Stem Cell Transplant for Beta Thalassemia

Medically Reviewed by Jennifer Robinson, MD on November 18, 2021
3 min read

A stem cell transplant can cure beta thalassemia, but the treatment isn't the right choice for everyone. You'll need to learn the risks and side effects and talk to your doctor to see if it's a good idea for you.

If you or your child has beta thalassemia, the body doesn't make enough red blood cells to deliver the oxygen it needs. When you get a stem cell transplant, your doctor will try to fix the problem by putting "hematopoietic" stem cells into your bloodstream.

These stem cells are made in your bone marrow -- the spongy center of bones where blood cells develop. When you get a stem cell transplant, you receive stem cells from a donor that help you grow new, healthy red blood cells.

Before the transplant, you'll get chemotherapy to wipe out the stem cells in your bone marrow that aren't working right and prepare your bone marrow to receive new stem cells.

During the transplant, your doctor puts the donor's stem cells into your body through a plastic tube placed in your chest. After the cells go into your bloodstream, they travel to your bone marrow to begin making healthy blood cells.

If you get a stem cell transplant, you'll need to find a donor whose stem cells have a genetic makeup that matches yours. The transplant works best if you have a brother or sister who is a good match.

If you don't have a sibling who would be a good donor, it's still possible to find a match from a donor who's not related to you.

If you or your child get a stem cell transplant, one of the major risks is organ damage from too much iron in your body. That's why transplants work best in people who have had regular chelation therapy, a treatment that clears extra iron from the body.

If you already have some organ damage from your disease before the transplant, such as heart failure or cirrhosis, you'll have a greater chance of getting more organ damage.

Doctors don't recommend stem cell transplants if you have had renal failure or you're HIV positive, because there is too big a risk of organ damage.

Other risks include a condition called "graft versus host disease" (GVHD). When you get this problem, your body sees the new blood cells as a possible danger to you and begins attacking them.

Medications can prevent or stop GVHD, but because they work by curbing your immune system -- your body's defense against germs -- they raise your chance of getting infections.

Chemotherapy that you get before the transplant also raises your odds of infection. Your doctor can suggest medications, like antibiotics, to prevent this side effect.

Treatments for beta thalassemia, including stem cell transplants, are getting better all the time. In general, the younger you are, the better the stem cell transplant works. Stem cell transplants work best in children age 14 or younger.

One of the most common side effects of stem cell transplants is infertility. That's because the high doses of chemotherapy before the transplant can damage women's reproductive organs and cause you to stop having periods. The chemotherapy can also lower the amount of sperm that men make.

Your doctor can tell you about ways to keep your options to have children in the future. For instance, men may be able to preserve sperm before the transplant and women may be able to freeze their eggs.

Other side effects that can happen soon after transplant include:

  • Mouth or throat pain
  • Nausea and vomiting
  • Bleeding or bruising
  • Swelling in your lungs

It takes about 2 to 6 weeks for your blood count to return to normal after a stem cell transplant. Your doctor will follow you closely during this time to make sure you don't get infections or other serious side effects.