Real-world Outcomes Research Articles

Real-world ePRO use and clinical outcomes using electronic patient-reported symptom monitoring for patients with advanced non-small-cell lung cancer receiving first-line pembrolizumab.

Aim: This ambispective observational study assessed the impact of Noona, an electronic patient-reported outcomes (ePRO) platform, for patients with non-small cell lung cancer (NSCLC) treated in a community oncology setting. Methods: Adults with advanced NSCLC, ECOG performance status of 0-2, who received first-line (1L) pembrolizumab (monotherapy or with chemotherapy) were eligible. Those initiating pembrolizumab from 1 July 2017 to 30 June 2019, identified retrospectively (historical cohort), were compared with those initiating pembrolizumab from 1 October 2019 to 30 September 2021 who were prospectively offered Noona (standard of care [SoC] cohort). The Kaplan-Meier method and Cox proportional hazards models were used to compare pembrolizumab real-world time on treatment (rwToT; primary outcome measure) and rw time to next treatment or death (rwTTNTD) between historical and SoC cohorts. Healthcare resource use (HCRU) was compared using generalized linear models with Poisson distribution. Analyses were repeated to compare outcomes in the SoC cohort between Noona users (created a profile and used any function ≥one-time during 1L therapy) and nonusers with >42days on 1L pembrolizumab. Data cutoff was 30 June 2020 and 30 September 2022 for historical and SoC cohorts, respectively. Results: Median pembrolizumab rwToT was 4.4months (95% CI: 3.9-5.1) in the historical cohort (n=448) versus 4.1months (95% CI: 3.3-4.8) in the SoC cohort (n=462; adjusted hazard ratio [aHR], 0.9; 95% CI: 0.8-1.0; p=0.14 vs historical cohort). In the SoC cohort, 147 of 341 eligible patients (43%) established a Noona profile; 122/341 (36%) were Noona users. Median rwToT was 6.4months (95% CI: 5.1-7.4) and 6.9months (95% CI: 5.6-7.6) among Noona users and Noona nonusers (n=219), respectively (aHR, 1.1; 95% CI: 0.8-1.4; p=0.95 vs Noona users). The rwTTNTD and HCRU were comparable in historical versus SoC cohorts and for Noona users versus nonusers. During the first year after establishing a Noona profile, 92 of 147 patients (63%) used the platform; monthly use was 32-42%, and checking laboratory results was the most used function overall (by 52% of the 147). Conclusion: Notwithstanding the null findings of this study, positive results of ePRO use in clinical trials and observational studies support the treatment-related symptom monitoring and survival benefits of ePRO utilization.

Real-world outcomes of first-line maintenance niraparib monotherapy in patients with epithelial ovarian cancer.

To assess real-world progression-free survival (rwPFS) and time to next treatment (rwTTNT) among patients with epithelial ovarian cancer (EOC) who received first-line maintenance (1LM) niraparib monotherapy. In this US-nationwide, electronic health record-derived, deidentified database study, eligible patients with EOC initiated 1LM niraparib monotherapy (1 January 2017-1 December 2022) following first-line platinum-based chemotherapy. Median rwPFS and rwTTNT were estimated with Kaplan-Meier methodology overall and in a homologous recombination-deficient (HRd) subgroup (further stratified as BRCA wild-type [BRCAwt] or BRCA-mutated [BRCAm]). Observed median rwPFS was 11.4 (95% CI, 10.1-12.7) months overall (N = 560), 18.2 (95% CI, 13.9-24.2) months for the HRd subgroup (n = 144), and 25.4 (95% CI, 15.9-not reached) and 14.2 (95% CI, 8.6-18.6) months for HRd patients with BRCAm and BRCAwt tumors, respectively. Observed median rwTTNT was 12.4 (95% CI, 11.5-13.8) months overall, 19.6 (95% CI, 14.9-23.9) months for the HRd subgroup, and 24.9 (95% CI, 16.0-not reached) and 15.1 (95% CI, 10.3-19.8) months for HRd patients with BRCAm and BRCAwt tumors, respectively. The real-world observed median rwPFS and rwTTNT were longer for patients with EOC who received 1LM niraparib monotherapy in the HRd subgroup (specifically for the BRCAm subgroup).

Comparison of Outcomes of Deep Sclerectomy, Canaloplasty, and Viscocanaloplasty: A Multi-Centred Study.

Deep sclerectomy (DS) and canaloplasty provide better intraocular pressure (IOP) control than viscocanalostomy. DS required less glaucoma medications but more interventions to reach target IOP. To compare real-world outcomes of three non-penetrating glaucoma surgery (NPGS) techniques. Retrospective, cohort study of consecutive patients undergoing canaloplasty (CP), deep sclerectomy (DS), and viscocanalostomy (VC), across nine European glaucoma units. Four intraocular pressure (IOP) criteria were used to define success at 2-year follow-up: (A)IOP≤21mmHg and ≥20% reduction; (B)IOP≤18mmHg and ≥20% reduction; (C)IOP≤15mmHg and ≥25% reduction; (D)IOP≤12mmHg and ≥30% reduction. Secondary outcomes included IOP control, BCVA, number of medications over time, risk factors for failure, complications, and post-operative interventions. Success was distinguished as qualified or complete, if reached with or without antiglaucoma medications, respectively. 600 eyes (545 patients) undergoing standalone CP (201 eyes), DS (200 eyes), and VC (199 eyes) were included. Qualified success rates of CP, DS, and VP at 24 months were, respectively: (Criterion A) 85.1%, 67.6% and 64.6%; (Criterion B) 85.1%, 66.1% and 58.6%; (Criterion C) 76.6%, 55.5% and 39.0%; (Criterion D) 27.7%, 28.5% and 22.1%. Success rates were significantly different across the three techniques (P=0.04 or below), except for complete success according to criterion A (P=0.07). Mean IOP(±SD) reduced from 25.2(±6.9), 20.5(±6.7), and 22.7(±7.2)mmHg pre-operatively to 13.1(±3.1), 12.9(±4.5), and 14.7(±4.6)mmHg at post-operative year 2 in the CP, DS, and VC groups respectively (P<0.001 between pre-operative and post-operative time points for all groups). All three NPGS provide sustained IOP reduction, but DS and CP provide better success rates and IOP control. Success rates were low for the most stringent cut-offs, suggesting that other techniques such as trabeculectomy may be indicated when a very low target IOP is demanded.

Real-world study of patients with HR+/ HER2- metastatic breast cancer treated with palbociclib and fulvestrant.

To investigate real-world treatment patterns and outcomes among patients with hormone receptor-positive/human epidermal growth factor 2-negative (HR+/HER2-) metastatic breast cancer (mBC) who initiated first-line palbociclib-fulvestrant. Retrospective observational study of iKnowMed electronic health records among patients who initiated first-line palbociclib-fulvestrant between 1 February 2016 and 31 December 2019 and were followed through 30 June 2020. Demographic, clinical, and treatment characteristics were evaluated descriptively. Endpoints including real-world progression-free survival, overall survival, time to chemotherapy, real-world duration of therapy, and time to next treatment were assessed using Kaplan-Meier methods from first-line treatment initiation. 317 patients were included (median age 67.3 years, 90.5% post-menopausal, 36.9% bone-only disease, 15.9 months median follow-up). Among those with prior adjuvant treatment (n = 269), 66.2% (n = 178) had disease-free intervals less than 12 months. Median real-world progression-free survival was 19.6 months (95% CI 15.2-23.6). These results suggest favorable real-world clinical outcomes associated with first-line palbociclib-fulvestrant among patients with HR+/HER2- mBC. (clinical trial registration: NCT04498481).

Real-world comparative outcomes and toxicities after definitive radiotherapy using proton beam therapy versus intensity-modulated radiation therapy for prostate cancer: a retrospective, single-institutional analysis.

This retrospective study aimed to compare the clinical outcomes of intensity-modulated radiation therapy (IMRT) and proton beam therapy (PBT). A total of 606 patients diagnosed with prostate cancer between January 2008 and December 2018 were included. Of these patients, 510 received PBT up to a dose of 70-78Gy (relative biological effectiveness) and 96 patients received IMRT up to a dose of 70-78Gy. The median follow-up period was 82months (range: 32-140months). Patients in the PBT group had significantly higher 7-year rates of biochemical relapse-free survival (bRFS) and disease-free survival (DFS) rates: 95.1% for PBT vs 89.9% for IMRT (P = 0.0271) and 93.1% for PBT vs 85.0% for IMRT (P = 0.0019). After matching analysis, 94 patients were assigned to both groups, and the PBT group showed significantly higher 7-year bRFS and DFS rates: 98.9% for PBT vs 89.7% for IMRT (P = 0.023) and 93.4% for PBT vs 84.6% for IMRT (P = 0.022), respectively. In the subgroup analysis of intermediate-risk patients, the PBT group showed a significantly higher 7-year bRFS rate (98.3% for PBT vs 90.5% for IMRT; P = 0.007). The V60 of the bladder in the PBT group (18.1% ± 10.1%) was higher than that in the IMRT group (14.4% ± 7.6%) (P = 0.024). This study found that the treatment outcomes of PBT potentially surpassed those of IMRT specifically concerning bRFS and DFS in real-world settings. However, it should be noted that attention is warranted for late bladder complication of PBT.

Making sense of body-worn cameras as a violence prevention tool for ambulance staff

Body-worn cameras (BWCs) were introduced for ambulance crews in England in 2021 in response to rising rates of abuse targeted towards ambulance crews by members of the public. As part of an evaluation of the effectiveness of BWCs as a tool for reducing occupational violence, we produced a non-systematic literature review to examine the real-world outcomes and cost-effectiveness of BWCs in a variety of settings and countries. Our review identified minimal literature on the topic pertaining to the ambulance sector. While there is considerable evidence relating to BWCs and violence prevention more generally, much of it is poor quality, anecdotal, and focused on specific professions with limited scope for making generalisations. There is some evidence that BWCs have the potential to address violence and abuse directed towards ambulance staff. However, our analysis of costs and benefits also raises questions about evidence concerning the value of BWCs and their potential negative impacts on staff wellbeing. The literature is unclear about the underlying mechanisms through which BWCs might support violence reduction, and it is likely that any benefits will be mediated by the complex and unpredictable environment in which BWCs are being used, which is likely to have strong independent effects on outcomes. The more that BWC research is able to explore such mechanisms in detail to inform our understanding of how and under what conditions specific outcomes are observed, the more we will be able to make sense of the variable findings produced in this area to date.

Methodological challenges using routine clinical care data for real-world evidence: a rapid review utilizing a systematic literature search and focus group discussion

BackgroundThe integration of real-world evidence (RWE) from real-world data (RWD) in clinical research is crucial for bridging the gap between clinical trial results and real-world outcomes. Analyzing routinely collected data to generate clinical evidence faces methodological concerns like confounding and bias, similar to prospectively documented observational studies. This study focuses on additional limitations frequently reported in the literature, providing an overview of the challenges and biases inherent to analyzing routine clinical care data, including health claims data (hereafter: routine data).MethodsWe conducted a literature search on routine data studies in four high-impact journals based on the Journal Citation Reports (JCR) category “Medicine, General & Internal” as of 2022 and three oncology journals, covering articles published from January 2018 to October 2023. Articles were screened and categorized into three scenarios based on their potential to provide meaningful RWE: (1) Burden of Disease, (2) Safety and Risk Group Analysis, and (3) Treatment Comparison. Limitations of this type of data cited in the discussion sections were extracted and classified according to different bias types: main bias categories in non-randomized studies (information bias, reporting bias, selection bias, confounding) and additional routine data-specific challenges (i.e., operationalization, coding, follow-up, missing data, validation, and data quality). These classifications were then ranked by relevance in a focus group meeting of methodological experts. The search was pre-specified and registered in PROSPERO (CRD42023477616).ResultsIn October 2023, 227 articles were identified, 69 were assessed for eligibility, and 39 were included in the review: 11 on the burden of disease, 17 on safety and risk group analysis, and 11 on treatment comparison. Besides typical biases in observational studies, we identified additional challenges specific to RWE frequently mentioned in the discussion sections. The focus group had varied opinions on the limitations of Safety and Risk Group Analysis and Treatment Comparison but agreed on the essential limitations for the Burden of Disease category.ConclusionThis review provides a comprehensive overview of potential limitations and biases in analyzing routine data reported in recent high-impact journals. We highlighted key challenges that have high potential to impact analysis results, emphasizing the need for thorough consideration and discussion for meaningful inferences.

Transarterial Radioembolisation with Y90 Resin Microspheres and the Effect of Reimbursement Criteria in France: Final Results of the CIRT-FR Prospective Observational Study.

This analysis of the CIRSE Registry for SIR-Spheres Therapy in France, CIRT-FR, reports on real-world outcomes of transarterial radioembolisation (TARE) with Y90 resin microspheres for hepatocellular carcinoma (HCC) and colorectal cancer liver metastases (CRLM) patients in France, focusing on safety, effectiveness and health-related quality of life (HRQoL). Results on patients treated based on national reimbursement criteria are discussed here. Prospective, multicentre, observational study of HCC and CRLM patients treated between August 2017 and July 2020 with TARE Y90 resin microspheres. Patients were assigned to different analysis groups based on reimbursement recommendations. Follow-up period was at least 24months with patient data collected every 3months. In total, 252 (193 HCC, 59 CRLM) patients of CIRT-FR were included in the analysis. No differences in effectiveness, safety and HRQoL were found between analysis groups based on reimbursement recommendations. Median overall survival for HCC and CRLM was 19.0 (95% CI, 16.1-22.4) and 10.8 (95% CI, 8.0-13.5) months, respectively. Serious procedure-related adverse events occurred in 13% of the patients. HRQoL generally remained stable, with some fluctuations in function scores and symptoms. In our cohorts, patients performed similarly regarding clinical outcomes irrespective of their analysis group based on reimbursement recommendations. Our results suggest that instead of restrictive reimbursement criteria, more decision-making power in selecting suitable patient groups could be given to multidisciplinary tumour boards. Results confirm that TARE with Y90 resin microspheres is an effective and safe treatment for liver cancer, with maintained HRQoL in most patients.

Real world outcomes with Ibrutinib monotherapy in Chronic Lymphocytic Leukemia: a single center experience

Introduction. The advent of Bruton’s tyrosine kinase (BTK) inhibitors brought about a paradigm shift in the management of chronic lymphocytic leukemia (CLL), by offering a well-tolerated chemotherapy-free approach. Here, we share the experience with ibrutinib of a major Romanian regional cancer center. Methods. We screened patients treated for CLL in our center over 6 years (2017- 2022) and included those who were treated with ibrutinib either in the first line of therapy or in subsequent lines. Results. We enrolled 61 patients, 40 with treatment-naïve (TN) CLL and 21 with relapsed/refractory (R/R) CLL, with a median age at treatment initiation of 65 years. Concerning the prognostic-predictive workup, IgHV mutational status was available for 78.7% of the patients, TP53 sequencing for 82%, assessment of 17p deletion for 82%, and CD38 marker analysis was performed for 70.5%. With a median follow-up period of 55 months, the overall response rate (ORR) was 90.2%, with a median progression-free survival (PFS) of 33 months and a median overall survival (OS) that has not been reached. In our cohort, albeit non-significant statistically, patients with TP53 mutation had a shorter OS and those with mutated IgHV, a shorter PFS. Rai 3-4 and Binet C stages at diagnosis were associated with a shorter PFS, but not OS. In our cohort, the correlation between survival and high Cumulative Illness Rating Scale (CIRS) index was not statistically significant. Ibrutinib was generally well tolerated in our cohort, as only 14.8% of our patients discontinued treatment due to adverse effects. Conclusion. Our study suggests that ibrutinib is a valid therapeutic option for TN or R/R CLL patients, with a high ORR and a good safety profile.

Biosimilars in Focus: Evaluating Their Role Compared to Adalimumab in Clinical Practice

Introduction and Purpose: Adalimumab, a TNF-α inhibitor, is a cornerstone treatment for autoimmune and inflammatory diseases such as rheumatoid arthritis and inflammatory bowel disease. Despite its efficacy, the high cost of adalimumab (Humira®) limits accessibility. The emergence of biosimilars offers cost-effective alternatives with comparable efficacy, safety, and immunogenicity. This article reviews the role of adalimumab biosimilars, their clinical equivalence, and potential to address healthcare challenges. Materials and Methods: A comprehensive literature review was conducted to evaluate the structural, functional, and clinical parity of adalimumab biosimilars with the reference product. Key studies on pharmacokinetics, efficacy, safety, therapeutic drug monitoring (TDM), and real-world outcomes were analyzed. Results:Adalimumab biosimilars demonstrate equivalent pharmacokinetic profiles and clinical efficacy across conditions, with comparable remission rates and safety profiles. Innovations, such as citrate-free formulations and advanced delivery devices, enhance patient adherence. Biosimilars significantly reduce treatment costs, increasing accessibility, especially in resource-constrained settings. Challenges remain in patient acceptance and managing therapy transitions. Conclusion:Biosimilars are transformative in addressing the cost barrier of biologic therapies, maintaining therapeutic equivalence to adalimumab while expanding access globally. Enhanced TDM and patient-centered strategies are essential for optimizing outcomes and maximizing their adoption in clinical practice.

Niraparib as First-Line Maintenance Therapy in Patients with Stage III Ovarian Cancer and No Visible Residual Disease: AR1ZE Real-World Study.

Niraparib was approved for first-line (1L) maintenance (1LM) treatment of patients with advanced epithelial ovarian cancer (EOC) following the PRIMA/ENGOT-OV26/GOG-3012 (PRIMA) trial. PRIMA was restricted to patients at higher risk of progression (excluded stage III EOC with no visible residual disease [NVRD] after primary cytoreductive surgery [PCS]). This retrospective study evaluated the potential impact of excluding stage III EOC with NVRD from PRIMA by assessing real-world treatment outcomes following 1LM niraparib monotherapy in this patient population. Data from a US-nationwide electronic health record-derived deidentified database comprised adult patients diagnosed with stage III/IV EOC who received 1L platinum-based chemotherapy and initiated niraparib 1LM monotherapy (01Jan2017-28Feb2023). Patients were classified as PRIMA-like (EOC with higher-risk prognostic factors for disease progression) or stage III disease with NVRD after PCS. Real-world time to next treatment (rwTTNT) and progression-free survival (rwPFS), assessed from the end date of 1L platinum-based chemotherapy, were measured via Kaplan-Meier methods. Among 453 patients who received niraparib 1LM (PRIMA-like cohort, n = 390; stage III NVRD cohort, n = 63), median follow-up from index was 14.9 (interquartile range [IQR], 7.3-25.1) and 18.4 (IQR, 9.3-29.1) months in the PRIMA-like and stage III NVRD cohorts, respectively. Median rwTTNT was significantly longer in the stage III NVRD cohort (22.5 [95% confidence interval (CI), 17.3-not reached] months) than in the PRIMA-like cohort (11.7 [95% CI, 10.8-12.9] months; P < 0.001). Median rwPFS in the stage III NVRD cohort (25.2 [95% CI, 12.6-not reached] months) was more than double that in the PRIMA-like cohort (10.1 [95% CI, 9.1-11.9] months; P < 0.001). In the stage III NVRD cohort, rwTTNT and rwPFS were significantly longer than in the PRIMA-like cohort, consistent with clinical expectation. Results suggest niraparib clinical benefit may have been underestimated in PRIMA because of the exclusion of patients with stage III EOC with NVRD after PCS.

Adjuvant Immunotherapy After Resected Melanoma: Survival Outcomes, Prognostic Factors and Patterns of Relapse.

Anti-PD-1-based immunotherapy has improved outcomes in stage IIB to IV resected melanoma patients in clinical trials. However, little is known about real-world outcomes, prognostic factors and patterns of relapse. This is a retrospective multicenter observational study including patients with resected melanoma treated with subsequent anti-PD-1-based adjuvant immunotherapy. Data on clinical and demographic characteristics, delivered treatment, prognostic factors, time and pattern of relapse were collected. We included 245 patients from eight centers; 4% of patients were at stage IIB-C, 80% at stage IIIA-D and 16% at stage IV. Recurrence-free survival (RFS) rates at 18 and 36 months were 60% and 48%, respectively, with a median RFS of 33.7 months. Prognostic factors associated with recurrence were melanoma primary site (HR 2.64, 95% CI 1.15-6.01) and starting adjuvant therapy more than 12 weeks after the last resection (HR 1.68, 95% CI 1.13-2.5); presence of serious immune-related adverse events was associated with better RFS (HR 0.4, 95% CI 0.19-0.87). Early relapses accounted for 63% of the total recurrences, with a higher number of metastatic sites (18%); in contrast, late relapses presented more frequently with brain metastases (20%). In our patients with resected melanoma who underwent anti-PD-1-based adjuvant immunotherapy, survival outcomes were worse than those reported in clinical trials. Primary melanoma site and time interval between the last resection and the start of adjuvant therapy were associated with survival.

Mobile Health for Obsessive-Compulsive Disorder: Patients' Preferences and Perception of Patient-Centeredness.

The increasingly fast development of mobile health technologies holds significant value for individuals dealing with mental health conditions. However, inadequate consideration of patients' preferences and expectations undermines real-world outcomes, including sustained adherence. Driven by the belief that specific characteristics, such as youth and higher education, of individuals with obsessive-compulsive disorder make them suitable for digital adoption, we investigated mHealth-related desirability factors within this patient group. Fifty-one conveniently selected adults with obsessive-compulsive disorder filled in a self-report questionnaire about symptom self-management preferences, with an emphasis on assessing mobile health options and perceptions of patient-centeredness. The smartphone phone app emerged as the top choice of most of the sample for receiving information about symptom status (82.4%), obtaining general information about obsessive-compulsive disorder (74.5%), and symptom self-registration (66.7%), with no significant effect of sex or living location. Although only 23.5% of participants were using a health-related app, most expressed interest in using it for receiving symptom management tips (98.1%), medical advice (94.2%), symptom evolution updates (90.2%), lifestyle information (92.2%), medication tracking (88.2%) and short symptom self-reports (90.2%). Median expectations regarding mobile health's impact on patient-centeredness, satisfaction, and adherence were positive or very positive. Our data confirm that individuals with obsessive-compulsive disorder exhibit strong inclinations and optimistic expectations toward technology-based solutions. We highlight some of the preferences within this patient group, which can inform the design of practical, real-world applications.

Early life racial/ethnic discrimination effects on behavioral control and health outcomes in young adults.

Early life trauma has been shown to facilitate habitual behavior, which may predispose individuals toward perpetuating maladaptive behaviors. However, previous investigations did not account for other traumatic childhood experiences like racial/ethnic discrimination exposure, nor have they examined the interaction of trauma and habits on real-world adverse outcomes. To examine these effects, we recruited 96 young adults (20.06 ± 1.89 years old) in a study probing early life racial/ethnic discrimination influences on habitual learning, and the conjunctive influences of early life discrimination and habit on disordered eating and substance use. To measure habit responses, participants completed a noise avoidance task during which they responded to abstract stimuli via associated keyboard presses to avoid an aversive screaming sound, after which they performed a devaluation test to measure avoidance habit responses. Participants then completed a series of questionnaires examining early life racial/ethnic discrimination exposure, disordered eating and substance use, and other psychological characteristics. Hierarchical regression results showed that certain early life discrimination subtypes, particularly threat/aggression experienced due to racial/ethnic background, significantly predicted habitual responding above and beyond the effects of psychological confounds. Additionally, overall early life discrimination exposure positively predicted binge eating, but no variables of interest predicted alcohol and drug use. These results expand on extant literature showing the negative impacts of childhood stressors on behavioral control and real-world outcomes.

Evaluation of Real-World Evidence to Assess Effectiveness Outcomes of Janus Kinase Inhibitors for Rheumatoid Arthritis: A Systematic Review of US Studies.

This review summarized the real-world effectiveness outcomes of Janus kinase inhibitors (JAKi) for rheumatoid arthritis (RA) based on observational studies. A systematic review followed PRISMA guidelines, with searches conducted in PubMed, Embase, and CINAHL from each database's inception to June 2, 2023. Studies were included if they evaluated real-world effectiveness outcomes of JAKi for US RA patients. Search terms included "RA", "JAKi", and "real-world". All citations were imported into COVIDENCE platform. Two reviewers independently performed title/abstract screening and full-text eligibility. For each article, study characteristics and effectiveness measures focusing on treatment pattern, clinical response, and patient-reported outcomes (PROs) of JAKi were extracted. Newcastle-Ottawa Scale (NOS) was utilized to assess the quality of the included articles. In total, 35 studies representing 252-30,556 patients were included. A majority used the administrative claims datasets (n=23, 65.71%), followed by 9 studies using electronic medical record (EMR) data and 3 studies using patient registry databases. Across claims-based studies, adherence, persistence, and effectiveness of JAKi were common outcomes. Adherence rates varied, with a proportion of days covered (PDC) ranging from 0.53 to 0.83 across 11 studies. Persistence of JAKi in RA patients was reported in 14 studies, where the median persistence time in treatment was reported to be between 121-516 days. Six studies applied effectiveness algorithms, with 14.8-26% of patients meeting effective treatment criteria. In addition, the most common measure of clinical response throughout the studies was Clinical Disease Activity Index (CDAI), with 10 articles reporting mean CDAI changes between -4.7 and 5.1. Across 12 studies that measured the PROs, the most prevalent PRO was pain, with the mean change in pain ranging from -9.3 to 8.9 across these studies. Real-world studies on JAKi for RA reflect a range of effectiveness measures, illustrating the expanding role of JAKi in clinical practice.

Real-world outcomes on myopia management efficacy of diverse segmented defocus optics (DSDO) and defocus incorporated multiple segments (DIMS) spectacle lenses in Chinese children: An initial 12-month prospective clinical study.

To investigate the 12-month effectiveness of Diverse Segmented Defocus Optics (DSDO) and Defocus Incorporated Multiple Segments (DIMS) spectacle lenses in a real-world clinical population in myopic and pre-myopic Chinese children. About 364 subjects prescribed DSDO or DIMS were enrolled. Axial length (AL) and cycloplegic spherical equivalent refraction (SER) changes over 12 months were measured. The subjects were further divided into age sub-group (6-9; 10-14) and SER sub-group (+0.75D≤SER<-0.50D; -0.50D≤SER<-2.00D; -2.00D≤SER<-4.00D; SER≤-4.0D). Contrast sensitivity and visual experience were also reported. The rate of myopia progression was compared with historical single-vision spectacles (SVS) lenses data to evaluate the effectiveness of the regime. 317 subjects were analyzed. At 12-month, AL changes in the DSDO and DIMS group were 0.16±0.16 mm and 0.21±0.22 mm, respectively (P = 0.0202). DSDO spectacle lenses had better control effect in +0.75D≤SER<-0.50D and SER≤-2.0D sub-groups. The proportion of participants had no greater than 0.20 mm AL elongation was 65.00% and 55.41% of in DSDO and DIMS group separately. Myopia control effect in DSDO group was 47%-69% and 33%-62% in DIMS group compared to historical SVS lenses. Both DSDO and DIMS spectacle lenses retarded AL elongation. DSDO showed more stable myopia control effect comparing to DIMS, especially in groups of SER≤-2.0D sub-groups and older patients. DSDO showed initial potential myopia prevention effect in pre-myopic children compared with historical SVS lenses data. However, the small sample and no control group in pre-myopes of this study are key limitations. Further research is needed to confirm and understand DSDO's role for pre-myopic children.

Real-World Treatment Outcomes of an Artificial Tear Containing Arabinogalactan, Hyaluronic Acid and Trehalose Among Subjects with Dry Eye.

To assess the efficacy, adherence, and tolerability of a new artificial tear based on arabinogalactan, hyaluronic acid, and trehalose in a population with dry eye disease (DED). A retrospective, real-world, post-marketing study identified 96 adult patients (aged 18-80 years) with signs and symptoms of dry eye. These patients received fixed combination therapy with eye drops containing arabinogalactan, hyaluronic acid, and trehalose at various dosing schedules. The data for this study were collected from April 2022 to June 2023. Patients underwent evaluation at baseline (T0) and after a follow-up period of two-three months (T1) using a patient-reported questionnaire. In 96 adult patients (71 women and 25 men) with dry eye due to various conditions, the results indicated a 98% positive response to therapy. This response included improvements in vision (13%), comfort (39%), redness (13%), itching (16%), photophobia (4%), and tearing (14%). Additionally, 61% of the patients experienced 1-2hours of comfort following instillation. This real-life post-marketing study demonstrated clinical improvement of signs and symptoms in patients with dry eye disease using a new artificial tear medical device based on arabinogalactan, hyaluronic acid, and trehalose.

Evaluation of Drug-Drug Interactions in Pharmacoepidemiologic Research.

Drug-drug interactions (DDIs) represent a significant concern for clinical care and public health, but the health consequences of many DDIs remain largely underexplored. This knowledge gap underscores the critical need for pharmacoepidemiologic research to evaluate real-world health outcomes of DDIs. In this review, we summarize the definitions commonly used in pharmacoepidemiologic DDI studies, discuss common sources of bias, and illustrate through examples how these biases can be mitigated.

Travoprost Intracameral Implant in Eyes with Glaucoma or Ocular Hypertension: Early Short-Term Real-World Outcomes

Travoprost Intracameral Implant in Eyes with Glaucoma or Ocular Hypertension: Early Short-Term Real-World Outcomes

Real-World Outcomes After Switch From Aflibercept to Faricimab in Eyes With Diabetic Macular Edema.

To assess the anatomic and functional outcomes in eyes with diabetic macular edema (DME) switched from intravitreal aflibercept to faricimab in a real-world setting. Retrospective, interventional consecutive case series. Patients with DME were switched from aflibercept to faricimab and categorized based on central subfield thickness (CST) 4 weeks after last aflibercept injection into responding DME (rDME, CST reduction >20% or CST ≤ 250 µm) and nonresponding DME (nrDME, CST unchanged or increased). Patients received a loading dose of two monthly faricimab injections followed by a treat-and-extend regimen. Differences in response between rDME and nrDME were analyzed based on injection interval, change in CST, and visual acuity (VA) 12 weeks postswitch. Fifty-two eyes of 40 patients met inclusion criteria (rDME: n = 26, nrDME: n = 26). Baseline and week 12: VA (logMAR) rDME 0.29 ± 0.23 and 0.22 ± 0.28, nrDME 0.42 ± 0.32 and 0.36 ± 0.29; CST (µm) rDME 370 ± 99 and 288 ± 80, nrDME 384 ± 85 and 380 ± 129. After 12 weeks, 54% rDME and 25% nrDME eyes showed a CST decrease of >20% or CST ≤ 250 µm. Forty-six percent rDME and 50% nrDME eyes had a ±20% CST change, 25% of nrDME eyes had a >20% CST increase, and 73% of rDME eyes and 47% of nrDME eyes reached an extended interval of 8 weeks or longer after 12 weeks. Most DME eyes previously responding or not responding to aflibercept experienced a reduction or stabilization of DME after 12 weeks of faricimab treatment. rDME showed a better anatomic response, and treatment intervals could be extended earlier and longer than nrDME.