Shu Ye,Tan Xin,Liu Xiangyu,et al.Study of oral sirolimus in the treatment of vascular anomaliesin infants and children[J].Journal of Clinical Pediatric Surgery,,20():1064-1069.[doi:10.12260/lcxewkzz.2021.11.013]
Study of oral sirolimus in the treatment of vascular anomaliesin infants and children
- CLC:
- R722;R986
- Abstract:
- Objective To investigate the efficacy of oral sirolimus in the treatment of vascular anomalies in infants and children. Methods From December 2019 to December 2020, 14 infant and children with vascular anomalies were hospitalized in the dermatology department of Hunan Children’s Hospital. Among them, there were 11 males and 3 females, aged from 6 months to 10 years. There were 4 cases of Kaposi hemangioendothelioma, 2 cases of Sturge Weber syndrome, 3 cases of venous malformation, 3 cases of mixed vascular malformation, 1 case of CLOVES syndrome and 1 case of tuberous sclerosis. Sirolimus 1-1.5 mg/m2 was given twice a day at an interval of 12 hours. The adverse reactions were monitored during hospitalization, and the drugs were taken orally after discharge. Regular outpatient examinations were conducted to detect the children’s blood and urine routine, liver and kidney function, blood lipids, coagulation function and blood drug concentration. Results All children were treated with sirolimus for 3-7 months. After the whole course of treatment, they were followed up for 1-3 months. Four patients with Kaposi hemangioendothelioma were all effective, two patients with Sturge Weber syndrome, one partially effective and one ineffective. Of the 3 patients with venous malformation, one was partially effective and two were ineffective. In 3 cases of mixed vascular malformation, one case was partially effective and two cases were ineffective. Patients with CLOVES syndrome were partially effective, but patients with tuberous sclerosis were ineffective. No obvious adverse reactions occurred in all children. Conclusion Oral sirolimus has good efficacy in the treatment of Kaposi hemangioendothelioma in infants and children. There was no obvious adverse reaction. The efficacy of sirolimus in the treatment of vascular malformations is uncertain and needs further study.
References:
1 孔亮亮, 韩涛, 高庆文, 等. 口服西罗莫司治疗难治性脉管性疾病的有效性与安全性[J].中华整形外科杂志, 2020, 36(5):487-493.DOI:10.3760/cma.j.cn114453-20200316-00159. Kong LL, Han T, Gao QW, et al. Efficacy and safety of oral sirolimus in the treatment of refractory vascular anomalies[J].Chin J Plast Surg, 2020, 36(5):487-493.DOI:10.3760/cma.j.cn114453-20200316-00159.
2 赵亚梅, 高怡瑾, 周莺, 等. 儿童卡梅现象13例回顾性分析并文献复习[J].临床儿科杂志, 2017, 35(6):458-466.DOI:10.3969/j.issn.1000-3606.2017.06.015. Zhao YM, Gao YJ, Zhou Y, et al. Retrospective analysis in 13 children with Kasabach-Merritt phenomenon and review of literature[J].J Clin Pediatr, 2017, 35(6):458-466.DOI:10.3969/j.issn.1000-3606.2017.06.015.
3 代诗懿, 彭素华, 陈思源, 等. 卡波西样血管内皮瘤的研究进展[J].临床小儿外科杂志, 2020, 19(6):544-547.DOI:10.3969/j.issn.1671-6353.2020.06.016. Dai SY, Peng SH, Chen SY, et al. Recent advances in researches and treatments of kaposiform hemangioendothelioma[J].J Clin Ped Sur, 2020, 19(6):544-547.DOI:10.3969/j.issn.1671-6353.2020.06.016.
4 中华医学会整形外科分会血管瘤和脉管畸形学组. 血管瘤和脉管畸形的诊断及治疗指南(2019 版)[J]. 组织工程与重建外科杂志, 2019, 15(5):277-317.DOI:10.3969/j.issn.1673-0364.2019.05.001. Group of Angiomas and Vascular Malformations, Branch of Reconstructive Surgery, Chinese Medical Association:Guideline of Diagnosing & Treating Angiomas and Vascular Malformations (2019 Edition)[J].Journal of Tissue Engineering and Reconstructive Surgery, 2019, 15(5):277-317.DOI:10.3969/j.issn.1673-0364.2019.05.001.
5 刘文英.脉管异常类疾病的分类及相关临床问题[J].临床小儿外科杂志, 2019, 18(8):621-625.DOI:10.3969/j.issn.1671-6353.2019.08.001. Liu WY.Classifications and clinical managements of vascular abnormalities[J].J Clin Ped Sur, 2019, 18(8):621-625.DOI:10.3969/j.issn.1671-6353.2019.08.001.
6 Hammill AM, Wentzel M, Gupta A, et al. Sirolimus for the treatment of complicated vascular anomalies in children[J].Pediatr Blood Cancer, 2011, 57(6):1018-1024.DOI:10.1002/pbc.23124.
7 ?zg?nenel B, Martin A.Low-dose sirolimus controls recurrent iron deficiency in a patient with blue rubber bleb nevus syndrome[J].Pediatr Blood Cancer, 2015, 62(11):2054-2055.DOI:10.1002/pbc.25590.
8 Hammer J, Seront E, Duez S, et al. Sirolimus is efficacious in treatment for extensive and/or complex slow-flow vascular malformations:a monocentric prospective phase Ⅱ study[J].Orphanet J Rare Dis, 2018, 13(1):191.DOI:10.1186/s13023-018-0934-z.
9 Nozawa A, Ozeki M, Yasue S, et al. Immunologic Effects of Sirolimus in Patients With Vascular Anomalies[J].J Pediatr Hematol Oncol, 2020, 42(5):e355-e360.DOI:10.1097/MPH.0000000000001650.
10 Russell TB, Rinker EK, Dillingham CS, et al. Pneumocystis Jirovecii Pneumonia during sirolimus therapy for kaposiform hemangioendothelioma[J].Pediatrics, 2018, 141(Suppl5):S421-S424.DOI:10.1542/peds.2017-1044.
11 Ying H, Qiao C, Yang X, et al. A case report of 2 sirolimus-related deaths among infants with kaposiform hemangioendotheliomas[J].Pediatrics, 2018, 141(Suppl5):S425-S429.DOI:10.1542/peds.2016-2919.
12 Ji Y, Yang K, Chen S, et al. Musculoskeletal complication in kaposiform hemangioendothelioma without Kasabach-Merritt phenomenon:clinical characteristics and management[J].Cancer ManagRes, 2018, 10(9):3325-3331.DOI:10.2147/CMAR.S171223.eCollection 2018.
13 Ji Y, Chen S, Li L, et al. Kaposiform hemangioendothelioma without cutaneous involvement[J].J Cancer Res Clin Oncol, 2018, 144(12):2475-2484.DOI:10.1007/s00432-018-2759-5.
14 Schmid I, Klenk AK, Sparber-Sauer M, et al. Kaposiform hemangioendothelioma in children:a benign vascular tumor with multiple treatment options[J].World J Pediatr, 2018, 14(4):322-329.DOI:10.1007/s12519-018-0171-5.
15 Greveling K, Prens EP, van Doorn MB.Treatment of port wine stains using pulsed dye laser, erbium yag laser, and topical rapamycin (sirolimus)-a randomized controlled trial[J].Lasers Surg Med, 2017, 49(1):104-109.DOI:10.1002/lsm.22548.
16 Marqués L, Nú?ez-Córdoba JM, Aguado L, et al. Topical rapamycin combined with pulsed dye laser in the treatment of capillary vascular malformations in Sturge-Weber syndrome:phase Ⅱ, randomized, double-blind, intraindividual placebo-controlled clinical trial[J].J Am Acad Dermatol, 2015, 72(1):151-158.e1.DOI:10.1016/j.jaad.2014.10.011.
17 Freixo C, Ferreira V, Martins J, et al. Efficacy and safety of sirolimus in the treatment of vascular anomalies:A systematic review[J].J Vasc Surg, 2020, 71(1):318-327.DOI:10.1016/j.jvs.2019.06.217.
Memo
收稿日期:2020-03-25。
基金项目:湖南省科技创新计划项目(编号:2018SK50408)
通讯作者:周斌,Email:zhoubin957@sina.com