A Fibromyalgia-Specific Extension of the PROMIS Network

描述(由申请人提供)：现有的评估纤维肌痛(FM)疾病状态和治疗反应性的方法受到批评，理由是(A)现有的患者报告结果衡量标准(PRO)未能充分评估与FM患者相关的领域，(B)大多数现有工具是从其他疾病借用的，因此针对疾病的PRO基本上没有开发用于FM，以及(C)许多现有衡量标准具有不利的心理测量学特性和/或缺乏足够的变化敏感度。因此，有必要为FM提供新的方法上合理的专业知识，短期内可以通过传统方式提供，长期来说可以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来提供。对FM的相关评估领域缺乏共识，阻碍了FM有意义的多维工具的开发；但世卫组织ICF倡议和OMERACT内的项目最近达成的协议表明，启动这类工具开发的时机是正确的。为此，这项建议包含四个目标：首先，全国纤维肌痛协会(NFA)将对FM患者进行重点小组调查，以生成特定疾病的项目，这些项目可以补充NIH路线图倡议PROMIS正在开发的更通用的慢性病题库。目的二将在通过NFA招募的具有代表性的FM患者的大样本中，对疾病特定项目以及PROMIS题库进行现场测试。将在目标三项下进行一系列验证研究，以支持在临床试验中以传统方式使用新的疾病特定量表。最后，在目标四下，将开发利用PROMIS和FM疾病特定项的组合的CAT算法并进行现场测试。该项目的最终目标是将新的FM-CAT模块整合到PROMIS网络中。这项建议的研究人员已经与ICF、OMERACT和PROMIS积极合作，在成功的结果测量开发和先进的定量方法方面拥有跟踪记录，并拥有FM的临床专业知识。该项目产生的产品将在涉及FM的临床试验中直接适用和实用，并代表着下一个关键步骤，以完善为这种难治性疾病确定有用和有意义的治疗方法的能力。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仪器，从而导致评估和治疗纤维肌痛的更有效的方法。

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.