Association between end‐induction response according to the revised International Neuroblastoma Response Criteria (INRC) and outcome in high‐risk neuroblastoma patients

Barr, Erin K.; Laurie, Kathryn; Wroblewski, Kristen; Applebaum, Mark A.; Cohn, Susan L.

doi:10.1002/pbc.28390

Cited by 8 publications

(9 citation statements)

References 28 publications

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“…More than 90% of the transplantation group achieved CR/VGPR after induction chemotherapy, which was higher than the 40%-70% reported in the literature and may be responsible for the better prognosis [4,6,9]. As shown in previous studies, the treatment response after induction is also an important prognostic factor for high-risk neuroblastoma [4,19]. However, some medical centres in China have a conservative view of the prognostic value of stem cell transplantation.…”

Section: Stem Cell Transplantation Significantly Improves Prognosismentioning

confidence: 78%

Analysis of the efficacy of autologous peripheral blood stem cell transplantation in high-risk neuroblastoma

Jin¹,

Li²,

Yang³

et al. 2022

Int. J. Med. Sci.

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Objective: This study aimed to analyze the efficacy of autologous peripheral blood stem cell transplantation for high-risk neuroblastoma in China. Methods: The data of 90 high-risk neuroblastoma patients treated with the CCCG-NB 2015 regimen were reviewed. The baseline clinicopathological characteristics and prognosis were analyzed and compared. In addition, the prognoses of tandem autologous stem cell transplantation and single autologous stem cell transplantation groups were compared. Results: The results of survival analysis showed that autologous peripheral blood stem cell transplantation based on this pretreatment regimen significantly improved the prognosis of children in the high-risk group. The 3-year event-free survival (EFS) and overall survival (OS) rates for the transplantation group and the nontransplantation group were 65.5% vs. 41.3% ( p =0.023) and 77.1% vs. 57.9% ( p =0.03), respectively. There was no difference in the distribution of baseline clinical case characteristics between the single transplantation group and the tandem transplantation group ( p >0.05), and there was no significant difference in EFS and OS between the two groups ( p >0.05). Conclusion: Based on this pretreatment programme, autologous peripheral blood stem cell transplantation is safe and tolerable and significantly improves the prognosis of children in the high-risk group. The value of tandem autologous stem cell transplantation is worthy of further discussion, which should consider various aspects such as the transplantation medication regimen and the patient's state.

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Section: Stem Cell Transplantation Significantly Improves Prognosismentioning

confidence: 78%

Analysis of the efficacy of autologous peripheral blood stem cell transplantation in high-risk neuroblastoma

Jin¹,

Li²,

Yang³

et al. 2022

Int. J. Med. Sci.

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“…In brief, all patients were diagnosed with neuroblastoma between the ages of 0 and 29 years and samples were collected at two local children's hospitals after approval by local Institutional Review Boards. Response to treatment was assessed according to the 2017 International Neuroblastoma Response Criteria 15 . Patients were excluded if they did not have high‐risk disease.…”

Section: Methodsmentioning

confidence: 99%

“…Response to treatment was assessed according to the 2017 International Neuroblastoma Response Criteria. 15 Patients were excluded if they did not have high-risk disease. Blood samples were collected in EDTA tubes and processed within two hours of collection.…”

Section: Patients and 5-hmc Libraries From Cfdna Datamentioning

confidence: 99%

Adrenergic and mesenchymal signatures are identifiable in cell‐free DNA and correlate with metastatic disease burden in children with neuroblastoma

Vayani,

Kaufman,

Moore

et al. 2023

Pediatric Blood & Cancer

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BackgroundCell‐free DNA (cfDNA) profiles of 5‐hydroxymethylcytosine (5‐hmC), an epigenetic marker of open chromatin and active gene expression, are correlated with metastatic disease burden in patients with neuroblastoma. Neuroblastoma tumors are comprised of adrenergic (ADRN) and mesenchymal (MES) cells, and the relative abundance of each in tumor biopsies has prognostic implications. We hypothesized that ADRN and MES‐specific signatures could be quantified in cfDNA 5‐hmC profiles and would augment the detection of metastatic burden in patients with neuroblastoma.MethodsWe previously performed an integrative analysis to identify ADRN and MES‐specific genes (n = 373 and n = 159, respectively). Purified DNA from cell lines was serial diluted with healthy donor cfDNA. Using Gene Set Variation Analysis (GSVA), ADRN and MES signatures were optimized. We then quantified signature scores, and our prior neuroblastoma signature, in cfDNA from 84 samples from 46 high‐risk patients including 21 patients with serial samples.ResultsSamples from patients with higher metastatic burden had increased GSVA scores for both ADRN and MES gene signatures (p < .001). While ADRN and MES signature scores tracked together in serially collected samples, we identified instances of patients with increases in either MES or ADRN score at relapse.ConclusionsWhile it is feasible to identify ADRN and MES signatures using 5‐hmC profiles of cfDNA from neuroblastoma patients and correlate these signatures to metastatic burden, additional data are needed to determine the optimal strategies for clinical implementation. Prospective evaluation in larger cohorts is ongoing.

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“…[1][2][3][4] Accordingly, the International Neuroblastoma Risk Group has adopted MIBG scanning for predicting treatment response. 5,6 However, there is a dearth of research examining the role of MIBG scanning after the introduction of tandem high-dose chemotherapy (HDCT) with autologous stem cell transplantation (auto-SCT),…”

Section: Introductionmentioning

confidence: 99%

“…Metaiodobenzylguanidine (MIBG) scintigraphy labeled with iodine‐131 ( 131 I) or iodine‐123 ( 123 I) is a well‐established imaging tool for response assessment, and early changes in MIBG uptake have been found to predict clinical outcomes in neuroblastoma 1–4 . Accordingly, the International Neuroblastoma Risk Group has adopted MIBG scanning for predicting treatment response 5,6 …”

Section: Introductionmentioning

confidence: 99%

Clinical implication of residual MIBG‐positive disease in the follow‐up of high‐risk neuroblastoma treated with tandem high‐dose chemotherapy and autologous stem cell transplantation

Seo

Shin

Lim

et al. 2021

Pediatric Blood & Cancer

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Background: The implication of residual metaiodobenzylguanidine (MIBG)-positive disease in the era of tandem high-dose chemotherapy (HDCT) with autologous stem cell transplantation (auto-SCT) has not yet been established in neuroblastoma. Moreover, most published studies have not evaluated the long-term prognosis of patients with residual MIBG-positive disease following treatment completion. Therefore, we investigated the prognostic significance of residual MIBG-positive disease at each treatment phase and after treatment completion. Methods: We assessed MIBG scans labeled with either iodine-123 ( 123 I) or 131 I from 150 patients with MIBG-avid and high-risk neuroblastoma enrolled in the NB-2004, -2009, and -2014 trials at postinduction, posttandem HDCT/auto-SCT, and completion of treatment. Results:The residual MIBG-positive disease at postinduction and posttandem HDCT/auto-SCT evaluation was highly correlated with the risk of progression. However, at treatment completion, there was no significant difference in survival and risk of progression between patients with residual MIBG-positive disease and MIBG-negative patients. Patients with persistent MIBG-positive disease at the end of treatment were more likely to have indolent tumor characteristics, such as favorable histology at diagnosis, lower incidence of MYCN amplification, and slow response to chemotherapy. Conclusion:Residual MIBG-positive disease during treatment predicted unfavorable outcomes for patients with high-risk neuroblastoma, even under tandem HDCT/auto-SCT. However, persistent MIBG uptake at the completion of all treatments may not always indicate an active disease.

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Association between end‐induction response according to the revised International Neuroblastoma Response Criteria (INRC) and outcome in high‐risk neuroblastoma patients

Cited by 8 publications

References 28 publications

Analysis of the efficacy of autologous peripheral blood stem cell transplantation in high-risk neuroblastoma

Analysis of the efficacy of autologous peripheral blood stem cell transplantation in high-risk neuroblastoma

Adrenergic and mesenchymal signatures are identifiable in cell‐free DNA and correlate with metastatic disease burden in children with neuroblastoma

Clinical implication of residual MIBG‐positive disease in the follow‐up of high‐risk neuroblastoma treated with tandem high‐dose chemotherapy and autologous stem cell transplantation

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