Dinutuximab for maintenance therapy in pediatric neuroblastoma

Purpose. The pharmacology, clinical efficacy, safety, dosage and administration, and role in therapy of dinutuximab for the treatment of high-risk pediatric neuroblastoma are reviewed. Summary. Dinutuximab (Unituxin, United Therapeutics) is a novel monoclonal antibody recently approved for use in combination with granulocyte macrophage colony-stimulating factor, interleukin-2, and isotretinoin for the treatment of pediatric patients with high-risk neuroblastoma. Its approval has led to the first major change in standard recommended first line maintenance therapy for high-risk pediatric neuroblastoma in over a decade. Dinutuximab causes antibody-dependent cell-mediated cytotoxicity and complement-dependent cytotoxicity by binding to GD2, a tumor associated antigen. The recommended dosage of dinutuximab is 17.5 mg/m(2)/day for 4 consecutive days of each 24- or 32-day cycle, for a maximum of 5 cycles. In a Phase III trial, patients who received dinutuximab as part of combination immunotherapy in addition to standard maintenance therapy had significantly improved 2-year event-free survival relative to those who received standard maintenance therapy alone (66% versus 46%, p < 0.01). Dinutuximab has a unique adverse-effect profile that includes infusion reactions, neuropathic pain, and electrolyte abnormalities; the most common adverse effects observed with dinutuximab use in clinical trials were pain, pyrexia, myelosuppression, infusion reactions, and electrolyte abnormalities. Conclusion. Dinutuximab is a novel monoclonal antibody that is efficacious as part of combination immunotherapy in pediatric patients with high-risk neuroblastoma.