Gene and Cell Therapies for Beta-Globinopathies by Punam Malik & John Tisdale

Author:Punam Malik & John Tisdale
Language: eng
Format: epub
Publisher: Springer New York, New York, NY

Umbilical Cord Blood Transplantation for Patients with Hemoglobin Disorders

Since the first report of a successful umbilical cord blood (CB) transplant in 1989 in a pediatric patient with Fanconi Anemia [23], this option has increasingly been used to treat patients with malignant and nonmalignant hematologic diseases . While CB grafts are smaller in cellularity and volume as compared to bone marrow, the proliferative capacity and numbers of progenitor cells contained within the graft are often greater [24, 25]. Further, due to immunological differences such as lower numbers of CD3+, CD4+, and CD8+ T-cells with a higher CD4/CD8 ratio and a higher percentage of naïve CD45RA+ T-cells [26], CB transplants have been associated with a lower risk of GvHD as compared to bone marrow transplants. Besides the lower risk of GvHD, other advantages associated with CB grafts include easy availability, reduced time to complete the pre-transplant process, little or no donor morbidity, a decreased risk of transmitting latent viral infections, possibility of directed sibling banking, transplant at earlier disease stages, potential for greater HLA-mismatching, an expandable donor pool, and a greater frequency of rare HLA haplotypes in the donor pool as compared to bone marrow registries [27–32].

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