Patient-reported Health-related Quality Of Life Research Articles

Health-related quality of life and utility of maternity health states amongst post-partum Australians.

This study aimed to measure patient-reported health-related quality of life amongst post-partum women in Queensland, Australia. Patient-reported health-related quality of life data was prospectively collected from 134 post-partum women using the EQ-5D-5L at weekly intervals during the first six weeks following birth. Data across the five health domains of the EQ-5D-5L was converted to a single health utility value to represent overall health status. Linear mixed modelling and regression analysis were used to examine changes in utility over the first six weeks post-birth and determine associations between utility and clinical and demographic characteristics of post-partum women. Gestation at birth and weeks post-partum were significantly associated with utility values when considered in a multivariate linear mixed model. Mean utility values increased by 0.01 for every week increase in gestation at birth, and utility values were 0.70 at one week post-partum and increased to 0.85 at six weeks post-partum, with the largest increase occurring between one- and two-weeks post-birth. When controlling for variables that were found to predict utility values across the first six weeks post-partum, no single state of health predicted utility values at one-week post-partum. Maternity services can use our data and methods to establish norms for their own service, and researchers and maternity services can partner to conduct cost-effectiveness analysis using our more relevant utility values than what is currently available. Time since birth and gestational age of the woman's baby should be considered when selecting post-partum health state utility values for maternity services cost-effectiveness analyses.

Postprostatectomy Radiotherapy Timing and Long-Term Health-Related Quality of Life

The association between radiotherapy (RT) timing after radical prostatectomy and long-term patient-reported health-related quality of life (HRQOL) in men with prostate cancer is unknown. To measure long-term HRQOL in men with prostate cancer up to 15 years after prostatectomy with or without RT and examine whether early vs late postprostatectomy RT is associated with differences in sexual, urinary, and bowel HRQOL. A prospective, multicenter, longitudinal cohort analysis using HRQOL data from the PROST-QA (2003-2006) and RP2 consortium (2010-2013) studies was conducted. Men with localized prostate cancer undergoing radical prostatectomy were included. Data were analyzed between May 8, 2023, and March 1, 2024. The study was conducted in 12 high-volume academic medical centers in the US. Men were stratified based on receipt and timing of postprostatectomy RT: prostatectomy only, early RT (<12 months), and late RT (≥12 months). Longitudinal sexual, incontinence, urinary irritation, bowel, and hormonal/vitality HRQOL were measured via the Expanded Prostate Cancer Index Composite at baseline; months 2, 6, and 12; and annually thereafter. Treatment groups were compared using multivariable linear mixed-effects models of change in longitudinal domain scores. Pad use for incontinence was measured longitudinally among men receiving postprostatectomy RT. A total of 1203 men were included in the study: prostatectomy only (n = 1082), early RT (n = 57), and late RT (n = 64). Median age for the entire cohort was 60.5 (range, 38.8-79.7) years, and 1075 men (92.0%) were White. Median follow-up was 85.6 (IQR, 35.8-117.2) months. Compared with men receiving prostatectomy alone, those receiving postprostatectomy RT had significantly greater decreases in sexual, incontinence, and urinary irritation HRQOL. However, timing of postprostatectomy RT, specifically early vs late, was not associated with a long-term decrease in any HRQOL domain. There was evidence of improved recovery of sexual, continence, and urinary irritation scores among men receiving early RT compared with those receiving late RT after prostatectomy. Before the start of postprostatectomy RT, 39.3% of men in the early RT cohort and 73.4% of men in the late RT cohort were pad-free. By the sixth visit post-RT, 67.4% in the early RT cohort and 47.6% in the late RT cohort were pad-free. In this multicenter, prospective analysis, postprostatectomy RT appeared to be negatively associated with long-term HRQOL across all domains. However, receipt of early vs late postprostatectomy RT may result in similar long-term HRQOL outcomes.

Internalizing Symptoms and Their Impact on Patient-Reported Health-Related Quality of Life and Fatigue among Patients with Craniopharyngioma During Proton Radiation Therapy.

The aim of this study was to describe fatigue, health-related quality of life (HRQOL) and brain tumor-associated symptoms after surgical resection and during proton radiotherapy, using latent class analysis (LCA), and to determine if there is class membership change among pediatric patients with craniopharyngioma. For all patients (n = 92), demographic and disease-related/clinical variables were attained, and patient reported outcomes were collected prior to proton therapy, at week three, and at the completion of proton therapy. The mean scores for fatigue, HRQOL, and brain tumor symptoms were compared over time and profiles were identified. Factors that influenced profile status and transition probability were examined. Fatigue, HRQOL, and brain tumor symptoms improved over time during proton therapy; however, a subset remained in the lower profile, profile 1, associated with increased internalizing behaviors, compared to profile 2. Future study should explore the bidirectional relationship of sleep, worry and anxiety in the context of ongoing radiotherapy.

Capivasertib and fulvestrant for patients with hormone receptor-positive, HER2-negative advanced breast cancer (CAPItello-291): patient-reported outcomes from a phase 3, randomised, double-blind, placebo-controlled trial

CAPItello-291 is an ongoing phase 3 trial in which capivasertib-fulvestrant significantly improved progression-free survival versus placebo-fulvestrant in patients with hormone receptor-positive, HER2-negative advanced breast cancer who had relapse or disease progression during or after aromatase inhibitor treatment, in both the overall population and in patients with PIK3CA, AKT1, or PTEN-altered tumours. This study further explored patient-reported health-related quality of life (HRQOL), functioning, symptoms, and symptom tolerability in CAPItello-291. This phase 3, randomised, double-blind, placebo-controlled trial, which was conducted across 193 hospitals and cancer centres in 19 countries, enrolled women with any menopausal status or men, aged ≥18 years (≥20 years in Japan), with hormone receptor-positive, HER2-negative locally advanced or metastatic breast cancer who had relapse or disease progression during or after treatment with an aromatase inhibitor, with or without previous cyclin-dependent kinase (CDK) 4 or 6 inhibitor therapy. Patients had an Eastern Cooperative Oncology Group/WHO performance score of 0 or 1 and could have received up to two previous lines of endocrine therapy and up to one previous line of chemotherapy for advanced disease. Patients were randomly assigned (1:1) using block randomisation (stratified according to the presence or absence of liver metastases, previous use of a CDK4/6 inhibitor [yes vs no], and geographical region) to receive oral capivasertib 400 mg (twice daily for 4 days, followed by 3 days off) plus intramuscular fulvestrant 500 mg (every 14 days for the first three injections, then every 28 days) or placebo with matching fulvestrant dosing. The dual primary endpoint of the trial was investigator-assessed progression-free survival assessed both in the overall population and among patients with PIK3CA, AKT1, or PTEN-altered tumours. The EORTC Quality of Life Questionnaire 30-item core module (QLQ-C30) and breast module (QLQ-BR23), Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), and Patient Global Impression of Treatment Tolerability (PGI-TT) questionnaires were used to assess patient-reported outcomes. Evaluation of EORTC QLQ-C30 and EORTC QLQ-BR23 were secondary endpoints and evaluation of PRO-CTCAE and PGI-TT were pre-defined exploratory endpoints, and these endpoints are the subject of analysis in this Article. Data were collected at baseline and prespecified timepoints. Patient-reported outcomes were analysed in all randomly assigned patients with an evaluable baseline assessment and at least one evaluable post-baseline assessment. Change from baseline was assessed using mixed model with repeated measures for EORTC QLQ-C30 and summarised for QLQ-BR23. Time to deterioration was described using the Kaplan-Meier method. PGI-TT and PRO-CTCAE responses were summarised at each treatment cycle. Patient-reported outcomes were not prospectively powered for statistical comparison. The trial is registered with ClinicalTrials.gov, NCT04305496. Between June 2, 2020, and Oct 13, 2021, 901 patients were enrolled, of whom 708 patients were randomly assigned to receive capivasertib-fulvestrant (n=355) or placebo-fulvestrant (n=353). The median age of the patients was 59 years (IQR 51-67) in the capivasertib-fulvestrant group and 58 years (IQR 49-66) in the placebo-fulvestrant group. At data cutoff (Aug 15, 2022), the median duration of follow-up for progression-free survival in censored patients was 13·0 months (IQR 9·1-16·7) for capivasertib-fulvestrant and 12·7 months (IQR 2·0-16·4) for placebo-fulvestrant in the overall population. EORTC QLQ-C30 global health status/quality of life (GHS/QOL) scores were maintained from baseline and were similar between treatment groups throughout the study period (difference in mean change from baseline of -2·5 [95% CI -4·5 to -0·6] with capivasertib-fulvestrant vs -5·6 [-7·9 to -3·4] with placebo-fulvestrant; treatment difference 3·1 [95% CI 0·2 to 6·0]). Median time to deterioration in EORTC QLQ-C30 GHS/QOL was 24·9 months (95% CI 13·8 to not reached) in the capivasertib-fulvestrant group and 12·0 months (10·2 to 15·7) in the placebo-fulvestrant group (hazard ratio [HR] 0·70, 95% CI 0·53 to 0·92). Time to deterioration HRs for all EORTC QLQ-C30 and QLQ-BR23 subscale scores showed little difference between the treatment groups, except for diarrhoea, which was worse in the capivasertib-fulvestrant group than in the placebo-fulvestrant group (HR 2·75, 95% CI 2·01-3·81). In PRO-CTCAE symptom assessment, the proportion of patients reporting loose and watery stools "frequently" or "almost constantly" was 29% higher at cycle 1, day 15 in the capivasertib-fulvestrant group than in the placebo-fulvestrant group, decreasing at subsequent cycles. Other PRO-CTCAE-reported symptoms (rash, mouth or throat sores, itchy skin, and numbness or tingling in hands or feet) were absent or mild in most patients in both groups throughout treatment. According to the PGI-TT, most patients in both groups reported "not at all" or "a little bit" of bother from treatment side-effects. Patient-reported outcomes from CAPItello-291 demonstrated that capivasertib-fulvestrant delayed time to deterioration of GHS/QOL and maintained other dimensions of HRQOL (except symptoms of diarrhoea) similarly to fulvestrant. With the clinical efficacy and manageable safety profile, these exploratory results further support the positive benefit-risk profile of capivasertib-fulvestrant in this population. AstraZeneca.

Assessing core outcome set uptake in randomized controlled trials for chronic kidney disease: Cross-sectional analysis

Main problemChronic kidney disease (CKD) is a progressive condition that affects millions of people worldwide. A standardized core outcome set (COS) was developed for CKD by the International Consortium for Health Outcomes and Measurements in 2019. This study aims to evaluate the frequency of measurement for these outcomes before and after the publication of the COS. MethodsA literature search was done to gather the phase III/IV clinical trials evaluating chronic kidney disease through ClinicalTrials.gov. Data extraction of included studies was completed in a masked, duplicate fashion. The included studies were evaluated for characteristics such as survival, burden of disease, patient-reported health-related quality of life, and treatment modality-specific outcomes. ResultsOur results showed that the majority of all COS domains were inadequately measured in CKD clinical trials before and after publication of the COS. Despite the increase in COS measurements following publication, the average percent of COS outcomes measured was less than 40 % per year even after four years. ConclusionThere is a notable deficiency in the complete measurement of COS among all domains both before and after COS publication. We suggest efforts be made to improve the adoption of consistent outcome measures that would benefit the growing population of patients affected by CKD.

Comparative Efficacy of Biologics and Small Molecule Therapies in Improving Patient-Reported Outcomes in Ulcerative Colitis: Systematic Review and Network Meta-Analysis

Abstract Background Ulcerative colitis (UC) is a chronic disorder with a considerable negative impact on health-related quality of life (HRQoL), which has been recently recognized as an important treatment target. The purpose of this study is to compare the efficacy of different biologics and small molecule therapies in achieving better patient-reported outcomes and HRQoL in patients with UC. Methods We performed a systematic review and network meta-analysis of the EMBASE, MEDLINE, and Cochrane Central databases from inception until February 1, 2024. The primary endpoint was clinical remission in the patient-reported outcome (PRO-2) score in UC patients who were treated with different biologics or small molecules during induction and maintenance phases. PRO-2 score is the sum of both stool frequency and rectal bleeding subscores. The secondary outcome was improvement of HRQoL defined as an increase in Inflammatory Bowel Disease Questionnaire score of ≥16 points from baseline or any change in total score from baseline. A random effects model was used, and outcomes were reported as odds ratio with 95% confidence interval. Interventions were ranked per the SUCRA (surface under the cumulative ranking curve) score. Results A total of 54 studies were included in the primary outcome analysis and 15 studies were included in the secondary outcome analysis. The primary analysis showed that during the induction phase all of included drugs were better than placebo in improving the PRO-2 score. Interestingly, upadacitinib was found to be superior to most medications in improving PRO-2 scores. The secondary analysis showed that guselkumab ranked first in the improvement of the Inflammatory Bowel Disease Questionnaire score, followed by upadacitinib during the induction phase. Conclusion Upadacitinib ranked first in PRO-2 clinical remission during the induction and maintenance phases. Guselkumab, mirikizumab, tofacitinib, and upadacitinib were the only novel medications that were superior to placebo in improving HRQoL in UC, with guselkumab ranking the highest, followed by tofacitinib and upadacitinib. During maintenance of remission, tofacitinib ranked highest in improving HRQoL.

The Impact of Postoperative Complications on Recovery of Health-Related Quality of Life and Functional Capacity After Pancreatectomy: Findings From a Prospective Observational Study.

To evaluate the extent to which postoperative complications impact patient health-related quality of life (HRQoL) and survival after pancreatic surgery. Pancreatectomy is frequently associated with severe postoperative morbidity, which can affect patient recovery. Few and conflicting data are available regarding the effect of post-pancreatectomy complications on patient-reported HRQoL. This is an observational cohort study including consecutive patients enrolled in a prospective clinical trial (NCT04431076) who underwent elective pancreatectomy (2020-2022). Before surgery and on postoperative days 15, 30, 90, and 180, patients completed the PROMIS-29 profile and Duke Activity Status Index questionnaires to assess their HRQoL and functional capacity. Mean differences in HRQoL scores were obtained using multivariable linear regression adjusting for preoperative scores and confounders. Of 528 patients, 370 (70%) experienced morbidity within 90 days, and 154 (29%) had severe complications (Clavien-Dindo grade >2). Delayed gastric emptying had the greatest impact on HRQoL, showing decreased mental health up to POD90 and physical health up to POD180 compared with uncomplicated patients. An inverse relationship between complication severity grade and HRQoL was evident for most domains, with Clavien-Dindo grade 3b to 4 patients showing worse HRQoL and functional capacity scores up to 6 months after surgery. In 235 pancreatic cancer patients, grade 3b and 4 complications were associated with reduced disease-specific survival (median 25 vs 41mo, P <0.001). In patients undergoing pancreatic resection, postoperative complications significantly impact all domains of patient quality of life with a dose-effect relationship between complication severity and impairment of HRQoL and functional capacity.

Health-related quality of life is a predictor of readmission following emergency laparotomy.

Health-related quality of life (HRQoL) is a multidimensional concept used to examine the impact of patient-perceived health status on quality of life. Patients' perception of illness affects outcomes in both medical and elective surgical patients; however, not much is known about how HRQoL effects outcomes in the emergency surgical setting. This study aimed to examine if patient-reported HRQoL was a predictor of unplanned readmission after emergency laparotomy. This study included 215 patients who underwent emergency laparotomy at the Copenhagen University Hospital, Herlev, between August 1, 2021, and July 31, 2022. Patient-reported HRQoL was assessed with the EuroQol group EQ5D index (EQ5D5L descriptive system and EQ-VAS). The population was followed from 0 to 180days after discharge, and readmissions and days alive and out of hospital were registered. A Cox proportional hazard model was used to examine HRQoL and the risk of readmission within 30 and 180days. Within 30days, 28.4% of patients were readmitted; within 180days, the number accumulated to 45.1%. Low self-evaluated HRQoL predicted 180-day readmission and was significantly associated with fewer days out of hospital within both 90 and 180days. Low HRQoL and discharge with rehabilitation were independent risk factors for short- (30-day) and long-term (180-day) emergency readmission. Patient-perceived quality of life is an independent predictor of 180-day readmission, and the number of days out of hospital was correlated to self-reported HRQoL.

Stereotactic Body Proton Therapy Versus Conventionally Fractionated Proton Therapy for Early Prostate Cancer: A Randomized, Controlled, Phase 3 Trial

PurposeWe aimed to determine if ultrahypofractionated proton therapy delivered via stereotactic body proton therapy (SBPT) is noninferior to conventionally fractionated proton therapy (CFPT) in patients with early prostate cancer. Methods and MaterialsThis study was a multicenter, randomized, controlled, noninferiority phase 3 trial that included patients with histologically confirmed low-risk prostate adenocarcinoma defined by Gleason score grouping 1, Prostate-specific antigen <10 ng/mL, and clinical stage T1-T2a N0 M0 according to 7th edition of the American Joint Committee on Cancer tumor-node-metastasis cancer staging system. Eligible participants were randomly assigned initially at a 1:1 ratio and later at a 2:1 ratio to SBPT (38 Gy in 5 fractions) or CFPT (79.2 Gy in 44 fractions). The primary endpoint was freedom from failure (FFF) at 2 years from the date of randomization. Noninferiority for FFF was determined based on 1-sided confidence intervals. Toxicities were compared at different time points using Fisher's exact test. Health-related quality-of-life (HRQoL) was analyzed at different time points using a mixed-effects linear model. This trial is registered with ClinicalTrials.gov, NCT01230866, and is closed to accrual. ResultsBetween December 10, 2010, and September 29, 2020, 144 patients were enrolled and 135 were randomly assigned (90 to the SBPT group and 45 to the CFPT group). The median follow-up was 5 years (IQR, 3.9-5.2). The 2-year FFF was 100% for both groups, with the 1-sided 5-year risk difference in FFF between groups reported as 2.63% (90% CI, −1.70% to 6.96%), favoring the SBRT arm, thus fulfilling the prespecified criteria for noninferiority of SBPT compared with CFPT. Rates of gastrointestinal and genitourinary G2 and G3 toxicities did not differ significantly between groups. Further, HRQoL metrics did not differ significantly between groups over the study's median follow-up. ConclusionsSBPT is noninferior to CFPT regarding FFF, with similar long-term genitourinary and gastrointestinal toxicity rates and minimal impact in patient-reported HRQoL over time.

Health-related quality scores in childhood interstitial lung disease: Good agreement between patient and caregiver reports.

Childhood interstitial lung disease (chILD) is a heterogeneous group of mostly chronic respiratory disorders. Assessment of health-related quality of life (HrQoL) in chILD has become increasingly important in clinical care and research. The aim of this study was to assess differences between patient-reported (self) and caregiver-reported (proxy) HrQoL scores. This study used data obtained from the chILD-EU Register. After inclusion (baseline), the patient's health status was followed up at predefined study visits. At each study visit, caregivers and patients were handed validated, age-specific HrQoL questionnaires. HrQoL data entered at baseline were used to compare self- and proxy-reported HrQoL scores. For the longitudinal analysis, we compared HrQoL scores between the baseline and the next follow-up visit. No differences between patient- and caregiver-reported HrQoL scores were found for school functioning, chILD-specific questionnaire score, and physical health summary score. Self-reported HrQoL scores were higher for the subscales emotional functioning (77.4 vs. 70.7; p < .001), social functioning (81.9 vs. 76.2; p < .001), as well as psycho-social summary score (76.5 vs. 71.8; p < .001) and total score (74.7 vs. 70.8; <.001). The longitudinal analysis showed that a significant change in a patient-reported HrQoL score resulted in a significant change in a caregiver-reported HrQoL score after a mean time of 11.0 months (SD 9.4). We found a good agreement between children- and caregiver-related HrQoL scores. In chILD, caregivers are able to sense changes in children's HrQoL scores over time and may be used as a proxy for children unable to complete HrQoL questionnaires.

Patients’ voices matter: Translating pretreatment patient-reported outcomes into high and low health-related quality of life provides a new clinical aspect on prognosis and survival in patients with previously untreated diffuse large B‐cell lymphoma.

e23165 Background: Patient-reported health-related quality of life (HRQoL) measures collected prior to treatment initiation have been shown to be prognostic of disease outcomes and survival in patients with previously untreated diffuse large B-cell lymphoma (DLBCL), including progression-free survival (PFS) and overall survival (OS), However, these HRQoL measures are usually expressed as numbers from a scale (e.g. from 0 to 100), which limit their ease of clinical utility compared to binary scales (i.e. high vs. low). Using HRQoL measures collected from the phase 3 study GOYA, we aim to convert these measures into binary scales by identifying optimal cutoffs and demonstrate their clinical utility in differentiating patient risks in DLBCL. Methods: HRQoL data from the physical functioning (PF2), global health status (QL2), and fatigue (FA) scales from the EORTC QLQ-C30 questionnaire and the lymphoma subscale (LYMS) from the FACT-LYM questionnaire were used in this analysis. The optimal cutoff points were identified using statistical metrics that evaluate the prognostic value and goodness-of-fit for each assessed cutoff point. The top performing binary HRQoL scale was further assessed in its clinical utility as an addition to the Internal Prognostic Index (IPI), a standard risk classification tool in DLBCL based on 5 binary measures of patient baseline characteristics, to evaluate whether adding the HRQoL scale would potentially improve the risk classification. Results: As shown in the table, while all binary scales can clearly differentiate patient overall survival (demonstrated by the hazard ratios), PF2 numerically exhibited the best performance, and was further assessed as an addition to IPI. After adding PF2 to the base OS model containing only IPI, we observed improvements in model performance metrics, including the concordance index and the goodness-of-fit. In addition, among patients classified as high risk by IPI (n = 185, 3-yr OS: 65% [95% CI 68-72%]), those with low HRQoL (n = 102, 3-yr OS: 56% [95% CI 47-67%]) had a substantially worse OS, suggesting that further refinement of patient-risk defined by IPI may be feasible with HRQoL measures. Conclusions: Our work highlights the potential of the binary HRQoL scales to provide additional prognostic value to IPI, and suggests that patient reported outcomes may be useful in further refining patient risk classification in addition to IPI in untreated DLBCL. [Table: see text]

The one-minute sit-to-stand-test performance is associated with health-related quality of life in patients with pulmonary hypertension.

Patients with pulmonary hypertension (PH) have an impaired functional capacity and poor health-related quality of life (HRQoL). The one-minute sit-to-stand test (1-min STST) can be used for the assessment of functional capacity. Our aim was to evaluate the 1-min STST performance and its association with patient-reported HRQoL in patients with PH. We prospectively assessed functional capacity in 98 PH patients (mean age 66 ± 15 years, 55% female) using the 1-min STST. Patients had to stand up and sit down from a chair as many times as possible within one minute. Patients' HRQoL was evaluated with the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) questionnaire, which consists of the three subcategories symptoms, activities and quality of life (QoL). We observed a significant correlation of the 1-min STST performance with all HRQoL subcategories assessed with the CAMPHOR questionnaire: A lower number of 1-min STST repetitions correlated with more symptoms (rs = -.398, p < .001), worse functioning (rs = -.551, p < .001) and a decreased QoL (rs = -.407, p < .001). Furthermore, in the multivariable linear regression analysis, adjusted for age, sex, body mass index (BMI) and mean pulmonary artery pressure (mPAP), lower 1-min STST performance was an independent predictor for worse symptoms (est. β = -0.112, p = .003), activities (est. β = -0.198, p < .001) and QoL (est. β = -0.130, p < .001) assessed with the CAMPHOR questionnaire. Our results indicate that regardless of age, sex, BMI and mPAP the 1-min STST performance is associated with all CAMPHOR HRQoL subcategories in patients with PH. Therefore, the 1-min STST performance might be a new option to assess functional capacity correlated to HRQoL in patients with PH.

Protocol for the nationwide registry of patients with polycystic kidney disease: japanese national registry of PKD (JRP).

Autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD) are major genetic polycystic kidney diseases that can progress to end-stage kidney disease (ESKD). Longitudinal data on the clinical characteristics associated with clinical outcomes in polycystic kidney disease (PKD), including the development of ESKD and cardiovascular disease (CVD) are lacking in Japan. To address this unmet need the authors are establishing a novel, web-based, Nationwide Cohort Registry Study-the Japanese Registry of PKD (JRP). The JRP is a prospective cohort study for ADPKD (aim to recruit n = 1000 patients), and both a retrospective and prospective study for ARPKD (aim to recruit n = 100). In the prospective registry, patients will be followed-up for 10years every 6months and 12months for patients with ADPKD and ARPKD, respectively. Data collection will be recorded on Research Electronic Data Capture (REDCap) starting on April 1, 2024, with recruitment ending on March 31, 2029. (jRCT 1030230618). Data to be collected include: baseline data, demographics, diagnostic and genetic information, radiological and laboratory findings, and therapeutic interventions. During follow-up, clinical events such as development of ESKD, hospitalization, occurrence of extra kidney complications including CVD events, and death will be recorded, as well as patient-reported health-related quality of life for patients with ADPKD. The JRP is the first nationwide registry study for patients with ADPKD and ARPKD in Japan, providing researchers with opportunities to advance knowledge and treatments for ADPKD and ARPKD, and to inform disease management and future clinical practice.

Garadacimab for hereditary angioedema attack prevention: long-term efficacy, quality of life, and safety data from a phase 2, randomised, open-label extension study

Garadacimab is a fully human immunoglobulin G4 monoclonal antibody targeting activated factor XII. This study evaluated long-term efficacy, health-related quality of life (HRQoL), and safety data for garadacimab in adults with hereditary angioedema. This global phase 2 study comprised a treatment period 1 (TP1: 12 weeks, double-blind, placebo-controlled) and a treatment period 2 (TP2: ≥44-week open-label extension). Patients aged 18-65 years with clinically confirmed hereditary angioedema were eligible. In TP1, 32 patients were randomly assigned (1:1:1:1) to receive subcutaneous garadacimab (75 mg, 200 mg, or 600 mg) or placebo every 4 weeks (once monthly). Randomisation was done using interactive response technology via block randomisation (block sizes 1-4). Subsequently, six additional patients in TP1 were assigned to open-label garadacimab 400 mg every 2 weeks. At the start of TP2, patients were re-randomised (if receiving placebo, garadacimab 75 mg, or garadacimab 400 mg) or continued to receive garadacimab 200 mg or garadacimab 600 mg once monthly. After a protocol amendment on March 20, 2020, patients originally assigned to the 600 mg dose were down-titrated to 200 mg at their next visit. The primary endpoint (published previously) was monthly attack rate for patients receiving 200 mg or 600 mg garadacimab in TP1 in the intention-to-treat population. Here, we assessed the impact of garadacimab on patient-reported and investigator-reported outcomes and HRQoL as well as long-term efficacy and safety. This trial is registered with ClinicalTrials.gov, NCT03712228, and is completed. Of 54 patients screened between Oct 29, 2018, and Aug 28, 2019, 32 randomised and six open-label patients completed TP1 and entered TP2 (20 in the garadacimab 200 mg group; 18 in the garadacimab 600 mg group; total 38 patients). Median age was 39·0 years (IQR 27·0-53·0), and 21 patients (55%) were female and 17 (45%) were male. In TP2, the median garadacimab exposure was 87·9 weeks (IQR 50·0-106·6) in the garadacimab 200 mg group and 44·1 weeks (24·1-56·1) in the garadacimab 600 mg group. Median monthly attack rates were 0·0 (IQR 0·0-0·1) in the garadacimab 200 mg group and 0·1 (0·0-0·4) in the garadacimb 600 mg group. Median reduction in monthly attack rate versus run-in was 100% (IQR 98-100) with garadacimab 200 mg. HRQoL improvements observed during TP1 with garadacimab were sustained throughout TP2. TP2 safety signals were consistent with TP1. Two patients experienced serious adverse events of diverticular perforation and asthma (not garadacimab-related). Treatment-emergent adverse events were mostly mild or moderate in severity. The most common adverse events were headache (nine of 38, 24%) and abdominal pain (seven of 38, 18%). There were no treatment-related deaths. Once-monthly garadacimab for more than 2 years in patients with hereditary angioedema was well tolerated and efficacious in reducing monthly attack rate and improving HRQoL. These results reveal the potential of long-term prophylactic treatment with 200 mg once-monthly garadacimab towards complete disease control of patients with hereditary angioedema. CSL Behring.

Abstract PS02-09: Nurse-led individualized follow-up versus regular physician-led visits after early breast cancer (MyHealth) - a randomized, controlled trial

Abstract Background: Follow-up after breast cancer with regular specialist-led visits has failed to prove superiority over other strategies in terms of recurrence detection and survival, but it is still a cornerstone in breast cancer follow-up in many healthcare systems. Follow-up provided by the general practitioner or a specialist nurse has been reported more cost-effective and non-inferior in terms of patient-reported health-related quality of life (HRQoL) and in meeting the needs of breast cancer survivors. However, no follow-up strategies have demonstrated indisputable superiority over other strategies. Methods: In this randomized controlled trial (MyHealth), patients who recently completed surgery, and chemo-/radiotherapy if indicated, for stage I–II breast cancer were randomly assigned to intervention or control follow-up at Zealand University Hospital, Denmark. The nurse-led intervention comprised three to five individual self-management sessions during the first six months, and regular reporting of symptoms with nurse navigation to relevant healthcare services during three years of follow-up. The control comprised outpatient visits with oncologist every six months for three years. The primary outcome was breast cancer-specific HRQoL measured by the Trial Outcome Index-Physical/Functional/Breast (TOI-PFB) summary score of the Functional Assessment of Cancer Therapy-Breast (FACT-B) two years after randomization. Secondary outcomes were fear of recurrence, anxiety, depression, and healthcare utilization. Results: From January 2017 to January 2019, 503 patients were randomly assigned to intervention (n=251) or control (n=252) follow-up. At two years, patients in the intervention group reported a significantly higher HRQoL (mean 75·69 (SD 12·27)) than patients in the control group (mean 71·26 (SD 14·08)), a mean difference of 5·05 (95% CI; 3·30–6·79), which is considered clinically relevant. The intervention group also reported significantly less fear of recurrence, anxiety, and depression as shown in Table 1. Furthermore, patients in the intervention group had fewer physician consultations but more nurse contacts, and an unchanged diagnostic imaging pattern as shown in Table 2. The effect on all outcomes was stable through three years of follow up. Conclusions: The MyHealth study suggests a new strategy for follow-up after early breast cancer providing significant improvements in HRQoL, fear of recurrence, anxiety, and depression without inflicting extra expenses to the healthcare system. (Funded by the Danish Cancer Society, Region Zealand and Copenhagen University Hospital; Clinicaltrials.gov number; NCT02949167.) Table 1: Outcome findings by study group at baseline, 6, 12, 24, and 36 months Data are mean (SD) unless otherwise stated. The effect estimates are based on the mixed model assuming no difference between groups at baseline. The QoL and fear of recurrence variables are continuous, and the estimates describe the mean difference in score between patients in the two groups. For anxiety and depression, data were zero-inflated. These estimates describe the exponential coefficients of anxiety and depression scores in the intervention group compared to the control group. Cohen’s d is a measure of effect size, and interpreted as small for Cohen’s d = 0·2, medium for Cohen’s d = 0·5, and large for Cohen’s d = 0·8 Table 2: Number of contacts and diagnostic imaging examinations during the first three years of follow-up by study group Data are numbers and mean (SD) per patient during the first three years of follow-up. 1 Most telephone consultations were scheduled as outpatient visits according to the protocol, but changed to telephone consultations due to the COVID-19 pandemic Citation Format: Lena Saltbæk, Pernille Bidstrup, Randi Karlsen, Beverley Høeg, Trine Horsboel, Federica Belmonte, Elisabeth Andersen, Vibeke Zoffmann, Anne Friberg, Mads Svendsen, Helle Christensen, Vesna Glavicic, Dorte Nielsen, Susanne Dalton, Christoffer Johansen. Nurse-led individualized follow-up versus regular physician-led visits after early breast cancer (MyHealth) - a randomized, controlled trial [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PS02-09.

Correlation between disease activity and patient-reported health-related quality of life in rheumatoid arthritis: a cross-sectional study

ObjectiveWe aimed to provide a comprehensive assessment of health-related quality of life (HRQoL) in patients with rheumatoid arthritis (RA) of different activities and to evaluate the correlation between clinical activity...

Associations between health-related quality of life and subsequent need for specialized palliative care and hospital utilization in patients with gastrointestinal cancer—a prospective single-center cohort study

BackgroundWe lack knowledge of which factors are associated with the risk of developing complex palliative care needs. The aim of this study was to investigate the associations between patient-reported health-related quality of life and subsequent referral to specialized palliative care (SPC) and hospital utilization.MethodsThis was a prospective single-center cohort study. Data on patient-reported outcomes were collected through the European Organization of Research and Treatment of Cancer Questionnaire-Core-15-Palliative Care (EORTC QLQ-C15-PAL) at the time of diagnosis. Covariates and hospital utilization outcomes were collected from medical records. Adjusted logistic and Poisson regression were applied in the analyses. Participants were newly diagnosed with incurable gastrointestinal cancer and affiliated with a palliative care case management intervention established in a gastroenterology department.ResultsOut of 397 patients with incurable gastrointestinal cancer, 170 were included in the study. Patients newly diagnosed with incurable gastrointestinal cancer experienced a substantial burden of symptoms. Pain was significantly associated with subsequent referral to SPC (OR 1.015; 95% CI 1.001–1.029). Patients with lower education levels (OR 0.210; 95% CI 0.056–0.778) and a Charlson Comorbidity Index score of 2 or more (OR 0.173; 95% CI 0.041–0.733) were less likely to be referred to SPC. Pain (IRR 1.011; 95% CI 1.005–1.018), constipation (IRR 1.009; 95% CI 1.004–1.015), and impaired overall quality of life (IRR 0.991; 95% CI 0.983–0.999) were significantly associated with increased risk of hospital admissions.ConclusionThe study indicates a need for interventions in hospital departments to identify and manage the substantial symptom burden experienced by patients, provide palliative care, and ensure timely referral to SPC.

Exploring Quality of Life and Mortality in Pertrochanteric Fragility Hip Fractures in Northern Greece: A Single Tertiary Center Study.

Background: Fragility-related pertrochanteric fractures have become a significant public health concern, with a rising incidence attributed to the expanding elderly demographic. Assessing patient-reported health-related quality of life (HRQoL), mortality, and factors correlated with them serves as a crucial metric in evaluating the effectiveness of hip fracture surgery. Methods: In a single-center retrospective study, 259 patients underwent surgical treatment with a cephalomedullary nail, with a mean follow-up of 21.7 months. Health-related quality of life (HRQoL) was assessed using SF-12 (12-item Short Form) and EQ-5D (EuroQoL-5 Dimensions) questionnaires. Mobility status was measured by the Crude Mobility Index (CMI). Surveys were administered during hospitalization and six months postoperatively. Statistical analysis involved descriptive statistics, non-parametric controls (Kendall, Mann-Whitney, and Wilcoxon), and Spearman correlation and logistic regression analysis, which were conducted using IBM SPSS version 28. Results: A statistically significant decrease was observed in the mean EQ-5D and SF-12 scores at 6 months post-op compared to the pre-fracture status. The ASA (American Society of Anaesthesiologists) score showed a significant correlation with the decrease in HRQoL measured by the SF-12 questionnaire. The 30-day post-operative mortality rate was 9.3%, increasing to 32.4% at 1 year. Notably, the 30-day mortality significantly rose during the pandemic era (5.0% vs. 12.0%; p = 0.003). Conclusions: Pertrochanteric hip fractures cause a lasting decline in quality of life. Annual mortality is high, and further investigations are needed to formulate policies that prevent hip fractures and reduce mortality rates.

Patient-reported outcomes after personalised dose-escalation for stage II-III non-small-cell lung cancer patients: Results from the randomised ARTFORCE PET-Boost trial

Background and purposeThe ultimate challenge in dose-escalation trials lies in finding the balance between benefit and toxicity. We examined patient-reported outcomes (PROs), including health-related quality of life (HRQoL) in patients with locally advanced non-small cell lung cancer (LA-NSCLC), treated with dose-escalated radiotherapy. Materials and methodsThe international, randomised, phase 2 ARTFORCE PET-Boost study (NCT01024829) aimed to improve 1-year freedom from local failure rates in patients with stage II-III NSCLC, with a ≥ 4 cm primary tumour. Treatment consisted of an individualised, escalated fraction dose, either to the primary tumour as a whole or to its most FDG-avid subvolume (24 x 3.0–5.4 Gy). Patients received sequential or concurrent chemoradiotherapy, or radiotherapy only. Patients were asked to complete the EORTC QLQ-C30, QLQ-LC13, and the EuroQol-5D at eight timepoints. We assessed the effect of dose-escalation on C30 sum score through mixed-modelling and evaluated clinically meaningful changes for all outcomes. ResultsBetween Apr-2010 and Sep-2017, 107 patients were randomised; 102 were included in the current analysis. Compliance rates: baseline 86.3%, 3-months 85.3%, 12-months 80.3%; lowest during radiation treatment 35.0%. A linear mixed-effect (LME) model revealed no significant change in overall HRQoL over time, and no significant difference between the two treatment groups. Physical functioning showed a gradual decline in both groups during treatment and at 18-months follow-up, while clinically meaningful worsening of dyspnoea was seen mainly at 3- and 6-months. ConclusionIn patients with LA-NSCLC treated with two dose-escalation strategies, the average patient-reported HRQoL remained stable in both groups, despite frequent patient-reported symptoms, including dyspnoea, dysphagia, and fatigue.

Physician Empathy and Chronic Pain Outcomes

Empathy is an aspect of the patient-physician relationship that may be particularly important in patients with chronic pain. To measure the association of physician empathy with pain, function, and health-related quality of life (HRQOL) among patients with chronic low back pain. This cohort study included adult enrollees from the Pain Registry for Epidemiological, Clinical, and Interventional Studies and Innovation national pain research registry. Study dates were from April 1, 2016, to July 25, 2023, with up to 12 months of follow-up. Physician empathy was assessed with the Consultation and Relational Empathy measure and dichotomized to yield very empathic physician and slightly empathic physician groups. Main outcomes were patient-reported pain, function, and HRQOL measured with a numerical rating scale for low back pain intensity, the Roland-Morris Disability Questionnaire for back-related disability, and the Patient-Reported Outcomes Measurement Information System for HRQOL deficits pertaining to anxiety, depression, fatigue, sleep disturbance, and pain interference. Data were collected at 5 quarterly encounters from registry enrollment through 12 months and analyzed with generalized estimating equations, including multivariable models to measure temporal trends and to adjust for baseline and longitudinal covariates. Among the 1470 patients, the mean (SD) age was 53.1 (13.2) years, and 1093 (74.4%) were female. Patients completed 5943 encounters in which multivariable analyses demonstrated that greater physician empathy was inversely associated with pain intensity (β = -0.014; 95% CI, -0.022 to -0.006; P < .001), back-related disability (β = -0.062; 95% CI, -0.085 to -0.040; P < .001), and HRQOL deficits on each measure (eg, pain interference: β = -0.080; 95% CI, -0.111 to -0.049; P < .001). Correspondingly, compared with the slightly empathic physician group, the very empathic physician group reported lower mean pain intensity (6.3; 95% CI, 6.1-6.5 vs 6.7; 95% CI, 6.5-6.9; P < .001), less mean back-related disability (14.9; 95% CI, 14.2-15.6 vs 16.8; 95% CI, 16.0-17.6; P < .001), and fewer HRQOL deficits on each measure (eg, fatigue: 57.3; 95% CI, 56.1-58.5 vs 60.4; 95% CI, 59.0-61.7; P < .001). All physician empathy group differences were clinically relevant, with Cohen d statistics ranging from 0.21 for pain intensity to 0.30 for back-related disability, fatigue, and pain interference. Physician empathy was associated with more favorable outcomes than non-pharmacological treatments, opioid therapy, and lumbar spine surgery. In this cohort study of adult patients with chronic pain, physician empathy was associated with better outcomes over 12 months. Greater efforts to cultivate and improve physician empathy appear warranted.