Babies who are treated within the first few months of life have a higher chance of successful treatment. That’s because as time goes by, the baby is being exposed to more germs. Once an infection develops in a baby with SCID, it may reduce the effectiveness of treatment.
The current standard treatment for SCID is hematopoietic stem cell transplant, or HSCT, also known as bone marrow transplant. In HSCT, doctors take healthy blood-forming cells from a donor and put them into your baby. The blood-forming cells reproduce and provide your child with an immune system.
HSCT is a months-long process which requires a lengthy hospital stay. Once in the hospital, a baby first will undergo a number of tests and other procedures to get the baby ready for HSCT. Importantly, the doctors will determine who is the best match to provide blood forming cells for the HSCT. The baby will also be put on medicines to reduce the chance of infection.
Next the baby will undergo the treatment itself (HSCT). Following HSCT, the baby will undergo months of monitoring the immune system to determine if the HSCT was successful. With HSCT, there can be a number of complications and sometimes the HSCT needs to be repeated.
Your health plan might dictate which transplant center you can use or you might have a choice of where you would like the procedure to take place. Either way, there are crucial questions you should ask the transplant doctors so that you understand: the level of experience doctors have with HSCT, and specifically with SCID; the role isolation will play in the process; your responsibilities in the process; and the general length of time the transplant process will take to complete.
Gene therapy is another treatment, but it is still in the clinical trial stages. In clinical trials, doctors are still experimenting with how best to perfect the treatment so that it can be approved for regular use.
In gene therapy, doctors take the stem cells which have the incorrect copy of the gene out of the baby and put a corrected copy of the gene into those cells. They then put the cells back into the baby. The cells with the corrected gene make copies of themselves and create an immune system in the baby.
Currently, gene therapy is only available for X-linked SCID and Artemis SCID. Trials for ADA-SCID are on hold and updates will be made when they are available.
If your baby has ADA-SCID, you can also use PEG-ADA, an enzyme replacement therapy, administered to a child through injections that must be given on at least a monthly basis. The enzyme boosts the baby’s immune system. This treatment is only temporary and a more long-term treatment should be pursued.
As you explore treatments and begin your journey with SCID, be sure to keep paper copies of all of your baby’s test results and other health related materials. Recordkeeping is important for your baby’s future.
With early treatment, most children with SCID should be able to develop their own working immune system. Visit the SCID Treatment Overview page which includes links to in-depth information on Hematopoietic Stem Cell Transplantation (HSCT), Gene Therapy, and PEG-ADA Therapy.
Audrey and TJ Duggan explore the treatment options they chose for son, Dean.