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Real-world clinical course of HTLV-1-associated myelopathy/tropical spastic paraparesis (HAM/TSP) in Japan.
Orphanet Journal of Rare Diseases ( IF 3.4 ) Pub Date : 2019-10-21 , DOI: 10.1186/s13023-019-1212-4
Shuntaro Tsutsumi 1 , Tomoo Sato 2 , Naoko Yagishita 2 , Junji Yamauchi 2 , Natsumi Araya 2 , Daisuke Hasegawa 2 , Misako Nagasaka 1, 3 , Ariella L G Coler-Reilly 2 , Eisuke Inoue 4 , Ayako Takata 5 , Yoshihisa Yamano 1, 2
Affiliation  

BACKGROUND As human T-cell leukemia virus type 1 (HTLV-1)-associated myelopathy/tropical spastic paraparesis (HAM/TSP) is a rare chronic neurological disease, large scale studies to collect continuous clinical data have been difficult to conduct. Therefore, the incidence of comorbidities and drug utilization data remain unknown. When conducting trials to develop new drugs in rare disease such as HAM/TSP, historical control data obtained from registry studies would be useful, as cohorts in rare disease tend to be small. Long-term follow-up of patients with a chronic disease can also be challenging. In this study, we addressed the following two goals using registry data on patients (n = 486) enrolled in the Japanese HAM/TSP patient registry "HAM-net" from 2012 to 2016: 1) to clarify the epidemiological information of HAM/TSP such as the incidence of comorbidities and drug utilization and 2) to provide the real-world data on changes in lower limb motor dysfunction. RESULTS In HAM-net-registered patients, common comorbidities were fractures, herpes zoster, and uveitis, with incidences of 55.5, 10.4, and 6.5, respectively, per 1000 person-years. Every year, oral steroid treatment was administered in 48.2-50.7% of the HAM-net-registered patients and interferon-α treatment was used in 2.6-3.5% of patients. The median dose of oral prednisolone was low at 5.0 mg/day. The incidence of fractures and herpes zoster tended to be higher in the steroid-treated group than in the untreated group (fractures: 61.0 vs. 48.3, herpes zoster: 12.7 vs. 8.8, per 1000 person-years). The analysis of chronological change in Osame motor disability score (OMDS) indicated that the mean change in OMDS was + 0.20 [95% confidence intervals (CI): 0.14-0.25] per year in the one-year observation group (n = 346) and + 0.57 (95% CI: 0.42-0.73) over four years in the four-year observation group (n = 148). Significant deterioration of OMDS was noted in all subgroups with varying steroid use status. CONCLUSIONS This study revealed the incidence of comorbidities and drug utilization data in patients with HAM/TSP using registry data. Furthermore, this study provided real-world data on chronological changes in lower limb motor dysfunction in patients with HAM/TSP, indicating the utility of these data as historical controls.

中文翻译:

日本HTLV-1相关性脊髓病/热带痉挛性轻瘫(HAM / TSP)的真实临床过程。

背景技术由于人类T细胞白血病病毒1型(HTLV-1)相关的脊髓病/热带痉挛性轻瘫(HAM / TSP)是一种罕见的慢性神经系统疾病,因此难以进行用于收集连续临床数据的大规模研究。因此,合并症的发生率和药物利用数据仍然未知。在进行针对罕有疾病的新药(例如HAM / TSP)开发试验时,从注册研究中获得的历史对照数据将很有用,因为稀有疾病的人群往往很小。慢性病患者的长期随访也可能具有挑战性。在本研究中,我们使用2012年至2016年在日本HAM / TSP患者注册表“ HAM-net”中注册的患者(n = 486)的注册表数据解决了以下两个目标:1)阐明HAM / TSP的流行病学信息,例如合并症和药物利用的发生率; 2)提供有关下肢运动功能障碍变化的真实数据。结果在HAM网络登记的患者中,常见的合并症是骨折,带状疱疹和葡萄膜炎,每1000人年的发生率分别为55.5、10.4和6.5。每年,在HAM-net注册的患者中,口服类固醇治疗率为48.2-50.7%,在2.6-3.5%的患者中采用干扰素-α治疗。口服泼尼松龙的中位剂量低至5.0 mg / day。类固醇治疗组的骨折和带状疱疹的发生率往往高于未治疗组(骨折率:每1000人年61.0 vs. 48.3,带状疱疹:12.7 vs. 8.8)。芝麻运动障碍评分(OMDS)的时间变化分析表明,在一年观察组中,OMDS的平均变化为每年+ 0.20 [95%置信区间(CI):0.14-0.25](n = 346)四年观察组(n = 148)的四年内为+ 0.57(95%CI:0.42-0.73)。在所有类固醇使用状态不同的亚组中,OMDS均显着下降。结论本研究使用注册表数据揭示了HAM / TSP患者合并症和药物利用数据的发生率。此外,这项研究提供了有关HAM / TSP患者下肢运动功能障碍的时间变化的真实世界数据,表明这些数据可作为历史对照。[25]在一年观察组中(n = 346)每年;在四年观察组(n = 148)中,在四年内每年+ 0.57(95%CI:0.42-0.73)。在所有类固醇使用状态不同的亚组中,OMDS均显着下降。结论本研究使用注册表数据揭示了HAM / TSP患者合并症和药物利用数据的发生率。此外,这项研究提供了有关HAM / TSP患者下肢运动功能障碍的时间变化的真实世界数据,表明这些数据可作为历史对照。[25]在一年观察组中(n = 346)每年;在四年观察组(n = 148)中,在四年内每年+ 0.57(95%CI:0.42-0.73)。在所有类固醇使用状态不同的亚组中,OMDS均显着下降。结论本研究使用注册表数据揭示了HAM / TSP患者合并症和药物利用数据的发生率。此外,这项研究提供了有关HAM / TSP患者下肢运动功能障碍的时间变化的真实世界数据,表明这些数据可作为历史对照。结论本研究使用注册表数据揭示了HAM / TSP患者合并症和药物利用数据的发生率。此外,这项研究提供了有关HAM / TSP患者下肢运动功能障碍的时间变化的真实世界数据,表明这些数据可作为历史对照。结论本研究使用注册表数据揭示了HAM / TSP患者合并症和药物利用数据的发生率。此外,这项研究提供了有关HAM / TSP患者下肢运动功能障碍的时间变化的真实世界数据,表明这些数据可作为历史对照。
更新日期:2019-10-21
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