Your immune system is a powerful tool against infection. But sometimes the immune system goes into overdrive, working against the very body it was designed to protect. These “autoimmune” disorders can affect blood cells. At MSK Kids, our hematologists have experience treating rare, severe autoimmune blood cell disorders that don’t respond to treatment. We carefully assess your child’s health with tests, look for the effects of the disorder on his or her life, and combine our knowledge as a team to design the best plan of care for your child.
Immune thrombocytopenia (ITP) happens when the immune system attacks and destroys platelets, the blood cells responsible for clotting. As a result, there are not enough platelets in the body to control bleeding. Children with ITP have increased bruising, may bleed from the gums or nose, and have blood in the urine or stool. Girls who are getting their period and who have ITP may have very heavy periods. If your child has ITP, we will monitor the platelet count with the goal of getting it up to a safe level that reduces the risk of serious bleeding. We can accomplish this with a variety of medications. Some children may also have the spleen removed, since the spleen is responsible for normal platelet destruction. If these treatments don’t work, we may consider certain chemotherapy drugs. In rare cases, we may consider stem cell transplantation. This gives your child stem cells from a healthy donor to promote the growth of a healthy blood system.
Autoimmune hemolytic anemia is when the immune system destroys red blood cells faster than the bone marrow can make new ones. Children with this rare disorder may look pale and feel weak. They may also have shortness of breath, headaches, jaundice (yellowed skin and eyes), dark brown urine, nausea, and vomiting. We may treat your child with transfusions of red blood cells and medications. If these approaches are not enough, we may remove the spleen, and in rare cases, perform stem cell transplantation.
Evans syndrome is a very rare disorder in which the immune system attacks and destroys all types of blood cells, including red and white blood cells and platelets. Children with Evans syndrome may appear pale and weak. They may also have headaches, jaundice (yellowed skin and eyes), dark brown urine, and rashes, and be prone to bleeding and infections.
Evans syndrome is challenging and requires the input and expertise of multiple specialists. At MSK Kids, when our team sees a child with these symptoms, we first rule out other possible causes. Then we design a course of treatment to enhance your child’s well-being, which may include a combination of medications, immunoglobulin to support immunity given through an IV, the chemotherapy drug rituximab (Rituxan®), and other drugs to dampen the increased immune response. Some children benefit from removal of the spleen, and stem cell transplantation may be a possible treatment for others. We’ll determine the most appropriate treatment for your child and explain what you can expect. While Evans syndrome cannot be cured, there are steps we can take to improve your child’s health.