2019
DOI: 10.1111/pedi.12895
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Hematopoietic stem cell transplantation recovers insulin deficiency in type 1 diabetes mellitus associated with IPEX syndrome

Abstract: Immune dysregulation, polyendocrinopathy, enteropathy, and X‐linked (IPEX) syndrome is an autoimmune disorder caused by the dysfunction of FOXP3, which leads to regulatory T‐(Treg) cell dysfunction and subsequently autoimmunity including type 1 diabetes mellitus (T1D). Presently, allogeneic hematopoietic stem cell transplantation (HSCT) is a potential curative therapy for IPEX syndrome, but not for T1D. Generally, after complete loss of pancreatic β‐cells, HSCT cannot improve the prognosis of T1D. Here, we rep… Show more

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Cited by 15 publications
(5 citation statements)
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“…Currently, hematopoietic stem cell transplantation (HSCT) is the only curative therapy for IPEX, and survival after HSCT is significantly impacted by disease severity [ 18 ]. Amelioration of insulin dependency has been reported in an adolescent treated with HSCT 1 month after diabetes onset [ 19 ], suggesting the period from onset may also impact efficacy. This further underlines the importance of rapid genetic testing and variant classification.…”
Section: Discussionmentioning
confidence: 99%
“…Currently, hematopoietic stem cell transplantation (HSCT) is the only curative therapy for IPEX, and survival after HSCT is significantly impacted by disease severity [ 18 ]. Amelioration of insulin dependency has been reported in an adolescent treated with HSCT 1 month after diabetes onset [ 19 ], suggesting the period from onset may also impact efficacy. This further underlines the importance of rapid genetic testing and variant classification.…”
Section: Discussionmentioning
confidence: 99%
“… 45 Nevertheless, there are reports of the curative potential of allogeneic HSCT in T1DM, in cases of allogeneic HSCT performed due to other indications, with cure of diabetes as an incidental outcome. 46 , 47 …”
Section: Discussionmentioning
confidence: 99%
“… 96 , 97 The key to the aetiological treatment of T1DM lies in preventing early islet loss in susceptible individuals, promoting islet regeneration during remission, or islet transplantation in the case of chronic disease, each of which can be regulated by Tregs. 98 In recent years, animal models and clinical trials have also confirmed that promoting increases in the number of Tregs in the body, regulate Tregs homeostasis, and improve the progression of T1DM, the effects of which tend to be notably more pronounced during the early stages of T1DM development. 99 , 100 However, although this would appear to imply that the activation of Tregs is highly beneficial from the perspective of T1DM treatment, 100 long-term observations in a large number of patients are needed for confirmation.…”
Section: Irisin Tregs and T1dmmentioning
confidence: 93%