Why The PFIC Network Patient Registry Matters
A message from our Executive Director, Emily Ventura
When Cedar was 15 months old, her dad and I were faced with an important decision.
Cedar’s itch was unfathomable. She never slept, and when she did, she woke up crying in bloody sheets, her ears and face mangled from the scratches. There were no medications available for her itch at the time, and her doctors had tried every combination of things they could think of. Was it time for a liver transplant? No, her liver was still doing “ok.”
We held onto hope for three reasons:
1. We wanted her to grow up as much as possible before putting her through transplant, as we felt that would award her the best possible outcome.
2. With a liver that was “ok”, putting her on the transplant list may not be a quick path.
3. We hoped that if we held on and continued trying different interventions, that one would work and she may not need a transplant at all.
This was 2012. The IBAT inhibitors were in the early phases of clinical trials, and we did not meet the criteria to enroll. The only option left was diversion surgery. But which one, internal or external? A list of considerations for each was presented to us, with no way of telling which one was the better option.
Ultimately, we were faced with a choice. We chose for her to have a partial external biliary diversion. I believe that choice afforded Cedar time before her disease progressed toward transplant, but unfortunately, it did not help reduce her itch.
If we would have known then what doctors know now, we would have known that Cedar would not have responded to diversion surgery…and we would have made different choices.
But I chose not to let the thought of “what would have happened if she didn’t have surgery” burden me. There’s no point – we made the best possible decision based on the information available to us. What I still do think about is the impact that today’s knowledge and understanding of the disease would have had on that decision – and the impact that knowledge we don’t yet have could exercise on decisions being made by families today and in the near future.
I envision a world where every patient diagnosed with PFIC can make informed decisions that are based on knowledge gained through decades of understanding each unique disease that falls under the PFIC umbrella. We as patients, parents and caregivers have an opportunity to support and accelerate that gain in knowledge. We can help get there faster.
The PFIC Network chose to professionalize and internationalize our patient registry so that we establish a patient reported database that will aim to accelerate these outcomes. The registry has been built by a statistician under the direction of patients and parents, and with clinician guidance. The registry is a powerful tool, but it is only as strong as we make it.
Not only do we have the opportunity as parents and patients to contribute to such a tool, we have a duty to do so. Enrolling in the registry takes less than 30 minutes. Keeping up with your follow up surveys takes less than 10 minutes every 6 months. Even if you have not experienced any changes, that is important to know! Have you been on a healthy trajectory, not experiencing symptoms or progression? Phenomenal, fill out your surveys to document those wins!
If you have any questions about the patient registry, whether it be about data privacy, research opportunities, or information you’d like to gain from your registry data, please reach out to the PFIC Network team. We are here to answer any questions you have to help you feel good about your decision to enroll in and keep up with your follow-up surveys.
We are in this together. The registry is unique to us, it’s a powerful tool, but its impact solely hinges on the full participation of our community.
Please enroll and fill out your follow up surveys every 6 months, so we can all follow along with knowledge and progress gained from your decision to do so!
With hope and deep gratitude,
Emily Ventura
