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darbepoetin alfa (Darbatitor)

✓ Approved

Torrent Pharmaceuticals Limited · EPOR · 重组蛋白

什么是 darbepoetin alfa？

darbepoetin alfa 是一种重组蛋白，由Torrent Pharmaceuticals Limited研发。该药已获批，用于治疗相关适应症。

药物档案

商品名Darbatitor
公司Torrent Pharmaceuticals Limited
药物类别重组蛋白
分子靶点EPOR
状态Approved
来源： ClinicalTrials.gov, Torrent Pharmaceuticals Limited

作用机制

分子靶点

darbepoetin alfa 作用于 1 个分子靶点：

EPORerythropoietin receptor (EPO-R)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

darbepoetin alfa 针对 1 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Blood and lymphatic system disordersNephrogenic anaemia✓ Approved

相关研究文献

PubMedKidney diseases (Basel, Switzerland)2026-08-05

Efficacy and Cardiovascular Safety of Pegmolesatide in Patients with Anemia of Chronic Kidney Disease: Post hoc Analysis of Two Phase 3 Randomized Trials.

Ye Zhiming Z, Zhang Ping P, Hu Zhizhen Z, Cheng Xue X et al.

Pegmolesatide selectively binds to the erythropoietin receptor homodimer with higher binding stability and prolonged residency than erythropoietin, potentially offering an improved therapeutic profile for anemia management in chronic kidney disease (CKD) patients. This post hoc analysis of two randomized phase 3 trials compared pegmolesatide with epoetin alfa to further evaluate pegmolesatide's potential benefits. This analysis included a total of 545 patients: 372 with dialysis-dependent (DD)-CKD (248 pegmolesatide, 124 epoetin alfa) and 173 with non-dialysis-dependent (NDD)-CKD (115 pegmolesatide, 58 epoetin alfa). The proportion of patients maintaining mean hemoglobin (Hb) levels ≥10 g/dL and intraindividual hemoglobin variability (Hb-Var) were analyzed. Cardiovascular (CV) safety was assessed using five-point major adverse cardiovascular events (MACE), expanded CV events, and three-point MACE. During the efficacy evaluation period (weeks 17-24), pegmolesatide showed numerically higher proportions of patients who maintained mean Hb levels ≥10 g/dL versus epoetin alfa in both DD-CKD {91.8% vs. 88.6%; difference: 7.1% (95% confidence interval [CI]: 0.1, 14.2); p = 0.2802} and NDD-CKD (87.0% vs. 85.4%; difference: 3.3% [95% CI: -8.1, 14.6]; p = 0.6091) patients. From the efficacy evaluation period to the extended period (weeks 17-52), patients receiving pegmolesatide exhibited lower Hb-Var compared with those receiving epoetin alfa as measured by within-patient residual standard deviation (DD-CKD: 0.678 g/dL vs. 0.730 g/dL, p = 0.0061; NDD-CKD: 0.647 g/dL vs. 0.677 g/dL, p = 0.3158). The incidences of five-point MACE (DD-CKD: 3.7% vs. 6.5%, hazard ratio [HR] = 0.54 [95% CI: 0.21, 1.39]; NDD-CKD: 0.9% vs. 6.9%, HR = 0.14 [95% CI: 0.02, 1.28]), expanded CV events (DD-CKD: 9.8% vs. 11.3%, HR = 0.83 [95% CI: 0.43, 1.61]; NDD-CKD: 5.2% vs. 12.1%, HR = 0.49 [95% CI: 0.16, 1.45]) and three-point MACE (DD-CKD: 2.0% vs. 3.2%, HR = 0.60 [95% CI: 0.16, 2.23]; NDD-CKD: 0 vs. 1.7%, HR not estimable) were consistently lower in the pegmolesatide group across both patient populations. Pegmolesatide showed effectiveness in Hb management for both NDD-CKD and DD-CKD populations, with overall numerically favorable CV safety outcomes compared to epoetin alfa.

PMID 42553958
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PubMedJournal of plastic and reconstructive surgery2026-08-04

Iatrogenic Pan-Craniosynostosis Due to Alkaline Phosphatase Enzyme Replacement Therapy Using Asfotase Alfa for Hypophosphatasia.

Kyutoku Shigeo S

Hypophosphatasia is a relatively rare congenital bone metabolic disease, occurring in approximately 1 in 100,000 to 150,000 births. It is diagnosed in infancy by low bone calcification, rickets-like symptoms visible on X-ray, and decreased serum alkaline phosphatase (ALP) levels in the blood. Prognosis varies by type, and while treatment has not been long established, ALP enzyme replacement therapy has been developed and has achieved improved outcomes since 2015. Pan-craniosynostosis, as an unfavorable side effect of this symptomatic therapy, is not well known even among experienced craniofacial surgeons, likely because it is often regarded as part of the bone transformation process, especially when the unusual discrepancy between a hard cranium and fragile limb bones is observed, or is overlooked due to poor prognosis. The author's purpose is to present rare experiences of two cases of pan-craniosynostosis considered to be iatrogenic after the therapy for hypophosphatasia. Some reports in Japan indicate that this iatrogenic complication may appear in 15.3% of cases after treatment. As craniofacial surgeons, we need to recognize this causal relationship between hypophosphatasia treatment and craniosynostosis.

PMID 42548985
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PubMedCureus2026-08-04

Therapy-Associated Vitiligo: Hypopigmentation Secondary to Programmed Cell Death Protein 1 Inhibitors, Interferon Alfa-2b, Cytotoxic T-Lymphocyte-Associated Protein 4 Inhibitors, and B-Raf/Mitogen-Activated Protein Kinase Kinase Inhibitors.

Verma Kritin K KK, Reddy Sreeya S, Beckham Caleb C, Nguyen Kevin T KT et al.

Background Malignant melanoma (MM) arises from melanocytes, and vitiligo is an autoimmune condition targeting these cells. Although an association between MM and vitiligo has been proposed, underlying mechanisms remain unclear. Because several melanoma treatments modulate immune responses, this study evaluated the relationship between MM and vitiligo across commonly used systemic therapies. Methods In September 2025, using the TriNetX network, patients with MM receiving programmed cell death protein 1 (PD-1) inhibitors, interferon alpha-2b (IFN-α2b), B-Raf serine/threonine kinases (BRAF)/ mitogen-activated protein kinase kinases (MEK) inhibitors, or cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) inhibitors were identified and 1:1 propensity score-matched to patients without MM receiving the same therapy. Demographics and vitiligo incidence were analyzed using risk ratios (RRs) and 95% confidence intervals (CIs) via Wald's method. Counts <10 were suppressed per platform guidelines. Results Baseline demographics were well balanced between MM and matched non-MM patients. MM was significantly associated with higher vitiligo risk among those treated with PD-1 inhibitors (RR: 24.84; 95% CI: 16.92-36.45), IFN-α2b (RR: 3.10; 95% CI: 1.53-6.30), and CTLA-4 inhibitors (RR: 32.04; 95% CI: 18.05-56.88). In the BRAF/MEK cohort, vitiligo occurred only in patients with MM, preventing RR calculation. Conclusions Across multiple therapeutic classes, MM consistently conferred a greater risk of developing vitiligo compared with matched non-MM patients. The strong associations observed with immune-modulating agents support vitiligo as a potential marker of antitumor immune activation rather than a coincidental adverse event. Even rare cases in BRAF/MEK-treated patients suggest that targeted therapy may also influence melanocyte-directed immunity. Further prospective studies are needed to characterize the clinical and immunologic significance of treatment-associated vitiligo.

PMID 42549405
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PubMedEULAR rheumatology open2026-08-02

Management of major organ involvement in Behçet syndrome: a systematic literature review informing the 2025 update of the EULAR recommendations.

Ozguler Yesim Y, Esatoglu Sinem Nihal SN, Tomasson Gunnar G, Falzon Louise L et al.

The objective of this study was to evaluate and update the evidence on pharmacologic and interventional treatments for major organ involvement in Behçet syndrome (BS), in order to inform the 2025 update of the European Alliance of Associations for Rheumatology recommendations. A systematic literature review was conducted using 2 distinct search strategies for pharmacologic and surgical/interventional therapies, covering major databases from October 2015 to November 2024. Controlled studies comparing active interventions with placebo or another active treatment were included. If no controlled trials were available for a specific research question, uncontrolled studies or case series with at least 10 patients with BS were included. Of 7128 citations, 69 studies on major organ involvement and tapering/withdrawal of immunosuppressives were included. Only 5 were randomised controlled trials (RCTs), 4 of them included patients with eye involvement, and 1 included patients with vascular or nervous system involvement. RCTs and comparative observational studies for eye involvement supported the use of monoclonal tumour necrosis factor alfa inhibitors and interferon alfa. The RCT that included patients with vascular or nervous system involvement favoured infliximab over cyclophosphamide based on complete response rates and safety. Observational data additionally supported the use of interferon alfa for venous thrombosis, whereas the role of adjunctive anticoagulation remains unclear. Noncomparative observational studies showed benefit with monoclonal tumour necrosis factor alfa inhibitors in patients with gastrointestinal involvement. This systematic literature review provided evidence on the management of major organ involvement to inform the task force for developing the 2025 update of the European Alliance of Associations for Rheumatology recommendations for the management of BS.

PMID 42540035
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PubMedEClinicalMedicine2026-08-02

Comparative efficacy and safety of four long-acting granulocyte colony-stimulating factors for primary prophylaxis in patients with breast cancer: a prospective observational cohort study in China.

Guo Zihan Z, Hu Jinwei J, Mo Miao M, Wang Mengmeng M et al.

Long-acting granulocyte colony-stimulating factors (G-CSFs) are the standard of care for primary prophylaxis against chemotherapy-induced neutropenia in breast cancer, but comparative data remain limited. This prospective observational cohort study was conducted at a single center in Shanghai, China. Patients with breast cancer scheduled for high-febrile-neutropenia-risk chemotherapy and planned for long-acting G-CSF primary prophylaxis were eligible. Patients received subcutaneous pegfilgrastim, mecapegfilgrastim, telpegfilgrastim, or efbemalenograstim alfa once per cycle per clinical practice. The primary outcome was the incidence of grade 3/4 neutropenia (leukopenia), assessed during chemotherapy cycles and at follow-up visits. Safety was assessed via bone pain-related treatment-emergent adverse events (BPR TEAEs). Between March 25 and September 30, 2025, 407 patients were analyzed. Grade 3/4 neutropenia (leukopenia) occurred in 24.1%, varying numerically across agents (telpegfilgrastim 30.2%, mecapegfilgrastim 16.4%; overall P = 0.061). After multivariable adjustment, telpegfilgrastim was associated with a higher risk of grade 3/4 neutropenia (leukopenia) versus mecapegfilgrastim (OR = 2.46, P = 0.011). BPR TEAEs occurred in 62.4%, with similar incidence across groups (P = 0.32). Lower BPR TEAE risk was associated with ddEC chemotherapy (OR = 0.32, P = 0.0010) and postmenopausal status (OR = 0.53, P = 0.031). In exploratory analyses, administration at 24-48 h (versus <24 h) was associated with lower grade 4 neutropenia (leukopenia) (9.9% versus 27.3%, P = 0.011) and severe BPR TEAEs (0.5% versus 4.5%, P = 0.032), and a 3 mg pegfilgrastim dose was associated with comparable myeloprotection to 6 mg (29.4% versus 26.5%, P = 0.81) but fewer BPR TEAEs (29.4% versus 60.3%, P = 0.022). All four long-acting G-CSFs demonstrated clinical efficacy. Numerical variations and adjusted analyses indicated distinct clinical profiles, supporting tailored prophylaxis. Optimizing administration timing and considering dose reduction could improve benefit-risk balance. Larger prospective studies are needed to confirm these exploratory findings. None.

PMID 42541270
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PubMedOrphanet journal of rare diseases2026-08-01

Defining the therapeutic corridor of stability in enzyme replacement therapy for Pompe disease: a position statement.

Schoser Benedikt B

Pompe disease, also known as glycogen storage disease type II, is a rare, progressive lysosomal storage disorder caused by pathogenic variants in the GAA gene. Enzyme replacement therapy has transformed the natural history of the disease; however, with three agents now approved - alglucosidase alfa, avalglucosidase alfa, and cipaglucosidase alfa plus miglustat - clinicians lack a consensus quantitative framework for determining when a patient has left the zone of therapeutic stability and requires treatment reassessment, escalation, or switching. A structured synthesis was conducted of Phase 2 and Phase 3 randomized controlled trials, open-label extension studies, registry analyses, real-world observational datasets, switching studies, scoping review evidence on global diagnostic and epidemiological variation, and consensus recommendations evaluating approved enzyme replacement therapies for Pompe disease, with an emphasis on late-onset Pompe disease. Outcome domains evaluated included forced vital capacity percentage predicted, six-minute walk test distance, urinary hexose tetrasaccharide, serum creatine kinase, maximal inspiratory and expiratory pressures, anti-drug antibody titers, functional motor scales, and patient-reported outcomes. Across LOTS, COMET and its 97- and 145-week extensions, PROPEL and ATB200-07, ATB200-02, the STIG real-world cohort, the International Pompe Registry, European Pompe Consortium start-switch-stop criteria, post hoc PROPEL switching analyses, real-world alglucosidase-to-avalglucosidase switching data, and the first multicenter real-world analysis of switching to next-generation enzyme replacement therapies, a coherent therapeutic corridor of stability emerges. Forced vital capacity change within approximately - 1% to + 5% per year defines the respiratory stability corridor; a confirmed decline exceeding 5% over 12 months constitutes an alarm threshold. For the six-minute walk test, stabilization within approximately ± 25 m of the peak is an adequate response, whereas a confirmed decline of more than 25 m from the peak is actionable. Biomarker improvement or normalization of urinary hexose tetrasaccharide and creatine kinase is achievable with next-generation enzyme replacement therapies and serves as an early surrogate of enhanced lysosomal glycogen clearance. Multicenter real-world switching data indicate that transitions between enzyme replacement therapy preparations are generally feasible and associated with clinical stability in late-onset Pompe disease. This formal, multi-domain therapeutic corridor of stability for enzyme replacement therapy in Pompe disease is grounded in available Phase 2, Phase 3, extension, registry, consensus, and real-world evidence. The framework supports structured monitoring, timely reassessment of treatment, and personalized management for patients receiving long-term enzyme replacement therapy. Prospective validation with standardized monitoring is required. Not applicable.

PMID 42538546
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