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Pediatr Blood Cancer. 2018 May;65(5):e26940. doi: 10.1002/pbc.26940. Epub 2018 Jan 19.

Predictors of response, progression-free survival, and overall survival using NANT Response Criteria (v1.0) in relapsed and refractory high-risk neuroblastoma.

Author information

1
Department of Pediatrics, Saban Research Institute, Children's Hospital Los Angeles, Keck School of Medicine of the University of Southern California, Los Angeles, California.
2
Department of Preventative Medicine Statistics, Keck School of Medicine of the University of Southern California, Los Angeles, California.
3
Department of Pediatric Hematology-Oncology, The Ruth Rappaport Children's Hospital, Haifa, Israel.
4
Department of Pathology, Saban Research Institute, Children's Hospital Los Angeles, Keck School of Medicine of the University of Southern California, Los Angeles, California.
5
Department of Radiology, University of California San Francisco, San Francisco, California.
6
Department of Pediatric Radiology, Children's Hospital Orange County, Orange, California.
7
Department of Radiology, Children's Hospital Los Angeles, Keck School of Medicine of the University of Southern California, Los Angeles, California, USA.
8
Department of Pediatrics, Seattle Children's Hospital, University of Washington, Seattle, Washington.
9
Department of Pediatrics, University of California San Francisco, San Francisco, California.

Abstract

PURPOSE:

The New Approaches to Neuroblastoma Therapy Response Criteria (NANTRC) were developed to optimize response assessment in patients with recurrent/refractory neuroblastoma. Response predictors and associations of the NANTRC version 1.0 (NANTRCv1.0) and prognostic factors with outcome were analyzed.

METHODS:

A retrospective analysis was performed of patients with recurrent/refractory neuroblastoma enrolled from 2000 to 2009 on 13 NANT Phase 1/2 trials. NANTRC overall response integrated CT/MRI (Response Evaluation Criteria in Solid Tumors [RECIST]), metaiodobenzylguanidine (MIBG; Curie scoring), and percent bone marrow (BM) tumor (morphology).

RESULTS:

Fourteen (6.9%) complete response (CR) and 14 (6.9%) partial response (PR) occurred among 203 patients evaluable for response. Five-year progression-free survival (PFS) was 16 ± 3%; overall survival (OS) was 27 ± 3%. Disease sites at enrollment included MIBG-avid lesions (100% MIBG trials; 84% non-MIBG trials), measurable CT/MRI lesions (48%), and BM (49%). By multivariable analysis, Curie score of 0 (P < 0.001), lower Curie score (P = 0.003), no measurable CT/MRI lesions (P = 0.044), and treatment on peripheral blood stem cell (PBSC) supported trials (P = 0.005) were associated with achieving CR/PR. Overall response of stable disease (SD) or better was associated with better OS (P < 0.001). In multivariable analysis, MYCN amplification (P = 0.037) was associated with worse PFS; measurable CT/MRI lesions (P = 0.041) were associated with worse OS; prior progressive disease (PD; P < 0.001/P < 0.001), Curie score ≥ 1 (P < 0.001; P = 0.001), higher Curie score (P = 0.048/0.037), and treatment on non-PBSC trials (P = < 0.001/0.003) were associated with worse PFS and OS.

CONCLUSIONS:

NANTRCv1.0 response of at least SD is associated with better OS in patients with recurrent/refractory neuroblastoma. Patient and tumor characteristics may predict response and outcome. Identifying these variables can optimize Phase 1/2 trial design to select novel agents for further testing.

KEYWORDS:

clinical trials; neuroblastoma; response criteria

PMID:
29350464
DOI:
10.1002/pbc.26940
[Indexed for MEDLINE]

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