Treatment and outcomes of 1041 pediatric patients with neuroblastoma who received multidisciplinary care in China

Su, Yan; Qin, Hong; Chen, Chenghao; Wang, Shengcai; Zhang, Shihan; Zhang, Dawei; Jin, Mei; Peng, Yun; He, Lejian; Wang, Xiaoman; Qian, Suyun; Qin, Maoquan; Ge, Ming; Zhang, Fuquan; Zeng, Qi; Wang, Huanmin; Ma, Xiaoli; Ni, Xin

doi:10.1002/ped4.12214

Cited by 21 publications

(12 citation statements)

References 24 publications

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“…In this study, chemotherapy was administered according to the guidelines for the diagnosis and treatment of NB (2019) published by the National Health Commission of the People’s Republic of China [ 9 ]. Alternating chemotherapy regimens of CBVP (carboplatin, etoposide) were applied to children in the low- and intermediate-risk groups.…”

Section: Methodsmentioning

confidence: 99%

Safety and feasibility of laparoscopic resection of abdominal neuroblastoma without image-defined risk factors: a single-center experience

et al. 2023

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Objective To explore the criteria, safety and efficacy of laparoscopic surgery in pediatric neuroblastoma (NB). Methods A retrospective study of 87 patients with NB without image-defined risk factors (IDRFs) between December 2016 and January 2021 at Beijing Children’s Hospital was conducted. Patients were divided into two groups according to the surgical procedure. Results Between the 87 patients, there were 54 (62.07%) cases in the open surgery group and 33 (37.93%) cases in the laparoscopic surgery group. There were no significant differences between the two groups regarding demographic characteristics, genomic and biological features, operating time or postoperative complications. However, in terms of intraoperative bleeding (p = 0.013) and the time to start postoperative feeding after surgery (p = 0.002), the laparoscopic group was obviously better than the open group. Furthermore, there was no significant difference in the prognosis between the two groups, and no recurrence or death was observed. Conclusion For children with localized NB who have no IDRFs, laparoscopic surgery could be performed safely and effectively. Surgeons who are skilled in this can help children reduce surgical injuries, speed up postoperative recovery, and obtain the same prognosis as open surgery.

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Section: Methodsmentioning

confidence: 99%

Safety and feasibility of laparoscopic resection of abdominal neuroblastoma without image-defined risk factors: a single-center experience

et al. 2023

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“…The survival of the patients was also evaluated. Event-free survival (EFS) was a composite endpoint, which was defined as the time from the day of diagnosis to the day of relapse or progression of the NB, the date of death for any reason, or the last follow-up date (2). Overall survival (OS) was defined as the time from the day of diagnosis to the date of death for any reason (2).…”

Section: Data Collection and Evaluationmentioning

confidence: 99%

“…Neuroblastoma (NB) is the most common childhood extracranial solid tumor. Its characteristics include rapid progression, and it accounts for 8-10% of pediatric cancers (1,2). The clinical presentation of NB can be quite heterogeneous in terms of histology and genetics, ranging from asymptomatic incidental tumors to widespread metastases with systemic manifestations (3).…”

Section: Introductionmentioning

confidence: 99%

Impact of 11q Loss of Heterozygosity Status on the Response of High-Risk Neuroblastoma With MYCN Amplification to Neoadjuvant Chemotherapy

Duan

et al. 2022

Front. Pediatr.

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PurposeThe aim of this study was to investigate whether 11q loss of heterozygosity (LOH) aberration would impact the response of the primary tumor to neoadjuvant chemotherapy or to the degree of surgical resection in neuroblastoma (NB) patients with MYCN amplification.MethodsThe clinical data of 42 NB patients with MYCN amplification who were newly diagnosed and received treatments at our hospital from 2011 to 2020 were retrospectively analyzed. According to the results of the segmental chromosome aberration analysis, the patients enrolled were assigned to an 11qLOH positive group and an 11qLOH negative group.ResultsThere was no significant difference in the mean number of chemotherapy courses completed before surgery between the 11qLOH positive and 11qLOH negative groups (p = 0.242). Each of the 42 patients had metaiodobenzylguanidine (MIBG) scans both before and after neoadjuvant chemotherapy. The percentage of patients who had a clinical MIBG change in the 11qLOH positive group was lower than the percentage in the 11qLOH negative group (27.27 vs. 66.67%, p = 0.030). The 11qLOH negative group seemed to have a higher rate of surgical resection (≥90%); however, the difference between the two groups was not statistically significant (p = 0.088). Furthermore, the 11qLOH negative group did not show significantly superior event-free survival and overall survival rates compared with the 11qLOH positive group.ConclusionsThis study showed that patients with NB and MYCN amplification in combination with 11qLOH might be less likely to respond to neoadjuvant chemotherapy when compared with patients with NB and MYCN amplification without 11qLOH.

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“…Patients with refractory and/or recurrent NB have a poor prognosis, even after intensive treatment with chemotherapy, surgery, stem cell transplantation, radiation, and/or antibody-based therapy (Mallepalli et al 2019). Although metaiodobenzylguanidine (MIBG) and disialoganglioside 2 (GD2) monoclonal antibody treatment might be effective, they are difficult to access, resulting in worse prognosis of NB in China (Su et al 2020).…”

Section: Introductionmentioning

confidence: 99%

GD2-specific chimeric antigen receptor-modified T cells for the treatment of refractory and/or recurrent neuroblastoma in pediatric patients

Huang

Lin

et al. 2021

J Cancer Res Clin Oncol

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Purpose This study aimed to evaluate the safety and efficacy of chimeric antigen receptor (CAR) disialoganglioside 2 (GD2)-specific (4SCAR-GD2) T cells for treatment of refractory and/or recurrent neuroblastoma (NB) in pediatric patients. Experimental design A phase I clinical study using 4SCAR-GD2 T cells for the treatment of NB in pediatric patients was conducted. This study was registered at www.clinicaltrials.gov (NCT02765243). A lentiviral CAR with the signaling domains of CD28/4-1BB/CD3ζ-iCasp9 was transduced into activated T cells. The response to 4SCAR-GD2 T-cell treatment, and 4SCAR-GD2 T-cell expansion and persistence in patients were evaluated. Toxicities were determined based on the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) v4.03. Results Twelve patients were enrolled and finally ten patients were included in this clinical trial which started from January 1, 2016, to August 1, 2017. These patients had progressive disease (PD) before CAR T-cell infusion. After 4SCAR-GD2 T-cell treatment, 6 (6/10) had stable disease (SD) at 6 months, and 4 (4/10) remained SD at 1 year and alive after 3–4 years of follow-up. Six patients died due to disease progression by the end of July 1, 2020. The median overall survival (OS) time was 25 months (95% CI, 0.00–59.43), and the median progression-free survival (PFS) time was 8 months (95% CI, 0.25–15.75). Grade 3 or 4 hematological toxicities were the common adverse events frequently occurred after fludarabine and cyclophosphamide (Flu/cy) chemotherapy. Grade 1–2 toxicities such as cytokine release syndrome (CRS) and neuropathic pain were common, but were transient and mild. Conclusions The 4SCAR-GD2 T-cell therapy demonstrated antitumor effect and manageable toxicities, indicating its potential to benefit children with refractory and/or recurrent NB.

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Treatment and outcomes of 1041 pediatric patients with neuroblastoma who received multidisciplinary care in China

Abstract: , et al. Treatment and outcomes of 1041 pediatric patients with neuroblastoma who received multidisciplinary care in China.

Cited by 21 publications

References 24 publications

Safety and feasibility of laparoscopic resection of abdominal neuroblastoma without image-defined risk factors: a single-center experience

Safety and feasibility of laparoscopic resection of abdominal neuroblastoma without image-defined risk factors: a single-center experience

Impact of 11q Loss of Heterozygosity Status on the Response of High-Risk Neuroblastoma With MYCN Amplification to Neoadjuvant Chemotherapy

GD2-specific chimeric antigen receptor-modified T cells for the treatment of refractory and/or recurrent neuroblastoma in pediatric patients

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