OBJECTIVES: We assess prescription patterns and describe the long-term real-life effectiveness of different TNF inhibitors (TNFis) in juvenile idiopathic arthritis-related uveitis (JIA-U). METHODS: Patients with JIA-U tr...OBJECTIVES: We assess prescription patterns and describe the long-term real-life effectiveness of different TNF inhibitors (TNFis) in juvenile idiopathic arthritis-related uveitis (JIA-U). METHODS: Patients with JIA-U treated with TNFi were retrospectively enrolled. RESULTS: Ninety-six JIA-U patients (77% female) with an age at diagnosis of 2.44 [interquartile range (IQR) 1.56-3.81] and a median follow-up of 19 years were included. Adalimumab was the most frequently prescribed TNFi (61%), followed by etanercept (19%), infliximab (15%), and golimumab (5%). Overall, adalimumab showed the lowest complication rate (51%; < 0.001) and median number of uveitis relapse (1; IQR 0-2; = 0.012). Conversely, etanercept showed the highest median number of uveitis relapses (4; IQR 1-5). The cumulative incidence curves for 'uveitis relapse' were similar among TNFi. Conversely, when 'treatment change' was considered as the event, a significantly higher risk for patients treated with infliximab compared with those receiving adalimumab emerged (HR 3.06, 95% CI 1.41-6.63; < 0.01). CONCLUSIONS: All TNFis appear to be effective for long-term management of JIA-U. We observed some differences in the number of uveitis relapses and ocular complication rates favoring adalimumab over infliximab and etanercept, findings that need to be further confirmed in prospective studies.
INTRODUCTION: Over the past two decades, targeted biological therapies have profoundly transformed the management of type 2 (T2) inflammatory diseases, including severe asthma, atopic dermatitis, chronic rhinosinusitis w...INTRODUCTION: Over the past two decades, targeted biological therapies have profoundly transformed the management of type 2 (T2) inflammatory diseases, including severe asthma, atopic dermatitis, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, chronic spontaneous urticaria, and eosinophilic granulomatosis with polyangiitis. This paper reviews the key milestones of this twenty-year journey, focusing on the evolving therapeutic goals, from disease control to clinical remission. AREAS COVERED: This paper synthesizes evidence from pivotal clinical trials, real-world registries, extension studies, and systematic reviews on biologics in T2 inflammatory diseases. A targeted literature search was conducted across PubMed and major respiratory, allergy, and dermatology journals, prioritizing publications from 2020 to 2026, with inclusion of landmark earlier studies. EXPERT OPINION: Clinical remission in biologic-treated T2 inflammatory diseases is achievable but not yet reliably predictable; the field urgently requires globally standardized, validated composite remission indices for each major T2 condition that integrate symptom scores, organ function measures, and biomarker profiles. The defining challenge of the next decade is determining whether early biologic intervention can genuinely modify the disease trajectory and achieve sustained off-treatment remission.
INTRODUCTION: Adult secondary hemophagocytic lymphohistiocytosis (asHLH) is a fulminant hyperinflammatory condition triggered by cancers, infections, autoimmune conditions or drugs. The hallmark of its pathogenesis invol...INTRODUCTION: Adult secondary hemophagocytic lymphohistiocytosis (asHLH) is a fulminant hyperinflammatory condition triggered by cancers, infections, autoimmune conditions or drugs. The hallmark of its pathogenesis involves dysregulated immune effector cell activation and excessive cytokine release. AREAS COVERED: This narrative review updates the reader on modern treatment options for asHLH. The traditional one-size-fits-all strategy derived from the pediatric HLH-2004 protocol is increasingly viewed as outdated today. A significant shift toward a more individualized, trigger- directed treatment approach has occurred in the HLH literature of recent years. EXPERT OPINION: Apart from treating promptly the underlying trigger conditions, earlier incorporation of targeted anti-cytokine therapy such as anakinra is strongly favored these days. The cytotoxic etoposide shall be chiefly reserved for malignancy-associated HLH, including B-cell lymphomas, and refractory disease. Rituximab has led to superior outcomes when utilized to clear EBV-infected B-cell reservoirs. IVIG should be considered in infection-associated HLH. The anti-IFN-γ monoclonal antibody emapalumab has been approved for refractory HLH cases. The JAK 1/2 inhibitor ruxolitinib may prove useful in this space as well. The therapeutic arsenal is likely to evolve further in the direction of agents addressing specific pathophysiologic steps in asHLH. Targeting effector cytokines, their receptors, inflammasome pathways are promising future directions in asHLH therapeutics.
INTRODUCTION: Childhood-onset systemic lupus erythematosus (cSLE) accounts for 15-20% of all forms of SLE. It is associated with greater disease severity and requires more aggressive treatment than adult-onset SLE. Despi...INTRODUCTION: Childhood-onset systemic lupus erythematosus (cSLE) accounts for 15-20% of all forms of SLE. It is associated with greater disease severity and requires more aggressive treatment than adult-onset SLE. Despite therapeutic advances, many patients do not achieve sustained remission, highlighting the need for more effective treatment strategies. AREAS COVERED: This review summarizes the literature published in the past two decades on the use of biologic medications for the treatment of cSLE. It is based on the narrative analysis of recent clinical trials, observational studies and patient series or individual clinical cases. We discuss the rationale, indications, effectiveness and safety of the biologic agents utilized thus far in patients with cSLE, with a primary focus on rituximab and belimumab. In addition, we address the future perspectives of the management of cSLE. EXPERT OPINION: The improved understanding of the mechanisms involved in immunopathogenesis of SLE has led to the development of increasing numbers of biologic agents that target specific cells or pathways. This advance has increased the therapeutic options available for the management of cSLE and holds great promise for transforming the landscape of cSLE care through the implementation of precision medicine and personalized therapeutic approaches, with the ultimate goal of reaching sustained, glucocorticoid-free disease remission.
Giovannini M, de Las Vecillas L, De Filippo M
… +12 more, Castagnoli R, Votto M, Klain A, Dinardo G, Trincianti C, Marseglia GL, Indolfi C, Del Giudice MM, Tosca MA, Ciprandi G, Mori F, Licari A
INTRODUCTION: Atopic dermatitis (AD) is a chronic inflammatory skin disease that frequently affects children and is associated with significant morbidity and impaired quality of life. Recent advances in our understanding...INTRODUCTION: Atopic dermatitis (AD) is a chronic inflammatory skin disease that frequently affects children and is associated with significant morbidity and impaired quality of life. Recent advances in our understanding of AD immunopathogenesis have led to the development of targeted biological and small-molecule therapies. AREAS COVERED: This review focuses on the available and emerged targeted therapies for pediatric AD, linking mechanistic rationale to currently available and emerging biologics and JAK inhibitors and highlighting the limits of pediatric evidence, real-world data, and treatment selection. The literature was reviewed by searching PubMed and other relevant clinical trial databases, focusing on studies that evaluated targeted systemic treatments in children and adolescents with AD. EXPERT OPINION: Targeted therapies are reshaping the management of pediatric AD and improving disease control and quality of life. However, long-term safety data, validated biomarker-guided treatment strategies, and equitable access to these therapies remain key challenges.
INTRODUCTION: Biologic therapies have transformed the management of moderate-to-severe psoriasis; however, long-term treatment requires optimization strategies to maintain efficacy, minimize adverse effects, and improve...INTRODUCTION: Biologic therapies have transformed the management of moderate-to-severe psoriasis; however, long-term treatment requires optimization strategies to maintain efficacy, minimize adverse effects, and improve cost-effectiveness in real-world practice. AREAS COVERED: This narrative review summarizes current evidence on biologic optimization in psoriasis, including dose adjustment strategies (dose reduction and escalation), interval modification, switching between biologics, and treatment tapering. The role of therapeutic drug monitoring and immunogenicity in guiding treatment decisions is also discussed, along with emerging approaches such as combination biologic therapy. In addition, evolving biomarkers and computational tools, like machine learning, may enable the prediction of treatment response and guide personalized therapy. A structured literature review of published clinical trials, real-world studies, and review articles was undertaken to evaluate existing evidence and identify gaps in current practice. EXPERT OPINION: Biologic optimization should follow an individualized, mechanism-based approach integrating clinical response, pharmacokinetic variability, and patient-specific factors. While current evidence is heterogeneous, future research focusing on validated biomarkers, therapeutic drug monitoring, and artificial intelligence-driven models will be critical in advancing precision medicine and improving the optimization, durability and cost-effectiveness of biologic therapy.
BACKGROUND: Psoriasis is an immune-mediated inflammatory skin disease with variable biologic therapy response. Current treatment selection lacks reliable predictive biomarkers for personalized decisions. OBJECTIVES: To i...BACKGROUND: Psoriasis is an immune-mediated inflammatory skin disease with variable biologic therapy response. Current treatment selection lacks reliable predictive biomarkers for personalized decisions. OBJECTIVES: To identify distinct immunological patient subgroups based on peripheral blood immune cells and evaluate their association with biologic therapy efficacy. METHODS: This retrospective cohort study included 329 plaque psoriasis patients initiating first-time biologic therapy and 169 healthy controls. Peripheral blood flow cytometry data were analyzed using hierarchical clustering. Kaplan-Meier analysis assessed time to PASI90 achievement among subgroups. RESULTS: Psoriasis patients showed significantly elevated lymphocyte populations. Three distinct immunological clusters were identified: Cluster 1 (57.8%) with moderate T cells and low NK cells; Cluster 2 (27.7%) with decreased T cells and increased NK cells; and Cluster 3 (14.6%) with increased total lymphocytes. Cluster 1 patients achieved PASI90 significantly faster with both IL-17 and IL-23 inhibitors ( < 0.001). CONCLUSION: Peripheral blood flow cytometry identified distinct psoriasis immunophenotypes with significant differences in biologic therapy response, offering potential biomarkers for treatment selection and personalized medicine approaches.
INTRODUCTION: Biosimilar development is undergoing regulatory change, with growing emphasis on analytical comparability and targeted clinical pharmacology rather than routine comparative efficacy studies. A focused revie...INTRODUCTION: Biosimilar development is undergoing regulatory change, with growing emphasis on analytical comparability and targeted clinical pharmacology rather than routine comparative efficacy studies. A focused review is therefore needed to understand how global regulatory expectations are evolving, where they are converging, and what this means for future development. AREAS COVERED: This review examines biosimilar regulatory frameworks across agencies, including the FDA, EMA, PMDA, WHO, Health Canada, and ANVISA. It outlines the shift toward a stepwise, totality-of-evidence approach in which analytical similarity, functional characterization, pharmacokinetic/pharmacodynamic comparability, and immunogenicity assessment provide the basis for establishing biosimilarity. It also considers recent regulatory changes supporting a reduced role for comparative efficacy studies, broader use of foreign comparators and reliance pathways, and the emerging application of artificial intelligence in comparability assessment and model-informed development. Relevant regulatory documents and peer-reviewed literature were reviewed to summarize current trends, differences, and likely future directions. EXPERT OPINION: For many well-characterized biosimilars, particularly monoclonal antibodies, robust analytical and clinical pharmacology data can address most uncertainty, making routine comparative efficacy studies less necessary in many cases. Greater alignment in comparator policies, study expectations, and responsible use of artificial intelligence could help streamline development, reduce duplication, and improve patient access.
BACKGROUND: Clinical remission is an emerging goal in severe asthma (SA) management. While tezepelumab demonstrates broad efficacy, its real-world impact on remission-specifically in the context of small airway dysfuncti...BACKGROUND: Clinical remission is an emerging goal in severe asthma (SA) management. While tezepelumab demonstrates broad efficacy, its real-world impact on remission-specifically in the context of small airway dysfunction (SAD)-remains incompletely defined. METHODS: This prospective, multicentre study enrolled 39 adults with SA treated with tezepelumab for 12 months. Clinical remission was defined by SANI criteria: complete oral corticosteroid (OCS) withdrawal, zero severe exacesrbations, Asthma Control Test score ≥20, and stable/improved FEV. SAD remission required normalization of oscillometric parameters. RESULTS: At baseline, 44% of patients exhibited SAD. Tezepelumab eliminated severe exacerbations and achieved complete OCS withdrawal in 100% of evaluable patients. At 12 months, 77.4% of the overall evaluable cohort and 80.0% of those with baseline SAD achieved clinical remission. However, SAD remission was not observed; these patients maintained persistent oscillometric abnormalities despite reaching all clinical goals. CONCLUSION: Tezepelumab enables high rates of clinical remission regardless of baseline small airway status. In the SAD phenotype, symptomatic and inflammatory recovery dissociates from physiological SAD remission. Persistent mechanical abnormalities highlight the necessity of oscillometry to identify hidden residual disease in patients who otherwise appear to be in remission.
INTRODUCTION: The concept of difficult-to-treat (D2T)/difficult-to-manage (DTM) disease was first defined in rheumatoid arthritis (RA). Recent definitions for axial spondyloarthritis (axSpA) and psoriatic arthritis (PsA)...INTRODUCTION: The concept of difficult-to-treat (D2T)/difficult-to-manage (DTM) disease was first defined in rheumatoid arthritis (RA). Recent definitions for axial spondyloarthritis (axSpA) and psoriatic arthritis (PsA) combine non-response to treatment including targeted and biologic treatment, inflammatory disease activity, and patient symptoms. This concept could apply to other chronic inflammatory diseases such as psoriasis (PsO) and inflammatory bowel disease (IBD). A consensus definition of D2T IBD is available but is yet to be fully articulated in PsO. AREAS COVERED: This article aims to provide an expert overview of the shared elements and potential differences between the definitions and concepts of D2T/D2M in these diseases. EXPERT OPINION: D2T/D2M definitions should allow better evaluation of refractory diseases, accurate determination of disease severity, and effective treatment of the D2T areas or domains. Definitions should consider response to previous lines of treatment, control of inflammation, and global disease improvement, and consider the impact of D2T disease on patient status/quality of life. Proof-of-concept studies need to assess the current and future definitions of D2T/D2M populations in axSpA, PsA, PsO, and IBD, to accurately determine the prevalence of patients meeting each of those D2T criteria sets, and to identify risk factors, disease burden, and appropriate management strategies.
INTRODUCTION: Refractory myasthenia gravis (RMG) affects 10-20% of patients with generalized MG (gMG) and is associated with persistent disability, recurrent myasthenic crises, and elevated healthcare costs despite multi...INTRODUCTION: Refractory myasthenia gravis (RMG) affects 10-20% of patients with generalized MG (gMG) and is associated with persistent disability, recurrent myasthenic crises, and elevated healthcare costs despite multiple lines of immunosuppressive therapy. Efgartigimod (EFG), an engineered IgG1 Fc fragment that competitively inhibits the neonatal Fc receptor (FcRn), reduces pathogenic IgG and is approved for gMG in more than 30 countries worldwide. AREAS COVERED: This review synthesizes evidence on EFG in RMG from a literature search of PubMed and Embase (inception to March 2026), covering international refractory definitions, the ADAPT trial, multicenter real-world cohorts, rescue therapy in myasthenic crisis, treatment switching after biologic failure, and safety. EXPERT OPINION: EFG provides consistent symptom relief and substantial corticosteroid-sparing benefits across diverse RMG subtypes, including seronegative patients and those unresponsive to C5 complement inhibitors or B-cell depleting agents. EFG also serves as an effective rapid rescue therapy for refractory myasthenic crises. Despite reducing circulating IgG by approximately 60%, EFG does not increase severe infection rates. Therapeutic limitations include autoantibody rebound and inefficacy against non-IgG pathogenic autoantibodies. Further prospective trials utilizing modernized RMG definitions and validated predictive biomarkers are essential to establish EFG's therapeutic role in this challenging population.
Provencio M, Aguiar D, Antoñanzas M
… +27 more, Areses MC, Azkárate A, Barba A, Bernabé R, Blanco R, Borregón M, Campos B, Coves J, de Castro J, Domènech M, Dómine M, García Campelo R, Lucía-Gozálvez C, Lázaro-Quintela M, Insa A, Juan-Vidal Ó, López Castro R, Martínez-Marti A, Medina S, Moreno MÁ, Nadal E, Olivares-Hernández A, Reguart N, Sala MÁ, Medina K, Vila L, Massutí B
Lung cancer remains the leading cause of cancer-related deaths worldwide. Despite advances to improve treatment in resectable early-stage and locally advanced non-small cell lung cancer (NSCLC), survival rates after 5 ye...Lung cancer remains the leading cause of cancer-related deaths worldwide. Despite advances to improve treatment in resectable early-stage and locally advanced non-small cell lung cancer (NSCLC), survival rates after 5 years remain low, with a high risk of disease progression. Patients who fail to achieve a pathological complete response (pCR) after neoadjuvant treatment have unfavorable outcomes. ARIAN is a phase III, multicenter, open-label, randomized trial evaluating adjuvant treatment in adults with stage IB-IIIB (N2) NSCLC who have undergone neoadjuvant chemo-immunotherapy but did not achieve pCR after completing surgical resection. Patients will be stratified based on programmed death receptor-ligand 1 (PD-L1) tumor expression and randomized (1:1:1) to observation for 10 months (Arm 1), zimberelimab for 13 cycles (Arm 2), or sacituzumab govitecan and zimberelimab for 8 cycles followed by zimberelimab monotherapy for 5 cycles (Arm 3). The primary endpoint is disease-free survival, and secondary endpoints include overall survival and safety.
BACKGROUND: Phase-III INSIGHT study subgroup analyses observed best corrected visual acuity (BCVA) and central subfield thickness (CST) outcomes at Week 8/52 in diabetic macular edema treated with aflibercept biosimilar...BACKGROUND: Phase-III INSIGHT study subgroup analyses observed best corrected visual acuity (BCVA) and central subfield thickness (CST) outcomes at Week 8/52 in diabetic macular edema treated with aflibercept biosimilar (MYL-1701P/Yesafili™) or reference aflibercept (Eylea®). RESEARCH DESIGN AND METHODS: Two mg (0.05 mL) MYL-1701P ( = 179) or reference aflibercept ( = 176) was given intravitreally every 4 weeks for 5 doses, followed by 8-weekly dosing through Week 48. Subgroups were stratified by baseline BCVA/baseline CST/age/gender/race/ethnicity/region/glycated hemoglobin (HbA1c)/anti-drug antibody status/anti-vascular endothelial growth factor therapy in fellow eye. Main outcome measures included mean change in BCVA/CST from baseline to Week 8/52 with 90% confidence interval (CI). RESULTS: For MYL-1701P and reference aflibercept, participants with baseline BCVA score (73-55 letters) had an adjusted mean difference of 0.03 letters in BCVA (90% CI: -1.26,1.31) and 15.46 µm in CST (90% CI: -0.02,30.93) at 8 weeks and 0.81 letters in BCVA (90% CI: -0.58,2.2) and 6.41 µm in CST (90% CI: 17.31,30.12) at 52 weeks. Subgroup categories with ≥45% participants, including CST (<400/≥400 µm) and HbA1c (<8%/>8%) had an adjusted mean difference within -3 to 3 letters (90% CI) in BCVA at 8/52 weeks. CONCLUSIONS: The exploratory subgroup analyses supported clinical equivalence between MYL-1701P and reference aflibercept showing clinically comparable changes in BCVA/CST across most subgroups. TRIAL REGISTRATION: ClinicalTrials.gov identifier is NCT03610646.
INTRODUCTION: Melanomas cause the vast majority of skin-cancer related deaths, despite accounting for only around 2% of skin cancer cases. Moreover, due to genetic and environmental factors, the melanomas that predominat...INTRODUCTION: Melanomas cause the vast majority of skin-cancer related deaths, despite accounting for only around 2% of skin cancer cases. Moreover, due to genetic and environmental factors, the melanomas that predominate in East Asian populations, generally acral and mucosal melanomas, are extremely rare in Caucasian populations. These melanomas differ from other cutaneous melanomas in clinicopathological features and mutational landscapes, including a lower prevalence of commonly targetable mutations such as BRAF V600, and are usually diagnosed at a more advanced stage with a poor prognosis. The advent of immunotherapies therefore represents a great stride forward in the treatment of melanoma, including in East Asian countries, with immune checkpoint blockade now available as first-line treatment in China. AREAS COVERED: This review briefly outlines the epidemiology and unique clinical characteristics of melanoma in East Asian patients; summarizes existing immunotherapy approvals, treatment landscape, and unmet needs; and aims to enable clinicians to make more informed treatment decisions for patients. EXPERT OPINION: Further research is urgently needed into novel immunotherapeutic combinations, and biomarkers of response, to fully unlock the potential of immunotherapy for East Asian AM and MM and guide future therapeutic strategies.
INTRODUCTION: Advanced non-squamous non-small cell lung cancer (NSCLC) remains associated with substantial mortality despite major advances in targeted therapy and immuno-oncology. c-Met protein overexpression represents...INTRODUCTION: Advanced non-squamous non-small cell lung cancer (NSCLC) remains associated with substantial mortality despite major advances in targeted therapy and immuno-oncology. c-Met protein overexpression represents a biologically relevant and relatively prevalent phenotype that may define a therapeutically vulnerable population lacking canonical genomic drivers. AREAS COVERED: This review examines the scientific rationale for targeting c-Met protein overexpression and critically evaluates telisotuzumab vedotin (Teliso-V), a c-Met-directed antibody-drug conjugate (ADC) delivering the cytotoxic microtubule polymerization inhibitor MMAE. The structure, mechanism of action, dose optimization strategy, and exposure-toxicity relationships are discussed alongside emerging efficacy data from early-phase studies and the phase II LUMINOSITY trial. The evolving role of biomarker-driven ADC therapy in previously treated EGFR-wildtype non-squamous NSCLC, companion diagnostics, and regulatory considerations are also addressed. EXPERT OPINION: Teliso-V represents an important extension of the ADC paradigm into a protein-expression-defined NSCLC population, demonstrating clinically meaningful activity with a predictable and manageable safety profile dominated by cumulative risk for peripheral neuropathy. While accelerated approval underscores its therapeutic promise, long-term positioning will depend on confirmatory trials, refinement of biomarker testing, and optimization of patient selection. If validated, this strategy may redefine later-line treatment expectations by aligning cytotoxic payload delivery with biologically enriched disease subsets.
INTRODUCTION: Hereditary angioedema (HAE) is a rare, potentially life-threatening, unpredictable disease characterized by recurrent subcutaneous and/or submucosal edema (HAE attacks). HAE imposes a significant biopsychos...INTRODUCTION: Hereditary angioedema (HAE) is a rare, potentially life-threatening, unpredictable disease characterized by recurrent subcutaneous and/or submucosal edema (HAE attacks). HAE imposes a significant biopsychosocial burden on patients and their families owing to the erratic nature and variable severity of HAE attacks. Current guidelines appoint sustained disease control as one of the main treatment goals, achievable through the initiation of long-term prophylaxis (LTP). AREAS COVERED: This review focuses on the evaluation of three monoclonal antibodies developed for LTP of HAE, namely lanadelumab (Takhzyro®), garadacimab (Andembry®), and navenibart, in this order. Lanadelumab inhibits plasma kallikrein and has been an approved LTP option since 2018. Garadacimab inhibits activated factor XII (FXIIa) and has been approved for LTP in 2025. Navenibart, a drug currently under development, inhibits plasma kallikrein and has been modified to extend its circulating half-life. EXPERT OPINION: The transition from older non-targeted LTP options to the use of monoclonal antibody-based therapies has fundamentally changed the treatment landscape by offering a safe, effective, and highly targeted solution. Although having different pharmacological characteristics, these LTP options share the same objective: to achieve complete disease control.
BACKGROUND: This study aimed to show equivalent efficacy, pharmacodynamics, pharmacokinetics (PK), safety, and immunogenicity of biosimilar BAT2506 to reference golimumab (Ref-GLM) in patients with active psoriatic arthr...BACKGROUND: This study aimed to show equivalent efficacy, pharmacodynamics, pharmacokinetics (PK), safety, and immunogenicity of biosimilar BAT2506 to reference golimumab (Ref-GLM) in patients with active psoriatic arthritis. RESEARCH DESIGN AND METHODS: This study comprised a 24-week initial treatment period 1. Patients were randomly assigned (1:2:1) into three groups: Ref-GLM, BAT2506, or start with Ref-GLM and switch to BAT2506 at week 24. The assessment of therapeutic equivalence between BAT2506 and Ref-GLM via percentage of patients achieving an American College of Rheumatology 20 (ACR 20) response at Week 14 was the primary endpoint. RESULTS: 351 patients were randomized into the BAT2506 group and 353 patients in those groups that started the treatment with Ref-GLM. The 90% confidence interval (CI; -2.91, 7.63) and 95% CI (-3.92, 8.64) for the common risk difference in ACR 20 response between the BAT2506 group and groups that started the treatment with Ref-GLM fully fell within predefined equivalence margins supporting efficacy equivalence between BAT2506 and Ref-GLM at Week 14. Comparable safety, PK, and immunogenicity profiles were observed for BAT2506 and Ref-GLM groups. CONCLUSIONS: Data supported equivalence efficacy and comparable pharmacodynamics, PK, safety, and immunogenicity profiles between BAT2506 and Ref-GLM till Week 24. CLINICAL TRIAL REGISTRATION: www.clinicaltrials.gov identifier NCT05046431.
INTRODUCTION: Chronic graft-versus-host disease remains a major cause of late morbidity and mortality after allogeneic hematopoietic stem cell transplantation. Conventional prevention and management strategies, heavily r...INTRODUCTION: Chronic graft-versus-host disease remains a major cause of late morbidity and mortality after allogeneic hematopoietic stem cell transplantation. Conventional prevention and management strategies, heavily reliant on prolonged immunosuppression, are limited by suboptimal efficacy, substantial toxicity, and a failure to address disease heterogeneity. AREAS COVERED: This review synthesizes recent advances in the biological understanding of cGVHD, particularly the immune dysregulation and fibrotic progression that underpin its diverse clinical manifestations. We discuss a paradigm shift from uniform immunosuppression toward a structured framework encompassing four sequential phases: comprehensive assessment, risk-adapted prevention, mechanism-driven treatment, and holistic long-term survivorship care. Key innovations highlighted include the use of predictive biomarkers for early intervention, the use of steroid-sparing targeted therapies (such as JAK/ROCK2 inhibitors), and the integration of patient-reported outcomes and functional measures into routine evaluation. EXPERT OPINION: The field is poised for transformative change. We advocate for the routine implementation of risk-stratified prevention, preemptive antifibrotic strategies, and organ-specific multidisciplinary management. Crucially, therapeutic success should be redefined around patient-centered goals: safe steroid discontinuation, symptom control, functional recovery, and preserved quality of life. Embedding lifestyle support and longitudinal follow-up into standard care is essential to reduce the overall burden of cGVHD and improve long-term survival.