DRUGS - REAL WORLD OUTCOME
Drugs real world outcomes: Latest results from PubMed
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Emerging targeted therapies for primary immune thrombocytopenia: novel agents, combination strategies, and future directions
CONCLUSION: This article analyses recent therapeutic trials with Bruton's tyrosine kinase inhibitors, splenic tyrosine kinase inhibitors, and neonatal Fc receptor antagonists, demonstrating an improvement in platelet counts and patients' quality of life. Furthermore, combination therapy regimens show great promise in terms of synergistic effects, while cellular therapies, such as chimeric antigen receptor T-cell immunotherapy and mesenchymal stem cell therapy, appear to offer new treatment... -
Implementation factors shaping British Columbia's drug decriminalization pilot: A systematic review with narrative synthesis
CONCLUSION: BC's decriminalization pilot suggests the effects of legal reform are shaped by implementation context. Early outcomes may reflect design features, institutional readiness, and system capacity rather than legal change alone; longer-term impacts remain uncertain. Future reforms should align legal change with coordinated implementation, operational guidance, public communication, and adequate service infrastructure. -
Disease Stabilization with MEK Inhibitors in NF1-Associated Plexiform Neurofibromas: A Systematic Review and Meta-Analysis with Subgroup Analyses by Age and Study Design
CONCLUSIONS: The use of MEK inhibitors is associated with high rates of disease control and minimal tumor progression in patients with NF1-related PN, with consistent effects observed across clinical trial and real-world environments. Although tumor reduction occurs in some patients, the predominant therapeutic benefit appears to be sustained disease stabilization, with response variability noted among different age groups and study designs. -
Efficacy and safety of antibody-drug conjugates for HR+/HER2-low advanced breast cancer: a systematic review with Bayesian network meta-analysis and real-world study
CONCLUSIONS: ADCs significantly improved the clinical outcomes of patients with HR+/HER2 low expression advanced breast cancer. Compared with other ADC drugs, T-DXd showed relatively better treatment characteristics, better PFS benefit, and relatively low incidence of serious AEs (SAEs). Combined with RCTs and real-world data, T-DXd has potential advantages in this population. -
The Clinical and Economic Impact of Biologic Agents in Asthma Management: a Systematic Review
CONCLUSIONS: Biologic therapies achieve substantial clinical benefits and favorable economic value through healthcare cost offsets. Precision medicine approaches and early response assessment optimize patient selection and clinical outcomes in severe asthma management. -
Efficacy and safety of Serenoa repens in benign prostatic disorders: a systematic review of recent clinical evidence
This systematic review assessed the potential effectiveness and safety of Serenoa repens (saw palmetto) for benign prostatic disorders, mainly benign prostatic hyperplasia and associated lower urinary tract symptoms. Following PRISMA guidance (PROSPERO: CRD420251032255), we searched PubMed, Cochrane and ScienceDirect for human studies from January 2020 to May 2025. Sixteen studies (>3000 participants) met criteria, including randomized trials, observational cohorts, a post hoc analysis, an... -
Adverse events in bedaquiline- and pretomanid-based regimens for drug-resistant tuberculosis from trial, implementation and pharmacovigilance studies
The availability of safety data, particularly concerning adverse events (AEs) associated with the new shorter regimen for drug-resistant tuberculosis (TB) containing a bedaquiline-pretomanid-based regimen, is still limited. This systematic review aims to provide a comprehensive and updated analysis of AEs related to this new regimen by combining safety data from clinical trials, implementation and pharmacovigilance studies. We conducted a search using PubMed, Medline and Web of Science to... -
Comparative Efficacy, Safety, and Cost-Utility of DPP-4 Inhibitors and Metformin Combination Therapy in Type 2 Diabetes: A Systematic Review of Real-World Clinical and Economic Outcomes
INTRODUCTION: The management of type 2 diabetes with metformin as the first-line therapy has long been established. However, combination therapy of metformin and other oral antidiabetics became necessary to achieve optimal glycemic targets. Recently, the rising cost of these combinations poses a challenge for the healthcare system and patients, particularly in low- and middle-income settings, highlighting the need to balance clinical benefits with economic considerations to ensure access to... -
Evaluating oritavancin for Gram-positive infections: a systematic review of on-label and off-label use
CONCLUSIONS: Oritavancin is effective for ABSSSI and may be a promising treatment alternative for selected Gram-positive off-label indications requiring prolonged therapy. Standardized dosing strategies and prospective trials are needed to define optimal regimens for off-label indications. -
From Methotrexate Resistance to Biologic and Targeted Pharmacotherapy: A Decade of Phase 4 Clinical Trials Evidence in Rheumatoid Arthritis
CONCLUSION: Phase 4 evidence highlights the continued role of TNF inhibitors alongside newer biologic and targeted synthetic therapies in MTX-resistant RA. Trial heterogeneity reflects real world clinical complexity, underscoring the importance of treatment optimization, safety monitoring, and emerging precision strategies in contemporary RA management. -
Global Long-Term Cost Effectiveness of Newer Antidiabetic Drugs for Type 2 Diabetes Mellitus: A Systematic Review
CONCLUSIONS: These findings should be interpreted cautiously given the substantial heterogeneity across studies and methodological limitations inherent to long-term economic modelling. Overall, NADs generally provide favourable long-term cost effectiveness, owing to their cardiovascular and renal benefits; however, recently introduced agents and early-line use tend to be cost effective only at higher willingness-to-pay thresholds or after significant price reductions. Future evaluations should... -
Hand-dominant joint involvement pattern associates with favourable, and polyarthritis with unfavourable, treatment response to both csDMARDs and bDMARDs in early rheumatoid arthritis: a combined analysis of NORD-STAR and BeSt trials
CONCLUSIONS: In early RA, csDMARD and bDMARD treatments resulted in the greatest improvement in disease activity in JIP-Hand and the least improvement in JIP-Poly. -
Diagnosis and treatment of human epidermal growth factor receptor 2-positive metastatic colorectal cancer: a systematic literature review
Human epidermal growth factor receptor 2 (HER2)-targeted therapies have been investigated for therapeutic benefit in RAS/BRAF wild-type/HER2+ metastatic colorectal cancer (mCRC). Unlike HER2+ breast and gastric cancer, there are no regulatory criteria for determining HER2 overexpression in patients with mCRC. This systematic literature review describes unmet needs for patients with HER2+ mCRC in relation to testing and treatment, highlights the importance of early HER2 testing at mCRC diagnosis,... -
Multi-Targeted TKIs in Patients with Advanced Ewing Sarcoma: A Systematic Review and Single-Arm Meta-Analysis
Background/Objectives: Standard treatment of multiply relapsed Ewing sarcoma remains to be established. Recent studies evaluating tyrosine kinase inhibitors (TKIs) with anti-angiogenic properties have shown encouraging results. Therefore, we conducted a systematic review and meta-analysis to explore the efficacy and safety of TKIs in patients with Ewing sarcoma. Methods: We comprehensively searched PubMed, Embase, and Cochrane databases for clinical trials (CTs) and cohort studies assessing TKIs... -
Unmanned aerial vehicles in prehospital emergency care: current evidence, technological advances, and future perspectives
CONCLUSION: UAVs demonstrate significant potential to enhance the speed, coverage, and coordination of prehospital emergency care. Future research should focus on integrating UAVs within established emergency medical service networks, developing unified policy and airspace regulations, and validating cost-effectiveness and clinical impact through large-scale, real-world studies. -
Treatments for renal cell carcinoma: NICE Pilot Treatment Pathways Appraisal
CONCLUSIONS: This pilot demonstrated the feasibility of producing a reference model, which is open source and available to relevant stakeholders without restriction. This will improve consistency in the National Institute for Health and Care Excellence's decision-making and allow for the evaluation of optimum treatment sequences for advanced renal cell carcinoma. -
Treatment strategies for motor fluctuations in Parkinson's disease: a systematic review of efficacy, functionality, and drug accessibility with a focus on Latin America
CONCLUSION: While multiple pharmacological and device-based interventions effectively reduce OFF-time in Parkinson's disease, their real-world impact is constrained by uneven global access and affordability. The Latin American region exemplifies these disparities, with limited regulatory availability, heterogeneous pricing, and insufficient inclusion of novel agents in national formularies. Integrating efficacy evidence with accessibility analyses highlights the need for coordinated regional... -
Restoring the Sense of Smell: A Systematic Review and Meta-Analysis of Biologic Therapies for Chronic Rhinosinusitis with Nasal Polyps
PURPOSE OF REVIEW: Smell loss is among the most debilitating symptoms of chronic rhinosinusitis with nasal polyps (CRSwNP). We aimed to synthesize current evidence on the smell-restoring efficacy of biologics, focusing on outcomes measured by psychophysical tests. -
Real-world effectiveness and safety of galcanezumab for the treatment of migraine: A systematic review and meta-analysis
CONCLUSION: Galcanezumab appears to be associated with clinically meaningful improvements in migraine and favorable safety outcomes, although the evidence certainty is limited by heterogeneity. -
Machine learning for predicting treatment response to biologic and targeted synthetic disease-modifying antirheumatic drugs in rheumatoid arthritis: a scoping review
CONCLUSION: ML methods are increasingly used to predict RA treatment response, but vary widely in methodology and performance. Standardization, external validation, and transparent reporting are critical for advancing clinical application. -
Comparative outcomes of aflibercept biosimilars and reference aflibercept in nAMD: a systematic review and meta-analysis
CONCLUSION: This systematic review and meta-analysis found no statistically significant differences in functional, anatomical or safety outcomes between aflibercept biosimilars and the reference product for nAMD, based on moderate to high certainty evidence. Functional and anatomical outcomes appeared stable across multiple timepoints. Further long-term pharmacovigilance studies and real-world data beyond 56 weeks are warranted. -
The association of direct-acting antiviral therapy with health care utilization and costs in patients with chronic hepatitis C virus infection: A systematic review of real-world studies in the United States
CONCLUSIONS: This systematic review reveals significant gaps in the real-world economic evaluation of DAA therapy. In particular, longitudinal data, geographic granularity, and state-specific analyses are lacking. Future research should address these limitations and further explore the long-term impacts of DAA therapy and variations in access and costs across different populations and insurance programs. -
Drugs for transthyretin amyloidosis under the microscope: Survival, safety, and a meta-analysis with certainty of evidence assessment
Transthyretin amyloidosis (ATTR) is an infiltrative disease caused by the deposition of misfolded transthyretin (TTR) fibrils in organs and tissues, with incidence and prevalence rapidly increasing worldwide. Current therapeutic strategies fall into two main classes: TTR stabilizers and TTR gene silencers. To date, no comprehensive reviews cover all the available pharmacological treatments for ATTR, both approved and off-label. In addition, previous meta-analyses have often excluded real-world... -
Strategies to Improve PrEP Adherence in People Who Use Drugs: A Systematic Review
People who use drugs (PWUD) face a disproportionately high risk of HIV, accounting for a considerable share of cases in the United States. Pre-exposure prophylaxis (PrEP) has been shown to be effective in preventing HIV transmission through both sexual contact and injection drug use. However, adherence is essential for real-world success. Following PRISMA guidelines, we conducted the first systematic review to examine PrEP interventions among PWUD and identify strategies to enhance adherence.... -
Current Evidence on Celecoxib Safety in the Management of Chronic Musculoskeletal Conditions: An Umbrella Review
CONCLUSIONS: Celecoxib appears to offer better gastrointestinal safety than non-selective NSAIDs. Although data on cardiovascular, renal, and mortality outcomes suggest possible advantages, the evidence remains limited and of low certainty. Moreover, some real-world evidence raises concerns in specific high-risk populations. Future research should integrate data from both randomized trials and observational studies to better inform long-term safety assessments and guide individualized treatment... -
Long-Term Safety of Anti-Interleukin-1 Medications in Children with Rheumatic Diseases: a Systematic Review
CONCLUSIONS: Our results are consistent with the known safety profile of anti-IL-1 medications and show that they are generally safe for use in the context of childhood rheumatic and autoinflammatory diseases. This review of clinical trial and real-world data will help inform clinical decision-making and family counselling when initiating anti-IL-1 medications in children. -
Comparative effectiveness and safety of biosimilars versus reference biologics in rheumatoid arthritis during treatment initiation: a systematic review of real-world evidence
CONCLUSION: In real-world settings, biosimilars generally demonstrate comparable effectiveness to originators. Future investigations are warranted to examine the comparative safety profiles of biosimilars and originators.
