糖皮质激素治疗先天性纯红细胞再生障碍性贫血的远期并发症

Investigation on long - term complications of glucocorticoid in treatment of Diamond - Blackfan anemia

目的总结糖皮质激素（GC）治疗先天性纯红细胞再生障碍性贫血（DBA）的远期并发症。方法收集2009年12月至2012年11月首都医科大学附属北京儿童医院诊治的应用GC治疗〉1年的17例DBA患儿资料，记录患儿一般情况、开始治疗时间、用药情况、治疗反应、不同时期身高和体质量、治疗相关不良反应等数据，采用SPSS16．0软件进行数据录入及统计学分析。结果符合纳入标准并具有详细长期随访资料的DBA共17例。58．8％（10／17例）的患儿开始GC治疗时年龄〈6个月。76．4％（13／17例）的患儿初始泼尼松用量≥2mg／（kg·d），此剂量维持治疗中位时间为2个月（1～5个月）。泼尼松用量〉0．5mg／（kg·d）的中位时间为6个月（3～48个月）。以用药时年龄是否≥6个月，将患儿分为小年龄组及大年龄组。开始治疗时，小年龄组中位身高为同年龄同性别标准身高的-1．0标准差（SD）（-3．5～1．0SD），用药0．5年后降为-3．5SD（-3．5SD～-2．0SD）；而大年龄组则分别为0．0SD（-1．5～2．0SD）和-0．5SD（-1．5～0．5SD）。治疗过程中，出现1例骨折，2例麻疹肺炎，3例反复肺炎，3例多毛，5例鹅口疮，12例向心性肥胖。结论DBA患儿长期应用GC治疗，可出现激素相关不良反应。建议临床医师尽量于患儿6个月后开始GC治疗，尽量缩短〉0．5mg／（kg·d）泼尼松口服维持时间。

Objective To collect and summarize long - term complications of glueocortieoid （GC） in treat- ment of Diamond - Blaekfan anemia （ DBA）. Methods A total of 17 DBA patients,treated with GC more than 1 year from December 2009 to November 2012 in Being Children＇s Hospital, Capital Medical University,were retrospectively investigated. The data of general information, drug treatment, treatment response, height and body mass in different the- rapy periods, and therapy related adverse reaction were collected. The data entry and the statistical analysis were per- formed using SPSS 16.0 software. Results Seventeen cases which fulfilled the research criteria were enrolled. The 58.8 percent of cases （10/17） began GC therapy from the age younger than 6 months. The 76.4 percent of the cases （14/17） started prednisone therapy with the dosage≥2 mg/（ kg · d）,and the median time of maintenance therapy with this dosage was 2 months （ 1 -5 months）. The median time of prednisone dosage greater than 0.5 mg/（ kg · d） was 6 months （3 -48 months）. Patients were divided into 2 groups at the beginning of therapy according to whether their age was younger or older than 6 mouths. The median height of younger age group was - 1.0 SD （ - 3.5 - 1.0 SD） of corresponded age - sex - standard height at the beginning of prednisone therapy, and was dropped to - 3.5 SD （ -3.5 - -2.0 SD）after half year treatment. For older age group, it was 0.0 SD （ - 1.5 -2.0 SD）and -O. 5 SD （ - 1.5 - 0. 5 SD） respectively. During the therapy, there were 1 fracture,2 measles pneumonia, 3 pneumonia, 3 hi- rsuitisms ,5 thrushes, and 12 central obesity cases. Conclusions GC related adverse reaction might appear when app- lying prednisone for the treatment of DBA in long term. It was suggested that GC therapy should start after 6 months old if possible,and the duration of 0.5 mg/（ kg · d） GC treatment should be reduced as short as possible.