Weight management in youth with rapid-onset obesity with hypothalamic dysregulation, hypoventilation, autonomic dysregulation, and neural crest tumor (ROHHAD-NET): literature search and case report

Ian Marpuri; Esther Ra; Monica N. Naguib; Alaina P. Vidmar

doi:10.1515/jpem-2021-0600

Article

Weight management in youth with rapid-onset obesity with hypothalamic dysregulation, hypoventilation, autonomic dysregulation, and neural crest tumor (ROHHAD-NET): literature search and case report

Ian Marpuri , Esther Ra , Monica N. Naguib and Alaina P. Vidmar

Published/Copyright: December 23, 2021

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From the journal Journal of Pediatric Endocrinology and Metabolism Volume 35 Issue 4

Abstract

Objectives

Rapid-onset obesity with hypothalamic dysfunction, hypoventilation, autonomic dysregulation, and neural endocrine tumor (ROHHAD-NET) syndrome is a youth-onset constellation of symptoms including rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation. Despite growing understanding of the clinical classification of this syndrome there is limited investigation into treatment of the rapid-onset obesity which can be progressive and life-limiting. The purpose of this case report is to describe the clinical timeline and treatment of severe obesity in a patient with of ROHHAD-NET and propose recommendations for the treatment of associated obesity.

Case presentation

We present the case of a 10-year-old female with a clinical presentation consistent with ROHHAD-NET who achieved clinically meaningful weight loss with a combination of lifestyle modification and anti-obesity pharmacotherapies. We report on the use of three separate pharmacological agents and ultimately the referral for bariatric surgery.

Conclusions

Given that early-onset obesity and hypoventilation are life-limiting components of this condition, early recognition and treatment are essential to improve health outcomes.

Keywords: anti-obesity medications; early-onset obesity; rapid-onset obesity with hypothalamic dysfunction, hypoventilation, autonomic dysregulation, and neural endocrine tumor (ROHHAD-NET) syndrome

Corresponding author: Alaina Vidmar, MD, Department of Pediatrics, Center for Diabetes, Endocrinology and Metabolism, Children’s Hospital Los Angeles, 4650 Sunset Boulevard, Mailstop #61, Los Angeles, CA 90027, USA; and Keck School of Medicine of USC, Los Angeles, CA, USA, Phone: +1 (323) 361 3385, E-mail: avidmar@chla.usc.edu

Funding source: NIH/NCRR SC-CTSI

Award Identifier / Grant number: UL1 TR000130

Acknowledgments

The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. Dexcom supported this study by providing the continuous glucose monitor equipment required.

Research funding: This work was supported in part by grants 1) NIH/NCRR SC-CTSI, Grant Number: UL1 TR000130.
Author contributions: Conceptualization, I.M, E.R, M.N, A.P.V.; methodology, I.M, E.R, M.N, A.P.V.; formal analysis, I.M, E.R, M.N, A.P.V.; data curation, I.M, E.R, M.N, A.P.V; writing—original draft preparation, A.P.V., M.N.; writing—review and editing, I.M, E.R, M.N, A.P.V.; supervision, A.P.V. All authors have read and agreed to the published version of the manuscript. All authors have accepted responsibility for the entire content of this manuscript and approved its submission.
Competing interests: The authors have no financial relationships or conflict of interest relevant to this article to disclose. The funders had no role in the design of the study; in the collection, analyses, or interpretation of data; in the writing of the manuscript, or in the decision to publish the results. Authors state no conflict of interest.
Informed consent: Written informed consent was obtained from all participants and a parent or guardian involved in the study.
Ethical approval: The study was conducted according to the guidelines of the Declaration of Helsinki and approved by the Institutional Review Board (or Ethics Committee) of Children’s Hospital Los Angeles (CHLA-000243, date of approval – 12/20/2019).
Data availability statement: The datasets from this study will be available from the corresponding author on written request.

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Received: 2021-09-21

Accepted: 2021-12-06

Published Online: 2021-12-23

Published in Print: 2022-04-26

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https://doi.org/10.1515/jpem-2021-0600

Keywords for this article

anti-obesity medications; early-onset obesity; rapid-onset obesity with hypothalamic dysfunction, hypoventilation, autonomic dysregulation, and neural endocrine tumor (ROHHAD-NET) syndrome