Watch Behind the Mystery: Living with Polycythemia Vera


The Lifetime Channel’s The Balancing Act featured a story this week on polycythemia vera (PV), with experts Dr. Richard T. Silver, a professor of medicine at NewYork–Presbyterian/Weill Cornell Medical Center, and Dr. Srdan Verstovsek of the MD Anderson Cancer Center, who discuss the latest inpatient care and clinical trials for PV, as well as the future for those living with PV as a chronic illness. Learn More

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Challenges Remain in MPN Diagnosis and Drug Development

Drug development in myeloproliferative neoplasms (MPNs), specifically polycythemia vera (PV) and myelofibrosis, have both remained similar to their diagnosis: difficult, explained Andrew T. Kuykendall, MD.

Regarding therapeutic advances, the FDA approved ruxolitinib (Jakafi) for the treatment of patients with myelofibrosis and polycythemia vera in 2011 and 2014, respectively. However, researchers are diligently working to bring more novel agents to the field.

The JAK2/FLT3 inhibitor pacritinib, though put on a clinical hold in 2016, is back on trial. The agent is being tested in patients with primary or secondary myelofibrosis who did not benefit from ruxolitinib or were not eligible to receive the agent in the ongoing PAC203 study (NCT03165734). Though momelotinib has been explored, it has demonstrated mixed findings in clinical trials, Kuykendall explained.

“Trying to get additional agents on board especially for different phenotypes of [myelofibrosis] is where I see the field going,” said Kuykendall, an assistant member of Moffitt Cancer Center. “In PV, we need to make some strides in understanding how to better treat the disease overall. We also have to understand what it means when we decrease the JAK2-mutant allele burden.” Read more

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Common Therapy for Rare Blood Cancer May Activate ‘Dormant’ Lymphoma, Study Suggests

Treatment of myelofibrosis, a rare type of blood cancer, with Jakafi (ruxolitinib) gives a patient 16 times the risk of developing aggressive B-cell lymphoma, through the activation of a pre-existing “dormant” lymphoma, according to a recent study.

The results suggest that the detection of this “dormant” lymphoma may be useful to identify myelofibrosis patients at risk.

The study, “Aggressive B-cell lymphomas in patients with myelofibrosis receiving JAK1/2 inhibitor therapy,” was published in the journal Blood.

Myelofibrosis belongs to the group of myeloproliferative neoplasms, in which the bone marrow stem cells that produce the body’s blood cells develop and function abnormally, resulting in increased numbers of specific types of blood cells.

The six types of myeloproliferative neoplasms are defined by the type of blood cell abnormally produced, in which myelofibrosis results in an increased number of immature white and red blood cells.

Jakafi, a suppressor of JAK1 and JAK2 — two proteins involved in the production of blood cells —became the first FDA-approved therapy for myelofibrosis and is widely used to treat this and other types of myeloproliferative neoplasms.

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First MPN Pediatric & Young Adult Symposium to be held in Chicago

There was a time when we didn’t think young children could have an MPN. We now know MPNs do not discriminate. Infants, children and young adults are being diagnosed regularly. Pediatric MPN specialist Dr. Nicole Kucine, MD, MS, Weill Cornell Medicine, partnered with us to bring attention to this group of patients and to their very specific challenges.
 “The study of MPN in children is far behind the field in adults. We are working to better understand the symptoms children with MPNs experience, and we are trying to identify causes of these diseases and their symptoms. With this information we will be able to create treatment plans specifically designed for children with MPNs.” (view interview with Dr. Kucine.  Dr. Kucine explained. She recently published a study identifying the challenges in distinguishing ET from PV in children with the JAK2 mutation. (Read Dr. Kucine’s study)

Parents want to know about dosing-what can their child can take and how much is safe. Can their child be in a clinical trial? Is their child able to have a transplant? Young adults (18-40 years), have issues and questions regarding pregnancy, life expectancy, if their offspring will have an MPN.

MPN Advocacy & Education will bring three specialists to Chicago for a half day, morning program, on September 29, 2018. Dr. Kucine will be joined by Drs. Linda Resar, Johns Hopkins Medicine, and Raajit Rampal, Memorial Sloan Kettering, we hope you can join us for this groundbreaking event. If the cost of registration is a financial hardship we will waive the registration fee.

Speakers for the Pediatric & Young Adult MPN Program from left, Drs. Nicole Kucine, MD, MS, Raajit Rampal, MD, PhD, and Linda Resar, MD

MPN Advocacy in Australia

Getting Pegasys onto the Government’s Approved Drug Listing

By Nathalie Cook

MPN Patient and Advocate, Nathalie Cook, works as an Accredited Practicing Dietitian in Community Health in Melbourne

In Australia over the last 20 years, myeloproliferative neoplasm (MPN) consumer advocacy has been strong. This began with the tireless support that the late Ian Sweet offered fellow MPN sufferers via the US based MPN-NET email list from the late 1990s and continued when Ken Young established MPD-oz, the first Australian email list, which he still manages today. Together these men have responded to thousands of emails from people with MPN both in Australia and around the world with messages of knowledge, wisdom and reassurance. In recent years other Australian online MPN groups have been setup to provide peer support to the MPN community.

In 2004, Ian and Ken began advocating for Pegasys® (the slow release, better-tolerated formulation of interferon (IFN)), seeking the Government’s subsidised Pharmaceutical Benefits Scheme (PBS) for MPN, as an affordable treatment option. They approached Roche who suggested ‘off-label’ access. At that time, no international clinical trials had been done with Pegasys for MPN treatment, so evidence of efficacy was lacking. This made government approval of Pegasys for MPN seem bureaucratically insurmountable.

In 2015, MPN patient Lara Chapman encouraged people with MPN in Australia to write to the Government’s Senate Enquiry into ‘The Availability of new, innovative and specialist cancer drugs in Australia’. This resulted in 40 MPN consumer submissions expressing a desire for PBS access to Pegasys for MPN, out of a total of 205 senate enquiry submissions.

In 2015 Lara, together with five other MPN patient volunteers from around the country, (Jolanda Visser, Sue Taylor, Angela Willet, Ken Young and Nathalie Cook) formed the MPN Alliance Australia (MPN AA), with goals of increasing community awareness of MPN, educating and supporting the MPN community, raising funds for MPN research and advocating for Australians with MPN.

The MPN AA co-hosted Australia’s first MPN doctor/patient conference in 2017, in partnership with the US-based MPN Education and Advocacy International. This conference brought together 150 MPN patients from around Australia and New Zealand and 10 haematologists three US-based and seven Australian.

Thanks to generous donations from the community, in 2018 MPN AA donated $20,000.00 to support Brisbane based haematologists. Assoc. Professor Steven Lane’s contribution to the Chicago-based MPN Research Foundation’s Interferon Initiative. This international multi-center collaboration of experts seeks to deepen understanding of how IFN works to treat MPN.

In 2011, in addition to all the other Australian MPN advocacy, MPN patient Nathalie Cook began a seven year campaign to Roche Products and the government for PBS access to Pegasys for people with MPN. This led to Pegasys becoming available on the PBS for MPN from 1 August 2018, in the Australia’s first consumer-led PBS drug listing! This was also the first government approved Pegasys listing for MPN to occur worldwide.

After many years of phone calls, emails and letters to Roche and the government, the breakthrough started in 2016 when Nathalie was invited to the Rare Cancers Australia CanForum in Canberra, as a consumer representative with the Leukaemia Foundation. At the CanForum she spoke with Prof. Andrew Wilson, Chair of the Pharmaceutical Benefits Advisory Committee (PBAC), (whose role is to advise the government on new PBS drug listings) about the unmet need for Pegasys to treat MPN.

This discussion led to Nathalie writing a submission directly to Prof. Wilson, seeking the addition of Pegasys on the PBS for MPN. In her submission she included information on her own experience with both types of IFN, (Roferon and Pegasys) and her better tolerance of Pegasys compared with Roferon.  She included extensive references to peer reviewed medical literature providing evidence of safety and efficacy of Pegasys in MPN.

The basis of her submission was that the older form of IFN (Roferon) was on the PBS for MPN, but was poorly tolerated, due to adverse side effects. She highlighted that Pegasys, the slow release better tolerated formulation of IFN should be on the PBS for MPN, because it is equally effective in controlling the disease, however the ‘off label’ price of about AUD$500.00 per weekly dose made it unaffordable for most patients.

When Nathalie’s submission was accepted by the PBAC for consideration in January 2017, she immediately contacted haematologists around Australia, the Leukaemia Foundation and Rare Cancers Australia, seeking support to get approval of Pegasys for MPN on the PBS. These groups subsequently sent the PBAC correspondence confirming a clinical need for Pegasys to treat MPN.

MPN-AA members Nathalie Cook and Ken Young attend the official announcement to put Pegasys on the government drug listing

The MPN AA used social media to encourage MPN patients around Australia to contribute comments via the PBS online consumer portal, stating why they wanted Pegasys on the PBS for MPN and what this change would mean to them in terms of quality of life.

Although the PBAC recognised a clinical need for Pegasys in MPN and knew it was safe long term with low toxicity, after a decade of experience with Hepatitis C treatment, the path to approval was not simple.

Firstly, Pegasys did not have Therapeutic Goods Administration (TGA) approval for MPN. TGA approval for a specific disease indication is usually required before a drug company can make a PBAC submission. Secondly, no Australian clinical trials had been conducted for Pegasys in MPN, another usual PBAC requirement for submissions. 

Additionally, Roche had no plans to write a submission for PBS listing of Pegasys for MPN, because the drug was considered ‘old’ and was close to coming off patent. In this exceptional case, the PBAC requested Roche lodge a submission for PBS listing of Pegasys for MPN, in response to Nathalie’s consumer submission.

During 2017, the PBAC worked with Roche, Rare Cancers Australia, haematologists and the Leukaemia Foundation on the issue of access to Pegasys for MPN. In the meantime, Nathalie continued discussions with Roche on the matter. Just before the December 2017 holiday season, she received a phone call from Roche saying the PBAC had approved inclusion of Pegasys on the PBS for MPN.

The next step was for the government and Roche to finalise price negotiations, before doctors could prescribe Pegasys for their MPN patients at the PBS price of AUD$39.50 (or AUD$6.40 for concessional patients). The MPN AA was delighted to share this wonderful news with the MPN community!

In July 2018, Nathalie was invited and with Ken Young attended, the Health Minister’s announcement that Pegasy and also three other cancer drugs that would be available to patients on the PBS from 1 August 2018. 

The achievement to list Pegasys on the PBS for MPN in Australia demonstrates the power of consumer advocacy and patient networking, and the importance of collaboration between consumers, the medical community, the pharmaceutical industry and government. 

View Videos from the Australian MPN Patient/Caregiver Program

PEGASYS ® is a registered trademark of Hoffmann-La Roche Inc.

Lymphadenopathy May Be an Early Manifestation of Primary MF

Extramedullary hematopoiesis (EMH)—the presence of hematopoietic activity in sites outside of the bone marrow, such as the liver or spleen—can occur in malignant and non-malignant hematologic diseases, and they may be a complication of primary myelofibrosis (PMF). EMH has been observed in the lymph nodes of some patients with PMF, and a recent case reportdescribes a presentation of lymphadenopathy, secondary to EMH, that occurred as a patient’s first manifestation of PMF.

The report describes a 63-year-old patient who experienced generalized lymphadenopathy for 9 months, and had fever, night sweating, and weight loss. She had no history of hematologic illness. Computed tomography revealed lymphadenopathy in the mediastinal, abdominal, and pelvic lymph nodes, as well as enlargement of the liver and spleen. Additionally, sclerotic lesions throughout the patient’s skeletal system were observed.

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A Patient’s Story: How I Diagnosed Myself

My journey with essential thrombocytosis (ET) began in May 2016.  Although I am sure I had it for at least four years prior to to that.  I self diagnosed myself after noticing my gums were bleeding when I brushed and flossed.  I am a dentist, so how could this be?  I have immaculate oral hygiene, floss and brush at least two times a day and get my teeth cleaned every three months.  A little voice from one of my lectures in dental school went off in my head;  I recalled my professor’s words, “in the absence of gum disease or dental issues, bleeding gums can indicate a blood cancer and you should refer your patient to their doctor immediately.”  I didn’t think I would be the one to need the referral.  After some research on Dr. Google I put the puzzle pieces together.  I had tingling fingers and toes for at least a few years and had actually gone to a neurologist who tested me for carpal tunnel syndrome.  Again, being a dentist, that is not unusual to get carpal tunnel. However, after that diagnosis was negative, I just brushed it off.  I had also had major hives a couple of years ago all over my legs,  I had gone to an immunologist and she said it was allergies and put me on allergy shots.  After they didn’t go away, I had gone to a primary who told me my symptoms were stress related, “psychogenic” as he called it and I needed to manage my stress.
Six months before my diagnosis, I had a case of vertigo.  Again, I went to my doctor who reassured me it was a viral infection and it would go away in a few days.  I never had vertigo again so I believed him.  No blood work or additional testing was done.
However, the bleeding gums and my professor’s voice was what made me suspicious.  I started researching blood cancers and put all my symptoms together for what pointed toward ET.  I took charge of my own health and went to a hematologist and asked him to test me for JAK2 mutation.  Sure enough that came back positive and my bone marrow biopsy confirmed it.  Platelets were in 900 range and I just didn’t feel like myself.  He insisted I start on Anagralide that day and that I was going to stroke any minute.
I wasn’t comfortable with his rush to treatment as I had read a lot on ET prior to my diagnosis, so I knew that Anagralide was definitely not first line of treatment and neither is Hydrea if you are at low risk like me.  I am otherwise very healthy, work out religiously, grow my own vegetables and juice fruits and vegetables at least 5 times a week. I am not a smoker and I don’t  drink. I have a normal BMI and have no other health conditions.  I ran out of that office and went to UCSD in search of a doctor that actually listens.
My new doctor suggested that I start on a low dose Hydrea immediately and I declined.  I had research and information on my side-I was under 60, platelets under one million and otherwise very healthy with no other cardiovascular issues or dispositions.  So I declined once more and she agreed to monitor me.  After 18 months, I had a lot of headaches and could hardly feel my feet and my hands felt horrible, almost numb.  Again, I am a dentist, so this scared me and I agreed to do treatment but wanted to try Pegasys instead of Hydrea.  My doctor was very reluctant about Pegasys because she believed the side effects are not worth the benefits.  I produced a lot of studies and literature on how it works better for some people and in my mind I would rather be on Immunotherapy rather than Chemotherapy.  I did take Hydrea for one month while awaiting insurance authorization for Pegasys and I knew immediately that I was right in my intuition. It wasn’t the right treatment for me. I was nauseous all the time, couldn’t sleep, had major brain fog, red dots on my chest and legs, my nail beds even hurt.
The answer to my symptoms by a different doctor at UCSD was to take more drugs.  One to help me sleep, and an anti-nausea medication.  I am very much against taking drugs if I don’t have to so this didn’t sit well with me.  Not to mention, they wanted to increase my dose every week since my numbers weren’t coming down the way my doctor wanted them to.  For the first time since my diagnosis, I broke into tears because I knew I wouldn’t have a quality of life if I increased my dose of 2000 mg a day.  I have a demanding job and need to have a clear brain!  When my insurance authorized the Pegasys, my doctor agreed to let me try it and I have been doing great on it at 90 MCG per week and in one month my numbers are down to 920.
I am not suggesting Interferon is for everyone because you all know we are all different and respond differently to different medications.  In some studies that compare Pegasys to Hydrea the dosing of Pegasys was so high and toxic that patients dropped out, I would too.  But at a low steady dose, it is working for me. Someday, I may have to go back to Hydrea. No one knows how our bodies respond to certain drugs long-term but for me personally, I wanted Pegasys as my first line of treatment.  I am 50 years old and 20-30 years of Hydrea ahead of me was not going to be my first choice.
I guess the lesson I have learned and continue to learn is to be your own advocate, research and study your disease.  Doctors are busy people with perhaps thousands of patients.  I only have one disease and one patient; MYSELF.  I will continue to fight for what is right for my body and luckily I have a great doctor that listens to my wants and needs. If I didn’t, I wouldn’t hesitate to switch till I found the right doctor.

Gender Effects on MPN Patients: Results from the MYSEC Project


Survival difference between men and women in the general population is well recognized and consistent sex-discrepancies are common within hematologic malignancies. Additionally, there is developing interest in understanding how gender influences the pathogenesis, phenotype and outcome in myeloproliferative neoplasms (MPN).

The aim of the current study is to evaluate the prognostic impact of gender in the study-population of the multicentric MYSEC (Myelofibrosis Secondary to PV and ET Collaboration) project including 781 patients with post-polycythemia vera (PPV-MF) and post-essential thrombocythemia (PET-MF) myelofibrosis.

The study was approved by the Ethical Committee of each Institution and conducted in accordance with the Declaration of Helsinki. Diagnoses of secondary myelofibrosis (SMF) were performed between 1981 and 2015 and were locally reviewed according to the International Working Group on Myeloproliferative Neoplasm Research and Treatment criteria (IWG-MRT 2008). Wilcoxon rank sum and Pearson’s chi-squared tests were performed to report differences between groups whereas Kaplan-Meier estimators, log-rank tests and Cox regression models were used for time-to-event analysis.

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New Tool Helps Patients With MF Time Stem Cell Transplantation for Better Outcomes

An allogeneic stem cell transplant (ASCT) is the only treatment that has the potential to cure myelofibrosis (MF), one of the three main types of myeloproliferative neoplasms (MPN); however, a stem cell transplant comes with risks — some potentially life-threatening. Fortunately, there is a new tool that can help with these potential concerns.

Patients with MF can now evaluate risk through a new tool designed to improve transplant outcomes. The SCT Spectrum Transplant Timing Tool (SSTT) was created by Zhenya Senyak, a patient with MF and editor of MPN Forum, with input from an 18-person taskforce made up of MPN experts, patient advocates and SCT patient survivors. The project is sponsored by the MPN Research Foundation (MPNRF).

“What we really want this to do is be a place for patients to facilitate conversations with their doctor,” Michelle Woehrle, executive director of MPN Research Foundation (MPNRF), told CURE in an interview.

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Increased B-cell lymphoma risk with JAK1/2 inhibitors


Patients with myeloproliferative neoplasms treated with Janus-kinase (JAK) 1/2 inhibitors may be at significantly increased risk of aggressive B cell non-Hodgkin lymphomas, according to a study published in Blood.

A retrospective cohort study of 626 Viennese patients with myeloproliferative neoplasms – 69 of whom were treated with JAK1/2 inhibitors – found that 4 of the 69 patients (5.8%) developed aggressive B-cell lymphoma, compared with just 2 patients (0.36%) in the rest of the group. This represented a significant, 16-fold higher risk of aggressive B cell lymphoma associated with JAK1/2 inhibitor therapy (P = .0017).

The lymphoma was diagnosed within 13-35 months of starting JAK1/2 inhibitors. In three patients, the disease was in the bone marrow and peripheral blood, one patient had it in mammary tissue, and another had it in mucosal tissue. All four lymphomas showed positive MYC and p53 staining.