Clinical Reviews in Allergy & Immunology

, Volume 46, Issue 2, pp 131–144

Recent Advances in Transplantation for Primary Immune Deficiency Diseases: A Comprehensive Review

Article

DOI: 10.1007/s12016-013-8379-6

Cite this article as:
de la Morena, M.T. & Nelson, R.P. Clinic Rev Allerg Immunol (2014) 46: 131. doi:10.1007/s12016-013-8379-6

Abstract

Hematopoietic cell transplantation (HCT) is a curative therapeutic option for severe combined immunodeficiency (SCID), a group of diseases which otherwise carry life expectancies that are of limited duration and quality. Survival following HCT for SCID has improved from approximately 23 to 91 % over the last 40 years. Success with SCID prompted efforts to apply HCT to the therapeutic challenge of well over 20 molecularly defined primary immune deficiency diseases (PID). Such success is due to both early recognition of PIDs and advances in the field of transplantation. Such advances include high-resolution HLA DNA donor–recipient matching, expansion of donor sources, better tolerated conditioning, new antibiotics, and wider availability. International collaborative efforts have provided patients and caregivers information that permit better treatment decisions now, and direct clinicians and investigators to ensure progress in the future. Pioneers in screening for SCID have taken steps to correct the fundamental challenge to successful treatment, which is the rapid discovery and characterization of cases and offering the transplant option to an affected child early in life; blood spot testing for T and B cell receptor quantification is now available to a growing fraction of newborns. Organizations including the Primary Immune Deficiency Treatment Consortium in the USA, The European Society for Primary Immunodeficiency, the European Group for Blood and Marrow Transplantation, the Pediatric Blood and Marrow Transplant Consortium, the United States Immunodeficiency Network, the Immune Deficiency Foundation, and the Jeffrey Modell Foundation are contributing mightily to increase awareness and standardize optimal utilization to the benefit of patients. This review will update the allergist–immunologist concerning disease presentations, indications for transplantation, methodologies, conditioning regimens, and clinical outcomes for patients with PID for which timely HCT is critical.

Keywords

Primary immune deficiency Immunodeficiency Stem cell transplantation Bone marrow transplantation 

Copyright information

© Springer Science+Business Media New York 2013

Authors and Affiliations

  • M. Teresa de la Morena
    • 1
  • Robert P. NelsonJr.
    • 2
    • 3
  1. 1.Division of Allergy and ImmunologyUniversity of Texas Southwestern Medical Center in Dallas and Children’s Medical Center DallasDallasUSA
  2. 2.Divisions of Hematology and OncologyIndiana University School of MedicineIndianapolisUSA
  3. 3.The IU Melvin and Bren Simon Cancer Center and Riley Hospital for ChildrenIndiana University School of MedicineIndianapolisUSA

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