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!

By Ron K.

Fortunately, my MPN journey has been a long one. I was first diagnosed 32 years ago and I know have post-Polycythemia Vera Myelofibrosis. Looking back it was almost archaic in nature. Today the progress has been immense on many fronts, not just in treatment, but with treating the patient as a whole. As many of you know, there is no exact recipe book for life with cancer and its treatment journey is unique.  My hope is that sharing some of my experiences may help someone else along the way.

There is a quote by Albert Einstein that reflects my approach to my medical journey

Learn from yesterday, Live for today, Hope for tomorrow. The important part is to not stop questioning.

Learn, Live, Hope – three good words for any cancer patient.

As those with an MPN know this is a chronic neoplasm and unfortunately there is no treatment yet to cure this illness. Some of the latest MPN and MF drugs do improve one’s quality of life and even appear to extend one’s survival, a significant milestone from what was available just a handful of years ago. Finding ways to slow or stop the progression is my only hope until medical knowledge can surpass me with a real solution.

Having a chronic cancer and having it for a long time has enabled and encouraged me to evolve into a healthier lifestyle. One does not have to change everything overnight but just evolve and continue to build healthier ways, one step at a time. While there is no evidence that a healthy lifestyle will cure me, it is something I can do and have control over.

This all started back in 1985 when I went to my family doctor with a cold and I mentioned that my colds seem to last longer and hit me harder than others.  A simple blood test began my journey.  I would not be alive today if I followed my first hematologist’s treatment plan. He wanted to treat me in a traditional manner at that time, with a radio isotope that kills off some of the marrow, they know now that treatment leads to an acute leukemia years down the road. Instead my intuition questioned this approach, since I was only 31 and I thought that I might need that marrow later in life.  I called a major metro medical center and asked for an appointment with their Chief of Hematology. Unknown to me at the time, and not commonly termed in the 1980’s, I was going to get something called a second opinion. I got an appointment and his approach was quite different. He wanted to understand me, my rate of progression, severity, etc as that would lead him to figure out how to best treat me. He even commented that the current radio isotope treatment (my first hematologist’s plan) to kill off marrow was likely going away as he saw it.

Today, I am a patient of Dr. Mesa’s. I found that they could answer all my questions. Answers to questions that my previous hematologists could not answer. They have knowledge that is very leading edge as to direction of MPN treatments as they are part of an elite group of hematologists that are making many of the MPN discoveries, designing the clinical trials and studies with other key hematologists around the world.  So my personal doctor advice is to make sure you find the right doctor and treatment strategy for you. Listen to your intuition and ask questions, seek information and acquire knowledge in your cancer and health. Expect answers or find those professionals that can give them to you. While I did not need a MPN specific doctor early on I do need one now as I am living well beyond the averages.

For me, finding the right doctor has been a key in my cancer treatment journey. Getting a second opinion is also key. What you will likely find is the different doctors will agree on the diagnosis but their treatment strategy will often be different.

I attend conferences, seminars, and special programs such as those that the MPN Advocacy and Education International puts on. I have done online webinars or conferences as well as belong to online MPN listing groups to learn and share information. I find this information to be helpful to ask the right questions to my doctors and understand what may be option in the future.

I believe support makes a difference. It is helpful to have support from your spouse, kids, parents or friends. I also find that support groups can be very helpful as well. As they can fill a support void that is missing from one’s non-cancer family and friends. This is no fault of family, spouses, or friends in that they do listen and they do try to understand and relate and they do support the cancer patient, but I have found that belonging to a cancer support group often helps to fulfill things non-cancer patients cannot quite grasp. I have belonged to both hematology specific support groups and all cancers support groups. You might want to try both and see if one works better than another.

As I live my life, I am grateful for every day. Everyday is a great day, just some are even better than others.  I even celebrated the point in my life where I have lived longer with cancer as part of my life than without cancer. That was at about 31 1/2 years and I had a bakery make me a cake to celebrate the occasion. The cake was symbolic of my MPN journey and view of life. I celebrated this occasion with my all cancer support group. So while cancer is a part of my life I run my life with cancer being a part of it and I do not let my cancer rule over me.  I am hopeful that a cure is in our much sunnier MPN journey. May the best be yet to come, and enjoy the day!

Suggestions for being an effective caregiver

Caring for a person with cancer may seem complex and like too much to cope with at first. Depending on the person’s needs, you may provide different types of support, such as:

• Emotional support
• Help with medical care
• Assist with financial and insurance issues
• Serve as the communicator between the patient and the health care team

You may find the following tips can help you become a successful caregiver:

Remember that caregiving is a team effort. A caregiver is a member of an important team of family members, friends, volunteers, and the health care team. Each member of the team offers different skills and strengths to provide effective care.

If you are the main caregiver, help each team member express concerns, opinions, and emotions. Also, make sure that the person with cancer has a central role in all discussions and decisions, if possible. It is very important for the person with cancer to feel like an active member in their care.

CancerCare provides a special website for caregivers called My Cancer Circle.  This website can help caregivers organize the family and friends who want to help. Find other online communities for support.

Create a list of tasks. Caregiving, like any responsibility, involves tasks of varying importance. Start by making a list of all of your caregiving tasks. Then, use it to decide how to divide the tasks between friends, family, professionals, and other volunteers.

Be proactive. Being proactive means taking charge and planning as much as possible to prevent last-minute emergencies. This can also help provide a sense of control and order. Create schedules that list which relative, friend, or other volunteer is available when and for what tasks. Make sure that all of the caregivers involved have some time to be away without feeling guilty or concerned. Long-distance caregiving takes even more planning. Find out more about how to be an effective long-distance caregiver.

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 At what point in your MPN diagnosis did you join a clinical trial?  What trial (drug) were you in?

I was diagnosed with PV in 2001.  Only on phlebotomy and 81mg aspirin until 2007 when I began hydroxyurea because I turned 60 in 2006 and was now “high risk.”  In 2009 my spleen was enlarging. I had been hearing that Interferon was best taken in the early stage of MF and might reduce myelofibrosis.  My hematologist advised me he would be having a trial with Pegasys (Interferon) and I should wait for that. I actually waited until 2012 when the trial became available.  I was now showing signs through bone marrow biopsies of secondary MF.
Did you feel as though you received accurate information getting into the trial?

Yes, I had been reading about Interferon treatment and knew I wanted to try it.  Perhaps I didn’t know how quickly I could be removed from the trial because of my worsening symptoms.
What didn’t you know going into the trial that would be useful for others to know or questions they might ask?

It would have helped to know at what point you may be pulled from the trial and what happens after you are removed.  Also, I did not know that the FDA can shut a trial down at any point due to adverse reactions to few patients.

How many clinical trials have you participated in?

I am currently on my 3rd clinical trial.  (First trial wasn’t improving my symptoms, second trial was shut down by the FDA and the pharma company decided not to proceed , and the third trial I have begun my 4th year.)

Were you able to get the drugs after the trial(s) if you were doing well?

I can continue with the drug I am currently on as long as I continue to have benefit and as long as there is funding for this drug by the pharma company, and that it doesn’t get shut down prior to FDA approval

What advice would you impart to those who are skeptical about clinical trials who are not doing well on available treatments?

What do you have to lose?  If you are currently not well, you certainly may have good results and feel better.  If you don’t show benefit you will be removed to try something else.  We do not know the future after taking these drugs.  That is a gamble we must take.

Would you participate in other trials knowing what you know now?  

If I was not feeling well and I met the qualifications, I would try again.

Marylin C. has been living with her diagnosis for many years.  She has graciously volunteered for trials not only for her own reasons, but for the sake of MPN patients and their futures.

Ron Anderson

Ron Anderson, LA Support Group Coordinator, received a bone marrow transplant eight years ago.  His brother, a perfect match, was his donor.  We asked his brother to share his experience to get a donor’s perspective.

Was there ever a time, once you knew you were a perfect match for your brother, that you thought you would not be his donor?

No, by the time his doctor told him he needed a bone marrow transplant he was seriously ill and probably would not survive for very long without a total transplant.  As it turned out, my brother and I have type A+ blood and (thank God) I turned out to be a very good match for him.  I just felt very fortunate I would be a great donor and could help him.

When I told him I was willing to be his donor I thought I would be going into a hospital operating room and have doctors use needles to remove my marrow from my pelvic bone.  Later, I found out about Peripheral Blood Stem Cells (PBSCs) and a process called apheresis.  My stem cells could be collected by drawing blood out of my veins, filtering them out and the rest of my blood would be given back to me.   I was very pleased to learn about this nonsurgical method.

How has this experience changed you?

For the first time I took a hard look at my entire family’s age/health issues and felt there was a real possibility that I might not have any family members alive in the next 5 to 10 years.  That was not a very comforting thought for me.  I think this experience with Ron not only strengthened my relationship with him but with my entire family.  Since Ron’s transplant in 2009 I have tried not to take my family for granted.  Since 2009 both of my parents have passed away but I am very thankful I still have my two brothers.

What, if any, were the side effects?

The only side effects I can remember was some mild hip and shoulder joint pain issues after taking the drug Neupogen to increase my stem cell production.  It went away within a reasonable time frame (I think about a couple of weeks) after I stopped taking that medication.  The only other issue I experienced was during my hospital blood collection time when I had some tingling around my mouth apparently from decreased calcium blood levels from the filtering process of my stem cells.   That tingling totally stopped after the apheresis when completed.

Can you briefly explain the experience in the hospital?

All the hospital staff were great and they gave a very good explanation of what would be happening during my stay.  They were very caring and made me feel very comfortable during the apheresis process.

In the hospital, I sat in a comfortable lounge chair for about 6 hours a day for 2 days (because I also agreed to give extra cells to stem cell researchers) squeezing a soft ball with my hand during the stem cell collection process.  As my blood calcium levels dropped during the apheresis procedure calcium had to be replaced back into my vein which sometimes caused that vein to fail and infiltrate.  I think the worst part of the hospital stay was the restarting of new I.Vs.  I had about 4 or 5 new I.V. restarts a day and some pretty good bruises on the back of my hands and forearms by the time the apheresis was completed.  At the end of the day, a very small price to pay for helping my brother.

I served in the Army during a more recent war than many of the veterans who have been diagnosed with an MPN.  I was diagnosed with Essential Thrombocythemia (ET) in early 2013. My son was only a year old and I had spent two weeks in a VA hospital while hematologists tried to rule out other illnesses. I was put on Hydroxyurea (Hydrea) almost immediately due to the aggressive rise of my platelet count. It was eventually controlled and I was sent off with a permanent Hydrea prescription.

My background in health care and my university education prompted me to research this illness and I learned a lot.  I read about alternative drugs, but the drug that stood out to me the most was peginterferon alfa-2a (Pegasys). It’s what was advised for young females of childbearing age, as it isn’t harmful to a fetus. I also learned that Pegasys is the only known drug that gives a glimmer of hope for remission. That’s when I decided I wanted to be on Pegasys. Hydrea was giving me terrible side effects.

I joined several MPN support groups on Facebook. This is probably the best thing I ever did for my condition. The wealth of information in those groups is astounding. I met many people who were very proactive in finding the correct treatments that alleviated some of their symptoms.

I was fortunate to have the wherewithal to find this out on my own.  I’m sure there are many veterans who do not or cannot advocate for themselves and are probably too tired, too sick or too confident in the VA to research this for themselves. Or maybe many of them had but had been denied, like I was.

At my visit with my VA oncologist, I mentioned Pegasys and my desire to be on it. I was told that Hydrea is the standard drug for ET and that all other patients are on it. I was adamant to get on Pegasys and was told that only Melanoma patients received it. Luckily, I had a receptive and understanding oncologist who said he’d research it first before giving it to me. After doing his own research, he agreed. I think I was the first ET-VA patient in Central Texas to be put on Pegasys. To this day, I believe I’m still the only one.

I was very lucky.  I’m sure this does not happen often with other VA physicians.  My other mission was to be seen by an MPN expert.  Making all these discoveries on my own really showed me how rare MPNs are and how unaware many VA hematologist/oncologists are about the best current available treatments.  This is my life…but not just mine, my son’s too.  I owe it to him to improve my health and extend my life.

My hematologist/oncologist agreed to submit a request to get seen at MD Anderson in Houston. I was placed into the Veteran’s Choice program and repeatedly given appointments with local civilian physician.  After letting the program know time and time again, that I wanted to go to MD Anderson, my oncologist told me it had been rejected because the VA doesn’t approve “second opinions.”

I was disappointed but grateful that, at least, I was on Pegasys. About a year later, I decided to prepare to have another baby. My VA gynecologist advised me to let my oncologist know of this. At my appointment, I told my oncologist my plans and he said he’d put in another request for the VA to cover a visit to see an MPN expert. I didn’t get my hopes up.

Sometime after, I got a call saying I was approved for two visits at MD Anderson. I saw an MPN expert who basically told me I was on the right medication (Pegasys) but that I was taking it too often because my white blood cell count was low and I was very fatigued (among other disturbing symptoms). He decreased the frequency and I felt much better with my white blood cell count being within normal range for the first time in three years. MD Anderson also tested me for mutations that the VA couldn’t perform. I had many questions answered and was finally at peace with how my condition was being handled. I was ready to move forward confidently, I felt closure.

None of this would have happened if I had been a man. The VA only approved my MD Anderson visit after learning about my intention to get pregnant. As a female, I’m a minority at the VA. There are many male vets who aren’t benefiting from a visit with an MPN expert.

If I hadn’t done my own research on ET, I would still be on Hydrea today. The side effects were very trying. Pegasys wasn’t even on their radar. Even if the VA approves prescriptions for Pegasys, Anagrelide, Jakafi or something else, the patients could be suffering from dosage/frequency problems, like I was. An MPN expert can help sort this out, and so much more.

I encourage MPN patients and their oncologists to do their own research. Veterans have the right to know about alternate treatments. Veterans also need to see knowledgeable experts and have access to blood tests that are relevant to their condition.

My daughter has always been a very active, happy child. She is very creative, loves animals and art. She has always participated in sports. Currently, she runs cross country and plays field hockey and loves her ceramics and art classes. If you look at her, it is impossible to see a sick child, you’d never think she’s different or that there is something wrong with her health.

When she was 8 years old our family moved to Kentucky. Her new pediatrician requested a blood test, which was something she said she did for all of her new patients. My daughter had never had blood drawn as part of a normal check in any of the states that we had previously lived. At first it felt very invasive, but we agreed. To our surprise, it was discovered that her blood platelet count was at almost 2 million. The pediatrician of course requested a second test for validation and again the results were the same.

As parents we were shocked because my daughter had never had headaches, bad bruising without reason or ever felt fatigued. Every question we were asked regarding any symptoms she may have exhibited, our answer was no. She never presented any symptoms. After the first visit to the hematologist, we were told my daughter needed to get a bone marrow biopsy. At this point our world was upside down.  Why a bone marrow biopsy? What are the doctors talking about?  We had little to no answers. This is when we decided to start looking for help, after all she is our daughter and we needed guidance. At our church one of our deacons is on the medical board of a university, so we reached out to him for guidance and a better explanation. He was able to ease our worries by explaining the reasons for the bone marrow biopsy was to give us a better understanding of what my daughter had.

Once the bone marrow biopsy was done, we got the diagnosis that it was a JAK2 mutation causing Essential Trombocythemia, again we didn’t really understand what we were facing, this was a rare illness for adults and even rarer in children. They wanted to start a treatment that was usually given for another illness, but that it might help keep the blood platelets under control. Our whole life changed, we had a diagnosis but no real answers about this condition. She was the first patient in our area to be diagnosed with ET and there was little to no information about it, our child didn’t look sick or feel sick, however she needed to take hydroxyurea, a type of chemotherapy.

I can tell you that I was very upset and sad, I’m supposed to protect my child, but how? Having no answers from doctors made me feel even worse. For my own peace of mind, I decided to do my own research, I spent hours on the computer and talking to our deacon trying to figure something out. Then I came across the MPN organization and MPN Advocacy and I subscribed to get their emails and magazines, I soon realized that there was very little to no information about this condition in pediatrics.

Meanwhile my daughter started the treatment and she would ask me why, I don’t feel sick, why do I have to take this? Because of her age it was difficult to try and explain everything in a way she could understand, but we did it the best way we could. We told her we loved her and we would never let her down and everything was going to be OK.

My daughter went from doing regular, normal 8 year old things to having to visit the hematologist every other week, going through all kinds of testing and taking this chemo medicine. Her platelets went down, but after a couple of months she started vomiting and having headaches, my daughter had never had headaches or presented anything like this. There were constant visits to the ER or the pediatrician every time she was feeling sick, there were constant tests and blood draws, but nobody knew exactly what was wrong. At one point her blood platelet count went down below normal and that’s when the doctor adjusted the medicine to a lower dosage and she started feeling better, the headaches still continued, just a little more infrequently but now she presented another symptom, she felt tingling in her hands and feet.

We tried to keep her life as normal as we could but the constant headaches made it impossible, it affected her school work and attendance, I would have to pick her up every time she was sick. It affected her activities so she didn’t want to take the medicine any more. We visited all kinds of specialists, a neurologist, an ophthalmologist, just to make sure there wasn’t something we were missing but every single test came back fine. Our frustration grew and so did our need for help.

My daughter didn’t want to go to the hematologist any more, she was trying so hard to be happy, yet we can see that she was sad. She had never spent so much time in bed due to headaches, she didn’t want to take the medicine that made her feel sick anymore, she would ask, why me?

At age 9 she needed to get another bone marrow biopsy, it was very scary for her, I still remember when they put her to sleep, she looked so little and so fragile. You’re supposed to protect your children, but what can I do against this?

I continued to research ET and found that MPN Advocacy was having a seminar in Boston. My husband and I decided we needed to attend it to see if we could get some answers. I contacted Ann Brazeau and told her about our daughter and that we needed help finding more answers about ET. We wanted to learn about this condition but also wanted to try and seek out help from somebody that knew more about this. Ann not only had answers for us but she put us in contact with Dr. Nicole Kucine. We attended the seminar and it was the best decision we could have made, we learned so much about the MPN conditions and the medicines used to treat it and we also got the opportunity to meet Dr. Nicole Kucine, a leading ET specialist who specializes in pediatrics. We later took our daughter to see Dr. Kucine and she was able to answer many of our questions and give us some different options and ideas that put us in a more confident state of mind.

My daughter now feels more confident when it comes to talking about her condition, she understands that we can’t keep it a secret. In school, teachers need to know that even though she looks fine and can function like any other 9 year old, she has ET and some days are going be bad for her. She can’t understand everything at her age, but she has a strong spirit and just keeps going.

The biggest difference between the first day of this journey and now is the understanding and knowledge we gained by attending the MPN Advocacy and Education’s MPN program in Boston. We learned so much about our daughter’s condition and we gained new tools to support and help our daughter. We understand there is still so much research to do and knowledge to gain about ET in children. We know that we may never get all the questions answered for my daughter, but we take it one day at a time. She continues to attend school, she runs cross country, plays field hockey and takes ceramics classes. She continues to do well in school and for better or worse when she becomes sick she has been able to adapt and can control it better and recovers quickly. But we are looking forward to the day that we can lower her medicine to a level where she can live with no side effects. We let her know that we love her and we would do everything in our power to help her. We are thankful to God, the support of friends and family, people like Ann Brazeau, the MPN Advocacy and doctors like Dr. Nicole Kucine who dedicate their time and lives to teaching and informing parents like us.

By Silvia