Cyclophosphamide for rapid-onset obesity, hypothalamic dysfunction, hypoventilation, and autonomic dysregulation syndrome

Ido Paz-Priel, David W Cooke, Allen R Chen

Research output: Contribution to journalArticle

Abstract

Patients with rapid-onset obesity, hypothalamic dysfunction, hypoventilation, autonomic dysregulation, and neural crest tumor syndrome have poor long-term outcomes. We report a patient who was treated successfully with high-dose cyclophosphamide immunoablation. This experience offers a novel therapeutic approach and an indirect insight into the underlying pathogenesis of this syndrome.

Original languageEnglish (US)
Pages (from-to)337-339
Number of pages3
JournalJournal of Pediatrics
Volume158
Issue number2
DOIs
StatePublished - Feb 2011

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Hypoventilation
Cyclophosphamide
Obesity
Therapeutics
Neural crest tumor

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

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abstract = "Patients with rapid-onset obesity, hypothalamic dysfunction, hypoventilation, autonomic dysregulation, and neural crest tumor syndrome have poor long-term outcomes. We report a patient who was treated successfully with high-dose cyclophosphamide immunoablation. This experience offers a novel therapeutic approach and an indirect insight into the underlying pathogenesis of this syndrome.",
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