About this trial
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases.
Eligibility criteria
Qualifiers
AA will be defined as having peripheral blood cytopenias with a hypocellular bone marrow for age and a clinical diagnosis of aplastic anemia as determined by their treating physicians.
Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study.
Individuals with bone marrow failure due to clinical or molecularly diagnosed inherited bone marrow failure, inborn errors of immunity or other cause will be included.
Patients must receive an HCT or GT for management of their underlying disease. Allogeneic transplants including all conditioning regimens, donors, and GVHD prophylaxis regimens are eligible. This study does not define how the transplant or transplant-supportive care will be performed.
Disqualifiers
None
Trial design
Treatments tested in this trial
- Not listed
Trial groups
Sponsors and collaborators
Medical College of Wisconsin
Lead sponsor
National Cancer Institute (NCI)
Collaborator
National Institutes of Health (NIH)
Collaborator
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator