Advances in Therapy最新文献

{"title":"Comparison of On-Label Treatment Persistence in Real-World Patients with Psoriatic Arthritis Receiving Guselkumab Versus Subcutaneous Interleukin-17A Inhibitors","authors":"Philip J. Mease,&nbsp;Shannon A. Ferrante,&nbsp;Natalie J. Shiff,&nbsp;Timothy P. Fitzgerald,&nbsp;Soumya D. Chakravarty,&nbsp;Jessica A. Walsh","doi":"10.1007/s12325-024-03042-1","DOIUrl":"10.1007/s12325-024-03042-1","url":null,"abstract":"<div><h3>Introduction</h3><p>Psoriatic arthritis (PsA) is a chronic, multidomain, inflammatory disease requiring long-term treatment. Guselkumab, a fully human interleukin [IL]-23p19-subunit inhibitor, and the IL-17A inhibitors (IL-17Ai) ixekizumab and secukinumab are approved by the US Food and Drug Administration (FDA) for adults with active PsA. Real-world data evaluating on-label treatment persistence is an important consideration for patients.</p><h3>Methods</h3><p>This retrospective claim-based analysis (IQVIA PharMetrics® Plus) included adults with PsA receiving guselkumab or their first subcutaneous (SC) IL-17Ai (ixekizumab/secukinumab) per FDA label (“on-label”) between July 14, 2020, and June 30, 2022. Baseline demographic and disease characteristics were collected in the 12 months preceding the index date (date of first guselkumab or SC IL-17Ai claim); follow-up extended through the earlier of the end of continuous insurance eligibility or end of data availability. Baseline characteristics were balanced between the cohorts by propensity score weighting (standardized mortality ratio [SMR]). Discontinuation was defined as a gap 2 × the FDA-approved maintenance dosing interval (guselkumab:112 days; SC IL-17Ai: 56 days); on-label persistence in the weighted cohorts was assessed using Kaplan-Meier curves and compared with a Cox proportional hazards model.</p><h3>Results</h3><p>Weighted demographic and disease characteristics were well balanced between the cohorts (guselkumab: <i>N</i> = 910, mean age = 50.4 years, 60.4% female; SC IL-17Ai: <i>N</i> = 2740, mean age = 50.2, 59.4% female). At 12 months, the guselkumab cohort was 1.85 × more likely to remain persistent with on-label therapy vs the SC IL-17Ai cohort (<i>p</i> &lt; 0.001); median time to discontinuation was not reached for guselkumab and was 12.3 months for SC IL-17Ai. At 3, 6, 9, and 12 months, persistence rates in the weighted cohorts were higher with guselkumab than with SC IL-17Ai (<i>p</i> &lt; 0.001).</p><h3>Conclusion</h3><p>In this real-world claims data analysis in adults with PsA, on-label persistence rates were statistically significantly higher with guselkumab, as early as 3 months, with ~ 2 × greater likelihood of persistence at 12 months relative to SC IL-17Ai.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"734 - 751"},"PeriodicalIF":3.4,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03042-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142764777","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Addressing the Human Experience of Chronic Kidney Disease: A Call to Transform Kidney Care","authors":"Ricardo Correa-Rotter,&nbsp;Steven J. Chadban,&nbsp;Laura Christen,&nbsp;Kelli Collins Damron,&nbsp;Lweendo Hamusankwa,&nbsp;Sarah Jarvis,&nbsp;Surendra Pentakota,&nbsp;Marisol Robles,&nbsp;Petrina Stevens,&nbsp;Christoph Wanner","doi":"10.1007/s12325-024-03048-9","DOIUrl":"10.1007/s12325-024-03048-9","url":null,"abstract":"<div><p>Chronic kidney disease (CKD), a long-term condition in which kidney function declines over time, is a growing global healthcare concern. CKD can have a major impact on the quality of life of patients and their caregivers. Recent research by the International Society of Nephrology highlights that current treatment strategies and policies do not fully address patients’ needs. This commentary provides patient insights into the real-life concerns of those who are living with CKD, with main concerns focusing on relationships and support, work and finances, and awareness, prevention, and intervention. Strong support networks are essential for patients and caregivers, but the burden of CKD can make it difficult to maintain personal connections. Limiting disease progression and providing mental health support can help patients and caregivers to maintain their relationships. Work or education can be challenging to manage with CKD; however, employers and educational institutions can create supportive environments that meet the diverse needs of people with CKD. Although delaying disease progression can preserve patient quality of life, people are often unaware of their disease prior to diagnosis, the severity of their CKD, and the risk factors for progression. This presents an opportunity to involve patients in their care by improving education about the benefits of maintaining kidney health. Early identification and holistic intervention could slow disease progression and protect the well-being of patients with CKD and their caregivers. This commentary brings together the diverse perspectives of patients and patient advocacy groups, as well as primary care and specialist healthcare professionals, to advocate for a transformation of CKD management that encourages patient self-care and that prioritizes timely intervention.</p><p>A patient perspective video and a graphical abstract are available with this article.</p>\u0000<div><div><div></div><script></script></div><div><p>Addressing the Human Experience of Chronic Kidney Disease: A Call to Transform Kidney Care. Watch Lweendo Hamusankwa discuss his experience of living with chronic kidney disease, from how it impacted his family life to the importance of support networks and patient-facing information. Lweendo advocates for patients to be educated on CKD progression, treatment options, and lifestyle interventions to encourage people to manage their own health and well-being.</p></div></div><h3>Graphical Abstract</h3><div><figure><div><div><picture><source><img></source></picture></div></div></figure></div></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"559 - 567"},"PeriodicalIF":3.4,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03048-9.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142765513","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Baseline Characteristics of the DISCOVER CKD Prospective Cohort.","authors":"Carol Pollock, Juan-Jesus Carrero, Eiichiro Kanda, Richard Ofori-Asenso, Hungta Chen, Juan Jose Garcia Sanchez, Surendra Pentakota, Roberto Pecoits-Filho, Steven Fishbane, Carolyn S P Lam, Naoki Kashihara, David C Wheeler","doi":"10.1007/s12325-024-03028-z","DOIUrl":"10.1007/s12325-024-03028-z","url":null,"abstract":"<p><strong>Introduction: </strong>Real-world data from patients with chronic kidney disease (CKD) are limited, particularly regarding clinical management, treatment patterns and health-related quality of life (HRQoL) in the context of new therapies and updated standard of care guidelines.</p><p><strong>Methods: </strong>DISCOVER CKD is an observational cohort study enrolling adult patients with CKD, defined by an International Classification of Diseases, 10th Revision code, or with two estimated glomerular filtration rate measures < 75 ml/min/1.73 m² recorded 91-730 days apart. We describe the demographics, baseline characteristics and patient-reported outcomes of patients enrolled in the prospective phase.</p><p><strong>Results: </strong>Of 1052 patients (mean age 62.5 years; 36.9% female) enrolled from the USA, UK, Spain, Italy, Sweden and Japan, 727 (69.1%) had stage 2-3b CKD and 325 (30.9%) had stage 4-5 CKD. Overall, 72.4%, 43.0% and 37.5% of patients had histories of hypertension, diabetes and hyperlipidaemia, respectively. In total, 58.7% and 14.0% were receiving renin-angiotensin-aldosterone system inhibitors (RAASi) and sodium-glucose co-transporter 2 inhibitors (SGLT2i), respectively. Compared with patients with stage 2-3b CKD, patients with stage 4-5 CKD reported numerically greater symptom burden across all 11 symptoms measured, numerically worse HRQoL across all eight categories measured using the 36-item Short Form (SF-36) questionnaire, and numerically greater impairment at work across all four categories measured using the Work Productivity and Activity Impairment chronic kidney disease (WPAI-CKD) questionnaire. Compared with patients with stage 2-3b CKD, a higher proportion of patients with stage 4-5 CKD had anaemia, hyperkalaemia and oedema (49.8% vs. 16.9%, 21.8% vs. 8.4% and 17.5% vs. 9.5%, respectively).</p><p><strong>Conclusions: </strong>These contemporary real-world data from six countries highlight the substantial symptom, medication and psychosocial burden associated with CKD, and continued gaps in treatment. Graphical abstract available for this article.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier, NCT04034992.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142749717","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Secondary Risk-Reducing Strategies for Contralateral Breast Cancer in BRCA1/2 Variant Carriers: A Systematic Review and Meta-analysis","authors":"Jing Yu,&nbsp;Shan Jiang,&nbsp;Taoran Liu,&nbsp;Yangyang Gao,&nbsp;Xinyang Ma,&nbsp;Ginenus Fekadu,&nbsp;Yunqiu Xia,&nbsp;Bonny Parkinson,&nbsp;Wai-kit Ming,&nbsp;Yuanyuan Gu","doi":"10.1007/s12325-024-03054-x","DOIUrl":"10.1007/s12325-024-03054-x","url":null,"abstract":"<div><h3>Introduction</h3><p>Breast cancer poses significant challenges, especially the increased risk of contralateral breast cancer (CBC) in <i>BRCA1/2</i> variant carriers. This study systematically reviews and analyzes the effectiveness of secondary risk-reducing strategies for CBC in <i>BRCA1/2</i> carriers.</p><h3>Methods</h3><p>A systematic review and meta-analysis were conducted from January 2000 to December 2023, including RCTs, cohort, or case–control studies involving <i>BRCA</i> carriers with unilateral breast cancer. Random-effects models were used for odds ratios (ORs) on CBC incidence and hazard ratios (HRs) for overall survival (OS), with bias assessed via the Newcastle–Ottawa Scale.</p><h3>Results</h3><p>A total of 23,840 participants from 26 studies were included. Secondary risk-reducing interventions reduced CBC incidence by 38% [OR 0.62, 95% confidence interval (CI) 0.57–0.68] and improved OS by 45% (HR 0.55, 95% CI 0.46–0.67). Subgroup analyses showed differences by <i>BRCA</i> type, menopausal status, and treatment duration. For <i>BRCA</i>1 carriers, chemotherapy was most effective, while, for <i>BRCA</i>2, it was endocrine therapy. Postmenopausal interventions reduced CBC by 47% (OR 0.53, 95% CI 0.40–0.71), while premenopausal carriers saw a 34% reduction (OR 0.66, 95% CI 0.53–0.82). Tamoxifen’s effect diminished over time.</p><h3>Conclusion</h3><p>Secondary prophylaxis reduces CBC and improves OS in <i>BRCA1/2</i> carriers, with variations by genetic and physiological factors. These findings underscore the need for personalized strategies, considering menopausal status and treatment duration.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 1","pages":"106 - 131"},"PeriodicalIF":3.4,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142749719","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient Satisfaction Scale Following a Laxative for Antibiotic Washout Prior to Oral Microbiome Therapy","authors":"Hubert C. Chua,&nbsp;Sissi Pham,&nbsp;David A. Lombardi,&nbsp;Edina Hot,&nbsp;Lorie Mody","doi":"10.1007/s12325-024-03065-8","DOIUrl":"10.1007/s12325-024-03065-8","url":null,"abstract":"<div><h3>Introduction</h3><p>Administration of fecal microbiota spores, live-brpk [Vowst Oral Spores (VOS)], an oral microbiome therapeutic approved for prevention of recurrent <i>Clostridioides difficile</i> infection in adults, requires antibiotic washout using a laxative prior to administration. Patient acceptability of the prerequisite laxative is important. This study assessed psychometric properties of the Antibiotic Washout Patient Satisfaction Scale (AWPSS) which was minimally modified from a previously validated patient satisfaction scale for bowel preparation prior to colonoscopy.</p><h3>Methods</h3><p>Patients from the ECOSPOR IV trial who received a laxative preparation prior to oral administration of VOS and were administered the AWPSS were included. Reliability and construct validity of the AWPSS were evaluated.</p><h3>Results</h3><p>AWPSS data were available for 110 patients; all completed all 6 items of the AWPSS, supporting its acceptability. Domain 1 mean/median transformed total scores of 105.9/100 [range (best–worst), 0–300] suggested that patients were satisfied with the laxative preparation; a Cronbach’s alpha of 0.81 showed acceptable reliability. Almost all patients (97.3%) reported they were able to consume the entire laxative solution as instructed and would take it again if needed (95.5%). Higher satisfaction with the laxative preparation predicted higher acceptability of future use if needed (lower score) with mean/median of 101.7/100 and 195.0/200.00 for those who were willing or not willing to accept, respectively (<i>P</i> = 0.008).</p><h3>Conclusions</h3><p>AWPSS is a valid and reliable 6-item patient-reported outcome measure for use in patients requiring a laxative prior to oral microbiome therapy. AWPSS showed antibiotic washout was well tolerated and predicted that patients would be willing to consume the laxative in the future if needed.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 1","pages":"490 - 499"},"PeriodicalIF":3.4,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782395/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142738090","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Our Experience in Treating Infantile Hemangioma: Prognostic Factors for Relapse After Propranolol Discontinuation","authors":"Hiba Zaaroura,&nbsp;Afik Tibi,&nbsp;Emily Avitan-Hersh,&nbsp;Ziad Khamaysi","doi":"10.1007/s12325-024-03017-2","DOIUrl":"10.1007/s12325-024-03017-2","url":null,"abstract":"<div><h3>Introduction</h3><p>Infantile hemangioma (IH) is a common benign tumor in infants. While most cases exhibit a self-limiting nature, some require medical treatment to avoid complications. Propranolol is the first-line therapy for IH, it has a high success rate, and is safe to use. Unfortunately, some patients might experience rebound growth after propranolol discontinuation. Currently, it is unclear which factors predict this phenomenon. This study aimed to identify factors affecting the rebound growth of IH after propranolol cessation. We also aimed to identify predictors for an excellent response to oral propranolol.\u0000</p><h3>Methods</h3><p>We performed a retrospective cohort study using clinical data from all patients referred to our clinic with IH and placed on systemic oral propranolol between January 2009 to December 2023 in the dermatology outpatient clinic of Rambam Healthcare Campus.</p><h3>Results</h3><p>Out of a total of 552 patients with IH, 301 received oral propranolol for at least 6 months. A relapse phenomenon was observed in 38 (12.6%) patients. We found a significant association between limb involvement and the least likelihood of hemangioma rebound (<i>p</i> &lt; 0.001). An excellent response to oral propranolol was observed in 57.8% of patients. Younger age at initiation of oral propranolol was associated with an excellent response (<i>p</i> = 0.015). Also, IHs located on the limbs (67) showed a higher rate of excellent response to oral propranolol compared to other anatomical sites (<i>p</i> = 0.02). Interestingly, patients who were treated with a dose of 2 mg/kg/day were associated with excellent response to treatment (<i>p</i> = 0.007).</p><h3>Conclusions</h3><p>IHs located on the limbs demonstrated less rebound growth and a better treatment response. When oral propranolol was initiated earlier or when the target dose was 2 mg/kg/day, there was a higher rate of excellent response to treatment.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 1","pages":"537 - 547"},"PeriodicalIF":3.4,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782296/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142714938","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hyporesponsiveness to Erythropoiesis-Stimulating Agents in Dialysis-Dependent Patients with Anaemia of Chronic Kidney Disease: A Retrospective Observational Study","authors":"Christopher Atzinger,&nbsp;Hans-Jürgen Arens,&nbsp;Luca Neri,&nbsp;Otto Arkossy,&nbsp;Mario Garbelli,&nbsp;Alina Jiletcovici,&nbsp;Robert Snijder,&nbsp;Kirsten Leyland,&nbsp;Najib Khalife,&nbsp;Mahmood Ali,&nbsp;Astrid Feuersenger","doi":"10.1007/s12325-024-03015-4","DOIUrl":"10.1007/s12325-024-03015-4","url":null,"abstract":"<div><h3>Introduction</h3><p>Hyporesponsiveness to erythropoiesis-stimulating agents (ESAs) in patients with anaemia of chronic kidney disease may lead to increased ESA doses to achieve target haemoglobin levels; however, elevated doses may be associated with increased mortality. Furthermore, patients with hyporesponsiveness to ESAs have poorer clinical outcomes than those who respond well to ESAs. Incidence and clinical characteristics of patients with ESA hyporesponsiveness were explored in a real-world setting.</p><h3>Methods</h3><p>This was a retrospective study of electronic medical records of adults with stage 5 chronic kidney disease receiving renal replacement therapy and ESA treatment, from 1 January 2015 to 31 December 2021. The primary objective was ESA hyporesponsiveness rate/1000 days, with a hyporesponsive event defined as ESA use at an elevated dose, according to National Institute for Health and Care Excellence (NICE) criteria. Other hyporesponsiveness definitions applied were erythropoietin resistance index-defined ESA hyporesponsiveness (ERI) Kidney Disease Improving Global Outcomes (KDIGO) and a clinical practicality algorithm.</p><h3>Results</h3><p>In total, 85,259 patients were included in the analysis; 59.9% were male, median (interquartile range) ESA starting dose was 733.3 (400.0, 1200.0) IU/week and follow-up duration was 2.2 (1.0, 4.2) years. Incidence of ESA hyporesponsiveness varied when applying different definitions; NICE 0.05/1000 days (5.2% of patients), ERI 0.40/1000 days (40.7%), KDIGO 0.15/1000 days (15.4%), and clinical practicality algorithm 0.48/1000 days (47.9%). ESA doses remained higher in hyporesponsive versus responsive patients, yet haemoglobin levels were similar between groups.</p><h3>Conclusion</h3><p>The results from this study, which applied multiple hyporesponsiveness definitions to a large, geographically diverse population of patients with anaemia of CKD, showed variation in ESA hyporesponsiveness incidence rates depending on definitions used and higher ESA doses in hyporesponsive versus responsive patients. These results underscore the need for individualised clinical assessment and thorough evaluation when considering ESA dose adjustments to reach haemoglobin targets.</p><p>Graphical abstract available for this article.</p><h3>Trial Registration</h3><p>NCT05530291.</p><h3>Graphical Abstract</h3><div><figure><div><div><picture><source><img></source></picture></div></div></figure></div></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 1","pages":"471 - 489"},"PeriodicalIF":3.4,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782378/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142708953","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia A Without Inhibitors","authors":"María Teresa Álvarez Román,&nbsp;Nana Kragh,&nbsp;Patricia Guyot,&nbsp;Amanda Wilson,&nbsp;Piotr Wojciechowski,&nbsp;Wojciech Margas,&nbsp;Marlena Wdowiak,&nbsp;Elena Santagostino,&nbsp;Alix Arnaud","doi":"10.1007/s12325-024-03031-4","DOIUrl":"10.1007/s12325-024-03031-4","url":null,"abstract":"<div><h3>Introduction</h3><p>The phase 3 XTEND-1 trial (NCT04161495) demonstrated that efanesoctocog alfa prophylaxis provided superior bleed protection compared with pre-trial factor VIII (FVIII) prophylaxis in patients with severe haemophilia A. The aim of this study was to indirectly compare the efficacy of efanesoctocog alfa with non-factor replacement therapy emicizumab in adolescent and adult patients with severe haemophilia A without inhibitors.</p><h3>Methods</h3><p>A systematic literature review was conducted to identify phase 3 trials of emicizumab. Matching-adjusted indirect comparisons were used to compare annualised bleeding rates (ABRs) for any, treated, joint, and spontaneous bleeds, and joint health (measured using Hemophilia Joint Health Score [HJHS]), between efanesoctocog alfa and emicizumab. Estimated effects for different emicizumab regimens were pooled using random-effect meta-analysis to evaluate the overall difference in bleed outcomes between efanesoctocog alfa and emicizumab.</p><h3>Results</h3><p>One emicizumab trial was included (HAVEN 3), which investigated three dosing regimens. In meta-analyses, efanesoctocog alfa once-weekly (Q1W) was associated with significantly lower ABRs for any (incidence rate ratio [95% CI] 0.33 [0.20; 0.53]), any treated (0.49 [0.30; 0.80]) and treated joint (0.51 [0.28; 0.91]) bleeds compared with emicizumab Q1W in non-inhibitor patients with prior prophylaxis or on-demand treatment. Efanesoctocog alfa Q1W was also associated with a significantly better improvement from baseline in HJHS Joint Score (mean difference [95% CI] −2.06 [−3.97; −0.14]) and Total Score (−2.37 [−4.36; −0.39]) versus emicizumab Q1W or every 2 weeks.</p><h3>Conclusion</h3><p>Efanesoctocog alfa prophylaxis was associated with significantly lower rates of any, treated, and joint bleeds and improved joint health compared with emicizumab in patients with severe haemophilia A.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 1","pages":"442 - 455"},"PeriodicalIF":3.4,"publicationDate":"2024-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782328/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142685782","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Utility of Loneliness Status to Risk Stratification and Prediction of Recurrent Atrial Fibrillation After Catheter Ablation","authors":"Huaxin Sun,&nbsp;Wenchao Huang,&nbsp;Yan Luo,&nbsp;Shiqiang Xiong,&nbsp;Yan Tang,&nbsp;Guoshu Yang,&nbsp;Duan Luo,&nbsp;Xiaolin Zhou,&nbsp;Zhen Zhang,&nbsp;Hanxiong Liu","doi":"10.1007/s12325-024-03046-x","DOIUrl":"10.1007/s12325-024-03046-x","url":null,"abstract":"<div><h3>Introduction</h3><p>Socioeconomic determinants are increasingly important factors in the integrated management of atrial fibrillation (AF). The impact of loneliness status on AF recurrence remains unclear.</p><h3>Methods</h3><p>We conducted a cohort study based on patients with AF undergoing catheter ablation from 2017 to 2022. The Chinese version of the De Jong Gierveld scale (DJGLS) for evaluating loneliness degree was used. Multivariate Cox regression was performed to identify the independent risk factors for recurrent AF. A multivariate model was used to estimate the hazard ratio (HR) with 95% confidence interval (CI) when adjusting the known risk covariates in several kinds of subgroups.</p><h3>Results</h3><p>Nine hundred fifty-five patients with AF and mean age &gt; 65 years finished long-term follow-up. The AF cluster with severe/extreme loneliness tended to live alone according to DJGLS scores. Multivariate Cox regression showed that loneliness status is an independent risk factor for AF recurrence by using a multivariate model with adjustments of some covariates (moderate loneliness: HR 2.02; 95% CI 1.47–2.77, <i>P</i> &lt; 0.001; severe/extreme loneliness: HR 5.28; 95% CI 3.56—7.84, <i>P</i> &lt; 0.001). Survival analysis demonstrated that patients with AF with a more severe degree of loneliness are more likely to have AF recurrence in the long-term follow-up (log-rank test, <i>P</i> &lt; 0.001). Restricted cubic spline (RCS) showed a mainly non-linear relationship between feeling lonely and AF recurrence (<i>P</i> overall &lt; 0.001, <i>P</i> non-linear = 0.195). Receiver-operator characteristic curve (ROC) and time-dependent ROC curve indicated that the diagnostic value of loneliness status in predicting AF recurrence is stable and acceptable.</p><h3>Conclusion</h3><p>A more severe degree of loneliness was positively associated with increased risk of AF recurrence. Loneliness status showed an acceptable diagnostic value in discriminating AF recurrence as an independent tripartite variable.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 1","pages":"456 - 470"},"PeriodicalIF":3.4,"publicationDate":"2024-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142685786","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors","authors":"Robert Klamroth,&nbsp;Nana Kragh,&nbsp;Alix Arnaud,&nbsp;Patricia Guyot,&nbsp;Amanda Wilson,&nbsp;Piotr Wojciechowski,&nbsp;Marlena Wdowiak,&nbsp;Wojciech Margas,&nbsp;Linda Bystrická,&nbsp;Alberto Tosetto","doi":"10.1007/s12325-024-03032-3","DOIUrl":"10.1007/s12325-024-03032-3","url":null,"abstract":"<div><h3>Introduction</h3><p>In the Phase 3 XTEND-1 trial, (NCT04161495) efanesoctocog alfa prophylaxis provided superior bleed protection versus pre-study factor VIII (FVIII) replacement therapy in patients with severe haemophilia A. The aim of this study was to indirectly compare bleed outcomes between efanesoctocog alfa and standard/extended half-life (SHL and EHL) FVIII replacement therapies in adolescent and adult patients with severe haemophilia A without inhibitors.</p><h3>Methods</h3><p>A systematic literature review was conducted to identify Phase 3 trials of EHL and SHL FVIII replacement therapies for comparison with efanesoctocog alfa data from XTEND-1. Matching-adjusted indirect comparisons were used to compare annualised bleeding rates (ABRs) for any, treated, joint, and spontaneous bleeds between efanesoctocog alfa and comparators. The estimates from respective comparisons were pooled using random-effect meta-analyses to evaluate the overall difference between efanesoctocog alfa and comparator therapies.</p><h3>Results</h3><p>Four EHL therapies (rurioctocog alfa pegol, efmoroctocog alfa, turoctocog alfa pegol, damoctocog alfa pegol) and two octocog alfa SHL therapies were included. In meta-analyses, efanesoctocog alfa was associated with significantly lower ABRs for any [mean difference (95% CI) – 2.24 ( – 3.24; – 1.25)], spontaneous [ – 1.52 ( – 2.33; – 0.72)], and joint bleeds [ – 1.60 ( – 2.32; – 0.88)] versus EHL therapies, and with significantly lower ABRs for any [ – 3.61 ( – 4.43; – 2.79)], treated [ – 1.55 ( – 1.89; – 1.20)], spontaneous [ – 2.52 ( – 3.31; – 1.72)], and joint bleeds [ – 3.42 ( – 4.77; – 2.08)] versus SHL therapies.</p><h3>Conclusion</h3><p>Efanesoctocog alfa was associated with significantly lower ABRs (any, spontaneous and joint) compared with EHL or SHL prophylaxis therapies. Patients had, on average, 2.2 and 3.6 fewer bleeds per year versus EHL and SHL therapies, respectively.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 1","pages":"427 - 441"},"PeriodicalIF":3.4,"publicationDate":"2024-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782424/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142685784","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}