Dr. Gary McClain explains the delicate balance between empowering your child to manage their own bleeding disorder while managing your own worries. Learn how to face the “what-ifs,” build your own stress tolerance, and give your child the tools they need to make decisions and grow.
Reflect on your own definition of a fulfilling life and explore the multifaceted concept of quality of life with seasoned therapist, Dr. Gary McClain. Find guidance to advocate for yours and your family's well-being through ideas to embrace life's possibilities and acquire a future with improved quality of life despite the challenges posed by chronic conditions.
Those of us in the inhibitor community who attended the Inhibitor Summits have been hearing about future therapies that would potentially provide more efficacious treatment for years. As with all product development, the process to get to market is long, and not always successful. Product safety remains a priority, especially for our bleeding disorder community. These new therapies, however, bring hope to the inhibitor community; especially for those with hemophilia B and an inhibitor who have very few options for effective treatment. Rebalancing therapies that increase thrombin by lowering anticoagulants are within reach. There is a lot to know and understand about their mechanism of action.
Familiarize yourself with the vocabulary
Take advantage of webinars or educational resources as they present themselves
And ask your healthcare professional (HCP) LOTS of questions
Product choice is ultimately a decision made between you/your child and your HCP. We know well in this community, that not every product works the same for everyone, and if you know one inhibitor patient-you know ONE inhibitor patient.
Shellye Horowitz, a well-known and active member of the bleeding disorders community, seized the opportunity to attend CHES Foundation's 1st (hemophilia with) inhibitors program that includes adults, teens, and adolescents, as well as caregivers. InhibitCon is the first time members of nearly all ages affected or indirectly affected by inhibitors gathered since the Inhibitor Summits.
by Ashley Hall
Published: LifeLines for Health, vol. 21 (2024)
We first learned that our son Maverick had developed inhibitors after spending the first few months of his life in the hospital. We were devastated by the news and after hearing that word, we were in total shock! How could this happen? Why us? But most importantly why him? It was such a scary and frightening time in our lives, and we were so lost! We thought we were already pros at dealing with the hemophilia part since it ran in my family and my middle son had already been diagnosed as having severe hemophilia A.
That’s when I set out on a mission to start learning anything and everything I could about what life with an inhibitor was about to start looking like for us. We were in uncharted territory as no two cases I had found were the same. All I knew is that it had never ran in my family previously, so we were clueless when it came to the inhibitor aspect. I started to read stories where some kids beat them, while some never could. We hardly knew any families with young kids, and I just remember feeling so scared and confused with so many questions still left unanswered.
That’s when someone messaged me one day telling me about CHES Foundation, Inc and invited us to attend their camp, After the Shock, so that we could meet families just like ours going through some of the exact same things! That someone also just so happened to take me under her wing and helped teach me everything she knew about inhibitors and treated us like family from the start! I will forever be grateful for Mrs. Janet! She is and forever will be my inhibitor mama!!
We jumped at the opportunity and I’m so glad we did! CHES provided us with a safe space to vent about medical issues going on in our lives, yet we were also able to get much needed clarity and guidance by other families going through similar treatments. We bonded instantly with other families and my kids were able to meet other kids going through very similar situations! They were able to have a weekend of some type of normalcy where they could laugh and play and run free knowing we had the proper help on standby if anything were to occur.
The After the Shock Inhibitor Family camp provided onsite nurses, industry representatives who were available to answer any questions they could about certain medications and most importantly we had HELP! We had a personal helper called a Pal who had inhibitors himself so we knew we could relax and be stress free knowing they knew how to help in case an injury occurred. It was a weekend of fun and fellowship and most importantly education.
My kids loved fishing, archery, and we all enjoyed the cooking class together. Although Maverick at some point seemed to be enjoying eating the food more than the actual cooking part! Just seeing our kids run around with other kids like them made the journey up to Camp Zeke in Pennsylvania for the After the Shock camp well worth it! The educational classes were spot on and just what my husband and I needed to help us make more educated decisions in our future! This is a smaller community that is much needed to help give us the mental and physical support we desperately needed! Nobody should fight inhibitiors alone, and having an army of people who understand exactly what you’re going through makes you feel like you can conquer anything! Especially these pesky inhibitors!
We made friendships that turned into family and bonds that will last a lifetime. Whenever we attend a new program and learn something new that we didn’t know before, that is always a win in our book. We have been fighting Maverick’s inhibitor through every other day Immune Tolerance Induction Treatment for the past year and a half and currently still fighting. Although everyday seems like a challenge, we remain hopeful and can’t wait until we get the chance to reunite with our inhibitor families once again!
Reflect on your own definition of a fulfilling life and explore the multifaceted concept of quality of life with seasoned therapist, Dr. Gary McClain. Find guidance to advocate for yours and your family's well-being through ideas to embrace life's possibilities and acquire a future with improved quality of life despite the challenges posed by chronic conditions.
Published: LifeLines for Health, vol. 3 (Summer 2014)
They say that a picture is worth a thousand words. But what if three people were to describe that same picture? Is it still a thousand words, or is it now three-thousand? After all, we all seem to hold different viewpoints on everything from the sun to the bumps on a crocodile’s back. The Zhao family of Pennsylvania has 3 stories to share, but all about the same experience. Learn how Inhibitor Family Camp means a little something different to each of them.
A Mom’s Perspective
by Sha Ha
“We come from the mountains, living in the mountains. Go back to the mountains, and turn the world around.” (Clap! Clap! Clap!) Singing coming from the seats in front of me woke me up on a late night flighthomefromCalifornia.Obviously, Ryan and Lily were still having fun because of the new friends they made from the weekend. I smiled, but felt tears coming down my cheeks. Images from the past 13 years flashed back through my mind. I remembered how my world almost collapsed when my son Ryan, six months after birth, developed a huge hematoma in his right eye from a slight bump of his head on the crib. Ryan bled for more than ten days while my husband and I waited desperately for a diagnosis. To this day, the image of Ryan with a bloody eye still wakes me up at night from time to time. Ryan was eventually diagnosed with severe Hemophilia A, and later with inhibitors. In the years that followed, emergency room and operating room visits almost became a routine in our lives. I often couldn’t remember how many ports Ryan had during that time, only to be reminded by counting the scars on his chest. Because he didn’t respond to the immune tolerance treatments, Ryan kept bleeding in his joints, muscle, neck, and most scary of all, his spinal cord. We were drained, helpless, and alone. We didn’t know what the future would hold. We were desperate to find other families we could talk to. We learned about the Inhibitor Family Camp in 2012 and decided to give it a try, not sure how the experience would turn out. We came back with full luggage: a Big Stick Award for Ryan for his first self- infusion and a list of families who rode the same roller coaster as we did and who taught us that there were no hurdles we could not overcome. I made a photobook of this life-changing event and have formed a habit to open it whenever I need an extra dose of encouragement.
We have become loyal campers ever since. This year we went to The Painted Turtle, a beautiful camp snuggled against the mountains of Southern California. We met new families and reunited with old ones. The children’s cheers and laughter echoed all weekend long in the mountains and on the lake. We the parents, gathered at evening parents’ café. There, we no longer felt lonely and helpless. There, families from all over the country bonded together. There, we saw a future for our kids.
“We come from the Turtle, living in the Turtle. Go out from the Turtle, and turn the world around.” (Clap! Clap! Clap!) The kids’ humming of the camp song brought me back to the airplane ride. Our world has indeed been turned around - by Inhibitor Family Camp. The world has become smallerbecause we have gotten to know families from other parts of the country; the world has become warmer because we no longer feel alone; the world has become brighter because we have built such strong community bonds. Thank you, CHES, The Painted Turtle, and the wonderful volunteers. You have made us stronger. We have a mission to accomplish and it is every bit POSSIBLE!
A Camper’s Perspective
by Ryan Zhao
The trip to the Painted Turtle was a wonderful experience for me. The best thing about it was that I felt a sense of belonging. In normal camps, I always feel awkward, or held back because of my hemophilia. I was different from everyone else and that created a barrier between other campers and me. At the Painted Turtle, however, I was free! I could do what I really wanted to do, instead of trying to act like a “normal” person. I could talk to others about hemophilia as opposed to hiding it. Everyone there had experienced the challenges of hemophilia and an inhibitor. That brought us closer together and allowed me to make many new friends.
The activities at the Painted Turtle were also amazing! They had everything from fishing to woodshop to horseback riding. There was a gym for the more active people, and a wide outdoor pavilion at arts and crafts for those who like to be indoors. I loved the woodshop and the arts and crafts center. They had such a wide variety of things to do. I could spend days there without ever getting bored! At the woodshop, my mom and I worked hard on a Pinewood Derby car. My mom had a great idea of making a painted turtle, and I turned it into a reality. We called our car “The Painted Turtle,” feeling confident that it would be a great ride. And it sure was! At the Pinewood Derby where the whole camp gathered, each family participated in the race. Creativity and competitiveness ran high. Pun’s humorous commentary filled the room with laughter. The competition was friendly but intense. In the end, “The Painted Turtle” won! That is a moment I will treasure for the rest of my life. Finally, the trip would not have been the same without the wonderful peoplethere at camp. The CHESorganizers, camp staff andactivity pals were so nice! In the woodshop, Pops was a big help. He knew how to use the woodcutting tools,and helped me shape my derby car. Pun’s puns filled my days with laughter and taught me many great life lessons too. The family pals were like family. Kelsie and K-Pod accompanied me everywhere and showed me where everything was. They were like the big sisters I always wanted to have. They made camp feel like a vacation home. The three days at the Painted Turtle passed too fast. As I waved good-bye to the red cabins, the warm California sun, and the many new friends, I promised myself that, “Painted Turtle, I WILL BE BACK!”
A Sibling’s Perspective
by Lily Zhao
This past April, my family went to the Painted Turtle. We went because my brother, Ryan, has hemophilia with an inhibitor. My family and I had so much fun at the Painted Turtle. I wanted to stay there for another week!
The activities were my favorite part. There was archery, boating, fishing, wood shop, arts and crafts, and horseback riding. In the evening, we also had a carnival and stage night. I liked all of the activities, but if Ihadtopickmy three favorites, I would choose horseback riding, the carnival, and arts and crafts. In horseback riding, we got to ride on a horse/pony and go through obstacles. The horse I rode on was named Diamond. In arts and crafts, you could make almost anything you wanted. You could even decorate a rock and put it in the “rock garden.” The carnival was so much fun. There were fun games, dancing and even prizes.
Other things I liked about the Painted Turtle were the weather and the cabins. The weather was warm and breezy, perfect for a hike. The cabins were amazing! They were nice and cozy. Last but not least (which I thought was the best), was having the family pals and staff joining us. They were helpful, funny, and kind. “Pun” and “Pops” were very funny and exciting. Kelsie (our family pal) was like a big sister. We were sad she had to leave early.“Pun”toldveryfunnyjokes.Forexample,whenIfirstmet him he said that my mom was my older sister and that I was 29 years old. I had a very good time at the Painted Turtle. I hope next year my family will come here again!
by Mark Zatyrka
Published: LifeLines for Health, vol. 3 (Summer 2014)
Over the past ten years our bleeding disorders community has been dealing with the major issue of not having access to qualified factor providers. Many families are losing their choice of providers, or at best being frequently challenged in that choice.
When a member of the bleeding disorders community finds a good HHC (home healthcare company, also known as homecare or home infusion company, specialty pharmacy, and for the sake of this article, HTCs who sell factor through its’ 340B program), they tend to grow very tight bonds and have a level of comfort and trust with their HHC. It can be invaluable to have someone who you can always call, 24/7, who knows your exact needs and preferences, your history, etc. Having a reliable HHC can ease the burdens that come with living with hemophilia and its’ complications, such as an inhibitor. It is important that when it comes to the health of your child or yourself, you know the right factor will arrive at the right place at the right time and should an emergency arise, your HHC will be there for you.
This reality is all especially true for families living with an inhibitor, who may need even more specialized and personalized services. You need to be able to have access to a company who knows inhibitors inside and out. You have enough to deal with and no one has the time to explain every month what an inhibitor is, why you need so much factor, why you need multiple brands of factor, what a bypassing agent is and so on.
Accessing the Situation
Before you begin to fight the battle with an insurer who won’t let you use the HHC you trust and are comfortable with, it can be helpful to know why they are making the decisions they are making.
There are usually two reasons why an insurer will not allow a subscriber, who has out-of-network benefits, to use the HHC of their choice.
1. The insurer signed a “one-stop-shop” deal with one or two large pharmacies
These pharmacies may not have the lowest per unit price on factor, but the insurer sees value in the pharmacies’ pricing on all the drugs across the board as well as cost savings in only having to deal with one or two pharmacy providers.
2. “Vertical Integration”
This trend is one of the scariest in healthcare today. One definition states, “vertical integration is where the supply chain of a company is owned by that company.” In healthcare, vertical integration happens when the insurer owns the pharmacy benefit manager (PBM), the pharmacy, and even the hospital. For example, Xyz Insurance Company tells all their subscribers with hemophilia that they need to use Xyz Specialty Pharmacy if they want their factor paid for. This is a huge conflict of interest issue and we, the patients, are the ones who suffer. Like the above reason #1, the pharmacies have zero incentive to provide quality service. Once insurers remove your ability to bring your business elsewhere, it no longer matters if you are happy.
Because of these two reasons, you can see why getting the insurer to make an exception for you is such a hard battle to win.
We always hear that insurers are doing what they are doing to “control costs,” but when you look at the big picture, it is clear that it is much more about increasing profits than it is about controlling costs.
In the past, a HHC could offer a lower per unit price for factor in efforts to work with families to keep them on their services. In the two reasons listed above, neither one cares about the per unit price. HHCs today rarely if ever set their reimbursement price. Insurers decide what they will pay and that’s what HHCs accept. In fact, most insurers will not even accept lower rates when offered. When we have a client at AHF who will need large amounts of factor, we usually contact the insurer to try to negotiate a lower rate. Surprisingly, insurers have never taken us up on it. We get the sense that they do not want to create more paperwork and exceptions that they need to keep track of. We have even offered to accept a much lower rate to help a family stay on our service and again, that offer has always been declined. These experiences prove the “per unit price” is not the insurer’s priority when choosing which HHCs their subscribers are allowed to access.
Below are some tips you can try when your insurer is preventing you from choosing a new HHC or prohibiting you from staying with your current HHC. But keep in mind, the way insurers and PBMs are set up now, these battles are lost more than they are won, so the odds are stacked up against you. (But we are used to that, right!)
Tip 1: Know your plan
You can ask your HR department or call your insurer’s Customer Service dept. and ask for a written copy of your “Certificate of Coverage” (COC). This should be provided free of charge. This document explains the health benefits you and your dependents have under your plan. It details the services that will and will not be covered. This is a large document, usually 50-200 pages long. The 1-2 page outline of your co-pays and deductibles has some good information, but in order to really know what your rights are and how your factor is billed, you need your full COC. Every insurer is different, every plan type within the insurer is different, and every individual plan can be different.
Tip 2: Be prepared
If you are told a change is happening, be sure to place your monthly factor order with your current HHC prior to the deadline given to you by your insurer. You do not want to be left without factor and need to order from your insurer’s preferred provider. Not only can it take a week or more to get set up with these big PBMs, it also becomes nearly impossible to get permission to switch back to your preferred HHC once you place an order with the insurer’s preferred provider. Verifying your insurance, waiting on your physician to send over your prescription, waiting on prior authorizations and/or pre-certifications and delivery time all can take time to happen. Make sure you have adequate factor on hand for the transition.
Tip 3: Find out if your HHC is willing to fight for you?
It is difficult enough to win these battles. You really need to have your HHC fight for you because this process can consume many hours. Who you need to talk to, what questions do you need to ask, and how to ask those questions are all very important. Your HHC needs to put in the hours, and then be willing to assume some level of risk.
Tip 4: Do not take their word for it
Many times you will receive a letter from your insurer informing you that you will no longer be able to use your current HHC and you will have to switch to the insurer’s preferred provider. Some of these letters are real and some are a scare tactic. You and your HHC should call to see if there are any changes to your plan. A lot of times there are not. It is always good to get the necessary prior authorizations or pre-certifications before shipping just to make sure.
Some treatment teams will recommend their patients call their insurer to find out which providers they can use, which is definitely the easiest path to picking your HHC. However, if there were a particular HHC you would like to use, I would not recommend calling your insurer. They will tell you who they want you to use. I would suggest calling the HHC you would like to use and ask them to run your insurance information. A lot of times the HHC can bill differently, such as out-of-network, through a third party payer (TPA), or through your medical
plan, which may then allow you to use them.
Tip 5: Find out if you have out-of-network benefits
This information can be found in your COC. If you or someone in your family has a chronic illness, I always recommend picking a plan with out-of-network benefits, such as a PPO, if you can afford the higher premiums. It not only can affect which HHC you are allowed to use, but also which doctors and which hospitals you are allowed to go to for care.
If you do have a plan with out-of-network benefits, it becomes very difficult for insurers to
prevent you from using the HHC of your choice. Unfortunately insurers still find a way to deny people their out-of-network benefits even when they pay extra to have those out-of-network benefits. Some payers have moved factor to their pharmacy benefit, which requires you to use their preferred pharmacy (that they usually own), or they can simply “carve out” hemophilia and mandate which provider people with hemophilia are allowed to use, which is a discriminatory practice.
That said, many plans with out-of-network benefits will still allow you to choose your HHC. They may just give you and your HHC the runaround for a while. That is why it is so important to have your COC so you can recite the actual verbiage of your plan.
Be aware that when you do exercise your out-of-network benefits, there is usually higher co-pays or deductibles. Ask your HHC if they have a hardship program or if they work with the manufacturer of the brand of factor you use, as almost all of the manufacturers have generous co- pay assistance programs and patient assistance programs (PAPs).
Tip 6: Find out if your plan is fully-funded or self-funded
Fully-funded plans are your traditional plans. Self-funded plans are usually plans offered by large employers where the employer pays the claims themselves and they hire an insurer to administer the plan. Again, this can be found in the COC. Employers with self-funded plans usually have an additional insurance policy specifically to offset the cost of an employee with a high cost disorder, such as an inhibitor.
There are pros and cons to self-funded plans. A con for self-funded plans would be that they are not regulated by the government. So they could decide not to cover factor. That is usually not the case because good employers want happy employees. But it does happen. That said, the fact that the employer can have a lot more say and has the power to make exceptions would be a major pro for self-funded plans. So if you have a self-funded plan, you would want to go to your HR department and tell them how important it is for you to have access to your HHC. If they value you enough, they can make that change.
Tip 7: Find out if factor is covered under your medical benefit or your pharmacy benefit
Factor used to always be covered under the major medical benefit. The trend lately has been to
move factor over to the pharmacy benefit. Insurers have a bit more control when factor is billed on the pharmacy side, but we usually have much more freedom to choose our HHC when it is billed on the medical side. Check your COC. It will tell you which side factor is billed on. It will also tell you if there are exceptions. Often times, if a nurse or professional is doing the infusion, the factor can be billed under the medical benefit, which usually allows you to stay with your HHC. Also, if factor is billed under the pharmacy benefit, you are at risk of paying those high specialty tier co-
pays.
Tip 8: Ask to appeal
If your insurer is telling you that you need to switch to a different HHC, ask if there is an urgent appeal process or special consideration process. Follow that process exactly and act fast...because they do not. You can usually ask your doctor for a letter and your HHC can help you construct a letter from you stating your case. Don’t be afraid to share how difficult life can be, what services you are receiving from your current HHC, and why they are so important. Even though it does not carry the weight it used to, stress how important continuity of care is for your family. Share what you have been through and what could happen if you receive poor services.
Tip 9: Contact your hemophilia patient organizations
Contact your local, state, regional, or national hemophilia organizations and ask for support. Some organizations are definitely more helpful than others. They may be able to put you in touch with the right individuals. Ask if they can help you prepare or defend your case to your insurer or provide you with up to date info on any insurance law changes in your state. They may also be able to put you in touch with other families who are having the same problems, usually with the same insurer. Your voices together will be much stronger than your voice alone. They may also help get the media involved if it’s a big enough issue.
Published: LifeLines for Health, vol. 3 (Summer 2014)
Today, nearly 40 years after effective treatments for hemophilia A became widely available, there are many of us living with an inhibitor that causes uncontrolled bleeding episodes, increased disability and decreased quality of life. For some of us, these effects can be compared to those individuals who lived with hemophilia prior to factor replacement. In approximately 30% of all individuals with severe hemophilia A, inhibitors form in response to FVIII therapy, rendering any routine treatment ineffective.
Due to the history of pathogen transmission in the 70’s and mid 80’s from human-source FVIII, recombinant
FVIII is often the only treatment option patients are given. As many of us have discovered, however, not all FVIII products work the same for each of us. Some of us have found that after years of unsuccessfully trying to treat with different recombinant products, bleeding episodes can be managed better with human-source FVIII.
Due to the changes and advances in plasma collection, manufacturing and production over the past 20 years, perhaps it is reasonable to consider human- source FVIII as an option for patients with difficult to treat symptoms of hemophilia A.
History of Factor VIII
Human-source FVIII became widely available in the 1970’s, allowing hemophilia A patients, for the first time, to treat bleeding episodes at home, to travel and to have surgery1. These products created a significant improvement in the quality of life for individuals with hemophilia. Lengthy hospital stays and lengthy treatments became a thing of the past. The lifespan of an individual with hemophilia A could now be increased dramatically. It wasn’t long after their introduction however, that it was discovered the use of human-source FVIII carried a risk of pathogen transmission. Many of us had family members who became infected in the 1970’s and 1980’s. It was by far the most tragic era in the history of hemophilia treatment.
Recombinant versions of FVIII, first introduced in the early 1990’s,1 were not just hailed as welcome additions to the hemophilia A treatment toolbox, but signified a complete change in the way we treat hemophilia A to this day. Along with increased product choice and supply of available treatments, the risk of pathogen transmission was significantly decreased. Prophylaxis treatment became the standard of care, drastically reducing the number and severity of bleeds and the subsequent joint damage they cause. For many of us, prophylaxis meant the ability to live an otherwise “normal” life with the opportunities to participate in sports and other activities that would have been considered too risky, dangerous or impossible.
Managing a chronic neutralizing inhibitor is a daunting challenge. Its’ impact on the individual and the family’s overall quality of life is debilitating.Many of us have spent precious years and resources attempting immune tolerance induction (ITI) with large, frequent infusions of recombinant FVIII,or large, frequent volumes of bypassing agents with little to no success. The options for treatment are extremely limited, while the bleeds andcomplications are abundant. Reconsidering human-source FVIII – not an easy choice given our community’s history, as a way to manage bleeding episodes soon became the only choice for many of us. Faced with a diminished quality of life, many of us consulted with our medical teams or began researching the safety of human- source plasma on our own as a possible treatment option. What was found was that our bleeding episodes could again be managed. For many, immune tolerance was successfully completed with human-source product.
It is highly doubtful that there is a single medication, prescription or otherwise that does not carry the risk of side effects, some more harmful than others. As stewards of our own (or our child’s) health, it is imperative to know the risks, consequences and benefits of any medication we use. Our community should never forget the devastating effects of products that were not stringently screened, tested and manufactured.
The biggest challenge in the hemophilia community today is inhibitors. Although no one can say with certainty what causes them, we do know that at least 30% of the severe hemophilia A population will develop one. We as custodians of our own (or child’s) health in conjunction with our health care team need to know all of the options for treatment available to us, along with their possible consequences. This knowledge for those of us living with an inhibitor could make a drastic difference in bleed management, joint health and overall quality of life.
Final Thought
We do not all have the same response to FVIII products. There are those of us who develop inhibitors or are poor half-life responders. We have learned that some of us do respond successfully to human-source FVIII products.
No one knows for sure why, but after years of uncontrolled bleeding, many of us wish we had considered human-source products long ago. We would like to encourage physicians, nurses, and caregivers that it is okay to bring up the topic of human-source FVIII products in the discussion with patients and parents, and vice-versa.
Authored by:
Jake Rollins - Adult with severe hemophilia A. Human-source FVIII successfully tolerized his inhibitor.
Janet Brewer - Mother of 2 sons with severe hemophilia A. One of her sons developed an inhibitor which was partially tolerized after switching to human-source FVIII.
Eric Lowe - Adult with severe hemophilia A. Human-source FVIII successfully tolerized his inhibitor.
References
1. Lusher JM. Development and introduction of recombinant factor VIII - a children’s experience. Haemophilia. 2012;18:483-86
2. Viel KR, Afshin A, Abshire TC, lyer, RV et.al. Inhibitors of Factor VIII in black patients with hemophilia. N Engl J Med. 2009;360:1618-27.
3. www.pptaglobal.org Plasma Protein Therapeutics Association (PPTA)
4. Klamroth R et al. Pathogen inactivation and remocal methods for plasma-derived clotting factor concentrates. Transfusion. 2013:9.
