The Journal of Pediatrics
Volume 158, Issue 2 , Pages 337-339, February 2011

Cyclophosphamide for Rapid-Onset Obesity, Hypothalamic Dysfunction, Hypoventilation, and Autonomic Dysregulation Syndrome

  • Ido Paz-Priel, MD

      Affiliations

    • Division of Pediatric Oncology, Johns Hopkins University, Baltimore, MD
    • Corresponding Author InformationReprint requests: Ido Paz-Priel, MD, Bunting-Blaustein Cancer Research Building I, Room 208, 1650 Orleans Street, Baltimore, MD 21231.
    • ,
  • David W. Cooke, MD

      Affiliations

    • Division of Pediatric Endocrinology, Johns Hopkins University, Baltimore, MD
    • ,
  • Allen R. Chen, MD, PhD

      Affiliations

    • Division of Pediatric Oncology, Johns Hopkins University, Baltimore, MD

Received 8 February 2010; received in revised form 14 June 2010; accepted 9 July 2010. published online 23 August 2010.

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.

OMA, Opsoclonus myoclonus ataxia, ROHHAD, Rapid onset of obesity, hypothalamic dysfunction, hypoventilation, autonomic dysregulation

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 I.P.-P. is a St. Baldrick's Foundation Scholar. The authors declare no conflicts of interest.

PII: S0022-3476(10)00584-6

doi:10.1016/j.jpeds.2010.07.006

The Journal of Pediatrics
Volume 158, Issue 2 , Pages 337-339, February 2011

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