Epidermolysis Bullosa (EB) Center
Bone Marrow Transplant
Bone marrow transplant is a complex medical procedure and is not appropriate for every patient. It is a multi-step process that replaces the blood-making cells in a patient’s body with cells from a donor who does not have the genetic predisposition for epidermolysis bullosa. As the donor cells take up residence in the patient’s bone marrow, they produce cells that are capable of making the missing protein that helps hold the layers of the skin together.
To prepare for a bone marrow transplant, a donor must be identified (related, unrelated matched, or cord blood). Then chemotherapy and low-dose total body irradiation are used to make room for the donor cells in the patient’s bone marrow. This effectively wipes out the patient’s immune system. The transplant itself consists of cells being infused through an IV, much like a blood transfusion. Afterwards, the patient is hospitalized for around 4-6 weeks while the donor cells engraft in the bone marrow and begin producing healthy cells. Once the patient’s cell production has normalized, they are released, but due to their lowered immunity, they need to remain fairly isolated and with 30 minutes of the hospital. Through this recovery period, patients are closely monitored with frequent clinic appointments until 100 days after transplant or their immune system recovers.