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, C., Doerken, S., Aiuti, A., Albert, M.H., Al-Herz, W., Allende, L.M., Scarselli, A., Avcin, T., Perez-Becker, R., Cancrini, C., Cant, A., Di Cesare, S., Finocchi, A., Fischer, A., Gaspar, H.B., Ghosh, S., Gennery, A., Gilmour, K., González-Granado, L.I., Martinez-Gallo, M., et al.. - 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
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/20.500.11768/78490
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