Our pediatric experts are focused on how treatments today
affect growing bodies in the future.
When Xander Speidel’s bone marrow stopped producing new blood cells, he and his mom, Liberty Speidel, travelled to Seattle from Kansas City to
participate in the nation’s first clinical trial using Treosulfan – a drug that readies the body for transplant without the harsh side effects that medically
fragile patients can’t always tolerate. After spending five months in Seattle, Xander, now 3, is back home with a brand-new immune system and the
promise of a normal life span.
One third of U.S. hospitals have no
pediatric specialists on call, and about
50% of emergency rooms care for
fewer than 10 kids a day. It’s easy to see
how kids sometimes receive treatment
better suited for adults.
“Problems arise when providers
aren’t used to seeing kids and use adult
patients as their point of reference,”
says Dr. Chappie Conrad, head of the
Orthopedics team at Seattle Children’s.
“Younger children can’t describe their
symptoms and are harder to examine,
and clinicians can easily miss red flags
that signal what care is required.”
Case in point: A recent drill conducted
in 35 emergency departments (EDs)
in North Carolina found that nearly
all failed to stabilize injured children
properly, to treat kids with a lifethreatening
drop in blood sugar correctly
or to order proper IV fluids. A different
study conducted nationally showed
only 6% of EDs have the supplies they
need to safely treat kids.
Children don’t react to illness, injury,
pain and medication in the same way
as adults. They require – and deserve – care designed specifically for them
and provided by people specially
trained to understand and meet
Drs. Akiko Shimamura (left) and Lauri Burroughs are national leaders in the care of kids with noncancerous
blood diseases. They are dedicated to developing safer protocols for patients whose
health makes it difficult for them to tolerate the intense drug and radiation conditioning that comes
before a bone marrow transplant.
Liberty and Nathan Speidel were
days away from celebrating their 10th
wedding anniversary when they got
a phone call that literally knocked the
wind out of them. Their 2-year-old
son Xander needed a bone marrow transplant.
As an infant, the boy was diagnosed
with Shwachman-Diamond syndrome
(SDS) – a relatively rare condition that
decreases one’s ability to digest food
and produce blood cells. Now the Kansas City–based family faced a major
decision and the Speidels wanted a
second opinion. Their SDS support
group (a Web-based network of families
across the country) provided unanimous
advice: call Dr. Akiko Shimamura at
“I called the phone number on
Children’s website for the SDS registry
and within two hours, Dr. Shimamura
called me back,” recalls Liberty Speidel.
“I was flabbergasted. I’d never even had a
phone call from my own doctor and now
I was having a 45-minute conversation
with the national expert on SDS.”
A chance for life
After a lifesaving bone marrow transplant,
Xander Speidel, 3, has so much energy that his
6-year-old sister wondered if he could go back
to being sick!
Shimamura and her colleague Dr. Lauri Burroughs reviewed Xander’s records.
They concurred that the toddler’s
bone marrow was no longer effectively producing blood cells and platelets.
Even more concerning, Xander’s
marrow was starting to show changes
connected to the development of
leukemia. A transplant would correct
his marrow function and stop the
leukemia in its tracks.
Burroughs and Shimamura
specialize in treating children who
have non-cancerous blood diseases,
such as marrow failure disorders,
primary immune deficiencies and
sickle cell disease. Bone marrow
transplants are often the only known
cure for kids with these conditions,
but the organ damage and underlying
infections caused by their illnesses
make it difficult for them to tolerate
the powerful drug and radiation
conditioning used to prepare their
bodies for transplant.
“We’re developing safer, less intense
conditioning regimens so more kids
like Xander can undergo potentially
lifesaving transplants and live healthy,
productive lives,” says Burroughs.
Burroughs is running the nation’s
first clinical trial for patients with noncancerous
diseases using the new drug
Treosulfan as part of the transplant
conditioning regimen. Several European
studies have shown decreased
transplant-related mortality with
Treosulfan – particularly important for
medically fragile patients like Xander.
“My favorite part of the treatment is
telling parents, ‘you need to start
saving for college.’”
Speidel agonized over whether to
have Xander’s bone marrow transplant
in Kansas City near family, church and
home, or to travel nearly 2,000 miles
to Seattle, which had Shimamura,
Burroughs and the clinical trial using
Treosulfan. “Once we made the
decision to go to Seattle, I felt so at
peace about it,” she remembers.
Xander and his mom came to Seattle
in March 2013 to begin the conditioning
regimen with Treosulfan prior to his
bone marrow transplant in May. By
mid-August, they were headed home
with new bone marrow, a brand new immune system and the promise of
a normal life span for Xander.
Although the clinical trial using
Treosulfan is still underway, Burroughs
notes that initial results are very
“My favorite part of this treatment
is telling parents, ‘you need to start
saving for college,’” she says.
Care for now and later
Jessica Stewart, 16, discusses next steps in her treatment with Drs. Rich Grady (left) and Jeff
Avansino. “My doctors think about my future as much as I do. They
give me the right treatments now so I'll thrive as an adult,” says Jessica, who travels from Portland
for her care.
Doctors at the Oregon hospital where
Jessica Stewart was born so doubted
she would survive that they secretly
asked her family’s minister to prepare
for her inevitable passing.
Jessica was born with OEIS complex,
a severe birth defect in which the bladder
and intestines develop on the outside
of the body, the rectum is malformed
and the spinal cord is tethered.
Being treated on the pediatric wing
of an adult hospital for the first 10 years
of her life was eye-popping for Jessica
and her family. She witnessed violent
outbursts in the ER, having an elderly
person collapse in front of her from
a heart attack at the lab, and being
forcibly restrained for blood draws
and other procedures.
After Jessica’s mom met Dr. Richard Grady, a pediatric urologist here at
Children’s, she began to wonder if Seattle might be a better place for
her daughter’s ongoing care.
The difference was quickly apparent.
“Before I came to Children’s, doctors
would say things like, ‘I’m so excited
to work with someone with your
anatomy,’” muses Jessica, now 16.
“At Children’s, no one treats me like
a science experiment.”
Here, Jessica’s team – all experts
in the care of kids with complex pelvic
anatomies – work together to make
sure that she is healthy today and
emotionally and physically primed
for college, career and relationships.
one treats me
like a science
“All of the specialists on the
Reconstructive Pelvic Medicine team – from general surgery,
gastroenterology and gynecology
to urology, adolescent medicine and
radiology – love helping patients
and families understand and navigate
what can be very overwhelming medical
issues,” explains Dr. Jeff Avansino, a
pediatric general surgeon.
“My doctors think about my future
as much as I do,” says Jessica, a
budding singer and actress. “They were
great at dealing with what I needed as
a child, but they’re also looking ahead
to what I’ll need as an adult. I feel really
safe in their hands.”
Pediatric Specialists Know Kids Best
Orthopedic surgeon Dr. Chappie Conrad and his
team are improving care for kids with fractures.
Every year about 2,500 children
come through our doors with
fractures. The latest thinking on how
best to care for them continues to
evolve – and orthopedists at Seattle
Children’s are helping establish new
50 years ago, a child could
spend a year in the hospital healing
from a hip fracture. Today, minimally
invasive surgical treatments take
only a day; yet, for a child to regain
a lifetime of normal function,
factors such as age, family history
of growth, the type of fracture
and level of bone deformity must
be considered before treatment
“At every age and developmental
stage, kids’ bones are changing and
it can take years of training to be able
to assess what treatment approach
will work best,” says Dr. Chappie
Conrad, head of the Orthopedics
team at Children’s.
2,500: Approximate number of fractures treated each year at Seattle Children’s
For example, a simple elbow fracture – common among kids under 10 – can
cut off blood supply to an adjacent
artery. If the fracture is treated without
repairing the artery, the oversight could
damage the arm and lead to amputation.
“We treat lots of kids with fractures
whose families have been to one, two
and sometimes three other places
before they come to us,” says Conrad.
“If I could tell parents anything, I’d say
‘see us first.’”
Published in Connection magazine, November 2013