Long-Term Outcomes of Patients With Relapsed/Refractory Neuroblastoma Treated on ANBL1221: A Report From the Children's Oncology Group.

Results of COG ANBL1221 changed treatment paradigms for patients with relapsed/refractory neuroblastoma. Chemoimmunotherapy with irinotecan/temozolomide/dinutuximab is now used as both bridge therapy following suboptimal response to induction and as treatment in first relapse. Prospective data regarding long-term outcomes following this therapy have not been reported. Five-year progression-free survival (PFS) and overall survival (OS) for patients treated with chemoimmunotherapy were 22.6% ± 6.6% and 54.7% ± 7.7%, respectively. Patients with early disease progression following treatment on ANBL1221 tended to have been diagnosed with high-risk neuroblastoma at a younger age than those without early disease progression. A subset of chemoimmunotherapy-treated patients survived ≥2 years, but most all received additional therapy.