Improvements In Patient-reported Outcomes Research Articles

Prolonged Improvement in Patient-Reported Outcomes (PROs) and Well-being in Older Patients With Treatment-Naïve (TN) Chronic Lymphocytic Leukemia Treated With Ibrutinib (Ibr): 3-Year Follow-up of the RESONATE-2 Study

Prolonged Improvement in Patient-Reported Outcomes (PROs) and Well-being in Older Patients With Treatment-Naïve (TN) Chronic Lymphocytic Leukemia Treated With Ibrutinib (Ibr): 3-Year Follow-up of the RESONATE-2 Study

Quantitative Sensory Testing of Spinal Cord and Dorsal Root Ganglion Stimulation in Chronic Pain Patients

The physiological mechanisms underlying the pain-modulatory effects of clinical neurostimulation therapies, such as spinal cord stimulation (SCS) and dorsal root ganglion stimulation (DRGS), are only partially understood. In this pilot prospective study, we used patient-reported outcomes (PROs) and quantitative sensory testing (QST) to investigate the physiological effects and possible mechanisms of action of SCS and DRGS therapies. We tested 16 chronic pain patients selected for SCS and DRGS therapy, before and after treatment. PROs included pain intensity, pain-related symptoms (e.g., pain interference, pain coping, sleep interference) and disability, and general health status. QST included assessments of vibration detection theshold (VDT), pressure pain threshold (PPT) and tolerance (PPToL), temporal summation (TS), and conditioned pain modulation (CPM), at the most painful site. Following treatment, all participants reported significant improvements in PROs (e.g., reduced pain intensity [p < 0.001], pain-related functional impairment [or pain interference] and disability [p=0.001 for both]; better pain coping [p=0.03], sleep [p=0.002]), and overall health [p=0.005]). QST showed a significant treatment-induced increase in PPT (p=0.002) and PPToL (p=0.011), and a significant reduction in TS (p=0.033) at the most painful site, but showed no effects on VDT and CPM. We detected possible associations between a few QST measures and a few PROs. Notably, higher TS was associated with increased pain interference scores at pre-treatment (r=0.772, p=0.009), and a reduction in TS was associated with the reduction in pain interference (r=0.669, p=0.034) and pain disability (r=0.690, p=0.027) scores with treatment. Our preliminary findings suggest significant clinical and therapeutic benefits associated with SCS and DRGS therapies, and the possible ability of these therapies to modulate pain processing within the central nervous system. Replication of our pilot findings in future, larger studies is necessary to characterize the physiological mechanisms of SCS and DRGS therapies.

Individual Participant Symptom Responses to Intra-Articular Lorecivivint in Knee Osteoarthritis: Post Hoc Analysis of a Phase 2B Trial.

IntroductionEstablished thresholds for patient-reported outcomes (PROs) provide clinically relevant responder data from trials. Lorecivivint (LOR) is an intra-articular (IA) therapy in development for knee osteoarthritis (OA). A post hoc analysis from a phase 2b trial (NCT03122860) determined proportions of LOR responders.MethodsA 24-week, randomized trial of 0.07 mg LOR demonstrated PRO improvements compared with PBO in moderate-to-severe knee OA participants. Participants treated with LOR and PBO achieving 30%/50%/70% improvements at weeks 12 and 24 in Pain Numeric Rating Scale (NRS), WOMAC Pain/Function subscales, Patient Global Assessment (PtGA), and OMERACT-OARSI responder criteria were determined. Odds ratios (ORs) and 95% confidence intervals [CIs] were compared with PBO.ResultsThere were 115 and 116 participants in the LOR and PBO groups, respectively. For Pain NRS, LOR increased ORs of achieving 30% [week 12, OR = 2.47 (1.45, 4.19), P < 0.001; week 24, OR = 2.37 (1.40, 4.02), P < 0.01] and 50% [week 24, OR = 1.89 (1.11, 3.23), P < 0.05] improvements over baseline. For WOMAC Pain, LOR increased ORs of achieving 30% [week 24, OR = 1.79 (1.06, 3.01), P < 0.05] and 50% [week 12, OR = 1.79 (1.06, 3.03), P < 0.05; week 24, OR = 1.73 (1.02, 2.93), P < 0.05] improvements. For WOMAC Function, LOR increased ORs of achieving 30% [week 12, OR = 1.85 (1.10, 3.12), P < 0.05; week 24, OR = 1.93 (1.14, 3.26), P < 0.05] improvements. For PtGA, LOR increased ORs of achieving 50% [week 12, OR = 2.28 (1.25, 4.16), P < 0.01] improvements. LOR produced numerical increases at the 70% threshold. LOR increased ORs of achieving OMERACT-OARSI responses [week 12, OR = 2.21 (1.29, 3.78); P < 0.01; week 24, OR = 2.57 (1.49, 4.43), P < 0.001] and strict responses [week 12, OR = 2.13 (1.26, 3.61), P < 0.01; week 24, OR = 2.05 (1.21, 3.47), P < 0.01].ConclusionsLOR (0.07 mg) demonstrated improved PRO threshold responses across single and composite measures of pain, function, and patient global assessment compared with PBO, with benefits sustained to 24 weeks.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40744-021-00316-w.

DOP88 Effect of risankizumab on patient-reported outcomes in patients with Crohn’s Disease who had an inadequate response or intolerance to conventional and/or biologic treatments: Results from phase 3 MOTIVATE and ADVANCE trials

Abstract Background Risankizumab (RZB), an IL-23 inhibitor, is being investigated for Crohn’s disease (CD). MOTIVATE (NCT03105128) and ADVANCE (NCT03104413) were two double-blind, randomised, placebo (PBO)-controlled phase 3 trials evaluating the efficacy and safety of RZB as induction therapy for CD after inadequate response or intolerance to biologic treatment, or conventional or biologic treatment. We investigated the effect of RZB on patient-reported outcomes (PROs) in these two trials. Methods Patients with moderately to severely active CD (CD activity index of 220–450, Simple Endoscopic Score for CD excluding the narrowing component ≥ 6 or ≥ 4 for isolated ileal disease, and average daily stool frequency ≥ 4 and/or average daily abdominal pain score ≥ 2) were randomised 1:1:1 (MOTIVATE; N=569) or 2:2:1 (ADVANCE; N=850) to intravenous RZB 600 mg, RZB 1200 mg or PBO at Weeks 0, 4 and 8. PROs assessed were Inflammatory Bowel Disease Questionnaire (IBDQ), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), and 36-Item Short Form Health Survey (SF 36). Least-squares mean change from baseline to Week 4 and 12 were assessed for each PRO. Additionally, the proportion of patients achieving IBDQ remission (IBDQ total score ≥ 170 points) was evaluated. Comparisons between RZB dosage groups and PBO were based on Cochran-Mantel-Haenszel or chi-square/Fisher’s exact tests; missing data were reported using non-responder imputation. Results In the MOTIVATE (100% biologic inadequate responder [bio-IR]) and ADVANCE (57% bio-IR) trials, significant (P&amp;lt;0.05) improvements in nearly all PROs evaluated were seen as early as Week 4 (Figures 1–4). At Week 12, significant improvements in all PROs were reported for both doses of RZB compared with PBO in both trials, except for the SF-36 Mental Component Summary score (RZB 1200 mg in MOTIVATE), which improved, but not significantly. In MOTIVATE, at Week 4 and 12, IBDQ remission was achieved by significantly (P&amp;lt;0.05) more patients taking RZB (600 mg, 25.1%/38.3%; RZB 1200 mg, 31.9%/44.6%) than PBO (15.5%/26.8%). Results were similar in ADVANCE: RZB 600 mg 30.9%/44.5%, RZB 1200 mg 33.7%/51.5%, and PBO 18.4%/29.8% at weeks 4 and 12, respectively (P&amp;lt;0.001 for both RZB groups compared to PBO). Conclusion Patients with active CD and an inadequate response to conventional or biologic treatment who received RZB (600 mg or 1200 mg) induction therapy reported significant improvements in disease-specific (IBDQ) and general health-related PROs (FACIT and SF-36) compared with PBO, with improvements seen as early as Week 4. Funding statement: Study was funded by AbbVie. Acknowledgement: Medical writing support was provided by Joann Hettasch of Fishawack Facilitate Ltd, and was funded by AbbVie.

Do Outcomes of Meniscal Allograft Transplantation Differ Based on Age and Sex? A Comparative Group Analysis

To analyze the effect of patient age, sex, and associated preoperative factors on patient-reported outcome (PRO) measures and graft survival following primary meniscal allograft transplantation (MAT). A prospectively collected database was retrospectively reviewed to identify patients who underwent primary MAT with a minimum of 2 years of follow up between 1999 and 2017. Demographic, intraoperative, and postoperative outcome data were collected for each patient. Postoperative outcomes were stratified based on age and sex, and comparative statistical analysis was performed between sexes, both >40 and <40. A total of 238 patients underwent primary MAT during the study period, of which 212 patients (mean age, 28.5 ± 9.0 years; range, 15.01-53.67 years) met the inclusion criteria with a mean follow-up of 5.1 ± 3.4 years (range 2.0-15.9 years). At final follow-up, patients ≥40 and <40 years of age demonstrated statistically significant improvements in nearly all PRO scores (P < .05 for both groups). There were no significant differences between either group for achievement of minimal clinically important difference for International Knee Documentation Committee (P= .48) or Knee Injury and Osteoarthritis Outcome Score symptoms (P= .76). Because of insufficient numbers, a statistically significant difference could not be demonstrated in reoperation rate (≥40: 1.49 ± 1.77 years, <40: 1.87 ± 1.98 years, P= .591), failure rate (≥40: 7/32 [21.9%], <40: 19/180 [10.6%], P= .072), or complication rate (≥40: 2/32 [6.3%], <40: 12/180 [6.7%], P= .930) based on age. Both sexes showed a significant improvement in PROs, whereas female patients were more likely to undergo revision surgery (P= .033), with no significant differences based on time to reoperation, failure, or complication rates. PROs similarly improved following MAT in both patients aged ≥40 and those <40 at final follow-up with no significant differences in minimal clinically important difference achievement rate, complication rate, reoperation rate, time to reoperation, or failure rate between groups. Female patients may be more likely to undergo revision surgery after MAT. III; therapeutic retrospective comparison study.

Do Adult Spinal Deformity Patients Undergoing Surgery Continue to Improve From 1-Year to 2-Years Postoperative?

Evaluate clinical improvement as measured by patient-reported outcomes (PROs) during the 1 to 2-year interval. Retrospective Cohort. A single-institution registry of ASD patients undergoing surgery was queried for patients with ≥6 level fusions. Demographics and radiographic variables were collected. PROs collected were the ODI and SRS-22r scores at: preoperative, 1-year and 2-years. Outcome measures of clinical improvement during the 1-2 year time interval were: 1) group medians, 2) percent minimum clinically important difference (MCID), and 3) percent minimal symptom scale (MSS)(ODI < 20 or SRS-pain + function >8). Wilcoxon rank-sum tests, chi-squared tests, Kruskal-Wallis tests, and logistic regression were performed. 157 patients undergoing ASD surgery with minimum of 1-year follow-up were included. Mean age was 53.2 and mean instrumented levels was 13.1. Preoperative alignment was: Neutral Alignment (NA) 49%, Coronal Malalignment (CM) 17%, Sagittal Malalignment (SM 17%), and Combined Coronal/Sagittal Malalignment (CCSM) 18%. Preoperative to 1-year, and preoperative to 2-years, all ODI/SRS-22r significantly improved (P < .001). In all patients, the only significant improvement in PROs between 1-and 2-year postoperative were those reaching ODI MCID (69% 1-year vs. 84% 2-years; P < .001). Subgroup analysis: ≥55 years had an improved median ODI (18 vs. 8; P = .047) and an improved percent achieving ODI MCID (73% vs. 84%, P = .048). CCSM patients experienced significant improvement in SRS-appearance score (75% vs. 100%; P = .050), along with those with severe preoperative SM >7.5 cm (73% vs. 100%; P = .032). Most ASD patients experience the majority of PRO improvement by 1-year postoperative. However, subsets of patients that may continue to improve up to 2-years postoperative include patients ≥55 years, combined coronal/sagittal malalignment, and those with severe sagittal malalignment ≥7.5 cm.

Effect of golimumab on health-related quality of life, other patient-reported outcomes and healthcare resource utilization in patients with moderate-to-severe ulcerative colitis: a real-world multicenter, noninterventional, observational study in Greece

This real-world study assessed the impact of golimumab on health-related quality of life (HRQoL) and other patient-reported outcomes (PROs) in patients with ulcerative colitis over 12 months in Greece. GO-LIFE was a noninterventional, prospective, multicenter, 12-month study. Patients who had moderately-to-severely active ulcerative colitis were naïve to antitumor necrosis factor (anti-TNFα) therapy and had failed previous conventional therapy. Patients received golimumab as per label. The primary endpoint was patients achieving inflammatory bowel disease questionnaire 32-item (IBDQ-32) remission at 12 months. Secondary endpoints, at 6 and 12 months, included patients achieving IBDQ-32 response; the mean change in the treatment satisfaction questionnaire for medication (TSQM) and the work productivity and activity impairment in ulcerative colitis (WPAI:UC) questionnaires; changes in healthcare utilization; patients achieving clinical response and remission; adherence rates and the percentage of patients who discontinued golimumab. IBDQ-32 remission was achieved by 76.9% of patients at 12 months. Mean changes in all TSQM and WPAI:UC domain scores at 12 months were statistically significant. Clinical remission was achieved by 49.4 and 50.6% of patients at 6 and 12 months, and clinical response by 59.3 and 56.8%, respectively. All patients but one (80/81) had high adherence (≥80%) to golimumab treatment over 12 months. Ulcerative colitis-related health care resource utilization was reduced during the follow-up period. In real-world settings, treatment with golimumab resulted in meaningful improvements in HRQoL and other PROs, and in disease activity at 6 and 12 months in patients with moderately-to-severely active ulcerative colitis who were naïve to anti-TNFa therapy.

POS0661 RAPID RESPONSE TO BARICITINIB IN PATIENTS WITH RHEUMATOID ARTHRITIS AND AN INADEQUATE RESPONSE TO METHOTREXATE AND AT LEAST ONE BIOLOGIC DMARD: A CLINICAL AND POWER DOPPLER ULTRASOUND STUDY

Background:Baricitinib, an oral Janus kinase (JAK) 1-2 inhibitor, is currently used among biologic DMARDs (bDMARDs) after the failure of methotrexate (MTX) in rheumatoid arthritis (RA). Power Doppler ultrasound (PDUS) is a promising, non-invasive imaging method to assess synovitis in RA: results from numerous studies suggest that it provides additional information to clinical and conventional radiographic examinations.Objectives:The main objective of our study was to evaluate short-term efficacy of Baricitinib in reducing synovitis, using the composite semi quantitative scale (0–3 grades) PDUS synovitis score, developed by the Outcome Measures in Rheumatology– European League Against Rheumatism (OMERACT– EULAR)-Ultrasound Task Force. Moreover both synovial hyperplasia and intrasynovial power Doppler (PD) signal were also scored as single components/parameters on 0-3 scales. Secondary objective was to assess the concordance between patient reported outcomes (PROs), markers of inflammation, physical examination and US.Methods:We enrolled 30 patients fulfilling 2010 ACR and EULAR criteria for RA. All patients had failed at least one anti-TNF. Each patient was prescribed Baricitinib 4 mg/daily at T0, in addition to MTX and/or oral steroids at a dosage ≤ 7, 5 mg/day of Prednisone or equivalent, at T’. All patients were evaluated at baseline (T0) and then after one month (T1), 3 months (T2) and 6 months (T3) of treatment. Swollen and tender joints (out of 28)were evaluated and recorded, as well as patient (PGA) and physician global assessment (PhGA) and pain, expressed in a visual analog scale (VAS). Disease activity was evaluated at each visit using DAS28 (Disease activity score 28), CDAI (Clinical disease activity index)and SDAI(Simplified disease activity index), accompanied by a complete blood count, Erythrocyte sedimentation rate (ESR) and C- reactive protein (CRP) collection. Statistical analysis was performed using GraphPad version 9.0.0. PDUS examination, was carried out by two rheumatologists (PF and CB) blinded to clinical conditions of the patients, using an Esaote Mylab Twice (Genoa, Italy), equipped with a high-frequency (6-18 MHz) linear probe. With standardised Doppler parameters (pulse repetition frequency between 500-750 Hz; Doppler frequency between 7–11.1 MHz). PDUS was performed at each visit bilaterally for 22 joint sites [MCPs 1–5, proximal interphalangeal joints (PIPs) 1–5, wrist, elbow, glenohumeral, knee, tibiotalar, talonavicular and calcaneocuboidal and metatarsophalangeal joints (MTPs) 1–5] for a total of 44 joints for each patient.Results:we observed a reduction of VAS pain (T0 vs, T6&lt;0,0001) PDUS composite score (T0 vs. T6 p&lt;0,0001), Power Doppler (T0 vs. T6 p&lt;0,0001) synovial hyperplasia (T0 vs. T6 p=0,0002), CRP (T0 vs. T6 p&lt;0,0001) and ESR (T0 vs. T6 p &lt;0,0001) was observed in our patients. Accordingly, DAS-28, CDAI and SDAI displayed a significant reduction too (DAS-28: T0 vs. T6 p&lt; 0, 0001; CDAI: T0 vs. T6 p&lt; &lt;0, 0001; SDAI: T0 vs. T6 p= 0, 0003).Conclusion:We investigated the efficacy of Baricitinib in real life, evaluating both from a clinimetric and ultrasound point of view. Baricitinib, demonstrated a significant parallel and fast improvement in VAS, PDUS and CRP was found at follow up assessment as early as one month of therapy. In conclusion, these results demonstrated the short term efficacy of Baricitinib 4mg for up to 6 months and providing a prompt improvement of PROs within the first weeks of treatment.Figure 1.The difference between the means of PD and of the VAS pain over time (T0, T1, T3 and T6). Power Doppler (T0 vs. T6** p&lt;0,0001), VAS: (T0 vs. T1 *p&lt;0,0098;T0 vs. T3 **** p&lt;0,0001; T0 vs. T6 ****p&lt;0,0001)Disclosure of Interests:None declared

Characterisation of depressive symptoms in rheumatoid arthritis patients treated with tocilizumab during routine daily care.

To assess whether tocilizumab treatment is associated with changes in depression symptoms in patients with rheumatoid arthritis (RA) during routine daily care. We retrospectively analysed data from a German non-interventional study (ARATA) of adult, tocilizumab-naïve RA patients who initiated subcutaneous tocilizumab and were followed for 52 weeks. The Beck Depression Inventory II (BDI-II) was used to assess symptoms of depression and create baseline subgroups of no (BDI-II<14), mild (14-19), moderate (20-28), and severe (≥29) depression. Other key outcomes included Disease Activity Score-28 joints (DAS28), patient-reported outcomes (PROs), and adverse events. Mixed model repeated measures (MMRM) assessed the impact of DAS28 on BDI-II over time, and Pearson correlation analyses evaluated associations between changes from baseline. Of 474/1155 ARATA patients who completed the BDI-II at baseline, 47.7% had evidence of depression: 18.4% mild, 17.7% moderate, and 11.6% severe. 229 patients (48.3%) completed the BDI-II at both baseline and week 52. Two-thirds of patients with moderate or severe depression at baseline improved to a milder or no depression subgroup at week 52 (44/65 [67.7%]). Improvements in disease activity and PROs were observed in all subgroups, but patients with depression had lower response and higher adverse events rates. We observed an association between DAS28 and BDI-II over time in MMRM analyses, but the Pearson correlation for change from baseline was weak (r=0.10). Depression is common in patients receiving routine care for RA. Improvements in depressive symptoms in RA during tocilizumab therapy appear to be distinct from changes in disease activity.

TLANDO, a Novel Oral TRT, Improves Sexual and Mental Domain Outcomes in Hypogonadal Men

Male hypogonadism is characterized by symptoms and deficiency (<300 ng/dL) in levels of in total testosterone (TT), a critical hormone for sexual, cognitive, and body function and development. TLANDO, a testosterone undecanoate (TU) comprising lymphatically delivered oral testosterone replacement therapy (TRT) option not requiring dose titration, treatment has demonstrated effective restoration in hypogonadal men of TT levels to the eugonadal range in multiple clinical studies. TLANDO therapy resulted in decreased sex hormone binding globulin with increased free testosterone (FT). TLANDO’s unique delivery system enables consistent restoration of TT regardless of meal fat content. Moreover, TLANDO has shown potential to improve liver health through resolution of fatty liver disease in hypogonadal men and is not known to have any adverse liver effects. However, it is unclear if fixed dose TLANDO therapy without dose adjustment improves symptoms of psychosexual functions. The objective is to assess key sexual and mental domain Patient Reported Outcomes (PRO) post 52 weeks of treatment using TLANDO on the to-be-marketed dosing regimen in comparison with a widely used topical TRT, Androgel 1.62%. Data analysis was performed in in hypogonadal males post TLANDO treatment without dose adjustment, and in patients on the active control from a randomized, multi-center, open label, active controlled 52-week trial (SOAR, NCT02081300). Sexual and mental domain function PROs were measured at baseline (BL) and end of study (EOS) using Psychosexual Daily Questionnaire (PDQ) and Short Form (SF)-36 surveys and compared between TLANDO and active control. Post treatment with TLANDO dosing regimen not requiring dose titration, key sexual domain function PROs at week 52 were significantly (p<0.05) improved from BL: positive mood (BL:4.5 vs EOS:5.1, p<0.001), negative mood (1.8 vs 1.4, p<0.01), overall sexual desire (2.5 vs 3.7, p<0.001), sexual activity (2.5 vs 4.0, p<0.001), highest pleasure with partner (2.0 vs 2.8, p=0.06), highest pleasure without partner (1.8 vs 2.4, p<0.05), weekly maintained erection (3.3 vs 4.5, p<0.001), and weekly full erection % (50.5% vs 68.9%, p<0.001). Most sexual and mental function PROs were comparable to Androgel 1.62. TLANDO therapy was well tolerated through 52 weeks of treatment exposure. In conclusion, TLANDO, a novel easy to use and prescribe TRT not requiring dose titration, demonstrated improvement in sexual and mental PROs, a significant unmet need in hypogonadal males. Further placebo-controlled studies are warranted to better elucidate these improvements.

Treatment of COPD with Long-Acting Bronchodilators: Association Between Early and Longer-Term Clinically Important Improvement.

IntroductionThis post hoc analysis of the “Early MAXimization of bronchodilation for improving COPD stability” (EMAX) trial investigated whether patients achieving early clinically important improvement (CII) sustained longer-term improvements and lower risk of clinically important deterioration (CID).MethodsPatients were randomized to umeclidinium/vilanterol, umeclidinium, or salmeterol for 24 weeks. The patient-reported outcomes (PROs) Transition Dyspnea Index (TDI), Evaluating Respiratory Symptoms, St George’s Respiratory Questionnaire (SGRQ) and COPD Assessment Test (CAT) were assessed. CII, defined as attaining minimum clinically important differences (MCID) in ≥2 PROs, was assessed at Weeks 4, 12 and 24. CID was defined as a deterioration in CAT, SGRQ, TDI by the MCID and/or a moderate/severe exacerbation from Day 30.ResultsOf 2425 patients, 50%, 53% and 51% achieved a CII at Weeks 4, 12 and 24, respectively. Patients with a CII at Week 4 versus those without had significantly greater odds of achieving a CII at Weeks 12 and 24 (odds ratio: 5.57 [95% CI: 4.66, 6.66]; 4.09 [95% CI: 3.44, 4.86]). The risk of a CID was higher in patients who did not achieve a CII at Week 4 compared with patients who did (hazard ratio [95% CI]: 2.09 [1.86, 2.34]). Patients treated with umeclidinium/vilanterol versus either monotherapy had significantly greater odds of achieving CII at Weeks 4, 12 and 24.ConclusionAchieving a CII at Week 4 was associated with longer-term improvement in PROs and a reduced risk of deterioration. Further research is required to investigate the importance of an early response to treatment on the long-term disease course.

Improvement in Quality of Life Following Surgical Resection of Benign Intradural Extramedullary Tumors: A Prospective Evaluation of Patient-Reported Outcomes.

Historically, symptomatic, benign intradural extramedullary (IDEM) spine tumors have been managed with surgical resection. However, minimal robust data regarding patient-reported outcomes (PROs) following treatment of symptomatic lesions exists. Moreover, there are increasing reports of radiosurgical management of these lesions without robust health-related quality of life data. To prospectively analyze PROs among patients with benign IDEM spine tumors undergoing surgical resection to define the symptomatic efficacy of surgery. Prospective, single-center observational cohort study of patients with benign IDEM spine tumors undergoing open surgical resection. Pre- and postoperative Brief Pain Index (BPI) and MD Anderson Symptom Inventory (MDASI) questionnaires were used to quantitatively assess their symptom control after surgical intervention. Matched pairs were analyzed with the Wilcoxon signed-rank test. A total of 57 patients met inclusion criteria with both pre- and postoperative PROs. There were 35 schwannomas, 18 meningiomas, 2 neurofibromas, 1 paraganglioma, and 1 mixed schwannoma/neurofibroma. Most patients were American Spinal Injury Association Impairment (ASIA) E (93%) with high-grade spinal cord compression (77%), and underwent either a 2 or 3 level laminectomy (84%). Surgical resection resulted in statistically significant improvement in all 3 composite BPI constructs of pain-severity, pain-interference, and overall patient pain experience (P<.0001). Surgical resection resulted in statistically significant improvements in all composite scores for the MDASI core symptom severity, spine tumor, and disease interference constructs (P<.01). Three patients (5%) had postoperative complications requiring surgical interventions (2 wound revisions and 1 ventriculo-peritoneal shunt). Surgical resection of IDEM spine tumors provides rapid, significant, and durable improvement in PROs.

An evaluation of clinical and quality of life outcomes after ventral hernia repair with poly-4-hydroxybutyrate mesh.

Despite continued efforts, recurrence after ventral hernia repair (VHR) remains a common problem. Biosynthetic Phasix™ (Poly-4-Hydroxybutyrate, P4HB) mesh combines the durability of synthetic mesh with the bio-resistance of biologics. P4HB has shown promising early outcomes, but long-term data are lacking. We examine patients following VHR with P4HB with at least 3years of follow-up to assess clinical and patient reported outcomes (PROs). Adult patients (≥ 18years old) undergoing VHR with P4HB mesh between 10/2015 and 01/2018 by a single surgeon were retrospectively identified. Patients with < 36months of follow-up were excluded unless they had a documented recurrence. Clinical outcomes and quality of life using the Hernia-Related Quality of Life Survey (HerQLes) were assessed. Seventy-one patients were included with a median age and body mass index of 61.2 and 31kg/m2, respectively. Mesh was placed in the retromuscular (79%) and onlay (21%) planes with 1/3 of patients having hernias repaired in contaminated fields. There were no mesh infections, enterocutaneous fistulas, or mesh explantations. Nine patients (12.7%) developed recurrence at a median follow-up of 43.1months [38.2-49.1]. Mesh plane, fixation technique, and Ventral Hernia Working Group were not associated with recurrence. Significant improvement in disease-specific PROs was observed and maintained at 3-year follow-up. Longitudinal clinical and quality of life outcomes after clean and contaminated VHR with P4HB are limited. Here, we conclude that P4HB is an effective and versatile mesh option for use in abdominal wall reinforcement.

Perioperative Modifications to the Open TLIF Provide Comparable Short-term Outcomes to the MIS-TLIF.

This study is a retrospective review of patients' charts and data from longitudinally collected clinical outcomes and opioid use. In the current study, we aim to compare short-term outcomes data for 139 Open transforaminal interbody fusion (TLIF) patients to recently published data for tubular and endoscopic MIS-TLIF. In response to the downsides associated with Open TLIF, such as large incision, blood loss, delayed ambulation, prolonged hospitalization, and opioid-reliance, spine surgeons developed tubular retractor based "minimally-invasive" TLIF. However, the traditional Open TLIF retains its significance in terms of providing successful fusion and improved patient-reported outcomes (PROs). We adapted several techniques with an aim to improve short-term outcomes for our Open TLIF patients that combined extensive perioperative counselling, an emphasis on early mobilization, avoidance of overuse of opioid analgesics, early discharge with home care arrangements, use of a posthospitalization drainage tube with intraoperative surgical modifications using small incisions (4-5 cm), a narrow 20 mm retractor, minimal muscle injury, and use of a cell saver to minimize net blood loss. The demographics and perioperative results were compared with data from recent MIS-TLIF studies using Student t test for continuous and χ2/exact test for categorical variables. Among the total 139 patients, 115 underwent a single-level procedure, 90% of whom were discharged on the first postoperative day (length of stay=1.13±0.47 d) with an average net estimated blood loss of 176.17±87.88 mL. There were 24 two-level procedures with an average length of stay of 1.57±0.84 days, average net estimated blood loss was 216.96±85.70 mL. The patients had statistically significant improvements in PROs at 3 and 12 months. The results of this study identify that patients who underwent modified Open TLIF demonstrated favorable short-term outcomes, as compared with the tubular MIS-TLIF, by virtue of avoidance of blood transfusions, shorter hospital stays, and significantly less opioid usage while experiencing satisfactory PROs.

Onlay Poly-4-Hydroxybutyrate (P4HB) Mesh for Complex Hernia: Early Clinical and Patient Reported Outcomes

While mesh re-enforcement and advanced surgical techniques are cornerstones of complex ventral hernia repair (CVHR), the risk of complications and recurrence is common. We aim to evaluate the efficacy, safety, and patient reported outcomes (PROs) of patients undergoing CVHR with onlay Poly-4-hydroxybutyrate (P4HB). Adult (>18 y old) patients undergoing VHR with P4HB (Phasix) in the onlay plane by a single surgeon from 01/2015 to 05/2020 were reviewed. VHR was considered complex if patients had significant co-morbidities, large abdominal wall defects, a history of extensive abdominal surgery, and/or concurrent intra-abdominal pathology. A composite of postoperative outcomes including surgical site occurrences (SSO), surgical site infection (SSI), and surgical site occurrences requiring procedural intervention (SSOpi), as well as PROs as defined by the Abdominal Hernia-Q (AHQ), were analyzed. A total of 51 patients were included with average age and body mass index of 56.4 and 29.9 kg/m2. Median follow up was 20 mo with a hernia recurrence rate of 5.9% (n = 3). 21 patients had an SSO (41.2%), 8 had an SSI (15.7%), and 6 had an SSOpi (11.8%). There was an association with Ventral Hernia Working Group ≥ 2 and development of SSO. There was a significant improvement in overall PROs (P < 0.0001) with no difference in those patients with and without complications (P > 0.05). For hernia patients with large defects and complex intra-abdominal pathology, a safe and effective repair is difficult. The use of onlay P4HB was associated with acceptable postoperative outcomes and recurrence rate.

Early responders within seven days of dupilumab treatment for severe asthma evaluated by patient-reported outcome: a pilot study.

BackgroundThe management of severe asthma-associated symptoms is essential since they are distressing to the affected patients, and also greatly impair their quality of life. Dupilumab, a monoclonal antibody, blocks interleukin (IL)-4 and IL-13 signaling, both of which are crucial in acquired and innate immunity pathways through fast signal transduction, leading to an early response to treatment. Although rapid improvement within 1–3 days after dupilumab treatment was observed in moderate-to-severe atopic dermatitis, an early response within 7 days of dupilumab treatment in severe asthma has not been reported.MethodsTwelve consecutive patients with severe asthma who were newly treated with dupilumab between July 2019 and April 2020 were retrospectively investigated. We evaluated the early response (within 7 days) of patients with severe asthma receiving dupilumab therapy. Asthma control test (ACT) and the daily ACT, which was modified from the ACT to evaluate daily symptoms associated with asthma, were adopted as patient-reported outcomes (PROs) at week 8 and within 7 days, respectively. Patients were stratified into early responders (7 days), late responders (week 8), and non-responders without significant improvement in PROs. Descriptive statistics were adopted due to the limited number of patients.ResultsFour of these 12 patients were early responders, with the following baseline characteristics: body mass index, <25 kg/m2; without depression; baseline forced expiratory volume in 1 second, <1.50 L; and more than one exacerbation in 1 year. On the other hand, five were late responders, and 44.4% of the nine responders were early responders. The higher the eosinophilic count and/or FeNO did not show any relationship between the early responder and nonresponder.ConclusionsThe effect of dupilumab on severe asthma in patients with atopic features could be started earlier than 2 weeks, similar to atopic dermatitis. Daily ACT may be useful in monitoring the early efficacy of dupilumab in treating severe asthma.

The Effectiveness of Closed-Incision Negative-Pressure Therapy Versus Silver-Impregnated Dressings in Mitigating Surgical Site Complications in High-Risk Patients After Revision Knee Arthroplasty: The PROMISES Randomized Controlled Trial

BackgroundRevision total knee arthroplasty (rTKA) is associated with significant risk of wound-related morbidity. The present study aimed to evaluate the 1) efficacy of closed-incision negative-pressure therapy (ciNPT) vs silver-impregnated antimicrobial dressing (AMD) in mitigating postoperative surgical site complications (SSCs), 2) the effect of ciNPT vs AMD on certain postoperative health utilization parameters, and on 3) patient-reported outcomes (PROs) improvement at 90-day postoperative follow-up. MethodsThis multicenter randomized controlled trial was conducted between December 2017 and August 2019. Patients ≥22 years, at high risk for SSC, and receiving rTKA with full exchange and reimplantation of new prosthetic components or open reduction and internal fixation of periprosthetic fractures were screened for inclusion. Eligible patients were randomized to receive a commercially available ciNPT system or a silver-impregnated AMD (n = 147, each) for minimum of 5-day duration. Primary outcome was the 90-day incidence of SSCs with stratification in accordance with revision type (aseptic/septic). Secondary outcomes were the 90-day health care utilization parameters (readmission, reoperation, dressing changes, and visits) and PROs. ResultsOf 294 patients randomized (age: 64.9 ± 9.0 years, female: 59.6%), 242 (82.0%) patients completed the study (ciNPT: n = 124; AMD: n = 118). The incidence of 90-day SSCs was lower for the ciNPT cohort (ciNPT: 3.4% vs AMD: 14.3%; odds ratio (OR): 0.22, 95% confidence interval (0.08, 0.59); P = .0013). Readmission rates (3.4% vs 10.2%, OR: 0.30(0.11, 0.86); P = .0208) and mean dressing changes (1.1 ± 0.3 vs 1.3 ± 1.0; P = .0003) were lower with ciNPT. The differences in reoperation rates, number of visits, and PRO improvement between both arms were not statistically significant (P > .05). ConclusionciNPT is effective in reducing the 90-day postoperative SSCs, readmission, and number of dressing changes after rTKA. Recommending routine implementation would require true-cost analyses.

Does exercise therapy improve patient-reported outcomes in rheumatoid arthritis? A systematic review and meta-analysis for the update of the 2020 JCR guidelines for the management of rheumatoid arthritis.

This study aimed to evaluate the impact of exercise therapy on patient-reported outcomes (PROs) in rheumatoid arthritis (RA) as part of the process of updating the 2020 Japanese guidelines for the management of RA according to the Grading of Recommendations, Assessment, Development, and Evaluation system. We searched PubMed, Japana Centra Revuo Medicina Web, and the Cochrane Library (from 2009 to 2018) to identify articles that evaluated PROs of exercise therapy and RA disease activity. A total of 662 articles were identified, including nine RCTs, and meta-analyses were performed on six RCTs on systemic exercise therapy and three RCTs on upper extremity exercise therapy. Analyzed exercise therapies were diverse, differing in target population, intervention method, and duration. Significant improvements were observed in the Health Assessment Questionnaire Disability Index (mean difference -0.35, 95% confidence interval (CI): -0.60 to -0.10), pain (standardized mean difference -2.04, 95% CI: -3.77 to -0.32), and SF-36. For upper extremity exercise therapy, significant improvements in PROs (Disabilities of the Arm, Shoulder, and Hand Questionnaire, Michigan Hand Outcome Questionnaire) were observed. Exercise therapy in RA treatment improves patient subjective assessment of pain, physical function, and quality of life.

Abstract PS9-21: Patient reported outcomes of newly diagnosed women with breast cancer enrolled in a digital health coaching program

Abstract Background: With breast cancer (BC) treatment primarily managed in the ambulatory setting opportunities exist to provide support services to individuals that enhance their ability to manage side effects of treatment and their overall health outcomes. The aim of this randomized control trial was to evaluate the effect of a digital health coaching program on patient reported outcomes (PROs) and experience of women with BC undergoing chemotherapy treatment in the ambulatory setting. Methods: Women newly diagnosed with BC were randomized to receive either a 3-month digital health coaching intervention or standard of care support services provided by the treating hospital. PROs were captured at baseline and monthly for 3 months. The primary objective compared Patient Reported Outcomes Measurement Information System (PROMIS) Global Health Scale scores between arms at the 3-month assessment. Secondary objectives evaluated the PROMIS physical and mental subscales, and the MD Anderson Symptom Inventory (MDASI) for differences between arms and over time. Summary statistics were used to describe demographic and baseline clinical characteristics by arm. Linear mixed effects models were used to assess PROMIS and MDASI over time. Statistical analysis were performed using Stata/MP v15.0. Results: A total of 210 subjects were randomized to the control arm and 208 to the digital health coaching intervention. A significant time (Table 1) but not intervention effect was observed. The mean (SD) of the PROMIS physical health t-score for the control arm was 43.9 (4.7) and 44.1 (4.3) for the intervention arm (p = 0.699). The mean (SD) of the PROMIS mental health t-score for the control arm was 45.3 (5.4) and 45.1 (5.0) for the intervention arm (p = 0.742). The mean (SD) of MDASI severity score for the control arm was 2.4 (1.6) and was 2.5 (1.8) for the intervention arm (p = 0.715). The mean (SD) of the MDASI interference for the control arm was 2.6 (2.3) and was 2.8 (2.3) for the intervention arm (p = 0.633). Of 208 individuals randomized to the intervention, the majority (n=195; 94%) preferred engagement by phone, email and text. Participants averaged 9.17 calls with a mean duration of 13.04 minutes per call, 12.3 outbound texts to and 3.8 inbound texts from the participant, and a 47% email open rate over the course of 12-weeks. Conclusions: Study results suggest that while digital health coaching did not produce a statistically significant improvement in PRO over the course of the first 3 months of treatment, it did result in engagement that could be leveraged during and potentially beyond primary treatment for women with BC. PRO scores reflected minimal symptom burden and high global health at baseline before the start of treatment, which predictably declined over the course of treatment. Results informed the development of a currently enrolling expansion trial for individuals with diverse treatment types to evaluate which population receives the greatest benefit. Given the need for increasing engagement using telehealth solutions, the optimization of digital health coaching for individuals with BC is timely and increasingly relevant to promote optimal health self-efficacy and PROs. Table 1. Linear Mixed Models of PROMIS and MDASI Scores Over TimePROMISEffectBeta95% LB95% UBp-valuePhysicalTime&amp;lt;0.00130-0.88-1.50-0.260.00660-0.74-1.390.090.02690-1.88-2.53-1.22&amp;lt;0.001Intervention0.12-0.730.980.980MentalTime&amp;lt;0.00130-3.53-4.22-2.85&amp;lt;0.00160-3.94-4.66-3.22&amp;lt;0.00190-4.64-5.36-3.92&amp;lt;0.001Intervention-0.25-1.130.630.583MDASIEffectBeta95% LB95% UBp-valueSeverityTime&amp;lt;0.001301.170.991.35&amp;lt;0.001600.990.811.18&amp;lt;0.001901.261.061.45&amp;lt;0.001Intervention-0.08-0.330.160.499InterferenceTime&amp;lt;0.001301.321.081.58&amp;lt;0.001601.190.921.46&amp;lt;0.001901.561.291.83&amp;lt;0.001Intervention-0.09-0.420.240.587 Citation Format: Kelly J Brassil, Bryan Fellman, Brett Wisse, Diana Urbauer, Valerie Shelton, Carolyn Harty, Mazi Rasulnia. Patient reported outcomes of newly diagnosed women with breast cancer enrolled in a digital health coaching program [abstract]. In: Proceedings of the 2020 San Antonio Breast Cancer Virtual Symposium; 2020 Dec 8-11; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2021;81(4 Suppl):Abstract nr PS9-21.

The Feasibility of Prehabilitation as Part of the Breast Cancer Treatment Pathway.

BackgroundThere is compelling support for implementing prehabilitation to optimize perioperative risk factors and to improve postoperative outcomes. However, there is limited evidence studying the application of multimodal prehabilitation for patients with breast cancer.ObjectiveTo determine the feasibility of multimodal prehabilitation as part of the breast cancer treatment pathway.DesignThis was a prospective, cohort observational study. Breast cancer patients undergoing surgery were recruited. They were assigned to an intervention or control group according to patient preference.SettingUK prehabilitation center.ParticipantsA total of 75 patients were referred during the study period. Forty eight patients (64%) did not participate; 20 of those opted to be in the control group. Twenty four patients engaged with prehabilitation and returned completed questionnaires. In total, 44 patients were included in the analysis.InterventionsThe program consisted of supervised exercise, nutritional advice, smoking cessation, and psychosocial support.Outcome MeasuresFeasibility was determined by the center's ability to deliver the program. This was measured by the number of patients who wanted to access the service, compared with those able to. Service uptake, patient satisfaction, and project costs were recorded. Patient‐reported outcomes (PROs) and the use of healthcare resources were also evaluated.ResultsA total of 61 patients (81%) wanted to participate; 24 (32%) were able to partake and return questionnaires. Reasons for nonparticipation included surgery within weeks, full‐time commitments, and transportation difficulties. A total of 25 (93%) prehabilitation patients recorded high satisfaction with the program. There was a significant reduction in anxiety among prehabilitation patients. There were no significant improvements in the other PROs. There were no changes to hospital length of stay, readmissions, and complications.ConclusionsMultimodal prehabilitation is a feasible intervention. Logistical challenges need to be addressed to improve engagement. These results are limited and would require a larger sample to confirm the findings. Work on a thorough cost‐benefit analysis is also required.