This is a prospective, longitudinal, non-therapeutic study which includes routine assessment for long-term effects, as per FDA guidelines after receipt of an allogeneic HCT or autologous genetically modified cellular products for hemoglobin disorders.
Primary objective:
\- To provide long term follow up, for individuals with hemoglobin disorders undergoing allogeneic hematopoietic stem cell transplantation (HCT) or receipt of an autologous genetically modified cellular product to treat their hemoglobinopathy. For individuals receiving a genetically modified cellular product, this long term follow up study is in accordance with the guidelines provided by the Food and Drug Administration (FDA).
Receipt, or planned receipt, of an allogeneic HSCT or infusion of genetically modified autologous cells for hemoglobin disorders within 15 years prior to enrollment
Inability or unwillingness of research participant and/or legal guardian/ representative to provide written informed consent.
200 patients with hemoglobin disorders that have received, or are planning to receive, either a genetically modified cell product or allogenic HCT will be invited to enroll on this long term follow up study.
Cohort
Prospective
Not listed
To provide ongoing review of long-term clinical and psychosocial outcomes and late effects of hematopoietic stem cell transplant and gene therapy recipients at St. Jude Children's Research Hospital.
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