Tag Archives: cystic fibrosis

We Can Be Heroes: Patricia George, M.D.

Posted on May 18, 2014 by Charlie

Patricia George, M.D., spoke with Community about her work in pulmonary transplant medicine, her HIV-PAH research, and her motivation as a member of PHA’s four-woman Team PHenomenal Hope in the nine-day 2014 Race Across America.

Going into medical school, I had passion for and experience in immunology research, so transplantation was something in which I was always interested. And like many who go into pulmonary medicine, I was initially drawn to it through my medical school and residency rotations in the medical intensive care unit. I enjoyed pulmonary physiology, and the work and pace of critical care medicine.

In addition, as a medical student, I met a patient with cystic fibrosis awaiting a lung transplant in the medical intensive care unit. I got to know her and her mom, and some of her life story, and wanted to be able to help people like her with lung disease. So that led me to pulmonary medicine and pulmonary transplant medicine.

My research involves looking at the mechanisms of HIV-associated pulmonary arterial hypertension (HIV-PAH). Pulmonary arterial hypertension is quite rare, however in patients with HIV, it affects at least 0.5 percent, and perhaps more, according to recent studies. That’s at least one in [every] 200 patients!

Advances in HIV care have changed the landscape for people with HIV, and many now label HIV a chronic disease. So medical complications, like HIV-PAH, become extremely important to study and hopefully help people live longer and live better.

Team PHenomenal Hope came together with people passionate about cycling and raising awareness. As an avid cyclist, it had long been a dream to someday race in the Race Across America (RAAM).

Stacie Truszkowski, one of my close friends in the cycling community, also shared this dream. So, in 2011 and 2012, Stacie and I reached out to our endurance cycling friends whom we thought might be crazy—er—passionate—enough to do this as well, and in 2012, with the addition of Anne-Marie Alderson, Ryanne Palermo, and Kate Bennett as our crew chief, our four-woman cycling team was born.

We organized this Pittsburgh-based team, met with our friends at PHA, as well as our earliest sponsors, and formed Team PHenomenal Hope. Later that summer, we added to this group Greta Daniels, assistant crew chief and alternate racer, and Sara Harper, alternate racer and crew.

Our mission is to dedicate our training and racing to those who live with pulmonary hypertension, to raise public awareness about the disease, and to raise funds to find a cure.

I started biking during pulmonary fellowship. I wanted to get back in shape, and a new women’s cycling team called Steel City Endurance was forming. I joined them in the inaugural year, and became totally enamored with biking and bike racing and met a lot of really neat people.

I enjoy being outside, escaping the stresses of my sometimes hectic lifestyle. As for endurance cycling, I enjoy pushing my body and mind to some sort of limit. It allows you to lose yourself in the present—how you’re feeling at that time.

We’re working with our team coach, who’s helping coordinate our training schedules so that they build and peak at the right time. Training is about consistency—getting the workouts in, getting stronger every day (except rest days). Eating healthy and getting enough sleep are crucial too.

As a team, we’re racing the whole race as a relay. To make the time cut-off and make it to the finish line as fast as we can, we divide up the ride into 20-to-30-minute segments.

On a four-person team, typically two riders will be out on the road, trading places in 20-to-30-minute pulls (one riding, the other in a support vehicle leapfrogging ahead for the exchange to happen). This pair of the four-woman team will ride for four to six hours, while the other pair rests, eats, sleeps, and recovers. It goes 24/7, from the time the gun goes off until we cross the finish line.

From what I hear, mental toughness will be one of the biggest challenges during RAAM. Those who have done it say that, at about day four or five, the sleep deprivation kicks in, and the reality of the Midwest flatlands also hits you. I know there is beauty in rolling plains, but at that point in the race, it may be tough to see it.

During RAAM, the crew is the essential group of people that will get us from Oceanside, California, to Annapolis, Maryland. The crew chief, Kate Bennett, is in charge of coordinating the drivers, navigators, medics, mechanics, nutrition, making sure we’re on course, and that people— including crew—are getting enough sleep, food, etc. A race with this relay between four racers, moving across the country with an RV, two support vehicles, and 13 crewmembers is quite an undertaking.

The greatest source of inspiration is the PH community. When I think about how hard it may be to be on the bike, mentally or physically, I think about what my patients go through on a daily basis.

Team PHenomenal Hope members Ryanne Palermo, Patty George, Stacie Truszkowski, Greta Daniels and Kate Bennett.

I get to choose to ride my bike, to push myself through discomfort. My patients don’t have such a choice. They wake up and live with pulmonary hypertension every day, and face whatever that day may bring, and many do so with such grace. So when I’m feeling less than motivated, I often think of people I know living with PH, and it motivates me to get this job done.

Likewise, in my practice, I am regularly reminded of the need for a cure. I often evaluate patients with pulmonary hypertension in need of a lung transplant. For this group of patients, they often no longer are responding to medications. It is a reminder that, while we have come so far, and many patients do respond to medical therapies, we still need a cure.

In my job, I also conduct PH research, and know firsthand how important funding is to exploring the frontiers in science. It makes it all the more important to me that Team PHenomenal Hope is raising money for PHA to fund grants and help other scientists have funds needed to find a cure.

We have something truly special with our partnership with the Pulmonary Hypertension Association. PHA launched a Race of Our Lives campaign, and we have been amazed how people in the community have organized their own Unity events, walking, riding their bikes, doing whatever they can to raise awareness about PH and join us in raising funds to find a cure.

Team PHenomenal Hope is bigger than four of us on bikes, or the 17 of us crossing the country. This is actually a huge team that spans coast-to-coast.

Pulmonary hypertension is a rare disease that can affect anyone, from children to adults, men and women, and people of all races and ethnic backgrounds. Initially, it is often misdiagnosed as another pulmonary condition, taking on average over a year to make the correct diagnosis and get the proper treatment.

Although it is a rare disease, it is important for doctors to at least think about pulmonary hypertension in their differential diagnosis when faced with a patient with shortness of breath, because without considering it, the diagnosis won’t be made.

Fortunately there are many medical treatments on the market, changing the prognosis for many who have this disease; however there still are people who do not respond to therapy, and to date there is no cure. Team PHenomenal Hope is working with PHA to do something to try to change that.

Team members Palermo, George, Truszkowski, and Anne-Marie Alderson at University of Pittsburgh Medical Center.

Posted in About Us, Diseases, Featured | Tagged anne marie alderson, bicycle, bicycling, bike, biking, chronic disease, cycling, cystic fibrosis, greta daniels, hiv-pah, hivpah, kate bennett, lung disease, pah, patricia george, patty george, PH, PHA, phenomenal hope, Pulmonary, pulmonary arterial hypertension, Pulmonary Hypertension, Pulmonary Hypertension Association, pulmonary medicine, pulmonary physiology, raam race, race across america, race of our lives, ryanne palermo, sara harper, stacie truszkowski, steel city endurance, team phenomenal hope, transplant, transplantation | Leave a comment

Newsmaker Q&A with Thomas L. Spray, M.D.

Posted on November 1, 2013 by Charlie

Thomas L. Spray, M.D., is chief of the Division of Cardiothoracic Surgery at The Children’s Hospital of Philadelphia, director of the hospital’s Thoracic Organ Transplantation Program, and Professor of Surgery at the University of Pennsylvania School of Medicine. Community recently interviewed Dr. Spray about pediatric lung transplantation.

What are the most important considerations for parents when making decisions about pediatric lung transplants?

The fundamental issue here is that, lung transplantation is, unlike heart transplantation, not associated with as good long-term survival. In some ways it’s somewhat palliative. Five-year survival for lung transplant is about 50 percent. And despite lung transplants being around now for about 25 years, there hasn’t been a lot of improvement in survival.

The lung is an immunocompetent organ, unlike most other organ transplants. It has the disadvantages of being connected to the outside, so it’s always at risk of infection. Also, there are lymph nodes in the lungs, which probably make them more susceptible to rejection and latent chronic rejection, which is where most lung transplants eventually fail; something called bronchiolitis obliterans—scarring of the small airways of the lungs—which is thought to largely be a chronic rejection problem.

Having said that, there are isolated patients who do extremely well and do well over the long term, but if you just take the averages, the average survival is about five years. So that’s the disadvantage.

On the other hand, for many patients, especially pediatric patients, their survival is going to be measured in a matter of a year or less if they don’t get a lung transplant. So it’s successful, but it’s not perfect by any means. It’s generally very much palliative for children as well.

To what degree should the child be involved in the decision?

To what degree the child should be involved in the decision is somewhat age-dependent. When you have a lung transplant, you’re basically exchanging one disease for another one, because you then have to be on medicines to prevent rejection, and you have a certain amount of medical surveillance necessary.

Unfortunately lung transplantation is still a procedure that has a significant risk associated with it. The lungs cannot work sometimes. The outcome cannot be positive if you get a viral infection of the transplant early on—it can destroy the lungs. And that’s very hard for anyone to survive.

I can remember some patients though, with cystic fibrosis, after lung transplant, even when the lungs went bad, and they ultimately died, having said during the time they were alive, that it was worth everything just to breathe normally for a change. So you have to put it in perspective.

When you get into the teenage years, I think it’s appropriate for the child to be involved in the decision. I think it’s especially important for teenagers, who unfortunately have a fairly high risk of not being compliant with medical management, and then they reject their organs and die.

Just being an adolescent is a risk factor for transplant, it appears. It’s a difficult time. That’s why when you’re talking teenagers, it’s very important that they be involved in the decision process, so that they recognize that they have to be involved in the medical management. They have to take their medicines faithfully. They have to be seen frequently. And if they aren’t willing to do that, then it’s kind of foolish to go down that pathway.

What have been some of the advances in pediatric lung transplantation?

Patient room at The Children’s Hospital of Philadelphia

Most of the advances in transplantation, in terms of survival, over the last 15 years, have been at the early period after transplant. In other words, we’ve been able to transplant more complex patients, who’ve had multiple previous operations, who were sicker waiting for transplant.

We’ve evolved to the point where it’s now possible to support patients on an assist device called ECMO while waiting for transplant and still have them able to be up and around and maintain their ability to breathe, so that it makes it easier after the transplant. So there are some advances, but there haven’t been any major advances in immunosuppression.

We have experience now transplanting newborns all the way up to adults. We’ve had experience with many kinds of complex congenital heart disease and lung transplants. We have experience in pulmonary hypertension. Those are the more common causes in children.

I think it’s important to note that in the pediatric world, the most common indications for transplant are considered the higher risk indications in the adult world. For example, in adult lung transplant, the most common indication is emphysema. Emphysema is a more straightforward condition to treat with lung transplant than any of the diseases that we see in children, and emphysema is virtually unheard of in children, so pediatric transplant by its very nature is a higher risk population.

What percentage of pediatric transplant patients have pulmonary hypertension?

At The Children’s Hospital of Philadelphia, at least 50 percent or more have congenital heart disease or pulmonary hypertension, and relatively fewer have cystic fibrosis. Part of that is because the management of cystic fibrosis has improved over the years such that there are relativity fewer children who require transplantation for cystic fibrosis than in the past. So, most people with cystic fibrosis can get to adulthood before they have enough lung deterioration to require transplant, and therefore get transplanted in adult centers now.

When we first started doing lung transplant in about 1990, when I was in St. Louis, cystic fibrosis was a common indication, because children under 18 with severe cystic fibrosis would come to St. Louis for transplant. In Philadelphia, we see less of that and more pulmonary hypertension and congenital heart disease.

What drew you to the lung transplantation field?

I started out as a congenital heart surgeon. Congenital heart surgeons all have to be adult heart surgeons first. I did adult and congenital heart surgery at St. Louis Children’s Hospital. I was recruited to come here to take over from William Norwood, a very well known surgeon, who went to Europe to start a new program.

When I came to Philadelphia, there wasn’t a lung transplant program in this area. So I started a pediatric lung transplant program when I came in 1994, having started the program in St. Louis in 1990.

I think my interest in lung transplant came from patients I saw in St. Louis who had no real, good option for repair of their heart, because they didn’t have good lungs to push blood into. There are certain types of congenital heart disease where that’s the case, where you could repair the heart defect if you had pulmonary arteries to pump blood into.

But children who don’t have that became progressively more cyanotic. I saw some of these children and I thought, ‘If we could just put new lungs in, we could fix the heart.’ So that’s what got me interested initially, and then that expanded to all the other potential reasons for lung transplant, of which there are many.

What are some of the differences in quality of life after a successful pediatric lung transplant?

Art therapy at The Childrens Hospital of Philadelphia

Quality of life depends on the patient’s condition before the transplant, but the majority in the pediatric population are extremely debilitated. They often have cystic fibrosis. They’re chronically infected. They have poor lung function. They have very little exercise tolerance. Patients with PH may have heart failure also. So the quality of life prior to transplant is very poor.

The waiting times are so long for lung transplant that most children deteriorate significantly while waiting in the hospital. They’re sometimes waiting in the hospital for a year or more.

So the quality of life after transplant, while vastly improved, takes a while for them to recuperate. I think what people often don’t realize is that if you’re sick for months and months prior to lung transplantation, there’s a lot of rehabilitation necessary. Even after a successful lung transplant, it’s not like patients are going home in a week. Many of them have to stay in the hospital for months while they’re literally recuperating and rehabilitating themselves from being chronically ill for the previous several years.

How much interaction do your patients have with the Child Life, Education and Creative Arts Therapy department at The Children’s Hospital of Philadelphia?

They have a great deal of interaction, especially while waiting. Children who are sick and in the hospital and literally waiting for sometimes months and months for organs to become available need that kind of interaction. They need to have play.

They need to have creative interactions, and that’s why the Child Life Department is so important for all the patients in the hospital, but even more important for those who are chronically in the hospital for long periods of time. They need to have that kind of stimulation. They need to be able to be involved in arts and crafts to keep them occupied, literally, to help them develop while they’re waiting.

What are some ways that families can cope with being on a waiting list?

Everyone, I think, does that differently. I think it’s always difficult for families to recognize that a waiting list is exactly that. You have no idea when organs might become available.

There are times when organs are available, and we tentatively accept them, and then they deteriorate to the point that we can’t use them. So the families have situations where their hopes are raised that they’ll have a transplant, and then it falls through, and then they’re back waiting again. So it’s a difficult thing for the families.

I think the families need to recognize that donor organs are hard to come by; they’re very scarce. The organ system is as fair and open as people can make it. It’s constantly trying to be as completely open and straightforward as possible and to not disadvantage anybody and make everybody on an equal playing field. But these are difficult things sometimes.

Transplantation in some ways is kind of a fundamentally flawed strategy, because you have to have a tragedy to have a miracle. Someone has to die for organs to be available, and as a physician and surgeon, I don’t want anyone to die. In a way, I don’t want there to be more donors.

On the other hand, I think the donors who are available should be used as maximally as possible, because there are so many children who need the organs. It’s a constant battle to try to use everyorgan you possibly can. But then some of them have problems and don’t work. That’s unfortunately the chance you take.

What programs are in place at The Children’s Hospital of Philadelphia to help families afford the cost of pediatric lung transplants?

Art Therapy at The Children’s Hospital of Philadelphia

There’s a whole process before someone is listed for transplantation that involves evaluating their financial situation. The hospital has many programs to try to get them into programs that will provide coverage. Because it makes little sense to do a lung transplant if you have no coverage for medications after the transplant, which happens in some crazy insurance arrangements.

The hospital sometimes will help families get Medicaid or some other government program that will at least provide them with follow-up medications and follow-up care. There’s a whole financial counseling group that works with families in relation to transplant, because you have to recognize it’s not a one-time deal. It’s like a new disease, if you will, transplantation.

What else is important to stress about how pediatric lung transplants are different from adult transplants?

The diseases are different, and they’re more complex. Many of the [pediatric] patients have had previous surgeries, which complicate the transplant significantly, in terms of bleeding and other issues.

I’ve said many times that the hardest part of transplantation is not putting the new organs in; it’s getting the old ones out. That can be extremely difficult due to the scarring and inflammation and previous infections. It can sometimes be extremely difficult just to get the old organs out without damaging other important structures. They can all be stuck together in the chest.

That’s one major challenge with lung transplant, especially in children. It’s not as common in adults. Most of the adults with emphysema have not had significant previous surgeries; they don’t have a lot of extra blood vessels.

Many of the children who need transplant are also ‘blue.’ They’re cyanotic. They don’t have normal oxygen levels. That stimulates development of blood vessels in the chest that can be very difficult to control at the time of transplant, and bleeding is much more of an issue.

Then of course we’re dealing with different sizes. In adults, they’re using mostly adult lungs and some teenage lungs. But in pediatric transplantation, we have to list patients in a very discrete size range, because we do transplants in patients all the way from newborns up to adult-size teenagers. So we have to have the ability to put very small lungs in small children.

We do occasionally use lobes or parts of lungs from adults in small children, which is something the adult world rarely, if ever does, because we have to deal with this wide size range.