Off-Label use

标签外使用
  • DOI:
    文章类型: Journal Article
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  • DOI:
    文章类型: Letter
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  • 文章类型: Journal Article
    背景:A型肉毒杆菌毒素是一种公认的美学和医学治疗方法。虽然B型毒素的使用不太常见,人们对使用B型毒素越来越感兴趣,尤其是那些对治疗有抵抗力的人。
    目的:为了评估FDA批准的利莫他林毒素B的主要临床应用,以及已建立和正在出现的标签外临床适应症。
    方法:文章来自PubMed数据库和食品和药物管理指南。
    结果:面部韵律倾向于在A型和B型毒素之间使用较高的转化率,由于B型毒素对肌肉的亲和力较弱,对汗腺的亲和力较高。特别是,1:100到1:50的比例用于眉骨,1:25到1:50的比率为眼周Rhytis,宫颈肌张力障碍的比例为1:50至1:66.6,多汗症的比例为1:20至1:50,和1:25到30的比例流涎。
    结论:B型毒素已经证明了其在治疗面部韵律方面的安全性和有效性,宫颈肌张力障碍,流涎和多汗症,具有正在调查的新颖应用的潜力。无论注射位置和临床应用,口干和吞咽困难仍然是最常见的副作用。在所有迹象中,B型毒素似乎起效更快,剂量依赖性临床持续时间,和剂量依赖性不良反应。
    BACKGROUND: Botulinum type-A toxin is a well established aesthetic and medical treatment. While the usage of type-B toxin is less common, there is a growing interest in using type-B toxin, especially in those who are treatment resistant.
    OBJECTIVE: To evaluate the primary FDA-approved clinical applications of rimabotulinumtoxinB, along with established and emerging off-label clinical indications.
    METHODS: Articles were reviewed from PubMed database and Food and Drug Adminstration guidelines.
    RESULTS: Facial rhytids tend to use a higher conversion ratio between type A and type B toxin, due to type B toxin\'s weaker affinity to muscles and higher affinity for sweat glands. Specially, a 1:100 to 1:50 ratio was utilized for glabellar rhytids, a 1:25 to 1:50 ratio for periocular rhytids, a 1:50 to 1:66.6 ratio for cervical dystonia, a 1:20 to 1:50 ratio for hyperhidrosis, and a 1:25 to 30 ratio for sialorrhea.
    CONCLUSIONS: Type B toxin has demonstrated its safety and efficacy in treating facial rhytids, cervical dystonia, sialorrhea and hyperhidrosis, with potential for novel applications under investigation. Regardless of injection location and clinical applications, dry mouth and dysphagia remained the most common side effects. Across all indications, type B toxin appeared to have a faster onset of action, a dose-dependent clinical duration, and a dose-dependent adverse effect profile.
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  • 文章类型: Case Reports
    数十年来,迷走神经刺激(VNS)已被用作耐药性癫痫的辅助治疗选择。传统上,左迷走神经用于刺激,而右迷走神经很少使用。正确的VNS(R-VNS)在人类中的长期疗效和安全性尚不清楚。我们介绍了三名接受R-VNS治疗的患者,随访时间长达8年。所有三名患者均耐受R-VNS,并发症最少。R-VNS在所有3例患者中均显示出合理的有效性。一名患者反应良好,无癫痫发作。与先前的左VNS治疗相比,其他两名患者对R-VNS的反应较差。
    Vagus nerve stimulation (VNS) has been used as an adjunctive therapeutic option for drug-resistant epilepsy for decades. Traditionally, the left vagus nerve is used for stimulation, while the right vagus nerve is rarely used. The long-term efficacy and safety of the right VNS (R-VNS) in humans are unknown. We presented three patients who were treated with R-VNS over a follow-up period of up to eight years. All three patients tolerated R-VNS well with minimal complications. R-VNS displayed reasonable effectiveness in all three patients. One patient had an excellent response and became seizure-free. The other two patients demonstrated a less favorable response to R-VNS compared to their previous left VNS therapy.
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  • 文章类型: Journal Article
    背景:2005年,美国食品和药物管理局发布了一个黑匣子警告,关于痴呆症患者使用超标签抗精神病药物(APM)导致死亡率和心血管事故增加。为了提高养老院(NH)痴呆患者的护理质量,2012年初,医疗保险和医疗补助服务中心启动了国家伙伴关系(NP),旨在打击过度使用APM。在合作的五年内,NHs的抗精神病药物处方数量减少了34%.本研究的目的是检查APM的使用如何影响其他结果,包括死亡率和主要心血管事件,如中风和心肌梗死(MI)在NH居民痴呆。
    方法:我们分析了611,773名患有痴呆症的长期NH居民的医疗保险数据,50岁及以上,从2011年到2016年。在此期间接受过临终关怀的居民被排除在外。进行了横断面(按年份)逻辑回归,以检查APM使用与死亡的关联。中风,和MI在2011-2016年。根据人口统计和合并症指标调整回归模型。
    结果:Logistic回归(参见表1中的结果)显示,从2011年到2015年,与未使用APM的NH居民相比,使用APM的NH居民的死亡概率明显更高。然而,2016年,APM用户的死亡概率低于非用户,虽然协会很弱。此外,从2011年到2016年,接受APM的NH居民比非使用者患中风或MI的可能性较小。
    结论:目前的研究结果表明,使用APM可能与长期NH居民的死亡概率增加有关,这与以前的文献是一致的。APM使用与卒中和MI负相关的发现与普遍的理解不一致,即APM使用会导致这些结果的风险更高。2016年死亡可能性的变化以及与中风和MI的负相关,需要进一步调查NH居民服用抗精神病药物的死亡和心血管事件的可能影响因素。
    BACKGROUND: In 2005, the Food and Drug Administration issued a black box warning regarding increased mortality and cardiovascular accidents with the off-label use of antipsychotic medication (APM) in patients with dementia. To improve the quality of care for patients with dementia in nursing homes (NH), in early 2012, the Centers for Medicare & Medicaid Services launched the National Partnership (NP) aimed at combatting the excessive use of APM. Within five years of the partnership, the number of antipsychotic prescriptions in NHs decreased by 34%. The objective of the present study was to examine how APM use has impacted other outcomes, including mortality and major cardiovascular events such as stroke and myocardial infarction (MI) among NH residents with dementia.
    METHODS: We analyzed Medicare data on a sample of 611,773 long-term NH residents with dementia, aged 50 years and older, from 2011 through 2016. Residents who had received hospice care during the time period were excluded. Cross-sectional (by year) logistic regressions were conducted to examine the associations of APM use with death, stroke, and MI in the years 2011-2016. Regression models were adjusted for demographic and co-morbidity indicators.
    RESULTS: Logistic regressions (see results in Table 1) revealed that NH residents prescribed APM had a significantly higher probability of death compared to non-users from 2011 through 2015. However, in 2016, the probability of death was lower among APM users than non-users, although the association was weak. Additionally, NH residents who were on APM were less likely to have a stroke or MI than non-users from 2011 through 2016.
    CONCLUSIONS: Current study findings suggest that APM use is likely associated with a heightened probability of death among long-term NH residents, which is consistent with previous literature. The finding that APM use is negatively associated with stroke and MI is inconsistent with prevailing understandings that APM use would lead to higher risks of these outcomes. The shift in the likelihood of death in 2016, and negative associations with stroke and MI warrant further investigation as to the possible contributing factors of death and cardiovascular events in NH residents on antipsychotic medications.
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  • DOI:
    文章类型: Journal Article
    大多数新药没有针对某些人群的标签,如婴儿和儿童;“标签外”药物的使用在儿科患者中很常见。在这篇文章中,作者向儿科医生介绍复合药剂师的服务。他讨论了局部麻醉组合,泻药配方,治疗注意力缺陷多动障碍的药物,抗恶心药物,尿布疹药物,痤疮药物和头部冰药物。他得出的结论是,复合药剂师必须运用创新的思维来制定成人药物的儿科滴定法,并使这些滴定法调味,以使其更适合儿童。
    Most new drugs are not labeled for certain populations, such as infants and children; and \"off-label\" use of drugs is common in pediatric patients. In this article, the author introduces pediatricians to the services of compounding pharmacists. He discusses topical anesthetic combinations, laxative formulations, medications for attention-deficit hyperactivity disorder, antinausea medications, diaper-rash medications, acne medications and head -ice medications. He concludes that the compounding pharmacist must use innovative thinking to formulate pediatric titrations of adult medications and to flavor those titrations to make them more palatable for children.
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  • 文章类型: Journal Article
    氨苯砜,最初合成用于纺织染色,在1930年代因其抗菌性能而获得认可,导致其用于麻风病和疱疹样皮炎的皮肤病学。尽管美国食品和药物管理局(FDA)批准了这些条件,dapsone的标签外用途已经扩展,使其成为各种皮肤病的有价值的选择。这篇综述旨在强调氨苯砜在其FDA适应症和标签外适应症中的常见用途。回顾了氨苯砜被认为是一线治疗或辅助治疗的疾病,包括资源中的亮点。氨苯砜的药代动力学概述,药效学,适应症,剂量,和安全性也审查。氨苯砜的多功能性和安全性使其成为皮肤科具有成本效益的治疗选择,特别是对于获得专门药物的患者。还描述了正在进行的临床试验,以探索氨苯砜在新型皮肤病学用途中的功效。多年来,氨苯砜一直是各种皮肤病的有价值的辅助疗法,其使用的证据也在不断扩大。
    Dapsone, initially synthesized for textile dyeing, gained recognition in the 1930s for its antibacterial properties, leading to its utilization in dermatology for leprosy and dermatitis herpetiformis. Despite US Food and Drug Administration (FDA) approval for these conditions, dapsone\'s off-label uses have expanded, making it a valuable option in various dermatologic conditions. This review seeks to highlight the common uses of dapsone in its FDA indications and off-label indications. Diseases in which dapsone is considered first-line therapy or adjunctive therapy are reviewed, with highlights from the resources included. An overview of dapsone\'s pharmacokinetics, pharmacodynamics, indications, dosages, and safety profile are also reviewed. Dapsone\'s versatility and safety profile make it a cost-effective treatment option in dermatology, particularly for patients with limited access to specialized medications. Ongoing clinical trials are also described exploring dapsone\'s efficacy in novel dermatologic uses. Dapsone has been a valuable adjunctive therapy across various dermatologic conditions for years and evidence for its use continues to expand.
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  • 文章类型: Interview
    这篇医学新闻文章是对罗伯特·加贝的采访,MD,PhD,美国糖尿病协会首席科学和医学官。
    This Medical News article is an interview with Robert Gabbay, MD, PhD, the American Diabetes Association’s chief scientific and medical officer.
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  • 文章类型: Journal Article
    目标:有些人正在使用药物来尝试增强认知和社会情感功能,并要求医生提供非标签处方以增强神经功能(例如,兴奋剂)。几个医学协会发布了关于处方神经增强剂的指南,其中一些是指神经增强的潜在社会影响(例如,分配正义),除了给用户带来的风险和好处。哪些机构(例如,医学社会,政府,大学)应该做出允许神经增强的决定,他们是否应该考虑潜在的社会影响还不清楚。我们研究了允许药用神经增强剂的支持是否以及多少受神经增强剂的机构以及潜在的个人和社会影响的影响。
    方法:我们使用美国成年公众的代表性样本进行了离散选择实验。使用多项logit模型对数据进行分析。
    结果:参与者(n=927)在人口统计学上与美国人口相似。严重副作用的风险(OR0.20,CI0.18-0.22)和用户缺乏益处(OR0.31,CI0.26-0.38)对参与者支持神经增强剂的负面影响最大。轻度副作用的风险对参与者支持允许神经增强剂(OR0.67,CI0.62-0.74)和更有意义的前景有中等的负面影响,对用户的长期益处为中度正效应(OR1.74,CI1.61-1.87)。神经增强剂对社会中人们的平均福祉和平等的积极或消极影响对参与者允许神经增强剂的支持有中等影响。例如,参与者支持允许对社会福祉有负面影响的增强剂的几率约为一半(OR0.45,CI0.40-0.50),允许不平等恶化的增强剂的几率比没有这种效应的增强剂低约40%(OR0.62,CI0.55-0.71).如果增强剂降低了使用者的真实性,则参与者允许神经增强剂的几率略低(10%)(OR0.90,CI0.84-0.97)。调节神经增强子和神经增强子为用户提供不公平优势的机构不会影响参与者的决定。
    结论:当提出个人和社会考虑时,公众似乎支持医疗社会和其他机构根据用户的风险和利益做出关于神经增强剂的政策决定,还有,但在较小程度上,对平等和社会福利的影响。
    OBJECTIVE: Some individuals are using drugs to try to enhance cognitive and social-affective functioning and asking physicians for off-label prescriptions for neuroenhancement (e.g., stimulants). Several medical societies released guidance on prescribing neuroenhancers, some of which refer to potential societal effects of neuroenhancement (e.g., distributive justice), besides risks and benefits to users. Which institutions (e.g., medical societies, government, universities) should make decisions on allowing neuroenhancement and whether they should consider potential societal effects are unclear. We examined whether and how much support for allowing pharmaceutical neuroenhancers was influenced by the institution and potential individual and societal effects of neuroenhancers.
    METHODS: We conducted a discrete-choice experiment using a constructed representative sample of the US adult public. Multinomial logit models were used to analyze the data.
    RESULTS: Participants (n = 927) demographically resembled the US population. Risks of serious side effects (OR 0.20, CI 0.18-0.22) and a lack of benefits for users (OR 0.31, CI 0.26-0.38) had the largest negative effect on participants\' support for allowing neuroenhancers. A risk of mild side effects had a moderate negative effect on participants\' support for allowing neuroenhancers (OR 0.67, CI 0.62-0.74) and the prospect of more meaningful, long-lasting benefits for users a moderate positive effect (OR 1.74, CI 1.61-1.87). Positive or negative effects of neuroenhancers on the average well-being of people in society and on equality had moderate effects on participants\' support for allowing neuroenhancers. For example, the odds of participants\' support for allowing enhancers with a negative effect on societal well-being were around half (OR 0.45, CI 0.40-0.50) and the odds of allowing enhancers that worsen inequality were approximately 40% lower compared with enhancers without such effects (OR 0.62, CI 0.55-0.71). The odds of participants allowing neuroenhancers were slightly (10%) lower if enhancers reduced users\' authenticity (OR 0.90, CI 0.84-0.97). The institution regulating neuroenhancers and neuroenhancers providing users with an unfair advantage did not affect participants\' decisions.
    CONCLUSIONS: When presented with both individual and societal considerations, the public seems to support medical societies and other institutions making policy decisions about neuroenhancers based on risks and benefits for users, as well as, but to a lesser extent, effects on equality and societal well-being.
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  • 文章类型: Journal Article
    这项研究的目的是评估贝伐单抗-awwb在新生血管性年龄相关性黄斑变性(n-AMD)和糖尿病性黄斑水肿(DME)的非标签治疗中的疗效和安全性。
    所有n-AMD和DME患者在维持阶段按照“治疗和延长”策略治疗,从2022年10月至2023年4月,在罗马的TorVergata综合诊所接受了从贝伐单抗到贝伐单抗-awwb的强制药物替代,在一项回顾性研究中进行了评估。主要结果是药物替代后中央视网膜厚度(CRT)随时间的变化。次要结果是药物耐久性的变化,最佳矫正视力(BCVA)和视网膜液,以及与药物相关的局部和全身严重不良事件的发生率。
    纳入76例n-AMD患者的80只眼和44例DME患者的55只眼,在药物替代之前和之后,平均CRT无统计学差异;q8,q12和q16时间间隔内的患者比例无差异;平均BCVA保持不变.在累计3496种贝伐单抗-awwb治疗中(n-AMD患者为2154种,DME患者为1342种),未发现局部严重并发症.在总共544名患者中(342名受n-AMD影响,202名受DME影响),未报告严重不良事件.
    在我们的n-AMD和DME维持期患者队列中,贝伐单抗-awwb似乎代表了一种可行且具有成本效益的玻璃体内治疗方法,其疗效和安全性与鼻祖相当.
    本研究对玻璃体内贝伐单抗-awwb的疗效和安全性进行了初步评估,这是广泛用于视网膜血管疾病的标签外。
    UNASSIGNED: The purpose of this study was to evaluate the efficacy and safety of bevacizumab-awwb in the off-label treatment of neovascular age-related macular degeneration (n-AMD) and diabetic macular edema (DME).
    UNASSIGNED: All patients with n-AMD and DME treated in the maintenance phase according to the \"treat and extend\" strategy, who underwent forced drug substitution from bevacizumab to bevacizumab-awwb from October 2022 to April 2023 at the Tor Vergata Polyclinic in Rome, were evaluated in a retrospective study. The primary outcome was changes in central retinal thickness (CRT) over time following drug substitution. The secondary outcomes were variations in drug durability, best corrected visual acuity (BCVA) and retinal fluid, and the incidence of drug-related local and systemic serious adverse events.
    UNASSIGNED: Of 80 eyes of 76 patients with n-AMD and 55 eyes of 44 patients with DME included, before and after drug substitution, the average CRT did not statistically differ; the proportion of patients within time intervals of q8, q12, and q16 was not different; and the mean BCVA remained constant. Of a cumulative 3496 bevacizumab-awwb treatments (2154 for patients with n-AMD and 1342 for patients with DME), no local severe complications were detected. Out of a total of 544 patients (342 affected by n-AMD and 202 affected by DME), no serious adverse events were reported.
    UNASSIGNED: In our cohort of patients with n-AMD and DME in the maintenance phase, bevacizumab-awwb seems to represent a viable and cost-effective intravitreal therapy with comparable efficacy and safety to the originator.
    UNASSIGNED: This study provides a preliminary assessment of the efficacy and safety of intravitreal bevacizumab-awwb, which is widely used off-label in retinal vascular diseases.
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