IPNA临床实践建议:儿童激素敏感型肾病综合征的诊断和治疗  

IPNA Clinical Practice Recommendations for the Diagnosis and Management of Children with Steroid-Sensitive Nephrotic Syndrome

作　　者：Agnes Trautmann Olivia Boyer Elisabeth Hodson Arvind Bagga Debbie S.Gipson Susan Samuel Jack Wetzels Khalid Alhas an Sushmita Banerjee Rajendra Bhimma Melvin Bonilla-Felix Francisco Cano Martin Christian Deirdre Hahn Hee Gyung Kang Koichi Nakanishi Hesham Safouh Howard Trachtman Hong Xu Wendy Cook Marina Vivarelli Dieter Hafner on behalf of the International Pediatric Nephrology Association 韩思雨(翻译) 季丽娜(翻译)[2] HAN Si-yu;JI Li-na(Department of Pediatrics,Beijing Tsinghua Changgung Hospital Affiliated to Tsinghua University,Beijing 102218,China)

机构地区：[1]不详 [2]清华大学附属北京清华长庚医院儿科,北京102218

出　　处：《中国合理用药探索》2023年第2期1-35,共35页Chinese Journal of Rational Drug Use

摘　　要：特发性肾病综合征是最常见的儿童肾小球疾病,其特征是大量蛋白尿、低蛋白血症和(或)伴有水肿,全球每年儿童患病率为(1.15~16.90)每10万例。约有85%~90%的患者在接受糖皮质激素治疗的4~6周内可达到蛋白尿完全缓解,表现为激素敏感型肾病综合征(SSNS)。约70%~80%的SSNS患者在随访期间至少复发1次,其中多达50%的患者出现频繁复发或依赖糖皮质激素以维持蛋白尿缓解。为延长复发间隔时间,激素治疗的用药剂量和用药时长始终是热点问题之一,且患者出现激素相关并发症的概率较高。临床已应用多种激素助减药物(免疫抑制药物),但在疾病治疗过程中这些药物的选择和用药时机存在明显差异。因此,需要国际循证临床实践建议(CPRs)来指导临床实践、减少实践差异。国际儿科肾脏病学会(IPNA)召集专家小组通过对12个临床相关PICO(研究对象、干预措施、对照、结果/结局)问题进行系统的文献回顾,制定了关于儿童SSNS诊断和治疗的建议和分级。对治疗转归提出了新的定义,有助于指导治疗改变,并为重要的研究问题提供了参考。Idiopathic nephrotic syndrome is the most frequent pediatric glomerular disease,affecting from 1.15 to 16.90 per 100000 children per year globally.It is characterized by massive proteinuria,hypoalbuminemia,and/or concomitant edema.Approximately 85%~90%of patients attain complete remission of proteinuria within 4~6 weeks of treatment with glucocorticoids,and therefore,have steroid-sensitive nephrotic syndrome(SSNS).Among those patients who are steroid sensitive,70%~80%will have at least one relapse during follow-up,and up to 50%of these patients will experience frequent relapses or become dependent on glucocorticoids to maintain remission.The dose and duration of steroid treatment to prolong time between relapses remains a subject of much debate,and patients continue to experience a high prevalence of steroidrelated morbidity.Various steroid-sparing immunosuppressive drugs have been used in clinical practice;however,there is marked practice variation in the selection of these drugs and timing of their introduction during the course of the disease.Therefore,international evidence-based clinical practice recommendations(CPRs)are needed to guide clinical practice and reduce practice variation.The International Pediatric Nephrology Association(IPNA)convened a team of experts including pediatric nephrologists,an adult nephrologist,and a patient representative to develop comprehensive CPRs on the diagnosis and management of SSNS in children.After performing a systematic literature review on 12 clinically relevant PICO(Patient or Population covered,Intervention,Comparator,Outcome)questions,recommendations were formulated and formally graded at several virtual consensus meetings.New definitions for treatment outcomes to help guide change of therapy and recommendations for important research questions are given.

关 键 词：激素敏感型肾病综合征 儿童 频复发型肾病综合征 激素依赖型肾病综合征 激素毒性 儿科 免疫抑制治疗 

分 类 号：R726.9[医药卫生—儿科]