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Paeds Vivashaematology-oncology-and-transfusion

Paeds Vivas · haematology-oncology-and-transfusion

Neuroblastoma: Viva

Branching clinical structured oral on neuroblastoma in children, covering the neural crest origin and the catecholamine secretion, the International Neuroblastoma Risk Group staging with the image-defined risk factors, the MYCN amplification as the most powerful prognostic marker, the iodine-123 metaiodobenzylguanidine scan, the opsoclonus-myoclonus-ataxia syndrome, the risk-adapted treatment from observation to the intensive multimodal therapy, and the spontaneous regression of stage MS.

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Prompt
A two-year-old girl presents with a firm abdominal mass crossing the midline, irritability, and bilateral periorbital ecchymosis. The urinary vanillylmandelic acid and homovanillic acid are markedly elevated. The examiner asks how you frame the problem, how you stage and risk-stratify the disease, what the MYCN amplification means for the prognosis, and how your approach changes for an infant with the opsoclonus-myoclonus-ataxia syndrome.

This is a branching oral built to probe the reasoning that holds the risk stratification and the paraneoplastic syndrome at the centre, and to expose the candidate who has memorised the headline without the safety-critical corners. The questions escalate from the framing to the staging, the molecular markers, the treatment pathway and the paraneoplastic syndrome, with deliberate probes into the spontaneous regression and the late effects. [1]

Opening question: framing the problem

The examiner opens with the mass and the periorbital ecchymosis and asks: how do you frame this problem in a single sentence, and what is your first step? [1]

A strong answer names the neuroblastoma from the abdominal mass crossing the midline, the catecholamine secretion and the periorbital ecchymosis of the orbital metastasis, and states that the first step is the confirmation of the catecholamine secretion and the staging of the disease extent. [1]

Model answer. This child has a metastatic neuroblastoma from the abdominal mass crossing the midline, the catecholamine secretion and the periorbital ecchymosis. The first step is to confirm the diagnosis with the urinary vanillylmandelic acid and homovanillic acid, to stage the disease with the iodine-123 metaiodobenzylguanidine scan and the magnetic resonance imaging, and to risk-stratify with the biopsy and the MYCN amplification status. [1][2]

Probe one: the staging system

The examiner presses: describe the International Neuroblastoma Risk Group staging system, and tell me what the image-defined risk factors are. [2]

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References8ShowHide
  1. [1]Matthay KK, Maris JM, Schleiermacher G, et al Neuroblastoma Nat Rev Dis Primers, 2016.PMID 27830764
  2. [2]Monclair T, Brodeur GM, Ambros PF, et al The International Neuroblastoma Risk Group (INRG) staging system: an INRG Task Force report J Clin Oncol, 2009.PMID 19047290
  3. [5]Huang M, Weiss WA Neuroblastoma and MYCN Cold Spring Harb Perspect Med, 2013.PMID 24086065
  4. [6]Du H, Cai W Opsoclonus-myoclonus syndrome associated with neuroblastoma: Insights into antitumor immunity Pediatr Blood Cancer, 2022.PMID 36094353
  5. [9]Rossor T, Yeh EA, Khakoo Y, et al Diagnosis and management of opsoclonus-myoclonus-ataxia syndrome in children: An international perspective Neurol Neuroimmunol Neuroinflamm, 2022.PMID 35260471
  6. [10]Ladenstein R, Pötschger U, Valteau-Couanet D, et al. Interleukin 2 with anti-GD2 antibody ch14.18/CHO (dinutuximab beta) in patients with high-risk neuroblastoma (HR-NBL1/SIOPEN): an open-label, randomised, phase 3 trial Lancet Oncol, 2018.PMID 30442501
  7. [11]Brodeur GM Spontaneous regression of neuroblastoma Cell Tissue Res, 2018.PMID 29305654
  8. [12]Cohn SL, Pearson AD, London WB, et al The International Neuroblastoma Risk Group (INRG) classification system: an INRG Task Force report J Clin Oncol, 2009.PMID 19047291
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