Hydration and Movement Are Important for Patients With MPNs

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October 18, 2023

Alex Biese

Patients with myeloproliferative neoplasms (MPNs) — a group of blood cancers that causes the bone marrow to overproducer red or white blood cells or platelets and which includes polycythemia vera, essential thrombocythemia and myelofibrosis) — are at particular risk for thrombotic and cardiovascular events.

“It’s really a problem for all patients with cancer because it does increase your chances of developing a clot, for example,” said Kim Noonan, nursing and patient care services chief nurse practitioner at the Dana-Farber Cancer Institute in Boston, during a conversation about the risks faced by patients, and the ways in which nurses can help patients prevent cardiovascular and thrombotic events.

However, such risks are of particular concern for patients with MPNs, as literature has shown.

“Clinicians have long recognized an association between MPNs and increased risk for both thrombotic and hemorrhagic complications,” stated the authors of an article published in JACC: CardioOncology. “A recent meta-analysis of 13,436 patients with MPNs showed a pooled prevalence of arterial thrombosis of 16.2% and of hemorrhagic complications of 6.2%. Indeed, cardiovascular events often accompany this cluster of hematologic conditions and contribute significantly to morbidity and mortality.”

Symptoms of a clot or pulmonary embolus include shortness of breath and chest pain, especially following exertion, as well as an elevated or irregular heart rate and an unexplained fever noted Noonan, the co-author of a Journal of Oncology Nursing study on the assessment and prevention of venous thromboembolism and cardiovascular disease among patients with another form of blood cancer, multiple myeloma.

“I’m always thinking about thrombosis first, and then I can relax if I have maybe another explanation for their shortness of breath,” Noonan said. “But we’re always working it up, we really do due diligence to not miss some kind of thrombotic event that’s going on.”

Transcript:
I think when people see me, they just feel like they have to start jogging, because I’m always saying, “You (have) got to move, you have to move, you have to move.” And it’s so, so important. That person that is sedentary is really at a huge risk to the point of where if they have a sedentary lifestyle, we’re thinking maybe that person needs to be on needs to be on anticoagulant therapy right from the beginning, as opposed to just using an aspirin.

We really want people to stay hydrated. We want them to get up and walk around. We want them to be aware of what the symptoms are (and be aware that) they can be doing everything right and still develop a clot because of the medication that they’re on. But I think also, education is essential, (letting a patient know) that you are on a medication that can increase your chances of developing a clot or thrombosis, and (you should) just be aware of what the symptoms are.

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Advancing the Treatment Landscape for Patients With Myeloproliferative Neoplasms

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Pearl Steinzor

Despite recent advancements in treatments for patients with myeloproliferative neoplasms, a term encompassing several rare cancers, many challenges persist when it comes to these hematologic malignancies, primarily due to lack of uniform treatment plans for each of the classical subgroups.

These findings were presented in a landscape analysis by the Association of Community Cancer Centers (ACCC) at the 2023 National Oncology Conference from October 4-6, 2023, in Austin, Texas.

In 2008, the World Health Organization (WHO) reclassified myeloproliferative disease from a blood disorder to clonal hematopoietic stem cell malignancies, and in 2016, the WHO identified 4 classic types of myeloproliferative neoplasms: chronic myeloid leukemia (BCR-ABL1 positive), polycythemia vera (BCR-ABL1 negative), essential thrombocythemia (BCR-ABL1 negative), and primary myelofibrosis (ABCR-ABL1 negative).

Patients with myeloproliferative neoplasms report debilitating symptoms, including fevers, night sweats, fatigue, sleep disturbances, weight loss, bone pain, itchy skin, headaches, difficulty concentrating, anxiety, and depressive symptoms.

Although these patients are considered to have favorable life expectancy, with 60% of patients living up to 15 years after diagnosis, about 84% of these patients have reported a reduced quality of life directly due to these symptoms. Moreover, many low-risk patients are given a “watch-and-wait” treatment plan and receive no drug therapy despite experiencing a moderate to high symptom burden.

Gaps in care coordination also exist, and current standard-of-care pharmacological treatments are unable to fully relieve symptom burden. For example, approximately 84% of patients with myeloproliferative neoplasms reported reduced quality of life due to the effects of their current pharmacological interventions.

Moreover, patient and physicians perceptions regarding treatment are often misaligned, with patients reporting that their most important treatment goal was to slow or delay disease progression, whereas physicians reported the most important goal was symptom improvement and prevention of vascular or thrombotic events. Furthermore, an average of 30% of patients with myeloproliferative neoplasms did not believe their physician had a treatment plan, and an average of 35% of patients believed their physician was not providing updates on new treatments.

The researchers also identified health literacy as an important factor in understanding and managing symptom burdens, shared decision-making, and proper disease management. Additionally, a 2016 online survey of 904 adults found significant employment disruptions were common, with at least 1 employment change reported among 65.5% of patients with myelofibrosis, 48% of patients with polycythemia vera, and 38.8% of patients with essential thrombocythemia. Furthermore, respondents reported reduced working hours, medical disability leave, early retirement, dropping to part-time hours, and switching to a lower-paying job due to their disease.

For these reasons, myeloproliferative neoplasms have a significant disease burden and impact on patients, commonly due to anxieties about disease advancement and disruptive symptoms that affect a person’s quality of life.

Furthermore, lack of uniform treatment, risk diagnostic assessments, and care coordination can result in late diagnosis and rapid potential disease progression, which may also lead to a lack of transparency and patients feeling excluded in decision-making of their care.

Moreover, a lack of awareness surrounding available care guidelines, standardized symptom alleviation, and disease progression control plans are stressors that add to this disease burden, so further knowledge on the management of myeloproliferative neoplasms and related symptoms is necessary to improve patient outcomes.

Reference

Advancing care for patients with myeloproliferative neoplasms. Association of Community Cancer Centers. Accessed October 17, 2023. https://www.accc-cancer.org/home/learn/cancer-types/hematologic-malignancies/advancing-care-for-patients-with-myeloproliferative-neoplasms.

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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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Bomedemstat as an investigative treatment for myeloproliferative neoplasms

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By Hugh Young Rienhoff Jr. & Harinder Gill

Abstract

Introduction

Myeloproliferative neoplasm (MPN) is a heterogeneous group of hematopoietic stem cell disorders characterized by clonal proliferation of one of more of the hematopoietic stem cell lineages. Clinical manifestations result from uncontrolled myeloproliferation, extramedullary hematopoiesis with splenomegaly and excessive inflammatory cytokine production. Currently available therapy improves hematologic parameters and symptoms but does not adequately address the underlying neoplastic biology. Bomedemstat has thus far demonstrated clinical efficacy and tolerability in the treatment of MPNs with recent evidence of impacting the malignant stem cell population.

Areas covered

This review summarizes the mechanisms of action, pharmacokinetics and pharmacodynamics, safety and efficacy of bomedemstat in MPN with specific emphasis on essential thrombocythemia (ET) and myelofibrosis (MF).

Expert opinion

In patients with MPNs, bomedemstat appears effective and well tolerated. The signs and symptoms of these diseases are managed as a reduction in the frequency of mutant cells was demonstrated in patients with ET and MF. Ongoing and planned studies of bomedemstat in MPN will establish the position of bomedemstat in MPNs and may help to redefine treatment endpoints of MPNs in the future.

KEYWORDS:

  • Bomedemstat
  • lysine-specific demethylase-1
  • LSD1
  • myeloproliferative neoplasm
  • polycythemia vera
  • essential thrombocythemia
  • myelofibrosis

Article highlights

  • Myeloproliferative neoplasm (MPN) are clonal hematopoietic stem cell disorders characterized by uncontrolled cellular proliferation, cytokine mediated symptoms and clonal instabilty leading to leukemic progression.

  • Conventional therapeutic approaches in MPN are not adequate in altering the underlying disease biology.

  • Lysine specific demethylase 1 (LSD1) is overexpressed in the malignant hematopoietic stem cell population in MPN.

  • Bomedemstat, an inihibitor of MPN, effectively controls cellular proliferation and reverses disease biology in mouse models of MPN.

  • Phase 2 studies in essential thrombocythemia and myelofibrosis has demonstrated that Bomedemstat is highly efficacious and safe.

Declarations of interests

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

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Blood Cancer Symptoms That Shouldn’t Be Ignored: Early Warning Signs and Diagnosis

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Published on October 9, 2023

By Dr. Suraj D Chiraniya, Clinical Hematologist,  Hemato-oncologist and BMT Physician, HCG Cancer Centre, Borivali

A type of cancer that affects the blood cells. Some common types of blood cancer include leukaemia, lymphoma, and myeloma. Though rare, Myelodysplastic Syndromes (MDS) and Myeloproliferative Neoplasms (MPN) are some other types of blood cancer.

Caused by mutations in DNA within the blood cells, blood cancer results in blood cells behaving abnormally. Treatment, symptoms, and prognosis will vary depending on the type of blood cancer. Further, some types of blood cancer affect children, for which treatment can vary.

Understanding The Various Types Of Blood Cancer

One of the most common types of blood cancer affecting most blood cancer patients is leukaemia, defined as cancer of the blood cells. There are three main categories of blood cells: Red Blood Cells (RBC), White Blood Cells (WBC) and platelets. Leukaemia affects the WBC within the bloodstream and causes them to divide rapidly and eventually crowd out the normal cells. As a result of this, the WBC is unable to perform its function of fighting infections within the human body.

Lymphoma is another type of blood cancer that affects the human immune system. It specifically affects white blood cells called lymphocytes, a vital part of the immune system. Lymphoma can also be called a cancer of the lymphatic system or lymphatic cancer.

In general, there are two main types of lymphoma, namely Hodgkin lymphoma and non-Hodgkin lymphoma, which both affect the blood’s lymphocytes.

The third most common type of blood cancer is myeloma, which affects the blood’s plasma cells. Plasma cells help fight infections in the body, and when a person has myeloma, the body cannot make these antibodies properly. Additionally, abnormal cells multiply in this situation, causing various symptoms.

The majority of the people diagnosed with myeloma are men over the age of 70, although cases of it occurring in young men are also possible.

Who is at risk for blood cancer?

Although there are no specific conditions for blood cancer, the risk can sometimes increase with age. Further, a family history of this disease can also increase the risk.

In addition to the above, the risk of leukaemia can also increase due to the following factors:

•            exposure to radiation

•            treatment with certain chemotherapy drugs

•            a past diagnosis of blood cancer

•            Down syndrome and other genetic syndromes

•            Smoking

Risk factors for lymphoma, on the other hand, include the following:

•            exposure to radiation

•            Epstein-Barr or human T-cell lymphotropic virus

•            HIV, organ transplant, or genetic immune disorders

Some risk factors for myeloma include exposure to radiation, obesity, and other plasma cell diseases.

What are some common signs and symptoms of blood cancers?

Since blood cancer is a chronic condition that progresses slowly, symptoms may not appear until the end stage. While coughing, chest pain, frequent infections, fever and unexplained weight loss can occur in all the types of blood cancer, other symptoms may be specific to the condition.

Some symptoms of leukaemia include fever and lethargy, paleness and shortness of breath due to anaemia and increased bruising and bleeding. When it comes to lymphoma, symptoms may include swollen lymph nodes, fever, night sweats and fatigue and unexplained weight loss. Finally, in the case of myeloma, specific signs may include bone pain, particularly in the back and ribs, weakness, fatigue, and paleness due to anaemia and frequent bacterial infections, such as pneumonia.

Can blood cancer be prevented?

While there is no way to prevent blood cancer completely, certain lifestyle factors can reduce the risk of developing cancer. These include maintaining a healthy diet, exercising regularly, and not indulging in habits like smoking and excessive alcohol consumption.

Preventive health check-ups are highly recommended, especially if a person has a personal or family history of blood cancer, previous sessions of radiation therapy/chemotherapy or radiation exposure. If a person has experienced any symptoms related to blood cancer, it is best to consult an expert, as early diagnosis can ensure the best possible outcome.

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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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Novartis makes sculptures to shape knowledge of cancer symptoms

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By Nick Paul Taylor

September 18, 2023

Novartis has enlisted artists to help raise awareness of rare blood cancers, commissioning sculptures that portray the 10 key symptoms of the conditions to create a temporary exhibition that will tour the U.K.

The art exhibition is focused on symptoms of myeloproliferative neoplasms (MPN), a group of rare blood cancers that affect around 4,100 people in the U.K. each year. Novartis sells Jakavi for the treatment of MPNs in the U.K., recently securing coverage for one of the three main types of the blood cancer, and wants to spread knowledge that enables more cases to be identified early.

MPNs cause symptoms, such as fatigue, loss of concentration and night sweats, that overlap with other conditions, leading to delayed diagnoses. Novartis wants to bump MPNs up the list of potential causes that patients and physicians consider when they encounter the symptoms.

“More needs to be done to recognize and identify these symptoms early so that people living with the condition can seek help from medical professionals to better manage their symptoms, and those who are undiagnosed can seek a potential diagnosis sooner,” Alisia O’Sullivan, a MPN patient and MPN Voice volunteer, said in a statement.

To support that goal, the Swiss drugmaker has identified 10 key symptoms and commissioned sculptures to represent each of them. Each sculpture is in the shape of the number assigned to the symptom and features art that portrays the symptom. For example, symptom two, inactivity, displays a painting of someone sleeping under a blue sky and symptom three, weight loss, features a quote about being thin.

Novartis worked with MPN Voice and five mural artists from around the U.K. to create the sculptures and began showing them at Westfield, a shopping center in London, last week. The art exhibition will run for one week at Westfield and then go on a tour of U.K. cities.

Encouraging people to consider MPNs as an explanation for the 10 symptoms could support growth products for the condition. Novartis’ sales of Jakavi increased by 11%, rising to $435 million, in the second quarter on the back of demand in emerging markets, Japan and Europe.

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FDA Grants Approval to GSKs Ojjaara A GameChanger in Myelofibrosis Treatment

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by Yasmim Mendonça
 September 18, 2023

As of September 18, 2023, the United States Food and Drug Administration (FDA) has granted approval to GSK’s groundbreaking medication, Ojjaara (momelotinib), for the treatment of intermediate or high-risk myelofibrosis in adults suffering from anemia. This approval encompasses both primary myelofibrosis and secondary myelofibrosis, including post-polycythemia vera and post-essential thrombocythemia cases. Ojjaara is the sole authorized treatment that effectively addresses the primary symptoms of myelofibrosis, such as anemia, constitutional symptoms, and splenomegaly, making it a game-changer in the field of medicine.

Myelofibrosis, a form of blood cancer, affects a significant number of individuals in the United States, with an estimated 25,000 patients grappling with this condition. The approval of Ojjaara marks a monumental milestone for GSK, as it introduces a formidable rival to the widely-used Incyte drug, Jakafi. With this groundbreaking development, patients diagnosed with myelofibrosis now have an additional treatment option that holds immense promise in effectively managing their condition and improving their quality of life.

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