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Spring 2014

A third of all pediatric bone marrow transplants (BMT) are now for non-malignant disorders. In this issue of Pediatric BMT Update, we explain why so many children with rare primary immunodeficiency diseases, marrow failure syndromes, or other blood disorders now benefit from hematopoietic cell transplantation.

Welcome from Scott Baker, Program Director

A third of all pediatric bone marrow transplants (BMT) are now for nonmalignant disorders.

In this issue of Pediatric BMT Update, we explain why so many children with rare primary immunodeficiency diseases, marrow failure syndromes, or other blood disorders now benefit from hematopoietic cell transplantation.

One key, as explained by Akiko Shimamura, MD, PhD, has been prompt patient referral for diagnostic and medical evaluation. This allows meticulous pretransplant planning as well as preemption of complications that might otherwise disqualify a patient.

The other game-changer, as described by Lauri M. Burroughs, MD, has been the introduction of lower toxicity conditioning regimens—such as the treosulfan protocol now available at Seattle Cancer Care Alliance (SCCA)—that allows more patients to safely undergo the procedure.

Of course, to access such specialized transplant protocols, families often must decide to travel for hundreds or thousands of miles and stay in a new city for many months to get the best care available for their child.

And that’s the other story in this issue: how our extraordinary SCCA services and support systems are ready to ease the whole arduous process for families—from first consultation, through hospitalization and recovery, and the transition back home. It’s never easy, but it can be comfortable—and ultimately worth the effort. I encourage families to explore all their transplant options and to partner with their primary hematology/oncology specialists and pediatricians to choose the transplant destination that is best for their child.

As always, I welcome inquiries—from any state or country—about our world-class programs for children with nonmalignant diseases.

K. Scott Baker, MD, MS
Director, Pediatric Blood and Marrow Transplantation
Director, Survivorship Programs, Fred Hutchinson Cancer Research Center and Seattle Children’s Hospital
Professor of Pediatrics, University of Washington School of Medicine

More Children with Rare Blood Disorders are Now Receiving Stem Cell Transplants

During the past decade, the number of bone marrow transplants (BMT) for non-malignant disorders has tripled (Figure 1). In fact, a third of all pediatric stem cell transplants are now done to treat these rare disorders.

Many of the forces driving this trend—better understanding of disease pathophysiology, earlier diagnosis, less toxic transplant conditioning regimens, more options for high-risk patients without matched donors, and improved follow-up care—were developed right here at Seattle Children’s and the Fred Hutchinson Bone Marrow Transplant Program at SCCA.

According to National Marrow Donor Program® (NMDP) / Be The Match® statistics, SCCA has performed over 3,000 unrelated transplants in adults and children, including a high volume of unrelated donor transplantations for non-malignant disorders:

  • Myelodysplastic and myeloproliferative disorders (528)
  • Histiocytic, plasma cell, or stem cell disorders (198)
  • Inherited erythrocyte/platelet or immune system disorders (66)
  • Inherited metabolic disorders (17)

Overall, SCCA has now performed 448 transplants for nonmalignant disease in children.

Figure 1

Why the Boom in Transplants for Nonmalignant Disease?

According to Akiko Shimamura, MD, PhD, pediatric hematologist-oncologist at Seattle Children’s, rising usage of stem cells relates in great part to earlier diagnosis and improved surveillance.

“Today, cutting-edge laboratory tests facilitate diagnosis and treatment planning,” she said. “And when a transplant is not needed immediately, we now partner with the patient’s hematologist to monitor the patient’s condition closely. If they develop complications requiring a bone marrow transplant, we can act swiftly with a customized protocol.”

Smarter surveillance beginning soon after a prompt referral leads to improved BMT timing and outcomes. “For example,” Shimamura said, “we have become much better at catching most Fanconi anemia patients before they develop leukemia, and that’s one reason more are benefitting from transplantation.”

Another factor driving up transplantation rates in rare conditions is the availability of safer and more customized transplant protocols. At the most experienced transplant centers, protocols are now finely tuned—in terms of timing, donor requirements, conditioning intensity and type, and risk management—based on the patient’s unique genetic and clinical risk factors.

“It’s not like one transplant protocol works for all patients,” Shimamura said. “Indeed, different reduced-intensity conditioning regimens may vary significantly with respect to their risks and benefits. Many patients with marrow failure disorders are ultrasensitive to specific toxicities and those with primary immunodeficiencies may come in with infections. This is where the experience of our large multidisciplinary team—hematologists, immunologists, subspecialists, researchers, and transplant experts—really pays off.”

Lower-toxicity transplant conditioning regimens are also opening new doors for many young patients with rare nonmalignant diseases. “Patients with rare nonmalignant diseases often have underlying problems, like infections or organ dysfunction that makes transplant very challenging and as a result, historically there was a high incidence in mortality,” said Lauri M. Burroughs, MD, pediatric hematologist-oncologist at Seattle Children’s and Fred Hutchinson Cancer Research Center and director of the Non-Malignant Disease Transplant Program at SCCA.

“We needed to develop better regimens that were safer for patients,” Burroughs said as she explained why she developed the first U.S. prospective clinical trial using a new drug called treosulfan as part of the conditioning regimen.

“Treosulfan is particularly appealing to use in the conditioning regimen for high-risk patients with nonmalignant diseases,” she said, “because it is effective in establishing the new marrow graft but with far less toxicity, which has translated into better survival.”

Lowered BMT Risk Leads to Changing Perspectives

Steadily improving transplant results (Table 1) have changed the thinking of many physicians caring for patients with rare bone marrow failure syndromes or primary immunodeficiency diseases.

“Back when I was starting my fellowship training,” Shimamura recalled, “the last thing I wanted to do was send my patient to transplant because the outcomes were often so poor. But now, with the encouraging results emerging with treosulfan and our other new protocols, I’m much more comfortable referring my patients to transplant if clinically warranted. Although the risks versus benefits of transplant must always be carefully weighed for each individual patient, transplant risks continue to decrease as new regimens are developed.”

“It’s still a rigorous procedure, no question, for these complicated patients with multiple medical issues,” she said. “But at a medical center with subspecialists who are experienced in these rare diseases, the outcomes tend to be much better.”

Burroughs agreed: “Because these diseases are so rare it is very important for patients to be seen at a center that is familiar with their child’s disease.”

Table 1


At Seattle Children’s, Burroughs and colleagues formed the first national non-malignant board composed of experts in the fields of transplantation, immunology, hematology, infectious disease, and other subspecialties. This board, with top specialists from ten different U.S. centers, is entirely dedicated to the care of patients with rare non-malignant diseases.

“I wanted patients with rare non-malignant diseases to get the same dedicated attention as patients with cancer,” Burroughs said. “The board discusses every new patient with a rare non-malignant disease and we evaluate the best treatment options. The board has definitely improved patient care.”

Families More Willing to Travel for the Cure

The families of children newly diagnosed with these rare conditions are also recognizing the value of partnering with an experienced transplant center. Increasingly, according to Shimamura, families are sharing stories and information on websites of patient advocacy groups such as the Fanconi Anemia Research Fund (www.fanconi.org), the Shwachman-Diamond Syndrome Foundation (www.shwachman-diamond.org), and the Aplastic Anemia & MDS International Foundation (www.aamds.org).

The result, she said, is that more families are willing to put up with the inconvenience of traveling out of their region to get the most expert care and access to the special protocols they want for their child.

With its history as the destination pediatric transplant center servicing a huge geographic area of Alaska and the Northwestern U.S., the Fred Hutchinson Bone Marrow Transplant Program at SCCA is uniquely equipped to help families coming from afar for transplant therapy. For children with non-malignant disorders, there is even a dedicated nurse facilitator who assists physicians and families with the practical logistics of insurance, transportation, housing, and school. Post-transplant care programs at SCCA are also well coordinated, with long-term monitoring in close partnership with the patient’s own hematology specialist and with special programs for graft-versus-host disease prevention and clinics for late-effects, survivorship, and quality of life.

As treatment options for children with rare blood disorders improve, and as more of these children grow into adulthood, the role of SCCA as a destination for advanced therapy and a resource for follow-up care will continue to grow.

SCCA’s Non-Malignant Transplant Program Offers Ample Support for Families and Referring Physicians

When families uproot their lives to spend three months away from home for medical treatment, it is never easy. But when parents choose the pediatric bone marrow transplantation (BMT) specialists at SCCA, Seattle Children’s and Fred Hutchinson Cancer Research Center to offer the best treatment for their child’s non-malignant disease, an array of support services are available to make that period as comfortable as possible. An infrastructure of support ensures that referring physicians are kept in the loop so they can be ready to assist the family upon their return home.

The support available to families begins well before the tough decision to come to Seattle is ever made. Here’s why: when children are diagnosed with a non-malignant disease that may require hematopoietic cell transplantation, the family often has a window of opportunity to evaluate different transplant centers. This is very different from the sense of transplant urgency seen in most children diagnosed with cancer.

“Some patients with marrow failure or immunological diseases are monitored for years as they wait for the trigger that says it’s time to get a transplant,” said Kathleen McGregor, MN, MPH, of the Blood and Marrow Disorders and Immunological Disease Programs at Seattle Children’s.

“Others may be waiting for flu season to end. Whatever the reason, these families often have time before the transplant is needed, and increasingly they are using this time to research transplant centers, join chat groups, ask questions, and fully explore their options.”

A Consistent Presence for Parents in Research Mode

McGregor said this “shopping” of transplant centers makes perfect sense and that’s one main reason her dual position as non-malignant transplant coordinator was created: to make sure families coming to SCCA for a first transplant consultation have a consistent presence available to answer logistical questions, ensure they see the right people, and give them a tour of the new inpatient unit.

Parents typically consult with a transplant specialist to discuss the treatment options and a social worker to learn about all the practical issues such as where they’ll stay for three months, schooling at the fully accredited K-12 Hutch School, transportation, neighborhood services, and financial assistance (Table 2).

“Then what most parents really want is to see the hospital where they might be spending a month after the transplant,” McGregor said. “It’s a lengthy period of time to be away from your support system.”

McGregor takes families on these visits to the newly opened wing at Seattle Children’s. She explained that most families are reassured when they see the comfortable rooms with bedside accommodations where parents can stay overnight, as well as the quiet surrounding neighborhood with parks and the nearby housing options—including Ronald McDonald House right across the street.

After this initial visit, McGregor stays in touch with families, answering questions and helping to coordinate any further testing.

The Transplant, Then Home

Once families arrive in Seattle, McGregor makes rounds several times per week—ensuring that parents understand their child’s clinical care and that they are getting all they need from social workers, the palliative care team, teachers, counselors, and volunteers.

“I’m a familiar face,” she said. “They see me consistently throughout the transplant inpatient and outpatient phases.”

After engraftment, a child and caregiver usually leave the hospital but find housing within a 30-minute drive of Seattle Children’s. McGregor and her social work colleagues assist families in choosing from the list of high-quality housing options, including the nearby Ronald McDonald House, Pete Gross House, and SCCA House.

As they recover, the children come back to the posttransplant outpatient clinic to see their pediatric specialists and transplantation doctors and they continue tapping resources such as the Hutch School and support groups. They also start preparing for their return home with the help of one-on-one counseling, detailed guidebooks, and a personalized care plan.

When recovery is complete, typically after 100 days, the family moves back home and returns to the care of their primary hematologist/oncologist or pediatrician.

The Long-Term Follow-Up Program

The Pediatric Long-Term Follow-up (LTFU) Clinic at SCCA helps families transition back home and manage any long-term transplant effects. This program is part of SCCA’s overall LTFU Program, which monitors the recovery of more than 6,000 patients and is one of the most respected post-transplant care programs in the world.

The Pediatric LTFU Clinic for nonmalignant disease is directed by Lauri M. Burroughs, MD, pediatric hematologist-oncologist and director of the Non-Malignant Disease Transplant Program at Seattle Children’s and the Fred Hutchinson Cancer Research Center.

“Following transplantation, patients need close monitoring to make sure that they are doing OK,” Burroughs said. “We developed the Pediatric LTFU Clinic in order to provide long-term care following transplantation for patients in Seattle as well as when they return home.”

The Pediatric LTFU Clinic also works with each patient’s referring physician to help coordinate care once the patient returns home. “We want the transition home to be as seamless as possible and we want families and referring physicians to feel supported,” Burroughs said.

The LTFU office provides a direct line of communication for families and their local physicians. They can call anytime for advice, and the LTFU webpage provides a wealth of information about posttransplant care—including protocols for vaccination and photos of how to recognize graft-versus-host-disease (GVHD). In cases where GVHD becomes an issue, the local physician receives immediate support from LTFU specialists on how to monitor the condition.

Most patients return to SCCA once a year for a routine appointment. In many cases, the patient’s primary specialist can perform tests in advance to shorten the time required for this checkup.

Finally, the pediatric LTFU staff also track patients to study late effects, quality of life, and survivorship. As bone marrow transplants for non-malignant conditions become even more common, this long-term monitoring will be the key to improving transplant outcomes—and making difficult choices for parents a bit easier.

Through the whole transplantation process—from their first phone call to their transition home—parents of children with nonmalignant diseases can count on substantial support from SCCA.

Referring physicians or parents can contact Kathleen McGregor, MN, MPH, directly with any questions about bone marrow transplantation for non-malignant disease.

Phone: (206) 987-7021
Email: kathleenmcgregor@seattlechildrens.org

Table 2

Services Available at Seattle Children’s

Social Work

  • Counseling, education, advocacy, interpreters
  • Navigation of health care system
  • Help in adjusting to illness and coping
  • Guest Services
  • Assistance in finding and arranging nearby housing

Financial Counseling Programs

  • Information and applications for financial assistance
  • Help in understanding bills and insurance processing
  • Assistance in claiming medical necessity for out-of-network care

Family Resource Center

  • Education about diseases, treatments, hospital services, etc
  • Stress relief for parents and caregivers
  • Information on housing, restaurants, transportation
  • Access to computers, internet, wireless, phones, fax, copier
  • Reserved kitchen, showers, laundry, storage
  • Mobile in-room resource cart with coffee, newspapers, books, toiletries

Hutch School

  • Up-front assessments, consultations, and recommendations
  • Fully accredited kindergarten through high-school
  • Classroom teaching and/or individual tutoring
  • Programs for preschool children with special needs
  • Programs for family members (siblings)
  • Special arts programs and summer sessions
  • Discharge coordination with the child’s teachers
  • Rarely do children go home behind in their schoolwork

Other

  • Child-Life trained volunteers offering companionship, games, and crafts for pediatric inpatients and their siblings
  • Volunteer network for help in transportation, social activities, companionship
  • Spiritual and emotional care for all
  • Caregiver preparation and training
  • Free van service between SCCA facilities and to and from airport/ferry/bus

From a Library Card to the Latest Cure

In recalling one family’s recent visit, Kathleen McGregor, MN, MPH, described how help with the little things can create a home-like feel that opens the door to the big thing—in this case, access to a research protocol with treosulfan that provided an effective reduced-intensity treatment for their child.

“We recently had a family from the Midwest who researched their child’s marrow failure and reached out to Dr. Shimamura. Their local hematologist sent information to us and then the family came out and met with Dr. Shimamura and Dr. Burroughs. They heard about the treosulfan protocol and made the decision to come out here.

It was quite a distance for them, so we definitely had some long discussions…about internet access, closet space, signing up for the local library to get books, nearby areas to walk. All the things a mom and dad want to know to feel a little more comfortable. Now they’ve had the transplant and returned back home and their local hematologist is managing them quite well.

It’s always a difficult decision to travel so far. But if a family decides we have the protocol that is the best for them, then we will do everything it takes to make them feel comfortable coming here.”


Adult Bone Marrow Transplant News

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Pediatric Bone Marrow Transplant News

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Each quarterly Leading Edge newsletter will highlight a new topic to give you the latest news on leading-edge therapies that SCCA physicians are offering.