Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland.
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, the Netherlands.
JAMA Ophthalmol. 2018 Nov 1;136(11):1217-1225. doi: 10.1001/jamaophthalmol.2018.3456.
Identifying and prioritizing unanswered clinical questions may help to best allocate limited resources for research associated with the treatment of age-related macular degeneration (AMD).
To identify and prioritize clinical questions and outcomes for research associated with the treatment of AMD through engagement with professional and patient stakeholders.
DESIGN, SETTING, AND PARTICIPANTS: Multiple cross-sectional survey questions were used in a modified Delphi process for panel members of US and international organizations, the American Academy of Ophthalmology (AAO) Retina/Vitreous Panel (n=7), health care professionals from the American Society of Retinal Specialists (ASRS) (n=90), Atlantic Coast Retina Conference (ACRC) and Macula 2017 meeting (n=34); and patients from MD (Macular Degeneration) Support (n=46). Data were collected from January 20, 2015, to January 9, 2017.
The prioritizing of clinical questions and patient-important outcomes for AMD.
Seventy clinical questions were derived from the AAO Preferred Practice Patterns for AMD and suggestions by the AAO Retina/Vitreous Panel. The AAO Retina/Vitreous Panel assessed all 70 clinical questions and rated 17 of 70 questions (24%) as highly important. Health care professionals assessed the 17 highly important clinical questions and rated 12 of 17 questions (71%) as high priority for research to answer; 9 of 12 high-priority clinical questions were associated with aspects of anti-vascular endothelial growth factor agents. Patients assessed the 17 highly important clinical questions and rated all as high priority. Additionally, patients identified 6 of 33 outcomes (18%) as most important to them (choroidal neovascularization, development of advanced AMD, retinal hemorrhage, gain of vision, slowing vision loss, and serious ocular events).
Input from 4 stakeholder groups suggests good agreement on which 12 priority clinical questions can be used to underpin research related to the treatment of AMD. The 6 most important outcomes identified by patients were balanced between intended effects of AMD treatment (eg, slowing vision loss) and adverse events. Consideration of these patient-important outcomes may help to guide clinical care and future areas of research.
确定和优先考虑未解决的临床问题，可能有助于为与年龄相关性黄斑变性（AMD）治疗相关的研究分配有限的资源。
通过与专业人员和患者利益相关者的合作，确定和优先考虑与 AMD 治疗相关的研究的临床问题和结果。
设计、地点和参与者：使用美国和国际组织、美国眼科学会（AAO）视网膜/玻璃体小组（n=7）、美国视网膜专家学会（ASRS）的医疗保健专业人员（n=90）、大西洋海岸视网膜会议（ACRC）和 Macula 2017 会议（n=34）的多项横断面调查问题，采用修改后的 Delphi 流程对美国和国际组织、美国眼科学会（AAO）视网膜/玻璃体小组（n=7）、美国视网膜专家学会（ASRS）的医疗保健专业人员（n=90）、大西洋海岸视网膜会议（ACRC）和 Macula 2017 会议（n=34）的小组人员进行评估；以及来自马里兰州（黄斑变性）支持组织的患者（n=46）。数据收集于 2015 年 1 月 20 日至 2017 年 1 月 9 日。
AMD 的临床问题和患者重要结果的优先级排序。
从 AAO 关于 AMD 的最佳实践指南和 AAO 视网膜/玻璃体小组的建议中得出了 70 个临床问题。AAO 视网膜/玻璃体小组评估了所有 70 个临床问题，并将其中 70 个问题中的 17 个（24%）评为高度重要。医疗保健专业人员评估了 17 个高度重要的临床问题，并将其中 12 个（71%）评为研究回答的高优先级；12 个高优先级临床问题中有 9 个与抗血管内皮生长因子药物的各个方面有关。患者评估了这 17 个高度重要的临床问题，并将所有问题都评为高度优先。此外，患者确定了 33 个结果中的 6 个（18%）是他们最关心的（脉络膜新生血管、晚期 AMD 的发展、视网膜出血、视力增益、视力丧失减缓、严重眼部事件）。
来自 4 个利益相关者群体的意见表明，对于可以用来支持与 AMD 治疗相关的研究的 12 个优先临床问题，意见基本一致。患者确定的 6 个最重要的结果在 AMD 治疗的预期效果（如视力丧失减缓）和不良事件之间取得了平衡。考虑这些患者重要的结果可能有助于指导临床护理和未来的研究领域。
