Improvements In Patient-reported Outcomes Research Articles

Hip Arthroscopy for Femoroacetabular Impingement Syndrome Shows Good Outcomes and Low Revision Rates, With Young Age and Low Postoperative Pain Score Predicting Excellent 5-Year Outcomes

To evaluate the clinical outcomes of hip arthroscopy for femoroacetabular impingement syndrome (FAIS) and their predictors at a minimum 5 years' follow-up. We retrospectively analyzed patients with FAIS after first-time unilateral hip arthroscopy between January 2010 and July 2016. Patient-reported outcomes (PROs) included the validated modified Harries Hip Score (mHHS) and Visual Analog Scale for Pain (Pain VAS). We included patients with Tönnis grade 0 or 1 and reported PROs, and excluded patients with previous hip diseases or bilateral symptoms. Bivariate and multivariate analyses were used for data analysis. We included 159 patients with a mean follow-up of 6.4 years, aged 36.18 ± 8.61 years, 41.5% female, and a mean body mass index of 23.61 ± 3.45. The mean postoperative mHHS was 88.82 ± 11.60, and the mean Pain VAS was 1.93 ± 1.89, significantly better than before surgery (P < .001). Postoperative alpha angle (P= .003) and lateral center edge angle (P < .001) were significantly decreased. Most patients (83.7%) achieved clinically important improvement based on patient-acceptable symptom state and minimal clinically important difference (MCID). The overall revision surgery rate was 2.5%. There were no conversions to total hip arthroplasty. Bivariate analysis indicated that age (P < .001), preoperative mHHS (P= .002), and postoperative Pain VAS (P <.001) correlated with postoperative mHHS at a minimum 5 years' follow-up. Multivariate regression analysis of MCID showed that age (P <.001), preoperative PROs (P < .01 for both), and postoperative Pain VAS (P < .001) were significant outcome predictors. Patients with FAIS after first-time unilateral hip arthroscopy showed significant improvement in PROs at mid-term follow-up, with a low revision surgery rate. Young patients and those with low postoperative Pain VAS showed excellent outcomes at a minimum 5 years' follow-up. Level IV, retrospective case series.

Impact of Infliximab-dyyb (Infliximab Biosimilar) on Clinical and Patient-Reported Outcomes: 1-Year Follow-up Results from an Observational Real-World Study Among Patients with Inflammatory Bowel Disease in the US and Canada (the ONWARD Study).

IntroductionTo date, there are limited real-world studies published on the use of infliximab-dyyb, a biosimilar to reference product (RP) infliximab approved for the treatment of moderate to severe inflammatory bowel disease (IBD), including Crohn’s disease (CD) and ulcerative colitis (UC) in North America. This study examined utilization patterns and the effects of infliximab-dyyb on clinical outcomes, patient-reported outcomes (PROs), and healthcare resource use (HCRU) in IBD patients in a real-world setting.MethodsIn this prospective, observational study, adult IBD patients in the US and Canada were recruited to initiate treatment with infliximab-dyyb and followed for 12 months. Patients included biologic-naïve users of infliximab-dyyb and patients switching from RP infliximab or other biologics to infliximab-dyyb. Partial Mayo (pMAYO) and Harvey Bradshaw Index (HBI) scores measured clinical outcomes for the UC and CD cohorts, respectively. Key PRO measures included the SIBDQ, EQ-VAS, and psychological outcomes. In addition, work productivity, HCRU, and adverse events (AEs) were assessed.ResultsA total of 67 CD and 48 UC patients were enrolled (51% female; mean age 44 years; 87% Caucasian; mean BMI 27.9). Thirty-nine patients were biologic-naïve, 57 switched from RP infliximab, and 19 switched from other biologics. Among UC biologic-naïve users, pMAYO decreased from 5.67 to 1.09 (p < 0.0001) and the remission rate increased from 5.6 to 90.9% (p = 0.0015). For UC patients switching from RP infliximab, pMAYO decreased from 1.38 to 0.29 (p = 0.0103). For CD biologic-naïve users, HBI scores and remission rates did not significantly change. The scores on all the PROs significantly improved from baseline to 12 months. A total of 22 AEs occurred consistent with the known AE profile for infliximab.ConclusionsClinical outcomes among biologic-naïve users of infliximab-dyyb improved for UC and were maintained for CD patients. Biologic-naïve users of infliximab-dyyb showed significant improvements in PROs. Patients switching from RP infliximab to infliximab-dyyb maintained their clinical outcomes and PROs.Trial RegistrationClinicalTrials.gov Registration Number: NCT03801928 (February 23, 2018).Supplementary InformationThe online version contains supplementary material available at 10.1007/s12325-022-02104-6.

The effect of secukinumab on patient-reported outcomes in patients with active psoriatic arthritis in a randomised phase 3 trial.

The phase 3 FUTURE 5 trial (NCT02404350) showed the clinical and radiographical efficacy of secukinumab in patients with psoriatic arthritis. This analysis aimed to assess the effect of secukinumab on patient-reported outcomes (PROs). FUTURE 5 was a phase 3, multicentre, parallel-group randomised trial in which patients who were 18 years old or older, met the classification criteria for psoriatic arthritis at screening, and had symptoms of moderate-to-severe psoriatic arthritis for at least 6 months were randomly assigned to receive secukinumab 300 mg, 150 mg, 150 mg no loading dose (NL), or placebo weekly from baseline to week 4 and every 4 weeks thereafter. The prespecified PROs of the FUTURE 5 trial were assessed first in the overall population. We report mean changes from baseline and the proportion of patients reporting improvements equal to or more than the minimum clinically important differences (MCIDs) and scores equal to or more than the normative values for patient global assessments (PtGA) of disease activity; psoriasis and arthritis visual analogue scale (VAS) scores; pain VAS; Health Assessment Questionnaire Disability Index (HAQ-DI); 36-item Short Form Health Survey (SF-36); Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F); and quality of life questionnaires. Patients were then stratified and assessed according to their tumour necrosis factor (TNF) inhibitor status (TNF-naive and TNF-inadequate responder [TNF-IR] populations) as a post-hoc analysis. Patients in all secukinumab groups reported significant least-squares mean changes from placebo at week 16 in all PROs except SF-36 mental component summary (MCS), irrespective of TNF inhibitor use. These included PtGA (300 mg difference vs placebo -12·2 [95% CI -16·3 to -8·1], 150 mg -8·22 [-12·4 to -4·1], 150 mg NL -8·3 [-12·5 to -4·2]; all p<0·0001), pain VAS (300 mg -14·3 [-18·3 to -10·2], 150 mg -11·5 [-15·6 to -7·5], 150 mg NL -11·3 [-15·3 to -7·2]; all p<0·0001), HAQ-DI (300 mg -0·33 [-0·42 to -0·24], 150 mg -0·23 [-0·32 to -0·14], 150 mg NL -0·24 [-0·33 to -0·15]; all p<0·0001), and FACIT-F (300 mg 4·8 [3·2 to 6·4], 150 mg 4·2 [2·6 to 5·8], 150 mg NL 3·5 [1·9 to 5·1]; all p<0·0001). Similarly, the proportion of patients with improvements equal to or better than MCID at week 16 was higher in the secukinumab group compared with the placebo group for most PROs except SF-36 (MCS), regardless of TNF inhibitor use. Secukinumab resulted in early, statistically significant, clinically meaningful, sustained improvements in PROs across all doses compared with placebo in patients with active psoriatic arthritis. These improvements were seen irrespective of previous TNF inhibitor use, in a post-hoc analysis. These results indicate that secukinumab provides comprehensive improvement for patients with psoriatic arthritis, regardless of previous therapy. Novartis.

122 ECAP-Controlled Closed-Loop Spinal Cord Stimulation (CL SCS): Double-Blind, Randomized Trial for the Treatment of Chronic Pain – 24-Month Outcomes

INTRODUCTION: Evoked compound action potential (ECAP) recording provides an objective measure of spinal cord (SC) activation during SCS and can assist in programing of the SCS system. ECAP-controlled CL SCS, in which the stimulation output is automatically adjusted to maintain a desired ECAP amplitude, has been shown to provide effective pain relief in an open-label study (ACTRN12615000713594). METHODS: 134 subjects were enrolled and randomized after trial leads were implanted. The primary endpoint evaluated =50% reduction in overall back and leg pain measured by the Visual Analog Scale (VAS). Opioid usage and other patient-reported outcomes (PROs) including emotional/physical functioning, sleep quality, and quality of life were also collected. Additionally, objective neurophysiological data, including SC activation and time spent in the therapeutic range, were collected. RESULTS: Herein the Evoke Study Group reports the 24-month outcomes from this ongoing RCT. The proportion of implanted subjects with =50% overall back and leg pain reduction at 24 months was statistically superior in the CL vs. OL group (84.0% vs. 65.9% subjects, respectively; p = 0.040). Long-term improvements in all other PROs, including POMS, ODI, PSQI, EQ-5D-5L, and SF-12, were also demonstrated. Opioids were reduced or eliminated in 66.7% (CL) and 60.9% (OL), respectively. The most frequent level of SC activation was three times greater for CL (median ECAP Amplitude: 22.5 µV CL vs. 7.5 µV OL). SC activation was better maintained within the therapeutic range with CL (median: 93.9% CL vs. 46.1% OL). There were no differences in the safety profiles between treatment groups, and the type, nature, and severity of adverse events were similar to other SCS studies. CONCLUSION: In this ongoing study, ECAP-controlled CL SCS provided statistically superior pain relief and greater improvement in other PROs compared with OL SCS at 24 months. CL delivered greater levels of SC activation and better maintained SC activation in the subject’s therapeutic range.

Open Fixation of Displaced Greater Tuberosity Fractures Yields Similar Patient-Reported Outcomes to Acute Arthroscopic Rotator Cuff Repair at 2 Years: A Matched Cohort Analysis

PurposeTo evaluate and compare patient-reported outcomes (PROs) after isolated greater tuberosity (GT) fracture fixation versus acute rotator cuff repair (RCR) at a minimum of 2 years.MethodsPatients who underwent isolated GT fracture fixation were compared in a 1-to-3 fashion with patients who underwent arthroscopic RCR for an acute rotator cuff tear by a single surgeon from January 2006 and to July 2018. Data were prospectively collected and retrospectively reviewed. PROs were compared pre- and postoperatively as well as between groups (American Shoulder and Elbow Surgeons [ASES], General Health Short Form-12 Physical Component [SF-12 PCS], Single Assessment Numerical Evaluation [SANE], Quick Disabilities of the Arm, Shoulder, and Hand [QuickDASH], and satisfaction). Reoperation rates were analyzed.ResultsA total of 57 patients (14 with isolated GT fracture fixation, mean age 45.7 years; and 43 who underwent ARCR for acute tears, mean age 56.6 years) were evaluated (P = .050). ASES scores significantly improved from 39.7 to 94.1 (P = .018) in the isolated GT fracture fixation group and from 51.0 to 95.2 (P < .001) in acute RCR group. At final follow-up, mean QuickDASH scores were 8.9 and 7.9 (P = .677) and SANE scores were 91.1 and 87.3 (P = .616) for the GT and acute RCR groups, respectively. The median satisfaction was 10/10 for the GT group and 10/10 for the RCR group. Additional comparison of patients who underwent double-row repair for an acute rotator cuff tear or isolated GT fracture revealed no significant difference in outcomes (P > .404).ConclusionMinimum 2-year PROs after fixation of isolated GT fractures show relatively high outcome scores whether treated by open reduction and internal fixation or arthroscopic fixation using a double-row bridging technique. The improvements in PROs are similar to those achieved with acute rotator cuff tears that were fixed arthroscopically with RCR. Further analysis of these results suggest that the functional outcomes of tendon-to-bone healing with linked, double-row rotator cuff repairs are similar to those of bone-to-bone healing as seen with GT fractures.Level of EvidenceIII, retrospective comparative study.

Patient-reported outcomes, health-related quality of life, and acute medication use in patients with a\u2009\u2265\u200975% response to eptinezumab: subgroup pooled analysis of the PROMISE trials

BackgroundPROMISE-1 and PROMISE-2 evaluated the preventive efficacy, tolerability, and safety of eptinezumab, a calcitonin gene-related peptide–targeted monoclonal antibody, in adults with episodic (EM) and chronic migraine (CM), finding significant reductions in migraine frequency. This post hoc analysis compared patient-reported outcomes (PROs), health-related quality of life (HRQoL) and acute medication use in patients with a ≥ 75% migraine responder rate (MRR) after treatment with eptinezumab to patients with a ≥ 50– < 75% MRR.MethodsPROMISE-1 and PROMISE-2 were phase 3, randomized, double-blind, placebo-controlled studies. This analysis included patients from both studies treated with eptinezumab 100 mg or 300 mg who experienced ≥ 75% and ≥ 50–< 75% MRR over Weeks 1–12 (wks1–12). In both studies, HRQoL was measured by the 36-item Short-Form Health Survey (SF-36) and acute medication usage. PROMISE-2 also included the 6-item Headache Impact Test (HIT-6), patient-identified most bothersome symptom (PI-MBS), and Patient Global Impression of Change (PGIC).ResultsIn PROMISE-1, a total of 115/443 (26.0%; 100 mg, n = 49, 300 mg, n = 66) and 120/443 (27.0%; 100 mg, n = 61, 300 mg, n = 59) eptinezumab-treated patients achieved ≥ 75% and ≥ 50–< 75% MRR over wks1–12, respectively. In PROMISE-2, a total of 211/706 (30.0%; 100 mg, n = 95; 300 mg, n = 116) and 209/706 (29.6%; 100 mg, n = 110, 300 mg, n = 99) eptinezumab-treated patients achieved ≥ 75% and ≥ 50–< 75% MRR over wks1–12, respectively. EM and CM patients with ≥ 75% and ≥ 50–< 75% MRR over wks1–12 showed reduced use of acute headache medication and increased HRQoL to normative levels across SF-36 domains of bodily pain, social functioning, and physical functioning. In CM patients with ≥ 75% and ≥ 50–< 75% MRR over wks1–12, the mean change in HIT-6 total score with eptinezumab (pooled) was − 11.7 and − 7.6, respectively. “Very much” or “much” improvement responses were reported in 41.8% and 16.5% on PI-MBS and 36.2% and 20.0% on PGIC in ≥ 75% and ≥ 50–< 75% MRR, respectively.ConclusionEptinezumab treatment induced a ≥ 75% MRR over wks1–12 in the majority of patients. This patient subgroup reported substantial improvements in PROs associated with headache-related life impact and HRQoL, and reductions in acute headache medication use, which were more marked than those in the ≥ 50–< 75% responders. This study supports the clinical meaningfulness of ≥ 75% MRR for patients with either EM or CM.Trial registrationClinicalTrials.gov identifiers: NCT02559895 (PROMISE-1), NCT02974153 (PROMISE-2).

Influence of Self-Identified Gender on Clinical Outcomes and Postoperative Patient Satisfaction After Lumbar Decompression: Cohort-Matched Analysis

We compared the patient-reported outcomes (PROs), minimal clinically important difference (MCID) achievement, and perceived postoperative satisfaction after minimally invasive lumbar decompression of patients stratified by self-identified gender. Patients who had undergone single minimally invasive lumbar decompression were identified. The PRO measures were administered preoperatively and postoperatively and included the PRO measurement information system-physical function, visual analog scale (VAS) for back and leg pain, Oswestry disability index (ODI), and 12-item short form physical and mental component scores. The patients were grouped by self-identified gender. Propensity score matching was performed. The mean PROs and postoperative satisfaction scores were compared between cohorts using a 2-sample t test. The postoperative PRO improvement within each cohort was calculated using a paired t test. MCID achievement was determined by comparison to previously established threshold values. The MCID achievement rates were compared among the groups using simple logistic regression. A total of 128 propensity score-matched patients were included: 44 in the female group and 84 in the male group. The male group demonstrated worse VAS scores for back pain at 12 weeks and a worse ODI at 6 weeks (P < 0.046 for all). The female cohort had achieved greater rates of a MCID for the ODI at 6 months (P < 0.049). Patients in the self-identified female group demonstrated higher levels of postoperative satisfaction for the VAS score for leg pain at 6 and 12 weeks), the VAS score for back pain at 12 weeks, and the ODI at 6 and 12 weeks (P < 0.028 for all). Additionally, patients in the self-identified female group demonstrated greater levels of satisfaction for lifting at 6 and 12 weeks (P < 0.014 for all). Despite the similar preoperative baseline values, postoperative improvement, and clinical outcomes, our results suggest that the self-identified male patients will have poorer short-term satisfaction for disability, leg pain, back pain, and lifting versus patients in the self-identified female group. Self-identified gender might influence patient satisfaction and could be attributed to differing preoperative expectations at baseline for short-term recovery.

High Body Mass Index Does Not Adversely Affect Outcomes in High-Level Athletes Undergoing Primary Hip Arthroscopy: A Propensity-Matched Comparison With Minimum 2-Year Follow-up

Background: The effect of high body mass index (BMI) on outcomes in athletes has not been established. Purpose: (1) To report minimum 2-year patient-reported outcome (PRO) scores and return to sports (RTS) for high-level athletes with high BMI undergoing hip arthroscopy for femoroacetabular impingement syndrome (FAIS) and (2) to compare results with a propensity-matched control group of high-level athletes with a normal BMI. Study Design: Cohort study; Level of evidence, 3. Methods: Data were collected on all professional, collegiate, and high school athletes who had a high BMI (>30) and who had undergone primary hip arthroscopy for FAIS between January 2010 and December 2018. RTS status and minimum 2-year PROs were collected for the modified Harris Hip Score (mHHS), Non-Arthritic Hip Score (NAHS), Hip Outcome Score–Sports Specific Subscale (HOS-SSS), and visual analog scale (VAS) for pain. The percentage of patients achieving the minimal clinically important difference (MCID) and Patient Acceptable Symptom State (PASS) were also recorded. These patients were propensity matched in a 1:3 ratio to high-level athletes with a normal BMI for comparison. Results: A total of 30 high-level athletes with a high BMI were included with a mean follow-up of 49.4 ± 29.5 months. They demonstrated significant improvement from preoperatively to latest follow-up for mHHS, NAHS, HOS-SSS, and VAS (P < .001). When outcomes were compared with a propensity-matched control group of 90 athletes with a normal BMI, athletes with a high BMI had worse acetabular cartilage injury and were more likely to undergo acetabular microfracture (P < .001). Athletes with a high BMI demonstrated lower postoperative scores for NAHS when compared with athletes with a normal BMI (88.06 ± 9.37 [range, 60-100] and 90.25 ± 10.79 [range, 48.75-100], respectively; P = .049). Athletes with a high BMI also demonstrated worse postoperative scores for HOS-SSS when compared with athletes with a normal BMI (77.22 ± 18.31 [range, 22.22-100] and 82.38 ± 22.79 [range, 2.78-100], respectively; P = .038). Rates of achieving MCID for the high-BMI and normal-BMI groups were comparable in mHHS (90.0% and 77.8%, respectively; P = .185) and HOS-SSS (90.0% and 82.2%, respectively; P = .397). PASS rates were also comparable between the high- and normal-BMI groups for mHHS (90.0% and 87.8%, respectively; P > .999) and HOS-SSS (70.0% and 71.1%, respectively; P = .908). Athletes with a high BMI also returned to sports at a lower rate compared with athletes with a normal BMI, but this did not reach statistical significance (P = .479). Conclusion: Athletes with a high BMI undergoing primary hip arthroscopy for FAIS demonstrated significant improvement in PROs and favorable rates achieving clinically meaningful improvement. When compared with a control group of high-level athletes with a normal BMI, they exhibited similar rates of achieving psychometric thresholds and RTS rates. At short-term follow-up, high BMI did not adversely affect outcomes of high-level athletes undergoing primary hip arthroscopy.

Maintenance of Patient-Reported Outcomes in Baricitinib-Treated Patients with Moderate-to-Severe Active Rheumatoid Arthritis: Post Hoc Analyses from Two Phase 3 Trials.

IntroductionBaricitinib has been shown to improve patient-reported outcomes (PROs) in patients with rheumatoid arthritis (RA) who are inadequate responders (IR) to conventional synthetic and biologic disease-modifying antirheumatic drugs (csDMARDs and bDMARDs, respectively). We assessed the ability of baricitinib 2-mg to maintain minimal clinically important differences (MCIDs) in PROs until week 24 among week 4 and 12 responders.MethodsData were from two phase 3 trials, RA-BUILD (NCT01721057; csDMARD-IR patients) and RA-BEACON (NCT01721044; bDMARD-IR patients). PROs included Pain Visual Analogue Scale, Health Assessment Questionnaire-Disability Index, Functional Assessment of Chronic Illness Therapy-Fatigue, Short-Form 36 Physical Component Score, and Patient’s Global Assessment of Disease Activity. Outcomes were evaluated by proportions of patients achieving MCID improvements, number needed to treat (NNT) at weeks 4, 12, and 24, proportions of patients maintaining MCID responses at week 24 among week 4 or 12 responders, and median time to achieve substantial response with baricitinib 2-mg versus placebo.ResultsA higher proportion of baricitinib-treated patients achieved MCID improvements, with NNTs ranging from 5 to 8 for baricitinib 2-mg versus placebo at week 24. Generally, early MCID responses in PROs at weeks 4 or 12 were better maintained through week 24 in RA patients treated with baricitinib 2-mg versus placebo. Patients treated with baricitinib 2-mg also achieved substantial PRO responses or normative values more quickly than placebo.ConclusionsThese results suggest baricitinib-treated patients with RA achieving MCID improvement in PROs at weeks 4 and 12 maintained those improvements over time and that substantial PRO responses were achieved quickly.

The impact of filgotinib on patient-reported outcomes and health-related quality of life for patients with active rheumatoid arthritis: a post hoc analysis of Phase 3 studies

BackgroundThe effects of filgotinib on patient-reported outcomes (PROs) from 3 trials in patients with active rheumatoid arthritis were investigated.MethodsMethotrexate (MTX)-naïve patients received filgotinib 200 or 100 mg plus MTX (FIL200+MTX, FIL100+MTX), filgotinib 200 mg monotherapy (FIL200), or MTX monotherapy through 52 weeks (NCT02886728). Patients with inadequate response (IR) to MTX (MTX-IR) received FIL200+MTX, FIL100+MTX, adalimumab 40 mg +MTX (ADA+MTX), or placebo (PBO)+MTX (rerandomized to FIL200+MTX or FIL100+MTX at week 24) through 52 weeks (NCT02889796). Patients with IR to biologic disease-modifying antirheumatic drugs (bDMARD-IR) received FIL200 or FIL100 or PBO with background stable conventional synthetic (cs) DMARDs for up to 24 weeks (NCT02873936). PROs included Health Assessment Questionnaire-Disability Index (HAQ-DI), Medical Outcomes Study 36-Item Short Form Health Survey (SF-36) physical/mental component summary (PCS/MCS), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), Work Productivity and Activity Impairment Questionnaire-Rheumatoid Arthritis (WPAI-RA), and Patient Global Assessment of Disease Activity (PtGA). Data are reported as least-squares mean changes from baseline with standard error to the timepoint representing each study’s primary endpoint. All statistical comparisons are of filgotinib groups vs their respective control groups.ResultsAt week 24, among MTX-naïve patients, change from baseline (standard deviation) in HAQ-DI was − 1.00 (0.03; P < 0.001) with FIL200+MTX, − 0.94 (0.04; P < 0.01) with FIL100+MTX, and − 0.91 (0.04; P < 0.05) with FIL200 alone compared with − 0.81 (0.03) with MTX alone. At week 12, among MTX-IR patients, change from baseline in HAQ-DI was − 0.69 (0.04; P < 0.001 vs PBO+MTX, P < 0.05 vs ADA) with FIL200+MTX, − 0.57 (0.04; P < 0.001 vs placebo) with FIL100+MTX, and − 0.60 (0.04) with ADA vs − 0.40 (0.04) with PBO+MTX. At week 12, among bDMARD-IR patients, change from baseline in HAQ-DI was − 0.50 (0.06; P < 0.001) with FIL200+csDMARD and − 0.46 (0.05; P < 0.001) with FIL100+csDMARD vs − 0.19 (0.06) with placebo+csDMARD. Changes in SF-36 PCS and MCS, FACIT-Fatigue, WPAI, and PtGA tended to favor filgotinib over PBO, MTX, and ADA. Greater proportions of patients experienced clinically meaningful differences with either dosage of FIL in combination with csDMARDs (including MTX) and with FIL200 monotherapy vs comparators.ConclusionsFilgotinib provided improvements in PROs across patient populations. These findings suggest filgotinib can be an effective treatment option for patients with insufficient response to MTX or bDMARDs and patients who are MTX-naïve.Trial registrationClinicalTrials.gov, FINCH 1, NCT02889796, first posted September 7, 2016; FINCH 2, NCT02873936, first posted August 22, 2016, retrospectively registered; FINCH 3, NCT02886728, first posted September 1, 2016, retrospectively registered.

Patient-reported outcome after primary and aseptic revision hip arthroplasty: 1-year follow-up of 3,559 primary and 406 revision THAs in an institutional registry.

Background and purposePatient-reported outcomes (PROMs) after primary total hip arthroplasty (THA) and revision THA are important information in the preoperative shared decision-making process. We present 1-year results on pain, function, and quality of life following primary and revision THA.Patients and methodsFrom 2010 to 2018, 3,559 primary THA and 406 revision THAs were included in our institutional quality registry. PROMs were registered preoperatively, 3 months, and 1 year after surgery, numeric rating scale (0–10) for pain during mobilization and at rest, health-related quality of life (EQ-5D), and a hip-specific physical function score (HOOS-PS). 2 anchor questions were asked 1 year after surgery concerning joint function and willingness to go through surgery again.ResultsThere were statistically significant improvements in all PROMs at the 3-month follow-up in both groups. All PROMs improved more in the primary group relative to the revision group. 1 year after surgery, pain during mobilization was reduced with a mean change of 5.1 (SD 2.6) for primary THA and 2.9 (SD 3.0) for revision THA. 93% of primary THA patients reported both better function 1 year after surgery and that they would have gone through surgery again, compared with 78% and 79% in the revision THA group.InterpretationPrimary THA patients reported better function and more pain relief than the revision THA group 1 year after surgery. Pain during mobilization shows the most marked improvement in both groups, which is important preoperative information for patients.

Delayed treatment with a tumor necrosis factor alpha blocker associated with worse outcomes in patients with spondyloarthritis: data from the Czech National Registry ATTRA.

Introduction:The administration of biologic disease-modifying antirheumatic drugs, including tumor necrosis factor (TNF)-α inhibitors, is observed to interfere with the disease activity and progression. In this study, we aimed to assess the effectiveness and response predictors of adalimumab (ADA), a TNF-α blocker, in patients with axial spondyloarthritis (AxSpA).Methods:This study was a historical prospective, registry-based observational study on patients with AxSpA treated with first-line ADA after conventional drug failure. For evaluation and comparison, patients were divided into three groups according to the number of years from AxSpA diagnosis to initiation of ADA treatment: (A) <5 years, (B) 5–10 years, and (C) >10 years. The assessment instruments ankylosing spondylitis disease activity score (ASDAS), Bath ankylosing spondylitis activity index (BASDAI), Bath ankylosing spondylitis functional index (BASFI), health assessment questionnaire (HAQ), Short Form 36 questionnaire (SF-36), and EuroQoL 5 dimension questionnaire (EQ-5D) were regularly administered for up to 24 months of follow-up.Results:This study included 1043 patients with AxSpA (9.2% with non-radiographic AxSpA, 68.9% men). By month 6, a significantly higher proportion of patients with ASDAS remission (<1.3) was achieved upon earlier intervention in group A (30.1%) and B (32.9%) than in the late intervention group C (22.6%) (p ⩽ 0.05). At month 6, lower age and better BASFI at treatment initiation were identified as the strongest predictors of ASDAS remission in both univariable [odds ratio (OR): 0.956, p ⩽ 0.001; OR: 0.834, p ⩽ 0.001, respectively] and multivariable analyses (OR: 0.963, p ⩽ 0.001; OR: 0.859, p ⩽ 0.001, respectively). Earlier intervention also led to improvement in most patient-reported outcomes (PROs) based on HAQ, SF-36, and EQ-5D.Conclusion:Results from the ATTRA registry concur with previous clinical trials that supported efficacy of TNF-α blockers and showed better treatment outcomes with early interventions, including reduction of disease activity and improvement in PROs. We identified age and BASFI as the main factors influencing treatment effectiveness.

Study Protocol for a Multicenter Nationwide Prospective Cohort Study to Investigate the Natural Course and Clinical Outcome in Benign Liver Tumors and Cysts in The Netherlands: The BELIVER Study

Introduction: Benign liver tumours and cysts (BLTCs) comprise a heterogeneous group of cystic and solid lesions, including hepatic hemangioma, focal nodular hyperplasia and hepatocellular adenoma. Some BLTCs, for example (large) hepatocellular adenoma, are at risk of complications. Incidence of malignant degeneration or hemorrhage is low in most other BLTCs. Nevertheless, the diagnosis BLTC may carry a substantial burden and patients may be symptomatic, necessitating treatment. The indications for interventions remain matter of debate. The primary study aim is to investigate patient reported outcomes (PROs) of patients with BLTCs, with special regard to the influence of invasive treatment as compared to the natural course of the disease. Methods and analysis: A nationwide observational cohort study of BLTC patients will be performed between August 2021 and August 2026. Inclusion will be open from August 2021 until August 2025. During surveillance, a questionnaire regarding symptoms and their impact will be sent to participants on a biannual basis and more often in case of invasive intervention. The questionnaire was previously developed based on patient reported outcomes (PROs) considered relevant to patients with BLTCs and their caregivers. Most questionnaires will be administered by computerized adaptive testing through the Patient-Reported Outcomes Measurement Information System (PROMIS). Data, such as treatment outcomes, will be extracted from electronic patient files. Multivariable analysis will be performed to identify patient and tumour characteristics associated with significant improvement in PROs or a complicated postoperative course.

Association between low hemoglobin, clinical measures, and patient-reported outcomes in patients with rheumatoid arthritis: results from post hoc analyses of three phase III trials of sarilumab

BackgroundAnemia is common in patients with rheumatoid arthritis (RA). Higher hemoglobin (Hb) levels may be associated with better clinical outcomes and patient-reported outcomes (PROs). To assess this hypothesis, we conducted two post hoc analyses in three sarilumab phase III studies: TARGET, MOBILITY, and MONARCH.MethodsPooled data from combination therapy from placebo-controlled MOBILITY (sarilumab + methotrexate) and TARGET (sarilumab + conventional synthetic disease-modifying antirheumatic drugs [csDMARDs]) and monotherapy data from active-controlled MONARCH (sarilumab vs. adalimumab) studies were included. Associations between Hb levels and clinical measures and PROs were assessed over 24 weeks. The mean changes from baseline in clinical outcomes and PROs (to week 24) and radiographic outcomes (to week 52) were evaluated between low and normal Hb levels (based on the World Health Organization [WHO] criteria).ResultsFrom TARGET, MOBILITY, and MONARCH, 546, 1197, and 369 patients, respectively, were stratified according to Hb levels (low vs. normal). Over 24 weeks, higher Hb levels were found to be consistently associated with better clinical outcomes and PROs in combination therapy and monotherapy groups and were more pronounced among the patients treated with sarilumab than those treated with placebo and adalimumab. The mean change from baseline to week 24 in clinical efficacy measures and PROs was similar in patients with low vs. normal Hb at baseline. Differences between sarilumab and/or adalimumab, for all outcomes, were larger for low Hb subgroups. In MOBILITY, by week 52, the inhibition of progression of structural damage (assessed via Modified Total Sharp Score [mTSS]) was 84% (sarilumab 200 mg) and 68% (sarilumab 150 mg) vs. placebo in patients with low Hb and 97% (sarilumab 200 mg) and 68% (sarilumab 150 mg) vs. placebo in patients with normal Hb. Similar results were observed for other radiographic outcomes.ConclusionsIn these post hoc analyses, a consistent relationship was observed between higher Hb levels and better clinical outcomes and PROs in patients with RA. Irrespective of the baseline Hb levels, sarilumab treatment was associated with improvements in clinical measures and PROs over 24 weeks (improvements were more pronounced than those with adalimumab treatment) and mitigation of joint damage progression over 52 weeks.Trial registrationClinTrials.gov NCT01061736, NCT01709578, and NCT02332590

Efficacy and safety of guselkumab and adalimumab for pustulotic arthro-osteitis and their impact on peripheral blood immunophenotypes

ObjectivesWe compared the treatment effectiveness between guselkumab and adalimumab in patients with pustulotic arthro-osteitis (PAO). In addition, we performed peripheral blood immunophenotyping to elucidate the immunological background and analyzed the impact of therapeutic drugs to verify the validity of immunological phenotypes as therapeutic targets.MethodsPatients were treated with guselkumab 100 mg (guselkumab group; n = 12) and adalimumab 40 mg (adalimumab group; n = 13). Arthritis disease activity, skin lesion activity, and patient-reported outcomes (PROs) were evaluated and compared between the two groups. The retention rate and adverse events were evaluated. Comprehensive phenotyping of peripheral immune cells was performed in both groups, and phenotypes were compared before and after treatment.ResultsAt 6 months, both groups showed significant improvement in arthritis disease activity and PROs. In the guselkumab group, skin symptoms significantly improved. The 6-month continuation rates were 91.7% (11/12) and 69.2% (9/13) in the guselkumab and adalimumab groups, respectively. Adverse events occurred in 2/12 and 5/13 patients in the guselkumab (16.7%) and adalimumab (38.5%) groups, respectively. Peripheral blood immunophenotyping showed that the proportion of activated T helper (Th) 1 cells was significantly lower in patients with PAO than in healthy controls and that the proportion of activated Th17 cells was significantly higher in patients with PAO, which significantly decreased after treatment with guselkumab.ConclusionAlthough guselkumab and adalimumab have comparable efficacy for PAO, their impact on immunophenotypes varies.

Post Hoc Analysis of the Correlation Between Patient-Reported Outcomes and Clinical Response to Repository Corticotropin Injection for Persistently Active Rheumatoid Arthritis.

PurposeApproximately 6% of patients with rheumatoid arthritis (RA) in the USA have refractory disease that is resistant to standard-of-care therapies. A recent phase IV clinical trial affirmed the safety and efficacy of repository corticotropin injection (RCI; Acthar® Gel) for refractory RA. This post hoc analysis of the clinical trial data assessed whether changes in clinical measures correlated with patient-reported outcome (PRO) improvements.MethodsData were assessed from the trial’s open-label period when patients received RCI (80 U) twice weekly for 12 weeks. Clinical assessments included hemoglobin A1c, C-reactive protein, erythrocyte sedimentation rate (ESR), total joint count (TJC), swollen joint count (SJC), Disease Activity Score with 28 joint count and ESR (DAS28-ESR), and Clinical Disease Activity Index (CDAI). PROs included pain (Visual Analog Scale), fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue [FACIT-F]), disability (Health Assessment Questionnaire-Disability Index [HAQ-DI]), and activity impairment (Work Productivity and Activity Impairment [WPAI] questionnaire). Patients grouped by minimal clinically important difference (MCID) improvement vs no improvement in PROs were compared with clinical measures at week 12. Correlations were determined by multivariable linear regression analysis and standardized coefficient estimates.ResultsRCI responders, defined as patients with DAS28-ESR < 3.2 at week 12, reported significantly greater PRO improvements for pain, disability, fatigue, activity impairment, current work impairment, and overall work impairment than nonresponders. Patients with MCID improvements in all PROs showed significantly greater decreases in mean values for TJC, DAS28-ESR, and CDAI, whereas those with pain, fatigue, and disability improvements had significantly greater SJC and ESR reductions. Multivariable linear regression analysis determined that improvement from baseline in all PROs correlated with significant decreases in TJC, DAS28-ESR, and CDAI. ESR reduction significantly correlated with improvements in pain and disability, but not fatigue or WPAI.ConclusionsThese results confirm that clinical responses to RCI were directly correlated with patient perception of improvement.

Mobility-related outcomes for periacetabular osteotomy in persons with acetabular dysplasia: setting the stage for measurement of real-world outcomes.

ABSTRACTPeriacetabular osteotomy (PAO) is a surgery for persons with symptomatic acetabular dysplasia (AD) that increases acetabular coverage of the femoral head for reducing hip pain and improving function. Patient-reported outcomes (PROs) are significantly improved following PAO, yet little is known regarding mobility-related outcomes. This narrative review provides a synthesis of evidence regarding PROs and mobility-related outcomes in persons with AD following PAO. We further identified important future research directions, chiefly the need for measurement of real-world outcomes. We searched PubMed using comprehensive predefined search terms. We included studies that (i) enrolled persons with AD undergoing PAO, (ii) included PROs and/or mobility-related outcomes and (iii) were written in English. We synthesized and summarized study characteristics and findings. Twenty-three studies were included in this review. Commonly evaluated PROs included pain (n = 14), hip function (n = 19) and quality of life (n = 9). Mobility-related outcomes included self-reported physical activity (PA; n = 11), walking speed and cadence (n = 4), device-measured PA (n = 2), and sit-to-stand, four-square-step and timed stair ascent tests (n = 1). Persons with AD had significant improvements in PROs following PAO, yet mobility-related outcomes (e.g. walking speed and device-measured PA levels) did not change over 1 year following PAO. Few studies have evaluated mobility-related outcomes following PAO, and these studies were of a low methodological quality. Future research might include experience sampling data collection approaches and body-worn devices as free-living, technology-driven methodologies to evaluate mobility and other outcomes in persons with AD undergoing PAO.

Patient-Reported Outcomes Following Total Thyroidectomy for Graves' Disease.

Background: Graves' disease accounts for ∼80% of all cases of hyperthyroidism and is associated with significant morbidity and decreased quality of life. Understanding the association of total thyroidectomy with patient-reported quality-of-life and thyroid-specific symptoms is critical to shared decision-making and high-quality care. We estimate the change in patient-reported outcomes (PROs) before and after surgery for patients with Graves' disease to inform the expectations of patients and their physicians. Methods: PROs using the MD Anderson Symptom Inventory (MDASI) validated questionnaire were collected prospectively from adult patients with Graves' disease from January 1, 2015, to November 20, 2020, on a longitudinal basis. Survey responses were categorized as before surgery (≤120 days), short term after surgery (<30 days; ST), and long term after surgery (≥30 days; LT). Negative binomial regression was used to estimate the association of select covariates with PROs. Results: Eighty-five patients with Graves' disease were included. The majority were female (83.5%); 47.1% were non-Hispanic white and 35.3% were non-Hispanic black. The median thyrotropin (TSH) value before surgery was 0.05, which increased to 0.82 in ST and 1.57 in LT. In bivariate analysis, the Total Symptom Burden Score, a composite of all patient-reported burden, significantly reduced shortly after surgery (before surgery mean of 56.88 vs. ST 39.60, p < 0.001), demonstrating improvement in PROs. Furthermore, both the Thyroid Symptoms Score, including patient-reported thermoregulation, palpitations, and dysphagia, and the Quality-of-Life Symptom Score improved in ST and LT (thyroid symptoms, before surgery 13.88 vs. ST 8.62 and LT 7.29; quality of life, before surgery 16.16 vs. ST 9.14 and LT 10.04, all p < 0.05). After multivariate adjustment, the patient-reported burden in the Thyroid Symptom Score and the Quality-of-Life Symptom Score exhibited reduction in ST (thyroid symptoms, rate ratio [RR] 0.55, confidence interval [CI]: 0.42-0.72; quality of life, RR 0.57, CI: 0.40-0.81) and LT (thyroid symptoms, RR 0.59, CI: 0.44-0.79; quality of Life, RR 0.43, CI: 0.28-0.65). Conclusions: Quality of life and thyroid-specific symptoms of Graves' patients improved significantly from their baseline before surgery to both shortly after and longer after surgery. This work can be used to guide clinicians and patients with Graves' disease on the expected outcomes following total thyroidectomy.

Significant improvement after hip arthroscopy for femoroacetabular impingement in women.

This study was designed to evaluate outcomes in women after hip arthroscopy (HA) for femoroacetabular impingement syndrome (FAIS) and compare these results with men. Retrospective study of a prospective database of patients undergoing hip arthroscopy between 2015 and 2018. Inclusion criteria were patients between 18 and 50years of age, diagnosis of FAIS, complete clinical patient-reported outcomes (PROs), radiographic measurements, and underwent at least a 2-year HA follow-up. For each woman, two men underwent HA were matched in a 1:2 ratio based on age within 5years, and date of surgery within 6months. Diagnostic arthroscopy was performed to evaluate labral and chondral status. Radiographic evaluation, a self-administered Hip Outcome Score (HOS) questionnaire, with activity of daily living (ADL) and sports subscale (SSS), and a self-administered short version of the International Hip Outcome Tool (iHOT-12) were assessed at 6months, 12months, and then yearly. Clinical relevance was measured with the minimal clinical important difference (MCID), patient acceptable symptom state (PASS), and substantial clinical benefit (SCB) for HOS-ADL, HOS-SSS, and iHOT-12. One hundred and eighty-five patients met the inclusion criteria. Fifty-two women were matched with 104 men. Significant differences in terms of demographics, radiographic results, intraoperative findings and arthroscopic procedures were found between women and men. Mean follow-up was 48.4months in women and 50.2months in men. Both groups showed significant improvement from preoperative PROs to the latest follow-up. There were no significant differences between groups in preoperative PROs, latest follow-up PROs and PROs improvements. The difference in frequency of patients achieving MCID, PASS, and SCB was only significant greater for iHOT-12 PASS in women compared with men. Significant improvements in PROs in women after HA for FAIS at 4-year follow-up were found. Differences between women and men in PROs and rates of achieving MCID, SCB and PASS were only significant for iHOT-12 PASS. IV.

P085 CLINICAL AND LONGITUDINAL PATIENT REPORTED OUTCOMES AFTER TRANSVERSUS ABDOMINIS RELEASE: A SINGLE SURGEON EXPERIENCE

Abstract Aim Posterior component separation with transversus abdominis release (TAR) is a novel complex abdominal wall repair technique that maximizes medial myofascial flap advancement in a vascularized, pre-peritoneal plane. Here, we add to a growing body of literature on this technique by assessing longitudinal clinical and patient reported outcomes (PROs) after ventral hernia repair (VHR) with TAR. Material and Methods Adult patients undergoing VHR with TAR between 10/1/2015 and 01/15/2020 by a single surgeon were retrospectively identified. Patients with parastomal hernias and &amp;lt;12 months of follow-up were excluded. Clinical outcomes and PROs using the Abdominal Hernia Questionnaire (AQH) and Hernia Related Quality of Life Survey (HerQLes) were assessed. Results 57 patients were included with a median age and body mass index of 60 and 30.6 kg/m2, respectively. The average hernia defect was 384 cm2 [IQR 205-471], and all patients had retro-muscular mesh placed. The most common complications were delayed healing (19.3%) and seroma (14.0%). One patient required return to the OR for management of a complication and there were no cases of mesh infection or explantation. Previous hernia repair and concurrent panniculectomy were risk factors for developing any complication (p &amp;lt; 0.05). One patient (1.8%) recurred at a median follow-up of 25.7 months [IQR 18.2-42.1]. Significant improvement in disease-specific PROs was observed and maintained throughout the follow-up period (pre to post p &amp;lt; 0.05). Conclusions Longitudinal clinical and patient-reported outcomes after VHR with TAR are limited. We conclude that TAR is a safe and efficacious adjunct in the repair of complex hernia defects.