Ongoing Research in Plasminogen Deficiency Type 1: Dr Amy Shapiro


Amy Shapiro, MD, medical director, Indiana Hemophilia and Thrombosis Center, discusses an ongoing trial and novel development in plasminogen deficiency type 1.

In part 3 of this interview, Amy Shapiro, MD, medical director, Indiana Hemophilia and Thrombosis Center, discussed the importance of receiving care at a hemophilia center and the most surprising findings from their trials on plasminogen (Ryplazim, Kedrion Biopharma), the first FDA-approved treatment for plasminogen deficiency.

In the final part of her interview, Shapiro explores more ongoing research in plasminogen deficiency type 1 and the development of a novel web-based system that was designed to guide treatment approaches in affected patients.

This transcript has been lightly edited for clarity and length.


To your knowledge, are there any plans for additional trials with Ryplazim?

There is a trial that our center is doing that I would like people to know about. It's called the HISTORY trial (NCT03797495). It's listed on, is not sponsored by the pharmaceutical agent, and it doesn't necessarily include Ryplazim, but it is looking at the natural history of individuals with plasminogen deficiency.

One of the goals of the study is to try to develop what we call “severity categories.” Because this disease is so rare, we don't have the ability at this point in time when a patient comes to us to say, “You have severe disease, you need lifelong prophylaxis with this agent or treatment on a regular basis to help prevent and suppress any manifestations of the disease,” or “Your disorder is more mild or moderate. You need intermittent treatment when symptoms occur or before an intervention."

So at this point in time, the level of the plasminogen activity and the genetic defect don't predict the severity category for this disorder. And so what we're trying to do in this study is to help develop those. And it's an international study, we've got near 100 individuals and we also enroll all siblings and parents—what we call first degree relatives—because we're trying to sort out what other genetic contributions, other background genetic data may impact the manifestations of this disorder. So, I would encourage anyone who has someone identified with plasminogen deficiency to contact me. You can see on the website how to get ahold of us to possibly enroll in this study.

We also have worked with a group in McMaster [University], Canada to develop a specific module for the use of Ryplazim. And that system is called WAPPS-Hemo. It's a web-based system that is a Bayesian model for pharmacokinetics in populations. You can, with very limited testing of an individual, develop one or two data points—knowing when they were infused or treated—and put this into the model and develop a curve of what the pharmacokinetics look like in that individual patient. You can target a trough—or a lowest level—before next infusion with the system and help treat your patient and model your therapy over time.

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