Patients With Plasminogen Deficiency Should Receive Care at Hemophilia Treatment Centers: Dr Amy Shapiro


Amy Shapiro, MD, medical director, Indiana Hemophilia and Thrombosis Center, discusses the clinical trials that influenced plasminogen's (Ryplazim; Kedrion Biopharma) FDA approval, as well as details valuable information for the treatment of patients with plasminogen deficiency.

Part 2 of this interview featured a discussion with Amy Shapiro, MD, medical director, Indiana Hemophilia and Thrombosis Center, on the recent FDA approval of plasminogen (Ryplazim; Kedrion Biopharma). Ryplazim is the first treatment indicated for plasminogen deficiency type 1, a rare disorder that impacts the blood clotting processes.

Here, Shapiro further discussed plasminogen’s impact by talking through the takeaways of the clinical trials that led up to its approval in this disorder, as well as provided helpful resources for locating hemophilia treatment centers (HTCs).

This transcript has been lightly edited for clarity and length.


Were there any surprising findings that emerged throughout the clinical trials that led up to Ryplazim’s FDA approval, and how should these inform patient care?

Yeah, I think the most surprising findings is how rapidly the product works. If you have a very small patient, a pediatric patient, with respiratory lesions, those first infusions need to be given in a very controlled setting. Because airway lesions in small children are smaller, if they slough off the surface, they can obstruct an airway and that can lead to significant difficulties breathing, if not cardiorespiratory arrest. So, in small children with respiratory lesions, the caveat is: treat under observation with ability to resuscitate if there are respiratory lesions in those patients initially, until you feel they're safe to treat at home.

Now, the other thing that's great about this product, because we as hemophilia treatment centers have experience with patients, teaching them to treat themselves at home—which is the goal of therapy, to make them have as normal a life as possible—we do the same with patients with plasminogen deficiency. We've taught them self-infusion or parents infusing children at home and it's been very successful, and again, allowed them to lead as normal a life as possible.

One thing I do want to say that's important for the care of these patients is they should be treated by a hemophilia treatment center. Hematologists who care for patients with hemophilia are very attuned to the pharmacokinetic profiles of coagulation factors, of monitoring this, and to the multi-system manifestations of disorders have coagulation. So, for example, in hemophilia, we understand that the musculoskeletal system is significantly affected. We understand that other systems of the body can be affected, and we're used to looking for symptoms in those other areas. So even if a patient presents initially with eye lesions, an ophthalmologist should refer the patient to hemophilia treatment center because we're used to taking very careful histories and doing detailed physical exams to make sure that other areas that could be affected are not being missed. And oftentimes what we find is that other systems are affected but have been diagnosed as more common disorders. So, for example, recurrent otitis media in a child really could be a symptom of plasminogen deficiency with lesions in the middle ear. What is felt to be reactive airway disease in a child could be respiratory symptoms due to plasminogen deficiency. Hoarse voice, often attributed to an undercurrent viral infection which could precipitated, but it could be due to plasminogen deficiency with development of lesions on the vocal cords or in the subglottic area. So, again, it is important that the physicians who take primary responsibility for these patients should be through a hemophilia treatment center. And they're easy to find. There's a website on the American Thrombosis and Hemostasis Network Athens website, and also on the CDC website so you can look up where an HTC is in your state.

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