Clinical validation and study of stem cell transplantation in treatment of vitiligo.
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ABSTRACT:
SUBMITTER: Liu J
PROVIDER: S-EPMC10615963 | biostudies-literature | 2023 Dec
REPOSITORIES: biostudies-literature
Publications
Clinical validation and study of stem cell transplantation in treatment of vitiligo.
Liu Jingwei J Liu Shiyu S Guo Min M Yong Miao M Hu Zhiqi Z
Archives of dermatological research 20230907 10
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Project description:ObjectiveAutologous transplantation of epidermal cells has been used increasingly to treat vitiligo patients and is a simple, safe, and relatively efficient method. However, the outcome is not always satisfactory, and some patients show less or no response to this treatment. This study was evaluated to identify genes expressed differently among responders and non-responders to cell transplantation to find potential markers that could predict 'patients' responses to this type of cell therapy.Materials and methodsEleven stable vitiligo patients who received autologous epidermal cell transplantation were included in this clinical trial study. Before cell transplantation, skin samples were obtained from the recipient's vitiligo lesions. After epidermal cell transplantation, patients were followed for at least six months to assess the response to epidermal cell injection. RNA sequencing was used to determine potential gene expression profile differences between responder and non-responder vitiligo patients.ResultsThe RNA sequencing results showed differences in expression levels of 470 genes between the skin specimens of responder versus non-responder patients. There were 269 up-regulated genes and 201 down-regulated genes. Upregulated genes were involved in processes, such as Fatty Acid Omega Oxidation. Down-regulated genes were related to PPAR signaling pathway, and estrogen signaling pathway. Among the most differentially expressed genes (DEGs) with the most altered RNA expression levels in responders versus non-responder patients, we selected three genes (up-regulated genes KRTAP10-11 and down-regulated genes IP6K2 and C9) as potential biomarkers, which are involved in associated pathways.ConclusionBased on our findings, it is estimated that proposed genes might predict the response of vitiligo patients to cell therapy. However, further studies are required to clarify the role of these genes in pathogenesis and to characterize gene expression in a larger number of vitiligo patients in the context of epidermal cell transplantation therapy (registration number: IRCT201508201031N16).
| S-EPMC9315216 | biostudies-literature
Project description:Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) is one of approximately 50 known lysosomal storage disorders. MPS VI is characterized by an absence or deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) resulting in accumulation of dermatan sulfate. Prior to the availability of enzyme replacement therapy (ERT), the clinical management of MPS VI was limited to supportive care and allogeneic hematopoietic stem cell transplantation (HSCT); however, due to the rarity of this disease, little is known about the long-term outcomes of HSCT for MPS VI. The following retrospective study was performed using aggregate data gathered by the Center for International Blood and Marrow Transplant Research (CIBMTR) between 1982 and 2007 to determine survival probability for patients with MPS VI following allogeneic HSCT. This analysis identified 45 MPS VI patients with a median age of 5 years (range, 1-22 years) at the time they received an allogeneic HSCT. Cumulative incidence (95% CI) of acute graft-vs.-host disease at 100 days was 36% (21-53%). Probability of survival was 78% (65-89%) at 100 days and 66% (52-79%) at 1 and 3 years. While these data are based upon small numbers of recipients, they represent the largest series to date and may help clinicians assess the relative risks and benefits of currently available therapies.
| S-EPMC3367500 | biostudies-literature
Project description:BackgroundPatient-reported outcome measures are rarely used in vitiligo trials. The Vitiligo Noticeability Scale (VNS) is a new patient-reported outcome measure assessing how 'noticeable' vitiligo patches are after treatment. The noticeability of vitiligo after treatment is an important indicator of treatment success from the patient's perspective.ObjectivesTo evaluate the construct validity, acceptability and interpretability of the VNS.MethodsClinicians (n = 33) and patients with vitiligo (n = 101) examined 39 image pairs, each depicting a vitiligo lesion pre- and post-treatment. Using an online questionnaire, respondents gave a global assessment of treatment success and a VNS score for treatment response. Clinicians also estimated percentage repigmentation of lesions (< 25%; 25-50%; 51-75%; > 75%). Treatment success was defined as 'yes' on global assessment, a VNS score of 4 or 5, and > 75% repigmentation. Agreement between respondents and the different scales was assessed using kappa (κ) statistics.ResultsVitiligo Noticeability Scale scores were associated with both patient- and clinician-reported global treatment success (κ = 0·54 and κ = 0·47, respectively). Percentage repigmentation showed a weaker association with patient- and clinician-reported global treatment success (κ = 0·39 and κ = 0·29, respectively). VNS scores of 4 or 5 can be interpreted as representing treatment success. Images depicting post-treatment hyperpigmentation were less likely to be rated as successful.ConclusionsThe VNS is a valid patient-reported measure of vitiligo treatment success. Further validation of the VNS is required, using larger sets of clinical pre- and post-treatment images, affecting a wider range of anatomical sites.
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Project description:This review highlights problems related to translation of advanced therapy medicinal products (ATMPs) from bench to bedsite. Regenerative medicine within the current regulatory frame reveals common hitches in the course of development, translation, and clinical application. This paper suggests outlining a path from the few examples of successfully approved vs unsuccessful advanced therapies.In the multitude of ongoing studies, few of them achieved positive results with a final treatment available to patients; this result was possible due to multidisciplinary teams working together from the beginning of the development and during the hard route to standardization and clinical application.The root of success of an advanced therapy requires not only the inescapable scientific and biological knowledge but also requires several contributions as regulatory, ethical, medical, and bio-engineering expertise, from the real beginning. A strong scientific rationale and an integrated network of expertises would contribute to a successful investment of available resources in advanced therapy medicinal products and to a greater confidence in future medicine.
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Project description:The aim of this study is to assess the Fecal Microbiota Transplantation (FMT) efficacy in the prevention of allogeneic hematopoietic stem cell transplantation (allo-HSCT) complications and particularly Graft versus Host Disease (GvHD).
The hypothesis of this study is that allogeneic FMT may improve outcomes of these patients.
| 2381286 | ecrin-mdr-crc
Project description:Stem cells (SCs) play a major role in advanced fields of regenerative medicine and other research areas. They are involved in the regeneration of damaged tissue or cells, due to their self-renewal characteristics. Tissue or cells can be damaged through a variety of diseases, including hematologic and nonhematologic malignancies. In regard to this, stem-cell transplantation is a cellular therapeutic approach to restore those impaired cells, tissue, or organs. SCs have a therapeutic potential in the application of stem-cell transplantation. Research has been focused mainly on the application of hematopoietic SCs for transplantation. Cord blood cells and human leukocyte antigen-haploidentical donors are considered optional sources of hematopoietic stem-cell transplantation. On the other hand, pluripotent embryonic SCs and induced pluripotent SCs hold promise for advancement of stem-cell transplantation. In addition, nonhematopoietic mesenchymal SCs play their own significant role as a functional bone-marrow niche and in the management of graft-vs-host disease effects during the posttransplantation process. In this review, the role of different types of SCs is presented with regard to their application in SC transplantation. In addition to this, the therapeutic value of autologous and allogeneic hematopoietic stem-cell transplantation is assessed with respect to different types of leukemia. Highly advanced and progressive scientific research has focused on the application of stem-cell transplantation on specific leukemia types. We evaluated and compared the therapeutic potential of SC transplantation with various forms of leukemia. This review aimed to focus on the application of SCs in the treatment of leukemia.
| S-EPMC7493021 | biostudies-literature
Project description:BackgroundHuman umbilical cord mesenchymal stem cells (HUC-MSCs) present in the umbilical cord tissue are self-renewing and multipotent. They can renew themselves continuously and, under certain conditions, differentiate into one or more cell types constituting human tissues and organs. HUC-MSCs differentiate, among others, into osteoblasts, chondrocytes, and adipocytes and have the ability to secrete cytokines. The possibility of noninvasive harvesting and low immunogenicity of HUC-MSCs give them a unique advantage in clinical applications. In recent years, HUC-MSCs have been widely used in clinical practice, and some progress has been made in their use for therapeutic purposes.Main bodyThis article describes two aspects of the clinical therapeutic effects of HUC-MSCs. On the one hand, it explains the benefits and mechanisms of HUC-MSC treatment in various diseases. On the other hand, it summarizes the results of basic research on HUC-MSCs related to clinical applications. The first part of this review highlights several functions of HUC-MSCs that are critical for their therapeutic properties: differentiation into terminal cells, immune regulation, paracrine effects, anti-inflammatory effects, anti-fibrotic effects, and regulating non-coding RNA. These characteristics of HUC-MSCs are discussed in the context of diabetes and its complications, liver disease, systemic lupus erythematosus, arthritis, brain injury and cerebrovascular diseases, heart diseases, spinal cord injury, respiratory diseases, viral infections, and other diseases. The second part emphasizes the need to establish an HUC-MSC cell bank, discusses tumorigenicity of HUC-MSCs and the characteristics of different in vitro generations of these cells in the treatment of diseases, and provides technical and theoretical support for the clinical applications of HUC-MSCs.ConclusionHUC-MSCs can treat a variety of diseases clinically and have achieved good therapeutic effects, and the development of HUC-MSC assistive technology has laid the foundation for its clinical application.
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Project description:PurposeThe aim of this study was to evaluate the therapeutic efficacy and safety of mesenchymal stem cells (MSCs) for the treatment of patients with knee osteoarthritis (OA).MaterialsWe performed a meta-analysis of relevant published clinical studies. An electronic search was conducted for randomized controlled trials (RCTs) of MSC-based therapy in knee OA. The visual analogue scale (VAS), International Knee Documentation Committee (IKDC) form, Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), Lequesne algofunctional indices (Lequesne), Lysholm knee scale (Lysholm), Tegner activity scale (Tegner) and adverse events (AEs) were evaluated.ResultsEleven eligible trials with 582 knee OA patients were included in the present meta-analysis. We demonstrated that MSC treatment could significantly decrease VAS and increase IKDC scoresafter a 24-month follow-up compared with controls (P<0.05). MSC therapy also showed significant decreases in WOMAC and Lequesne scores after the 12-month follow-up (P<0.01). Analysis of Lysholm (24-month) and Tegner (12- and 24-month) scores also demonstrated favorable results for MSC treatment (P<0.05).ConclusionOverall, MSC transplantation treatment was shown to be safe and has great potential as an efficacious clinical therapy for patients with knee OA.
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Project description:Organ transplantation is the ultimate treatment for end-stage diseases such as heart and liver failure. However, the severe shortage of donor organs has limited the organ transplantation progress. Xenogeneic stem cell transplantation provides a new strategy to solve this problem. Researchers have shown that xenogeneic stem cell transplantation has significant therapeutic effects and broad application prospects in treating liver failure, myocardial infarction, advanced type 1 diabetes mellitus, myelosuppression, and other end-stage diseases by replacing the dysfunctional cells directly or improving the endogenous regenerative milieu. In this review, the sources, problems and solutions, and potential clinical applications of xenogeneic stem cell transplantation will be discussed.
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Project description:Although autologous stem cell transplantation (auto-HCT) is the standard of care for patients with refractory/relapsed (R/R) classical Hodgkin's lymphoma (cHL), there is still a significant proportion of patients that relapse after the procedure. This review contemplates different treatment strategies for patients with cHL that relapse or progress after auto-HCT. Allogeneic stem cell transplantation (allo-HCT) has, for many years, been the only curative option for this group of patients. Although the advent of haploidentical donors has allowed for the possibility to allograft almost all patients that are in need of it and to eventually improve historical results, allo-HCT is still associated with substantial morbidity and mortality. Brentuximab vedotin (BV) is an antibody drug conjugate that binds to CD30 antigen; BV is able to give up to 34% metabolic complete remissions (mCR) in HL patients that fail auto-HCT. Unleashing the immune system with PD-1 inhibitors has resulted in remarkable responses in a number of malignancies, including HL. Nivolumab and pembrolizumab offer a 20%-25% mCR and 40%-50% partial remissions, with an acceptable safety profile. R/R cHL do have several options nowadays that, without any doubt, have significantly improved the long-term outcome of this hard-to-treat population.
| S-EPMC7290321 | biostudies-literature