Keller San, Yang Manshu, Treadwell Marsha J, Hassell Kathryn L
American Institutes for Research, 100 Europa Drive, Suite 315, Chapel Hill, NC, 27517-2357, USA.
Children's Hospital & Research Center Oakland, 747 52nd Street, Oakland, CA, 94609, USA.
Health Qual Life Outcomes. 2017 Jun 2;15(1):117. doi: 10.1186/s12955-017-0661-5.
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℠, was developed for this purpose. ASCQ-Me℠ 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®) and one designed specifically for SCD (ASCQ-Me℠).
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.
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.
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.
镰状细胞病（SCD）会给患者带来巨大痛苦，并导致日常功能下降。对于能够系统记录这些影响的有效且可靠的测量方法的需求日益增长，尤其是在成年人中。成人镰状细胞生活质量测量系统（ASCQ-Me℠）就是为此目的而开发的。ASCQ-Me℠是由美国国立卫生研究院资助的以患者为中心的评估资源（PCAR）中的四个测量系统之一，用于支持临床研究。为了帮助用户为患有SCD的成年人选择最佳测量方法，我们对PCAR的两个系统进行了评估和比较：一个设计为“普遍适用”（患者报告结果测量信息系统，PROMIS®），另一个专门为SCD设计（ASCQ-Me℠）。
PROMIS和ASCQ-Me问题的受访者是来自美国七个地理位置分散的诊所的490名患有SCD的成年人。收集了六项ASCQ-Me测量指标的数据（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）以及十项PROMIS测量指标的数据（疼痛影响、疼痛行为、身体功能、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、睡眠障碍和与睡眠相关的损害）。进行了包括方差分析和多元线性回归在内的统计分析，以确定测量指标对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。
对于患有最严重SCD的患者，在十个健康领域中的九个领域，PROMIS评分显示其健康状况比一般人群更差：差异幅度在0.5至1.1个标准差单位之间。受影响最严重的PROMIS领域是身体功能和疼痛（影响和行为）。大多数PROMIS简表以及所有ASCQ-Me简表和固定形式在SCD-MHC三分位数上均显示出显著差异。在大多数模型中，ASCQ-Me测量指标在统计上解释了SCD-MHC评分中与相应PROMIS测量指标所解释的互补的显著独特方差。
研究结果支持PROMIS和ASCQ-Me测量指标在患有SCD的成年人中使用的有效性。与可比的PROMIS评分相比，大多数ASCQ-Me评分是SCD疾病严重程度更好的预测指标，通过病史清单进行测量。这些结果的临床意义需要进一步研究。
