Absent T-cell immunity resulting in life-threatening infections provides a clear rationale for hematopoetic stem cell transplantation (HSCT) in patients with severe combined immunodeficiency (SCID). Combined immunodeficiencies (CIDs) and "atypical" SCID show reduced, not absent T-cell immunity. If associated with infections or autoimmunity, they represent profound combined immunodeficiency (P-CID), for which outcome data are insufficient for unambiguous early transplant decisions.

A prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysis / Speckmann, Carsten; Doerken, Sam; Aiuti, Alessandro; Albert, Michael H; Al-Herz, Waleed; Allende, Luis M; Scarselli, Alessia; Avcin, Tadej; Perez-Becker, Ruy; Cancrini, Caterina; Cant, Andrew; Di Cesare, Silvia; Finocchi, Andrea; Fischer, Alain; Gaspar, H Bobby; Ghosh, Sujal; Gennery, Andrew; Gilmour, Kimberly; González-Granado, Luis I; Martinez-Gallo, Monica; Hambleton, Sophie; Hauck, Fabian; Hoenig, Manfred; Moshous, Despina; Neven, Benedicte; Niehues, Tim; Notarangelo, Luigi; Picard, Capucine; Rieber, Nikolaus; Schulz, Ansgar; Schwarz, Klaus; Seidel, Markus G; Soler-Palacin, Pere; Stepensky, Polina; Strahm, Brigitte; Vraetz, Thomas; Warnatz, Klaus; Winterhalter, Christine; Worth, Austen; Fuchs, Sebastian; Uhlmann, Annette; Ehl, Stephan. - In: THE JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY. - ISSN 1097-6825. - 139:4(2017), p. 1302-1310.e4. [10.1016/j.jaci.2016.07.040]

A prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysis

Aiuti, Alessandro;
2017-01-01

Abstract

Absent T-cell immunity resulting in life-threatening infections provides a clear rationale for hematopoetic stem cell transplantation (HSCT) in patients with severe combined immunodeficiency (SCID). Combined immunodeficiencies (CIDs) and "atypical" SCID show reduced, not absent T-cell immunity. If associated with infections or autoimmunity, they represent profound combined immunodeficiency (P-CID), for which outcome data are insufficient for unambiguous early transplant decisions.
2017
T-cell deficiency; combined immunodeficiency; hematopoietic stem cell transplantation; natural history; Adolescent; Child; Child, Preschool; Female; Humans; Infant; Male; Prospective Studies; Research Design; Severe Combined Immunodeficiency; Hematopoietic Stem Cell Transplantation
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/20.500.11768/78490
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus 54
  • ???jsp.display-item.citation.isi??? 51
social impact