METHODS: Mice received 3.5% DSS for 7 days to establish IBD models. Intraperitoneal STV-Na was given 2 days before DSS and lasted for 9 days. Commercially available drugs used in treating IBDs (5-aminosalicylic acid, dexamethasone, and infliximab) were used as positive controls. Samples were collected 7 days after colitis induction. Histopathological score, biochemical parameters, molecular biology methods, and metabolomics were used for evaluating the therapeutic effect of STV-Na.
RESULTS: Our data revealed that STV-Na could significantly alleviate colon inflammation in mice with colitis. Specifically, STV-Na treatment improved body weight loss, increased colon length, decreased histology scores, and restored the hematological parameters of mice with colitis. The untargeted metabolomics analysis revealed that metabolic profiles were restored by STV-Na treatment. Furthermore, STV-Na therapy suppressed the number of CD68 macrophages and F4/80 cell infiltration. And STV-Na suppressed M1 and M2 macrophage numbers along with the mRNA expressions of proinflammatory cytokines. Moreover, STV-Na administration increased the number of regulatory T (Treg) cells while decreasing Th1/Th2/Th17 cell counts in the spleen. Additionally, STV-Na treatment restored intestinal barrier disruption in DSS-triggered colitis tissues by ameliorating the TJ proteins, increasing goblet cell proportions, and mucin protein production, and decreasing the permeability to FITC-dextran, which was accompanied by decreased plasma LPS and DAO contents.
CONCLUSION: These results indicate that STV-Na can ameliorate colitis by modulating immune responses along with metabolic reprogramming, and could therefore be a promising therapeutic strategy for IBDs.
METHODS: Patients with IIM, excluding inclusion body myositis, were recruited along with age- and sex-matched healthy controls (HC). All participants underwent muscle ultrasound and clinical assessments. Six limb muscles were unilaterally scanned using a standardized protocol, measuring muscle thickness (MT) and echo intensity (EI). Results were compared with HC, and correlations were made with outcome measures.
RESULTS: Twenty IIM patients and 24 HC were recruited. The subtypes of IIM were dermatomyositis (6), necrotizing myositis (6), polymyositis (3), antisynthetase syndrome (3), and nonspecific myositis (2). Mean disease duration was 8.7 ± 6.9 years. There were no significant differences in demographics and anthropometrics between patients and controls. MT of rectus femoris in IIM patients was significantly lower than HC. Muscle EI of biceps brachii and vastus medialis in IIM patients were higher than HC. There were moderate correlations between MT of rectus femoris and modified Rankin Scale, Physician Global Activity Assessment, and Health Assessment Questionnaire, as well as between EI of biceps brachii and Manual Muscle Testing-8.
DISCUSSION: Muscle ultrasound can detect proximal muscle atrophy and hyperechogenicity in patients with IIM. The findings correlate with clinical outcome measures, making it a potential tool for evaluating disease activity of patients with IIM in the late phase of the disease.
METHODS: Among the >2,500 patients in a local muscle biopsy database, patients with LSM were identified and their genomic DNA were extracted from muscle samples and peripheral blood. All 13 exons of the electron-transfer flavoprotein dehydrogenase gene (ETFDH) were subsequently sequenced. Fifty controls were included to determine the prevalence of identified mutations in the normal population.
RESULTS: Fourteen (82%) of the 17 LSM patients had MADD with ETFDH mutations. Twelve (86%) were Chinese and two were Malay sisters. Other unrelated patients reported that they had no relevant family history. Nine (64%) were females. The median age at onset was 18.5 years (interquartile range=16-37 years). All 14 demonstrated proximal limb weakness, elevated serum creatine kinase levels, and myopathic changes in electromyography. Three patients experienced a metabolic crisis at their presentation. Sanger sequencing of ETFDH revealed nine different variants/mutations, one of which was novel: c.998A>G (p.Y333C) in exon 9. Notably, 12 (86%) patients, including the 2 Malay sisters, carried a common c.250G>A (p.A84T) variant, consistent with the hotspot mutation reported in southern China. All of the patients responded well to riboflavin therapy.
CONCLUSIONS: Most of our Malaysian cohort with LSM had late-onset, riboflavin-responsive MADD with ETFDH mutations, and they demonstrated phenotypic and genetic features similar to those of cases reported in southern China. Furthermore, we report a novel ETFDH mutation and possibly the first ever MADD patients of Malay descent.
METHODS: Medical records of MG patients from 1976 to 2023 were reviewed retrospectively. Patients with pure ocular MG were excluded. Data on demographic, clinical characteristics, laboratory features, and outcomes were analyzed.
RESULTS: This study included 120 GMG patients. There was a slight preponderance of female patients over male patients (male:female ratio=1:1.3), with the age at onset exhibiting a bimodal distribution. Female patients peaked at a lower age (21-30 years) whereas male patients peaked at a higher age (61-70 years). Most (92%, 105 of 114) patients had positive anti-acetylcholine receptor antibodies. Five patients were also tested for anti-muscle-specific tyrosine kinase antibodies, with two showing positivity. Thymectomy was performed in 62 (52%) patients, of which 30 had thymoma, 16 had thymic hyperplasia, 7 had an involuted thymus, and 6 had a normal thymus. There were significantly more female patients (68% vs. 45%, p=0.011) with early-onset disease (<50 years old) and thymic hyperplasia (33% vs. 0%, p<0.025). Most (71%) of the patients had a good outcome based on the Myasthenia Gravis Foundation of America postintervention status. GMG patients with early-onset disease had a significantly better outcome than patients with a late onset in univariate (58% vs. 37%, p=0.041) and multivariate (odds ratio=4.68, 95% confidence interval=1.17-18.64, p=0.029) analyses.
CONCLUSIONS: Female patients with early-onset MG and thymic hyperplasia had significantly better outcomes, but only early-onset disease was independently associated with a good outcome. These findings are comparable with those of other studies.
METHODS: Between December 2020 to February 2023, parents of ARM and HD patients with and without DS aged 3-17 years who had undergone surgery > 12 months prior at four tertiary referral centers were recruited. We used the Pediatric Quality of Life Inventory™ (PedsQL™) Generic Core Scales, General Well-Being (GWB) Scale and Family Impact (FI) Module questionnaires, and the Rintala bowel function score (BFS).
RESULTS: There were 101 ARM, 9 (8.9%) of whom had DS; and 87 HD, of whom 6 (6.9%) had DS. Parent-reported Core scores in ARM and HD with DS were comparable to those without DS. However, ARM and HD with DS had worse scores in the FI Module and bowel function than those without DS.
CONCLUSION: Although parent-reported QOL in ARM and HD with DS is similar to those without DS, family impact and BFS are worse. Our findings are limited by small sample size in proportion of DS patients.
OBJECTIVE: The study aimed to examine the linguistic and construct validity of the Malay version of the Coping Self-Efficacy Scale (CSES-My) among parents of children with hematological cancer.
METHODS: The CSES-My was created through a sequential series of steps, starting with the translation of the original English version, followed by cultural adaptation, and then cognitive interviewing. The 26-item CSES-My was self-administered in a sample of parents of children with leukemia and lymphoma from October 2021 until February 2022. They were recruited via online or face-to-face methods. Exploratory factor analysis was performed to examine the construct validity of the CSES-My.
RESULTS: A total of 165 complete responses were analyzed. The scale has two factors, including Personal Coping and Social Coping, accounting for 58.3% of the variance. Personal Coping (19 items, Cronbach's alpha = 0.958) represented the respondents' self-efficacy for independently executing coping strategies, whether through solving problems or changing the way they think about the situation. Social Coping (6 items, Cronbach's alpha = 0.867) represented their confidence in executing coping strategies, which involved using social resources such as seeking external support, distraction, and avoiding loneliness.
CONCLUSIONS: The CSES-My had reliable and valid psychometric properties, providing evidence for its utility in evaluating coping self-efficacy among parents of children with leukemia or lymphoma in Malaysia. The CSES-My is a valuable tool for nurses and other healthcare professionals investigating coping self-efficacy in Malay-speaking populations, and it may also aid in the development of future coping interventions.
METHODS: We recruited ARM and HD patients aged 5-17 years and their parents at four tertiary referral centres between December 2020 to February 2023 who had corrective surgery done >12 months prior. Healthy controls were age-matched and gender-matched. They completed the Pediatric Quality of Life Inventory™ (PedsQL™) Generic Core Scales and General Well-Being (GWB) Scale. The questionnaires were administered in parallel parent-proxy-report and child self-report formats. Appropriate statistical analysis was performed with p
METHODS: The medical records of 24 patients who underwent repeat MUS surgery at a single tertiary center from January 2004 to February 2014 were reviewed. The types of MUS used for the repeat surgey were transobturator, retropubic and single incision slings. Objective cure was defined as no demonstrable involuntary leakage of urine during increased abdominal pressure in the absence of a detrusor contraction observed during filling cystometry, and subjective cure was defined as a negative response to Urogenital Distress Inventory six (UDI-6) question 3 during follow-up between 6 months and 1 year postoperatively. The change in the inclination angle between the urethra and pubic axis was measured with introital ultrasonography and the cotton swab test performed.
RESULTS: The objective and subjective cure rates were 79.2 % and 75 %, respectively. There were no differences in demographics between the patients with failure of surgery and those with successful surgery. Significant independent risk factors for failure of repeat MUS surgery were a change in cotton swab angle at rest and straining of <30° (OR 4.6, 95 % CI 2.5 - 7.9°), a change in inclination angle of <30° (OR 4.6, 95 % CI 2.5 - 7.9°), intrinsic sphincter deficiency (OR 3.4, 95 % CI 1.8 - 6.1) and a mean urethral closure pressure of <60 cm H2O (OR 2.9, 95 % CI 1.5 - 4.5). In one patient the bladder was perforated.
CONCLUSIONS: Repeat MUS surgery is safe and has a good short-term success rate, both objectively and subjectively, with independent risk factors for failure related to bladder neck hypomobility and poor urethral function.