Advances in Therapy最新文献

{"title":"Patient Burden in the Treatment of Wilson Disease in the United States: An Analysis of Real-World Health Insurance Claims Data from the Komodo database","authors":"Peter Hedera,&nbsp;Megan Teynor,&nbsp;Carey Strader,&nbsp;Halley Costantino,&nbsp;Michael Schultze,&nbsp;Divine Akumo,&nbsp;Karl Heinz Weiss","doi":"10.1007/s12325-026-03501-x","DOIUrl":"10.1007/s12325-026-03501-x","url":null,"abstract":"<div><h3>Introduction</h3><p>Wilson disease (WD) is a rare inherited disorder that causes copper accumulation and can be fatal if untreated. This study used real-world data from the US Komodo Health claims database to describe healthcare resource utilization (HCRU) and evaluate direct economic costs among patients with WD.</p><h3>Methods</h3><p>This retrospective observational study identified patients with WD using ICD-9/10 codes (excluding Menkes disease) between 2016 and 2019, with data spanning 2012–2020. Sociodemographic characteristics, HCRU, and costs were analyzed using SPSS v23, SAS v9.4, and R v3.6.0. The study was approved by Pearl Pathways IRB (#20-KANT-224).</p><h3>Results</h3><p>A total of 2115 patients with prevalent WD, including 360 ever-treated (with reimbursable WD prescriptions), were identified. During the 2-year follow-up, about 25% were hospitalized, with a mean stay of 9 days, and most visited the ER three times annually. Hepatic patients with WD were more likely to undergo liver biopsy or transplant but had fewer home health visits and less use of assistive mobility devices. Annual liver transplant costs averaged $9094.72 ± 8110.23 per prevalent WD patient and $10,147.98 ± 7030.83 per ever-treated patient. Mean annual costs per prevalent versus ever-treated patients with WD were inpatient ($716.52 ± 2675.06 vs. $252.75 ± 333.39), pharmacy ($270.35 ± 1348.79 vs. $1284.51 ± 2994.85), and outpatient ($73.93 ± 156.89 vs. $60.82 ± 62.17), respectively. Pharmacy costs for adherent patients averaged $157,505.28 for any medication, $252,617.11 for <span>d</span>-penicillamine, $189,328.52 for trientine, and $1574.91 for zinc. Among ever-treated patients with WD, respective costs were lower at $85,117.60, $123,190.73, $100,017.20, and $820.35.</p><h3>Conclusion</h3><p>Estimated annual HCRU and treatment costs for patients with WD were lower than previously reported. These findings provide updated real-world insights into the economic burden of WD and highlight the cost implications of medication adherence in managing this rare disorder in the USA.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1705 - 1722"},"PeriodicalIF":4.0,"publicationDate":"2026-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146257031","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":"Medical Absenteeism and Premature Death in Spinal Muscular Atrophy in Sweden: A Population-Based Matched Register Study of People of Working Age","authors":"Thomas Sejersen,&nbsp;Anne-Berit Ekström,&nbsp;Anna-Karin Kroksmark,&nbsp;Nahila Justo,&nbsp;Michael L. Ganz,&nbsp;Charlotte Pettersson,&nbsp;Sophie Graham,&nbsp;Karl Gertow,&nbsp;Sreeram Ramagopalan,&nbsp;Alex Simpson","doi":"10.1007/s12325-026-03503-9","DOIUrl":"10.1007/s12325-026-03503-9","url":null,"abstract":"<div><h3>Introduction</h3><p>Spinal muscular atrophy (SMA) is a rare, progressive, neuromuscular disorder that leads to loss of motor and respiratory function, affecting the individual’s ability to work and life expectancy. The magnitude of productivity losses due to medical absenteeism and premature death associated with SMA is unknown. We estimated the productivity losses attributable to SMA in a working age population compared with the general working age population in Sweden.</p><h3>Methods</h3><p>This was a population-based, 1:4 matched cohort study of patients with SMA aged ≥ 18 years identified in the Swedish National Patient Registry from 2007 to 2019. Amongst those of working age (18–65 years), morbidity-induced productivity losses compared with a matched-reference cohort were estimated as the difference in the number of workdays lost attributable to SMA, monetised using mean income. Mortality-induced productivity losses were estimated as foregone lifetime earnings due to premature deaths for individuals with SMA compared with matched references, after adjusting for sex, age, and background unemployment.</p><h3>Results</h3><p>Overall, 172 adult patients with SMA were identified. Amongst those of working age, their average annual medical absenteeism was 100 days (95% confidence interval 61.5–138.1) higher than that of their references, leading to morbidity-induced productivity losses of €31,638 per patient per year alive whilst of productive age, of which €25,650 was directly attributable to SMA. Average mortality-induced productivity losses due to premature death were €108,253 for men and €87,160 for women with SMA.</p><h3>Conclusions</h3><p>Productivity losses due to medical absenteeism and premature mortality place a significant burden on adult patients with SMA of working age and Swedish society.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1741 - 1755"},"PeriodicalIF":4.0,"publicationDate":"2026-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03503-9.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146257036","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":"Flash Glucose Monitoring System Enhances Glycemic Control and Metabolic Health in People with Type 2 Diabetes on GLP-1RA-Based Therapy: Real-World Evidence","authors":"Ayman Al Hayek,&nbsp;David C. Klonoff,&nbsp;Wael M. Al Zahrani,&nbsp;Nouf Yasin Indarkiri,&nbsp;Mohammed A. Al Dawish","doi":"10.1007/s12325-026-03495-6","DOIUrl":"10.1007/s12325-026-03495-6","url":null,"abstract":"<div><h3>Introduction</h3><p>To evaluate the effect of adding flash glucose monitoring (Flash GM) to glucagon-like peptide-1 receptor agonist (GLP-1RA) therapy on glycemic and metabolic outcomes in people with type 2 diabetes (PwT2D) who failed to achieve target HbA1c levels.</p><h3>Methods</h3><p>A prospective cohort study enrolled 264 PwT2D who were on stable GLP-1RA therapy and had failed to reach an HbA1c target of ≤ 7.0% at our institution. Participants were assigned to Flash GM + GLP-1RA or GLP-1RA alone, matched by age and baseline HbA1c using a nearest-neighbor approach, and followed for 12 months. Changes in HbA1c, body weight, lipid profile, and continuous glucose monitoring (CGM) metrics were assessed.</p><h3>Results</h3><p><b>A</b>fter 12 months, the Flash GM group achieved a mean HbA1c reduction of − 0.58% (from 8.24 to 7.66%) versus − 0.35% (from 8.32 to 7.97%) in the GLP-1RA-only group (<i>p</i> &lt; 0.001). Weight decreased by − 4.03 kg (from 83.97 to 79.95 kg) with Flash GM, significantly more than the − 2.29 kg reduction (from 84.75 to 82.50 kg) in the GLP-1RA alone group (<i>p</i> &lt; 0.001). Total cholesterol and triglyceride levels decreased significantly in the Flash GM group, while HDL cholesterol levels increased, compared to minimal changes in the controls. LDL levels decreased within the Flash GM group; however, the between-group change did not reach statistical significance. Mean time-in-range was 69.6%, with low hypoglycemia exposure and no signal for increased severe events or diabetes-related emergencies.</p><h3>Conclusion</h3><p>Adding Flash GM to GLP-1RA therapy significantly improved glycemic control, weight, and lipid parameters without added risk. This supports the consideration of integrating Flash GM into routine GLP-1RA-based management to optimize outcomes in PwT2D.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1671 - 1688"},"PeriodicalIF":4.0,"publicationDate":"2026-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146257106","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":"Safety and Tolerability of Nintedanib in Japanese Patients with Progressive Fibrosing Interstitial Lung Diseases: Final Results of 2-Year Post-Marketing Surveillance","authors":"Tomohiro Ito,&nbsp;Akira Shibuya,&nbsp;Hiroko Noguchi","doi":"10.1007/s12325-026-03502-w","DOIUrl":"10.1007/s12325-026-03502-w","url":null,"abstract":"<div><h3>Introduction</h3><p>Nintedanib, a tyrosine kinase inhibitor, is approved for treatment of progressive fibrosing interstitial lung disease (PF-ILD). Existing clinical safety data for nintedanib treatment of PF-ILD in Japanese patients is based on relatively few patients, therefore, further evaluation is needed. An interim report of the present surveillance has been previously published; here, we report data from the full surveillance.</p><h3>Methods</h3><p>Non-interventional, prospective, 2-year post-marketing surveillance evaluated the safety of nintedanib in a real-world setting in Japan, in patients with PF-ILD other than idiopathic pulmonary fibrosis or systemic sclerosis-associated ILD between October 2, 2020, and January 22, 2025. Patients newly initiated on nintedanib, 150 mg bid or 100 mg bid, were included. The primary outcome was the incidence of adverse drug reactions (ADRs). Other safety outcomes included the incidence of adverse events (AEs) leading to treatment discontinuation, AEs leading to death, and serious adverse events (SAEs).</p><h3>Results</h3><p>ADRs were reported in 246/408 (60.29%) patients; the most common were diarrhea (30.88%), nausea (8.82%), and hepatic function abnormal (8.33%). There were no noticeable differences in the incidence of ADRs between subgroups by clinical ILD diagnosis. AEs were the main reason for treatment discontinuation. AEs leading to treatment discontinuation were reported for 145 (35.54%) patients. The most common reason was diarrhea (6.86%). The adjusted annual rate of decline in forced vital capacity was − 93.2 mL/year [SE: 65.6, 95% confidence interval − 223.1 to 36.8 (<i>n</i> = 200)].</p><h3>Conclusions</h3><p>The safety profile of nintedanib in clinical practice is consistent with previous reports. No new safety concerns were observed. However, the effectiveness of nintedanib in this real-world setting was not sufficient to stop disease progression, and the treatment discontinuation rate due to AEs was high.</p><h3>Trial registration</h3><p>NCT04559581.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1723 - 1740"},"PeriodicalIF":4.0,"publicationDate":"2026-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03502-w.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146257056","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":"Predicting Long-Term Weight Loss Using Self-Reported, Digitally Collected, Real-World Data After Initiation of Semaglutide for Overweight or Obesity","authors":"Kristine Færch,&nbsp;Mikel M. Gomes,&nbsp;Maja Bramming,&nbsp;Mads R. Sørensen,&nbsp;Anders Strathe","doi":"10.1007/s12325-026-03507-5","DOIUrl":"10.1007/s12325-026-03507-5","url":null,"abstract":"<div><h3>Introduction</h3><p>We hypothesised that an exposure–response weight predictor algorithm developed from randomised controlled trial data can predict long-term individual body weight changes in both men and women in response to subcutaneous semaglutide treatment for weight management using self-reported, real-world data, collected through a digital patient support program (PSP).</p><h3>Methods</h3><p>An exposure–response body weight prediction model developed from clinical trials with semaglutide in people with overweight or obesity was applied to a real-world dataset from patients prescribed semaglutide (0.25–2.4 mg) by their treating healthcare provider (HCP) and enrolled into an app-based PSP. Model variables were baseline sex and body weight, and self-reported dosing and body weight during semaglutide treatment. Predictions were assessed in two scenarios. In the first scenario, body weight at 26 ± 4 weeks was predicted from baseline data with model updates at weeks 4, 8, and 16. In the second scenario, body weight at 52 ± 4 weeks was predicted from baseline data with updates at weeks 8, 16, and 28. Model bias was calculated as the difference between predicted and self-reported body weight, while precision for predicting categorical weight loss (≥ 10%, ≥ 15%, ≥ 20%) was assessed using area under the curve (AUC).</p><h3>Results</h3><p>The study included 1797 WegovyCare® app users, predominantly women (81%), with a mean (SD) age of 48.0 (11.8) years. Mean (SD) self-reported body weight in the entire population was 105 (19.5) kg at baseline. In the half-year scenario, users lost an average of 15% (15.6 kg). Model bias was low (0.7–1.4 kg) and precision for predicting categorical weight loss was high (AUC 0.74–0.95). In the full-year scenario, average weight decreased by 21% (22.0 kg) with similarly low bias (−0.6 to 0.6 kg) and high prediction precision for categorical weight loss (AUC 0.75–0.92).</p><h3>Conclusion</h3><p>This study successfully applied an exposure–response weight predictor algorithm to self-reported data collected from users in the real world. Integrating weight predictors into digital PSPs may be valuable for both patients and HCPs in managing weight loss and setting or monitoring treatment targets.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1697 - 1704"},"PeriodicalIF":4.0,"publicationDate":"2026-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03507-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146257084","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":"Hospital-Treated Severe Hypoglycemia in Type 1 Diabetes in the US: A Retrospective Claims-Based Study of Diabetes-Related Complications and Direct Medical Costs","authors":"Adriana Boateng-Kuffour,&nbsp;Nanxin Li,&nbsp;Keval Chandarana,&nbsp;Vamshi Ruthwik Anupindi,&nbsp;Liang Chen,&nbsp;Michael Hull,&nbsp;Xiaoyu Zhou,&nbsp;Jason Gaglia,&nbsp;Mitch DeKoven,&nbsp;Beth Barber","doi":"10.1007/s12325-026-03505-7","DOIUrl":"10.1007/s12325-026-03505-7","url":null,"abstract":"<div><h3>Introduction</h3><p>Severe hypoglycemic events (SHEs) impose substantial clinical and economic burden on people with type 1 diabetes (pwT1D), yet real-world data describing this burden remain limited in the U.S. hospital setting. This study examined T1D-related complications and direct medical costs in pwT1D hospitalized or treated in the emergency department (ED) for hypoglycemia.</p><h3>Methods</h3><p>IQVIA’s PharMetrics Plus database was used to identify adults with T1D who experienced at least 1 hypoglycemic event requiring an inpatient hospitalization or ED visit from April 2016-April 2020. The SHE date was defined as the date of the first hospital-treated SHE: a claim with a hypoglycemia diagnosis and an inpatient hospitalization or ED visit. Patients were followed up until the end of continuous enrollment or end of the study period (April 30, 2022). Prevalence of T1D-related complications were descriptively summarized, and all-cause direct medical costs were calculated as per-patient-per-year (PPPY). The study was conducted during a period of early adoption of advanced diabetes technologies, such as hybrid closed-loop systems, and only included direct medical costs, potentially underestimating total costs.</p><h3>Results</h3><p>Among 4627 adults with T1D and hospital-treated SHEs, mean age was 41.4 years, 57.3% were male, and 93.4% had commercial insurance. Common comorbidities included hypertension (24.6%), anxiety (9.5%), and depression (9.3%). Prevalence of retinopathy, neuropathy, chronic kidney disease (any stage), coronary artery disease, and peripheral vascular disease were 38.6%, 38.6%, 17.7%, 13.2%, and 11.4%, respectively. Total all-cause direct medical costs averaged $52,849 PPPY in 2022 USD ($59,719 in 2025 USD), driven primarily by inpatient hospitalization (mean—$22,981 in 2022 USD, $25,968 in 2025 USD). Among patients with a hospitalization, mean (SD) number of hospitalizations (PPPY) were 1.5 (3.6) and the average length of stay per patient (PPPY) was 5.0 (20.7) days.</p><h3>Conclusion</h3><p>High T1D-related complication rates and elevated direct medical costs highlight the complexity of pwT1D who experienced hospital-treated SHEs.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1689 - 1696"},"PeriodicalIF":4.0,"publicationDate":"2026-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03505-7.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146257074","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":"Comparative Effectiveness and Risk of Severe Infection in Adult Patients With MS Treated With Diroximel Fumarate Versus Anti-CD20 Monoclonal Antibodies: A Real-World Claims Analysis","authors":"Ahmed Z. Obeidat,&nbsp;Michelle Betz,&nbsp;Rebecca Straus Farber,&nbsp;Erica Goff,&nbsp;Mark Gudesblatt,&nbsp;Le H. Hua,&nbsp;Yang Mao-Draayer,&nbsp;Derrick Robertson,&nbsp;Jonathan D. Santoro,&nbsp;Tony Wang,&nbsp;Daniel Gomes,&nbsp;Ivan Bozin,&nbsp;Jason P. Mendoza,&nbsp;Boyang Bian,&nbsp;James B. Lewin,&nbsp;Nicholas Belviso,&nbsp;Sai L. Shankar","doi":"10.1007/s12325-025-03480-5","DOIUrl":"10.1007/s12325-025-03480-5","url":null,"abstract":"<div><h3>Introduction</h3><p>Multiple sclerosis (MS) is a chronic, immune-mediated neurological disease, leading to significant morbidity. Over 25 disease-modifying therapies (DMTs) are approved for MS; however, older patients may benefit less from high-efficacy DMTs. We compared the risk of severe infections (SIs) and annualized relapse rate (ARR) by age (&lt; 45 and ≥ 45 years) between diroximel fumarate (DRF) and anti-CD20 monoclonal antibodies (mAbs) in patients with MS.</p><h3>Methods</h3><p>This retrospective study utilized the Komodo Health Claims database to identify patients treated with DRF or anti-CD20 agents. Patients were propensity score matched 1:1 on baseline characteristics and stratified by age (younger: &lt; 45 years; older: ≥ 45 years). Infection-related encounters were identified by diagnosis codes; SIs required hospitalization or intravenous antibiotics. MS relapses were based on inpatient or outpatient claims and associated treatments.</p><h3>Results</h3><p>Between 2016 and 2025, 2894 propensity score-matched patients with MS who initiated DRF (<i>n</i> = 1447) or anti-CD20s (<i>n</i> = 1447) were included. DRF-treated patients had a lower proportion of SIs at 12 and 24 months compared with anti-CD20-treated patients (<i>p</i> ≤ 0.002 at 24 months). Younger DRF-treated patients had significantly fewer SIs (<i>p</i> = 0.005), while older DRF-treated patients had lower non-SI rates. COVID-19-related SIs were also significantly lower in DRF-treated patients (<i>p</i> &lt; 0.001). ARRs were similar between the two groups.</p><h3>Conclusion</h3><p>DRF-treated patients with MS had a significantly lower risk of SI compared with anti-CD20-treated patients, with no difference in ARR. More real-world studies are needed to understand the efficacy and safety of DMTs in the setting of de-escalation in aging patients with MS.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1653 - 1670"},"PeriodicalIF":4.0,"publicationDate":"2026-02-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03480-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146218284","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 Unresectable Stage III EGFR-Mutated Non-Small Cell Lung Cancer in China","authors":"Shun Lu,&nbsp;Xingya Li,&nbsp;Changchun Wang,&nbsp;Ken Chen,&nbsp;Yong Liang,&nbsp;Zheng Yin,&nbsp;Feruza Nasirova,&nbsp;YongJin Kim","doi":"10.1007/s12325-025-03488-x","DOIUrl":"10.1007/s12325-025-03488-x","url":null,"abstract":"<div><h3>Introduction</h3><p>Following the LAURA study (NCT03521154), the third-generation epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI) osimertinib was approved for unresectable stage III EGFR-mutated non-small cell lung cancer (NSCLC) after chemoradiotherapy. To inform clinical decision-making, we report real-world (rw) treatment patterns and outcomes in Chinese patients with unresectable stage III EGFR-mutated NSCLC who underwent chemoradiotherapy.</p><h3>Methods</h3><p>Data were retrospectively extracted from the China Multicentre Lung Cancer Precision Medicine Registry medical records of adults with unresectable stage III EGFR-mutated (exon 19 deletion [Ex19del]/leucine-to-arginine substitution at position 858 [L858R]) NSCLC (diagnosed Jan 2016–Dec 2019) who received standard-of-care chemoradiotherapy. The primary outcome was rw progression-free survival (rwPFS) and secondary outcomes were mutation testing patterns, treatment sequencing post-diagnosis, and rw time-to-next treatment or death (rwTTNTD).</p><h3>Results</h3><p>Of 51 patients, 65%/33% had Ex19del/L858R mutations. As first treatment, 31 patients (61%) received chemoradiotherapy plus EGFR-TKI (mainly first-generation) and 20 (39%) chemoradiotherapy alone. Of 34 (67%) patients who received first-subsequent treatment, most were given EGFR-TKI (88%). In patients who received chemoradiotherapy plus EGFR-TKIs (first-generation) or chemoradiotherapy alone, median (95% confidence interval) rwPFS/rwTTNTD was 22.6 (14.2–34.6)/25.0 (21.5–44.7) or 12.2 (10.5–not estimable)/13.6 (10.5–not estimable) months, respectively.</p><h3>Conclusion</h3><p>These rw results show that while first-generation EGFR-TKIs are associated with numerically longer rwPFS and rwTTNTD relative to chemoradiotherapy alone in Chinese patients with unresectable stage III EGFR-mutated NSCLC, there remains an unmet need to prolong survival.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1639 - 1652"},"PeriodicalIF":4.0,"publicationDate":"2026-02-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03488-x.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146199833","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":"Impact of Diffusing Lung Capacity for Carbon Monoxide on Mortality Risk in Patients with ASMD: Insights from a Post Hoc Analysis","authors":"Wim A. Wuyts,&nbsp;Francesco Bonella,&nbsp;Maurizio Scarpa,&nbsp;Venediktos Kapetanakis,&nbsp;Pragya Shukla,&nbsp;Marie Fournier,&nbsp;Maja Gasparic,&nbsp;Ruth Pulikottil-Jacob","doi":"10.1007/s12325-025-03481-4","DOIUrl":"10.1007/s12325-025-03481-4","url":null,"abstract":"<div><h3>Introduction</h3><p>Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder with heterogenous clinical manifestations, including interstitial lung disease (ILD). Respiratory manifestations are the leading causes of mortality in patients with ASMD type B and type A/B. In ILD, the percent predicted diffusing capacity of the lungs for carbon monoxide (DL_CO) is a common clinical endpoint; however, its clinical relevance in patients with ASMD remains unclear.</p><h3>Methods</h3><p>This post hoc analysis explored the relationship between DL_CO and mortality risk in patients with ASMD type B and type A/B. Data from a prospective natural history study (NCT02004704) and a retrospective cohort study conducted in the United States were pooled based on the eligibility criteria. Percent predicted DL_CO was imputed for 10 records (9/68 patients), assuming an annual 1% linear decrease in DL_CO. A Cox proportional hazards model was fitted using percent predicted DL_CO as a time-varying predictor at &lt; 60% and ≥ 60%.</p><h3>Results</h3><p>A total of 68 patients (prospective study,<i> n</i> = 40; retrospective study, <i>n</i> = 28) diagnosed with ASMD type B or type A/B during childhood (aged 1–12 years) with ≥ 1 DL_CO measurement were included in the analysis. A total of 12 deaths were recorded. The estimated hazard ratio (95% confidence interval) was 0.77 (0.22–2.67), indicating a potential trend toward an association of lower mortality risk with higher percent predicted DL_CO (≥ 60%). However, the result was not statistically significant (<i>p</i> = 0.69) because of the limited sample size, thus warranting further prospective validation.</p><h3>Conclusions</h3><p>To our knowledge, this is the first analysis to explore the relationship between DL_CO and mortality risk in patients with ASMD type B and type A/B. Overall, these findings underscore how DL_CO affects the mortality risk in patients with ASMD.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1845 - 1853"},"PeriodicalIF":4.0,"publicationDate":"2026-02-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03481-4.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146199817","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":"Practice-Based Consensus Amongst Indian Neurologists: Adopting Remote Electrical Neuromodulation for Effective Management of Migraine","authors":"Debashish Chowdhury,&nbsp;Jaydip Ray Chaudhuri,&nbsp;Pravar Passi,&nbsp;Rajasekar Reddi,&nbsp;Rajiv Anand,&nbsp;Sumit Singh,&nbsp;T. K. Banerjee,&nbsp;Vikram Sharma,&nbsp;Mayank Ravindra Dhore,&nbsp;Sujeet Narayan Charugulla,&nbsp;Bhavesh P. Kotak,&nbsp;Sukhpreet Singh","doi":"10.1007/s12325-026-03500-y","DOIUrl":"10.1007/s12325-026-03500-y","url":null,"abstract":"<div><h3>Introduction</h3><p>Migraine is a major cause of disability in India, disproportionately affecting women and adolescents. Although traditional pharmacological treatments exist, side effects and poor adherence have increased interest in non-pharmacological options such as remote electrical neuromodulation (REN). However, clinical adoption of REN remains limited because of the absence of clear, India-specific guidelines.</p><h3>Methods</h3><p>An Indian consensus was developed through a two-phase Delphi process involving 49 neurologists. Phase 1 included a scientific committee of eight neurologists who conducted a targeted literature review on REN and formulated 27 consensus statements. In phase 2, 41 neurologists from across India participated in modified Delphi rounds to finalize these statements.</p><h3>Results</h3><p>Statements achieving over 60% agreement identified REN as a promising and well-tolerated non-pharmacological therapy that addresses limitations of current migraine treatments. High consensus (75–97.22%) highlighted REN’s favorable safety profile, minimal adverse events, and suitability for adolescents and women of childbearing age.</p><h3>Conclusion</h3><p>This Indian consensus provides practical guidance for integrating REN into migraine management. The REN device shows potential as a first-line or adjunct therapy, particularly for patients facing challenges with pharmacological treatment adherence, supporting its wider adoption in clinical practice.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1622 - 1638"},"PeriodicalIF":4.0,"publicationDate":"2026-02-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03500-y.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146199797","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}