30 facts about MPNs

Fact #1 

Polycythemia vera (PV) and essential thrombocythemia (ET) are often found during routine bloodwork. It’s common for patients to have no symptoms or mild symptoms for years.  

Fact #2 

Bringing new treatments to MPN patients takes many years, from discoveries in the laboratory, to pre-clinical research that test for safety and effectiveness. Successful pre-clinical therapeutic drug candidates move to human clinical trials and ultimately require approval by a regulatory agency, such as the US Food and Drug Administration (FDA).  

Fact #3 

MPN clinical trials have gone beyond JAK inhibitors and are now looking at new targets and therapeutic pathways that will expand the universe of options for patients. This is especially promising for patients who could not tolerate or loose their response to JAK inhibitors.  

Fact #4 

Anemia is a major contributor to the hallmark fatigue associated with MPNs.  

Fact #5 

MPN research is rapidly accelerating our understanding of what causes these chronic diseases and how to treat them. While there are only 4 FDA approved MPN drug treatments, many clinical trials are underway across the globe, studying potential new and better treatment options.  

(Constantinescu et al., 2026) 

Fact #6 

Three key gene mutations drive most MPNs. More than 90% of MPN patients have a mutation in the genes JAK2, CALR, or MPL. These changes cause blood stem cells to stay “switched on,” producing more blood cells than the body needs. (Luque Paz et al., 2023)

Fact #7

With research advancing so rapidly, many people living with an MPN feel that clinical trials offer better treatment options for their disease symptoms. Consider speaking withyour doctor about the MPN trials for which you might be eligible.. 

Fact #8 

Inflammation plays many roles in MPN cancers. Research points towards inflammation contributing to the mutations that drive MPNs, symptom severity, and the progression of MPNs from one disease state to another. 

(Craver et al., 2018; Fleischman, 2015) 

Fact #9 

MPNs are considered chronic caners. This means they are ongoing, slow to progress, and require long-term monitoring and treatment. 

Fact #10 

There are more clinical trials every year for people living with ET, PV, or MF. Get a personalized list of trials that match your diagnosis and medical history, then share with the medical team that manages your MPN. Get started with the MPN Clinical Trial Finder

Fact #11 

Before a clinical study can begin it is carefully evaluated by the health authorities. The safety and scientific validity of a clinical study are then carefully monitored by a study sponsor and investigators. Along with the benefits of participating in a clinical trial, it’s important to discuss the potential risks with healthcare professionals. Speak to your MPN care team to see if you qualify for any trials in your area. 

Fact #12 

The genetic mutations associated with MPNs can arise early, long before symptoms appear. Research shows some driver mutations occur in childhood or early adulthood, growing silently for years before diagnosis. This helps explain why many people feel something was “off” long before they had a name for it.(Williams et al., 2022) 

Fact #13 

Your body constantly makes new blood cells from stem cells in the bone marrow through a process called hematopoiesis. Blasts are early, still-developing cells that are in a transitionary stage between stem cells and fully mature blood cells. In MPNs, a sustained rise in the number of blasts in the blood or bone marrow can mean the disease is more seriously disrupting the production of blood cells. 

(What Are Blast Cells?, 2024) 

Fact #14 

Because MPNs are rare, it is often challenging to find enough people with ET, PV, and MF who are willing and able to participate in a clinical trial. Unfortunately, this can slow new drug development and approvals. Ask your doctor what MPN trial might be a good match for you. 

Fact #15 

MPNs are a chronic cancer that can progress. Disease progression refers to the worsening or advancement of a disease over time, often marked by new symptoms, changes in laboratory results, or the spread of disease to other parts of the body. In myeloproliferative neoplasms (MPNs), progression can include a shift from one phase or disease state to another — such as the development of overt myelofibrosis or acute myeloid leukemia. 

Fact #16 

MPN Research Foundation focuses on gaps in MPN research to better understand and treat ET, PV, and MF, investing more than $21 million over the past two decades. You can be a part of the solution by making an investment toward MPN research today. 

Fact #17 

Some MPN cancer treatments such as hydroxyurea and JAK inhibitors are associated with increased UV light sensitivity and higher risk of non-melanoma skin cancer That’s one of the many reasons why MPN patients should talk to their physician about referrals to other specialties including dermatologists. 

(Landtblom et al., 2018; Rampotas et al., 2024; Starace et al., 2024) 

Fact #18 

From night sweats to fatigue, MPN patients can experience very different symptoms. If you or a loved one have an MPN and you are not satisfied with the information or course of treatment provided to you, consider finding an MPN specialist, familiar with the various ways MPNs manifest. 

Fact #19 

Ruxolitinib was approved in 2011 for myelofibrosis and has provided symptom relief for many patients. But some patients don’t see results or lose their response to ruxolitinib over time. With more treatments on the market, many current clinical trials are now testing the effectiveness of drug combinations.  

Fact #20 

Participating in a clinical trial doesn’t always mean moving your care to an academic medical center. With community-based practitioners referring patients to trial sites, now more than ever, flexible trial designs include a diversity of participation options.  

Fact #21 

Research projects funded by MPN Research Foundation have led to important advances for people living with MPNs. These include discovery of the CALR mutation, understanding more about interferon and disease progression, and identifying possible new treatments. 

Fact #22 

Founded in 1999 as the first organization dedicated to MPNs, MPN Reseach Foundation funds projects that addresses the critical gaps in current MPN research with the potential to move answers from the laboratory to the clinic. The Foundation plays a uniquely independent role in the MPN community, convening academia, industry, patients and caregivers, advocates, and regulatory agencies, ultimately to improve the lives of people living with MPNs. 

Fact #23

Not all research looks like a clinical trial — and that’s a good thing. Scientists call research that asks how and why biology works basic research. Basic research discoveries lay the groundwork for pre‑clinical studies and, eventually, clinical trials. The MPN Research Foundation invests early because today’s laboratory insights can become tomorrow’s trials — and more options for people living with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF). 

Fact #24 

Because symptoms can vary significantly among MPN patients, it is important to track changes in symptoms and their severity between doctor visits. Keep current on the latest MPN research updates and speak to your doctor about how changes in your symptoms may call for a change in treatment. 

Fact #25 

Whether the number of MPN mutated cells increases or not may depend on inherited DNA, the bone marrow environment, inflammation, and possibly lifestyle. These layers of influence help explain why MPNs progress differently from person to person. 

Fact #26 

MPN patients can expect a variety of different tests to confirm their diagnosis or check on their current condition. Blood counts and blood smears measure blood cell production and looking for immature blood cells, called blasts, in the bloodstream. Bone marrow biopsy or aspirate tallying blasts in the marrow and assessing fibrosis and chromosomes of certain cells. And molecular testing finds types and rates of mutations which may guide treatment choices. 

Fact #27 

If you are considering a clinical trial, you have the right to informed consent. FDA has guidelines for sponsors and doctors to ensure participants fully comprehend potential risks and benefits, and what participation in the clinical trial will entail. Get all of your questions answered before enrolling. 

Fact #28 

Allogeneic hematopoietic stem cell transplant, or allo-HCT, is considered the only treatment strategy that can “cure” myelofibrosis (MF).It has substantial risks but is also linked with long-term disease-free survival. It’s recommended that all MPN patients discuss their allogeneic stem cell transplant options with an MPN specialist. 

(Polverelli et al., 2022) 

Fact #29 

Patients with MPNs are more likely to have blood clots, also called thrombotic events. These cardiovascular events are associated with inflammation, are dangerous for patients and may indicate a risk of disease progression. (Barbui et al., 2024) 

Fact #30 

Biomarkers are biological molecules found in blood, other body fluids, or tissues. Their presence or quantity can be interpreted by clinicians to better understand how a patient is being affected by a disease. They are also used to see how well a patient is responding to a treatment for a disease or condition. Identifying and assessing patient health using biomarkers has the potential to lead to more personalized treatments.  

Sources: 

Barbui, T., Ghirardi, A., Carobbio, A., De Stefano, V., Rambaldi, A., Tefferi, A., & Vannucchi, A. M. (2024). Thrombosis in myeloproliferative neoplasms: A viewpoint on its impact on myelofibrosis, mortality, and solid tumors. Blood Cancer Journal, 14(1), 1–8. https://doi.org/10.1038/s41408-024-01169-6 

Constantinescu, S. N., Vainchenker, W., & Pecquet, C. (2026). Next-Generation JAK Inhibitors in the Treatment of Myeloproliferative Neoplasms. Blood, blood.2025028645. https://doi.org/10.1182/blood.2025028645 

Craver, B. M., El Alaoui, K., Scherber, R. M., & Fleischman, A. G. (2018). The Critical Role of Inflammation in the Pathogenesis and Progression of Myeloid Malignancies. Cancers, 10(4), 104. https://doi.org/10.3390/cancers10040104 

Fleischman, A. G. (2015). Inflammation as a Driver of Clonal Evolution in Myeloproliferative Neoplasm. Mediators of Inflammation, 2015, 606819. https://doi.org/10.1155/2015/606819 

Landtblom, A. R., Bower, H., Andersson, T. M.-L., Dickman, P. W., Samuelsson, J., Björkholm, M., Kristinsson, S. Y., & Hultcrantz, M. (2018). Second malignancies in patients with myeloproliferative neoplasms: A population-based cohort study of 9379 patients. Leukemia, 32(10), 2203–2210. https://doi.org/10.1038/s41375-018-0027-y 

Luque Paz, D., Kralovics, R., & Skoda, R. C. (2023). Genetic basis and molecular profiling in myeloproliferative neoplasms. Blood, 141(16), 1909–1921. https://doi.org/10.1182/blood.2022017578 

Polverelli, N., Farina, M., D’Adda, M., Damiani, E., Grazioli, L., Leoni, A., Malagola, M., Bernardi, S., & Russo, D. (2022). How We Manage Myelofibrosis Candidates for Allogeneic Stem Cell Transplantation. Cells, 11(3), 553. https://doi.org/10.3390/cells11030553 

Rampotas, A., Carter-Brzezinski, L., Somervaille, T. C. P., Forryan, J., Panitsas, F., Harrison, C., Witherall, R., Innes, A. J., Wallis, L., Butt, N. M., Psaila, B., Mead, A. J., Carter, M., Godfrey, A. L., Laing, H., Garg, M., Francis, S., Ewing, J., Teh, C. H., … Lambert, J. (2024). Outcomes and characteristics of nonmelanoma skin cancers in patients with myeloproliferative neoplasms on ruxolitinib. Blood, 143(2), 178–182. https://doi.org/10.1182/blood.2023022345 

Starace, M., Rapparini, L., & Cedirian, S. (2024). Skin Malignancies Due to Anti-Cancer Therapies. Cancers, 16(11), 1960. https://doi.org/10.3390/cancers16111960 

What Are Blast Cells? (2024, November 21). Cleveland Clinic. https://my.clevelandclinic.org/health/body/blast-cells 

Williams, N., Lee, J., Mitchell, E., Moore, L., Baxter, E. J., Hewinson, J., Dawson, K. J., Menzies, A., Godfrey, A. L., Green, A. R., Campbell, P. J., & Nangalia, J. (2022). Life histories of myeloproliferative neoplasms inferred from phylogenies. Nature, 602(7895), 162–168. https://doi.org/10.1038/s41586-021-04312-6