Patient reported outcome measures (PROMs) as primary and secondary outcomes in total hip and knee arthroplasty randomized controlled trials: a systematic review.

BACKGROUND: Significant heterogeneity exists regarding patient reported outcome measures (PROMs) used in total hip (THA) and knee (TKA) arthroplasty randomized controlled trials (RCTs). This study investigates the PROMs used as primary and secondary outcomes in contemporary arthroplasty RCTs. METHODS: A literature search identified THA and TKA RCTs that were published in top ten impact factor orthopaedic journals from 2017 to 2021. Screening identified 241 trials: 76 THA, 157 TKA, and eight combined. Data were extracted to identify PROMs utilized as either primary or secondary outcomes and the time period of measurement. RESULTS: Visual Analog Scale (VAS) Pain was the most reported primary PROM in THA (9.2%) and TKA (22.9%) trials. This was followed by Numeric Rating Scale (NRS) Pain (7.9%) and the Harris Hip score (6.6%) in THA trials and NRS Pain (4.5%) and the Knee Society score (4.5%) in TKA trials. Many THA (37.0%) and TKA (52.1%) trials did not clearly specify primary outcome time points. Only pain scales were reported at time points less than one week, while various joint-specific functional outcomes were reported at later time points. As secondary outcomes, the Harris Hip score (28.9%) was most common in THA trials and the Knee Society score (26.1%) was most common in TKA trials. Indeterminate primary or secondary outcomes were reported in 18.2% of studies. CONCLUSIONS: Contemporary THA and TKA trials exhibit heterogeneity of PROMs as study outcomes after the first postoperative week. Our findings highlight the need for consensus in PROM reporting and better methodological reporting to improve the interpretability of RCT outcomes. PROSPERO REGISTRATION NUMBER: CRD42022337255.

Six-month pain and function outcome expectations were established for total knee arthroplasty using the smallest worthwhile effect.

INTRODUCTION: Interpretations of patient-reported outcome measures following knee arthroplasty lack context and typically do not account for costs, risks and benefits compared to an alternative treatment. The primary purpose of our paper is to estimate expectations patients have for pain and function destination outcome, six-months following surgery relative to the outcome expected if knee arthroplasty was not done. Secondary purposes were to determine if statistically significant changes in the smallest worthwhile six-month outcome occurred following an interactive discussion and to assess the construct validity of the expected six-month outcome obtained at baseline. METHODS: This was a secondary analysis of a prospective longitudinal cohort study of 121 patients undergoing knee arthroplasty. Smallest worthwhile effect estimates were determined and expected six-month KOOS Pain and Function, daily activity measures were established during a pre-operative visit. RESULTS: The average six-month expected (the destination of interest) KOOS Pain score was 75 (IQR = 64 to 86) and the average KOOS Function, daily activity score was 74 (IQR = 59 to 86). The smallest worthwhile effect discussion led to significant changes in expected destination scores. For example, KOOS Pain expected outcome changed from 87.7 (9.8) to 75.0 (13.6), a statistically significant reduction in expected outcome (t(119) = 16.942, p < 0.001. CONCLUSION: Six-month expected KOOS outcomes following knee arthroplasty were established and approximate the average six-month outcomes reported in the literature. Validity of these estimates was established. These data can be used to aid shared decision-making discussions regarding patient expectations of knee arthroplasty outcomes during a patient encounter.

Using Two Predictive Models to Capture Two Types of Poor Outcomes in Knee Arthroplasty: A Multisite Longitudinal Cohort Study.

OBJECTIVE: Poor outcome after knee arthroplasty (KA), a common major surgery worldwide, reportedly occurs in approximately 20% of patients. These patients demonstrate minimal improvement, at least moderate knee pain, and difficulty performing many routine daily activities. The purposes of our study were to comprehensively determine poor outcome risk after KA and to identify predictors of poor outcome. METHODS: Data from 565 participants with KA in the Osteoarthritis Initiative and the Multicenter Osteoarthritis studies were used. Previously validated latent class analyses (LCAs) of good versus poor outcome trajectories of Western Ontario and McMaster Universities Arthritis Index (WOMAC) Pain and Disability were generated to describe minimal improvement and poor final outcome. The modified Escobar RAND appropriateness system was used to generate classifications of appropriate, inconclusive, and rarely appropriate. Multivariable prediction models included LCA-based good versus poor outcome, modified Escobar classifications, and evidence-driven preoperative prognostic variables. RESULTS: Modified Escobar appropriateness classifications were nonsignificant predictors of WOMAC Pain good versus poor outcomes, indicating the methods provide independent outcome estimates. For WOMAC Pain and WOMAC Disability, approximately 34% and 45% of participants, respectively, had a high probability of either minimal improvement via "rarely appropriate" classifications or poor outcome via LCA. In multivariable prediction models, greater contralateral knee pain consistently predicted poor outcome (eg, odds ratio 1.21, 95% confidence interval 1.10-1.33). CONCLUSION: Appropriateness criteria and LCA estimates provided combined poor outcome estimates that were approximately double the commonly reported poor outcome of 20%. Rates of poor outcome could be reduced if clinicians screened patients using appropriateness criteria and LCA predictors before surgery to optimize outcome.

Perioperative Opioid Use and Dosage Trajectories Vary Depending on Pain Outcome Classification and Bodily Pain in Patients who Catastrophize About Their Pain: A Secondary Analysis of a Randomized Trial in Knee Arthroplasty.

Opioid use and dosage following knee arthroplasty (KA) has not been reported for subgroups with persistent moderate pain versus rapidly improving mild pain, externally validated from prior work. We determined if opioid use and dosage varied for persons classified into these externally validated subgroups. A secondary purpose determined if bodily pain scores are associated with the outcome subgroup. This was a secondary analysis of a prospective no-effect randomized clinical trial conducted on 384 participants with pain catastrophizing and scheduled for KA. Data were collected preoperatively and at 2-, 6-, and 12-month following surgery. Two-piece latent class growth curve analyses applied previously validated pain outcomes to determine subgroup outcome trajectories for the proportion of opioid users and oral morphine equivalent (OME) dosages. Substantial trajectory separation was found for opioid use and OME. Specifically, the average OME dosage for the persistent moderate pain subgroup was more than double that for the other outcome subgroup. The average preoperative opioid daily OME dosage for 170 patients reporting opioid use was 24.94 (95% [confidence interval] CI = 20.52, 29.38). Bodily pain was consistently higher for the persistent moderate pain subgroup compared to the other subgroup. Outcome subgroups in patients with pain catastrophizing demonstrated substantial differences in opioid use and dosage and were predicted by high pain catastrophizing, more bodily pain, and changes in bodily pain over time. The persistent moderate pain subgroup is at greater risk of opioid use and greater opioid dosages and should be targeted for preoperative screening and interventions to reduce opioid use and potential opioid misuse. PERSPECTIVE: More frequent and higher opioid dosage following KA was found for the persistent moderate pain subgroup compared to the other subgroup. Patients with persistent pain had worse catastrophizing, contralateral and ipsilateral lower extremity pain, low back pain, and whole body pain compared to the rapidly improving mild pain subgroup.

A Latent Change Score Approach to Understanding Chronic Bodily Pain Outcomes Following Knee Arthroplasty: A Secondary Analysis of Longitudinal Data.

BACKGROUND: The extent to which chronic bodily pain changes following total knee arthroplasty (TKA) is unknown. We determined the extent of chronic bodily pain changes at 1 year following TKA. METHODS: Data from our randomized trial of pain coping skills, which revealed no effect of the studied interventions, were used. The presence and severity of chronic pain in 16 body regions, excluding the surgically treated knee, were determined prior to and 1 year following surgery. The Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain scale was used to quantify the extent of surgical knee pain. Latent change score (LCS) models were used to determine the extent to which true chronic bodily pain scores change after TKA. RESULTS: The mean age of the sample of 367 participants was 63.4 ± 8.0 years, and 247 (67%) were female. LCS analyses showed significant 20% to 54% reductions in pain in the surgically treated lower limb (not including the surgically treated knee), pain in the non-surgically treated lower limb, and whole body pain. In bivariate LCS analyses, greater improvement in the WOMAC pain score, indicating surgical benefit of TKA, led to greater improvement in all 4 bodily pain areas beyond the surgically treated knee, even after controlling for the latent change in pain catastrophizing. CONCLUSIONS: Clinically important chronic bodily pain reductions occurred following TKA and may be causally linked to the surgical procedure. Reduction in chronic bodily pain in sites other than the surgically treated knee is an additional benefit of TKA. LEVEL OF EVIDENCE: Prognostic Level II . See Instructions for Authors for a complete description of levels of evidence.

Responder Analyses: A Methodological Mess.

SYNOPSIS: Responder analyses are methods for analyzing randomized controlled trials, which purport to identify individuals or subgroups of study participants who experienced a "clinically meaningful" improvement from a treatment. Unfortunately, responder analyses have numerous methodological shortcomings, which preclude inferences concerning individual response to treatments and, thus, adoption into clinical practice. In this Viewpoint, we summarize 2 major limitations of responder analyses: (1) their thresholds of success involve arbitrary criteria and (2) responder analyses do not capture true individual treatment effects. J Orthop Sports Phys Ther 2023;53(XX):1-3. Epub: 20 June 2023. doi:10.2519/jospt.2023.11853.

KOOS score maps were externally validated to inform knee arthroplasty shared decision making.

PURPOSE: The Knee injury and Osteoarthritis Outcome Survey (KOOS) profile of outcome measures are among the most commonly used outcome measures in knee arthroplasty (KA). The purpose was to develop and externally validate "score maps" (one-page figural depictions of most likely scores) for KOOS Pain and Function subscales to facilitate a variety of clinical decisions related to shared decision making prior to KA. METHODS: Presurgical KA data collected within 1 year of surgery and obtained in two independent studies were used in this cross-sectional study. Score maps were designed to be easily understandable, single-page graphical depictions of predicted KOOS Pain, and KOOS Function, daily activity subscales. To create the score maps, individual item scores from one dataset were used to determine the most probable responses for each item for the entire range of possible scores. Predicted KOOS score maps were derived from Osteoarthritis Initiative (OAI) data and externally validated using an independent single site KA cohort study. Score map predicted scores from OAI were compared to actual presurgical KOOS subscale scores using Weighted Kappa (Κw) agreement coefficients and actual versus predicted differences in scores. RESULTS: The score maps derived from OAI and applied to actual scores in the validation sample demonstrated moderate to substantial chance-corrected agreement for both KOOS Pain and KOOS Function, daily activity subscale items. For example, KOOS Pain score map scores applied to the external validation dataset showed chance-corrected agreement with Κw ranging from 0.43 to 0.73. Score maps predicted actual item scores within ± 1 point at least 94% of the time. Findings for the KOOS Function, daily activity subscale items were similar. CONCLUSIONS: Score maps derived from OAI data agreed with actual KOOS scores obtained on an independent dataset at an acceptable degree of precision. Easy-to-use KOOS Pain and Function, daily activity score maps have potential to facilitate a variety of important clinical decisions during discussions between patients and surgeons prior to KA. LEVEL OF EVIDENCE: Level III prognostic study.

Sequential and Comparative Evaluation of Pain Treatment Effectiveness Response (SCEPTER), a pragmatic trial for conservative chronic low back pain treatment.

BACKGROUND: Chronic low back pain (cLBP) is a common and highly disabling problem world-wide. Although many treatment options exist, it is unclear how to best sequence the multitude of care options to provide the greatest benefit to patients. METHODS: The Sequential and Comparative Evaluation of Pain Treatment Effectiveness Response (SCEPTER) trial uses a pragmatic, randomized, stepped design. Enrollment targets 2529 participants from 20 Veterans Affairs (VA) medical centers. Participants with chronic low back pain will first be randomized to one of three options: 1) an internet-based self-management program (Pain EASE); 2) a tailored physical therapy program (Enhanced PT); or 3) continued care with active monitoring (CCAM), a form of usual care. Participants not achieving a 30% or 2-point reduction on the study's primary outcome (Brief Pain Inventory Pain Interference (BPI-PI) subscale), 3 months after beginning treatment may undergo re-randomization in a second step to cognitive behavioral therapy for chronic pain, spinal manipulation therapy, or yoga. Secondary outcomes include pain intensity, back pain-related disability, depression, and others. Participants will be assessed every three months until 12 months after initiating their final trial therapy. Companion economic and implementation analyses are also planned. RESULTS: The SCEPTER trial is currently recruiting and enrolling participants. CONCLUSIONS: Trial results will inform treatment decisions for the stepped management of chronic low back pain - a common and disabling condition. Additional analyses will help tailor treatment selection to individual patient characteristics, promote efficient resource use, and identify implementation barriers of interventions. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT04142177.

Patient Acceptable Symptom State Versus Latent Class Analysis Outcome Classification: A Comparative Longitudinal Study of Knee Arthroplasty.

OBJECTIVE: To determine whether Patient Acceptable Symptom State (PASS), a single-item deterministic binary measure of pain and function outcome satisfaction, leads to better differentiation of outcome classification versus latent class analysis probability-based outcome subgroups 1 year after knee arthroplasty (KA). METHODS: We used data from Knee Arthroplasty Skills Training for Pain (KASTPain), a 1-year no-effect multicenter randomized clinical trial of participants with KA, along with prior work that developed and externally validated good and poor outcome trajectories. Confirmatory latent class analyses were conducted on 2 exemplar outcome measures (Euroquol visual analog scale single-item self-rated health and 4-item pain ratings) and compared with PASS scores. Separation of trajectories were used to compare good and poor latent class self-rated health/4-item pain trajectories and PASS score trajectories. RESULTS: Prevalence rates for poor outcomes were 10% for self-rated health and 20% for 4-item pain and PASS. Probabilistic latent class-derived classifications of self-rated health and 4-item pain outcomes outperformed PASS in separating growth trajectories. The effect size point estimates for 12-month 4-item pain scale score separation was approximately 3 times larger for latent class analyses as compared with PASS. CONCLUSIONS: When used for outcome classification, observed PASS scores consistently underperform relative to probabilistic latent class-derived subgroups of pain and self-rated health outcome. PASS is a weak substitute for probabilistic classification of other patient-reported outcome measures of KA outcome. Clinicians and researchers should rely on latent class analyses over PASS to differentiate between outcome subgroups after KA.

Incorporating Expected Outcomes Into Clinical Decision-Making for Total Knee Arthroplasty.

OBJECTIVE: Expected outcomes (e.g., expected survivorship after a cancer treatment) have improved decision-making around treatment options in many clinical fields. Our objective was to evaluate the effect of expected values of 3 widely available total knee arthroplasty (TKA) outcomes (risk of serious complications, time to revision, and improvement in pain and function at 2 years after surgery) on clinical recommendation of TKA. METHODS: The RAND/University of California Los Angeles appropriateness criteria method was used to evaluate the role of the 3 expected outcomes in clinical recommendation of TKA. The expected outcomes were added to 5 established preoperative factors from the modified Escobar appropriateness criteria. The 8 indication factors were used to develop 279 clinical scenarios, and a panel of 9 clinicians rated the appropriateness of TKA for each scenario as inappropriate, inconclusive, and appropriate. Classification tree analysis was applied to these ratings to identify the most influential of the 8 factors in discriminating TKA appropriateness classifications. RESULTS: Ratings for the 279 appropriateness scenarios deemed 34.4% of the scenarios as appropriate, 40.1% as inconclusive, and 25.5% as inappropriate. Classification tree analyses showed that expected improvement in pain and function and expected time to revision were the most influential factors that discriminated among the TKA appropriateness classification categories. CONCLUSION: Our results showed that clinicians would use expected postoperative outcome factors in determining appropriateness for TKA. These results call for further work in this area to incorporate estimates of expected pain/function and revision outcomes into clinical practice to improve decision-making for TKA.

Effects of psychological distress on the general health to self-reported pain and function outcome relationship in knee arthroplasty: A causal mediation study.

Objectives: We examined two potential causal pathways that could be intervention targets to enhance knee arthroplasty outcomes. Data from a no-effect trial of persons with moderate to high pain catastrophizing were used to determined whether pain catastrophizing, depressive symptoms causally mediate the effect of preoperative general health on postoperative knee pain and functional difficulty. Methods: We used natural-effects models to conduct causal mediation analyses using the preoperative dichotomized EQ-5D-5L general health measure as the exposure, 2-month postoperative pain catastrophizing, depressive symptoms, and localized knee pain as potential mediators, and 12-month dichotomized Western Ontario and McMaster's University Osteoarthritis Index (WOMAC) Pain and Function scores reflecting good versus poor outcome as the outcomes. Results: Estimates of the indirect (mediating) effect suggested that pain catastrophizing mediated the effect of preoperative general health on 12-month WOMAC pain score by increasing odds of a good outcome by 8% (natural indirect effect odds ratio â€‹= â€‹1.08, 95% CI: 0.88, 1.29). The direction of mediating effects and their magnitude were similar for depressive symptoms; Sensitivity analyses suggested similar magnitudes and mediating effects to those reported for the main analyses. Conclusions: Our findings suggested that pain catastrophizing and depressive symptoms have a mediating role on the effect of baseline general health on self-reported pain and function outcomes. These findings support the continued treatment of pain catastrophizing and depressive symptoms as viable targets for interventions to potentially enhance pain and function outcomes for patients with moderate to high levels of psychological distress prior to surgery.

The smallest worthwhile effect is superior to the MCID for estimating acceptable benefits of knee arthroplasty.

BACKGROUND AND OBJECTIVES: Traditionally, the minimal clinically important difference (MCID) is used to judge the meaningfulness of outcomes in total knee arthroplasty (TKA). However, MCID estimates do not consider patient costs, potential side effects, and inconveniences. MCIDs vary substantially across TKA studies and have several conceptual and psychometric problems. A more scientifically sound alternative for estimating benefits patients expect TKA is the smallest worthwhile effect (SWE), measured with the benefit-harm trade-off method. METHODS: We recruited 121 participants and followed them for 6 months after surgery. All participants completed Knee Injury and Osteoarthritis Score (KOOS) Pain and Function, and underwent an interview using the benefit-harm trade-off method. RESULTS: The absolute SWE at the 50th percentile (approximating the average patient) was 31 points KOOS Pain improvement and 28 points for KOOS Function, daily living. Construct validity was supported with strong associations between meeting SWE (yes or no) and satisfaction with 6-month outcome (yes or no) using Pearson Chi Square (24.5, P < 0.001). CONCLUSION: Current MCID-based methods for interpreting KOOS change scores have flaws while the SWE method is conceptually superior. We determined the SWE of TKA that would justify the costs, risks, and inconveniences of surgery. SWE estimates suggest that patients expect a substantially greater change in KOOS scores than would be expected using MCID estimates. Clinicians can use SWE estimates when discussing likely outcomes and potential TKA benefits and risks with their patients.

Development of a score map to guide interpretation of WOMAC Pain scores prior to knee arthroplasty.

BACKGROUND: Scores from patient reported outcome measures (PROMs) are challenging to interpret for both clinicians and patients. PROMs obtained prior to knee arthroplasty (KA) could be used to inform important decisions related to KA made by both patients and clinicians. The purpose of this study was to develop a "score map" to allow for efficient and meaningful use of PROMs scores for patients considering KA. METHODS: Knee arthroplasty data obtained between one day and twelve months preoperatively from two multicenter studies were combined and used to develop and test the accuracy of a Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Pain Scale score map. To develop the score map, individual item scores were used to determine the most probable responses to items for the entire range of possible WOMAC Pain scores. Predicted WOMAC Pain scores, using the most probable response for each possible score on the score map, were compared to actual presurgical WOMAC Pain scores using Weighted Kappa (Κw) agreement coefficients. The score map is an easy-to-use graphical display of the entire range of WOMAC Pain scores from no pain to extreme pain for each item comprising the WOMAC Pain scale. RESULTS: Data from 780 patients were used in the analyses. The score map predicted WOMAC Pain scores and showed substantial agreement with actual WOMAC Pain scores Κw = 0.68 (95 %CI = 0.58, 0.77) to Κw = 0.77 (95 % CI = 0.75, 0.79). Perfect prediction of actual scores occurred between 55.1 % and 62.5 % of the time for all WOMAC Pain items. CONCLUSION: The WOMAC Pain score map has potential for facilitating a variety of important clinical decisions and discussions between patients and practitioners during healthcare encounters related to KA candidacy. For example, by comparing a patents' scores to literature-based estimates, patients may better understand how their WOMAC Pain scores compare to other persons who underwent KA, how much a score may change, on average, after surgery and whether this change might be acceptable to them.

Examination of Randomized Trials and Corresponding Trial Registry Entries: Registration Timing and Primary Outcome Analysis in the Journal of Arthroplasty.

BACKGROUND: Prospective trial registration enhances transparency and rigor of trial reporting. We conducted an in-depth examination of randomized clinical trials (RCTs) published in The Journal of Arthroplasty (JOA) from 2010 to 2020 and their associated trial registries. METHODS: We examined all RCTs published in the JOA during the even years between 2010 and 2020. We determined the proportion of trials that were registered and prospectively registered as well as the extent of consistency between primary outcome characteristics in the trials vs the registries. Trial characteristics published between 2010 and 2014 were compared to trials published between 2016 and 2020. RESULTS: A total of 57 (33.7%) of 169 primary RCTs over the study period reported being registered and of these, 20 (11.8%) were prospectively registered. For the registered primary RCTs, 75% reported primary outcome findings that were inconsistent with the corresponding registry. Trial registration proportion substantially improved from 13.6% between 2010 and 2014 to 53% between 2016 and 2020 (z-test = -5.315, P < .001). CONCLUSION: High proportions of retrospectively registered or unregistered trials and a very high proportion of inconsistencies in reporting of primary outcomes compared to the trial registries were found. These data argue for a well-developed strategy by JOA to enhance editorial policies, reviewer and editorial board member training and oversight, and improved arthroplasty researcher awareness to improve the current state of RCT reporting in JOA.