Preliminary real-world efficacy and safety of telitacicept in pediatric immunoglobulin A nephropathy: An exploratory retrospective case series

Immunoglobulin A nephropathy is a predominant cause of chronic kidney disease in the pediatric population. Despite standard-of-care treatments, a significant subset of children remains refractory to conventional immunosuppressive therapies, necessitating exploration of novel targeted interventions. Telitacicept, a recombinant fusion protein targeting both B lymphocyte stimulator and a proliferation-inducing ligand, has demonstrated efficacy in adult immunoglobulin A nephropathy; however, real-world evidence in pediatric cases remains limited. This exploratory retrospective case series evaluated the efficacy and safety of telitacicept in seven pediatric patients with refractory immunoglobulin A nephropathy. Telitacicept was administered subcutaneously once weekly using a weight-stratified dosing regimen (80 mg for patients weighing <40 kg and 160 mg for patients weighing ≥40 kg) over a 24-week period. At the end of follow-up, clinical remission (defined as urine protein-to-creatinine ratio <0.2 g/g) was achieved in five of the seven patients (71.4%). Notably, a rapid therapeutic response was observed, with 85.7% of patients achieving at least partial remission by month 3. Although estimated glomerular filtration rate remained stable throughout the study, significant reductions were observed in serum immunoglobulin A, immunoglobulin G, and immunoglobulin M levels (all p < 0.05), correlating with clinical improvement. However, two patients with specific pathological features, including minimal change disease or advanced chronic lesions, showed limited response to treatment. The treatment was well tolerated, with only one instance of mild upper respiratory tract infection and no serious adverse events reported. These preliminary findings suggest that weight-stratified telitacicept is a promising and safe adjuvant therapy for pediatric patients with refractory immunoglobulin A nephropathy, although its efficacy may be influenced by individual clinicopathological characteristics.