Cyclophosphamide for rapid-onset obesity, hypothalamic dysfunction, hypoventilation, and autonomic dysregulation syndrome.
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.
Paz-Priel
Ido
I
Division of Pediatric Oncology, Johns Hopkins University, Baltimore, MD 21231, USA. ipazpri1@jhmi.edu
Cooke
David W
DW
Chen
Allen R
AR
D000208:Acute Disease; D000310:Adrenal Gland Neoplasms / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy; D001342:Autonomic Nervous System Diseases / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007027:Hypothalamic Diseases / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D009765:Obesity / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D010845:Obesity Hypoventilation Syndrome / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D020551:Pulse Therapy, Drug; D012720:Severity of Illness Index; D013577:Syndrome; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D003520:Cyclophosphamide