曲前列尼尔治疗重症特发性肺动脉高压患儿的短期临床效果和安全性

Short-term clinical effect and safety of treprostinil on the treatment of severe pediatric idiopathic pulmonary arterial hypertension

目的评价曲前列尼尔治疗重症特发性肺动脉高压(IPAH)患儿的短期临床效果和安全性。方法收集2018年5月至2020年12月就诊于首都医科大学附属北京安贞医院小儿心脏中心年龄<18周岁的16例短期持续皮下泵入曲前列尼尔治疗的重症IPAH患儿的临床资料进行回顾性分析。收集其病史、临床症状、治疗策略及随访数据,比较随访及基线时的相关临床指标,观察不良反应发生情况。结果入组患儿年龄为(9±5)岁,纽约心脏病协会(NYHA)心功能分级Ⅲ级11例、Ⅳ级5例,在为期(68±34)d的随访中,患儿心功能明显改善,Ⅱ级15例、Ⅲ级1例。患儿随访时B型脑钠肽、三尖瓣反流最大峰值速度、三尖瓣反流压差、主肺动脉内径与主动脉根部内径比值、右心室前后径与左心室舒张末期内径比值均较基线时明显下降[(135±98)ng/L比(380±353)ng/L、(426±58)cm/s比(467±49)cm/s、(73±19)mm Hg(1 mm Hg=0.133 k Pa)比(88±19)mm Hg、(1.32±0.22)比(1.62±0.47)、(0.6±0.3)比(0.9±0.6)],差异均有统计学意义(均P<0.05)。起始用药5 d内,2例患儿诉输注部位疼痛,3例诉间断头痛,2例表现为面部潮红,调整用药剂量及对症支持治疗后均能耐受,无患儿因无法耐受而终止治疗。结论曲前列尼尔治疗重症IPAH患儿安全有效,且耐受性良好。

Objective To evaluate the short-term clinical effect and safety of treprostinil on the treatment of severe pediatric idiopathic pulmonary arterial hypertension(IPAH).Methods From May 2018 to December2020,clinical data of 16 children(<18 years old)with severe IPAH receiving short-term continuous subcutaneous pumping of treprostinil admitted to Pediatric Heart Center,Beijing Anzhen Hospital,Capital Medical University were retrospectively analyzed.The medical history,clinical symptoms,treatment strategies and follow-up data were collected,the relevant clinical indicators at follow-up and baseline were compared,and adverse reactions were observed.Results The age of children was(9±5)years old.There were 11 cases of New York Heart Association(NYHA)gradeⅢand 5 cases of gradeⅣ.During the follow-up of(68±34)d,the cardiac function of children was significantly improved,including 15 cases of NYHA gradeⅡand 1 case of gradeⅢ.Brain natriuretic peptide,maximum peak velocity of tricuspid regurgitation,differential pressure of tricuspid regurgitation,ratio of aortopulmonary artery inside diameter to aortic root inside diameter,ratio of right ventricular anteroposterior diameter to left ventricular end diastolic diameter of children at follow-up were significantly lower than those at baseline[(135±98)ng/L vs(380±353)ng/L,(426±58)cm/s vs(467±49)cm/s,(73±19)mm Hg vs(88±19)mm Hg,(1.32±0.22)vs(1.62±0.47),(0.6±0.3)vs(0.9±0.6)](all P<0.05).Within 5 d of initial medication,2 cases complained about pain in infusion site,3 cases complained about head pain,and 2 cases presented facial flushing.After adjusting the dosage of medication and supportive treatment,children were able to tolerate,and no child discontinued treatment due to intolerance.Conclusion Treprostinil is safe,effective and well tolerated in children with severe IPAH.