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Descriptive Analysis of Types and Diagnoses Associated with Lower Extremity Amputation: Analysis of the US Veterans Health Administration Database 2019–2023 与下肢截肢相关的类型和诊断的描述性分析:2019-2023年美国退伍军人健康管理局数据库分析。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-16 DOI: 10.1007/s12325-024-03005-6
Brajesh Lal, Chi Gao, Fan Mu, Grace Chen, Qi Hua, Jared Calish, Marie Parker

Introduction

Veterans in the US have higher rates of lower extremity amputation (LEA) compared to the general population and these rates have increased between 2008 and 2018. There is limited data which directly evaluate the potential underlying comorbidities associated with LEA in the veterans’ population especially with the most recent data. Such information is critical to help inform clinical management strategies to reduce the risk of amputations among our veterans.

Methods

This was a retrospective observational study of adults in the Veterans Health Administration database who underwent LEA from January 1, 2019 to December 31, 2023. The date of the first LEA procedure was defined as the index date. Index LEA type, patient demographic at baseline, and clinical characteristics (including diagnoses for conditions associated with LEA and other comorbidities) 1 year before and 30 days after the index LEA procedure (except for bacterial infections which the identification period was 30 days before and 30 days after the index LEA procedure) were described.

Results

Of the 27,134 Veterans with LEA, 67.3% were ≥ 65 years of age, 97.0% were male, and 65.3% were non-Hispanic white. The most common type of LEA was transmetatarsal (52.9%), followed by toe (21.9%), above-knee (15.4%), and below-knee (9.8%). The most prevalent diagnoses associated with LEA were diabetes (81.6%), bacterial infections (79.1%), and peripheral artery disease (PAD; 63.3%). Only 15 Veterans (< 0.1%) had a diagnosis for combat-related injuries to lower extremities.

Conclusion

Diabetes and PAD are highly prevalent and among the main conditions associated with LEA among US Veterans. Earlier and more effective preventative and clinical management of these conditions offer an opportunity to significantly reduce the rates of LEA in this population.

导言:与普通人群相比,美国退伍军人的下肢截肢(LEA)率较高,而且在 2008 年至 2018 年期间,这一比率有所上升。直接评估退伍军人群体中与下肢截肢相关的潜在合并症的数据有限,尤其是最新数据。这些信息对于帮助制定临床管理策略以降低退伍军人截肢风险至关重要:这是一项回顾性观察研究,研究对象是退伍军人健康管理局数据库中在 2019 年 1 月 1 日至 2023 年 12 月 31 日期间接受 LEA 的成年人。首次 LEA 手术的日期被定义为索引日期。对指数 LEA 类型、基线时的患者人口统计学特征以及指数 LEA 手术前 1 年和手术后 30 天的临床特征(包括与 LEA 相关的疾病诊断和其他合并症)(细菌感染除外,其识别期为指数 LEA 手术前 30 天和手术后 30 天)进行了描述:在 27,134 名患有 LEA 的退伍军人中,67.3% 年龄≥ 65 岁,97.0% 为男性,65.3% 为非西班牙裔白人。最常见的 LEA 类型是跨跖骨(52.9%),其次是脚趾(21.9%)、膝盖以上(15.4%)和膝盖以下(9.8%)。与 LEA 相关的最常见诊断是糖尿病(81.6%)、细菌感染(79.1%)和外周动脉疾病(PAD;63.3%)。只有 15 名退伍军人(结论:在美国退伍军人中,糖尿病和 PAD 发病率很高,是与 LEA 相关的主要疾病之一。更早、更有效地对这些疾病进行预防和临床治疗,是大幅降低该人群 LEA 发生率的良机。
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引用次数: 0
A Systematic Literature Review and Network Meta-analysis of Azilsartan Medoxomil Compared to Other Anti-hypertensives Efficacy in Lowering Blood Pressure Amongst Mild to Moderate Hypertensive Patients 阿齐沙坦美多米与其他抗高血压药降低轻度至中度高血压患者血压疗效的系统性文献综述和网络 Meta 分析。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-16 DOI: 10.1007/s12325-024-02997-5
Juying Qian, Mengjun Zhang, Zhangwei Chen

Introduction

A systematic literature review and network meta-analysis was conducted on azilsartan medoxomil (AZL-M) versus other antihypertensive drugs’ efficacy in hypertensive patients.

Methods

The search utilized English platforms, from January 2000 until December 2023, resulting in 10,380 articles being screened. Screening criteria included hypertension (mild or moderate); first-line treatment and washout periods; studies (monotherapy) with AZL-M, angiotensin type II receptor blockers (ARBs), angiotensin-converting enzyme inhibitors (ACEIs), angiotensin receptor neprilysin inhibitor (ARNIs), beta-blockers, calcium channel blockers (CCBs), and diuretics, either as intervention or comparator; and antihypertension efficacy as an outcome measure. Study design was randomized clinical trials. Efficacy variables included absolute office systolic and diastolic blood pressure (BP) reductions. A total of 21 publications provided adequate data for analysis, of which 20 studies reported both systolic and diastolic BP and one study reported only the diastolic BP.

Results

In 21 studies on systolic BP, against the common comparator placebo, the differences in systolic BP were significantly in favor of AZL-M, amlodipine, candesartan, irbesartan, nebivolol, nifedipine, olmesartan, sacubitril valsartan, telmisartan, and valsartan. The surface under the cumulative ranking curve (SUCRA) ranking shows that AZL-M 80 mg had the highest ranking, with a possibility of 93% being the best in all other included treatments. In 20 studies on diastolic BP, against the common comparator placebo, the differences in diastolic BP were significantly in favor of AZL-M, amlodipine, bisoprolol, nebivolol, olmesartan, sacubitril valsartan, telmisartan, and valsartan. The SUCRA ranking shows that AZL-M 80 mg had the highest ranking, with a possibility of 90% being the best in all other included treatments.

Conclusion

AZL-M at 40 mg and 80 mg shows favorable efficacy compared to other anti-hypertensives, and the 80 mg dosage seemed to be the most efficacious of all the included treatments in reducing both office systolic and diastolic BP in patients with mild-to-moderate hypertension.

简介本研究对阿齐沙坦酯(AZL-M)与其他抗高血压药物在高血压患者中的疗效进行了系统性文献综述和网络荟萃分析:检索利用英语平台,检索期从 2000 年 1 月至 2023 年 12 月,共筛选出 10,380 篇文章。筛选标准包括:高血压(轻度或中度);一线治疗和冲洗期;使用 AZL-M、血管紧张素 II 型受体阻滞剂(ARB)、血管紧张素转换酶抑制剂(ACEI)、血管紧张素受体肾酶抑制剂(ARNI)、β-受体阻滞剂、钙通道阻滞剂(CCB)和利尿剂作为干预或比较药物的研究(单药治疗);以及作为结果测量指标的抗高血压疗效。研究设计为随机临床试验。疗效变量包括办公室收缩压和舒张压(BP)的绝对降低幅度。共有 21 篇文献提供了足够的分析数据,其中 20 项研究同时报告了收缩压和舒张压,1 项研究仅报告了舒张压:结果:在 21 项关于收缩压的研究中,与常用的比较药安慰剂相比,AZL-M、氨氯地平、坎地沙坦、厄贝沙坦、奈比洛尔、硝苯地平、奥美沙坦、沙库比特利缬沙坦、替米沙坦和缬沙坦的收缩压差异显著。累积排名曲线下表面(SUCRA)排名显示,AZL-M 80 毫克的排名最高,有 93% 的可能性在所有其他纳入的治疗中名列前茅。在 20 项关于舒张压的研究中,与常用的参照安慰剂相比,AZL-M、氨氯地平、比索洛尔、奈比洛尔、奥美沙坦、沙库比特利缬沙坦、替米沙坦和缬沙坦的舒张压差异显著。SUCRA 排名显示,AZL-M 80 毫克的排名最高,90% 的可能性是所有其他治疗方法中最好的:结论:与其他降压药相比,AZL-M 40 毫克和 80 毫克剂量显示出良好的疗效,在所有纳入的治疗中,80 毫克剂量似乎对降低轻中度高血压患者的办公室收缩压和舒张压最有效。
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引用次数: 0
Cost-Effectiveness of Teduglutide for Pediatric Patients with Short Bowel Syndrome in Japan, Including Caregiver Burden 特度鲁肽治疗日本小儿短肠综合征患者的成本效益,包括护理人员的负担。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-16 DOI: 10.1007/s12325-024-02995-7
Hisato Deguchi, Masafumi Kato

Introduction

Short bowel syndrome (SBS) is associated with a significant mental and physical burden for patients and caregivers. Standard of care (SOC) for SBS includes parenteral support (PS) to optimize intestinal function. Teduglutide, a recombinant human glucagon-like peptide 2 analogue, reduces the need for PS in patients with SBS. In this study, we assessed the cost-effectiveness of teduglutide in pediatric patients with SBS from multiple perspectives, considering the caregiver’s burden.

Methods

A Markov model was used to evaluate cost (Japanese yen, JPY) and effectiveness (quality-adjusted life years, QALYs) of teduglutide compared with SOC for pediatric patients with SBS in Japan. We conducted a base-case analysis and selected sensitivity and scenario analyses from three perspectives: (1) the public healthcare payer, (2) the public healthcare and long-term care payer, and (3) society.

Results

In the base-case analysis, the incremental cost-effectiveness ratio (ICER) was 9,533,412 JPY per QALY from the public healthcare payer perspective, 6,335,980 JPY per QALY from the public healthcare and long-term care payer perspective, and 3,510,371 JPY per QALY from the societal perspective. The probability that cost-effectiveness of teduglutide is favorable from a societal perspective was 59.3%. In all scenario analyses, consistent with the base-case analysis, ICERs for teduglutide compared with SOC were different depending on whether caregiver utility and productivity loss were considered.

Conclusions

Incorporating the caregiver’s burden in the cost-effectiveness analysis of teduglutide for pediatric patients with SBS provided a more comprehensive assessment of the value of teduglutide for patients, their families, and society. This approach enhances our understanding of the overall value of a treatment, especially for diseases with significant caregiver burden.

简介:短肠综合征(SBS)给患者和护理人员带来了巨大的精神和身体负担。治疗 SBS 的标准疗法(SOC)包括肠外支持疗法(PS),以优化肠道功能。泰度鲁肽是一种重组人胰高血糖素样肽 2 类似物,可减少 SBS 患者对肠外支持的需求。在这项研究中,我们从多个角度评估了特度鲁肽治疗儿科SBS患者的成本效益,并考虑了护理人员的负担:方法:我们使用马尔可夫模型评估了日本儿童 SBS 患者使用特度鲁肽与 SOC 相比的成本(日元,JPY)和疗效(质量调整生命年,QALYs)。我们从以下三个角度进行了基本情况分析、敏感性分析和情景分析:(1) 公共医疗支付方;(2) 公共医疗和长期护理支付方;(3) 社会:在基础案例分析中,从公共医疗支付方的角度来看,每 QALY 的增量成本效益比为 9,533,412 日元;从公共医疗和长期护理支付方的角度来看,每 QALY 的增量成本效益比为 6,335,980 日元;从社会角度来看,每 QALY 的增量成本效益比为 3,510,371 日元。从社会角度看,特度鲁肽的成本效益有利的概率为 59.3%。在所有情景分析中,与基础情景分析一致的是,与SOC相比,特度鲁肽的ICER因是否考虑护理人员的效用和生产力损失而有所不同:将照顾者的负担纳入特度鲁肽治疗儿童 SBS 患者的成本效益分析,可更全面地评估特度鲁肽对患者、患者家庭和社会的价值。这种方法提高了我们对治疗总体价值的认识,尤其是对护理负担较重的疾病。
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引用次数: 0
A Phase 3, Open-Label, Single-Arm Trial of the Efficacy and Safety of Triptorelin 6-Month Formulation in Chinese Children with Central Precocious Puberty 特普瑞林 6 个月制剂对中国中枢性性早熟儿童疗效和安全性的 3 期、开放标签、单臂试验。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-16 DOI: 10.1007/s12325-024-02991-x
Xiao Yu, Xinran Cheng, Haiyan Wei, Xu Xu, Chunxiu Gong, Guimei Li, Hui Yao, Li Zhou, Yan Zhong, Yu Yang, Feihong Luo, Yining Zhang, Frank Huang, Xiaofeng Shi, Patrick Cabri, Xiaoping Luo

Introduction

This phase 3 study assessed the efficacy, safety, and pharmacokinetics of the 6-month prolonged release (PR) formulation in Chinese children with central precocious puberty (CPP).

Methods

In this open-label study (NCT05029622), Chinese children (girls < 9 years, boys < 10 years) received two doses of triptorelin pamoate 22.5 mg (day 1 and month 6). Primary endpoint was the proportion at month 6 with luteinizing hormone (LH) suppression (stimulated peak LH ≤ 5 IU/L after gonadotropin-releasing hormone stimulation). Secondary endpoints included safety assessments, hormone level changes, and clinical parameters from baseline.

Results

Overall, 66 children completed the study (93.9% girls; median age 8.0 [range 5–9] years). At month 6, all patients had LH suppression; this was maintained at month 12 in 98.5% of patients. Mean basal and peak LH and follicle-stimulating hormone levels were suppressed throughout follow-up. All patients at months 3 to 12 had sex hormone suppression to prepubertal levels. Stable or reduced breast development was seen for 98.4% and 93.5% of girls at month 6 and 12, respectively; all boys had regression or stable genital development until month 12. Compared with baseline (9.82 cm/year), mean growth velocity was 5.88 cm/year at month 6 and 5.17 cm/year at month 12. Mean bone age/chronological age ratio decreased from 1.27 at baseline to 1.23 and 1.21 at month 6 and 12, respectively. In girls, 64.5% showed decreased uterine length at month 6 and 12 versus baseline, while 75.0% of boys showed stable testicular volume versus baseline. Thirteen patients (19.7%) had 22 drug-related treatment emergent adverse events (TEAEs); no grade ≥ 3 TEAEs were reported.

Conclusion

The efficacy and safety profile of triptorelin 6-month PR in Chinese children with CPP was consistent with data previously reported in non-Chinese children with CPP, supporting this as a viable treatment option for Chinese children with CPP.

Trial Registration

Trial registration: ClinicalTrials.gov identifier, NCT05029622.

简介:本3期研究评估了6个月长效缓释制剂(PR)对中国中枢性性早熟(CPP)儿童的疗效、安全性和药代动力学:本 3 期研究评估了中枢性性早熟(CPP)患儿服用 6 个月长效缓释制剂(PR)的疗效、安全性和药代动力学:在这项开放标签研究(NCT05029622)中,中国儿童(女孩 结果:共有 66 名儿童完成了研究:共有 66 名儿童完成了研究(93.9% 为女孩;中位年龄为 8.0 [5-9]岁)。第 6 个月时,所有患者的 LH 均得到抑制;第 12 个月时,98.5% 的患者的 LH 均得到抑制。在整个随访期间,LH和卵泡刺激素的基础和峰值平均水平均受到抑制。在第 3 至 12 个月期间,所有患者的性激素水平都被抑制到了青春期前的水平。在第 6 个月和第 12 个月,分别有 98.4% 和 93.5% 的女孩乳房发育稳定或减弱;所有男孩的生殖器发育在第 12 个月前都有所减弱或稳定。与基线(9.82 厘米/年)相比,第 6 个月和第 12 个月的平均生长速度分别为 5.88 厘米/年和 5.17 厘米/年。平均骨龄/同步年龄比从基线时的1.27降至第6个月时的1.23和第12个月时的1.21。与基线相比,64.5%的女孩在第 6 个月和第 12 个月的子宫长度有所减少,而 75.0%的男孩的睾丸体积与基线相比保持稳定。13名患者(19.7%)发生了22起与药物相关的治疗突发不良事件(TEAEs);没有≥3级TEAEs的报告:结论:曲普瑞林6个月PR在中国儿童CPP患者中的疗效和安全性与之前在非中国儿童CPP患者中报道的数据一致,支持将其作为中国儿童CPP患者的可行治疗方案:试验注册ClinicalTrials.gov identifier,NCT05029622。
{"title":"A Phase 3, Open-Label, Single-Arm Trial of the Efficacy and Safety of Triptorelin 6-Month Formulation in Chinese Children with Central Precocious Puberty","authors":"Xiao Yu,&nbsp;Xinran Cheng,&nbsp;Haiyan Wei,&nbsp;Xu Xu,&nbsp;Chunxiu Gong,&nbsp;Guimei Li,&nbsp;Hui Yao,&nbsp;Li Zhou,&nbsp;Yan Zhong,&nbsp;Yu Yang,&nbsp;Feihong Luo,&nbsp;Yining Zhang,&nbsp;Frank Huang,&nbsp;Xiaofeng Shi,&nbsp;Patrick Cabri,&nbsp;Xiaoping Luo","doi":"10.1007/s12325-024-02991-x","DOIUrl":"10.1007/s12325-024-02991-x","url":null,"abstract":"<div><h3>Introduction</h3><p>This phase 3 study assessed the efficacy, safety, and pharmacokinetics of the 6-month prolonged release (PR) formulation in Chinese children with central precocious puberty (CPP).</p><h3>Methods</h3><p>In this open-label study (NCT05029622), Chinese children (girls &lt; 9 years, boys &lt; 10 years) received two doses of triptorelin pamoate 22.5 mg (day 1 and month 6). Primary endpoint was the proportion at month 6 with luteinizing hormone (LH) suppression (stimulated peak LH ≤ 5 IU/L after gonadotropin-releasing hormone stimulation). Secondary endpoints included safety assessments, hormone level changes, and clinical parameters from baseline.</p><h3>Results</h3><p>Overall, 66 children completed the study (93.9% girls; median age 8.0 [range 5–9] years). At month 6, all patients had LH suppression; this was maintained at month 12 in 98.5% of patients. Mean basal and peak LH and follicle-stimulating hormone levels were suppressed throughout follow-up. All patients at months 3 to 12 had sex hormone suppression to prepubertal levels. Stable or reduced breast development was seen for 98.4% and 93.5% of girls at month 6 and 12, respectively; all boys had regression or stable genital development until month 12. Compared with baseline (9.82 cm/year), mean growth velocity was 5.88 cm/year at month 6 and 5.17 cm/year at month 12. Mean bone age/chronological age ratio decreased from 1.27 at baseline to 1.23 and 1.21 at month 6 and 12, respectively. In girls, 64.5% showed decreased uterine length at month 6 and 12 versus baseline, while 75.0% of boys showed stable testicular volume versus baseline. Thirteen patients (19.7%) had 22 drug-related treatment emergent adverse events (TEAEs); no grade ≥ 3 TEAEs were reported.</p><h3>Conclusion</h3><p>The efficacy and safety profile of triptorelin 6-month PR in Chinese children with CPP was consistent with data previously reported in non-Chinese children with CPP, supporting this as a viable treatment option for Chinese children with CPP.</p><h3>Trial Registration</h3><p>Trial registration: ClinicalTrials.gov identifier, NCT05029622.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"41 12","pages":"4537 - 4556"},"PeriodicalIF":3.4,"publicationDate":"2024-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-02991-x.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142455871","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-World Safety and Effectiveness of Infliximab in 255 Patients with Intestinal, Neurological, and Vascular Behçet’s Disease: A Post-Marketing Surveillance 英夫利西单抗对 255 例肠道、神经和血管性白塞氏病患者的实际安全性和有效性:上市后监测
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-16 DOI: 10.1007/s12325-024-02993-9
Toshifumi Hibi, Shunsei Hirohata, Tadakazu Hisamatsu, Hirotoshi Kikuchi, Mitsuhiro Takeno, Noriko Sato, Naomi Mizuno, Mayumi Tashiro, Yutaka Susuta, Yoshiaki Ishigatsubo

Introduction

Behçet’s disease (BD) with intestinal, neurological (NBD), and vascular (VBD) manifestations often leads to poor outcomes. Infliximab is approved for the treatment of intestinal BD, NBD, and VBD in Japan; however, evidence regarding its safety and effectiveness in these patients is limited. We conducted a 2-year post-marketing surveillance to evaluate the safety and effectiveness of infliximab in patients with intestinal BD, NBD, and VBD in Japan.

Methods

This 2-year, multicenter, prospective, observational study included all patients with intestinal BD, NBD, or VBD, who had experienced an insufficient response to conventional therapies (e.g., glucocorticoids and immunosuppressants/immunomodulators), and initiated infliximab for the first time at participating medical institutions. The safety endpoints included adverse events and adverse drug reactions (ADRs), and the effectiveness endpoints included global improvement, and for patients with acute NBD, acute attacks.

Results

Between October 2015 and August 2018, 255 patients (171 intestinal BD, 49 NBD, and 51 VBD; including 16 with two disease types) were enrolled from 133 medical centers and treated with infliximab. Adverse events, ADRs, and serious ADRs occurred in 100 (39.2%), 72 (28.2%), and 38 (14.9%) patients, respectively; incidences were generally similar across intestinal BD, NBD, and VBD groups. No new safety concerns were identified. At the final evaluation, 68.8% of patients with intestinal BD showed improvement, most patients with chronic progressive NBD and VBD had not worsened (100% and 91.7%, respectively), and 93.3% of patients with acute NBD had no new acute attacks during the observation period.

Conclusion

These results confirmed the safety and effectiveness of infliximab in clinical practice in 255 patients with intestinal BD, NBD, and VBD. There were no new safety concerns.

简介:伴有肠道、神经(NBD)和血管(VBD)表现的贝赫切特病(BD)往往疗效不佳。在日本,英夫利西单抗被批准用于治疗肠道白塞氏病、NBD和VBD;然而,有关其对这些患者的安全性和有效性的证据却很有限。我们开展了一项为期两年的上市后监测,以评估英夫利昔单抗在日本肠道BD、NBD和VBD患者中的安全性和有效性:这项为期 2 年的多中心前瞻性观察研究纳入了所有对常规疗法(如糖皮质激素和免疫抑制剂/免疫调节剂)反应不佳,并在参与研究的医疗机构首次使用英夫利西单抗的肠道 BD、NBD 或 VBD 患者。安全性终点包括不良事件和药物不良反应(ADRs),有效性终点包括总体改善,对于急性NBD患者,有效性终点包括急性发作:2015年10月至2018年8月期间,来自133个医疗中心的255名患者(171名肠道BD患者、49名NBD患者和51名VBD患者;包括16名患有两种疾病类型的患者)接受了英夫利西单抗治疗。发生不良事件、不良反应和严重不良反应的患者分别为 100 例(39.2%)、72 例(28.2%)和 38 例(14.9%);肠道 BD、NBD 和 VBD 组的发生率基本相似。未发现新的安全问题。在最终评估中,68.8%的肠道BD患者病情有所改善,大多数慢性进行性NBD和VBD患者病情没有恶化(分别为100%和91.7%),93.3%的急性NBD患者在观察期间没有新的急性发作:这些结果证实了英夫利西单抗在255例肠道BD、NBD和VBD患者的临床实践中的安全性和有效性。结论:这些结果证实了英夫利昔单抗在255例肠道BD、NBD和VBD患者临床实践中的安全性和有效性,没有新的安全问题。
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引用次数: 0
Efficacy and Safety of Advanced Therapies in Moderately-to-Severely Active Ulcerative Colitis: a Systematic Review and Network Meta-analysis 中度至重度活动性溃疡性结肠炎先进疗法的疗效和安全性:系统综述和网络 Meta 分析。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-15 DOI: 10.1007/s12325-024-03003-8
Axel Dignass, Claire Ainsworth, Susanne Hartz, Niels Dunnewind, Isabel Redondo, Christophe Sapin, Sonja Kroep, Nicholas Halfpenny, Emanuele Arcà, Sami Hoque

Introduction

This study aimed to compare the efficacy and safety of biologics and small molecules for treatment of adults with moderately-to-severely active ulcerative colitis (UC).

Methods

A systematic literature review was conducted to identify randomised controlled trials evaluating approved and emerging targeted therapies for patients with UC. A Bayesian network meta-analysis (NMA) approach was applied. Outcomes assessed included clinical response and remission, endoscopic mucosal healing, and safety.

Results

Thirty studies were included in the NMA following a feasibility assessment comparing approved induction dosing regimens and 22 studies comparing approved maintenance dosing regimens. In the biologic/Janus kinase inhibitor (JAKi)-naïve population, induction studies showed similar clinical response and remission rates across most interventions, with upadacitinib demonstrating significant improvements versus most other interventions. For maintenance studies, mirikizumab demonstrated significant improvements in clinical response and remission versus most other interventions. In the biologic/JAKi-experienced population, no significant differences were observed between most interventions in induction studies, except for significantly improved clinical response and remission for mirikizumab versus adalimumab, and upadacitinib demonstrated significant improvement versus all other interventions. Few differences between active treatments were observed in maintenance studies. In both populations, all active interventions had similar efficacy in terms of endoscopic mucosal healing in both induction and maintenance studies. Regardless of prior treatment exposure, similar rates of serious adverse events were seen across all active interventions in the induction period.

Conclusion

Among the available interventions, owing to its favourable efficacy and safety profile, mirikizumab has a relevant role in the long-term treatment of UC.

简介本研究旨在比较生物制剂和小分子药物治疗中度至重度活动性溃疡性结肠炎(UC)成人患者的疗效和安全性:方法: 我们进行了系统性文献综述,以确定对已获批准的和新出现的治疗溃疡性结肠炎患者的靶向疗法进行评估的随机对照试验。采用贝叶斯网络荟萃分析(NMA)方法。评估的结果包括临床反应和缓解、内镜下粘膜愈合以及安全性:在对已获批准的诱导给药方案和已获批准的维持给药方案分别进行可行性评估后,30 项研究和 22 项研究被纳入 NMA。在生物制剂/破伤风激酶抑制剂(JAKi)未获批准的人群中,大多数干预措施的诱导研究显示出相似的临床反应和缓解率,其中达达替尼与大多数其他干预措施相比有显著改善。在维持治疗研究中,米利珠单抗与其他大多数干预措施相比,临床反应和缓解率均有显著改善。在有生物制剂/JAKi经验的人群中,除了米利珠单抗与阿达木单抗相比在临床反应和缓解方面有明显改善外,在诱导研究中大多数干预措施之间没有观察到显著差异,而达达替尼与所有其他干预措施相比有明显改善。在维持治疗研究中,几乎没有观察到活性疗法之间的差异。在这两种人群中,在诱导和维持研究中,所有积极干预措施在内镜粘膜愈合方面都具有相似的疗效。无论之前是否接受过治疗,在诱导期,所有积极干预措施的严重不良反应发生率相似:结论:在现有的干预措施中,米利珠单抗因其良好的疗效和安全性,在UC的长期治疗中具有重要作用。
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引用次数: 0
Recommendations for Interchangeability in a Growing Biosimilar Market in Latin America 关于在拉丁美洲不断增长的生物仿制药市场中实现互换性的建议。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-09 DOI: 10.1007/s12325-024-02990-y
Gilberto Castañeda-Hernández, Manuel Antonio Espinoza, Luis Eduardo Pino, Mariana Rico-Restrepo, Bianca Schiavetti, Enrique Terán, Valderilio Feijo Azevedo

Background

Biosimilars offer significant advantages for improving access to biologic treatments in Latin America. However, their uptake has been slow due to misconceptions, regulatory uncertainties, and inadequate pharmacovigilance.

Objective

To address these issues, Americas Health Foundation convened a multidisciplinary panel of regional experts in biosimilar use and interchangeability from Latin America. The panel assessed the current landscape and recommended steps to enhance access.

Results

Key recommendations include strengthening biosimilar regulations, ensuring transparent enforcement, implementing robust pharmacovigilance, and promoting collaboration among stakeholders to educate about the safety, efficacy, and economic advantages of biosimilars and their interchangeability.

Conclusions

By embracing biosimilars and interchangeability, Latin American countries can expand patient access, foster competition, diversify treatment sources, and enhance the sustainability of their healthcare systems. However, achieving these goals requires addressing knowledge gaps and biases among healthcare providers, patients, regulators, and government agencies. This can be accomplished through clear communication and the use of real-world evidence.

背景:生物仿制药在改善拉丁美洲生物治疗的可及性方面具有显著优势。然而,由于误解、监管不确定性和药物警戒不足等原因,生物仿制药的使用一直进展缓慢:为了解决这些问题,美洲健康基金会召集了一个由拉丁美洲生物仿制药使用和互换性方面的地区专家组成的多学科小组。该小组评估了目前的状况,并提出了提高可及性的建议:主要建议包括加强生物仿制药法规、确保透明执法、实施强有力的药物警戒、促进利益相关者之间的合作,以宣传生物仿制药的安全性、有效性和经济优势及其互换性:通过接受生物仿制药和互换性,拉美国家可以扩大患者就医途径,促进竞争,使治疗来源多样化,并增强其医疗保健系统的可持续性。然而,实现这些目标需要解决医疗服务提供者、患者、监管者和政府机构之间的知识差距和偏见。这可以通过清晰的沟通和使用真实世界的证据来实现。
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引用次数: 0
Reduced Sense of Smell in Patients with Severe Chronic Rhinosinusitis and its Implications for Diagnosis and Management: A Narrative Review 严重慢性鼻窦炎患者嗅觉减退及其对诊断和管理的影响:叙述性综述。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-09 DOI: 10.1007/s12325-024-02984-w
Zachary M. Soler, Scott Nash, Andrew P. Lane, Zara M. Patel, Stella E. Lee, Wytske J. Fokkens, Mark Corbett, Juby A. Jacob-Nara, Harry Sacks

Reduced sense of smell is a common symptom in patients with chronic rhinosinusitis (CRS). Although it is often under-diagnosed by healthcare providers, reduced sense of smell can have a substantial negative impact on patient’s quality of life as measured by health-related quality of life (HRQoL) assessments and patient-reported outcomes. This narrative review describes current smell loss diagnosis and management guidelines in CRS, and the relationship between smell loss and CRS. Reduced sense of smell can be an indication of CRS disease severity in patients with (CRSwNP) and without nasal polyps (CRSsNP), and recovery of smell can be an indicator of successful CRS treatment. The current first-line therapeutic options for smell loss are intranasal corticosteroids and nasal irrigation, and second-line therapeutic options include systemic steroids and surgery. Shared decision-making between patient, caregiver, and healthcare provider is important when choosing the most appropriate CRS treatment option. Emerging biologic therapies that target type 2 inflammation signaling pathways, such as dupilumab, omalizumab, and mepolizumab, have been shown to improve smell and taste in randomized controlled trials of patients with CRSwNP.

A graphical abstract and video abstract are available with this article.

嗅觉减退是慢性鼻炎(CRS)患者的常见症状。虽然医疗服务提供者往往对其诊断不足,但通过健康相关生活质量(HRQoL)评估和患者报告结果来衡量,嗅觉减退会对患者的生活质量产生严重的负面影响。这篇叙事性综述介绍了当前 CRS 的嗅觉减退诊断和管理指南,以及嗅觉减退与 CRS 之间的关系。嗅觉减退可作为有鼻息肉(CRSwNP)和无鼻息肉(CRSsNP)患者CRS疾病严重程度的指标,而嗅觉恢复可作为CRS治疗成功的指标。目前,嗅觉丧失的一线治疗方案是鼻内皮质类固醇和鼻腔冲洗,二线治疗方案包括全身性类固醇和手术。在选择最合适的 CRS 治疗方案时,患者、护理人员和医疗服务提供者之间的共同决策非常重要。在针对 CRSwNP 患者的随机对照试验中,针对 2 型炎症信号通路的新兴生物疗法(如杜匹单抗、奥马珠单抗和美泊珠单抗)已被证明可以改善嗅觉和味觉。
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引用次数: 0
Patient and Healthcare Provider Perspectives on the Pathway to Diagnosis of Seborrheic Dermatitis in the United States 美国患者和医护人员对脂溢性皮炎诊断途径的看法。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-09-30 DOI: 10.1007/s12325-024-02986-8
Raj Chovatiya, Meredith T. Polaskey, Lakshi Aldredge, Candrice Heath, Moises Acevedo, David H. Chu, Diane Hanna, Melissa S. Seal, Matthew Zirwas

Introduction

Seborrheic dermatitis (SD) is a common, chronic inflammatory skin condition associated with significant impact on quality of life, yet its etiology and pathophysiology are not well understood. With significant impact on patients’ quality of life, understanding the diagnostic pathway from the perspectives of patient and healthcare providers (HCPs) is crucial.

Methods

An online survey was developed and administered in conjunction with the Harris Poll to gain insight into patient and HCP perspectives about SD diagnosis and management from December 2021 to January 2022.

Results

Most patients were unaware of SD before their diagnosis (71%) and experienced difficulty finding information online (56%). Patients delayed seeking medical attention for SD by an average of 3.6 years, with most patients feeling their symptoms did not require medical attention (63%), a perception that HCPs correctly anticipated. Additionally, most patients (58%) reported embarrassment discussing their SD symptoms with HCPs, a factor HCPs underestimated. HCPs also underestimated the percentage of patients self-reporting moderate-severity SD. Patients preferred dermatology HCPs for SD treatment (79%), and reported visiting an average of 2.3 different HCPs, with 75% of patients seeing more than one provider.

Conclusion

These insights highlight the complexities in the diagnostic and management pathways of SD and underscore the need for a more nuanced understanding and approach in addressing the condition.

Infographic available for this article.

Infographic

简介:脂溢性皮炎(SD)是一种常见的慢性炎症性皮肤病,对患者的生活质量有很大影响,但人们对其病因和病理生理学还不甚了解。由于该病对患者的生活质量有重大影响,因此从患者和医疗服务提供者(HCPs)的角度了解诊断途径至关重要:方法:2021 年 12 月至 2022 年 1 月,我们与哈里斯民意调查(Harris Poll)联合开发并实施了一项在线调查,以深入了解患者和医护人员对 SD 诊断和管理的看法:大多数患者在确诊前不了解 SD(71%),并且在网上查找信息时遇到困难(56%)。患者因 SD 而推迟就医的时间平均为 3.6 年,大多数患者认为他们的症状不需要就医(63%),而保健医生正确地预见到了这一点。此外,大多数患者(58%)表示与保健医生讨论他们的 SD 症状时会感到尴尬,而保健医生低估了这一因素。保健医生还低估了自我报告为中度严重 SD 的患者比例。患者在接受 SD 治疗时首选皮肤科保健医生(79%),并称平均看了 2.3 位不同的保健医生,其中 75% 的患者看了一位以上的保健医生:这些见解凸显了 SD 诊断和管理途径的复杂性,并强调了对该疾病进行更细致入微的了解和治疗的必要性。本文附有信息图表。信息图表。
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引用次数: 0
Efficacy of Ixekizumab in Chinese Patients with Moderate-to-Severe Psoriasis and Special Body Area Involvement: Sub-analysis of a Randomized, Double-Blind, Multicenter Phase 3 Study. 伊昔单抗对中重度银屑病和特殊体质患者的疗效:一项随机、双盲、多中心 3 期研究的子分析。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-09-30 DOI: 10.1007/s12325-024-02976-w
Xia Li, Yangfeng Ding, Chunlei Zhang, Yan Lu, Fuqiu Li, Weili Pan, Shuping Guo, Jinnan Li, Bilian Zhao, Jie Zheng

Introduction: Special body area involvement is common in psoriasis and can be challenging to treat. We investigated the efficacy of ixekizumab (IXE) in Chinese patients with moderate-to-severe psoriasis and fingernail, scalp, or palmoplantar involvement.

Methods: A post-hoc sub-analysis of a phase 3 trial, in which patients were randomized to receive placebo, IXE 80 mg every 2 (IXE Q2W) or 4 (IXE Q4W) weeks. At Week 12, patients classified as IXE responders [static Physician's Global Assessment (sPGA) score of 0 or 1 [0,1)] were re-randomized (2:1) to IXE Q4W or placebo until Week 60. Efficacy was assessed by body-region specific parameters including Nail Psoriasis Severity Index (NAPSI), Psoriasis Scalp Severity Index (PSSI), and Palmoplantar Psoriasis Area Severity Index (PPASI).

Results: Of 438 patients, 99.1% (434) had ≥ 1 special area involvement [fingernail (76.5%, 335), scalp (97.3%, 426), palmoplantar (27.9%, 122)]. Significantly greater improvements from baseline in NAPSI score were observed with IXE Q4W and Q2W at Week 12 versus placebo (p < 0.001 for both). These improvements were further increased and sustained over 60 weeks in IXE Q4W and Q2W responders who were re-randomized to IXE Q4W, who achieved a 77.9% and 89.7% improvement from baseline, respectively, at Week 60. Significantly higher proportions of patients receiving IXE Q4W and Q2W achieved NAPSI 50 at Week 12 versus placebo (44.4%, 36.6% vs. 14.1%; p < 0.001 and < 0.01, respectively). Similarly, significantly higher proportions of patients receiving IXE Q4W and Q2W achieved PSSI 100 and PPASI 100 at Week 12 versus placebo (60.6% and 65.1% vs. 1.2%, and 67.4%, 84.3% vs. 21.4%, respectively; p < 0.001 for all comparisons). Improvements across all outcomes were sustained in patients re-randomized to IXE Q4W until Week 60.

Conclusion: IXE led to a rapid onset of action and sustained efficacy over 60 weeks in Chinese patients with moderate-to-severe psoriasis and special body area involvement.

Clinicaltrials: gov identifier, NCT03364309.

简介:特殊身体部位受累是银屑病的常见症状,治疗起来具有挑战性。我们研究了ixekizumab(IXE)对中国中重度银屑病患者以及指甲、头皮或掌跖受累患者的疗效:对一项三期试验进行事后子分析,患者被随机分配接受安慰剂、IXE 80 毫克,每两周一次(IXE Q2W)或四周一次(IXE Q4W)。第12周时,被归类为IXE应答者(静态医生总体评估(sPGA)评分为0或1 [0,1))的患者被重新随机分配(2:1)至IXE Q4W或安慰剂,直至第60周。疗效通过身体区域特定参数进行评估,包括指甲银屑病严重程度指数(NAPSI)、头皮银屑病严重程度指数(PSSI)和掌跖银屑病面积严重程度指数(PPASI):在 438 名患者中,99.1%(434 人)有≥ 1 个特殊部位受累[指甲(76.5%,335 人)、头皮(97.3%,426 人)、掌跖(27.9%,122 人)]。与安慰剂相比,IXE Q4W 和 Q2W 在第 12 周的 NAPSI 评分与基线相比有显著提高(p 结论:IXE 在第 12 周的 NAPSI 评分与基线相比有显著提高:IXE对中重度银屑病和特殊身体部位受累的中国患者起效迅速,疗效持续60周。
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引用次数: 0
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