Moreno, Lucas, Guo, Dongjing, Irwin, Meredith S., Berthold, Frank 
ORCID: 0000-0002-7613-1723, Hogarty, Michael ORCID: 0000-0002-9221-4852, Kamijo, Takehiko, Morgenstern, Daniel ORCID: 0000-0002-4859-1108, Pasqualini, Claudia, Ash, Shifra, Potschger, Ulrike, Ladenstein, Ruth, Valteau-Couanet, Dominique, Cohn, Susan L., Pearson, Andrew D. J. and London, Wendy B.
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A nomogram of clinical and biologic factors to predict survival in children newly diagnosed with high-risk neuroblastoma: An International Neuroblastoma Risk Group project.
Pediatr. Blood Cancer.
HOBOKEN:
WILEY.
ISSN 1545-5017
Abstract
Background Long-term outcome remains poor for children with high-risk neuroblastoma (five-year overall survival [OS] similar to 50%). Our objectives were to (a) identify prognostic biomarkers and apply them in a nomogram to identify the subgroup of ultra-high-risk patients at highest risk of disease progression/death, for whom novel frontline therapy is urgently needed; and (b) validate the nomogram in an independent cohort. Methods A total of 1820 high-risk patients (>= 18 months old with metastatic neuroblastoma), diagnosed 1998-2015, from the International Neuroblastoma Risk Groups (INRG) Data Commons were analyzed in a retrospective cohort study. Using multivariable Cox regression of OS from diagnosis, a nomogram was created from prognostic biomarkers to predict three-year OS. External validation was performed using the SIOPEN HR-NBL1 trial cohort (n = 521), evidenced by receiver operating characteristic curves. Results The nomogram, including MYCN status (P < 0.0001), lactate dehydrogenase (LDH) (P = 0.0007), and presence of bone marrow metastases (P = 0.004), had robust performance and was validated. Applying the nomogram at diagnosis (a) gives prognosis of an individual patient and (b) identifies patients predicted to have poor outcome (three-year OS was 30% +/- 5% for patients with a nomogram score of > 82 points; 58% +/- 1% for those <= 82 points). Median follow-up time was 5.5 years (range, 0-14.1). Conclusions In high-risk neuroblastoma, a novel, publicly available nomogram using prognostic biomarkers (MYCN status, LDH, presence of bone marrow metastases; ) has the flexibility to apply a clinically suitable and context-specific cutoff to identify patients at highest risk of death. This will facilitate testing urgently needed new frontline treatment options to improve outcome for these children.
| Item Type: | Journal Article | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| URN: | urn:nbn:de:hbz:38-311213 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| DOI: | 10.1002/pbc.28794 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Journal or Publication Title: | Pediatr. Blood Cancer | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Publisher: | WILEY | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Place of Publication: | HOBOKEN | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| ISSN: | 1545-5017 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Language: | English | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Faculty: | Unspecified | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Divisions: | Unspecified | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Subjects: | no entry | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| URI: | http://kups.ub.uni-koeln.de/id/eprint/31121 |
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