Ouadani Hanen, Ben-Mustapha Imen, Ben-ali Meriem, Ben-khemis Leila, Larguèche Beya, Boussoffara Raoudha, Maalej Sonia, Fetni Ilhem, Hassayoun Saida, Mahfoudh Abdelmajid, Mellouli Fethi, Yalaoui Sadok, Masmoudi Hatem, Bejaoui Mohamed, Barbouche Mohamed-Ridha
Laboratory of Transmission, Control and Immunobiology of Infection (LR11IPT02), Institut Pasteur de Tunis and University Tunis El Manar, Tunis, Tunisia.
Department of Pediatrics, Tahar Sfar Hospital, Mahdia, Tunisia.
Immunogenetics. 2016 Jan;68(1):19-28. doi: 10.1007/s00251-015-0878-6. Epub 2015 Nov 6.
Immunoglobulin class switch recombination deficiencies (Ig-CSR-D) are characterized by normal or elevated serum IgM level and absence of IgG, IgA, and IgE. Most reported cases are due to X-linked CD40L deficiency. Activation-induced cytidine deaminase deficiency is the most frequent autosomal recessive form, whereas CD40 deficiency is more rare. Herein, we present the first North African study on hyper IgM (HIGM) syndrome including 16 Tunisian patients. Phenotypic and genetic studies allowed us to determine their molecular basis. Three CD40LG mutations have been identified including two novels (c.348_351dup and c.782_*2del) and one already reported mutation (g.6182G>A). No mutation has been found in another patient despite the lack of CD40L expression. Interestingly, three AICDA mutations have been identified in 11 patients. Two mutations were novel (c.91T>C and c.389A>C found in one and five patients respectively), and one previously reported splicing mutation (c.156+1T>G) was found in five patients. Only one CD40-deficient patient, bearing a novel mutation (c.109T>G), has been identified. Thus, unlike previous reports, AID deficiency is the most frequent underlying molecular basis (68%) of Ig-CSR-D in Tunisian patients. This finding and the presence of specific recurrent mutations are probably due to the critical role played by inbreeding in North African populations.
免疫球蛋白类别转换重组缺陷(Ig-CSR-D)的特征是血清IgM水平正常或升高,而缺乏IgG、IgA和IgE。大多数报道的病例是由于X连锁的CD40L缺陷。活化诱导的胞苷脱氨酶缺陷是最常见的常染色体隐性形式,而CD40缺陷则较为罕见。在此,我们展示了第一项关于高IgM(HIGM)综合征的北非研究,包括16名突尼斯患者。表型和基因研究使我们能够确定其分子基础。已鉴定出三个CD40LG突变,包括两个新突变(c.348_351dup和c.782_*2del)和一个已报道的突变(g.6182G>A)。尽管缺乏CD40L表达,但在另一名患者中未发现突变。有趣的是,在11名患者中鉴定出三个AICDA突变。两个突变是新的(分别在一名和五名患者中发现的c.91T>C和c.389A>C),并且在五名患者中发现了一个先前报道的剪接突变(c.156+1T>G)。仅鉴定出一名携带新突变(c.109T>G)的CD40缺陷患者。因此,与先前的报道不同,AID缺陷是突尼斯患者中Ig-CSR-D最常见的潜在分子基础(68%)。这一发现以及特定反复突变的存在可能归因于近亲繁殖在北非人群中所起的关键作用。