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Spring 2009

masthead pediatric bmt newsletter

 

Transplant Program at SCCA Ranks First in One-Year Survival Rates

The Fred Hutchinson Transplant Program at Seattle Cancer Care Alliance (SCCA) was one of only two bone marrow transplant programs nationwide that consistently outperformed the expected one-year survival rate for unrelated donor transplants, according to an independent report that assessed 122 transplant centers over a five-year period.

 

Transplant Research at SCCA 

Ongoing clinical trials at SCCA and Seattle Children’s provide transplant patients with access to promising treatment protocols that may improve their outcomes. Read about:

Meet Lauri Burroughs, MD

Dr. Lauri Burroughs specializes in bone marrow transplantation at Seattle Cancer Care Alliance and Seattle Children’s for patients with childhood cancers and nonmalignant disorders. In particular, she has an interest in patients with primary immunodeficiency disorders, metabolic disorders, and marrow failure. Learn more about Dr. Burroughs’ outstanding work with pediatric transplantation.

 

Patient Referrals and Transplant Consultations

If you have pediatric patients who have diseases that could be treated with transplantation please refer them to the Transplant Program at SCCA for a consultation. For Online Referrals: http://www.seattlecca.org/physician-referral.cfm. Or call 1-800-804-8824.

 

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Transplant Program at SCCA Ranks First in One-Year Survival Rates

CIBMTR Report Assessed 122 Transplant Centers Over Five Years
 

The Fred Hutchinson Transplant Program at Seattle Cancer Care Alliance (SCCA) was one of only two hematopoietic cell transplant programs nationwide that consistently outperformed the expected one-year survival rate for unrelated donor transplants, according to an independent report that assessed 122 transplant centers over a five-year period.

  • The Center for International Blood and Marrow Transplant Registry (CIBMTR) examined the survival rates of 8,847 transplants performed to treat blood cancers at U.S. centers in the National Marrow Donor Program (NMDP) network.
  • The survival data was collected for the years 2002 through 2006 and patient outcomes were measured at the end of 2007.
  • In each of five years, the Hutchinson Center “over performed” in terms of expected one-year survival rates after transplant. Only one other transplant center in the nation matched that record.
  • The data was adjusted for several risk factors that could influence patient outcomes such as type of disease, its stage, patient age, and degree of donor cell match.

Transplant Research at SCCA

 

Transplants for Aplastic Anemia

“Aplastic anemia represents a non-malignant, but nevertheless highly fatal disease for which transplantation has been uniquely successful,” says Rainer Storb, MD, Head of the Transplantation Biology Program at Fred Hutchinson Cancer Research Center. “Transplantation for aplastic anemia has taught us a lot about transplantation for malignant diseases since transplantation-related issues can be looked at in ‘pure’ form without the constant threat of recurrent malignant disease which otherwise tends to muddle the statistical analyses.”

 

Current survival for aplastic anemia patients who receive a transplant is around 90 percent, according to Storb, who is principal investigator for a clinical trial for patients with severe aplastic anemia. Protocol 2054 relies on existing transplant strategies but limits the number of transplanted cells. The purpose is to determine whether limiting the number of cells will decrease mortality (now around 10 percent) by reducing infections and decrease morbidity by reducing chronic graft-versus-host diseases, which now affect about 25 percent of patients.

 

Protocol 2054 looks at Cyclophosphamide and Antithymocyte Globulin conditioning regimen for marrow transplantation from HLA-matched family members for severe aplastic anemia and the effect of marrow cell dose on chronic graft-vs.-host disease. (A multi-center trial. Principal Investigator: Rainer Storb, MD.

 

Non-myeloablative Transplants in Children
Another series of trials at SCCA focuses on non-myeloablative transplants in children who have related or unrelated HLA-matched donors and nonmalignant diseases, such as severe immunodeficiency diseases, inborn errors of metabolism, or congenital bone marrow failure syndromes.

 

Hutchinson Center doctors pioneered the use of the “mini bone-marrow transplant” (non-myeloablative) in nonmalignant diseases and have shown this procedure to be very successful in reducing the risks of transplant in young patients.

 

Mini-transplants, also called mixed chimeric transplants, “do not rely on high-dose cytotoxic therapy,” says Storb, “but rather rely on graft-versus-tumor effects for killing the patient’s cancer cells.” The low-dose conditioning regimen is administered to weaken the patient’s immune system so it accepts the donor’s stem cells.

 

Ideally, the transplanted cells engraft and a new immune system develops alongside the patient’s remaining immune system. For a time, the patient has two immune systems (mixed chimerism) before the patient’s system eventually disappears.

 

Studies for which your patients may be eligible include:

  • Protocol 1227: Induction of Mixed Hematopoietic Chimerism in Patients with Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow from an HLA-Matched Related Donor and Post-Transplant Immunosuppression with Cyclosporine and Mycophenolate Mofetil. PI: Lauri Burroughs, MD.
  • Protocol 2007: Hematopoietic Cell Transplantation for Treatment of Patients with Primary Immunodeficiencies and Other Nonmalignant Inherited Disorders Using Low-dose TBI and Fludarabine With or Without Campath®. PI: Lauri Burroughs, MD.
  • Protocol 2032: HLA-Haploidentical Related Marrow Grafts for the Treatment of Immunodeficiency and Other Nonmalignant Disorders Using Conditioning with Low-Dose Cyclophosphamide, 200 cGy TBI and Fludarabine; Postgrafting Immunosuppression Will Consist of a Single Low Dose of Cyclophosphamide, MMF and Tacrolimus. PI: Lauri Burroughs, MD.

Treating bone marrow failure and immune deficiency diseases 

Diagnosing a child with an immune deficiency disease is not always immediately straightforward. Immunologists, transplant biologists, and hematologists from Seattle Children’s and Seattle Cancer Care Alliance work together to diagnose and treat patients for these diseases. Often times, the only cure for a bone marrow failure disease or an immune deficiency disease is a bone marrow transplant. Our doctors are world-leaders in this treatment and have outstanding success treating children.
 
Most primary immune deficiency diseases begin in childhood, which means the doctors at Seattle Children's see a lot of these patients, and as a result have developed a regional expertise. Children’s is the referral center for children and adults with immune problems in the Pacific Northwest and home to the Center for Immunity and Immunotherapies and The Northwest Genome Engineering Consortium (NGEC) which brings together researchers at Seattle Children's Hospital Research Institute, Fred Hutchinson Cancer Research Center, and the University of Washington who are working to advance gene repair.
 
Children’s is building a gene sequencing service unlike any other in the region and serves as an international center for prenatal and neonatal testing, and for testing of adults to identify carriers of immune deficiency disorders.
 
Many bone marrow failure syndromes also begin to cause symptoms during childhood.  Children's is the referral center for children and adults with bone marrow failure in the Pacific Northwest.  Experts at Seattle Children's offer state-of-the-art diagnosis and treatment for aplastic anemia, Fanconi anemia, Shwachman-Diamond syndrome, Diamond-Blackfan anemia, congenital thrombocytopenia, Kostmann's syndrome, myelodysplastic syndrome, and other disorders of bone marrow dysfunction. Researchers at Seattle Children's Hospital Research Institute, Fred Hutchinson Cancer Research Center, and the University of Washington are working to optimize bone marrow transplant and gene therapy for treatment of these disorders.
 
In addition, immunologists work with transplant biologists to produce the best treatments possible for your patients with these rare, non-malignant diseases. This is the only place in the country to offer this level of collaboration for patients pre- and post-transplant. It’s how we are able to provide outstanding outcomes for your patients.

 

Lauri Burroughs, MD

Dr. Lauri Burroughs is a pediatric hematology/oncologist at Seattle Children’s, where she specializes in bone marrow transplantation for the treatment of patients with childhood cancers and nonmalignant disorders. She is a graduate of the University of Wisconsin School of Medicine and Public Health in Madison and received her residency training at Indiana University School of Medicine. She received her fellowship training at the University of Washington School of Medicine in Pediatric Hematology-Oncology. She is board certified in Pediatrics.

 

Dr. Burroughs’ clinical interests include primary immunodeficiency disorders, metabolic disorders, and bone marrow failure. Her research interests are in bone marrow transplantation, Hodgkin’s disease, and graft versus host disease. Read more.

 


Refer Patients and Transplant Consultations

If your patients are candidates for transplantation, please contact the Transplant Program at SCCA for a consultation with one of our physicians at 1-800-804-8824. Online referrals can be made at: http://www.seattlecca.org/physician-referral.cfm.

 


Adult Bone Marrow Transplant News

The SCCA Adult Bone Marrow Transplant News is a publication presenting the latest information on bone marrow transplant research at SCCA, providing up-to-date information for all health care professionals caring for transplant patients.

Pediatric Bone Marrow Transplant News

Read about important outcomes research at the Fred Hutch that may benefit your patients.

Clinical Trials Monthly

Each issue of Clinical Trials Monthly highlights several of the more than 200 clinical trials that are currently recruiting patients at SCCA.

The Leading Edge Newsletter

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.