Advances in Therapy最新文献

{"title":"Genetic Etiologies and Outcomes in Malignancy and Mortality in Activated Phosphoinositide 3-Kinase Delta Syndrome: A Systematic Review","authors":"Katharina Büsch,&nbsp;Heidi L. Memmott,&nbsp;Heather M. McLaughlin,&nbsp;Julia E. M. Upton,&nbsp;Amanda Harrington","doi":"10.1007/s12325-024-03066-7","DOIUrl":"10.1007/s12325-024-03066-7","url":null,"abstract":"<div><h3>Introduction</h3><p>This analysis evaluated literature on patients with activated phosphoinositide 3-kinase delta syndrome (APDS) to better understand the genetic etiologies and occurrence of mortality in this population.</p><h3>Methods</h3><p>A systematic review was performed according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses approach, including all articles published in English prior to March 13, 2023, in PubMed and Embase. Patients included in the study had reported either (1) APDS diagnosis or (2) ≥ 1 clinical sign consistent with APDS and a first-degree relative with genetically confirmed APDS. Reported age at last observation was also a required outcome. Publications not meeting these criteria were excluded. Data were summarized using descriptive statistics.</p><h3>Results</h3><p>The search identified 108 publications describing 351 unique patients with 39 distinct disease-causing variants. Among these, 41 (12%) deaths were reported, with a mean age at last follow-up of 19.6 (range, 1–64) years. A cause of death was reported for 80% (33/41) of deaths; lymphoma (24%, 10/41) and infections (22%, 9/41) were the most common causes. Types of infections causing death were severe uncontrollable infections (<i>n</i> = 3), sepsis (<i>n</i> = 2), viral infection (varicella zoster pneumonitis [<i>n</i> = 1], cytomegalovirus and adenovirus [<i>n</i> = 1], and Epstein-Barr virus [<i>n</i> = 1]), and infection (<i>n</i> = 1). Mean age at death for lymphoma was 24.9 (range, 1–41) years, and all nine patients who died from infections died before the age of 15 years. The mean age at first APDS symptom was 2.0 (range, &lt; 1–22) years, and mean age at APDS diagnosis was 13.4 (range, 0–56) years; the mean time between symptoms and diagnosis was 10.6 (range, 0–44) years. Limitations of the study were primarily related to the data source.</p><h3>Conclusion</h3><p>Patients with APDS suffer early mortality, largely from lymphoma and infection, with large time gaps between symptoms and diagnosis. These findings highlight the need for improved diagnostics, earlier genetic testing for APDS, increased awareness of familial testing, and targeted therapies.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"752 - 771"},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03066-7.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783602","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":"Pharmacokinetics, Pharmacodynamics, Bioavailability, and Immunogenicity of Obexelimab Following Subcutaneous Administration in Healthy Japanese and Non-Japanese Volunteers","authors":"Xiaodong Wang,&nbsp;Rachel Kirk,&nbsp;Mark Matijevic,&nbsp;Minggeng Gao,&nbsp;Allen Poma,&nbsp;Shauna Quinn,&nbsp;Sujata Arora,&nbsp;Tanya Fischer","doi":"10.1007/s12325-024-03067-6","DOIUrl":"10.1007/s12325-024-03067-6","url":null,"abstract":"<div><h3>Introduction</h3><p>Obexelimab is an investigational, bifunctional, non-depleting, humanized monoclonal antibody that binds CD19 and FcγRIIb to inhibit B cells, plasmablasts, and CD19-expressing plasma cells. In clinical trials, intravenous (IV) administration of obexelimab has been well-tolerated, and demonstrated clinical activity in patients with rheumatoid arthritis, systemic lupus erythematosus, and immunoglobulin G4-related disease. This study was performed to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of obexelimab following subcutaneous (SC) administration, and compare PK/PD profiles between healthy Japanese and non-Japanese volunteers.</p><h3>Methods</h3><p>This was a Phase I, open-label, parallel group, multiple-dose study. Participants were randomized to five cohorts to receive three doses of obexelimab as 125 mg SC every 14 days (q14d), 250 mg SC q14d, 375 mg SC q14d, 250 mg IV q14d, and 125 mg SC every 7 days, then monitored during a 28-day safety follow-up period. PK/PD assessments were performed after the first and third doses.</p><h3>Results</h3><p>A total of 50 healthy volunteers (25 Japanese and 25 non-Japanese) were enrolled and distributed evenly between dose cohorts. All SC dosing regimens were well-tolerated. Dose-proportional PK was observed following SC doses with a bioavailability of approximately 60%. No clinically meaningful differences in PK parameters were found between healthy Japanese and non-Japanese participants. Antidrug antibodies (ADA) were detected in 6/50 (12%) participants after dosing. ADA had no or minimal impact on PK in all six ADA positive participants. Near-complete CD19 receptor occupancy and an absolute B-cell count nadir of approximately 50% baseline levels were maintained for the duration of the study in both populations.</p><h3>Conclusion</h3><p>Obexelimab SC administration demonstrated favorable bioavailability, was well-tolerated, and showed no clinically meaningful ethnic differences in PK/PD. These results support further clinical development of SC obexelimab to treat B-cell mediated autoimmune diseases.</p><h3>Trial Registration</h3><p>NCT02867098.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"813 - 829"},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783535","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":"Adherence and Persistence with Single-Inhaler Triple Therapy Among Patients with COPD Using Commercial and Medicare Advantage US Health Plan Claims Data","authors":"Corinne Young,&nbsp;Lydia Y. Lee,&nbsp;Kristi K. DiRocco,&nbsp;Guillaume Germain,&nbsp;Jacob Klimek,&nbsp;François Laliberté,&nbsp;Dominique Lejeune,&nbsp;Stephen G. Noorduyn,&nbsp;Rosirene Paczkowski","doi":"10.1007/s12325-024-03055-w","DOIUrl":"10.1007/s12325-024-03055-w","url":null,"abstract":"<div><h3>Introduction</h3><p>Previously, adherence and persistence to treatment have been shown to improve outcomes among patients with chronic obstructive pulmonary disease (COPD). This study aimed to evaluate adherence and persistence to single-inhaler triple therapy with fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI; one inhalation, once-daily) compared with budesonide/glycopyrrolate/formoterol fumarate (BUD/GLY/FOR; two inhalations, twice-daily) among patients with COPD in the USA.</p><h3>Methods</h3><p>This retrospective weighted cohort study used claims data from the IQVIA PharMetrics^® Plus Database from October 1, 2019 to March 31, 2023, to identify patients with COPD newly initiating FF/UMEC/VI or BUD/GLY/FOR. Index date was the first pharmacy claim for FF/UMEC/VI or BUD/GLY/FOR on or after October 1, 2020. The longest follow-up period was 12 months. Inverse probability of treatment weighting was used to balance baseline characteristics between cohorts. Adherence was measured as mean proportion of days covered (PDC); the proportion of patients with PDC ≥ 0.5 and PDC ≥ 0.8 was also assessed. Persistence was assessed as time to treatment discontinuation using Kaplan–Meier rates.</p><h3>Results</h3><p>Overall, 8912 and 2685 patients were included in the FF/UMEC/VI and BUD/GLY/FOR cohorts, respectively. After weighting, mean age and proportion of patients with Medicare Advantage insurance was 64.62 years and 40.0% in the FF/UMEC/VI cohort and 63.96 years and 36.1% in the BUD/GLY/FOR cohort. At 6 months post-index, mean PDC was greater in the FF/UMEC/VI versus the BUD/GLY/FOR cohort (0.65 versus 0.59; <i>P</i> &lt; 0.001). A significantly greater proportion of patients in the FF/UMEC/VI versus the BUD/GLY/FOR cohort had PDC ≥ 0.8 (45.6% versus 34.5%; <i>P</i> &lt; 0.001) and PDC ≥ 0.5 (71.8% versus 64.3%; <i>P</i> &lt; 0.001). Results were consistent at 12 months post-index. When a 30-day gap was used to define treatment discontinuation, the FF/UMEC/VI cohort had statistically significantly greater treatment persistence versus the BUD/GLY/FOR cohort at all time points.</p><h3>Conclusion</h3><p>In this study, patients initiating FF/UMEC/VI had significantly greater adherence and persistence to treatment than patients initiating BUD/GLY/FOR.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"830 - 848"},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03055-w.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783854","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":"Efficacy of Dapagliflozin + Sitagliptin + Metformin Versus Sitagliptin + Metformin in T2DM Inadequately Controlled on Metformin Monotherapy: A Multicentric Randomized Trial","authors":"Awadhesh Kumar Singh,&nbsp;Ashok Kumar Das,&nbsp;L. Sreenivasa Murthy,&nbsp;Samit Ghosal,&nbsp;Rakesh Sahay,&nbsp;K. V. S. Harikumar,&nbsp;Ganesh Hosahithlu Keshava,&nbsp;Mayur Agarwal,&nbsp;G. Vijayakumar,&nbsp;Pramila Kalra,&nbsp;Piyush Lodha,&nbsp;Sambit Das,&nbsp;Shehla Shaikh,&nbsp;Soumik Goswami,&nbsp;T. P. Ajish,&nbsp;Prashant Kumthekar,&nbsp;Mihir Upadhyay,&nbsp;Anthuvan Thamburaj,&nbsp;Aushili Mahule,&nbsp;Ashish Prasad,&nbsp;Abhijit Pednekar","doi":"10.1007/s12325-024-03037-y","DOIUrl":"10.1007/s12325-024-03037-y","url":null,"abstract":"<div><h3>Introduction</h3><p>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><h3>Methods</h3><p>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 &lt; 7.0% by week 16 of the study period.</p><h3>Results</h3><p>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; <i>P</i> &lt; 0.0001)]. Arm X showed a marked decrease in FPG [ETD − 15.42 mg/dl; 95% CI (17.63, 13.22; <i>P</i> &lt; 0.0001)], PPG [ETD − 30.39 mg/dl; 95% CI (35.59, 25.19; <i>P</i> &lt; 0.0001)], and weight [ETD − 1.47 kg; 95% CI (1.59, 1.28; <i>P</i> &lt; 0.0001)] after 16 weeks. In arm X, 54% of patients reached HbA1c &lt; 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><h3>Conclusion</h3><p>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><h3>Trial Registration</h3><p>CTRI/2022/01/039857</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"801 - 812"},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03037-y.pdf","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":"OA","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,&nbsp;Erick Álvarez,&nbsp;Mónica Novoa,&nbsp;Ivonne Lozano,&nbsp;Maribel Trujillo","doi":"10.1007/s12325-024-03040-3","DOIUrl":"10.1007/s12325-024-03040-3","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"1290 - 1290"},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03040-3.pdf","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":"OA","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,&nbsp;Carla Tordoff-Gibson,&nbsp;Patricia Dorling,&nbsp;Irene Koulinska,&nbsp;Swati Kunduri,&nbsp;Victor Laliman-Khara,&nbsp;Anna Forsythe","doi":"10.1007/s12325-024-03062-x","DOIUrl":"10.1007/s12325-024-03062-x","url":null,"abstract":"<div><h3>Objectives</h3><p>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><h3>Methods</h3><p>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><h3>Results</h3><p>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><h3>Conclusions</h3><p>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></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"579 - 596"},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03062-x.pdf","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":"OA","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,&nbsp;Runa Ono,&nbsp;Keita Odanaka,&nbsp;Hiroshi Ohara,&nbsp;Shigeki Kisara,&nbsp;Kitae Ito","doi":"10.1007/s12325-024-02988-6","DOIUrl":"10.1007/s12325-024-02988-6","url":null,"abstract":"<div><h3>Introduction</h3><p>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><h3>Methods</h3><p>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><h3>Results</h3><p>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><h3>Conclusions</h3><p>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></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"772 - 787"},"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,&nbsp;Elena Biagini,&nbsp;Vittoria Cianci,&nbsp;Federico Pieruzzi,&nbsp;Antonio Pisani,&nbsp;Antonino Tuttolomondo,&nbsp;Maurizio Pieroni","doi":"10.1007/s12325-024-03041-2","DOIUrl":"10.1007/s12325-024-03041-2","url":null,"abstract":"<div><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></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 2","pages":"597 - 635"},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03041-2.pdf","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":"OA","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,&nbsp;Pablo Fernández-Peñas,&nbsp;Marjolein de Bruin-Weller,&nbsp;Peter A. Lio,&nbsp;Chia-Yu Chu,&nbsp;Khaled Ezzedine,&nbsp;Helena Agell,&nbsp;Marta Casillas,&nbsp;Yuxin Ding,&nbsp;Fan Emily Yang,&nbsp;Evangeline Pierce,&nbsp;Thomas Bieber","doi":"10.1007/s12325-024-03010-9","DOIUrl":"10.1007/s12325-024-03010-9","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 1","pages":"144 - 145"},"PeriodicalIF":3.4,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782380/pdf/","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":"OA","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,&nbsp;Chia-Yu Chu,&nbsp;Hilde Lapeere,&nbsp;Mariateresa Rossi,&nbsp;Silvia M. Ferrucci,&nbsp;Wen-Hung Chung,&nbsp;Anne-Claire Fougerousse,&nbsp;Daria S. Fomina,&nbsp;Gregor Holzer,&nbsp;Jarmila Čelakovská,&nbsp;Mona Al-Ahmad,&nbsp;Thrasyvoulos Tzellos,&nbsp;Jiangming Wu,&nbsp;Marius Ardeleanu,&nbsp;Kwinten Bosman","doi":"10.1007/s12325-024-03049-8","DOIUrl":"10.1007/s12325-024-03049-8","url":null,"abstract":"<div><h3>Introduction</h3><p>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><h3>Methods</h3><p>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><h3>Results</h3><p>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><h3>Conclusions</h3><p>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><h3>Clinical Trial Registration</h3><p>ClinicalTrials.gov Identifier NCT03992417.</p><h3>Graphical Abstract</h3>\u0000<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":"720 - 733"},"PeriodicalIF":3.4,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03049-8.pdf","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}