Study Of Patient-reported Outcomes Research Articles

Stereotactic Magnetic Resonance–Guided Daily Adaptive Radiation Therapy for Localized Prostate Cancer: Acute and Late Patient-Reported Toxicity Outcomes

ObjectiveTo report acute and late bowel, urinary and sexual dysfunction patient reported outcome measures (PROMS), amongst localised prostate cancer patients who underwent Stereotactic MRI-guided daily adaptive radiotherapy (SMART). MethodsAll patients who completed a baseline 12 item Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), before undergoing 36.25 Gy in 5 fractions by SMART, were subsequently followed up with the same graded questionnaire at set time points. Latest PSA levels were recorded. The percentage of patients who reported no change from their baseline AE or reported a new ≥ ‘frequent’ or’ severe’ AE grade during follow up was calculated. The maximum PRO-CTCAE grade for each item was recorded for each patient. The percentage of toxicity levels for each separate AE item at set time points were calculated. ResultsThe total number of patients was 69 with a median follow-up of 27 months. Median age of cohort 73 (range 54-85). The median pre-treatment PSA, T stage and Gleason score was 7.5 mmol/l (4.5-32 mmol/l), T2b (T2-T3b) and Gleason Score of 7 (3+4, range: 6-9) respectively. No patient had biochemical failure during follow up. Regarding bowel symptoms, >80% of men reported no change from baseline toxicity during follow up. New ≥ frequent diarrhoea was reported in 9% of patients. ‘Almost constant’ diarrhoea peaked at 1 month but was absent at >33 months. Regarding urinary symptoms, increased urinary urgency was the most common complaint (39%). 20% of men reported new ≥ frequent urinary urgency incidence peaking at 1 month but absent at >33 months. New “severe” sexual dysfunction was seen in 26% of patients and was persistent at >33 months. ConclusionOur study is one the largest patient reported outcomes study post prostate SMART. It shows acceptable levels of toxicity even up to 2 years post treatment.

A real-world, patient-reported outcomes study in patients with platinum-sensitive recurrent ovarian cancer receiving long-term maintenance therapy with niraparib.

e23164 Background: Niraparib has been approved for the maintenance treatment of newly diagnosed advanced and platinum-sensitive recurrent ovarian cancer (PSR OC). The phase 3 PRIMA and NOVA studies showed that maintenance niraparib did not worsen quality of life (QoL) compared with placebo. However, no QoL data have been reported for Asian OC patients treated with niraparib. Here, we report patient-reported outcomes (PROs) for PSR OC patients who received niraparib as maintenance therapy for more than two years in a real-world setting. Methods: This prospective, real-world study included OC patients participating in the Niraparib Patient Assistance Program (PAP) in China (NCT06037213). Eligible patients had histologically confirmed PSR epithelial OC, fallopian tube, or primary peritoneal cancer, had been maintained on niraparib for more than two years without disease progression and were still receiving niraparib within 28 days. PROs were assessed every 4-6 weeks for three times using the EQ-5D-5L questionnaire and the FOSI. In addition, a 6-question niraparib medication questionnaire including starting dose, adaptive dose, dose adjustments and reasons, missed doses, concomitant medications was administered at baseline. Results: The PAP enrolled 3154 PSR OC patients, 1151 had been on niraparib for more than two years and could be followed up. From August 2023 to January 2024, 604 eligible patients were recruited and completed at least one assessment. The median age was 58 years (range, 27-82 years). The median duration of treatment with niraparib was 34.8 months (range, 30.5-46.7 months). Among 445 patients with BRCAm status, 104 were BRCAm (23.4%) and 341 were BRCAwt (76.6%). A total of 491 patients (81.3%) received niraparib 200 mg as a starting dose, while 107 (17.7%) required dose adjustments in clinical practice. Presently, 503 (83.3%) were on 200mg. 523 (86.6%) were compliant with the daily oral dose of niraparib, without missing any doses in the previous 6 months. 193 patients (32.0%) had common chronic disease, and 176 (29.1%) were taking concomitant medications during maintenance. The EQ-5D-5L HUI and VAS scores remained stable across the three assessments. The baseline mean EQ-5D-5L HUI and VAS scores were 0.98 (SD 0.05) and 84.31 (SD 10.50), respectively, and the least squares (LS) mean changes from baseline to the third assessment were -0.004 (SE 0.001) and -0.609 (SE 0.248), respectively. A similar situation was observed for the FOSI score. The baseline mean FOSI score was 31.13 (SD 1.37), and the LS mean change from baseline to the third assessment was -0.359 (SE 0.033). Conclusions: In the real world, patients with PSR OC who underwent long-term maintenance treatment with niraparib exhibited a consistently stable QoL. This finding highlights the significant clinical value of niraparib in maintaining QoL in these populations.

Characterising paroxysmal supraventricular tachycardia episodes by patient-perceived episode duration, symptoms, and severity: longitudinal patient-reported outcomes

Abstract Background/Information Paroxysmal supraventricular tachycardia (PSVT) encompasses a range of heart rhythms that are often associated with clinical symptoms. The duration and symptoms a patient experiences during a PSVT episode are variable. The impact of episode duration and symptom type on a patient’s perceived severity of an episode of PSVT has not been well defined. Purpose To characterise the relationship between duration, symptom type, and patient-perceived episode severity in a longitudinal study of patient-reported outcomes in the US and UK. Methods The survey included patients >18 years of age with a self-reported diagnosis of PSVT, without a history of ablation. Surveys included a 30-minute baseline survey (Mar 2019), 5-minute weekly tracking surveys (Mar 2019 to Dec 2019), and a 15-minute closing survey (Dec 2019). Patients were asked during the baseline survey to rate the severity of their PSVT (mild/moderate/severe). Weekly episode surveys assessed patient-reported episode duration, symptoms, severity, and interventions. If multiple episodes occurred, characteristics of the most recent episode were assessed. Results Among 117,252 patients screened, 368 met the inclusion criteria and completed the baseline survey, while 247 patients in the final cohort met the quality control checks. Most patients were White (92%), female (70%), and from the US (80%) and had a mean age of 52 years. At baseline, 47% of patients described their PSVT as moderate severity, while 39% reported mild and 14% reported severe. Overall, 5277 longitudinal weekly surveys were completed, of which 48% captured a PSVT episode (n=2518). Median reported duration of an episode was 15 minutes, with severe episodes having numerically longer duration (71 minutes) than moderate (20 minutes) or mild (6 minutes) episodes. Among severe episodes, 57% lasted 1 hour or longer; only 9% of mild episodes lasted 1 hour or longer (Figure 1). Among all assessed episodes of PSVT, 89% had palpitations, 35% shortness of breath, 26% dizziness, and 17% chest pain; mean number of symptoms per episode was 3.1, while numerically more symptoms were reported with severe episodes (mean 6.0) than with moderate (mean 3.5) or mild (mean 2.1) episodes. Shortness of breath was more frequently reported with severe episodes (81%) than with moderate (43%) or mild (17%) episodes. Similar findings were seen with dizziness and chest pain (Figure 2). Overall, approximately half of the reported episodes during follow-up were classified as moderate severity (47%), followed by mild (44%) and severe (9%). Conclusions Episodes of PSVT of longer duration and/or accompanied by a greater number of symptoms are reported by patients to be more severe. However, heterogeneity of symptoms and episode duration is substantial within a given classification of PSVT severity. Additional studies are needed to better understand how the optimal treatment of patients with PSVT may vary by duration, symptoms, and severity.

Linking genetic counseling communication skills to patient outcomes and experiences using a community-engagement and provider-engagement approach: research protocol for the GC-PRO mixed methods sequential explanatory study

IntroductionIn over 50 years since the genetic counseling (GC) profession began, a systematic study of GC communication skills and patient-reported outcomes in actual sessions across multiple clinical specialties has never...

Clinical Depression and Anxiety Are Relieved by Microvascular Decompression in Patients with Trigeminal Neuralgia-A Prospective Patient-Reported Outcome Study.

Objective: Patients with idiopathic trigeminal neuralgia (TN) live in constant fear of triggering shock-like pain episodes, which may cause symptoms of depression and a reduction in quality of life. Microvascular decompressive surgery has been demonstrated to achieve satisfactory and stable results. With this study, we wanted to investigate prevalence and risk factors for depression and perceived stress in correlation with symptom relief after surgical treatment. Methods: In this prospective study, patients undergoing microvascular decompression (MVD) for TN were included. The Barrow Neurological Institute Pain Score (BNI), Beck Depression Inventory (BDI), Chronic Pain Acceptance Questionnaire (CPAQ), Perceived Stress Questionnaire (PSQ) and McGill questionnaire were used to evaluate depression, stress and anxiety disorders before and 3 months after MVD. Results: A total of 35 patients (16 male (46%)) with a mean age of 55.4 (SD 15) years were included in this study. The BDI revealed that 24 (68.8%) patients harbored mild-to-extreme depression preoperatively (2.4 ± 1.4), which improved to 1.2 (±0.6, p < 0.0001). Pain acceptance also changed from 64 (±11.3) to 67.7 (±9.3, p = 0.006). Perceived stress decreased from 46.9 (±21.9) to 19.6 (±18.6) (p < 0.0001) postoperatively, and pain decreased from 31.0 (±11.7) to 9.4 (±12.9, p < 0.0001). Microvascular decompression reduced the mean BNI pain score significantly from 4.6 to 1.8 postoperatively (p < 0.00001). Conclusions: Depression and perceived stress are prevalent in patients with idiopathic TN. Adequate treatment not only provides a high rate of satisfaction through pain relief, but also leads to immediate and significant improvements in depression and stress. Thus, in patients with TN who do not reach an adequate and timely pain reduction through medical management and develop signs of depression, early treatment with microvascular decompression should be considered.

Comparison of the Real-World Reporting of Symptoms and Well-Being for the HER2-Directed Trastuzumab Biosimilar Ogivri With Registry Data for Herceptin in the Treatment of Breast Cancer: Prospective Observational Study (OGIPRO) of Electronic Patient-Reported Outcomes.

Trastuzumab has had a major impact on the treatment of human epidermal growth factor receptor 2 (HER2)-positive breast cancer (BC). Anti-HER2 biosimilars such as Ogivri have demonstrated safety and clinical equivalence to trastuzumab (using Herceptin as the reference product) in clinical trials. To our knowledge, there has been no real-world report of the side effects and quality of life (QoL) in patients treated with biosimilars using electronic patient-reported outcomes (ePROs). The primary objective of this prospective observational study (OGIPRO study) was to compare the ePRO data related to treatment side effects collected with the medidux app in patients with HER2-positive BC treated with the trastuzumab biosimilar Ogivri (prospective cohort) to those obtained from historical cohorts treated with Herceptin alone or combined with pertuzumab and/or chemotherapy (ClinicalTrials.gov NCT02004496 and NCT03578731). Patients were treated with Ogivri alone or combined with pertuzumab and/or chemotherapy and hormone therapy in (neo)adjuvant and palliative settings. Patients used the medidux app to dynamically record symptoms (according to the Common Terminology Criteria for Adverse Events [CTCAE]), well-being (according to the Eastern Cooperative Oncology Group Performance Status scale), QoL (using the EQ-5D-5L questionnaire), cognitive capabilities, and vital parameters over 6 weeks. The primary endpoint was the mean CTCAE score. Key secondary endpoints included the mean well-being score. Data of this prospective cohort were compared with those of the historical cohorts (n=38 patients; median age 51, range 31-78 years). Overall, 53 female patients with a median age of 54 years (range 31-87 years) were enrolled in the OGIPRO study. The mean CTCAE score was analyzed in 50 patients with available data on symptoms, while the mean well-being score was evaluated in 52 patients with available data. The most common symptoms reported in both cohorts included fatigue, taste disorder, nausea, diarrhea, dry mucosa, joint discomfort, tingling, sleep disorder, headache, and appetite loss. Most patients experienced minimal (grade 0) or mild (grade 1) toxicities in both cohorts. The mean CTCAE score was comparable between the prospective and historical cohorts (29.0 and 30.3, respectively; mean difference -1.27, 95% CI -7.24 to 4.70; P=.68). Similarly, no significant difference was found for the mean well-being score between the groups treated with the trastuzumab biosimilar Ogivri and Herceptin (74.3 and 69.8, respectively; mean difference 4.45, 95% CI -3.53 to 12.44; P=.28). Treatment of patients with HER2-positive BC with the trastuzumab biosimilar Ogivri resulted in equivalent symptoms, adverse events, and well-being as found for patients treated with Herceptin as determined by ePRO data. Hence, integration of an ePRO system into research and clinical practice can provide reliable information when investigating the real-world tolerability and outcomes of similar therapeutic compounds. ClinicalTrials.gov NCT05234021; https://clinicaltrials.gov/study/NCT05234021.

Neurobehavioral and developmental profiles: genotype–phenotype correlations in individuals with Cornelia de Lange syndrome

BackgroundCornelia de Lange (CdLS) is a rare genetic disorder that affects most body systems. Variants in multiple genes including NIPBL and SMC1A, can cause the syndrome. To date, literature on genotype–phenotype associations in individuals with CdLS is extremely limited, although studies suggest some differences in clinical phenotype severity across variants. This study aimed to examine and compare neurobehavioral differences and developmental variability across CdLS genes, specifically NIPBL and SMC1A, and identify genotype–phenotype correlations.Participants and methodsThis patient-reported outcomes study included accessing data from the Coordination of Rare Diseases registry at Sanford. Parents of a total of 26 children/adults with CdLS and a known variant in NIPBL (Mean age = 20.46 years, SD = 11.21) and 12 with a known variant in SMC1A (Mean age = 11.08 years, SD = 9.04) completed a series of questionnaires regarding their child’s developmental history. This included attainment of common language and motor milestones, intervention history, and behavior functioning. Developmental history and reported behavior regulation difficulties were compared across variant groups.ResultsOverall, individuals with a pathogenic variant in NIPBL or SMC1A were similarly delayed across motor and language milestones with about 70% not using phrase speech and 30–50% not walking by 5 years of age. However, those with NIPBL variants showed more severity in behavioral phenotype, namely with more repetitive behaviors, tantrums, and withdrawn behaviors. In addition, these individuals were more likely than those with SMC1A variants to demonstrate self-injurious behaviors, and anxiety. Both groups yielded a similar proportion of participants who participated in speech and occupational therapy, however those with SMC1A variants were more likely to engage in physical therapy. Both clinical groups report low rate of communicative or assistive device use despite a large proportion of participants never mastering single word or sentence use.ConclusionsStudy results are consistent with recent investigations highlighting more severe behavioral phenotype, particularly autistic features, anxiety, and behavior regulation challenges, among those with NIPBL variants albeit comparable developmental milestones. Both groups endorsed very elevated attention problems. Findings highlight importance of early interventions, including behavioral health services.

Local anaesthesia under sedation versus general anaesthesia for brachytherapy-treated patients with uveal melanoma: A patient-reported outcome study.

Local anaesthesia under sedation versus general anaesthesia for brachytherapy-treated patients with uveal melanoma: A patient-reported outcome study.

Impact of frailty on cancer-related fatigue and quality of life in outpatients with prostate cancer: a cross-sectional study of patient-reported outcomes.

To investigate the prevalence of frailty and its effects on cancer-related fatigue and quality of life among patients with prostate cancer. In this cross-sectional study, questionnaires were administered to 254 outpatients who visited the Department of Urology at Kagawa University Hospital for prostate cancer; finally, 108 outpatients were analyzed. Frailty, cancer-related fatigue and quality of life were assessed using the G8 screening tool, Japanese version of the Brief Fatigue Inventory and Japanese version of the Short Form 8 Health Survey, respectively. We defined frailty based on a score≤14 points and divided the patients into frailty and no-frailty groups. We also compared the severity of cancer-related fatigue and quality of life between groups. The prevalence of frailty among 108 outpatients was 63%. Older age correlated with frailty severity (P=0.0007) but not cancer-related fatigue severity (P=0.2391). The proportion of patients on treatment or with metastasis was not significantly different between groups. The frailty group had higher cancer-related fatigue severity (P=0.004) and decreased levels of general activity, mood, walking ability, normal work and enjoyment of life, especially on the Brief Fatigue Inventory subscale. The frailty group had lower physical and mental quality of life than the no-frailty group or general population. The frailty rate for these patients increased with age, exceeding 60% regardless of the treatment status, and was associated with worsened cancer-related fatigue severity and reduced quality of life. Our study highlights the importance of assessing frailty when selecting treatment, especially in older patients.

Impact of Electronic Patient-Reported Outcomes on Unplanned Consultations and Hospitalizations in Patients With Cancer Undergoing Systemic Therapy: Results of a Patient-Reported Outcome Study Compared With Matched Retrospective Data.

The evaluation of electronic patient-reported outcomes (ePROs) is increasingly being used in clinical studies of patients with cancer and enables structured and standardized data collection in patients' everyday lives. So far, few studies or analyses have focused on the medical benefit of ePROs for patients. The current exploratory analysis aimed to obtain an initial indication of whether the use of the Consilium Care app (recently renamed medidux; mobile Health AG) for structured and regular self-assessment of side effects by ePROs had a recognizable effect on incidences of unplanned consultations and hospitalizations of patients with cancer compared to a control group in a real-world care setting without app use. To analyze this, the incidences of unplanned consultations and hospitalizations of patients with cancer using the Consilium Care app that were recorded by the treating physicians as part of the patient reported outcome (PRO) study were compared retrospectively to corresponding data from a comparable population of patients with cancer collected at 2 Swiss oncology centers during standard-of-care treatment. Patients with cancer in the PRO study (178 included in this analysis) receiving systemic therapy in a neoadjuvant or noncurative setting performed a self-assessment of side effects via the Consilium Care app over an observational period of 90 days. In this period, unplanned (emergency) consultations and hospitalizations were documented by the participating physicians. The incidence of these events was compared with retrospective data obtained from 2 Swiss tumor centers for a matched cohort of patients with cancer. Both patient groups were comparable in terms of age and gender ratio, as well as the distribution of cancer entities and Joint Committee on Cancer stages. In total, 139 patients from each group were treated with chemotherapy and 39 with other therapies. Looking at all patients, no significant difference in events per patient was found between the Consilium group and the control group (odds ratio 0.742, 90% CI 0.455-1.206). However, a multivariate regression model revealed that the interaction term between the Consilium group and the factor "chemotherapy" was significant at the 5% level (P=.048). This motivated a corresponding subgroup analysis that indicated a relevant reduction of the risk for the intervention group in the subgroup of patients who underwent chemotherapy. The corresponding odds ratio of 0.53, 90% CI 0.288-0.957 is equivalent to a halving of the risk for patients in the Consilium group and suggests a clinically relevant effect that is significant at a 2-sided 10% level (P=.08, Fisher exact test). A comparison of unplanned consultations and hospitalizations from the PRO study with retrospective data from a comparable cohort of patients with cancer suggests a positive effect of regular app-based ePROs for patients receiving chemotherapy. These data are to be verified in the ongoing randomized PRO2 study (registered on ClinicalTrials.gov; NCT05425550). ClinicalTrials.gov NCT03578731; https://www.clinicaltrials.gov/ct2/show/NCT03578731. RR2-10.2196/29271.

Comparative, Controlled, Retrospective Study of Patient-Reported Outcomes After Meniscectomy With Adjunctive Use of Platelet-Rich Plasma or Amniotic Umbilical Cord Tissue.

Background: Meniscal tears are one of the most frequent injuries to the knee, with an estimated incidence of 222 per 100,000 individuals aged 18 to 55 years based on magnetic resonance imaging. Poor outcomes following meniscal surgical interventions are common and have led many surgeons to use biologic augmentation strategies to enhance the healing. Methods: We conducted a single-center, retrospective, observational study of patients who underwent arthroscopic meniscectomy with and without adjunctive platelet-rich plasma (PRP) or the particulate form of amniotic umbilical cord (AMUC) tissue. We evaluated patient-reported outcomes on the visual analog scale for pain, International Knee Documentation Committee (IKDC) Subjective Knee Evaluation Form, Lysholm Knee Scoring Scale, 12-Item Short Form Survey, and Knee Injury and Osteoarthritis Outcome Score (KOOS) during a 1-year postoperative period. Complications and follow-up procedures were also evaluated. Results: We evaluated 113 patients who underwent meniscectomies from November 2010 to March 2017. Pain severity was significantly decreased only in the AMUC group at 6 months (P=0.0143). Patients in the AMUC group demonstrated significant improvement in functional recovery based on the IKDC and the KOOS subscales of pain, symptoms, activities of daily living, and sport and recreation function at 6 months. Patients in the PRP group had a significant benefit in the KOOS subscales of pain, symptoms, sport and recreation function, and knee-related quality of life at 3 months. Improvement in the control group was less substantial. Patients in the PRP group had more complications and follow-up procedures (30.0%) than patients in the AMUC group (8.3%). Conclusion: In our study population, arthroscopic meniscectomy with adjunctive use of AMUC tissue improved patient-reported outcomes and reduced the reoperation rate compared to conventional technique or adjunctive use of PRP.

Pilot study of remote electronic patient-reported outcomes (ePRO) symptom monitoring of palliative care needs among outpatients with advanced solid cancers.

250 Background: Early specialty palliative care for patients with advanced cancer is an evidence-based practice that improves health outcomes, yet patients do not receive timely referrals. ePRO symptom monitoring provides a ready care delivery platform to monitor patients for unmet palliative care needs and direct palliative care referral based on patient report. This study sought to obtain feasibility data for ePRO symptom monitoring of palliative care needs. Methods: We conducted a single-arm pilot study of longitudinal ePRO symptom monitoring of multidimensional palliative care needs among outpatients with advanced breast, gastrointestinal, and gynecologic cancers at an academic oncology practice. Adult patients on active treatment with a prognosis &gt; 6 months were approached consecutively. Patients completed baseline measures and enrolled on a third-party symptom monitoring platform for 12 weeks. Patients received weekly monitoring of 14 common symptoms using the National Cancer Institute's PRO version of the common terminology criteria for adverse events (PRO-CTCAEs). Patients received monthly monitoring of palliative needs using 11 PRO items from validated measures and a clinician-developed item for patient request for palliative care. Weekly symptom alerts and monthly palliative care PRO assessments were reported to the primary oncology team for interdisciplinary clinical management. Acceptability and appropriateness of the intervention were assessed using the Acceptability of Intervention Measure (AIM) and Intervention Appropriateness Measure (IAM). Results: From May 2022 to April 2023, 29 of 37 eligible patients (78%) consented to the study, of which 25 completed baseline measures and enrolled on the platform (68% enrollment rate; most common reason for refusal was not enough time). Median age was 53 years (range 29-77), 18 (72%) female, 12 (48%) non-Hispanic White, 32% reported an ECOG-PRO score 2-3. 96% of patients met pre-defined ePRO adherence thresholds and 92% completed study end measures (1 patient censored for death). Completion of the palliative PRO items was &gt; 94% for 9 of 11 items. Two PRO items for spiritual distress screening were skipped most often. 80% of patients reported a severe score for at least one palliative PRO symptom during the study period. Mean AIM and IAM scores were 3.92 and 4.26, respectively (range 3.25 – 5; best possible total score 5). Conclusions: Expanding evidence-based ePRO symptom monitoring to include monthly palliative care needs assessment is highly feasible, acceptable, and appropriate to patients. Adherence to PRO items was high, and most patients had at least one area of need. Future work will refine the ePRO palliative assessment with attention to spiritual distress screening and determine severity thresholds to develop a PRO-directed referral system to specialty palliative care.

Comparison of Innervated and Non-innervated Free Glabrous Skin Flap Transfers for Volar Digital Defect Reconstructions - A patient-reported Outcome Study

Comparison of Innervated and Non-innervated Free Glabrous Skin Flap Transfers for Volar Digital Defect Reconstructions - A patient-reported Outcome Study

Advantages of Totally Stapled Collard Over Circular Stapled Technique for Cervical Esophagectomy Anastomosis

BackgroundThe quality of life between the totally stapled Collard (TSC) and circular stapled (CS) techniques from the patient’s perspective has not been assessed. This longitudinal patient-reported outcome and objective data assessment study aimed to compare the effects of TSC vs CS for cervical anastomosis with minimally invasive esophagectomy (MIE) on the quality of life. MethodsThis single-center, retrospective study included patients with esophageal cancer who underwent MIE and cervical anastomosis between April 2019 and February 2021; of whom, 105 and 216 patients received TSC and CS anastomosis, respectively. The objective metrics were evaluated based on postoperative complications. Patient-reported outcomes instruments using the European Organization for Research and Treatment of Cancer modules were assessed preoperatively. The Quality of Life Questionnaire (QLQ) Core 30 and the QLQ-Oesophagus Module 18 assessed the quality of life. Mixed-effects models examined changes in questionnaire scores. ResultsThe stenosis rate in the TSC group was significantly lower than that in the CS group. There were no significant differences in other postoperative complications between the groups. In the QLQ Core 30 scale, the TSC group had better emotional function, less fatigue, less appetite loss, and better function than the CS group, with significant differences in different time points between the groups. QLQ-Oesophagus Module 18 revealed that the TSC group had significantly less appetite loss, dysphagia, dry mouth, pain, and obstruction, and better eating and speech than the CS group. ConclusionsTSC has significant advantages over the CS technique in lowering postoperative symptom burden and improving quality of life for cervical anastomosis on patient-reported outcomes.

Disc prosthesis versus fusion with cage in single level cervical degenerative spine disease – A retrospective case-control patient reported outcome study

AimTo compare the clinical outcome of single level cervical degenerative spine disease treated surgically with motion preserving anterior cervical disc arthroplasty versus anterior cervical discectomy and fusion (ACDF). MethodsPatients treated with cervical disc arthroplasty at Aalborg University Hospital and ACDF at Aarhus University Hospital were matched 1:2.Primary outcome measures were Neck Disability Index (NDI), EQ-5D-3 L and Numeric rating scale (NRS) for arm- and neck pain.Data was collected by telephone interviews regarding present and retrospective data. Results50 patients treated with cervical disc arthroplasty were matched to 100 ACDF patients covering November 2011 to December 2018.Mean improvements for NRS neck pain three-months postoperative, and NDI were significantly better in the cervical disc arthroplasty group, with intergroup differences of 1.56 (p = 0.02) and 5.01 (p = 0.01) respectively.A subgroup analysis of the half of the cohort with the longest follow-up (mean 7.6 years) showed, in favour of cervical disc arthroplasty, mean improvements of NDI: 8.80 (p = 0.00), EQ5D: − 0.19 (p = 0.04), NRS neck three months follow-up: 3.70 (p = 0.00) and long follow-up: 2.54 (p = 0.01) and NRS arm three months follow-up: 2.02 (p = 0.01).Radiologic examination indicated preserved mobility in 80% of the implanted protheses at 24-month post-surgical follow-up. ConclusionSurgical treatment of one level degenerative cervical spine disease with cervical disc arthroplasty or anterior cervical discectomy and fusion has a similar good clinical outcome after a mean follow-up of 5.6 years.However, cervical disc arthroplasty displayed long-term superiority in the half of the cohort with the longest follow-up time averaging 7.6 years.

The Europa Uomo Patient Reported Outcome Study 2.0—Prostate Cancer Patient-reported Outcomes to Support Treatment Decision-making

BackgroundTo further strengthen the voice of patients, Europa Uomo initiated the Europa Uomo Patient Reported Outcome Study 2.0 (EUPROMS 2.0) in October 2021. ObjectiveTo collect the self-reported perspective of prostate cancer (PCa) patients on physical and mental well-being after PCa treatment outside a clinical trial setting to inform future fellow patients about the impact of PCa treatment. Design, setting, and participantsEuropa Uomo invited PCa patients to complete a cross-sectional survey including the validated EQ-5D-5L, EORTC-QLQ-C30, and the EPIC-26 questionnaires. Furthermore, the nine-item Shared Decision Making Questionnaire (SDM-Q-9) and diagnostic clinical scenarios were included. Outcome measurements and statistical analysisDescriptive statistics was used to assess the demographic and clinical characteristics and to analyze the patient-reported outcome data. Results and limitationsBetween October 25, 2021 and January 17, 2022, 3571 men from 30 countries completed the EUPROMS 2.0 survey. The median age of respondents was 70 yr (interquartile range 65–75 yr). Half of the respondents underwent one treatment, most often radical prostatectomy. Men who are treated actively experience lower health-related quality of life than men on active surveillance, mainly regarding sexual function, fatigue, and insomnia. Lower urinary incontinence levels were seen for men who underwent radical prostatectomy (single treatment or in combination with other treatments). Of the respondents, 42% indicated that the determination of the prostate-specific antigen (PSA) value was part of a routine blood test; 25% wanted to undergo screening/early detection for PCa, and 20% indicated that the determination of the PSA value had a clinical reason. ConclusionsA large sample of 3571 international patients has contributed patient experience after PCa treatment in the EUPROMS 2.0 study, confirming that treatment for PCa mainly affects urinary incontinence, sexual function, fatigue, and insomnia. Such information can be used to direct toward a better patient-doctor relationship, to offer patients ready access to responsible information and a better understanding of their disease and treatment. Patient summaryThrough the EUPROMS 2.0 survey, Europa Uomo has strengthened the voice of the patient. Such information can be used to inform future prostate cancer (PCa) patients about the impact of PCa treatment and to engage them in informed and shared decision-making.

Malnutrition in Head and Neck Cancer: A Patient-reported Outcome Study.

Head and neck cancer (HNC) is associated with a high risk of malnutrition. Malnutrition is defined as acute weight loss greater than 5% and increases mortality 1.7-fold for HNC patients. The aim of the study was to investigate the social and nutritional impairments that patients face throughout cancer-survivorship. The study was conducted nationwide via the self-help network and in a single oncological center. We analysed 134 patient reported outcome (PRO) questionnaires with a mean age of 65.5±9.4 years, including 88 males, 36 females, and ten participants of undisclosed sex. The questionnaire contained 47 items enquiring about demography, weight development, and how treatment impaired nutrition. The patient data showed a weight loss of 8% after surgery, 13% after (chemo)radiotherapy [(C)RT] and a return to baseline weight in convalescence. However, patients with a baseline weight >100 kilogram (kg), had a 22% weight loss after (C)RT (p<0.0001) and this remained permanent at 11% (p=0.0041). Treatment-associated side-effects gradually decreased in the course compared to the time of treatment: loss of taste (55% to 21%), xerostomia (56% to 42%), dysphagia (57% to 43%), and dental problems (33%/ to 21%). (C)RT immediately led to more loss of taste (p=0.0461) and dysphagia (p=0.0334), and surgery as a singular modality scored the lowest odds ratio for side-effects. Social Impact: mood, supporting networks, and supplement satisfaction were rated "good" (Likert Scale). Malnutrition is common among HNC patients. High baseline weight and extensive multimodal treatment are important risk factors that require enhanced stewardship.

Patients' attitudes regarding genetic counseling before germline BRCA1/2 pathogenic variants testing in Taiwan: Asingle-country, multi-center, patient-reported outcome study.

Germline pathogenic variants of BRCA1 or BRCA2 cause hereditary breast and ovarian cancer syndromes. The present study investigated the participants' understanding and awareness of germline BRCA1/2 pathogenic variants before genetic counseling, the expectations and obstacles for genetic testing from the perspective of participants and their families, and their attitudes towards genetic testing after counseling. In this single-country, multicenter, non-interventional, patient-reported outcome study, untested cancer patients and their families who visited genetic counseling clinics or who wanted to receive pre-test genetic counseling were eligible to fill in the questionnaire after pre-test counseling for germline BRCA1/2 testing. Demographic information, clinical characteristics, and information collected from the questionnaires, including the understanding of BRCA1/2 pathogenic variants before genetic counseling, understanding of BRCA1/2 pathogenic variants and feelings after genetic counseling, willingness to share results of genetic testing with family, and willingness to receive genetic testing, were summarized using descriptive statistics. A total of 88 participants were enrolled. The proportion of slight understanding of BRCA1/2 pathogenic variants increased from 11.4% to 67.0%, and the proportion of full understanding increased from 0% to 8.0%. After genetic counseling, most participants were willing to undergo genetic testing (87.5%) and share the results with their families (96.6%). The main factors that may affect participants' willingness to undergo BRCA1/2 testing were management (61.2%) and testing costs (25.9%). After pre-test counseling, there was a high acceptance of BRCA1/2 testing and in-family information sharing in Taiwanese patients with cancer and their families, which may serve as a reference for implementing genetic counseling in Taiwan.

A cross-sectional study of patient-reported outcomes and symptom burden using PROMIS and PRO-CTCAE measures in light chain amyloidosis.

We conducted a cross-sectional study to characterize health-related quality of life and symptom burden in individuals living with light chain (AL) amyloidosis. Members of the Amyloidosis Support Groups, Inc. with AL amyloidosis who consented to this IRB-approved survey provided information on their amyloidosis diagnosis, treatment, symptoms, and functioning. HRQL was measured using PROMIS and PRO-CTCAE questionnaires. Among 297 participants who responded, the median age at diagnosis was 60years (23-82) with 52% female and 90% white race. There were 69% AL (lambda) and 39% reported 3 or more organs involved with amyloidosis (58% cardiac, 58% renal, 30% neurological AL). Time from diagnosis was less than 2years in 64 (22%), 2-5years in 105 (36%), > 5years in 126 (43%), and unknown in 2 (< 1%) individuals. Therapy included prior chemotherapy in 88% and stem cell transplant in 52%. Fifty percent of the cohort was on active treatment. Multiple domains were impaired in AL amyloidosis compared to the general population, including physical function, fatigue, and social roles. While highest among those within 2years of diagnosis, high symptom burden was also seen in long-term survivors. A trend to decreased severity and number of impaired symptoms was seen with longer treatment-free interval but many symptoms remained persistent. Significant and persistent symptom burden is seen in AL amyloidosis. Patient-reported outcomes should be routinely measured and used to provide best supportive care to all AL amyloidosis patients, including long-term survivors and those not on active therapy.

P783 Symptom Improvement of ulceRative colitis after an Injection dose of Ustekinumab in Japanese clinical practice, measured using patient-reported outcomes (SIRIUS) study, Interim Analysis

Abstract Background Ustekinumab (UST) is treated to moderate to severe adult ulcerative colitis (UC) patients. We conducted the SIRIUS study (Symptom Improvement of ulceRative colitis after an Injection dose of UStekinumab in Japanese clinical practice, measured using patient-reported outcomes (PRO)) to record daily symptoms for 8 weeks after the introduction of intravenous ustekinumab in moderate to severe UC patients, which aimed to reveal early response patterns of symptoms during the induction of remission period with ustekinumab, and to explore symptoms suitable for monitoring and predicting treatment effect. This interim analysis focuses on stool frequency (SF) and rectal bleeding (RB) scores in the PRO-2 during the 8-week induction period. Methods This single-arm prospective observational study conducted in 23 centers in Japan aimed to recruit 140 patients with moderate-to-severe UC between July 2021 and July 2022. RB score, SF score, abdominal pain, nocturnal diarrhea, tenesmus, and patient perception of improvement were recorded daily using a smartphone app. To be eligible for the study, participants must have a documented diagnosis of UC, be ≥16 years of age, have UC (partial Mayo score of 5~9), have had an inadequate response or intolerance to prior UC therapies, and be able to understand and complete PRO instruments using a smartphone. Patients who had prior exposure to UST, history of extensive colectomy, or were hospitalized when UST was started were excluded. Results In this analysis,137 patients were included. Seventy-four (54.4%) patients were male and the median age was 35 years. Seventy-six (55.9%) patients were Bio-naïve. This high proportion of Bio-naïve patients reflects the fact that UST can be used for Bio-naive patients without restrictions in the Japanese health insurance. At weeks 2 and 4, 56 (41.2%) and 57 (41.9%) patients attended outpatient clinics, respectively. The data collection rate of the PROs was 95%. Mean SF and RB scores were demonstrated stratified by prior treatment with Bio (Figure 1&amp;3). Significant decreases in mean SF and RB scores were observed within 1 week in both Bio-naïve and -experienced patients. the time-course change in SF was similar between Bio-naïve and -experienced patients, but the decrease in RB scores was greater in the Bio-naïve patients than the Bio-experienced patients. Conclusion A daily PRO-based analysis was conducted in the Sirius study to monitor symptoms during the first 8 weeks after the introduction of UST in patients with moderate-to-severe UC. This interim analysis revealed that SF and RB scores started to decrease within 1 week, demonstrating a rapid symptomatic response to UST. The analysis of the other PROs and predictors of efficacy will be performed in further studies.