Pajno R, Visconti C, Bucolo C, Guarneri MP, Del Barba P, Silvani P, Gregnanin M, Barera G. Diazoxide toxicity in congenital hyperinsulinism: A case report. World J Clin Pediatr 2024; 13(4): 94156 [DOI: 10.5409/wjcp.v13.i4.94156]
Corresponding Author of This Article
Roberta Pajno, MD, Attending Doctor, Department of Pediatrics, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Institute, Via Olgettina 60, Milan 20132, Lombardy, Italy. pajno.roberta@hsr.it
Research Domain of This Article
Pediatrics
Article-Type of This Article
Case Report
Open-Access Policy of This Article
This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
World J Clin Pediatr. Dec 9, 2024; 13(4): 94156 Published online Dec 9, 2024. doi: 10.5409/wjcp.v13.i4.94156
Diazoxide toxicity in congenital hyperinsulinism: A case report
Roberta Pajno, Camilla Visconti, Carmen Bucolo, Maria Pia Guarneri, Paolo Del Barba, Paolo Silvani, Marco Gregnanin, Graziano Barera
Roberta Pajno, Camilla Visconti, Carmen Bucolo, Maria Pia Guarneri, Paolo Del Barba, Graziano Barera, Department of Pediatrics, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Institute, Milan 20132, Lombardy, Italy
Camilla Visconti, Università Vita-Salute San Raffaele, Facoltà di Medicina e Chirurgia, Milan 20132, Lombardy, Italy
Paolo Silvani, Department of Anesthesia and Intensive Care, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan 20132, Lombardy, Italy
Marco Gregnanin, Graziano Barera, Department of Neonatology, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Institute, Milan 20132, Lombardy, Italy
Author contributions: Pajno R conduct writing original draft; Visconti C conduct data collection; Bucolo C conduct conceptualization of the case report; Guarneri MP, Barera G and Silvani P proceed manuscript review; Barba PD and Gregnanin M proceed manuscript editing.
Informed consent statement: The authors obtained informed consent from both caregivers for the publication of the images and clinical data contained herein prior to study enrollment.
Conflict-of-interest statement: All the authors declare that they have no competing interests.
CARE Checklist (2016) statement: The authors have read the CARE Checklist (2016), and the manuscript was prepared and revised according to the CARE Checklist (2016).
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Roberta Pajno, MD, Attending Doctor, Department of Pediatrics, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Institute, Via Olgettina 60, Milan 20132, Lombardy, Italy. pajno.roberta@hsr.it
Received: March 12, 2024 Revised: June 6, 2024 Accepted: July 2, 2024 Published online: December 9, 2024 Processing time: 231 Days and 21.2 Hours
Abstract
BACKGROUND
Diazoxide is the sole approved drug for congenital hyperinsulinism; however, diuretic administration and vigilant monitoring are crucial to prevent and promptly identify potentially life-threatening adverse effects. This report aims to highlight a seldom-considered rare side effect of diazoxide. We believe that this brief report is of general interest to World Journal of Clinical Pediatric readership and increase the physicians’ awareness of the guideline importance. Moreover, it underlines the importance of stopping immediately the drug if suspected side effects.
CASE SUMMARY
The manuscript describes a patient diagnosed with congenital hyperinsulinism (CHI) treated with diazoxide not overlapping with diuretic. He resulted in sudden respiratory distress and therefore was transferred to the Neonatal Intensive Care Unit. The cardiological evaluation showed pericardial effusion and left ventricular myocardial hypertrophy, absent before. In suspicion of an iatrogenic effect of diazoxide it was progressively reduced until stop while introducing diuretic treatment, with resolution of symptoms. Once clinically stabilized, an 18 fluoro-diydroxy-phenylalanine positron emission tomography/computed tomography (PET/CT) was performed to differentiate between a focal or diffuse form of CHI. The PET/CT highlighted the presence of a single focal accumulation of the tracer located in the pancreatic tail, consistent with a focal form of hyperinsulinism. At the age of four months, the patient underwent a distal pancreatectomy with histological confirmation of a focal form of nesidioblastosis, resulting in a curative operation.
CONCLUSION
Diuretic administration and vigilant monitoring of diazoxide therapy are crucial to prevent and promptly identify potentially life-threatening adverse effects.
Core Tip: Diazoxide can be effective in treating congenital hyperinsulinism, but its use requires diuretic administration and careful surveillance to prevent and promptly diagnose potential life-threatening adverse effects. Although diazoxide cardiopulmonary side effects were previously described in sporadic case reports, the United States Food and Drug Administration (FDA) has clearly listed them as severe adverse events since 2015. Despite this, numerous reports were published after the FDA statement, describing the increasing rate of these diazoxide side effects. Further studies are needed to identify the underlying mechanism and precise predisposing factors.
