Han Moon, Raymond Jaime, Larson Theodore C, Mehta Paul, Horton D Kevin
Office of Innovation and Analytics, Agency for Toxic Substances and Disease Registry/Centers for Disease Control and Prevention, 4770 Buford Hwy NE, Atlanta, GA, 30341, USA.
J Racial Ethn Health Disparities. 2024 Jul 8. doi: 10.1007/s40615-024-02047-4.
To characterize the participant demographics in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database compared with the web-portal National Amyotrophic Lateral Sclerosis (ALS) Registry (the Registry).
Demographics and ALS symptom information were compared between the self-reported registrant data in the Registry web portal (2010-2021) and the latest available PRO-ACT data (updated August 2022), which is a collection of clinical trials data.
Greater percentages of younger (≤ 59 years old) but smaller percentages of older (60 + years old) participants were represented in PRO-ACT compared to Registry. Enrollment for minority race groups was greater in the Registry portal data, but race information was largely missing/unknown in PRO-ACT database. Median age at the time of diagnosis and age at the time of symptom onset were significantly higher for Registry enrollees compared to the participants of PRO-ACT. Symptom onset sites were similarly reported, but duration between self-noted symptom onset and diagnosis was slight, but significantly longer for the Registry enrollees (11 vs. 9 months). Hispanic were as likely as non-Hispanic to participate in research studies, based on the Registry data.
There was a notable difference in the age distribution and minority representation of enrollees between the PRO-ACT and Registry study populations. Age distribution in the PRO-ACT database skewed to a younger and less diverse cohort. Despite the clinical heterogeneity and complex disease mechanism of ALS, identifying the underrepresented demographic niche in the PRO-ACT and Registry study populations can help improve patient participation and criteria for patient selection to enhance generalizability.
与门户网站全国肌萎缩侧索硬化症（ALS）登记处（登记处）相比，对汇总资源开放获取ALS临床试验（PRO-ACT）数据库中的参与者人口统计学特征进行描述。
比较登记处门户网站（2010 - 2021年）自我报告的登记人数据与最新可用的PRO-ACT数据（2022年8月更新）之间的人口统计学和ALS症状信息，后者是一个临床试验数据集合。
与登记处相比，PRO-ACT中年龄较小（≤59岁）的参与者百分比更高，但年龄较大（60岁及以上）的参与者百分比更低。登记处门户网站数据中少数种族群体的登记人数更多，但PRO-ACT数据库中种族信息大多缺失/未知。与PRO-ACT的参与者相比，登记处登记者的诊断时中位年龄和症状出现时年龄显著更高。症状出现部位的报告相似，但自我记录的症状出现到诊断之间的持续时间略有不同，登记处登记者的持续时间明显更长（11个月对9个月）。根据登记处数据，西班牙裔参与研究的可能性与非西班牙裔相同。
PRO-ACT和登记处研究人群中登记者的年龄分布和少数群体代表性存在显著差异。PRO-ACT数据库中的年龄分布偏向于更年轻且多样性较低的队列。尽管ALS存在临床异质性和复杂的疾病机制，但确定PRO-ACT和登记处研究人群中代表性不足的人口细分群体有助于提高患者参与度和患者选择标准，以增强普遍性。
