Outcome of children with relapsed or refractory neuroblastoma: A meta‐analysis of ITCC/SIOPEN European phase II clinical trials

Moreno, Lucas; Rubie, H; Varo, A; Deley, Marie-Cécile Le; Amoroso, Loredana; Chevance, Astrid; Garaventa, Alberto; Gambart, Marion; Bautista, Francisco; Valteau‐Couanet, Dominique; Geoerger, Birgit; Vassal, Gilles; Paolettí, Xavier; Pearson, Andrew D.J.

doi:10.1002/pbc.26192

Cited by 75 publications

(75 citation statements)

References 19 publications

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“…The reported 5-year OS rate for patients after the first relapse of neuroblastoma is 20% [94], with outcomes dependent on the time of relapse and the initial patient tumor stage [94][95][96]. A recently published meta-analysis of three phase II clinical trials run through the SIOPEN group in Europe reported median progression free survival rates of 12.5% and 5.7% for patients with refractory and relapsed disease, respectively, while median OS rates were 27.9 months for patients with refractory disease versus 11.0 months for patients with relapsed disease, confirming the poor outcomes in both cohorts of patients [97].…”

Section: Treatment -Relapsed and Refractory Neuroblastomamentioning

confidence: 87%

Overview and recent advances in the treatment of neuroblastoma

Whittle

Smith

Doherty

et al. 2017

Expert Review of Anticancer Therapy

325

258

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Introduction: Children with neuroblastoma have widely divergent outcomes, ranging from cure in >90% of patients with low risk disease to <50% for those with high risk disease. Recent research has shed light on the biology of neuroblastoma, allowing for more accurate risk stratification and treatment reduction in many cases, although newer treatment strategies for children with high-risk and relapsed neuroblastoma are needed to improve outcomes. Areas covered: Neuroblastoma epidemiology, diagnosis, risk stratification, and recent advances in treatment of both newly diagnosed and relapsed neuroblastoma. Expert commentary: The identification of newer tumor targets and of novel cell-mediated immunotherapy agents may lead to novel therapeutic approaches, and clinical trials for regimens designed to target individual genetic aberrations in tumors are underway. A combination of therapeutic modalities will likely be required to improve survival and cure rates for patients with high-risk neuroblastoma.ARTICLE HISTORY

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Section: Treatment -Relapsed and Refractory Neuroblastomamentioning

confidence: 87%

Overview and recent advances in the treatment of neuroblastoma

Whittle

Smith

Doherty

et al. 2017

Expert Review of Anticancer Therapy

325

258

View full text Add to dashboard Cite

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Section: Introductionmentioning

confidence: 99%

“…The prognosis of children with recurrences of high‐risk neuroblastoma is dismal . The reported time periods from the observation of first recurrence to the subsequent progression are short (median intervals 58 days, 4.7 months, 6.4 months), and the overall survival proportions have been poor (20% after 4 years, 20% after 5 years, 7% after 10 years). 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 .…”

Section: Introductionmentioning

confidence: 99%

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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“…Despite recent advances in high‐risk neuroblastoma (HR‐NBL) therapy, long‐term survival for patients with metastatic or high‐risk disease remains less than 40% . Further, patients who experience a relapse of their disease or fail to achieve complete remission have an extremely poor prognosis with 2‐year survival rates of less than 25% . Better understanding of the biology of NBL is critical in identifying new therapeutic targets and improving outcomes for this patient population.…”

Section: Introductionmentioning

confidence: 99%