Origins: Part 4 Turning Point

It was 1989. My son with hemophilia was two, and we had had some experiences with bleeds. There was the potential head bleed at nine months, when our baby was sitting up but still wobbly, and pitched backwards and hit his head on the hard wood floor. We rushed him to Children’s Hospital for his first infusion. I was crying more than him, and was ordered out of the room, while our nurse tried to secure a vein. I wandered the hospital halls, crying to myself, and when I returned, saw everyone, including my baby, laughing and just fine. That’s when it dawned on me that I had more problems with the infusion than my baby!

There was the bleed at age 10 months, when our son stood up in his crib. He was teething and gnawed on the crib railing. I saw blood dripping from his mouth and realized he had cut his gums. But suddenly the bleed stopped! He was moderate, after all, and perhaps the bleeding would stay stopped. I put him down to sleep.

In the morning, I opened his door, and there he stood, one little hand on the crib rail, and one holding his bottle, covered from head to waist in blood. Only his round white eyes seemed visible. He was smiling a grisly, bloody smile. If I hadn’t known it was from a small cut in his gum, I would have panicked. This time I remained calm, and cleaned him up as best I could. Blood was in his ears, hair, nose… everywhere and the gum was actively bleeding now. So back to Children’s Hospital for an infusion. We had to wrap him in a bedsheet to sneak him out of the house. We lived in Medford, Massachusetts, a town where the houses are crammed together and where there is no privacy! We spent the morning at the hospital, and he got a second infusion.

When sharing this story, now with a bit of humor, at our monthly parents’ support group meeting, one of the moms asked if we had used Amicar. Never heard of it! She was surprised, and we were surprised. Amicar helps neutralize saliva so that it won’t break down a clot in the mouth. Why had no one at Children’s told us about this? Had we known, and given him a dose that first night, when the bleeding stopped temporarily, maybe we could have avoided a hospital visit and infusion.

It dawned on me that parents have first-hand knowledge and advice that maybe even the medical team doesn’t know or remember.

We remained tethered to our HTC. The turning point came September 29, 1989, when my son had just turned two. It was like a new me was born. An advocate. A real hemo-mom!

I will never forget that morning. I was dressed up to go to work (it was the ‘80s!): silk dress, big hair, big earrings, jewelry, and high heels. I worked at DRI/McGraw-Hill, a private economic forecasting company. My dream job. I worked with intelligent, interesting people creating and delivering economic forecasts for different industries. I felt at last everything was going great and hemophilia was under control. That all would change.

Wearing those high heels, I drove my son to the babysitter, as usual. Carrying him from my car to the front door, I slipped on something, Maybe mulch, a stone… who knows? The baby pitched forward from me, and landed headfirst on the driveway. Our babysitter cried, I cried, the baby cried. I put him back in the car and we rushed to the hospital. My husband met us there, after the sitter called him. Imagine, no cell phones!

I recall waiting and waiting at the hospital. No infusion at first, just x-rays, and many doctors standing around discussing what should be done. We waited hours. No food, no juice, and finally, no patience. My son, who had been running around the hospital halls, started to fuss and cry. A nurse tried to pin him down on a table for an MRI, but my son would have none of it. She said, “I’m going to get a doctor to help hold him down.” My husband looked angry (and he never did), and frustrated. We talked about what was happening, and I said, “Let’s get out of here.”

It seemed radical, controversial. We were saying we were leaving the hospital without approval. The team had finally infused him, but were not making any decisions about next steps. And our son hit the breaking point. We knew the symptoms of a head bleed. We could monitor him. So we announced we were leaving, and were told, No you can’t! Yes, we could. And somehow I knew I was right.

We signed a stack of papers, and left. All weekend we monitored him, and into the next week. He was fine. No sign of a head bleed.

When I shared this episode with our support group, our nurse Jocelyn made the suggestion that perhaps I wanted to write this down? I guess I sounded emotional, and she might have felt I needed to sort out my feelings. But I actually was feeling very empowered. I had made a medical decision. I could make decisions about my son’s health. It was a revelation. I was a person who seemed to escape all physical problems as a child; the worst thing that happened to me was getting stitches in 5th grade. I had no experience dealing with hospitals and doctors until I actually had my baby. It was a revelation that I could make this medical decision.

So I started writing. But I also remembered all the wisdom I had heard in the parent support group. I started writing that down. Little by little, the writing exercise became something more. What if I were to ask parents of children with hemophilia to share their stories? How empowering would that be? I know I needed their information and advice.

I called the National Hemophilia Foundation and asked the advice of two women who worked in their educational department. They were adamant: a book would be a great idea, but not just stories. “We want a guidebook,” said one of the women. “At each stage of life,” added the other.

That was a lot more than I had originally dreamed up. But why not? A book about how to raise a child with hemophilia, based on advice from parents. I had a degree in child psychology and had published research. I knew how to research.

The book idea became even more urgent when I learned there was not just one brand of factor, and one homecare company, as I was led to believe. I had a choice. And I was a consumer, not just a patient. I could make my own decisions there too. My husband knew all about the medicine, because he worked in the field. We could include a whole chapter on being a consumer.

The book idea became a reality, a passion, a need. Now all I needed was a way to solicit stories… and get funding.

Next: Phone calls pour in, and one postcard turns the tide.

Know Your Factor Choices

It’s truly a new era in bleeding disorders with new products, new therapies and new indications. Octapharma’s wilate just got approval for indication in adults and adolescents with hemophilia A; previously it was indicated only for VWD. With this news, I realized that we should remind everyone to know what product they use, where it comes from, and why they are using it.

Who chose your product? Was it your hematologist? Did you have any input? You should, and this means bearing the responsibility to read about products, as questions about them and be part of the decision making process.

Back in 1990, when Raising a Child with Hemophilia was first published, we were the first ones to describe parents and patients as consumers, and to treat products like any other commercial product you would buy. We provided the question to ask your doctor, and we explained the different manufacturing processes, and the difference between safety and purity. And we urged parents not to be passive in the treatment process but to help make medical decisions.

It’s more important now than ever. We have a dazzling choice of products, and novel therapies like Hemlibra. Maybe there’s no reason for you to switch products, but you might want to get up to speed on what’s available. Please note: all US FDA-approved products are considered safe and efficacious.

We’ve been writing about products and product choice for decades. I hope you read up on them and be prepared! To help you, we created a chart of products that you can download. Take it to the HTC with you and discuss with your hematologist about choice. You can download it right from our home page or go here.

We also have a chart for VWD products too!

We did our part. Now do yours; know your product, manufacturer, and why you use it. Keep learning… more therapy choices are coming!

A Personal Approach to Healthcare


Ever hear of personalized healthcare (PHC)? PHC means tailoring a treatment regimen specific to an individual patient. It acknowledges that every patient is different, with different physiology, biology, reactions to medicine, and lifestyle. A treatment regimen that works for one patient may not work for another with the exact same ailment.

The goal of PHC is to improve your quality of life as a patient with a specific disease or disorder, by taking into
account your individual needs and lifestyle, and then tailoring a treatment plan. How does that happen? It’s a collaborative process involving you and your medical team. Your HTC medical team knows all the treatment options, but you know yourself, your body, and your lifestyle. To develop your own PHC, you’ll need to share with the team every aspect of your past health, current health, needs, and desires. Only then can you all explore treatment options that best suit you.

Your Personalized Healthcare Team

The best place to get help with hemophilia and all bleeding disorders is at a hemophilia treatment center (HTC). These are centers of excellence that specialize in diagnosing, treating, and monitoring bleeding disorders. There are over 140 HTCs throughout the US. They follow the model of comprehensive healthcare, which means that they’re not just treating bleeding episodes, but all aspects of living with a bleeding disorder. From orthopedics to psychosocial needs to genetics, the HTC team knows how a bleeding disorder can impact your life.

By using comprehensive healthcare, isn’t your HTC team already implementing PHC?

Maybe not. First, not all HTCs are created equal. Some may lack certain experts you need, such as a pediatric hematology department or a geneticist. And some may have strong beliefs, different from yours, about when—or whether—to start prophylaxis (prophy), or about whether you should try new products.

Parents and patients need to become partners in PHC. Are they ready for this collaboration? Parents of newly
diagnosed children may be too shocked at first, and not even know what questions to ask. Older patients may be overlooked for PHC because they’ve been on the same treatment plan for a long time, their blood work is good, and they don’t complain or ask questions.

It’s important for you to be ready to partner with your HTC and let the team know your needs. But even if you’re comfortable standing up and being heard, what will you say? What will you ask?


One of the first decisions you need to make about your PHC is which treatment to use. All products licensed in the US are considered safe and effective, but they’re not all the same. How do you find one that’s best for you?

You know that new products are entering the market. You can choose between plasma-derived (made from human blood) and recombinant (made from animal cells containing human genes). Within the recombinant products, there are categories: first, second, or third generation. And there are novel therapies, that are not even factor! So begin by asking your hematologist for opinions on all products.

Your decision about treatment may come down to lifestyle, when selecting a particular brand for prophy. Perhaps the brand you’re using now, three times a week, works well for you or your child. Does a product with an extended half-life offer fewer infusions, saving veins from wear and tear? Or would you benefit from a new, subcutaneous product? Some products might not be available through your insurance. Talk through these choices with your HTC.


Perhaps nothing is more personal than your individual bleeding pattern. People with hemophilia bleed differently, in different places, from different causes. Once your child begins getting bleeds, notice his bleeding pattern, if any. This is where you can really help your HTC team personalize your treatment. The information you share can help prevent a target joint from starting, or can compel the HTC team to put your child on prophy.

You may hear severity levels described like this: Children with severe hemophilia will bleed from trauma, or spontaneously, with no known trauma. Children with moderate hemophilia may bleed on average once a month, with known trauma. Children with mild hemophilia may bleed only after dental extractions and surgery. But what’s the reality? Some children with severe hemophilia bleed only monthly; some with moderate bleed every week, with no known trauma. Your child’s bleeding is unique!

And know his personal symptoms of a bleed: Tingling? Hot? Swollen? Your HTC staff can help you identify symptoms, so you can infuse more quickly at the first sign of a bleed.


Personalized healthcare really shines when it’s time to devise a treatment plan. You can use the new, subcutaneous novel therapy. Or you have two options for infusing factor: on-demand (also called episodic) or prophylaxis. On-demand means infusing at the first sign of a bleed. Prophy is the scheduled infusing of factor. It’s designed to keep factor levels in the bloodstream high enough—greater than 1%—to prevent most spontaneous bleeds.

Prophy is the recommended therapy for children with hemophilia in countries like the US with ready access to clotting factor.

And what about your schedule for prophy? This is about as personal as PHC gets! Your HTC team will offer a schedule based on your child’s needs: your family lifestyle, activity level, perhaps pharmacokinetics (PK) data to determine how quickly factor is cleared from the blood after an infusion, and any breakthrough bleeding that might happen.


Personalized healthcare is not the same as personalized medicine, a common term. Personalized medicine often refers to using a person’s genetic profile—genomes and specific genetic markers—to guide therapy for cancer and other diseases and disorders, including hemophilia. But PHC includes personalized medicine, and it’s worth discussing with your HTC team because more and more, the hemophilia community is focusing on personalized medicine.

National Hemophilia Foundation (NHF) recognized the importance of this genetic research and launched My Life, Our Future (MLOF). Through this program, you can get a blood test that enables you to learn more about the specific genetic mutation that caused your child’s hemophilia.

So PHC uses personalized medicine to examine your genetic makeup, help predict which medical disorders or diseases your child is most at risk for, and suggest which treatments will be safe and effective (or not) for him. This is particularly important for complications like the risk of getting inhibitors.

We’ve shown you examples of treatment and lifestyle areas to focus on, potential needs to address, and questions to ask your HTC team to design the best life possible with your bleeding disorder. Your HTC team will become one of your most important partners.

Dosage Denied! Where Does Insurance’s Power End?

Samantha* lives in Montana, our fourth-largest state but with one of the lowest populations, at 1.05 million. This means that families with hemophilia have an extra burden of traveling long distances to a hemophilia treatment center (HTC). Samantha’s three sons with severe hemophilia, ages 18, 13, and 11, were used to the 11-hour drive to Denver’s renowned HTC and a 7-hour drive to the closest hemophilia hematologist.

            What they were not used to was a sudden denial by their insurance company of their hematologist-approved factor dosage.

            On January 1, 2016, Samantha switched to a new insurance company, Assurance*, which was registered with the state exchange, or Marketplace. Six months later, Assurance made a curious request. The company asked Samantha to provide the weights of each of her three sons. This seemed odd, because Samantha always provided weights when she ordered factor, but she complied. Then her hematologist and specialty pharmacy received the same request from Assurance. The hematologist sent the weights, along with a three-month prescription. But Samantha was baffled.

            The reason for the company’s request soon became clear. On July 11, 2016, Assurance sent a letter to Samantha’s specialty pharmacy and hematologist explaining that the boys were using factor outside the recommendations of the product insert (PI). The PI is the set of instructions and medical information provided by the manufacturer inside each package of factor. The insurance company claimed that by using more factor per dose than was recommended, the family was acting outside of FDA regulations.

            Shortly after, Assurance sent Samantha a letter declaring it would only authorize the average dose according to the PI, contradicting the prescription of the boys’ hematologist, and cutting the hematologist-ordered doses by 65%.

            This letter shocked Samantha. “I reached out to Assurance via email to ask what was going on,” she recalls. “The company replied, said the hematologist had been notified, and I needed to contact the hematologist and have her reissue a script at a lower dosage. The insurance company kept me out of loop as much as possible.”

            But Samantha was smart: she made notes on every phone call and documented everything.

            Then Assurance sent a one-page letter about the dosage change to the hematologist and specialty pharmacy. Although Samantha requested a copy, she didn’t receive one; her specialty pharmacy finally forwarded her a copy.

            The letter stated that Assurance had an external review board investigate the case, but deceptively, the insurance company requested only a review of the PI’s average dosing, neglecting to send the bleed history of each boy and the recommendations of the hematologist! The letter did not verify who this review board was, or what outside consulting company was used and whether it had included hematologists knowledgeable about hemophilia.

            Samantha knew a fight was coming. Luckily, she was prepared. Her hematologist had recommended using an amount higher than the dose shown on the PI. Each boy’s dose was well documented. “We have 150 pages of documentation showing why each of our sons is on doses higher than the PI,” says Samantha. “Their half-lives, their recovery studies were all done consistently. So the scientific and medical facts were there.”

            The changes recommended by Assurance were shocking: Samantha’s youngest son had been prescribed 130 IU/kg of recombinant factor IX, three times a week. Assurance changed his dose to 80 IU/kg twice a week, a decrease of 67% per month. Another son was prescribed 150 IU/kg, three times a week. Assurance decreased this by 64.5%, down to 80 IU/kg, twice a week.

            Samantha notes, “My boys are very athletic and extremely toned; there’s no obesity. They are big boys. My youngest now weighs 184 lbs and is almost six feet tall. They needed the dosage prescribed by their hematologist.”

            Meanwhile, Samantha’s world became more stressful. “Raising three boys, fighting this battle, it was overwhelming!” Within one week of the lower-dosage change, all three boys had bleeds, flu-like symptoms, aching joints, and headaches. “My oldest son told me everything aches inside him. My middle son took a shower late one night. Afterward, it looked like a blood bath. He had horrific bloody noses. He was ghostly white. Finally we started taking [our sons] to the ER, even for nosebleeds, which we normally treat at home. We weren’t going to the ER for products (the ER doesn’t stock them), but for documentation—proof of their bleeds for the insurance company!” The ER doctor told Samantha that her sons needed factor now. “We told the doctor we couldn’t get the medicine in the correct dosage. Then he wanted to get involved in the fight!”

            Samantha’s youngest son had his first-ever joint bleed in his knee, which bled for a month. He was on crutches for six weeks and needed physical rehabilitation. “We started keeping the boys inside, like in a bubble, so they wouldn’t risk getting hurt. Yet, the boys had spontaneous bleeds.”

            Assurance tried many tactics. The company didn’t return Samantha’s phone calls. It denied and even delayed preauthorization from the hematologist, denied bleed doses, and once, incredibly, even refused to send factor.

            Despite the barrage of phone calls to Assurance from the specialty pharmacy, hematologist, and Samantha, the request for the prescribed dosage was denied. Together, Samantha and her healthcare team filed a grievance with the state against Assurance, following the online procedure. “Assurance became belligerent, to be honest,” Samantha remembers. “It was horrible. We said this change in dosage was life-threatening, and they said it wasn’t their problem. Our hematologist had never heard of any insurance company changing a physician’s script. She would have screaming phone calls with them. But they just would not budge. It was all about the money. They said, if we weren’t happy, we could go elsewhere. They wanted to get rid of us.”

            After filing the grievance, Samantha contacted her state insurance commissioner’s office. She filed an online appeal. It was tricky, because applications are limited to a certain number of characters. But Samantha was clever: she filed an appeal for each child separately, giving her more space to describe what was happening. Samantha also contacted Michelle Rice, vice president, public policy and stakeholder relations of National Hemophilia Foundation, for advice about including key words and points.

            A second external review was requested by the commissioner. When Samantha received the results of this review, she couldn’t believe it. “It was nuts! It agreed with the dosage change by Assurance, and this time we knew who the company was that conducted the review. What info did they base this on? What info was given to the external review company? What do they know about my boys? I started crying. My kids could die.”

            Samantha phoned the commissioner’s office, but the commissioner wasn’t there. In desperation, she called Donnie Ackers of Hemophilia Federation of America (HFA), who coached her on using the right words: “Call the commissioner’s office right away and get a manager. Don’t lose this!” With Donnie’s urging, Samantha called back: “I got ahold of Brenda*…she was literally a godsend. She said this was not acceptable. She took over our case. If not for her, we would still be fighting this. She said what they are doing is feeding you half-truth and lies.” Brenda asked for only one lawyer from Assurance to be her contact, and spent hours on the phone with the boys’ hematologist.

            By now it was August 2016, and Samantha and her boys had gone a month and a half with limited factor. “Brenda emailed me, and said she was going to force Assurance to send the prescribed amount of factor. She challenged them on the external review board’s finding. She called their bluff: Assurance wrote a letter saying it was not their fault; it was the external review board’s fault. Assurance had the audacity to claim the review board put their own numbers in the review, which coincidently matched the lower preauthorization!”

            To keep medical records up to date, Samantha recalls, “We eventually had a new pharmacokinetic [PK] test on all three boys, a 7-hour drive for us to our hematologist. At that time, my youngest had a knee bleed, and had to endure having all these labs drawn. I’ll never forget the drive to the hematologist; they all hurt so much that they slept a long way to overcome the pain.”

            The stress took its toll. Samantha says, “It was a tough time for all five of us. On top of this, we are working full-time jobs; our oldest had to quit his summer job for a while. We were so busy making phone calls, taking notes, caring for bleeds, and worrying about the long-term joint damage on the boys that we never stopped to breathe until Christmas!”

            The state insurance commissioner’s office made Assurance conduct a third review, with the hematologist’s prescription this time. In her incredible efforts to prepare, Brenda compiled records from birth to present for each child, including all scientific evidence (lab results, PK testing), a list of 20 items that an external review board needed, the PI, the original script, the changed script, and documented bleeds.

            “This third external review came not only in our favor, but even more in our favor,” says Samantha. “The board said these boys are not getting enough factor, and they changed the dosage to every 48 hours! And Assurance can never, ever change this, because it’s from the insurance commissioner’s office and follows the procedures outlined in the Americans with Disabilities Act.

            Samantha never dreamed that a manufacturer’s PI would be used as a tool in an insurance company’s attempt to lower costs by lowering dosage. When an insurance company comes between a hematologist’s prescription and a patient’s need, this is playing with life and death. Samantha’s story shows the value of documenting everything—every call, every email, every letter. She was smart to work with her healthcare team, and to never quit. When all seemed lost, Samantha called HFA, and from there, the commissioner’s office…one more time. And that was the straw that broke the back of Assurance’s ludicrous claim.

            Assurance hid behind the PI to start the battle. But the hemophilia community takes any challenge to children’s health head-on. To win.

 *Names have been changed due to pending legal action.

From: Publication: PEN’s Insurance Pulse 09.18

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