Immunologic Research

, Volume 44, Issue 1, pp 4–17

Single-center analysis of long-term outcome after hematopoietic cell transplantation in children with congenital severe T cell immunodeficiency

Authors

  • Evelina Mazzolari
    • Divisione di Emato-Oncologia PediatricaOspedale dei Bambini
    • Dipartimento Materno-infantile e Tecnologie BiomedicheUniversità di Brescia
  • Donatella de Martiis
    • Divisione di Emato-Oncologia PediatricaOspedale dei Bambini
  • Concetta Forino
    • Divisione di Emato-Oncologia PediatricaOspedale dei Bambini
  • Arnalda Lanfranchi
    • Divisione di Emato-Oncologia PediatricaOspedale dei Bambini
  • Silvia Giliani
    • “Angelo Nocivelli” Institute for Molecular MedicineUniversity of Brescia
  • Roberto Marzollo
    • Divisione di Emato-Oncologia PediatricaOspedale dei Bambini
  • Paolo Airò
    • Servizio di Immunologia Clinica
  • Luisa Imberti
    • Laboratorio di Biotecnologie, Diagnostic Department
  • Fulvio Porta
    • Divisione di Emato-Oncologia PediatricaOspedale dei Bambini
    • Dipartimento Materno-infantile e Tecnologie BiomedicheUniversità di Brescia
    • Division of ImmunologyChildren’s Hospital, Harvard Medical School
Article

DOI: 10.1007/s12026-008-8022-4

Cite this article as:
Mazzolari, E., de Martiis, D., Forino, C. et al. Immunol Res (2009) 44: 4. doi:10.1007/s12026-008-8022-4

Abstract

We review clinical outcome and immune reconstitution in a consecutive series of 74 infants with severe T cell immunodeficiency who received hematopoietic cell transplantation (HCT) from January 1991 to May 2003. Fifty-three patients (71.6%) are alive. Results were significantly better for recipients of HCT from HLA-matched related donors (100% survival) and unrelated donors (86.4%) than from mismatched related donors (51.6%). A detailed analysis of immune reconstitution and clinical status was performed in 49 surviving patients, most of which have attained robust T and B cell reconstitution and are in very good clinical conditions. No cases of late deaths or of chronic graft-versus-host disease (GvHD) have been observed. However, infections and autoimmunity at >1 year after HCT have been observed in a significant number of patients. Persistence of a low number of circulating naive T cells and long-term requirement for intravenous immunoglobulin were associated with a higher incidence of clinical events.

Keywords

Hematopoietic cell transplantationSevere combined immunodeficiencyImmune reconstitutionT lymphocytesB lymphocytesIntravenous immunoglobulinsAutoimmunity

Copyright information

© Springer Science+Business Media, LLC 2008