Apr 18, 2023
Dr. Steven Abman is Professor of Pediatrics-Pulmonary Medicine and Director of the Pediatric Heart Lung Center (PHLC) at the University of Colorado Denver Anschutz School of Medicine and Children’s Hospital Colorado.
In this episode, Dr. Abman gives an overview of The Pediatric Pulmonary Hypertension Network (PPHNet), a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease. He also discusses takeaways from the 15th International Neonatal & Childhood Pulmonary Vascular Disease Conference.
My name is Steven
Abman and I'm a pediatrician trained in Pulmonary and Critical Care
Medicine from the University of Colorado. I've been working locally
with our team here in Colorado at the Children's Hospital to really
develop our own pulmonary hypertension program.
What we recognized very early on, of course, is that it's a disease
that knows no boundaries, that occurs in various settings and
affects neonates or newborns, infants, children, older children,
and then can continue into adulthood. We also realized like most,
that this is a disease without knowing boundaries. That means it
crosses over into different specialties. So we include members of
our cardiology team, our pulmonary team, our critical care team. It
takes a village of really talented advanced practice nurses and
therapists of all sorts. So in other words, it's an
interdisciplinary program that manages children of all ages with
various forms of pulmonary hypertension.
The program links inpatient with outpatient care. But what we
realized quite a while ago is that there aren't many places that do
this kind of thing. Also, we had very limited data, very limited
information on what treatments really work, what kind of diagnostic
studies are really necessary, and in what sequence. We relied very
heavily on our adult colleagues and friends and collaborators of
what works in adults and how do we apply them to children? If we
apply them to children, how do we apply them to infants and
newborns? We know that infants and newborns, of course, have
different physiologies, meaning how the heart and the lungs work
together and how pulmonary hypertension develops. We also know that
their bodies metabolize drugs differently and the effects of growth
and what that means with early interventions that perhaps one
cannot do in the adult. So many, many things were just unknown at
the time.
We went ahead and we were in touch with some of the sites around
the country, in North America more broadly. This was a little bit
over 15 years ago. We met together, first of all 8 sites, and now
we have 13 sites. These are sites in major institutions that
include Boston, Cincinnati, Philadelphia and Columbia in New York.
In California, we have Stanford and the University of California in
San Francisco, Texas Children's Hospital, Vanderbilt University. We
have Alberta in Canada. So we have many different teams of folks
from all over who are engaged here. So as these programs began to
develop, we formed this network, the Pediatric Pulmonary
Hypertension Network, or PPHNet (www.pphnet.org).
Over the years we've had some grant dollars from the government,
the NIH. We have had many, many meetings, have done research
together, written papers together. What we've done is really formed
the infrastructure that we can now move forward to advance our
studies and understanding of pediatric pulmonary hypertension.
Now we have a number of programs that address several missions.
Number one is how to overall improve the outcomes of our children
and how to work closely with families to achieve that goal. Number
two, how could we better inform ourselves so we do the best thing?
Educate the community, meaning other medical centers that are
seeing children with pulmonary hypertension to develop optimal
programs in their region or through centers. Also to then advocate
for our families and our kids through the FDA to the National
Institute of Health for research through government. Then finally,
how to educate the next generation to make sure that our younger
subspecialists in the field that I mentioned can be actively
engaged in solving the problems related to pulmonary
hypertension.
Over the 15 years then, the PPHNet has really developed a nice
patient registry, which provides a lot of information from well
over 1600 patients already. That provides us with a sense of the
natural history of the disease, how we're treating them, thinking
about their disease, how we can improve outcomes in the different
diseases associated with pulmonary hypertension. These include the
idiopathic forms, that is, we don't really know their cause, the
genetic or heritable causes, causes related to lung diseases, heart
diseases or systemic, meaning diseases from anywhere in the body
that could be liver, blood cancer, many other things where we see
pulmonary hypertension.
Every year we've had a meeting, our organizational meeting in San
Francisco. It's also paired with a scientific conference, which is
called the Neonatal and Pediatric Pulmonary Vascular Disease
Conference, organized by Dr. Jeff Fineman as our leader. He's been
fantastic pulling members from the PPHNet and others together to
help organize the best of experts in various aspects of childhood
diseases.
We're also expanding our engagement of families, getting them more
involved so we can make sure we advocate for them, meet their
needs, do the educational part that's so vital, and get families
talking to each other. So most recently we formed in the network,
largely thanks to Dr. Russell Hirsch at Cincinnati Children's, this
idea of PHriends of PPHNet. The PHriends really means non-medical
people and people who could really help us make sure that we're
meeting our goals of being advocates and colleagues and meeting the
needs of the community and their families. So we're launching that
as a major activity and that's quite exciting.
Along these lines, we've developed a website and the website will
include a number of resources and information about our centers,
but also different projects and goals and activities that PPHNet
will be achieving. We also opened it up now from our 13 centers to
individual members. So we'll have a number of folks from all over
the country and from all over Canada as well, North America right
now, who could join us. Then they could work towards developing
their sites into successful programs. Then their sites can
subsequently join us as we get the word out on how to better
approach children from both our clinical research as well as our
laboratory research, how to then communicate advances into
effective therapies by working closely with pharmaceuticals, with
regulatory agents, especially the Food and Drug Administration, the
FDA and others, and to really finally try to just change the
course.
Things are already getting better. Very exciting data was presented
at this year's meeting. We're already seeing nice responses with
very complex diseases to some of the therapies that we're using
when applied to the right populations who are evaluated in the
right fashion and followed closely. The meeting also highlighted
certain causes of pulmonary hypertension or associated diseases.
Some of them are what we call developmental lung diseases where
there is a genetic basis for the lungs not growing properly, which
then contributes to the pulmonary hypertension.
So how can we get smarter about that? One of our big workshops
included experts in genetics, in pathology, to look at the lung
tissue, the structure of the vessels and the airspaces that they
travel with. We had members from neonatology and cardiology speak
out. We had family and parents' perspective talking about all the
issues of the care of their child and what it was like being in the
intensive care unit going through such a difficult course when
we've had very little to offer in that time window to improving the
outcomes of their children. So we've had all these folks coming
together, and that's a good marker for the kinds of missions that
we have.
One of the major goals of the PPHNet is to really work closely with
physicians and care providers from all over the country. We provide
a resource for families as well. That's what PPHNet does. We talk
with all sorts of very, very talented care providers, physicians
and nurses and others. We advise and we'll discuss details on cases
and we're available for those kinds of consultations. There is a
lot of talent around the country absolutely, that are not
necessarily PPHNet sites, but what we try to do is encourage people
then to develop programs that really sort of provide the kind of
depth and breadth of support from having multiple disciplines
involved. Again, lung specialists, pulmonologists, neonatologists,
specialists for the newborn, hematologist, oncologist. We work as a
team to make sure that we could provide a team approach by talking
about the kids together, caring for them together. We're trying to
optimize care at as many sites as possible, but sometimes because
we have these as sites that have this experience and expertise, and
also we have striking interest in research with the registry and
collecting patient data and things like that. That's where the
PPHNet provides additional service.
So if you're the parent of a child who has pulmonary hypertension
in a place where the medical center just doesn't have all these
specialties, or they haven't yet formed a pulmonary hypertension
program, that don't have talented individuals who really could
provide great care for your kiddo, we're a resource to help advise
and counsel. At those centers, they could then work with us to
develop the kind of care program more broadly. Short of that, of
course, we do get children who will travel to some of the sites
that are close to their home for further in-depth evaluation. We're
really not trying to take over the care, but simply to really help
highlight and review and offer other opportunities or
recommendations for care. So it really is still a part of the team
with your local doctor and your local center. Communication's
essential. We're certainly available to consult and get engaged
with local care providers and to work together to help the
families.
Overall, the scientific conference that we've just had, our
organizational meeting for PPHNet and its growth, all of these
things are very, very exciting times. We're really looking forward
to really changing the course of all forms of pulmonary
hypertension, improving not just survival, but the quality of life.
I think exciting advances are happening. So it's wonderful to be
part of the PPHNet.
My name is Steven Abman, and I'm aware that our patients are
rare.
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