Expert Opinion on Biological Therapy最新文献

Objective: To evaluate indications, clinical response, outcomes, and tolerability of intravenous immunoglobulin (IVIG) therapy in patients with juvenile dermatomyositis (JDM) in a multicenter cohort.

Methods: This retrospective multicenter study included 29 patients with JDM who received IVIG. Demographic data, clinical features, laboratory parameters, and standardized disease activity scores were recorded at disease onset, before and after IVIG treatment, and at final follow-up. Treatment response and adverse events were analyzed.

Results: IVIG was initiated a median of 1.2 months (IQR: 0.1-18.9) after diagnosis and continued for a median of 8.2 months (IQR: 5.6-21.6). Indications included refractory muscle weakness, persistent skin disease, calcinosis, and interstitial lung disease (ILD). IVIG therapy was associated with significant improvements in disease activity measures and laboratory parameters (p < 0.001). Calcinosis partially or completely resolved in 3 of 6 patients, while no change was observed in the single ILD case. IVIG was well tolerated, with only mild adverse effects.

Conclusion: IVIG appears to be a safe and effective treatment option for JDM, particularly in refractory or skin-predominant disease. Although causal inferences are limited by the retrospective design and the absence of a control group, these real-world findings support the inclusion of IVIG in an individualized treatment approach.

Introduction: The antibody therapeutic landscape has been dominated by monoclonal antibodies (mAbs) since the early 2000s, leading to polyclonal antibodies (pAbs) assuming a less prominent role in clinical use. However, the multiepitope targeting capability of pAbs has generated renewed interest over the last five years. Emerging technologies such as recombinant antibody pools are prompting a revisit of the distinction between traditional mAb and pAb therapies.

Areas covered: Using listed FDA-approved polyclonal therapies as a basis for our literature searches, we explore the applications of current pAb therapies and examine whether mAbs may eventually replace them. We also discuss the costs and cost-effectiveness of directly competing mAb and pAb therapies for a variety of clinical indications. Finally, we review emerging technologies, such as glyco-humanized and recombinant pAbs, covering their modes of action, manufacturing approaches, and current clinical trial statuses.

Expert opinion: The current clinical pAb therapy landscape is varied, evolving, and continues to be challenged by mAbs. For the pAb therapies explored in this review, multiple competing mAb alternatives are progressing toward clinical approval. Future hybrid technologies like recombinant pAbs may allow multiple epitope targeting while achieving enhanced safety profiles and batch-to-batch consistency; however, their cost-effectiveness remains uncertain.

Background: To evaluate early real-world clinical outcomes on the safety and efficacy of the ranibizumab biosimilar (Oceva, Sun Pharmaceuticals, India).

Research design and methods: A multicenter, retrospective, uncontrolled observational study evaluating data from 404 eyes that received a total of 742 intravitreal injections of the ranibizumab biosimilar (Oceva 0.5 mg) across four centers in India, administered between August 2024 and May 2025 in variable approved and off label indications. Of the total eyes, 288 were treatment-naïve naïve, while 116 eyes were previously treated.

Results: The mean (SD) follow-up was 14.39 ± 12.5 weeks. The mean BCVA improved significantly from 0.87 to 0.59 LogMAR (p < 0.0001; d = 0.61), and mean CFT reduced from 406.8 µm to 306.5 µm (p < 0.0001; d = 0.66). Naïve eyes showed greater improvements than previously treated ones. No serious ocular or systemic adverse events were observed.

Conclusions: The preliminary real-world data from this limited early series suggest that ranibizumab biosimilar (Oceva) appears to be efficacious and safe across the approved indications. However, long-term data with a larger population are needed to further strengthen the findings of this study.

Introduction: Small-cell lung cancer (SCLC) is a rapidly progressive form of cancer often expressing DLL3. Tarlatamab is a bispecific antibody which blocks DLL3-related downstream pathways and activates antitumor host immunity. It is currently authorized in the U.S.A. for the treatment of extensive SCLC.

Areas covered: This is a review evaluating the basic pharmacology and clinical data regarding tarlatamab. Most data come from SCLC; in the other two diseases, tarlatamab is in early clinical development.

Expert opinion: Tarlatamab is an effective therapy mainly in relapsed/progressed SCLC, and its assessment in limited SCLC alone or combined with other therapies is supported by the existing data.

Background: Biologic agents have significantly improved psoriasis treatment, but patient responses exhibit considerable heterogeneity, highlighting the urgent need for practical predictive biomarkers of therapeutic efficacy.

Research design and methods: This prospective cohort study enrolled 422 psoriasis patients and 150 healthy controls. Sixteen CPD parameters were measured using a hematology analyzer. We analyzed associations between baseline CPD and disease severity as well as inflammatory markers, assessed their predictive value for treatment response over 48 weeks of biologic therapy, and monitored early dynamic changes in CPD and their relationship with treatment response in 169 patients.

Results: All CPD parameters were significantly elevated in psoriasis patients compared to healthy controls (all p < 0.001). Baseline mean lymphocyte volume (MN-V-LY) demonstrated sustained negative correlations with PASI improvement rates from weeks 4 to 48 (ρ = -0.278 to -0.449, all p < 0.001). Early reduction in monocyte volume heterogeneity (SD-V-MO) was significantly associated with long-term efficacy (ρ = -0.355 to -0.546, all p < 0.001).

Conclusions: As simple, standardized hematological parameters, CPD show potential for clinical application in predicting biologic therapy response in psoriasis.

Introduction: Bispecific antibodies (BsAbs) are a novel class of immunotherapy agents that engage endogenous T cells to target and eradicate malignant B cells by simultaneously binding CD3 and CD20. BsAbs such as epcoritamab, glofitamab, and mosunetuzumab have demonstrated substantial efficacy across both aggressive and indolent B-cell non-Hodgkin lymphomas (B-NHL), with largely manageable safety profiles. Their off-the-shelf administration offers a practical alternative to CAR T-cell therapy, particularly for patients who are transplant-ineligible or lack access to cellular approaches.

Areas covered: This review provides a comprehensive overview of BsAbs in B-NHL. We detail pivotal studies in relapsed/refractory (R/R) DLBCL, FL, and MCL, highlighting single-agent outcomes and emerging combination strategies. Approaches incorporating BsAbs into cytotoxic backbones and antibody-drug conjugates, as well as chemotherapy-free regimens such as BsAbs combined with lenalidomide, are discussed. We also examine their potential to address poor-risk disease features and historically refractory subsets.

Expert opinion: BsAbs represent a major advance in B-NHL, offering high rates of deep remission with predictable safety and outpatient feasibility. As trial data continues to mature, BsAb-based regimens are poised to become foundational across multiple lines of therapy, reshaping expectations for patients with both aggressive and indolent lymphomas.

Introduction: Natural killer (NK) cells are innate immune effectors that can eliminate malignant cells without prior sensitization. By recognizing cellular stress signals and releasing inflammatory mediators, they contribute to immune surveillance and regulation. Their therapeutic potential lies in their ability to act across donor barriers with a reduced risk of graft-related complications; however, clinical translation remains challenging due to tumor immune evasion and limited persistence in suppressive environments.

Areas covered: This review summarizes the biological roles of NK cells in cancer immunity and examines recent therapeutic approaches that harness their cytotoxic and regulatory properties. We discuss barriers to clinical application, including immune suppression, antigen loss, and manufacturing limitations. In addition, we highlight emerging strategies, such as gene editing, rational combination therapies, and standardized clinical trial designs, aimed at improving therapeutic efficacy.

Expert opinion: NK cell-based therapies represent a promising avenue in cancer immunotherapy but require carefully designed solutions to overcome their inherent limitations. Advances in biomarker-guided patient selection, integration with existing treatment modalities, and international collaboration will be critical for translating NK cell biology into effective and durable clinical outcomes.

Introduction: Atopic dermatitis (AD) is a prevalent chronic inflammatory skin disorder and has a considerable impact on patients' quality of life. Dupilumab, the first biologic approved for moderate-to-severe AD, is a human monoclonal antibody targeting the interleukin (IL)-4 receptor α, thereby blocking both IL-4 and IL-13 signaling, key drivers of type 2 inflammation in AD. Its introduction has marked a paradigm shift in the management of AD and paved the way for future therapies. As real-world evidence on biologics and Janus kinase (JAK) inhibitors is rapidly emerging, comprehensive and up-to-date reviews are important.

Areas covered: This review primarily summarizes real-world data on dupilumab, with a focus on its effectiveness and safety in clinical practice, highlighting recent findings and their implications for optimizing patient management.

Expert opinion: Real-world evidence consistently confirms the high effectiveness, favorable safety profile, and long-term treatment durability of dupilumab across various patient subgroups, including children, elderly, and patients with different underlying immune and genetic profiles. Additionally, dupilumab has demonstrated beneficial effects in patients with comorbid asthma and food allergies. Emerging data further support its potential safety during pregnancy, breastfeeding, and (non-)live vaccinations. Dose tapering is often successful and lowers the socio-economic impact of dupilumab.