Abstract: FR-PO0717
Preliminary Study of Tofacitinib in Pediatric Nephrotic Syndrome with Atopic Dermatitis
Session Information
- Pediatric Nephrology: CKD, ESKD, and Glomerular Diseases
November 07, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Pediatric Nephrology
- 1900 Pediatric Nephrology
Authors
- Yin, Lu, Shengjing Hospital of China Medical University, Shenyang, Liaoning, China
- Du, Yue, Shengjing Hospital of China Medical University, Shenyang, Liaoning, China
Introduction
Pediatric nephrotic syndrome (NS) and atopic dermatitis (AD) share inflammatory pathways. Traditional therapies (corticosteroids, immunosuppressants) have side effects and high relapse rates. Tofacitinib (TOF), a JAK inhibitor, shows efficacy in AD but lacks evaluation in NS.
Case Description
Three male NS+AD patients (mean age 3.33 years) received TOF for refractory AD. SCORAD scores improved significantly. All had prior NS relapses despite tacrolimus/cyclophosphamide/mycophenolate. Post-TOF, NS relapses decreased, prednisone doses reduced, and one discontinued tacrolimus. Two patients showed improved growth; one developed mild pneumonia at 11 months.
TOF targets were screened (SuperPred, SwissTarget). Shared AD/NS genes (81 overlaps) were analyzed via Venn. PPI networks identified 10 core targets (IFNG, STAT3, IL6, TNF, etc.). Molecular docking confirmed TOF binding to IFNG, IL6, STAT3, and TNF.
Discussion
Tofacitinib, a JAK inhibitor, demonstrates dual benefits for pediatric patients with both nephrotic syndrome (NS) and atopic dermatitis (AD) by targeting JAK-STAT pathway genes (IFNG, IL6, STAT3, TNF). Clinical observations show reduced NS relapses and improved AD symptoms, though infection risks remain. Further studies are needed to confirm efficacy and safety in this population.
Characteristics of patients before and after tofacitinib
| Patient | sex | Age at diagnosis of nephrotic syndrome (years) | Prior Treatment for nephrotic syndrome | Main Reason for TOF initiation | Nephrotic syndrome duration at TOF initiation(months) | Age at TOF initiation(years) | Relapses in nephrotic syndrome within 1 year before and after TOF | Scorad Score | Minimum prednisone dose | Height increase per month |
| 1 | male | 3.5Y | Prednisone，Tacrolimus, Losartan potassium | Severe AD | 49 | 5.5 | Pre-TOF:3 Post-TOF: 0 | Pre-TOF:63.4 Post-TOF: 28.1 | Pre-TOF:0.27mg/kg.Qd Post-TOF:0.05mg/kg.Qod | Pre-TOF: 0.30cm Post-TOF: 0.63cm |
| 2 | male | 4.5Y | Prednisone, CTX, Tacrolimus | Severe AD | 59 | 6.5 | Pre-TOF: 2 Post-TOF: 0 | Pre-TOF:59.4 Post-TOF: 27.8 | Pre-TOF: 0.03mg/kg. Qd Post-TOF: 0.03mg/kg.Qod | Pre-TOF: 0.69cm Post-TOF: 0.49cm |
| 3 | male | 2Y | Prednisone, Tacrolimus | Severe AD | 67 | 7.5 | Pre-TOF: 3 Post-TOF:0 | Pre-TOF:60.4 Post-TOF:26.4 | Pre-TOF: 0.0125mg/kg. Qd Post-TOF:0.025mg/kg.Qod | Pre-TOF: 0.25cm Post-TOF: 0.63cm |