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A Phase II Study of Tocilizumab to Prevent Graft-versus-Host Disease after Cord Blood Transplantation

Full Title

IIT: IL-6 Receptor Blockade to Ameliorate Acute Graft versus Host Disease and Early Toxicity after Double Unit Cord Blood Transplantation in Adults with Hematologic Malignancies


The standard treatment for aggressive or advanced blood cancers is a stem cell transplant using blood-forming cells from a donor. For patients who don’t have a matched donor, stem cells from umbilical cord blood (cord blood transplant) is an option. There is a risk of a complication called graft-versus-host disease (GVHD) in people who receive stem cells from a donor, where immune cells from the donor attack tissues of the recipient, causing inflammation.

Patients already receive the drugs cyclosporine and mycophenolate mofetil to reduce the immune response and the risk of GVHD. In this study, researchers want to see if adding one dose of the drug tocilizumab to this regimen further lowers the risk of GVHD after cord blood transplantation. It works by inhibiting inflammation and is already used to treat other patients with inflammatory diseases and conditions, such as arthritis.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, non-Hodgkin lymphoma, or Hodgkin lymphoma and be eligible for cord blood transplantation.
  • Patients may not have received an autologous (self) stem cell transplant in the last year, an allogeneic (donated) stem cell transplant in the preceding two years, or two prior stem cell transplants of any kind.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients ages 18-65.

For more information and to inquire about eligibility for this study, please contact Dr. Ioannis Politikos at 212-639-3468.