Immune cells are your body’s arsenal against infection. There are different types of immune cells, each with its own specialized job. Disorders that reduce the numbers of normal, functioning white blood cells and other immune cells impair your child’s immunity and make him or her more prone to infections. At MSK Kids, our pediatric hematologists have treated every type of white blood cell disorder and primary immunodeficiency in children. Our team performs comprehensive testing to identify the problem and designs the most effective plan of care for your child.
We often treat children with white blood cell and primary immunodeficiencies using medication. But the only known cure for these ailments is an allogeneic stem cell transplant — stem cells donated by a healthy donor to replace a child’s ineffective bone marrow. At MSK Kids, we have successfully performed transplants where we remove certain T cells from the donated marrow to reduce the risk of complications after transplant. We also give chemotherapy instead of radiation therapy before the transplant to avoid the high risks of radiation in young children. Because children treated for these disorders may have an increased risk of certain cancers later, your child’s care includes lifelong monitoring and the expertise of other specialists to address any other health problems that may arise.
Chronic granulomatous disease (CGD) is a rare inherited disorder that results in low levels of phagocytes — a type of white blood cells called neutrophils that engulf foreign bodies such as bacteria and fungi, eating and digesting them and keeping them from causing harm. Children who have low phagocyte levels due to CGD are more prone to bacterial and fungal infections. They may also develop “granulomas” — areas of inflamed tissue — most often in the digestive and urinary tracts. They may be prone to abscesses (tender masses), swollen lymph nodes, persistent diarrhea, and a chronic runny nose.
The most common treatments for CGD are antibiotic and antifungal medications to treat and prevent infections. Some children benefit from treatment with injections of a medication called interferon gamma-1b, which can reduce the frequency of severe infections by boosting the body’s immune response against infection. For severe cases not helped by medication, MSK Kids offers stem cell transplantation, the only cure for CGD.Back to top
In children with cyclic neutropenia — a rare disorder that may be inherited or acquired later in life — the level of white blood cells called neutrophils fluctuates and sometimes drops to very low levels. When the number of neutrophils is too low, your child may experience infections, including fever, skin infections, mouth sores, and sore throat. Sometimes we can treat these infections with antibiotics. When your child’s neutrophil count is too low, we may prescribe G-CSF to boost white blood cell counts. This medicine makes the bone marrow produce blood stem cells that mature into functioning white blood cells. In severe cases when medical treatment is not sufficient, a child may be a candidate for a stem cell transplant.Back to top
Leukocytes are a type of white blood cell. In children with leukocyte adhesion deficiency, a rare inherited primary immune deficiency, leukocytes lack a certain protein on their surfaces that they need to work properly. As a result, they cannot fight off bacteria and other invaders the way healthy leukocytes do, and children are prone to infections. Children with leukocyte adhesion deficiency may have gum, skin, and muscle infections and slow-healing wounds.
If your child has leukocyte adhesion deficiency, we may prescribe antibiotic treatments, sometimes continuously, to prevent infections. Your child may also have periodic transfusions of healthy white blood cells called granulocytes. We treat severe leukocyte adhesion deficiency with a stem cell transplant from a donor. This is the only known cure for this disorder and a lifesaving treatment for young children who cannot be helped with medication and transfusions alone. Your MSK Kids team will let you know the best course of care for your child.Back to top
Myeloperoxidase is an enzyme that helps white blood cells called neutrophils fight off bacteria and other causes of infection. An inherited deficiency in myeloperoxidase is actually fairly common, but rarely causes problems unless someone is also diabetic. Children with diabetes and myeloperoxidase deficiency (MPO deficiency) are more prone to infections caused by bacteria and fungi. If this is the case for your child, the MSK Kids team will perform a full evaluation and make recommendations about the best treatment, which may include medications. We provide long-term care for as long as your child needs, bringing together hematologists, endocrinologists, and other healthcare providers necessary to achieve and maintain the best possible health.Back to top
Children with severe chronic neutropenia (SCN) develop serious infections as early as infancy or later in life. This inherited disorder is caused by a genetic mutation and leads to very low levels of white blood cells called neutrophils. Severe chronic neutropenia causes skin infections, mouth ulcers, gum disease, and sinus infections and is usually found when a child is very young.
We use a drug called granulocyte colony-stimulating factor, or G-CSF, to treat SCN. It makes the bone marrow produce blood stem cells that mature into functioning white blood cells. Memorial Sloan Kettering researchers developed G-CSF, which has been used around the world to raise white blood cell counts in people receiving chemotherapy. It is also effective in many children with low white blood cell counts. If your child’s SCN persists despite G-CSF treatment, we may perform a stem cell transplant.Back to top