Knowing How Care Today Affects a Child’s Future

Our pediatric experts are focused on how treatments todayaffect growing bodies in the future.

Xander Speidel and mom Liberty

When Xander Speidel’s bone marrow stopped producing new blood cells, he and his mom, Liberty Speidel, travelled to Seattle from Kansas City toparticipate in the nation’s first clinical trial using Treosulfan – a drug that readies the body for transplant without the harsh side effects that medicallyfragile patients can’t always tolerate. After spending five months in Seattle, Xander, now 3, is back home with a brand-new immune system and thepromise of a normal life span.

One third of U.S. hospitals have nopediatric specialists on call, and about50% of emergency rooms care forfewer than 10 kids a day. It’s easy to seehow kids sometimes receive treatmentbetter suited for adults.

“Problems arise when providersaren’t used to seeing kids and use adultpatients as their point of reference,”says Dr. Chappie Conrad, part of theOrthopedics team at Seattle Children’s. “Younger children can’t describe theirsymptoms and are harder to examine,and clinicians can easily miss red flagsthat signal what care is required.”

Case in point: A recent drill conductedin 35 emergency departments (EDs)in North Carolina found that nearlyall failed to stabilize injured childrenproperly, to treat kids with a lifethreateningdrop in blood sugar correctlyor to order proper IV fluids. A differentstudy conducted nationally showedonly 6% of EDs have the supplies theyneed to safely treat kids.

Children don’t react to illness, injury,pain and medication in the same wayas adults. They require – and deserve – care designed specifically for themand provided by people speciallytrained to understand and meettheir needs.

National expertise

Drs. Shimamura and Burroughs

Drs. Akiko Shimamura (left) and Lauri Burroughs are national leaders in the care of kids with noncancerousblood diseases. They are dedicated to developing safer protocols for patients whosehealth makes it difficult for them to tolerate the intense drug and radiation conditioning that comesbefore a bone marrow transplant.

Liberty and Nathan Speidel weredays away from celebrating their 10thwedding anniversary when they gota phone call that literally knocked thewind out of them. Their 2-year-oldson Xander needed a bone marrow transplant.

As an infant, the boy was diagnosedwith Shwachman-Diamond syndrome (SDS) – a relatively rare condition thatdecreases one’s ability to digest foodand produce blood cells. Now the Kansas City–based family faced a majordecision and the Speidels wanted asecond opinion. Their SDS supportgroup (a Web-based network of familiesacross the country) provided unanimousadvice: call Dr. Akiko Shimamura atSeattle Children’s.

“I called the phone number onChildren’s website for the SDS registryand within two hours, Dr. Shimamuracalled me back,” recalls Liberty Speidel. “I was flabbergasted. I’d never even had aphone call from my own doctor and nowI was having a 45-minute conversationwith the national expert on SDS.”

A chance for life

Xander running

After a lifesaving bone marrow transplant,Xander Speidel, 3, has so much energy that his6-year-old sister wondered if he could go backto being sick!

Shimamura and her colleague Dr. Lauri Burroughs reviewed Xander’s records.They concurred that the toddler’sbone marrow was no longer effectively producing blood cells and platelets.Even more concerning, Xander’smarrow was starting to show changesconnected to the development ofleukemia. A transplant would correcthis marrow function and stop theleukemia in its tracks.

Burroughs and Shimamuraspecialize in treating children whohave non-cancerous blood diseases,such as marrow failure disorders,primary immune deficiencies andsickle cell disease. Bone marrowtransplants are often the only knowncure for kids with these conditions,but the organ damage and underlyinginfections caused by their illnessesmake it difficult for them to toleratethe powerful drug and radiationconditioning used to prepare theirbodies for transplant.

“We’re developing safer, less intenseconditioning regimens so more kidslike Xander can undergo potentiallylifesaving transplants and live healthy,productive lives,” says Burroughs.

Burroughs is running the nation’sfirst clinical trial for patients with noncancerousdiseases using the new drugTreosulfan as part of the transplantconditioning regimen. Several Europeanstudies have shown decreasedtransplant-related mortality withTreosulfan – particularly important formedically fragile patients like Xander.

“My favorite part of the treatment istelling parents, ‘you need to startsaving for college.’”

Dr. Lauri Burroughs

Speidel agonized over whether tohave Xander’s bone marrow transplantin Kansas City near family, church andhome, or to travel nearly 2,000 milesto Seattle, which had Shimamura,Burroughs and the clinical trial usingTreosulfan. “Once we made thedecision to go to Seattle, I felt so atpeace about it,” she remembers.

Xander and his mom came to Seattlein March 2013 to begin the conditioningregimen with Treosulfan prior to hisbone marrow transplant in May. Bymid-August, they were headed homewith new bone marrow, a brand new immune system and the promise ofa normal life span for Xander.

Although the clinical trial usingTreosulfan is still underway, Burroughsnotes that initial results are verypromising.

“My favorite part of this treatmentis telling parents, ‘you need to startsaving for college,’” she says.

Care for now and later

Jessica Stewart, mom, and Drs. Grady and Avansino

Jessica Stewart, 16, discusses next steps in her treatment with Drs. Rich Grady (left) and JeffAvansino. “My doctors think about my future as much as I do. Theygive me the right treatments now so I'll thrive as an adult,” says Jessica, who travels from Portlandfor her care.

Doctors at the Oregon hospital whereJessica Stewart was born so doubtedshe would survive that they secretlyasked her family’s minister to preparefor her inevitable passing.

Jessica was born with OEIS complex,a severe birth defect in which the bladderand intestines develop on the outsideof the body, the rectum is malformedand the spinal cord is tethered.

Being treated on the pediatric wingof an adult hospital for the first 10 yearsof her life was eye-popping for Jessicaand her family. She witnessed violentoutbursts in the ER, having an elderlyperson collapse in front of her froma heart attack at the lab, and beingforcibly restrained for blood drawsand other procedures.

After Jessica’s mom met Dr. Richard Grady, a pediatric urologist here atChildren’s, she began to wonder if Seattle might be a better place forher daughter’s ongoing care.

The difference was quickly apparent. “Before I came to Children’s, doctorswould say things like, ‘I’m so excitedto work with someone with youranatomy,’” muses Jessica, now 16. “At Children’s, no one treats me likea science experiment.”

Here, Jessica’s team – all expertsin the care of kids with complex pelvicanatomies – work together to makesure that she is healthy today andemotionally and physically primedfor college, career and relationships.

“At SeattleChildren’s, noone treats melike a scienceexperiment.”

Jessica Stewart, 16

“All of the specialists on theReconstructive Pelvic Medicine team – from general surgery,gastroenterology and gynecologyto urology, adolescent medicine andradiology – love helping patientsand families understand and navigatewhat can be very overwhelming medicalissues,” explains Dr. Jeff Avansino, apediatric general surgeon.

“My doctors think about my futureas much as I do,” says Jessica, abudding singer and actress. “They weregreat at dealing with what I needed asa child, but they’re also looking aheadto what I’ll need as an adult. I feel reallysafe in their hands.”

Pediatric Specialists Know Kids Best

Chappie Conrad on fractures

Orthopedic surgeon Dr. Chappie Conrad and histeam are improving care for kids with fractures.

Every year about 2,500 childrencome through our doors withfractures. The latest thinking on howbest to care for them continues toevolve – and orthopedists at SeattleChildren’s are helping establish newbest practices.

50 years ago, a child couldspend a year in the hospital healingfrom a hip fracture. Today, minimallyinvasive surgical treatments takeonly a day; yet, for a child to regaina lifetime of normal function,factors such as age, family historyof growth, the type of fractureand level of bone deformity mustbe considered before treatmentever begins.

“At every age and developmentalstage, kids’ bones are changing andit can take years of training to be ableto assess what treatment approachwill work best,” says Dr. ChappieConrad, head of the Orthopedicsteam 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 – cancut off blood supply to an adjacentartery. If the fracture is treated withoutrepairing the artery, the oversight coulddamage the arm and lead to amputation.

“We treat lots of kids with fractureswhose families have been to one, twoand sometimes three other placesbefore they come to us,” says Conrad. “If I could tell parents anything, I’d say‘see us first.’”

Published in Connection magazine, November 2013