口服普萘洛尔治疗PHACES综合征4例临床特点分析并文献复习

Clinical characteristics of oral Propranolol in the treatment of 4 patients with PHACES syndrome and literature review

目的分析口服普萘洛尔治疗PHACES综合征的临床及影像学特点。方法回顾性分析2018年10月至2022年10月首都儿科研究所附属儿童医院使用普萘洛尔治疗的4例PHACES综合征患儿的临床资料。在PubMed、中国知网和万方数据库中检索相关文献,对口服普萘洛尔治疗PHACES综合征的临床研究进行总结。结果4例PHACES综合征患儿,3例面部血管瘤经治疗后均消退,1例由于就诊时间较晚,并发心血管畸形病情严重,家长放弃治疗最终死亡。共检索到口服普萘洛尔治疗PHACES综合征的临床研究7篇,其中6篇回顾性研究,1篇观察性研究。大部分研究显示,口服普萘洛尔治疗PHACES综合征耐受性良好,面部血管瘤大部分消退,影响预后的主要因素为大脑、主动脉、胸部、颈部等中等大小动脉受损程度,普萘洛尔有助于改善预后。结论PHACES综合征目前以口服普萘洛尔为一线治疗方案,多数患者对口服普萘洛尔治疗耐受性良好。

Objective To summarize the clinical data and imaging characteristics of patients with PHACES syndrome treated with oral Propranolol.Methods The clinical data of 4 cases of PHACES syndrome treated with oral Propranolol in Children′s Hospital,Capital Institute of Pediatrics from October 2018 to October 2022 were retrospectively analyzed.Relevant studies reporting the treatment of PHACES with Propranolol were retrieved in PubMed,China National Knowledge Infrastructure(CNKI)and Wanfang Data.Results Three cases of the 4 patients with PHACES syndrome treated with Propranolol obtained relieved symptoms of facial hemangioma,and 1 case died due to late treatment,complication of severe cardiovascular malformation,and treatment abandonment by parents.A total of 7 clinical studies on the use of Propranolol in the treatment of PHACES were retrieved,including 6 retrospective studies and 1 observational study.Most studies have shown that Propranolol is well tolerated in the treatment of PHACES syndrome,and most of cases have relieved facial hemangioma.The main factors affecting the prognosis are the degree of damage to middle and small arteries such at brain,aorta,chest and neck.Propranolol treatment can improve the prognosis.Conclusions Oral Propranolol is currently the first-line treatment for PHACES syndrome,and most patients tolerate oral Propranolol well.