When your child is diagnosed with pulmonary hypertension, there are so many emotions, questions and unknowns. As a parent, you adopt a “whatever it takes” mentality and will drive anywhere to get them the care they need. When my son Cash was diagnosed with PH, there weren’t any pediatric PH care centers near Indianapolis.

I found a list of PHA-accredited PH Care Centers on the PHA website, which led us to Cincinnati Children’s Hospital Medical Center. Cash still goes there for treatment. The hospital has a robust research program, which reaffirmed our decision to go there for our son’s care.

In 2015, pediatric cardiologist Russel Hirsch approached us about participating in a pediatric clinical trial studying Uptravi (selexipag).

We felt confident in Cash participating in the study because we had a clear understanding of what was expected of him and the potential side effects. We had the mindset that Cash’s participation was also doing something for the greater PH community. Cash comes from a place of privilege to have a choice in where he goes for treatment, and since he met the qualifications for the trial, it just felt like the right thing to do.

Participation in the trial was straightforward and included various doses of medication, along with routine blood work. Although Cash’s pulmonary pressure didn’t drastically improve, it did improve his quality of life.

Hope for PH families

For parents, there’s a lot of hope surrounding these medications and trials. I want to share Cash’s positive experience with others, while being mindful that there are kids who aren’t doing well and are on waiting lists to participate.

I’ve connected with many parents of children with PH on social media, including parents of the kids participating in the same trials as Cash. It’s an active online community where parents can ask questions and get advice from others in similar situations.

During my conversations with other parents, I never want to pressure them to choose a specific doctor or care facility. Each child is different, so what works for Cash might not work for someone else. This is why access to treatment, medication and research is so important. Positive results and hope on the horizon are a complete game-changer for families in this community.

Navigating trial logistics

In February 2023, Cash began participating in a clinical trial to examine the effects of sotatercept (Winrevair) in kids with PH. We were given all the information we needed to make an informed decision, including a list of potential side effects based on a similar clinical trial for adults.

At the time, Cash’s medication regimen wasn’t working and his condition was progressing much quicker than expected. We hoped his participation in the study would improve his condition. Cash hoped his condition would improve so he could reduce his oxygen flow rate or come off supplemental oxygen altogether.

Every three weeks we drive an hour and 45 minutes from Indianapolis to Cincinnati for blood work and the next dose of medication. In addition to office visits, the trial requires home health visits, which initially came with a lot of logistical challenges for us.

There weren’t any pediatric home health nurses near us. The closest we could find was a nurse who lived west of Chicago, which is about a five-hour drive depending on traffic. That was problematic because the medicine must be transported in a temperature-controlled cooler and administered within a specific window of time.

In addition, some courier drivers opened the medication box, which compromised the temperature and security of the medicine. There were also instances where the medication was delivered at the wrong time.

Eventually, we found a nurse in Indiana who would drive about an hour to our house. However, if a home health nurse cancels, we have to drop everything and drive to Cincinnati so Cash can still receive the injection.

We shared our concerns and frustrations with Dr. Hirsch and his PH team. We told them that if these issues continued, it would jeopardize our participation in the trial. Dr. Hirsch immediately contacted Merck representatives to get the issues resolved.

As a result, we secured a professional and consistent home health nurse in Indianapolis. There was also a significant improvement in the shipping and delivery of the medication. The shipping containers now come sealed with tape and “Do not open” stickers all over the box.

It was a frustrating process, but I think our experience has helped work out issues that other families won’t have to experience. Now, the home health process is more consistent, with a greater sense of urgency and attention to detail.

Advice for other families

When this clinical trial began, we didn’t realize the impact of frequent travel on our family. Cash has been participating for over three years, and there have been times he’s said he didn’t want to participate anymore.

To a child, living with PH is not just taking medication. It’s waking up in the middle of the night to eat a snack and take your medication. It’s being put on oxygen or experiencing side effects from medications while going to school.

Throughout Cash’s clinical trial experiences, we shared concerns with his care team, and they never pressured us to keep participating. It was always our family’s decision, and we really appreciated that. Having the right care facility can really make or break your experience.

My biggest piece of advice is to have a full understanding of the trial requirements and obligations. Make sure you know what participation will look like before making a decision. I also encourage families to visit PHA’s website to find a PHA-accredited PH Care Center with a strong research program. Finding PHA’s website changed everything for our family. It led us to Cincinnati for Cash’s care and allowed our son to live his life instead of being bedridden.