Clinical Efficacy In Treatment Research Articles

Clinical characteristics and treatment efficacy in patients with primary severe IGF-1 deficiency treated with recombinant IGF-1

Aim of the studyTo evaluate the clinical characteristics and treatment efficacy of patients with severe primary IGF-1 deficiency (PSIGFD) using a recombinant IGF-1 (rhIGF-1).Objectives of the studyTo examine the clinical characteristics of patients with PSIGFD before starting treatment with a rIGF-1. To assess the height changes in patients with PSIGFD, before and after treatment with a rhIGF-1. To analyze the clinical characteristics, side effect frequency, and treatment efficacy with a rhIGF-1 analog in patients with PSIGFD.MethodsA retrospective analysis was conducted on patients with PSIGFD treated with the rhIGF-1 (mecasermin). Data were collected from patients’ medical records, focusing on the impact of treatment on their growth and monitoring any side effects.ResultsThe study showed that treatment with rhIGF-1 positively affects growth rate, especially in the first years of treatment. However, the growth rate decreases over time. The change in height from the beginning to the end of the treatment was 0.76 ± 0.64 SD, with the first quartile at 0.29 SD and the third quartile at 1.14 SD. During the treatment period, patients’ average body mass increased by 0.37 ± 1.35 SD, with the first quartile at −0.33 SD and the third quartile at 0.92 SD. Side effects occurred in 50% of patients, with 40% of patients treated with rhIGF-1 experiencing hypoglycemia during treatment.ConclusionsTreatment with rhIGF-1 is effective in treating patients with PSIGFD, causing significant improvement in growth, but requires continuous monitoring and treatment adjustment.

Efficacy and safety of Shenmai injection in the treatment of viral myocarditis: a systematic review and meta-analysis

PurposeTo evaluate the efficacy and safety of Shenmai injection for the treatment of viral myocarditis (VMS) through systematic evaluation and meta-analysis.MethodsSeven databases were searched to identify randomized controlled trials that examined the use of Shenmai injection for the treatment of VMS. The databases were searched from inception to 20 January 2024. The quality of the included studies was evaluated via the Cochrane risk of bias tool (RoB 2) version 2. Data analysis was performed using Review Manager 5.4 and Stata 16.ResultsIn total, 18 randomized controlled trials were included. The trials were conducted in 2006–2024 and included 1,661 patients with VMS. The results reveal that Shenmai injection combined with conventional treatment was superior to conventional treatment alone in terms of the following outcomes: total effective rate [RR = 1.22, 95% CI (1.16, 1.28)], CKMB [SMD = −3.33, 95% CI (−4.85, −1.81)], electrocardiogram (ECG) efficacy [RR = 1.30, 95% CI (1.20, 1.40)], AST [SMD = -0.70, 95% CI (−1.28, −0.11)], LDH [SMD = −1.17, 95% CI (−1.37, −0.97], p < 0.00001], CK [SMD = −1.74, 95% CI (−2.34, −1.13)], TNF-α [SMD = −1.35, 95% CI (−1.85, −0.84)], and IL-6 [SMD = −1.40, 95% CI (−1.76, −1.05)]. There were no significant differences in the incidence of adverse reactions [RR = 1.56, 95% CI (0.73, 3.33), p = 0.25] or cTnI levels [SMD = −3.35, 95% CI (−6.81, 0.11)] between the groups.ConclusionShenmai injection with conventional treatment can reduce the degree of myocardial injury in patients with VMS, weaken the inflammatory response and improve the clinical efficacy of the conventional treatment. This approach was found to be safe. However, additional high-quality studies are necessary to confirm the reliability of this treatment method.Systematic Review Registration:https://www.crd.york.ac.uk/PROSPERO/logout.php, identifier PROSPERO (CRD42024518665).

Age Is a Key Factor Influencing the Choice of Treatment for Primary Patellar Dislocation: A Systematic Review and Meta-analysis.

Primary patellar dislocation has a certain recurrence rate after either conservative or surgical treatment, and the optimal treatment for patients with primary patellar dislocation of different ages remains unclear. This study aims to compare the clinical efficacy of surgical and conservative treatments for primary patellar dislocation across different age groups. According to the Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) checklist, we conducted a systematic search for randomized controlled trials. We searched the PubMed, EMBASE, Cochrane Library, and Web of Science databases for randomized controlled trials of primary patellar dislocation treated surgically or conservatively up to January 2023. Data searching, extraction, analysis, and quality assessment were carried out in accordance with the Cochrane Collaboration guidelines. A total of nine studies with 433 patients were included in our study. There was no statistically significant difference between the two treatment modalities in terms of Kujala score, pain score, patient satisfaction, reoperation, and Tegner score. The rate of re-dislocation after surgical treatment is lower than that after conservative treatment. Subgroup analysis based on mean age showed that when the age was >20 years, Kujala scores were higher after surgical treatment than after conservative treatment (p < 0.0001, 95% confidence interval [CI] = 10.41-21.30). When the age was ≤20 years, the difference in Kujala scores between the two treatment modalities was not statistically significant. When the age was >20 years, the recurrence rate of patellar dislocation was lower after surgical treatment than after conservative treatment (p = 0.009, 95% CI = 0.08-0.70). When the age was ≤20 years, the difference in the recurrence rate of patellar dislocation between the two treatment modalities was not statistically significant. When the age of patients with primary patellar dislocation is ≤20 years, both surgical and conservative treatments result in similar clinical outcomes. When the age is >20 years, better clinical outcomes can be achieved by opting for surgical treatment. Therefore, surgery may be a better option for patients with primary patellar dislocation whose age is >20 years.

Neurofeedback technique for treating male schizophrenia patients with impulsive behavior: a randomized controlled study.

Schizophrenia is one of the most severe mental disorders, frequently associated with aggression and violence, particularly in male patients. The underlying mechanisms of violent behavior in these patients remain unclear, limiting effective treatment options and highlighting the need for further research into interventions for impulsive behaviors. This study aims to evaluate the clinical efficacy of neurofeedback treatment in hospitalized male schizophrenia patients exhibiting impulsive behaviors. The study was designed as a single-center, randomized, single-blind, sham-controlled parallel trial. Eighty patients were randomly assigned to either a study group or a control group. The control group received risperidone and sham neurofeedback, while the study group received risperidone and active neurofeedback therapy. Both groups underwent training five times per week, with each session lasting 20 minutes, over a six-week period. Clinical symptoms were assessed at baseline, three weeks and six weeks using the Positive and Negative Syndrome Scale (PANSS), the Modified Overt Aggression Scale (MOAS), and the Rating Scale for Extrapyramidal Side Effects (RSESE). Statistical analyses were conducted to compare the therapeutic effects between the two groups at the study's conclusion. Initial comparisons showed no significant differences in baseline data, except for the number of prior hospitalizations (P<0.018). By the end of the study, the study group demonstrate significant improvements in MOAS and PANSS scores (including the Excited, Positive, Cognitive, and Depressive/Anxiety Components), with no significant changes in RSESE scores. Both time and group interactions were significant across most outcomes, underscoring the efficacy of neurofeedback in reducing the severity of impulsive behaviors and associated schizophrenia symptoms. chictr.org.cn, identifier ChiCTR2200063407.

Assessing Clinical Outcomes and Treatment Efficacy in a Patient with Crescentic Glomerulonephritis (CGN)- A Case Report

Crescentic glomerulonephritis (CGN) is a rapidly progressive renal condition marked by crescent formation in the glomeruli, often resulting in end-stage renal disease (ESRD) if not promptly addressed. This case report examines a 54-year-old female with a sudden onset of generalized edema and reduced urine output, which led to her hospitalization. Laboratory evaluations revealed acute renal failure with markedly elevated serum creatinine levels. Despite a background of Antineutrophil Cytoplasmic Antibodies (ANCA)-associated vasculitis, her management was complicated by inconsistent adherence to immunosuppressive therapy. Notably, severe hypertension exacerbated her renal impairment, necessitating immediate intervention. The clinical progression of CGN highlights the critical importance of early diagnosis, continuous monitoring, and a multidisciplinary approach for effective management. Emphasizing patient education and lifestyle modifications, particularly in dietary habits and blood pressure control, can significantly influence treatment outcomes. This case illustrates the urgency of addressing CGN and the necessity of a collaborative healthcare strategy to improve prognosis and enhance the patient's quality of life.

Antitumour activity of oleanolic acid: A systematic review and meta‑analysis.

Oleanolic acid (OA), a compound known for its potent antitumour properties, has been the subject of investigations in both cell and animal models. Although OA has good biological activity, its low water solubility and bioavailability limit its therapeutic use, and therefore translating the potential of OA into the clinical oncology setting remains challenging. The present systematic review and meta-analysis utilized evidence from animal model studies to gain insights into the antitumour mechanisms of OA to address the gap in understanding, and to provide guidance for future research directions and potential clinical applications. The guidelines outlined in the Preferred Reporting Items for Systematic Reviews and Meta-Analyses were applied in the present study and a comprehensive search was conducted across the PubMed/MEDLINE, Web of Science, Cochrane Library and Embase databases, with a cut-off date of June 30, 2023. The primary focus was on randomized controlled trials that used animal models to assess the antitumour effects of OA. The methodological quality appraisal was conducted using the Systematic Review Centre for Laboratory Animal Experimentation risk of bias tool, and tumour volume and weight served as the principal outcome measures. Data were analysed using the RevMan (version 5.3) and Stata SE11 software packages, with an assessment of heterogeneity conducted using the I2 statistical test, sensitivity analysis conducted using the leave-one-out approach, and evaluation of publication bias performed using Egger's test and funnel plot analysis. The present study demonstrated a significant inhibitory effect of OA intervention on tumour growth and a decrease in tumour weight in animal models. Despite the broad spectrum of antitumour effects exhibited by OA, further investigations are warranted to optimize the dosage and administration routes of OA to maximize its efficacy in clinical cancer treatment.

Treatment of Macrolide-resistant Mycoplasma pneumoniae Pneumonia in Children: A Meta-analysis of Macrolides Versus Tetracyclines.

The global prevalence of macrolide-resistant Mycoplasma pneumoniae (MRMP) pneumonia infections, particularly in children, is on the rise. It is imperative to assess the clinical efficacies of alternative antibiotics such as tetracyclines to ensure effective treatment, mitigate antibiotic resistance, enhance clinical outcomes, and minimize the spread of resistant strains among MRMP-infected children. The objective of this study was to compare the therapeutic efficacies of macrolides and tetracyclines in treating MRMP pneumonia in children. We systematically searched the literature to identify comparative studies that examined the clinical outcomes of macrolide and tetracycline antibiotics in children with MRMP pneumonia. We conducted a meta-analysis of the mean duration of fever, hospital stay duration, therapeutic efficacies, and time to defervescence to compare macrolides and tetracyclines. Eleven studies involving 1143 patients compared the clinical efficacies of macrolides and tetracyclines in children with MRMP pneumonia. The studies were conducted in China, Japan, and Korea, and the outcomes of febrile days, hospital stay duration, therapeutic efficacy, and time to defervescence were analyzed. The macrolides studied were azithromycin and clarithromycin, whereas the tetracyclines included minocycline and doxycycline. The pooled estimate of 5 studies showed that the mean duration of febrile days and hospital stay was longer in the macrolides group than tetracycline group [weighted mean difference = 1.64 days, 95% confidence interval (CI): 0.68-2.59, weighted mean difference = 1.22 days, 95% CI: 0.82-1.62, respectively]. The therapeutic efficacy was significantly lower in the macrolide group than in the tetracycline group (odds ratio: 0.33, 95% CI: 0.20-0.57). The clinical efficacy of tetracycline treatment was superior to that of macrolide treatment in children with MRMP pneumonia. However, further research is required to validate these findings and inform evidence-based clinical practice guidelines.

Cepharanthine sensitizes gastric cancer cells to chemotherapy by targeting TRIB3-FOXO3-FOXM1 axis to inhibit autophagy

BackgroundGastric cancer is among the common solid tumors. Chemotherapy resistance is the most common issue in gastric cancer treatment. Inhibiting intracellular autophagy may be a feasible method for overcoming chemotherapy resistance. Cepharanthine (CEP), a natural small molecule extracted from the stephania cephalantha Hayata plant, has been demonstrated to significantly inhibit cancer growth and can regulate autophagy. Although CEP can significantly inhibit cancer growth, it remains unclear whether CEP can regulate autophagy in gastric cancer. This study aimed to investigate whether CEP can enhance the sensitivity of gastric cancer to chemotherapy and elucidate its molecular mechanism. MethodsThree gastric cancer cell lines (AGS, SGC7901, and MFC) and one normal gastric mucosal epithelial cell line (GES-1) were used for in vitro experiments. The characterization of autophagy in gastric cancer cells included the detection of autophagy markers and autophagy flux through immunofluorescence staining and Western blotting, as well as the assessment of lysosomal function using fluorescence staining (LysoTracker Red DND-99, Acridine Orange staining) and Western blotting. The cytotoxicity of CEP, autophagy inhibitors (chloroquine [CQ] and 3-methyladenine [3MA]), and chemotherapy drugs (doxorubicin [DOX] and cisplatin [CIS]) was evaluated using the 3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyltetrazolium bromide assay, cell colony formation, and fluorescence staining techniques (H2DCFDA, Dihydroethidium, and JC-1 staining). The interaction between CEP and autophagy inhibitors was tested in a 615 mice model, and changes in the gut microbiota were determined through accurate 16S absolute quantification sequencing. The signaling pathway and autophagy regulatory target TRIB3-FOXO3-FOXM1 were confirmed through molecular docking, RNA sequencing, bioinformatic analysis, transfection techniques, and Western blotting. ResultsCEP blocked autophagic flux in gastric cancer cells without affecting lysosomal function. As a novel autophagy inhibitor, CEP could combine with conventional autophagy inhibitors (CQ and 3MA) to block intracellular autophagy, thereby inhibiting gastric cancer growth. During this process, changes in the gut microbiota were observed, including low-level changes in Odoribacterium, Erysipelatoclostridium, and ParaPrevotella and high-level changes in Ileibacterium, Enterorhabdus, and Bifidobacterium. Additionally, CEP synergistically inhibited the growth of gastric cancer when combined with chemotherapy drugs. Mechanistically, the TRIB3-FOXO3-FOXM1 signaling axis was found to be involved in the inhibition of gastric cancer by CEP combined with autophagy inhibitors and chemotherapy drugs, thereby mediating cell apoptosis. ConclusionThis study links the TRIB3-FOXO3-FOXM1 axis with chemotherapy efficacy. Our findings demonstrated that CEP inhibits autophagy by modulating the FOXO3-FOXM1 axis. When combined with chemotherapy drugs (DOX and CIS), CEP, as an autophagy inhibitor, can limit TRIB3 protein expression, thereby regulating the FOXO3-FOXM1 axis and enhancing its ability to prevent gastric cancer growth. These findings may contribute to improving the prognosis of patients with gastric cancer. Furthermore, these results enrich the fundamental understanding of how autophagy inhibition can enhance clinical cancer treatment efficacy and provide insights into the potential mechanisms by which CEP functions as an anti-tumor drug, thereby exploring its value for clinical application.

Prospective Randomized Controlled Clinical Trial to Evaluate the Safety and Efficacy of ACTLINK Plasma Treatment for Promoting Osseointegration and Bone Regeneration in Dental Implants.

Recent studies have explored surface treatments, such as increasing the hydrophilicity of implant fixtures, to enhance the osseointegration of implants. This prospective clinical study aimed to assess the clinical stability and efficacy of plasma treatment applied to implants with sandblast-acid etching (SLA) surfaces before placement. Twenty-eight patients requiring implant placement provided consent and were assigned randomly to either the SLA group without plasma treatment or the SLA/plasma group with plasma treatment. Recall checks were conducted one and three months after the first-stage surgery, followed by a second surgery at four months. Although no significant differences in buccal bone defects or implant stability were observed between the groups, the SLA/plasma group showed significant increases in marginal bone changes on the mesial and distal sides, as assessed using periapical radiographs. This study underscores the potential of pre-implantation plasma treatment to enhance bone regeneration around implants.

Evaluation of Chemokines MIG and IP-10 as Immunological Biomarkers of Human Visceral Leishmaniasis: A Systematic Review.

Visceral leishmaniasis (VL) is a neglected tropical disease that is potentially fatal when untreated. Current diagnostic methods have limitations that contribute to ongoing transmission and poor prognosis. Thus, new tests are needed to provide quick, accurate diagnoses and evaluate clinical progression and treatment efficacy. The monokine induced by interferon-gamma (MIG) and interferon-gamma-inducible protein 10 (IP-10) has been associated with the host susceptibility to VL with potential diagnostic and prognostic purposes. We performed a systematic review using four search databases (Scopus, PubMed, Web of Science, and MEDLINE) to identify studies assessing MIG and IP-10 as potential biomarkers in patients with VL across various clinical conditions. A total of 13 studies were potentially eligible and included in this review. The articles, in general, reveal that the chemokines MIG and IP-10 are elevated in response to infection by Leishmania spp., acting on the host's resistance to the development of the disease. They are associated with asymptomatic conditions and after VL treatment, and this relationship can be observed in both immunocompetent and immunocompromised individuals. Consequently, these chemokines hold relevance in the diagnoses and appropriate management of individuals with VL.

Clinical efficacy and safety of perampanel monotherapy as primary anti-seizure medication in the treatment of pediatric epilepsy: A single-center, prospective, observational study.

To assess the efficacy and safety of perampanel (PER) as primary monotherapy in patients aged 4-18 years old with epilepsy. A single-center, prospective, observational study was conducted from October 2021 to October 2023, to evaluate PER monotherapy's efficacy and safety as initial therapy for pediatric epilepsy. Changes in seizure frequency, safety, and retention rate were observed at 3, 6, 9, and 12 months after initiating PER primary monotherapy. A total of 124 children aged 4-15 years (mean age = 8.25 ± 2.50 years) were included in the Analysis Sets. The retention rates at 3, 6, 9, and 12 months were 88.71% (110/124), 84.68% (105/124), 78.26% (90/115), and 71.58% (68/95), respectively. Seizure freedom rates at 3, 6, 9, and 12 months were 85.45%, 79.09%, 76.24%, and 75.31%, respectively. The responder rates (≥50% but <100%) at the same endpoints were 9.09%, 14.55%, 12.87%, and 7.41%, respectively. Seizure freedom rate of PER was independent of age at PER initiation, seizure onset age, gender, baseline frequency, seizure types, and family history of epilepsy (p > 0.05) but associated with duration of treatment (p = 0.001) and maintenance dose (p = 0.022). Additionally, 124 patients were included in the safety analysis set. The overall adverse event rate was 38.71% (48/124), with irritability (19 cases, 15.32%) and dizziness (18 cases, 14.52%) being the most common adverse effects. One patient discontinued PER monotherapy within 1 month due to unbearable itching of the skin. PER monotherapy as the primary anti-seizure medication (ASM) for pediatric epilepsy demonstrates high efficacy and safety in real-world clinical treatment. Patients who respond well to this drug and adhere to long-term treatment can achieve favorable seizure control. Furthermore, patients achieving seizure freedom with a relatively lower dose can opt for the same dose as the maintenance dose. This study provided the efficacy and safety of PER monotherapy as the primary ASM for Chinese pediatric epilepsy. In total, 124 patients took part. The seizure freedom rates were over 70% at different observation points (OPs), along with a retention rate of 71.58% at the 12-month OP. Most of adverse effects observed were mild to moderate.

As a novel prognostic model for breast cancer, the identification and validation of telomere-related long noncoding RNA signatures

BackgroundTelomeres are a critical component of chromosome integrity and are essential to the development of cancer and cellular senescence. The regulation of breast cancer by telomere-associated lncRNAs is not fully known, though. The goals of this study were to describe predictive telomere-related LncRNAs (TRL) in breast cancer and look into any possible biological roles for these RNAs.MethodsWe obtained RNA-seq data, pertinent clinical data, and a list of telomere-associated genes from the cancer genome atlas and telomere gene database, respectively. We subjected differentially expressed TRLs to co-expression analysis and univariate Cox analysis to identify a prognostic TRL. Using LASSO regression analysis, we built a prognostic model with 14 TRLs. The accuracy of the model’s prognostic predictions was evaluated through the utilization of Kaplan-Meier (K-M) analysis as well as receiver operating characteristic (ROC) curve analysis. Additionally, immunological infiltration and immune drug prediction were done using this model. Patients with breast cancer were divided into two subgroups using cluster analysis, with the latter analyzed further for variations in response to immunotherapy, immune infiltration, and overall survival, and finally, the expression of 14-LncRNAs was validated by RT-PCR.ResultsWe developed a risk model for the 14-TRL, and we used ROC curves to demonstrate how accurate the model is. The model may be a standalone prognostic predictor for patients with breast cancer, according to COX regression analysis. The immune infiltration and immunotherapy results indicated that the high-risk group had a low level of PD-1 sensitivity and a high number of macrophages infiltrating. In addition, we’ve discovered a number of small-molecule medicines with considerable for use in treating high-risk groups. The cluster 2 subtype showed the highest immune infiltration, the highest immune checkpoint expression, and the worst prognosis among the two subtypes defined by cluster analysis, which requires more attention and treatment.ConclusionAs a possible biomarker, the proposed 14-TRL signature could be utilized to evaluate clinical outcomes and treatment efficacy in breast cancer patients.

Analysis of clinical efficacy and long-term prognosis of patients with hepatocellular carcinoma treated with PD-L1 inhibitor targeting.

The most effective clinical treatment for hepatocellular carcinoma (HCC) is surgery, but most patients are diagnosed when the disease has progressed. To examine the long-term prognosis and clinical effectiveness of PD-L1 inhibitor-targeted therapy for patients suffering from HCC. Ninety-six patients with advanced HCC who were admitted to our hospital between December 2019 and April 2022 were split into two groups based on the treatment plan after a retrospective analysis: 43 patients in the control group underwent sorafenib-based targeted therapy, while dulvalizumab was used to treat 53 patients in the observation group. Observation indexes were used to assess the clinical effectiveness and long-term prognosis of HCC patients receiving targeted therapy with dulvalizumab, which included the disease control rate, tumor markers, immune function, survival, quality of survival, and the occurrence of unfavorable side effects such as thrombocytopenia, leukopenia, vomiting, and rash. The initial KPS scores, CEA, CA199, AFP, CD3+, CD4+, CD4+/CD8+, IgG, IgM, and IgA levels did not differ significantly between the two groups (P> 0.05). After treatment, the observation group showed a significantly higher disease control rate (92.45% vs. 74.42%) and improved KPS score, OS, PFS, CD3+, CD4+, CD4+/CD8+, IgG, IgM, and IgA levels compared to the control group. Additionally, the observation group exhibited significantly reduced CEA, CA199, and AFP levels, and a lower overall incidence of adverse reactions (16.98% vs. 51.16%) compared to the control group (P< 0.05). The clinical efficacy of dulvalizumab-targeted treatment of HCC among PD-L1 inhibitors is better, enhancing the disease's ability to be controlled considerably lowering patients' levels of tumor markers. This greatly boosts patients' immune systems, extends their lives and improves the quality of their survival. The frequency of negative reactions is minimal and safe.

Expanding clinical characteristics and genotypic profiling of Yao syndrome in Chinese patients.

Yao syndrome (YAOS, OMIM# 617321) is a kind of systemic autoinflammatory diseases (SAIDs) linked to the nucleotide-binding oligomerization domain containing 2 (NOD2). Clinical reports of YAOS in China are sparse. Herein, we reported the largest YAOS cohort of Chinese patients to expand the understanding of its phenotype, genotype, and therapeutic responses. This study enrolled 15 adult patients diagnosed with YAOS at Peking Union Medical College Hospital from April 2015 to May 2024. Whole-exome sequencing was performed on all patients. Clinical data, genetic variations, and treatment responses were documented and compared with a Caucasian cohort. The mean age of disease onset was 35 ± 17 years old. The most common clinical manifestations included recurrent high-grade fever (100%), gastrointestinal symptoms (73.3%), arthralgia/arthritis, fatigue, myalgia, and lower extremity swelling (46.7%). All patients exhibited elevated acute-phase reactants during episodes. 12 heterozygous NOD2 variants were identified, with Q902K in 4 patients, R471C in 3, and variants c.-14C>T, A110T, S127L, R311W, A432V, Y514H, R541P, A661P, K818Q, A886V each found in individual patients. 90% of the patients responded well to glucocorticoids, and 55.6% to sulfasalazine. 66.7% of patients who received TNF inhibitors achieved complete resolution of symptoms. Additionally, one patient each responded favorably to canakinumab and tocilizumab. Compared to the Caucasian cohort, our cohort exhibited a more balanced gender ratio and a higher proportion of recurrent fever, proteinuria/hematuria as well as more frequent leukocytosis, elevated acute phase reactants, and anemia. Lower proportions of arthralgia/arthritis, skin rashes, headaches, and sicca-like symptoms were noted in our cohort. Moreover, a higher proportion of patients in our cohort showed a good response to TNF inhibitors. Chinese patients with YAOS had more pronounced inflammatory manifestations compared to the Caucasian cohort. Variants c.-14C>T, A110T, S127L, A661P, K818Q, A886V, R471C, and A432V were identified as novel NOD2 variants in YAOS. TNF, IL-6, and IL-1 inhibitors are the promising treatment options. These findings expand the clinical spectrum, genetic profile, and treatment efficacy of YAOS, underscoring the need for heightened awareness of this disease in diverse populations.

Hyaluronic acid-covered ferric ion-rich nanobullets with high zoledronic acid payload for breast tumor-targeted chemo/chemodynamic therapy

Due to the heterogeneity of the tumor microenvironment, the clinical efficacy of tumor treatment is not satisfied, highlighting the necessity for new strategies to tackle this issue. To effectively treat breast tumors by tumor-targeted chemo/chemodynamic therapy, herein, the Fe3+-rich MIL-88B nanobullets (MNs) covered with hyaluronic acid (HA) were fabricated as vehicles of zoledronic acid (ZA). The attained ZA@HMNs showed a high ZA payload (ca 29.6 %), outstanding colloidal stability in the serum-containing milieu, and accelerated ZA as well as Fe3+ release under weakly acidic and glutathione (GSH)-rich conditions. Also, the ZA@HMNs consumed GSH by GSH-mediated Fe3+ reduction and converted H2O2 into OH via Fenton or Fenton-like reaction with pH reduction. After being internalized by 4T1 cells upon CD44-mediated endocytosis, the ZA@HMNs depleted intracellular GSH and degraded H2O2 into OH, thus eliciting lipid peroxidation and mitochondria damage to suppress cell proliferation. Also, the ZA@HMNs remarkably killed macrophage-like RAW 264.7 cells. Importantly, the in vivo studies and ki67 and GPX4 staining of tumor sections demonstrated that the ZA@HMNs efficiently accumulated in 4T1 tumors to hinder tumor growth via ZA chemotherapy combined with OH-mediated ferroptosis. This work presents a practicable strategy to fabricate ZA@HMNs for breast tumor-targeted chemo/chemodynamic therapy with potential clinical translation.

Poor outcome despite modern treatments: A retrospective study of 99 patients with primary and secondary plasma cell leukemia.

Plasma cell leukemia (PCL) is a rare monoclonal gammopathy, associated with short survival. Because of its very low incidence, only a few cohorts have been reported and thus, information on this disease is scarce. The goal of this study was to better understand the clinical features, prognostic factors, and efficacy of modern treatments in both primary PCL (pPCL) and secondary PCL (sPCL). We performed a retrospective, multicenter study of patients diagnosed with PCL, defined as circulating plasma cells ≥20% of total leukocytes and/or ≥2 × 109/L. We identified 99 eligible PCL patients, of whom 33 were pPCL and 66 were sPCL. The median progression-free survival (PFS) to frontline treatment and overall survival (OS) were, respectively, 4.8 (95% CI, 0.4-9.2) and 18.3 months (95% CI, 0.0-39.0) for pPCL and 0.8 (95% CI, 0.5-1.1) and 1.2 months (95% CI, 0.9-1.5) for sPCL (both p < 0.001). We observed no improvement in OS over time (2005-2012 vs. 2013-2020, p = 0.629 for pPCL and p = 0.329 for sPCL). Finally, our data suggested that sPCL originates from a high-risk multiple myeloma (MM) population with a short OS (median 30.2 months), early relapse after stem cell transplant (median 11.9 months) and a high proportion of patients with multiple cytogenetic abnormalities (36% with ≥2 abnormalities). This study is one of the largest PCL cohorts reported. We are also the first to investigate characteristics of MM before its transformation into sPCL and demonstrate that high-risk biologic features already present at the time of MM diagnosis. Moreover, our data highlights the lack of improvement in PCL survival in recent years and the urgent need for better treatment options.

Impact of Microsurgery and Postoperative Radiotherapy on Neurological Function in Intramedullary Spinal Cord Gliomas.

To assess the clinical efficacy of combined microsurgery and postoperative radiotherapy for the treatment of intramedullary spinal gliomas and its impact on neurological function. An observational study. Place and Duration of the Study: Department of Neurosurgery, Baoding No.1 Central Hospital, Hebei, China, between January 2020 and 2023. Sixty patients diagnosed with spinal cord intramedullary gliomas were divided equally into an experimental and control group. The control group received microsurgical treatment, and the experimental group received microsurgical treatment combined with postoperative radiotherapy. The treatment effectiveness, neurological function, and follow-up results of the two groups were compared. After treatment, the clinical efficacy of the experimental group treatment was significantly better than that of the control group (p <0.05). The National Institutes of Health Stroke Scale (NIHSS) scores were significantly lower, and the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-30 (EORTC QLQ-C30) scores were significantly higher in the experimental group than in the control group (p <0.05). The 1-3-year survival rate and median survival time of the experimental group were significantly higher than those of the control group (p <0.05). The incidence of complications was 3.33% in the experimental group and 6.67% in the control group, but the difference was not statistically significant (p >0.05). The postoperative recurrence rate was significantly lower in the experimental (0%) than in the control group (13.33%, p <0.05). Combined microsurgery and postoperative radiotherapy was found to be more effective than microsurgery alone. It was also more conducive to the recovery of neurological function and improved the patient's quality of life. Intramedullary spinal cord glioma, Microsurgery, Neurological function, Radiotherapy.

Discussion on the upper limit of HIFU sound pressure measurement based on laser interferometry

Abstract High intensity focused ultrasound (HIFU) technology is a non-invasive treatment technique that can achieve non-invasive treatment of tumors and other diseases. The accuracy of HIFU sound pressure is crucial for the clinical efficacy and safety of HIFU treatment. The sound pressure measurement technology based on laser interferometry is the recommended method for ICE TC87, which has the advantages of high measurement accuracy and good traceability, and is therefore widely adopted as the new generation of sound pressure standards. However, the upper limit of the laser interferometric HIFU sound pressure measurement currently reported is about 10 MPa, which is far lower than the actual sound pressure of HIFU technology. In response to this issue, the upper limit of HIFU sound pressure measurement was discussed from the perspective of laser interference systems. The mathematical relationship between the HIFU sound pressure and the optical and electrical signals of the laser interference system under the condition of nonlinear sound field is established. The mathematical analysis of HIFU sound pressure requirements for the bandwidth of laser interference systems is discussed. Using experimental and numerical simulation methods, the effect of HIFU sound pressure on the bandwidth of a laser interference system under nonlinear acoustic field conditions was studied.

Recent advances in the diagnosis of rectal cancer and evaluation of clinical treatment efficacy using magnetic resonance imaging

Accurate preoperative tumor staging is essential for precise treatment of rectal cancer, and magnetic resonance imaging (MRI) is the preferred method. In addition to accurately determining tumor (T) and stages node (N), MRI can also be used to evaluate factors related to the prognosis of rectal cancer, such as depth of tumor invasion into the muscular layer, extramural vascular invasion of the tumor, and distance to the circumferential resection margin. MRI is also an important method for evaluating tumor regression after neoadjuvant therapy, and quantitative biomarkers of functional MRI can objectively reflect treatment efficacy. Moreover, MRI can predict response after neoadjuvant therapy, local recurrence rate, and disease-free survival before treatment. The present article reviews recent advances in the preoperative diagnosis and staging of rectal cancer, reassessment after neoadjuvant therapy, and evaluation of clinical treatment efficacy using MRI.

Comparing the clinical trial efficacy versus real-world effectiveness of treatments for multiple myeloma: a population-based study.

Not available.