Health-related Quality Of Life Tools Research Articles

Low-level laser therapy: an efficient supplement to treatments of vulvar Lichen sclerosus to improve quality of life

Background Lichen sclerosus (LS) is a chronic, inflammatory disease of the genital and extra genital skin, causing pruritus, soreness, pain and dyspareunia. The aim of this study was to investigate whether Low Level Laser Therapy (LLLT) can improve the quality of life in women with Lichen sclerosus (LS) and insufficient topical treatment. Methods In a descriptive prospective observational study conducted between 02.01.2016 and 08.01.2018, we included 100 women with LS with insufficient topical treatment because of poor response of symptoms. All participants received ten LLLT treatments (808 nm and 500 mW) over a period of 8 weeks. The first four treatments were planned as two treatments per week. The remaining six treatments were planned as once a week. A Danish health-related quality of life tool (HRQoL test) monitored the effect. Results A total of 94 patients completed the study, median age of 62 [InterQuartile Range 53–69]. There was a statistically significant improvement in seven of the eight domains of the HRQoL test after ten LLLT. We found the results of DoloTest to be statistically significant in all of the groups except for smoking (p < 0.094). Conclusions LLLT treatment can improve the quality of life in women with LS.

Development and validation of a questionnaire to assess health-related quality-of-life in cats with hyperthyroidism.

Health-related quality-of-life (HRQoL) assessment tools are becoming increasingly important for the assessment of diseases in veterinary medicine. To develop a tool to assess the HRQoL of hyperthyroid cats and their owners. Cats with hyperthyroidism (n = 229) and without hyperthyroidism (n = 322). Cross-sectional study design. A preliminary list of 28 questions relating to the HRQoL of hyperthyroid cats and the influence their cat's disease might have on owners was created. Each question consisted of 2 subquestions: (1) "how often does the item apply"; (2) "how strongly does the item affect HRQoL." The questionnaire was refined based on statistical analysis, including Mann-Whitney-U tests on each item, comparing the results from cats with and without hyperthyroidism. Internal consistency and reliability of the questions were measured by Cronbach's alpha (α). P < .05 was considered significant. Overall, 25/28 questions were retained within the final HRQoL tool, which had an excellent internal consistency (α = .92). The tool produced a score between 0 and 382 (lower scores meaning better HRQoL). The median HRQoL score was 87.5 (range, 2-348) for cats with hyperthyroidism, and 27 (range, 0-249) for cats without (P < .001), suggesting the HRQoL was poorer in hyperthyroid cats. This validated HRQoL tool is useful to reliably quantify the influence of hyperthyroidism on the quality-of-life of affected cats and their owners. In the future, it could be considered of assistance in the clinical assessment of cats with hyperthyroidism.

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.

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 lifethreatening adverse events (AE), including cytokine release syndrome and immune effector cellassociated neurotoxicity syndrome. While clinical trials evaluating CAR T-cell therapy consistently report the incidence of these AE, most trials do not collect health-related quality of life (HRQoL) data. As such, the impact of CAR T-cell therapy process and related AE on the physical and psychological well-being of patients remains uncertain. HRQoL and other patientreported 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 patient care and costs, and enable comparison between different studies. We conducted a literature search up to June 2023 to identify the HRQoL tools used in clinical trials and in realworld 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.

Health-related quality of life and its influencing factors in patients with primary cutaneous B-cell lymphomas: A multicentric study in 100 patients.

Primary cutaneous B-cell lymphomas (CBCL) are a group of rare malignant skin diseases that represent approximately 20%-30% of all primary cutaneous lymphomas (PCL). Previous studies revealed impaired health-related quality of life (HRQoL) in patients diagnosed with primary cutaneous T-cell lymphoma (CTCL). Currently, only small-sized studies investigated HRQoL in CBCL patients and lacked detailed analysis of respective subtypes. This study aims to investigate HRQoL in CBCL patients to identify independent factors of HRQoL impairment in CBCL patients. One hundred CBCL patients were recruited from eight German PCL centres in this multicentric, cross-sectional study from 2021 to 2022. The patients completed the dermatologic HRQoL questionnaire Skindex-29 and an investigator-designed 'CBCL-Questionnaire' with additional questions on HRQoL and clinical characteristics. The Skindex-29 revealed that HRQoL in CBCL patients is impaired on a mild to moderate level. The multiple regression analysis identified parameters like worries about dying, feeling prejudiced/discriminated and impairment of daily activities to be independently associated with impairment of HRQoL. Highest scores for HRQoL impairment were found in patients with primary cutaneous follicle centre lymphoma while on rituximab treatment and in patients with primary cutaneous marginal zone lymphoma while on watchful waiting. HRQoL is impaired in CBCL patients, even though, in the face of indolent disease course and favourable prognosis in the majority of cases. Of note, our investigator-designed tool identified worries about dying, feeling prejudiced/discriminated, and the type of treatment to have a negative impact on patients' HRQoL. Our study highlights the importance of a thorough patient-doctor communication to capture overall disease burden because generic HRQoL tools might lack of disease-specific items.

Management and health-related quality of life among patients with prostate cancer in a Kenyan tertiary health facility

Introduction Advances made in the screening, diagnosis and management of prostate cancer have improved the survival rates of the patients. However, many of these treatments including surgery, radiotherapy, and pharmacotherapy, have an impact on the subsequent health-related quality of life (HRQoL) of these patients. Since it is an important prognostic factor of survival, failure to evaluate the HRQoL and its predictors in these patients typically results in long-term deficits in their overall well-being, that is, their physical, social, emotional, and mental health. The objective of this study was to evaluate the management and HRQoL among patients with prostate cancer at Kenyatta National Hospital. Methods This was a descriptive cross-sectional study. The sample size of 62 patients who met the eligibility criteria was selected through simple random sampling on the respective clinic days of the cancer treatment centre and urology clinic. Data was collected through a pre-tested structured questionnaire and HRQoL tools which are EORTC-QLQ-C30 and EORTC-QLQ-PR25 and analysed using STATA version 13 software. Descriptive analysis was used to summarise the continuous and categorical variables. Spearman's rho (rs) correlation was used to determine the predictors of HRQoL based on the strength and significance of association at 0.05 level of significance. Results The mean age of the participants was 70.5 (±7.35) years. The majority (52, 83.9%) of the patients had a prostate specific antigen (PSA) above 20 ng/ml. Twenty-one (33.9%) were graded as Gleason group 5 and 41 (66.1%) had stage IV disease at diagnosis. Fifty (80.9%) participants were on hormonal therapy, with most of them being on combined androgen blockade. The overall HRQoL was 65.1. Fatigue, one of the major complaints among these patients, was negatively associated with physical functioning (p = 0.0005), role functioning (p = 0.0026), social functioning (p = 0.0001), financial difficulties (p = 0.0077) and quality of life (p = 0.0050). Conclusion Fatigue was the most common predictor of poor HRQoL in several scales of measurement. For those on management, frequent assessment of HRQoL should be carried out and interventions instituted immediately.

The validity and reliability of Indonesian version of atrial fibrillation effect on quality of life (AFEQT) questionnaire for atrial fibrillation patients

BackgroundMore than 60% of patients with atrial fibrillation (AF) have a significant health-related quality of life (HRQoL) impairment. HRQoL, a patient-reported outcome (PRO), has become an important endpoint to assess treatment success in AF patients. The Atrial Fibrillation Effect on Quality of Life (AFEQT) questionnaire is an AF-specific HRQoL tool shown to be feasible, reliable, and valid, with translations in various languages. Since this questionnaire has never been translated or validated in Indonesian, we aimed to determine the validity and reliability of the Indonesian version of the AFEQT questionnaire for AF patients.ResultsThis cross-sectional, observational study was conducted in the Integrated Cardiovascular Service Polyclinic, Cipto Mangunkusumo Hospital, Indonesia, from December 2021 to March 2022. A total of 30 participants were recruited for cross-cultural adaptation process, which consisted of translation and adaptation process, and a total of 102 participants were consecutively recruited to participate in the validation process, which consisted of validity test (construct validity) and reliability tests (internal consistency and test-retest). The retest was conducted within a 1–2-week interval after the baseline assessment, by analyzing the intraclass correlation coefficient (ICC). The construct validity was determined by multitrait scaling analysis, and the convergent and divergent validity was compared to SF-36 domains. Multitrait scaling analysis revealed that all items in the Indonesian version of the AFEQT questionnaire had a strong negative correlation towards their respective domains (r -0.639–-0.960). For convergent and divergent validity, AFEQT domains had weak to strong positive correlations to all SF-36 domains (r 0.338–0.693). This questionnaire also had acceptable internal consistency (Cronbach’s α for overall score: 0.947; Domains: Symptoms: 0.818, Daily Activities: 0.943, Treatment Concern: 0.894, and Treatment Satisfaction: 0.865), as well as moderate-to-good test-retest reliability (0.521–0.828).ConclusionsThe Indonesian version of the AFEQT questionnaire has good validity and reliability for assessing quality of life of atrial fibrillation patients in Indonesia.

Quality-of-life measurement long-term after resection of colorectal liver metastases – is there an optimal assessment tool?

BackgroundThere is limited literature on health-related quality-of-life (HRQoL) in patients with colorectal liver metastases (CRLM). Furthermore, there is no consensus on which HRQoL tool is most appropriate. This study assessed the long-term HRQoL in patients who undergo liver resection for CRLM and assessed which HRQoL tool is most useful. MethodsThis was a cross-sectional study of patients who had curative resection for CRLM between 2010 and June 2021. Three validated instruments were used: The European Organisation for Research and Treatment of Cancer (EORTC), which consists of the QLQ-C30 (a generic questionnaire) and QLQ-LMC21 (CRLM specific); the EuroQol-5D (EQ-5D) and the 36-Item Short Form Survey. Results121 patients underwent liver resection for CRLM, of which 85 were alive. There was a 61 % response rate (n = 52). The median post-operative time when the survey was completed was 4.0 years. Across all three questionnaires, patients performed exceptionally well in all domains, with median functional scores >90. The EQ-5D-5L VAS and the EROTC QLQ-C30 produced similar results. ConclusionThis study demonstrates excellent long-term HRQoL in patients who undergo resection for CRLM. The EQ-5D questionnaire is the preferred questionnaire because it is shorter and simpler to complete than the other tools without compromising accuracy.

Effect of immunotherapy on health-related quality of life outcomes in patients with advanced gastro-esophageal cancers: A meta-analysis.

245 Background: Immune checkpoint inhibitors (ICIs) have revolutionized cancer care, with increasing data demonstrating improved survival outcomes using ICIs among patients with advanced gastroesophageal cancer (aGEC). ICIs are also associated with a lower incidence of grade ≥3 adverse effects (AEs) compared to chemotherapy (CT), suggesting that ICIs may have favorable effects on health-related quality of life (HRQoL). This meta-analysis sought to evaluate the effects of ICIs on the HRQoL of patients with aGEC. Methods: An online bibliographic search was conducted on Medline via PubMed using MeSH-based terms to retrieve randomized controlled trials (RCTs) that evaluated the effects of ICIs on HRQoL in patients with aGEC. We included RCTs that incorporated ICIs as part of the intervention arm either as monotherapy (second-line) or as a combination therapy (first-line) with another ICI or CT. We combined the HRQoL measures into a meta-analysis using standard random effects models, from which estimates of the average mean difference (MD) were obtained with 95% confidence intervals. We assessed heterogeneity of the study outcomes using the Q and I2 statistics. Results: 11 phase-3 RCTs were included, with a mean enrollment of 820 patients. Most (n=X) of the RCTs had ICI plus CT combination in the intervention arm, three had ICIs as monotherapy, and one had doublet ICI therapy in the intervention arm. All RCTs had CT for the control arm. Collectively, trials reported 37 HRQoL measures using 5 different HRQoL tools. The pooled analysis favored the intervention over the control arm in terms of Functional Assessment of Cancer Therapy-Esophageal (FACT-E) scores [MD 2.7 (95% CI 0.1 to 5.3), p&lt;0.05]. In a subgroup analysis of 8 RCTs comparing combination therapy with ICIs plus CT versus CT alone, the effect estimates favored the ICI arm regarding the FACT-E [MD 2.7 (95% CI 0.1 to 5.3), p&lt;0.05] and the EORTC QLQ-OES18 pain scale [MD -2.2 (95% CI -4.3 to -0.2), p&lt;0.05]. We found no statistically significant differences in HRQoL scales in subgroup analysis of ICI monotherapy versus CT. Conclusions: In this meta-analysis, we found that the inclusion of ICIs as first-line treatment for aGECs yielded better HRQoL outcomes than CT. Further research on the impact of ICIs on HRQoL is needed, yet with increasing evidence that ICIs improve survival outcomes in patients with aGEC, these corresponding benefits with HRQoL are encouraging.[Table: see text]

Circumstances and outcome of active transportation injuries: protocol of a British Columbian inception cohort study

IntroductionActive transport (AT) is promoted by urban planners and health officials for its environmental, economic and societal benefits and its uptake is increasing. Unfortunately, AT users can be injured or...

Effect of Physical Activity on Health-Related Quality of Life of Older Adults Using Newly Developed Health-Related Quality of Life Tool for the Korean Population.

This study aimed to investigate the effect of physical activity on health-related quality of life. Data from the 2019 Korean National Health and Nutrition Examination Survey were used. The participants were 1495 (56.7% women) older individuals older than 65 years of age. A one-way analysis of variance (ANOVA) and multiple regression analysis were performed to determine the effect of walking and strength exercise on the health-related quality of life using the Korean version of the health-related quality of life questionnaire called the Korean Health-Related Quality of Life Instrument with 8 Items (HINT-8). The one-way ANOVA revealed that the health-related quality of life (according to the HINT-8) differed depending on increases in walking and strength exercise. Furthermore, walking and strength exercise positively influenced the health-related quality of life according to the HINT-8. Therefore, this study showed that walking and strength exercise were associated with the health-related quality of life among older Korean individuals. This study will be helpful for future studies on the health-related quality of life of older individuals.

Effects of aerobic, resistance, and combined exercise training on health-related quality of life in multiple sclerosis: Systematic review and meta-analysis.

Multiple sclerosis (MS) is an immune-mediated, neurodegenerative disease of the central nervous system that manifests in symptoms that compromise health-related quality of life (HRQOL). HRQOL focuses on a person's overall, subjective evaluation of health status primarily in the physical and mental domains. Exercise training is a form of rehabilitation for managing MS-related outcomes that might influence HRQOL. Reviews on exercise training are available, but we are unaware of a recent comprehensive review and meta-analysis of exercise effects for improving physical and mental domains of HRQOL. This analysis provides an updated review and meta-analysis of randomized controlled trials (RCTs) examining interventions consisting of aerobic, resistance and combined exercise training for improving HRQOL in persons with MS. This systematic review 1) assessed the overall strength of evidence for exercise interventions on HRQOL, 2) evaluated the relative effect of exercise interventions on physical and mental domains of HRQOL, and 3) determined moderators of exercise intervention effects on HRQOL. Seven databases were searched for RCTs evaluating physical and/or mental domains of HRQOL with adults diagnosed with MS and undergoing an intervention of aerobic, resistance or combined exercise training compared with a non-exercise comparator. Data extraction included participant and intervention characteristics, and pre- and post-intervention HRQOL outcome data. Effect sizes (ESs) were calculated as standardized mean differences (SMDs) and a multilevel random-effects model was used to generate an aggregated SMD that compared exercise with non-exercise control conditions. Twelve RCTs met the inclusion criteria and yielded 23 ESs to be analyzed. Participants (N=593; 308 intervention vs. 285 control conditions) had a mean (±standard deviation) age of 42.4 (6.5) years and 80% (18.3%) were female. Results generated a medium effect of exercise for improving overall HRQOL (ES=0.64, p=0.0001) with high heterogeneity (Q11=58.8, I2=86.7%). Exercise training yielded a large effect on the physical domain (k=12, ES=0.82, p<0.0009) and a medium effect on the mental domain (k=11, ES=0.41, p<0.0001). Moderator analyses identified exercise modality, supervision level, intervention delivery and length, HRQOL tool, and number of participants with relapsing-remitting MS as significant influences of ES for HRQOL. Exercise training is clinically effective for improving overall HRQOL in MS and produces greater improvements in the physical domain of HRQOL than the mental domain. The moderator analysis suggests that supervised, aerobic, and group-delivered exercise training of ≥3 months yields the most influence on HRQOL. Such results may have major implications for MS treatment and care.

Dyspnea Associates With a Widely Impaired Quality of Life in Idiopathic Pulmonary Fibrosis Patients: A Longitudinal Study Using 15D.

Background: Research on health-related quality of life (HRQoL) is crucial for developing comprehensive palliative care in idiopathic pulmonary fibrosis (IPF). Objectives: To study IPF patients' HRQoL compared with general population and its association with dyspnea in a longitudinal follow-up. Design: Assessment of IPF patients' HRQoL by a generic tool. Comparison of baseline data with the general population and a 30-month follow-up with 6 months intervals. Setting/Subjects: In total, 246 IPF patients were recruited from the Finnish nationwide real-life study, FinnishIPF. Measurements: Modified Medical Research Council (MMRC) dyspnea scale for dyspnea and the generic HRQoL tool 15D for the total and dimensional HRQoL were used. Results: At baseline, the mean 15D total score was lower (0.786, standard deviation [SD] 0.116) in IPF patients than in the general population (0.871, SD 0.043) (p < 0.001) and among the IPF patients with MMRC ≥2 compared with those with MMRC <2 (p < 0.001). In patients with MMRC ≥2, significant impairment compared with general population existed in 11 dimensions of HRQoL, such as breathing, usual activities, and sexual activity, whereas this was true in only 4 dimensions in MMRC <2 category. Mental function was not impaired in either group. During the follow-up, 15D total score decreased in both MMRC categories (p < 0.001) but stayed constantly worse in the MMRC ≥2 group. Seven and two dimensions of HRQoL significantly declined in the categories of MMRC <2 and MMRC ≥2, respectively. Conclusions: Patients with IPF, especially if dyspnea limits everyday life, suffer from widely impaired HRQoL, although self-assessed mental capability is preserved. Integrated palliative care is supported to face the multiple needs of IPF patients.

The SMILE scale: A pilot study among patients with cancer.

e24036 Background: In the US, it is projected that more than 22 million people will be living with cancer by 2030. As survivorship increases, there is a need for simple, convenient tools to quantify patient quality of life and encourage healthy behaviors. To fill this gap, a new psychometric tool named the SMILE Scale was created. The SMILE Scale is a brief self-administered questionnaire assessing the frequency of behaviors intended to improve wellness in five domains: Sleep, physical activity (Move), stress management (Inhale and Exhale), social interactions (Love and connect), and nutrition (Eat to nourish). In this study, we aimed to determine the feasibility of collecting daily self-reported SMILE Scale data and weekly quality of life data, via electronic surveys in patients with cancer. We also aimed to measure the association between SMILE Scale responses and validated health-related quality of life (HRQOL) tools (PROMIS-29+2 and SymTrak-8) as a pilot test of the hypothesis that increased wellness behaviors may impact quality of life. Methods: We surveyed 100 patients with cancer at the IU Simon Comprehensive Cancer Center. Participants were asked to complete daily SMILE Scale assessments over a two-week period, as well as weekly PROMIS-29+2 and SymTrak-8 surveys. The primary endpoint was the SMILE Scale completion rate. Secondary endpoints in this single-arm pilot study included correlations between SMILE Scale results and the other HRQOL tools. Results: Daily completion rate of the SMILE Scale ranged from 57% to 65% of participants over a 14-day period. Among the 61% of participants who completed SMILE on day 1, 87% completed SMILE on 10 of 14 days. Further, 77% completed SMILE at least 1 of the 14 days. By end of study, participants who self-reported more wellness behaviors (i.e., higher daily SMILE scores) demonstrated significantly higher PROMIS physical health (p = .003), higher PROMIS mental health (p = .008), and lower (better) SymTrak total symptom burden (p = .006). Further, among those who completed at least 1 of 14 daily SMILE assessments, quality of life significantly improved over the two-week period for PROMIS mental health (p = .018) and SymTrak total symptom burden (p = .014). Conclusions: The SMILE Scale completion rate on any given day did not satisfy our pre-planned ≥70% threshold for feasibility; however, the rate for completing SMILE at least once during the 14 days (77%) met this threshold. There is growing recognition that interventions with modest uptake may benefit interested patients. The SMILE completion rate across 14 days among those who completed at least one day indicates benefit for providing a daily or on-demand option for those interested. Further, significant correlative results between SMILE scores and secondary QOL endpoints suggest a potential benefit for delivering SMILE in clinical practice and warrants testing the SMILE Scale’s causal impact in a future randomized trial.

Real world impact of hepatocellular carcinoma stage and hepatic function on health-related quality of life.

e16215 Background: Health-Related Quality of Life (HRQoL) is an important prognostic indicator and clinical endpoint for patients with hepatocellular carcinoma (HCC). The relationship between cancer stage, liver function, performance status, and HRQoL in HCC in a real-world clinical setting is not well defined. Methods: A retrospective chart review was undertaken of consecutive patients with HCC treated at our institution's liver multidisciplinary clinic between February 2020 and August 2022. We examined the relationship between Barcelona Clinic Liver Cancer (BCLC) stage, Child Pugh (CP) score, and Eastern Cooperative Oncology Group (ECOG) performance status on HRQoL at diagnosis. HRQoL was assessed using the Functional Assessment of Cancer Therapy-Hepatobiliary (FACT-Hep) questionnaire. Results: Fifty-one patients met our inclusion criteria. The median age was 69 and the mean FACT-Hep score was 130.3 ± 20.3. FACT-Hep total and subscales showed no significant association with BCLC stages A, B, and C (p =0.224), except in the Emotional Well Being (EWB) subscale (p =0.001). There was no impact of ECOG performance status on the FACT-Hep scores, except for the Functional Well Being (FWB) subscale (p=0.006). Patients with a CP B had significantly more impairment in FACT-Hep than patients with CP A, including FACT-Hep total score (118.5 ±27.3 versus 134.3 ±23.8; p =0.021) and subscales including the Physical Well Being (p =0.018), FWB (p =0.030), Hepatobiliary Cancer Subscale (p &lt;0.001), and FACT-Hep Trial Outcome Index score (p =0.003). Conclusions: Cancer stage poorly correlates with HRQoL at diagnosis in HCC patients. Impaired liver function reduces overall quality of life in HCC patients. Further large-scale prospective studies should be undertaken which incorporate validated HRQoL tools in the routine assessment of HCC patients. [Table: see text]

Clinical, biochemical and molecular characterization of 12 patients with pyruvate carboxylase deficiency treated with triheptanoin.

Pyruvate carboxylase (PC) deficiency is a rare autosomal recessive mitochondrial neurometabolic disorder of energy deficit resulting in high morbidity and mortality, with limited therapeutic options. The PC homotetramer has a critical role in gluconeogenesis, anaplerosis, neurotransmitter synthesis, and lipogenesis. The main biochemical and clinical findings in PC deficiency (PCD) include lactic acidosis, ketonuria, failure to thrive, and neurological dysfunction. Use of the anaplerotic agent triheptanoin on a limited number of individuals with PCD has had mixed results. We expand on the potential utility of triheptanoin in PCD by examining the clinical, biochemical, molecular, and health-related quality-of-life (HRQoL) findings in a cohort of 12 individuals with PCD (eight with Type A and two each with Types B and C) treated with triheptanoin ranging for 6days to about 7years. The main endpoints were changes in blood lactate and HRQoL scores, but collection of useful data was limited to about half of subjects. An overall trend of lactate reduction with time on triheptanoin was noted, but with significant variability among subjects and only one subject reaching close to statistical significance for this endpoint. Parent reported HRQoL assessments with treatment showed mixed results, with some subjects showing no change, some improvement, and some worsening of overall scores. Subjects with buried amino acids in the pyruvate carboxyltransferase domain of PC that undergo destabilizing replacements may be more likely to respond (with lactate reduction or HRQoL improvement) to triheptanoin compared to those with replacements that disrupt tetramerization or subunit-subunit interface contacts. The reason for this difference is unclear and requires further validation. We observed significant variability but an overall trend of lactate reduction with time on triheptanoin and mixed parent reported outcome changes by HRQoL assessments for subjects with PCD on long-term triheptanoin. The mixed results noted with triheptanoin therapy in this study could be due to endpoint data limitation, variability of disease severity between subjects, limitation of the parent reported HRQoL tool, or subject genotype variability. Alternative designed trials and more study subjects with PCD will be needed to validate important observations from this work.

Linguistic accuracy and authenticity of the content of the Russian version of the module of gastrointestinal symptoms of the PedsQL&lt;sup&gt;™&lt;/sup&gt; questionnaire for pediatric patients

Background. Health Related Quality of Life (HRQL) is a person’s perception of the effect of a disease on physical, social and psychological functioning and wealth. Questionnaires that help assess the HRQL can give a concept of child’s state, identify arcane issues and they are of great importance for a full understanding of how the state of health affects the child. Universal HRQL tools allow to compare results with general parameters, indicators with specific symptoms better reflect clinically significant moments. The most widespread in the Russian Federation was the general PedsQLTM 4.0 questionnaire, widely used in practice to assess HRQL in children with various pathologies. Objective.The aim of the study is to describe the analysis of linguistic accuracy and authenticity of the content of the Russian version of the module of gastrointestinal symptoms of the PedsQLTM questionnaire, measuring HRQL, in children with gastrointestinal disorders (GIDs).Materials and methods. The establishment of linguistic accuracy and authenticity of the content was carried out according to international standards. The process included forward translation, scientific assessment and coordination, reverse translation, verification of the reverse translation and interviews with 17 children aged 5–18 years with symptoms of GIDs and 20 parents of children with symptoms of GIDs aged 2–18 years.Results. The Russian version of PedsQLTM module of gastrointestinal symptoms (report from children 5-18 years old, report from parents for children 2–18 years old) was developed without significant difficulties. Eight questions required discussion after the forward translation, one change was made after the reverse translation, and three changes were made after the study of the symptom’s module by patients and parents.Conclusion. A conceptually equivalent version of the of PedsQLTM module of gastrointestinal symptoms in Russian has been developed for children aged 2–18 years. It allows to improve the assessment of HRQL in children with GIDs in the Russian Federation. To assess authenticity and reliability of the Russian version of the module, it’s recommended to conduct further research using a larger sample

Leveraging patient-reported outcomes to discern mobility trajectories in Parkinson’s disease (P10-11.003)

<h3>Objective:</h3> To characterize subgroups of persons with Parkinson’s disease (PWP) with similar longitudinal mobility impairment patterns based on an easy-to-use patient-reported outcome (PRO) measure. <h3>Background:</h3> Mobility impairments are common, complex symptoms experienced by PWP. The underlying mechanisms and accrual patterns are not well understood, which impedes therapeutic shared-decision making. <h3>Design/Methods:</h3> Latent class growth analysis (LCGA) uses finite mixture models to estimate discrete groups of trajectory parameters using maximum likelihoods. Using longitudinal data available through Fox Insight Data Exploration Network, LCGA was applied to 16,863 PWP stratified by early (N=8612; &lt;3 years), mid (N=6181; 3–10 years) and later (N=2070; &gt;10 years) disease duration spanning ~4.5 years, to empirically discern clusters of PWP with similar longitudinal mobility impairment patterns. Mobility impairment was measured using a generic health-related quality of life tool that has shown construct validity in PWP (EuroQol 5 dimensions [EQ-5D-5L] mobility component). Associations were evaluated between latent trajectory classes and sociodemographic/clinical factors. <h3>Results:</h3> In the early and mid-disease duration strata, four clusters were identified, while a fifth cluster was identified in the later disease duration strata. The subgroup trajectories ranged from none to moderate mobility problems, with a smaller cluster of subjects with severe mobility problems. The percentage of subjects with moderate (early=17.5%, mid=26.4%, later=32.5%) and severe (early=3.8%, mid=7.4%, later=15.4%) mobility problems at baseline increased across disease duration groups. The trajectories tended to be variable or slightly worsening in the early and later duration group and more stable in the mid-duration group. Across strata, the clusters with moderate to severe mobility problems were associated with more severe impairment, depression, anxiety and arthritis, higher BMI, lower income, and lower education. There were no differences by race. <h3>Conclusions:</h3> The approach described and applied to PWP provides a novel template for using a easy-to-use PRO to improve our understanding of mobility trajectories in Parkinson’s disease. <b>Disclosure:</b> Doug Gunzler has nothing to disclose. Dr. Gunzler has received research support from NIH/NINDS. The institution of Dr. Gunzler has received research support from Biogen. The institution of Dr. Gunzler has received research support from Amneal. Dr. Gunzler has received research support from Michael J Fox Foundation. The institution of Prof. Briggs has received research support from NIH. The institution of Prof. Briggs has received research support from Michael J. Fox Foundation.

Effect of Mayan Abdominal Massage Technique on Sexuality and Quality of Life among Women with Uterine Prolapse

Pelvic organ prolapse dramatically compromises sexual, bowel, and bladder function and lowersquality of life. Mayan abdominal massage is a form of non-invasive external self-treatment thatpromotes the natural healing of the abdomen and pelvic organs. The aim of this study was toevaluate the effect of Mayan abdominal massage technique on sexuality and quality of lifeamong women with uterine prolapse. Research design: A quasi experimental design was utilizedto fulfill the aim of the study. Sample: A purposive sample of 75 women was selected accordingto the inclusion criteria. Setting: In gynecological outpatient and the family planning clinics atTeaching University Hospital, Menoufia Governorate. Egypt. Tools used to collect the datawere the Structured Interview Questionnaire, Female Sexual Function Index, and Health-relatedQuality of life tool. Results: The total mean scores of female sexual function index weresignificantly improved after three months of intervention from 18.7 ± 4.5 to 28.1 ± 4.3. Also,there are statistically significant differences of the mean scores of Health-related Quality of lifedomains between before and after three months of intervention. Conclusion: Mayan abdominalmassage significantly improves sexual function and quality of life after three months'intervention among women with uterine prolapse. Recommendation: Mayan abdominalmassage is considered one of the most effective therapeutic options accompanied with changinglifestyle for improving sexual function and quality of life among women with uterine prolapseshould be used as an integral part of the gynecological treatment in the maternity hospitals

EQ-5D in dialysis units: a PROM with a view

Summary Chronic kidney disease (CKD) is a silent worldwide epidemic responsible for a high clinical and socioeconomic burden. Beyond disease-related outcomes, there is an urgent need for clinicians to focus on implementation of validated patient-reported outcome measures (PROMs) in routine care practice. This updated concept of high-quality renal care implies a changing paradigm, with a focus on patient experiences and health-related quality of life (HRQL) measures. This is even more crucial in end-stage renal disease, where adequate dialysis should aim at a multidimensional approach instead of only analytical targets. It is vital to emphasize interventions that positively affect the quality of life of the patient with CKD beyond improving their survival. Although the importance of using HRQL measures is well established, there has been resistance to their use in routine care. There are numerous tools to assess HRQL, but not all are easy to apply. It is essential to overcome these possible barriers and better adequate the HRQL tools to the patients. The shorter and simpler instruments are more appealing, as well as the electronic health questionnaires. The EuroQol-5 Dimensions tool (EQ5D) is a standardized measure of health status, is simple and quick, and provides information that can be used in economic assessments of healthcare.In this era of limited health resources, cost analysis and economic evaluations are becoming increasingly relevant. In dialysis units, sustainability management should include a pathway of integrated care, including home and center dialysis, that values the better adjustment of prescriptions to the individual patient. The authors advocate using the EQ5D to support this pathway of quality in dialysis units toward global health gains. The EQ5D is a PROM with a view centered on patient and sustainable health services.