Advances in Therapy最新文献

{"title":"The Critical Role of Oxygen Supplementation in Epithelium-On Corneal Cross-Linking: A Narrative Review","authors":"Christopher J. Rapuano,&nbsp;Kenneth A. Beckman,&nbsp;Rajesh Rajpal","doi":"10.1007/s12325-026-03521-7","DOIUrl":"10.1007/s12325-026-03521-7","url":null,"abstract":"<div><p>Keratoconus is a progressive corneal disorder characterized by thinning and steepening of the cornea, leading to visual impairment and reduced quality of life. Although epithelium-off corneal cross-linking has emerged as the gold-standard therapy, it can be associated with postoperative pain, delayed healing, and increased risk of complications due to removal of the corneal epithelium. To overcome these limitations, epithelium-on cross-linking was developed as a less invasive alternative. In epithelium-on cross-linking, three essential components must be present to maximize the photochemical reaction: oxygen, riboflavin formulation with permeation enhancers, and high-irradiance pulsed ultraviolet-A (UV-A) light. Oxygen plays an especially critical rate-limiting role, as it interacts with riboflavin and UV-A irradiation to produce the reactive oxygen species (ROS) that promote covalent cross-links, thereby strengthening the corneal stroma. Given that the intact epithelium during epithelium-on cross-linking can act as a barrier to oxygen diffusion, in addition to riboflavin penetration, enhancing oxygen availability during epithelium-on cross-linking has been shown to improve treatment outcomes. Oxygen-enriched epithelium-on cross-linking is poised to emerge as a mainstay therapy for keratoconus. This narrative review examines the influence of oxygen supplementation, a key rate-limiting factor, in enhancing the efficacy of epithelium-on cross-linking.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1423 - 1438"},"PeriodicalIF":4.0,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03521-7.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147343214","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 Home-Based Respiratory Physiotherapy in Nusinersen-Treated Patients with Spinal Muscular Atrophy","authors":"Moria Be’er,&nbsp;Lior Shperling,&nbsp;Mika Rochman,&nbsp;Israel Amirav,&nbsp;Michal Cahal,&nbsp;Revital Lavi,&nbsp;Efraim Sadot,&nbsp;Yotam Lior,&nbsp;Moran Lavie","doi":"10.1007/s12325-026-03524-4","DOIUrl":"10.1007/s12325-026-03524-4","url":null,"abstract":"<div><h3>Introduction</h3><p>Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder associated with respiratory complications and reduced quality of life (QOL). Although disease-modifying therapies have altered the clinical course of SMA, the role of home-based respiratory physiotherapy as an adjunct to pharmacologic treatment remains underexplored. The aim of this study is to evaluate the impact of a 1-year home-based respiratory physiotherapy program on QOL and pulmonary function in nusinersen-treated patients with SMA types 2 and 3.</p><h3>Methods</h3><p>This mixed prospective interventional and retrospective analytical study assessed the impact of weekly home-based, personally tailored respiratory physiotherapy sessions in addition to standard multidisciplinary care. Group allocation was determined by patient or parental willingness to participate and geographical feasibility for weekly home visits. Pulmonary function tests (PFTs) were performed at baseline and after 12 months. QOL was assessed with the SF-36 questionnaire and the Global Rating of Change (GROC) scale.</p><h3>Results</h3><p>Twenty-nine patients with spinal muscular atrophy (SMA) types 2 and 3 receiving nusinersen were included. The intervention group (<i>n</i> = 15) and the control group (<i>n</i> = 14) were comparable at baseline. Objective respiratory parameters remained stable in both groups with no significant differences at the end of the intervention (forced vital capacity % predicted: 72.7 ± 25.1 in the intervention group vs. 69.4 ± 26.5 in the control group, <i>p</i> = 0.7). In contrast, the intervention group demonstrated significantly higher scores in multiple SF-36 domains, including physical functioning and energy/fatigue (41.3 ± 43.7 vs. 2.1 ± 3.7 and 71.7 ± 16.9 vs. 51.4 ± 14.5, respectively; <i>p</i> &lt; 0.05). The median GROC score in the intervention group was 3.0, indicating a clinically meaningful perceived benefit in QOL by exceeding the minimal clinically important difference threshold.</p><h3>Conclusion</h3><p>Home-based respiratory physiotherapy was associated with stable pulmonary indices as well as significant improvements in perceived health status and QOL in patients with SMA treated with nusinersen.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1816 - 1826"},"PeriodicalIF":4.0,"publicationDate":"2026-03-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03524-4.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147324367","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":"Overcoming Hyperkalaemia as a Barrier to Achieving Optimal RAASi Therapy and Cardiorenal Protection in Individuals with Cardiorenal Disease: A Podcast Discussion","authors":"Andrew H. Frankel,&nbsp;Kate Bramham,&nbsp;Barbara Byrne,&nbsp;Geraldine Chiu,&nbsp;Ruby Chumber,&nbsp;Sarah Jane Davies,&nbsp;Ahmet Fuat,&nbsp;Laura Gray,&nbsp;Darren Green,&nbsp;William Priestman,&nbsp;Mandie Welch,&nbsp;Simon G. Williams,&nbsp;Stephen Wheatcroft","doi":"10.1007/s12325-026-03518-2","DOIUrl":"10.1007/s12325-026-03518-2","url":null,"abstract":"<div><p>Renin–angiotensin–aldosterone system inhibitor (RAASi) therapies are a cornerstone of guideline-directed medical therapy in the management of cardiorenal disease, including chronic kidney disease and heart failure. Management guidelines state that these therapies should be prescribed at the maximum licensed or tolerated dose in order to prevent disease progression and adverse events. However, both cardiorenal disease and RAASi therapies increase the risk of hyperkalaemia. When hyperkalaemia occurs, clinicians often down-titrate or discontinue these important RAASi therapies, leaving patients at risk of adverse cardiorenal outcomes. Eleven cardiorenal experts considered how hyperkalaemia can act as a barrier to optimised RAASi therapy in patients with cardiorenal disease and how these issues could be mitigated. Four key areas of suboptimal and variable clinical practice were identified: optimisation of RAASi therapy; definition and management of acute hyperkalaemia; secondary to primary care communications; and patient education. In this podcast article, two of the experts discuss the clinical challenges and principles of optimal care for each of the four areas identified. The experts agreed that hyperkalaemia should be considered a predictable and manageable condition, requiring a pre-emptive and long-term approach. When hyperkalaemia occurs, down-titrating or discontinuing RAASi therapy should be a last resort, after all other management approaches have been employed. Healthcare professionals in secondary and primary care must communicate and collaborate to ensure effective, consistent management of patients with cardiorenal disease. Finally, patients should be educated to understand their cardiorenal disease and its management and the importance of optimal RAASi therapy.</p>\u0000<div><div><iframe></iframe></div><div><p>Supplementary file1 (MP4 173853 KB)</p></div></div></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1369 - 1379"},"PeriodicalIF":4.0,"publicationDate":"2026-03-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03518-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147324318","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":"Cognitive Decline and Illness Progression in Bipolar Disorder","authors":"Isabella S. Ji,&nbsp;Kayla M. Teopiz,&nbsp;William Cheung,&nbsp;Roger S. McIntyre","doi":"10.1007/s12325-026-03527-1","DOIUrl":"10.1007/s12325-026-03527-1","url":null,"abstract":"<div><h3>Introduction</h3><p>Bipolar disorder (BD) is a chronic and heterogeneous condition associated with global and specific cognitive deficits, including but not limited to impaired executive function, memory, and processing speed. As cognitive impairment represents a therapeutic target, this systematic review aims to synthesize available evidence investigating the association between clinical markers of illness progression and cognitive decline in adults with BD.</p><h3>Methods</h3><p>A systematic search was conducted in PubMed, OVID (PsycINFO), and Scopus from inception to September 27, 2025. Eligible studies included adults (≥ 18 years) with BD (I, II, or mixed) that reported a direct comparison between subgroups of illness progression markers (illness duration and/or number of mood episodes). The primary outcome was cognitive function assessed by validated neuropsychological tests. Eligible study designs were cross-sectional, longitudinal, case-control, cohort, or baseline clinical trial data.</p><h3>Results</h3><p>Available evidence from cross-sectional studies suggest that a higher number of mood episodes or longer illness duration was associated with decreased cognitive function, particularly in domains of executive function and verbal/visual memory. In contrast, several longitudinal studies reported no significant change in cognitive outcomes over time in persons with BD relative to healthy controls.</p><h3>Conclusion</h3><p>Findings on the influence of illness progression markers on cognitive decline in BD is mixed. Future research should prioritize large-scale longitudinal designs and integrate biomarker and neuroimaging methods to investigate underlying mechanisms that may contribute to cognitive worsening in individuals with BD.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1439 - 1485"},"PeriodicalIF":4.0,"publicationDate":"2026-03-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147324322","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":"Joint Assessment Matters: Diagnostic and Therapeutic Benefits of Interdisciplinary Psoriasis Care","authors":"Tassilo Dege,&nbsp;Oliver Steffens,&nbsp;Özlem Kiesel,&nbsp;Iana Bychkova,&nbsp;Lara Leichtfuss,&nbsp;Theresa Schulze-Hagen,&nbsp;Matthias Goebeler,&nbsp;Marc Schmalzing,&nbsp;Jan Leipe,&nbsp;Victor Olsavszky,&nbsp;Patrick-Pascal Strunz,&nbsp;Caroline Glatzel,&nbsp;Astrid Schmieder","doi":"10.1007/s12325-026-03520-8","DOIUrl":"10.1007/s12325-026-03520-8","url":null,"abstract":"<div><h3>Introduction</h3><p>Psoriasis is a chronic inflammatory disease often accompanied by musculoskeletal symptoms and psoriatic arthritis (PsA). Early identification of PsA remains challenging, underscoring the need for interdisciplinary care between dermatology and rheumatology. To evaluate the diagnostic and therapeutic impact of an interdisciplinary dermatology-rheumatology board (IDRB) for patients with psoriasis, we initiated a non-randomized, prospective bicentric study.</p><h3>Methods</h3><p>A total of 182 patients with psoriasis were enrolled at baseline (V0), of whom 111 completed the 12-month follow-up (V2). Forty-seven (25.8%) patients participated in the IDRB, and 135 (74.2%) patients received standard dermatological care. Psoriasis Area and Severity Index (PASI), Dermatology Life Quality Index (DLQI), Hospital Anxiety and Depression Scale (HADS-A/D), pain, systemic inflammation, psoriatic arthritis (PsA) diagnosis, and systemic therapy courses were analyzed. Group differences and changes over time were assessed using non-parametric and parametric tests, and predictors of therapy modification were explored using univariate logistic regression.</p><h3>Results</h3><p>Over 12 months, patients in the IDRB group showed statistically significant improvements in PASI, DLQI, and HADS-A (all <i>p</i> ≤ 0.05). Among participants without PsA at baseline and with complete PsA documentation at follow-up, new PsA diagnoses occurred more often in the IDRB cohort (31%) than in standard care (9.8%) (Fisher’s exact <i>p</i> = 0.0295; <i>χ</i>² <i>p</i> = 0.0360; OR = 4.14). In univariate analyses, higher baseline PASI, DLQI, and HADS-A values were each associated with subsequent therapy modification. Within the IDRB group, biologic treatments shifted over time toward IL-17- and IL-23-targeted agents, indicating a move toward more streamlined and targeted systemic therapy patterns compared with standard care.</p><h3>Conclusion</h3><p>An IDRB may contribute to more structured PsA assessment and to more informed therapeutic decisions in patients with psoriasis. Integrating objective clinical measures together with patient-reported burden appears crucial for guiding treatment modification and optimizing outcomes. Given the non-randomized, self-selected design, these findings should be interpreted as associations.</p><h3>Trial Registration</h3><p>DRKS-Deutsches Register Klinischer Studien listing: DRKS00037907.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1790 - 1801"},"PeriodicalIF":4.0,"publicationDate":"2026-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03520-8.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147315843","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":"Matching-Adjusted Indirect Comparison of Olutasidenib and Ivosidenib in Isocitrate Dehydrogenase 1-Mutated Relapsed/Refractory Acute Myeloid Leukemia","authors":"Justin M. Watts,&nbsp;Eunice S. Wang,&nbsp;Brian A. Jonas,&nbsp;Florence R. Wilson,&nbsp;Julie E. Park,&nbsp;Shannon Cope,&nbsp;Aaron Sheppard,&nbsp;Amber Thomassen,&nbsp;Stéphane de Botton,&nbsp;Jorge E. Cortes","doi":"10.1007/s12325-026-03522-6","DOIUrl":"10.1007/s12325-026-03522-6","url":null,"abstract":"<div><h3>Introduction</h3><p>Olutasidenib and ivosidenib are isocitrate dehydrogenase 1 (IDH1) inhibitors approved for relapsed/refractory (R/R) <i>IDH1</i> mutant (<i>IDH1</i>m) acute myeloid leukemia (AML).</p><h3>Methods</h3><p>A matching-adjusted indirect comparison estimated relative treatment effects using registrational Phase I/II data for olutasidenib (Study 2102-HEM-101; individual patient data) and ivosidenib (Study AG120-C-001; study-level data) since a head-to-head trial is unlikely. Weights were estimated using a logistic propensity score model adjusted for pre-defined covariates identified from a literature review, validated by clinical experts. Eight covariates were determined to be the most important prognostic factors/effect modifiers for the target population as reported in the Food and Drug Administration labels: number of prior systemic therapies, age, prior hematopoietic stem cell transplantation, AML type, relapse type, cytogenetic risk, Eastern Cooperative Oncology Group performance status, and IDH1 mutation.</p><h3>Results</h3><p>Olutasidenib versus ivosidenib adjusted rates of complete remission (CR; odds ratio [OR] 1.12, 95% confidence interval [CI] 0.61–2.08), CR plus CR with partial hematologic recovery (CR + CRh; OR 0.83, 95% CI 0.46–1.50), and median CR duration (difference in medians 11.18 months, 95% CI − 4.30 to 22.72) were not significantly different. Median CR + CRh duration was significantly longer for olutasidenib (difference in medians 9.84 months, 95% CI 3.24–22.28), accompanied by a numerical non-significant trend in overall survival that should be considered exploratory (hazard ratio 0.75, 95% CI 0.53–1.07).</p><h3>Conclusion</h3><p>While not confirmatory, these findings may be clinically relevant in the context of this difficult-to-treat R/R <i>IDH1</i>m AML population.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1802 - 1815"},"PeriodicalIF":4.0,"publicationDate":"2026-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03522-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147315884","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":"Pathways to Facilitate Early Recognition and Diagnosis of Hypochondroplasia","authors":"Melita Irving,&nbsp;Elena Greco,&nbsp;Alessandra Cocca,&nbsp;Andrew Dauber,&nbsp;Alexander Augusto de Lima Jorge,&nbsp;Svein Fredwall,&nbsp;Amy Patterson,&nbsp;Juan Llerena Jr.,&nbsp;Keiichi Ozono,&nbsp;Rui Santos,&nbsp;Dominique Kelly,&nbsp;Elena Muslimova,&nbsp;Tejaswini Reddi,&nbsp;Renée Shediac,&nbsp;Ravi Savarirayan,&nbsp;V. Reid Sutton,&nbsp;Julie Hoover-Fong,&nbsp;Moira Cheung","doi":"10.1007/s12325-026-03526-2","DOIUrl":"10.1007/s12325-026-03526-2","url":null,"abstract":"<div><h3>Introduction</h3><p>Hypochondroplasia (HCH) is a disproportionate short-statured skeletal dysplasia condition caused by gain-of-function pathogenic variants in the fibroblast growth receptor 3 gene (<i>FGFR3</i>). Although HCH typically becomes clinically apparent after the first year of life, when height discrepancy compared with the general population becomes more pronounced, diagnosis is often delayed by several years. Early recognition of HCH is challenging because of wide phenotypic variability and subtle clinical and radiographic features, leading to delayed or missed diagnosis. Furthermore, wide variant heterogeneity and restrictive testing criteria can contribute to diagnostic delays. Early diagnosis may facilitate timely clinical management and psychosocial support. However, no standardized diagnostic criteria for HCH currently exist, nor are diagnostic pathways well described in the literature.</p><h3>Methods</h3><p>In October 2024, 14 experts across multiple specialties completed an online survey on current clinical practices for diagnosing HCH. A subset convened in person to discuss strategies to optimize clinical diagnostic pathways, which were subsequently refined by the collective group.</p><h3>Results</h3><p>Age-specific diagnostic opportunities were identified. Prenatally, sonographic features of HCH may be detectable from approximately 20 weeks’ gestation. Postnatally, features suggestive of HCH include a sustained fall in length/height centiles over the first 2 years of life, relative macrocephaly, neonatal seizures, and specific radiographic and neuroimaging findings. Between ages 2–3 years, a characteristic growth pattern including limb shortening and body disproportion may become evident. Neurocognitive involvement including neurodevelopmental challenges may become apparent. HCH should be considered in the differential diagnosis of idiopathic or isolated short stature. Genetic testing panels that include <i>FGFR3</i> and evaluation of short-statured parents can support diagnosis.</p><h3>Conclusion</h3><p>Early diagnosis of HCH is achievable when age-specific key clinical and radiologic features are recognized and supported by molecular testing using appropriate diagnostic platforms. This work represents an important first step towards developing consensus-based diagnostic guidelines for HCH.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 5","pages":"2018 - 2033"},"PeriodicalIF":4.0,"publicationDate":"2026-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03526-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147315862","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 Sore Throat and Antibiotic Resistance (STAR) Study in Malaysia: Nationwide Survey of Antibiotic Use for Upper Respiratory Tract Infection and Sore Throat","authors":"Baharudin Abdullah,&nbsp;So Fie Tan,&nbsp;Shinee Tan,&nbsp;Zahiruddin Wan Mohammad,&nbsp;Andy Watson,&nbsp;Adrian Shephard","doi":"10.1007/s12325-026-03515-5","DOIUrl":"10.1007/s12325-026-03515-5","url":null,"abstract":"<div><h3>Introduction</h3><p>Antimicrobial resistance (AMR) is exacerbated by the ongoing misuse of antibiotics for upper respiratory tract infections (URTIs), and it is vital to try and determine factors associated with antibiotic misuse. The Sore Throat and Antibiotic Resistance (STAR) study aims to evaluate public knowledge, perceptions, and behaviors to antibiotic use for acute URTIs and sore throat in Malaysia.</p><h3>Methods</h3><p>A nationwide online survey was carried out in June 2024 among Malaysian adults who reported having sore throats with other URTI symptoms in the past six months using a random sampling method. A 26-item (plus 5 for screening) self-administered questionnaire, adapted from the Eurobarometer survey on AMR, was shared through the Toluna online panel. The questionnaire included four sections that covered demographic details, URTI symptom patterns, antibiotic use, and self-medication practices for symptomatic relief, as well as knowledge, perceptions, and behaviors about these practices.</p><h3>Results</h3><p>Among 1031 respondents, sore throat was the most common symptom (62%). It often occurred significantly (<i>p</i> &lt; 0.001) with cough (51.4%) and flu-like symptoms (53.8%). Antibiotic use was high, with 72% using them for respiratory illnesses and 33.4% for sore throat. Self-medication for symptomatic relief occurred mostly for sore throat (62%). While 78% had seen AMR messages and 70% knew about antibiotic risks, many held misconceptions: 66.1% thought antibiotics relieve pain and 72.5% believed they speed recovery. Confidence in non-antibiotic care was low at 42%, and 80% expressed anxiety about not using antibiotics. Unsafe behaviors included keeping leftover antibiotics (34%) and stopping treatment early (45%).</p><h3>Conclusions</h3><p>This study found a significant gap between perceived knowledge and practice as well as behaviors that contribute to inappropriate antibiotic use. These findings emphasize the need for targeted interventions such as public education, improved communication between healthcare providers and patients, including the benefits of a pro-symptomatic approach to first line management.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1775 - 1789"},"PeriodicalIF":4.0,"publicationDate":"2026-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03515-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147288756","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":"System Preparedness for Rising Kidney Replacement Therapy Demand: Late-Stage CKD Drives Costs, Healthcare Resource Utilisation and Environmental Impact in Switzerland","authors":"Thomas Campbell-James,&nbsp;Stefan Buff,&nbsp;Giliane Nanchen,&nbsp;Andrew Hall,&nbsp;Thomas Rosemann,&nbsp;Lindsay Nicholson,&nbsp;Stacey Priest,&nbsp;Anthony Zara,&nbsp;Luke Hubbert,&nbsp;Caterina Vecchio Rodriguez,&nbsp;George Wharton,&nbsp;Menno Pruijm","doi":"10.1007/s12325-026-03519-1","DOIUrl":"10.1007/s12325-026-03519-1","url":null,"abstract":"<div><h3>Introduction</h3><p>Chronic kidney disease (CKD) is a leading cause of global morbidity and mortality, affecting one in ten Swiss adults. Patient numbers are expected to increase due to demographic shift and increasing comorbidity rates, necessitating strategic healthcare planning. Holistic burden remains unknown and detailed projections—including expectations of prospective economic and environmental impact—are lacking. To inform Switzerland’s public health strategies and support its 2050 net-zero healthcare goal, this study aims to forecast the multidimensional burdens of CKD.</p><h3>Methods</h3><p>The IMPACT-CKD microsimulation model was utilised to project CKD-progression and clinical, healthcare resource utilisation, economic, societal, and environmental outcomes in Switzerland over 10 years (2023–2032). Modelled individuals were assigned CKD-relevant characteristics—including kidney function, comorbidities and clinical events—based on real-world data. Individuals were categorised into non-CKD or one of six CKD stages and progressed through the disease, diagnosed or undiagnosed. Model inputs and outcomes were validated and calibrated against literature, real-world evidence, and expert consultation.</p><h3>Results</h3><p>By 2032, IMPACT-CKD projects Switzerland’s cases of CKD to rise by 6.7% (0.96m to 1.03m) with late-stage CKD and kidney replacement therapy (KRT) surging by 18.1% (437k to 516k) and 57.2% (8.4k to 13.2k). CKD-related healthcare costs are projected to increase by 27.6% (Swiss francs [CHF] 3.3b to CHF 4.2b), driven by a 77.3% rise KRT-related costs. CKD would account for 4.6% of the Swiss healthcare budget, incur CHF 12.9b in lost productivity, 43.2m missed workdays and CHF 606m in lost tax revenue. Greenhouse gas emissions are projected to increase by 12.9%, driven by rising dialysis demand.</p><h3>Conclusion</h3><p>IMPACT-CKD projects substantial increases in the multidimensional burdens of CKD in Switzerland. Impact could be mitigated via earlier detection and broader uptake of guideline-directed medical therapy. Comprehensive health policies and strategic public health planning are necessary to reduce burden and sustain Switzerland’s 2050 net-zero healthcare ambition.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1756 - 1774"},"PeriodicalIF":4.0,"publicationDate":"2026-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03519-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147281810","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":"Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series","authors":"Melissa D. Svoboda,&nbsp;Nancy Kuntz,&nbsp;Carmen Leon-Astudillo,&nbsp;Barry J. Byrne,&nbsp;Jena Krueger,&nbsp;Jennifer M. Kwon,&nbsp;Cory Sieburg,&nbsp;Diana Castro","doi":"10.1007/s12325-026-03509-3","DOIUrl":"10.1007/s12325-026-03509-3","url":null,"abstract":"<div><p>This Summary of Research summarizes a previously published original article, “Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series.” Spinal muscular atrophy (SMA) is a rare genetic disease that causes muscle weakness and is associated with swallowing and breathing difficulties. Risdiplam (EVRYSDI^®) and onasemnogene abeparvovec (OA, ZOLGENSMA^®) are two medications approved by the US Food and Drug Administration for the treatment of individuals with SMA. This study explored the clinical benefits and safety of using risdiplam after OA in children with SMA. All children whose muscle movement was assessed showed stability or improvement after risdiplam initiation, and around one in three children saw improvements in swallowing and decreased usage of respiratory support. Risdiplam treatment was well tolerated. This study may help to improve understanding of the potential risks and benefits of using risdiplam treatment after OA treatment in children with SMA. Further studies including more children are necessary.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"43 4","pages":"1380 - 1383"},"PeriodicalIF":4.0,"publicationDate":"2026-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-026-03509-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146257102","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}