Sensitivity of alternative measures of functioning and wellbeing for adults with sickle cell disease: comparison of PROMIS® to ASCQ-MeSM
Sensitivity of alternative measures of functioning and wellbeing for adults with sickle cell disease: comparison of PROMIS® to ASCQ-MeSM
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DOI：
10.1186/s12955-017-0661-5
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发表时间：
2017-06-02
影响因子：
3.6
通讯作者：
Hassell, Kathryn L.
中科院分区：
文献类型：
--
作者：
Keller, San;Yang, Manshu;Hassell, Kathryn L.
背景：镰状细胞病 (SCD) 会导致严重的痛苦和日常功能下降。对有效且可靠的措施来系统地记录这些影响（尤其是对成年人）的需求不断增长。成人镰状细胞生命质量测量系统 ASCQ-Me-SM 就是为此目的而开发的。 ASCQ-Me-SM 是以人为中心的评估资源 (PCAR) 内的四个测量系统之一，由美国国立卫生研究院资助，用于支持临床研究。为了帮助用户为 SCD 成人选择最佳措施，我们评估并比较了两种 PCAR 系统：一种设计为“普遍适用”（患者报告结果测量信息系统，PROMIS (R)），另一种专门为 SCD 设计 (ASCQ-Me-SM)。 方法：PROMIS 和 ASCQ-Me 问题的受访者是来自美国 7 个地理分布诊所的 490 名 SCD 成人。收集了六项 ASCQ-Me 指标（情绪影响、睡眠影响、社会影响、僵硬影响、疼痛影响、SCD 疼痛发作频率和严重程度）和十项 PROMIS 指标（疼痛影响、疼痛行为、身体机能、焦虑、抑郁、疲劳、对自主社交活动的满意度、对社会角色的满意度、睡眠障碍和睡眠相关障碍）的数据。进行统计分析，包括方差分析和多元线性回归，以确定措施对 SCD 严重程度的敏感性。通过相关治疗和病症清单评估 SCD 严重程度。结果：对于那些患有最严重 SCD 的人，PROMIS 评分显示，与普通人群相比，十个健康领域中有九个领域的健康状况较差：差异幅度为 0.5 至 1.1 个标准差单位。受影响最严重的 PROMIS 领域是身体功能和疼痛（影响和行为）。对于大多数 PROMIS 简短形式以及所有 ASCQ-Me 简短和固定形式，SCD-MHC 三分位数显示出显着差异。在大多数模型中，ASCQ-Me 测量解释了 SCD-MHC 分数中统计显着的独特方差，与相应的 PROMIS 测量所解释的差异互补。结论：研究结果支持 PROMIS 和 ASCQ-Me 测量用于 SCD 成人的有效性。与可比较的 PROMIS 评分相比，大多数 ASCQ-Me 评分可以更好地预测 SCD 疾病的严重程度（根据病史检查表进行测量）。这些结果的临床意义需要进一步研究。
Background: Sickle Cell Disease (SCD) causes profound suffering and decrements in daily functioning. Demand is growing for valid and reliable measures to systematically document these effects, particularly in adults. The Adult Sickle Cell Quality of Life Measurement System, ASCQ-Me-SM, was developed for this purpose. ASCQ-Me-SM is one of four measurement systems housed within the Person-Centered Assessment Resource (PCAR), funded by the National Institutes of Health, to support clinical research. To help users select the best of these measures for adults with SCD, we evaluated and compared two PCAR systems: one designed to be "universally applicable" (the Patient-Reported Outcome Measurement Information System, PROMIS (R)) and one designed specifically for SCD (ASCQ-Me-SM).Methods: Respondents to PROMIS and ASCQ-Me questions were 490 adults with SCD from seven geographically-disbursed clinics within the US. Data were collected for six ASCQ-Me measures (Emotional Impact, Sleep Impact, Social Impact, Stiffness Impact, Pain Impact, SCD Pain Episode Frequency and Severity) and ten PROMIS measures (Pain Impact, Pain Behavior, Physical Functioning, Anxiety, Depression, Fatigue, Satisfaction with Discretionary Social Activities, Satisfaction with Social Roles, Sleep Disturbance, and Sleep-Related Impairment). Statistical analyses, including analysis of variance and multiple linear regression, were conducted to determine the sensitivity of measures to SCD severity. SCD severity was assessed via a checklist of associated treatments and conditions.Results: For those with the most severe SCD, PROMIS scores showed worse health compared to the general population for nine of ten health domains: the magnitude of the difference ranged 0.5 to 1.1 standard deviation units. The PROMIS domains most severely affected were Physical Functioning and Pain (Impact and Behavior). Significant differences by tertile of the SCD-MHC were shown for most PROMIS short forms and all ASCQ-Me short and fixed forms. In most models, ASCQ-Me measures explained statistically significant unique variance in SCD-MHC scores complementary to that explained by corresponding PROMIS measures.Conclusions: Study results supported the validity of both PROMIS and ASCQ-Me measures for use in adults with SCD. Compared to comparable PROMIS scores, most ASCQ-Me scores were better predictors of SCD disease severity, as measured by a medical history checklist. The clinical implications of these results require further investigation.