A single-center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

Laura Dinardo, Valerie Brown, Elena Perez, Nancy Bunin, Kathleen E. Sullivan

Research output: Contribution to journalArticle

7 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)63-72
Number of pages10
JournalPediatric Transplantation
Volume16
Issue number1
DOIs
StatePublished - Feb 1 2012

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Hematopoietic Stem Cell Transplantation
Transplants
X-Linked Combined Immunodeficiency Diseases
Tissue Donors
Intravenous Immunoglobulins
Immunoglobulins
Survival Rate
Transplantation
Pediatrics
Survival
Mortality
Incidence
Infection

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

Cite this

Dinardo, Laura ; Brown, Valerie ; Perez, Elena ; Bunin, Nancy ; Sullivan, Kathleen E. / A single-center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD). In: Pediatric Transplantation. 2012 ; Vol. 16, No. 1. pp. 63-72.
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A single-center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD). / Dinardo, Laura; Brown, Valerie; Perez, Elena; Bunin, Nancy; Sullivan, Kathleen E.

In: Pediatric Transplantation, Vol. 16, No. 1, 01.02.2012, p. 63-72.

Research output: Contribution to journalArticle

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