Patient-reported Outcome Questionnaires Research Articles

Abstract PO4-17-02: A multicenter, prospective, observational study of patients receiving trastuzumab deruxtecan for the treatment of HER2-positive and HER2-low unresectable and/or metastatic breast cancer: DESTINY-Breast-RESPOND

Abstract Background: Approximately 20% of breast cancer (BC) cases are human epidermal growth factor receptor 2-positive (HER2+; immunohistochemistry [IHC] 3+, IHC 2+/in-situ hybridization [ISH]+), and up to ~50% of patients with primary or metastatic BC (mBC) have HER2-low tumors (IHC 1+, IHC 2+/ISH−), a new therapeutically targetable subset of BC. Trastuzumab deruxtecan (T-DXd) is a HER2-directed antibody-drug conjugate approved in the USA, EU, and other countries for the treatment of adult patients with unresectable or metastatic HER2+ BC who have received a prior anti-HER2-based regimen, or with unresectable or metastatic HER2-low BC who have received a prior chemotherapy (CTx) in the metastatic setting or developed disease recurrence during or within 6 months of completing adjuvant CTx. Approvals followed positive results from the DESTINY-Breast03 and DESTINY-Breast04 clinical trials. In DESTINY-Breast03, T-DXd improved median progression-free survival (mPFS) compared with trastuzumab emtansine (28.8 vs 6.8 months, respectively; P< 0.0001) in previously treated (prior trastuzumab and taxane) patients with HER2+ mBC. In DESTINY-Breast04, T-DXd significantly improved mPFS vs physician’s choice of CTx (9.9 vs 5.1 months, respectively; P< 0.001) in all randomized patients with HER2-low mBC who were previously treated with one or two lines of CTx. Alongside these efficacy benefits, T-DXd has demonstrated an acceptable and generally manageable safety profile. To optimize care and maximize treatment benefit, insights into real-world T-DXd use and safety events of interest, including patient management and experience, are needed. This study aims to describe the effectiveness and tolerability of T-DXd in patients with HER2+ or HER2-low unresectable and/or mBC in a real-world setting. Trial design: DESTINY-Breast-RESPOND (NCT05592483) is a multicenter (120 sites planned as of June 2023), multi-country (HER2+ cohort: several regions globally; HER2-low cohort: North America only), prospective, observational study characterizing real-world clinical use, effectiveness, tolerability, and patient experience among adults who initiated T-DXd monotherapy, per standard of care, for HER2+ (second line or earlier) and HER2-low unresectable and/or mBC (T-DXd not provided by study sponsor). The decision to initiate T-DXd will be made by the physician prior to, and independent from, participation in this study. Approximately 750 patients with HER2+ and 250 patients with HER2-low disease will be enrolled. Patients must have received prior treatment with a trastuzumab-containing regimen in the metastatic setting or have evidence of disease progression within 6 months of neoadjuvant or adjuvant treatment (HER2+ cohort), or prior CTx in the metastatic setting or have evidence of disease progression within 6 months of adjuvant CTx (HER2-low cohort). Data will be collected via chart abstraction, patient-reported outcome questionnaires on tolerability and safety (PGI-TT, NCI PRO-CTCAE, and EORTC IL19), and a daily patient nausea/vomiting symptom diary. Endpoints will be analyzed separately for HER2+ and HER2-low cohorts. The primary endpoint of interest is time to next treatment from T-DXd initiation, a measure of real-world effectiveness. Other important endpoints include treatment patterns, safety events of interest (nausea/vomiting, fatigue, alopecia, interstitial lung disease/pneumonitis, left ventricular ejection fraction decrease) and their management, time to T-DXd discontinuation, and patient-reported tolerability. Patients will be observed until end of study (~60% of patients receive subsequent treatment or have died), withdrawal from study, or loss to follow up, whichever occurs first. Citation Format: Joyce O'Shaughnessy, Reva Basho, Maryam Lustberg, Gary H Lyman, Manoj Prahladan, Gareth D. James, Della Varghese, Flavia Lujan, Hans Tesch. A multicenter, prospective, observational study of patients receiving trastuzumab deruxtecan for the treatment of HER2-positive and HER2-low unresectable and/or metastatic breast cancer: DESTINY-Breast-RESPOND [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 PO4-17-02.

Rhinoplasty Health Inventory and Nasal Outcomes (RHINO) Scale: Translation, Cross-Cultural Adaptation and Validation for Hebrew-Speaking Patients.

Patient-reported outcomes questionnaires are accepted measurement tools to evaluate procedures results. The Rhinoplasty Health Inventory and Nasal Outcomes (RHINO) scale is an English-language validated and reliable quality-of-life instrument that evaluates both functional and aesthetic outcomes after rhinoplasty, and is not available in Hebrew. We followed the forward-and-back-translation method, defined by the European Organization for Research and Treatment of Cancer. Translation of the RHINO scale and merging it into one version were performed, following back-translation and validation on native Hebrew-speaking patients who underwent rhinoplasty and control healthy group. Lastly, we review and analyzed the results. The translated questionnaire had high reliability measures, demonstrating homogeneity (α value 0.800 in the rhinoplasty group and 0.896 in the control group), test-retest reproducibility with no significant difference (p = 0.5), and high agreement scores represented by the Bland-Altman plot (95% limits of agreement ranged from 8.78 to 9.80). Validity demonstrated by significant differences between the two study groups' mean questionnaire scores (p < 0.001 for rhinoplasty vs. controls, p < 0.001 for preoperative vs. postoperative, and p = 0.002 for postoperative vs. control group) and also between the aesthetic scores and functional scores of the rhinoplasty group before and after the surgery (p < 0.001). There were no reported understanding problems. The Hebrew version of the RHINO scale is a reliable and valid tool for pre- and post-rhinoplasty surgery outcomes measuring among Hebrew-speaking patients, and can improve coordination of expectations, follow-up, and quantify subjective evaluation of the surgery. Level of evidence: IV.

Once-weekly insulin efsitora alfa: Design and rationale for the QWINT phase 3 clinical development programme.

Insulin efsitora alfa (efsitora) is a once-weekly basal insulin. This review describes the study design and rationale of the efsitora phase 3 Once Weekly (QW) Insulin Therapy (QWINT) clinical development programme, including the five trials, QWINT-1 through QWINT-5. The five trials included insulin-naïve adults (QWINT-1 and -2) with type 2 diabetes (T2D), adults with T2D previously treated with basal insulin (QWINT-3 and -4), and QWINT-5 in adults with type 1 diabetes. All five trials were designed as multicentre, randomized, controlled, open-label, treat-to-target studies to investigate the efficacy and safety of efsitora versus active once-daily basal insulin comparators (insulin glargine U100 or insulin degludec U100). The primary objective of each trial is to compare the change in HbA1c from baseline to week 26 or 52 between efsitora and the active comparator. The key secondary objectives include change in fasting glucose, insulin dose and continuous glucose monitoring variables, and patient-reported outcome questionnaires. The QWINT development programme includes a racially and geographically diverse population to provide important information regarding the efficacy and safety of efsitora and its clinical management of people with diabetes.

Long-term Outcomes of Surgically Treated Congenital Dislocation of the Knee.

Congenital dislocation of the knee (CDK) may be idiopathic or associated with another condition, such as Larsen syndrome or arthrogryposis. Surgical reduction of type-3 dislocation may require quadricepsplasty (QP) or femoral diaphyseal shortening (FS). Because it is unknown which treatment is more effective, we evaluated long-term outcomes using patient-reported questionnaires and gait analysis, comparing results by surgery type and underlying diagnosis. Twelve patients (mean age, 19mo) were treated surgically for CDK from 1985 to 2015 and studied 9 to 30 years postoperatively. Three participants had idiopathic CDK, 5 had Larsen syndrome, and 4 had arthrogryposis. Eleven knees underwent QP and 7 underwent FS. Participants were evaluated in our movement science laboratory and completed patient-reported outcome questionnaires. Data were compared with healthy, age-matched control values at the same visit. Surgically treated knees had less flexion during swing (P<0.01), less overall motion (P<0.01), greater coronal instability (P<0.04), and slower gait (P<0.01) compared with controls. QP knees had more instability in midstance (P=0.03) and less flexion during gait compared with FS knees, less sagittal power generation than controls (P<0.01), and trended toward lower scores on Knee Injury and Osteoarthritis Outcome and Lysholm Knee Questionnaires than FS patients did. The idiopathic group had the gait most similar to that of controls, followed by the Larsen syndrome group and then the arthrogryposis group. The idiopathic group also had a better UCLA Activity Score (P=0.03) than the arthrogryposis group did. Surgical treatment of type-3 CDK will not likely restore normal knee function, suggesting teratologic joint abnormality. In this small series, FS produced better gait mechanics and patient-reported outcomes compared with QP. Not surprisingly, patients with idiopathic CDK had better outcomes than those with a syndromic diagnosis, likely related to having only a single joint affected. Level III.

The validity and reliability of the Turkish version of the quality of recovery-15 (QoR-15) questionnaire.

Quality of recovery (QoR) is a significant component of peri-operative health status and is influenced by patients' characteristics and surgical and anesthetic methods. The QoR-15 scale is a patient-reported outcome questionnaire that measures postoperative QoR. The validity of the QoR-15 scale has been proven in many languages. In this study, we aimed to translate the QoR-15 questionnaire into Turkish and evaluate its validity in the Turkish population. After being translated into Turkish, the questionnaire was administered to 190 patients who underwent obstetric, gynecological, orthopedic, or thoracic surgery under general or regional anesthesia. The Turkish version of QoR-15 (QoR-15T) was administered 2 times: before surgery and 24 hour after surgery. The feasibility, reliability, validity and responsiveness of the QoR-15T were evaluated. Because 13 patients were discharged within 24 hour postoperatively, the study was completed with 177 patients. The recruitment and completion rates of questionnaire were 95% and 93.1% respectively. The completing time of the questionnaire was 2.5 minutes preoperatively and 3.5 minutes postoperatively. The scale yielded a Cronbach α value of 0.75, a Cohen effect size of 1.42, and a standardized response mean of 1.39. There was a significant positive correlation (95% confidence interval; R = 0.68, P < .001) between QoR-15T and visual analog scale postoperatively. The correlation of the items with the total QoR-15T score ranged from 0.19 to 0.60. The total scores of preoperative and postoperative QoR-15T were mean: 130.67, standard deviation: 15.78 and mean: 108.23, standard deviation: 13.06, respectively, with a significant difference between them (P < .01). The QoR-15T is feasible, reliable, valid, and responsive among patients undergoing surgery under general and regional anesthesia.

Clinical and patient-reported outcomes in patients with chronic hepatitis B and C and non-alcoholic fatty liver disease from real-world practices in Saudi Arabia, Turkey and Egypt.

Patients with chronic liver disease (CLD) experience health-related quality of life (HRQoL) and patient-reported outcomes (PROs) impairments. We assessed and identified predictors of HRQoL and PROs in CLD patients from Saudi Arabia (SA), Turkey and Egypt. Patients enrolled in Global Liver Registry™ with chronic hepatitis B (CHB), chronic hepatitis C (CHC) and non-alcoholic fatty liver disease (NAFLD) or non-alcoholic steatohepatitis (NASH) were included. Clinical data and PRO questionnaires (FACIT-F, CLDQ and WPAI) were compared across countries. Linear regression identified PRO predictors. Of the 4014 included patients, 26.9% had CHB, 26.9% CHC and 46.1% NAFLD/NASH; 19.2% advanced fibrosis. Compared across countries, CHB patients were younger in Egypt (mean age [years] 41.2 ± 11.4 vs. 45.0 ± 10.3 SA, 46.1 ± 12.0 Turkey), most often employed in SA (64.8% vs. 53.2% Turkey) and had the lowest prevalence of obesity in Turkey (26.7% vs. 37.8% SA, 38.5% Egypt). In SA, CHB patients had lowest prevalence of fibrosis and comorbidities (all p < .01). There was a higher frequency of males with NAFLD/NASH in SA (70.0% vs. 49.6% Turkey, and 35.5% Egypt). Among NAFLD/NASH patients, CLDQ-NAFLD/NASH scores were highest in SA (mean total score: 5.3 ± 1.2 vs. 4.8 ± 1.2 Turkey, 4.1 ± 0.9 Egypt, p < .01). Independent predictors of worse PROs included younger age, female sex, advanced fibrosis, non-hepatic comorbidities and lack of regular exercise (all p < .05). Clinical presentation and PRO scores of CLD patients vary across SA, Turkey and Egypt. Impairment of HRQoL is associated with demographic factors, lack of regular exercise, advanced fibrosis and non-hepatic comorbidities.

A study demonstrating users' preference for the adapted-REQUITE patient-reported outcome questionnaire over PRO-CTCAE® in patients with lung cancer.

The use of patient-reported outcomes (PROs) has been shown to enhance the accuracy of symptom collection and improve overall survival and quality of life. This is the first study comparing concordance and patient preference for two PRO tools: Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE®) and the adapted-REQUITE Lung Questionnaire. Patients with lung cancer were recruited to the study while attending outpatient clinics at a tertiary cancer centre. Clinician-reported outcomes were generated through initial patient assessment with CTCAE v4.03. Participants then completed the PRO-CTCAE® and adapted-REQUITE questionnaires. Concordance between the 2 questionnaires was assessed by calculating Pearson correlation coefficient. PRO-CTCAE® and CTCAE concordance was demonstrated by calculating Pearson correlation coefficient from the linear predictors of an ordinal logistic regression. P-values were also calculated. Out of 74 patients approached, 65 provided written informed consent to participate in the study. 63 (96.9%) patients completed both PRO-CTCAE® and adapted-REQUITE questionnaires. Pearson correlation coefficient between PRO tools was 0.8-0.83 (p <.001). Correlation between CTCAE and PRO-CTCAE® ranged between 0.66-0.82 (p <.001). Adapted-REQUITE and CTCAE correlation was higher for all symptoms ranging between 0.79-0.91 (p <.001). Acceptable discrepancies within one grade were present in 96.8%-100% of symptom domains for REQUITE and in 92.1%-96.8% for all domains in the PRO-CTCAE®. 54% of the total participant cohort favored the adapted-REQUITE questionnaire due to reduced subjectivity in the questions and ease of use. The adapted-REQUITE questionnaire has shown a superior correlation to clinician-reported outcomes and higher patient preference than the PRO-CTCAE®. The results of this study suggest the use of the REQUITE questionnaire for patients with lung cancer in routine clinical practice.

BCLA CLEAR Presbyopia: Evaluation and diagnosis

It is important to be able to measure the range of clear focus in clinical practice to advise on presbyopia correction techniques and to optimise the correction power. Both subjective and objective techniques are necessary: subjective techniques (such as patient reported outcome questionnaires and defocus curves) assess the impact of presbyopia on a patient and how the combination of residual objective accommodation and their natural DoF work for them; objective techniques (such as autorefraction, corneal topography and lens imaging) allow the clinician to understand how well a technique is working optically and whether it is the right choice or how adjustments can be made to optimise performance. Techniques to assess visual performance and adverse effects must be carefully conducted to gain a reliable end-point, considering the target size, contrast and illumination. Objective techniques are generally more reliable, can help to explain unexpected subjective results and imaging can be a powerful communication tool with patients. A clear diagnosis, excluding factors such as binocular vision issues or digital eye strain that can also cause similar symptoms, is critical for the patient to understand and adapt to presbyopia. Some corrective options are more permanent, such as implanted inlays / intraocular lenses or laser refractive surgery, so the optics can be trialled with contact lenses in advance (including differences between the eyes) to better communicate with the patient how the optics will work for them so they can make an informed choice.

Outcomes and Quality of Life After Resorbable Synthetic Ventral Hernia Repair in Contaminated Fields.

The ideal mesh type for hernia repair in a contaminated field remains controversial. Data regarding outcomes beyond 1 year and the impact on quality of life (QoL) are unknown. This study aims to evaluate the long-term surgical outcomes and patient-reported outcomes (PROs) after contaminated repair with poly-4-hydroxybutyrate (P4HB) mesh. Patients undergoing a contaminated hernia repair with P4HB between 2015 and 2020 were identified. Surgical site occurrences and hernia recurrences were recorded. Long-term PROs as defined by the Abdominal Hernia-Q and Hernia-Related Quality-of-Life Survey were assessed. Fifty-five patients were included with a mean age of 54.5 years, a body mass index of 34 kg/m2, and a defect size of 356.9 ± 227.7 cm2. Median follow-up time was 49 months with a reoperation rate of 14.5% and a hernia recurrence rate of 7.3%. Of the 55 patients, 37 completed preoperative and postoperative PRO questionnaires. When comparing preoperative with postoperative Abdominal Hernia-Q, there was significant improvement in overall PROs (2.1 vs 3.5, P < 0.001). This improvement was maintained with no significant changes between postoperative scores over the course of 5 years. Patients with complications saw the same improvement in postoperative PRO scores as those without complications. Our study found a low hernia recurrence and acceptable long-term reoperation rates in patients undergoing hernia repair with P4HB mesh in a contaminated setting. We demonstrate a sustained significant improvement in QoL scores after hernia repair. These data yield insight into the long-term outcomes and QoL improvement, providing physicians further knowledge to better counsel their patients.

ROAR-A: re-optimization based Online Adaptive Radiotherapy of anal cancer, a prospective phase II trial protocol

BackgroundChemo-radiotherapy with curative intent for anal cancer has high complete remission rates, but acute treatment-related gastrointestinal (GI) toxicity is significant. Toxicity occurs due to irradiation of surrounding normal tissue. Current radiotherapy requires the addition of large planning margins to the radiation field to ensure target coverage regardless of the considerable organ motion in the pelvic region. This increases the irradiated volume and radiation dose to the surrounding normal tissue and thereby toxicity. Online adaptive radiotherapy uses artificial intelligence to adjust the treatment to the anatomy of the day. This allows for the reduction of planning margins, minimizing the irradiated volume and thereby radiation to the surrounding normal tissue.This study examines if cone beam computed tomography (CBCT)-guided oART with daily automated treatment re-planning can reduce acute gastrointestinal toxicity in patients with anal cancer.Methods/designThe study is a prospective, single-arm, phase II trial conducted at Copenhagen University Hospital, Herlev and Gentofte, Denmark. 205 patients with local only or locally advanced anal cancer, referred for radiotherapy with or without chemotherapy with curative intent, are planned for inclusion. Toxicity and quality of life are reported with Common Terminology Criteria of Adverse Events and patient-reported outcome questionnaires, before, during, and after treatment. The primary endpoint is a reduction in the incidence of acute treatment-related grade ≥ 2 diarrhea from 36 to 25% after daily online adaptive radiotherapy compared to standard radiotherapy. Secondary endpoints include all acute and late toxicity, overall survival, and reduction in treatment interruptions.ResultsAccrual began in January 2022 and is expected to finish in January 2026. Primary endpoint results are expected to be available in April 2026.DiscussionThis is the first study utilizing online adaptive radiotherapy to treat anal cancer. We hope to determine whether there is a clinical benefit for the patients, with significant reductions in acute GI toxicity without compromising treatment efficacy.Trial registrationClinicalTrials.gov Identifier: NCT05438836. Danish Ethical Committee: H-21028093

Pediatric Spastic Wrist Contractures Can Be Well Managed With Wrist Arthrodesis.

Severe spastic wrist contractures secondary to cerebral palsy (CP) or alike can have significant implications for patient hand function, hygiene, skin breakdown, and cosmesis. When these contractures become rigid, soft tissue procedures alone are unable to obtain or maintain the desired correction. In these patients' wrist arthrodesis is an option-enabling the hand to be stabilized in a more functional position for hygiene, dressing, and general cosmesis, though are patients satisfied? All children who had undergone a wrist arthrodesis for the management of a severe wrist contracture at Shriners Hospital, Portland between January 2016 and January 2021 were identified (n=23). A chart review was undertaken to obtain data-demographic, operative, clinical, and radiographic. All patients were then contacted to participate in 2 patient-reported outcome questionnaires (74% response agreement), a numerical rating scale (NRS), based on the visual analog scale (VAS) and the disability analog scale (DAS). Results were assessed with the aid of descriptive statistics, means and percentages with the primary focus of determining overall patient satisfaction with the procedure. Twenty-three patients were included in the review, and 74% took part in the prospective survey. Included were 10 patients with hemiplegia, 4 with triplegia, 7 with quadriplegia, 1 with a diagnosis of Rhett syndrome, and 1 with a history of traumatic brain injury. All patients achieved radiologic union by a mean of 8 weeks, and 87% obtained a neutral postoperative wrist alignment. The NRS showed 88% of patients were highly satisfied with their results; specifically, 82% had an improved appearance, 53% improved function, 71% improved daily cares, and 65% improved hygiene. The postoperative DAS score averaged 4.7 of 12 indicating mild disability. When looking at how a patient's diagnostic subtype affected outcome results, patients with triplegia reported less improvement and greater disability on the NRS and DAS, averaging 9.5 (severe disability on the DAS). The GMFCS classification had less correlation with outcomes. Wrist arthrodesis is a good option for the management of pediatric spastic wrist contractures, with limited complications and an overall high patient/carer satisfaction rate. Level IV-case series.

Statistical analysis plan for the PRO B study: open-label, superiority randomised controlled trial of alarm-based patient-reported outcome monitoring in patients with metastatic breast cancer

BackgroundWith an increasing collection of patient-reported outcomes (PROs) to measure health-related quality of life (HRQoL) in oncological patients, there is still a lack of standardised strategies on how to interpret and use these data in patient care. Prior research has shown support for the use of digital PRO monitoring together with alarm systems to notify clinicians when the PRO values are deteriorating. This system has demonstrated advantages in improving HRQoL and increasing survival rates among oncology patients. Hence, we designed the PRO B study, a superiority multi-centre randomised controlled trial, to investigate the effects of alarm-based monitoring in metastatic breast cancer patients in Germany. The study protocol for the PRO B study was published in September 2021, and this manuscript describes a formal statistical analysis plan (SAP) for the PRO B study to improve the transparency and quality of this trial.Methods and designThe trial aimed to recruit 1000 patients with metastatic breast cancer. However, as of the completion of recruitment on June 15, 2023, we have successfully enrolled 924 patients from 52 breast cancer centres. Patients were 1:1 stratified randomised to the intervention and control groups. App-based PRO questionnaires are sent weekly to the intervention group and every 3 months to the control group. Only patients in the intervention group trigger an alarm if their PRO scores deteriorate, and they are subsequently contacted by the local care team within 48 h. The primary outcome is the fatigue score at 6 months, and secondary outcomes are other HRQoL and overall survival. Evaluation of the superiority of the intervention will be done using a linear mixed model with random intercepts for study centres.ConclusionThis detailed SAP defines the main components of the statistical analysis for the PRO B study to assist the statistician and prevent bias in selecting analysis and reporting findings. Version 1 of the SAP was finalised on January 18, 2024.Trial registrationDRKS (German Clinical Trials Register) DRKS00024015. Registered on February 15, 2021.

(040) Anterolateral Thigh Flap Phalloplasty: Outcomes Following Genital Gender Affirmation Surgery

Abstract Introduction Genital gender affirmation surgery (gGAS) is a key part of the management of patients with gender dysphoria. The anterolateral thigh (ALT) free flap was proposed as an alternative to the more common radial forearm free flap (RFFP) phalloplasty due to the twin advantages of a more easily hidden scar and improved flexibility in the length of the flap. A pedicled variant of the ALT flap offers greater security of vascular supply for suitable patients, and if suitable and desired the flap can be formed with an integrated urethra, facilitating voiding while standing. Objective The aim of the study is to report the contemporary surgical and functional outcomes of ALT phalloplasty. Methods All individuals undergoing ALT flap phalloplasty for genital gender affirmation surgery (GAS) between 2009 and 2023 nationwide were included. Individuals were managed in accordance with the World Professional Association for Transgender Health (WPATH) Standards of Care version 7. Data were extracted from a prospectively collected single surgeon database and medical records. Functional outcomes were assessed by non-validated questionnaire. Statistical analysis included Mann-Whitney, chi-squared, Fisher’s exact and unpaired T tests. Results Surgical outcomes: A total of 87 patients were included in the study. More than half (45, 51.7%) had an integrated urethra. The median length of stay following surgery was six days (IQR 6-7). Eleven patients (12.6%) had a 30-day Clavien-Dindo 3b complication or greater. Three patients (3.4%) experienced total flap loss. A further three patients (3.4%) had partial flap loss. Three patients (3.4%) required a salvage phalloplasty later. Thirteen patients (out of 42, 31.0%) without an integrated urethra went onto have a free flap urethroplasty. Functional outcomes: Thirty-nine individuals (44.8%) completed the patient reported outcomes questionnaire. Most patients (77%) were generally satisfied with their phalloplasty. More than half (56%) with an erectile device used their phallus for penetrative intercourse, while one in five reported experiencing orgasm using their phallus alone. Following urethral lengthening, all respondents were able to void standing. There was no significant difference in mean satisfaction scores for those with an integrated urethra compared to those without (4.18/5 vs 3.82/5, p=.45), as well as between those with or without an erectile device, respectively (3.78/5 vs 4.10/5, p=.26). Conclusions This study is one of the largest single centre cohorts of ALT flap phalloplasty reported to date. It further supports the use of the ALT flap as one of the main genital GAS options for transmen. Over half of the patients studied had an integrated urethra, the gold standard for urethral lengthening. Patient reported functional outcomes were promising, with similar scores regardless of urethral lengthening or erectile device insertion. Disclosure No.

Psychological support for individuals with differences of sex development (DSD)

ObjectiveCongenital conditions with atypical development of chromosomal, gonadal, or anatomic sex characteristics are referred to as Differences of Sex Development (DSD). Psychosocial care is recommended to be an integral part of clinical management for individuals with DSD. Few studies have examined the perceived need for, utilization of and the opinions of individuals with DSD regarding psychological support. MethodsThis cross-sectional study was part of a European multicentre study in 14 different medical centres in six countries. In total, 1040 individuals with DSD participated in a patient-reported outcome questionnaire asking about experiences and opinions regarding psychological support in DSD care. ResultsA majority of the participants reported that they had not received psychological support, in childhood and/or adolescence (70.6%, n = 692) nor in adulthood (67.9%, n = 661). Need for psychological support in childhood and/or adolescence was reported by 51.3% (n = 503), need for psychological support in adulthood, was reported by 49.5% (n = 482). The majority (80.2%; n = 718) agreed with the statement that people with DSD should always be offered psychological support. According to 78.7% (n = 697) of the participants, parents of children with DSD should always be offered psychological support. ConclusionOur findings support the existing consensus that psychological support should be an integral part of DSD care. The discrepancy between received and perceived need for psychological support suggests that individuals with DSD experience barriers to access mental health care services. Psychosocial and psychological services for children, adolescents and adults should therefore be available and offered throughout the lifespan to individuals with DSD.

How glaucoma patients balance between the advantages and disadvantages of acceptance to the recommended treatment regimen in real-life: Perspectives from a middle-income country.

Patients with primary open-angle glaucoma (POAG) are required to take long-term treatments with topical medications to halt disease progression. This cross-sectional survey aimed to describe the level of acceptance of Brazilian patients toward the long-term treatment with eyedrops and to find possible correlates of high acceptance. POAG patients were recruited from the Glaucoma Service-Santa Casa of Sao Paulo, Sao Paulo, Brazil. Clinical and demographic data were retrieved from participants' electronic records. All patients answered the ACCEPT© questionnaire. This is a generic patient-reported outcome questionnaire specifically developed to assess patients' acceptance of long-term medications and not adherence. Summed scores and those for each domain were calculated to range from 0 to 100 with a higher score indicating greater acceptance. The sample comprised 96 patients with POAG. The mean age was 63.2 ± 8.9 years; 48 were male and 48 female; 55 (57.3%) were white, 36 (37.5%) African-Brazilian, and 5 (5.2%) were of mixed color; most patients (97.9%) had less than high school degree and all had a family income <USD10,000. The mean summed ACCEPT score was 66.2 ± 5.0. Scores for specific domains were higher for your medication (82.5 ± 35.9), side effects of your medication (86.9 ± 37.4), and effectiveness of your medication (73.3 ± 8.3). There was no association between higher scores and any sociodemographic variables. In this cohort of Brazilian patients with glaucoma, acceptance of treatment was high. Patients seems to have noticed more benefits than risks to the recommended treatment regimen.

Validation of a patient-reported knee-specific outcome questionnaire specifically intended to assess pain, stiffness, and functional activities in Indians with osteoarthritis knees: an observational cross-sectional study

Background: Patient-reported outcome measurement assessments that are frequently used for knee joint disorders have ceiling effect. This restriction is allegedly not there in New Hindi score. Purpose of this study is to validate New Hindi Score in patients with osteoarthritis knee. Methods: Level II Prospective cohort study was conducted. In pilot study, 20 patients had their comprehension of New Hindi Score assessed. A prospective cohort study involving 200 individuals with osteoarthritis knee Kl grade 2 and 3 was conducted in AIIMS, Rishikesh, Uttarakhand. Patients' overall age 57.76±8.63 years, both mean and SD. There were 125 females &amp; 75 males. Men's mean &amp; SD were 59.52±9.39 while women's 56.70±8.00. All patients were requested to complete WOMAC, KOOS, OKS &amp; New Hindi Score questionnaires. Validity, reliability, repeatability of New Hindi Score for knee function in KOA patients were evaluated. There's no floor ceiling effect. New Hindi Score's validity, responsiveness, and floor ceiling effect were assessed. Validity was measured using the Pearson correlation coefficient. Results: In pilot study participants answered all question accurately. New Hindi Score shows moderate correlation with WOMAC &amp; weak correlation with OKS and KOOS (Pearson coefficients of 0.45, 0.21, and 0.28, respectively) with 95% confidence interval, indicating strong construct validity in primary study. There was no floor or ceiling effect seen. Conclusions: For KOA patients, New Hindi Score exhibits strong levels of validity, reliability, and reliability. As result, it can be effectively applied to research knee function in Indian population.  

The Patient Journey in Facial Aesthetics: Findings from a European Consensus Meeting on Improving the Quality of Life for Patients Receiving Botulinum Toxin Injections.

Successful treatment of facial lines with botulinum toxin is largely dependent on patient satisfaction; thus, a structured treatment journey that uses patient-reported outcomes (PROs) is helpful for maximizing botulinum toxin results. To develop a patient-centric approach for botulinum toxin injections in facial aesthetics, a group of clinicians met to provide opinions on an optimal treatment journey that uses PROs to quantify treatment benefits on patient quality of life. A multidisciplinary panel of 9 clinicians with expertise in facial aesthetic procedures convened for an advisory board that was preceded by and followed up with a structured, multistep consensus discussion. Based on current literature, the panel's expertise, structured questions, and group discussion, panelists assessed, reconciled, and agreed upon on a patient journey for botulinum toxin treatment in facial aesthetics. Panelists agreed that an optimal patient journey includes screening, assessment, treatment, posttreatment, and follow-up visits. A compact, easy-to-complete, and digital PRO questionnaire should be provided before the visit. During screening, thorough assessments are integral for a successful patient journey because they provide an opportunity to understand treatment goals, address patient concerns, discuss risks and benefits, obtain medication lists/medical history, and take pretreatment photographs. Treatment strategies should include discussing and educating on the approach/choice of botulinum toxin and ensuring patients are comfortable. Posttreatment, clinicians should request intense muscle movements to enhance product uptake and be available to address patient concerns. Finally, during follow-up, PRO questionnaires can be provided to gauge patient satisfaction with treatment, and pretreatment photographs can be provided to allow patients to track their progression. Follow-ups should be scheduled with new patients or those reporting low satisfaction. Establishing a relationship, being aware of the patient's goals, and developing an individualized care plan allows for a structured, patient-centered treatment journey that promotes positive aesthetic outcomes.

Characteristics and patient-reported outcomes of long-term lung cancer survivors.

Due to advances in screening and treatment of lung cancer, there has been increased interest in long-term lung cancer survivors (LTLCS). The aim of this study was to evaluate the prevalence of LTLCS, their characteristics and patient-reported outcomes (PROs) of LTLCS. Cross-sectional study that included patients diagnosed with primary lung cancer between Jan 2012 and Dec 2016 whose overall survival (OS) was greater than 5 years. A self-administered questionnaire was applied, including European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30), Patient Health Questionnaire-4 (PHQ-4) and two open questions regarding quality of life (QoL) and suggestions for improvements. Factors potentially related to QoL were analysed. Of 767 lung cancer patients, 158 (20.6%) were LTLCS and LTLCS' proportion increased yearly. Most patients were male (70.9%) with median age of 65 [interquartile range (IQR), 56-71] years. Fifty-seven percent had adenocarcinoma, 66.2% were diagnosed at early stages but 8.9% were at stage IV. During follow-up, 77.1% quitted smoking, 31.8% had disease progression/relapse and 15.2% developed other tumours. Of all living LTLCS, 100 (85%) patients answered the PROs questionnaire. The median Global Health score was 66.67 (IQR, 50-83), social functioning had the best score and emotional functioning the worst. Pain and fatigue were the symptoms with the worst impact on QoL. PHQ-4 identified mental distress in 36% and patients with a lower QoL were more likely to present anxiety (35.3% vs. 9.4%, P=0.007) or depression (27.9% vs. 3%, P=0.006). In the open questions, patients reported pain (17%), lack of familiar/financial support (16%), dyspnoea (14%), depression (8%), concern for the future (8%) and limitations performing daily activities (8%) as the aspects with most impact in QoL. The most suggested measures were improvement of care provided by health institutions (25%) and better social support (16%). Prevalence of LTLCS is increasing and survivors may experience a high prevalence of anxiety and depression as well as a high disease burden affecting QoL. Therefore, it's important to provide multidisciplinary continuous patient-centred care and a careful follow-up for all lung cancer patients, including LTLCS.

Administering selected subscales of patient-reported outcome questionnaires to reduce patient burden and increase relevance: a position statement on a modular approach

Patient-reported outcome (PRO) questionnaires considered in this paper contain multiple subscales, although not all subscales are equally relevant for administration in all target patient populations. A group of measurement experts, developers, license holders, and other scientific-, regulatory-, payer-, and patient-focused stakeholders participated in a panel to discuss the benefits and challenges of a modular approach, defined here as administering a subset of subscales out of a multi-scaled PRO measure. This paper supports the position that it is acceptable, and sometimes preferable, to take a modular approach when administering PRO questionnaires, provided that certain conditions have been met and a rigorous selection process performed. Based on the experiences and perspectives of all stakeholders, using a modular approach can reduce patient burden and increase the relevancy of the items administered, and thereby improve measurement precision and eliminate wasted data without sacrificing the scientific validity and utility of the instrument. The panelists agreed that implementing a modular approach is not expected to have a meaningful impact on item responses, subscale scores, variability, reliability, validity, and effect size estimates; however, collecting additional evidence for the impact of context may be desirable. It is also important to recognize that adequate rationale and evidence (e.g., of fit-for-purpose status and relevance to patients) and a robust consensus process that includes patient perspectives are required to inform selection of subscales, as in any other measurement circumstance, is expected. We believe that the considerations discussed within (content validity, administration context, and psychometric factors) are relevant across multiple therapeutic areas.

Patient reported outcomes (PROs) and user experiences of young children with type 1 diabetes using t:slim X2 insulin pump with control-IQ technology

ObjectiveExamine patient-reported outcomes (PROs) after the use of t:slim X2 insulin pump with Control-IQ technology (CIQ) in young children with type 1 diabetes. MethodsChildren with type 1 diabetes, ages 2 to < 6 years (n = 102), were randomly assigned 2:1 to either CIQ or standard care (SC) with pump or multiple daily injections (MDI) plus continuous glucose monitoring (CGM) for 13 weeks. Both groups were offered to use CIQ for an additional 13 weeks after the randomized control trial’s (RCT) completion. Guardians completed PRO questionnaires at baseline, 13-, and 26-weeks examining hypoglycemia concerns, quality of life, parenting stress, and sleep. At 26 weeks, 28 families participated in user-experience interviews. Repeated measures analyses compared PRO scores between systems used. ResultComparing CIQ vs SC, responses on all 5 PRO surveys favored the CIQ group, showing that CIQ was superior to SC at 26 weeks (p values < 0.05). User-experience interviews indicated significant benefits in optimized glycemic control overall and nighttime control (28 of 28 families endorsed). All but 2/28 families noted substantial reduction in management burden resulting in less mental burden and all but 4 stated that they wanted their children to continue using CIQ. ConclusionsFamilies utilizing CIQ experienced glycemic benefits coupled with substantial benefits in PROs, documented in surveys and interviews. Families utilizing CIQ had reduced hypoglycemia concerns and parenting stress, and improved quality of life and sleep. These findings demonstrate the benefit of CIQ in young children with type 1 diabetes that goes beyond documented glycemic benefit.