Targeted Therapy for Systemic Epstein-Barr Virus–Positive T-Cell Lymphoma of Childhood

Systemic Epstein-Barr virus-positive T-cell lymphoma of childhood (STCLC) is a rare and highly aggressive malignancy with a dismal prognosis and no standard curative treatment. This report describes the first reported case of STCLC in a White child who, after experiencing failure with intensive chemotherapy regimens, achieved a complete response following targeted immunotherapy. Initially, the patient had a rapidly progressive illness with multisystem involvement, presenting as a diagnostic and treatment dilemma due to its rarity. Histomolecular workup was most consistent with a diagnosis of STCLC. Chemotherapy-induced temporary partial response was followed by brisk clinical and radiologic progression. Subsequent immunotherapy resulted in a complete response, which was consolidated with an allogeneic hematopoietic stem cell transplant. The patient currently remains disease-free 3 years later. This case underscores the role of precision medicine in guiding the diagnosis and treatment of rare malignancies. It also highlights the role of immunotherapy as a novel targeted treatment option in STCLC, warranting further investigation.