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eculizumab (Ablyze)

✓ Approved

Cinnagen Co · C5 · 单克隆抗体

什么是 eculizumab？

eculizumab 是一种单克隆抗体，由Cinnagen Co研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)、Intravenous (IV)。

药物档案

商品名Ablyze
公司Cinnagen Co
药物类别单克隆抗体, 抗体
分子靶点C5
给药途径Injectable (Others), Intravenous (IV)
状态Approved
来源： ClinicalTrials.gov, Cinnagen Co

作用机制

分子靶点

eculizumab 作用于 1 个分子靶点：

C5complement C5 (C5b, C5D)
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

eculizumab 针对 4 个适应症，涉及 3 个治疗领域。

治疗领域疾病/病症分期
Blood and lymphatic system disordersHaemolytic uraemic syndrome✓ Approved
Nervous system disordersMyasthenia gravis✓ Approved
Renal and urinary disordersParoxysmal nocturnal haemoglobinuria✓ Approved
Nervous system disordersNeuromyelitis optica spectrum disorder✓ Approved

相关研究文献

PubMedInfection and drug resistance2026-07-26

Eculizumab-Induced Acute Heart Failure Decompensation in a Patient with Clinically Suspected Complement-Mediated Thrombotic Microangiopathy: A Case Report.

Huang Jiayang J, Ni Tongtian T, Liu Hong H, Yao Yi Y et al.

Eculizumab is a highly effective and generally safe therapy for atypical hemolytic uremic syndrome (aHUS), a rare subtype of thrombotic microangiopathy (TMA). This report presents a case of recurrent episodes of acute decompensated heart failure temporally related to its administration, a possible adverse effect that is exceedingly rare and poorly documented in the literature. A 45-year-old woman presented with altered mental status and received a clinical diagnosis of complement-mediated thrombotic microangiopathy based on the presence of thrombotic microangiopathy and marked complement activation. Within hours of the first and subsequent eculizumab infusions, she developed recurrent episodes of acute decompensated heart failure, evidenced by respiratory distress, tachycardia, soaring pro-BNP levels, and echocardiographic confirmation of left ventricular dysfunction. These episodes consistently impeded weaning from mechanical ventilation. The temporal association was reinforced when withholding eculizumab was associated with symptom resolution, while a subsequent reduced-dose challenge promptly reinduced cardiac decompensation. This case raises the possibility that eculizumab may trigger acute decompensated heart failure in susceptible patients. It alerts clinicians to monitor for this potentially life-threatening complication and underscores a significant therapeutic dilemma when first-line therapy is associated with severe adverse effects. Further investigation is needed to understand the mechanism and identify at-risk patients.

PMID 42502762
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PubMedPediatric nephrology (Berlin, Germany)2026-07-25

Typical or atypical hemolytic uremic syndrome? That is the question.

Ardissino Gianluigi G, Bernardi Silvia S, Dato Letizia L, Mancuso Maria Cristina MC et al.

An 11-month-old boy with a clinical diagnosis of hemolytic uremic syndrome (HUS) associated with Shiga toxin-producing Escherichia coli infection (STEC-HUS) and a documented presence of both Shiga toxin (Stx) 1 and 2 in his stool showed an unexpected drop in platelet count during recovery (day 13 after diagnosis, day 10 after platelet nadir). Although clinically mild (from 305,000/mm3 to 216,000/mm3), this decline diverged from the expected platelet course observed in a cohort of 148 confirmed STEC-HUS patients treated at our center during the last decade and described in detail elsewhere, raising suspicion of an overlapping condition. Differential diagnoses were therefore investigated. ADAMTS13 activity and homocysteine levels were within normal range, and blood tests revealed a C3 level of 0.62 g/L. Given the unusual course of platelet count, which further dropped to 157,000/mm3, alongside the decreased C3 level, atypical HUS (aHUS) was suspected and treated accordingly with intravenous eculizumab. Platelet count peaked at 417,000/mm3 within 7 days, kidney function normalized, and the patient was discharged 23 days after admission. Genetic analysis revealed two heterozygous rare variants of uncertain significance: p.(Gly759Arg) and p.(Gly110Arg) in the complement factor H (CFH) and factor I (CFI) genes, respectively. Given the diagnostic uncertainty, C5 inhibition (C5i) was discontinued, but HUS relapsed 3.5 months later following a febrile upper respiratory tract infection. This case highlights the diagnostic challenge of discriminating aHUS when STEC infection coexists. As variants in complement regulatory genes (including pathogenic, likely pathogenic, or variants of unknown significance) are not uncommon in the general population, careful monitoring of platelet count using disease-specific reference trajectories may represent a clinically useful tool to detect early deviation from classical STEC-HUS evolution and prompt timely C5i, preventing severe consequences.

PMID 42501081
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PubMedHematology reports2026-07-24

Clinical Spectrum and Differential Diagnosis of Adult Thrombotic Microangiopathies: Real-World Experience from a Tertiary Referral Center.

Kırkayak Nazlı Pelin NP, Ozet Gulsum G, Dagdas Simten S, Ceran Funda F et al.

Background/Objectives: Thrombotic microangiopathies are rare, life-threatening hematological disorders characterized by microangiopathic hemolytic anemia, thrombocytopenia, and end-organ injury. This study was conducted in a setting where ADAMTS13 activity testing became available only from 2015 onward and complement-targeted therapy (eculizumab) had limited accessibility throughout most of the study period, conditions that shaped both diagnostic classification and treatment outcomes. Their clinical presentation, treatment response, and prognosis vary according to etiology, making early recognition and subtype classification clinically important. This study aimed to evaluate the etiological distribution, clinical features, treatment responses, and outcomes of adult patients with thrombotic microangiopathy at a tertiary-center real-world cohort. Methods: This retrospective cohort study included 47 adult patients (≥18 years) hospitalized with thrombocytopenia and microangiopathic hemolytic anemia (MAHA) in a nine-year period. Patients were classified as thrombotic thrombocytopenic purpura (TTP), hemolytic uremic syndrome (HUS), or secondary TMA based on clinical and laboratory evaluation. Demographic characteristics, clinical manifestations, laboratory parameters, treatments, and outcomes were analyzed. Results: The mean age was 45.3 ± 15.2 years, and 72.3% of patients were female. Primary thrombotic microangiopathy accounted for 74.5% of cases, including thrombotic thrombocytopenic purpura in 53.2% and hemolytic uremic syndrome in 21.2%; secondary thrombotic microangiopathy accounted for 25.5%. Hemodialysis was required in all patients with hemolytic uremic syndrome compared with 16% of those with thrombotic thrombocytopenic purpura. The complete response rate was 74.5%, and in-hospital mortality was 25.5%. In multivariable Cox regression analysis, treatment non-response and reduced post-treatment estimated glomerular filtration rate independently predicted mortality. Conclusions: Adult TMAs are characterized by considerable etiological and clinical heterogeneity, which makes differential diagnosis challenging, particularly in settings where access to contemporary diagnostic tests and targeted treatments is limited. In this cohort, in the absence of ADAMTS13 testing, TTP was the most frequent subtype, while treatment non-response and renal impairment emerged as the main factors associated with mortality. These findings emphasize the need for early clinical recognition and careful subtype-based differential diagnosis, which will reduce morbidity and mortality by permitting rapid initiation of pathophysiology-based appropriate interventions, i.e., PEx, immune suppression and caplacizumab for immune TTP and anti-complement therapy for aHUS, and limiting the inappropriate use of PEx with its complications, including sepsis.

PMID 42496448
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PubMedKidney international2026-07-23

Biallelic pathogenic variants in EXOSC3 mediate renal thrombotic microangiopathy of the kidney.

Walsh Patrick R PR, Basu Uttiya U, Barakat Tahsin Stefan TS, Beck Bodo B BB et al.

Thrombotic microangiopathy (TMA) is characterized by the classical triad of microangiopathic hemolytic anemia, thrombocytopenia and acute kidney injury. Complement inhibition with eculizumab is highly efficacious in TMA secondary to complement dysregulation. However, there are a growing number of eculizumab nonresponsive TMAs reported. Recently a syndromic form of TMA due to recessive variants in RNA exosome components (EXOSC3, EXOSC5) has been identified. The underlying pathogenesis remains unclear. We identified 34 children across Europe with pontocerebellar hypoplasia 1b (PCH1b) due to EXOSC3 rare variants and reviewed their clinical history for signs of TMA. To further examine the pathogenesis, a tamoxifen-inducible whole body Exosc3 conditional knockout mouse model (Exosc3KO) was used. Thirteen (eight male, five female) cases of EXOSC3-TMA were identified. In the United Kingdom the incidence of EXOSC3-TMA was 0.004/million/year. Three children received long-term eculizumab therapy, one child failed to respond and two relapsed on treatment. Exosc3KO demonstrated cell cycle arrest and apoptosis resulting in death in a median of eight days with sequelae noted in actively dividing cells in the bone marrow and large intestine. In this timeframe no kidney pathology was identified. EXOSC3-TMA is a severe, early-onset, C5 inhibitor resistant TMA. EXOSC3-TMA should be considered in eculizumab resistant pediatric TMA, particularly in the context of neurodevelopmental disease.

PMID 42486191
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PubMedBMJ open2026-07-22

Clinical similarity in cost-comparison evaluations: a systematic review of current methods in NICE appraisals and the development of a framework for the formal assessment of clinical similarity.

Edwards Steven J SJ, Burgess Benjamin J BJ, Downes Nicole N, Ip Sophie S et al.

To review how statistically non-significant indirect treatment comparison (ITC) results are interpreted within National Institute for Health and Care Excellence (NICE) cost-comparison evaluations (CCEs) and develop a framework to support interpretations of these results from Bayesian network meta-analyses (NMAs). A systematic review of CCEs between 2017 (first introduced) and April 2025. A framework (point-and-density plots) was developed to better interpret statistically non-significant NMA results for CCEs. CCEs were identified through NICE website searches, references of similar reviews and communications with NICE. NICE technology appraisals (from 2017) that followed a CCE approach ab initio, had final guidance available and used non-statistically significant ITC results were included. A single reviewer performed screening and data extraction with validation by a second reviewer. Narrative syntheses were performed separately for company, External Assessment Group (EAG) and committee perspectives. Point-and-density plots combine elements of forest plots and density plots alongside reporting the probability that a treatment is non-inferior relative to a comparator. These were applied to a recent CCE (TA1019) for crovalimab for patients with paroxysmal nocturnal haemoglobinuria. Among 41 CCEs, EAGs raised concerns about statistically non-significant ITC results while companies relied heavily on them. Only ∼32% of CCEs applied formal methods to explore ITC result uncertainty.For the example framework analysis, comparisons of crovalimab to eculizumab (mean difference (MD): 0.018; 95% CIs -0.22 to 0.25) and ravulizumab (MD: 0.079; 95% CIs -0.25 to 0.41) were statistically non-significant, with non-inferiority not demonstrated. However, point-and-density plots indicated a 95.9% and 86.3% probability of non-inferiority of crovalimab versus eculizumab and ravulizumab. Interpretations of statistically non-significant ITC results are inconsistent within individual CCEs and across appraisals. Implementation of the presented recommendations and framework would improve the consistency and robustness of CCEs. CRD420251034143.

PMID 42481194
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PubMedNephron2026-07-21

Sustained terminal complement inhibition on the endothelium with ravulizumab in patients with atypical Haemolytic Uremic Syndrome.

Gastoldi Sara S, Bresin Elena E, Pasini Andrea A, Nardini Beatrice B et al.

Atypical haemolytic uremic syndrome (aHUS) is a rare and severe form of thrombotic microangiopathy caused by dysregulation of the alternative complement pathway, leading to sustained complement activation at endothelial level and microvascular thrombosis, predominantly affecting the kidney. Anti-C5 therapy with eculizumab has markedly improved patient outcomes, with ex-vivo studies showing suppression of endothelial C5b-9 deposition by sera from treated patients, in contrast to the marked C5b-9 formation induced by acute-phase sera. Ravulizumab, a long-acting anti-C5 antibody derived from eculizumab, has recently been approved for aHUS, offering extended dosing intervals, and maintaining (in patients switched from eculizumab) or inducing (in de novo treated patients) disease remission. Its ability to inhibit terminal complement activation at the endothelial level has not been yet evaluated. In this retrospective study, six patients with primary aHUS were assessed, including four switched from eculizumab and two treated de novo with ravulizumab. Serum-induced C5b-9 formation on human microvascular endothelial cells (HMEC-1) was measured ex-vivo at multiple time points during treatment. C5b-9 formation was in normal range on HMEC-1 exposed to sera collected from all aHUS patients treated with ravulizumab, regardless of prior eculizumab exposure, and all patients remained in or achieved clinical remission. These findings provide mechanistic evidence supporting ravulizumab as an effective inhibitor of terminal complement activation and highlight ex-vivo C5b-9 assessment as a potential tool for monitoring treatment efficacy in aHUS.

PMID 42479640
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