Progression-Free Survival and Patterns of Response in Patients with Relapsed High-Risk Neuroblastoma Treated with Irinotecan/Temozolomide/Dinutuximab/Granulocyte-Macrophage Colony-Stimulating Factor

Benjamin J. Lerman, Yimei Li, Cecilia Carlowicz, Meaghan Granger, Thomas Cash, Arhanti Sadanand, Katherine Somers, Aeesha Ranavaya, Brian D. Weiss, Michelle Choe, Jennifer H. Foster, Navin Pinto, Daniel A. Morgenstern, Margarida Simão Rafael, Keri A. Streby, Rachel N. Zeno, Rajen Mody, Sahr Yazdani, Ami V. Desai, Margaret E. MacySuzanne Shusterman, Sara M. Federico, Rochelle Bagatell

Research output: Contribution to journalArticlepeer-review

8 Scopus citations

Abstract

PURPOSEAlthough chemoimmunotherapy is widely used for treatment of children with relapsed high-risk neuroblastoma (HRNB), little is known about timing, duration, and evolution of response after irinotecan/temozolomide/dinutuximab/granulocyte-macrophage colony-stimulating factor (I/T/DIN/GM-CSF) therapy.PATIENTS AND METHODSPatients eligible for this retrospective study were age < 30 years at diagnosis of HRNB and received ≥ 1 cycle of I/T/DIN/GM-CSF for relapsed or progressive disease. Patients with primary refractory disease who progressed through induction were excluded. Responses were evaluated using the International Neuroblastoma Response Criteria.RESULTSOne hundred forty-six patients were included. Tumors were MYCN-amplified in 50 of 134 (37%). Seventy-one patients (49%) had an objective response to I/T/DIN/GM-CSF (objective response; 29% complete response, 14% partial response [PR], 5% minor response [MR], 21% stable disease [SD], and 30% progressive disease). Of patients with SD or better at first post-I/T/DIN/GM-CSF disease evaluation, 22% had an improved response per International Neuroblastoma Response Criteria on subsequent evaluation (13% of patients with initial SD, 33% with MR, and 41% with PR). Patients received a median of 4.5 (range, 1-31) cycles. The median progression-free survival (PFS) was 13.1 months, and the 1-year PFS and 2-year PFS were 50% and 28%, respectively. The median duration of response was 15.9 months; the median PFS off all anticancer therapy was 10.4 months after discontinuation of I/T/DIN/GM-CSF.CONCLUSIONApproximately half of patients receiving I/T/DIN/GM-CSF for relapsed HRNB had objective responses. Patients with initial SD were unlikely to have an objective response, but > 1 of 3 patients with MR/PR on first evaluation ultimately had complete response. I/T/DIN/GM-CSF was associated with extended PFS in responders both during and after discontinuation of treatment. This study establishes a new comparator for response and survival in patients with relapsed HRNB.

Original languageEnglish (US)
Pages (from-to)508-516
Number of pages9
JournalJournal of Clinical Oncology
Volume41
Issue number3
DOIs
StatePublished - Jan 20 2023
Externally publishedYes

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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