Role of reduced intensity conditioning in T-cell and B-cell immune reconstitution after HLA-identical bone marrow transplantation in ADA-SCID

Caterina Cancrini, Francesca Ferrua, Alessia Scarselli, Immacolata Brigida, Maria Luisa Romiti, Graziano Barera, Andrea Finocchi, Maria Grazia Roncarolo, Maurizio Caniglia, Alessandro Aiuti

Research output: Contribution to journalArticle

Abstract

The treatment of choice for severe combined immunodeficiency is bone marrow transplantation from an HLA-identical donor sibling without conditioning. However, this may result in low donor stem cell chimerism, leading to reduced long-term immune reconstitution. We compared engraftment, metabolic, and T-cell and B-cell immune reconstitution of HLA-identical sibling bone marrow transplantation performed in 2 severe combined immunodeficiency infants with adenosine deaminase deficiency from the same family treated with or without a reduced intensity conditioning regimen (busulfan/fludarabine). Only the patient who received conditioning showed a stable mixed chimerism in all lineages, including bone marrow myeloid and B cells. The use of conditioning resulted in higher thymus-derived naïve T cells and T-cell receptor excision circles, normalization of the T-cell repertoire, and faster and complete B-cell and metabolic reconstitution. These results suggest the utility of exploring the use of reduced intensity conditioning in bone marrow transplantation from HLA-identical donor in severe combined immunodeficiency to improve long-term immune reconstitution.

Original languageEnglish
Pages (from-to)1778-1782
Number of pages5
JournalHaematologica
Volume95
Issue number10
DOIs
Publication statusPublished - Oct 2010

Keywords

  • Bone marrow transplantation
  • Primary immunodeficiency
  • Reduced intensity conditioning

ASJC Scopus subject areas

  • Hematology

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