Patient Care

For the past three decades, the Duke Division of Pediatric Transplant and Cellular Therapy (PTCT) has provided life-saving transplants to more than 2200 patients, including 70 percent receiving transplants from family, cord blood or unrelated donors and 30 percent receiving autologous cells. Nearly 1,000 transplants have been performed for patients with leukemia or lymphoma and approximately 200 for neuroblastoma. Collectively our faculty is world-renowned in the transplantation of children with inherited metabolic diseases (including lysosomal storage and peroxisomal disorders), performing nearly 350 transplants. In partnership with our Duke pediatric immunology colleagues, we have transplanted more than 200 patients with inherited immunodeficiency disease. In addition, we have transplanted more than 200 patients with sickle cell disease, thalassemia and other non-malignant bone marrow disorders and are distinguished for our experience in treating Krabbe disease, metachromatic leukodystrophy, adrenoleukodystrophy, Hurler syndrome, Hunter syndrome, Batten disease and numerous other rare genetic diseases.

Over the years, our expertise has evolved from a program providing pioneering cord blood transplants to one utilizing any type of donor and transplantation regimen to achieve the best outcomes for our patients. We are well established in all areas of autologous and allogeneic hematopoietic cell transplantation and CAR-T therapies. We also have established programs in gene therapy and a wide range of cell therapies for transplant patients as well as in the emerging field of regenerative medicine.

Specifically, the division has extensive expertise and experience in performing:

Hematopoietic Cell Transplantation

• Haplo-identical donor transplant using post-transplant cyclophosphamide as well as magnetic T cell depletion protocols
• Related and unrelated bone marrow transplantation
• Peripheral blood stem cell transplantation
• Umbilical cord blood transplantation
• Non-myeloablative, reduced intensity and reduced toxicity transplantation

CAR-T Therapy for Hematologic Malignancies

Gene Therapy

• Sickle Cell Disease
• Severe Combined Immunodeficiency Diseases (SCID)

Cell Therapies

• DUOC-01 for inherited metabolic diseases and multiple sclerosis
• Cord blood infusions for children with brain injuries (cerebral palsy (CP), autism, hypoxic-ischemic encephalopathy (HIE), acute ischemic stroke)
• Cord tissue derived mesenchymal stromal cell (MSC) infusions for graft versus host disease (GvHD), autism, hypoxic-ischemic encephalopathy (HIE), COVID-ARDS, COVID-MISC
• Bone marrow derived MSCs for GvHD

Thymus Implantation in collaboration with the Division of Allergy and Immunology

• Congenital Athymia