Ma Cindy S, Hare Nathan J, Nichols Kim E, Dupré Loic, Andolfi Grazia, Roncarolo Maria-Grazia, Adelstein Stephen, Hodgkin Philip D, Tangye Stuart G
Centenary Institute of Cancer Medicine and Cell Biology, Newtown, New South Wales, Australia.
J Clin Invest. 2005 Apr;115(4):1049-59. doi: 10.1172/JCI23139. Epub 2005 Mar 3.
X-linked lymphoproliferative disease (XLP) is an often-fatal immunodeficiency characterized by hypogammaglobulinemia, fulminant infectious mononucleosis, and/or lymphoma. The genetic lesion in XLP, SH2D1A, encodes the adaptor protein SAP (signaling lymphocytic activation molecule-associated [SLAM-associated] protein); however, the mechanism(s) by which mutations in SH2D1A causes hypogammaglobulinemia is unknown. Our analysis of 14 XLP patients revealed normal B cell development but a marked reduction in the number of memory B cells. The few memory cells detected were IgM(+), revealing deficient isotype switching in vivo. However, XLP B cells underwent proliferation and differentiation in vitro as efficiently as control B cells, which indicates that the block in differentiation in vivo is B cell extrinsic. This possibility is supported by the finding that XLP CD4(+) T cells did not efficiently differentiate into IL-10(+) effector cells or provide optimal B cell help in vitro. Importantly, the B cell help provided by SAP-deficient CD4(+) T cells was improved by provision of exogenous IL-10 or ectopic expression of SAP, which resulted in increased IL-10 production by T cells. XLP CD4(+) T cells also failed to efficiently upregulate expression of inducible costimulator (ICOS), a potent inducer of IL-10 production by CD4(+) T cells. Thus, insufficient IL-10 production may contribute to hypogammaglobulinemia in XLP. This finding suggests new strategies for treating this immunodeficiency.
X连锁淋巴增殖性疾病(XLP)是一种常致命的免疫缺陷病,其特征为低丙种球蛋白血症、暴发性传染性单核细胞增多症和/或淋巴瘤。XLP中的基因病变SH2D1A编码衔接蛋白SAP(信号淋巴细胞激活分子相关[SLAM相关]蛋白);然而,SH2D1A突变导致低丙种球蛋白血症的机制尚不清楚。我们对14例XLP患者的分析显示,B细胞发育正常,但记忆B细胞数量显著减少。检测到的少数记忆细胞为IgM(+),表明体内同种型转换存在缺陷。然而,XLP B细胞在体外增殖和分化的效率与对照B细胞一样高,这表明体内分化受阻是B细胞外源性的。XLP CD4(+) T细胞不能有效地分化为IL-10(+)效应细胞或在体外提供最佳的B细胞辅助,这一发现支持了这种可能性。重要的是,通过提供外源性IL-10或异位表达SAP可改善SAP缺陷型CD4(+) T细胞提供的B细胞辅助,这导致T细胞产生的IL-10增加。XLP CD4(+) T细胞也不能有效地上调诱导性共刺激分子(ICOS)的表达,ICOS是CD4(+) T细胞产生IL-10的有效诱导剂。因此,IL-10产生不足可能导致XLP中的低丙种球蛋白血症。这一发现为治疗这种免疫缺陷病提出了新的策略。