Refractory Pediatric Autoimmune Enteropathy Successfully Treated with Hematopoietic Stem Cell Transplant

We report a pediatric case of autoimmune enteropathy (AIE) that was successfully treated with haploidentical SCT and post-transplant cyclophosphamide.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 455 Source Type: research

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Abstract The ovary serves as the source of oocytes for the maintenance of female fertility and is a major supplier of sex hormones for endocrine homeostasis. Various circumstances such as genetic defects, autoimmune disorders, natural aging and environmental toxins can damage the ovaries leading to diminished ovarian function, and there are currently no effective treatment regimens for such loss of function. Stem cells show promising for treating many refractory diseases, and stem cell transplantation has been shown to be effective and safe as a new therapeutic method for ovarian injuries and ovarian aging in both...
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research
Primary immunodeficiencies (PIDs) encompass a heterogeneous group of diseases affecting all components of the immune system 1. They are caused by mutations in various genes and may phenotypically present with infections, as well as with autoimmune phenomena and malignancies 2. Despite advances in gene therapy, allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment for most children with various malignant or nonmalignant diseases, including selected PIDs.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Authors: Roudsari PP, Alavi-Moghadam S, Rezaei-Tavirani M, Goodarzi P, Tayanloo-Beik A, Sayahpour FA, Larijani B, Arjmand B Abstract Rheumatoid arthritis as a common autoimmune inflammatory disorder with unknown etiology can affect 0.5-1% of adults in developed countries. It involves more than just the patient's joints and can be accompanied by several comorbidities and affect cardiovascular, pulmonary, and some other systems of the human body. Although cytokine-mediated pathways are mentioned to have a central role in RA pathogenesis, adaptive and innate immune systems and intracellular signaling pathways all have...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
In this study we show that psychological stress attenuates therapeutic effects of MSCT in a DSS-induced colitis mouse model by elevating the levels of exosomal Mir7k/mmu-let-7k (microRNA 7k) in circulation. Mechanistically, Mir7k inhibits STAT3 pathway in donor MSCs, leading to upregulated expression of BECN1 (beclin 1, autophagy related) and, thus, activation of macroautophagy/autophagy. Inhibition of autophagy by blocking Mir7k or activating STAT3 signaling can restore MSCT-mediated therapy in psychologically stressed colitis mice. Our study identifies a previously unknown role of autophagy in regulating MSCT therapy via...
Source: Autophagy - Category: Cytology Authors: Tags: Autophagy Source Type: research
Conclusions: These data suggest that combined intravenous and intrathecal low-dose hUC-MSCs transplantation is safe and feasible in RRMS and NMO patients in the long term. The conclusion requires confirmation by future clinical trials in a larger cohort.
Source: Frontiers in Neurology - Category: Neurology Source Type: research
Abstract Cell-based therapies are emerging for Type I diabetes mellitus (T1D), an autoimmune disease characterized by the destruction of insulin producing pancreatic β-cells, as a means to provide long term restoration of glycemic control. Biomaterial scaffolds provide an opportunity to enhance the manufacturing and transplantation of islets or stem cell-derived β-cells. In contrast to encapsulation strategies that prevent host contact with the graft, recent approaches aim to integrate the transplant with the host to facilitate glucose sensing and insulin distribution, while also needing to modulate the ...
Source: Endocrinology - Category: Endocrinology Authors: Tags: Endocrinology Source Type: research
In conclusion, using a large cohort with rich health and DNA methylation data, we provide the first comparison of six major epigenetic measures of biological ageing with respect to their associations with leading causes of mortality and disease burden. DNAm GrimAge outperformed the other measures in its associations with disease data and associated clinical traits. This may suggest that predicting mortality, rather than age or homeostatic characteristics, may be more informative for common disease prediction. Thus, proteomic-based methods (as utilised by DNAm GrimAge) using large, physiologically diverse protein sets for p...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
AbstractHeterozygous gain-of-function (GOF) mutations inSTAT1 in patients with chronic mucocutaneous candidiasis (CMC) and hypothyroidism were discovered in 2011. CMC is the recurrent or persistent mucocutaneous infection byCandida fungi, and hypothyroidism results from autoimmune thyroiditis. Patients with these diseases develop other infectious diseases, including viral, bacterial, and fungal diseases, and other autoimmune manifestations, including enterocolitis, immune cytopenia, endocrinopathies, and systemic lupus erythematosus.STAT1-GOF mutations are highly penetrant with a median age at onset of 1  year and oft...
Source: Journal of Clinical Immunology - Category: Allergy & Immunology Source Type: research
Autoimmune manifestations after allogeneic hematopoietic stem cell transplantation (AHSCT) are rare and poorly understood due to the complex interplay between the reconstituting immune system and transplant-associated factors. While autoimmune manifestations following AHSCT have been observed in children with graft-versus-host disease (GvHD), an alloimmune process, they are distinct from the latter in that they are generally restricted to the hematopoietic compartment, i.e., autoimmune hemolytic anemia, thrombocytopenia, and/or neutropenia. Autoimmune cytopenias in the setting of ASHCT represent a donor against donor immun...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Chronic graft-versus-host disease (GVHD) occurs in 30% to 70% of allogeneic stem cell transplant recipients and represents a major cause of morbidity and late mortality. [1,2] Chronic GVHD is a syndrome that can affect the skin, mouth, eye, muscle, fascia, joints, gastrointestinal tract (GIT), liver and lungs resembling autoimmune and other immunologic disorders such as scleroderma, Sjogren's syndrome, primary biliary cirrhosis, bronchiolitis obliterans or immune cytopenia. [1,2]
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
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