Advances in Therapy最新文献

{"title":"The IMPACT Survey: The Economic Impact of Caring for an Individual with Osteogenesis Imperfecta.","authors":"Frank Rauch, Taco van Welzenis, Lena Lande Wekre, Cathleen Raggio, Oliver Semler, Ingunn Westerheim, Heather Mulhall, Melanie Anderson, Samantha Prince, Tracy Hart","doi":"10.1007/s12325-025-03373-7","DOIUrl":"https://doi.org/10.1007/s12325-025-03373-7","url":null,"abstract":"<p><strong>Introduction: </strong>The IMPACT Survey (\"IMPACT\") investigated the economic, clinical, and humanistic challenges of osteogenesis imperfecta (OI) on affected individuals, caregivers, and the broader community. Prior publications detail the methodology, initial findings, healthcare expenditures, and quality of life (QoL) impact on adults with OI. Here, data is presented on the productivity and finances of caregivers and any predictors of impact. We hypothesise that caring for an individual with OI will impact the productivity and finances of caregivers.</p><p><strong>Methods: </strong>IMPACT, fielded July through September 2021 in eight languages, targeted adults and adolescents with OI, caregivers (with or without OI), and close relatives. Survey items covered demographics, socioeconomic factors, clinical characteristics, treatment patterns, QoL, and health economics. We performed descriptive analyses of caregivers' productivity and finances and exploratory regression analyses to identify independent associations between care recipient and caregiver characteristics (\"predictors\"), and their economic impact on caregivers.</p><p><strong>Results: </strong>Of 528 caregivers (without OI) with one care recipient, 64% were in paid employment. Of these, 50% reported missing workdays in the preceding 4 weeks (mean 1.9 days). Caregivers reported impacted finances, spending a mean total of €209 out of pocket (OOP) with the most spent on travel to medical appointments (mean €83) and medicine (mean €46) in the preceding 4 weeks. Caregiver spending varied across regions. Caregivers in the USA spent more in 4 weeks (mean €334) than caregivers in EU4 (France, Germany, Italy, and Spain) and UK (mean €163) or Nordic countries (mean €33). Predictors of productivity and OOP spending included caregiver age, sex and employment status, care recipient age, and various signs, symptoms, and events.</p><p><strong>Conclusion: </strong>Our results suggest that caring for an individual with OI may impact caregivers' productivity and finances. The degree of impact may be predicted by caregiver and care recipient age, fracture frequency, and dental problems.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145123883","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":"Number Needed to Treat and Cost Per Responder Analysis of Anti-CGRP Monoclonal Antibodies for Migraine Prevention in Adults for Whom Prior Preventive Treatments have Failed.","authors":"Dimos D Mitsikostas, Susanne F Awad, Rikke Kongerslev, Line Pickering Boserup, Xin Ying Lee, Ravinder Phul, Simona Sacco","doi":"10.1007/s12325-025-03348-8","DOIUrl":"https://doi.org/10.1007/s12325-025-03348-8","url":null,"abstract":"<p><strong>Introduction: </strong>Four monoclonal antibodies (mAbs) targeting calcitonin gene-related peptide (CGRP) signaling are approved for migraine prevention and commonly prescribed/reimbursed after the failure of repurposed anti-migraine medications. Participants achieving clinical response [e.g., ≥ 50% monthly migraine days (MMDs) reduction] during an anti-CGRP mAb trial are likely to continue treatment. We calculated number needed to treat (NNT) and quarterly cost per responder (CPR) across four anti-CGRP mAbs.</p><p><strong>Methods: </strong>Data were from randomized, double-blind, placebo-controlled phase 3b clinical trials that evaluated anti-CGRP mAbs (eptinezumab, fremanezumab, galcanezumab, erenumab) for migraine prevention in adults with episodic or chronic migraine for whom 2-4 prior preventive treatments have failed. NNT was calculated as 1 divided by absolute risk reduction (difference between active treatment and placebo in the proportion of participants with ≥ 50% or ≥ 75% MMD reduction over Weeks 1-12). CPR was calculated by multiplying NNT by the quarterly (3-month) drug acquisition CPR (£), based on the reimbursed list price in the United Kingdom (CPR could not be calculated for eptinezumab 300 mg). Statistical comparisons were not made.</p><p><strong>Results: </strong>All anti-CGRP mAbs demonstrated higher rates of ≥ 50% and ≥ 75% MMD reduction than their respective placebo (p < 0.05). The NNT to achieve ≥ 50% MMD reduction ranged from 2.7 (eptinezumab 300 mg) to 6.0 (erenumab 140 mg), and for ≥ 75%, 6.0 (eptinezumab 300 mg) to 16.2 (fremanezumab 675 mg/q). The cost per ≥ 50% responder ranged from £4647 (eptinezumab 100 mg) to £7009 (erenumab 140 mg), and for ≥ 75%, £9850 (eptinezumab 100 mg) to £21,862 (fremanezumab 675 mg/q).</p><p><strong>Conclusions: </strong>These results show that, for most anti-CGRP mAbs, a low number of participants (< 10) with migraine need to be treated to achieve one person with a ≥ 50% or ≥ 75% reduction in MMDs over Weeks 1-12, with CPR ranging from £4647 (eptinezumab 100 mg) to £21,862 (fremanezumab 675 mg/q).</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145123893","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Outcomes in Patients with COPD Initiating Budesonide/Glycopyrronium/Formoterol Fumarate Dehydrate in Spain: ORESTES Study.","authors":"Bernardino Alcázar-Navarrete, Juan Marco Figueira-Gonçalves, Carmen Corregidor-García, Eunice Fitas, Joaquín Sánchez-Covisa","doi":"10.1007/s12325-025-03361-x","DOIUrl":"https://doi.org/10.1007/s12325-025-03361-x","url":null,"abstract":"<p><strong>Introduction: </strong>Chronic obstructive pulmonary disease (COPD) often results in progressive airflow limitation and is a major cause of morbidity, mortality, and healthcare resource utilization (HCRU). Single-inhaler triple therapy with budesonide/glycopyrronium/formoterol fumarate dehydrate (BGF) is recommended for maintenance in adults with moderate-to-severe COPD not adequately controlled by dual therapy. The ORESTES study aimed to describe the occurrence of exacerbations, clinical characteristics, and HCRU in patients with COPD initiating BGF in a real-life setting in Spain.</p><p><strong>Methods: </strong>Observational, retrospective, multicenter study in patients with COPD aged ≥ 40 years starting BGF treatment at their physician's discretion in routine clinical practice. Data were retrieved 12 months before and up to 12 months after BGF initiation. Occurrence of exacerbations, HCRU, and use of additional COPD medications, together with the patient demographic and clinical profiles, were evaluated.</p><p><strong>Results: </strong>A total of 718 patients were evaluated, of whom 89.3% completed 12 months of BGF treatment. At BGF initiation, most patients were classified as having high-risk phenotype (72.3%), 78.4% presented with dyspnea (mMRC grade ≥ 2), 50.9% were GOLD E, 93.0% had ≥ 3 comorbidities, and 79.7% ≥ 1 cardiovascular comorbidity. Prior initiation of BGF, 41.1% had received dual therapy and 49.6% triple therapy. After BGF initiation, the proportions of patients experiencing moderate and severe exacerbations decreased by 20.8% and 23.1%, respectively. Additionally, the use of all rescue medication decreased by 21.3%, with a similar reduction (21.1%) observed specifically for short-acting beta-2 agonists (SABA). Oral corticosteroids and antibiotics use decreased 17.1%, and 18.2%, respectively. Primary care visits, admissions to the emergency room, and hospitalizations decreased by 18.0%, 25.5%, and 24.7%, respectively.</p><p><strong>Conclusions: </strong>These real-world findings suggest that BGF may provide clinical benefit with high treatment persistence in complex, high-risk patients with COPD, even following high-intensity therapy. The observed improvements, despite advanced disease, raise the possibility that earlier initiation of BGF may help optimize outcomes; however, further study is warranted.</p><p><strong>Clinical trial registration: </strong>NCT06321731.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145111680","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":"Intraoperative Ventilatory Strategies in Patients Undergoing Video-Assisted Thoracic Surgery: A Narrative Review.","authors":"Silvia Coppola, Bruno Pastene, Isabella Fratti, Mert Sentürk, Ebru Emre Demirel, Marc Leone, Davide Alberto Chiumello","doi":"10.1007/s12325-025-03369-3","DOIUrl":"https://doi.org/10.1007/s12325-025-03369-3","url":null,"abstract":"<p><p>Video-assisted thoracic surgery (VATS) and robotic-assisted thoracic surgery (RATS) are commonly used in thoracic surgery, yet postoperative complications still occur in up to 10% of the patients. The aim of our narrative review was to summarize the best available evidence on mechanical ventilation settings, particularly with regard positive end-expiratory pressure (PEEP) selection, tidal volume (VT) and ventilation mode, as well as the feasibility of two-lung ventilation in patients undergoing thoracic surgery using VATS or RATS techniques. We searched the MEDLINE/PubMed database using the terms \"VATS\" or \"RATS\" and \"ventilation\" between 1 January 2007 and 1 February 2025. Publications were screened by title or abstract. We discussed studies according to their methodological quality, ventilation mode, as well as the feasibility of two-lung ventilation in patients undergoing thoracic surgery using VATS or RATS techniques. In patients receiving one-lung ventilation (OLV), the application of a protective lung ventilation using an intermediate VT ranged between 5 and 8 ml (mL) of predicted body weight (PBW) and a PEEP of 5-8 cmH₂O was not found to be associated with a lower incidence of postoperative pulmonary complications and improved hospital outcomes. Titrating PEEP based on mechanical properties appears to enhance perioperative oxygenation and ventilatory mechanics and to reduce postoperative pneumonia. However, no conclusions can be drawn regarding ventilation modes, because only few studies have compared the same low VT using different pressure versus volume control modes. The feasibility of two-lung ventilation during specific thoracic surgery procedures has recently been positively evaluated, with no differences in postoperative complications found. The level of evidence for the ventilatory settings in patients undergoing VATS or RATS remains low. In conclusion, large randomized controlled trials (RCTs) are needed to determine whether certain intraoperative ventilatory strategies can reduce postoperative pulmonary complications (PPCs) in patients.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145085052","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":"Comparing Real-World Outcomes of Prophylaxis with Extended Half-life Factor IX (rIX-FP vs. rFIXFc and N9-GP) for Haemophilia B: An Analysis of Medical Chart Data from Germany.","authors":"Martin Olivieri, Songkai Yan, Ying Yang, Radovan Tomic, Thomas Linhoff, Xiang Zhang, Douglass Drelich, Natalie Jakobs, Wolfgang Miesbach","doi":"10.1007/s12325-025-03336-y","DOIUrl":"https://doi.org/10.1007/s12325-025-03336-y","url":null,"abstract":"<p><strong>Introduction: </strong>In Germany, three extended half-life factor IX (FIX) products are commonly used to treat people with haemophilia B (PwHB). However, there remains a critical need to differentiate treatments for PwHB. The aim of this study was to assess the effectiveness and utilisation of rIX-FP compared with rFIXFc and N9-GP for prophylaxis in clinical practice in Germany.</p><p><strong>Methods: </strong>A retrospective chart review was performed for PwHB aged ≥ 12 years with moderate/severe haemophilia B, who received prophylaxis with rIX-FP, rFIXFc or N9-GP for ≥ 12 months. The primary outcome was FIX consumption; secondary outcomes included annualised bleeding rate (ABR), annualised spontaneous bleeding rate (AsBR) and annualised joint bleeding rate (AjBR).</p><p><strong>Results: </strong>The study included 138 PwHB: rIX-FP, n = 52; rFIXFc, n = 55; and N9-GP, n = 31. Mean FIX consumption with rIX-FP (46.9 IU/kg/week) was significantly lower than that of rFIXFc (70.1 IU/kg/week, p = 0.0083) but not significantly different from N9-GP (47.2 IU/kg/week, p = 0.9331). PwHB receiving rIX-FP prophylaxis had significantly lower mean bleeding rates than those receiving N9-GP (ABR: 0.8 vs. 1.5, p = 0.0472; AsBR: 0.1 vs. 0.6, p = 0.0092; and AjBR: 0.2 vs. 0.6, p = 0.0140). Bleeding rates for rIX-FP and rFIXFc did not differ significantly.</p><p><strong>Conclusion: </strong>rIX-FP prophylaxis was associated with significantly lower FIX consumption and numerically (but not significantly) lower bleeding rates compared with rFIXFc. Compared to N9-GP, prophylaxis with rIX-FP was associated with similar FIX consumption and significantly lower bleeding rates.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145079316","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":"Mohs Micrographic Surgery: A Narrative Review of Current Practices, Emerging Trends, and Case-Based Insights.","authors":"Magdalena Maciejewska, Aleksandra Bętkowska, Joanna Czuwara, Lidia Rudnicka, Laura Banciu, Tiberiu Tebeica, Mihaela Leventer","doi":"10.1007/s12325-025-03354-w","DOIUrl":"https://doi.org/10.1007/s12325-025-03354-w","url":null,"abstract":"<p><p>Mohs micrographic surgery (MMS) is a method used to treat skin cancers. It ensures a complete microscopic examination of the tumor margins with the conservation of surrounding tissue, leading to high cure rates and favorable cosmetic outcomes. In each Mohs stage, the excised specimen is frozen and sectioned horizontally, and its margins are evaluated for the presence of malignant cells. A variety of staining methods can be employed, including immunohistochemical. The process is repeated until all margins are negative. The most common indications for MMS are non-melanoma skin cancers, but it is increasingly utilized for other skin lesions, such as melanoma, lentigo maligna, and dermatofibrosarcoma protuberans. A growing body of evidence indicates significantly higher cure rates and a lower risk of local recurrence associated with MMS compared to other surgical modalities. Numerous modifications of the Mohs surgical technique have been developed to enhance the accuracy of margin control and to tackle specific challenges associated with various tumor types. Such alternative approaches include the \"spaghetti technique,\" the \"slow Mohs\" technique, and other variations such as the Munich method, the square method, the muffin technique, or the perimeter technique. Complications are rare and include infections, bleeding, or impaired wound healing. The increasing popularity of noninvasive imaging, digital pathology, and artificial intelligence models will likely enhance the efficiency of MMS in the future. Machine learning can predict diagnoses, recommend treatment options, predict responses to treatment and potential drug interactions, or help plan surgical procedures, enabling dermatologists to tailor therapies to individual patient characteristics. In addition to presenting the latest trends in Mohs micrographic surgery, this article includes a selection of clinical cases, with an overview of the treatment protocols followed by our institution, as well as data on recurrences based on our clinical experience.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145079356","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":"Fatigue Experience in Oncology; a Targeted Qualitative Literature Review and Novel Patient-Centric Conceptual Model.","authors":"Chloe Carmichael, Cecile Gousset, Danielle Burns, Jordan Miller, Sophie Van Tomme, Helen Kitchen, Harriet Makin, Natalie Aldhouse, Paul Cordero","doi":"10.1007/s12325-025-03330-4","DOIUrl":"https://doi.org/10.1007/s12325-025-03330-4","url":null,"abstract":"<p><strong>Introduction: </strong>Traditionally, treatment endpoints in oncology have focused on increasing overall survival and progression-free survival. Although fatigue may be a meaningful outcome measure across different cancers, it is assessed less frequently. To explore the impact of fatigue on patients with cancer, confirm the importance of fatigue measurement and inform future measurement strategies in oncology, we aimed to characterise the patient experience of fatigue and the associated impact on how patients feel and function, including aspects of health-related quality of life (HRQoL).</p><p><strong>Methods: </strong>A targeted literature review of published fatigue-related qualitative publications in oncology was conducted in MEDLINE, Embase and PsycINFO, limited to studies published between 2018 and 2023. Concepts were extracted from the qualitative literature and patient quotes or author descriptions/interpretations were subject to secondary analysis using semantic, qualitative, directed content analysis techniques via ATLAS.ti v9. The literature review informed the development of a preliminary patient-centric conceptual model of fatigue in oncology.</p><p><strong>Results: </strong>Of 1210 identified publications, 26 were selected for data extraction, including 512 patients living with 28 different oncology indications across geographies. Frequently reported fatigue triggers included treatments and cancer itself. Patients used various fatigue descriptors, including low/lack/loss of energy, tiredness and exhaustion, across disease stages. Physiological and cognitive manifestations of fatigue were reported, and fatigue episodes were described as unpredictable and of variable severity, duration and frequency. Cancer-related fatigue had a substantial negative effect on patients' QoL, including emotional, physical, physiological, social, activities of daily living, sleep and work impacts. Unmet needs included poor support from healthcare professionals (HCPs).</p><p><strong>Conclusions: </strong>This review demonstrated that fatigue is an impactful symptom across oncology indications and provides the first holistic conceptual model of cancer-related fatigue across disease stages and treatments. Future research should review clinical outcome assessments (COA) that may be fit-for-purpose for measuring fatigue in oncology clinical trials.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145079345","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":"Tiprelestat for treatment of hospitalized COVID-19: results of the double-blind randomized placebo-controlled COMCOVID trial.","authors":"Ingmar Bergs, Stephan Budweiser, Hans-Heinrich Henneicke-von Zepelin, Hagen Kelm, Tom Bollmann, Johannes-Josef Tebbe, Stephan Sorichter, Stefan Lüth, Stephan Walterspacher, Henning Wege, Oliver Wiedow, Michael Dreher","doi":"10.1007/s12325-025-03362-w","DOIUrl":"https://doi.org/10.1007/s12325-025-03362-w","url":null,"abstract":"<p><strong>Introduction: </strong>Tiprelestat (recombinant human elafin) reversibly inhibits human neutrophil elastase. This can reduce overactivity of this enzyme in COVID-19-patients and might prohibit further organ damage and progression to severe disease. This protein had yet not been tested in COVID-19 patients.</p><p><strong>Methods: </strong>This prospective, multicenter, randomized, double-blind, placebo-controlled trial investigated Tiprelestat in adult patients hospitalized for COVID-19 in 7 hospitals in Germany between May 2023 and May 2024. Patients received 100 mg Tiprelestat or placebo twice a day for 7 days, or shorter if no longer hospitalized due to COVID-19. Data about efficacy, safety, and pharmacokinetic trough levels were collected over 29 days.</p><p><strong>Results: </strong>A total of 296 patients were planned. Due to slow recruitment during the end of the pandemic, only 17 patients were finally included. Of these, 9 received Tiprelestat and 8 placebo. The mean treatment exposition was 9.1 ± 4.8 (SD, maximum 15) 30-min infusions with Tiprelestat and 7.8 ± 3.2 (maximum 14) with placebo. No relevant abnormalities in clinical or laboratory blood parameters were suspected to be caused by Tiprelestat. None of the Tiprelestat-treated patients developed severe COVID-19 (WHO Clinical Progression Scale ≥ 6). The number of days with any oxygen support tended to be lower in the Tiprelestat group (2.4 ± 3.6 days) compared to placebo (4.0 ± 6.2 days). Stable plasma trough levels of Tiprelestat were shown upon repeated administration for up to 7 days, even in patients with impaired renal function (eGFR > 31 to < 60 mL/min) as comorbidity.</p><p><strong>Conclusion: </strong>Tiprelestat infused for 30 min twice daily in patients with moderate COVID-19 requiring hospitalization was well tolerated and appears to be safe. Beneficially, plasma trough levels of the drug over time were high and stable. The small sample size does not facilitate reliable statements about efficacy. Further studies are necessary including other inflammatory severe respiratory diseases.</p><p><strong>Trial registrations: </strong>EudraCT 2022-000714-33, DRKS00031463.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145068816","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Narrative Review of the Immunomodulatory Effects of Acthar^® Gel Beyond Its Steroidogenic Properties.","authors":"Mehdi Mirsaeidi, Jeffrey Kaplan, John Affeldt, Regina Berkovich, Anca Askanase","doi":"10.1007/s12325-025-03359-5","DOIUrl":"https://doi.org/10.1007/s12325-025-03359-5","url":null,"abstract":"<p><p>Corticosteroids have long been a standard-of-care treatment for chronic immune-mediated inflammatory diseases (IMIDs), such as rheumatoid arthritis, systemic lupus erythematosus, and multiple sclerosis. However, corticosteroids are associated with potentially serious adverse effects and may be ineffective in cases of resistant or refractory disease. Acthar^® Gel, a naturally sourced complex mixture of porcine pituitary peptides, is a noncorticosteroid alternative that is approved by the US Food and Drug Administration to treat a variety of IMIDs and autoimmune conditions. These include infantile spasms, systemic lupus erythematosus, exacerbations of multiple sclerosis and rheumatoid arthritis, and several ocular inflammatory disorders. Preclinical and clinical studies have shown that the immunomodulatory effects of Acthar Gel are distinct from those of corticosteroids and other adrenocorticotropic hormone (ACTH)-class therapeutics. For example, Acthar Gel stimulates both steroid-dependent and steroid-independent pathways that may mitigate inflammation. In this narrative review, we summarize the immunomodulatory effects of Acthar Gel, with a focus on its potential mechanisms in immune cells, such as B cells, T cells, and macrophages. These effects are thought to be mediated by binding and activation of transmembrane melanocortin receptors expressed on these immune cells. Receptor binding initiates an intracellular signal transduction cascade that ultimately regulates the expression of anti-inflammatory genes. Collectively, the experimental and clinical studies reviewed here suggest that Acthar Gel acts as an immunomodulator via melanocortin receptors and may be an effective anti-inflammatory therapeutic option for patients with IMIDs who are refractory or intolerant to corticosteroids.Graphical Abstract available for this article.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145063156","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":"Association of CPAP Prescription and Pneumonia Among Patients with Obstructive Sleep Apnea in the United States: A Retrospective Database Analysis.","authors":"Maria J Tort, Birol Emir, Jennifer L Nguyen, Deepa Malhotra, Chai Hyun Kim, Vincenza T Snow","doi":"10.1007/s12325-025-03343-z","DOIUrl":"https://doi.org/10.1007/s12325-025-03343-z","url":null,"abstract":"<p><strong>Introduction: </strong>Continuous positive airway pressure (CPAP) is the first-line therapy for obstructive sleep apnea (OSA). We sought to understand whether CPAP prescription among adults diagnosed with OSA was associated with a higher likelihood of pneumonia diagnosis and inpatient hospitalization for pneumonia after adjusting for patient demographic characteristics and comorbidities.</p><p><strong>Methods: </strong>This retrospective cohort study used de-identified data from the Optum Clinical Electronic Health Record (EHR) database for the time period January 2012 to December 2019. The study population comprised adults with an OSA diagnosis with and without CPAP prescription after the initial diagnosis. The primary outcome of interest was a pneumonia diagnosis in the first year following OSA diagnosis. Propensity score matching was used to balance the cohorts. Logistic regression was used to estimate the odds of developing pneumonia.</p><p><strong>Results: </strong>There were 328,340 patients with an OSA diagnosis and evidence of CPAP prescription and 964,199 patients with an OSA diagnosis without evidence of CPAP prescription. After 1:1 propensity score matching, 326,145 were included in each cohort. The risk of pneumonia in any setting and in the inpatient setting was higher among patients with CPAP prescription, odds ratio (OR), 1.06 (95% CI: 1.03, 1.09) and 1.24 (95% CI, 1.20, 1.29), respectively. The odds of developing pneumonia increased in any setting when CPAP was initiated on the date of OSA diagnosis (OR = 1.68, 95% CI: 1.62-1.72) among all patients, for patients aged 65 + years (OR = 1.68, 95% CI: 1.47, 1.92), patients with obesity (OR = 1.59, 95% CI: 1.47, 1.72), and patients with a history of pneumonia (OR = 1.68, 95% CI: 1.41, 2.00).</p><p><strong>Conclusion: </strong>This study indicates that among patients with OSA, CPAP initiation was associated with incident pneumonia, particularly inpatient pneumonia hospitalizations. Longer CPAP prescription, older age, obesity, and previous history of pneumonia further increased the risk of incident pneumonia associated with CPAP prescription.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145063105","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}