Clinician-reported Outcomes Research Articles

NCOG-23. CLINICIAN AND PATIENT REPORTED OUTCOMES IN YOUNG ADULTS WITH GLIOBLASTOMA: RESULTS FROM A MULTIMODAL PROSPECTIVE STUDY

Abstract The median age for glioblastoma (GBM) is 65 years old, however, younger patients have a similarly dismal prognosis. The adolescent and young adult (AYA) population (15-39 years) is a unique and understudied population. Our study aimed to identify survivorship needs in adult patients in AYA population (<40 yrs) with GBM through analysis of patient and clinician-reported outcomes (PROs and CROs). The Molecular, Imaging and Neurological assessment Database (MIND-CNS) captures Neurological Assessment in Neuro-Oncology (NANO) scores, MDASI-BT and FACIT-SP12 responses at first clinic visit and follow-ups for patients with GBM. We tested associations of the two age groups (18-39 vs. 40 years and older) with each domain of NANO, MDASI-BT and FACIT-SP12 at baseline and at follow-up visits using multivariate logistic regression models, adjusting for MGMT promoter methylation and extent of resection. There were 104 patients with GBM enrolled, including 11 young adults, (median =30 years) and 93 older adults (median =65 years). Young and older adults scored similarly in the domains of NANO, MDASI-BT, and FACIT-SP12 at baseline. MDASI domains at follow-up showed statistically significant lower scores on weakness, fatigue, distress, irritability, drowsiness, and disturbed sleep in younger vs. older adults (adjusted p<0.05). Younger adults had less difficulty with memory and concentration, and their symptoms interfered less with work, walking, and overall enjoyment of life. They also reported feeling more peaceful, finding greater comfort and strength in their faith and beliefs. A comparison of NANO score at baseline and progression will be performed at data maturation. While PROs were similar at baseline, young adults reported lower severity in their physical symptoms and higher spiritual well-being compared to older adults at follow-up visits, highlighting different survivorship experiences. Data maturation will allow for deeper analysis to better inform supportive strategies.

Concordance of patient- and clinician-reported outcomes of acute radiation dermatitis in breast cancer.

The study evaluated the concordance between patient-reported outcomes (PRO) and clinician-reported outcomes (CRO) of acute radiation dermatitis (RD) symptoms following adjuvant radiotherapy for early-stage and locally advanced breast cancer. This is a secondary analysis of a multi-center randomized phase 3 trial (376 patients). Ordinal logistic regression analysis was used to compare the Skin Symptom Assessment (SSA) independently reported by both patients and clinicians. Concordance between patient- and clinician-reported SSAs for RD symptoms was measured by percent concordance, concordance index (C-statistic), and Cohen's Kappa. Analyses were performed across all patients in the original modified intention-to-treat analysis and those with only grade 2-3 (CTCAE) RD. PROs were significantly more severe than CROs across all RD symptoms (Odds Ratio [OR] > 1; p < 0.0001). Pigmentation (OR 5.4), blistering/peeling (OR 4.0), and pain/soreness (OR 3.9) were the most differentially reported symptoms. Poor-to-low concordance was noted between patient- and clinician-reported SSAs for all RD symptoms for the entire cohort (percent concordance < 50%, C-statistic 0.52-0.63, Cohen's Kappa 13.9-23.4%) and those with grade 2-3 RD (percent concordance < 50%, C-statistic 0.56-0.66, Cohen's Kappa 2.0-24.5%). Similarly, poor-to-low concordance was noted in both Mepitel film and standard-of-care arms. PROs and CROs have poor concordance in breast RD, and patients report worse outcomes than clinicians, regardless of RD severity or prophylaxis. PROs must be further integrated into routine clinical practice and clinical trial design to reduce the risk of underreporting symptoms.

Patient-Reported Hair Loss and Its Impacts as Measured by the Alopecia Areata Patient Priority Outcomes Instrument in Patients Treated with Ritlecitinib: The ALLEGRO Phase 2b/3 Randomized Clinical Trial.

The ALLEGRO phase 2b/3 study investigated the efficacy and safety of ritlecitinib in patients with alopecia areata (AA). To describe the impact of ritlecitinib on patient-reported hair loss using the Alopecia Areata Patient Priority Outcomes (AAPPO) instrument and evaluate the relationship between clinically meaningful hair regrowth and improvements in patient-reported impacts. In ALLEGRO-2b/3, patients aged ≥ 12 years with AA and ≥ 50% scalp hair loss received once-daily ritlecitinib 50 or 30 mg (± 4-week 200-mg daily loading dose), 10 mg, or placebo for 24 weeks andthen continued ritlecitinib or switched from placebo to ritlecitinib 200/50 or 50 mg for 24 weeks. The AAPPO instrument evaluated improvement in hair loss, emotional symptoms (ES), and activity limitations (AL) from weeks 4 to 48 (secondary endpoint). Mean changes in ES and AL domain scores and individual items at weeks 24 and 48 were calculated for Severity of Alopecia Tool (SALT) score ≤ 20 responders and nonresponders (exploratory endpoint). Overall, 718 patients were randomized. At week 24, 5-36% of patients receiving ritlecitinib 10-200/50 mg reported improvement in scalp hair loss versus 9% receiving placebo. The results for eyebrow, eyelash, and body hair loss were similar. Mean change from baseline in ES and AL scores at weeks 24 and 48 was small and similar between groups. Mean change was larger for individual hair loss and ES items at weeks 24 and 48 in SALT score ≤ 20 responders versus nonresponders. The AAPPO instrument demonstrated the beneficial impact of ritlecitinib on patient-reported hair growth, which was consistent with improvements in clinician-reported outcomes. NCT03732807. INFOGRAPHIC.

Platelet-rich plasma applications in the larynx: snake oil or stupendous?

To explore and summarize recent work examining the use of platelet-rich plasma (PRP) applications for vocal fold disorder. Intrachordal PRP injections have recently emerged as a treatment option for patients with damage to the lamina propria of the vocal fold (scar, atrophy, sulcus and inflammatory lesions). Studies support significant improvements in patient-reported and clinician-reported outcomes, and objective phonatory parameters. PRP demonstrates a good safety profile, absence of immune reactions, affordable cost model and improvement in mucosal wave features with resultant improved voice quality up to 12 months following treatment. PRP injection of the vocal folds is a novel repurposing of a previously validated technique, and emerging data shows consistent results across multiple centres, increasing confidence in use of this freely available material. Current published studies confirm improved voice outcomes compared with pretreatment measures and high patient satisfaction. Further research is required to properly assign the role of and ideal candidate for PRP applications, and to delineate durability of treatment.

Assessing disease control in inflammatory bowel disease: a real world cross-sectional study in the UK (PODCAST-IBD)

Objective Proportion Of suboptimal Disease Control And Strategy of Treatment in IBD (PODCAST-IBD) was an international real-world study which aimed to quantify disease control in IBD using STRIDE-II recommendations. Design/Method Cross-sectional assessment of IBD patients attending routine clinic appointments in four UK centers October 2022 to January 2023. Clinician-reported outcomes, patient-reported outcomes and retrospective data from medical chart review were used to assess IBD control against red flags aligned to STRIDE-II. Results Data were available from 198 UK patients. IBD was suboptimally controlled in 52.4% (54/103) of patients with Crohn’s disease (CD) and 45.3% (43/95) with ulcerative colitis (UC). Impaired quality of life (QOL), defined as Short inflammatory bowel disease questionnaire (SIBDQ) score <50, was the main contributor to suboptimal disease control. Suboptimal disease control has a detrimental impact on fatigue and disability with significantly lower mean Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F) score in suboptimally controlled disease (CD: 81.5 vs 125, UC: 87.4 vs 122.8) and IBD Disk. Suboptimal disease control results in higher health care resource use (HCRU) (CD: £4,746 vs £1,924; UC: £2,428 vs £1,121) and higher rates of work productivity loss (CD: 41.7% vs 11.9%, UC: 38.0% vs 22.6%). Conclusion IBD was suboptimally controlled in around one-half of patients. Impaired QOL was the most common contributor (64%, 62/97) to suboptimal control. Suboptimal control had a considerable economic impact; HCRU more than doubled and productivity fell. Physicians could consider regular QOL assessments to prompt timely disease monitoring to enable identification of early active disease and appropriate treatment.

The Eaton-Littler Ligament Reconstruction in Thumb Carpometacarpal Joint Instability: Outcomes and Prognostic Factors in 74 Patients.

The Eaton-Littler ligament reconstruction is widely used for thumb carpometacarpal (CMC) instability, yet the existing literature lacks a thorough analysis of the outcomes for nontraumatic instability. This study aimed to assess the outcomes of the Eaton-Littler ligament reconstruction and to identify prognostic factors associated with postoperative pain. Patients with nontraumatic CMC joint instability, unresponsive to conservative treatment, were included in this prospective study. The Visual Analogue Scale (VAS, range 0-100) for pain and the Michigan Hand Outcome Questionnaire (MHQ, range 0-100) total score were measured at intake and three and 12-months postoperative. Multivariable linear regression was used to analyze the association between preoperative variables and the 12-month MHQ pain score. Seventy-four patients undergoing Eaton-Littler ligament reconstruction were included. The median VAS pain score improved significantly (P<0.001) from intake (70 [interquartile range (IQR) 63-78]) to 12 months postoperatively (27 [IQR 7-56]). The mean MHQ total score also improved significantly (P<0.001) from intake (52, standard deviation (SD) 13) to 12 months (74, SD 17). All thumbs were stable at follow-up whilst preserving the range of motion. Grip and pinch strength also improved significantly following surgery. Undergoing a concurrent surgery during ligament reconstruction and a better MHQ pain score at intake were found to be predictors of a favorable postoperative MHQ pain score. Patient- and clinician-reported outcomes improved significantly at three and 12 months after Eaton-Littler ligament reconstruction. We advise concurrent hand pathologies resulting from instability (e.g., tendinitis, synovitis) to be treated simultaneously during ligament reconstruction. Therapeutic, II.

83 Prospective Evaluation of Gastrointestinal and Genitourinary Side Effects of Pelvic Radiotherapy: Association Between Patient-Reported Outcomes and Clinician-Reported Outcomes with a View to Improving Quality of Care

83 Prospective Evaluation of Gastrointestinal and Genitourinary Side Effects of Pelvic Radiotherapy: Association Between Patient-Reported Outcomes and Clinician-Reported Outcomes with a View to Improving Quality of Care

52758 Patient and clinician-reported outcomes with tirbanibulin 1% treatment for actinic keratosis in routine clinical practice across the U.S. according to treated area location (PROAK study)

52758 Patient and clinician-reported outcomes with tirbanibulin 1% treatment for actinic keratosis in routine clinical practice across the U.S. according to treated area location (PROAK study)

A systematic review of outcome measures evaluating treatment efficacy in vulval lichen sclerosus and evaluation of patients' priorities.

Vulval lichen sclerosus (VLS) is an inflammatory skin disease characterised by itching, apareunia, loss of vulval architecture and scarring. Heterogeneity in outcome reporting precludes comparison between treatments. This study aimed to systematically review outcome measures used to evaluate the efficacy of VLS treatments and present patients' treatment priorities. This review followed the PRISMA guidelines using a registered protocol (PROSPERO: CRD42022356738). Multiple databases were searched, along with grey literature on Clinicaltrials.gov, European Union Clinical Trials and International Standard Randomised controlled trial (RCT) registries. All RCTs assessing any treatment for VLS were eligible for inclusion. A total of 775 patients were assessed across 21 RCTs. The assessment tools reported outcomes in the following domains: patient-reported symptoms assessed with one validated scale in 12 studies and seven non-validated scales in nine studies; sexual function with validated female sexual function index and female sexual distress scale in two studies and two non-validated scales in two studies; quality of life with three validated scales in three studies and clinician-reported objective outcomes with two validated scales in three studies and six non-validated scales in fourteen studies. Histological changes were assessed in 10 studies and tissue biomechanics in one study. We also carried out an online survey completed by 809 women with VLS to assess their research and disease treatment priorities and identified validated outcome measures to assess these. There is high variability in assessing treatment outcomes for VLS. We identified validated assessment tools which could be implemented in VLS studies to evaluate the effectiveness of treatments.

Socioeconomic Differences in Patient Reported Outcome Measures 3 Months After Stroke: A Nationwide Swedish Register-Based Study.

There is a well-known association between low socioeconomic status (SES), poor survival, and clinician-reported outcomes after stroke. We aimed to assess socioeconomic differences in Patient Reported Outcome Measures 3 months after stroke. This nationwide cohort study included patients registered with acute stroke in the Swedish Stroke Register 2015-2017. Patient Reported Outcome Measures included activities of daily living (mobility, toileting, and dressing), and poststroke symptoms (low mood, fatigue, pain, and poor general health). Information on SES prestroke was retrieved from Statistics Sweden and defined by a composite measure based on education and income tertiles. Associations between SES and Patient Reported Outcome Measures were analyzed using logistic regression adjusting for confounders (sex and age) and additionally for potential mediators (stroke type, severity, cardiovascular disease risk factors, and living alone). Subgroup analyses were performed for stroke type, men and women, and younger and older patients. The study included 44 511 patients. Of these, 31.1% required assistance with mobility, 18% with toileting, and 22.2% with dressing 3 months after stroke. For poststroke symptoms, 12.3% reported low mood, 39.1% fatigue, and 22.7% pain often/constantly, while 21.4% rated their general health as poor/very poor. Adjusted for confounders, the odds of needing assistance with activities of daily living were highest for patients with low income and primary school education, for example, for mobility, odds ratio was 2.06 (95% CI, 1.89-2.24) compared with patients with high income and university education. For poststroke symptoms, odds of poor outcome were highest for patients with low income and university education (eg, odds ratio, 1.79 [95% CI, 1.49-2.15] for low mood). Adjustments for potential mediators attenuated but did not remove associations. The associations were similar in ischemic and hemorrhagic strokes and more pronounced in men and patients <65 years old. There are substantial SES-related differences in Patient Reported Outcome Measures poststroke. The more severe outcome associated with low SES is more pronounced in men and in patients of working age.

Health utility of patients with established rheumatoid arthritis and its influencing factors: a multi-center study in China

To assess the health utility value (HUV) of Rheumatoid Arthritis (RA) patients and its influencing factors in China. A cross-sectional survey was conducted in 8 tertiary hospitals across four capital-cities. The demographic characteristics, patient-reported outcomes including the HUV got by EQ-5D-5L, clinical characteristics, and clinician-reported outcomes of 171 RA patients were collected both from themselves and their physicians. Both the univariate and multivariate analyses were used to assess the potential factors of EQ-5D-5L HUV of the patients. The mean age of the patients was 50.7 years, with female being 64.9% (n = 111). The mean HUV and EQ visual analogue scale score of all patients were 0.586 and 47.3, respectively. The univariate analysis showed that the patients who were female, older, living in rural areas, with lower education level, advanced disease stage, higher the patient's assessment of arthritis pain visual analogue scale (PtAAP-VAS), the patient's global assessment of disease activity visual analogue scale (PtGADA-VAS), and the Physician’s global assessment of disease activity visual analogue scale (PhGADA-VAS) scores had significantly lower EQ-5D-5L HUVs. The multivariate analysis further suggested that older age, female, higher body mass index and higher PtGADA-VAS score were statistically significantly related to lower HUVs. The study provided the HUVs for RA patients with different characteristics and outcomes, which could be used in the economic evaluation of interventions for the RA patients. The identified factors could also assist the health care managing and improving the health-related quality of life on RA patients.

Validation of the Manchester Cosmetic Clinician-Reported Outcomes Questionnaire (MCCRO-Q)

SummaryPatient-reported outcome measures (PROM) are collected pre- and post-operatively following cosmetic surgery. Clinicians assess their own outcomes following surgery, but without a validated clinician-reported outcome measure (CROM), there is no way to compare clinical results between clinicians or compare these to PROM. Assessment of clinical outcomes is important to address patients' expectations in the consenting process and to provide recommendations to patients pre-operatively to improve post-operative appearance.To address the lack of validated CROM for cosmetic surgery, the Manchester Cosmetic Clinical Reported Outcome Questionnaire (MCCRO-Q) was developed. This study assessed the inter-rater reliability of assessors using the four-point scale pre-operative and post-operative MCCRO-Q questionnaires. Fifteen assessors reviewed photos from pre- and post-operative assessments in the UK between July 2016 and February 2020. Ninety-five patients were included in this validation study, with each patient assessed by a minimum of three assessors.MCCRO-Q showed consistency between reviewers, with all intra-class coefficient averages >0.5 for pre-operative assessment and post-operative assessment. Twenty-six per cent of preoperative assessments demonstrated perfect agreement, 56% had a maximum one-point difference, and 18% had a two-point difference to the median score. One per cent of post-operative assessments post-operatively demonstrated perfect agreement, 36% had a one-point difference and 63% had a maximum point difference of two to the median score.The inter-rate agreement shows MCCRO-Q is a reliable tool when used with three or more assessors to judge patient appearance clinically, pre-operatively and post-operatively, concerning abdominoplasty.

Patient-reported burden in adults with atopic dermatitis: an international qualitative study

The objective was to study a large, international, ethnically diverse population of patients with atopic dermatitis (AD) to support the creation of patient-centric recommendations for AD management. Qualitative data were generated from 45-min, 1:1 telephone interviews conducted across 15 countries in each patient’s native language. Interviews explored the impact of AD on patients’ lives, patients’ most important symptoms, treatment expectations, and treatment decision-making. Participants were also questioned on their current knowledge of AD scoring systems and what was most important to include in these tools. In total, 88 adult patients (≥ 18 years old) receiving treatment for AD were recruited through a market research database, clinician referrals, and local advertising. All patients were screened to ensure a balanced and diverse sample in terms of age, gender, educational level, employment status, geographic location, and AD severity. Patients involved in market research or activities supporting advocacy groups within the previous 6 months or affiliated with or employed by pharmaceutical companies were excluded. AD had a substantial impact on patients’ lives. Itch, skin redness, and dry/flaky skin were the most frequently reported symptoms, with > 75% of patients experiencing these symptoms every 1–3 days. Mental health issues were common and resulted in the greatest negative impact on patients’ daily lives. Patients perceived clinicians to underestimate the burden of their AD. Patients had little awareness of AD scoring systems and indicated a preference for these to be more clearly incorporated in clinical practice. For an ideal scoring system, patients favored using a combination of patient-reported and clinician-reported outcomes to reflect disease burden and ensure consistency across all settings. This global study generated diverse patient perspectives on the disease burden of AD, their expectations of treatment, and their views on AD scoring methods. These data provide evidence to support the development of patient-centric recommendations for AD management.

Concordance between clinician-reported and patient-reported outcomes of eyebrow and eyelash hair loss in patients with severe alopecia areata: Results from BRAVE-AA1 and BRAVE-AA2 studies

Concordance between clinician-reported and patient-reported outcomes of eyebrow and eyelash hair loss in patients with severe alopecia areata: Results from BRAVE-AA1 and BRAVE-AA2 studies

Trends and landscape of clinical outcome assessment use to support oncology product approvals.

e23186 Background: The regulatory landscape in oncology trials has evolved with growing emphasis on patient-focused drug development. Further to tumor and survival outcomes, measuring how patients feel and function via clinical outcome assessments (COAs) provides critical information on symptoms and treatment burden and may help to inform risk/benefit evaluation for new treatments. This study aimed to examine trends in the use of COAs to support efficacy or safety in oncology product labeling. Methods: Product labeling information was obtained from PROLABELS (Mapi Research Trust), a database containing COA-related data sourced from the efficacy or safety sections of FDA Product labels and EMA SmPC where at least one COA is mentioned. Oncology product labels were screened based on therapeutic indications and study information. The clinical study details published in the label/SPC between 1990 and 2022 were used for further analysis. Results: A total of 180 EMA and FDA approved drugs and devices for oncology indications where COAs were mentioned in the product label were identified. 288 studies with COAs supported these drug approvals. These studies were in 52 different indications, with most in multiple myeloma (31), followed by non-small-cell lung cancer (24), prostate cancer (22), and breast cancer (22). Of the COAs that were used, 47% were patient-reported outcomes, 18% were clinician-reported outcomes, 30% were composites, and 5% were unspecified. The number of studies conducted with COAs supporting a product label increased over time, with 3x more studies in the past 10 years (189 from 2013-2022) compared to the previous 10 years (60 from 2003-2012). For studies listing specific COA names, 63 unique COAs were used. Excluding response criteria assessments, the most common COAs were the EORTC-QLQ-C30 (57 studies), EORTC-QLQ-LC13 (18 studies), and EQ-5D-3L (17 studies). The two indications with the largest variety of COAs used were prostate cancer (10 unique COAs) and non-small-cell lung cancer (9 unique COAs). Conclusions: In line with regulators highlighting the utility of COAs in evaluating clinical benefit, results show an increase in studies using COAs to support efficacy and safety in oncology product approvals. With COAs providing critical information on symptoms, side effect impact, and quality of life, including this information on labels may help patients and providers make more informed healthcare decisions, and for market differentiation.

CAD/CAM versus traditional complete dentures: A systematic review and meta-analysis of patient- and clinician-reported outcomes and costs.

Computer-aided design and manufacturing (CAD/CAM) have been increasingly used to enhance the patient and clinician experiences with removable complete dentures (CDs). Yet, evidence from systematic reviews is lacking to validate the clinical significance of these digital prostheses. The purpose of this systematic review was to compare CAD/CAM CDs with the traditional ones in terms of patient and clinician-reported outcomes, post-insertion adjustment visits and costs. An electronic search of four databases [Medline (Ovid), Embase, Scopus and Cochrane CENTRAL; last update: May 2022] was performed to retrieve clinical studies comparing CAD/CAM and traditional CDs. Two independent reviewers screened the articles, extracted data (methods and outcomes) and assessed risk of bias of the included studies. The following outcomes underwent meta-analysis (random-effects model): overall patient and clinician satisfaction, oral health-related quality of life (OHRQoL), number of post-insertion adjustment visits, as well as laboratory and total costs. This review included 11 studies. Meta-analysis revealed that CAD/CAM CDs are comparable to the traditional CDs in terms of overall patient satisfaction and OHRQoL. Clinician-reported data depended on the manufacturing technique: whereas milled CDs performed better than traditional CDs in terms of clinician satisfaction and number of adjustments, 3D printed and traditional CDs were similar. Fabrication of CAD/CAM CDs required significantly less laboratory and overall costs than the traditional CDs. There is some evidence showing that CAD/CAM CDs are at least comparable to traditional CDs. Further well-designed randomized clinical trials are needed to evaluate the performance of specific CAD/CAM approaches for manufacturing CDs, however.

The impact of social deprivation on rotator cuff repair outcomes

BackgroundRotator cuff tears are a common orthopedic injury and the role of social determinants of health (SDoH) in surgical outcomes remains underexplored. The goal of this study was to investigate the correlation between social deprivation, measured by the Area Deprivation Index (ADI), and outcomes following arthroscopic rotator cuff repair. MethodsWe conducted a retrospective chart review on patients undergoing primary arthroscopic rotator cuff repair at a level one academic center between 2006 and 2019. Patient demographics (age, gender, race), comorbidities, ADI scores, range of motion, visual analog pain scores, and patient-reported outcomes (SST, ASES, and QuickDASH) were collected. Patients were stratified into terciles based on their relative level of deprivation. Statistical analysis was performed using ANOVA, t-tests, chi-square tests, and univariate/multivariate logistic regression. Results322 patients were included in this study. The most deprived group had a higher prevalence of diabetes compared to the least and intermediately deprived group (p<0.001). Massive tear occurrence was greater in the least deprived group (p=0.003) compared to the most deprived group. There was no difference in objective outcomes between groups. Patient-reported outcomes (SST, ASES, and QuickDASH) were worse in the most deprived group compared to the least and intermediate deprived groups. ConclusionSocial deprivation significantly affects patient-reported outcomes in rotator cuff repair surgery. While clinician-reported outcomes were consistent, patients' perceptions varied based on social determinants. Integrating SDoH considerations in orthopedic care is a promising next step in securing equitable approaches. However, more research is needed to validate and expand these findings.

Patient- and Clinician-Reported Outcomes for Tirbanibulin in Actinic Keratosis in Clinical Practice Across the United States (PROAK).

The Patient-Reported Outcomes in Actinic Keratosis (PROAK) study evaluated patient- and clinician-reported outcomes (PRO; ClinRO) during 24 weeks of follow-up among adult patients with actinic keratosis (AK) on the face or scalp who were administered tirbanibulin 1% ointment in real-world community practices in the United States.&nbsp; Methods: Quality of life (QoL) was assessed by Skindex-16 at week (W) 8. Additionally, effectiveness (Investigator Global Assessment [IGA]), PRO and ClinRO (Treatment Satisfaction Questionnaire for Medication and Expert Panel Questionnaire), safety, and tolerability were assessed at W8 and W24. The safety population included 300 patients; the full analysis set included 290 patients (278 patients at W24). At W8, a statistically significant difference (P&lt;0.03) was observed for Skindex-16 domains in all assessed subgroups. Clinicians and patients reported high global satisfaction (mean [SD] scores of 74.9 [23.9] and 72.0 [24.6], respectively) at W24. Overall skin appearance improved from baseline to W24 (83.6% clinicians; 78.5% patients). IGA success (IGA score of 0-1) was achieved by 71.9% of patients at W24 with a similar % at W8 (73.8%) suggesting a stable effectiveness over time. About 5% of patients reported at least one adverse event, 4% reported at least one serious adverse event and no patients reported serious adverse drug reactions. At W8, the most frequently reported local skin reactions were mild/moderate erythema (47.6%) and flaking/scaling (49.6%). Treatment with tirbanibulin demonstrated effectiveness in the management of AK lesions and a favorable safety and tolerability profile. Furthermore, QoL was improved as early as W8, and both patients and clinicians reported high levels of treatment satisfaction, independently of patients' characteristics. J Drugs Dermatol. 2024;23(5):338-346. doi:10.36849/JDD.8264.

Impact of residual skin lesions and previous biologic treatment failure on patient-reported outcomes in patients with psoriasis receiving biologic treatment.

Recent advances in biologic treatments have made clear skin a realistic treatment goal for psoriasis. However, clear skin may not uniformly translate to an absence of impact on patients' quality of life. This retrospective observational study aimed to elucidate the factors influencing patient-reported outcomes in patients with psoriasis who have demonstrated successful clinician-reported outcomes on using biologics. A total of 96 patients who have achieved a ≥75% improvement in Psoriasis Area and Severity Index (PASI) scores with ≥6 months of biologic treatment were included. Their median PASI score was 0.4, with 37.5% having achieved PASI 100 (clear skin). Furthermore, 47.9% reported no impact of psoriasis on their quality of life (Dermatology Life Quality Index [DLQI] score 0 or 1), while 52.1% reported a negative impact (DLQI score ≥2). Notably, 28.1% of the participants had a history of biologic treatment failure, defined as the inability to achieve or sustain a 75% PASI improvement with the previously used biologic agent. Multivariable logistic regression analysis revealed a positive association between achieving PASI 100 and reporting no impact of psoriasis on quality of life (adjusted odds ratio [aOR] 3.88, 95% confidence interval [CI] 1.49-10.91, P = 0.007). Conversely, prior biologic treatment failure was negatively associated with reporting no impact of psoriasis on quality of life (aOR 0.13, 95% CI 0.02-0.65, P = 0.023). Furthermore, among patients with clear skin, those with experience of previous biologic treatment failure reported significantly lower quality of life than those without such experience (P = 0.033). In conclusion, minimal residual skin lesions and prior biologic treatment failure were associated with poorer patient-reported outcomes in patients with psoriasis. Opting for a biologic agent with the highest predicted efficacy, rather than pursuing a "step-up" approach with a higher possibility of treatment failure, may be a more suitable strategy in the biologic treatment of psoriasis.

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.