A prospective multicenter study of sirolimus for complicated vascular anomalies

Ji, Yi; Chen, Siyuan; Yang, Kaiying; Zhou, Jiangyuan; Zhang, Xuepeng; Jiang, Xian; Xu, Xuewen; Lu, Guoyan; Qiu, Liqing; Kong, Feiteng; Zhang, Yongbo

doi:10.1016/j.jvs.2021.04.071

Cited by 39 publications

(47 citation statements)

References 31 publications

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“…After a careful full-text review, 12 studies were eligible for the final data extraction and analysis. Finally, nine studies [ 22 , 23 , 24 , 25 , 29 , 30 , 31 , 32 , 33 ] were included in the analyses. The remaining three studies were excluded, including a prospective cohort study and two randomized controlled trials.…”

Section: Resultsmentioning

confidence: 99%

“…The characteristics of the subsumed studies are summarized in Table 1 . In total, the 9 studies consisted of 575 patients, and there were 7 prospective studies [ 22 , 23 , 24 , 29 , 30 , 31 , 32 ] and 2 randomized controlled trials [ 25 , 33 ]. Of these studies, four were related to tuberous sclerosis complex [ 25 , 31 , 32 , 35 ], two were related to lymphatic anomalies [ 31 , 32 ], two were related to vascular anomalies [ 22 , 25 ] and one was related to nephrotic syndrome [ 24 ].…”

Section: Resultsmentioning

confidence: 99%

“…In the protocol, the age of the patients was limited to under 14 years of age because a prospective study with a large sample size defined the age as 0–14 years [ 22 ]. However, during the literature search, it was found that the age boundary of many prospective studies was 17 or 18 years old [ 23 , 24 , 25 ].…”

Section: Methodsmentioning

confidence: 99%

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Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review

Zhang

et al. 2022

Children

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Sirolimus, a mammalian target of rapamycin inhibitor, has been widely used in pediatric patients, but the safety of sirolimus in pediatric patients has not been well determined. Objective: The objective of this study was to systematically evaluate prospective studies reporting the safety of sirolimus in the treatment of childhood diseases. Methods: The following data were extracted in a standardized manner: study design, demographic characteristics, intervention, and safety outcomes. Results: In total, 9 studies were included, encompassing 575 patients who received oral sirolimus for at least 6 months. Various adverse events occurred. The most common adverse event was oral mucositis (8.2%, 95% CI: 0.054 to 0.110). Through comparative analysis of the subgroups based on the targeted concentration range, we discovered that many adverse events were significantly higher in the high concentration group (≥10 ng/mL) than in the low concentration group (<10 ng/mL) (p < 0.01). More interestingly, we found that oral mucositis was more frequently reported in children with vascular anomalies than tuberous sclerosis complex. Conclusions: This study shows that oral sirolimus in the treatment of childhood diseases is safe and reliable. However, sirolimus treatment in the pediatric population should be strictly monitored to reduce the occurrence of serious or fatal adverse events.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

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Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review

Zhang

et al. 2022

Children

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“…9,10 However, reports evaluating the efficacy of sirolimus in high-flow malformations are scarce, being limited to isolated cases or small series of patients, most of which showed poor results. 9,[11][12][13] The aim of this study was to contribute our experience in using sirolimus to treat complex high-flow vascular malformations in real clinical practice, providing data on dosage, efficacy and safety. To our knowledge, this is the second largest case series of patients treated with sirolimus for high-flow vascular malformations.…”

Section: Introductionmentioning

confidence: 99%

“…Recently, some prospective clinical trials assessing the efficacy and safety of sirolimus showed promising results in low‐flow vascular malformations 9,10 . However, reports evaluating the efficacy of sirolimus in high‐flow malformations are scarce, being limited to isolated cases or small series of patients, most of which showed poor results 9,11–13 …”

Section: Introductionmentioning

confidence: 99%

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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Long‐term outcomes of sirolimus treatment for kaposiform hemangioendothelioma: Continuing successes and ongoing challenges

Zhou

Qiu

et al. 2023

Intl Journal of Cancer

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Treatment with sirolimus, an inhibitor of the mammalian target of rapamycin pathway, has improved the prognosis of patients with kaposiform hemangioendothelioma (KHE). However, the efficacy, durability and tolerability of long-term sirolimus treatment in patients with KHE have not been well elucidated. We performed efficacy and safety assessments based on more than 4.5 years of follow-up in patients receiving sirolimus therapy for KHE. One hundred sixtyseven patients were analyzed, including 102 (61.1%) patients with the Kasabach-Merritt phenomenon (KMP). Follow-up was conducted after a median of 56.0 months. A total of 154 (92.2%) patients had a durable response to sirolimus treatment. No difference in durable response was found between patients without KMP and patients with KMP (95.4% vs 90.2%; difference, 5.2%; 95% confidence interval [CI], À4.0% to 13.1%). Rebound growth occurred in 17.3% of patients upon sirolimus discontinuation. Early treatment discontinuation (odds ratio [OR]: 3.103; 95% CI: 1.529-6.299; P = .002) and mixed lesion type (OR: 2.271; 95% CI: 0.901-5.727; P = .047) were associated with tumor rebound growth. No KHE-related deaths occurred in this cohort. At the last follow-up, approximately 17.4% of patients had active disease and/or changes in body structures to a variable extent. Serious adverse events occurred most commonly during the first year of sirolimus therapy. Follow-up of almost 4.5 years demonstrated that the efficacy of sirolimus persisted over time and that long-term treatment Abbreviations: CI, confidence interval; KHE, kaposiform hemangioendothelioma; KMP, Kasabach-Merritt phenomenon; MRI, magnetic resonance imaging; mTOR, mammalian target of rapamycin; OR, odds ratio; ROM, range of motion.Jiangyuan Zhou, Yanan Li and Tong Qiu have contributed equally to this work.

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A prospective multicenter study of sirolimus for complicated vascular anomalies

Cited by 39 publications

References 31 publications

Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review

Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review

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

Long‐term outcomes of sirolimus treatment for kaposiform hemangioendothelioma: Continuing successes and ongoing challenges

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