Life-saving stem cell transplants for children

Our team provides stem cell transplants and exceptional care to help restore your child to health

Pediatric Stem Cell Transplantation

If your child has an immune disorder, has a blood disease or metabolic condition, or is fighting cancer, stem cell transplantation could be a chance at a cure and a return to a healthy life. Stem cell transplantation essentially replaces your child’s immune system with a new one, and this approach is a promising option to treat many children who don’t currently have a cure for their condition. We also offer several clinical trials that use gene therapy—removing a broken gene and replacing it with a healthy copy—to treat several different blood and immune diseases that were previously incurable.

Our Pediatric Stem Cell Transplantation program was established in 1986. Since then, we have transplanted more than 1,000 patients.

Why choose us for your child’s care

  • We offer unique treatments not available elsewhere, including clinical trials testing gene therapy for blood diseases like sickle cell disease and pyruvate kinase deficiency, and a first-in-the-world treatment that combines kidney and stem cell transplantation to give patients a new kidney that will last without long-term immune suppression.
  • We can offer stem cell transplantation to patients who may not have qualified for one in the past. Stanford’s Alice Bertaina, MD, PhD, is a world-renowned pioneer in alpha/beta T-cell depleted haploidentical (partially matched) stem cell transplantation. She removes alpha/beta T cells (the immune system’s fighter cells) from the donor cells, which enables your child to have a successful transplant with partially matched donor cells, like those from a parent. This method also reduces the risk of graft-versus-host-disease and makes it safer for very medically fragile children to receive a stem cell transplant.
  • Our doctors and researchers are developing ways to make stem cell transplantation safer and more effective, including testing whether regulatory T cells can prevent graft-versus-host disease and whether a patient’s body can be prepared to receive donor stem cells using a new antibody-based method instead of traditional chemotherapy or radiation.
  • Our Stanford Laboratory for Cell and Gene Medicine allows us to produce cells involved in many treatments right here on campus, helping us bring a potential cure to your child faster.
  • Your child will need to stay in the hospital during his or her stem cell transplant. Our patients stay in a dedicated unit with positive-pressure ventilation to keep every room cleaner and prevent infections, and with a specialized nursing staff who treat stem cell transplant patients exclusively.
  • We are an active member of the Children’s Oncology Group, the Pediatric Transplantation & Cellular Therapy Consortium, and the Blood & Marrow Transplant Clinical Trials Network, enabling us to share research and innovations with other centers around the world. Our doctors are all certified by the Foundation for the Accreditation of Cellular Therapy (FACT).

Pediatric stem cell transplant patient and family support

We recognize that stem cell transplantation places enormous emotional, financial, psychological, and logistical burdens on your family and your child. In addition to a dedicated inpatient and outpatient nursing staff, we provide multifaceted support for patients and families, including dedicated social workers and child life specialists; a hospital-based school; physical, occupational, and speech therapy; and spiritual support. Our social workers also conduct group therapy sessions for families. The hospital-based school is a unique collaboration between the hospital and the Palo Alto Unified School District that helps your child continue their schoolwork during transplantation and aids in their transition back to their original school.

In addition, the newly renovated Ronald McDonald House is available for housing patients and families. A shuttle is available to transport you and your child between the house and the hospital.


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Testing an approach to reduce graft-versus-host disease

Graft-versus-host disease is one of the most common complications of stem cell transplantation. Stanford’s Maria Grazia Roncarolo, MD, and her team have launched a clinical trial to test whether T cells can help prevent graft-versus-host disease after stem cell transplantation. This trial is only available at Lucile Packard Children’s Hospital Stanford.


Pediatric Stem Cell Transplantation Patient Stories