Studies Open for Enrollment

Allogeneic Hematopoietic Stem Cell Transplantation for Patients with High Risk Hemoglobinopathy Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism

The goal of this pilot study is to develop less toxic treatments for patients with severe blood disorders, such as sickle cell disease (SCD) and thalassemia, that are associated with organ damage and premature death. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only therapy that can cure a blood disorder of this type. This multi-center study will: explore a novel nontoxic approach to stem cell transplantation; determine optimization of an immunosuppressive regimen using busulfan and mycophenolic acid; and examine success rates as related to donor matches in treating patients with SCD.

Candidates
Children’s Hospital of Pittsburgh of UPMC will be recruiting 10 of the total 20 candidates for this study. Eligible male and female candidates with SCD or thalassemia must be between 3 and 35 years of age.
Males: 3 to 35 years
Females: 3 to 35 years

Requirements
Candidates are given a specific 12-day pharmaceutical dosage-reducing treatment leading up to a stem cell transplant. A bone marrow draw and biopsy is performed 1 month after donor lymphocyte infusions. A small amount of blood will be collected from recipients prior to transplantation and at key times following transplantation.

Status: Open for Enrollment

How to Participate
To get started, please contact:
Lakshmanan Krishnamurti, MD
412-692-7192

Additional Resources
Bone Marrow Transplant
Sickle Cell Disease
Alpha Thalassemia
Beta Thalassemia

Principal Investigator
Lakshmanan Krishnamurti, MD
Pediatric Hematology/Oncology

Source of Support
Research Advisory Committee, Children’s Hospital of Pittsburgh of UPMC