progression‐free survival

无进展生存期
  • 文章类型: Journal Article
    目的:高级别胶质瘤(HGG)是高度恶性的,侵略性,发病率和死亡率都很高。这项研究的目的是调查HGs患者的生存结果和预后因素。
    方法:在这项回顾性研究中,共纳入了159例经组织学证实的HGG患者.招聘期为2011年1月至2019年12月。我们评估了患者的人口统计数据,肿瘤特征,治疗方法,免疫细胞化学结果,总生存期(OS)时间,和无进展生存期(PFS)时间使用Kaplan-<>Meier生存分析与对数秩检验。此外,我们采用Cox回归分析来确定与生存结局相关的独立因素.
    结果:Kaplan-Meier生存分析显示,1-,2-,5年OS率为81.8%,50.3%,12.6%,分别。同样,1-,2-,5年PFS率为50.9%,22.4%,和3.1%,分别。中位OS持续时间为35.0个月。单因素分析显示术后病理分型,grade,和年龄与患者预后显著相关(p<0.01)。在患者中,147人接受同步放化疗,12没有。ki-67、MGMT、IDH1R132H,和p53在预后影响方面表现出统计学上的显着差异(分别为p=0.001,p=0.020,p=0.003和p=0.021)。总之,我们发现成绩,年龄,病理分类,ki-67,MGMT,和IDH1R132H表达与PFS有统计学意义(p<0.01,p=0.004,p=0.003,p=0.001,p=0.036,p=0.028)。此外,在生存期较短的患者中,TRIB3和AURKA的免疫组织化学表达显着升高(p=0.015和p=0.023)。
    结论:肿瘤分级和术后同步放化疗是影响患者生存的独立预后因素。此外,肿瘤分级和MGMT表达是影响无进展生存期(PFS)的独立因素.值得注意的是,在生存结局较差的患者中,TRIB3和AURKA的表达较高.
    OBJECTIVE: High-grade gliomas (HGGs) are highly malignant, aggressive, and have a high incidence and mortality rate. The aim of this study was to investigate survival outcomes and prognostic factors in patients with HGGs.
    METHODS: In this retrospective study, a total of 159 patients with histologically confirmed HGGs were included. The recruitment period was from January 2011 to December 2019. We evaluated patient demographic data, tumor characteristics, treatment methods, immunocytochemistry results, overall survival (OS) time, and progression-free survival (PFS) time using Kaplan-<>Meier survival analysis with log-rank testing. Additionally, we employed Cox regression analysis to identify independent factors associated with survival outcomes.
    RESULTS: Kaplan-Meier survival analysis revealed that the 1-, 2-, and 5-years OS rates were 81.8%, 50.3%, and 12.6%, respectively. Similarly, the 1-, 2-, and 5-years PFS rates were 50.9%, 22.4%, and 3.1%, respectively. The median OS duration was 35.0 months. The univariate analysis indicated that postoperative pathological classification, grade, and age were significantly associated with patient outcomes (p < 0.01). Among the patients, 147 received concurrent chemoradiotherapy, while 12 did not. The immunohistochemical markers of ki-67, MGMT, IDH1R132H, and p53 demonstrated statistically significant differences in their prognostic impact (p = 0.001, p = 0.020, p = 0.003, and p = 0.021, respectively). In conclusion, we found that grades, age, pathological classification, ki-67, MGMT, and IDH1R132H expression were statistically significantly associated with PFS (p < 0.01, p = 0.004, p = 0.003, p = 0.001, p = 0.036, and p = 0.028). Additionally, immunohistochemical expressions of TRIB3 and AURKA were significantly higher in patients with shorter survival (p = 0.015 and p = 0.023).
    CONCLUSIONS: Tumor grade and the use of concurrent chemoradiotherapy after surgery were independent prognostic factors that significantly influenced patient survival. Additionally, tumor grade and MGMT expression were found to be independent factors affecting progression-free survival (PFS). Notably, the expression of TRIB3 and AURKA was higher in patients with poor survival outcomes.
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  • 文章类型: Journal Article
    目的:目的是在对现有文献进行系统综述的基础上,确定自体造血干细胞移植(aHSCT)作为进行性多发性硬化(PMS)治疗干预措施的价值。
    方法:系统回顾了2024年2月之前以英文发表的PubMed和GoogleScholar数据库中提供PMS患者个体数据的所有研究。PICO被定义为人口(P),原发性进展性MS和继发性进展性MS患者;干预(I),用aHSCT治疗;比较(C),无,疾病改善治疗/复发缓解型MS队列(如果可用);结果(O),移植相关死亡率,无进展生存期(PFS),没有疾病活动的证据。
    结果:共有15项研究符合标准,包括665名PMS患者(74名原发性进展型MS,591例继发性进行性MS)和801例复发缓解型MS患者作为对照。有647例患者的PFS数据。PMS患者在基线时表现出比复发缓解型MS患者更严重的残疾。在10项研究中,PMS的平均移植相关死亡率为1.9%,528例患者中有10例死亡。治疗开始后5年,PMS组的PFS范围为0%至78%,表现出高度的可变性。没有证据表明5年时的疾病活动性评分范围为0%至75%。
    结论:根据现有数据,aHSCT不停止进步与PMS的人。然而,在选定的患者中似乎有改善预后的证据.由于现有数据的异质性,迫切需要更全面的临床试验来评估aHSCT在不同患者组中的疗效,以减少变异性并改善患者分层.
    OBJECTIVE: The aim was to determine the value of autologous haematopoietic stem cell transplantation (aHSCT) as a therapeutic intervention for progressive multiple sclerosis (PMS) based on a systematic review of the current literature.
    METHODS: All studies from the databases PubMed and Google Scholar published in English before February 2024 which provided individual data for PMS patients were systematically reviewed. PICO was defined as population (P), primary progressive MS and secondary progressive MS patients; intervention (I), treatment with aHSCT; comparison (C), none, disease-modifying therapy treated/relapsing-remitting MS cohorts if available; outcome (O), transplant-related mortality, progression-free survival (PFS) and no evidence of disease activity.
    RESULTS: A total of 15 studies met the criteria including 665 patients with PMS (74 primary progressive MS, 591 secondary progressive MS) and 801 patients with relapsing-remitting MS as controls. PFS data were available for 647 patients. PMS patients showed more severe disability at baseline than relapsing-remitting MS patients. The average transplant-related mortality for PMS in 10 studies was 1.9%, with 10 deaths in 528 patients. PFS ranged from 0% to 78% in PMS groups 5 years after treatment initiation, demonstrating a high variability. No evidence of disease activity scores at 5 years ranged from 0% to 75%.
    CONCLUSIONS: Based on the available data, aHSCT does not halt progression in people with PMS. However, there appears to be evidence of improved outcome in selected patients. Due to the heterogeneity of the available data, more comprehensive clinical trials assessing the efficacy of aHSCT across different patient groups are urgently needed to reduce variability and improve patient stratification.
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  • 文章类型: Journal Article
    背景:早期发现高危鼻咽癌(NPC)复发至关重要。我们通过结合三个机器学习(ML)模型来预测非转移性NPC患者的无进展生存期(PFS),从而创建了机器学习衍生的预后特征(MLDPS)。
    方法:将653例非转移性NPC患者的队列分为训练(n=457)和验证(n=196)数据集(7:3比例)。该研究包括临床病理特征,血液学标志物,和MRI在三种机器学习模型-随机森林(RF)中的发现,极端梯度提升(XGBoost),和最小绝对收缩和选择算子(LASSO)-预测无进展生存期(PFS)。维恩图从三种ML算法中识别出重叠的签名。Cox比例风险分析确定了PFS的MLDPS。
    结果:RF,XGBoost,LASSO算法从33个签名中确定了6个共识因子。Cox比例风险分析表明,MLDPS包括年龄,淋巴细胞计数,阳性淋巴结数,和区域淋巴结密度。此外,MLDPS有效分层预后,低风险个体比高风险个体表现出更好的PFS(p<0.001)。
    结论:MLDPS,根据临床病理特征,血液学标志物,和MRI检查结果,对于指导非转移性NPC患者的临床管理和个性化治疗至关重要。
    BACKGROUND: Early detection of high-risk nasopharyngeal carcinoma (NPC) recurrence is essential. We created a machine learning-derived prognostic signature (MLDPS) by combining three machine learning (ML) models to predict progression-free survival (PFS) in patients with non-metastatic NPC.
    METHODS: A cohort of 653 patients with non-metastatic NPC was divided into a training (n = 457) and validation (n = 196) dataset (7:3 ratio). The study included clinicopathological characteristics, hematologic markers, and MRI findings in three machine learning models-random forest (RF), extreme gradient boosting (XGBoost), and least absolute shrinkage and selection operator (LASSO)-to predict progression-free survival (PFS). A Venn diagram identified the overlapping signatures from the three ML algorithms. Cox proportional hazard analysis determined the MLDPS for PFS.
    RESULTS: The RF, XGBoost, and LASSO algorithms identified six consensus factors from the 33 signatures. Cox proportional hazards analysis showed that the MLDPS includes age, lymphocyte count, number of positive lymph nodes, and regional lymph node density. Additionally, MLDPS effectively stratified prognosis, with low-risk individuals showing better PFS than high-risk individuals (p < 0.001).
    CONCLUSIONS: MLDPS, based on clinicopathological characteristics, hematologic markers, and MRI findings, is crucial for guiding clinical management and personalizing treatments for patients with non-metastatic NPC.
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  • 文章类型: Journal Article
    目的:本研究旨在评估局部治疗(LT)的疗效,包括放疗(RT)和肿瘤细胞减灭术(CRP),改善寡转移前列腺癌(OmPCa)患者的预后。
    方法:对PubMed,Embase,和2010年至2023年11月出版的WebofScience进行了研究。这项研究包括11篇文章,包括3项随机对照试验(RCT)和8项回顾性分析。该研究评估了总生存期(OS),放射学无进展生存期(rPFS),前列腺特异性抗原(PSA)PFS,癌症特异性生存率(CSS),并发症发生率(CR)。
    结果:LT组OS明显改善,RCT和非RCT均显示统计学意义[风险比(HR)=0.64;95%置信区间(95%CI),0.51-0.80;p<0.0001;HR=0.55;95%CIs,0.40-0.77;p=0.0004]。对于rPFS,随机对照试验结果无统计学意义(HR=0.60;95%CI,0.34-1.07;p=0.09),而非随机对照试验显示了显著的结果(HR=0.42;95%CI,0.24-0.72;p=0.002)。RCT和非RCT均显示PSA-PFS显着改善(HR=0.44;95CI,0.29-0.67;p=0.0001;HR=0.51;95%CIs,0.32-0.81;p=0.004)。对于CSS,RCT显示出统计学差异(HR=0.65;95%CIs,0.47-0.90;p=0.009),而非随机对照试验则没有(HR=0.61;95%CI,0.29-1.27;p=0.19)。关于CR,风险差为-0.22(95%CI,-0.32至-0.12;p<0.00001)。
    结论:LT显著改善OmPCa患者的OS和PFS。需要进一步的RCT来证实这些结果。
    OBJECTIVE: This study aims to evaluate the efficacy of local treatment (LT), including radiotherapy (RT) and cytoreductive prostatectomy (CRP), in improving outcomes for patients with oligometastatic prostate cancer (OmPCa).
    METHODS: A systematic review and meta-analysis of articles from PubMed, Embase, and Web of Science published between 2010 and November 2023 were conducted. The study included 11 articles, comprising three randomized controlled trials (RCTs) and eight retrospective analyses. The study assessed overall survival (OS), radiographic progression-free survival (rPFS), prostate-specific antigen (PSA) PFS, cancer-specific survival (CSS), and complication rate (CR).
    RESULTS: OS was significantly improved in the LT group, with both RCTs and non-RCTs showing statistical significance [hazard ratios (HR) = 0.64; 95% confidence intervals (95% CIs), 0.51-0.80; p < 0.0001; HR = 0.55; 95% CIs, 0.40-0.77; p = 0.0004]. For rPFS, RCTs did not show statistically significant outcomes (HR = 0.60; 95% CIs, 0.34-1.07; p = 0.09), whereas non-RCTs demonstrated significant results (HR = 0.42; 95% CIs, 0.24-0.72; p = 0.002). Both RCTs and non-RCTs showed a significant improvement in PSA-PFS (HR = 0.44; 95%CI, 0.29-0.67; p = 0.0001; HR = 0.51; 95% CIs, 0.32-0.81; p = 0.004). For CSS, RCTs demonstrated statistical differences (HR = 0.65; 95% CIs, 0.47-0.90; p = 0.009), whereas non-RCTs did not (HR = 0.61; 95% CIs, 0.29-1.27; p = 0.19). Regarding CR, the risk difference was -0.22 (95% CIs, -0.32 to -0.12; p < 0.00001).
    CONCLUSIONS: LT significantly improved OS and PFS in patients with OmPCa. Further RCTs are necessary to confirm these results.
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  • 文章类型: Journal Article
    背景:本研究的目的是评估以氟喹替尼为基础的疗法作为晚期或转移性肉瘤患者的抢救疗法的疗效和安全性,包括软组织肉瘤(STS)和骨肉瘤。
    方法:将晚期或转移性肉瘤患者分为两组。一组接受氟喹替尼单药治疗,而另一例则接受了氟喹替尼联合治疗。对以氟喹替尼为基础的治疗的安全性和有效性进行了记录和回顾性评价。包括无进展生存期(PFS),总反应率(ORR),和不良事件(AE)。
    结果:在2021年8月至2022年12月之间,回顾性纳入了38例肉瘤患者。共有14例患者单独接受了氟喹替尼(包括6例STS和8例骨肉瘤),而24例接受了氟喹替尼联合治疗(包括2例STS和22例骨肉瘤)。中位随访时间为10.2个月(95%CI,6.4-11.5)。对于整个人口来说,中位PFS为8.0个月(95%CI,5.5-13.0).ORR为13.1%,疾病控制率(DCR)为86.8%。单因素分析显示放疗史(HR,4.56;95%CI,1.70-12.24;p=0.003),骨肉瘤(HR,0.34;95%CI,0.14-0.87;p=0.024),和氟喹替尼的治疗方法(HR,0.36;95%CI,0.15-0.85;p=0.021)与PFS显著相关。多因素分析显示,无放疗史患者PFS较好(HR,3.71;95%CI:1.31-10.55;p=0.014)比有放疗史的患者多。联合组患者报告气胸(8.3%),白细胞减少症(33.3%),血小板减少症(12.5%),腹泻(4.2%),贫血(4.2%)是最常见的3级或更高的治疗紧急AE(TEAE),而单药治疗组无严重TEAE发生。
    结论:基于Fruquintinib的治疗在晚期或转移性肉瘤患者中显示出最佳的肿瘤控制和可接受的安全性。
    BACKGROUND: The purpose of this study was to evaluate the efficacy and safety of fruquintinib-based therapy as a salvage therapy for patients with advanced or metastatic sarcoma, including soft tissue sarcoma (STS) and bone sarcoma.
    METHODS: Patients with advanced or metastatic sarcoma were divided into two groups. One group received fruquintinib monotherapy, while the other received fruquintinib combined therapy. Safety and efficacy of fruquintinib-based therapy were recorded and reviewed retrospectively, including progression-free survival (PFS), overall response rate (ORR), and adverse events (AEs).
    RESULTS: Between August 2021 and December 2022, 38 sarcoma patients were retrospectively included. A total of 14 patients received fruquintinib alone (including 6 STS and 8 bone sarcoma), while 24 were treated with fruquintinib combined therapy (including 2 STS and 22 bone sarcoma). The median follow-up was 10.2 months (95% CI, 6.4-11.5). For the entire population, the median PFS was 8.0 months (95% CI, 5.5-13.0). The ORR was 13.1%, while the disease control rate (DCR) was 86.8%. The univariate analysis showed that radiotherapy history (HR, 4.56; 95% CI, 1.70-12.24; p = 0.003), bone sarcoma (HR, 0.34; 95% CI, 0.14-0.87; p = 0.024), and treatment method of fruquintinib (HR, 0.36; 95% CI, 0.15-0.85; p = 0.021) were significantly associated with PFS. The multivariate analysis showed that patients without radiotherapy history were associated with a better PFS (HR, 3.71; 95% CI: 1.31-10.55; p = 0.014) than patients with radiotherapy history. Patients in combination group reported pneumothorax (8.3%), leukopenia (33.3%), thrombocytopenia (12.5%), diarrhea (4.2%), and anemia (4.2%) as the most frequent grade 3 or higher treatment-emergent AEs (TEAEs), while there was no severe TEAEs occurred in the monotherapy group.
    CONCLUSIONS: Fruquintinib-based therapy displayed an optimal tumor control and an acceptable safety profile in patients with advanced or metastatic sarcoma.
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  • 文章类型: Journal Article
    目的:本研究旨在评估lenvatinib射频消融(RFA)序贯疗法对某些肝细胞癌(HCC)患者的疗效和安全性。
    方法:回顾性招募了119例Child-PughA中期不可切除的HCC患者。lenvatinibRFA序贯治疗组的患者最初接受lenvatinib,其次是RFA和lenvatinib的再治疗。该研究比较了总生存期(OS),无进展生存期(PFS),肿瘤反应,接受序贯治疗和乐伐替尼单药治疗的患者之间的不良事件(AE)。
    结果:在倾向得分匹配后,对25例接受序贯疗法的患者和50例接受单一疗法的患者进行了评估。影响OS的独立因素被确定为序贯治疗,改良白蛋白-胆红素(mALBI)等级,和相对剂量强度(%),风险比(HRs)为0.381(95%置信区间[CI],0.186-0.782),2.220(95%CI,1.410-3.493),和0.982(95%CI,0.966-0.999),分别。基于mALBI等级的分层分析证实了治疗策略对所有mALBI等级对OS的独立影响(HR,0.376;95%CI,0.176-0.804)。此外,序贯治疗被确定为PFS的独立因素(HR,0.382;95%CI,0.215-0.678)。序贯疗法在生存获益方面显著优于单一疗法(OS:38.27vs.18.96个月的序贯疗法和单一疗法,分别,p=0.004;PFS:13.80vs.5.32个月的序贯疗法和单一疗法,分别,p<0.001)。通过改良的实体瘤反应评估标准,序贯治疗与完全缓解显着相关(比值比,63.089)。119例患者中有10例出现3级不良事件,未观察到超过3级的AE。
    结论:与乐伐替尼单药治疗相比,乐伐替尼RFA序贯治疗可能提供良好的耐受性和潜在的预后改善。
    OBJECTIVE: This study aims to evaluate the efficacy and safety of lenvatinib radiofrequency ablation (RFA) sequential therapy for certain hepatocellular carcinoma (HCC) patients.
    METHODS: One hundred and nineteen patients with unresectable HCC in the intermediate stage with Child-Pugh A were retrospectively recruited in a multicenter setting. Those in the lenvatinib RFA sequential therapy group received lenvatinib initially, followed by RFA and the retreatment with lenvatinib. The study compared overall survival (OS), progression-free survival (PFS), tumor response, and adverse events (AEs) between patients undergoing sequential therapy and lenvatinib monotherapy.
    RESULTS: After propensity score matching, 25 patients on sequential therapy and 50 on monotherapy were evaluated. Independent factors influencing OS were identified as sequential therapy, modified albumin-bilirubin (mALBI) grade, and relative dose intensity (%) with hazard ratios (HRs) of 0.381 (95% confidence interval [CI], 0.186-0.782), 2.220 (95% CI, 1.410-3.493), and 0.982 (95% CI, 0.966-0.999), respectively. Stratified analysis based on mALBI grades confirmed the independent influence of treatment strategy across all mALBI grades for OS (HR, 0.376; 95% CI, 0.176-0.804). Furthermore, sequential therapy was identified as an independent factor of PFS (HR, 0.382; 95% CI, 0.215-0.678). Sequential therapy significantly outperformed monotherapy on survival benefits (OS: 38.27 vs. 18.96 months for sequential therapy and monotherapy, respectively, p = 0.004; PFS: 13.80 vs. 5.32 months for sequential therapy and monotherapy, respectively, p < 0.001). Sequential therapy was significantly associated with complete response by modified Response Evaluation Criteria in Solid Tumors (odds ratio, 63.089). Ten of 119 patients experienced grade 3 AEs, with no AE beyond grade 3 observed.
    CONCLUSIONS: Lenvatinib RFA sequential therapy might offer favorable tolerability and potential prognostic improvement compared to lenvatinib monotherapy.
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  • 文章类型: Journal Article
    目的:本研究的目的是探讨腹膜后副神经节瘤(RPGL)患者的长期预后危险因素,并检查其临床和病理特征。
    方法:使用免疫组织化学(IHC)鉴定生物标志物的表达,并对病例数据库进行回顾性检索。使用Kaplan-Meier和Cox风险回归进行生存分析,以确定影响RPGL患者术后无进展生存的因素。
    结果:共有105名患者,其中大多数患有位于主动脉旁区域的肿瘤,平均肿瘤大小为8.6厘米,参加了这项研究。平均随访时间为51个月,死亡率为19%,复发和转移率为41.9%。使用改良的肾上腺嗜铬细胞瘤和副神经节瘤(GAPP)分级系统评估肿瘤,和SDHB,在所有情况下使用IHC对S-100和Ki-67进行染色。在检查的所有病例中,在18.1%的病例中观察到SDHB阴性表达,36.2%的病例中S-100表达为阴性,大约25.7%的病例存在血管内肿瘤。单因素分析结果表明,以下几个因素显着影响PGL患者的无进展生存期:最大肿瘤直径(>5.5cm),肿瘤形态学特征,肿瘤分级(改良GAPP评分>6),SDHB阴性,S-100阴性,增殖指数Ki-67表达(>3%)(X2=4.217-27.420,p<0.05)。多变量分析结果表明,S-100(p=0.021)和SDHB(p=0.038)阴性,以及血管内肿瘤血栓(p=0.047)的表达是患者无进展生存期的独立危险因素。
    结论:RPGL的特点是生物学特征多样,对复发和转移的易感性升高。SDHB和S-100都可以作为传统的IHC指标来预测PGL的转移风险。而肿瘤组织形态学-血管内肿瘤囊泡有助于确定RPGL的转移风险。
    OBJECTIVE: The aim of this study is to explore the long-term prognostic risk factors associated with patients diagnosed with retroperitoneal paraganglioma (RPGL) and examine their clinical and pathological characteristics.
    METHODS: Expressions of biomarkers were identified using immunohistochemistry (IHC) and case databases were retrospectively searched. Survival analysis was performed using Kaplan-Meier and Cox risk regression to identify the factors that influence the postoperative progression-free survival of patients with RPGL.
    RESULTS: A total of 105 patients, most of whom had tumors situated in the paraaortic region, and whose average tumor size was 8.6 cm, were enrolled in this study. The average follow-up duration was 51 months, with a mortality rate of 19% and a recurrence and metastasis rate of 41.9%. Tumors were assessed using the modified Grading system for Adrenal Pheochromocytoma and Paraganglioma (GAPP), and SDHB, S-100, and Ki-67 were stained using IHC in all cases. Out of the total cases examined, negative in SDHB expression were observed in 18.1% of cases, S-100 expression was negative in 36.2% of cases, and endovascular tumor enboluswas present in approximately 25.7% of cases. The results of the univariate analysis indicated that several factors significantly influenced the progression-free survival of patients with PGL as follow: maximum tumor diameter (>5.5 cm), tumor morphological features, tumor grading (modified GAPP score > 6), SDHB negative, S-100 negative, and expression of proliferation index Ki-67 (>3%) (X2 = 4.217-27.420, p < 0.05). The results of the multivariate analysis indicated that negative of S-100 (p = 0.021) and SDHB (p = 0.038), as well as intravascular tumor thrombus (p = 0.047) expression were independent risk factors for progression-free survival in patients.
    CONCLUSIONS: RPGL is characterized by diverse biological features and an elevated susceptibility to both recurrence and metastasis. Both SDHB and S-100 can be employed as traditional IHC indicators to predict the metastatic risk of PGL, whereas the tumor histomorphology-endovascular tumor enbolus assists in determining the metastasis risk of RPGL.
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  • 文章类型: Journal Article
    背景:含沙利度胺的治疗方案会引起不良事件(AEs),这可能需要降低多发性骨髓瘤患者的治疗强度或甚至停止治疗。由于沙利度胺的毒性是剂量依赖性的,确定每位患者的最合适剂量至关重要。
    目的:本研究旨在研究沙利度胺剂量递增策略对治疗反应和无进展生存期(PFS)的影响。
    结果:这项前瞻性观察研究包括93例接受硼替佐米治疗的新诊断多发性骨髓瘤(NDMM)患者,沙利度胺,和地塞米松(VTD)。本研究评估了沙利度胺剂量减少和停药的发生率,总剂量强度,以及它们对治疗效果的影响。此外,本研究使用Cox比例风险模型分析了导致沙利度胺不能耐受的因素.结果显示,所有患者和可评价患者的总有效率分别为78.5%和98.7%,分别。未达到研究队列中的中位PFS。最常见的沙利度胺相关不良事件为便秘(32.3%)和皮疹(23.7%),导致剂量减少和停药率为22.6%和21.5%,分别。响应者的平均沙利度胺剂量强度明显高于无响应者(88.6%vs.42.9%,p<.001)。
    结论:沙利度胺剂量递增方法对于接受VTD诱导治疗的NDMM患者是一种可行的选择,具有满意的疗效和耐受性。然而,沙利度胺不耐受可能导致剂量减少或因不可预测的不良事件而停药。导致较低的剂量强度和潜在较差的治疗结果。除了剂量增加策略,最佳支持治疗对于接受VTD诱导治疗的多发性骨髓瘤患者至关重要.
    BACKGROUND: Thalidomide-containing regimens cause adverse events (AEs) that may require a reduction in treatment intensity or even treatment discontinuation in patients with multiple myeloma. As thalidomide toxicity is dose-dependent, identifying the most appropriate dose for each patient is essential.
    OBJECTIVE: This study aimed to investigate the effects of a thalidomide dose step-up strategy on treatment response and progression-free survival (PFS).
    RESULTS: This prospective observational study included 93 patients with newly diagnosed multiple myeloma (NDMM) who received bortezomib, thalidomide, and dexamethasone (VTD). The present study assessed the incidence of thalidomide dose reduction and discontinuation, the overall dose intensity, and their effects on therapeutic efficacy. Furthermore, this study used Cox proportional hazard models to analyze the factors contributing to thalidomide intolerability. The results showed the overall response rates in all patients and the evaluable patients were 78.5% and 98.7%, respectively. The median PFS in the study cohort was not reached. The most common thalidomide-related AEs were constipation (32.3%) and skin rash (23.7%), resulting in dose reduction and discontinuation rates of 22.6% and 21.5%, respectively. The responders had a significantly higher average thalidomide dose intensity than the nonresponders (88.6% vs. 42.9%, p < .001).
    CONCLUSIONS: The thalidomide dose step-up approach is a viable option for patients with NDMM receiving VTD induction therapy with satisfactory efficacy and tolerability. However, thalidomide intolerance may lead to dose reduction or discontinuation due to unpredictable AEs, leading to lower dose intensity and potentially inferior treatment outcomes. In addition to a dose step-up strategy, optimal supportive care is critical for patients with multiple myeloma receiving VTD induction therapy.
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  • 文章类型: Journal Article
    时间至事件终点被广泛用作患者幸福感和预后指标。在基于成像的生物标志物研究中,越来越多的研究专注于检查成像生物标志物对这些终点的预后或预测效用,无论是在试验还是观察性研究环境中。在这篇教育评论文章中,我们简要介绍了时间至事件终点的一些基本概念,并指出了影像学生物标志物研究的潜在陷阱,以期提高放射科医师对相关学科的理解.此外,我们纳入了一些关于使用时间至事件终点的益处的回顾和讨论,以及选择总生存期或无进展生存期进行主要分析的考虑因素.证据水平:5技术效率:第3阶段。
    Time-to-event endpoints are widely used as measures of patients\' well-being and indicators of prognosis. In imaging-based biomarker studies, there are increasingly more studies that focus on examining imaging biomarkers\' prognostic or predictive utilities on those endpoints, whether in a trial or an observational study setting. In this educational review article, we briefly introduce some basic concepts of time-to-event endpoints and point out potential pitfalls in the context of imaging biomarker research in hope of improving radiologists\' understanding of related subjects. Besides, we have included some review and discussions on the benefits of using time-to-event endpoints and considerations on selecting overall survival or progression-free survival for primary analysis. LEVEL OF EVIDENCE: 5 TECHNICAL EFFICACY: Stage 3.
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  • 文章类型: Journal Article
    滤泡性淋巴瘤(FL)是最常见的惰性B细胞非霍奇金淋巴瘤。循环淋巴瘤(CL)细胞可以在某些FL患者的诊断中看到,然而,先前的研究对此进行了评估,结果好坏参半。因此,我们试图使用来自单个中心的数据评估CL在诊断时对新诊断的FL患者结局的影响.通过外周血(PB)流式细胞术根据免疫表型将患者分为CL和CL-。CL定义为与FL的实际或预期B细胞免疫表型匹配的可检测的克隆限制性B细胞。主要终点是一线治疗后的无进展生存期(PFS),次要终点包括总缓解率(ORR)。总生存期(OS),诊断至治疗间隔(DTI),诊断后2年内的疾病进展(POD24),两组间转化的累积发生率。在541例FL患者中,204在诊断时进行了PB流式细胞术,在排除不符合资格标准的患者后,诊断时仍有147例患者24(16%)CL。CL+组的患者年龄较小(53vs.58年,p=0.02),结外受累更多(83%vs.44%,p<0.01),滤泡性淋巴瘤国际预后指数3-5(55%vs.31%,p=0.01),接受一线免疫化疗的比例更高(75%vs.43%,与CL组相比,p=0.01)。CL+之间的中位PFS没有显着差异(6.27年,95%CI=3.61-NR)和CL-(6.61年,95%CI=5.10-9.82)队列,无论一线治疗或绝对PBCL细胞水平如何。ORR没有显著差异,中位数OS,DTI,POD24和两组间转化的累积发生率。在我们的研究中,我们发现,在当代的FL诊断中,CL细胞的存在并不影响预后和生存率.
    Follicular lymphoma (FL) is the most common indolent B-cell non-Hodgkin lymphoma. Circulating lymphoma (CL) cells can be seen at diagnosis in some FL patients, however, previous studies evaluating this have shown mixed results. Therefore, we sought to evaluate the impact of CL at diagnosis on outcomes in patients with newly diagnosed FL using data from a single center. Patients were divided into CL+ and CL- based on immunophenotyping via peripheral blood (PB) flow cytometry. CL was defined as detectable clonally restricted B-cells that matched the actual or expected B-cell immunophenotype of FL. The primary endpoint was progression-free survival (PFS) after first-line treatment and secondary endpoints included overall response rate (ORR), overall survival (OS), diagnosis to treatment interval (DTI), progression of disease within 2 years of diagnosis (POD24), and cumulative incidence of transformation between the two groups. Among the 541 patients with FL, 204 had PB flow cytometry performed at diagnosis, and after excluding patients not meeting the eligibility criteria, 147 cases remained with 24 (16%) CL+ at diagnosis. Patients in the CL+ group were younger (53 vs. 58 years, p = 0.02), had more extranodal involvement (83% vs. 44%, p < 0.01), follicular lymphoma international prognostic index 3-5 (55% vs. 31%, p = 0.01), and a higher proportion received first-line immunochemotherapy (75% vs. 43%, p = 0.01) compared to the CL-group. The median PFS was not significantly different between CL+ (6.27 years, 95% CI = 3.61-NR) and CL- (6.61 years, 95% CI = 5.10-9.82) cohorts regardless of the first-line treatment or level of absolute PB CL cells. There was no significant difference in ORR, median OS, DTI, POD24, and cumulative incidence of transformation between the two groups. In our study, we found that the presence of CL cells at diagnosis in FL in the contemporary era did not impact outcomes and survival.
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