Tung Wynn, MD, Assistant Professor, Department of Pediatrics and Division of Pediatrics Hematology/Oncology, UF Health Shand’s Children’s Hospital talks about an alternative treatment for hemophilia patients who resist the standard treatment option.
Interview Conducted by Ivanhoe Broadcast News in July 2017.
Tell me a little about Cayden and his condition and how you were able to start working with him?
Dr. Wynn: Cayden came to me when he was first born. He was born in Tallahassee and I was on call the night that he had his circumcision done. The circumcision lead to a whole lot of bleeding and at that point the physician was up at Tallahassee Memorial. Capital Regional Memorial gave us a call and said, hey, we need your help this boy is bleeding and it looks like he might have hemophilia.
Did you drive to Tallahassee?
Dr. Wynn: We did not, we actually got into an ambulance and transported him down here and we were able to evaluate him and determine his hemophilia, and then get his bleeding under control by giving him the appropriate factor for him.
How long have you been treating hemophilia patients?
Dr. Wynn: My whole 15 years in practice. I had spent 10 years in Tampa, I was doing hemophilia as a part of my practice and then I came up here to do primarily hemophilia for the last five years. .
For you is it something personal? Why did you pick hemophilia and oncology?
Dr. Wynn: For me it is about the patients, the families, and the relationships. I enjoy being able to help families with a disease that is very, very rare. There are very few physicians who have the expertise to handle it and are willing to treat it appropriately, so I have the opportunity to help the patients with the disorder. Educate the families that it is a generational disease; so it is a disease that will run in generations within the families, so we are helping that entire family across generations. I also have the opportunity to reach out to the communities through my position, providing education and knowledge about the disorder and I have the opportunity to provide education to other physicians; the residencies as well as current active physicians such as the ones in the emergency rooms, to provide them with education about hemophilia
So Cayden, let’s talk about the treatment that he has been getting?
Dr. Wynn: The normal treatment for hemophilia is to provide them with the factor that they are missing; in Cayden’s case he is missing factor eight. The missing factor is given to them and it brings the levels up to then what would be normal. With Cayden what has happened is that because his levels were so severe and his immune system is slightly different; his immune system has developed an antibody or what is called an inhibitor to block the factor that we give him. So it inactivates it and makes it ineffective as a treatment.
So it is actually his body that is fighting the treatment you were trying to give him?
Dr. Wynn: His body is completely rejecting it.
And it was not clotting properly?
Dr. Wynn: That is right, the levels are never normalized. His levels never increased with the replacements that we gave him.
So what is the remedy?
Dr. Wynn: The remedy is something called bypass therapy; in order to get him to clot we have to activate the clotting system in an alternative pathway or an alternative arm of the clotting system.
Is that common?
Dr. Wynn: It is very rare, there are probably just a few hundred patients that have it in the United States and our center has two or three of them.
Cayden is one of them?
Dr. Wynn: Cayden is one of them.
From another energetic little boy on the playground, what does that treatment mean for someone like Cayden? I mean, is it working? Would you never notice just by looking at him after treatment, you wouldn’t be able to tell?
Dr. Wynn: Right, yeah, and that is how we define our success. We want to see that. Our guys in the past, treatment for hemophilia used to entail protecting them. Keeping him away from other kids, keeping him away from playgrounds, keeping him away from sports and activities… That is not how we define success with hemophilia anymore. Success is really how can we get the kids to do all these things just like other children would? The fact that he is up and around; playing, running, climbing, and doing all those things that normal child would do is kind of what we look as successful treatment for him.
Why is hemophilia most common with males?
Dr. Wynn: Hemophilia is caused by a mutation that occurs on the X-chromosome and we mostly think about the X-chromosome as what defines a woman, but a woman actually has two X-chromosomes. When a woman inherits the one gene the other X-chromosome protects her. Whereas, a boy is defined by a having only one X-chromosome, so if the boy inherits the hemophilia gene on the X-chromosome they have no additional X-chromosome to protect them so they have the disease.
What is Cayden’s treatment moving forward, is this something that may become unmanageable as he get older?
Dr. Wynn: We actually have a lot of therapies that we are going to try to get for him. The immediate goal is try to get him on his immune suppressive therapy that would dampen his immune system and then work on trying even tolerance again for him once his immune system has been suppressed enough. Beyond that if he does not tolerate once again, there are actually therapies that are going to be available that will provide longer-term bypass therapy for him. That will continue to help him form clots that would be more longer-acting, that he could currently receive in a much safer fashion. A lot of those drugs are currently in the research and development pipeline, but part of the studies that we do here will help to bring that to the market within the next few years and will be available for Cayden.
So it will be something he will deal with probably for his lifetime?
Dr. Wynn: Or if we could get him to tolerate the treatment, then we will be able to return back to regular replacement factor like all the other hemophilia patients. If he does not tolerate treatment he has some additional therapies that will be available to him in the very near future.
Are there any concerns that you may know of?
Dr. Wynn: There are so few patients that there is not a whole lot of standardize therapy for these patients. I think there are that some concerns about the immune-suppressive therapy that we are going to try, there are going to be concerns that it is going to cause him to be more at risk for infection and that would be adding extra risk to his therapy.
Is this therapy that you are giving Cayden funded by the hospital, does his insurance pay for it?
Dr. Wynn: Yeah, it is standard. The drugs themselves are standard drugs; there is not a research component to what we are doing, so you know it is billed to his insurance like the others on therapy would be.
Will the hospital benefit in any way by the treatment you are giving Cayden?
Dr. Wynn: The only benefit the hospital would receive is the knowledge that Cayden is doing better and it is well controlled with this disease; there is no financial benefit that the hospital will receive.
END OF INTERVIEW
This information is intended for additional research purposes only. It is not to be used as a prescription or advice from Ivanhoe Broadcast News, Inc. or any medical professional interviewed. Ivanhoe Broadcast News, Inc. assumes no responsibility for the depth or accuracy of physician statements. Procedures or medicines apply to different people and medical factors; always consult your physician on medical matters.
If you would like more information, please contact:
Tung Wynn, MD
352-273-9120
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