Therapeutic efficacy analysis of diazoxide in treating 145 cases of congenital hyperinsulinism

Objective To analyze the efficacy and safety of diazoxide treatment in patients with congenital hyperinsulinism(CHI). Methods The clinical data of 145 patients diagnosed with CHI hospitalized in Beijing Children′s Hospital affiliated to Capital Medical University from February 2002 to January 2016 who received diazoxide treatment were retrospectively analyzed. We conducted a detailed analysis on the efficacy, side effects and prognosis of diazoxide treatment for CHI. Results In 145 patients, there were 89 patients (61.4%) who were responsive to diazoxide and 23 patients (15.9%) unresponsive to diazoxide, and the treatment effect of diazoxide for the other 33 cases (22.8%) was still unclear. In the diazoxide effective group, there were 22 cases (24.7%) of neonatal onset, 32 cases (36.0%)of onset from 1-6 months after birth and 35 cases(39.3%)of onset after 6 months of birth, and the birth weight was normal in 67 cases, macrosomia in 16 cases, low in 5 cases and unknown in 1 case. In the diazoxide ineffective group, there were 14 cases (60.8%) of neonatal onset, 7 cases (30.4%) of onset from 1-6 months after birth and 2 cases(8.6%)of onset after 6 months of birth, and the birth weight was normal in 9 cases and macrosomia in 14 cases. In the unclear diazoxide effect group, there were 20 cases (60.6%) of neonatal onset, 9 cases (27.34%) of onset from 1-6 months after birth and 4 cases(12.1%)of onset after 6 months of birth, and the birth weight was normal in 15 cases and macrosomia in 18 cases. After the application of diazoxide, 65 cases (44.8%) had hirsutism, and 43 patients (29.7%) had gastrointestinal side effects. In the diazoxide effective group, the symptom of hypoglycemia was spontaneously alleviated at the age of from 3 months to 7 years old in 20 patients (22.5%), and 33 patients continued to be treated by diazoxide. In the diazoxide ineffective group, the symptom of hypoglycemia was spontaneously alleviated around the age of 1 years old in 4 patients (17.3%), 5 patients were treated by octreotide in long term to maintain normal blood sugar level, 4 patients received pancreectomy including 3 with normal blood sugar and 1 with occasional hypoglycemia after surgery. In the unclear diazoxide effect group, the symptom of hypoglycemia was spontaneously alleviated at the age of from 10 months to 3 years old in 4 patients (12.1%), 2 patients were treated by octreotide in long term to maintain normal blood sugar level, 8 patients received pancreatectomy including 5 with controlled blood sugar after surgery. Conclusions Diazoxide is effective in treating CHI children. The efficiency may be higher for the CHI with normal birth weight or whose onset age is after the neonatal period. Key words: Congenital hyperinsulinism; Diazoxide; Remission, spontaneous; Treatment outcome