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Oct 15Thu
  1. N Engl J Med76

    Long-Acting HIV Therapy in African Adults with Adherence Challenges.

    Long-acting injectable cabotegravir plus rilpivirine every 8 weeks maintained viral suppression in African adults with HIV and adherence challenges, matching daily oral dolutegravir-based therapy. At week 48, 91.0% of the long-acting group (244 of 268) had a viral load below 50 copies per milliliter versus 89.2% of the oral group (240 of 269), meeting noninferiority. Confirmed virologic failure occurred in 5 participants (1.9%) on long-acting therapy and none on oral therapy.

    Why it matters: It tests long-acting injectable HIV therapy in the population most affected by adherence failure: African adults with suboptimal adherence.

  2. N Engl J Med77

    Phase 2 Trial of Icotrokinra for Patients with Ulcerative Colitis.

    Once-daily oral icotrokinra, a peptide that blocks the interleukin-23 receptor, beat placebo for clinical response at week 12 in adults with moderately to severely active ulcerative colitis: 63% at 400 mg, 58% at 200 mg and 55% at 100 mg versus 27% on placebo (P<0.001 for all). The 400-mg group also showed more clinical remission (30% vs. 11%), symptomatic remission (46% vs. 19%) and endoscopic improvement (37% vs. 14%). It offers an oral alternative to injected interleukin-23-targeted antibodies.

    Why it matters: An oral interleukin-23 receptor blocker matched injected antibodies' mechanism with positive phase 2 results.

  4. N Engl J Med71

    Intermittent or Continuous Renal-Replacement Therapy in Acute Kidney Injury.

    Starting renal-replacement therapy with intermittent hemodialysis rather than continuous renal-replacement therapy was not noninferior in critically ill adults with acute kidney injury. The trial stopped early after 554 patients were randomized because of higher mortality with intermittent hemodialysis: day-90 death occurred in 63.1% versus 53.5%, and major adverse kidney events in 78.1% versus 71.6%. Clinicians choosing an initial modality now have mortality evidence favoring continuous therapy.

    Why it matters: It gives clinicians comparative mortality evidence for choosing the initial renal-replacement modality in acute kidney injury.

  5. JAMA69

    Effect of Vasopressin Initiation Threshold on Septic Shock Mortality: The VASSPR Cluster Crossover Randomized Strategy Trial.

    A hospital-level strategy of starting fixed-dose vasopressin at 0.1 µg/kg/min norepinephrine-equivalent did not reduce 28-day mortality compared with starting at 0.4 µg/kg/min in septic shock (31.8% vs 33.0%; adjusted OR 0.98, 95% CI 0.87-1.06). The trial randomized 13 hospitals in a cluster-crossover design and included 3959 patients; kidney replacement therapy-free days were also unchanged. Clinicians can defer second-line vasopressin without a mortality penalty.

    Why it matters: Large pragmatic trial showing the timing of adding vasopressin in septic shock does not change mortality.

  6. JAMA67

    Isoflurane-Based Inhaled Sedation in Mechanically Ventilated Adults With Acute Hypoxemic Respiratory Failure: The SAVE-ICU Randomized Clinical Trial.

    An isoflurane-based inhaled sedation strategy did not reduce hospital mortality in mechanically ventilated adults with acute hypoxemic respiratory failure compared with usual intravenous sedation. In the SAVE-ICU trial of 780 adults across 14 Canadian ICUs, 42.5% died in the inhaled sedation group versus 43.8% with intravenous sedation (risk ratio, 0.98; P = .85). Isoflurane patients received more adjunctive therapies for hypoxemia and had fewer delirium- and coma-free days.

    Why it matters: Large randomized trial shows no mortality benefit and some signals of harm for isoflurane sedation in ventilated AHRF patients.

  7. doi.org48

    Open-label extension study of leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety

    Long-term leniolisib was generally well tolerated in 37 patients with activated phosphoinositide 3-kinase delta syndrome treated for a median of 4.3 years. Annual infection rates (P = 0.0005), respiratory infection rates (P = 0.0017) and immunoglobulin replacement therapy use (P = 0.0095) all declined per year of treatment, with clinically meaningful gains in 36-Item Short Form Survey general health scores. This extends the efficacy and safety evidence for leniolisib in APDS well beyond the pivotal trials.

Oct 14Wed
  1. nature.com62

    Talazoparib plus enzalutamide in high-volume metastatic hormone-naive prostate cancer: a randomized phase 2 trial

    Adding the PARP inhibitor talazoparib to enzalutamide improved prostate-specific antigen response in patients with high-volume metastatic hormone-sensitive prostate cancer in the randomized phase 2 ZZ-FIRST trial. Spatial transcriptomic analysis of paired biopsies showed that androgen receptor inhibition suppresses homologous recombination repair-related programs, and this suppression correlated with proliferation arrest rather than functional HRR deficiency.

    Why it matters: Links AR inhibition to HRR program suppression, giving a mechanistic basis for combining PARP inhibition with androgen receptor inhibitors.

  2. nature.com50

    Personalized neoantigen-targeting cancer vaccines in ovarian cancer: a phase 1 trial

    Personalized neoantigen vaccines combined with PD-1 blockade were feasible in patients with high-grade serous ovarian cancer in a phase 1 trial. Many patients mounted tumor-specific immune responses. Cell-free DNA analysis identified putative resistance mechanisms related to antigen presentation. The trial establishes feasibility of this approach in ovarian cancer and points to antigen presentation as a resistance pathway.

Oct 13Tue
  1. Nat Med65

    Intravenous hyaluronidase-expressing oncolytic adenovirus with chemotherapy in metastatic pancreatic cancer: a randomized phase 2b trial.

    Adding intravenous VCN-01, a hyaluronidase-expressing oncolytic adenovirus, to gemcitabine and nab-paclitaxel improved outcomes as first-line therapy in metastatic pancreatic ductal adenocarcinoma. In the full analysis set, median overall survival was 10.8 versus 8.6 months (HR 0.57) and progression-free survival was 7.0 versus 4.6 months (HR 0.55, P = 0.011); response duration was 11.2 versus 5.4 months. The phase 3 evaluation is warranted.

    Why it matters: First randomized evidence that an intravenous oncolytic virus plus chemotherapy extends survival in metastatic pancreatic cancer.

  2. medrxiv.org41

    FORESEE: A prospective feasibility trial of functional precision oncology for metastatic breast cancer patients

    Organoid-based drug testing plus genomic characterization was feasible but limited by PDO establishment in HER2-negative metastatic breast cancer. Of 15 patients enrolled, 10 biopsies had adequate tumor content and PDOs were established in 4, an overall success rate of 40%, missing the primary endpoint of at least 46%. Results returned within 12 weeks informed treatment selection in all evaluable cases, and physicians used the information in 100% of successful cases.

    Why it matters: Quantifies the real-world success rate of functional precision oncology in metastatic breast cancer, showing PDO establishment as the bottleneck.

Oct 11Sun
  1. N Engl J Med82

    Vasopressors or Fluids in Early Septic Shock.

    Early vasopressor therapy with restricted fluids did not improve outcomes over higher fluid volumes with later vasopressors in emergency department patients with septic shock. In 963 intention-to-treat patients, days alive and out of the hospital at day 90 were a median of 76 in both groups (difference, 0.0 days; 95% CI, -2.7 to 2.7; P = 1.00). Pulmonary edema was less frequent with the vasopressor strategy (0.6% vs. 5.0%; P<0.001).

    Why it matters: It resolves equipoise over early vasopressors versus larger fluid volumes in early septic shock resuscitation.

Oct 10Sat
  1. Nat Med62

    Extended follow-up of in vivo BCMA CAR-T therapy in relapsed/refractory multiple myeloma.

    Extended follow-up of in vivo BCMA CAR-T (ESO-T01), given without leukapheresis or lymphodepletion to four patients with relapsed/refractory multiple myeloma, shows no new important adverse events beyond the initial report at up to 15 months. One patient maintained stringent complete response for 15 months; median progression-free survival was 4.0 months (range 3.0-15.0). Three patients relapsed or progressed, all with extramedullary involvement, and died during follow-up, indicating limited CAR-T persistence requiring platform optimization.

Oct 9Fri
  1. Lancet75

    Oral deucrictibant for on-demand treatment of hereditary angioedema attacks: a phase 3, multicentre, randomised, double-blind, placebo-controlled crossover trial.

    A 20 mg oral deucrictibant capsule gave faster onset of symptom relief than placebo in hereditary angioedema attacks, with median relief times of under one and a half hours for deucrictibant and beyond twelve hours for placebo, a statistically significant difference. In the phase 3 RAPIDe-3 crossover trial, 113 participants treated 201 attacks, and no severe, serious or treatment-related discontinuation events occurred. Deucrictibant could become the first orally administered bradykinin B2 receptor antagonist for treating attacks of bradykinin-mediated angioedema.

    Why it matters: Phase 3 data show an oral capsule can relieve hereditary angioedema attacks far faster than placebo.

  2. JAMA74

    Nicotinamide Riboside for Patients With Early-Stage Parkinson Disease: The NOPARK Randomized Clinical Trial.

    Nicotinamide riboside, 500 mg twice daily for 52 weeks, did not improve clinical outcomes in early Parkinson disease and was worse than placebo. In the phase 3 NOPARK trial of 393 patients, the MDS-UPDRS parts I-III score worsened by 2.72 points with nicotinamide riboside relative to placebo (P = .02), and nonmotor symptom burden worsened more on treatment (NMSS difference 4.22 points, P = .009). The findings do not support nicotinamide riboside as a disease-modifying treatment.

    Why it matters: Phase 3 trial shows a proposed disease-modifying therapy for Parkinson disease was ineffective and worse than placebo.

Oct 8Thu
  1. JAMA59

    Advance Care Planning for Black and White Older Adults With Serious Illness: The EQUAL ACP Randomized Trial.

    Facilitated and self-guided advance care planning produced similar 1-year completion rates overall, but the facilitated approach worked relatively better for Black participants. Among Black participants, 71% completed ACP with facilitated versus 62% with self-guided care; among White participants, completion was 68% versus 78%. The race-by-intervention interaction was significant (adjusted RR, 1.32 [95% CI, 1.03-1.69]) among 789 participants, and facilitator racial concordance showed no association with completion.

Oct 7Wed
  1. biospace.com40

    Genmab and AbbVie’s Epkinly win, a rush of deals, Sarepta’s turnaround hopes, more

    Genmab and AbbVie's bispecific antibody Epkinly, approved in May 2023 for diffuse large B cell lymphoma after at least two prior systemic lines, cut risk of disease progression or death by 51% in a Phase 3 trial when combined with R-CHOP in the frontline setting. In deals, Shionogi bought IntraBio for $2 billion, Novartis committed up to $7.8 billion to Abogen Biosciences, Eli Lilly added up to $870 million with Gate Bioscience, and Sanofi and Regeneron renewed their collaboration with a $1 billion upfront. Sarepta shared Elevidys data in older Duchenne patients at the World Muscle Society meeting, with Jefferies citing upcoming readouts and an FDA decision on Amondys 45 and Vyondys 53.

  2. JAMA61

    Smoking Cessation Among Underserved Patients Referred for Lung Cancer Screening: A Randomized Clinical Trial.

    Financial incentives of up to $600 roughly doubled sustained tobacco abstinence among medically underserved adults referred for lung cancer screening. In the trial of 3220 patients, biochemically confirmed abstinence through 6 months was 8.8% with incentives added to usual care and free pharmacotherapy, versus 4.3% with ask-advise-refer alone and 5.1% with pharmacotherapy added. Free pharmacotherapy alone did not improve abstinence over usual care.

  3. JAMA63

    Iron or Multiple Micronutrient Powder Supplements With Malaria Chemoprevention in Rural Malawian Children: The IRMA Randomized Clinical Trial.

    Universal iron supplementation, given with malaria chemoprevention, did not improve cognitive outcomes in rural Malawian infants. In the IRMA randomized trial of 2168 six-month-old infants, neither iron syrup (MD, -0.52 [95% CI, -2.52 to 1.48]) nor iron-containing micronutrient powders (MD, -0.85 [95% CI, -2.80 to 1.09]) differed from malaria chemoprevention alone on the Bayley-III Cognitive Scale composite score at 1 year of age. Both iron interventions raised ferritin but did not reduce anemia prevalence, and neither increased malaria risk.

    Why it matters: It tests WHO-recommended universal iron intervention in a randomized trial and finds no cognitive benefit.

Oct 6Tue
  1. N Engl J Med84

    Obexelimab for the Treatment of IgG4-Related Disease.

    Weekly subcutaneous obexelimab, 250 mg for 52 weeks, cut the risk of IgG4-related disease flare requiring rescue therapy by more than half in a phase 3 trial of 194 patients (hazard ratio 0.44; flares in 26.8% vs. 54.6% with placebo). Complete remission at week 52 was reached by 37.1% versus 19.6%, and cumulative glucocorticoid rescue dose was 329.5 mg versus 929.8 mg. The results position this non-depleting anti-CD19/FcγRIIb antibody as an alternative to glucocorticoid-dependent management.

    Why it matters: Phase 3 evidence that a non-depleting B-cell-targeted antibody reduces flares and steroid exposure in IgG4-related disease.

Oct 5Mon
  1. nature.com54

    Oral small-molecule GLP-1RA safiglipron versus dapagliflozin in type 2 diabetes: a randomized, double-blind, active-comparator-controlled phase 3 trial

    Once-daily oral safiglipron, a small-molecule GLP-1RA, was non-inferior to dapagliflozin for HbA1c reduction at all tested doses and superior at 90 mg in adults with type 2 diabetes in a randomized, double-blind, active-comparator-controlled phase 3 trial. Safiglipron gave greater improvements in glycemic control, similar modest weight loss, and an acceptable safety profile. It offers an oral alternative in the GLP-1 receptor agonist class.

    Why it matters: Phase 3 data show an oral GLP-1RA matching and at 90 mg beating dapagliflozin on HbA1c.

Oct 3Sat
  1. N Engl J Med73

    Phase 3 Trial of Weekly Oral Islatravir-Lenacapavir for HIV-1 Treatment.

    Once-weekly oral islatravir-lenacapavir maintained viral suppression as well as once-daily bictegravir-emtricitabine-tenofovir alafenamide in a phase 3 trial of 607 adults with virologically suppressed HIV-1. At week 48, HIV-1 RNA of 50 copies per milliliter or higher was reported in no ISL/LEN participants and 1 (0.3%) on B/F/TAF, meeting a 4-percentage-point noninferiority margin. The finding supports a long-acting weekly oral option for suppressed patients.

    Why it matters: Phase 3 data show a weekly oral HIV regimen can replace daily tablets without loss of suppression.

Oct 2Fri
  1. N Engl J Med69

    Survodutide Once Weekly in Adults with Obesity and Type 2 Diabetes.

    Once-weekly survodutide, a glucagon receptor-GLP-1 receptor dual agonist, produced mean weight reductions of 8.2% at 3.6 mg and 9.8% at 6.0 mg over 76 weeks in 752 adults with type 2 diabetes and BMI of 27 or more, versus 3.9% with placebo. At least 5% weight loss occurred in 57.6% and 64.5% of the survodutide groups versus 35.1% on placebo, with glycated hemoglobin falling 0.9 and 0.8 percentage points. Gastrointestinal adverse events, mostly mild to moderate, occurred in 72.8% and 77.7% of survodutide recipients.

    Why it matters: Phase 3 data show the dual agonist survodutide beats placebo for weight loss in adults with obesity and type 2 diabetes.

  2. Lancet44

    8 weeks versus 12 weeks of sofosbuvir-velpatasvir for treatment-naive, non-cirrhotic, chronic hepatitis C (RESOLVE): a multicentre, open-label, non-inferiority, randomised controlled trial in India.

    Eight weeks of sofosbuvir-velpatasvir cleared chronic hepatitis C as well as the standard 12 weeks in treatment-naive, non-cirrhotic patients. In the per-protocol analysis, SVR12 was achieved by 410 of 415 participants with 8 weeks and 397 of 401 with 12 weeks, meeting the non-inferiority margin. The intention-to-treat analysis also showed non-inferiority, with no drug-related serious adverse events.

  3. Lancet71

    Cardiovascular safety of orforglipron versus insulin glargine in adults with type 2 diabetes at increased cardiovascular risk (ACHIEVE-4): a phase 3, event-driven, randomised, open-label, non-inferiority, active comparator trial.

    Orforglipron, an oral non-peptide GLP-1 receptor agonist, was non-inferior to insulin glargine for four-component major adverse cardiovascular events in 2749 adults with type 2 diabetes at increased cardiovascular risk: MACE-4 occurred in 57 (4·2%) of 1358 orforglipron participants versus 67 (5·0%) of 1343 insulin glargine participants (HR 0·84; 95% CI 0·59-1·20) over a median follow-up of 2 years. Clinically significant or severe hypoglycaemia was less frequent with orforglipron (93, 6·8%) than insulin glargine (260, 19·2%), while gastrointestinal adverse events were more frequent (62·1% vs 14·2%). The results establish cardiovascular safety for a non-peptide GLP-1 receptor agonist, a class not previously studied for this outcome.

    Why it matters: First cardiovascular safety data for an oral non-peptide GLP-1 receptor agonist, a class not previously studied for cardiovascular outcomes.

Oct 1Thu
  1. JAMA59

    Permissive Blood Pressure Targets in Pediatric Critical Care: The PRESSURE Randomized Clinical Trial.

    A permissive mean arterial pressure target in hypotensive, invasively ventilated children did not change a composite of 30-day mortality and ventilation duration versus usual care (probabilistic index, 0.52; P = .23), while cutting median norepinephrine-equivalent total dose by 37.5%. The trial randomized 1900 patients across 23 PICUs in 3 countries. Kidney replacement therapy, adverse events and most secondary outcomes were similar, though surviving children left the PICU sooner (median 159 vs 166 hours). Clinicians can accept lower MAP targets and reduce vasoactive exposure without worsening outcomes.

    Why it matters: Large multicenter trial showing lower blood pressure targets in PICUs reduce vasoactive drug dose without harming 30-day outcomes.

  2. N Engl J Med82

    Retatrutide, a Triple Hormone Receptor Agonist, for Treatment of Obesity.

    Once-weekly retatrutide produced mean body-weight reductions of 17.6%, 23.7%, and 25.0% at 4, 9, and 12 mg over 80 weeks in 2339 adults with obesity without diabetes, versus 3.9% with placebo. In subgroups, WOMAC pain fell 3.5 and 3.6 points at 9 and 12 mg versus 1.9 with placebo in 574 participants with knee osteoarthritis, and apnea-hypopnea events per hour fell 34.3 and 31.7 versus 9.9 in 243 participants with obstructive sleep apnea. The phase 3 results extend triple-receptor agonist efficacy beyond weight to obesity-related pain and sleep outcomes.

    Why it matters: Phase 3 data show retatrutide's weight loss and benefits in knee osteoarthritis pain and obstructive sleep apnea.

  3. N Engl J Med64

    Giredestrant plus Everolimus in Advanced Breast Cancer.

    An all-oral regimen of giredestrant plus everolimus significantly prolonged progression-free survival over standard endocrine therapy plus everolimus in ER-positive, HER2-negative advanced breast cancer after a CDK4/6 inhibitor. Among 207 patients with ESR1-mutated tumors, median progression-free survival was 10.0 versus 5.5 months (hazard ratio 0.38); in the overall population of 373 patients, it was 8.8 versus 5.5 months (hazard ratio 0.56). Adverse-event rates were similar between groups.

    Why it matters: Phase 3 data show an all-oral targeted combination doubles progression-free survival after CDK4/6 inhibition, especially in ESR1-mutated tumors.

  4. Nat Med53

    Oral small-molecule GLP-1 receptor agonist safiglipron in early type 2 diabetes: a randomized, double-blind, placebo-controlled trial.

    Once-daily oral safiglipron reduced HbA1c by 1.20 to 1.45 percentage points versus placebo at 32 weeks in 284 adults with early type 2 diabetes managed with diet and exercise alone (OUTSTAND-1, phase 3). HbA1c below 7.0% was reached by 71.4-77.8% of safiglipron recipients versus 25.0% on placebo, with modest weight loss and mostly mild or moderate gastrointestinal adverse events. It offers an oral GLP-1 receptor agonist option taken without fasting or dietary restrictions.

    Why it matters: Phase 3 evidence that an oral small-molecule GLP-1 receptor agonist matches injectable-class glycemic efficacy.

  5. Lancet73

    Retatrutide in adults with obesity and type 2 diabetes (TRIUMPH-2): a double-blind, parallel-group, randomised, placebo-controlled, phase 3 trial.

    Once-weekly retatrutide produced dose-dependent weight loss in adults with obesity and type 2 diabetes over 80 weeks: mean bodyweight change was -16·8% with 9 mg and -18·8% with 12 mg versus -5·1% with placebo. In the phase 3 TRIUMPH-2 trial of 1152 participants, gastrointestinal adverse events were the most frequent, and discontinuation due to adverse events or death was higher with 9 mg and 12 mg than with placebo. The findings support retatrutide as a treatment option for obesity in people with type 2 diabetes.

    Why it matters: Phase 3 data show triple-agonist retatrutide achieves up to -18·8% bodyweight change in obesity with type 2 diabetes.

  6. Nat Med60

    Werner helicase inhibitor for advanced microsatellite instability solid tumors: a phase 1 trial.

    RO7589831 (VVD-133214), a first-in-class covalent inhibitor of Werner syndrome helicase, showed manageable safety and early antitumor activity in 88 patients with MSI and/or mismatch repair-deficient advanced solid tumors in part 1 of a phase 1 trial. TEAEs were mostly grade 1-2, with no grade 5 events and discontinuation in 3.4% (3/88). In 66 MSI efficacy-evaluable patients, disease control rate was 74.2%, objective response rate 10.6% (7 partial responses), median PFS 6.7 months and median OS 17.6 months; 150 mg and 600 mg BID were selected as recommended phase 2 doses.

    Why it matters: First-in-human data on a WRN covalent inhibitor, a synthetic lethal target for MSI cancers.

Sep 30Wed
  1. nature.com70

    Intravenous hyaluronidase-expressing oncolytic adenovirus with chemotherapy in metastatic pancreatic cancer: a randomized phase 2b trial

    Intravenous zabilugene almadenorepvec (VCN-01), a hyaluronidase-expressing oncolytic adenovirus, plus gemcitabine and nab-paclitaxel prolonged overall survival over chemotherapy alone in the randomized phase 2b VIRAGE trial in treatment-naive metastatic pancreatic cancer. The finding supports adding an oncolytic virus to standard first-line chemotherapy in this setting.

    Why it matters: First randomized evidence that an intravenous oncolytic adenovirus improves survival in metastatic pancreatic cancer.

Sep 29Tue
  1. nature.com54

    Werner helicase inhibitor for advanced microsatellite instability solid tumors: a phase 1 trial

    RO7589831, a first-in-class inhibitor of the synthetic lethal target Werner syndrome helicase, showed some partial responses in the dose-escalation stage of a phase 1 trial in patients with microsatellite instability and/or DNA mismatch repair-deficient solid tumors. The maximum tolerated dose was not reached.

    Why it matters: First clinical data for a Werner helicase inhibitor, a new synthetic lethal strategy in MSI tumors.

  2. nature.com54

    Oral small-molecule GLP-1 receptor agonist safiglipron in early type 2 diabetes: a randomized, double-blind, placebo-controlled trial

    Once-daily oral small-molecule GLP-1 receptor agonist safiglipron reduced HbA1c by up to 1.45% versus placebo over 32 weeks in people with early type 2 diabetes not taking glucose-lowering medication, in the randomized OUTSTAND-1 trial. Body weight effects were modest. It offers an oral option with glycemic efficacy in this population.

    Why it matters: Oral small-molecule GLP-1 receptor agonists would replace injectable dosing for type 2 diabetes.

Sep 24Thu
  1. Nature Medicine56

    Evorpacept plus trastuzumab, ramucirumab and paclitaxel in HER2-positive gastric cancer: a randomized phase 2 trial

    Adding the CD47 inhibitor evorpacept to trastuzumab, ramucirumab and paclitaxel (TRP) produced a higher objective response rate than TRP alone in patients with HER2-positive advanced gastric cancer that had progressed on anti-HER2 treatment. The finding comes from the phase 2 part of the randomized ASPEN-06 trial. Response was associated with CD47 and HER2 expression.

    Why it matters: It tests CD47 blockade as a way to overcome resistance to anti-HER2 therapy in gastric cancer.

Sep 22Tue
  1. Nature Medicine50

    Performance and safety of a multi-cancer early detection test: the PATHFINDER 2 study

    The PATHFINDER 2 study evaluated the safety and performance of a blood-based multi-cancer early detection test in an intended-use population of more than 35,000 participants aged 50 years or older. It reports outcomes on how the test performs when used prospectively in people without a prior cancer diagnosis. The findings add interventional evidence on safety and performance in the intended-use setting.

    Why it matters: Large interventional evidence on a multi-cancer early detection blood test in its intended-use population.

  2. Nature Medicine52

    Performance of a multi-cancer early detection test in the randomized controlled NHS-Galleri trial

    Secondary endpoint analyses from the NHS-Galleri randomized controlled trial characterize how a blood-based multi-cancer early detection test performed when added to usual care in more than 142,000 participants without suspicion of cancer. The trial evaluated the test's performance as a screening tool in a general population, offering data on its behavior outside diagnostic settings.

    Why it matters: Reports performance data for a multi-cancer early detection blood test in a randomized trial of over 142,000 people.

  3. Nature Medicine52

    Trastuzumab plus nivolumab with gemcitabine and cisplatin as first-line therapy for HER2-positive unresectable biliary tract cancer: a phase 1b/2 trial

    A phase 1b/2 trial tested trastuzumab combined with nivolumab, gemcitabine and cisplatin as first-line therapy for HER2-positive unresectable biliary tract cancer. The regimen showed encouraging clinical response rates, and HER2 expression was associated with clinical benefit. The results support HER2 status as a marker guiding trastuzumab-based treatment in this population.

    Why it matters: First data on adding trastuzumab and nivolumab to standard gemcitabine-cisplatin in HER2-positive biliary tract cancer.

  4. JAMA47

    Oxygen vs Air at Birth for Moderate- to Late-Preterm Infants: The AIROPLANE Cluster Randomized Crossover Trial.

    Starting resuscitation of 32-to-35-week neonates with Fio2 0.30 instead of 0.21 did not change the primary outcome: ongoing respiratory support when leaving the delivery room occurred in 72.6% vs 73.3% (risk difference, -0.83). In the cluster randomized crossover trial across 26 Australian maternity hospitals, 1818 newborns were enrolled, and Fio2 0.30 was associated with less escalation of delivery room support and less endotracheal ventilation beyond the delivery room (6.4% vs 9.3%).

    Why it matters: It answers an open question about initial oxygen for moderate- to late-preterm resuscitation with a large randomized comparison.

Sep 17Thu
  1. medrxiv.org42

    Validating LLM judges for automated oversight of patient communication

    LLM judges match clinician ratings for safety screening and factual verification of patient communication about trial informed consent (Spearman ρ > 0.80), but agree less on communication quality (ρ < 0.60). Using a seven-criterion rubric validated on ICON-Bench, 19 LLM judges were benchmarked; safety-specialized guard models underperformed general-purpose models, and patient advocates rated communication quality lower than both clinicians and LLM judges. The results support LLM judges for scalable oversight, with recalibration needed to patient-centered standards.

    Why it matters: It quantifies where LLM judges can and cannot replace clinician review of patient-facing communication.

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