Stem cell transplants are a treatment option that can potentially cure Beta (β)‑Thalassemiaa genetic blood disorder that reduces or eliminates the production of β‑globin by addressing the disease at the genetic level. This treatment is also known as a bone marrow transplant (BMT).
What Happens During a Stem Cell Transplant?
Stem cell transplants aim to replace Blood Stem Cellsan immature cell that has the potential to develop into all types of blood cells, including white blood cells, red blood cells, and platelets that contain a change to the HBB gene with blood stem cells from a donor who does not have the change. During a stem cell transplant, new healthy stem cells are added to the body.
Before the new cells are added, a form of treatment called conditioning (which includes chemotherapy medicines) is used to clear out the existing bone marrow cells for the new stem cells. Over time, the goal of a stem cell transplant is for the healthy blood stem cells to make their way to the bone marrow and start to make new blood cells; this process is known as engraftment.
Stem Cell Transplants: What Should You Know?
In order to receive a stem cell transplant, an appropriate stem cell donor must be identified. This is done so that the transplanted cells have the best chance of being accepted by your body. For the most favorable outcome, the donor should be genetically similar to the person receiving the stem cells. Generally, siblings are the people who are most genetically similar. Only about 25–30% of people have a matched sibling donor.
If you want to learn more about this treatment option, it’s important to talk to your doctor about the benefits and potentially severe risks involved with stem cell transplants.
Although stem cell transplants have the potential to cure β-thalassemia, there are also serious risks associated with this type of transplant.
One major complication is a condition called Graft versus host disease (GvHD). In GvHD, the transplanted stem cells see the body's cells are foreign and so attack them. GvHD severity can vary, but for some it can be a potentially serious complication, and even life-threatening in some cases.