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sirolimus (Excel / Excel II)

✓ Approved

JW Medical Systems · MTOR · 小分子

什么是 sirolimus？

sirolimus 是一种小分子，由JW Medical Systems研发。该药已获批，用于治疗相关适应症，给药途径：Surgical Implantation。

药物档案

商品名Excel, Excel II
公司JW Medical Systems
药物类别小分子
分子靶点MTOR
给药途径Surgical Implantation
状态Approved
来源： ClinicalTrials.gov, JW Medical Systems

作用机制

分子靶点

sirolimus 作用于 1 个分子靶点：

MTORmechanistic target of rapamycin kinase (FRAP2, RAPT1)
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

sirolimus 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
Injury, poisoning and procedural complicationsRestenosis✓ Approved

相关研究文献

PubMedHuman gene therapy2026-08-05

Safety, Tolerability, and Efficacy of a Prophylactic Sirolimus Protocol for Patients Receiving Delandistrogene Moxeparvovec-Rokl Gene Therapy.

Siddiqui Zahraa Z, Neumann Serena S, Guillen Daniel D, Patel Ruchee R et al.

Delandistrogene moxeparvovec-rokl (DMR) is the only U.S. Food and Drug Administration-approved gene therapy for patients with Duchenne muscular dystrophy (DMD). After reports of two deaths due to acute liver injury (ALI)/acute liver failure, our center began prophylaxis with sirolimus to help prevent ALI. The objective of this study is to report the initial safety, tolerability, and efficacy of sirolimus prophylaxis. The clinical course of patients receiving sirolimus prophylaxis with a target trough of 4-6 ng/mL was retrospectively evaluated and compared with those who did not receive sirolimus. Laboratory testing, particularly gamma glutamyl transferase, aspartate aminotransferase, alanine aminotransferase, and total bilirubin, was collected. A Fisher's exact test was used to assess the difference between groups. Twelve patients were started on sirolimus prophylaxis, 10 prior to DMR infusion and 2 within 2 weeks of DMR. The median age at DMR dosing was 11 years old (interquartile range = 8.5-14); 11 patients were ambulatory. No patients experienced a serious adverse event related to sirolimus administration. Seven patients developed elevated triglyceride or cholesterol levels and began omega-3 supplementation. None of the patients receiving sirolimus prophylaxis experienced ALI compared with 3 of the 14 patients (21%) infused prior to implementation of the sirolimus protocol. Sirolimus prophylaxis appears to be safe and well-tolerated in DMD patients receiving DMR. While not statistically significant, these results suggest that further study of sirolimus prophylaxis is warranted.

PMID 42552850
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PubMedJournal of pediatric hematology/oncology2026-08-05

A Case Report of Bilateral Pulmonary Emboli in a Patient With Fibroadipose Venous Anomaly Treated With Sirolimus.

Farrell Meredith M, McDaniel Janice J, Kendel Nicole E NE

Sirolimus is a first-line treatment for venous malformations in pediatric patients. Current studies describing adverse events have not shown an increased incidence of venous thromboembolism (VTE). We present a pediatric patient with a vascular anomaly who developed bilateral pulmonary emboli while on sirolimus with minimal other risk factors for clot development. While it is not possible to conclusively prove that the development of pulmonary emboli was related to sirolimus, given the possible association, clinicians who treat patients with vascular anomalies may need to consider patients treated with sirolimus at a higher risk for VTE.

PMID 42554458
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PubMedClinical research in cardiology : official journal of the German Cardiac Society2026-08-04

Performance of a sirolimus-eluting balloon with phospholipid nanocarriers in the treatment of long de novo coronary artery disease: the prospective GINGER trial.

Ielasi Alfonso A, Gitto Mauro M, Galli Stefano S, Sangiorgi Giuseppe Massimo GM et al.

Sirolimus-coated balloons (SCB) may offer improved antiproliferative efficacy over paclitaxel-coated balloons; however, data regarding their performance in de novo coronary lesions are limited. The GINGER study aimed to evaluate the angiographic efficacy and clinical safety of a SCB (Magic Touch®) in the treatment of long (≥ 25 mm) de novo coronary artery lesions. This observational, prospective, multicenter, single-arm cohort study enrolled patients with at least one long de novo lesion treated with SCB. Co-primary endpoints were late lumen loss (LLL) and net lumen gain at 9 months. Secondary endpoints included: procedural success, periprocedural myocardial infarction, binary restenosis and a device-oriented composite endpoint (DOCE), defined as the composite of cardiac death, target vessel myocardial infarction (TV-MI) and clinically-driven target lesion revascularization (CD-TLR). The study was registered at ClinicalTrials.gov (NCT05471245). A total of 104 patients was enrolled at 8 centers. Mean lesion length was 28.9 ± 16.0 mm and reference vessel diameter was 2.3 ± 0.6 mm. Procedural success was achieved in all cases. At 9-month angiographic follow-up, LLL was 0.28 ± 0.68 mm, net lumen gain 0.62 ± 0.63 mm, and binary restenosis was observed in 33.3% of lesions. Clinical events at 12-month included DOCE in 6.7% of patients, cardiac death in 1.0%, TV-MI in 4.8% and CD-TLR in 3.8%. No lesion thrombosis was reported. In a real-world cohort of patients with long de novo coronary lesions, the Magic Touch SCB was associated with a LLL of 0.28 mm, which should be interpreted in light of the high complexity of treated lesions. Future randomized trials are warranted to evaluate its clinical efficacy in comparison with new-generation drug-eluting stents.

PMID 42550208
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PubMedMolecular syndromology2026-08-04

Hypomelanosis of Ito with Local Overgrowth due to a Somatic Complex MTOR Gene Variant Consistent with Smith-Kingsmore Syndrome in One of Monozygotic Twins Detectable Only by RNA Sequencing: A Case Report.

Karaca Nazlı Balcan NB, Ceylaner Serdar S, Yeniay Süt Nurşah N, Alıcı Nurettin N et al.

Smith-Kingsmore syndrome is a rare neurodevelopmental disorder that is caused by somatic variants in the MTOR gene. The present study reports on a monozygotic twin exhibiting hypomelanosis of Ito and localized brain overgrowth, attributable to a complex somatic MTOR variant, which is only detectable by RNA sequencing (RNAseq). A 9-year-old girl with a healthy monozygotic twin developed seizures at the age of 3 months. Due to refractory epilepsy and focal cortical dysplasia identified on brain magnetic resonance imaging, she underwent resective epilepsy surgery at the age of 5 years. Despite initial seizure control, seizures later persisted, and by the age of 9 years, she exhibited progressive autism spectrum features and intellectual impairment. Whole exome sequencing performed on a blood sample was unremarkable. On re-evaluation, faint Blaschko lines were observed under mobile phone light, raising suspicion of somatic mosaicism. Due to technical limitations of DNA-based testing in paraffin-embedded brain tissue, RNAseq of affected tissue was performed, revealing a complex somatic MTOR variant. Treatment with the mTOR inhibitor sirolimus resulted in approximately 50% reduction in seizure frequency and improvement in autistic features and verbal communication. This case highlights several important clinical insights. The presence of discordant clinical features in monozygotic twins may be indicative of mosaicism. Cutaneous findings, such as Blaschko lines, may be subtle and easily overlooked. The concept of mosaicism should be considered in the context of localized brain dysplasias. RNAseq has been identified as a valuable diagnostic tool, particularly in challenging samples such as paraffin-embedded tissues, where conventional genomic methods may be limited.

PMID 42549377
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PubMedCatheterization and cardiovascular interventions : official journal of the Society for Cardiac Angiography & Interventions2026-08-03

Clinical Outcomes After Scoring-Balloon and Sirolimus-Eluting Balloon Strategy in Diffuse Small-Vessel Coronary Artery Disease (SCA-DEB Study): A Single-Center Prospective Study.

Vassilev Dobrin D, Mileva Niya N, Panayotov Panayot P, Shurupov Vladimir V et al.

The treatment of diffuse coronary artery disease is challenging, with high rates of repeat revascularization without satisfactory treatment. We designed a prospective, single-center, single-arm study to evaluate the feasibility and safety of a new strategy for the treatment of diffuse, small-vessel coronary artery disease using a systematic combination of Scoring-balloon Angioplasty and Sirolimus-eluting balloon Angioplasty. We included patients with diffuse (lesion length ≥ 20 mm) and small vessel (diameter 1.5-2.75 mm) coronary disease. Patients with STEMI, left main stenosis, and a life expectancy of less than 24 months were excluded. The procedure strategy was scoring-balloon angioplasty (SBA), 1:1 diameter ratio with distal reference plus sirolimus-eluting balloon angioplasty (SEB) using Mozec balloon (Meril, India). From December 2022 until June 2025, 167 patients with 194 vessels were included. The mean age was 67 ± 9 years, 71% males, 92% with dyslipidemia, and 44% diabetics. The left anterior descending artery was the most frequently treated vessel (66%). The mean reference vessel diameter was 2.1 ± 0.74 mm, MLD 0.71 ± 0.31 mm, and %DS 67 ± 14%. The mean lesion length was 35 ± 15 mm. For a median clinical follow-up of 15 [8-29] months, the overall rate of MACE was 5.4%, 4.8% patients had TLR, and the total percentage of POCE was 10.2%. A novel strategy using a combination of scoring balloon angioplasty followed by sirolimus-eluting balloon inflation for the treatment of diffuse coronary artery disease yields promising angiographic and clinical outcomes.

PMID 42543702
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PubMedExperimental and clinical transplantation : official journal of the Middle East Society for Organ Transplantation2026-08-01

Neoplastic Complications Under mTOR Inhibitors in Kidney Transplant Recipients: 2 Case Reports.

Dahmane Rihem R, Ben Aicha Narjess N, Dziri Sonia S, Azzabi Awatef A et al.

Malignancy remains a major cause of late morbidity and mortality in kidney transplant recipients, largely due to chronic immunosuppression and impaired tumor immune surveillance. Mammalian target of rapamycin inhibitors have antiproliferative and antiangiogenic properties and are frequently used in recipients considered to be at increased oncologic risk. However, their protective effect against de novo malignancy is not absolute. Here, we report 2 cases of severe malignancies that developed in kidney transplant recipients after conversion from calcineurin inhibitors to sirolimus following polyomavirus-associated nephropathy. The first patient, a 55-year-old man, developed prostate adenocarcinoma 6 years after transplant and 4 years after conversion to sirolimus. The diagnosis was established during evaluation for severe anemia and graft dysfunction. The second patient, a 35-year-old woman, developed primary central nervous system posttransplant lymphoproliferative disorder 4 years after transplant and 2 years after conversion to sirolimus. Histopathologic examination confirmed an aggressive lymphoma without detectable Epstein-Barr virus infection. These cases illustrate that mammalian target of rapamycin inhibitor-based immunosuppression does not eliminate the risk of solid or hematologic malignancy. Cumulative immunosuppressive exposure, viral complications, and delayed conversion may contribute to persistent oncogenic risk. Careful long-term oncologic surveillance and individualized immunosuppressive management remain essential in kidney transplant recipients.

PMID 42538715
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