Author/Authors :
Cuvelier، نويسنده , , Geoff D.E. and Schultz، نويسنده , , Kirk R. and Davis، نويسنده , , Jeff and Hirschfeld، نويسنده , , Aaron F. and Junker، نويسنده , , Anne K. and Tan، نويسنده , , Rusung and Turvey، نويسنده , , Stuart E.، نويسنده ,
Abstract :
This FOCIS Centers of Excellence Short Analytical Review is based on the clinical vignette of two boys from the same family with very different outcomes following hematopoietic stem cell transplantation (HSCT) for X-linked severe combined immunodeficiency (SCID). We review the kinetics of immune reconstitution following HSCT in SCID and emphasize the latest information regarding optimizing transplant outcomes for this disorder. The cases illustrate the difficulties and controversies surrounding the optimal strategies for planning SCID transplants. Specifically, we will focus on 3 areas of current debate and investigation: (i) factors involved in donor selection; (ii) the role of pretransplant conditioning; and (iii) benefits of early HSCT for SCID.
Keywords :
Primary immunodeficiency , Hematopoietic stem cell transplantation , Severe combined immunodeficiency
