Improved responsiveness and reduced sample size requirements of PROMIS physical function scales with item response theory.

Improved responsiveness and reduced sample size requirements of PROMIS physical function scales with item response theory.
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DOI:
10.1186/ar3461
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发表时间:
2011
影响因子:
4.9
通讯作者:
Bruce B
Bruce B
中科院分区:
医学2区
文献类型:
--
作者:
Fries JF;Krishnan E;Rose M;Lingala B;Bruce B

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健康评估问卷残疾指数 (HAQ) 和 SF-36 PF-10 等工具可产生敏感且有效的残疾(身体功能)终点。现代技术,例如项目反应理论(IRT),现在可以使用改进的项目开发更精确的仪器。 NIH 患者报告结果测量信息系统 (PROMIS) 负责开发改进的基于 IRT 的工具。我们将使用原始(传统)仪器与基于 Item-Improved 和 PROMIS IRT 的仪器检测身体功能变化的能力进行了比较。我们研究了两种传统(原始)身体功能/残疾工具(HAQ、PF-10)、其项目改进衍生品(Item-Improved HAQ 和 PF-10)以及基于 IRT 的 PROMIS 身体功能 10-(PROMIS PF 10)和 20 项(PROMIS PF 20)工具。我们比较了检测 451 名类风湿关节炎 (RA) 患者 12 个月身体功能变化的敏感性,并使用 P 值、效应量 (ES) 和样本量要求评估了相对反应性。研究样本中 81% 为女性,87% 为白人,年龄 65 岁,受过 14 年教育,基线有中度残疾。所有仪器对于检测一年内身体机能的变化(< 0.05)都很敏感。这些患者中反应最灵敏的仪器是 Item-Improved HAQ 和 PROMIS PF 20。IRT 改进的仪器可以以 80% 的功效检测到 1.2% 的差异,而参考仪器只能检测到 2.3% 的差异 (P < 0.01)。最好的基于 IRT 的仪器仅需要传统 (PF-10) 比较器四分之一的样本量(95 对 427)。在更多受损人群中,HAQ 的表现优于 PF-10;在更正常的人群中情况恰恰相反。特别考虑到测量的严重程度范围,PROMIS PF 20 似乎是响应最灵敏的仪器。使用改进项目或基于 IRT 的项目的身体功能量表可以在更广泛的身体功能范围内提高响应能力和精确度。这可以减少样本量要求,从而降低研究成本。
The Health Assessment Questionnaire Disability Index (HAQ) and the SF-36 PF-10, among other instruments, yield sensitive and valid Disability (Physical Function) endpoints. Modern techniques, such as Item Response Theory (IRT), now enable development of more precise instruments using improved items. The NIH Patient Reported Outcomes Measurement Information System (PROMIS) is charged with developing improved IRT-based tools. We compared the ability to detect change in physical function using original (Legacy) instruments with Item-Improved and PROMIS IRT-based instruments. We studied two Legacy (original) Physical Function/Disability instruments (HAQ, PF-10), their item-improved derivatives (Item-Improved HAQ and PF-10), and the IRT-based PROMIS Physical Function 10- (PROMIS PF 10) and 20-item (PROMIS PF 20) instruments. We compared sensitivity to detect 12-month changes in physical function in 451 rheumatoid arthritis (RA) patients and assessed relative responsiveness using P-values, effect sizes (ES), and sample size requirements. The study sample was 81% female, 87% Caucasian, 65 years of age, had 14 years of education, and had moderate baseline disability. All instruments were sensitive to detecting change (< 0.05) in physical function over one year. The most responsive instruments in these patients were the Item-Improved HAQ and the PROMIS PF 20. IRT-improved instruments could detect a 1.2% difference with 80% power, while reference instruments could detect only a 2.3% difference (P < 0.01). The best IRT-based instruments required only one-quarter of the sample sizes of the Legacy (PF-10) comparator (95 versus 427). The HAQ outperformed the PF-10 in more impaired populations; the reverse was true in more normal populations. Considering especially the range of severity measured, the PROMIS PF 20 appears the most responsive instrument. Physical Function scales using item improved or IRT-based items can result in greater responsiveness and precision across a broader range of physical function. This can reduce sample size requirements and thus study costs.
DOI: 10.1016/j.jclinepi.2006.06.025
发表时间: 2008-01-01
影响因子: 7.2
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DOI: 10.1186/ar2890
发表时间: 2009
影响因子: 4.9
作者:
Bruce B;Fries JF;Ambrosini D;Lingala B;Gandek B;Rose M;Ware JE Jr
通讯作者: Ware JE Jr