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Integrating patient advocacy groups in the development of clinical practice guidelines: comment on the article by Johnson et al. 将患者权益团体纳入临床实践指南的制定过程。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-28 DOI: 10.1002/acr.25474
Nishant Gupta, Steven E Carsons, Nancy L Carteron, Robert Hal Scofield, Augustine S Lee, Donald E Thomas, Teng Moua, Kamonpun Ussavarungsi, E William St Clair, Richard Meehan, Kieron Dunleavy, Matt Makara, Katherine Morland Hammitt
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引用次数: 0
Relationship Between Number of Different Lower-Limb Resistance Exercises Prescribed in a Program and Exercise Outcomes in People With Knee Osteoarthritis: A Systematic Review With Meta-Regression. 膝关节骨性关节炎患者在一个项目中规定的不同下肢阻力运动的数量与运动结果的关系:一项meta回归的系统综述。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-28 DOI: 10.1002/acr.25476
Belinda J Lawford, Kim L Bennell, Libby Spiers, Alexander J Kimp, Andrea Dell'Isola, Alison R Harmer, Martin Van der Esch, Michelle Hall, Rana S Hinman

Objective: We determine whether there is a relationship between the number of different lower-limb resistance exercises prescribed in a program and outcomes for people with knee osteoarthritis.

Methods: We used a systematic review with meta-regression. We searched the Cochrane Central Register of Controlled Trials, MEDLINE, and Embase up to January 4, 2024. We included randomized controlled trials that evaluated land-based resistance exercise for knee osteoarthritis compared with nonexercise interventions. We conducted meta-regressions between number of different exercises prescribed and standardized mean differences (SMDs) for pain and function. Covariates (intervention duration, frequency per week, use of resistance exercise machine[s], and comparator type) were applied to attempt to reduce between-study heterogeneity.

Results: Forty-four trials (3,364 participants) were included. The number of resistance exercises ranged from 1 to 12 (mean ± SD 5.0 ± 3.0). Meta-regression showed no relationship between the number of prescribed exercises and change in pain (slope coefficient: -0.04 SMD units [95% confidence interval {95% CI} -0.14 to 0.05]) or self-reported function (SMD -0.04 [95% CI -0.12 to 0.05]). There was substantial heterogeneity and evidence of publication bias. However, even after removing 31 trials that had overall unclear/high risk of bias, there was no change in relationships.

Conclusion: There was no relationship between the number of different lower-limb resistance exercises prescribed in a program and change in knee pain or self-reported function. However, given that we were unable to account for all differences in program intensity, progression, and adherence, as well as the heterogeneity and overall low quality of included studies, our results should be interpreted with caution.

目的:确定一个项目中规定的不同下肢阻力运动的数量与膝关节骨关节炎患者的预后之间是否存在关系。方法:采用meta回归法进行系统评价。我们检索了截至2024年1月4日的Cochrane中央对照试验注册库(Central)、MEDLINE和Embase。我们纳入了随机对照试验,以评估陆上阻力运动与非运动干预对膝关节骨关节炎的治疗效果。我们在不同运动的数量和疼痛和功能的标准化平均差异(SMD)之间进行了meta回归。采用协变量(干预持续时间、每周频率、使用阻力运动机和比较者类型)试图减少研究间的异质性。结果:共纳入44项试验(3364名受试者)。阻力运动次数为1-12次(平均5.0;标准偏差3.0)。meta回归显示,规定的运动次数与疼痛变化之间没有关系(斜率系数:-0.04 SMD单位[95%置信区间(CI): -0.14至0.05)或自我报告功能(-0.04[-0.12至0.05])。存在大量异质性和发表偏倚的证据。然而,即使在排除了31项总体偏倚风险不明确/高的试验后,两者之间的关系也没有变化。结论:在一个项目中规定的不同下肢阻力运动的数量与膝关节疼痛或自我报告功能的变化之间没有关系。然而,考虑到我们无法解释项目强度、进展和依从性的所有差异,以及纳入研究的异质性和总体低质量,我们的结果应谨慎解释。
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引用次数: 0
Incidence of Side Effects Associated With Acetaminophen in People Aged 65 Years or More: A Prospective Cohort Study Using Data From the Clinical Practice Research Datalink. 65 岁及以上人群对乙酰氨基酚相关副作用的发生率:利用临床实践研究数据链接数据进行的前瞻性队列研究。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-24 DOI: 10.1002/acr.25471
Jaspreet Kaur, Georgina Nakafero, Abhishek Abhishek, Christian Mallen, Michael Doherty, Weiya Zhang

Objective: The main objective of this study is to examine the safety of oral acetaminophen at its therapeutic dose in adults aged ≥65 years.

Methods: This population-based cohort study used the Clinical Practice Research Datalink-Gold data. Participants were aged ≥65 years registered with a UK general practice for at least 12 months between 1998 and 2018. Acetaminophen exposure was defined as at least two acetaminophen prescriptions within six months of the first acetaminophen prescription, the first prescription date being the index date. Acetaminophen nonexposure was defined as the absence of two acetaminophen prescriptions within six months over the study period. We calculated propensity score (PS) for acetaminophen prescription and undertook inverse probability treatment weighting using PS and PS-matched analyses to account for confounding. Missing data were handled using multiple imputation. The adjusted hazard ratio (aHR) and 95% confidence interval (95% CI) were calculated using the Cox proportional hazards regression model.

Results: In total, 180,483 acetaminophen exposed and 402,478 unexposed participants were included in this study. Acetaminophen exposure was associated with an increased risk of perforation or ulceration or bleeding (aHR 1.24; 95% CI 1.16-1.34), uncomplicated peptic ulcers (aHR 1.20; 95% CI 1.10-1.31), lower gastrointestinal bleeding (aHR 1.36; 95% CI 1.29-1.46), heart failure (aHR 1.09; 95% CI 1.06-1.13), hypertension (aHR 1.07; 95% CI 1.04-1.11), and chronic kidney disease (aHR 1.19; 95% CI 1.13-1.24).

Conclusion: Despite its perceived safety, acetaminophen is associated with several serious complications. Given its minimal analgesic effectiveness, acetaminophen as the first-line oral analgesic option for long-term conditions in older people requires careful reconsideration.

研究目的本研究的主要目的是检查 65 岁及以上成年人口服对乙酰氨基酚治疗剂量的安全性:这项基于人群的队列研究使用了临床实践研究数据-金数据。参与者年龄≥65岁,1998年至2018年期间在英国全科诊所注册至少12个月。对乙酰氨基酚暴露的定义是,在首次开具对乙酰氨基酚处方后的六个月内至少开具过两次对乙酰氨基酚处方,首次开具处方日期为索引日期。对乙酰氨基酚非暴露定义为在研究期间的 6 个月内没有两次对乙酰氨基酚处方。我们计算了对乙酰氨基酚处方的倾向分数(PS),并利用倾向分数和倾向分数匹配分析进行了逆概率治疗加权(IPTW),以考虑混杂因素。缺失数据采用多重估算法处理。采用 Cox 比例危险度回归模型计算调整后危险度比(aHR)和 95% 置信区间(CI):本研究共纳入了 180,483 名对乙酰氨基酚使用者和 402,478 名非使用者。使用对乙酰氨基酚与消化性溃疡出血(aHR 1.24;95% CI 1.16,1.34)、无并发症消化性溃疡(aHR 1.20;95% CI 1.10,1.31)、下消化道出血(aHR 1.36;95% CI 1.29,1.46)、心脏病(aHR 1.09;95% CI 1.06,1.13)、高血压(aHR 1.07;95% CI 1.04,1.11)和慢性肾病(aHR 1.19;95% CI 1.13,1.24):结论:尽管对乙酰氨基酚被认为是安全的,但它与多种严重并发症有关。鉴于对乙酰氨基酚的镇痛效果微乎其微,因此需要重新慎重考虑将对乙酰氨基酚作为治疗老年人长期疾病的一线口服镇痛药。
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引用次数: 0
Introduction to the Special Theme Issue: Environmental and Geographic Factors and Rheumatic Disease 专题特刊简介:环境和地理因素与风湿病。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-24 DOI: 10.1002/acr.25472
Kelli D. Allen, S. Sam Lim
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引用次数: 0
Musculoskeletal Ultrasound Practices of Graduates of a Blended-Learning Program: A Survey of Rheumatologists From the United States. 混合式学习课程毕业生的肌肉骨骼超声实践:美国风湿病学家调查。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-22 DOI: 10.1002/acr.25470
Midori Nishio, Karina D Torralba, Sonja I Ziniel, Eugene Kissin, Fawad Aslam

Objective: Not much is known regarding musculoskeletal ultrasound (MSUS) practices of rheumatologists in the United States. We sought to determine the current use of MSUS among past participants of the Ultrasound School of North American Rheumatologists (USSONAR) training program and, by extension, MSUS-practicing rheumatologists and to understand barriers to its MSUS use.

Methods: An online survey was sent to 374 participants in the eight-month USSONAR blended course (Fundamentals in Musculoskeletal Ultrasound and Train the Trainer) between 2009 and 2020. Each respondent had a unique identifier linked to their total number of submitted practice scans and examination scores during training.

Results: The survey response rate was 28.1% (105 of 374), comprising 82% adult and 18% pediatric rheumatologists. Of the respondents, 71% were MSUS certified: 86.7% performed and/or interpreted diagnostic MSUS, 81.0% performed/interpreted procedural MSUS, 59.8% billed for at least 50% of diagnostic studies, and 78.8% billed for at least 50% of procedural studies. The top reasons for not doing diagnostic and procedural ultrasonography were lack of administrative support and limited time, respectively. For 25% of diagnostic ultrasonography and 12.9% of procedural ultrasonography, billing was done <50% of the time. Of the respondents, 78.0% reported that USSONAR training made them better rheumatologists.

Conclusion: Most USSONAR-trained rheumatologists are certified, practicing both diagnostic and procedural MSUS and billing for most of their work. However, a substantial number of studies are not being billed due to time constraints, limited administrative support, and legal liability. Participants agreed that USSONAR training made them better rheumatologists.

目的:关于美国风湿病学家的肌肉骨骼超声(MSUS)实践,我们所知甚少。我们试图确定北美风湿病学家超声学校(USSONAR)培训项目的学员目前使用 MSUS 的情况,进而确定风湿病学家使用 MSUS 的情况,并了解使用 MSUS 的障碍:2009年至2020年间,374名学员参加了为期8个月的USSONAR混合课程(MSUS基础和培训师培训),我们向他们发送了一份在线调查。每位受访者都有一个唯一的标识符,该标识符与其在培训期间提交的练习扫描总数和考试成绩相关联:调查回复率为 28.1%(105/374),其中 82% 为成人风湿病学家,18% 为儿童风湿病学家。71%的受访者获得了 MSUS 证书:86.7%的受访者进行和/或解释了诊断性MSUS,81.0%的受访者进行/解释了程序性MSUS,59.8%的受访者至少为50%的诊断性研究开具了账单,78.8%的受访者至少为50%的程序性研究开具了账单。不进行诊断性和程序性超声检查的首要原因分别是缺乏行政支持和时间有限。在 25% 的诊断性超声检查和 12.9% 的程序性超声检查中,计费率低于 50%。78.0%的受访者表示,USSONAR培训使他们成为了更好的风湿病医生:结论:大多数接受过 USSONAR 培训的风湿病学家都获得了认证,可以同时进行 MSUS 诊断和手术,并对其大部分工作进行收费。然而,由于时间限制、行政支持有限以及法律责任等原因,大量研究并未计费。与会者一致认为,USSONAR培训使他们成为了更好的风湿病学家。
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引用次数: 0
Oral Glucocorticoids for Skin Fibrosis in Early Diffuse Systemic Sclerosis: A Target Trial Emulation Study From the European Scleroderma Trials and Research Group Database. 口服糖皮质激素治疗早期弥漫性系统性硬化症的皮肤纤维化:来自欧洲硬皮病试验和研究小组数据库的目标试验模拟研究。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-14 DOI: 10.1002/acr.25469
Denis Mongin, Marco Matucci-Cerinic, Ulrich A Walker, Oliver Distler, Radim Becvar, Elise Siegert, Lidia P Ananyeva, Vanessa Smith, Juan Jose Alegre-Sancho, Sule Yavuz, Massimiliano Limonta, Gabriela Riemekasten, Elena Rezus, Madelon Vonk, Marie-Elise Truchetet, Francesco Del Galdo, Delphine S Courvoisier, Michele Iudici

Objective: The objective of this study is to evaluate whether adding oral glucocorticoids to immunosuppressive therapy improves skin scores and ensures safety in patients with early diffuse cutaneous systemic sclerosis (dcSSc).

Methods: We performed an emulated randomized trial comparing the changes from baseline to 12 ± 3 months of the modified Rodnan skin score (mRSS: primary outcome) in patients with early dcSSc receiving either oral glucocorticoids (≤20 mg/day prednisone equivalent) combined with immunosuppression (treated) or immunosuppression alone (controls), using data from the European Scleroderma Trials and Research Group. Secondary end points were the difference occurrence of progressive skin or lung fibrosis and scleroderma renal crisis. Matching propensity score was used to adjust for baseline imbalance between groups.

Results: We matched 208 patients (mean age 49 years; 33% male; 59% anti-Scl70), 104 in each treatment group, obtaining comparable characteristics at baseline. In the treated group, patients received a median prednisone dose of 5 mg/day. Mean mRSS change at 12 ± 3 months was similar in the two groups (decrease of 2.7 [95% confidence interval {95% CI} 1.4-4.0] in treated vs 3.1 [95% CI 1.9-4.4] in control, P = 0.64). Similar results were observed in patients with shorter disease duration (≤ 24 months) or with mRSS ≤22. There was no between-group difference for all prespecified secondary outcomes. A case of scleroderma renal crisis occurred in both groups.

Conclusion: We did not find any significant benefit of adding low-dose oral glucocorticoids to immunosuppression for skin fibrosis, and at this dosage, glucocorticoid did not increase the risk of scleroderma renal crisis.

研究目的本研究的目的是评估在免疫抑制疗法中添加口服糖皮质激素是否能改善早期弥漫性皮肤系统性硬化症(dcSSc)患者的皮肤评分并确保其安全性:我们利用欧洲硬皮病试验和研究小组(European Scleroderma Trials and Research Group)的数据,进行了一项模拟随机试验,比较了接受口服糖皮质激素(≤20 毫克/天泼尼松当量)联合免疫抑制治疗(治疗组)或单纯免疫抑制治疗(对照组)的早期弥漫性皮肤系统性硬化症(dcSSc)患者从基线到 12±3 个月的改良罗德南皮肤评分(mRSS:主要结果)的变化。次要终点是进行性皮肤或肺纤维化以及硬皮病肾危象发生率的差异。匹配倾向评分用于调整组间基线不平衡:我们匹配了 208 名患者(年龄 49 岁;33% 为男性;59% 抗 Scl70),每组 104 人,基线特征相当。在治疗组中,患者接受的泼尼松剂量中位数为 5 毫克/天。两组患者在12±3个月时的平均mRSS变化相似(治疗组下降2.7 [95% CI 1.4 - 4.0],对照组下降3.1 [95% CI 1.9 - 4.4],P = 0.64)。在病程较短(≤ 24 个月)或 mRSS ≤ 22 的患者中也观察到了类似的结果。所有预设的次要结果均无组间差异。两组均有一例硬皮病肾危象发生:我们没有发现在治疗皮肤纤维化的免疫抑制基础上加用小剂量口服糖皮质激素有任何明显的益处,而且在此剂量下,糖皮质激素不会增加硬皮病肾危象的风险。
{"title":"Oral Glucocorticoids for Skin Fibrosis in Early Diffuse Systemic Sclerosis: A Target Trial Emulation Study From the European Scleroderma Trials and Research Group Database.","authors":"Denis Mongin, Marco Matucci-Cerinic, Ulrich A Walker, Oliver Distler, Radim Becvar, Elise Siegert, Lidia P Ananyeva, Vanessa Smith, Juan Jose Alegre-Sancho, Sule Yavuz, Massimiliano Limonta, Gabriela Riemekasten, Elena Rezus, Madelon Vonk, Marie-Elise Truchetet, Francesco Del Galdo, Delphine S Courvoisier, Michele Iudici","doi":"10.1002/acr.25469","DOIUrl":"10.1002/acr.25469","url":null,"abstract":"<p><strong>Objective: </strong>The objective of this study is to evaluate whether adding oral glucocorticoids to immunosuppressive therapy improves skin scores and ensures safety in patients with early diffuse cutaneous systemic sclerosis (dcSSc).</p><p><strong>Methods: </strong>We performed an emulated randomized trial comparing the changes from baseline to 12 ± 3 months of the modified Rodnan skin score (mRSS: primary outcome) in patients with early dcSSc receiving either oral glucocorticoids (≤20 mg/day prednisone equivalent) combined with immunosuppression (treated) or immunosuppression alone (controls), using data from the European Scleroderma Trials and Research Group. Secondary end points were the difference occurrence of progressive skin or lung fibrosis and scleroderma renal crisis. Matching propensity score was used to adjust for baseline imbalance between groups.</p><p><strong>Results: </strong>We matched 208 patients (mean age 49 years; 33% male; 59% anti-Scl70), 104 in each treatment group, obtaining comparable characteristics at baseline. In the treated group, patients received a median prednisone dose of 5 mg/day. Mean mRSS change at 12 ± 3 months was similar in the two groups (decrease of 2.7 [95% confidence interval {95% CI} 1.4-4.0] in treated vs 3.1 [95% CI 1.9-4.4] in control, P = 0.64). Similar results were observed in patients with shorter disease duration (≤ 24 months) or with mRSS ≤22. There was no between-group difference for all prespecified secondary outcomes. A case of scleroderma renal crisis occurred in both groups.</p><p><strong>Conclusion: </strong>We did not find any significant benefit of adding low-dose oral glucocorticoids to immunosuppression for skin fibrosis, and at this dosage, glucocorticoid did not increase the risk of scleroderma renal crisis.</p>","PeriodicalId":8406,"journal":{"name":"Arthritis Care & Research","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142613804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Immunosuppressive Drugs in Early Systemic Sclerosis and Prevention of Damage Accrual. 早期局限性皮肤系统性硬化症的免疫抑制药物可防止全局性损伤累积
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-14 DOI: 10.1002/acr.25467
Murray Baron, Mandana Nikpour, Dylan Hansen, Susanna Proudman, Wendy Stevens, Mianbo Wang

Objective: Organ damage in patients with systemic sclerosis (SSc) in individual organs such as the lungs may be prevented by receiving immunosuppressive drugs (ISs). A new measure of global organ damage, the Scleroderma Clinical Trials Consortium Damage Index (SCTC-DI), has allowed us to investigate whether receiving ISs may reduce global organ damage accrual in patients with early SSc.

Methods: This was a retrospective study of patients with two or less years of disease duration in Canadian and Australian cohorts with SSc. Patients with either limited cutaneous SSc (lcSSc) or diffuse cutaneous SSc (dcSSc) were observed separately and divided into groups who were either ever or never exposed to ISs. The SCTC-DI was the outcome, and inverse probability of treatment weighting (IPTW) was used to balance the study groups and to fit a marginal structural generalized estimating equation model.

Results: In the cohort with lcSSc, there were 210 patients, of whom 34% were exposed to ISs at some time. Exposure to ISs was associated with lower damage scores. In the cohort with dcSSc, there were 192 patients, of whom 76% were exposed to ISs at some time. Exposure to ISs was not associated with damage scores.

Conclusion: In this retrospective observational cohort study, using IPTW to adjust for confounders, we found a protective effect of receiving ISs on damage accrual in patients with lcSSc. We were unable to determine such an effect in patients with dcSSc, but unknown confounders may have been present, and prospective studies of patients with dcSSc receiving ISs should include the SCTC-DI to determine the possible effect of ISs on damage accrual.

目的:免疫抑制剂(IS)可预防系统性硬化症(SSc)中肺部等个别器官的损伤。硬皮病临床试验联盟损伤指数(SCTC-DI)是衡量全身器官损伤的一种新方法,它使我们能够研究免疫抑制剂是否能减少早期系统性硬化症的全身器官损伤:这是一项回顾性研究,研究对象是加拿大和澳大利亚SSc队列中病程小于2年的患者。分别观察了局限性皮肤型(lcSSc)或弥漫性皮肤型(dcSSc)SSc 患者,并将其分为曾经接触过 IS 组和从未接触过 IS 组。研究结果以SCTC-DI为指标,并采用逆概率治疗加权法(IPTW)来平衡各研究组,并拟合边际结构广义估计方程(GEE)模型:在lcSSc队列中,共有210名受试者,其中34%的受试者曾经接触过IS。暴露于 IS 与较低的损伤评分有关。在 dcSSc 队列中,有 192 名受试者,其中 76% 的人曾在一段时间内接触过 IS。暴露于 IS 与损伤评分无关:在这项回顾性观察队列研究中,我们使用 IPTW 对混杂因素进行了调整,发现在 lcSSc 中使用 IS 对损伤累积有保护作用。我们无法确定在 dcSSc 中是否存在这种效应,但可能存在未知的混杂因素,因此对 dcSSc 中 IS 的前瞻性研究应包括 SCTC-DI,以确定 IS 对损伤累积的可能效应。
{"title":"Immunosuppressive Drugs in Early Systemic Sclerosis and Prevention of Damage Accrual.","authors":"Murray Baron, Mandana Nikpour, Dylan Hansen, Susanna Proudman, Wendy Stevens, Mianbo Wang","doi":"10.1002/acr.25467","DOIUrl":"10.1002/acr.25467","url":null,"abstract":"<p><strong>Objective: </strong>Organ damage in patients with systemic sclerosis (SSc) in individual organs such as the lungs may be prevented by receiving immunosuppressive drugs (ISs). A new measure of global organ damage, the Scleroderma Clinical Trials Consortium Damage Index (SCTC-DI), has allowed us to investigate whether receiving ISs may reduce global organ damage accrual in patients with early SSc.</p><p><strong>Methods: </strong>This was a retrospective study of patients with two or less years of disease duration in Canadian and Australian cohorts with SSc. Patients with either limited cutaneous SSc (lcSSc) or diffuse cutaneous SSc (dcSSc) were observed separately and divided into groups who were either ever or never exposed to ISs. The SCTC-DI was the outcome, and inverse probability of treatment weighting (IPTW) was used to balance the study groups and to fit a marginal structural generalized estimating equation model.</p><p><strong>Results: </strong>In the cohort with lcSSc, there were 210 patients, of whom 34% were exposed to ISs at some time. Exposure to ISs was associated with lower damage scores. In the cohort with dcSSc, there were 192 patients, of whom 76% were exposed to ISs at some time. Exposure to ISs was not associated with damage scores.</p><p><strong>Conclusion: </strong>In this retrospective observational cohort study, using IPTW to adjust for confounders, we found a protective effect of receiving ISs on damage accrual in patients with lcSSc. We were unable to determine such an effect in patients with dcSSc, but unknown confounders may have been present, and prospective studies of patients with dcSSc receiving ISs should include the SCTC-DI to determine the possible effect of ISs on damage accrual.</p>","PeriodicalId":8406,"journal":{"name":"Arthritis Care & Research","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142613784","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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. 多关节病程幼年特发性关节炎儿科患者服用乌达帕替尼的药代动力学、疗效和安全性:一项开放标签 1 期试验的中期分析。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-14 DOI: 10.1002/acr.25465
Hermine I Brunner, Anna Shmagel, Gerd Horneff, Ivan Foeldvari, Jordi Antón, Athimalaipet V Ramanan, Yuli Qian, Kristina Unnebrink, Shuai Hao, Heidi S Camp, Nasser Khan, Wei Liu, Mohamed-Eslam F Mohamed

Objective: 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).

Methods: In an open-label, phase 1 study (SELECT-YOUTH), enrolled patients, aged 2 to <18 years with pcJIA, received body weight-based upadacitinib doses using a twice-daily oral solution or once-daily 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.

Results: A total of 57 patients received upadacitinib. The median time to maximum upadacitinib concentration was approximately three hours and one hour for the tablet and oral solution regimens, respectively; the harmonic mean functional half-life was approximately five hours and two hours, respectively. Juvenile idiopathic arthritis American College of Rheumatology 30, 50, 70, 90, and 100 responses at week 12 were 91.8%, 89.8%, 69.4%, 49.0%, and 32.7%, respectively. Efficacy was generally maintained through week 48, and improvement in additional efficacy end points 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.

Conclusion: This interim analysis demonstrates that the bodyweight-based dosing regimen of upadacitinib was well tolerated and efficacious in pediatric patients with pcJIA.

研究目的本研究旨在评估口服选择性 JAK 抑制剂达帕替尼(upadacitinib)在多关节病程幼年特发性关节炎(pcJIA)儿童患者中的药代动力学、疗效和安全性:在一项开放标签的一期研究(SELECT-YOUTH)中,入组患者的年龄为2至6岁:共有57名患者接受了达达替尼治疗。片剂和口服溶液剂达到最高达帕他替尼浓度的中位时间分别约为3小时和1小时;调和平均功能半衰期分别约为5小时和2小时。第12周时,青少年特发性关节炎(JIA)美国风湿病学会(ACR)30/50/70/90/100应答率分别为91.8%/89.8%/69.4%/49.0%/32.7%。疗效普遍维持到第48周,其他疗效终点也有所改善。中位暴露持续时间为 412 天,57 例患者中有 52 例报告了不良事件,其中 6 例出现严重不良事件。不良事件的严重程度以轻度至中度为主,符合已知的奥达替尼安全性特征:这项中期分析表明,基于体重的达达替尼给药方案在儿童pcJIA患者中具有良好的耐受性和疗效。
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引用次数: 0
National Institute of Health and Care Excellence Clinical Criteria for the Diagnosis of Knee Osteoarthritis: A Prospective Diagnostic Accuracy Study in Individuals with Type 2 Diabetes. 国家健康与护理卓越研究所诊断膝骨关节炎的临床标准:2型糖尿病患者前瞻性诊断准确性研究》。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-14 DOI: 10.1002/acr.25464
Lauren K King, Ian Stanaitis, Vivian Hung, Sahil Koppikar, Esther J Waugh, Lorraine Lipscombe, Gillian A Hawker

Objective: The National Institute of Health and Care Excellence (NICE) criteria for osteoarthritis (OA) obviate the need for physical exam or imaging, and their use may improve timely diagnosis of OA. However, they have not been validated.

Methods: Within a larger study of individuals with type 2 diabetes, participants with and without self-reported knee pain underwent assessment of the NICE criteria for knee OA by questionnaire (index test), and clinical evaluation for established or possible knee OA by a rheumatologist (reference standard). We calculated the sensitivity, specificity, likelihood ratio positive (LR+) and likelihood ratio negative (LR-) of the NICE criteria and modified NICE criteria without the stiffness criterion.

Results: Our study included 96 participants: mean age 65.4 (SD 8.3) years and 52% were women. Individuals who fulfilled NICE criteria for knee OA (55.2%) included a spectrum of pain severity on a 11-point pain numeric rating scale with a median score of 5 (range: 1-9). Rheumatologist assessment identified 56 (58.3%) participants with symptomatic knee OA. The sensitivity, specificity, LR+, and LR- of NICE criteria for symptomatic knee OA were 0.84 (95% CI 0.74, 0.94), 0.85 (95% CI 0.74, 0.96), 5.6 and 0.19, respectively. For modified NICE criteria, these were 0.89 (95% CI 0.82, 0.97), 0.85 (95% CI 0.74, 0.96), 5.93 and 0.13.

Conclusion: The NICE criteria have high sensitivity and specificity for detecting symptomatic knee OA in a population with type 2 diabetes. We found that a modified version, omitting the stiffness criterion, performed similarly. These criteria should be validated in other settings and populations.

目的:美国国家健康与护理优化研究所(NICE)的骨关节炎(OA)标准无需进行体格检查或影像学检查,使用这些标准可提高对 OA 的及时诊断率。然而,这些标准尚未得到验证:在一项针对 2 型糖尿病患者的大型研究中,有膝关节疼痛和没有自述膝关节疼痛的参与者都接受了 NICE 膝关节 OA 标准的问卷评估(指标测试),并接受了风湿免疫科医生对已确诊或可能确诊的膝关节 OA 的临床评估(参考标准)。我们计算了 NICE 标准和不含僵硬度标准的修改版 NICE 标准的敏感性、特异性、似然比阳性(LR+)和似然比阴性(LR-):我们的研究包括 96 名参与者:平均年龄为 65.4 岁(标准差为 8.3 岁),52% 为女性。符合 NICE 标准的膝关节 OA 患者(55.2%)包括 11 点疼痛数字评分表中的疼痛严重程度,中位数为 5 分(范围:1-9)。风湿免疫科医生的评估确定了 56 人(58.3%)为有症状的膝关节 OA 患者。NICE标准对无症状膝关节OA的敏感性、特异性、LR+和LR-分别为0.84(95% CI 0.74,0.94)、0.85(95% CI 0.74,0.96)、5.6和0.19。修改后的 NICE 标准分别为 0.89(95% CI 0.82,0.97)、0.85(95% CI 0.74,0.96)、5.93 和 0.13:NICE 标准在检测 2 型糖尿病患者的无症状膝关节 OA 方面具有较高的灵敏度和特异性。我们发现,省略了僵硬度标准的修改版也有类似的效果。这些标准应在其他环境和人群中进行验证。
{"title":"National Institute of Health and Care Excellence Clinical Criteria for the Diagnosis of Knee Osteoarthritis: A Prospective Diagnostic Accuracy Study in Individuals with Type 2 Diabetes.","authors":"Lauren K King, Ian Stanaitis, Vivian Hung, Sahil Koppikar, Esther J Waugh, Lorraine Lipscombe, Gillian A Hawker","doi":"10.1002/acr.25464","DOIUrl":"https://doi.org/10.1002/acr.25464","url":null,"abstract":"<p><strong>Objective: </strong>The National Institute of Health and Care Excellence (NICE) criteria for osteoarthritis (OA) obviate the need for physical exam or imaging, and their use may improve timely diagnosis of OA. However, they have not been validated.</p><p><strong>Methods: </strong>Within a larger study of individuals with type 2 diabetes, participants with and without self-reported knee pain underwent assessment of the NICE criteria for knee OA by questionnaire (index test), and clinical evaluation for established or possible knee OA by a rheumatologist (reference standard). We calculated the sensitivity, specificity, likelihood ratio positive (LR+) and likelihood ratio negative (LR-) of the NICE criteria and modified NICE criteria without the stiffness criterion.</p><p><strong>Results: </strong>Our study included 96 participants: mean age 65.4 (SD 8.3) years and 52% were women. Individuals who fulfilled NICE criteria for knee OA (55.2%) included a spectrum of pain severity on a 11-point pain numeric rating scale with a median score of 5 (range: 1-9). Rheumatologist assessment identified 56 (58.3%) participants with symptomatic knee OA. The sensitivity, specificity, LR+, and LR- of NICE criteria for symptomatic knee OA were 0.84 (95% CI 0.74, 0.94), 0.85 (95% CI 0.74, 0.96), 5.6 and 0.19, respectively. For modified NICE criteria, these were 0.89 (95% CI 0.82, 0.97), 0.85 (95% CI 0.74, 0.96), 5.93 and 0.13.</p><p><strong>Conclusion: </strong>The NICE criteria have high sensitivity and specificity for detecting symptomatic knee OA in a population with type 2 diabetes. We found that a modified version, omitting the stiffness criterion, performed similarly. These criteria should be validated in other settings and populations.</p>","PeriodicalId":8406,"journal":{"name":"Arthritis Care & Research","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142613792","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Incidence of and Risk of Mortality After Hip Fractures in Rheumatoid Arthritis Relative to the General Population. 与普通人群相比,类风湿关节炎患者髋部骨折的发生率和死亡风险。
IF 3.7 2区 医学 Q1 RHEUMATOLOGY Pub Date : 2024-11-14 DOI: 10.1002/acr.25466
C Allyson Jones, Pierre Guy, Hui Xie, Eric C Sayre, Kai Zhao, Diane Lacaille

Objective: Osteoporosis, a known complication of rheumatoid arthritis (RA), increases the risk of hip fracture, which is associated with high morbidity and mortality. Fracture risk estimates in patients with RA treated with contemporary treatment strategies are lacking. The objectives were (1) estimate age-specific and sex-specific incidence rates and compare the risk of hip fractures in RA relative to age-matched and sex-matched general population controls, and (2) compare the risk of all-cause mortality in RA and general population controls after hip fracture.

Methods: A longitudinal study of a population-based incident cohort of patients with RA diagnosed between 1997 and 2009, followed until 2014, with age-matched and sex-matched controls from the general population of British Columbia, using administrative health data. Hip fracture outcomes (International Classification of Diseases, Ninth Edition, Clinical Modification [ICD-9-CM] codes 820.0 or 820.2; ICD-10-Canada code S72.0 to S72.2) and mortality at predefined intervals after fracture (in hospital, 90 days, 1-year, 5-year) were identified. Hip fracture incidence rates for RA and controls, and incidence rate ratios (IRRs), were calculated. Cox proportional hazards models compared hip fracture and mortality risk in RA versus controls; logistic regression compared in-hospital mortality risk.

Results: Overall, 1,314 hip fractures over 360,521 person-years were identified in 37,616 individuals with RA and 2,083 over 732,249 person-years in 75,213 controls, yielding a 28% greater fracture risk in RA (IRR 1.28 [95% confidence interval 1.20-1.37]). Mean age at time of fracture was slightly younger for RA than controls (79.6 ± 10.8 vs 81.6 ± 9.3 years). Postfracture mortality risk at one-year and five-years did not differ between RA and general population controls. Results were similar in a sensitivity analysis including only individuals with RA who received disease-modifying antirheumatic drugs.

Conclusion: People with RA had a greater risk of hip fractures, but no greater risk of mortality post fracture, than the general population. The relative risk of hip fractures observed was not as high as previously reported, likely reflecting better treatment of inflammation and management of osteoporosis and its risk factors.

目的:骨质疏松症是类风湿性关节炎(RA)的一种已知并发症,会增加髋部骨折的风险,而髋部骨折与高发病率和高死亡率相关。目前还缺乏对接受现代治疗策略的 RA 患者骨折风险的估计。研究目的是:1)估算年龄和性别特异性发病率,并比较 RA 患者与年龄和性别匹配的普通人群对照组的髋部骨折风险;2)比较 RA 患者和普通人群对照组在髋部骨折后的全因死亡风险:方法:利用健康管理数据,对 1997 年至 2009 年间确诊的 RA 患者与年龄和性别匹配的不列颠哥伦比亚省普通人群对照组进行纵向研究。该研究确定了髋部骨折的结果(ICD9-CM代码820.0或820.2;ICD10-CA代码S72.0至S72.2)以及骨折后预定时间间隔(院内、90天、1年、5年)内的死亡率。计算了 RA 和对照组的髋部骨折发病率和发病率比 (IRR)。Cox比例危险模型比较了RA与对照组的髋部骨折和死亡风险;逻辑回归比较了院内死亡风险:总体而言,37,616 名 RA 患者在 360,521 人年中发生了 1314 次髋部骨折,75,213 名对照者在 732,249 人年中发生了 2083 次髋部骨折,RA 患者的骨折风险比对照者高出 28%(IRR 为 1.28 [95%CI, 1.20;1.37])。RA患者骨折时的平均年龄略小于对照组(79.6 + 10.8 岁 vs. 81.6 + 9.3 岁)。RA和普通人群对照组骨折后1年和5年的死亡风险没有差异。在一项敏感性分析中,仅包括接受改变病情抗风湿药(DMARDs)治疗的RA患者,结果与上述分析相似:结论:与普通人群相比,RA患者发生髋部骨折的风险更高,但骨折后死亡的风险并不比普通人群高。观察到的髋部骨折相对风险并没有之前报道的那么高,这可能反映了炎症治疗和骨质疏松症及其风险因素管理的改善。
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Arthritis Care & Research
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