Autoimmune Focal Segmental Glomerulosclerosis (FSGS)

Read more about our new revolutionary approach to treating and potentially curing FSGS, called DISOT. 

What is autoimmune focal segmental glomerulosclerosis (FSGS)?

Autoimmune FSGS is a rare disease where the immune system produces a chemical that causes the filters in the kidneys to leak. The tiny filters, called glomeruli, become scarred. Scarring often leads to nephrotic syndrome (a disorder where the kidneys excrete extra protein in the urine), which in turn can lead to progressive renal failure. It is most common in children and teenagers.

What causes autoimmune FSGS?

The cause of autoimmune FSGS is unknown, but we do know that it involves a problem with the cells of the immune system.

What are the signs and symptoms of autoimmune FSGS?

Symptoms of autoimmune FSGS include swelling in the legs, ankles, and around the eyes. Because the body is retaining fluids, you might notice that your child is gaining weight. Another sign is foamy urine. Your child might test with high cholesterol or high blood pressure. If kidney failure is involved, your child could experience fatigue, headaches, itchy skin, a poor appetite, and vomiting. Also, with autoimmune FSGS, the body can’t hold on to protein well, making it hard for children to grow well and be healthy. High-risk treatments, such as steroids, also set children up for infections.

Is there a cure for autoimmune FSGS?

When kidneys are scarred, that scarring is permanent, which means your child will eventually need a kidney transplant. Unfortunately, traditional kidney transplants fail in children with autoimmune FSGS over 90% of the time. This is because the immune system attacks the donor kidney and destroys it, usually within a few weeks. Doctors at Stanford Medicine Children’s Health have developed a two-transplant approach that gives children with autoimmune FSGS a chance at a successful kidney transplant. This approach, called dual immune/solid organ transplant (DISOT), is essentially a cure for autoimmune FSGS. Once we give your child a new immune system with a stem cell transplant, it will not attack a new kidney from the same donor.

How is autoimmune FSGS traditionally treated?

• Autoimmune FSGS can be treated with diet changes, medicines, and high doses of immunosuppressive drugs and corticosteroids given intravenously to suppress the immune system.
• We can also perform plasma freezes to replace plasma, the liquid part of the blood, with healthy plasma. This washes toxins out of the blood.
• If your child’s kidneys fail, he or she will need to undergo regular dialysis treatments, which can be done at an infusion center or at home. The dialysis machine removes waste from your child’s blood. However, dialysis cannot replace real kidneys, which work 24 hours a day, so your child will eventually need a kidney transplant.

What is Stanford Medicine Children’s Health’s groundbreaking treatment for autoimmune FSGS?

We have researched, developed, and had initial success with a two-transplant approach to treating autoimmune FSGS called dual immune/solid organ transplant (DISOT). DISOT was recently featured in the New England Journal of Medicine. We are the only hospital in the world that is currently offering this revolutionary approach.

DISOT is a stem cell transplant, which provides your child with a new immune system, followed by a kidney transplant from the same donor, usually a parent. Since your child’s new immune system recognizes its new kidney, it isn’t likely to reject it.

What if I am not a match with my child for a kidney transplant?

We are able to use a haploidentical (partially matched) donor, which is usually a parent. We selectively eliminate alpha/beta T-cells (the immune system’s fighter cells) from the donor’s stem cells to reduce the risk of graft-versus-host disease, which is otherwise a frequent complication of these kinds of transplants. This revolutionary stem cell transplant method is led by Alice Bertaina, MD, PhD—the worldwide pioneer and foremost expert in alpha/beta T-cell depleted haploidentical stem cell transplantation. Even though alpha/beta T-cells are removed, these cells recover 60–90 days after transplant, so your child regains his or her full immune function. 

What is the benefit of DISOT for treating autoimmune FSGS?

There are several benefits to our novel dual immune/solid organ transplant (DISOT) approach:

• When a new immune system is adopted from a stem cell donor—one that recognizes the transplanted kidney as its own—it essentially reboots your child’s immune system. This means the possibility of chronic rejection of the kidney is eliminated, along with the need for long-term immunosuppressive medication.
• By removing the need for medication, doctors also remove toxicities associated with these medications. This means your child will feel better.
• Your child’s body is no longer in a state of constantly fighting itself, and he or she will likely experience more energy and renewed growth and development.
• Without medications and long hours of dialysis, your child’s quality of life is greatly improved, and he or she can get back to the business of being a child.
• The donor kidney could last a long time, maybe even a lifetime. The average living donor kidney transplant lasts about 19 years, requiring another kidney transplant as an adult. Because our dual immune/solid organ transplant (DISOT) method often eliminates the need for transplant-rejection drugs—which are hard on kidneys—there’s a good chance that your child might not need a future kidney transplant.

Have you tried DISOT with other children, and if so, how are they doing?

Yes, and it has been successful. One patient with severe FSGS now has normal kidney function and is off all medications. He reports feeling well, and he is gaining physical strength and energy.

My child with autoimmune FSGS has had a failed kidney transplant already—are they a candidate for DISOT?

Yes. They should be a candidate, and we are happy to evaluate your child. Have their nephrologist contact us at (650) 498-4905.

Your child’s first kidney transplant failed because their immune system was still programmed by the FSGS. Because we replace your child’s immune system before the kidney transplant—basically wiping out the FSGS—the risk of rejection is nearly nonexistent.

Why should I choose Stanford Medicine Children’s Health for autoimmune FSGS treatment?

Most important, because we offer a chance at a cure of your child’s kidney disease and immune problems. We also are experts at what we do. Stanford Medicine Children’s Health has performed over 1,000 stem cell transplants since our program’s inception, and we are a national leader in pediatric kidney transplant volumes. Our program in alpha/beta T-cell depletion, which reduces the risk of the stem cell transplant being rejected as well as the risk of graft-versus-host-disease, is the biggest in the country.

Also, when children with FSGS come to us, they receive care from a multidisciplinary team of internationally respected stem cell and kidney transplant specialists, nephrologists, neuroimmunologists, oncologists, and other specialists. With our revolutionary DISOT method, we give hope to parents who experienced little before.

Where can I find more information on general FSGS?

A great resource to learn more about living with kidney disease, particularly nephrotic syndrome (which can be caused by FSGS), is Nephcure Kidney International. It’s a site created by families for families with nephrotic syndrome and FSGS. You can also learn more on the National Kidney Foundation’s FSGS page.

Learn more about our pediatric stem cell transplant, kidney transplant, and kidney care programs at Stanford Medicine Children’s Health.

Want your child to be evaluated for DISOT?

Have your child’s nephrologist contact us at (650) 498-4905.