Reappraisal of in utero stem cell transplantation based on long-term results

Jean Louis Touraine, Daniel Raudrant, François Golfier, Albert Rebaud, Rachel Sembeil, Maria Grazia Roncarolo, Rosa Bacchetta, Roseline D'Oiron, Thierry Lambert, Lucette Gebuhrer

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish
Pages (from-to)305-312
Number of pages8
JournalFetal Diagnosis and Therapy
Volume19
Issue number4
DOIs
Publication statusPublished - 2004

Keywords

  • Fetal liver
  • Hemophilia
  • Human fetus
  • Immunodeficiency
  • Immunoreconstitution
  • In utero transplant
  • Inborn errors of metabolism
  • Prolonged engraftment
  • Stem cell
  • Thalassemia

ASJC Scopus subject areas

  • Obstetrics and Gynaecology
  • Pediatrics, Perinatology, and Child Health

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