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Background <p>The present study was conducted as a randomized controlled trial to investigate the combined impact of rituximab and target care on efficacy, quality of life, adverse reactions, and recurrence rate among children with nephrotic syndrome.</p> Methods <p>This was a prospective, randomized, open-label, controlled trial conducted at a single tertiary children’s hospital. Ninety-one pediatric patients with nephrotic syndrome were enrolled between January 2021 and June 2023. Patients were randomly assigned (1:1) to the study group (receiving initial corticosteroid therapy followed by rituximab combined with target care, <i>n</i> = 46) or the control group (receiving initial corticosteroid therapy followed by oral tacrolimus capsules combined with target care, <i>n</i> = 45). The primary outcome was clinical efficacy at 3 months. Secondary outcomes included quality of life (IS-LQ questionnaire at baseline and 3 months), adverse reactions, 6-month recurrence rate, and CD19 + B-cell counts (study group only). Analysis was by intention-to-treat.</p> Results <p>Of 115 children assessed for eligibility, 91 were randomized. All randomized patients completed the 3-month efficacy assessment and were included in the analysis. After treatment, the study group was associated with significantly better efficacy (45/46, 97.83%) than that of the control group (38/45, 84.44%) (Risk Difference 13.39%, 95% CI 1.63–25.15%; <i>P</i> = 0.029). After treatment, the quality of life in both groups was significantly improved (<i>P</i> &lt; 0.001 compared to baseline for each group). The quality of life in the study group was slightly increased as compared to the control group, but the difference was not statistically significant (<i>P</i> &gt; 0.05). All patients in the study group achieved adequate B-cell depletion. The recurrence rate at 6 months in the study group (18/46, 39.13%) was lower as compared to the control group (30/45, 66.67%) (Risk Difference − 27.54%, 95% CI − 47.00% to − 8.08%; <i>P</i> = 0.009). The incidence of adverse reactions, including mild transient neutropenia, was comparable between the two groups (<i>P</i> &gt; 0.05).</p> Conclusion <p>The rituximab-based regimen combined with target care exhibited beneficial outcomes by effectively improving clinical efficacy and reducing recurrence for children with nephrotic syndrome, with a safety profile comparable to a tacrolimus-based regimen over the study period. Quality of life improved in both groups. Further studies with longer follow-up are needed to assess long-term outcomes and safety.</p> Trial registration <p>ClinicalTrials.gov, NCT07049172.</p>

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Combined impact of rituximab and target care on efficacy, quality of life, adverse reactions and recurrence rate in children with nephrotic syndrome: a randomized controlled trial

  • 期刊论文

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  • 发表日期：

    2025年9月24日

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  • Lanfen He,
  • Huazhen Mei,
  • Yan Gu,
  • Zeng Liu,
  • Lian Yang,
  • Xiaolu Li,
  • Xiaofeng Wang,
  • Ting Xu

摘要

Background

The present study was conducted as a randomized controlled trial to investigate the combined impact of rituximab and target care on efficacy, quality of life, adverse reactions, and recurrence rate among children with nephrotic syndrome.

Methods

This was a prospective, randomized, open-label, controlled trial conducted at a single tertiary children’s hospital. Ninety-one pediatric patients with nephrotic syndrome were enrolled between January 2021 and June 2023. Patients were randomly assigned (1:1) to the study group (receiving initial corticosteroid therapy followed by rituximab combined with target care, n = 46) or the control group (receiving initial corticosteroid therapy followed by oral tacrolimus capsules combined with target care, n = 45). The primary outcome was clinical efficacy at 3 months. Secondary outcomes included quality of life (IS-LQ questionnaire at baseline and 3 months), adverse reactions, 6-month recurrence rate, and CD19 + B-cell counts (study group only). Analysis was by intention-to-treat.

Results

Of 115 children assessed for eligibility, 91 were randomized. All randomized patients completed the 3-month efficacy assessment and were included in the analysis. After treatment, the study group was associated with significantly better efficacy (45/46, 97.83%) than that of the control group (38/45, 84.44%) (Risk Difference 13.39%, 95% CI 1.63–25.15%; P = 0.029). After treatment, the quality of life in both groups was significantly improved (P < 0.001 compared to baseline for each group). The quality of life in the study group was slightly increased as compared to the control group, but the difference was not statistically significant (P > 0.05). All patients in the study group achieved adequate B-cell depletion. The recurrence rate at 6 months in the study group (18/46, 39.13%) was lower as compared to the control group (30/45, 66.67%) (Risk Difference − 27.54%, 95% CI − 47.00% to − 8.08%; P = 0.009). The incidence of adverse reactions, including mild transient neutropenia, was comparable between the two groups (P > 0.05).

Conclusion

The rituximab-based regimen combined with target care exhibited beneficial outcomes by effectively improving clinical efficacy and reducing recurrence for children with nephrotic syndrome, with a safety profile comparable to a tacrolimus-based regimen over the study period. Quality of life improved in both groups. Further studies with longer follow-up are needed to assess long-term outcomes and safety.

Trial registration

ClinicalTrials.gov, NCT07049172.