Department of Neuroscience, St. Agostino-Estense Hospital, Azienda Ospedaliero Universitaria di Modena, University of Modena and Reggio Emilia, Via Pietro Giardini n. 1355, 41100, Modena, Italy.
Department of Neuroscience, IRCCS-Istituto di Ricerche Farmacologiche Mario Negri, Milan, Italy.
J Neurol. 2018 Apr;265(4):817-827. doi: 10.1007/s00415-018-8778-y. Epub 2018 Feb 5.
In this prospective population-based registry study on ALS survival, we investigated the role of riluzole treatment, together with other clinical factors, on the prognosis in incident ALS cases in Emilia Romagna Region (ERR), Italy.
A registry for ALS has been collecting all incident cases in ERR since 2009. Detailed clinical data from all patients diagnosed with ALS between 1.1.2009 and 31.12.2014 have been analyzed for this study, with last follow up date set at 31.12.2015.
During the 6 years of the study, there were 681 incident cases with a median tracheostomy-free survival of 40 months (95% CI 36-44) from onset and of 26 months (95% CI 24-30) from diagnosis; 573 patients (84.14%) were treated with riluzole, 207 (30.39%) patients underwent gastrostomy, 246 (36.12%) non invasive ventilation, and 103 (15.15%) invasive ventilation. Patients who took treatment for ≥ 75% of disease duration from diagnosis had a median survival of 29 months compared to 18 months in patients with < 75% treatment duration. In multivariable analysis, factors independently influencing survival were age at onset (HR 1.04, 95% CI 1.02-1.05, p < 0.001), dementia (HR 1.56, 95% CI 1.05-2.32, p = 0.027), degree of diagnostic certainty (HR 0.88, 95% CI 0.78-0.98, p = 0.021), gastrostomy (HR 1.46, 95% CI 1.14-1.88, p = 0.003), NIV (HR 1.43, 95% CI 1.12-1.82, p = 0.004), and weight loss at diagnosis (HR 1.05, 95% CI 1.03-1.07, p < 0.001), diagnostic delay (HR 0.98, 95% CI 0.97-0.99, p = 0.004), and % treatment duration (HR 0.98, 95% CI 0.98-0.99, p < 0.001).
Independently from other prognostic factors, patients who received riluzole for a longer period of time survived longer, but further population based studies are needed to verify if long-tem use of riluzole prolongs survival.
在这项针对肌萎缩侧索硬化症（ALS）生存的前瞻性基于人群的登记研究中，我们调查了利鲁唑治疗与其他临床因素共同对意大利艾米利亚-罗马涅地区（ERR）ALS 新发病例预后的作用。
自 2009 年以来，一项 ALS 登记处一直在收集 ERR 中的所有新发病例。对所有在 2009 年 1 月 1 日至 2014 年 12 月 31 日期间被诊断为 ALS 的患者的详细临床数据进行了分析，随访截止日期为 2015 年 12 月 31 日。
在研究的 6 年期间，有 681 例新发病例，从发病开始的中位无气管造口术生存时间为 40 个月（95%CI 36-44），从诊断开始的中位生存时间为 26 个月（95%CI 24-30）；573 例（84.14%）患者接受了利鲁唑治疗，207 例（30.39%）患者接受了胃造口术，246 例（36.12%）患者接受了无创通气，103 例（15.15%）患者接受了有创通气。从诊断开始接受≥75%治疗时间的患者中位生存时间为 29 个月，而接受治疗时间<75%的患者中位生存时间为 18 个月。多变量分析显示，影响生存的独立因素为发病年龄（HR 1.04，95%CI 1.02-1.05，p<0.001）、痴呆（HR 1.56，95%CI 1.05-2.32，p=0.027）、诊断确定性程度（HR 0.88，95%CI 0.78-0.98，p=0.021）、胃造口术（HR 1.46，95%CI 1.14-1.88，p=0.003）、无创通气（HR 1.43，95%CI 1.12-1.82，p=0.004）和诊断时体重减轻（HR 1.05，95%CI 1.03-1.07，p<0.001）、诊断延迟（HR 0.98，95%CI 0.97-0.99，p=0.004）和治疗时间比例（HR 0.98，95%CI 0.98-0.99，p<0.001）。
除其他预后因素外，接受利鲁唑治疗时间较长的患者生存时间更长，但需要进一步的基于人群的研究来验证长期使用利鲁唑是否能延长生存时间。
