Purpose: Hemophilia A is a genetic disorder characterized by a lack of factor VIII (FVIII). Emicizumab, a recombinant humanized bispecific monoclonal antibody, mimics the function of FVIII. In this article, we present data on an initial real-world evaluation of emicizumab use in Korean children with severe hemophilia A without inhibitors. Methods: This study was conducted from June 2020 to March 2024 at 4 centers in Korea. The participants were pediatric patients with severe hemophilia A without inhibitors who had received emicizumab treatment for over 6 months. The mean and median annualized bleeding rates (ABRs) and mean and median annual joint bleeding rates (AJBRs) were compared. Results: Each of the 21 patients in the study received an emicizumab loading regimen of 3 mg/kg weekly for 4 weeks, followed by a modified maintenance regimen of which 2 patients (9.5%) received a 1.5 mg/kg weekly dose, 3 patients (14.3%) received a 6 mg/kg dose every 4 weeks, and the remaining 16 patients (76.2%) received a 3 mg/kg dose every 2 weeks. Before emicizumab prophylaxis initiation, the mean and median ABRs for all patients were 7.04 (SD ± 5.83) and 6.52 (range 0–21.74), respectively. After receiving emicizumab treatment, the mean and mediam ABRs decreased to 0.41 and zero, respectively. Additionally, 85.7% of the patients achieved no bleeding events within 6 months of starting the treatment. Conclusion These first real-world data in Korea indicate that emicizumab is effective and safe for pediatric patients with severe hemophilia A without inhibitors.

Purpose: Hemophilia A is a genetic disorder characterized by a lack of factor VIII (FVIII). Emicizumab, a recombinant humanized bispecific monoclonal antibody, mimics the function of FVIII. In this article, we present data on an initial real-world evaluation of emicizumab use in Korean children with severe hemophilia A without inhibitors. Methods: This study was conducted from June 2020 to March 2024 at 4 centers in Korea. The participants were pediatric patients with severe hemophilia A without inhibitors who had received emicizumab treatment for over 6 months. The mean and median annualized bleeding rates (ABRs) and mean and median annual joint bleeding rates (AJBRs) were compared. Results: Each of the 21 patients in the study received an emicizumab loading regimen of 3 mg/kg weekly for 4 weeks, followed by a modified maintenance regimen of which 2 patients (9.5%) received a 1.5 mg/kg weekly dose, 3 patients (14.3%) received a 6 mg/kg dose every 4 weeks, and the remaining 16 patients (76.2%) received a 3 mg/kg dose every 2 weeks. Before emicizumab prophylaxis initiation, the mean and median ABRs for all patients were 7.04 (SD ± 5.83) and 6.52 (range 0–21.74), respectively. After receiving emicizumab treatment, the mean and mediam ABRs decreased to 0.41 and zero, respectively. Additionally, 85.7% of the patients achieved no bleeding events within 6 months of starting the treatment. Conclusion These first real-world data in Korea indicate that emicizumab is effective and safe for pediatric patients with severe hemophilia A without inhibitors.

Type 1 diabetes (T1D) is a chronic autoimmune disease characterized by the destruction of insulin-producing beta-cells in the pancreas.Genetic variations within the major histocompatibility complex (MHC) significantly influence the development of T1D, with diseaseprogression often indicated by the presence of autoantibodies. Until recently, insulin therapy was the sole treatment for T1D. However, in 2022, the Food and Drug Administration approved teplizumab, an anti-CD3 monoclonal antibody, as a novel immunomodulatory therapy to delay the onset of T1D. Various immunologic agents, including anti-CD antibodies and anti-cytokine autoantibodies, have been investigated across various stages of T1D in clinical trials. This article examines the current status of drug development for theprevention and treatment of T1D and summarizes key studies that aimed at delaying the onset of T1D using these agents. While effortsto halt or prevent the disease prior to clinical diagnosis have yielded limited success, post-diagnosis interventions have shown promising potential in slowing disease progression by preserving beta-cell function. Further investigation into long-term clinical outcomes related to the delay of T1D onset is necessary, and ongoing studies require extended follow-up to assess their full potential.

Type 1 diabetes (T1D) is a chronic autoimmune disease characterized by the destruction of insulin-producing beta-cells in the pancreas.Genetic variations within the major histocompatibility complex (MHC) significantly influence the development of T1D, with diseaseprogression often indicated by the presence of autoantibodies. Until recently, insulin therapy was the sole treatment for T1D. However, in 2022, the Food and Drug Administration approved teplizumab, an anti-CD3 monoclonal antibody, as a novel immunomodulatory therapy to delay the onset of T1D. Various immunologic agents, including anti-CD antibodies and anti-cytokine autoantibodies, have been investigated across various stages of T1D in clinical trials. This article examines the current status of drug development for theprevention and treatment of T1D and summarizes key studies that aimed at delaying the onset of T1D using these agents. While effortsto halt or prevent the disease prior to clinical diagnosis have yielded limited success, post-diagnosis interventions have shown promising potential in slowing disease progression by preserving beta-cell function. Further investigation into long-term clinical outcomes related to the delay of T1D onset is necessary, and ongoing studies require extended follow-up to assess their full potential.

Type 1 diabetes (T1D) is a chronic autoimmune disease characterized by the destruction of insulin-producing beta-cells in the pancreas.Genetic variations within the major histocompatibility complex (MHC) significantly influence the development of T1D, with diseaseprogression often indicated by the presence of autoantibodies. Until recently, insulin therapy was the sole treatment for T1D. However, in 2022, the Food and Drug Administration approved teplizumab, an anti-CD3 monoclonal antibody, as a novel immunomodulatory therapy to delay the onset of T1D. Various immunologic agents, including anti-CD antibodies and anti-cytokine autoantibodies, have been investigated across various stages of T1D in clinical trials. This article examines the current status of drug development for theprevention and treatment of T1D and summarizes key studies that aimed at delaying the onset of T1D using these agents. While effortsto halt or prevent the disease prior to clinical diagnosis have yielded limited success, post-diagnosis interventions have shown promising potential in slowing disease progression by preserving beta-cell function. Further investigation into long-term clinical outcomes related to the delay of T1D onset is necessary, and ongoing studies require extended follow-up to assess their full potential.

Purpose: Hemophilia A is a genetic disorder characterized by a lack of factor VIII (FVIII). Emicizumab, a recombinant humanized bispecific monoclonal antibody, mimics the function of FVIII. In this article, we present data on an initial real-world evaluation of emicizumab use in Korean children with severe hemophilia A without inhibitors. Methods: This study was conducted from June 2020 to March 2024 at 4 centers in Korea. The participants were pediatric patients with severe hemophilia A without inhibitors who had received emicizumab treatment for over 6 months. The mean and median annualized bleeding rates (ABRs) and mean and median annual joint bleeding rates (AJBRs) were compared. Results: Each of the 21 patients in the study received an emicizumab loading regimen of 3 mg/kg weekly for 4 weeks, followed by a modified maintenance regimen of which 2 patients (9.5%) received a 1.5 mg/kg weekly dose, 3 patients (14.3%) received a 6 mg/kg dose every 4 weeks, and the remaining 16 patients (76.2%) received a 3 mg/kg dose every 2 weeks. Before emicizumab prophylaxis initiation, the mean and median ABRs for all patients were 7.04 (SD ± 5.83) and 6.52 (range 0–21.74), respectively. After receiving emicizumab treatment, the mean and mediam ABRs decreased to 0.41 and zero, respectively. Additionally, 85.7% of the patients achieved no bleeding events within 6 months of starting the treatment. Conclusion These first real-world data in Korea indicate that emicizumab is effective and safe for pediatric patients with severe hemophilia A without inhibitors.

Type 1 diabetes (T1D) is a chronic autoimmune disease characterized by the destruction of insulin-producing beta-cells in the pancreas.Genetic variations within the major histocompatibility complex (MHC) significantly influence the development of T1D, with diseaseprogression often indicated by the presence of autoantibodies. Until recently, insulin therapy was the sole treatment for T1D. However, in 2022, the Food and Drug Administration approved teplizumab, an anti-CD3 monoclonal antibody, as a novel immunomodulatory therapy to delay the onset of T1D. Various immunologic agents, including anti-CD antibodies and anti-cytokine autoantibodies, have been investigated across various stages of T1D in clinical trials. This article examines the current status of drug development for theprevention and treatment of T1D and summarizes key studies that aimed at delaying the onset of T1D using these agents. While effortsto halt or prevent the disease prior to clinical diagnosis have yielded limited success, post-diagnosis interventions have shown promising potential in slowing disease progression by preserving beta-cell function. Further investigation into long-term clinical outcomes related to the delay of T1D onset is necessary, and ongoing studies require extended follow-up to assess their full potential.

OBJECTIVE: Acupotomy is a modern acupuncture method that includes modern surgical methods. Since acupotomy is relatively more invasive than filiform acupuncture treatment, it is important to establish the safety profile of this practice. To justify further large-scale prospective observational studies, this preliminary study was performed to assess the feasibility of the approach and investigate the safety profile and factors potentially associated with adverse events (AEs). METHODS: This was a prospective pilot study that assessed the feasibility of a large-scale forthcoming safety study on acupotomy treatment in a real-world setting. The feasibility (call response rate, drop-out rate, response rate for each variable and recruitment per month) and safety profile (incidence, type, severity and causality of AEs, and factors potentially associated with AEs) were measured. RESULTS: A total of 28 participants joined the study from January to May 2018. A follow-up assessment was achieved in 258 (1185 treatment points) out of 261 sessions (1214 treatment points). The response rate via telephone on the day after treatment was 87.3%. There were 8 systemic AEs in all the sessions (8/258; 3.11%) and 27 local AEs on the total points treated (27/1185; 2.28%). Severe AEs did not occur. Total AE and local AE occurrence were associated with blade width and the number of needle stimulations per treatment point. CONCLUSION: The findings suggest that it could be feasible to analyze the safety of acupotomy in a real-world setting. Moreover, the primary data on some relevant AEs could be determined. We are planning large-scale prospective studies based on these findings. TRIAL REGISTRATION Clinical Research Information Service (CRIS) KCT0002849 (https://cris.nih.go.kr/cris/search/detailSearch.do/11487).
Humans ; Feasibility Studies ; Prospective Studies ; Pilot Projects ; Acupuncture Therapy/methods* ; Research Design ; Treatment Outcome

BACKGROUND: The repair of large bone defects remains a significant challenge in clinical practice and requires bone grafts or substitute materials. In this study, we developed a unique hybrid bone scaffold comprising a three dimensional (3D)-printed metal plate for weight bearing and a biodegradable polymer tube serving as bone conduit. We assessed the long-term effect of the hybrid bone scaffold in repairing radial bone defects in a beagle model. METHODS: Bone defects were created surgically on the radial bone of three beagle dogs and individually-tailored scaffolds were used for reconstruction with or without injection of autologous bone and decellularized extracellular matrix (dECM). The repaired tissue was evaluated by X-ray, micro-computed tomography, and histological observation 6 months after surgery. The functional integrity of hybrid bone scaffold-mediated reconstructions was assessed by gait analysis. RESULTS: In vivo analysis showed that the hybrid bone scaffolds maintained the physical space and bone conductivity around the defect. New bone was formed adjacent to the scaffolds. Addition of autologous bone and dECM in the polymer tube improved healing by enhancing bone induction and osteoconduction. Furthermore, the beagles’ gait appeared normal by 4 months. CONCLUSION The future of bone healing and regeneration is closely related to advances in tissue engineering. Bone production using autologous bone and dECM loaded on 3D-printed hybrid bone scaffolds can successfully induce osteogenesis and provide mechanical force for functional bone regeneration, even in large bone defects.