Using flow cytometry to screen patients for X-linked lymphoproliferative disease due to SAP deficiency and XIAP deficiency

J Immunol Methods. 2010 Oct 31;362(1-2):1-9. doi: 10.1016/j.jim.2010.08.010. Epub 2010 Sep 9.

Abstract

X-linked lymphoproliferative disease is a rare congenital immunodeficiency that is most often caused by mutations in SH2D1A, the gene encoding signaling lymphocyte activation molecule (SLAM)-associated protein (SAP). XLP caused by SAP deficiency is most often characterized by fulminant mononucleosis/EBV- associated hemophagocytic lymphohistiocytosis (HLH), lymphoma, and dysgammaglobulinemia. XLP has also been found to be caused by mutations in BIRC4, the gene encoding X-linked inhibitor of apoptosis (XIAP). Patients with XIAP deficiency often present with HLH or recurrent HLH, which may or may not be associated with EBV. XLP is prematurely lethal in the majority of cases. While genetic sequencing can provide a genetic diagnosis of XLP, a more rapid means of diagnosis of XLP is desirable. Rapid diagnosis is especially important in the setting of HLH, as this may hasten the initiation of life-saving medical treatments and expedite preparations for allogeneic hematopoietic cell transplantation (HCT). Flow cytometry offers a means to quickly screen patients for XLP. Flow cytometry can be used to measure lymphocyte SAP or XIAP protein expression, and can also be used to observe lymphocyte phenotypes and functional defects that are unique to XLP. This review will give a brief overview of the clinical manifestations and molecular basis of SAP deficiency and XIAP deficiency, and will focus on the use of flow cytometry for diagnosis of XLP.

MeSH terms

  • Flow Cytometry / methods*
  • Flow Cytometry / standards
  • Gene Expression Regulation
  • Genetic Diseases, X-Linked* / blood
  • Genetic Diseases, X-Linked* / diagnosis
  • Genetic Diseases, X-Linked* / genetics
  • Genetic Diseases, X-Linked* / therapy
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Intracellular Signaling Peptides and Proteins / blood
  • Intracellular Signaling Peptides and Proteins / genetics*
  • Lymphocytes / metabolism
  • Lymphoproliferative Disorders* / blood
  • Lymphoproliferative Disorders* / diagnosis
  • Lymphoproliferative Disorders* / genetics
  • Mutation*
  • Signaling Lymphocytic Activation Molecule Associated Protein
  • Time Factors
  • Transplantation, Homologous
  • X-Linked Inhibitor of Apoptosis Protein / blood
  • X-Linked Inhibitor of Apoptosis Protein / genetics*

Substances

  • Intracellular Signaling Peptides and Proteins
  • SH2D1A protein, human
  • Signaling Lymphocytic Activation Molecule Associated Protein
  • X-Linked Inhibitor of Apoptosis Protein
  • XIAP protein, human