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HFA: 25 Years of Advocacy

Laurie Kelley April 7, 2019

Hemophilia Federation of America (HFA) celebrated its 25th anniversary this past week in San Diego. I remember HFA in its infancy, when its annual meeting drew about 100 attendees. This past week over 1,200 people, mostly consumers, gathered from all parts of the country to learn, socialize, hold remembrances, and give awards.

Laurie Kelley with Carri Nease and family

An HFA meeting is also a chance to network, to brainstorm new ideas to meet the ongoing needs of the bleeding disorders community. Three hot topics? Women and bleeding disorders: how to include them in our data, discussions, treatment and future. Gene therapy: what will it look like, cost and be available? And perhaps the most urgent: mental health issues and how to identify and treat them.

This last issue for me is paramount. A year ago we lost Barry Haarde, who was a prominent contributor and attendee at HFA. His absence was very much felt. Since his passing, so many community members have opened up about their own mental health issues, including depression and anxiety. To address these issues, I was privileged to listen in on some exciting programs and ideas that could help not only patients but caregivers better deal with stress, sadness and overwhelm.

My good friend Debbie de la Riva, who knew Barry, a fellow Texan, started “Mental Health Matters Too,” following his death, to bring the issue forth in meetings across the country. Her program is appearing everywhere it seems. Visit Debbie’s website, www.MentalHealthMattersToo.com, to see if it will be coming to your state. I know it’s coming to New England in October, and I hope to attend.

HFA CEO Kimberly Haugstad
at Award Ceremony

The three days of meetings felt like a week, and I mean that in a great way! It’s always a wonderful feeling to spend time in this community, which is like family. I’ve been in this community for 31 years, and one of my highlights of the week is to bump into people like Sam Doughty of Tennessee, who I have known since he was a child. Age 34 now, and president of the Tennessee chapter, Sam is a fine example of young men who have taken the reins not only of their disorder but also of the future of the hemophilia community.

The conference was capped off with an incredible final night aboard the USS Midway. Above the sea and under stars we had drinks, food, dancing and fireworks! Check out photos here to see more! Thank you, HFA, for making such a huge difference in so many lives, and for giving us the chance to have a reunion; it strengthen the bonds and brings us joy. Bleeding disorders can never defeat us!

Remembering a Visionary

Laurie Kelley April 1, 2019

March is Hemophilia Awareness Month, and this year, also the 15th anniversary of the passing of a visionary in our community: Dave Madeiros. Dave passed away from complication of hepatitis C at age 47 in Boca Raton, Florida, just two days after I had visited him. He was a friend, mentor, businessman and amazing visionary.

If you ask what the greatest complication of hemophilia is in the US now, many will say insurance. It was Dave who first sounded the alarm in 2003 that insurance was undergoing massive changes that would eventually impact hemophilia care. Each quarter, Dave would invite a select group of people to his headquarters in Boca, to share and brainstorm about what we called “The Coming Storm.” Included in this group was Dana Kuhn, founder and president of PSI, very knowledgeable about insurance issues. And Ed Burke, who worked for Dave. Dave was founder and president of Factor Foundation of America, a specialty pharmacy that gave away profits back into the community. Dave’s dream was to become the number one specialty pharmacy serving the bleeding disorders community, where 70% of profits would be channeled back into the community.

Dave predicted the coming changes in insurance amazingly well. He told us as early as 2003 that insurers would: start restricting access to certain therapies; would challenge whether patients needed so much factor; would start step therapy; would focus on the bottom line more than on patient needs; that PBMs would become megaplayers in the industry.

Of course, he was right. All that he predicted came true. And he predicted this at a time when no one else in our community seemed to know anything about what was to happen. NHF had no clue; they were busy undergoing an internal restructuring. Based on what Dave shared, I wrote an article called “The Coming Storm” in PEN, outlining the predicted changes. It became one of our most popular articles, and indeed, the community started referring to the rapidly changing landscape as the coming storm. These changes continue even today. NHF and HFA got on board after the article was published, and now are the foremost advocates for preserving access to care nationally. (Download the article here)

I feel we as a community owe a lot to Dave. Myriad presentations, meetings, marches, and more are all due to his early efforts to be like Paul Revere, and warn us that the “Payers are coming!” He had insight and shared it; he changed the way we thought about our community, roused us from complacency, and put us on the right path to protect our needs and interest.

We lost a great man when he passed away February 29, 2004. He left behind a wonderful wife, Kim, who you might meet these days at a hemophilia meeting. He left behind his prized car, a Mustang Shelby. But mostly he left behind an incredible legacy. Let’s remember him and honor him for eternally helping our community, as a visionary leader.

Sage Advice to Younger Guys

Laurie Kelley March 24, 2019

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.

Our Celtic Logo

Laurie Kelley March 17, 2019

It’s St. Patrick’s Day, and being of Irish descent (fourth generation), I thought I’d share the origins of our Celtic swirl in our logo. Logos are important; the name itself derives from the Latin word for “meaning.” Logos can be in script (like Coca-Cola’s logo), a graphic (like Nike’s “swoosh”) or an image (like KFC’s Colonel).

NHF’s logo shows two human figures: a red figure with an arm around a white figure. The red figure represents a person with factor. The white figure represents a person without factor.

We chose a graphic. The green swirl in our logo is a modern adaptation of the centuries-old Celtic swirls carved into rocks surrounding the base of the ancient tomb at Newgrange, Boyne Valley, Ireland. Built more than 3,500 years ago, Newgrange is thought to be one of the earliest manmade structures, predating the pyramids and even Stonehenge.

The meaning of the elegant swirls remains unknown, but the design is one of humankind’s earliest and simplest forms of communication.

The color green represents the Irish heritage of the name Kelley. The Celtic swirl demonstrates energy and momentum. At LA Kelley Communications, we believe that written or oral communication can be simple and elegant, yet can convey a powerful message. Our communication is dynamic, educating and compelling people to take action for change.

For more information about Newgrange, please visit; www.knowth.com/newgrange.htm.

And Happy St. Pat’s Day!

New Therapies, New Era

Laurie Kelley March 11, 2019

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 Amazon.com 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.

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.