Advances in Therapy最新文献

{"title":"Cycling to TNFi vs. Switching to IL-17Ai Among Patients with Psoriatic Arthritis and Axial Spondyloarthritis: Real-World CorEvitas PsA/SpA Registry Data","authors":"Alexis Ogdie,&nbsp;Nicole Middaugh,&nbsp;Maya Marchese,&nbsp;Jessica A. Walsh,&nbsp;Natalia Bello,&nbsp;Marcus Ngantcha,&nbsp;Andris Kronbergs,&nbsp;Elsie L. Grace,&nbsp;Philip J. Mease","doi":"10.1007/s12325-026-03531-5","DOIUrl":"10.1007/s12325-026-03531-5","url":null,"abstract":"<div><h3>Introduction</h3><p>Tumor necrosis factor inhibitors (TNFis) are a common first-line treatment option for patients with psoriatic arthritis (PsA) or axial spondyloarthritis (axSpA) with persistent disease activity despite conventional treatment. There is limited real-world evidence available to inform selection of therapy after first TNFi failure.</p><h3>Methods</h3><p>This study compares the effectiveness of cycling to a second TNFi versus switching to an interleukin-17A inhibitor (IL-17Ai) at 6-month follow-up, among patients with PsA and axSpA who discontinued a first TNFi. Patients were stratified into two groups: those who (1) initiated a second TNFi (cyclers) and (2) initiated an IL-17Ai (switchers). Inverse probability of treatment weighting (IPTW) was used to account for baseline differences between cyclers and switchers. Separate models were fit for the PsA and axSpA cohorts.</p><h3>Results</h3><p>The PsA cohort included 277 (59%) cyclers and 194 (41%) switchers. Prior to IPTW, switchers demonstrated greater improvement (− 4.1 [95% confidence interval (CI) − 5.6, − 2.5]) in the primary outcome of change from baseline (CFB) in the Clinical Disease Activity Index for Psoriatic Arthritis (cDAPSA) versus cyclers. Post IPTW, switchers showed greater improvements across most secondary outcomes, and significantly greater improvements in Physician Global Assessment (PhGA) of arthritis and psoriasis (− 5.0 [95% CI − 9.3, − 0.8], <i>p</i> = 0.021) and non-work activity impairment (− 4.7 [95% CI − 9.2, − 0.2], <i>p</i> = 0.039).</p><p>The axSpA cohort included 119 (69%) cyclers and 53 (31%) switchers. Prior to IPTW, switchers demonstrated greater improvement (− 0.7 [95% CI − 1.2, − 0.2]) in the primary outcome of CFB in Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) versus cyclers. Post IPTW, switchers showed significantly greater improvement in patient fatigue (− 9.4 [95% CI − 15.8, − 3.1], <i>p</i> = 0.004) and numerically greater improvements across remaining secondary outcomes.</p><h3>Conclusion</h3><p>Switching to IL-17Ai vs. cycling to another TNFi provided similar or numerically improved outcomes in both cohorts, underscoring the importance of considering alternative mechanisms of action for these patients.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2139 - 2162"},"PeriodicalIF":4.0,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03531-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442071","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":"Safety and Effectiveness of Lisdexamfetamine Dimesylate in Children and Adolescents with ADHD in Japan: An Interim Analysis of Post-Marketing Surveillance","authors":"Takashi Okada,&nbsp;Hiroyuki Ida,&nbsp;Kouji Yamashita,&nbsp;Kazuhiko Saito","doi":"10.1007/s12325-025-03459-2","DOIUrl":"10.1007/s12325-025-03459-2","url":null,"abstract":"<div><h3>Introduction</h3><p>This post-marketing surveillance study investigated safety and effectiveness of lisdexamfetamine dimesylate (LDX) in paediatric attention-deficit/hyperactivity disorder (ADHD) in routine clinical practice in Japan.</p><h3>Methods</h3><p>Patients (aged 6 to &lt; 18 years) were enrolled in this prospective, observational study from January 2020 to December 2023 and received an LDX dose determined by the investigator. Safety (including dependence and abuse) and effectiveness were evaluated for 2 years after treatment initiation. Dependence and abuse were also assessed for 3 additional years in those still receiving LDX. The data cut-off date for this interim analysis was 21 January 2024.</p><h3>Results</h3><p>Overall, 1819 patients were enrolled across 188 sites and case report forms were collected for 1012 patients. Of these, 1006 and 916 patients were included in the safety and effectiveness analyses, respectively. The adverse drug reactions (ADRs) of decreased appetite (19.3%), insomnia (3.7%), nausea (3.4%), weight decrease (2.8%), headache (2.6%), aggression (2.4%), initial insomnia (1.8%), irritability (1.8%), tic (1.6%), and agitation (1.3%), were reported in ≥ 1% of patients. Eight serious ADRs occurred in five patients: decreased appetite (<i>n</i> = 3), and anger, hyperthyroidism, palpitations, poor weight gain, and suicide attempt (<i>n</i> = 1 each). For ADRs defined as important identified risks or important potential risks of LDX, one non-serious syncope event was observed. No dependence- or abuse-related events of LDX were reported. An increasing proportion of patients had improvements in the Clinical Global Impression–Improvement scale during the first year of observation, and improvements were maintained through the second year. Improvements from baseline in ADHD-Rating Scale-IV total and subscale scores increased over the 2-year observation period; significant improvements in the total score were observed at all time points (<i>p</i> &lt; 0.0001).</p><h3>Conclusions</h3><p>No ADRs requiring new safety measures were observed. The effectiveness of LDX was confirmed in real-world practice. This study is ongoing, and the final results will be reported upon completion.</p><h3>Trial Registration</h3><p>University Hospital Medical Information Network (UMIN) Clinical Trials Registry (identifier: UMIN000039018).</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2163 - 2181"},"PeriodicalIF":4.0,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03459-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442062","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":"Indirect Comparison of Nipocalimab Versus Efgartigimod and Rozanolixizumab in the Treatment of Generalized Myasthenia Gravis","authors":"Saiju Jacob,&nbsp;Mahmoud Hashim,&nbsp;Brian Hutton,&nbsp;Kavita Gandhi,&nbsp;Suzy Van Sanden,&nbsp;Rafal Slowik,&nbsp;Christopher Drudge,&nbsp;Antoine C. El Khoury,&nbsp;Mi Jun Keng,&nbsp;Sumeet Singh,&nbsp;Sindhu Ramchandren,&nbsp;Nils Erik Gilhus","doi":"10.1007/s12325-026-03543-1","DOIUrl":"10.1007/s12325-026-03543-1","url":null,"abstract":"<div><h3>Introduction</h3><p>Nipocalimab, efgartigimod, and rozanolixizumab (the last two cyclically dosed) are approved neonatal Fc receptor (FcRn) blockers for treating generalized myasthenia gravis (gMG). No trials have directly compared these therapies; hence, indirect treatment comparisons (ITCs) were conducted to evaluate their relative efficacy.</p><h3>Methods</h3><p>Matching-adjusted indirect comparisons (MAICs) and Bucher ITCs were used to compare nipocalimab vs. efgartigimod and rozanolixizumab for changes from baseline (CFB) in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score observed in their phase 3 registration trials. Bucher ITCs used the relative treatment effect vs. placebo. As there was considerable cross-trial heterogeneity, including uncertainty in using the placebo arm as a common comparator, active treatment arms were used in unanchored MAICs. MG-ADL CFB was compared between trials (1) at multiple timepoints to evaluate onset of action and disease control over time, and (2) using area under the curve (AUC) as a measure of cumulative effect normalized per week of follow-up.</p><h3>Results</h3><p>In both Bucher ITCs and MAICs, nipocalimab had a comparable MG-ADL CFB at week 1 vs. the other FcRn blockers. In MAICs, MG-ADL CFB was significantly greater with nipocalimab vs. efgartigimod at week 8 sustained up to 24 weeks (<i>p</i> &lt; 0.05), and vs. rozanolixizumab at week 10 sustained up to 14 weeks (<i>p</i> &lt; 0.05); results numerically favored nipocalimab in corresponding Bucher ITCs. Using normalized AUC, MG-ADL CFB with nipocalimab was significantly greater in MAICs (<i>p</i> &lt; 0.05) and numerically greater in Bucher ITCs vs. the other FcRn blockers.</p><h3>Conclusions</h3><p>Sustained disease control is an important consideration in managing chronic diseases with fluctuating symptoms such as gMG. Study results showed that nipocalimab provided a comparable onset of action and consistent and sustained disease control that was numerically or statistically significantly greater (depending on ITC method) when compared with the symptom-based cyclic FcRn blockers efgartigimod and rozanolixizumab.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2125 - 2138"},"PeriodicalIF":4.0,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03543-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442088","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: Assessing the Value Contribution of Vyvgart® (Efgartigimod Alfa) in the Treatment of Generalized Myasthenia Gravis with Acetylcholine Receptor Antibody in Spain Through Multi-criteria Decision Analysis","authors":"Elena Cortés-Vicente,&nbsp;Antonio Guerrero,&nbsp;Carmina Díaz,&nbsp;Eva Martínez,&nbsp;Francisco J. Toja-Camba,&nbsp;María R. Abad,&nbsp;José M. Serra,&nbsp;Jose L. Trillo,&nbsp;Celia Martín Machín,&nbsp;Alicia Gil","doi":"10.1007/s12325-026-03532-4","DOIUrl":"10.1007/s12325-026-03532-4","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2317 - 2318"},"PeriodicalIF":4.0,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03532-4.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442133","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":"Long-Term Treatment with Single-Tablet Combination of Macitentan and Tadalafil in Pulmonary Arterial Hypertension: Results from A DUE and Its Open-Label Period","authors":"H. James Ford,&nbsp;Kelly M. Chin,&nbsp;Fenling Fan,&nbsp;Michael Friberg,&nbsp;Ekkehard Grünig,&nbsp;Jakob A. Hauser,&nbsp;Matthieu Pannaux,&nbsp;Hany Rofael,&nbsp;Pavel Jansa","doi":"10.1007/s12325-026-03525-3","DOIUrl":"10.1007/s12325-026-03525-3","url":null,"abstract":"<div><h3>Introduction</h3><p>In the A DUE study, a fixed-dose combination of macitentan 10 mg and tadalafil 40 mg (M/T FDC) as a single tablet significantly improved pulmonary vascular resistance at Week 16 versus corresponding monotherapies in patients with pulmonary arterial hypertension (PAH). Safety was consistent with known profiles of macitentan and tadalafil. The open-label (OL) period of A DUE provides long-term safety/efficacy data for M/T FDC.</p><h3>Methods</h3><p>In A DUE (NCT03904693), patients were randomized (2:1:1) to double-blind M/T FDC, macitentan 10 mg or tadalafil 40 mg and followed for 16 weeks. They then transitioned to OL M/T FDC and were followed for up to 2 years to end of study (EOS). Efficacy analyses, including survival, changes in six-minute walk distance (6MWD) and N-terminal pro-brain natriuretic peptide (NT-proBNP) from baseline, are reported in patients randomized to M/T FDC at start of A DUE (efficacy set). Safety is reported for all patients receiving M/T FDC at any time during the double-blind (DB) and/or OL (safety set).</p><h3>Results</h3><p>In A DUE, 185 patients received M/T FDC for median (range) of 105.1 (0.6, 182.6) weeks. In the efficacy set, 91% of patients were alive at EOS. Mean (SD) change in 6MWD from M/T FDC initiation to OL Week 120 was 60.5 m (84.2). For NT-proBNP, geometric mean percent of baseline was 50.2% at OL Week 120. In the safety set, adverse events (AEs) occurred in 94.1% and serious AEs in 33.5% of patients; 10.3% discontinued study treatment due to an AE. Seven on-treatment deaths occurred in those receiving M/T FDC; all were evaluated as unrelated to treatment.</p><h3>Conclusions</h3><p>Long-term treatment with single-tablet combination of macitentan/tadalafil was well tolerated, with no new safety findings identified. Most patients were alive at EOS. Incremental improvements in 6MWD and NT-proBNP observed in the DB with M/T FDC were sustained over 2 years.</p><h3>Trial Registration</h3><p>ClinicalTrials.gov Identifier NCT03904693.</p><p>A graphical abstract is also available for this article.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2106 - 2124"},"PeriodicalIF":4.0,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03525-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442067","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":"Budget Impact of Venetoclax for Newly Diagnosed Patients with Acute Myeloid Leukemia Aged ≥ 75 Years or with Comorbidities Precluding Intensive Chemotherapy in the United States","authors":"Yanqing Xu,&nbsp;Janet Nguyen,&nbsp;Kaylee Miu,&nbsp;Chia-Wei Lin,&nbsp;Melissa Montez,&nbsp;Xinglei Chai,&nbsp;Xin Chen,&nbsp;Thomas W. LeBlanc","doi":"10.1007/s12325-026-03542-2","DOIUrl":"10.1007/s12325-026-03542-2","url":null,"abstract":"<div><h3>Introduction</h3><p>Venetoclax plus azacitidine or decitabine is approved in the US for treatment of newly diagnosed patients with acute myeloid leukemia (AML) aged ≥ 75 years or who have comorbidities precluding use of intensive chemotherapy. As novel targeted regimens expand treatment options for AML, this study evaluated the budget impact of adopting venetoclax combinations for this population from a US third-party payer perspective to inform affordability and access at the health plan level.</p><h3>Methods</h3><p>The model estimated the 3-year budget impact of adopting venetoclax combinations in a hypothetical US health plan with 1 million members (60% commercial, 40% Medicare). Eligible patients were estimated using public data. Market share projections assumed venetoclax + azacitidine or decitabine captured a 53% and 15% share, respectively, from existing treatments (azacitidine, low-dose cytarabine [LDAC], decitabine, ivosidenib, gemtuzumab ozogamicin, glasdegib + LDAC, and ivosidenib + azacitidine). The model considered costs associated with drug acquisition/administration, adverse events, hospitalization, disease monitoring, blood transfusions, and subsequent AML management (2024 USD). Clinical inputs for venetoclax combinations were informed by final VIALE-A and M14-358 data, respectively. Incremental budget impact was calculated as per-member-per-month (PMPM), with one-way sensitivity analyses performed.</p><h3>Results</h3><p>In a 1-million-member health plan, 48 patients were eligible for venetoclax combinations. Annual costs of venetoclax + azacitidine ($258,498) or decitabine ($259,921) were lower than those of the highest-cost comparators, ivosidenib + azacitidine ($477,520) and ivosidenib ($404,869). Drug acquisition costs of adopting venetoclax combinations were offset by lower subsequent AML management costs, resulting in savings of $0.0476 PMPM in years 1–3. Results remained robust in sensitivity analyses. In a 100% Medicare scenario, 117 patients were eligible for venetoclax combinations, with savings of $0.1137 PMPM over years 1–3.</p><h3>Conclusion</h3><p>Inclusion of venetoclax combinations for newly diagnosed patients with AML aged ≥ 75 years or with comorbidities precluding intensive chemotherapy reduced the budget impact, providing potential financial benefits for US payers.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2182 - 2201"},"PeriodicalIF":4.0,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03542-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442151","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":"The PKD Daily: Development and Content Validation of a Hybrid Diary for Tracking Urinary Events in Adults with Autosomal Dominant Polycystic Kidney Disease (ADPKD)","authors":"Meaghan O’Connor,&nbsp;Lynne Broderick,&nbsp;Laura Tesler Waldman,&nbsp;Elizabeth Costa,&nbsp;Michelle Carty,&nbsp;Sasikiran Nunna,&nbsp;Dorothee Oberdhan","doi":"10.1007/s12325-026-03528-0","DOIUrl":"10.1007/s12325-026-03528-0","url":null,"abstract":"<div><h3>Introduction</h3><p>For patients with autosomal dominant polycystic kidney disease (ADPKD), the only approved treatment is tolvaptan, a twice-daily medication that works to slow kidney function decline. Patients on tolvaptan report a considerable burden related to the drug’s aquaretic effects which can intensify immediately following a dose of the medication.</p><h3>Methods</h3><p>A targeted literature review and individual concept elicitation (CE) interviews with nephrologists and adult patients with ADPKD taking tolvaptan were conducted to inform the development of a hybrid diary, programmed as a smartphone application. A sample of adults with ADPKD taking tolvaptan tested the diary for 7 days; a subset also participated in cognitive debriefing (CD) interviews. The diary was then revised and finalized.</p><h3>Results</h3><p>CE interviews confirmed the importance of tracking urinary frequency and urgency and timing of tolvaptan doses in real time. Results from the usability/feasibility test and CD interviews confirmed the understandability and relevance of the diary and provided critical insights to improve its content and functionality. The resulting PKD Daily includes two daily entries to report dose timing, overnight urination, and daily impacts of frequent/urgent urination; real-time entries to capture frequency/urgency of daytime urination; and reminders to encourage regular data entry.</p><h3>Conclusion</h3><p>Originally developed for use in a clinical trial, the PKD Daily can also provide valuable information to clinicians and patients to support treatment decisions and inform dose selection in clinical practice, becoming a valuable part of a clinician’s toolbox. Further, the development of the PKD Daily can serve as a case study for the successful creation of a hybrid diary.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2054 - 2070"},"PeriodicalIF":4.0,"publicationDate":"2026-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03528-0.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147430114","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":"Screening for Pulmonary Hypertension in Interstitial Lung Disease: Preliminary Results from the PHINDER Study","authors":"David Zisman,&nbsp;Sandeep Sahay,&nbsp;Debabrata Bandyopadhyay,&nbsp;Amro Al-Astal,&nbsp;Meredith Broderick,&nbsp;Danielle Caudell Stamper,&nbsp;Hunter Champion,&nbsp;Maral DerSarkissian,&nbsp;Matthew Hunsucker,&nbsp;Dasom Lee,&nbsp;Kevin Maher,&nbsp;Andrew Nelsen,&nbsp;Raj Parikh,&nbsp;Franck Rahaghi,&nbsp;Abhijit Raval,&nbsp;Claire M. Thrasher,&nbsp;Tejaswini Kulkarni,&nbsp;Oksana A. Shlobin,&nbsp;David G. Kiely,&nbsp;Steven Nathan,&nbsp;Mary Beth Scholand","doi":"10.1007/s12325-026-03508-4","DOIUrl":"10.1007/s12325-026-03508-4","url":null,"abstract":"<div><h3>Introduction</h3><p>Interstitial lung disease (ILD) is frequently complicated by pulmonary hypertension (PH) resulting in reduced functional capacity, diminished quality of life, and increased mortality. However, standardized screening for PH in ILD is lacking, causing delays in diagnosis and treatment. PHINDER (NCT05776225) is a prospective multicenter study that aims to identify parameters for the detection of PH in ILD.</p><h3>Methods</h3><p>Data were collected prospectively in patients with ILD from predefined routine testing, including clinical, physiological, and imaging assessments. Precapillary PH was defined as mean pulmonary arterial pressure &gt; 20 mmHg, pulmonary artery wedge pressure ≤ 15 mmHg, and pulmonary vascular resistance (PVR) &gt; 2 Wood units (WU). Investigators estimated probability of precapillary PH based on noninvasive evaluations before confirmation by right heart catheterization (RHC).</p><h3>Results</h3><p>Preliminary results included 190 participants; 105 (55%) had precapillary PH and 26 (14%) had severe PH (PVR &gt; 5 WU). Notable parameters associated with precapillary PH included supplemental oxygen use (OR 3.6, <i>p</i> = 0.004), diffusing capacity of the lung for carbon monoxide ([DLCO] OR 0.9, <i>p</i> = 0.005), forced vital capacity % to DLCO % ratio (OR 1.1, <i>p</i> = 0.008), tricuspid annular plane systolic excursion to right ventricular systolic pressure ratio (OR 0.8, <i>p</i> = 0.020), tricuspid regurgitant velocity (OR 4.4, <i>p</i> = 0.006), pulmonary artery (PA) enlargement (OR 10.6, <i>p</i> &lt; 0.001), PA/aorta diameter ratio (OR 1.7, <i>p</i> = 0.004), and right to left ventricle diameter ratio (OR 1.5, <i>p</i> = 0.021). There was a trend toward higher likelihood of PH with higher clinician suspicion of PH before RHC, but gestalt-based assessment showed limited accuracy relative to hemodynamic confirmation (positive predictive value, 59%; negative predictive value, 68%; accuracy, 60%).</p><h3>Conclusions</h3><p>Preliminary findings support the composite use of pulmonary function testing, lung imaging, and echocardiography to improve early detection of precapillary PH in ILD and guide structured screening strategies. The final data set from PHINDER will provide guidance on thresholds for continuous variables with application in diagnosing PH in ILD, facilitating the development of a validated evidence-based screening tool to aid the detection of PH in ILD.</p><h3>Trail Registration</h3><p>NCT05776225.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2071 - 2089"},"PeriodicalIF":4.0,"publicationDate":"2026-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03508-4.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147430016","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":"Treatment Patterns and Clinical Outcomes in Metastatic HR+/HER2− and Triple-Negative Breast Cancer in Canada: The HER2− TRENDS Study","authors":"Karen Gambaro,&nbsp;Kahina Rachedi,&nbsp;Mark Basik,&nbsp;Gerald Batist,&nbsp;Fred Saad,&nbsp;Saima Hassan,&nbsp;Anne-Marie Mes-Masson,&nbsp;Dominique Boudreau,&nbsp;Francois Vincent,&nbsp;Eve St-Hilaire,&nbsp;Helen Mackay,&nbsp;Mahmoud Abdelsalam,&nbsp;Steven M. Yip,&nbsp;Robert Hanel,&nbsp;Simran Shokar,&nbsp;Zhor Senhaji Mouhri,&nbsp;Matthew Badin,&nbsp;Kristoph Klein-Panneton,&nbsp;Arif Ali Awan,&nbsp;Maud Marques","doi":"10.1007/s12325-026-03545-z","DOIUrl":"10.1007/s12325-026-03545-z","url":null,"abstract":"<div><h3>Introduction</h3><p>The treatment landscape for human epidermal growth factor receptor 2-negative (HER2−) metastatic breast cancer (mBC) in Canada is rapidly evolving. This retrospective cohort study described real-world historical treatment patterns and clinical outcomes for patients with HER2− mBC.</p><h3>Methods</h3><p>Adults enrolled in the pan-Canadian ‘Personalize My Treatment’ cancer registry and diagnosed with stage IV HER2− mBC (01/01/2015–03/01/2022) were eligible and followed until 03/01/2023. Hormone receptor-positive (HR+)/HER2− mBC and triple-negative mBC (mTNBC) were analyzed separately and further stratified by line of therapy. Main study outcomes included treatment patterns, <i>PIK3CA</i>/<i>AKT1</i>/<i>PTEN</i> alteration testing/positivity rates, and overall survival (OS), all analyzed descriptively.</p><h3>Results</h3><p>A total of 507 patients with HER2− mBC were included (HR+/HER2− mBC: 387; mTNBC: 120; median follow-up: 54.1 months). The most common HR+/HER2− mBC treatments were cyclin‑dependent kinase 4/6 inhibitors (CDK4/6is) plus endocrine therapy (ET) in first line (1L; 55.0%), targeted therapies (22.9%) and CDK4/6i + ET (22.0%) in second line (2L), and chemotherapy (CT) monotherapy (42.7%) in third line (3L). CT monotherapy was the most common mTNBC treatment in 1L (35.1%), 2L (50.6%), and 3L (54.8%). Attrition was similar for HR+/HER2− mBC and mTNBC from 1L–2L (16.9% and 16.3%, respectively) but was lower for HR+/HER2− mBC from 2L–3L (33.2% and 38.6%) and 3L–4L (48.1% and 62.1%). <i>PIK3CA</i>/<i>AKT1</i>/<i>PTEN</i> alteration testing was performed in 31.8% of patients with HR+/HER2− mBC and 50.0% with mTNBC, with alterations identified in 15.4% and 20.0% of patients, respectively. Median OS (65.9 and 31.4 months, respectively), OS rates (1 year: 94.8% and 79.8%; 5 years: 56.0% and 21.7%), and time to next treatment (1L–2L: 24.5 and 8.1 months; 2L–3L: 10.1 and 5.1 months) were greater for HR+/HER2− mBC than mTNBC.</p><h3>Conclusions</h3><p>These findings describe historical HER2− mBC treatment patterns and associated outcomes in Canada. Ongoing research is needed to optimize therapeutic strategies, expand novel treatment access, and improve patient outcomes.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2034 - 2053"},"PeriodicalIF":4.0,"publicationDate":"2026-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147430036","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":"Content Validation of the Flares Diary: A Qualitative Analysis of the Flares Experience Within the Fibrodysplasia Ossificans Progressiva (FOP) Population","authors":"Jessica Baldasaro,&nbsp;Robert J. Sanchez,&nbsp;Christopher Hartford,&nbsp;Kathryn M. Dahir,&nbsp;Richard Keen,&nbsp;Thomas Funck-Brentano,&nbsp;Robert J. Pignolo,&nbsp;Michelle Davis,&nbsp;Danielle E. Altman","doi":"10.1007/s12325-026-03506-6","DOIUrl":"10.1007/s12325-026-03506-6","url":null,"abstract":"<div><h3>Introduction</h3><p>Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, autosomal dominant disorder characterized by episodic yet cumulative heterotopic ossification (HO) of connective tissues. Flare-ups, i.e., sites of local soft tissue inflammation, are associated with swelling, joint stiffness, and pain are commonly associated with FOP. This study aimed to investigate the content validity of the Flares Diary by gaining an in-depth understanding of the experience of FOP flare-ups in people with FOP and by debriefing the Flares Diary to assess the relevance, comprehensiveness, and understandability of the instrument.</p><h3>Methods</h3><p>Adults with FOP who participated in the Phase 2 LUMINA-1 trial (NCT03188666) of garetosmab, or who were recruited by email via the International Fibrodysplasia Ossificans Progressiva Association, were enrolled in this study. Trained qualitative researchers conducted in-depth semi-structured interviews with all participants comprising concept elicitation and cognitive debriefing of the Flares Diary, with participants encouraged to describe flare-up symptoms and impacts in their own words. All interviews were conducted between March 3, 2022, and March 7, 2023. A conceptual model was developed based on participant-reported symptoms and impacts of flare-ups.</p><h3>Results</h3><p>Thematically analyzed interview transcripts from 20 adults with FOP identified concepts of localized and systemic symptoms, as well as impacts on participants’ daily lives associated with flare-ups. Minimal issues were reported with understanding the Flares Diary, and participants found that the items were comprehensive and matched their own experience of flare-ups.</p><h3>Conclusion</h3><p>The results confirm the adequacy of the Flares Diary and that the symptoms assessed are important to people with FOP.</p><p>Graphical abstract available for this article.</p><h3>Trial Registration</h3><p>ClinicalTrials.gov identifier, NCT03188666.</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":"43 4","pages":"1827 - 1844"},"PeriodicalIF":4.0,"publicationDate":"2026-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03506-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147429957","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}