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ET and PV
Bridging Gaps in ET and PV: Lessons Learned From a Survey of Healthcare Professionals 

Released: May 15, 2026

Andrew T. Kuykendall
Andrew T. Kuykendall, MD
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Key Takeaways
  • Molecular testing, particularly for driver mutations in JAK2, MPL, and CALR, remains foundational in the diagnosis and risk stratification of ET and PV.
  • Treatment decisions require individualized approaches based on symptoms, thrombotic risk, patient age, and long-term disease goals.
  • Familiarity with emerging therapies being evaluated in clinical trials may enhance support for patients progressing on standard of care therapies.

Understanding Where We Are and Where We Need to Go in Myeloproliferative Neoplasms
As our understanding of myeloproliferative neoplasms (MPNs) continues to evolve, so does the complexity of managing patients with essential thrombocythemia (ET) and polycythemia vera (PV). Although these are relatively rare disorders, increasing awareness among healthcare professionals (HCPs) of the diagnostic criteria for ET and PV has improved the ability to identify these diseases earlier and more accurately. At the same time, the growing therapeutic landscape introduces both new opportunities and challenges in tailoring care for individual patients.

We recently conducted a survey of physicians who treat patients with ET or PV (N = 18) to measure their ability and confidence in diagnosing, treating, and educating those patients as well as their familiarity with emerging therapies. Most respondents were community based (n = 13) and located in the United States (n = 17).

Refining Diagnosis Through Molecular and Clinical Context
One of the clearest themes from the survey was the widespread recognition of molecular testing as a cornerstone of ET and PV diagnosis. That is reassuring because abnormal blood counts alone are only the beginning of the story. In some patients, a thrombotic event leads to an MPN diagnosis via subsequent molecular testing coupled with abnormal blood counts. In most patients, the diagnostic process ultimately depends on integrating clinical presentation, molecular findings, blood counts, and bone marrow morphology.

HCPs were queried regarding molecular testing for a patient with a history of deep vein thrombosis presenting with a myocardial infarction accompanied by frank erythrocytosis and splenomegaly. HCPs appropriately recognized the importance of testing for JAK2 mutations (78%). One half of the respondents indicated that they would test for CALR and MPL mutations. The survey also reflected an increasing openness toward broader next-generation sequencing approaches (50%). I think this is an important shift. Although targeted testing for JAK2, CALR, and MPL mutations remains highly practical and effective, broader molecular profiling can sometimes offer additional insight and, in some settings, may be more practical than individual testing.

Importantly, respondents demonstrated a strong understanding of situations in which a bone marrow biopsy/aspirate may or may not be necessary. Continuing with this patient case, a JAK2 mutation was confirmed with low serum erythropoietin. HCPs (83%) correctly recognized that current WHO criteria allow for a definitive PV diagnosis without requiring a marrow biopsy. That does not diminish the value of marrow evaluation, but it reflects a nuanced understanding of diagnostic criteria. Conversely, in a younger patient with isolated thrombocytosis and a JAK2 mutation, HCPs (61%) appropriately acknowledged the need for a bone marrow biopsy to distinguish ET from prefibrotic myelofibrosis or masked PV. Of note, in younger women, concomitant iron deficiency can obscure erythrocytosis and lead to underrecognition of PV. In those scenarios, marrow morphology remains critically important.

Risk Stratification Remains Grounded in Thrombosis Prevention
The survey findings also reinforced the central clinical priority in ET and PV: thrombosis prevention. Age, prior thrombosis, and JAK2 mutation status remain the most established thrombotic risk factors, and survey respondents (>75%) identified these risk factors. These factors are aligned with current evidence-based models such as the IPSET-thrombosis, Revised IPSET-thrombosis, and the conventional risk stratification approaches. One half of HCPs also recognized the contribution of traditional cardiovascular risk factors that are particularly relevant for arterial events.

There is growing evidence suggesting that elevated white blood cell counts may contribute to vascular complications and possibly disease evolution. Despite longstanding assumptions, extremely elevated platelet counts have not consistently correlated with increased thrombosis risk and may actually increase bleeding risk in some patients. Some HCPs (19%) selected CALR mutations as a thrombotic risk factor; patients with CALR mutations have a lower thrombotic risk compared with JAK2-mutated disease.

Therapeutic Familiarity Reflects Real-world Experience
We then asked HCPs to rank their confidence in counseling patients about available therapies. Hydroxyurea remains the therapy HCPs are most comfortable prescribing and counseling patients about, which is not surprising given its longstanding role as frontline cytoreductive therapy. HCPs also noted a relatively high familiarity with ruxolitinib, likely reflecting years of experience using JAK inhibition in myelofibrosis and more recently in second-line PV management.

HCPs are less confident discussing therapeutic options that are less frequently prescribed. This includes ropeginterferon and peginterferon, with HCPs reporting moderate confidence in counseling on these agents. Older therapies such as busulfan and anagrelide appear to be losing favor in clinical practice with availability of better, less toxic options. HCPs report limited confidence in counseling on busulfan while confidence about anagrelide ranged widely.

HCPs noted several barriers to shared decision-making including patient education level, cognitive concerns, health literacy, time constraints, and lack of HCP training.

Individualizing Treatment Beyond a One-Size-Fits-All Approach
Three case scenarios were used to assess HCP ability to manage patients with ET or PV. In the first case, hydroxyurea was the preferred option (75%) for an older patient with high-risk CALR-mutant ET. Three respondents selected ruxolitinib, which is not currently approved for ET but can be useful in this setting, specifically for patients with extreme itching. One HCP selected peginterferon as an option. I think peginterferon has a role in ET and we do use it for younger patients, especially those with JAK2 mutations. For older patients where there is less concern about disease progression, we tend to favor hydroxyurea, although peginterferon can certainly be used if patients do not tolerate hydroxyurea.

The next case was a patient with PV who developed worsening symptoms and splenomegaly while receiving hydroxyurea. Ruxolitinib was the preferred option (69%) and remains an excellent choice because of its ability to improve constitutional symptoms and reduce burden associated with splenomegaly.

Although hydroxyurea is frequently frontline therapy, patients may not tolerate it for a variety of reasons, including mucocutaneous complications, fatigue, gastrointestinal symptoms, or rare idiosyncratic reactions like fevers and ankle ulcerations. HCPs differed on their choice of therapy for a patient with PV who developed an intolerance to hydroxyurea. One half chose ruxolitinib and the other half selected an interferon—either ropeginterferon (19%) or peginterferon (31%). All of these are reasonable options for a patient who is switching because of intolerance. Interferon formulations probably have the best data for being able to “disease modify” or reduce the JAK2 allele fraction and potentially prolong the progression to myelofibrosis. For younger patients concerned about progression, interferon could be a favored option in this setting. Survey respondents indicated that the selection of second-line therapy was one of the challenges that they face in clinical practice.

The characteristics of each agent matter because no single therapy currently addresses every aspect of disease biology equally well. Some agents are more effective for symptom control. Others may offer deeper molecular responses or potential disease modification. The challenge of managing ET and PV lies in matching the right therapy to the right patient at the right time.

Looking Ahead: Emerging Therapies and Educational Opportunities
Familiarity with emerging agents such as rusfertide, bomedemstat, givinostat, and mutant CALR–targeted approaches remains low, particularly in community settings. Bomedemstat is currently in phase III trials (NCT06456346, NCT06079879) and a regulatory decision on rusfertide is anticipated later in 2026. However, the rapid pace of therapeutic development in MPNs suggests that closing this knowledge gap may become increasingly important to enable HCPs to incorporate these agents into practice if approved. Ongoing collaboration among community HCPs, academic medical centers, and specialty MPN programs will become even more valuable.

Conclusion
The management of ET and PV has evolved substantially over the past decade, driven by advances in molecular understanding, refined diagnostic criteria, and growing therapeutic options. Encouragingly, HCPs appear increasingly comfortable navigating many aspects of diagnosis and frontline management.

At the same time, important opportunities remain to improve confidence with nuanced diagnostic scenarios, treatment selection, and emerging investigational therapies. As the treatment landscape becomes more sophisticated, continued education and collaboration will be essential to delivering individualized, evidence-based care for patients with MPNs.

Your Thoughts
How confident are you in selecting therapy for patients with ET or PV intolerant or resistant to hydroxyurea? Are you aware of the latest evidence on emerging therapies? Answer the polling question and leave a comment to join the discussion.

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