Advances in Therapy最新文献

{"title":"Efficacy of Dapagliflozin + Sitagliptin + Metformin Versus Sitagliptin + Metformin in T2DM Inadequately Controlled on Metformin Monotherapy: A Multicentric Randomized Trial.","authors":"Awadhesh Kumar Singh, Ashok Kumar Das, L Sreenivasa Murthy, Samit Ghosal, Rakesh Sahay, K V S Harikumar, Ganesh Hosahithlu Keshava, Mayur Agarwal, G Vijayakumar, Pramila Kalra, Piyush Lodha, Sambit Das, Shehla Shaikh, Soumik Goswami, T P Ajish, Prashant Kumthekar, Mihir Upadhyay, Anthuvan Thamburaj, Aushili Mahule, Ashish Prasad, Abhijit Pednekar","doi":"10.1007/s12325-024-03037-y","DOIUrl":"https://doi.org/10.1007/s12325-024-03037-y","url":null,"abstract":"<p><strong>Introduction: </strong>A slower adoption rate of fixed dose combinations (FDC) in diabetes management is partly due to insufficient data. This study evaluates the safety and efficacy of an FDC of dapagliflozin + sitagliptin + metformin hydrochloride extended release (XR), compared to a dual FDC of sitagliptin + metformin hydrochloride XR among patients with type 2 diabetes mellitus (T2DM) with poor glycemic control when treated with metformin monotherapy.</p><p><strong>Methods: </strong>A total of 274 patients with T2DM were randomized (1:1) to either arm X, receiving FDC of dapagliflozin (10 mg) + sitagliptin (100 mg) + metformin hydrochloride XR (1000 mg) (Dapa + Sita + Met) tablets, or arm Y, receiving sitagliptin phosphate (100 mg) + metformin hydrochloride XR (1000 mg) (Sita + Met) tablets, and treated for 16 weeks. The outcome measures included changes in hemoglobin A1c (HbA1c)(%), fasting plasma glucose (FPG), 2-h post-prandial glucose (PPG), weight, and the proportion of patients achieving target HbA1c levels of < 7.0% by week 16 of the study period.</p><p><strong>Results: </strong>The reduction in HbA1c at week 16 was significantly higher in arm X than in arm Y [estimated treatment difference (ETD), - 0.65% (95% CI - 0.76 to - 0.53; P < 0.0001)]. Arm X showed a marked decrease in FPG [ETD - 15.42 mg/dl; 95% CI (17.63, 13.22; P < 0.0001)], PPG [ETD - 30.39 mg/dl; 95% CI (35.59, 25.19; P < 0.0001)], and weight [ETD - 1.47 kg; 95% CI (1.59, 1.28; P < 0.0001)] after 16 weeks. In arm X, 54% of patients reached HbA1c < 7.0% compared to 29.9% in arm Y. The incidence of adverse events was comparable [13.14% (arm X) vs 12.4% (arm Y)]. There was no severe hypoglycemia-led treatment discontinuation.</p><p><strong>Conclusion: </strong>Among patients with T2DM who have poor glycemic control with metformin monotherapy, triple FDC (Dapa + Sita + Met) effectively helped achieve better glycemic response compared to dual FDC (Sita + Met), with a comparable safety and tolerability profile.</p><p><strong>Trial registration: </strong>CTRI/2022/01/039857.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783517","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":"Correction to: Oleogel-S10 in Dystrophic Epidermolysis Bullosa: A Case Series Evaluating the Impact on Wound Burden Over Two Years.","authors":"Mauricio Torres Pradilla, Erick Álvarez, Mónica Novoa, Ivonne Lozano, Maribel Trujillo","doi":"10.1007/s12325-024-03040-3","DOIUrl":"https://doi.org/10.1007/s12325-024-03040-3","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783506","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":"Influence of Treatment Effect Modifiers in Fabry Disease: A Systematic Literature Review.","authors":"Khashayar Azimpour, Carla Tordoff-Gibson, Patricia Dorling, Irene Koulinska, Swati Kunduri, Victor Laliman-Khara, Anna Forsythe","doi":"10.1007/s12325-024-03062-x","DOIUrl":"https://doi.org/10.1007/s12325-024-03062-x","url":null,"abstract":"<p><strong>Objectives: </strong>Fabry disease (FD) is a rare metabolic disorder which presents with considerable heterogeneity in disease characteristics. Given the absence of interventional studies comparing all available treatments, it is important for indirect treatment comparisons (ITCs) to account for potential treatment effect modifiers (TEMs). This systematic literature review (SLR) aimed to identify patient characteristics that may impact clinical outcomes by analyzing real-world evidence (RWE) in FD.</p><p><strong>Methods: </strong>An SLR was conducted according to PRISMA guidelines, with searches performed in the EMBASE, MEDLINE, and Cochrane databases (1946-2022; with a recent update in April 2023). Full-text articles reporting clinical outcomes from RWE studies of pharmacological therapies for the treatment of FD were included.</p><p><strong>Results: </strong>Including studies from the recent SLR update, a total of 119 original studies met the PICOS criteria and 25 studies provided insights into TEMS. Potential TEMs in FD were identified: sex, age, timing of treatment initiation (early/delayed), left ventricular hypertrophy (LVH), estimated glomerular filtration rate (eGFR), proteinuria, presence of anti-drug-antibodies (ADAs) at baseline, and previous enzyme replacement therapy (ERT). In three studies (two including ERT-treated patients and one study of migalastat-treated patients) males showed worse renal outcomes than females. Five studies found that younger patients and those who received initial ERT before the age of 25 years had greater reductions in plasma-lysoGb3, as well as more favorable renal, cardiac, and biochemical outcomes. Seven studies identified associations between LVH and reduced eGFR at baseline, along with an increased risk of cardiovascular, renal, and neurological events. In four studies, lower baseline eGFR and proteinuria were associated with faster annual eGFR decline despite ERT; high baseline proteinuria was a significant predictor of renal disease progression. Baseline ADAs were linked to lower eGFR, increased left ventricular mass, and reduced treatment impact on plasma/urine-lysoGb3. Migalastat was effective in treatment-naïve patients, while those previously treated with ERT experienced deteriorations in mean lysoGb3, eGFR, and left ventricular mass.</p><p><strong>Conclusions: </strong>This SLR highlighted several patient characteristics that influence treatment effectiveness in FD. It is important to account for these characteristics in ITCs to ensure unbiased outcomes.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783615","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":"A Pharmacoeconomic Study of Post-Exposure Prophylaxis Strategies for Influenza Virus Infections in Japan.","authors":"Naoto Nakagawa, Runa Ono, Keita Odanaka, Hiroshi Ohara, Shigeki Kisara, Kitae Ito","doi":"10.1007/s12325-024-02988-6","DOIUrl":"https://doi.org/10.1007/s12325-024-02988-6","url":null,"abstract":"<p><strong>Introduction: </strong>Vaccines can prevent influenza (flu) infections and are cost-effective for society and healthcare. However, the cost-effectiveness of post-exposure prophylaxis as a follow-up strategy is unclear. This study aims to evaluate the cost utility of post-exposure prophylaxis and treatment strategies with neuraminidase inhibitors and a cap-dependent endonuclease inhibitor for flu infections from the perspective of healthcare costs in Japan.</p><p><strong>Methods: </strong>A base-case analysis was used to compare oseltamivir, zanamivir, and laninamivir for neuraminidase inhibitors and baloxavir marboxil for the endonuclease inhibitor. The costs of the first visit to a physician and pharmacy were excluded because of the policy on out-of-pocket expenses for post-exposure prophylaxis in Japan. Direct medical costs include the second physician visit, pharmacy and hospital admission expenses, and drug prices, based on the 2020 Japanese Medical Fee Index. The EuroQol-5Dimention-5Level was utilized to measure healthy participants' quality of life scores, with a time horizon of 14 days. Deterministic and probabilistic sensitivity analyses were conducted.</p><p><strong>Results: </strong>We have found baloxavir marboxil as the post-exposure prophylaxis agent and laninamivir as the treatment agent to be the most cost-effective strategy in Japan, followed by oseltamivir as the post-exposure prophylaxis agent and zanamivir as the treatment agent.</p><p><strong>Conclusions: </strong>Baloxavir marboxil and oseltamivir are cost-effective prophylactic agents for flu from the perspective of healthcare costs in Japan. This strategy to select baloxavir marboxil or oseltamivir would be helpful to manage a formulary for post-exposure prophylaxis in Japan.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783853","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":"Effects of Current Therapies on Disease Progression in Fabry Disease: A Narrative Review for Better Patient Management in Clinical Practice.","authors":"Renzo Mignani, Elena Biagini, Vittoria Cianci, Federico Pieruzzi, Antonio Pisani, Antonino Tuttolomondo, Maurizio Pieroni","doi":"10.1007/s12325-024-03041-2","DOIUrl":"https://doi.org/10.1007/s12325-024-03041-2","url":null,"abstract":"<p><p>Fabry disease (FD) is a rare lysosomal storage disorder that is characterized by renal, neurological, and cardiovascular dysfunction. Four treatments are currently available for patients with FD; three enzyme replacement therapies (ERTs; agalsidase alfa, agalsidase beta, and pegunigalsidase alfa) and one pharmacological chaperone (migalastat). This review focuses on the evidence for the benefits of ERTs and migalastat, and provides an overview of their impact on disease manifestations and quality of life (QoL). Agalsidase beta is associated with renal, neurological, and cardiovascular benefits, and may prevent renal disease progression. Agalsidase alfa provides stabilizing effects across all main organ systems, although minor sex-specific differences exist in patients with more advanced baseline disease. The benefits of agalsidase alfa and agalsidase beta are similar but depend on the extent of baseline disease. Some data indicate that agalsidase beta may be preferable over the longer term. Both agalsidase alfa and agalsidase beta are associated with improved gastrointestinal and pain symptoms, as well as improved QoL. Patients with advanced end-organ damage tend not to respond as optimally to ERTs as those who initiate ERTs before irreversible organ fibrosis develops, highlighting the need for early treatment initiation. Migalastat, which is only approved for patients with amenable missense gene variants, generally stabilizes renal parameters and provides cardiovascular benefits. Migalastat also improves diarrhea and pain, and stabilizes QoL (although ERT may be more effective for pain management), but the neurological effects of migalastat have not been studied. Real-world data raise concerns about effective in vivo amenability of some genetic variants. Future studies with direct treatment comparisons in patients with FD are needed.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783512","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":"Correction to: Improvement Across Dimensions of Disease with Lebrikizumab Use in Atopic Dermatitis: Two Phase 3, Randomized, Double-Blind, Placebo-Controlled Monotherapy Trials (ADvocate1 and ADvocate2).","authors":"Eric Simpson, Pablo Fernández-Peñas, Marjolein de Bruin-Weller, Peter A Lio, Chia-Yu Chu, Khaled Ezzedine, Helena Agell, Marta Casillas, Yuxin Ding, Fan Emily Yang, Evangeline Pierce, Thomas Bieber","doi":"10.1007/s12325-024-03010-9","DOIUrl":"https://doi.org/10.1007/s12325-024-03010-9","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783478","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":"One-Year Insights into the GLOBOSTAD Multinational Prospective Observational Study of Patients Receiving Dupilumab for Atopic Dermatitis.","authors":"Piergiacomo Calzavara-Pinton, Chia-Yu Chu, Hilde Lapeere, Mariateresa Rossi, Silvia M Ferrucci, Wen-Hung Chung, Anne-Claire Fougerousse, Daria S Fomina, Gregor Holzer, Jarmila Čelakovská, Mona Al-Ahmad, Thrasyvoulos Tzellos, Jiangming Wu, Marius Ardeleanu, Kwinten Bosman","doi":"10.1007/s12325-024-03049-8","DOIUrl":"https://doi.org/10.1007/s12325-024-03049-8","url":null,"abstract":"<p><strong>Introduction: </strong>Currently, limited data are available on long-term use of dupilumab to treat atopic dermatitis (AD) in a multinational real-world setting. The aim of this analysis was to report the interim 1-year data for patients with AD enrolled in the GLOBOSTAD registry, including treatment patterns, dupilumab effectiveness and safety, and healthcare burden.</p><p><strong>Methods: </strong>GLOBOSTAD is an ongoing, 5-year, multinational, prospective, observational study of adult/adolescent (aged ≥ 12 years at baseline) patients with AD who initiated dupilumab in real-world settings according to their local country-specific prescribing guidelines. Outcomes were evaluated at baseline and at 3, 6 and 12 months and included Eczema Area and Severity Index (EASI) total score, SCORing Atopic Dermatitis (SCORAD) total score, percent body surface area (BSA) affected, Patient-Oriented Eczema Measure (POEM), Dermatology Life Quality Index (DLQI) total score for adults or Children's Dermatology Life Quality Index (CDLQI) total score for adolescents and pruritus Numeric Rating Scale (NRS) total score.</p><p><strong>Results: </strong>At the interim 1-year cut-off (March 2023), 955 patients were enrolled in GLOBOSTAD, and follow-up data were obtained from 903 patients. After dupilumab initiation, mean improvements in effectiveness outcome measures from baseline to month 3 were EASI from 25.1 to 6.1, SCORAD 59.3 to 25.3, POEM 19.7 to 8.7, DLQI 13.7 to 5.3, CDLQI 12.2 to 2.7 and pruritus NRS 6.3 to 2.5, with each measure exceeding the minimal clinically important difference. These positive changes in effectiveness outcomes were maintained or further improved through 12 months since treatment initiation. AD-related hospitalizations and emergency room or urgent care facility visits decreased from 11.1% to 1.7% from baseline to month 12.</p><p><strong>Conclusions: </strong>In a multinational real-world setting, dupilumab demonstrated rapid, robust and sustained effectiveness in patients with moderate-to-severe AD across multiple disease domains, including AD signs, symptoms, quality of life and emergency/urgent care visits. Safety was consistent with the known dupilumab safety profile.</p><p><strong>Clinical trial registration: </strong>ClinicalTrials.gov Identifier NCT03992417.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142764739","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":"Addressing the Human Experience of Chronic Kidney Disease: A Call to Transform Kidney Care.","authors":"Ricardo Correa-Rotter, Steven J Chadban, Laura Christen, Kelli Collins Damron, Lweendo Hamusankwa, Sarah Jarvis, Surendra Pentakota, Marisol Robles, Petrina Stevens, Christoph Wanner","doi":"10.1007/s12325-024-03048-9","DOIUrl":"https://doi.org/10.1007/s12325-024-03048-9","url":null,"abstract":"<p><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.A patient perspective video and a graphical abstract are available with this article. 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>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"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, Shannon A Ferrante, Natalie J Shiff, Timothy P Fitzgerald, Soumya D Chakravarty, Jessica A Walsh","doi":"10.1007/s12325-024-03042-1","DOIUrl":"https://doi.org/10.1007/s12325-024-03042-1","url":null,"abstract":"<p><strong>Introduction: </strong>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><p><strong>Methods: </strong>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><p><strong>Results: </strong>Weighted demographic and disease characteristics were well balanced between the cohorts (guselkumab: N = 910, mean age = 50.4 years, 60.4% female; SC IL-17Ai: N = 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 (p < 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 (p < 0.001).</p><p><strong>Conclusion: </strong>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>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","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":"https://doi.org/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}