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ciclosporin (Gengraf / ciclosporin, AbbVie)

✓ Approved

AbbVie, Inc. · PPIA · 小分子

什么是 ciclosporin？

ciclosporin 是一种小分子，由AbbVie, Inc.研发。该药已获批，用于治疗相关适应症，给药途径：Oral (PO)。

药物档案

商品名Gengraf, ciclosporin, AbbVie
公司AbbVie, Inc.
药物类别小分子
分子靶点PPIA
给药途径Oral (PO)
状态Approved
来源： ClinicalTrials.gov, AbbVie, Inc.

作用机制

分子靶点

ciclosporin 作用于 1 个分子靶点：

PPIApeptidylprolyl isomerase A (HEL-S-69p, CYPH)
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

ciclosporin 针对 3 个适应症，涉及 3 个治疗领域。

治疗领域疾病/病症分期
Skin and subcutaneous tissue disordersPsoriasis✓ Approved
Musculoskeletal and connective tissue disordersRheumatoid arthritis✓ Approved
Immune system disordersSolid organ transplant rejection✓ Approved

相关研究文献

PubMedJournal of the European Academy of Dermatology and Venereology : JEADV2026-09-09

Management of chronic urticaria: Current status and future prospect.

Salman Andaç A, Al-Ahmad Mona M, Al-Nesf Maryam M, Chu Chia-Yu CY et al.

Chronic urticaria is a mast cell-driven inflammatory disease characterized by recurrent wheals, angioedema or both for more than 6 weeks, with substantial effects on sleep, quality of life, mental health and daily functioning. Type I autoallergic and type IIb autoimmune mechanisms represent major endotypes, but many patients show overlapping or mixed features, contributing to heterogeneous clinical courses and variable treatment responses. Current management relies on confirming the diagnosis, excluding differential diagnoses, identifying aggravating factors and comorbidities and monitoring disease activity and control using validated patient-reported outcome measures. A limited, clinically guided diagnostic workup is preferred, with extended investigations reserved for selected cases based on history, examination, red flags or isolated angioedema. Second-generation H1-antihistamines remain the first-line therapy, with up-dosing recommended in insufficient responders. Omalizumab has transformed the treatment of antihistamine-refractory disease, while newer options such as dupilumab and remibrutinib further expand the therapeutic landscape. Ciclosporin remains an effective option in selected patients, particularly those with severe or difficult-to-treat disease, but requires careful safety monitoring. In chronic inducible urticaria, provocation testing, threshold assessment, trigger counselling and individualized treatment are central to care. Despite recent advances, important unmet needs remain, including reliable biomarkers for endotyping, evidence-based selection among emerging therapies, better data in children and other special populations, standardized definitions of remission and relapse and disease-modifying strategies. Future management is expected to move towards biomarker-driven, personalized care, enabling more precise treatment selection and sustained disease control.

PMID 42712073
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PubMedArthritis & rheumatology (Hoboken, N.J.)2026-09-07

AMETHYST: a Retrospective Cohort Study of Treatment Patterns and Outcomes in Patients With Glucocorticoid-refractory Macrophage Activation Syndrome Complicating Still's Disease.

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.

PMID 42703797
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PubMedArtificial organs2026-08-29

The Effects of Continuous Venovenous Hemofiltration on Immunosuppressive Drug concentrations in an Ex-Vivo Model of the critically Ill Adult: (CITRIC) study.

Chumas Lucy A LA, Wallis Steven C SC, Sumi Chandra C, Abdul-Aziz Mohd Hafiz MH et al.

The pharmacokinetics of immunosuppressive drugs during continuous kidney replacement therapy (CKRT) modalities such as continuous venovenous hemofiltration (CVVH) are poorly understood, yet these drugs are crucial for transplant and critically ill patients. An ex vivo adult CVVH circuit with AN69ST membrane was established, using whole blood (WB) or blood-crystalloid (BC) to simulate hypoproteinemia and anemia, across a range of ultrafiltration rates (UFR) (1000-4000 mL/h) and point-of-dilution. Study drugs (tacrolimus, ciclosporin, mycophenolic acid (MPA), hydrocortisone, and methylprednisolone) were administered at clinically relevant concentrations. Drug concentrations were quantified by ultra-high-performance liquid chromatography-tandem mass spectrometry, and sieving coefficient (Sc) and clearance (Cl) were calculated. Mean Sc and Cl were significantly higher for BC than WB: methylprednisolone (Sc: 0.44 vs. 0.19 p < 0.001; Cl: 17.31 mL/min vs. 6.99 mL/min p < 0.001), hydrocortisone (Sc: 0.20 vs. 0.09 p = 0.005; Cl: 7.12 mL/min vs. 3.15 mL/min p = 0.01), MPA (Sc: 0.05 vs. 0.02 p < 0.001; Cl: 1.83 mL/min vs. 0.71 mL/min p < 0.001), ciclosporin (Sc: 0.00 vs. 0.00 p = 0.008; Cl: 0.02 mL/min vs. 0.01 mL/min p = 0.004). Tacrolimus was undetectable in ultrafiltrate. Point-of-dilution had minimal effect, except at UFR 4000 mL/h where pre-dilution increased MPA and methylprednisolone clearance. Hydrocortisone and methylprednisolone were cleared by CVVH, while tacrolimus, ciclosporin, and MPA Cl were negligible or absent. Hypoproteinemia and anemia significantly increased drug Cl. These findings provide mechanistic insights and support the need for validation in clinical studies to guide dosing during CKRT.

PMID 42666083
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PubMedDermatology and therapy2026-08-28

Dual-Targeted Therapy in Refractory Pyoderma Gangrenosum: A Focused Systematic Review of Convergent Pathway Targeting.

Shanshal Mohammed M, Karanasios George G

Refractory pyoderma gangrenosum (PG) may persist despite corticosteroids, ciclosporin, biologics or sequential treatment escalation. Dual-targeted therapy (DTT) is increasingly used in medically complex inflammatory bowel disease (IBD), but PG is often recorded only as an extraintestinal manifestation. We examined the direction, extractability and attribution limits of PG outcomes reported during concurrent DTT. PubMed, Embase via Ovid and Web of Science Core Collection were searched from inception to 26 July 2026. Eligible reports described at least one patient with PG receiving concurrent systemic DTT, defined as biologic-biologic, biologic-Janus kinase (JAK) inhibitor or biologic-phosphodiesterase-4 inhibitor combinations. Screening and extraction were performed independently and in duplicate. The analytic unit was the PG exposure. Outcomes were retained as source-reported and were not pooled because PG assessment, follow-up and co-intervention reporting were non-standardised. Fourteen reports described 17 PG exposures. Of these, 14 had an extractable PG-specific cutaneous outcome, all source-reported using non-standardised assessments as improved or resolved. This 14-of-14 pattern reflects the direction of published outcome-bearing observations, not a denominator-based response estimate. Three additional cohort-level records documented PG exposure without a separable cutaneous end-point. A total of 16 exposures were IBD-associated and 11 involved JAK-containing regimens. Individual regimen mapping, diagnostic ascertainment, follow-up and co-intervention reporting were frequently incomplete. All evidence was uncontrolled, safety reporting was limited and the complete exposure denominator was unknown. Favourable PG outcomes were reported across several DTT pathway pairings, but these uncontrolled published observations do not establish efficacy, comparative advantage or added benefit from simultaneous pathway blockade. The next step is a dermatology-embedded prospective registry within IBD DTT programmes, with standardised PG diagnosis, lesion documentation, co-intervention recording and exposure-specific safety ascertainment. Until such evidence is available, DTT should remain an exceptional multidisciplinary consideration rather than an established PG treatment strategy.

PMID 42663862
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PubMedSurgical case reports2026-08-28

Simultaneous Liver-Kidney Transplantation from a Deceased Donor for Glycogen Storage Disease Type Ia: A Case Report.

Hirata Yuta Y, Sanada Yukihiro Y, Takadera Kiichiro K, Akimoto Ryosuke R et al.

Glycogen storage disease type Ia (GSD Ia) is an indication for simultaneous liver-kidney transplantation (SLKT) due to multiple hepatocellular adenomas and chronic renal failure. In this report, we describe a deceased-donor SLKT in a patient with GSD Ia who presented with hypoglycemic attacks, progressive multiple hepatocellular adenomas, and chronic renal failure. The patient was diagnosed with hepatomegaly at the age of 1 year and 6 months, with a diagnosis of GSD Ia, and was started on a special diet. She had been aware of liver tumors since 24 years of age. At 29 years of age, she underwent left lateral segmentectomy and radiofrequency ablation for multiple liver tumors. She began to suffer from renal dysfunction around 39 years of age, and then at 40 years of age, she was diagnosed with chronic renal failure and therefore was started on hemodialysis. She began having recurrent hypoglycemic attacks after the introduction of dialysis. Due to repeated hypoglycemic attacks, multiple hepatocellular adenomas, and chronic renal failure, she was deemed eligible for SLKT and thus was registered for deceased-donor SLKT at 41 years of age. She underwent SLKT at 46 years of age. The liver graft weight was 1060 g, and the graft-to-recipient body weight ratio was 2.41. The kidney graft weight was 170 g. The weight of the excised liver was 2710 g. Although no malignant findings were observed in the excised liver, multiple hepatocellular adenomas were identified. She was able to urinate independently immediately after the SLKT and was able to discontinue dialysis immediately afterward. Immunosuppressive therapy was initiated with basiliximab, ciclosporin A, methylprednisolone, and mycophenolate mofetil (MMF). However, due to abdominal pain and liver dysfunction caused by MMF, she was switched to mizoribine on POD 46, and her abdominal pain improved thereafter. She progressed well and was discharged on POD 49. Her liver and kidney functions remained good approximately 2 years after the SLKT. Adult patients with GSD Ia require transplantation due to hypoglycemic attacks, multiple liver tumors, and chronic renal failure.

PMID 42662052
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PubMedGenes2026-08-27

Integrated Transcriptomic and Machine-Learning Analyses Identify Shared Na+ Overload-Related Gene Signatures in Inflammatory Bowel Disease and Ankylosing Spondylitis.

Wu Luojin L, Ou Chenghao C, Liu Xuan X, Yan Miaohan M et al.

Background: Ankylosing Spondylitis (AS) and inflammatory bowel disease (IBD) exhibit substantial pathophysiological overlap, including dysregulated innate immunity, barrier dysfunction, and Th17-mediated inflammation. However, whether Na+ overload-related genes (NRGs) exhibit shared transcriptional alterations in IBD and AS remains unclear. This study aimed to characterize the expression patterns of NRGs across IBD and AS and to identify candidate genes associated with both diseases. Methods: Differential expression analysis was performed to identify differentially expressed NRGs (DE-NRGs) in diseased tissues relative to normal tissues. Shared DE-NRGs between IBD and AS were screened and defined as common differentially expressed NRGs (Co-DE-NRGs). We then analyzed the correlations of these Co-DE-NRGs and explored their relationships with immune cell infiltration in target tissues. Four machine learning algorithms were applied to screen key NRGs associated with both IBD and AS. Potential therapeutic agents targeting these core biomarkers were predicted using drug-gene interaction databases, and molecular docking was conducted for further validation. Results: A total of 32 shared Co-DE-NRGs were identified for IBD and AS, with nine key regulatory NRGs recognized: CALR, CD63, CYBA, DYSF, HYOU1, IL1B, JAK1, MMP9, and STAT3. Exploratory MR analysis identified disease-specific associations between genetically predicted expression of NRGs and CD, UC, and AS. Genetically predicted STAT3 expression showed positive associations with CD and UC but an inverse association with AS and therefore did not represent a consistent risk factor across the three diseases. Furthermore, transcriptome-based drug-response analysis identified four candidate agents shared between AS and at least one IBD dataset: ciclosporin, BCL-LZH-4, BRD-K79669418, and CID-5951923. Exploratory molecular docking generated STAT3 binding poses for BCL-LZH-4 and CID-5951923, with DOCK Grid Scores of -35.321896 and -28.361128, respectively. CID-5951923 was selected for representative visualization of its predicted interaction with STAT3. Single-cell RNA-sequencing analysis identified tissue- and cell-type-specific STAT3 mRNA expression patterns in the analyzed IBD colonic and AS peripheral-blood datasets, with monocytes representing a major cell population exhibiting detected STAT3 expression in the AS dataset. Conclusions: These findings identify shared NRG-related transcriptional alterations in IBD and AS, with STAT3 emerging as a candidate gene associated with both diseases. Further experimental studies are required to determine whether these alterations reflect the involvement of NECSO and to evaluate their potential diagnostic or therapeutic relevance.

PMID 42650131
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