Rituximab therapy for childhood onset idiopathic nephrotic syndrome: experience of a Portuguese tertiary center

Abstract

Introduction: Rituximab (RTX) is a therapeutic option in pediatric difficult-to-treat idiopathic nephrotic syndrome (NS). We aimed to assess the efficacy and safety of RTX use in these patients. Method: A retrospective study of all patients with idiopathic NS treated with RTX was conducted in a pediatric nephrology division of a tertiary hospital. Demographic, anthropometric, clinical and analytical data were collected prior to treatment and at 6, 12, and 24 months. Results: Sixteen patients were included (11 males), with a median (25th–75th percentile, P25–P75) age at diagnosis of 2 (2.0–2.8) years. Fifteen were steroid-sensitive and 1 was steroid-resistant and sensitive to cyclosporine. The median age at administration of RTX was 10 (6.3–14.0) years. Throughout a median follow-up time of 2.5 (1.0–3.0) years, 6 (37.5%) patients achieved partial remission and 7 (43.8%) had no relapses and were not taking any immunosuppressants at the 24-month follow-up visit. Regarding complications, 1 patient presented persistent hypogammaglobulinemia. Compared with the 12-month period before RTX, there was a decrease in the median number of relapses at 6 and 12 months [3 (3.0–4.0) vs 0 (0–0.8) and 0.50 (0–1.0), respectively; p = 0.001] and in the daily steroids dose (mg/kg/day) at 6, 12, and 24 months [0.29 (0.15–0.67)vs [0.10 (0.07–0.13); p = 0.001], [0.12 (0.05–0.22); p = 0.005] and [0.07(0.04–0.18); p = 0.021]], respectively. There was also a reduction in the median BMI z score at 24 months [2.11 (0.45–3.70) vs. 2.93 (2.01–3.98); p = 0.049]. Conclusion: Our results confirm the efficacy and safety of RTX use in pediatric idiopathic NS and highlight its’ potential cardiometabolic benefits.

Rituximab in idiopathic nephrotic syndrome: still waiting for stronger evidences

“…Most studies suggest that RTX is reasonably safe in the short term and relatively effective compared to other SSA, as also demonstrated by Gomes et al 1 . However, unlike other immunosuppressive therapies, there is a lack of long-term follow-up data on RTX use in INS.…”

“…Gomes et al 1 present in the current issue of BJN a report on the promising outcomes achieved with the use of rituximab (RTX) in 16 pediatric and adolescent nephrotic patients from Portugal. Their findings demonstrate a reduction in the frequency of relapses, a decrease in steroid dosage, and improvements in the patients’ bone mass index after RTX, which is consistent with existing literature.…”

Rituximab na síndrome nefrótica idiopática: ainda à espera de evidências mais robustas

“…Gomes et al 1 apresentam na edição atual do BJN um relatório sobre os desfechos promissores alcançados com o uso de rituximab (RTX) em 16 pacientes nefróticos, pediátricos e adolescentes, de Portugal. Seus achados demonstram uma redução na frequência de recidivas, uma diminuição na dosagem de esteroides e melhorias no índice de massa óssea dos pacientes após o RTX, o que é consistente com a literatura existente.…”

“…A maioria dos estudos sugere que o RTX é razoavelmente seguro em curto prazo e relativamente eficaz em comparação com outros APE, como também demonstrado por Gomes et al 1 . No entanto, ao contrário de outras terapias imunossupressoras, há uma falta de dados de acompanhamento de longo prazo sobre o uso de RTX na SNI.…”