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New England Journal of Medicine, Volume 394, Issue 10, Page 969-982, March 5, 2026.

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New England Journal of Medicine, Ahead of Print.

New England Journal of Medicine, Ahead of Print.

New England Journal of Medicine, Ahead of Print.

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Neoadjuvant gemcitabine–oxaliplatin, lenvatinib, and anti–PD-1 antibody led to longer event-free survival than surgery alone in resectable high-risk intrahepatic cholangiocarcinoma, with mainly low-grade adverse events.

This review summarizes advances in radiotherapy that reduce injury to normal tissue, including improvements in the precision of imaging and delivery, and outlines strategies to prevent and manage treatment-related side effects.

A CD19-targeted therapy induced remission in a woman with refractory immune thrombocytopenia and antiphospholipid syndrome by stabilizing platelet counts, eliminating pathogenic antibodies, and enabling safer long-term anticoagulation.

In a phase 1 study of the oral p53 reactivator rezatapopt in heavily pretreated patients with TP53 Y220C–mutated solid tumors, the most common adverse events were nausea and vomiting, and the overall response was 20%.

In patients with HER2-positive early breast cancer and residual disease after neoadjuvant therapy, trastuzumab deruxtecan improved invasive disease–free survival but carried a risk of interstitial lung disease.

When a physician’s own incidental finding sends her down a long and winding path to find answers, she finds herself overcome with worry — but also, eventually, a new appreciation for life.

Recent FDA actions related to leucovorin have caused confusion. There is still a lack of scientific evidence establishing that the drug is a safe and effective treatment for autism.

Medicare will achieve greater savings on the 15 drugs in its second round of price negotiations than it did in the first round. Despite new challenges, the effort should continue to pay growing dividends.

The author describes the scientific foundations of a study of rezatapopt, which targets a variant form of p53, to treat patients with solid tumors.

Over the course of the past three decades, human epidermal growth factor receptor 2 (HER2)–targeted treatment has dramatically transformed outcomes for patients with HER2-positive breast cancer across both early- and late-stage disease. HER2 has also emerged as a clinically meaningful biomarker in several tumor types, with HER2-targeted therapies showing substantial...

In relapsed or refractory multiple myeloma, teclistamab plus daratumumab significantly prolonged progression-free survival as compared with DPd or DVd (83.4% vs. 29.7%) and led to higher response rates.

Gene therapy for cystinosis, a lysosomal storage disease, led to decreased white-cell cystine levels and stopping or decreasing cysteamine; adverse events were mainly consistent with the myeloablative regimen and underlying disease.

Cystinosis is a prototypical lysosomal storage disease caused by recessive pathogenic variants in CTNS, which encodes cystinosin, a ubiquitous proton-driven lysosomal cystine transporter.1 Loss of cystinosin leads to intralysosomal cystine accumulation, impaired lysosomal signaling, defective autophagy, and widespread cellular dysfunction.2-4 The most severe and...

Progress in the treatment of relapsed or refractory multiple myeloma has been rapid since the introduction of T-cell–redirecting immunotherapies. The identification of B-cell maturation antigen (BCMA) led to the approvals of three bispecific antibodies, two chimeric antigen receptor T-cell (CAR-T) therapies, and an antibody drug conjugate. Talquetamab, a bispecific antibody...

A 91-year-old man was admitted to the hospital with weight loss, dyspnea, and eosinophilia. An anteroposterior chest radiograph obtained 2 months earlier showed bibasilar patchy opacities. A diagnosis was made.

In Latin America, though overall smoking rates have declined, nondaily and low-intensity smoking are increasingly common, and tobacco-cessation resources remain underdeveloped in many countries.

Survival in patients with cancer, affected by myriad factors, is notoriously difficult to predict. But while oncologists find it hard to communicate the uncertainty, patients are the ones who must live it.

In cisplatin-ineligible patients with muscle-invasive bladder cancer, enfortumab vedotin–pembrolizumab plus surgery led to better event-free survival (74.7%, vs. 39.4%) and overall survival (79.7%, vs. 63.1%) than surgery alone at 2 years.

Although bladder preservation with trimodal therapy is a category 1 recommendation in the National Comprehensive Cancer Network (NCCN) guidelines for appropriately selected patients with muscle-invasive bladder cancer, the real-world adoption of this type of therapy in the United States remains disappointingly low. Contemporary data suggest that only half the patients...

VITT is caused by a somatic hypermutation of an anti–adenovirus pVII antibody that generates more avid binding of platelet factor 4 than of adenovirus pVII, its original target, which results in platelet activation.

On Feb 4, 2026—World Cancer Day—the long-overdue National Cancer Plan for England was finally published. The plan sets ambitious targets, promising that three quarters of all patients diagnosed with cancer will survive at least 5 years and that the UK will become a global leader in cancer survival by 2035. However, the feasibility of these ambitions is doubtful. The UK's current 5-year survival is among the lowest of all high-income countries, and internal reviews have presented the National Health Service (NHS) as broken.

The Lancet Oncology Global Burden of Diseases, Injuries, and Risk Factors Study (GBD) study on global breast cancer burden provides comprehensive epidemiological trends from 1990 to 2023, with projections to 2050.1 According to this study, breast cancer was the most diagnosed cancer up until 2023.2 Although this ambitious undertaking addresses crucial needs in global oncology, several methodological concerns warrant attention before these estimates inform policy decisions and resource allocation.

The therapeutic landscape of early-stage HER2-positive breast cancer has been transformed over the past two decades.1 The introduction of HER2-directed therapy, including the antibody trastuzumab, has fundamentally altered the natural history of this disease, converting an aggressive breast cancer subtype associated with poor outcomes into one with high rates of pathological complete response and excellent long-term survival.1,2 These gains were initially achieved using intensive multi-agent regimens incorporating HER2-directed therapies with anthracyclines, taxanes, and platinum chemotherapy in both the adjuvant and neoadjuvant settings.

Metastatic uveal melanoma is a rare and life-threatening cancer with limited treatment options. A characteristic feature of uveal melanoma is its strong liver tropism, resulting in liver metastases in approximately 90% of patients who develop metastatic disease. This distinct pattern has led to investigations of liver-directed therapeutic strategies in patients with uveal melanoma.

Timo F W Soeterik and colleagues1 are to be congratulated for their important contribution to guiding personalised treatment in patients with oligorecurrent prostate cancer, published in The Lancet Oncology. In this multicentre retrospective analysis, the authors curated a cohort of more than 500 men with metachronous oligometastatic prostate cancer who underwent prostate-specific membrane antigen (PSMA) PET for disease characterisation, followed by ablative radiotherapy to all sites of oligometastases.

20 years ago, the combination of radiotherapy with concomitant and adjuvant temozolomide, also known as the Stupp protocol,1 transformed glioblastoma treatment. It was the first multimodal regimen to show a survival advantage in newly diagnosed glioblastoma and is the standard of care two decades later. The trial1 identified a biomarker (MGMT) to predict benefit from temozolomide; yet this represents only the beginning of what biologically informed patient selection could achieve. Advances in single-cell profiling, spatial imaging, and systems immunology are revealing fundamental determinants of glioblastoma behaviour, including dynamic cell states, lineage plasticity, metabolic constraints, and the role of the brain's resident and infiltrating immune cells.

In December, 2025, the Trump administration announced completion of the second cycle of historic price negotiations between US Medicare and pharmaceutical manufacturers.1 Concurrently, it released plans to establish international reference pricing to lower the costs of cancer drugs and other medicines in Medicare to levels in peer countries.2 These policy changes could have important effects on access to cancer medicines globally.

I appreciate the excellent work by Natólia Alves and colleagues,1 comparing artificial intelligence (AI) and radiologists in pancreatic cancer detection using standard of care CT scans (PANORAMA study). The paper is a milestone in evaluating the real-world diagnostic performance of an AI system for the detection of pancreatic ductal adenocarcinoma (PDAC) on contrast-enhanced CT. This important advance will facilitate PDAC surveillance of individuals who are at high risk.2 However, PANORAMA study's AI system1 is not suitable for PDAC screening of people who are at average risk of PDAC and are asymptomatic.

We thank Hans Scherübl and Misha D P Luyer1 for their insightful comments on the PANORAMA study,2 regarding the challenges and opportunities of artificial intelligence (AI)-assisted detection of pancreatic ductal adenocarcinoma (PDAC), particularly in early-stage disease.

We congratulate Huiping Li and colleagues on the FABULOUS trial,1 which establishes fuzuloparib, with or without apatinib, as a viable option for HER2-negative metastatic breast cancer with germline BRCA1/2 mutations. As clinicians practising in a prefecture-level hospital in the Hunan province, we consider these findings from the perspective of accessibility and real-world applicability in resource-constrained settings.

We thank Ziran Qiu and Na Jin for their Correspondence regarding the FABULOUS trial1 and appreciate their practical perspectives.

We read with great interest the phase 2 KUNPENG trial by Yi-Long Wu and colleagues.1 The reported objective response rate of 48·8% with vebreltinib in MET amplification-driven non-small-cell lung cancer is promising. However, the use of a gene copy number (GCN) of six or higher as the sole enrolment criterion obscures crucial biological heterogeneity within this population. This methodological choice fundamentally limits the interpretability of the efficacy signal and poses a considerable risk for the design of the planned phase 3 trial.

We thank Yuxin Liu and colleagues for their interest in the KUNPENG study and concur that a clinically meaningful MET amplification cutoff is crucial to trial design and interpretation.1 Indeed, our selection of a gene copy number (GCN) of six or higher assessed by fluorescence in-situ hybridisation (FISH) was grounded in a biological rationale and clinical precedent. Mechanistically, MET amplification promotes oncogenesis through GCN gain-induced MET protein phosphorylation, and a GCN of six or higher confers a potent oncogenic driver function independent of hepatocyte growth factor.

The National Health Service (NHS) in England, UK, has set up the world's first national genetic cancer risk register to help track and screen individuals at higher risk of developing cancer and offer preventive and personalised care.

Lithuania has now joined a growing list of more than 100 countries worldwide that have introduced a tax on sugar-sweetened beverages (SSBs). The tax, introduced from Jan 1, 2026, means the Baltic nation is one of many countries using fiscal policies to help encourage healthier diets across its population. Lithuania will also be taxing artificially sweetened beverages.

On Jan 27, 2026, India and the EU announced the conclusion of a comprehensive free trade agreement (FTA) as part of the 16th EU–India Summit, held in New Delhi, India. The new agreement creates a free trade zone affecting more than 2 billion people by eliminating or reducing tariffs on chemical inputs, medical devices, and pharmaceuticals, including cancer drugs.

On Jan 27, 2026, Zimbabwe's Medicines Control Authority (MCAZ) alerted citizens about the circulation of an unauthorised formulation of goserelin acetate, a hormone therapy used in the treatment of prostate and breast cancer. It warned that the product could pose risks to patient safety. “Our records show that this product was not formally imported into Zimbabwe. The MCAZ cannot therefore ascertain the safety, quality and efficacy of this product”, the alert read.

The UK National Health Service (NHS) will meet all three core cancer standards by 2029, according to a new National Cancer Plan for England, published on Feb 4, 2026. This is despite national treatment targets having not been hit for more than a decade.

A coalition of 13 Democratic state attorney generals is urging the Trump administration to drop a “dangerous and scientifically unsupported” revision to a federal formaldehyde-inhalation risk assessment they say would put workers, consumers, and communities at risk of formaldehyde-linked cancers and asthma.

On Feb 4, 2026, Health Minister Stéphanie Rist announced that the French Government will launch a targeted lung cancer screening trial in March, 2026, with the aim of making screening systematic by 2030.

The US Food and Drug Administration (FDA) has fast-tracked a promising new gene therapy, KB707, which is inhaled as a fine mist for the targeted treatment of advanced or metastatic non-small-cell lung cancer.

The Musée Curie, nestled in the historic heart of Paris near the Panthéon, offers a compelling journey through the extraordinary legacy of Marie and Pierre Curie—pioneers whose groundbreaking work in radioactivity transformed both science and medicine. As a dedicated centre for preserving and showcasing their contributions, the museum provides invaluable insights into the origins of radiological research and its profound impact on modern cancer therapies.

Earthquakes constitute major natural hazards, with the potential to cause extensive damage to infrastructure, substantial loss of life, and economic consequences. Türkiye is situated at the intersection of the Arabian and Eurasian tectonic plates and is traversed by both the North Anatolian and East Anatolian fault lines, resulting in high seismic activity across the region. In the last century, the country has experienced 16 major earthquakes with magnitudes of Mw 7·0 or higher. Among these earthquakes were the two major seismic events of 2023 (Mw 7·7 and Mw 7·6), which profoundly impacted southeastern Türkiye and northern Syria, resulting in more than 50 000 deaths and widespread structural destruction, including critical damage to health care facilities.

This 10-year follow-up confirms the non-inferiority of the ultra-hypofractionated radiotherapy regimen compared with the conventionally fractionated, with similar toxicity profiles. The findings support the seven-fraction schedule as a safe, effective, and practical standard-of-care option for patients with intermediate-risk prostate cancer.

The stable incidence and declining mortality rates of female breast cancer in high-income nations reflect success in screening, diagnosis, and treatment. In contrast, the concurrent rise in incidence and mortality in other regions signals health system deficits. Without effective interventions, many countries will fall short of the WHO Global Breast Cancer Initiative's ambitious target of achieving an annual reduction of 2·5% in age-standardised mortality rates by 2040. The mounting breast cancer burden, disproportionately affecting some of the world's most vulnerable populations, will further exacerbate health inequalities across the globe without decisive immediate action.

Vibostolimab coformulated with pembrolizumab did not provide additional clinical benefit versus pembrolizumab as adjuvant therapy in participants with resected stage IIB–IV melanoma. Pembrolizumab monotherapy remains a standard of care for resected high-risk melanoma.

Global access to brachytherapy remains profoundly unequal, with adequate coverage largely limited to HICs. Resource gaps are greatest in lower-income settings, where patients also travel farthest. Investment in new centres is urgently needed to reduce geographical and economic barriers to access.

MRI-guided optimisation of neoadjuvant chemotherapy duration was associated with favourable 3-year event-free survival outcomes in patients with stage II–III HER2-positive breast cancer. This approach represents a novel strategy that reduces treatment burden, minimises toxicity, and preserves quality of life in a subset of patients with early HER2-positive breast cancer.

Cancer-related NHS hospital care costs by stage at diagnosis differed between cancer types; this heterogeneous pattern could inform detailed and nuanced economic evaluations of early detection initiatives.

Adding ipilimumab and nivolumab to percutaneous hepatic perfusion significantly improved progression-free survival, but with a higher rate of adverse events. The combination therapy offers a promising new treatment paradigm for patients with metastatic uveal melanoma. These results would ideally be validated in larger, multicentre randomised trials; however, conducting such studies is challenging due to the low incidence of uveal melanoma.

This nomogram predicts ADT-free survival after MDT in oligorecurrent prostate cancer. While individual-level discrimination was modest, risk-group stratification showed meaningful separation, supporting its potential use in treatment selection.

Concurrent chemoradiotherapy followed by immune checkpoint inhibitor (ICI) consolidation is now the standard of care for unresectable stage III non-small-cell lung cancer (NSCLC) and is increasingly applied to other thoracic malignancies. Although survival outcomes have improved, concerns about cardiac toxicity have emerged, as both chemoradiotherapy and ICIs are independently cardiotoxic and their combined effects remain unclear. This scoping review first examines chemoradiotherapy-associated and ICI-associated cardiac toxicity separately, and then evaluates their convergence in combined chemoradiotherapy and ICI therapy.

Currently, no consensus exists regarding the definition of oligometastatic pancreatic ductal adenocarcinoma, its necessary diagnostic measures, and potential treatment approaches. To address these knowledge gaps, the OligoPanc project brought together an interdisciplinary group of experts to establish consensus using a modified Delphi process and clinical vignettes. Participants agreed that the number of metastatic lesions and the number of affected organs are key elements in defining oligometastatic pancreatic ductal adenocarcinoma.

Radiotherapy and systemic therapies have revolutionised cancer treatments and improved survival, with generally acceptable toxicity. Modern radiotherapy techniques offer precision in targeting tumours, while minimising harm to the surrounding tissues. Novel systemic therapies represent opportunities for combining innovative treatments with radiotherapy. New agents are increasingly more tumour-specific than chemotherapy, and new rational combinations with radiotherapy are emerging, allowing for a more personalised approach.

This international Delphi study, led by the European Society of Radiation Oncology as part of their Value-Based Radiation Oncology programme, brought together key experts from the radiation oncology community to build consensus on both the level of evidence and the endpoints that are essential to support clinical implementation or policy decisions (eg, reimbursement) for different types of radiotherapy innovations. Although randomised trial evidence remained a high priority across most innovation types, other evidence, such as high-quality prospective observational studies or alternative designs such as pragmatic trials, was found to be a suitable alternative in specific scenarios.

Historically, perioperative cisplatin based neoadjuvant therapy has remained the gold standard for muscle-invasive bladder cancer. In the past 5 years, several practice changing trials have enabled the incorporation of immune checkpoint inhibitors in the perioperative setting for muscle-invasive bladder cancer. The phase 3 NIAGARA study led to the approval of durvalumab in combination with gemcitabine or cisplatin followed by adjuvant immune checkpoint inhibitor monotherapy.1 The CheckMate-274 and AMBASSADOR trials showed improved outcomes in patients treated with adjuvant immune checkpoint inhibitors.

Perioperative sacituzumab govitecan plus pembrolizumab revealed a promising clinical complete response rate, without the occurrence of grade 4 or higher adverse events, allowing a bladder preservation with sustained remission in approximately 40% of patients.

On Feb 15 (commemorating the World Childhood Cancer Day), Jordan announced three linked initiatives: a National Cancer Control Strategy 2026–2030, six national paediatric cancer clinical guidelines, and its entry into the Global Platform for Access to Childhood Cancer Medicines. The event was jointly organised by the Ministry of Health and WHO under the patronage of Princess Ghida Talal of the King Hussein Cancer Foundation and King Hussein Cancer Center (KHCC; Amman, Jordan). The event also included participation of civil society organisations supporting patients with cancer (among panellists), to foster meaningful engagement of patients in line with WHO guidance.

The US Food and Drug Administration (FDA) has fast-tracked a promising new gene therapy, KB707, which is inhaled as a fine mist for the targeted treatment of advanced or metastatic non-small-cell lung cancer.

Timo F W Soeterik and colleagues1 are to be congratulated for their important contribution to guiding personalised treatment in patients with oligorecurrent prostate cancer, published in The Lancet Oncology. In this multicentre retrospective analysis, the authors curated a cohort of more than 500 men with metachronous oligometastatic prostate cancer who underwent prostate-specific membrane antigen (PSMA) PET for disease characterisation, followed by ablative radiotherapy to all sites of oligometastases.

The therapeutic landscape of early-stage HER2-positive breast cancer has been transformed over the past two decades.1 The introduction of HER2-directed therapy, including the antibody trastuzumab, has fundamentally altered the natural history of this disease, converting an aggressive breast cancer subtype associated with poor outcomes into one with high rates of pathological complete response and excellent long-term survival.1,2 These gains were initially achieved using intensive multi-agent regimens incorporating HER2-directed therapies with anthracyclines, taxanes, and platinum chemotherapy in both the adjuvant and neoadjuvant settings.

This nomogram predicts ADT-free survival after MDT in oligorecurrent prostate cancer. While individual-level discrimination was modest, risk-group stratification showed meaningful separation, supporting its potential use in treatment selection.

MRI-guided optimisation of neoadjuvant chemotherapy duration was associated with favourable 3-year event-free survival outcomes in patients with stage II–III HER2-positive breast cancer. This approach represents a novel strategy that reduces treatment burden, minimises toxicity, and preserves quality of life in a subset of patients with early HER2-positive breast cancer.

Cancer-related NHS hospital care costs by stage at diagnosis differed between cancer types; this heterogeneous pattern could inform detailed and nuanced economic evaluations of early detection initiatives.

Vibostolimab coformulated with pembrolizumab did not provide additional clinical benefit versus pembrolizumab as adjuvant therapy in participants with resected stage IIB–IV melanoma. Pembrolizumab monotherapy remains a standard of care for resected high-risk melanoma.

On Feb 4, 2026, Health Minister Stéphanie Rist announced that the French Government will launch a targeted lung cancer screening trial in March, 2026, with the aim of making screening systematic by 2030.

The UK National Health Service (NHS) will meet all three core cancer standards by 2029, according to a new National Cancer Plan for England, published on Feb 4, 2026. This is despite national treatment targets having not been hit for more than a decade.

On Jan 27, 2026, India and the EU announced the conclusion of a comprehensive free trade agreement (FTA) as part of the 16th EU–India Summit, held in New Delhi, India. The new agreement creates a free trade zone affecting more than 2 billion people by eliminating or reducing tariffs on chemical inputs, medical devices, and pharmaceuticals, including cancer drugs.

This 10-year follow-up confirms the non-inferiority of the ultra-hypofractionated radiotherapy regimen compared with the conventionally fractionated, with similar toxicity profiles. The findings support the seven-fraction schedule as a safe, effective, and practical standard-of-care option for patients with intermediate-risk prostate cancer.

Lithuania has now joined a growing list of more than 100 countries worldwide that have introduced a tax on sugar-sweetened beverages (SSBs). The tax, introduced from Jan 1, 2026, means the Baltic nation is one of many countries using fiscal policies to help encourage healthier diets across its population. Lithuania will also be taxing artificially sweetened beverages.

February 15 is International Childhood Cancer Day, with 2026 marking the cumulation of a 3-year campaign, “Equal Access to Care for All Children with Cancer.” 400 000 children are diagnosed with cancer annually and, by disability-adjusted life-years, childhood cancer ranks as the sixth leading cause of cancer burden and ninth highest in childhood disease burden. Childhood cancer is now the leading cause of non-communicable disease-related death among children and adolescents in low-income and middle-income countries (LMICS) as well as in high-income countries.

The editorial team at The Lancet Haematology would like to extend our thanks to the many experts who offered their time and expertise to help us make decisions on and improve papers in 2025. In this issue we publish a list of clinical and statistical peer reviewers—we hope that some small recognition of an important academic task can be given to those who give their time, knowledge, and expertise. The Journal's success was partially reflected in our 2024 Impact Factor of 17·7 and Scopus CiteScore of 23·8.

In their study in The Lancet Haematology, Mahé and colleagues report a post-hoc analysis of the API-CAT randomised, double-blind, non-inferiority trial, identifying predictors of clinically relevant bleeding during 12 months of extended apixaban therapy in patients with active cancer who had completed at least 6 months of anticoagulation for venous thromboembolism (VTE).1 Among 1766 participants, clinically relevant bleeding occurred in 238 patients (cumulative incidence 13·9%), including 61 major bleeding events (3·6%).

We read with interest the post-hoc analysis of the pivotal API-CAT trial by Isabelle Mahé and colleagues, published in The Lancet Haematology,1 that found anaemia and/or thrombocytopenia, age 75 years or older, pulmonary embolism as the index event, and male sex to be associated with an increased risk of clinically relevant bleeding during extended anticoagulation for cancer-associated venous thromboembolism (VTE). The authors concluded that these results were homogenous across cancer sites. However, upon closer inspection of the subgroup analyses, we noted potentially substantial heterogeneity among the identified predictors of clinically relevant bleeding according to cancer site.

We thank Faizan Khan and Nick van Es, and Zhang Cheng and Yiqi Jin for their Correspondence regarding our post-hoc analysis of the API-CAT trial1,2 in which we identified four non-cancer related, non-modifiable predictors of clinically relevant bleeding during 12 months of extended apixaban therapy in patients with cancer-associated venous thromboembolism (VTE).

Iron deficiency during pregnancy is associated with an increased risk of postpartum haemorrhage, preterm birth, pre-eclampsia, fetal growth restriction, maternal mortality, and neurodevelopmental abnormalities in infants and during early childhood.1,2 The American College of Obstetricians and Gynaecologists recommends prophylactic oral iron (30 mg per day) rather than treatment of non-anaemic iron deficiency due to insufficient high-quality evidence concerning the effect of treatment on fetomaternal outcomes.

Kabbara F, Charbel N, Klim J, et al. Thalassaemia clinical trials in war-stricken Lebanon: a story of struggle and resilience. Lancet Haematol 2026; 13: e6–7—In this In Focus piece, Ghida Ismail and Farah Ismail should have been included in the author list. Ghida Ismail's affiliation of EpiMaCT - Epidemiology of chronic diseases in tropical zone, Institute of Epidemiology and Tropical Neurology, Omega Health, University of Limoges, CHU Limoges, INSERM U1094, IRD U270, Limoges, France, should have been included and both Ghida Ismail and Farah Ismail should have been added to the affiliation of Chronic Care Center, Hazmieh, Lebanon.

My Blood, Your Blood, a new children's book by author and producer Laura Henry-Allain MBE and illustrated by Zenovia Grant, is full of heroes. Based on the real-life experience of 13-year-old Angel Salami, it follows her, aged 4 years, bravely enduring a sickle cell crisis and receiving a blood transfusion in hospital. Back playing with her friends at the end of the story, she pays tribute to the other anonymous heroes in the book: “Every day I think about the person whose blood was given to me.”

Allogeneic haematopoietic cell transplantation (HCT) remains a potentially curative option for patients with high-risk haematological malignancies, albeit at the cost of substantial morbidity and mortality. Advances in transplantation practices and supportive care have expanded eligibility to older patients and to those with significant comorbidities. In this evolving landscape, frailty assessment has become a crucial component of pre-transplantation evaluation, as it is consistently associated with increased transplantation-related complications, prolonged hospitalisation, and inferior survival.

Var-cel induced deep remissions with low incidence of severe cytokine release syndrome and any-grade immune effector cell-associated neurotoxicity syndrome, supporting fractionated dose escalation as a strategy that preserves activity, limits acute toxic effects, and supports a hospital-based approach that could expand access to CAR T-cell therapy.

Adverse events during induction and reinduction were consistent with those reported in previous infant acute lymphoblastic leukaemia studies. Infections remain a substantial cause of morbidity and mortality in this immunocompromised patient population and extended beyond the induction and reinduction chemotherapy phases.

High-dose cytarabine and mitoxantrone plus venetoclax appeared to be safe, showed promising activity, and could be a new therapeutical approach for medically fit patients with relapsed or refractory acute myeloid leukaemia, especially as a bridge to allogeneic HCT.

The results indicate that quizartinib plus standard chemotherapy prolongs overall survival without adversely affecting patient-reported outcomes and health-related quality of life, with no substantial differences between groups. Future research in real-world settings is warranted to assess the generalisability of these patient-reported outcome results.

The distinction between myelodysplastic syndromes and acute myeloid leukaemia remains a subject of debate, with direct implications for clinical decisions, trial eligibility, and allogeneic hematopoietic cell transplantation allocation. Although the historical 20% blast threshold in bone marrow is commonly used to separate myelodysplastic syndromes from acute myeloid leukaemia, emerging evidence shows that morphological, cytogenetic, and molecular features provide a more accurate framework for diagnosis and risk stratification.

A 47-year-old man was admitted to Ruijin Hospital (Shanghai, China) in July, 2025, with widespread, tender, and violaceous cutaneous nodules on his trunk and extremities. PET–CT revealed hypermetabolic activity in the skin lesions, which involved cervical and inguinal lymph nodes (maximum standardised uptake value of 3·6–7·1). Laboratory studies revealed a white blood cell count of 11 × 109 cells per L. A skin biopsy showed a diffuse dermal infiltrate of monomorphic blastoid cells. Immunohistochemical analysis revealed that the neoplastic cells expressed CD123, TCF4, TCL1, CD4, CD56, HLA-DR, and aberrant cytoplasmic CD3 (figure A), with partial expression of CD2 and CD7.

Hemophagocytic lymphohistiocytosis (HLH) is a syndrome of life-threatening hyperinflammation that challenges pediatric and adult hematologists. Deputy Editors Helen E. Heslop and Jorge Di Paola introduce a 2-piece review series on this rare condition that, once seen, is not forgotten. Its descriptive name reflects an era when we did not understand the pathobiology of a group of diseases unified in common presenting features reflecting uncontrolled immune activation and excessive proinflammatory cytokine production. Verkamp, Jordan, and Allen address pediatric HLH, which is now well recognized to be underpinned by genetic lesions that undermine immune-cell cytotoxicity. They describe how presentations are driven by the intersection of genetic susceptibility and environmental triggers. Hsu, Nikiforow, and Berliner review adult-onset HLH, which is more heterogeneous with a wide array of provoking stimuli and which often occurs in the absence of a known predisposing genetic immune defect. Both reviews cover recent advances arising from the use of targeted therapies directed at activated T cells and cytokine signaling pathways. The role of allogeneic stem cell transplantation is emphasized for pediatric patients, and the recently described entity “immune effector cell–associated HLH-like syndrome” after chimeric antigen receptor T-cell therapy is also explained.

Hemophagocytic lymphohistiocytosis (HLH) is a syndrome of life-threatening hyperinflammation that challenges pediatric and adult hematologists. Deputy Editors Helen E. Heslop and Jorge Di Paola introduce a 2-piece review series on this rare condition that, once seen, is not forgotten. Its descriptive name reflects an era when we did not understand the pathobiology of a group of diseases unified in common presenting features reflecting uncontrolled immune activation and excessive proinflammatory cytokine production. Verkamp, Jordan, and Allen address pediatric HLH, which is now well recognized to be underpinned by genetic lesions that undermine immune-cell cytotoxicity. They describe how presentations are driven by the intersection of genetic susceptibility and environmental triggers. Hsu, Nikiforow, and Berliner review adult-onset HLH, which is more heterogeneous with a wide array of provoking stimuli and which often occurs in the absence of a known predisposing genetic immune defect. Both reviews cover recent advances arising from the use of targeted therapies directed at activated T cells and cytokine signaling pathways. The role of allogeneic stem cell transplantation is emphasized for pediatric patients, and the recently described entity “immune effector cell–associated HLH-like syndrome” after chimeric antigen receptor T-cell therapy is also explained.

Hemophagocytic lymphohistiocytosis (HLH) is a syndrome of life-threatening hyperinflammation that challenges pediatric and adult hematologists. Deputy Editors Helen E. Heslop and Jorge Di Paola introduce a 2-piece review series on this rare condition that, once seen, is not forgotten. Its descriptive name reflects an era when we did not understand the pathobiology of a group of diseases unified in common presenting features reflecting uncontrolled immune activation and excessive proinflammatory cytokine production. Verkamp, Jordan, and Allen address pediatric HLH, which is now well recognized to be underpinned by genetic lesions that undermine immune-cell cytotoxicity. They describe how presentations are driven by the intersection of genetic susceptibility and environmental triggers. Hsu, Nikiforow, and Berliner review adult-onset HLH, which is more heterogeneous with a wide array of provoking stimuli and which often occurs in the absence of a known predisposing genetic immune defect. Both reviews cover recent advances arising from the use of targeted therapies directed at activated T cells and cytokine signaling pathways. The role of allogeneic stem cell transplantation is emphasized for pediatric patients, and the recently described entity “immune effector cell–associated HLH-like syndrome” after chimeric antigen receptor T-cell therapy is also explained.

The optimal induction regimen for fit older adults with acute myeloid leukemia (AML) is controversial. Knapper and colleagues report the results of a large, randomized trial of daunorubicin (D) and cytosine arabinoside (A) with 2 doses of gemtuzumab ozogamicin (GO) (DA-GO2) vs liposomal fixed-ratio D and A (CPX-351) in AML without adverse cytogenetics, with DA-GO2 providing superior event-free and overall survival and less prolonged hematological toxicity. Now we need to know how this augmented regimen compares with lower-intensity venetoclax-based regimens.

CD22 is a potential antigen target for cellular therapy of B-cell malignancies, but expression can be low and this is a disadvantage for chimeric antigen receptor (CAR) approaches. Rhein and colleagues report a novel, high-affinity T-cell receptor (TCR) that recognizes CD22 and, when integrated into autologous T cells, results in improved antitumor activity compared to CAR T cells, both in vitro and in model systems in vivo. Use of TCRs with these qualities warrants evaluation in future clinical trials.

Anti–B-cell maturation antigen (BCMA) bispecific antibodies (bsAbs) are transforming treatment for relapsed multiple myeloma (MM), but infection is a leading cause of morbidity and mortality with these agents in this setting. Jelinek et al help explain this by demonstrating that BCMA is expressed from the early pre-B-cell maturation stage onwards, and consequently anti-BCMA bsAbs deplete B-cell subsets as well as plasma cells, especially mature B cells. In contrast, the expression of G protein–coupled receptor class C group 5 member D (GPRC5D) is highly restricted to malignant and normal plasma cells, and the data from this study suggest that, while still common, infections are less frequent with anti-GPRC5D bsAb therapy.

Prior studies indicate that quiescent, chemotherapy-resistant acute myeloid leukemia stem cells (AML LSCs) are characterized by low oxidative phosphorylation and high expression of microRNA-126 (miR-126). Zhang and colleagues demonstrate that pharmacologic inhibition of miR-126 potently perturbs the metabolic homeostasis and leukemogenic activity of AML LSCs and can resensitize resistant AML cells to venetoclax. Their data offer new mechanistic insights and offer translational promise for overcoming resistance to regimens such as venetoclax-azacitidine.

Should we test for measurable residual disease (MRD) longitudinally after MRD-negative complete remission of acute myeloid leukemia (AML) is achieved? If so, should we intervene at the point of detection of MRD reemergence? Platzbecker et al report the final results of the single-arm RELAZA2 trial, the first prospective study to evaluate the clinical impact of early therapeutic intervention for MRD relapse, using azacitidine for up to 2 years. While 63% of patients initiating on azacitidine maintained remission at 6 months, most eventually relapsed, although approximately 50% of those whose MRD converted to negative remained relapse-free more than 2 years after cessation of azacitidine, suggesting a major benefit. This pioneering study has inspired a suite of ongoing MRD intervention studies using a range of targeted agents.

Therapeutic efforts to increase nonsickling hemoglobin in sickle cell disease (SCD) have generally focused on induction of fetal hemoglobin. However, adult hemoglobin A2 (HbA2; α2δ2) also inhibits HbS polymerization. Zhu et al reveal that interferon gamma (IFN-γ) therapy produces a significant activation of δ-globin gene transcription via STAT1/3 phosphorylation and HbA2 induction in a model erythroid cell line and normal and sickle-derived erythroid progenitors. This work opens new avenues for exploration of induction of HbA2 as alternative therapy for SCD.

A significant minority of patients with advanced-stage Hodgkin lymphoma (HL) will relapse after frontline therapy, and some will have primary refractory disease. In a retrospective study of almost 1300 patients, Desai et al report that programmed death protein 1 inhibitor (PD-1i)–based salvage regimens prior to autologous transplantation are associated with improved progression-free survival compared to regimens based on chemotherapy or brentuximab vedotin, independent of potential confounders such as pretransplant remission state. These data reinforce the findings of small studies and make the case for incorporation of PD-1i in salvage for patients not previously treated with these agents in first-line therapy.

Although BR patients demonstrated a higher SPM risk at 5 years in univariate models, this finding was not present on multivariable analysis. An increased number of BR cycles was associated with borderline increased SPM risk. These findings require prospective validation and studies examining risk‐adapted abbreviation of chemotherapy.

Smoking demonstrates significant concurrent and longitudinal associations with poorer HRQOL in prostate cancer survivors, with a clear dose–response pattern. These results underscore the critical need to integrate structured smoking assessment and cessation support as a core component of comprehensive prostate cancer survivorship care.

This large‐scale multiomic analysis revealed significant associations between SCLC subtypes and specific immune signatures and comutations. These findings provide insights into the molecular heterogeneity of SCLC, and highlight potential biomarkers for targeted therapies.

Childhood overweight and obesity increase BTC risk in adulthood.

Early dosing decisions shaped subsequent chemotherapy delivery. Most patients who began at full dose maintained consistent dosing, whereas early reductions did not stave off subsequent changes, underscoring the need to balance safety with adequate dose intensity.

Patients with cancer who experience a mental health condition are at an increased risk of all‐cause mortality. This reinforces and emphasizes existing recommendations for prompt screening and management of distress and mental health following a cancer diagnosis.

Compared to a detailed informational booklet on sex/intimacy, the couple‐based AIE intervention yielded long‐term benefits for MBC patients’ sexual outcomes and partners’ sexual distress and psychosocial outcomes. Future research should identify intervention mediators and optimal dissemination methods.

Severe radiation-induced lymphopenia (RIL) during concurrent chemoradiotherapy (CCRT) for NSCLC has been associated with poorer outcomes and reduced immunotherapy efficacy. Because RIL often develops late during CCRT, identifying patients at risk before treatment may be clinically relevant. This study aimed to develop and validate a nomogram based on pretreatment predictors for severe RIL, and secondarily to explore associations between predicted RIL risk and adjuvant durvalumab-associated survival.

: Differentiating recurrent brain metastases (BM) from treatment-related changes post-radiotherapy remains challenging. PURSUE (NCT04410367) evaluated various lesion uptake metrics to establish image interpretation criteria to detect recurrent BM with 18F-fluciclovine PET.

Proton beam radiotherapy (PBT) offers superior accuracy of dose deposition, reducing the risk of adverse effects to surrounding healthy tissues. However, despite high medical need, molecular and cellular determinants of radiosensitivity to proton radiotherapy remain underexplored, and prognostic biomarkers and therapeutic targets informing precision medicine strategies for PBT are mostly missing. This study aimed to investigate the role of DNA double-strand break (DSB) repair pathways in shaping tumor response to proton versus photon radiation.

Women treated with radiation therapy (RT) for breast cancer have an increased risk of developing radiation-associated contralateral breast cancer (CBC). Predicting CBC events is challenging due to the complex interplay of genomic, treatment, personal, and clinical factors. This study investigated computational methods that integrate genome-wide single nucleotide polymorphisms (SNPs) and non-genomic data to develop a risk stratification model for developing CBC in women treated with RT for their first primary breast cancer.

Resident and faculty impressions of novel mini-fellowship concentrations in multiple disease sites during radiation oncology training were assessed.

To evaluate PSA and PSMA kinetics following PSMA-PET and MR guided stereotactic body radiotherapy (SBRT) and short-term androgen deprivation therapy (ADT) with dominant intraprostatic lesion (DIL) boost in localised prostate cancer.

For breast cancer patients undergoing mastectomy and reconstruction, postmastectomy radiation therapy (PMRT) jeopardizes reconstruction outcomes, yet no universal consensus exists for integrating PMRT with breast reconstruction. The recent expansion of proton therapy has intensified a debate over its role in this setting. While protons offer superior dosimetry and cardiac sparing compared to photons, several studies -focusing on implant-based reconstruction- report higher rates of capsular contracture and reconstruction failure with protons, whereas others show comparable protons outcomes to photons.

The multicentre, single-arm, phaseⅡSHIFT trial evaluated an ultra-hypofractionated (UHF) radiotherapy (RT) regimen that completes both whole-breast irradiation (WBI) and a sequential tumor bed boost within 1.5 weeks, with the primary endpoint of acute toxicity.

Breathing-induced motion of abdominothoracic lesions is an important source of uncertainty in radiotherapy. Beam gating improves delivery precision, but lengthens treatment delivery time, which can potentially be counteracted by biofeedback (BF). In this study, we investigated if providing BF results in higher delivery efficiency and benefits the patient experience.

The Proliferation Saturation Index (PSI) model is a patient-specific mathematical model designed to simulate and predict tumour volume regression (TVR) during radiation therapy (RT) using early treatment response dynamics. This study validates the PSI model in an independent external cohort of patients with non-small cell lung cancer (NSCLC) using previously derived model parameters.

To collect national data from medical physicists on work effort, challenges, and job satisfaction in HDR brachytherapy as a function of procedure complexity.

To assess the dosimetric impact to organs-at-risk (OARs) and target coverage of a small margin weekly adaptive treatment for glioblastoma delivered on an MR-linac.

177Lu-based radiopharmaceutical therapy (RPT) has shown increasing promise in the treatment of neuroendocrine and metastatic prostate cancer. Delivering optimal radiation dose to tumors while minimizing dose to organs-at-risk (OAR) remains an unmet need due to significant patient-to-patient heterogeneity in treatment response, necessitating multiple snapshots of the in vivo activity distribution. Towards this goal, here we present a high temporal-resolution activity reconstruction method demonstrated on preclinical prostate cancer models.

To evaluate the impact of radiotherapy on sexual health in women with gynecological cancers.

Purpose: Currently, on-board kV imaging systems on proton therapy machines typically employ scintillator-based energy-integrating detectors (EIDs) to acquire planar images or cone-beam CT (CBCT) scans. However, EID-based CBCT is limited by suboptimal image quality and poor quantification accuracy, falling short of the requirements for online adaptive proton therapy and other advanced imaging tasks for proton therapy. This study aims to experimentally demonstrate the feasibility of on-board photon-counting detector (PCD) CT imaging by integrating a PCD with a proton therapy gantry Methods and Materials: A CdTe-based PCD, featuring a pixel size of 100 μm and two energy channels, was mounted onto the surface of the existing flat-panel EIDs of an IBA Proteus ONE proton therapy system.

: Radiation dose selection for neoadjuvant chemoradiotherapy (nCRT) in esophageal squamous cell carcinoma (ESCC) varies widely in clinical practice, and prospective randomized data addressing this question are lacking. This trial compared two commonly used radiation dose regimens in nCRT for resectable thoracic ESCC.

Researchers have identified that the reporting of venous and arterial thrombotic events in cancer clinical trials is inconsistent and potentially inaccurate, according to a comments article published in the Journal of Clinical Oncology.¹

Quadrivalent human papillomavirus (HPV) vaccination led to a significantly reduced risk of invasive cervical cancer that was sustained through long-term follow-up, according to the results of a Swedish nationwide, register-based cohort study published in The BMJ. 

As reported in the Journal of Clinical Oncology by Scherpereel et al, 5-year findings from the phase III CheckMate 743 trial showed continued overall survival benefit of first-line nivolumab plus ipilimumab vs chemotherapy in patients with unresectable pleural mesothelioma.

On March 5, 2026, the U.S. Food and Drug Administration (FDA) approved teclistamab (Tecvayli) in combination with daratumumab hyaluronidase-fihj for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a...

In time for the assigned deadline of February 23, 2026, medical societies, companies, health-care systems, and more have responded to a request for information from the Department of Health and Human Services (HHS) regarding the use of artificial intelligence (AI) in clinical practice.

In a retrospective study reported in The Lancet Oncology, Lalchungnunga et al tested the classification accuracy of a molecular inference–based artificial intelligence (AI) model (Neuropath-AI) in central nervous system (CNS) tumor diagnosis.

A novel KRAS G12D inhibitor produced disease control in almost 80% of patients with heavily pretreated advanced or metastatic KRAS G12D–mutated pancreatic cancer in an early-phase study reported at the 2026 ASCO Gastrointestinal (GI) Cancers Symposium.¹

Short-term exposure to low-dose vaginal estrogen therapy may relieve some symptoms of menopause for younger survivors of endometrial cancer without increasing the risk of endometrial cancer recurrence, according to findings from a study published in Menopause.

A large national study in Denmark following nearly 1,900 patients over almost a decade found that minimally invasive ablation is as effective as surgery for treating small kidney cancers, with faster recovery and fewer complications. Results of the study were published by Ahrenfeldt et al in

Thursday, March 5, 2026 9:00 AM

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Social Cue Prompts Reduce Sharing of Cancer Treatment Misinformation

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Black Patients With AML Face Younger Onset and Significantly Worse Survival Outcomes

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CD123-Targeting ADC Shows Activity in AML and BPDCN

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Combined Tests Could Avoid Unnecessary Cystectomy, Study Finds

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Adjuvant Pembrolizumab Plus Belzutifan in Patients With RCC at Increased Risk of Recurrence

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Research Suggests AI Pathology Models May Take Unreliable 'Shortcuts' to Identify Cancer Biomarkers

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Colorectal Cancer: ACS Report Shows Increasing Incidence, Mortality Rates in Younger Adults

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Lisocabtagene Maraleucel in Relapsed or Refractory MZL

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Perioperative Enfortumab Vedotin Plus Pembrolizumab in Cisplatin-Ineligible Bladder Cancer

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Can ctDNA Negativity Predict Metastasis-Free Survival in Muscle-Invasive Bladder Cancer?

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Addition of Hormone Therapy to Postoperative Radiotherapy in Prostate Cancer

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Perioperative Enfortumab Vedotin Plus Pembrolizumab May Reduce Risk of Recurrence in Patients With Muscle-Invasive Bladder Cancer

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Microplastics Found in 90% of Prostate Cancer Samples

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Dietary Influence on Leukemia and Lymphoma Progression

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Fifteen-Year Results From SWOG S0016 Suggest Follicular Lymphoma May Be Curable

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AI Model May Predict Cancer Risk in Patients With Colitis-Associated Low-Grade Dysplasia

Right ventricle (RV) dysfunction is associated with poorer outcomes in heart failure with preserved ejection fraction (HFpEF). Females are more likely to have HFpEF but males have worse prognosis and resting RV function. The contribution of dynamic RV-pulmonary artery (RV-PA) coupling between sex and its impact on peak exercise capacity (VO2) in HFpEF is not known.

A Phase I, single-center investigation demonstrated that 8 weeks of antimycobacterial therapy improved sarcoidosis forced vital capacity (FVC). Safety and efficacy assessments have not been performed in a multicenter cohort.

All aspects of medical education were affected by the Novel Coronavirus Infectious Disease-19 (COVID-19) pandemic. Several challenges were experienced by trainees and programs alike due to the economic repercussions of the pandemic, how social distancing affected the delivery of medical education, testing and interviewing, how the surge of patients affected redeployment of personnel, potential compromise in core training and the overall impact on the wellness and mental health of trainees and educators.

In this meta-analysis of observational studies, RV dysfunction was associated with higher short-term and long-term mortality in sepsis and septic shock.

Legionella is an uncommon cause of CAP, occurring primarily from late spring through early autumn. Testing is uncommon, even among patients with risk factors, and many positive patients failed to receive empiric coverage for LP.

The nS classification could be used to provide a more accurate prognosis in patients with resected NSCLC. The nS is worth taking into consideration when defining nodal category in the forthcoming ninth edition of the staging system.

NLP successfully identified a large cohort with CC. Most patients were identified through NLP alone, rather than diagnoses or medications. NLP improved detection of patients nearly seven-fold, addressing the gap in ability to identify and characterize CC disease burden. Nearly all cases appeared to be managed in primary care. Identifying these patients is important for characterizing treatment and unmet needs.

Pulmonary arterial hypertension (PAH) is a rare disease and much of our understanding stems from single-center studies, which are limited by sample size and generalizability. Administrative data offer an appealing opportunity to inform clinical, research, and quality improvement efforts for PAH. Yet, there is currently no standardized, validated method to distinguish PAH from other subgroups of pulmonary hypertension (PH) within this data source.

A transparent DL algorithm improves on traditional clinical criteria to predict the need for MV in hospitalized patients, including in those with COVID-19. Such an algorithm may help clinicians optimize timing of tracheal intubation, better allocate resources and staff, and improve patient care.

To assess airway and lung parenchymal damage non-invasively in cystic fibrosis (CF), chest MRI has been historically out of the scope of routine clinical imaging due to technical difficulties such as the low proton density and respiratory and cardiac motion. However, technological breakthroughs have recently emerged to dramatically improve lung MRI quality (including signal-to-noise ratio, resolution, speed, contrast). At the same time, novel treatments have changed the landscape of CF clinical care.

Bronchoscopic lung volume reduction with one-way endobronchial valves is a guideline treatment option for patients with advanced emphysema, supported by extensive scientific data. Patients limited by severe hyperinflation, with a suitable emphysema treatment target lobe and with absence of collateral ventilation are the responders to this treatment. Detailed patient selection, a professional treatment performance, and dedicated follow-up of the valve treatment, including management of complications, are key ingredients to success.

Chronic Obstructive Pulmonary Disease (COPD) may cause profound dyspnea, functional impairment, and reduced quality of life. Available pharmacologic therapy provides suboptimal symptom improvement in many patients. Bronchoscopic lung volume reduction (BLVR), achieved with endobronchial valve (EBV) placement, can effectively improve dyspnea and functional status in appropriately selected patients. Operationalizing a safe and effective BLVR program requires appropriate oversight which can be achieved by a BLVR Nurse Coordinator (NC).

Empathy-based, stigma-reducing communication may lead to improved assessments of tobacco use and smoking cessation for patients with smoking-related cancers. These findings support the dissemination and further testing of a new ECS model for training OCPs in best practices for assessment of smoking history and engagement of patients who currently smoke in tobacco treatment delivery.

In clinical practice, reslizumab may have been initiated in response to heavy symptom burden and CAEs. Reslizumab was associated with improved clinical and patient-reported outcomes and significant reductions in asthma-related HRU.

Quantitative emphysema measured on LDCT of the chest can be leveraged to improve lung cancer risk prediction and help diagnose COPD in individuals who currently or formerly smoked undergoing lung cancer screening.

Although oncology advances stem from clinical research, <5% of global cancer trials actively recruit patients in Latin America. Herein, I discuss why expanding inclusive trial designs and fostering multisector partnerships are essential to reduce disparities and improve the role of Latin America in global oncology research. I present examples such as the recent regulatory reforms in Brazil that aim to accelerate study approvals, as well as collaborative initiatives between local and international research groups to further strengthen clinical trial capacity.

Lisocabtagene maraleucel has high efficacy in R/R MZL

Cholangiocarcinomas, comprising intrahepatic, extrahepatic and perihilar subtypes as well as gallbladder cancer, are a heterogeneous group of malignancies that necessitates a personalized approach to both surgery and systemic therapy. In this Review, the authors describe progress in the development of personalized perioperative therapy strategies as well as the most promising future research directions in this diverse and often difficult-to-treat group of cancers.

Enfortumab vedotin plus pembrolizumab effective in patients with resectable bladder cancer

Despite substantial interest in exploiting ferroptosis — an iron-mediated, lipid peroxidation-driven form of cell death — for cancer therapy, progress towards clinical translation has been limited. This comprehensive Review describes the major barriers that have restricted ferroptosis-based therapeutic development as well as emerging opportunities presented by new ferroptosis-inducing agents, biomarker-guided patient selection and rational combination treatments.

Effective early detection and intervention strategies for lung cancer are urgently needed. The authors of this Review summarize the current state of lung cancer screening and provide future directions for optimal implementation, including biomarker development. They also discuss precancer interception strategies that could transform both lung cancer prevention and early intervention.

Analysis of tumour tissue to guide the clinical management of patients with cancer poses several challenges. This Review outlines the potential of liquid biopsy-based analysis of circulating tumour DNA (ctDNA) to address these limitations and thereby improve precision medicine in the context of metastatic colorectal cancer, as well as opportunities to refine the management of locoregional disease using recurrence risk-tailored approaches predicated on ctDNA-based evaluation of minimal residual disease status.

Repotrectinib is a next-generation TKI for NTRK fusion-positive solid tumours

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Zealand Pharma’s shares fell 32% in early morning trading Friday after its Roche-partnered asset petrelintide failed to meet investor expectations in a mid-stage clinical trial.

In this episode of Denatured, you’ll be listening to Indu Navar, CEO and founder of EverythingALS and Dr. Olga Uspenskaya, chief medical officer at VectorY Therapeutics. We’ll be speaking about patient-pharma collaborations accelerating trials and hope, advances in ALS biology understanding and biomarker-driven endpoints.

A trial of a MacroGenics’ drug temporarily paused enrollment after several safety events, including a fatality. The deceased patient had developed a severe case of neutropenia and concurrent septic shock.

Following Monday’s clinical defeat by Eli Lilly, Novo Nordisk cut the 2027 list prices for its three GLP-1 medicines by as much as 50%, while boasting Phase 2 data for its invesigational triple-G agonist.

Disc Medicine’s leadership tried to express optimism that its rare disease therapy bitopertin can be approved based on a Phase 3 trial set to begin shortly. However, analysts are worried that the protocol was developed with former FDA leaders.

The percentage of patients achieving total clearance of eczema lesions in a Phase 2b trial increased with prolonged rezpeg treatment.

The newly public Evommune shared data showing that EVO301, an IL-18 targeting protein, cleared symptoms comparably to Regeneron and Sanofi’s mega-blockbuster in a mid-stage atopic dermatitis clinical trial.

Kailera will launch a global Phase 2 study of ribupatide this year, while Hengrui will push the asset into Phase 3 in China.

According to Priovant, the Phase 2 BEACON study is the first industry-sponsored placebo-controlled trial in cutaneous sarcoidosis to deliver positive data.

With data from a mid-stage trial showing weight loss of up to 12.3% at 28 weeks in patients treated with PF’3944, “Pfizer is moving full speed into obesity clinical development,” BMO Capital Markets said Tuesday.

The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later.

Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.

Aardvark Therapeutics is down 54% since Friday after the biotech said it detected “reversible cardiac observations” in a healthy volunteer study of its drug to treat extreme hunger in patients with the rare genetic disease.

A combination of Merck’s Keytruda and Pfizer’s Padcev could offer a chemotherapy-free treatment alternative for patients with muscle-invasive bladder cancer, even those eligible for cisplatin treatment.

Novo Nordisk reported a loss in a head-to-head trial of CagriSema against Lilly’s Zepbound earlier this week. This time around, Lilly’s orforglipron bested Novo’s oral semaglutide in blood sugar control and weight reduction—albeit with a few extra discontinuations as compared to its rival.

At the 2026 Conference on Retroviruses and Opportunistic Infections, Gilead and Merck demonstrated that their respective daily oral HIV drugs can match current therapies in keeping the virus at bay.

Following a disappointing Phase 3 performance and given Gossamer Bio’s balance sheet, seralutinib’s path to the market for pulmonary arterial hypertension has become unclear, according to analysts at Guggenheim Partners.

Evidence of durability of psilocybin-based COMP360 is a key point for the FDA, according to Compass Pathways Chief Medical Officer Guy Goodwin. By providing 26 weeks’ worth of such data instead of the requested 12, the company is delivering “in spades,” he said.

Following the successful late-stage study in wet age-related macular degeneration, Ocular plans to meet with the FDA to determine a regulatory path for Axpaxli.

Infigratinib topped “even the most optimistic expectations” for efficacy and safety in the late-stage PROPEL 3 study in achondroplasia, Truist Securities analysts said Thursday.

Zealand Pharma’s shares fell 32% in early morning trading Friday after its Roche-partnered asset petrelintide failed to meet investor expectations in a mid-stage clinical trial.

In this episode of Denatured, you’ll listen to Ram May-Ron, managing partner at FreeMind Group, and Ravi Kiron, managing director at Biopharma Strategy Advisors. We’ll be speaking about how to combine nondilutive funding and family office money into a unified strategy that gets companies through the drug development valley of death.

With fresh billions unlocked in the 2026 U.S. budget and mission‑driven family offices recalibrating after a “nuclear winter,” early stage biotechs are rewriting their financing strategies around nondilutive capital and targeted private wealth.

This webinar explores how collaborative-initiated trials and Investigator-Initiated Trials (IITs) are complementing traditional drug development to accelerate innovation, advance precision medicine, and address clinically meaningful questions.

The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later.

Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.

Aardvark Therapeutics is down 54% since Friday after the biotech said it detected “reversible cardiac observations” in a healthy volunteer study of its drug to treat extreme hunger in patients with the rare genetic disease.

A combination of Merck’s Keytruda and Pfizer’s Padcev could offer a chemotherapy-free treatment alternative for patients with muscle-invasive bladder cancer, even those eligible for cisplatin treatment.

In this episode of Denatured, you’ll be listening to Indu Navar, CEO and founder of EverythingALS and Dr. Olga Uspenskaya, chief medical officer at VectorY Therapeutics. We’ll be speaking about patient-pharma collaborations accelerating trials and hope, advances in ALS biology understanding and biomarker-driven endpoints.

Novo Nordisk reported a loss in a head-to-head trial of CagriSema against Lilly’s Zepbound earlier this week. This time around, Lilly’s orforglipron bested Novo’s oral semaglutide in blood sugar control and weight reduction—albeit with a few extra discontinuations as compared to its rival.

On March 5, 2026, the Food and Drug Administration approved teclistamab (Tecvayli, Janssen Biotech, Inc.) in combination with daratumumab hyaluronidase-fihj for adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor and an immunomodulatory agent. 

On February 26, 2026, the Food and Drug Administration granted accelerated approval to zongertinib (Hernexeos, Boehringer Ingelheim Pharmaceuticals, Inc.), a kinase inhibitor, for an expanded indication for adults with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-authorized test.

On February 24, 2026, the Food and Drug Administration granted traditional approval to encorafenib (Braftovi, Array BioPharma Inc., a subsidiary of Pfizer Inc.) in combination with cetuximab and fluorouracil-based chemotherapy for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, as detected by an FDA-authorized test. Encorafenib received accelerated approval in combination with cetuximab and mFOLFOX6 for metastatic colorectal cancer with BRAF V600E mutation in 2024. 

On February 19, 2026, the Food and Drug Administration approved acalabrutinib (Calquence, AstraZeneca) tablets and capsules in combination with venetoclax (Venclexta, AbbVie Inc. and Genentech Inc.) for adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

On February 10, 2026, the Food and Drug Administration approved pembrolizumab (Keytruda, Merck) as well as pembrolizumab and berahyaluronidase alfa-pmph (Keytruda Qlex, Merck) in combination with paclitaxel, with or without bevacizumab, for adult patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal carcinoma whose tumors express PD-L1 (CPS≥1) as determined by an FDA-authorized test, and who have received one or two prior systemic treatment regimens.

On January 27, 2026, the Food and Drug Administration approved daratumumab and hyaluronidase-fihj (Darzalex Faspro, Janssen Biotech, Inc.) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for adults with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT).

On December 17, 2025, the Food and Drug Administration approved amivantamab and hyaluronidase-lpuj (Rybrevant Faspro, Janssen Biotech, Inc.) for subcutaneous injection for adult patients across all indications approved for the intravenous formulation of amivantamab (Rybrevant, Janssen Biotech, Inc.). See the prescribing information for the specific indications.

On December 17, 2025, the Food and Drug Administration approved rucaparib (Rubraca, pharmaand GmbH) for adults with a deleterious BRCA mutation (BRCAm) (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) previously treated with an androgen receptor-directed therapy. Patients should be selected for therapy using an FDA-approved companion diagnostic (CDx).

On December 15, 2025, the Food and Drug Administration approved fam-trastuzumab deruxtecan-nxki (Enhertu, Daiichi Sankyo, Inc.) in combination with pertuzumab for the first-line treatment of adults with unresectable or metastatic HER2-positive (IHC 3+ or ISH+) breast cancer as determined by an FDA-approved test.

On December 12, 2025, the Food and Drug Administration approved niraparib and abiraterone acetate (Akeega, Janssen Biotech, Inc.) with prednisone for adults with deleterious or suspected deleterious BRCA2-mutated (BRCA2m) metastatic castration-sensitive prostate cancer (mCSPC), as determined by an FDA-approved test.

On December 4, 2025, the Food and Drug Administration approved lisocabtagene maraleucel (Breyanzi, Juno Therapeutics, Inc., a Bristol-Myers Squibb Company) for adults with relapsed or refractory marginal zone lymphoma (MZL) who have received at least two prior lines of systemic therapy

On December 3, 2025, the Food and Drug Administration granted traditional approval to pirtobrutinib (Jaypirca, Eli Lilly and Company) for adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have previously been treated with a covalent BTK inhibitor. In 2023, FDA granted accelerated approval to pirtobrutinib for adults with CLL/SLL who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor.

On November 25, 2025, the Food and Drug Administration approved durvalumab (Imfinzi, AstraZeneca) with fluorouracil, leucovorin, oxaliplatin, and docetaxel (FLOT) chemotherapy as neoadjuvant and adjuvant treatment, followed by single agent durvalumab, for adults with resectable gastric or gastroesophageal junction adenocarcinoma (GC/GEJC).

On November 21, 2025, the Food and Drug Administration approved pembrolizumab (Keytruda, Merck) or pembrolizumab and berahyaluronidase alfa-pmph (Keytruda Qlex, Merck) with enfortumab vedotin-ejfv (Padcev, Astellas Pharma) as neoadjuvant treatment followed by adjuvant treatment after cystectomy for adults with muscle invasive bladder cancer (MIBC) who are ineligible for cisplatin.

On November 19, 2025, the Food and Drug Administration granted traditional approval to tarlatamab-dlle (Imdelltra, Amgen Inc.) for adults with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy. Tarlatamab-dlle received accelerated approval for this indication in 2024.

On November 19, 2025, the Food and Drug Administration granted accelerated approval to sevabertinib (Hyrnuo, Bayer HealthCare Pharmaceuticals Inc.), a kinase inhibitor, for adults with locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy.

On November 19, 2025, the Food and Drug Administration granted traditional approval to daratumumab and hyaluronidase-fihj (Darzalex Faspro, Janssen Biotech Inc.) with bortezomib, cyclophosphamide, and dexamethasone (VCd) for newly diagnosed light chain (AL) amyloidosis. FDA granted accelerated approval for this indication in 2021.

On November 19, 2025, the Food and Drug Administration approved selumetinib (KOSELUGO, AstraZeneca Pharmaceuticals LP) for adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). FDA previously approved selumetinib capsules and granules for pediatric patients 1 year of age and older for this indication.

On November 18, 2025, the Food and Drug Administration approved epcoritamab-bysp (Epkinly, Genmab US, Inc.) with lenalidomide and rituximab for relapsed or refractory follicular lymphoma (FL). The FDA also granted traditional approval to epcoritamab-bysp as monotherapy for relapsed or refractory FL after two or more lines of systemic therapy (epcoritamab-bysp was granted accelerated approval for this indication in 2024). 

On November 13, 2025, the Food and Drug Administration approved Poherdy (pertuzumab-dpzb, Shanghai Henlius Biologics Co. Ltd.) as an interchangeable biosimilar to Perjeta (pertuzumab, Genentech Inc.). This is the first approval of a biosimilar for Perjeta.

On November 13, 2025, the Food and Drug Administration approved ziftomenib (Komzifti, Kura Oncology, Inc.), a menin inhibitor, for adults with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options.

On November 6, 2025, the Food and Drug Administration approved daratumumab and hyaluronidase-fihj (Darzalex Faspro, Janssen Biotech, Inc.) for adults with high-risk smoldering multiple myeloma (SMM).

On October 24, 2025, the Food and Drug Administration approved revumenib (Revuforj, Syndax Pharmaceuticals, Inc.), a menin inhibitor, for relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options.

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