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Prevalence of Adverse Events in Children With Congenital Hyperinsulinism Treated With Diazoxide
Adriana Herrera1, Mary Ellen Vajravelu1,2, Stephanie Givler1
1Division of Endocrinology and Diabetes, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Insights
Diazoxide treatment for hyperinsulinemic hypoglycemia in children can cause adverse events like pulmonary hypertension and neutropenia. Proactive screening for side effects is recommended in pediatric patients.
Area of Science:
- Pediatric Endocrinology
- Pharmacology
- Clinical Pediatrics
Background:
- Diazoxide is the sole FDA-approved drug for hyperinsulinemic hypoglycemia (HI).
- Concerns exist regarding diazoxide's adverse events, with limited data from small studies.
- Adverse event prevalence and risk factors in pediatric HI patients require further investigation.
Purpose of the Study:
- To determine the prevalence of adverse events in infants and children treated with diazoxide.
- To identify clinical factors associated with diazoxide-induced adverse events.
- To inform safety monitoring protocols for pediatric diazoxide use.
Main Methods:
- Retrospective cohort study of 295 pediatric patients with hyperinsulinism (HI).
- Data collected between 2003 and 2014 from The Congenital Hyperinsulinism Center.
- Analysis included prevalence determination and logistic regression for risk factors of pulmonary hypertension, edema, neutropenia, thrombocytopenia, and hyperuricemia.
Main Results:
- Pulmonary hypertension (PH) occurred in 2.4% of patients, associated with earlier gestational age and risk factors like respiratory failure.
- Other adverse events included neutropenia (15.6%), thrombocytopenia (4.7%), and hyperuricemia (5.0%).
- No specific risk profile was identified for edema, neutropenia, thrombocytopenia, or hyperuricemia.
Conclusions:
- Pulmonary hypertension in diazoxide-treated infants is linked to pre-existing risk factors.
- The study highlights the need for proactive screening for neutropenia, thrombocytopenia, and hyperuricemia.
- Findings support enhanced monitoring for diazoxide-related side effects in pediatric HI management.
Context:
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.
Objective:
To determine prevalence of and clinical factors associated with adverse events in infants and children treated with diazoxide.
Design:
Retrospective cohort study of children with hyperinsulinism (HI) treated with diazoxide between 2003 and 2014.
Setting:
The Congenital Hyperinsulinism Center at the Children's Hospital of Philadelphia.
Patients:
Children and infants with laboratory-confirmed diagnosis of HI.
Main Outcome Measures:
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.
Results:
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 < 0.0001 and P = 0.005). Other adverse events included neutropenia (15.6%), thrombocytopenia (4.7%), and hyperuricemia (5.0%).
Conclusion:
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.
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