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Neuroblastoma

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1. Anti-GD2 antibody-containing immunotherapy postconsolidation therapy for people with high-risk neuroblastoma treated with autologous haematopoietic stem cell transplantation. (PubMed)

Anti-GD2 antibody-containing immunotherapy postconsolidation therapy for people with high-risk neuroblastoma treated with autologous haematopoietic stem cell transplantation. Neuroblastoma is a rare malignant disease that primarily affects children. The tumours mainly develop in the adrenal medullary tissue, and an abdominal mass is the most common presentation. High-risk disease is characterised by metastasis and other primary tumour characteristics resulting in increased risk for an adverse (...) outcome. The GD2 carbohydrate antigen is expressed on the cell surface of neuroblastoma tumour cells and is thus a promising target for anti-GD2 antibody-containing immunotherapy.To assess the efficacy of anti-GD2 antibody-containing postconsolidation immunotherapy after high-dose chemotherapy (HDCT) and autologous haematopoietic stem cell transplantation (HSCT) compared to standard therapy after HDCT and autologous HSCT in people with high-risk neuroblastoma. Our primary outcomes were overall

2019 Cochrane

2. Dinutuximab (Unituxin) - the treatment of high-risk neuroblastoma in pediatric patients

Dinutuximab (Unituxin) - the treatment of high-risk neuroblastoma in pediatric patients Search Page - Drug and Health Product Register Language selection Search and menus Search Search website Search Topics menu You are here: Summary Basis of Decision - - Health Canada Expand all Summary Basis of Decision (SBD) for Contact: Summary Basis of Decision (SBD) documents provide information related to the original authorization of a product. The for is located below. Recent Activity for SBDs written

2019 Health Canada - Drug and Health Product Register

3. Dinutuximab beta for treating neuroblastoma

Dinutuximab beta for treating neuroblastoma Dinutuximab beta for treating Dinutuximab beta for treating neuroblastoma neuroblastoma T echnology appraisal guidance Published: 22 August 2018 nice.org.uk/guidance/ta538 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent the view of NICE, arrived at after careful consideration (...) to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible. Dinutuximab beta for treating neuroblastoma (TA538) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 2 of 25Contents Contents 1 Recommendations 4 2 Information about dinutuximab beta 5 3 Committee discussion 7 The condition 7 Current treatments 7

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

5. Neuroblastoma

Neuroblastoma Neuroblastoma - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Neuroblastoma Last reviewed: February 2019 Last updated: September 2018 Summary Rare, malignant tumour arising from the embryological neural crest element of the peripheral sympathetic nervous system. The most common extracranial solid tumour in children; the majority of patients are diagnosed by 5 years of age. Diagnosis can usually (...) (high-risk) disease is difficult to cure, and is associated with extremely low survival. Definition A malignant tumour arising from the embryological neural crest element of the peripheral sympathetic nervous system. It most commonly arises from the adrenal gland(s), but can form anywhere that sympathetic nervous tissue is present, including paraspinal sympathetic ganglia in the chest and abdomen. Brodeur G, Hogarty M, Bagatell R, et al. Neuroblastoma. In: Pizzo P, Poplack D, eds. Principles

2018 BMJ Best Practice

6. Neuroblastoma

Neuroblastoma Neuroblastoma - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Neuroblastoma Last reviewed: February 2019 Last updated: September 2018 Summary Rare, malignant tumour arising from the embryological neural crest element of the peripheral sympathetic nervous system. The most common extracranial solid tumour in children; the majority of patients are diagnosed by 5 years of age. Diagnosis can usually (...) (high-risk) disease is difficult to cure, and is associated with extremely low survival. Definition A malignant tumour arising from the embryological neural crest element of the peripheral sympathetic nervous system. It most commonly arises from the adrenal gland(s), but can form anywhere that sympathetic nervous tissue is present, including paraspinal sympathetic ganglia in the chest and abdomen. Brodeur G, Hogarty M, Bagatell R, et al. Neuroblastoma. In: Pizzo P, Poplack D, eds. Principles

2018 BMJ Best Practice

7. Difluoromethylornithine for the Treatment of Neuroblastoma in the Pediatric Population: Clinical Effectiveness, Cost-Effectiveness, and Guidelines

Difluoromethylornithine for the Treatment of Neuroblastoma in the Pediatric Population: Clinical Effectiveness, Cost-Effectiveness, and Guidelines Difluoromethylornithine for the Treatment of Neuroblastoma in the Pediatric Population: Clinical Effectiveness, Cost-Effectiveness, and Guidelines | CADTH.ca Find the information you need Difluoromethylornithine for the Treatment of Neuroblastoma in the Pediatric Population: Clinical Effectiveness, Cost-Effectiveness, and Guidelines (...) Difluoromethylornithine for the Treatment of Neuroblastoma in the Pediatric Population: Clinical Effectiveness, Cost-Effectiveness, and Guidelines Last updated: October 5, 2018 Project Number: RA0971-000 Product Line: Research Type: Drug Report Type: Reference List Result type: Report Question What is the clinical effectiveness of difluoromethylornithine (DFMO) for the treatment or preventative treatment of neuroblastoma in the pediatric population? What is the cost-effectiveness of difluoromethylornithine (DFMO

2018 Canadian Agency for Drugs and Technologies in Health - Rapid Review

8. Unituxin for Neuroblastoma – Details

Unituxin for Neuroblastoma – Details Unituxin for Neuroblastoma – Details | CADTH.ca Find the information you need Unituxin for Neuroblastoma – Details Unituxin for Neuroblastoma – Details Project Number pCODR 10154 Brand Name Unituxin Generic Name Dinutuximab Strength 3.5 mg / mL Tumour Type Neurological Indication Neuroblastoma Funding Request To be used in combination with GM-CSF, IL-2 and Retinoic acid (RA) for the treatment of pediatric patients with high-risk neuroblastoma who achieve

2019 CADTH - Pan Canadian Oncology Drug Review

9. Retinoic acid postconsolidation therapy for high-risk neuroblastoma patients treated with autologous haematopoietic stem cell transplantation. (PubMed)

Retinoic acid postconsolidation therapy for high-risk neuroblastoma patients treated with autologous haematopoietic stem cell transplantation. Neuroblastoma is a rare malignant disease and mainly affects infants and very young children. The tumours mainly develop in the adrenal medullary tissue, with an abdominal mass as the most common presentation. About 50% of patients have metastatic disease at diagnosis. The high-risk group is characterised by metastasis and other features that increase (...) the risk of an adverse outcome. High-risk patients have a five-year event-free survival of less than 50%. Retinoic acid has been shown to inhibit growth of human neuroblastoma cells and has been considered as a potential candidate for improving the outcome of patients with high-risk neuroblastoma. This review is an update of a previously published Cochrane Review.To evaluate the efficacy and safety of additional retinoic acid as part of a postconsolidation therapy after high-dose chemotherapy (HDCT

2017 Cochrane

10. Iodine-131-meta-iodobenzylguanidine therapy for patients with newly diagnosed high-risk neuroblastoma. (PubMed)

Iodine-131-meta-iodobenzylguanidine therapy for patients with newly diagnosed high-risk neuroblastoma. Patients with newly diagnosed high-risk (HR) neuroblastoma (NBL) still have a poor outcome, despite multi-modality intensive therapy. This poor outcome necessitates the search for new therapies, such as treatment with 131I-meta-iodobenzylguanidine (131I-MIBG).To assess the efficacy and adverse effects of 131I-MIBG therapy in patients with newly diagnosed HR NBL.We searched the following (...) electronic databases: the Cochrane Central Register of Controlled Trials (CENTRAL; the Cochrane Library 2016, Issue 3), MEDLINE (PubMed) (1945 to 25 April 2016) and Embase (Ovid) (1980 to 25 April 2016). In addition, we handsearched reference lists of relevant articles and reviews. We also assessed the conference proceedings of the International Society for Paediatric Oncology, Advances in Neuroblastoma Research and the American Society of Clinical Oncology; all from 2010 up to and including 2015. We

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2017 Cochrane

11. Dinutuximab beta EUSA for the treatment of high-risk neuroblastoma

Dinutuximab beta EUSA for the treatment of high-risk neuroblastoma 1 Cost-effectiveness of dinutuximab beta (Qarziba®) for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment (...) of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures. In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, Qarziba® should be combined with interleukin-2 (IL-2). The NCPE has issued a recommendation regarding the cost-effectiveness of dinutuximab beta (Qarziba®). Following assessment of the applicant’s submission, the NCPE recommends that dinutuximab beta (Qarziba

2018 Pediatric Endocrine Society

12. LDHA in neuroblastoma is associated with poor outcome and its depletion decreases neuroblastoma growth independent of aerobic glycolysis. (PubMed)

LDHA in neuroblastoma is associated with poor outcome and its depletion decreases neuroblastoma growth independent of aerobic glycolysis. Purpose: To investigate whether lactate dehydrogenase A (LDHA), an important component of the LDH tetramer crucial for aerobic glycolysis, is associated with patient outcome and constitutes a therapeutic target in neuroblastoma (NB).Experimental Design: Expression of LDHA mRNA and protein was determined in 709 and 110 NB patient samples, respectively

2018 Clinical Cancer Research

13. Phase I study of vorinostat in combination with isotretinoin in patients with refractory/recurrent neuroblastoma: A new approaches to Neuroblastoma Therapy (NANT) trial (PubMed)

Phase I study of vorinostat in combination with isotretinoin in patients with refractory/recurrent neuroblastoma: A new approaches to Neuroblastoma Therapy (NANT) trial Vorinostat combined with retinoids produces additive antitumor effects in preclinical studies of neuroblastoma. Higher systemic exposures of vorinostat than achieved in pediatric phase I trials with continuous daily dosing are necessary for in vivo increased histone acetylation and cytotoxic activity. We conducted a phase I (...) trial in children with relapsed/refractory neuroblastoma to determine the maximum tolerated dose (MTD) of vorinostat on an interrupted schedule, escalating beyond the previously identified pediatric MTD.Isotretinoin (cis-13-retinoic acid) 80 mg/m2 /dose was administered by mouth twice daily on days 1-14 in combination with escalating doses of daily vorinostat up to 430 mg/m2 /dose (days 1-4; 8-11) in each 28-day cycle using the standard 3 + 3 design. Vorinostat pharmacokinetic testing and histone

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2018 Pediatric blood & cancer

14. Heterogeneous MYCN amplification in neuroblastoma: a SIOP Europe Neuroblastoma Study. (PubMed)

Heterogeneous MYCN amplification in neuroblastoma: a SIOP Europe Neuroblastoma Study. In neuroblastoma (NB), the most powerful prognostic marker, the MYCN amplification (MNA), occasionally shows intratumoural heterogeneity (ITH), i.e. coexistence of MYCN-amplified and non-MYCN-amplified tumour cell clones, called heterogeneous MNA (hetMNA). Prognostication and therapy allocation are still unsolved issues.The SIOPEN Biology group analysed 99 hetMNA NBs focussing on the prognostic significance

2018 British Journal of Cancer

15. Guidelines on nuclear medicine imaging in neuroblastoma

Guidelines on nuclear medicine imaging in neuroblastoma Guidelines on nuclear medicine imaging in neuroblastoma | SpringerLink This service is more advanced with JavaScript available, learn more at Advertisement Hide Search SpringerLink October 2018 , Volume 45, , pp 2009–2024 | Guidelines on nuclear medicine imaging in neuroblastoma Authors Zvi Bar-Sever Lorenzo Biassoni Barry Shulkin Grace Kong Michael S. Hofman Egesta Lopci Irina Manea Jacek Koziorowski Rita Castellani Ariane Boubaker Bieke (...) Lambert Thomas Pfluger Helen Nadel Susan Sharp Francesco Giammarile Guidelines First Online: 25 June 2018 1.4k Downloads Abstract Nuclear medicine has a central role in the diagnosis, staging, response assessment and long-term follow-up of neuroblastoma, the most common solid extracranial tumour in children. These EANM guidelines include updated information on 123 I-mIBG, the most common study in nuclear medicine for the evaluation of neuroblastoma, and on PET/CT imaging with 18 F-FDG, 18 F-DOPA

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2018 European Association of Nuclear Medicine

16. Interleukin 2 with anti-GD2 antibody ch14.18/CHO (dinutuximab beta) in patients with high-risk neuroblastoma (HR-NBL1/SIOPEN): a multicentre, randomised, phase 3 trial

Interleukin 2 with anti-GD2 antibody ch14.18/CHO (dinutuximab beta) in patients with high-risk neuroblastoma (HR-NBL1/SIOPEN): a multicentre, randomised, phase 3 trial Immunotherapy with the chimeric anti-GD2 monoclonal antibody dinutuximab, combined with alternating granulocyte-macrophage colony-stimulating factor and intravenous interleukin-2 (IL-2), improves survival in patients with high-risk neuroblastoma. We aimed to assess event-free survival after treatment with ch14.18/CHO (dinutuximab (...) beta) and subcutaneous IL-2, compared with dinutuximab beta alone in children and young people with high-risk neuroblastoma.We did an international, open-label, phase 3, randomised, controlled trial in patients with high-risk neuroblastoma at 104 institutions in 12 countries. Eligible patients were aged 1-20 years and had MYCN-amplified neuroblastoma with stages 2, 3, or 4S, or stage 4 neuroblastoma of any MYCN status, according to the International Neuroblastoma Staging System. Patients were

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2019 EvidenceUpdates

17. Effect of Tandem Autologous Stem Cell Transplant vs Single Transplant on Event-Free Survival in Patients With High-Risk Neuroblastoma: A Randomized Clinical Trial. (PubMed)

Effect of Tandem Autologous Stem Cell Transplant vs Single Transplant on Event-Free Survival in Patients With High-Risk Neuroblastoma: A Randomized Clinical Trial. Induction chemotherapy followed by high-dose therapy with autologous stem cell transplant and subsequent antidisialoganglioside antibody immunotherapy is standard of care for patients with high-risk neuroblastoma, but survival rate among these patients remains low.To determine if tandem autologous transplant improves event-free (...) survival (EFS) compared with single transplant.Patients were enrolled in this randomized clinical trial from November 2007 to February 2012 at 142 Children's Oncology Group centers in the United States, Canada, Switzerland, Australia, and New Zealand. A total of 652 eligible patients aged 30 years or younger with protocol-defined high-risk neuroblastoma were enrolled and 355 were randomized. The final date of follow-up was June 29, 2017, and the data analyses cut-off date was June 30, 2017.Patients

2019 JAMA

18. Feasibility, toxicity and response of upfront metaiodobenzylguanidine therapy therapy followed by German Pediatric Oncology Group Neuroblastoma 2004 protocol in newly diagnosed stage 4 neuroblastoma patients. (PubMed)

Feasibility, toxicity and response of upfront metaiodobenzylguanidine therapy therapy followed by German Pediatric Oncology Group Neuroblastoma 2004 protocol in newly diagnosed stage 4 neuroblastoma patients. Radiolabelled meta-iodobenzylguanidine (MIBG) is an effective option in treatment of neuroblastoma (NBL) tumours. We studied feasibility, toxicity and efficacy of upfront 131I-MIBG and induction treatment in stage 4 NBL patients.Retrospective, multi-centre (AMC and EMC) pilot regimen (1/1

2017 European Journal of Cancer

19. Neuroblastoma survivors are at increased risk for second malignancies: A report from the International Neuroblastoma Risk Group Project. (PubMed)

Neuroblastoma survivors are at increased risk for second malignancies: A report from the International Neuroblastoma Risk Group Project. The incidence of second malignant neoplasm (SMN) within the first ten years of diagnosis in high-risk neuroblastoma patients treated with modern, intensive therapy is unknown. Further, the underlying germline genetics that contribute to SMN in these survivors are not known.The International Neuroblastoma Risk Group (INRG) database of patients diagnosed from (...) 1990 to 2010 was analysed. SMN risk was accessed by cumulative incidence, standardised incidence ratios (SIRs) and absolute excess risk. A candidate gene-based association study evaluated genetic susceptibility to SMN in neuroblastoma survivors.Of the 5987 patients in the INRG database with SMN data enrolled in a clinical trial, 43 (0.72%) developed a SMN. The 10-year cumulative incidence of SMN for high-risk patients was 1.8% (95% confidence interval [CI] 1.0-2.6%) compared with 0.38% (95% CI

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2017 European Journal of Cancer

20. Expression of Five Neuroblastoma Genes in Bone Marrow or Blood of Patients with Relapsed/Refractory Neuroblastoma Provides a New Biomarker for Disease and Prognosis. (PubMed)

Expression of Five Neuroblastoma Genes in Bone Marrow or Blood of Patients with Relapsed/Refractory Neuroblastoma Provides a New Biomarker for Disease and Prognosis. Purpose: We determined whether quantifying neuroblastoma-associated mRNAs (NB-mRNAs) in bone marrow and blood improves assessment of disease and prediction of disease progression in patients with relapsed/refractory neuroblastoma.Experimental Design: mRNA for CHGA, DCX, DDC, PHOX2B, and TH was quantified in bone marrow and blood (...) independently with PFS in relapsed/refractory neuroblastoma. Clin Cancer Res; 23(18); 5374-83. ©2017 AACR.©2017 American Association for Cancer Research.

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2017 Clinical Cancer Research

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