Component Summary Scores Research Articles

Efficacy of a cognitive-behavioral digital therapeutic on psychosocial outcomes in rheumatoid arthritis: randomized controlled trial

Cognitive behavioral therapy improves psychosocial outcomes in rheumatoid arthritis (RA), but access is limited. We conducted a randomized controlled trial to evaluate the efficacy of a cognitive-behavioral digital therapeutic, reclarit, on psychosocial outcomes in adult RA patients with impaired health-related quality of life. Participants were randomized to reclarit plus treatment as usual (TAU) or TAU plus educational and informational material (active control). The primary outcome was SF-36 mental (MCS) and physical (PCS) component summary scores at 3 months, with additional assessments at 6 months. reclarit significantly improved SF-36 MCS scores compared to control (mean difference 3.3 [95% CI 0.7, 5.9]; p = 0.014), with high user satisfaction and sustained improvements at 6 months. Depression, anxiety, fatigue, and social/work functioning also improved significantly, while SF-36 PCS, pain, and disability scores did not differ. In conclusion, reclarit offers immediate, effective, evidence-based and personalized psychological support for RA patients.

Effect of nano-curcumin supplementation on cardiometabolic risk factors, physical and psychological quality of life, and depression in patients with coronary slow flow phenomenon: a randomized double-blind clinical trial

BackgroundExtensive evidence has suggested the cardio-protective properties of the polyphenol curcumin. The aim of this study was to investigate the effects of a highly bioavailable curcumin supplement on cardiometabolic risk factors, health-related quality of life, and depression in patients with coronary slow flow phenomenon (CSFP).MethodsThis randomized double-blind placebo-controlled clinical trial was conducted in 42 patients with CSFP (age 35–70 years, 25 ≤ body mass index < 40 kg/m2). Patients received either 80 mg/day nano-curcumin or placebo for 12 weeks. Serum levels of visfatin, high-sensitivity C-reactive protein (hs-CRP), and glycemic indices were measured before and after the intervention. The short form 36-item quality of life (SF-36) and Beck’s Depression Inventory-II (BDI-II) questionnaires were assessed, as well.ResultsNo significant improvements were observed in circulating hs-CRP and visfatin following the intervention. A significant increase was observed in pre- to post-fasting blood glucose (− 0.9 ± 12.2 vs. 7.7 ± 12.4 mg/dl, p = 0.02) and hemoglobin A1C (− 0.1 ± 0.8 vs. 0.5 ± 0.8%, p = 0.04) levels, in the placebo compared with the intervention group. Physical (8.2 ± 8.1 vs. − 1.2 ± 6.5, p < 0.001) and mental (6.8 ± 11.8 vs. − 1.1 ± 10.4, p = 0.02) component summary scores were significantly improved in the nano-curcumin than the placebo group. Additionally, the number of patients with lower degrees of depression was significantly better in the intervention than the placebo group following the supplementation (p = 0.046).ConclusionCurcumin supplementation prevented deterioration of glycemic control and improved physical and psychological quality of life and depression in patients with CSFP.Trial registrationIranian Registry of Clinical Trials (IRCT20131125015536N8), June 19, 2019.

Health-related quality of life (HRQoL) results for adjuvant alectinib vs chemotherapy in patients with resected ALK+ non-small cell lung cancer (NSCLC): Data from ALINA.

8006 Background: ALINA (NCT03456076) is a global, open-label, phase 3, randomized trial evaluating the efficacy and safety of adjuvant alectinib versus chemotherapy in patients with resected stage IB (≥4 cm)–IIIA, ALK+ NSCLC. A significant disease-free survival benefit was observed with alectinib vs chemotherapy (HR 0.24, 95% CI: 0.13–0.43, p&lt;0.0001; N=257 patients in the ITT). In the adjuvant setting, where patients have received surgery with curative intent, the impact of treatment on HRQoL is an important clinical consideration. We report HRQoL results from ALINA. Methods: Patients ≥18 years old with ECOG PS 0/1 and completely resected, stage IB–IIIA, ALK+ NSCLC (UICC/AJCC 7th edition) were randomized 1:1 to receive oral 600 mg alectinib twice-daily for 2 years, or four 21-day cycles of platinum-based chemotherapy. HRQoL was an exploratory endpoint measured using the Short Form-36 version 2 (SF-36v2) health survey, which assesses functional health and well-being across 8 domains and 2 aggregated summary scores: the physical (PCS) and mental (MCS) component summary scores. Patients completed the SF-36v2 at baseline, every 3 weeks to Week 12, then every 12 weeks until disease recurrence, withdrawal of consent, death, or Week 96 (or equivalent post-chemotherapy follow-up visit). SF-36v2 was scored using norm-based scoring relative to the 2009 US general population (mean ± standard deviation: 50 ± 10), with higher scores indicating better health. Within-group minimal important differences (MIDs) were used as benchmarks for each domain, MCS and PCS. Data cut-off: 26 June 2023. Results: Completion rates were generally high (&gt;90%) throughout the study. At baseline, most mean scores were low (&lt;50) and were similar between study arms. With alectinib, mean scores were ≥50 for the Bodily Pain, Mental Health and Vitality domains by Week 96, suggesting that patients functioned as well as the general population on these aspects. The mean change from baseline met or exceeded MIDs for 5 domains and MCS by Week 12, and exceeded MIDs for 6 domains, MCS and PCS by Week 96. In the chemotherapy arm, mean scores were low during treatment; mean change from baseline for General Health and Vitality exceeded negative MIDs, indicating worsening in these domains. Mean scores improved in the post-chemotherapy period; in the final assessment, mean scores were ≥50 for the Bodily Pain, Mental Health and Vitality domains, and MIDs were met or exceeded for MCS, PCS and 5 domains. Conclusions: Alectinib demonstrated an improvement in most domains of HRQoL by Week 12, followed by maintenance of HRQoL on all 8 domains, MCS and PCS to Week 96. With chemotherapy, HRQoL was low during treatment, but improved in the post-chemotherapy period. Together with the DFS benefit seen in ALINA, these data support alectinib as an important new adjuvant treatment for patients with resected ALK+ NSCLC. Clinical trial information: NCT03456076 .

#1562 Longitudinal changes in quality of life—a comparison between institutional hemodialysis, home-hemodialysis and peritoneal dialysis

Abstract Background and Aims Quality of life is an important factor for patients when planning for future dialysis care and for some individuals, it might be even more important than a prolonged life. Knowledge about differences between dialysis modalities is essential for patients making informed decisions. Most previous research on differences in quality of life between dialysis modalities have been cross-sectional and subjected to selection bias; few large contemporary studies have investigated the changes in quality of life between the three dialysis modalities; institutional hemodialysis (IHD), home hemodialysis (HHD) and peritoneal dialysis. The aim of the present study was to compare longitudinal changes in health-related physical and mental quality of life between patients on IHD, PD, and HHD. Method Patients on dialysis with at least two Research and Development 36 (RAND-36) questionnaires between 2017-2021 in the Swedish Renal Registry (SRR) were eligible for inclusion. Information on dialysis modality, patient characteristics and medication were obtained from the SRR and other national registries. Patients were followed for three years and remained in their designated dialysis modality in the analysis until end of the follow-up (intention-to-treat). Changes in physical (PCS) and mental (MCS) component summary score were analyzed in adjusted linear mixed models and joint models where informative censoring due to mortality and kidney transplantation was considered. Results 930 patients (IHD 714, HHD 88, PD 128), were followed for 1.8 years (IQR 1.0–2.1). Patients on HHD were younger, had lower Charlson comorbidity index (CCI) and higher serum albumin compared with IHD and PD (Table 1). At baseline, patients on IHD had a mean PCS 30.7 (95% CI 29.9–31.4) and MCS 45.6 (95% CI 44.7–46.5). The unadjusted PCS at baseline was higher in HHD patients (4.5 [95% CI 2.2–6.9]) and PD patients (2.5 [95% CI 0.5–4.5]) compared with patients on IHD but showed no difference in the adjusted models. Both unadjusted and adjusted MCS was similar at baseline. The PCS declined over time in all modalities; faster for patients on PD compared with IHD (−1.2 per year [95% CI −2.1 – −0.3]; Fig. 1). MCS decreased similarly for both PD and IHD over time, while patients on HHD had an improving MCS (1.5 per year [95% CI 0.1–2.8]) compared with patients on IHD (Fig. 2). Conclusion There are differences in the development of physical and mental quality of life over time for patients on different dialysis modalities. The dialysis modality with the overall most positive development was HHD. Offering HHD to more patients could potentially improve the quality-of-life for patients on dialysis.

QOL-06. HEALTH RELATED QUALITY OF LIFE INFLUENCES ON OVERALL SURVIVAL IN GLIOBLASTOMAS

Abstract BACKGROUND Currently, in treatment of glioblastomas (GBM), emphasis is placed not only on prolonging prognosis but also on maintaining quality of life (QOL). The purpose of this study was to investigate the relationship between health related QOL and prognosis in GBMs and to examine factors that influence on QOL. Method: Participants were 34 patients with GBMs who underwent surgery (mean age, 56.4 years). All patients assessed Karnofsky performance status (KPS), several kinds of neurological and neuropsychological functions, and SF-36 for QOL. In SF-36, we calculated three summary component scores: physical, mental, and role/social component score (PCS, MCS, RCS, respectively). Cox-regression analysis was used to analyze relationship between QOL and progression-free survival (PFS) and overall survival (OS). To examine clinical and functional factors and brain regions associated with QOL, multiple regression analysis and the Voxel-based lesion symptom mapping (VLSM) were utilized. Result: When association between PFS/OS and QOL (PCS, MCS, and RCS) were analyzed, significant correlation was found only between OS and PCS (p = 0.039). Then, we investigated factors influencing on PCS. Using univariate analysis, the clinical factors associated with PCS were lesion location and KPS at 6 months (p = 0.037, p = 0.030, respectively). As for brain functions, visuospatial cognition and paralysis were significantly associated with PCS (p = 0.012 and p = 0.0056, respectively). Among these significant factors in univariate analyses, only visuospatial cognition and paralysis were associate with PCS via multiple regression analysis. Result of VLSM revealed that patients who resected the right medial superior frontal gyrus and deep part of the middle frontal gyrus showed significantly low PCS. Patients with VLSM positive region resected were more likely to have paresis and/or visuospatial cognitive deficit (p = 0.0013). CONCLUSION Health related QOL was associated with OS in GBMs. Factors influencing on QOL were visuospatial cognition and paresis, that may cause by lesion of the right frontal lobe.

Quality of life of the Canadian population using the VR-12: population norms for health utility values, summary component scores and domain scores

ObjectivesTo estimate Canadian population norms (health utility values, summary component scores and domain scores) for the VR-12.MethodsEnglish and French speaking Canadians aged 18 and older completed an online survey that included sociodemographic questions and standardized health status instruments, including the VR-12. Responses to the VR-12 were summarized as: (i) a health utility value; (ii) mental and physical component summary scores (MCS and PCS, respectively), and (iii) eight domain scores. Norms were calculated for the full sample and by gender, age group, and province/territory (univariate), and for several multivariate stratifications (e.g., age group and gender). Results were summarized using descriptive statistics, including number of respondents, mean and standard deviation (SD), median and percentiles (25th and 75th), and minimum and maximum.ResultsA total of 6761 people who clicked on the survey link completed the survey (83.4% completion rate), of whom 6741 (99.7%) were included in the analysis. The mean health utility score was 0.698 (SD = 0.216). Mean health utility scores tended to be higher in older age groups, ranging from 0.661 (SD = 0.214) in those aged 18–29 to 0.728 (SD = 0.310) in those aged 80+. Average MCS scores were higher in older age groups, while PCS scores were lower. Females consistently reported lower mean health utility values, summary component scores and domain scores compared with males.ConclusionsThis is the first study to present Canadian norms for the VR-12. Health utility norms can serve as a valuable input for Canadian economic models, while summary component and domain norms can help interpret routinely-collected data.

Association between PROMIS10, SF-36 and NeuroQoL in persons with multiple sclerosis

BackgroundPatient reported outcome measures (PROs) are considered promising tools for use in clinical settings to measure the impact of disease on physical, mental and social well-being from the patient's perspective. The Patient Reported Outcome Measurement Information System Scale v1.1-Global Health (PROMIS-10) is a measure that is well-suited to clinical practice, but the relationships between this measure and longer PRO measures used in multiple sclerosis (MS) research are unknown. MethodsSubjects enrolled in SysteMS: A Systems Biology Study of Clinical, Radiological, and Molecular Markers in Subjects with MS at the Brigham and Women's Hospital were eligible to contribute to the study. 349 subjects completed three PRO measures at study entry: PROMIS-10, Medical Outcomes Study Short-Form 36 (SF-36), and Quality of Life in Neurological Disorders (Neuro-QoL™). All questions and global scores from PROMIS-10 were correlated with all domain and summary component scores for SF-36 and all domain scores for Neuro-QoL using Pearson's correlation coefficient. Further, the global scores from PROMIS-10 were correlated with the expanded disability status scale (EDSS) and compared between disease categories (relapsing vs progressive MS). ResultsStrong correlations were observed between PROMIS-10 questions and SF-36 domains aimed at measuring the same construct. Further, the PROMIS-10 Global Physical Health score was correlated with the Physical Component Score from the SF-36 (r = 0.798), and the PROMIS Global Mental Health score was correlated with the Mental Component Score from the SF-36 (r = 0.726). Strong correlations between PROMIS-10 questions and two Neuro-QoL domains (fatigue and lower extremity function) were observed, but other Neuro-QoL domains were not strongly correlated with PROMIS-10 questions. PROMIS-10 Global Physical Health had stronger relationship to EDSS and disease category compared to the Global Mental Health. ConclusionsPROMIS-10 questions and global scores are highly correlated with the corresponding domains of SF-36 in PwMS. Neuro-QoL provides different information regarding HRQOL since different domains are being measured.

Persistently Decreased Quality of Life and its Determinants in Previous Illicit Androgen Users.

Quality of life (QoL) has never been assessed in previous illicit users of androgens years following androgen cessation. Therefore, the objective of this study was to assess QoL in previous illicit androgen users compared with current illicit androgen users and controls who had never used androgens. Cross-sectional study including men involved in recreational strength training grouped according to their history of androgen use. We used the RAND Short-Form-36 questionnaire to assess physical and mental health-related QoL. We included 77 previous and 118 current androgen users and 39 healthy nonusers. The mean (SD) age of all participants was 33 (8) years. The elapsed duration since androgen cessation, geometric mean (95% CI), was 2.0 (1.5-2.6) years in former users. Median (25th-75th percentiles) serum total testosterone was lower in former users than controls, 14 (11-17) vs 19 (16-21) nmol/L, P < .001. Previous users displayed lower mean (SD) across both mental and physical (PCS) component summary scores, 48 (10) vs 54 (4) (P = .004) and 48 (9) vs 53 (3) (P = .002) compared with controls.Using multivariate linear regressions, evaluating physical and mental component scores as dependent variables, lower serum total testosterone, longer duration since androgen cessation, study recruitment from an endocrine outpatient clinic, and established chronic diseases were all independently associated with reduced QoL in previous users, P < .05. Previous illicit androgen users exhibited reduced QoL 2 years after androgen discontinuation, which may be a persistent condition.

Multimorbidity in persons with non-traumatic spinal cord injury and its impact on healthcare utilization and health outcomes.

Cross-sectional surveyin Canada. To explore multimorbidity (the coexistence of two/more health conditions) in persons with non-traumatic spinal cord injury (NTSCI) and evaluate its impact on healthcare utilization (HCU) and health outcomes. Community-dwelling persons. Data from the Spinal Cord Injury Community Survey (SCICS) was used. A multimorbidity index (MMI) consisting of 30 secondary health conditions (SHCs), the 7-item HCU questionnaire, the Short Form-12 (SF-12), Life Satisfaction-11 first question, and single-item Quality of Life (QoL) measure were administered. Additionally, participants were grouped as "felt needed healthcare was received" (Group 1, n = 322) or "felt needed healthcare was not received" (Group 2, n = 89) using the HCU question. Associations among these variables were assessed using multivariable analysis. 408 of 412 (99%) participants with NTSCI reported multimorbidity. Constipation, spasticity, and fatigue were the most prevalent self-reported SHCs. Group 1 had a higher MMI score compared to Group 2 (p < 0.001). A higher MMI score correlated with the feeling of not receiving needed care (OR1.4, 95% CI 1.08-1.21), lower SF-12 (physical/mental component summary scores), being unsatisfied with life, and lower QoL (all p < 0.001). Additionally, Group 1 had more females (p < 0.001), non-Caucasians (p = 0.034), and lower personal annual income (p = 0.025). Persons with NTSCI have multimorbidity, and the MMI score was associated with increased HCU and worse health outcomes. This work emphasizes the critical need for improved healthcare and monitoring. Future work determining specific thresholds for the MMI could be helpful for triage screening to identify persons at higher risk of poor outcomes.

753-P: Tirzepatide Improves HRQoL Compared with Insulin Lispro in Poorly Controlled Basal Insulin–Treated Adults with Long-Standing T2D (SURPASS-6)

Tirzepatide, a first-in-class GIP/GLP-1 receptor agonist, resulted in significant improvements in HbA1c and body weight compared to insulin lispro (iLispro) at 52 weeks in the SURPASS-6 study. We compared the HRQoL of participants with T2D treated with tirzepatide (all doses pooled) versus iLispro, both added to basal insulin, in SURPASS-6. Adults with T2D and inadequate glycemic control were randomized (1:1:1:3) to tirzepatide 5, 10, or 15 mg QW, or iLispro TID, as add-on to optimized insulin glargine, with or without metformin, for 52 weeks in this open-label, multicenter, Phase 3b study. HRQoL was measured at baseline and endpoint (Week 52) using the SF-36v2 acute form comprised of 8 domains and 2 component summary scores. Overall, 1428 participants with mean baseline age 59 y, T2D duration 14 y, HbA1c 8.8% and BMI 33.1 kg/m2 were randomized. At endpoint, tirzepatide-treated participants had statistically significantly improved scores across all SF-36v2 domains and component summaries compared to iLispro-treated participants, with the largest differences observed in the General health (LS mean [SE] change 3.0 [0.31] vs. -0.1 [0.32]) and Vitality (1.5 [0.31] vs. -1.1 [0.32]) domains (Table). Tirzepatide treatment resulted in greater improvements across multiple domains of HRQoL than iLispro in adults with long standing T2D already treated with basal insulin. Disclosure K.Boye: Employee; Eli Lilly and Company. H.Sapin: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. J.Poon: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. L.Fernandez lando: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. R.Huh: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. M.Wang: None. H.Patel: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. Funding Eli Lilly and Company

953-P: The Use of isCGM Improves the Mental Aspect of Quality of Life in Overweight Individuals with Impaired Glucose Tolerance or Mild Diabetes Mellitus

Objective: We aimed to investigate the relationship between the use of intermittently scanned continuous glucose monitoring (isCGM) and quality of life (QoL) in overweight individuals with impaired glucose tolerance (IGT) or mild type 2 diabetes mellitus (T2DM). Methods: Forty overweight individuals (BMI ≥25kg/m2) having IGT or mild T2DM (without taking antidiabetic drugs and with HbA1c ≤7.0%) were recruited. We randomly divided the subjects into two groups: those using isCGM in addition to diet and exercise therapy (isCGM group) and those treated with diet and exercise therapy alone (control group). We conducted a 6-month intervention and assessed QoL using SF36 before and after the intervention. Results: One participant in the isCGM group withdrew the consent. We thus analyzed 19 participants in the isCGM group (53.8±11.5 years old, BMI 35.2±5.7 kg/m2, HbA1c 6.2±0.3%) and 20 participants in the control group (54.3±14.0 years old, BMI 31.6±6.8 kg/m2, HbA1c 6.2±0.3%). There were no differences in changes in each subscale of the SF36 and component summary scores between the isCGM and control groups. In the isCGM group, Mental Component Summary (MCS) improved significantly after the intervention (43.6/48.2, p&amp;lt;0.05). Changes in SD and Time Above Range (TAR) were negatively correlated with changes in general health perception (GH) (r= -0.50, p&amp;lt;0.05; r= -0.53, p&amp;lt;0.05). The number of scans positively correlated with vitality, mental health, and MCS before and after the intervention. Conclusions: The use of isCGM improved MCS in overweight individuals with IGT or drug-naïve T2DM. The improvement in SD and TAR with the use of isCGM likely contributed to the improvement in GH. In addition, the number of scans correlated with MCS, suggesting that vitality and mental health are essential for the effective use of isCGM. Disclosure S. Nishikage: None. Y. Nakagawa: None. Y. Hirota: Other Relationship; Lilly, Sanofi K.K., Terumo Corporation, Sumitomo Dainippon Pharma Co., Ltd. Research Support; Sumitomo Dainippon Pharma Co., Ltd. K. Yoshimura: None. M. Ueda: None. A. Yamamoto: None. T. Takayoshi: None. A. Matsuoka: None. M. Takahashi: None. K. Yokota: None. T. Nakamura: None. K. Sakaguchi: Research Support; Sumitomo Dainippon Pharma Co., Ltd. W. Ogawa: Advisory Panel; Abbott Japan Co., Ltd. Research Support; Boehringer Ingelheim Japan, Inc., Eli Lilly Japan K.K., Novo Nordisk, Teijin Pharma Limited, Kowa Company, Ltd., Sumitomo Dainippon Pharma Co., Ltd., Abbott Japan Co., Ltd. Speaker's Bureau; Sumitomo Dainippon Pharma Co., Ltd.

Two-Year Clinical and Radiologic Outcomes Following Surgical Treatment of Insertional Achilles Tendinopathy Using a Central Tendon-Splitting Approach.

If conservative treatment of insertional Achilles tendinopathy (IAT) fails, surgery is often considered. Various surgical approaches have been used including the central Achilles tendon splitting approach. This study aimed to report the 2-year clinical and radiologic outcomes after surgical treatment of IAT with a central tendon-splitting approach. Seventy-five cases of IAT treated surgically via the open central tendon-splitting approach were analyzed. Clinical outcomes included the American Orthopaedic Foot & Ankle Society (AOFAS) hindfoot score, visual analog scale (VAS), 36-Item Short Form Health Survey (SF-36) physical (PCS) and mental (MCS) component summary scores, all measured at baseline and 6 and 24 months postoperatively. Radiologic parameters measured included the Fowler-Philip angle (FPA) and parallel pitch lines (PPL). Three patients had clinically significant wound issues but healed completely by 3 weeks. Mean AOFAS score improved from 45.63 preoperatively to 94.71 at 24 months. Mean VAS score improved from 6.73 preoperatively to 0.55, mean SF-36 PCS from 35.98 to 48.74, and mean SF-36 MCS from 53.04 to 55.43 at 24 months. Satisfaction at 2 years was 94.3%. Mean FPA decreased from 62.0 degrees preoperatively to 34.0 degrees postoperatively. PPL was positive in 82.7% (62 of 75) of cases preoperatively, decreasing to 1.3% (1 of 75) postoperatively. Increasing age and higher preoperative VAS and SF-36 MCS scores were significantly associated with improvements in postoperative AOFAS, SF-36 PCS, and MCS scores. Surgical treatment of IAT via the central tendon-splitting approach achieved substantial improvements in all patient-reported outcome measures measured. These excellent clinical outcomes continued to show improvement 2 years postoperatively. Level III, retrospective cohort study.

Comparison of long-term quality of life based on surgical procedure in patients with rectal cancer.

Reports on the long-term quality of life (QOL) over 3 years after surgery in patients who have undergone surgery for rectal cancer are limited. Therefore, we aimed to evaluate the long-term QOL of patients who underwent high anterior resection (HAR), low anterior resection (LAR), internal sphincter resection (ISR), or abdominoperineal resection (APR) for rectal cancer. A questionnaire regarding QOL was sent to 360 patients with rectal cancer who underwent curative resection by HAR, LAR, ISR, or APR between January 2005 and December 2015. QOL was assessed using the short-form 36 (SF-36) and modified fecal incontinence QOL (mFIQL) questionnaire. QOL between surgical procedures was analyzed using a multivariate model adjusted for age, sex, and postoperative time. A total of 144 patients responded with a median follow-up period of 94 months (range 38-233 months). According to surgical procedure, HAR was performed in 26 patients, LAR in 80 patients, ISR in 32 patients, and APR in 6 patients. Patients who underwent HAR had significantly better mFIQL scores than those who underwent LAR and ISR (p=0.013 and p=0004, respectively) and significantly better role/social component summary scores on the SF-36 subscales (p=0.007). No difference was observed in the mFIQL scores between patients who underwent ISR and those who underwent APR (p=0.8423). In addition, postoperative anastomotic leakage sutures did not influence the mFIQL and SF-36 scores after surgery. The QOL of patients who underwent anus-preserving surgery was best in the HAR group, with the QOL of other groups similar to the APR group. These results suggest that anus- preserving surgery is acceptable from a QOL standpoint. However, a colostomy may be a more satisfactory procedure in some patients.

Household socioeconomic status relates to specific hippocampal subfield volumes across development.

The hippocampus is composed of cytoarchitecturally distinct subfields that support specific memory functions. Variations in total hippocampal volume across development have been linked to socioeconomic status (SES), a proxy for access to material resources, medical care, and quality education. High childhood household SES is associated with greater cognitive abilities in adulthood. Currently, it is not known whether household SES differentially impacts specific hippocampal subfield volumes. We assessed susceptibility of subfields to variations in household SES across development in a sample of 167 typically developing 5- to 25-year-old. Bilateral cornu ammonis (CA) 1-2, combined CA3-dentate gyrus (DG), and subiculum (Sub) volumes were measured by highly reliable manual segmentation of high-resolution T2-weighted images and adjusted for intracranial volume. A summary component score of SES measures (paternal education, maternal education, and income-to-needs ratio) was used to examine variability in volumes across ages. We did not identify age-related differences in any of the regional volumes, nor did age modify SES-related effects. Controlling for age, larger volumes of CA3-DG and CA1-2 were associated with lower SES, while Sub volume was not. Overall, these findings support the specific impact of SES on CA3-DG and CA1-2 and highlight the importance of considering environmental influences on hippocampal subfield development.

Physical function across the lifespan in adults with multiple sclerosis: An application of the Short Physical Performance Battery.

Multiple sclerosis (MS) typically has its onset in early and middle adulthood, but the population is steadily becoming more dominated by older adults. One of the primary consequences of both MS and aging involves declines of lower extremity physical function and mobility. This cross-sectional study compared physical function status based on Short Physical Performance Battery (SPPB) summary and component scores between persons with MS and healthy controls across 6 age groups. We further examined associations between SPPB summary scores and component scores as well as associations between summary scores and measures of physical and cognitive function for identifying the strongest correlates of SPPB summary scores. The study involved secondary analysis of cross-sectional data from multiple studies. Ambulatory adults with MS who were relapse-free for the last 30 days were recruited, and controls were recruited based on similar criteria to adults with MS except without the diagnosis of MS or relapses. The sample of 345 persons with MS and 174 controls completed questionnaires regarding demographic and clinical information and underwent assessments of physical and cognitive function including the SPPB, 6-Minute Walk, Timed 25-Foot Walk, Symbol Digit Modalities Test, California Verbal Learning Test-Second Edition, and Brief Visuospatial Memory Test-Revised. The two-way ANOVA indicated a main effect of MS status (F(5,500)=34.74, p<.01, η2=0.065), a main effect of age (F(1,500)=3.88, p<.01, η2=0.037), and no MS status by age interaction (F(5,500)=1.20, p=.31, η2=0.012) on SPPB scores. The bivariate correlation analysis indicated that summary SPPB scores were associated with component SPPB scores in the overall samples of persons with MS (rs=0.71 to 0.83) and controls (rs=0.42 to 0.91) as well as within most age groups of MS (rs=0.63 to 0.91) and controls (rs=0.34 to 1.00). The associations between SPPB scores and physical function outcomes were larger in the sample of persons with MS (rs=-0.72 to 0.76) than controls (rs=-0.47 to 0.48). SPPB scores were further significantly associated with scores on cognitive outcomes in persons with MS (rs=0.31 to 0.43), whereas these associations were weaker in controls (rs=0.09 to 0.32). Overall, the associations between SPPB scores and physical function outcomes were stronger than the associations between SPPB scores and cognitive function outcomes. Overall, MS status and aging have additive effects on physical function, and the summary SPPB score may be driven by a specific component within each age group. SPPB scores may be driven more by mobility rather than cognition, and are consistent with cognitive-motor coupling in MS. The novelty of this study provides evidence of worsening physical function based on the application of the SPPB and its scores across the lifespan in persons with MS and controls, and this has important implications particularly given the increasing prevalence of older adults with MS.

The burden of nonalcoholic steatohepatitis (NASH) in the United States

BackgroundThere is limited data on the comparative economic and humanistic burden of non-alcoholic steatohepatitis (NASH) in the United States. The objective was to examine the burden of disease comparing NASH to a representative sample of the general population and separately to a type 2 diabetes mellitus (T2DM) cohort by assessing health-related quality of life (HRQoL) measures, healthcare resource use (HRU) and work productivity and activity impairment (WPAI).MethodsData came from the 2016 National Health and Wellness Survey, a nationally representative patient-reported outcomes survey conducted in the United States. Respondents with physician-diagnosed NASH, physician-diagnosed T2DM, and respondents from the general population were compared. Humanistic burden was examined with mental (MCS) and physical (PCS) component summary scores from the Short-Form (SF)-36v2, concomitant diagnosis of anxiety, depression, and sleep difficulties. Economic burden was analysed based on healthcare professional (HCP) and emergency room (ER) visits, hospitalizations in the past six months; absenteeism, presenteeism, overall work impairment, and activity impairment scores on WPAI questionnaire. Bivariate and multivariable analysis were conducted for each outcome and matched comparative group.ResultsAfter adjusting for baseline demographics and characteristics, NASH (N = 136) compared to the matched general population cohort (N = 544), reported significantly lower (worse) mental (MCS 43.19 vs. 46.22, p = 0.010) and physical (PCS 42.04 vs. 47.10, p < 0.001) status, higher % with anxiety (37.5% vs 25.5%, p = 0.006) and depression (43.4% vs 30.1%, p = 0.004), more HCP visits (8.43 vs. 5.17), ER visits (0.73 vs. 0.38), and hospitalizations (0.43 vs. 0.2) all p’s < 0.05, and higher WPAI scores (e.g. overall work impairment 39.64% vs. 26.19%, p = 0.011). NASH cohort did not differ from matched T2DM cohort (N = 272) on mental or work-related WPAI scores, but had significantly worse physical status (PCS 40.52 vs. 44.58, p = 0.001), higher % with anxiety (39.9% vs 27.8%, p = 0.043), more HCP visits (8.63 vs. 5.68, p = 0.003) and greater activity impairment (47.14% vs. 36.07%, p = 0.010).ConclusionThis real-world study suggests that burden of disease is higher for all outcomes assessed among NASH compared to matched general controls. When comparing to T2DM, NASH cohort has comparable mental and work-related impairment but worse physical status, daily activities impairment and more HRU.

P434 Upadacitinib improved fatigue and health-related quality of life in patients with moderately to severely active Crohn’s disease: Results from induction and maintenance phase 3 trials

Abstract Background Upadacitinib (UPA) demonstrated efficacy and safety in the phase 3 U-EXCEED (NCT03345836) and U-EXCEL (NCT03345849) induction, and U-ENDURE (NCT03345823) maintenance trials in patients (pts) with moderately to severely active Crohn’s disease (CD). We evaluated the impact of UPA on health-related quality of life (QoL) in pts with CD. Methods Clinical responders to UPA 45 mg (UPA45) in U-EXCEED and U-EXCEL induction trials were re-randomised at Week 12 (1:1:1) to UPA 15 mg (UPA15), UPA 30 mg (UPA30), or placebo (PBO) for 52 weeks of maintenance treatment in U-ENDURE; data from these induction and maintenance trials were included in this analysis. Endpoints included the percentage of pts who achieved an Inflammatory Bowel Disease Questionnaire (IBDQ) response (≥16 points), IBDQ remission (≥170 points), a meaningful within-person change (MWPC) from baseline of ≥9 points on the Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F), and of ≥4.1 and ≥3.9 points on the 36-item Short-Form Health Survey (SF-36) physical (PCS) and mental (MCS) component summary scores, respectively, at Weeks 4/12 of induction and Week 52 of maintenance. Adjusted risk differences compared to PBO, 95% confidence intervals, and p-values were calculated based on the Cochran-Mantel-Haenszel test. Results In U-EXCEED (n=324 UPA; 171 PBO) and U-EXCEL (n=350 UPA; 176 PBO), a greater proportion of UPA45- vs PBO-treated pts had an IBDQ response at Weeks 4 (U-EXCEED: 71.6% vs 47.6%; U-EXCEL: 71.0% vs 50.2%; p≤0.001) and 12 (U-EXCEED: 63.9% vs 44.4%; U-EXCEL: 68.4% vs 46.5%; p≤0.001; Figure 1). In UPA- vs PBO-treated pts, a greater proportion achieved IBDQ remission at Weeks 4 (U-EXCEED: 41.4% vs 22.4%; U-EXCEL: 44.2% vs 23.7%; p≤0.001) and 12 (all p≤0.001; Figure 1). At Week 12, a significantly greater proportion of UPA-treated pts vs PBO demonstrated a MWPC in FACIT-F, and SF-36 PCS and MCS from baseline (p≤0.001; Figure 1). Similar results for UPA45 vs PBO were achieved as early as Week 4 in FACIT-F (U-EXCEED: 42.3% vs 20.0%; U-EXCEL: 42.0% vs 27.0%), SF-36 PCS (U-EXCEED: 56.6% vs 37.2%; U-EXCEL: 53.0% vs 37.5%; p≤0.001) and MCS (p≤0.05). In the maintenance study U-ENDURE (n=168 UPA30; 169 UPA15; 165 PBO), a greater proportion of pts receiving UPA15 and UPA30 vs PBO achieved an IBDQ response (39.1%, 53.5% vs 20.4%), IBDQ remission (36.1%, 45.2% vs 13.5%), and a meaningful improvement from baseline in FACIT-F, SF-36 PCS and MCS through 52 weeks (p≤0.01; Figure 2). Conclusion In pts with moderately to severely active CD, UPA treatment significantly improved fatigue, disease-specific and generic QoL, compared with PBO, as early as Week 4 of induction. The effects were sustained through 52 weeks of maintenance treatment.

Health-Related Quality of Life in Patients with Hemophilia A or B with Inhibitors on Concizumab Prophylaxis: Results from the Phase 3 explorer7 Study

Introduction: Concizumab is currently in clinical development as a humanized, monoclonal anti-tissue factor pathway inhibitor antibody that enhances the initiation phase of coagulation. In the phase 3 explorer7 study (NCT04083781), patients with hemophilia A or B with inhibitors received once-daily subcutaneous prophylactic treatment with concizumab for the prevention of bleeding episodes. Patients with hemophilia generally report impaired health-related quality of life (HRQoL), particularly in the areas of pain and physical functioning. Here, we present an analysis of changes in reported HRQoL for patients treated with concizumab for 24 weeks in the explorer7 study. Methods: A total of 133 male patients aged ≥12 years were randomized to no prophylaxis (arm 1) or concizumab prophylaxis (arm 2) or assigned to non-randomized concizumab prophylaxis (arms 3 or 4) depending on the treatment they received before entering the study. All patients were asked to complete the 36-item Short Form Health Survey (SF-36v2; Ware & Sherbourne. Med Care 1992; 30:473-83) and patients aged ≥17 years could complete the Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL; von Mackensen & Gringeri, Handbook of Disease Burdens and Quality of Life 2010:1896-920; von Mackensen et al.Haemophilia 2017;23(3):383-391; Wyrwich et al.Haemophilia 2015;(5):578-84). Both questionnaires could be completed at baseline (Week 0), and at Weeks 4, 8, 16 and 24. The SF-36v2 health scales "bodily pain" (BP) and "physical functioning" (PF) were analyzed as key secondary endpoints. Changes in Haem-A-QoL total score and "physical health" score, as well as the remaining SF-36v2 health scales and component summary scores, were analyzed as exploratory endpoints. Unfortunately, due to technical issues, several patient-reported outcomes recordings could not be collected for statistical analysis. As the preplanned statistical analysis could not be implemented, a post-hoc mixed model for repeated measures (MMRM) was applied after the randomization code was broken. The MMRM analysis included patients from arms 1 and 2 who had completed the questionnaire at baseline and had at least one post-baseline visit on the new dosing regimen. Results: The mean estimated change in score from baseline to Week 24 on the SF-36v2 BP scale was 2.2 points (95% CI: -5.1; 9.5) for patients not on prophylaxis (n=9), and 9.2 points (95% CI: 5.1; 13.3) for those on concizumab (n=23), which yielded an estimated treatment difference (ETD) of 7.0 points (95% CI: -1.6; 15.6) (Figure 1). For the SF-36v2 PF scale, the mean change in score from baseline to Week 24 was 1.2 points (95% CI: -4.8; 7.1) in the no prophylaxis arm (n=9) versus 4.5 points (95% CI: 0.8; 8.2) in the concizumab treatment arm (n=23), which yielded an ETD of 3.3 points (95% CI: -3.8; 10.4) (Figure 1). While BP and PF scores were not significantly different between both arms, several other health scales, including "general health", "mental health", "role-emotional" and "vitality", showed statistically significant differences (Figure 1). The total score from the Haem-A-QoL questionnaire showed a significant improvement for patients on concizumab prophylaxis (n=13) compared to no prophylaxis (n=4) (ETD: -22.6 points [95% CI: -42.5; -2.7]; Figure 2). A significant difference was not observed for "physical health", but there were significant improvements in "feeling", "treatment", "view of yourself", and "sport and leisure" (Figure 2). Conclusion: Patients on daily subcutaneous concizumab prophylaxis generally reported better scores on generic (SF-36v2) and disease-specific (Haem-A-QoL) questionnaires when compared with patients treated on-demand. The differences were mostly in favor of concizumab, with the largest ETDs being related to mental and general health. SF-36v2 and Haem-A-QoL are being investigated as part of the explorer8 concizumab phase 3 study in patients with hemophilia A or B without inhibitors. These data reflect the potential benefit of concizumab prophylaxis to improve HRQoL in patients with hemophilia A and B with inhibitors. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Abstract 13142: Development of a 30-Day Health Status Prediction Model for High-Risk Transfemoral Carotid Artery Stenting Using a Simple Machine Learning Algorithm

Introduction: Individualized prediction models for transfemoral carotid artery stenting (TF-CAS) health status outcomes have not been developed. We applied a machine learning algorithm to identify the most robust pre-procedural predictors of 30-day TF-CAS health status, and validated models across different health status measures to maximize generalizability. Methods: The 390-center SAPPHIRE registry enrolled high-risk patients undergoing TF-CAS from 2006-2014, containing two cohorts with pre-procedural and 30-day health status assessments: Cohort 1 (n=4,667) had EQ-5D-3L (Index and Visual Analog Scale [VAS]); Cohort 2 (n=3,594) had SF-36 (Mental [MCS] and Physical [PCS] Component Summary scores). A random forest algorithm ranked the importance of 50 pre-procedural variables (pre-procedural health status, modified Rankin and NIH stroke scores, demographics, comorbidities, and disease characteristics) for 30-day health status in each cohort. Selection thresholds were based on visual inspection of the importance plots to evaluate the information gain value of each variable considered. Overlapping variables across health status measures were retained to develop a multivariable linear regression model to predict 30-day health status in each cohort. Results: Importance plot ( Figure ) inspection yielded 23 variables, each of which had importance values &gt;15% for ≥2 of the 4 health status measures. From these, 7 overlapping variables were ultimately retained in the linear regression analyses that predicted ~40% of the variance (R 2 =.39-.45) for 30-day TF-CAS health status, including pre-procedural health status, age, heart failure, history of stroke, Rankin stroke score, renal disease, and pulmonary disease. Conclusions: Seven easily assessed pre-procedural variables robustly predicted TF-CAS health status. This model can inform the risk-benefit analysis underlying the TF-CAS medical decision-making process for patients and clinicians.

Associations of Symptom Clusters and Health Outcomes in Adult Survivors of Childhood Cancer: A Report From the St Jude Lifetime Cohort Study.

To identify symptom clusters among adult survivors of childhood cancers and test associations with health-related quality of life (HRQOL) and physical and neurocognitive performance. This cross-sectional study included 3,085 survivors (mean age at evaluation 31.9 ± 8.3 years; mean years from diagnosis 28.1 ± 9.1) participating in the St Jude Lifetime Cohort Study. Survivors self-reported the presence of 37 symptoms capturing 10 domains (cardiac, pulmonary, sensory, motor/movement, nausea, pain, fatigue, memory, anxiety, and depression). The Short Form-36's Physical/Mental Component Summaries assessed HRQOL; the Physical Performance Test evaluated physical performance; and neurocognitive batteries tested attention, processing/psychomotor speed, memory, and executive function. Latent class analysis identified subgroups of survivors experiencing different patterns of symptom burden (ie, symptom clusters). Multivariable regression models identified risk of cluster membership and tested associations with health outcomes. Four symptom clusters were identified including cluster 1 (prevalence 52.4%; low physical, somatization, and psychologic domains), cluster 2 (16.1%; low physical, moderate somatic, and high psychologic domains), cluster 3 (17.6%; high physical, moderate somatic, and low psychologic domains), and cluster 4 (13.9%; high in all three domains). Compared with cluster 1, survivors in cluster 4 were more likely to have less than high school education (odds ratio [OR], 7.71; 95% CI, 4.46 to 13.31), no insurance (OR, 1.49; 95% CI, 1.04 to 2.13), and exposure to corticosteroids (OR, 1.76; 95% CI, 1.02 to 3.03); survivors in cluster 3 were more likely to have received platinum agents (OR, 2.22; 95% CI, 1.34 to 3.68) and brain radiation ≥ 30 Gy (OR, 3.99; 95% CI, 2.33 to 6.86). Survivors in cluster 4 reported the poorest Physical Component Summary/Mental Component Summary scores (31.0/26.7) and physical and neurocognitive performance versus survivors in the other clusters (P < .001). Nearly 50% of survivors had moderate to high multisymptom burden, which was associated with sociodemographic, treatment factors, HRQOL, and functional outcomes.