Legacy Patient-reported Outcome Measures Research Articles

Comparing the Responsiveness of the Global Rating Scale With Legacy Knee Outcome Scores: A Delaware-Oslo Cohort Study

Background: The selection of patient-reported outcome measures (PROMs) is essential for obtaining meaningful information to treat a patient, determine a plan of care, and make clinical decisions; however, the process of selecting PROMs for clinical care is difficult, with the need to balance these multiple factors. Variation makes it difficult to compare data across providers and studies. Hypothesis/Purpose: The purpose was to determine the responsiveness of 4 PROMs via effect size and the presence of a ceiling effect in the 5 years after anterior cruciate ligament reconstruction (ACLR). We hypothesized that the single-item Global Rating Scale (GRS) would have an effect size and ceiling effect similar to the commonly used legacy PROMs. Study Design: Cohort study (diagnosis); Level of evidence, 2. Methods: Of the 300 participants, 218 had ACLR, completed postoperative progressive criterion-based rehabilitation early after surgery, and were followed for 5 years. We collected data based on the GRS, the Knee Outcome Survey–Activities of Daily Living Scale (KOS-ADLS), the International Knee Documentation Committee–Subjective Knee Form (IKDC-SKF), and the KOOS (Knee injury and Osteoarthritis Outcome Score) before and after training and at 6, 12, 24, and 60 months after ACLR. Results: The IKDC-SKF had the largest effect sizes and lowest ceiling effects. The GRS had a similar size and change in both effect size and ceiling effect when compared with the longer PROMs. The GRS and IKDC-SKF had a correlation of 0.72, and the GRS had a minimal detectable change of 2.9 or 4.8, depending on methodology. Conclusion: The GRS responded similarly to the IKDC-SKF, KOS-ADLS, and KOOS and was responsive to patient change. The ease of use and patient-specific nature of the question means that it may be appropriate to use the GRS in clinical care as a consistent measure throughout the course of rehabilitation.

PROMIS® and Neuro-QoLTM measures are valid measures of health-related quality of life among patients with familial chylomicronemia syndrome

ABSTRACT Background FCS significantly affects health-related quality of life (HRQOL). Legacy patient-reported outcome measures are often not sensitive to FCS’s impact. NIH PROMIS and Neuro-QoL measures may accurately capture HRQOL in FCS patients. This study assessed a broad range of PROMIS and Neuro-QoL measures covering physical, mental, and social HRQOL to determine their suitability for the FCS population. Methods Adult FCS patients in the United States (N = 25) were recruited to an online survey study and completed several PROMIS short forms and Neuro-QoL computer adaptive tests. Results Scores were more than 0.5 standard deviations (SD) worse than the normative mean on 10 of 16 normed measures, and more than 0.75 SDs worse than the normative mean on two measures. Responses at the floor and ceiling were occasionally observed, marginal reliabilities were strong, and significant differences across performance status (ps < 0.05) provided preliminary support for construct validity. The measures correlated with each other strongly and as expected. Conclusion Results support the ability of PROMIS and Neuro-QoL measures to detect HRQOL impairment among patients with FCS. PROMIS and Neuro-QoL measures captured the functional impact and symptom burden associated with FCS, and the broad range of symptom severity experienced by patients with FCS.

Relationship between the Patient-Reported Outcomes Measurement Information System (PROMIS) computer adaptive testing and legacy instruments in patients undergoing isolated biceps tenodesis

The purpose of this study was to correlate the Patient-Reported Outcomes Measurement Information System (PROMIS) computer adaptive testing (CAT) domains with commonly used legacy patient-reported outcome measures (PROMs) preoperatively in a population of patients presenting to a tertiary care orthopedic center for biceps and labral pathology. Prospective data were collected on 175 patients scheduled to undergo isolated biceps tenodesis at a tertiary center. Enrollees completed legacy scores (Veterans RAND 12-Item Health Survey, Short Form 12, American Shoulder and Elbow Surgeons [ASES] Assessment Form, Single Assessment Numeric Evaluation, and Constant-Murley score) and PROMIS CAT questionnaires (Upper Extremity [UE], Pain Interference [PIF], and Depression). In addition, patients were asked to provide an assessment of the strength, function, and pain of the affected shoulder using a custom visual analog scale (VAS) questionnaire. Spearman rank correlations of the PROMIS CAT with legacy measures and the custom VAS were calculated. Floor and ceiling effects were assessed. The UE CAT yielded moderate correlations with the ASES score (r = 0.57) and the custom VAS strength score (r = 0.50-0.57). The PIF CAT demonstrated moderate correlations with the VAS pain score (r = 0.45) and high-moderate correlations with the ASES score, VR6D score, and UE CAT (r = 0.61-0.66). The Depression CAT demonstrated high-moderate correlations with the mental health legacy measures (r = -0.64 to -0.61). There was a significant relative floor effect for the Depression CAT at a score of 34.2 (19%). The PIF CAT was comparable to the ASES score, the current gold standard. Compared with legacy measures, both the UE CAT and PIF CAT are less burdensome and have few floor or ceiling effects. The PIF CAT may be a viable alternative to describe the physical and psychosocial impact of pain in biceps tenodesis patients.

PROMIS Versus Legacy Patient-Reported Outcome Measures in Patients Undergoing Surgical Treatment for Symptomatic Acetabular Dysplasia

Background: No previous study has investigated how the Patient-Reported Outcomes Measurement Information System (PROMIS) performs compared with legacy patient-reported outcome measures in patients with symptomatic acetabular dysplasia treated with periacetabular osteotomy (PAO). Purpose: To (1) measure the strength of correlation between the PROMIS and legacy outcome measures and (2) assess floor and ceiling effects of the PROMIS and legacy outcome measures in patients treated with PAO for symptomatic acetabular dysplasia. Study Design: Cohort study (Diagnosis); Level of evidence, 2. Methods: This study included 220 patients who underwent PAO for the treatment of symptomatic acetabular dysplasia. Outcome measures included the Hip disability and Osteoarthritis Outcome Score (HOOS) pain, HOOS activities of daily living (ADL), modified Harris Hip Score (mHHS), PROMIS pain, and PROMIS physical function subsets, with scores collected preoperatively and/or postoperatively at a minimum 12-month follow-up. The change in mean scores from preoperatively to postoperatively was calculated only in a subgroup of 57 patients with scores at both time points. Distributions of the PROMIS and legacy scores were compared to evaluate floor and ceiling effects, and Pearson correlation coefficients were calculated to evaluate agreement. Results: The mean age at the time of surgery was 27.7 years, and 83.6% were female. The mean follow-up time was 1.5 years. Preoperatively, neither the PROMIS nor the legacy measures showed significant floor or ceiling effects. Postoperatively, all legacy measures showed significant ceiling effects, with 15% of patients with a maximum HOOS pain score of 100, 29% with a HOOS ADL score of 100, and 21% with an mHHS score of 100. The PROMIS and legacy instruments showed good agreement preoperatively and postoperatively. The PROMIS pain had a moderate to strong negative correlation with the HOOS pain (r = −0.66; P < .0001) and mHHS (r = −0.60; P < .0001) preoperatively and the HOOS pain (r = −0.64; P < .0001) and mHHS (r = −0.64; P < .0001) postoperatively. The PROMIS physical function had a moderate positive correlation with the HOOS ADL (r = 0.51; P < .0001) and mHHS (r = 0.49; P < .0001) preoperatively and a stronger correlation postoperatively with the HOOS ADL (r = 0.56; P < .0001) and mHHS (r = 0.56; P < .0001). Conclusion: We found good agreement between PROMIS and legacy scores preoperatively and postoperatively. PROMIS scores were largely normally distributed, demonstrating an expanded ability to capture variability in patients with improved outcomes after treatment.

Preoperative Validation of the Patient-Reported Outcomes Measurement Information System in Patients With Articular Cartilage Defects of the Knee

To validate the Patient-Reported Outcomes Measurement Information System (PROMIS) physical function computer adaptive test (PF CAT) with current patient-reported outcome (PRO) instruments in patients with cartilage injuries of the knee. Patients scheduled for osteochondral autograft or allograft transplant, microfracture, autologous chondrocyte implantation, allograft cartilage resurfacing, and chondroplasty were prospectively enrolled in the study and completed PROMIS PF CAT, Knee Injury and Osteoarthritis Outcome Score (KOOS activities of daily living, pain, symptoms, sport, and quality of life), Short Form-36 Health Survey (SF-36 physical function [PF] and Physical Component Summary), and EuroQol-5 Dimension questionnaires. The Spearman correlation coefficient was used to compare instruments. Instrument correlations were defined as excellent (>0.7), excellent to good (0.61-0.69), good (0.4 to 0.6), and poor (<0.39), with significance defined as P < .05. A total of 293 knees in 275 patients (54.5% male) undergoing 319 cartilage procedures were analyzed. The most commonly performed cartilage procedure was chondroplasty (n= 118; 37.0%), followed by microfracture (n= 100; 31.3%). The mean age was 34.0 ± 14.7 and the mean body mass index was 30.0 ± 6.9. The PROMIS PF CAT had an excellent correlation with the SF-36 PF (r= 0.819; P < .001), SF-36 Physical Component Summary (r= 0.766; P < .001), KOOS activities of daily living (r= 0.733; P < .001), KOOS Sport (r= 0.709; P < .001), and EuroQol-5 Dimension (r= 0.752; P < .001) instruments; an excellent-good correlation with the KOOS pain (r= 0.662; P < .001), and KOOS quality of life (r= 0.640; P < .001) scores; and a good correlation with the KOOS symptoms (r= 0.519; P < .001) scale. The PROMIS PF CAT had no floor or ceiling effects and the smallest question burden (mean 4.17 ± 0.93 questions). Dimensionality analyses demonstrated that the smallest amount of unexplained variance was present in the PROMIS PF CAT (3.6%). The PROMIS PF CAT is an effective tool for preoperative outcome assessment in patients with cartilage defects of the knee. It correlates strongly with legacy PRO measures of physical function with no ceiling and floor effects and a minimal time burden for completion. Further study is warranted to determine postoperative performance and to evaluate the responsiveness of PROMIS to change within a patient. III; Prognostic retrospective comparative study.

Preoperative Performance of Patient-Reported Outcomes Measurement Information System in Patients with Meniscal Root Tears.

The purpose of this study was to establish preoperative validity of the Patient-Reported Outcomes Measurement Information System physical function computer adaptive test (PROMIS PF-CT) with legacy patient-reported outcome measures (PROMs) for meniscal root tears (MRTs). Our study included 51 patients (52 knees) with MRT. Patients completed PROMIS PF-CT, Short Form 36 (SF-36 physical function, pain, general health, vitality, social function, emotional well-being, role limitations due to physical health, and role limitations due to emotional problems), Knee Injury and Osteoarthritis Outcome Score (KOOS pain, symptoms, activities of daily living [ADLs], sports, and quality of life [QOL]), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC pain, stiffness, and function), EuroQol-5 dimensions (EQ-5D), and Knee Activity Scale questionnaires at their preoperative visit. Correlations between the PROMs listed above were evaluated along with floor and ceiling effects. Correlations were defined as weak (0.2-0.39), moderate (0.4-0.59), strong (0.6-0.79), and very strong (0.8-1.0). Preoperative data showed that PROMIS PF-CT has a strong correlation with SF-36 PF, KOOS-ADL, WOMAC-function, and EQ-5D; and moderate correlation with KOOS-sport, KOOS-pain, KOOS-symptoms, KOOS-QOL, WOMAC-pain, and WOMAC-stiffness. The Knee Activity Scale did not show any significant correlation with PROMIS PF-CT (r = 0.12, p = 0.2080). Of all the PROMs administered, PROMIS PF-CT demonstrated no floor or ceiling effects compared with 11.54% ceiling effect in KOOS-sports, and 5.77% floor effect in KOOS-ADL. On average, patients answered fewer PROMIS PF-CT questions (4.15 ± 0.72). PROMIS PF-CT is a valuable tool to assess preoperative patient-reported physical function in patients that may undergo MRT repair. It correlates strongly with other well-established PROMs. It also demonstrated no floor or ceiling effects and demonstrated a low test burden in our sample of 52 knees. This is a level III, prognostic retrospective comparative study.

A comparison of PROMIS Global Health-Mental and legacy orthopedic outcome measures for evaluating preoperative mental health status

The influence of mental health on postoperative outcomes necessitates comparison of the mental health constructs captured by patient reported outcome measures (PROMs). 1,026 patients completed preoperative PROMs including the PROMIS Physical Function (PF), PROMIS Global Health (GH), and a subspecialty-specific PROM. Across specialties, PROMIS GH-Physical, PF-CAT, and PF-SF scores correlated more strongly with all legacy PROMs than the GH-Mental score. Moderate to strong correlation between the PF-SF, PF-CAT, and GH-Physical questionnaires and subspecialty-specific PROMs across specialties suggest they assess similar concepts. Lower correlation between the GH-Mental and subspecialty-specific PROMs suggest that the GH-Mental questionnaire captures distinct patient-reported mental health.

The Impact of Workers' Compensation on Patient-Reported Outcomes Measurement Information System Upper Extremity and Legacy Outcome Measures in Patients Undergoing Arthroscopic Rotator Cuff Repair

To examine the preoperative performance of the Patient-Reported Outcomes Measurement Information System (PROMIS) Upper Extremity (UE) Computer Adaptive Test (CAT) with respect to legacy scores in patients receiving rotator cuff repair (RCR). In addition, to define the impact of Workers' Compensation (WC) status on both performance and floor and ceiling effects. The PROMIS UE CAT was administered preoperatively alongside legacy patient-reported outcome measures (PROMs) to patients undergoing isolated arthroscopic RCR from November 2017 to September 2018. Performance was assessed using Spearman correlation coefficients, and floor and ceiling effects were examined. One hundred twenty-two patients (WC, n= 32; non-WC, n= 90; 62.3% male, 53.6 ± 11.5years) were included. PROMs assessing physical function (r= 0.41-0.77) correlated more strongly to the PROMIS UE CAT than did multidomain or mental health PROMs (r= 0.25-0.61). In WC patients, the PROMIS UE CAT demonstrated diminished correlative strength relative to shoulder function PROMs. WC patients also demonstrated relative floor effects for Single Assessment Numerical Evaluation (SANE; 18.8%) and Constant-Murley (15.6%) and relative ceiling effects for the BriefResilience Scale (53.1%), Short Form 12 Mental Component Score (50%), and Veterans Rand 12 Mental Component Score (53.1%) and were more likely to report the minimum SANE score (P < .01) and the maximum Brief Resilience Scale score (P < .01). No absolute or relative floor/ceiling effects for the PROMIS UE CAT were found. Compared with a non-WC cohort, WC patients have significantly lower preoperative PROMIS UE CAT scores, are more likely to report the absolute minimum and maximum scores for various PROMs, and demonstrated relative floor and ceiling effects for PROMs assessing mental health. The absence of significant floor/ceiling effects for the PROMIS UE CAT may suggest improved outcome discrimination and may support the adoption of PROMIS UE for the assessment of functional status in WC patients with rotator cuff pathology. Level III, retrospective comparative trial.

P117. The use of patient-reported outcomes measurement information system (PROMIS) in spine surgery: a systematic review

BACKGROUND CONTEXT PROMIS was developed to provide a time-saving, easy to administer patient outcome questionnaire that was adaptable to a variety of medical and surgical subspecialties. Numerous authors have examined the effectiveness of PROMIS in various areas of spine surgery. PURPOSE To assess the correlation of PROMIS with legacy patient-reported outcome measures in spine surgery STUDY DESIGN/SETTING Systematic review. OUTCOME MEASURES Correlation coefficient (r) of PROMIS PF, Pin, PIf, PB with legacy outcomes. METHODS A systematic search of the Pubmed, EMBASE, and Cochrane databases using the Preferred Reporting Items for Systematic Reviews and Meta-Analysis guidelines was performed yielding 254 unique studies reporting on “PROMIS” in “spine.” Each study was independently reviewed. Fifty-four studies were selected for review, and 16 studies were selected for inclusion. RESULTS The pooled sample size yielded a total of 4,344 patients. In the cervical population, PROMIS physical function (PF), pain intensity (PIn), pain interference (PIf) and pain behavior (PB) all strongly correlated with the NDI (average |r|=0.64-0.76). PROMIS PF also strongly correlated with the mJOA (average |r|=0.66). Among patients with lumbar pathology and adult spinal deformities, the PF, PIn, PIf, PB, and social roles satisfaction (SR) all strongly correlated with the ODI (|r|=0.66-0.76). PF, PI, and anxiety (AX) also strongly correlated with the SRS-22/SRS-30. When comparing PROMIS to legacy measures of global health, PROMIS PF was strongly correlated with the SF-12 (|r|=0.67), while PROMIS AX and depression (D) correlated with the GAD and PHQ-8 measures. On average, all individual PROMIS domains required less time to complete (49.6-56 seconds) compared to ODI (176 seconds), NDI (190.3 seconds), SF-12 (214 seconds), and SF-36 PF (99 seconds). Responsiveness of PROMIS PF (ES=0.85, SRM=0.88), PIf (ES=1.14, SRM=0.92), and PB (ES=0.895) was comparable to ODI (ES=1.02, SRM=1.06), NDI (ES=0.75, SRM=0.96) and SF-12 (ES=0.675) CONCLUSIONS PROMIS PF, PIn, PIf and PB demonstrated strong correlations with NDI, mJOA, ODI, SRS, and SF-12 measures in various spine patient populations. Decreased time to completion and comparable responsiveness of PROMIS suggests that it may be used to assess patient-reported outcomes in spinal surgery patients. FDA DEVICE/DRUG STATUS This abstract does not discuss or include any applicable devices or drugs.

Preoperative Performance of the PROMIS in Patients Undergoing Hip Arthroscopic Surgery for Femoroacetabular Impingement Syndrome

Background:The Patient-Reported Outcomes Measurement Information System (PROMIS) is being increasingly evaluated for use in orthopaedic surgery. The performance of the PROMIS in patients undergoing hip preservation surgery is unknown.Purpose:To investigate the psychometric performance of the PROMIS compared with legacy patient-reported outcome measures (PROMs) in patients indicated for hip arthroscopic surgery for femoroacetabular impingement syndrome (FAIS).Study Design:Cohort study (diagnosis); Level of evidence, 2.Methods:Data from consecutive patients who underwent primary hip arthroscopic surgery between January and October 2018 for the treatment of FAIS were collected and analyzed. Baseline data, including preoperative PROM scores and demographics, were recorded. PROMs included the PROMIS Physical Function (PF), the Hip Outcome Score (HOS), the modified Harris Hip Score (mHHS), the International Hip Outcome Tool–12 (iHOT-12), and the Veterans RAND 12-item health survey (VR-12). Pearson and Spearman coefficient analyses were used to identify correlations between continuous and noncontinuous variables, respectively. Correlation was defined as excellent (>0.80), very good (0.71-0.80), good (0.61-0.70), fair (0.41-0.60), or poor (0.21-0.40). A multivariate linear regression analysis was used to identify preoperative predictors of having higher preoperative PROMIS scores.Results:A total of 197 patients with a mean age and body mass index (BMI) of 32.8 ± 12.6 years and 25.7 ± 5.6 kg/m2, respectively, were included in the final analysis. There were no ceiling or floor effects for the PROMIS PF score observed in the study group. With regard to hip-specific measures, PROMIS PF scores demonstrated excellent correlation with HOS–Activities of Daily Living (r = 0.801; P < .001) scores, very good correlation with mHHS (r = 0.721; P < .001) and iHOT-12 (r = 0.722; P < .001) scores, and good correlation with HOS–Sports Specific (r = 0.675; P < .001) scores. With regard to general health–related quality of life (HRQoL) measures, PROMIS PF scores demonstrated very good correlation with VR-12 mental (r = 0.721; P < .001) scores, good correlation with VR-12 physical (r = 0.618; P < .001) scores, and poor correlation with visual analog scale for pain (r = –0.365; P < .001) scores. Patients who reported being physically active were more likely to have a higher preoperative PROMIS score (β = 3.216; P = .004). Lower PROMIS scores were found in patients with a higher BMI (β = –0.236; P = .005) and in female patients (β = –2.608; P = .014).Conclusion:In patients with FAIS, the preoperative PROMIS PF scores demonstrated excellent to good correlation with legacy hip-specific instruments as well as with HRQoL measures. No ceiling or floor effects were identified. Notably, of the hip-specific PROMs administered, the PROMIS PF demonstrated the weakest correlation with the HOS–Sports Specific subscale. Physical activity, BMI, and sex were predictors of preoperative PROMIS scores in our patient population.

The Patient-Reported Outcomes Measurement Information System in spine surgery: a systematic review.

OBJECTIVEThe Patient-Reported Outcomes Measurement Information System (PROMIS) was developed to provide a standardized measure of clinical outcomes that is valid and reliable across a variety of patient populations. PROMIS has exhibited strong correlations with many legacy patient-reported outcome (PRO) measures. However, it is unclear to what extent PROMIS has been used within the spine literature. In this context, the purpose of this systematic review was to provide a comprehensive overview of the PROMIS literature for spine-specific populations that can be used to inform clinicians and guide future work. Specifically, the authors aimed to 1) evaluate publication trends of PROMIS in the spine literature, 2) assess how studies have used PROMIS, and 3) determine the correlations of PROMIS domains with legacy PROs as reported for spine populations.METHODSStudies reporting PROMIS scores among spine populations were identified from PubMed/MEDLINE and a review of reference lists from obtained studies. Articles were excluded if they did not report original results, or if the study population was not evaluated or treated for spine-related complaints. Characteristics of each study and journal in which it was published were recorded. Correlation of PROMIS to legacy PROs was reported with 0.1 ≤ |r| < 0.3, 0.3 ≤ |r| < 0.5, and |r| ≥ 0.5 indicating weak, moderate, and strong correlations, respectively.RESULTSTwenty-one articles were included in this analysis. Twelve studies assessed the validity of PROMIS whereas 9 used PROMIS as an outcome measure. The first study discussing PROMIS in patients with spine disorders was published in 2012, whereas the majority were published in 2017. The most common PROMIS domain used was Pain Interference. Assessments of PROMIS validity were most frequently performed with the Neck Disability Index. PROMIS domains demonstrated moderate to strong correlations with the legacy PROs that were evaluated. Studies assessing the validity of PROMIS exhibited substantial variability in PROMIS domains and legacy PROs used for comparisons.CONCLUSIONSThere has been a recent increase in the use of PROMIS within the spine literature. However, only a minority of studies have incorporated PROMIS for its intended use as an outcomes measure. Overall, PROMIS has exhibited moderate to strong correlations with a majority of legacy PROs used in the spine literature. These results suggest that PROMIS can be effective in the assessment and tracking of PROs among spine populations.

Relationship Between PROMIS Computer Adaptive Tests and Legacy Hip Measures Among Patients Presenting to a Tertiary Care Hip Preservation Center

Background: Legacy hip outcome measures may be burdensome to patients and sometimes yield floor or ceiling effects. Patient-Reported Outcomes Measurement Information System (PROMIS) computer adaptive tests (CATs) allow for low-burden data capture and limited ceiling and floor effects. Purpose/Hypothesis: The purpose of this study was to determine whether the PROMIS CAT domains demonstrate correlation against commonly used legacy patient-reported outcome measures in a population of patients presenting to a tertiary care hip preservation center. The authors hypothesized the following: (1) PROMIS CAT scores based on physical function (PF), pain interference (PIF), pain behavior, and pain intensity would show strong correlation with the following legacy scores: modified Harris Hip Score (mHHS), International Hip Outcome Tool–12 (iHOT-12), Hip Outcome Score (HOS) Sports and Activities of Daily Living subscales, and Veterans RAND–6D (VR-6D) utility measure. (2) The mental and physical health portions of the VR-6D legacy measure would show weak correlation with mental- and psychosocial-specific PROMIS elements—depression, anxiety, fatigue, sleep, and ability to participate in social roles and activities. (3) All PROMIS measures would exhibit fewer floor and ceiling effects than legacy scores. Study Design: Cohort study (diagnosis); Level of evidence, 3. Methods: Prospective data were collected on 125 patients in the hip preservation clinics. Enrollees completed legacy scores (visual analog scale for pain, mHHS, iHOT-12, HOS, and VR-6D) and PROMIS CAT questionnaires (PF, PIF, pain behavior, anxiety, depression, sleep, social roles and activities, pain intensity, fatigue). Spearman rank correlations were calculated, with rs values of 0 to 0.3 indicating negligible correlation; 0.3 to 0.5, weak correlation; 0.5 to 0.7, moderately strong correlation; and >0.7, strong correlation. Floor and ceiling effects were evaluated. Results: As anticipated, the PF-CAT yielded strong correlations with the iHOT-12, mHHS, HOS–Sports, HOS–Activities of Daily Living, and VR-6D, with rs values of 0.76, 0.71, 0.81, 0.87, and 0.71, respectively. The PIF-CAT was the only pain score to show moderately strong to strong correlation with all 14 patient-reported outcome measures. A strong correlation was observed between the VR-6D and the social roles and activities CAT (rs = 0.73). The depression CAT had a significant floor effect at 19%. No additional floor or ceiling effect was present for any other legacy or PROMIS measure. Conclusion: The PF-CAT shows strong correlation with legacy patient-reported outcome scores among patients presenting to a tertiary care hip preservation center. The PIF-CAT also correlates strongly with legacy and PROMIS measures evaluating physical and mental well-being. PROMIS measures are less burdensome and demonstrate no floor or ceiling effects, making them a potential alternative to legacy patient-reported outcome measures for the hip.

Physical function computer adaptive test outcomes in diabetic lumbar spine surgical patients

BACKGROUND CONTEXTDiabetes is a highly prevalent comorbid condition among patients undergoing spine surgery. Several studies have used legacy patient-reported outcome measures to implicate diabetes as a predictor of increased disability, pain, and decreased physical function and quality of life following spine surgery. The effect of diabetes on postoperative physical function has not yet been studied using the PROMIS Physical Function Computer Adaptive Test (PF CAT). PURPOSETo understand the effect of diabetes on physical function outcomes among patients undergoing lumbar spine surgery, as reported by the PF CAT. STUDY DESIGN/SETTINGA retrospective cohort study was performed at a single university-based spine clinic. PATIENT SAMPLEPatients who underwent lumbar spine surgery between October 1, 2013 and April 26, 2018 with both PF CAT and Oswestry Disability Index (ODI) scores available for review. OUTCOME MEASURESPROMIS PF CAT. Secondary measures of disability included the ODI. METHODSPF CAT and ODI questionnaires were administered to patients via electronic tablets. Data from these questionnaires were collected prospectively, and retrieved from a university database. Wilcox tests, Exact Wilcox tests, linear regression models, and descriptive analytics were applied. RESULTSPatients meeting inclusion criteria included 233 nondiabetic patients and 65 diabetic patients. Mean PF CAT scores among diabetics were lower than those of nondiabetics at all time-points from preoperative through 12 months postoperative, with significance found at both 6 months (p=.035) and 12 months (p=.039) postoperative. Mean ODI scores among diabetics were significantly higher than those of nondiabetics at 3 months (p=.018) and 12 months (p=.027) postoperative. By 12 months postoperative, a smaller proportion of diabetics reached PF CAT and ODI minimum clinically important difference thresholds when compared to nondiabetics. CONCLUSIONSDiabetes is associated with lower PF CAT scores up to one year following lumbar spine surgery. However, many of these patients achieve meaningful improvement in physical function during this time. The PF CAT is consistent with legacy outcome measures in assessing outcomes in diabetic patients undergoing lumbar spine surgery, with an added benefit of decreased patient burden.

Performance of PROMIS Global-10 Compared With Legacy Instruments for Rotator Cuff Disease

Background: The Patient-Reported Outcomes Measurement Information System (PROMIS) Global-10 was recently developed to assess physical and mental health and provide an estimated EuroQol-5 Dimension (EQ-5D) score. This instrument needs to be validated for specific patient cohorts such as those with rotator cuff pathology. Hypothesis: There is moderate to high correlation between the PROMIS Global-10 and legacy patient-reported outcome measures; PROMIS Global-10 will not show ceiling effects; and estimated EQ-5D scores will show good correlation and low variance with actual EQ-5D scores. Study Design: Cohort study (diagnosis); Level of evidence, 2. Methods: A total of 323 patients with rotator cuff disease were prospectively enrolled before treatment. Each patient completed the PROMIS Global-10, EQ-5D, American Shoulder and Elbow Surgeons (ASES) shoulder assessment form, and Single Assessment Numeric Evaluation (SANE), and those with known rotator cuff tears completed the Western Ontario Rotator Cuff Index (WORC). Spearman correlations were calculated. Bland-Altman agreement tests were conducted between estimated EQ-5D scores from the PROMIS and actual EQ-5D scores. Ceiling and floor effects were assessed, defined as ≥15% respondents with highest or lowest possible score. Results: Correlation between the PROMIS Global-10 and EQ-5D was excellent (0.70, P < .0001). Correlation of the PROMIS physical scores was excellent-good with the ASES (0.62, P < .0001), good with the WORC (0.47, P < .0001), and good with the SANE (0.41, P < .0005). Correlation between the PROMIS mental scores was poor with the ASES (0.34, P < .0001), the WORC (0.32, P = .0016), and the SANE (0.24, P < .0001). No floor or ceiling effects were found. Agreement analysis showed substantial variance in individual scores, despite the overall similarity in mean scores between the estimated and actual EQ-5D scores, indicating poor agreement. Bland-Altman 95% limits of agreement for estimated EQ-5D scores ranged from 34% below to 31% above actual EQ-5D scores. Conclusion: Physical function scores of the PROMIS Global-10 show high correlation with legacy patient-reported outcome instruments, suggesting that it is a reliable tool for outcome assessment in a population with rotator cuff pathology. The large variability in 95% limit of agreement suggested that the estimated EQ-5D scores from the PROMIS Global-10 cannot replace traditional EQ-5D scores.

Content validity and legacy patient-reported outcome measures in cancer.

The publication in 2009 of the Guidance for Industry by US Food and Administration (FDA) [1] ushered in a significant change in the way patient-reported outcome measures (PROMs) are evaluated when used by pharmaceutical companies to support product label claims. In particular, the FDA Guidance emphasised patient input in the development of PROMs. To underline this point, a review of the reasons for FDA rejections of PRO label claims [2] concluded that just under 40 % of all rejections were due to the PROM not being fit for purpose; 31 % of product PRO claims were rejected due to a lack of content validity. This approach adopted by the FDA may be problematic for those legacy PROMs whose development predates the Guidance, one of which is the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 [3]. There is no evidence that the early development of the EORTC QLQ-C30 incorporated patient involvement in the content validity other than in rating the acceptability of items. Despite this, the instrument continues to be widely used in oncology clinical trials: a search on clinicaltrials.gov for the period 1 January 2009 to 31 December 2013 revealed 207 registered trials that included the EORTC QLQ-C30. Furthermore, its inclusion has been recommended in comparative effectiveness research [4], and it has been shown to be of use in facilitating patient–clinician communication [5]. Beyond that the instrument has been part of a successful PRO product label claim granted by the FDA [6] after the publication of the Guidance document. Content validity is one particular psychometric property of the EORTC QLQ-C30 that has not been revisited since the early development of the instrument. Although the EORTC QLQ-C30 has been subsequently validated with other cancer diagnoses, it has undergone little or no change in terms of the original content. However, this needs to be balanced against the fact that the EORTC QLQ-C30 has been completed by thousands of patients with different types of cancers since its development. There are currently 31 studies registered on the EORTC clinical trials database with a total target recruitment of 29,106 patients across a spectrum of cancers including brain, breast, (paediatric) leukaemia, endocrine, gastrointestinal, genito-urinary, gynaecological, head and neck, lung, and melanoma (www.eortc.org/clinical-trials). It also should be stressed that the EORTC guidelines for the development of diseasespecific modules emphasise the necessity of patient input in order to enhance content validity [7]. It could therefore be argued that the EORTC QLQ-C30 has been subjected to a content validation process through exposure in numerous clinical trials, particularly given that the domains of the instrument, such as fatigue, sleeplessness, pain, appetite loss, nausea, and cognitive problems largely correspond with the core set of symptoms recommended for assessment by the National Cancer Institute’s Symptom Management & Health-related Quality of Life Steering Committee [8]. It remains for future research to determine whether the EORTC QLQC30 requires further modification or is—in its current form and owing to the significant post-development use across different cancers and treatments—fit for purpose in oncology trials. A. B. Smith (&) York Health Economics Consortium, Market Square, University of York, York YO10 5NH, UK e-mail: adam.smith@york.ac.uk

Validity of two new patient‐reported outcome measures in systemic sclerosis: Patient‐reported outcomes measurement information system 29‐item health profile and functional assessment of chronic illness therapy–dyspnea short form

Many patient-reported outcome (PRO) instruments used in systemic sclerosis (SSc) trials are limited by lack of validation, licensing fees, and complicated scoring systems. We assessed the construct validity for discriminative purposes of 2 new PRO instruments, the Patient-Reported Outcomes Measurement Information System 29-item Health Profile (PROMIS-29) and the Functional Assessment of Chronic Illness Therapy-Dyspnea short form (FACIT-Dyspnea), measuring health status and dyspnea in SSc patients. Seventy-three patients participated in a cross-sectional study at a tertiary SSc program. PROMIS-29, FACIT-Dyspnea, and legacy PRO instruments used in clinical trials (Medical Research Council Dyspnea Score, St. George's Respiratory Questionnaire, Health Assessment Questionnaire disability index, and Short Form 36) were administered. Composite severity scores using an adaptation of the Medsger Disease Severity Index were generated using clinical, diagnostic, and laboratory information. PROMIS-29 and FACIT-Dyspnea scores were compared with legacy PRO measures and composite severity scores. The mean patient age (84% women) was 51 years (range 22-72 years). The mean SSc disease duration from the onset of the first non-Raynaud's phenomenon symptom was 7.2 years (range 0-45 years). Spearman's correlation coefficients across FACIT-Dyspnea and PROMIS physical functioning scores with legacy PRO instruments were generally high (range 0.50-0.86); those between PROMIS and FACIT-Dyspnea with composite disease severity scores were more modest, but statistically significant (range 0.33-0.48, P < 0.01). PROMIS-29 and FACIT-Dyspnea are valid instruments to measure the health status of SSc patients. PROMIS-29 and FACIT-Dyspnea may be preferable to legacy instruments because they are freely available in multiple languages and simple to administer, score, and interpret.