Improvements In Health-related Quality Of Life Measures Research Articles

Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT)

There are substantial disease and health-related quality-of-life (HRQoL) burdens for many patients with myasthenia gravis (MG), especially for those whose disease symptoms are not well controlled. HRQoL measures such as the Myasthenia Gravis Quality of Life 15-item revised (MG-QOL15r) and EuroQoL 5-Dimensions 5-Levels (EQ-5D-5L) are vital for evaluating the clinical benefit of therapeutic interventions in patients with MG, as they assess the burden of disease and the effectiveness of treatment, as perceived by patients. The phase 3 ADAPT study (NCT03669588) demonstrated that efgartigimod—a novel neonatal Fc receptor inhibitor—was well tolerated and that acetylcholine receptor antibody–positive (AChR-Ab+) participants who received efgartigimod had statistically significant improvements in MG-specific clinical scale scores. The ancillary data reported here, which cover an additional treatment cycle, show that these participants had similar significant improvements in HRQoL measures, the MG-QOL15r and EQ-5D-5L utility and visual analog scales, and that these improvements were maintained in the second treatment cycle. Positive effects on HRQoL were rapid, seen as early as the first week of treatment in both treatment cycles, and maintained for up to 4 weeks in the follow-up–only portion of treatment cycles. The pattern of improvements in HRQoL paralleled changes in immunoglobulin G level, and correlational analyses show that improvements were consistent across HRQoL measures and with clinical efficacy measures in the ADAPT study. The substantial and durable improvements in HRQoL end points in this study demonstrate the broader benefit of treatment with efgartigimod beyond relief of immediate signs and symptoms of gMG.

Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood

Omidubicel is an advanced cell therapy derived from umbilical cord blood (UCB) for use in allogeneic hematopoietic cell transplantation (HCT). A recent randomized phase 3 clinical trial demonstrated faster engraftment, shorter length of hospital stays, and lower rates of infection with omidubicel compared with standard UCB transplantation in patients with high-risk hematologic malignancies. Despite the proven clinical benefits of omidubicel, its impact on health-related quality of life (HRQL) from the patient's perspective has not been described. This study analyzed patient-reported HRQL measures collected prospectively in the randomized phase 3 trial comparing omidubicel to standard UCB transplantation. A total of 108 patients at 33 international stem cell transplantation centers underwent myeloablative allogeneic HCT with either omidubicel or standard UCB. Patients completed serial HRQL questionnaires at screening and on days 42, 100, 180, and 365 post-transplantation. The HRQL surveys included the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT), a 50-item cancer-specific questionnaire assessing physical, functional, emotional, social/family, and HCT-specific well-being, and the EuroQol 5-Dimension 3-Level, a 5-item generic HRQL survey. A mixed model with repeated measures was used to compare changes in HRQL from baseline in the 2 treatment arms. The average change in HRQL scores over time was compared by estimating the difference in the area under the curve (AUC) in each treatment group. Seventy-five patients (omidubicel arm, n=37; standard UCB arm, n=38) who completed the FACT-BMT at baseline and on 1 or more follow-up visits were included in this study. Baseline characteristics were similar in the 2 treatment arms. Over the first year post-transplantation, the AUCs of mean changes in physical, functional, and total FACT-BMT scores indicated significantly better HRQL with omidubicel (P < .05), with mean differences across time points ranging from 1.4 to 3.1 points, 1.6 to 3.2 points, and 7.2 to 11.0 points, respectively. The minimal clinically important difference was exceeded at 1 or more time points for each of these measures. The HRQL improvements with omidubicel were observed as early as 42 days post-transplantation and persisted at 1 year, indicating the potential long-term benefits of omidubicel on HRQL. Across all patients, adverse clinical outcomes, such as grade 3 viral infections and lower rates of neutrophil engraftment, were associated with worse HRQL scores. The observed improvements in HRQL measures may reflect the known clinical benefits of omidubicel. Compared with standard UCB, allogeneic HCT with omidubicel resulted in significant and clinically meaningful improvements in patient-reported HRQL measures.

Ustekinumab Is Associated with Real-World Long-Term Effectiveness and Improved Health-Related Quality of Life in Crohn's Disease

BackgroundProspectively and systematically collected long-term real-world clinical data on ustekinumab (anti-interleukin-12/23) are still scarce.AimsTo assess the long-term effectiveness of ustekinumab in patients with active Crohn’s disease (CD).MethodsThis is a prospective multicenter study of adult patients with CD initiating ustekinumab according to recommended doses at 20 Swedish hospitals. The primary outcome was clinical remission (Harvey–Bradshaw Index (HBI) ≤ 4 points) at weeks 52 and 104. Secondary outcomes included clinical response (≥ 3-point-decrease in HBI among patients with initial HBI ≥ 5 points), treatment retention, and biomarkers (C-reactive protein (CRP), hemoglobin, fecal-calprotectin) at weeks 52 and 104 compared to baseline. We also reported Health-related Quality of Life (HRQoL) measures.ResultsOf 114 included patients, 107 (94%) had previously failed ≥ 1 and 58 (51%) ≥ 2 anti-tumor necrosis factor agents. Forty (35%) had failed anti-integrin agents. Ustekinumab retention rates at weeks 52 and 104 were 70% (n = 80/114) and 61% (n = 69/114), respectively. Clinical response was seen in 36% (n = 25/69) and 29% (n = 20/69) of the patients, and remission was achieved in 32% (n = 31/96) and 29% (n = 28/96) at weeks 52 and 104, respectively. Median HBI and CRP levels decreased significantly at both timepoints as compared to baseline. Significant improvements were also observed in HRQoL. Adverse events were reported in 11% (n = 13/114) of the patients, including five cases of severe adverse events. No malignancies were observed.ConclusionsIn this nationwide prospective real-world 104-week-follow-up study of adult patients with active CD, ustekinumab was associated with long-term clinical effectiveness and improvement in HRQoL measures when used in routine clinical care.

Impact of biologics on health-related quality of life in patients with Ankylosing spondylitis: A systematic review and meta-analysis of randomized controlled trials

ObjectiveTo examine the impact of novel biologic disease-modifying antirheumatic drugs (bDMARDs) with tumor necrosis factor (TNF) inhibitors or Interleukin-17A (IL-17A) antibody agents on health-related quality of life (HRQoL) outcomes in patients with radiographic axial spondyloarthritis (r-axSpA). MethodsA literature search on PubMed, Embase, and Clinicaltrials.gov databases was performed through February 2022 to identify randomized controlled trials (RCTs) reporting HRQoL outcomes of bDMARDs for treatment of r-axSpA. Approved bDMARD therapy for r-axSpA included five TNF-inhibitors (infliximab, etanercept, adalimumab, golimumab, and certolizumab pegol) and two IL-17A antibody agents (secukinumab, and ixekizumab). Heterogeneity (I2) was used to determine the variability between studies. ResultsSixteen RCTs, involving 3481 participants, were included. The placebo-controlled and treatment blinded durations ranged from 12 to 24 weeks. Compared with the placebo, bDMARD therapy was associated with significant improvement in HRQoL measures with the 36-item Short Form Survey (SF-36), European Quality of Life–5 Dimensions (EQ-5D), and Ankylosing Spondylitis Quality of Life (ASQoL). The pooled mean differences of changes from baseline for the SF-36 Physical Component Score (PCS), SF-36 Mental Component Score (MCS), EQ-5D, and ASQoL were 4.39 [95% Confidence Interval (CI): 3.24 to 5.54, P < 0.001]; 2.37 (95%-CI: 1.25 to 3.49, P = 0.003); 0.11 (95%-CI: 0.07 to 0.14, P < 0.001); and -2.45 (95%-CI: -3.21 to -1.70, P < 0.001), respectively. Heterogeneity was high (I2 = 79%) among studies reporting SF-36 PCS, and moderate (I2 = 61%, 34%, and 49%) among studies reporting SF-36 MCS, EQ-5D, and ASQoL, respectively. ConclusionsBiologic therapy was associated with a significant improvement in quality of life in patients with r-axSpA. The results were consistent among all three measures of HRQoL outcomes.

Changes in Quality-of-Life Parameters in Patients Receiving Therapeutic Interventions for Cancer-Associated Anemia: A Systematic Review

Introduction: Cancer-associated anemia (CAA) is multifactorial in etiology and can occur due to the malignancy itself or as a side effect of the treatment. The prevalence of CAA can exceed 90%. Several studies have reported positive correlations between increased hemoglobin (Hb) and improved health-related quality of life (HR-QoL) measures including fatigue scores and scores for physical, mental and social well-being. However, Hb increases and extent of improvement in HR-QoL measures varied by therapeutic interventions (Oral/intravenous iron, erythropoietin stimulating agents (ESAs), transfusions, and/or others), as did HR-QoL tools used in these studies. We conducted a systematic review to identify final target Hb levels as well as changes in Hb from baseline that resulted in significant improvement in HR-QoL parameters following interventions for CAA. We also sought to identify HR-QoL tools that effectively and comprehensively captured symptomatic improvement following Hb increases in patients with CAA.Methods: Randomized Controlled Trials (RCTs) and prospective studies which evaluated HR-QoL changes following drug interventions for CAA were identified through searches of the CINAHL Plus, Embase, PubMed, and Web of Science for articles in English from 1997-2021. Inclusion criteria were: RCT or prospective study; patients with CAA; a HR-QoL was used. Two reviewers independently screened abstracts. A third reviewer resolved disagreements. Bias assessment was performed using Joanna Briggs checklist.Results: Of the 8243 studies identified of which 1718 were duplicates and 6525 records were screened at the title and abstract level. Of the 59 full text articles reviewed, 38 articles met our eligibility criteria (Figure 1). A total of 19 RCTs and 19 prospective studies were identified. On average, studies were conducted over 14.2 ± 5.5 weeks, with a mean of 546 ± 693 study participants in each study. 23 studies provided information on the number of enrolled participants completing study. Mean study completion rates among participants was 70 ± 1 (%). A summary of HR-QoL measures utilized in the various studies is presented in Table 1.Hb changes and HR-QoL score changes were reported consistently in 30 studies and are presented in Table 2. Therapeutic interventions for CAA included ESAs (n=19; 65.5%), Oral/IV iron (n=1; 3.4%); transfusions (n=2; 6.9%), other agents used in specific conditions (luspatercept (MDS)) n=1; 3.3%, or combinations of these (n=7; 24.1%). Overall, 78% of studies that reported an Hb increase following drug interventions for CAA also reported an improvement in HR-QoL measures. Mean Hb increases was 1.9 g/dL. Median change in the most frequently used HR-QoL (FACT-An, FACT-F) was +3 points (Range: 0-4).Studies utilizing transfusions as the intervention for CAA reported the lowest final Hb (8.8 gm/dL) as well as the lowest change in Hb (Δ Hb= 1gm/dL) levels from study onset to completion. These studies also reported the least improvements in HR-QoL scores. HR-QoL tools used in transfusion-based studies were variable. With regard to other outcomes measured in the various studies, raising Hb levels by various interventions did not significantly impact bleeding scores, infections, hospitalizations, or mortality.Conclusions: Our data suggest the need to identify optimum Hb thresholds (final target Hb levels as well as changes in Hb levels from baseline) that result in meaningful improvements in HR-QoL parameters following the use of any therapeutic intervention for CAA. Specifically, studies utilizing transfusions alone as intervention for CAA may not reach the optimum threshold (>11gm/dL or a Δ Hb of greater than 2 gm/dL) to result in meaningful improvements in HR-QoL measures as has been shown in studies utilizing ESAs or iron or a combination of these agents. Further, it is imperative for studies in transfusion medicine to identify appropriate HR-QoL tool/s that comprehensively capture all aspects of well-being related to CAA. As such, studies may need to utilize more than one HR-QoL tool that gathers information on physical, social and emotional well-being before and after transfusions, while balancing the risk of questionnaire fatigue. A meta-analysis of our systematic review is ongoing and will provide additional information on data gathered thus far. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Impact of preoperative weakness and duration of symptoms on health-related quality-of-life outcomes following anterior cervical discectomy and fusion

BACKGROUND CONTEXTThe majority of patients with preoperative upper extremity weakness show improvements in motor function after anterior cervical discectomy and fusion (ACDF). Although numerous studies have examined both the extent and time course to which motor function can be expected to improve, few have shown that these improvements in motor function translate to improved health related quality of life (HRQOL) outcomes. PURPOSEThe purpose of this study was to examine the effect of preoperative weakness and duration of symptoms on HRQOL outcomes in patients who underwent ACDF. STUDY DESIGNRetrospective cohort study. PATIENT SAMPLEAdult patients who underwent an ACDF procedure at an academic hospital from January 2015 to December 2016 by a fellowship-trained spine surgeon. OUTCOME MEASURESThe primary outcomes of interest were HRQOL outcomes: Short Form-12 Physical/Mental Component Scores (SF-12 PCS/MCS), Neck Disability Index (NDI), and Visual Analog Scale Arm/Neck scores (VAS Arm/Neck). METHODSPatient demographics, surgical case characteristics, and motor strength exams were collected in patients who underwent ACDF over a 2-year period. Preoperative weakness was defined by a strength grade ≤3 (on a scale from 0 to 5) in at least one upper extremity muscle group. Multivariate linear regression analysis was performed to determine the effect of the preoperative weakness on HRQOL outcomes. RESULTSOf the 276 patients identified, 45 (16.3%) showed evidence of preoperative weakness, 44 (97.8%) of which showed subsequent postoperative motor improvements after ACDF. All patients reported significant improvements in all HRQOL outcome measures. Patients with preoperative weakness reported significantly worse preoperative VAS Arm (6.9 vs. 5.2; p=.01) and VAS Neck (6.1 vs. 4.8; p=.02) pain scores. Compared with patients without preoperative weakness, those with preoperative weakness reported significantly more improvement in NDI (β: −10.9; p=.001). Patients with symptoms greater than or equal to 12 months and preoperative weakness showed significantly less improvement in NDI (β: 14.8; p=.03). CONCLUSIONSPatients with preoperative weakness generally exhibited worse pain and HRQOL measures preoperatively, and showed greater potential for improvement after ACDF. Patients with a shorter duration of preoperative weakness had greater potential for improvement in HRQOL measures after ACDF compared with those with longer duration of symptoms. ACDF is an effective procedure to improve strength and HRQOL measures across all patient groups under appropriate indications.

Impact of pedicle subtraction osteotomy on health-related quality of life (HRQOL) measures in patients undergoing surgery for adult spinal deformity: a systematic review and meta-analysis.

Systematic review and meta-analysis. Three-column osteotomies (3-CO) have gained popularity in the last decade as part of the armamentarium for the surgical correction of sagittal imbalance in patients with adult spinal deformity (ASD). Three-column osteotomies in the form of pedicle subtraction osteotomy (PSO) may be necessary to achieve adequate correction for severe and rigid spinal deformity. Studies reporting improvement in health-related quality of life (HRQOL) with validated outcome measures after PSO surgery are sparse and currently consist of small series. Evaluate the improvement in HRQOL measures following PSO for adult spinal deformity. Two independent reviewers conducted a systematic review of the English literature between period 1996 and 2019 for articles reporting outcome of PSO in patients with ASD according to PRISMA (Preferred Reporting Items for Systematic reviews and Meta-Analyses) guidelines. Inclusion criteria were studies consisting of patient-reported outcome Oswestry Disability Index (ODI) and Scoliosis Research Society 22 or 24 (SRS) outcomes after PSO surgery for adult spine deformity patients (18years or older) with a minimum follow-up of 1year. Eight studies with 431 PSOs were included in the meta-analysis. The results showed a statistically significant improvement in ODI in PSO (P < 0.0001), and the mean clinically important difference was achieved with both ODI (50.46 (45.5-55.4) preoperatively to 32.78 (29.7-39) postoperatively) and SRS (2.49 (2.38-2.7) preoperatively to 3.26 (2.8-4.1) postoperatively) scores. This meta-analysis did find improvements in the health-related quality of life in patients undergoing PSO surgery for adult spinal deformity.

Surgical correction of severe adult lumbar scoliosis (major curves ≥ 75°): retrospective analysis with minimum 2-year follow-up.

Prior reports have demonstrated the efficacy of surgical correction for adult lumbar scoliosis. Many of these reports focused on mild to moderate scoliosis. The authors' objective was to report their experience and to assess outcomes and complications after deformity correction for severe adult scoliosis. The authors retrospectively analyzed consecutive adult scoliosis patients with major thoracolumbar/lumbar (TL/L) curves ≥ 75° who underwent deformity correction at their institution. Those eligible with a minimum 2 years of follow-up were included. Demographic, surgical, coronal and sagittal plane radiographic measurements, and health-related quality of life (HRQL) scores were analyzed. Among 26 potentially eligible patients, 22 (85%) had a minimum 2 years of follow-up (range 24-89 months) and were included in the study (mean age 57 ± 11 years; 91% women). The cohort comprised 16 (73%), 4 (18%), and 2 (9%) patients with adult idiopathic scoliosis, de novo degenerative scoliosis, and iatrogenic scoliosis, respectively. The surgical approach was posterior-only and multistage anterior-posterior in 18 (82%) and 4 (18%) patients, respectively. Three-column osteotomy was performed in 5 (23%) patients. Transforaminal and anterior lumbar interbody fusion were performed in 14 (64%) and 4 (18%) patients, respectively. All patients had sacropelvic fixation with uppermost instrumented vertebra in the lower thoracic spine (46% [10/22]) versus upper thoracic spine (55% [12/22]). The mean fusion length was 14 ± 3 levels. Preoperative major TL/L and lumbosacral fractional (L4-S1) curves were corrected from 83° ± 8° to 28° ± 13° (p < 0.001) and 34° ± 8° to 13° ± 6° (p < 0.001), respectively. Global coronal and sagittal balance significantly improved from 5 ± 4 cm to 1 ± 1 cm (p = 0.001) and 9 ± 8 cm to 2 ± 3 cm (p < 0.001), respectively. Pelvic tilt significantly improved from 33° ± 9° to 23° ± 10° (p < 0.001). Significant improvement in HRQL measures included the following: Scoliosis Research Society (SRS) pain score (p = 0.009), SRS appearance score (p = 0.004), and SF-12/SF-36 physical component summary (PCS) score (p = 0.026). Transient and persistent neurological deficits occurred in 8 (36%) and 2 (9%) patients, respectively. Rod fracture/pseudarthrosis occurred in 6 (27%) patients (supplemental rods were utilized more recently in 23%). Revisions were performed in 7 (32%) patients. In this single-center surgical series for severe adult scoliosis (major curves ≥ 75°), a posterior-only or multistage anterior-posterior approach provided major curve correction of 66% and significant improvements in global coronal and sagittal spinopelvic alignment. Significant improvements were also demonstrated in HRQL measures (SRS pain, SRS appearance, and SF-12/SF-36 PCS). Complications and revisions were comparable to those of other reports involving less severe scoliosis. The results of this study warrant future prospective multicenter studies to further delineate outcomes and complication risks for severe adult scoliosis correction.

Cultural Dance Program Improves Hypertension Management for Native Hawaiians and Pacific Islanders: a Pilot Randomized Trial

ObjectiveNative Hawaiians and Pacific Islanders (NHPI) bear an unequal burden of hypertension and cardiovascular disease. Hula, the traditional dance of Hawaii, has shown to be a culturally meaningful form of moderate-vigorous physical activity for NHPI. A pilot study was done in Honolulu, Hawaii, to test a 12-week hula-based intervention, coupled with self-care education, on blood pressure management in NHPI with hypertension in 2013.MethodNHPI with a systolic blood pressure (SBP) ≥140 mmHg were randomized to the intervention (n = 27) or a wait-list control (n = 28). Blood pressure, physical functioning, and eight aspects of health-related quality of life (HRQL) were assessed.ResultsThe intervention resulted in a reduction in SBP compared to control (−18.3 vs. −7.6 mmHg, respectively, p ≤ 0.05) from baseline to 3-month post-intervention. Improvements in HRQL measures of bodily pain and social functioning were significantly associated with SBP improvements in both groups.ConclusionUsing hula as the physical activity component of a hypertension intervention can serve as a culturally congruent strategy to blood pressure management in NHPI with hypertension.Trial registrationclinicaltrials.gov Identifier: NCT01995812

Eculizumab (ECU) Significantly Improves Health-Related Quality of Life (HRQoL) in Patients with Atypical Hemolytic Uremic Syndrome (aHUS)

Abstract 4772 BackgroundaHUS is a rare, genetic, life-threatening, disease caused by chronic uncontrolled complement activation, which leads to systemic thrombotic microangiopathy (TMA). Despite plasma exchange/infusion (PE/PI), >50% of patients (pts) will either die, require kidney dialysis or have permanent renal damage within the first year of diagnosis. In addition to causing progressive organ damage, the uncontrolled complement activation and subsequent TMA might be expected to impact HRQoL. In 2 prospective, controlled, single-arm phase II trials of aHUS pts receiving ECU, a terminal complement inhibitor, TMA was stopped, there was no longer a need for PE/PI or new dialysis. In one of the trials (C08-002), dramatically, 4/5 pts on dialysis permanently discontinued dialysis as of most recent follow-up. The current analysis evaluates the impact of ECU on HRQoL in aHUS pts. MethodsIn trial C08-002, 17 pts with aHUS resistant to PE/PI (persistent TMA despite ≥4 PE/PI sessions 1 wk before screening) received ECU. In trial C08-003, 20 pts previously received chronic PE/PI (≥1 every 2 wks and <3 per wk for ≥8 wks prior to ECU). Clinical data, including pt-reported outcomes, were collected at baseline and through the 26-wk treatment and ongoing extension studies up to data cut-off (mean [SD] duration of ECU treatment: C08-002=58 [29] wks and C08-003=60 [12] wks). HRQoL was measured with the EQ-5D self-reporting questionnaire and a 20-cm visual analogue scale (VAS) validated for a number of disease populations. EQ-5D has 5 dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression) and 3 levels (no problem, some problems and extreme problems). Change in HRQoL was calculated using the US time trade-off (TTO) index and VAS valuation techniques, and ANOVA analysis. A change of >0.06 is considered a clinically meaningful change ResultsC08-002: 15/17 pts received ECU until Wk 26 (2 discontinued at Wks 1 and 6; protocol violation and an adverse event unrelated to ECU, respectively) and 13 continued into the extension study. Median age=28 yrs and 29% were on dialysis immediately prior to ECU. Median time from diagnosis to screening =10 mo. All patients had eGFR ≤60 mL/min/1.73m2.C08-003: 20 pts received ECU for 26 wks and 19 continued into the extension study. Median age=28 yrs. Median time from diagnosis to screening=48 mo. Median duration of eGFR ≤60 mL/min/1.73m2=180 days at baseline.HRQoL: In both cohorts of distinct trial pts, ECU substantially improved HRQoL through Wk 26, and sustained this thereafter during long-term ECU therapy (table). In trial C08-002, improvements were seen as early as Day 7 (0.12, p=0.0346). Point estimate improvement (95%CI) in HRQoL from baseline to1 yr was 0.32 (0.27-0.36) for C08-002 and 0.09 (0.05-0.14) for C08-003. On an individual pt basis, 87% and 73% of C08-002 and C08-003 pts, respectively, achieved a clinically meaningful change that represented a clinically important large or moderate improvement in HRQoL measures, respectively. ConclusionsaHUS pts have an impaired HRQoL both from a direct impact of aHUS morbidity and management requirements (such as PE/PI), but also from the systemic nature of the disease. In patients with aHUS, significant improvements in HRQoL were achieved rapidly with ECU (as early as Day 7). Sustained long-term treatment with ECU maintained the improvements in HRQoL.C08-002 trialC08-003 trialECU Treatment (BL Through 1 yr)ECU Treatment (BL Through Wk 26)ECU Treatment (BL Through 1 yr)ECU Treatment (BL Through Wk 26)Point estimate change (95% CI) in EQ-5D from BL (using US TTO index scores)0.33 (0.28–0.37) (n=12) (p>0.0001)0.32 (0.27–0.36) (n=10) (p>0.0001)0.12 (0.07–0.17) (n=17) (p>0.0001)0.09 (0.05–0.14) (n=15) (p=0.0001)Mean (SD) change in VAS from BL31.9 (23) (n=12) (p=0.0010)35.5 (17) (n=10) (p=0.002)10.2 (17) (n=17) (p=0.02)9.1 (15) (n=15) (p=0.03)Percentage of pts with a clinically meaningful benefit in the US TTO value80%*87%*73%*73%*Effect Sizes† for EQ-5D (response is change in the index using the US TTO value)1.11.00.60.4BL=baseline; TTO=time trade off*15 pts were evaluable for C08-002 and 11 for C08-003†Effect sizes were considered large for C08-002 and medium for C08-003 Disclosures:Muus:Alexion: Honoraria, Research Funding. Off Label Use: The research described is a clinical trial of eculizumab in patients with aHUS, an indication in which its use is not approved. Licht:Alexion: Honoraria, Research Funding. Goodship:Alexion: Honoraria, Research Funding. Greenbaum:Alexion: Honoraria, Research Funding. Bedrosian:Alexion: Employment. Loirat:Alexion: Honoraria, Research Funding. Legendre:Alexion: Honoraria, Research Funding.

Diferencias en la calidad de vida en pacientes con flutter auricular típico sometidos a ablación del istmo cavotricuspídeo

Introduction and objectives The aim of the study was to assess and measure health-related quality of life (HRQoL) changes in patients with typical atrial flutter following catheter ablation. The outcome was standardized and normalized to the Spanish population adjusted by age and sex. Methods Ninety-five consecutive patients who had undergone cavotricuspid isthmus ablation were included. The SF-36 questionnaire was self-administered before the procedure and at 1-year follow-up. We used the effect size and the standardized response mean as measures of responsiveness to quantify the change in HRQoL and the minimum clinically important difference to assess the smallest difference in score that patients perceived as beneficial. Results Of the 95 patients initially included, 88 completed the 1-year follow-up. We observed a large improvement (effect size ≥0.8) on the physical functioning, role-physical , general health, and vitality scales and on the physical component summary. We detected a moderate improvement (effect size ≥0.5) on the role-emotional, social functioning, and mental health scales and on the mental component summary. On all scales except bodily pain and social activity, the improvement was clinically perceived by patients. Conclusions A clinically significant improvement in HRQoL measures was found in patients with typical atrial flutter who underwent cavotricuspid isthmus catheter ablation. Full English text available from: www.revespcardiol.org

Quality of Life Differences in Patients With Typical Atrial Flutter Following Cavotricuspid Isthmus Ablation

Introduction and objectives The aim of the study was to assess and measure health-related quality of life (HRQoL) changes in patients with typical atrial flutter following catheter ablation. The outcome was standardized and normalized to the Spanish population adjusted by age and sex. Methods Ninety-five consecutive patients who had undergone cavotricuspid isthmus ablation were included. The SF-36 questionnaire was self-administered before the procedure and at 1-year follow-up. We used the effect size and the standardized response mean as measures of responsiveness to quantify the change in HRQoL and the minimum clinically important difference to assess the smallest difference in score that patients perceived as beneficial. Results Of the 95 patients initially included, 88 completed the 1-year follow-up. We observed a large improvement (effect size ≥0.8) on the physical functioning, role-physical, general health, and vitality scales and on the physical component summary. We detected a moderate improvement (effect size ≥0.5) on the role-emotional, social functioning, and mental health scales and on the mental component summary. On all scales except bodily pain and social activity, the improvement was clinically perceived by patients. Conclusions A clinically significant improvement in HRQoL measures was found in patients with typical atrial flutter who underwent cavotricuspid isthmus catheter ablation.

Health-related quality of life and subjective neurocognitive function three months after coronary artery bypass graft surgery

We compared health-related quality of life (HRQL), including patient-perceived neurocognitive function at preoperative baseline and 3 months after coronary artery bypass (CABG) surgery. The design was prospective and comparative. The study took place in the cardiovascular units at two large metropolitan Midwestern hospitals. The study included a consecutive convenience sample of 64 patients who underwent CABG. Preoperative baseline and mailed survey at 3 months post-CABG included the Short-Form 12 (subjective health-status), State Trait Anxiety Inventory (state anxiety), Center for Epidemiological Studies of Depression (depressive symptoms), and Cantril Ladder of Life Satisfaction (global life satisfaction). Investigator-developed questions assessed satisfaction in life domains, cardiac symptoms, and frequency of symptoms related to neurocognitive function. Significant improvements were demonstrated 3 months postoperatively, including the Physical Component Summary, Mental Component Summary, depression, anxiety, satisfaction with social and mental life domains, and patient-perceived neurocognitive function related to memory and concentration. Patients reported improvements in HRQL measures, including two of three subjective neurocognitive measures. Health care providers facilitate preparation for the CABG recovery trajectory by discussing expected post-hospital experience and potential postoperative variations in emotions and neurocognitive function.

Inhaled Mometasone Furoate Improves Health-Related Quality of Life in Patients with Persistent Asthma

Results from two clinical trials of mometasone furoate administered via a dry powder inhaler (MF-DPI) were reviewed to evaluate the consistency of effects of MF-DPI administered once-daily in the evening (QD PM) or twice-daily (BID) on health-related quality of life (HRQOL) in adults with persistent asthma previously treated with inhaled corticosteroids.HRQOL data were collected from two 12-week, randomized, double-blind trials: in study 1 (n = 268), patients received MF-DPI 400 μ g QD PM (1 inhalation), MF-DPI 200 μ g BID, or placebo; in study 2 (n = 400), patients received MF-DPI 200 μ g QD PM, MF-DPI 400 μ g QD PM (1 inhalation), MF-DPI 200 μ g BID, MF-DPI 400 μ g QD PM (2 inhalations of 200 μ g), or placebo. In both studies, HRQOL was assessed using the Medical Outcomes Survey 36-item Short Form (SF-36) and an asthma-specific module.MF-DPI was associated with consistent, statistically significant improvements in asthma-specific total scores, breathlessness, asthma concerns, and physical symptoms compared with placebo in both trials (p < 0.05 vs. placebo). MF-DPI improved SF-36 Physical Component Summary scores at all doses except 200 μ g QD PM. In conclusion, the results from two placebo-controlled trials suggest that MF-DPI 400 μ g/d, administered once or twice-daily, produces consistent, statistically, and clinically significant improvement in HRQOL measures in patients with persistent asthma.

Pregabalin reduces pain and improves sleep and mood disturbances in patients with post-herpetic neuralgia: results of a randomised, placebo-controlled clinical trial

This study was designed to assess the efficacy and safety of pregabalin—a novel α 2-δ ligand with analgesic, anxiolytic, and anticonvulsant activity—for treating neuropathic pain in patients with post-herpetic neuralgia (PHN). Two hundred and thirty-eight patients were randomised into this multicentre, doubleblind, placebo-controlled trial to receive 150 ( n=81), 300 mg/day ( n=76) pregabalin, or placebo ( n=81) for 8 weeks. Among the exclusion criteria was failure to respond to previous treatment for PHN with gabapentin at doses ≥1200 mg/day. Endpoint mean pain scores were significantly reduced in patients receiving 150 or 300 mg/day pregabalin compared with placebo. Efficacy was observed as early as week 1 and was maintained throughout the study. Significantly more patients in both pregabalin groups (150 mg, 26%; 300 mg, 28%) were responders (≥50% decrease in mean pain score from baseline to endpoint) than in the placebo group (10%). Additionally, by week 1 and for the study's duration, 150 and 300 mg/day pregabalin significantly reduced weekly mean sleep interference scores. More pregabalin-treated patients than placebo-treated patients reported that they were ‘much improved’ or ‘very much improved’. Health-related quality-of-life (HRQoL) measurements using the SF-36 Health Survey demonstrated improvement in the mental health domain for both pregabalin dosages, and bodily pain and vitality domains were improved in the 300 mg/day group. The most frequent adverse events were dizziness, somnolence, peripheral oedema, headache, and dry mouth. Pregabalin efficaciously treated the neuropathic pain of PHN. Additionally, pregabalin was associated with decreased sleep interference and significant improvements in HRQoL measures.