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

Haematologica. 2010 Oct;95(10):1778-82. doi: 10.3324/haematol.2010.025098. Epub 2010 May 11.

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.

Publication types

  • Case Reports
  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenosine Deaminase / deficiency
  • Agammaglobulinemia / therapy
  • B-Lymphocytes / cytology
  • Bone Marrow Transplantation / immunology
  • Bone Marrow Transplantation / methods*
  • Graft Survival
  • Humans
  • Infant
  • Severe Combined Immunodeficiency / therapy*
  • Siblings
  • T-Lymphocytes / cytology
  • Transplantation Conditioning / methods*
  • Transplantation, Isogeneic

Substances

  • Adenosine Deaminase

Supplementary concepts

  • Severe combined immunodeficiency due to adenosine deaminase deficiency