A Fibromyalgia-Specific Extension of the PROMIS Network

描述（申请人提供）：用于评估纤维肌痛（FM）的疾病状态和对治疗的反应性的现有方法受到了批评，理由是（a）现有患者报告的结局指标（PRO）未能充分评估与FM患者相关的领域，（B）大多数现有工具都是从其他疾病借用的，因此FM的疾病特异性PRO基本上没有开发，以及（c）许多现有的测量具有不利的心理测量特性和/或缺乏足够的变化敏感性。因此，有必要为FM提供新的方法学上合理的PRO，可以在短期内以传统方式提供，并通过与计算机自适应测试（CAT）相关的更敏感和更有效的方法提供。由于对森林管理的相关评估领域缺乏共识，因此无法为森林管理开发有意义的多层面工具;但最近世卫组织ICF倡议内的项目之间以及OMERACT内的项目之间达成的协议表明，启动此类工具开发的时机已经成熟。为此，该提案包含四个目标：首先，FM患者的焦点小组将由国家纤维肌痛协会（NFA）进行，以产生疾病特异性项目，可以补充NIH路线图计划PROMIS正在开发的更通用的慢性疾病项目库。目标二将在通过NFA招募的FM患者的大型代表性样本中，对疾病特异性项目沿着PROMIS项目库进行现场测试。将根据目标三进行一系列验证研究，以支持以传统方式在临床试验中使用新的疾病特异性量表。最后，在目标四下，将开发和实地测试一种利用PROMIS和FM疾病特定项目相结合的CAT算法。该项目的最终目标是将新的FM-CAT模块纳入PROMIS网络。该提案的研究者已经与ICF、OMERACT和PROMIS积极合作，在成功的结局测量开发和先进的定量方法方面有良好的记录，并拥有FM的临床专业知识。该项目产生的产品将在涉及FM的临床试验中具有直接的适用性和实用性，并代表了改进识别这种难治性疾病的有用和有意义的治疗方法的能力的下一个关键步骤。该项目与公共卫生有关，因为它将提供一种用于临床试验的疾病特异性测量工具，从而为纤维肌痛的评估和治疗提供更有效的方法。

DESCRIPTION (provided by applicant): Existing methods for assessing disease status and responsiveness to treatment in Fibromyalgia (FM) have been criticized on the grounds that (a) existing patient reported outcomes measures (PROs) fail to adequately assess the domains of relevance to patients with FM, (b) most existing instruments have been borrowed from other diseases and thus disease-specific PROs are largely undeveloped for FM, and (c) many of the existing measures have unfavorable psychometric properties and/or lack sufficient sensitivity to change. Thus, there is need for new methodologically sound PROs for FM that can be delivered in a traditional manner in the short term and via the more sensitive and efficient methodology associated with computer adaptive testing (CAT) in the longer term. Lack of consensus regarding the relevant assessment domains for FM has prevented the development of meaningful multi-dimensional instruments for FM; but recent agreement between projects within the WHO ICF initiatives and within OMERACT suggests that the time is right to initiate this type of instrument development. To this end, this proposal contains four aims: In the first, focus groups of FM patients will be conducted by the National Fibromyalgia Association (NFA) so as to generate disease-specific items that can supplement the more generic chronic illness item banks being developed by the NIH roadmap initiative PROMIS. Aim two will field test the disease-specific items along with the PROMIS item banks in a large representative sample of FM patients recruited through the NFA. A series of validation studies will be conducted under Aim three so as to support the use of the new disease- specific scales for administration in clinical trials in a traditional manner. Finally, under aim four, a CAT algorithm utilizing the combined PROMIS and FM disease-specific items will be developed and field tested. The final goal of this project will be the integration of the new FM-CAT module into the PROMIS network. The investigators of this proposal are already actively collaborating with ICF, OMERACT, and PROMIS, have track records in successful outcomes measurement development and advanced quantitative methods and possess clinical expertise in FM. The products resulting from this project will have direct applicability and utility in clinical trials involving FM and represent the next critical steps in refining the ability to identify useful and meaningful treatments for this refractory condition. This project is relevant to public health as it will provide a disease-specific measurement instrument to use in clinical trials thus leading to more effective methods for assessment and treatment of Fibromyalgia.