Eflornithine for treating high-risk neuroblastoma with complete or partial response after immunotherapy [ID4060]

Neuroblastoma is a solid cancer of embryonic nerve cells called neural crest cells. It commonly occurs in the adrenal glands or in the nerve tissue of the sympathetic nervous system.

People may be classified as having high-risk neuroblastoma when they are older than 18 months of age their condition is metastatic or there is MYCN oncogene amplification and overexpression.(12)The initial symptoms of neuroblastoma are usually vague such as tiredness fever and loss of appetite. Specific symptoms depend on the location of the tumour.

Because neuroblastoma usually develops in the abdomen the most common symptom is an abdominal lump and children may also experience constipation or difficulty in passing urine.

The tumour may affect the chest or neck region and may cause breathlessness and difficulty in swallowing or a visible lump in the neck.Neuroblastoma usually affects children under the age of 5-years with a median age of diagnosis of 17 months.(3) Approximately 100 children are diagnosed with neuroblastoma in the UK each year.(4) Of these 40% may be considered high-risk. (2) High-risk neuroblastoma is associated with a 5-year survival rate of approximately 50%. (5)Treatment for high-risk disease is generally divided into 3 phases; induction consolidation and maintenance.

During induction and consolidation phases people in the high-risk category are initially treated with multi-agent chemotherapy surgery and radiotherapy followed by high-dose chemotherapy (which may cause severe or complete depletion of bone marrow cells; also known as myeloablative therapy) and autologous stem cell transplant.

Radiotherapy is usually also given after stem cell transplant. Following full treatment the maintenance phase aims to stop the cancer from coming back.

Standard of care in the maintenance phase is to treat for minimal residual disease with dinutuximab beta an immunotherapy-based regimen or isotretinoin.References1. Evageliou NF Haber M Vu A Laetsch TW Murray J Gamble LD et al. Polyamine Antagonist Therapies Inhibit Neuroblastoma Initiation and Progression.

Clinical Cancer Research. 2016 Aug 31; 22(17): 4391–404.2. Bassiri H Benavides A Haber M Gilmour SK Norris MD Hogarty MD. Translational development of difluoromethylornithine (DFMO) for the treatment of neuroblastoma. Translational Pediatrics [Internet]. 2015 Jul 1;4(3):226–38. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4729051/3.

Mahapatra S Challagundla KB. Neuroblastoma. [Updated 2023 Jul 10]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK448111/4. Neuroblastoma | Solving Kids’ Cancer UK [Internet]. www.solvingkidscancer.org.uk. Available from: https://www.solvingkidscancer.org.uk/neuroblastoma/5.

Neuroblastoma Cancer | American Cancer Society [Internet]. www.cancer.org. 2021. Available from: https://www.cancer.org/cancer/types/neuroblasta.html