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On Monday, September 22, several members of the bleeding disorders community came out to share their experiences with bleeding disorders treatment with the FDA.

HFA worked with partners like NORD, PPTA, and NHF to ensure that our community was effectively represented. Patients and advocates of every age, every background, and with several different conditions came out to share with the FDA their hopes, fears, highlights, and trials in treating their conditions. So many great opinions were shared, covering two open-ended topics set by the FDA.

Below is the testimony of Sonji, from Colorado. Not only does Sonji herself have hemophilia, but her son, Thomas, has hemophilia with an inhibitor. She explained what it was like to treat Thomas’ inhibitor, as well as her hopes and fears for the future. We hope that after reading reading Sonji’s compelling testimony, you will be encouraged to share your own treatment experiences with the FDA.

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My name is Sonji and I am from Englewood, Colorado. I have mild hemophilia. But today I will tell you about my 11-year-old son, Thomas. Thomas has severe Hemophilia A. He has been fighting an inhibitor since he was seven months old. He also has asthma and an acquired immune deficiency and needs IVIG treatments every four weeks. While his bleeding episodes have been numerous and frightening, the most challenging impacts for him and my entire family are situations that influence his quality of life beyond just his clinical conditions.

Venous access is our number one concern. Because he infuses factor concentrate daily for his immune tolerance, good venous access is paramount. When he bleeds and needs multiple infusions per day, the need for access is even more critical. As an infant, he had a Broviac catheter. Eventually, the Broviac succumbed to the wear and tear of repeated use. We made the decision to implant a port-a-cath. Little did we know that it would be the first of five ports to date, along with multiple PICC lines. Thomas’s veins are hard to stick on a regular basis and he has repeated history of compartment syndromes after peripheral infusions by medical professionals. It is uncertain if he will require another port, or even if his body will hold up to another insertion, not to mention the bleeding and infection risks involved with the surgical placement. It is a terrifying to have the very medication that can save his life in your hands, but not know if you’ll be able to administer it because of access issues.

Chronic and acute pain management also has been a significant challenge for Thomas. Repeated joint bleeds into his left ankle have miraculously caused little visible damage, but he consistently complains of soreness there and in other areas of his body. He recently said to me, “Mommy, my body is like that of an old man’s. I’m just sore all the time.” He does not tolerate most opioids or narcotics and the side effects often make him harder to manage medically. In 2013, we tried a combination of a transdermal opioid patch and an anti-nausea patch during a particularly nasty shoulder bleed. Despite my insistence for weaning, Thomas continued on these patches for three months – two months past the worst of the acute pain. He became dependent on the patches. The suffering my then 10-year-old son had to go through during the withdrawals from such powerful, yet mildly effective drugs was something I’d never wish on my worst enemy.

Like most boys, Thomas has a competitive spirit. Because of his physical limitations, most sports are off-limits. He started swim lessons at an early age and in the summer of 2011 he swam with a neighborhood swim team. He excelled and even qualified for the All-Stars meet. We continued with a year-round team, but Thomas spent much of 2011-2013 sidelined as he battled port infections and significant bleeds in his iliopsoas and quad muscles. Last summer he returned to the pool, but he found he didn’t have the stamina or speed that he once had. Despite sadness about not being able to do what he loves on the level that he’d like to participate at, he continued to swim to have fun with his peers.

Thomas misses an average of twenty days per school year. During the past year, he missed a trimester due to pain related to a shoulder bleed and the resulting dependency on pain medications. Our school district worked with us to provide a homebound teacher, but she was only with him for two hours a day and he fell behind. Even after he had recovered enough to attend school, he had to remain home one day a week for us to retain eligibility for the homebound teacher. Through sheer determination Thomas was able to move on to the sixth grade this year. Thomas’s father and I bought at our own expense a robot that travels from class to class, enabling Thomas to replicate himself from a distant location so that he interacts with his teachers and peers as if he was physically present.

Hemophilia hasn’t gotten easier over time – bleeds, pain, and line infections still happen despite all the preventative efforts, and as a family we’ve just become more skilled in coping. We’ve made multiple attempts at immune suppression therapy to eradicate his inhibitor and cannot sustain a zero Bethesda Unit.

From the time Thomas was born, we’ve tried to prevent bleeding to minimize joint damage. I didn’t want Thomas reliant on a wheelchair. Limited mobility is less of a worry now, as I see Thomas adapt when he needed. But the root cause of that limited mobility – whether or not his factor concentrate will address a bleed and minimize damage – is what worries me most.

Bleeding management of an inhibitor patient is staggering – currently we have only two products to choose from to control bleeding, and only one product approved for prophylaxis. Every time a bleed happens, I hold my breath to see if Thomas’s body is going to respond to treatment – and I never know if it’s going to take one treatment or a week of multiple infusions daily to stop a bleed. It’s scary. I worry that my child might literally lose life or limb and I only have a limited range of medical interventions to help him.

There’s worry over the affordability of the drugs available to him. There’s worry over access to his medical team and if my insurance will allow me to utilize their expertise. There’s worry over his mental and emotional condition. There’s enough worry to fill an ocean – but I’ll meet that worry with advocacy and empowerment so my child can live his best life despite any limitations.

Thank you for the opportunity to share our experiences.

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