Rituximab for Children With Difficult-to-Treat Nephrotic Syndrome: Its Effects on Disease Progression and Growth

Frontiers in Pediatrics

article

Rezan Topaloğlu¹  Bora Gülhan¹  Kübra Çelegen¹  Mihriban İnözü¹  Mutlu Hayran²  Ali Düzova¹  Fatih Ozaltin¹ 

[1] Division of Pediatric Nephrology, Department of Pediatrics, School of Medicine, Hacettepe University;Department of Preventive Oncology, School of Medicine, Hacettepe University;Nephrogenetics Laboratory, School of Medicine, Hacettepe University;Center for Biobanking and Genomics, School of Medicine, Hacettepe University

关键词: rituximab;    nephrotic syndrome;    steroid;    cyclosporine;    growth;   

DOI  :  10.3389/fped.2019.00313

学科分类：社会科学、人文和艺术（综合）

来源: Frontiers

【 摘 要 】

Background: Since the early 2000s rituximab (RTX) has been thought of as an alternative treatment for steroid-sensitive nephrotic syndrome (SSNS) and steroid-resistant nephrotic syndrome (SRNS). Objective: This study aimed to determine the effects of RTX treatment on disease outcome and growth in pediatric SSNS and SRNS patients. Materials and Methods: The medical records of pediatric SSNS and SRNS patients that began RTX treatment at the mean age of 10.8 ± 5.1 years between 2009 and 2017 were retrospectively reviewed. Additionally, the effect of RTX on growth was evaluated based on patient height, weight, and BMI z scores. Results: The study included 41 children, of which 21 had SSNS and 20 had SRNS. Mean age at diagnosis of NS was 5.8 ± 4.7 years. Mean duration of post-RTX treatment follow-up was 2.3 ± 1.6 years. Among the SSNS patients, 6 and 11 patients were steroid free and calcineurin inhibitor free at the last follow-up visit, respectively. The 1-year cumulative steroid and calcineurin inhibitor doses both decreased after RTX treatment, as compared to before RTX ( P = 0.001 and P = 0.015, respectively). The median height z -score at the time of RTX initiation was −1.2 and the median height z -score at the last follow-up visit was −0.6 ( P = 0.044). The median BMI z -score decreased from 1.6 (IQR; 0.9–3.0) at the time RTX was initiated to 1.1 IQR; [(−0.7)−2.5] at the last follow-up visit ( P = 0.007). At the last follow-up visit 4 SRNS patients had complete remission and 4 had partial remission. The 1-year cumulative steroid dosage in the SRNS patients decreased significantly after RTX, as compared to before RTX ( P = 0.001). The median height z -score at the time of RTX initiation was −0.8 and the median height z -score at the last follow-up visit was −0.7 ( P = 0.81). The median BMI z -score decreased from 0.3 at the time RTX was initiated to −0.1 at the last follow-up visit ( P = 0.11). Conclusion: RTX has a more positive effect on disease outcome and growth in SSNS patients than in those with SRNS.

【 授权许可】

CC BY