Advances in Therapy最新文献

{"title":"Tabelecleucel in Post-transplant Epstein-Barr Virus-Associated Lymphoproliferative Disease: Patient and Oncologist Perspectives.","authors":"Antonio Perez-Martinez, Anarbella Lastra Rodrigo, Esther Ramos Boluda, Berta Gonzalez Martinez, Alida Alcolea Sánchez, Francisco Hernandez Oliveros","doi":"10.1007/s12325-025-03246-z","DOIUrl":"https://doi.org/10.1007/s12325-025-03246-z","url":null,"abstract":"<p><p>This article is coauthored by the parents of a young patient and the medical team who cared for her.Together, they recount their shared experience with tabelecleucel, a novel therapy for Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV + PTLD). The patient's parents offer a deeply personal perspective, reflecting on several years of struggle marked by unwavering hope and trust in the medical professionals who have supported their daughter throughout her journey. The pediatric hemato-oncologist who advocated for the use of tabelecleucel described the case as unique worldwide.Their joint testimony tells the remarkable story of how tabelecleucel, despite an initially unclear biological rationale, achieved a reversal of a severe case of EBV + PTLD that had progressed to a point considered beyond recovery and associated with a dire prognosis.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144186209","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":"The Potential Impact of Sotatercept on Long-Term Disability in Commercially Insured Patients with Pulmonary Arterial Hypertension in the United States: An Exercise in Actuarial Modeling.","authors":"Anna Watzker, Adnan Alsumali, Christine Ferro, Xun Jun Li, Gabriela Dieguez, Clare Park, Jestinah Chevure, Dominik Lautsch, Karim El-Kersh","doi":"10.1007/s12325-025-03224-5","DOIUrl":"https://doi.org/10.1007/s12325-025-03224-5","url":null,"abstract":"<p><p>Pulmonary arterial hypertension (PAH) is a progressive disease commonly leading to functional impairment that can impact the ability to work. In the US, disabled workers often qualify for employer-sponsored long-term disability (LTD) benefits and Social Security Disability Insurance (SSDI). We used actuarial techniques to model scenarios of disability costs associated with PAH, with and without treatment with sotatercept, a first-in-class therapy that slowed PAH disease progression in clinical trials. Annual disability costs were measured as the net present value of lifetime benefits per claimant with PAH and multiplied by the number of new disability insurance claimants with PAH nationwide each year. We estimated that sotatercept therapy would result in disability benefit costs savings in the US ranging from $86.9 to $245.5 million per year or 32-90% of current costs for LTD and SSDI benefits associated with PAH. Our findings suggest that therapies that potentially prevent disability and enable disabled patients to return to work can help reduce disability costs.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144186223","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":"Risk Factors Associated with Suboptimal Real-World Outcomes in Patients with EGFR-Mutated Non-Small Cell Lung Cancer Treated with Front-Line Recommended Therapy.","authors":"Alexander I Spira, Tao Ran, Iris Lin, Cindy Chen, Andy He, Neel Belani, Shawn Du","doi":"10.1007/s12325-025-03234-3","DOIUrl":"https://doi.org/10.1007/s12325-025-03234-3","url":null,"abstract":"<p><strong>Introduction: </strong>Osimertinib, a recommended front-line (1L) treatment option for non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) mutations [exon 19 deletion (ex19del)/exon 21 L858R substitution (L858R)], demonstrated significantly improved progression-free survival and overall survival (OS) compared with other EGFR tyrosine kinase inhibitors in the FLAURA trial. However, most patients developed secondary resistance, and subpopulations experienced disparate outcomes. This retrospective, observational study aimed to describe real-world treatment outcomes along with patient clinical characteristics associated with different clinical outcomes among patients with EGFR-mutated (ex19del/L858R) NSCLC who received osimertinib as 1L therapy.</p><p><strong>Methods: </strong>This retrospective cohort study of patient clinical and genomic data from a de-identified, US-based, nationwide NSCLC clinico-genomic database evaluated OS, time-to-next-treatment (TTNT), and time-to-discontinuation (TTD) among patients with EGFR-mutated NSCLC who received 1L osimertinib monotherapy and identified patient characteristics associated with differences in clinical outcomes as related to real-world 1L osimertinib use.</p><p><strong>Results: </strong>Among 703 identified patients, mean age was 69 years, 67% of patients were female, and 53% were White. Median OS was 29.8 months, TTNT was 17.6 months, and TTD was 15.9 months. A large majority of the overall population had risk factors associated with suboptimal real-world outcomes (97% for OS, 95% for TTNT, and 87% for TTD), even among patients with ECOG PS scores of 0-1. Key individual risk factors identified included metastases of the liver, bone, and central nervous system; ECOG PS scores ≥ 2; and TP53, CDK-4, and EGFR L858R mutations.</p><p><strong>Conclusions: </strong>More than 90% of the assessed real-world patients with EGFR-mutated (ex19del/L858R) NSCLC who received 1L osimertinib had risk factors associated with suboptimal outcomes, highlighting the urgent unmet need for new treatments and emphasizing the importance of administering the most effective therapy in the 1L setting.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172359","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":"Matching-Adjusted Indirect Comparison of Osilodrostat Versus Metyrapone for the Treatment of Cushing's Syndrome.","authors":"Conor Hickey, Beatrice Gueron, Fabian Schmidt, Emma Tyas, Grzegorz Binowski, Rosario Pivonello","doi":"10.1007/s12325-025-03229-0","DOIUrl":"https://doi.org/10.1007/s12325-025-03229-0","url":null,"abstract":"<p><strong>Introduction: </strong>Cushing's syndrome (CS) is a rare, chronic condition caused by prolonged exposure to elevated levels of circulating cortisol, and characterized by high morbidity and mortality. The primary treatment option for CS is surgery; however, medical therapy may be useful when surgery is unsuitable, refused, or has not been curative, or a rapid control of hypercortisolism is required. While osilodrostat and metyrapone are two treatments for controlling cortisol levels, they have not been compared directly in a clinical trial. This study evaluated the comparative efficacy and tolerability of osilodrostat versus metyrapone for the treatment of CS using indirect treatment comparison methods.</p><p><strong>Methods: </strong>Unanchored matching-adjusted indirect comparison was used to synthesize relative treatment effects by reweighting patient-level data from two clinical trials for osilodrostat to published aggregate data for metyrapone. Efficacy endpoints included complete response (CR), defined as mean urinary free cortisol ≤ 1.0 × the upper limit of normal, at Weeks 12, 24, and 36. Tolerability endpoints included all-cause treatment discontinuation and treatment discontinuation due to lack of efficacy (LoE) or adverse events (AEs).</p><p><strong>Results: </strong>The base case analysis demonstrated that osilodrostat provides increased odds of CR versus metyrapone at Week 12 [odds ratio (OR) 2.75; 95% confidence interval (CI) 1.29, 5.88], Week 24 (OR 3.28; 95% CI 1.58, 6.84) and Week 36 (OR 10.50; 95% CI 1.84, 59.96), implying a greater proportion of patients experience normalized cortisol levels at these time-points. Although the base case analysis showed that the odds of all-cause discontinuation and discontinuation due to LoE or AEs were numerically lower for osilodrostat, the evidence was insufficient to show a statistically significant difference.</p><p><strong>Conclusion: </strong>These analyses show that osilodrostat increases the odds of achieving CR at Weeks 12, 24, and 36 versus metyrapone, demonstrating that osilodrostat is a more efficacious treatment option for normalizing cortisol levels in CS patients.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172316","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":"Mobility and Quality of Life in Children with Paediatric-Onset Hypophosphatasia Treated with Asfotase Alfa: Results from UK Managed Access Agreement.","authors":"Raja Padidela, Nick Bishop, Paul Arundel, Shona Fang, Alexandros Zygouras, M Zulf Mughal, Nick Shaw, Vrinda Saraff","doi":"10.1007/s12325-025-03225-4","DOIUrl":"https://doi.org/10.1007/s12325-025-03225-4","url":null,"abstract":"<p><strong>Introduction: </strong>Hypophosphatasia (HPP) is a rare, inherited metabolic bone disease with a high degree of morbidity and mortality in children. Asfotase alfa is an enzyme replacement therapy for HPP reimbursed in the UK since 2017 under a Managed Access Agreement (MAA). This analysis assessed the effectiveness and safety of asfotase alfa in children < 18 years of age.</p><p><strong>Methods: </strong>The MAA was a prospective, longitudinal data collection in children with paediatric-onset HPP. Effectiveness outcomes were evaluated in children who were treated with asfotase alfa for ≥ 6 months. Data were collected on respiratory support, growth, mobility, motor development, analgesic use, quality of life, and safety at enrolment and throughout the 5-year MAA.</p><p><strong>Results: </strong>Twenty-four children enrolled in the MAA and 20 were included in the analysis. Twelve children had received asfotase alfa before enrolment through a clinical trial or compassionate use program. From baseline to month 60, the median (minimum, maximum) change in height and weight Z-scores were 0.20 (- 0.9, 1.2; n = 6) and - 0.5 (- 1.9, 1.5; n = 6), respectively. The median (minimum, maximum) percent of predicted distance walked in the 6-Minute Walk Test increased by 3.8% (- 8.6, 4.3; n = 5) at month 3 and was sustained through follow-up. Median (minimum, maximum) child- and parent-reported Pediatric Quality of Life Inventory scores were 59.2 (15.2, 91.3; n = 11) and 53.4 (16.3, 100.0; n = 18) at baseline and increased by 21.7 (5.4, 37.0; n = 3) and 16.3 (9.8, 45.7; n = 4) at month 60, respectively. Treatment-naïve children had a greater clinical response than treatment-experienced participants, who maintained their status. No deaths occurred in the study. The most common adverse events were injection site reactions, reported in 8/24 participants (33.3%).</p><p><strong>Conclusion: </strong>This analysis confirmed the clinical benefit of asfotase alfa in children with HPP. Asfotase alfa was well tolerated, with no new safety signals identified.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172317","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":"Economic Burden and Provider Referral Patterns Among Patients with Unresectable Stage III EGFR-Mutated NSCLC Receiving Chemoradiotherapy in the United States.","authors":"YongJin Kim, Yong Zhu, Kristin J Moore, Mary DuCharme, Dan James, Arber Shehu, Yanique Rattigan-Brown, Kim Ohaegbulam","doi":"10.1007/s12325-025-03239-y","DOIUrl":"https://doi.org/10.1007/s12325-025-03239-y","url":null,"abstract":"<p><strong>Introduction: </strong>Among patients with unresectable stage III non-small cell lung cancer (NSCLC), those whose tumors harbor epidermal growth factor receptor mutations (EGFRm) are associated with comparatively fewer treatment options and worse prognosis. With the recent approval of targeted treatment, characterizing the economic burden and EGFRm testing and provider referral patterns is crucial to understanding the unmet needs of these patients.</p><p><strong>Methods: </strong>This was a retrospective analysis of Optum's Market Clarity Dataset from January 1, 2018 to June 30, 2023. Eligibility criteria included diagnosis with unresectable stage III EGFRm NSCLC and chemoradiotherapy (CRT) initiation (index date) within 90 days. Primary outcomes were per patient per month (PPPM) all-cause and NSCLC-related health care resource utilization (HCRU) and costs, and EGFRm testing and provider referral patterns.</p><p><strong>Results: </strong>A total of 144 patients were followed for a median of 15.5 months; 56.3% of patients underwent EGFRm testing before CRT initiation. All-cause and NSCLC-related costs during follow-up were $28,020 and $22,816 PPPM, respectively. Ambulatory utilization was the major driver of this economic burden. Pharmacy costs accounted for $4244 (15.1%) and $3736 (16.4%) of the total all-cause and NSCLC-related costs, respectively. Between diagnosis and CRT initiation, the most common specialties visited were oncology/hematology (seen by 67.4% of patients), radiology (26.4%), pulmonology (22.2%), and cardiology (21.5%). Patients who visited three or more specialties on separate days before CRT initiation had a median time to CRT initiation of 33.0 days versus 22.0 days when patients visited multiple specialties on the same day (suggestive of a multidisciplinary care team, MDT).</p><p><strong>Conclusion: </strong>Patients with unresectable stage III EGFRm NSCLC incur substantial economic burden, especially in ambulatory HCRU and costs. With the recent approval of targeted treatment for these patients, reflex EGFRm testing in all early-stage NSCLC at diagnosis is encouraged. Our results also suggest MDT involvement may improve completeness in diagnosis and staging, resulting in acceleration of treatment planning and management.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172315","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":"Dietary Intake of Major Minerals and Trace Elements in Patients with Metabolic Dysfunction-Associated Steatotic Liver Disease: Implications for Dietary Intervention.","authors":"Vasily Isakov, Armida Sasunova, Sergey Morozov, Alexei Goncharov","doi":"10.1007/s12325-025-03238-z","DOIUrl":"https://doi.org/10.1007/s12325-025-03238-z","url":null,"abstract":"<p><strong>Introduction: </strong>An imbalanced diet is one of the leading causes of metabolic dysfunction-associated steatotic liver disease (MASLD) development. Diet modification remains the leading approach in the disease management. However, the role of minerals in MASLD development and treatment is poorly understood. In this retrospective study we compared minerals intake in patients with MASLD and age- and sex-matched controls, based on the data of a food frequency questionnaire.</p><p><strong>Methods: </strong>A retrospective database search was performed to identify eligible data of the nutritional assessment with software based on a food frequency questionnaire. The institutional medical records of the obtained cohort were then searched for medical conditions in accordance with the inclusion/exclusion criteria. On the basis of the presence of MASLD, the subjects were allocated to either MASLD or the control group. Sex- and age-matched pairs were formed for the analysis. Consumption of major minerals and trace elements was compared using non-parametric statistics.</p><p><strong>Results: </strong>Records of 15,862 subjects were screened, and the data of 226 sex- and age-matched pairs of patients with MASLD and controls were selected for the analysis. The absolute average daily intake of most of the minerals, except silicon, cobalt, molybdenum, nickel, and chromium, was greater in the MASLD group than in the control group. However, relative value (per 1000 kcal) analysis revealed that only boron intake was greater in the MASLD group (28.3 ± 38.5 vs 19.5 ± 24.7 μg/day, p = 0.013). Subjects with MASLD exceeded the recommended daily allowance (RDA) for sodium (241% of RDA), phosphorus (211%), vanadium (1576%), manganese (410%), and selenium (197%) intake, but consumed less than the recommended amounts of silicon (5% of the RDA), molybdenum (28%), fluorine (3%), zinc (91%), and chromium (37%).</p><p><strong>Conclusion: </strong>Patients with MASLD consumed greater amounts of most minerals than did the control group due to overeating. When diet modification for patients with MASLD is planned, the intake of calcium, zinc, and boron needs to be controlled, and the diet may be modified with food supplements or specific foods rich in these minerals.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172314","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-Life Growth Hormone Treatment Patterns in Children from China: A Report from Two Databases.","authors":"Wei Wu, Nan Li, Tingting Wang, Xiaoping Luo","doi":"10.1007/s12325-025-03204-9","DOIUrl":"https://doi.org/10.1007/s12325-025-03204-9","url":null,"abstract":"<p><strong>Introduction: </strong>Although recombinant human growth hormone (rhGH) treatment has been established in China for many years for treating short stature (SS), there exists a lacuna on the current status and potential problems associated with effective usage of rhGH. The objective of this study is to investigate the real-world status of rhGH treatment in Chinese children with SS and its associated factors.</p><p><strong>Methods: </strong>This multicenter, retrospective, observational study included children treated with rhGH from January 1, 2017, to April 30, 2024, from two registry databases in China, which included children who were treated with at least one rhGH owing to GH deficiency (GHD), small for gestational age (SGA), or idiopathic short stature (ISS). Data collected included baseline characteristics, the type of rhGH treatment [long-acting (LAGH) or short-acting (SAGH)], and treatment compliance.</p><p><strong>Results: </strong>A total of 41,942 children (15,975 with GHD, 24,445 with ISS, and 1522 with SGA) with a median age of 8.29 (5.88-10.64) years were included. The mean Ht SDS and BMI-SDS were - 2.51 ± 0.42 and -0.48 ± 1.11, respectively. More children started using SAGH than LAGH (79.53% vs. 20.47%). Although most children with SGA and GHD initiated with an appropriate dose, 33.26% of children with ISS started with an insufficient dose. In the total population, 42.1% of children discontinued medication. The most common reasons were irregular administration, limited medication access, refusal by patients or families, and observational discontinuation. In addition, the LAGH group exhibited a significantly longer duration of therapy compared with the SAGH group.</p><p><strong>Conclusion: </strong>In China, children with SS tended to start rhGH treatment at a relatively older age and to exhibit a high rate of treatment discontinuation. To achieve better treatment outcomes for children with SS, good adherence and guideline-recommended medication patterns need to be followed.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172358","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":"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":"https://doi.org/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":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Oral Peptide Therapeutics as an Emerging Treatment Modality in Immune-Mediated Inflammatory Diseases: A Narrative Review.","authors":"Linda Stein Gold, Kilian Eyerich, Joseph F Merola, Joana Torres, Laura C Coates, Jessica R Allegretti","doi":"10.1007/s12325-025-03213-8","DOIUrl":"https://doi.org/10.1007/s12325-025-03213-8","url":null,"abstract":"<p><p>Immune-mediated inflammatory diseases (IMIDs), such as psoriasis, psoriatic arthritis, and inflammatory bowel disease, encompass a heterogenous group of conditions associated with chronic inflammation. Systemic treatments for patients with IMIDs include parenterally delivered monoclonal antibodies (mAbs) that disrupt specific cytokine and cytokine receptor binding interactions, and orally delivered small molecules that inhibit certain enzymes involved in the regulation of inflammatory signaling. Many patients prefer oral alternatives to injectables, but currently available oral advanced therapies are less effective than mAbs and/or have tolerability concerns. Thus, an unmet need exists for additional oral treatment options for patients with IMIDs. Therapeutic peptides can be designed to possess characteristics that provide both the target selectivity typically associated with parenterally delivered mAbs and an oral route of administration. Oral peptide therapeutics are an area of intense research in several therapeutic areas, and, although some oral peptides are available for certain indications, such as diabetes, there are currently no targeted oral peptides available for the treatment of patients with IMIDs. Icotrokinra (JNJ-77242113), which is currently in development to treat patients with various IMIDs, is the first targeted oral peptide designed to selectively inhibit interleukin (IL)-23 signaling by blocking the IL-23 receptor on human immune cells. In a phase 2b study in adults with moderate-to-severe psoriasis, icotrokinra showed a significant dose-response effect versus placebo, and a tolerable safety profile at Week 16. Sustained skin clearance and no safety signals were observed through Week 52 in the extension study to the phase 2b study. Ongoing phase 2 and phase 3 clinical studies in patients with psoriasis, psoriatic arthritis, and ulcerative colitis will provide data to inform the therapeutic potential of icotrokinra to address the unmet need in these diseases.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172318","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}