ArticleTranslational pediatrics2025
Rapid-onset obesity, hypothalamic dysfunction, hypoventilation, and autonomic dysregulation as a paraneoplastic syndrome in neuroblastoma: case report.
Article in Translational pediatrics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Neuroblastoma, the most common extracranial solid tumour in childhood, is occasionally associated with paraneoplastic syndromes. These syndromes can occur before tumour diagnosis or recurrence. Notably, approximately 40% of patients with rapid-onset obesity, hypothalamic dysfunction, hypoventilation, and autonomic dysregulation (ROHHAD) have underlying neural crest tumours, such as ganglioneuromas or ganglioneuroblastomas. ROHHAD may also manifest as a paraneoplastic syndrome in neuroblastomas. Here, we describe three neuroblastoma cases with ROHHAD to help improve oncologists' recognition of ROHHAD as a paraneoplastic entity. Case Description: The three neuroblastoma cases (two females, one male) were diagnosed at 102, 44, and 9 months of age, respectively. The histopathological subtypes included ganglioneuroblastomas (n=2) and a neuroblastoma (n=1). Rapid-onset obesity occurred at 95 months (7.9 years), 33 months (2.8 years), and 72 months (6 years) of age. All three children underwent resection of their primary tumour, with one receiving adjuvant chemotherapy. For the treatment of ROHHAD, case 1 received immunomodulatory therapy comprising glucocorticoids, intravenous immunoglobulin (IVIG), and rituximab; case 2 did not receive immunotherapy; and case 3 was treated with oral prednisone therapy. All cases demonstrated sustained survival at last follow-up. Conclusions: ROHHAD syndrome may manifest as a paraneoplastic phenomenon in neuroblastoma patients. Oncologists should suspect ROHHAD in children with neuroblastoma who develop rapid-onset obesity, even in the absence of classic features such as hypoventilation or hypothalamic endocrine dysregulation. Implementing a multidisciplinary collaborative approach to care will enable early recognition and intervention. This will critically impact patient prognosis by facilitating prompt tumour surveillance while significantly improving long-term survival rates.
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