The stem cell transplantation program at NYP/Morgan Stanley Children’s is one of the oldest in the nation, with expertise in treating both malignant and non-cancerous conditions. Our stem cell transplantation program is accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) and the National Marrow Donor Program (NMDP) for autologous, allogeneic, and umbilical cord blood transplantation.
Our program is committed to improving outcomes for patients by decreasing the toxicities of stem cell transplantation through a two-pronged approach. First, we have pioneered novel reduced-intensity approaches to stem cell transplantation, in which we are able to give lower doses of chemotherapy with immune therapy to allow donor cell engraftment while minimizing toxicity. We have used these reduced-intensity approaches most extensively to treat patients with sickle cell disease, and have refined our approaches with over a decade of experience. We have one of the largest programs nationwide offering stem cell transplantation to patients with sickle cell disease.
Over the last five years, we have achieved 100 percent event-free survival in our sickle cell patients undergoing transplant using a matched sibling donor. We are actively working to translate these exceptional outcomes to patients transplanted with alternative donor sources.
Second, we offer a number of supportive therapies — including investigational therapies that are not available elsewhere — to treat or prevent the possible complications of stem cell transplantation, such as:
- Photophoresis to treat acute and chronic graft-versus-host disease (GVHD), a technique in which white blood cells are removed from the patient’s bloodstream, exposed to a drug that makes the cells sensitive to light, and then treated with ultraviolet radiation to kill large numbers of the white blood cells that cause GVHD.
- CD34-selected stem cell transplantation to prevent GVHD, where the T cells that cause GVHD are removed from the stem cell product prior to infusion into patients.
- Defibrotide to treat veno-occlusive disease (VOD), a complication that may occur early in the course of transplantation, resulting in liver dysfunction due to microscopic clots. Our program has an active treatment protocol and we have defibrotide in our onsite pharmacy, allowing us to initiate therapy rapidly and achieving a dramatic reduction in mortality from this complication.
- Mesenchymal stem cells (MSC), an investigational therapy to manage acute GVHD, are cells that give rise to bone, cartilage, tendons, muscles, and fat. MSC travel to areas of the body that require healing, such as tissues affected by GVHD. They are especially useful in patients with GVHD who are refractory to standard steroid therapy.
- Pediatric Intensive Care Unit (PICU), which allows us to provide intensive supportive measures such as ventilator support, extracorporeal membrane oxygenation (ECMO), and left ventricular assist device (LVAD) support