The mAb was approved in combination with the cytokines granulocyte macrophage colony-stimulating factor (GM-CSF) and interleukin-2 (IL-2), and the oral retinoid isotretinoin (RA). The approval was based on a pivotal, phase 3, multicenter, open-label, randomized trial conducted by the Children’s Oncology Group between October 2001 and January 2009 that was stopped early after the combination demonstrated superiority over standard therapy with respect to event-free survival (EFS).2
Two hundred and twenty-six patients (mostly pediatric patients, though age up to 31 years at diagnosis was allowed) with high-risk neuroblastoma were enrolled based on the following criteria: age of ≤31 years at diagnosis; completion of induction therapy, autologous stem-cell transplant (SCT), and radiation therapy, with autologous SCT performed within 9 months of initiation of induction therapy; achievement of at least partial response prior to autologous SCT; enrollment between 50-100 days after final autologous SCT; absence of progressive disease; adequate organ function; life expectancy of at least 2 months; and prior enrollment in the COG biology study (ANBL00B1). An additional 25 patients with biopsy-proven residual disease after autologous SCT were also enrolled, but were nonrandomly assigned to the immunotherapy arm and were excluded from the primary outcome analysis. Patients with systemic infections or a requirement for concomitant systemic corticosteroids or immunosuppressant usage were ineligible.
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