Hematopoietic Stem Cell Transplantation (HSCT)

The current accepted treatment for SCID is hematopoietic stem cell transplant, or HSCT. HSCT, is a multi-step medical process that requires months of stay at the hospital. Though this time can be stressful, remember that it is only temporary.

Finding a Match

Once you’ve decided to treat your baby with HSCT, doctors will begin the search for a donor who best matches your child. The donor will provide hematopoietic stem cells that will be infused into your baby. The proteins on the cells from the donor have to match or come close to matching the proteins on the cells of your baby. A closer match increases the chances of the transplant being successful.

Donor sources include: a sibling matched donor; an unrelated matched donor; an unrelated umbilical cord blood donor; or a half-matched family donor. After a donor is found, he or she will have to undergo a stem cell donation procedure.

Pre-transplantation Steps

Your baby will undergo several pre-transplantation procedures before he or she gets the transplant. The first is an evaluation to make sure that your child is healthy enough to receive the transplant. If your baby is ready for the transplant, doctors will put your baby under general anesthesia to put a central line into his or her chest so that medications, fluids, and blood can be easily administered. The central line is an IV and is used when treatments have to be given over long periods of time, such as weeks and months.

Doctors administer immunoglobulin (antibodies) as supportive therapy before the HSCT. Immunoglobulin is protein preparation that contains antibodies from donated blood that helps the baby fight infection. The baby may also receive conditioning medicines, including chemotherapy. Conditioning eliminates the baby’s immune cells to make room for the new donor cells.

Transplant Day

On transplant day doctors infuse the donor cells into your baby through his or her central line. Your baby will be awake and you may be present during the procedure. The transplantation is similar to a blood transfusion and only takes a few hours at most.

Gene Therapy

Unlike HSCT which can be used to treat all types of SCID, gene therapy is tailored to treat a specific gene and is performed for special genetic types of SCID. In addition, gene therapy is still in clinical trials and is not yet a treatment approved by the Food and Drug Administration.

In gene therapy, hematopoietic stem cells containing the faulty gene are removed from the child and transferred to a lab. In the lab, doctors insert a working copy of the gene into those cells. Those cells are infused back into the child. The corrected cells make copies of themselves and provide the child with an immune system.

Advocacy

Finally, remember that advocacy in the hospital for your child is an important part of your role as a parent. Although doctors decide much of what happens during treatment, you should feel free to discuss the procedure with them. Also, there may be certain situations in which you will need to make sure that protocol is followed. For example, you will want to ensure that staff wash their hands and wear mask and gown when entering your child’s room, if that is what you and the doctor have discussed. Remember, it is your right to do the following when interacting with healthcare workers, including doctors:

• Ask questions. If you don’t understand the answers, ask them again.
• Bring up concerns you might have with procedures being performed on your child.
• Keep a notebook to record information about your child’s care, condition, and recovery.