Phase II Study on the Effect of Disease Sites, Age, and Prior Therapy on Response to Iodine-131-Metaiodobenzylguanidine Therapy in Refractory Neuroblastoma

Matthay, Katherine K.; Yanik, Gregory A.; Messina, Julia A.; Quach, Alekist; Huberty, John P.; Cheng, Su–Chun; Veatch, Janet; Goldsby, Robert E.; Brophy, Patricia; Kersun, Leslie S.; Hawkins, Randall A.; Maris, John M.

doi:10.1200/jco.2006.09.3484

Cited by 231 publications

(206 citation statements)

References 23 publications

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“…In a large phase II study, 164 patients with relapsed or refractory neuroblastoma were treated with 131 I-MIBG. Approximately one-third of patients (36%) had evidence of clinical response; with approximately one-third (34%) having stable disease for a median of 6 months [117], leading to efforts to include 131 I-MIBG therapy as a part of upfront consolidation treatment for children with high-risk neuroblastoma. Additional clinical trials are ongoing through the New Agents in Neuroblastoma Therapy (NANT) consortium to identify the best of anticancer agents to combine with MIBG therapy (NCT02035137).…”

Section: Treatment -Relapsed and Refractory Neuroblastomamentioning

confidence: 99%

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: 99%

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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“…It is thought that emission of ionising radiation at this site results in tumour decay (Mukherjee et al, 2001;Rose et al, 2003;Pasieka et al, 2004). This therapy has been used to treat NETs of various types, including gastroenteropancreatic NETs (Pathirana et al, 2001;Pasieka et al, 2004;Safford et al, 2004;Sywak et al, 2004;Buscombe et al, 2005), paraglangliomas (Mukherjee et al, 2001;Safford et al, 2003;Fitzgerald et al, 2006), phaeochromocytomas (Castellani et al, 2000;Rose et al, 2003;Safford et al, 2004;Fitzgerald et al, 2006), medullary carcinoma of the thyroid (Castellani et al, 2000(Castellani et al, , 2003Mukherjee et al, 2001) and neuroblastomas (Howard et al, 2005;Matthay et al, 2007). A number of previous studies had demonstrated significant symptomatic benefit in 40 -60% of patients with metastatic NETs following 131 I-MIBG therapy (Safford et al, 2004;Sywak et al, 2004), in a safe and cost-effective manner (Pathirana et al, 2001).…”

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confidence: 99%

Assessment of the efficacy and toxicity of 131I-metaiodobenzylguanidine therapy for metastatic neuroendocrine tumours

et al. 2008

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131 I-metaiodobenzylguanidine ( 131 I-MIBG) is a licensed palliative treatment for patients with metastatic neuroendocrine tumours. We have retrospectively assessed the consequences of 131 I-MIBG therapy in 48 patients (30 gastroenteropancreatic, 6 pulmonary, 12 unknown primary site) with metastatic neuroendocrine tumours attending Royal Liverpool University Hospital between 1996 and 2006. Mean age at diagnosis was 57.6 years (range 34 -81). 131 I-MIBG was administered on 88 occasions (mean 1.8 treatments, range 1 -4). Twenty-nine patients had biochemical markers measured before and after 131 I-MIBG, of whom 11 (36.7%) showed 450% reduction in levels post-therapy. Forty patients had radiological investigations performed after 131 I-MIBG, of whom 11(27.5%) showed reduction in tumour size post-therapy. Twenty-seven (56.3%) patients reported improved symptoms after 131 I-MIBG therapy. Kaplan -Meier analysis showed significantly increased survival (P ¼ 0.01) from the date of first 131 I-MIBG in patients who reported symptomatic benefit from therapy. Patients with biochemical and radiological responses did not show any statistically significant alteration in survival compared to non-responders. Eleven (22.9%) patients required hospitalisation as a consequence of complications, mostly due to mild bone marrow suppression. 131 I-MIBG therefore improved symptoms in more than half of the patients with metastatic neuroendocrine tumours and survival was increased in those patients who reported a symptomatic response to therapy.

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“…With the widespread use of therapeutic 131 I-mIBG as a targeted radiopharmaceutical treatment of neuroblastoma both in newly diagnosed and relapsed patients, diagnostic mIBG scans will determine the eligibility for this modality (Garaventa et al, 1999;Gaze et al, 2005;Matthay et al, 2006Matthay et al, , 2007de Kraker et al, 2008;DuBois and Matthay, 2008). Previous studies have indicated that a positive mIBG scan after induction chemotherapy or just before myeloablative therapy may be a prognostic marker for a high likelihood of relapse (Ladenstein et al, 1998;Perel et al, 1999;Schmidt et al, 2008).…”

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confidence: 99%

Criteria for evaluation of disease extent by 123I-metaiodobenzylguanidine scans in neuroblastoma: a report for the International Neuroblastoma Risk Group (INRG) Task Force

et al. 2010

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BACKGROUND: Neuroblastoma is an embryonic tumour of the sympathetic nervous system, metastatic in half of the patients at diagnosis, with a high preponderance of osteomedullary disease, making accurate evaluation of metastatic sites and response to therapy challenging. Metaiodobenzylguanidine (mIBG), taken into cells via the norepinephrine transporter, provides a sensitive and specific method of assessing tumour in both soft tissue and bone sites. The goal of this report was to develop consensus guidelines for the use of mIBG scans in staging, response assessment and surveillance in neuroblastoma. METHODS: The International Neuroblastoma Risk Group (INRG) Task Force, including a multidisciplinary group in paediatric oncology of North and South America, Europe, Oceania and Asia, formed a subcommittee on metastatic disease evaluation, including expert nuclear medicine physicians and oncologists, who developed these guidelines based on their experience and the medical literature, with approval by the larger INRG Task Force. RESULTS: Guidelines for patient preparation, radiotracer administration, techniques of scanning including timing, energy, specific views, and use of single photon emission computed tomography are included. Optimal timing of scans in relation to therapy and for surveillance is reviewed. Validated semi-quantitative scoring methods in current use are reviewed, with recommendations for use in prognosis and response evaluation. CONCLUSIONS: Metaiodobenzylguanidine scans are the most sensitive and specific method of staging and response evaluation in neuroblastoma, particularly when used with a semi-quantitative scoring method. Use of the optimal techniques for mIBG in staging and response, including a semi-quantitative score, is essential for evaluation of the efficacy of new therapy.

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Phase II Study on the Effect of Disease Sites, Age, and Prior Therapy on Response to Iodine-131-Metaiodobenzylguanidine Therapy in Refractory Neuroblastoma

Abstract: The high response rate and low nonhematologic toxicity with 131I-MIBG suggest incorporation of this agent into initial multimodal therapy of neuroblastoma.

Cited by 231 publications

References 23 publications

Overview and recent advances in the treatment of neuroblastoma

Overview and recent advances in the treatment of neuroblastoma

Assessment of the efficacy and toxicity of 131I-metaiodobenzylguanidine therapy for metastatic neuroendocrine tumours

Criteria for evaluation of disease extent by 123I-metaiodobenzylguanidine scans in neuroblastoma: a report for the International Neuroblastoma Risk Group (INRG) Task Force

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