2011 Cardiac Stewardship Report

A REPORT ON OUR EFFORTS TO HELP AND HEAL: 2011

FOR CHILDREN’S

Hearts

giftofchildhood.org/supportcardiac

Kareim, 8, heart transplant patient

dear friend, 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

with all my heart

T H A N K YO U

THOMAS L. SPRAY, M.D.

SUSAN C. NICOLSON, M.D.

Chief, Division of Cardiothoracic Surgery

Medical Director, Cardiac Center

Alice Langdon Warner Endowed Chair

Chief, Division of Cardiothoracic Anesthesia

in Pediatric Cardiothoracic Surgery

Josephine J. Templeton Endowed Chair in Pediatric Anesthesiology Clinical Education

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

THERESA R. SCHULTZ, M.B.A., R.N.

Jennifer Terker Endowed Chair

Interim Director of Cardiac Nursing

in Pediatric Cardiology

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

GRATITUDE FOR GRANDDAUGHTER’S HEALTH LEADS TO DAISY DAY GIFT

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

His program brings together physicians from different

had grown as the pregnancy progressed, and both pumping

specialties, including cardiology, pulmonology, gastroenterology,

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

hepatology and endocrinology, to address the complications.

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

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

the more radical surgeries required for single ventricle defects.

focus on this group and to acquire solutions so these children

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 fire,” 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,”

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 nonprofit 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 sildenafil improves heart function in single ventricle patients.

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/

The Daisy Day Luncheon 2011 Park Hyatt Philadelphia at the Bellevue Wednesday, April 27, 2011

McAndrews family is the honorary chair sponsor of the 2011 Daisy Day Luncheon, which will benefit the Single Ventricle Survivorship Program.

Founded by the Auxiliary of The Children’s Hospital of Philadelphia in 1953, Daisy Day is the largest

“We are grateful to CHOP for the excellent care and compassion showed to Madeline and our family during

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

GREAT GIFTS

annual volunteer fundraising effort benefiting Children’s

extremely terrifying times. The outcome was beyond

Hospital. Since its inception,

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

when volunteers sold daisies

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

on street corners throughout

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

the city, Daisy Day has grown

Karen and Brian McAndrews went home knowing their baby

to help others similarly affected like Madeline through a

might be born with a heart with only one functioning pumping

meaningful contribution.”

chamber — a single ventricle. Karen went to her computer and learned that single ventricle heart defects are among the most

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

While Madeline McAndrews was still in the womb, her parents

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

survive. Now, after surgery to alter their circulatory systems

came to The Children’s Hospital of Philadelphia and faced

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

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

frightening news.

that’s what we were facing.”

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

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

Looking back, the McAndrews feel hugely fortunate. The

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

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

Cardiac Center team helped them through the pregnancy, and

expected. He would track the heart defect as the pregnancy

serious complications, including abnormal heart rhythms,

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

progressed to get a better idea of its severity.

growth and puberty delays, liver disease, and gastrointestinal

by ambulance to CHOP.

and lung disorders.”

growing alarm that these patients are unlikely to have a normal

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 4

A PRESSING CHALLENGE IN PEDIATRIC CARDIAC CARE

Families whose children have surgery or other procedures at CHOP take comfort in knowing the Cardiac Center team will be with them for years,

SURVIVAL IS NOT ENOUGH

helping immediately if problems arise.

Children with complex heart defects, particularly those severe enough to require surgery in the first 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

As cure rates for childhood cancers began climbing

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

in the 1970s, doctors soon realized survivors were

only one functioning pumping chamber (ventricle).

experiencing “late effects,” complications from their

As these children are, for the first time, living into their

disease and treatment.

teens and twenties, they’re experiencing a range of complications. Rychik brings together a team from

Pediatric oncologists responded to ensure complications

endocrinology, gastroenterology, hepatology and other

were treated and even prevented. Helping lead the way

specialties to focus on their care.

was The Children’s Hospital of Philadelphia, where the first program in the country to care for and track long-

As patients outgrow pediatrics, it’s imperative that

term survivors of childhood cancers was founded in 1983.

they continue to have doctors who know their unique physiology. The Philadelphia Adult Congenital

Another survivorship challenge is happening right now,

Heart Center, a partnership between CHOP and

in a different field — pediatric cardiac care. Surgeons

Penn Medicine, provides that team of experts.

began attempting new procedures in the 1970s,

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

but into the 1980s, most infants with serious heart

treating adult survivors of congenital heart defects,

defects died. As the available treatments became more

was recently hired as the new director of the Center.

sophisticated, survival rates began climbing significantly

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.

in the 1990s. Most babies with heart defects now

“Right now there are more adult survivors of

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

congenital heart defects than there are children

rising to meet the unique challenges of cardiac survivors.

under the age of 16 (with those defects),” says Stephanie Fuller, M.D., a cardiothoracic surgeon

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

who operates on both pediatric and adult patients.

one of the most comprehensive cadres of programs for

“An adult center sets a platform for having surgeons

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

and physicians who really are lifelong partners in

of the NeuroCardiac Care Program at CHOP. “Our

managing your disease. We don’t say goodbye as

responsibility to our patients doesn’t end with their

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

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

specialists who are truly devoted to their patients.”

With a Little Help: Women’s Committee Provides Grant to NeuroCardiac Care Children with complex heart defects, particularly those who require surgery in the first months of life, are at higher risk for learning disabilities, attention deficit 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 floor 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.”

6

GIFT ADVANCES RESEARCH ON EFFECTS OF STRESS CAUSED BY PRENATAL DIAGNOSES

MOTHER’S WORRY, BABY’S HARM? For a pregnant woman, discovering that the baby she is

also step up our efforts towards stress relief and perhaps refer

carrying has a heart defect is devastating. Stress can harm

mothers to proven and effective stress-reduction programs such

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

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

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

Scientists have proved that some factors during pregnancy,

The Children’s Hospital of Philadelphia plan to measure stress

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

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

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

fetus, in the mother and between mother and fetus.

health is a relatively new field of study. Some research has shown that maternal stress during pregnancy is associated with

Prenatal diagnoses are helpful, because parents have time to

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

absorb the news and doctors can monitor the pregnancy and

other developmental problems.

plan to immediately stabilize the newborn, if necessary. But can the stress of the diagnosis before birth disrupt fetal circulation,

There has been surprisingly little research on stress in women with

potentially resulting in grave consequences for fetal development

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

and even the long-term health of the child?

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

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

A remarkable gift several years ago from Anne and John Bazik

toward more carefully monitoring the mom in fetal care. The

made such research possible. Their gift funded two Fetal Heart

researchers have applied for funds from the March of Dimes.

Program studies showing that women with a prenatal diagnosis of a heart defect have higher rates of depression and anxiety

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

than women with a normal fetus. These results were crucial

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

preliminary data in applying for the March of Dimes funding

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

for the larger study.

Dolores Harrington Endowed Chair in Pediatric Cardiology.

7

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

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

effects. Eventually it may also help us address the question

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

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

of how private gifts from generous and devoted individuals are

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

so vital to our efforts.”

8

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.

DECORATED FOR BRAVERY Beads of Courage is a program in which patients receive

the establishment of the Bridgette Lauren Gori Child Life

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

Fund have supported the program in both cardiology and

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

oncology ever since.

poke, a black bead; for every admission to the intensive care unit, a glass star. When children show particular strength or

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

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

recipient at CHOP. During her stay, Bridgette loved getting

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

stickers after different procedures. She collected three albums

to mark every imaginable procedure. Depending on their

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

length of stay, participating kids collect anywhere from a few

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

dozen to a few thousand beads.

Bridgette passed away in 2001 at the age of four, the Goris continue to support the Child Life department, which gave

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

Bridgette so much joy.

in the hospital didn’t just happen to you — this is something you did,” says Child Life specialist Sherry Polise, who runs the

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

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

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

You got through this.”

commemorate and honor John M. Rodak, Joanne’s brother who was killed in the World Trade Center attack the same week

The beads can also facilitate conversations, opening the door

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

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

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

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

funds raised through the tournament are donated to Bridgette’s

classmates understand what she had gone through. The beads

fund at CHOP. When children begin the Beads of Courage

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

program, they receive beads spelling out their name plus two

the child when he or she grows up.

starter beads, one bearing Bridgette’s name, the other with John’s. The two sit side-by-side.

Beads of Courage is a national non-profit program used by more than 60 children’s hospitals. Polise brought Beads of

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

Courage to CHOP in 2007, thanks to the financial support of

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

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

and John would have been proud.”

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

10

PHILANTHROPY SUSTAINS WORK OF RENOWNED RESEARCHER

BETTER VALVES, BETTER LIVES

In his first years as a

Another option is valves from animals, most commonly pigs.

Endowed Fund for Cardiology Research, named for their

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

pediatric cardiologist

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

granddaughter, a Cardiac Center patient who is now a healthy

fields, comparable but far lower in magnetic strength than those

in the late 1970s,

with no anticoagulants required. But in children, because they

teenager. And Adam and Christine Kibel, whose son was

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

children born with

are experiencing rapid bone growth, the valves quickly became

treated at CHOP for an aortic valve defect, provide support

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

heart defects faced a

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

through the Kibel Fund for Cardiac Research.

and arteries open. The nanoparticles slowly released medicine

very difficult road.

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

Many did not make it.

calcified valves.

and prevented blockages. This localized treatment could prove Levy’s work is ongoing. Through the efforts of his team,

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

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

diabetics who suffer from poor circulation because of damaged blood vessels.

“It drove all of us crazy

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

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

to stand by and see a

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

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

child die like that,” recalls

Hospital of Philadelphia is an international leader in the quest

calcification. Another of his valves, the St. Jude Medical

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

Robert J. Levy, M.D.

for better replacement valves.

Trifecta Valve, is in clinical trials.

childhood cancer neuroblastoma. Dr. Levy is collaborating with

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

Meaningful research takes time. Reflecting on the past decades,

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

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

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

different directions.

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

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

®

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

was fascinated by a particular problem: replacement valves for

research into the major projects we have now.”

children with faulty heart valves. One option is mechanical

nanoparticles as carriers of chemotherapy to tumors.

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

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

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

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

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

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

Two generous donors, families of Cardiac Center patients,

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

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

clots. Children with mechanical valves take anticoagulants

helped this progress. Richard and Ann Seidel have supported

medical research: nanoparticles as targeted delivery systems for

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

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

research for more than a decade through the Erin Beatty

medicines and other therapeutics.

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

From a Seed, Forests

“The impact on my laboratory was quite significant, 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 outflow 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.

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 significantly improved. In addition, our magnetic delivery strategy enabled efficient cell guidance and prolonged residence at the stented vessels, which was not achievable without the magnetic targeting.”

Nanoparticles with (bottom) and without (top) surface modification

When 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.

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 modification to improve their function and provide magnetically

The results justified 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

NURSES’ PROJECTS EDUCATE COLLEAGUES AND FAMILIES

TEACHING ELEVATES CARE

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

Whether it’s monitoring patients after surgery or keeping an

Creating Opportunities for Parent Empowerment (COPE)

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

program with Cardiac Center families. This program educates

with cardiac problems often falls on two groups: nurses and

parents about how they can better monitor their children’s

parents. Ensuring these important caregivers have all of the

health and participate in their care. Although the research is

knowledge, skills and resources they need has been the focus

not yet complete, families using COPE at the Cardiac Center

of numerous initiatives this past year.

are responding positively.

Cardiac Center nurses proposed, developed and carried out

In a separate project, two Cardiac Center nurses used the

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 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

DAUGHTER’S HEART TRANSPLANT INSPIRES GIFTS

“A FOUNDATION FOR OUR FAMILY” Katrina Penney was only 24 hours old when her parents, Kati

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

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

to leave the Hospital. While she will remain on medication

had been diagnosed in utero with serious heart irregularities

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

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

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

Philadelphia from Bethesda, Md., specifically for the Cardiac

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

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

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

her problem could be corrected with surgery.

“It’s a great place to go and visit.”

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

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

more complicated than they’d realized.

it a priority to give back to other families at the Cardiac Center, bringing thoughtful donations like food for the

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

family lounge, DVDs and videogames for the playroom,

birth, CHOP had been mentally preparing her parents for

and loads of toys every Christmas. With every donation,

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

the Penneys leave behind a letter of hope to let families

Cardiac Intensive Care Unit, the operating rooms, and the

know that things do get better. Through their Penney Girls

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

Foundation, the family holds an annual golf tournament,

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

the proceeds of which have gone to support Child Life and

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

15

Katrina, 8, heart transplant patient

cardiac research at CHOP.

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

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

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

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

they heard a helicopter approach, the lights and blades casting

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

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

for our family.”

than a walnut, had arrived.

16

THE CARDIAC CENTER’S EFFORTS TO ELEVATE RESEARCH AND CARE NEVER STOP. HERE WE HIGHLIGHT JUST A FEW OF THE MANY RECENT PROJECTS.

CHOP-led Team Finds First Common Gene for Congenital Heart Disease A team led by a cardiothoracic surgeon at CHOP has discovered,

Gruber’s collaborators included his brother, Stephen B.

for the first time, common genetic variants that strongly raise

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

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

the University of Michigan Medical School, and Hakon Hakonarson, M.D., Ph.D., director of the Center for

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

Applied Genomics at CHOP.

Cardiac Center, and a team from Philadelphia and Michigan found that variants in the ISL1 gene had strong associations

Peter Gruber collected DNA samples from 300 CHOP

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

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

Public Library of Science One.

for Applied Genomics. Hakonarson’s team did the initial genotyping, looking for gene variants in the DNA of genes

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

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

chromosome 5, was crucial in regulating the development

common in children with certain variations in the gene.

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

EVERY DAY, EXCELLENCE

instead of assuming separate genes would govern each specific

Adding DNA from medical programs in Canada and the

defect, we formed the hypothesis that a common gene variant

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

operates early in the biological pathway of heart formation,

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

thus affecting multiple subtypes of congenital heart disease,”

controls, and confirmed in replication studies that variants in

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

ISL1 had strong associations with CHD.

ISL1 was a likely candidate.”

The Knowledge to Predict

New Standard for Sedation for Echocardiography Echocardiography, ultrasound imaging of the heart, is one

anesthesia takes effect more quickly than choral hydrate, and

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

The physiology of the heart changes over the course of the

of the most important and common tests in diagnosing and

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

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

pregnancy. Two fetuses with similar echo images at 18 weeks

monitoring heart problems in children.

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

don’t receive a medicine called prostaglandin or undergo

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

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

specialized treatment.

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

To obtain ideal images with echocardiography (“echo”), the patient must stay still. But children are not always known for

chloral hydrate. The results of the study were published in the Journal of the American Society of Echocardiographers in January.

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

However, if doctors know about the heart defect ahead of time and have a plan for care ready, babies are less likely

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

chloral hydrate has been used for children who must be sedated

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

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

during echo. However, doctors became dissatisfied with chloral

of Cardiothoracic Anesthesia, is the Josephine J. Templeton

echocardiography (ultrasound of the heart) allows doctors to

hydrate due to unpredictable onset and offset of sedation and

Endowed Chair in Pediatric Anesthesiology Clinical Education.

diagnose many heart defects before birth. But predicting the

With better standards by which to predict severity, doctors can

concern about the safety of the drug.

By supporting outstanding doctors such as Nicolson, endowments

severity can be difficult.

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

help elevate the level of care CHOP offers to children.

A team of pediatric cardiothoracic anesthesiologists and

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

helping to push that effort forward.”

should be born in the Garbose Family Special Delivery Unit at CHOP. Here, newborns can be in the care of a specially trained

cardiologists at CHOP decided to examine an alternative.

“Face mask anesthesia offers numerous advantages and is preferred

is working to establish some very important standards by

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

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

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

mask anesthesia.

become the standard for sedated echo at our institution. We

of heart defects. Their findings have appeared in the Journal

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

hope other institutions will examine our findings and consider

of the American Society of Echocardiography, with more

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

making the same change.”

publications pending.

standards for prenatal monitoring and for delivery that will

After comparing the charts of 507 patients, the team found significant advantages to face mask anesthesia. Since face mask

cardiac care team within seconds.

result in better health for our patients.”

Thank you!

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.

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.

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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.