Thalassemia Major Clinical Trial
Official title:
Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major
The purpose of this study is to collect peripheral blood and bone marrow aspirate samples from thalassemia patients in Tehran, in a collaborative effort to develop an erythroid lineage specific chimerism assay applicable to patients with thalassemia. Development of such an assay would be useful both for identification of the exact mutation causing the disease, as well as for providing a direct method to measure and monitor the kinetics of donor erythropoiesis in this patient population following transplant.
- In the first phase of the study we will develop a panel of reagents designed to
identify predominant thalassemia mutations in the Iranian population, as well as
alternative highly variable erythroid specific polymorphisms. To determine how
informative this panel is, these reagents will be applied to samples collected from
patients homozygous for the disorder, and compared to patients with thalassemia trait
and normal Iranian donors.
- In the second phase, we propose to serially measure and compare erythroid lineage
chimerism with overall genomic chimerism following transplant. Samples will be
collected from participants before and at 1, 2, 3, 6 and 12 months following transplant
and cryopreserved. Peripheral blood from the stem cell donor will also be collected and
cryopreserved. Participants will undergo myeloablative or nonmyeloablative transplant.
;
Time Perspective: Retrospective
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