A single-center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)
Version of Record online: 17 NOV 2011
© 2011 John Wiley & Sons A/S
Volume 16, Issue 1, pages 63–72, February 2012
How to Cite
DiNardo, L., Brown, V., Perez, E., Bunin, N. and Sullivan, K. E. (2012), A single-center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD). Pediatric Transplantation, 16: 63–72. doi: 10.1111/j.1399-3046.2011.01606.x
- Issue online: 17 JAN 2012
- Version of Record online: 17 NOV 2011
- Accepted for publication 14 September 2011
- severe combined immune deficiency;
- X-linked severe combined immune deficiency;
- hematopoietic stem cell transplantation;
- bone marrow;
DiNardo L, Brown V, Perez E, Bunin N, Sullivan KE. A single-center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD). Pediatr Transplantation 2012: 16: 63–72. © 2011 John Wiley & Sons A/S.
Abstract: PIDD are rare inherited disorders that can result in life-threatening infections. Allogeneic HSCT is the only cure for many primary immune deficiencies; however, the specific diseases and optimal type(s) of transplants are not clear. This study compares transplant outcomes in a large cohort with a relatively uniform pre- and post-transplant management strategies. We conducted a retrospective analysis of 39 pediatric patients who underwent HSCT for SCID (n = 25) or other immune deficiencies (n = 14) from 1986 to 2010. A structured case report form was used to collect clinical information. The outcomes of survival, immune reconstitution, engraftment, incidence of GvHD and IVIG dependency were tabulated. Overall survival rates were 88% for SCID and 86% for other primary immune deficiencies, which are high compared to other historical series. No single variable was associated with mortality. Immunoglobulin dependence occurred only in patients who had X-linked SCID and a parental donor haploidentical transplant. Because of improved supportive care and use of alternative donors and conditioning regimens, HSCT has become an acceptable option for an increasing number of PIDD subtypes not previously transplanted with high frequency. This study encourages greater use of transplantation.