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努南综合征中与淋巴水肿相关的复发性蜂窝织炎:伴RIT1变异的病例报告及文献综述

Recurrent cellulitis associated with lymphoedema in Noonan syndrome: case reports with RIT1 variants and literature review.

作者信息

Kobayashi Yuki, Adachi Takeya, Tahara Umi, Tanaka Moemi, Arakawa Hiroki, Funatsu Yohei, Moritani Kazunori, Yamada Mamiko, Kosaki Kenjiro, Inazumi Toyoko

机构信息

Department of Dermatology, Tachikawa Hospital, Federation of National Public Service Personnel Mutual Aid Associations, Tokyo, Japan.

Department of Dermatology, Keio University School of Medicine, Tokyo, Japan.

出版信息

Hum Genome Var. 2025 Jun 4;12(1):12. doi: 10.1038/s41439-025-00315-1.

Abstract

Noonan syndrome (NS) is a RASopathy, a disorder caused by genetic alterations involving the Ras/mitogen-activated protein kinase pathway. It causes characteristic clinical manifestations, including facial dysmorphism and congenital cardiac defects. Occasionally, lymphoedema and recurrent cellulitis occur in patients with NS, potentially escalating to lethal conditions. Despite the frequent association of cellulitis with lymphoedema in NS, features susceptible to these complications have not been fully characterized. We encountered two patients with NS carrying RIT1 pathogenic variants, who were treated for recurrent lower leg cellulitis since their teenage years, which occasionally progressed to sepsis. Here we retrospectively examined these patients with NS and recurrent cellulitis on the background of lymphoedema and reviewed published cases of NS with lymphoedema and cellulitis up to March 2024 to elucidate the clinical and genetic features of this subgroup. Our literature review identified 16 additional patients with NS with similar complications. Among the 18 patients (15 men), genetic analyses revealed pathogenic variants in PTPN11 and RIT1 in 4 patients each, with the latter occurring more frequently than commonly observed. The patients developed lymphoedema by 15 years of age, predisposing them to cellulitis by 23 years of age. Notably, four of the five patients with sepsis had congenital heart defects, with a higher prevalence than that generally reported in NS. This study highlights the characteristics of genetic variants, congenital cardiac anomalies and heightened risk of recurrent cellulitis in patients with NS, emphasizing the need for early intervention with prophylactic antibiotics and surgical treatment to mitigate these risks.

摘要

努南综合征(NS)是一种RAS病,由涉及Ras/丝裂原活化蛋白激酶途径的基因改变引起。它会导致特征性临床表现,包括面部畸形和先天性心脏缺陷。偶尔,NS患者会出现淋巴水肿和复发性蜂窝织炎,可能会发展为致命状况。尽管在NS中蜂窝织炎与淋巴水肿经常相关,但易患这些并发症的特征尚未完全明确。我们遇到了两名携带RIT1致病变异的NS患者,他们从青少年时期就开始接受复发性小腿蜂窝织炎的治疗,偶尔会发展为败血症。在此,我们回顾性研究了这些伴有淋巴水肿背景的NS和复发性蜂窝织炎患者,并回顾了截至2024年3月已发表的伴有淋巴水肿和蜂窝织炎的NS病例,以阐明该亚组的临床和遗传特征。我们的文献综述又发现了16例患有类似并发症的NS患者。在这18例患者(15名男性)中,基因分析显示4例患者的PTPN11和RIT1存在致病变异,后者的发生频率高于通常观察到的情况。这些患者在15岁时出现淋巴水肿,到23岁时易患蜂窝织炎。值得注意的是,五名败血症患者中有四名患有先天性心脏缺陷,其患病率高于NS中一般报道的情况。这项研究突出了NS患者的基因变异特征、先天性心脏异常以及复发性蜂窝织炎的高风险,强调需要通过预防性抗生素和手术治疗进行早期干预以降低这些风险。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/578b/12137658/97f498ffdd53/41439_2025_315_Fig1_HTML.jpg

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