Stem-cell transplantation for primary immunodeficiencies

Bella Bielorai, Hana Golan, Gideon Rechavi, Amos Toren*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

3 Scopus citations

Abstract

Until further improvement in gene therapy is achieved, stem-cell transplantation is still the main option for cure of patients with primary immunodeficiency diseases. Performing the transplant in early infancy before severe infections and organ damage develop has the highest chance for success. The remarkable progress that has been achieved in understanding these disorders has made it possible to identify most of the genetic abnormalities. This enables an early transplant immediately after birth or even in utero. However, the optimal approach for stem-cell transplantation in children with immunodeficiency has yet to be determined. The non-myeloablative protocols increasingly used in recent years have the potential for immune reconstitution without considerable toxicity, but the experience is still limited. A better understanding of the specific immune dysfunction and the exact degree of residual immunity may enable the tailoring of the most appropriate conditioning regimen for each patient so that engraftment will be ensured with little morbidity.

Original languageEnglish
Pages (from-to)648-652
Number of pages5
JournalIsrael Medical Association Journal
Volume4
Issue number8
StatePublished - 2002
Externally publishedYes

Keywords

  • Primary immunodeficiencies
  • Severe combined immune deficiency
  • Stem-cell transplantation

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