*Sickle Cell Disease Treatment with Alternative Donor Bone Marrow Transplant – Phase I/II

T-Cell Depleted, Alternative Donor Transplant in Pediatric and Young Adult Patients with Severe Sickle Cell Disease

Protocol Description

This research study by the UPMC Immune Transplant and Therapy Center (ITTC) evaluates what effect, if any, hematopoietic stem cell transplant (HSCT) from mismatched unrelated donors and certain related donors may have on patients with severe sickle cell disease, beta-thalassemia major anemia, or Diamond-Blackfan anemia. Typically, physicians seek donor cells from a sibling with matched human leukocyte antigen (HLA); however, such donors are not always available. By using stem cells from mismatched unrelated donors or HLA-haploidentical (half-related) donor stem cells, this study will increase the number of patients who can undergo a HSCT for the specified disease. The study also applies a technique for reducing T-cells to help prevent graft-versus-host disease in the setting of mismatched donor transplantation.

Study Level: Phase I/II

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling patients who have severe sickle cell disease, beta-thalassemia major, or Diamond-Blackfan anemia and require an HSCT.
Males and Females: Ages 5 through 40


Participants in this study will have a HSCT, a procedure in which the stem cells in the participant’s blood and bone marrow are replaced by stem cells from a healthy donor. A 30-day conditioning period to lower the participant’s white blood count through chemotherapy and radiation will precede transplant. Patients will then receive an infusion of bone marrow cells that have had 99% of T and B lymphocytes removed. After this transplant, participants will remain in the hospital for several weeks until their white blood counts recover. The research team will monitor participants’ health for 2 years over 11 follow up visits.
Visits: 11 follow-up visits after transplant
Duration: About 26 months

Status: Open to Enrollment

Source(s) of Support

University of Pittsburgh Medical Center

Primary Investigator

Jessie Barnum, MD

Contact Information

For more information about the study or enrollment, please contact:
Shawna McIntyre, Clinical Research Supervisor

*Original research initiated by investigators at UPMC Children’s Hospital of Pittsburgh.