非霍奇金淋巴瘤患者的真实世界进展和反应不足变量及相关终点分析。

IF 3.4 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL

Advances in Therapy Pub Date : 2025-03-03 DOI:10.1007/s12325-025-03143-5

Madeline Richey, Christina Fullerton, Qianyi Zhang, Tori Williams, Douglas Donnelly, Hannah C. Wise, Aaron Dolor, Niquelle Wadé, Kelly Magee

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引用次数: 0

摘要

简介：评估肿瘤负荷变化的方法在临床试验和常规临床护理环境中有所不同，这为设计从电子健康记录（EHR）数据中获取临床结果的新方法提供了独特的机会。我们采用了先前建立的实体瘤方法，并对其进行了修改，以捕获非霍奇金淋巴瘤（NHL）的真实进展（rwP）和真实反应不足（rwIR）事件。方法：本研究使用了一个全国性的ehr数据库。第一阶段评估了rwP/rwIR方法在滤泡性淋巴瘤（FL）和弥漫性大B细胞淋巴瘤（DLBCL）患者中的应用。第二阶段在更大的套细胞淋巴瘤（MCL）患者队列中评估该方法。通过对事件发生、日期和类型、临床医生评估和源数据完整性、rwP/rwIR事件后的下游事件、事件时间分析（真实世界无进展[rwPFS]和无事件[rwEFS]生存）及其与真实世界总生存（rwOS）的相关性来评估性能。结果：研究共纳入6162例NHL患者，其中FL患者672例(中位年龄64岁；女性,49%;男性，51%)，405例DLBCL患者(中位年龄70岁；女性,42%;男性，58%)和5085例MCL患者(中位年龄69岁；女性,27%;男性,73%)。所有队列中事件发生的摘要间一致性为96-97%，30天内事件日期一致性为85-89%。发生rwP/rwIR事件的患者比例在FL队列中为26%，在DLBCL队列中为27%，在MCL队列中为42%。在58%的FL队列、63%的DLBCL队列和73%的MCL队列中观察到临床相关的下游事件。在MCL队列中，中位rwPFS为34.5个月，中位rwEFS为32.2个月。实际操作系统与rwPFS（85%）的相关性比与rwEFS（80%）的相关性更强。结论：nhl特异性rwP/rwIR方法可行、可靠、可扩展。观察到的抽象者之间的一致性和rwP/rwIR事件频率显示了NHL队列的适用性。在现实世界人群中，终点分析和与rwOS的相关性证明了这种方法的临床相关性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Analysis of Real-World Progression and Insufficient Response Variables and Related Endpoints Among Patients with Non-Hodgkin Lymphoma

Introduction

Methods for assessing change in tumor burden differ between clinical trial and routine clinical care settings, presenting a unique opportunity to design novel methods to capture clinical outcomes from electronic health record (EHR) data. We adapted a previously established approach for solid tumors and modified it to capture real-world progression (rwP) and real-world insufficient response (rwIR) events in non-Hodgkin lymphoma (NHL).

Methods

This study used a nationwide EHR-derived deidentified database. The first phase assessed the rwP/rwIR approach in patients with follicular lymphoma (FL) and diffuse large B cell lymphoma (DLBCL). The second phase assessed the approach in a larger cohort of patients with mantle cell lymphoma (MCL). Performance was assessed through inter-abstractor agreement on event occurrence, date, and type, clinician assessment and source data completeness, downstream events after an rwP/rwIR event, and time-to-event analyses (real-world progression-free [rwPFS] and event-free [rwEFS] survival) and their correlation with real-world overall survival (rwOS).

Results

A total of 6162 patients with NHL were included in the study, comprising 672 patients with FL (median age, 64 years; female, 49%; male, 51%), 405 patients with DLBCL (median age, 70 years; female, 42%; male, 58%), and 5085 patients with MCL (median age, 69 years; female, 27%; male, 73%). Inter-abstractor agreement among all cohorts was 96–97% for event occurrence and 85–89% for event date within 30 days. The proportion of patients with an rwP/rwIR event was 26% in the FL cohort, 27% in the DLBCL cohort, and 42% in the MCL cohort. Clinically relevant downstream events were observed in 58% of the FL cohort, 63% of the DLBCL cohort, and 73% of the MCL cohort. In the MCL cohort, median rwPFS was 34.5 months, and median rwEFS was 32.2 months. Real-world OS correlated more strongly with rwPFS (85%) than with rwEFS (80%).

Conclusions

The NHL-specific rwP/rwIR approach is feasible, reliable, and scalable. Observed inter-abstractor agreement and rwP/rwIR event frequency show applicability across NHL cohorts. Endpoint analyses and correlations with rwOS in a real-world population demonstrate the clinical relevance of this approach.

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来源期刊

Advances in Therapy 医学-药学

CiteScore

7.20

自引率

2.60%

发文量

353

审稿时长

6-12 weeks

期刊介绍： Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.