Evaluation Of Efficacy Research Articles

Evaluation of efficacy of mentzer index for screening of beta - Thalassemia trait in antenatal women

People with thalassemia trait should be distinguished from those who have anemia due to iron deficiency since they typically have mild microcytic hypochromic anemia and are mostly asymptomatic. : To evaluate efficacy of Mentzer index for screening Beta- Thalassemia trait in pregnant women.From January 2023 to October 2023, the observational study was carried out in the Department of Gynecology & Obstetrics at Sri Guru Ram Das Charitable Hospital Amritsar. The study comprised 130 pregnant women with anemia. To assess microcytic hypochromic anemia, tests like complete blood count (CBC), peripheral blood smears, iron studies, and hemoglobin electrophoresis were performed. Mentzer Index (MI) was computed, and its specificity and sensitivity assessed. Of the 130 patients, 112 had Mentzer Index greater than 13 and 18 had Mentzer Index less than 13 but only 12 out of these18 had beta thal-trait according to HPLC. There were total 15 females having beta thal-trait as per HPLC, however 3 of the 15 had Mentzer >13. Conserding Beta thal-trait, the Mentzer Index's sensitivity and specificity were 80% and 95.65% in this study, and 95.33% and 86.96% for IDA. Mentzer Index (MI) is a helpful tool to distinguish iron deficiency from thalassemia trait as its specificity and sensitivity is high as electrophoresis in diagnosis. In situations with limited resources, this proves to be advantageous in terms of cost-effectiveness.

Genetic variation perspective reveals potential drug targets for subtypes of endometrial cancer.

The study aims to identify potential drug targets for endometrial cancer (EC) subtypes through a Mendelian randomization (MR) approach, assessing their clinical relevance. We utilized genetic instruments for 4,907 plasma proteins from the deCODE Genetics study dataset, and data with EC (n = 12,906) from a genome-wide study (GWAS) meta-analysis in European populations for MR analyses. Complementary analyses included protein-protein interactions (PPI) network analysis, therapeutic efficacy evaluation, differential gene expression assessment, and prognosis evaluation. The expression levels of key drug targets were quantitatively measured at both the transcriptional and translational stages utilizing reverse transcription quantitative PCR (RT-qPCR) and immunohistochemistry (IHC). Additionally, we analyzed various clinicopathological features. Five drug targets for EC (CBR3, GSTO1, HHIP, IGF2R, and MMP10), seven for endometrioid subtypes (ACAP2, CBR3, GSTO1, HHIP, IGF2R, MMP10, and TLR2), and seven for non-endometrioid subtypes (CST3, DNAJB14, FSTL5, GMPR2, IFI16, MAPK9, and NEO1) were identified. Among these, IGF2R (OR = 1.165; 95% CI 1.067-1.272; p = 1.046 × 10- 2) and CST3 (OR = 0.523; 95% CI 0.339-0.804; p = 7.010 × 10- 3) were highlighted as key drug targets with causal evidence both at transcriptional and translational levels. This study preliminarily confirms that IGF2R and CST3 may serve as novel targets for the treatment of EC, providing a foundational reference for innovative clinical approaches to this disease.

Pharmacokinetics, Efficacy, and Safety of Upadacitinib in Pediatric Patients with Polyarticular-Course Juvenile Idiopathic Arthritis: An Interim Analysis of an Open-label, Phase 1 Trial.

This work aimed to evaluate the pharmacokinetics, efficacy, and safety of upadacitinib, an oral selective JAK inhibitor, in pediatric patients with polyarticular-course juvenile idiopathic arthritis (pcJIA). In an open-label, phase 1 study (SELECT-YOUTH), enrolled patients, aged 2 to <18 years with pcJIA, received bodyweight-based upadacitinib doses using a twice-daily (BID) oral solution or once-daily (QD) extended-release tablet based on their body weight and ability to swallow tablets. The study included a 7-day pharmacokinetic assessment, followed by a long-term efficacy and safety evaluation for up to 156 weeks, including an additional long-term safety cohort. This interim analysis included available pharmacokinetic and safety data and efficacy data collected through Week 48. A total of 57 patients received upadacitinib. The median time to maximum upadacitinib concentration was approximately 3 hours and 1 hour for the tablet and oral solution regimens, respectively; the harmonic mean functional half-life was approximately 5 hours and 2 hours, respectively. Juvenile idiopathic arthritis (JIA) American College of Rheumatology (ACR)30/50/70/90/100 responses at Week 12 were 91.8/89.8/69.4/49.0/32.7%, respectively. Efficacy was generally maintained through Week 48, and improvement in additional efficacy endpoints was also observed. At a median exposure duration of 412 days, 52 of 57 patients reported adverse events, of these 6 experienced serious adverse events. Adverse events were predominately mild to moderate in severity and consistent with the known safety profile of upadacitinib. This interim analysis demonstrates that the bodyweight-based dosing regimen of upadacitinib was well tolerated and efficacious in pediatric patients with pcJIA.

HPB SO19 - Comparison of perioperative outcomes of open (CUSA) versus laparoscopic (LOTUS) major hepatectomy – revisited. First evaluation of efficacy and safety of AEONTM stapler

Abstract Background In 2019, preliminary data matching 40 cases demonstrated improved outcomes of laparoscopic major liver resections using the BOWA-LOTUSTM liver blade (Devon, UK). An updated and expanded comparison between open and laparoscopic major hepatectomies is presented as well as evaluating of an endovascular stapler AEON™ (Lexington Medical, Massachusetts, USA). Method January 2020 to June 2023 patients were identified from a prospectively maintained database. Propensity score matching analysis used R statistical tool in SPSS (IBM, New York, USA) to isolate matched open and laparoscopic cases. LOTUSTM ultrasonic energy device was used for parenchymal transection in laparoscopic cases and CUSA (Valleylab, Colorado, USA) in open procedures. AEONTM endovascular stapler was used from early 2021. Results 86 patients constitute the study population after applying propensity score matching analysis in a 1:1 ratio. The median (range) age in the matched sample was 63 (27-83) yrs. Hospital stay (mean 7.8 v 14.7 days; p=0.025) and blood transfusion requirement (0 v 4 units; p=0.035) were all significantly improved when comparing laparoscopic to open. Comparison of the AEONTM stapler with Endo-GIA (Medtronic, MN, USA) showed no significant difference in any outcome measure, although AEONTM demonstrated (non-significantly) a reduced trend in operating time comparing all cases (56 v 69 mins; p=0.300) and laparoscopic (56 v 71 min; p=0.295). Conclusion The LOTUS liver blade continues to be associated with acceptable outcomes in laparoscopic liver resections. The AEONTM endovascular stapler was equivalent to other staplers with a trend towards a shorter operating time in liver transectional surgery.

BN SO50 - The Resurgence of Veress Needle Insufflation: An Evaluation of Efficacy and Safety of One Surgeon’s Practice

Abstract Background Veress needle insufflation has been established as a safe and effective method of establishing pneumoperitoneum for laparoscopic surgery, with comparable risk profile to open methods. However, the blind nature of the procedure with potential complications such as iatrogenic bowel and blood vessel injury has seen this technique fall out of fashion with general surgeons. This study reviews the safety and efficacy of a single surgeon’s practice using Veress needle insufflation for Laparoscopic surgical procedures. Method A retrospective analysis was conducted on minimally invasive procedures performed between January 2019 and June 2024. In our practice, a standard or bariatric Veress needle is placed in a small incision at Palmer’s point. A manometer test is performed with CO2 insufflation at a low flow rate and raising the abdominal wall. If a low pressure is established, then the flow is increased to achieve adequate pneumoperitoneum before introduction of a midline 5mm camera port. Primary outcomes of this study include rate of conversion to open techniques (either Hasson’s or open cut down) and any complications. Results Of the 702 cases reviewed, five patients (0.71%) required conversion to open insufflation techniques. Two (0.28%) had bleeding from the omentum, and one patient (0.14%) experienced inadvertent bowel insufflation which was managed conservatively with no long-term morbidity. Notably there were no port site hernias. Conclusion This study supports the continued use of the Veress needle as a safe and effective method of insufflation in laparoscopic surgery. It offers minimal incision size, easier access in high BMI patients, and an alternative when adhesions and scarring limit access. Furthermore, adopting this technique can be particularly useful for appropriate port placement in robotic surgery, making it a valuable tool for modern surgical practices.

Intra-articular Injection of a Cross-Linked Hyaluronic Acid Combined with Triamcinolone Hexacetonide Improves Pain at 6 Months in Patients with Mild to Moderate Hip Osteoarthritis: A Prospective Observational Study.

Joint-preserving interventions, such as intra-articular viscosupplementation injections, are evolving, requiring efficacy and safety evaluation through an evidence-based approach. The objective of this study was to assess the use of a hyaluronic acid and corticosteroid-based injection (Cingal™; Anika Therapeutics, Bedford, MA, USA) in improving pain and functional outcomes for patients aged 40 to 65 with mild to moderate hip osteoarthritis (OA) six months post-injection. This prospective observational study included 100 patients receiving a single ultrasound-guided intra-articular injection of Cingal™. Eligible patients seen in participating orthopaedic, physiatry and sports medicine clinics, were evaluated at baseline, one-month, and six-month follow-up. The primary outcome was patient-reported hip pain (Visual Analogue Scale, VAS) at six months post-injection. Secondary outcomes included hip function (Hip Disability and Osteoarthritis Outcome Score, HOOS), quality of life (Short-Form 12, SF-12), pain medication use, range of motion (ROM), physical activity (activity tracker), and adverse events. 96 patients received the injection; 91 had complete data for primary outcome analysis. Statistically significant improvements were observed in VAS (p<0.001), HOOS (p<0.001), and SF-12 scores (Physical Component Summary, p=0.005; Mental Component Summary, p=0.022) from baseline to six months post-injection. Pain medication use decreased from 50.0% to 34.0% (p=0.035). No statistically significant change was observed in ROM or activity level. Adverse events were reported in 9.5% of patients: five (5.3%) experienced hip pain for less than seven days, one for greater than seven days but less than one month, and three (3.2%) underwent hip arthroplasty. Patients receiving an ultrasound-guided Cingal™ injection for hip OA reported statistically significantly reduced hip pain, improved function and quality of life, and reduced pain medication use at six months. The most common adverse event was transient hip pain. Prospective observational study, Level III.

Preclinical safety and efficacy evaluation of the intrathecal transplantation of GMP-grade human umbilical cord mesenchymal stem cells for ischemic stroke

Intrathecal administration of human umbilical cord mesenchymal stem cells may be a promising approach for the treatment of stroke, but its safety, effectiveness, and mechanism remain to be elucidated. In this study, good manufacturing practice–grade human umbilical cord mesenchymal stem cells (5 × 105 and 1 × 106 cells) and saline were administered by cerebellomedullary cistern injection 72 hours after stroke induced by middle cerebral artery occlusion in rats. The results showed (1) no significant difference in mortality or general conditions among the three groups. There was no abnormal differentiation or tumor formation in various organs of rats in any group. (2) Compared with saline-treated animals, those treated with human umbilical cord mesenchymal stem cells showed significant functional recovery and reduced infarct volume, with no significant differences between different human umbilical cord mesenchymal stem cell doses. (3) Human umbilical cord mesenchymal stem cells were found in the ischemic brain after 14 and 28 days of follow-up, and the number of positive cells significantly decreased over time. (4) Neuronal nuclei expression in the human umbilical cord mesenchymal stem cell group was greater than that in the saline group, while glial fibrillary acidic protein and ionized calcium binding adaptor molecule 1 expression levels decreased. (5) Human umbilical cord mesenchymal stem cell treatment increased the number of CD31+ microvessels and doublecortin-positive cells after ischemic stroke. Human umbilical cord mesenchymal stem cells also upregulated the expression of CD31+/Ki67+. (6) At 14 days after intrathecal administration, brain-derived neurotrophic factor expression in the peri-infarct area and the concentrations of brain-derived neurotrophic factor in the cerebrospinal fluid in both human umbilical cord mesenchymal stem cell groups were significantly greater than those in the saline group and persisted until the 28th day. Taken together, these results indicate that the intrathecal administration of human umbilical cord mesenchymal stem cells via cerebellomedullary cistern injection is safe and effective for the treatment of ischemic stroke in rats. The mechanisms may include alleviating the local inflammatory response in the peri-infarct region, promoting neurogenesis and angiogenesis, and enhancing the production of neurotrophic factors.

Abstract 4138704: Safety and Efficacy of Generic Atorvastatin in Korean Dyslipidemia Patients: A Non-Interventional Observational Study

Background: Statins are widely used for controlling dyslipidemia. However, most evidences for dyslipidemia prevention and treatment with statins are based on studies involving Caucasian populations, resulting in limited data on Korean patients. This study was conducted to evaluate the efficacy lipid profile and safety of generic atorvastatin in a real-world clinical setting among Korean dyslipidemia patients. Methods: This study was conducted as a non-interventional, multi-center observational study. It was carried out across 264 institutions and clinics from October 2017 to July 2021. Total 16,992 participants were enrolled. The safety and efficacy evaluation cohorts consisted of 16,803 and 11,035 subjects, respectively. The liver function evaluation cohort included 13,889 subjects. The main eligibility criteria were dyslipidemia patients prescribed generic atorvastatin according to approved indications. Adverse event rates were collected for safety evaluation, and lipid profiles, including LDL-C, were collected for efficacy evaluation. Results: In the safety endpoint evaluation, 1,612 adverse events were reported in 1,027 subjects (6.11%). Common adverse events such as dizziness, headache, and constipation, which were frequently observed in this study, were within the approved labels. Muscle pain occurred in 0.14% of subjects, and myopathy, myositis, rhabdomyolysis, and CK abnormalities or increases were reported in 0.01% of subjects, respectively. Most of the adverse events were confirmed to be mild or resolved, and the study was completed without changing the dose. All abnormal liver function test results were reported in less than 1% of subjects (AST increase: 0.14%, ALT increase: 0.1%, ALP increase: 0.01%, γ-GT increase: 0.01%). The mean LDL-C decreased from 117.31 mg/dL to 89.97 mg/dL, showing a statistically significant improvement in LDL-C levels (p &lt; 0.0001). Conclusions: Generic atorvastatin demonstrated tolerable safety as well as significant efficacy on lipid profile. Generic atorvastatin might be safe and effective for Korean patients with dyslipidemia.

CTNI-76. A PHASE 1/2 DOSE ESCALATION AND EXPANSION STUDY OF SONODYNAMIC THERAPY WITH SONALA-001 IN COMBINATION WITH NON-ABLATIVE MR-GUIDED FOCUSED ULTRASOUND IN SUBJECTS WITH PROGRESSIVE OR RECURRENT GLIOBLASTOMA

Abstract Recurrent glioblastoma (rGBM) is a lethal brain tumor with no effective standard of care option and an extremely poor prognosis (median survival 6 to 8 months after first recurrence). Sonodynamic therapy (SDT) is a non-invasive combination therapy that utilizes a drug, aminolevulinic acid HCL (SONALA-001, ALA), and a device, the Exablate 4000 Type 2.0 to deliver MR-guided focused ultrasound (MRgFUS). Preclinical studies have shown that SDT activates protoporphyrin IX (PpIX) to generate reactive oxygen species that lead to tumor cell death and improved survival in animal glioma models. A first-in-human Phase 0 trial (NCT04559685) indicated that ALA SDT was well-tolerated and not associated with off-target cellular or radiographic effects and provided direct evidence of reactive oxygen species formation and targeted tumor cell death in recurrent high-grade glioma (rHGG). SDT-202 is a Phase 1/2 multicenter, open-label, dose escalation and expansion study (NCT05370508) of iv SONALA-001 (ALA) in combination with MRgFUS for SDT in patients with progressive or recurrent GBM. Dose escalation will include an initial drug and energy dose escalation of ALA SDT utilizing an accelerated titration design with single subject cohorts for dose level cohorts 1 and 2, followed by cohort 3 with 3 + 3 design for additional dose escalation. SONALA-001 is administered 6 to 9 hours prior to MRgFUS treatment. Once the RP2D is determined, Phase 2 dose expansion will enroll approximately 36 additional subjects to assess safety and efficacy of repeated cycles of ALA SDT at the optimized RP2D. Endpoints of this study are safety evaluation, maximum tolerated dose (MTD), recommended Phase 2 dose (RP2D), and determination of pharmacokinetic (PK) parameters. The Phase 2 portion will further characterize safety of RP2D along with evaluation of efficacy with PFS6 (mRANO), ORR, CBR, DOR, DOCR, TTR, and OS. Preliminary PK and safety results will be presented.

CTNI-72. VAL-083 IN PATIENTS WITH RECURRENT GLIOBLASTOMA TREATED UNDER EXPANDED ACCESS PROGRAM

Abstract BACKGROUND Current standard-of-care for glioblastoma (GBM) includes surgery followed by chemoradiation with temozolomide (TMZ) followed by adjuvant TMZ. Almost all GBM patients experience disease progression despite upfront standard-of-care treatment, with a median overall survival of 3-9 months after first recurrence. There are limited treatment options at the time of disease progression, apart from participation in clinical trials. VAL-083 is a bi-functional DNA damaging agent that induces inter-strand DNA cross-links at N7-guanine in a manner independent of O6-methylguanine-DNA-methyltransferase (MGMT). METHODS Under an Expanded Access program (NCT03138629), we used VAL-083 to treat 30 patients with recurrent GBM who were not eligible to participate in clinical trials. Here we report safety and efficacy results. RESULTS Four patients (13.3%) with leptomeningeal disease were excluded from efficacy evaluation. All patients received chemoradiation with TMZ. Twelve (12/26; 46.2%) patients had ≥2 recurrences and 9/26 (34.6%) had prior lomustine. All tumors had ≥1 mutation, with 14/26 (53.8%) having ≥5 mutations; one patient had a hypermutator phenotype. The most common mutations were TERT (57.7%), PTEN (38.5%), TP53 (26.9%), and NF1 (26.9%). All patients started treatment with VAL-083 at 30 mg/m2/day administered on 3 consecutive days every 21 days. Eight (8/26; 30.8%) patients received bevacizumab concurrently with VAL-083. Eight (8/26; 30.8%) patients had a VAL-083 dose reduction. Patients received a median of 3.0 (5-95pth 1-10) VAL-083 treatment cycles. Median progression free survival (mPFS) and median overall survival (mOS) from last disease progression was 5.1 (95%CI: 2.7-7.2) and 9.8 (95%CI: 5.3-16.4) months, respectively. VAL-083 was well-tolerated and the most frequent adverse events were consistent with prior experience, i.e., thrombocytopenia and neutropenia. CONCLUSIONS Use of VAL-083 continues to show benefit in the treatment of GBM patients who have had multiple recurrences and have limited therapeutic options.

A novel HIV triple broadly neutralizing antibody (bNAb) combination-based passive immunization of infant rhesus macaques achieves durable protective plasma neutralization levels and mediates anti-viral effector functions.

To eliminate vertical HIV transmission and achieve therapy-free viral suppression among children living with HIV, novel strategies beyond antiretroviral therapy (ART) are necessary. Our group previously identified a triple broadly neutralizing antibody (bNAb) combination comprising of 3BNC117, PGDM1400 and PGT151 that mediates robust in vitro neutralization and non-neutralizing effector functions against a cross-clade panel of simian human immunodeficiency viruses (SHIVs). In this study, we evaluated the safety, pharmacokinetics, and antiviral potency of this bNAb combination in infant rhesus macaques (RMs). We demonstrate that subcutaneous infusion of the triple bNAb regimen was well tolerated in pediatric monkeys and resulted in durable systemic and mucosal distribution. Plasma obtained from passively-immunized RMs demonstrated potent HIV-neutralizing and Fc-mediated antiviral effector functions. Finally, using the predicted serum neutralization 80% inhibitory dilution titer (PT80) biomarker threshold of >200, which was recently identified as a surrogate endpoint for evaluation of the preventative efficacy of bNAbs against mucosal viral acquisition in human clinical trials, we demonstrated that our regimen has PT80>200 against a large panel of plasma and breast milk-derived HIV strains and cross-clade SHIV variants. This data will guide the development of combination bNAbs for eliminating vertical HIV transmission and for achieving ART-free viral suppression among children living with HIV.

Improving precision of vaccine efficacy evaluation using immune correlate data in time-to-event models.

Understanding potential differences in vaccine-induced protection between demographic subgroups is key for vaccine development. Vaccine efficacy evaluation across these subgroups in phase 2b or 3 clinical trials presents challenges due to lack of precision: such trials are typically designed to demonstrate overall efficacy rather than to differentiate its value between subgroups. This study proposes a method for estimating vaccine efficacy using immunogenicity (instead of vaccination status) as a predictor in time-to-event models. The method is applied to two datasets from immunogenicity sub-studies of vaccine phase 3 clinical trials for zoster and dengue vaccines. Results show that using immunogenicity-based estimation of efficacy in subgroups using time-to-event models is more precise than the standard estimation. Incorporating immune correlate data in time-to-event models improves precision in estimating efficacy (i.e., yields narrower confidence intervals), which can assist vaccine developers and public health authorities in making informed decisions.

Changes in lipid abundance are associated with disease progression and treatment response in chronic Trypanosoma cruzi infection

BackgroundChagas disease, caused by the parasite Trypanosoma cruzi, is a zoonosis that affects more than seven million people. Current limitations on the diagnosis of the disease hinder the prognosis of patients and the evaluation of treatment efficacy, slowing the development of new therapeutic options. The infection is known to disrupt several host metabolic pathways, providing an opportunity for the identification of biomarkers.MethodsThe metabolomic and lipidomic profiles of a cohort of symptomatic and asymptomatic patients with T. cruzi infection and a group of uninfected controls were analysed using liquid chromatography/mass spectrometry. Differences among all groups and changes before and after receiving anti-parasitic treatment across those with T. cruzi infection were explored.ResultsThree lipids were found to differentiate between symptomatic and asymptomatic participants: 10-hydroxydecanoic acid and phosphatidylethanolamines PE(18:0/20:4) and PE(18:1/20:4). Additionally, sphinganine, 4-hydroxysphinganine, hexadecasphinganine, and other sphingolipids showed post-treatment abundance similar to that in non-infected controls.ConclusionsThese molecules hold promise as potentially useful biomarkers for monitoring disease progression and treatment response in patients with chronic T. cruzi infection.Graphical

Efficacy and safety evaluation of a novel hemostatic gelatin matrix for intraoperative hemostasis: a prospective, randomized controlled trial

Borayflo Haemostatic matrix is a new absorbable hemostatic gelatin matrix which can be used for intraoperative assisted hemostasis. In a prospective, multicenter, non-inferiority, randomized controlled trial, a total of 354 subjects were recruited from the departments of hepatobiliary surgery, obstetrics and gynaecology and orthopaedics of 4 hospitals and randomly allocated to test group (Borayflo Haemostatic matrix) or control group (Surgiflo Haemostatic matrix) in a 1:1 ratio. In the modified intention-to-treat population, 163 (93.14%) of 175 subjects in the test group versus 167 (94.89%) of 176 subjects in the control group successfully achieved hemostasis within 5 min (P > 0.05). Non-inferiority for effective rates of hemostasis at 5 min to Surgiflo Haemostatic matrix was shown in the study (treatment difference: -1.74% [95%CI, -6.70–3.22%] for modified intention-to-treat population). In terms of efficacy and safety, the new hemostatic gelatin matrix (Borayflo Haemostatic matrix) is equivalent to Surgiflo Haemostatic matrix. There was no significant difference in the incidence of AEs or SAEs between the test and control groups(P > 0.05). In addition, Borayflo Haemostatic matrix is a domestically produced haemostatic gelatin product, an advantage that will reduce the cost of surgical haemostasis for Chinese patients.

PP 1/336 (1) Efficacy evaluation of herbicides used with mechanical weeders

<scp>PP</scp> 1/336 (1) Efficacy evaluation of herbicides used with mechanical weeders

Two types of vidian neurectomy show efficacy in treating allergic rhinitis and vasomotor rhinitis

This study aimed to evaluate the efficacies of endoscopic vidian neurectomy (VN) and highly selective vidian neurectomy (HSVN) for the treatment of allergic rhinitis (AR) and vasomotor rhinitis (VMR). All AR and VMR patients were divided into two groups, Group VN and Group HSVN. The efficacy evaluation methods were visual analog scale (VAS), rhinoconjunctivitis quality of life questionnaire (RQLQ), and medication score. The efficacy evaluations were used to assess patient rhinitis symptoms, quality of life and drug usage. The time points of follow-up period were preoperative, 3 months, 6 months, 1 year, 2 years, and 3 years post operation. By analyzing pre- and postoperative VAS, medication score and RQLQ score, the results showed that rhinitis symptoms and quality of life in both VN and HSVN groups were significantly improved at 3 years, and the rate of improvement decreased gradually with time. Post operation, there were no significant differences in VAS and medication scores between the VN and HSVN groups. With respect to RQLQ, postoperative 2 years and 3 years improvements in sleep disorders, non-nasal symptoms and eye symptoms were significantly greater in the HSVN group than in the VN group. Improvements in rhinitis symptoms and quality of life in AR and VMR were sustained by VN and HSVN until 3 years post operation, with greater quality of life improvements in the HSVN group.

Evaluation of the potential efficacy of the nitric oxide donor molsidomine for the treatment of schizophrenia.

Schizophrenia is a chronic devastating psychiatric disease characterized by a high recurrence rate. Pharmacological management of this disorder appears disappointing since it is associated with a lack of efficacy for negative symptoms and cognitive deficits, typical features of schizophrenia, and the presence of severe undesired side effects. Thus, novel molecules with high efficacy and low toxicity for the treatment of schizophrenia are urgently needed. The involvement of the gaseous molecule nitric oxide in the pathogenesis of schizophrenia is well documented since low concentrations of nitric oxide are associated with this psychiatric disease. Therefore, chemicals able to normalize nitric oxide levels, such as nitric oxide donors, might be useful for the management of this type of schizophrenia. Molsidomine is a nitric oxide donor and is under investigation as a novel antischizophrenia agent. The aim of this review is to critically evaluate the potential efficacy of this molecule for the treatment of schizophrenia.

Global research hotspots and trends of Buyang huanwu decoction: A visual analysis of the literature based on CiteSpace.

Buyang huanwu decoction (BYHWD) has shown significant clinical efficacy in the treatment of several diseases, particularly stroke. However, bibliometric research has not been comprehensive. BYHWD articles were collected from literature databases published from January 1, 1915, to March 31, 2024, including the China National Knowledge Infrastructure, Weipu, Wanfang, Pubmed, Scopus, and Web of Science Core Collection. Knowledge network graphs of annual publication volume, authors, institutions, countries, keywords, and references were constructed. Nine thousand two hundred thirty-eight Chinese literature and 559 English articles published between 1915 and 2024 showed an overall upward trend. The countries, institutions, journals, and authors with the highest output were China, Hunan University of Chinese Medicine, Guangzhou University of Chinese Medicine, Guangxian Cai, and Changqing Deng, respectively. Research teams outside China were located at Daejeon University, Gachon University, Aga Khan University, Yale University, etc. The results of keyword co-occurrence and burst analysis included clinical applications, animal experiments, action mechanisms, clinical efficacy, and safety evaluations based on systematic reviews and meta-analyses. Literature co-citation analysis revealed that BYHWD was highly correlated with neuroprotection and reduction of cerebral ischemia/reperfusion injury. Both Chinese and English literature have shown overall growth trend since 1984 and 1989, respectively. Clinical applications, pharmacological effects, mechanisms, active ingredients, evaluation of clinical efficacy and safety, modified BYHWD, methods, and biological techniques may be hotspots and focus of future research on BYHWD. Hotspot analytical methods and biological techniques include systematic reviews, meta-analyses, data mining, network pharmacology, and molecular docking. Future valuable research fields may include studies on neuroprotection, anti-inflammatory activity, ischemic stroke, bioactive compounds, and their underlying mechanisms.

Investigation of Novel Isatinylhydantoin Derivatives as Potential Anti-Kinetoplastid Agents.

Neglected tropical diseases are a group of infectious diseases with a high endemicity in developing countries of Africa, Asia, and the Americas. Treatment for these diseases depends solely on chemotherapy, which is associated with severe side effects, toxicity, and the development of parasitic resistance. This highlights a critical need to develop new and effective drugs to curb these diseases. As a result, a series of novel isatinylhydantoin derivatives were synthesized and evaluated for in vitro anti-kinetoplastid activity against seven human- or animal-infective Trypanosoma and two human-infective Leishmania species. The synthesized derivatives were tested for potential cytotoxicity against human, animal, and parasite host-related cell lines. The isatinylhydantoin hybrid 4 b bearing 5-chloroisatin and p-bromobenzyl moieties, showed strong trypanocidal activity against blood-stage T. congolense parasites; however, the promising in vitro trypanocidal potency of 4 b could not be translated to in vivo treatment efficacy in a preliminary animal study. Compounds 5, 2 b, and 5 b, were the most active against amastigotes of L. donovani, showing higher leishmanicidal activity than the reference drug, amphotericin B. These compounds were identified as early antileishmanicidal leads, and future investigations will focus on confirming their antileishmanial potential through in vivo efficacy evaluation as well as their exact mechanism of action.

Safety and efficacy evaluation of intracavernosal injection of platelets rich plasma in treatment of vasculogenic erectile dysfunction

ABSTRACT Objectives The study’s objective is to compare platelet-rich plasma (PRP) injections to placebo in terms of effectiveness and safety for patients with vasculogenic erectile dysfunction (ED). Methods This randomized placebo-controlled clinical trial was done on 50 male patients with organic vasculogenic ED and sexually active in a stable heterosexual relationship for a period of over three months. The patients were randomized into two main groups: Group (A) (n = 25) underwent PRP treatment. Group (B) (n = 25) control underwent placebo treatment. Results The two groups under study differed in a statistically significant way regarding the achievement of minimal clinically important differences (MCID) at 3 months and 24 months. Statistically significant variations existed between the mild subgroup and placebo regarding achieving MCID after 3 months and 24 months. The level of satisfaction was statistically significantly higher in the PRP group. A statistically significant variation was present between the mild subgroup and placebo at 3 months and 24 months in terms of changes from baseline in the erectile function domain of the International Index of Erectile Function (IIEF-EF) questionnaire score in each subgroup. Conclusions PRP is an effective alternative modality of treatment in cases of ED, and they offer an intermediate stage between pharmaceutical therapy and surgical interventions at least in mild and mild to moderate cases where unsatisfactory results or unpleasant side effects compel the patients to abandon all hope on medical treatment.