For some children with GATA2 deficiency, treatment includes a transplant of blood-forming stem cells from a healthy person (stem cell transplant). It is also called a bone marrow transplant or hematopoietic cell transplant. Hematopoietic (him-at-oh-poy-EH-tik) stem cells are young cells that grow into blood cells.
This treatment replaces your child’s defective blood and immune cells with healthy ones. With normal levels of healthy infection-fighting blood cells, a child is no longer at risk for severe infections and skin conditions like warts.
A transplant also lowers the risk of a pre-leukemic condition called myelodysplastic syndrome, which is higher in kids with GATA2 deficiency. A transplant would not improve conditions that are not related to the blood or immune system. GATA2 deficiency can cause problems affecting the lymph system, hearing and thyroid. The transplant would not help with these.
Our Non-Malignant Transplant Program specializes in stem cell transplants for children with noncancer conditions. Some children with GATA2 deficiency are too sick to tolerate the powerful drugs or radiation (called conditioning) that are usually used to prepare their bodies for the transplant. Our team – led by Dr. Lauri Burroughs – has developed better ways to prepare them, called reduced-intensity conditioning. We continue to fine-tune conditioning treatments to improve survival and reduce complications.
We perform the transplants here at Seattle Children’s, working closely with our partner in the Seattle Cancer Care Alliance, Fred Hutch. Fred Hutch pioneered this lifesaving procedure and is one of the largest stem cell transplant centers in the world.