Hematopoietic stem cell transplantation for complete IFN-γ receptor 1 deficiency: A multi-institutional survey

Research output: Contribution to journalResearch articleContributedpeer-review

Contributors

  • Joachim Roesler - , University Hospital Carl Gustav Carus Dresden, Department of Child and Adolescent Psychiatry and Psychotherapy (Author)
  • Mitchell E. Horwitz - (Author)
  • Capucine Picard - (Author)
  • Pierre Bordigoni - (Author)
  • Graham Davies - (Author)
  • Ewa Koscielniak - (Author)
  • Mike Levin - (Author)
  • Paul Veys - (Author)
  • Ursula Reuter - (Author)
  • Ansgar Schulz - (Author)
  • Christian Thiede - , Department of Internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Thomas Klingebiel - (Author)
  • Alain Fischer - (Author)
  • Steven M. Holland - (Author)
  • Jean Laurent Casanova - (Author)
  • Wilhelm Friedrich - (Author)

Abstract

To evaluate the outcome of hematopoietic stem cell transplantation (HSCT) in a series of patients with inherited complete IFN-γ receptor 1 (IFNγR1) deficiency. We report 8 patients who received altogether 11 HSCT from family donors, including 10 HLA-identical (5 siblings and 5 relatives) and 1 HLA-haplo-identical donors. Five grafts were T-cell depleted, and conditioning regimens varied in intensity. Four patients died within 8 months after HSCT. Two of these deaths were due to specific complications related to mycobacterial infection. There was no or very low (2%) donor cell engraftment in 2 survivors. Only 2 patients are in full remission of mycobacterial disease 5 years after HSCT. These are the only patients who received non-T-cell-depleted grafts from an HLA-identical sibling after a fully myeloablative conditioning regimen. HSCT can lead to prolonged remission of mycobacterial disease in children with complete IFNγR1 deficiency. However, optimal control of mycobacterial infection before HSCT and the use of a non-T-cell-depleted transplant from an HLA-identical sibling after a fully myeloablative conditioning regimen are recommended.

Details

Original languageEnglish
Pages (from-to)806-812
Number of pages7
JournalJournal of Pediatrics
Volume145
Issue number6
Publication statusPublished - Dec 2004
Peer-reviewedYes

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