MPN Hub

This independent educational activity is supported by GSK.​ All content is developed independently by the faculty; the funder is allowed no influence on the content.

During the European School of Haematalogy (ESH) 4th How to Diagnose and Treat CML/MPN conference, the MPN hub held a symposium on March 9, 2025, titled, Anemia in myelofibrosis: Sequencing therapies to optimize patient outcomes. Here, we share a presentation by Paola Guglielmelli, University of Florence, Florence, IT​, discussing the optimal sequencing of therapies in patients with myelofibrosis and anemia.

Guglielmelli discussed the differences between myeloproliferative and cytopenic myelofibrosis (MF), highlighting the limited treatment options in patients with severe cytopenias. She discussed the British Society of Haematology and National Comprehensive Cancer Network guidelines for the treatment of anemia in patients with MF, which both suggest the use of momelotinib in patients with MF and anemia and symptomatic splenomegaly.

Guglielmelli then presented three patient cases and discussed the clinical evidence that influences treatment decisions specific to each case.

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This independent educational activity was supported by GSK.​ All content was developed independently by the faculty; the funder is allowed no influence on the content.

During the European School of Haematalogy (ESH) 4th How to Diagnose and Treat CML/MPN conference, the MPN Hub held a symposium on March 9, 2025, titled Anemia in myelofibrosis: Sequencing therapies to optimize patient outcomes. Here, we share a presentation by Francesco Passamonti, Università degli Studi di Milano, Milan, IT, discussing the treatment landscape for patients with myelofibrosis and anemia.

Firstly, Passamonti provided an overview of current treatment strategies for patients with myelofibrosis-related anemia, including red blood cell transfusion, erythropoiesis-stimulating agents, androgens, splenectomy, immunomodulatory drug agents, and Janus kinase inhibitors. He then discussed key efficacy and safety data from phase III trials of ruxolitinib, fedratinib, pacritinib, and momelotinib, with a focus on anemia.

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This educational resource is independently supported by GSK. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

During an MPN Hub symposium presented at the European School of Haematology (ESH) 4th How to Diagnose and Treat: CML/MPN conference, MPN Hub steering committee Chair Jean-Jacques Kiladjian, Université Paris Cité and Hôpital Saint-Louis, Paris, FR, presented a review of anemia in myelofibrosis (MF), with a focus on the impact of anemia on patient outcomes.

Key points 

• MF is associated with a range of clinical manifestations and constitutional symptoms, including anemia, that significantly impact patient outcomes.  
• The cause of anemia in MF is multifactorial, including splenomegaly-induced hemodilution and increases in plasma volume, red blood cell sequestration and destruction, and ineffective erythropoiesis; it can also be caused or exacerbated by certain MF treatments. 
• Anemia is prevalent in patients with MF, with ~40% of patients anemic at diagnosis and ~25% of patients transfusion dependent at diagnosis, rising to almost 60% and 50% respectively within the first year of diagnosis and increasing further over time. It is often severe (defined as <8g/dL hemoglobin or transfusion dependence). 
• Anemia is included in all MF prognostic scoring systems, and both anemia severity and transfusion dependence are strongly predictive of reduced QoL and survival outcomes in patients with anemia. 

- Anemia and increasing severity of anemia significantly reduce OS for patients with MF.  

- The median OS in transfusion-independent patients is ~62 months, and only ~22 months in highly transfusion-dependent patients. 

- Transfusion burden can impact employment and social life. 

• Improving anemia is an important goal alongside targeting spleen size and symptoms for improving everyday QoL and long-term outcomes in patients with MF. 

- Anemia therefore represents an important consideration for treatment decision-making. 

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During the MPN Hub Steering Committee meeting, Ruben Mesa discussed anemia as an unmet need and the impact on quality of life and survival outcomes in patients with myelofibrosis (MF).

1. Anemia in patients with MF is a complex, multifactorial process, and can be exacerbated by treatment with some Janus kinase inhibitors, including ruxolitinib and fedratinib.
2. Anemia is common in patients with primary MF, with one study reporting hemoglobin levels <10 g/dL in 38% of patients at the time of diagnosis, increasing to 58% in patients seen within 1 year since diagnosis.
3. Anemia can negatively impact overall survival; in a study of 1,109 consecutive patients with primary MF, the median overall survival among patients with no, mild, moderate, and severe anemia was 7.9 years, 4.9 years, 3.4 years, and 2.1 years, respectively.
4. Anemia also negatively impacts patients' quality of life; a study of 85 patients with MF showed that anemia responders had greater improvements in quality of life vs nonresponders.
5. There is a need for effective treatments to address MF-related anemia.

This educational resource is independently supported by GSK and Bristol Myers Squibb. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

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The MPN Hub was pleased to speak to Aaron Gerds, Cleveland Clinic Taussig Cancer Institute, Cleveland, US. Gerds discussed treating anemia in patients with myelofibrosis (MF).

Gerds highlights the impact of anemia on survival outcomes in patients with MF, and reviews key data from the phase II ACE-536-MF-001 trial (NCT03194542) evaluating the safety and efficacy of luspatercept in patients with MF and anemia, including patients with and without transfusion dependence and concurrent Janus kinase (JAK) inhibition. He then highlights the ongoing randomized, phase III INDEPENDENCE trial (NCT04717414) assessing the safety and efficacy of luspatercept in combination with ruxolitinib in transfusion-dependent patients with MF. Finally, Gerds discusses potential future combinations of luspatercept with ACVR1 inhibitors. 

This educational resource is independently supported by Bristol Myers Squibb. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

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The MPN Hub was pleased to speak to Aaron Gerds, Cleveland Clinic Taussig Cancer Institute, Cleveland, US. We asked, How might luspatercept benefit patients with myelofibrosis (MF)? 

Aaron Gerds opens by discussing the prevalence and issue of anemia in patients with MF, noting that almost all patients will become anemic at some point in the course of their disease. Gerds discusses luspatercept as a treatment option in this indication, sharing the latest clinical trial data and emphasizing the benefit of a reduction in transfusion dependency for patients treated with luspatercept. The interview closes with a look to ongoing clinical trials, including the INDEPENDENCE study and the implications of these data on future management strategies for anemic myelofibrosis.

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During the European Hematology Association (EHA) 2024 Hybrid Congress, the MPN Hub was pleased to speak with Haifa Kathrin Al-Ali, University Hospital Halle, Halle, DE. We asked, What more can be done to improve treatment for patients with polycythemia vera?

Al-Ali provides an overview of how to improve treatment and outcomes for patients with polycythemia vera, opening with a discussion of the latest advancements in the field and key areas for improvement. Al-Ali shares insights on symptoms management, risk stratification, and the potential applications for machine learning and artificial intelligence in MPN. This interview concludes with a discussion of the goals for treatment and how these may vary between patients and physicians, highlighting the importance of the patient voice in the treatment of MPN.

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The MPN Hub was pleased to speak to Laura Michaelis, Medical College of Wisconsin, Milwaukee, US and Anand Patel, University of Chicago, Chicago, US. We asked, When to refer patients with MPN to clinical trials: Part II - Patients with myelofibrosis (MF).

Patel opens by highlighting the need for more long-term effective MF treatments and presents long-term data on the cessation of existing therapies such as ruxolitinib. Michaelis and Patel then discuss factors that influence the decision to refer to a clinical trial in patients with newly diagnosed MF; outlining anemia and other symptoms as indicators for referral. They also discuss add-on clinical trials as options for patients who do not achieving optimal response with their current standard of care treatment.

The podcast concludes with a discussion on clinical trial referral versus transplantation in patients not achieving an optimal treatment response with standard of care. Finally, the importance of communicating effectively with the patient and considering their comorbidities is highlighted.

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The MPN Hub was pleased to speak to Laura Michaelis, Medical College of Wisconsin, Milwaukee, US and Anand Patel, University of Chicago, Chicago, US. We asked, When to refer patients with MPN to clinical trials: Part I — Patients with essential thrombocytopenia/polycythemia vera.

In this podcast, Michaelis and Patel discuss the factors which determine whether standard of care treatment or referral to a clinical trial is the most appropriate course of action. Patel begins by outlining drugs currently under clinical trial for essential thrombocytopenia and polycythemia vera.  Michaelis and Patel then consider the importance of aligning trial endpoints with the individualized treatment goals of patients. They conclude by discussing how the progression of a patient to second-line therapy, as well as cytogenetic abnormalities that indicate more aggressive disease progression, may underly the decision to consider clinical trials that align with a patient’s goals.

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During the 63rd ASH Annual Meeting and Exposition, the MPN Hub was pleased to speak to Laura Michaelis (Medical College of Wisconsin, Milwaukee, US) and Claire Harrison (Guy's and St Thomas' NHS Foundation Trust, London, UK). We asked, Patient follow-up in myeloproliferative neoplasms: Best practices and myths. 

In this podcast, Michaelis and Harrison discuss the best practices in MPN, in particular in regards to diagnosis. They also discuss the new data that was presented at ASH 2021, such as the MOMENTUM (NCT04173494) study and momelotinib.  

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