MPN Specialists Videos
MPN Advocacy & Education International partnered with MPN specialists to offer insights on patient concerns and updates on drug treatments during this pandemic. The videos are now available on our YouTube channel, click here to subscribe.
These videos are made possible by a grant from Bristol Myers Squibb
Naveen Pemmaraju, MD-MD Anderson Cancer Center
Mark Heaney, MD, PhD-Columbia University Medical Center
Ellen Ritchie, MD-Weill Cornell Medicine
Linda Smith-Resar, MD-Johns Hopkins
Dr. Resar’s presentation will be posted as soon as it is available.
A Mother’s Story: When Your Child is Diagnosed with an MPN
No one can prepare you for a cancer diagnosis of a child. Our daughter was four years old and began to complain about headaches. I assumed it was her eyes and made an appointment with an optometrist. Her sight was perfectly normal. A visit to her pediatrician lasted an hour with little insight, only suggestions to watch her diet, limit TV time and give her lots of water. She rarely watched TV and carried water with her throughout the day. Our diet doesn’t include sugar except from fresh fruit and we don’t eat boxed or canned food. I wasn’t optimistic. Eventually, her headaches became more severe on occasion, similar to migraines. We were sent to a neurologist. He requested blood work after she underwent an MRI. Thankfully, the blood work identified the problem-ET or essential thrombocythemia. We had never heard of it and had no idea what this meant for the future. A hematologist became our savior. With the proper diagnosis and medication, our daughter began to feel better, albeit a few side effects from the meds. Yogurt is a staple to help with GI issues, and a nap and early bedtime help fatigue. Yes, our little girl had what I would call fatigue. Our lives have changed but we do not let her ET control us. We control her ET. We manage her diagnosis as part of our daily lives as we would manage any other chronic disease. That is not to say it’s been easy. We have our moments of fear and doubt, but that doesn’t last as long as it used to. We keep very good records of her doctor visits, her blood levels and her overall health. We ask how she is feeling and pay attention to any changes that could be due to her ET. She is now 12 and enjoying a normal childhood. We are looking at Interferfon as a possible “next protocol,” if we think it will be better for her. We stay informed and are very pleased to see all of the clinical trials and new drugs on the horizon. It’s easy to say don’t panic if your child is diagnosed with an MPN. I would simply say, gather the facts, stay informed, be the voice they cannot be, and remember to take good care of yourself.
Click here to learn more about Pediatric and Young Adult MPNs
One Patient’s Point of View on “Living” with Myelofibrosis
David told his story at the Cleveland MPN Patient Program in November
On a beautiful fall day in late August 2013, I received a call that changed the course of my life. The voice on the other end told me that they had reviewed my blood counts and determined that I had some sort of leukemia. They had pre-admitted me to the local hospital to meet an oncologist and have the necessary tests. After about two weeks I received my diagnosis of Primary Myelofibrosis, Intermediate 1. I was told that treatment options were limited and the only true cure was allogeneic bone marrow transplan
It is tempting to focus all our energies on our hope in medical interventions. But diagnosis brings fear, denial, anger, & depression. These impact your relationships and can throw you into a downward spiral. Being diagnosed with a life-threatening disease like an MPN is an existential challenge. It raises all the questions: Why are we here? What is life about? What lies beyond this life? How you answer these questions will affect how you deal with your disease and its physical effects.
We are all tempted to be sad and maybe even angry. But you do not have to give in to the negative. You can choose to respond with a positive attitude.
My diagnosis has changed the course of my life – but for the better. Because of myelofibrosis, I realized that I was spending far too much of my time and energy focused on some sort of future achievement. I was super-busy every day and the days passed in a blur. But myelofibrosis woke me up to the truth that life is not about some future achievement. Life is about today. Since my diagnosis, I have come to have a heightened enjoyment of the simple pleasures of daily living.
These last six years have been wonderful and I have enjoyed them more because of my disease. My son and his wife have been kind enough to give us two new grandchildren in the past four years – and I am making the most of that. I’ve made many good friends in the MPN community. I took up motorcycle riding. The more aware I am of my mortality – the more I savor every experience of life.
Your life only comes one day at a time. Today is the day you have – make it into something good. Refuse to let an uncertain future rob you of today’s joys.
David shared his story in the MPN Community Connection Newsletter click here to view
David is the support group coordinator for the northern Pennsylvania/Ohio area, if you are interested in participating you can contact us for more information. Click here to contact us.
Fedratinib and Ruxolitinib: Advice for Deciding Which Agent to Give and When
The introduction of fedratinib (Inrebic) to the treatment landscape of myelofibrosis (MF) and the challenges that have arisen over deciding between administering fedratinib or ruxolitinib (Jakafi) means more community oncologists should consult specialists when treating these patients, said Andrew Kuykendall, MD.
Research shows that fedratinib and the earlier JAK inhibitor, ruxolitinib have similar efficacy in patients with MF. However, their toxicity profiles differ, and the potential for encephalopathy with fedratinib is an ongoing concern, resulting in a black box warning on the label. Now that the agent is FDA approved for the treatment of MF, oncologists are left with a decision of which JAK inhibitor to give to which patients and when to prescribe them.
How to continue using ruxolitinib now that fedratinib is available remains an unanswered question, said Kuykendall, assistant member, Moffitt Cancer Center; however, experts in treating myeloproliferative neoplasms (MPNs) can be a helpful resource for other oncologists.
Another resource for treatment decision-making is clinical data from the JAKARTA-2 trial, which studied fedratinib in patients with MF who were previously treated with ruxolitinib. Findings from a re-analysis of the study were presented at the 2019 ASCO Annual Meeting and showed that 46 of the 83 assessable patients achieved a spleen response (55%; 95% CI, 44%-66%), meeting the primary endpoint of the study.
The most common adverse events included diarrhea (n = 60), nausea (n = 54), vomiting (n = 40), constipation (n = 20), and others. Additionally, hematologic abnormalities including, grade 3/4 anemia (n = 96), thrombocytopenia (n = 68), and neutropenia (n = 23) were seen. Eighteen patients (19%) discontinued treatment due to adverse events.
These data suggest that fedratinib may be a second-line option for patients who are resistant or sensitive to ruxolitinib. The management of the gastrointestinal (GI)-related toxicities and checking of thymine levels to prevent encephalopathy, however, are newer management concerns that physicians must be aware of when administering fedratinib to patients with MF and is another point when consulting an MPN specialist may come in handy.
A Patient’s Story: What Box Do I Fit In And Does It Even Matter?
Linda, Grandmother and MPN Patient pictured above with her granddaughter.
I’m 58 and the proud mother of five beautiful children, their families and seven grandchildren. In 2017, I was diagnosed with essential thrombocythemia (ET). Looking back, I believe my symptoms began in 2011. I worked in a college library where I did research and helped organize lectures on various subjects for students and faculty. I also taught voice at night at a music school and sang at charity events and with various bands. Life was good. One day I got up from my desk and went to help students in the computer lab when all of a sudden everyone was a complete blur. Later, I experienced similar problems when teaching breathing techniques at the music school. I was in perimenopause so I wrote it off.
On Mother’s Day in 2011, I was out enjoying a band at a restaurant and while they were setting up their speakers, one blew right by my ear. I felt like I was underwater for an hour. Later that week I got a cold and had a loud heartbeat sound (pulsatile tinnitus) in my left ear. I then began a journey of symptoms that have not changed to this day. Early on I was diagnosed with various possibilities, Meniere’s Disease, MS, Vestibular Neuritis, Vestibular Migraine, maybe Lyme- so many ideas were entertained. I tried working for years in a reclined chair at my job. If I got up quickly without thinking, I would often see black spots. I would get odd brain fog at times and blamed it on the various drugs I was taking. After getting bounced around from neurologists to ENTs to cardiologists, I was finally diagnosed with atypical Vestibular Migraine.
In 2017, my platelets started climbing and my local neurologist, who had spent hours with me testing my blood pressure in different positions, felt I had a form of dysautonomia called POTs and needed more testing. He repeated an electromyography (EMG) study which showed severe neuropathy in 2012 and it came back the same in 2018. Eventually, I got to the point that the feeling of fainting was so strong I couldn’t stand. I tried to hide it whenever I could because it was so inexplicable even to myself. I was anxious because I never knew when a symptom would occur when I had to be up for any length of time and I looked normal on the outside and was embarrassed. My family and friends were frustrated with me because I went from being an active mom and grandmother to being disabled and limited in what I could do. My local neurologist sent me to a hematologist who diagnosed me with ET, CALR 1 mutation. He told me I would need a biopsy to confirm which I did at Sloan Kettering.,
As scary as it is to get diagnosed with a rare blood cancer, I felt slightly relieved that it might explain some of my symptoms and was told there was hope on the horizon with these blood cancers. It seemed that my neurological symptoms could not all be explained by the MPN only. and I probably have something else going on. I noticed that is a common complexity of MPN patients, we usually have other things going on and have been to many types of specialists. Being treated as a whole person can be challenging for us. I noticed that a lot of the symptoms were shared by the other groups I belonged to especially the Vestibular Migraine Group and Pots. It occurred to me that if these different chronic illnesses could be studied together maybe drugs used to help one could be used to help another especially if you are in a “watch and wait” situation. I’m sure this is being done all over the world.
I realized after joining some of the social media groups, that I am not alone in this feeling especially when it comes to the atypical migraines, brain fog and dizziness. Being in a box, is not so important anymore. Especially in the MPN world where you can have one type one day and potentially can learn it progressed or changed to another. We are in this together. No matter what. I’ve been lucky to have been referred to the Cleveland Clinic where I’m being evaluated by neurology and oncology to come up with an answer. I’m inspired by that institution and the kindness of everyone from the shuttle drivers, to the technicians and doctors who work there.
If I had any advice to share it would be to be your own advocate. Not believing everything you read in the groups is also important because it may never be part of your story and there is so much being researched and studied. If anything happens to be written that is inaccurate, you can put yourself in a state of fear even if you try to tell yourself otherwise. Also, there are wonderful friendships to be made with people who know what you are going through. I’m looking forward to finally meeting a fellow MPN patient, who I have been communicating with for a year at MPN Advocacy & Education International’s program in Cleveland this November. I’m realizing the importance of yoga and nutrition and I still try to keep busy for as long as I can stand before I give myself permission to rest when I can’t. I’ve since learned that life is unpredictable and can change in a moment. All in all, I try to be optimistic and feel most people are kind, loving, and caring, but no one knows what you feel better than yourself. I’ve also learned I have the best family, friends, and people in my life who provide love and support.
Treatments for MPNs During Pregnancy With Birth Rates and Maternal Outcomes
Question
Are use of aspirin, heparin, interferon, or combinations associated with live birth rate and adverse maternal outcomes in pregnant women with myeloproliferative neoplasms?
Findings
In this systematic review and meta-analysis of 22 studies, reporting on 1210 pregnancies, the live birth rate was 71.3%; most studies reported on pregnancy with essential thrombocythemia. The use of aspirin and interferon—but not heparin—was associated with higher odds of live birth.
Meaning
Moderate-quality evidence suggests that treatment with aspirin or interferon is associated with higher odds of live birth in pregnant patients with myeloproliferative neoplasms.
Myeloproliferative neoplasms (MPNs) are increasingly being identified in women of childbearing potential. Pregnancy in women with MPNs is associated with maternal thrombosis, hemorrhage, and placental dysfunction leading to fetal growth restriction or loss.
Objective
To evaluate the association between the use of aspirin, heparin, interferon, or combinations and live birth rate and adverse maternal outcomes in pregnant women with MPNs.
Data Sources
Systematic searches of MEDLINE, Embase, Cochrane Database of Systematic Reviews, Cochrane Central Register of Controlled Trials, and the MEDLINE Epub Ahead of Print and In-Process and Other Non-Indexed Citations from inception to July 19, 2018, with no language restrictions, was conducted. Key search terms included myeloproliferative disorders, polycythemia vera, essential thrombocythemia, and primary myelofibrosis.
Study Selection
A study was eligible if it included pregnant patients with MPNs; interventions included aspirin, heparin, and/or interferon; there was a comparison group in which patients did not receive the intervention; the study reported on at least 1 of the study outcomes; and it was a randomized, case-control, or cohort study or series of at least 10 pregnancies. Data were extracted in duplicate; 0.5% of identified studies met selection criteria.
Data Extraction and Synthesis
The review was conducted according to Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) guidelines and reported in accordance with Meta-analysis of Observational Studies in Epidemiology (MOOSE) guidelines. Data were pooled using the Mantel-Haenszel approach.
Main Outcomes and Measures
Outcomes were the number of live births and maternal complications, specifically, arterial or venous thrombosis, hemorrhage, and preeclampsia.
Results
Twenty-two studies reporting on 1210 pregnancies were included. The live birth rate was 71.3% (95% CI, 65.1%-77.6%). Use of aspirin (11 studies, 227 patients; unadjusted odds ratio, 8.6; 95% CI, 4.0-18.1) and interferon (6 studies, 90 patients; unadjusted odds ratio, 9.7; 95% CI, 2.3-41.0) were associated with higher odds of live birth. Addition of heparin to aspirin was not associated with higher odds of live birth (6 studies, 96 patients; unadjusted odds ratio, 2.1; 95% CI, 0.5-9.0). The most common adverse maternal event was preeclampsia, with an incidence of 3.1% (95% CI, 1.7%-4.5%).
Conclusions and Relevance
Most studies reported on pregnancy with essential thrombocythemia. Few studies reported on pregnancy with polycythemia vera and none with myelofibrosis met the inclusion criteria. Most studies were retrospective and early pregnancy losses may have been underreported. Moderate-quality evidence suggests that aspirin or interferon is associated with higher odds of live birth in pregnant women with MPN.
View Dr. Hobbs Presentation on Fertility, Child Bearing and Beyond
at the 5th Annual Women & MPN Conference
Health Insurance: What You Should Know
By Pam Trexel, American Cancer Society, Senior Vice President, Alliance Development and Philanthropy
Pam Trexel
Access to health care is a significant determinant in whether an individual diagnosed with cancer will survive. Uninsured individuals are more likely to be diagnosed with cancer at a later stage and more likely to die from the disease. The American Cancer Society Cancer Action Network (ACS CAN), the public policy arm of the American Cancer Society, believes all Americans should have access to affordable, quality health insurance.
Fortunately, in recent years there has been an increase in the number of Americans who have health insurance coverage. Since 2014, Americans have had access to comprehensive coverage that includes key consumer protections vitally important to cancer patients. These protections include: prohibiting insurance companies from denying coverage or charging more due to a consumer’s pre-existing conditions, restrictions against insurers imposing arbitrary caps on coverage, and a requirement that all insurance offered to individuals cover a broad set of benefits called essential health benefits.
Yet there are still challenges. Many cancer patients have difficulty finding specialists who participate in their insurance plan’s network, affording their prescription medications, and understanding their out-of-pocket expense liability. Recent regulatory and legislative approaches on both the federal and state levels have the potential to weaken current patient protections, segment the insurance market, allow for more insurance plans with inadequate coverage, and reduce access to healthcare for cancer patients and survivors.
Federal Activities
In 2018 the administration finalized a rule that would expand access to short-term, limited-duration (STLD) policies. The rule allows STLD products to be sold for a coverage period of up to 12 months and be renewed for three years. ACS CAN urged the administration to withdraw the rule due to concern that these policies are exempt from many of the key patient protections that ensure individuals with cancer and survivors have access to quality health care needed to treat their disease.
Additionally, the current administration has repeatedly reduced enrollment education and outreach funding,which limits efforts to inform consumers about open enrollment and plan options. Concerns remain about enrollment trends in future years and the abilities of non-governmental groups to continue outreach and enrollment efforts.
State Activities
Faced with uncertainty from the federal government, some states have implemented policies that seek to either strengthen or weaken the individual health insurance market.
Short-Term Limited-Duration Policies
As federal regulations try to expand access to STLD policies, some states are trying to prohibit or minimize their expansion. For example, New York state law permits the sale of short-term limited duration policies, but requires these plans abide by the consumer protections required for ACA-compliant plans.Other states are considering legislation that would limit STLD policies to a coverage period of less than three months without the option for renewal.
State Individual Mandates
The federal individual health insurance mandate penalty ended January 1, 2019. In response a few states have begun considering state-level individual mandates requiring state residents to maintain health insurance. Massachusetts has had a state individual insurance mandate since before the implementation of the ACA and never rescinded it. New Jersey has also enacted legislation to impose an individual mandate requirement.
Non-Comprehensive Coverage
Following administrative actions encouraging creation of association health plans (AHPs) – plans wherein small businesses join together to purchase health coverage – some states are considering legislation that exempts AHPs from state regulation. These plans are already exempt from the important patient protections provided under the Affordable Care Act (ACA). ACS CAN is concerned these plans will be able to discriminate against people based on their health status and will syphon off younger, healthier people, leaving older and sicker people in the state’s individual market (which would increase premiums).
Utilization Management
Cancer patients often need to choose a health plan based, in part, on the plan’s prescription drug coverage. Utilization management programs are health insurer practices used to control spending. These practices may include: prior authorization or approval of a drug by the patient’s health insurer before a prescription can be filled; and step therapy which requires patients to try, and fail, on an insurer-chosen prescription drug before gaining access to the drug that was prescribed by their doctor but may be more expensive. ACS CAN is concerned that if used inappropriately, utilization management may delay care or impede access to prescription drugs for cancer patients. Several states are considering legislation to ensure that utilization management practices are timely, efficient, clearly described for both patients and doctors, and allow for appeals and exceptions when appropriate.
Study Unveils Family Risk for Certain Types of Blood Cancers
Individuals with a parent, sibling or child with blood cancer appear to have a higher likelihood of also being diagnosed with the disease, according to study results published in Blood.
Moreover, age of diagnosis; whether the relative is a parent, sibling or child; and the number of affected first-degree relatives were substantially associated with familial risk for certain blood cancers.
“Although many hematological malignancies are individually rare, collectively they contribute significantly to the overall cancer burden in the population,” the researchers wrote, adding that the etiological basis of most blood cancers is poorly understood.
Therefore, they analyzed data from over 16 million individuals from the Swedish Family-Cancer Database to determine the familial risk of the different blood cancer and their possible inter-relationship.
Those with a familial link to the disease represented 4.1% of all blood cancer diagnoses – higher than patients with cancers of the nervous system, kidney and pancreas. However, this was lower than those with cancers of the breast, colon and prostate, which ranged from 8% to 15%.
In total, the researchers identified 153,115 patients who were diagnosed with a primary blood cancer, including myeloproliferative neoplasms (MPN; polycythemia vera, essential thrombocythemia, myelofibrosis and MPN not otherwise specified), chronic myeloid leukemia, myelodysplastic syndrome, acute myeloid leukemia, acute lymphocytic leukemia, Hodgkin lymphoma, non-Hodgkin lymphoma, diffuse large B cell lymphoma, follicular lymphoma, mantle cell lymphoma, marginal zone lymphoma, Burkitt lymphoma, small lymphocytic lymphoma, hairy cell leukemia, chronic lymphocytic leukemia and multiple myeloma.
The researchers found the highest relative risks for familial risk among patients with certain Hodgkin lymphoma subtypes (lymphoplasmacytic lymphoma and mantle cell lymphoma) and with polycythemia vera, myelodysplasia and essential thrombocythemia.
“This information improves our understanding of the causes of – and potential inherited predisposition to – blood cancers and should inform the identification and characterization of genetic risk factors for blood cancer, as well as how we best clinically manage patients and their relatives,” lead study author Dr. Amit Sud, from The Institute of Cancer Research in London, said in a press release. “The results should also encourage conversations among families, clinicians and patients about familial risk.” (Cure Magazine)