History and exam

Key diagnostic factors

common

presence of risk factors

Patients may have a family history of neuroblastic tumours or neurocristopathies.

age <4 years

The majority of neuroblastoma cases occur in children ≤4 years of age (34% in children <1 year; 49.9% in those aged 1-4 years).[12] 

Median age at diagnosis is 17 months.[12] 

Infants typically present with an asymptomatic adrenal mass detected on routine prenatal ultrasound. Patients with localised disease may also be asymptomatic.

abdominal distention

Most common symptom, caused by the presence of a large abdominal mass.[29][30]​​

abdominal mass

May be felt on palpation if the primary site is the abdomen.

In the UK, the National Institute for Health and Care Excellence (NICE) recommend very urgent referral (an appointment within 48 hours) for specialist assessment for neuroblastoma in children with a palpable abdominal mass or an unexplained enlarged abdominal organ.[31]

pain

Abdominal pain is common if the primary site is the abdomen.

Bone pain is common in patients with metastatic disease. Back pain may indicate spinal disease. The presence of neurological signs and symptoms may indicate spinal cord compression.

decreased appetite

Patients commonly present with general systemic symptoms which may indicate metastatic spread.

weight loss

Patients commonly present with general systemic symptoms which may indicate metastatic spread.

fussiness (infants)

Patients commonly present with general systemic symptoms which may indicate metastatic spread.

fatigue

Patients commonly present with general systemic symptoms which may indicate metastatic spread.

periorbital ecchymosis (panda eyes)

Common in patients with orbital metastases.

subcutaneous skin nodules

Palpable, non-tender, non-erythematous skin nodules are common in infants, and are associated with metastatic spread.[35][36]​​

uncommon

family history of neuroblastic tumours

Approximately 1% to 2% of patients have a family history of neuroblastoma.[13] 

Most hereditary neuroblastomas are caused by alterations in the ALK (anaplastic lymphoma kinase) gene.[14] Germline PHOX2B (paired-like homeobox 2b) mutations are a relatively rare cause of hereditary neuroblastoma.[15][16]

signs of spinal cord compression

If the tumour extends into the spinal canal, patients may initially present with signs of spinal cord compression including loss of bowel and bladder function, lower-extremity weakness, and back pain.

signs of Horner's syndrome

If the primary lesion is in the upper portion of the thoracic outlet or cervical sympathetic chain, patients will often present with Horner's syndrome (characterised by ptosis, miosis, anhidrosis) due to sympathetic nerve compression.[1][5][6]

signs of superior vena cava syndrome

If the primary lesion is in the upper portion of the thoracic outlet or cervical sympathetic chain, patients will often present with superior vena cava syndrome (characterised by dyspnoea, facial swelling, cough, distended neck/chest veins, oedema of the upper extremities) due to mass effect on the vascular system.[1]​ 

intractable secretory diarrhoea

May occur in patients with tumour secretion of vasoactive intestinal protein (VIP), a paraneoplastic syndrome associated with neuroblastoma.[9]

opsoclonus-myoclonus ataxia (OMA)

Paraneoplastic syndrome is sometimes associated with neuroblastoma. OMA (also known as opsoclonus-myoclonus syndrome) occurs in <3% of patients with neuroblastoma.[33] 

In patients with OMA, nearly one half will be diagnosed with neuroblastoma; therefore, all children with OMA should be evaluated for neuroblastoma.[34] Rapid, dancing eye movements, rhythmic jerking of limbs/trunk, and ataxia are pathognomonic for OMA.[8] 

Other diagnostic factors

common

constipation

Common presenting symptom. Usually secondary to mass effect of abdominal tumour.

uncommon

pallor

May indicate metastatic spread to bone marrow.

bleeding

May indicate metastatic spread to bone marrow.

infections

May indicate metastatic spread to bone marrow.

hypertension

Caused by mass effect of abdominal tumour, which may compromise renal vasculature resulting in alterations of the renin-activating system pathway, or may be a result of tumour-associated catecholamine secretion.​[1]

hepatomegaly

May indicate metastatic spread to the liver.

Severe hepatomegaly secondary to liver metastases may be present in patients <18 months with stage MS disease (International Neuroblastoma Risk Group Staging System).[2][32]

Risk factors

weak

neurocristopathies or other syndromes

A few medical conditions, some of which are related to aberrant neural crest development, have been shown to predispose a patient to developing neuroblastoma, including Turner syndrome, Hirschsprung's disease, congenital central hypoventilation syndrome, and neurofibromatosis type 1.[17][18]​ Given that neuroblastoma arises from the sympathetic nervous system, the rationale that it may be associated with other disorders of the neural crest is biologically sound.

family history of neuroblastic tumours or genetic predisposition

Approximately 1% to 2% of patients have a family history of neuroblastoma.[13]

Most hereditary neuroblastomas are caused by alterations in the ALK (anaplastic lymphoma kinase) gene.[14]​ Germline PHOX2B (paired-like homeobox 2b) mutations are a relatively rare cause of hereditary neuroblastoma.[15][16]

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