Authors' objectives: APN311 is intended to be used as first line therapy for the treatment of high risk neuroblastoma in children and adolescents. If licensed, APN311 will offer an additional treatment option for such patients who currently have few effective therapies available. APN311 does not currently have Marketing Authorisation in the EU for any indication. Neuroblastoma is the most common extracranial solid tumour diagnosed in children, comprising 8-10% of all childhood cancers. It is most commonly diagnosed in children under the age of 5 years, with 90% of cases diagnosed in the first 10 years of life. The median age of diagnosis is 17 months. High risk neuroblastoma can be characterised by age (>1 year), disease which has spread, MYCN oncogene amplification, and unfavourable histopathologic findings. It is estimated that 40% of children presenting with the condition may be classified as high risk and research suggests that these patients have a five-year event-free survival of less than 50%. Treatment for patients with high risk neuroblastoma is generally divided into three phases: induction, consolidation and maintenance. The majority of UK patients are registered and treated on the SIOPEN HR-NB1 trial. Within this trial patients receive platinum-based rapid COJEC chemotherapy or a modified Kusher N7 regime in the induction phase. The consolidation phase may involve myeloablative chemotherapy and autologous stem cell transplant. Patients may also receive 21Gy radiotherapy to their primary tumour bed, followed by differentiation and immunotherapy in the maintenance phase. APN311 is currently in one phase III clinical trial comparing its effect on event free survival against treatment with APN311 and interleukin 2 combined. This trial is expected to complete in September 2016.

Project Status: Completed

Year Published: 2015

URL for published report: http://www.hsric.nihr.ac.uk/topics/apn311-for-high-risk-neuroblastoma-in-children-and-adolescents-first-line/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Horizon Scanning Centre

Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269

Contact Name: c.packer@bham.ac.uk

Contact Email: c.packer@bham.ac.uk

Copyright: NIHR Horizon Scanning Research&Intelligence Centre (NIHR HSRIC)