A REPORT ON OUR EFFORTS TO HELP AND HEAL: 2011

FOR CHILDREN’S

Hearts

giftofchildhood.org/supportcardiac

We are so pleased to share with you this collection of updates on the Cardiac Center at The Children’s Hospital

of Philadelphia. Your generous gifts are vital to funding the unique research, care and education detailed in

the following pages. So much of what makes CHOP the best place for children with heart conditions is only

possible because of our community of philanthropic supporters. Whether you give every month or gave a

one-time gift a year ago, you should be proud of your contribution.

We hope you will enjoy learning more about what your dollars have helped to accomplish. The Cardiac Center

offers a continuum of care for heart patients, starting with the Fetal Heart Program that diagnoses, evaluates

and manages congenital heart disease before birth. The continuum extends through the Philadelphia Adult

Congenital Heart Center, which is increasingly important as our patients live longer, healthier lives.

In between, the Cardiac Center helps children with everything from addressing elevated cholesterol levels,

to surgical correction of structural problems, to heart transplants. The follow-up for children who have had

surgery or other serious interventions involves monitoring their mental and emotional development as well

as their hearts, highlighting CHOP’s dedication to caring for the whole child.

In the following pages, you will read about a wide range of exciting research, innovative approaches to care and

even ways we help our young patients keep up their spirits. You enable all of these things to happen. Thank you

for your generosity to the Cardiac Center and the children and families we serve.

Sincerely, The Cardiac Center Executive Committee

dear friend,

THOMAS L. SPRAY, M.D.Chief, Division of Cardiothoracic Surgery

Alice Langdon Warner Endowed Chair

in Pediatric Cardiothoracic Surgery

ROBERT E. SHADDY, M.D.Chief, Division of Cardiology

Jennifer Terker Endowed Chair

in Pediatric Cardiology

SUSAN C. NICOLSON, M.D.Medical Director, Cardiac Center

Chief, Division of Cardiothoracic Anesthesia

Josephine J. Templeton Endowed Chair

in Pediatric Anesthesiology Clinical Education

THERESA R. SCHULTZ, M.B.A., R.N.Interim Director of Cardiac Nursing

MARY JO TIMLIN-HOAG R.N., B.S.N., M.P.H.Director, Cardiac Center Service Line

Kareim, 8, heart transplant patient

THANK YOUwith all my heart

Madeline had two heart surgeries before she was a year old. Her family wanted to give back.

GREAT GIFTSWhile Madeline McAndrews was still in the womb, her parents

came to The Children’s Hospital of Philadelphia and faced

frightening news.

After fetal echocardiography, their doctor, Jack Rychik, M.D.,

told them one side of their daughter’s heart was smaller than

expected. He would track the heart defect as the pregnancy

progressed to get a better idea of its severity.

Karen and Brian McAndrews went home knowing their baby

might be born with a heart with only one functioning pumping

chamber — a single ventricle. Karen went to her computer and

learned that single ventricle heart defects are among the most

severe, requiring at least three open heart surgeries. “We were so

scared,” she recalls. “It’s a very hard diagnosis, and we thought

that’s what we were facing.”

Looking back, the McAndrews feel hugely fortunate. The

Cardiac Center team helped them through the pregnancy, and

after Madeline was born on October 5, 2007, she was taken

by ambulance to CHOP.

GRATITUDE FOR GRANDDAUGHTER’S HEALTH

LEADS TO DAISY DAY GIFT

It was a happy ending — and beginning: Madeline’s heart

had grown as the pregnancy progressed, and both pumping

chambers (ventricles) were functioning. While she still had a

serious heart defect, atrio-ventricular canal, she would not need

the more radical surgeries required for single ventricle defects.

Madeline had heart surgery at 5 days and at 8 months. Today,

at 3, she is an energetic “girly-girl” who loves music and old

movies like “The Wizard of Oz.”

“She is just a little ball of fi re,” Mrs. McAndrews says. “Tons

of energy. We do dance class. We do gymnastics. And she has

to wear her red sparkly ‘Dorothy’ shoes every day.”

The family remains in the care of the Cardiac Center.

Dr. Rychik is Madeline’s primary cardiologist, and she visits

annually for checkups. “We have this wonderful little girl,”

her mother says, “and we feel indebted to CHOP.”

When Madeline’s parents and maternal grandfather, Bob

Klimasewski, heard Dr. Rychik was starting a program focused

on single ventricle patients, they knew they wanted to help

other families who faced these severe defects. The Klimasewski/

McAndrews family is the honorary chair sponsor of the 2011

Daisy Day Luncheon, which will benefi t the Single Ventricle

Survivorship Program.

“We are grateful to CHOP for the excellent care and

compassion showed to Madeline and our family during

extremely terrifying times. The outcome was beyond

expectations,” says Mr. Klimasewski. “We can’t do surgery,

advise on cardiac issues or prescribe meds. But we can assist

CHOP and, in particular, Dr. Rychik to advance his ability

to help others similarly affected like Madeline through a

meaningful contribution.”

Thirty years ago, patients with single ventricle defects didn’t

survive. Now, after surgery to alter their circulatory systems

and hearts, most survive. Many are living into their teens and

twenties. However, Dr. Rychik says, “We are realizing with

growing alarm that these patients are unlikely to have a normal

quality of life or a normal duration of life. They are experiencing

serious complications, including abnormal heart rhythms,

growth and puberty delays, liver disease, and gastrointestinal

and lung disorders.”

His program brings together physicians from different

specialties, including cardiology, pulmonology, gastroenterology,

hepatology and endocrinology, to address the complications.

“Survival alone is not adequate,” he says. “Our purpose is to

focus on this group and to acquire solutions so these children

can have long and healthy lives.” The team provides clinical

care to single ventricle patients of any age, conducts research,

and is developing screening and treatment protocols. Without

philanthropic support, their efforts wouldn’t be nearly as robust.

Big Hearts to Little Hearts, a nonprofi t group of grateful

families of Cardiac Center patients, is also a presenting

sponsor of the Daisy Day Luncheon. The group has supported

Dr. Rychik’s work in the past, including helping fund a study

that showed the drug sildenafi l improves heart function in

single ventricle patients.

The Daisy Day Luncheon 2011Park Hyatt Philadelphia at the Bellevue

Wednesday, April 27, 2011

4

Founded by the Auxiliary of

The Children’s Hospital of Philadelphia

in 1953, Daisy Day is the largest

annual volunteer fundraising

effort benefi ting Children’s

Hospital. Since its inception,

when volunteers sold daisies

on street corners throughout

the city, Daisy Day has grown

to include the prestigious Daisy Day

Luncheon and the Daisy Day Community

Campaign, which is supported by schools and

businesses across the region. To date, Daisy Day

has raised more than $12 million for patient-care

programs at Children’s Hospital.

More information at

DaisyDayLuncheon.org

6

With a Little Help: Women’s Committee Provides Grant to NeuroCardiac Care

Children with complex heart defects, particularly those who require

surgery in the fi rst months of life, are at higher risk for learning disabilities,

attention defi cit disorder and other academic, behavioral and emotional

problems than the general population. Thanks to a generous grant from the

Women’s Committee of The Children’s Hospital of Philadelphia, the

Cardiac Center’s NeuroCardiac Care Program (NCCP) will be able to help

those children and their parents get the best start and possibly avoid those

problems. The NCCP brings together specialists to provide screening and

long-term evaluation for cardiac patients who may be at risk. The unique

team includes cardiologists, neurologists, developmental pediatricians,

nurses, nutritionists, social workers, psychologists, and physical, speech

and occupational therapists. Beginning in infancy, patients visit at regular

intervals for evaluations of developmental milestones, such as crawling,

so problems may be addressed immediately.

The grant will be used for a variety of purposes, including developing

materials to educate families about ways to help their children recover

from open heart surgery, such as scar massage, tummy time and much

more. Massaging the scar promotes healing and keeps chest muscles

loose — so movement isn’t inhibited as the child grows. And tummy

play-time — to give babies a chance to work their arms and learn to

crawl — is even more essential for babies who have been hooked to

machines in intensive care and are used to being on their backs.

The Women’s Committee gift will also purchase fl oor mats, therapy balls

and other equipment, and software, questionnaires and screening tools

for neurodevelopmental issues. The NCCP will use the funds to create a

registry, tracking every patient’s progress. The database could serve as

a template for other institutions and eventually be combined with others

into a national registry to help researchers learn about and plan care for

the ever-growing group of cardiac survivors.

One of the most important uses of the Women’s Committee grant will be

a small fund for vouchers for meals, public transportation, gas, parking

and the other costs that can quickly become unmanageable for families

visiting CHOP frequently.

“It’s absolutely wonderful that philanthropy is helping to cover those costs

right now,” says Jean M. Carroll, R.N., M.S.N., NCCP program manager.

“In these economic times families are facing even greater challenges in

getting to their appointments. That people really care and have reached

out to help means the world.”

As cure rates for childhood cancers began climbing

in the 1970s, doctors soon realized survivors were

experiencing “late effects,” complications from their

disease and treatment.

Pediatric oncologists responded to ensure complications

were treated and even prevented. Helping lead the way

was The Children’s Hospital of Philadelphia, where the

fi rst program in the country to care for and track long-

term survivors of childhood cancers was founded in 1983.

Another survivorship challenge is happening right now,

in a different fi eld — pediatric cardiac care. Surgeons

began attempting new procedures in the 1970s,

but into the 1980s, most infants with serious heart

defects died. As the available treatments became more

sophisticated, survival rates began climbing signifi cantly

in the 1990s. Most babies with heart defects now

survive. Just as it did with cancer survivors, CHOP is

rising to meet the unique challenges of cardiac survivors.

“The Cardiac Center has built, and continues to build,

one of the most comprehensive cadres of programs for

cardiac survivors,” says Gil Wernovsky, M.D., director

of the NeuroCardiac Care Program at CHOP. “Our

responsibility to our patients doesn’t end with their

hospitalizations. As they grow, we’re still here for them.”

Families whose children have surgery or other

procedures at CHOP take comfort in knowing the

Cardiac Center team will be with them for years,

helping immediately if problems arise.

Children with complex heart defects, particularly

those severe enough to require surgery in the fi rst

months of life, have a higher incidence of academic,

behavioral and coordination problems than children

without complex heart defects. The NeuroCardiac

Care Program brings together a team of specialists

to provide screening and long-term evaluation for

patients who may be at risk. (See accompanying article.)

The Single Ventricle Survivorship Program, led by

Jack Rychik, M.D., is for patients born with hearts with

only one functioning pumping chamber (ventricle).

As these children are, for the fi rst time, living into their

teens and twenties, they’re experiencing a range of

complications. Rychik brings together a team from

endocrinology, gastroenterology, hepatology and other

specialties to focus on their care.

As patients outgrow pediatrics, it’s imperative that

they continue to have doctors who know their unique

physiology. The Philadelphia Adult Congenital

Heart Center, a partnership between CHOP and

Penn Medicine, provides that team of experts.

Yuli Y. Kim, M.D., who has special training in

treating adult survivors of congenital heart defects,

was recently hired as the new director of the Center.

“Right now there are more adult survivors of

congenital heart defects than there are children

under the age of 16 (with those defects),” says

Stephanie Fuller, M.D., a cardiothoracic surgeon

who operates on both pediatric and adult patients.

“An adult center sets a platform for having surgeons

and physicians who really are lifelong partners in

managing your disease. We don’t say goodbye as

soon as you turn 18. It’s a continuum of care from

specialists who are truly devoted to their patients.”

SURVIVAL IS NOT ENOUGH

A PRESSING CHALLENGE IN PEDIATRIC CARDIAC CARE

Abby, 18 months, has a complex

heart defect, double inlet left

ventricle, and is followed by

numerous teams at CHOP.

Years ago, when survival rates

for complex heart defects were

low, Abby’s grandma lost a

daughter to the same heart

defect. Today, the family rejoices

in hope for Abby’s long life.

8

For a pregnant woman, discovering that the baby she is

carrying has a heart defect is devastating. Stress can harm

the mother’s health. How might it affect the baby?

In a new study, researchers at the Fetal Heart Program at

The Children’s Hospital of Philadelphia plan to measure stress

levels after fetal diagnosis and their effect on blood flow in the

fetus, in the mother and between mother and fetus.

Prenatal diagnoses are helpful, because parents have time to

absorb the news and doctors can monitor the pregnancy and

plan to immediately stabilize the newborn, if necessary. But can

the stress of the diagnosis before birth disrupt fetal circulation,

potentially resulting in grave consequences for fetal development

and even the long-term health of the child?

The study will ask these difficult questions and may be a step

toward more carefully monitoring the mom in fetal care. The

researchers have applied for funds from the March of Dimes.

“By making a prenatal diagnosis, we think we’re helping, but is

it possible we are also causing some harm?” says Jack Rychik,

M.D., director of the Fetal Heart program and the Robert and

Dolores Harrington Endowed Chair in Pediatric Cardiology.

“This study will help us gain a better understanding of those

effects. Eventually it may also help us address the question

of how we can help modify stress. What we may find is that

when we make a prenatal diagnosis of a birth defect, we should

MOTHER’S WORRY, BABY’S HARM?

GIFT ADVANCES RESEARCH ON EFFECTS OF STRESS

CAUSED BY PRENATAL DIAGNOSES

also step up our efforts towards stress relief and perhaps refer

mothers to proven and effective stress-reduction programs such

as group counseling, or even yoga or massage therapy.”

Scientists have proved that some factors during pregnancy,

such as smoking, are harmful to the fetus. However, the effects

of maternal stress on fetal development and the child’s long-term

health is a relatively new field of study. Some research has

shown that maternal stress during pregnancy is associated with

early labor, low birth weight and, later, cognitive delays and

other developmental problems.

There has been surprisingly little research on stress in women with

high-risk pregnancies. The Fetal Heart Program study would be

one of the first of its kind in this important and emerging field.

A remarkable gift several years ago from Anne and John Bazik

made such research possible. Their gift funded two Fetal Heart

Program studies showing that women with a prenatal diagnosis

of a heart defect have higher rates of depression and anxiety

than women with a normal fetus. These results were crucial

preliminary data in applying for the March of Dimes funding

for the larger study.

“The Bazik gift clearly laid the foundation for a whole new field

of research for us,” Dr. Rychik says. “It’s a remarkable example

of how private gifts from generous and devoted individuals are

so vital to our efforts.”7

DECORATED FOR BRAVERYBeads of Courage is a program in which patients receive

a bead for any event or milestone during their stay: For every

echocardiogram, a glow-in-the-dark bead; for every needle

poke, a black bead; for every admission to the intensive care

unit, a glass star. When children show particular strength or

bravery, they receive an “act of courage” bead, a unique hand-

made glass bead donated by artists. There are dozens of beads

to mark every imaginable procedure. Depending on their

length of stay, participating kids collect anywhere from a few

dozen to a few thousand beads.

“The beads help to remind kids that this experience of being

in the hospital didn’t just happen to you — this is something

you did,” says Child Life specialist Sherry Polise, who runs the

program in the Cardiac Center. “You were brave and strong.

You got through this.”

The beads can also facilitate conversations, opening the door

for Child Life to discuss different procedures with a child. One

girl brought her beads to school for show and tell, helping her

classmates understand what she had gone through. The beads

can also give parents of an infant patient a memento to show

the child when he or she grows up.

Beads of Courage is a national non-profi t program used by

more than 60 children’s hospitals. Polise brought Beads of

Courage to CHOP in 2007, thanks to the fi nancial support of

the family and friends of Joanne and Peter Gori, who through

the establishment of the Bridgette Lauren Gori Child Life

Fund have supported the program in both cardiology and

oncology ever since.

The couple’s daughter, Bridgette, was a double lung transplant

recipient at CHOP. During her stay, Bridgette loved getting

stickers after different procedures. She collected three albums

full. “The stickers meant so much to her,” says Joanne. “I can

only imagine how much she would have loved beads.” While

Bridgette passed away in 2001 at the age of four, the Goris

continue to support the Child Life department, which gave

Bridgette so much joy.

Each year since 2003, Joanne and Peter, together with their

family, hold the JMR Charity Classic, a golf tournament to

commemorate and honor John M. Rodak, Joanne’s brother

who was killed in the World Trade Center attack the same week

that Bridgette passed away. John was Bridgette’s godfather, and

the event is another symbol of their connection. Most of the

funds raised through the tournament are donated to Bridgette’s

fund at CHOP. When children begin the Beads of Courage

program, they receive beads spelling out their name plus two

starter beads, one bearing Bridgette’s name, the other with

John’s. The two sit side-by-side.

“We wanted to donate to the Hospital in a way that directly

touched the kids’ lives,” says Joanne. “I know both Bridgette

and John would have been proud.”

Visitors to the Cardiac Center at The Children’s Hospital of Philadelphia might notice strands of colorful glass beads hanging from IV poles or sitting by the bedside. At first glance, they may seem like another cheerful decoration. But these beads are more than decorative. They tell the story of each child’s journey at CHOP.

This beautiful string of beads was created by a patient while she waited for a new heart. 10

In his fi rst years as a

pediatric cardiologist

in the late 1970s,

children born with

heart defects faced a

very diffi cult road.

Many did not make it.

“It drove all of us crazy

to stand by and see a

child die like that,” recalls

Robert J. Levy, M.D.

Over the next 30 years, a legion of scientist-physicians,

Dr. Levy among them, would rise to the many challenges

presented by their patients’ hearts. From the beginning, he

was fascinated by a particular problem: replacement valves for

children with faulty heart valves. One option is mechanical

(man-made) valves, which work well but are prone to blood

clots. Children with mechanical valves take anticoagulants

(“blood-thinners”) for their rest of their lives.

BETTER PHILANTHROPY SUSTAINS WORK OF RENOWNED RESEARCHER

Another option is valves from animals, most commonly pigs.

At the start of Levy’s career, these valves were working in adults,

with no anticoagulants required. But in children, because they

are experiencing rapid bone growth, the valves quickly became

covered in calcium. Dr. Levy saw waves of patients returning to

the hospital, often within months of surgery, severely ill with

calcifi ed valves.

Troubled by what he saw, Dr. Levy went to his lab and began

searching for answers. Today his research team at The Children’s

Hospital of Philadelphia is an international leader in the quest

for better replacement valves.

Meaningful research takes time. Refl ecting on the past decades,

Dr. Levy says, “It was really the private philanthropy that

kept us going. Because of that I was able to build my earliest

research into the major projects we have now.”

Two generous donors, families of Cardiac Center patients,

helped this progress. Richard and Ann Seidel have supported

research for more than a decade through the Erin Beatty

From a Seed, ForestsWhen a philanthropist makes a large donation to the Cardiac Center,

it is often only because of that donation that one of the many extra,

groundbreaking projects in research or care is possible. In those

cases, it is clear how the money given leads directly to expanded

services. But the hundreds of modest gifts that come into the Cardiac

Center every year also provide support for projects that could not

happen otherwise.

For several years, the Cardiac Center has been allocating money

from all of those donations made for the general support of the

Center to provide seed money to promising research projects that

would otherwise have no money to get started.

“The impact on my laboratory was quite signifi cant, as the preliminary

work that was collected with the help of the Cardiac Center was used

to secure a two-year grant from the March of Dimes Foundation,”

says Fraz Ahmed Ismat, M.D., who was provided funding for his study

of microRNA’s role in regulating the cardiac outfl ow tract. Dr. Ismat

notes that knowledge of microRNA’s role in disease is currently

advancing quickly, and that scientists suspect it plays a large part

in fetal development and congenital heart disease.

Cardiac Center donors are funding other work at the leading edge of

science. Michael Chorny, Ph.D., received funding for a study of using

magnetically guided nanoparticles to aid with the recovery of the

inner cell lining of an artery that has had a stent implanted.

“We developed biodegradable magnetic nanoparticles enabling genetic

cell modifi cation to improve their function and provide magnetically

Endowed Fund for Cardiology Research, named for their

granddaughter, a Cardiac Center patient who is now a healthy

teenager. And Adam and Christine Kibel, whose son was

treated at CHOP for an aortic valve defect, provide support

through the Kibel Fund for Cardiac Research.

Levy’s work is ongoing. Through the efforts of his team,

CHOP holds four valve-related patents. Tens of thousands of

patients have the St. Jude Medical Epic Valve®, created by

Levy. It is a porcine (pig) valve treated with a coating that slows

calcifi cation. Another of his valves, the St. Jude Medical

Trifecta Valve, is in clinical trials.

As so often happens, Levy’s research has branched off in

different directions.

Because heart valve disease is localized (in one place in the

body; not systemic), his focus has been on treatments targeting

one area. His team is now a leader in a burgeoning area of

medical research: nanoparticles as targeted delivery systems for

medicines and other therapeutics.

In a recent animal study, Dr. Levy’s team used uniform magnetic

fi elds, comparable but far lower in magnetic strength than those

present in MRI scanners, to drive iron-bearing nanoparticles to

metal stents, tiny devices used to keep narrow or injured veins

and arteries open. The nanoparticles slowly released medicine

and prevented blockages. This localized treatment could prove

helpful not only to heart patients, but also to the millions of

diabetics who suffer from poor circulation because of damaged

blood vessels.

Dr. Levy is also examining nanoparticles in another disease: the

childhood cancer neuroblastoma. Dr. Levy is collaborating with

Garrett Brodeur, M.D., an oncologist at CHOP, in a study of

nanoparticles as carriers of chemotherapy to tumors.

For Dr. Levy, the progress and the paths can be traced back to

the children who were so sick, and even died, when he was a

young doctor. Today, because of scientists like Dr. Levy, and their

supporters, the vast majority of children with heart defects live.

“It’s been revolutionary, what I’ve seen,” he says. “It’s miraculous.

We’re talking about quality of life now, instead of mortality.”

driven cell delivery to the injured arterial segment,” Chorny

explains. “Our studies showed that after the magnetic nanoparticle

treatment, the ability of cells to produce nitric oxide, an essential

element in the normal vessel homeostasis, was signifi cantly

improved. In addition, our magnetic delivery strategy enabled

effi cient cell guidance and prolonged residence at the stented

vessels, which was not achievable without the magnetic targeting.”

The results justifi ed additional studies as the technique moves

towards a possible place as a treatment for children in the Cardiac

Center in the future.

In all, from 2007 through 2009, 21 research studies were

supported by Cardiac Center donors. Through advancing basic and

clinical science in all aspects of cardiac care, donors like you are

building a better future for children with cardiac conditions.12

Nanoparticles with (bottom)and without (top) surface modifi cation

VALVES, BETTER LIVES

TEACHING ELEVATES CARE

NURSES’ PROJECTS EDUCATE COLLEAGUES AND FAMILIES

Whether it’s monitoring patients after surgery or keeping an

eye on them at home for signs of distress, caring for children

with cardiac problems often falls on two groups: nurses and

parents. Ensuring these important caregivers have all of the

knowledge, skills and resources they need has been the focus

of numerous initiatives this past year.

Cardiac Center nurses proposed, developed and carried out

six educational projects for parents and nurses last year, all

made possible by a donation from Big Hearts to Little Hearts,

a group of patient families and supporters who have raised

more than $650,000 for a variety of Cardiac Center programs

over the past seven years.

“The competence and the confidence of the nurse who is

providing the care at the bedside and beyond really heavily

relies upon education,” says Theresa Schultz, M.B.A., R.N.,

interim director of Cardiac Center nursing. “These are nurses

identifying what nurses’ needs are and then pulling together

all the expertise to meet that learning need.”

One project brought together nurses from CHOP and the

Hospital of the University of Pennsylvania to collaborate on

how best to transition childhood cardiac patients to adult

care. Another provided an opportunity for nurses to learn

about and practice procedures that are rarely needed, but are

vitally important.

Big Hearts to Little Hearts funding allowed CHOP nurses to

develop and host a pediatric cardiovascular nursing conference

in September, the contents of which were turned into online

training for nurses. As a separate project, Cardiac Center

nurses researched best practices in developmental care, in which

factors like skin-to-skin contact with a parent or different

lighting configurations can promote a child’s well-being.

While all six projects have shown positive results and will

be continued in some form in the future, some of the most

dramatic improvements seen so far were from the two

projects focused on family education. One study is testing the

Creating Opportunities for Parent Empowerment (COPE)

program with Cardiac Center families. This program educates

parents about how they can better monitor their children’s

health and participate in their care. Although the research is

not yet complete, families using COPE at the Cardiac Center

are responding positively.

In a separate project, two Cardiac Center nurses used the

Big Hearts to Little Hearts grant to produce a manual for

parents of children who are being evaluated for potential

heart transplants, helping parents distinguish normal events

from symptoms that should cause concern. Thanks to the

manual, the nurses are now receiving fewer worried phone

calls from parents.

Lynne Ramsay, president of Big Hearts to Little Hearts,

was impressed by the transplant manual and says that the

members of her organization are pleased with the effect

the nursing education projects are having.

“It is really good to know your child is in the very best

informed, capable hands,” Ramsay says, adding that she hopes

the community of donors supporting the Cardiac Center

continues to grow to provide that level of care. “Congenital

heart disease is so much more prevalent and common than

people realize. It is so grossly underfunded, and I don’t think

people are aware how many children are affected by it.”Hailee-Anne, 1 month, born with double inlet left ventricle, in the Evelyn and Daniel M. Tabas Cardiac Intensive Care Unit

14

“A FOUNDATION FOR OUR FAMILY”

DAUGHTER’S HEART TRANSPLANT INSPIRES GIFTS

Katrina Penney was only 24 hours old when her parents, Kati

and Jon, learned she would need a heart transplant. Katrina

had been diagnosed in utero with serious heart irregularities

— her parents had come to The Children’s Hospital of

Philadelphia from Bethesda, Md., specifically for the Cardiac

Center’s expertise — but Katrina’s doctors anticipated that

her problem could be corrected with surgery.

But now the doctors could see Katrina’s problem was even

more complicated than they’d realized.

Kati and Jon were shocked. In the months before Katrina’s

birth, CHOP had been mentally preparing her parents for

her surgery, showing them the Evelyn and Daniel M. Tabas

Cardiac Intensive Care Unit, the operating rooms, and the

step-down unit where she would go after surgery. The plan had

now changed: Katrina was on the list waiting for a transplant.

Katrina was fortunate — it took just nine weeks to find her

a donor heart. As Thomas L. Spray, M.D., took Katrina into

surgery, Kati and Jon sat in the lobby atrium. At 12:50 a.m.,

they heard a helicopter approach, the lights and blades casting

shadows onto the walls and floor. The donor heart, no bigger

than a walnut, had arrived.

The surgery was a success. Twelve days later, Katrina was able

to leave the Hospital. While she will remain on medication

for life to prevent rejection, Katrina is now a healthy second-

grader. She continues to return to CHOP for medical care,

but she and her sisters love stopping by even without a medical

appointment. “To them, it’s not just a building,” says Kati.

“It’s a great place to go and visit.”

Since Katrina’s surgery in 2003, the Penneys have made

it a priority to give back to other families at the Cardiac

Center, bringing thoughtful donations like food for the

family lounge, DVDs and videogames for the playroom,

and loads of toys every Christmas. With every donation,

the Penneys leave behind a letter of hope to let families

know that things do get better. Through their Penney Girls

Foundation, the family holds an annual golf tournament,

the proceeds of which have gone to support Child Life and

cardiac research at CHOP.

“We would never wish this experience on anyone, but the

result of what we’ve experienced we would never take back,”

says Kati. “It taught us so much and has been a foundation

for our family.” 1615 Katrina, 8, heart transplant patient

EVERY DAY, EXCELLENCE

THE CARDIAC CENTER’S EFFORTS TO ELEVATE

RESEARCH AND CARE NEVER STOP. HERE WE HIGHLIGHT

JUST A FEW OF THE MANY RECENT PROJECTS.

Echocardiography, ultrasound imaging of the heart, is one

of the most important and common tests in diagnosing and

monitoring heart problems in children.

To obtain ideal images with echocardiography (“echo”), the

patient must stay still. But children are not always known for

their ability to stay still. For many years, a drug called oral

chloral hydrate has been used for children who must be sedated

during echo. However, doctors became dissatisfi ed with chloral

hydrate due to unpredictable onset and offset of sedation and

concern about the safety of the drug.

A team of pediatric cardiothoracic anesthesiologists and

cardiologists at CHOP decided to examine an alternative.

In July 2007, the staff began offering sedated echo using face

mask anesthesia.

After comparing the charts of 507 patients, the team found

signifi cant advantages to face mask anesthesia. Since face mask

anesthesia takes effect more quickly than choral hydrate, and

usually wears off more quickly, visits for echo took much less

time. Also, the echo study was completed in all cases with face

mask anesthesia, as compared with a 6 percent failure rate with

chloral hydrate. The results of the study were published in the

Journal of the American Society of Echocardiographers in January.

Study co-author Susan C. Nicolson, M.D., chief of the Division

of Cardiothoracic Anesthesia, is the Josephine J. Templeton

Endowed Chair in Pediatric Anesthesiology Clinical Education.

By supporting outstanding doctors such as Nicolson, endowments

help elevate the level of care CHOP offers to children.

“Face mask anesthesia offers numerous advantages and is preferred

by both families and the medical team,” says Nicolson. “It has

become the standard for sedated echo at our institution. We

hope other institutions will examine our fi ndings and consider

making the same change.”

When a child is born with a severe heart defect, time is the

enemy. Babies can quickly get very sick, or even die, if they

don’t receive a medicine called prostaglandin or undergo

specialized treatment.

However, if doctors know about the heart defect ahead of

time and have a plan for care ready, babies are less likely

to go into shock or develop end-stage organ failure. Fetal

echocardiography (ultrasound of the heart) allows doctors to

diagnose many heart defects before birth. But predicting the

severity can be diffi cult.

A Cardiac Center team led by Michael D. Quartermain, M.D.,

is working to establish some very important standards by

using fetal echocardiography (“echo”) to predict the severity

of heart defects. Their fi ndings have appeared in the Journal

of the American Society of Echocardiography, with more

publications pending.

A team led by a cardiothoracic surgeon at CHOP has discovered,

for the fi rst time, common genetic variants that strongly raise

the risk of non-syndromic congenital heart disease (CHD).

Peter J. Gruber, M.D., Ph.D., an attending surgeon in the

Cardiac Center, and a team from Philadelphia and Michigan

found that variants in the ISL1 gene had strong associations

with CHD. The study appeared online in May in the journal

Public Library of Science One.

In Gruber’s previous research, he found that ISL1, on

chromosome 5, was crucial in regulating the development

of early cardiac progenitor cells. “In designing our study,

instead of assuming separate genes would govern each specifi c

defect, we formed the hypothesis that a common gene variant

operates early in the biological pathway of heart formation,

thus affecting multiple subtypes of congenital heart disease,”

said Gruber. “Based on previous research, we suspected that

ISL1 was a likely candidate.”

New Standard for Sedation for Echocardiography The Knowledge to Predict

The physiology of the heart changes over the course of the

pregnancy. Two fetuses with similar echo images at 18 weeks

gestation can be different at birth. One may be critically ill

because of a heart defect, while the other may have a milder

form. “We haven’t fully discovered all the markers that allow us

to reliably predict which fetuses will require immediate medical

attention after birth,” Dr. Quartermain says. “This work is

helping to push that effort forward.”

With better standards by which to predict severity, doctors can

make better plans. For example, they can decide if the baby

should be born in the Garbose Family Special Delivery Unit at

CHOP. Here, newborns can be in the care of a specially trained

cardiac care team within seconds.

“The more we know going into the delivery room, the better,”

Dr. Quartermain says. “We hope this research will help set

standards for prenatal monitoring and for delivery that will

result in better health for our patients.”

Gruber’s collaborators included his brother, Stephen B.

Gruber, M.D., Ph.D., a geneticist and epidemiologist at

the University of Michigan Medical School, and Hakon

Hakonarson, M.D., Ph.D., director of the Center for

Applied Genomics at CHOP.

Peter Gruber collected DNA samples from 300 CHOP

patients with CHD and 2,200 healthy children at the Center

for Applied Genomics. Hakonarson’s team did the initial

genotyping, looking for gene variants in the DNA of genes

in or near ISL1. The researchers found that CHD was more

common in children with certain variations in the gene.

Adding DNA from medical programs in Canada and the

Netherlands to the U.S. samples, the researchers studied genes

from a total of 1,344 children with CHD and 6,135 healthy

controls, and confi rmed in replication studies that variants in

ISL1 had strong associations with CHD.

CHOP-led Team Finds First Common Gene for Congenital Heart Disease

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34th Street and Civic Center Boulevard, Philadelphia, PA 19104-4399 • 267-426-5332

The Children’s Hospital of Philadelphia is one of only eight pediatric hospitals in the nation to be named to the prestigious U.S.News & World Report Best Children’s Hospitals Honor Roll for 2010-11.

The children you see throughout this report are our patients. It is for them, and for the many others in our care, that we relentlessly pursue the next advances in treatment and research. Your support is vital to the work of the Cardiac Center. To learn more about how you can help, please contact Robin Austin, associate vice president, at 267-426-5332 or austinr@email.chop.edu.

Thank you!

On the cover: Marisa, 7, born with heterotaxy syndrome, has been a patient of the Cardiac Center since birth. She is now part of the Single Ventricle Survivorship Program.