Advances in Therapy最新文献

{"title":"Neuromodulation in Chronic Migraine: Evidence and Recommendations from the GRADE Framework.","authors":"Claudio Tana, David Garcia-Azorin, Bianca Raffaelli, Mira Pauline Fitzek, Marta Waliszewska-Prosół, Sonia Quintas, Paolo Martelletti","doi":"10.1007/s12325-025-03206-7","DOIUrl":"10.1007/s12325-025-03206-7","url":null,"abstract":"<p><p>Chronic migraine (CM) affects approximately 2% of the general population and is defined by the persistence of migraine symptoms for at least 15 days per month for at least 3 months. CM is often refractory to common drug treatments and is associated with a significant burden in functions of daily life during ictal phases, productivity loss, and direct costs. Modulation of pain is considered pivotal to reduce its impact and to improve the quality of life among patients with CM. In recent years, neuromodulation in CM has received growing attention; however, there remains no consensus regarding the effectiveness and safety of these procedures. Previous invasive methods such as occipital nerve neurolysis and interruption of the trigeminal dorsal root are not indicated due to high rates of relapsing pain and frequent procedural complications. Although emerging neuromodulation methods, both noninvasive, such as vagus nerve stimulation (VNS), transcranial magnetic stimulation (TMS), remote electrical neuromodulation (REM), and invasive, such as deep brain stimulation (DBS), occipital nerve stimulation (ONS), and high-frequency 10-Hz spinal cord stimulation (HF-10 SNS) have demonstrated promising outcomes in early clinical trials, their use has yet to be integrated into routine clinical practice. In this review, study evidence and strength of recommendations are assessed by the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) system. Other conditions such as therapeutic risk/benefit, direct and indirect costs, use of resources, and patient/clinician preferences are also evaluated.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"3020-3044"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182506/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143962911","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":"Reality of Patient-Reported Symptoms in 200 Patients with Eosinophilic Granulomatosis with Polyangiitis: A Cross-Sectional Survey (The KUNPU Study).","authors":"Koichi Amano, Keita Ono, Kazuya Sumi, Hitomi Uchimura, Hayato Oka, Naoyuki Makita, Masami Taniguchi","doi":"10.1007/s12325-025-03197-5","DOIUrl":"10.1007/s12325-025-03197-5","url":null,"abstract":"<p><strong>Introduction: </strong>Eosinophilic granulomatosis with polyangiitis (EGPA) is a type of antineutrophil cytoplasmic antibody-associated vasculitis characterized by inflammation of small- and medium-sized vessels, causing symptoms in multiple organs. The symptoms and daily life problems reported by patients with EGPA themselves are largely unknown. We conducted a cross-sectional survey to investigate the reality of EGPA-related symptoms in patients with EGPA.</p><p><strong>Methods: </strong>Specialists and specialized facilities with experience in treating patients with EGPA cooperated in the survey; specialists from 28 facilities across Japan participated. Patients diagnosed with EGPA by their physician and treated for ≥ 1 year who agreed to answer the online questions were enrolled and completed the survey between March and June 2024. Patients answered questions about their general symptoms, asthma symptoms, and quality of life.</p><p><strong>Results: </strong>We analyzed valid responses from 200 patients (61.0% female/38.5% male/0.5% prefer not to answer) with EGPA. The mean age was 57.9 years and 34.5% were ≥ 65 years old. Patients were treated at rheumatology departments (48.0%), respiratory/allergy departments (48.0%), and other departments (4.0%). Basic treatments included oral glucocorticoids (63.0%) and anti-interleukin-5/receptor α biologics (61.0%). Symptoms in > 50.0% of patients (past month) were pain/numbness (73.5%), fatigue/malaise (68.0%), asthmatic symptoms (56.0%), nasal/paranasal symptoms (55.0%), and joint/muscle pain (54.5%). Pain/numbness was considered the most painful symptom (29.5%). Nearly all patients experienced symptoms affecting two or more organs/systems. Patients reported that EGPA symptoms had detrimental impacts on physical and mental health; 67.0% of patients thought they were not understood by others because their disease is invisible, and symptoms frequently affected their daily life (61.5%), work (53.0%), sleep (49.5%), and social life (36.5%).</p><p><strong>Conclusion: </strong>This is the largest survey of patients with EGPA. We have revealed the reality of patients' perceptions of EGPA-related symptoms. These results are expected to contribute to improvement in patient-centered EGPA management.</p><p><strong>Trial registration: </strong>jRCT1050230186.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"3369-3385"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182462/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144109357","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":"How do Japanese Patients with Chronic Heart Failure View Their Disease, Self-care, and Support?","authors":"Shoko Nagata, Kosuke Shiki, Nobutaka Yagi, Naoki Sato","doi":"10.1007/s12325-025-03203-w","DOIUrl":"10.1007/s12325-025-03203-w","url":null,"abstract":"<p><strong>Introduction: </strong>Chronic heart failure (HF) is a progressive disease with increasing prevalence in Japan. Adherence to lifestyle modifications is important to prevent disease progression and to improve outcomes. An accumulation of insufficient self-care behaviors is an independent risk factor of hospitalization for HF and/or cardiac death; however, there is a lack of self-care awareness in patients with chronic HF. This study aims to increase the understanding of self-care behavior of people living with chronic HF in Japan.</p><p><strong>Methods: </strong>This non-interventional, cross-sectional study used an explanatory sequential design in mixed methods research with a quantitative and consecutive qualitative phase. In the quantitative research phase, an anonymous online survey questionnaire was distributed to 262 people and completed by 162 participants. In the qualitative phase, 15 participants who had completed the quantitative phase were selected using stratified random sampling from the survey participants and took part in a semi-structured interview. Endpoints included participant demographics and HF characteristics, physical and emotional burdens associated with HF, knowledge of disease, self-care, and treatment support in daily life.</p><p><strong>Results: </strong>In the quantitative study, 61.5% of participants were male, and 68.9% were ≥ 60 years old. In the qualitative study, 80.0% were male and 46.7% were ≥ 70 years old. Participants experienced physical and emotional burden due to their HF symptoms with > 60% finding brisk walking, and > 40% finding going up stairs to the second floor, burdensome or very burdensome, and approximately 70% experiencing some emotional burden. In general, participants trusted their physicians' instructions around self-care and had a high level of self-care adherence. Participants would like more information regarding HF treatment (68.0%), diet and nutrition (57.3%), and daily lifestyle tips to reduce HF burden (57.3%).</p><p><strong>Conclusion: </strong>This study highlights the importance of patient education, and the need for multidisciplinary support for patients with chronic HF.</p><p><strong>Trial registration: </strong>UMIN000047055, March 1, 2022 and UMIN000049065, September 29, 2022.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"3207-3222"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182497/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143951858","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":"Real-World Evidence Acceptability and Use in Breast Cancer Treatment Decision-Making in the United States: Call-to-Action from a Multidisciplinary Think Tank.","authors":"Sean Khozin, Nancy A Dreyer, Dominic Galante, Raymond Liu, Peter Neumann, Nathan Nussbaum, Joyce O'Shaughnessy, Debra Patt, Mothaffar Rimawi, Hope Rugo, Sara M Tolaney, Marisa Weiss, Adam Brufsky","doi":"10.1007/s12325-025-03201-y","DOIUrl":"10.1007/s12325-025-03201-y","url":null,"abstract":"<p><p>Complementing randomized controlled trials, real-world evidence (RWE) from observational analyses can extend clinical insights in oncology. While healthcare stakeholders have published rigorous RWE frameworks and resources, a multidisciplinary think tank was established to further advance acceptance and use of RWE in treatment decision-making, with the focus on breast cancer (while recognizing relevance in oncology more broadly). Members discussed perceptions of RWE from a clinical perspective, across domains of data, methodology, and mindset, and \"calls-to-action\" for stakeholders. Agreement was reached on a primary \"call-to-action,\" to develop clinically-relevant, patient-informed, real-world endpoints, and secondary \"calls-to-action\": establish a multidisciplinary consensus forum; publish examples of unique RWE value; build upon existing frameworks and resources; and tailor an approach for exhibiting utility to guideline bodies.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"2973-2987"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182450/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143965233","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":"Conjunctivitis in Adults with Atopic Dermatitis Treated with Dupilumab: An Observational Study of Clinical Characteristics, Symptomatology, and Treatment.","authors":"Penny Asbell, Esen Akpek, Winston Chamberlain, Zhen Chen, Emma Lawless, Michael Van Spall, Zafer E Ozturk, Brad Shumel","doi":"10.1007/s12325-025-03209-4","DOIUrl":"10.1007/s12325-025-03209-4","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Conjunctivitis is more prevalent in patients with atopic dermatitis (AD) than the general population, and is the most common adverse event during dupilumab treatment for AD. We aimed to characterize conjunctivitis diagnosed during AD dupilumab treatment and to assess effectiveness of ophthalmic treatments.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This prospective observational study enrolled adults with AD who received the approved dupilumab regimen in a real-world setting for at least 8 weeks prior to enrollment. At baseline, we compared ophthalmic signs and symptoms in two cohorts of patients who had received dupilumab for similar duration: one with conjunctivitis (group 1) and one with no conjunctivitis (group 2). All patients continued treatment with dupilumab, and group 1 patients received treatment for conjunctivitis at the discretion of the investigator. We assessed effectiveness of prescribed treatments and the outcomes of conjunctivitis up to Week 60. All analyses were descriptive.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 35 patients were assessed in group 1 and 11 in group 2. Mean AD duration was 22.9 ± 15.2 years (group 1) and 13.1 ± 16.9 years (group 2). In group 1, 91% of patients reported a history of facial lesions during AD flares and 54% had current AD facial lesions, compared with 64% and 9%, respectively, in group 2. Ongoing facial contact dermatitis and rosacea were only reported in group 1 (11% and 9%, respectively). Common physical findings at baseline in group 1 compared with group 2 included periocular eczematous rash (65% vs. 18%) and/or lichenification (47% vs. 27%), posterior blepharitis with meibomian gland dysfunction (83% vs. 55%), bulbar hyperemia (89% vs. 46%) and conjunctival papillary pattern (69% vs. 27%); 24% of patients in group 1 and none in group 2 had corneal neovascularization, mostly in a single peripheral quadrant. There was no evidence of tear volume insufficiency. One patient in group 1 (3%) and two in group 2 (18%) had eyelash mites. Three patients (9%) in group 1 and one in group 2 (9%) had Staphylococcus aureus-positive conjunctival swabs. Most patients (88%) received multiple ophthalmic treatments for the qualifying conjunctivitis event. Investigators assessed topical corticosteroids and topical calcineurin inhibitors administered either as eye drops or ointment as the most effective treatments. Most conjunctivitis events recovered/resolved (84%) or were resolving/recovering (12%) at study end. Mean ± SD time to recovery from the initial visit was 171.9 ± 54.6 days. No participants discontinued treatment with dupilumab.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Prolonged history of AD and facial and/or eyelid AD lesions increased the likelihood of a conjunctivitis diagnosis during AD dupilumab treatment. Treatment of conjunctivitis with topical corticosteroids and/or calcineurin inhibitors resolved or controlled the event in most patients without need to discontinue dupilumab.&lt;","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"3285-3305"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144092395","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":"Patient Number and Treatment Patterns in Cytomegalovirus Viremia and Disease Following Solid Organ and Hematopoietic Stem Cell Transplantation in Germany: Results of a Delphi Consensus Study.","authors":"Daniel Teschner, Jana Knop, Christian Piehl, Tino Schubert, Oliver Witzke","doi":"10.1007/s12325-025-03210-x","DOIUrl":"10.1007/s12325-025-03210-x","url":null,"abstract":"<p><strong>Introduction: </strong>Management of cytomegalovirus (CMV) viremia/disease in transplant recipients may be complicated by toxicities and resistance to conventional antivirals, adding to the overall healthcare burden. Despite advances in analyzing real-world data in current years, little is known about refractory and resistant CMV. This study therefore aimed to characterize treatment patterns and patient numbers with special focus on refractory and resistant CMV.</p><p><strong>Methods: </strong>Two classical three-round Delphi consensus panels with German clinical experts in CMV following solid organ transplantations (SOT) and hematopoietic stem cell transplantations (HSCT) were held between October and December 2021 using online questionnaires. Consensus was defined as agreement of 75% of participants.</p><p><strong>Results: </strong>Following SOT, experts agreed that on average 65% of SOT patients are not affected by CMV at all, while 35% of patients experience CMV viremia or disease. Of SOT patients treated with antiviral therapies, experts agreed that 90% respond to their first-line treatment and 10% do not. For HSCT, experts agreed that 62% of patients experience no CMV, while 38% of patients are diagnosed with either CMV viremia or CMV disease. It was further estimated that 23% HSCT patients receiving antiviral treatment do not respond to their first-line CMV treatment. Experts reached consensus on the reasons for non-response, suggesting that among non-responders, 55% were intolerant, while 45% of non-responders were refractory/resistant to first-line treatment.</p><p><strong>Conclusion: </strong>Based on the current incidence of transplantations in Germany, experts estimated that 103 SOT and 225 HSCT patients need second-line CMV treatment annually.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"3419-3436"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182504/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144118514","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":"Overall Survival with Apalutamide Versus Enzalutamide in Metastatic Castration-Sensitive Prostate Cancer.","authors":"Mehmet A Bilen, Benjamin Lowentritt, Ibrahim Khilfeh, Carmine Rossi, Shawn Du, Frederic Kinkead, Lilian Diaz, Dominic Pilon, Lorie Ellis, Neal D Shore","doi":"10.1007/s12325-025-03207-6","DOIUrl":"10.1007/s12325-025-03207-6","url":null,"abstract":"<p><strong>Introduction: </strong>Survival outcomes associated with different androgen receptor pathway inhibitors (ARPIs) prescribed for the treatment of metastatic castration (hormone)-sensitive prostate cancer (mCSPC) have not been directly compared. The objective of this study was to compare overall survival (OS) by 24 months among ARPI-naïve patients with mCSPC initiating apalutamide or enzalutamide.</p><p><strong>Methods: </strong>A retrospective, causal longitudinal inverse probability of treatment weighted analysis was conducted to compare OS between patients initiating apalutamide or enzalutamide between December 2019 and December 2023 using de-identified linked US clinical and insurance claims data. Patients were excluded if they had prior exposure to ARPIs, had evidence of castration resistance, had < 12 months of database activity prior to ARPI initiation, were diagnosed with other primary cancers, or were treated with other advanced prostate cancer (PC)-related treatment (except docetaxel). Using an intention-to-treat approach, weighted Cox proportional hazards models were used to compare OS by 24 months between patients treated with apalutamide or enzalutamide (primary analyses; exploratory analyses used all available follow-up).</p><p><strong>Results: </strong>Overall, 1810 and 1909 ARPI-naïve patients who initiated apalutamide or enzalutamide, respectively, were included. Measured baseline characteristics between cohorts were well balanced after weighting (for both: mean age 73.0 years, ~ 60% white, ~ 23% black or African American, ~ 78% Medicare-insured, mean Quan-CCI 8.6, ~ 20% with visceral metastasis, 56.2% with de novo PC). At 24 months post index, there was a statistically significant 23% reduction in the risk of mortality among patients who initiated apalutamide compared with enzalutamide (hazard ratio [HR] 0.77; 95% confidence interval [CI] 0.62, 0.96; p = 0.019). Results remained consistent when using all available follow-up metrics (HR 0.77; 95% CI 0.64, 0.93; nominal p = 0.008).</p><p><strong>Conclusion: </strong>In this head-to-head causal analysis among ARPI-naïve patients with mCSPC, treatment with apalutamide resulted in better survival outcomes at 24 months compared with enzalutamide. Longer follow-up studies are required to fully determine the therapeutic comparator impact of these agents.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"3437-3454"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182483/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172356","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":"Dupilumab Efficacy in Children with Atopic Dermatitis with Different Phenotypes and Endotypes: A Case Series.","authors":"Ana B Rossi, Adriana M Mello, Joseph Zahn","doi":"10.1007/s12325-025-03150-6","DOIUrl":"10.1007/s12325-025-03150-6","url":null,"abstract":"<p><strong>Introduction: </strong>Atopic dermatitis (AD) is a prevalent disease in infants and young children worldwide. Dupilumab has been shown to rapidly and significantly improve AD signs, symptoms, and quality of life in pediatric patients with moderate-to-severe AD.</p><p><strong>Methods: </strong>In LIBERTY AD PRESCHOOL, a randomized, double-blind, placebo-controlled, phase 3 clinical trial, patients aged 6 months to 5 years with moderate-to-severe AD received subcutaneous dupilumab or matched placebo every 4 weeks for 16 weeks. All patients received concomitant low-potency topical corticosteroids. Here, we present 12 photographic cases of patients with different phenotypes and endotypes in the dupilumab group, representative of the study population, before and after treatment. Each case is presented with clinical outcome measures of AD severity and quality of life, as well as relevant biomarkers, with percent improvement from baseline.</p><p><strong>Results: </strong>Treatment with dupilumab led to visible improvements in signs of lichenification, erythema, excoriations, skin dryness, and oozing/crusting. Clinically meaningful improvements in the measured outcomes were observed in most of the patients, including AD extent and severity, clinical lesions, itch, sleep loss, frequency of symptoms, and quality of life. These improvements were associated with substantial reductions in AD-related biomarkers.</p><p><strong>Conclusion: </strong>Treatment with dupilumab improved signs, symptoms, and quality of life, and reduced AD-related serum biomarkers in young children with moderate-to-severe AD with different phenotypes and endotypes.</p><p><strong>Trial registration: </strong>The trial was registered with ClinicalTrials.gov with ID number NCT03346434 on November 17, 2017. Video abstract available for this article. Video abstract (MP4 1,02,609 KB).</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"3186-3206"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182520/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143959572","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":"Real-World Insights on Satisfaction and Experience with Acthar Gel via SelfJect (RISE™): A Cross-Sectional Patient Survey.","authors":"Jas Bindra, Ishveen Chopra, Kyle Hayes, John Niewoehner, Mary Panaccio, Sheila Elliott, Bobby Trawick, George J Wan","doi":"10.1007/s12325-025-03232-5","DOIUrl":"10.1007/s12325-025-03232-5","url":null,"abstract":"<p><strong>Introduction: </strong>Acthar^® Gel Single-Dose Pre-filled SelfJect™ Injector (\"Acthar Gel via SelfJect\") is approved by the US Food and Drug Administration for appropriate patients with chronic and acute inflammatory and autoimmune conditions. It has fewer steps than a multi-dose vial. This survey assessed patients' perceptions of their experience with this device, including their satisfaction, confidence, convenience, and ease of use, as well as persistence and compliance.</p><p><strong>Methods: </strong>Real-World Insights on Patient Satisfaction and Experience with Acthar Gel via SelfJect (RISE™) gathered responses via a non-interventional, observational, cross-sectional online survey with a pre-specified protocol (November 2024-January 2025). Eligible participants were aged ≥18 years, had a diagnosis of an indication of Acthar Gel based on the prescribing information, and had used Acthar Gel via SelfJect for ≥6 self-injections during the survey.</p><p><strong>Results: </strong>Fifty-four participants completed the survey (mean age 55.4 years, 76% women). Thirty-three percent (18/54) had chronic or recurring ocular inflammatory disease, and 26% (14/54) had rheumatoid arthritis. Thirty-nine percent (21/54) reported dexterity or visual problems, and 39% (21/54) had prior experience with Acthar Gel multi-dose vial. On average, administering Acthar Gel via SelfJect took 3.4 min. Overall, 91% (49/54) of participants reported that they were satisfied or very satisfied with Acthar Gel via SelfJect; 89% (48/54) felt very or extremely confident injecting with Acthar Gel via SelfJect; and 91% (49/54) found it to be convenient or very convenient. Seventy-six percent (41/54) felt that it was very or extremely easy to self-inject with this device. Eighty-seven percent (47/54) perceived that they were likely or very likely to maintain persistence and compliance with this device.</p><p><strong>Conclusions: </strong>Survey participants reported a favorable experience with Acthar Gel via SelfJect, with a high level of satisfaction, confidence, convenience, and ease of use. Participants reported perceptions of anticipated positive persistence and compliance with this device, which may correlate with the possibility of improved continuity of care.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"3386-3399"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144118516","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":"Progressive Pulmonary Fibrosis: Current Status in Terminology and Future Directions.","authors":"Yasuhiro Kondoh, Yoshikazu Inoue","doi":"10.1007/s12325-025-03215-6","DOIUrl":"10.1007/s12325-025-03215-6","url":null,"abstract":"<p><p>The latest clinical practice guidelines for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) were jointly published by the American Thoracic Society (ATS), European Respiratory Society (ERS), Japanese Respiratory Society (JRS), and Asociacion Latinoamericana de Thorax (ALAT) in 2022, and a new term-\"PPF\"-has been proposed to describe patients with non-IPF fibrosing interstitial lung diseases (ILDs), with defined criteria. However, the proposal of this new term has caused confusion amongst experts at a time when use of the term \"progressive fibrosing interstitial lung disease\" (PF-ILD), proposed in the phase 3 INBUILD trial of nintedanib, has become widely adopted by pulmonologists and rheumatologists in clinical practice. In this commentary, we discuss the background and concepts underpinning the terms PPF and PF-ILD and seek to provide pulmonologists and rheumatologists with a deeper understanding of the concept of PPF.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":"2988-3001"},"PeriodicalIF":3.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182512/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144092507","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}