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综合征性腹泻/毛发肝肠综合征中的炎症性肠病样特征

IBD-Like Features in Syndromic Diarrhea/Trichohepatoenteric Syndrome.

作者信息

Busoni Veronica B, Lemale Julie, Dubern Beatrice, Frangi Fernando, Bourgeois Patrice, Orsi Marina, Badens Catherine, Fabre Alexandre

机构信息

*Pediatric Gastroenterology, Hepatology, and Liver Intestinal Transplantation Unit, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina †Nutrition et Gastroentérologie Pédiatriques, Hôpital Armand-Trousseau ‡UMR_S U1166 Nutriomics, UPMC, Sorbonne University, Paris, France §Pediatric Department, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina ||Département de Génétique Médicale, Hôpital de la Timone Enfant ¶UMR_S 910, Aix-Marseille Université #Service de Pédiatrie Multidisciplinaire, Hôpital de la Timone Enfant, Assistance Publique des Hôpitaux de Marseille, Marseille, France.

出版信息

J Pediatr Gastroenterol Nutr. 2017 Jan;64(1):37-41. doi: 10.1097/MPG.0000000000001218.

Abstract

BACKGROUND

Very early onset inflammatory bowel disease (VEOIBD) (inflammatory bowel disease [IBD] before 6 years of age) may manifest as a monogenic disease affecting the gastrointestinal tract. Syndromic diarrhea/trichohepatoenteric syndrome (SD/THE), a rare disorder caused by alteration of a complex involved in RNA degradation, has been reported to present with some degree of colitis and in some cases an IBD-like presentation.

METHODS

We reviewed clinical and biological data of 4 previously published cases and added detailed data of 2 new cases of SD/THE with an IBD-like presentation.

RESULTS

All the 6 patients presented with typical intractable diarrhea and hair abnormalities. The colon was affected in all of the patients: 1 had ileitis, 2 had panenteritis, and 2 presented with perianal disease. Fecal calprotectin level and erythrosedimentation rate were elevated in 2 cases each. All the therapeutic classes of IBD treatment (mesalazine, steroids, immunomodulators, and biological therapy) were used in the 6 cases. In 2 patients, treatment had no effect. Three showed a partial effect, and 1 patient sustained only a transient effect.

CONCLUSIONS

SD/THE can have a similar presentation as VEOIBD, often as pancolitis. IBD treatments appear to have little efficacy for SD/THE, suggesting a different pathogenesis for the IBD-like features in SD/THE compared with classical IBD.

摘要

背景

极早发型炎症性肠病(VEOIBD)(6岁前的炎症性肠病[IBD])可能表现为影响胃肠道的单基因疾病。综合征性腹泻/毛发肝肠综合征(SD/THE)是一种由参与RNA降解的复合体改变引起的罕见疾病,据报道会出现一定程度的结肠炎,在某些情况下还会有类似IBD的表现。

方法

我们回顾了4例先前发表病例的临床和生物学数据,并补充了2例有类似IBD表现的新SD/THE病例的详细数据。

结果

所有6例患者均有典型的顽固性腹泻和毛发异常。所有患者的结肠均受累:1例有回肠炎,2例有全肠炎,2例有肛周疾病。粪便钙卫蛋白水平和红细胞沉降率各有2例升高。6例患者均使用了IBD治疗的所有类别(美沙拉嗪、类固醇、免疫调节剂和生物疗法)。2例患者治疗无效。3例有部分疗效,1例仅维持短暂疗效。

结论

SD/THE可能有与VEOIBD相似的表现,常为全结肠炎。IBD治疗对SD/THE似乎疗效甚微,这表明SD/THE中类似IBD的特征与经典IBD的发病机制不同。

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