Health-related Quality Of Life Outcomes Research Articles

Impact of Preoperative LUTS on Health-related Quality of Life Following Radical Prostatectomy: A Propensity Score Matched Longitudinal Study

To assess the impact of preoperative lower urinary tract symptoms (LUTS) on long-term health-related quality of life (HRQOL) up to 10 years after radical prostatectomy (RP) for prostate cancer (PC). Within our prospective institutional database of 6487 patients treated with RP for PC (2008-2020), 2727 patients with preoperative LUTS (IPSS score of ≥8) were identified. A 1:1 propensity-score matched analysis of 3056 men (n=1528 LUTS, n=1528 no LUTS) was conducted. Primary endpoint was HRQOL (based on EORTC QLQ-C30 and PR25). Linear regression models tested the effect of preoperative LUTS on the net change in general HRQOL (P<.05). Median follow-up was 48 months. Preoperative mean global health status (GHS) score (67.4 vs75.7) was significantly lower in the LUTS cohort (P<.001). Post-RP the difference in general HRQOL between the LUTS cohort and the no-LUTS cohort became smaller (65.7 vs67.8), however, remaining statistically significant (P=.037). In long-term follow-up, general HRQOL was comparable between both subcohorts (P-range 0.716-0.876). Multivariable linear regression analysis revealed increased preoperative IPSS as an independent predictor for increased perioperative improvement of IPSS (P<.001) CONCLUSION: For patients undergoing RP, preoperative LUTS were associated with a postoperative improvement of HRQOL outcomes. In long-term follow-up, HRQOL was comparable to patients without preoperative LUTS. Hence, RP is an efficient option to treat PC as well as LUTS in those patients.

Extrarenal symptoms associate with worse quality of life in patients enrolled in the AMP RA/SLE Lupus Nephritis Network.

Lupus nephritis (LN) can occur as an isolated component of disease activity or be accompanied by diverse extrarenal manifestations. Whether isolated renal disease is sufficient to decrease health related quality of life (HRQOL) remains unknown. This study compared Patient-Reported Outcomes Measurement Information System 29-Item (PROMIS-29) scores in LN patients with isolated renal disease to those with extrarenal symptoms to evaluate the burden of LN on HRQOL and inform future LN clinical trials incorporating HRQOL outcomes. A total of 181 LN patients consecutively enrolled in the multicentre multi-ethnic/racial Accelerating Medicines Partnership completed PROMIS-29 questionnaires at the time of a clinically indicated renal biopsy. Raw PROMIS-29 scores were converted to standardized T scores. Seventy-five (41%) patients had extrarenal disease (mean age 34, 85% female) and 106 (59%) had isolated renal (mean age 36, 82% female). Rash (45%), arthritis (40%) and alopecia (40%) were the most common extrarenal manifestations. Compared with isolated renal, patients with extrarenal disease reported significantly worse pain interference, ability to participate in social roles, physical function, and fatigue. Patients with extrarenal disease had PROMIS-29 scores that significantly differed from the general population by > 0.5 SD of the reference mean in pain interference, physical function, and fatigue. Arthritis was most strongly associated with worse scores in these three domains. Most patients had isolated renal disease and extrarenal manifestations associated with worse HRQOL. These data highlight the importance of comprehensive disease management strategies that address both renal and extrarenal manifestations to improve overall patient outcomes.

Pain and Health-Related Quality of Life in Autosomal Dominant Polycystic Kidney Disease: Results from a National Patient-Powered Registry

Rationale and ObjectiveADPKD impacts health-related quality of life (HRQoL) including pain, discomfort, fatigue, emotional distress, and impaired mobility. Stakeholders prioritized kidney cyst-related pain as an important core outcome domain in clinical trials, leading to development of disease-specific assessment tools. Study DesignThe ADPKD Registry is hosted online with multiple disease-specific patient reported outcomes modules to characterize the patient experience in the United States. Setting and ParticipantsThe ADPKD Registry allows consented participants access to a Core Questionnaire which includes demographics, comorbid conditions, current symptoms, and kidney function. Participants complete subsequent modules on a 3-month schedule, including two validated HRQoL tools, the ADPKD Pain and Discomfort Scale (ADPKD-PDS), the ADPKD Impact Scale (ADPKD-IS) and a Healthcare Access and Utilization module. ExposuresPatient-reported latest eGFR or creatinine used to calculate stage of chronic kidney disease. OutcomesHealth-related quality of life, measured using validated ADPKD-specific tools; access to PKD-specific healthcare. Analytical ApproachFor the two HRQoL tools, scores were calculated for physical, emotional, and fatigue domains, pain severity, and pain interference (based on the licensed user manuals). Associations to health care access were also assessed. ResultsBy July 2022, 1,086 individuals with ADPKD completed at least one of the HRQoL modules, 319 completed four over a year. Participants were an average age of 53, 71% women and 91% white, with all CKD stages represented. 2.5% reported being on dialysis and 23% had a kidney transplant. CKD Stage 4/5 participants reported the most dull kidney pain, while sharp kidney pain was evenly distributed across early CKD stages. Dull kidney pain had an impact on sleep regardless of CKD stage. There was a strong positive correlation between the ADPKD-PDS and ADPKD-IS. Patients with a neutral or positive HRQoL were less likely to have been denied access to imaging or other care. LimitationsCurrently all the information collected is patient-reported without health record validation of clinical variables. ConclusionsUse of the HRQoL tools in the ADPKD Registry provided a broad cross-sectional assessment in the U.S. and provided granular information on the burden of pain across the CKD spectrum in ADPKD. The ADPKD Registry allowed assessment of ADPKD impact in a community that experiences decline in health and kidney function over decades.

Abstract 4834: Racial disparities in pain and psychiatric medication use in patients with metastatic colorectal cancer

Abstract Background: In the United States, colorectal cancer (CRC) is a public health concern, as it is the second leading cause of cancer-related death, accounting for over 50,000 deaths annually. In addition to its high mortality, CRC is associated with significant comorbidities including pain and psychiatric symptoms, particularly in late-stage disease. Supportive care medications (SCM) are frequently used for symptom management and improving health-related Quality of Life (HRQoL). Racial disparities in clinical outcomes including HRQoL have been reported in CRC. While racial disparities have been described in the use of SCM for some cancer types, no investigation of SCM use in late-stage CRC have been performed. The purpose of our study was to explore the relationship between SCM use and race/ethnicity in patients with metastatic CRC. Methods: We used the National Cancer Institute Surveillance Epidemiology and End Results (NCI SEER)- Medicare-linked database to analyze all patients with a CRC diagnosis and a newly initiated outpatient prescription claim for an SCM of interest from 2009-2018. Patients on SCM before cancer diagnosis or who had non-metastatic CRC at diagnosis were excluded. We assessed six SCM classes: opioids, non-opioid analgesics, skeletal muscle relaxants, antidepressants, anxiolytics, and antipsychotics. Results: After application of inclusion/exclusion criteria, 28, 212 patients were included in the final analysis (White: 72.8%, Black: 12.7%, Hispanic: 9.5%, Asian: 4.6%, Native American: 0.4%). After controlling for socioeconomic factors and compared to non-Hispanic White patients, there were significant differences across multiple SCM drug classes. When considering pain medications, these differences were bi-directional. Black patients were less likely to receive opioids (adjusted Odds Ratio [aOR] 0.86, 95% CI: 0.80-0.93), while Hispanic patients were more likely to receive opioids (aOR: 1.12, 95% CI: 1.03-1.22) and non-opioid analgesics (aOR 1.22, 95% CI: 1.06-1.40). Asian patients were more likely to receive non-opioid analgesics (aOR 1.71, 95% CI: 1.45-2.03), but less likely to receive skeletal muscle relaxants (aOR 0.60, 95% CI: 0.43-0.82). When considering psychiatric medications, decreased use among all racial groups was noted for antidepressants (Black: aOR 0.53, 95% CI: 0.50-0.60; Hispanic: aOR 0.82, 95% CI: 0.75-0.90; Asian: aOR 0.48, 95% CI: 0.42-0.56), and anxiolytics (Black: aOR 0.43, 95% CI: 0.38-0.49; Hispanic: aOR 0.76, 95% CI: 0.67-0.85; Asian: aOR 0.45, 95% CI: 0.37-0.55). Among antipsychotics, only Asian patients had less use compared to non-Hispanic White patients. Conclusion: Racial disparities in the use of pain and psychiatric medications among patients with late-stage CRC were present, even after controlling for multiple confounders. Further investigation is needed to understand drivers and their impact on HRQoL outcomes. Citation Format: John M. Allen, Olga M. Trejos Kweyete, Yi Guo, Sherise C. Rogers, Lisa Scarton, David L. DeRemer, Chardae Whitner, Diana J. Wilkie. Racial disparities in pain and psychiatric medication use in patients with metastatic colorectal cancer [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 4834.

The Assessment of SF-36 Survey for Quality-of-Life Measurement after Radical Cystectomy for Muscle-Invasive Bladder Cancer: A Systematic Review.

This study presents a systematic review of the literature on individuals' health-related quality of life (HRQoL) following radical cystectomy for muscle-invasive bladder cancer (MIBC), utilizing the Short Form-36 Health Survey (SF-36) as a primary assessment tool. The review was designed as an exhaustive literature search across three major databases including PubMed, Scopus, and Embase up to December 2023, using the PRISMA guidelines. The selection process refined 2281 identified articles down to 11 studies meeting our inclusion criteria. These studies encompassed a diverse demographic and clinical profile of 774 participants, with follow-up durations ranging from 3 to 130 months, thereby offering insights into both short-term and long-term HRQoL outcomes. The results highlighted significant alterations in individuals' HRQoL across various domains post-radical cystectomy. Notably, the Physical Functioning (PF) and Bodily Pain (BP) domains generally scored higher, indicating a moderate to high perceived physical health status. However, the Role Physical (RP) and Role Emotional (RE) domains showed variability, reflecting the challenges in daily role fulfillment and emotional adjustment post-surgery. A marked variability in physical recovery was observed, with studies reporting significant differences in PF and RP scores between patient groups. The General Health (GH) and Vitality (VT) domains sometimes reflected perceived deteriorations, whereas the Mental Health (MH) scores suggested that many patients maintained or achieved high levels of well-being post-operatively. The conclusions drawn from this systematic review underscore the profound and multi-faceted impact of radical cystectomy on HRQoL, varying widely between studies, being influenced by geographic factors, surgical methods, and the time of evaluation. The findings emphasize the necessity for holistic patient care approaches that address both physical and emotional rehabilitation, aiming to improve HRQoL outcomes.

Health literacy mediates the relationships of cognitive and physical functions with health-related quality of life in older adults.

Declining cognitive function (CF) and physical function (PF) relate to poorer health-related quality of life (HRQoL) in older adults. As health literacy (HL) facilitates health information utilization, it may mediate links between functionality and HRQoL appraisals. This study examined HL as an intermediary between joint CF and PF contributions and HRQoL in Hong Kong older adults. 490 older adults aged 50-80 years completed assessments from March to July 2021. Health Literacy Survey Questionnaire Short Form 12 questions (HLS-SF12), Montreal Cognitive Assessment (MoCA), Senior Fitness Test (SFT) and 12-Item Short-Form Health Survey version 2 (SF-12v2) were used to assess HL, CF, PF and HRQoL, respectively. Path analysis tested a model with HL mediating CF/PF predictors and HRQoL outcome. Results for direct effects indicated that CF significantly associated with PF (β = 0.115, SE = 0.012, p < 0.001), PF significantly connected to HL (β = 0.101, SE = 0.022, p < 0.001), and HL significantly related to HRQoL (β = 0.457, SE = 0.049, p < 0.001). Meanwhile, PF significantly linked to HRQoL directly (β = 0.156, SE = 0.025, p < 0.001) as well as indirectly (β = 0.046, 95% CI [0.028, 0.067]). Significant mediating effect of HL was found on the relationship of PF and HRQoL. Findings confirm CF and PF joint impacts on HL and HRQoL in older adults, elucidating HL's mediating role in translating functionality levels into HRQoL. Fostering enduring health knowledge access may thus buffer effects of age-related declines on well-being. Results can inform interventions leveraging this pathway to promote resilient trajectories.

Use of the Patient Generated Index to Identify Physical Health Challenges among People Living with HIV: A Cross-sectional Study

Many people living with HIV experience physical health challenges including mobility problems, pain, and fatigue. Purpose: To estimate how many people living with HIV identify physical health challenges as important using the patient generated index (PGI). Secondary Objectives: a. Identify factors associated with reporting physical health challenges; b. Identify relationships between reporting physical health challenges and standardized health-related quality of life (HRQOL) items; and c. Estimate the extent to which reporting a physical health challenge explains downstream HRQOL outcomes. Methods: Cross-sectional data came from a large Canadian cohort. We administered the PGI and three standardized HRQOL measures. PGI text threads were coded according to the World Health Organization's International Classification of Functioning, Disability, and Health. Regression, discriminant analysis, and chi-square tests were used. Results: Of 865 participants, 248 [28.7%; 95% CI (25.7%, 31.8%)] reported a physical health challenge on the PGI. Participants with better pain (OR: 0.81, 95% CI: 0.71, 0.90) and vitality (OR: 0.71, 95%CI: 0.63, 0.80) by 20 points had lower odds of reporting a physical health challenge. Those who reported a physical health challenge had significantly lower HRQOL on some standardized items. Conclusion: The PGI is well-suited to identify the physical challenges of people living with HIV.

First knee for pain and function versus second knee for quality of life.

The aim of this study was to describe and compare joint-specific and generic health-related quality of life outcomes of the first versus second knee in patients undergoing staged bilateral total knee arthroplasty (BTKA) for osteoarthritis. This retrospective cohort study used Australian national arthroplasty registry data from January 2013 to January 2021 to identify participants who underwent elective staged BTKA with six to 24 months between procedures. The primary outcome was Oxford Knee Score (OKS) at six months postoperatively for the first TKA compared to the second TKA, adjusted for age and sex. Secondary outcomes compared six-month EuroQol five-dimension five-level (EQ-5D-5L) domain scores, EQ-5D index scores, and the EQ visual analogue scale (EQ-VAS) between knees at six months postoperatively. The cohort included 635 participants (1,270 primary procedures). Preoperative scores were worse in the first knee compared to the second for all instruments; however, comparing the first knee at six months postoperatively with the second knee at six months postoperatively, the mean between-knee difference was minimal for OKS (-0.8 points; 95% confidence interval (CI) -1.4 to -0.2), EQ-VAS (3.3; 95% CI 1.9 to 4.7), and EQ-5D index (0.09 points; 95% CI 0.07 to 0.12). Outcomes for the EQ-5D-5L domains 'mobility', 'usual activities', and 'pain/discomfort' were better following the second TKA. At six months postoperatively, there were no clinically meaningful differences between the first and second TKA in either the joint-specific or overall generic health-related quality of life outcomes. However, individual domain scores assessing mobility, pain, and usual activities were notably higher after the second TKA, likely reflecting the cumulative improvement in quality of life after both knees have been replaced.

Cost-utility analysis and impact on the environment of videoconference in pressure injury. A randomized controlled trial in individuals with spinal cord injury

Study designA prospective randomized controlled trial (RCT) in persons with spinal cord injury (SCI) and ongoing pressure injury (PI).ObjectivesThe main aim was to perform a cost-utility analysis (CUA) alongside the RCT comparing regular care to regular care with additional videoconference consultations. Secondary aims were to assess costs and greenhouse gas emission related to transportation in the two study groups.SettingTwo spinal cord units in Norway.MethodsParticipants were allocated to a regular care group (RCG) and a regular care group with additional videoconference (VCG), in a 1-year follow-up between 2016 and 2018. Costs were prospectively collected, and health-related quality of life (HRQoL) data were collected at baseline and 12 months. The outcome was quality-adjusted life years (QALYs), derived from the EQ-5D-5L questionnaire. Results are reported as incremental cost-effectiveness ratio (ICER), expressed as the cost per additional QALY gained. Transportation related costs and environmental emissions were compared by t-tests.ResultsThere were 56 participants included, 28 in each group. Of these 27 in the VCG and 26 in the RCG completed. Three participants died. The mean cost per patient was € 8819 in the VCG and € 3607 in the RCG, with 0.1 QALYs gained in the VCG. No significant differences were identified regarding HRQoL or secondary outcomes.ConclusionThe VCG costs € 5212 more for an additional 0.1 QALYs, giving an ICER of € 52,120 per QALY. No significant differences were found regarding transportation-related costs, or emission of greenhouse gases.Trial registrationwww.ClinicalTrials.gov; NCT02800915, TeleSCIpi. CRISTIN.no. https://app.cristin.no/projects/show.jsf?id=545284. Sunnaas Rehabilitation hospital’s web page, available at https://www.sunnaas.no/fag-og-forskning/fagstoff/sar.

Health-related quality of life in patients with hematologic malignancies treated with chimeric antigen receptor T-cell therapy: review and current progress.

Chimeric antigen receptor (CAR) T-cell therapy has transformed the care of patients with relapsed/refractory B-cell-derived hematologic malignancies. To date, six CAR T-cell therapies, targeting either CD19 or B-cell maturation antigen, have received regulatory approval. Along with the promising survival benefit, CAR T-cell therapy is associated with potentially life-threatening adverse events, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. While clinical trials evaluating CAR T-cell therapy consistently report the incidence of these adverse events, most trials do not collect health-related quality of life (HRQoL) data. As such, the impact of the CAR T-cell therapy process and related adverse events on the physical and psychological well-being of patients remains uncertain. HRQoL and other patient-reported outcome (PRO) assessments in patients with relapsed or refractory hematologic malignancies are of utmost importance, as individuals may have unmet needs and a high demand for tolerable therapy if a cure is not obtained. In addition, it is important to standardize methods of data collection to better assess the impact of CAR T-cell therapy on quality of life, optimize patients' care and costs, and enable comparisons between different studies. We conducted a literature search up to June 2023 to identify the HRQoL tools used in clinical trials and in real-world studies investigating CAR T-cell therapy in patients with lymphomas or leukemias. In the present comprehensive review, we summarize the most commonly used CAR T-cell specific and non-specific HRQoL tools and discuss how the use of HRQoL and other PRO tools may be optimized.

Comparative Clinical Effectiveness and Cost-Effectiveness of the Cochlear Osia System and Baha Attract System in Patients with Conductive or Mixed Hearing Loss or Single-Sided Deafness.

The aim of this study was to evaluate the comparative clinical effectiveness and cost-utility of the active transcutaneous Osia® System versus the passive transcutaneous Baha® Attract System for patients with conductive or mixed hearing loss or single-sided deafness in an Australian healthcare setting. In the absence of direct comparative evidence, an indirect treatment comparison (ITC) of the clinical effectiveness and utility gains was needed. The ITC was informed by three studies identified through a systematic literature review. A Markov model was developed to evaluate the cost-utility of the Osia System. The literature review identified three studies suitable to inform an ITC: Mylanus et al. 2020 and Briggs et al. 2022 (Osia System) and den Besten et al. 2019 (Baha Attract System). The Osia System was found to be clinically superior to the Baha Attract System, across objective audiological outcomes resulting in a clinically meaningful utility benefit of 0.03 measured by the Health Utility Index with at least equivalent safety. In conclusion, the Osia System is more effective than the Baha Attract System, providing better hearing and health-related quality of life outcomes. In an Australian healthcare setting, the Osia System is cost-effective as demonstrated in a cost-utility analysis versus the Baha Attract System.

Health-related quality of life following hysterectomy by vNOTES versus conventional laparoscopy

Objective To evaluate safety, efficacy, pre- and postoperative outcomes of vNOTES hysterectomy compared to conventional laparoscopic (CL) hysterectomy. Material and methods This prospective study was conducted on patients who underwent CL or vNOTES hysterectomy for benign gynecological indications between January and July 2021. Sociodemographic, clinical, and surgical data were collected. Health-related quality of life (HRQoL) outcomes were evaluated using the EQ-5D-5L questionnaire. Results A total of 228 patients (CL vs. vNOTES: 147 vs. 81) were included. No significant differences were observed between the two groups in terms of blood loss, uterine weight, complications, length of hospital stay, and readmission rate. Patients in the vNOTES group experienced significantly less postoperative pain and required less analgesia compared to those who underwent CL hysterectomy (p < 0.001). The CL group had a shorter operative time (p < 0.001). There was a significant difference between the two groups in the EQ-5D-5L pain/discomfort dimension (p = 0.047). Conclusion This research showed that vNOTES has various advantages, such as less postoperative pain, reduced analgesic usage, and better HRQoL outcomes three months after surgery.

Efficacy of a digital lifestyle intervention on health-related QUAlity of life in non-small cell LUng CAncer survivors following inpatient rehabilitation: protocol of the QUALUCA Swiss multicentre randomised controlled trial

IntroductionNon-small cell lung cancer (NSCLC) survivors suffer from impaired physical and psychological functioning and reduced health-related quality of life (HRQoL) that persist after active treatment ends. Sustaining rehabilitation benefits, promoting...

Familism, family cohesion, and health-related quality of life in Hispanic prostate cancer survivors.

Familism, the cultural value that emphasizes feelings of loyalty and dedication to one's family, has been related to both positive and negative outcomes in Hispanic cancer survivors. One potential source of observed inconsistencies may be limited attention to the family environment, as familism may be protective in a cohesive family whereas it can exacerbate distress in a conflictive family. The current study explored the associations of familism with general and disease-specific health-related quality of life (HRQoL) in Hispanic men who completed prostate cancer (PC) treatment, and whether family cohesion may help explain these relationships. Hispanic men treated for localized PC (e.g., radiation, surgery) were enrolled in a randomized controlled stress management trial and assessed prior to randomization. Familism (familial obligation) was assessed using Sabogal's Familism Scale and family cohesion was measured using the Family Environment Scale (ranging from high to low). The sexual, urinary incontinence, and urinary obstructive/irritative domains of the Expanded Prostate Cancer Index Composite - Short Form measured disease-specific HRQoL. The physical, emotional, and functional well-being subscales of the Functional Assessment of Cancer Therapy - General captured general HRQoL. Hierarchical linear regression and the SPSS PROCESS macro were used to conduct moderation analyses, while controlling for relevant covariates. Participants were 202 older men on average 65.7 years of age (SD = 8.0) who had been diagnosed with PC an average of 22 months prior to enrollment. Familism was not directly associated with general and disease-specific HRQoL. Moderation analyses revealed that greater familism was related to poorer urinary functioning in the incontinence (p = .03) and irritative/obstructive domains (p = .01), and lower emotional well-being (p = .02), particularly when family cohesion was low. These findings underscore the importance of considering contextual factors, such as family cohesion, in understanding the influence of familism on general and disease-specific HRQoL among Hispanic PC patients. The combined influence of familism and family cohesion predicts clinically meaningful differences in urinary functioning and emotional well-being during the posttreatment phase. Culturally sensitive psychosocial interventions to boost family cohesion and leverage the positive impact of familistic attitudes are needed to enhance HRQoL outcomes in this population.

The Role of Physical Exercise in the Prevention of Musculoskeletal Disorders in Manual Workers: A Systematic Review and Meta-Analysis.

Work-related musculoskeletal disorders (WMSDs) are the most common occupational health problem in the European Union. Physical exercise interventions have been investigated in the prevention of WMSDs in many sectors. Therefore, our aim was to assess the effect of physical exercise in manual workers for the primary and secondary prevention of WMSDs. We conducted a systematic search of the literature and papers were included if: the participants were adult employees exclusively engaged in manual labor tasks; non-acute physical exercise intervention; pain, disability, physical functioning, or health-related quality of life outcome, with pre-post intervention measurements. We retrieved 10419 unique records and included 23 studies. A random effect meta-analysis was conducted on the studies with a control group design, using a three level model to estimate the pooled effect for pain outcomes (g = 0.4339, 95% CI : 0.1267 - 0.7412, p < 0.01), and a two-level model for disability outcomes (g = 0.6279, 95% CI : 0.3983 - 0.8575, p < 0.0001). Subset analysis revealed a moderate-to-large effect on the VAS outcome (g = 0.5866, 95% CI: 0.3102 - 0.8630, p < 0.0001). Meta-regression on pain outcomes revealed a significant effect for sex, age, study quality, and body segments tested. The analyses on all outcomes except VAS showed substantial heterogeneity (I2pain = 93%, of which 72% at the study level, I2disability = 78%, and I2vas = 56%, of which 44% at the study level). Physical exercise programs seem to have a positive effect on pain and disability stemming from WRMSDs in manual workers.

Tislelizumab versus Sorafenib in First-Line Treatment of Unresectable Hepatocellular Carcinoma: Impact on Health-Related Quality of Life in RATIONALE-301 Study

Introduction: RATIONALE-301 (NCT03412773) was a global, phase 3 study comparing the efficacy and safety of tislelizumab with sorafenib as first-line (1L) treatment in adult patients with unresectable hepatocellular carcinoma (HCC) that met its primary endpoint of noninferiority in overall survival (OS). This analysis compared health-related quality-of-life (HRQOL) outcomes between the arms. Methods: Systemic therapy-naive adults with HCC were randomized 1:1 to receive tislelizumab (n = 342) or sorafenib (n = 332). HRQOL was assessed using EORTC QLQ-C30, QLQ-HCC18, and EQ-5D-5L. At cycles 4 and 6, a mixed model for repeated measures was performed using key-prespecified patient-reported outcome (PRO) endpoints of the QLQ-C30 and the QLQ-HCC18. Time to deterioration was analyzed with the Kaplan-Meier method using the PRO endpoints. Results: At cycles 4 and 6, patients in the tislelizumab arm had better HRQOL outcomes than the patients in the sorafenib arm per mean-change difference in GHS/QOL, QLQ-C30 physical functioning and fatigue, and QLQ-HCC18 symptom index; however, no differences for pain were observed. Patients in the tislelizumab arm had lower risk of deterioration in GHS/QOL (HR: 0.68; 95% CI: 0.49–0.94), QLQ-C30 physical functioning (HR: 0.45; 95% CI: 0.32–0.63) and fatigue (HR: 0.47; 95% CI: 0.36–0.61), QLQ-HCC18 symptom index (HR: 0.52; 95% CI: 0.34–0.81), and HCC-specific fatigue (HR: 0.59; 95% CI: 0.45–0.79). For pain, both arms had similar risk of deterioration (HR: 0.78; 95% CI: 0.56–1.09). At cycles 4 and 6, patients in the tislelizumab arm maintained in EQ-5D-5L visual analog scale, whereas scores decreased for the patients in the sorafenib arm. Conclusion: Patients with 1L HCC treated with tislelizumab had favorable HRQOL outcomes compared with patients treated with sorafenib, particularly in fatigue and physical functioning. These results, along with favorable safety profile, better response rate, and OS noninferiority, support tislelizumab as a potential 1L treatment option for unresectable HCC.

Functional and Health-Related Quality of Life Outcomes into Adulthood for Females Surgically Treated for Anorectal Malformation

BackgroundControlled outcomes into adulthood for females with anorectal malformation (ARM) are still scantily studied. The primary aim was to investigate bowel function, bladder function and health-related quality of life (HRQoL) in females operated for ARM. MethodsA cross-sectional questionnaire-based observational study was performed including females treated for ARM at our institution between 1994 and 2017. The bowel function was assessed with bowel function score (BFS) and urinary tract function with lower urinary tract symptoms (LUTS) questionnaires. HRQoL was investigated with validated age-dependent questionnaires. Patient characteristics were retrospectively retrieved from the medical records and descriptive statistics were used for analysis. HRQoL outcomes were compared with normative data whilst bowel and bladder function outcomes were compared to age-matched female controls. ResultsForty-four (41.5 %) of 106 females responded to the questionnaires. Ten of 29 patients (34.5 %) aged 4–17 years and 4 of 14 patients (28.6%) aged ≥18 years, reported a well-preserved bowel function (BFS≥17). Constipation issues decreased with age. BFS was similar in patients with perineal and vestibular fistulas. Thirty-six (83.7%) of the patients had at least one LUTS. No adult patients had issues with involuntary urinary leakage. Adults scored significantly (p = 0.004) lower than normative data regarding HRQoL, while children and adolescents scored comparably to norm data. ConclusionsOnly 28.6 % of the adult patients reported a well-preserved bowel function, similar to the proportion reported by children 4–17 years of age. Adult patients appear to have a diminished HRQoL, however the correlation with BFS was weak. Level of EvidenceIII.

Assessing health-related quality of life and clinical outcomes of patients with diabetes accessing healthcare in two public hospitals in south-eastern Ghana: a cross-sectional descriptive study.

Diabetes is a significant contributor to kidney failure, heart attack, strokes, lower limb amputation, blindness, and other complications that negatively impact health-related quality of life (HRQOL). This study assessed the HRQOL and clinical outcomes of patients with diabetes accessing healthcare in south-eastern Ghana. This was a hospital-based cross-sectional descriptivestudy conducted in the Ho Municipal and Ho Teaching Hospitals in Ghana among patients with type 2 diabetes who were seeking healthcare at both hospitals for at least 12months. Systematic random sampling technique was used to select 310 respondents out of the total sample size of 326 patients with type 2diabetes and data were collected using diabetes-39 questionnaire. The data were analysed using STATA 16.0. Socio-demographic and clinical variables were expressed as frequencies and percentages. Differences between proportions were tested using Chi-square to identify predictors of poor HRQOL and Pearson correlation for association. The p < 0.05 was considered significant. Out of 310 respondents, 171 (55.0%) had poor HRQOL. The predictors of poor HRQOL were age (p < 0.008), education (p < 0.028), employment (p < 0.001), residence (p < 0.01), duration of diabetes (p < 0.002), diabetes education (p < 0.001), BMI (p < 0.005), and glycaemic control (p < 0.001). Energy and mobility (63.2%), anxiety and worry (53.9%), and diabetes control (49.6%) dimensions were the most prevalent of poor HRQOL. Diabetes education, complications, being diabetic for 16years and above, earning income, resident in rural area, being married, being pensioner and national Service Personnel, and diabetes comorbidities were significantly associated with HRQOL. More than half of the respondents had poor HRQOL. Clinical and public health efforts should focus on effective control and screening measures for the individual patients and general population.

500 - Patient-reported outcomes following 24 weeks of treatment with upadacitinib in adults with non-segmental vitiligo: results from a phase 2, randomized, double-blind, dose-ranging study

Abstract Introduction &amp; Objectives Vitiligo has a considerable negative impact on patients’ health-related quality of life (HRQoL). Patients often experience social stigma, low self-esteem, anxiety, and depression. Assessment of HRQoL is therefore recommended as part of the evaluation of disease severity and treatment benefits. Accordingly, the impacts of treatment with upadacitinib (UPA), an oral Janus kinase inhibitor, on a range of patient-reported HRQoL outcomes were evaluated as part of a phase 2, multicenter, randomized, double-blind, placebo-controlled, dose-ranging study in adults with non-segmental vitiligo. Materials &amp; Methods Adults aged 18–65 years with non-segmental vitiligo with a Facial Vitiligo Area Scoring Index score ≥0.5 and a Total Vitiligo Area Scoring Index score ≥5 at baseline were included in this 52-week study (NCT04927975). During a 24-week, double-blind, placebo-controlled treatment period (period 1), patients were randomly assigned to receive UPA 6 mg (UPA6), UPA 11 mg (UPA11), UPA 22 mg (UPA22), or placebo once daily (QD). Patient-reported HRQoL outcomes assessed at the week 24 primary analysis timepoint (ie, end of period 1) included change from baseline in vitiligo QoL (VitiQoL) total score (lower scores reflect better HRQoL), achievement of a Vitiligo Noticeability Scale (VNS) response of 4 (“a lot less noticeable”) or 5 (“no longer noticeable”), change from baseline in Dermatology Life Quality Index (DLQI; lower scores reflect better HRQoL), change from baseline in Hospital Anxiety and Depression Scale (HADS) anxiety and depression scores (where lower scores reflect less anxiety and/or depression), and achievement of a Patient’s Global Impression of Change-Vitiligo (PaGIC-V) response of 1 (“much better”) or 2 (“a little better”). Results A total of 185 patients were enrolled in the study (UPA6 n=49; UPA11, n=47; UPA22, n=43; placebo n=46). Mean reductions in VitiQoL scores from baseline to week 24 with UPA22 (−6.6) and UPA6 (−7.5) were numerically larger than with placebo (−5.5), although not statistically significant. At week 24, more patients treated with UPA22 reported a VNS response of “a lot less noticeable” or “no longer noticeable” than with placebo (11.6% vs 0%, respectively; P &amp;lt; .05). Mean reductions in DLQI scores from baseline to week 24 were significantly larger with UPA22 than with placebo (−2.2 vs −0.6; P &amp;lt; .05). HADS anxiety scores decreased from baseline to week 24 in the UPA6 and UPA22 groups, with similar reductions in HADS depression scores observed for the UPA11 and UPA22 groups; changes in HADS scores were not significantly different vs placebo. A significantly greater proportion of patients achieved a PaGIC-V response of “much better” or “a little better” with any UPA dose vs placebo: UPA6 (34.7%), UPA11 (55.3%), and UPA22 (60.5%) vs placebo (19.6%; P &amp;lt; .05 vs placebo for all UPA doses). Conclusion Among adults with non-segmental vitiligo, 24 weeks of treatment with UPA at all doses resulted in improvements compared to placebo in patient impressions of disease improvement based on PaGIC results. These initial findings suggest that UPA22 may improve patient HRQoL and perceptions of vitiligo noticeability; longer term data from the extension phase of this study will further clarify the impact of UPA treatment on QoL.

Real-world health-related quality of life outcomes for patients with resected stage III/IV melanoma treated with adjuvant anti-PD1 therapy

BackgroundWhile adjuvant therapy with anti-programmed cell death protein-1 (anti-PD1) for patients with resected stage III/IV melanoma has been shown to improve recurrence-free survival, the overall survival benefit remains uncertain. This study aims to evaluate the impact of adjuvant anti-PD1 therapy on the health-related quality of life (HRQOL) of patients with resected stage III/IV melanoma MethodsData was used from two melanoma registries in Australia and the Netherlands. Patients with resected stage III/IV melanoma treated with adjuvant anti-PD1 who completed a baseline and at least one post-baseline HRQOL assessment were included. HRQOL was assessed using the EORTC QLQ-C30 at baseline, 3, 6, and 12 months. Established thresholds were used for interpreting changes in QLQ-C30 scores. Results92 patients were included. Mean symptom and functioning scores improved or remained stable at 12 months compared to baseline. However, a substantial proportion of patients experienced a clinically significant decline in role (39%, μ = −50.8), social (41%, μ = −32.7), or emotional (50%, μ = −25.1) functioning at 12 months compared to baseline. Younger patients were more likely to experience clinically significant deteriorations in role (OR=1.07, 95% CI: 1.02–1.13, p < 0.01) and social (OR=1.06, 95% CI: 1.01–1.11, p = 0.013) functioning. ConclusionA significant proportion of patients with resected stage III/IV melanoma who received adjuvant anti-PD1 experienced clinically significant declines in role, social and emotional functioning at 12 months compared to baseline. This highlights the HRQOL issues that may arise during adjuvant anti-PD1 therapy which may require supportive care intervention.