Advances in Therapy最新文献

{"title":"Safety of Ravulizumab Use in Pregnancy: Insights from a Global Pharmacovigilance Analysis.","authors":"Cynthia Carrillo Infante, Arshad Mujeebuddin, Meghana Koneru, Cory Williams","doi":"10.1007/s12325-026-03816-9","DOIUrl":"https://doi.org/10.1007/s12325-026-03816-9","url":null,"abstract":"<p><strong>Introduction: </strong>Ravulizumab, a second-generation terminal complement C5 inhibitor (engineered from eculizumab) is approved for the treatment of rare complement-mediated disorders paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). While eculizumab has an established pregnancy safety profile across these indications, data on ravulizumab during pregnancy and lactation remain limited. This analysis evaluated pregnancy-related safety and outcomes following ravulizumab exposure using global post-marketing data.</p><p><strong>Methods: </strong>All globally reported pregnancy cases with ≥ 1 documented ravulizumab dose were identified from the Alexion pharmacovigilance database (December 21, 2018 through December 31, 2025). Descriptive statistics were used to summarize patient demographics, treatment patterns, and pregnancy outcomes.</p><p><strong>Results: </strong>The 354 pregnancy cases with ≥ 1 dose of ravulizumab exposure that were identified include all reported cases: PNH (n = 160), aHUS (n = 72), no condition reported (n = 45), gMG (n = 38), NMOSD (n = 34), and other (n = 5). Among cases with pregnancy outcome data (n = 106), the ravulizumab exposure included full-pregnancy exposure (17.0%), ravulizumab-to-eculizumab switch during pregnancy (24.5%), and incomplete exposure details reported (58.5%). Pregnancy outcomes included 68 live births (64.2%), 29 spontaneous abortions (27.4%), 3 fetal deaths (2.8%), and 6 elective terminations (5.7%). For a nested subset of 44 post-marketing cases with detailed ravulizumab exposure data, as well as information on pregnancy outcomes (PNH [n = 26], aHUS [n = 10], gMG [n = 3], NMOSD [n = 3], and not reported [n = 2]), live births were reported in 32 cases (72.7%), spontaneous abortions in 11 cases (25.0%), and elective terminations in 1 case (2.3%).</p><p><strong>Conclusion: </strong>This post-marketing analysis provides real-world insights on ravulizumab exposure during pregnancy and suggests favorable outcomes and no unexpected safety signals similar to eculizumab. The findings may support shared decision-making between prescribers and patients when considering the ravulizumab use during pregnancy. An ongoing global observational ravulizumab pregnancy study will further inform the safety profile (NCT06312644).</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149147263","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":"Correction: Risk of protein intake deficiency during treatment with GLP-1 and GIP/GLP-1 receptor agonists: considerations for secondary sarcopenia.","authors":"Konstantinos Prokopidis, Stefano Cacciatore, Stephen D Anton, John A Batsis","doi":"10.1007/s12325-026-03813-y","DOIUrl":"https://doi.org/10.1007/s12325-026-03813-y","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149147898","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":"Estimating Long-Term Organ Damage Risk in Patients with Congenital Thrombotic Thrombocytopenic Purpura Receiving Recombinant ADAMTS13 Prophylaxis: A Structured Expert Elicitation Study.","authors":"Amie Padhiar, Colin Burke, Mathilde Puls, Annie Berkley, Oliver Heard, Harneet Kaur, Harold Wolff, Linda Murphy","doi":"10.1007/s12325-026-03786-y","DOIUrl":"https://doi.org/10.1007/s12325-026-03786-y","url":null,"abstract":"<p><strong>Introduction: </strong>Longitudinal real-world data in congenital thrombotic thrombocytopenic purpura (cTTP) are scarce. Structured expert elicitation (SEE) was conducted to estimate the projected risk of long-term organ damage in children and adolescents and in adult patients with cTTP receiving recombinant ADAMTS13 (rADAMTS13) prophylaxis, without history of organ damage.</p><p><strong>Methods: </strong>Six UK clinicians with experience in managing cTTP provided individual judgments via an online SEE survey. The probability of developing ≥ 1 long-term organ damage outcomes, including major organ events such as stroke/transient ischemic attack, myocardial infarction, and chronic kidney disease, at predefined ages was estimated. Responses were collected using the quartile method and combined via linear opinion pooling.</p><p><strong>Results: </strong>In children and adolescents, median (95% credible interval) estimated cumulative long-term organ damage projected risks were 3.25% (1.09-20.26%) by age 15 years, 4.48% (0.86-16.11%) by 20 years, and 5.76% (0.96-19.20%) by 25 years. In adults, estimated risks were 4.85% (0.59-16.96%) by 45 years and 7.42% (1.47-29.29%) by 55 years.</p><p><strong>Conclusion: </strong>Experts consistently projected lower long-term risks of organ damage with continuous rADAMTS13 prophylaxis, although these estimates remain hypothesis-generating. These expert-derived estimates provide structured insights into potential long-term outcomes associated with rADAMTS13 prophylaxis, which can inform future research, health technology assessments, and evidence-generation efforts in cTTP. Prospective empirical studies are required to confirm the findings. Graphical Abstract available for this article.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149147200","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":"Summary of Research: Two-Stage Estimation of Overall Survival in the Phase 3 CheckMate 9ER Trial, Adjusting for the Impact of Subsequent Therapy.","authors":"Marc-Oliver Grimm, Emma Karim, Venediktos Kapetanakis, Mickael Lothgren, Alessia Ogareva, Pragya Shukla, James Truscott, Camillo Porta","doi":"10.1007/s12325-026-03773-3","DOIUrl":"https://doi.org/10.1007/s12325-026-03773-3","url":null,"abstract":"<p><p>This is a summary of a post hoc analysis of the phase 3 CheckMate 9ER study. CheckMate 9ER demonstrated a significant overall survival (OS) benefit for first-line cabozantinib plus nivolumab (CaboNivo) versus sunitinib (SUN) in patients with advanced renal cell carcinoma (aRCC). However, receiving subsequent therapy can distort true OS benefits. This analysis used two-stage estimation (TSE) to model a hypothetical scenario to assess the OS benefit of CaboNivo versus SUN, removing the effect of subsequent treatment. In stage 1, multivariate modeling estimated the extent to which subsequent therapy extended OS in patients who discontinued their original study treatment. In stage 2, an adjusted hazard ratio (HR) for OS assessed the relative impact of CaboNivo versus SUN without the effects of subsequent treatment. TSE-adjusted counterfactual median OS [95% confidence interval (CI)] was 42.7 (38.4-49.1) months for CaboNivo and 21.3 (18.1-24.9) months for SUN [HR 0.53 (95% CI 0.43-0.65; p < 0.001)]; unadjusted estimates were 46.5 (40.6-53.7) months for CaboNivo and 35.5 (29.2-42.8) months for SUN [HR 0.79 (95% CI 0.65-0.96; p = 0.0196)]. Removing the effect of subsequent therapy increased the estimated OS benefit of CaboNivo versus SUN, reinforcing CaboNivo as a first-line standard of care for aRCC.Trial Registration NCT03141177.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149134702","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":"Summary of Research: Health-Related Quality-of-Life Profile and Clinical Outcomes in First-Line Advanced Renal Cell Carcinoma: A Modeling Analysis Based on the CheckMate 9ER Study.","authors":"David Cella, Michael Derosa, Lysbeth Floden, Rachel H Giles, Mickael Lothgren, Alessia Ogareva, Thomas Powles, Intan Purnajo, Toni K Choueiri, Cristiane Bergerot, Robert J Motzer, Jens Bedke","doi":"10.1007/s12325-026-03780-4","DOIUrl":"10.1007/s12325-026-03780-4","url":null,"abstract":"<p><p>This is a summary of a post hoc analysis of the phase 3 CheckMate 9ER study. CheckMate 9ER demonstrated a significant improvement in progression-free survival and overall survival with first-line cabozantinib plus nivolumab (CaboNivo) versus sunitinib (SUN) in patients with advanced renal cell carcinoma. First-line CaboNivo also maintained or improved health-related quality of life (HRQoL) versus SUN. However, the relationship between clinical efficacy and patient treatment experience remains underexplored. This post hoc analysis investigated the relationship between changes in HRQoL and clinical outcomes, and used latent class analysis (LCA) to identify symptom-based profiles at week 13 predictive of disease course. Early worsening of bone pain [hazard ratio (HR) 1.45, p = 0.010] and sleep quality (HR 1.45, p = 0.007) were associated with increased risk of mortality. LCA identified three distinct patient profiles at week 13; those in the limited symptoms class were more likely to have received CaboNivo and to have a longer treatment duration, and were generally less likely to experience disease progression than patients in other symptom classes. This analysis demonstrated a link between early changes in patient-reported outcomes and efficacy in the CheckMate 9ER study, highlighting the importance of balancing efficacy, tolerability, and HRQoL to optimize treatment outcomes and support shared decision-making.Trial Registration NCT03141177.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149134700","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 Characteristics, Treatment Patterns, and Clinical Outcomes of Patients Initiated on Macitentan/Tadalafil Single-Tablet Combination Therapy in the United States.","authors":"Mrinalini Krishnan, Nicholas A Kolaitis, Marinella Sandros, Ankita D Adhia, David Lopez, Natalie Gearhart, Alexander K Salomon, John J Ryan","doi":"10.1007/s12325-026-03811-0","DOIUrl":"https://doi.org/10.1007/s12325-026-03811-0","url":null,"abstract":"<p><strong>Introduction: </strong>Pulmonary arterial hypertension (PAH) is life-threatening and often complicated by comorbidities. Although endothelin receptor antagonist (ERA) plus phosphodiesterase 5 inhibitor (PDE5i) dual therapy is guideline recommended, real-world treatment varies. Macitentan/tadalafil fixed-dose combination (M/T FDC) is a once-daily single-tablet dual therapy approved in March 2024. This study characterized patient characteristics, treatment patterns, and exploratory outcomes.</p><p><strong>Methods: </strong>In this retrospective chart review, 50 US healthcare providers reviewed patient charts during structured interviews. Patients were categorized as ERA/PDE5i/soluble guanylate cyclase (sGC)-naïve, escalated from monotherapy, or switched from ERA plus PDE5i/sGC combination therapy. Outcomes included baseline characteristics, treatment patterns, reasons for M/T FDC initiation, 6-min walk distance (6MWD), brain natriuretic peptide (BNP), and PAH-related hospitalizations.</p><p><strong>Results: </strong>Providers reviewed 144 charts; 83.3% of patients had at least one comorbidity. Overall, 51.4% switched from dual therapy, 28.5% escalated from monotherapy, and 20.1% were ERA/PDE5i/sGC-naïve. Naïve patients frequently had connective tissue disease-associated or drug- and toxin-induced PAH. Reduced pill burden was the leading reason for initiation. Among patients without concomitant prostacyclin or activin signaling inhibitor initiation or escalation and with paired baseline and follow-up data, mean favorable numerical changes were observed: 6MWD increased by 51.0 m (355.2-406.2; n = 36), BNP decreased by 167.8 pg/mL (357.0-189.2; n = 29), and PAH-related hospitalizations decreased from 0.18 to 0.05 events per patient (n = 65) over approximately 3 months.</p><p><strong>Conclusion: </strong>M/T FDC is being adopted across diverse US PAH populations, including patients with comorbidities, most commonly to reduce pill burden and simplify treatment administration. Exploratory descriptive analyses showed favorable numerical changes in selected clinical parameters among patients with available paired data. However, because the study was retrospective, non-comparative, and not powered for hypothesis testing, these findings should be interpreted as hypothesis-generating. These results provide early insights into use of M/T FDC use and identify areas for future longitudinal and comparative research.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149134685","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":"Retifanlimab Versus Avelumab for the Treatment of Metastatic Merkel Cell Carcinoma: A Matching-Adjusted Indirect Comparison Analysis.","authors":"Camilla Porta, Gianni Ghetti, Massimiliano Povero","doi":"10.1007/s12325-026-03802-1","DOIUrl":"https://doi.org/10.1007/s12325-026-03802-1","url":null,"abstract":"<p><strong>Introduction: </strong>Merkel cell carcinoma (MCC) is a rare and aggressive skin cancer with poor prognosis and low survival rate. Retifanlimab and avelumab are monoclonal immune checkpoint inhibitor antibodies approved for metastatic or recurrent locally advanced MCC. The objective of this study was to analyze clinical outcomes with retifanlimab versus avelumab using a matching-adjusted indirect comparison (MAIC).</p><p><strong>Methods: </strong>Individual patient data from the phase 2 POD1UM-201 trial (NCT03599713, data cutoff July 2024) were used for the retifanlimab treatment group, and aggregate data from part B of the phase 2 JAVELIN Merkel 200 trial (NCT02155647) were used for the avelumab treatment group. Key prognostic factors/effect modifiers identified from published literature (age, Eastern Cooperative Oncology Group performance status, programmed cell death ligand 1 status, and disease characteristics) were selected for adjustment in the base case. Scenario analyses were also conducted, including additional covariates. An unanchored MAIC was conducted for overall survival (OS), progression-free survival (PFS), and overall response rate (ORR).</p><p><strong>Results: </strong>After weighing characteristics, the effective sample size of the adjusted retifanlimab population was approximately 42 patients (47% of original sample size). In the base case analysis, weighted hazard ratio (95% CI) numerically favored retifanlimab versus avelumab for OS (0.800 [0.492, 1.303]) and was statistically significant for PFS (0.590 [0.396, 0.881]), with the latter driven by separation during the first 2 months of treatment. Weighted odds ratio numerically favored retifanlimab for ORR (1.871 [0.896, 3.908)]. Scenario analyses generally confirmed base case analyses.</p><p><strong>Conclusion: </strong>Results of this MAIC generally favored retifanlimab compared with avelumab, including a statistically significant difference in PFS and numerical trends in OS and ORR, building on the clinical evidence supporting retifanlimab for the treatment of metastatic MCC. These findings are subject to residual confounding and should be interpreted with caution.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149126531","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":"Confounder Identification in Diffuse Large B-Cell Lymphoma: Findings from an Expert Panel of German and Austrian Hematologists.","authors":"Jan-Michel Heger, Philipp Gödel, Stefan Habringer, Ulrich Jäger, Nadine Kutsch, Bastian von Tresckow, Ruiyu Zhang, Stefanie Rungaldier, Julia Oddsdottir, Maria Zacharioudaki, Jörg Mahlich","doi":"10.1007/s12325-026-03820-z","DOIUrl":"https://doi.org/10.1007/s12325-026-03820-z","url":null,"abstract":"<p><strong>Introduction: </strong>Robust confounder identification is essential for valid indirect treatment comparisons (ITCs), particularly in relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL), where treatment pathways are heterogeneous and comparative evidence between chimeric antigen receptor T-cell (CAR-T) therapies remains limited. This study aimed to validate clinically relevant prognostic factors and treatment-effect modifiers for comparative effectiveness analyses in DLBCL using a structured expert consensus approach.</p><p><strong>Methods: </strong>Candidate variables were identified through a prior systematic literature review and refined by clinical review. A panel of six experienced hematologists and oncologists from Germany and Austria participated in a two-round expert elicitation process. They assessed the importance of each variable as a prognostic factor and/or treatment-effect modifier using a three-point Likert scale. Variables were categorized into relevance tiers according to predefined consensus thresholds and subsequently allocated to specific clinical endpoints, including progression-free survival (PFS), overall survival (OS), response outcomes, and CAR-T-related toxicities.</p><p><strong>Results: </strong>Several variables achieved high consensus as prognostic factors. Tier A prognostic factors included ECOG performance status, age, disease histology, International Prognostic Index (IPI), disease stage, elevated lactate dehydrogenase (LDH), bulky disease, extranodal involvement, tumor burden, primary refractory disease, number of relapses, time to relapse, and prior lines of therapy. Unanimous agreement was observed for ECOG performance status, IPI, disease histology, disease stage, elevated LDH, and primary refractory disease as prognostic factors for survival outcomes. Compared with prognostic factors, substantially lower agreement was observed for treatment-effect modifiers, particularly for response-based outcomes. Tier A-effect modifiers included primary refractory disease, time to first relapse, best response to prior therapy, bridging therapy, CAR-T product type, and CAR-T-related response characteristics. Bridging therapy was the only modifier achieving unanimous agreement for complete and overall response rates. Tumor burden-related variables were considered important for both efficacy and toxicity outcomes, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.</p><p><strong>Conclusions: </strong>Clinicians demonstrated strong consensus regarding established prognostic factors related to disease aggressiveness, treatment resistance, patient fitness, and tumor burden in R/R DLBCL. Agreement on treatment-effect modifiers was more limited, reflecting the limited evidence currently available.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149126518","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":"Capturing the Patient Voice: Qualitative Assessment of Daridorexant's Impact on Patients with Insomnia and Comorbid Nocturia from a Randomized, Placebo-Controlled Crossover Trial.","authors":"Dario Arnaldi, Donald L Bliwise, Duygu Alpan, Patricia Koochaki, Michael Meinel, Antonio Olivieri, Racheal Rowles, Annyesha Satapathy, Katharina Lederer","doi":"10.1007/s12325-026-03803-0","DOIUrl":"https://doi.org/10.1007/s12325-026-03803-0","url":null,"abstract":"<p><strong>Introduction: </strong>Chronic insomnia frequently co-exists with nocturia, contributing to impaired sleep quality and reduced health-related quality of life. A recent randomized cross‑over study found that daridorexant improved sleep duration, insomnia severity, daytime functioning, and nocturnal voiding in patients with chronic insomnia and nocturia. To complement these quantitative outcomes, the study incorporated qualitative free‑text surveys capturing patients' lived experiences, addressing an existing gap in patient‑reported insights for this multifaceted condition. Here, we report these qualitative findings assessed at baseline and post‑treatment.</p><p><strong>Methods: </strong>This double-blind, placebo-controlled, two-way crossover trial of patients with insomnia disorder and comorbid nocturia included 60 adults ≥ 55 years with ≥ 3 months of insomnia symptoms, an Insomnia Severity Index ≥ 13, and ≥ 3 nocturnal voids per night. Patients were randomized to daridorexant 50 mg or placebo for 4 weeks, followed by crossover after a washout period. Free‑text surveys at baseline and after each treatment period captured patient‑reported impacts of their conditions on daily life and perceived changes during treatment.</p><p><strong>Results: </strong>At baseline, patients reported broad negative impacts across physical, emotional, cognitive, and daily functioning domains. During treatment, more patients reported improvements in sleep (87% vs. 50%) and nocturia (79% vs. 32%) on daridorexant than on placebo, and improvements in functioning, particularly physical, cognitive, emotional, and daily activities.</p><p><strong>Conclusion: </strong>Patients reported meaningful improvements with daridorexant 50 mg, including better sleep, reduced nocturia, and enhanced physical, cognitive, and emotional functioning. Daridorexant consistently elicited more positive patient‑reported changes than placebo. These findings highlight the value of free‑text surveys for capturing patient‑centered outcomes.</p><p><strong>Clinical trial registration: </strong>NCT05597020.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149102961","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":"Questionable Evidence for Berberine in Polyendocrine Metabolic Ovarian Syndrome: An Integrity-Based Reappraisal.","authors":"Eshani Perera, Evangeline Mantzioris, Lisa Moran, Nicole Scannell, Anthony Villani, Vibhuti Rao, Aya Mousa, Madeline Flannagan, Carolyn Ee","doi":"10.1007/s12325-026-03799-7","DOIUrl":"https://doi.org/10.1007/s12325-026-03799-7","url":null,"abstract":"<p><strong>Introduction: </strong>Prior systematic reviews suggest that berberine may match or exceed the effects of metformin on metabolic and hormonal outcomes in polyendocrine metabolic ovarian syndrome (PMOS; previously referred to as polycystic ovary syndrome). This review aimed to assess the reported efficacy of berberine and act as an integrity-based reappraisal of available evidence in women with PMOS.</p><p><strong>Methods: </strong>A systematic review was conducted in accordance with PRISMA guidelines and prospectively registered. MEDLINE, EMBASE, the Cochrane Library and CINAHL were searched from inception to October 2025. Randomised controlled trials examining berberine supplementation in women with polycystic ovary syndrome were eligible for inclusion. Outcomes of interest included metabolic, hormonal, and anthropometric measures. All eligible trials underwent independent integrity assessment using two validated frameworks: the Research Integrity in Guidelines and Evidence Synthesis framework and the Trustworthiness in Randomised Controlled Trials checklist. Studies rated as moderate or high risk of compromised integrity were excluded from quantitative synthesis. Authors were contacted for clarification prior to exclusion.</p><p><strong>Results: </strong>All four trials (n = 452) that met eligibility criteria were independently rated as high risk for compromised integrity, and were not included in quantitative synthesis. Concerns included absent trial registration, implausible recruitment timelines, and potential duplication of data. No clarifying responses were received. While improvements in insulin resistance, lipid profiles, and androgen markers were reported, these findings cannot be considered reliable given the identified concerns.</p><p><strong>Conclusions: </strong>Our review demonstrates that reliable conclusions regarding the efficacy of berberine in PMOS cannot be drawn from the current evidence base.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149102547","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}