一次性ネフローゼ症候群

BACKGROUND: Patients with nephrotic syndrome (NS) treated with glucocorticoids often suffer from growth disorders. This study aimed to explore the impact of recombinant human growth hormone treatment in children with growth disorders secondary to NS. METHODS: This retrospective study enrolled patients with NS treated at Guangzhou Women and Children's Medical Center between May 2015 and November 2023. Patients were divided into the recombinant human growth hormone group and the conventional group. The primary outcome was height increment. Secondary outcomes included height standard deviation score, predicted adult height standard deviation score, and height velocity. RESULTS: Among 129 enrolled children (89 in the recombinant human growth hormone group, 40 in the conventional group), the recombinant human growth hormone group showed significantly greater height increments at 6 months (4.80 ± 1.42 vs. 2.02 ± 0.91 cm), 12 months (9.07 ± 2.46 vs. 4.39 ± 1.73 cm), and 24 months (15.34 ± 5.20 vs. 7.88 ± 3.43 cm; all P < .001). Similarly, height standard deviation score and predicted adult height standard deviation score improvements were significantly greater in the recombinant human growth hormone group at all time points (all P < .001). Height velocity was also higher in the recombinant human growth hormone group at 6 and 12 months (both P < .001). These associations remained significant after multivariate adjustment. No significant differences in glucocorticoid exposure during follow-up were observed between groups. Multivariate analysis at 12 months indicated that cumulative glucocorticoid dosage was negatively associated with height standard deviation score (β=-.0022, 95% CI: -0.0041, -0.0004; P = .020) and predicted adult height (β=-.0148, 95% CI: -0.0288, -0.0008; P = .038). CONCLUSION: The results suggested that recombinant human growth hormone treatment in children with NS significantly improved height, height standard deviation score, predicted adult height standard deviation score, and height velocities, compared with conventional treatment.

BACKGROUND: Idiopathic nephrotic syndrome is a common primary glomerular disease in children. Circulating anti-nephrin autoantibodies have emerged as clinically relevant biomarkers, but existing detection methods are complex and time-consuming. We developed a quantum dot microsphere-based lateral flow immunoassay (QD-LFIA) for rapid serum anti-nephrin autoantibody detection. METHODS: Quantum dot microspheres were conjugated to recombinant human nephrin protein, and the reading time, T-line coating concentration, and nephrin protein labeling amount were optimized. Analytical performance was evaluated by receiver operating characteristic (ROC) analysis based on test line/control line ratios generated by the dry fluorescence immunoanalyzer, together with interference and accelerated stability testing. Clinical performance was assessed in 191 pediatric serum samples, with qualitative QD-LFIA results compared against immunoprecipitation-Western blotting (IP-WB). Discordant samples were evaluated by recombinant nephrin protein inhibition assay. Clinical indices were compared between anti-nephrin autoantibody positive and negative patients. RESULTS: The optimized reaction time, T-line coating concentration, and recombinant nephrin protein labeling amount were 10 min, 0.5 mg/mL, and 92.4 μg, respectively. ROC analysis yielded an AUC of 0.90 (95% CI 0.84-0.97), with 93% sensitivity and 92% specificity at a test line/control line cutoff of 0.003. The QD-LFIA resisted the tested interferents and maintained stable performance for 40 days at 37 °C. QD-LFIA achieved overall, positive, and negative percent agreements of 92%, 89%, and 93%, respectively, relative to IP-WB. Recombinant nephrin protein inhibition abolished the signals in all 12 QD-LFIA-positive/IP-WB-negative samples. Anti-nephrin autoantibody positive patients had higher 24-h urinary protein excretion and lower serum albumin levels than negative patients. CONCLUSIONS: These findings suggest that the QD-LFIA enabled rapid detection of anti-nephrin autoantibodies in children with idiopathic nephrotic syndrome. The QD-LFIA showed good agreement with IP-WB and identified additional anti-nephrin-positive samples undetected by IP-WB.

BACKGROUND: Frequently relapsing nephrotic syndrome (FRNS) and steroid-dependent nephrotic syndrome (SDNS) require steroid-sparing agents to maintain remission and reduce steroid-related toxicity. Comparative evidence between mycophenolate mofetil (MMF) and tacrolimus (TAC) remains limited. The objective of this study was to compare the efficacy and safety of MMF and TAC for maintenance of remission in children with FRNS/SDNS. METHODS: This prospective, open-label, pilot randomized controlled trial was conducted at a tertiary care centre in India between October 2023 and March 2025. Children aged 2-18 years with FRNS or SDNS receiving only prior corticosteroids were randomized (1:1) to MMF (1200 mg/m2/day) or TAC (0.1-0.15 mg/kg/day). The primary outcome was relapse-free survival at 6 months. Secondary outcomes included relapse rates over 12 months, steroid exposure, kidney function, blood pressure, growth parameters, and adverse events. RESULTS: Forty patients were enrolled (20 in each group). Relapse-free survival at 6 months was comparable between groups, with no significant difference in time to first relapse. Sustained remission and overall relapse rates over 12 months were also similar. Kidney function was significantly better preserved in the MMF group, whereas TAC was associated with higher serum creatinine and lower eGFR. Blood pressure and body mass index were higher in the TAC group, while growth velocity was greater in the MMF group. Adverse events were mostly mild and comparable. CONCLUSIONS: MMF and TAC showed comparable efficacy in maintaining remission in children with FRNS/SDNS. However, TAC was associated with less favourable kidney, cardiovascular, and growth profiles. MMF may represent a safer alternative for long-term therapy, although larger multicentre trials are needed.

Spontaneous bacterial peritonitis (SBP) is a life-threatening complication of idiopathic nephrotic syndrome (INS) in children. Evidence on the optimal duration of antibiotics in this population is limited. We investigated whether 5-day antibiotic therapy is non-inferior to 7-day therapy for SBP in children with INS. In this single-center, open-label, randomized controlled non-inferiority trial, 50 children aged 1-14 years with INS and SBP were allocated 1:1 to intravenous ceftriaxone for 5 days (Group A, n = 25) or 7 days (Group B, n = 25). The primary outcome was composite clinical response (resolution of fever, abdominal pain, and tenderness) at the end of treatment, analyzed by intention-to-treat. The pre-specified non-inferiority margin was -15%. Secondary outcomes included bacteriological cure, cytological response, and the need for additional antibiotics. Fifty children [median age 50 (30.8-92) months] were randomized. Composite clinical response was achieved in 25/25 (100%) in Group A versus 22/25 (88%) in Group B (risk difference 12%, 95% CI -3.5% to 30.0%; P = 0.23); the lower CI bound exceeded the -15% non-inferiority margin, establishing non-inferiority. Bacteriological cure was achieved in 96% of both groups. Cytological response (64% vs 72%; P = 0.54) was inconclusive for non-inferiority. Additional or modified antibiotic therapy was required in 16% versus 20% (P = 1.00). Median hospitalization was comparable (10 vs 11 days; P > 0.05). No ceftriaxone-related adverse events were recorded. Five-day intravenous ceftriaxone is non-inferior to 7-day therapy for SBP in children with INS, with respect to composite clinical response; the secondary outcome of cytological response was inconclusive for non-inferiority.

Membranous nephropathy (MN) is a common cause of nephrotic syndrome in adults, broadly classified as primary or secondary based on etiology and pathologic findings. We present a single unusual case report of a 75-year-old woman with an 11-year course of MN featuring concurrent characteristics of both primary and secondary disease. On two separate kidney biopsies for the same patient, she demonstrated histopathologic features consistent with Class V lupus nephropathy alongside positive PLA2R staining - a combination that creates significant diagnostic uncertainty. Despite the overlapping features, the patient achieved remission on two separate occasions using different immunosuppressive regimens: cyclosporine initially, followed by Mycophenolate mofetil and prednisone upon relapse over a decade later. This case contributes to the emerging literature on PLA2R positivity in lupus membranous nephropathy and highlights the diagnostic and therapeutic challenges clinicians face when primary and secondary MN features coexist. The long-term follow-up and dual treatment response on a single patient reported here add to a limited body of published cases and carry implications for management of this increasingly recognized overlap syndrome.