Trial-level factors affecting accrual rate of systemic sclerosis randomized clinical trials

IF 4.6 2区医学 Q1 RHEUMATOLOGY

Seminars in arthritis and rheumatism Pub Date : 2025-05-03 DOI:10.1016/j.semarthrit.2025.152749

Barbara Russo , Iulia-Simona Chirică , Delphine Sophie Courvoisier , Michele Iudici

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引用次数: 0

Abstract

Objectives

To estimate the average time to complete patient enrollment and identify factors associated with accrual rates in systemic sclerosis (SSc) randomized controlled trials (RCTs).

Methods

We searched published SSc-RCTs indexed in PubMed from 2000 to 2024, selecting those with recruitment completed before the COVID-19 pandemic. We recorded key trial features (country, phase, randomization ratio, intervention, blinding, funding source, outcome type) and enrollment year(s). We measured enrollment duration and accrual rate (participants per month). A multivariable negative binomial generalized linear model was used to identify factors associated with accrual rate.

Results

We included 80 studies, mostly single-country (75.0 %) and industry-funded (57.5 %), mainly recruiting in Europe (36.2 %) and North America (22.5 %). In 65 % of studies, both limited and diffuse SSc patients were enrolled. The median sample size was 40.5 patients, with 20 % of RCTs enrolling ≥100 patients. The median recruitment time was 15 months (IQR 9.9 – 30.0), with a median accrual rate of 3.1 (IQR 1.6 - 5.5) participants per month. Recruitment rates varied over time, with faster accrual early in the 2000s and after 2012, and a slower period in between. Multivariable analysis showed that accrual rate was positively associated with skewed randomization, blinding, non-industry funding, international recruitment, and inclusion of both SSc subsets, especially compared to studies involving only dcSSc patients.

Conclusions

Recruiting SSc patients for RCTs has been challenging, with generally slow accrual over the past 20 years and no significant improvement over time.

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影响系统性硬化发生率的试验水平因素随机临床试验

目的估计完成系统性硬化症（SSc）随机对照试验（rct）患者入组的平均时间，并确定与应计率相关的因素。方法检索PubMed检索的2000 - 2024年已发表的ssc - rct，选择在COVID-19大流行前完成招募的ssc - rct。我们记录了关键试验特征（国家、阶段、随机化比例、干预、盲法、资金来源、结果类型）和入组年份。我们测量了入组时间和累计率（每月参与者）。采用多变量负二项广义线性模型来确定与应计费率相关的因素。结果我们纳入了80项研究，主要是单一国家（75.0%）和行业资助（57.5%），主要在欧洲（36.2%）和北美（22.5%）招募。65%的研究纳入了有限和弥漫性SSc患者。中位样本量为40.5例患者，20%的rct入组≥100例患者。中位招募时间为15个月（IQR 9.9 - 30.0），每月中位累积率为3.1 （IQR 1.6 - 5.5）。招聘率随着时间的推移而变化，21世纪初和2012年之后的增长速度较快，而这两者之间的增长速度较慢。多变量分析显示，应计率与偏随机化、盲法、非行业资助、国际招募和纳入两个SSc亚群呈正相关，特别是与仅涉及dcSSc患者的研究相比。招募SSc患者进行随机对照试验是具有挑战性的，在过去的20年里，SSc患者的累积速度通常很慢，并且随着时间的推移没有显著的改善。

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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来源期刊

Seminars in arthritis and rheumatism 医学-风湿病学

CiteScore

9.20

自引率

4.00%

发文量

176

审稿时长

46 days

期刊介绍： Seminars in Arthritis and Rheumatism provides access to the highest-quality clinical, therapeutic and translational research about arthritis, rheumatology and musculoskeletal disorders that affect the joints and connective tissue. Each bimonthly issue includes articles giving you the latest diagnostic criteria, consensus statements, systematic reviews and meta-analyses as well as clinical and translational research studies. Read this journal for the latest groundbreaking research and to gain insights from scientists and clinicians on the management and treatment of musculoskeletal and autoimmune rheumatologic diseases. The journal is of interest to rheumatologists, orthopedic surgeons, internal medicine physicians, immunologists and specialists in bone and mineral metabolism.