Rituximab treatment in children with difficult-to-treat nephrotic syndrome

Year 2022, Volume: 47 Issue: 2, 489 - 496, 30.06.2022

Gönül Parmaksız

https://doi.org/10.17826/cumj.1038641

Abstract

Purpose: Rituximab (RTX) has been offered as rescue therapy for patients with difficult-to-treat nephrotic syndrome (frequent relapsing, steroid-dependent and steroid resistant). We aimed to assess the efficacy and long-term outcomes of RTX treatment in children with difficult nephrotic syndrome and shared our experiences
Materials and Methods: Medical records of children with difficult nephrotic syndrome who were treated with RTX were retrospectively evaluated. The relapse-free survival rate at 12 month and monitoring of B-cell depletion were assessed.
Results: In the study included 20 children of which 8 had steroid-dependent (SDNS), 6 had frequent relapsing (FRNS), and 6 had steroid-resistant nephrotic syndrome (SRNS). The median number of relapses at 1 year before and after treatment in FRNS/SDNS patients receiving RTX treatment were compared. The median number of relapses decreased from 2 (1-4) to 0 (0-1) times/year. The mean duration of the follow-up period after RTX treatment was 23 (12-59) months, and 8 patients developed relapse. Repeated doses of RTX were administered to 5 patients who relapsed after RTX treatment. In these patients, CD19+B cells re-emerged during remission, while depletion of memory B-cells remained.
Conclusion: The RTX treatment prolonged the remission time in FRNS/SDNS patients, but it was ineffective in SRNS patients. It was determined that the RTX doses can be repeated to maintain remission in these patients, and the best memory B-cell counts can help in timing the repeat doses.

Keywords

Rituximab, nephrotic syndrome, memory b-cell count, children

Supporting Institution

Baskent University Research Fund

Project Number

KA20/269

Thanks

I would like to thank Professor Dr. Zekiye Aytül Noyan at the Pediatric Nephrology Department for Dr. Turgut Noyan Research and Training Center of Başkent University. I would like to thank Professor Dr. İlknur Kozanoğlu at the Physiology Department for Dr. Turgut Noyan Research and Training Center Apheresis Unit of Başkent University. Başkent Üniversitesi Pediatrik Nefroloji Anabilim Dalı'ndan Dr. Turgut Noyan Araştırma ve Eğitim Merkezi'nden Prof. Dr. Zekiye Aytül Noyan'a teşekkür ederim. Başkent Üniversitesi Dr. Turgut Noyan Araştırma ve Eğitim Merkezi Aferez Ünitesi Fizyoloji Bölümü'nden Prof. Dr. İlknur Kozanoğlu'na teşekkür ederim.

Tedavisi zor nefrotik sendromlu çocuklarda rituksimab tedavisi

Abstract

Amaç: Rituksimab (RTX), tedavisi zor nefrotik sendromlu (sık tekrarlayan, steroide bağımlı ve steroide dirençli) hastalar için kurtarma tedavisi olarak önerilmektedir. Amacımız zor nefrotik sendromlu çocuklarda RTX tedavisinin etkinliğini ve uzun dönem sonuçlarını değerlendirmek ve deneyimlerimizi paylaşmaktır.
Gereç ve Yöntem: RTX ile tedavi edilen zor nefrotik sendromlu çocukların tıbbi kayıtları geriye dönük olarak değerlendirildi. Oniki ayda nükssüz sağkalım oranı ve B hücre deplesyonun izlemi değerlendirildi.
Bulgular: Çalışmaya 8'i steroide bağımlı (SBNS), 6'sı sık tekrarlayan (STNS) ve 6'sı steroide dirençli nefrotik sendromlu (SDNS) 20 çocuk dahil edildi. RTX tedavisi alan STNS/SBNS hastalarının tedavi öncesi ve sonrası 1 yıllık ortalama nüks sayısı karşılaştırıldı. Ortalama nüks sayısı 2 (1-4)’den 0 (0-1) kez/yıla geriledi. RTX tedavisi sonrası ortalama takip süresi 23 ay (12-59) ve 8 hastada nüks gelişti. RTX tedavisi sonrası nüks eden 5 hastaya, tekrarlayan dozlarda RTX uygulandı. Bu hastalarda, CD19+B hücreleri remisyon sırasında yeniden ortaya çıkarken, hafıza B hücrelerinin deplesyonu devam etti.
Sonuç: RTX tedavisi, STNS/SBNS hastalarında remisyon süresini uzattı, ancak SDNS hastalarında etkisiz kaldı. Bu hastalarda remisyonun idamesi için RTX dozunun tekrarlanabileceği ve tekrarlama dozlarının zamanlamasında en iyi hafıza B hücre sayısının yardımcı olabileceği saptandı.

Keywords

Rituximab, nephrotic syndrome, memory b-cell count, children

Project Number

KA20/269

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