Journalz

Phase 3 RCT: 3-year structured exercise post-adjuvant chemotherapy raised 5-year disease-free survival to 80.3% vs 73.9% in controls at 8-year median follow-up. First RCT to show an exercise-survival link in colon cancer.

Addition of PD-L1 blockade to perioperative FLOT chemotherapy produced significant event-free and overall survival gains in resectable gastric/GEJ cancer, establishing a new perioperative standard.

Dual checkpoint blockade doubled progression-free survival vs nivolumab alone (median 38.4 vs 13.1 months) in MSI-H/dMMR mCRC, cementing combined IO as first-line standard for this biomarker-selected subgroup.

CheckMate 816 update: neoadjuvant nivolumab+chemo significantly improved OS vs chemo alone (NR vs 45.5 months; HR 0.71) in resectable NSCLC, providing definitive OS support for neoadjuvant IO in early-stage lung cancer.

First RCT of CLDN18.2-targeted CAR-T in solid tumors: significant OS benefit (14.4 vs 9.6 months) over physician's choice in CLDN18.2+ gastric/GEJ cancer, extending CAR-T success to solid-tumor oncology.

Individual patient-data meta-analysis of 4 RCTs (n=3,373): IV iron reduced HF hospitalisations or CV death by 21% (HR 0.79) across HFrEF and HFmrEF, resolving prior conflicting trial results and supporting guideline expansion.

ALONE-AF RCT (n=840): stopping oral anticoagulation ≥12 months after AF ablation with no recurrence was non-inferior to continuing DOAC for stroke/TIA at 2 years (0.5% vs 0.9%), supporting a shared-decision approach to anticoagulation cessation post-ablation.

RCT in HFpEF: 12-month combined aerobic+resistance training improved peak VO2 by 3.1 mL/kg/min, reduced E/e′, and improved KCCQ vs usual care—the first trial to show exercise remodels diastolic function in HFpEF.

RCT (n=407): liberal fluid intake (no restriction) was non-inferior to standard 1.5 L/day restriction for HF hospitalizations or death (HR 0.97), definitively challenging decades-old fluid-restriction guidelines and improving patient quality of life.

10-year follow-up: intensive risk-factor management (weight, BP, sleep apnea, alcohol, exercise) alongside AF ablation maintained sinus rhythm in 86% vs 38% for ablation alone, confirming lifestyle modification as essential co-therapy after ablation.

CLARITY AD phase 3 RCT (n=1,795): lecanemab slowed CDR-SB decline by 27% vs placebo at 18 months with significant amyloid clearance. First anti-amyloid antibody with unequivocal phase 3 efficacy, earning FDA traditional approval and shaping global Alzheimer's treatment.

CATIS-3 RCT (n=680): IV tenecteplase before thrombectomy increased functional independence at 90 days (52.9% vs 44.1%; RR 1.20) in LVO stroke within 4.5 h of onset, supporting tenecteplase as the preferred bridging thrombolytic before EVT.

Head-to-head RCT (n=751): tirzepatide 15 mg produced greater weight loss than semaglutide 2.4 mg (20.2% vs 13.7% at 72 weeks) with superior waist circumference reduction, establishing tirzepatide as the more effective GLP-1–class agent for obesity.

SOUL CVOT (n=9,650): oral semaglutide reduced 3-point MACE by 14% vs placebo (HR 0.86) in T2D patients with established ASCVD or CKD—extending CV outcome benefits of GLP-1 agonists to oral administration for the first time.

NOTUS phase 3 RCT (n=935): dupilumab reduced moderate/severe COPD exacerbations by 34% vs placebo in eosinophilic COPD (eos ≥300/µL), confirming BOREAS and establishing the first approved biologic for COPD.

Double-blind RCT: a single dose of benralizumab (anti-IL-5Rα) during eosinophilic exacerbations of asthma or COPD reduced treatment failure at 90 days compared with placebo add-on, supporting eosinophil-guided biologic use in acute exacerbations.

RCT (n=1,060): population-based spirometric screening followed by treatment in screen-positive individuals reduced symptom burden, exacerbations, and health-care utilization over 2 years, providing strong evidence that early COPD/asthma detection and treatment is beneficial.

MAESTRO-NASH (n=966): resmetirom 100 mg resolved MASH in 29.9% vs 9.7% placebo and improved fibrosis ≥1 stage in 25.9% vs 14.2%, earning FDA approval as the first drug specifically indicated for MASH — a landmark in hepatology.

CONFIDENCE RCT (n=807): finerenone + empagliflozin combination reduced UACR by 38% vs either drug alone at 12 months in CKD+T2D, with no unexpected adverse events — the first head-to-head evidence supporting dual MRA+SGLT2i combination therapy for CKD.

15 patients with refractory SLE, systemic sclerosis, or idiopathic inflammatory myositis treated with autologous anti-CD19 CAR-T; all achieved drug-free remission with a median follow-up of 15 months. Transformative proof-of-concept for CAR-T in autoimmune disease.

CLIMB SCD-121 trial: CRISPR/Cas9 gene-edited HSC therapy (Casgevy) eliminated vaso-occlusive crises in 28/29 patients (97.2%) with 12 months' follow-up, achieving FDA approval as the first CRISPR-based cure for sickle cell disease.

ELEVATE-TN long-term update: acalabrutinib + obinutuzumab maintained a significant PFS advantage over FCR or chlorambucil+obinutuzumab at 6-year follow-up (70% vs 38% PFS), confirming fixed-duration BTK inhibitor combinations as frontline CLL standard.

COMP360 phase 2b RCT (n=233): psilocybin 25 mg produced significantly greater MADRS score reduction than 1 mg at 3 weeks (−12.0 vs −7.9 points), with 29% remission vs 8%. The largest psilocybin RCT to date, informing ongoing phase 3 programs.

Case report of a neonate with CPS1 deficiency treated with a custom base-editor designed, manufactured, and administered in under 7 months. Normalized plasma ammonia and achieved normal developmental milestones — proof-of-concept for individualized gene editing in pediatric metabolic disease.

French nationwide cohort (n=383,398 infants): nirsevimab effectiveness against RSV-related hospitalization was 79.5% (95% CI 72.8–84.6%) during the 2023–2024 RSV season, confirming large-scale real-world efficacy of the prophylactic monoclonal antibody.

APPAC 10-year update: 63% of patients randomized to antibiotics for uncomplicated appendicitis remained recurrence-free without surgery at 10 years. No appendiceal cancers were missed in the antibiotic arm, providing the definitive long-term safety evidence for non-operative appendicitis management.

RCT in high-risk pregnancies: low-dose aspirin initiated at 11–14 weeks significantly reduced early preterm pre-eclampsia (<34 weeks) incidence and delayed the gestational age at preterm delivery, providing data supporting earlier and higher-risk aspirin prophylaxis in obstetric practice.

Multicenter RCT targeting capillary refill time normalization vs lactate-guided resuscitation in early septic shock. Provides updated evidence on perfusion-based endpoints vs lactate targets for hemodynamic resuscitation, informing bedside sepsis management in the ED and ICU.