What is the Non-Malignant Transplant Program?
Our Non-Malignant Transplant Program provides care for children who have a disease other than cancer that may be helped by a hematopoietic cell transplant. Transplants may greatly improve or cure several kinds of diseases. A transplant works by replacing the body's system for making blood cells, including the cells needed for a healthy immune system.
The three major types of non-malignant diseases we treat with transplants are:
- Primary immunodeficiency disorders. Children with these disorders get a lot of infections because they are missing one or more parts of their immune system. The goal of a transplant is to replace the body's incomplete immune system with a system that works.
- Bone marrow failure syndromes. Children with bone marrow failure are not able to make all the types of blood cells their body needs. The goal of a transplant is to replace the bone marrow that does not work with marrow that makes normal blood cells.
- Histiocytic disorders. Children with these disorders have immune cells that do not work as they should. Instead, their immune cells attack the bone marrow, liver and other organs. The goal of a transplant is to replace the cells that do not work with cells that work as they are supposed to and that do not attack the body.
We use transplants to treat other diseases, too, including:
- Metabolic diseases. Children with metabolic diseases are not able to break down or get rid of toxic substances in the body, like certain fats or proteins. This happens when certain kinds of enzymes are missing from cells. Enzymes are the proteins in the body that help to break down fats, proteins and other things in the body into substances that the body can use or process. The goal of a transplant is to give the child cells that contain the missing enzymes.
- Severe autoimmune diseases. In these diseases, the child's own immune system attacks their body. If medicines do not help, a transplant may be an option. The goal of the transplant is to destroy the immune cells that are attacking the body and replace them with cells that will work as they should.
Through our Non-Malignant Transplant Program, we diagnose and treat these and other conditions in children of all ages. Our caring team of experts prepares children and their families for the entire transplant process. We perform the transplant itself here at Seattle Children's. We also provide the many types of care children need for their short-term as well as long-term recovery.
What conditions do you treat?
We treat these non-malignant conditions using transplants:
- Immunodeficiency disorders
- Severe combined immunodeficiency (SCID)
- Wiskott Aldrich syndrome (WAS)
- X-linked hyper-IgM syndrome (XHIM)
- Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX)
- Chronic granulomatous disease (CGD)
- Common variable immune deficiency (CVID)
- Cartilage hair hypoplasia (CHH)
- Leukocyte adhesion deficiency (LAD)
- Chediak-Higashi syndrome (CHS)
- X-linked lymphoproliferative disorder (XLP)
- Bone marrow failure syndromes
- Histiocytic disorders
- Metabolic storage diseases
- Hurler syndrome (MPS 1)
- Maroteaux-Lamy syndrome (MPS IV)
- Cerebral X-linked adrenoleukodystrophy
- Metachromatic leukodystrophy (MLD)
- Globoid-cell leukodystrophy (GLD)/Krabbe disease
- Autoimmune diseases
- Juvenile rheumatoid arthritis (JRA)
- Systemic lupus erythematosus (SLE)
- Systemic sclerosis (SSC)
- Hematologic conditions
- Acquired immunodeficiency disease
How do you diagnose non-malignant diseases and treat with transplant?
The way we find out about what condition your child has and how to best treat them may be different depending on their symptoms or other medical issues. For instance, the first sign of an immunodeficiency disorder may be that your child gets sick often, gets uncommon infections or gets infections that are worse than usual. Or, if their bone marrow cannot produce enough blood cells, they may be fatigued, have paler skin than normal, bruise or bleed easily or get infections. Or they may seem to have an autoimmune disease that does not respond to treatment the way doctors expect. Our team will work to diagnose your child's condition based on all of their symptoms and their medical history.
Most of the time, these are the steps that our team will go through to diagnose your child:
- Take a detailed health history
- Carefully check your child for signs of illness
- Do blood tests to check the level and function of the various blood cells and immune cells
- Do genetic tests (on a sample of blood or other tissue) to see if there if it a condition that is passed down through family (genetic cause)
- Remove a sample of bone marrow (called bone marrow aspiration or biopsy) to find out if there are any problems with the marrow
- Sometimes, our team might have to do other tests. For example, we might have to get images of the inside of your child's body using a test like an ultrasound or CT scan. These use painless methods to take pictures of the inside of the body.
Your child's doctor will create a treatment plan that is best suited to your child's problems and needs. Depending on the illness that your child has, there may be a range of treatment options - transplant may be only one of them. For instance, for some immunodeficiency disorders, children can get monthly infusions of medicines called immunoglobulins. For bone marrow failure syndromes, sometimes medicines can be used to help the bone marrow produce more blood cells. For other conditions, transplant is the main treatment and possibly a cure. At Seattle Children's, we have experts that can provide the full range of treatments, based on what's best for your child.
What's special about the Non-Malignant Transplant Program at Seattle Children's?
Seattle Children's works with the Fred Hutchinson Cancer Research Center and Seattle Cancer Care Alliance (SCCA) to offer hematopoietic cell transplants. We also work with these partners to do research on non-malignant diseases so we can understand the causes and develop better treatments. The Hutchinson Center is a world leader in transplants. They have a great deal of experience in the kind of transplants that many patients with non-malignant diseases need.
Our non-malignant transplant team includes specialists in many areas: transplants, immunology, infectious disease, nephrology, gastroenterology and others. Our team members meet, work and talk with each other throughout the entire transplant process so your child gets the very best care for all aspects of their health. This continues after your child's transplant. The team also follows your child throughout the recovery process.
For immunodeficiency disorders, Seattle Children's has a large team of clinical immunologists in our Immunology Clinic and Center for Immunity and Immunotherapies with over 40 years of experience diagnosing and treating these conditions in children. These specialists are experts in understanding how the immune system works and caring for patients with unusual or serious infections or autoimmune problems. Seattle Children's also has an Immunology Diagnostic Laboratory. This means we can do complex tests offered at only a few places in the world. Results from these tests help us know how to best treat your child's disease. We are working on important new research to improve diagnosis and treatment of primary immunodeficiency disorders. We are also actively involved in organizations that provide support for patients and families, including the Immune Deficiency Foundation and the Jeffrey Modell Foundation.
For bone marrow failure disorders, Seattle Children's has a team of hematologists with expertise in diagnosis and treatment who care for children through our Bone Marrow Failure Program. Many of the specific bone marrow failure syndromes require a specialized transplant regimen to reduce the risk of severe side effects. Our transplant team has extensive experience transplanting patients with aplastic anemia and marrow failure syndromes. Our team pioneered many of the transplant regimens used now, and we are continuing to develop better transplant regimens designed specifically for patients with these conditions. We are also pursuing research to improve diagnosis and treatment. Seattle Children's hematologists participate in many national organizations providing promoting research and providing support to patients and families. Our hematologists are involved in the national registries for severe chronic neutropenia and for Shwachman-Diamond syndrome, both of which are based right here in Seattle.
Who needs the Non-Malignant Transplant Program?
This program is for children:
- Who have a known non-malignant disorder that needs treatment
- Who have symptoms of a non-malignant disorder, such as immunodeficiency or bone marrow failure, that needs diagnosis and treatment
- Who might have an immunodeficiency disorder, bone marrow failure syndrome or other non-malignant disease treated with transplant, but need a diagnosis and ongoing management
At Seattle Children's, we work with many children and families from the western United States and beyond. You may live as close as Seattle, or you may be traveling from far away to come to Seattle Children's. Either way, we can help you with things like getting ready for your child's clinic visit or hospital stay, getting schooling for your child and their siblings through the Hutch School, transportation, neighborhood services, billing and financial assistance, places to stay, and more.