• Journal of mucopolysaccharidosis and rare diseases
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• v.3 no.2
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• pp.37-40
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• 2017

Mucopolysaccharidosis (MPS) is caused by accumulation of the glycosaminoglycans in all tissues due to decreased activity of the lysosomal enzyme. Patients exhibit multisystemic signs and symptoms in a chronic and progressive manner, especially with changes in the skeleton, cardiopulmonary system, central nervous system, cornea, skin, liver, and spleen. In the past, treatment of MPS was limited to enzyme replacement therapy (ERT). The outcome for affected patients improved with the introduction of new technologies as hematopoietic stem cell transplantation, relegated to specific situations after ERT became available. Intrathecal ERT may be considered in situations of high neurosurgical risk but still it is experimental in humans. New insights on the pathophysiology of MPS disorders are leading to alternative therapeutic approaches, as gene therapy, inflammatory response modulators and substrate reduction therapy. In this paper, we will highlight the recent novel treatment and clinical trials for MPS and discuss with the goal of fostering an understanding of this field.

• Pediatric Gastroenterology, Hepatology & Nutrition
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• v.22 no.1
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• pp.80-85
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• 2019

Purpose: Abernethy malformation is a rare condition, which was first described in 1793 as a congenital extrahepatic porto-systemic shunt (CEPS) directing splanchnic blood flow into the inferior vena cava. Eighty cases have been published so far that reported CEPS, while in Korea, very few cases have been reported. Through this study, we present 6 cases of patients diagnosed with CEPS at Samsung Medical Center and compare these with other such cases published in France and China. Methods: We reviewed clinical, laboratory, and imaging data of 6 children with CEPS in our pediatric clinic between 2004 and 2017. Results: A total of 6 children with CEPS was included in this study, namely, one with type 1a, two with type 1b, and three with type 2 CEPS. The most common presenting symptom was gastrointestinal bleeding (50.0%). Therapeutic interventions included shunting vessel ligation (16.7%) in type 2 CEPS and liver transplantation (16.7%) in type 2 CEPS patient with suddenly developed hepatic encephalopathy. Conclusion: There is no consensus guideline for the optimal management of patients with CEPS. Large-sample studies regarding CEPS are needed to evaluate the characteristics of patients with CEPS and determine the treatment guideline for CEPS.

• Journal of Life Science
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• v.16 no.6
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• pp.1060-1065
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• 2006

Non-invasive evaluation of liver function in animal models remains a challenge. Hepatoscintigraphy provides information about changes in liver size and shape, and enables to understand general liver function. Futhermore it is readily used to diagnosis complications of liver transplantation like hepatitis, rejections and biliary complications. In this study, we investigated the usefulness of evaluating the liver function in miniature pigs with $^{99m}Tc-Tin$ colloid and $^{99m}Tc-DISIDA$ which are the most commonly used radiopharmaceuticals in human medicine. In result, $^{99m}Tc-Tin$ colloid was uptaked in lung, liver, gastric wall and kidney in miniature pigs. And $^{99m}Tc-DISIDA$ showed continuous uptake images of heart, lung, liver, gallbladder and duodenum, and it was similar to human's. Therefore we could conclude $^{99m}Tc-Tin$ colloid would not be suitable for evaluating hepatic function because of it's nonspecific affinity, however $^{99m}Tc-DISIDA$ scintigraphy would be an effective method for detecting hepatobiliary function in miniature pigs.

• Pediatric Gastroenterology, Hepatology & Nutrition
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• v.8 no.2
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• pp.177-193
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• 2005

Purpose: To identify genes specifically expressed in biliary atresia, we compared the patterns of gene expression between biliary atresia and neonatal hepatitis syndrome using cDNA microarray analysis. Methods: Liver tissues were taken from livers of 11 patients (7 patients with biliary atresia and four with neonatal hepatitis) with neonatal cholestasis by needle biopsy. Normal control could be obtained from donor liver tissue during living-related liver transplantation. Total RNA was extracted from each samples and reversely transcribed to make cDNA. Then fluorescent cDNA were pooled and hybridized to the clones on the microarray. Fluorescence intensities at the immobilized targets were measured. Utilizing cDNA arrays of 4.7 K human genes, gene expression profiles were analyzed. Results: Among 4,700 microarray clones, 17 cDNA clones were significantly over-expressed in all 11 patients with neonatal cholestasis, while 20 clones were significantly decreased. Genome-wide expression analysis was carried out in livers obtained at the time of diagnosis. We could identify 49 genes, in which there showed differential expression between biliary atresia and neonatal hepatitis syndrome. Conclusion: This study shows the pattern of differentially expressed genes in biliary atresia and neonatal hepatitis syndrome. We believe that this study can contribute to the understanding of pathogenesis of neonatal cholestasis.

• Korean Journal of Clinical Laboratory Science
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• v.51 no.3
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• pp.370-378
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• 2019

The hepatic ischemic model has recently been widely used for the epidemiological study of ischemic reperfusion injury. This study was carried out to investigate the protective effect of vanillin, which is known to have antioxidant and anti-inflammatory effects, against hepatic and renal injury using an ischemia-reperfusion rat model, and we also investigated the mechanism related to vanillins' protective effect. The test material was administered at a concentration of 100 mg/kg for 3 days, followed by ligation of the liver for 60 minutes to induce ischemia reperfusion. As control groups, there was a negative control, sham control and ischemia-reperfusion-only ischemia reperfusion control, and the controls groups were compared with the drug administration group. In the vanillin group, aspartate aminotransferase (AST) and alanine aminotransferase (ALT) activities were significantly inhibited compared with the AST and ALT activities of the ischemia-reperfusion group, and histopathological examination showed significant reduction of both inflammation and necrosis. The malondialdehyde (MDA) and superoxide dismutase (SOD) levels were significantly different from the ischemia-reperfusion group. In conclusion, vanillin showed a hepatocyte protective action by alleviating the cellular inflammation and cell necrosis caused by hepatic ischemia-reperfusion, and vanillin mitigated inflammatory changes in the kidney glomeruli and distal tubules. The protective effect is considered to be caused by vanillin's antioxidant function. Further studies such as on cell death and possibly vanillin's same effect on damaged tissue will be necessary for clinical applications such as organ transplantation.

• Korean Journal of Radiology
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• v.22 no.8
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• pp.1289-1299
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• 2021

Objective: We aimed to evaluate the usefulness of arterial subtraction images for predicting the viability of hepatocellular carcinoma (HCC) after locoregional therapy (LRT) using gadoxetic acid-enhanced MRI and the Liver Imaging Reporting and Data System treatment response (LR-TR) algorithm. Materials and Methods: This study included 90 patients (mean age ± standard deviation, 57 ± 9 years) who underwent liver transplantation or resection after LRT and had 73 viable and 32 nonviable HCCs. All patients underwent gadoxetic acid-enhanced MRI before surgery. Two radiologists assessed the presence of LR-TR features, including arterial phase hyperenhancement (APHE) and LR-TR categories (viable, nonviable, or equivocal), using ordinary arterial-phase and arterial subtraction images. The reference standard for tumor viability was surgical pathology. The sensitivity of APHE for diagnosing viable HCC was compared between ordinary arterial-phase and arterial subtraction images. The sensitivity and specificity of the LR-TR algorithm for diagnosing viable HCC was compared between the use of ordinary arterial-phase and the use of arterial subtraction images. Subgroup analysis was performed on lesions treated with transarterial chemoembolization (TACE) only. Results: The sensitivity of APHE for viable HCCs was higher for arterial subtraction images than ordinary arterial-phase images (71.2% vs. 47.9%; p < 0.001). LR-TR viable category with the use of arterial subtraction images compared with ordinary arterial-phase images showed a significant increase in sensitivity (76.7% [56/73] vs. 63.0% [46/73]; p = 0.002) without significant decrease in specificity (90.6% [29/32] vs. 93.8% [30/32]; p > 0.999). In a subgroup of 63 lesions treated with TACE only, the use of arterial subtraction images showed a significant increase in sensitivity (81.4% [35/43] vs. 67.4% [29/43]; p = 0.031) without significant decrease in specificity (85.0% [17/20] vs. 90.0% [18/20]; p > 0.999). Conclusion: Use of arterial subtraction images compared with ordinary arterial-phase images improved the sensitivity while maintaining specificity for diagnosing viable HCC after LRT using gadoxetic acid-enhanced MRI and the LR-TR algorithm.

• Asian Pacific Journal of Cancer Prevention
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• v.14 no.8
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• pp.4583-4589
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• 2013

Objective: This study aimed to investigate the effect of multimodality treatment of advanced paediatric hepatoblastoma (HB) and the factors affecting prognosis. Methods: A total of 35 children underwent multimodality treatments consisting of chemotherapy, surgery, interventional therapy, and autologous peripheral blood stem cell transplantation. The patients were followed up every month. Results: Serum AFP levels in 33 out of 35 patients in this study were significantly increased (P = 0.0002). According to the statistical scatter plot, the values of serum AFP on the 25th, 50th, and 75th percentages were 1,210, 1,210 and 28,318 ng/dl, respectively. Of the 35 cases, 21 were stage IV. 18 cases were treated with systemic chemotherapy before surgery, and 3 cases with locally interventional chemotherapy before surgery. Statistical analysis showed that the preferred interventional treatment affected prognosis, and that there was a statistically significant difference (P = 0.024). Some 33 patients completed the follow-up, of which 17 were in complete remission (CR), 5 were in partial remission (PR), 1 became disease progressive (DP), and 10 died. The remission and overall survival rates were 66.7% (22/33) and 69.7% (23/33), respectively. Patients with the mixed HB phenotypes had worse prognoses than the epithelial phenotype (P < 0.001), and patients in stage IV had a lower survival rate than those in stage III (P < 0.001). Conclusion: Multimodality treatment can effectively improve remission rate and prolong the survival of children with advanced HB. In addition, alpha-fetoprotein (AFP), a tumor marker of liver malignant tumors, HB pathological classification, and staging are highly useful in predicting prognosis.

• Korean Journal of Clinical Laboratory Science
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• v.50 no.4
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• pp.391-398
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• 2018

Mesenchymal stem cells (MSCs) are an attractive resource for refractory patients because of their anti-inflammatory/immunomodulatory capability and multi-lineage differentiation potential. The transplantation of MSCs has led to positive results in preclinical and clinical application to various diseases, including autoimmune disease, cardiovascular disease, cancer, liver cirrhosis, and ischemic stroke. On the other hand, studies have shown that paracrine factors, not direct cell replacement for damaged cells or tissue, are the main contributors in MSC-based therapy. More recently, evidence has indicated that MSC-derived exosomes play crucial roles in regulating the paracrine factors that can mediate tissue regeneration via transferring nucleic acids, proteins, and lipids to the local microenvironment and cell-to-cell communication. The use of these exosomes is likely to be beneficial for the therapeutic application of MSCs because their use can avoid harmful effects, such as tumor formation involved in cell transplantation. Therefore, therapeutic applications using MSC-derived exosomes might be safe and efficient strategies for regenerative medicine and tissue engineering. This review summarizes the recent advances and provides a comprehensive understanding of the role of MSC-derived exosomes as a therapeutic agent.

• Journal of Hospice and Palliative Care
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• v.20 no.3
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• pp.177-187
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• 2017

Purpose: From August 2017, hospice-palliative care (HPC) will be provided to patients with acquired immunodeficiency syndrome (AIDS), chronic obstructive pulmonary disease (COPD), and liver cirrhosis in Korea. To contribute to building a non-cancer (NC) hospice-palliative care model, NC specialists were interviewed regarding the goals, details, and provision methods of the model. Methods: Four physicians specializing in HPC of cancer patients formulated a semi-structured interview with questions extracted from literature review of 85 articles on NC HPC. Eleven NC disease specialists were interviewed, and their answers were analyzed according to the qualitative content analysis process. Results: The interviewees said as follows: It is difficult to define end-stage NC patients. HPC for cancer patients and that for NC patients share similar goals and content. However, emphasis should be placed on alleviating other physical symptoms and emotional care rather than pain control. Timing of the care provision should be when patients are diagnosed as "end stage". Special issues should be considered for each NC disease (e.g., use of anti-retroviral drugs for AIDS patients, oxygen supply for COPD patients suffering from dyspnea, liver transplantation for patients with liver cirrhosis) and education should be provided to healthcare professionals. NC patients tend to negatively perceive HPC, and the government's financial assistance is insufficient. Conclusion: It is necessary to define end-stage NC patients through in-depth discussion to minimize issues that will likely accompany the expansion of care recipients. This requires cooperation between medical staff caring for NC patients and HPC givers for cancer patients.

• Preventive Nutrition and Food Science
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• v.5 no.3
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• pp.126-130
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• 2000

To enhance the antitumor activity of Chinese cabbage kimchi, four kinds of kimchi, which ere differently prepared in kinds and levels of sub-ingredients, were fermented at 15$^{\circ}C$ for 1 day and then at 5$^{\circ}C$ up to pH 4.3. The solid tumor formation, hepatic glutathione S-transferase activity and glutathione contents in the liver, and natural killer (NK) cell activity of spleen were determined from the sarcoma-180 cell injected Balb/c mice that were treated with methanol extracts of the kimchi samples. Kimchi IV, prepared with organically cultivated Chinese cabbage, red pepper powder, garlic, Chinese pepper powder mustard leaf and heat processed salt (Gueun salt), reduced the tumor formation by 39.3% compared to the sarcoma-180 cell treated group, resulting in the smallest tumor weight. Methanol extracts of the kimchi III and kimchi IV recovered the activities of hepatic glutathione S-transferase(GST) that was decreased by the transplantation of the sarcoma-180 cells to th mice. The injections of methanol extracts of kimchi II and kimchi IV increased glutathione contents in sarcoma-180 cells treated mice. The methanol extract of kimchi IV increased the natural killer (NK) cell activity of spleen lymphocytes a more effectively (p<0.05) than those the other kimchi samples. These results suggest that the anticancer activities of kimchi can be increased by changing the kinds and levels of sub-ingredients.