Special Interview: Living with an MPN in Childhood

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Diagnosed at age seven with Essential Thrombocythemia, Portia shared her story at the 2nd Annual Pediatric & Young Adult MPN Patient Program.

Portia, a young adult MPN patient, shared her story at the 2nd annual Pediatric & Young Adult Program

Do you remember experiencing any symptoms?

I had occasional nosebleeds that would last about twenty minutes or so. But over time, the time decreased to about ten minutes. I also experience fatigue, especially when I’m sick, all I do is sleep to try and regain any energy. Also, I’m very active and I play competitive squash, so I do experience fatigue more than an average person.

How do you cope with essential thrombocythemia (ET) symptoms and/or side effects from Hydroxyurea?

I’m very lucky that I don’t really experience too many symptoms, but I usually push through any pain that I have since I’m such a wimp about medication. I have not had any side effects from Hydroxyurea. For fatigue, I don’t take any other medication, I pretty much just work hard and try to be smart about how I utilize my energy. When playing squash, I work extra hard to make up for my fatigue, but if I really can’t breathe due to lack of oxygen, I will talk to my coach and ask for a small break to recuperate. Most coaches are very reasonable and will allow a break.

Has ET curtailed your involvement in school activities?  Sports?

When I was younger, elementary and middle school age, I would occasionally have to miss school for lab appointments, so I would have to make up work. One symptom of ET is fatigue, so I do have to deal with that in sports. But I also have Hemoglobin H, which I believe has a bigger impact on my fatigue in sports than ET. But overall, I still go about my life and continue to do the things I love.

How do you explain what you have to your friends?

As I’ve gotten older, I’ve done a lot more of my own research to further understand my condition, but to my friends, I explain that I have way too many platelets, which help clot your blood when you get a cut, and because of that, I bleed for longer.

What advice would you give other younger individuals with an MPN when peers say they don’t look sick or they’re faking?

I would tell them to do their best to ignore their hateful comments and try to explain their condition by telling them it’s something internal rather than external, that’s why they don’t appear sick. And most important, find friends who won’t judge you, and people who do, clearly aren’t your real friends, for real friends should accept you no matter what.

You are very energetic and positive, when you reach out to others your age who aren’t feeling well, what do you say to encourage them?

First, I would listen to their concerns and possible issues, and then I would tell them to keep their head held high and know that it does get better. This is just a phase and eventually, the negative parts will fade away. Also, it’s very important to know who your close friends are and be able to talk to them since many kids would rather talk to their friends rather than a parent or even a doctor since it can be intimidating. I would also say, take one day at a time and find joy in the little things, whether it’s going for ice cream or just taking a nice walk.

If you could wave a wand and change one thing in the world of MPNs, what would you change?

Personally, I would change the medicine. I really hate swallowing pills, so I would much prefer something fun to eat or drink as my medicine. I’m also very strange and would much rather have the medicine get injected into me, which I know is an option, but the majority of people aren’t a big fan of needles.

 

 

 

A Caregiver’s Story: The Journey of a Spouse Through the Transplant Process

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In March, MPN Advocacy & Education International highlighted the story of a recent stem cell patient, Andrea (click here to view story). This month, Andrea’s wife, Denise, shares her story as a caregiver in an interview with us. 

Andrea and Denise

How did you feel about your spouse’s decision to have a transplant? Were you apprehensive? 

I have been a part of Andrea’s medical journey since her diagnosis from ET to myleofibrosis.  I recall it took me about a week to learn how to pronounce the name correctly, and it has been an education ever since.  Being present during doctor appointments and multiple clinical trials provided first-hand information in addition to our ongoing discussions.  Observing and experiencing her five years of transfusion independence was a gift.  We took advantage of the opportunity to cycle and travel together and separately. Knowing the clinical trial would either end or the drug would quit working was only a matter of time.  And knowing how she physically felt prior to and after this time period played a role in my ‘wrapping my head’ around a possible future transplant.  All the while, we adjusted and modified activities as needed in order to continue allowing her to live life to the fullest.

When medications were no longer yielding the same results, and Andrea’s blood transfusions became more frequent, the doctors felt the alternatives were to continue clinical trials and hope for the best or discuss a stem cell or bone marrow transplant. I felt she was strong enough physically to beat the odds. It was now or never. I never doubted our decision, and have had no regrets.

How did you prepare yourself as a caregiver? 

In order to prepare, I scoured MD Anderson’s educational resources to understand what a ‘typical’ transplant journey would entail.  I developed multiple spreadsheets to track medications, nutrition, recovery, therapy, etc. I left the online myleofibrosis forum readings to Andrea as she would report her findings from reading first-hand accounts.  She also talked to many individuals who had had transplants, pummeling them with questions.

Another important piece of this journey was my mindset.  I focused on the end goal – her successful transplant and healthy recovery.  I looked at the journey in three parts:

  1. Pre-transplant
  2. Transplant/hospital stay
  3. Post-transplant

My goal was to have as much of her daily care and needs become second nature to me prior to her hospital release. When she was released, the additional environmental interaction was familiar without the distraction of ‘everything new all at one time’.

What tools did you find useful as the caregiver and advocate?

I created a 3-ring notebook with tabs to manage spreadsheets, medical records/test,  and doctor questions, so I could access information easily and quickly. I created a spreadsheet to track her medication schedule.  The purpose was to help me identify what was needed, including dosage when refilling medications both inside and outside the hospital. Click here to view medication tracker template. (For an Excel spreadsheet version of the attached please email kmichael@mpnadvocacy.com).

I also knew there was a good possibility that after her hospital release and within the first 100 days, she would have a trip to the emergency room and be re-admitted to the hospital.  Therefore, medications and ‘the notebook’ were kept in one location and easily picked up and transported with us.  The notebook was with us each time we visited the care team.

I created additional spreadsheets to help track:

  • Food and water daily intake and output. This is useful for doctor & nutrition appointments.
  • Daily stats: blood pressure, temperature, pain levels, exercise, spirometer therapy.  This is useful for tracking blood pressure and temperature anomalies.  Because if her temperature rose to a specific number, she had to go to the emergency room immediately.
  • Signs of graft vs host disease (GVHD).
  • I choose to stay in the hospital 24/7 to understand how the nursing staff/care team handled her care. This experience helped me understand her routine.
  • I figured if I learned her hospital routine, it would be familiar when she was released.
  • I used my smartphone and set alarms with labels so I knew what drugs were due when. (Used upon hospital release.)
  • I utilized Caring Bridge (www.caringbridge.org) to communicate Andrea’s transplant journey to her friends and family. My intent with each post was to create an engaging story yet convey “a day in her life” so everyone could get a sense of being there.

What was the most challenging part of your role and why?

Coming home and changing environments automatically kicked us back to normal behaviors and patterns. I felt we had to be more careful and diligent in preventing infection.  Even though we were home, I had to be more watchful because familiarity brings about a relaxed state.  Her immune system was developing and the risk of infection was too great.  Andrea quickly tired of me saying “No, you can’t do that or touch that.”

Once home, Andrea’s friends visited.  This was a considerable risk to her because the natural tendency is to hug and touch. To reduce her risk, we asked people to use hand sanitizer when they were around her.  We developed a routine where I would greet her friends first with a hug and explain she couldn’t hug yet. Andrea stood back a few feet to reduce her availability.

What I learned :

Every recovery and journey is different.

While it’s tempting, don’t measure your progress against someone else.  It’s your journey.  That goes for both the patient and caregiver!

The “notebook” was a great tool.  It kept us on schedule for all medications and were able to provide information to the doctors as needed.

The hospital care team staff is a critical part of your recovery.  Don’t opt for staying close to home if you feel a facility’s care team is better in another location.

Deciding where to have the transplant included researching the number of myleofibrosis transplants, versus other blood disease transplants, and the success that facility had.

Participate in the journey. I chose to shave my head at the same time Andrea did.  It sure made showering quick and easy!

Take in the outdoors and/or change the scenery.

Exercise or go for walks.  Listen to music.  Visit a friend. Go to the grocery store.  Do something to clear your head and regain perspective.

Have a confidante. It’s normal to question, or become frustrated, and to second guess.  But remember – it’s temporary.

You are the coach, cheerleader, and guardian all at once.  Embrace the many hats you will wear!

Click here to read Andrea’s Transplant Story 

MPN Patient Daily Stats 06-10-18

A Parent’s Story: Navigating the Health Insurance Maze

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By Sarah W.

Sarah is a member of MPN Advocacy & Education International’s Patient Advisory Council

I know there are good people who work at insurance companies, however, some are the bane of my existence. Every month I gear up for the fight to get my son, Jedi, his medicine. Jedi has an MPN. My hope is that my journey can be used to make this process easier and better for other patients and their families.

Hydroxyurea was the initial drug prescribed for my son. It wasn’t a difficult drug to get filled, even though it was a little overwhelming walking my son through the adult oncology unit at MD Anderson Cancer Center, to the “specialty pharmacy.” It was frightening for him to see adults that look so sickly skinny and with their bald heads from their treatments. However, the real frustration began when he was put on Pegasys. MD Anderson tried to call numerous pharmacies to his prescription filled, but they were unable to find a pharmacy that had it in stock. So we left the hospital with a handwritten script in search of the drug.

Read tips from Speciality Pharmacist/MPN Patient Jennifer Powers w/ links to treatment assistance programs

At that time, we were insured by United Health Care. This was my first introduction to a specialty drug. For background purposes, specialty drugs are a recent designation of pharmaceuticals that are classified as high-cost, high complexity and/or high touch, such as injectables. A chemotherapy that is administered orally, like Hydroxyurea, is covered as part of a normal prescription. A chemotherapy that is administered as a subcutaneous (jiggly fat) shot, like Pegasys, is not. In other words, it comes out of your deductible. For us, that means we owed approximately $4,000 for the first month of Pegasys. We had to prove to the insurance company that Pegasys had been approved for polycythemia vera. This was done by producing a memo my husband found on the internet from United Health Care specifically stating Pegasys was approved for polycythemia vera.

Then, our insurance was switched to Blue Cross Blue Shield of Texas (BCBSTX). (I will skip the conversation about trying to get our member number early so I could start the pre-approval process.  I had been told in a Facebook group that it took about six weeks to approve Pegasys with BCBS. This was cause for concern because we could only get four weeks of medicine at a time.)

I started the pre-approval process with BCBS after we received a member ID number the first week of January. As expected, Pegasys was initially declined.  In the state of Texas I should be able to submit an emergency appeal and get a response in three days, but I had to get the appeal submitted first.  It is very difficult to get the appeal started. Not to mention that BCBSTX outsources the pharmaceuticals to Prime.

After a few weeks of deep frustration and the looming possibility we could run out of his medication, I finally posted the following on Blue Cross Blue Shield of Texas Facebook page:

January 15, 2019. Your appeals process is TERRIBLE. I have spent HOURS trying to figure this out. I have a 10 year old with CANCER. His medicine is out next week. I have to get it from a specialty pharmacy. His doctor called BCBS yesterday (a number I got after spending TWO HOURS on the phone last Friday) and was told that he has to wait 7-10 business days to start the process. Today I received a NEW peer-to-peer phone number. Does this start the process? No, it sets up an appointment for our doctor to talk to your doctors. Just to give you an idea. This medicine controls platelets. If his platelets go up he could have a heart attack, stroke, embolism for example. You can see my concern. My next steps are to ask my doctor to call AGAIN to START the appeals process – you know the one he tried to start yesterday? I will also have on my TO DO list to file a complaint with Texas Department of Insurance, contact my state Senator and Representative. Thank you for making this such a HARD process. Oh, by the way, the National Comprehensive Cancer Network, other BCBS insurance (different states), United Healthcare ALL have approved this medicine for his diagnosis

 I received a phone call from BCBSTX within 24 hours of posting this. I was told I had made it to the “special escalation” team because I posted on social media. On one hand, I am grateful he was approved. On the other hand, I am sad that this is what it took. I think it also helped that I had gathered the information to make it easy for them to approve. I had whitepapers, Jedi’s medical records and the details from the National Comprehensive Cancer Network. (Click here to view).

I wish I could say this was the end of the drama. But this is a story for another day. Just writing this was overwhelming. I can’t imagine what people who are sick go through. I am the caregiver, not the patient. I do not have chemo brain and I am exhausted by the process and the stress and anxiety to ensure my child receives his treatment. I hope this information will help other families move through the process a little more smoothly and I will continue to write about the trial and tribulations I experience on my website.

View Videos from the MPN Pediatric & Young Adult Program in 2018

 

 

Interview with a Patient: Making the Decision to have a Transplant

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Andrea was initially diagnosed with essential thrombocythemia after her primary care doctor noticed her platelets steadily rising. Ten years later it had progressed to myelofibrosis. She lives in Texas with her wife (and caregiver), three dogs and one cat. After working with American Airlines and Sabre Holdings for 30 years she chose an early retirement and pursued other opportunities, including working in a bike shop (she’s an avid cyclist), flower shop, consulting firm and for the last six years with Apple. 
What made you decide to have a Stem Cell Transplant (SCT)?
It was an exceptionally hard decision to make. I struggled with it up to and including the day before transplant. I had been feeling good, I was in and out of several clinical trials, the last of which had worked well for six years. But I realized my options for the future were very limited. I had become red blood cell transfusion dependent. My age was advancing, I had exhausted all relevant clinical trials and available drugs and I was physically pushing harder to do things I loved. It was clear to me that waiting “it” out and hoping for another miracle drug or a SCT were my only options.
How did you prepare for the procedure?
I like to be as prepared as possible, so I found speaking with actual survivors helped me get answers to all my questions that doctors may not have known or had time for. I spoke to several SCT recipients, especially those who had the same MPN as me. I read as much information as possible such as newsletters, blogs, and other online sources. It did not dwell on the less positive comments! Gathering as much information as possible from a variety of sources gave me an idea of what was to come. Keeping in mind that everyone is different, everyone I connected with gave me tidbits that I could refer to before, during and after transplant.
Andrea was initially diagnosed with essential thrombocythemia after her primary care doctor noticed her platelets steadily rising. Ten years later it had progressed to myelofibrosis. She lives in Texas with her husband (and caregiver), three dogs and one cat. After working with American Airlines and Sabre Holdings for 30 years she chose an early retirement and pursued other opportunities, including working in a bike shop (she’s an avid cyclist), flower shop, consulting firm and for the last six years with Apple. 

 

A Mother’s Story: Children DO Get MPNs-Our Loss May Save Your Child

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In memory of Jordan

In 2014, our daughter Jordan, age 14, complained of headaches and neck pain. Ultimately the doctors diagnosed her with mastoiditis, with a rare complication of cerebral venous sinus thrombosis (a 5% chance of that complication). The doctors then fixated on a diagnosis of migraines before, during and after shunt evaluations, placement and revisions.

In January 2015, unbeknownst to us, one doctor mentioned polycythemia vera as a possible diagnosis. He requested a hematology consult, but no action was taken. Due to the CVST, Jordan developed papilledema and required shunting due to intracranial hypertension. She was at risk of losing her vision.

Jordan suffered from headaches, nausea, vomiting, blurry vision, joint and bone aches and pains, and itching which was diagnosed as an allergic reaction to medication she had taken. Nobody looked at the bigger picture. One doctor said her issues were the result of complications due to migraines. After which,  every other doctor followed suit despite the symptoms, abnormal imaging, and abnormal lab results. All of this happened over a three year period.

In February 2018, Jordan, now 17, was diagnosed with portal vein thrombosis. Her symptoms remained unchanged for three years, yet the doctors still fixated on migraines. At this point, I started doing my own online research and requested testing for polycythemia vera (PV). We were told by a hematologist that tests including a bone marrow biopsy, were not necessary as her labs were normal. They were not.  She was reluctantly tested for the JAK2 mutation on February 21, 2018. Nobody read the results.

On March 4, 2018, we were told Jordan could possibly have an MPN and a bone marrow biopsy was needed. The biopsy was performed on March 5, 2018. Two days later, our Jordan passed away on March 7, 2018.

The medical community failed our daughter. We were told on March 4 they didn’t test her earlier because “this isn’t seen in children.” We told them this was no excuse not to test, diagnose and treat. Their expectations resulted in our daughter’s death. Instead of trying to find out why our daughter was sick, and doing further investigation into her MPN symptoms, they labeled her with a default diagnosis that did not fit.

Afterward, we were not told of her official diagnosis. No doctor called us. Patient Relations refused to speak to us and suggested we request our daughter’s records and have our primary care doctor review and advise. We found out six weeks after she passed away she suffered from primary myelofibrosis.

If Jordan were tested for JAK2 and or had a bone marrow biopsy in January 2015, when one doctor requested hematology do an exam because of suspicion of PV she would still be here. She could have had treatment. She could have had a stem cell transplant. Due to lack of awareness, ignorance, ego, expectation or a combination of these, she was left to suffer. We lost our beautiful daughter because they didn’t want to look beyond migraines. The sad part is that she was at a well-known level 3 tertiary care children’s hospital in southern CA.

We have now become advocates for awareness. We want doctors who see children not to be swayed by “expectation” as MPNs can affect anyone regardless of age, gender, or ethnicity. We want more research. The medical community needs to be educated. We advocate for funding. We advocate for Jordan. We are sharing her story on Facebook in the hopes of raising awareness, www.facebook.com/jordansstory .

MPN Advocacy & Education International’s Pediatric and Young Adult MPN initiative advocates for young patients. As part of that effort, we are in the process of creating a private, online, space for parents to communicate with each other and seek input from our Pediatric MPN advisors, Dr. Nicole Kucine, Weill Cornell and Dr. Linda Smith-Resar, Johns Hopkins Medicine. Click here to learn more about our Pediatric and Young Adult advocacy efforts 

 Click here to download MPN Advocacy & Education International’s Pediatric & Young Adult Booklet

Join Us for the 2ndAnnual Pediatric & Young Adult MPN Program on May 16 in New York City

 

 

Watch Behind the Mystery: Living with Polycythemia Vera

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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

Learn more about MPN Clinical Trials

A Patient’s Story: How I Diagnosed Myself

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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.
 

A Mother’s Story: Coping with a Sick Child

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Young MPN patient “Jedi” with his companion Chewy

In the opening credits of the television show “The Fresh Prince of Bel Air” Will Smith sings, “This is the story/all about how/my life was turned/upside down.” This article is the story all about how OUR lives were turned upside down when our son, who we affectionately refer to as a “Jedi” because of his special blood, was diagnosed with a Myeloproliferative Neoplasm (MPN).

Our story is probably different from other adults/children with an MPN because Jedi wasn’t extremely sick before we discovered he had an MPN. For about two years, Jedi had experienced a variety of unexplained health issues — random fevers, flu, an estimated seven times over the preceding twelve months, and extreme pain in his legs. At the time, I attributed these conditions to allergies, or growing pains, things that boys normally experience at that age. This soon changed, however, when I took him in for his annual physical exam. The doctor suggested a blood test for Jedi after hearing about his recent health issues. I am not one who immediately agrees to testing, but I agreed when the doctor said to me, “if it was my son, I would do the test.”  A week later, the doctor called and told me the blood test was contaminated and to immediately re-test Jedi, which we did. Two weeks later, I received a phone call from him, who told me he believed Jedi had Essential Thrombocythemia (ET), a condition I had never heard used before. His doctor then recommended we see a specialist who specialized in ET. It can be hard to diagnose a child with a MPN, because it is so rare. However, the doctor was a General Practitioner and had seen it in other adults. Thus, Jedi didn’t get extremely sick before diagnosis as so many of the children do.

The following Monday, I called the recommended specialist. The scheduler answered the phone by stating the name of the organization, which was “something something oncology.” Her words, more specifically one word – oncology – startled me. I held it together long enough to explain who I was and to ask for an appointment. Once the call was completed, I hung up the phone, closed my door and cried. Oncology? Why was an oncologist being recommended to see my beautiful eight-year old?

The results of Jedi’s first bone marrow biopsy revealed he had the JAK2 gene mutation. Jedi asked what a gene mutation was. I frantically tried to explain a gene mutation to my child. My first instinct was to tell him he was a mutant. I knew his next question was going to be, “What is my superpower?” I didn’t have an answer for that question, but realized he is like a Jedi, who has midi-cholorians, or special blood. That is what I explained to him.

Pediatric MPN Specialist Dr. Nicole Kucin, MD, MS, New York Presbyterian Hospital/Weill Cornell Medicine. 

After initial difficulty finding a specialist who understood MPNs, we now have a talented team of specialists. He sees a local doctor every month. He also sees Dr. Nicole Kucine, MD, MS, an MPN specialist with Weill Cornell in New York City, click here to learn more. Dr. Kucine is performing a study on children with MPNs through the National Institutes of Health (NIH). If you have a child who has an MPN, I highly recommend contacting her. Last, Dr. Srdan Verstovsek (aka “Dr. V”) who is affiliated with MD Anderson in Houston, Texas, is part of the team. He is an Adult MPN specialist but performs a lot of work related to the JAK2 mutation. He has agreed to consult with Jedi’s Pediatric Hematologist, Dr. Michael Rytting, who is also at MD Anderson. As a result of Dr. V and Dr. Rytting’s recommendation, we have changed Jedi’s treatment plan from Hydroxyurea (HU) to Interferon.

In determining the appropriate treatment plan, some questions we asked were:

What are our options for treatments? What is the difference between each treatment? Are there timeframe limitations for each treatment? (The effectiveness of one of the drugs used to treat ET is limited to 5 years. That was information I did not know but extremely important to know given Jedi’s age – now nine years old!)

Are there any other patients using this same treatment? Have they experienced any side effects not listed on the medicine? Is the basis for our understanding of how this treatment affects the patient based on a different disease? For example, HU is commonly prescribed to patients with sickle cell anemia. Some doctors’ understanding of how HU impacts a patient is based on the their patients who have sickle cell anemia, which is a completely unrelated disease.

Additional questions to consider:

Is there a way to mitigate side effects?

Can we start with a lower dose and see if it works?

How long will it take for the medicine to start working?

What are the risks of not taking any medicine? What are the risks of taking this medicine?

Is there any research being done on these treatments?

In finding a local doctor, some questions we asked are:

Are you willing to work with other specialists in this field of medicine?

How do you propose to communicate with them?

Are you willing to follow the specialist’s instruction when treating my child?

Are you willing to consider diet as part of the treatment?

There a few ways to connect with other with MPN patients. I have gotten great information from Facebook support groups. Attending MPN conferences is another way to become informed and connected. We attended an MPN conference in February. Listening first-hand to specialists providing updates in the field and answering questions was like drinking from a firehose. Thankfully, MPN Advocacy & Education International posted the videos on the website, which allowed me the opportunity to repeatedly watch them to fully absorb the information the specialists provided, view conference videos. Being able to converse with the attendees at the conference was also extremely helpful. They shared their first-hand experiences and provided insight into what my child is going through. It is more difficult for a child to describe how he or she feels because what he or she experiences on an everyday basis is their “normal.” By sharing their experiences with me, the attendees were able to help me find the words to help my child describe how HE is feeling.

Part of this disease is a feeling of loneliness – for Jedi, Jedi’s brother (our other son), and my husband and me. Unlike more common disorders, finding and becoming part of a support group can be difficult for those with an MPN—especially since it is so rare in a child. That is why it is important for us to participate in conferences whenever possible. This Fall MPN Advocacy & Education Int’l is hosting a conference especially for children and young adults with MPNs. This is a fantastic opportunity for both parents and children to meet and get to know one another. We plan to attend this conference. Learn more about the Pediatric MPN event.

Finally, a plea to adults with an MPN. Please consider using the resources the MPN groups has provided, such as the tool that tracks symptoms. I know it can be concerning to share that information with a third party. (Believe me, I am wary of doing that myself.) But, any information YOU provide will help those that come behind you. Working together, we can collectively help each other and future generations better understand how to combat and defeat these diseases.

 

 

A Veteran’s Story: The Frustrations of Filing a Claim with the VA

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By Wayne E.

MPN Patient and Vietnam Veteran Wayne E.

I served in the USAF Security Service, 6924th Security Squadron, stationed in Da Nang, Vietnam for one year (1970-1971) and was exposed to the deadly Agent Orange/Dioxin. In 2007, after a simple pre-op blood test, I was diagnosed with essential thrombocythemia (ET). Upon further study I was told I had an incurable, but manageable, blood cancer, coupled with a gene mutation (JAK2). The word cancer scared me. I had never heard of ET and I was at a loss for what to do. I didn’t know where to go next. After much reading about these potentially deadly diseases, I found out I was one of many Vietnam Veterans who had an MPN.

In 2011, I filed my first claim with the VA. Until this filing, I was unable to get any substantial information from my primary care physician (PHP) or my hematologist/oncologist, as to what may have caused or contributed to my ET. They knew virtually nothing about Agent Orange. I contacted the National Institutes of Health, The Centers for Disease Control, and as many online medical sites as possible, all ending with a bigger question mark. Nothing could be explained to satisfy my inquiry.

It was by chance that I connected with a most remarkable group, MPN Advocacy and Education International. I could never thank them enough for the compassion and the understanding they extended to me.

After my initial rejection from the VA, I filed three more times and each time I was denied because MPNs are not on the “presumptive” list of Agent Orange-related illnesses. The same message I kept getting was I needed “clinical rationale” to support my claims. My doctors have not been able to provide me with this needed information. I don’t know what to do today. I understand there are many Vietnam vets that have won their appeals and now get benefits, but there are many others who were not approved and just gave up. I don’t plan to give up.

To my fellow Vietnam Veterans who may be dealing with one of these MPNs, don’t give up. If you have been denied, file an appeal. There is hope, comfort, and assistance available. With the help of MPN Advocacy and Education International.

 Learn more about filing a claim with the VA

 Learn more about Veterans and MPNs

A Veteran’s Story Told By His Wife

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Bill C. Veteran and MPN Patient

In January 2017, my husband, Bill, was enjoying his consulting business and writing a book that has been in his mind for years, when an annual physical changed everything. Some lab work was “off” according to the Veterans Administration (VA) physician so Bill sent the lab results to our personal physician, who is also our best friend. Within ten minutes after receiving it, our friend asked Bill to come to his clinic immediately.  Further blood tests yielded an initial diagnosis of Chronic Myeloid Leukemia (CML).

While we were reeling from that shock and trying to ground ourselves, our physician friend sent us to a local hematologist for additional work-ups and treatment.  Following a bone marrow test, we were further shocked to find out that the CML we had become somewhat resolved with was indeed Myelofibrosis (MF).  We sat in a dumb stupor trying to figure out what that was, how serious it was, where it came from, what we could do about it, etc.  We were encouraged to start on the only medication for Myelofibrosis, Jakafi, and were told the only drawback to the drug was its cost–$10,000/month!  We immediately started working with the VA for them to supply the medication.  After numerous telephone calls and in-person visits with both our hematologist and the VA (in a city 40 miles from our home), we secured VA support for Bill’s Jakafi.  It now routinely comes to our home in an innocuous package.  The initial symptoms Bill was experiencing responded to the Jakafi but so did the platelets and hemoglobin which are continuing to drop so we played with the dosage to, hopefully, continue to drive the white blood count down while keeping the platelet count and hemoglobin up closer to where they should be.  The drop in Jakafi was too drastic and symptoms immediately returned so our local hematologist moved Bill back to the original dosage. Symptoms once again are gone and platelets somewhat controlled but still very low. Hemoglobin is recovering which is good.

While all of this was going on, we decided, with the support of our local hematologist (who is wonderful realizing this is all about Bill and not about the hematologist’s ego), to go to Rochester, MN to Mayo Clinic to see one of the leading researchers in Myelofibrosis, Dr. Tefferi.  While there was no proactive guidance offered from this visit, we did learn that we should only approach researchers whose field of study is a fit between their interests and Bill’s current health status.  To that end, we went to Northwestern University in Chicago to see Dr. Brady Stein, another renowned Myelofibrosis researcher. He listened and answered all of our questions while assessing Bill’s fitness for ongoing clinical trials. His ultimate recommendation was for us to consider a transplant—again, another shock as we had hoped that we would have a variety of alternative treatments  Dr. Stein is concerned that Bill’s Next Gen Sequence report that showed other mutations limit the time he will have before he converts to Acute Myeloid Leukemia (AML). Since Bill is in such good health right now (ironically), Dr. Stein found him to be amongst 10% of people with MF that even qualify for a transplant consultation and while a transplant is a “rough ride” encouraged us to explore it.

To that end, we have met with Dr. Tom Chauncey who is the Program Director at the VA in Puget Sound-who along with the University of Washington at Seattle are the number one transplant center (particularly for people with MF) in the country. Dr. Chauncey was very generous with his time and counsel and offered to work with us as we continue to explore transplant options. With Dr. Stein’s support, we also will meet with the Director of the Northwestern University Transplant Program, Dr. Mehta.

Simultaneously, we filed a VA benefits claim related to Myelofibrosis, believing Bill’s exposure to Agent Orange most likely caused this illness, but we were denied.  Bill is a Vietnam Veteran having served in Quang Tri—I Corp from November, 1968 – November, 1969 and was exposed to Agent Orange/Dioxin.  While compiling our appeal information, we found numerous Citations where the VA had granted benefits for veterans (on appeal) who have been diagnosed with Myelofibrosis due to exposure to Agent Orange, so will be using that information to move our claim forward.  During the exploration for more information, we also discovered that the VA is finalizing a rule to add to the benefits structure Stem Cell Transplant coverage as well as treatment protocol to include myelosuppressive therapies of which Jakafi is one. This proposed change is set to take effect in FY18 (which begins as soon as October 1, 2017) which is exciting for veterans waiting for coverage of Myelofibrosis because, at least, some of the symptoms and associated therapies will be addressed.

MPN Advocacy & Education International continues to advocate for essential thrombocythemia, myelofibrosis and polycythemia vera to be included in the VA’s ‘presumptive’ list of illnesses related to Agent Orange exposure. Please click here if you are in the process of filing a claim or appealing a claim for more details.

Like many of you, we are sure, our world right now is exploring drugs in Phase II or III of clinical trials that are successful in producing remission in MF as well as other drug trials/existing drugs that will hold down the “blasts” that would otherwise convert Bill to AML. We are also exploring transplant centers, protocol, outcomes, experiences, etc. to get a better sense of whether that is something we even want to consider.

In the midst of all of this, we continue to work hard to enjoy our lives. Having a daughter with Down Syndrome who is now 43 years old taught us that nothing is ever guaranteed and that we would have to fight for anything and everything we wanted.  We “cut our teeth” on the fights for our daughter, Mindie, against insurmountable odds and won.  Now we are using those skills on Bill’s behalf.  While sad and scared, we remain determined that there are many, many opportunities for Bill to remain as healthy as he is today and live a long and enjoyable life beyond the current prognosis.   We know there is a lot to learn from all of you “in our same boat” and look forward to sharing stories and guidance between all of us.  In the interim, all of you touched by an MPN are in our thoughts and prayers.  Together we can change the face of these diseases!