背景:医学界开始认识到色素性视网膜炎(RP),由于它的残疾进展,最终导致患者生活质量下降,直接的经济影响,以及增加医疗保健系统的负担。这种疾病的起源没有治愈性的治疗方法,当前的大多数干预措施都未能减少相关的负面心理状态,比如焦虑和抑郁,这导致视力变异性增加,并对患者的独立性构成持续威胁。
目的:本研究的目的是评估单独口服褪黑素(OM)和短波光(SWL)阻断滤光片对RP患者的影响,并测试其在改善许多患者的压力水平和睡眠问题方面的有效性。
方法:我们为患有睡眠障碍和负面心理压力的RP患者开发了一种低成本的治疗方案。患者将随机接受SWL阻断过滤器和OM的联合干预,单独使用SWL阻塞滤波器,或OM单独。还将有一个非干预组作为对照组。这项研究将在患有睡眠障碍和高感知压力和焦虑评分报告的RP患者的2个视网膜单位进行。将在干预前对患者进行评估,在4周的干预期间,每周一次,然后在干预后6个月。主要结果是从基线到干预后激素释放的变化差异(α-淀粉酶,皮质醇,和褪黑激素)和睡眠质量,用视觉模拟量表测量。次要结果指标包括临床黄斑改变,用光学相干断层扫描和光学相干断层扫描血管造影测量;视网膜功能,使用视野和最佳矫正视力进行测量;从个人可穿戴设备收集的睡眠数据;以及几个患者报告的变量,比如自录的睡眠日记,生活质量,感知压力,和功能状态。
结果:该项目仍是研究方案,尚未开始。为证明其合理性而进行的书目信息研究始于2020年,该工作组目前正在寻求启动资金。一旦我们有了必要的手段,我们将在初步阶段之前进行注册和组织。
结论:在这项可行性随机对照试验中,我们将比较单独的SWL阻断的效果,仅管理OM,并对RP患者进行联合干预。我们提出了这项研究,以便可以复制并纳入其他机构的未来研究中,以及适用于其他遗传性视网膜营养不良。提出该协议的目的是帮助最近的努力,以减少睡眠障碍和其他心理障碍对RP患者生活质量的影响,并恢复其自我自主性。此外,这项研究的结果将代表着为RP患者开发一种新的低成本疗法并验证一种新的治疗靶点的重要一步.
■PRR1-10.2196/49196。
BACKGROUND: The medical community is beginning to recognize that retinitis pigmentosa (RP), due to its disabling progression, eventually leads to a reduction in the patient´s quality of life, a direct economic impact, and an increase in the burden on the health care system. There is no curative treatment for the origin of the disease, and most of the current interventions fail in reducing the associated negative psychological states, such as anxiety and depression, which lead to increased variability of vision and pose a continuous threat to the patient\'s independence.
OBJECTIVE: The aim of this
study is to assess the effect of oral melatonin (OM) administration alone and combined with short-wavelength light (SWL)-blocking filters on patients with RP and test their effectiveness in improving the level of stress and sleep problems in many of these patients.
METHODS: We have developed a low-cost therapy protocol for patients with RP with sleep disorders and negative psychological stress. Patients will be randomized to receive a combined intervention with SWL-blocking filters and OM, SWL-blocking filters alone, or OM alone. There will also be a nonintervention arm as a control group. This
study will be conducted across 2 retinal units in patients with RP with sleep disorders and high perceived stress and anxiety score reports. Patients will be assessed in the preintervention period, weekly during the 4 weeks of intervention, and then at 6 months postintervention. The primary outcomes are the differences in changes from baseline to postintervention in hormone release (α-amylase, cortisol, and melatonin) and sleep quality, as measured with the visual analog scale. Secondary outcome measures include clinical macular changes, as measured with optical coherence tomography and optical coherence tomography angiography; retinal function, as measured using the visual field and best-corrected visual acuity; sleep data collected from personal wearables; and several patient-reported variables, such as self-recorded sleep diaries, quality of life, perceived stress, and functional status.
RESULTS: This project is still a
study protocol and has not yet started. Bibliographic research for information for its justification began in 2020, and this working group is currently seeking start-up funding. As soon as we have the necessary means, we will proceed with the registration and organization prior to the preliminary phase.
CONCLUSIONS: In this feasibility randomized clinical controlled
trial, we will compare the effects of SWL blocking alone, administration of OM alone, and a combined intervention with both in patients with RP. We present this study so that it may be replicated and incorporated into future studies at other institutions, as well as applied to additional inherited retinal dystrophies. The goal of presenting this protocol is to aid recent efforts in reducing the impact of sleeping disorders and other psychological disorders on the quality of life in patients with RP and recovering their self-autonomy. In addition, the results of this
study will represent a significant step toward developing a novel low-cost therapy for patients with RP and validating a novel therapeutic target.
UNASSIGNED: PRR1-10.2196/49196.