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Background <p>Hydroxychloroquine (HCQ) has been identified as an interesting treatment option in adult IgA nephropathy. HCQ targets the active toll-like receptors in IgA nephropathy. The purpose of this study was to explore the effects and potential benefits of HCQ for childhood-onset IgA nephropathy (cIgAN).</p> Methods <p>We conducted a retrospective multicenter cohort study including 16 children with biopsy-proven cIgAN treated with HCQ in tertiary pediatric nephrology centers in Canada and France. HCQ was prescribed as an adjunctive therapy for persistent proteinuria despite standard-of-care treatment and/or with a steroid-sparing intent. Clinical and histological data were collected at baseline, and urine protein-to-creatinine ratios (UPCR) and estimated glomerular filtration rates (eGFR) were documented at HCQ initiation, 3, 6, 9, and 12&#xa0;months of follow-up. Iterative biopsies before and after the introduction of HCQ (<i>n</i> = 8) were compared. Steroid exposure was assessed in both groups, and safety monitoring data for HCQ therapy were documented.</p> Results <p>Median age was 12.74 [11.33–14.07] years, with 37.5% male participants. Most (15/16, 93.8%) participants received steroid therapy, and all (16/16, 100%) were treated with renin–angiotensin–aldosterone system blockers. HCQ was initiated within a median time of 519 [249–1016.5] days from initial diagnosis. UPCR at 3, 6, 9, and 12&#xa0;months after HCQ initiation significantly decreased, both in absolute and relative metrics. A decrease of 50% was noted at 12&#xa0;months of follow-up. eGFR remained stable over the follow-up period. Follow-up biopsies in the HCQ-treated group showed stability or improvement of the Oxford score. No complications of HCQ were documented.</p> Conclusions <p>In this first pediatric cohort reporting on real-life experience with HCQ, HCQ use was associated with a sustained reduction in proteinuria and stable kidney function in children with difficult-to-control cIgAN, with an excellent safety profile. Although causality cannot be inferred due to the observational design and concomitant therapies, these findings support further prospective evaluation of HCQ as a safe adjunctive treatment in selected cases of cIgAN.</p> Graphical abstract <p>A higher resolution version of the Graphical abstract is available as <InternalRef RefID="MOESM1">Supplementary information</InternalRef>.</p> <p></p>

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Hydroxychloroquine use in pediatric IgA nephropathy

  • 期刊论文

  • 发表日期：

    2026年3月14日

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  • Karma Abukasm,
  • Lison Lachize Neanne,
  • Andrea Lafitte,
  • Zoe Reverand,
  • Véronique Phan,
  • Olivia Boyer,
  • Geneviève Benoit,
  • Kevin Côté,
  • Marion Rabant,
  • Anne-Laure Lapeyraque,
  • Claire Dossier,
  • Alexandra Cambier

摘要

Background

Hydroxychloroquine (HCQ) has been identified as an interesting treatment option in adult IgA nephropathy. HCQ targets the active toll-like receptors in IgA nephropathy. The purpose of this study was to explore the effects and potential benefits of HCQ for childhood-onset IgA nephropathy (cIgAN).

Methods

We conducted a retrospective multicenter cohort study including 16 children with biopsy-proven cIgAN treated with HCQ in tertiary pediatric nephrology centers in Canada and France. HCQ was prescribed as an adjunctive therapy for persistent proteinuria despite standard-of-care treatment and/or with a steroid-sparing intent. Clinical and histological data were collected at baseline, and urine protein-to-creatinine ratios (UPCR) and estimated glomerular filtration rates (eGFR) were documented at HCQ initiation, 3, 6, 9, and 12 months of follow-up. Iterative biopsies before and after the introduction of HCQ (n = 8) were compared. Steroid exposure was assessed in both groups, and safety monitoring data for HCQ therapy were documented.

Results

Median age was 12.74 [11.33–14.07] years, with 37.5% male participants. Most (15/16, 93.8%) participants received steroid therapy, and all (16/16, 100%) were treated with renin–angiotensin–aldosterone system blockers. HCQ was initiated within a median time of 519 [249–1016.5] days from initial diagnosis. UPCR at 3, 6, 9, and 12 months after HCQ initiation significantly decreased, both in absolute and relative metrics. A decrease of 50% was noted at 12 months of follow-up. eGFR remained stable over the follow-up period. Follow-up biopsies in the HCQ-treated group showed stability or improvement of the Oxford score. No complications of HCQ were documented.

Conclusions

In this first pediatric cohort reporting on real-life experience with HCQ, HCQ use was associated with a sustained reduction in proteinuria and stable kidney function in children with difficult-to-control cIgAN, with an excellent safety profile. Although causality cannot be inferred due to the observational design and concomitant therapies, these findings support further prospective evaluation of HCQ as a safe adjunctive treatment in selected cases of cIgAN.

Graphical abstract

A higher resolution version of the Graphical abstract is available as Supplementary information.