法利昔单抗对代表性不足患者糖尿病性黄斑水肿的影响

IF 2.3 4区医学 Q1 MEDICINE, GENERAL & INTERNAL

Journal of the National Medical Association Pub Date : 2025-09-01 DOI:10.1016/j.jnma.2025.08.055

Joseph M. Coney MD, FACS, Manuel Amador MD, Jeremiah Brown MS, MD, Matthew A. Cunningham MD, Andres Emanuelli MD, Adrienne W. Scott MD, FASRS, Sofia Milunovich, Ming Yang PhD, Xiao-Yu Lu, Ruby Ha

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引用次数: 0

摘要

糖尿病性黄斑水肿（DME）是糖尿病患者视力丧失的主要原因，在美国黑人和西班牙裔患者中尤为严重。尽管如此，这些患者在临床试验中的代表性不足。(1) ELEVATUM （NCT05224102）是一项4期、开放标签、单臂、多中心研究，评估DME患者对faricimab治疗的反应，传统上在眼科试验中代表性不足。我们报告了美国患者使用ELEVATUM的第一年疗效和安全性结果。MethodsTreatment-naïve DME患者（n=123），自我认定为黑人或非裔美国人，西班牙裔或拉丁美洲人，或美洲原住民/阿拉斯加原住民/夏威夷原住民/太平洋岛民，每4周（Q4W）至第20周，然后Q8W至第52周接受法昔单抗6.0 mg。在第56周对黑人/非裔美国患者、西班牙裔/拉丁裔美国患者和美国总体研究人群进行评估，评估最佳矫正视力（BCVA）和中央子视野厚度（CST）与基线相比的变化，以及糖尿病视网膜病变严重程度量表（DRSS）改善≥2级的患者比例。结果在基线时，西班牙裔/拉丁裔美国患者的BCVA（平均[标准差]:59.2[11.4]个字母）和CST （491.1 [157.2] μm）的数值差于黑人/非洲裔美国患者（62.2[10.2]个字母和467.4[126.0]个μm）和整个美国研究人群（61.1[11.1]个字母和476.9[138.5]个μm）。在第56周，西班牙裔/拉丁裔美国患者、黑人/非裔美国患者和整个美国研究人群中，调整后的平均（95%置信区间）BCVA较基线变化分别为+14.1（9.9,18.2）、+11.3（9.0,13.5）和+12.3（10.2,14.5）个字母。与基线相比，第56周的CST变化在西班牙/拉丁美洲患者、黑人/非裔美国患者和整个美国研究人群中分别为-230.4 (-252.1,-208.6)μm、-193.9 (-215.2,-172.7)μm和-206.3 (-217.0,-195.7)μm。41.5%的西班牙裔/拉丁裔美国患者、17.6%的黑人/非洲裔美国患者和32.1%的美国总体研究人群实现了≥2步的DRSS改善。Faricimab耐受性良好；没有发现新的安全隐患。结论：在美国，对于代表性不足的DME患者，ELEVATUM治疗的第一年结果显示faricimab对视力和CST有显著改善。西班牙/拉丁美洲患者在BCVA、CST和糖尿病视网膜病变疾病严重程度方面的改善最大。Faricimab耐受性良好，安全性与YOSEMITE/RHINE （NCT03622580/NCT03622593）试验一致。

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Effect of Faricimab on Diabetic Macular Edema in Underrepresented Patients

Introduction

Diabetic macular edema (DME) is the leading cause of vision loss among patients with diabetes mellitus and disproportionately affects Black and Hispanic patients in the US. Despite this, these patients are underrepresented in clinical trials.(1) ELEVATUM (NCT05224102) is a phase 4, open-label, single-arm, multicenter study assessing the response to faricimab treatment among patients with DME traditionally underrepresented in ophthalmology trials. We report year 1 efficacy and safety results from ELEVATUM for patients in the US.

Methods

Treatment-naïve patients with DME (n=123), who self-identified as Black or African American, Hispanic or Latin American, or Native American/Alaska Native/Native Hawaiian/Pacific Islander, received faricimab 6.0 mg every 4 weeks (Q4W) to week 20, then Q8W to week 52. Best-corrected visual acuity (BCVA) and central subfield thickness (CST) changes from baseline at week 56, and the proportion of patients with ≥2-step Diabetic Retinopathy Severity Scale (DRSS) improvement, were assessed for Black/African American patients, Hispanic/Latino American patients, and the overall US study population.

Results

At baseline, Hispanic/Latino American patients had numerically worse BCVA (mean [standard deviation]: 59.2 [11.4] letters) and CST (491.1 [157.2] μm) compared with Black/African American patients (62.2 [10.2] letters and 467.4 [126.0] μm) and the overall US study population (61.1 [11.1] letters and 476.9 [138.5] μm). At week 56, adjusted mean (95% confidence interval) BCVA changes from baseline were +14.1 (9.9, 18.2), +11.3 (9.0, 13.5), and +12.3 (10.2, 14.5) letters in Hispanic/Latino American patients, Black/African American patients, and the overall US study population, respectively. Corresponding CST changes from baseline at week 56 were -230.4 (-252.1, -208.6) μm, -193.9 (-215.2, -172.7) μm, and -206.3 (-217.0, -195.7) μm in Hispanic/Latino American patients, Black/African American patients, and the overall US study population, respectively. DRSS improvement of ≥2 steps was achieved by 41.5% of Hispanic/Latino American patients, 17.6% of Black/African American patients, and 32.1% of the overall US study population. Faricimab was well tolerated; no new safety concerns were identified.

Conclusions

Year 1 results from ELEVATUM demonstrated robust vision and CST improvements with faricimab in underrepresented patients with DME in the US. Hispanic/Latino American patients had the greatest improvements in BCVA, CST, and diabetic retinopathy disease severity. Faricimab was well tolerated, with a safety profile consistent with that established in the YOSEMITE/RHINE (NCT03622580/NCT03622593) trials.

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来源期刊

Journal of the National Medical Association 医学-医学：内科

CiteScore

4.80

自引率

3.00%

发文量

139

审稿时长

98 days

期刊介绍： Journal of the National Medical Association, the official journal of the National Medical Association, is a peer-reviewed publication whose purpose is to address medical care disparities of persons of African descent. The Journal of the National Medical Association is focused on specialized clinical research activities related to the health problems of African Americans and other minority groups. Special emphasis is placed on the application of medical science to improve the healthcare of underserved populations both in the United States and abroad. The Journal has the following objectives: (1) to expand the base of original peer-reviewed literature and the quality of that research on the topic of minority health; (2) to provide greater dissemination of this research; (3) to offer appropriate and timely recognition of the significant contributions of physicians who serve these populations; and (4) to promote engagement by member and non-member physicians in the overall goals and objectives of the National Medical Association.