Quality Of Life Metrics Research Articles

Impact of opioids on esophageal motility

Opioids are well known to cause adverse effects on the gastrointestinal tract including nausea, vomiting, and constipation. Data regarding how opioids affect the esophagus are more limited. Opioid-induced esophageal dysfunction (OIED) is a clinical syndrome defined by chronic opioid use (≥3 months), esophageal symptoms (mainly dysphagia), and esophageal motility abnormalities diagnosed by manometry including achalasia type III, hypercontractile esophagus, distal esophageal spasm, and esophagogastric junction outflow obstruction. Up until now, the effect of opioids on esophageal motility assessed by the functional lumen imaging probe (FLIP) had not been described. In this issue of NGM, Patel et al. report that FLIP assessment in patients with esophageal symptoms showed that chronic opioid users have a significant increase in repetitive retrograde contractions, but no significant reduction in distensibility at the esophagogastric junction compared to non-users. Additionally, perceptive symptoms were higher, and quality of life metrics were lower in the chronic opioid users. This review article will discuss our current understanding of OIED and provide context for this latest study in chronic opioid users. Further investigation with larger prospective studies is needed to understand the pathophysiology, diagnosis, and management of OIED.

Long term outcomes of robotic-assisted abdominal wall reconstruction: a single surgeon experience.

Robotic abdominal wall reconstruction (RAWR) is one of the most significant advances in the management of complex abdominal wall hernias. The objective of this study was to evaluate long term outcomes in a cohort of patients that underwent complex RAWR in a single center. This was a longitudinal retrospective review of a cohort of 56 patients who underwent complex RAWR at least 24months prior by a single surgeon at a tertiary care institution. All patients underwent bilateral retro-rectus release (rRRR) with or without robotic transversus abdominis release (rTAR). Data collected include demographics, hernia details, operative and technical details. The prospective analysis included a post-procedure visit of at least 24months from the index procedure with a physical examination and quality of life survey using the Carolinas Comfort Scale (CCS). Patients with reported symptoms concerning for hernia recurrence underwent radiographic imaging. Descriptive statistics (mean ± standard deviation or median) were calculated for continuous variables. Chi-square or Fisher's exact test as deemed appropriate for categorical variables, and analysis of variance or the Kruskal-Wallis test for continuous data, were performed among the separate operative groups. A total score for the CCS was calculated and analyzed in accordance with the user guidelines. One-hundred and-forty patients met the inclusion criteria. Fifty-six patients consented to participate in the study. Mean age was 60.2years. Mean BMI was 34.0. Ninety percent of patients had at least one comorbidity and 52% of patients were scored ASA 3 or higher. Fifty-nine percent were initial incisional hernias, 19.6% were recurrent incisional hernias and 8.9% were recurrent ventral hernias. The mean defect width was 9cm for rTAR and 5cm for rRRR. The mean implanted mesh size was 945.0cm2 for rTAR and 362.5cm2 for rRRR. The mean length of follow-up was 28.1months. Fifty-seven percent of patients underwent post-op imaging at an average follow-up of 23.5months. Recurrence rate was 3.6% for all groups. There were no recurrences in patients that underwent solely bilateral rRRR. Two patients (7.7%) that underwent rTAR procedures were found with recurrence. Average time to recurrence was 23months. Quality of life survey demonstrated an overall CCS score of 6.63 ± 13.95 at 24months with 12 (21.4%) patients reporting mesh sensation, 20 (35.7%) reporting pain, and 13 (23.2%) reporting movement limitation. Our study contributes to the paucity of literature describing long term outcomes of RAWR. Robotic techniques offer durable repairs with acceptable quality of life metrics.

A Hierarchical Approach to Realignment Strategies in Adult Cervical Deformity Surgery.

Retrospective cohort study. Construct an individualized cervical realignment strategy based on patient parameters at the presentation that results in superior 2-year health-related quality of life metrics and decreased rates of junctional failure and reoperation following adult cervical deformity surgery. Research has previously focused on adult cervical deformity realignment thresholds for maximizing clinical outcomes while minimizing complications. However, realignment strategies may differ based on patient presentation and clinical characteristics. We included adult cervical deformity patients with 2-year data. The optimal outcome was defined as meeting good clinical outcomes without distal junctional failure or reoperation. Radiographic parameters assessed included C2 Slope, C2-C7, McGregor's slope, TS-CL, cSVA, T1 slope, and preoperative lowest-instrumented vertebra (LIV) inclination angle. Conditional inference trees were used to establish thresholds for each parameter based on achieving the optimal outcome. Analysis of Covariance and multivariable logistic regression analysis, controlling for age, comorbidities, baseline deformity and disability, and surgical factors, assessed outcome rates for the hierarchical approach within each deformity group. One hundred twenty-seven patients were included. After correction, there was a significant difference in meeting the optimal outcome when correcting the C2 slope below 10 degrees (85% vs. 34%, P <0.001), along with lower rates of distal junctional failure (DJF) (7% vs. 42%, P <0.001). Next, after isolating patients below the C2 slope threshold, the selection of LIV with an inclination between 0 and 40 degrees demonstrated lower rates of distal junctional kyphosis and higher odds of meeting optimal outcome(OR: 4.2, P =0.011). The best third step was the correction of cSVA below 35mm. This hierarchical approach (11% of the cohort) led to significantly lower rates of DJF (0% vs. 15%, P <0.007), reoperation (8% vs. 28%, P <0.001), and higher rates of meeting optimal outcome (93% vs. 36%, P <0.001) when controlling for age, comorbidities, and baseline deformity and disability. Our results indicate that the correction of C2 slope should be prioritized during cervical deformity surgery, with the selection of a stable LIV and correction of cervical SVA below the idealized threshold. Among the numerous radiographic parameters considered during preoperative planning for cervical deformity correction, our determinations help surgeons prioritize those realignment strategies that maximize the health-related quality of life outcomes and minimize complications. Level-III.

The presence, nature and network characteristics of behavioural phenotypes in temporal lobe epilepsy.

The relationship between temporal lobe epilepsy and psychopathology has had a long and contentious history with diverse views regarding the presence, nature and severity of emotional-behavioural problems in this patient population. To address these controversies, we take a new person-centred approach through the application of unsupervised machine learning techniques to identify underlying latent groups or behavioural phenotypes. Addressed are the distinct psychopathological profiles, their linked frequency, patterns and severity and the disruptions in morphological and network properties that underlie the identified latent groups. A total of 114 patients and 83 controls from the Epilepsy Connectome Project were administered the Achenbach System of Empirically Based Assessment inventory from which six Diagnostic and Statistical Manual of Mental Disorders-oriented scales were analysed by unsupervised machine learning analytics to identify latent patient groups. Identified clusters were contrasted to controls as well as to each other in order to characterize their association with sociodemographic, clinical epilepsy and morphological and functional imaging network features. The concurrent validity of the behavioural phenotypes was examined through other measures of behaviour and quality of life. Patients overall exhibited significantly higher (abnormal) scores compared with controls. However, cluster analysis identified three latent groups: (i) unaffected, with no scale elevations compared with controls (Cluster 1, 37%); (ii) mild symptomatology characterized by significant elevations across several Diagnostic and Statistical Manual of Mental Disorders-oriented scales compared with controls (Cluster 2, 42%); and (iii) severe symptomatology with significant elevations across all scales compared with controls and the other temporal lobe epilepsy behaviour phenotype groups (Cluster 3, 21%). Concurrent validity of the behavioural phenotype grouping was demonstrated through identical stepwise links to abnormalities on independent measures including the National Institutes of Health Toolbox Emotion Battery and quality of life metrics. There were significant associations between cluster membership and sociodemographic (handedness and education), cognition (processing speed), clinical epilepsy (presence and lifetime number of tonic-clonic seizures) and neuroimaging characteristics (cortical volume and thickness and global graph theory metrics of morphology and resting-state functional MRI). Increasingly dispersed volumetric abnormalities and widespread disruptions in underlying network properties were associated with the most abnormal behavioural phenotype. Psychopathology in these patients is characterized by a series of discrete latent groups that harbour accompanying sociodemographic, clinical and neuroimaging correlates. The underlying neurobiological patterns suggest that the degree of psychopathology is linked to increasingly dispersed abnormal brain networks. Similar to cognition, machine learning approaches support a novel developing taxonomy of the comorbidities of epilepsy.

Letter: Risk for Hemorrhage the First 2 Years After Gamma Knife Surgery for Arteriovenous Malformations: An Update.

To the Editor: We have read with great interest the article by Karlsson et al1 titled “Risk for Hemorrhage the First 2 Years After Gamma Knife Surgery for Arteriovenous Malformations: An Update.” The premise of this study was based on the findings of a 2001 study that demonstrated the risk of hemorrhage after gamma knife surgery (GKS) for arteriovenous malformations (AVMs) to be strongly correlated with large AVM size, older age, and lower therapeutic dose.2 However, contradictory data collected 17 years later revealed the risk of hemorrhage was not related to AVM size or patient age.3 To clearly define predictors of hemorrhage, the authors retrospectively reviewed the outcome of 5037 patients treated with GKS for AVM.1 The average rate of hemorrhage was determined to be 2.4%, where larger AVM size, older age, and lower therapeutic dose served as significant (P < .01) risk factors, consistent with findings from the original study.1,2 AVM location (P = .16), embolization (P = .27), and history of pre-GKS hemorrhage (P = .20) were found to be nonsignificant predictors for post-GKS hemorrhage.1 The identification of clear hemorrhage risk factors after GKS for AVM in this large, heterogeneous cohort provides clinicians with more concrete guidelines for AVM management.1 The clinical management of cerebral AVMs remains challenging due to AVM heterogeneity in size, biological composition, anatomic location, and associated symptoms.4 Thus, the treatment of AVMs to reduce hemorrhage risk must be conducted in an individualized manner specific to patient presentation and AVM profile. Although no formal guidelines exist regarding treatment of unruptured AVMs, a landmark randomized trial of unruptured brain arteriovenous malformations (trial) revealed the superiority of medical management compared with interventional therapy.5 Interventional therapy was associated with an increased risk of stroke or death as compared with medical management alone (P < .0001; hazard ratio 3.70; 95% CI 1.85–7.14).5 Five-year follow-up data confirmed these findings.6 With this understanding, along with identifying general risk factors and prevalence rates of AVM hemorrhage after GKS, it would be valuable to investigate the impact of hemorrhage on mortality and morbidity in this cohort of patients undergoing interventional therapy. Doing so, while controlling for confounding variables, such as the Spetzler-Martin grade, AVM nidus morphology or location, would be clinically significant and aid in deciding which patients would truly benefit from GKS. Hemorrhage is the leading cause of morbidity and mortality associated with AVMs.7 Consequently, common symptoms of AVM-related hemorrhage such as seizures and headaches severely impair quality of life (QOL).8,9 While it can be hypothesized that post-GKS AVM hemorrhage would have profound implications for QOL outcomes, there are no reports in the literature detailing this relationship. Future studies should investigate the impact of post-GKS hemorrhage on QOL metrics, especially as GKS continues to improve and be used for more complex disease. This study by Karlsson et al1 provides a strong foundation for the future investigation of invaluable QOL metrics to guide medical decision-making in a manner that targets patient-specific QOL discrepancies while bridging this apparent gap in the literature.

Patient-Important Needs and Goals Related to Nutrition Interventions during Cancer Treatment

Patients with cancer often experience nutrition-related challenges for which they are subsequently connected with nutritional support. Yet to date there are no validated tools to assess whether nutrition interventions sufficiently address patients’ needs. A vital step toward developing a tool is to identify primary patient-important goals related to receiving nutrition support during cancer care. To that end, we interviewed patients and clinicians to identify nutrition-related needs and goals of patients undergoing cancer treatment. We interviewed 31 patients undergoing cancer treatment and 17 clinicians at the Sidney Kimmel Cancer Center at Thomas Jefferson University in Philadelphia, PA. Two coders analyzed transcripts using a conventional qualitative content analysis approach. Patients and clinicians both identified weight maintenance, improved food satisfaction and intake, and improved quality of life metrics – such as reduced emotional and financial stress – as top nutrition-related goals. Participants also highlighted the importance of patients receiving food they like and having control over what they eat when designing optimal nutrition interventions. These findings will be used in future work to create a patient-centered assessment tool designed to capture a range of patient goals related to nutrition interventions.

TRANSCUTANEOUS OSSEOINTEGRATION RECONSTRUCTION FOR WHEELCHAIR-BOUND TRANSFEMORAL AMPUTEES (K0 LEVEL)

Traditional socket prosthesis (TSP) rehabilitation for amputees is associated with substantial dissatisfaction due to poor mobility and pain from soft tissue squeezing. Osseointegration (OI), eliminates skin compression and prosthesis fit issues, providing superior mobility and quality of life (QOL) for most amputees. The potential for OI to benefit patients with exceptionally poor mobility is understudied. To address this knowledge gap, this project investigated the mobility and QOL changes.A retrospective review was performed to identify transfemoral amputees who presented with wheelchair-bound mobility (K0) and had at least five years of post-osseointegration follow-up. Outcome measures included changes in mobility (K-level, daily prosthesis wear hours, Timed Up and Go (TUG), 6 minute walk test (6MWT)) and QOL (Questionnaire for persons with a Transfemoral Amputation (QTFA); Short Form Health Survey 36 (SF36). Adverse events recorded included debridement for infection, implant revision surgery, fractures, and implant failures.9 patients with mean age 48.2±7.7 (range 34-59) years were included. The proportion of patients achieving K-level &gt;2 improved from 0/9=0% to [9/9=100%], p&lt;0.001. The 6MWT improved from 0±0 to 320.65±57 meters (p&lt;0.001). The TUG improved from incapable to 10.68±2.7 seconds (p&lt;0.001). Statistically significant QOL metrics included QTFA global score (33.33±31 vs 68.52±21, p=0.039), SF36 physical component score (30.03±6.3 vs 42.34±12, p=0.023) and the SF36 mental component score (47.89±8.8 vs 51.95±10.4, p=0.332). Six patients required operative intervention to address complications. Five developed infections: three had debridement, and 2 required implant removal and reimplantation.Osseointegration does confer significant sustained mobility and quality of life improvement for wheelchair bound transfemoral amputees. All 9 K0 patients had statistically improved mobility and QOL after osseointegrated reconstruction. This supports osseointegration as an effective rapid and long-term rehabilitation alternative for patients who have the neurologic and muscular capacity to walk but are unable due to issues related to their TSP.

A prospective randomized crossover trial of systemic chemotherapy in patients with low-grade mucinous appendiceal adenocarcinoma.

163 Background: Appendiceal adenocarcinoma is both a rare and heterogenous tumor, with marked contrast in the natural history of low-grade and high-grade tumors (5-year OS 68% for low-grade vs. 7% for high-grade). While low-grade appendiceal adenocarcinoma is primarily treated with surgical resection sometimes followed by hyperthermic intraperitoneal chemotherapy (HIPEC), many inoperable candidates are treated with systemic chemotherapy although there is no prospective data supporting this practice. The purpose of our study was to objectively evaluate the effectiveness of systemic chemotherapy in low-grade mucinous appendiceal adenocarcinoma. Methods: A randomized crossover trial of surgically unresectable low-grade (well differentiated) mucinous appendiceal adenocarcinoma was performed with patients randomized to either 6 months observation followed by 6 months of chemotherapy (physician’s choice), or initial chemotherapy followed by observation. In this way each patient would serve as their own control. Enrollment of 30 patients was planned to have complete 6- and 12-month tumor measurements for 24 patients, providing 80% power at 0.05 significance level to detect a 5.0% difference in change in tumor size as measured by peritoneal RECIST in observation vs. treatment periods. Results: The trial closed early due to slow accrual. A total of 24 patients were enrolled. The majority of patients were treated with either 5FU or capecitabine (n = 15, 63%), bevacizumab was added for 3 (13%), and 3 were treated with doublet chemotherapy (FOLFOX/FOLFIRI). 15 patients who completed both treatment and observation periods were available for the primary analysis, the mean difference in tumor size was -4.5% (95% CI: -12.6, 3.7), indicating a slight trend towards faster growth on treatment than observation. This difference was not statistically or clinically significant (8.4% growth on treatment vs. 4.0% observation, p=0.26). Of the 18 patients who received any chemotherapy during the study period, zero achieved an objective response, 14 (77.8%) had stable disease during the entire year of follow up, and 4 (12.2%) had progression on study. Patient reported quality of life metrics identified that fatigue (p=0.02), peripheral neuropathy (p=0.014), and financial difficulty (p=0.0013) were all significantly worse while on treatment. There was not a significant difference in rate of bowel obstruction between the treatment first vs. observation first arms (12.5%, (n=3) vs 8.3%, (n=2)). Conclusions: These data from a prospective, randomized crossover trial indicate that patients with low-grade mucinous appendiceal adenocarcinoma do not derive benefit from 5FU based chemotherapy but do incur toxicity. These data further highlight the unique biology of low-grade appendiceal cancer and demonstrates the need to identify novel systemic therapies for this patient population. Clinical trial information: NCT01946854 .

Dietary Exposures and Interventions in Inflammatory Bowel Disease: Current Evidence and Emerging Concepts.

Diet is intimately linked to the gastrointestinal (GI) tract and has potent effects on intestinal immune homeostasis. Inflammatory bowel disease (IBD) is characterized by chronic inflammation of the GI tract. The therapeutic implications of diet in patients with IBD have received significant attention in recent years. In this review, we provide a contemporary and comprehensive overview of dietary exposures and interventions in IBD. Epidemiological studies suggest that ultra-processed foods, food additives, and emulsifiers are associated with a higher incidence of IBD. Exclusion and elimination diets are associated with improved symptoms in patients with IBD, but no effects on objective markers of inflammation. Specific dietary interventions (e.g., Mediterranean, specific carbohydrate, high fiber, ketogenic, anti-inflammatory diets) have been shown to reduce symptoms, improve inflammatory biomarkers, and quality of life metrics to varying degrees, but these studies are limited by study design, underpowering, heterogeneity, and confounding. To date, there is no robust evidence that any dietary intervention alone may replace standard therapies in patients with IBD. However, diet may play an adjunct role to induce or maintain clinical remission with standard IBD therapies. The results of novel dietary trials in IBD such as personalized fiber, intermittent fasting, and time-restricted diets are eagerly awaited.

Comorbidity and health-related quality of life in people with a chronic medical condition in randomised clinical trials: An individual participant data meta-analysis.

Health-related quality of life metrics evaluate treatments in ways that matter to patients, so are often included in randomised clinical trials (hereafter trials). Multimorbidity, where individuals have 2 or more conditions, is negatively associated with quality of life. However, whether multimorbidity predicts change over time or modifies treatment effects for quality of life is unknown. Therefore, clinicians and guideline developers are uncertain about the applicability of trial findings to people with multimorbidity. We examined whether comorbidity count (higher counts indicating greater multimorbidity) (i) is associated with quality of life at baseline; (ii) predicts change in quality of life over time; and/or (iii) modifies treatment effects on quality of life. Included trials were registered on the United States trials registry for selected index medical conditions and drug classes, phase 2/3, 3 or 4, had ≥300 participants, a nonrestrictive upper age limit, and were available on 1 of 2 trial repositories on 21 November 2016 and 18 May 2018, respectively. Of 124 meeting these criteria, 56 trials (33,421 participants, 16 index conditions, and 23 drug classes) collected a generic quality of life outcome measure (35 EuroQol-5 dimension (EQ-5D), 31 36-item short form survey (SF-36) with 10 collecting both). Blinding and completeness of follow up were examined for each trial. Using trials where individual participant data (IPD) was available from 2 repositories, a comorbidity count was calculated from medical history and/or prescriptions data. Linear regressions were fitted for the association between comorbidity count and (i) quality of life at baseline; (ii) change in quality of life during trial follow up; and (iii) treatment effects on quality of life. These results were then combined in Bayesian linear models. Posterior samples were summarised via the mean, 2.5th and 97.5th percentiles as credible intervals (95% CI) and via the proportion with values less than 0 as the probability (PBayes) of a negative association. All results are in standardised units (obtained by dividing the EQ-5D/SF-36 estimates by published population standard deviations). Per additional comorbidity, adjusting for age and sex, across all index conditions and treatment comparisons, comorbidity count was associated with lower quality of life at baseline and with a decline in quality of life over time (EQ-5D -0.02 [95% CI -0.03 to -0.01], PBayes > 0.999). Associations were similar, but with wider 95% CIs crossing the null for SF-36-PCS and SF-36-MCS (-0.05 [-0.10 to 0.01], PBayes = 0.956 and -0.05 [-0.10 to 0.01], PBayes = 0.966, respectively). Importantly, there was no evidence of any interaction between comorbidity count and treatment efficacy for either EQ-5D or SF-36 (EQ-5D -0.0035 [95% CI -0.0153 to -0.0065], PBayes = 0.746; SF-36-MCS (-0.0111 [95% CI -0.0647 to 0.0416], PBayes = 0.70 and SF-36-PCS -0.0092 [95% CI -0.0758 to 0.0476], PBayes = 0.631. Treatment effects on quality of life did not differ by multimorbidity (measured via a comorbidity count) at baseline-for the medical conditions studied, types and severity of comorbidities and level of quality of life at baseline, suggesting that evidence from clinical trials is likely to be applicable to settings with (at least modestly) higher levels of comorbidity. A prespecified protocol was registered on PROSPERO (CRD42018048202).

Retrospective dataset and survey analyses identify gaps in data collection for craniopharyngioma and priorities of patients and families affected by the disease

IntroductionCraniopharyngioma is a rare, low-grade tumor located in the suprasellar region of the brain, near critical structures like the pituitary gland. Here, we concurrently investigate the status of clinical and genomic data in a retrospective craniopharyngioma cohort and survey-based data to better understand patient-relevant outcomes associated with existing therapies and provide a foundation to inform new treatment strategies. MethodsClinical, genomic, and outcome data for a retrospective cohort of patients with craniopharyngioma were collected and reviewed through the Children's Brain Tumor Network (CBTN) database. An anonymous survey was distributed to patients and families with a diagnosis of craniopharyngioma to understand their experiences throughout diagnosis and treatment. ResultsThe CBTN repository revealed a large proportion of patients (40 – 70%) with specimens that are available for sequencing but lacked relevant quality of life (QoL) and functional outcomes. Frequencies of reported patient comorbidities ranged from 20–35%, which is significantly lower than historically reported. Survey results from 159 patients/families identified differences in treatment considerations at time of diagnosis versus time of recurrence. In retrospective review, patients and families identified preference for therapy that would improve QoL, rather than decrease risk of recurrence (mean 3.9 vs. 4.4 of 5) and identified endocrine issues as having the greatest impact on patients’ lives. ConclusionsThis work highlights the importance of prospective collection of QoL and functional metrics alongside robust clinical and molecular correlates in individuals with craniopharyngioma. Such comprehensive measures will facilitate biologically relevant therapeutic strategies that also prioritize patient needs.

Quality of Life Metrics in Terms of Facilities and Urban Reputation: A Case Study in the City of Ventanilla, Lima Peru

The availability and access to city facilities are key metrics in the assessment of the quality of life of its inhabitants. In our case study, it became evident that there is a gap between the supply of facilities (education, health, supply, security, culture, recreation, and sports) and the context of urban planning, which must structure a comprehensive and long-term vision through urban plans. This management tool must integrate facilities with housing, transportation, facilities, workplaces, etc. Likewise, it must cover various scales (neighborhoods, districts, municipalities, metropolitan areas, regions) and a multi-stakeholder vision (government authorities, private entrepreneurs, academics, and civil society). The existing gap generates an inequitable territorial distribution of facilities and services, which leads to progressive social segregation in the city and a progressive decrease in the quality of life of its inhabitants. On the other hand, the concept of a city's reputation has two main connotations. On the one hand, it is based on the functional value that a city has for its inhabitants; on the other hand, it echoes the emotional ties between them and the city, according to social roots. This research addresses, from an empirical perspective, the supply of facilities within the city of Ventanilla and its influence on the city's reputation. The analysis was based on metrics of the spatial distribution of facilities and the perception of its inhabitants. The results show that the supply of facilities, within a decontextualized diagnosis of needs and without a long-term vision, through a Master Plan, causes a progressive deterioration of the quality of life. At the same time, quantitative metrics related to the provision of facilities can be a misleading approach to assessing the quality of life in a city. Our research made it clear that it is necessary to include metrics, methodologies, and qualitative analyses so that underlying issues such as accessibility, placemaking, and social cohesion are also assessed. Post -COVID-19 scenario concepts such as

Severity of depressive and motor symptoms impacts quality of life in Parkinson’s disease patients at an academic movement clinic: A cross-sectional study

IntroductionParkinson’s disease (PD) is a neurodegenerative disease with motor and non-motor manifestations that have been previously reported to affect patient quality of life (QoL). Our objective is to investigate the factors that contribute to QoL in a cohort of PD patients receiving care at a major academic institution. MethodsIn this cross-sectional study of 124 participants (71.77% male, mean age 65.20, mean UPDRS-III score 11.25), we analyzed if certain clinical features such as UPDRS-III, QIDS-C, and total disease duration contributed to QoL as measured by two different metrics (PDQ-39 and EQ-5D) in PD patients at a university Movement Disorders Clinic. ResultsMotor symptoms of PD, with the exception of tremor, as well as depression and specific depressive symptoms were significantly and positively correlated with lower QoL metrics for patients with Parkinson’s, with total depressive symptom severity (QIDS-C16 Total score) contributing most to QoL scores. Of the specific depressive and motor symptoms, anhedonia and rigidity contributed the most to QoL. Disease duration was significantly correlated with lower QoL for participants with Parkinson’s according to the QoL metric PDQ-39 but not ED-5D. Parkinson’s patients with access to high-quality healthcare are at risk for having diminished QoL due to both depressive and motor symptoms. ConclusionWhile severity of motor symptoms certainly impacted QoL in our cohort, our findings suggest that depressive symptoms contribute more to impaired QoL than severe motor symptoms do. This phenomenon suggests that concomitant depression in PD as well as one’s psychological adjustment to disability may have a greater impact on QoL than severe motor symptoms.

Sickle Cell Disease (SCD) Burden on Patients Treated With Hydroxyurea (HU): Results from the Real-World Assessment Survey for SCD in Saudi (ROARS)

The prevalence of SCD in the Kingdom of Saudi Arabia (KSA) is one of the highest in the world with up to 2.6% of the population affected. The Real-World Assessment Survey for Sickle Cell Disease in Saudi (ROARS) was conducted to evaluate the impact of SCD on the quality of life (QoL) of this population. Here, we present data on the QoL burden experienced by patients with SCD in KSA treated with hydroxyurea (HU). Between September and December 2021, patients in KSA with SCD (age: 5-65 years) were surveyed (caregivers were surveyed for patients 5-14 years) using structured, bilingual ROARS questionnaires (English/Arabic) through personal quantitative web-assisted telephone interviews. The survey includes the Patient-Reported Outcomes Measurement Information System (PROMIS) global health domain, a validated tool for assessing QoL among SCD patients and caregivers. The survey evaluated topics such as the impact of SCD on QoL, symptoms, impact of SCD on productivity at work/school, and treatment goals. A total of 1011 responses to the survey were collected; of these, 32.3% (n=327) of patients were currently being treated with HU (47% of adult and 17% of pediatric patients surveyed) and are the focus of this analysis. Amongst these patients, the median age (range) was 25 (5-83) years. Patients were responsible for reporting their SCD genotype: 58% of respondents were diagnosed with HbSS (n=189), 26% with HbSC (n=86), 2% with HbSβ0 thalassemia (n=8), and 5% with HbSβ+ thalassemia (n=15). Overall, 35% (adults, n=84; pediatric n=25) of patients in the eastern province were treated with HU, 27% (adult, n=71; pediatric, n=17) in the western province, 24% (adult, n=55; pediatric, n=24) in the southern province, and 14% (adult, n=29; pediatric, n=22) in the central province. Among HU-treated patients, 64% reported never ceasing/stopping treatment, 21% rarely ceased/stopped treatment and 15% of patients cease/stop treatment sometimes, often, or all the time. The most common reason for patients ceasing/stopping treatment was forgetting to take their medication (79%). Of patients who cease/stop treatment sometimes, often, or all the time (n=48), 17% of these stated that their medication was not available while 4% could not afford their medication. The mean (SD) number of VOCs reported by HU-treated patients in the 12 months prior to survey completion was 4.6 (4.09), while 32% (n=104) of these patients reported experiencing ≥5 VOCs in the same period. The most common SCD symptoms (other than VOCs) experienced by HU-treated patients were bone ache (reported by 92% of respondents), fatigue/tiredness (85%), headache (79%), anxiety (61%), joint stiffness (56%) and numbness/tingling (51%). Most HU-treated patients reported poor/fair or good health for all QoL metrics assessed (Figure 1). Almost half of patients (49%) reported that their SCD was impactful/extremely impactful on their emotional wellbeing. Of those treated with HU, 71% of patients reported that HU is effective in treating their SCD symptoms. However, 84% of patients report a desire for alternatives to their current pain management medication while 72% report concerns about the long-term side effects of HU. The most important treatment goals for these patients were improvement in quality of life (82%), improvement in overall symptoms (79%), and reduction in the number of severe pain crises and increase in the number of days free from severe pain crises (79%). A total of 48% of HU-treated patients were bothered by limitations in performing everyday activities; 48% and 55% of patients also reported that their SCD has prevented them from progressing in their career or education, respectively. Patients treated with HU reported missing a mean (SD) of 6.9 (10.4) hours of work in the 7 days prior to survey completion because of their SCD. Access to HU treatment continues to be a challenge, with approximately 70% of patients surveyed not receiving HU. Despite the observed clinical benefit in symptom control, patients receiving HU continue to experience disease burden including substantial impact on QoL and productivity at work/school (as evident from the treatment goals identified by patients). These data highlight the necessity of optimizing HU accessibility and treatment strategies to improve patient outcomes. These data also illustrate the need for novel therapeutic options and management strategies for patients with SCD to target patient-reported treatment goals. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

QOL-04. THE EFFECT OF ART THERAPY ON PATIENT AND CAREGIVER QUALITY OF LIFE IN NEWLY DIAGNOSED HIGH GRADE GLIOMA: A PROSPECTIVE PILOT STUDY

Abstract BACKGROUND High grade gliomas (HGGs) are incurable primary brain tumors, with limited survival times. Patients experience a variety of physical, cognitive, psycho-social changes that may negatively impact patient’s quality of life (QOL). Moreover, patient caregivers’ QOL are also often negatively impacted. As such, interventions to improve patient and caregiver QOL are needed. This study aims to evaluate whether art therapy positively impacts QOL among patients with HGG and their caregivers. METHODS This is a planned study with a target of 20 newly diagnosed or early treatment HGG patients and their caregiver matches. Each patient (with the option of one caregiver per patient) will participate in six (6) monthly art therapy sessions with licensed art therapists and structured curriculum. Patient and caregiver QOL will be assessed before, during, and after the six-month study period using validated scales including the HADS, EORTC-QL-C30, and COQL-C. The data will be aggregated following study completion for statistical modeling. DISCUSSION The positive impact of art therapy on QOL has previously been well established in patients with oncologic and neurologic conditions. However, there is a gap in our understanding of the impact of such therapies among patients with primary neuro-oncologic disorders. These patients face uniquely difficult physical, cognitive, and psychosocial challenges as a result of their unique disease process. It would stand to reason that newly diagnosed HGG patients may benefit from early enrolment and participation in these therapies to improve their overall QOL, particularly shortly after diagnosis and during active anti-cancer therapies. CONCLUSION To our knowledge, this is the first study to assess the impact of such intervention in newly diagnosed HGG patients. This pilot study will assess if early intervention with such therapies can lead to significant changes in patients and/or caregiver measurable QOL metrics.

Abstract 12772: Transcatheter Aortic Valve Replacement Improves Functional and Quality Of Life Outcomes in Patients With Low Gradient Aortic Stenosis

Introduction: Aortic stenosis (AS) is the most common valvular disease, and severe disease can significantly impact morbidity. Less data exists examining the functional and quality of life (QOL) effects of transcatheter aortic valve replacement (TAVR) in patients with severe low-gradient AS in both low-flow and normal-flow states. Hypothesis: Patients with symptomatic, severe, low-gradient AS would have improvements in functional and QOL outcomes at 30-days and 1-year following TAVR procedures. Methods: A single center, retrospective study examined symptomatic, severe, low-gradient AS variants. These had an aortic valve area of ≤1.0 cm 2 , mean transvalvular gradient &lt;40 mmHg, and peak aortic valve velocity of &lt;4 m/s. Classical low-flow low-gradient (CLFLG) AS was defined as a left ventricular ejection fraction (LVEF) &lt;50%. CLFLG group underwent dobutamine stress echo to assess AS severity (Stage D2). Paradoxical low-flow low-gradient (PLFLG) AS was defined as LVEF ≥50% and a stroke volume index (SVi) ≤35mL/m 2 (Stage D3). Normal-flow low-gradient (NFLG) AS was defined as LVEF ≥50% and SVi &gt;35mL/m 2 (Stage D4). The primary outcome was change in New York Heart Association (NYHA) classification and a 12-item Kansas City Cardiomyopathy Questionnaire overall score (KCCQ-OS) at 30-days and 1-year following TAVR. Results: A total of 191 patients were included for analysis: 46 CLFLG, 83 PLFLG, and 62 NFLG AS. At baseline, 130 (68%) had NYHA class III or IV symptoms. Mean baseline KCCQ-OS for all groups was 38±23 (Figure 1). The mean KCCQ-OS at 30-days and 1 year were 62±22 and 68±20. There were no significant differences in KCCQ-OS between the groups at 30-days and 1-year. Following TAVR, 92% and 88% of the cohort had NYHA class I or II symptoms at 30-days and 1 year. Conclusions: Patients with symptomatic, severe low-gradient AS had significant and sustained improvements in their functional and QOL metrics at 30-days and 1-year following TAVR procedures regardless of flow-state.

Evaluation of inguinal hernia repair using post-operative pain and quality of life metrics.

Elective inguinal hernia repair is a ubiquitous procedure that carries risks; chronic pain and impacts on quality of life (QoL) must be considered when advising patients around repair. The length of time from surgery date and impacts on quality of life are often limited to only a few years of follow-up and despite hernia repair being quite common, long-term outcomes are not often reported. A cohort of patients who had received Lichtenstein inguinal hernia repair over the previous 10years were contacted and surveyed using the Brief Pain Inventory Short Form (BPI) to assess chronic pain and its effects on their QoL. Patient and operative factors were correlated with pain through linear regression and t-test analysis provided statistical significance for mean comparisons (P < 0.05). The rate of chronic pain was 17.2% with recurrence of 3.1% at an average post-operative interval of 5.84years. Of the various metrics compared between groups, age was one of the only significant predictors of chronic pain with younger patients reporting higher pain. Further time from surgery also translated to significantly less pain with a difference of 1.3years. BPI respondents identified pain that interfered to varying degrees in different aspects of life but had relatively low average magnitudes (range: 1.82/10-2.91/10). These long-term considerations of post-surgical impact should be considered alongside potential benefits when advising patients about surgery and may help moderate post-operative expectations to optimize the outcome of common inguinal hernia repairs.

Clinical Improvements in Myelopathy Result in Improved Patient-Reported Outcomes Following Anterior Cervical Discectomy and Fusion.

Retrospective cohort study. To determine if myelopathy severity predicted the magnitude of improvement in health-related quality of life metrics following anterior cervical discectomy and fusion (ACDF). Surgery for myelopathy is primarily performed to halt disease progression. However, it is still controversial if these patients can expect significant health-related quality-of life improvements following ACDF. We explore the relationship between modified Japanese Orthopaedic Association (mJOA) improvements and its effect on other health-related quality-of life metrics. Patients undergoing ACDF for myelopathy were grouped based on preoperative mJOA scores into mild (15-17), moderate (12-14), and severe (<12) groups. Patients were subsequently categorized based on if they attained the minimum clinically detectable improvement (MCID) threshold for mJOA. Multivariate linear regression was performed to determine the magnitude of improvement in ∆patient-reported outcome measures. A total of 374 patients were identified for inclusion. Of those, 169 (45.2%) had mild myelopathy, 125 (33.4%) had moderate, and 80 (21.4%) had severe myelopathy. Only the moderate and severe groups had significant improvements in mJOA following surgery (mild: P =0.073, moderate: P <0.001, severe: P <0.001). There were no significant differences in the magnitude of improvement for any patient-reported outcome measure based on myelopathy severity, except for mJOA (mild: 0.27, moderate: 1.88, severe: 3.91; P <0.001). Patients meeting the MCID for mJOA had better ∆Short-Form 12 Mental Component Score (3.29 vs. -0.21, P =0.007), ∆Short-Form 12 Physical Component Score (6.82 vs. 1.96, P <0.001), ∆Visual Analog Scale Neck (-3.11 vs. -2.17, P =0.001), ∆Visual Analog Scale Arm (-2.92 vs. -1.48, P <0.001), ∆Neck Disability Index (-18.35 vs. -7.86, P <0.001), and ∆mJOA (3.38 vs. -0.56, P <0.001) compared with patients who did not. Worse baseline myelopathy severity predicts worse postoperative outcomes. However, baseline myelopathy severity is not predictive of the magnitude of postoperative improvement with the exception of mJOA. Patients who attain MCID improvement in mJOA had greater postoperative improvement for other health-related quality of life metrics.

Odevixibat: a promising new treatment for progressive familial intrahepatic cholestasis

ABSTRACT Introduction Progressive familial intrahepatic cholestasis (PFIC) refers to a group of heterogeneous, mostly autosomal recessive disorders resulting from the inability to properly form and excrete bile from hepatocytes. The resulting shared phenotype is one of hepatocellular cholestasis. Clinical management targeting refractory itch and surgical interventions to interrupt the enterohepatic circulation are often pursued with variable efficacy. Recent development of the family of IBAT inhibitor therapeutics has introduced a novel tool in the armamentarium for the treatment of PFIC. Areas covered Data from Phase 3 and 3 clinical trials were reviewed. The primary endpoints in most studies included effect on pruritus, serum bile acid levels, and quality of life metrics, with the duration of the study ranging between 24 and 72 weeks. Most common adverse events included diarrhea, vomiting, and elevation in transaminases. Expert opinion IBAT inhibition with therapeutics such as odevibixat have shown that it is well-tolerated and efficacious in mitigating itch and reducing serum bile acid levels. While the few early published trials with odevixibat have shown good efficacy, what remains to be seen is long-term, sustainable improvement and if or how these medications will supplement or replace the current medical and surgical therapies available for managing PFIC disorders.

Quality of Life After Orthopedic Procedures at Buraydah Central Hospital and King Fahad Specialist Hospital in Qassim Region, Saudi Arabia.

Aim Patients' perspectives on their condition and treatment, their sense of need for healthcare, and their preferences for care and outcomes are all addressed by quality of life metrics. Therefore, it is important to all health professionals and patients involved in orthopedic surgery. This study aimed to evaluate the quality of life after orthopedic procedures and how its results could potentially be used for future improvement. Methods This is a cross-sectional study conducted among patients who underwent orthopedic procedures at Buraydah Central Hospital (BCH) and King Fahad Specialist Hospital (KFSH). A self-administered questionnaire was distributed among the patients using a paper questionnaire. The questionnaire was composed of socio-demographic data (e.g. age, gender, education, etc.) and the 36-Item Short Form Survey (SF-36) to measure patients' quality of life. Results In this study, 215 patients were able to complete the survey (male 82.3% vs female 17.7%). The most common age group was 18 to 30 years old (30.2%) and the most common surgery performed on patients was thigh surgery (19.5%) and hand surgery (13%). The overall mean physical health score after the surgery was 51.1 (SD 11.8) higher than the mental health score (mean 47.7; SD 11.2).Poor quality of life was significantly more common among patients with chronic disease while poor physical functioning and general health subdomains were more associated among patients who underwent hand surgery. Conclusion Patients suffering from chronic diseases tend to exhibit a low quality of life as compared to other patients. The quality of life after the surgery is an important indicator of patient satisfaction which may have a direct impact on the future outlook of a patient. More research is needed to determine the overall quality of life in patients who underwent a surgical procedure in our region.