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患者对雷珠单抗经Port 给药系统与玻璃体内注射治疗新生血管性年龄相关性黄斑变性的偏好和治疗满意度:一项随机临床试验。

Patient Preference and Treatment Satisfaction With a Port Delivery System for Ranibizumab vs Intravitreal Injections in Patients With Neovascular Age-Related Macular Degeneration: A Randomized Clinical Trial.

机构信息

Retinal Consultants, Sacramento, California.

Genentech, Inc, South San Francisco, California.

出版信息

JAMA Ophthalmol. 2022 Aug 1;140(8):771-778. doi: 10.1001/jamaophthalmol.2022.1091.


DOI:10.1001/jamaophthalmol.2022.1091
PMID:35708706
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9204622/
Abstract

IMPORTANCE: The port delivery system (PDS) with ranibizumab has demonstrated noninferior and equivalent efficacy compared with monthly intravitreal injections of ranibizumab, an anti-vascular endothelial growth factor (VEGF) agent, in patients with neovascular age-related macular degeneration (nAMD), but evaluating patient preference is important to help inform clinical decision-making. OBJECTIVE: Evaluate treatment satisfaction for ranibizumab delivered via PDS vs intravitreal injections as well as patient preference among those assigned to PDS. DESIGN, SETTING, AND PARTICIPANTS: Archway was a phase 3 randomized active-comparator open-label clinical trial conducted at 78 sites in the US. Patients 50 years and older with nAMD diagnosed within 9 months of screening with a documented response to anti-VEGF therapy were included. Of 619 patients screened, 418 were enrolled; 415 were included in the primary analysis and 234 were included in the secondary exploratory analysis. The Archway study ran from September 12, 2019, through primary readout on May 22, 2020. INTERVENTIONS: Patients were randomized 3:2 to PDS with ranibizumab, 100 mg/mL, with fixed refill exchanges every 24 weeks or intravitreal ranibizumab injections, 0.5 mg, every 4 weeks. MAIN OUTCOMES AND MEASURES: Treatment satisfaction was measured using the Macular Disease Treatment Satisfaction Questionnaire in the PDS and intravitreal injection arms at week 40. Patient preference was assessed using the content-validated PDS Patient Preference Questionnaire (PPPQ), which measured the proportion of patients in the PDS arm with monthly monitoring who preferred treatment with the PDS at week 40 over previous intravitreal injections or concurrent fellow-eye injections. Both outcomes were exploratory end points. RESULTS: The mean (SD) age of participants at baseline was 75.0 (7.9) years; 234 participants (59%) were women and 162 (41%) were men. At week 40, differences in overall treatment satisfaction scores were minimal for the PDS and intravitreal injection arms (mean, 68.0; 95% CI, 67.4-68.6; n = 237 and mean, 66.1; 95% CI, 64.9-67.3; n = 159, respectively; difference, 1.9; 95% CI, 0.7-3.1). A total of 234 of 248 patients (94.4%) in the PDS arm were included in the PPPQ analysis. At week 40, almost all patients in the PDS arm preferred treatment via PDS (218 of 234 [93.2%]) vs previous intravitreal injections (3 of 234 [1.3%]), including 172 of 234 (73.5%) with a very strong preference for the PDS. In patients who received concurrent fellow-eye injections (n = 78), 72 (92.3%) preferred the PDS. CONCLUSIONS AND RELEVANCE: Although PDS treatment was preferred by almost all patients assigned to PDS over previous intravitreal injections, both delivery methods have high treatment satisfaction. These findings provide further evidence for the PDS as a meaningful alternative treatment option for patients with nAMD. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT03677934.

摘要

重要性:与每月玻璃体腔内注射抗血管内皮生长因子(VEGF)药物雷珠单抗相比,雷珠单抗端口输送系统(PDS)在新生血管性年龄相关性黄斑变性(nAMD)患者中显示出非劣效性和等效性,但评估患者的偏好对于帮助制定临床决策很重要。

目的:评估与玻璃体腔内注射相比,接受 PDS 治疗的雷珠单抗的治疗满意度,以及在接受 PDS 治疗的患者中的患者偏好。

设计、地点和参与者:Archway 是一项在美国 78 个地点进行的 3 期随机活性对照开放标签临床试验。纳入了年龄在 50 岁及以上、在筛选后 9 个月内被诊断为 nAMD、有抗 VEGF 治疗反应记录的患者。在 619 名筛选的患者中,有 418 名患者入组;415 名患者纳入主要分析,234 名患者纳入次要探索性分析。Archway 研究于 2019 年 9 月 12 日开始,于 2020 年 5 月 22 日进行主要结果分析。

干预措施:患者按 3:2 的比例随机分为 PDS 组(100mg/ml 雷珠单抗,每 24 周固定更换)和玻璃体腔注射组(0.5mg 雷珠单抗,每 4 周)。

主要观察指标:在第 40 周时,使用黄斑疾病治疗满意度问卷(Macular Disease Treatment Satisfaction Questionnaire)评估 PDS 组和玻璃体腔注射组的治疗满意度。使用内容验证的 PDS 患者偏好问卷(PDS Patient Preference Questionnaire,PPPQ)评估患者偏好,该问卷衡量了在第 40 周时,每月监测的 PDS 组中有多少患者更喜欢使用 PDS 治疗,而不是之前的玻璃体腔注射或同时对侧眼注射。这两个结果都是探索性的终点。

结果:参与者的平均(SD)年龄为 75.0(7.9)岁;234 名参与者(59%)为女性,162 名(41%)为男性。在第 40 周时,PDS 组和玻璃体腔注射组的总体治疗满意度评分差异很小(分别为 68.0,95%置信区间[CI]:67.4-68.6;n=237;66.1,95%CI:64.9-67.3;n=159;差异:1.9,95%CI:0.7-3.1)。共有 248 名 PDS 组患者中的 234 名(94.4%)患者纳入 PPPQ 分析。在第 40 周时,几乎所有的 PDS 组患者(234 名中的 218 名[93.2%])都更喜欢通过 PDS 进行治疗,而不是之前的玻璃体腔注射(234 名中的 3 名[1.3%]),包括 234 名中的 172 名(73.5%)非常强烈地偏好 PDS。在接受同时对侧眼注射的患者(n=78)中,有 72 名(92.3%)患者更喜欢 PDS。

结论和相关性:尽管几乎所有被分配到 PDS 的患者都更倾向于使用 PDS 治疗,而不是之前的玻璃体腔注射,但这两种治疗方法的治疗满意度都很高。这些发现为 PDS 作为 nAMD 患者的一种有意义的替代治疗选择提供了进一步的证据。

试验注册:ClinicalTrials.gov 标识符:NCT03677934。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/e5248b1f8b90/jamaophthalmol-e221091-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/eb2cc08bcd8a/jamaophthalmol-e221091-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/cfcca0072da5/jamaophthalmol-e221091-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/566ec7e05e13/jamaophthalmol-e221091-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/e5248b1f8b90/jamaophthalmol-e221091-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/eb2cc08bcd8a/jamaophthalmol-e221091-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/cfcca0072da5/jamaophthalmol-e221091-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/566ec7e05e13/jamaophthalmol-e221091-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b31c/9204622/e5248b1f8b90/jamaophthalmol-e221091-g004.jpg

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[2]
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[3]
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[4]
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[5]
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[6]
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