March 2019

Sage Advice to Younger Guys

Stephen C. Place

Here’s my advice for “bleeders.”  A bleeding disorder is lifelong, from birth and until death.  My perspective is from someone age 64, with mild hemophilia A.  My motto is “Firsthand personal experience trumps all.”

The most important priority in the life of a bleeder and their family is to accept the disorder.  Over time, consider it a blessing.  I most certainly do. Fighting the diagnosis will result in disaster.  

Once you have accepted that your blood will not clot like others, your journey will be positive for you and your family.  For sure, it’s not all about you.  Your family is affected by every decision you make in all situations. 

My experience tells me that having a hematologist on board is a first priority.  I love regular doctors, NPs, and nurses, but their training is not sufficient to advise you about the treatment of this special disorder.  Hematologists are the experts.  They are up-to-date on all the new technology and treatments.  Have an annual exam and LISTEN TO THEM!

I have learned over the course of my lifetime to treat a bleed early and often.  Follow your treatment protocol to the letter. Your hemophilia team should be able to prescribe a treatment plan for a particular bleed via a phone call.  Text them a picture of the situation. A picture is worth a thousand words. 

Think.  Think.  Think of the consequences of what you are about to do.  After a very serious head injury from riding my bicycle “no handed” (bad decision), I was told to avoid contact sports.  OUCH!  That really hurt, more so emotionally, than the physical injury.  Well, after I got over the shock and awe of my new lifestyle, I decided the ball was in my court (pun intended).  I made a firm decision to take responsibility for my disorder, become my own advocate, and get on with life.   This is one of the best decisions I have ever made. 

Even with this first step, I continued with the idea of proving to the world, and myself, that I really did not have a bleeding disorder. I refused to learn to self-infuse until I was 60 years old (another bad decision).  Imagine going to an ER for a long over-due infusion where a nurse said to me, “I have been a nurse for 30 years and have never seen factor VIII before.” WOW!  What was I thinking? Now as a “self-infuser” my bleeds are shorter and the life-long consequences of an untreated bleed are minimized.  Joint bleeds can cause serious problems for an older bleeder.

We make decisions every day.  Some are non-consequential, while others can have serious consequences.  Remember, again, it’s not all about you.  Every decision you make will affect other people. 

My firm opinion, with great wisdom, is to avoid participating in contact sports.  Yes, this is tough for a young person because of school age children and parents that are uninformed about bleeding disorders.  You will experience discrimination because of hemophilia throughout your life.  Be ready for it.  Plan your response and be strong.  This will be a great character builder.

Parents, as the most important people in your child’s life, steer them to a life as free from bleeds as possible. Please don’t wrap them in cotton, but allow them to experience life as much as possible, helping them to make good decisions.   Steer them into activities free from danger, that will help to build character and a profession that will benefit humanity.  Prompt them to opt for music, science, medicine, a trade, finance and so many others that will protect them from serious consequences.  Help them to choose a good life course.  If it becomes their decision, you have done your job.

Back to bleeds.  I strongly believe, from experience, that if I need to treat, I screwed up.  Plain and simple.  Again, this is from a mild hemophiliac who treats on demand.  “If I had just thought for a second, I wouldn’t be infusing and missing the opportunity to complete my job”.  Again, think, think, think.  Time is my most valuable asset.  Treating an avoidable injury is the biggest waste of time and money.   Aruggh!

Now, about insurance.  Picking a good plan is vital.  Look for assistance everywhere.  Check for state and federal programs that will assist you in paying the premiums.  Check for state plans that will cover your deductibles and co-pays.  Check with your pharmaceutical manufacturer.  There is funding available. Don’t feel guilty accepting help. The best source for this information is your HTC social worker. Just ask!

Life with a bleeding disorder can be great, if you choose to accept it and use it to your personal benefit. Think of the situations you can respectfully avoid that you really don’t want to do. Yes, you have a legitimate reason, not excuse, to avoid sky diving, knife twirling, drag racing and other potentially life threatening activities.  Get smart.  Log your bleeds, their causes, and the result of your treatment.  Help yourself and others to avoid a bleed by documenting the cause of the injury. Embrace your disorder by learning everything and anything you can about it. 

New Therapies, New Era

I attended the New England Hemophilia Association’s Consumer Medical Symposium Saturday in Portland, Maine. It was fabulous! Over 200 consumers, medical staff and guest speakers attended. As always, after 31 years in this community, it’s like a family reunion. But it’s also a time of great learning.

Rich Pezzillo, executive director of NEHA, opens the symposium

We were fortunate to have Dr. Steven Pipe, chair of NHF’s Medical and Scientific Advisory Committee. He provided an excellent and understandable overview of current products, new products and gene therapy, and how they all coexist. It was truly a brilliant presentation. Dr. Pipe started by saying that our community is having some of the most monumental changes in hemophilia that we’ve ever gone through. He talked about standard factor, the factor used prior to newer therapies like extended half-life (EHL) products. Why was EHL developed? To help preserve joints better; to reduce the “burden” of prophylaxis (frequent infusions, wear and tear on the veins). But he reminds us that even though EHL products extend the half life, these products also have a trough, when levels will dip and when a child will be less protected from a bleed.

In discussing the newest therapy, Dr. Pipe calls Hemlibra “substitution therapy.” It is not a factor product. It bridges factor IXa and factor X, bringing them together to allow a clot to form. He made everyone laugh when he called Hemlibra the “eHarmony” of clotting. Factor VIII normally acts as a scaffolding, allowing FIX and FIXa to come together. When there is no factor VIII, the scaffolding cannot support the two. So Hemlibra can now do this. And this new structure looks nothing like factor VIII so it is less likely to encourage inhibitor formation. It has a long half life, in some cases up to 30 days. And it’s administered subcutaneously, so no more trying to secure little wiggly veins. The outcomes of the HAVEN studies were displayed and discussed, causing Dr. Pipe to say that he had never seen anything like them. This nonfactor therapy provides a steady trough as well, not the dipping troughs of EHL or standard factor. It’s also effective in inhibitor and noninhibitor patients. (Download PEN’s Hemlibra article here)

What’s not to like? Well, there are unknowns. Although Dr. Pipe has used Hemlibra in a two year old, patients are being monitored for thrombotic risks, assay issues, annual expenses, and peak bleeding risk events. You can’t do an assay of Hemlibra, unlike factor concentrates. And one third of patients using it will experience some bleeding issues.

Dr. Pipe also showed on a slide the scales of hemostasis; how everything needs to be in balance. Not only do we need to try to clot the blood, but we need the blood not to overclot. The body has its own natural mechnisms for stopping the blood clotting process with naturally occurring antifibrinolytics. Some new therapies will try to remove the antifibrinolytics, which might allow the blood to continue to clot. These are all low burden therapies, desgined to restore the balance in hemostasis.

Dr. Pipe used as a metaphor for how gene therapy could work. The box your order comes in is like the vector carrying the human gene that will be inserted into the body. It’s targeted to the liver, and once implanted, will manufacture factor VIII normally. There are currently 5 gene therapy trials underway for FVIII and even more for FIX.

Dr. Steven Pipe, Chair, MASAC

And I learned something new. Victoria Kuhn asked a pertinent question: why weren’t women being included in the clinical trials of these products and gene therapy? Dr. Pipe replied that women and children were considered “unique high risk groups” by the ethics committee of the FDA. A woman’s eggs could be affected by gene therapy; no one knows for sure. The good news is that Dr. Pipe believes gene therapy will be viable in 2020 or 2021.

He ended his superb presentation by agreeing that there is no one product that everyone should be on, as everyone’s body is different. Glenn Roy, a nurse from Maine, agreed and added that his center does PK studies on every patient, to determine the best therapy for them. What’s important to you? Shaving off one extra infusion? Avoided vein wear and tear? Out of pocket costs?

Mary Fitzgerald, a mother of an adult son with hemophilia who I have known for decades, reminded the younger parents in the audience to be cautious about rushing to new therapies: an entire generation did that and was lost. There are known risks, and risks that are as yet unknown.

Laurie Kelley and Rich Pezzillo, part of a “wicked strong family”

It was a multifaceted look at new therapies and therapies yet to come, to prepare ourselves with knowledge, when the time comes to make an informed decision.

See photo gallery of the day here.

Kudos to NEHA for a wonderful day and event! Thanks to everyone who traveled so far to come and share their wisdom with us in Maine.

Honoring a Little Boy and a Great Man

Last week, I traveled to California, for an exclusive showing of the wonderful movie “Bombardier Blood” to employees of NuFactor, the hemophilia distribution program of FFF Enterprises. We gathered at the Omni Resort in Carlsbad, where Chris Bombardier and I marveled at the brilliant blue skies, warm sunshine and swaying palm trees. The movie was warmly received; there was a standing O before it even ended, in honor of Chris’s achievements! But the best moment for me was being able to tell employees the gratitude I have for their founder and CEO, Patrick M. Schmidt.

Laurie Kelley and
Patrick M. Schmidt

Patrick and I have a long history, meeting on August 12,1995, when he was attending a business meeting in Boston. He called me the previous week, out of the blue—I had no idea who he was. He said he had heard of me, saw my books, admired my work and independent stance (I was not part of an HTC, specialty pharmacy, manufacturer or nonprofit. Back then, you were one of these). He was just entering the hemophilia community, and wanted to pick my brain. So we met over a cup of “chowda” in downtown Boston. I found Patrick sincere, eager to get involved, and respectful. And he remains so to this day.

During lunch, he asked me a question that reverberates today: What can I do to make a difference?

It happened that I was still reeling from the murder the previous summer of a beautiful little 5-year-old boy. On a hot summer day in August 1994, I was leaning against my kitchen counter and opened the Boston Globe. The headlines screamed that a little boy from Easthampton, Massachusetts had been murdered. According to his stepmother, some strangers entered the house a few days before, on a quiet Saturday, smothered the child with a pillow, hit the stepmother in the head, and left the baby in the crib unharmed. Nothing was taken. There were no footprints, fingerprints and no one saw anything.

The greatest shock was that I knew this child. He had hemophilia. His grandmother, Jennie Gosselin, had been sending me photos and stories to publish in a children’s newsletter I once had, called “Factor Fun.” The headlines dominated for the week, until the stepmother was arrested for premeditated murder. She remains in prison to this day, and will be for life. The reason she killed this child? His medical bills were too expensive, and she wanted another child but couldn’t afford one.

This touched Patrick deeply. That a child would be murdered because his medical bills were too expensive was abhorrent. Also, Eric was the same age as his daughter, Natalie. I can see even now the look of horror and dismay on his face as he considered all this. Did he want to make a difference? I suggested a scholarship in Eric’s name, so that he would not be forgotten. Patrick immediately approved. The scholarship is now in its 23rd year! It has helped hundreds of students attend college.

Eric Dostie with grandpa George

I remained friends with Eric’s grandmother right up until she passed away two years ago. Before she moved to North Carolina, I would visit her in Easthampton and we would stop by Eric’s grave. She was forever grateful to a man she never met for his act of compassion and generosity.

If you are a US student with a bleeding disorder in your family, you can apply for the Eric Dostie Memorial Scholarship. This year’s application is closed, but mark it on your calendar for the fall, when you can apply again. And check out our scholarship page on our website, which lists even more you can apply to.

Many thanks to Patrick M. Schmidt for his friendship, mentorship and support. The movie “Bombardier Blood” showcases Chris’s climbs, but also the work we do at Save One Life. Patrick, by the way, is our number one sponsor, supporting 180 children who live in poverty. And he is our top donor. He believes in giving children a chance in life.

He has changed so many lives around the world… including mine.

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