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Pediatric cardiologist Oscar J. Benavidez, Jr.,

MD, began his appointment as chie o pediatric

cardiology at MassGeneral Hospital forChildren

(MGHfC) in April 2011. Dr. Benavidez received

his medical degree at Harvard Medical School

and completed a residency and ellowship at

Childrens Hospital Boston. He is an assistant

proessor at Harvard Medical School. His primary

areas o ocus span a large age spectrum rom

etal to adolescent care.

Clinics are currently available in Boston, at

MGHfC at Newton-Wellesley Hospital and

at the Mass General/North Shore Center or

Outpatient Care in Danvers, MA. The combined

strength o expertise in noninvasive imaging and

collaborations with obstetricians, neonatologists

and pediatricians will provide patients with

improved diagnostic accuracy and earlier

identication o heart problems, leading to more

eective delivery planning and pediatric care.

Deeper collaboration with Massachusetts

General Hospital adult congenital cardiologists

and related specialists, Dr. Benavidez says, can

help improve patient care by providing in-depth

skill with the ull range o imaging equipment

used in the clinic and operating room.

Research aims or

the division include

identiying risk actors

that aect optimal

outcomes in congenital

heart surgery in both the

pediatric and adult population and uncovering

new strategies or improving the sensitivity o

the obstetrical screen or a etal diagnosis o

congenital heart disease.

For more information, call 888-644-3241

101MerrimacStreet,Suite200

Boston,MA02114

massgeneralforchildren.org

CascadesNewsletter to PhysiciaNs Fall 2011

NON-PROFITORG

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Inthisissue:TissueEngineeringandRegenerative

Medicine:Progresstowardalleviating

donororganshortages

QualityandCareEortsRefect

PassionorCareExcellence

NewChieoPediatricCardiology

PediatricTransplantation:Singlesite,

lietimecare

massgeneral forchildren.org | 888-64

CascadesFall 2011 Newsletter to PhysiciaNs

Tissue Engineering nd RegenerMedicine: Progress toward alleviatdonor organ shortages

Con

Research advances in the MassGeneral Hospital for

Children (MGHfC) Center or R egenerative Medicine

have brought the eld closer to engineering entire living

organs that are ree o immunosuppression challenges

and that can be used to replace organs in end-stage

ailure. As researchers work toward that goal, says

Joseph P. Vacanti, MD, deputy director o the Center or

Regenerative Medicine and surgeon in chie at MGHfC,

interim steps will produce living structures made rom the

patients own cells that can replace damaged or diseased

tissue. Already, many o these interim products have been

incorporated into clinical practice and hundreds more are

in the pipeline.

Deveoping orgn structureswith poymersDr. Vacanti began working in the eld o tissue

engineering 25 years ago, when the eld was in its

inancy. Throughout the 1980s, he experimented with

methods or growing cells on a palette o

polymers, eventually ocusing on the imp

three-dimensional scaold or solid orga

The addition o stem cells to the eld o

medicine urther advanced the value o D

Biodegradable scaolds, he notes, provi

structure or tissue-specic cell seeding

development. Normal healing processes

scaolding as it degrades.

Currently Dr. Vacantis lab ocuses on ncombination o synthetic polymer, cellula

growth actors and modications to the s

fexibility and surace eatures o the ma

unctional hearts, lungs and kidneys. Thr

eld o regenerative medicine, virtually e

in the human body is under study. To bec

says Dr. Vacanti, these components mus

bioreactor that exposes them to tissue-s

orces, such as pressure and fow or blo

compression and tension or cartilage an

In addition to generating progress towar

or tissues, knowledge and designs rom

Lab have resulted in assist devices that p

mechanical unction o organs but do not

cells. These devices have been used in a

or lung, liver and kidney. Once available

they will provide bridge therapy or patie

transplantation or destination therapy o

are not candidates or transplantation.

Joseph P. Vacanti, MD in his lab

New Chie o Peditric Crdioogylunches Fet Congenit Crdic Progrm

Oscar J. Benavidez, Jr., MD


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Newsletter to Phys

massgeneral forchildren.org | 888-644-3

Cascades

Tissue Engineering nd Regenertive Medicine: Progress towrd

eviting donor orgn shortgescontinued from page one

Page twomassgeneral forchildren.org | 888-644-3211

Ntur mtrices usingcdveric orgnsHarald Ott, MD, in the Center or R egenerative

Medicine, has worked with Dr. Vacanti to

expand on the value o m atrices in a dierent

direction by developing organ prousion

decellularization, a technology to remove all

cells rom a cadaveric organ. The remaining

collagen matrix is then inused with new adult-

derived stem cells in a process that is identical

to that used with the synthetic matrices. This

method o reseeding and engratment with

native cells potentially eliminates the need

or lie-long immunosuppression in transplant

patients, a particularly critical issue in pediatrictransplantation since children undergoing

organ transplant ace a lietime o imm uno-

suppressive medications.

Dr. Otts initial work was in the heart. Following

early animal studies that involved parts o

recellularized hearts, Dr. Ott is working with the

New England Organ Bank and the Mass General

Transplant Center in an attempt to upscale the

process o scaold generation to human hearts.

Additionally, his lab is attempting to isolate

human cardiomyocytes, cells critical to heart

unction. The lab is also applying the technique

in the lung which, says Dr. Ott, has an advantage

over heart regeneration in that it does not need

to provide active mechanical work. Instead, it

provides gas exchange through a membrane

that can be accessed on both sides to acilitate

cell seeding. To date, Drs. Ott and Vacanti have

reported the ability o transplanted regenerated

lungs to unction in vivo in animal models or

a period o up to 10 days. Failure appears due

to the need or cell subpopulations required or

mucociliary clearing. Dr. Ott is collaborating

with the Rajagopal Lab at Mass General to try

to reconstitute mucocilary clearance. Additional

eorts include applying the same approaches in

the pancreas, kidney and liver.

Since the results o this work were published in

2008, interest in using decellularized cadaveric

organs as a platorm or organ development has

exploded. While the goal o providing these new

organs in the clinic is still years away, says Dr.

Ott, the work will produce intermediary steps

o more immediate value to patients, including

the creation o patches that can be used to

replace damaged portions o hearts, lungs,

airways or other organs.

Successully bringing together all o the actors

necessary to create clinically viable living tissue

and organs requires the resources o MGHfC

and its collaborative relationship with MIT,

which provide researchers in the Center or

Regenerative Medicine with access to experts

in surgical science, chemical engineering,

cellular and molecular biology, and technology

development. Equally as critical, says Dr.

Vacanti, is the expertise o specialists in the

Mass General Transplant Center, who provide

insight into the most current understanding o

biology o the infammatory response.

Even though these living organs will be m ade

rom stem cells o the patient who needs

the organ, explains Joren C. Madsen, MD,

DPHL, director o the Mass General Transplant

Center and section chie o Cardiac S urgery,

the structure itsel is not o the patients

tissues. As a result, transplant specialists

and immunologists need to participate in

living organ development to address potential

immunological eects. As the eld o tissue

engineering and regenerative medicine m atures,

MGHfC clinical researchers remain committed

to continuing their leadership role in overcoming

the remaining biological and engineering

challenges. Each step o the way, says Dr.

Vacanti, brings medicine closer to the goal

o providing patients in need with biological

substitutes that restore, maintain or improve

tissue unction or patients.

New Medic Genetics Chie Focuses on

Expnsion nd lieong Genetic ServicesWhile the traditional purview o medical

genetics was inherited birth deects, says David

Sweetser, MD, PhD, MassGeneral Hospital

forChildrens (MGHfC) new chie o Medical

Genetics, the eld now encompasses inborn

errors o metabolism that lead to abnormalities

in children and adults. While some o these

disorders are detected and managed rom

birth, others may not be diagnosed until they

become problematic in adulthood. Identiying

these disorders early or even identiying the

risk during pregnancy counseling could help

protect individuals and amilies rom some o

the long-term eects.

We are one o the ew programs in the

country that can oer genetic services

throughout the liespan, rom prenatal

counseling and newborns and into adulthood,

says Dr. Sweetser. This comprehensive range

o services acilitates care and also leads

to improved understanding o the clinical

symptoms and management o underlying

genetic mutations. Individuals with a connective

tissue disorder called Maran syndrome, or

example, are at high risk or aortic rupture.

Following these patients with serial ultrasounds

allows or identication o the optimal time or

a surgical grat.

Under Dr. Sweetsers leadership, the division

will increase the scope o its services. These

eorts include consultations with other

specialists concerned about the possibility

o a genetic cause or their abnormalities,

prenatal genetic counseling, genetic counseling

and screening o amily members, and

multidisciplinary clinics or patients with rare

or more common syndromes, as well as a clinic

or adult patients with Down syndrome. The

division is expanding geographically as well,

with a new clinic at Mass General/North Shore

Center or Outpatient Care in Danvers, MA.

In collaboration with Massachusetts Eye and

Ear Inrmary, the division has created a new

sensory-neuro hearing loss multidisciplinary

clinic or patients with hearing loss attributable

to genetic causes, and a new partnership

with Shriners Hospital will ocus on genetic

components o inherited skeletal dysplasias and

abnormalities o bone metabolism. Dr. Sweetser

is recruiting new clinicians and scientists to

reduce wait time or appointments and to

uncover new genetic mutations that may point

the way to new targeted therapies.

Dr. Sweetsers research ocuses on the genetic

basis o cancer with the goal o nding novel

targeted therapies. His lab identied new

class o tumor suppressor gen

in leukemia that may be implic

more malignancies. The lab al

new compounds in model syst

published ndings relating to t

COX2 inhibitors to reduce risk

breast and prostate cancer. Ad

working with colleagues at the

and in local academic hospita

what aspects o whole genom

appropriate in the clinical sett

Dr. Sweetser received his MD

Washington University School

(Missouri), and completed a re

pediatrics at St. Louis Children

has also done ellowships in m

at Washington University and

Washington School o Medicinas well as a ellowship in ped

oncology at University o Wash

Medical School assistant proe

he joined the sta at MGHfC i

We are one of the few proin the country that can offegenetic services throughoulifespan, from prenatal couand newborns and into adu

David Sweetse

Since the results of this work were published in2008, interest in using decellularized cadavericorgans as a platform for organ development hasincreased dramatically.


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Page fourmassgeneral forchildren.org | 888-644-3211

Newsletter to Phys

massgeneral forchildren.org | 888-64

At MassGeneral Hospital forChildren

(MGHfC), perormance measurement is

viewed as an opportunity to improve in

meaningul areas o patient care. According

to Esther Israel, MD, director o Inpatient

Quality and Saety at MGHfC and associate

unit chie o Pediatric Gastroenterology and

Nutrition, quality and saety arent about

collecting and reporting data. Theyre an

opportunity to dene and respond to indicators

o quality that provide patients, amilies and

colleagues with the assurance that care is

sae, ecient and eective.

Initiatives at MGHfC ocus on the six aims

dened by the Institute o Medicine or

achieving quality in pediatric care: Saety,

eectiveness, eciency and equality, as well as

timely and amily-centered care. Individual

projects, identied by members o the care team

rom day-to-day experience on the foor, and by

the Quality and Saety Team using input rom

patient experience surveys, saety report orms

available on the inpatient units and other data

sources, build on MGHfCs long history o

service-wide and unit-based committees and

projects relating to quality and saety.

Improvements, says Kristen Solemina, MPH,

quality and saety manager or MGHfC, can

range rom the small and quickly accomplished

to larger-scale initiatives that are tackled by

teams o nurse managers, physicians, residents,child lie specialists, parent volunteers and

other sta. The quality and saety mindset

is integrated into our daily processes, says

Solemina. Everyone involved in the treatment

o our patients is always thinking about how

to do things better and provide the highest

quality o care.

Eorts in improving amily-centered care include

involving amilies with the management o the

patients disease. Five years ago, MGHfC moved

to bedside rounding. Rather than standing

outside the patients room to discuss the case,

the team gathers around the patient and amily

to discuss the treatment plan and include them

in the process. A second initiative aimed at

improving amily-centered care helps ensure

parents are kept updated during the pediatric

intensive care unit (PICU) admissions process.

Quity nd Cre Eorts Refect

Pssion or Cre ExceenceHaving your child admitted to intensive

care is incredibly stressul or parents, says

Phoebe Yaeger, MD, PICU physician, who

leads the project. Our goal is to do what

we can to alleviate that stress by connecting

regularly with the parents to let them know

how their child is doing.

Needle insertion pain and distress or children

undergoing IV insertion and phlebotomy is an

issue recently identied by inpatient comments.

One o the current Quality initiatives ocuses on

improving the management o needle insertions.

Representatives rom nursing, pain management

and Child Lie have joined orces with Dr. Israel

to create a brochure and teaching module on

reducing needle insertion pain and distress. The

materials, based on extensive literature review,

input rom Mass General clinical experts and

review o national best practices, include using

a local anesthetic to numb the injection site,

distraction techniques and holding techniques.

Inormation is disseminated to nurses,physicians, and other practitioners in the

hospital. A separate handout has been

developed or parents.

But beyond these specic projects, Dr. Israel

says, is the need or meaningul measures o

whether good care is being provided. The

Quality and Saety Team is working with each o

the subspecialties individually and with national

groups to dene and measure quality. At the

national level, MGHfC is participating in the

Value in Pediatrics (VIP) project, a national

collaborative aimed at benchmarking the care

o inpatients with bronchiolitis and creating

national care standards. The g

length o stay, age, readmissio

and bronchodilator and steroid

data or all bronchiolitis admis

goal o dening treatment gui

reduce readmissions nationall

Within MGHfC, eorts are und

establish baseline values or a

pediatric colonoscopy. The tea

establishing baseline values t

requency with which pre-proc

documented, the quality o the

whether the procedure was in

the ileum and, i so, whether i

initiative improves documenta

physicians as well as ensuring

diagnostic procedure.

According to Dr. Israel, these e

participation in other national

allow service areas within MG

the PICU and newborn intensiv

measure quality o care and m

and to compare perormance t

organizations. Using this ino

can identiy and celebrate our

says Dr. Israel. We can also

the care team in their number

Providing the best possible ca

Everyone involved

in the treatment

of patients is always

thinking about how

to do things better

and provide the

highest quality

of care.

Kristen Solemina, MPH

For more information about quality and safety initiatives at MGHfC, please v

massgeneralforchildren.org/qualityandsafety

A MGHfC child life specialist uses dist raction techniques to reduce needle insertion pain.


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massgeneral forchildren.org | 888-644-3

Cse review:kidney trnspntIn a case covered extensively by the Boston

Heraldearlier this year, Dicken Ko, MD, director

o Renal Transplant Program and surgical

director o the Kidney Transplant Program, and

other members o the m ultidisciplinary pediatric

organ transplant team, removed a kidney rom

39-year-old Tara Johnson and placed it in her

2-and-a-hal-year-old son, Andrew.

Andrews condition, a blockage in his urinary

tract, was diagnosed during a 19-week

ultrasound. Andrew was not expected to

survive, but the blockage improved, leaving

Andrews kidneys badly damaged. MGHfC

pediatric nephrologist, Avram Traum, MD,medical director o Renal Transplant Program,

began caring or Andrew the day he was

born, including the 11 days the inant spent

in the neonatal intensive care unit and

during a surgery to ully relieve the blockage

Andrew underwent when he was 3 days o

age. Following discharge, Andrew was seen

weekly by MGHfC transplantation specialists

and underwent nightly dialysis beginning at

10 months o age while his organ transplant

team waited or his body to be large enough to

receive an adult kidney. Finally, in March, ater

assistance rom the MGHfC nutritional team to

help Andrew reach the goal weight o 10 kg,

Andrew was ready.

This is a rare operation, says Dr. Ko. Only

about 140 kidney transplants done annually

involve an adult kidney placed in a child under

5. In all o New England, there are probably

only 30 o these procedures done each year.

Both parents were willing to donate but Taras

evaluation, perormed at the Mass General

Transplant Center, showed her to be the preerred

donor. Her procedure was done laparoscopically

in a room that adjoined the surgical suite whereAndrew awaited his new kidney. The kidney,

says Dr. Ko, began working almost immediately.

To date, Andrew has experienced no serious

complications, and continues to grow and

develop. He will be ollowed by his care team

and, ollowing adolescence, will likely require

another transplant.

Esing the compexityo creOne overarching goal or the MGHfC transplant

program, says Dr. Traum, is to ease the

complexity o care or patients and amily

members, both in the time leading up to and

during the procedure and in ollow-up care.

Having the same doctor beore, during and

ater transplant is reassuring or amilies, Dr.

Traum says. This goal is urther demonstrated

in the seamless transition o pediatric care

to adult care. According to M ass General

transplant surgeon Heidi Yeh,

patients and their amily memb

same specialists, in the same

the doctors already know one

communicate on a regular bas

to the ability to prevent or trea

issues that arise, this continuo

solutions or post-transplant ch

as compliance with medication

the team is working on a text m

application that will send remi

time take to take medications

is received, a second reminder

activity is logged so that mem

team can check compliance. T

will help middle adolescents le

responsibility or their medica

Reducing complexity helps com

pediatricians as well by provid

point o access or questions w

management o their transplan

or or reerrals to adult special

dermatologists and OB/GYNs

care o transplant patients. T

care involved in organ transpla

a wide variety o specialists w

eectively, even in a critical si

Dr. Traum. Our goal at MGHfC

this level o expertise to patien

reerring physicians in a single

single phone call.

For more information about

Transplant Program, please

massgeneralforchildren.org/

Newsletter to Phys

As a comprehensive service within Mass

General, MassGeneral Hospital forChildrens

(MGHfC) transplant program provides

multidisciplinary care or inants, children and

adolescents beore, during and ater organ

transplantation, including seamless transition

to adult care at the appropriate time. This care,

perormed by a team o experts that includes

pediatric specialists such as hepatologists,

nephrologists, urologists, gastroenterologists,

surgeons, psychiatrists, inectious disease

specialists, nutritionists and social workers,

as well as surgeons rom Mass Generals

Transplant Center. This care team is inormed

by the most current understanding o the

diseases process, diagnostic procedures,

treatment options and psycho-social

repercussions related to late-stage organ

disease in inants and children.

Patients reerred to our pediatric specialists

receive the ull complement o medical care

beginning with initial contact and extending

throughout their lietime, says Uzma Shah, MD,

medical director o the Liver Transplant Program

and director o the Hepatobiliary and Pancreatic

Program. For those children requiring organ

transplantation, we work in conjunction with the

transplant surgeons to ensure high-quality, well-

coordinated, patient- and amily-ocused care

beore, during and ater surgery.

This ully integrated relationship with Mass

Generals adult transplant program is a benetto patients as they age. It also provides

important benets or parent-to-child organ

donations. Unique among New Englands

pediatric organ transplant programs, MGHfC can

oer surgical and medical care to both donor

and recipient, allowing amilies to stay together

during this challenging time.

Cse review:iver trnspntIn 2004, a pediatric gastroenterologist at

Dartmouth-Hitchcock Medical Center in Lebanon,

New Hampshire, reerred a 5-month-old girl,

S, with congenital biliary atresia to MGHfC.

According to Martin Hertl, MD, surgical director

o the Liver Transplant Program, the child had

undergone a hepatoportoenterostomy, or Kasai

procedure, to connect her small intestine directly

to the liver at a N ew York Hospital, but the

procedure had ailed. Weighing only 5.4 kg, S

was gaining water weight at a rate o one pound

per week. Her pediatric end stage liver disease

(PELD) score, which uses lab values and growth

parameters to assess the risk or probability o

death within three months i the patient does not

receive a transplant, was 24 out o a 6-40 range.

On April 15, when Dr. H ertl rst met his new

patient, she had diarrhea and symptoms o an

upper respiratory inection (URI). Following a

consult with MGHfC pediatric inectious disease

specialists, the baby was admitted to MGHfC or

transplantation evaluation and URI treatment.

While she was released two days later, she

was on the operating table in just over a month.

This is ast, Dr. Hertl says, but evaluation can

be done within a day i neededa particular

advantage in children who have overdosed

on Tylenol. Under one roo, he says, we

can do everything required or transplantation

preparation and evaluation and operate the

next morning.

Both parents were willing to donate to their

child, but the ather, M, was the preerred

donor. On May 12, Dr. Hertl and his team

removed Ss liver while, in the next surgical

suite, a separate transplant team removed

segments 2 and 3 o Ms liver and delivered

them to Dr. Hertl, who successully placed them

in Ss body. Six days ater surgery, S suered

hepatic vein thrombosis, caused by ailure oa vein in the piece o liver received rom her

ather. This complication necessitated a second

transplant, this time o a hepatic vein received

rom a blood-group compatible cadaveric donor.

This procedure was also done at MGHfC. Ss

recovery was urther slowed by duodenal

peroration and candida peritonitis, which were

managed by members o the multidisciplinary

care team. In late July, S returned home to her

parents, returning to MGHfC only briefy or

treatment or viral gastroenteritis. Now a happy

7-year-old, S is seen annually in the MGHfC

transplant clinic where she is ollowed by a

team o specialists at each visit. B etween visits,

her care is managed by her pediatrician at

Dartmouth-Hitchcock.

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Peditric Trnspnttion: Single site, lifetime care

Page sixmassgeneral forchildren.org | 888-644-3211

Avram Traum, MD, with his patient,Andrew Johnson.

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