Patient-reported Questionnaires Research Articles

The persian version of the fear-avoidance beliefs questionnaire among iranian post-surgery patients: a translation and psychometrics

IntroductionFear-avoidance beliefs (FAB) play a crucial role in the treatment outcomes of post-surgery patients. These beliefs can lead to activity avoidance, increased pain, and decreased quality of life. Therefore, accurately measuring these beliefs in Iranian patients is of significant importance. The Fear-Avoidance Belief Questionnaire (FABQ) is a patient-reported questionnaire that evaluates individuals’ FAB. Since the validity and reliability of the Persian version of FABQ (FABQ-P) have not been assessed based on the Iranian population and sociocultural contexts, the current study has been implemented to determine the reliability and validity of the FABQ-P among Iranian post-operative patients by translation and psychometric properties.MethodsThis methodological study conducted in 2023, a sample of 400 patients who had undergone surgery were selected using a convenience sampling method. The scale used in the study was translated and its psychometric properties were evaluated through network analysis and assessments of construct validity (including exploratory and confirmatory factor analysis), convergent validity, and discriminant validity. Additionally, the study assessed the internal consistency of the scale.ResultsThe MLEFA results with Promax and Kaiser Normalization rotation yielded two factors explaining 57.91% of the variance, encompassing 13 items. Also, the model was approved by CFA. Convergent and discriminant validity have been confirmed through the following criteria: Average Variance Extracted (AVE) exceeding 0.5, Composite Reliability (CR) surpassing 0.7, and Heterotrait-Monotrait Ratio of Correlations (HTMT) equating to 0.597. As for reliability, Cronbach’s alpha, composite reliability (CR), and MaxR for all constructs were greater than 0.7, demonstrating good internal consistency.ConclusionAs demonstrated by the results, the FABQ-P has a satisfactory level of reliability along with authentic validity according to the sociocultural contexts of Iranian post-operative patients.

GRAPPA 2023 Annual Meeting Report: Hot Topic Session on Measurement of Musculoskeletal Symptoms in Psoriatic Disease.

During the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA) 2023 annual meeting, the International Dermatology Outcome Measures (IDEOM) psoriatic disease (PsD) workgroup presented an update on their efforts toward measurement of musculoskeletal (MSK) symptoms in patients with PsD. Dr. Joseph Merola initiated the presentation emphasizing the vital importance of assessing MSK symptoms in patients with psoriasis (PsO) regardless of whether they have been diagnosed with psoriatic arthritis (PsA). He also discussed existing challenges for evaluating MSK symptoms in patients with PsO without a PsA diagnosis. Dr. Lourdes Perez-Chada then presented their work on the development and validation of the IDEOM Musculoskeletal Questionnaire (MSK-Q), a patient-reported questionnaire developed by the IDEOM to capture the intensity and impact of MSK symptoms on quality of life in patients with PsO with or without PsA. Dr. Perez-Chada also introduced a set of ongoing studies employing the IDEOM MSK-Q, highlighting the potential effects of the data collected through this innovative tool.

Anti‑CGRP monoclonal antibodies in resistant migraine: preliminary real-world effectiveness and clinical predictors of response at twoyears.

Monoclonal antibodies targeting calcitonin gene-related peptide (anti-CGRP mAbs) have shown clinical effectiveness and safety in randomized clinical studies. However, long-term studies in clinical practice remain limited. To assess the long-term effectiveness, clinical predictors and safety of three anti-CGRP mAbs (erenumab, galcanezumab, fremanezumab) in resistant migraine patients. A single-center retrospective study was conducted from December 2019 to June 2023 involving 120 resistant migraine patients who received at least a month of anti-CGRP mAbs treatment. Patients completed a headache diary that included monthly acute medication intake (MAM), monthly migraine days (MMD), adverse events as well as completed Patient-Reported Outcome questionnaires (MIDAS [Migraine Disability Assessment] and Headache Impact Test 6 [HIT-6]). The number of patients achieving a ≥ 50% reduction in monthly migraine days was determined and classified as ≥ 50% responders, and baseline parameters and logistic regression between responders and non-responders were analyzed to identify potential predictors of response. Adverse events were registered in every follow-up. Treatment with anti-CGRP mAbs led to reductions in MIDAS, HIT-6, MMD and MAM from baseline to 6-24months. At 6-12months, responders (61% and 57%, respectively) exhibited lower baseline MMD and MAM. Medication overuse was associated with non-responders from 6 to 24months and it was identified as a negative predictor of treatment effectiveness (OR 0.23, 95% CI 0.07-0.74; p = 0.014). Anti-CGRP mAbs prove effectiveness and safety over a 24-month period in a RM population. Patients with no medication overuse and lower basal MMDs and MAM may respond better to anti-CGRP mAbs.

The Revised Reflux Symptom Index (R-RSI): development, internal and external validation study.

This study proposes a revised version of the Reflux Symptom Index (R-RSI), a seventeen-item questionnaire that was revised to increase the suspicion of laryngo-pharyngeal reflux disease (LPRD). Internal validation involved 213 participants, comprising160 subjects without a previous LPRD diagnosis and 53 subjects with a self-reported previous diagnosis of LPRD with or without gastro-esophageal reflux disease (GERD). Test-retest reliability and internal consistency were calculated. For the external validation, 56 patients (independent from the previous cohort) were enrolled to explore the R-RSI Screening Properties and determine a cut-off using 24-h MII-pH as the gold standard. R-RSI test-retest reliability was high, both for the total score (ICC: 0.970) and for each item (ranging from 0.876 to 0.980). Cronbach's alpha was 0.910, indicating excellent internal consistency of the questionnaire. Participants with a previous self-reported diagnosis scored significantly higher (mean 24.94 ± 7.4; median 26, IQR 20-29) than those without a previous diagnosis (mean 4.66 ± 5.3; median 4, IQR 1-6) (p-value < 0.0001). Participants with both previous LPRD and GERD diagnoses had higher scores (27.20 ± 7.8) compared to those with only LPRD (21.77 ± 5.5) (p-value=0.003). Using 24-h MII-pH diagnosis as a gold standard, the optimal R-RSI cut-off point was determined to be 18, with a sensitivity of 84.5% and a specificity of 81.8%, positive predictive value of 95% and negative predictive value of 60%. Our results suggest that the R-RSI may be useful to suspect LPRD, with or without GERD. The R-RSI is a self-administered patient-reported outcome questionnaire that demonstrates excellent reliability and high screening properties. Employing a cut-off of ≥18 the R-RSI can assist in diagnosing and monitoring LPRD.

Eustachian Tube Dysfunction Diagnostic Pathway-What Is the Current State of the Art and How Relevant Is Chronic Nasal Disease?

Background: Eustachian tube dysfunction (ETD) presents a complex diagnostic challenge in otolaryngology, compounded by its multifaceted nature and overlapping symptoms with chronic nasal disease. This article examines the intricacies of ETD diagnosis, emphasising the necessity for a consensus on diagnostic procedures. Methods: A review of the literature was performed through the OVID research tool in the Pubmed/Medline databases to identify relevant articles that discuss eustachian tube dysfunction diagnostics as well as its correlation with chronic nasal disease. Results: The literature review harvested 201 articles, and only 51 of them were included in the full text review. A consensus statement was identified on eustachian tube dysfunction, function and diagnostics. It appears that there is significant variability in the diagnostic tools used to identify eustachian tube dysfunction. The main diagnostic approaches used are tympanometry, tubomanometry and sonotubometry, combined with the Patient-Reported Outcome Measure ETDQ-7 questionnaire to support the diagnosis of the condition. Nasal pathology is mostly absent from the retrieved studies, while ear pathology is more commonly mentioned in the current literature. Conclusions: There is no gold standard diagnostic tool to determine the presence of eustachian tube dysfunction. Further discussion, large multicentre studies and focused research are required to achieve a consensus on a diagnostic approach. The authors suggest a diagnostic pathway that combines subjective and objective diagnostic tools to determine the presence of eustachian tube dysfunction. This pathway is simple and can be used in district ENT departments, highlighting the nasal pathology relevance to ETD.

Relationship of Fatigue, Pain Interference, and Physical Disability in Children Newly Diagnosed With Juvenile Idiopathic Arthritis.

Our objectives were to quantify the relationships among fatigue, pain interference, and physical disability in children with juvenile idiopathic arthritis (JIA) and to test whether fatigue mediates the relationship between pain interference and physical disability in JIA. Patients enrolled within three months of JIA diagnosis in the Canadian Alliance of Pediatric Rheumatology Investigators (CAPRI) Registry between February 2017 and May 2023 were included. Their parents completed the Patient-Reported Outcomes Measurement Information System fatigue and pain interference short proxy questionnaires and the Childhood Health Assessment Questionnaire disability index at registry enrollment. Associations were assessed using Pearson correlations and multiple linear regression. Structural equation modeling (SEM) was used to test if fatigue mediates the relationship between pain interference and physical disability. Among 855 patients (61.4% female, 44.1% with oligoarthritis), most reported fatigue and pain interference scores similar to those in the reference population, but 15.6% reported severe fatigue and 7.3% reported severe pain interference, with wide variation across JIA categories. Fatigue was strongly correlated with pain interference (r = 0.72, P < 0.001) and with physical disability (r = 0.60, P < 0.001). Pain interference (β = 0.027, P < 0.001) and fatigue (β = 0.013, P < 0.001) were both associated with physical disability after controlling for each other and potential confounders. SEM supported our hypothesis that fatigue partially mediates the relationship between pain interference and physical disability. Our findings suggest both fatigue and pain interference are independently associated with physical disability in children newly diagnosed with JIA, and the effect of pain interference may be partly mediated by fatigue.

Impact of chemotherapy-induced peripheral neuropathy permanence on patients' preference to discontinue chemotherapy.

12046 Background: Chemotherapy-induced peripheral neuropathy (CIPN) diminishes patients’ functional ability and quality of life; approximately 40% of patients experience CIPN symptoms 3 years after treatment. Due to the absence of effective CIPN treatments, ASCO guidelines recommend discontinuing chemotherapy in patients experiencing intolerable CIPN symptoms. Little is known about patient’s preference to continue or discontinue chemotherapy when experiencing CIPN, and whether the potential permanence of CIPN symptoms affects this decision. The objective of this prospective observational study was to determine the effect of CIPN symptom permanence on patients’ preference to discontinue chemotherapy. Methods: Patients receiving taxane and/or platinum chemotherapy for breast or colorectal cancer were enrolled in a prospective observational clinical study. Patients reported their CIPN severity using the EORTC CIPN20 patient-reported outcome questionnaire at the start of each treatment cycle. At the same time, patients were asked if they would prefer to continue or discontinue chemotherapy treatment under the hypothetical scenario that their current CIPN symptoms would be temporary or permanent. A generalized linear mixed effects model was used to determine the effect of CIPN severity, permanence, and other clinical variables on a patient’s decision to discontinue (vs. continue) chemotherapy treatment. Results: A total of 66 patients completed data collection at least one time, of whom 55 (83%) had breast cancer, 12 (18%) had metastatic disease, and 52 (79%) received a taxane. The odds a patient would prefer to discontinue chemotherapy treatment if their CIPN symptoms would be permanent (vs. temporary) were nearly 30 times greater (odds ratio (OR)=30.15, 95% confidence interval (CI): 15.73-57.79, p&lt;0.001, Table). There was a statistical trend toward preferring to discontinue chemotherapy treatment as CIPN symptom severity increased (OR=1.09, 95% CI: 1.00-1.19, p=0.063). Conclusions: The potential for CIPN symptoms to be permanent has a huge effect on whether patients want to continue or discontinue chemotherapy treatment. Additional data is needed on the incidence and predictors of permanent CIPN. More importantly, shared decision-making tools that convey the risk of permanent CIPN, and the potential risk of discontinuing chemotherapy treatment, are needed to facilitate patient-centered treatment decisions so that each patient with cancer achieves their personal treatment goals. [Table: see text]

#802 CENSUS-EU: a cross-sectional, study to assess the prevalence and burden of chronic kidney disease-associated pruritus in Europe

Abstract Background and Aims Chronic kidney disease-associated pruritus (CKD-aP) is a common condition that can negatively affect patients’ health-related quality of life (HRQoL). Despite this, there has been a lack of clarity on its prevalence and impact, leading to increasing interest in and need for an accurate overview of its epidemiology. Here we present the interim analyses of the real-world study CENSUS-EU to estimate the prevalence of CKD-aP and its impact on patients’ HRQoL. Method CENSUS-EU is a retrospective, cross-sectional, multicentre study across seven countries in Europe. Eligible patients (aged ≥18 years and on dialysis for ≥3 months) consenting to participate were asked to complete five patient-reported outcome questionnaires, one on CKD-aP severity (the Worst Itching Intensity Numerical Rating Scale) and four on HRQoL, including the 5-D itch scale and the integrated palliative care outcome scale symptom list for end-stage renal disease (IPOS-Renal). Patients also completed a survey on the communication and management of their pruritus, including current anti-pruritic medication use. In addition, the medical records of patients were assessed to gather retrospective information on clinical dialysis characteristics, treatment patterns and healthcare resource utilisation. In this presentation of the interim data, we report the prevalence of pruritus and the distribution of severity subgroups, how pruritus severity affects the disability score from the 5-D itch (impact on sleep, leisure, errands, and work/school), and the sleep and depression scores from the IPOS-Renal questionnaire. Results The interim analysis included 1,482 patients, of which 59.9% were ≥65 years of age and 61.2% were male. Patient characteristics were balanced across severity subgroups. Overall prevalence of CKD-aP was 52.6% (95% confidence interval 50.02, 55.10); 21.5% of patients experienced mild pruritus, 17.5% moderate pruritus, and 13.5% severe pruritus (Fig. 1). As pruritus severity increased from no pruritus to severe pruritus, patients reported greater difficulty sleeping, as shown by increasing scores for question 2 of the IPOS-Renal questionnaire (Fig. 2A). Patients with more severe pruritus also reported feeling depressed more often than those with less severe or no pruritus (Fig. 2B). There were also increasing scores for the 5-D itch disability subscale (Fig. 2C). Patients with moderate or severe pruritus were also hospitalised more often than patients with mild or no pruritus (mean 1.7 and 1.9 hospitalisations per year, respectively). Overall, 17.9% of patients were receiving at least one ongoing anti-pruritic treatment, and the proportion of patients using at least one anti-pruritic treatment became greater with increasing itch intensity. Despite the impact of pruritus on HRQoL, 40.6% of patients with severe pruritus who had ever suffered from itch while on dialysis were not receiving anti-itch treatment. Conclusion The interim results of this cross-sectional study suggest that approximately 50% of patients on haemodialysis experience CKD-aP. The data reveal that as pruritus severity increases, the effect on sleep and depression scores becomes greater, potentially impacting more deeply on patients’ HRQoL.

High-precision stereotactic irradiation for focal drug-resistant epilepsy versus standard treatment: a randomized waitlist-controlled trial (the PRECISION trial)

IntroductionThe standard treatment for patients with focal drug-resistant epilepsy (DRE) who are not eligible for open brain surgery is the continuation of anti-seizure medication (ASM) and neuromodulation. This treatment does not cure epilepsy but only decreases severity. The PRECISION trial offers a non-invasive, possibly curative intervention for these patients, which consist of a single stereotactic radiotherapy (SRT) treatment. Previous studies have shown promising results of SRT in this patient population. Nevertheless, this intervention is not yet available and reimbursed in the Netherlands. We hypothesize that: SRT is a superior treatment option compared to palliative standard of care, for patients with focal DRE, not eligible for open surgery, resulting in a higher reduction of seizure frequency (with 50% of the patients reaching a 75% seizure frequency reduction at 2 years follow-up).MethodsIn this waitlist-controlled phase 3 clinical trial, participants are randomly assigned in a 1:1 ratio to either receive SRT as the intervention, while the standard treatments consist of ASM continuation and neuromodulation. After 2-year follow-up, patients randomized for the standard treatment (waitlist-control group) are offered SRT. Patients aged ≥ 18 years with focal DRE and a pretreatment defined epileptogenic zone (EZ) not eligible for open surgery will be included. The intervention is a LINAC-based single fraction (24 Gy) SRT treatment. The target volume is defined as the epileptogenic zone (EZ) on all (non) invasive examinations. The seizure frequency will be monitored on a daily basis using an electronic diary and an automatic seizure detection system during the night. Potential side effects are evaluated using advanced MRI, cognitive evaluation, Common Toxicity Criteria, and patient-reported outcome questionnaires. In addition, the cost-effectiveness of the SRT treatment will be evaluated.DiscussionThis is the first randomized trial comparing SRT with standard of care in patients with DRE, non-eligible for open surgery. The primary objective is to determine whether SRT significantly reduces the seizure frequency 2 years after treatment. The results of this trial can influence the current clinical practice and medical cost reimbursement in the Netherlands for patients with focal DRE who are not eligible for open surgery, providing a non-invasive curative treatment option.Trial registrationClinicaltrials.gov Identifier: NCT05182437. Registered on September 27, 2021.

Online Adaptive MR-Guided Ultrahypofractionated Radiotherapy of Prostate Cancer on a 1.5 T MR-Linac: Clinical Experience and Prospective Evaluation.

The use of hypofractionated radiotherapy in prostate cancer has been increasingly evaluated, whereas accumulated evidence demonstrates comparable oncologic outcomes and toxicity rates compared to normofractionated radiotherapy. In this prospective study, we evaluate all patients with intermediate-risk prostate cancer treated with ultrahypofractionated (UHF) MRI-guided radiotherapy on a 1.5 T MR-Linac within our department and report on workflow and feasibility, as well as physician-recorded and patient-reported longitudinal toxicity. A total of 23 patients with intermediate-risk prostate cancer treated on the 1.5 T MR-Linac with a dose of 42.7 Gy in seven fractions (seven MV step-and-shoot IMRT) were evaluated within the MRL-01 study (NCT04172753). The duration of each treatment step, choice of workflow (adapt to shape-ATS or adapt to position-ATP) and technical and/or patient-sided treatment failure were recorded for each fraction and patient. Acute and late toxicity were scored according to RTOG and CTC V4.0, as well as the use of patient-reported questionnaires. The median follow-up was 12.4 months. All patients completed the planned treatment. The mean duration of a treatment session was 38.2 min. In total, 165 radiotherapy fractions were delivered. ATS was performed in 150 fractions, 5 fractions were delivered using ATP, and 10 fractions were delivered using both ATS and ATP workflows. Severe acute bother (G3+) regarding IPS-score was reported in five patients (23%) at the end of radiotherapy. However, this tended to normalize and no G3+ IPS-score was observed later at any point during follow-up. Furthermore, no other severe genitourinary (GU) or gastrointestinal (GI) acute or late toxicity was observed. One-year biochemical-free recurrence survival was 100%. We report the excellent feasibility of UHF MR-guided radiotherapy for intermediate-risk prostate cancer patients and acceptable toxicity rates in our preliminary study. Randomized controlled studies with long-term follow-up are warranted to detect possible advantages over current state-of-the-art RT techniques.

Prevalence and clinical characteristics of Sarcopenia in older adult patients with stable chronic obstructive pulmonary disease: a cross-sectional and follow-up study

BackgroundThe relationship between sarcopenia and chronic obstructive pulmonary disease (COPD) has been increasingly reported, and there is some overlap regarding their clinical features and pulmonary rehabilitation (PR) strategies. No Korean study has reported the actual prevalence of sarcopenia in patients with stable COPD who are recommended for pulmonary rehabilitation. This study evaluated the prevalence and clinical features of sarcopenia in older adult outpatients with stable COPD and the changes after 6 months.MethodsIn this cross-sectional and 6-month follow-up study, we recruited 63 males aged ≥ 65 diagnosed with stable COPD. Sarcopenia was diagnosed using the AWGS 2019 criteria, which included hand grip strength testing, bioelectrical impedance analysis, Short Physical Performance Battery administration, and Strength, Assistance with walking, Rising from a chair, Climbing stairs, and Falling screening tool administration. A 6-minute walk test (6 MWT) was conducted, forced vital capacity (FVC), forced expiratory volume in 1 s (FEV1), maximal inspiratory and expiratory pressures (MIP and MEP, respectively) and peak expiratory flow (PEF) were assessed, and patient-reported questionnaires were administered.ResultsAt baseline, 14 (22%) patients were diagnosed with possible sarcopenia, and eight (12.6%) were diagnosed with sarcopenia. There were significant differences in the age; body mass index; Body mass index, airflow Obstruction, Dyspnea, and Exercise index; modified Medical Research Council dyspnea scores; and International Physical Activity Questionnaire scores between the normal and sarcopenia groups. Whole-body phase angle, MIP, MEP, PEF, and 6-minute walk distance (6 MWD) also showed significant differences. Over 6 months, the proportion of patients with a reduced FEV1 increased; however, the proportion of patients with sarcopenia did not increase.ConclusionA relatively low prevalence of sarcopenia was observed in older adult outpatients with stable COPD. No significant change in the prevalence of sarcopenia was found during the 6-month follow-up period.Trial registrationThe study was registered with the Clinical Research Information Service (KCT0006720). Registration date: 30/07/2021.

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&amp;lt; 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&amp;lt; 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.

Efficiency assessment of follow-up methodology of patients with knee replacement to predict post-surgical functionality: a protocol for randomised control PROKnee trial

IntroductionEven when total knee arthroplasty (TKA) is an extended treatment, most patients experience a suboptimal evolution after TKA. The objectives of this study are the following: (1) to determine the...

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.

Non-infectious adverse events of transperineal prostate biopsies performed under local anaesthesia.

To report non-infectious adverse events associated with transperineal prostate biopsy (TPBx) performed under local anaesthesia (LA) in an outpatient setting. This study reports secondary outcomes from the Norwegian arm of the prospective NORAPP study (ClinicalTrials.gov identifier NCT04146142) and included all patients referred for prostate biopsy from November 2019 to February 2021. Transperineal magnetic resonance imaging-transrectal ultrasonography fusion TPBx were taken using 40 mL 1% lidocaine with 4 mL of 8.4% sodium bicarbonate placed in the perineal skin, under the prostatic apex, in the m. levator ani bilaterally, and along the path of the needle. Follow-up using patient-reported questionnaires was done immediately after TPBx, and after 2 weeks and 2 months. Pain was reported using a visual analogue scale (VAS) during placement of the LA, and during and after TPBx. Haematuria and acute urinary retention (AUR) rates were recorded. We included 402 patients, and the response rate was 99.8% (401/402). The median (interquartile range [IQR]) age was 69 (63-74) years, the prostate volume was 40 (27-58) mL, the prostate-specific antigen level was 7.0 (4.5-11) ng/mL, and the number of biopsy cores taken was 8 (6-10). The median (IQR) VAS pain score was 1 (1-2) during placement of LA, 1 (0-2) during TPBx, and 0 (0-0) after TPBx. Haematuria and AUR rates were 64% (95% confidence interval [CI] 60-69%) and 0.5% (95% CI 0.1-1.8%), respectively. No patients were hospitalised or required after the TPBx surgical intervention. Transperineal prostate biopsies can be performed under LA with limited discomfort to the patient and few post-TPBx adverse events.

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.

Sleep and circadian biomarkers of postoperative delirium (SLEEP-POD): protocol for a prospective and observational cohort study

IntroductionSurgical patients over 70 experience postoperative delirium (POD) complications in up to 50% of procedures. Sleep/circadian disruption has emerged as a potential risk factor for POD in epidemiological studies. This...

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.

Outcome of bleomycin electrosclerotherapy of slow-flow malformations in adults and children.

To evaluate the safety and clinical outcome of bleomycin electrosclerotherapy (BEST) for treating extracranial slow-flow malformations. In this retrospective investigation of a multicenter cohort presenting symptomatic slow-flow malformations, patient records were analyzed with respect to procedural details and complications. A treatment-specific, patient-reported questionnaire was additionally evaluated, obtained 3-12 months after the last treatment, to assess the subjective outcomes, including mobility, aesthetic aspects, and pain, as well as the occurrence of postprocedural skin hyperpigmentation. All outcome parameters were compared according to patients' age. Overall, 325 BEST treatments were performed in 233 patients after intralesional and/or intravenous bleomycin injection. The total complication rate was 10.2% (33/325), including 29/352 (8.9%) major complications. Patient-reported mobility decreased in 10/133 (8.8%), was stable in 30/113 (26.5%), improved in 48/113 (42.5%), and was rated symptom-free in 25/113 (22.1%) patients. Aesthetic aspects were rated impaired compared to baseline in 19/113 (16.8%), stable in 21/133 (18.6%), improved in 62/113 (54.9%), and perfect in 11/133 (9.7%) patients. Postprocedural skin hyperpigmentation occurred in 78/113 (69%) patients, remaining unchanged in 24/78 (30.8%), reduced in 51/78 (65.5%), and completely resolved in 3/78 (3.8%) patients. The median VAS pain scale was 4.0 (0-10) preprocedural and 2.0 (0-9) postprocedural. Children/adolescents performed significantly better in all parameters compared to adults (≥ 16 years) (mobility, p = 0.011; aesthetic aspects, p < 0.001; pain, p < 0.001). BEST is effective for treating slow-flow vascular malformations, with few but potentially significant major complications. Regarding patient-reported outcomes, children seem to benefit better compared to older patients, suggesting that BEST should not be restricted to adults. Bleomycin electrosclerotherapy is a safe and effective approach and therapy should not be restricted to adults due to good clinical outcomes in children.