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Infants treated with diazoxide for hyperinsulinism show high prevalence of adverse events

29 Nov 2018

Pulmonary hypertension (PH) is associated with underlying risk factors in children with congenital hyperinsulinism (HI) treated with diazoxide, reports a study. Other adverse events, however, do not show any identifiable risk profile.

“The relatively high prevalence of neutropaenia, thrombocytopaenia and hyperuricaemia suggests the value in proactively screening for these side effects in children treated with diazoxide,” the authors said.

A total of 295 patients (median age at diazoxide initiation, 29 days; interquartile range, 10–142 days; 129 females; n=226 available start dates) were included, of which 2.4 percent presented with PH following diazoxide initiation.

PH (p=0.003) or oedema (p=0.002) in children was associated with earlier gestational age at birth and with potential risk factors for PH, including respiratory failure and structural heart disease (p<0.0001 and p=0.005, respectively). Other adverse events were neutropaenia (15.6 percent), hyperuricaemia (5.0 percent) and thrombocytopaenia (4.7 percent).

“Diazoxide, the only US Food and Drug Administration–approved drug to treat hyperinsulinaemic hypoglycaemia, 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,” the authors noted.

In this retrospective study, children and infants with laboratory-confirmed diagnosis of HI treated with diazoxide between 2003 and 2014 were included. The authors determined the prevalence of PH, oedema, neutropaenia, thrombocytopaenia and hyperuricaemia. They identified potential risk factors using tests of association and logistic regression.

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5 days ago

Dr Michael Lim, a senior consultant at the Paediatric Pulmonary and Sleep Division, National University Hospital, Singapore, speaks to Roshini Claire Anthony on the rare disease that is cystic fibrosis.

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