Meaningful Improvements In Health-related Quality Of Life Research Articles

A multicenter randomized controlled feasibility trial of a digital self-management intervention for adults with epilepsy.

Self-management interventions may enhance health-related quality of life (HRQoL) in epilepsy. However, several barriers often impair their implementation in the real world. Digital interventions may help to overcome some of these barriers. Considering this, the Helpilepsy Plus Prototype was developed as a prototype smartphone-delivered self-care treatment program for adults with epilepsy. The 12-week Helpilepsy Plus Prototype was evaluated through a randomized controlled feasibility trial with a waiting-list control (WLC) group. Outcome measurement at baseline and at 12 weeks assessed adherence to the prototype intervention and changes in epilepsy-related outcomes. The primary endpoint was patient autonomy measured with EASE, and secondary endpoints included HRQoL measured with QOLIE-31, health literacy measured with HLQ, anxiety, and depression symptoms measured with HADS. Semi-structured interviews were conducted with a heterogeneous sample of participants to assess user-friendliness and usefulness. The prototype program was delivered through the Neuroventis Platform (Neuroventis, BV, Overijse, Belgium), a certified medical device (under EU/MDD Class I, and EU/MDR grace period). Ninety-two patients were included (46 in the intervention group, 46 in WLC). Most participants (63%, 58/92 women, median age 30 years) had pharmacoresistant epilepsy (61%, 56/92). Only 22% of participants (10/46) in the intervention group completed at least half of all intervention sessions. No significant differences between the intervention group and WLC were observed. Although there was a larger proportion of patients in the intervention group with meaningful improvements in HRQoL compared to WLC (19/46 versus 11/46), the difference was not significant (p = 0.119). Qualitative feedback showed that participants would appreciate more personalization, such as adaptation of the content to their current epilepsy knowledge level, a more interactive interface, shorter text sections, and interaction through reminders and notifications. Digital interventions should allow sufficient scope for personalization and interaction to increase patient engagement and enable benefits from self-care apps. Feedback loops allow the participatory development of tailored interventions. In this study, we investigated the effectiveness of an app-based self-help intervention. Study participants were either randomly assigned to a group that had access to the app or a group that received access to the app after the end of the study. Although a larger proportion of participants in the intervention group showed a relevant improvement in quality of life, the difference between the two groups was not statistically significant. Less than one-fifth of participants in the intervention group attended at least half of all intervention sessions; patient feedback showed that patients required more personalization and interactive options.

The changes in health-related quality of life in stable coronary artery disease patients and intermediante coronary lesions after elective percutaneous coronary intervention

The aim – to analyze the factors, associated with the change in health-related quality of life (HRQoL) in stable coronary artery disease (SCAD) patients with intermediate coronary lesions at 3-months follow-up after elective percutaneous coronary intervention (PCI).Materials and methods. A prospective single-center study enrolled 84 SCAD patients (mean age 64±8 years; males – 73 %) with intermediate coronary lesions (stenosis 50-90 %) (by invasive coronary angiography [ICA]), consecutively enrolled and hospitalized with the aim of elective PCI to be performed. HRQoL was evaluated by SF-36 (physical health summary [PHsum] and mental health summary [МНsum]) and SAQ (Seattle Angina Questionnaire»; the domains «physical limitation» [PL], «angina stability» [AS], «angina frequency» [AF], «treatment satisfaction» [TS] and «disease perception» [DP]), – at baseline and 3-months follow-up. The enrolled sample was subdivided in a dichotomous manner into the HRQoL «improvement» or «non-improvement» patterns, based on the cut-off-values (COVs) of clinically meaningful change of the certain HRQoL parameter score at 3-months follow-up after PCI.Results and discussion. We revealed an improvement of average (median) HRQoL parameters at 3-months follow-up after PCI, namely PHsum (score increase from 38.8 to 43.1; р < 0.001), SAQ-AS (25 to 75; р < 0.001), SAQ-AF (70 to 80; р < 0.001), SAQ-TS (58.8 to 70.6; р < 0.001) and SAQ-DP (45.8 to 66.7; р < 0.001). The clinically meaningful HRQoL improvement ranged from 14 % to 71 % cases, depending on the certain HRQoL parameter and the COVs of its change at follow-up after PCI. The worse baseline HRQoL, older age and higher baseline total cholesterol level were the factors, associated with the increased probability of HRQoL to be meaningfully improved after revascularization. In addition, a presence of diabetes mellitus, a history of acute cerebrovascular accident, and the ICA data on multivessel disease and in-stent restenosis after previous PCI, – all were associated with the lower odds of HRQoL «improvement» status to be achieved.Conclusions. A PCI favored the HRQoL improvement in SCAD patients at 3-months follow-up. The clinical and angiographic factors, associated with the post-procedure change in HRQoL, should be considered for a better selection of SCAD patients with intermediate coronary lesions for PCI.

Quality of Life in Patients With Chronic Limb-Threatening Ischemia Treated With Revascularization.

In the BEST-CLI trial (Best Endovascular Versus Best Surgical Therapy for Patients With Chronic Limb-Threatening Ischemia), a prespecified secondary objective was to assess the effects of revascularization strategy on health-related quality of life (HRQoL). Patients with chronic limb-threatening ischemia were randomized to surgical bypass (Bypass) or endovascular intervention (Endo) in 2 parallel trials. Cohort 1 included patients with single-segment great saphenous vein; cohort 2 included those lacking suitable single-segment great saphenous vein. HRQoL was assessed over the trial duration using Vascular Quality-of-Life (VascuQoL), European Quality-of-Life-5D (EQ-5D), the Short Form-12 (SF-12) Physical Component Summary (SF-12 PCS), SF-12 Mental Component Summary (SF-12 MCS), Utility Index Score (SF-6D R2), and numeric rating scales of pain. HRQoL was summarized by cohort and compared within and between groups using mixed-model linear regression. A total of 1193 and 335 patients in cohorts 1 and 2 with a mean follow-up of 2.9 and 2.0 years, respectively, were analyzed. In cohort 1, HRQoL significantly improved from baseline to follow-up for both groups across all measures. For example, mean (SD) VascuQoL scores were 3.0 (1.3) and 3.0 (1.2) for Bypass and Endo at baseline and 4.7 (1.4) and 4.8 (1.5) over follow-up. There were significant group differences favoring Endo when assessed with VascuQoL (difference, -0.14 [95% CI, -0.25 to -0.02]; P=0.02), SF-12 MCS (difference, -1.03 [95% CI, -1.89 to -0.18]; P=0.02), SF-6D R2 (difference, -0.01 [95% CI, -0.02 to -0.001]; P=0.03), numeric rating scale pain at present (difference, 0.26 [95% CI, 0.03 to 0.49]; P=0.03), usual level during previous week (difference, 0.26 [95% CI, 0.04 to 0.48]; P=0.02), and worst level during previous week (difference, 0.29 [95% CI, 0.02 to 0.56]; P=0.04). There was no difference between treatment arms on the basis of EQ-5D (difference, -0.01 [95% CI, -0.03 to 0.004]; P=0.12) or SF-12 PCS (difference, -0.41 [95% CI, -1.2 to 0.37]; P=0.31). In cohort 2, HRQoL also significantly improved from baseline to the end of follow-up for both groups based on all measures, but there were no differences between Bypass and Endo on any measure. Among patients with chronic limb-threatening ischemia deemed eligible for either Bypass or Endo, revascularization resulted in significant and clinically meaningful improvements in HRQoL. In patients with an available single-segment great saphenous vein for bypass, but not among those without one, Endo was statistically superior on some HRQoL measures; however, these differences were below the threshold of clinically meaningful difference.

DOP49 Guselkumab improves health-related quality of life as measured by PROMIS-29 in patients with moderately to severely active Ulcerative Colitis: Phase 3 QUASAR induction study

Abstract Background Patients (pts) with ulcerative colitis (UC) suffer from symptoms that impair health-related quality of life (HRQoL). The Phase 3 QUASAR induction study (NCT04033445) evaluated the efficacy and safety of guselkumab (GUS), an IL-23 p19 subunit antagonist, in pts with moderately to severely active UC who had demonstrated inadequate response, loss of response, or intolerance to conventional and/or advanced therapies (ie, tumor necrosis factor-alpha antagonists, integrin receptor antagonists, and/or Janus kinase inhibitors). Here we report the effect of GUS IV induction on HRQoL at Week (Wk) 12 as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS)-29. Methods Pts were randomized 3:2 to receive GUS 200 mg IV or placebo (PBO) IV at Wks 0/4/8. PROMIS-29 consists of 7 domains (anxiety, depression, fatigue, pain interference, sleep disturbance, physical function, and social participation) and a pain intensity numeric rating scale (NRS, range 0-10). The raw score of each domain was converted into a standardized T-score based on a general population mean of 50 and standard deviation (SD) of 10. Higher scores indicate better outcomes for physical function and social participation and worse outcomes for all other domains. The physical component summary (PCS) and mental component summary (MCS) scores were calculated based on the domain T-scores for physical and mental HRQoL, with higher scores representing better outcomes. Minimum clinically meaningful improvement was defined as a ≥3-point improvement in the pain intensity NRS score, a ≥5-point (one-half SD of the population) improvement in each domain, and a ≥5-point improvement in the PCS/MCS scores. More stringent thresholds (eg, ≥7- or ≥9-point improvements) were also examined and will be presented. Results Among the primary analysis population, baseline mean PROMIS-29 domain scores were similar between treatment groups, with functional domain scores <50 and symptom domain scores >50, indicating impaired HRQoL (Table). GUS-treated pts achieved a greater mean change from baseline at Wk 12 in each domain T-score, the pain intensity NRS score, and the PCS/MCS than PBO-treated pts. Additionally, the percentage of pts achieving minimum clinically meaningful improvement at Wk 12 was greater for GUS vs PBO in each domain, the pain intensity NRS score, and the PCS/MCS scores (Figure). Conclusion Pts with moderately to severely active UC treated with GUS IV induction experienced broad and clinically meaningful improvements in HRQoL as measured by the PROMIS-29 at Wk 12.

It Is Smart to Set Treatment Goals, But Are Set Treatment Goals SMART? A Qualitative Assessment of Goals Described in the Assessment of the Burden of COPD Tool

The Assessment of the Burden of COPD (ABC) tool facilitates shared decision-making and goal setting to develop a personalized care plan. In a previous trial (RCT), the ABC tool was found to have a significant effect on patients’ Health-related Quality of Life (HRQoL). In this exploratory study we used data from the intervention group of the RCT to investigate if patients with health-related goals had an improved HRQoL compared to those without goals, and if the quality and types of goals differed for those who have a clinically meaningful improvement in HRQoL. We hypothesized that the quality and the type of the goal described in the ABC tool, relates to an improved HRQoL. We assessed the quality of the goals according to the Specificity, Measurability, Achievability, Relevance and Timeliness (SMART) criteria, and coded and counted each type of goal. We found that having a goal or not, did not differ significantly for those who had a clinically meaningful improved HRQoL versus those who had not, nor was the quality or type of goal significantly different. The most common types of goals were exercise more, smoke less, and improve weight. Based on the results, we speculate that when a clinically meaningful improvement in HRQoL is achieved, it is not related to a single component (i.e. goal setting as part of shared decision-making) but that the different components of the ABC tool (visualization of burden, shared decision making, utilization of tailored evidence based interventions, and regular monitoring of progress) may have a synergistic effect on disease cognition and/or behavior change. Noteworthy, the sample size was small while the calculated effect size was moderate, making it unlikely to find a significant effect.

The Effects of Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, on Bleeding Symptoms and Health-Related Quality of Life in Patients with Immune Thrombocytopenia

Introduction: Patients with immune thrombocytopenia (ITP) have a high incidence of comorbid conditions, elevated risk for bleeding and thrombosis, and reduced health-related quality of life (HRQoL). Rilzabrutinib is a potent oral, reversible inhibitor of Bruton tyrosine kinase with the potential to modulate multiple immunological pathways. In part A of a phase 1/2 study (LUNA 2) of ITP patients, rilzabrutinib treatment led to rapid and durable platelet responses with a favorable safety profile. This abstract reports the effects of rilzabrutinib on bleeding and HRQoL on patients in part B of the LUNA 2 study. Methods: The multicenter, open-label, phase 1/2, part B study evaluated the efficacy and safety of rilzabrutinib 400 mg BID in relapsed ITP (NCT03395210). Adults aged 18-80 y were eligible with ≥2 baseline platelet counts <30x10 9/L no less than 7 days apart in the 15 days before the first dose. Eligible patients were required to have a past response (achievement of platelet count ≥50x10 9/L) to intravenous immunoglobulin (IVIg)/anti-D or corticosteroid (CS) that was not sustained, and have failed ≥1 other ITP therapy (not IVIg or CS). Stable doses of concomitant CS/thrombopoietin receptor agonists (TPO-RA) were allowed with rilzabrutinib. The primary endpoints were safety and durable platelet response defined as platelet counts ≥50x10 9/L on ≥8 of the last 12 weeks of rilzabrutinib without rescue medication. Change from baseline in the ITP Bleeding Scale (IBLS) score (0 none to 2 marked bleeding) was a secondary endpoint to assess bleeding across 9 anatomical sites (8 for male/postmenopausal women). An exploratory HRQoL endpoint evaluated the EuroQol-5 Dimensions 5-Level (EQ-5D-5L) + Visual Analog Scale (EQ-VAS) and ITP Patient Assessment Questionnaire (ITP-PAQ; scores 0 worst to 100 best HRQoL). Results: For 26 enrolled patients with data cut-off of 31Jan2023, 15 patients completed 24 weeks of treatment and 11 discontinued due to lack of response (n=5); adverse events (AEs, n=3); and n=1 each lack of response/AE, noncompliance, and erroneous enrollment. At baseline, patients had a median age of 57 y (range, 20-75) with median platelet count of 13x10 9/L (range, 2-24x10 9/L). Patients had a median duration of ITP of 10.3 y (range, 0.7-48.2) and had received a median of 6 prior unique ITP therapies (range, 3-19; 46% splenectomy). Prior ITP medication included CS (100%), TPO-RA (85%), immunosuppressants or IVIg (81%), and rituximab (50%). During the study, 17 patients (65%) received concomitant non-rescue CS and/or TPO-RA. The median compliance to rilzabrutinib for patients with 24 weeks of treatment was 99%. Nine patients (35%; 95% CI, 17-56%) achieved the primary endpoint of durable platelet response. Treatment-related AEs were mainly grade 1/2, with no treatment-related grade ≥2 bleeding/thrombotic events or infections, serious AEs, or deaths. At baseline, the IBLS average score across sites was a mean of 0.27 (SD, 0.27) and mean change from baseline at week 25 was -0.07 (SD, 0.12). The anatomical site with the highest proportion of patients having a baseline score of 2 was skin (n=7 per medical history, n=6 per physical examination), all of whom had a decrease at 25 weeks. At baseline, median EQ-VAS was 78 (range, 8-100) with a median change from baseline (N=26) to week 25 (n=15) of +7 (range, -5 to 77) indicating an improvement in HRQoL. Median changes in ITP-PAQ domain scores from baseline to week 25 (Figure 1) showed clinically meaningful improvement in women's reproductive health with a 21-point increase (range, 0-38; median baseline: 54); clinically meaningful improvement in fatigue/sleep, activity, and overall HRQoL scales with ~13-point increases (range, -23 to 75; median baseline: 53, 50, and 52, respectively); and improvement in psychological, symptoms, social activity, and bother-physical health domains with 4- to 8-point increases (range, -13 to 56; median baseline: 70, 60, 75, 69, respectively). Fear and work domains had high scores at baseline (≥90) with minimal changes over time. Conclusion: Overall, rilzabrutinib showed durable platelet responses, high compliance, and improvements on HRQoL measures in difficult to treat patients with relapsed ITP. There was no evidence of increased bleeding with rilzabrutinib. Clinically meaningful improvements in HRQoL were observed in multiple individual and overall HRQoL health domains following rilzabrutinib.

Strategies in activating lymphatic system on symptom distress and health-related quality of life in patients with heart failure: secondary analysis of a pilot randomized controlled trial.

Abnormal interstitial fluid accumulation remains the major cause for patients with heart failure (HF) to endure a myriad of distressing symptoms and a decline in their health-related quality of life (HRQoL). The lymphatic system is essential in regulating fluid balance within the interstitial compartment and has recently been recognized as an important target for the prevention and mitigation of congestion. This study aimed to investigate the effects of exercises in activating lymphatic system on symptom distress and HRQoL among patients with HF. This was a pre-determined, secondary analysis of the TOLF-HF [The-Optimal-Lymph-Flow for Heart Failure (TOLF-HF)] study, a two-arm pilot randomized controlled trial evaluating the preliminary effects of the lymphatic exercise intervention in enhancing interstitial decongestion among patients with HF. Participants were randomized to receive either a four-week TOLF-HF program in addition to standard care or standard care alone. The Chinese version of the Minnesota Living with Heart Failure Questionnaire (MLHFQ) was employed to measure symptom distress and HRQoL before and after the intervention. Data analyses included descriptive statistics, the independent sample t-test, Pearson's chi-square test, the Mann-Whitney U test, and covariance analysis. Of the 66 patients enrolled, 60 completed the study. The study results exhibited that the TOLF-HF intervention were effective in alleviating both physical and psychological symptom distress. The intervention group yielded significantly lower MLHFQ total scores in comparison to the control group. The odd ratio of achieving meaningful improvement in HRQoL in TOLF-HF group was 2.157 times higher than those in the control group. The TOLF-HF program focusing on activating lymphatic system was effective in alleviating physical and psychological symptom distress as well as improving HRQoL for patients with HF. The tolerability, feasibility, and effectiveness of the TOLF-HF intervention make it a promising intervention for patients to manage HF. http://www.chictr.org.cn/index.aspx, identifier (ChiCTR2000039121).

Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1

BackgroundSevere hemophilia A (HA) negatively impacts health-related quality of life (HRQOL). ObjectivesWe aimed to analyze HRQOL in adult men with severe HA without inhibitors after valoctocogene roxaparvovec gene transfer in the phase 3 trial GENEr8-1. MethodsParticipant-reported outcomes were the hemophilia-specific quality of life questionnaire for adults (Haemo-QOL-A), the EQ-5D-5L instrument, the Hemophilia Activities List (HAL), and the Work Productivity and Activity Impairment Questionnaire: Hemophilia Specific (WPAI+CIQ:HS). Participants completed the questionnaires at baseline and through 104 weeks postinfusion with 6 × 1013 vg/kg of valoctocogene roxaparvovec. Scores were analyzed per participant characteristics and outcomes. ResultsFor 132 HIV-negative participants, mean change from baseline in Haemo-QOL-A Total Score met the anchor-based clinically important difference (CID: 5.5) by week 12; the mean (SD) increase was 7.0 (12.6) at week 104. At week 104, improvement in Consequences of Bleeding, Treatment Concern, Worry, and Role Functioning domain scores exceeded the CID (6). EQ-5D-5L Utility Index scores improved above the CID at week 52, but not at week 104. EQ-5D-5L visual analog scale and HAL scores increased from baseline to week 104. Participants reported less activity and work impairment at week 104 than baseline. Participants with problem joints had lower mean baseline Haemo-QOL-A Total and domain scores than those without them, but improved over 104 weeks, except for 11 participants with ≥3 problem joints. Participants with 0 bleeds during the baseline prophylaxis period reported Haemo-QOL-A score improvements above the CID, including in the Consequences of Bleeding domain. ConclusionValoctocogene roxaparvovec provided clinically meaningful HRQOL improvement for men with severe HA.

Health related quality of life (HRQoL) in patients with triple-class-exposed relapsed/refractory multiple myeloma (TCE RRMM) treated with idecabtagene vicleucel (ide-cel) versus standard regimens: Patient-reported outcomes (PROs) from KarMMa-3 phase 3 randomized controlled trial (RCT).

8032 Background: Patients (pts) with TCE RRMM have few treatment (Tx) options and poor HRQoL. Ide-cel, the first in class CAR T cell Tx for pts with TCE RRMM, improved PFS versus standard (std) regimens in the KarMMa-3 trial. We report the PRO results comparing the Tx arms. PROs provide further understanding of Tx benefit of ide-cel from the pts’ perspective. Methods: KarMMa-3 (NCT03651128) is an open-label phase 3 RCT comparing the efficacy and safety of ide-cel with std regimens in pts with TCE RRMM, who had received 2–4 prior regimens. In addition to clinical endpoints, we evaluated the impact of ide-cel compared with std regimens on the changes in HRQoL, measured by the EORTC QLQ-C30, EORTC QLQ-MY20, and the EQ-5D-5L questionnaires. PROs were collected at baseline (screening), day of infusion and monthly from 2–24 months (mo) and thereafter every 3 mo. This interim analysis reports PROs through 20 mo. Comparisons were performed on least squares mean (LSM) changes from baseline over time between arms using constrained longitudinal data analysis (cLDA). Results: In total, 386 pts were randomized (ide-cel, 254; std regimens, 132). PRO compliance was high over time ( > 80%). At baseline, PROs were similar between arms. LSM changes from baseline to 20 mo showed significant differences ( P < 0.05), with effect sizes of 0.3–0.7, in favor of ide-cel for most domains, including global health status/QoL, cognitive functioning, fatigue, and pain (EORTC QLQ-C30); side effects of Tx (EORTC QLQ-MY20); and the EQ-5D-5L VAS. The difference in overall LSM change reached or exceeded the pre-specified between-groups minimal importance difference (MID) for improvement in most domains in favor of ide-cel (Table). Conclusions: Ide-cel showed statistically significant and clinically meaningful improvements in HRQoL, including key MM symptoms and functioning, for pts with TCE RRMM compared with std regimens. The PRO data for ide-cel expands upon the clinical outcomes observed in the KarMMa-3 trial. Clinical trial information: NCT03651128 . [Table: see text]

Effect of Upadacitinib on Quality of Life and Work Productivity in Active Non-radiographic Axial Spondyloarthritis: Results From Randomized Phase 3 Trial SELECT-AXIS 2.

To evaluate the effect of upadacitinib vs. placebo on health-related quality of life (HRQoL) and work productivity in patients with active non-radiographic axial spondyloarthritis (nr-axSpA) enrolled in the SELECT-AXIS 2 phase 3 randomized controlled trial. Adult patients with active nr-axSpA and an inadequate response to non-steroidal anti-inflammatory drugs were randomized 1:1 to receive upadacitinib 15mg once daily or placebo. Mean changes from baseline in measures of HRQoL (Ankylosing Spondylitis QoL [ASQoL], Assessment of SpondyloArthritis international Society Health Index [ASAS HI], Short-Form 36 Physical Component Summary [SF-36 PCS] score) and Work Productivity and Activity Impairment (WPAI) were assessed through 14weeks based on mixed-effects repeated measures or analysis of covariance models. The proportions of patients with improvements ≥ minimum clinically important differences (MCID) were assessed in HRQoL measures at week 14 using non-responder imputation with multiple imputation. At week 14, upadacitinib- vs. placebo-treated patients reported greater improvements from baseline in ASQoL and ASAS HI (ranked, P < 0.001) and in SF-36 PCS and WPAI overall work impairment (nominal P < 0.05). Improvements were observed as early as week 2 in ASAS HI. Greater proportions of upadacitinib vs. placebo-treated patients reported improvements ≥ MCID in ASQoL (62.6 vs. 40.9%), ASAS HI (44.8 vs. 28.8%), and SF-36 PCS (69.3 vs. 52.0%), with numbers needed to treat < 10 for all (nominal P ≤ 0.01). Improvements ≥ MCID were consistently observed irrespectively of prior exposure to tumor necrosis factor inhibitors. Upadacitinib provides clinically meaningful improvements in HRQoL and work productivity in patients with active nr-axSpA. NCT04169373, SELECT-AXIS 2.

Self-evidence-based digital care programme improves health-related quality of life in adults with a variety of autoimmune diseases and long COVID: a retrospective study

ObjectiveTo retrospectively investigate the feasibility and impact on health-related quality of life (HRQoL) of a digital care programme (DCP) designed to guide personalised diet and integrative interventions in a variety...

Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema

BackgroundHereditary angioedema (HAE) is associated with a substantial disease burden. Lanadelumab reduced the HAE attack rate during 132 weeks of follow-up in the HELP open-label extension (OLE) Study (NCT02741596). ObjectiveTo measure the impact of long-term lanadelumab treatment on patient-reported outcomes (PROs). MethodsRollover patients (completed the 26-week HELP study [NCT02586805]) and nonrollovers (newly enrolled) received lanadelumab 300 mg every 2 weeks. PROs (Angioedema Quality of Life Questionnaire [AE-QoL], Short Form Health Survey 12-item version 2, Hospital Anxiety and Depression Scale, Work Productivity and Activity Impairment–General Health Questionnaire, and EQ-5D-5L questionnaire) were assessed at baseline (day 0 of HELP OLE) and various time points until the end-of-study visit. The Angioedema Control Test, Treatment Satisfaction Questionnaire for Medication, and Global Impression of Treatment Response were administered starting at week 52. ResultsThe mean (SD) change in AE-QoL total score from baseline to end-of-study for rollovers (n = 90) was −10.2 (17.9), exhibiting further improvement from HELP in health-related quality of life (HRQoL); 48.9% of rollovers achieved the previously defined 6-point minimal clinically important difference. Nonrollovers (n = 81) reported a change of −19.5 (21.3). Controlled disease (Angioedema Control Test total score ≥10) was reported by 90.2% of rollovers and 95.9% of nonrollovers at the end of the study. Excellent treatment response was reported by 78.7% of patients and 82.4% of investigators. Results from other PROs indicated a slight improvement in anxiety, a high level of satisfaction with treatment, and increased work productivityor activity. ConclusionClinically meaningful improvement in HRQoL was exhibited with long-term lanadelumab treatment, supporting the benefit of lanadelumab therapy associated with attack prevention. Trial RegistrationClinicalTrials.gov Identifiers: NCT02586805 (HELP Study) and NCT02741596 (HELP open-label extension).

Health-Related Quality of Life in Adults With Adolescent- and Adult-Onset Systemic Lupus Erythematosus: A Longitudinal Study of a Multiethnic US Cohort.

The long-term impact of childhood-onset systemic lupus erythematosus (SLE) on health-related quality of life (HRQoL) in adult SLE patients in comparison to those with adult-onset SLE is unknown. We aim to examine and compare HRQoL trajectories in adults with adolescent- and adult-onset SLE. Patients enrolled in the LUpus in MInorities: NAture versus Nurture cohort were included. Adolescent-onset SLE were those diagnosed before 24 years of age, and adult-onset SLE were those diagnosed otherwise. Sociodemographic, clinical, medications, behavioral/psychological, and functioning data were obtained. Longitudinal trajectories of the physical component summary (PCS) and the mental component summary (MCS) Short Form 36 health survey scores were compared between the groups using a linear mixed model accounting for time-dependent and independent covariates. A total of 470 SLE patients were included (95 with adolescent-onset SLE and 375 with adult-onset SLE). The mean ± SD age at diagnosis was 19.7 ± 2.8 years in the adolescent group and 39.3 ± 11.0 years in the adult group. The baseline PCS scores were higher (better physical functioning) in adolescent-onset SLE than in adult-onset SLE (38.9 versus 34.3, respectively; P < 0.001); however, the baseline MCS scores were comparable between the groups (41.4 versus 40.5, respectively; P=0.53). The HRQoL improved equally in both groups with no statistically significant difference within and between the groups (last mean PCS and MCS scores 43.9 and 45.3 in adolescent-onset SLE; 38.1 and 43 in adult-onset SLE). Adults with adolescent-onset SLE exhibited better physical functioning than those in the adult SLE group, despite more severe disease; noteworthy, HRQoL was below the general US population, despite clinically meaningful improvement in HRQoL over time in both groups.

Does kidney transplant improve health-related quality of life for youth with chronic kidney disease?

Youth with chronic kidney disease are known to have impaired health-related quality of life (HRQOL), particularly as the disorder increases in severity. Few prospective, longitudinal investigations of HRQOL within the context of pediatric chronic kidney disease exist. In the current issue, Guha etal. provide a longitudinal assessment of HRQOL for a cohort of youth with chronic kidney disease. Their findings suggest that children may experience meaningful improvement in HRQOL when they transition from dialysis to transplant.

SUSTAINED IMPROVEMENT IN HEALTH-RELATED QUALITY OF LIFE WITH CONTINUED LANADELUMAB TREATMENT IN HEREDITARY ANGIOEDEMA

<h3>Introduction</h3> Lanadelumab is approved for the prevention of hereditary angioedema attacks. Reduced attack rate is associated with improved health-related quality of life (HRQoL). HRQoL was assessed in patients who completed the HELP Study (NCT02586805) and rolled into the HELP OLE Study (NCT02741596) using the Angioedema Quality of Life Questionnaire (AE-QoL). <h3>Methods</h3> Rollover patients completed 26 weeks' treatment in HELP, where they received placebo or lanadelumab (150mg every 4 weeks [Q4W], 300mg Q4W, or 300mg every 2 weeks [Q2W]), then continued into HELP OLE with lanadelumab 300mg Q2W for ≤33 months. A decrease in AE-QoL total score of ≥6 was considered clinical meaningful improvement. <h3>Results</h3> From the baseline of HELP to the end of study (EOS) visit in HELP OLE, rollovers (n=98) had a mean(SD) change in AE-QoL total score of -25.1(19.8). The reduction of total score in rollovers (n=97) during HELP OLE was -10.2(17.9). Among patients who received lanadelumab 300mg Q2W in HELP (n=24), total scores for patients with baseline attack rates of 1–<2 (n=7), 2–<3 (n=5), and ≥3 (n=12) attacks/month were reduced from 23.1(27.3), 15.3(9.5), and 24.4(22.0), respectively, at HELP OLE baseline, to 10.3(8.9) 18.0(12.9), and 20.4(20.6), respectively, at EOS. For rollovers treated with placebo in HELP (n=33), total scores for patients with baseline attack rates of 1–<2 (n=10), 2–<3 (n=7), and ≥3 (n=16) attacks/month were reduced from 37.8(20.0), 36.3(14.2), and 43.2(23.4), respectively, at HELP OLE baseline, to 17.5(15.3), 8.6(8.4), and 27.4(24.6), respectively, at EOS. <h3>Conclusion</h3> These data demonstrate sustained clinically meaningful improvement in HRQoL with continued lanadelumab treatment.

Rate versus rhythm control in patients with newly diagnosed atrial fibrillation: Effects of the treatment timing on health status outcomes

Recent randomized clinical trials have demonstrated that applying rhythm control during the early stage of atrial fibrillation (AF) may lead to improved clinical outcomes. However, the effects of this modality on health-related quality of life (HRQoL) have not been fully investigated. We aimed to assess the association between the AF stage, determined by the time between AF diagnosis and referral to the cardiology clinic, and HRQoL outcomes. Using an outpatients-based multicenter AF registry (n=3,313), we analyzed 2,070 patients with AF diagnosed within 5 years. The patients were divided into 2 groups according to AF stage: early and late AF (AF duration ≤1 and >1 year, respectively). All patients had HRQoL information collected at baseline and 1 year after their initial treatment (assessed via the Atrial Fibrillation Effect on Quality-of-Life-overall summary [AFEQT-OS] score, with higher scores reflecting better HRQoL). The change in AFEQT-OS was adjusted for patient characteristics using a generalized linear mixed model. The early AF group (n=1,644) was older (early, 68.5 ± 11.1, late, 64.4 ± 10.6 years, P < .001) and had more heart failure (early, 19.9%, late, 12.7%, P < .001) than the late AF group (n=426). At 1 year after treatment, the adjusted changes in AFEQT-OS were similar in patients with rhythm (adjusted difference [SE], early, 8.4 [1.2], late, 7.2 [1.4], P=.15) or rate (early, 4.0 [0.7], late, 2.3 [1.4], P=.16) control, regardless of AF stage. Furthermore, the improvement in HRQoL was similar between early and late AF in patients undergoing catheter ablation (early, 10.2 [2.1], late, 9.8 [2.4], P=.78), whereas a significant difference was observed in those receiving antiarrhythmic drug therapy alone (early, 10.2 [1.4], late, 3.5 [2.2], P < .001). Rhythm control therapy provided clinically meaningful improvements in HRQoL, regardless of AF stage. For patients with impaired HRQoL, AF duration should not be a deterrent to treatment, especially catheter ablation.

Impact of Comorbidities on Health-related Quality of Life in Nontransplant Eligible Patients With Newly Diagnosed Multiple Myeloma.

Impact of Comorbidities on Health-related Quality of Life in Nontransplant Eligible Patients With Newly Diagnosed Multiple Myeloma.

Health-related quality of life in patients with relapsed/refractory multiple myeloma (RRMM) treated with teclistamab, a B-cell maturation antigen (BCMA) x CD3 bispecific antibody: Patient-reported outcomes in MajesTEC-1.

8033 Background: As multiple myeloma (MM) negatively affects patients’ (pts) health-related quality of life (HRQoL), assessment of patient-reported outcomes (PROs) in addition to clinical outcomes is important. Teclistamab (tec; JNJ-64007957) is an off-the-shelf bispecific antibody that redirects CD3+ T cells to mediate T-cell activation and subsequent lysis of BCMA-expressing MM cells. Initial results from the pivotal cohort of the phase 1/2 MajesTEC-1 study demonstrated that tec was well tolerated with encouraging efficacy in pts who received ≥3 prior lines of treatment (LOT; including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody). Here we report PROs from this cohort. Methods: Pts (aged ≥18 years) had documented RRMM (International Myeloma Working Group criteria), progressive/measurable disease, and had previously received ≥3 prior LOT; prior anti-BCMA treatment (tx) was not allowed. Pts received weekly subcutaneous tec at the recommended phase 2 dose (1.5 mg/kg with step-up doses of 0.06 and 0.3 mg/kg). PROs were assessed at screening and every even cycle (cycles 2–8 reported here) using the EORTC QLQ-C30 (range: 0–100; higher scores indicate better global health status [GHS] but greater symptom severity [symptom scales]) and the EuroQol 5-dimensional descriptive system (visual analog scale [VAS] range: 0 [worst imaginable health state] to 100 [best imaginable]). Tx effect was assessed by a mixed-effects model with repeated measures; the proportion of pts with meaningful improvement was defined as a change ≥10 points. Time to worsening was determined using the Kaplan-Meier estimate. Results: A total of 110 pts were included (median follow-up: 7.8 mos). Overall PRO compliance rates were high (baseline [BL]: 85–90%; cycles 2–8: 80–94%). Tec improved overall HRQoL as evidenced by improvements in GHS scores (cycles 2–8) and reduction in pain (-4.2 [cycle 2] to -15.1 [cycle 8]; Table), with no overall change in physical functioning and fatigue. The proportions of pts with meaningful improvements from BL at cycle 8 were GHS: 50%; physical functioning: 35%; pain: 65%; fatigue: 73%; 50% of pts reported meaningful improvement in their overall health (VAS). Median time to improvement from baseline was ̃1.5 months (with nausea/vomiting and fatigue taking longer to improve), while median time to worsening (all symptoms) ranged from 2 months to not estimable. Conclusions: Consistent with clinical outcomes, pts treated with tec reported rapid, clinically meaningful improvements in HRQoL. Clinical trial information: NCT04557098. [Table: see text]

Updated health-related quality of life of patients with TRK-fusion cancer treated with larotrectinib in clinical trials.

6563 Background: NTRK gene fusions have been identified as oncogenic drivers in patients (pts) with TRK fusion cancer across multiple solid tumors. Larotrectinib, a highly selective, CNS-active TRK inhibitor has shown high response rates, durable disease control, and a favorable safety profile in pts with TRK fusion cancer and is approved in over 40 countries. Larotrectinib has demonstrated rapid health-related quality of life (HRQOL) improvement in a group of 57 adult and pediatric pts (Kummar et al, Curr Prob Canc 2021). Here, we report updated HRQOL results for larger group of pts treated with larotrectinib. Methods: HRQOL data were collected in two ongoing trials (NCT02576431, NCT02637687) of larotrectinib in pts with TRK-fusion cancer using the EORTC QLQ-C30, EQ-5D-5L questionnaires and were analyzed descriptively and longitudinally. Scores from the EORTC QLQ-C30 Global Health Status (GHS), EQ-5D-5L VAS range from 0 to 100, with higher scores indicating better QOL. We also calculated the proportion of pts with either below normal or normal and above normal HRQOL scores against values in the literature for the US general population. Results: By July 2021, 113 adults with TRK-fusion cancer had received larotrectinib and completed the baseline (BL) and ≥ 1 post-BL questionnaire. The majority of pts had clinically meaningful HRQOL improvements during treatment (Table). For EORTC QLQ-C30 GHS, most adults maintained or improved scores from BL at or above the normal population level category. HRQOL improvements (change from BL &gt; 0) occurred after ̃2 months of treatment in 75% of adults. Median duration of pts with sustained improvement in EORTC QLQ-C30 GHS, and EQ-5D-5L VASs was 12.5 months (range, 1.8-34.1), and 12.9 months (range, 1.8-34.0), respectively. HRQOL results were consistent across multiple data cuts. Conclusions: Patients with TRK-fusion cancer treated with larotrectinib continued to have rapid, clinically meaningful, and sustained improvements in HRQOL. Clinical trial information: NCT02576431, NCT02122913, NCT02637687.

Short- and long-term effect of high versus low-to-moderate intensity exercise to optimise health-related quality of life after oncological treatment—results from the Phys-Can project

PurposeThis study aimed to evaluate the effect of high intensity (HI) vs low-to-moderate intensity (LMI) exercise on health-related quality of life (HRQoL) up to 18 months after commencement of oncological treatment in patients with breast, colorectal or prostate cancer. In addition, we conducted a comparison with usual care (UC).MethodsPatients scheduled for (neo)adjuvant oncological treatment (n = 577) were randomly assigned to 6 months of combined resistance and endurance training of HI or LMI. A longitudinal descriptive study (UC) included participants (n = 89) immediately before the RCT started. HRQoL was assessed by EORTC QLQ-C30 at baseline, 3, 6 and 18 months (1 year after completed exercise intervention) follow-up. Linear mixed models were used to study the groups over time.ResultsDirectly after the intervention, HI scored significant (P = 0.02), but not clinically relevant, higher pain compared with LMI. No other significant difference in HRQoL was found between the exercise intensities over time. Clinically meaningful improvements in HRQoL over time were detected within both exercise intensities. We found favourable significant differences in HRQoL in both exercise intensities compared with UC over time.ConclusionThis study adds to the strong evidence of positive effect of exercise and shows that exercise, regardless of intensity, can have beneficial effects on HRQoL during oncological treatment and also for a substantial time after completion of an exercise intervention. In this study, for one year after.Implications for cancer survivorsPatients can be advised to exercise at either intensity level according to their personal preferences, and still benefit from both short-term and long-term improvements in HRQoL.