Pharmacotherapy最新文献

{"title":"Pharmaceutical Considerations for Oncology Risk, Screening, and Drug Dosing in the Transgender Population.","authors":"Amy Broadwater, Kevin N Astle","doi":"10.1002/phar.70136","DOIUrl":"10.1002/phar.70136","url":null,"abstract":"<p><p>Transgender individuals make up a significant portion of the United States population, yet little data are available regarding oncologic considerations for this population. Transgender individuals may utilize gender-affirming hormone therapy to develop the desired secondary sex characteristics. Although there are few studies assessing hormone therapy and cancer risk in transgender individuals, available studies show no increased risk of cervical, ovarian, testicular, and prostate cancer associated with gender-affirming hormone therapy. Caution is advised when using estrogen therapy in transgender women who may be at higher risk for breast cancer due to family history or other considerations. Transgender patients should also be counseled on cancer screening recommendations depending on which tissues they have retained, even if those tissues are incongruous with their gender identity. Additionally, when providing oncologic care for transgender individuals, health care providers often utilize calculations for dosing and renal function that contain gender modifiers. Duration of hormone therapy can be utilized as a factor for determining whether to utilize gender assigned at birth or gender identity for these calculations. Through education and cultural awareness, health care providers can provide a simultaneously effective and gender-affirming medical experience to the transgender population.</p>","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":"46 4","pages":"e70136"},"PeriodicalIF":4.0,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147481339","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":"Models Integrating Disease Heterogeneity and MPA Exposure Predict Systemic and Renal Efficacy of Mycophenolate in Pediatric Lupus Nephritis.","authors":"Baojing Liu, Lizhi Chen, Jingxuan Lin, Xiaoman Liu, Xiaohong Liu, Zhijun Huang, Min Huang, Kejing Tang, Xiaoyun Jiang, Pan Chen","doi":"10.1002/phar.70127","DOIUrl":"10.1002/phar.70127","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to develop and validate integrated prediction models for pediatric lupus nephritis (LN) treated with a mycophenolate mofetil (MMF)-based induction regimen (combined with glucocorticoids and hydroxychloroquine), incorporating both disease heterogeneity and pharmacokinetic variability. The primary model predicts the achievement of low disease activity (Systemic Lupus Erythematosus Disease Activity Index 2000 [SLEDAI-2 K] ≤ 4) at 12 months, while a secondary model predicts renal complete response during the same period.</p><p><strong>Methods: </strong>A total of 120 children with LN treated with MMF between 2001 and 2025 were included. The follow-up time from MMF initiation was 12 months. Comprehensive data encompassing clinical phenotypes, organ function, immunological profiles, and pharmacokinetic parameters were collected. We employed a data-driven approach to select key features from a comprehensive set of variables reflecting disease heterogeneity and drug exposure. Seven machine learning (ML) algorithms were evaluated, with their performance assessed using area under the curve (AUC), precision, recall, F1-score, and accuracy. For the secondary outcome of complete renal response, a separate model was constructed using seven similar ML algorithms. For the primary outcome, the incremental value of adding pharmacokinetic data was quantified using the Net Reclassification Improvement (NRI). The optimal models were further validated using calibration curves and decision curve analysis, and interpreted via SHAP analysis.</p><p><strong>Results: </strong>The final integrated model for predicting low disease activity, based on a logistic regression framework, demonstrated robust performance, achieving an AUC of 0.77 in the test set, with a supporting F1-score of 0.86. The incorporation of the area under the concentration-time curve for mycophenolic acid (MPA-AUC) provided significant incremental predictive value over a clinical-only model (ΔAUC = +0.05), which was further confirmed by an NRI of 0.1309. For predicting complete renal response, a random forest model achieved an AUC of 0.88. Key variables influencing treatment response across models included MPA exposure, corticosteroid dose, immunological, renal, and hepatic parameters. Both models were well-calibrated and provided significant net benefit in decision curve analysis.</p><p><strong>Conclusion: </strong>We present predictive models that incorporate a comprehensive profile of disease and drug-related heterogeneity to stratify pediatric patients with LN by their likelihood of responding to MMF. This integrated approach offers a strategy to optimize initial treatment selection by directly addressing the challenge of clinical variability.</p>","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":"46 4","pages":"e70127"},"PeriodicalIF":4.0,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147390492","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":"Comprehensive Review of Candidozyma (Candida) auris Management: Insights From the Society of Infectious Diseases Pharmacists.","authors":"Caleb E Rux, Melissa D Johnson, Victoria C Grant, Skip G Katipunan, Elias B Chahine, Dallas J Smith, Shawn R Lockhart, Emily Drwiega, Paul O Gubbins","doi":"10.1002/phar.70122","DOIUrl":"10.1002/phar.70122","url":null,"abstract":"<p><p>Candidozyma (Candida) auris has emerged over the past two decades as a formidable global health threat due to its multidrug resistance, persistence in healthcare environments, and rapid nosocomial spread. Recently reclassified into the genus Candidozyma based on phylogenomic analysis, C. auris poses major challenges for both clinical management and infection control. Its ability to tolerate heat, salinity, and disinfectants supports long-term survival on surfaces and medical devices, facilitating transmission. Biofilm formation further enhances virulence and resistance to antifungal therapy. Clinical presentations range from asymptomatic colonization to invasive infections, with mortality rates approaching 50%. Echinocandins remain an important first-line treatment option, but their fungistatic activity, limited tissue penetration, and emerging resistance contribute to suboptimal outcomes, highlighting the need for new agents and optimized dosing strategies. The role of triazoles and amphotericin B is significantly limited by resistance and associated toxicities, while newer agents such as ibrexafungerp, fosmanogepix, and rezafungin show promising in vitro activity but lack substantial supporting clinical data. Combination therapy may also offer potential benefit, though supporting evidence is sparse. Infection control methods including active surveillance, contact precautions, and environmental disinfection with sporicidal agents and avoidance of ineffective quaternary ammonium compounds are key to preventing the nosocomial spread of C. auris. Despite growing awareness, effective decolonization strategies are lacking, and recurrence and transmission continue to pose challenges. Ongoing efforts to refine antifungal therapy, improve rapid diagnostics, and strengthen infection control practices are essential to mitigating the spread of this pathogen and optimizing outcomes for patients.</p>","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":"46 4","pages":"e70122"},"PeriodicalIF":4.0,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147444654","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":"Outcomes of CDK4/6 Inhibitor-Based Therapy in Premenopausal HR+/HER2- Metastatic Breast Cancer: Real World Data From A Multicenter Study.","authors":"Hatice Odabaş, Omar Alomari, Seval Orman, Nadiye Sever, Esmanur Kaplan Tuzun, Aydin Acarbay, Eda Ercin, Miray Aydogan, Ali Kaan Guren, Muhammed Edib Mokresh, Alper Topal, Sedat Yildirim, Melike Ozcelik","doi":"10.1002/phar.70134","DOIUrl":"10.1002/phar.70134","url":null,"abstract":"<p><strong>Background: </strong>Breast cancer is the leading cause of cancer-related death in women worldwide. Hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer (MBC) represents the most common subtype. Cyclin-dependent kinase 4/6 (CDK4/6) inhibitors combined with endocrine therapy (ET) constitute standard first-line treatment, yet evidence comparing ribociclib and palbociclib in peri-/premenopausal patients remains limited. This study aimed to evaluate and compare the real-world efficacy and safety profiles of ribociclib versus palbociclib in combination with ET in this patient population.</p><p><strong>Methods: </strong>This multicenter, retrospective study included 267 peri-/premenopausal patients with HR+/HER2- MBC treated with palbociclib (n = 86) or ribociclib (n = 181) plus ET across five centers in Turkey between 2020 and 2024. Primary end points were progression-free survival (PFS) and overall survival (OS); secondary end points included objective response rate (ORR) and safety. Survival analyses used Kaplan-Meier and Cox regression methods using R software; adverse events were graded per Common Terminology Criteria for Adverse Events (CTCAE) v5.0.</p><p><strong>Results: </strong>The mean age was 42.96 years. Median follow-up was 19.6 months for palbociclib and 13.3 months for ribociclib (p = 0.003). No statistically significant differences were observed between palbociclib and ribociclib in median PFS (24.05 vs. 24.41 months; HR: 1.06; p = 0.94) or OS (not reached vs. 41.03 months; p = 0.86). The ORR was comparable at 64% for palbociclib and 69% for ribociclib (p = 0.459). Safety profiles were similar; any-grade adverse events occurred in 77.9% of palbociclib and 80.7% of ribociclib patients (p = 0.600). Neutropenia was the most common toxicity (72.1% palbociclib vs. 74.0% ribociclib). Grade 3-4 adverse events occurred in 29.1% of the palbociclib group and 40.3% of the ribociclib group (p = 0.074). Subgroup analyses showed consistent treatment effects across age, visceral involvement, and line of therapy.</p><p><strong>Conclusion: </strong>In real-world peri-/premenopausal patients with HR+/HER2- MBC, ribociclib and palbociclib combined with ET demonstrate comparable efficacy and tolerability. These findings support flexible treatment choice based on individual patient profiles and preferences.</p>","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":"46 4","pages":"e70134"},"PeriodicalIF":4.0,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147481336","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":"Probiotic Supplementation as an Adjuvant Therapy in Pediatric Drug-Resistant Epilepsy: A Double-Blind Placebo-Controlled Trial.","authors":"Amira R Rashdan, Sahar M El-Haggar, Ahmed Mustafa Kishk, Tarek M Mostafa","doi":"10.1002/phar.70117","DOIUrl":"10.1002/phar.70117","url":null,"abstract":"<p><strong>Background: </strong>Drug-resistant epilepsy (DRE) is increasingly linked to neuroinflammatory mechanisms driven by gut dysbiosis. These mechanisms compromise blood-brain barrier integrity, enhance seizure susceptibility, and modulate immune pathways. These insights underscore the therapeutic potential of microbiota-targeted interventions in epilepsy.</p><p><strong>Aim: </strong>The study aimed at assessing the effectiveness of probiotics as an adjunctive therapy to enhance drug sensitivity and clinical outcomes in children with DRE.</p><p><strong>Methods: </strong>This randomized, double-blind, placebo-controlled trial enrolled 60 pediatric patients with DRE who were assigned to either the control group (n = 30), which received a standard antiepileptic regimen (valproic acid, oxcarbazepine, and levetiracetam at the maximum tolerated doses) plus a daily placebo capsule, or the probiotic group (n = 30), which received the same antiepileptic regimen plus a daily probiotic (Lactobacillus acidophilus) supplement. The study duration was 6 months. Assessments of clinical and biochemical outcomes were conducted at baseline and 6 months after intervention. Primary end points included seizure frequency and change in quality of life as measured by the quality of life in childhood epilepsy (QOLCE-55) questionnaire. Secondary end points included the change in the serum levels of high-mobility group box 1 protein (HMGB1), interleukin-1β (IL-1β), homocysteine (Hcy), and NLR family pyrin domain-containing 3 (NLRP3).</p><p><strong>Results: </strong>After 6 months and relative to the control group, the probiotic (Lactobacillus acidophilus) group experienced a significant decline in seizure frequency (p = 0.04) and a significant improvement in the QOLCE-55 total score (p < 0.0001). Additionally, the probiotic group exhibited significant decreases in the serum levels of HMGB1 (p = 0.0005), NLRP3 (p = 0.002), Hcy (p = 0.001), and IL-1β (p = 0.05) compared with the control group.</p><p><strong>Conclusion: </strong>Lactobacillus acidophilus supplementation appears to enhance the effectiveness of conventional antiepileptic drugs, reduce systemic inflammation, improve quality of life, and reduce seizure frequency in children with DRE. However, further validation is necessary.</p><p><strong>Clinicaltrials: </strong></p><p><strong>Gov registration number: </strong>NCT05539287.</p>","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":"46 3","pages":"e70117"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146202227","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":"Cohort Study to Determine the Impact of CYP3A5 Genotype on Tacrolimus Dosing Requirements and Trough Concentrations in Heart Transplant Recipients.","authors":"Yanting Wu, Michael T Eadon, Laurie Schenkelberg, Roopa A Rao, Todd C Skaar, Emma M Tillman, Tyler Shugg","doi":"10.1002/phar.70116","DOIUrl":"10.1002/phar.70116","url":null,"abstract":"<p><strong>Background: </strong>Tacrolimus is primarily metabolized by Cytochrome P450 (CYP)3A4/5. The Clinical Pharmacogenetics Implementation Consortium recommends increasing the initial dose 1.5- to 2-fold in CYP3A5 expressers to enhance transplant outcomes. Our objective was to investigate the impact of CYP3A5 expresser status on tacrolimus dosing requirements and attainment of target trough concentrations in heart transplant recipients.</p><p><strong>Methods: </strong>We performed a retrospective cohort analysis of tacrolimus dose, concentration, demographics, CYP3A4/5 genotype, concomitant medications, and biochemical data in heart transplant recipients from December 2020 to August 2023. The primary outcome was the time to first therapeutic trough concentration, compared by CYP3A5 expression status. Secondary outcomes included the tacrolimus dose at target trough and dose-adjusted tacrolimus trough concentration (C₀/D). Stepwise multiple regression was performed to account for potential covariates. Moreover, clinical outcomes were assessed at 1-year post-transplantation and compared based on CYP3A5 expression status.</p><p><strong>Results: </strong>Among 33 patients, CYP3A5 expressers (27.3%) required longer to achieve therapeutic trough concentrations (median [Q1, Q3]: expressers: 14 [9.5, 16] days vs. nonexpressers 7.5 [6.0, 11] days; p = 0.0073) and required nearly double the tacrolimus dose to reach target concentrations (10 [5.5, 13] mg/day for expressers vs. 5 [3.3, 5.9] mg/day for nonexpressers; p = 0.0019). Conversely, the C₀/D was nearly 2-fold higher in nonexpressers 2.0 [1.6, 3.4] ng/(mL*mg) than expressers (1.1 [0.83, 1.7] ng/(mL*mg); p = 0.0015). Stepwise regression identified route of administration (sublingual vs. oral) at therapeutic trough and initial dose as covariates for all outcomes. All clinical outcomes showed no significant differences based on CYP3A5 expression status, with the exception that poor metabolizers demonstrated higher serum creatinine elevation at 1-week post-transplantation (p = 0.047).</p><p><strong>Conclusion: </strong>Our findings highlight the impact of CYP3A5 expresser status on the time needed and dosing requirements to attain tacrolimus therapeutic concentrations in heart transplant recipients, suggesting CYP3A5-guided dosing strategies may improve rapid attainment of therapeutic tacrolimus concentrations.</p>","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":"46 3","pages":"e70116"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12884575/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146143160","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":"Maternal and Pediatric Precision in Therapeutic Knowledge Portal (MPRINT-KP): Landscape Analysis of Pharmacology Research in Maternal and Pediatric Patient Populations.","authors":"Xiaofu Liu, Aditi Shendre, Lei Wang, Aislinn O'Kane, Hanson Ma, Chien-Wei Chiang, Syed S Zaidi, Omar A Aboshady, Gerald So, Emma M Tillman, Lindsey M Kirkpatrick, Maged Costantine, Shaun Grannis, Colin M Rogerson, Christopher Bartlett, Saurabh Rahurkar, Lijun Cheng, Jiayi Ouyang, Ping Wei, Zhimo Zhu, Shangjia Li, Yirui Huang, Lingling Wang, Weidan Cao, Haoran Jiang, Jianing Liu, Samuel-Richard Oteng, Andrew Goodwin, Jiezel Ann Faith Deypalubos, Shijun Zhang, Robert Bies, Sara K Quinney, Lang Li","doi":"10.1002/phar.70096","DOIUrl":"10.1002/phar.70096","url":null,"abstract":"<p><p>Pregnant women and children have been underrepresented in clinical studies due to ethical concerns and perceived vulnerabilities. This resulted in a significant gap in knowledge regarding the safety and efficacy of medications for these populations. Maternal and Pediatric PRecision In Therapeutics Knowledge Portal (MPRINT-KP) is designed to provide a comprehensive view of pharmacokinetic, pharmaco-epidemiology, and clinical trial research evidence in maternal and pediatric patient populations. MPRINT-KP Silver was generated upon BioBERT models, due to their supreme performance in classifying pharmacokinetic, pharmaco-epidemiology, and clinical trial papers from PubMed, with an F1-score > 0.91. As of April 1, 2025, MPRINT-KP Silver contains 758,560 clinical pharmacology research papers in maternal and pediatric populations. The landscape analysis revealed a large number of drugs with pharmacology knowledge gaps, which is calculated as the relative frequency of drugs with no or weak (i.e., less than five publications) evidence in each of pharmacokinetic, pharmaco-epidemiology, or clinical trial study type. The highest pharmacotherapy knowledge gaps are in postpartum women, pregnant women, and pediatric patients between 0-12 and 12-18 years of age (51.37%, 32.47%, 25.82%, 32.11%, respectively). Pharmacovigilance analyses were conducted on highly prescribed drugs with limited pharmacology evidence in pediatric patients 0-2 year old and pregnant women using United States Food and Drug Administration Adverse Event Reporting System (FAERS) and MarketScan claims data. A number of new drug-associated adverse drug events (ADEs) were discovered. In pediatric patients, there were crisaborole-associated hemorrhage and moxifloxacin-associated cardiac toxicities. In pregnant women, the analysis revealed terconazole-associated abnormalities of heartbeat; benzonatate-associated depressive and anxiety disorders; buspirone-associated abnormalities of heartbeat; cyclobenzaprine-associated tubulo-interstitial nephritis; and pantoprazole-associated hearing loss and voice and resonance disorders. For the first time, a large-scale landscape analysis of pharmacotherapy knowledge gap in both maternal and pediatric patient populations was conducted and identified new drug associated ADE evidence using real world data.</p>","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":"46 3","pages":"e70096"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12929201/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147276081","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":"Pharmacokinetic and Pharmacodynamic Principles and Concepts Remain Relevant in the Era of Artificial Intelligence and Machine Learning.","authors":"Keith A Rodvold","doi":"10.1002/phar.70097","DOIUrl":"10.1002/phar.70097","url":null,"abstract":"","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":" ","pages":"e70097"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145952771","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":"Harnessing the Power of Artificial Intelligence to Enhance Drug Therapy Research.","authors":"William L Baker, Alexandre Chan","doi":"10.1002/phar.70105","DOIUrl":"10.1002/phar.70105","url":null,"abstract":"","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":" ","pages":"e70105"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145966520","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":"Current and Emerging Pharmacologic Therapies for Diabetic Retinopathy.","authors":"Jennifer N Clements, Julia Mesawich, Cassandra Twisdale","doi":"10.1002/phar.70119","DOIUrl":"10.1002/phar.70119","url":null,"abstract":"<p><p>Diabetic retinopathy, a prevalent microvascular complication of diabetes mellitus, is a leading cause of blindness among adults in developed countries. Risk factors include prolonged duration of diabetes, elevated glycosylated hemoglobin levels, hypertension, and dyslipidemia, with rapid glucose reduction also increasing risk for developing diabetic retinopathy. The American Academy of Ophthalmology classifies diabetic retinopathy into non-proliferative diabetic retinopathy and proliferative diabetic retinopathy, distinguished by the absence or presence of neovascularization, respectively. Complications such as diabetic macular edema, retinal detachment, and irreversible vision loss further underscore the need for effective therapies. Current treatments include laser photocoagulation, vitrectomy, and anti-vascular endothelial growth factor agents. This narrative review evaluates established pharmacotherapy and explores emerging therapies targeting novel pathways such as inflammation, oxidative stress, and vascular stability. Investigational agents, including integrin inhibitors, gene therapies, and small-molecule antagonists, alongside innovative delivery systems such as topical formulations and sustained-release implants, offer the potential to enhance treatment efficacy, patient adherence, and overall outcomes in diabetic retinopathy. By examining these advancements, including innovative delivery systems, this review aims to inform practitioners about the evolving landscape of pharmacotherapy for diabetic retinopathy.</p>","PeriodicalId":20013,"journal":{"name":"Pharmacotherapy","volume":"46 3","pages":"e70119"},"PeriodicalIF":4.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12907467/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146202251","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}