Blood and Marrow Transplantation

We offer a range of cutting-edge therapies that expand treatment options and improve outcomes for children with complex conditions, such as: 

• Gene therapy for sickle cell disease and beta thalassemia, including FDA-approved treatments such as CASGEVY™, LYFGENIA™ and ZYNTEGLO®
• CAR T-cell therapy, including KYMRIAH®, for certain types of leukemia and lymphoma
• Extracorporeal photopheresis for managing graft-versus-host disease 
• Ruxolitinib, a JAK1/JAK2 inhibitor, for steroid-refractory graft-versus-host disease in children
• Virus-specific T-cell therapy to prevent and treat viral infections post-transplant

Our care extends beyond medical treatment. We offer:

• Integrative therapies through the PANDA team, including aromatherapy, massage therapy, acupressure and acupuncture
• Child Life services, music therapy and art therapy
• Educational specialists to support school reintegration
• Psychology and social work support for emotional and family well-being

Children’s National is the only hospital in the Washington, D.C., area that works with the Oncofertility Consortium. This group includes doctors, scientists and teachers who focus on helping young cancer patients keep the option to have children in the future.

Our Pediatric Gynecology and Urology teams talk with patients and families to explain their choices. They help families understand the options and make the best decisions for their child. Our doctors explain fertility preservation in ways that are right for each child’s age, so we can support their future plans to have a family.

As the region’s only dedicated pediatric National Marrow Donor Program transplant center and a participating site in the NIH-funded BMT Clinical Trials Network, we offer access to national and international clinical trials. Our physicians lead and collaborate on studies through the Center for International Blood and Marrow Transplant Research and the Pediatric Blood and Marrow Transplant Consortium. 

We also operate an in-house stem cell processing laboratory, ensuring timely and precise cell preparation. Our Center for Cancer and Immunology Research (CETI) supports ongoing innovation in both malignant and non-malignant transplant therapies.

Learn more about clinical trials

A blood or bone marrow transplant replaces damaged, diseased or blood-forming cells with healthy donor cells (stem cells). These stem cells must closely match your child's unique genetic profile. In many cases, the donor is a sibling or family member. However, for some children, finding a suitable match can be challenging and may delay treatment. At Children’s National, we are committed to expanding access to transplant by successfully using a wide range of stem cell sources. Our team has deep experience with matched sibling and unrelated donors, umbilical cord blood and half-matched (haploidentical) family donors. We are one of the few programs in the country offering a clinical trial using a biological parent as a half-matched donor, opening new possibilities for children who do not have a traditional match. Through ongoing research and clinical trials, we continue to explore and refine alternative donor options to ensure that more children can receive timely, life-saving transplants.

To help you understand the full process, we’ve created a step-by-step guide that walks you through each stage of care—from diagnosis and evaluation to treatment, recovery and long-term follow-up.