Advances in Therapy最新文献

{"title":"Investigating the Pharmacokinetics, Injection Speed, and Usability of Subcutaneous Efgartigimod PH20 Administered with a Prefilled Syringe.","authors":"Cassandra De Muynck, Jan Noukens, Koen Allosery, Clotilde Cettour-Rose, Ségolène Pastouret, Anthony D Andre, Filip Borgions","doi":"10.1007/s12325-026-03733-x","DOIUrl":"https://doi.org/10.1007/s12325-026-03733-x","url":null,"abstract":"<p><strong>Introduction: </strong>Efgartigimod is a human immunoglobulin G1 (IgG) antibody Fc fragment that reduces levels of circulating IgG, including pathogenic autoantibodies, by blocking neonatal Fc receptor. The original 1000-mg fixed-dose formulation of efgartigimod coformulated with recombinant human hyaluronidase PH20 for subcutaneous injection (PH20 SC) is supplied in a single-dose vial and administered with a separate syringe (V + S). To increase patient convenience, a 5-ml prefilled syringe (PFS) of efgartigimod PH20 SC has been developed. These studies examined the pharmacokinetics, injection speed, and usability of the efgartigimod PH20 SC PFS.</p><p><strong>Methods: </strong>Bioequivalence of efgartigimod PH20 SC administered via PFS or V + S was assessed in an open-label study in healthy participants. Feasibility, safety, and tolerability of injecting efgartigimod PH20 SC at different injection speeds ranging from 20 to 60 s was examined in a separate open-label study in healthy participants. Finally, two human factor (HF) validation studies investigated usability of the efgartigimod PH20 SC PFS by participants with generalized myasthenia gravis (gMG) or chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and lay caregivers in a simulated at-home environment.</p><p><strong>Results: </strong>Pharmacokinetic bioequivalence was established between efgartigimod PH20 SC administered by PFS and V + S. No meaningful differences were observed between mean fluid leakage/backflow volume at the injection site between the injection speed groups, and no meaningful differences in tolerability across injection speeds occurred. All participants in the HF studies successfully used the efgartigimod PH20 SC PFS and associated instructional materials without training to deliver the full injection volume in an average of 30 s.</p><p><strong>Conclusion: </strong>These studies support the bioequivalence, safety, and feasibility of an efgartigimod PH20 SC PFS. Both participants with gMG or CIDP and lay caregivers successfully administered the full dose of the efgartigimod PH20 SC PFS. Rapid-administration efgartigimod PH20 SC PFS is safe and feasible for both patients with autoimmune diseases and lay caregivers.</p><p><strong>Trial identification number: </strong>ClinicalTrials.gov identifier: NCT05817435.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148872547","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":"Travoprost Intracameral Implant as a Standalone Procedure or Combined with Minimally Invasive Glaucoma Surgery (MIGS).","authors":"Arkadiy Yadgarov","doi":"10.1007/s12325-026-03778-y","DOIUrl":"https://doi.org/10.1007/s12325-026-03778-y","url":null,"abstract":"<p><strong>Introduction: </strong>The primary objective of this study was to report real-world outcomes of the travoprost intracameral implant (iDose TR) as standalone surgery or combined with minimally invasive glaucoma surgery (MIGS). The secondary objective was to compare efficacy across subgroups stratified by surgical category, severity, and race/ethnicity.</p><p><strong>Methods: </strong>This was a retrospective nonrandomized single-center consecutive case series of iDose TR implantations, with or without MIGS. Inclusion criteria were primary open-angle glaucoma, mild-to-severe stage, and minimum 3 months' follow-up. Eyes with visually significant cataract undergoing combined surgery also underwent concomitant phacoemulsification. Efficacy endpoints were mean and proportional analyses of IOP and medication burden (at baseline vs 3 months).</p><p><strong>Results: </strong>The study included 85 eyes. iDose TR was a standalone procedure in 40 eyes (47.1%) and combined with MIGS in 45 eyes (52.9%): iDose + iStent infinite (n = 22), iDose + iStent infinite + phacoemulsification (n = 16), and iDose + other MIGS ± phacoemulsification (n = 7). IOP and medication burden reduced significantly from 17.2 ± 5.1 and 1.9 ± 1.1 mmHg preoperatively to 14.5 ± 4.3 and 0.9 ± 0.9 mmHg at 3 months in all eyes (p < 0.001). Subgroup analyses revealed significant reductions in IOP and medication burden across all subgroups with no significant between-subgroup differences, except in the surgical category analysis which showed statistically significantly greater medication reduction after combined procedures than standalone procedures. There were no intraoperative complications. Postoperatively, IOP elevation (n = 1) and branch retinal vein occlusion (n = 1) were observed. No secondary glaucoma interventions were required.</p><p><strong>Conclusion: </strong>iDose TR achieved significant and safe IOP and medication reduction when performed as standalone surgery or combined with trabecular outflow-based MIGS with or without concomitant phacoemulsification. Greater medication-reducing effect was observed following combined iDose TR + MIGS surgery than with standalone iDose TR implantation. Study limitations include the short follow-up, nonrandomized design, and small, unmatched subgroups; further randomized studies with large, matched cohorts will be helpful to validate these findings.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148872632","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":"Meaningful Improvement Thresholds for Pruritus and Sleep Disturbance in PBC Using the Worst Itch and Sleep Interference Numerical Rating Scales.","authors":"Brooke M Currie, Katelyn Cutts, Ren Yu, Yipin Han, Gideon M Hirschfield, Cynthia Levy, Megan M McLaughlin, Marlyn J Mayo, David E Jones, Heather L Gelhorn","doi":"10.1007/s12325-026-03694-1","DOIUrl":"https://doi.org/10.1007/s12325-026-03694-1","url":null,"abstract":"<p><strong>Introduction: </strong>Cholestatic pruritus is common in primary biliary cholangitis (PBC), adversely affecting quality of life. This study aimed to establish the within-patient meaningful change threshold (MCT) for the Worst Itch Numerical Rating Scale (WI-NRS) in patients with PBC and pruritus. Determining MCTs for patient-reported outcomes is crucial for interpreting treatment efficacy in clinical trials. Given the high impact of pruritus on sleep in PBC, an MCT was also estimated for the Sleep Interference NRS (SI-NRS), which assesses pruritus-related sleep interference.</p><p><strong>Methods: </strong>Analyses were performed on blinded data from the Phase 3 GLISTEN study of linerixibat in patients with PBC and moderate-to-severe pruritus (NCT04950127). Anchor-based methods included the Patient Global Impression of Severity (PGI-S) and Change (PGI-C) as primary and supportive anchors, respectively. Correlations between weekly averages of daily WI-NRS and SI-NRS change scores and the anchors were assessed. A '1-point' improvement in PGI-S and 'moderately improved' on PGI-C were considered key reference groups for MCT determination. MCTs were evaluated in patients who reported changes in their itch and/or sleep were meaningful, as indicated by PGI-C item 2. Cumulative distribution function curves and distribution-based methods provided supporting evidence.</p><p><strong>Results: </strong>Data from 238 patients were included. Among patients reporting meaningful change in itch at Week 24, mean (standard deviation) change in WI-NRS was - 3.0 (1.7) for the '1-point' PGI-S improvers and - 2.9 (2.0) for the 'moderately improved' PGI-C group. Corresponding values for SI-NRS were - 2.6 (2.1) and - 2.4 (2.1). Distribution-based estimates for the change in WI-NRS and SI-NRS each ranged from 0.42 to 0.85.</p><p><strong>Conclusion: </strong>This study demonstrated that a reduction of ≥ 3 points in WI-NRS and ≥ 2.5 points in SI-NRS weekly scores represent clinically meaningful improvement thresholds for patients with PBC and moderate-to-severe pruritus. These results support use of WI-NRS and SI-NRS in future PBC trials evaluating new treatments for pruritus.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148857163","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":"Summary of Research: Brentuximab Vedotin and Nivolumab in Combination with Chemotherapy for Nonbulky, Early-Stage Classical Hodgkin Lymphoma.","authors":"Jeremy S Abramson, David J Straus, Nancy L Bartlett, John M Burke, Ryan C Lynch, Eva Domingo Domenech, Brian Hess, Steven R Schuster, Yuliya Linhares, Mitul Gandhi, Harsh R Shah, Wojciech Jurczak, Alessandro Re, Uwe Hahn, H Miles Prince, Wenchuan Guo, Griffith Davis, Linda Ho, Michelle Fanale, Christopher A Yasenchak, Hun Ju Lee","doi":"10.1007/s12325-026-03723-z","DOIUrl":"https://doi.org/10.1007/s12325-026-03723-z","url":null,"abstract":"<p><p>This is a summary of the original research article \"Brentuximab Vedotin and Nivolumab in Combination with Chemotherapy for Nonbulky, Early-Stage Classical Hodgkin Lymphoma.\" The phase 2 SGN35-027 study (NCT03646123) is a multiple-part clinical trial of brentuximab vedotin (BV), with nivolumab, doxorubicin, and dacarbazine (AN + AD), in classical Hodgkin lymphoma (cHL). Here, we present the efficacy and safety of AN + AD from part C of this study in patients with nonbulky, early-stage cHL. At the time of this analysis, 154 patients had received ≥ 1 dose of AN + AD and 98% had received all 4 cycles of treatment. The objective response rate at end of treatment was 96%, and complete response rate was 92% (95% for the favorable subgroup; 91% for the unfavorable subgroup). The proportion of patients with complete response lasting ≥ 2 years was 96%. At a median follow-up of 27.9 months, the estimated 2-year progression-free survival rate was 97%. Any-grade and grade ≥ 3 treatment-related side effects occurred in 97% and 34% of patients, respectively. No events of febrile neutropenia were reported. Any-grade treatment-emergent immune-mediated adverse events occurred in 22% of patients. Collectively, results from this study support the use of BV and nivolumab in combination with limited chemotherapy in patients with nonbulky, early-stage cHL.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148857106","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":"Technology and Obesity: A Year in Review.","authors":"Shinjan Patra, Akhila Bhandarkar, Nitin Kapoor, Sanjay Kalra","doi":"10.1007/s12325-026-03775-1","DOIUrl":"https://doi.org/10.1007/s12325-026-03775-1","url":null,"abstract":"<p><p>SMART technological advancements help diagnose, treat, and monitor various diseases at the earliest stages. It presents an opportunity to maintain the key components of conventional obesity management programming while reducing costs and provider time inputs. Various machine learning models have helped predict the risks of obesity and metabolic syndrome. Additionally trained convolutional neural networks can now automatically segment and quantify different adipose tissue compartments. Various multicenter series and randomized clinical trials showed clinically meaningful total and excess weight loss at 6-24 months after minimally invasive robotic bariatric surgical procedures with an acceptable safety profile and shorter recovery compared with surgical sleeve gastrectomy. Virtual reality cue exposure therapy (VR-CET), a Website interactive voice response system, and various mobile applications for tracking diet and activity are the most advanced technological uses in the field of obesity management. The technological advancements should be adaptable, affordable, accessible, accurate, intuitive, sustainable and scalable. The conventional face-to-face interactions in obesity management have yielded short-term dividends, whereas technology-driven methods have the potential to produce long-lasting effects. This narrative review presented the different types of technologies used for obesity management and their outcomes in recent times.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148857150","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":"Effectiveness and Safety of Trastuzumab Deruxtecan in Chinese Patients with HER2-Mutant Metastatic Non-Small Cell Lung Cancer (RERUN): Study Protocol for a Real-World, Multicenter, Prospective, Observational Study.","authors":"Jianchun Duan, Minglei Zhuo, Chunxia Su, Yibing Liu, Lifeng Wang, Nong Yang, Baoshan Cao, Mingming Hu, Shi Jin, Hui Li, Xiaoyan Li, Yueyin Pan, Jie Hu, Degan Lu, Chuanhao Tang, Bin Yang, Yunjian Huang, Guanming Jiang, Wen Li, Sujie Ni, Wang Sheng, Kejing Tang, Jing Wang, Qiming Wang, Zhijie Wang, Si Si, Jie Wang","doi":"10.1007/s12325-026-03729-7","DOIUrl":"https://doi.org/10.1007/s12325-026-03729-7","url":null,"abstract":"<p><strong>Introduction: </strong>Trastuzumab deruxtecan (T-DXd) is a human epidermal growth factor receptor 2 (HER2)-targeting antibody-drug conjugate that has demonstrated encouraging efficacy and a manageable safety profile in the second-line or later setting for non-small cell lung cancer (NSCLC) harboring HER2 mutations. While T-DXd was approved for treating patients with locally advanced or metastatic HER2-mutant NSCLC in China, real-world data on its use in Chinese clinical practice are lacking. This study will collect real-world data on T-DXd to evaluate its effectiveness and safety in Chinese patients with HER2-mutant metastatic NSCLC, thereby providing additional evidence to the oncology community in China.</p><p><strong>Methods: </strong>RERUN is a prospective, multicenter, observational cohort study conducted at approximately 30 sites in China. Approximately 150 adult patients (≥ 18 years) with pathologically documented unresectable and/or metastatic non-squamous NSCLC harboring any known activating HER2 mutation are currently being enrolled. The follow-up period will last approximately 6 months after the last patient is enrolled, when sufficient progression-free survival (PFS) maturity (approximately 60% of patients with events) is expected to be reached. All data will be prospectively collected and analyzed descriptively.</p><p><strong>Planned outcomes: </strong>The primary outcome is real-world PFS assessed by investigators in patients receiving T-DXd as second-line or later therapy. Secondary outcomes include the time to treatment discontinuation or death, best overall response rate, overall survival, and safety and tolerability. This study will support treatment decision-making in routine oncology practice and improve outcomes for Chinese patients with HER2-mutant NSCLC across diverse healthcare settings.</p><p><strong>Trial registration: </strong>Study registration number NCT06809764.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148856997","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 Use of Abrocitinib for Moderate-to-Severe Atopic Dermatitis in the United States Based on Electronic Health Records and Administrative Claims.","authors":"Raj Chovatiya, Amanda Mummert, Stella Chang, Mark Levenberg, Griffith Bell, Amie Scott, Melissa Watkins","doi":"10.1007/s12325-026-03761-7","DOIUrl":"https://doi.org/10.1007/s12325-026-03761-7","url":null,"abstract":"<p><strong>Introduction: </strong>Abrocitinib is approved for moderate-to-severe atopic dermatitis (AD) treatment. Real-world abrocitinib usage data are limited. The aim of this study was to characterize patients initiating abrocitinib in clinical settings in the United States.</p><p><strong>Methods: </strong>Retrospective, observational study of patients aged ≥ 12 years with AD using linked claims (index date: abrocitinib initiation) and electronic health records in the OMNY Health real-world data platform (January 1, 2017-October 31, 2025). Baseline demographics, characteristics, comorbidities, medication history, and healthcare resource utilization were evaluated. Abrocitinib dose changes ≤ 12 months post-index, early persistence (prescription refill within 60 days of index), and AD severity changes from baseline were assessed.</p><p><strong>Results: </strong>Overall, 401 patients initiated abrocitinib (8.5% adolescent; 91.5% adult). Mean time since diagnosis was 4.2 years. Most patients (72.1%) received abrocitinib as their first advanced systemic therapy and the most common starting dose was 100 mg (81.8%). The most frequent pre-index AD therapy overall was topical treatment (72.6%). Within 12 months post-index, 90.8% of patients remained on the starting dose and 60.8% demonstrated early persistence.</p><p><strong>Conclusion: </strong>Most patients with moderate-to-severe AD prescribed abrocitinib receive 100 mg as the starting dose, refill their prescription within 60 days of index, and maintained their starting dose. Limitations include incomplete patient data and lack of generalizability to other settings.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148857084","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 Retrospective, Observational Study of Adherence, Persistence and Clinical Outcomes with Perindopril-Based Single-Pill or Free-Pill Antihypertensive Treatments in Italy.","authors":"Ana Maria Vintila, Stefano Masi, Luca Degli Esposti, Melania Dovizio, Julien Magne, Marta Nugnes, Chiara Veronesi, Jacek Wolf, Gianluigi Savarese","doi":"10.1007/s12325-026-03765-3","DOIUrl":"https://doi.org/10.1007/s12325-026-03765-3","url":null,"abstract":"<p><strong>Introduction: </strong>Poor adherence limits the effectiveness of pharmacological treatments. Single-pill combinations (SPCs) improve adherence in short-term observational studies, but whether advantages are sustained long term and translate into clinical benefits remains uncertain. We evaluated long-term improvements in adherence, persistence, and clinical outcomes associated with SPCs.</p><p><strong>Methods: </strong>Using patient record linking across Italian administrative healthcare databases, we performed a non-interventional, retrospective, observational, longitudinal analysis of hypertension treated with perindopril (PER)-based combinations. We compared adherence [proportion of days covered (PDC)], treatment discontinuation, all-cause mortality, and cardiovascular (CV) event rates between SPCs and free-pill combinations (FPCs) over 10 years. Being the first introduced in Italy, PER-based SPCs were chosen to allow the longest follow-up.</p><p><strong>Results: </strong>Between 2010 and 2022, 24,121 patients with hypertension were treated with PER-based SPCs (n = 22,663) or FPCs (n = 1458). Throughout the 10-year period, ≥ 73% of SPC users had high adherence (PDC ≥ 80%), compared with ≤ 44% of FPC users. Over a 3-year follow-up, discontinuation rates were lower with SPCs (20%) than FPCs (50%). SPCs were associated with lower risks of all-cause mortality (- 27%), CV events (- 31%), ischaemic heart disease (IHD) (- 22%), cerebrovascular events (- 46%), and a composite of mortality/CV events (- 29%) compared with FPCs. Compared with patients with low adherence, those with high adherence had lower risks of all-cause mortality (- 22%), CV events (- 21%), IHD (- 17%), cerebrovascular events (- 33%), and the composite of mortality/CV events (- 22%).</p><p><strong>Conclusion: </strong>SPCs are associated with sustained, better adherence and persistence to antihypertensive pharmacological treatments compared with FPCs, which may contribute to real-world clinical benefits. Graphical abstract available for this article.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148823663","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":"Cost Per Remission of Vedolizumab Versus Risankizumab for the Treatment of Moderately to Severely Active Crohn's Disease in Italy.","authors":"Elizabeth Brook, Chloë Hardern, Jakob Langer, Raffaella Viti, Arnaud Nucit","doi":"10.1007/s12325-026-03665-6","DOIUrl":"https://doi.org/10.1007/s12325-026-03665-6","url":null,"abstract":"<p><strong>Introduction: </strong>Crohn's disease (CD) involves chronic gastrointestinal inflammation and requires lifelong treatment, creating an economic burden for patients and healthcare systems. Approved treatment options for CD in Italy include vedolizumab (VDZ) and risankizumab (RZB). In this analysis, a cost-per-responder model was used to evaluate the comparative value of VDZ and RZB in inducing disease remission after 1 year of treatment.</p><p><strong>Methods: </strong>A cost-per-responder model assessed the cost per patient to achieve clinical remission after 1 year of treatment. Efficacy inputs were obtained from a previously reported matching-adjusted indirect comparison of VDZ versus RZB. Costs included drug acquisition and administration in Italy. Sensitivity analyses assessed the robustness of the results to variations in efficacy rates, efficacy outcomes, and acquisition costs.</p><p><strong>Results: </strong>Cost-per-responder analysis showed that the cost per remission for patients with CD was lower for VDZ than for RZB over 1 year (€114,098 vs. €285,939; - 60.1%). A sensitivity analysis varying clinical remission rates by ± 25% showed that VDZ maintained a lower cost per remission, even when comparing simultaneous decreased VDZ and increased RZB remission rates (€152,130 vs. €228,751). Similarly, the cost per clinical response was also lower for VDZ after 1 year of treatment (€182,987 vs. €245,696), despite RZB exhibiting a numerically higher clinical response rate. Price sensitivity analysis showed that even when the acquisition costs of both RZB and VDZ were substantially discounted, VDZ consistently provided a lower cost per remission.</p><p><strong>Conclusions: </strong>In Italy, VDZ may represent a more efficient use of healthcare resources than RZB to achieve clinical remission in patients with CD. Graphical abstract available for this article https://doi.org/10.6084/m9.figshare.32456697 .</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148817205","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":"Physician and Patient Perception of Disease, Symptomatology, and Treatment Administration Preference in Paroxysmal Nocturnal Haemoglobinuria: Results from a Real-World Survey.","authors":"Bing Han, Maria-Magdalena Balp, Josefin Snellman, Anggie Wiyani, Gifty Cobby Amporful, Niall Hatchell, Yasmin Taylor, Sarah Weatherby, Scarlett Che, Jordi Cruz, Talha Munir","doi":"10.1007/s12325-026-03749-3","DOIUrl":"https://doi.org/10.1007/s12325-026-03749-3","url":null,"abstract":"<p><strong>Introduction: </strong>Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare condition, characterised by complement-mediated red blood cell breakdown, resulting in haemolytic anaemia. Associated symptoms, such as fatigue, impact on patient quality of life. Little is known about the comparative perceptions of symptom burden and treatment preferences among patients and physicians.</p><p><strong>Methods: </strong>This study analysed secondary data from the Adelphi Real World PNH Disease Specific Programme™, a cross-sectional survey of physicians and patients with PNH in France, Germany, Italy, Spain, China, and Japan conducted January-November 2022. Haematologists and haematologist-oncologists reported demographic and clinical data for ≤10 consecutive consulting adult patients with PNH. Only patients with both physician-reported and patient-reported data were included in the study. Patients and physicians reported on clinical signs and symptom burden, treatment satisfaction, and preferred route of administration. Bivariate analyses were used to compare physician and patient responses.</p><p><strong>Results: </strong>Forty-two physicians reported data for 225 patients. Median (interquartile range) patient age was 44.0 (35.0-56.0), 56.9% were male and 96.0% received treatment at survey. Most common clinical signs and symptoms were fatigue, anaemia, haemoglobinuria, dyspnoea, and headaches. All common signs/symptoms, except haemoglobinuria, were reported as significantly more frequent and severe by patients than physicians (p < 0.05). Most bothersome signs/symptoms reported were anaemia (physicians: 31.3%, patients: 21.3%) and fatigue (physicians: 29.9%, patients: 45.5%). Physicians were significantly more satisfied with treatment than patients (p < 0.001), regardless of treatment. Although oral administration was preferred by both physicians (60.2%) and patients (74.2%), there was significant difference in overall preference (p < 0.001), with 30.8% of physicians preferring intravenous administration, compared to 16.3% of patients.</p><p><strong>Conclusion: </strong>There was significant patient-physician disconnect in perception of PNH symptoms burden, treatment satisfaction, and administration preferences. Improved communication and patient involvement in disease management may contribute to optimised patient outcomes and quality of life.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148817207","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}