Research

Severe Blood Disorder Treatment with Allogenic Stem Cell Transplant - Pilot

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

Protocol Description

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.

Eligibility Criteria

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

Additional Resources
Bone Marrow Transplant
Sickle Cell Disease
Alpha Thalassemia
Beta Thalassemia
Study description at National Institutes of Health