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利妥昔单抗、环磷酰胺和他克莫司作为儿童复杂性复发/类固醇依赖型肾病综合征的一线类固醇替代药物的比较:对健康相关生活质量的评估

Comparison of rituximab, cyclophosphamide, and tacrolimus as first steroid-sparing agents for complicated relapsing/steroid-dependent nephrotic syndrome in children: an evaluation of the health-related quality of life.

作者信息

Wang Li, Zhu Jialiang, Xia Mingyun, Hua Ran, Deng Fang

机构信息

Children's Hospital of Anhui Medical University, Anhui, China.

The First Affiliated Hospital of Anhui Medical University, Anhui, China.

出版信息

Arch Med Sci. 2022 Jan 14;18(1):275-278. doi: 10.5114/aoms/145587. eCollection 2022.

DOI:10.5114/aoms/145587
PMID:35154548
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8826811/
Abstract

INTRODUCTION

Frequently-relapsing/steroid-dependent nephrotic syndrome (FRSDNS) leads to steroid toxicity impairing quality of life (QOL), thus prompting the use of steroid-sparing drugs.

METHODS

51 FRSDNS children not previously treated with steroid-sparing agents were randomized to receive rituximab, cyclophosphamide, or tacrolimus. Clinical findings and QOL were evaluated before and after treatment.

RESULTS

The mean relapse rate in all groups declined six months after treatment, however, 1-year relapse-free survival rate, number of relapses, and cumulative prednisolone dosage were lower with rituximab than with tacrolimus and cyclophosphamide. Cyclophosphamide group had twice frequency of infections compared to the other groups. At 1 year after treatment, total scores showed greater improvement with rituximab.

CONCLUSIONS

As first-line steroid-sparing agent, rituximab is more effective and safer than cyclophosphamide and tacrolimus in FRSDNS, and improve QOL.

摘要

引言

频繁复发/依赖类固醇的肾病综合征(FRSDNS)会导致类固醇毒性,损害生活质量(QOL),从而促使人们使用类固醇节约药物。

方法

51名此前未接受过类固醇节约药物治疗的FRSDNS儿童被随机分为接受利妥昔单抗、环磷酰胺或他克莫司治疗。在治疗前后评估临床结果和生活质量。

结果

治疗6个月后,所有组的平均复发率均下降,然而,利妥昔单抗治疗组的1年无复发生存率、复发次数和累积泼尼松龙剂量低于他克莫司和环磷酰胺治疗组。与其他组相比,环磷酰胺组的感染频率是其他组的两倍。治疗1年后,利妥昔单抗治疗组的总评分改善更大。

结论

作为一线类固醇节约药物,利妥昔单抗在FRSDNS治疗中比环磷酰胺和他克莫司更有效、更安全,并且能改善生活质量。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7b1f/8826811/c933e7e7f813/AMS-18-1-145587-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7b1f/8826811/c933e7e7f813/AMS-18-1-145587-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7b1f/8826811/c933e7e7f813/AMS-18-1-145587-g001.jpg

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The effect of rituximab on the quality of life of children with refractory nephrotic syndrome.利妥昔单抗对难治性肾病综合征患儿生活质量的影响。
Pediatr Int. 2022 Jan;64(1):e14725. doi: 10.1111/ped.14725. Epub 2021 Dec 2.
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Single Dose of Rituximab in Children with Steroid-Dependent / Frequently Relapsing Nephrotic Syndrome, Clinical Efficacy and Evaluation of Health-Related Quality of Life.单剂量利妥昔单抗治疗激素依赖/频繁复发型肾病综合征患儿的临床疗效及健康相关生活质量评估
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Rituximab vs Low-Dose Mycophenolate Mofetil in Recurrence of Steroid-Dependent Nephrotic Syndrome in Children and Young Adults: A Randomized Clinical Trial.
Prolonged remission after cyclophosphamide or tacrolimus treatment in childhood nephrotic syndrome: a cohort study.
环磷酰胺或他克莫司治疗儿童肾病综合征后的长期缓解:一项队列研究
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Non-corticosteroid immunosuppressive medications for steroid-sensitive nephrotic syndrome in children.儿童类固醇敏感型肾病综合征的非皮质类固醇类免疫抑制药物。
Cochrane Database Syst Rev. 2024 Nov 8;11(11):CD002290. doi: 10.1002/14651858.CD002290.pub6.
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Rituximab versus tacrolimus as corticosteroid-sparing therapy for children with steroid-dependent nephrotic syndrome: A systematic review and meta-analysis of randomized and nonrandomized controlled trials.利妥昔单抗与他克莫司作为激素依赖型肾病综合征儿童的糖皮质激素节省疗法:随机对照试验和非随机对照试验的系统评价与荟萃分析
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An update on the incidence, risk factors and mechanisms of rituximab-associated neutropenia.利妥昔单抗相关中性粒细胞减少症的发病率、危险因素及机制的最新进展。
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Predictive factors of long-term disease remission after rituximab administration in patients with childhood-onset complicated steroid-dependent nephrotic syndrome: a single-center retrospective study.利妥昔单抗治疗儿童起病的激素依赖型肾病综合征后长期缓解的预测因素:一项单中心回顾性研究。
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利妥昔单抗与低剂量霉酚酸酯治疗儿童和青年成人激素依赖型肾病综合征复发的随机临床试验
JAMA Pediatr. 2021 Jun 1;175(6):631-632. doi: 10.1001/jamapediatrics.2020.6150.
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Rituximab in childhood steroid-sensitive nephrotic syndrome: are multiple subsequent courses safe and effective?利妥昔单抗治疗儿童激素敏感性肾病综合征:多次后续治疗安全有效吗?
Arch Dis Child. 2021 Jul 19;106(8):815-818. doi: 10.1136/archdischild-2020-319609.
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Retrospective single-arm cohort study of steroid-dependent minimal change nephrotic syndrome treated with very low-dose rituximab.极低剂量利妥昔单抗治疗激素依赖型微小病变肾病综合征的回顾性单臂队列研究
Clin Nephrol. 2021 Jan;95(1):29-36. doi: 10.5414/CN110245.
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Impact of steroids and steroid-sparing agents on quality of life in children with nephrotic syndrome.类固醇和类固醇节约剂对肾病综合征患儿生活质量的影响。
Pediatr Nephrol. 2021 Jan;36(1):93-102. doi: 10.1007/s00467-020-04684-3. Epub 2020 Jul 15.
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Rituximab versus cyclophosphamide as first steroid-sparing agent in childhood frequently relapsing and steroid-dependent nephrotic syndrome.利妥昔单抗与环磷酰胺作为儿童频复发型和激素依赖型肾病综合征的一线激素保肾药物。
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Non-corticosteroid immunosuppressive medications for steroid-sensitive nephrotic syndrome in children.用于儿童激素敏感型肾病综合征的非皮质类固醇免疫抑制药物。
Cochrane Database Syst Rev. 2020 Apr 16;4(4):CD002290. doi: 10.1002/14651858.CD002290.pub5.
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Rituximab for very low dose steroid-dependent nephrotic syndrome in children: a randomized controlled study.利妥昔单抗治疗儿童极低剂量激素依赖型肾病综合征:一项随机对照研究。
Pediatr Nephrol. 2020 Aug;35(8):1437-1444. doi: 10.1007/s00467-020-04540-4. Epub 2020 Mar 30.
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Both the rituximab dose and maintenance immunosuppression in steroid-dependent/frequently-relapsing nephrotic syndrome have important effects on outcomes.利妥昔单抗剂量和维持性免疫抑制在激素依赖型/频繁复发型肾病综合征中对治疗结果均有重要影响。
Kidney Int. 2020 Feb;97(2):393-401. doi: 10.1016/j.kint.2019.09.033. Epub 2019 Oct 31.