solve.”
What is disease progression?
Disease progression in MPNs refers to changes in the disease over time that result in a more advanced disease state or are associated with poorer long-term outcomes. While progression is often recognized when a patient's diagnosis changes or the disease becomes more severe, the biological processes underlying progression is graduate and may begin years earlier.
One of the challenges highlighted by the publication is that there is no widely accepted consensus definition of progression beyond obvious disease transformations. Researchers continue to debate which clinical findings, molecular changes, and biomarkers should be considered evidence of progression and which of those factors can reliably predict future outcomes. Previous PRN discussions have identified progressive symptoms, clonal evolution, bone marrow changes, and biomarker shifts as potential indicators, but additional research is needed to determine how these factors relate to long-term outcomes
What does the publication conclude?
“This manuscript results from several years of thought and effort among many of the scientific and clinical thought leaders in MPN who are focused on progression,” says Dr. Rampal. “It is a blueprint for affirmative steps forward that we as a community can take to improve outcomes for patients.”
The authors argue that a biomarker-driven approach may help the field better define and predict disease progression. They emphasize the need to identify and validate biomarkers that can detect important biological changes before traditional clinical transitions occur.
The publication also highlights the need for consensus progression criteria, predictive indicators, improved clinical trial endpoints, and continued collaboration among researchers, industry, regulators, and patient advocates. These themes closely align with priorities identified through the PRN's collaborative work over the past several years.
From PRN Summit discussions to published research
This publication is also an example of the value of scientific convening.
“When the MPN Progression Research Network launched in 2020, our goal was to bring together leaders across the MPN community to help develop better tools for predicting disease progression, including biomarkers and clinical trial endpoints that more reliably reflect long-term patient outcomes,” Dr. Richard Winneker, PhD, former Scientific Director, current Foundation Scientific Advisor, and co-author on the publication explains. “The MPN PROGRESSion Registry and this Leukemia publication are tangible outcomes from those discussions.”
The publication in Leukemia demonstrates how conversations fostered through the PRN can develop into concrete resources for advancing progression research.
Advancing the MPNRF mission
MPN Research Foundation believes that disease progression is a key research priority. As a scientific convener at the intersection of the MPN community, we are uniquely positioned to bring researchers, clinicians, patients, and industry partners together to address this challenge collaboratively.
The PRN and its annual summit put that imperative into action. MPN Research Foundation congratulates doctors Rampal, Heidel, Winneker, and all contributing authors on this important publication and their continued commitment to advancing MPN progression research.
Sources
Rampal, R. K., Winneker, R., Kuykendall, A., Michaelis, L. C., Abu-Zeinah, G., Koschmieder, S., Bewersdorf, J. P., Ng, Z. Y., Hobbs, G., Marcellino, B., Resar, L., Scandura, J., Patel, A., Mascarenhas, J., Gerds, A., Tremblay, D., Mayack, S., Derkach, A., & Heidel, F. H. (2026). Defining and predicting disease progression in myeloproliferative neoplasms: A proposed biomarker-driven approach. Leukemia, 1–11. https://doi.org/10.1038/s41375-026-03103-0
Winneker, R. (2024) MPN Progression Research Network 4th Annual Summit Executive Summary. MPN Research Foundation. https://static1.squarespace.com/static/69dd3b420c08bb2042fac568/t/6a0365a1e8350b32117fff67/1778607521393/MPNRF_ProgressionResearchNetworkSummit2024.pdf