Primary immune thrombocytopenia (ITP) in children is often self‑limiting, but a subset becomes refractory to conventional treatments or progresses to chronic disease, posing a major clinical challenge. This case report aims to explore the potential benefit of telitacicept, a novel biologic agent targeting B‑cell activating factor (BAFF) and a proliferation‑inducing ligand (APRIL), in a pediatric patient with refractory ITP who had insufficient responses to standard therapies.

We describe the case of a 5‑year‑old child diagnosed with refractory ITP. The patient had previously received standard first‑line treatments, including intravenous immunoglobulin (IVIG) and high‑dose corticosteroids, without achieving sustained disease control. Subsequent therapy with rituximab, an anti‑CD20 monoclonal antibody, also resulted in only limited and transient improvement. Following disease recurrence and persistent thrombocytopenia with active bleeding symptoms, the decision was made to administer telitacicept. The treatment regimen, monitoring of platelet counts and bleeding episodes, as well as any adverse events, were carefully recorded. The diagnostic evaluation and the complete therapeutic course are presented in detail.

After initiation of telitacicept, the patient exhibited marked and sustained clinical improvement. Specifically, platelet counts rose to safe levels within a few weeks, and all bleeding manifestations resolved completely. Throughout the follow‑up period, the patient maintained stable platelet counts without the need for any additional rescue therapy. Importantly, no infusion‑related reactions or other significant adverse effects were observed during or after treatment. As a result of this favorable response, concurrent immunosuppressive medications could be gradually tapered without disease recurrence.

This case suggests that telitacicept may be a highly effective and well‑tolerated salvage therapy for pediatric patients with refractory ITP who have already exhausted all available conventional treatment options. Given the current lack of large‑scale studies in children, further prospective observational research and well‑designed rigorous clinical trials are still definitely necessary to confirm its efficacy, define optimal dosing, and evaluate long‑term safety in the pediatric population.