Sensitivity of alternative measures of functioning and wellbeing for adults with sickle cell disease: comparison of PROMIS® to ASCQ-Me℠

Abstract

BackgroundSickle 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℠).MethodsRespondents 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.ResultsFor 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.ConclusionsStudy 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.

Clinical and genetic factors are associated with pain and hospitalisation rates in sickle cell anaemia in Cameroon

“…The issue of chronic pain was difficult to address with the study design, as it seems highly likely that individuals with 40 pain events per year are experiencing chronic pain. However, self-reported VOC in SCD has also been useful as a clinical endpoint in drug trials, patient quality of life measures and as a prognostic marker for mortality (Platt et al , 1991, Charache et al , 1995; Keller et al , 2017; Machado et al , 2011; Hoots and Shurin, 2012). The possible poor definition of VOC is also tempered by its strong association with the use of health services, which was more objectively assessed; validating, to some extent, the use of such cost-effective patient reported outcomes for genetic association study.…”

Pain and opioid use after reversal of sickle cell disease following HLA‐matched sibling haematopoietic stem cell transplant

“…Patient Reported Outcomes Measurement Information System (PROMIS) measures were also prospectively collected at the same time points in a subgroup of these patients ( n = 20). The PROMIS domains assessed included pain intensity, pain impact, anxiety, depression, satisfaction with social role, physical function, fatigue and sleep disturbance (Cella et al , ; Keller et al , ). All PROMIS raw data were converted to T‐scores.…”

End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain

“…44,45 In adults, PROMIS Pain Interference was validated in adults with SCD in a comparative study with ASCQ-ME and was found to be sensitive to differences in the presence of clinical complications. 46 ASCQ-ME Pain Impact was shown to be valid 47 with regard to overall SCD severity, but its longitudinal validity is not yet established.…”

“…48,49 Pain Behavior and Pain Quality domains have been used in adults with SCD. 46,47 For assessment of pain behavior, the PROs panel recommends using the PROMIS Pain Behavior domain for children and adults with SCD.…”