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).
This protocol will provide a mechanism to appropriately monitor individuals with hemoglobin disorders that have received an allogenic HCT or an autologous genetically modified cellular product for hemoglobinopathies. Monitoring will include potential long-term adverse effects after receipt of these treatments, as well as long-term monitoring after the receipt of the genetically modified cellular product.
Study Type
OBSERVATIONAL
Enrollment
200
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
RECRUITINGTo 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.
Time frame: long term follow up. Can also be stated as "up to 15 years after transplant"
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