BACKGROUND Hereditary angioedema (HAE) is a rare, potentially fatal disorder that may present solely with non-specific gastrointestinal symptoms. CASE SUMMARY We report a 30-year-old woman with a 10-year history of recurrent, unexplained abdominal pain and vomiting, without any cutaneous edema. The initial presentation suggested acquired angioedema because of the lack of a clear family history. However, the early age of onset prompted genetic testing. Lower endoscopy performed during an acute attack provided crucial evidence, revealing marked edema of the terminal ileum. Laboratory tests demonstrated low complement C4 levels and severely reduced C1 esterase inhibitor activity. Genetic analysis revealed of a novel heterozygous frameshift mutation in SERPING1 (p.Thr285Tyrfs*20, c.852dupT), establishing the diagnosis of HAE type 1. CONCLUSION HAE can be fatal from laryngeal edema and intestinal involvement and should be considered in patients with recurrent abdominal pain.
In light of the rapidly increasing number of people with diabetes worldwide and the resulting surge in medical costs, we have attempted to reassess the effectiveness of inexpensive and readily available balneotherapy on diabetes and obesity by summarizing recent related papers. For this analysis, 22 studies were selected through searches of multiple databases, including MEDLINE, Embase, CENTRAL, Medical Central Journal Web, and Web of Science. The selected studies consisted of 12 full-body baths, six on foot baths, two on saunas, and two on drinking water therapies. Among participants receiving full-body bath therapy, reductions in blood glucose levels and weight loss were reported. Moreover, immersion in hot water at high temperatures appeared to be more effective. In balneotherapy, using water containing solutes was more effective than using plain hot water. Saunas were effective in reducing participants’ blood glucose levels and weight. Hot-spring foot baths were effective for diabetic complications such as peripheral circulatory disorders and peripheral neuropathy. Drinking water containing sulfur or bicarbonate was effective in reducing the blood glucose levels. The previously reported effects of hot-spring therapy on diabetes and obesity were reconfirmed, and it became evident that the solutes present in hot-spring water contributes to its therapeutic effects. However, in the present analysis, only three studies included more than 100 participants. Although larger studies are desirable, hot-spring interventions are not well-suited to large-scale trials requiring uniform conditions. Therefore, it is essential to continue collecting new findings and comprehensively evaluating the therapeutic effects of balneotherapy, as demonstrated in this review.
562 Background: Systemic therapy for hepatocellular carcinoma (HCC) has rapidly evolved, and immune checkpoint inhibitor (ICI)-based regimens are now widely used as first-line treatment. However, in patients at risk of gastrointestinal bleeding or those contraindicated for ICIs, tyrosine kinase inhibitors (TKIs) such as sorafenib (SOR) and lenvatinib (LEN) remain clinically important options. While randomized trials and retrospective studies have compared these agents, prospective real-world data are limited. This study aimed to evaluate outcomes of SOR and LEN as first-line therapy for unresectable HCC (uHCC) in a multicenter prospective observational setting. Methods: One hundred sixty-seven patients with uHCC initiating SOR (n=32) or LEN (n=135) at 20 institutions of the Kyushu Study Group of Clinical Cancer (KSCC) were enrolled. Eligible patients had no prior systemic therapy. The primary endpoint was overall survival (OS). Secondary endpoints included progression-free survival (PFS), objective response rate (ORR), disease control rate (DCR), and safety. Tumor response was assessed by RECIST v1.1 and mRECIST. To adjust for baseline differences, inverse probability of treatment weighting (IPTW) based on propensity scores was applied. Results: In the unadjusted cohort, OS was significantly longer in the SOR group than in the LEN group (median 35.1 vs. 15.7 months, HR 0.49, p=0.0088). However, after IPTW adjustment, OS was comparable (HR 1.13, 95% CI 0.68–1.88). Median PFS was 4.8 months for SOR and 5.4 months for LEN. IPTW-adjusted analysis showed significantly shorter PFS with SOR (median 4.1 vs. 5.5 months, HR 1.50, p=0.047). ORR was significantly higher in the LEN group compared with the SOR group (RECIST v1.1: 30.7% vs. 7.8%, p=0.045; mRECIST: 43.3% vs. 14.7%, p=0.036). DCR was also numerically superior with LEN. Grade ≥3 adverse events occurred in 31.3% (SOR) and 45.2% (LEN), with hand-foot syndrome and proteinuria being the most frequent severe toxicities, for SOR and LEN respectively. Conclusions: This prospective multicenter observational study demonstrated that SOR and LEN achieve comparable OS in the real-world first-line treatment of uHCC. LEN showed superior PFS and tumor response, while SOR maintained favorable tolerability. These findings highlight the continued clinical relevance of both TKIs and emphasize the importance of individualized therapy selection based on tumor burden, biomarker profiles, and patient-specific tolerability. Clinical trial information: 000038867 .
BACKGROUND & AIMS:Direct-acting antiviral agents (DAA)-mediated HCV cure correlates with better outcomes, but there are insufficient data on detailed mortality-related risk factors after cure. This study sought to clarify mortality and associated risk factors post-HCV cure. METHODS:The study included HCV patients with sustained virological response following DAA (DAA-SVR) from 39 REAL-C centres in North America, Europe and Asia-Pacific. The primary outcome was all-cause mortality in DAA-SVR patients. Mortality rate per 1000 patient-years (PY) was calculated as the number of deaths divided by total PY multiplied by 1000. RESULTS:A total of 10 034 DAA-SVR patients (stratified by cirrhosis status: 5611 non-cirrhosis, 4153 compensated, 270 decompensated) were included. With a median follow-up of 4.76 PY, 4.9% (491) died. The all-cause mortality rates were 6.2, 13.1, 60.0 and 10.4 per 1000 PY for patients without cirrhosis, compensated and decompensated cirrhosis, and overall patients, respectively. The 5-year cumulative survival was 95.1% (94.5%-95.6%) overall, with the lowest rate of 73.9% (67.0%-79.5%) in decompensated cirrhosis. Non-liver-related death was the main cause in non-cirrhosis (non-liver-related vs. liver: 5.2 vs. 0.8 per 1000 PY)/compensated cirrhosis (8.2 vs. 4.8 per 1000 PY), while liver-related death was dominant in decompensated cirrhosis (24.7 vs. 33.8 per 1000 PY). Risk factors for higher mortality included age > 65 (3.2-fold), male (1.5-fold), cirrhosis (decompensated 7.6-fold) and baseline DM (1.5-fold). CONCLUSION:This study showed significant age, sex, fibrosis stage and DM differences in mortality and causes among DAA-SVR patients. It provided granular subgroup data for precision medicine to support individualised care, future modelling studies and public health planning.
Delayed emergence from general anesthesia has multiple etiologies; in rare cases, it may unmask an unrecognized neurodegenerative disorder. A 64-year-old man underwent laparoscopic cholecystectomy under general anesthesia. No neurological abnormalities were identified preoperatively. After surgery, response to verbal stimuli was inadequate; however, other extubation criteria were met and he was extubated. Approximately 45 min later, rapid nystagmus-like eye movements consistent with opsoclonus developed, initially oscillating horizontally and then vertically, and persisted for about six minutes. A similar episode recurred 125 min after extubation. Head computed tomography showed no acute lesions but revealed marked cerebellar and brainstem atrophy. Subsequent neurological evaluation, including magnetic resonance imaging demonstrating the “hot cross bun” sign, fulfilled the Movement Disorder Society diagnostic criteria for clinically established multiple system atrophy with predominant parkinsonism. In unexplained delayed emergence from general anesthesia, opsoclonus should prompt evaluation for an underlying neurodegenerative disorder.
