Improvement In Physical Component Summary Research Articles

E071 Bimekizumab demonstrates sustained improvement in quality of life up to week 52 in patients across the full spectrum of axial spondyloarthritis: results from two phase 3 studies

Abstract Background/Aims Bimekizumab (BKZ), a monoclonal IgG1 antibody that selectively inhibits IL-17F in addition to IL-17A, has demonstrated rapid and clinically meaningful improvements in disease activity in patients across the full spectrum of axial spondyloarthritis (axSpA; active non-radiographic axSpA [nr-axSpA; BE MOBILE 1, NCT03928704] and ankylosing spondylitis [AS]/ radiographic axSpA [r-axSpA; BE MOBILE 2, NCT03928743]). Here we report health-related quality of life (HRQoL) outcomes (ASQoL, SF-36 and EQ-5D-3L) from the two parallel phase 3 studies up to Week (Wk)52. Methods BE MOBILE 1 and 2 have similar study designs: 16-Wk double-blind placebo (PBO)-controlled period followed by 36-Wk maintenance period. Patients were randomised (1:1 in BE MOBILE 1; 2:1 in BE MOBILE 2) to receive BKZ 160 mg Q4W or PBO. All patients received BKZ 160 mg Q4W from Wk16 to Wk52. We report ASQoL, SF-36 - physical component summary (PCS) and SF-36 - mental component summary (MCS) scores up to Wk52 and proportion of patients with ’no problem’ in each of the 5 EQ- 5D-3L domains (mobility, selfcare, usual activity, pain/discomfort, and anxiety/ depression). The results are presented as observed cases (OC), and missing data were imputed using multiple imputation (MI). Results 254 nr-axSpA (BKZ: 128; PBO: 126) and 332 r-axSpA(BKZ: 221; PBO: 111) patients were randomised with 86.6% and 89.8% completing to Wk52, respectively. At Wk16, BKZ treated patients reported greater improvements in ASQoL and SF-36 PCS compared to PBO (p < 0.001, all comparisons, using Analysis of Covariance [ANCOVA]). By Wk52, these improvements across all HRQoL outcomes were sustained in BKZ treated patients and improved in patients who switched to BKZ at Wk16. (Table). The baseline SF-36 MCS levels indicated that on average patients had normal scores which remained unchanged throughout the study. The proportion of patients reporting ‘no problem’ across all five EQ-5D-3L domains improved over time up to Wk52. In patients with ASQoL ≥4 at baseline, >60% of patients had ≥4-point improvement in ASQoL at Wk52. Conclusion Across the axSpA disease spectrum, treatment with BKZ compared with PBO at Wk16 significantly improved quality of life outcomes measured by EQ-5D-3L, ASQoL and SF-36 PCS scores which were sustained to Wk52. Disclosure N.D. McKay: Consultancies; NDM has received consultancy fees from Gilead and UCB Pharma. Other; NDM has received a registration fees for attendance at a conference from Abbvie, Novartis and UCB Pharma, NDM has received a fee for lecturing from Gilead, NDM has taken part in education meetings, programmes and examinations for a wide range of pharmaceutical companies. A. Bennett: Honoraria; AB received teaching honorarium from Abbvie Ltd, Pfizer, UCB Pharma, Novartis and Biogen. Grants/research support; AB received research grants from Pfizer. Other; AB received advisory board fees from Abbvie Ltd, Pfizer, UCB Pharma, MSD, Novartis and Lilly. N. Goodson: Honoraria; NG has received honoraria from UCB Pharma and Novartis. C. Fleurinck: Other; CF is an employee of UCB Pharma. C. de la Loge: Consultancies; CdlL is a consultant to UCB Pharma, Brussels, Belgium. U. Massow: Other; UM is an employee of UCB Pharma. V. Taieb: Other; VT is employee of UCB Pharma. H. Marzo-Ortega: Grants/research support; HMO has received grants/research support from Janssen, Novartis and UCB Pharma. Other; HMO has received honoraria and/or consultancy and/or speaker fees from Abbvie, Biogen, Celgene, Janssen, Eli-Lilly, Moonlake, Novartis, Pfizer and UCB Pharma. K. Gaffney: Shareholder/stock ownership; KG is a shareholder of Rheumatology Events. Grants/research support; KG has received grants/research support from NASS, Versus Arthritis, AbbVie, Pfizer, UCB Pharma, Norvartis, Eli Lilly, Cellgene, Celltrion, Janssen, Gilead and Biogen. Other; KG has received honoraria/ consultation fees from Novartis, AbbVie, UCB Pharma, Lilly and Pfizer, KG has received speaker’s bureau from Novartis, UCB Pharma, AbbVie and Lilly; meeting expenses from AbbVie, Lilly, Roche, Novartis, Pfizer and UCB Pharma.

Mirikizumab Improves Quality of Life in Patients With Moderately-to-Severely Active Ulcerative Colitis: Results From the Phase 3 LUCENT-1 Induction and LUCENT-2 Maintenance Studies.

Mirikizumab, an anti-IL-23p19 antibody, demonstrated efficacy in phase 3, randomized, double-blind, placebo-controlled LUCENT-1 (induction/NCT03518086) and LUCENT-2 (maintenance/NCT03524092) ulcerative colitis (UC) studies. We evaluated the effect of mirikizumab on quality-of-life (QoL) outcomes in these studies. In LUCENT-1, 1162 patients with moderately-to-severely active UC were randomized 3:1 to receive mirikizumab 300 mg intravenous or placebo every 4 weeks (Q4W) for 12 weeks. In LUCENT-2, mirikizumab induction responders (N = 544) were re-randomized 2:1 to receive mirikizumab 200 mg subcutaneous or placebo Q4W through week (W) 40 (W52 of treatment). QoL was assessed at W12 and W52 using patient-reported outcomes. Treatments were statistically compared using analysis of covariance model (continuous outcomes) and Cochran-Mantel-Haenszel test (binary outcomes). At W12 and W52, mirikizumab showed significant improvement in Inflammatory Bowel Disease Questionnaire (IBDQ) total and domain scores (P < .001); 36-Item Short Form Health Survey (SF-36) Physical Component Summary (PCS), Mental Component Summary (MCS), and domain scores (P < .05); EQ-5D-5L scores (P < .001); Work Productivity and Activity Impairment Questionnaire (UC) scores (P < .05); Patient Global Rating of Severity (P < .001); and Patient Global Rating of Change (P < .01) scores. A significantly higher proportion of mirikizumab-treated patients achieved IBDQ response (W12: 72.7% vs 55.8%; W52: 79.2% vs 49.2%; P < .001), IBDQ remission (W12: 57.5% vs 39.8%; W52: 72.3% vs 43.0%; P < .001), and clinically important improvements in PCS (W12: 50.6% vs 41.5%; W52: 61.9% vs 36.9%; P < .01) and MCS (W12: 44.2% vs 37.8%; W52: 51.2% vs 34.6%; P < .05) scores. Mirikizumab improved QoL in patients with moderately-to-severely active UC in phase 3 LUCENT-1 and LUCENT-2 studies. LUCENT-1: NCT03518086; LUCENT-2: NCT03524092.

P194 Bimekizumab is associated with improvement in quality of life in patients across the full spectrum of axial spondyloarthritis at week 16: results from two phase 3 studies

Abstract Background/Aims Bimekizumab (BKZ) is a monoclonal IgG1 antibody that selectively inhibits IL-17F in addition to IL-17A and has demonstrated rapid and clinically meaningful improvements in disease activity (ASAS40, primary outcome measure) in patients across the full spectrum of axial spondyloarthritis (axSpA; active non-radiographic axSpA [nr-axSpA; BE MOBILE 1, NCT03928704] and ankylosing spondylitis [AS] / radiographic axSpA [r-axSpA; BE MOBILE 2, NCT03928743]). Here we report results related to HRQoL instruments (EQ-5D-3L, ASQoL and SF-36) from the two ongoing phase 3 studies at week (wk) 16. Methods BE MOBILE 1 and 2 were conducted in parallel with similar study designs: 16-wk double-blind placebo-controlled period followed by a 36-wk maintenance period. Patients were randomised (1:1 in BE MOBILE 1 and 2:1 in BE MOBILE 2) to BKZ 160 mg Q4W or placebo (PBO). All patients received BKZ 160 mg Q4W from wk16. We report change from baseline (CfB) in the overall EQ-5D-3L utility index, ASQoL and SF-36 Physical component Summary (PCS) scores at wk16 compared to PBO. Missing data is imputed using multiple imputation. Results Overall, 240/254 (94.5%; nr-axSpA) and 313/332 (94.3%; r-axSpA) patients completed the studies up to wk24. Both studies met their primary and all ranked secondary endpoints at wk16. In both studies, BKZ treatment resulted in improved health status as measured with the EQ-5D-3L utility score versus PBO. This improvement at wk16 was consistent across both tumour necrosis factor inhibitor (TNFi)-naive and TNFi-inadequate responders (IR) patients. Significantly greater improvements were also observed with BKZ compared to PBO in ASQoL and SF-36 PCS in both studies (p &amp;lt; 0.001, all comparisons) at wk16 (Table). At wk16, a higher proportion of BKZ- versus PBO-treated patients achieved ≥5-point improvement in SF-36 PCS (nr-axSpA: 64.8% vs 41.3% and r-axSpA: 63.8% vs 49.5%) and ≥4-point improvement in ASQoL (nr-axSpA: 58.6% vs 33.3% and r-axSpA: 57.5% vs 45.0%), respectively. Conclusion Dual inhibition of IL-17A and IL-17F with BKZ improved quality of life measured by EQ-5D-3L, ASQoL and SF-36 PCS scores compared with PBO in patients across the full spectrum of axSpA. Disclosure N. McKay: Member of speakers’ bureau; NM has received Speaker' bureau from Gilead. Other; NM has received travel fees from UCB Pharma and Gilead. A. Bennett: Honoraria; AB has received teaching honorarium from Abbvie Ltd, Pfizer, UCB Pharma, Novartis and Biogen. Grants/research support; AB has received research grants from Pfizer. Other; AB has received advisory board fees from Abbvie Ltd, Pfizer, UCB Pharma, MSD, Novartis and Eli Lilly. N. Goodson: Honoraria; NG has received honoraria from UCB Pharma and Novartis. C. Fleurinck: Other; CF is an employee of UCB Pharma. C. de la Loge: Consultancies; CdlL is a consultant to UCB Pharma, Brussels, Belgium. U. Massow: Other; UM is an employee of UCB Pharma. V. Taieb: Other; VT is employee of UCB Pharma. H. Marzo-Ortega: Grants/research support; HMO has received grants/research support from Janssen, Novartis and UCB Pharma. Other; HMO has received honoraria and/or consultancy and/or speaker fees from Abbvie, Biogen, Celgene, Janssen, Eli-Lilly, Moonlake, Novartis, Pfizer and UCB Pharma. K. Gaffney: Consultancies; KG has received honoraria or consultancy fees from Novartis, AbbVie, UCB Pharma, Eli Lilly and Pfizer. Shareholder/stock ownership; KG is a shareholder of Rheumatology Events. Member of speakers’ bureau; KG has paticipated in speaker's bureau for Novartis, UCB Pharma, AbbVie and Eli Lilly. Grants/research support; KG has received grants/research support from NASS, Versus Arthritis, AbbVie, Pfizer, UCB Pharma, Norvartis, Eli Lilly, Cellgene, Celltrion, Janssen, Gilead and Biogen. Other; KG has received meeting expenses from AbbVie, Eli Lilly, Roche, Novartis, Pfizer and UCB Pharma.

Long-Term Change and Predictors of Change in Physical and Mental Function after Rehabilitation: A Multi-Centre Study

To investigate changes and predictors of change in physical and mental function over a 3-year period after rehabilitation. Prospective cohort. Patients, across diseases, living in western Norway, accepted for somatic specialized interprofessional rehabilitation (n = 984). Physical and mental function were assessed at admittance (baseline), and after 1 and 3 years using the Medical Outcome Study Short Form 36 (SF-36). Associations between changes in SF-36 component summary scores and sense of coherence, pain, disease group (musculoskeletal, neoplasm, cardiovascular, neurological, other), exercise habits and demographic variables were analysed using linear mixed modelling. In the total group, mean (standard deviation) physical component summary scores improved by 2.9 (8.4) and 3.4 (9.3) points at 1 and 3 years, respectively. Mental component summary scores improved by 2.1 (9.7) and 1.6 (10.8) points. Improvement in physical component summary was significantly greater for patients with higher sense of coherence (b = 0.09, p = 0.001) and for the neoplasm disease group (b = 2.13, p = 0.046). Improvement in mental component summary was significantly greater for patients with low sense of coherence (b = -0.13, p = < 0.001) and higher level of education (b = 3.02, p = 0.0302). Interaction with age (physical component summary: b = 0.22, p = 0.039/mental component summary b = 0.51, p = 0.006) indicated larger effect at 1 year than at 3 years. Physical and mental function improved in the total study group over the 3-year period. Sense of coherence at baseline was associated with improved physical and mental function, suggesting that coping resources are important in rehabilitation.

Effect of age on quality of life after gastric bypass: data from the Scandinavian Obesity Surgery Registry.

Whether patients aged 60 years or older should be recommended bariatric surgery is still controversial. To assess the effect of age on health-related quality of life (QoL) over time after gastric bypass. Data from the Swedish national registry for bariatric surgery. Data of 57,215 patients undergoing gastric bypass were retrieved from the Scandinavian Obesity Surgery Register with a follow-up rate at 1,2, and 5 years at 89%, 69%, and 59%, respectively. Patients were divided into 5-years age intervals. Odds ratios for the relative mean changes in QoL were compared by logistic regression. Preoperatively, patients aged 60 years or older scored better on mental aspects (Mental Component Summary score, MCS) of RAND-36 (Short Form Health Survey (higher values better)) as well as OP (Obesity related Problem scale (lower values better)) better than the entire cohort of patients (MCS: mean [95% CI], 46.2 [45.5-46.9] versus 43.5 [43.4-43.7], respectively; OP: mean [95% CI], 55.3 [54.0-56.6] versus 64.1 [63.9-64.4], respectively), whereas the Physical Component Summary (PCS) scores of patients aged 60 years or older were lower (mean [95% CI], 32.3 [31.7-32.8] for the ≥60-yr cohort versus 36.4 [36.2-36.5] for the entire cohort; P < .001 for all). In all age groups, MCS was improved at 1 and 2 years but decreased to baseline at 5 years. The postoperative improvements in PCS and OP were sustained in all age groups. Although the relative increases for PCS and OP in patients aged ≥60 years were somewhat lower compared with the entire cohort at 5 years, the values were well above baseline levels (mean [95% CI], 41.0 [40.0-42.0] versus 32.3 [31.7-32.8] and 22.2 [20.3-24.0] versus 55.3 [54.0-56.6], respectively; P < .001). Mental QoL is transiently improved after bariatric surgery without marked differences between age groups. However, patients aged ≥60 years report pronounced and sustained improvements in physical and obesity-specific QoL 5 years postoperatively. These observations support previous studies that older patients should not be denied bariatric surgery from a risk-benefit perspective, solely based on age.

Preoperative mental distress is associated with poorer physical improvements after revision total hip arthroplasty

AimsDespite the increasing prevalence of mental health disorders in revision arthroplasty patients, the impact of preoperative mental distress on functional outcomes after revision total hip arthroplasty (rTHA) remains unclear. Our study aims to investigate the impact of preoperative mental distress on functional outcomes after rTHA. MethodsProspectively collected data of 84 rTHAs was extracted from a single institution's joint replacement registry. Preoperative Short Form-36 (SF-36) mental component summary (MCS) was used to dichotomize patients to those who were distressed (MCS <50) and non-distressed (MCS ≥50). Multivariable analysis was used to analyze the impact of preoperative mental distress on various outcomes such as the SF-36 physical component summary (PCS), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), Oxford Hip Score (OHS), patient satisfaction and expectation fulfilment at 6 months and 2 years postoperatively. ResultsCompared to non-distressed patients, patients with preoperative mental distress had poorer improvements in PCS (+11.4 vs + 16.9, p = 0.007) and lower rates of minimal clinically important difference (MCID) attainment (55.2% vs 73.0%, p = 0.026) at 2 years postoperatively. However, there were no significant differences in improvements for WOMAC and OHS (p > 0.05). Despite experiencing greater absolute improvements in MCS (+12.1 vs −2.1, p < 0.001) and higher MCID attainment rates (65.5% vs 24.3%, p = 0.005) at 2 years, preoperatively distressed patients had lower MCS levels compared to their non-distressed counterparts at all time points (p-value<0.05). There were no significant differences in patient satisfaction (p = 0.509) or expectation fulfilment (p-value = 0.342) at 2 years postoperatively. ConclusionRevision THA led to substantial mental health improvements in patients with preoperative mental distress. Despite this, preoperative mental distress was associated with poorer physical improvements after surgery.

A randomized controlled trial of an mHealth application with nursing interaction to promote quality of life among community-dwelling older adults.

Using mHealth apps alone at home without the support of healthcare experts could mean that older adults might not fully utilize the functions of the apps, recognize their benefits, and sustain their use. Incorporating an integrated health-social partnership model to support the app usage when further help is needed by the older adults might maximize the apps' benefits in the long term. This study aimed to examine the benefits of adding nursing interaction supported by a health-social partnership model in the use of mHealth, and the sustained beneficial effects on psychological outcomes, including quality of life, self-efficacy, and depression, among older adults after the completion of the program. A three-arm, randomized controlled trial design was adopted. Community-dwelling older adults with chronic pain, hypertension, or diabetes, were randomly assigned to either the mHealth, mHealth with interactivity, or control group. Subjects in both the mHealth and the mHealth with interactivity groups received the mHealth application. In addition, the mHealth with interactivity group received support from a nurse case manager, who was supported by a health-social partnership team. mHealth apps and services from a nurse case manager were not provided to the control group. The primary outcome measure was quality of life, and secondary outcomes were self-efficacy and depression. Data were collected at pre-intervention (T1), post-intervention (T2), and at 3 months post-intervention to measure the sustained effect of the program. There were 74 mHealth+I, 71 mHealth, and 76 control group subjects enrolled in the program. No statistically significant between-group, within-group, and interaction effects between group and time in both physical component summary (PCS) and mental component summary (MCS) scores were found among the three groups. The mHealth group showed an improvement in PCS and depression scores from T1 to T2, sustained at T3; while the mHealth+I group demonstrated improved self-efficacy from T1 to T2, with a decrease at T3. Adding nurse-directed telephone calls may be of little to no benefit at all in the long term. Future studies may consider a longer intervention period to build and sustain quality of life and self-efficacy levels among community-dwelling older adults. www.ClinicalTrials.gov, identifier: NCT03878212.

The impact of minimally invasive palliative decompression of bile ducts on quality of life in patients with distal malignant mechanical jaundice

Palliative treatment for mechanical jaundice is aimed at improving patient quality of life (QoL) and prolonging life. The current QoL studies indicate that there is limited evidence on various techniques for decompression of bile ducts (BD) in terms of their impact on quality of life in patients with distal malignant mechanical jaundice, and their data are contradictory.&#x0D; The aim of the study is to evaluate the effect of minimally invasive palliative techniques on QoL in patients with distal mechanical jaundice (MJ) after palliative decompression of bile ducts.&#x0D; Materials and methods. From 2017 to 2021, 98 patients who underwent palliative decompression of bile ducts for distal MJ of malignant origin were examined. A validated MOS SF‑36 questionnaire was used to assess patient QoL. The survey was conducted before the minimally invasive intervention and 2 months after it. Depending on the technique used for decompression of BD, patients were divided into the following groups: percutaneous transhepatic biliary drainage (PTBD) — 25, internal‑external transpapillary biliary drainage (IETBD) — 19, internal‑external biliary‑jejunal drainage (IEBJD) — 29, and endoscopic retrograde biliary stenting (ERBS) — 25.&#x0D; Results. Before treatment, a low level of patient QoL was detected in all groups on all scales of physical and psychological components of health (all p &gt; 0.05). After 2 months, in the PTBD group, the indicator of the Mental Component Summary (MCS) decreased by an average of 6.9 ± 1.2 points (due to the deterioration of the indicator of social functioning scales by 12.5 ± 5.0 points and mental health by 11.1 ± 1.8 points), while the indicator of the Physical Component Summary (PCS) improved by 11.3 ± 1.1 points. In the IETBD and IEBJD groups, there was an improvement in MCS (by 11.7 ± 1.3 points and 13.0 ± 1.1 points, respectively) and PCS (by 5.2 ± 1.2 and 8.6 ± 1.1 points). With regard to MCS and PCS, slight improvement (by 4.7 ± 2.2 and 2.0 ± 1.9 points) was observed in the ERBS group.&#x0D; Conclusions. The IEBJD technique provided important advantages in comparison with other minimally invasive palliative techniques for decompression of BD in terms of its impact on patient QoL. Patients in the IEBJD group had better PCS scores (on average, 47.3 ± 1.3 points) compared to the IETBD (42.1 ± 1.5 points) and ERBS (39.1 ± 1.3 points, p &lt; 0.05) groups, and those in the PTBD group (46.2 ± 1.4 points) had better scores than patients in the IETBD group (p &lt; 0.05). In the IETBD and IEBJD groups, MCS scores were better 41.9 ± 1.1 and 40.3 ± 1.1 points, respectively) compared to the PTBD (22.6 ± 0.9 points) and ERBS (34.0 ± 1.1 points, p &lt; 0.05) groups, and in the ERBS group, they were better than in the PTBD group.&#x0D;

Improvement in Health-Related Quality of Life Measures after Long-Term, Daily, Subcutaneous Concizumab Prophylaxis in Patients with Hemophilia A/B with and without Inhibitors: Results from the Main and Extension Parts of Phase 2 Clinical Trials

Relative to the general population, health-related quality of life (HRQoL) is impaired in patients with hemophilia, who report increased pain and reduced physical activity. It is therefore of interest to assess the change in HRQoL after long-term prophylactic treatment. Concizumab is an anti-tissue factor pathway inhibitor monoclonal antibody in clinical development for a subcutaneous, prophylactic treatment of patients with hemophilia. Here, we present results from exploratory analyses assessing changes in HRQoL after long-term concizumab treatment (≤126 treatment weeks), which includes data from the main and extension parts of explorer4 (NCT03196284) and explorer5 (NCT03196297) phase 2 trials.Both trials comprised a main part (≤24 weeks), and an extension part (explorer4: ≤94 weeks; explorer5: ≤102 weeks). In explorer4, patients with hemophilia A and B with inhibitors (HAwI/HBwI) were recruited and randomized 2:1 to concizumab prophylaxis (n=17) or recombinant activated factor VII on-demand treatment (n=9). Patients in the on-demand arm switched to concizumab prophylaxis in the extension part. Twenty-six patients were recruited for explorer4, 25 patients were exposed to concizumab in the extension part, and 22 patients completed the trial. In explorer5, 36 patients with severe hemophilia A (HA) were recruited, 32 patients entered the extension part, and 30 patients completed the trial. Patients were asked to complete the 36-item Short Form Health Survey (SF-36v2) at baseline (for this analysis, baseline was defined as the last assessment before first treatment with concizumab), and throughout the main and extension parts. Scoring was conducted according to the SF-36v2 scoring software (version 5.0). Only patients who completed the entire trial were included for analysis.T score points of SF-36v2 domains were used to determine clinically meaningful differences at group level from baseline to the end of the extension part, based on minimally important difference criteria (MID; SF-36v2 manual 3rd edition, 2013). At an individual level, a responder analysis was conducted to identify the proportion of patients who had improved scores in the physical component summary (PCS), physical function (PF) and bodily pain (BP) domains, based on the recommended individual-level response threshold of 3.4, 4.3 and 6.2, respectively (based on 2009 United States general population norms; SF-36v2 manual). The results presented here demonstrate the change in HRQoL before and after long-term concizumab use in the main and extension parts of explorer4 and explorer5.For explorer4, 22 patients (14 HAwI; 8 HBwI) were included in the current exploratory analysis, which showed that the difference in improvement from baseline to end of extension part exceeded the MID thresholds for PCS score (Table 1), at group level. Additionally, the MID thresholds were also met for PF, BP, role-physical, general health, vitality, social functioning, and mental health domains in explorer4 (Table 1). At an individual level, the responder analysis revealed that 63.6%, 54.5% and 50.0% of 22 patients had an improvement that met or exceeded the response threshold for PCS, PF and BP scores. In the 30 patients with severe HA included in the explorer5 analysis, PCS score met the MID threshold for difference in improvement from baseline to end of extension, although large standard deviations were observed (Table 1). At an individual level, the responder analysis revealed that 43.3%, 33.3% and 33.3% of 30 patients had an improvement that met or exceeded the response threshold for PCS, PF and BP scores.While these analyses are exploratory and should be interpreted with caution, they illustrated that patients with HAwI/HBwI reported improved HRQoL after long-term, subcutaneous concizumab prophylaxis, particularly in clinically relevant domains such as PF and BP, suggesting a potential positive effect of concizumab prophylaxis on physical functioning and reduced pain. Interestingly, PCS improvement was observed across all hemophilia subgroups, suggesting better functional health, albeit the large standard deviation reported. The potential beneficial effect of concizumab prophylaxis on HRQoL in hemophilia patients is being investigated further in the ongoing phase 3 trials. [Display omitted] DisclosuresFaller: Novo Nordisk Health Care AG: Current Employment. Marie Tønder: Novo Nordisk Health Care AG: Current Employment. Porstmann: Novo Nordisk Health Care AG: Current Employment.

The Impact of Preoperative Mental Health and Depression on Outcomes After Total Ankle Arthroplasty.

Preoperative mental health and depression have been shown to negatively impact patient-reported outcome measures after a broad array of orthopaedic procedures including total ankle arthroplasty. The hypothesis for this study was that decreased Short Form (SF)-36 Mental Component Summary (MCS) scores will modulate the impact of depression on patient-reported outcome measures after total ankle arthroplasty. All patients undergoing primary total ankle arthroplasty between January 2007 and December 2016 who were enrolled into a prospective outcomes study and who had at least 1-year minimum study follow-up were retrospectively reviewed. Patients were separated into 4 groups based on the presence or absence of an SF-36 MCS score of <35 points and diagnosis of depression. SF-36 Physical Component Summary (PCS) and MCS scores, Short Musculoskeletal Function Assessment (SMFA) function and bother components, and visual analog scale (VAS) pain were collected preoperatively and in the 1 to 2-year follow-up. The Wilcoxon rank sum was used to assess differences in outcomes by depression and low preoperative MCS scores. Multivariable models were then constructed to evaluate between-group differences in change scores according to preoperative SF-36 MCS scores and a diagnosis of depression, with adjustment for baseline patient and treatment characteristics. Patients with depression and those with low preoperative MCS scores had significantly worse final outcome scores along with reduced improvement in SF-36 PCS and VAS pain scores compared with patients without these risk factors. Among patients with depression, low preoperative MCS scores helped to differentiate patients with poor final outcome scores. Similarly, in patients with low preoperative MCS scores, depression helped to differentiate patients with poor final outcome scores. Although patients achieved significant improvements in functional outcomes regardless of cohort, decreased preoperative mental health modulated the impact of depression on outcomes. Patients with diminished preoperative mental health and depression are at an increased risk for sustaining smaller improvements in outcomes. Prognostic Level III. See Instructions for Authors for a complete description of levels of evidence.

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial

Time Course of Physical and Mental Well-being Improvements After Cervical Surgery.

Retrospective cohort study. To elucidate the postoperative time course of improvements in physical and mental well-being in patients with cervical spondylotic myelopathy (CSM). Spinal surgeons should understand the postoperative course in detail. However, data are still needed regarding the time course of improvements in well-being, a fundamental aspect of human life, after cervical surgery for CSM. One hundred consecutive patients who underwent laminoplasty for CSM, with complete clinical data preoperatively and 3 months and 2 years postoperatively, were enrolled. The Short Form-36 physical component summary (PCS) and mental component summary (MCS) scores were used as parameters of physical and mental well-being, respectively, and 4.0 was defined as the minimal clinically important difference (MCID) for both parameters. On average, PCS and MCS scores were significantly improved after surgery (P < 0.001, P=0.004, respectively). Moreover, 64 and 48 patients achieved meaningful improvement (>MCID) in PCS and MCS scores at 3 months postoperatively, with maintained improvement (to 2 yr) in 46/64 (71.9%) and 34/48 patients (70.8%), respectively (PCS vs. MCS: P = 0.912). Additionally, 15 of 36 patients (41.7%) and 8 of 52 patients (15.4%) achieved late improvement (meaningful improvement at 2 yr but not at 3 months) in PCS and MCS scores, respectively (PCS vs. MCS: P = 0.007). In multivariate regression analysis, improvement in cJOA score was significantly associated with PCS improvement, but not MCS improvement, at both 3 months and 2 years (P = 0.001, P > 0.001, respectively). The overall outcome of physical well-being improvement is decided within 3 months postoperatively, in proportion to the recovery in myelopathy, with a relatively high chance of meaningful improvement over the next 21 months. The outcome of improvement in mental well-being is decided within 3 months postoperatively, independently from the recovery in myelopathy, with a low chance of meaningful improvement over the next 21 months.Level of Evidence: 3.

Clinical, Patient-Reported, and Ultrasound Outcomes from an Open-Label, 12-week Observational Study of Certolizumab Pegol in Spanish Patients with Rheumatoid Arthritis with or without Prior Anti-TNF Exposure

ObjectivesTo assess the effectiveness and safety of certolizumab pegol (CZP) in Spanish patients with RA. Materials and methodsSONAR (NCT01526434), a 12-week, open-label, prospective, observational, multicenter study. Patients with active RA for ≥3 months, according to ACR criteria, were treated with CZP (400mg at Weeks 0, 2 and 4, then 200mg every 2 weeks). The primary effectiveness endpoint was change from baseline (CFB) in Health Assessment Questionnaire-Disability Index (HAQ-DI) at Week 12. Other assessments included DAS28(ESR), patient's assessment of arthritis pain (PtAAP-VAS) and Short Form 36-item Health Survey (SF-36) physical component summary (PCS) and mental component summary (MCS). Joint inflammation was investigated using Power Doppler (PD) ultrasound (US), to detect effusion, synovial hypertrophy and synovial PD signal. PDUS outcomes assessed CFB to Week 12 in synovial hypertrophy, effusion and PD signal indices. ResultsA total of 77/80 enrolled patients received ≥1 dose of CZP. The 12-week mean reduction from baseline (SD) was −0.6 (0.6) for HAQ-DI and −2.2 (1.5) for DAS28(ESR). PtAAP-VAS was reduced from baseline (mean [SD]: −36.8 [26.8]) and improvements in SF-36 PCS and SF-36 MCS were reported. Synovial hypertrophy, effusion and PD signal indices were reduced from baseline to Week 12. One death was reported during the study. ConclusionsSpanish patients with RA demonstrated improvements in clinical, PDUS and patient-reported outcomes over 12 weeks of CZP treatment. No new safety signals were identified, and the safety profile was in line with previous CZP studies. These results support previous clinical trial findings investigating CZP treatment for active RA.

Clinical, Patient-Reported, and Ultrasound Outcomes from an Open-Label, 12-week Observational Study of Certolizumab Pegol in Spanish Patients with Rheumatoid Arthritis with or without Prior Anti-TNF Exposure.

To assess the effectiveness and safety of certolizumab pegol (CZP) in Spanish patients with RA. SONAR (NCT01526434), a 12-week, open-label, prospective, observational, multicenter study. Patients with active RA for ≥3 months, according to ACR criteria, were treated with CZP (400mg at Weeks 0, 2 and 4, then 200mg every 2 weeks). The primary effectiveness endpoint was change from baseline (CFB) in Health Assessment Questionnaire-Disability Index (HAQ-DI) at Week 12. Other assessments included DAS28(ESR), patient's assessment of arthritis pain (PtAAP-VAS) and Short Form 36-item Health Survey (SF-36) physical component summary (PCS) and mental component summary (MCS). Joint inflammation was investigated using Power Doppler (PD) ultrasound (US), to detect effusion, synovial hypertrophy and synovial PD signal. PDUS outcomes assessed CFB to Week 12 in synovial hypertrophy, effusion and PD signal indices. A total of 77/80 enrolled patients received ≥1 dose of CZP. The 12-week mean reduction from baseline (SD) was -0.6 (0.6) for HAQ-DI and -2.2 (1.5) for DAS28(ESR). PtAAP-VAS was reduced from baseline (mean [SD]: -36.8 [26.8]) and improvements in SF-36 PCS and SF-36 MCS were reported. Synovial hypertrophy, effusion and PD signal indices were reduced from baseline to Week 12. One death was reported during the study. Spanish patients with RA demonstrated improvements in clinical, PDUS and patient-reported outcomes over 12 weeks of CZP treatment. No new safety signals were identified, and the safety profile was in line with previous CZP studies. These results support previous clinical trial findings investigating CZP treatment for active RA.

Changes in Health-Related Quality of Life During the First Year in Lung Transplant Recipients

BackgroundHealth-related quality of life (HRQOL) has increasingly been accepted as a supplementary outcome measure for patients before and after lung transplantation (LT). This longitudinal study was conducted to recognize the tracks of HRQOL during the first year after transplantation and the main factors associated with HRQOL of LT recipients. The research was conducted in accordance with the 2000 Declaration of Helsinki and the Declaration of Istanbul 2008. The transplant organs were from volunteer donation, and next of kin provided written informed consents of their own free will. No prisoners were used, and donors were neither paid nor coerced. MethodsA total of 118 patients were investigated before and 3, 6, 9, and 12 months post-transplantation. The Medical Outcomes SF-36 (Chinese version) was used to measure the HRQOL. The recipients’ demographic characteristics and clinical data were evaluated to determine the relative contributions to HRQOL outcomes. ResultsRecipients reported a mean physical component summary of 39.62 ± 6.57, 57.90 ± 9.99, 59.15 ± 8.73, 58.79 ± 8.52, and 58.72 ± 8.99 before transplantation and at 3, 6, 9, and 12 months after LT (F = 64.960, P < .001). By 3 months after transplant, patients experienced significant improvement in physical component summary (MD = 18.27, SE = 1.52, P < .001); but between 3 and 12 months, no significant improvement was observed (MD = 0.82, SE = 1.77, P = .645). Patients reported a continuous rise with means of 44.63 ± 5.35, 51.13 ± 10.25, 51.92 ± 9.72, 53.23 ± 10.34, and 55.40 ± 8.83 for the mental component summary before LT and at 3, 6, 9, and 12 months after transplant (F = 13.059, P < .001). By 3 months after transplant, patients experienced significant improvement in mental component summary (MD = 6.50, SE = 1.50, P < .001). Between 3 and 12 months, a continuous significant improvement was observed (MD = 4.27, SE = 1.92, P = .030). The generalized estimated equation showed that age, marital status, residence, disease diagnosis, transplant type, sleep disorders, gastrointestinal complications, and BODE index (body mass index, obstruction, dyspnea, exercise) were all found to be related to HRQOL. ConclusionThe HRQOL of LT patients improved significantly at 3 months after transplantation, but between 3 and 12 months after transplantation, the changes were not obvious. Health practitioners should pay more attention to elderly patients, unmarried patients, patients living in urban areas, patients diagnosed with pneumoconiosis, patients with left single-lung transplantation, patients with sleep disorders, patients with high BODE indexes, and patients with gastrointestinal complications.

Improved Physical Function And Quality Of Life In People With Blood Cancer After An Exercise Intervention

In blood cancer, aerobic physical activity may improve fatigue and depression, though its effect on quality of life and physical function is less clear. Assessment of Fit to Thrive (FTT), a community-based individualised, supervised exercise program for people with blood cancer, may assist with future recommendations. PURPOSE: To assess the effect of the FTT program on physical function and quality of life (QoL) in people with blood cancer. METHODS: The 12-week FTT program utilised progressive aerobic and resistance training, supervised by an Accredited Exercise Physiologist, in small groups (n=6), twice weekly, for 8 weeks, with associated psychosocial and peer support. Physical function was assessed via the six minute walk (6MW), functional forward reach (FFR) and 5 times-sit-to-stand (5TSTS) tests. QoL was measured using the 36-item Short Form Survey Instrument (SF-36) and the Functional Assessment of Cancer Therapy General (FACT-G). Minimally important differences (MID) involved a change of 2 points for the SF-36, 3 points for the FACT-G and +50m for 6MW. RESULTS: Participants (n=106) who attended the FTT program between 2014 and 2016 were included. Significant improvements in functional measures were observed for 6MW (+54.7m [95% CI 40.5, 69.0] p<0.001), 5TSTS (-1.9 sec [95% CI -2.5, -1.2] p<0.001) and FFR (+2.7cm [95% CI 1.5, 4.0] p<0.001), with the MID achieved by 56% for the 6MW. The SF-36 physical component summary (PCS) significantly increased (+4.99 [95% CI 3.29, 6.68] p<0.001) immediately following the intervention, with 68% (n=72) achieving the MID. The improvement in the SF-36 mental component summary (MCS) also achieved statistical significance (+2.81 [95% CI 0.54, 5.08] p=0.02), with 51% (n=54) achieving the MID. FACT-G scores improved significantly from pre- to post-intervention (+5.50 [95%CI 2.52, 8.47], p<;0.001) with 58% (n=62) meeting the MID. MID improvements in PCS and FACT-G were maintained in 77% (n=20/26) and 95% (n=19/20) of participants 3 months following completion of the program. CONCLUSION: The FTT program was effective in improving physical function and QoL, with some evidence for long-term effects. An individually-prescribed exercise program supervised by an Accredited Exercise Physiologist should be considered as part of standard care in people with stable blood cancer.

Machine learning algorithms for prediction of health-related quality-of-life after surgery for mild degenerative cervical myelopathy

BACKGROUNDDegenerative cervical myelopathy (DCM) is the most common cause of spinal cord dysfunction worldwide. Current guidelines recommend management based on the severity of myelopathy, measured by the modified Japanese Orthopedic Association (mJOA) score. Patients with moderate to severe myelopathy, defined by an mJOA below 15, are recommended to undergo surgery. However, the management for mild myelopathy (mJOA between 15 and 17) is controversial since the response to surgery is more heterogeneous. PURPOSETo develop machine learning algorithms predicting phenotypes of mild myelopathy patients that would benefit most from surgery. STUDY DESIGNRetrospective subgroup analysis of prospectively collected data. PATIENT SAMPLESData were obtained from 193 mild DCM patients who underwent surgical decompression and were enrolled in the multicenter AOSpine CSM clinical trials. OUTCOME MEASURESThe mJOA score, an assessment of functional status, was used to isolate patients with mild DCM. The primary outcome measures were change from baseline for the Short Form-36 (SF-36) mental component summary (MCS) and physical component summary (PCS) at 1-year postsurgery. These changes were dichotomized according to whether they exceeded the minimal clinically important difference. METHODSThe data were split into training (75%) and testing (25%) sets. Model predictors included baseline demographic variables and clinical presentation. Seven machine learning algorithms and a logistic regression model were trained and optimized using the training set, and their performances were evaluated using the testing set. For each outcome (improvement in MCS or PCS), the machine learning algorithm with the greatest area under the curve (AUC) on the training set was selected for further analysis. RESULTSThe generalized boosted model (GBM) and earth models performed well in the prediction of significant improvement in MCS and PCS respectively, with AUCs of 0.72 to 0.78 on the training set. This performance was replicated on the testing set, in which the GBM and earth models showed AUCs of 0.77 and 0.78, respectively, as well as fair to good calibration across the predicted range of probabilities. Female patients with a low initial MCS were less likely to experience significant improvement in MCS than males. The presence of certain signs and symptoms (eg, lower limb spasticity, clumsy hands) were also predictive of worse outcome. CONCLUSIONSMachine learning models showed good predictive power and provided information about the phenotypes of mild DCM patients most likely to benefit from surgical intervention. Overall, machine learning may be a useful tool for management of mild DCM, though external validation and prospective analysis should be performed to better solidify its role.

PS1514 PARTICIPATION IN A SPECIALISED EXERCISE INTERVENTION IMPROVED PHYSICAL FUNCTION AND HEALTH‐RELATED QUALITY OF LIFE IN PEOPLE WITH HAEMATOLOGICAL CANCERS

Background:Exercise has been shown to improve quality of life and physical function in patients with solid tumours, such as colorectal, breast and prostate. In haematological cancers, there is some evidence that aerobic physical activity improves fatigue and depression, though its effect on quality of life and physical function is less clear. Assessment of programs, such as Fit to Thrive (FTT), a community‐based individualised, supervised exercise program offered to people with haematological cancers, may assist with future recommendations in this population.Aims:This study aimed to assess the effect of the FTT program on physical function and health‐related quality of life (HRQoL) in people with haematological cancers.Methods:The 12‐week FTT program utilised progressive aerobic and resistance training, supervised by an Accredited Exercise Physiologist, in small groups (n = 6), twice weekly, for 8 weeks, with an additional 4 weeks of associated psychosocial and peer support. Participants had stable disease and were off treatment or on maintenance therapy. Physical function was assessed at baseline and immediately after the intervention via the six minute walk (6MW), functional forward reach (FFR) and 5 times‐sit‐to‐stand (5TSTS) tests. HRQoL was measured using the 36‐item Short Form Survey Instrument (SF‐36) and the Functional Assessment of Cancer Therapy General (FACT‐G) at baseline, immediately post‐intervention and, for a subset of participants, at 3 months post‐intervention. Minimally important differences (MID) involved a change of 2 points for the SF‐36, 3 points for the FACT‐G and +50m for 6MW. A reach of ≤25 cm on the FFR predicted a moderate risk of falls.Results:Participants (n = 106; lymphoma 35%, myeloma 30%, leukaemia 26%, other 8%) who attended the FTT program between 2014 and 2016 were included in this analysis. Significant improvements in functional measures were observed for 6MW (+54.7m [95% CI 40.5, 69.0] p &lt; 0.001), 5TSTS (‐1.9 sec [95% CI −2.5, −1.2] p &lt; 0.001) and FFR (+2.7 cm [95% CI 1.5, 4.0] p &lt; 0.001), with the MID achieved by 56% of participants for the 6MW. Of participants with a predicted moderate risk of falls on initial assessment (n = 6), 67% (n = 4) achieved &gt;25 cm on the FFR after the intervention. The SF‐36 physical component summary (PCS) significantly increased (+4.99 [95% CI 3.29, 6.68] p &lt; 0.001) immediately following the intervention, with 68% (n = 72) of participants achieving the MID. The improvement in the SF‐36 mental component summary (MCS) also achieved statistical significance (+2.81 [95% CI 0.54, 5.08] p = 0.02), with 51% (n = 54) achieving the MID. FACT‐G scores improved significantly from pre‐ to post‐intervention (+5.50 [95%CI 2.52, 8.47], p &lt; 0.001) with 58% (n = 62) of participants meeting the MID. MID improvements in PCS and FACT‐G were maintained in 77% (n = 20/26) and 95% (n = 19/20) of participants 3 months following completion of the program.Summary/Conclusion:The FTT program was effective in improving and maintaining physical function and HRQoL, with some evidence for long‐term effects. An individually‐prescribed exercise program supervised by an Accredited Exercise Physiologist should be considered as part of standard care to improve function and HRQoL in patients with stable haematological cancer.

Patient outcomes after total hip arthroplasty in extreme elderly patients older than 80 years.

While extreme elderly patients (age 80 and above) benefit from joint replacement, there is controversy about whether their physical function improves as much as younger individuals following total hip arthroplasty. We completed a retrospective cohort study comparing extreme elderly total hip arthroplasty (THA) patients to younger patients. We obtained data from a large institutional repository of 2327 consecutive THAs performed from April 2011 through July 2016 at an American academic medical centre. We performed multivariate regression analyses to determine associations between age group and clinically significant improvement in the Patient-Reported Outcome Measurement Information System (PROMIS)-10 physical component summary (PCS) score. Secondary outcomes included the magnitude of PCS change, length of stay (LOS), and facility discharge. There were 187 THAs (8.0%) in patients age ⩾ 80 years compared to 2140 THA procedures in patients < age 80. Extreme elderly patients had similar adjusted odds of achieving clinically significant PCS improvement after THA (p = 0.528) and there were no statistical differences in adjusted postoperative PCS score improvements between the cohorts. Extreme elderly patients were associated with a 0.68 day longer adjusted LOS (p < 0.001) and demonstrated higher adjusted odds of facility discharge following THA (OR 8.96, p < 0.001). Compared to younger patients, extreme elderly individuals had similar adjusted postoperative functional outcomes following THA but utilised substantially more resources in the form of increased time in the hospital and higher rates of facility discharges.

Quality of Life in Hypoparathyroidism Improves With rhPTH(1-84) Throughout 8 Years of Therapy.

Calcium and vitamin D treatment does not improve reduced quality of life (QOL) in hypoparathyroidism. Recombinant human (rh) PTH(1-84) therapy improves QOL metrics for up to 5 years. Data on QOL beyond this time point are not available. To evaluate the effects of 8 years of rhPTH(1-84) therapy on QOL and factors associated with long-term benefit. Prospective, open-label trial. Referral center. Twenty patients with hypoparathyoidism. RAND 36-Item Short Form Health Survey (SF-36). rhPTH therapy led to substantial improvement in five of the eight SF-36 domains [vitality, social functioning (SF), mental health (MH), bodily pain (BP) and general health] and three of these domains (SF, MH, BP) were no longer lower than the reference population. The improvement in the mental component summary (MCS) score was sustained through 8 years, while the physical component summary (PCS) score improved through 6 years. A lower baseline QOL score was associated with greater improvement. A threshold value <238 (MCS) and <245 (PCS) predicted long-term improvement in 90% and 100% of the cohort, respectively. In patients whose calcium supplementation was reduced, MCS and PCS scores improved more than those whose supplementation did not decline to the same extent. Improvement in PCS was greater in patients whose calcitriol dosage was reduced and duration of disease was shorter. rhPTH(1-84) improves long-term well-being in hypoparathyroidism. The improvements are most prominent in those with impaired SF-36 at baseline and those whose requirements for conventional therapy decreased substantially.