Changes In Symptom Burden Research Articles

A randomized controlled trial of a novel home-based palliative care program: A report of a trial that could not be completed.

In response to a growing need for accessible, efficient, and effective palliative care services, we designed, implemented, and evaluated a novel palliative care at home (PC@H) model for people with serious illness that is centered around a community health worker, a registered nurse, and a social worker, with an advanced practice nurse and a physician for support. Our objectives were to measure the impact of receipt of PC@H on patient symptoms, quality of life, and healthcare utilization and costs. We enrolled 136 patients with serious illness in this parallel, randomized controlled trial. Our primary outcome was change in symptom burden at 6 weeks. Secondary outcomes included change in symptom burden at 3 months, change in quality of life at 6 weeks and 3 months, estimated using a group t-test. In an exploratory aim, we examined the impact of PC@H on healthcare utilization and cost using a generalized linear model. PC@H resulted in a greater improvement in patient symptoms at 6 weeks (1.30 score improvement, n = 37) and 3 months (3.14 score improvement, n = 21) compared with controls. There were no differences in healthcare utilization and costs between the two groups. Unfortunately, due to the COVID-19 pandemic and a loss of funding, the trial was not able to be completed as originally intended. A palliative care at home model that leverages community health workers, registered nurses, and social workers as the primary deliverers of care may result in improved patient symptoms and quality of life compared with standard care. We did not demonstrate significant differences in healthcare utilization and cost associated with receipt of PC@H, likely due to inability to reach the intended sample size and insufficient statistical power, due to elements beyond the investigators' control such as the COVID-19 public health emergency and changes in grant funding.

Symptom burden among men treated for castration-resistant prostate cancer: a longitudinal study

ObjectivesDespite rapid expansion of treatments for metastatic castration-resistant prostate cancer (mCRPC) and the importance of symptom management for enhancing quality of life, few studies have focused on men’s experiences of...

Distinct Longitudinal Trajectories of Symptom Burden Predict Clinical Outcomes in End-Stage Liver Disease.

Little has been reported about the clinical relevance and trajectories of symptoms in end-stage liver disease (ESLD). The purpose of this prospective study was to identify trajectories of change in symptom burden over the course of 12 months in adults with ESLD. Patients were recruited from hepatology clinics at 2 healthcare systems. Validated measures were used to assess physical and psychological symptoms. Latent growth mixture modeling and survival and growth modeling were used to analyze the survey data. Data were available for 192 patients (mean age 56.5 ± 11.1 years, 64.1% male, mean Model for ESLD (MELD) 3.0 19.2 ± 5.1, ethyl alcohol as primary etiology 33.9%, ascites 88.5%, encephalopathy 70.8%); there were 38 deaths and 39 liver transplantations over 12 months. Two symptom trajectories were identified: 62 patients (32.3%) had high and unmitigated symptoms, and 130 (67.7%) had lower and improving symptoms. Patients with high and unmitigated symptoms had twice the hazard of all-cause mortality (subhazard ratio 2.53, 95% confidence interval: 1.32-4.83) and had worse physical ( P < 0.001) and mental quality of life ( P = 0.012) compared with patients with lower and improving symptoms. Symptom trajectories were not associated with MELD 3.0 scores ( P = 0.395). Female sex, social support, and level of religiosity were significant predictors of symptom trajectories ( P < 0.05 for all). There seems to be 2 distinct phenotypes of symptom experience in patients with ESLD that is independent of disease severity and associated with sex, social support, religiosity, and mortality. Identifying patients with high symptom burden can help optimize their care.

Changes in symptom burden and quality of life among women with uterine fibroids receiving relugolix combination therapy: a plain language summary.

This is a summary of findings from two research studies (known as clinical trials). The studies looked at how well a medicine called relugolix combination therapy worked in women with heavy menstrual bleeding (heavy bleeding during a period) with uterine fibroids (noncancerous or benign growths in the uterus). In this analysis of the studies, researchers looked at how patients self-reported their uterine fibroid symptoms before and after taking relugolix combination therapy. Researchers also looked at how patients self-reported the impact of uterine fibroids on their health-related quality of life before and after taking relugolix combination therapy. Women took either relugolix combination therapy or placebo (a pill that contains no medicine) by mouth once daily for 24weeks. Women completed the Uterine Fibroid Symptom and Quality of Life questionnaire (where "quality of life" refers to the women's health-related quality of life related to uterine fibroids) before, during, and after treatment. The questionnaire let researchers see if the women felt that relugolix combination therapy decreased the burden of uterine fibroid symptoms and improved the women's health-related quality of life related to uterine fibroids. More women said that they felt less distress due to their uterine fibroid symptoms and that their health-related quality of life related to uterine fibroids was better after taking relugolix combination therapy compared with women who took placebo. Relugolix combination therapy may lessen distress associated with uterine fibroid symptoms and improve health-related quality of life related to uterine fibroids.

The use of the "group traumatic episode protocol (G-TEP) " in an outpatient randomized and hospital setting: Implementation and effects in heterogeneous patient samples.

The resource-based eye movement desensitization and reprocessing group intervention group traumatic episode protocol G-TEP showed promising results in reducing posttraumatic stress disorder, depression, and anxiety. Using a high structured G-TEP worksheet, the patients conduct self-bilateral stimulation while (re)processing their targets. We examined the effectiveness on multiple symptoms and practicability of G-TEP in an outpatient setting and its feasibility in hospital treatment. Study A: Forty-four patients suffering from different symptoms and diagnoses received four ambulatory G-TEP sessions within three eye movement desensitization and reprocessing-G-TEP sessions in a randomized delayed waitlist control group design. We investigate the improvements in symptoms and advantages of G-TEP as an upstream treatment. Study B: The targeted changes in symptom burden in 23 patients and the implementation process of G-TEP as an additional treatment option in a psychiatric hospital were examined. The measures used were Impact of Event Scale-Revised, Beck Depression Inventory-II, Brief Symptom Check List, and the Questionnaire on Dissociative Symptoms at pre- and posttreatment and follow-up. Both studies showed significant and long-lasting reductions in subjective distress and concomitant impairments. The distressing experiences became more "ego-syntonic," levels of avoidance decreased, abilities to manage negative emotions got strengthened, and they gained hope. All effects had a positive impact on subsequent treatment processes; some patients already went free of symptoms only with this short G-TEP treatment. G-TEP effectively alleviates symptoms caused by stressful experiences. G-TEP can be used as a stand-alone intervention in outpatients and can easily be integrated into the offer of multimodal therapy in a psychiatric ward. It can prevent the exacerbation of symptoms and chronification of disease and should be implemented into the (German) health system. (PsycInfo Database Record (c) 2024 APA, all rights reserved).

Abstract PO3-01-10: Changes in Patient-Reported Outcomes with 3 Months of Pre-operative Endocrine Therapy in the POWER Trial

Abstract Background: The Pre-Operative Window of Endocrine Therapy to Inform Radiation Therapy Decisions Trial (POWER, NCT04272801) aims to determine whether 3 months of pre-operative endocrine therapy (pre-ET) affects decision-making regarding adjuvant radiation therapy (RT) among older women with early-stage, estrogen receptor (ER) positive breast cancer. The goal of the present study is to evaluate changes in patient-reported outcomes (PROs) during the course of pre-ET. We hypothesize that PROs will change over the course of pre-ET and that those changes can be used to identify patients who are at-risk for long-term noncompliance to adjuvant ET and who may benefit from RT. Methods: Participants in the POWER Trial receive 3 months of pre-ET prior to breast-conserving surgery and final treatment decisions regarding adjuvant RT. Health-related quality of life (HRQOL) (EORTC QLQ-C30 and QLQ-BR23), depression (Center for Epidemiologic Studies Depression Scale Revised), and general symptom burden (Breast Cancer Prevention Trial Symptom Checklist (BCPT-SCL)) are assessed at baseline and at day 90 after pre-ET. Perceived sensitivity to medicine (Perceived Sensitivity to Medicine (PSM) survey) is assessed at baseline. The BCPT-SCL was used to examine common ET side effects, including hot flashes, musculoskeletal symptoms, cognitive problems, weight gain, and bladder control. Graphical methods are used to display results. Results: The first 46 participants who completed pre-ET in the POWER trial were included in this preliminary assessment. From baseline to day 90, there was notable variability in HRQOL and no definitive shift towards worsening HRQOL with a median change (IQR) of 0 (-8.33, 8.33). Similarly, the median change in symptom profiles for the whole cohort was 0 over the 90 days of pre-ET. However, a subset of patients experienced changes in symptom burden throughout the 90-day period. Approximately one-fourth of participants reported an increase in hot flashes and musculoskeletal symptoms from baseline to day 90. There does not appear to be worsening fatigue, depression scores, cognitive problems, or weight gain. After stratifying by self-perceived sensitivity to medicine, patients with high PSM scores have a greater median change (0.5 and 0.33) in symptoms compared to those with low PSM scores (0 and 0) for hot flashes and musculoskeletal symptoms, respectively (Figure 1). Conclusions: When looking at this cohort, there was no change in symptoms or quality of life after the 3-month course of pre-ET. Evaluation of the changes in PROs identified a subset of participants with worsening symptoms after pre-ET. Future analyses, when trial accrual is complete, will examine the relationship between PROs and long-term adherence to adjuvant ET and will elucidate if the patients who experience worsening BCPT-SCL symptoms during the 90-day period are more likely to opt for RT. Figure 1 Change in symptoms after pre-ET Change in hot flash burden and musculoskeletal symptom burden is greater in patients with high perceived sensitivity to medicine. Citation Format: Trish Millard, Lena Turkheimer, Christiana Brenin, David Brenin, Patrick Dillon, Einsley Janowski, Anneke Schroen, Gina Petroni, Shayna Showalter. Changes in Patient-Reported Outcomes with 3 Months of Pre-operative Endocrine Therapy in the POWER Trial [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO3-01-10.

Mental health, COVID-19 burden and quality of life of kidney transplant recipients two years after the COVID-19 pandemic.

Few studies have evaluated the psychological distress of COVID-19 in kidney transplantation and the psychological impact that the COVID-19 pandemic has had on kidney transplant recipients is not yet well understood. The present study aimed to investigate the change in symptom burden and health-related quality of life in the two years after initial assessment, by outlining the change over time of symptoms at 12 and 24 months of follow-up. This is a follow-up study. We performed a study published in 2021 (phase 1 of COVID-19); of the 89 kidney transplant recipients evaluated in this study, 60 completed the 12 months follow-up (March 2021 June 2021, phase 2 of COVID-19) and 57 completed the 24 months follow-up (March 2022 June 2022, post COVID-19). The same tools as in previous study were administered: the ad hoc questionnaire on emotional state and psychophysical well-being during COVID-19, the Middlesex Hospital Questionnaire (MHQ) to provide a simple and rapid quantification of the psychological and somatic symptoms and the Short Form Health Survey 36 (SF-36) was used to assess health-related quality of life. Compared to the first and second phase of COVID-19, the mean score of quality of life variables were higher in the post COVID-19 phase; thus the recipients physical health, mental health and their perception of their general health improved. Regarding the psychopathology variables the levels of Anxiety, Depression and Phobia in the Post COVID-19 phase decreased, while the Somatization score was higher. Lastly, burden of COVID-19 scores in the third phase, significantly decreased. Our study highlights a significant association between mental health and the burden of COVID-19 pandemic in kidney transplant recipients. This study showed, a significant worsening, over time, of some specific symptoms, such as somatization and phobias. However, the results showed that depressive symptoms improved during the study period. Long-term monitoring of kidney transplant recipients therefore remains fundamental. These results confirmed the need to provide integrated multidisciplinary services to adequately address the long-term effects of the COVID-19 pandemic on the mental health of the most vulnerable subjects.

Evaluating the effect of a mobile-based symptom monitoring system for improving physical function in patients with cancer during chemotherapy: study protocol for a multicentre randomised controlled trial

IntroductionSymptoms due to chemotherapy are common in patients with cancer. Cancer-related symptoms are closely associated with the deterioration of physical function which can be associated with decreased quality of life...

Abstract 2135: Racial disparities in symptom burden among women receiving early stage breast cancer chemotherapy

Abstract Purpose: The purpose of this study was to compare prevalent symptoms (pain, fatigue, and physical function) and changes over time between Black and White women undergoing chemotherapy for early-stage breast cancer (ESBC) employing a longitudinal, repeated measures design over the course of ESBC. Methods: Data collected as per protocol of 1R01MD012245, comparing racial differences in chemotherapy tolerance and dose modifications during chemotherapy treatment at baseline, midpoint, and endpoint, tailored to each patient's individual chemotherapy schedule. Race was collected by patient self-report and chart review. Symptom burden (fatigue, pain, and physical functioning) was measured by the PROMIS-29 Profile v 2.0). Area deprivation was measured using the Area Deprivation Index (ADI). Linear mixed models were used to assess racial differences in symptom burden across timepoints, and changes in symptom burden over time, adjusting for ADI. Results: A total of 147 patients participated in this study (36% Black and 64% white). Pain: Black patients reported significantly higher pain scores than White patients at pretherapy (MD=3.7, p=.034), midpoint (MD=5.8, p: 0.002), and endpoint (MD=7.8, p&amp;lt;.001). Even after adjusting for area deprivation, this disparity persisted (F(1, 390)=29.43, p&amp;lt;.001), with Black patients having higher pain scores at midpoint (MD=4.7, p: 0.02) and endpoint (MD=7.8, p&amp;lt;.001). Pain scores worsened over time for Black patients, increasing significantly by the end of chemotherapy (MDT1-T3 =6.2, p=.010; MDT2-T3=5.8, p=.024). No significant changes in pain scores were observed over time among White patients. Fatigue: After adjusting for area deprivation, fatigue significantly increased at the endpoint (MDT1-T3=8.9, p&amp;lt;.001) for Black patients. Among White patients, fatigue increased at midpoint (MDT1-T2=5.7) and continued to worsen at the endpoint (MDT1-T3=10.1, p&amp;lt;.001; MDT2-T3=4.3, p=.017). Physical Function: Black patients had significantly lower physical function than White patients at the midpoint (MD=4.0, p=.027). By the end of chemotherapy, physical function deteriorated significantly for Black patients (MDT1-T3=7.8, p&amp;lt;.001), and White patients (MDT1-T3=7.7, p&amp;lt;.001; MDT2-T3=4.8, p=.002). Correlations between the ADI and symptom severity and physical function revealed that living in more deprived areas was associated with greater pain severity (r=-.227, p=.002, 56.3 vs. 50.1) and worse physical function (r=-.190, p=-.035, 40.6 vs. 42.4) at the completion of chemotherapy. Conclusions: The findings indicate that race and area deprivation are associated with symptom trajectories over the course of ESBC chemotherapy. Further research is warranted on how to best assess and relieve symptom burden among Black ESBC patients so that existing disparities in treatment and treatment outcomes are not perpetuated. Citation Format: Elham Nasrollahi, Hiba Abujaradeh, Susan Mazanec, Julia O'Brien, Isabelle M. Schlemmer, Margaret Rosenzweig. Racial disparities in symptom burden among women receiving early stage breast cancer chemotherapy [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 2135.

Fifteen-year symptomatic outcome of patients with nonactionable motor findings on high-resolution manometry.

High-resolution manometry (HRM) is performed for evaluation of esophageal symptoms, but patient outcome is unclear when no actionable motor disorder is identified. We evaluated long-term symptomatic outcome of patients with nonactionable HRM findings. Patients who underwent (HRM) studies in 2006-2008 were tracked. Patients with achalasia spectrum disorders, foregut surgery before or after HRM, and incomplete symptom documentation were excluded. Symptom questionnaires assessing dominant symptom intensity (DSI, product of symptom severity and frequency recorded on 5-point Likert scales) and global symptom severity (GSS, from 10 cm visual analog scale) were repeated. Change in symptom burden was compared against HRM motor findings using Chicago Classification 4.0 (CCv4.0), applied retroactively to 2006-2008 data. Overall, 134 patients (median age 68 years, 64.5% female) could be contacted. The majority (73.1%) had normal motility; others had ineffective esophageal motility (8.2%), esophagogastric junction outflow obstruction (13.4%), hypercontractile esophagus (3.0%), or absent contractility (2.2%), none managed invasively. Over 15 years of follow-up, DSI decreased from 8.0 (4.0-16.0) to 1.0 (0.0-6.0) (p < 0.001) and GSS improved from 5.5 (3.3-7.7) to 2.0 (0.0-4.0) (p < 0.001); improvement was consistent across CCv4.0 diagnoses and subgroups. The majority (82.8%) reported improvement over time, and antisecretory medication was the most effective intervention (83.0% improvement). There was no difference in medication efficacy (p = 0.75) or improvement in symptoms (p = 0.20) based on CCv4 diagnosis. Esophageal symptoms improve with conservative symptomatic management over long-term follow-up when no conclusive obstructive motor disorders or achalasia spectrum disorders are found on HRM.

Pain Catastrophizing in Cancer Patients

Background: Pain catastrophizing is a group of negative irrational cognitions in the context of anticipated or actual pain. The aim of this study was to decipher the possible role of catastrophism on pain expression and outcomes after a comprehensive palliative care treatment. Methods: A consecutive sample of patients with uncontrolled pain was assessed. Demographic characteristics, symptom intensity included in the Edmonton symptom assessment system (ESAS), and opioid drugs used were recorded at admission (T0). The Pain Catastrophizing Scale (PCS) was measured for patients. Patients were also asked about their personalized symptom goal (PSG) for each symptom of ESAS. One week after a comprehensive palliative care treatment (T7), ESAS and opioid doses used were recorded again, and the number of patients who achieved their PSG (PSGR) were calculated. At the same interval (T7), Minimal Clinically Important Difference (MCID) was calculated using patient global impression (PGI). Results: Ninety-five patients were eligible. A significant decrease in symptom intensity was reported for all ESAS items. PGI was positive for all symptoms, with higher values for pain, poor well-being, and poor sleep. Only the rumination subscale of catastrophism was significantly associated with pain at T0 (B = 0.540; p = 0.034). Conclusions: Catastrophism was not associated with the levels of pain intensity, PSG, PSGR, and PGI for pain, except the rumination subscale that was associated with pain intensity at T0. A comprehensive palliative care management provided the relevant changes in symptom burden, undoing the pain expression associated with rumination.

Randomized Controlled Trial of the Effect of an Exercise Rehabilitation Program on Symptom Burden in Maintenance Hemodialysis: A Clinical Research Protocol.

People receiving hemodialysis experience high symptom burden that contributes to low functional status and poor health-related quality of life. Management of symptoms is a priority for individuals receiving hemodialysis but limited effective treatments exist. There is emerging evidence that exercise programming can improve several common dialysis-related symptoms. The primary aim of this study is to evaluate the effect of an exercise rehabilitation program on symptom burden in individuals receiving maintenance hemodialysis. Multicenter, randomized controlled, 1:1 parallel, open label, prospective blinded end point trial. Three facility-based hemodialysis units in Winnipeg, Manitoba, Canada. Adults aged 18 years or older with end-stage kidney disease receiving facility-based maintenance hemodialysis for more than 3 months, with at least 1 dialysis-related symptom as indicated by the Dialysis Symptom Index (DSI) severity score >0 (n = 150). Supervised 26-week exercise rehabilitation program and 60 minutes of cycling during hemodialysis thrice weekly. Exercise intensity and duration were supervised and individualized by the kinesiologist as per participant baseline physical function with gradual progression over the course of the intervention. Usual hemodialysis care (no exercise program). Our primary outcome is change in symptom burden at 12 weeks as measured by the DSI severity score. Secondary outcomes include change in modified DSI severity score (includes 10 symptoms most plausible to improve with exercise), change in DSI severity score at 26 and 52 weeks; time to recover post-hemodialysis; health-related quality of life measured using EuroQol (EQ)-5D-5L; physical activity behavior measured by self-report (Godin-Shepherd questionnaire) and triaxial accelerometry; exercise capacity (shuttle walk test); frailty (Fried); self-efficacy for exercise; and 1-year hospitalization and mortality. Change in primary outcome will be compared between groups by independent 2-tailed t test or Mann-Whitney U test depending on data distribution and using generalized linear mixed models, with study time point as a random effect and adjusted for baseline DSI score. Similarly, change in secondary outcomes will be compared between groups over time using appropriate parametric and nonparametric statistical tests depending on data type and distribution. The COVID-19 pandemic restrictions on clinical research at our institution delayed completion of target recruitment and prevented collection of accelerometry and physical function outcome data for 15 months until restrictions were lifted. The application of an exercise rehabilitation program to improve symptom burden in individuals on hemodialysis may ameliorate common symptoms observed in individuals on hemodialysis and result in improved quality of life and reduced disability and morbidity over the long term. Importantly, this pragmatic study, with a standardized exercise intervention that is adaptable to baseline physical function, addresses an important gap in both clinical care of hemodialysis patients and our current knowledge.

Symptom Burden in Idiopathic Multicentric Castleman Disease: Protocol for the Development of a Novel Standardized Patient Reported Outcome Measure

Background and Significance Idiopathic multicentric Castleman disease (iMCD) is a rare lymphoproliferative disorder with a high symptom burden. These symptoms impact many aspects of daily life for people living with iMCD, including work/education, social life, travel, mobility, personal relationships and sexual functioning. Despite this significant impact [Shupo F et al. Hemasphere 2022, Jun 23;6(Suppl):802-803], no validated disease-specific measure exists to assess symptom burden in iMCD. This is a major unmet need, considering the mainstay of iMCD treatment is symptom control and preventing serious complications. A novel symptom burden scale for iMCD is therefore timely and important and would be a beneficial clinical tool, both in routine clinical practice and clinical trials, for the comprehensive assessment of disease burden at diagnosis, during progression, and for treatment efficacy. Study Design and Methods A protocol for the development of a novel, standardized, patient reported outcome measure (PROM) for assessing symptom burden in iMCD has been developed and approved by a multi-stakeholder group (including patients, clinicians, industry representatives, and researchers). A four-stage development process has been proposed, alongside the generation of a novel patient advisory group and wider multi-stakeholder advisory group. In stage one, draft PROM content will be generated from existing literature and expert opinions. In stage two, the content validity of the draft PROM will be assessed in online qualitative interviews with people living with iMCD, with any revisions to the content decided in consultation with patient and expert advisors. In stage three, the revised PROM will be administered alongside existing measures of symptom burden and/or health-related quality of life to evaluate its psychometric performance and inform decisions on content for the final PROM, using classical test theory and/or Rasch psychometric analyses. The PROM will be finalized based on the qualitative and quantitative evidence generated in consultation with project advisors. Finally, in stage four, the PROM will be re-administered to observe change in symptom burden over time. This will be complemented with qualitative interviews in a mixed methods design to estimate a minimally clinically important difference (MCID) for the measure. The project will involve investigators and iMCD patients from the U.S., U.K., Canada, Australia, New Zealand and Brazil. Conclusion This is an international effort to develop and validate a novel iMCD symptom burden PROM through a collaboration of iMCD patients, clinical, health economics, public health, statistical, psychometrics, and industry experts. The development of the measure follows U.S. Food and Drug Administration (FDA) regulatory guidance, with modifications for rare diseases as required. Special Considerations This project has been submitted to clintrials.gov, documentation sent to ASH and Secretary, and awaiting approval of the submission.

Relugolix combination therapy in Black/African American women with symptomatic uterine fibroids: LIBERTY Long-Term Extension study

BackgroundIn the LIBERTY Long-Term Extension study, once-daily relugolix combination therapy (40 mg relugolix, estradiol 1 mg, norethindrone acetate 0.5 mg) significantly improved uterine fibroid-associated heavy menstrual bleeding over the 52-week treatment period in the overall study population. Objectives(s)Black/African American women typically experience greater extent of disease and symptom burden of uterine fibroids versus other racial groups and have traditionally been underrepresented in clinical trials. This secondary analysis aimed to assess the efficacy and safety of relugolix combination therapy in the subgroup population of Black/African American women with uterine fibroids in the LIBERTY Long-Term Extension study. Study designBlack/African American premenopausal women (aged 18–50 years) with uterine fibroids and heavy menstrual bleeding who completed the 24-week randomized, placebo-controlled, double-blind LIBERTY 1 (NCT03049735) or LIBERTY 2 (NCT03103087) trials were eligible to enroll in the 28-week LIBERTY Long-Term Extension (NCT03412890), in which all women received once-daily, open-label relugolix combination therapy. The primary endpoint of this subanalysis was the proportion of Black/African American treatment responders: women who achieved a menstrual blood loss volume of <80 mL and at least a 50% reduction in menstrual blood loss volume from pivotal study baseline to the last 35 days of treatment by pivotal study randomized treatment group. Secondary outcomes included rates of amenorrhea, as well as changes in symptom burden and quality of life. ResultsIn total, 241/477 (50.5%) women enrolled into the LIBERTY Long-Term Extension study self-identified as Black or African American. In Black/African American women receiving continuous relugolix combination therapy for up to 52 weeks, 82.9% [n=58/70; 95% confidence interval: 72.0%, 90.8%] met treatment responder criteria for reduction in heavy menstrual bleeding (primary endpoint). A substantial reduction in menstrual blood loss volume from pivotal study baseline to Week 52 was demonstrated (least squares mean percentage change: 85.0%); 64.3% of women achieved amenorrhea; 59.1% of women with anemia at the pivotal study baseline achieved a substantial improvement (>2 g/dL) in hemoglobin levels; and decreased symptom severity and distress due to uterine fibroid-associated symptoms was demonstrated, as well as improvements in health-related quality of life through 52 weeks. The most frequently reported adverse events during the cumulative 52-week treatment period were hot flush (12.9%), headache (5.7%) and hypertension (5.7%). Bone mineral density was preserved through 52 weeks. Conclusion(s)Once-daily relugolix combination therapy improved uterine fibroid-associated heavy menstrual bleeding in the majority of Black/African American women who participated in the LIBERTY Long-Term Extension. The safety and efficacy profile of relugolix combination therapy in Black/African American women was consistent with previously published results from the overall study population through 52 weeks. Findings from this subanalysis will assist shared decision-making by helping providers and Black/African American women understand the efficacy and safety of relugolix combination therapy as a pharmacologic option for the management of uterine fibroid-associated symptoms.

No Increase in Symptoms Toward the End of the Ocrelizumab Infusion Cycle in Patients With Multiple Sclerosis: Symptom Burden on Ocrelizumab: A Longitudinal Study (SymBOLS).

Some patients with multiple sclerosis (MS) receiving ocrelizumab (OCR) report worsening symptoms toward the end of the 6-month infusion cycle ('wearing off'). The objective of our study was to comprehensively assess changes in symptom burden across 2 consecutive OCR infusion cycles. SYMptom Burden on Ocrelizumab, a Longitudinal Study (SymBOLS; NCT04855617) was an investigator-initiated, 2-center study of patients with MS starting or receiving OCR. Patients' symptoms were assessed with NeuroQoL short forms, SymptoMScreen, and Work Productivity and Activity Impairment Questionnaire at the start-cycle, mid-cycle, and end-cycle time points in each of the 2 infusion cycles. Symptom scores at the 3 time points within each cycle were compared with repeated-measures ANOVA or the Friedman rank-sum test for non-normal variables. The proportions of patients with a meaningful symptomatic change from the start to the end of each infusion cycle were calculated, and patients whose symptoms improved, worsened, and stayed the same from the start to the end of the cycle were compared with respect to demographic and clinical characteristics. One hundred three patients with MS provided longitudinal data for analyses (mean age [SD]: 46.7 [12.2] years, 68% female, 33% non-White, disease duration: 15.5 [5] years, 41% with the Extended Disability Status Scale score >3). On a group level, NeuroQoL and SymptoMScreen scores mostly remained stable or even improved slightly toward the end of each cycle. On an individual level, symptoms remained unchanged across either cycle for most patients, and meaningful symptom worsening from the start to the end of the cycle was no more common than improvement. Meaningful change in symptoms in both cycles was very rare and generally in the direction of improvement toward the end cycle. Despite the lack of evidence for symptom worsening with a longer time from infusion, 54% of patients endorsed feeling of "wearing off" at least sometimes, most commonly as an increase in fatigue. Our prospective study failed to uncover evidence for the worsening of symptoms with a longer time from OCR infusion. These findings cast doubt on the existence of wearing off as a physiologic phenomenon in OCR-treated patients with MS. The perception of wearing off is likely the result of natural fluctuations in MS symptoms and attribution bias.

Impact of an outpatient palliative care consultation and symptom clusters in terminal patients at a tertiary care center in Pakistan

BackgroundPatients with terminal diseases may benefit physically and psychosocially from an outpatient palliative care visit. Palliative care services are limited in Pakistan. An improved understanding of the symptom clusters present in our population is needed. The first outpatient palliative care center in Karachi, Pakistan, was established at our tertiary care institution. The primary aim of this study was to evaluate the impact of a palliative care outpatient consultation on symptom burden in patients with a terminal diagnosis. The secondary aim was to analyze the symptom clusters present in our population.MethodsPatients with a terminal diagnosis referred to our outpatient palliative department between August 2020-August 2022 were enrolled. The Edmonton Symptom Assessment Scale (ESAS) questionnaire was administered at the initial visit and the first follow-up visit at one month. Change in symptom burden was assessed using a Wilcoxon signed ranks test. A principal component analysis with varimax rotation was performed on the symptoms reported at the initial visit to evaluate symptom clusters. The palliative performance scale (PPS) was used to measure the performance status of palliative care patients.ResultsAmong the 78 patients included in this study, the average age was 59 ± 16.6 years, 52.6% were males, 99% patients had an oncological diagnosis, and the median duration between two visits was 14 (Q1-Q3: (7.0, 21.0) days. The median PPS level was 60% (Q1-Q3: 50–70). Overall, ESAS scores decreased between the two visits (6.0 (2.8, 11.0), p < 0.001) with statistically significant improvement in pain (5.0 vs. 2.5, p < 0.001), loss of appetite (5.0 vs. 4.0, p = 0.004), depression (2.0 vs. 0.0, p < 0.001), and anxiety (1.5 vs. 0.0, p = 0.032). Based on symptoms at the initial visit, 3 clusters were present in our population. Cluster 1 included anxiety, depression, and wellbeing; cluster 2 included nausea, loss of appetite, tiredness, and shortness of breath; and cluster 3 included drowsiness.ConclusionAn outpatient palliative care visit significantly improved symptom burden in patients with a terminal diagnosis. Patients may benefit from further development of outpatient palliative care facilities to improve the quality of life in terminally ill patients.

Improving palbociclib adherence among women with metastatic breast cancer using a CONnected CUstomized Treatment Platform: A pilot study.

To pilot test a mobile health intervention using a CONnected CUstomized Treatment Platform that integrates a connected electronic adherence monitoring smartbox and an early warning system of non-adherence with bidirectional automated texting feature and provider alerts. In total, 29 adult women with hormone-receptor-positive, human epidermal growth factor receptor 2-negative metastatic breast cancer and a prescription for palbociclib were asked to complete a survey and participate in a CONnected CUstomized Treatment Platform intervention, including use of a smartbox for real-time adherence monitoring, which triggered text message reminders for any missed or extra dose, and referrals to (a) participant's oncology provider after three missed doses or an episode of over-adherence, or (b) a financial navigation program for any cost-related missed dose. Use of smartbox, number of referrals, palbociclib adherence, CONnected CUstomized Treatment Platform usability measured by System Usability Scale, and changes in symptom burden and quality of life were assessed. Mean age was 57.6 and 69% were white. The smartbox was used by 72.4% of participants, with palbociclib adherence rate of 95.8%±7.6%. One participant was referred to oncology provider due to missed doses and one was referred to financial navigation. At baseline, 33.3% reported at least one adherence barrier including inconvenience to get prescription filled, forgetfulness, cost, and side effects. There were no changes in self-reported adherence, symptom burden or quality of life over 3 months. CONnected CUstomized Treatment Platform usability score was 61.9 ± 14.2. The CONnected CUstomized Treatment Platform interventions is feasible, resulting in a high palbociclib adherence rate without any decline in overtime. Future efforts should focus on improving usability.

Changes in symptom burden from 2019 to 2021 amongst community-dwelling older adults in Finland.

Symptom burden causes suffering amongst older adults and is associated with healthcare visits and prognosis. We evaluated the prevalence of 10 symptoms and changes in symptom burden amongst home-dwelling older adults in 2019 and 2021 using Finnish cohort data. We analysed factors associated with symptom burden increase during follow-up. Altogether 1,637 people aged 75+ participated in the Helsinki Ageing Study postal survey in 2019, where they reported the presence of 10 common symptoms over the past 2 weeks. Of them, 785 participated in a follow-up in 2021, where the same symptoms were queried. We compared the prevalence of various symptoms and symptom burden scores in the 2-year interval and evaluated factors associated with increased symptom burden during this time. Of participants, 33% reported at least one daily symptom in 2019 versus 44% in 2021. Symptom burden increased by a mean ratio of 1.29 between 2019 and 2021. The most common symptoms were joint pain, back pain, urinary incontinence and fatigue. The prevalence of four symptoms increased between 2019 and 2021: joint pain, urinary incontinence, dizziness and shortness of breath. Higher age, reduced functional capacity and comorbidities were associated with higher odds of symptom burden increase during follow-up. Psychological well-being (PWB) was strongly associated with lower odds of symptom burden increase in the logistic regression model. Symptom burden increased in our cohort aged 75+ between 2019 and 2021 before and during the COVID-19 pandemic. PWB was associated with lower odds of acquiring additional symptoms over time.

Determinants of quality of life improvements in anxiety and depressive disorders-A longitudinal study of inpatient psychotherapy.

Quality of life (QoL) is substantially impaired in patients with anxiety disorders (AD) and depressive disorders (DD) and improvements in symptom burden after psychotherapy are not always paralleled by similar improvements in QoL. So far, little is known about treatment outcome in terms of QoL and predictors of QoL improvements following inpatient psychotherapy with a focus on cognitive behavior therapy (CBT). The current study aimed at investigating the relationship between changes in symptoms and QoL across different life domains. Additionally, predictors of a positive treatment outcome were evaluated. 122 patients with AD and/or DD undergoing an 8-weeks inpatient CBT program completed self-report measures of psychopathological symptoms and QoL at pre- and post-treatment. Mixed effects models were used to investigate changes, a confirmatory factor analysis was applied to analyze the latent factor structure of the anxiety sensitivity index and binary logistic regression analyses were performed for predictors of QoL improvements. Patients showed moderate to strong decreases in anxious and depressive symptoms and moderate to strong improvements in general QoL, particularly in the psychological and physical QoL subdomains. Changes in symptom burden correlated most strongly with psychological and physical QoL. In addition, poor QoL before treatment and low levels of specific anxiety sensitivity symptoms (items 1 and 5) significantly predicted improvement in QoL. Patients with poor QoL who are not as inhibited to openly express their anxious feelings particularly benefit from inpatient psychotherapy (individual and group) to improve their QoL. In contrast, our research suggests that patients who are too anxious to openly express their nervousness should receive additional social skills training, more focused treatment to build sufficient self-confidence to better engage in the treatment program.

Symptom Burden in Glioblastoma: A Prospective Pilot Study from Diagnosis to First Progression

Introduction: The evaluation of symptom burden, performance status, and neurological function is still challenging in glioblastoma (GBM) patients. Patients may suffer from a wide spectrum of neurological symptoms like cognitive deficits, aphasia, or hemiparesis, which interfere to report to comprehensive questionnaires. However, an integrated and reliable neuro-oncological assessment is the key in the clinical management and in the evaluation of treatment benefits for GBM patients. Methods: We implemented an easy-to-use clinical toolkit for the prospective assessment and follow-up evaluation of GBM patients using the Karnofsky performance status (KPS), the National Institute of Health Stroke Scale (NIH-SS), and simple scores for the evaluation of key symptoms like fatigue, depression, and headache. Results: We prospectively followed 50 patients. The composite score (headache, depression, and fatigue), fatigue alone, the NIHSS, and the KPS were suitable biomarkers to evaluate symptom burden in GBM patients and indicate clinical disease progression. Discussion/Conclusion: The proposed clinical toolkit seems feasible in routine clinical practice and reflects changes in symptom burden in different stages of the postsurgical course of GBM patients in this monocentric clinical pilot trial.