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了批评，理由是：(a)现有的患者报告的结果测量（PROs）不能充分评估与FM患者相关的领域，(b)大多数现有的仪器都是从其他疾病借来的，因此疾病特异性的PROs在很大程度上没有开发用于FM。(c)许多现有的测量方法具有不利的心理测量特性和/或缺乏足够的敏感性来改变。因此，有必要为FM提供新的方法学上可靠的优点，这些优点可以在短期内以传统的方式提供，并在长期内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学提供。由于对调频的相关评估领域缺乏共识，妨碍了为调频开发有意义的多维工具；但最近世卫组织ICF倡议内的项目与OMERACT内的项目之间达成的协议表明，现在是开始开发这类工具的时候了。为此，本提案包含四个目标：第一，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，可以补充由NIH路线图倡议PROMIS开发的更通用的慢性病项目库。目标二将在通过NFA招募的FM患者的大量代表性样本中对疾病特异性项目以及PROMIS项目库进行实地测试。将在目标3下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，将开发利用PROMIS和FM疾病特异性项目的CAT算法并进行实地测试。该项目的最终目标是将新的FM-CAT模块集成到PROMIS网络中。该提案的研究人员已经与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有记录，并拥有FM的临床专业知识。该项目产生的产品将在FM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。该项目与公共卫生相关，因为它将提供一种用于临床试验的疾病特异性测量工具，从而导致更有效的方法来评估和治疗纤维肌痛。

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.