Category Archives: Polycythemia Vera

Dr Kuykendall on Frontline Cytoreductive Therapies in Polycythemia Vera

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October 20, 2023

Andrew Kuykendall, MD

Andrew Kuykendall, MD, assistant member, Department of Malignant Hematology, Moffitt Cancer Center, discusses frontline cytoreductive treatment options for patients with polycythemia vera.

In the management of polycythemia vera, the choice of treatment with frontline cytoreductive therapies is primarily between pegylated interferon alfa-2a or hydroxyurea, 2 agents that possess distinct characteristics, Kuykendall says. Pegylated interferon alfa-2a is administered subcutaneously and provides long-acting efficacy, whereas hydroxyurea is an oral agent with a shorter duration of action, Kuykendall explains. Additionally, although both agents are relatively well tolerated, their adverse event profiles differ from one another and inform the use of these agents in different patient populations, Kuykendall notes.

For instance, older patients with polycythemia vera typically receive hydroxyurea, whereas younger patients will often receive pegylated interferon alfa-2a, according to Kuykendall. Although both agents are reasonable frontline treatment options for patients with polycythemia vera, the rationales for the use of each agent differs, Kuykendall emphasizes.

A randomized phase 3 trial compared pegylated interferon alfa-2 vs hydroxyurea in patients with polycythemia vera and essential thrombocytopenia. In the patients with polycythemia vera, the 12-month complete response (CR) rates with pegylated interferon alfa-2 and hydroxyurea were 28% and 30%, respectively (P = .80). At 24 months, the CR rates were 25% with pegylated interferon alfa-2 and 17% with hydroxyurea. At 36 months, the CR rates with pegylated interferon alfa-2 and hydroxyurea were 29% and 17%, respectively. Additionally, of the patients who received post-baseline imaging for spleen response, the median spleen reduction was –6% (best response on treatment range, –37% to 54%) with pegylated interferon alfa-2 vs –5% (best response on treatment range, –24% to 17%) with hydroxyurea.

The use of pegylated interferon alfa-2 has increased over the past few years because of ongoing developments with this agent, including the advent of more tolerable formulations and its potential to elicit long-term disease modification, Kuykendall explains. This growing inclination toward using pegylated interferon alfa-2 underscores the ongoing interest in finding polycythemia vera treatments that can do more than simply reduce thrombotic potential for patients, Kuykendall says.

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Does the Use of Statins Improve Survival in Essential Thrombocythemia, Polycythemia Vera?

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By Patrick Daly – Last Updated: October 16, 2023

In patients with polycythemia vera and essential thrombocythemia, statins improved survival and decreased the risk of thrombosis after being diagnosed with myeloproliferative neoplasms (MPNs), according to a study published in Cancer Medicine.

“We found that among patients with [polycythemia vera] and [essential thrombocythemia], the use of statins improved survival and decreased risk of thrombosis after MPN diagnosis,” wrote the researchers, led by Nikolai Podoltsev, MD, PhD, of Yale University in New Haven, Connecticut

Dr. Podoltsev and colleagues noted that prior studies have suggested statins may improve the survival of patients with various cancers. They performed an analysis to characterize the effects of statins in older patients with polycythemia vera and essential thrombocythemia.

Based on their results, they suggested that this novel finding supports the use of statins “to address hyperlipidemia as one of the modifiable cardiovascular risk factors” in this group of patients. They also suggested that statins could be additionally relevant given the current use of ruxolitinib, which may lead to development or worsening of hypercholesterolemia.

Data Support Statin Use in Polycythemia Vera, Essential Thrombocythemia

Their conclusions were based on analysis of 1809 and 2201 older adults with polycythemia vera and essential thrombocythemia, respectively, in the Surveillance, Epidemiology, and End Results (SEER) database. Analysts used propensity score matching (PSM) and inverse probability of treatment weighting (IPTW) to evaluate the impact of statins on overall survival, and multivariable competing risk models to evaluate associations between statins and thrombosis risk.

Overall, 55.8% of patients used statins within the first year of polycythemia vera and essential thrombocythemia diagnosis. Over a median follow-up of 3.92 years (interquartile range, 2.58-5.75 years), statin use was associated with a 22% reduction in all-cause mortality (PSM hazard ratio [HR], 0.78; 95% CI, 0.63-0.98; P=.03; and IPTW HR, 0.79; 95% CI, 0.64-0.97; P=.03). Statin use was also shown to reduce the risk of thrombosis (PSM HR, 0.63; 95% CI, 0.51-0.78; P<.01; and IPTW HR, 0.57; 95% CI, 0.49-0.66; P<.01).

Noting that a randomized controlled trial of statins in patients with MPNs is unlikely, the investigators suggested that, “based on our results the recommendation can be made for hematologists taking care of patients with [polycythemia vera and essential thrombocythemia] to either be directly involved in or advocate for prescribing statins to these patients who are at a high risk for cardiovascular events.”

 Reference

Podoltsev NA, Wang R, Shallis RM, et al. Statin use, survival and incidence of thrombosis among older patients with polycythemia vera and essential thrombocythemia. Cancer Med. 2023;12(18):18889-18900. doi:10.1002/cam4.6528

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NCCN-Directed Guidelines Driving PV Treatment in Clinical Practice

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September 22, 2023

Aaron Gerds, MD, MS: The NCCN [National Comprehensive Cancer Network] guidelines for polycythemia vera and the other MPNs [myeloproliferative neoplasms] are updated regularly. So certainly there’s a large effort to annually update them, where we go over the entire sets of guidelines to update and refine them. But as new developments come along, we update on the fly. Say a new therapy is approved for polycythemia vera. We would quickly add that to the guidelines in an ad hoc update, just simply keeping the link to the NCCN and guidelines readily available, so whatever is on the NCCN guidelines website is the most up-to-date version. Printing it out and having it on your desk might [allow it to] become outdated at some point. So certainly relying heavily on the website. The NCCN also has apps available where the guidelines are automatically updated within the app, so you don’t have to worry about visiting the website or printing out new guidelines. And the NCCN, as well as other entities, support regular education efforts, both virtually and in person. There’s the annual NCCN Hematologic Malignancies conference, where the guidelines are a key piece of that, where updates are given not only within the disease state, but specifically with an eye to the guidelines. Within the MPN world, there are lots of conferences and educational opportunities as well, both virtually and in-person. The big event here in North America is the American Society Hematology annual meeting, where updates are given within the disease field. But, certainly, looking toward the NCCN, the resources available there can keep you up to date, especially a lot of people have found the app to be very, very helpful.

Adherence to the NCCN guidelines for polycythemia vera is actually kind of a chicken and the egg issue. What came first? I think NCCN guidelines reflected everyday practice of taking care of patients with polycythemia vera before they were invented. So the MPN guidelines in general are relatively new compared to other guidelines for breast cancer or colon cancer. At that time, we focused on what the current practice was. Identifying patients [as] high risk, low risk,…reductive therapies in the high-risk cases, and so on and so forth. Now, over time, we’ve had to adapt the guidelines for new therapies, in particular the approval of ropeginterferon, and incorporate that in additional data has been published about ruxolitinib, particularly the MAJIC-PV trial. We’ve incorporated this information into the guidelines, which has altered or strengthened some of our recommendations. So certainly I think the guidelines…will help guide someone learning how to take care of patients with polycythemia vera. But I think it really does reflect the best practice in how practice is sustained throughout North America. So it’s kind of a bidirectional effort, where absorbing what is currently the standard of care practice [and] putting [it] in the guidelines to then help unify practice throughout the country. And while there [is] some data out there that looks at adherence to the guidelines in everyday practice, I would say that polycythemia vera is actually one of the ones that is a little bit more stringent. There are fewer treatment options. Kind of clear, low risk, high risk, “what do you do” process. So I think it’s a lot easier to take those guidelines and adhere to them closely.

Transcript is AI-generated and edited for clarity and readability.

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MWTX-003 Wins FDA Fast Track Designation for Polycythemia Vera

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Kristi Rosa

The FDA has granted fast track designation to the investigational, anti-TMPRSS6 monoclonal antibody, MWTX-003 (DISC-3405), for use in the treatment of patients with polycythemia vera, according to an announcement from Disc Medicine, Inc.1

Patients with hematologic diseases such as polycythemia vera, myelodysplastic syndrome (MDS), and beta-thalassemia are known to develop high levels of iron, which leads to survival and quality-of-life complications.2 MWTX-003 was designed to boost the production of hepcidin, which suppresses serum iron. Preclinical data in animal models of beta-thalassemia and polycythemia vera have confirmed this ability.

“We are delighted to have received fast track designation for MWTX-003, which highlights the unmet need for [patients with] polycythemia vera and the potential of MWTX-003 in a disease where there are few treatment options,” John Quisel, JD, PhD, president and chief executive officer of Disc Medicine, Inc., stated in a press release.1 “We believe MWTX-003 is uniquely positioned to address the needs of [patients with] polycythemia vera and are excited to initiate a phase 1 trial in the coming months.”

Preclinical studies have demonstrated strong pharmacodynamic effects that are reflective of TMPRSS6 inhibition.3 Specifically, a single administration of MWTX-003 led to an approximate 70% suppression of serum iron that lasted for 3 weeks. Moreover, in non-clinical GLP safety studies, the agent showcased a strong toxicity profile.

In a model of beta-thalassemia, treatment with MWTX-003 resulted in significant effects on disease hallmarks such as iron overload, ineffective erythropoiesis, and splenomegaly. The production of hepcidin was boosted up to 4-fold, serum and liver iron was reduced by approximately 60% to 65%, red blood cell production increased, and spleen weight decreased.

MWTX-003 was in-licensed from Mabwell Therapeutics, and in November 2022, the FDA accepted an investigational new drug application for the agent.1 In January 2023, the clinical-stage biopharmaceutical company shared development plans for MWTX-003 which consisted of establishing phase 1 proof-of-mechanism; this was planned for initiation in the second half of 2023, and would examine hepcidin, iron, and other hematologic parameters.3

They also shared plans to advance the agent into point-of-care studies focused on polycythemia vera. In a phase 1b/2a proof-of-concept study, they hope to evaluate the safety and pharmacokinetic profile of MWTX-003 in patients with polycythemia vera. These data could provide clarity on the regulatory development path for the agent, according to Disc Medicine.

There is interest in examining the agent in additional POC studies spanning a range of indications, including hereditary hemochromatosis, beta-thalassemia, and MDS.

References

  1. Disc Medicine receives FDA fast track designation for MWTX-003 for the treatment of polycythemia vera. News release. Disc Medicine, Inc. September 20, 2023. Accessed September 21, 2023. https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-receives-fda-fast-track-designation-mwtx-003
  2. MWTX-003. Disc Medicine, Inc. website. Accessed September 21, 2023. https://www.discmedicine.com/our-pipeline/mat-2-inhibitor/
  3. Novel anti-TMPRSS6 monoclonal antibody portfolio: exclusive in-licensing agreement with Mabwell Therapeutics. Disc Medicine, Inc. January 20, 2023. Accessed September 21, 2023. https://ir.discmedicine.com/static-files/549caf12-e7be-45ff-8667-86908e4e6bdd

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Ruxolitinib Receives Positive NICE Opinion for Polycythemia Vera

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Russ Conroy

The National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending the approval of ruxolitinib (Jakafi) as a treatment for adult patients with polycythemia vera that is intolerant or resistant to hydroxycarbamide or hydroxyurea, according to a press release from Novartis.1 This may help to make the agent available for patients residing in England and Wales.

“We welcome this recommendation from NICE, as polycythemia vera can be an extremely debilitating illness that has a significant impact on patients’ lives in terms of day-to-day symptoms,” Jon Mathias, co-chair of MPN Voice, said in the press release. “Ruxolitinib addresses a significant unmet need in patients who cannot tolerate or no longer respond to [hydroxycarbamide/hydroxyurea].”

According to a previous report, treatment with hydroxycarbamide or hydroxyurea leads to the development of resistance or intolerance in 24% of patients with polycythemia vera, which correlates with a higher risk of disease progression.2

In an international MPN Landmark survey, 72% of patients with polycythemia vera reported that they experienced reduced quality of life (QOL) due to disease symptoms.3 Additionally, 14.0% of surveyed patients with polycythemia vera reported that they were experiencing emotional hardship due to their condition, and 29.0% stated that they felt worried or anxious about their disease. A further 33.0% of the surveyed polycythemia vera population stated that they experienced impairment at work, and 40.3% reported impairment with respect to overall activity.

“There is a significant unmet need for people with polycythemia vera in England and Wales, who live with a large symptom burden as a result of their condition,” Claire Harrison, MD, FRCP, FRCPath, consultant hematologist at Guy’s and St Thomas’ NHS Foundation Trust in London, said in the press release.1 “Today’s decision is a step in the right direction for providing additional treatment options that reduce the burden of these symptoms and improve disease progression, in this under-represented patient population.”

Investigators sent the MPN LANDMARK survey to patients with polycythemia vera, myelofibrosis, and essential thrombocythemia, and physicians across the United Kingdom, Australia, Germany, Canada, Japan, and Italy from April 2016 to October 2016. The survey was designed to evaluate how MPNs affected QOL, patients’ ability to work, and implementation of disease-management strategies.

Patients 18 years and older diagnosed with myelofibrosis, polycythemia vera, or essential thrombocytopenia were able to respond to the survey. Those enrolled on clinical trials were not eligible to take the survey.

Overall, 219 physicians and 699 patients—including 174 with myelofibrosis, 223 with polycythemia vera, and 302 with essential thrombocythemia—completed the survey.

The FDA approved ruxolitinib as a treatment for patients with polycythemia vera who are intolerant to hydroxyurea in December 2014.4 Supporting data for the FDA approval came from the phase 3 RESPONSE trial (NCT01243944), in which treatment with ruxolitinib produced improvements in hematocrit control and spleen volume reductions compared with best available therapy. Additionally, a higher number of patients receiving ruxolitinib experienced complete hematologic remission. Investigators reported that the most common hematologic adverse effects included thrombocytopenia and anemia.

References

  1. NICE recommends Novartis Jakavi® (ruxolitinib) for patients living with polycythaemia vera (PV). News release. Novartis. September 14, 2023. Accessed September 15, 2023. https://shorturl.at/hixAD
  2. Alvarez-Larran A, Pereira A, Cervantes F, et al. Assessment and prognostic value of the European LeukemiaNet Criteria for clinicohematologic response, resistance, and intolerance to hydroxyurea in polycythemia vera. Blood. 2012;119(6):1363-1369. Doi:10.1182/blood-2011-10-387787
  3. Harrison CN, Koschmieder S, Foltz L, et al. The impact of myeloproliferative neoplasms (MPNs) on patient quality of life and productivity: results from the international MPN Landmark survey. Ann Hematol. 2017;96(10):1653-1665. doi:10.1007/s00277-017-3082-y
  4. FDA approves Jakafi® (ruxolitinib) for the treatment of patients with uncontrolled polycythemia vera. News release. Incyte Corporation. December 4, 2014. Accessed September 15, 2023. https://shorturl.at/aARU3

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PharmaEssentia and MPN Advocacy & Education International Launch New Educational Initiative to Empower People Living With Polycythemia Vera (PV)

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Published: Sep 14, 2023

PV&ME™ campaign features personal stories from people living with PV and their journeys navigating the rare blood cancer

BURLINGTON, Mass. & EAST LANSING, Mich.–(BUSINESS WIRE)– PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, and MPN Advocacy & Education International, a leading advocacy group dedicated to providing the knowledge, support and resources patients will need as they adjust to living with a myeloproliferative neoplasm (MPN), today announced a new educational initiative for the MPN community called PV&ME. The goal of the campaign is to bring to light the unique and challenging experiences of living with polycythemia vera (PV) in the hopes of raising awareness, empowering patients to advocate for themselves and ensuring newly diagnosed patients feel supported in their journeys. PV&ME features the stories of four inspiring individuals – Buzz, Deb, Patti and Steven – living with this chronic cancer and their perspectives on navigating diagnosis, addressing burdensome symptoms and seeking comprehensive care.

PV is the most common MPN and a long-term, potentially life-threatening cancer that has had limited treatment options for many years. Patients with PV are at a more significant increased risk of developing thromboembolic events than the general population with cardiovascular disease, due to increased blood cell counts. They also have a long-term risk of progression to myelofibrosis or transformation to acute myeloid leukemia.1-5

“People living with PV often face feelings of isolation as they navigate a long and confusing road to diagnosis and adjust to extreme fatigue or other often debilitating symptoms,” said Ann Brazeau, Chief Executive Officer, MPN Advocacy & Education International. “The stories shared in this new PV&ME campaign show just how important the right support and resources can be for this community. We hope this new initiative will help people with PV feel connected and empowered to advocate for themselves on their PV journeys.”

“At PharmaEssentia, we are committed to being an essential partner for the MPN community and know that a critical component of that is listening to and amplifying stories from individuals living with PV themselves,” said Raymond Urbanski, M.D., Ph.D., Senior Vice President and U.S. Head of Clinical Development and Medical Affairs at PharmaEssentia. “This MPN Awareness Day, we are proud to partner with MPN Advocacy & Education International to share these inspiring stories with the MPN community and help encourage patients to take a proactive approach in their care.”

The PV&ME educational video series launched on MPN Awareness Day (September 14) and can be found by visiting us.pharmaessentia.com/patients/patient-stories/. Throughout Blood Cancer Awareness Month, PharmaEssentia and MPN Advocacy & Education International will continue to share important educational content for the MPN community.

Follow PharmaEssentia USA on Twitter and LinkedIn for news and updates.

About Polycythemia Vera (PV)

Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.6

About PharmaEssentia

PharmaEssentia (TPEx: 6446), headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology and oncology, with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan.

For more information about PharmaEssentia USA, visit the website, LinkedIn or Twitter.

About MPN Advocacy & Education International

MPN Advocacy and Education International provides educational programs, materials, and resources for patients, caregivers, physicians, and entire healthcare teams to improve their understanding of myelofibrosis, polycythemia vera, and essential thrombocythemia. They are dedicated to making a difference in the lives of those affected by MPNs and strive to grow awareness and advocate on behalf of the MPN community.

For more information about MPN Advocacy and Education International, visit the website, Facebook or Twitter.

© 2023 PharmaEssentia Corporation. All rights reserved.

PharmaEssentia, the PharmaEssentia logo, and PV&ME are trademarks or registered trademarks of PharmaEssentia Corporation.

1 Griesshammer M, Kiladjian J-J, Besses C. Thromboembolic events in polycythemia vera. Ann Hematol. 2019;98:1071–82. DOI: 10.1007/s00277-019-03625-x
2 Antithrombotic Trialists (ATT) Collaboration. Aspirin in the primary and secondary prevention of vascular disease: collaborative meta-analysis of individual participant data from randomised trials. Lancet. 2009;373:1849-1860. https://doi.org/10.1016/S0140-6736(09)60503-1
3 Yusef S, et al. Cholesterol lowering in intermediate-risk persons without cardiovascular disease. NEJM. 2016;374(21):2021-2031. DOI: 10.1056/NEJMoa1600176
4 Risk and Prevention Study Collaborative Group;​ Roncaglioni M, et al. N-3 fatty acids in patients with multiple cardiovascular risk factors. NEJM. 2013;368:1800-1808. DOI: 10.1056/NEJMoa1205409
5 Barbui T, et al. In contemporary patients with polycythemia vera, rates of thrombosis and risk factors delineate a new clinical epidemiology. Blood. 2014;124:3021-3023. https://doi.org/10.1182/blood-2014-07-591610
6 Cerquozzi S, Tefferi A. Blast transformation and fibrotic progression in polycythemia vera and essential thrombocythemia: a literature review of incidence and risk factors. Blood Cancer J. 2015;5, e366; DOI:10.1038/bcj.2015.95

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PharmaEssentia and MPN Advocacy & Education International Launch New Educational Initiative to Empower People Living With Polycythemia Vera (PV)

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PV&ME™ campaign features personal stories from people living with PV and their journeys navigating the rare blood cancer

BURLINGTON, Mass. & EAST LANSING, Mich.–(BUSINESS WIRE)–PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, and MPN Advocacy & Education International, a leading advocacy group dedicated to providing the knowledge, support and resources patients will need as they adjust to living with a myeloproliferative neoplasm (MPN), today announced a new educational initiative for the MPN community called PV&ME. The goal of the campaign is to bring to light the unique and challenging experiences of living with polycythemia vera (PV) in the hopes of raising awareness, empowering patients to advocate for themselves and ensuring newly diagnosed patients feel supported in their journeys. PV&ME features the stories of four inspiring individuals – Buzz, Deb, Patti and Steven – living with this chronic cancer and their perspectives on navigating diagnosis, addressing burdensome symptoms and seeking comprehensive care.

PV is the most common MPN and a long-term, potentially life-threatening cancer that has had limited treatment options for many years. Patients with PV are at a more significant increased risk of developing thromboembolic events than the general population with cardiovascular disease, due to increased blood cell counts. They also have a long-term risk of progression to myelofibrosis or transformation to acute myeloid leukemia.1-5

“People living with PV often face feelings of isolation as they navigate a long and confusing road to diagnosis and adjust to extreme fatigue or other often debilitating symptoms,” said Ann Brazeau, Chief Executive Officer, MPN Advocacy & Education International. “The stories shared in this new PV&ME campaign show just how important the right support and resources can be for this community. We hope this new initiative will help people with PV feel connected and empowered to advocate for themselves on their PV journeys.”

“At PharmaEssentia, we are committed to being an essential partner for the MPN community and know that a critical component of that is listening to and amplifying stories from individuals living with PV themselves,” said Raymond Urbanski, M.D., Ph.D., Senior Vice President and U.S. Head of Clinical Development and Medical Affairs at PharmaEssentia. “This MPN Awareness Day, we are proud to partner with MPN Advocacy & Education International to share these inspiring stories with the MPN community and help encourage patients to take a proactive approach in their care.”

The PV&ME educational video series launched on MPN Awareness Day (September 14) and can be found by visiting us.pharmaessentia.com/patients/patient-stories/. Throughout Blood Cancer Awareness Month, PharmaEssentia and MPN Advocacy & Education International will continue to share important educational content for the MPN community.

Follow PharmaEssentia USA on Twitter and LinkedIn for news and updates.

About Polycythemia Vera (PV)

Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.6

About PharmaEssentia

PharmaEssentia (TPEx: 6446), headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology and oncology, with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan.

For more information about PharmaEssentia USA, visit the website, LinkedIn or Twitter.

About MPN Advocacy & Education International

MPN Advocacy and Education International provides educational programs, materials, and resources for patients, caregivers, physicians, and entire healthcare teams to improve their understanding of myelofibrosis, polycythemia vera, and essential thrombocythemia. They are dedicated to making a difference in the lives of those affected by MPNs and strive to grow awareness and advocate on behalf of the MPN community.

For more information about MPN Advocacy and Education International, visit the website, Facebook or Twitter.

© 2023 PharmaEssentia Corporation. All rights reserved.

PharmaEssentia, the PharmaEssentia logo, and PV&ME are trademarks or registered trademarks of PharmaEssentia Corporation.

1 Griesshammer M, Kiladjian J-J, Besses C. Thromboembolic events in polycythemia vera. Ann Hematol. 2019;98:1071–82. DOI: 10.1007/s00277-019-03625-x
2 Antithrombotic Trialists (ATT) Collaboration. Aspirin in the primary and secondary prevention of vascular disease: collaborative meta-analysis of individual participant data from randomised trials. Lancet. 2009;373:1849-1860. https://doi.org/10.1016/S0140-6736(09)60503-1
3 Yusef S, et al. Cholesterol lowering in intermediate-risk persons without cardiovascular disease. NEJM. 2016;374(21):2021-2031. DOI: 10.1056/NEJMoa1600176
4 Risk and Prevention Study Collaborative Group;​ Roncaglioni M, et al. N-3 fatty acids in patients with multiple cardiovascular risk factors. NEJM. 2013;368:1800-1808. DOI: 10.1056/NEJMoa1205409
5 Barbui T, et al. In contemporary patients with polycythemia vera, rates of thrombosis and risk factors delineate a new clinical epidemiology. Blood. 2014;124:3021-3023. https://doi.org/10.1182/blood-2014-07-591610
6 Cerquozzi S, Tefferi A. Blast transformation and fibrotic progression in polycythemia vera and essential thrombocythemia: a literature review of incidence and risk factors. Blood Cancer J. 2015;5, e366; DOI:10.1038/bcj.2015.95

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Patients With Polycythemia Vera Seek Guidance on Exercise

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August 23, 2023

Andrea S. Blevins Primeau, PhD, MBA

In a multicenter survey, patients with polycythemia vera (PV) shared their preferences and needs regarding physical activity (PA), including the frequency of PA per week and the type and location of activity. Notably, lower educational level was associated with higher levels of inactivity.

The study, which was published in the journal Cancer Medicine, surveyed 182 adult patients with PV about their disease burden and PA preferences and needs. The mean age of the cohort was 61 and 68% of patients were female. There were 57% of patients with more than 10 years of education and 48% were currently working. The mean time since diagnosis was 8 years.

Moderate-to-severe symptoms were present among 60% of patients, which most commonly included fatigue, concentration problems, and bone/muscle pain. Skin reactions, splenomegaly, tendency to bleed, and weight gain were also commonly reported symptoms.

There were 67% of patients who reported that they would like more information about PA. Patients with a lower educational level were significantly less likely to be engaged in PA at 50% compared with 69% of patients with a higher education level (P =.021).

Patients who were currently inactive preferred PA sessions 1 to 2 times per week for a duration of 15 to 45 minutes. Patients who already exercised, preferred PA 3 to 4 times per week for a duration of 30 to 60 minutes.

The majority of patients preferred individual training at 79%, whereas 40% preferred group training. The most common location that was preferred for PA was outdoors at 79% followed by at home at 56%. There was no significant difference in training setting or location among patients who were currently active or inactive.

The authors provided recommendations for the frequency, intensity, duration, and type of exercise for patient, as well as special recommendations depending on the symptom or side effect that an individual is experiencing. For example, yoga or tai-chi was recommended for patients with fatigue or concentration problems, whereas endurance training or yoga was recommended for patients with anxiety or depression.

Reference
Felser S, Rogahn J, Hollenbach L, et al. Physical exercise recommendations for patients with polycythemia vera based on preferences identified in a large international patient survey study of the East German Study Group for Hematology and Oncology (OSHO #97). Cancer Med. Published online August 9, 2023. doi: 10.1002/cam4.6413

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Optimizing Hydroxyurea Management Among Patients With Polycythemia Vera

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August 8, 2023

Amber Denham

Results of a retrospective, real-world analysis determined that many patients with chronic myeloproliferative neoplasm (MPN) polycythemia vera (PV) received underdosed hydroxyurea, which led to lower complete response and toxicity rates, as well as proceeded with hydroxyurea regardless of poor clinical or hematological responses. These results highlight the need for more optimized [hydroxyurea] management and intervention in less-than-optimal responders.

“Hydroxyurea is currently the most used cytoreductive therapy for PV patients at high risk of thrombosis, with most patients achieving adequate control of the disease with acceptable tolerance,” Francesca Palandri, MD, Universitaria di Bologna, Bologna, Italy, and coauthors explained. “However, many patients may only obtain a poor response to [hydroxyurea] or develop drug-related toxicities during therapy.”

This study sought to define clinical characteristics associated with the achievement of complete response (CR) to hydroxyurea, investigate whether the type of suboptimal response may influence a decision to switch to ruxolitinib, and assess if a patient achieving CR to hydroxyurea would improve outcome parameters. The analysis included 563 patients with polycythemia vera treated with hydroxyurea for ≥12 months during an observational “PV-NET” Italian study. Among this patient population, 166 patients achieved complete response (CR), 264 achieved partial response (PR), and 133 achieved no response (NR).

In a multivariate analysis, the absence of splenomegaly (P = 0.03), pruritus (P = 0.002), and a median hydroxyurea dose of ≥1 g/day (P < 0.001) remained associated with CR. Overall, 283 patients who received either CR or PR continued hydroxyurea, and 114 patients switched to ruxolitinib. It was noted that many patients continued hydroxyurea despite a PR/NR and that splenomegaly and other symptoms were the main drivers of an early switch.

A median hydroxyurea dose of ≥1 g/day was correlated with more frequent adverse events. In the 449 patients who were receiving only hydroxyurea, rates of thrombosis, hemorrhages, progression, and overall survival (OS) were comparable among the CR, PR, and NR groups.

Palandri et al concluded, “Better [hydroxyurea] management, standardization of the criteria for and timing of responses to [hydroxyurea], and adequate intervention in poor responders should be advised.”

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New Study Demonstrates Ropeginterferon Alfa-2b-njft Is a Cost-Effective Treatment Option for a Broad Range of Patients with Polycythemia Vera

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Published August 4, 2023

Analysis published in the Journal of Comparative Effectiveness Research shows ropeginterferon alfa-2b-njft provided a cost-effective benefit in quality-adjusted life years compared to a commonly used treatment pathway

BURLINGTON, Mass.–(BUSINESS WIRE)– PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced the publication of a cost-effectiveness analysis of ropeginterferon alfa-2b-njft (marketed as BESREMi®) in the Journal of Comparative Effectiveness Research. The analysis, titled “Cost-Effectiveness of Ropeginterferon Alfa-2b-njft for the Treatment of Polycythemia Vera,” showed that ropeginterferon alfa-2b-njft provided a cost-effective benefit for a broad range of patients with polycythemia vera (PV) versus first-line hydroxyurea followed by ruxolitinib. Cost effectiveness was demonstrated in a modeled population including both low- and high-risk patients receiving first- or second-line treatment with ropeginterferon alfa-2b-njft.

“PV is a chronic blood cancer that requires lifelong therapy, which can prevent or delay negative clinical outcomes, but is also associated with additional costs to patients and payers. These new data demonstrate ropeginterferon alfa-2b-njft’s value not only to people living with PV, but also to the healthcare system at large,” said Dr. Aaron Gerds, MD, MS, Medical Director at Case Comprehensive Cancer Center and lead study author. “This study shows ropeginterferon alfa-2b-njft is a cost-effective treatment option over the modeled lifetime and that earlier initiation of treatment of PV with effective therapy can translate to more favorable cost to benefit ratios.”

PV is the most common myeloproliferative neoplasm (MPN) and a long-term, potentially life-threatening disease with limited approved treatment options. Patients with PV are at risk for disease progression to myelofibrosis (post-PV MF) or transformation to blast phase (MPN-BP) which is akin to acute myeloid leukemia1-3. Patients are also at an increased risk for arterial and venous thromboembolic events (TEs) which are associated with higher mortality, lower quality of life, and higher healthcare costs4-7.

This new study used an economic model developed from the United States healthcare system perspective. Inputs were informed by the data from randomized clinical trials, including the PROUD-PV and CONTINUATION-PV studies, and from real-world sources. The model compared ropeginterferon alfa-2b-njft used either as first- or second-line therapy versus an alternative treatment pathway of first-line hydroxyurea followed by ruxolitinib. To reflect the long-term consequences of treating PV, results were presented over a lifetime horizon.

Findings from the study conclude ropeginterferon alfa-2b-njft is a cost-effective treatment option for a broad range of patients with PV, including both low- and high-risk patients and patients with and without prior cytoreductive treatment with hydroxyurea. Of note:

  • These data show that over the modeled lifetime, patients who receive ropeginterferon alfa-2b-njft have more years alive (0.4), higher quality-adjusted life years (QALYs) (0.4), and higher cost ($60,175) as compared to the alternative treatment pathway. Weighing the additional costs versus the additional QALY gains results in a cost per QALY of $141,783.
  • This cost per QALY is less than a standard willingness to pay threshold of $150,000 per QALY8.
  • In this study, treating patients at a younger age or those with low-risk disease led to more cost-effective results, suggesting that earlier initiation of treatment of PV with effective therapy can translate to more favorable cost to benefit ratios.
  • The model was sensitive to treatment costs, the percentage of patients who discontinue hydroxyurea, the percentage of ropeginterferon alfa-2b-njft users who switch to monthly dosing, the percentage of ropeginterferon alfa-2b-njft users as 2nd line treatment, and the treatment response rates.

“Ropeginterferon alfa-2b-njft has demonstrated safety and efficacy in studies including both low- and high-risk patients and patients with and without prior cytoreductive treatment with HU. Recently, the National Comprehensive Cancer Network (NCCN) updated their treatment guidelines to include ropeginterferon as a preferred treatment regimen for both low- and high-risk PV patients9. As ropeginterferon continues to be more widely used in clinical practice, this study was imperative to demonstrate the cost-effectiveness of ropeginterferon alfa-2b-njft for a broad range of patients with PV,” said Raymond Urbanski, M.D., Ph.D., Senior Vice President and U.S. Head of Clinical Development and Medical Affairs at PharmaEssentia. “This analysis also underscores the importance of treating low-risk patients and patients early in their disease journey to help minimize more severe events and their corresponding costs to the healthcare system.”

The full benefits of ropeginterferon alfa-2b-njft may not be fully captured in the model, and in particular, data on disutility of phlebotomy are lacking. Although some patients may tolerate regular phlebotomies, others can experience iron deficiency which can negatively impact quality of life. While the results from a scenario analysis incorporating a small decrement in utility had minimal impact on the cost-effectiveness results, due to the lack of data in this area, this estimate remains conservative. This study took a U.S. healthcare perspective, and the results may not generalize to other countries given the differences in healthcare resource use, costs and cost-effectiveness thresholds.

About Polycythemia Vera (PV)

Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.10

About BESREMi® (ropeginterferon alfa-2b-njft)

BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients.

BESREMi has orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. The product was approved by the European Medicines Agency (EMA) in 2019, in the United States in 2021, and has recently received approval in Taiwan and South Korea. The drug candidate was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications.

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