For 35-year-old Christian Alfredo De La Peña Alvarez, living with hemophilia A has meant a lifetime of planning around treatment, managing injuries, and navigating the challenges that come with a chronic condition. After decades of factor VIII infusions, Christian recently pursued gene therapy at the Center for Inherited Blood Disorders (CIBD), opening a new chapter in his journey.
A husband, father of three, and small business owner, Christian enjoys spending time outdoors with his family, traveling, visiting the beach, and volunteering in his community. Staying active and being present for the people he loves have always been important to him, but living with hemophilia has sometimes meant having to think twice about everyday activities.
A Lifetime of Managing Hemophilia
Christian was diagnosed with hemophilia A at birth. Because the condition runs on his mother’s side of the family, his family knew to have him tested right away.
Growing up, he experienced frequent bruising, ankle injuries, and joint pain. He was also advised to avoid certain activities, including football and full-contact martial arts. Even as a child, he had to be mindful of the possibility of getting hurt and how an injury could affect his daily life.
His treatment journey began with factor VIII infusions, which his mother administered until he was around eight years old. He eventually learned to infuse himself, a routine that became part of his everyday life.
One of the most difficult parts of treatment was finding a vein. “Sometimes it took multiple needle sticks, which was painful and frustrating, especially as a child,” says Christian. As he got older, he also faced challenges maintaining access to treatment.
As a small business owner, Christian experienced periods when he struggled to qualify for health insurance or maintain coverage. At times, he had difficulty obtaining the factor he needed, resulting in painful bleeding and added stress.
“Trying to run my business while figuring out how to obtain essential treatment was very stressful,” Christian shared. “The mental burden was a significant part of living with hemophilia.”
Discovering Gene Therapy as an Option
Christian first heard about gene therapy for hemophilia about four years ago. At the time, he didn’t think it was a treatment he could have because of the cost.
That started to change when his comprehensive care team at CIBD spoke with him about a potential path toward qualifying for treatment. After learning more about gene therapy and what the process would involve, Christian expressed interest in moving forward.
Christian worked with Dr. Gomez and the CIBD team to determine whether he was a candidate for gene therapy. The evaluation included discussions about the treatment, antibody testing, and additional lab work and imaging to assess his overall health and eligibility.
Once his results confirmed he was medically eligible, the CIBD team helped navigate the insurance authorization process, coordinating with his insurance company and providing the necessary clinical documentation to help make treatment possible.
Christian has received care at CIBD since he was very young, and having a team familiar with his medical history made a difference as he explored this new option.
“The possibility of needing fewer infusions felt like a dream I never thought I would experience,” he said.
His previous struggles with insurance and access to factor also influenced his decision. He hoped gene therapy could reduce his dependence on frequent infusions and ease some of the stress of managing treatment access.
He understood that gene therapy was not a guarantee of permanent results, and that continued monitoring would be important. Still, the possibility of a reduced treatment burden and greater peace of mind made it an opportunity he wanted to pursue.
Taking the Next Step with CIBD
Earlier this year, Christian received Roctavian gene therapy for hemophilia A at CIBD.
For him, the experience represented much more than a treatment appointment. It was the culmination of years of managing hemophilia, navigating treatment challenges, and working toward an option he once thought was out of reach.
One moment from the experience stands out in particular: the time immediately after his infusion was completed.
“Dr. Gomez and the CIBD team came in, talked with me, and congratulated me. They made me feel safe and supported, and they helped me feel that I had accomplished something huge,” Christian recalled. “Their kindness made that moment very special, and it is something I will always remember.”
While everyone’s response to gene therapy is different, Christian is hopeful about what the future may hold. He looks forward to continuing to be active, spending time with his family, and participating in his community with less of the day-to-day worry surrounding treatment.
Helping Others Learn About Gene Therapy
Sharing his experience is an opportunity for Christian to help others understand both hemophilia and the treatment options that may be available.
He recognizes that many people don’t fully understand what hemophilia is or how significantly it can affect someone’s daily life, particularly when access to necessary treatment becomes a challenge.
He also hopes that greater awareness of gene therapy will encourage more conversations about access and financial assistance for eligible patients.
“Cost should not be what prevents someone from exploring an appropriate treatment with their doctor. Sharing my experience is one way I can help others feel less alone and encourage conversations about access to care,” he shared.
Christian knows that considering gene therapy can bring questions and uncertainty. He experienced some of those same feelings before making his decision. His advice to other patients and families is to take the time to learn about the option, talk with their care team, and ask questions about the potential benefits, risks, and whether it may be appropriate for them.
One of the most meaningful changes for Christian has been the reduced day-to-day mental burden of managing treatment, including carrying factor, keeping it cool, and planning travel around treatment.
He hopes that sharing his story helps someone else feel less alone, less afraid, and more comfortable starting a conversation about gene therapy.
We’re grateful to Christian for allowing us to be part of his journey and for sharing his experience to help others learn about the possibilities of gene therapy for hemophilia.
Interested in Learning More About Gene Therapy for Hemophilia?
Gene therapy may be an option for some adults living with hemophilia. Our team works with patients to help them understand available treatment options, determine eligibility, and navigate the gene therapy process.
Learn more about our Gene Therapy Program or contact our team to start a conversation.