treatment outcomes

治疗结果
  • 文章类型: Journal Article
    小儿复发性多软骨炎(RP)是一种罕见的自身免疫性疾病,可引起儿童炎症和软骨损伤。常见的症状包括疼痛,耳朵肿胀和畸形,鼻子,气管,接头,和眼睛。缺乏对儿科人群的研究,有必要对儿科RP的文献进行进一步评估,以总结现有的表现模式。管理,和治疗。
    从1947年至2023年4月,对遵守2020PRISMA清单的21岁以下患者的RP进行了系统评价。仅包括符合McAdamRP标准并包括管理信息的患者陈述。
    从304项初步研究来看,54项研究纳入最终分析,共68例患者,以女性为主(65%)。平均诊断延迟1年,平均发病年龄为12岁.最常见的症状包括双侧耳廓软骨炎(69%),鼻软骨炎症(62%),和呼吸道软骨炎(63%)。文献中最常见的初始检查信息通常包括CT/MRI(72%),支气管镜检查(57%),活检(51%),和实验室(88%),最常见的是ESR升高(59%)。最常见的药物是皮质类固醇(91%)和甲氨蝶呤(35%),最常见的手术治疗是气管切开术(38%)。最有效的治疗选择是单克隆抗体(87%,n=15)和皮质类固醇(66%,n=62)用于22%和91%的患者,分别。最常用的单克隆抗体疗法是英夫利昔单抗(13%,n=9)。
    小儿RP最常见的表现包括耳软骨炎,鼻子,和呼吸道。最有效的治疗方案包括皮质类固醇和单克隆抗体治疗,如英夫利昔单抗。我们的发现强调了抗风湿药和单克隆抗体治疗的缓解增加,尤其是皮质类固醇.
    UNASSIGNED: Pediatric Relapsing Polychondritis (RP) is a rare autoimmune disorder that causes inflammation and damage to cartilage in children. Common symptoms include pain, swelling and deformities in the ears, nose, trachea, joints, and eyes. The lack of research on the pediatric population necessitates further evaluation of the literature on pediatric RP to summarize existing patterns in presentation, management, and treatment.
    UNASSIGNED: A systematic review was conducted on PubMed and Embase from 1947 to April 2023 on RP in patients under 21 years old abiding by the 2020 PRISMA checklist. Only patient presentations meeting McAdam criteria for RP and including information on management were included.
    UNASSIGNED: From the 304 initial studies, 54 studies were included for final analysis with a total of 68 patients, who were predominantly female (65%). With a median diagnostic delay of 1 year, the mean age of onset was 12 years old. The most common symptoms on presentation included bilateral auricular chondritis (69%), nasal cartilage inflammation (62%), and respiratory tract chondritis (63%). The most commonly reported information in the literature for the initial workup usually included CT/MRI (72%), bronchoscopy (57%), biopsy (51%), and labs (88%), which most commonly displayed elevated ESR (59%). The most common medications were corticosteroids (91%) and methotrexate (35%) and the most common procedural treatment was tracheostomy (38%). The most efficacious treatment options were monoclonal antibodies (87%, n = 15) and corticosteroids (66%, n = 62) used in 22% and 91% of patients, respectively. The most commonly used monoclonal antibody therapy was infliximab (13%, n = 9).
    UNASSIGNED: The most common presentation for pediatric RP includes chondritis of the ear, nose, and respiratory tract. The most effective treatment options include corticosteroids and monoclonal antibody therapy, such as infliximab. Our findings highlight increasing remission achieved with anti-rheumatic drugs and monoclonal antibody treatment, especially alongside corticosteroids.
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  • 文章类型: Journal Article
    目的:确定结果测量工具(OMIs)以评估声带萎缩和/或沟患者的治疗效果。
    方法:通过搜索Pubmed和EMBASE对2021年3月之前发布的记录进行系统审查。纳入的研究报告成人(>18岁)由于声带萎缩引起的声门功能不全,伴有或不伴有沟,他们被纳入一项随机对照试验,一项非随机对照试验,病例对照研究或队列研究。所有纳入的研究都描述了至少一项结果测量的干预措施。
    结果:共确定了5456项研究。删除重复项后,筛选标题和摘要,并对选定的记录进行全文筛选,最终分析包括34种出版物。根据DeJonckere等人的ELS方案,从这50个单独的OMI中进行记录和分类。(欧耳拱门奥托鼻喉258:77-82,2001)。由于大多数OMI被用于多项研究,报告的OMI总数为265。十九(19)个单独的OMI占报告的80%。根据类别,最常用的OMI是:VHI和VHI-10(主观评估);GRBAS的G(感知评估);F0,抖动和闪光(声学评估);MPT和MFR(空气动力学评估)以及声门闭合和粘膜波(内窥镜评估)。在这些OMI中,VHI具有90%的高的显著性百分比。
    结论:本系统评价确定了由于声带萎缩和/或沟而导致声门功能不全的患者中最常用的OMI,这是朝着为该人群定义核心结果集(COS)迈出的一步。
    238274。
    OBJECTIVE: Identifying outcome measurements instruments (OMIs) to evaluate treatment efficacy in patients with vocal fold atrophy and/or sulcus.
    METHODS: Systematic review of records published before March 2021 by searching Pubmed and EMBASE. Included studies reported on adults (> 18 year) with dysphonia caused by glottic insufficiency due to vocal fold atrophy with or without sulcus, who were enrolled into a randomized controlled trial, a non-randomized controlled trial, a case-controlled study or a cohort study. All included studies described an intervention with at least one outcome measurement.
    RESULTS: A total of 5456 studies were identified. After removing duplicates, screening title and abstract and full text screening of selected records, 34 publications were included in final analysis. From these 50 separate OMIs were recorded and categorized according to the ELS protocol by DeJonckere et al. (Eur Arch Otorhinolaryngol 258: 77-82, 2001). With most OMIs being used in multiple studies the total number of OMIs reported was 265. Nineteen (19) individual OMIs accounted for 80% of reports. The most frequently used OMIs according to category were: VHI and VHI-10 (subjective evaluation); G of GRBAS (perceptual evaluation); F0, Jitter and Shimmer (acoustic evaluation); MPT and MFR (aerodynamic evaluation) and glottic closure and mucosal wave (endoscopic evaluation). Of these OMIs VHI had a high percentage of significance of 90%.
    CONCLUSIONS: This systematic review identifies the most used OMIs in patients with glottic incompetency due to vocal fold atrophy and/or sulcus as a step toward defining a Core Outcome Set (COS) for this population.
    UNASSIGNED: 238274.
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  • 文章类型: Journal Article
    背景:产科瘘是由产科并发症引起的生殖道与下泌尿和/或胃肠道之间的异常开口,影响了全世界近200万女性。它强加了物理,经济,社会,以及对受影响妇女的心理后果。治疗结果各不相同,大多数情况下,手术治疗可改善存活者的生活质量,并成功再次妊娠.
    目的:本综述旨在绘制和检查中低收入国家产科瘘外科修复的治疗结果。
    方法:这是一项范围审查研究,旨在确定中低收入国家产科瘘手术修复的治疗结果。搜索是从数据库中进行的(PUBMED,Embase,CINAHL,Scopus,和WebofScience),和灰色文学(谷歌学者,Google,和会议记录)。
    方法:资格标准是使用参与者构建的,概念,和背景框架,并包括主要研究的研究类型,reviews,和报告。没有全文和英语以外语言的研究被排除在外。
    方法:在Excel电子表格中提取纳入研究的相关特征,并进行分析以绘制治疗结果。
    结果:本综述审查了57项关于产科瘘治疗结果的研究全文。研究结果分为两个主题:早期结果和晚期结果。早期结果包括尿失禁,手术部位感染,尿潴留,出血,并保留导管。晚期结果包括瘘管复发,残余失禁,生活质量,生殖问题,心理健康,家庭和社会支持,和财务状况。
    结论:产科瘘的治疗结果可分为短期和长期结果。尽管这篇综述发现了足够的分析研究,大多数研究设计都很糟糕。建议将来进行更强有力的研究,以指导政策和决策。我们建议研究人员对短期和长期结果独立进行系统评价和荟萃分析。
    BACKGROUND: Obstetrical fistula is an abnormal opening between the reproductive tract and lower urinary and/or gastrointestinal tract resulting from obstetrical complications, affecting nearly two million women worldwide. It imposes physical, economic, social, and mental consequences on the affected women. Treatment outcomes vary and, mostly, surgical treatment results in improved quality of life and successful subsequent pregnancy for survivors.
    OBJECTIVE: The review aimed to chart and examine the treatment outcomes of obstetrical fistula surgical repair in low- and middle-income countries.
    METHODS: This is a scoping review study to identify treatment outcomes of obstetrical fistula surgical repair in low- and middle-income countries. The search was conducted from databases (PUBMED, Embase, CINAHL, Scopus, and Web of Science), and gray literature (Google Scholar, Google, and conference proceedings).
    METHODS: The eligibility criteria were constructed using a participant, concept, and context framework and included study types of primary research, reviews, and reports. Studies without full text and in languages other than English were excluded.
    METHODS: The relevant characteristics of the included studies were extracted on an Excel spreadsheet and analyzed to chart treatment outcomes.
    RESULTS: The review examined the full text of 57 studies on the treatment outcomes of obstetrical fistula. The findings were grouped into two themes: early and late outcomes. The early outcomes included incontinence, surgical-site infection, urine retention, hemorrhage, and retained catheter. The late outcomes included fistula recurrence, residual incontinence, quality of life, reproductive issues, mental health, family and social support, and financial status.
    CONCLUSIONS: The treatment outcomes of obstetrical fistula can be grouped into short-term and long-term outcomes. Although this review found adequate studies for the analysis, most study designs were poor. Stronger studies are recommended in the future to guide policy and decision-making. We would like to suggest that researchers conduct systematic reviews and meta-analyses independently for short-term and long-term outcomes.
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  • 文章类型: Journal Article
    这篇全面的综述研究了精神疾病背景下家庭治疗对表达情绪(EE)的影响。EE,以高度批评为特征,敌意,或者情感上的过度参与,一直与较差的治疗结果和精神症状严重程度增加相关.该综述探讨了各种家庭治疗方法及其在降低精神病患者家庭高EE水平方面的有效性。它综合了现有的文献,突出了EE变化的潜在机制,例如改变沟通模式和增强家庭凝聚力。此外,这篇综述讨论了对临床实践的影响,强调将家庭治疗纳入精神病治疗计划的重要性,并提供心理教育,使家庭能够有效地管理情绪。还概述了未来的研究方向,包括调查家庭治疗带来的变化的长期可持续性,并探索治疗方法中的文化考虑。总的来说,该综述强调了家庭治疗在解决精神病患者及其家人的EE和促进康复和复原力方面的关键作用.
    This comprehensive review examines the impact of family therapy on expressed emotions (EE) within the context of psychiatric disorders. EE, characterized by high levels of criticism, hostility, or emotional over-involvement, have been consistently linked to poorer treatment outcomes and increased severity of psychiatric symptoms. The review explores various family therapy approaches and their effectiveness in reducing high EE levels in families of psychiatric patients. It synthesizes existing literature to highlight the mechanisms underlying the changes in EE, such as modifying communication patterns and enhancing family cohesion. Additionally, the review discusses the implications for clinical practice, emphasizing the importance of integrating family therapy into psychiatric treatment plans and providing psychoeducation to empower families to manage emotions effectively. Future research directions are also outlined, including investigating the long-term sustainability of changes brought about by family therapy and exploring cultural considerations in therapeutic approaches. Overall, the review underscores the pivotal role of family therapy in addressing EE and promoting recovery and resilience in psychiatric patients and their families.
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  • 文章类型: Journal Article
    就患者预后而言,关于前列腺癌(PCa)最有效的主要治疗方法存在争议,如手术或放射治疗(RT)。这项研究评估了前列腺癌根治术(RP)和RT治疗PCa的比较疗效和长期预后。对相关数据库进行了全面的文献综述,专注于2019年以来发表的学术和临床研究。纳入标准包括随机对照试验(RCT)和其他观察性研究,比较接受手术和RT治疗的患者的生存结果。我们遵循系统评价和荟萃分析(PRISMA)指南的首选报告项目,以提供数据的概述。我们根据纳入标准选择了19项研究。在总共19项研究中,12主张RP作为改善PCa患者生存结果的首选治疗方法。我们的综合结果显示,接受RT治疗的患者的前列腺癌特异性死亡率(PCSM)较低。分析的总效应大小计算为Z=1.19(p值=0.23)。研究中的异质性如下:Tau2=0.09,Chi2=20.25,df=4,I2=80%。此外,结果显示,接受前列腺切除术的患者的总生存期(OS)更高.发现分析的组合效应为:HR=0.97(0.93,1.01)。总效应计算为Z=1.33(p值=0.18)。发现异质性为Tau2=0.00,Chi2=1.33,df=2,I2=0%。然而,总死亡率(OM)与治疗方式无关.RT是PCa治疗的首选策略,因为它平衡了疗效和长期结果。临床决策应考虑患者的个体特征,未来的研究应深入研究特定的亚群和长期结果,以进一步完善治疗指南。
    There is controversy regarding the most effective primary treatment of choice for prostate cancer (PCa) in terms of patient outcomes, such as surgery or radiotherapy (RT). This study evaluated the comparative efficacy and long-term outcomes of radical prostatectomy (RP) and RT for PCa treatment. A thorough literature review of relevant databases was conducted, focusing on academic and clinical studies published from 2019 onwards. The inclusion criteria included randomized controlled trials (RCTs) and other observational studies comparing survival outcomes in patients treated with surgery and RT. We followed the Preferred Reporting Items for Systematic Review and Meta-Analysis (PRISMA) guidelines to provide an overview of the data. We selected 19 studies based on the inclusion criteria. Of the total 19 studies, 12 advocated RP as the preferred treatment to improve survival outcomes in patients with PCa. The results of our synthesis showed that prostate cancer-specific mortality (PCSM) was lower in patients treated with RT. The total effect size for the analysis was calculated as Z=1.19 (p-value=0.23). The heterogeneity in the studies was as follows: Tau2=0.09, Chi2=20.25, df=4, I2=80%. Moreover, overall survival (OS) was shown to be higher in patients who underwent prostatectomy. The combined effect for the analysis was found to be: HR=0.97 (0.93, 1.01). The total effect was calculated as Z=1.33 (p-value= 0.18). The heterogeneity was found to be Tau2=0.00, Chi2=1.33, df=2, and I2=0%. However, overall mortality (OM) was shown to be independent of the treatment modality. RT is the preferred strategy for PCa treatment, as it balances efficacy and long-term outcomes. Clinical decision-making should consider individual patient characteristics and future research should delve into specific subpopulations and long-term outcomes to further refine the treatment guidelines.
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  • 文章类型: Journal Article
    目的:本范围审查旨在评估当前文献中关于乡村性的以下结果:诊断阶段,临床特征,治疗特点,和头颈部癌症(HNC)的生存结果。
    方法:使用PubMed(MEDLINE)进行文献检索,科学直接,EMBASE,Scopus,和WebofScience数据库。
    方法:从最初的研究开始,20年的研究截止时间被用来增加关于人群和临床护理标准的研究的可比性。这些搜索旨在捕获所有报告HNC发生率的主要研究。呈现特点,治疗,和治疗结果。两名审稿人独立筛选摘要,选择排除的文章,提取的数据,和评估的研究。严格评估是根据JoannaBriggs研究所队列研究质量评估工具进行的。
    结果:包括20篇合格的原创文章。诊断阶段,临床特征,治疗特点,和生存结果进行了测量。我们的审查表明,尽管这种关系尚不清楚,根据地理位置和农村居住状况,喉癌的治疗选择可能会有所不同。评估HNC结果的研究与诊断阶段相关,临床特征,治疗特点,总体生存率显示出相互矛盾的发现,表明需要进一步研究检查HNC结果,重点是将农村作为主要暴露。
    结论:HNC与城乡状况之间的关系尚不清楚。需要更多的研究,以及用于衡量农村和城市地区可比人口的农村和招募的一致指标。喉镜,2024.
    OBJECTIVE: This scoping review sought to evaluate the current literature regarding the following outcomes in relation to rurality: stage at diagnosis, clinical characteristics, treatment characteristics, and survival outcomes of head and neck cancer (HNC).
    METHODS: A literature search was performed using PubMed (MEDLINE), Science Direct, EMBASE, SCOPUS, and Web of Science databases.
    METHODS: A 20-year study cutoff from the initial search was used to increase the comparability of the studies regarding population and standards of clinical care. These searches were designed to capture all primary studies reporting HNC incidence, presenting characteristics, treatments, and treatment outcomes. Two reviewers independently screened abstracts, selected articles for exclusion, extracted data, and appraised studies. Critical appraisal was done according to the Joanna Briggs Institute Quality Assessment Tool for Cohort Studies.
    RESULTS: Twenty eligible original articles were included. Stage at diagnosis, clinical characteristics, treatment characteristics, and survival outcomes were measured. Our review indicates that although this relationship is unclear, there may be variations in treatment choice for laryngeal cancer based on geographic location and rural residency status. The studies assessing HNC outcomes related to stage at diagnosis, clinical characteristics, treatment characteristics, and overall survival demonstrated conflicting findings, indicating a need for further research examining HNC outcomes with a focus on rurality as the main exposure.
    CONCLUSIONS: The relationship between HNC and rural-urban status remains unclear. More studies are needed, along with a consistent metric for measuring rurality and recruitment of comparable populations from both rural and urban areas. Laryngoscope, 2024.
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  • 文章类型: Journal Article
    与原发肿瘤部位相比,转移部位对治疗的抵抗力更强,对治疗方案的反应也不同.这可能是由于转移部位和原发性肿瘤之间微环境的异质性。肿瘤相关成纤维细胞(CAFs)作为肿瘤微环境的关键成分广泛存在于肿瘤基质中。原发性肿瘤CAFs(pCAFs)和转移性CAFs(mCAFs)在来源方面是异质的,激活模式,标记和功能表型。它们可以根据器官塑造肿瘤微环境,显示原发性肿瘤和转移瘤之间的异质性,这可能会影响这些部位对治疗的敏感性。假设了解pCAFs和mCAFs之间的异质性可以提供对治疗结果差异的一瞥。为提高各种癌症的转移控制率提供新思路。
    Compared with primary tumor sites, metastatic sites appear more resistant to treatments and respond differently to the treatment regimen. It may be due to the heterogeneity in the microenvironment between metastatic sites and primary tumors. Cancer‑associated fibroblasts (CAFs) are widely present in the tumor stroma as key components of the tumor microenvironment. Primary tumor CAFs (pCAFs) and metastatic CAFs (mCAFs) are heterogeneous in terms of source, activation mode, markers and functional phenotypes. They can shape the tumor microenvironment according to organ, showing heterogeneity between primary tumors and metastases, which may affect the sensitivity of these sites to treatment. It was hypothesized that understanding the heterogeneity between pCAFs and mCAFs can provide a glimpse into the difference in treatment outcomes, providing new ideas for improving the rate of metastasis control in various cancers.
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  • 文章类型: Journal Article
    大疱性表皮松解症(EB)是一种遗传性皮肤病,缺乏有效的治疗方法,需要对其严重的支持治疗,危及生命的表现。骨髓移植(BMT)及其衍生细胞已被认为可以改善临床症状和生活质量。对PubMed/MEDLINE中评估BMT和骨髓间充质干细胞(BM-MSC)治疗EB的出版物进行了全面搜索,谷歌学者,和Cochrane数据库从成立到2023年6月。共有55名患有严重EB形式的参与者患有BMT和/或BM-MSC,以隐性营养不良型EB最为常见;53例(96.4%)患者伤口愈合较好,3例(5.5%)患者死于脓毒症。报告的最常见的不良事件是移植物失败,脓毒症,移植物抗宿主病,和肾功能不全。同种异体BMT是一种具有可能的益处和不良事件的高风险手术。BM-MSCs显示出有利的结果,可通过将严重不良事件的风险降至最低来提高EB细胞治疗的安全性。减少水泡,加速伤口愈合.需要进一步的研究来评估治疗的长期效果,并阐明手术与常规治疗的风险/获益比。
    Epidermolysis bullosa (EB) is a genodermatosis that lacks effective treatments and requires supportive care for its severe, life-threatening manifestations. Bone marrow transplantation (BMT) and its derived cells have been suggested to improve clinical symptoms and quality of life. A comprehensive search was conducted for publications evaluating BMT and bone marrow-derived mesenchymal stem cell (BM-MSC) therapy for EB in PubMed/MEDLINE, Google Scholar, and Cochrane databases from inception until June 2023. A total of 55 participants with severe forms of EB had BMT and/or BM-MSCs, with recessive dystrophic EB as the most common EB type; 53 (96.4%) patients had better wound healing, and 3 (5.5%) patients died of sepsis. The most common adverse events reported were graft failure, sepsis, graft-versus-host disease, and renal insufficiency. Allogeneic BMT is a high-risk procedure with possible benefits and adverse events. BM-MSCs revealed favorable outcomes to improve the safety of EB cell-based therapy by minimizing the risk of serious adverse events, reducing blisters, and accelerating wound healing. Further studies are needed to assess the treatment\'s long-term effects and clarify the risk/benefit ratio of procedure versus conventional therapy.
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  • 文章类型: Systematic Review
    关于药物和酒精治疗的交叉,文献基础有限,暴力,和创伤。虽然研究证实,接触暴力和创伤会影响滥用药物的倾向,临床试验和实践中的概念化在很大程度上是狭隘和性别化的,仅指亲密伴侣或家庭暴力。我们的系统映射审查探索了对暴力和创伤的幸存者和肇事者的更具包容性和更广泛的审查(例如,亲密伴侣暴力,性侵犯,跟踪,虐待儿童,政治和社区暴力,刑事暴力,微暴力,结构性暴力,和压迫)建立:1)干预研究中包括的治疗设置类型,2)记录的成功或共同结果的共同指标,和3)了解谁正在寻求治疗药物和酒精使用与暴力的历史。
    进行了系统的绘图审查,以确定2011年至2022年发表的任何同行评审文章。WebofScience数据库是使用一系列与暴力相关的布尔搜索词进行搜索的,物质使用障碍,和治疗。从系统审查中确定了8,800多条记录,共有48篇文章符合纳入标准。
    本综述中的大多数研究包括报告暴力行为的人群(n=23,48%)与报告创伤/暴力存活的参与者(n=17,35%)。结果还表明,女性识别人群(n=19;40%)主要得到服务,在美国接受治疗(n=33;69%),在门诊就诊(n=24;50%)。作者还关注包括性和性别少数群体的研究,发现只有三项研究(6%)明确承认纳入跨性别参与者或参与者与同性伴侣的关系;另外三项研究(6%)集中于具有性工作历史或从事性工作的参与者。
    本综述概述了直接位于本综述中发现的服务提供差距中的治疗和研究意义。具体来说,结果阐明了对基于性别的小型化和排斥身份的影响,性偏好,刑事法律地位和研究和治疗方向,以增加包容性,representation,以及研究和治疗环境中的公平性。
    UNASSIGNED: There is a limited literature base regarding the intersection of drug and alcohol treatment, violence, and trauma. While research substantiates that exposure to violence and trauma impacts the propensity to misuse substances, the conceptualization in clinical trials and practice has largely been narrow and gendered, referring only to intimate partner or domestic violence. Our systematic mapping review explored a more inclusive and expansive review of survivors of and perpetrators of violence and trauma (e.g., intimate partner violence, sexual assault, stalking, child abuse, political and community violence, criminal violence, micro violence, structural violence, and oppression) to establish: 1) the types of treatment settings included in intervention studies, 2) the common indicators of success or common outcomes recorded, and 3) understanding who is seeking treatment for drug and alcohol use with histories of violence.
    UNASSIGNED: A systematic mapping review was conducted to identify any peer-reviewed articles published from 2011 to 2022. The Web of Science database was searched using a broad set of Boolean search terms related to violence, substance use disorders, and treatment. Over 8,800 records were identified from the systematic review with a total of 48 articles meeting inclusion criteria.
    UNASSIGNED: Most studies in this review included populations reporting perpetration of violence (n=23, 48%) versus participants reporting survival of trauma/violence (n=17, 35%). Results also indicated female identifying populations (n=19; 40%) were predominantly served, were treated in the US (n=33; 69%) and seen in an outpatient setting (n=24; 50%). Authors also were attentive to studies that included sexual and gender minorities and discovered only three studies (6%) explicitly acknowledging inclusion of transgender participants or participants in relationship with partners of the same sex; three more studies (6%) were focused on participants with histories of or engaging in sex work.
    UNASSIGNED: This review outlines treatment and research implications directly situated in the gap of service delivery found in this review. Specifically, the results elucidate the impact on minoritized and excluded identities based on gender, sexual preference, criminal legal status and directions for research and treatment to increase inclusion, representation, and equity across research and treatment settings.
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  • 文章类型: Review
    治疗晚期甲状腺癌由于其对各种治疗方式的抵抗力而面临挑战,从而限制了治疗选择。据我们所知,这项研究首次报道了替西罗莫司联合纳武单抗/ipilimumab双重免疫疗法治疗重度治疗的晚期PDTC的疗效.一名50岁的女性最初表现为右脖子上的肿块迅速扩大。随后的诊断显示低分化甲状腺癌,导致甲状腺全切除术,然后进行术后放射消融治疗。四年后,对持续性咳嗽的检查显示该疾病在多个纵隔淋巴结中复发。血液样本的遗传分析发现了肿瘤中的体细胞突变,特别涉及PTEN和TP53。尽管有姑息性辐射,疾病还是进展了,lenvatinib,和nivolumab/ipilimumab治疗。因此,替西罗莫司,作为mTOR抑制剂,作为nivolumab/ipilimumab方案的辅助手段。这种组合方法在大约六个月的持续时间内产生了显着的临床改善和疾病控制。坦西罗莫司可能抑制异常激活的PI3K/AKT/mTOR信号通路,由PTEN基因改变促进,从而产生有效的治疗反应。靶向药物和免疫疗法之间的这种协同作用为具有有限治疗选择的晚期PDTC患者提供了有希望的治疗策略。在之前的临床试验中,mTOR抑制剂已证明有能力在65%至74%的晚期甲状腺癌患者中维持稳定的疾病(SD),包括PDTC。当与其他靶向治疗相结合时,观察到的SD或部分缓解率范围为80%至97%。许多试验主要涉及分化型甲状腺癌,具有不同的基因突变。PI3K/mTOR/Akt改变的甲状腺癌患者似乎最受益于mTOR抑制剂。然而,mTOR抑制剂的疗效与特定组织学或基因突变之间没有明确关联.未来的研究有必要阐明这些关联。
    Treating advanced thyroid cancer presents challenges due to its resistance to various treatment modalities, thereby limiting therapeutic options. To our knowledge, this study is the first to report the efficacy of temsirolimus in conjunction with dual immunotherapy of nivolumab/ipilimumab to treat heavily treated advanced PDTC. A 50-year-old female initially presented with a rapidly enlarging mass on her right neck. Subsequent diagnosis revealed poorly differentiated thyroid carcinoma, leading to a total thyroidectomy followed by post-operative radioablation therapy. After four years, an examination for persistent cough revealed a recurrence of the disease within multiple mediastinal nodes. Genetic analysis of blood samples uncovered somatic mutations in the tumor, specifically involving PTEN and TP53. The disease progressed despite palliative radiation, lenvatinib, and nivolumab/ipilimumab therapy. Consequently, temsirolimus, functioning as an mTOR inhibitor, was introduced as an adjunct to the nivolumab/ipilimumab regimen. This combination approach yielded remarkable clinical improvement and disease control for a duration of approximately six months. Temsirolimus likely suppressed the aberrantly activated PI3K/AKT/mTOR signaling pathway, facilitated by the PTEN genetic alteration, thus engendering an effective treatment response. This synergy between targeted agents and immunotherapy presents a promising therapeutic strategy for advanced PDTC patients with limited treatment alternatives. In previous clinical trials, mTOR inhibitors have demonstrated the ability to maintain stable disease (SD) in 65% to 74% for advanced thyroid cancer patients, including those with PDTC. When combined with other targeted therapies, the observed SD or partial response rates range from 80% to 97%. Many of these trials primarily involved differentiated thyroid carcinoma, with diverse genetic mutations. Thyroid cancer patients with alterations in the PI3K/mTOR/Akt appeared to benefit most from mTOR inhibitors. However, no clear association between the efficacy of mTOR inhibitors and specific histologies or genetic mutations has been established. Future studies are warranted to elucidate these associations.
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