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

Villablanca, Judith G.; Ji, Lingyun; Shapira-Lewinson, Adi; Marachelian, Araz; Shimada, Hiroyuki; Hawkins, Randall A.; Pampaloni, Miguel Hernandez; Lai, Hollie; Goodarzian, Fariba; Sposto, Richard; Park, Julie R.; Matthay, Katherine K.

doi:10.1002/pbc.26940

Cited by 10 publications

(29 citation statements)

References 40 publications

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“…Many studies have shown that MYCN is an independent factor for unfavorable prognosis. [ 11 12 ] The key action of MYCN in inducing NB has been confirmed in vivo and in vitro. [ 13 14 ] The number of MYCN copies required to be defined as amplification is still controversial.…”

Section: Discussionmentioning

confidence: 99%

Bone and bone marrow involvement in neuroblastoma

Huang

Jiang²,

Liao³

et al. 2020

Medicine

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Rationale: Neuroblastoma (NB) can occur in any part of the sympathetic nervous system, and it is highly heterogeneous. Tumors that only involve bone marrow and bone lesions without solid masses have rarely been reported. Patient concerns: A 2-year-old girl child presented with recurrent fever, accompanied by pain in both lower limbs for more than 1 month. Diagnose: Bone marrow examination revealed NB cell invasion. Femoral and multiple vertebral lesions were observed by MRI, while head MRI, chest CT, abdominal CT, and pelvic CT showed no solid mass. Interventions: The child received the standard therapy for high-risk NB. Outcomes: She was sensitive to the initial chemotherapy protocol. Two years later, a bone marrow examination confirmed NB recurrence. Lessons: The prognosis of this special type of NB was not improved mainly based on common chemotherapy and local radiotherapy, and new treatment strategies should be explored.

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Section: Discussionmentioning

confidence: 99%

Bone and bone marrow involvement in neuroblastoma

Huang

Jiang²,

Liao³

et al. 2020

Medicine

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“…Risk factors for an inferior outcome included stage 4, age ≥18 months at first diagnosis, MYCN amplification, loss of heterozygosity of chromosome 11q, shorter time from diagnosis to first recurrence, abdominal primary tumor, bone marrow metastasis at first diagnosis, recurrent disease (vs refractory disease), and increased lactate dehydrogenase blood levels . Other predictors for poor outcome were measurable tumor on computed tomography/ magnetic resonance imaging at second‐line trial enrollment, high Curie score by metaiodobenzylguanidine scintigraphy, and stem cell supported therapy . The analyzed cohorts varied and included recurrences from localized tumors, patients aged less than 18 months at diagnosis, and patients with refractory disease …”

Section: Introductionmentioning

confidence: 99%

“…Other predictors for poor outcome were measurable tumor on computed tomography/ magnetic resonance imaging at second‐line trial enrollment, high Curie score by metaiodobenzylguanidine scintigraphy, and stem cell supported therapy . The analyzed cohorts varied and included recurrences from localized tumors, patients aged less than 18 months at diagnosis, and patients with refractory disease …”

Section: Introductionmentioning

confidence: 99%

“…[1][2][3][4][5][6][7][8][9] The reported time periods from the observation of first recurrence to the subsequent progression are short (median intervals 58 days, 1 4.7 months, 2 6.4 months 3 ), and the overall survival proportions have been poor (20% after 4 years, 1 20% after 5 years, 4 7% after 10 years 5 ). Risk factors for an inferior outcome included stage 4, 4,5 age ≥18 months at first diagnosis, 4,5 MYCN amplification, 1,2,4,6,7 loss of heterozygosity of chromosome 11q, 1 shorter time from diagnosis to first recurrence, 4,7 abdominal primary tumor, 5 bone marrow metastasis at first diagnosis, 2 recurrent disease (vs refractory disease), 3,8,10 and increased lactate dehydrogenase blood levels. 5 Other predictors for poor outcome were measurable tumor on computed tomography/ magnetic resonance imaging at second-line trial enrollment, high Curie score by metaiodobenzylguanidine scintigraphy, and stem cell supported therapy.…”

Section: Introductionmentioning

confidence: 99%

“…5 Other predictors for poor outcome were measurable tumor on computed tomography/ magnetic resonance imaging at second-line trial enrollment, high Curie score by metaiodobenzylguanidine scintigraphy, and stem cell supported therapy. 10 The analyzed cohorts varied and included recurrences from localized tumors, 1,2,[4][5][6][7][8]10 patients aged less than 18 months at diagnosis, 1,2,4,5,9,10 and patients with refractory disease. 3,8,10 For parents, the decision to undergo therapy a second time after recurrence of stage 4 neuroblastoma is heavily dependent on the potential survival chances.…”

Section: Introductionmentioning

confidence: 99%

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A new risk score for patients after first recurrence of stage 4 neuroblastoma aged ≥18 months at first diagnosis

Kreitz¹,

Ernst

Schmidt³

et al. 2019

Cancer Medicine

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Background The prognosis of patients with recurrences from stage 4 neuroblastoma is not uniformly dismal. The evaluation of new therapies therefore needs to consider the individual risks of the treated patients. This study aims to define clinically useful risk criteria. Patients and Methods Inclusion criteria were: first recurrence of neuroblastoma stage 4 aged ≥18 months and enrollment in first line trials between 1997 and 2016. Patients were randomized into a training set (N = 310) and an independent validation set (N = 159). The primary endpoint was secondary event‐free survival. The individual treatment elements the patients received during initial and recurrent disease were analyzed as binary and time‐dependent variables. A five‐step multiple time‐dependent Cox regression analysis was performed on the training set to identify prognostic variables adjusted for the individual frontline treatment. The selected variables resulted in a prognostic index (PI) and were used to build a risk score system. The score was validated with the validation set. Results Of the 469 patients, 372 were treated with curative intent and 97 with palliative intent. The PI included the variables number of recurrence organs (hazard ratio [HR] = 2.27), time to recurrence (HR = 2.03), liver metastasis at diagnosis (HR = 1.77), first recurrence at site of the primary tumor (HR = 1.55), and age (HR = 1.29). Three risk groups were built and confirmed in the validation set. The scoring system was likewise useful for the curatively or palliatively treated subgroups. Conclusion A new risk score system for patients with first recurrence of stage 4 neuroblastoma aged ≥18 months at diagnosis is proposed.

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Early‐phase clinical trial eligibility and response evaluation criteria for refractory, relapsed, or progressive neuroblastoma: A consensus statement from the National Cancer Institute Clinical Trials Planning Meeting

Park

Villablanca

Hero

et al. 2022

Cancer

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BACKGROUND:International standardized criteria for eligibility, evaluable disease sites, and disease response assessment in patients with refractory, progressive, or relapsed high-risk neuroblastoma enrolled in early-phase clinical trials are lacking. METHODS: A National Cancer Institute-sponsored Clinical Trials Planning Meeting was convened to develop an international consensus to refine the tumor site eligibility criteria and evaluation of disease response for early-phase clinical trials in children with high-risk neuroblastoma. RESULTS: Standardized data collection of patient and disease characteristics (including specified genomic data), eligibility criteria, a definition of evaluable disease, and response evaluations for primary and metastatic sites of disease were developed. Eligibility included two distinct patient groups: progressive disease and refractory disease. The refractory disease group was subdivided into responding persistent disease and stable persistent disease to better capture the clinical heterogeneity of refractory neuroblastoma. Requirements for defining disease evaluable for a response assessment were provided; they included requirements for biopsy to confirm viable neuroblastoma and/or ganglioneuroblastoma in those patients with soft tissue or bone disease not avid for iodine-123 meta-iodobenzylguanidine. Standardized evaluations for response components and time intervals for response evaluations were established. CONCLUSIONS: The use of international consensus eligibility, evaluability, and response criteria for early-phase clinical studies will facilitate the collection of comparable data across international tri-als and promote more rapid identification of effective treatment regimens for high-risk neuroblastoma.

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

Cited by 10 publications

References 40 publications

Bone and bone marrow involvement in neuroblastoma

Bone and bone marrow involvement in neuroblastoma

A new risk score for patients after first recurrence of stage 4 neuroblastoma aged ≥18 months at first diagnosis

Early‐phase clinical trial eligibility and response evaluation criteria for refractory, relapsed, or progressive neuroblastoma: A consensus statement from the National Cancer Institute Clinical Trials Planning Meeting

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