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

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Joachim Roesler - , Universitätsklinikum Carl Gustav Carus Dresden, Klinik und Poliklinik für Kinder- und Jugendmedizin (Autor:in)
  • Mitchell E. Horwitz - (Autor:in)
  • Capucine Picard - (Autor:in)
  • Pierre Bordigoni - (Autor:in)
  • Graham Davies - (Autor:in)
  • Ewa Koscielniak - (Autor:in)
  • Mike Levin - (Autor:in)
  • Paul Veys - (Autor:in)
  • Ursula Reuter - (Autor:in)
  • Ansgar Schulz - (Autor:in)
  • Christian Thiede - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Thomas Klingebiel - (Autor:in)
  • Alain Fischer - (Autor:in)
  • Steven M. Holland - (Autor:in)
  • Jean Laurent Casanova - (Autor:in)
  • Wilhelm Friedrich - (Autor:in)

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

OriginalspracheEnglisch
Seiten (von - bis)806-812
Seitenumfang7
FachzeitschriftJournal of Pediatrics
Jahrgang145
Ausgabenummer6
PublikationsstatusVeröffentlicht - Dez. 2004
Peer-Review-StatusJa