Stem cell transplants hold great potential for alleviating diseases that involve the blood or the immune system. Children born with severely deficient immune systems are at increased risk for infections. Without a working immune system, these children cannot guard against bacteria and viruses. Often times the only cure for primary immune deficiency disorders, bone marrow failure syndromes, metabolic disorders, and sickle cell disease, is a bone marrow transplant.
Our hematologists and immunologists work with transplant biologists to produce the best treatments possible for patients with these rare, non-malignant diseases. Seattle Cancer Care Alliance (SCCA) is the only place in the country to offer this level of collaboration for patients before and after transplant, and is part of the reason we are able to provide outstanding outcomes for our patients with:
- Severe aplastic anemia
- Fanconi anemia
- Shwachman-Diamond syndrome
- Diamond-Blackfan anemia
- Dyskeratosis congenital
- Congenital thrombocytopenia
- Kostman’s syndrome
- Myelodysplastic syndrome
- Juvenile idiopathic arthritis (JIA)
- Systemic lupus erythematosis
- Systemic sclerosis (scleroderma)
Human immunodeficiency virus (HIV)
Nonmyeloablative transplants (mini-transplants) have also been used for people who have human immunodeficiency virus (HIV) to (1) provide a healthy immune system to those whose immune system is not responding to the medicines, or (2) to treat HIV-positive people who develop leukemia or some other kind of cancer that can be treated with a transplant.
Very few facilities do transplants for people who are HIV positive because of concern that this procedure could allow the virus to grow uncontrollably. As HIV becomes a chronic, manageable condition that people live with for many years, the option of a transplant may be increasingly important for some infected people, which makes this an important area to explore and develop.
“The genetic conditions we are studying are rare, and patients with non-malignant diseases have unique underlying medical problems that require the expertise of multiple subspecialists,” says Lauri M. Burroughs, MD, a pediatric blood and marrow transplantation specialist at SCCA. Dr. Burroughs leads the Non-Malignant Board, a collaboration across eight institutions across the United States, which was established to provide a lasting forum for developing more patient-specific transplant treatment options.
The dilemma with transplants in non-malignant disease is that patients may avoid or delay a transplant (because of the risks associated with transplant) until their disease has progressed to the point that they don’t have other options.
“Unfortunately, if you wait too long, you miss the best opportunity for a successful transplant,” says Dr. Burroughs. “Many of these patients have underlying problems, like infections or organ disease. Lowering the toxicity associated with transplantation allows us to consider transplantation earlier in the course of the disease and gives more patients a chance to receive this potentially life-saving therapy and have a chance to live a long, productive life.”
The results of a long-term study1 by Jean Sanders, MD, (retired from Fred Hutchinson Cancer Research Center in 2012) confirm that transplantation is effective therapy for children with severe aplastic anemia and the majority of people transplanted for aplastic anemia during childhood grow up to become normal functioning adults and have productive lives. The unique long-term perspective of this study makes it extremely instructive in guiding the counseling of future pediatric patients undergoing transplantation for severe aplastic anemia.
Children with autoimmune diseases usually get adequate relief with standard treatments and do not need a transplant. “These diseases are not common, and most of them can be taken care of medically,” says Ann E. Woolfrey, MD. But a small number of children are not helped by regular treatments and may become crippled by their disease and have a poor quality of life. If all available medical therapies fail, then these children may be considered for a transplant.
"The goal of the transplant is to reset the immune system, to remove the autoreactive immune system and allow the immune system to rebuild in the body without attacking the body,” Dr. Woolfrey explains. “In Europe autologous transplants have been used to treat children with autoimmune diseases.”
Doctors at the Hutchinson Center pioneered the procedure in the United States, and have performed transplants for children and adults affected by autoimmune conditions.
1Sanders et al., Blood 2011; 118:1421-1428