Efficacy and Tolerance of Sirolimus (Rapamycin) for Extracranial Arteriovenous Malformations in Children and Adults

Gabeff, R.; Boccara, O.; Soupre, V.; Lorette, G; Bodemer, Christine; Herbreteau, Denis; Tavernier, Elsa; Maruani, Annabel

doi:10.2340/00015555-3273

Cited by 18 publications

(30 citation statements)

References 16 publications

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“…Treatment with sirolimus did not result in resolution in our study, as evident by persistent flow in the AVM. This is in line with the findings of other studies which showed none or only minor improvements at best with the use of sirolimus for AVMs [21,22]. However, it should be considered as a palliative treatment option for management of symptoms.…”

Section: Discussionsupporting

confidence: 90%

Peripheral Arteriovenous Malformations and Their Response to Treatment Modalities: A Single Surgeon’s Experience at Tertiary Care Hospitals

Rashid

Hussain

Malik

et al. 2021

Preprint

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Background Peripheral arteriovenous malformations (AVMs) are congenital defects resulting in abnormal connections between veins and arteries. We investigated a group of patients with peripheral AVMs to determine whether there were any gender differences in disease presentation and the response of AVMs to various modalities of treatment. Methodology The patients in this cross-sectional study were divided into two groups based on gender and their demographic data, clinical presentation at the time of diagnosis and response to treatment was assessed. Both surgical and nonsurgical treatment options were used. Nonsurgical options employed in the study included immunotherapy, embolotherapy and ultrasound-guided foam sclerotherapy (UGFS) Results Out of 43 patients, 74.4% were females with a male to female ratio of 1:3. The mean age at presentation in males was 27 years and 17 years in females. 60% of the male patients presented with high-flow AVMs while 81% of the female patients presented with low-flow lesions instead. Half of the AVMs in males were on the trunk whereas, in females, 93.9% were on the extremities. UGFS alone was used in 95.3% of patients while 32.5% of patients underwent UGFS followed by surgical excision. One patient was treated with sirolimus. In 4.6% of cases, embolization followed by surgical excision was performed. Recurrence was recorded in 20.9% of cases. Conclusions The clinical presentation of AVMs is notably different among the two genders. Sclerotherapy and embolotherapy proved to be effective treatment options. Larger studies, however, are needed to substantiate these claims. Keywords arteriovenous malformations, vascular malformations, sclerotherapy, embolotherapy, sirolimus

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Section: Discussionsupporting

confidence: 90%

Peripheral Arteriovenous Malformations and Their Response to Treatment Modalities: A Single Surgeon’s Experience at Tertiary Care Hospitals

Rashid

Hussain

Malik

et al. 2021

Preprint

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“…From the start of sirolimus treatment, 88.9% of the patients with a high‐flow vascular malformation showed improvement in their baseline symptoms, especially the size of the lesion, and the number of episodes of swelling and bleeding. Similar to results reported in previous studies, the median response time was 2 months 12 . However, some patients in our series took 6 months to respond and based on previous studies, some patients may take up to 1 year to respond 12 .…”

Section: Discussionsupporting

confidence: 90%

Efficacy and safety of oral sirolimus for high‐flow vascular malformations in real clinical practice

Durán‐Romero

Hernández‐Rodríguez

Ortiz‐Álvarez

et al. 2021

Clin Experimental Derm

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Background. Vascular malformations are a complex pathology with few treatment options. In previously published studies, oral sirolimus (rapamycin) has shown promising results in the treatment of low-flow vascular malformations, but its usefulness in high-flow vascular malformations is controversial. Aim. To evaluate the efficacy and safety of sirolimus for the treatment of high-flow vascular malformations in real-life practice. Methods. In a unit specializing in vascular anomalies, patients treated with oral sirolimus for high-flow vascular malformations were located by consulting the drug dispensations. Reviewing the electronic medical records, data on patient demographics, vascular malformation characteristics, treatments, toxicity and clinical course were collected and statistically analysed. Results. Nine patients with vascular malformations were included: eight had arteriovenous malformation and one had arteriovenous fistula. Six of these malformations were isolated while three were part of a syndrome. Sirolimus was initiated at a dosage of 1-4 mg/day to be taken as a single dose. Partial response was observed in eight of the nine patients (88.9%) with high-flow vascular malformation, while worsening was observed in the remaining patient. The treatment was well tolerated and at the most recent follow-up, five patients remained on treatment with oral sirolimus. Conclusion. Our results show that oral sirolimus is a well-tolerated therapeutic option, with an excellent safety profile, which can be useful in the long-term stabilization of patients with high-flow vascular malformations. Single-daily dosage may improve long-term adherence to treatment without worsening its effectiveness.

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“…mTOR inhibitors are recently used alternatives to surgical management of large, superficial AVMs in the pediatric population, with rapamycin being the drug of choice in many published reports [3,6]. To date, our study details the 22nd and 23rd known patients to be treated with an mTOR inhibitor for management of a cranial or facial AVM [6,[11][12][13][14][15][16]. There has been substantial variability in dosing and treatment duration across institutions, with treatment duration ranging from 3 to 24.5 months, with a median treatment duration of 12 months across all reported cases [17,18].…”

Section: Review Of Literaturementioning

confidence: 99%

“…One earlier study suggested rapamycin promoted coagulopathy in patients treated with low-flow AVMs based on correlation with significant decrease in D-dimer levels [25]. However, in a case series of both pediatric and adult patients, it was noted that children developed oral ulcers and oral mucositis following rapamycin treatment for extracranial AVMs [13]. Other authors also reported relatively infrequent and minor complications, such as transient hyperlipidemia and minor opportunistic infection [16].…”

Section: Review Of Literaturementioning

confidence: 99%

Medical Adjuvants in the Treatment of Surgically Refractory Arteriovenous Malformations of the Head and Face: Case Report and Review of Literature

et al. 2021

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Background: Arteriovenous malformations (AVMs) of the brain and face present unique challenges for clinicians. Cerebral AVMs may induce hemorrhage or form aneurysms, while facial AVMs can cause significant disfigurement and pain. Moreover, facial AVMs often draw blood supply from arteries providing critical blood flow to other important structures of the head which may make them impossible to treat curatively. Medical adjuvants may be an important consideration in the management of these patients. Summary: We conducted a systematic review of the literature to identify other instances of molecular target of rapamycin (mTOR) inhibitors used as medical adjuvants for the treatment of cranial and facial AVMs. We also present 2 cases from our own institution where patients were treated with partial embolization, followed by adjuvant therapy with rapamycin. After screening a total of 75 articles, 7 were identified which described use of rapamycin in the treatment of inoperable cranial or facial AVM. In total, 21 cases were reviewed. The median treatment duration was 12 months (3–24.5 months), and the highest recorded dose was 3.5 mg/m2. 76.2% of patients demonstrated at least a partial response to rapamycin therapy. In 2 patients treated at our institution, symptomatic and radiographic improvement were noted 6 months after initiation of therapy. Key Messages: Early results have been encouraging in a small number of patients with inoperable AVM of the head and face treated with mTOR inhibitors. Further study of medical adjuvants such as rapamycin may be worthwhile.

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Efficacy and Tolerance of Sirolimus (Rapamycin) for Extracranial Arteriovenous Malformations in Children and Adults

Cited by 18 publications

References 16 publications

Peripheral Arteriovenous Malformations and Their Response to Treatment Modalities: A Single Surgeon’s Experience at Tertiary Care Hospitals

Peripheral Arteriovenous Malformations and Their Response to Treatment Modalities: A Single Surgeon’s Experience at Tertiary Care Hospitals

Efficacy and safety of oral sirolimus for high‐flow vascular malformations in real clinical practice

Medical Adjuvants in the Treatment of Surgically Refractory Arteriovenous Malformations of the Head and Face: Case Report and Review of Literature

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