- Research Article
- 10.1182/blood.2025031894
NXTAGE: a phase 1/2 study of NXT007 to assess safety, pharmacokinetics, and efficacy in hemophilia A without inhibitors.
- May 07, 2026
- Blood
- Keiji Nogami + 14 more +14
Publications from 2021 to 2026
Showing 10 of 1,734 papers
NXTAGE: a phase 1/2 study of NXT007 to assess safety, pharmacokinetics, and efficacy in hemophilia A without inhibitors.
AI-driven appendicular skeletal muscle mass index (ASMI) prediction and low muscle mass detection from routine hip X-rays: a novel opportunistic case finding tool.
Plasma <scp>Immunoglobulin A</scp> and intestinal ultrasound: Novel indicators for disease activity monitoring in inflammatory bowel disease—Insights from a Taiwan cohort
Abstract Intestinal ultrasound (IUS) is a non‐invasive imaging method used to evaluate bowel wall inflammation and monitor disease activity in inflammatory bowel disease (IBD) patients. Both the European Crohn's and Colitis Organization and the American Gastroenterological Association endorse IUS for monitoring disease activity and identifying complications at the point of care. However, to date, no published data are available regarding the use of IUS for IBD management in Taiwan. Similar to the specificity of IUS in visualizing gut inflammation, immunoglobulin A (IgA) is a key immunoglobulin central to gut mucosal immunity. Recent studies have explored the involvement of IgA in IBD, but the relationship between IgA levels and bowel wall inflammation was unknown. We enrolled 28 IBD patients, including 19 with ulcerative colitis (UC) and 9 with Crohn's disease (CD), from the outpatient clinic of China Medical University Medical Center. All patients underwent IUS between July 2023 and August 2024. Ultrasound scores were based on the Milan ultrasound criteria (MUC) for UC and the bowel ultrasound score (BUSS) for CD. Plasma levels of erythrocyte sedimentation rate, C‐reactive protein (CRP), albumin, IgA, partial Mayo score, and Crohn's disease activity index (CDAI) were collected at baseline, during treatment, and after 52 weeks of advanced therapy (Adalimumab, Vedolizumab, or Tofacitinib). IUS findings were closely correlated with disease activity. In UC, the MUC score was positively correlated with CRP ( R 2 = 0.4888, p < .0001), partial Mayo score ( R 2 = 0.3652, p = .0002), and negatively with albumin levels ( R 2 = 0.3747, p = .0002). In CD, the BUSS score showed a positive correlation with CDAI ( R 2 = 0.2694, p = .0394). Plasma IgA levels were significantly associated with bowel wall thickness (BWT) in both UC and CD ( R 2 = 0.1759, p = .0041). Furthermore, plasma IgA was strongly correlated with the BUSS score ( R 2 = 0.6585, p = .0001) in CD patients. Notably, when BWT exceeded 3.5 mm, plasma IgA levels increased significantly ( p = .0025). These results demonstrate that IUS is an effective tool for monitoring disease activity and evaluating therapeutic responses in IBD patients. Besides, plasma IgA reflects the bowel wall inflammation, particularly in conjunction with IUS.
Read moreInfluence of interhospital transfer on endovascular thrombectomy outcome in acute ischemic stroke patients: an analysis of the TREAT-AIS registry
BackgroundThe outcome of patients undergoing endovascular thrombectomy (EVT) for large vessel occlusion in a comprehensive stroke center (CSC) is affected by the onset-to-treatment time. Whether the pathway to CSC arrival (direct vs. interhospital transfer) is thus associated with EVT outcomes among such patients is unknown.MethodsUsing the Taiwan Registry of Endovascular Thrombectomy for AIS registry, patients ≥20 years of age and receiving EVT for AIS within 24 h of onset between January 2019 and December 2022 were included. Patients were categorized according to CSC arrival pathway into direct arrival and transfer groups. The primary outcome was 3-month functional independence, defined as a modified Rankin Scale (mRS) of 0–2.ResultsOf the1830 patients included, 79% arrived at a CSC directly and 21% via transfer. More patients in the direct arrival than the transfer group achieved a 3-month mRS of 0–2. A significant interaction was found between onset-to-puncture (OTP) time and arrival pathway for achieving 3-month mRS 0–2 (pinteraction = 0.017). Arrival by transfer was associated with reduced odds of achieving mRS 0–2 when OTP time was < 6 h (aOR, 0.55), but with increased odds when OTP was ≥6 h (aOR, 1.95).ConclusionsDirect arrival was associated with improved outcomes if OTP < 6 h. Patients who arrive via transfer may still benefit substantially from EVT during the later treatment window. Further study is warranted to examine the predictors of favorable post-EVT outcomes within a 24-h window to facilitate timely treatment.
Read moreBio-Printed PCL Tracheal Graft in a Large Animal Model: Reproducible Short-Segment Regeneration and Preliminary Upgraded Long-Segment Reconstruction.
Three-dimensional (3D) bioprinting provides new options for airway reconstruction by enabling the fabrication of customizable, biodegradable scaffolds designed to support in situ tissue regeneration. Building on our established large-animal platform, in which two cm bioprinted tracheal grafts combined with refined surgical techniques and adjunctive laser intervention have achieved long-term survival exceeding three months, the present study aims to explore long-segment (≥four cm) tracheal transplantation. We evaluated the fabrication feasibility and regeneration patterns of extrusion-based 3D bioprinted polycaprolactone (PCL) tracheal grafts in a porcine model. The grafts were implanted via end-to-end anastomosis with adjunctive mechanical stabilization and followed by serial bronchoscopic surveillance, gross examination, and histological analysis. The two cm PCL tracheal grafts achieved reproducible survival exceeding three months when combined with refined surgical techniques, structured postoperative airway management, and optimized wound coverage. Histological analysis revealed multi-lineage tissue formation-including cartilage, muscle, glands, and epithelium-was observed. Cartilage regeneration followed a staged maturation process, compared to epithelial regeneration, although continuous by 12 weeks, remained developmentally immature. A single long-segment transplantation was explored in a single preliminary case, providing an initial technical observation of feasibility; however, definitive conclusions regarding long-term survival or regeneration cannot be drawn. These findings further characterize regenerative responses in a large-animal model and highlight critical translational barriers-fabrication constraints, airway biomechanics, and delayed epithelial maturation-that require systematic investigation before long-segment tracheal reconstruction can advance toward clinical application.
Read morePhoton-Counting CT Enables Preoperative Diagnosis of Perilymphatic Fistula.
515 AIxTHY Digital Cytopathology Platform Improves Diagnostic Accuracy and Reduces Nondiagnostic Interpretations in Thyroid Fine-Needle Aspiration Cytology
Evaluation of healthcare utilization and burden of chronic cough in Taiwan using patient interviews and medical chart review.
PT7:08 Longitudinal association between LLDAS and remission attainment and improvement in health-related quality of life in patients with systemic lupus erythematosus:a multinational prospective cohort study
Adjunctive Molecular Hydrogen Therapy Modulates T Cell Markers and Reduces Anti-Ro Antibody in Refractory Oral Ulcers of Behçet's Disease and Sjögren's Syndrome: A Case Report.
Behçet's Disease is a systemic vasculitis affecting both venous and arterial vessels, characterized by painful oral and genital ulcers. Primary Sjögren's Syndrome is an autoimmune disorder mainly targeting exocrine glands, especially the salivary and lacrimal glands, with possible systemic involvement. Molecular hydrogen has emerged as a potential adjuvant therapy due to its antioxidant and immunomodulatory effects. This report evaluates the efficacy of molecular hydrogen as adjunctive therapy for refractory oral ulcers in a patient with Behçet's disease and primary Sjögren's syndrome. A 67-year-old Taiwanese woman with Behçet's disease and primary Sjögren's syndrome had recurrent painful oral ulcers and mucosal atrophy since 2018, meeting the International Study Group (ISG) criteria. In 2020, she fulfilled the 2016 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) criteria for Sjögren's syndrome based on Schirmer's test (0/0 mm), positive anti-Ro antibody (29 U/ml), and labial salivary gland biopsy findings. Despite long-term, hydroxychloroquine, colchicine, and monthly high-dose intravenous vitamin C and glutathione, her oral ulcers recurred with petechiae and bleeding. In January 2024, oral molecular hydrogen capsules were added as adjunctive therapy. Over several months, she showed marked clinical improvement, with less mucosal bleeding and decreased anti-Ro antibody levels. Immunophenotyping revealed increased naïve T helper Fas cell surface death receptor-positive (Fas+) and cytotoxic T cell programmed death-1-positive (PD-1+) subsets, suggesting immunomodulatory effects. The therapy was well tolerated, with no major adverse events. By August 2025, oral ulcers had markedly improved, and systemic disease remained stable on maintenance with hydroxychloroquine, azathioprine, corticosteroid infusions, and hydrogen therapy. Molecular hydrogen may aid refractory oral ulcers in Behçet's and primary Sjögren's syndromes, with clinical and immunologic benefits.
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