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In Utero Transplantation for Thalassemia
Author(s) -
FLAKE ALAN W.,
ZANJANI ESMAIL D.
Publication year - 1998
Publication title -
annals of the new york academy of sciences
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.712
H-Index - 248
eISSN - 1749-6632
pISSN - 0077-8923
DOI - 10.1111/j.1749-6632.1998.tb10487.x
Subject(s) - thalassemia , transplantation , context (archaeology) , medicine , hematopoietic stem cell transplantation , erythropoiesis , in utero , haematopoiesis , immunology , hemoglobinopathy , fetus , stem cell , anemia , hemolytic anemia , pregnancy , biology , genetics , paleontology
A bstract : In utero hematopoietic stem cell transplantation is a promising approach for the treatment of a variety of congenital hematologic diseases. Although the approach has been successful for immunodeficiency syndromes, attempts thus far to treat the hemoglobinopathies have failed. In most of these cases the late gestational age at transplantation, source of donor cells, or procedure‐related complications, provide an explanation for failure. Nevertheless the biology of thalassemia, in the context of prenatal transplantation, requires examination. In contrast to postnatal bone marrow transplant regimens, engraftment after in utero transplantation requires donor cells to effectively compete for developing receptive sites in the recipient hematopoietic microenvironment. Effective prenatal treatment of thalassemia will depend on the ability of normal cells to engraft and compete in the thalassemic microenvironment. Clinical observations after bone marrow transplantation of amelioration of anemia in β‐thalassemia by relatively low degrees of mixed chimerism, and the apparent selective advantage observed for donor erythropoiesis, suggest prenatal transplantation could succeed. Prenatal strategies involving multiple transplants, donor‐specific tolerance induction, and postnatal same‐donor transplants should be considered.