Treatments for Severe Combined Immunodeficiency (SCID) in Children

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What are the treatment options for SCID?

Guarding against infection
Nothing is more important to the health of a child with severe combined immunodeficiency (SCID) than warding off potential infections. Your child's clinician can advise you about the exact steps you should be taking to help reduce the risk of infection.

Mothers of newborn children with SCID should discuss the pros and cons of breastfeeding with their clinicians, as some infections can be passed through breast milk.

As a rule, children with SCID should not receive the standard childhood vaccinations. Because the B cells of children with SCID do not function properly, their bodies cannot produce the normal antibodies that fight off viruses. Since many vaccines are actually live viruses, they pose too high a risk of infection to be safe for a child with a drastically weakened immune system.

Other ways to avoid potential infections include:

Basic precautions

You should be sure to always:

  • keep your child away from crowds, dirty places or anyone who is ill or seems to be "coming down with something"
  • follow a strict hand washing regimen for your child, your family and any visitors
  • use protective face masks at the recommendation of your child's doctor
  • give your child antibiotics, antifungal or antiviral medications at the direction of your child's doctor

Antibody infusions
Because your child's body does not have healthy B cells that produce antibodies against infection, he may need regular intravenous (IV, administration through a vein) or subcutaneous infusions (administration under the skin) of the antibody immunoglobin (also known as immune globin, gammaglobin, IVIG or SCIG).

Stem cell/bone marrow transplants
Nearly every child with SCID is treated with a stem cell transplant (also known as a bone marrow transplant). This is the only available treatment option that has a chance of providing a permanent cure. The bone marrow cells or stem cells are administered through an IV, similar to a blood transfusion.

Stem cells are a versatile type of cell found in bone marrow. These cells have a unique and powerful ability: they can develop into several different types of specialized cells.

In the case of a child with SCID, the transplanted stem cells are injected into the bloodstream. They will then become healthy white blood cells that replenish immune functions—essentially building a whole new, functional immune system for the child. If the immune system regains complete function, the child may be permanently cured.

The effectiveness of a stem cell transplant for SCID depends on:

  • your child's overall health at the time of the procedure
  • the match between your child and donor's bone marrow (if the donated marrow comes from a matched sibling, the procedure has a better than 90 percent success rate)
  • your child's age at the time of the transplant (For optimal success, a baby should have the transplant performed no later than the age of 3 months; some studies have shown that, if stem cell transplants are performed within the first 28 days of a baby's life, the success rate is as high as 95 percent.)

There can be some obstacles to a successful stem cell transplant. For instance, a suitable donor may not be available.

In addition, any transplant carries a risk of a phenomenon known as graft-versus-host disease. This means that the donated bone marrow attacks the recipient, which can be a fatal complication.

At Boston Children's Hospital, stem cell transplants are conducted through the Stem Cell Transplant Center at Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

Gene therapy
A promising field, gene therapy, may have the answers to the obstacles that are present with stem cell transplants. In gene therapy, patients receive stem cells from their own bone marrow (rather than from another person). These cells also have an added ingredient: they've been infused with a working, healthy version of the mutated gene responsible for the child's disease.

If the gene therapy is successful, the child will enjoy all the benefits of a stem cell transplant without the potential hazard of graft-versus-host disease.

Importantly, gene therapy is still an experimental treatment and has its own complications. Children for whom there are no suitable donors or who have a high risk of major complications after a transplant may be candidates for gene therapy.

In 2010, Boston Children's began an international trial of gene therapy to treat SCID-X1. Read the story of a child who was part of the trial. 

The study is currently open for enrollment. To learn more, visit the trial listing on ClinicalTrials.gov. To find out if your child may be eligible to participate, please contact David Williams, MD (email); Luigi Notarangelo, MD (email); or Sung-Yun Pai, MD (email).  

Long-term Outlook

If your child's immune system is restored to good working order, she should be able to enjoy a rich, active adult life, including having a family.

It is important to note, though, that some forms of SCID can run in families. For example, SCID-X1 is passed on to boys from mothers who carry the gene mutation on the X chromosome. Genetic counseling is essential for prospective parents with a family history of SCID or any other immune deficiency.

Helpful links

Please note that neither Boston Children's Hospital, the Division of Allergy and Immunology at Boston Children's nor the Dana-Farber/Boston Children's Cancer and Blood Disorders Center’s Stem Cell Transplantation Program unreservedly endorses all of the information found at the sites listed below. These links are provided as a resource.

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

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