Your health can’t wait. Learn how we’re making our facilities safer and schedule your care now.
Children's Hospital is part of the UPMC family.
Be safe anytime, anywhere.
To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.
A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
Ranked #8 Nationally by U.S. News & World Report.
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.
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
University of Pittsburgh Medical Center
Study Description at the National Institutes of Health
Blood and Marrow Transplantation and Cellular Therapies Research
Beth Carella, DO
For more information about the study or enrollment, please contact:
Shawna McIntyre, Clinical Research Supervisor
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.