塞来昔布治疗儿童难治性淋巴管畸形的临床效果

Efficacy of celecoxib in the treatment of refractory lymphatic anomalies

目的:探讨口服塞来昔布治疗儿童难治性淋巴管畸形(LM)的临床效果。方法:回顾性分析2020年9月至2022年1月在南京医科大学附属儿童医院采用塞来昔布治疗的难治性LM患儿的临床资料。在其他方法治疗效果不佳或伴有疼痛的情况下口服塞来昔布胶囊,每天5 mg/kg,分2次口服,疗程为6个月,治疗期间观察药物不良反应。在用药治疗前和治疗后6个月行磁共振(MRI)检查,评估病灶大小的变化;通过儿童生活质量量表(PedsQL)4.0进行评分。结合MRI检查结果及PedsQL评分对药物治疗效果进行评价,分为治愈、改善、稳定和无效。使用SPSS 21.0软件进行数据分析,用药前后病灶体积及PedsQL评分以M(Q1,Q3)表示,并采用Wilcoxon符号秩和检验进行比较,P<0.05表示差异有统计学意义。结果:共纳入12例患儿,男7例,女5例,年龄1岁5个月至9岁9个月。12例中2例临床症状得到改善,8例病情保持稳定,2例无效。12例患儿用药前病灶体积为59.13(38.19,107.15)cm 3,用药后6个月为66.08(37.37,119.98)cm 3,用药前后体积比较差异无统计学意义(Z=0.40,P=0.686);用药前PedsQL评分为30.5(29.0,32.0)分,用药后6个月为29.0(28.0,30.0)分,用药前后PedsQL评分比较差异无统计学意义(Z=1.73,P=0.084)。其中仅有1例患儿出现了短期的睡眠质量下降,无其他不良反应发生。结论:塞来昔布无法使LM病灶缩小,但可能有助于稳定难治性LM患儿的病情,并改善其症状,提高生活质量,可作为辅助治疗方法。

Objective To determine the efficacy of treatment of refractory lymphatic anomalies with celecoxib.Methods The authors retrospectively analyzed the characteristics,treatments and complications of patients treated with celecoxib in Children’s Hospital of Nanjing Medical University from September 2020 to January 2022.Treatment was started after poor response to other method or pain,which consisted of a continuous dosing schedule of oral celecoxib at 5 mg.kg-1.d-1,twice daily,for 6 months.Efficacy and safety were evaluated according to radiologic assessment,quality of life and incidence of adverse events.The efficacy was divided into complete response,partial response,stablizing response and non-responsive.SPSS 21.0 was used for data analysis.The data were expressed by M(Q1,Q3).Wilcoxon test was used to compare the lesion volume and PedsQL score before and after treatment.P<0.05 indicated that the difference was statistically significant.Results A total of 12 patients with lymphatic malformation were enrolled,including 7 males and 5 females aging from 1 year and 5 months to 9 years and 9 months.Eight patients kept stable disease while two patients achieve partial response and two patients presented progressive disease.The median volume of lesions before treatment was 59.13(38.19,107.15)cm3 and was 66.08(37.37,119.98)cm3 after the treatment.There was no significant difference in volume changes(Z=0.40,P=0.686).The PedsQL score was 30.5(29.0,32.0)before and 29.0(28.0,30.0)after the treatment.There was no significant difference in the change of PedsQL score(Z=1.73,P=0.084).The side effects occurred in one patient as poor sleeping.Conclusion Celecoxib can not reduce the lesion but may be helpful for improving life quality in lymphatic malformations.