pruritus

瘙痒
  • 文章类型: Journal Article
    很少有研究检查老年患者的瘙痒,一种常见的皮肤病。该研究检查了皮肤科老年患者的瘙痒患病率和特征,在热那亚的Galliera医院.检查了所有有任何皮肤状况的门诊患者的人口统计学特征,专注于65岁以上的瘙痒患者。瘙痒存在于36/262患者中(14%;M:F=20:16;平均年龄:59.55岁)。140名年龄≥65岁的患者中约有14%有瘙痒,20/262(8%;M:F=14:6;平均年龄:74.6岁)表现出来。在≥65岁(20/36)和<65岁(16/36)的患者之间,皮肤瘙痒的视觉模拟评分没有统计学差异。在89%的患者中,瘙痒与皮肤病有关,主要是牛皮癣。在年龄>65岁的患者中,仅皮肤外疾病更为常见。在这些患者中,药物使用和瘙痒之间没有发现记忆联系。我们确认瘙痒是影响两性的常见皮肤问题,年轻和年老,并且几乎总是由潜在的皮肤状况(主要是牛皮癣)引起的。它很少是由新药引起的。
    Few studies have examined pruritus in elderly patients, a common dermatological condition. The study examines pruritus prevalence and characteristics in elderly patients referred to the Dermatology Unit, at Genoa\'s Galliera Hospital. The demographic characteristics of all Outpatient Clinic patients with any skin condition were examined, focusing on pruritus patients over 65. Pruritus was present in 36/262 patients (14%; M:F =20:16; mean age: 59.55 years). About 14% of 140 patients aged ≥65 years had pruritus, with 20/262 (8%; M:F =14:6; mean age: 74.6 years) exhibiting it. Visual analog score pruritus did not differ between patients aged ≥65 years (20/36) and <65 years (16/36) statistically. In 89% of patients, itch was related to a dermatological condition, mainly psoriasis. Only extracutaneous diseases resulted more frequently in the patients aged >65. No anamnestic link was found between drug use and pruritus in these patients. We confirm that pruritus is a common skin problem that affects both sexes, young and old, and is almost always caused by an underlying skin condition (mainly psoriasis). It is rarely caused by a new drug.
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  • 文章类型: Journal Article
    目的:结节性痒疹(PN)是一种皮肤疾病,其特征是皮肤结节严重发痒,与重要的医疗保健资源利用(HCRU)有关。这项研究旨在评估英格兰PN总体和中度至重度PN(MSPN)患者的HCRU。
    方法:这项回顾性队列研究使用了来自英国临床实践研究数据链和医院事件统计的数据。在主要分析中,将轻度PN(MiPN)患者与MSPN患者的年龄和性别进行匹配。患者在2007年4月1日至2019年3月1日期间纳入研究。计算了全因HCCU,包括初级和二级保健接触者和费用(成本年2022)。
    结果:在23,522名确定的患者中,8,933符合纳入标准,与2,479名PN患者的主要匹配队列。随访期间,MSPN组和MiPN组的匹配队列初级护理访视次数分别为21.27/患者年(PPY)和11.35PPY.MSPN和MiPN组的任何门诊量为10.72PPY和4.87PPY,分别。MSPN和MiPN组的门诊皮肤科访视为1.96PPY和1.14PPY,分别。
    结论:PN,尤其是MSPN,在英国有很高的HCCU负担,强调需要新的和改进的疾病管理治疗。
    Purpose: Prurigo nodularis (PN) is a skin disease characterized by intensely itchy skin nodules and is associated with a significant healthcare resource utilization (HCRU). This study aimed to estimate the HCRU of patients in England with PN overall and moderate-to-severe PN (MSPN) in particular.
    Methods: This retrospective cohort study used data from the Clinical Practice Research Datalink and Hospital Episode Statistics in England. Patients with Mild PN (MiPN) were matched to patients with MSPN by age and gender for the primary analysis. Patients were enrolled in the study between 1st April 2007 and 1st March 2019. All-cause HCRU was calculated, including primary and secondary care contacts and costs (cost-year 2022).
    Results: Of 23,522 identified patients, 8,933 met the inclusion criteria, with a primary matched cohort of 2,479 PN patients. During follow up, the matched cohort\'s primary care visits were 21.27 per patient year (PPY) for MSPN group and 11.35 PPY for MiPN group. Any outpatient visits were 10.72 PPY and 4.87 PPY in MSPN and MiPN groups, respectively. Outpatient dermatology visits were 1.96 PPY and 1.14 PPY in MSPN and MiPN groups, respectively.
    Conclusion: PN, especially MSPN, has a high HCRU burden in England, highlighting the need for new and improved disease management treatments.
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  • 文章类型: Journal Article
    背景:胆汁淤积性瘙痒和疲劳是与原发性胆汁性胆管炎(PBC)相关的衰弱病症,可显著影响患者的生活质量。PBC中的瘙痒经常在晚上恶化,患者经常报告睡眠障碍,这有助于认知症状和疲劳。Linerixibat是临床开发中用于治疗与PBC相关的瘙痒的回肠胆汁酸转运蛋白抑制剂,最近在2b期GLIMMER试验中与安慰剂进行了评估。该事后分析评估了与治疗组无关的GLIMMER参与者的瘙痒严重程度与睡眠障碍之间的关系。
    方法:GLIMMER(NCT02966834),一个多中心,双盲,随机化,安慰剂对照试验,招募了147例PBC和中度至重度瘙痒患者。经过4周的单盲安慰剂,患者(随机3:1)接受linerixibat或安慰剂治疗12周(至第16周).参与者使用0-10数字评分量表(NRS)在电子日记中对瘙痒(每天两次)及其对睡眠的干扰(每天一次)进行评分。每周和每月瘙痒评分被计算为各自时间段内最差的每日瘙痒评分的平均值。在研究访问中,参与者完成了5-D瘙痒量表和PBC-40生活质量问卷,两者都包含与瘙痒相关的睡眠障碍特定的项目。通过NRS变化之间的相关性,事后评估瘙痒对睡眠的影响。5-D痒,PBC-40
    结果:在治疗结束时(第16周),每周瘙痒的基线变化与睡眠NRS评分(r=0.88[95%置信区间(CI):0.83;0.91])之间存在很强的相关性,以及每月瘙痒和睡眠NRS评分(r=0.84[95%CI:0.80;0.87])。每周瘙痒评分严重程度类别改善的患者平均表现出减少的睡眠干扰。瘙痒反应者(每周瘙痒评分与基线相比改善≥2点)显示每周睡眠NRS评分有较大改善,5-D痒,和PBC-40睡眠项目,比痒无反应者(<2点改善)。
    结论:PBC患者瘙痒严重程度的变化与睡眠干扰之间存在很强的相关性;瘙痒减轻可同时改善睡眠。
    BACKGROUND: Cholestatic pruritus and fatigue are debilitating conditions associated with primary biliary cholangitis (PBC) and can significantly impact patients\' quality of life. Pruritus in PBC often worsens at night and patients frequently report sleep disturbance, which contributes to cognitive symptoms and fatigue. Linerixibat is an ileal bile acid transporter inhibitor in clinical development for the treatment of pruritus associated with PBC and was recently assessed versus placebo in the Phase 2b GLIMMER trial. This post-hoc analysis assesses the relationship between pruritus severity and sleep disturbance in participants of GLIMMER regardless of treatment group.
    METHODS: GLIMMER (NCT02966834), a multicenter, double-blind, randomized, placebo-controlled trial, recruited 147 patients with PBC and moderate-to-severe pruritus. Following 4 weeks single-blind placebo, patients (randomized 3:1) received linerixibat or placebo for 12 weeks (to Week 16). Participants graded their itch (twice daily) and its interference with sleep (once daily) in an electronic diary using a 0-10 numerical rating scale (NRS). Weekly and monthly itch scores were calculated as the mean of the worst daily itch score over the respective time period. At study visits, participants completed the 5-D itch scale and the PBC-40 quality of life questionnaire, both of which contain an item specific to itch-related sleep disturbance. The impact of pruritus on sleep was assessed post hoc through correlations between the changes in NRS, 5-D itch, and PBC-40.
    RESULTS: Strong correlations were found between change from baseline in weekly itch and sleep NRS scores (r = 0.88 [95% confidence interval (CI): 0.83; 0.91]) at the end of treatment (Week 16), as well as in monthly itch and sleep NRS scores (r = 0.84 [95% CI: 0.80; 0.87]). Patients with improved weekly pruritus score severity category demonstrated reduced perceived sleep interference on average. Itch responders (≥2-point improvement in weekly itch score from baseline) displayed larger improvements in weekly sleep NRS score, 5-D itch, and PBC-40 sleep items, than itch non-responders (<2-point improvement).
    CONCLUSIONS: A strong correlation exists between changes in pruritus severity and sleep interference in patients with PBC; pruritus reduction could generate concomitant improvement in sleep.
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  • 文章类型: Journal Article
    感觉异常疼痛(NP)是一种慢性疾病,其特征是在肩胛骨下背部单侧出现瘙痒和其他令人不快的感觉障碍。其具体的潜在机制在很大程度上是未知的,虽然假设是神经病。确定可能的体感因素可以为新的治疗方法铺平道路。
    考虑到非瘙痒机制在NP中的潜在参与,我们的目标是使用在无痛对照和疼痛性神经性疾病中标准化的方法,广泛表征NP感染和未受影响的皮肤的体感功能.我们假设,如果NP是由不直接针对皮肤瘙痒感受器的神经病理机制引起的,体感异常不会发痒。第二,鉴于背部对侧没有症状,我们假设这个地区通常是敏感的。
    在这项研究中,定量感觉测试(QST)用于综合评估15例成年NP患者的体感功能。在NP受影响的区域进行标准化的QST指标,并使用年龄与对侧无症状皮肤和无痒个体进行比较,性别,和站点匹配的参考数据集。
    有症状和无症状的皮肤之间的敏感性没有显着差异,除了瘙痒一侧的机械诱发瘙痒增加。然而,参考数据集比较显示,NP患者对无害感冒和有害针刺的双侧敏感性低,疼痛的时间总和更高。此外,与参考数据相比,NP患者对寒冷和针刺的敏感性降低,存在矛盾的热感觉,增加了疼痛的结束。
    这些结果表明Aδ纤维途径和中枢敏化在NP相关瘙痒中起作用。需要更多的研究来确定感觉差异是否超出了受NP影响的皮肤瘤水平,以及什么可能导致特定靶向Aδ纤维的神经病变。
    UNASSIGNED: Notalgia paresthetica (NP) is a chronic condition characterized by pruritus and other unpleasant dysesthetic sensations unilaterally on the subscapular back. Its specific underlying mechanisms are largely unknown, though hypothesized to be neuropathic. Determination of possible somatosensory contributors to the condition could pave the way for novel treatments.
    UNASSIGNED: Given the potential involvement of non-pruritic mechanisms in NP, our objective was to broadly characterize the somatosensory function in NP-affected and unaffected skin using methods that have been standardized in pain-free controls and painful neuropathic disorders. We hypothesized that if NP is caused by neuropathic mechanisms not targeted directly to pruritoceptors in the skin, somatosensory abnormalities would not be itchspecific. Second, given the lack of symptoms on the contralateral side of the back, we hypothesized that this region would be normally sensitive.
    UNASSIGNED: In this study, quantitative sensory testing (QST) was used to comprehensively assess the somatosensory function in 15 adult patients with NP. Standardized QST metrics were performed in the NP-affected region and compared with the contralateral asymptomatic skin and itch-free individuals using an age, gender, and site-matched reference data set.
    UNASSIGNED: There were no significant differences in sensitivity between symptomatic and asymptomatic skin, except for increased mechanical-evoked itch on the itchy side. However, reference data set comparisons revealed bilateral hyposensitivity to innocuous cold and noxious pinprick and higher temporal summation of pain in patients with NP. In addition, compared with reference data, patients with NP demonstrated decreased sensitivity to cold and pinprick, presence of paradoxical heat sensations, and increased wind-up of pain.
    UNASSIGNED: These results suggest a role for Aδ fiber pathways and central sensitization in NP-associated itch. More research is needed to determine whether sensory differences extend beyond the NP-affected dermatomal level and what might cause neuropathy specifically targeting Aδ fibers.
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  • 文章类型: Journal Article
    基于与肥大细胞活性和神经源性炎症相关的潜在共同病理生理学,可能将瘙痒和慢性咳嗽(CC)联系起来,这项研究,利用我们所有人的数据库,检查这两个条件之间的关联。
    使用嵌套的病例对照比较来检查关联,识别SNOMED代码为418363000(瘙痒)和68154008(CC)的病例。按年龄按1:4的比例进行匹配,性别,和种族在R中使用MatchIt包,然后用最大似然法从2x2列联表中估计比值比(ORs)和95%置信区间。
    CC患者(n=2,388)被诊断为瘙痒(OR:2.65)的可能性是2倍以上,瘙痒患者(n=22,496)被诊断为CC(OR:2.57)的可能性是2倍以上。比各自匹配的控件。
    这些结果突出了CC和瘙痒之间的潜在双向关系,提示可能共有的免疫和神经通路。像difikefalin和纳布啡这样调节这些途径的治疗方法,与P2X3靶向剂一起,鉴于可能相互关联的病理生理学,正在成为瘙痒和慢性咳嗽的潜在治疗方法。本研究对瘙痒和CC之间关联的见解可能为解决其共同机制的靶向治疗策略铺平道路。
    UNASSIGNED: Based on a potential shared pathophysiology tied to mast cell activity and neurogenic inflammation that may link pruritus and chronic cough (CC), this study, leveraging the All of Us database, examines the association between the two conditions.
    UNASSIGNED: A nested case-control comparison was used to examine the association, identifying cases with SNOMED codes 418363000 (pruritus) and 68154008 (CC). Matching was performed on a 1:4 ratio by age, sex, and ethnicity using the MatchIt package in R, followed by maximum likelihood method to estimate odds ratios (ORs) and 95% confidence intervals from 2x2 contingency tables.
    UNASSIGNED: CC patients (n = 2,388) were more than twice as likely to be diagnosed with pruritus (OR: 2.65) and pruritus patients (n = 22,496) were more than twice as likely to be diagnosed with CC (OR: 2.57), than respective matched controls.
    UNASSIGNED: These results highlight the potential bidirectional relationship between CC and pruritus, suggesting possible shared immune and neural pathways. Treatments like difelikefalin and nalbuphine that modulate these pathways, alongside P2X3 targeting agents, are emerging as potential therapeutic approaches for itch and chronic cough given the possible interconnected pathophysiology. This study\'s insights into the associations between pruritus and CC may pave the way for targeted therapeutic strategies that address their shared mechanisms.
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  • 文章类型: Journal Article
    目的:描述澳大利亚和新西兰(ANZ)中重度特应性皮炎(AD)患者的疾病负担,并与其他地理区域进行比较。
    方法:这个多中心,横截面,观察性研究(MEASURE-AD)招募了28个国家/地区连续参加皮肤科诊所的青少年和成人患者。收集的数据包括瘙痒的得分,疾病严重程度,睡眠,疼痛,疾病控制,工作和生活质量。
    结果:该研究包括2019年12月至2020年12月的112名ANZ参与者(澳大利亚n=72;新西兰n=40)。治疗包括局部用药(85.7%的患者),非生物系统治疗(28.6%),光疗(9.8%)和dupilumab(4.5%)。平均湿疹面积和严重程度指数(EASI)评分为22.3(95%CI19.6-25.0),以患者为中心的湿疹测量(POEM)评分为18.4(95%CI16.8-20.0)。瘙痒数字评定量表(NRS)为6.0(95%CI5.5-6.6)(50%患有严重瘙痒),皮肤病生活质量指数(DLQI)为14.3(95%CI12.8-15.8)。ADerm-Impact睡眠领域得分为15.1(95%CI13.2-16.9)。ADerm-症状量表最差皮肤疼痛领域评分为5.0(95%CI4.3-5.6)。工作生产力和活动减值(WPAI)百分比表示工作和生产力减值。41%的AD自我报告未得到充分控制,过去6个月有9.7次耀斑。瘙痒的分数,疾病严重程度,睡眠,疼痛,澳新银行的疾病控制和生活质量通常是所研究的所有地理区域中最高的。
    结论:ANZ型AD患者具有较高的疾病负担,延伸到日常生活的多个方面。许多人在现有治疗中没有得到充分控制。
    OBJECTIVE: To describe disease burden in individuals with moderate-to-severe atopic dermatitis (AD) in Australia and New Zealand (ANZ) and compare it with other geographic regions.
    METHODS: This multicentre, cross-sectional, observational study (MEASURE-AD) recruited consecutive adolescent and adult patients attending dermatology clinics in 28 countries. Data collected included scores of pruritus, disease severity, sleep, pain, disease control, work and quality of life.
    RESULTS: This study included 112 ANZ participants (Australia n = 72; New Zealand n = 40) from December 2019 to December 2020. Treatments included topicals (85.7% of patients), non-biologic systemic therapy (28.6%), phototherapy (9.8%) and dupilumab (4.5%). Mean Eczema Area and Severity Index (EASI) score was 22.3 (95% CI 19.6-25.0) and Patient-Oriented Eczema Measurement (POEM) score was 18.4 (95% CI 16.8-20.0). Pruritus Numerical Rating Scale (NRS) was 6.0 (95% CI 5.5-6.6) (50% had severe pruritus) and Dermatology Life Quality Index (DLQI) 14.3 (95% CI 12.8-15.8). ADerm-Impact sleep domain score was 15.1 (95% CI 13.2-16.9). ADerm-Symptom Scale worst skin pain domain score was 5.0 (95% CI 4.3-5.6). Work Productivity and Activity Impairment (WPAI) percentages indicated work and productivity impairment. Inadequately controlled AD was self-reported by 41%, with 9.7 flares in the past 6 months. Scores of pruritus, disease severity, sleep, pain, disease control and quality of life in ANZ were often the highest of all the geographic regions studied.
    CONCLUSIONS: ANZ patients with AD have a high disease burden, which extends across multiple facets of daily life. Many are inadequately controlled with existing therapies.
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  • 文章类型: Journal Article
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  • 文章类型: Journal Article
    背景:进行性家族性肝内胆汁淤积(PFIC)是一组常染色体隐性遗传疾病,最普遍的是BSEP缺乏症,导致胆汁形成中断,胆汁淤积,还有瘙痒.在先前的第二阶段研究的基础上,我们旨在评估回肠胆汁酸转运体抑制剂maralixibat在所有类型PFIC参与者中的疗效和安全性.
    方法:MARCH-PFIC是多中心,随机化,双盲,安慰剂对照,在欧洲16个国家的29个社区和医院中心进行的第3阶段研究,美洲,和亚洲。我们招募了年龄为1-17岁的PFIC持续瘙痒的参与者(>6个月;在过去4周的筛查中,早晨瘙痒报告的结果[观察者;ItchRO(Obs)]平均≥1·5)和进行性肝病的生化异常或病理证据,或者两者兼而有之。我们定义了三个分析队列。BSEP(或主要)队列仅包括双等位基因,非截短的BSEP缺乏症,没有低或波动的血清胆汁酸或以前的胆道手术。全PFIC队列将BSEP队列与患有双等位基因FIC1,MDR3,TJP2或MYO5B缺陷的参与者相结合,没有先前手术但不考虑胆汁酸。整个队列没有排除。参与者被随机分配(1:1)接受口服maralixibat(起始剂量142·5μg/kg,然后升级到570μg/kg)或安慰剂,每天两次,持续26周。主要终点是BSEP队列中基线和15-26周之间的平均早晨ItchRO(Obs)严重程度评分的平均变化。关键次要疗效终点是BSEP队列中基线与第18、22和26周平均值之间总血清胆汁酸的平均变化。在意向治疗人群(所有随机分配的人群)中进行疗效分析,并在接受至少一剂研究药物的所有参与者中进行安全性分析。这项已完成的试验已在ClinicalTrials.gov注册,NCT03905330和EudraCT,2019-001211-22。
    结果:在2019年7月9日至2022年3月4日之间,对125名患者进行了筛查,其中93人被随机分配到maralixibat(n=47;BSEP队列14人,全PFIC队列33人)或安慰剂(n=46;BSEP队列17人,全PFIC队列31人),至少接受了一剂研究药物,并纳入意向治疗和安全性人群.中位年龄为3·0岁(IQR2·0-7·0),93名参与者中有51名(55%)为女性,42名(45%)为男性。在BSEP队列中,maralixibat早晨ItchRO(Obs)从基线的最小二乘平均变化为-1·7(95%CI-2·3至-1·2),安慰剂为-0·6(-1至-0·1),组间差异为-1·1(95%CI-1·8至-0·3;p=0·0063)。血清总胆汁酸从基线的最小二乘平均变化为-176μmol/L(95%CI-257至-94),安慰剂为11μmol/L(-58至80),也代表-187μmol/L的显着差异(95%CI-293至-80;p=0·0013)。最常见的不良事件是腹泻(服用maralixibat的47例患者中有27[57%],服用安慰剂的46例患者中有9例[20%];均为轻度或中度,且大部分为短暂性)。maralixibat组中有5名(11%)参与者出现严重的因治疗引起的不良事件,而安慰剂组中有3名(7%)。无治疗相关死亡发生。
    结论:Maralixibat改善了PFIC中瘙痒和天然肝脏存活的预测因子(例如,血清胆汁酸)。Maralixibat代表非手术,药物选择中断肝肠循环和改善PFIC患者的护理标准。
    背景:Mirum制药。
    BACKGROUND: Progressive familial intrahepatic cholestasis (PFIC) is a group of autosomal recessive disorders, the most prevalent being BSEP deficiency, resulting in disrupted bile formation, cholestasis, and pruritus. Building on a previous phase 2 study, we aimed to evaluate the efficacy and safety of maralixibat-an ileal bile acid transporter inhibitor-in participants with all types of PFIC.
    METHODS: MARCH-PFIC was a multicentre, randomised, double-blind, placebo-controlled, phase 3 study conducted in 29 community and hospital centres across 16 countries in Europe, the Americas, and Asia. We recruited participants aged 1-17 years with PFIC with persistent pruritus (>6 months; average of ≥1·5 on morning Itch-Reported Outcome [Observer; ItchRO(Obs)] during the last 4 weeks of screening) and biochemical abnormalities or pathological evidence of progressive liver disease, or both. We defined three analysis cohorts. The BSEP (or primary) cohort included only those with biallelic, non-truncated BSEP deficiency without low or fluctuating serum bile acids or previous biliary surgery. The all-PFIC cohort combined the BSEP cohort with participants with biallelic FIC1, MDR3, TJP2, or MYO5B deficiencies without previous surgery but regardless of bile acids. The full cohort had no exclusions. Participants were randomly assigned (1:1) to receive oral maralixibat (starting dose 142·5 μg/kg, then escalated to 570 μg/kg) or placebo twice daily for 26 weeks. The primary endpoint was the mean change in average morning ItchRO(Obs) severity score between baseline and weeks 15-26 in the BSEP cohort. The key secondary efficacy endpoint was the mean change in total serum bile acids between baseline and the average of weeks 18, 22, and 26 in the BSEP cohort. Efficacy analyses were done in the intention-to-treat population (all those randomly assigned) and safety analyses were done in all participants who received at least one dose of study drug. This completed trial is registered with ClinicalTrials.gov, NCT03905330, and EudraCT, 2019-001211-22.
    RESULTS: Between July 9, 2019, and March 4, 2022, 125 patients were screened, of whom 93 were randomly assigned to maralixibat (n=47; 14 in the BSEP cohort and 33 in the all-PFIC cohort) or placebo (n=46; 17 in the BSEP cohort and 31 in the all-PFIC cohort), received at least one dose of study drug, and were included in the intention-to-treat and safety populations. The median age was 3·0 years (IQR 2·0-7·0) and 51 (55%) of 93 participants were female and 42 (45%) were male. In the BSEP cohort, least-squares mean change from baseline in morning ItchRO(Obs) was -1·7 (95% CI -2·3 to -1·2) with maralixibat versus -0·6 (-1·1 to -0·1) with placebo, with a significant between-group difference of -1·1 (95% CI -1·8 to -0·3; p=0·0063). Least-squares mean change from baseline in total serum bile acids was -176 μmol/L (95% CI -257 to -94) for maralixibat versus 11 μmol/L (-58 to 80) for placebo, also representing a significant difference of -187 μmol/L (95% CI -293 to -80; p=0·0013). The most common adverse event was diarrhoea (27 [57%] of 47 patients on maralixibat vs nine [20%] of 46 patients on placebo; all mild or moderate and mostly transient). There were five (11%) participants with serious treatment-emergent adverse events in the maralixibat group versus three (7%) in the placebo group. No treatment-related deaths occurred.
    CONCLUSIONS: Maralixibat improved pruritus and predictors of native liver survival in PFIC (eg, serum bile acids). Maralixibat represents a non-surgical, pharmacological option to interrupt the enterohepatic circulation and improve the standard of care in patients with PFIC.
    BACKGROUND: Mirum Pharmaceuticals.
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  • 文章类型: Journal Article
    目的:特应性皮炎(AD)对生活质量(QoL)产生不利影响。我们评估了upadacitinib的效果,口服选择性Janus激酶抑制剂被批准用于中度至重度AD,加上局部皮质类固醇(+TCS)对患者报告的结果(PRO)超过52周。
    材料和方法:在3期ADUp研究(NCT03568318)中,患有中度至重度AD的成人和青少年随机分为1:1:1,每天一次upadacitinib15mg,30毫克,或安慰剂+TCS。痒,皮肤疼痛/症状,睡眠,QoL,日常活动,情绪状态,心理健康,并评估患者对疾病严重程度/改善/治疗满意度的印象。
    结果:该分析包括901例患者。在1-2周内,upadacitinib两种剂量的PRO改善均大于安慰剂(p<0.05)。改善在第4-8周增加;率通常维持到第52周。在第52周,瘙痒有临床意义的改善的患者比例(最严重瘙痒数字评分量表改善≥4),皮肤疼痛(AD症状量表皮肤疼痛改善≥4),睡眠(AD影响量表[ADerm-IS]睡眠改善≥12),日常活动(ADerm-IS日常活动改善≥14),和情绪状态(ADerm-IS情绪状态改善≥11)在upadacitinib15mg+TCS的情况下为62.1%-77.7%,在upadacitinib30mg+TCS的情况下为71.3%-83.6%。
    结论:Upadacitinib+TCS结果迅速,持续改善严重的AD症状和QoL。
    Purpose: Atopic dermatitis (AD) adversely impacts quality of life (QoL). We evaluated the effect of upadacitinib, an oral selective Janus kinase inhibitor approved for moderate-to-severe AD, plus topical corticosteroids (+TCS) on patient-reported outcomes (PROs) over 52 weeks.
    Materials and methods: In the phase 3 AD Up study (NCT03568318), adults and adolescents with moderate-to-severe AD were randomized 1:1:1 to once-daily upadacitinib 15 mg, 30 mg, or placebo + TCS. Itch, skin pain/symptoms, sleep, QoL, daily activities, emotional state, mental health, and patient impressions of disease severity/improvement/treatment satisfaction were assessed.
    Results: This analysis included 901 patients. Within 1-2 weeks, PRO improvements were greater with both upadacitinib doses than with placebo (p <.05). Improvements increased through weeks 4-8; rates were generally maintained through week 52. At week 52, the proportion of patients with clinically meaningful improvements in itch (Worst Pruritus Numerical Rating Scale improvement ≥4), skin pain (AD Symptom Scale Skin Pain improvement ≥4), sleep (AD Impact Scale [ADerm-IS] Sleep improvement ≥12), daily activities (ADerm-IS Daily Activities improvement ≥14), and emotional state (ADerm-IS Emotional State improvement ≥11) ranged from 62.1%-77.7% with upadacitinib 15 mg + TCS and 71.3%-83.6% with upadacitinib 30 mg + TCS.
    Conclusions: Upadacitinib + TCS results in rapid, sustained improvements in burdensome AD symptoms and QoL.
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