Advances in Therapy最新文献

{"title":"Therapeutic Advances in Hereditary Angioedema: A Focus on Present and Future Options.","authors":"Kelsey Uminski, Dawn Goodyear, Stephen Betschel","doi":"10.1007/s12325-025-03382-6","DOIUrl":"https://doi.org/10.1007/s12325-025-03382-6","url":null,"abstract":"<p><p>Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable and potentially life-threatening episodes of swelling, driven primarily by excessive bradykinin production. These episodes commonly involve the skin, gastrointestinal tract, and upper airway, significantly impacting patients' quality of life. Recent advances in understanding the underlying pathophysiology of HAE have transformed clinical care, enabling the development of highly targeted treatments that disrupt critical steps within the kallikrein-kinin pathway. Current on-demand therapies rapidly relieve acute symptoms, while contemporary prophylactic strategies have substantially reduced attack frequency and improved patient autonomy and health-related quality of life. Emerging therapies-including novel oral agents, monoclonal antibodies, RNA therapies, and pioneering gene editing approaches-continue to evolve, aiming to simplify treatment and further personalize care. These innovative treatments collectively strive to address remaining unmet needs, ensuring broader accessibility, convenience, and long-term sustainability of care for individuals living with HAE. This narrative review highlights the progression of therapeutic options in HAE, summarizing current advances and exploring future strategies toward personalized and patient-centered care.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145273300","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":"Immunosuppressive Therapy for Pulmonary Arterial Hypertension Associated with Connective Tissue Diseases: Insights from Japanese Registry.","authors":"Yudai Tamura, Yuichi Tamura, Ryo Takemura, Yu Taniguchi, Ichizo Tsujino, Takumi Inami, Hiromi Matsubara, Ayako Shigeta, Masaru Hatano, Shiro Adachi, Nobuhiro Tahara, Keiichi Sakurai, Koshin Horimoto, Nobuhiro Yaoita, Kohtaro Abe, Yoshihiro Dohi, Kazuhiro Kimura, Kayoko Kubota, Noriko Kikuchi, Hidekata Yasuoka, Yuichi Baba, Toshiro Shinke, Mari Amino, Natsumi Yamaguchi, Satoshi Ikeda, Teruki Sato, Masaru Ishida, Fusako Sera, Naohiko Nakanishi, Hakuoh Konishi, Koichiro Kinugawa, Takeshi Kashimura, Kaoru Dohi, Kazufumi Nakamura, Soichiro Usui, Sumiaki Tanaka, Shuji Kubota, Nobutaka Ikeda, Masanori Yoshikawa, Keiichi Odagiri, Sadatomo Tasaka, Yasuchika Takeishi, Teruyasu Sugano, Koichiro Sugimura, Koichiro Tatsumi, Masataka Kuwana","doi":"10.1007/s12325-025-03389-z","DOIUrl":"https://doi.org/10.1007/s12325-025-03389-z","url":null,"abstract":"<p><strong>Introduction: </strong>The combination of immunosuppressive therapy (IST) and pulmonary artery vasodilators has demonstrated potential effectiveness in treating pulmonary arterial hypertension (PAH) associated with connective tissue diseases (CTD-PAH) other than systemic sclerosis (SSc). However, large-scale studies of this topic are limited. This study aimed to evaluate the effectiveness of early IST in treating PAH in patients diagnosed with non-SSc CTD-PAH.</p><p><strong>Methods: </strong>Clinical data for patients with non-SSc CTD-PAH were collected from the Japan Pulmonary Hypertension Registry spanning 2008-2021. Early IST was defined as the initiation or intensification of therapy within 3 months of PAH diagnosis.</p><p><strong>Results: </strong>The study included 141 patients (mean age 51 ± 16.7 years; 95% female), with 57 receiving early IST, across 43 centers in Japan. The primary underlying diseases were systemic lupus erythematosus, mixed connective tissue disease, and Sjögren syndrome. At baseline, there were no significant differences in hemodynamics or PAH treatment regimens between the IST and non-IST groups. However, the IST group was notably younger, had higher plasma IgG levels, and maintained better renal function. The IST group showed significantly greater improvements in mean pulmonary artery pressure (mPAP) and pulmonary vascular resistance (PVR) over 12 months (p = 0.032 and p = 0.028), along with significant reductions in all-cause (p = 0.039) and PAH-related (p = 0.020) mortalities. No significant differences in deaths due to infections or malignancies were observed between groups.</p><p><strong>Conclusion: </strong>Our data suggest that early initiation of IST may be associated with hemodynamic improvement; prospective confirmation in international cohorts is warranted.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145249242","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":"Efficiency of Gla-300 versus Gla-100 Across Age Groups in People Living with Type 2 Diabetes in France: A Post Hoc Analysis of an Observational Longitudinal Study.","authors":"Patrice Darmon, Olivier Hanon, Pierre Gourdy, Isabelle Borget, Bruno Detournay, Pierre Evenou, Isabelle Bureau, Noemie Allali, Aymeric Mahieu, Corinne Emery, Alfred Penfornis","doi":"10.1007/s12325-025-03381-7","DOIUrl":"https://doi.org/10.1007/s12325-025-03381-7","url":null,"abstract":"<p><strong>Introduction: </strong>The EF-BI study, a nationwide observational and retrospective study conducted in France, found better insulin persistence, fewer insulin therapy-related acute hospitalizations, and lower healthcare costs in adults with Type 2 diabetes (T2D) who initiated treatment with insulin glargine 300 U/mL (Gla-300) compared to those who started on insulin glargine 100 U/mL (Gla-100) over a 3-year follow-up period. Given the heterogeneity of the T2D population, the benefits observed may vary according to individuals' characteristics. A post hoc analysis of this study was conducted to verify whether these findings were consistent in all age groups, including the elderly population.</p><p><strong>Methods: </strong>Using the French national health insurance information system, adults with T2D who initiated basal insulin therapy with Gla-300 or Gla-100 between January 1, 2016, and December 31, 2020, and had no prior insulin use in the previous 6 months, were selected. Covariate adjustment using a propensity score based on key individuals' characteristics was applied to estimate outcomes and costs between the two basal insulins. Analyses were conducted using several age cut-offs (65, 75, and 80 years).</p><p><strong>Results: </strong>Overall, 235,894 people with T2D were included: 175,537 initiated treatments with Gla-100 and 60,357 with Gla-300. Across both age groups (< 65 years or older), treatment with Gla-300 was associated with better persistence and a lower frequency of acute insulin-related events. Cost comparisons per individual treated over 3 years showed significant results favoring Gla-300:- €2,031 (- 7.3%, p < 0.0001) in the < 65 years group, and- €2,914 (- 7%, p < 0.0001) in the older group.</p><p><strong>Conclusion: </strong>This EF-BI post hoc analysis found that Gla-300 was associated with lower overall costs, better persistence, and fewer insulin-related acute events compared to Gla-100 in several age subgroups in people with T2D in France. Further investigations are needed to confirm these results, including glycemic control and comparison with other second-generation basal insulins.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145249250","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":"Real-World Health Care Resource Utilization and Costs Among Patients with Activated Phosphoinositide 3-Kinase Delta (PI3Kδ) Syndrome in the United States.","authors":"Nicholas L Rider, François Laliberté, Guillaume Germain, Ana Urosevic, Malena Mahendran, Amanda Harrington","doi":"10.1007/s12325-025-03377-3","DOIUrl":"https://doi.org/10.1007/s12325-025-03377-3","url":null,"abstract":"<p><strong>Introduction: </strong>This study sought to describe health care resource utilization (HRU) and treatment use among patients with activated phosphoinositide 3-kinase delta syndrome (APDS) and assess the incremental HRU and health care costs associated with APDS relative to a non-APDS control cohort.</p><p><strong>Methods: </strong>Patients with APDS were identified via Symphony Health Solutions Integrated Dataverse database. Controls were selected using a 10:1 ratio and matched to patients with APDS. Rates of HRU (hospitalizations, emergency department [ED] visits, outpatient [OP] visits, other visits) per person-year (PPY) and annual health care costs were compared using rate ratios (RRs) estimated from Poisson regression models and cost ratios (CRs) estimated from gamma regression models, respectively. Use of symptomatic treatments associated with APDS was also assessed.</p><p><strong>Results: </strong>Mean (standard deviation [SD]) age of patients with APDS (n = 42) and controls (n = 420) was 16.0 (15.1) and 16.9 (17.4) years, respectively. Patients with APDS had significantly higher PPY rates of all-cause hospitalizations (RR 4.31, 95% confidence interval [CI] 1.83-8.03; p < 0.001) and OP visits (RR 1.77, 95% CI 1.24-2.61; p < 0.001) vs controls, with OP visits driven by higher rates of OP hospital visits (RR 5.32, 95% CI 3.30-8.29; p < 0.001). Patients with APDS had annual all-cause total health care costs 10.54 times greater (95% CI 4.56-20.67; p < 0.001) than controls, driven by higher medical costs (CR 12.39, 95% CI 5.04-25.82; p < 0.001). Significantly higher costs for hospitalizations and ED, OP (office/clinic and hospital), and other visits were observed in patients with APDS vs controls. HRU rates and costs (except other visits) remained significantly higher in patients with APDS vs controls after excluding visits related to immunoglobulin replacement therapy.</p><p><strong>Conclusion: </strong>Higher HRU and health care costs among patients with APDS vs controls reflect the high clinical and economic burden of APDS.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145249290","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":"Matching-Adjusted Indirect Treatment Comparison of Tarlatamab Versus Comparator Therapies in England in Patients with Extensive-Stage Small Cell Lung Cancer Who Have Received Two or More Prior Lines of Therapy.","authors":"Rumbidzai Takundwa, Gaurav Suri, Franziska Dirnberger, Andre Verhoek, Elizabeth Vinand, Jessie Wang, Stephen Puntis, Xerxes Pundole, Fiona Blackhall","doi":"10.1007/s12325-025-03376-4","DOIUrl":"https://doi.org/10.1007/s12325-025-03376-4","url":null,"abstract":"<p><strong>Introduction: </strong>The non-comparative phase 2 DeLLphi-301 trial found that tarlatamab, a bispecific T cell engager immunotherapy, can provide sustained objective response rates and improved survival outcomes for patients with previously treated extensive-stage small cell lung cancer (ES-SCLC). In the absence of direct head-to-head evidence, an indirect treatment comparison was conducted to estimate the efficacy of tarlatamab relative to comparator therapies in England.</p><p><strong>Methods: </strong>The outcomes of patients in the DeLLphi-301 trial were compared against those of a comparator therapies cohort identified through linkage of population-level real-world databases in England. The study population consisted of patients with relapsed ES-SCLC who received two or more prior lines of therapy and who met key eligibility criteria from the DeLLphi-301 trial. Outcomes of interest included overall survival (OS) and progression-free survival (PFS). An unanchored matching-adjusted indirect comparison (MAIC) was used to estimate the efficacy of tarlatamab relative to available comparator therapies, adjusting for key baseline characteristics to match the patient cohort from DeLLphi-301 with the comparator therapies cohort. A weighted Cox proportional hazards model with robust variance estimation was used to estimate the hazard ratio (HR) and 95% confidence interval (CI) for both OS and PFS.</p><p><strong>Results: </strong>The analyses included 97 patients enrolled in the DeLLphi-301 trial and 540 patients receiving available treatment options in England. After matching, tarlatamab was associated with improved OS (HR 0.24; 95% CI 0.14, 0.43) and PFS (HR 0.18; 95% CI 0.11, 0.31) relative to the comparator therapies. Findings were similar in sensitivity analyses performed by changing the variables for adjustment.</p><p><strong>Conclusion: </strong>Tarlatamab provides a clinically meaningful survival benefit compared to currently available treatments in England. These findings support the use of tarlatamab as an effective treatment option for patients with previously treated ES-SCLC.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145249224","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: The Effect of Leuprolide Acetate 11.25 mg 3-Month Formulation in Children with Central Precocious Puberty: A Systematic Review and Meta-analysis.","authors":"Linqi Han, Chunjuan Zhai, La Da, Yan Sun","doi":"10.1007/s12325-025-03315-3","DOIUrl":"https://doi.org/10.1007/s12325-025-03315-3","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145249203","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":"The Global Patient Perspective on Uncontrolled Moderate-to-Severe Asthma: Reducing Delays in Diagnosis and Treatment.","authors":"Karen Rance, Brenda Young, Gretchen McCreary, Stephanie Williams, Marilyn Urrutia-Pereira, Kristen Willard, Ghulam Mustafa, Purvi Parikh, Tonya Winders, Ruth Tal-Singer","doi":"10.1007/s12325-025-03346-w","DOIUrl":"https://doi.org/10.1007/s12325-025-03346-w","url":null,"abstract":"<p><strong>Introduction: </strong>Uncontrolled asthma greatly affects quality of life globally and highlights unmet medical needs. Despite advances in treatment and care, many patients still experience delayed diagnoses, poor symptom control, and a reliance on emergency care. The Global Allergy and Airways Patient Platform (GAAPP) surveyed patients with moderate-to-severe uncontrolled asthma to assess their care experiences.</p><p><strong>Methods: </strong>The GAAPP Time Clock Survey is a cross-sectional, online, multilingual survey of adults living in Brazil, Germany, Italy, Japan, Saudi Arabia, the United Arab Emirates, and the US. The survey examined diagnosis, symptoms, treatment outcomes, challenges in self-management, and timelines for care coordination.</p><p><strong>Results: </strong>A total of 1401 individuals with self-reported asthma using combination inhaler therapy and experiencing symptoms were enrolled in this study. Among these participants, 56% reported waiting more than 1 month to undergo pulmonary function testing for diagnosis. Additionally, 51% indicated minimal to no improvement in quality of life despite treatment interventions. Difficulties in asthma management were reported by 42% of participants, with some describing the process as difficult or very difficult. Approximately 32% of individuals used daily corticosteroids. Nearly half of the cohort consulted three or more healthcare providers in their pursuit of effective asthma management. Emergency department visits were common, with 50% seeking urgent care for uncontrolled symptoms and 35% requiring hospitalization.</p><p><strong>Conclusion: </strong>This study underscores the importance of policy reforms that prioritize timely diagnosis, shared decision-making, and long-term disease control. Improving outcomes for patients with uncontrolled asthma will require both clinical innovation and structural transformation.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145231326","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 Functional Genetic Score in the ZMIZ1/TGF-β/STAT Pathway Predicts Early Biologic Discontinuation in Psoriasis Patients Treated with Anti-TNF and Anti-IL12/23 Agents.","authors":"Juan de Luque, Carmen Mochón-Jiménez, Irene Rivera-Ruiz, Jesús Gay-Mimbrera, Judilyn Fuentes-Duculan, Israel Coats, Macarena Aguilar-Luque, Beatriz Isla-Tejera, Antonio Vélez-García Nieto, Manuel Galán-Gutiérrez, Teresa López-Viñau López, Mayte Suarez-Fariñas, James G Krueger, Juan Ruano","doi":"10.1007/s12325-025-03350-0","DOIUrl":"https://doi.org/10.1007/s12325-025-03350-0","url":null,"abstract":"<p><strong>Introduction: </strong>Biologic drug survival in psoriasis is variable. While clinical factors such as obesity and comorbidities contribute to early discontinuation, genetic predictors are less defined. The ZMIZ1/TGF-β/STAT axis regulates immune-metabolic responses and represents a promising pharmacogenetic target.</p><p><strong>Methods: </strong>We retrospectively analyzed 875 biologic treatment courses from 312 patients with moderate-to-severe psoriasis (NCT07041112). A pathway-based genetic score was derived from seven single nucleotide polymorphisms (SNPs) in the ZMIZ1/TGF-β/STAT axis and dichotomized at the median. The primary outcome was time to biologic discontinuation, assessed with Kaplan-Meier curves and Cox proportional hazards models adjusted for demographic, clinical, and inflammatory covariates.</p><p><strong>Results: </strong>Patients with a high genetic score had significantly longer drug survival (hazard ratio [HR] = 0.74; 95% confidence interval [CI]: 0.62-0.89; p = 0.0015), despite elevated baseline tumor necrosis factor (TNF)-α, interleukin (IL)-1β, IL-15, and leptin. This indicates that genetic background simultaneously conditioned immune-metabolic activation and treatment persistence. Predictive value was strongest for anti-IL12/23 agents (HR = 0.44; 95% CI: 0.26-0.75; p = 0.002) and anti-TNF therapies (HR = 0.79; 95% CI: 0.62-0.99; p = 0.045), but absent for anti-IL17/IL-23 agents after adjustment. None of the circulating biomarkers independently predicted survival.</p><p><strong>Conclusion: </strong>A functional genetic score in the ZMIZ1/TGF-β/STAT pathway independently predicted long-term biologic persistence in psoriasis, particularly with anti-TNF and anti-IL12/23 therapies. Its association with immune-metabolic activation suggests that genetic background shapes both inflammatory status and treatment durability. Incorporating such profiling into predictive algorithms may improve treatment personalization and biologic retention.</p><p><strong>Trial registration: </strong>Trial Registration NCT07041112.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145231363","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":"Quantifying the Clinical and Economic Burden of Graft-Versus-Host Disease Following Allogeneic Haematopoietic Stem Cell Transplantation in England: A Retrospective Cohort Study.","authors":"Francesca Kinsella, Nadia Quignot, Stephanie H Read, Gaelle Gusto, Kazue Kikuchi, Dawn Reichenbach, Anita Burrell, Shicheng Weng, Charlotte Pollet, Kris Thiruvillakkat","doi":"10.1007/s12325-025-03355-9","DOIUrl":"https://doi.org/10.1007/s12325-025-03355-9","url":null,"abstract":"<p><strong>Introduction: </strong>Evidence on the burden of graft-versus-host disease (GVHD) among allogeneic haemopoietic stem cell transplantation (allo-HSCT) recipients in England is lacking. We compared clinical and economic outcomes among adult allo-HSCT recipients who developed acute GVHD (aGVHD) or chronic GVHD (cGVHD) versus those who did not develop GVHD.</p><p><strong>Methods: </strong>This retrospective cohort study included adult patients with haematological malignancies who underwent allo-HSCT in England between 2011 and 2023 and were captured in the National Cancer Registration and Analysis Service database. Patients were grouped according to GVHD development following allo-HSCT. Propensity score matching was used to balance covariates between aGVHD, cGVHD and without-GVHD groups. Clinical (mortality, severe infections) and economic (healthcare resource utilization, costs) outcomes observed during follow-up were extracted from the Hospital Episode Statistics database and were compared across GVHD groups.</p><p><strong>Results: </strong>Among 9450 eligible patients, 5022 (53.14%) developed GVHD. Hazard ratios (95% confidence intervals [CI]) indicated that patients with GVHD were at increased risk of mortality (aGVHD 1.61 [1.49-1.75]; cGVHD 2.12 [1.83-2.44]) and severe infection (aGVHD 1.94 [1.79-2.10]; cGVHD 3.11 [2.45-3.95]) versus patients without GVHD. Patients with GVHD had considerably greater overnight hospitalization rates than patients without GVHD (aGVHD 1.32 [95% CI 1.30-1.35] versus 0.55 [95% CI 0.54-0.57]; cGVHD 1.18 [95% CI 1.15-1.22] versus 0.33 [95% CI 0.32-0.35] overnight hospitalization rate per person-year, respectively). Total healthcare costs were approximately doubled for both patients with aGVHD compared with patients without GVHD (mean [standard deviation (SD)] £12,026 [£21,325] versus £6916 [£14,767], p < 0.05) and patients with cGVHD compared with patients without GVHD (mean [SD] £11,707 [£20,722] versus £5227 [£11,041]).</p><p><strong>Conclusions: </strong>GVHD is associated with considerable burden in England, underscoring the urgent need for improved prevention and treatment options.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145231393","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":"Peak Nasal Inspiratory Flow and the Association with Nasal Obstruction in Patients with Severe CRSwNP from the SINUS-24/-52 Studies.","authors":"Martin Desrosiers, Scott Nash, Andrew Lane, Stella E Lee, Eugenio De Corso, Changming Xia, Mark Corbett, Amr Radwan, Paul J Rowe, Yamo Deniz","doi":"10.1007/s12325-025-03378-2","DOIUrl":"https://doi.org/10.1007/s12325-025-03378-2","url":null,"abstract":"<p><strong>Introduction: </strong>Nasal congestion/obstruction (NC) contributes to the high disease burden in patients with severe chronic rhinosinusitis with nasal polyps (CRSwNP). Patient perception of NC may not accurately reflect nasal patency, while peak nasal inspiratory flow (PNIF) is an objective method with established thresholds for normal nasal airflow. This analysis evaluated the association between NC and PNIF and the impact of baseline PNIF on dupilumab efficacy in patients with severe CRSwNP.</p><p><strong>Methods: </strong>This was a post hoc analysis of patients treated with placebo or dupilumab 300 mg every 2 weeks in the SINUS-24 (NCT02912468) and SINUS-52 (NCT02898454) phase III studies. Patients provided daily e-diary measures of PNIF (L/min) using PNIF meters, and NC by patient-reported evaluation of severity (scored 0-3). Other assessed outcomes were nasal polyp score (NPS), 22-item Sinonasal Outcome Test (SNOT-22), loss of smell (LoS), University of Pennsylvania Smell Identification Test (UPSIT), and Lund-Mackay computed tomography. Outcomes were assessed in two subgroups: baseline PNIF < 120 L/min and ≥ 120 L/min.</p><p><strong>Results: </strong>Of 724 patients, 552 (76%) had PNIF < 120 L/min and 172 (24%) had PNIF ≥ 120 L/min at baseline. The PNIF < 120 L/min subgroup had higher mean scores for NPS and SNOT-22 and more smell impairment (LoS and UPSIT). PNIF weakly correlated with NC at baseline (Spearman coefficient - 0.348 [95% CI - 0.410, - 0.282], P < 0.0001). Correlations between change from baseline in PNIF and NC at week 24 were weak in the dupilumab group (- 0.390 [- 0.468, - 0.305], P < 0.0001) and moderate in the placebo group (- 0.497 [- 0.582, - 0.399], P < 0.0001).</p><p><strong>Conclusion: </strong>These results confirm PNIF as a valuable method for assessing nasal obstruction in patients with severe CRSwNP. The degree of nasal flow impairment at baseline does not impact dupilumab's efficacy. A graphical abstract and video abstract are available for this article.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145172183","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}