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• #1 Treatment and prognosis of neuroblastoma – UpToDate

  https://www.uptodate.com/contents/treatment-and-prognosis-of-neuroblastoma

  Treatment and prognosis of neuroblastoma […] Neuroblastomas, which account for 97 percent of all neuroblastic tumors, are clinically heterogeneous, varying in location, histopathologic appearance, and biologic characteristics. They are most remarkable for their broad spectrum of clinical behavior, which can range from spontaneous regression to maturation to a benign ganglioneuroma, or aggressive disease with metastatic dissemination leading to death. Clinical diversity correlates closely with numerous clinical and biologic factors, although its molecular basis remains largely unknown. For example, most infants with disseminated disease have a favorable outcome after treatment with chemotherapy and surgery, while approximately half of children over the age of 18 months who have advanced neuroblastoma die from progressive disease despite intensive multimodality therapy.

• #2 Predicting Neuroblastoma Patient Risk Groups, Outcomes, and Treatment Response Using Machine Learning Methods: A Review

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10801560/

  Risk stratification in NB patients divides cases into low, intermediate, and high-risk groups based on multiple parameters such as histological features, MYCN amplification, DNA ploidy, age, chromosomal alterations (including alterations to 11q), differentiation status, and stage at diagnosis. Risk groups bear substantial differences in prognoses; for example, low- and intermediate-risk groups may display an overall survival (OS) of 95%, while high-risk cases will display a dismal survival of less than 50% and may experience a higher rate of recurrence despite treatment. […] Many studies have used machine learning (ML) and statistical methods to develop multi-gene models or other predictor models to anticipate patient subgrouping, risk, outcome (including survival, relapse, and recurrence), and treatment response.

• #3 Predicting Neuroblastoma Patient Risk Groups, Outcomes, and Treatment Response Using Machine Learning Methods: A Review

  https://www.mdpi.com/2076-3271/12/1/5

  Neuroblastoma, a paediatric malignancy with high rates of cancer-related morbidity and mortality, is of significant interest to the field of paediatric cancers. High-risk NB tumours are usually metastatic and result in survival rates of less than 50%. […] Risk stratification in NB patients divides cases into low, intermediate, and high-risk groups based on multiple parameters such as histological features, MYCN amplification, DNA ploidy, age, chromosomal alterations (including alterations to 11q), differentiation status, and stage at diagnosis. Risk groups bear substantial differences in prognoses; for example, low- and intermediate-risk groups may display an overall survival (OS) of 95%, while high-risk cases will display a dismal survival of less than 50% and may experience a higher rate of recurrence despite treatment.

• #4 Neuroblastoma Survival Rates | American Cancer Society | American Cancer Society

  https://www.cancer.org/cancer/types/neuroblastoma/detection-diagnosis-staging/survival-rates.html

  Survival rates are a way to get an idea of the outlook for children with a certain type of cancer. They cant tell you for sure if treatment will be successful, but they may help give you a better understanding of how likely this is. […] The survival rates below are based on the risk group of the childs cancer. The risk group, in turn, is based on the stage (extent) of the cancer, as well as other prognostic factors (such as the childs age). But other factors can also affect a childs outlook, such as the location of the tumor and how well the cancer responds to treatment. Your childs doctor can tell you how the numbers below might apply to your child, as they know your situation best. […] Children in the low-risk group have a 5-year survival rate that is higher than 95%. […] Children in the intermediate-risk group have a 5-year survival rate of around 90% to 95%. […] Children in the high-risk group have a 5-year survival rate of around 50%.

• #5 Survival statistics for neuroblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/neuroblastoma/prognosis-and-survival/survival-statistics

  Survival statistics for neuroblastoma are very general estimates and must be interpreted very carefully. These statistics are based on the experience of groups of children and cannot be used to predict a particular child’s chances of survival. […] The 5-year observed survival for neuroblastoma in children 0 to 14 years of age is 81%. This means that, on average, 81% of children diagnosed with neuroblastoma are expected to live at least 5 years after their diagnosis. […] Survival varies with each risk group of neuroblastoma. […] Generally, younger children with neuroblastoma have a better outcome. […] The actual survival rates change during treatment depending on how the tumour responds to treatment. […] Only a doctor familiar with these factors can put all of this information together with survival statistics to arrive at a prognosis.

• #6 Pediatric Neuroblastoma: Practice Essentials, Pathophysiology, Etiology

  https://emedicine.medscape.com/article/988284-overview

  According to the SEER data, the overall 5-year survival rate for children with neuroblastoma has improved from 24% in 1960-1963 to 55% in 1985-1994. […] Most patients with neuroblastoma present with disseminated disease, which confers a poor prognosis and is associated with a high mortality rate. Tumors in these patients usually have unfavorable pathologic and/or molecular features. The 3-year EFS for high-risk patients treated with conventional chemotherapy, radiation therapy, and surgery is less than 20%.

• #7 Prognosis and survival for neuroblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/neuroblastoma/prognosis-and-survival

  If your child has neuroblastoma, you will have questions about their prognosis. A prognosis is the doctor’s best estimate of how cancer will affect someone and how it will respond to treatment. Prognosis and survival depend on many factors. Only a doctor familiar with your child’s medical history, the type, stage and other features of the cancer, the treatments chosen and the response to treatment can put all of this information together with survival statistics to arrive at a prognosis. […] The following are prognostic and predictive factors for neuroblastoma. […] Children younger than 18 months of age usually have a better outcome. […] The earlier the stage at diagnosis, the better the prognosis. […] High-risk neuroblastoma that doesn’t respond well to initial induction chemotherapy (for example, if neuroblastoma remains in the bone marrow or if there is little improvement in the extent of disease shown on an MIBG scan) often has a poorer prognosis.

• #8 Pediatric Neuroblastoma: Practice Essentials, Pathophysiology, Etiology

  https://emedicine.medscape.com/article/988284-overview

  Neuroblastoma is the most common extracranial solid tumor of infancy. […] Age, stage, and biological features encountered in tumor cells are important prognostic factors and are used for risk stratification and treatment assignment. The differences in outcome for patients with neuroblastoma are striking. Patients with low-risk and intermediate-risk neuroblastoma have excellent prognosis and outcome. However, those with high-risk disease continue to have very poor outcomes despite intensive therapy. […] Stage of the tumor at the time of diagnosis and age of the patient are the most important prognostic factors. Although patients with localized tumors (regardless of age) have an excellent outcome (80-90% 3-year event-free survival [EFS] rate), patients older than 18 months with metastatic disease fare poorly. Generally, more than 50% of patients present with metastatic disease at the time of diagnosis, 20-25% have localized disease, 15% have regional extension, and approximately 7% present during infancy with disseminated disease limited to the skin, liver, and bone marrow (stage 4S).

• #9 Neuroblastoma | Nature Reviews Disease Primers

  https://www.nature.com/articles/nrdp201678

  Neuroblastoma is the most common extracranial solid tumour occurring in childhood and has a diverse clinical presentation and course depending on the tumour biology. […] The most malignant tumours have amplification of the MYCN oncogene (encoding a transcription factor), which is usually associated with poor survival, even in localized disease. […] Segmental chromosome alterations are frequent in neuroblastoma and are associated with worse outcome. […] Risk-stratified therapy has facilitated the reduction of therapy for children with low-risk and intermediate-risk disease. […] Advances in therapy for patients with high-risk disease include intensive induction chemotherapy and myeloablative chemotherapy, followed by the treatment of minimal residual disease using differentiation therapy and immunotherapy; these have improved 5-year overall survival to 50%.

• #10 Prognosis and survival for neuroblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/neuroblastoma/prognosis-and-survival

  The lower the risk group, the better the prognosis. Low-risk neuroblastoma responds better to treatment, requires less treatment and is less likely to come back (recur) than high-risk neuroblastoma. […] Hyperdiploid (DNA index 1) tumours tend to respond better to treatment and usually have a better prognosis. […] A single copy of the gene (described as non-amplified) is normal and is linked with a less aggressive form of cancer. […] Tumours with segmental chromosomal aberrations have a less favourable prognosis than tumours with only numerical abnormalities. […] Survival statistics for neuroblastoma are very general estimates. Survival is different for each risk group.

• #11 Neuroblastoma in Vietnam: A retrospective analysis of MYCN status and clinical features to inform prognosis and improve outcome

  https://www.oatext.com/neuroblastoma-in-vietnam-a-retrospective-analysis-of-mycn-status-and-clinical-features-to-inform-prognosis-and-improve-outcome.php

  This study demonstrates poor outcome for NB patients in Vietnam. Furthermore, prognosis is significantly worsened by their MYCN status; NB stage and age at diagnosis. […] MYCN amplification accounted for 17.69% of the total cohort in Vietnam. The result is relatively consistent with previous studies in Europe and Thailand in that there was a strong association of MYCN amplification with poorer survival rate in NB. MYCN-amplification in NB associated with decreased OS and EFS rates to 13.04% 7.02% and 28.12% 13.21%, respectively after 5-year observation; while the study in Europe found a higher percentage of survival rates (72% 7% for OS and 53% 8% for EFS). In multivariate analysis, MYCN was independently associated with a decrease in OS and EFS; compatible with the findings of studies in Europe and Thailand. […] MYCN status and INSS stage significantly associated with OS and EFS in both univariate and multivariate analyses. Multivariate analysis illustrated that MYCN status predicted OS and EFS independently of gender, age and INSS stage of patients.

• #12 Nomogram for predicting overall survival in children with neuroblastoma based on SEER database

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7406400/

  This study was performed to establish and validate a nomogram for predicting the overall survival in children with neuroblastoma. […] The nomogram possess the potential to be a new tool in predicting the survival rate of neuroblastoma patients. […] By mining the SEER database, we, for the first time, established and validated a nomogram that could facilitate a survival rate prediction for patients with NB. This predictive modelling involved 8 prognostic factors; age, sex, race, grade, radiotherapy, chemotherapy, tumor site, and tumor size. […] The novel predictive nomogram integrated with multiple variables including age, sex, race, tumor grade, radiotherapy, chemotherapy, tumor site, and tumor size from SEER possess the potential to be a new tool in predicting the survival rate of NB patients. […] The nomogram could accurately predict 1-, 3-, and 5-year overall survival of patients with NB. […] All these results confirmed that the nomogram had considerable prognostic potential, which was validated internally and externally.

• #13 Predicting Neuroblastoma Patient Risk Groups, Outcomes, and Treatment Response Using Machine Learning Methods: A Review

  https://www.mdpi.com/2076-3271/12/1/5

  Many studies have used machine learning (ML) and statistical methods to develop multi-gene models or other predictor models to anticipate patient subgrouping, risk, outcome (including survival, relapse, and recurrence), and treatment response. […] One example of a multi-gene predictor model is a study that identified a 59-gene signature to predict NB patient survival. […] Moreover, ML has been applied to a multitude of patient data, including multi-omics (genomic, transcriptomic, and methylome-sequencing data) in addition to microarray data and histological and medical imaging data to establish risk and predict outcomes and survival and treatment outcomes. […] Many studies use ML tools for patient outcome prediction. […] Given this background, in this study, the utility of multi-omics, histological, and radiological data for ML processing was discussed. Finally, studies reporting data processing by ML to stratify risk groups and predict outcomes, survival, and treatment response were examined.

• #14 Predicting Neuroblastoma Patient Risk Groups, Outcomes, and Treatment Response Using Machine Learning Methods: A Review

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10801560/

  One example of a multi-gene predictor model is a study that identified a 59-gene signature to predict NB patient survival. […] Moreover, ML has been applied to a multitude of patient data, including multi-omics (genomic, transcriptomic, and methylome-sequencing data) in addition to microarray data and histological and medical imaging data to establish risk and predict outcomes and survival and treatment outcomes. […] Many studies use ML tools for patient outcome prediction. […] Given this background, in this study, the utility of multi-omics, histological, and radiological data for ML processing was discussed. Finally, studies reporting data processing by ML to stratify risk groups and predict outcomes, survival, and treatment response were examined. […] The authors then performed literature mining and found that the expression of six genes, including ALK, BIRC5, MYCN, CCND1, NTRK1, and PHOX2B, distinguished four clusters.

• #15 Predicting Neuroblastoma Patient Risk Groups, Outcomes, and Treatment Response Using Machine Learning Methods: A Review

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10801560/

  In conclusion, this study identified a 6-gene signature for NB classification. […] The study also utilised KaplanMeier survival plots and Cox regression. […] In conclusion, this study utilised statistical and ML methods to extract features of DNA methylome data to retrieve signatures predictive of MYCN amplification status and patient prognosis, which may be useful for early patient diagnosis and risk stratification. […] In conclusion, the proposed method extracted valuable information, performed well, and may be able to assist clinical judgements.

• #16 Predicting clinical outcomes in neuroblastoma with genomic data integration | Biology Direct | Full Text

  https://biologydirect.biomedcentral.com/articles/10.1186/s13062-018-0223-8

  Current risk group definitions are problematic as patients within the same risk group can still show variable prognosis. […] One promising direction is to utilize the recently available genomic datasets to discover complex molecular markers that can improve patient stratification. […] Our supervised model can accurately predict overall survival and event-free survival profiles of neuroblastoma patients in independent cohorts. […] We observed that the prognosis of HR patients is harder to predict. One strategy to improve the prediction performance of HR patients is to focus on only HR patients in the training set. […] The clusters that we identified in both patient groups indicates that the current stratification of patients to high risk and low risk groups can be improved via the integrated use of genomic datasets. […] Both supervised and unsupervised approaches can contribute to improved treatment stratification of neuroblastoma patients.

• #17 Predicting clinical outcomes in neuroblastoma with genomic data integration | Biology Direct | Full Text

  https://biologydirect.biomedcentral.com/articles/10.1186/s13062-018-0223-8

  Neuroblastoma is a heterogeneous disease with diverse clinical outcomes. Current risk group models require improvement as patients within the same risk group can still show variable prognosis. […] Our supervised model trained on the SEQC cohort can accurately predict overall survival and event-free survival profiles of patients in two independent cohorts. […] Our results indicate that predicting OS and EFS is more challenging for high risk (HR) patients. […] Altogether, our results indicate that integration of multiple genomic characterizations enables the discovery of subtypes that improve over existing definitions of risk groups. Effective prediction of survival times will have a direct impact on choosing the right therapies for patients. […] Accurate prediction of disease prognosis is critical to improve the choice of therapies.

• #18 Neuroblastoma arises in early fetal development and its evolutionary duration predicts outcome | Nature Genetics

  https://www.nature.com/articles/s41588-023-01332-y

  Neuroblastoma, the most frequent solid tumor in infants, shows very diverse outcomes from spontaneous regression to fatal disease. […] We find in the discovery cohort (n=100), and validate in an independent cohort (n=86), that the duration of evolution is an accurate predictor of outcome. […] Thus, insight into neuroblastoma evolution may prospectively guide treatment decisions. […] A striking feature of neuroblastoma is the wide spectrum of clinical outcomes, ranging from low-risk cases requiring light or no treatment to high-risk disease that remains fatal for ~50% of patients. […] Nevertheless, the prospective stratification of patients into observation and different treatment groups remains a formidable challenge. […] Remarkably, early MRCA timing clearly identified cases with long event-free survival and long overall survival.

• #19 Sensitive liquid biopsy monitoring correlates with outcome in the prospective international GPOH-DCOG high-risk neuroblastoma RT-qPCR validation study | Journal of Experimental & Clinical Cancer Research | Full Text

  https://jeccr.biomedcentral.com/articles/10.1186/s13046-024-03261-y

  Liquid biopsies offer less burdensome sensitive disease monitoring. Bone marrow (BM) metastases, common in various cancers including neuroblastoma, is associated with poor outcomes. In pediatric high-risk neuroblastoma most patients initially respond to treatment, but in the majority the disease recurs with only 40% long-term survivors, stressing the need for more sensitive detection of disseminated disease during therapy. […] BM infiltration 10% by RT-qPCR at diagnosis was prognostic for survival (adjusted hazard ratio (HR) 1.82 [95%CI 1.25-2.63] and 2.04 [1.33-3.14] for EFS and OS, respectively). Any post-induction RT-qPCR positivity correlated with poor EFS and OS, with a HR of 2.10 [1.27-3.49] and 1.76 [1.01-3.08] and 5-years EFS of 26.6% [standard error 5.2%] versus 60.4% [6.7] and OS of 43.8% [5.9] versus 65.7% [6.6] for RT-qPCR-positive patients versus RT-qPCR-negative patients.

• #20 Neuroblastoma | Nature Reviews Disease Primers

  https://www.nature.com/articles/nrdp201678

  Currently, new approaches targeting the noradrenaline transporter, genetic pathways and the tumour microenvironment hold promise for further improvements in survival and long-term quality of life. […] This is a comprehensive regression tree analysis of 13 potential prognostic factors on an international cohort of 8,800 patients with neuroblastoma, establishing the current basis of international risk stratification. […] This seminal report of a multicentre randomized controlled study showed the significant improvement in EFS for children treated with myeloablative chemoradiotherapy and purged AHSCT compared with standard-dose chemotherapy, and for subsequent treatment with isotretinoin maintenance compared with no further therapy, with current incorporation into standard care for high-risk neuroblastoma.

• #21 Prediction of Composite Clinical Outcomes for Childhood Neuroblastoma Using Multi-Omics Data and Machine Learning

  https://www.mdpi.com/1422-0067/26/1/136

  Neuroblastoma is a common malignant tumor in childhood that seriously endangers the health and lives of children, making it essential to find effective prognostic markers to accurately predict their clinical outcomes. […] Accurately predicting the clinical outcomes for neuroblastoma patients not only helps patients know about their life expectancy but also helps clinicians make well-founded decision and further develop appropriate treatments. […] The event we are interested in is a composite clinical outcome of 5-year survival time and vital status (i.e., death or not) for neuroblastoma patients, which constitute two binary classification tasks. […] The reliability and performance of predicting clinical outcomes can be improved. […] In this study, we aimed to investigate composite clinical outcomes, i.e., survival time and vital status, by integrating clinical data and two types of multi-omics data, i.e., gene expression and DNA methylation data.

• #22 Treatment and prognosis of neuroblastoma – UpToDate

  https://www.uptodate.com/contents/treatment-and-prognosis-of-neuroblastoma

  The treatment and prognosis of neuroblastoma will be reviewed here and is meant to be an overview for the general oncologist. Due to the rarity of this disease, patients should be managed in a setting where appropriate expertise in the treatment of neuroblastoma is available. […] There is international consensus to use the International Neuroblastoma Risk Group Staging System (INRGSS) to describe the extent of disease for neuroblastoma. This topic is organized using a risk-stratified approach that incorporates the INRGSS, along with molecular, pathologic, and other clinical characteristics.

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