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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

PubMedVascular health and risk management2026-09-10

Short-Term Efficacy and Safety of Topical Sirolimus Gel in Pediatric Superficial Microcystic Lymphatic Malformations: A Single-Center Interventional Study of 30 Patients.

Xu Weiyang W, Ding Yu Y, Zhao Lei L, Pi Mengqi M et al.

To evaluate the efficacy and safety of 0.2% topical sirolimus gel for pediatric superficial microcystic lymphatic malformations (LMs). This single-center interventional study included 30 pediatric patients (14 males and 16 females; aged 2-15 years) with superficial microcystic lymphatic malformations. All patients received 0.2% topical sirolimus gel twice daily for 12 weeks according to a predefined treatment and follow-up protocol. Clinical efficacy was assessed at baseline and week 12 using clinical examination, standardized photography, symptom scores, and available imaging findings, together with a four-grade physician-assessed response scale. Adverse events were monitored throughout treatment, and blood sirolimus concentrations were measured at weeks 4 and 12. All patients showed lesion reduction, with an overall response rate of 96% (29/30). At week 12, efficacy was graded as IV in 10 patients, III in 11, II in 8, and I in 1. Among patients with pain symptoms, pain scores decreased significantly after treatment (2.88 ± 0.78 vs 1.18 ± 0.88; P < 0.05). In 10 patients with measurable lesions, maximum lesion diameter decreased significantly from 3.22 ± 1.36 cm to 1.89 ± 1.33 cm (P < 0.05). Only mild local adverse effects were observed, and blood sirolimus concentrations remained below 2.0 ng/mL in all patients. Topical sirolimus gel demonstrated favorable efficacy and safety for pediatric superficial microcystic LMs.

PMID 42718634
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PubMedRevista espanola de enfermedades digestivas2026-09-09

Blue rubber bleb nevus syndrome as an uncommon cause of gastrointestinal overt bleeding: a diagnostic and therapeutic challenge.

López-Picazo García María M, Alajarín Cervera Miriam M, Ono Ono Akiko A, Argumánez Tello Víctor V et al.

A 76-year-old woman on anticoagulation for atrial fibrillation presented with melaena. Her medical history included previous jejunal resection, aortic aneurysm repair, and cervical cancer in remission. Initial upper gastrointestinal endoscopy and complete colonoscopy showed no source of bleeding. Despite discontinuation of anticoagulation, she developed recurrent gastrointestinal bleeding with severe transfusion-dependent anaemia. Capsule endoscopy demonstrated bluish submucosal vascular lesions in the jejunum, suggestive of Blue Rubber Bleb Nevus Syndrome (BRBNS), a diagnosis further supported by dermatological findings. Single-balloon enteroscopy revealed multiple jejunal venous blebs, with no active bleeding, throughout the first 240 cm of examined small bowel. Treatment with somatostatin and sirolimus was started; however, ongoing bleeding and clinical instability prompted an enteroscopy-guided surgical strategy. At laparotomy, multiple subserosal jejunal lesions were identified, corresponding to the endoscopic findings. A 20-cm bleeding jejunal segment was resected, followed by end-to-end anastomosis, with preservation of 250 cm of jejunum. BRBNS, also known as Bean syndrome, is a rare vascular malformation disorder characterised by multiple venous malformations, most commonly affecting the skin and gastrointestinal tract. Gastrointestinal involvement may lead to chronic occult bleeding, recurrent overt haemorrhage, iron-deficiency anaemia, and transfusion dependence. Current evidence is largely limited to case reports and small series, making management challenging. Endoscopic therapy is generally preferred when lesions are accessible, but extensive or multifocal small-bowel disease may limit its effectiveness. Sirolimus has shown promising results as systemic therapy, although its delayed onset of action may be unsuitable in patients with severe ongoing haemorrhage or clinical instability. This case highlights the value of capsule endoscopy and device-assisted enteroscopy not only for diagnosing small-bowel BRBNS, but also for guiding bowel-sparing surgery when endoscopic treatment is insufficient and systemic therapy cannot provide timely haemorrhage control.

PMID 42714101
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PubMedFrontiers in medicine2026-09-08

Topical sirolimus for the treatment of verrucous epidermal nevus associated with an HRAS mosaic mutation: a case report.

Wei Qiu-Ju QJ, Xiang Wan-Yan WY, Zheng Wen-Jun WJ, Li Qiu-Ju QJ

Verrucous epidermal nevus is a benign epidermal hamartoma associated with somatic mosaic mutations, which shows poor response to conventional therapies and has a high risk of recurrence. To date, there are no standardized treatment guidelines for this condition. In recent years, targeted treatments for specific gene mutations have provided new directions for the clinical management of this disease. We report a case of a 13-year-old child with verrucous epidermal nevus in whom genetic testing revealed an HRAS mosaic mutation. The child showed poor response to conventional treatments, including surgical excision, topical retinoic acid cream, and oral acitretin. However, after treatment with topical 0.5% (w/w) sirolimus ointment, the child's skin lesions showed reduced area, thinning, improved pigmentation, and decreased roughness, and no local or systemic adverse reactions were observed during the 6-month treatment period. This case suggests that topical sirolimus may represent a potentially effective therapeutic option for patients with verrucous epidermal nevus who are refractory to conventional therapy.

PMID 42707900
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PubMedFuture cardiology2026-09-07

Contemporary sirolimus-based stent platforms versus durable-polymer limus-eluting stents in patients with diabetes undergoing percutaneous coronary intervention: a systematic review and meta-analysis.

Kim Jaehoon J, Nugroho Michael Putra MP, Hong Jinpyo J

We performed a systematic review and meta-analysis of randomized evidence comparing sirolimus-based stent platforms with durable-polymer limus-eluting drug-eluting stents (DES) in patients with diabetes undergoing percutaneous coronary intervention (PCI). PubMed/MEDLINE and Embase were searched through April 2026. Randomized trials and diabetic cohorts from randomized trials reporting approximately 1-year clinical outcomes were included; nonrandomized and surrogate-only reports were excluded. Risk of bias was assessed using the Cochrane Risk of Bias 2 (RoB 2) tool. Random-effects meta-analysis used restricted maximum likelihood estimation with Hartung-Knapp-Sidik-Jonkman confidence intervals. Eight randomized comparisons (5,988 patients) were included. Target lesion failure yielded a risk ratio (RR) of 1.14 (95% confidence interval [CI] 0.57-2.26; I2 = 79.4%); among everolimus-eluting comparators, RR was 1.49 (95% CI 1.03-2.14; I2 = 0%), favoring comparator DES. Target lesion revascularization yielded RR 1.46 (95% CI 0.66-3.25; I2 = 62.9%). Definite/probable stent thrombosis yielded RR 1.50 (95% CI 1.12-2.02), favoring comparator DES, although events were few. Death and target-vessel myocardial infarction estimates were imprecise. Current evidence does not support preferential adoption of dedicated diabetic stent platforms over conventional durable-polymer DES. Longer-term trials are needed. www.crd.york.ac.uk/prospero identifier is CRD420261356994.

PMID 42702707
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PubMedRespiratory medicine case reports2026-09-06

Sporadic lymphangioleiomyomatosis with lymphocyte-predominant pleural effusion misdiagnosed as tuberculosis: a case report.

Lamboro Beniam Tadewos BT, Belachew Dejene Mariamawit M, Belisa Telila Kumneger TK, Gashaw Dessie Mulualem M

Lymphangioleiomyomatosis (LAM) is a rare cystic lung disease that primarily affects women of reproductive age. The disease may involve extrapulmonary sites, including the lymphatic system, and can present with a lymphocyte-predominant pleural effusion mimicking tuberculosis. We report a 35-year-old woman who presented with a three-year history of progressive dyspnea and recurrent lymphocyte-predominant exudative pleural effusions. She was empirically started on anti-TB therapy at a local hospital but showed no clinical improvement. Upon evaluation at a tertiary center, there was decreased air entry over the posterior third of the chest. Repeat pleural fluid analysis demonstrated a milky appearance with elevated triglyceride levels, consistent with chylous effusion. High-resolution CT showed diffuse, bilateral, thin-walled pulmonary cysts and bilateral pleural effusion with retroperitoneal lymphatic involvement, which confirmed the diagnosis of LAM. Treatment with sirolimus was started and showed significant clinical improvement, but the patient discontinued therapy after two months due to financial constraints. In this patient, LAM presented as tuberculous pleuritis that delayed the definitive diagnosis. This should alert clinicians in high TB burden settings to consider this rare disease in reproductive-aged women who present with progressive dyspnea and a lymphocyte-predominant pleural effusion, particularly when there is no response to anti-TB therapy.

PMID 42701434
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PubMedJournal of drugs in dermatology : JDD2026-09-04

Topical Sirolimus for Refractory Cutis Marmorata Telangiectatica Congenita in Adulthood: A Case Report.

Bhatt Ram D RD, Karpoff Kateryna K, Heitman Nicholas N, Aslam Rabail R et al.

Cutis marmorata telangiectatica congenita (CMTC) is a rare congenital vascular disorder characterized by persistent, violaceous, reticulated skin changes that may be complicated by painful ulcerations. Although localized disease often improves with age, generalized CMTC can persist into adulthood and may be associated with limb asymmetry, ocular abnormalities, and neurologic sequelae. Diagnosis can be challenging in atypical adult presentations despite established major and minor criteria. We report a case of a 50-year-old woman with congenital livedo reticularis and Raynaud syndrome who presented with lifelong unilateral left lower-extremity hypoplasia and fixed lacy violaceous patches. Over the preceding decade, she developed recurrent, spontaneous, painful ulcerations exacerbated by cold exposure. Examination revealed a hypoplastic left leg with reticulated violaceous patches and tender, crusted erosions without venectasia; biopsy findings ruled out vasculitis, and the overall clinicopathologic picture met all three major and multiple minor Kienast-Hoeger criteria for CMTC. Multiple therapies targeting vasospasm and microvascular flow (including sildenafil, pentoxifylline, diosmiplex, nifedipine, and aspirin) failed to improve symptoms. Initiation of once-daily topical sirolimus (1 mg/mL) resulted in marked pain reduction within four weeks and cessation of new ulcerations, with visible improvement and healed erosions by two months. This case supports topical sirolimus as a promising off-label option for adult CMTC with chronic ulcerative disease.

PMID 42696321
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