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

  • Evelina Mazzolari
  • Donatella de Martiis
  • Concetta Forino
  • Arnalda Lanfranchi
  • Silvia Giliani
  • Roberto Marzollo
  • Paolo Airò
  • Luisa Imberti
  • Fulvio Porta
  • Luigi D. Notarangelo
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

Authors and Affiliations

  • Evelina Mazzolari
    • 1
    • 2
  • Donatella de Martiis
    • 1
  • Concetta Forino
    • 1
  • Arnalda Lanfranchi
    • 1
  • Silvia Giliani
    • 3
  • Roberto Marzollo
    • 1
  • Paolo Airò
    • 4
  • Luisa Imberti
    • 5
  • Fulvio Porta
    • 1
  • Luigi D. Notarangelo
    • 2
    • 6
  1. 1.Divisione di Emato-Oncologia PediatricaOspedale dei BambiniBresciaItaly
  2. 2.Dipartimento Materno-infantile e Tecnologie BiomedicheUniversità di BresciaBrescia Italy
  3. 3.“Angelo Nocivelli” Institute for Molecular MedicineUniversity of BresciaBrescia Italy
  4. 4.Servizio di Immunologia ClinicaBresciaItaly
  5. 5.Laboratorio di Biotecnologie, Diagnostic DepartmentBresciaItaly
  6. 6.Division of ImmunologyChildren’s Hospital, Harvard Medical SchoolBostonUSA