Autoimmune Enteropathy: An Updated Review with Special Focus on Stem Cell Transplant Therapy.
Autoimmune Enteropathy: An Updated Review with Special Focus on Stem Cell Transplant Therapy.
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自身免疫性肠病：一项更新的评论，特别关注干细胞移植疗法。
DOI：
10.1007/s10620-018-5364-1
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发表时间：
2019-03
影响因子：
3.1
通讯作者：
Acra S
中科院分区：
文献类型：
--
作者：
Ahmed Z;Imdad A;Connelly JA;Acra S
自身免疫性肠病（AIE）是一种影响儿童和成人的复杂疾病。尽管与显着的发病率和死亡率相关，但该疾病的病理生理学及其治疗尚未得到很好的表征。本研究的目的是回顾这种罕见但具有临床意义的疾病的医学文献，以帮助更好地了解其病理生理学，并列举可用的诊断和治疗方式。使用自身免疫性肠病和难治性腹泻相关的关键术语在PubMed上进行文献检索，对出版日期或语言没有限制。我们共发现98份以病例报告和病例系列形式发表的AIE报告。回顾的证据表明，AIE是一种多方面的疾病，需要在适当的临床环境中高度怀疑才能做出早期诊断。目前的证据支持使用支持性治疗来纠正营养和代谢缺陷，并将免疫抑制剂和免疫调节剂作为定向治疗。造血干细胞移植是一种积极的，但成功的AIE患者作为免疫失调，多内分泌病，肠病，X连锁（IPEX）综合征的一部分的治疗方式。尽管AIE患者的发病率和死亡率仍然很高，但AIE管理的累积临床经验已经改善了移植和非移植AIE患者的结局。需要更多的研究来进一步确定新疗法对AIE的作用，多个机构参与的中央登记处可能有助于分享和标准化对这种罕见但严重疾病患者的护理。
Autoimmune enteropathy (AIE) is a complex disease affecting both children and adults. Although associated with significant morbidity and mortality, the pathophysiology of the disease and its treatment have not been well characterized. This study aims to review the medical literature available on this rare but clinically significant ailment, to help establish a better understanding of its pathophysiology and enumerate the available diagnostic and treatment modalities. A literature search was conducted on PubMed using key terms related to autoimmune enteropathy and intractable diarrhea, with no restrictions on the date of publication or language. We found a total of 98 reports of AIE published in the form of case reports and case series. The evidence reviewed suggests that AIE is a multifaceted disorder that requires a high index of suspicion in the appropriate clinical setting to be able to make an early diagnosis. Current evidence supports the use of supportive care to correct nutritional and metabolic deficiencies, and immunosuppressives and immunomodulators as directed therapies. Hematopoietic stem cell transplant is an aggressive, but successful curative modality for patients with AIE as part of immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome. Cumulative clinical experience with management of AIE has allowed improved outcomes in transplanted and non-transplanted AIE patients even though morbidity and mortality with are still high in patients with this condition. More research is needed to further define the role of new therapies for AIE, and a central registry with participation of multiple institutions might help share and standardize care of patients with this rare but serious condition.
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DOI：
10.1016/j.jaci.2017.10.041
发表时间：
2018-03
期刊：
The Journal of allergy and clinical immunology
影响因子：
--
作者：
Barzaghi F;Amaya Hernandez LC;Neven B;Ricci S;Kucuk ZY;Bleesing JJ;Nademi Z;Slatter MA;Ulloa ER;Shcherbina A;Roppelt A;Worth A;Silva J;Aiuti A;Murguia-Favela L;Speckmann C;Carneiro-Sampaio M;Fernandes JF;Baris S;Ozen A;Karakoc-Aydiner E;Kiykim A;Schulz A;Steinmann S;Notarangelo LD;Gambineri E;Lionetti P;Shearer WT;Forbes LR;Martinez C;Moshous D;Blanche S;Fisher A;Ruemmele FM;Tissandier C;Ouachee-Chardin M;Rieux-Laucat F;Cavazzana M;Qasim W;Lucarelli B;Albert MH;Kobayashi I;Alonso L;Diaz De Heredia C;Kanegane H;Lawitschka A;Seo JJ;Gonzalez-Vicent M;Diaz MA;Goyal RK;Sauer MG;Yesilipek A;Kim M;Yilmaz-Demirdag Y;Bhatia M;Khlevner J;Richmond Padilla EJ;Martino S;Montin D;Neth O;Molinos-Quintana A;Valverde-Fernandez J;Broides A;Pinsk V;Ballauf A;Haerynck F;Bordon V;Dhooge C;Garcia-Lloret ML;Bredius RG;Kałwak K;Haddad E;Seidel MG;Duckers G;Pai SY;Dvorak CC;Ehl S;Locatelli F;Goldman F;Gennery AR;Cowan MJ;Roncarolo MG;Bacchetta R;Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT)
通讯作者：
Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT)
影响因子：
29.4
作者：
CUENOD, B;BROUSSE, N;CERFBENSUSSAN, N
通讯作者：
CERFBENSUSSAN, N
影响因子：
3.1
作者：
Elwing, JE;Clouse, RE
通讯作者：
Clouse, RE
影响因子：
2.9
作者：
Gupta, Nitin K.;Yilmaz, Omer;Yajnik, Vijay
通讯作者：
Yajnik, Vijay
影响因子：
3.2
作者：
Bennett, CL;Brunkow, ME;Chance, PF
通讯作者：
Chance, PF