Non-Oncologic Blood Disorders Patient & Family Handbook

A Family Guide to Non-Oncologic Blood Disorders

Some SCID patients require chemotherapy before their trans- plant. Chemotherapy will destroy cells in the bone marrow to make room for the donated cells and help prevent the child’s immune cells from attacking the donated cells. Some children with SCID may not need chemotherapy, especially if they have very few immune cells to start with. The use of chemotherapy depends on the severity of the immune deficiency, type of SCID, donor used, and recommendations made by your child’s doctor. Stem Cell Transplant Indications SCT provides SCID patients with a functioning immune system that is capable of protecting them from infections. It is a cure for the disease and is highly effective in the majority of patients.

• Gene therapy currently is being studied. Your medical team will determine the best treatment plan for your child. How successful is the treatment? Medications and IVIG can temporarily keep children with SCID healthy, but ultimately, almost all patients with SCID require stem cell transplant. To provide the best chances for success, a transplant is usually done using the bone marrow of a sibling. However, a parent’s bone marrow also is acceptable. Some children do not have family members who are suitable donors, in which case, doc- tors may use stem cells from an unrelated donor. The likelihood of a good outcome also is greater if the trans- plant is done early, within the first 3 months of life.

Notes from Your Child’s Medical Team

Severe Combined Immunodeficiency

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