Dorst Johannes, Dreyhaupt Jens, Wernecke Deborah, Weiland Ulrike, Parlak Özlem, Wiesenfarth Maximilian, Elmas Zeynep, Herrmann Christine, Bäzner Hansjörg, Boertlein Axel, Dempewolf Silke, Foerch Christian, Hecht Martin, Kohler Andreas, Opherk Christian, Althaus Katharina, Clauer-Bredt Monika, Lindner Alfred, Ruf Wolfgang, Brenner David, Witzel Simon, Peter Raphael S, Schuster Joachim, Ludolph Albert C, Rosenbohm Angela, Nagel Gabriele
Department of Neurology, University of Ulm, Ulm, Germany.
German Center for Neurodegenerative Diseases (DZNE), Ulm, Germany.
Eur J Neurol. 2025 Apr;32(4):e70137. doi: 10.1111/ene.70137.
Over the past years, some studies in amyotrophic lateral sclerosis (ALS) have provided heterogeneous findings regarding demographic and clinical data as well as the impact of various prognostic factors. It is well known that these inconsistencies might be caused by a selection bias in hospital-based data sets. In this study, we sought to further characterize this selection bias.
We compared hospital-based data from the ALS center at Ulm University (UC; n = 3833; 1997-2021) with the population-based ALS registry Swabia (SR; n = 852; 2010-2020).
Patients from UC were younger (age of onset 60.9 [IQR 52.4-68.9] vs. 65.0 [57.0-72.7]), had a higher share of males (60.5% vs. 56.3%), a longer diagnostic delay (10.5 [IQR 6.4-18.4] months vs. 6.9 [IQR 3.4-12.1] months), a higher prevalence of the "definite" category according to El Escorial diagnostic criteria (60.9% vs. 11.2%), a higher share of familial cases (12.9% vs. 6.3%), a slower progression rate (points of ALS functional rating scale revised lost per month -0.54 [IQR -1.02 to -0.28] vs. -0.79 [IQR -1.47 to -0.43]), and (among all deceased patients) a higher share of percutaneous endoscopic gastrostomy (26.7% vs. 17.7%) and non-invasive ventilation (34.3% vs. 25.3%).
The observed differences likely indicate a selection bias in hospital-based data, which may be attributed, among others, to the willingness to travel large distances to a specialized center, the desire to participate in clinical studies, and the attitude toward life-prolonging measures. These differences must be considered when interpreting and generalizing study results from hospital-based populations.
在过去几年中，一些关于肌萎缩侧索硬化症（ALS）的研究在人口统计学和临床数据以及各种预后因素的影响方面得出了不一致的结果。众所周知，这些不一致可能是由基于医院的数据集的选择偏倚引起的。在本研究中，我们试图进一步描述这种选择偏倚的特征。
我们将乌尔姆大学ALS中心（UC；n = 3833；1997 - 2021年）的基于医院的数据与基于人群的施瓦本ALS登记处（SR；n = 852；2010 - 2020年）的数据进行了比较。
来自UC的患者更年轻（发病年龄60.9[四分位间距52.4 - 68.9]岁对65.0[57.0 - 72.7]岁），男性比例更高（60.5%对56.3%），诊断延迟更长（10.5[四分位间距6.4 - 18.4]个月对6.9[四分位间距3.4 - 12.1]个月），根据埃尔埃斯科里亚尔诊断标准“确诊”类别的患病率更高（60.9%对11.2%），家族性病例比例更高（12.9%对6.3%），疾病进展速度更慢（ALS功能评定量表修订版每月丧失的分数 - 0.54[四分位间距 - 1.02至 - 0.28]对 - 0.79[四分位间距 - 1.47至 - 0.43]），并且（在所有死亡患者中）经皮内镜下胃造瘘术的比例更高（26.7%对17.7%）以及无创通气的比例更高（34.3%对25.3%）。
观察到的差异可能表明基于医院的数据存在选择偏倚，这可能归因于愿意长途前往专科中心、参与临床研究的意愿以及对延长生命措施的态度等因素。在解释和推广基于医院人群的研究结果时，必须考虑这些差异。
