Reappraisal of in utero stem cell transplantation based on long-term results.
The therapeutic field of in utero transplantation of stem cells, into human fetuses, has developed since 1988 with the hope of improved probability of engraftment and tolerance, due to immune immaturity of the host. Fifteen years later, it is possible to evaluate the results that we and others have obtained in the treatment of several fetal diseases. Seven fetal patients have been treated in Lyon: In 2 cases, pregnancy termination was induced by the in utero injection; in the 5 other cases, engraftment was obtained and repeatedly documented with presence of donor HLA antigens and/or Y chromosome in recipients. In the 2 patients with combined immunodeficiency disease, a sustained reconstitution of immunity was obtained as a result of the transplant but other complications occurred thereafter. In patients with thalassemia major, Niemann-Pick disease or hemophilia, a very partial and very transitory benefit was only obtained. Approximately 33 other patients with immunodeficiencies, hemoglobinopathies or inborn errors of metabolism have been treated worldwide, over the last 13 years, with a comparable method, using parental or fetal stem cells transplanted in utero. Successful treatment has usually been recorded in immunodeficiencies, and insufficient results have been obtained in the other cases. This form of treatment can therefore be recommended after prenatal diagnosis of combined immunodeficiency but additional research is required to improve the degree of engraftment, the lack of resistance of the host and the 'space' available for hematopoiesis in the other conditions.
Touraine
Jean-Louis
JL
Department of Transplantation and Clinical Immunology, Claude Bernard University, Lyon, and Department of Obstetrics, Paris University, Paris, France. jean-louis.touraine@chu-lyon.fr
Raudrant
Daniel
D
Golfier
François
F
Rebaud
Albert
A
Sembeil
Rachel
R
Roncarolo
Maria-Grazia
MG
Bacchetta
Rosa
R
D'Oiron
Roseline
R
Lambert
Thierry
T
Gebuhrer
Lucette
L
D000818:Animals; D005260:Female; D005315:Fetal Diseases / Q000000981:diagnostic imaging / Q000601:surgery; D046128:Fetal Therapies / Q000379:methods / Q000706:statistics & numerical data*; D006453:Hemoglobinopathies / Q000000981:diagnostic imaging / Q000601:surgery; D006801:Humans; D011247:Pregnancy; D011296:Prenatal Diagnosis / Q000379:methods / Q000706:statistics & numerical data; D033581:Stem Cell Transplantation / Q000379:methods / Q000706:statistics & numerical data*; D013995:Time; D014463:Ultrasonography