MPN Monthly Rewind: Key News and Expert Insights You May Have Missed in September 2025

September brought a series of key developments in hematologic oncology, highlighted by data presented at the 2025 SOHO Annual Meeting, updates from ongoing phase 3 trials, and expert insights on evolving diagnostic and treatment strategies.

Highlights include phase 3 results from the SURPASS-ET trial (NCT04285086) showing the efficacy of ropeginterferon alfa-2b (Besremi) in essential thrombocythemia, real-world findings on pacritinib (Vonjo) use in myelofibrosis with anemia, and outcomes from the VERONA trial (NCT04401748) evaluating venetoclax (Venclexta) plus azacitidine (Vidaza) in higher-risk myelodysplastic syndrome (MDS). Additional discussions explored molecular risk assessment, JAK inhibitor use, and investigational approaches like imetelstat, setting the stage for further updates at the 2025 ESMO Congress.

Check out this month-in-review recap of OncLive®’s coverage of the top news and expert insights in myeloproliferative neoplasms (MPNs):

Conference Updates

The 2025SOHO Annual Meeting was held in Houston, Texas, and ran from September 3-6, 2025, providing the hematologic oncology community with a host of trials and presentations to write home about, from notably effective treatment combinations to agents displaying the promise of novel mechanisms of action. OncLive editors spoke with several experts to spotlight the most important updates to come out of the meeting—those insights can be found in this exclusive recap.¹

Ropeginterferon Alfa-2b Generates Superior Efficacy vs Anagrelide in High-Risk Essential Thrombocythemia

Second-line ropeginterferon alfa-2b demonstrated superior efficacy and a favorable safety profile vs anagrelide in patients with high-risk essential thrombocythemia.² Phase 3 SURPASS-ET data showed that 42.9% of patients in the ropeginterferon alfa-2b arm achieved durable responses per modified European Leukemia Net criteria at months 9 and 12 vs just 6% in the anagrelide arm. The treatment also demonstrated higher response rates across platelet and white blood cell counts, peripheral blood count remission, symptom improvement, and absence of hemorrhagic or thrombotic events. In terms of safety, those who received ropeginterferon alfa-2b experienced a significant reduction in symptoms and thrombotic or hemorrhagic events.

Real-World Data Show Hematologic Responses With Pacritinib in Myelofibrosis With Anemia

In real-world clinical settings, pacritinib improved hemoglobin levels and stabilized platelet counts in patients with myelofibrosis and anemia.³ Data showed that 44% of patients (n = 148) who had data for at least 90 days post-index experienced a hemoglobin response. Responses were observed across varying degrees of baseline anemia, and the median time to hemoglobin response was 33 days. “These real-world findings align with the guideline recommendations for the use of pacritinib in patients with [myelofibrosis] and anemia,” Raajit K. Rampal, MD, PhD, of Memorial Sloan Kettering Cancer Center, noted in a presentation of the data.

Venetoclax Plus Azacitidine Fails to Provide OS Advantage in Treatment-Naive, Higher-Risk MDS

In the phase 3 VERONA trial, the addition of venetoclax to azacitidine did not improve median overall survival (OS) vs azacitidine plus placebo in previously untreated patients with intermediate- or high-risk MDS.⁴ The median OS outcomes in the respective arms was 22.18 months (95% CI, 17.28-27.27) and 21.68 months (95% CI, 17.81-23.79; HR, 0.908; 95% CI, 0.733-1.126; P = .38). The combination did demonstrate higher modified overall response rates (76.2% vs 57.7%) and hematologic response rates (49.4% vs 41.2%) vs placebo, along with greater transfusion independence (55.7% vs 33.6%). No new safety signals were reported.

In an exclusive interview, Sanam Loghavi, MD, of The University of Texas MD Anderson Cancer Center, discussed standard-of-care frontline testing approaches for patients with suspected MDS.⁵ “Right now, the standard-of-care testing for a patient with suspected MDS is to get a bone marrow aspirate and a biopsy for morphologic evaluation at some centers. It’s not universally done, but we have specialized flow cytometry that [looks] for immunophenotypic aberrations and hematopoietic stem cells.”

In another interview, Francesca Palandri, MD, of University of Bologna, unpacked additional research that could elucidate the role of predictive markers for ruxolitinib (Jakafi) response in myelofibrosis.⁶ “In the future, I hope that we can have an integrated model sharing clinical and molecular parameters in order to guide our treatment decisions. The implementation of molecular findings over time could be a step forward in our understanding of [this] disease and also in optimizing the management of myelofibrosis.”

Looking Ahead to ESMO 2025

Although solid tumor research will take center stage at the 2025 ESMO Congress, several key presentations will highlight ongoing advances in hematologic malignancies. Oral abstracts across two dedicated sessions will feature studies spanning CAR T-cell therapy, combination immunotherapy, molecular risk stratification, and emerging targeted approaches in lymphoma, multiple myeloma, and MDS. Check out the exclusive preview for more information.⁷

Top Expert Insights of The Month: OncLive TV Highlights

John O. Mascarenhas, MD, of Icahn School of Medicine at Mount Sinai, shed light on the potential role of imetelstat (Rytelo) in the treatment of patients with myelofibrosis,⁸ as well as the primary objective and design of the ongoing phase 3 Impact-MF trial (NCT04576156) in those with relapsed/refractory disease.⁹ “One application of [imetelstat treatment] is as a bridge to transplant for some patients, but not every patient is a transplant candidate. Many patients are not. For many patients, this drug could be used outside of the usual world of JAK inhibition, for patients who are looking to achieve disease course modification.”

In another interview, Naseema Gangat, MBBS, of Mayo Clinic, discussed the role of ruxolitinib vs other JAK inhibitors in the treatment of patients with myelofibrosis.¹⁰ “The new JAK inhibitors have been on the market for the last couple of years, specifically momelotinib [Ojjaara], which was approved specifically for anemia related to myelofibrosis, and it does have a superior anemia profile compared with ruxolitinib, but with respect to spleen and tolerability, it is inferior to ruxolitinib.”

OncLive On Air Podcast Spotlight

In a recent episode of OncLive On Air®, Andrew Kuykendall, MD, of Moffitt Cancer Center, provided an overview of the myelofibrosis treatment paradigm.¹¹ Some key topics covered include:

• Prevalence of patients with mutations in JAK2, CALR, or MPL
• Crucial roles of the JAK1, JAK2, and IRAK1 pathways in disease progression
• Consideration of JAK inhibition in eligible patients
• Challenges linked with cytopenic myelofibrosis management

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References

1. Wahner A. SOHO spotlight: Hematologists share practice-informing insights From the 2025 Annual Meeting. OncLive.com. September 10, 2025. Accessed October 8, 2025. https://www.onclive.com/view/soho-spotlight-hematologists-share-practice-informing-insights-from-the-2025-annual-meeting
2. Masarova L, Gill H, Zhang L, et al. Phase 3 study comparing ropeginterferon alfa-2b with anagrelide as second-line treatment for essential thrombocythemia: SURPASS-ET trial. Presented at: 2025 SOHO Annual Meeting; September 3-6, 2025; Houston, TX. Abstract MPN-524.
3. Rampal RK, Marrone M, Morere L, et al. Hematologic response in patients with myelofibrosis treated with pacritinib in real-world clinical settings. Clin Lymphoma Myeloma Leuk. 2025;25 (suppl 1): S686. doi:10.1016/S2152-2650(25)02157-3
4. Garcia-Manero G, Platzbecker U, Fenaux P, et al. Primary analysis of the randomized phase 3 VERONA study of venetoclax plus azacitidine versus placebo with azacitidine in patients with treatment-naïve, intermediate and higher-risk myelodysplastic syndromes. Presented at: 2025 SOHO Annual Meeting; September 3-6, 2025; Houston, TX;. Abstract MDS-1497.
5. Loghavi S. Dr Loghavi on SOC frontline testing approaches in MDS. OncLive.com. September 5, 2025. Accessed October 8, 2025. https://www.onclive.com/view/dr-loghavi-on-soc-frontline-testing-approaches-in-mds
6. Palandri F. Dr Palandri on efforts to elucidate the role of predictive markers for ruxolitinib in myelofibrosis. OncLive.com. September 5, 2025. Accessed October 8, 2025. https://www.onclive.com/view/dr-palandri-on-efforts-to-elucidate-the-role-of-predictive-markers-for-ruxolitinib-in-myelofibrosis
7. Ryan C. Hematologic oncology abstracts to watch at the 2025 ESMO Congress. OncLive.com. September 30, 2025. Accessed October 8, 2025. https://www.onclive.com/view/hematologic-oncology-abstracts-to-watch-at-the-2025-esmo-congress
8. Mascarenhas JO. Dr Mascarenhas on the role of imetelstat in the myelofibrosis treatment paradigm. OncLive.com. September 30, 2025. Accessed October 8, 2025. https://www.onclive.com/view/dr-mascarenhas-on-the-role-of-imetelstat-in-the-myelofibrosis-treatment-paradigm
9. Mascarenhas JO. Dr Mascarenhas on the IMpact-MF trial of imetelstat in R/R myelofibrosis. OncLive.com. September 26, 2025. Accessed October 8, 2025. https://www.onclive.com/view/dr-mascarenhas-on-the-impact-mf-trial-of-imetelstat-in-r-r-myelofibrosis
10. Gangat N. Dr Gangat on ruxolitinib as the standard of care in myelofibrosis. OncLive.com. September 18, 2025. Accessed October 8, 2025. https://www.onclive.com/view/dr-gangat-on-ruxolitinib-as-the-standard-of-care-in-myelofibrosis
11. Kuykendall A. Expanding JAK inhibitor use offers increased treatment options for cytopenia myelofibrosis: with Andrew Kuykendall, MD. OncLive.com. September 29, 2025. Accessed October 8, 2025. https://www.onclive.com/view/expanding-jak-inhibitor-use-offers-increased-treatment-options-for-cytopenic-myelofibrosis-with-andrew-kuykendall-md