A Phase II Study of a New Approach to Stem Cell Transplantation for Patients with Noncancerous Blood and Immune System Disorders


Full Title

A Phase II Trial of Alpha Beta T-cell and CD19 B-cell depleted Peripheral Blood Stem Cell Transplantation using the CliniMACS System for patients with Non-Malignant Hematologic Disorders from matched or mismatched, related or unrelated donors


An infusion of stem cells from a donor (“allogeneic stem cell transplant”) is one way of treating noncancerous disorders of the blood and immune system. One potential complication of stem cell transplantation is graft-versus-host disease (GvHD). GvHD is caused when cells from the donor view the recipient’s cells as foreign, resulting in symptoms such as rash, diarrhea, nausea, and vomiting, among others. Removing the T cells (“T-cell depletion”) from the donor’s stem cells lowers the risk of GvHD.

Research shows that removing T cells in the lab after the stem cells are taken from the donor, but before they are given to the patient, helps stop GVHD, but may slow down recovery of the immune system and increase the risk of infection after transplant. However, not all types of T cells cause GVHD. Patients in this study will only have the alpha beta T cells, which doctors believe are responsible for GVHD, removed from their donor’s stem cells. This technique will keep the “good” T cells in the donor stem cells so they can help the patient’s immune system recover faster to prevent infections. The removal of alpha beta T cells will be done using a machine called CliniMACS.

In order to try to prevent another serious complication of transplantation, called post-transplant Epstein-Barr virus (EBV)-associated lymphoproliferative disease (PTLD), the researchers will also remove B cells (CD19+ lymphocytes) from the donor stem cells and give patients a medication called rituximab to remove B cells from the body. PTLD is a disorder caused when EBV reproduces uncontrollably in B cells, causing enlargement of the lymph nodes and lymphoma.

This study is open to patients with diverse stem cell donor options, including matched and partially matched related and unrelated donors.


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

  • Patients must have a noncancerous blood or immune system disorder that requires treatment with stem cell transplantation. Examples include hemoglobinopathies like sickle cell disease and thalassemia; bone marrow failure syndromes (aplastic anemia and inherited marrow failure disorders); autoimmune cytopenias; and histiocytic disorders.
  • This study is open to pediatric and adult patients under age 70.

For more information about this study and to inquire about eligibility, please contact Dr. Maria Cancio at 212-639-2446.





Disease Status

Newly Diagnosed & Relapsed/Refractory