MT2014-10C : Allogeneic Hematopoietic Stem Cell Transplant for Patients with High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders


The primary purpose of this study is to confirm the findings of our previous allogeneic hematopoietic stem cell transplant trial for non-malignant hematologic disorders that are transfusion dependent or represent other potentially life-threatening cytopenias. Participation in this study will not alter treatment or medical care. All information for this study will be collected from medical records.

I'm interested

Male or Female
Not specified
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• up to 55 years old
• diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
• suitable stem cell donor has been identified
• see link to for complete inclusion and exclusion criteria
Exclusion Criteria:

• active, uncontrolled infection
• women who are pregnant or breastfeeding
• HIV positive

Drug: Myeloablative Preparative Regimen, Drug: Reduced Intensity Preparative Regimen, Drug: Reduced Toxicity Ablative Regimen

Blood Disorders

Clinics and Surgery Center (CSC), Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Sickle Cell Disease, Thalassemia

Holly Franceen -
Ashish Gupta
See this study on