First name
Adriana
Last name
Herrera

Title

Prevalence of Adverse Events in Children With Congenital Hyperinsulinism Treated With Diazoxide.

Year of Publication

2018

Number of Pages

4365-4372

Date Published

2018 Dec 01

ISSN Number

1945-7197

Abstract

<p><strong>Context: </strong>Diazoxide, the only U.S. Food and Drug Administration-approved drug to treat hyperinsulinemic hypoglycemia, has been associated with several adverse events, which has raised concerns about the safety of this drug. Existing reports are limited to small studies and case reports.</p>

<p><strong>Objective: </strong>To determine prevalence of and clinical factors associated with adverse events in infants and children treated with diazoxide.</p>

<p><strong>Design: </strong>Retrospective cohort study of children with hyperinsulinism (HI) treated with diazoxide between 2003 and 2014.</p>

<p><strong>Setting: </strong>The Congenital Hyperinsulinism Center at the Children's Hospital of Philadelphia.</p>

<p><strong>Patients: </strong>Children and infants with laboratory-confirmed diagnosis of HI.</p>

<p><strong>Main Outcome Measures: </strong>Prevalence of pulmonary hypertension (PH), edema, neutropenia, thrombocytopenia, and hyperuricemia was determined. Tests of association and logistic regression were used to identify potential risk factors.</p>

<p><strong>Results: </strong>A total of 295 patients (129 female) met inclusion criteria. The median age at diazoxide initiation was 29 days (interquartile range, 10 to 142 days; n = 226 available start dates); 2.4% of patients were diagnosed with PH after diazoxide initiation. Children with PH (P = 0.003) or edema (P = 0.002) were born at earlier gestational age and more frequently had potential PH risk factors, including respiratory failure and structural heart disease (P &lt; 0.0001 and P = 0.005). Other adverse events included neutropenia (15.6%), thrombocytopenia (4.7%), and hyperuricemia (5.0%).</p>

<p><strong>Conclusion: </strong>In this large cohort, PH occurred in infants with underlying risk factors, but no identifiable risk profile emerged for other adverse events. The relatively high prevalence of neutropenia, thrombocytopenia, and hyperuricemia suggests the value in proactively screening for these side effects in children treated with diazoxide.</p>

DOI

10.1210/jc.2018-01613

Alternate Title

J. Clin. Endocrinol. Metab.

PMID

30247666

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