PersisGen Par · IL1R1 · 重组蛋白
anakinra 是一种重组蛋白，由PersisGen Par研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)、Intravenous (IV)、Subcutaneous Injection。
| 商品名 | PerkinRA |
| 公司 | PersisGen Par |
| 药物类别 | 重组蛋白 |
| 分子靶点 | IL1R1 |
| 给药途径 | Injectable (Others), Intravenous (IV), Subcutaneous Injection |
| 状态 | Approved |
anakinra 作用于 1 个分子靶点：
| IL1R1 | interleukin 1 receptor type 1 (P80, CD121A) |
anakinra 针对 11 个适应症，涉及 5 个治疗领域。
| 治疗领域 | 疾病/病症 | 分期 |
|---|---|---|
| Musculoskeletal and connective tissue disorders | Rheumatoid arthritis | ✓ Approved |
| Musculoskeletal and connective tissue disorders | Still's disease | ✓ Approved |
| Musculoskeletal and connective tissue disorders | Juvenile idiopathic arthritis | ✓ Approved |
| Congenital, familial and genetic disorders | Chronic infantile neurological cutaneous and articular syndrome | ✓ Approved |
| Congenital, familial and genetic disorders | Muckle-Wells syndrome | ✓ Approved |
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Nedel Wagner W
Hemophagocytic lymphohistiocytosis (HLH) is a critical syndrome of immune dysregulation, marked by a hyperinflammatory cytokine storm and multiorgan failure. While primary forms are genetic, secondary HLH in adults is more prevalent and frequently precipitated by infections (notably viral), malignancies, or autoimmune diseases. Distinguishing secondary HLH from sepsis and multiorgan failure presents a significant clinical challenge due to overlapping clinical features, such as persistent fever and cytopenias. Diagnosis relies on clinical judgment and predictive frameworks like the HLH-2004 criteria or the HScore. Although elevated ferritin levels are a hallmark biomarker, isolated values lack sufficient specificity, necessitating a multifaceted diagnostic approach. Treatment strategies focus on controlling hyperinflammation while addressing the underlying trigger. Initial therapies often include corticosteroids, intravenous immunoglobulin, or the interleukin-1 receptor antagonist anakinra, with etoposide reserved for severe or refractory cases. Despite advances in management, mortality rates in the intensive care unit remain high, particularly when invasive organ support is required. Early recognition and multidisciplinary collaboration are essential to improve outcomes in this heterogeneous patient population. Further research is necessary to refine diagnostic cutoffs and identify personalized, phenotype-specific therapeutic interventions.
Rapti Vassiliki V, Foutadakis Spyros S, Kakavoulis Nikolaos N, Skorda Lamprini L et al.
To map and compare the serum metabolome of hospitalized patients with COVID-19 or community-acquired pneumonia (CAP). Observational matched cohort study using an untargeted metabolomics approach. Serum samples were obtained at the time of hospital admission as part of two clinical trials conducted in hospitalized patients. A matched cohort design was applied, including patients with COVID-19 and CAP, matched according to age, sex, and Charlson Comorbidity Index. The patient samples were obtained from two clinical studies, namely the suPAR-guided Anakinra Treatment for Validation of the Risk and Management of Respiratory Failure by COVID-19 (SAVE) trial (ClinicalTrials.gov identifier: NCT04357366; European Union Drug Regulating Authorities (EudraCT) number: 2020-001466-11) and the A randomized clinical trial of oral Clarithromycin in Community-acquired pneumonia to attenuatE inflammatory responseS and improve outcomeS (ACCESS trial) (ClinicalTrials.gov identifier: NCT04724044; EudraCT number: 2020-004452-15). The total study population comprised 92 patients. None. A total of 3555 metabolites were detected, and differential analysis recovered more than 70% of the metabolome as altered between conditions. Metabolite pathway analysis highlighted pathways related to the citric acid cycle and arachidonic metabolism as activated in CAP, while COVID-19 was mainly driven by alterations in amino acid metabolism and metabolites related to mitochondrial function. Extensive divergence was observed in the metabolomic landscape of patients with COVID-19 or CAP, underscoring the disease-specific metabolic adaptations and providing potential targets for diagnostic and therapeutic development.
Grom Alexei A, Antón Jordi J, Behrens Edward M EM, Best Corey C et al.
Information is limited on the natural history and current treatment patterns in macrophage activation syndrome (MAS), a life-threatening hyperinflammatory syndrome complicating Still's disease (systemic juvenile idiopathic arthritis [sJIA] and adult-onset Still's disease [AOSD]). AMETHYST aimed to describe real-world treatment patterns and outcomes in glucocorticoid (GC)-refractory MAS complicating Still's disease. In this retrospective cohort study, medical data from January 1, 2012, to March 31, 2023, were abstracted from charts of all eligible patients across eight sites in Europe, Canada, and the US for index MAS episodes (occurring between January 1, 2012, and September 30, 2022, and meeting eligibility criteria). Overall, 55/64 (86%) included patients had sJIA and 9/64 (14%) had AOSD. Most patients (53/64 [82.8%]) were children at index (median age: 7.0 years). MAS was characterized by rash (60.4%), fever (52.8%), and hepatic involvement (49.1%). All patients received GCs; most were also treated with anakinra (48/64 [75%]) and/or ciclosporin (33/64 [51.6%]). Normalization of 7 (complete MAS laboratory remission) or ≥3 (partial remission) prespecified laboratory parameters occurred in 7/64 (10.9%) and 41/64 (64.1%) patients, respectively. GCs were tapered in 50/64 (78.1%) patients (median: 39.9 days). Per investigator assessment of clinical signs/symptoms for the index MAS episode, 24/64 (37.5%) and 26/64 (40.6%) patients had a complete and partial response, respectively. MAS recurred in 20/64 (31.3%) patients. There were 7/64 (10.9%) deaths; estimated 1-year survival probability was 93.75%. Low MAS laboratory remission rates and toxicities of high-dose GCs combined with other treatments highlight the need for safer, more effective therapies.
Eyre Michael M, Han Velda X VX, Thomas Terrence T, Sakuma Hiroshi H et al.
Infection-triggered encephalopathy syndromes (ITES) are acute, para-infectious disorders that can cause disability or death. Recognition is increasingly important in viral pandemics, but clinical features and outcomes remain poorly understood. PubMed search (inception to 6 March 2024) identified studies with published individual patient data (raw data were not collected from authors). The search was updated on 14 May 2026 using the same terms to identify reports and cases published after the data extraction cutoff. Data were extracted by paediatric neurologists using a standardised proforma. Major syndromes included acute encephalopathy with biphasic seizures and late reduced diffusion (AESD), acute necrotising encephalopathy (ANE), acute shock with encephalopathy and multiorgan failure (ASEM), hemiconvulsion-hemiplegia-epilepsy syndrome (HHE), febrile infection-related epilepsy syndrome (FIRES) and mild encephalopathy with reversible splenial lesion (MERS). We performed a pooled analysis of individual-level published data investigating patient characteristics, infections, and clinical outcomes measured by six-month modified Rankin Scale (mRS). Infection-syndrome associations were analysed with chi-square normalised residuals. 1946 patients from 656 studies (by ascending age of onset) included 164 ASEM (median age 0.78 years), 217 AESD (1.3 years), 95 HHE (2 years), 414 ANE (3.4 years), 562 FIRES (9 years), and 422 MERS (9.25 years). An updated search identified 658 cases from 171 studies that could be eligible for inclusion. AESD was linked to human herpesvirus 6 (z = 12.87); ANE with influenza A (z = 11.1), SARS-CoV-2 (z = 9.1) and influenza B (z = 4.3); MERS with rotavirus infection (z = 7.48); and FIRES with absence of microbiological identification (z = 18.2) (all p < 0.001).Neuroimaging was often delayed. Magnetic resonance imaging (MRI) brain restricted diffusion was the commonest finding, typically bilateral (except HHE). Characteristic patterns included thalamic involvement with or without haemorrhagic changes in ANE, corpus callosum involvement in MERS, and subcortical white matter involvement in AESD and HHE. Cerebrospinal fluid pleocytosis occurred mostly in FIRES and MERS, and elevated protein in ANE.Immunotherapy (commonly steroids, immunoglobulin) including biologics (anakinra, tocilizumab) was used most in ANE and FIRES. Acute mortality was 12% (227/1946), highest in ASEM (50%) and ANE (35%). There was good six-month outcome (mRS 0-2) in 52% (95% CI 50-55; 615/1174): MERS 99% (95% CI 97-100; 323/326), AESD 54% (95% CI 44-64; 50/93), FIRES 38% (95% CI 33-44; 120/314), ANE 33% (95% CI 27-38; 88/269), HHE 16% (95% CI 7-32; 5/32), and ASEM 15% (95% CI 9-24; 14/91). ITES showed distinct demographic, clinico-radiological features and outcomes. This largest ITES cohort to date highlights the importance of timely imaging, intervention, and future global collaboration to advance diagnosis, treatment, and pandemic preparedness. No funding was received for this work.
Mazloum Hala H, Buchholz Shenja S, Schifter Hanna H, Ameling Sabine S et al.
Pancreatitis is a life-threatening inflammatory disease of the pancreas. The cytokine interleukin-1α has been demonstrated to act as an alarmin released by necrotic cells. In the present study, we investigated the influence of IL-1α on the immune response during acute and chronic pancreatitis. Following tissue injury, pancreatic acinar cells released IL-1α, which activates tissue-resident fibroblasts to differentiate toward a pro-inflammatory phenotype. By secreting chemokines and cytokines such as CXCL5, CCL2, and IL-6, these fibroblasts recruit immune cells to the pancreas. The absence of IL-1α reduces disease severity in acute pancreatitis and chemokine release. Furthermore, IL-1α primes fibroblasts to enhance the production of extracellular matrix-components by the up-regulation of pro-fibrotic receptors such as Il4ra, Il13ra1, and Tgfbr3. Therefore, the deletion of IL-1α significantly reduced the development of tissue fibrosis. A therapeutic blockade of the IL1R1-signaling by i.p. administration of the IL-1-receptor antagonist Anakinra showed the same effect; the severity of acute pancreatitis and fibrogenesis during chronic pancreatitis were reduced. In conclusion, the crosstalk between necrotic acinar cells and fibroblasts mediated by IL-1α plays a crucial role in acute inflammation of the pancreas and fibrogenic signaling. Blockade of IL1R1-signaling by Anakinra is therefore a promising therapeutic intervention for both acute and chronic pancreatitis.
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